Cover
Cover - shares | 3 Months Ended | |
Mar. 31, 2022 | May 03, 2022 | |
Cover [Abstract] | ||
Document Type | 10-Q | |
Amendment Flag | false | |
Document Quarterly Report | true | |
Document Transition Report | false | |
Document Period End Date | Mar. 31, 2022 | |
Document Fiscal Period Focus | Q1 | |
Document Fiscal Year Focus | 2022 | |
Current Fiscal Year End Date | --12-31 | |
Entity File Number | 001-15771 | |
Entity Registrant Name | ABEONA THERAPEUTICS INC. | |
Entity Central Index Key | 0000318306 | |
Entity Tax Identification Number | 83-0221517 | |
Entity Incorporation, State or Country Code | DE | |
Entity Address, Address Line One | 1330 Avenue of the Americas | |
Entity Address, Address Line Two | 33rd Floor | |
Entity Address, City or Town | New York | |
Entity Address, State or Province | NY | |
Entity Address, Postal Zip Code | 10019 | |
City Area Code | (646) | |
Local Phone Number | 813-4701 | |
Title of 12(b) Security | Common Stock, $0.01 par value | |
Trading Symbol | ABEO | |
Security Exchange Name | NASDAQ | |
Entity Current Reporting Status | Yes | |
Entity Interactive Data Current | Yes | |
Entity Filer Category | Non-accelerated Filer | |
Entity Small Business | true | |
Entity Emerging Growth Company | false | |
Entity Shell Company | false | |
Entity Common Stock, Shares Outstanding | 146,949,529 |
Condensed Consolidated Balance
Condensed Consolidated Balance Sheets - USD ($) $ in Thousands | Mar. 31, 2022 | Dec. 31, 2021 |
Current assets: | ||
Cash and cash equivalents | $ 20,326 | $ 32,938 |
Short-term investments | 10,989 | 12,086 |
Restricted cash | 5,891 | 5,891 |
Accounts receivable | 3,000 | |
Prepaid expenses and other current assets | 1,998 | 2,377 |
Total current assets | 39,204 | 56,292 |
Property and equipment, net | 8,408 | 12,339 |
Right-of-use lease assets | 7,540 | 9,403 |
Licensed technology, net | 1,384 | |
Other assets | 20 | 168 |
Total assets | 55,172 | 79,586 |
Current liabilities: | ||
Accounts payable | 1,601 | 4,325 |
Accrued expenses | 4,206 | 5,585 |
Current portion of lease liability | 1,822 | 1,818 |
Current portion of payable to licensor | 4,708 | 4,599 |
Deferred revenue | 296 | |
Total current liabilities | 12,337 | 16,623 |
Payable to licensor | 3,919 | 3,828 |
Other long-term liabilities | 200 | 200 |
Long-term lease liabilities | 7,273 | 7,560 |
Total liabilities | 23,729 | 28,211 |
Commitments and contingencies | ||
Stockholders’ equity: | ||
Preferred stock - $0.01 par value; authorized 2,000,000 shares; No shares issued and outstanding as of March 31, 2022 and December 31, 2021, respectively | ||
Common stock - $0.01 par value; authorized 200,000,000 shares; 147,079,899 and 147,205,422 shares issued and outstanding as of March 31, 2022 and December 31, 2021, respectively | 1,471 | 1,472 |
Additional paid-in capital | 706,433 | 705,570 |
Accumulated deficit | (676,431) | (655,640) |
Accumulated other comprehensive loss | (30) | (27) |
Total stockholders’ equity | 31,443 | 51,375 |
Total liabilities and stockholders’ equity | $ 55,172 | $ 79,586 |
Condensed Consolidated Balanc_2
Condensed Consolidated Balance Sheets (Parenthetical) - $ / shares | Mar. 31, 2022 | Dec. 31, 2021 |
Statement of Financial Position [Abstract] | ||
Preferred stock, par value | $ 0.01 | $ 0.01 |
Preferred stock, shares authorized | 2,000,000 | 2,000,000 |
Preferred stock, shares issued | 0 | 0 |
Preferred stock, shares outstanding | 0 | 0 |
Common stock, par value | $ 0.01 | $ 0.01 |
Common stock, shares authorized | 200,000,000 | 200,000,000 |
Common stock, shares issued | 147,079,899 | 147,205,422 |
Common stock, shares outstanding | 147,079,899 | 147,205,422 |
Condensed Consolidated Statemen
Condensed Consolidated Statements of Operations and Comprehensive Loss (Unaudited) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2022 | Mar. 31, 2021 | |
Revenues: | ||
License and other revenues | $ 346 | |
Expenses: | ||
Research and development | 10,545 | 8,317 |
General and administrative | 4,224 | 6,280 |
Licensed technology impairment charge | 1,355 | |
Lease impairment charge | 1,561 | |
Construction-in-progress impairment charge | 3,252 | |
Total expenses | 20,937 | 14,597 |
Loss from operations | (20,591) | (14,597) |
Interest and miscellaneous income | 1 | 15 |
Interest expense | (201) | (1,420) |
Net loss | $ (20,791) | $ (16,002) |
Basic and diluted loss per common share | $ (0.14) | $ (0.17) |
Weighted average number of common shares outstanding – basic and diluted | 144,877,693 | 94,234,653 |
Other comprehensive income: | ||
Change in unrealized gains related to available-for-sale debt securities | $ 3 | $ 13 |
Comprehensive loss | $ (20,788) | $ (15,989) |
Condensed Consolidated Statem_2
Condensed Consolidated Statements of Stockholders' Equity (Unaudited) - USD ($) $ in Thousands | Common Stock [Member] | Additional Paid-in Capital [Member] | Retained Earnings [Member] | AOCI Attributable to Parent [Member] | Total |
Beginning balance, value at Dec. 31, 2020 | $ 961 | $ 672,304 | $ (570,704) | $ (10) | $ 102,551 |
Beginning balance, shares at Dec. 31, 2020 | 96,131,678 | ||||
Stock-based compensation expense | 1,950 | 1,950 | |||
Issuance of common stock under open market sale agreement | $ 16 | 5,195 | 5,211 | ||
Issuance of common stock under open market sale agreement, shares | 1,578,324 | ||||
Issuance of common stock in connection with the exercise of stock options | $ 5 | 662 | 667 | ||
Issuance of common stock in connection with the exercise of stock options, shares | 488,204 | ||||
Net loss | (16,002) | (16,002) | |||
Other comprehensive income | 13 | 13 | |||
Issuance of common stock in connection with restricted share awards, net of cancellations | $ 8 | (8) | |||
Issuance of common stock in connection with restricted share awards, net of cancellations, shares | 840,727 | ||||
Ending balance, value at Mar. 31, 2021 | $ 990 | 680,103 | (586,706) | 3 | 94,390 |
Ending balance, shares at Mar. 31, 2021 | 99,038,933 | ||||
Beginning balance, value at Dec. 31, 2021 | $ 1,472 | 705,570 | (655,640) | (27) | 51,375 |
Beginning balance, shares at Dec. 31, 2021 | 147,205,422 | ||||
Stock-based compensation expense | 862 | 862 | |||
Issuance of common stock in connection with restricted share awards, net of cancellations | $ (1) | 1 | |||
Cancellations, net , shares | (125,523) | ||||
Net loss | (20,791) | (20,791) | |||
Other comprehensive income | (3) | (3) | |||
Ending balance, value at Mar. 31, 2022 | $ 1,471 | $ 706,433 | $ (676,431) | $ (30) | $ 31,443 |
Ending balance, shares at Mar. 31, 2022 | 147,079,899 |
Condensed Consolidated Statem_3
Condensed Consolidated Statements of Cash Flows (Unaudited) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2022 | Mar. 31, 2021 | |
Cash flows from operating activities: | ||
Net loss | $ (20,791) | $ (16,002) |
Adjustments to reconcile net loss to cash used in operating activities: | ||
