UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
WASHINGTON, DCD.C. 20549
FORM 10-Q
(Mark One)
☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934
☒ | QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 |
For the quarterly period ended June 30, 20222023
OR
☐ |
|
For the transition period from _______________________________________ to _______________________________________
Commission File Number: 001-39438
GEMINI THERAPEUTICS, INC.Disc Medicine, Inc.
(Exact Namename of Registrantregistrant as Specifiedspecified in its Charter)charter)
Delaware | 85-1612845 |
(State or other jurisdiction of incorporation or organization) | (I.R.S. Employer
|
Watertown, |
|
(Address of principal executive offices) | (Zip Code) |
Registrant’s telephone number, including area code: (617) 401-4400674-9274
(Registrant’s telephone number, including area code)
Securities registered pursuant to Section 12(b) of the Act:
Title of each class | Trading Symbol(s) | Name of each exchange on which registered | ||
Common |
| The Nasdaq Global Market |
Indicate by check mark whether the registrant (1) has filed all reports required to be filed by Section 13 or 15(d) of the Securities Exchange Act of 1934 during the preceding 12 months (or for such shorter period that the registrant was required to file such reports), and (2) has been subject to such filing requirements for the past 90 days. Yes ☒No ☐
Indicate by check mark whether the registrant has submitted electronically every Interactive Data File required to be submitted pursuant to Rule 405 of Regulation S-T (§232.405 of this chapter) during the preceding 12 months (or for such shorter period that the registrant was required to submit such files). Yes ☒No ☐
Indicate by check mark whether the registrant is a large accelerated filer, an accelerated filer, a non-accelerated filer, a smaller reporting company, or an emerging growth company. See the definitions of “large accelerated filer,” “accelerated filer,” “smaller reporting company,”company” and “emerging growth company” in Rule 12b-2 of the Exchange Act.
Large accelerated filer | ☐ | Accelerated filer | ☐ | |||
Non-accelerated filer | ☒ | Smaller reporting company | ☒ | |||
Emerging growth company | ☒ | |||||
If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐
Indicate by check mark whether the registrant is a shell company (as defined in Rule 12b-2 of the Exchange Act). Yes ☐No ☒
As of August 5, 2022, the registrant had8, 2023, there were 43,244,45322,877,552 shares of common stock,Common Stock, $0.0001 par value per share, outstanding.
Table of Contents
Page | ||
1 | ||
3 | ||
PART I. | 5 | |
Item 1. | 5 | |
5 | ||
Condensed Consolidated Statements of Operations and Comprehensive Loss | 6 | |
Condensed Consolidated Statements of Preferred Stock and Stockholders’ Equity (Deficit) | 7 | |
9 | ||
Notes to Unaudited Condensed Consolidated Financial Statements | 10 | |
Item 2. | Management’s Discussion and Analysis of Financial Condition and Results of Operations | 22 |
Item 3. | 31 | |
Item 4. | 31 | |
PART II. | 32 | |
Item 1. | 32 | |
Item 1A. | 32 | |
Item 2. | 80 | |
Item 3. | 80 | |
Item 4. | 80 | |
Item 5. | 80 | |
Item 6. | 81 | |
82 | ||
i
RISK FACTOR SUMMARY
The risk factors detailed in Item 1A entitled “Risk Factors” in this Quarterly Report on Form 10-Q are the risks that we believe are material to our investors and a reader should carefully consider them. Those risks are not all of the risks we face and other factors not presently known to us or that we currently believe are immaterial may also affect our business if they occur. The following is a summary of the risk factors detailed in Item 1A:
1
This section contains forward-looking statements. You should refer to the explanation of the qualifications and limitations on forward-looking statements under the caption “Forward Looking Statements” of this Quarterly Report on Form 10-Q.
2
CAUTIONARY NOTE REGARDING FORWARD-LOOKINGFORWARD LOOKING STATEMENTS
This Quarterly Report on Form 10-Q, or Quarterly Report, of Disc Medicine, Inc., or the Company, contains or incorporates statements that constitute forward-looking statements within the meaning of the federal securities laws. Our forward-looking statements include, but are not limited to, statements regarding our or our management team’s expectations, hopes, beliefs, intentions or strategies regarding the future. In addition, any statements that involverefer to projections, forecasts or other characterizations of future events or circumstances, including any underlying assumptions, are forward-looking statements. The words “anticipate,” “believe,” “contemplate,” “continue,” “could,” “estimate,” “expect,” “intends,” “may,” “might,” “plan,” “possible,” “potential,” “predict,” “project,” “should,” “will,” “would” and similar expressions may identify forward-looking statements, but the absence of these words does not mean that a statement is not forward-looking. Forward-looking statements in this Quarterly Report on Form 10-Q may include, for example, statements about:
3
These forward-looking statements are based on information available as ofto us at the datetime of this reportQuarterly Report on Form 10-Q and our management’s current expectations, forecasts and assumptions, and involve a number of judgments, risks and uncertainties. Accordingly, forward-looking statements should not be relied upon as representing our views as of any subsequent date. Wedate, and we do not undertake any obligation to update forward-looking statements to reflect events or circumstances after the date they were made, whether as a result of new information, future events or otherwise, except as may be required under applicable securities laws. Specifically,
The outcome of the events described in these forward-looking statements may include, but are not limitedis subject to statements relating to or about:
i
Other statements preceded by, followed by or that include the words “may,” “can,” “should,” “will,” “estimate,” “plan,” “project,” “forecast,” “intend,” “expect,” “anticipate,” “believe,” “seek,” “target” or similar expressions, or the negative of these terms, are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words.
Any or all forward-looking statements may turn out to be incorrect. Because forward-looking statements are inherently subject to risks, uncertainties, and uncertainties, some of which cannot be predicted or quantified, you should not rely on these forward-looking statements as predictions of future events.other factors. As a result of a number of known and unknown risks and uncertainties, our actual results or performance may be materially different from those expressed or implied by these forward-looking statements. Some factors that could cause actual results to differ include:
ii4
Table of Contents
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iii
PART I—FINANCIAL INFORMATION
ItemITEM 1. Financial Statements.FINANCIAL STATEMENTS
Gemini Therapeutics, Inc.DISC MEDICINE, INC.
Condensed Consolidated Balance SheetsCONDENSED CONSOLIDATED BALANCE SHEETS
(In thousands, except share and per share amounts)
(Unaudited)
| June 30, |
|
| December 31, |
| |||
| 2023 |
|
| 2022 |
| |||
Assets | |
|
|
|
|
| ||
Current assets: | |
|
|
|
|
| ||
Cash and cash equivalents | | $ | 377,602 |
|
| $ | 194,611 |
|
Prepaid expenses and other current assets | |
| 4,131 |
|
|
| 3,880 |
|
Total current assets | |
| 381,733 |
|
|
| 198,491 |
|
Property and equipment, net | |
| 159 |
|
|
| 168 |
|
Right-of-use assets, operating leases | |
| 2,166 |
|
|
| 1,430 |
|
Other assets | |
| 116 |
|
|
| 116 |
|
Total assets | | $ | 384,174 |
|
| $ | 200,205 |
|
Liabilities and Stockholders’ Equity | |
|
|
|
|
| ||
Current liabilities: | |
|
|
|
|
| ||
Accounts payable | | $ | 5,214 |
|
| $ | 16,162 |
|
Accrued expenses | |
| 4,148 |
|
|
| 6,109 |
|
Operating lease liabilities, current | |
| 321 |
|
|
| 307 |
|
Total current liabilities | |
| 9,683 |
|
|
| 22,578 |
|
Operating lease liabilities, non-current | |
| 1,772 |
|
|
| 1,027 |
|
Contingent value right liability | |
| 1,500 |
|
|
| — |
|
Total liabilities | |
| 12,955 |
|
|
| 23,605 |
|
Commitments and contingencies (Note 14) | |
|
|
|
|
| ||
Stockholders’ equity: | |
|
|
|
|
| ||
Common stock, $0.0001 par value; 100,000,000 shares authorized as of June 30, | |
| 2 |
|
|
| 2 |
|
Additional paid-in capital | |
| 522,161 |
|
|
| 288,814 |
|
Accumulated deficit | |
| (150,944 | ) |
|
| (112,216 | ) |
Total stockholders’ equity | |
| 371,219 |
|
|
| 176,600 |
|
Total liabilities and stockholders’ equity | | $ | 384,174 |
|
| $ | 200,205 |
|
The accompanying notes are an integral part of these condensed consolidated financial statements.
5
DISC MEDICINE, INC.
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS
(In thousands, except share and per share amounts)
(Unaudited)
| Three Months Ended |
| | Six Months Ended |
| |||||||||||
| 2023 |
| | 2022 |
| | 2023 |
| | 2022 |
| |||||
Operating expenses: | |
|
| |
|
| |
|
| |
|
| ||||
Research and development | | $ | 12,100 |
| | $ | 7,714 |
| | $ | 32,280 |
| | $ | 15,535 |
|
General and administrative | |
| 5,228 |
| |
| 4,301 |
| |
| 10,173 |
| |
| 6,440 |
|
Total operating expenses | |
| 17,328 |
| |
| 12,015 |
| |
| 42,453 |
| |
| 21,975 |
|
Loss from operations | |
| (17,328 | ) | |
| (12,015 | ) | |
| (42,453 | ) | |
| (21,975 | ) |
Other income (expense), net: | |
|
| |
|
| |
|
| |
|
| ||||
Interest income | |
| 2,910 |
| |
| 45 |
| |
| 5,277 |
| |
| 52 |
|
Change in fair value of contingent value right liability | |
| (1,500 | ) | |
| — |
| |
| (1,500 | ) | |
| — |
|
Change in fair value of derivative liability | |
| — |
| |
| 2,390 |
| |
| — |
| |
| 2,490 |
|
Other expense | |
| (5 | ) | |
| — |
| |
| (5 | ) | |
| — |
|
Total other income (expense), net | |
| 1,405 |
| |
| 2,435 |
| |
| 3,772 |
| |
| 2,542 |
|
Loss before income taxes | |
| (15,923 | ) | |
| (9,580 | ) | |
| (38,681 | ) | |
| (19,433 | ) |
Income tax expense |
|
| (24 | ) |
|
| — |
|
|
| (47 | ) |
|
| — |
|
Net loss and comprehensive loss | | $ | (15,947 | ) | | $ | (9,580 | ) | | $ | (38,728 | ) | | $ | (19,433 | ) |
Net loss attributable to common stockholders-basic and diluted | | $ | (15,947 | ) | | $ | (9,580 | ) | | $ | (38,728 | ) | | $ | (19,433 | ) |
Weighted-average common shares outstanding-basic and diluted | |
| 21,484,955 |
| |
| 944,706 |
| |
| 20,226,923 |
| |
| 934,286 |
|
Net loss per share attributable to common stockholders-basic and diluted | | $ | (0.74 | ) | | $ | (10.14 | ) | | $ | (1.91 | ) | | $ | (20.80 | ) |
The accompanying notes are an integral part of these condensed consolidated financial statements.
6
DISC MEDICINE, INC.
CONDENSED CONSOLIDATED STATEMENTS OF CONVERTIBLE PREFERRED STOCK
AND STOCKHOLDERS’ EQUITY (DEFICIT)
(In thousands, except share and per share amounts)
(Unaudited)
|
| June 30, |
|
| December 31, |
| ||
Assets |
|
|
|
|
|
| ||
Current assets: |
|
|
|
|
|
| ||
Cash and cash equivalents |
| $ | 108,659 |
|
| $ | 136,627 |
|
Restricted cash, current |
|
| 0 |
|
|
| 223 |
|
Prepaid expenses and other current assets |
|
| 4,470 |
|
|
| 3,250 |
|
Total current assets |
|
| 113,129 |
|
|
| 140,100 |
|
|
|
|
|
|
|
| ||
Restricted cash, non-current |
|
| 0 |
|
|
| 100 |
|
Other assets |
|
| 204 |
|
|
| 237 |
|
Total assets |
| $ | 113,333 |
|
| $ | 140,437 |
|
|
|
|
|
|
|
| ||
Liabilities and stockholders' equity |
|
|
|
|
|
| ||
Current liabilities: |
|
|
|
|
|
| ||
Accounts payable |
| $ | 1,618 |
|
| $ | 2,950 |
|
Accrued expenses and other current liabilities |
|
| 1,905 |
|
|
| 6,884 |
|
Term loan, current portion |
|
| 2,913 |
|
|
| 5,000 |
|
Total current liabilities |
|
| 6,436 |
|
|
| 14,834 |
|
|
|
|
|
|
|
| ||
Other liabilities |
|
| 0 |
|
|
| 358 |
|
Term loan, net of current portion and discount |
|
| 0 |
|
|
| 404 |
|
Total liabilities |
|
| 6,436 |
|
|
| 15,596 |
|
|
|
|
|
|
|
| ||
Stockholders' equity: |
|
|
|
|
|
| ||
Preferred stock, $0.0001 par value; 10,000,000 shares authorized; 0 shares issued |
|
| 0 |
|
|
| 0 |
|
Common stock, $0.0001 par value; 250,000,000 shares authorized; 43,244,453 and |
|
| 4 |
|
|
| 4 |
|
Additional paid-in capital |
|
| 313,904 |
|
|
| 309,527 |
|
Accumulated deficit |
|
| (207,011 | ) |
|
| (184,690 | ) |
Total stockholders' equity |
|
| 106,897 |
|
|
| 124,841 |
|
|
|
|
|
|
|
| ||
Total liabilities and stockholders' equity |
| $ | 113,333 |
|
| $ | 140,437 |
|
7
| Convertible Preferred Stock |
| |
|
|
| |
|
| |
|
| |
|
| |
|
| |||||||||||||||||||||||||||
| Series Seed |
|
| Series A |
| | Series B |
| |
| Common Stock |
| | Additional |
| | Accumulated |
| | Total |
| ||||||||||||||||||||||||
| Shares |
| | Amount |
| | Shares |
| | Amount |
| | Shares |
| | Amount |
| |
| Shares |
| | Amount |
| | Capital |
|
| Deficit |
|
| Deficit |
| ||||||||||||
Balance at December 31, 2021 | |
| 5,000,000 |
| | $ | 2,350 |
| |
| 41,666,666 |
| | $ | 49,762 |
| |
| 37,499,999 |
| | $ | 89,744 |
| |
|
| 909,418 |
| | $ | 1 |
| | $ | 1,185 |
| | $ | (65,389 | ) | | $ | (64,203 | ) |
Issuance of common stock | |
| — |
| |
| — |
| |
| — |
| |
| — |
| |
| — |
| |
| — |
| |
|
| 31,725 |
| |
| — |
| |
| 55 |
| |
| — |
| |
| 55 |
|
Vesting of restricted | |
| — |
| |
| — |
| |
| — |
| |
| — |
| |
| — |
| |
| — |
| |
|
| 2,285 |
| |
| — |
| |
| — |
| |
| — |
| |
| — |
|
Stock-based compensation | |
| — |
| |
| — |
| |
| — |
| |
| — |
| |
| — |
| |
| — |
| |
|
| — |
| |
| — |
| |
| 332 |
| |
| — |
| |
| 332 |
|
Net loss | |
| — |
| |
| — |
| |
| — |
| |
| — |
| |
| — |
| |
| — |
| |
|
| — |
| |
| — |
| |
| — |
| |
| (9,853 | ) | |
| (9,853 | ) |
Balance at March 31, 2022 | |
| 5,000,000 |
| | $ | 2,350 |
| |
| 41,666,666 |
| | $ | 49,762 |
| |
| 37,499,999 |
| | $ | 89,744 |
| |
|
| 943,428 |
| | $ | 1 |
| | $ | 1,572 |
| | $ | (75,242 | ) | | $ | (73,669 | ) |
Issuance of common stock | |
| — |
| |
| — |
| |
| — |
| |
| — |
| |
| — |
| |
| — |
| |
|
| 8,768 |
| |
| — |
| |
| 86 |
| |
| — |
| |
| 86 |
|
Vesting of restricted | |
| — |
| |
| — |
| |
| — |
| |
| — |
| |
| — |
| |
| — |
| |
|
| 2,285 |
| |
| — |
| |
| — |
| |
| — |
| |
| — |
|
Stock-based compensation | |
| — |
| |
| — |
| |
| — |
| |
| — |
| |
| — |
| |
| — |
| |
|
| — |
| |
| — |
| |
| 382 |
| |
| — |
| |
| 382 |
|
Net loss | |
| — |
| |
| — |
| |
| — |
| |
| — |
| |
| — |
| |
| — |
| |
|
| — |
| |
| — |
| |
| — |
| |
| (9,580 | ) | |
| (9,580 | ) |
Balance at June 30, 2022 | |
| 5,000,000 |
| | $ | 2,350 |
| |
| 41,666,666 |
| | $ | 49,762 |
| |
| 37,499,999 |
| | $ | 89,744 |
| |
|
| 954,481 |
| | $ | 1 |
| | $ | 2,040 |
| | $ | (84,822 | ) | | $ | (82,781 | ) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
| |||||||||||
| Convertible Preferred Stock |
| |
|
|
| |
|
| |
|
| |
|
| |
|
| |||||||||||||||||||||||||||
| Series Seed |
|
| Series A |
| | Series B |
| |
| Common Stock |
| | Additional |
| | Accumulated |
| | Total |
| ||||||||||||||||||||||||
| Shares |
| | Amount |
| | Shares |
| | Amount |
| | Shares |
| | Amount |
| |
| Shares |
| | Amount |
| | Capital |
|
| Deficit |
|
| Equity |
| ||||||||||||
Balance at December 31, 2022 | |
| — |
| | $ | — |
| |
| — |
| | $ | — |
| |
| — |
| | $ | — |
| |
|
| 17,403,315 |
| | $ | 2 |
| | $ | 288,814 |
| | $ | (112,216 | ) | | $ | 176,600 |
|
Issuance of common stock | |
| — |
| |
| — |
| |
| — |
| |
| — |
| |
| — |
| |
| — |
| |
|
| 74,753 |
| |
| — |
| |
| 1,067 |
| |
| — |
| |
| 1,067 |
|
Vesting of restricted | |
| — |
| |
| — |
| |
| — |
| |
| — |
| |
| — |
| |
| — |
| |
|
| 958 |
| |
| — |
| |
| — |
| |
| — |
| |
| — |
|
Stock-based compensation | |
| — |
| |
| — |
| |
| — |
| |
| — |
| |
| — |
| |
| — |
| |
|
| — |
| |
| — |
| |
| 1,024 |
| |
| — |
| |
| 1,024 |
|
Sale of common stock in | |
| — |
| |
| — |
| |
| — |
| |
| — |
| |
| — |
| |
| — |
| |
|
| 1,488,166 |
| |
| — |
| |
| 34,148 |
| |
| — |
| |
| 34,148 |
|
Sale of pre-funded warrants |
|
| — |
| |
| — |
| |
| — |
| |
| — |
| |
| — |
| |
| — |
|
|
|
| — |
|
|
| — |
|
|
| 28,206 |
|
|
| — |
|
|
| 28,206 |
|
Sale of common stock in |
|
| — |
| |
| — |
| |
| — |
| |
| — |
| |
| — |
| |
| — |
|
|
|
| 608,050 |
|
|
| — |
|
|
| 14,591 |
|
|
| — |
|
|
| 14,591 |
|
Net loss | |
| — |
| |
| — |
| |
| — |
| |
| — |
| |
| — |
| |
| — |
| |
|
| — |
| |
| — |
| |
| — |
| |
| (22,781 | ) | |
| (22,781 | ) |
Balance at March 31, 2023 | |
| — |
| | $ | — |
| |
| — |
| | $ | — |
|
|
| — |
| | $ | — |
|
|
|
| 19,575,242 |
| | $ | 2 |
|
| $ | 367,850 |
| | $ | (134,997 | ) |
| $ | 232,855 |
|
Issuance of common stock | |
| — |
| |
| — |
| |
| — |
| |
| — |
| |
| — |
| |
| — |
| |
|
| 41,475 |
| |
| — |
| |
| 242 |
| |
| — |
| |
| 242 |
|
Vesting of restricted | |
| — |
| |
| — |
| |
| — |
| |
| — |
| |
| — |
| |
| — |
| |
|
| 958 |
| |
| — |
| |
| — |
| |
| — |
| |
| — |
|
Stock-based compensation | |
| — |
| |
| — |
| |
| — |
| |
| — |
| |
| — |
| |
| — |
| |
|
| — |
| |
| — |
| |
| 1,309 |
| |
| — |
| |
| 1,309 |
|
Sale of common stock in |
|
| — |
| |
| — |
| |
| — |
| |
| — |
| |
| — |
| |
| — |
|
|
|
| 3,015,919 |
|
|
| — |
|
|
| 138,508 |
|
|
| — |
|
|
| 138,508 |
|
Sale of pre-funded warrants |
|
| — |
| |
| — |
| |
| — |
| |
| — |
| |
| — |
| |
| — |
|
|
|
| — |
|
|
| — |
|
|
| 9,373 |
|
|
| — |
|
|
| 9,373 |
|
Sale of common stock in |
|
| — |
| |
| — |
| |
| — |
| |
| — |
| |
| — |
| |
| — |
|
|
|
| 217,300 |
|
|
| — |
|
|
| 4,879 |
|
|
| — |
|
|
| 4,879 |
|
Net loss | |
| — |
| |
| — |
| |
| — |
| |
| — |
| |
| — |
| |
| — |
| |
|
| — |
| |
| — |
| |
| — |
| |
| (15,947 | ) | |
| (15,947 | ) |
Balance at June 30, 2023 | |
| — |
| | $ | — |
| |
| — |
| | $ | — |
|
|
| — |
| | $ | — |
|
|
|
| 22,850,894 |
| | $ | 2 |
|
| $ | 522,161 |
| | $ | (150,944 | ) |
| $ | 371,219 |
|
The accompanying notes are an integral part of thethese condensed consolidated financial statements.
18
Gemini Therapeutics, Inc.DISC MEDICINE, INC.
Condensed Consolidated Statements of Operations and Comprehensive LossCONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS
(In thousands)
(Unaudited)
| Six Months Ended |
| ||||||
| 2023 |
|
| 2022 |
| |||
Cash flows from operating activities |
|
|
|
|
|
| ||
Net loss |
| $ | (38,728 | ) |
| $ | (19,433 | ) |
Adjustments to reconcile net loss to net cash used in operations: |
|
|
|
|
|
| ||
Depreciation and amortization |
|
| 50 |
|
|
| 40 |
|
Stock-based compensation |
|
| 2,333 |
|
|
| 714 |
|
Change in fair value of contingent value right liability |
|
| 1,500 |
|
|
| — |
|
Change in fair value of derivative liability |
|
| — |
|
|
| (2,490 | ) |
Noncash lease expense |
|
| 134 |
|
|
| 47 |
|
Changes in operating assets and liabilities: |
|
|
|
|
|
| ||
Prepaid expenses and other current assets |
|
| 128 |
|
|
| (1,860 | ) |
Other assets |
|
| — |
|
|
| 64 |
|
Accounts payable |
|
| (11,476 | ) |
|
| 1,003 |
|
Accrued expenses |
|
| (1,991 | ) |
|
| (920 | ) |
Operating lease liabilities |
|
| (140 | ) |
|
| (60 | ) |
Net cash used in operating activities |
|
| (48,190 | ) |
|
| (22,895 | ) |
Cash flow from investing activities |
|
|
|
|
|
| ||
Purchases of property and equipment |
|
| (41 | ) |
|
| (120 | ) |
Net cash used in investing activities |
|
| (41 | ) |
|
| (120 | ) |
Cash flow from financing activities |
|
|
|
|
|
| ||
Proceeds from sale of common stock in offerings, net of issuance costs paid |
|
| 192,226 |
|
|
| — |
|
Proceeds from sale of pre-funded warrants in offerings, net of issuance costs paid |
|
| 37,637 |
|
|
| — |
|
Proceeds from stock option exercises |
|
| 1,298 |
|
|
| 141 |
|
Net cash provided by financing activities |
|
| 231,161 |
|
|
| 141 |
|
Net increase (decrease) in cash, cash equivalents and restricted cash |
|
| 182,930 |
|
|
| (22,874 | ) |
Cash, cash equivalents and restricted cash, beginning of period |
|
| 194,788 |
|
|
| 88,213 |
|
Cash, cash equivalents and restricted cash, end of period |
| $ | 377,718 |
|
| $ | 65,339 |
|
Supplemental disclosure of non-cash activities |
|
|
|
|
|
| ||
Right-of-use assets obtained in exchange for new operating lease liabilities |
| $ | 1,106 |
|
| $ | — |
|
Decrease in right-of-use assets related to lease modification |
| $ | 237 |
|
| $ | — |
|
Decrease in operating lease liabilities due to lease modification |
| $ | 207 |
|
| $ | — |
|
Receivable related to deferred issuance costs on sale of common stock and pre-funded warrants in |
| $ | 400 |
|
| $ | — |
|
Deferred issuance costs on sale of common stock and pre-funded warrants in |
| $ | 558 |
|
| $ | 4 |
|
(In thousands, except share and per share amounts)
|
| Three Months Ended |
|
| Six Months Ended |
| ||||||||||
|
| 2022 |
|
| 2021 |
|
| 2022 |
|
| 2021 |
| ||||
Operating expenses: |
|
|
|
|
|
|
|
|
|
|
|
| ||||
Research and development |
| $ | 2,580 |
|
| $ | 10,842 |
|
| $ | 12,384 |
|
| $ | 22,628 |
|
General and administrative |
|
| 4,654 |
|
|
| 5,478 |
|
|
| 10,027 |
|
|
| 10,182 |
|
Total operating expenses |
|
| 7,234 |
|
|
| 16,320 |
|
|
| 22,411 |
|
|
| 32,810 |
|
Loss from operations |
|
| (7,234 | ) |
|
| (16,320 | ) |
|
| (22,411 | ) |
|
| (32,810 | ) |
Other income (expense): |
|
|
|
|
|
|
|
|
|
|
|
| ||||
Interest expense |
|
| (48 | ) |
|
| (121 | ) |
|
| (114 | ) |
|
| (1,969 | ) |
Interest income |
|
| 149 |
|
|
| 5 |
|
|
| 158 |
|
|
| 6 |
|
Loss on conversion of convertible notes |
|
| 0 |
|
|
| 0 |
|
|
| 0 |
|
|
| (711 | ) |
Other expense |
|
| 0 |
|
|
| (11 | ) |
|
| 0 |
|
|
| (11 | ) |
Other income |
|
| 43 |
|
|
| 0 |
|
|
| 46 |
|
|
| 0 |
|
Net loss and comprehensive loss |
| $ | (7,090 | ) |
| $ | (16,447 | ) |
| $ | (22,321 | ) |
| $ | (35,495 | ) |
Net loss per share, basic and diluted |
| $ | (0.16 | ) |
| $ | (0.38 | ) |
| $ | (0.52 | ) |
| $ | (0.94 | ) |
Weighted average common shares outstanding, basic and diluted |
|
| 43,230,523 |
|
|
| 43,041,856 |
|
|
| 43,221,712 |
|
|
| 37,564,936 |
|
The accompanying notes are an integral part of the condensed consolidated financial statements.
2
Gemini Therapeutics, Inc.
Condensed Consolidated Statements of Stockholders’ Equity
(Unaudited)
(In thousands, except share amounts)
|
| Series A |
|
| Series B |
|
|
| Old Gemini |
|
| Common Stock |
|
| Additional |
|
| Accumulated |
|
| Total |
| |||||||||||||||||||||||
|
| Shares |
|
| Amount |
|
| Shares |
|
| Amount |
|
|
| Shares |
|
| Amount |
|
| Shares |
|
| Amount |
|
| capital |
|
| deficit |
|
| equity |
| |||||||||||
Balance at December 31, 2020 |
|
| 39,722,088 |
|
| $ | 47,113 |
|
|
| 24,790,938 |
|
| $ | 33,336 |
|
|
|
| 6,900,493 |
|
| $ | 7 |
|
|
| - |
|
| $ | - |
|
| $ | 10,504 |
|
| $ | (112,821 | ) |
| $ | (102,310 | ) |
Retroactive application of the |
|
| (39,722,088 | ) |
|
| (47,113 | ) |
|
| (24,790,938 | ) |
|
| (33,336 | ) |
|
|
| (6,900,493 | ) |
|
| (7 | ) |
|
| 15,565,380 |
|
|
| 2 |
|
|
| 80,454 |
|
|
| - |
|
|
| 80,449 |
|
Balance at December 31, 2020, |
|
| - |
|
|
| - |
|
|
| - |
|
|
| - |
|
|
|
| - |
|
|
| - |
|
|
| 15,565,380 |
|
|
| 2 |
|
|
| 90,958 |
|
|
| (112,821 | ) |
|
| (21,861 | ) |
Issuance of common stock upon |
|
| - |
|
|
| - |
|
|
| - |
|
|
| - |
|
|
|
| - |
|
|
| - |
|
|
| 25,041,150 |
|
|
| 2 |
|
|
| 195,880 |
|
|
| - |
|
|
| 195,882 |
|
Conversion of promissory notes (Note 2) |
|
| - |
|
|
| - |
|
|
| - |
|
|
| - |
|
|
|
| - |
|
|
| - |
|
|
| 2,341,316 |
|
|
| - |
|
|
| 14,515 |
|
|
| - |
|
|
| 14,515 |
|
Issuance of common stock upon |
|
| - |
|
|
| - |
|
|
| - |
|
|
| - |
|
|
|
| - |
|
|
| - |
|
|
| 15,257 |
|
|
| - |
|
|
| 76 |
|
|
| - |
|
|
| 76 |
|
Vesting of restricted common stock |
|
| - |
|
|
| - |
|
|
| - |
|
|
| - |
|
|
|
| - |
|
|
| - |
|
|
| 35,561 |
|
|
| - |
|
|
| - |
|
|
| - |
|
|
| - |
|
Issuance of common stock upon |
|
| - |
|
|
| - |
|
|
| - |
|
|
| - |
|
|
|
| - |
|
|
| - |
|
|
| 3,480 |
|
|
| - |
|
|
| 4 |
|
|
| - |
|
|
| 4 |
|
Stock-based compensation expense |
|
| - |
|
|
| - |
|
|
| - |
|
|
| - |
|
|
|
| - |
|
|
| - |
|
|
| - |
|
|
| - |
|
|
| 1,593 |
|
|
| - |
|
|
| 1,593 |
|
Net loss |
|
| - |
|
|
| - |
|
|
| - |
|
|
| - |
|
|
|
| - |
|
|
| - |
|
|
| - |
|
|
| - |
|
|
| - |
|
|
| (19,048 | ) |
|
| (19,048 | ) |
Balance at March 31, 2021 |
|
| - |
|
|
| - |
|
|
| - |
|
|
| - |
|
|
|
| - |
|
|
| - |
|
|
| 43,002,144 |
|
|
| 4 |
|
|
| 303,026 |
|
|
| (131,869 | ) |
|
| 171,161 |
|
Issuance of common stock upon |
|
| - |
|
|
| - |
|
|
| - |
|
|
| - |
|
|
|
| - |
|
|
| - |
|
|
| 52,968 |
|
|
| - |
|
|
| 106 |
|
|
| - |
|
|
| 106 |
|
Stock-based compensation expense |
|
| - |
|
|
| - |
|
|
| - |
|
|
| - |
|
|
|
| - |
|
|
| - |
|
|
| - |
|
|
| - |
|
|
| 2,862 |
|
|
| - |
|
|
| 2,862 |
|
Net loss |
|
| - |
|
|
| - |
|
|
| - |
|
|
| - |
|
|
|
| - |
|
|
| - |
|
|
| - |
|
|
| - |
|
|
| - |
|
|
| (16,447 | ) |
|
| (16,447 | ) |
Balance at June 30, 2021 |
|
| - |
|
| $ | - |
|
|
| - |
|
| $ | - |
|
|
|
| - |
|
| $ | - |
|
|
| 43,055,112 |
|
| $ | 4 |
|
| $ | 305,994 |
|
| $ | (148,316 | ) |
| $ | 157,682 |
|
|
| Series A |
|
| Series B |
|
|
| Old Gemini |
|
| Common Stock |
|
| Additional |
|
| Accumulated |
|
| Total |
| |||||||||||||||||||||||
|
| Shares |
|
| Amount |
|
| Shares |
|
| Amount |
|
|
| Shares |
|
| Amount |
|
| Shares |
|
| Amount |
|
| capital |
|
| deficit |
|
| equity |
| |||||||||||
Balance at December 31, 2021 |
|
| - |
|
| $ | - |
|
|
| - |
|
| $ | - |
|
|
|
| - |
|
| $ | - |
|
|
| 43,208,159 |
|
| $ | 4 |
|
| $ | 309,527 |
|
| $ | (184,690 | ) |
| $ | 124,841 |
|
Issuance of common stock upon |
|
| - |
|
|
| - |
|
|
| - |
|
|
| - |
|
|
|
| - |
|
|
| - |
|
|
| 19,000 |
|
|
| - |
|
|
| 25 |
|
|
| - |
|
|
| 25 |
|
Stock-based compensation expense |
|
| - |
|
|
| - |
|
|
| - |
|
|
| - |
|
|
|
| - |
|
|
| - |
|
|
| - |
|
|
| - |
|
|
| 1,976 |
|
|
| - |
|
|
| 1,976 |
|
Net loss |
|
| - |
|
|
| - |
|
|
| - |
|
|
| - |
|
|
|
| - |
|
|
| - |
|
|
| - |
|
|
| - |
|
|
| - |
|
|
| (15,231 | ) |
|
| (15,231 | ) |
Balance at March 31, 2022 |
|
| - |
|
|
| - |
|
|
| - |
|
|
| - |
|
|
|
| - |
|
|
| - |
|
|
| 43,227,159 |
|
|
| 4 |
|
|
| 311,528 |
|
|
| (199,921 | ) |
|
| 111,611 |
|
Issuance of common stock upon |
|
| - |
|
|
| - |
|
|
| - |
|
|
| - |
|
|
|
| - |
|
|
| - |
|
|
| 7,334 |
|
|
| - |
|
|
| 9 |
|
|
| - |
|
|
| 9 |
|
Proceeds from sale of stock under |
|
| - |
|
|
| - |
|
|
| - |
|
|
| - |
|
|
|
| - |
|
|
| - |
|
|
| 9,960 |
|
|
| - |
|
|
| 11 |
|
|
| - |
|
|
| 11 |
|
Stock-based compensation expense |
|
| - |
|
|
| - |
|
|
| - |
|
|
| - |
|
|
|
| - |
|
|
| - |
|
|
| - |
|
|
| - |
|
|
| 2,356 |
|
|
| - |
|
|
| 2,356 |
|
Net loss |
|
| - |
|
|
| - |
|
|
| - |
|
|
| - |
|
|
|
| - |
|
|
| - |
|
|
| - |
|
|
| - |
|
|
| - |
|
|
| (7,090 | ) |
|
| (7,090 | ) |
Balance at June 30, 2022 |
|
| - |
|
| $ | - |
|
|
| - |
|
| $ | - |
|
|
|
| - |
|
| $ | - |
|
|
| 43,244,453 |
|
| $ | 4 |
|
| $ | 313,904 |
|
| $ | (207,011 | ) |
| $ | 106,897 |
|
The accompanying notes are an integral part of the condensed consolidated financial statements.
3
Gemini Therapeutics, Inc.
Condensed Consolidated Statements of Cash Flows
(Unaudited)
(In thousands)
|
| Six Months Ended June 30, |
| |||||
|
| 2022 |
|
| 2021 |
| ||
Cash flows from operating activities: |
|
|
|
|
|
| ||
Net loss |
| $ | (22,321 | ) |
| $ | (35,495 | ) |
Adjustments to reconcile net loss to net cash used in operating activities: |
|
|
|
|
|
| ||
Depreciation expense |
|
| 0 |
|
|
| 127 |
|
Stock-based compensation expense |
|
| 4,332 |
|
|
| 4,455 |
|
Non-cash interest expense |
|
| 49 |
|
|
| 225 |
|
Loss on conversion of convertible notes |
|
| 0 |
|
|
| 711 |
|
Accretion of discount on convertible notes |
|
| 0 |
|
|
| 1,600 |
|
Changes in operating assets and liabilities: |
|
|
|
|
|
| ||
Prepaid expenses and other current assets |
|
| (1,219 | ) |
|
| (3,272 | ) |
Deferred offering costs |
|
| 0 |
|
|
| 1,341 |
|
Other assets |
|
| 33 |
|
|
| (559 | ) |
Accounts payable |
|
| (1,333 | ) |
|
| 42 |
|
Accrued expenses and other current liabilities |
|
| (5,377 | ) |
|
| (110 | ) |
Net cash used in operating activities |
|
| (25,836 | ) |
|
| (30,935 | ) |
Cash flows from financing activities: |
|
|
|
|
|
| ||
Proceeds from Business Combination, net |
|
| 0 |
|
|
| 195,882 |
|
Proceeds from exercise of stock options |
|
| 34 |
|
|
| 110 |
|
Proceeds from sale of stock under employee stock purchase plan |
|
| 11 |
|
|
| 0 |
|
Principal payments on term loan |
|
| (2,500 | ) |
|
| (2,083 | ) |
Net cash provided by (used in) financing activities |
|
| (2,455 | ) |
|
| 193,909 |
|
Increase (decrease) in cash, cash equivalents and restricted cash |
|
| (28,291 | ) |
|
| 162,974 |
|
Cash, cash equivalents and restricted cash at beginning of period |
|
| 136,950 |
|
|
| 4,826 |
|
Cash, cash equivalents and restricted cash at end of period |
| $ | 108,659 |
|
| $ | 167,800 |
|
|
|
|
|
|
|
| ||
Supplemental disclosure |
|
|
|
|
|
| ||
Cash paid for interest |
| $ | 83 |
|
| $ | 144 |
|
|
|
|
|
|
|
| ||
Noncash financing activities |
|
|
|
|
|
| ||
Proceeds from exercise of stock options included in other current assets |
| $ | (9 | ) |
| $ | 0 |
|
Conversion of convertible notes to Series B preferred stock |
| $ | 0 |
|
| $ | 14,515 |
|
Exercise of warrants |
| $ | 0 |
|
| $ | 76 |
|
Property and equipment purchases included in accounts payable and accrued expenses |
| $ | 0 |
|
| $ | 61 |
|
The accompanying notes are an integral part of the condensed consolidated financial statements.
4
Gemini Therapeutics, Inc.
Notes to Condensed Consolidated Financial Statements
(Unaudited)
1. Nature of the business
Gemini Therapeutics, Inc. (the “Company” or “Gemini”) is a clinical-stage precision medicine company developing novel therapeutic compounds to treat genetically defined, age-related macular degeneration. The Company was founded on March 3, 2015.
Unless the context otherwise requires, references in these notes to “Gemini”, “the Company”, “we”, “us” and “our” and any related terms are intended to mean Gemini Therapeutics, Inc. and its consolidated subsidiary following the Business Combination (as defined below).
Since its inception, the Company has devoted substantially all its efforts and financial resources to organizing and staffing the Company, business planning, raising capital, discovering product candidates and securing related intellectual property rights and conducting research and development activities for its product candidates. In January 2022, the Company discontinued both of its Phase 2a clinical trials for GEM103. The Company's other product candidate, GEM307, is in the preclinical stage of development.
In February 2022, the Company announced a corporate restructuring and that it initiated a process to evaluate strategic alternatives. After a comprehensive review of strategic alternatives, including identifying and reviewing potential candidates for a strategic transaction, on August 9, 2022, the Company entered into an Agreement and Plan of Merger and Reorganization (the “Disc Merger Agreement”) with Disc Medicine, Inc., a Delaware corporation (“Disc”), and Gemstone Merger Sub, Inc., a Delaware corporation and a wholly-owned subsidiary of the Company (“Gem Merger Sub”), pursuant to which, subject to the satisfaction or waiver of the conditions therein, Gem Merger Sub will merge with and into Disc (the “Disc Merger”), with Disc continuing as the surviving company and a wholly-owned subsidiary of the Company. In connection with the Disc Merger, the Company will distribute to the pre-closing Gemini stockholders contingent value rights (“CVRs”), representing the contractual right to receive payments from the post-closing combined company upon receipt of certain proceeds derived from consideration paid as a result of the disposition of the Company’s pre-Disc Merger assets, net of certain permitted deductions for expenses. The Disc Merger was unanimously approved by the Company’s board of directors (the “Board”), and the Board resolved to recommend approval of the Disc Merger Agreement to the Company’s shareholders. The closing of the Disc Merger is subject to approval by the stockholders of the Company and Disc as well as other customary closing conditions, including the effectiveness of a registration statement filed with the SEC in connection with the transaction. If the merger is completed, the business of Disc will continue as the business of the combined company.
The Company’s future operations are highly dependent on the success of the Disc Merger and there can be no assurances that the Disc Merger will be successfully consummated. In the event that the Company does not complete the transaction with Disc, the Company may explore strategic alternatives, including, without limitation, another strategic transaction and/or pursue a dissolution and liquidation of the Company.
On February 5, 2021 (the “Closing Date”), FS Development Corporation, a Delaware corporation (“FSDC”), consummated the previously announced business combination (the “Business Combination”) pursuant to the terms of the Agreement and Plan of Merger, dated as of October 15, 2020 (as amended, supplemented or otherwise modified from time to time, the “Merger Agreement”), by and among Gemini Therapeutics, Inc., a Delaware corporation (“Old Gemini”), Shareholder Representative Services LLC, a Colorado limited liability company solely in its capacity as the representative, agent and attorney-in-fact of the Company Securityholders (the “Stockholders’ Representative”), FSDC and FSG Merger Sub Inc., a Delaware corporation (“Merger Sub”).
FSDC was incorporated in Delaware on June 25, 2020 and was formed for the purpose of effecting a merger, capital stock exchange, asset acquisition, stock purchase, reorganization or similar business combination with one or more businesses.
On the day prior to the Closing Date, Old Gemini changed its name to “Gemini Therapeutics Sub, Inc.” Pursuant to the Merger Agreement, on the Closing Date, (i) FSDC changed its name to “Gemini Therapeutics, Inc.” and (ii) Old Gemini merged with and into Merger Sub (the “Merger”), with Old Gemini as the surviving company in the Merger and, after giving effect to such Merger, Old Gemini becoming a wholly-owned subsidiary of Gemini. Upon the closing of the Business Combination, and pursuant to the terms of the Merger Agreement, the existing shareholders of Old Gemini exchanged their interests for shares of common stock of Gemini.
In connection with the Business Combination, certain investors purchased an aggregate of $95.1 million of the Company’s Common Stock in a private placement of public equity (the “PIPE Financing”). Together with FSDC’s cash resources and funding of the PIPE Financing, the Company received net proceeds of approximately $195.9 million.
For additional information on the Business Combination, please refer to Note 2, Business Combination, to these condensed consolidated financial statements.
59
DISC MEDICINE, INC.
NOTES TO CONDENSED CONSOLIDATED FINANCIAL STATEMENTS
(Unaudited)
1. Organization and Nature of the Business
Disc Medicine, Inc. (together with its subsidiaries, the “Company”) is a clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of novel treatments for patients suffering from serious hematologic diseases. The Company has assembled a portfolio of clinical and preclinical product candidates that aim to modify fundamental biological pathways associated with the formation and function of red blood cells, specifically heme biosynthesis and iron homeostasis. The Company’s current pipeline includes, bitopertin for the treatment of erythropoietic porphyrias (“EPs”) including erythropoietic protoporphyria (“EPP”), and X-linked protoporphyria (“XLP”), and Diamond-Blackfan Anemia (“DBA”); DISC-0974 for the treatment of anemia of myelofibrosis (“MF”), and anemia of chronic kidney disease (“CKD”); and MWTX-003 for the treatment of polycythemia vera (“PV”), and other hematologic disorders. In addition, the Company’s preclinical programs also include DISC-0998, for the treatment of anemia associated with inflammatory diseases. The Company’s approach to product candidate development leverages well-understood molecular mechanisms that have been validated in humans. The Company believes that each of its product candidates, if approved, has the potential to improve the lives of patients suffering from hematologic diseases. The Company was founded in October 2017. The Company’s principal offices are in Watertown, Massachusetts.
The Company is subject to a number of risks and uncertainties common to early-stagedevelopment stage companies in the biotechnology industry, including, but not limited to, uncertainty ofrisks associated with completing preclinical studies and clinical trials, receiving regulatory approvals for product development and commercialization, lack of marketing and sales history,candidates, development by its competitors of new technological innovations,biopharmaceutical products, dependence on key personnel, market acceptance of products, product liability,reliance on third-party organizations, protection of proprietary technology, ability to raise additional financing, compliance with government regulations, and the impact of the ongoingCOVID-19 pandemic or other health crises and evolving novel coronavirus disease (“COVID-19”) pandemic. If the Company does not successfully commercialize any of its product candidates, it will be unableability to generate recurring product revenue or achieve profitability.
secure additional capital to fund operations. The Company’s product candidatesresearch and development programs will require significant additional research and development efforts, including extensive preclinical and clinical testing and regulatory approval, prior to commercialization. These efforts require significant amounts of additional capital, adequate personnel and infrastructure and extensive compliance-reporting capabilities. There can be no assurance that the Company’s research and development will be successfully completed, that adequate protection for the Company’s intellectual property will be obtained, that any products developed will obtain necessary government regulatory approval or that any approved products will be commercially viable. Even if the Company’s product development efforts are successful, it is uncertain when, if ever, the Company will generate significantrealize revenue from product sales. The Company operates in an environment of rapid change in technology and is dependent upon the services of its employees, consultants, third-party contract research organizations and other third-party organizations.
Prior to the Business Combination, the Company primarily financed its operations through the sale of convertible preferred stock, borrowings under convertible promissory notes and borrowings under loan agreements. The Company believes that its $108.7 million of cash and cash equivalents as of June 30, 2022 will enable it to fund its planned operations for at least twelve months from the issuance date of these condensed consolidated financial statements, though the Company may raise additional capital through a combination of equity offerings, debt financings, collaborations, strategic alliances and marketing, distribution or licensing arrangements. Management’s expectations with respect to its ability to fund current planned operations is based on estimates that are subject to risks and uncertainties. Its operating plan may change as a result of many factors currently unknown to management, and there can be no assurance that the current operating plan will be achieved in the time frame anticipated by the Company, and it may need to seek additional funds sooner than anticipated. Furthermore, the operating plan could materially change depending on the outcome of our ongoing strategic alternative review process, including if the Disc Merger is not successfully consummated on a timely basis or at all. If adequate funds are not available to the Company on a timely basis, on acceptable terms or at all, management may be required to delay, limit, reduce or terminate certain of its research, product development or future commercialization efforts, obtain funds through arrangements with collaborators on terms unfavorable to the Company, or pursue merger or acquisition strategies, all of which could adversely affect the holdings or the rights of its stockholders.
Impact of the COVID-19 Pandemic
The ongoing COVID-19 pandemic and the increased prevalence of variants of the virus, and government measures taken in response, have had a significant impact, both direct and indirect, on businesses and commerce, as worker shortages have occurred; supply chains have been disrupted; facilities and production have been suspended; and demand for certain goods and services, such as medical services and supplies, has spiked, while demand for other goods and services, such as travel, has fallen. The ongoing COVID-19 pandemic and related impacts have resulted in and will likely continue to result in significant disruptions to the global economy and capital markets around the world. The Company cannot predict the future progression or full impact of the outbreak and its effects on the Company’s business and operations.
