Cover
Cover - shares | 9 Months Ended | |
Sep. 30, 2022 | Nov. 04, 2022 | |
Cover [Abstract] | ||
Document Type | 10-Q | |
Document Quarterly Report | true | |
Document Period End Date | Sep. 30, 2022 | |
Document Transition Report | false | |
Entity File Number | 001-35670 | |
Entity Registrant Name | Regulus Therapeutics Inc. | |
Entity Incorporation, State or Country Code | DE | |
Entity Tax Identification Number | 26-4738379 | |
Entity Address, Address Line One | 4224 Campus Point Court, Suite 210 | |
Entity Address, City or Town | San Diego | |
Entity Address, State or Province | CA | |
Entity Address, Postal Zip Code | 92121 | |
City Area Code | 858 | |
Local Phone Number | 202-6300 | |
Title of 12(b) Security | Common Stock, par value $0.001 per share | |
Trading Symbol | RGLS | |
Security Exchange Name | NASDAQ | |
Entity Current Reporting Status | Yes | |
Entity Interactive Data Current | Yes | |
Entity Filer Category | Non-accelerated Filer | |
Entity Small Business | true | |
Entity Emerging Growth Company | false | |
Entity Shell Company | false | |
Entity Common Stock, Shares Outstanding | 16,840,261 | |
Amendment Flag | false | |
Document Fiscal Year Focus | 2022 | |
Document Fiscal Period Focus | Q3 | |
Entity Central Index Key | 0001505512 | |
Current Fiscal Year End Date | --12-31 |
CONDENSED BALANCE SHEETS
CONDENSED BALANCE SHEETS - USD ($) $ in Thousands | Sep. 30, 2022 | Dec. 31, 2021 |
Current assets: | ||
Cash and cash equivalents | $ 20,014 | $ 60,383 |
Short-term investments | 25,310 | 0 |
Restricted cash | 62 | 62 |
Prepaid materials, net | 3,010 | 3,010 |
Prepaid expenses and other current assets | 977 | 1,780 |
Total current assets | 49,373 | 65,235 |
Property and equipment, net | 513 | 281 |
Intangibles, net | 67 | 83 |
Right of use asset | 2,174 | 2,564 |
Other assets | 0 | 291 |
Total assets | 52,127 | 68,454 |
Current liabilities: | ||
Accounts payable | 796 | 285 |
Accrued liabilities | 615 | 821 |
Accrued research and development expenses | 1,081 | 810 |
Accrued compensation | 1,698 | 2,016 |
Current portion of term loan, less debt issuance costs | 4,478 | 0 |
Other current liabilities | 1,853 | 1,295 |
Total current liabilities | 10,521 | 5,227 |
Term loan, less debt issuance costs | 0 | 4,673 |
Lease liability, less current portion | 1,936 | 2,417 |
Other long-term liabilities | 0 | 1,179 |
Total liabilities | 12,457 | 13,496 |
Commitments and Contingencies | ||
Stockholders’ equity: | ||
Common stock, $0.001 par value; 300,000,000 and 400,000,000 shares authorized at September 30, 2022 and December 31, 2021, respectively; 16,840,261 and 14,597,118 shares issued and outstanding at September 30, 2022 (unaudited) and December 31, 2021, respectively | 17 | 15 |
Additional paid-in capital | 516,060 | 509,791 |
Accumulated other comprehensive loss | (31) | 0 |
Accumulated deficit | (476,381) | (454,853) |
Total stockholders’ equity | 39,670 | 54,958 |
Total liabilities and stockholders’ equity | 52,127 | 68,454 |
Class A-1 Convertible Preferred Stock | ||
Stockholders’ equity: | ||
Preferred stock | 0 | 0 |
Class A-2 Convertible Preferred Stock | ||
Stockholders’ equity: | ||
Preferred stock | 1 | 1 |
Class A-3 Convertible Preferred Stock | ||
Stockholders’ equity: | ||
Preferred stock | 0 | 0 |
Class A-4 Convertible Preferred Stock | ||
Stockholders’ equity: | ||
Preferred stock | $ 4 | $ 4 |
CONDENSED BALANCE SHEETS (Paren
CONDENSED BALANCE SHEETS (Parenthetical) - $ / shares | Sep. 30, 2022 | Dec. 31, 2021 |
Common stock par value (in usd per share) | $ 0.001 | $ 0.001 |
Common stock, shares authorized (in shares) | 300,000,000 | 400,000,000 |
Common stock, shares issued (in shares) | 16,840,261 | 14,597,118 |
Common stock, shares outstanding (in shares) | 16,840,261 | 14,597,118 |
Class A-1 Convertible Preferred Stock | ||
Preferred stock, par value (in usd per share) | $ 0.001 | $ 0.001 |
Preferred stock, shares authorized (in shares) | 256,700 | 256,700 |
Preferred stock, shares issued (in shares) | 256,700 | 256,700 |
Preferred stock, shares outstanding (in shares) | 256,700 | 256,700 |
Class A-2 Convertible Preferred Stock | ||
Preferred stock, par value (in usd per share) | $ 0.001 | $ 0.001 |
Preferred stock, shares authorized (in shares) | 1,330,832 | 1,330,832 |
Preferred stock, shares issued (in shares) | 1,330,832 | 1,330,832 |
Preferred stock, shares outstanding (in shares) | 1,330,832 | 1,330,832 |
Class A-3 Convertible Preferred Stock | ||
Preferred stock, par value (in usd per share) | $ 0.001 | $ 0.001 |
Preferred stock, shares authorized (in shares) | 258,707 | 258,707 |
Preferred stock, shares issued (in shares) | 258,707 | 258,707 |
Preferred stock, shares outstanding (in shares) | 258,707 | 258,707 |
Class A-4 Convertible Preferred Stock | ||
Preferred stock, par value (in usd per share) | $ 0.001 | $ 0.001 |
Preferred stock, shares authorized (in shares) | 3,725,720 | 3,725,720 |
Preferred stock, shares issued (in shares) | 3,725,720 | 3,725,720 |
Preferred stock, shares outstanding (in shares) | 3,725,720 | 3,725,720 |
CONDENSED STATEMENTS OF OPERATI
CONDENSED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2022 | Sep. 30, 2021 | Sep. 30, 2022 | Sep. 30, 2021 | |
Operating expenses: | ||||
Research and development | $ 5,310 | $ 5,915 | $ 13,697 | $ 13,385 |
General and administrative | 2,253 | 2,504 | 7,610 | 7,471 |
Total operating expenses | 7,563 | 8,419 | 21,307 | 20,856 |
Loss from operations | (7,563) | (8,419) | (21,307) | (20,856) |
Other income (expense): | ||||
Interest and other income | 187 | 1 | 272 | 823 |
Interest and other expense | (175) | (210) | (492) | (641) |
Loss before income taxes | (7,551) | (8,628) | (21,527) | (20,674) |
Income tax expense | 0 | 0 | (1) | (1) |
Net loss | (7,551) | (8,628) | (21,528) | (20,675) |
Other comprehensive loss: | ||||
Unrealized gain (loss) on short-term investments, net | 5 | 0 | (31) | 0 |
Comprehensive loss | $ (7,546) | $ (8,628) | $ (21,559) | $ (20,675) |
Net loss per share, basic (in usd per share) | $ (0.50) | $ (0.99) | $ (1.46) | $ (2.63) |
Net loss per share, diluted (in usd per share) | $ (0.50) | $ (0.99) | $ (1.46) | $ (2.63) |
Weighted average shares used to compute basic net loss per share (in shares) | 14,969,574 | 8,703,637 | 14,727,591 | 7,855,479 |
Weighted average shares used to compute diluted net loss per share (in shares) | 14,969,574 | 8,703,637 | 14,727,591 | 7,855,479 |
CONDENSED STATEMENTS OF CHANGES
CONDENSED STATEMENTS OF CHANGES IN STOCKHOLDERS' EQUITY (DEFICIT) - USD ($) $ in Thousands | Total | Preferred Stock Convertible preferred stock | Common stock | Additional paid-in capital | Accumulated other comprehensive income (loss) | Accumulated deficit |
Beginning balance, preferred stock shares (in shares) at Dec. 31, 2020 | 1,931,860 | |||||
Beginning balance, common stock shares (in shares) at Dec. 31, 2020 | 6,743,271 | |||||
Beginning balance at Dec. 31, 2020 | $ 26,026 | $ 2 | $ 7 | $ 453,062 | $ 0 | $ (427,045) |
Increase (Decrease) in Stockholders' Equity [Roll Forward] | ||||||
Issuance of common stock upon vesting of restricted stock units (in shares) | 1,684 | |||||
Issuance of common stock upon exercise of options (in shares) | 2,673 | |||||
Issuance of common stock upon exercise of options | 26 | 26 | ||||
Issuance of common stock upon exercise of warrants (in shares) | 241,866 | |||||
Issuance of common stock upon exercise of warrants | 666 | 666 | ||||
Issuance of common stock through ATM (in shares) | 400,970 | |||||
Issuance of common stock through ATM | 5,713 | $ 1 | 5,712 | |||
Issuance of common stock under employee stock purchase plan (in shares) | 411 | |||||
Issuance of common stock under Employee Stock Purchase Plan | 2 | 2 | ||||
Conversions of convertible preferred stock (in shares) | (78,036) | 78,036 | ||||
Conversions of convertible preferred stock | 0 | |||||
Stock-based compensation expense | 691 | 691 | ||||
Net loss | (6,013) | (6,013) | ||||
Ending balance, preferred stock shares (in shares) at Mar. 31, 2021 | 1,853,824 | |||||
Ending balance, common stock shares (in shares) at Mar. 31, 2021 | 7,468,911 | |||||
Ending balance at Mar. 31, 2021 | 27,111 | $ 2 | $ 8 | 460,159 | 0 | (433,058) |
Beginning balance, preferred stock shares (in shares) at Dec. 31, 2020 | 1,931,860 | |||||
Beginning balance, common stock shares (in shares) at Dec. 31, 2020 | 6,743,271 | |||||
Beginning balance at Dec. 31, 2020 | 26,026 | $ 2 | $ 7 | 453,062 | 0 | (427,045) |
Increase (Decrease) in Stockholders' Equity [Roll Forward] | ||||||
Net loss | (20,675) | |||||
Unrealized (loss) gain on short-term investments | 0 | |||||
Ending balance, preferred stock shares (in shares) at Sep. 30, 2021 | 1,846,239 | |||||
Ending balance, common stock shares (in shares) at Sep. 30, 2021 | 8,704,780 | |||||
Ending balance at Sep. 30, 2021 | 29,012 | $ 2 | $ 9 | 476,721 | 0 | (447,720) |
Beginning balance, preferred stock shares (in shares) at Mar. 31, 2021 | 1,853,824 | |||||
Beginning balance, common stock shares (in shares) at Mar. 31, 2021 | 7,468,911 | |||||
Beginning balance at Mar. 31, 2021 | 27,111 | $ 2 | $ 8 | 460,159 | 0 | (433,058) |
Increase (Decrease) in Stockholders' Equity [Roll Forward] | ||||||
Issuance of common stock upon vesting of restricted stock units (in shares) | 1,679 | |||||
Issuance of common stock upon exercise of options (in shares) | 95 | |||||
Issuance of common stock upon exercise of warrants (in shares) | 25,000 | |||||
Issuance of common stock upon exercise of warrants | 166 | 166 | ||||
Issuance of common stock through ATM (in shares) | 1,200,761 | |||||
Issuance of common stock through ATM | 14,831 | $ 1 | 14,830 | |||
Conversions of convertible preferred stock (in shares) | (7,585) | 7,585 | ||||
Stock-based compensation expense | 754 | 754 | ||||
Net loss | (6,034) | (6,034) | ||||
Ending balance, preferred stock shares (in shares) at Jun. 30, 2021 | 1,846,239 | |||||
Ending balance, common stock shares (in shares) at Jun. 30, 2021 | 8,704,031 | |||||
Ending balance at Jun. 30, 2021 | 36,828 | $ 2 | $ 9 | 475,909 | 0 | (439,092) |
Increase (Decrease) in Stockholders' Equity [Roll Forward] | ||||||
Issuance of common stock under employee stock purchase plan (in shares) | 749 | |||||
Issuance of common stock under Employee Stock Purchase Plan | 5 | 5 | ||||
Stock-based compensation expense | 807 | 807 | ||||
Net loss | (8,628) | (8,628) | ||||
Unrealized (loss) gain on short-term investments | 0 | |||||
Ending balance, preferred stock shares (in shares) at Sep. 30, 2021 | 1,846,239 | |||||
Ending balance, common stock shares (in shares) at Sep. 30, 2021 | 8,704,780 | |||||
Ending balance at Sep. 30, 2021 | $ 29,012 | $ 2 | $ 9 | 476,721 | 0 | (447,720) |
Beginning balance, preferred stock shares (in shares) at Dec. 31, 2021 | 5,571,959 | |||||
Beginning balance, common stock shares (in shares) at Dec. 31, 2021 | 14,597,118 | 14,597,118 | ||||
Beginning balance at Dec. 31, 2021 | $ 54,958 | $ 5 | $ 15 | 509,791 | 0 | (454,853) |
Increase (Decrease) in Stockholders' Equity [Roll Forward] | ||||||
Issuance of common stock under employee stock purchase plan (in shares) | 996 | |||||
Issuance of common stock under Employee Stock Purchase Plan | 2 | 2 | ||||
Stock-based compensation expense | 1,000 | 1,000 | ||||
Net loss | (6,719) | (6,719) | ||||
Ending balance, preferred stock shares (in shares) at Mar. 31, 2022 | 5,571,959 | |||||
Ending balance, common stock shares (in shares) at Mar. 31, 2022 | 14,598,114 | |||||
Ending balance at Mar. 31, 2022 | $ 49,241 | $ 5 | $ 15 | 510,793 | 0 | (461,572) |
Beginning balance, preferred stock shares (in shares) at Dec. 31, 2021 | 5,571,959 | |||||
Beginning balance, common stock shares (in shares) at Dec. 31, 2021 | 14,597,118 | 14,597,118 | ||||
Beginning balance at Dec. 31, 2021 | $ 54,958 | $ 5 | $ 15 | 509,791 | 0 | (454,853) |
Increase (Decrease) in Stockholders' Equity [Roll Forward] | ||||||
Net loss | (21,528) | |||||
Unrealized (loss) gain on short-term investments | $ (31) | |||||
Ending balance, preferred stock shares (in shares) at Sep. 30, 2022 | 5,571,959 | |||||
Ending balance, common stock shares (in shares) at Sep. 30, 2022 | 16,840,261 | 16,840,261 | ||||
Ending balance at Sep. 30, 2022 | $ 39,670 | $ 5 | $ 17 | 516,060 | (31) | (476,381) |
Beginning balance, preferred stock shares (in shares) at Mar. 31, 2022 | 5,571,959 | |||||
Beginning balance, common stock shares (in shares) at Mar. 31, 2022 | 14,598,114 | |||||
Beginning balance at Mar. 31, 2022 | 49,241 | $ 5 | $ 15 | 510,793 | 0 | (461,572) |