Depreciation and amortization | 811 | 817 |
Stock-based compensation expense | 862 | 1,950 |
Non-cash licensed technology impairment charge | 1,355 | |
Non-cash lease impairment charge | 1,561 | |
Non-cash construction-in-progress impairment charge | 3,252 | |
Accretion and interest on short-term investments | (84) | 125 |
Amortization of right-of-use lease assets | 302 | 268 |
Non cash interest | 200 | |
Change in operating assets and liabilities: | ||
Accounts receivable | 3,000 | |
Prepaid expenses and other current assets | 379 | 882 |
Other assets | 148 | (20) |
Accounts payable, accrued expenses and lease liabilities | (4,386) | (3,024) |
Change in payable to licensor | (296) | 1,419 |
Net cash used in operating activities | (13,687) | (13,585) |
Cash flows from investing activities: | ||
Capital expenditures | (103) | (444) |
Purchases of short-term investments | (7,487) | (15,164) |
Proceeds from maturities of short-term investments | 8,665 | 24,984 |
Net cash provided by investing activities | 1,075 | 9,376 |
Cash flows from financing activities: | ||
Proceeds from open market sales of common stock | 5,211 | |
Proceeds from exercise of stock options | 667 | |
Net cash provided by financing activities | 5,878 | |
Net increase/(decrease) in cash and cash equivalents | (12,612) | 1,669 |
Cash and cash equivalents at beginning of period | 32,938 | 13,571 |
Cash and cash equivalents at end of period | 20,326 | 15,240 |
Supplemental cash flow information: | ||
Cash and cash equivalents | 20,326 | 14,265 |
Restricted cash | 5,891 | 975 |
Total cash, cash equivalents and restricted cash | $ 26,217 | $ 15,240 |
NATURE OF OPERATIONS AND SIGNIF
NATURE OF OPERATIONS AND SIGNIFICANT ACCOUNTING POLICIES | 3 Months Ended |
Mar. 31, 2022 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
NATURE OF OPERATIONS AND SIGNIFICANT ACCOUNTING POLICIES | NOTE 1 – NATURE OF OPERATIONS AND SIGNIFICANT ACCOUNTING POLICIES Background Abeona Therapeutics Inc. (together with our subsidiaries, “we,” “our,” “Abeona” or the “Company”), a Delaware corporation, is a clinical-stage biopharmaceutical company developing gene and cell therapies for life-threatening rare genetic diseases. Our lead clinical program is EB-101, an autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa (“RDEB”), which is currently in the pivotal Phase 3 VIITAL™ clinical trial. Following a comprehensive portfolio review in early 2022, we have decided to focus our research and development resources on the VIITAL™ readout while actively pursuing a potential commercialization partner for EB-101 with the objective of reducing operating expenses and extending our cash runway. As part of this portfolio prioritization, we have intensified our pursuit of a strategic partnership to take over development activities for our adeno-associated virus (“AAV”)-based gene therapy ABO-102 for Sanfilippo syndrome type A (“MPS IIIA”) and we have discontinued development of our AAV-based gene therapy ABO-101 for Sanfilippo syndrome type B (“MPS IIIB”). We plan to continue development of AAV-based gene therapies designed to treat ophthalmic and other diseases and next-generation AAV-based gene therapies using the novel AIM™ capsid platform that we have exclusively licensed from the University of North Carolina at Chapel Hill (“UNC”), and internal AAV vector research programs. Basis of Presentation The Company’s unaudited interim condensed consolidated financial statements have been prepared in conformity with accounting principles generally accepted in the United States of America (“U.S. GAAP”). All intercompany balances and transactions have been eliminated in consolidation. In the opinion of management, all adjustments, consisting only of normal recurring adjustments, except as otherwise disclosed, necessary for the fair presentation of the financial position, results of operations, and changes in financial position for such periods, have been made. These unaudited interim condensed financial statement results are not necessarily indicative of results to be expected for the full fiscal year or any future period. Certain information that is normally required by U.S. GAAP has been condensed or omitted in accordance with rules and regulations of the U.S. Securities and Exchange Commission (“SEC”). Therefore, these unaudited interim condensed consolidated financial statements should be read in conjunction with the audited consolidated financial statements and notes thereto included in our Annual Report on Form 10-K for the year ended December 31, 2021, which was filed with the SEC on March 31, 2022. Uses and Sources of Liquidity The unaudited interim condensed consolidated financial statements have been prepared on the going concern basis, which assumes the Company will have sufficient cash to pay its operating expenses, as and when they become payable, for a period of at least 12 months from the date the financial report is issued. As of March 31, 2022, we had cash, cash equivalents, restricted cash and short-term investments of $ 37.2 13.7 We are subject to a number of risks similar to other life science companies, including, but not limited to, risks related to the successful discovery and development of product candidates, obtaining the necessary regulatory approval to market our product candidates, raising additional capital to continue to fund our operations, development of competing drugs and therapies, protection of proprietary technology and market acceptance of our products. As a result of these and other risks and the related uncertainties, there can be no assurance of our future success. Following a comprehensive portfolio review in early 2022, we have decided to focus our research and development resources on the EB-101 program with the objective of reducing operating expenses and extending our cash runway. As part of this portfolio prioritization, we have intensified our pursuit of a strategic partnership to take over development activities for our AAV-based gene therapy ABO-102 for MPS IIIA and we have discontinued development of our AAV-based gene therapy ABO-101 for MPS IIIB. Based upon these current operating plans, our ability to access additional financial resources and/or our financial flexibility to further reduce operating expenses if required, we believe that we have sufficient resources to fund operations through at least the next 12 months from the date of this Quarterly Report on Form 10-Q. We will need to secure additional funding beyond the next 12 months to carry out all of our planned research and development activities. If we are unable to obtain additional financing or generate license or product revenue, the lack of liquidity and sufficient capital resources could have a material adverse effect on our future prospects. Use of Estimates The preparation of unaudited interim condensed consolidated financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amount of