The Company has not incurred impairment losses in the carrying values of its assets as a result of the ongoing COVID-19 pandemic, and it is not aware of any specific related event or circumstance that would require it to revise its estimates reflected in these condensed consolidated financial statements. Although the COVID-19 pandemic did not have a significant impact on the Company’s condensed consolidated financial results in the second quarter of 2022, the full extent to which the ongoing COVID-19 pandemic may impact the Company’s business, results of operations, financial condition and cash flows will depend on future developments that are highly uncertain, and the estimates of the impact on the Company’s business may change based on new information that may emerge concerning COVID-19, including the duration of the pandemic, any potential subsequent waves or strains of COVID-19 infection, the effectiveness, distribution and acceptance of COVID-19 vaccines and the actions to contain it or treat its impact and the economic impact on local, regional, national and international markets.
2. Business CombinationReverse Merger with Gemini
On February 5, 2021, OldAugust 9, 2022, Gemini and FSDC completed the Business Combination pursuant to theTherapeutics, Inc., a Delaware corporation (“Gemini”), Gemstone Merger Agreement with Old Gemini surviving the merger asSub, Inc., a Delaware corporation and a wholly owned subsidiary of FSDC. Net proceeds from the Business Combination totaled approximately $195.9 million, which included funds held in FSDC’s trust accountGemini (“Merger Sub”), and the completionDisc Medicine, Inc., a Delaware corporation (“Private Disc”), entered into an Agreement and Plan of the concurrent PIPE Financing.
Merger and Reorganization (the “Merger Agreement”). The merger was completed on December 29, 2022. In accordance with the termsMerger Agreement, the Merger Sub merged with and subjectinto Private Disc, with Private Disc surviving as a wholly-owned subsidiary of the Company (the “merger”). Gemini changed its name to Disc Medicine Inc., and Private Disc, which remains as a wholly-owned subsidiary of the Company, changed its name to Disc Medicine Opco, Inc. On December 30, 2022, the combined company’s common stock began trading on The Nasdaq Capital Market under the ticker symbol “IRON.”
Except as otherwise indicated, references herein to “Disc,” the “Company,” or the “combined company”, refer to Disc Medicine, Inc. on a post-merger basis, and the term “Private Disc” refers to the conditionsbusiness of privately-held Disc Medicine, Inc., prior to completion of the merger. References to Gemini refer to Gemini Therapeutics, Inc. prior to completion of the merger.
Pursuant to the terms of the Merger Agreement, at the effective time of the Merger, (i) all sharesmerger (the “Effective Time”), each then outstanding share of Old Gemini’s Series B Preferred StockPrivate Disc common stock (including shares of Series B Preferred Stockcommon stock issued upon conversion of outstandingthe Company’s preferred stock (see Note 9) and shares of the Company’s common stock issued in the Private Disc pre-closing financing defined below) was exchanged for 0.1096
6
convertible promissory notes) shares of Gemini’s common stock (the “Exchange Ratio”), Series A Preferredafter taking into account the Reverse Stock Split, as defined below. In addition, each option to purchase Private Disc shares that was outstanding and Common Stock (collectively, “Old Gemini Stock”) issued and outstandingunexercised immediately prior to the effective timeEffective Time was converted into an option to purchase shares of Gemini based on the Exchange Ratio. Immediately following the merger, stockholders of Private Disc owned approximately 74% of the Merger, whether vestedoutstanding common stock of the combined company. The merger was intended to qualify for federal income tax purposes as a tax-free reorganization under the provisions of Section 368(a) of the Internal Revenue Code of 1986, as amended.
At the Effective Time, each person who as of immediately prior to the Effective Time was a stockholder of record of Gemini or unvested, were converted intohad the right to receive their pro rata portion of the 17,942,274 shares of FSDC Class A Common Stock (the “Common Stock”Gemini’s common stock received a contractual contingent value right (“CVR”) issued as Merger consideration (the “Merger Consideration”), provided that 2,150,000 shares of Common Stock are heldby Gemini subject to and in escrow for a period of 12 months fromaccordance with the Closing Date to satisfy any indemnification obligations of Old Gemini under the Merger Agreement; (ii) each option exercisable for Old Gemini Stock that was outstanding immediately prior to effective time of the Merger was assumed and continues in full force and effect on the same terms and conditions as were previously applicableof a Contingent Value Rights Agreement between Gemini, the holder’s representative and the rights agent (the “CVR Agreement”), representing the contractual right to such options, subject to adjustments to exercise price and numberreceive consideration from the post-closing combined company upon the receipt of shares Common Stock issuable upon exercise based oncertain proceeds from a disposition of Gemini’s pre-merger assets during the final conversion ratioperiod that is one year after the closing of the merger, calculated in accordance with the Merger Agreement, and (iii) 4,264,341 sharesCVR Agreement. As of Common Stock were reserved for issuance under the newly adopted 2021 Stock Option and Incentive Plan (the “2021 Plan”).
The Company accounted for the Business Combination as a reverse recapitalization, which is the equivalent of Old Gemini issuing stock for the net assets of FSDC, accompanied by a recapitalization, with FSDC treated as the acquired company for accounting purposes. The determination of FSDC as the “acquired” company for accounting purposes was primarily based on the fact that immediately subsequent to the Business Combination, shareholders of Old Gemini prior to the Business Combination have a majority of the voting power of the combined company, the operations of Old Gemini will comprise all of the ongoing operations of the combined entity, and Old Gemini’s senior management will comprise all of the senior management of the combined company. The net assets of FSDC were stated at historical cost withJune 30, 2023, no goodwill or other intangible assets recorded. Reported results from operations included herein prior to the Business Combination are those of Old Gemini. The shares and corresponding capital amounts and loss per share related to Old Gemini’s outstanding convertible preferred stock and common stock prior to the Business Combinationsuch proceeds have been retroactively restated to reflect the conversion ratio established in the Merger Agreement (1.00 Old Gemini share for 0.2180 shares of the Company) (the “Conversion Ratio”)received (See Note 4).
In connection with the Business Combination, the Company incurred equity issuance costs and other costs considered direct and incremental to the transaction totaling $21.0 million, consisting of legal, accounting, financial advisory and other professional fees. These amounts are reflected within additional paid-in capital in the condensed consolidated balance sheet as of June 30, 2022.
PIPE Financing
Concurrent with the execution of the Business Combination, the CompanyMerger Agreement, certain third parties entered into a subscription agreementsagreement with certain investors (the “PIPE Investors”) pursuantPrivate Disc to which the PIPE Investors subscribed for and purchased an aggregate of 9,506,000purchase shares of Common StockPrivate Disc’s common stock for an aggregate purchase price of $95.153.5 million.million (the “pre-closing financing”).
Summary10
Shares of Net ProceedsPrivate Disc’s common stock issued pursuant to the pre-closing financing were converted into shares of the Company’s common stock based on the Exchange Ratio.
The following table summarizesmerger was accounted for as a reverse recapitalization in accordance with U.S. GAAP. For accounting purposes, Private Disc was considered to be acquiring the elementsassets and liabilities of Gemini in the merger based on the terms of the Merger Agreement and other factors, including: (i) Private Disc’s stockholders own a majority of the voting rights in the combined company; (ii) Private Disc designated a majority (eight of nine) of the initial members of the board of directors of the combined company; (iii) Private Disc’s executive management team became the management of the combined company; (iv) the pre-combination assets of Gemini were primarily cash and cash equivalents and other non-operating assets (the in-process research and development assets potentially remaining as of the combination were considered to be of de minimis value); and (v) the combined company was named Disc Medicine, Inc. and is headquartered in Private Disc’s office in Watertown, Massachusetts. Accordingly, the merger was treated as the equivalent of Private Disc issuing stock to acquire the net proceeds fromassets of Gemini. As a result of the Business Combination (in thousands):merger, the net assets of Gemini were recorded at their acquisition-date fair value in the financial statements of the Company and the reported operating results prior to the merger are those of Private Disc.
Cash - FSDC Trust Account and cash (net of redemptions) |
| $ | 121,782 |
|
Cash - PIPE Financing |
|
| 95,060 |
|
Less: Equity issuance costs and other costs paid |
|
| (20,960 | ) |
Net proceeds from the Business Combination |
| $ | 195,882 |
|
SummaryPursuant to the terms of Shares Issued
The following table summarizes the number of shares of Common Stock outstandingRoche Agreement (see Note 4), immediately following the consummationEffective Time, the Company issued 482,313 shares of the Business Combination:combined company to Roche for no consideration.
Reverse Stock Split and Exchange Ratio
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7On December 29, 2022, in connection with, and prior to the completion of, the merger, Gemini effected a one-for-ten reverse stock split of its then outstanding common stock (the “Reverse Stock Split”). The par value and the authorized shares of the common stock were not adjusted as a result of the Reverse Stock Split. All of Gemini’s issued and outstanding common stock have been retroactively adjusted to reflect this Reverse Stock Split for all periods presented.
All issued and outstanding Private Disc common stock, convertible preferred stock and options prior to the effective date of the merger have been retroactively adjusted to reflect the 0.1096 Exchange Ratio, which reflects the impact of the reverse stock split, for all periods presented.
Liquidity and Capital Resources
The Company’s condensed consolidated financial statements have been prepared on the basis of the Company continuing as a going concern. The Company expects that its existing cash and cash equivalents as of June 30, 2023 of $377.6 million will enable the Company to fund its planned operating expense and capital expenditure requirements for at least twelve months from the date of issuance of these condensed consolidated financial statements. The Company has incurred recurring losses and negative cash flows from operations since inception. As of June 30, 2023, the Company had an accumulated deficit of $150.9 million. The Company expects its operating losses and negative operating cash flows to continue into the foreseeable future. The future viability of the Company is dependent on its ability to generate cash from operating activities or to raise additional capital to finance its operations. There can be no assurance that the Company will ever earn revenues or achieve profitability, or if achieved, that the revenues or profitability will be sustained on a continuing basis. In addition, the Company’s preclinical and clinical development activities, manufacturing and commercialization of the Company’s product candidates, if approved, will require significant additional financing.
3.2. Summary of Significant Accounting Policies
BasisSummary of presentationSignificant Accounting Policies
The accompanying unauditedsignificant accounting policies used in preparation of the condensed consolidated financial statements are described in the Company’s audited consolidated financial statements as of and for the year ended December 31, 2022, and the notes thereto, which are included in the Company’s Annual Report on Form 10-K. There have been no material changes to the significant accounting policies previously disclosed in the Company’s Annual Report on Form 10-K for the year ended December 31, 2022.
Basis of Presentation and Principles of Consolidation
The condensed consolidated financial statements include those of the Company and its subsidiary, Gemini Therapeutics Sub, Inc., after elimination of allwholly-owned subsidiaries. All intercompany accountstransactions and transactions. balances have been eliminated in consolidation.
The accompanying unauditedCompany’s condensed consolidated financial statements and notes hereto have been prepared in conformity with the rules and regulations of the Securities and Exchange Commission (“SEC”) for interim financial reporting and, therefore, omit or condense certain footnotes and other information normally included in financial statementsare prepared in accordance with U.S. generally accepted accounting principles generally accepted in the United States of America (“GAAP”) as set forth in the Financial Accounting Standards Board’s (“FASB”). Any reference in these notes to applicable guidance is meant to refer to the authoritative GAAPaccounting principles generally accepted in the United States as found in the Accounting StandardsStandard Codification (“ASC”) and Accounting Standards Updates (“ASU”) of the FASB.Financial Accounting Standards Board (“FASB”).
Unaudited Interim Condensed Consolidated Financial Information
In the opinion11
The accompanying condensed consolidated financial statements as of management, all adjustments necessary for a fair statement of the financial information, which are of a normalJune 30, 2023 and recurring nature, have been made for the interim periods reported. Results of operations for the three and six months ended June 30, 2023 and 2022 are unaudited. The financial data and 2021 are not necessarily indicativeother information contained in the notes hereto as of the results for the entire fiscal year or any other period. The condensed consolidated financial informationJune 30, 2023 and for the three and six months ended June 30, 2023 and 2022 and 2021are also unaudited. The condensed consolidated balance sheet data as of December 31, 2022 was derived from the Company’s audited consolidated financial statements included in the Company’s Annual Report on Form 10-K.
The unaudited interim condensed consolidated financial statements have been prepared on the same basis as and should be read in conjunction with the audited annual consolidated financial statements, and notes theretoin the opinion of management, reflect all adjustments, which include only normal recurring adjustments necessary for the year ended December 31, 2021 included infair presentation of the Company’s Annual Report on Form 10-Kfinancial position as filed withof June 30, 2023 and the SEC on March 10, 2022.
As a resultresults of the Business Combination, the sharesits operations and corresponding capital amounts and loss per share related to Old Gemini’s outstanding convertible preferred stock and common stock prior to the Business Combination have been retroactively restated to reflect the Conversion Ratio established in the Merger Agreement. For additional information regarding the Business Combination, please refer to Note 2, Business Combination, to these condensed consolidated financial statements.
The significant accounting policies used in preparation of these unaudited condensed consolidated financial statementscash flows for the three and six months ended June 30, 2022 are consistent with those discussed in Note 3 to the2023 and 2022. These unaudited condensed consolidated financial statements should be read in conjunction with the audited consolidated financial statements as of and for the year ended December 31, 2022, and the notes thereto, included in the Company’s 2021 Annual Report on Form 10-K and are updated below as necessary.
CARES Act10-K.
The CARES Act provided refundable employee retention credits, which could be used to offset payroll tax liabilities. On March 11, 2021, President Biden signed the American Rescue Plan Act (“ARPA”). The ARPA includes several provisions, such as measures that extend and expand the employee retention credit, previously enacted under the CARES Act, through December 31, 2021.
Measures not related to income-based taxes within the CARES Act include (1) allowing an employer to pay its share of Social Security payroll taxes that would otherwise be due from the date of enactment through December 31, 2020 over the following two years and (2) allowing eligible employers subject to closure due to the COVID-19 pandemic to receive a 50% credit on qualified wages against their employment taxes each quarter, with any excess credits eligible for refunds.
As there is no authoritative guidance under GAAP for accounting for grants to for-profit business entities, the Company accounts for the grant by analogy to International Accounting Standards 20 – Accounting for Government Grants and Disclosure of Government Assistance (“IAS 20”). During the three months ended June 30, 2022, the Company recorded an employee retention credit of $1.0 million upon completion of an analysis providing reasonable assurance that it met the conditions set forth in the CARES Act and it was reasonably assured that it will receive the employee retention credit. The employee retention credit is recorded in research and development expenses and general and administrative expenses in the manner in which the qualified wages and related costs were classified. $0.7 million and $0.3 million have been recorded to reduce research and development and general and administrative expenses, respectively, in the accompanying condensed consolidated statement of operations and comprehensive lossresults for the three and six months ended June 30, 2022. As2023 and 2022 are not necessarily indicative of June 30, 2022,results to be expected for the employee retention credit receivable of $1.0 million is included in prepaid expenses and other current assets in the accompanying condensed consolidated balance sheet.
Recently adopted accounting pronouncements
In February 2016, the FASB issued ASU No. 2016-02, Leases (Topic 842), Amendments to the FASB Accounting Standards Codification (“ASU 2016-02”), which replaces the existing guidance for leases. ASU 2016-02 requires the identification of arrangements that should be accounted for as leases by lessees. In general, for lease arrangements exceeding a twelve-month term, these arrangements must now be recognized as assets and liabilities on the balance sheet of the lessee. Under ASU 2016-02, a right-of-use asset and a lease liability will be recorded for all leases, whether operatingfull years, or financing, while the income statement will reflect lease expense for operating leases and amortization/interest expense for financing leases. The balance sheet amount recorded for existing leases at the date of adoption of ASU 2016-02 must be calculated using the applicable incremental borrowing rate at the date of adoption. The Company adopted ASU 2016-02 on January 1, 2022 using the modified retrospective approach. The Company elected the package of practical expedients which
8
allows entities to not reassess (i) whether an arrangement is or contains a lease, (ii) the classification of its leases, and (iii) the accounting for initial direct costs. Further, the Company elected, by class of underlying asset, the short-term lease exception for leases with terms of twelve months or less. In doing so, the Company did not recognize a lease liability or right-of-use asset on its consolidated balance sheets for such short-term leases. Finally, the Company elected, by class of underlying asset, the practical expedient to not separate lease and non-lease components. The Company terminated its lease agreement on December 31, 2021 for its office and laboratory space. The Company does not have any other leases within the scope of ASU 2016-02. Therefore, the adoption of ASU 2016-02 did not have an impact on the Company's condensed consolidated financial statements and related disclosures.
In December 2019, the FASB issued ASU 2019-12, Income Taxes (Topic 740): Simplifying the Accounting for Income Taxes (“ASU 2019-12”), which is intended to simplify the accounting for income taxes. ASU 2019-12 removes certain exceptions to the general principles in Topic 740 and also clarifies and amends existing guidance to improve consistent application. The Company adopted ASU 2019-12 on January 1, 2022. The adoption did not have a material effect on the Company’s condensed consolidated financial statements.
In November 2021, the FASB issued ASU No. 2021-10, Government Assistance (Topic 832): Disclosures by Business Entities about Government Assistance. This standard increases the transparency of transactions with the government that are accounted for by applying a grantinterim periods, or contribution accounting model, and aims to reduce diversity that currently exists in the recognition, measurement, presentation, and disclosure of government assistance received by business entities due to the lack of specific authoritative guidance in GAAP. This standard requires an entity to provide information regarding the nature of the transaction with a government and the related accounting policy used to account for this transaction, the line item on the condensed consolidated balance sheet and condensed consolidated statement of operations and comprehensive loss that are affected by the transaction and the amounts applicable to each financial statement line item, and the significant terms and conditions of the transaction, including commitments and contingencies. The Company adopted ASU 2021-10 on January 1, 2022 using the prospective approach. The adoption did not have a material effect on the Company’s condensed consolidated financial statements.any future year or period.
Use of estimatesEstimates
The preparation of the unauditedCompany’s condensed consolidated financial statements in conformity with GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities and disclosure of contingent assets and liabilities at the date of the condensed consolidated financial statements and the reported amounts of expenses during the reporting period. Significant estimates contained withinand assumptions reflected in these condensed consolidated financial statements include, but are not limited to the accruals ofaccrued research and development expenses, share-based awards utilized forexpenses; stock-based compensation purposes and, prior toexpense; the Business Combination, the estimated fair value of the Company’s common stock prior to the effective date of the merger; the fair value determinations for instruments accounted for at fair value including contingent amounts payable to third parties upon the consummation of specified transactions, including a Roche Qualified Transaction (see Note 4); the fair value of Gemini’s development programs at the Effective Time; the fair value of the CVR; the incremental borrowing rate for determining lease liabilities and warrant liability.right-of-use assets and income taxes. The Company bases its estimates on historical experience, known trends and other market-specific or other relevant factors that it believeshas concluded to be reasonable under the circumstances. On an ongoing basis, management evaluates its estimates as there are changes in circumstances, facts and experience. Changes in estimates are recorded in the period in which they become known. Actual results may differ materially from those estimates or assumptions.
Concentration of Credit Risk and of Significant Suppliers
Financial instruments that potentially subject the Company to significant concentrations of credit risk consist primarily of cash and cash equivalents. The Company maintains deposits in federally insured financial institutions in excess of federally insured limits and limits its exposure to cash risk by placing its cash with high credit quality accredited financial institutions. The Company has concluded that it is not subject to unusual credit risk beyond the normal credit risk associated with commercial banking relationships.
The Company relies, and expects to continue to rely, on a small number of vendors to manufacture supplies and to process its product candidates for its development programs. These programs could be adversely affected by a significant interruption in the manufacturing process or supply chain.
Restricted cashCash
RestrictedThe Company maintains a letter of credit for the benefit of its landlord related to its current leased office space in Watertown, Massachusetts which is classified within other assets. The Company is required to maintain a separate cash amountedbalance to $0 and $0.3 million assecure its letter of June 30, 2022 and December 31, 2021, which consistedcredit. The Company maintained a letter of credit of less than $0.1 million to collateralize the Company’s credit cardfor leased space in Cambridge, Massachusetts, which was classified within prepaids and $other current assets. This lease was terminated in 0.22021 million to collateralize its irrevocable standbyand the letter of credit for its facility lease arrangement.was subsequently returned and released from restricted cash.
Recently Adopted Accounting Pronouncements
In June 2016, the FASB issued ASU No. 2016-13, Financial Instruments - Credit Losses (Topic 326): Measurement of Credit Losses on Financial Instruments, which has been subsequently amended by ASU 2018-19, ASU 2019-04, ASU 2019-05, ASU 2019-10, ASU 2019-11, ASU 2020-03, and ASU 2022-02 (“ASU 2016-13”). This standard requires that credit losses be recorded using an expected losses model rather than the incurred losses model that was previously used and establishes additional credit risk disclosures associated with financial assets. The facility lease arrangement was terminatedamendments in this standard should be applied on December 31, 2021.a modified retrospective basis to all periods presented. The Company adopted ASU 2016-13 on January 1, 2023 using the modified retrospective approach. The adoption of this standard did not have a material effect on the Company’s financial position, results of operations or disclosures.
A reconciliation of12
Recently Issued Accounting Pronouncements Not Yet Adopted
Other accounting standards that have been issued or proposed by the cash and cash equivalents and restricted cash as presented inFASB or other standards-setting bodies that do not require adoption until a future date are not expected to have a material impact on the Company’s condensed consolidated balance sheetsfinancial statements upon adoption.
3. Reverse Merger with Gemini
As described in Note 1, Private Disc merged with Gemini on December 29, 2022. The merger was accounted for as a reverse recapitalization with Private Disc as the accounting acquirer. The primary pre-combination assets of Gemini were cash and cash equivalents. Under reverse recapitalization accounting, the assets and liabilities of Gemini were recorded at their fair value which approximated book value due to the Company’s condensed consolidated statements of cash flows is as follows (in thousands):
|
| June 30, |
|
| December 31, |
| ||
Cash and cash equivalents |
| $ | 108,659 |
|
| $ | 136,627 |
|
Restricted cash |
|
| 0 |
|
|
| 323 |
|
Total cash, cash equivalents and restricted cash |
| $ | 108,659 |
|
| $ | 136,950 |
|
Emerging growth company status
The Company qualifies as an “emerging growth company” (“EGC”), as defined in the Jumpstart Our Business Startups Act (“JOBS Act”), and may take advantage of certain exemptions from various reporting requirements that are applicable to other public companies that are not EGCs. The Company may take advantage of these exemptions until it is no longer an EGC under Section 107short-term nature of the JOBS Act, which provides that an EGC can take advantage of the extended transition period afforded by the JOBS Act for the implementation of newaccounts. No goodwill or revised accounting standards. The Company has elected to avail itself of the extended transition period and, therefore, while the Company is an EGC it will not be subject to new or revised accounting standards at the same time that they become applicable to other public companies that are not EGCs, unless it chooses to early adopt a new or revised accounting standard. As a result of this
9
election,intangible assets were recognized. Consequently, the condensed consolidated financial statements may notof the Company reflect the operations of Private Disc for accounting purposes, together with a deemed issuance of shares equivalent to the shares held by the former stockholders of Gemini, the legal acquirer, and a recapitalization of the equity of Private Disc, the accounting acquirer.
As part of the reverse recapitalization, the Company acquired $97.4 million of cash and cash equivalents. The Company also obtained prepaids and other assets of $1.8 million and assumed accounts payable and accrued expenses of $7.8 million. Gemini’s development programs had ceased prior to the merger and were deemed to be comparable to companies that comply with public company FASB standards’ effective dates.de minimis in value at the transaction date.
In addition, the Company recognized $0.6 million in share-based compensation expense as a result of the acceleration of vesting of stock options, performance stock units and restricted stock units at the time of merger. This amount was recorded in general and administrative expense in the consolidated statements of operations and comprehensive loss for the year ended December 31, 2022. The Company also incurred transaction costs of $7.9 million and this amount is recorded in additional paid-in capital in the consolidated statements of convertible preferred stock and stockholders’ equity (deficit) for the year ended December 31, 2022.
4. Fair value measurementsValue Measurements
The following tables present information about the Company’s financial assets and liabilities that are regularly measured and carried at fair value (in thousands) on a recurring basis and indicate the level ofwithin the fair value hierarchy usedof the valuation techniques the Company utilized to determine such fair values:value, which is described further within Note 2 of the Company’s Annual Report on Form 10-K for the year ended December 31, 2022.
Financial assets and liabilities measured at fair value on a recurring basis are summarized as follows (in thousands):
June 30, 2022 |
| Level 1 |
|
| Level 2 |
|
| Level 3 |
|
| Total |
| ||||
Assets |
|
|
|
|
|
|
|
|
|
|
|
| ||||
Money market funds in cash and cash equivalents |
| $ | 107,659 |
|
| $ | - |
|
| $ | - |
|
| $ | 107,659 |
|
| June 30, 2023 |
| ||||||||||
| Level 1 |
| | Level 2 |
| | Level 3 |
| ||||
Assets | |
|
| |
|
| |
|
| |||
Cash equivalents: | |
|
| |
|
| |
|
| |||
Money market funds | | $ | 80,132 |
| | $ | — |
| | $ | — |
|
U.S. Treasury notes | |
| — |
| |
| 190,768 |
| |
| — |
|
Total | | $ | 80,132 |
| | $ | 190,768 |
| | $ | — |
|
Liabilities | |
|
| |
|
| |
|
| |||
Contingent value right liability | | $ | — |
| | $ | — |
| | $ | 1,500 |
|
Total | | $ | — |
| | $ | — |
| | $ | 1,500 |
|
December 31, 2021 |
| Level 1 |
|
| Level 2 |
|
| Level 3 |
|
| Total |
| ||||
Assets |
|
|
|
|
|
|
|
|
|
|
|
| ||||
Money market funds in cash and cash equivalents |
| $ | 135,631 |
|
| $ | - |
|
| $ | - |
|
| $ | 135,631 |
|
| December 31, 2022 |
| ||||||||||
| Level 1 |
|
| Level 2 |
|
| Level 3 |
| ||||
Assets | |
|
|
|
|
|
|
|
| |||
Cash equivalents: | |
|
| |
|
| |
|
| |||
Money market funds | | $ | 40,783 |
|
| $ | — |
| | $ | — |
|
Total | | $ | 40,783 |
|
| $ | — |
|
| $ | — |
|
The valuesfair value of the Company’s Level 1 cash equivalents, are classified as Level 1 measurements under the fair value hierarchy as these assets have been valued usingconsisting of money market funds, is based on quoted market prices in active markets and do not have any restrictions on redemption. Aswith no valuation adjustment. The fair value of June 30, 2022 and December 31, 2021,the Company’s Level 2 cash equivalents, were comprisedconsisting of funds in money market accounts.U.S. treasury notes with original maturities of three months or less, is determined through third-party pricing services. The amortized cost of the U.S. treasury notes approximates the fair value. There werehave been no transfers or reclassifications between Level 1, Level 2impairments of the Company’s assets measured and Level 3carried at fair value during the three and six months ended June 30, 2022.
5. Accrued expenses2023 and other current liabilities
Accrued expenses2022. In addition, there were no changes in valuation techniques or transfers between Level 1, Level 2 and other current liabilities consisted of the following (in thousands):
|
| June 30, |
|
| December 31, |
| ||
Accrued external research and development |
| $ | 762 |
|
| $ | 4,031 |
|
Accrued payroll and benefits |
|
| 559 |
|
|
| 2,603 |
|
End of term charge for Term Loan |
|
| 389 |
|
|
| 0 |
|
Accrued professional fees |
|
| 186 |
|
|
| 233 |
|
Accrued interest |
|
| 9 |
|
|
| 17 |
|
|
| $ | 1,905 |
|
| $ | 6,884 |
|
6. Corporate restructuring
In October 2021, the Company announced a restructuring plan that resulted in a reduction of the Company’s workforce by 11 positions, or approximately 26% of the Company’s then workforce. A majority of these employees’ separation from the Company occurred in mid-October 2021, and the remaining affected employees transitioned by the end of 2021. As a result, the Company incurred costs of $1.4 million during the year ended December 31, 2021 related to severance benefits for the affected employees.
In February 2022, the Company announced an additional restructuring plan to reduce the Company's operations to preserveLevel 3 financial resources, resulting in a reduction of the Company’s workforce by 24 positions, or approximately 80% of the Company’s then workforce, by the end of the second quarter of 2022. As a result, the Company incurred costs of $1.7 millionassets during the three months ended March 31, 2022 related to severance benefits for the affected employees.
The severance benefits for both restructuring plans include severance payments, limited reimbursement of medical insurance premiums, outplacement services and other restructuring costs and expenses. Each affected employee’s eligibility for the severance benefits is contingent upon such employee’s execution (without revocation, as applicable) of a separation agreement, which includes a general release of claims against the Company. The October 2021 restructuring plan was completed by the end of 2021. The February 2022 restructuring plan was completed by the end of the second quarter of 2022.
Of the $1.7 million in costs recognized related to the February 2022 restructuring plan, $1.0 million and $0.7 million have been charged to research and development and general and administrative expenses, respectively, in the accompanying condensed consolidated statement of operations and comprehensive loss for the six months ended June 30, 2023 and 2022. DuringThe Company did not have any non-recurring fair value measurements on any assets or liabilities during the three and six months ended June 30, 2022, the Company paid $2.2 million in severance benefits to separating employees related to the restructuring plans, with $0.9 million related to the October 2021 restructuring plan2023 and $1.3 million related to the February 2022 restructuring plan. As of June 30, 2022 and December 31, 2021, unpaid severance costs of $0.4 million and $0.9 million, respectively, are included in accrued expenses and other current liabilities in the accompanying condensed consolidated balance sheet for each period.2022.
1013
7. Term loan
In February 2019,May 2021, the Company entered into a term loan facility of up to $10.0 millionlicense agreement (the “Term Loan”“Roche Agreement”) with Silicon Valley Bank (“SVB”F. Hoffmann-La Roche Ltd. and Hoffmann-La Roche Inc. (together, “Roche”). pursuant to which Roche granted the Company an exclusive and sublicensable worldwide license under certain patent rights and know-how to develop, manufacture and commercialize certain compounds (the “Compounds”) as further described in Note 8. The proceeds were used for general corporate and working capital purposes. ConcurrentCompany recognized a liability in connection with the Term Loan, the Company issued SVB warrantsRoche Agreement which included an obligation to purchase 15,257issue a variable number of shares of the Company’s Series A preferredcommon stock atto Roche for no additional consideration upon the Company’s completion of an exercise priceinitial public offering or certain merger transactions, a “Roche Qualified Transaction.” Prior to settlement in Q4 2022, the fair value measurement of $5.46. Asthe derivative liability was classified as Level 3 under the fair value hierarchy as it was valued using certain unobservable inputs. These inputs included: (1) the Company’s estimated shares outstanding and fair value per share upon completion of June 30, 2022a Roche Qualified Transaction and December 31, 2021,(2) the probability of the Company had $2.9 million and $5.4 million, respectively, in principal outstanding under the Term Loan.
The Term Loan is governed bycompleting a loan and security agreement, entered into in February 2019, between the Company and SVB (the “SVB Loan Agreement”). The SVB Loan Agreement provided for 2 separate tranches under which the Company could borrow. In April 2019, the Company borrowed $7.5 million under the first tranche, and in December 2019, the Company borrowed $2.5 million under the second tranche.
The Term Loan initially matured in July 2022 and accrues interest at a floating rate per annum equal to the greater of 3.75% or the prime rate minus 1.5% (3.25% as of June 30, 2022). The Term Loan initially provided for monthly interest-only payments until July 2020. Thereafter, payments are payable in equal monthly installments of principal, plus all accrued and unpaid interest.Roche Qualified Transaction. The Company may prepaysettled the Term Loan in whole upon 5 days’ prior written noticederivative liability by issuing common stock to SVB. Any such prepaymentRoche immediately following the completion of the Term Loan is subjectmerger. The number of shares of common stock to a prepayment charge ofbe issued to Roche was estimated to be approximately 0.52.85% of the then outstanding principal balance. Amounts outstanding during an event of default are payable upon SVB’s demand and will accrue interest at an additional rate of 5.0% per annum of the past due amount outstanding.
In April 2020, the Company entered into a deferral agreement with SVB to defer scheduled principal repayments on its Term Loan by six months. The deferral agreement was offered to the Company in connection with SVB’s venture debt relief initiative, which was started due to the COVID-19 pandemic. The Company’s first principal payment under its credit facility occurred in February 2021. The required monthly interest-only payment was not impacted by the deferral. The Term Loan’s new maturity date is in January 2023. After considering the debt guidance in ASC 470, the Company concluded that it did not meet the indicators of a troubled debt restructuring and accounted for the deferral of principal payment as a debt modification. Since there were no fees paid to SVB in connection with the deferral agreement, the Company did not record any adjustments to the Company’s condensed consolidated financial statements related to this deferral.
At the end of the loan term (whether at maturity, by prepayment in full or otherwise), the Company is required to pay a final end of term charge to SVB in the amount of 4.0% of the aggregate original principal amount advanced by SVB. The amount of the end of term charge is being accrued over the loan term as interest expense. As of June 30, 2022 and December 31, 2021, the Company had a liability related to the end of term charge of $0.4 million, which has been classified within accrued expenses and other current liabilities as of June 30, 2022 and other long-term liabilities as of December 31, 2021.
The SVB Loan Agreement includes a provision under which SVB may accelerate the scheduled maturities of the Term Loan under conditions that are not objectively determinable. The Company evaluated the likelihood of such acceleration and determined that it is not probable and classified the Term Loan on the balance sheet in accordance with the repayment schedule as of June 30, 2022.
As of June 30, 2022, scheduled principal payments for the Term Loan are as follows (in thousands):
Year Ending December 31, |
|
|
| |
2022 (remaining six months) |
| $ | 2,500 |
|
2023 |
|
| 417 |
|
Total principal |
|
| 2,917 |
|
Unamortized discounts |
|
| (4 | ) |
Carrying amount |
|
| 2,913 |
|
Less current portion |
|
| (2,913 | ) |
Long-term portion |
| $ | 0 |
|
Interest expense for the Term Loan was $0.1 million for each of the three months ended June 30, 2022 and 2021 and $0.1 million and $0.3 million for the six months ended June 30, 2022 and 2021, respectively.
8. Convertible promissory notes
In August 2020, Old Gemini entered into a purchase agreement with various investors to issue $14.0 million in convertible promissory notes (the “Notes”). The Notes accrued simple interest at 8% per annum. The Company determined that a beneficial conversion feature (“BCF”) existed and should be recognized on the issuance date. The Company recorded the Notes at the original issuance price, net of
11
the BCF discount. The BCF discount was accreted to the face value of the Notes over the period from the issuance date until the maturity date, offset against interest expense.
The Notes served as a bridge loan prior to the PIPE Financing that was completed in connection with the closing of the Business Combination. The Notes were intended to automatically convert into shares of common stock issuedof the combined company as of immediately after the completion of a Roche Qualified Transaction, including the exercise by the underwriters thereof of any overallotment option, if applicable. The Company remeasured the derivative liability based on the stock price of its publicly-traded common stock on December 29, 2022. The change in the PIPE Financing at a per share conversion price equal to the lowest per share price paid for such shares of common stock in the PIPE Financing. The Notes were amended to allowfair value for the principal and interest to convert to shares of Series B preferred stock prior to the closing of the Business Combination. Accordingly, immediately prior to the closing of the Business Combination, the outstanding principal and interest under the Notes converted into 2,341,316 shares of Series B preferred stock at a per share conversion price of $6.1986, and the Notes liabilityperiod was extinguished. The Company recorded a loss on conversion of convertible notes of $0.7 million for the difference between the reacquisition price of the Notes and the net carrying amount of the Notes in the condensed consolidated statements of operations and comprehensive loss in the change in fair value of derivative liability. Upon completion of the merger, the Company issued 482,313 shares of common stock to Roche, thereby settling the derivative liability, with the fair value of the common stock at the time of issuance recorded as additional paid-in capital.
As described in Note 1, in connection with the merger, the stockholders of Gemini at the Effective Time received a CVR to receive consideration from the Company upon its receipt of certain proceeds, resulting from a disposition of Gemini’s pre-merger assets within one year after the closing of the merger, calculated in accordance with the CVR Agreement. The fair value of the CVR liability was $1.5 million as of June 30, 2023 and de minimis as of December 31, 2022. The fair value of the CVR liability was determined based on significant inputs not observable in the market, which represents a Level 3 measurement within the fair value hierarchy. The Company used a discounted cash flow approach to value the CVR liability. As inputs into the valuation, the Company considered the probabilities of success of certain potential payments, the amount of the payments, and a discount rate of 13.0% determined using an implied credit spread adjusted based on companies with similar credit risk.
The following table provides a summary of changes in fair value of the Level 3 liabilities for the three months ended March 31, 2021.
Interest expense for the Notes was $0 for each of the threeand six months ended June 30, 2023 and 2022 (in thousands):
|
| Three and Six Months Ended |
|
| Three and Six Months Ended |
| ||
|
| Contingent value right liability |
|
| Derivative liability |
| ||
Balance at December 31 |
| $ | — |
|
| $ | 6,450 |
|
Change in fair value |
|
| — |
|
|
| (100 | ) |
Balance at March 31 |
|
| — |
|
|
| 6,350 |
|
Change in fair value |
|
| 1,500 |
|
|
| (2,390 | ) |
Balance at June 30 |
| $ | 1,500 |
|
| $ | 3,960 |
|
5. Cash, Cash Equivalents and 2021Restricted Cash
Cash, cash equivalents and restricted cash consisted of the following (in thousands):
| June 30, |
|
| December 31, |
| |||
| 2023 |
| | 2022 |
| |||
Cash and cash equivalents | | $ | 377,602 |
|
| $ | 194,611 |
|
Restricted cash | |
| 116 |
| |
| 177 |
|
Total cash, cash equivalents and restricted cash as shown on the condensed | | $ | 377,718 |
|
| $ | 194,788 |
|
14
6. Property and Equipment, Net
Property and equipment, net consisted of the following (in thousands):
| June 30, |
|
| December 31, |
| |||
| 2023 |
|
| 2022 |
| |||
Computer equipment |
| $ | 210 |
|
| $ | 169 |
|
Furniture and fixtures |
|
| 144 |
|
|
| 144 |
|
Less: Accumulated depreciation |
|
| (195 | ) |
|
| (145 | ) |
Property and equipment, net |
| $ | 159 |
|
| $ | 168 |
|
7. Accrued Expenses
Accrued expenses consisted of the following (in thousands):
| June 30, |
|
| December 31, |
| |||
| 2023 |
|
| 2022 |
| |||
Accrued research and development |
| $ | 1,617 |
|
| $ | 1,817 |
|
Accrued employee-related expenses |
|
| 2,122 |
|
|
| 3,623 |
|
Accrued professional fees |
|
| 220 |
|
|
| 463 |
|
Accrued other |
|
| 189 |
|
|
| 206 |
|
Total accrued expenses |
| $ | 4,148 |
|
| $ | 6,109 |
|
8. Development and License Agreements
License Agreement and Master Service Agreement with Aurigene Discoveries Technology Limited (“Aurigene”)
In February 2018, the Company entered into a license agreement with Aurigene, pursuant to which Aurigene granted the Company an exclusive worldwide license, with the right to grant sublicenses, to certain Aurigene intellectual property. Concurrent with the execution of the Aurigene license agreement, the parties entered into a master services agreement, which provided for Aurigene to provide future development services to the Company on a full-time equivalent cost basis and consumable costs incurred basis. In December 2022, the Company provided a 90-day notice to terminate the master service agreement effective in March 2023.
Pursuant to the license agreement, the Company agreed to pay an upfront fee of $00.1 million and annual maintenance fees up to $1.70.2 million for the licensed intellectual property. The Company may also be obligated to make future milestone payments of up to $7.1 million for the first licensed product based on the achievement of certain development and regulatory milestones. The term of the license agreement expires on a licensed product-by-licensed product and country-by-country basis on the expiration of the last-to-expire valid claim under the licensed intellectual property rights in such country. The Company can terminate the agreement, for convenience, with 90 days’ notice to Aurigene. The agreement can also be terminated by either party due to insolvency or by Aurigene due to a material breach after a specified cure period.
During the three and six months ended June 30, 2023, the Company recorded research and development expense related to its arrangements with Aurigene of $0.3 million and $0.3 million, respectively, compared to expense for the three and six months ended June 30, 2022 of $0.2 million and 2021,$0.4 million, respectively.
License and Stock Purchase Agreement with AbbVie Deutschland GmbH & Co. KG (“AbbVie”)
9. Stockholders’ EquityIn September 2019, the Company entered into an agreement with AbbVie, pursuant to which AbbVie granted the Company an exclusive license, with the right to grant sublicenses, to certain AbbVie intellectual property.
Under this agreement, the Company paid a non-refundable, non-creditable upfront fee of $0.6 million. The Company is also obligated to make future payments upon the achievement of certain development, commercialization and sales-based milestones up to $18.0 million, $45.0 million and $87.5 million, respectively on a licensed product-by-licensed product basis. In addition, the Company is also obligated to pay royalties based on net sales of the licensed products on a licensed product-by-licensed product and country-by-country basis. As of June 30, 2023, none of the milestones had been achieved.
The condensed consolidated statementCompany’s royalty obligation expires on a licensed product-by-licensed product and country-by-country basis upon the expiration of stockholders’ equity has been retroactively adjustedthe last-to-expire valid claim under the licensed intellectual property rights in such country. Unless terminated earlier, the agreement expires upon the expiration of the Company’s royalty obligation for all periods presentedlicensed products. AbbVie can terminate the agreement if the Company fails to reflectmake any payments within a specified period after receiving written notice of such failure, or in the Business Combinationevent of a material breach by the Company and reverse recapitalization as definedfailure to cure such breach within a certain period of time.
15
License Agreement with Roche
In connection with the Roche Agreement, the Company paid Roche an upfront, non-refundable exclusivity payment of $0.5 million in March 2021. Upon execution of the Roche Agreement in May 2021, the Company paid Roche an additional upfront, non-refundable payment of $4.0 million.
The Company is obligated to make contingent payments to Roche totaling up to $205.0 million upon achievement of certain development, regulatory and commercial milestones. Roche is also eligible to receive tiered royalties on net sales of commercialized products, at rates ranging from high single-digits to high teens.
A description of the derivative liability related to the Roche agreement settled in 2022 is included in Note 2, Business Combination.4.
License Agreement with Mabwell
In January 2023, the Company entered into an exclusive license agreement with Mabwell Therapeutics, Inc. (“Mabwell”), pursuant to which Mabwell granted the Company an exclusive and sublicensable license to certain Mabwell intellectual property.
In connection with the agreement, the Company paid Mabwell an upfront payment of $10.0 million in March 2023. In addition, the Company is obligated to pay certain development and regulatory milestone payments for the licensed products, for up to three indications, up to a maximum aggregate amount of $127.5 million, as well as certain commercial milestone payments for certain licensed product net sales achievements, up to a maximum aggregate amount of $275.0 million. The Company is further obligated to pay a tiered percentage of revenue that the Company receives from its sublicensees ranging from a low third decile percentage to a low first decile percentage. In addition, the Company is obligated to pay Mabwell a royalty on annual net sales of all licensed products at a tiered rate ranging from low single-digits to high single-digits.
9. Convertible Preferred Stock
Upon closingAs of the Business Combination and pursuant to the terms of the Company's Amended and Restated Certificate of Incorporation entered into on February 5, 2021 (the “Certificate of Incorporation”),June 30, 2023, the Company was authorized to issue up to 10,000,000 shares of preferred stock with a par value $0.0001 per share. The Company’s board of directors has the authority, without further action by the stockholders, to issue such shares of preferred stock in one or more series, to establish from time to time the number of shares to be included in each such series, and to fix the designations, powers, voting, and other rights, preferences and privileges of the shares. There were 0 issued and outstanding shares of preferred stock as of June 30, 2022.
In connection with the closing of the Business Combination, all previously issued and outstanding Series A convertible preferred stock and Series B convertible preferred stock were exchanged for common stock of the Company pursuant to the Conversion Ratio established in the Merger Agreement. All fractional shares were rounded down.
Common Stock
Pursuant to the terms of the Company's Certificate of Incorporation, the Company authorized 250,000,000 shares of common stock withat a par value of $0.0001 per share., with no shares issued or outstanding.
Immediately prior to the Effective Time, each share of Private Disc’s preferred stock was converted into a share of Private Disc’s common stock. At the closing of the merger, the shares of Private Disc’s common stock were converted into shares of the Company’s common stock based on the Exchange Ratio.
10. Common Stock
At the closing of the merger, the shares of Private Disc’s common stock were converted into shares of the Company’s common stock based on the Exchange Ratio.
As discussed in Note 2, Business Combination,of June 30, 2023, the authorized capital stock of the Company has retroactively adjusted the shares issued and outstanding prior to February 5, 2021 to give effect to the Conversion Ratio established in the Merger Agreement to determine the number ofincluded 100,000,000 shares of common stock, into which they$0.0001 par value per share. Prior to the merger, the holders of Private Disc’s common stock were converted.
Voting
subject to and qualified by the rights, powers and preferences of the holders of the Preferred Stock. Each share of common stock entitles the holder to one vote on all matters submitted to the stockholders for a votevote.
As of June 30, 2023, the Company has 2,571,395 shares of common stock reserved for the exercise of stock options and 1,433,305 shares of common stock reserved for the exercise of pre-funded warrants.
Registration Statements Resulting from the Merger
In January 2023, as a result of the Company’s stockholders.
Dividends
Common stockholders are entitled to receive dividends, as may be declared by the board of directors. NaN dividends have been declared to date.
12
10. Equity incentive plans
2017 Old Gemini Equity Incentive Plan
Old Gemini’s 2017 Stock Option and Grant Plan, as amended (the “2017 Plan”), provided formerger, the Company filed a resale registration statement on Form S-3 with the Securities and Exchange Commission (“SEC”), which covered the proposed resale or other disposition by certain stockholders of up to grant qualified incentive options, nonqualified options, stock grants and other stock-based awards to employees and non-employees to purchasean aggregate of 12,635,956 shares of the Company’s common stock. The 2017 Plan was administered byCompany also filed a registration statement on Form S-8 with the board of directors, or at the discretion of the board of directors, by a committee of the board of directors.SEC, which registered
The exercise price for incentive options was determined at the discretion of the board of directors. All incentive options granted to any person possessing less than 10% of the total combined voting power of all classes of stock may not have an exercise price of less than 1,672,599100% of the fair market value of the common stock on the grant date. All incentive options granted to any person possessing more than 10% of the total combined voting power of all classes of stock may not have an exercise price of less than 110% of the fair market value of the common stock on the grant date.
The option term for incentive awards may not be greater than ten years from the date of the grant. Incentive options granted to persons possessing more than 10% of the total combined voting power of all classes of stock may not have an option term of greater than five years from the date of the grant. The vesting period for equity-based awards under the 2017 Plan was determined at the discretion of the board of directors, which was generally four years. For awards granted to employees and non-employees with four-year vesting terms, 25% of the options vest on the first anniversary of the grant date and the remaining options vest equally each month for three years thereafter.
Upon completion of the Business Combination, the Company ceased granting awards under the 2017 Plan.
Conversion of Awards
Each Old Gemini option from the 2017 Plan and each option from Old Gemini’s 2015 Stock Option and Grant Plan (the “2015 Plan”) that was outstanding immediately prior to the Business Combination, whether vested or unvested, was converted into an option to purchase a number of shares of common stock (each such option, an “Exchanged Option”) equalissuable with respect to Private Disc options assumed by the Company pursuant to the product (rounded downMerger Agreement as well as 2,035,103 additional shares of common stock reserved and available for future issuance under the 2021 Plan and 180,894 additional shares of common stock reserved and available for future issuance under the 2021 ESPP.