Increase (Decrease) in Stockholders' Equity [Roll Forward] | ||||||
Issuance of common stock upon vesting of restricted stock units (in shares) | 36,300 | |||||
Stock-based compensation expense | 420 | 420 | ||||
Net loss | (7,258) | (7,258) | ||||
Unrealized (loss) gain on short-term investments | (36) | (36) | ||||
Ending balance, preferred stock shares (in shares) at Jun. 30, 2022 | 5,571,959 | |||||
Ending balance, common stock shares (in shares) at Jun. 30, 2022 | 14,634,414 | |||||
Ending balance at Jun. 30, 2022 | 42,367 | $ 5 | $ 15 | 511,213 | (36) | (468,830) |
Increase (Decrease) in Stockholders' Equity [Roll Forward] | ||||||
Issuance of common stock through ATM (in shares) | 2,205,100 | |||||
Issuance of common stock through ATM | 4,488 | $ 2 | 4,486 | |||
Issuance of common stock under employee stock purchase plan (in shares) | 747 | |||||
Issuance of common stock under Employee Stock Purchase Plan | 1 | 1 | ||||
Stock-based compensation expense | 360 | 360 | ||||
Net loss | (7,551) | (7,551) | ||||
Unrealized (loss) gain on short-term investments | $ 5 | 5 | ||||
Ending balance, preferred stock shares (in shares) at Sep. 30, 2022 | 5,571,959 | |||||
Ending balance, common stock shares (in shares) at Sep. 30, 2022 | 16,840,261 | 16,840,261 | ||||
Ending balance at Sep. 30, 2022 | $ 39,670 | $ 5 | $ 17 | $ 516,060 | $ (31) | $ (476,381) |
CONDENSED STATEMENTS OF CASH FL
CONDENSED STATEMENTS OF CASH FLOWS - USD ($) $ in Thousands | 9 Months Ended | |
Sep. 30, 2022 | Sep. 30, 2021 | |
Operating activities | ||
Net loss | $ (21,528) | $ (20,675) |
Adjustments to reconcile net loss to net cash used in operating activities | ||
Depreciation and amortization expense | 89 | 418 |
Stock-based compensation | 1,780 | 2,252 |
Amortization of discount on investments, net | (52) | 0 |
Gain on PPP Loan forgiveness | 0 | (662) |
Other | 101 | 39 |
Change in operating assets and liabilities: | ||
Contracts and other receivables | 0 | 503 |
Prepaid materials | 0 | 304 |
Prepaid expenses and other assets | 802 | 784 |
Accounts payable | 511 | 51 |
Accrued liabilities | (217) | 193 |
Accrued research and development expenses | 271 | 1,250 |
Accrued compensation | (319) | (316) |
Operating lease right-of-use assets and liabilities, net | (46) | 291 |
Other liabilities | (665) | (729) |
Net cash used in operating activities | (19,273) | (16,297) |
Investing activities | ||
Purchases of short-term investments | (30,289) | 0 |
Sales of short-term investments | 5,000 | 0 |
Purchases of property and equipment | (298) | (210) |
Net cash used in investing activities | (25,587) | (210) |
Financing activities | ||
Proceeds from issuance of common stock, net | 4,491 | 21,384 |
Proceeds from exercise of common stock options | 0 | 27 |
Payments on financing leases | 0 | (81) |
Net cash provided by financing activities | 4,491 | 21,330 |
Net (decrease) increase in cash and cash equivalents | (40,369) | 4,823 |
Cash and cash equivalents at beginning of period | 60,445 | 31,087 |
Cash and cash equivalents at end of period | 20,076 | 35,910 |
Reconciliation of cash, cash equivalents and restricted cash | ||
Cash and cash equivalents | 20,014 | 35,848 |
Restricted cash | 62 | 62 |
Total cash, cash equivalents and restricted cash | 20,076 | 35,910 |
Supplemental disclosure of cash flow information | ||
Paycheck Protection Program loan forgiveness | 0 | 662 |
Interest paid | (348) | (329) |
Income taxes paid | $ (1) | $ (1) |
Basis of Presentation and Summa
Basis of Presentation and Summary of Significant Accounting Policies | 9 Months Ended |
Sep. 30, 2022 | |
Accounting Policies [Abstract] | |
Basis of Presentation and Summary of Significant Accounting Policies | Basis of Presentation and Summary of Significant Accounting Policies Basis of Presentation The accompanying unaudited condensed financial statements have been prepared in accordance with U.S. generally accepted accounting principles (“GAAP”) for interim financial information and the instructions to Form 10-Q and Article 10 of Regulation S-X. Accordingly, they do not include all of the information and footnotes required by GAAP for complete financial statements. In management’s opinion, the accompanying financial statements reflect all adjustments, consisting of normal recurring adjustments, considered necessary for a fair presentation of the results for the interim periods presented. Interim financial results are not necessarily indicative of results anticipated for the full year. These unaudited condensed financial statements should be read in conjunction with the audited financial statements and footnotes included in our Annual Report on Form 10-K for the year ended December 31, 2021, from which the balance sheet information herein was derived. On June 24, 2022, we filed a Certificate of Amendment of Amended and Restated Certificate of Incorporation with the Secretary of State of the state of Delaware to effect a 1-for-10 reverse stock split of our issued and outstanding common stock. The reverse stock split became effective at 5:00 p.m. Eastern Time on June 28, 2022 and our common stock began trading on a split-adjusted basis on The Nasdaq Capital Market on June 29, 2022. The accompanying condensed financial statements and notes thereto give retrospective effect to the reverse stock split for all periods presented. All issued and outstanding common stock, options exercisable for common stock, restricted stock units, common stock issuable upon conversion of our outstanding convertible preferred stock, warrants and per share amounts contained in our condensed financial statements have been retrospectively adjusted. Liquidity The accompanying financial statements have been prepared on a basis which assumes we are a going concern, and does not include any adjustments to reflect the possible future effects on the recoverability and classification of assets or the amounts and classifications of liabilities that may result from any uncertainty related to our ability to continue as a going concern. Through the date of the issuance of these financial statements, we have principally been financed through proceeds received from the sale of our common stock and other equity securities, debt financings, up-front payments and milestones received from collaboration agreements, totaling $542.5 million. As of September 30, 2022, we had approximately $45.3 million of cash, cash equivalents and short-term investments. Based on our operating plans, we believe our cash, cash equivalents and short-term investments may not be sufficient to fund our operations for the period one year following the issuance of these financial statements. As a result, there is substantial doubt about our ability to continue as a going concern. All amounts due under the Term Loan (see note 5) have been classified as a current liability as of September 30, 2022 due to the considerations discussed above and the assessment that the material adverse change clause under the Term Loan is not within our control. We are in compliance with all Loan Agreement covenants. We intend to seek additional capital through equity and/or debt financings, collaborative or other funding arrangements with partners or through other sources of financing. Should we seek additional financing from outside sources, we may not be able to raise such financing on terms acceptable to us or at all. If we are unable to raise additional capital when required or on acceptable terms, we may be required to scale back or discontinue the advancement of product candidates, reduce headcount, file for bankruptcy, reorganize, merge with another entity, or cease operations. If we become unable to continue as a going concern, we may have to liquidate our assets, and might realize significantly less than the values at which they are carried on our financial statements, and stockholders may lose all or part of their investment in our common stock. Use of Estimates Our condensed financial statements are prepared in accordance with GAAP, which requires us to make estimates and assumptions that affect the reported amounts of assets, liabilities, revenues and expenses and the disclosure of contingent assets and liabilities in our financial statements and accompanying notes. An estimated loss contingency is accrued in our financial statements if it is probable that a liability has been incurred and the amount of the loss can be reasonably estimated. Although these estimates are based on our knowledge of current events and actions we may undertake in the future, actual results may ultimately differ from these estimates and assumptions. Additionally, the impact of the COVID-19 pandemic to our business and operating results presents additional uncertainty. Stock-Based Compensation We account for stock-based compensation expense related to stock options granted to employees and members of our board of directors by estimating the fair value of each stock option on the date of grant using the Black-Scholes option pricing model. We recognize stock-based compensation expense using the accelerated multiple-option approach. Under the accelerated multiple-option approach (also known as the graded-vesting method), we recognize compensation expense over the requisite service period for each separately vesting tranche of the award as though the award was in substance multiple awards, resulting in accelerated expense recognition over the vesting period. For performance-based awards granted to employees (i) the fair value of the award is determined on the grant date, (ii) we assess the probability of the individual milestones under the award being achieved and (iii) the fair value of the shares subject to the milestone is expensed over the implicit service period commencing once management believes the performance criteria is probable of being met. We account for restricted stock units by determining the fair value of each restricted stock unit based on the closing market price of our common stock on the date of grant. We recognize stock-based compensation expense using the accelerated multiple-option approach over the requisite service periods of the awards, and forfeitures are recognized as they occur. Clinical Trial and Preclinical Study Accruals We make estimates of our accrued expenses for clinical trial and preclinical study activities as of each balance sheet date in our financial statements based on the facts and circumstances known to us at that time. These accruals are based upon estimates of costs incurred and fees that may be associated with services provided by clinical trial investigational sites and CROs and for other clinical trial-related activities. Payments under certain contracts with such parties depend on factors such as site initiation, successful enrollment of patients and the completion of clinical trial milestones. In accruing for these services, we estimate the time period over which services will be performed and the level of effort to be expended in each period. If possible, we obtain information regarding unbilled services directly from these service providers. However, we may be required to estimate these services based on other information available to us. If we underestimate or overestimate the activities or fees associated with a study or service at a given point in time, adjustments to research and development expenses may be necessary in future periods. Historically, our estimated accrued liabilities have approximated actual expense incurred. Subsequent changes in estimates may result in a material change in our accruals. Prepaid Materials We capitalize the purchase of certain raw materials and related supplies for use in the manufacturing of drug product in our preclinical and clinical development programs, as we have determined that these materials have alternative future use. We can use these raw materials and related supplies in multiple clinical drug products, and therefore these raw materials have future use independent of the development status of any particular drug program until it is utilized in the manufacturing process. We expense the cost of materials when used. We periodically review these capitalized materials for continued alternative future use and write down the asset to its net realizable value in the period in which an impairment is identified. Recent 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 . Subsequently, in November 2018, the FASB issued ASU 2018-19, Codification Improvements to Topic 326, Financial Instruments-Credit Losses . ASU 2016-13 requires entities to measure all expected credit losses for most financial assets held at the reporting date based on an expected loss model which includes historical experience, current conditions, an d reasonable and supportable forecasts. ASU 2016-13 also requires enhanced disclosures to help financial statement users better understand significant estimates and judgments used in estimating credit losses. This ASU is effective for smaller reporting companies for fiscal years beginning after December 15, 2022, with early adoption permitted. We are assessing the impact this standard will have on our financial statements and disclosures. In March 2020, the FASB issued ASU No. 2020-04, Reference Rate Reform (Topic 848), |