assets and disclosure of contingent assets and liabilities at the date of the unaudited interim condensed consolidated financial statements and the reported amounts of revenue and expenses during the reported period. Actual results could differ from these estimates and assumptions. Summary of Significant Accounting Policies There have been no new or material changes to the significant accounting policies discussed in the Company’s Annual Report on Form 10-K for the year ended December 31, 2021 that are of significance, or potential significance, to the Company. Reclassifications Certain comparative figures have been reclassified to conform to the current year presentation. The Company reclassified depreciation and amortization costs of $ 0.8 35,000 0.3 5.0 0.9 Net Loss Per Share Basic and diluted net loss per share is computed by dividing net loss by the weighted-average number of shares of common stock. We do not include the potential impact of dilutive securities in diluted net loss per share, as the impact of these items is anti-dilutive. Potential dilutive securities result from outstanding restricted stock, stock options, and stock purchase warrants. The following table sets forth the potential securities that could potentially dilute basic income/(loss) per share in the future that were not included in the computation of diluted net loss per share because to do so would have been anti-dilutive for the periods presented: SCHEDULE OF ANTI-DILUTIVE SECURITIES EXCLUDED FROM COMPUTATION OF EARNINGS PER SHARE 2022 2021 For the three months ended March 31, 2022 2021 Stock options 7,101,803 7,091,879 Restricted stock 1,948,334 2,636,216 Warrants 44,700,000 - Total 53,750,137 9,798,095 |
SHORT-TERM INVESTMENTS
SHORT-TERM INVESTMENTS | 3 Months Ended |
Mar. 31, 2022 | |
Investments, Debt and Equity Securities [Abstract] | |
SHORT-TERM INVESTMENTS | NOTE 2 – SHORT-TERM INVESTMENTS Short-term investments consisted of the following marketable securities as of: SCHEDULE OF AVAILABLE FOR SALE SHORT-TERM INVESTMENTS (in thousands) March 31, 2022 Amortized Cost Gross Unrealized Gain Gross Unrealized Loss Fair Value Available-for-sale, short-term investments U.S. treasury securities $ 10,986 $ 3 $ - $ 10,989 Total $ 10,986 $ 3 $ - $ 10,989 December 31, 2021 Amortized Cost Gross Unrealized Gain Gross Unrealized Loss Fair Value Available-for-sale, short-term investments U.S. treasury securities $ 12,077 $ 9 $ - $ 12,086 Total $ 12,077 $ 9 $ - $ 12,086 As of March 31, 2022, the available-for-sale securities classified as short-term investments mature in one year or less. Unrealized losses on available-for-sale securities as of March 31, 2022 were not significant and were primarily due to changes in interest rates, including market credit spreads, and not due to increased credit risks associated with specific securities. None of the short-term investments have been in a continuous unrealized loss position for more than 12 months. Accordingly, no other-than-temporary impairment was recorded for the three months ended March 31, 2022. There were no |
PROPERTY AND EQUIPMENT, NET
PROPERTY AND EQUIPMENT, NET | 3 Months Ended |
Mar. 31, 2022 | |
Property, Plant and Equipment [Abstract] | |
PROPERTY AND EQUIPMENT, NET | NOTE 3 – PROPERTY AND EQUIPMENT, NET Property and equipment are stated at cost and depreciated or amortized using the straight-line method based on useful lives as follow: SCHEDULE OF PROPERTY AND EQUIPMENT (in thousands) Useful lives (years) March 31, December 31, Laboratory equipment 5 $ 9,138 $ 9,081 Furniture, software and office equipment 3 5 1,908 1,896 Leasehold improvements Shorter of remaining lease term or useful life 8,603 8,603 Construction-in-progress 3,252 3,219 Subtotal 22,901 22,799 Less: accumulated depreciation (11,241 ) (10,460 ) Less: construction-in-progress impairment (3,252 ) - Property and equipment, net $ 8,408 $ 12,339 Depreciation expense was $ 0.8 On March 31, 2022, the Company announced that we were pursuing a strategic partner to take over development activities of ABO-102 and that we were discontinuing development of ABO-101. As a result of this shift in priorities, the Company determined the construction-in-progress which was dedicated to the ABO-101 and ABO-102 programs, had no future value and thus, we recorded an impairment charge of $ 3.3 million for the three months ended March 31, 2022. |
LICENSED TECHNOLOGY
LICENSED TECHNOLOGY | 3 Months Ended |
Mar. 31, 2022 | |
Goodwill and Intangible Assets Disclosure [Abstract] | |
LICENSED TECHNOLOGY | NOTE 4 – LICENSED TECHNOLOGY On May 15, 2015, we acquired Abeona Therapeutics LLC, which had an exclusive license through Nationwide Children’s Hospital to the AB-101 and AB-102 patent portfolios for developing treatments for patients with Sanfilippo Syndrome Type A and Type B. The license is amortized over the life of the license of 20 years. On March 31, 2022, the Company announced that it was pursuing a strategic partner to take over development activities of ABO-102 and that it was discontinuing development of ABO-101. As a result of this shift in priorities, the Company determined the remaining value of the licensed technology had no future value and thus, recorded an impairment charge of $ 1.4 million for the three months ended March 31, 2022. Licensed technology consists of the following: SCHEDULE OF LICENSED TECHNOLOGY (in thousands) March 31, December 31, Licensed technology $ 2,156 $ 2,156 Less accumulated amortization (801 ) (772 ) Less impairment charge (1,355 ) - Licensed technology, net $ - $ 1,384 Amortization expense on licensed technology was $ 29,000 |
SETTLEMENT LIABILITY
SETTLEMENT LIABILITY | 3 Months Ended |
Mar. 31, 2022 | |
Commitments and Contingencies Disclosure [Abstract] | |
SETTLEMENT LIABILITY | NOTE 5 – SETTLEMENT LIABILITY On November 12, 2021, we entered into a settlement agreement (“Settlement Agreement”) with our prior licensor, REGENXBIO Inc. (“REGENXBIO”) to resolve all existing disputes between the parties. In accordance with the Settlement Agreement, we agreed to pay REGENXBIO a total of $ 30.0 million, payable as follows: (1) $20.0 million paid in November 2021 after execution of the Settlement Agreement, (2) $5.0 million on the first anniversary of the effective date of the Settlement Agreement, and (3) $5.0 million upon the earlier of: (i) the third anniversary of the effective date of the Settlement Agreement or (ii) the closing of a Strategic Transaction, as defined in the Settlement Agreement. As of March 31, 2022, we recorded the payables due to REGENXBIO in the condensed consolidated balance sheets based on the present value of the remaining payments due to REGENXBIO under the Settlement Agreement using an interest rate of 9.6 %. The current portion of the payable due in November 2022 is $ 4.7 million and the long-term portion due in November 2024 is $ 3.9 million as of March 31, 2022. As of March 31, 2022, we have recorded $ 5.0 million of restricted cash in the balance sheet that serves as collateral for the payment owed to REGENXBIO in November 2022. |