ATM Program
In January 2023, the Company filed a shelf registration statement on Form S-3 with the SEC, which covered the offering, issuance and sale by the Company of up to an aggregate of $300.0 million of the Company’s common stock, preferred stock, debt securities, warrants or units. Subsequently in January 2023, the Company entered into a Sales Agreement (the “Sales Agreement”) with SVB Securities LLC, as sales agent, to provide for the offering, issuance and sale by the Company of up to $100.0 million of the Company’s common stock from time to time in “at-the-market” offerings under the shelf. Under this program, the Company is able to offer and sell, from time to time at its sole discretion, shares of its common stock through SVB Securities LLC as its sales
16
agent. In an ATM offering, exchange-listed companies incrementally sell newly issued shares into the secondary trading market through a designated broker-dealer at prevailing market prices. Effective June 12, 2023, the ATM program was suspended. The Company will not make any sales of common stock pursuant to the nearest whole number)Sales Agreement unless and until a new prospectus supplement or registration statement is filed with the SEC; however, the Sales Agreement remains in full force and effect. In connection with the ATM suspension, the Company recognized the remaining capitalized issuance costs of (i)$0.3 million as general and administrative expense in the consolidated statements of operations and comprehensive loss for the three and six months ended June 30, 2023.
As of June 30, 2023, the Company had sold an aggregate of 825,350 shares of common stock in at-the-market offerings under the shelf. Aggregate gross proceeds from the transactions were $20.0 million and the Company received $19.5 million in net proceeds, after deducting placement agent fees and offering expenses.
Registered Direct Offering
In February 2023, the Company entered into a securities purchase agreement, with certain investors. Pursuant to the securities purchase agreement, the Company sold an aggregate of 1,488,166 shares of the Company’s common stock, at a purchase price of $23.00 per share, and with respect to a certain investor, in lieu of shares of the Company’s common stock, pre-funded warrants to purchase an aggregate of 1,229,224 shares of the Company’s common stock, at a purchase price of $22.9999 per pre-funded warrant, for aggregate net proceeds of $62.4 million, after deducting offering expenses of $0.1 million.
The pre-funded warrants provide that the holder will not have the right to exercise any portion of its warrants if such holder, together with its affiliates, would beneficially own in excess of 9.99% of the number of shares of Old Geminithe Company’s common stock subjectoutstanding immediately after giving effect to such Old Gemini option immediately priorexercise (the “Beneficial Ownership Limitation”); provided, however, that the holder may increase or decrease the Beneficial Ownership Limitation by giving 61 days’ notice, but not to the Business Combination and (ii) the Conversion Ratio, at an exercise price per share (rounded up to the nearest whole cent) equal to (A) the exercise price per shareany percentage in excess of such Old Gemini option immediately prior to the consummation19.99%. The investors or their affiliates are beneficial holders of more than 5% of the Business Combination, divided by (B)Company’s capital stock. The pre-funded warrants meet the Conversion Ratio. Each Exchanged Option will continue to be governed by the same termscondition for equity classification and conditions (including vesting and exercisability terms)were therefore recorded as were applicable to the corresponding former Old Gemini option immediately prior to the consummationa component of the Business Combination. All stock option activity was retroactively restated to reflect the Exchanged Options.stockholders’ equity within additional paid-in capital.
As of the Closing Date, the 10,567,508 options and 163,157 restricted stock units (“RSUs”) outstanding under the 2017 Plan and 2015 Plan were converted into 2,303,309 options and 35,561 RSUs, respectively, upon completion of the Business Combination after the effect of the Conversion Ratio. This effect of the Conversion Ratio has been retroactively adjusted throughout the Company’s unaudited condensed consolidated financial statements.
2021 Gemini Equity Incentive PlanFollow-On Public Offering
In February 2021, FSDC’s stockholders approvedJune 2023, the Company issued 3,015,919 shares of its common stock upon the completion of its public follow-on offering, which included the exercise in full by the underwriters of their option to purchase up to 420,000 additional shares of common stock, at a public offering price of $49.00 per share. The Company also sold, in lieu of shares of the Company’s common stock, pre-funded warrants to purchase an aggregate of 204,081 shares of common stock at a price of $48.9999 per pre-funded warrant. The Company received aggregate net proceeds of $147.9 million, after deducting offering expenses of $9.9 million.
The pre-funded warrants provide that the holder will not have the right to exercise any portion of its warrants if such holder, together with its affiliates, would beneficially own in excess of 24.99% of the number of shares of Common Stock outstanding immediately after giving effect to the exercise (the “Ownership Limit”). Purchasers of the pre-funded warrants may also elect to set the initial Ownership Limit at 4.99%, 9.99% or 19.99%. Upon at least 61 days’ prior notice from the holder to the Company, the holder may increase or decrease the Ownership Limit up to 24.99%, provided however that purchasers that select an Ownership Limit of 19.99% or less will only be allowed to increase the Ownership Limit above 19.99% if such increase would not result in a change of control under the rules and regulations of the Nasdaq Stock Market LLC. The pre-funded warrants meet the condition for equity classification and were therefore recorded as a component of stockholders’ equity within additional paid-in capital.
11. Stock-Based Compensation
The Company grants stock-based awards under its 2021 Stock Option and Incentive Plan (the “2021 Plan”), pursuant to which 4,264,341 shares of common stock were reserved for issuance.was approved by its stockholders in February 2021 and amended and restated in January 2023. The 2021 Plan provides for the Company to grant incentive stock options or nonqualified stock options for the purchase of common stock, stock appreciation rights, restricted stock awards, restricted stock units, unrestricted stock awards, cash-based awards and dividend equivalent rights to employees, officers, directors and consultants of Gemini. Incentive stock options may only be granted to employees. The 2021 Plan is administered by the plan administrator, which is the Compensation Committee of Gemini’s board of directors, provided therein, whichalso has discretionary authority, subject only to the express provisions of the 2021 Plan, to interpret the 2021 Plan; determine eligibility for and grant awards; determine form of settlement of awards (whether in cash, shares of stock, other property or a combination of the foregoing), determine, modify or waive the terms and conditions of any award; prescribe forms, rules and procedures; and otherwise do all things necessary to carry out the purposes of the 2021 Plan. The number of shares of common stock reserved for issuance under the 2021 Plan automatically increases on January 1 of each calendar year, starting on January 1, 2022 and continuing through January 1, 2031, in an amount equal to 4% of the total number of shares of the Company’s capital stock outstanding on the last day of the calendar month before the date of each automatic increase, or a lesser number of shares determined by the Company’s board of directors. Subject to this provision, the Company added 1,728,326 shares available for grant to the 2021 Plan effective January 1, 2022. As of June 30, 2022, 2,162,435 shares remained available for future issuance under the 2021 Plan.
The exercise price of each stock option grantedawards under its 2017 Stock Option and Grant Plan (the “Private Disc Plan”), the 20212017 Stock Option and Grant Plan will be 100% of the fair market value of the underlying stock subject to the award, determined as of the date of the grant, or such higher amount as the plan administrator may determine in connection with
13
the grant,(the “Gemini 2017 Plan”), and the term of stock option may not be greater than ten years. The vesting and other restrictions are determined at the discretion of the plan administrator.
2021 Inducement Plan
In February 2021, the Company’s board of directors approved the 2021 Inducement Plan. The 2021 Inducement Plan is a non-stockholder approved stock plan under which the Company grants equity awards to induce highly-qualified prospective officers and employees who are not currently employed by the Company to accept employment and provide them with a proprietary interest in the Company. The Company intends that the 2021 Inducement Plan, be reserved for personsbut is no longer granting awards under these plans. The Company also has the option to whom the Company may issue securities without stockholder approval as an inducement pursuant to Rule 5635(c)(4) of the Marketplace Rules of the NASDAQ Stock Market, Inc. The 2021 Inducement Plan is administered by the board of directors or the Compensation Committee of the board, which determines the types ofgrant awards to be granted, including the number of shares subject to the awards, the exercise price and the vesting schedule. Awards granted under the 2021 Inducement Plan expire no later than ten years from the date of grant. As of June 30, 2022, 983,949 shares were available for issuance under the 2021 Inducement Plan.
2021 Employee Stock Purchase Plan
In July 2021, the Company’s board of directors approved the 2021 Employee Stock Purchase Plan (“(the “2021 ESPP”), which was approved by shareholders in July 2021 ESPP”). and amended and restated in January 2023.
17
The first offeringfollowing table summarizes stock option activity for the six months ended June 30, 2023.
| Number of |
| | Weighted- |
| | Weighted- |
| | Aggregate |
| |||||
Outstanding at December 31, 2022 | |
| 2,640,590 |
| | $ | 16.19 |
| |
| 8.08 |
| | $ | 25,513 |
|
Granted | |
| 117,463 |
| | $ | 35.36 |
| |
|
| |
|
| ||
Exercised | |
| (116,228 | ) | | $ | 11.94 |
| |
|
| |
|
| ||
Forfeited | |
| (70,430 | ) | | $ | 102.56 |
| |
|
| |
|
| ||
Outstanding at June 30, 2023 | |
| 2,571,395 |
| | $ | 14.90 |
| |
| 7.98 |
| | $ | 82,589 |
|
Exercisable at June 30, 2023 | |
| 1,191,600 |
| | $ | 16.57 |
| |
| 7.01 |
| | $ | 39,795 |
|
The aggregate intrinsic value of options is calculated as the difference between the exercise price of the stock options and the fair value of the Company’s common stock for those stock options that had exercise prices lower than the fair value of the common stock as of the end of the period. The aggregate intrinsic value of stock options exercised during the six months ended June 30, 2023 was $2.0 million.
The weighted-average assumptions used to estimate the fair value of stock options granted were as follows:
| Six Months Ended |
| ||||||
| 2023 |
| | 2022 |
| |||
Risk-free interest rate | |
| 3.67 | % | |
| 1.99 | % |
Expected term (in years) | |
| 7.73 |
| |
| 6.00 |
|
Expected volatility | |
| 58 | % | |
| 56 | % |
Expected dividend yield | |
| 0 | % | |
| 0 | % |
Fair value per share of common stock | | $ | 35.36 |
| | $ | 14.69 |
|
The weighted-average grant date fair value of options granted during the six months ended June 30, 2023 and 2022 was $22.49 and $7.85 per share, respectively.
The total fair value of options vested during the six months ended June 30, 2023 was $2.1 million.
As of June 30, 2023, the Company had issued a total of 63,061 shares of restricted common stock to the founders of Private Disc pursuant to subscription agreements and to certain key employees pursuant to the Private Disc Plan at $0.0001 per share. The stock restrictions relate to the sale and transferability of the stock and lapse over the defined vesting period underin the 2021 ESPP began on December 1, 2021.restricted stock agreement. The vesting period is generally contingent upon continued employment or consulting services being provided to the Company. In the event of termination, the Company has the right, but not yet issued anythe obligation to repurchase the unvested shares underat the 2021 ESPP. The Companyoriginal purchase price.
A summary of restricted common stock activity is as follows:
| Six Months Ended |
| ||||||
| 2023 |
| | 2022 |
| |||
Unvested at the beginning of the year | |
| 1,916 |
| |
| 10,170 |
|
Vested | |
| (1,916 | ) | |
| (4,570 | ) |
Unvested at the end of the period | |
| — |
| |
| 5,600 |
|
As of June 30, 2023, all awards of restricted common stock were fully vested.
Stock-Based Compensation Expense
Total stock-based compensation expense recorded $as research and development and general and administrative expenses, respectively, for employees, directors and non-employees is as follows (in thousands):6
thousand and $22
| Three months ended |
|
| Six Months Ended |
| |||||||||||
| 2023 |
| | 2022 |
|
| 2023 |
| | 2022 |
| |||||
Research and development | | $ | 367 |
| | $ | 149 |
|
| $ | 712 |
| | $ | 282 |
|
General and administrative | |
| 942 |
| |
| 233 |
|
|
| 1,621 |
| |
| 432 |
|
Total stock-based compensation expense | | $ | 1,309 |
| | $ | 382 |
|
| $ | 2,333 |
| | $ | 714 |
|
thousand
18
As of June 30, 2023, the total unrecognized stock-based compensation expense related to the 2021 ESPPoutstanding awards was $10.5 million and is expected to be recognized over a weighted-average period of 2.74 years.
12. Income Taxes
The Company did not record a provision or benefit for federal income taxes during the three and six months ended June 30, 2022, respectively. As2023 and 2022. The Company recorded state income tax expense of less than $0.1 million for the three and six months ended June 30, 2022,2023, due to interest income. There was 420,591no shares remained availablestate income tax expense for future issuance under the 2021 ESPP.three and six months ended June 30, 2022. The numberCompany continues to maintain a full valuation allowance against all of sharesits deferred tax assets.
The Company has evaluated the positive and negative evidence involving its ability to realize its deferred tax assets and has considered its history of common stock reserved for issuance undercumulative net losses incurred since inception and its lack of any commercially ready products. The Company has concluded that it is more likely than not that it will not realize the 2021 ESPP automatically increases on January 1benefits of its deferred tax assets. The Company reevaluates the positive and negative evidence at each calendar year, starting on January 1, 2023 and continuing through January 1, 2031, in an amount equal to the least of (a) reporting period.
1% of the total number of shares of the Company’s capital stock outstanding on the last day of the calendar month before the date of each automatic increase, (b) 430,551 shares of common stock, or (c) such number of shares determinedThe Company has never been examined by the Company’s boardInternal Revenue Service or any other jurisdiction for any tax years and, as such, all years within the applicable statutes of directors.limitations are potentially subject to audit.
13. Net Loss Per Share
Stock-based compensation expense
The Company recorded stock-based compensation expense in the following expense categories of its condensed consolidated statements of operationsBasic and comprehensive loss (in thousands):
|
| Three Months Ended |
|
| Six Months Ended |
| ||||||||||
|
| 2022 |
|
| 2021 |
|
| 2022 |
|
| 2021 |
| ||||
Research and development |
| $ | 268 |
|
| $ | 1,303 |
|
| $ | 677 |
|
| $ | 1,959 |
|
General and administrative |
|
| 2,088 |
|
|
| 1,559 |
|
|
| 3,655 |
|
|
| 2,496 |
|
Total stock-based compensation expense |
| $ | 2,356 |
|
| $ | 2,862 |
|
| $ | 4,332 |
|
| $ | 4,455 |
|
11. Netdiluted net loss per share
is computed by dividing net loss attributable to common stockholders by the weighted-average common shares outstanding. The following table summarizesweighted-average common shares outstanding used in the computation of basic and diluted net loss per share attributable to common stockholderscalculation includes the pre-funded warrants issued in connection with the Company’s follow-on public offering in June 2023 and registered direct offering in February 2023 as the pre-funded warrants are exercisable for nominal cash consideration. As of the Company (in thousands, except shareJune 30, 2023, no pre-funded warrants have been exercised and per share amounts):
1,433,305 pre-funded warrants are outstanding.
|
| Three Months Ended |
|
| Six Months Ended |
| ||||||||||
|
| 2022 |
|
| 2021 |
|
| 2022 |
|
| 2021 |
| ||||
Net loss attributable to common stockholders |
| $ | (7,090 | ) |
| $ | (16,447 | ) |
| $ | (22,321 | ) |
| $ | (35,495 | ) |
Weighted average common shares outstanding, basic and diluted |
|
| 43,230,523 |
|
|
| 43,041,856 |
|
|
| 43,221,712 |
|
|
| 37,564,936 |
|
Net loss per share attributable to common stockholders, basic and diluted |
| $ | (0.16 | ) |
| $ | (0.38 | ) |
| $ | (0.52 | ) |
| $ | (0.94 | ) |
The Company’s unvested restricted common shares have been excluded fromCompany has generated a net loss in all periods presented, so the computation of basic and diluted net loss per share attributable to common stockholders.
The Company’sare the same, as the inclusion of the potentially dilutive securities which include unvested restricted stock and common stock options outstanding, have beenwould be anti-dilutive. The Company excluded the following from the computation of diluted net loss per share attributable to common stockholders as the effect would be to reduce the net loss per share attributable to common stockholders. Therefore, the weighted average number of common shares outstanding used to calculate both basic and diluted net loss per share attributable to common stockholders is the same. The Company excluded the
14
following potential common shares from the computation of diluted net loss per share attributable to common stockholders for the periods indicated because including them would have had an anti-dilutive effect:
|
| As of June 30, |
| |||||
|
| 2022 |
|
| 2021 |
| ||
Unvested restricted stock |
|
| 871,204 |
|
|
| - |
|
Common stock options outstanding |
|
| 4,807,045 |
|
|
| 6,506,146 |
|
|
|
| 5,678,249 | �� |
|
| 6,506,146 |
|
| June 30, |
| ||||||
| 2023 |
| | 2022 |
| |||
Series Seed convertible preferred stock | |
| — |
| |
| 5,000,000 |
|
Series A convertible preferred stock | |
| — |
| |
| 41,666,666 |
|
Series B convertible preferred stock | |
| — |
| |
| 37,499,999 |
|
Unvested restricted common stock | |
| — |
| |
| 5,600 |
|
Options to purchase common stock | |
| 2,571,395 |
| |
| 1,591,619 |
|
12.14. Commitments and contingencies
Commitments
The Company’s long-term contractual obligations include commitments entered into in the normal course of business. The Company’s most significant contracts relate to agreements with clinical research organizations (“CROs”) for clinical trials and preclinical studies and clinical manufacturing organizations (“CMOs”), which the Company enters into in the normal course of business. The contracts with CROs and CMOs are generally cancellable, with notice, at the Company’s option. The Company also has commitments related to debt obligations, license agreements and other purchase obligations.
License agreements
In April 2017, the Company entered into a Research Collaboration and License Agreement with Sanquin Blood Supply Foundation (“Sanquin”) (the “2017 License Agreement”) to develop antibodies that bind and enhance the activity of CFH. As consideration for the license, the Company paid a one-time, non-refundable upfront payment of $0.1 million. The 2017 License Agreement includes additional consideration upon the achievement of certain development and commercial milestones (i.e., once net sales targets exceed certain thresholds) totaling up to an aggregate amount of $29.0 million. Finally, the Company is required to make royalty payments of between 1.25% and 2.50% of net product sales if commercialization is achieved. On March 7, 2022, the Company entered into an amendment to the 2017 License Agreement (the “2022 Amendment”) to clarify that certain patent rights directed to CFH potentiating antibodies are jointly owned by the Company and Sanquin. Under the 2022 Amendment, Sanquin granted the Company an exclusive (even as to Sanquin) royalty-bearing license, with the right to sublicense through multiple tiers, to the portion of these patent rights owned by Sanquin. The condensed consolidated financial statements as of June 30, 2022 do not include liabilities with respect to this agreement as the Company has not yet generated revenue and the achievement of certain milestones is not deemed probable.
In June 2018, the Company entered into a Cell Line License Agreement with Life Technologies Corporation (the “2018 License Agreement”) to obtain non-exclusive use of 293 H cells in support of GEM-103 manufacturing activities. As consideration for the license, the Company paid a one-time, non-refundable, non-creditable initial license fee of $0.1 million. In addition, an annual non-refundable, non-creditable development fee of $0.1 million is due on each anniversary date. The 2018 License Agreement includes additional consideration of $0.3 million contingent upon future commercialization of each licensed product. The condensed consolidated financial statements as of June 30, 2022 do not include a liability with respect to the additional consideration under this agreement as the Company has not yet generated revenue.
In March 2019, the Company entered into a second Cell Line License Agreement with Life Technologies Corporation (the “2019 License Agreement”) to obtain non-exclusive use of a CTS Viral Production cell line for producing genetically engineered adeno-associated virus particles to be used in human therapeutics. In October 2021, the Company terminated the 2019 License Agreement. As consideration for the license, the Company paid a one-time, non-refundable, non-creditable initial license fee of $0.1 million. In addition, an annual non-refundable, non-creditable development fee of $0.1 million was due on each anniversary date, beginning on the second anniversary date. The 2019 License Agreement included additional consideration of $0.4 million contingent upon future commercialization of each licensed product. The condensed consolidated financial statements as of June 30, 2022 do not include a liability with respect to the additional consideration under this agreement as the Company has not yet generated revenue.
In October 2018, the Company entered into a Master License Agreement with Avitide, Inc. (the “2018 Master License Agreement”) to license, on an exclusive basis, certain of Avitide’s affinity chromatography resins comprised of proprietary ligands for affinity purification of biopharmaceuticals. As consideration for the license, the Company paid an upfront license fee of $0.2 million. In addition, an annual license fee of $0.1 million is due on each anniversary date. The 2018 Master License Agreement includes additional consideration upon the achievement of certain development, commercial and sales milestones totaling up to $0.7 million, $2.2 million and $7.0 million, respectively. Finally, the Company is required to make royalty payments of 1.25% of net product sales if commercialization is achieved. The condensed consolidated financial statements as of June 30, 2022 do not include liabilities with respect to additional consideration under this agreement as the Company has not yet generated revenue and the achievement of certain milestones is not deemed probable.
In June 2019, the Company entered into a GPEx-Derived Cell Line Sale Agreement with Catalent Pharma Solutions, LLC (the “2019 Sale Agreement”) to purchase all right, title and interest in and to the GPEx Cell Line. As consideration for the GPEx Cell Line, theContingencies
15
Company is required to make one-time milestone payments totaling up to $Indemnification Agreements1.3 million in aggregate, as well as a contingent annual fee upon commercialization (1% of net sales, or $0.1 million, whichever is greater) and other fees after certain milestones are reached. Certain milestone payments may be waived if Catalent manufactures >50% of the total product required for the relevant clinical trial. The condensed consolidated financial statements as of June 30, 2022 do not include liabilities with respect to this agreement as the Company has not yet generated revenue and the achievement of certain milestones is not deemed probable.
Contingencies
Indemnification agreements
In the ordinary course of business, the Company may provide indemnification of varying scope and terms to its vendors, lessors, contract research organizations, business partners and other parties with respect to certain matters including, but not limited to, losses arising out of breach of such agreements or from intellectual property infringement claims made by third parties. In addition, the Company has entered into indemnification agreements with members of its board of directors and executive officersBoard that will require the Company, among other things, to indemnify them against certain liabilities that may arise by reason of their status or service as directors or officers.directors. The maximum potential amount of future payments the Company could be required to make under these indemnification agreements is, in many cases, unlimited. To date, theThe Company has not incurred any material costs as a result of such indemnifications. The Companyindemnifications and is not currently aware of any indemnification claims.
Legal Proceedings
The Company, from time to time, may be party to litigation arising in the ordinary course of business. The Company was not subject to any material legal proceedings during the six months ended June 30, 2023 and 2022 and, to the best of its knowledge, no material legal proceedings are currently pending or threatened.
Payments Upon Termination
The Company enters into contracts in the normal course of business with CROs, CDMOs and other third parties for preclinical studies, clinical trials and manufacturing services. These contracts typically do not contain minimum purchase commitments and are generally cancelable by the Company upon written notice. Payments due upon cancellation consist of payments for services provided or expenses incurred, including noncancelable obligations of the Company’s service providers, up to the date of
19
cancellation and, in the case of certain arrangements could havewith CROs and CDMOs, may include noncancelable fees. Under such agreements, the exact amounts owed by the Company in the event of termination will be based on the timing of the termination and the exact terms of the agreement.
15. Leases
In October 2021, Private Disc entered into a material effectfive-year lease through November 2026 of 7,566 square feet of office space located at 321 Arsenal Street, Watertown, Massachusetts to be used as its corporate headquarters. In June 2023, the lease was amended to allow the landlord to perform certain maintenance activities expected to be completed by the end of 2023 and to provide rent abatement of $0.2 million. The amendment was accounted for as a modification resulting in a new incremental borrowing rate and a reduction to the right-of-use asset and lease liability of $0.2 million.
In June 2023, the Company entered into a sublease for a term of approximately three and a half years through November 2026, consisting of 9,280 square feet of office space on its financial position, resultsthe second floor of operations orthe corporate headquarters in Watertown, Massachusetts. Fixed lease payments include base rent, subject to annual rent increases, and a management fee. Variable lease payments include the Company’s allocated share of costs incurred for real estate taxes, utilities, and other operating expenses applicable to the leased premises. Pursuant to the lease, the Company is also obligated to pay for certain administrative costs, taxes and operating expenses.
The components of lease expense were as follows (in thousands):
| Six Months Ended |
| ||||||
| 2023 |
| | 2022 |
| |||
Operating lease costs | | $ | 181 |
| | $ | 206 |
|
Variable lease costs | |
| 109 |
| |
| 94 |
|
Total lease expense | | $ | 290 |
| | $ | 300 |
|
Other information related to the Company’s leases is as follows (in thousands, except term and discount rate amounts):
| Six Months Ended |
| ||||||
| 2023 |
| | 2022 |
| |||
Weighted average remaining lease term | | 3.42 years |
| | 4.41 years |
| ||
Weighted average discount rate |
|
| 9.9 | % | |
| 5.5 | % |
Cash paid for amounts included in the measurement of lease liabilities: | |
|
| |
|
| ||
Operating cash flows used in operating leases | | $ | 187 |
| | $ | 181 |
|
A maturity analysis of the annual undiscounted cash flows reconciled to the carrying value of the operating lease liabilities as of June 30, 2023, reflective of the Company’s election to account for lease and itnon-lease components together, is as follows (in thousands):
Year Ending December 31, | | Operating Leases |
| |
Remaining 2023 | | $ | 149 |
|
2024 | |
| 789 |
|
2025 | |
| 850 |
|
2026 | |
| 729 |
|
Total minimum lease payments | |
| 2,517 |
|
Less imputed interest | |
| (424 | ) |
Present value of lease liabilities | | $ | 2,093 |
|
16. Related Party Transactions
The landlord of the Company’s leased office space in Watertown, Massachusetts is a related party of the Company due to its equity ownership.
In February 2023, certain existing investors participated in the Company’s registered direct offering (see Note 10).
In March 2023, the Company executed a promissory note for an aggregate principal amount of $0.5 million from an existing investor. The Company did not use these funds and repaid the note four days later, recording a de minimis amount of interest expense based on the then Federal funds rate for short term loans of 4.5% per annum.
20
In June 2023, an existing investor participated in the Company’s follow-on offering (see Note 10).
17. Subsequent Events
The Company has not accrued any liabilities relatedcompleted an evaluation of all subsequent events after the unaudited condensed consolidated balance sheet date of June 30, 2023 through the date these condensed consolidated financial statements were issued to such obligationsensure that these condensed consolidated financial statements include appropriate disclosure of events both recognized in itsthe condensed consolidated financial statements as of June 30, 2022.
Legal proceedings
From time to time, the Company may become involved in legal proceedings arising2023, and events which occurred subsequently but were not recognized in the ordinary course of business. As of June 30, 2022, thecondensed consolidated financial statements. The Company was not a party to any material legal matters or claims.had no non-recognizable subsequent events.
13. Related party transactions
The Company engaged a firm managed by an executive of the Company for professional services related to accounting, finance and other administrative functions. There were 0 costs incurred under this arrangement for either of the three months ended June 30, 2022 and 2021. For the six months ended June 30, 2022 and 2021, the costs incurred under this arrangement totaled $0 and $0.1 million, respectively, of which $0.1 million was recorded in stockholders’ equity as a reduction to additional paid-in capital as a result of the Business Combination and $10 thousand was recorded as general and administrative expense in the accompanying condensed consolidated statements of operations and comprehensive loss for the six months ended June 30, 2021. There were 0 amounts owed under this arrangement as of June 30, 2022 and December 31, 2021. The executive of the Company associated with this firm resigned from the Company in February 2021.
14. Subsequent events
On August 5, 2022, the Company formed a wholly-owned subsidiary, Gemstone Merger Sub, Inc. (“Gem Merger Sub”), a Delaware shell company formed solely for the purpose of merging with Disc (as defined below) in connection with the proposed merger transaction with Disc.
In February 2022, the Company announced a corporate restructuring and that it initiated a process to evaluate strategic alternatives. After a comprehensive review of strategic alternatives, including identifying and reviewing potential candidates for a strategic transaction, on August 9, 2022, the Company entered into an Agreement and Plan of Merger and Reorganization (the “Disc Merger Agreement”) with Disc Medicine, Inc., a Delaware corporation (“Disc”), and Gem Merger Sub, pursuant to which, subject to the satisfaction or waiver of the conditions therein, Gem Merger Sub will merge with and into Disc (the “Disc Merger”), with Disc continuing as the surviving company and a wholly-owned subsidiary of the Company. The Disc Merger was unanimously approved by the Company’s board of directors (the “Board”), and the Board resolved to recommend approval of the Disc Merger Agreement to the Company’s shareholders.
The transaction is expected to close in the fourth quarter of 2022. The closing of the Disc Merger is subject to approval by the stockholders of the Company and Disc as well as other customary closing conditions, including the effectiveness of a registration statement filed with the SEC in connection with the transaction and Nasdaq’s approval of the listing of the shares of the Company’s common stock to be issued in connection with the transaction. If the Company is unable to satisfy certain closing conditions or if other mutual closing conditions are not satisfied, Disc will not be obligated to complete the Disc Merger. The Disc Merger Agreement contains certain termination rights of each of the Company and Disc. Under certain circumstances detailed in the Disc Merger Agreement, the Company could be required to pay Disc a termination fee of $3,000,000 or Disc could be required to pay the Company a termination fee of $7,800,000. In addition, in certain circumstances upon the termination of the Disc Merger Agreement, we could be required to pay the costs and expenses of Disc in an amount not to exceed $750,000, or Disc could be required to pay our costs and expenses in an amount not to exceed $750,000. If the merger is completed, the business of Disc will continue as the business of the combined company.
1621
Subject to the terms and conditions of the Disc Merger Agreement, at the effective time of the Disc Merger (the "Effective Time"), each then outstanding share of Disc common stock (including shares of Disc common stock issued upon conversion of Disc preferred stock and shares of Disc common stock issued in the concurrent financing transaction) will be converted into the right to receive a number of shares of the Company’s common stock (subject to the payment of cash in lieu of fractional shares and after giving effect to a reverse stock split of the Company’s common stock) calculated in accordance with the Disc Merger Agreement (the “Exchange Ratio”). Further, at the Effective Time, each person who as of immediately prior to the Effective Time was a stockholder of record of the Company or had the right to receive the Company’s common stock will be entitled to receive a contractual contingent value right (“CVR”) issued by the Company subject to and in accordance with the terms and conditions of a Contingent Value Rights Agreement between the Company, the holder’s representative and the rights agent (the “CVR Agreement”), representing the contractual right to receive payments from the post-closing combined company upon receipt of certain proceeds derived from consideration paid as a result of the disposition of the Company’s pre-Disc Merger assets, net of certain permitted deductions for expenses.ITEM 2. MANAGEMENT’S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS OF OPERATIONS
Concurrently with the execution and delivery of the Disc Merger Agreement, certain parties have entered into agreements with Disc pursuant to which they have agreed, subject to the terms and conditions of such agreements, to purchase prior to the consummation of the Disc Merger shares of Disc common stock for an aggregate purchase price of approximately $53.5 million. Shares of Disc common stock issued pursuant to this financing transaction will be converted into shares of the Company’s common stock in the Disc Merger in accordance with the Exchange Ratio.
The Company’s future operations are highly dependent on the success of the Disc Merger and there can be no assurances that the Disc Merger will be successfully consummated. In the event that the Company does not complete the transaction with Disc, the Company may explore strategic alternatives, including, without limitation, another strategic transaction and/or pursue a dissolution and liquidation of the Company.
17
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of Operations.
The following discussion and analysis of our financial condition and results of operations should be read in conjunction with ourthe unaudited condensed consolidated financial statements and notes thereto appearing elsewhere in this Quarterly Report on Form 10-Q. Some of the information contained in this discussion and analysis or set forthincluded elsewhere in this Quarterly Report on Form 10-Q including information with respectand the audited consolidated financial statements and notes thereto included in our Annual Report on Form 10-K for the year ended December 31, 2022. Please refer to our plans and strategy for our business and related financing, includesnote regarding forward-looking statements that involve risks and uncertainties. As a resultunder the caption “Forward Looking Statements” of many factors, our actual results could differ materially from the results described in or impliedthis Quarterly Report on Form 10-Q, which is incorporated herein by the forward-looking statements contained in the following discussion and analysis.this reference.
Overview
We are a clinical-stage precision medicinebiopharmaceutical company developingfocused on the discovery, development, and commercialization of novel therapeutic compoundstreatments for patients suffering from serious hematologic diseases. We have assembled a portfolio of clinical and preclinical product candidates that aim to treat genetically defined, age-related macular degeneration (“AMD”).modify fundamental biological pathways associated with the formation and function of red blood cells, specifically heme biosynthesis and iron homeostasis. Our current pipeline includes, bitopertin for the treatment of erythropoietic porphyrias, or EPs, including erythropoietic protoporphyria, or EPP, and X-linked protoporphyria, or XLP, and Diamond-Blackfan Anemia, or DBA; DISC-0974 for the treatment of anemia of myelofibrosis, or MF, and anemia of chronic kidney disease, or CKD; and MWTX-003 for the treatment of polycythemia vera, or PV, and other hematologic disorders. In addition, our preclinical programs also include DISC-0998, for the treatment of anemia associated with inflammatory diseases. Our approach to product candidate development leverages well-understood molecular mechanisms that have been validated in humans. We believe that each of our product candidates, if approved, has the potential to improve the lives of patients suffering from hematologic diseases.
Bitopertin is the lead product candidate GEM103,in our heme biosynthesis modulation portfolio. Bitopertin was previously evaluated by F. Hoffmann-La Roche Ltd and Hoffmann-La Roche Inc., or collectively, Roche, in a comprehensive clinical program in over 4,000 individuals in other indications which demonstrated the activity of bitopertin as a glycine transporter 1, or GlyT1, inhibitor and its effect on heme biosynthesis. We are planning to initially develop bitopertin for the treatment of EPs, including EPP and XLP. In July 2022, we initiated BEACON, a Phase 2 open-label, parallel-dose clinical trial of bitopertin in EPP and XLP patients that is being conducted at sites in Australia. Separately, in July 2022, we received clearance of our Investigational New Drug application, or IND, from the U.S. Food and Drug Administration, or FDA, for, and in October 2022 we initiated, AURORA, a recombinant formPhase 2, randomized, double-blind, placebo-controlled clinical trial of bitopertin in EPP patients that is being conducted at sites in the human complement factor H protein (“CFH”)United States. We presented positive initial data from BEACON in June of 2023 at the European Hematology Association 2023 Congress, and we expect BEACON data from all patients to be presented year-end 2023 and AURORA topline data by early 2024. We entered into a collaborative research and development agreement with the National Institutes of Health, or NIH, to conduct an NIH-sponsored clinical trial of bitopertin in DBA. The FDA authorized the clinical trial to proceed and we announced on July 27, 2023 that the first patient was enrolled in this trial. We are planning additional trials of bitopertin in other indications.
DISC-0974 is the lead product candidate in our iron homeostasis portfolio. DISC-0974 is designed to suppress hepcidin production and increase serum iron levels. We submitted an IND to the FDA for DISC-0974 in June 2021, received clearance in July 2021, and participants completed a Phase 1 clinical trial in healthy volunteers in the U.S. in June 2022 with results showing an acceptable tolerability profile and evidence of target engagement, iron mobilization and augmented erythropoiesis. We initiated a Phase 1b/2 clinical trial in June 2022 in patients with anemia of MF, and initiated a separate Phase 1b/2 clinical trial in February 2023 in patients with non-dialysis dependent CKD and anemia. We expect initial data from both of these trials by end of 2023. In addition, we are developing a preclinical anti-hemojuvelin, or HJV, monoclonal antibody, DISC-0998, which also targets hepcidin suppression and was in-licensed from AbbVie Deutschland GmbH & Co. KG, or AbbVie. DISC-0998 is designed to increase serum iron levels and has an extended serum half-life as compared to DISC-0974. We believe this profile may be desirable in certain subsets of patients with anemia associated with inflammatory diseases.
Lastly, we are developing MWTX-003, a monoclonal antibody against Transmembrane Serine Protease 6, or TMPRSS6, that we licensed from Mabwell Therapeutics, Inc., or Mabwell. MWTX-003 is part of our iron homeostasis portfolio and is designed to address complement hyperactivityinduce hepcidin production and overall dysregulation causedreduce serum iron levels. An IND for MWTX-003 has been cleared by loss of function mutations thus restoring retinal healththe FDA, and we plan to initiate a Phase 1 clinical trial in patients with AMD. Native CFH serves multiple functions in maintaining retinal health, including regulating lipid metabolismhealthy adult volunteers in the retina, protectingsecond half of 2023. We expect to develop MWTX-003 for the retina against lipidtreatment of PV and protein by-products of oxidative stress, and regulating the complement system, which is part of the innate immune system. This multifaceted regulation plays an integral role in engagement and maintenance of complement-mediated immune responses that are involved in pathogen defense and cellular debris clearance.other hematologic disorders.
In January 2022, we announced that we discontinued both of our Phase 2a clinical trials of GEM103, the ReGAtta study and the GEM103 as an Add-On to Anti-VEGF Therapy for the Treatment of Wet-AMD study.Reverse Merger with Gemini
In February 2022, we announced a corporate restructuring and that we had initiated a process to evaluate strategic alternatives. After a comprehensive review of strategic alternatives, including identifying and reviewing potential candidates for a strategic transaction, onOn August 9, 2022, weDisc Medicine, Inc., a Delaware corporation, or Private Disc, entered into an Agreement and Plan of Merger and Reorganization, (the “Discor the Merger Agreement”)Agreement, with Disc Medicine,Gemini Therapeutics, Inc., a Delaware corporation, (“Disc”), andor Gemini, Gemstone Merger Sub, Inc., a Delaware corporation and a wholly-ownedwholly owned subsidiary of ours (“Gem Merger Sub”), pursuant to which, subject to the satisfaction or waiver of the conditions therein, Gem Merger Sub will merge with and into Disc (the “Disc Merger”), with Disc continuing as the surviving company and a wholly-owned subsidiary of the Company. The Disc Merger was unanimously approved by our board of directors (the “Board”), and the Board resolved to recommend approval of the Disc Merger Agreement to our shareholders.
The transaction is expected to close in the fourth quarter of 2022. The closing of the Disc Merger is subject to approval by our stockholders and the stockholders of Disc as well as other customary closing conditions, including the effectiveness of a registration statement filed with the SEC in connection with the transaction and Nasdaq’s approval of the listing of the shares of the Company’s common stock to be issued in connection with the transaction. If we are unable to satisfy certain closing conditions or if other mutual closing conditions are not satisfied, Disc will not be obligated to complete the Disc Merger. The Disc Merger Agreement contains certain termination rights of each of us and Disc. Under certain circumstances detailed in the Disc Merger Agreement, we could be required to pay Disc a termination fee of $3,000,000 or Disc could be required to pay us a termination fee of $7,800,000. In addition, in certain circumstances upon the termination of the Disc Merger Agreement, we could be required to pay the costs and expenses of Disc in an amount not to exceed $750,000, or Disc could be required to pay our costs and expenses in an amount not to exceed $750,000. If the merger is completed, the business of Disc will continue as the business of the combined company.
Subject to the terms and conditions of the Disc Merger Agreement, at the effective time of the Disc Merger (the "Effective Time"), each then outstanding share of Disc common stock (including shares of Disc common stock issued upon conversion of Disc preferred stock and shares of Disc common stock issued in the concurrent financing transaction) will be converted into the right to receive a number of shares of the Company’s common stock (subject to the payment of cash in lieu of fractional shares and after giving effect to a reverse stock split of the Company’s common stock) calculated in accordance with the Disc Merger Agreement (the “Exchange Ratio”). Further, at the Effective Time, each person who as of immediately prior to the Effective Time was a stockholder of record of Gemini, or had the right to receive our common stock will be entitled to receive a contractual contingent value right (“CVR”) issued by the Company subject to and in accordance with the terms and conditions of a Contingent Value Rights Agreement between the Company, the holder’s representative and the rights agent (the “CVR Agreement”), representing the contractual right to receive payments from the post-closing combined company upon receipt of certain proceeds derived from consideration paid as a result of the disposition of our pre-Disc Merger assets, net of certain permitted deductions for expenses.Sub.
Concurrently with the execution and delivery of the Disc Merger Agreement, certain parties have entered into agreements with Disc pursuant to which they have agreed, subject to the terms and conditions of such agreements, to purchase prior to the consummation of the Disc Merger shares of Disc common stock for an aggregate purchase price of approximately $53.5 million. Shares of Disc common stock issued pursuant to this financing transaction will be converted into shares of the Company’s common stock in the Disc Merger in accordance with the Exchange Ratio.
1822
The Company's future operations are highly dependent onOn December 29, 2022, Private Disc completed the success ofmerger with Gemini, or the Disc Merger and there can be no assurances that the Disc Merger will be successfully consummated. In the event that we do not complete the transaction with Disc, we may explore strategic alternatives, including, without limitation, another strategic transaction and/or pursue a dissolution and liquidation of the Company.
Since inception in 2015, we have devoted substantially all our efforts and financial resources to organizing and staffing our company, business planning, raising capital, discovering product candidates and securing related intellectual property rights and conducting research and development activities for our product candidates. We do not have any products approved for sale, and we have not generated any revenue from product sales. We may never be able to develop or commercialize a marketable product.
To the extent we continue to pursue clinical development of GEM103 or any other product candidate, our ability to generate revenue from product sales sufficient to achieve profitability will depend heavily on the successful development and eventual commercialization of one or more of our product candidates. We have not yet successfully completed any pivotal clinical trials, nor have we obtained any regulatory approvals, manufactured a commercial-scale drug, or conducted sales and marketing activities.
We are also working to advance GEM307, that could be effective for treatment of systemic diseases, towards IND filing.
COVID-19 pandemic
The ongoing coronavirus (“COVID-19”) pandemic and the increased prevalence of variants of the virus, and government measures taken in response, have had a significant impact, both direct and indirect, on businesses and commerce, as worker shortages have occurred; supply chains have been disrupted; facilities and production have been suspended; and demand for certain goods and services, such as medical services and supplies, has spiked, while demand for other goods and services, such as travel, has fallen. The ongoing COVID-19 pandemic and related impacts have resulted in and will likely continue to result in significant disruptions to the global economy and capital markets around the world. We cannot predict the future progression or full impact of the outbreak and its effects on our business and operations.
We have not incurred impairment losses in the carrying values of our assets as a result of the ongoing COVID-19 pandemic, and we are not aware of any specific related event or circumstance that would require us to revise our estimates reflected in our condensed consolidated financial statements. Although the COVID-19 pandemic did not have a significant impact on our financial results in the second quarter of 2022, the full extent to which the ongoing COVID-19 pandemic may impact our business, results of operations, financial condition and cash flows will depend on future developments that are highly uncertain, and the estimates of the impact on our business may change based on new information that may emerge concerning COVID-19, including the duration of the pandemic, any potential subsequent waves or strains of COVID-19 infection, the effectiveness, distribution and acceptance of COVID-19 vaccines and the actions to contain it or treat its impact and the economic impact on local, regional, national and international markets.
Business Combination
On February 5, 2021, FSDC consummated a previously announced business combination pursuant to the terms of the Agreement and Plan of Merger, dated as of October 15, 2020 (as amended, supplemented or otherwise modified from time to time, the “Merger Agreement”), by and among Old Gemini, Stockholders’ Representative, and Merger Sub (the “Business Combination”).
FSDC was incorporated in Delaware on June 25, 2020 and was formed for the purpose of effecting a merger, capital stock exchange, asset acquisition, stock purchase, reorganization or similar business combination with one or more businesses.
On the day prior to the Closing Date, Oldmerger. Gemini changed its name to “Gemini Therapeutics Sub,Disc Medicine Inc.” Pursuant to the Merger Agreement, on the Closing Date, (i) FSDC, and Private Disc changed its name to “Gemini Therapeutics,Disc Medicine Opco, Inc.”, and (ii) Old Gemini merged with and into Merger Sub (the “Merger”), with Old Gemini as the surviving company in the Merger and, after giving effect to such Merger, Old Gemini becoming a wholly-owned subsidiary of Gemini. Upon the closing of the Business Combination, and pursuant to the terms of the Merger Agreement, the existing shareholders of Old Gemini exchanged their interests for shares ofDisc Medicine, Inc. On December 30, 2022, our common stock of Gemini.
In connection withbegan trading on The Nasdaq Capital Market under the Business Combination, certain investors purchased an aggregate of $95.1 million of our Common Stock in a private placement of public equity (the “PIPE Financing”). Together with FSDC’s cash resources and funding of the PIPE Financing, we received net proceeds of approximately $195.9 million.ticker symbol “IRON.”
We accounted for the Business Combination as a reverse recapitalization, which is the equivalent of Old Gemini issuing stock for the net assets of FSDC, accompanied by a recapitalization, with FSDC treated as the acquired company for accounting purposes. The net assets of FSDC were stated at historical cost with no goodwill or other intangible assets recorded. Reported results from operations included herein prior to the Business Combination are those of Old Gemini. The shares and corresponding capital amounts and loss per share related to Old Gemini’s outstanding convertible preferred stock and common stock prior to the Business Combination have been
19
retroactively restated to reflect the conversion ratio established in the Merger Agreement (1.00 Old Gemini share for 0.2180 shares of our company (the “Conversion Ratio”).
For additional information on the Business Combination, please read Note 2, Business Combination, to our unaudited condensed consolidated financial statements included elsewhere in this Quarterly Report on Form 10-Q.
Financial Operations Overview
Revenue
We have not generated any revenue since our inception and do not expect to generate any revenue from the sale of products in the near future, if at all. If our development efforts were to continue and wereare successful and we were to commercialize anyresult in commercialization of ourone or more product candidates or if we enter into collaboration or license agreements with third parties, we may generate revenue in the future from product sales, as well as upfront, milestone and royalty payments from such collaboration or license agreements or a combination thereof.
Operating expensesExpenses
Research and development expensesDevelopment Expenses
Research and development expenses consist primarily of costs incurred forin connection with the research activities, including drug discovery efforts and the clinical development of our product candidates. We expense research and development costs as incurred, whichThese expenses include:
We recognize externalexpense research and development costs as incurred. AnyCosts incurred for external development activities are recognized based on an evaluation of the progress to completion of specific tasks using information provided to us by our vendors. Payments for these activities are based on the terms of the individual agreements, which may differ from the pattern of costs incurred, and may be reflected in our condensed consolidated financial statements as prepaid or accrued expenses. Nonrefundable advance payments that we make for goods or services to be received in the future for use in research and development activities are recorded as prepaid expenses. Such amounts areexpenses and expensed as the related goods are delivered or the related services are performed or untilwhen it is no longer expected that the goods will be delivered or the services rendered. We estimate and accrue for the value of goods and services received from CROs, CMOs and other third parties each reporting period based on an evaluation of the progress to completion of specific tasks using information provided to us by our service providers. This process involves reviewing open contracts and purchase orders, communicating with our personnel to identify services that have been performed on our behalf and estimating the level of service performed and the associated cost incurred for the service when we have not yet been invoiced or otherwise notified of actual costs.
We do not tracktypically use our researchemployee and infrastructure resources across product candidates and development expenses on a program-by-program basis. Our direct external research andprograms. We track outsourced development expenses consist primarily of external costs such as fees paid to outside consultants, CROs, CMOs and research laboratories in connection with our preclinicalby product candidate or development process development, manufacturing and clinical development activities. Weprogram, but we do not allocate employeepersonnel costs costs associated with our discovery efforts, laboratory supplies, and facilities, including depreciation or other indirectinternal costs to specific programs because these costs are deployed across multiple programs and, as such, are not separately classified. We use internal resources primarily to conduct our research and discovery as well as for managing our preclinicalproduct candidates or development process development, manufacturing and clinical development activities. These employees work across multiple programs and, therefore, we do not track their costs by program.programs.