Net Loss Per Share
Net Loss Per Share | 9 Months Ended |
Sep. 30, 2022 | |
Earnings Per Share [Abstract] | |
Net Loss Per Share | Net Loss Per Share Basic net loss per share is calculated by dividing net loss by the weighted average number of common shares outstanding for the period, without consideration for common stock equivalents. Diluted net loss per share is calculated by dividing net loss by the weighted-average number of common share equiva lents outstanding for the period determined using the treasury-stock method or if-converted method. Dilutive common stock equivalents are comprised of stock options, restricted stock units, warrants and convertible preferred stock outstanding. For all periods presented, there is no difference in the number of shares used to calculate basic and diluted net loss per share. |
Investments
Investments | 9 Months Ended |
Sep. 30, 2022 | |
Debt Securities, Available-for-Sale [Abstract] | |
Investments | Investments Historically, we have invested our excess cash primarily in debt instruments of financial institutions, corporations, U.S. government-sponsored agencies and the U.S. Treasury. We generally hold our investments to maturity and do not sell our investments before we have recovered our amortized cost basis. Unrealized Maturity (in years) Amortized cost Gains Losses Estimated fair value As of September 30, 2022 U.S. Treasury securities 1 or less $ 25,341 $ — $ (31) $ 25,310 $ 25,341 $ — $ (31) $ 25,310 |
Fair Value Measurements
Fair Value Measurements | 9 Months Ended |
Sep. 30, 2022 | |
Fair Value Disclosures [Abstract] | |
Fair Value Measurements | Fair Value Measurements We have certain financial assets recorded at fair value which have been classified as Level 1, 2, or 3 within the fair value hierarchy as described in the accounting standards for fair value measurements. Accounting standards define fair value as the exchange price that would be received for an asset or paid to transfer a liability (an exit price) in the principal or most advantageous market for the asset or liability in an orderly transaction between market participants as of the measurement date. Market participants are buyers and sellers in the principal market that are (i) independent, (ii) knowledgeable, (iii) able to transact, and (iv) willing to transact. The accounting standards provide an established hierarchy for inputs used in measuring fair value that maximizes the use of observable inputs and minimizes the use of unobservable inputs by requiring that the most observable inputs be used when available. Observable inputs are inputs that market participants would use in valuing the asset or liability and are developed based on market data obtained from independent sources. Unobservable inputs are inputs that reflect our assumptions about the factors that market participants would use in valuing the asset or liability. The accounting standards prioritize the inputs used in measuring the fair value into the following hierarchy: • Level 1 includes financial instruments for which quoted market prices for identical instruments are available in active markets. • Level 2 includes financial instruments for which there are inputs other than quoted prices included within Level 1 that are observable for the instrument such as quoted prices for similar instruments in active markets, quoted prices for identical or similar instruments in markets with insufficient volume or infrequent transactions (less active markets) or model-driven valuations in which significant inputs are observable or can be derived principally from, or corroborated by, observable market data. • Level 3 includes financial instruments for which fair value is derived from valuation techniques in which one or more significant inputs are unobservable, including management’s own assumptions. Financial Assets Measured at Fair Value The following table presents our fair value hierarchy for assets measured at fair value on a recurring basis as of September 30, 2022 and December 31, 2021 (in thousands): Fair value as of September 30, 2022 Total Level 1 Level 2 Level 3 Cash equivalents and short-term investments: Money market funds $ 17,308 $ 17,308 $ — $ — U.S. Treasury securities 25,310 25,310 — — $ 42,618 $ 42,618 $ — $ — Fair value as of December 31, 2021 Total Level 1 Level 2 Level 3 Cash equivalents: Money market funds $ 57,905 $ 57,905 $ — $ — $ 57,905 $ 57,905 $ — $ — |
Debt
Debt | 9 Months Ended |
Sep. 30, 2022 | |
Debt Disclosure [Abstract] | |
Debt | Debt Term Loan On June 17, 2016, we entered into a loan and security agreement ("Loan Agreement") with Oxford Finance, LLC, (the “ Lender ” ), pursuant to which we received $20.0 million in proceeds, net of debt issuance costs, on June 22, 2016 (the "Term Loan"). The outstanding Term Loan will mature on May 1, 2024 (the “Maturity Date”) and bears interest at a floating per annum rate equal to (i) 8.51% plus (ii) the greater of (a) the 30 day U.S. Dollar LIBOR rate reported in The Wall Street Journal on the last business day of the month that immediately precedes the month in which the interest will accrue and (b) 0.44%. Under the original Loan Agreement, we were required to make interest-only payments through June 1, 2018, followed by 24 equal monthly payments of principal and unpaid accrued interest. The Loan Agreement was amended ten times between October 2017 through August 2020. On December 31, 2021, we entered into an eleventh amendment to the Loan Agreement (the "Eleventh Amendment"). Under the terms of the Eleventh Amendment, our required monthly payments to the Lender are to be comprised of interest only through and including (i) December 1, 2022, if the 2022 Equity Event (as defined below) does not occur or (ii) December 1, 2023 if the 2022 Equity Event occurs. The “2022 Equity Event” means the receipt by us, during the calendar year 2022, of unrestricted net cash proceeds of at least $20.0 million from the sale and issuance of our equity securities. In addition, the maturity date for the Term Loan was extended to May 1, 2024. If the 2022 Equity Event does not occur, then commencing on January 1, 2023 and continuing on each successive payment date thereafter through and including the maturity date of May 1, 2024, we will be required to make consecutive equal monthly payments of principal, together with applicable interest, in arrears, to the Lender. If the 2022 Equity Event does occur, then commencing on January 1, 2024 and continuing on each successive payment date thereafter through and including the maturity date of May 1, 2024, we will be required to make consecutive equal monthly payments of principal, together with applicable interest, in arrears, to the Lender. The Eleventh Amendment also provides that we are required to maintain a minimum cash balance of $5.0 million. As consideration for the Lender’s entry into the Eleventh Amendment, we made a payment of $0.3 million to the Lender. We used the proceeds from the Term Loan solely for working capital and to fund our general business requirements. Our obligations under the Loan Agreement are secured by a first priority security interest in substantially all of our current and future assets, other than our intellectual property, for which the Lender currently has a positive lien. We have also agreed not to encumber our intellectual property assets, except as permitted by the Loan Agreement. The Loan Agreement includes customary events of default, including instances of a material adverse change in our operations, that may require prepayment of the outstanding Term Loan. We are in compliance with all Loan Agreement covenants as of the date of the filing of this Form 10-Q. As of September 30, 2022, $4.7 million of principal was outstanding under the Term Loan. An additional $1.3 million is also payable at the conclusion of the Term Loan (the related $1.2 million accrued liability balance is presented in other current liabilities on our balance sheet at September 30, 2022). We had less than $0.1 million of debt issuance costs outstanding as of September 30, 2022, which are being accreted to interest expense over the life of the Term Loan using an effective interest rate of 8.98%. The exit fees are being accrued over the life of the Term Loan through interest expense. As of September 30, 2022, future principal payments for the Term Loan due under the Loan Agreement are as follows (in thousands): 2022 $ — 2023 3,304 2024 1,377 $ 4,681 Paycheck Protection Program Loan On April 23, 2020, we received proceeds in the amount of approximately $0.7 million (the "PPP Loan") from Silicon Valley Bank, as lender, pursuant to the Paycheck Protection Program of the Coronavirus Aid, Relief, and Economic Security Act. The PPP Loan was set to mature on April 23, 2022 and bore interest at a rate of 1.0% per annum. The PPP Loan was evidenced by a promissory note dated April 23, 2020, which contained customary events of default relating to, among other things, payment defaults and breaches of representations and warranties. The PPP Loan was prepayable by us at any time prior to maturity with no prepayment penalties. |
Stockholders' Equity
Stockholders' Equity | 9 Months Ended |
Sep. 30, 2022 | |
Equity [Abstract] | |
Stockholders' Equity | Stockholders’ Equity Common Stock As of September 30, 2022, the re were 16,840,261 shares of common stock outstanding. Each share of common stock is entitled to one vote. The holders of the common stock are also entitled to receive dividends whenever funds are legally available and when declared by our Board of Directors. Reverse Stock Split On June 24, 2022, we filed a Certificate of Amendment of Amended and Restated Certificate of Incorporation with the Secretary of State of the state of Delaware to effect a 1-for-10 reverse stock split of our issued and outstanding common stock. The primary purpose of the reverse stock split was to raise the per share trading price of our common stock to seek to maintain the listing of our common stock on The Nasdaq Capital Market. At the effective time of the reverse stock split, 5:00 p.m. on June 28, 2022, each 10 shares of our issued and outstanding common stock were automatically combined and converted into one issued and outstanding share of common stock. All of our stock options, RSUs and warrants outstanding immediately prior to the reverse stock split, as well as the conversion ratio of our outstanding convertible preferred stock, were proportionately adjusted. All issued and outstanding common stock, options exercisable for common stock, restricted stock units, common stock issuable upon conversion of outstanding convertible preferred stock, warrants a nd per share amounts contained in these financial statements have been retrospectively adjusted. 2019 Equity Incentive Plan On June 15, 2019, the Company's board of directors approved, and on August 1, 2019 the Company's stockholders approved, the Company's 2019 Equity Incentive Plan (the "2019 Plan"). The 2019 Plan is the successor to and continuation of the Company's 2012 Equity Incentive Plan . The number of shares authorized for issuance under the 2019 Plan may be increased by (a) the shares subject to outstanding stock awards granted under the Company’s 2009 Equity Incentive Plan (the “2009 Plan”) and the Company’s 2012 Equity Incentive Plan (together the with 2009 Plan, the “Prior Plans”) that on or after the effective date of the 2019 Plan (i) expire or terminate for any reason prior to exercise or settlement; (ii) are forfeited because of the failure to meet a contingency or condition required to vest such shares or otherwise return to the Company, or (iii) are reacquired, withheld (or not issued) to satisfy a tax withholding obligation in connection with an award or to satisfy the purchase price or exercise price of a stock award. No further grants will be made under the Prior Plan s. In addition, on January 22, 2020, an additional 416,686 shares of common stock became available for issuance under the 2019 Plan pursuant to the Milestone Closing (defined below) of the May 2019 SPA (defined below). Further, on January 1 st of each year, for a period of not more than ten years, beginning on January 1, 2021 and continuing through January 1, 2029, the number of shares authorized for issuance under the 2019 Plan will increase by 5.0% of the total number of shares of our capital stock outstanding on December 31 of the preceding calendar year, or a lesser number of shares as may be determined by our Board of Directors. As of September 30, 2022, 365,628 shares of common stock were available for new equity award grants under the 2019 Plan and 1,470,768 shares of common stock were reserved for issuance pursuant to equity awards outstanding under the 2019 Plan as of September 30, 2022. 