FAIR VALUE MEASUREMENTS
FAIR VALUE MEASUREMENTS | 3 Months Ended |
Mar. 31, 2022 | |
Fair Value Disclosures [Abstract] | |
FAIR VALUE MEASUREMENTS | NOTE 6 – FAIR VALUE MEASUREMENTS We calculate the fair value of our assets and liabilities that qualify as financial instruments and include additional information in the notes to the consolidated financial statements when the fair value is different than the carrying value of these financial instruments. The estimated fair value of accounts receivable, prepaid expenses and other current assets, other assets, accounts payable, accrued expenses, loan payable, payable to licensor and deferred revenue approximate their carrying amounts due to the relatively short maturity of these instruments. U.S. GAAP defines 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 at the measurement date. This guidance establishes a three-level fair value hierarchy that prioritizes the inputs used to measure fair value. The hierarchy requires entities to maximize the use of observable inputs and minimize the use of unobservable inputs. The three levels of inputs used to measure fair value are as follows: ● Level 1 - Quoted prices in active markets for identical assets or liabilities. ● Level 2 - Observable inputs other than quoted prices included in Level 1, such as quoted prices for similar assets and liabilities in active markets; quoted prices for identical or similar assets and liabilities in markets that are not active; or other inputs that are observable or can be corroborated by observable market data. ● Level 3 - Unobservable inputs that are supported by little or no market activity and that are significant to the fair value of the assets and liabilities. This includes certain pricing models, discounted cash flow methodologies and similar valuation techniques that use significant unobservable inputs. We have segregated all financial assets and liabilities that are measured at fair value on a recurring basis (at least annually) into the most appropriate level within the fair value hierarchy based on the inputs used to determine the fair value at the measurement date in the table below. Financial assets measured at fair value on a recurring and non-recurring basis as of March 31, 2022 and December 31, 2021 are summarized below: SCHEDULE OF FAIR VALUE, ASSETS AND LIABILITIES MEASURED ON RECURRING AND NON-RECURRING BASIS (in thousands) Description Fair Value at Level 1 Level 2 Level 3 Recurring Assets: Cash equivalents Money market fund $ 16,694 $ 16,694 $ - $ - Short-term investments U.S. treasury securities 10,989 - 10,989 - Total assets measured at fair value $ 27,683 $ 16,694 $ 10,989 $ - Description Fair Value at Level 1 Level 2 Level 3 Recurring Assets: Cash equivalents Money market fund $ 28,590 $ 28,590 $ - $ - Cash equivalents fair value $ 28,590 $ 28,590 $ - $ - Short-term investments U.S. treasury securities 12,086 - 12,086 - Short-term investments fair value 12,086 - 12,086 - Total recurring assets 40,676 28,590 12,086 - Non-recurring Assets Licensed technology, net $ 1,384 $ - $ - $ 1,384 Total assets measured at fair value $ 42,060 $ 28,590 $ 12,086 $ 1,384 |
ACCRUED EXPENSES
ACCRUED EXPENSES | 3 Months Ended |
Mar. 31, 2022 | |
Accrued Expenses | |
ACCRUED EXPENSES | NOTE 7 – ACCRUED EXPENSES Accrued expenses consisted of the following as of: SCHEDULE OF ACCRUED EXPENSES (in thousands) March 31, December 31, Accrued employee compensation $ 745 $ 1,794 Accrued contracted services and other 3,461 3,091 Accrued sublicense fee owed to licensor - 700 Accrued expenses $ 4,206 $ 5,585 |
LEASES
LEASES | 3 Months Ended |
Mar. 31, 2022 | |
Leases | |
LEASES | NOTE 8 – LEASES We lease space under operating leases for manufacturing and laboratory facilities in Cleveland, Ohio, as well as administrative offices in New York, New York. We also lease office space in Madrid, Spain as well as certain office equipment under operating leases, which have a non-cancelable lease term of less than one year and, therefore, we have elected the practical expedient to exclude these short-term leases from our right-of-use assets and lease liabilities. On March 31, 2022, the Company announced that we were pursuing a strategic partner to take over development activities of ABO-102 and that we were discontinuing development of ABO-101. As a result of this shift in priorities, the Company determined the portion of the lease which was dedicated to the future facility for the ABO-101 and ABO-102 programs, had no future value and thus, we recorded an impairment charge of $ 1.6 Components of lease cost are as follows: SCHEDULE OF COMPONENTS OF LEASE COST (in thousands) 2022 2021 For the three months ended March 31, (in thousands) 2022 2021 Operating lease cost $ 472 $ 434 Variable lease cost $ 96 $ 135 Short-term lease cost $ 21 $ 5 Maturities of the Company’s operating lease liabilities, which do not include short-term leases, as of March 31, 2022 are as follows: SCHEDULE OF MATURITIES OF OPERATING LEASE LIABILITIES Maturity of lease liabilities: (in thousands) 2022, remainder $ 1,364 2023 1,834 2024 1,879 2025 1,896 2026 871 Thereafter 3,662 Total undiscounted operating lease payments 11,506 Less: imputed interest 2,411 Present value of operating lease liabilities $ 9,095 The weighted-average remaining term of the Company’s operating leases was 84 7.3 |
STOCK-BASED COMPENSATION
STOCK-BASED COMPENSATION | 3 Months Ended |
Mar. 31, 2022 | |
Share-Based Payment Arrangement [Abstract] | |
STOCK-BASED COMPENSATION | NOTE 9 – STOCK-BASED COMPENSATION We have two stock-based compensation plans: (1) Abeona Therapeutics Inc. 2015 Equity Incentive Plan (the “2015 Incentive Plan”), which was approved by stockholders on May 7, 2015 and last amended on May 20, 2020 and (2) Abeona Therapeutics Inc. 2005 Equity Incentive Plan (the “2005 Inventive Plan”), under which no further grants can be made. The following table summarizes stock-based compensation expense for the three months ended March 31, 2022 and 2021: SCHEDULE OF STOCK BASED COMPENSATION (in thousands) 2022 2021 For the three months ended March 31, (in thousands) 2022 2021 Research and development $ 372 $ 1,155 General and administrative 490 795 Stock based compensation expense $ 862 $ 1,950 Stock Options: ● Expected volatility - we estimate the volatility of our share price at the date of grant using a “look-back” period which coincides with the expected term, defined below. We believe using a “look-back” period which coincides with the expected term is the most appropriate measure for determining expected volatility. ● Expected term - we estimate the expected term using the “simplified” method, as outlined in Staff Accounting Bulletin No. 107, “Share-Based Payment.” ● Risk-free interest rate - we estimate the risk-free interest rate using the U.S. Treasury yield curve for periods equal to the expected term of the options in effect at the time of grant. ● Dividends - we use an expected dividend yield of zero because we have not declared or paid a cash dividend, nor do we have any plans to declare a dividend. The Company estimated the fair value of stock options granted in the periods presented utilizing a Black-Scholes option-valuation model utilizing the following assumptions: SCHEDULE OF WEIGHTED-AVERAGE ASSUMPTIONS TO ESTIMATE THE FAIR VALUE OF THE OPTIONS GRANTED 2022 2021 For the three months ended March 31, 2022 2021 Expected volatility 95 % 99 % Expected term 6.08 6.08 Risk-free interest rate 1.73 % 1.00 % Expected dividend yield 0 % 0 % The following table summarizes stock option activity for the 2015 Incentive Plan during the three months ended March 31, 2022 : SCHEDULE OF STOCK OPTIONS ACTIVITY Number of Options Weighted Average Exercise Price Weighted Average Remaining Contractual Term (years) Aggregate Intrinsic Value (in thousands) Outstanding at December 31, 2021 7,854,851 $ 1.54 7.63 $ - Granted 104,000 $ 0.26 - $ - Cancelled/forfeited (937,048 ) $ 1.40 - $ - Exercised - $ - - $ - Outstanding at March 31, 2022 7,021,803 $ 1.54 7.24 $ 6 Exercisable 3,516,716 $ 1.51 5.49 $ - Unvested 3,505,087 $ 1.57 8.98 $ 6 The aggregate intrinsic value of options is calculated as the difference between the exercise price of the underlying options and the fair value of the Company’s common stock for those options that had exercise prices lower than the fair value of the Company’s common stock. As of March 31, 2022, the total compensation cost related to non-vested option awards not yet recognized is approximately $ 5.0 2.6 The following table summarizes stock option activity for the 2005 Incentive Plan during the three months ended March 31, 2022 : SCHEDULE OF STOCK OPTIONS ACTIVITY Number of Options Weighted Average Exercise Price Weighted Average Remaining Contractual Term (years) Aggregate Intrinsic Value (in thousands) Outstanding at December 31, 2021 80,000 $ 1.28 1.80 $ Cancelled/forfeited - $ - - $ - Exercised - $ - - $ - Outstanding at March 31, 2022 80,000 $ 1.28 1.54 $ - Exercisable 80,000 $ 1.28 1.54 $ - Unvested - $ - - $ - Restricted Stock The following table summarizes restricted stock award activity during the three months ended March 31, 2022: SCHEDULE OF RESTRICTED STOCK AWARD ACTIVITY Number of Awards Weighted Average Grant Date Fair Value Outstanding at December 31, 2021 2,431,515 $ 1.86 Granted 252,000 $ 0.28 Cancelled/forfeited (377,523 ) $ 1.58 Vested (357,658 ) $ 2.31 Outstanding at March 31, 2022 1,948,334 $ 1.63 As of March 31, 2022, there is approximately $ 2.8 2.7 |
SUBSEQUENT EVENTS
SUBSEQUENT EVENTS | 3 Months Ended |
Mar. 31, 2022 | |
Subsequent Events [Abstract] | |
SUBSEQUENT EVENTS | NOTE 10 – SUBSEQUENT EVENTS On April 29, 2022, the Company, entered into a Securities Purchase Agreement (the “Purchase Agreement”) with certain institutional investors (the “Investors”), pursuant to which the Company agreed to issue and sell, in a private placement (the “Offering”), 1,000,006 0.01 250,005 0.01 19.00 5 20.00 25.0 0.45 0.01 The Company intends to call a special meeting of stockholders to consider an amendment (the “Amendment”) to the Company’s Restated Certificate of Incorporation (the “Charter”), to effect a reverse stock split of the outstanding shares of Common Stock by a ratio to be determined by the Board of Directors of the Company within a range to be specified in the proposal put to the stockholders for approval of the Amendment (the “Reverse Stock Split”). The Investors have agreed in the Purchase Agreement to not transfer, offer, sell, contract to sell, hypothecate, pledge or otherwise dispose of the shares of the Preferred Stock until the Reverse Stock Split, to vote the shares of the Series A Preferred Stock purchased in the Offering in favor of such Amendment and to vote the shares of the Series B Preferred Stock purchased in the Offering in a manner that “mirrors” the proportions on which the shares of Common Stock (excluding any shares of Common Stock that are not voted) and Series A Preferred Stock are voted on the Reverse Stock Split. The Reverse Stock Split requires the approval of the majority of the votes associated with our outstanding stock entitled to vote on the proposal. Because the Series B Preferred Stock will automatically and without further action of the purchaser be voted in a manner that “mirrors” the proportions on which the shares of Common Stock (excluding any shares of Common Stock that are not voted) and Series A Preferred Stock are voted on the Reverse Stock Split, abstentions by common stockholders will not have any effect on the votes cast by the holders of the Series B Preferred Stock. |
NATURE OF OPERATIONS AND SIGN_2
NATURE OF OPERATIONS AND SIGNIFICANT ACCOUNTING POLICIES (Policies) | 3 Months Ended |
Mar. 31, 2022 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
Background | Background Abeona Therapeutics Inc. (together with our subsidiaries, “we,” “our,” “Abeona” or the “Company”), a Delaware corporation, is a clinical-stage biopharmaceutical company developing gene and cell therapies for life-threatening rare genetic diseases. Our lead clinical program is EB-101, an autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa (“RDEB”), which is currently in the pivotal Phase 3 VIITAL™ clinical trial. Following a comprehensive portfolio review in early 2022, we have decided to focus our research and development resources on the VIITAL™ readout while actively pursuing a potential commercialization partner for EB-101 with the objective of reducing operating expenses and extending our cash runway. As part of this portfolio prioritization, we have intensified our pursuit of a strategic partnership to take over development activities for our adeno-associated virus (“AAV”)-based gene therapy ABO-102 for Sanfilippo syndrome type A (“MPS IIIA”) and we have discontinued development of our AAV-based gene therapy ABO-101 for Sanfilippo syndrome type B (“MPS IIIB”). We plan to continue development of AAV-based gene therapies designed to treat ophthalmic and other diseases and next-generation AAV-based gene therapies using the novel AIM™ capsid platform that we have exclusively licensed from the University of North Carolina at Chapel Hill (“UNC”), and internal AAV vector research programs. |