20
Research and development activities have historically been central to our business model. We anticipateexpect that our research and development expenses will decrease in 2022 compared to 2021 due toincrease substantially as we advance our planned reducedprograms into and through clinical efforts in 2022 and restructuring plans implemented in connection with our exploration of strategic alternatives. If we were to continue to pursue development efforts and we believe a regulatory approval of a product candidate appears likely, we would anticipate an increase in payroll and other expenses as a result of our preparation of regulatory filings and precommercial activities.
development. At this time, we cannot reasonablyaccurately estimate or know the nature, timing and costs of the efforts that wouldwill be necessary to complete the preclinical and clinical development of any of our product candidates or when, if ever, material net cash inflowswe may commence fromdevelop. A change in the outcome of any number of ourvariables with respect to product candidates.candidates we may develop could significantly change the costs and timing associated with the development of that product candidate. We may never succeed in obtaining regulatory approval for any product candidates we may develop. The successful development and commercialization of any of our product candidatescandidate is highly uncertain. This uncertainty is due to the numerous risks and uncertainties associated with product development, and commercialization, including the uncertainty of the following:
23
A change in any of these variables with respect to any of our programs would significantly change the costs, timing and viability associated with that program.
General and administrative expensesAdministrative Expenses
General and administrative expenses consist primarily of employee-related expenses, including salaries and related benefits, travel and stock-based compensationcosts for personnel in executive, finance, corporate and business development, finance, human resources, legal, information technology and administrative functions. General and administrative expenses also include insurance costslegal fees relating to patent and corporate matters, including noncapitalizable transaction costs; professional fees for legal, patent, consulting, investor and public relations, accounting, and audit services. We expense generalauditing, tax and administrative consulting services; insurance costs, as incurred.facility related expenses including maintenance and allocated expenses for rent and other operating costs.
We anticipate that our general and administrative expenses will decrease in 2022 as compared to 2021 due to restructuring plans we implemented. If we were to continue product development efforts and at any pointincrease substantially in the future as we believe a regulatory approval of a product candidate appears likely, we would anticipate an increase in payrollour headcount to support our continued research and other expenses as a resultdevelopment and potential commercialization of our preparation for commercial operations, especially as it relates to the salesproduct candidates. We also expect that we will incur increased expenses associated with being a public company, including costs of accounting, audit, legal, regulatory and marketing of that product candidate. Additionally, depending on the outcome of our ongoing strategic alternative review process, there may be an increase in generaltax compliance services, director and administrativeofficer insurance costs and investor and public relations expenses.
Other income (expense)Income
21
Interest expenseIncome
Interest expenseincome primarily consists of interest accruedearned on the Term Loancash equivalents, consisting of money market funds, U.S. treasury notes and certificates of deposit.
Change in Fair Value of Contingent Value Right Liability
In December 2022, we entered into the CVR Agreement, described in February 2019more detail in Notes 1 and for4 to our unaudited condensed consolidated financial statements, which provided a CVR to Gemini’s pre-merger common stockholders to receive common stock from the six months ended June 30, 2021, interest expense forCompany upon its receipt of certain proceeds, calculated in accordance with the Notes, including the accretionCVR Agreement, resulting from a disposition of the beneficial conversion feature discount recognized on the issuance date of the Notes.
Interest income
Interest income consists of income earned on our cash, cash equivalents and restricted cash.
Loss on conversion of convertible notes
Immediately prior toGemini’s pre-merger assets within one year after the closing of the Business Combination, the outstanding principal and interest under the Notes converted into sharesmerger. The CVR liability was measured at fair value as of Series B preferred stock, and we recorded other expense equal to the difference between the reacquisition price of the Noteseach reporting date and the net carrying amount ofchange in the Notesfair value for the period was recorded in the condensed consolidated statements of operations and comprehensive loss.loss in the change in fair value of contingent value right liability.
Provision for income taxesChange in Fair Value of Derivative Liability
We have not recorded any significant amounts relatedIn May 2021, we entered into the Roche Agreement, described in more detail in Notes 4 and 8 to income tax expense, we have not recognized any reserves relatedour unaudited condensed consolidated financial statements, which included an obligation to uncertain tax positions, nor have we recorded any income tax benefitsissue a variable number of shares of our common stock to Roche for no additional consideration upon the completion of a Roche Qualified Transaction as defined by the Roche Agreement. The liability was measured at fair value as of each reporting date and the change in the fair value for the majority of our net losses we have incurred to date or for our research and development tax credits.
We account for income taxes using the asset and liability method, which requires the recognition of deferred tax assets and liabilities for the expected future tax consequences of events that have been includedperiod was recorded in the condensed consolidated financial statements or our tax returns. Deferred tax assetsof operations and liabilities are determined based on the difference between the financial statement carrying amounts and tax bases of existing assets and liabilities and forcomprehensive loss and credit carryforwards, which are measured using the enacted tax rates and laws in effect in the yearschange in which the differences are expected to reverse.fair value of derivative liability. The realization of our deferred tax assets is dependentliability was settled upon the generation of future taxable income, the amount and timing of which are uncertain. Valuation allowances are provided, if, based upon the weight of available evidence, it is more likely than not that some or allcompletion of the deferred tax assets will not be realized. We continue to maintain a full valuation allowance against all of our net deferred tax assets basedmerger on our evaluation of all available evidence.December 29, 2022.
We file income tax returns in the U.S. federal tax jurisdiction and state jurisdictions and may become subject to income tax audit and adjustments by related tax authorities. Our tax return period for U.S. federal income taxes for the tax years since 2018 remain open to examination under the statute of limitations by the Internal Revenue Service and state jurisdictions. We record reserves for potential tax payments to various tax authorities related to uncertain tax positions, if any. The nature of uncertain tax positions is subject to significant judgment by management and subject to change, which may be substantial. These reserves are based on a determination of whether and how much a tax benefit taken by us in our tax filings or positions is more likely than not to be realized following the resolution of any potential contingencies related to the tax benefit. We develop our assessment of uncertain tax positions, and the associated cumulative probabilities, using internal expertise and assistance from third-party experts. As additional information becomes available, estimates are revised and refined. Differences between estimates and final settlement may occur resulting in additional tax expense. Potential interest and penalties associated with such uncertain tax positions is recorded as a component of our provision for income taxes. To date, no amounts are being presented as an uncertain tax position.
In addition, under Section 382 of the Internal Revenue Code of 1986, as amended, and corresponding provisions of state law, if a corporation undergoes an “ownership change,” which is generally defined as a greater than 50% change, by value, in its equity ownership over a three-year period, the corporation’s ability to use its pre-change net operating loss carryforwards and other pre-change tax attributes to offset its post-change income or taxes may be limited. We may experience ownership changes as a result of subsequent shifts in our stock ownership.
2224
Results of operationsOperations
Comparison of the three months endedThree Months Ended June 30, 20222023 and 20212022
The following table summarizes our results of operations for the three months ended June 30, 2023 and 2022 and 2021 (in thousands)(in thousands):
|
| Three Months Ended |
|
|
|
| ||||||
|
| 2022 |
|
| 2021 |
|
| Change |
| |||
Operating expenses: |
|
|
|
|
|
|
|
|
| |||
Research and development |
| $ | 2,580 |
|
| $ | 10,842 |
|
| $ | (8,262 | ) |
General and administrative |
|
| 4,654 |
|
|
| 5,478 |
|
|
| (824 | ) |
Total operating expenses |
|
| 7,234 |
|
|
| 16,320 |
|
|
| (9,086 | ) |
Loss from operations |
|
| (7,234 | ) |
|
| (16,320 | ) |
|
| 9,086 |
|
Other income (expense): |
|
|
|
|
|
|
|
|
| |||
Interest expense |
|
| (48 | ) |
|
| (121 | ) |
|
| 73 |
|
Interest income |
|
| 149 |
|
|
| 5 |
|
|
| 144 |
|
Other expense |
|
| - |
|
|
| (11 | ) |
|
| 11 |
|
Other income |
|
| 43 |
|
|
| - |
|
|
| 43 |
|
Net loss and comprehensive loss |
| $ | (7,090 | ) |
| $ | (16,447 | ) |
| $ | 9,357 |
|
| Three Months Ended |
| ||||||||||
| 2023 |
|
| 2022 |
| | Change |
| ||||
Operating expenses: | |
|
|
|
|
| |
|
| |||
Research and development | | $ | 12,100 |
|
| $ | 7,714 |
| | $ | 4,386 |
|
General and administrative | |
| 5,228 |
|
|
| 4,301 |
| |
| 927 |
|
Total operating expenses | |
| 17,328 |
|
|
| 12,015 |
| |
| 5,313 |
|
Loss from operations | |
| (17,328 | ) |
|
| (12,015 | ) | |
| (5,313 | ) |
Other income (expense), net: | |
|
|
|
|
| |
|
| |||
Interest income | |
| 2,910 |
|
|
| 45 |
| |
| 2,865 |
|
Change in fair value of contingent value right liability |
|
| (1,500 | ) |
|
| — |
|
|
| (1,500 | ) |
Change in fair value of derivative liability | |
| — |
|
|
| 2,390 |
| |
| (2,390 | ) |
Other expense |
|
| (5 | ) |
|
| — |
|
|
| (5 | ) |
Total other income (expense), net | |
| 1,405 |
|
|
| 2,435 |
| |
| (1,030 | ) |
Loss before income taxes |
|
| (15,923 | ) |
|
| (9,580 | ) |
|
| (6,343 | ) |
Income tax expense |
|
| (24 | ) |
|
| — |
|
|
| (24 | ) |
Net loss | | $ | (15,947 | ) |
| $ | (9,580 | ) | | $ | (6,367 | ) |
Research and Development Expenses
The following table summarizes our research and development expenses for the three months ended June 30, 2023 and 2022 (in thousands):
| Three Months Ended |
| ||||||||||
| 2023 |
|
| 2022 |
| | Change |
| ||||
Bitopertin | | $ | 4,179 |
|
| $ | 2,092 |
| | $ | 2,087 |
|
DISC-0974 | |
| 2,112 |
|
|
| 2,191 |
| |
| (79 | ) |
MWTX-003 | |
| 526 |
|
|
| — |
| |
| 526 |
|
Personnel-related (including equity-based compensation) | |
| 3,468 |
|
|
| 2,105 |
| |
| 1,363 |
|
Other research programs and expenses | |
| 1,815 |
|
|
| 1,326 |
| |
| 489 |
|
Total research and development expenses | | $ | 12,100 |
|
| $ | 7,714 |
| | $ | 4,386 |
|
Research and development expenses were $2.6$12.1 million for the three months ended June 30, 2022,2023, compared to $10.8$7.7 million for the three months ended June 30, 2021.2022. The decreaseincrease of $8.2$4.4 million in research and development expenses was primarily due to a decrease$2.1 million increase in external researchbitopertin development expense related to increased clinical study and drug manufacturing activity, as well as a $1.4 million increase in personnel-related costs related to the elimination of our lab space and a decrease in development costs related to lower clinical trial activities of GEM103. In addition,higher research and development personnel costs were lower period over period, including stock-based compensation, due to both a decrease in headcount inheadcount.
General and Administrative Expenses
The following table summarizes our researchgeneral and development function in connection with the restructuring plans we implemented and the employee retention credit receivable recorded duringadministrative expenses for the three months ended June 30, 2022.2023 and 2022 (in thousands):
General and administrative expenses
| Three Months Ended |
| ||||||||||
| 2023 |
|
| 2022 |
| | Change |
| ||||
Personnel-related (including equity-based compensation) | | $ | 2,495 |
|
| $ | 1,093 |
| | $ | 1,402 |
|
Legal, consulting and professional fees | |
| 1,791 |
|
|
| 2,988 |
| |
| (1,197 | ) |
Other expenses | |
| 942 |
|
|
| 220 |
| |
| 722 |
|
Total general and administrative expenses | | $ | 5,228 |
|
| $ | 4,301 |
| | $ | 927 |
|
General and administrative expenses were $4.7$5.2 million for the three months ended June 30, 2022,2023, compared to $5.5$4.3 million for the three months ended June 30, 2021.2022. The decreaseincrease of $0.8$0.9 million in general and administrative expenses was primarily due to loweran increase of $1.4 million in personnel-related costs due to both a decrease in headcount in ourhigher general and administrative functionheadcount, offset by a decrease of $1.2 million in connection withlegal, consulting and professional fees primarily due to the restructuring plans we implemented andrecognition of $2.2 million of deferred transaction costs in June 2022 related to a planned equity financing that was superseded by the employee retention credit receivable recorded duringmerger.
25
Other Income (Expense), Net
Other income (expense), net was $1.4 million for the three months ended June 30, 2022.
Interest expense
Interest expense was $0.12023, compared to $2.4 million for each of the three months ended June 30, 20222022. The change of $1.0 million was primarily due to the changes in fair value of the derivative and 2021 relatedcontingent value right liabilities, partially offset by an increase in interest income based on a larger cash and cash equivalents balance and higher interest rates.
Income Tax Expense
Income tax expense was less than $0.1 million for the three months ended June 30, 2023, compared to no expense for the three months ended June 30, 2022. The increase in expense relates to state income tax resulting from the increase in interest on the Term Loan.income.
Comparison of the six months endedSix Months Ended June 30, 20222023 and 20212022
The following table summarizes our results of operations for the six months ended June 30, 2023 and 2022 and 2021 (in thousands)(in thousands):
|
| Six Months Ended |
|
|
|
| | Six Months Ended |
| |||||||||||||||
|
| 2022 |
|
| 2021 |
|
| Change |
| | 2023 |
|
| 2022 |
| | Change |
| ||||||
Operating expenses: |
|
|
|
|
|
|
|
|
| |
|
|
|
| |
|
| |||||||
Research and development |
| $ | 12,384 |
|
| $ | 22,628 |
|
| $ | (10,244 | ) | | $ | 32,280 |
|
| $ | 15,535 |
| | $ | 16,745 |
|
General and administrative |
|
| 10,027 |
|
|
| 10,182 |
|
|
| (155 | ) | |
| 10,173 |
|
|
| 6,440 |
| |
| 3,733 |
|
Total operating expenses |
|
| 22,411 |
|
|
| 32,810 |
|
|
| (10,399 | ) | |
| 42,453 |
|
|
| 21,975 |
| |
| 20,478 |
|
Loss from operations |
|
| (22,411 | ) |
|
| (32,810 | ) |
|
| 10,399 |
| |
| (42,453 | ) |
|
| (21,975 | ) | |
| (20,478 | ) |
Other income (expense): |
|
|
|
|
|
|
|
|
| |||||||||||||||
Interest expense |
|
| (114 | ) |
|
| (1,969 | ) |
|
| 1,855 |
| ||||||||||||
Other income (expense), net: | |
|
|
|
| |
|
| ||||||||||||||||
Interest income |
|
| 158 |
|
|
| 6 |
|
|
| 152 |
| |
| 5,277 |
|
|
| 52 |
| |
| 5,225 |
|
Loss on conversion of convertible notes |
|
| - |
|
|
| (711 | ) |
|
| 711 |
| ||||||||||||
Change in fair value of contingent value right liability |
|
| (1,500 | ) |
|
| — |
|
|
| (1,500 | ) | ||||||||||||
Change in fair value of derivative liability | |
| — |
|
|
| 2,490 |
| |
| (2,490 | ) | ||||||||||||
Other expense |
|
| - |
|
|
| (11 | ) |
|
| 11 |
|
|
| (5 | ) |
|
| — |
|
|
| (5 | ) |
Other income |
|
| 46 |
|
|
| - |
|
|
| 46 |
| ||||||||||||
Net loss and comprehensive loss |
| $ | (22,321 | ) |
| $ | (35,495 | ) |
| $ | 13,174 |
| ||||||||||||
Total other income (expense), net | |
| 3,772 |
|
|
| 2,542 |
| |
| 1,230 |
| ||||||||||||
Loss before income taxes |
|
| (38,681 | ) |
|
| (19,433 | ) |
|
| (19,248 | ) | ||||||||||||
Income tax expense |
|
| (47 | ) |
|
| — |
|
|
| (47 | ) | ||||||||||||
Net loss | | $ | (38,728 | ) |
| $ | (19,433 | ) | | $ | (19,295 | ) | ||||||||||||
23
Research and Development Expenses
The following table summarizes our research and development expenses for the six months ended June 30, 2023 and 2022 (in thousands):
| Six Months Ended |
| ||||||||||
| 2023 |
|
| 2022 |
| | Change |
| ||||
Bitopertin | | $ | 6,762 |
|
| $ | 4,447 |
| | $ | 2,315 |
|
DISC-0974 | |
| 4,627 |
|
|
| 4,290 |
| |
| 337 |
|
MWTX-003 | |
| 10,567 |
|
|
| — |
| |
| 10,567 |
|
Personnel-related (including equity-based compensation) | |
| 6,588 |
|
|
| 4,292 |
| |
| 2,296 |
|
Other research programs and expenses | |
| 3,736 |
|
|
| 2,506 |
| |
| 1,230 |
|
Total research and development expenses | | $ | 32,280 |
|
| $ | 15,535 |
| | $ | 16,745 |
|
Research and development expenses were $12.4$32.3 million for the six months ended June 30, 2022,2023, compared to $22.6$15.5 million for the six months ended June 30, 2021.2022. The decreaseincrease of $10.2$16.7 million in research and development expenses was primarily due to a decrease$10.0 million upfront payment under the Mabwell license agreement, as well as a $2.3 million increase in external researchpersonnel-related costs related to the elimination of our lab space and a decrease in development costs related to lower clinical trial activities of GEM103. In addition,higher research and development personnel costs were lower period over period, including stock-based compensation, dueheadcount and a $2.3 million increase in bitopertin related to both a decrease in headcount in our researchincreased clinical study and development function in connection with the restructuring plans we implemented and the employee retention credit receivable recorded during the six months ended June 30, 2022.drug manufacturing activity.
26
General and administrative expensesAdministrative Expenses
GeneralThe following table summarizes our general and administrative expenses were $10.0 million for the six months ended June 30, 2023 and 2022 compared to(in thousands):
| Six Months Ended |
| ||||||||||
| 2023 |
|
| 2022 |
| | Change |
| ||||
Personnel-related (including equity-based compensation) | | $ | 4,577 |
|
| $ | 2,288 |
| | $ | 2,289 |
|
Legal, consulting and professional fees | |
| 3,719 |
|
|
| 3,777 |
| |
| (58 | ) |
Other expenses | |
| 1,877 |
|
|
| 375 |
| |
| 1,502 |
|
Total general and administrative expenses | | $ | 10,173 |
|
| $ | 6,440 |
| | $ | 3,733 |
|
General and administrative expenses were $10.2 million for the six months ended June 30, 2021. The decrease of $0.22023, compared to $6.4 million was primarily due to lower personnel-related costs due to both a decrease in headcount in our general and administrative function in connection with the restructuring plans we implemented and the employee retention credit receivable recorded duringfor the six months ended June 30, 2022. The increase of $3.7 million in general and administrative expenses was primarily due to a $2.3 million increase in personnel-related costs related to higher general and administrative headcount, as well as an increase of $1.5 million in other expenses related to increased costs as a public company.
Interest expenseOther Income (Expense), Net
InterestOther income (expense), net was $3.8 million for the six months ended June 30, 2023, compared to $2.5 million for the six months ended June 30, 2022. The change of $1.2 million in other income (expense), net was primarily due to an increase in interest income based on a larger cash and cash equivalents balance and higher interest rates, partially offset by the changes in fair value of the derivative and contingent value right liabilities.
Income Tax Expense
Income tax expense was less than $0.1 million for the six months ended June 30, 2022,2023, compared to $2.0 millionno expense for the six months ended June 30, 2021.2022. The decrease of $1.9 million is primarily dueincrease in expense relates to accretion duringstate income tax resulting from the six months ended June 30, 2021 of the beneficial conversion feature discount recognized at the issuance date of the Notesincrease in 2020.
Loss on conversion of convertible notes
The loss on conversion of convertible notes was $0 for the six months ended June 30, 2022, compared to $0.7 million for the six months ended June 30, 2021. The decrease reflects the difference between the reacquisition price of the Notes and the net carrying amount of the Notes at the time that the Notes converted into shares of Series B preferred stock immediately prior to the closing of the Business Combination.interest income.
Liquidity and capital resourcesCapital Resources
Sources of liquidity and capitalLiquidity
Since our inception, we have not generated any revenue from any product sales or any other sources and have incurred significant operating losses. We expect to continue to incur significant expenses and operating losses and negative cash flows from our operations. We have not yet commercialized anyin the foreseeable future as we advance the clinical development of our product candidatescandidates. We expect that our research and do not expectdevelopment and general and administrative costs will continue to generate revenue from sales of anyincrease significantly, including in connection with conducting clinical trials and manufacturing for our product candidates to support commercialization and providing general and administrative support for several years, if at all. Our net loss was $22.3 millionour operations, including the costs associated with operating as a public company. As a result, we will need additional capital to fund our operations, which we may obtain from additional equity or debt financings, collaborations, licensing arrangements or other sources. See “Risk Factors” for the six months ended June 30, 2022. As of June 30, 2022, we had an accumulated deficit of $207.0 million.additional risks associated with our substantial capital requirements.
Prior to the Business Combination,To date, we have funded our operations primarily with proceeds from the sale of our convertible preferred stock borrowings under convertible promissory notes and borrowings under loan agreements. In January 2020, we received grosscommon stock, the proceeds of $20.1 million from the sale of our preferred stock. In August 2020,merger with Gemini, proceeds from at-the-market offerings, proceeds from a registered direct offering and proceeds from a follow-on public offering. Through June 30, 2023, we received gross proceeds of $14.0 million from borrowings under convertible promissory notes. In February 2021, in connection with the Business Combination, wehave received net proceeds of $195.9 million.
$144.5 million from sales of our Series Seed, Series A and Series B convertible preferred stock, $89.5 million from the merger with Gemini, $53.5 million from sales of common stock in a pre-closing private financing, $62.4 million from sales of common stock and pre-funded warrants in a registered direct offering, $147.9 million from sales of common stock and pre-funded warrants in a public follow-on offering and $19.5 million from at-the-market offerings. As of June 30, 2022,2023, we had cash and cash equivalents of $108.7$377.6 million. Continued cash generation is highly dependent on our ability to finance our operations through a combination of equity offerings, debt financings, collaboration arrangements and strategic transactions. However, our resource requirements could materially change depending on the outcome of our ongoing strategic alternative review process, including to the extent we identify and enter into any potential strategic transaction.
Until requiredWe have incurred significant operating losses since inception and, as of June 30, 2023, had an accumulated deficit of $150.9 million. In addition, we expect to continue to incur significant and increasing expenses and operating losses for use inthe foreseeable future. We believe that our business, we typically investcurrent cash resources will enable us to fund our current planned operating expense and capital expenditure requirements well into 2026. We may also pursue additional cash in investments that are highly liquid, readily convertible to cash with original maturities of 90 daysresources through public or less at the date of purchase. We attempt to minimize the risks related to our cash and cash equivalents by maintaining balances in accounts only with accredited financial institutions and, consequently, we do not believe we are subject to unusual credit risk beyond the normal credit risk associated with ordinary commercial banking relationships.private equity, collaborations or debt financings.
On August 9, 2022, we entered into the Disc Merger Agreement pursuant to which, subject to the satisfaction or waiver of the conditions therein, Gem Merger Sub will merge with and into Disc, with Disc continuing as the surviving company and a wholly-owned subsidiary of the Company. The Disc Merger was unanimously approved by our board of directors (the “Board”), and the Board resolved to recommend approval of the Disc Merger Agreement to our shareholders. The closing of the Disc Merger is subject to approval by our stockholders and the stockholders of Disc and other customary closing conditions. The Company's future operations are highly dependent on the success of the proposed merger transaction with Disc.
2427
Cash flowsFlows
The following table summarizesprovides information regarding our cash flows for the six months ended June 30, 2022 and 2021 (in thousands)each period presented (in thousands):
|
| Six Months Ended |
| |||||
|
| 2022 |
|
| 2021 |
| ||
Net cash used in operating activities |
| $ | (25,836 | ) |
| $ | (30,935 | ) |
Net cash provided by (used in) financing activities |
|
| (2,455 | ) |
|
| 193,909 |
|
Net increase (decrease) in cash, cash equivalents and restricted cash |
| $ | (28,291 | ) |
| $ | 162,974 |
|
| Six months ended |
| ||||||
| 2023 |
| | 2022 |
| |||
Net cash provided by (used in): | |
|
| |
|
| ||
Operating activities | | $ | (48,190 | ) | | $ | (22,895 | ) |
Investing activities | |
| (41 | ) | |
| (120 | ) |
Financing activities | |
| 231,161 |
| |
| 141 |
|
Net increase (decrease) in cash, cash equivalents and restricted cash | | $ | 182,930 |
| | $ | (22,874 | ) |
Operating activitiesActivities
We do not generate any cash inflows from our operating activities. Our cash flows from operating activities are significantlygreatly influenced by our use of cash for operating expenses and working capital requirements to support theour business. We have historically experienced negative cash flows from operating activities as we invested in developing our platform,portfolio, drug discovery efforts and related infrastructure. The cash used in operating activities resulted primarily from our net losses adjusted for non-cash charges and changes in components of operating assets and liabilities, which are primarily the result of increased expenses and timing of vendor payments.
During the six months ended June 30, 2022, we2023, net cash used cash in operating activities of $25.8$48.2 million reflecting awas primarily due to our net loss of $22.3$38.7 million and a net change of $7.9 millionan increase in our operating assets and liabilities of $13.5 million primarily related to the payment of merger-related costs which were included in accounts payable at December 31, 2022, partially offset by non-cash chargesexpenses of $4.4$4.0 million. The non-cash charges consist primarily of $4.3 million of stock-based compensation expense. The net change in our operating assets and liabilities was primarily due to an increase in prepaid expenses and other current currents and a decrease in accounts payable and accrued expenses and other current liabilities.
During the six months ended June 30, 2021, we used cash in operating activities of $30.9 million, reflecting a net loss of $35.5 million, partially offset by non-cash charges of $7.1 million and a net change of $2.5 million in our operating assets and liabilities. The non-cash charges consist primarily of $4.5 million of stock-based compensation expense, $1.6 million accretion of the discount on the Notes, $0.7 million of expense related to the conversion of the Notes and $0.2 million of non-cash interest expense. The net change in our operating assets and liabilities was primarily due to an increase in prepaid expenses and other current currents, other assets and accounts payable, partially offset by a decrease in deferred offering costs and accrued expenses and other current liabilities.
Financing activities
During the six months ended June 30, 2022, net cash used in financingoperating activities of $22.9 million was $2.5primarily due to our net loss of $19.4 million, consisting primarilyand changes in operating assets and liabilities of principal payments made on our term loan.$1.8 million and non-cash expenses of $1.7 million.
Investing Activities
During the six months ended June 30, 2021,2023 and 2022, net cash used in investing activities was related to purchases of property and equipment.
Financing Activities
During the six months ended June 30, 2023, net cash provided by financing activities was $193.9of $231.2 million consistingconsisted primarily of $195.9 million ofaggregate net proceeds receivedof $147.9 million from sales of common stock and pre-funded warrants in a public follow-on offering, aggregate net proceeds of $62.5 million from sales of common stock and pre-funded warrants in a registered direct offering and aggregate net proceeds of $19.5 million from the Business Combination, partially offset by principal payments made on our term loan.at-the-market offerings.
Funding requirementsDuring the six months ended June 30, 2022, net cash provided by financing activities of $0.1 million was related to proceeds from stock option exercises.
Our primary use of cash is to fund operating expenses, primarily related to our research and development activities. However, our resource requirements could materially change depending on the outcome of our ongoing strategic alternative review process, including to the extent we identify and enter into any potential strategic transaction. Cash used to fund operating expenses is impacted by the timing of when we pay these expenses, as reflected in the change in our outstanding accounts payable, accrued expenses and prepaid expenses.Future Funding Requirements
We currently expect our expenses to decreaseincrease substantially in 2022 compared to 2021 due toconnection with our planned reduced clinical efforts in 2022 and the implementations of the restructurings announced in October 2021 and February 2022. To the extentongoing activities, particularly as we continue to pursue the development ofadvance our product candidates into and through clinical development and operate as a public company. Our funding requirements and the timing and amount of our operating expenditures will depend largely on our ability to:on:
28
25
Identifying potential product candidates and conducting preclinical studies and clinical trials is a time consuming, expensive and uncertain process that takes years to complete, and we may never generate the necessary data or results required to obtain marketing approval and achieve product sales. In addition, our product candidates, if approved, may not achieve commercial success. Our commercial revenues, if any, wouldwill be derived from sales of products that we do not expect to be commercially available for many years, if ever. Accordingly, we will need to obtain substantial additional funds to achieve our business objectives.
As of June 30, 2022, we had cash and cash equivalents of $108.7 million. We believe that our cash and cash equivalents will enable us to fund our operating expenses and capital expenditure requirements through at least the next twelve months from the filing of this Quarterly Report on Form 10-Q. We have based this estimate on assumptions that may prove to be wrong, and we could utilize our available capital resources sooner than we expect. However, our resource requirements could materially change depending on the outcome of our ongoing strategic alternative review process, including to the extent we identify and enter into any potential strategic transaction. Our future resource requirements will depend on the outcome of the proposed merger transaction with Disc.
Until such time, if ever, as we can generate substantial revenue from product revenue, if ever, and subject to our pursuit of a potential strategic transaction and the consummation of such potential transaction (including the proposed merger transaction with Disc),sales, we expect to finance our operationscash needs through a combination of equity offerings, debt financings, collaborations, strategic alliances, and marketing, distribution or licensing arrangements.arrangements with third parties. However, we may be unable to raise additional funds or enter into such other arrangements when needed on favorable terms or at all. To the extent that we raise additional capital through the sale of equity or convertible debt securities, the ownership interests willinterest of our existing stockholders may be materially diluted, and the terms of thesesuch securities maycould include liquidation or other preferences that adversely affect the rights of our common stockholders. Debt financing and preferred equity financing, if available, may involve agreements that include restrictive covenants limiting or restrictingthat limit our ability to take specificspecified actions, such as incurring additional debt, making acquisitions or capital expenditures or declaring dividends. If we raise additional funds through collaborations, strategic alliances or marketing, distribution or licensing arrangements with third parties, we may have to relinquish valuable rights to our technologies, future revenue streams, research programs or drugproduct candidates or grant licenses on terms that may not be favorable to us. If we are unable to raise additional funds through equity or debt financings or other arrangements when needed, we may be required to delay, limit, reduce or terminateeliminate our research, product development or future commercialization efforts, or grant rights to develop and market drugproduct candidates that we would otherwise prefer to develop and market ourselves.
Working capital
Because of the numerous risksContractual Obligations and uncertainties associated with research, development and commercialization of pharmaceutical product candidates, we are unable to estimate the exact amount of our working capital requirements. Subject to our pursuit of a potential strategic transaction and the consummation of such potential transaction, our future funding requirements will depend on and could increase significantly as a result of many factors, including:Other Commitments
26
Contractual obligations and commitments
Term loan
In February 2019, we entered into a term loan facility of up to $10.0 million (the “Term Loan”) with SVB. The proceeds were used for general corporate and working capital purposes. Concurrent with the Term Loan, we issued SVB warrants to purchase 15,257 shares of Old Gemini’s Series A preferred stock at an exercise price of $5.46. At the closing of the Business Combination, these warrants were automatically exercised for 15,257 shares of our common stock. As of June 30, 2022 and December 31, 2021, we had $2.9 million and $5.4 million, respectively, in principal outstanding under the Term Loan.
The Term Loan is governed by a loan and security agreement, entered into in February 2019, between Gemini and SVB (the “SVB Loan Agreement”). The SVB Loan Agreement provided for two separate tranches under which we could borrow. In April 2019, we borrowed $7.5 million under the first tranche, and in December 2019, we borrowed $2.5 million under the second tranche.
The Term Loan matures in January 2023 and accrues interest at a floating rate per annum equal to the greater of 3.75% or the prime rate minus 1.5% (3.25%following table summarizes our contractual obligations as of June 30, 2022).2023 and the effects that such obligations are expected to have on our liquidity and cash flows in future periods (in thousands):
| Payments Due by Period |
| ||||||||||||||||||
| Total |
| | Less Than |
| | 1 to 3 |
| | 3 to 5 |
| | More Than |
| ||||||
Operating lease commitments(1) | | $ | 2,517 |
| | $ | 522 |
| | $ | 1,701 |
| | $ | 294 |
| | $ | — |
|
Total | | $ | 2,517 |
| | $ | 522 |
| | $ | 1,701 |
| | $ | 294 |
| | $ | — |
|
(1) Amounts reflect payments due for our leased office spaces in Watertown, Massachusetts as of June 30, 2023. The Term Loan provides for monthly interest-only payments until February 2021. Thereafter, payments are payablelease terms began in equal monthly installments of principal, plus all accruedNovember 2021 and unpaid interest. We may prepay the Term Loan in whole upon 5 days’ prior written notice to SVB. Any such prepayment of the Term Loan is subject to a prepayment charge of 0.5% of the then outstanding principal balance. Amounts outstanding during an event of default are payable upon SVB’s demandJune 2023, and will accrue interest at an additional rate of 5.0% per annum of the past due amount outstanding.
In April 2020, we entered into a deferral agreement with SVB to defer scheduled principal repayments on its term loan by six months. The deferral agreement was offeredeach end in connection with SVB’s venture debt relief initiative, which was started due to the COVID-19 pandemic. Our first principal payment under our credit facility occurred in February 2021. The required monthly interest-only payment was not impacted by the deferral. The Term Loan’s new maturity date is January 2023.
At the end of the loan term (whether at maturity, by prepayment in full or otherwise), we are required to pay a final end of term charge to SVB in the amount of 4.0% of the aggregate original principal amount advanced by SVB.
Convertible promissory notes
In August 2020, we entered into a purchase agreement with existing investors to issue $14.0 million in convertible promissory notes, (the “Notes”). The Notes accrued simple interest at 8% per annum and matured in February 2021. The Notes served as a bridge loan prior to the PIPE Financing that was completed in connection with the closing of the Business Combination. The Notes were amended to allow for the principal and interest to convert to shares of Series B preferred stock prior to the closing of the Business Combination. Accordingly, immediately prior to the closing of the Business Combination, the outstanding principal and interest under the Notes converted into 2,341,316 shares of Series B preferred stock at a per share conversion price of $6.1986.
Contract research and manufacturing organizationsNovember 2026.
We enter into contracts in the normal course of business with CMOs, CROs, CDMOs and other third parties for the manufacture of our product candidates and to supportpreclinical studies, clinical trials and preclinical research studies and testing.manufacturing services. These contracts typically do not contain minimum purchase commitments and are generally cancelable at any time by us following a certain period ofupon written notice. Payments due upon cancellation consist only of payments for services provided or expenses incurred, including noncancelable obligations of our service providers, up to the date of cancellation. cancellation and, in the case of certain arrangements with CROs and CDMOs, may include non-cancelable fees. These payments are not included in the table above as the amount and timing of such payments are not fixed and estimable.
We recorded accrued expenseshave also entered into license agreements under which we are obligated to make specified milestone and royalty payments. We have not included future payments under these agreements in the table of approximately $0.8 millioncontractual obligations above since the payment obligations under these agreements are contingent upon future events such as regulatory milestones or generating product sales. We are unable to estimate the timing or likelihood of achieving these milestones or generating future product sales. For additional information about our license agreements and amounts that could become payable in the future under such agreements, see our condensed consolidated balance sheet for expenditures incurred by CROs and CMOs as of June 30, 2022.
Disc Merger Agreement
On August 9, 2022, we entered into the Disc Merger Agreement pursuant to which, subject to the satisfaction or waiver of the conditions therein, Gem Merger Sub will merge with and into Disc, with Disc continuing as the surviving company and a wholly-owned subsidiary of the Company. The Disc Merger was unanimously approved by our board of directors (the “Board”), and the Board resolved to
27
recommend approval of the Disc Merger Agreement to our shareholders. The closing of the Disc Merger is subject to approval by our stockholders and the stockholders of Disc and other customary closing conditions.
If we are unable to satisfy certain closing conditions or if other mutual closing conditions are not satisfied, Disc will not be obligated to complete the Disc Merger. The Disc Merger Agreement contains certain termination rights of each of us and Disc. Under certain circumstances detailed in the Disc Merger Agreement, we could be required to pay Disc a termination fee of $3,000,000 or Disc could be required to pay us a termination fee of $7,800,000. In addition, in certain circumstances upon the termination of the Disc Merger Agreement, we could be required to pay the costs and expenses of Disc in an amount not to exceed $750,000, or Disc could be required to pay our costs and expenses in an amount not to exceed $750,000.financial statements.
Critical accounting policiesAccounting Policies and significant judgments and estimatesEstimates
Our management’s discussion and analysis of our financial condition and results of operations is based on our condensed consolidated financial statements, which have been prepared in accordance with U.S. generally accepted accounting principles, in the United States (“GAAP”).or GAAP. The preparation of our condensed consolidated financial statements and related disclosures requires us to make estimates and judgments that affect the reported amounts of assets, liabilities, costs and expenses.expenses, and the disclosure of contingent assets and liabilities in our condensed consolidated financial statements. We base our estimates on historical experience, known trends and events and various other factors that we believe are reasonable under the circumstances, the results of which form the basis for
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making judgments about the carrying values of assets and liabilities that are not readily apparent from other sources. We evaluate our estimates and assumptions on an ongoing basis. Our actual results may differ from these estimates under different assumptions or conditions. See our Annual Report on Form 10-K for the year ended December 31, 2022 for more information about critical accounting policies as well as a description of our other significant accounting policies.
During the three and six months ended June 30, 2022, there wereThere have been no material changes to our critical accounting policies as reportedestimates from those described in Part II, Item 7. “Management’s Discussion and Analysis of Financial Condition and Results of Operations” included in our 2021 Annual Report on Form 10-K.10-K for the year ended December 31, 2022.
Recently Issued and Adopted Accounting Pronouncements
A description of recently issued and certain recently adopted accounting pronouncements
Refer that have or may potentially impact our financial position and results of operations is included in Note 2 to Note 3, Summary of Significant Accounting Policies, to our unaudited condensed consolidated financial statements included elsewhere in this Quarterly Report on Form 10-Q for information regarding recently issued accounting pronouncements.statements.
Emerging growth companyGrowth Company and smaller reporting company statusSmaller Reporting Company Status
We are an “emerging growth company,” as defined in the Jumpstart Our Business Startups Act of 2012, (“or the JOBS Act”),Act, and we may take advantage of certain exemptions from various reporting requirements that are applicable to other public companies that are not emerging growth companies. We may take advantage of these exemptions until we are no longer an emerging growth company under Section 107 of the JOBS Act, which provides that an emerging growth company can take advantage of the extended transition period afforded by the JOBS Act for the implementation of new or revised accounting standards. We have elected to avail ourselves of the extended transition period and, therefore, while we are an emerging growth company, we will not be subject to new or revised accounting standards at the same time that they become applicable to other public companies that are not emerging growth companies, unless we choose to early adopt a new or revised accounting standard. We will remain an emerging growth company until the earlier of (1) the last day of the fiscal year (a) following the fifth anniversary of the closing of our initial public offering which occurred in August 2020, (b) in which we have total annual gross revenue of at least $1.07$1.235 billion, or (c) in which we are deemed to be a large accelerated filer, which means the market value of our common stock that is held by non-affiliates exceeds $700 million as of the prior June 30th, and (2) the date on which we have issued more than $1.0 billion in non-convertible debt during the prior three-year period.
Additionally, we are a “smaller reporting company” as defined in Item 10(f)(1) of Regulation S-K. Smaller reporting companies may take advantage of certain reduced disclosure obligations, including, among other things, providing only two years of audited financial statements. We will remain a smaller reporting company until the last day of the fiscal year in which (i) the market value of our common stock held by non-affiliates exceeds $250 million as of the prior June 30, or (ii) our annual revenues exceed $100 million during such completed fiscal year and the market value of our common stock held by non-affiliates exceeds $700 million as of the prior June 30.
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ItemITEM 3. Quantitative and Qualitative Disclosures About Market Risk.QUANTITATIVE AND QUALITATIVE DISCLOSURES ABOUT MARKET RISKS
We areAs of June 30, 2023 and December 31, 2022, we had cash and cash equivalents of $377.6 million and $194.6 million, respectively, which consisted of cash and money market accounts. Interest income is sensitive to changes in the general level of interest rates; however, due to the nature of these investments, an immediate 10% change in market interest rates would not have a smaller reporting company as defined by Rule 12b-2material effect on the fair market value of the Exchange Act and Item 10(f)(1) of Regulation S-K. As such, we are not required to provide the information set forth in this item.our cash or cash equivalents.
Item 4. ControlsOur employees and Procedures.operations are primarily located in the United States. We have, from time to time, engaged in contracts with contractors or other vendors in a currency other than the U.S. dollar. To date, we have had minimal exposure to fluctuations in foreign currency exchange rates as the time period between the date that transactions are initiated, and the date of payment or receipt of payment is generally of short duration. Accordingly, we believe we do not have a material exposure to foreign currency risk.
Managements’ Inflation generally affects us by increasing our cost of labor and contract research. We do not believe that inflation had a material effect on our business, financial condition or results of operations during the six months ended June 30, 2023 and 2022.
ITEM 4. CONTROLS AND PROCEDURES
Evaluation of our Disclosure Controls and Procedures
As of June 30, 2022, our principal executive officerOur management, under the supervision and principal financial officer, after evaluating the effectiveness of our “disclosure controls and procedures” (as defined in the Exchange Act Rule 13a-15(e) or Rule 15d-15(e)), with the participation of our management,
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have each concluded that, asPrincipal Executive Officer (our Chief Executive Officer) and Principal Financial Officer (our Chief Financial Officer), has evaluated the effectiveness of the end of the period covered by this Quarterly Report on Form 10-Q, our disclosure controls and procedures were effectiveas of June 30, 2023. The term “disclosure controls and wereprocedures,” as defined in Rules 13a-15(e) and 15d-15(e) under the Exchange Act, means controls and other procedures of a company that are designed to ensure that information we are required to disclosebe disclosed by a company in the reports that we fileit files or submitsubmits under the Securities Exchange Act of 1934, as amended, is accumulated and communicated to management, including our principal executive officer and principal financial officer, as appropriate, to allow timely decisions regarding required disclosure, and is recorded, processed, summarized and reported, within the time periods specified in the Securities and Exchange CommissionSEC’s rules and forms. It should be noted that any system ofDisclosure controls isand procedures include, without limitation, controls and procedures designed to provide reasonable, but not absolute, assurancesensure that information required to be disclosed by a company in the system will achievereports that it files or submits under the Exchange Act is accumulated and communicated to the company’s management, including its stated goals under all reasonably foreseeable circumstances. Our principal executive officer and principal financial officer have each concludedofficers, as appropriate to allow timely decisions regarding required disclosure.
Management recognizes that any disclosure controls and procedures, no matter how well designed and operated, can provide only reasonable assurance of achieving their objectives, and management necessarily applies its judgment in evaluating the cost-benefit relationship of possible controls and procedures. Based on the evaluation of our disclosure controls and procedures as of June 30, 2022 are2023, our Principal Executive Officer and Principal Financial Officer concluded that, as of such date, our disclosure controls and procedures were effective at a level that provides suchthe reasonable assurances.assurance level.
Changes in Internal Control Over Financial Reporting
There were no changesNo change in our internal control over financial reporting (as such term is defined in Exchange Act Rules 13a-15(f) and 15d-15(f)) that under the Exchange Act) occurred during the threesix months ended June 30, 20222023 that havehas materially affected, or that areis reasonably likely to materially affect, our internal control over financial reporting.
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PART II—OTHER INFORMATION
ItemITEM 1. Legal Proceedings.LEGAL PROCEEDINGS
From time to time, we may be subject to legal proceedings and claims in the ordinary course of business. We are not currently subject toaware of any such proceedings or claims that we believe will have, individually or in the aggregate, a material legal proceedings.adverse effect on our business, financial condition or results of operations.
ItemITEM 1A. Risk Factors.RISK FACTORS
Set forth below are the risks that we believe are material to our investors and they should be carefully considered. If any of the following risks and uncertainties actually occurs, our business, prospects, financial condition and results of operations could be materially and adversely affected. The matters discussedrisks described below are not intended to be exhaustive and other factors not presently known to us or that we currently believe are immaterial may affect our business, prospects, financial condition and results of operations if they occur. This section contains forward-looking statements. You should refer to the explanation of the qualifications and limitations on forward-looking statements in this Quarterly Report on Form 10-Q include forward-looking statements that involve risks or uncertainties. These statements are neither promises nor guarantees, but are based on various assumptions by management regarding future circumstances, over many of which we have little or no control. A number of important risks and uncertainties, including those identified under the caption “Risk Factors” in Part I, Item 1A of our Annual Report on Form 10-K for the period ended December 31, 2021 and in subsequent filings, could cause our actual results to differ materially from those in the forward-looking statements. Other than the risk factors set forth below, there are no material changes to the risk factors described in our Annual Report on Form 10-K for the period ended December 31, 2021, as filed with the SEC on March 10, 2022. We may disclose changes to such factors or disclose additional factors from time to time in our future filings with the SEC.10-Q.
Risks Related to our Merger with Disc Medicine, Inc.Our Limited Operating History, Financial Position, and Capital Requirements
FailureOur limited operating history may make it difficult for you to complete,evaluate the success of our business to date and to assess our future viability.
We commenced operations in 2017 and are a clinical-stage biopharmaceutical company with a limited operating history. Biopharmaceutical product development is a highly speculative undertaking and involves a substantial degree of risk. Since our inception in October 2017, we have devoted substantially all of our efforts to organizing and staffing our company, business planning, capital raising, establishing and maintaining our intellectual property portfolio, building our pipeline of product candidates, conducting drug discovery activities, undertaking preclinical studies, conducting early-stage clinical trials, and providing general and administrative support for these operations. We have not yet demonstrated our ability to successfully develop any product candidate, obtain regulatory approvals, manufacture a commercial scale product or delays in completing, the proposed merger transaction with Disc (as defined below) announcedarrange for a third party to do so on August 10, 2022our behalf, or conduct sales and marketing activities necessary for successful product commercialization. Consequently, any predictions you make about our future success or viability may not be as accurate as they could materiallybe if we had a longer operating history or a history of successfully developing and adversely affect our results of operations, business, financial results and/or stock price.commercializing products.
In Februaryaddition, as our business grows, we may encounter unforeseen expenses, difficulties, complications, delays and other known and unknown factors. We will need to transition at some point from a company with a research and development focus to a company capable of supporting commercial activities. We may not be successful in such a transition.
We have incurred significant net losses since our inception and anticipate that we will continue to incur losses for the foreseeable future.