2021 Inducement Plan On November 23, 2021, our Board of Directors adopted the 2021 Inducement Plan (the “Inducement Plan”), which became effective immediately. Stockholder approval of the Inducement Plan was not required pursuant to Rule 5635(c)(4) of the Nasdaq Listing Rules. The Inducement Plan initially reserved 200,000 shares of common stock and provides for the grant of NSOs that was used exclusively for grants to individuals that were not previously employees or directors of the Company, as an inducement material to the individual’s entry into employment with the Company. Under the Inducement Plan, options are granted with varying vesting terms, but typically vested over four years, with 25% of the total grant vesting on the first anniversary of the effective date of the option grant and the remaining grant vesting monthly thereafter over the following 36 months. As of September 30, 2022, 160,000 shares of common stock were reserved for future issuance under the Inducement Plan and 40,000 shares of common stock were reserved for future issuance pursuant to equity awards outstanding under the Inducement Plan. 2022 Employee Stock Purchase Plan In June 2022, our stockholders approved and we adopted the 2022 Employee Stock Purchase Plan (“2022 Purchase Plan”), which enables participants to contribute up to 15% of such participant’s eligible compensation during a defined rolling six-month periods to purchase our common stock. The purchase price of common stock under the 2022 Purchase Plan will be the lesser of: (i) 85% of the fair market value of our common stock at the inception of the enrollment period or (ii) 85% of the fair market value of our common stock at the applicable purchase date. The 2022 Purchase Plan supersedes the 2012 Employee Stock Purchase Plan, and no further offerings will be made under the 2012 Employee Stock Purchase Plan. As of September 30, 2022, a maximum of 129,107 shares of our common stock were reserved for future issuance and have been authorized for purchase under the 2022 Purchase Plan. Private Placement of Common Stock and Non-Voting Preferred Stock On November 24, 2021, we entered into a Securities Purchase Agreement (the "November 2021 SPA") with certain institutional and other accredited investors, including one of the Company's directors (the “2021 Purchasers”), pursuant to which we agreed to sell and issue shares of our common stock and shares of newly designated non-voting convertible preferred stock (the “2021 PIPE”). At the closing under the November 2021 SPA that occurred on November 30, 2021 (the “2021 Closing”), we sold and issued to the 2021 Purchasers (i) 5,892,335 shares of common stock at a purchase price of $3.60 per share, and (ii) 372,572 shares of non-voting Class A-4 convertible preferred stock, in lieu of shares of common stock, at a price of $3.60 per share. Total gross proceeds from the 2021 Closing were approximately $34.6 million. Each share of non-voting Class A-4 convertible preferred stock is convertible into one share of common stock, subject to certain beneficial ownership conversion limitations. An aggregate of 222,222 shares of common stock were purchased for $0.8 million by a director of the Company at the 2021 Closing. We evaluated the non-voting Class A-4 convertible preferred stock sold in the 2021 PIPE under ASC 480, Distinguishing Liabilities from Equity, and ASC 815, Derivatives and Hedging, and determined permanent equity treatment was appropriate for these freestanding financial instruments and there were no embedded features that required bifurcation. The following table summarizes preferred stock conversions and warrant exercises (and the related impact on common stoc k) for the three and nine months ended September 30, 2022 and 2021 ( in thousands): Class A-1 Convertible Preferred Stock Class A-2 Convertible Preferred Stock Class A-3 Convertible Preferred Stock Class A-4 Convertible Preferred Stock Common Stock Warrants Common Stock Balance at December 31, 2021 257 1,331 259 3,726 6,187 Conversions/Exercises — — — — — — Balance at March 31, 2022 257 1,331 259 3,726 6,187 Conversions/Exercises — — — — — — Balance at June 30, 2022 257 1,331 259 3,726 6,187 Conversions/Exercises — — — — — — Balance at September 30, 2022 257 1,331 259 3,726 6,187 Balance at December 31, 2020 257 1,416 259 — 6,604 Conversions/Exercises — (78) — — (392) 320 Balance at March 31, 2021 257 1,338 259 — 6,212 Conversions/Exercises — (7) — — (25) 32 Balance at June 30, 2021 257 1,331 259 — 6,187 Conversions/Exercises — — — — — — Balance at September 30, 2021 257 1,331 259 — 6,187 ATM Offering On December 12, 2018, we entered into a Common Stock Sales Agreement (the “Stock Sales Agreement”) with H.C. Wainwright & Co., LLC (“HCW”), pursuant to which we may sell and issue shares of our common stock from time to time through HCW, as our sales agent (the “ATM Offering”). We have no obligation to sell any shares of common stock in the ATM Offering, and may at any time suspend offers under the Stock Sales Agreement or terminate the Stock Sales Agreement. Subject to the terms and conditions of the Stock Sales Agreement, HCW will use its commercially reasonable efforts to sell shares of our common stock from time to time based upon our instructions (including any price, time or size limits or other parameters or conditions that we may impose, subject to certain restrictions). We pay HCW a commission of 3.0% of the gross sales price of any shares sold under the Stock Sales Agreement. On August 10, 2021, we increased the amount of common stock available for sale in the ATM Offering under the Stock Sales Agreement to $50.0 million. A total of 2,205,100 shares were sold and settled for proceeds of $4.5 million (net of $0.1 million in offering costs) under the ATM Offering during the three and nine months ended September 30, 2022. No shares were sold under the ATM Offering during the three months ended September 30, 2021. A total of 16,017,131 shares were sold and settled for proceeds of $20.5 million (net of $0.8 million in offering costs) under the ATM Offering during the nine months ended September 30, 2021. At September 30, 2022, approximately $45.4 million remained eligible to be sold in the ATM Offering, subject to compliance with the rules applicable to sales on Form S-3. Shares Reserved for Future Issuance The following shares of common stock were reserved for future issuance as of September 30, 2022 (in thousands): Class A-1 convertible preferred stock outstanding (as-converted) 257 Class A-2 convertible preferred stock outstanding (as-converted) 1,331 Class A-3 convertible preferred stock outstanding (as-converted) 259 Class A-4 convertible preferred stock outstanding (as-converted) 3,726 2019 PIPE Initial Closing warrants 1,278 2019 PIPE Milestone Closing warrants 3,059 2020 PIPE warrants 1,849 Common stock options outstanding 1,386 RSUs outstanding 85 Common stock available for future grant under the 2019 Equity Incentive Plan 365 Common stock available for future grant under the 2021 Inducement Plan 160 2022 Employee Stock Purchase Plan 129 Total common shares reserved for future issuance 13,884 The following table summarizes our stock option and RSU (together Stock Awards) activity under all equity incentive plans for the nine months ended September 30, 2022 (shares in thousands): Number of Weighted Number of Weighted average grant date fair value Stock Awards outstanding at December 31, 2021 866 $ 11.72 40 $ 9.50 Granted 562 $ 2.60 85 $ 2.57 Exercised (options) or Vested (RSUs) — $ — (36) $ 9.50 Canceled/forfeited/expired (42) $ 26.10 (4) $ 9.50 Stock Awards outstanding at September 30, 2022 1,386 $ 7.58 85 $ 2.57 Stock-Based Compensation The following table summarizes the weighted average assumptions used to estimate the fair value of stock options and performance stock awards granted to employees under our 2019 Equity Incentive Plan, 2021 Inducement Plan and the shares purchasable under our Employee Stock Purchase Plans during the periods presented: Three months ended Nine months ended 2022 2021 2022 2021 Stock options Risk-free interest rate 3.1 % 0.9 % 2.0 % 1.0 % Volatility 97.5 % 96.0 % 96.1 % 95.8 % Dividend yield — — — — Expected term (years) 6.1 6.1 6.1 6.1 Performance stock options Risk-free interest rate — — — 1.0 % Volatility — — — 95.7 % Dividend yield — — — — Expected term (years) 0 0 0 6.1 Employee stock purchase plan shares Risk-free interest rate 1.7 % 0.1 % 0.9 % 0.1 % Volatility 105.9 % 103.1 % 99.3 % 104.4 % Dividend yield — — — — Expected term (years) 0.5 0.5 0.5 0.5 The following table summarizes the allocation of our stock-based compensation expense for all stock awards during the periods presented (in thousands): Three months ended Nine months ended 2022 2021 2022 2021 Research and development $ 56 $ 277 $ 425 $ 687 General and administrative 304 530 1,355 1,565 Total $ 360 $ 807 $ 1,780 $ 2,252 |
Collaborations
Collaborations | 9 Months Ended |
Sep. 30, 2022 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
Collaborations | Collaborations Revenue recognized from our strategic collaboration was zero for the three and nine months ended September 30, 2022 and 2021. Sanofi In July 2012, we amended and restated our collaboration and license agreement with Sanofi to expand the potential therapeutic applications of the micro RNA alliance targets to be developed under such agreement. The following elements were delivered as part of the strategic collaboration with Sanofi: (1) a license for up to four micro RNA targets; and (2) a research license under our technology collaboration. In June 2013, the original research term expired, upon which we and Sanofi entered into an option agreement pursuant to which Sanofi was granted an exclusive right to negotiate the co-development and commercialization of certain of our unencumbered micro RNA programs and we were granted the exclusive right to negotiate with Sanofi for co-development and commercialization of certain miR-21 anti-miRs in oncology and Alport syndrome. In February 2014, we and Sanofi entered into a second amended and restated collaboration and license agreement (the “2014 Sanofi Amendment”) to discover, develop and commercialize micro RNA therapeutics to focus on specific orphan disease and oncology targets. Under the terms of the 2014 Sanofi Amendment, Sanofi had opt-in rights to our clinical fibrosis program targeting miR-21 for the treatment of Alport syndrome, our preclinical program targeting miR-21 for oncology indications, and our preclinical program targeting miR-221/222 for hepatocellular carcinoma (“HCC”). We were responsible for developing each of these programs to proof-of-concept, at which time Sanofi had an exclusive option on each program. If Sanofi chooses to exercise its option on any of these programs, Sanofi would reimburse us for a significant portion of our preclinical and clinical development costs and would also pay us an option exercise fee for any such program, provided that $1.25 million of the $2.5 million upfront option fee paid to us by Sanofi in connection with the June 2013 option agreement will be creditable against such option exercise fee. We are eligible to receive royalties on micro RNA therapeutic products commercialized by Sanofi and will have the right to co-promote these products relating to our preclinical program targeting miR-221/222. As indicated below, we entered into an additional amendment with Sanofi in November 2018, under which Sanofi's opt-in rights to our miR-21 programs under the 