Basis of Presentation | Basis of Presentation The Company’s unaudited interim condensed consolidated financial statements have been prepared in conformity with accounting principles generally accepted in the United States of America (“U.S. GAAP”). All intercompany balances and transactions have been eliminated in consolidation. In the opinion of management, all adjustments, consisting only of normal recurring adjustments, except as otherwise disclosed, necessary for the fair presentation of the financial position, results of operations, and changes in financial position for such periods, have been made. These unaudited interim condensed financial statement results are not necessarily indicative of results to be expected for the full fiscal year or any future period. Certain information that is normally required by U.S. GAAP has been condensed or omitted in accordance with rules and regulations of the U.S. Securities and Exchange Commission (“SEC”). Therefore, these unaudited interim condensed consolidated financial statements should be read in conjunction with the audited consolidated financial statements and notes thereto included in our Annual Report on Form 10-K for the year ended December 31, 2021, which was filed with the SEC on March 31, 2022. |
Uses and Sources of Liquidity | Uses and Sources of Liquidity The unaudited interim condensed consolidated financial statements have been prepared on the going concern basis, which assumes the Company will have sufficient cash to pay its operating expenses, as and when they become payable, for a period of at least 12 months from the date the financial report is issued. As of March 31, 2022, we had cash, cash equivalents, restricted cash and short-term investments of $ 37.2 13.7 We are subject to a number of risks similar to other life science companies, including, but not limited to, risks related to the successful discovery and development of product candidates, obtaining the necessary regulatory approval to market our product candidates, raising additional capital to continue to fund our operations, development of competing drugs and therapies, protection of proprietary technology and market acceptance of our products. As a result of these and other risks and the related uncertainties, there can be no assurance of our future success. Following a comprehensive portfolio review in early 2022, we have decided to focus our research and development resources on the EB-101 program with the objective of reducing operating expenses and extending our cash runway. As part of this portfolio prioritization, we have intensified our pursuit of a strategic partnership to take over development activities for our AAV-based gene therapy ABO-102 for MPS IIIA and we have discontinued development of our AAV-based gene therapy ABO-101 for MPS IIIB. Based upon these current operating plans, our ability to access additional financial resources and/or our financial flexibility to further reduce operating expenses if required, we believe that we have sufficient resources to fund operations through at least the next 12 months from the date of this Quarterly Report on Form 10-Q. We will need to secure additional funding beyond the next 12 months to carry out all of our planned research and development activities. If we are unable to obtain additional financing or generate license or product revenue, the lack of liquidity and sufficient capital resources could have a material adverse effect on our future prospects. |
Use of Estimates | Use of Estimates The preparation of unaudited interim condensed consolidated financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amount of assets and disclosure of contingent assets and liabilities at the date of the unaudited interim condensed consolidated financial statements and the reported amounts of revenue and expenses during the reported period. Actual results could differ from these estimates and assumptions. |
Summary of Significant Accounting Policies | Summary of Significant Accounting Policies There have been no new or material changes to the significant accounting policies discussed in the Company’s Annual Report on Form 10-K for the year ended December 31, 2021 that are of significance, or potential significance, to the Company. |
Reclassifications | Reclassifications Certain comparative figures have been reclassified to conform to the current year presentation. The Company reclassified depreciation and amortization costs of $ 0.8 35,000 0.3 5.0 0.9 |
Net Loss Per Share | Net Loss Per Share Basic and diluted net loss per share is computed by dividing net loss by the weighted-average number of shares of common stock. We do not include the potential impact of dilutive securities in diluted net loss per share, as the impact of these items is anti-dilutive. Potential dilutive securities result from outstanding restricted stock, stock options, and stock purchase warrants. The following table sets forth the potential securities that could potentially dilute basic income/(loss) per share in the future that were not included in the computation of diluted net loss per share because to do so would have been anti-dilutive for the periods presented: SCHEDULE OF ANTI-DILUTIVE SECURITIES EXCLUDED FROM COMPUTATION OF EARNINGS PER SHARE 2022 2021 For the three months ended March 31, 2022 2021 Stock options 7,101,803 7,091,879 Restricted stock 1,948,334 2,636,216 Warrants 44,700,000 - Total 53,750,137 9,798,095 |
NATURE OF OPERATIONS AND SIGN_3
NATURE OF OPERATIONS AND SIGNIFICANT ACCOUNTING POLICIES (Tables) | 3 Months Ended |
Mar. 31, 2022 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
SCHEDULE OF ANTI-DILUTIVE SECURITIES EXCLUDED FROM COMPUTATION OF EARNINGS PER SHARE | The following table sets forth the potential securities that could potentially dilute basic income/(loss) per share in the future that were not included in the computation of diluted net loss per share because to do so would have been anti-dilutive for the periods presented: SCHEDULE OF ANTI-DILUTIVE SECURITIES EXCLUDED FROM COMPUTATION OF EARNINGS PER SHARE 2022 2021 For the three months ended March 31, 2022 2021 Stock options 7,101,803 7,091,879 Restricted stock 1,948,334 2,636,216 Warrants 44,700,000 - Total 53,750,137 9,798,095 |
SHORT-TERM INVESTMENTS (Tables)
SHORT-TERM INVESTMENTS (Tables) | 3 Months Ended |
Mar. 31, 2022 | |
Investments, Debt and Equity Securities [Abstract] | |
SCHEDULE OF AVAILABLE FOR SALE SHORT-TERM INVESTMENTS | Short-term investments consisted of the following marketable securities as of: SCHEDULE OF AVAILABLE FOR SALE SHORT-TERM INVESTMENTS (in thousands) March 31, 2022 Amortized Cost Gross Unrealized Gain Gross Unrealized Loss Fair Value Available-for-sale, short-term investments U.S. treasury securities $ 10,986 $ 3 $ - $ 10,989 Total $ 10,986 $ 3 $ - $ 10,989 December 31, 2021 Amortized Cost Gross Unrealized Gain Gross Unrealized Loss Fair Value Available-for-sale, short-term investments U.S. treasury securities $ 12,077 $ 9 $ - $ 12,086 Total $ 12,077 $ 9 $ - $ 12,086 |
PROPERTY AND EQUIPMENT, NET (Ta
PROPERTY AND EQUIPMENT, NET (Tables) | 3 Months Ended |
Mar. 31, 2022 | |
Property, Plant and Equipment [Abstract] | |