Our net losses were $38.7 million and $19.4 million for the six months ended June 30, 2023 and 2022, the Company announced a corporate restructuringrespectively. We had an accumulated deficit of $150.9 million as of June 30, 2023. Substantially all of our net losses have resulted from costs incurred in connection with our research and that it initiated a processdevelopment programs and from general and administrative costs associated with our operations. We expect our research and development expenses to evaluate strategic alternatives. After a comprehensive review of strategic alternatives, including identifying and reviewing potential candidates for a strategic transaction, on August 9, 2022, the Company entered into an Agreement and Plan of Merger and Reorganization (the “Disc Merger Agreement”) with Disc Medicine, Inc., a Delaware corporation (“Disc”), and Gemstone Merger Sub, Inc., a Delaware corporation and a wholly-owned subsidiary of the Company (“Gem Merger Sub”), pursuant to which, subject to the satisfaction or waiver of the conditions therein, Gem Merger Sub will merge with and into Disc (the “Disc Merger”), with Disc continuing as the surviving company and a wholly-owned subsidiary of the Company. The closing of the Disc Merger is subject to approval by the stockholders of the Company and Disc as well as other customary closing conditions, including the effectiveness of a registration statement filed with the SECincrease significantly in connection with the transaction. If the merger is completed, the businesscommencement and continuation of Discclinical trials of our product candidates. In addition, if we obtain regulatory approval for our product candidates, we will incur significant sales, marketing and manufacturing expenses. We also will continue to incur additional costs associated with operating as a public company and expect to continue to incur significant and increasing operating losses over the businessnext several years and for the foreseeable future. Because of the combined company. Any failurenumerous risks and uncertainties associated with developing pharmaceutical products, we are unable to satisfy a required condition to closing may prevent, delay or otherwise materially and adversely affectpredict the completionextent of the transaction, which could materially and adversely affect our results of operations, business, financial results and/or stock price. We cannot predict with certainty whetherany future losses or when we will become profitable, if at all. Even if we do become profitable, we may not be able to sustain or increase our profitability on a quarterly or annual basis.
The amount of our future losses is uncertain and our quarterly and annual operating results may fluctuate significantly in the future due to a variety of factors, many of which are outside of our control and may be difficult to predict, including the following:
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The cumulative effects of these factors could result in large fluctuations and unpredictability in our quarterly and annual operating results. As a result, comparing our operating results on a period-to-period basis may not be meaningful. This variability and unpredictability could also result in us failing to meet the expectations of industry or financial analysts or investors for any period. If our revenue or operating results fall below the expectations of analysts or investors or below any forecasts we may provide to the market, or if the forecasts we provide to the market are below the expectations of analysts or investors, the price of our common stock could decline substantially. Such a stock price decline could occur even if we have met any previously publicly stated guidance we may provide.
We have no products approved for commercial sale and have not generated any revenue from product sales.
Our ability to become profitable depends upon our ability to generate revenue. To date, we have not generated collaborative revenue from our product candidates and have not generated revenue from product sales, and do not expect to generate any revenue from the sale of products in the near future. We do not expect to generate significant revenue unless and until we obtain regulatory approval of, and begin to sell, one or more of our product candidates. Our ability to generate revenue depends on a number of factors, including, but not limited to, our ability to:
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If we do not achieve one or more of these factors in a timely manner or at all, we could experience significant delays or an inability to successfully commercialize our product candidates, which would materially harm our business. If we do not receive regulatory approvals for our product candidates, we may not be able to successfully consummate the proposed merger as currently contemplated under the Disc Merger Agreement or at all.continue our operations.
We will need to raise substantial additional funding. If we are unable to raise capital when needed or on terms acceptable to us, we may be forced to delay, reduce, or eliminate some of our product development programs or commercialization efforts.
The development of pharmaceutical products is capital-intensive. We are currently advancing our hematologic disease programs through preclinical and clinical development. We expect our expenses to significantly increase in connection with our ongoing activities, particularly as we continue the research and development of, initiate and complete clinical trials of, and seek regulatory approval for, our product candidates. In addition, depending on the status of regulatory approval or, if we obtain regulatory approval for any of our product candidates, we expect to incur significant commercialization expenses related to product sales, marketing, manufacturing and distribution. We may also need to raise additional funds sooner if we choose to pursue additional indications and/or geographies for our current or future product candidates or otherwise expand more rapidly than presently anticipated. Furthermore, we expect to incur additional costs associated with operating as a newly-public company. Accordingly, we will need to obtain substantial additional funding in connection with our continuing operations. If we are unable to raise capital when needed or on attractive terms, we may be forced to delay, reduce, or eliminate certain of our research and development programs or future commercialization efforts.
We believe that we have cash and cash equivalents that will enable us to fund operating expenses and capital expenditure requirements well into 2026. However, we have based this estimate on assumptions that may prove to be wrong, and we could exhaust our available capital resources sooner than expected. Our effortsfuture capital requirements will depend on and could increase significantly as a result of many factors, including:
Identifying potential product candidates and conducting preclinical development testing and clinical trials is a time-consuming, expensive and uncertain process that takes years to complete, and we may never generate the necessary data or results required to obtain regulatory approval and achieve product sales. In addition, our product candidates, if approved, may not achieve commercial success. Our commercial revenue, if any, will be derived from sales of products that we do not expect to be commercially available for several years, if at all. Accordingly, we will need to continue to rely on additional financing to achieve our business objectives.
Any additional fundraising efforts may divert our management from their day-to-day activities, which may materially adversely affect our results of operations and our business. Uncertainty as to whether the Disc Merger will be completed may affect our ability to recruit prospective employeesdevelop and commercialize our product candidates. Disruptions in the financial markets in general and more recently
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due to the COVID-19 pandemic may make equity and debt financing more difficult to obtain and may have a material adverse effect on our ability to meet our fundraising needs. We cannot guarantee that future financing will be available in sufficient amounts or on terms acceptable to retain and motivate existing employees. Employee retentionus, if at all.
If we are unable to obtain funding on a timely basis or on acceptable terms, we may be particularly challenging whilerequired to significantly curtail, delay or discontinue one or more of our research or development programs or the transaction is pending because employees may experience uncertainty about their roles following the transaction. Uncertaintycommercialization of any product that has received regulatory approval or be unable to expand our operations or otherwise capitalize on our business opportunities as to our futuredesired, which could adverselymaterially affect our business, financial condition and results of operations.
Raising additional capital may cause dilution to our relationship with collaborators, suppliers, vendors, regulatorsstockholders, restrict our operations or require us to relinquish rights to our technologies or product candidates.
Until such time, if ever, we can generate substantial product revenue, we expect to finance our cash needs through a combination of private and other business partners. For example, vendors, collaboratorspublic equity offerings, debt financings, collaborations, strategic alliances and other counterpartieslicensing arrangements. We do not have any committed external source of funds. The terms of any financing may defer decisions about working withadversely affect the holdings or the rights of our stockholders and the issuance of additional securities, whether equity or debt, by us, or seek to change existing business relationships with us. Changes to, or terminationthe possibility of existing business relationships could adversely affect our results of operations and financial condition, as well assuch issuance, may cause the market price of our common stock. The adverse effects of the pendency of the transaction could be exacerbated by any delays in completion of the transaction or termination of the Disc Merger Agreement.
Risks relatedshares to the failure to consummate, or delay in consummating, the proposed merger transaction with Disc include, but are not limited to, the following:
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The occurrence of any of these events individually or in combination could materiallyoperating results and adversely affect our results of operations, business, and our stock price.prospects.
We cannot be sure if or when the Disc Merger will be completed.
The consummation of the Disc Merger is subject to the satisfaction or waiver of various conditions, including the authorization of the Disc Merger by our shareholders and Disc’s shareholders. We cannot guarantee that the closing conditions set forth in the Disc Merger Agreement will be satisfied. If we are unable to satisfy certain closing conditions or if other mutual closing conditions are not satisfied, Disc will not be obligated to complete the Disc Merger. Under certain circumstances, we wouldalso could be required to pay Disc a termination fee of $3,000,000, and/seek funds through arrangements with collaborators or expense reimbursement of Disc of upotherwise at an earlier stage than otherwise would be desirable. If we raise funds through collaborations, strategic alliances or licensing arrangements with third parties, we may have to $750,000.
If the Disc Merger is not completed, our board of directors, in discharging its fiduciary obligationsrelinquish valuable rights to our shareholders, would evaluate other strategic alternativesintellectual property, future revenue streams, research programs or financing optionsproduct candidates, grant licenses on terms that may be available, which alternatives may not be as favorable to our shareholders as the Disc Merger, including a liquidationus or grant rights to develop and dissolution. Any future sale or merger, financing or other transaction, including a liquidation or dissolution, may be subjectmarket product candidates that we would otherwise prefer to further shareholder approval. We may also be unable to find, evaluate or complete other strategic alternatives,develop and market ourself, any of which may have a materiallymaterial adverse effect on our business.
Our efforts to complete the Disc Merger could cause substantial disruptions in,business, operating results and create uncertainty surrounding, our business, which may materially and adversely affect our results of operations and our business. Uncertainty as to whether the Disc Merger will be completed may affect our ability to recruit prospective employees or to retain and motivate existing employees. Employee retention may be particularly challenging while the transaction is pending because employees may experience uncertainty about their roles following the transaction. A substantial amount of our management’s and employees’ attention is being directed toward the completion of the transaction and thus is being diverted from our day-to-day operations. Uncertainty as to our future could adversely affect our business and our relationship with collaborators, suppliers, vendors, regulators and other business partners. For example, vendors, collaborators and other counterparties may defer decisions concerning working with us, or seek to change existing business relationships with us. Changes to, or termination of, existing business relationships could adversely affect our results of operations and financial condition, as well as the market price of our common stock. The adverse effects of the pendency of the transaction could be exacerbated by any delays in completion of the transaction or termination of the Disc Merger Agreement.
Until the Disc Merger is completed, the Disc Merger Agreement restricts Disc and us from taking specified actions without the consent of the other party, and requires us to operate in the ordinary course of business consistent with past practice. These restrictions may prevent Disc and us from making appropriate changes to our respective businesses or pursuing attractive business opportunities that may arise prior to the completion of the Disc Merger.
Any delay in completing the proposed Disc Merger may materially and adversely affect the timing and benefits that are expected to be achieved from the proposed Disc Merger.
The exchange ratio set forth in the Disc Merger Agreement is not adjustable based on the market price of our common stock, so the merger consideration at the closing of the Disc Merger may have a greater or lesser value than at the time the Disc Merger Agreement was signed.
The Disc Merger Agreement has set the exchange ratio for Disc capital stock being converted into the Company’s common stock, and the exchange ratio is based on the outstanding capital stock of Disc and the outstanding common stock of the Company, in each case immediately prior to the closing of the Disc Merger. Applying the exchange ratio formula in the Disc Merger Agreement, the former Disc equityholders immediately before the Disc Merger are expected to own approximately 72% of the outstanding capital stock of the combined company immediately following the Disc Merger, and the equityholders of Gemini immediately before the Disc Merger are expected to own approximately 28% of the outstanding capital stock of the combined company immediately following the Disc Merger, in each case, before giving effect to the concurrent financing and subject to certain assumptions detailed in the Disc Merger Agreement. Under certain circumstances further described in the Disc Merger Agreement, however, these ownership percentages may be adjustedprospects.
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upwardStockholders who hold contingent value rights, or downward based on the cash levels of the respective companies at the closing of the Disc Merger, and as a result, either our stockholders or the Disc stockholders could own less of the combined company than expected.
Any changes in the market price of our common stock before the completion of the Disc Merger will not affect the number of shares of our common stock issuable to Disc’s stockholders pursuant to the Disc Merger Agreement. Therefore, if before the completion of the Disc Merger the market price of our common stock declines from the market price on the date of the Disc Merger Agreement, then Disc’s stockholders could receive merger consideration with substantially lower value than the value of such merger consideration on the date of the Disc Merger Agreement. Similarly, if before the completion of the Disc Merger the market price of our common stock increases from the market price of our common stock on the date of the Disc Merger Agreement, then Disc’s stockholders could receive merger consideration with substantially greater value than the value of such merger consideration on the date of the Disc Merger Agreement. The Disc Merger Agreement does not include a price-based termination right.
The Disc Merger Agreement contains provisions that limit our ability to pursue alternatives to the Disc Merger, could discourage a potential competing acquiror of us from making an alternative transaction proposal and, in specified circumstances, could require us to pay a termination fee to Disc, which could significantly harm our financial condition and the market price of our common stock and negatively affect the future business and operations of each company.
The Disc Merger Agreement contains provisions that make it difficult for us to entertain a third-party proposal for an acquisition of our company. These provisions include our agreement not to solicit or initiate any additional discussions with third parties regarding other proposals for our acquisition, as well as restrictions on our ability to respond to such proposals, subject to fulfillment of certain fiduciary requirements of our board of directors.
If the proposed Disc Merger is not completed and the Disc Merger Agreement is terminated under certain circumstances, we may be required to pay Disc a termination fee of up to $3,000,000, and/or expense reimbursement of up to $750,000. Even if a termination fee is not payable in connection with a termination of the Disc Merger Agreement, the Company will have incurred significant fees and expenses, which must be paid whether or not the Disc Merger is completed. Further, if the proposed Disc Merger is not completed, it could significantly harm the market price of our common stock.
In addition, if the Disc Merger Agreement is terminated and the board of directors of the Company determines to seek another business combination, there can be no assurance that either we will be able to find a partner and close an alternative transaction on terms that are as favorable or more favorable than the terms set forth in the Disc Merger Agreement.
Lawsuits may be filed against us and the members of our board of directors arising out of the proposed merger, which may delay or prevent the proposed merger.
Putative stockholder complaints, including stockholder class action complaints, and other complaints may be filed against us, our board of directors, Disc, Disc’s board of directors and others in connection with the transactions contemplated by the Disc Merger Agreement. The outcome of litigation is uncertain, and we may not be successful in defending against any such future claims. Lawsuits that may be filed against us, our board of directors, Disc, or Disc’s board of directors could delay or prevent the Disc Merger, divert the attention of our management and employees from our day-to-day business and otherwise adversely affect our financial condition.
Our stockholdersCVRs, potentially may not receive any payment on the CVRs and the CVRs may otherwise expire valueless.valueless.
The Disc Merger Agreement contemplates that, at orOn December 29, 2022, prior to the Effective Time, the Company, the holder’s representative and theeffective time of our merger with Gemini, we entered into a Contingent Value Rights Agent (as defined inAgreement, or the CVR Agreement) will execute and deliverAgreement, with a contingent value rights agreement (the “CVR Agreement”),agent pursuant to which Gemini’s pre-merger common stockholders received one CVR for each person who asoutstanding share of immediately prior toGemini common stock held by such stockholders on December 29, 2022. Each CVR represents the Effective Time was a stockholder of record of the Company or had thecontractual right to receive payments, in the Company’s common stock will be entitled to receive a contractual contingent value right ("CVR") issued by the Company subject to and in accordance with the terms and conditions of the CVR Agreement. Each CVR will entitle the holder of the CVR to receive a certain numberform of shares of commonour stock, upon the actual receipt by us or our affiliates of certain proceeds derived from consideration paid to us as a result of the combined company calculated baseddisposition of Gemini’s pre-merger legacy assets, net of certain expenses and other deductions. Any payments under the CVR Agreement will be in the form of shares of our stock, determined on the quotientbasis of the gross proceeds, if any, received in connection with the sale, license, transfer, disposition or other monetizing event of any assets related to drug products, raw materials, and biological materials to which Gemini or any of its subsidiaries owned or had rights to immediately prior to the Effective Time and thea volume weighted average closing market price for the five (5) trading days prior to the date of issuance of the CVR. The right of our stockholders to derive any value from the CVRs will be contingent solely upon the disposition of such assets within the time periods specified in the CVR Agreement.issuance.
We may not be able to achieve successful results from the disposition of such assets as described above. If this is not achieved for any reason within the time periods specified in the CVR Agreement, no payments will be made under the CVRs, and the CVRs will expire valueless. There can be no assurance that any payment of any of our shares will be made or that any holders of CVRs will receive any amounts with respect thereto.
Risks Related to the Discovery and Development of Our Product Candidates
We have only successfully completed one Phase 1 clinical trial, and may be unable to successfully complete any additional clinical trials for any product candidates we develop. Certain of the Company’s officersour programs are still in preclinical development and directors may have interests in the proposed Disc Merger that are different from, or in conflict with or in additionnever advance to those of the Company’s stockholders generally.clinical development.
32We have completed one Phase 1 clinical trial and have not yet demonstrated our continued ability to successfully complete clinical trials, including large-scale, pivotal clinical trials, obtain regulatory approvals, manufacture a commercial scale product, or arrange for a third party to do so on our behalf, or conduct sales and marketing activities necessary for successful commercialization. Our programs are still in preclinical and early clinical development. Our clinical programs may not advance to the next stage of clinical development, and our preclinical programs may never advance to clinical development or through clinical development, as applicable. We currently only have two product candidates in clinical development. In July 2022, we initiated BEACON, a Phase 2 open-label, parallel-dose clinical trial of bitopertin in EPP and XLP patients that is being conducted at sites in Australia. Separately,
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in October 2022, we initiated AURORA, a Phase 2, randomized, double-blind, placebo-controlled clinical trial of bitopertin in EPP patients that is being conducted at sites in the United States. We completed our Phase 1 clinical trial of DISC-0974 in healthy volunteers. We initiated a Phase 1b/2 clinical trial of DISC-0974 in June 2022 in patients with anemia of MF, and initiated a separate Phase 1b/2 clinical trial in February 2023 in patients with non-dialysis dependent CKD and anemia. We plan to initiate a Phase 1 clinical trial of MWTX-003 in healthy volunteers in the second half of 2023. We may not initiate any clinical trial of our product candidates until we have submitted an IND to the FDA or comparable submissions with equivalent regulatory authorities and received regulatory clearance. We may not be able to submit INDs or other regulatory filings for bitopertin or any of our other product candidates on the timelines we expect, if at all. For example, we may experience manufacturing delays or other delays with IND-enabling studies. Moreover, we cannot be sure that submission of regulatory filings with the FDA or other regulatory authorities will result in such regulatory authorities allowing clinical trials to begin on a timely basis or at all, or that, once begun, such trials will be completed on schedule, if at all, or that issues will not arise that require us to revise, postpone, suspend or terminate our clinical trials. For example, we filed an IND in April 2022 with the FDA to initiate the AURORA Phase 2 trial of bitopertin in EPP patients, but the FDA initially placed the initiation of this trial on clinical hold; we received clearance to initiate the study in July 2022 after the study design was finalized with the FDA, and we initiated the study in October 2022. Commencing each of these clinical trials is subject to finalizing the trial design based on discussions with the FDA and other regulatory authorities. Any guidance we receive from the FDA or other regulatory authorities is subject to change. These regulatory authorities could change their position, including on the acceptability of our trial designs or the clinical endpoints selected, which may require us to complete additional clinical trials or result in the composition of stricter approval conditions than currently expected. For a further example, we relied on the data package generated by Roche to support our IND submission for bitopertin to initiate our AURORA Phase 2 clinical trial in patients with EPP, as well as our submission of an application with the Australian Therapeutic Goods Administration, or TGA, for our BEACON Phase 2 clinical trial in patients with EPP or XLP, and it is possible that the FDA or TGA, as applicable, may require us to conduct additional preclinical studies to support a future marketing application of bitopertin. Successful completion of our clinical trials is a prerequisite to submitting an NDA or a BLA to the FDA, a Marketing Authorisation Application, or MAA, to the European Medicines Agency, or EMA, or other marketing applications to regulatory authorities in other jurisdictions, for each product candidate and, consequently, the regulatory approval of each product candidate.
Certain officersA single well-controlled clinical trial may not be sufficient for approval. In general, the FDA requires two well-controlled clinical trials to support registration of a new drug or biologic. Exceptions may be made in cases of a severe disease with few treatment options, and directorsin principle this exception may appear applicable to many of the Companydiseases that we seek to treat, such as EPP, XLP, anemia of MF, DBA and others. Nonetheless, the FDA and other regulators may always require additional clinical trials to support regulatory approval.
If we are required to conduct additional preclinical studies or clinical trials or other testing of our product candidates beyond those that are currently contemplated, if we are unable to successfully complete clinical trials of our product candidates or other testing, if the results of these trials or tests are not positive or are only modestly positive or if there are safety concerns, we may:
Our programs are focused on the development of therapeutics for patients with hematologic diseases, which is a rapidly evolving area of science, and the approach we are taking to discover and develop product candidates is novel and may never lead to approved or marketable products.
The discovery and development of therapeutics for patients with hematologic diseases is an emerging field, and the scientific discoveries that form the basis for our efforts to discover and develop product candidates are relatively new. The scientific evidence to support the feasibility of developing product candidates based on these discoveries is both preliminary and limited. Although we believe, based on our preclinical work that our programs have intereststhe potential to provide disease-modifying therapies, clinical results in patients may not confirm this hypothesis or may only confirm it for certain alterations or certain indications. The patient populations for our product candidates are limited to those with specific hematologic diseases. We cannot be certain that the patient populations for each specific disease will be large enough to allow us to successfully obtain approval and commercialize our product candidates and achieve profitability.
Clinical product development involves a lengthy and expensive process, with an uncertain outcome.
Our preclinical studies and future and ongoing clinical trials may not be successful. Currently, all of our programs are in preclinical and early clinical development. It is impossible to predict when or if any of our product candidates will prove effective and safe in humans or will receive regulatory approval. Before obtaining regulatory approval from regulatory authorities for the
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sale of any product candidate, we must complete preclinical studies and then conduct extensive clinical trials to demonstrate the safety and efficacy of our product candidates or the safety, purity and potency of our biological product candidates in humans. There is no guarantee that our product candidates will advance in accordance with the timelines we anticipate, if at all. Clinical testing is expensive, difficult to design and implement, can take many years to complete and outcomes are uncertain. A failure of one or more clinical trials can occur at any stage of testing. The outcome of preclinical development testing and early clinical trials may not be predictive of the success of later clinical trials, and interim, top-line, initial, or preliminary results of a clinical trial do not necessarily predict final results. Moreover, preclinical and clinical data are often susceptible to varying interpretations and analyses, and many companies that have believed their product candidates performed satisfactorily in preclinical studies and clinical trials have nonetheless failed to obtain regulatory approval of their product candidates. Our preclinical studies and future and ongoing clinical trials may not be successful.
Additionally, some of the clinical trials we conduct may be open-label in study design and may be conducted at a limited number of clinical sites on a limited number of patients. An “open-label” clinical trial is one where both the patient and investigator know whether the patient is receiving the investigational product candidate or either an existing approved drug or placebo. Most typically, open-label clinical trials test only the investigational product candidate and sometimes may do so at different dose levels. Open-label clinical trials are subject to various limitations that may exaggerate any therapeutic effect as patients in open-label clinical trials are aware when they are receiving treatment. Open-label clinical trials may be subject to a “patient bias” where patients perceive their symptoms to have improved merely due to their awareness of receiving an experimental treatment. In addition, open-label clinical trials may be subject to an “investigator bias” where those assessing and reviewing the physiological outcomes of the clinical trials are aware of which patients have received treatment and may interpret the information of the treated group more favorably given this knowledge. The results from an open-label clinical trial may not be predictive of future clinical trial results when studied in a controlled environment with a placebo or active control.
In May 2021, we entered into the Roche Agreement, pursuant to which, among other things, Roche granted us an exclusive and sublicensable (subject to Roche’s consent, except with respect to affiliates) worldwide license under certain of Roche’s patent rights and know-how to develop and commercialize bitopertin. Although bitopertin was originally evaluated by Roche in over 4,000 individuals, Roche did not evaluate bitopertin in EPP or XLP, so the safety data generated from Roche’s clinical trials of bitopertin may not be predictive or indicative of the results of our clinical trials. Regulatory authorities may also raise questions regarding the transition in the future from Roche-manufactured drug substance to drug substance manufactured by us or another party, and we may be required to conduct comparability assessments, which could result in delays in development and additional costs. We may face similar challenges with respect to MWTX-003, for which pre-clinical results may not be indicative or predictive of future clinical trial results.
Because we are developing some of our product candidates for the treatment of diseases in which there is little clinical experience and, in some cases, using new endpoints or methodologies, the FDA or other regulatory authorities may not consider the endpoints of our clinical trials to predict or provide clinically meaningful results.
Many of our product candidates are designed to treat diseases for which there are few available therapeutic options. For example, in the United States there are currently no therapies approved to treat anemia of MF and there is only one approved therapy to treat EPP. As a result, the design and conduct of clinical trials of product candidates for the treatment of these diseases may take longer, be more costly or be less effective as part of the novelty of development in these diseases. In some cases, we may use new or novel endpoints or methodologies. The FDA or other regulatory authorities may not consider the endpoints of our clinical trials to be validated or clinically meaningful and we may need to conduct proof-of-concept studies or additional work to refine our endpoints and inform the design of future studies before initiating pivotal studies of our product candidates. Even if applicable regulatory authorities do not object to our proposed Disc Mergerendpoints in an earlier stage clinical trial, such regulatory authorities may require evaluation of additional or different clinical endpoints in later-stage clinical trials.
Even if the FDA does find our clinical trial success criteria to be sufficiently supported and clinically meaningful at the time, we may not achieve the pre-specified endpoint to a degree of statistical significance in any pivotal or other clinical trials we may conduct for our product candidates. Further, even if we do achieve the pre-specified criteria, our trials may produce results that are unpredictable or inconsistent with the results of the more traditional efficacy endpoints in the trial. The FDA also could change its view or give overriding weight to other efficacy endpoints over a primary endpoint, even if we achieve statistically significant results on that primary endpoint, if for example we do not do so on our secondary efficacy endpoints. The FDA also weighs the benefits of a product candidate against its risks and the FDA may view the efficacy results in the context of safety as not being supportive of approval. Other regulatory authorities in Europe and other countries may make similar findings with respect to these endpoints.
Interim, top-line, initial, and preliminary data from our clinical trials that we announce or publish from time to time may change as more patient data become available and are subject to confirmation, audit, and verification procedures that could result in material changes in the final data.
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From time to time, we may publicly disclose interim, top-line, initial, or preliminary data from our clinical trials, which is based on a preliminary analysis of then-available data, and the results and related findings and conclusions are subject to change following a more comprehensive review of the data. For example, we announced top-line results from the Phase 1 DISC-0974 clinical trial in June 2022 and initial results from our Phase 2 BEACON clinical trial of bitopertin in June 2023. We also may make assumptions, estimations, calculations and conclusions as part of our analyses of data, and may not have received or had the opportunity to fully and carefully evaluate all data. As a result, the interim, top-line, initial, or preliminary results that we report may differ from future results of the same trials, or different conclusions or considerations may qualify such results, once additional data have been received and fully evaluated. Interim, top-line, initial, and preliminary data from clinical trials are subject to the risk that one or more of the clinical outcomes may materially change as patient enrollment continues and more patient data become available. Interim, top-line, initial, and preliminary data also remain subject to audit and verification procedures that may result in the final data being materially different from the interests ofinterim, top-line, initial, or preliminary data we previously published. As a result, interim, top-line, initial, and preliminary data should be viewed with caution until the Company’s stockholders generally, including potentially, among others,final data are available. Adverse differences between interim, top-line, initial, or preliminary data and final data could significantly harm our business prospects and may cause the continued service as a director of the combined company, the acceleration of stock option vesting, and continued indemnification.
The closing of the Disc Merger will also result in the acceleration of vesting of options to purchase sharesprice of our common stock to fluctuate or decline.
Further, regulatory agencies and others, may not accept or agree with our assumptions, estimates, calculations, conclusions or analyses or may interpret or weigh the importance of data differently, which could adversely impact the potential of the particular program, the likelihood of obtaining regulatory approval of the particular product candidate, commercialization of any approved product and the business prospects of the company in general. In addition, the information we choose to publicly disclose regarding a particular study or clinical trial is derived from information that is typically extensive, and you or others may not agree with what we determine is material or otherwise appropriate information to include in our disclosure.
If the interim, top-line, initial, or preliminary data that we report differs from actual results, or if regulatory authorities or others, disagree with the conclusions reached, our ability to obtain approval for, and commercialize, our product candidates may be significantly impaired, which could materially harm our business, operating results, prospects or financial condition.
We may incur additional costs or experience delays in initiating or completing, or ultimately be unable to complete, the development and commercialization of our product candidates.
We may experience delays in initiating or completing our preclinical studies or clinical trials, including as a result of delays in obtaining, or failure to obtain, the FDA’s authorization to initiate clinical trials under future INDs. Additionally, we cannot be certain that preclinical studies or clinical trials for our product candidates will not require redesign, will enroll an adequate number of subjects on time, or will be completed on schedule, if at all. We may experience numerous unforeseen events during, or as a result of, preclinical studies and clinical trials that could delay or prevent our ability to receive regulatory authorizations, regulatory approval or commercialize our product candidates, including:
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We could encounter delays if a clinical trial is suspended or terminated by us, by the IRBs or ethics committees of the institutions at which such trials are being conducted or by the FDA or other regulatory authorities, or if the Data Safety Monitoring Board, or DSMB, for such trial recommends suspension or termination of the trial. Such authorities may impose or recommend such a suspension or termination or clinical hold due to a number of factors, including failure to conduct the clinical trial in accordance with regulatory requirements or our clinical protocols, adverse findings upon an inspection of the clinical trial operations or trial site by the FDA or other regulatory authorities, unforeseen safety issues or adverse side effects, failure to demonstrate a benefit from using a product, changes in governmental regulations or administrative actions or lack of adequate funding to continue the clinical trial. For example, we filed an IND in April 2022 with the FDA to initiate the AURORA Phase 2 trial of bitopertin in EPP patients, but the FDA initially placed the initiation of this trial on clinical hold; we received clearance to initiate the study in July 2022 after the study design was finalized with the FDA and initiated the study in October 2022. Many of the factors that cause, or lead to, a delay in the commencement or completion of clinical trials may also ultimately lead to the denial of regulatory approval of our product candidates. Further, the FDA may disagree with our clinical trial design or our interpretation of data from clinical trials or may change the requirements for approval even after it has reviewed and commented on the design for our clinical trials.
Moreover, principal investigators for our current and future clinical trials may serve as scientific advisors or consultants to us from time to time and receive compensation in connection with such services. Under certain circumstances, we may be required to report some of these relationships to the FDA or comparable foreign regulatory authorities. The FDA or comparable foreign regulatory authority may conclude that a financial relationship between us and a principal investigator has created a conflict of interest or otherwise affected the interpretation of the trial. The FDA or comparable foreign regulatory authority may therefore question the integrity of the data generated at the applicable clinical trial site, and the utility of the clinical trial itself may be jeopardized. This could result in a delay in approval, or rejection, of our marketing applications by the FDA or comparable foreign regulatory authority, as the case may be, and may ultimately lead to the denial of regulatory approval of one or more of our product candidates.
Our product development costs will also increase if we experience delays in testing or regulatory approvals. We do not know whether any of our future clinical trials will begin as planned, or whether any of our current or future clinical trials will need to be restructured or will be completed on schedule, if at all. Significant preclinical study or clinical trial delays also could shorten any periods during which we may have the exclusive right to commercialize our product candidates or allow our competitors to bring products to market before we do, which would impair our ability to successfully commercialize our product candidates and may significantly harm our business, operating results, financial condition and prospects.
If we experience delays or difficulties in the enrollment of patients in clinical trials, our receipt of necessary regulatory approvals could be delayed or prevented.
We may not be able to initiate or continue clinical trials for our product candidates if we are unable to locate and enroll a sufficient number of eligible patients to participate in these trials as required by the FDA or comparable foreign regulatory authorities, or as needed to provide appropriate statistical power for a given trial. In particular, because we are focused on patients with specific rare hematologic diseases for the development of our product candidates, our ability to enroll eligible patients may be limited or may result in slower enrollment than we anticipate.
We may experience difficulties with identifying specific patient populations for any defined trial cohorts. The patient eligibility criteria defined in our trial protocols, may limit the patient populations eligible for our clinical trials. We will also rely on the willingness and ability of clinicians to screen their patients, such as for specific genetic hematologic conditions, to indicate which patients may be eligible for enrollment in our clinical trials.
In addition, some of our competitors have ongoing clinical trials for product candidates that are intended to treat the same indications as our product candidates, and patients who would otherwise be eligible for our clinical trials may choose instead to enroll in clinical trials of our competitors’ product candidates.
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Additionally, the process of finding patients may prove costly. We also may not be able to identify, recruit or enroll a sufficient number of patients to complete our clinical trials because of the small patient populations with rare hematologic diseases, the perceived risks and benefits of the product candidates under study, the availability and efficacy of competing therapies and clinical trials, the proximity and availability of clinical trial sites for prospective patients, and the patient referral practices of physicians. If patients are unwilling to participate in our studies for any reason, the timeline for recruiting patients, conducting studies and obtaining regulatory approval of potential products may be delayed.
Patient enrollment may be affected by other factors, including:
Results from early preclinical studies and clinical trials of our programs and product candidates are not necessarily predictive of the results of later preclinical studies and clinical trials of our programs and product candidates. If we cannot replicate the results from earlier preclinical studies and clinical trials of our programs and product candidates in our later preclinical studies and clinical trials, we may be unable to successfully develop, obtain regulatory approval for and commercialize our product candidates.
Any results from early preclinical studies and clinical trials of bitopertin, DISC-0974, DISC-0998, MWTX-003 or our other product candidates or programs may not necessarily be predictive of the results from later preclinical studies and clinical trials. For example, DISC-0974 has undergone testing in healthy volunteers, a Phase 1b/2 clinical trial in patients with anemia of MF was initiated in June 2022 and a separate Phase 1b/2 clinical trial in patients with non-dialysis dependent CKD and anemia was initiated in February 2023. However, there can be no assurance that DISC-0974 will achieve the desired effects in these indications. Additionally, MWTX-003 has only been evaluated in pre-clinical models, which may not be indicative or predictive of future clinical trial results. Similarly, even if we are able to complete our planned preclinical studies and clinical trials of our product candidates according to our current development timeline, the results from such preclinical studies and clinical trials of our product candidates may not be replicated in subsequent preclinical studies or clinical trial results.
Many companies in the biopharmaceutical and biotechnology industries have suffered significant setbacks in late-stage clinical trials after achieving positive results in early-stage development, and we cannot be certain that we will not face similar setbacks. These setbacks have been caused by, among other things, preclinical findings made while clinical trials were underway, or safety or efficacy observations made in preclinical studies and clinical trials, including previously unreported adverse events. Moreover, preclinical and clinical data are often susceptible to varying interpretations and analyses and many companies that believed their product candidates performed satisfactorily in preclinical studies and clinical trials nonetheless failed to obtain regulatory approval.
Our clinical trials or those of our future collaborators may reveal significant adverse events not seen in prior preclinical studies or clinical trials and may result in a safety profile that could inhibit regulatory approval or market acceptance of any of our product candidates.
Before obtaining regulatory approvals for the commercial sale of any products, we must demonstrate through lengthy, complex and expensive preclinical studies and clinical trials that our product candidates regulated as drugs are safe and effective and our product candidates regulated as biologics are safe, pure and potent for use in each target indication. Clinical testing is expensive and can take many years to complete, and outcomes are inherently uncertain. Failure can occur at any time during the clinical trial process. Because our programs and product candidates are in an early stage of development, there is a high risk of failure, and we may never succeed in developing marketable products. There is typically an extremely high rate of attrition from the failure of product candidates proceeding through clinical trials. Product candidates in later stages of clinical trials also may fail to show the desired safety and efficacy profile despite having progressed through preclinical studies and initial clinical trials. For example, Roche had previously developed bitopertin as a potential therapy for certain symptoms of schizophrenia and obsessive-compulsive disorder, but discontinued the program for lack of efficacy in those indications after completing over 30 clinical trials in over 4,000 individuals. If the results of our ongoing or future preclinical studies and clinical trials are inconclusive with respect to the safety and efficacy of our programs and product candidates, if we do not meet the clinical endpoints with statistical and clinically meaningful significance, or if there are safety concerns associated with our product candidates, we may be prevented
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from, or delayed in, obtaining regulatory approval for such product candidates. In some instances, there can be significant variability in safety or efficacy results between different clinical trials of the same product candidate due to numerous factors, including changes in trial procedures set forth in protocols, differences in the size and type of the patient populations, changes in and adherence to the clinical trial protocols and the rate of dropout among clinical trial participants. Results of our trials could reveal a high and unacceptable severity and prevalence of side effects. In such an event, our trials could be suspended or terminated, and the FDA or comparable foreign regulatory authorities could order us to cease further development of or deny approval of our product candidates for any or all targeted indications. Treatment-related side effects could also affect patient recruitment or the ability of enrolled patients to complete the trial or result in potential product liability claims.
Further, our product candidates could cause undesirable side effects in clinical trials related to on-target toxicity. If on-target toxicity is observed, or if our product candidates have characteristics that are unexpected, we may need to abandon their development or limit development to more narrow uses or subpopulations in which the undesirable side effects or other characteristics are less prevalent, less severe or more acceptable from a risk-benefit perspective. In addition, our product candidates could cause undesirable side effects that have not yet been observed. For example, bitopertin may demonstrate toxicities in patients with hematologic diseases not previously observed by Roche when it was studied in different indications. Many compounds that initially showed promise in early-stage testing have later been found to cause side effects that prevented further development of the compound. Most product candidates that commence clinical trials are never approved as products, and there can be no assurance that any of our current or future clinical trials will ultimately be successful or support further clinical development or regulatory approval of any of our product candidates.
As is the case with many treatments for hematologic and rare diseases, it is likely that there may be side effects associated with the use of our product candidates. If significant adverse events or other side effects are observed in any of our current or future clinical trials, we may have difficulty recruiting patients to our clinical trials, patients may drop out of our trials, or we may be required to abandon the trials or development efforts of one or more product candidates altogether. We, the FDA or other applicable regulatory authorities, or an IRB may suspend or terminate clinical trials of a product candidate at any time for various reasons, including a belief that subjects in such trials are being exposed to unacceptable health risks or adverse side effects. Even if the side effects do not preclude the product from obtaining or maintaining regulatory approval, undesirable side effects may inhibit market acceptance of the approved product due to its tolerability versus other therapies. Any of these developments could materially harm our business, operating results, financial condition and prospects.
Some of our product candidates modulate pathways for which there are currently no approved or effective therapies, which may result in greater research and development expenses, regulatory issues that could delay or prevent approval, or discovery of unknown or unanticipated adverse effects on safety or efficacy.
Some of our product candidates modulate pathways for which there are currently no approved or effective therapies, which may result in uncertainty. We select programs for targets based on compelling biological rationale, including evidence of expected biological effects in humans. We explore new programs based on extensive preclinical data analysis which sometimes cannot predict efficacy or safety in humans. Regulatory approval of novel product candidates such as ours can be more expensive, riskier and take longer than for other, more well-known or extensively studied pharmaceutical or biopharmaceutical product candidates due to our and regulatory agencies’ lack of experience with them. The novelty of the mechanism of action of any of our product candidates may lengthen the regulatory review process, require us to conduct additional studies or clinical trials, increase our development costs, lead to changes in regulatory positions and interpretations, delay or prevent approval and commercialization of our product candidates or lead to significant post-approval limitations or restrictions. The novel mechanism of action also means that fewer people are trained in or experienced with product candidates of this type, which may make it more difficult to find, hire and retain personnel for research, development and manufacturing positions. If our product candidates utilize a novel mechanism of action that has not been the subject of extensive study compared to more well-known product candidates, there is also an increased risk that we may discover previously unknown or unanticipated adverse effects during our preclinical studies and clinical trials. Our product candidates may achieve lower efficacy in patients than expected. Any such events could adversely impact our business prospects, operating results and financial condition.
We are currently conducting a Phase 2 clinical trial for bitopertin in Australia and may in the future conduct additional clinical trials of our product candidates outside the United States, and the FDA and comparable foreign regulatory authorities may not accept data from such trials.
In July 2022, we initiated BEACON, a Phase 2 open-label, parallel-dose clinical trial of bitopertin in EPP and XLP patients that is being conducted at sites in Australia. We may in the future choose to conduct additional clinical trials of our product candidates outside the United States, including in Europe, Australia, or other foreign jurisdictions. The acceptance of trial data from clinical trials conducted outside the United States by the FDA may be subject to certain conditions. In cases where data from clinical trials conducted outside the United States are intended to serve as the sole basis for regulatory approval in the United States, the FDA will generally not approve the application on the basis of foreign data alone unless (i) the data are applicable to the United States population and United States medical practices, (ii) the trials were performed by clinical investigators of recognized competence and (iii) the data may be considered valid without the need for an on-site inspection by the FDA or, if the FDA considers
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such an inspection to be necessary, the FDA is able to validate the data through an on-site inspection or other appropriate means. Additionally, the FDA’s clinical trial requirements, including sufficient size of patient populations and statistical powering, must be met. Many foreign regulatory bodies have similar approval requirements. In addition, such foreign trials will be subject to the applicable local laws of the foreign jurisdictions where the trials are conducted. There can be no assurance that the FDA or any comparable foreign regulatory authority, including the TGA, will accept data from trials conducted outside of the United States or the applicable jurisdiction. If the FDA or any comparable foreign regulatory authority does not accept such data, it would result in the need for additional trials, which would be costly and time-consuming and delay aspects of our business plan, and which may result in our product candidates not receiving regulatory approval or clearance for commercialization in the applicable jurisdiction.
Although we intend to explore other therapeutic opportunities in addition to the programs and product candidates that we are currently developing, we may fail to identify viable new product candidates for clinical development for a number of reasons. If we fail to identify additional product candidates, our business could be materially harmed.
Research programs to pursue the development of our existing and planned product candidates for additional indications and to identify new product candidates and disease targets require substantial technical, financial and human resources whether or not they are ultimately successful. Our research programs may initially show promise in identifying potential indications and/or product candidates, yet fail to yield results for clinical development for a number of reasons, including:
Because we have limited financial and human resources, we intend to initially focus on research programs and product candidates for a limited set of indications. As a result, we may forego or delay pursuit of opportunities with other product candidates or for other indications that later prove to have greater commercial potential or a greater likelihood of success. Our resource allocation decisions may cause us to fail to capitalize on viable commercial products or profitable market opportunities.
Accordingly, there can be no assurance that we will ever be able to identify additional therapeutic opportunities for our product candidates or to develop suitable product candidates through internal research programs, which could materially adversely affect our future growth and prospects. We may focus our efforts and resources on potential product candidates or other potential programs that ultimately prove to be unsuccessful.
If we are not able to obtain, or if there are delays in obtaining, required regulatory approvals for our product candidates, we will not be able to commercialize, or will be delayed in commercializing, our product candidates, and our ability to generate revenue will be materially impaired.
Our product candidates and the activities associated with their development and commercialization, including their design, testing, manufacture, safety, efficacy, recordkeeping, labeling, storage, approval, advertising, promotion, sale, distribution, import and export are subject to comprehensive regulation by the FDA and other regulatory agencies in the United States and by comparable foreign regulatory authorities. Before we can commercialize any of our product candidates, we must obtain regulatory approval. Currently, all of our product candidates are in discovery, preclinical or clinical development, and we have not received approval to market any of our product candidates from regulatory authorities in any jurisdiction. It is possible that our product candidates, including any product candidates we may seek to develop in the future, will never obtain regulatory approval. We have limited experience in filing and supporting the applications necessary to gain regulatory approvals and rely on third-party CROs and/or regulatory consultants to assist us in this process. Securing regulatory approval requires the submission of extensive preclinical and clinical data and supporting information to the various regulatory authorities for each therapeutic indication to establish the product candidate’s safety and efficacy. Securing regulatory approval also requires the submission of information about the product manufacturing process to, and inspection of manufacturing facilities by, the relevant regulatory authority. Our product candidates may not be effective, may be only moderately effective or may prove to have undesirable or unintended side effects, toxicities or other characteristics that may preclude us from obtaining regulatory approval or prevent or limit commercial use. In addition, regulatory authorities may find fault with our manufacturing process or facilities or that of third-party contract manufacturers. We may also face greater than expected difficulty in manufacturing our product candidates.
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The process of obtaining regulatory approvals, both in the United States and abroad, is expensive and often takes many years. If the FDA or a comparable foreign regulatory authority requires that we perform additional preclinical studies or clinical trials, approval may be delayed, if obtained at all. The length of such a delay varies substantially based upon a variety of factors, including the type, complexity and novelty of the product candidate involved. Changes in regulatory approval policies during the development period, changes in or enactment of additional statutes or regulations, or changes in regulatory review policies for each submitted NDA, BLA, or equivalent application types, may cause delays in the approval or rejection of an application. The FDA and comparable foreign regulatory authorities have substantial discretion in the approval process and may refuse to accept any application or may decide that our data is insufficient for approval and require additional preclinical, clinical or other studies. Our product candidates could be delayed in receiving, or fail to receive, regulatory approval for many reasons, including the following:
Even if we were to obtain regulatory approval, regulatory authorities may approve any of our product candidates for fewer or more limited indications than we request, thereby narrowing the commercial potential of the product candidate. In addition, regulatory authorities may grant approval contingent on the performance of costly post-marketing clinical trials, or may approve a product candidate with a label that does not include the labeling claims necessary or desirable for the successful commercialization of that product candidate. Any of the foregoing scenarios could materially harm the commercial prospects for our product candidates.
If we experience delays in obtaining, or if we fail to obtain, approval of our product candidates, the commercial prospects for our product candidates may be harmed and our ability to generate revenue will be materially impaired.
The COVID-19 pandemic, or a similar pandemic, epidemic, or outbreak of an infectious or highly contagious disease, may materially and adversely affect our business and financial results and could cause a disruption to the development of our product candidates.
Public health crises such as pandemics, including the COVID-19 pandemic, or similar outbreaks could adversely impact our business. The extent to which COVID-19, or the future outbreak of other highly infectious or contagious diseases impacts our operations or those of our third-party partners, including our preclinical studies or clinical trial operations, will depend on future developments, which are highly uncertain and cannot be predicted with confidence, including the scope, severity and duration of the outbreak, actions taken to contain the outbreak or mitigate its impact, and the direct and indirect economic effects of the outbreak and containment measures, among others.
In addition, the patient populations that our product candidates target may be particularly susceptible to COVID-19 or other highly infectious or contagious diseases, which may make it more difficult for us to identify patients able to enroll in our current and future clinical trials and may impact the ability of enrolled patients to complete any such trials.
Risks Related to Commercialization
We face substantial competition, which may result in others discovering, developing, or commercializing products before or more successfully than we do.
The development and commercialization of new products in the biopharmaceutical and related industries is highly competitive. We compete in the segments of the pharmaceutical, biotechnology, and other related markets that develop therapies in the field of hematologic diseases. There are other companies focusing on developing therapies in the field of hematologic diseases.
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We also compete more broadly across the market for cost-effective and reimbursable treatments. Some of these competitive products and therapies are based on scientific approaches that are the same as or similar to our approach, and others are based on entirely different approaches. These companies include divisions of large pharmaceutical companies and biotechnology companies of various sizes. We face competition with respect to our current product candidates, and will face competition with respect to any product candidates that we may seek to develop or commercialize in the future, from major pharmaceutical companies, specialty pharmaceutical companies and biotechnology companies worldwide. Potential competitors also include academic institutions, government agencies and other public and private research organizations that conduct research, seek patent protection and establish collaborative arrangements for research, development, manufacturing and commercialization.
Any product candidates that we successfully develop and commercialize will compete with currently approved therapies and new therapies that may become available in the future from segments of the pharmaceutical, biotechnology and other related markets. Key product features that would affect our ability to effectively compete with other therapeutics include the efficacy, safety and convenience of our products. We believe principal competitive factors to our business include, among other things, our ability to successfully transition research programs into clinical development, ability to raise capital, and the scalability of the platform, pipeline, and business.