2014 Sanofi Amendment were relinquished. Sanofi's opt-in rights with regard to our miR-221/222 preclinical program under the 2014 Sanofi Amendment remained unchanged. We are eligible to receive milestone payments related to the development and commercialization of miR-221/222 for HCC of up to $38.8 million for proof-of-concept option exercise fees (net of $1.25 million creditable, as noted above), $25.0 million for clinical milestones and up to $130.0 million for regulatory and commercial milestones. In addition, we are entitled to receive royalties based on a percentage of net sales of any products from the miR-221/222 program which, in the case of sales in the United States, will be in the middle of the 10% to 20% range, and, in the case of sales outside of the United States, will range from the low end to the middle of the 10% to 20% range, depending upon the volume of sales. If we exercise our option to co-promote a miR-221/222 product, we will continue to be eligible to receive royalties on net sales of each product in the United States at the same rate, unless we elect to share a portion of Sanofi’s profits from sales of such product in the United States in lieu of royalties. In November 2018, we entered into an amendment to the 2014 Sanofi Amendment with Sanofi to modify the parties’ rights and obligations with respect to our miR-21 programs, including our RG-012 program (the “2018 Sanofi Amendment”). Under the terms of the 2018 Sanofi Amendment, we have granted Sanofi a worldwide, royalty-free, fee-bearing, exclusive license, with the right to grant sublicenses, under our know-how and patents to develop and commercialize miR-21 compounds and products for all indications, including Alport Syndrome. Sanofi will control and will assume all responsibilities and obligations for developing and commercializing each of our miR-21 programs, including our obligations regarding the administration and expense of clinical trials and all other costs, including in-license royalties and other in-license payments, related to our miR-21 programs. Under the terms of the 2018 Sanofi Amendment, we have assigned to Sanofi certain agreements, product-specific patents and all materials directed to miR-21 or to any miR-21 compound or product and are required to provide reasonable technical assistance to Sanofi for a period of 24 months after the date of the 2018 Sanofi Amendment. Under the terms of the 2018 Sanofi Amendment, we received approximately $6.8 million in upfront payments for the license and for miR-21 program-related materials (collectively, the “Upfront Amendment Payments”). We were also eligible to receive up to $40.0 million in development milestone payments, including a $10.0 million payment for an interim enrollment milestone (the "Enrollment Milestone"). In addition, Sanofi agreed to reimburse us for certain out-of-pocket transition activities and assume our upstream license royalty obligations. In 2019, we completed the performance obligations under the 2018 Sanofi Amendment and recognized revenue for the $6.8 million in Upfront Amendment Payments. In August 2020, we entered into an amendment to the 2018 Sanofi Amendment (the "2020 Sanofi Amendment"). Under the terms of the 2020 Sanofi Amendment, we agreed to transfer to Sanofi additional RG-012 development program materials (the “Materials”) in exchange for a payment from Sanofi of $1.0 million (the “Transfer Payment”). In addition, in lieu of the $10.0 million Enrollment Milestone under the 2018 Sanofi Amendment, Sanofi agreed to pay us a $4.0 million milestone upon the completion of the transfer and verification of the Materials, and $5.0 million upon achievement of the Enrollment Milestone. Additionally, we are eligible to receive $25.0 million upon achievement of an additional development milestone related to Sanofi's development of the miR-21 compounds. In September 2020, we received $1.0 million in exchange for the transfer of the Materials to Sanofi, and received an additional $4.0 million in October 2020 as a result of Sanofi's completion and verification of the Materials in September 2020. As the performance obligations associated with both of these payments had been satisfied under Topic 606 as of September 30, 2020, both amounts were recognized as revenue in the third quarter of 2020. In November 2020, we received $5.0 million upon achievement of the Enrollment Milestone. As the performance obligations associated with this payment had been satisfied under Topic 606 as of December 31, 2020, this amount was recognized as revenue in the fourth quarter of 2020. On July 12, 2022, we received notification from Sanofi of its decision to terminate the Phase 2 clinical study of lademirsen (RG-012) for the treatment of Alport syndrome for failure to meet Sanofi’s pre-defined futility criteria. Sanofi also notified us that they are evaluating different opportunities with respect to RG-012. We remain eligible to receive the $25.0 million milestone in the event of successful completion of a Phase 2 fibrosis proof of concept study. As of September 30, 2022, the $25.0 million in development milestone payments (variable consideration) is fully constrained and, therefore, does not meet the criteria for revenue recognition. |
Leases
Leases | 9 Months Ended |
Sep. 30, 2022 | |
Leases [Abstract] | |
Leases | Leases At the inception of a contractual arrangement, we determine whether the contract contains a lease by assessing whether there is an identified asset and whether the contract conveys the right to control the use of the identified asset in exchange for consideration over a period of time. For operating leases with an initial term greater than 12 months, we recognize operating lease right of use assets ("ROU assets") and operating lease liabilities based on the present value of lease payments over the lease term at the commencement date. Operating lease ROU assets are comprised of the lease liability plus any lease payments made and excludes lease incentives. Lease terms include options to renew or terminate the lease when we are reasonably certain that the renewal option will be exercised or when it is reasonably certain that the termination option will not be exercised. For our operating leases, we generally cannot determine the interest rate implicit in the lease, in which case we use our incremental borrowing rate as the discount rate for the lease. We estimate our incremental borrowing rate for our operating leases based on what we would normally pay to borrow on a collateralized basis over a similar term for an amount equal to the lease payments. Operating lease expense is recognized on a straight-line basis over the lease term. Leases with a lease term of 12 months or less at inception are not recorded on the unaudited condensed balance sheet. Instead, we recognize lease expense for these leases on a straight-line basis over the lease term. Our lease agreements do not contain any material variable lease payments, residual value guarantees or restrictive covenants. Certain leases require us to pay taxes, insurance, utilities, and maintenance costs for the building, which do not represent lease components. We elected to not separate lease and non-lease components. On June 19, 2019, we entered into a lease agreement (the “Prior Lease”) with ARE SD Region No.44 LLC ("Landlord") for the lease of approximately 8,727 square feet of rentable area of the building located at 10628 Science Center Drive, Suite 225, San Diego, California 92121 (the “Prior Premises”). The commencement date of the Prior Lease was July 1, 2019 (the “Prior Commencement Date”). We used the Prior Premises as our principal executive offices and as a laboratory for research and development and other related uses. The term of the Prior Lease (the “Prior Initial Term”) was two years, six months, ending December 31, 2021. The base rent payments due for the Prior Premises were $0.4 million in 2020 and $0.4 million in 2021, net of certain rent abatement terms. We were also responsible for the payment of additional rent to cover our share of the annual operating expenses of the building, the annual tax expenses of the building and the annual utilities cost of the building. On July 1, 2019, we recorded a $0.8 million lease liability for the Prior Lease, which was calculated as the present value of future lease payments to be made under the Prior Lease. A $0.6 million ROU asset was also recorded on July 1, 2019, which represents the difference between the lease liability and the remaining $0.2 million deferred credit for the reduction of the lease liability under the operating lease agreement with Landlord dated February 25, 2019. On February 11, 2021, we entered into a lease agreement (the "Campus Point Lease") with ARE-SD Region No. 61, LLC (as successor in interest to ARE-SD Region No. 58, LLC) ("Campus Point Landlord"), for the lease of approximately 13,438 square feet of rentable area located at 4224 Campus Point Court, Suite 210, San Diego, California, 92121 (the "Campus Point Premises"). The commencement date of the Campus Point Lease was April 15, 2021. However, for accounting purposes the lease commencement date was February 11, 2021. We are using the Campus Point Premises as our new principal executive offices and as a laboratory for research and development. The term of the Campus Point Lease (“Campus Point Initial Term”) is 60 months, ending April 30, 2026. The aggregate base rent due over the initial term of the Campus Point Lease is approximately $3.8 million. We are also responsible for the payment of additional amounts to cover our share of the annual operating expenses of the building, the annual tax expenses of the building and the utilities costs for the building. On February 11, 2021, concurrently with entry into the Campus Point Lease, we entered into an Assignment and Assumption of Lease (the “Assignment Agreement”) with Turning Point Therapeutics, Inc. (“Assignee”) and a Consent to Assignment (the "Consent") with Landlord. Pursuant to the Assignment Agreement, we assigned all rights, title, and interest under the Prior Lease to Assignee and delivered the Prior Premises to Assignee on April 22, 2021. Pursuant to the Assignment Agreement, Assignee paid us $60,000 in non-refundable assignment consideration. Additionally, the Consent stipulates that we were not required to pay a fee pursuant to the Prior Lease in connection with the assignment. The execution of the Campus Point Lease, Consent, and Assignment Agreement resulted in a modification which was not accounted for as a separate contract. Rather, we accounted for the three contracts with Campus Point Landlord in combination, as they were entered into at the same time and negotiated as a package to achieve the same commercial objective. We accounted for a $0.2 million reduction in the lease liability for the Prior Lease as a deferred credit that is amortized as a reduction to rent expense over the term of the Campus Point Lease. A lease liability of less than $0.1 million and ROU asset of less than $0.1 million remained with respect to the Prior Lease and was fully amortized as of April 30, 2021. On February 11, 2021, we recorded a $3.2 million lease liability for the Campus Point Lease, which was calculated as the present value of future lease payments to be made under the Campus Point Lease. A $3.0 million ROU asset was also recorded on February 11, 2021, which represents the difference between the lease liability and the $0.2 million deferred credit for the reduction of the lease liability under the Prior Lease. Our future lease payments under operating leases at September 30, 2022 are as follows (in thousands): Operating Leases Remaining 2022 $ 191 2023 776 2024 800 2025 824 2026 277 Total operating lease payments $ 2,868 Less: amount representing interest (298) Present value of obligations under operating leases 2,570 Less: current portion (634) Long-term operating lease obligations $ 1,936 |
Basis of Presentation and Sum_2
Basis of Presentation and Summary of Significant Accounting Policies (Policies) | 9 Months Ended |
Sep. 30, 2022 | |
Accounting Policies [Abstract] | |