SCHEDULE OF PROPERTY AND EQUIPMENT | Property and equipment are stated at cost and depreciated or amortized using the straight-line method based on useful lives as follow: SCHEDULE OF PROPERTY AND EQUIPMENT (in thousands) Useful lives (years) March 31, December 31, Laboratory equipment 5 $ 9,138 $ 9,081 Furniture, software and office equipment 3 5 1,908 1,896 Leasehold improvements Shorter of remaining lease term or useful life 8,603 8,603 Construction-in-progress 3,252 3,219 Subtotal 22,901 22,799 Less: accumulated depreciation (11,241 ) (10,460 ) Less: construction-in-progress impairment (3,252 ) - Property and equipment, net $ 8,408 $ 12,339 |
LICENSED TECHNOLOGY (Tables)
LICENSED TECHNOLOGY (Tables) | 3 Months Ended |
Mar. 31, 2022 | |
Goodwill and Intangible Assets Disclosure [Abstract] | |
SCHEDULE OF LICENSED TECHNOLOGY | Licensed technology consists of the following: SCHEDULE OF LICENSED TECHNOLOGY (in thousands) March 31, December 31, Licensed technology $ 2,156 $ 2,156 Less accumulated amortization (801 ) (772 ) Less impairment charge (1,355 ) - Licensed technology, net $ - $ 1,384 |
FAIR VALUE MEASUREMENTS (Tables
FAIR VALUE MEASUREMENTS (Tables) | 3 Months Ended |
Mar. 31, 2022 | |
Fair Value Disclosures [Abstract] | |
SCHEDULE OF FAIR VALUE, ASSETS AND LIABILITIES MEASURED ON RECURRING AND NON-RECURRING BASIS | Financial assets measured at fair value on a recurring and non-recurring basis as of March 31, 2022 and December 31, 2021 are summarized below: SCHEDULE OF FAIR VALUE, ASSETS AND LIABILITIES MEASURED ON RECURRING AND NON-RECURRING BASIS (in thousands) Description Fair Value at Level 1 Level 2 Level 3 Recurring Assets: Cash equivalents Money market fund $ 16,694 $ 16,694 $ - $ - Short-term investments U.S. treasury securities 10,989 - 10,989 - Total assets measured at fair value $ 27,683 $ 16,694 $ 10,989 $ - Description Fair Value at Level 1 Level 2 Level 3 Recurring Assets: Cash equivalents Money market fund $ 28,590 $ 28,590 $ - $ - Cash equivalents fair value $ 28,590 $ 28,590 $ - $ - Short-term investments U.S. treasury securities 12,086 - 12,086 - Short-term investments fair value 12,086 - 12,086 - Total recurring assets 40,676 28,590 12,086 - Non-recurring Assets Licensed technology, net $ 1,384 $ - $ - $ 1,384 Total assets measured at fair value $ 42,060 $ 28,590 $ 12,086 $ 1,384 |
ACCRUED EXPENSES (Tables)
ACCRUED EXPENSES (Tables) | 3 Months Ended |
Mar. 31, 2022 | |
Accrued Expenses | |
SCHEDULE OF ACCRUED EXPENSES | Accrued expenses consisted of the following as of: SCHEDULE OF ACCRUED EXPENSES (in thousands) March 31, December 31, Accrued employee compensation $ 745 $ 1,794 Accrued contracted services and other 3,461 3,091 Accrued sublicense fee owed to licensor - 700 Accrued expenses $ 4,206 $ 5,585 |
LEASES (Tables)
LEASES (Tables) | 3 Months Ended |
Mar. 31, 2022 | |
Leases | |
SCHEDULE OF COMPONENTS OF LEASE COST | Components of lease cost are as follows: SCHEDULE OF COMPONENTS OF LEASE COST (in thousands) 2022 2021 For the three months ended March 31, (in thousands) 2022 2021 Operating lease cost $ 472 $ 434 Variable lease cost $ 96 $ 135 Short-term lease cost $ 21 $ 5 |
SCHEDULE OF MATURITIES OF OPERATING LEASE LIABILITIES | Maturities of the Company’s operating lease liabilities, which do not include short-term leases, as of March 31, 2022 are as follows: SCHEDULE OF MATURITIES OF OPERATING LEASE LIABILITIES Maturity of lease liabilities: (in thousands) 2022, remainder $ 1,364 2023 1,834 2024 1,879 2025 1,896 2026 871 Thereafter 3,662 Total undiscounted operating lease payments 11,506 Less: imputed interest 2,411 Present value of operating lease liabilities $ 9,095 |
STOCK-BASED COMPENSATION (Table
STOCK-BASED COMPENSATION (Tables) | 3 Months Ended |
Mar. 31, 2022 | |
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items] | |
SCHEDULE OF STOCK BASED COMPENSATION | The following table summarizes stock-based compensation expense for the three months ended March 31, 2022 and 2021: SCHEDULE OF STOCK BASED COMPENSATION (in thousands) 2022 2021 For the three months ended March 31, (in thousands) 2022 2021 Research and development $ 372 $ 1,155 General and administrative 490 795 Stock based compensation expense $ 862 $ 1,950 |
SCHEDULE OF WEIGHTED-AVERAGE ASSUMPTIONS TO ESTIMATE THE FAIR VALUE OF THE OPTIONS GRANTED | The Company estimated the fair value of stock options granted in the periods presented utilizing a Black-Scholes option-valuation model utilizing the following assumptions: SCHEDULE OF WEIGHTED-AVERAGE ASSUMPTIONS TO ESTIMATE THE FAIR VALUE OF THE OPTIONS GRANTED 2022 2021 For the three months ended March 31, 2022 2021 Expected volatility 95 % 99 % Expected term 6.08 6.08 Risk-free interest rate 1.73 % 1.00 % Expected dividend yield 0 % 0 % |
SCHEDULE OF RESTRICTED STOCK AWARD ACTIVITY | The following table summarizes restricted stock award activity during the three months ended March 31, 2022: SCHEDULE OF RESTRICTED STOCK AWARD ACTIVITY Number of Awards Weighted Average Grant Date Fair Value Outstanding at December 31, 2021 2,431,515 $ 1.86 Granted 252,000 $ 0.28 Cancelled/forfeited (377,523 ) $ 1.58 Vested (357,658 ) $ 2.31 Outstanding at March 31, 2022 1,948,334 $ 1.63 |
2015 Equity Incentive Plan [Member] | |
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items] | |
SCHEDULE OF STOCK OPTIONS ACTIVITY | The following table summarizes stock option activity for the 2015 Incentive Plan during the three months ended March 31, 2022 : SCHEDULE OF STOCK OPTIONS ACTIVITY Number of Options Weighted Average Exercise Price Weighted Average Remaining Contractual Term (years) Aggregate Intrinsic Value (in thousands) Outstanding at December 31, 2021 7,854,851 $ 1.54 7.63 $ - Granted 104,000 $ 0.26 - $ - Cancelled/forfeited (937,048 ) $ 1.40 - $ - Exercised - $ - - $ - Outstanding at March 31, 2022 7,021,803 $ 1.54 7.24 $ 6 Exercisable 3,516,716 $ 1.51 5.49 $ - Unvested 3,505,087 $ 1.57 8.98 $ 6 |
2005 Equity Incentive Plan [Member] | |
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items] | |
SCHEDULE OF STOCK OPTIONS ACTIVITY | The following table summarizes stock option activity for the 2005 Incentive Plan during the three months ended March 31, 2022 : SCHEDULE OF STOCK OPTIONS ACTIVITY Number of Options Weighted Average Exercise Price Weighted Average Remaining Contractual Term (years) Aggregate Intrinsic Value (in thousands) Outstanding at December 31, 2021 80,000 $ 1.28 1.80 $ Cancelled/forfeited - $ - - $ - Exercised - $ - - $ - Outstanding at March 31, 2022 80,000 $ 1.28 1.54 $ - Exercisable 80,000 $ 1.28 1.54 $ - Unvested - $ - - $ - |
SCHEDULE OF ANTI-DILUTIVE SECUR
SCHEDULE OF ANTI-DILUTIVE SECURITIES EXCLUDED FROM COMPUTATION OF EARNINGS PER SHARE (Details) - shares | 3 Months Ended | |
Mar. 31, 2022 | Mar. 31, 2021 | |
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Total | 53,750,137 | 9,798,095 |
Stock Options [Member] | ||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Total | 7,101,803 | 7,091,879 |
Restricted Stock [Member] | ||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Total | 1,948,334 | 2,636,216 |