Many of the companies that we compete against or which we may compete against in the future have significantly greater financial resources and expertise in research and development, manufacturing, preclinical and clinical testing, obtaining regulatory approvals and marketing approved products than we do. Mergers and acquisitions in the pharmaceutical and biotechnology industries may result in even more resources being concentrated among a smaller number of our competitors. Smaller or early-stage companies may also prove to be significant competitors, particularly through collaborative arrangements with large and established companies. These competitors also compete with us in recruiting and retaining qualified scientific and management personnel and establishing clinical trial sites and patient registration for clinical trials, as well as in acquiring technologies complementary to, or necessary for, our programs. If these or other barriers to entry do not remain in place, other companies may be able to more directly or effectively compete with us.
Our commercial opportunity could be reduced or eliminated if our competitors develop and commercialize products that are safer, more effective, have fewer or less severe side effects, are more convenient or are less expensive than any products that we or our collaborators may develop. Our competitors also may obtain FDA or other regulatory approval for their products sooner than we may obtain approval for our product candidates, which could result in our competitors establishing a strong market position before we or our collaborators are able to enter the market. The key competitive factors affecting the success of all of our product candidates, if approved, are likely to be their efficacy, safety, convenience, price, level of generic competition and availability of reimbursement from government and other third-party payors.
If the market opportunities for our programs and product candidates are smaller than we estimate or if any regulatory approval that we obtain is based on a narrower definition of the patient population, our revenue and ability to achieve profitability could be materially adversely affected.
The incidence and prevalence for the target patient populations of our programs and product candidates have not been established with precision. Our lead heme biosynthesis modulation product candidate, bitopertin, is an oral, selective inhibitor of GlyT1. We are initially focused on developing bitopertin for the treatment of EPP and XLP, which are both diseases marked by severe photosensitivity and damage to the hepatobiliary system caused by the accumulation of PPIX. In July 2022, we initiated BEACON, a Phase 2 open-label, parallel-dose clinical trial of bitopertin in EPP and XLP patients that is being conducted at sites in Australia. Separately, in October 2022, we initiated AURORA, a Phase 2, randomized, double-blind, placebo-controlled clinical trial of bitopertin in EPP patients that is being conducted at sites in the United States. We initiated a Phase 1b/2 clinical trial of DISC-0974 in June 2022 in the United States in patients with anemia of MF, and initiated a separate Phase 1b/2 clinical trial in the February 2023 in patients with non-dialysis dependent CKD and anemia. We are initially focused on developing MWTX-003 for the treatment of PV. Our projections of both the number of people who have these diseases, as well as the subset of people with these diseases who have the potential to benefit from treatment with our programs and product candidates, are based on our estimates.
The total addressable market opportunity will ultimately depend upon, among other things, the diagnosis criteria included in the final label, the indications for which our product candidates are approved for sale, acceptance by the medical community and patient access, product pricing and reimbursement. The number of patients with erythropoietic porphyria and anemias of inflammation for which our product candidates may be approved as treatment may turn out to be lower than expected, patients may not be otherwise amenable to treatment with our products, or new patients may become increasingly difficult to identify or gain access to, all of which would adversely affect our results of operations and our business. We may not be successful in our efforts to identify additional product candidates. Due to our limited resources and access to capital, we must prioritize development of certain product candidates, which may prove to be the wrong choice and may adversely affect our business.
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If our current product candidates or any future product candidates do not achieve broad market acceptance, the revenue that we generate from our sales may be limited, and we may never become profitable.
We have never commercialized a product candidate for any indication. Even if our current product candidates and any future product candidates are approved by the appropriate regulatory authorities for marketing and sale, they may not gain acceptance among physicians, patients, third-party payors, and others in the medical community. If any product candidates for which we may obtain regulatory approval do not gain an adequate level of market acceptance, we may not generate significant revenue and may not become profitable or may be significantly delayed in achieving profitability. Market acceptance of our current product candidates and any future product candidates by the medical community, patients and third-party payors will depend on a number of factors, some of which are beyond our control. For example, physicians are often reluctant to switch their patients, and patients may be reluctant to switch, from existing therapies even when new and potentially more effective or safer treatments enter the market. If public perception is influenced by claims that the use of heme biosynthesis modulation therapies or hepcidin-targeted agents is unsafe, whether related to our or our competitors’ products, our products may not be accepted by the general public or the medical community. Future adverse events in the hematologic diseases or the biopharmaceutical industry could also result in greater governmental regulation, stricter labeling requirements and potential regulatory delays in the testing or approvals of our product candidates.
In the United States and markets in other countries, patients generally rely on third-party payors to reimburse all or part of the costs associated with their treatment. Adequate coverage and reimbursement from governmental healthcare programs, such as Medicare and Medicaid, and commercial payors is critical to new product acceptance. Our ability to successfully commercialize our product candidates will depend in part on the extent to which coverage and adequate reimbursement for these products and related treatments will be available from government health administration authorities, private health insurers and other organizations. Even if coverage is provided, the approved reimbursement amount may not be high enough to allow us to establish or maintain pricing sufficient to realize a sufficient return on our investment. Government authorities and third-party payors, such as private health insurers and health maintenance organizations, decide which medications they will pay for and establish reimbursement levels.
Efforts to educate the medical community and third-party payors on the benefits of our current product candidates and any future product candidates may require significant resources and may not be successful. If our current product candidates or any future product candidates are approved but do not achieve an adequate level of market acceptance, we could be prevented from or significantly delayed in achieving profitability. The degree of market acceptance of any of our current product candidates and any future product candidates will depend on a number of factors, including:
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There is also significant uncertainty related to the insurance coverage and reimbursement of newly approved products and coverage may be more limited than the purposes for which the medicine is approved by the FDA or comparable foreign regulatory authorities. In the United States, the principal decisions about reimbursement for new medicines are typically made by the Centers for Medicare & Medicaid Services, or CMS, an agency within the U.S. Department of Health and Human Services. CMS decides whether and to what extent a new medicine will be covered and reimbursed under Medicare and private payors tend to follow CMS to a substantial degree. Further, due to the COVID-19 pandemic, millions of individuals have lost or will be losing employer-based insurance coverage, which may adversely affect our ability to commercialize our products. It is unclear what effect, if any, the American Rescue Plan will have on the number of covered individuals.
We may not be successful in addressing these or other factors that might affect the market acceptance of our product candidates. Failure to achieve widespread market acceptance of our product candidates would materially harm our business, financial condition and results of operations.
Even if we receive regulatory approval for any of our product candidates, we will be subject to ongoing regulatory obligations and continued regulatory review, which may result in significant additional expense. Additionally, our product candidates, if approved, could be subject to post-market study requirements, marketing and labeling restrictions, and even recall or market withdrawal if unanticipated safety issues are discovered following approval. In addition, we may be subject to penalties or other enforcement action if we fail to comply with regulatory requirements.
If the FDA or a comparable foreign regulatory authority approves any of our product candidates, the manufacturing processes, labeling, packaging, distribution, import, export, adverse event reporting, storage, advertising, promotion, monitoring, and recordkeeping for the product will be subject to extensive and ongoing regulatory requirements. These requirements include submissions of safety and other post-marketing information and reports, establishment registration and listing, as well as continued compliance with cGMPs and GCPs for any clinical trials that we conduct post-approval. Any regulatory approvals that we receive for our product candidates may also be subject to limitations on the approved indicated uses for which the product may be marketed or to the conditions of approval, or contain requirements for potentially costly post-marketing studies, including Phase 4 clinical trials, and surveillance to monitor the safety and efficacy of the product. The FDA may also require a REMS in order to approve our product candidates, which could entail requirements for a medication guide, physician communication plans or additional elements to ensure safe use, such as restricted distribution methods, patient registries and other risk minimization tools. For certain commercial prescription drug and biological products, manufacturers and other parties involved in the supply chain must also meet chain of distribution requirements and build electronic, interoperable systems for product tracking and tracing and for notifying the FDA of counterfeit, diverted, stolen and intentionally adulterated products or other products that are otherwise unfit for distribution in the United States. Additionally, under the Food and Drug Omnibus Reform Act of 2022, or FDORA, sponsors of approved drugs and biologics must provide 6 months’ notice to the FDA of any changes in marketing status, such as the withdrawal of a drug, and failure to do so could result in the FDA placing the product on a list of discontinued products, which would revoke the product’s ability to be marketed. Later discovery of previously unknown problems with a product, including adverse events of unanticipated severity or frequency, or with our third-party manufacturers or manufacturing processes, or failure to comply with regulatory requirements, may result in, among other things:
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The FDA’s and other regulatory authorities’ policies may change and additional government regulations may be enacted that could prevent, limit or delay regulatory approval of our product candidates. If we are slow or unable to adapt to changes in existing requirements or the adoption of new requirements or policies, or if we are not able to maintain regulatory compliance, we may lose any regulatory approval that we may have obtained, which would adversely affect our business, prospects and ability to achieve or sustain profitability.
Risks Related to Our Reliance on Third Parties
We rely on third parties to conduct our Phase 2 clinical trials of bitopertin, Phase 1b/2 clinical trials of DISC-0974 and planned Phase 1 clinical trial of MWTX-003 and expect to rely on third parties to conduct other clinical trials for our product candidates, as well as potential investigator-sponsored clinical trials of our product candidates. If these third parties do not successfully carry out their contractual duties, comply with regulatory requirements, or meet expected deadlines, we may not be able to obtain regulatory approval for or commercialize our product candidates and our business could be substantially harmed.
We do not have the ability to independently conduct clinical trials. We rely and expect to continue to rely on medical institutions, clinical investigators, contract laboratories and other third parties, such as CROs, to conduct or otherwise support clinical trials for our product candidates, including our Phase 2 clinical trials of bitopertin, Phase 1b/2 clinical trials of DISC-0974 in patients with anemia of MF or non-dialysis dependent CKD and anemia, our planned Phase 1 trial of MWTX-003, as well as any other product candidates that we develop. We may also rely on academic and private non-academic institutions to conduct and sponsor clinical trials relating to our product candidates, such as the ongoing clinical trial of bitopertin in DBA, which is being conducted by NIH under a collaborative research and development agreement. We will not control the design or conduct of any investigator-sponsored trials, and it is possible that the FDA or non-U.S. regulatory authorities will not view these investigator-sponsored trials as providing adequate support for future clinical trials, whether controlled by us or third parties, for any one or more reasons, including elements of the design or execution of the trials or safety concerns or other trial results.
Such arrangements will likely provide us certain information rights with respect to the investigator-sponsored trials, including access to and the ability to use and reference the data, including for our own regulatory filings, resulting from the investigator-sponsored trials. However, we would not have control over the timing and reporting of the data from investigator-sponsored trials, nor would we own the data from the investigator-sponsored trials. If we are unable to confirm or replicate the results from the investigator-sponsored trials or if negative results are obtained, we would likely be further delayed or prevented from advancing further clinical development of our product candidates. Further, if investigators or institutions breach their obligations with respect to the clinical development of our product candidates, or if the data proves to be inadequate compared to the first-hand knowledge we might have gained had the investigator-sponsored trials been sponsored and conducted by us, then our ability to design and conduct any future clinical trials ourself may be adversely affected.
We rely and expect to continue to rely heavily on these parties for execution of clinical trials for our product candidates and control only certain aspects of our activities. Nevertheless, we are responsible for ensuring that each of our clinical trials is conducted in accordance with the applicable protocol, legal and regulatory requirements and scientific standards, and our reliance on CROs or other third parties will not relieve us of our regulatory responsibilities. For any violations of laws and regulations during the conduct of our clinical trials, we could be subject to warning letters or enforcement action that may include civil penalties up to and including criminal prosecution.
We, our principal investigators and our CROs are required to comply with regulations, including GCPs, for conducting, monitoring, recording and reporting the results of clinical trials to ensure that the data and results are scientifically credible and accurate, and that the trial patients are adequately informed of the potential risks of participating in clinical trials and their rights are protected. These regulations are enforced by the FDA, the Competent Authorities of the Member States of the European Economic Area, or the EEA, and comparable foreign regulatory authorities for any products in clinical development. The FDA enforces GCP regulations through periodic inspections of clinical trial sponsors, principal investigators and trial sites. If we, our principal
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investigators or our CROs fail to comply with applicable GCPs, the clinical data generated in our clinical trials may be deemed unreliable and the FDA or comparable foreign regulatory authorities may require us to perform additional clinical trials before approving our marketing applications. We cannot assure you that, upon inspection, the FDA will determine that any of our future clinical trials will comply with GCPs. In addition, our clinical trials must be conducted with product candidates produced under current Good Manufacturing Practice, or cGMP, regulations. Our failure or the failure of our principal investigators or CROs to comply with these regulations may require us to repeat clinical trials, which would delay the regulatory approval process, significantly increase our expenditures and could also subject us to enforcement action. We also are required to register ongoing clinical trials and post the results of completed clinical trials on a government-sponsored database, ClinicalTrials.gov, within certain timeframes. Failure to do so can result in fines, adverse publicity and civil and criminal sanctions.
Although we designed our Phase 1b/2 clinical trials of DISC-0974 and ongoing Phase 2 clinical trials of bitopertin and intend to design the future clinical trials for our product candidates, these trials are or will be conducted by CROs and we expect CROs will conduct all of our future clinical trials. As a result, many important aspects of our development programs, including their conduct and timing, are outside of our direct control. Our reliance on third parties to conduct future clinical trials also results in less direct control over the management of data developed through clinical trials than would be the case if we were relying entirely upon our own staff. Communicating with outside parties can also be challenging, potentially leading to mistakes as well as difficulties in coordinating activities. Outside parties may:
These factors may materially adversely affect the willingness or ability of third parties to conduct our clinical trials and may subject us to unexpected cost increases that are beyond our control. If the principal investigators or CROs do not perform clinical trials in a satisfactory manner, breach their obligations to us or fail to comply with regulatory requirements, the development, regulatory approval and commercialization of our product candidates may be delayed, we may not be able to obtain regulatory approval and commercialize our product candidates or our development program may be materially and irreversibly harmed. If we are unable to rely on clinical data collected by our principal investigators or CROs, we could be required to repeat, extend the duration of, or increase the size of any clinical trials we conduct and this could significantly delay commercialization and require significantly greater expenditures.
If any of our relationships with these third-party principal investigators or CROs terminate, we may not be able to enter into arrangements with alternative CROs. If principal investigators or CROs do not successfully carry out their contractual obligations or meet expected deadlines, if they need to be replaced or if the quality or accuracy of the clinical data they obtain is compromised due to the failure to adhere to our clinical protocols, regulatory requirements or for other reasons, any clinical trials such principal investigators or CROs are associated with may be extended, delayed or terminated, and we may not be able to obtain regulatory approval for, or successfully commercialize, our product candidates. As a result, we believe that our financial results and the commercial prospects for our product candidates in the subject indication would be harmed, our costs could increase and our ability to generate revenue could be delayed.
We might not realize the anticipated benefits of our current collaborations with Mabwell or NIH, or any other collaborations we enter into in the future.
Research, development, commercialization and/or strategic collaborations, including those that we have with Mabwell and NIH, are subject to numerous risks, which include the following:
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In addition, funding provided by a collaborator might not be sufficient to advance product candidates under the collaboration. If a collaborator terminates a collaboration or a program under a collaboration, including by failing to exercise a license or other option under the collaboration, whether because we fail to meet a milestone or otherwise, any potential revenue from the collaboration would be significantly reduced or eliminated. In addition, we will likely need to either secure other funding to advance research, development and/or commercialization of the relevant product candidate or abandon that program, the development of the relevant product candidate could be significantly delayed, and our cash expenditures could increase significantly if we are to continue research, development and/or commercialization of the relevant product candidates.
Any one or more of these risks, if realized, could reduce or eliminate future revenue from product candidates under our collaborations, and could have a material adverse effect on our business, financial condition, results of operations and/or growth prospects.
We have established collaborations with Mabwell and NIH and may seek to establish additional collaborations, and, if we are not able to establish them on commercially reasonable terms, or at all, we may have to alter our development and commercialization plans.
Our product development programs and the potential commercialization of our product candidates will require substantial additional cash to fund expenses. For some of our product candidates, we may decide to collaborate with pharmaceutical and biotechnology companies, such as our collaborations with Mabwell and NIH, for the development and potential commercialization of those product candidates.
We face significant competition in seeking appropriate collaborators. Whether we reach a definitive agreement for a collaboration will depend, among other things, upon our assessment of the collaborator’s resources and expertise, the terms and conditions of the proposed collaboration and the proposed collaborator’s own evaluation of a potential collaboration. Such factors a potential collaborator will use to evaluate a collaboration may include the design or results of clinical trials, the likelihood of approval by the FDA or comparable foreign regulatory authorities, the potential market for the subject product candidate, the costs and complexities of manufacturing and delivering such product candidate to patients, the potential of competing products, the existence of uncertainty with respect to our ownership of technology, which can exist if there is a challenge to such ownership without regard to the merits of the challenge and industry and market conditions generally. The collaborator may also consider alternative product candidates or technologies for similar indications that may be available to collaborate on and whether such a collaboration could be more attractive than the one with us for our product candidate. The terms of any additional collaborations or other arrangements that we may establish may not be favorable to us.
We are also restricted by Roche’s right of first negotiation under our current license agreement with them and may in the future be restricted under other license or collaboration agreements from entering into future agreements on certain terms with potential collaborators. Collaborations are complex and time-consuming to negotiate and document. In addition, there have been a significant number of recent business combinations among large pharmaceutical companies that have resulted in a reduced number of potential future collaborators.
We may not be able to negotiate collaborations on a timely basis, on acceptable terms, or at all. If we are unable to do so, we may have to curtail the development of the product candidate for which we are seeking to collaborate, reduce or delay our development program or one or more of our other development programs, delay our potential commercialization or reduce the scope of any sales or marketing activities, or increase our expenditures and undertake development or commercialization activities at our own expense. If we elect to increase our expenditures to fund development or commercialization activities on our own, we may need to obtain additional capital, which may not be available to us on acceptable terms or at all. If we do not have sufficient funds, we may not be able to further develop our product candidates or bring them to market and generate product revenue.
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In addition, any future collaborations that we enter into may not be successful. The success of our collaboration arrangements will depend heavily on the efforts and activities of our collaborators. Collaborators generally have significant discretion in determining the efforts and resources that they will apply to these collaborations. Disagreements between parties to a collaboration arrangement regarding clinical development and commercialization matters can lead to delays in the development process or commercializing the applicable product candidate and, in some cases, termination of the collaboration arrangement. These disagreements can be difficult to resolve if neither of the parties has final decision-making authority. Collaborations with pharmaceutical or biotechnology companies and other third parties often are terminated or allowed to expire by the other party. Any such termination or expiration would adversely affect us financially and could harm our business reputation.
We contract with third parties for the manufacture of our product candidates for preclinical development and clinical testing, and expect to continue to do so for commercialization. This reliance on third parties increases the risk that we will not have sufficient quantities of our product candidates or products or such quantities at an acceptable cost, which could delay, prevent, or impair our development or commercialization efforts.
We do not currently own or operate, nor do we have any plans to establish in the future, any manufacturing facilities. Although we believe we have obtained sufficient material to produce bitopertin tablets to complete our ongoing Phase 2 clinical trials, DISC-0974 vials to complete our ongoing Phase 1b/2 clinical trials, and vials to complete our planned Phase 1 trial of MWTX-003, we cannot be sure we have correctly estimated our drug product and API requirements or that such drug product or API will not expire before we want to use it. While we have identified a contract development and manufacturing organization, or CDMO, to produce our own GMP material, we are in the early stages of manufacturing such material. We rely, and expect to continue to rely, on third parties for the manufacture of our product candidates for preclinical development and clinical testing, including Mabwell for the supply of vials to complete our planned Phase 1 trial of MWTX-003. We also expect to rely on third parties for the commercial manufacture of our products if any of our product candidates receive regulatory approval. This reliance on third parties increases the risk that we will not have sufficient quantities of our product candidates or products or such quantities at an acceptable cost or quality, which could delay, prevent or impair our development or commercialization efforts.
The facilities used by our CDMOs to manufacture our product candidates must be inspected by the FDA pursuant to pre-approval inspections that will be conducted after we submit our marketing applications to the FDA. We do not control the manufacturing process of, and will be completely dependent on, our CDMOs for compliance with cGMPs in connection with the manufacture of our product candidates. If our CDMOs cannot successfully manufacture material that conforms to our specifications and the strict regulatory requirements of the FDA or others, they will not be able to pass regulatory inspections and/or maintain regulatory compliance for our manufacturing facilities. In addition, we have no control over the ability of our CDMOs to maintain adequate quality control, quality assurance and qualified personnel. If the FDA or a comparable foreign regulatory authority finds deficiencies with or does not approve these facilities for the manufacture of our product candidates or if it finds deficiencies or withdraws any such approval in the future, we may need to find alternative manufacturing facilities, which would significantly impact our ability to develop, obtain regulatory approval for or market our product candidates, if approved.
If any CDMO with whom we contract fails to perform its obligations, we may be forced to enter into an agreement with a different CDMO, which we may not be able to do on reasonable terms, if at all. In such a scenario, our clinical trials supply could be delayed significantly as we established alternative supply sources. In some cases, the technical skills required to manufacture our product candidates may be unique or proprietary to the original CDMO and we may have difficulty, or there may be contractual restrictions prohibiting us from, transferring such skills to a back-up or alternate supplier, or we may be unable to transfer such skills at all. In addition, if we are required to change CDMOs for any reason, we will be required to verify that the new CDMO maintains facilities and procedures that comply with quality standards and with all applicable regulations. We will also need to verify, such as through a manufacturing comparability study, that any new manufacturing process will produce our product candidate according to the specifications previously submitted to the FDA or another regulatory authority. The delays associated with the verification of a new CDMO could negatively affect our ability to develop product candidates or commercialize our products in a timely manner or within budget. In addition, changes in manufacturers often involve changes in manufacturing procedures and processes, which could require that we conduct bridging studies between our prior clinical supply used in our clinical trials and that of any new manufacturer. We may be unsuccessful in demonstrating the comparability of clinical supplies which could require the conduct of additional clinical trials.
Further, our failure, or the failure of our CDMOs, to comply with applicable regulations could result in sanctions being imposed on it, including clinical holds, fines, injunctions, civil penalties, delays, suspension or withdrawal of approvals, license revocation, seizures or recalls of product candidates or products, if approved, operating restrictions and criminal prosecutions, any of which could significantly and adversely affect our business and supplies of our product candidates.
We may be unable to establish any additional agreements with CDMOs or do so on acceptable terms. Reliance on CDMOs entails additional risks, including:
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Our product candidates and any products that we may develop may compete with other product candidates and approved products for access to manufacturing facilities. There are a limited number of CDMOs that operate under cGMP regulations and that might be capable of manufacturing for us.
Any performance failure on the part of our existing or future manufacturers could delay clinical development or regulatory approval. If our current CDMOs cannot perform as agreed, we may be required to replace such CDMOs. We may incur added costs and delays in identifying and qualifying any such replacement.
Our current and anticipated future dependence upon others for the manufacture of our product candidates or products may adversely affect our future profit margins and our ability to commercialize any products that receive regulatory approval on a timely and competitive basis.
The third parties upon whom we rely for the supply of the active pharmaceutical ingredients used in our product candidates are our sole sources of supply, and the loss of any of these suppliers could significantly harm our business.
The active pharmaceutical ingredients, or API, used in certain of our product candidates are supplied to us from single-source suppliers. Our ability to successfully develop our product candidates, and to ultimately supply our commercial products in quantities sufficient to meet the market demand, depends in part on our ability to obtain the API for these products in accordance with regulatory requirements and in sufficient quantities for clinical testing and commercialization. We do not currently have arrangements in place for a redundant or second-source supply of any such API in the event any of our current suppliers of such API cease their operations for any reason. We are also unable to predict how changing global economic conditions or potential global health concerns such as the COVID-19 pandemic will affect our third-party suppliers and manufacturers. Any negative impact of such matters on our third-party suppliers and manufacturers may also have an adverse impact on our results of operations or financial condition.
For all of our product candidates, we intend to identify and qualify additional manufacturers to provide such API prior to submission of an NDA to the FDA and/or an MAA to the EMA. We are not certain, however, that our single-source suppliers will be able to meet our demand for their products, either because of the nature of our agreements with those suppliers, our limited experience with those suppliers or our relative importance as a customer to those suppliers. It may be difficult for us to assess our ability to timely meet our demand in the future based on past performance. While our suppliers have generally met our demand for their products on a timely basis in the past, they may subordinate our needs in the future to their other customers.
Establishing additional or replacement suppliers for the API used in our product candidates, if required, may not be accomplished quickly. If we are able to find a replacement supplier, such replacement supplier would need to be qualified and may require additional regulatory inspection or approval, which could result in further delay. While we seek to maintain adequate inventory of the API used in our product candidates, any interruption or delay in the supply of components or materials, or our inability to obtain such API from alternate sources at acceptable prices in a timely manner could impede, delay, limit or prevent our development efforts, which could harm our business, results of operations, financial condition and prospects.
The manufacture of biologics is complex and our third-party manufacturers may encounter difficulties in production. If any of our third-party manufacturers encounter such difficulties, our ability to provide supply of product candidates for clinical trials or products for patients, if approved, could be delayed or prevented.
DISC-0974, DISC-0998, and MWTX-003 are monoclonal antibodies. Manufacturing biologics, like monoclonal antibodies, especially in large quantities, is often complex and may require the use of innovative technologies to handle living cells. Each lot of an approved biologic must undergo thorough testing for identity, strength, quality, purity and potency. Manufacturing biologics requires facilities specifically designed for and validated for this purpose, and sophisticated quality assurance and quality control procedures are necessary. Slight deviations anywhere in the manufacturing process, including filling, labeling, packaging, storage and shipping and quality control and testing, may result in lot failures, product recalls or spoilage. When changes are made to the manufacturing process, we may be required to provide preclinical and clinical data showing the comparable identity, strength, quality, purity or potency of the products before and after such changes. If microbial, viral or other contaminations are discovered at the facilities of our manufacturers, such facilities may need to be closed for an extended period of time to investigate and remedy the contamination, which could delay clinical trials and adversely harm our business.
In addition, there are risks associated with large scale manufacturing for clinical trials or commercial scale including, among others, cost overruns, potential problems with process scale-up, process reproducibility, stability issues, compliance with good manufacturing practices, lot consistency and timely availability of raw materials. Even if we obtain regulatory approval for any of our current product candidates or any future product candidates, there is no assurance that our manufacturers will be able to manufacture the approved product to specifications acceptable to the FDA or other comparable foreign regulatory authorities, to produce it in sufficient quantities to meet the requirements for the potential commercial launch of the product or to meet potential future demand. If our manufacturers are unable to produce sufficient quantities for clinical trials or for commercialization, our
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development and commercialization efforts would be impaired, which would have an adverse effect on our business, financial condition, results of operations and growth prospects.
Risks Related to Our Intellectual Property
If we are unable to obtain and maintain patent and other intellectual property protection for our technology and product candidates, or if the scope of the intellectual property protection obtained is not sufficiently broad, our competitors could develop and commercialize technology and drugs similar or identical to ours, and our ability to successfully commercialize our technology and drugs may be impaired, and we may not be able to compete effectively in our market.
Our commercial success depends in part on our ability to obtain and maintain proprietary or intellectual property protection in the U.S. and other countries for our current or future product candidates, including our current lead product candidates, bitopertin, DISC-0974 and MWTX-003, and our other current or future programs, including DISC-0998, as well as for their respective compositions, formulations, methods used to manufacture them, and methods of treatment, in addition to successfully defending these patents against third-party challenges. We seek to protect our proprietary and intellectual property position by, among other methods, filing patent applications in the U.S. and abroad related to our proprietary technology, inventions, and improvements that are important to the development and implementation of our business. Our ability to stop unauthorized third parties from making, using, selling, offering to sell, or importing our product candidates is dependent upon the extent to which we have rights under valid and enforceable patents or trade secrets that cover these activities. We also rely on trade secrets, know-how and continuing technological innovation to develop and maintain our proprietary and intellectual property position.
We have in-licensed, and may in the future in-license, a portion of our intellectual property, and, if we fail to comply with our obligations under these license arrangements, we could lose such intellectual property rights or owe damages to the licensor of such intellectual property. In particular, we have exclusively licensed intellectual property rights from Roche to develop and commercialize bitopertin, including certain back-up compounds and derivatives, for all prophylactic and therapeutic uses. The Roche license covers know-how, and certain specified Roche patent rights, including a composition of matter patent for bitopertin that expires in 2025. We also have exclusively licensed intellectual property rights from AbbVie Deutschland GmbH & Co. KG, or AbbVie, to develop and commercialize DISC-0974 and DISC-0998. The AbbVie license covers know-how, and certain specified AbbVie patent rights, including composition of matter and methods of use patents and patent applications for DISC-0974 and DISC-0998. We also have exclusively licensed intellectual property rights from Mabwell to develop and commercialize antibody products containing Mabwell’s MWTX-001, MWTX-002, and MWTX-003 antibodies. The Mabwell license covers know-how, and certain specified Mabwell patent rights, including composition of matter and methods of use patents and patent applications for MWTX-001, MWTX-002, and MWTX-003.
The patent position of biotechnology and pharmaceutical companies generally is highly uncertain, involves complex legal and factual questions and has in recent years been the subject of much litigation. The degree of patent protection we require to successfully commercialize our current or future product candidates may be unavailable or severely limited in some cases and may not adequately protect our rights or permit us to gain or keep any competitive advantage. We cannot provide any assurances that any of our patents have, or that any of our pending patent applications that mature into issued patents will include, claims with a scope sufficient to protect bitopertin, DISC-0974, MWTX-003 or our other current or future product candidates. In addition, if the breadth or strength of protection provided by our patent applications or any patents we may own or in-license is threatened, we could dissuade companies from collaborating with us to license, develop or commercialize current or future product candidates.
In addition, the laws of foreign countries may not protect our rights to the same extent as the laws of the U.S. For example, in jurisdictions outside the U.S., a license may not be enforceable unless all the owners of the intellectual property agree or consent to the license. Accordingly, any actual or purported co-owner of our patent rights could seek monetary or equitable relief requiring us to pay it compensation for, or refrain from, exploiting these patents due to such co-ownership. Furthermore, patents have a limited lifespan. In the U.S., and most other jurisdictions in which we have undertaken patent filings, the natural expiration of a patent is generally twenty years after it is filed, assuming all maintenance fees are paid. Various extensions may be available, on a jurisdiction-by-jurisdiction basis; however, the life of a patent, and thus the protection it affords, is limited. Additionally, our product candidates may or may not be eligible for such extensions or we may not be able to obtain such protections due to procedural or other reasons. Given the amount of time required for the development, testing and regulatory review of new product candidates, patents protecting such candidates might expire before or shortly after such candidates are commercialized. As a result, patents we may own or in-license may not provide us with adequate and continuing patent protection sufficient to exclude others from commercializing drugs similar or identical to our current or future product candidates, including generic versions of such drugs.
Other parties have developed technologies that may be related or competitive to our own, and such parties may have filed or may file patent applications, or may have received or may receive patents, claiming inventions that may overlap or conflict with those claimed in our own patent applications or issued patents, with respect to either the same compounds, methods, formulations or other subject matter, in either case that we may rely upon to dominate our patent position in the market. Publications of discoveries in the scientific literature often lag behind the actual discoveries, and patent applications in the U.S. and other jurisdictions are typically not published until at least 18 months after the earliest priority date of the patent filing, or, in some cases, not at all. Therefore, we cannot know with certainty whether we were the first to make the inventions claimed in patents we may own or in-license patents or pending patent applications, or that we were the first to file for patent protection of such inventions. As a result, the issuance, scope, validity, enforceability and commercial value of our patent rights cannot be predicted with any certainty.
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In addition, the patent prosecution process is expensive and time-consuming, and we may not be able to file and prosecute all necessary or desirable patent applications at a reasonable cost or in a timely manner. Further, with respect to certain pending patent applications covering our current or future product candidates, prosecution has yet to commence. Patent prosecution is a lengthy process, during which the scope of the claims initially submitted for examination by the relevant patent office(s) may be significantly narrowed by the time they issue, if they ever do. It is also possible that we will fail to identify patentable aspects of our research and development output before it is too late to obtain patent protection. Prosecution could require that claim scope narrow such that a clinical or product candidate or program is not adequately protected by the patent. Moreover, in some circumstances, we may not have the right to control the preparation, filing and prosecution of patent applications, or to maintain the patents, covering technology that we license from or to third parties. Therefore, these patents and applications may not be prosecuted and enforced in a manner consistent with the best interests of our business.
Even if we acquire patent protection that we expect should enable us to establish and/or maintain a competitive advantage, third parties may challenge the validity, enforceability or scope thereof, which may result in such patents being narrowed, invalidated or held unenforceable. The issuance of a patent is not conclusive as to its inventorship, scope, validity or enforceability, and our patents may be challenged in the courts or patent offices in the U.S. and abroad. We may become involved in post-grant proceedings such as opposition, derivation, reexamination, inter partes review, post-grant review, invalidation, or interference proceedings challenging our patent rights or the patent rights of others from whom we may in the future obtain licenses to such rights, in the U.S. Patent and Trademark Office, or USPTO, the European Patent Office, or EPO, or in other countries. In addition, we may be subject to a third-party submission to the USPTO, the EPO, or elsewhere, that may reduce the scope or preclude the granting of claims from our pending patent applications. Competitors may allege that they invented the inventions claimed in our issued patents or patent applications prior to us, or may file patent applications before we do. Competitors may also claim that we are infringing their patents and that we therefore cannot practice our technology as claimed under our patents or patent applications. Competitors may also contest our patents by claiming to an administrative patent authority or judge that the invention was not patent-eligible, was not original, was not novel, was obvious, and/or lacked inventive step, and/or that the patent application filing failed to meet relevant requirements relating to description, basis, enablement, clarity, and/or support; in litigation, a competitor could claim that our patents, if issued, are not valid or are unenforceable for a number of reasons. If a court or administrative patent authority agrees, we would lose our protection of those challenged patents.
In addition, we may in the future be subject to claims by our former employees or consultants asserting an ownership right in our patents or patent applications, as a result of the work they performed on our behalf. Although we generally require all of our employees, consultants and advisors and any other third parties who have access to our proprietary know-how, information or technology to assign or grant similar rights to their inventions to us, we cannot be certain that we have executed such agreements with all parties who may have contributed to our intellectual property, nor can we be certain that our agreements with such parties will be upheld in the face of a potential challenge, or that they will not be breached, for which we may not have an adequate remedy.
An adverse determination in any such submission or proceeding may result in loss of exclusivity or freedom to operate or in patent claims being narrowed, invalidated or held unenforceable, in whole or in part, which could limit our ability to stop others from using or commercializing similar or identical technology and drugs, without payment to it, or could limit the duration of the patent protection covering our technology and current or future product candidates. Such challenges may also result in our inability to manufacture or commercialize our current or future product candidates without infringing third-party patent rights. In addition, if the breadth or strength of protection provided by our patents and patent applications is threatened, it could dissuade companies from collaborating with us to license, develop or commercialize current or future product candidates.
Even if they are unchallenged, our issued patents and our pending patent applications, if issued, may not provide us with any meaningful protection or prevent competitors from designing around our patent claims to circumvent patents we may own or in-license by developing similar or alternative technologies or products in a non-infringing manner. For example, a third-party may develop a competitive product that provides benefits similar to one or more of our current or future product candidates but that has a different composition that falls outside the scope of our patent protection. If the patent protection provided by the patents and patent applications we hold or pursue with respect to our current or future product candidates is not sufficiently broad to impede such competition, our ability to successfully commercialize our current or future product candidates could be negatively affected, which would harm our business.
Furthermore, even if we are able to issue patents with claims of valuable scope in one or more jurisdictions, we may not be able to secure such claims in all relevant jurisdictions, or in a sufficient number to meaningfully reduce competition. Our competitors may be able to develop and commercialize their products, including products identical to ours, in any jurisdiction in which we are unable to obtain, maintain, or enforce such patent claims. Furthermore, generic manufacturers may develop, seek approval for and launch generic versions of our products, and may challenge the scope, validity or enforceability of our patents, requiring us to engage in complex, lengthy and costly litigation or other proceedings.
We also intend to rely on regulatory exclusivity for protection of our product candidates, if approved for commercial sale. Implementation and enforcement of regulatory exclusivity, which may consist of regulatory data protection and market protection, varies widely from country to country. Failure to qualify for regulatory exclusivity, or failure to obtain or to maintain the extent or duration of such protections that we expect for our product candidates, if approved, could affect our decision on whether to market the products in a particular country or countries or could otherwise have an adverse impact on our revenue or results of operations.
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Obtaining and maintaining our patent protection depends on compliance with various procedural, document submission, deadlines, fee payment and other requirements imposed by governmental patent agencies, and our patent protection could be reduced or eliminated if we fail to comply with these requirements. We may miss a filing deadline for patent protection on these inventions.
The USPTO and foreign governmental patent agencies require compliance with a number of procedural, documentary, fee payment and other similar provisions during the patent application process and after issuance of any patent. In addition, periodic maintenance fees, renewal fees, annuity fees and/or various other government fees are required to be paid periodically. While an inadvertent lapse can, in some cases, be cured by payment of a late fee, or by other means in accordance with the applicable rules, there are situations in which noncompliance can result in abandonment or lapse of the patent or patent application, resulting in partial or complete loss of patent rights in the relevant jurisdiction. Noncompliance events that could result in abandonment or lapse of a patent include, but are not limited to, failure to respond to official actions within prescribed time limits, non-payment of fees and failure to properly legalize and submit formal documents. In such an event, our competitors might be able to enter the market with similar or identical products or platforms, which could have a material adverse effect on our business prospects and financial condition.
If our trademarks and trade names for our products or company name are not adequately protected in one or more countries where we intend to market our products, we may delay the launch of product brand names, use different trademarks or tradenames in different countries, or face other potentially adverse consequences to building our product brand recognition.
Our trademarks or trade names may be challenged, infringed, diluted, circumvented or declared generic or determined to be infringing on other marks. We intend to rely on both registration and common law protection for our trademarks. We may not be able to protect our rights to these trademarks and trade names or may be forced to stop using these names, which we need for name recognition by potential partners or customers in our markets of interest. During the trademark registration process, we may receive Office Actions from the USPTO or from comparable agencies in foreign jurisdictions objecting to the registration of our trademark. Although we would be given an opportunity to respond to those objections, we may be unable to overcome such rejections. In addition, in the USPTO and in comparable agencies in many foreign jurisdictions, third parties are given an opportunity to oppose pending trademark applications and/or to seek the cancellation of registered trademarks. Opposition or cancellation proceedings may be filed against our trademark applications or registrations, and our trademark applications or registrations may not survive such proceedings. If we are unable to obtain a registered trademark or establish name recognition based on our trademarks and trade names, we may not be able to compete effectively and our business may be adversely affected.
If we are unable to adequately protect and enforce our trade secrets, our business and competitive position would be harmed.
In addition to the protection afforded by patents we may own or in-license, we seek to rely on trade secret protection, confidentiality agreements, and license agreements to protect proprietary know-how that may not be patentable, processes for which patents are difficult to enforce and any other elements of our product discovery and development processes that involve proprietary know-how, information, or technology that may not be covered by patents. Although we require all of our employees, consultants, advisors, and any third parties who have access to our proprietary know-how, information, or technology to enter into confidentiality agreements, trade secrets can be difficult to protect and we have limited control over the protection of trade secrets used by our collaborators and suppliers. We cannot be certain that we have or will obtain these agreements in all circumstances and we cannot guarantee that we have entered into such agreements with each party that may have or have had access to our trade secrets or proprietary information.
Moreover, any of these parties might breach the agreements and intentionally or inadvertently disclose our trade secret information and we may not be able to obtain adequate remedies for such breaches. In addition, competitors may otherwise gain access to our trade secrets or independently develop substantially equivalent information and techniques. Furthermore, the laws of some foreign countries do not protect proprietary rights and trade secrets to the same extent or in the same manner as the laws of the U.S. As a result, we may encounter significant problems in protecting and defending our intellectual property both in the U.S. and abroad. If we are unable to prevent unauthorized material disclosure of our intellectual property to third parties, we will not be able to establish or maintain a competitive advantage in our market, which could materially adversely affect our business, financial condition, results of operations and future prospects.
Enforcing a claim that a party illegally disclosed or misappropriated a trade secret is difficult, expensive and time-consuming, and the outcome is unpredictable. If we choose to go to court to stop a third party from using any of our trade secrets, we may incur substantial costs. These lawsuits may consume our time and other resources even if we are successful. Although we take steps to protect our proprietary information and trade secrets, including through contractual means with our employees and consultants, third parties may independently develop substantially equivalent proprietary information and techniques or otherwise gain access to our trade secrets or disclose our technology. If any of our trade secrets were to be lawfully obtained or independently developed by a competitor or other third party, we would have no right to prevent them from using that technology or information to compete with us.
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Thus, we may not be able to meaningfully protect our trade secrets. It is our policy to require our employees, consultants, outside scientific collaborators, sponsored researchers and other advisors to execute confidentiality agreements upon the commencement of employment or consulting relationships with us. These agreements provide that all confidential information concerning our business or financial affairs developed or made known to the individual or entity during the course of the party’s relationship with us is to be kept confidential and not disclosed to third parties except in specific circumstances. In addition, we take other appropriate precautions, such as physical and technological security measures, to guard against misappropriation of our proprietary technology by third parties. In the case of employees, the agreements provide that all inventions conceived by the individual, and which are related to our current or planned business or research and development or made during normal working hours, on our premises or using our equipment or proprietary information, are our exclusive property. Although we require all of our employees to assign their inventions to us, we may be unsuccessful in executing such an agreement with each party who, in fact, conceives or develops intellectual property that we regard as our own. The assignment of intellectual property rights may not be self-executing, or the assignment agreements may be ineffective or breached, and we may be forced to bring claims against third parties, or defend claims that they may bring against us, to determine the ownership of what we regard as our intellectual property. Such claims could have a material adverse effect on our business, financial condition, results of operations, and prospects.
We may initiate, become a defendant in, or otherwise become party to lawsuits to protect or enforce our intellectual property rights, which could be expensive, time-consuming, and unsuccessful.
Competitors may infringe any patents we may own or in-license. In addition, any patents we may own or in-license also may become involved in inventorship, priority, validity or unenforceability disputes. To counter infringement or unauthorized use, we may be required to file infringement claims, which can be expensive and time-consuming. We may not prevail in any lawsuits that we initiate, and the damages or other remedies awarded, if any, may not be commercially meaningful. In addition, in an infringement proceeding, a court may decide that one or more of any patents we may own or in-license is not valid or is unenforceable or that the other party’s use of our technology that may be patented falls under the safe harbor to patent infringement under 35 U.S.C. § 271(e)(1). There is also the risk that, even if the validity of these patents is upheld, the court may refuse to stop the other party from using the technology at issue on the grounds that any patents we may own or in-license do not cover the technology in question or that such third-party’s activities do not infringe our patent applications or any patents we may own or in-license. An adverse result in any litigation or defense proceedings could put one or more of any patents we may own or in-license at risk of being invalidated, held unenforceable, or interpreted narrowly and could put our patent applications at risk of not issuing. Such litigation or proceedings could substantially increase our operating losses and reduce the resources available for development activities or any future sales, marketing, patient support or distribution activities. We may not have sufficient financial or other resources to conduct such litigation or proceedings adequately. Some of our competitors may be able to sustain the costs of such litigation or proceedings more effectively than we can because of its greater financial resources and more mature and developed intellectual property portfolios. Uncertainties resulting from the initiation and continuation of patent litigation or other proceedings could have a material adverse effect on our ability to compete in the marketplace.
Post-grant proceedings provoked by third parties or brought by or before the USPTO or other patent granting authority may be necessary to determine the validity or priority of inventions with respect to our patent applications or any patents we may own or in-license. These proceedings are expensive and an unfavorable outcome could result in a loss of our current patent rights and could require us to cease using the related technology or to attempt to license rights to us from the prevailing party. Our business could be harmed if the prevailing party does not offer us a license on commercially reasonable terms. In addition to potential USPTO post-grant proceedings, we may become a party to patent opposition proceedings in the EPO, or similar proceedings in other foreign patent offices or courts where our patents may be challenged. The costs of these proceedings could be substantial, and may result in a loss of scope of some claims or a loss of the entire patent. An unfavorable result in a post-grant challenge proceeding may result in the loss of our right to exclude others from practicing one or more of our inventions in the relevant country or jurisdiction, which could have a material adverse effect on our business. Litigation or post-grant proceedings within patent offices may result in a decision adverse to our interests and, even if we are successful, may result in substantial costs and distract our management and other employees. We may not be able to prevent, misappropriation of our trade secrets or confidential information, particularly in countries where the laws may not protect those rights as fully as in the U.S.
Furthermore, because of the substantial amount of discovery required in connection with intellectual property litigation, there is a risk that some of our confidential information could be compromised by disclosure during this type of litigation. In addition, there could be public announcements of the results of hearings, motions or other interim proceedings or developments. If securities analysts or investors perceive these results to be negative, it could have a substantial adverse effect on the price of our common stock.
We may not be able to detect infringement against any patents we may own or in-license. Even if we detect infringement by a third party of any patents we may own or in-license, we may choose not to pursue litigation against or settlement with the third party. If we later sue such third-party for patent infringement, the third-party may have certain legal defenses available to it, which otherwise would not be available except for the delay between when the infringement was first detected and when the suit was brought. Such legal defenses may make it impossible for us to enforce any patents we may own or in-license against such third party.
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Intellectual property litigation and administrative patent office patent validity challenges in one or more countries could cause us to spend substantial resources and distract our personnel from their normal responsibilities.
Even if resolved in our favor, litigation or other legal proceedings relating to intellectual property claims may cause us to incur significant expenses, and could distract our technical and management personnel from their normal responsibilities. In addition, there could be public announcements of the results of hearings, motions or other interim proceedings or developments and if securities analysts or investors perceive these results to be negative, it could have a substantial adverse effect on the price of our common stock. Such litigation or proceedings could substantially increase our operating losses and reduce the resources available for development activities or any future sales, marketing, patient support or distribution activities. We may not have sufficient financial or other resources to conduct such litigation or proceedings adequately. As noted above, some of our competitors may be able to sustain the costs of such litigation or proceedings more effectively than we can because of their greater financial resources. Uncertainties resulting from the initiation and continuation of patent litigation or other proceedings could compromise our ability to compete in the marketplace, including compromising our ability to raise the funds necessary to continue our clinical trials, continue our research programs, license necessary technology from third parties, or enter into development collaborations that would help us commercialize our current or future product candidates, if approved. Any of the foregoing events would harm our business, financial condition, results of operations and prospects.
We may be subject to damages or settlement costs resulting from claims that we or our employees have violated the intellectual property rights of third parties, or are in breach of our agreements. We may be accused of, allege or otherwise become party to lawsuits or disputes alleging wrongful disclosure of third-party confidential information by us or by another party, including current or former employees, contractors or consultants. In addition to diverting attention and resources to such disputes, such disputes could adversely impact our business reputation and/or protection of our proprietary technology.