Basis of Presentation | Basis of Presentation The accompanying unaudited condensed financial statements have been prepared in accordance with U.S. generally accepted accounting principles (“GAAP”) for interim financial information and the instructions to Form 10-Q and Article 10 of Regulation S-X. Accordingly, they do not include all of the information and footnotes required by GAAP for complete financial statements. In management’s opinion, the accompanying financial statements reflect all adjustments, consisting of normal recurring adjustments, considered necessary for a fair presentation of the results for the interim periods presented. Interim financial results are not necessarily indicative of results anticipated for the full year. These unaudited condensed financial statements should be read in conjunction with the audited financial statements and footnotes included in our Annual Report on Form 10-K for the year ended December 31, 2021, from which the balance sheet information herein was derived. On June 24, 2022, we filed a Certificate of Amendment of Amended and Restated Certificate of Incorporation with the Secretary of State of the state of Delaware to effect a 1-for-10 reverse stock split of our issued and outstanding common stock. The reverse stock split became effective at 5:00 p.m. Eastern Time on June 28, 2022 and our common stock began trading on a split-adjusted basis on The Nasdaq Capital Market on June 29, 2022. The accompanying condensed financial statements and notes thereto give retrospective effect to the reverse stock split for all periods presented. All issued and outstanding common stock, options exercisable for common stock, restricted stock units, common stock issuable upon conversion of our outstanding convertible preferred stock, warrants and per share amounts contained in our condensed financial statements have been retrospectively adjusted. |
Use of Estimates | Use of Estimates Our condensed financial statements are prepared in accordance with GAAP, which requires us to make estimates and assumptions that affect the reported amounts of assets, liabilities, revenues and expenses and the disclosure of contingent assets and liabilities in our financial statements and accompanying notes. An estimated loss contingency is accrued in our financial statements if it is probable that a liability has been incurred and the amount of the loss can be reasonably estimated. Although these estimates are based on our knowledge of current events and actions we may undertake in the future, actual results may ultimately differ from these estimates and assumptions. Additionally, the impact of the COVID-19 pandemic to our business and operating results presents additional uncertainty. |
Stock-Based Compensation | Stock-Based Compensation We account for stock-based compensation expense related to stock options granted to employees and members of our board of directors by estimating the fair value of each stock option on the date of grant using the Black-Scholes option pricing model. We recognize stock-based compensation expense using the accelerated multiple-option approach. Under the accelerated multiple-option approach (also known as the graded-vesting method), we recognize compensation expense over the requisite service period for each separately vesting tranche of the award as though the award was in substance multiple awards, resulting in accelerated expense recognition over the vesting period. For performance-based awards granted to employees (i) the fair value of the award is determined on the grant date, (ii) we assess the probability of the individual milestones under the award being achieved and (iii) the fair value of the shares subject to the milestone is expensed over the implicit service period commencing once management believes the performance criteria is probable of being met. We account for restricted stock units by determining the fair value of each restricted stock unit based on the closing market price of our common stock on the date of grant. We recognize stock-based compensation expense using the accelerated multiple-option approach over the requisite service periods of the awards, and forfeitures are recognized as they occur. |
Clinical Trial and Preclinical Study Accruals | Clinical Trial and Preclinical Study Accruals We make estimates of our accrued expenses for clinical trial and preclinical study activities as of each balance sheet date in our financial statements based on the facts and circumstances known to us at that time. These accruals are based upon estimates of costs incurred and fees that may be associated with services provided by clinical trial investigational sites and CROs and for other clinical trial-related activities. Payments under certain contracts with such parties depend on factors such as site initiation, successful enrollment of patients and the completion of clinical trial milestones. In accruing for these services, we estimate the time period over which services will be performed and the level of effort to be expended in each period. If possible, we obtain information regarding unbilled services directly from these service providers. However, we may be required to estimate these services based on other information available to us. If we underestimate or overestimate the activities or fees associated with a study or service at a given point in time, adjustments to research and development expenses may be necessary in future periods. Historically, our estimated accrued liabilities have approximated actual expense incurred. Subsequent changes in estimates may result in a material change in our accruals. |
Prepaid Materials | Prepaid Materials We capitalize the purchase of certain raw materials and related supplies for use in the manufacturing of drug product in our preclinical and clinical development programs, as we have determined that these materials have alternative future use. We can use these raw materials and related supplies in multiple clinical drug products, and therefore these raw materials have future use independent of the development status of any particular drug program until it is utilized in the manufacturing process. We expense the cost of materials when used. We periodically review these capitalized materials for continued alternative future use and write down the asset to its net realizable value in the period in which an impairment is identified. |
Recent Accounting Pronouncements | Recent 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 . Subsequently, in November 2018, the FASB issued ASU 2018-19, Codification Improvements to Topic 326, Financial Instruments-Credit Losses . ASU 2016-13 requires entities to measure all expected credit losses for most financial assets held at the reporting date based on an expected loss model which includes historical experience, current conditions, an d reasonable and supportable forecasts. ASU 2016-13 also requires enhanced disclosures to help financial statement users better understand significant estimates and judgments used in estimating credit losses. This ASU is effective for smaller reporting companies for fiscal years beginning after December 15, 2022, with early adoption permitted. We are assessing the impact this standard will have on our financial statements and disclosures. In March 2020, the FASB issued ASU No. 2020-04, Reference Rate Reform (Topic 848), |
Fair Value Measurement | Fair Value Measurements We have certain financial assets recorded at fair value which have been classified as Level 1, 2, or 3 within the fair value hierarchy as described in the accounting standards for fair value measurements. Accounting standards define fair value as the exchange price that would be received for an asset or paid to transfer a liability (an exit price) in the principal or most advantageous market for the asset or liability in an orderly transaction between market participants as of the measurement date. Market participants are buyers and sellers in the principal market that are (i) independent, (ii) knowledgeable, (iii) able to transact, and (iv) willing to transact. The accounting standards provide an established hierarchy for inputs used in measuring fair value that maximizes the use of observable inputs and minimizes the use of unobservable inputs by requiring that the most observable inputs be used when available. Observable inputs are inputs that market participants would use in valuing the asset or liability and are developed based on market data obtained from independent sources. Unobservable inputs are inputs that reflect our assumptions about the factors that market participants would use in valuing the asset or liability. The accounting standards prioritize the inputs used in measuring the fair value into the following hierarchy: • Level 1 includes financial instruments for which quoted market prices for identical instruments are available in active markets. • Level 2 includes financial instruments for which there are inputs other than quoted prices included within Level 1 that are observable for the instrument such as quoted prices for similar instruments in active markets, quoted prices for identical or similar instruments in markets with insufficient volume or infrequent transactions (less active markets) or model-driven valuations in which significant inputs are observable or can be derived principally from, or corroborated by, observable market data. • Level 3 includes financial instruments for which fair value is derived from valuation techniques in which one or more significant inputs are unobservable, including management’s own assumptions. |
Investments (Tables)
Investments (Tables) | 9 Months Ended |
Sep. 30, 2022 | |
Debt Securities, Available-for-Sale [Abstract] | |
Schedule of Debt Securities | Unrealized Maturity (in years) Amortized cost Gains Losses Estimated fair value As of September 30, 2022 U.S. Treasury securities 1 or less $ 25,341 $ — $ (31) $ 25,310 $ 25,341 $ — $ (31) $ 25,310 |
Fair Value Measurements (Tables
Fair Value Measurements (Tables) | 9 Months Ended |
Sep. 30, 2022 | |
Fair Value Disclosures [Abstract] | |
Fair Value Hierarchy for Assets and Liabilities Measured at Fair Value on Recurring Basis | The following table presents our fair value hierarchy for assets measured at fair value on a recurring basis as of September 30, 2022 and December 31, 2021 (in thousands): Fair value as of September 30, 2022 Total Level 1 Level 2 Level 3 Cash equivalents and short-term investments: Money market funds $ 17,308 $ 17,308 $ — $ — U.S. Treasury securities 25,310 25,310 — — $ 42,618 $ 42,618 $ — $ — Fair value as of December 31, 2021 Total Level 1 Level 2 Level 3 Cash equivalents: Money market funds $ 57,905 $ 57,905 $ — $ — $ 57,905 $ 57,905 $ — $ — |
Debt (Tables)
Debt (Tables) | 9 Months Ended |
Sep. 30, 2022 | |
Debt Disclosure [Abstract] | |
Debt Maturities | As of September 30, 2022, future principal payments for the Term Loan due under the Loan Agreement are as follows (in thousands): 2022 $ — 2023 3,304 2024 1,377 $ 4,681 |
Stockholders' Equity (Tables)
Stockholders' Equity (Tables) | 9 Months Ended |
Sep. 30, 2022 | |
Equity [Abstract] | |
Summary of Activity of Shares Warrant Exercise | The following table summarizes preferred stock conversions and warrant exercises (and the related impact on common stoc k) for the three and nine months ended September 30, 2022 and 2021 ( in thousands): Class A-1 Convertible Preferred Stock Class A-2 Convertible Preferred Stock Class A-3 Convertible Preferred Stock Class A-4 Convertible Preferred Stock Common Stock Warrants Common Stock Balance at December 31, 2021 257 1,331 259 3,726 6,187 Conversions/Exercises — — — — — — Balance at March 31, 2022 257 1,331 259 3,726 6,187 Conversions/Exercises — — — — — — Balance at June 30, 2022 257 1,331 259 3,726 6,187 Conversions/Exercises — — — — — — Balance at September 30, 2022 257 1,331 259 3,726 6,187 Balance at December 31, 2020 257 1,416 259 — 6,604 Conversions/Exercises — (78) — — (392) 320 Balance at March 31, 2021 257 1,338 259 — 6,212 Conversions/Exercises — (7) — — (25) 32 Balance at June 30, 2021 257 1,331 259 — 6,187 Conversions/Exercises — — — — — — Balance at September 30, 2021 257 1,331 259 — 6,187 |
Common Stock Reserved for Future Issuance | The following shares of common stock were reserved for future issuance as of September 30, 2022 (in thousands): Class A-1 convertible preferred stock outstanding (as-converted) 257 Class A-2 convertible preferred stock outstanding (as-converted) 1,331 Class A-3 convertible preferred stock outstanding (as-converted) 259 Class A-4 convertible preferred stock outstanding (as-converted) 3,726 2019 PIPE Initial Closing warrants 1,278 2019 PIPE Milestone Closing warrants 3,059 2020 PIPE warrants 1,849 Common stock options outstanding 1,386 RSUs outstanding 85 Common stock available for future grant under the 2019 Equity Incentive Plan 365 Common stock available for future grant under the 2021 Inducement Plan 160 2022 Employee Stock Purchase Plan 129 Total common shares reserved for future issuance 13,884 |