The intellectual property landscape relevant to our product candidates and programs is crowded, and third parties may initiate legal proceedings alleging that we are infringing, misappropriating or otherwise violating their intellectual property rights, the outcome of which would be uncertain and could have a material adverse effect on the success of our business. Our commercial success depends upon our ability to develop, manufacture, market and sell our current and future product candidates and use our proprietary technologies without infringing, misappropriating or otherwise violating the intellectual property rights of third parties. There is a substantial amount of litigation involving patents and other intellectual property rights in the biotechnology and pharmaceutical industries, as well as administrative proceedings for challenging patents, including derivation, interference, reexamination, inter partes review and post grant review proceedings before the USPTO or oppositions and other comparable proceedings in foreign jurisdictions. We or any of our current or future licensors or strategic partners may be party to, exposed to, or threatened with, future adversarial proceedings or litigation by third parties having patent or other intellectual property rights alleging that our current or future product candidates and/or proprietary technologies infringe, misappropriate or otherwise violate their intellectual property rights. We cannot assure you that our current or future product candidates and other technologies that we have developed, are developing or may develop in the future do not or will not infringe, misappropriate or otherwise violate existing or future patents or other valid intellectual property rights owned by third parties. For example, many of our employees were previously employed at other biotechnology or pharmaceutical companies. Although we try to ensure that our employees, consultants and advisors do not use the proprietary information or know-how of others in their work for us, we may be subject to claims that we or these individuals have used or disclosed intellectual property, including trade secrets or other proprietary information, of any such individual’s former employer. We may also be subject to claims that patents and applications we have filed to protect inventions of our employees, consultants and advisors, even those related to one or more of our current or future product candidates, are rightfully owned by their former or concurrent employer. Litigation may be necessary to defend against these claims.
While certain activities related to development and clinical testing of our current or future product candidates may be subject to safe harbor of patent infringement, such as under 35 U.S.C. §271(e)(1), upon receiving regulatory approval for such candidates we or any of our current or future licensors or strategic partners may immediately become party to, exposed to, or threatened with, future adversarial proceedings or litigation by third parties having patent or other intellectual property rights alleging that such product candidates infringe, misappropriate or otherwise violate their intellectual property rights. Numerous U.S. and foreign issued patents and pending patent applications, which are owned by third parties, exist in the fields in which we are developing our current or future product candidates. As the biotechnology and pharmaceutical industries expand and more patents are issued, the risk increases that our current or future product candidates may give rise to claims of infringement of the patent rights of others. Moreover, it is not always clear to industry participants, including us, which patents cover various types of drugs, products or their methods of use or manufacture. Thus, because of the large number of patents issued and patent applications filed in our fields, there may be a risk that third parties may allege they have patent rights encompassing our current or future product candidates, technologies or methods.
If a third party claims that we infringe, misappropriate or otherwise violate its intellectual property rights, we may face a number of issues, including, but not limited to:
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Some of our competitors may be able to sustain the costs of complex patent litigation more effectively than we can because they have substantially greater resources. In addition, any uncertainties resulting from the initiation and continuation of any litigation could have a material adverse effect on our ability to raise the funds necessary to continue our operations or could otherwise have a material adverse effect on our business, results of operations, financial condition and prospects. The occurrence of any of the foregoing could have a material adverse effect on our business, financial condition, results of operations or prospects.
We may choose to challenge the patentability of claims in a third party’s U.S. patent by requesting that the USPTO review the patent claims in an ex-parte re-exam, inter partes review or post-grant review proceedings. These proceedings are expensive and may consume our time or other resources. We may choose to challenge a third party’s patent in patent opposition proceedings in the EPO, or other foreign patent office. The costs of these opposition proceedings could be substantial, and may consume our time or other resources. If we fail to obtain a favorable result at the USPTO, EPO or other patent office then we may be exposed to litigation by a third party alleging that the patent may be infringed by our current or future product candidates or proprietary technologies.
Third parties may assert that we are employing their proprietary technology without authorization. Patents issued in the U.S. by law enjoy a presumption of validity that can be rebutted in U.S. courts only with evidence that is “clear and convincing,” a heightened standard of proof. There may be issued third-party patents of which we are currently unaware with claims to compositions, formulations, methods of manufacture or methods for treatment related to the use or manufacture of our current or future product candidates. Patent applications can take many years to issue. In addition, because some patent applications in the U.S. may be maintained in secrecy until the patents are issued, patent applications in the U.S. and many foreign jurisdictions are typically not published until 18 months after their earliest priority filing date, and publications in the scientific literature often lag behind actual discoveries, we cannot be certain that others have not filed patent applications covering our current or future product candidates or technology. If any such patent applications issue as patents, and if such patents have priority over our patent applications or patents we may own or in-license, we may be required to obtain rights to such patents owned by third parties which may not be available on commercially reasonable terms or at all, or may only be available on a non-exclusive basis. There may be currently pending third-party patent applications which may later result in issued patents that our current or future product candidates may infringe. It is also possible that patents owned by third parties of which we are aware, but which we do not believe are relevant to our current or future product candidates or other technologies, could be found to be infringed by our current or future product candidates or other technologies. In addition, third parties may obtain patents in the future and claim that use of our technologies infringes upon these patents. Moreover, we may fail to identify relevant patents or incorrectly conclude that a patent is invalid, not enforceable, exhausted, or not infringed by our activities. If any third-party patents were held by a court of competent jurisdiction to cover the manufacturing process of our current or future product candidates, molecules used in or formed during the manufacturing process, or any final product itself, the holders of any such patents may be able to block our ability to commercialize the product candidate unless we obtained a license under the applicable patents, or until such patents expire or they are finally determined to be held invalid or unenforceable. Similarly, if any third-party patent were held by a court of competent jurisdiction to cover aspects of our formulations, processes for manufacture or methods of use, including patient selection methods, the holders of any such patent may be able to block our ability to develop and commercialize the product candidate unless we obtained a license or until such patent expires or is finally determined to be held invalid or unenforceable. In either case, such a license may not be
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available on commercially reasonable terms or at all. If we are unable to obtain a necessary license to a third-party patent on commercially reasonable terms, or at all, our ability to commercialize our current or future product candidates may be impaired or delayed, which could in turn significantly harm our business. Even if we obtain a license, it may be nonexclusive, thereby giving our competitors access to the same technologies licensed to us.
Parties making claims against us may seek and obtain injunctive or other equitable relief, which could effectively block our ability to further develop and commercialize our current or future product candidates. Defense of these claims, regardless of their merit, could involve substantial litigation expense and would be a substantial diversion of employee resources from our business. In the event of a successful claim of infringement, misappropriation or other violation against us, we may have to pay substantial damages, including treble damages and attorneys’ fees for willful infringement, obtain one or more licenses from third parties, pay royalties or redesign our infringing products, which may be impossible or require substantial time and monetary expenditure. We cannot predict whether any such license would be available at all or whether it would be available on commercially reasonable terms. Furthermore, even in the absence of litigation, we may need or may choose to obtain licenses from third parties to advance our research or allow commercialization of our current or future product candidates. We may fail to obtain any of these licenses at a reasonable cost or on reasonable terms, if at all. In that event, we would be unable to further develop and commercialize our current or future product candidates, which could harm our business significantly.
We may be unable to obtain patent or other intellectual property protection for our current or future product candidates or our future products, if any, in all jurisdictions throughout the world, and we may not be able to adequately enforce our intellectual property rights even in the jurisdictions where we seek protection.
We may not be able to pursue patent coverage of our current or future product candidates in all countries. Filing, prosecuting and defending patents on current or future product candidates in all countries throughout the world would be prohibitively expensive, and intellectual property rights in some countries outside the U.S. can be less extensive than those in the U.S. In addition, the laws of some foreign countries do not protect intellectual property rights to the same extent as federal and state laws in the U.S. Consequently, we may not be able to prevent third parties from practicing our inventions in all countries outside the U.S., or from selling or importing products made using our inventions in and into the U.S. or other jurisdictions. Competitors may use our technologies in jurisdictions where we have not obtained patent protection to develop their own products and further, may export otherwise infringing products to territories where we have patent protection, but where enforcement is not as strong as that in the U.S. These products may compete with our current or future product candidates and in jurisdictions where we do not have any issued patents, our patent applications or other intellectual property rights may not be effective or sufficient to prevent them from competing. Much of our patent portfolio is at the very early stage. We will need to decide whether and in which jurisdictions to pursue protection for the various inventions in our portfolio prior to applicable deadlines.
Many companies have encountered significant problems in protecting and defending intellectual property rights in foreign jurisdictions. The legal systems of certain countries, particularly certain developing countries, do not favor the enforcement of patents, trade secrets and other intellectual property protection, particularly those relating to biopharmaceutical products and/or methods of using biopharmaceutical products, which could make it difficult for us to stop the infringement of any patents we may own or in-license or marketing of competing products in violation of our proprietary rights generally. Proceedings to enforce any rights we may have in our patent applications or any patents we may own or in-license in foreign jurisdictions could result in substantial costs and divert our efforts and attention from other aspects of our business, could put any patents we may own or in-license at risk of being invalidated or interpreted narrowly and our patent applications at risk of not issuing and could provoke third parties to assert claims against us. We may not prevail in any lawsuits that we initiate and the damages or other remedies awarded, if any, may not be commercially meaningful. Accordingly, our efforts to enforce our intellectual property rights around the world may be inadequate to obtain a significant commercial advantage from the intellectual property that we develop or license.
Many countries have compulsory licensing laws under which a patent owner may be compelled to grant licenses to third parties. In addition, many countries limit the enforceability of patents against government agencies or government contractors. In these countries, the patent owner may have limited remedies, which could materially diminish the value of such patent. If we are forced to grant a license to third parties with respect to any patents we may own or license that are relevant to our business, our competitive position may be impaired, and our business, financial condition, results of operations, and prospects may be adversely affected.
We may not obtain or grant licenses or sublicenses to intellectual property rights in all markets on equally or sufficiently favorable terms with third parties.
It may be necessary for us to use the patented or proprietary technology of third parties to commercialize our products, in which case we would be required to obtain a license from these third parties. The licensing of third-party intellectual property rights is a competitive area, and more established companies may pursue strategies to license or acquire third-party intellectual property rights that we may consider attractive or necessary. More established companies may have a competitive advantage over us due to their size, capital resources and greater clinical development and commercialization capabilities. In addition, companies that perceive us to be a competitor may be unwilling to assign or license rights to us. We also may be unable to license or acquire third-party intellectual property rights on terms that would allow us to make an appropriate return on our investment or at all. If we are unable to license such technology, or if we are forced to license such technology on unfavorable terms, our business could be
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materially harmed. If we are unable to obtain a necessary license, we may be unable to develop or commercialize the affected current or future product candidates, which could materially harm our business, and the third parties owning such intellectual property rights could seek either an injunction prohibiting our sales, or, with respect to our sales, an obligation on our part to pay royalties or other forms of compensation. Even if we are able to obtain a license, it may be non-exclusive, thereby giving our competitors access to the same technologies licensed to us. Any of the foregoing could harm our competitive position, business, financial condition, results of operations and prospects.
If we fail to comply with our obligations in any agreements under which we may license intellectual property rights from third parties or otherwise experience disruptions to our business relationships with our licensors, we could lose license rights that are important to our business.
We are party to license agreements with Roche, AbbVie and Mabwell and we may from time to time in the future be party to other license and collaboration agreements with third parties to advance our research or allow commercialization of current or future product candidates. Such agreements may impose numerous obligations, such as development, diligence, payment, commercialization, funding, milestone, royalty, sublicensing, insurance, patent prosecution, enforcement and other obligations on us and may require us to meet development timelines, or to exercise commercially reasonable efforts to develop and commercialize licensed products, in order to maintain the licenses. See “Business-Collaborations and License Agreement” for more information regarding our license agreements with Roche, AbbVie and Mabwell. In spite of our best efforts, our licensors might conclude that we have materially breached our license agreements and might therefore terminate the license agreements, thereby removing or limiting our ability to develop and commercialize products and technologies covered by these license agreements.
Any termination of these licenses, or if the underlying patents fail to provide the intended exclusivity, could result in the loss of significant rights and could harm our ability to commercialize our current or future product candidates, and competitors or other third parties would have the freedom to seek regulatory approval of, and to market, products identical to ours and we may be required to cease our development and commercialization of certain of our current or future product candidates. Any of the foregoing could have a material adverse effect on our competitive position, business, financial conditions, results of operations, and prospects.
Disputes may also arise between us and our licensors regarding intellectual property subject to a license agreement, including:
In addition, the agreements under which we may license intellectual property or technology from third parties are likely to be complex, and certain provisions in such agreements may be susceptible to multiple interpretations. The resolution of any contract interpretation disagreement that may arise could narrow what we believe to be the scope of our rights to the relevant intellectual property or technology, or increase what we believe to be our financial or other obligations under the relevant agreement, either of which could have a material adverse effect on our business, financial condition, results of operations and prospects. Moreover, if disputes over intellectual property that we may license prevent or impair our ability to maintain future licensing arrangements on acceptable terms, we may be unable to successfully develop and commercialize the affected current or future product candidates, which could have a material adverse effect on our business, financial conditions, results of operations and prospects.
Any granted patents we may own or in-license covering our current or future product candidates or other valuable technology could be narrowed or found invalid or unenforceable if challenged in court or before administrative bodies in the U.S. or abroad, including the USPTO and the EPO. A patent asserted in a judicial court could be found invalid or unenforceable during the enforcement proceeding. Administrative or judicial proceedings challenging the validity of our patents or individual patent claims could take months or years to resolve.
If we or our licensors or strategic partners initiate legal proceedings against a third party to enforce a patent covering one of our current or future product candidates, the defendant could counterclaim that the patent covering our product candidate, as applicable, is invalid and/or unenforceable. In patent litigation in the U.S., defendant counterclaims alleging invalidity and/or unenforceability are commonplace, and there are numerous grounds upon which a third party can assert invalidity or unenforceability of a patent. Grounds for a validity challenge could be an alleged failure to meet any of several statutory requirements, including lack of patentable subject matter, lack of written description, lack of novelty, obviousness, or
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non-enablement. Grounds for an unenforceability assertion could be an allegation that someone connected with prosecution of the patent withheld relevant information from the USPTO that was material to patentability, or made a misleading statement, in the process of obtaining the patent during patent prosecution. Third parties may also raise similar claims before administrative bodies in the U.S. or abroad, even outside the context of litigation. Such mechanisms include re-examination, inter partes review, post grant review and equivalent proceedings in foreign jurisdictions (such as opposition proceedings). Such proceedings could result in revocation or amendment to our patent applications or any patents we may own or in-license in such a way that they no longer cover our current or future product candidates. The outcome following legal assertions of invalidity and unenforceability is unpredictable. An adverse determination in any such submission, proceeding or litigation could reduce the scope of, or invalidate or render unenforceable, any rights we may have from our patent applications or any patents we may own or in-license, allow third parties to commercialize our current or future product candidates or other technologies and compete directly with us, without payment to us, or result in our inability to manufacture or commercialize products without infringing third-party patent rights. Moreover, we may have to participate in interference proceedings declared by the USPTO to determine priority of invention or in post-grant challenge proceedings, such as oppositions in a foreign patent office, that challenge our or our current or future licensors’ priority of invention or other features of patentability with respect to our patent applications and any patents we may own or in-license. Such challenges may result in loss of patent rights, loss of exclusivity, or in patent claims being narrowed, invalidated, or held unenforceable, which could limit our ability to stop others from using or commercializing similar or identical technology and products, or limit the duration of the patent protection of our current or future product candidates and other technologies. With respect to the validity question, for example, we cannot be certain that there is no invalidating prior art, of which we or our current or future licensing partners and the patent examiner were unaware during prosecution. If a defendant were to prevail on a legal assertion of invalidity and/or unenforceability, or if we are otherwise unable to adequately protect our rights, we would lose at least part, and perhaps all, of the patent protection on our current or future product candidates. Such a loss of patent protection could have a material adverse impact on our business and our ability to commercialize or license our technology and current or future product candidates.
Such proceedings also may result in substantial cost and require significant time from our scientists and management, even if the eventual outcome is favorable to us. If we are unsuccessful in any such proceeding or other priority or inventorship dispute, we may be required to obtain and maintain licenses from third parties, including parties involved in any such interference proceedings or other priority or inventorship disputes. Such licenses may not be available on commercially reasonable terms or at all, or may be non-exclusive. If we are unable to obtain and maintain such licenses, we may need to cease the development, manufacture, and commercialization of one or more of the current or future product candidates we may develop. The loss of exclusivity or the narrowing of our patent application claims could limit our ability to stop others from using or commercializing similar or identical technology and products. Any of the foregoing could have a material adverse effect on our business, results of operations, financial condition and prospects.
Changes in patent law could diminish the value of patents in general, thereby impairing our ability to protect our current or future product candidates.
As is the case with other biopharmaceutical companies, our success is heavily dependent on intellectual property, particularly patents. Obtaining and enforcing patents in the biopharmaceutical industry involve both technological and legal complexity and is therefore costly, time consuming and inherently uncertain. Recent patent reform legislation in the U.S. and other countries, including the Leahy-Smith America Invents Act, or Leahy-Smith Act, signed into law on September 16, 2011, could increase those uncertainties and costs. The Leahy-Smith Act includes a number of significant changes to U.S. patent law. These include provisions that affect the way patent applications are prosecuted, redefine prior art and provide more efficient and cost-effective avenues for competitors to challenge the validity of patents. In addition, the Leahy-Smith Act has transformed the U.S. patent system into a “first inventor to file” system. The first-inventor-to-file provisions, however, only became effective on March 16, 2013. Accordingly, it is not yet clear what, if any, impact the Leahy-Smith Act will have on the operation of our business. However, the Leahy-Smith Act and its implementation could make it more difficult to obtain patent protection for our inventions and increase the uncertainties and costs surrounding the prosecution of our patent applications and the enforcement or defense of our issued patents, all of which could harm our business, results of operations and financial condition.
The U.S. Supreme Court has ruled on several patent cases in recent years, either narrowing the scope of patent protection available in certain circumstances or weakening the rights of patent owners in certain situations. Additionally, there have been recent proposals for additional changes to the patent laws of the U.S. and other countries that, if adopted, could impact our ability to obtain patent protection for our proprietary technology or our ability to enforce our proprietary technology. Depending on future actions by the U.S. Congress, the U.S. courts, the USPTO and the relevant law-making bodies in other countries, the laws and regulations governing patents could change in unpredictable ways that would weaken our ability to obtain new patents or to enforce our existing patents and patents that we might obtain in the future.
We may not identify relevant third-party patents or may incorrectly interpret the relevance, scope or expiration of a third-party patent, which might subject us to infringement claims or adversely affect our ability to develop and market our current or future product candidates.
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We cannot guarantee that any of our or our licensors’ patent searches or analyses, including the identification of relevant patents, the scope of patent claims or the expiration of relevant patents, are complete or thorough, nor can we be certain that we have identified each and every third-party patent and pending patent application in the U.S. and abroad that is relevant to or necessary for the commercialization of our current or future product candidates in any jurisdiction. For example, U.S. patent applications filed before November 29, 2000 and certain U.S. patent applications filed after that date that will not be filed outside the U.S. remain confidential until patents issue. As mentioned above, patent applications in the U.S. and elsewhere are published approximately 18 months after the earliest filing for which priority is claimed, with such earliest filing date being commonly referred to as the priority date. Therefore, patent applications covering our current or future product candidates could have been filed by third parties without our knowledge. Additionally, pending patent applications that have been published can, subject to certain limitations, be later amended in a manner that could cover our current or future product candidates or the use of our current or future product candidates. The scope of a patent claim is determined by an interpretation of the law, the written disclosure in a patent and the patent’s prosecution history. Our interpretation of the relevance or the scope and/or validity of a patent or a pending application may be incorrect, which may negatively impact our ability to market our current or future product candidates. We may incorrectly determine that our current or future product candidates are not covered by a third-party patent or may incorrectly predict whether a third party’s pending application will issue with claims of relevant scope. Our determination of the expiration date of any patent in the U.S. or abroad that we consider relevant may be incorrect, which may negatively impact our ability to develop and market our current or future product candidates. Our failure to identify and correctly interpret relevant patents may negatively impact our ability to develop and market our current or future product candidates.
If we fail to identify and correctly interpret relevant patents, we may be subject to infringement claims. We cannot guarantee that we will be able to successfully settle or otherwise resolve such infringement claims. If we fail in any such dispute, in addition to being forced to pay damages, which may be significant, we may be temporarily or permanently prohibited from commercializing any of our current or future product candidates that are held to be infringing. We might, if possible, also be forced to redesign current or future product candidates so that we no longer infringes the third-party intellectual property rights. Any of these events, even if we were ultimately to prevail, could require us to divert substantial financial and management resources that we would otherwise be able to devote to our business and could adversely affect our business, financial condition, results of operations and prospects.
Intellectual property rights do not guarantee commercial success of current or future product candidates or other business activities. Numerous factors may limit any potential competitive advantage provided by our intellectual property rights.
The degree of future protection afforded by our intellectual property rights, whether owned or in-licensed, is uncertain because intellectual property rights have limitations, and may not adequately protect our business, provide a barrier to entry against our competitors or potential competitors, or permit us to maintain our competitive advantage. Moreover, if a third party has intellectual property rights that cover the practice of our technology, we may not be able to fully exercise or extract value from our intellectual property rights. The following examples are illustrative:
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Should any of these events occur, they could significantly harm our business, financial condition, results of operations and prospects.
Risks Related to Government Regulation
Obtaining and maintaining regulatory approval of our product candidates in one jurisdiction does not mean that we will be successful in obtaining regulatory approval of our product candidates in other jurisdictions.
Obtaining and maintaining regulatory approval of our product candidates in one jurisdiction does not guarantee that we will be able to obtain or maintain regulatory approval in any other jurisdiction, while a failure or delay in obtaining regulatory approval in one jurisdiction may have a negative effect on the regulatory approval process in others. For example, even if the FDA grants regulatory approval of a product candidate, comparable regulatory authorities in foreign jurisdictions must also approve the manufacturing, marketing and promotion of the product candidate in those countries. Approval procedures vary among jurisdictions and can involve requirements and administrative review periods different from, and greater than, those in the United States, including additional preclinical studies or clinical trials as clinical trials conducted in one jurisdiction may not be accepted by regulatory authorities in other jurisdictions. In short, the foreign regulatory approval process involves all of the risks associated with FDA approval. In many jurisdictions outside the United States, a product candidate must be approved for reimbursement before it can be approved for sale in that jurisdiction. In some cases, the price that we may intend to charge for our products will also be subject to approval.
We may also submit marketing applications in other countries. Regulatory authorities in jurisdictions outside of the United States have requirements for approval of product candidates with which we must comply prior to marketing in those jurisdictions. Obtaining foreign regulatory approvals and compliance with foreign regulatory requirements could result in significant delays, difficulties and costs for us and could delay or prevent the introduction of our products in certain countries. If we fail to comply with the regulatory requirements in international markets and/or receive applicable regulatory approvals, our target market will be reduced and our ability to realize the full market potential of our product candidates will be harmed.
We may seek priority review designation for one or more of our product candidates, but we might not receive such designation, and even if we do, such designation may not lead to a faster regulatory review or approval process.
If the FDA determines that a product candidate offers a treatment for a serious condition and, if approved, the product would provide a significant improvement in safety or effectiveness, the FDA may designate the product candidate for priority review. A priority review designation means that the goal for the FDA to review an application is six months, rather than the standard review period of ten months. We may request priority review for our product candidates. The FDA has broad discretion with respect to whether or not to grant priority review status to a product candidate, so even if we believe a particular product candidate is eligible for such designation or status, the FDA may decide not to grant it. Moreover, a priority review designation does not necessarily result in an expedited regulatory review or approval process or necessarily confer any advantage with respect to approval compared to conventional FDA procedures. Receiving priority review from the FDA does not guarantee approval within the six-month review cycle or at all.
We may seek orphan drug designation for certain of our product candidates, and we may be unsuccessful or may be unable to maintain the benefits associated with orphan drug designation, including the potential for market exclusivity.
We have received orphan drug designation from the FDA for bitopertin for the treatment of EPP. As part of our business strategy, we may seek orphan drug designation for certain of our product candidates and indications, as appropriate. Regulatory authorities in some jurisdictions, including the United States and Europe, may designate drugs for relatively small patient populations as orphan drugs. Under the Orphan Drug Act, the FDA may designate a drug or biologic as an orphan drug if it is a product intended to treat a rare disease or condition, which is generally defined as a patient population of fewer than 200,000 individuals annually in the United States, or a patient population of 200,000 or more in the United States where there is no reasonable expectation that the cost of developing the product will be recovered from sales in the United States. In the United States, orphan drug designation entitles a party to financial incentives such as opportunities for grant funding towards clinical trial costs, tax advantages and user-fee waivers.
Similarly, in the European Union, the European Commission, upon the recommendation of the EMA’s Committee for Orphan Medicinal Products, grants orphan designation to promote the development of products that are intended for the diagnosis,
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prevention or treatment of life-threatening or chronically debilitating conditions affecting no more than 5 in 10,000 persons in the European Union and for which no satisfactory method of diagnosis, prevention, or treatment has been authorized for marketing in the European Union (or, if such a method exists, the product would be of a significant benefit to those affected by the condition). Additionally, designation is granted for products intended for the diagnosis, prevention, or treatment of a life-threatening, seriously debilitating or serious and chronic condition and when, without incentives, it is unlikely that sales of the product in the European Union would generate sufficient return to justify the necessary investment in developing the product. In the European Union, orphan designation entitles a party to financial incentives such as reduction of fees or fee waivers.
Generally, if a product with an orphan designation subsequently receives the first regulatory approval for the indication for which it has such designation, the product is entitled to a period of marketing exclusivity, which precludes the FDA or the EMA from approving another marketing application for the same product and indication for that time period, except in limited circumstances. The applicable period is seven years in the United States and ten years in the European Union. The European Union market exclusivity period can be reduced to six years if a product no longer meets the criteria for orphan designation or if the product is sufficiently profitable so that market exclusivity is no longer justified.
Even if we obtain orphan drug exclusivity for one of our product candidates, that exclusivity may not effectively protect our product candidate from competition because different products can be approved for the same condition. In addition, a designated orphan drug may not receive orphan drug exclusivity if it is approved for a use that is broader than the indication for which it received orphan designation. Moreover, orphan drug exclusive marketing rights in the United States may be lost if the FDA later determines that the request for designation was materially defective or if the manufacturer is unable to assure sufficient quantity of the product to meet the needs of patients with the rare disease or condition or if another product with the same active moiety is determined to be safer, more effective, or represents a major contribution to patient care. Orphan drug designation neither shortens the development time or regulatory review time of a product nor gives the product any advantage in the regulatory review or approval process. While we may seek orphan drug designation for our product candidates, we may never receive such designations. Even if we do receive such designations, there is no guarantee that we will enjoy the benefits of those designations.
The FDA may further reevaluate its regulations and policies under the Orphan Drug Act. We do not know if, when, or how the FDA may change its orphan drug regulations and policies in the future, and it is uncertain how any changes might affect our business. Depending on what changes the FDA may make to its orphan drug regulations and policies, our business could be adversely impacted.
We may seek rare pediatric disease designation for bitopertin. However, a marketing application for bitopertin, if approved, may not meet the eligibility criteria for a rare pediatric disease priority review voucher.
We may seek rare pediatric disease designation for bitopertin in patients with EPP and XLP. The FDA defines “rare pediatric disease” as a (i) serious or life-threatening disease in which the serious or life-threatening manifestations primarily affect ages from birth to 18 years, including age groups often called neonates, infants, children, and adolescents; and (ii) a rare disease or condition within the meaning of the Orphan Drug Act. Designation of a product candidate as a product for a rare pediatric disease does not guarantee that a marketing application for such product candidate will meet the eligibility criteria for a rare pediatric disease priority review voucher at the time the application is approved. Under the Federal Food, Drug, and Cosmetic Act, we will need to request a rare pediatric disease priority review voucher in our original marketing application for our product candidates for which we have received rare pediatric disease designation. The FDA may determine that a marketing application for bitopertin, if approved, does not meet the eligibility criteria for a priority review voucher.
Under the current statutory sunset provisions, after September 30, 2024, the FDA may only award a priority review voucher for an approved rare pediatric disease product application if the sponsor has rare pediatric disease designation for the drug or biologic that is the subject of such application, and that designation was granted by September 30, 2024. After September 30, 2026, the FDA may not award any rare pediatric disease priority review vouchers. However, it is possible the authority for FDA to award rare pediatric disease priority review vouchers will be further extended by Congress. As such, if we do not obtain approval of a marketing application for bitopertin in patients with EPP and XLP on or before September 30, 2026, and if the priority review voucher program is not extended by Congressional action, we may not receive a priority review voucher.
A breakthrough therapy designation and fast track designation by the FDA, even if granted for any of our product candidates, may not lead to a faster development, regulatory review or approval process, and each designation does not increase the likelihood that any of our product candidates will receive regulatory approval in the United States.
We may seek a breakthrough therapy designation for certain of our product candidates. A breakthrough therapy is defined as a drug or biologic that is intended, alone or in combination with one or more other drugs or biologics, to treat a serious or life-threatening disease or condition and preliminary clinical evidence indicates that the drug or biologic may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development. For product candidates that have been designated as breakthrough therapies, interaction and communication between the FDA and the sponsor of the trial can help to identify the most efficient path for clinical development while minimizing the number of patients placed in ineffective control regimens. Products designated as breakthrough therapies by
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the FDA may also be eligible for priority review and accelerated approval. Designation as a breakthrough therapy is within the discretion of the FDA. Accordingly, even if we believe one of our product candidates meets the criteria for designation as a breakthrough therapy, the FDA may disagree and instead determine not to make such designation. In any event, the receipt of a breakthrough therapy designation for a product candidate may not result in a faster development process, review or approval compared to therapies considered for approval under conventional FDA procedures and does not assure ultimate approval by the FDA. In addition, even if one or more of our product candidates are designated as breakthrough therapies, the FDA may later withdraw the designation if it determines that such product candidates no longer meet the conditions for such designation.
We may seek fast track designation for certain of our product candidates. If a drug or biologic is intended for the treatment of a serious or life-threatening condition and the drug or biologic demonstrates the potential to address unmet medical needs for this condition, the sponsor may apply for fast track designation. The FDA has broad discretion whether or not to grant this designation, so even if we believe a particular product candidate is eligible for this designation, the FDA may disagree and instead decide not to grant it. Even if we do receive fast track designation, we may not experience a faster development process, review or approval compared to conventional FDA procedures. The FDA may withdraw fast track designation if it believes that the designation no longer meets the conditions for such designation. Fast track designation alone does not guarantee qualification for the FDA’s priority review procedures.
Accelerated approval by the FDA, even if granted for our current or any other future product candidates, may not lead to a faster development or regulatory review or approval process and it does not increase the likelihood that our product candidates will receive regulatory approval.
We may seek accelerated approval of our current or future product candidates using the FDA’s accelerated approval pathway. A product may be eligible for accelerated approval if it treats a serious or life-threatening condition and generally provides a meaningful advantage over available therapies. In addition, it must demonstrate an effect on a surrogate endpoint that is reasonably likely to predict clinical benefit or on a clinical endpoint that can be measured earlier than irreversible morbidity or mortality, or IMM, that is reasonably likely to predict an effect on IMM or other clinical benefit. It is possible that at the time of submission of a marketing application, the FDA may determine that our product candidate is not eligible for accelerated approval or that accelerated approval is not warranted. Moreover, FDA may revise how it implements accelerated approval, which could negatively affect the development of our current or future product candidates.
As a condition of approval, the FDA generally requires that a sponsor of a drug or biologic receiving accelerated approval perform adequate and well-controlled post-marketing clinical trials. These confirmatory trials must be completed with due diligence. Under FDORA, the FDA is permitted to require, as appropriate, that a post-approval confirmatory trial or trials be underway prior to approval or within a specified time period after the date accelerated approval is granted. FDORA also requires sponsors to send updates to the FDA every 180 days on the status of such trials, including progress toward enrollment targets, and the FDA must promptly post this information publicly. FDORA also gives the FDA increased authority to withdraw approval of a drug or biologic granted accelerated approval on an expedited basis if the sponsor fails to conduct confirmatory studies in a timely manner, send the necessary updates to the FDA, or if such post-approval trials fail to verify the product’s predicted clinical benefit. Under FDORA, the FDA is empowered to take action, such as issuing fines, against companies that fail to conduct with due diligence any post-approval confirmatory trial or submit timely reports to the agency on their progress. In addition, the FDA currently requires, unless otherwise informed by the agency, pre-approval of promotional materials for products being considered for accelerated approval, which could adversely impact the timing of the commercial launch of the product. Even if we do receive accelerated approval, we may not experience a faster development or regulatory review or approval process, and receiving accelerated approval does not provide assurance of ultimate full FDA approval.
If our drug product candidates or any of our future drug product candidates obtain regulatory approval, additional competitors could enter the market with generic versions of such products, which may result in a material decline in sales of our competing products.
Under the Drug Price Competition and Patent Term Restoration Act of 1984, or the Hatch-Waxman Amendments to the Federal Food, Drug, and Cosmetic Act, or the FDCA, a company may file an abbreviated new drug application, or ANDA, seeking approval of a generic version of an approved innovator product. Under the Hatch-Waxman Amendments, a company may also submit an NDA under section 505(b)(2) of the FDCA that references the FDA’s prior approval of the innovator product or preclinical studies and/or clinical trials that were not conducted by, or for, the applicant and for which the applicant has not obtained a right of reference. A 505(b)(2) NDA product may be for a new or improved version of the original innovator product. The Hatch-Waxman Amendments also provide for certain periods of regulatory exclusivity, which preclude FDA approval (or in some circumstances, FDA filing and review) of an ANDA or 505(b)(2) NDA. In addition to the benefits of regulatory exclusivity, an innovator NDA holder may have patents claiming the active ingredient, product formulation or an approved use of the drug, which would be listed with the product in the FDA publication “Approved Drug Products with Therapeutic Equivalence Evaluations,” known as the Orange Book. If there are patents listed in the Orange Book for the applicable, approved innovator product, a generic or 505(b)(2) applicant that seeks to market our product before expiration of the patents must include in their applications what is known as a “Paragraph IV” certification, challenging the validity or enforceability, or claiming non-infringement, of the listed patent or patents.
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Notice of the certification must be given to the patent owner and NDA holder and if, within 45 days of receiving notice, either the patent owner or NDA holder sues for patent infringement, approval of the ANDA or 505(b)(2) NDA is stayed for up to 30 months.
Accordingly, if any of our product candidates that are regulated as drugs are approved, competitors could file ANDAs for generic versions of these products or 505(b)(2) NDAs that reference our products. If there are patents listed for such drug products in the Orange Book, those ANDAs and 505(b)(2) NDAs would be required to include a certification as to each listed patent indicating whether the ANDA applicant does or does not intend to challenge the patent. We cannot predict which, if any, patents in our current portfolio or patents we may obtain in the future will be eligible for listing in the Orange Book, how any generic competitor would address such patents, whether we would sue on any such patents or the outcome of any such suit.
We may not be successful in securing or maintaining proprietary patent protection for products and technologies we develop or licenses, despite expending a significant amount of resources that could have been focused on other areas of our business. Moreover, if any of our owned or in-licensed patents that are listed in the Orange Book are successfully challenged by way of a Paragraph IV certification and subsequent litigation, the affected product could immediately face generic competition and our sales would likely decline rapidly and materially.
If approved, our investigational products regulated as biologics may face competition from biosimilars or interchangeable products approved through an abbreviated regulatory pathway.
The Biologics Price Competition and Innovation Act of 2009, or BPCIA, created an abbreviated approval pathway for biologic products that are biosimilar to or interchangeable with an FDA-licensed reference biologic product. Under the BPCIA, an application for a biosimilar or interchangeable product may not be submitted to the FDA until four years following the date that the reference product was first licensed by the FDA. In addition, the approval of a biosimilar or interchangeable product may not be made effective by the FDA until 12 years from the date on which the reference product was first licensed. During this 12-year period of exclusivity, another company may still market a competing version of the reference product if the FDA approves a BLA for the competing product containing the sponsor’s own preclinical data and data from adequate and well-controlled clinical trials to demonstrate the safety, purity, and potency of the other company’s product. The law is complex and is still being interpreted and implemented by the FDA. As a result, its ultimate impact, implementation, and meaning are subject to uncertainty.
We believe that any of our product candidates approved as a biologic product under a BLA should qualify for the 12-year period of exclusivity. However, there is a risk that this exclusivity could be shortened due to congressional action or otherwise, or that the FDA will not consider our investigational medicines to be reference products for competing products, potentially creating the opportunity for generic competition sooner than anticipated. Other aspects of the BPCIA, some of which may impact the BPCIA exclusivity provisions, have also been the subject of recent litigation. Moreover, the extent to which a biosimilar or interchangeable product, once licensed, will be substituted for any one of our reference products in a way that is similar to traditional generic substitution for non-biologic products is not yet clear, and will depend on a number of marketplace and regulatory factors that are still developing. If competitors are able to obtain regulatory approval for biosimilars or interchangeable products referencing our products, our products may become subject to competition from such biosimilars or interchangeable products, with the attendant competitive pressure and consequences.
The FDA, the EMA and other regulatory authorities may implement additional regulations or restrictions on the development and commercialization of our product candidates, and such changes can be difficult to predict.
The FDA, the EMA and regulatory authorities in other countries have each expressed interest in further regulating biotechnology products. Agencies at both the federal and state level in the United States, as well as the U.S. Congressional committees and other governments or governing agencies, have also expressed interest in further regulating the biotechnology industry. Such action may delay or prevent commercialization of some or all of our product candidates. Adverse developments in clinical trials of products conducted by others may cause the FDA or other oversight bodies to change the requirements for approval of any of our product candidates. These regulatory review agencies and committees and the new requirements or guidelines they promulgate may lengthen the regulatory review process, require us to perform additional studies or trials, increase our development costs, lead to changes in regulatory positions and interpretations, delay or prevent approval and commercialization of our product candidates or lead to significant post-approval limitations or restrictions. As we advance our product candidates, we will be required to consult with these regulatory agencies and comply with applicable requirements and guidelines. If we fail to do so, we may be required to delay or discontinue development of such product candidates. These additional processes may result in a review and approval process that is longer than we otherwise would have expected. Delays as a result of an increased or lengthier regulatory approval process or further restrictions on the development of our product candidates can be costly and could negatively impact our ability to complete clinical trials and commercialize our current and future product candidates in a timely manner, if at all.
The FDA and other regulatory agencies actively enforce the laws and regulations prohibiting the promotion of off-label uses.
If any of our product candidates are approved and we are found to have improperly promoted off-label uses of those products, we may become subject to significant liability. The FDA and other regulatory agencies strictly regulate the promotional claims that may be made about prescription products, if approved. In particular, while the FDA permits the dissemination of truthful and
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non-misleading information about an approved product, a manufacturer may not promote a product for uses that are not approved by the FDA or such other regulatory agencies as reflected in the product’s approved labeling. If we are found to have promoted such off-label uses, we may become subject to significant liability. The federal government has levied large civil and criminal fines against companies for alleged improper promotion of off-label use and has enjoined several companies from engaging in off-label promotion. The FDA has also requested that companies enter into consent decrees, corporate integrity agreements or permanent injunctions under which specified promotional conduct must be changed or curtailed. If we cannot successfully manage the promotion of our product candidates, if approved, we could become subject to significant liability, which would materially adversely affect our business and financial condition.
Inadequate funding for the FDA, the SEC and other government agencies, including from government shut downs, or other disruptions to these agencies’ operations, could hinder their ability to hire and retain key leadership and other personnel, prevent new products and services from being developed or commercialized in a timely manner or otherwise prevent those agencies from performing normal business functions on which the operation of our business may rely, which could negatively impact our business.
The ability of the FDA to review and approve new products can be affected by a variety of factors, including government budget and funding levels, ability to hire and retain key personnel and accept the payment of user fees, and statutory, regulatory and policy changes. Average review times at the agency have fluctuated in recent years as a result. Disruptions at the FDA and other agencies may also slow the time necessary for new product candidates to be reviewed and/or approved by necessary government agencies, which would adversely affect our business. In addition, government funding of the SEC and other government agencies on which our operations may rely, including those that fund research and development activities, is subject to the political process, which is inherently fluid and unpredictable.
Disruptions at the FDA and other agencies may also slow the time necessary for new product candidates to be reviewed and/or approved by necessary government agencies, which would adversely affect our business. For example, over the last several years the U.S. government has shut down several times and certain regulatory agencies, such as the FDA and the SEC, have had to furlough critical FDA, SEC and other government employees and stop critical activities. If a prolonged government shutdown occurs, it could significantly impact the ability of the FDA to timely review and process our regulatory submissions, which could have a material adverse effect on our business. Further, future government shutdowns could impact our ability to access the public markets and obtain necessary capital in order to properly capitalize and continue our operations.
Healthcare legislative reform measures may have a material adverse effect on our business and results of operations.
The U.S. and many foreign jurisdictions have enacted or proposed legislative and regulatory changes affecting the U.S. and global healthcare systems that could prevent or delay regulatory approval of our current or future product candidates or any future product candidates, restrict or regulate post-approval activities and affect our ability to profitably sell a product for which we obtain regulatory approval. Changes in regulations, statutes or the interpretation of existing regulations could impact our business in the future by requiring, for example: (i) changes to our manufacturing arrangements, (ii) additions or modifications to product labeling, (iii) the recall or discontinuation of our products or (iv) additional record-keeping requirements. If any such changes were to be imposed, they could adversely affect the operation of our business. In the U.S., there have been and continue to be, on-going legislative initiatives to contain healthcare costs. For example, in March 2010, the Patient Protection and Affordable Care Act, or the ACA, was passed, as amended by the Health Care and Education Reconciliation Act of 2010, which substantially changed the way healthcare is financed by both governmental and private insurers, and significantly impacted the U.S. pharmaceutical industry. The ACA, among other things, subjects biological products to potential competition by lower-cost biosimilars, addresses a new methodology by which rebates owed by manufacturers under the Medicaid Drug Rebate Program are calculated for drugs that are inhaled, infused, instilled, implanted or injected, increases the minimum Medicaid rebates owed by manufacturers under the Medicaid Drug Rebate Program, and extends the rebate program to individuals enrolled in Medicaid managed care organizations, establishes annual fees and taxes on manufacturers of certain branded prescription drugs, creates a new Medicare Part D coverage gap discount program, in which manufacturers must agree to offer 50% (increased to 70% pursuant to the Bipartisan Budget Act of 2018, effective as of 2019) point-of-sale discounts off negotiated prices of applicable brand drugs to eligible beneficiaries during their coverage gap period, as a condition for the manufacturer’s outpatient drugs to be covered under Medicare Part D, and provided incentives to programs that increase the federal government’s comparative effectiveness research. Since then, the ACA risk adjustment program payment parameters have been updated annually.
Since the ACA’s enactment, there have been numerous judicial, administrative, executive, and legislative challenges to certain aspects of the ACA and we expect that there will be additional challenges and amendments to the ACA in the future. On June 17, 2021, the U.S. Supreme Court dismissed the most recent judicial challenge to the ACA brought by several states without specifically ruling on the constitutionality of the ACA. Prior to the Supreme Court’s decision, President Biden issued an Executive Order to initiate a special enrollment period from February 15, 2021 through August 15, 2021 for purposes of obtaining health insurance coverage through the ACA marketplace. The Executive Order also instructed certain governmental agencies to review and reconsider their existing policies and rules that limit access to healthcare, including among others, reexamining Medicaid demonstration projects and waiver programs that include work requirements, and policies that create unnecessary barriers to
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obtaining access to health insurance coverage through Medicaid or the ACA. It is unclear how other healthcare reform measures of the Biden administrations or other efforts, if any, to challenge repeal or replace the ACA, will impact our business.
In addition, other legislative and regulatory changes have been proposed and adopted in the United States since the ACA was enacted:
There has been increasing legislative and enforcement interest in the U.S. with respect to specialty drug pricing practices. Specifically, there have been several recent U.S. Congressional inquiries and proposed federal and state legislation designed to, among other things, bring more transparency to drug pricing, reduce the cost of prescription drugs under Medicare, review the relationship between pricing and manufacturer patient programs, and reform government program reimbursement methodologies for drugs. In addition, President Biden has issued multiple executive orders that have sought to reduce prescription drug costs. In February 2023, HHS also issued a proposal in response to an October 2022 executive order from President Biden that includes a proposed prescription drug pricing model that will test whether targeted Medicare payment adjustments will sufficiently incentivize manufacturers to complete confirmatory trials for drugs approved through FDA’s accelerated approval pathway. Although a number of these and other proposed measures may require authorization through additional legislation to become effective, and the Biden administration may reverse or otherwise change these measures, both the Biden administration and Congress have indicated that they will continue to seek new legislative measures to control drug costs.
The Inflation Reduction Act of 2022, or IRA, includes several provisions that may impact our business to varying degrees including provisions that reduce the out-of-pocket spending cap for Medicare Part D beneficiaries from $7,050 to $2,000 starting in 2025, thereby effectively eliminating the coverage gap; imposes new manufacturer financial liability on certain drugs under Medicare Part D, allows the U.S. government to negotiate Medicare Part B and Part D price caps for certain high-cost drugs and biologics without generic or biosimilar competition; requires companies to pay rebates to Medicare for certain drug prices that increase faster than inflation; and delays until January 1, 2032 the implementation of the HHS rebate rule that would have limited the fees that pharmacy benefit managers can charge. Further, under the IRA, orphan drugs are exempted from the Medicare drug price negotiation program, if they have one orphan designation and the only approved indication is for that rare disease or condition. If a product receives multiple rare disease designations or has multiple approved indications, it may not qualify for the orphan drug exemption. The implementation of the IRA is currently subject to ongoing litigation challenging the constitutionality of the IRA’s Medicare drug price negotiation program. The effects of the IRA on our business and the healthcare industry in general is not yet known.
At the state level, individual states are increasingly aggressive in passing legislation and implementing regulations designed to control pharmaceutical and biological product pricing, including price or patient reimbursement constraints, discounts, restrictions on certain product access and marketing cost disclosure and transparency measures, and, in some cases, designed to encourage importation from other countries and bulk purchasing. In addition, regional health care authorities and individual hospitals are increasingly using bidding procedures to determine what pharmaceutical products and which suppliers will be included in their prescription drug and other health care programs. These measures could reduce the ultimate demand for our products, once approved, or put pressure on our product pricing.
We expect that additional state and federal healthcare reform measures will be adopted in the future, any of which could limit the amounts that federal and state governments will pay for healthcare products and services, which could result in reduced demand for our current or future product candidates or additional pricing pressures. In particular any policy changes through CMS as well as local state Medicaid programs could have a significant impact on our business.
Our revenue prospects could be affected by changes in healthcare spending and policy in the U.S. and abroad. We operate in a highly regulated industry and new laws, regulations or judicial decisions, or new interpretations of existing laws, regulations or decisions, related to healthcare availability, the method of delivery or payment for healthcare products and services could negatively impact our business, operations and financial condition.
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There have been, and likely will continue to be, legislative and regulatory proposals at the foreign, federal and state levels directed at broadening the availability of healthcare and containing or lowering the cost of healthcare. We cannot predict the initiatives that may be adopted in the future, including repeal, replacement or significant revisions to the ACA. The continuing efforts of the government, insurance companies, managed care organizations and other payors of healthcare services to contain or reduce costs of healthcare and/or impose price controls may adversely affect:
Any reduction in reimbursement from Medicare or other government programs may result in a similar reduction in payments from private payors, which may adversely affect our future profitability.
Our relationships with customers, healthcare providers, physicians and third-party payors will be subject to applicable anti-kickback, fraud and abuse and other healthcare laws and regulations, which could expose us to criminal sanctions, civil penalties, exclusion from government healthcare programs, contractual damages, reputational harm and diminished future profits and earnings.