Summary of Stock Option Activity RSU | The following table summarizes our stock option and RSU (together Stock Awards) activity under all equity incentive plans for the nine months ended September 30, 2022 (shares in thousands): Number of Weighted Number of Weighted average grant date fair value Stock Awards outstanding at December 31, 2021 866 $ 11.72 40 $ 9.50 Granted 562 $ 2.60 85 $ 2.57 Exercised (options) or Vested (RSUs) — $ — (36) $ 9.50 Canceled/forfeited/expired (42) $ 26.10 (4) $ 9.50 Stock Awards outstanding at September 30, 2022 1,386 $ 7.58 85 $ 2.57 |
Summary of Assumptions Used to Estimate Fair Value of Stock Options and Performance Stock and Employee Stock Purchase Plan | The following table summarizes the weighted average assumptions used to estimate the fair value of stock options and performance stock awards granted to employees under our 2019 Equity Incentive Plan, 2021 Inducement Plan and the shares purchasable under our Employee Stock Purchase Plans during the periods presented: Three months ended Nine months ended 2022 2021 2022 2021 Stock options Risk-free interest rate 3.1 % 0.9 % 2.0 % 1.0 % Volatility 97.5 % 96.0 % 96.1 % 95.8 % Dividend yield — — — — Expected term (years) 6.1 6.1 6.1 6.1 Performance stock options Risk-free interest rate — — — 1.0 % Volatility — — — 95.7 % Dividend yield — — — — Expected term (years) 0 0 0 6.1 Employee stock purchase plan shares Risk-free interest rate 1.7 % 0.1 % 0.9 % 0.1 % Volatility 105.9 % 103.1 % 99.3 % 104.4 % Dividend yield — — — — Expected term (years) 0.5 0.5 0.5 0.5 |
Summary of Stock-Based Compensation | The following table summarizes the allocation of our stock-based compensation expense for all stock awards during the periods presented (in thousands): Three months ended Nine months ended 2022 2021 2022 2021 Research and development $ 56 $ 277 $ 425 $ 687 General and administrative 304 530 1,355 1,565 Total $ 360 $ 807 $ 1,780 $ 2,252 |
Leases (Tables)
Leases (Tables) | 9 Months Ended |
Sep. 30, 2022 | |
Leases [Abstract] | |
Operating Lease, Liability, Maturity | Our future lease payments under operating leases at September 30, 2022 are as follows (in thousands): Operating Leases Remaining 2022 $ 191 2023 776 2024 800 2025 824 2026 277 Total operating lease payments $ 2,868 Less: amount representing interest (298) Present value of obligations under operating leases 2,570 Less: current portion (634) Long-term operating lease obligations $ 1,936 |
Basis of Presentation and Sum_3
Basis of Presentation and Summary of Significant Accounting Policies (Details) $ in Millions | 9 Months Ended | |
Jun. 24, 2022 | Sep. 30, 2022 USD ($) | |
Accounting Policies [Abstract] | ||
Reverse stock split ratio | 0.1 | |
Financing from sale proceeds, debt, and agreement revenues | $ 542.5 | |
Cash, cash equivalents and short-term investments | $ 45.3 |
Net Loss Per Share (Details)
Net Loss Per Share (Details) - shares | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2022 | Sep. 30, 2021 | Sep. 30, 2022 | Sep. 30, 2021 | |
Earnings Per Share [Abstract] | ||||
Potentially dilutive securities not included in calculation of diluted net loss per share (in shares) | 13,229,539 | 9,050,339 | 13,229,539 | 9,050,339 |
Investments (Details)
Investments (Details) $ in Thousands | Sep. 30, 2022 USD ($) |
Debt Securities, Available-for-sale [Line Items] | |
Amortized cost | $ 25,341 |
Unrealized gains | 0 |
Unrealized losses | (31) |
Estimated fair value | 25,310 |
U.S. Treasury securities | |
Debt Securities, Available-for-sale [Line Items] | |
Amortized cost | 25,341 |
Unrealized gains | 0 |
Unrealized losses | (31) |
Estimated fair value | $ 25,310 |
Fair Value Measurements (Detail
Fair Value Measurements (Details) - USD ($) $ in Thousands | Sep. 30, 2022 | Dec. 31, 2021 |
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Assets measured at fair value on a recurring basis | $ 42,618 | $ 57,905 |
Level 1 | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Assets measured at fair value on a recurring basis | 42,618 | 57,905 |
Level 2 | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Assets measured at fair value on a recurring basis | 0 | 0 |
Level 3 | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Assets measured at fair value on a recurring basis | 0 | 0 |
Money market funds | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Assets measured at fair value on a recurring basis | 17,308 | 57,905 |
Money market funds | Level 1 | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Assets measured at fair value on a recurring basis | 17,308 | 57,905 |
Money market funds | Level 2 | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Assets measured at fair value on a recurring basis | 0 | 0 |
Money market funds | Level 3 | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Assets measured at fair value on a recurring basis | 0 | $ 0 |
U.S. Treasury securities | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Assets measured at fair value on a recurring basis | 25,310 | |
U.S. Treasury securities | Level 1 | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Assets measured at fair value on a recurring basis | 25,310 | |
U.S. Treasury securities | Level 2 | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Assets measured at fair value on a recurring basis | 0 | |
U.S. Treasury securities | Level 3 | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Assets measured at fair value on a recurring basis | $ 0 |
Debt (Narrative) (Details)
Debt (Narrative) (Details) | 12 Months Ended | 32 Months Ended | |||||
Jun. 22, 2016 USD ($) | Jun. 17, 2016 payment | Dec. 31, 2022 USD ($) | May 31, 2020 amendment | Sep. 30, 2022 USD ($) | Dec. 31, 2021 USD ($) | Apr. 23, 2020 USD ($) | |
Debt Instrument [Line Items] | |||||||
Number of times agreement was amended | amendment | 10 | ||||||
Other long-term liabilities | $ 0 | $ 1,179,000 | |||||
Debt Financing Agreement | |||||||
Debt Instrument [Line Items] | |||||||
Proceeds from borrowing under term loan | $ 20,000,000 | ||||||
Stated interest rate, percentage | 8.51% | ||||||
Number of monthly payments | payment | 24 | ||||||
Debt fee | 1,300,000 | ||||||
Long-term debt | 4,700,000 | ||||||
Debt issuance costs | $ 100,000 | ||||||
Debt instrument effective rate (as a percent) | 8.98% | ||||||
Debt Financing Agreement | London Interbank Offered Rate (LIBOR) | |||||||
Debt Instrument [Line Items] | |||||||
Basis spread on variable rate, percentage | 0.44% | ||||||
Eleventh Amendment | |||||||
Debt Instrument [Line Items] | |||||||
Cash collateral for borrowed securities | 5,000,000 | ||||||
Debt fee | $ 300,000 | ||||||
Eleventh Amendment | Forecast | |||||||
Debt Instrument [Line Items] | |||||||
Unrestricted net cash proceeds amount | $ 20,000,000 | ||||||
Term Loan | |||||||
Debt Instrument [Line Items] | |||||||
Other long-term liabilities | $ 1,200,000 | ||||||
PPP Loan | |||||||
Debt Instrument [Line Items] | |||||||
PPP loan | $ 700,000 |
Debt (Future Principal Payments
Debt (Future Principal Payments) (Details) - Debt Financing Agreement Tranche A $ in Thousands | Sep. 30, 2022 USD ($) |
Debt Instrument [Line Items] | |
2022 | $ 0 |
2023 | 3,304 |
2024 | 1,377 |
Long-term Debt | $ 4,681 |
Stockholders' Equity (Narrative
Stockholders' Equity (Narrative) (Details) $ / shares in Units, $ in Millions | 1 Months Ended | ||||||
Jun. 24, 2022 | Nov. 23, 2021 shares | Jan. 22, 2020 shares | Jun. 30, 2022 | Nov. 30, 2021 USD ($) $ / shares shares | Sep. 30, 2022 vote shares | Dec. 31, 2021 shares | |
Subsidiary, Sale of Stock [Line Items] | |||||||
Common stock, shares outstanding (in shares) | 16,840,261 | 14,597,118 | |||||
Common stock voting rights votes | vote | 1 | ||||||
Reverse stock split ratio | 0.1 | ||||||
Total common shares reserved for future issuance (in shares) | 13,884,000 | ||||||
2021 Closing | |||||||
Subsidiary, Sale of Stock [Line Items] | |||||||
Share sold (in shares) | 5,892,335 | ||||||
Sale of stock price (in usd per share) | $ / shares | $ 3.60 | ||||||
Consideration received | $ | $ 34.6 | ||||||
Shares issued upon conversion (in shares) | 1 | ||||||
Class A-4 Convertible Preferred Stock | 2021 Closing | |||||||
Subsidiary, Sale of Stock [Line Items] | |||||||
Sale of stock price (in usd per share) | $ / shares | $ 3.60 | ||||||
Nonvoting convertible preferred stock issued (in shares) | 372,572 | ||||||
Directors, Executive Officers and Employees | 2021 Closing | |||||||
Subsidiary, Sale of Stock [Line Items] | |||||||
Share sold (in shares) | 222,222 | ||||||
Directors, Executive Officers and Employees | Class A-4 Convertible Preferred Stock | 2021 Closing | |||||||
Subsidiary, Sale of Stock [Line Items] | |||||||
Proceeds from issuance of right to purchase common shares | $ | $ 0.8 | ||||||
2019 Plan | |||||||
Subsidiary, Sale of Stock [Line Items] | |||||||
Number of additional shares authorized (in shares) | 416,686 | ||||||
Number of years for increase of authorized shares | 10 years | ||||||
Number of additional shares authorized yearly increase (as a percent) | 5% | ||||||
Common stock available for future grant (in shares) | 365,628 | ||||||
Total common shares reserved for future issuance (in shares) | 1,470,768 | ||||||
Inducement Plan | |||||||
Subsidiary, Sale of Stock [Line Items] | |||||||
Common stock available for future grant (in shares) | 160,000 | ||||||
Total common shares reserved for future issuance (in shares) | 200,000 | 40,000 | |||||
Vesting period | 4 years | ||||||
Inducement Plan | Share-based Payment Arrangement, Tranche One | |||||||
Subsidiary, Sale of Stock [Line Items] | |||||||
Award vesting percentage | 25% | ||||||
Inducement Plan | Share-based Payment Arrangement, Tranche Two | |||||||
Subsidiary, Sale of Stock [Line Items] | |||||||
Vesting period for remaining vesting rights | 36 months | ||||||
Employee Stock Purchase Plan Two Thousand Twelve | 2022 Employee Stock Purchase Plan | |||||||
Subsidiary, Sale of Stock [Line Items] | |||||||
Share-based compensation arrangement by share-based payment award, discount from market price, purchase date | 15% | ||||||
Share-based compensation arrangement by share-based payment award, purchase price of common stock, percent | 85% | ||||||
Share-based compensation arrangement by share-based payment award, number of shares authorized (in shares) | 129,107 |
Stockholders' Equity (Share Act
Stockholders' Equity (Share Activity) (Details) - shares | 3 Months Ended | |||||
Sep. 30, 2022 | Jun. 30, 2022 | Mar. 31, 2022 | Sep. 30, 2021 | Jun. 30, 2021 | Mar. 31, 2021 | |
Activity of Shares [Roll Forward] | ||||||
Beginning balance, common stock shares (in shares) | 14,597,118 | |||||
Ending balance, common stock shares (in shares) | 16,840,261 | |||||
Class A-1 Convertible Preferred Stock | 2019 SPA and 2020 SPA | ||||||
Activity of Shares [Roll Forward] | ||||||
Beginning balance, preferred stock shares (in shares) | 257,000 | 257,000 | 257,000 | 257,000 | 257,000 | 257,000 |
Conversions of convertible preferred stock (in shares) | 0 | 0 | 0 | 0 | 0 | 0 |
Ending balance, preferred stock shares (in shares) | 257,000 | 257,000 | 257,000 | 257,000 | 257,000 | 257,000 |
Class A-2 Convertible Preferred Stock | 2019 SPA and 2020 SPA | ||||||
Activity of Shares [Roll Forward] | ||||||
Beginning balance, preferred stock shares (in shares) | 1,331,000 | 1,331,000 | 1,331,000 | 1,331,000 | 1,338,000 | 1,416,000 |
Conversions of convertible preferred stock (in shares) | 0 | 0 | 0 | 0 | (7,000) | (78,000) |
Ending balance, preferred stock shares (in shares) | 1,331,000 | 1,331,000 | 1,331,000 | 1,331,000 | 1,331,000 | 1,338,000 |
Class A-3 Convertible Preferred Stock | 2019 SPA and 2020 SPA | ||||||
Activity of Shares [Roll Forward] | ||||||
Beginning balance, preferred stock shares (in shares) | 259,000 | 259,000 | 259,000 | 259,000 | 259,000 | 259,000 |
Conversions of convertible preferred stock (in shares) | 0 | 0 | 0 | 0 | 0 | 0 |
Ending balance, preferred stock shares (in shares) | 259,000 | 259,000 | 259,000 | 259,000 | 259,000 | 259,000 |
Class A-4 Convertible Preferred Stock | ||||||
Activity of Shares [Roll Forward] | ||||||
Beginning balance, preferred stock shares (in shares) | 3,725,720 | |||||
Ending balance, preferred stock shares (in shares) | 3,725,720 | |||||
Class A-4 Convertible Preferred Stock | 2019 SPA and 2020 SPA | ||||||
Activity of Shares [Roll Forward] | ||||||
Beginning balance, preferred stock shares (in shares) | 3,726,000 | 3,726,000 | 3,726,000 | 0 | 0 | 0 |
Conversions of convertible preferred stock (in shares) | 0 | 0 | 0 | 0 | 0 | 0 |
Ending balance, preferred stock shares (in shares) | 3,726,000 | 3,726,000 | 3,726,000 | 0 | 0 | 0 |
Common Stock Warrants | 2019 SPA and 2020 SPA | ||||||
Activity of Shares [Roll Forward] | ||||||
Warrants outstanding (in shares) | 6,187,000 | 6,187,000 | 6,187,000 | 6,187,000 | 6,212,000 | 6,604,000 |
Conversions of convertible preferred stock (in shares) | 0 | 0 | 0 | 0 | (25,000) | (392,000) |
Warrants outstanding (in shares) | 6,187,000 | 6,187,000 | 6,187,000 | 6,187,000 | 6,187,000 | 6,212,000 |
Common stock | 2019 SPA and 2020 SPA | ||||||
Activity of Shares [Roll Forward] | ||||||
Beginning balance, common stock shares (in shares) | ||||||
Conversions of convertible preferred stock (in shares) | 0 | 0 | 0 | 0 | 32,000 | 320,000 |
Ending balance, common stock shares (in shares) |
Stockholders' Equity (ATM Offer
Stockholders' Equity (ATM Offering) (Details) - At The Moment - USD ($) $ in Millions | 3 Months Ended | 9 Months Ended | ||||
Sep. 30, 2022 | Sep. 30, 2021 | Sep. 30, 2022 | Sep. 30, 2021 | Aug. 10, 2021 | Dec. 12, 2018 | |
Class of Stock [Line Items] | ||||||
Commission fee rate (as a percent) | 3% | |||||
Amount eligible to be drawn down under the ATM | $ 45.4 | $ 45.4 | $ 50 | |||
Share sold (in shares) | 2,205,100 | 0 | 2,205,100 | 16,017,131 | ||
Consideration received | $ 4.5 | $ 4.5 | $ 20.5 | |||
Payments for commissions | $ 0.1 | $ 0.1 | $ 0.8 |
Stockholders' Equity (Common St
Stockholders' Equity (Common Stock Reserved for Future Issuance) (Details) shares in Thousands | Sep. 30, 2022 shares |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |
Common stock options outstanding (in shares) | 1,386 |
Total common shares reserved for future issuance (in shares) | 13,884 |
Common stock available for future grant under the 2019 Equity Incentive Plan | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |
Common stock available for future grant (in shares) | 365 |
Common stock available for future grant under the 2021 Inducement Plan | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |
Common stock available for future grant (in shares) | 160 |
2022 Employee Stock Purchase Plan | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |
Common stock available for future grant (in shares) | 129 |
RSUs outstanding | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |
RSUs outstanding (in shares) | 85 |
2019 PIPE Initial Closing warrants | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |
Warrants outstanding (in shares) | 1,278 |
2019 PIPE Milestone Closing warrants | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |
Warrants outstanding (in shares) | 3,059 |
2020 PIPE warrants | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |
Warrants outstanding (in shares) | 1,849 |
Class A-1 convertible preferred stock outstanding (as-converted) | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |
Preferred stock, shares outstanding (in shares) | 257 |
Class A-2 convertible preferred stock outstanding (as-converted) | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |
Preferred stock, shares outstanding (in shares) | 1,331 |
Class A-3 convertible preferred stock outstanding (as-converted) | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |
Preferred stock, shares outstanding (in shares) | 259 |
Class A-4 convertible preferred stock outstanding (as-converted) | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |
Preferred stock, shares outstanding (in shares) | 3,726 |
Stockholders' Equity (Stock Opt
Stockholders' Equity (Stock Option and RSU Activity) (Details) shares in Thousands | 9 Months Ended |
Sep. 30, 2022 $ / shares shares | |
Number of options | |
Ending balance (in shares) | 1,386 |
Stock options | |
Number of options | |
Beginning balance (in shares) | 866 |
Granted (in shares) | 562 |
Exercised (options) or Vested (RSUs) | 0 |
Canceled/forfeited/expired (in shares) | (42) |
Ending balance (in shares) | 1,386 |
Weighted average exercise price | |
Beginning balance (in usd per share) | $ / shares | $ 11.72 |
Granted (in usd per share) | $ / shares | 2.60 |
Exercised (options) or Vested (RSUs) (in usd per share) | $ / shares | 0 |
Canceled/forfeited/expired (in usd per share) | $ / shares | 26.10 |
Ending balance (in usd per share) | $ / shares | $ 7.58 |
Restricted Stock Units (RSUs) | |
Number of RSUs | |
Beginning balance (in shares) | 40 |
Shares granted (in shares) | 85 |
Exercised (options) or Vested (RSUs) (in shares) | (36) |
Canceled/forfeited/expired (in shares) | (4) |
Ending balance (in shares) | 85 |
Weighted average grant date fair value | |
Beginning balance (in usd per share) | $ / shares | $ 9.50 |
Granted (in usd per share) | $ / shares | 2.57 |
Exercised (options) or Vested (RSUs) (in usd per share) | $ / shares | 9.50 |
Canceled/forfeited/expired (in usd per share) | $ / shares | 9.50 |
Ending balance (in usd per share) | $ / shares | $ 2.57 |
Stockholders' Equity (Assumptio
Stockholders' Equity (Assumptions Used to Estimate Fair Value of Stock Options and Performance Stock and Employee Stock Purchase Plan) (Details) | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2022 | Sep. 30, 2021 | Sep. 30, 2022 | Sep. 30, 2021 | |
Stock options | ||||
Weighted average assumptions | ||||
Risk-free interest rate | 3.10% | 0.90% | 2% | 1% |
Volatility | 97.50% | 96% | 96.10% | 95.80% |
Dividend yield | 0% | 0% | 0% | 0% |
Expected term (years) | 6 years 1 month 6 days | 6 years 1 month 6 days | 6 years 1 month 6 days | 6 years 1 month 6 days |
Performance stock options | ||||
Weighted average assumptions | ||||
Risk-free interest rate | 0% | 0% | 0% | 1% |
Volatility | 0% | 0% | 0% | 95.70% |
Dividend yield | 0% | 0% | 0% | 0% |
Expected term (years) | 0 months | 0 months | 0 months | 6 years 1 month 6 days |
Employee stock purchase plan shares | ||||
Weighted average assumptions | ||||
Risk-free interest rate | 1.70% | 0.10% | 0.90% | 0.10% |
Volatility | 105.90% | 103.10% | 99.30% | 104.40% |
Dividend yield | 0% | 0% | 0% | 0% |
Expected term (years) | 6 months | 6 months | 6 months | 6 months |
Stockholders' Equity (Stock-Bas
Stockholders' Equity (Stock-Based Compensation Expense Allocation) (Details) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2022 | Sep. 30, 2021 | Sep. 30, 2022 | Sep. 30, 2021 | |
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | ||||
Stock-based compensation expenses | $ 360 | $ 807 | $ 1,780 | $ 2,252 |
Research and development | ||||
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | ||||
Stock-based compensation expenses | 56 | 277 | 425 | 687 |
General and administrative | ||||
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | ||||
Stock-based compensation expenses | $ 304 | $ 530 | $ 1,355 | $ 1,565 |
Collaborations (Details)
Collaborations (Details) | 1 Months Ended | 3 Months Ended | 9 Months Ended | 12 Months Ended | ||||||||||
Oct. 31, 2020 USD ($) | Sep. 30, 2020 USD ($) | Aug. 31, 2020 USD ($) | Nov. 30, 2018 USD ($) | Feb. 28, 2014 USD ($) | Jun. 30, 2013 USD ($) | Jul. 31, 2012 target | Sep. 30, 2022 USD ($) | Sep. 30, 2021 USD ($) | Sep. 30, 2022 USD ($) | Sep. 30, 2021 USD ($) | Dec. 31, 2019 USD ($) | Jul. 12, 2022 USD ($) | Nov. 30, 2020 USD ($) | |
Revenue under collaborations | ||||||||||||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | ||||||||||||||
Total revenues | $ 0 | $ 0 | $ 0 | $ 0 | ||||||||||
Sanofi | ||||||||||||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | ||||||||||||||
Number of collaborative areas granted | target | 4 | |||||||||||||
Initial upfront option payment | $ 4,000,000 | $ 1,000,000 | $ 1,000,000 | |||||||||||
Potential revenue through milestone payments | $ 6,800,000 | |||||||||||||
Agreement period | 24 months | |||||||||||||
Revenue recognized | $ 6,800,000 | |||||||||||||
Enrollment milestone | 10,000,000 | |||||||||||||
Contract with customer, asset | $ 5,000,000 | |||||||||||||
Development milestone not achievable | 25,000,000 | $ 25,000,000 | ||||||||||||
Sanofi | Minimum | United States | ||||||||||||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | ||||||||||||||
Royalties based on percentage of net sales | 10% | |||||||||||||
Sanofi | Minimum | Outside of the United States | ||||||||||||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | ||||||||||||||
Royalties based on percentage of net sales | 10% | |||||||||||||
Sanofi | Maximum | United States | ||||||||||||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | ||||||||||||||
Royalties based on percentage of net sales | 20% | |||||||||||||
Sanofi | Maximum | Outside of the United States | ||||||||||||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | ||||||||||||||
Royalties based on percentage of net sales | 20% | |||||||||||||
Sanofi | Development Commercialization And License Agreement | ||||||||||||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | ||||||||||||||
Deferred revenue creditable against future milestones | $ 1,250,000 | $ 1,250,000 | ||||||||||||
Initial upfront option payment | $ 2,500,000 | |||||||||||||
Sanofi | Proof-of-Concept Trial | ||||||||||||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | ||||||||||||||
Potential revenue through milestone payments | 38,800,000 | 38,800,000 | ||||||||||||
Sanofi | Clinical | ||||||||||||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | ||||||||||||||
Potential revenue through milestone payments | 25,000,000 | 25,000,000 | ||||||||||||
Sanofi | Regulatory and Commercialization Milestones | ||||||||||||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | ||||||||||||||
Potential revenue through milestone payments | $ 130,000,000 | $ 130,000,000 | ||||||||||||
Sanofi | Development Milestones | ||||||||||||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | ||||||||||||||
Potential revenue through milestone payments | 25,000,000 | $ 40,000,000 | $ 25,000,000 | |||||||||||
Sanofi | Enrollment Milestone | ||||||||||||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | ||||||||||||||
Potential revenue through milestone payments | $ 10,000,000 | |||||||||||||
Sanofi | Materials Sold | ||||||||||||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | ||||||||||||||
Potential revenue through milestone payments | 4,000,000 | |||||||||||||
Sanofi | Achievement of Enrollment Milestone | ||||||||||||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | ||||||||||||||
Potential revenue through milestone payments | $ 5,000,000 |
Leases (Narrative) (Details)
Leases (Narrative) (Details) $ in Thousands | Feb. 11, 2021 USD ($) ft² contract | Sep. 30, 2022 USD ($) | Dec. 31, 2021 USD ($) | Apr. 30, 2021 USD ($) | Dec. 31, 2020 USD ($) | Jul. 01, 2019 USD ($) | Jun. 19, 2019 ft² |
Lessee, Lease, Description [Line Items] | |||||||
Remaining liability to be paid | $ 191 | ||||||
Base rent payments due 2021 | 776 | ||||||
ROU asset | 2,174 | $ 2,564 | |||||
Operating lease payments due | $ 2,868 | ||||||
Prior Premises | |||||||
Lessee, Lease, Description [Line Items] | |||||||
Net rentable area | ft² | 8,727 | ||||||
Term of contract | 2 years 6 months | ||||||
Remaining liability to be paid | $ 400 | ||||||
Base rent payments due 2021 | $ 400 | ||||||
Initial direct cost liability | $ 800 | ||||||
ROU asset | 600 | ||||||
Deferred credit, operating lease liabilities | $ 200 | ||||||
Campus Point Lease | |||||||
Lessee, Lease, Description [Line Items] | |||||||
Term of contract | 60 months | ||||||
Initial direct cost liability | $ 3,200 | $ 100 | |||||
ROU asset | 3,000 | 100 | |||||
Deferred credit, operating lease liabilities | $ 200 | ||||||
Rented area | ft² | 13,438 | ||||||
Operating lease payments due | $ 3,800 | ||||||
Nonrefundable lease cost for assignment | $ 60 | ||||||
Number of operating lease contracts | contract | 3 | ||||||
Deferred cost of liability | $ 200 |
Leases (Operating and Finance M
Leases (Operating and Finance Maturities) (Details) - USD ($) $ in Thousands | Sep. 30, 2022 | Dec. 31, 2021 |
Lessee, Operating Lease, Liability, Payment, Due [Abstract] | ||
Remaining 2022 | $ 191 | |
2023 | 776 | |
2024 | 800 | |
2025 | 824 | |
2026 | 277 | |
Total operating lease payments | 2,868 | |
Less: amount representing interest | (298) | |
Present value of obligations under operating leases | 2,570 | |
Less: current portion | (634) | |
Long-term operating lease obligations | $ 1,936 | $ 2,417 |