Although we do not currently have any products on the market, once we begin commercializing our product candidates, we will be subject to additional healthcare statutory and regulatory requirements and enforcement by the federal government and the states and foreign governments in which we conduct our business. Healthcare providers, physicians and third-party payors will play a primary role in the recommendation and prescription of any product candidates for which we obtain regulatory approval. Our future arrangements with third-party payors and customers may expose us to broadly applicable fraud and abuse and other healthcare laws and regulations that may constrain the business or financial arrangements and relationships through which we market, sell and distribute our product candidates for which we obtain regulatory approval. Restrictions under applicable federal and state healthcare laws and regulations, include the following:
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We are also subject to state and foreign equivalents of each of the healthcare laws and regulations described above, among others, some of which may be broader in scope and may apply regardless of the payor. Many U.S. states have adopted laws similar to the federal Anti-Kickback Statute and False Claims Act, and may apply to our business practices, including, but not limited to, research, distribution, sales or marketing arrangements and claims involving healthcare items or services reimbursed by non-governmental payors, including private insurers. In addition, some states have passed laws that require pharmaceutical companies to comply with the April 2003 Office of Inspector General Compliance Program Guidance for Pharmaceutical Manufacturers and/or the Pharmaceutical Research and Manufacturers of America’s Code on Interactions with Healthcare Professionals. Several states also impose other marketing restrictions or require pharmaceutical companies to make marketing or price disclosures to the state and require the registration of pharmaceutical sales representatives. State and foreign laws, including for example the European Union General Data Protection Regulation, also govern the privacy and security of health information in some circumstances, many of which differ from each other in significant ways and often are not preempted by HIPAA, thus complicating compliance efforts. There are ambiguities as to what is required to comply with these state requirements and if we fail to comply with an applicable state law requirement we could be subject to penalties. Finally, there are state and foreign laws governing the privacy and security of health information, many of which differ from each other in significant ways and often are not preempted by HIPAA, thus complicating compliance efforts.
In the U.S., to help patients afford our approved product, we may utilize programs to assist them, including patient assistance programs and co-pay coupon programs for eligible patients. Government enforcement agencies have shown increased interest in pharmaceutical companies’ product and patient assistance programs, including reimbursement support services, and a number of investigations into these programs have resulted in significant civil and criminal settlements. In addition, at least one insurer has directed its network pharmacies to no longer accept co-pay coupons for certain specialty drugs the insurer identified. Our co-pay coupon programs could become the target of similar insurer actions. In addition, in November 2013, the CMS issued guidance to the issuers of qualified health plans sold through the ACA’s marketplaces encouraging such plans to reject patient cost-sharing support from third parties and indicating that the CMS intends to monitor the provision of such support and may take regulatory action to limit it in the future. The CMS subsequently issued a rule requiring individual market qualified health plans to accept third-party premium and cost-sharing payments from certain government-related entities. In September 2014, the OIG of the HHS issued a Special Advisory Bulletin warning manufacturers that they may be subject to sanctions under the federal anti-kickback statute and/or civil monetary penalty laws if they do not take appropriate steps to exclude Part D beneficiaries from using co-pay coupons. Accordingly, companies exclude these Part D beneficiaries from using co-pay coupons. It is possible that changes in insurer policies regarding co-pay coupons and/or the introduction and enactment of new legislation or regulatory action could restrict or otherwise negatively affect these patient support programs, which could result in fewer patients using affected products, and therefore could have a material adverse effect on our sales, business, and financial condition.
Third party patient assistance programs that receive financial support from companies have become the subject of enhanced government and regulatory scrutiny. The OIG has established guidelines that suggest that it is lawful for pharmaceutical manufacturers to make donations to charitable organizations who provide co-pay assistance to Medicare patients, provided that such organizations, among other things, are bona fide charities, are entirely independent of and not controlled by the manufacturer, provide aid to applicants on a first-come basis according to consistent financial criteria and do not link aid to use of a donor’s product. However, donations to patient assistance programs have received some negative publicity and have been the subject of multiple government enforcement actions, related to allegations regarding their use to promote branded pharmaceutical products over other less costly alternatives. Specifically, in recent years, there have been multiple settlements resulting out of government claims challenging the legality of their patient assistance programs under a variety of federal and state laws. It is possible that we may make grants to independent charitable foundations that help financially needy patients with their premium, co-pay, and co-insurance obligations. If we choose to do so, and if we or our vendors or donation recipients are deemed to fail to comply with relevant laws, regulations or evolving government guidance in the operation of these programs, we could be subject to damages,
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fines, penalties, or other criminal, civil, or administrative sanctions or enforcement actions. We cannot ensure that our compliance controls, policies, and procedures will be sufficient to protect against acts of our employees, business partners, or vendors that may violate the laws or regulations of the jurisdictions in which we operate. Regardless of whether we have complied with the law, a government investigation could impact our business practices, harm our reputation, divert the attention of management, increase our expenses, and reduce the availability of foundation support for our patients who need assistance.
The scope and enforcement of each of these laws is uncertain and subject to rapid change in the current environment of healthcare reform, especially in light of the lack of applicable precedent and regulations. Federal and state enforcement bodies have recently increased their scrutiny of interactions between healthcare companies and healthcare providers, which has led to a number of investigations, prosecutions, convictions and settlements in the healthcare industry.
Ensuring that our future business arrangements with third parties comply with applicable healthcare laws and regulations could involve substantial costs. It is possible that governmental authorities will conclude that our business practices do not comply with current or future statutes, regulations or case law involving applicable fraud and abuse or other healthcare laws and regulations. If our operations, including anticipated activities to be conducted by our sales team, were to be found to be in violation of any of these laws or any other governmental regulations that may apply to us, we may be subject to significant civil, criminal and administrative penalties, damages, fines, disgorgement, imprisonment, the exclusion from participation in federal and state government funded healthcare programs, such as Medicare and Medicaid, reputational harm, and the curtailment or restructuring of our operations. It may also subject us to additional reporting obligations and oversight, if we become subject to a corporate integrity agreement, deferred prosecution agreement, or other agreement to resolve allegations of non-compliance with these laws. If any of the physicians or other providers or entities with whom we expect to do business is found not to be in compliance with applicable laws, they may be subject to similar criminal, civil or administrative sanctions, including exclusions from government funded healthcare programs.
If we fail to comply with environmental, health and safety laws and regulations, we could become subject to fines or penalties or incur costs that could have a material adverse effect on the success of our business.
We are subject to numerous environmental, health and safety laws and regulations, including those governing laboratory procedures and the handling, use, storage, treatment and disposal of hazardous materials and wastes. Our operations involve the use of hazardous and flammable materials, including chemicals and biological and radioactive materials. Our operations also produce hazardous waste products. We generally contract with third parties for the disposal of these materials and wastes. We cannot eliminate the risk of contamination or injury from these materials. In the event of contamination or injury resulting from our use of hazardous materials, we could be held liable for any resulting damages, and any liability could exceed our resources. We also could incur significant costs associated with civil or criminal fines and penalties.
Although we maintain workers’ compensation insurance to cover us for costs and expenses we may incur due to injuries to our employees resulting from the use of hazardous materials, this insurance may not provide adequate coverage against potential liabilities. We do not maintain insurance for environmental liability or toxic tort claims that may be asserted against us in connection with our storage or disposal of biological, hazardous or radioactive materials. In addition, we may incur substantial costs in order to comply with current or future environmental, health and safety laws and regulations. Current or future environmental laws and regulations may impair our research, development and production efforts, which could harm our business, prospects, financial condition or results of operations.
Compliance with U.S. and global privacy and data security requirements could result in additional costs and liabilities to us or inhibit our ability to collect and process data globally, and the failure to comply with such requirements could expose us to criminal sanctions, civil penalties, contractual damages, reputational harm, administrative burdens and diminished profits and future earnings.
The regulatory framework for the collection, use, safeguarding, sharing, transfer and other processing of information worldwide is rapidly evolving and is likely to remain uncertain for the foreseeable future. We possess and process sensitive customer information. Other federal and state laws that restrict the use and protect the privacy and security of personally identifiable information, in many cases, are not preempted by HIPAA and may be subject to varying interpretations by the courts and government agencies. These varying interpretations can create complex compliance issues for us and our partners and potentially expose us to additional expense, adverse publicity and liability, any of which could adversely affect our business. There is ongoing concern from privacy advocates, regulators and others regarding data privacy and security issues, and the number of jurisdictions with data privacy and security laws has been increasing. Also, there are ongoing public policy discussions regarding whether the standards for de-identification, anonymization or pseudonymization of health information are sufficient, and the risk of re-identification sufficiently small, to adequately protect patient privacy. We expect that there will continue to be new proposed and amended laws, regulations and industry standards concerning privacy, data protection and information security in the United States, such as the California Consumer Privacy Act, or CCPA, as amended by the California Privacy Rights Act, or CPRA, creates specific obligations with respect to processing and storing personal information, and the CPRA amendments created a new state agency that is vested with authority to implement and enforce the CCPA.
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Laws similar to the CCPA have been passed in Montana, Texas, Iowa, Indiana, Tennessee, Connecticut, Colorado, Utah and Virginia. These laws are substantially similar in scope and contain many of the same requirements and exceptions as the CCPA, including a general exemption for clinical trial data and limited obligations for entities regulated by HIPAA. However, we cannot yet determine the full impact these laws or other such future laws, regulations and standards may have on our current or future business. Any of these laws may broaden their scope in the future, and similar laws have been proposed on both a federal level and in more than half of the states in the U.S. A number of other states have proposed new privacy laws, some of which are similar to the above discussed recently passed laws. Such proposed legislation, if enacted, may add additional complexity, variation in requirements, restrictions and potential legal risk, require additional investment of resources in compliance programs, impact strategies and the availability of previously useful data and could result in increased compliance costs and/or changes in business practices and policies. The existence of comprehensive privacy laws in different states in the country would make our compliance obligations more complex and costly and may increase the likelihood that we may be subject to enforcement actions or otherwise incur liability for noncompliance.
The collection, use, storage, disclosure, transfer, or other processing of personal data regarding individuals in the EEA, including personal health data, is subject to the EU General Data Protection Regulation 2016/679, or GDPR, which became effective on May 25, 2018. The GDPR is wide-ranging in scope and imposes numerous requirements on companies that process personal data, including requirements relating to processing health and other sensitive data, obtaining consent of the individuals to whom the personal data relates, providing information to individuals regarding data processing activities, implementing safeguards to protect the security and confidentiality of personal data, providing notification of data breaches, and taking certain measures when engaging third-party processors. The GDPR permits data protection authorities to impose large penalties for violations of the GDPR, including potential fines of up to €20 million or 4% of annual global revenues, whichever is greater. The GDPR also confers a private right of action on data subjects and consumer associations to lodge complaints with supervisory authorities, seek judicial remedies, and obtain compensation for damages resulting from violations of the GDPR. The GDPR increased our responsibility and liability in relation to personal data that we process where such processing is subject to the GDPR, and we may be required to put in place additional mechanisms to ensure compliance with the GDPR, including as implemented by individual countries. Compliance with the GDPR will be a rigorous and time-intensive process that may increase our cost of doing business or require us to change our business practices, and despite those efforts, there is a risk that we may be subject to fines and penalties, litigation, and reputational harm in connection with our European activities.
The GDPR provides that EEA Member States may make their own further laws and regulations in relation to the processing of genetic, biometric or health data, which could result in differences between Member States, limit our ability to use and share personal data or could cause our costs to increase, and harm our business and financial condition.
The GDPR also imposes strict rules on the transfer of personal data to countries outside the EEA, including the United States. We are subject to evolving and strict rules on the transfer of personal data out of the EEA to third countries such as the United States. Unless the destination country is an adequate country (as recognized by the European Commission), we will be required to incorporate a GDPR transfer mechanism (such as the European Commission approved standard contractual clauses, or SCCs) into our agreements with third parties to govern transfers of personal data outside the EEA. The new SCCs may also impact our business as companies based in the EEA may be reluctant to utilize the new clauses to legitimize transfers of personal data to third countries given the burdensome requirements of transfer impact assessments and the substantial obligations that the new SCCs impose upon exporters.
In addition, further to the UK’s exit from the European Union, or EU, on January 31, 2020 the UK’s European Union (Withdrawal) Act 2018 incorporated the GDPR into UK law, referred to as the UK GDPR. The UK GDPR and the UK Data Protection Act 2018 set out the UK’s data protection regime, which is independent from but currently still aligned to the EU’s data protection regime. Non-compliance with the UK GDPR may result in monetary penalties of up to £17.5 million or 4% of worldwide revenue, whichever is higher. Although the UK is regarded as a third country under the EU’s GDPR, the European Commission has now issued a decision recognizing the UK as providing adequate protection under the EU GDPR and, therefore, transfers of personal data originating in the EU to the UK remain unrestricted. Like the EU GDPR, the UK GDPR restricts personal data transfers outside the UK to countries not regarded by the UK as providing adequate protection. It is not subject to the new forms of SCCs but has issued its own transfer mechanism – the UK international data transfer agreement – which, like the SCCs, requires exporters to carry out a transfer impact assessment. The UK government has confirmed that personal data transfers from the UK to the EEA remain free flowing. The UK Government has also now introduced a Data Protection and Digital Information Bill, or the UK Bill, into the UK legislative process with the intention for this bill to reform the UK’s data protection regime following Brexit. If passed, the final version of the UK Bill may have the effect of further altering the similarities between the UK and EU data protection regime and threaten the UK Adequacy Decision from the EU Commission. This may lead to additional compliance costs and could increase our overall risk.
If we or third-party CDMOs, CROs or other contractors or consultants fail to comply with U.S. and international data protection laws and regulations, it could result in government enforcement actions (which could include civil or criminal penalties), private litigation, and/or adverse publicity and could negatively affect our operating results and business. Moreover, clinical trial subjects about whom we or our potential collaborators obtain information, as well as the providers who share this information with
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us, may contractually limit our ability to use and disclose the information. Claims that we have violated individuals’ privacy rights, failed to comply with data protection laws, or breached our contractual obligations, even if we are not found liable, could be expensive and time-consuming to defend and could result in adverse publicity that could harm our business.
Laws and regulations governing any international operations we may have in the future may preclude us from developing, manufacturing and selling certain products outside of the United States and require us to develop and implement costly compliance programs.
If we expand our operations outside of the United States, we must dedicate additional resources to comply with numerous laws and regulations in each jurisdiction in which we plan to operate. The Foreign Corrupt Practices Act, or FCPA, prohibits any U.S. individual or business from paying, offering, authorizing payment or offering of anything of value, directly or indirectly, to any foreign official, political party or candidate for the purpose of influencing any act or decision of the foreign entity in order to assist the individual or business in obtaining or retaining business. The FCPA also obligates companies whose securities are listed in the United States to comply with certain accounting provisions requiring the company to maintain books and records that accurately and fairly reflect all transactions of the corporation, including international subsidiaries, and to devise and maintain an adequate system of internal accounting controls for international operations.
Compliance with the FCPA is expensive and difficult, particularly in countries in which corruption is a recognized problem. In addition, the FCPA presents particular challenges in the pharmaceutical industry, because, in many countries, hospitals are operated by the government, and doctors and other hospital employees are considered foreign officials. Certain payments to hospitals in connection with clinical trials and other work have been deemed to be improper payments to government officials and have led to FCPA enforcement actions.
Various laws, regulations and executive orders also restrict the use and dissemination outside of the United States, or the sharing with certain non-U.S. nationals, of information classified for national security purposes, as well as certain products and technical data relating to those products. If we expand our presence outside of the United States, it will require us to dedicate additional resources to comply with these laws, and these laws may preclude us from developing, manufacturing, or selling certain products and product candidates outside of the United States, which could limit our growth potential and increase our development costs.
The failure to comply with laws governing international business practices may result in substantial civil and criminal penalties and suspension or debarment from government contracting. The SEC also may suspend or bar issuers from trading securities on U.S. exchanges for violations of the FCPA’s accounting provisions.
Risks Relating to Employee Matters and Managing Growth
Our future success depends on our ability to retain key executives and experienced scientists and to attract, retain, and motivate qualified personnel.
We are highly dependent on many of our key employees and members of our executive management team as well as the other principal members of our management, scientific and clinical teams. Although we have entered into employment letter agreements with certain of our executive officers, each of them may terminate their employment with us at any time. We do not maintain “key person” insurance for any of our executives or other employees. In addition, we rely on consultants and directors, whetheradvisors, including scientific and clinical advisors, to assist us in formulating our research and development and commercialization strategy. Our consultants and advisors may be employed by employers other than us and may have commitments under consulting or notadvisory contracts with other entities that may limit their availability to us. If we are unable to continue to attract and retain high quality personnel, our ability to pursue our growth strategy will be limited.
Recruiting and retaining qualified scientific, clinical, manufacturing and sales and marketing personnel will also be critical to our success. The loss of the services of our executive officers or other key employees, including temporary loss due to illness, could impede the achievement of our research, development and commercialization objectives and seriously harm our ability to successfully implement our business strategy. Furthermore, replacing executive officers and key employees may be difficult and may take an extended period of time because of the limited number of individuals in our industry with the breadth of skills and experience required to successfully develop, gain regulatory approval of and commercialize products. Competition to hire from this limited pool is intense, and we may be unable to hire, train, retain or motivate these key personnel on acceptable terms given the competition among numerous pharmaceutical and biotechnology companies for similar personnel. We also experience competition for the hiring of scientific and clinical personnel from universities and research institutions. Failure to succeed in clinical trials may make it more challenging to recruit and retain qualified scientific personnel.
In particular, we have experienced a very competitive hiring environment in the greater Boston area of Massachusetts, where we are headquartered. Many of the other pharmaceutical companies that we compete against for qualified personnel have greater financial and other resources, different risk profiles and a longer history in the industry than we do. They also may provide more diverse opportunities and better chances for career advancement. Some of these characteristics may be more appealing to
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high-quality candidates than what we have to offer. If we are unable to continue to attract and retain high-quality personnel, the rate and success with which we can discover and develop product candidates and our business will be limited.
We may be unable to adequately protect our information systems from cyberattacks, which could result in the disclosure of confidential or proprietary information, including personal data, damage our reputation, and subject us to significant financial and legal exposure.
We rely on information technology systems that we or our third-party providers operate to process, transmit and store electronic information in our day-to-day operations. In connection with our product discovery efforts, we may collect and use a variety of personal data, such as names, mailing addresses, email addresses, phone numbers and clinical trial information. A successful cyberattack could result in the theft or destruction of intellectual property, data, or other misappropriation of assets, or otherwise compromise our confidential or proprietary information and disrupt our operations. Cyberattacks are increasing in their frequency, sophistication and intensity, and have become increasingly difficult to detect. Cyberattacks could include wrongful conduct by hostile foreign governments, industrial espionage, wire fraud and other forms of cyber fraud, the deployment of harmful malware, denial-of-service, social engineering fraud or other means to threaten data security, confidentiality, integrity and availability. A successful cyberattack could cause serious negative consequences for us, including, without limitation, the disruption of operations, the misappropriation of confidential business information, including financial information, trade secrets, financial loss and the disclosure of corporate strategic plans. Although we devote resources to protect our information systems, we realize that cyberattacks are a threat, and there iscan be no assurance that our efforts will prevent information security breaches that would result in business, legal, financial or reputational harm to it, or would have a covered terminationmaterial adverse effect on our results of operations and financial condition. Any failure to prevent or mitigate security breaches or improper access to, use of, or disclosure of our clinical data or patients’ personal data could result in significant liability under state (e.g., state breach notification laws), federal (e.g., HIPAA, as amended by HITECH), and international law (e.g., the GDPR) and may cause a material adverse impact to our reputation, affect our ability to conduct new studies and potentially disrupt our business.
We rely on our third-party providers to implement effective security measures and identify and correct for any such failures, deficiencies or breaches. We also rely on our employees and consultants to safeguard their security credentials and follow our policies and procedures regarding use and access of computers and other devices that may contain our sensitive information. If we or our third-party providers fail to maintain or protect our information technology systems and data integrity effectively or fail to anticipate, plan for or manage significant disruptions to our information technology systems, we or our third-party providers could have difficulty preventing, detecting and controlling such cyber-attacks and any such attacks could result in the losses described above as well as disputes with physicians, patients and our partners, regulatory sanctions or penalties, increases in operating expenses, expenses or lost revenue or other adverse consequences, any of which could have a material adverse effect on our business, results of operations, financial condition, prospects and cash flows. Any failure by such third parties to prevent or mitigate security breaches or improper access to or disclosure of such officer's employment. information could have similarly adverse consequences for us. If we are unable to prevent or mitigate the impact of such security or data privacy breaches, we could be exposed to litigation and governmental investigations, which could lead to a potential disruption to our business. By way of example, the CCPA, which was modified by the CPRA, creates individual privacy rights for California consumers and increases the privacy and security obligations of entities handling certain personal data. The CCPA provides for civil penalties of up to $7,500 per violation, as well as a private right of action for data breaches. The CCPA may increase our compliance costs and potential liability, and many similar laws have been proposed at the federal level and in other states.
We expect to expand our development and regulatory capabilities and potentially implement sales, marketing and distribution capabilities, and as a result, we may encounter difficulties in managing our growth, which could disrupt our operations.
As of July 31, 2023, we had 55 full-time employees and no part-time employees. We expect to experience significant growth in the number of our employees and the scope of our operations, particularly as we function as a public company and in the areas of product development, regulatory affairs and, if any of our product candidates receives regulatory approval, sales, marketing and distribution. To manage our anticipated future growth, we must continue to implement and improve our managerial, operational and financial systems, expand our facilities and continue to recruit and train additional qualified personnel. Due to our limited financial resources, we may not be able to effectively manage the expansion of our operations or recruit and train additional qualified personnel. The expansion of our operations may lead to significant costs and may divert our management and business development resources. Any inability to manage growth could delay the execution of our business plans or disrupt our operations.
We may acquire additional businesses or products, form strategic alliances or create joint ventures with third parties that we believe will complement or augment our existing business. If we acquire businesses with promising markets or technologies, we may not be able to realize the benefit of acquiring such businesses if we are unable to successfully integrate them with our existing operations and company culture. We may encounter numerous difficulties in developing, manufacturing and marketing any new products resulting from a strategic alliance or acquisition that delay or prevent us from realizing their expected benefits or enhancing our business. We cannot assure you that, following any such acquisition, we will achieve the expected synergies to justify the transaction.
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General Risks
Adverse developments affecting the financial services industry, such as actual events or concerns involving liquidity, defaults, or non-performance by financial institutions or transactional counterparties, could adversely affect the Company’s current and projected business operations and its financial condition and results of operations.
Events involving limited liquidity, defaults, non-performance or other adverse developments that affect financial institutions, transactional counterparties or other companies in the financial services industry or the financial services industry generally, or concerns or rumors about any events of these kinds or other similar risks, have in the past and may in the future lead to market-wide liquidity problems. For example, on March 10, 2023, Silicon Valley Bank, or SVB, was closed by the California Department of Financial Protection and Innovation, which appointed the Federal Deposit Insurance Corporation, or FDIC, as receiver. Similarly, on March 12, 2023, Signature Bank and Silvergate Capital Corp. were each placed into receivership. Although a statement by the Department of the Treasury, the Federal Reserve and the FDIC indicated that all depositors of SVB (such as our company) would have access to all of their money after only one business day of closure, including funds held in uninsured deposit accounts, borrowers under credit agreements, letters of credit and certain other financial instruments with SVB, Signature Bank or any other financial institution that is placed into receivership by the FDIC may be unable to access undrawn amounts thereunder. Subsequent to these events, additional financial institutions have experienced similar failures and have been placed into receivership. It is possible that other banks will face similar difficulty in the future.
Although we assess our banking relationships as we believe necessary or appropriate, our access to funding sources and other credit arrangements in amounts adequate to finance or capitalize our current and projected future business operations could be significantly impaired by factors that affect the Company, the financial institutions with which the Company has credit agreements or arrangements directly, or the financial services industry or economy in general. These factors could include, among others, events such as liquidity constraints or failures, the ability to perform obligations under various types of financial, credit or liquidity agreements or arrangements, disruptions or instability in the financial services industry or financial markets, or concerns or negative expectations about the prospects for companies in the financial services industry. These factors could involve financial institutions or financial services industry companies with which the Company has financial or business relationships, but could also include factors involving financial markets or the financial services industry generally.
In addition, investor concerns regarding the U.S. or international financial systems could result in less favorable commercial financing terms, including higher interest rates or costs and tighter financial and operating covenants, or systemic limitations on access to credit and liquidity sources, thereby making it more difficult for us to acquire financing on acceptable terms or at all. Any decline in available funding or access to our cash and liquidity resources could, among other risks, adversely impact our ability to meet our operating expenses, financial obligations or fulfill our other obligations, result in breaches of our financial and/or contractual obligations or result in violations of federal or state wage and hour laws. Any of these impacts, or any other impacts resulting from the factors described above or other related or similar factors not described above, could have material adverse impacts on our liquidity and our current and/or projected business operations and financial condition and results of operations.
Changes in tax law may adversely affect us or our investors.
The rules dealing with U.S. federal, state and local income taxation are constantly under review by persons involved in the legislative process and by the Internal Revenue Service, or IRS, and the U.S. Treasury Department. Changes to tax laws (which changes may have retroactive application) could adversely affect us or holders of our common stock. In recent years, many such changes have been made and changes are likely to continue to occur in the future. It cannot be predicted whether, when, in what form or with what effective dates tax laws, regulations and rulings may be enacted, promulgated or issued, which could result in an increase in our or our stockholders’ tax liability or require changes in the manner in which we operate in order to minimize or mitigate any adverse effects of changes in tax law. Prospective investors should consult their tax advisors regarding the potential consequences of changes in tax law on our business and on the ownership and disposition of our common stock.
Our future taxable income may be subject to certain limitations.
As of Gemini’sDecember 31, 2022, we had federal and state net operating loss carryforwards of $69.3 million and $67.7 million, respectively, which begin to expire in various amounts in 2037. As of December 31, 2022, we also had federal and state research and development tax credit carryforwards of $2.7 million and $1.1 million, respectively, which begin to expire in 2032. These net operating loss and tax credit carryforwards could expire unused and be unavailable to offset future income tax liabilities. Under current directorslaw, unused U.S. federal and executive officerscertain state net operating losses generated for tax years beginning after December 31, 2017 are not subject to expiration and may be carried forward indefinitely. Such U.S. federal net operating losses generally may not be carried back to prior taxable years, except that, net operating losses generated in 2019, 2020 and 2021 may be carried back to each of the five tax years preceding the tax years of such losses. For taxable years beginning after December 31, 2020, the deductibility of U.S. federal net operating losses generated for tax years beginning after December 31, 2017 is limited to 80% of our taxable income in any future taxable year. In addition, in general, under Sections 382 and 383 of the Internal Revenue Code of 1986, as amended, or the Code, a corporation that undergoes an “ownership change” is subject to limitations on its ability to utilize its pre-change net operating losses or tax credits, or NOLs or credits, to offset future taxable income or taxes. For these purposes, an
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ownership change generally occurs when one or more stockholders or groups of stockholders who each owns at least 5% of a corporation’s stock increase their aggregate stock ownership by more than 50 percentage points over their lowest ownership percentage within a specified testing period. Our existing NOLs or credits may be subject to limitations arising from previous ownership changes, and if we undergo an ownership change after the merger, our ability to utilize NOLs or credits could be further limited by Sections 382 and 383 of the Code. In addition, future changes in our stock ownership, many of which are outside of our control, could result in an ownership change under Sections 382 and 383 of the Code. Our NOLs or credits may also be impaired under state law. Accordingly, we may not be able to utilize a material portion of our NOLs or credits.
We are subject to certain U.S. and foreign anti-corruption, anti-money laundering, export control, sanctions, and other trade laws and regulations. We can face serious consequences for violations.
Among other matters, U.S. and foreign anti-corruption, anti-money laundering, export control, sanctions, and other trade laws and regulations, which are collectively referred to as Trade Laws, prohibit companies and their employees, agents, CROs, legal counsel, accountants, consultants, contractors, and other partners from authorizing, promising, offering, providing, soliciting, or receiving directly or indirectly, corrupt or improper payments or anything else of value to or from recipients in the public or private sector. Violations of Trade Laws can result in substantial criminal fines and civil penalties, imprisonment, the loss of trade privileges, debarment, tax reassessments, breach of contract and fraud litigation, reputational harm, and other consequences. We have direct or indirect interactions with officials and employees of government agencies or government-affiliated hospitals, universities, and other organizations. We also expect our non-U.S. activities to increase in time. We currently engage, and expect to continue to engage, third parties for clinical trials and/or to obtain necessary permits, licenses, patent registrations, and other regulatory approvals and we can be held liable for the corrupt or other illegal activities of our personnel, agents, or partners, even if we do not explicitly authorize or have prior knowledge of such activities.
Unfavorable global economic conditions could adversely affect our business, financial condition or results of operations.
Our results of operations could be adversely affected by general conditions in the global economy and in the global financial markets. For example, in 2008, the global financial crisis caused extreme volatility and disruptions in the capital and credit markets and the COVID-19 pandemic has caused significant volatility and uncertainty in U.S. and international markets. See “Risks Related to the Discovery and Development of Our Product Candidates-The COVID-19 pandemic, or a similar pandemic, epidemic, or outbreak of an infectious disease may materially and adversely affect our business and financial results and could cause a disruption to the development of our product candidates.” Interest rates in the U.S. have recently increased to levels not seen in decades. In addition, the impact of geopolitical tension, such as a deterioration in the bilateral relationship between the United States and China or an escalation in conflict between Russia and Ukraine, including any resulting sanctions, export controls or other restrictive actions, also could lead to disruption, instability and volatility in the global markets. A severe or prolonged economic downturn could result in a variety of risks to our business, including, weakened demand for our product candidates and our ability to raise additional capital when needed on acceptable terms, if at all. A weak or declining economy could also strain our suppliers, possibly resulting in supply disruption, or cause our customers to delay making payments for our products. Any of the foregoing could harm our business and we cannot anticipate all of the ways in which the current economic climate and financial market conditions could adversely impact our business.
Our employees, principal investigators, CROs and consultants may engage in misconduct or other improper activities, including non-compliance with regulatory standards and requirements or insider trading.
We are exposed to the risk that our employees, principal investigators, CROs and consultants may engage in fraudulent conduct or other illegal activity. Misconduct by these parties could include intentional, reckless and/or negligent conduct or disclosure of unauthorized activities to us that violate the regulations of the FDA and other regulatory authorities, including those laws requiring the reporting of true, complete and accurate information to such authorities; healthcare fraud and abuse laws and regulations in the United States and abroad; or laws that require the reporting of financial information or data accurately. In particular, sales, marketing and business arrangements in the healthcare industry are subject to extensive laws and regulations intended to prevent fraud, misconduct, kickbacks, self-dealing and other abusive practices. These laws and regulations may restrict or prohibit a wide range of pricing, discounting, marketing and promotion, sales commission, customer incentive programs and other business arrangements. Other activities subject to these laws include the improper use of information obtained in the course of clinical trials or creating fraudulent data in our preclinical studies or clinical trials, which could result in regulatory sanctions and cause serious harm to our reputation. We have adopted a code of conduct applicable to all of our employees, but it is not always possible to identify and deter misconduct by employees and other third parties, and the precautions we take to detect and prevent this activity may not be effective in controlling unknown or unmanaged risks or losses or in protecting us from governmental investigations or other actions or lawsuits stemming from a failure to comply with these laws or regulations. Additionally, we are subject to the risk that a person could allege such fraud or other misconduct, even if none occurred. If any such actions are instituted against us, and we are not successful in defending ourself or asserting our rights, those actions could have a significant impact on our business, including the imposition of civil, criminal and administrative penalties, damages, monetary fines, possible exclusion from participation in Medicare, Medicaid and other federal healthcare programs, contractual damages, reputational harm,
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diminished profits and future earnings, and curtailment of our operations, any of which could adversely affect our ability to operate our business and our results of operations.
The market price of our common stock is expected to become directors of the surviving company upon the closing of the Disc Merger, and all of Gemini’s directors and executive officers are entitled to certain indemnification and liability insurance coverage pursuant to the terms of the Disc Merger Agreement. These interests, among others, may influence the officers and directors of the Company and cause them to view the Disc Merger differently from how the Company’s stockholders generally may view it.be volatile.
The market price of our common stock following the Disc Merger may decline as a resultmerger could be subject to significant fluctuations. Some of the Disc Merger.
Thefactors that may cause the market price of our common stock may decline as a result of the Disc Merger for a number of reasons, including if:to fluctuate include:
The Company’s equityholders willMoreover, the stock markets in general have experienced substantial volatility that has often been unrelated to the operating performance of individual companies. These broad market fluctuations may also adversely affect the trading price of our common stock. In addition, a reduced ownershiprecession, depression or other sustained adverse market event resulting from the spread of COVID-19 or otherwise could materially and voting interest in,adversely affect our business and will exercise less influence over the managementvalue of our common stock. In the Companypast, following the closingperiods of the Disc Merger as compared to their current ownership and voting interestvolatility in the Company.market price of a company’s securities, stockholders have often instituted class action securities litigation against such companies. Furthermore, market volatility may lead to increased shareholder activism if we experience a market valuation that activists believe is not reflective of our intrinsic value. Activist campaigns that contest or conflict with our strategic direction or seek changes in the composition of our board of directors could have an adverse effect on our operating results and financial condition.
AfterWe will incur additional costs and increased demands upon management as a result of complying with the completionlaws and regulations affecting public companies.
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We will incur significant legal, accounting and other expenses as a public company that we did not incur as a private company, including costs associated with public company reporting obligations under the Securities Exchange Act of 1934, as amended, or the Disc Merger, the current securityholdersExchange Act. Our management team consists of the Company will own a smaller percentage of the combined company than their ownership in the Companyour executive officers prior to the Disc Merger. Immediately aftermerger, some of whom have not previously managed and operated a public company. These executive officers and other personnel will need to devote substantial time to gaining expertise related to public company reporting requirements and compliance with applicable laws and regulations to ensure that we comply with all of these requirements. Any changes we make to comply with these obligations may not be sufficient to allow us to satisfy our obligations as a public company on a timely basis, or at all. These reporting requirements, rules and regulations, coupled with the Disc Merger,increase in potential litigation exposure associated with being a public company, could also make it is currently estimated that pre-merger Gemini’s equityholdersmore difficult for us to attract and retain qualified persons to serve on the board of directors or on board committees or to serve as executive officers, or to obtain certain types of insurance, including directors’ and officers’ insurance, on acceptable terms.
Once we are no longer an emerging growth company, a smaller reporting company or otherwise no longer qualify for applicable exemptions, we will own approximately 28% of the common stock of the combined company, and pre-merger Disc equityholders will own approximately 72% of the common stock of the combined company, in each case, before giving effect to the concurrent financing andbe subject to certain assumptions. These estimates are basedadditional laws and regulations affecting public companies that will increase our costs and the demands on the anticipated exchange ratiomanagement and could harm our operating results.
We are subject to adjustmentthe reporting requirements of the Exchange Act, which requires, among other things, that we file with the SEC, annual, quarterly and current reports with respect to our business and financial condition as providedwell as other disclosure and corporate governance requirements. However, as an emerging growth company we may take advantage of exemptions from various requirements such as an exemption from the requirement to have our independent auditors attest to our internal control over financial reporting under Section 404 of the Sarbanes-Oxley Act of 2002 as well as an exemption from the “say on pay” voting requirements pursuant to the Dodd-Frank Wall Street Reform and Consumer Protection Act of 2010. After we no longer qualify as an emerging growth company, we may still qualify as a “smaller reporting company” which may allow us to take advantage of some of the same exemptions from disclosure requirements including not being required to comply with the auditor attestation requirements of Section 404 of the Sarbanes-Oxley Act and reduced disclosure obligations regarding executive compensation in our periodic reports and proxy statements. Even after we no longer qualify as an emerging growth company, we expect to still qualify as a “smaller reporting company,” as such term is defined in Rule 12b-2 under the Disc Merger Agreement.Exchange Act, in at least the near term, which will allow us to take advantage of many of the same exemptions from disclosure requirements, including not being required to comply with the auditor attestation requirements of Section 404 of the Sarbanes-Oxley Act and reduced disclosure obligations regarding executive compensation in this Quarterly Report on Form 10-Q and in our periodic reports and proxy statements. Once we are no longer an emerging growth company, a smaller reporting company or otherwise qualify for these exemptions, we will be required to comply with these additional legal and regulatory requirements applicable to public companies and will incur significant legal, accounting and other expenses to do so. If we are not able to comply with the requirements in a timely manner or at all, our financial condition or the market price of our common stock may be harmed. For example, if we or our independent auditor identifies deficiencies in our internal control over financial reporting that are deemed to be material weaknesses we could face additional costs to remedy those deficiencies, the market price of our stock could decline or we could be subject to sanctions or investigations by the SEC or other regulatory authorities, which would require additional financial and management resources.
In addition,Provisions in our charter documents and under Delaware law could make an acquisition of us more difficult and may discourage any takeover attempts our stockholders may consider favorable, and may lead to entrenchment of management.
Provisions of our amended and restated certificate of incorporation and amended and restated bylaws could delay or prevent changes in control or changes in management without the nine-memberconsent of the board of directors. These provisions will include the following:
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In addition, these provisions would apply even if we were to receive an offer that some stockholders may consider beneficial.
We will also be subject to the anti-takeover provisions contained in Section 203 of the DGCL, or Section 203. Under Section 203, a corporation may not, in general, engage in a business combination with prior affiliationsany holder of 15% or more of its capital stock unless the holder has held the stock for three years or, among other exceptions, the board of directors has approved the transaction.
Our certificate of incorporation and bylaws provide that the Court of Chancery of the State of Delaware is the exclusive forum for substantially all disputes between us and our stockholders, which could limit our stockholders’ ability to obtain a favorable judicial forum for disputes with Gemini. Consequently, securityholdersus or our directors, officers or other employees.
Our certificate of Geminiincorporation and bylaws provide that the Court of Chancery of the State of Delaware is the sole and exclusive forum for any derivative action or proceeding brought on our behalf, any action asserting a breach of fiduciary duty, any action asserting a claim against it arising pursuant to any provisions of the DGCL, our certificate of incorporation or our bylaws, or any action asserting a claim against us that is governed by the internal affairs doctrine. The exclusive forum provision does not apply to actions arising under the Exchange Act. Our amended and restated bylaws also provide that the federal district courts of the United States of America will be the exclusive forum for the resolution of any complaint asserting a cause of action under the Securities Act. The provision may limit a stockholder’s ability to bring a claim in a judicial forum that it finds favorable for disputes with us or our directors, officers or other employees, which may discourage such lawsuits against us and our directors, officers and other employees. Alternatively, if a court were to find the choice of forum provision contained in the certificate of incorporation and bylaws to be inapplicable or unenforceable in an action, we may incur additional costs associated with resolving such action in other jurisdictions, which could materially and adversely affect our business, financial condition and results of operations.
We do not anticipate that we will pay any cash dividends in the foreseeable future.
The current expectation is that we will retain our future earnings, if any, to fund the growth of our business as opposed to paying dividends. As a result, capital appreciation, if any, of our common stock will be your sole source of gain, if any, for the foreseeable future.
An active trading market for our common stock may not develop and our stockholders may not be able to exercise less influence over the management and policiesresell their shares of the combined organization following the closing of the Disc Merger than they currently exercise over the management and policies of Gemini.common stock for a profit, if at all.
The Company’sPrior to the merger, there had been no public market for shares of our capital stock. An active trading market for shares of our common stock may never develop or be sustained. If an active market for our common stock does not develop or is not sustained, it may be difficult for our stockholders may not realize a benefit from the Disc Merger commensurate with the ownership dilution they will experience in connection with the Disc Merger.to sell their shares at an attractive price or at all.
Future sales of shares by existing stockholders could cause our stock price to decline.
If the combined company is unableour existing securityholders sell, or indicate an intention to realize the strategic and financial benefits currently anticipated from the proposed Disc Merger, the Company’s stockholders will have experiencedsell, substantial dilution of their ownership interests in the Company without receiving the expected commensurate benefit, or only receive part of the commensurate benefit to the extent the combined company is able to realize only part of the expected strategic and financial benefits currently anticipated from the proposed Disc Merger.
Because the lack of a public market for Disc’s capital stock makes it difficult to evaluate the value of Disc’s capital stock, the stockholders of Disc may receive sharesamounts of our common stock in the Disc Merger that have a value that is greater than the fair market value of Disc’s capital stock.
The outstanding capital stock of Disc is privately held and is not traded in any public market. The lack of a public market makes it extremely difficult to determineafter legal restrictions on resale discussed in our Annual Report on Form 10-K for the fair market value of Disc. Becauseyear ended December 31, 2022 lapse, the percentage of our common stock to be issued to Disc’s stockholders was determined based on negotiations between the parties, it is possible that Gemini may pay more than the aggregate fair market value for Disc, which could adversely impact the value of the holders of Gemini’s common stock prior to the consummation of the merger.
If we do not successfully consummate the Disc Merger or another strategic transaction, our board of directors may decide to pursue a dissolution and liquidation of the Company. In such an event, the amount of cash available for distribution to our stockholders will depend heavily on the timing of such liquidation as well as the amount of cash that will need to be reserved for commitments and contingent liabilities, as to which we can give you no assurance.
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There can be no assurance that the Disc Merger will be completed. If the Disc Merger is not completed, our board of directors may decide to pursue a dissolution and liquidation of the Company. In such an event, the amount of cash available for distribution to our stockholders will depend heavily on the timing of such decision and, ultimately, such liquidation, since the amount of cash available for distribution continues to decrease as we fund our operations while pursuing the Disc Merger. In addition, if our board of directors were to approve and recommend, and our stockholders were to approve, a dissolution and liquidation of the company, we would be required under Delaware corporate law to pay our outstanding obligations, as well as to make reasonable provision for contingent and unknown obligations, prior to making any distributions in liquidation to stockholders. Our commitments and contingent liabilities may include obligations under our employment and related agreements with certain employees that provide for severance and other payments following a termination of employment occurring for various reasons, including a change in control of the company, litigation against us, and other various claims and legal actions arising in the ordinary course of business, and other unexpected and/or contingent liabilities. As a result of this requirement, a portion of our assets would need to be reserved pending the resolution of such obligations.
In addition, we may be subject to litigation or other claims related to a dissolution and liquidation of the Company. If a dissolution and liquidation were to be pursued, our board of directors, in consultation with our advisors, would need to evaluate these matters and make a determination about a reasonable amount to reserve. Accordingly, holderstrading price of our common stock could losedecline. As of June 30, 2023, we had 22,850,894 shares of common stock outstanding. All outstanding shares of common stock, other than shares held by our affiliates will be freely tradable, without restriction, in the public market. In addition, shares of common stock that are subject to outstanding options of ours will become eligible for sale in the public market to the extent permitted by the provisions of various vesting agreements and Rules 144 and 701 under the Securities Act. If these shares are sold, the trading price of our common stock could decline.
Our executive officers, directors and principal stockholders have the ability to control or significantly influence all matters submitted to our stockholders for approval.
Our executive officers, directors and principal stockholders, in the aggregate, beneficially own approximately 54% of our outstanding shares of common stock. As a result, if these stockholders were to choose to act together, they would be able to control or significantly influence all matters submitted to our stockholders for approval, as well as our management and affairs. For example, these persons, if they choose to act together, would control or significantly influence the election of directors and approval of any merger, consolidation or sale of all or a significant portionsubstantially all of their investment inour assets. This concentration of voting power could delay or prevent an acquisition of us on terms that other stockholders may desire.
If equity research analysts do not publish research or reports, or publish unfavorable research or reports, about us, our business or our market, our stock price and trading volume could decline.
The trading market for our common stock will be influenced by the research and reports that equity research analysts publish about us and our business. Equity research analysts may elect not to provide research coverage of our common stock, and such lack of research coverage may adversely affect the market price of our common stock. In the event of a liquidation, dissolutionwe do have equity research analyst
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coverage, we will not have any control over the analysts or winding upthe content and opinions included in their reports. The price of our company. A liquidation would be a lengthy and uncertain process with no assurancecommon stock could decline if one or more equity research analysts downgrade our stock or issue other unfavorable commentary or research. If one or more equity research analysts ceases coverage of any value ever being returnedus or fails to publish reports on us regularly, demand for our stockholders.common stock could decrease, which in turn could cause our stock price or trading volume to decline.
We are substantially dependent onwill have broad discretion in the use of our remaining employees to facilitatecash and cash equivalents and may invest or spend our cash and cash equivalents in ways with which you do not agree and in ways that may not increase the consummationvalue of the merger.your investment.
AsWe will have broad discretion over the use of August 9, 2022, we had only four full-time employeesour cash and one part-time employee.cash equivalents. You may not agree with our decisions, and our use of our cash and cash equivalents may not yield any return on your investment. Our abilityfailure to successfully complete the Disc Merger depends in large part onapply these resources effectively could compromise our ability to retain certain remaining personnel. Despitepursue our effortsgrowth strategy and we might not be able to retainyield a significant return, if any, on our investment of these employees, one or more may terminate their employment with usnet proceeds. You will not have the opportunity to influence our decisions on short notice. The loss of the services of certain employees could potentially harmhow to use our ability to consummate the Disc Merger, to run our day-to-day business operations, as well as to fulfill our reporting obligations as a public company.cash resources.
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ItemITEM 2. Unregistered Sales of Equity Securities and Use of Proceeds.UNREGISTERED SALES OF EQUITY SECURITIES AND USE OF PROCEEDS
(a) Recent Sales of Unregistered Equity Securities and
None.
(b) Use of Proceeds from Initial Public Offering
None.Not applicable.
(b)(c) Issuer Purchases of Equity Securities
None.
Item 3. Defaults Upon Senior Securities.
None.
ItemITEM 3. DEFAULTS UPON SENIOR SECURITIES
None.
ITEM 4. Mine Safety Disclosures.MINE SAFETY DISCLOSURES
Not applicable.
ItemITEM 5. Other Information.OTHER INFORMATION
None.
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ItemITEM 6. Exhibits.EXHIBITS.
(a) ExhibitsRegistration No. 333-249785The exhibits filed as part of this Quarterly Report are set forth on the Exhibit Index, which is incorporated herein by reference.
EXHIBIT INDEX
Exhibit Number | Description | |
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3.1 | ||
3.2 | ||
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3.4 | ||
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31.1* | ||
31.2* | ||
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| Inline XBRL Instance Document | |
| Inline XBRL Taxonomy Extension Schema Document | |
| Inline XBRL Taxonomy Extension Calculation Linkbase Document | |
| Inline XBRL Taxonomy Extension Definition Linkbase Document | |
| Inline XBRL Taxonomy Extension Label Linkbase Document | |
| Inline XBRL Taxonomy Extension Presentation Linkbase Document | |
| Cover Page Interactive Data File (embedded within the Inline XBRL document) |
* Filed herewith.
+ These** The certifications furnished in Exhibit 32.1 hereto are beingdeemed to be furnished solelywith this Quarterly Report on Form 10-Q and will not be deemed to accompany this quarterly report pursuant to 18 U.S.C. Section 1350, and are not being filedbe “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended, except to the extent that the Registrant specifically incorporates it by reference.
† The annexes, schedules, and are notcertain exhibits to be incorporated by reference into any filingthe Merger Agreement have been omitted pursuant to Item 601(b)(2) of the registrant, whether made before or after the date hereof, regardlessRegulation S-K. The Registrant hereby agrees to furnish supplementally a copy of any general incorporation language in such filing.omitted annex, schedule or exhibit to the Commission upon request.
# Indicates management contract or compensatory plan.
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SIGNATURES
SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned thereunto duly authorized.
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Date: August 11, | By: | /s/ | |
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(Principal Executive Officer) | |||
Date: August 11, | By: | /s/ | |
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Chief Financial Officer (Principal Financial and Accounting Officer) | |||
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