Warrant [Member] | ||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Total | 44,700,000 |
NATURE OF OPERATIONS AND SIGN_4
NATURE OF OPERATIONS AND SIGNIFICANT ACCOUNTING POLICIES (Details Narrative) - USD ($) | 3 Months Ended | ||
Mar. 31, 2022 | Mar. 31, 2021 | Dec. 31, 2021 | |
Cash, cash equivalents, restricted cash and short-term investments | $ 37,200,000 | ||
Cash flow from operating activities | 13,687,000 | $ 13,585,000 | |
Depreciation and amortization costs | 811,000 | $ 817,000 | |
Restricted cash | 5,891,000 | $ 5,891,000 | |
General and Administrative to Research and Development Expenses [Member] | |||
Reclassification of Rent expense | 300,000 | ||
Prepaid Expenses, Other Current Assets to Restricted Cash [Member] | |||
Restricted cash | 5,000,000 | ||
Other Assets and Restricted Cash to Restricted Cash [Member] | |||
Restricted cash | 900,000 | ||
Research and Development Expense [Member] | |||
Depreciation and amortization costs | 800,000 | ||
General and Administrative Expense [Member] | |||
Depreciation and amortization costs | $ 35,000 |
SCHEDULE OF AVAILABLE FOR SALE
SCHEDULE OF AVAILABLE FOR SALE SHORT-TERM INVESTMENTS (Details) - USD ($) $ in Thousands | 3 Months Ended | 12 Months Ended |
Mar. 31, 2022 | Dec. 31, 2021 | |
Marketable Securities [Line Items] | ||
Amortized cost | $ 10,986 | $ 12,077 |
Gross Unrealized Gain | 3 | 9 |
Gross Unrealized Loss | ||
Fair Value | 10,989 | 12,086 |
US Treasury Securities [Member] | ||
Marketable Securities [Line Items] | ||
Amortized cost | 10,986 | 12,077 |
Gross Unrealized Gain | 3 | 9 |
Gross Unrealized Loss | ||
Fair Value | $ 10,989 | $ 12,086 |
SHORT-TERM INVESTMENTS (Details
SHORT-TERM INVESTMENTS (Details Narrative) - USD ($) | 3 Months Ended | |
Mar. 31, 2022 | Mar. 31, 2021 | |
Investments, Debt and Equity Securities [Abstract] | ||
Available-for-sale securities realized gain loss | $ 0 | $ 0 |
SCHEDULE OF PROPERTY AND EQUIPM
SCHEDULE OF PROPERTY AND EQUIPMENT (Details) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2022 | Dec. 31, 2021 | |
Property, Plant and Equipment [Line Items] | ||
Subtotal | $ 22,901 | $ 22,799 |
Less: accumulated depreciation | (11,241) | (10,460) |
Less: construction-in-progress impairment | (3,252) | |
Property and equipment, net | 8,408 | 12,339 |
Laboratory Equipment [Member] | ||
Property, Plant and Equipment [Line Items] | ||
Subtotal | $ 9,138 | 9,081 |
Useful lives (years) | 5 years | |
Furniture, Software and Office Equipment [Member] | ||
Property, Plant and Equipment [Line Items] | ||
Subtotal | $ 1,908 | 1,896 |
Furniture, Software and Office Equipment [Member] | Minimum [Member] | ||
Property, Plant and Equipment [Line Items] | ||
Useful lives (years) | 3 years | |
Furniture, Software and Office Equipment [Member] | Maximum [Member] | ||
Property, Plant and Equipment [Line Items] | ||
Useful lives (years) | 5 years | |
Leasehold Improvements [Member] | ||
Property, Plant and Equipment [Line Items] | ||
Subtotal | $ 8,603 | 8,603 |
Useful lives | Shorter of remaining lease term or useful life | |
Construction in Progress [Member] | ||
Property, Plant and Equipment [Line Items] | ||
Subtotal | $ 3,252 | $ 3,219 |
PROPERTY AND EQUIPMENT, NET (De
PROPERTY AND EQUIPMENT, NET (Details Narrative) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2022 | Mar. 31, 2021 | |
Property, Plant and Equipment [Line Items] | ||
Depreciation expense | $ 800 | $ 800 |
Impairment of Ongoing Project | 3,252 | |
Construction in Progress [Member] | ||
Property, Plant and Equipment [Line Items] | ||
Impairment of Ongoing Project | $ 3,300 |
SCHEDULE OF LICENSED TECHNOLOGY
SCHEDULE OF LICENSED TECHNOLOGY (Details) - USD ($) $ in Thousands | Mar. 31, 2022 | Dec. 31, 2021 |
Goodwill and Intangible Assets Disclosure [Abstract] | ||
Licensed technology | $ 2,156 | $ 2,156 |
Less accumulated amortization | (801) | (772) |
Less impairment charge | (1,355) | |
Licensed technology, net | $ 1,384 |
LICENSED TECHNOLOGY (Details Na
LICENSED TECHNOLOGY (Details Narrative) - USD ($) | May 15, 2015 | Mar. 31, 2022 | Mar. 31, 2021 |
Finite-Lived Intangible Assets [Line Items] | |||
Impairment of Intangible Assets, Finite-Lived | $ 1,355,000 | ||
Amortization of licensed technology | $ 29,000 | $ 29,000 | |
License [Member] | |||
Finite-Lived Intangible Assets [Line Items] | |||
Finite-Lived Intangible Asset, Useful Life | 20 years |
SETTLEMENT LIABILITY (Details N
SETTLEMENT LIABILITY (Details Narrative) - USD ($) $ in Thousands | Nov. 12, 2021 | Mar. 31, 2022 | Dec. 31, 2021 |
Defined Benefit Plan Disclosure [Line Items] | |||
Restricted Cash | $ 5,891 | $ 5,891 | |
REGENXBIO [Member] | |||
Defined Benefit Plan Disclosure [Line Items] | |||
Settlement Liabilities, Current | $ 30,000 | ||
Loss Contingency, Settlement Agreement, Terms | (1) $20.0 million paid in November 2021 after execution of the Settlement Agreement, (2) $5.0 million on the first anniversary of the effective date of the Settlement Agreement, and (3) $5.0 million upon the earlier of: (i) the third anniversary of the effective date of the Settlement Agreement or (ii) the closing of a Strategic Transaction, as defined in the Settlement Agreement. | ||
Debt Instrument, Interest Rate, Stated Percentage | 9.60% | ||
Long-Term Debt, Current Maturities | $ 4,700 | ||
Long-Term Debt, Excluding Current Maturities | 3,900 | ||
Restricted Cash | $ 5,000 |
SCHEDULE OF FAIR VALUE, ASSETS
SCHEDULE OF FAIR VALUE, ASSETS AND LIABILITIES MEASURED ON RECURRING AND NON-RECURRING BASIS (Details) - USD ($) $ in Thousands | Mar. 31, 2022 | Dec. 31, 2021 |
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total assets measured at fair value | $ 42,060 | |
Fair Value, Inputs, Level 1 [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total assets measured at fair value | 28,590 | |
Fair Value, Inputs, Level 2 [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total assets measured at fair value | 12,086 | |
Fair Value, Inputs, Level 3 [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total assets measured at fair value | 1,384 | |
Fair Value, Recurring [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total assets measured at fair value | $ 27,683 | 40,676 |
Fair Value, Recurring [Member] | Fair Value, Inputs, Level 1 [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total assets measured at fair value | 16,694 | 28,590 |
Fair Value, Recurring [Member] | Fair Value, Inputs, Level 2 [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total assets measured at fair value | 10,989 | 12,086 |
Fair Value, Recurring [Member] | Fair Value, Inputs, Level 3 [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total assets measured at fair value | ||
Fair Value, Recurring [Member] | Money Market Funds [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Cash equivalents fair value | 16,694 | 28,590 |
Fair Value, Recurring [Member] | Money Market Funds [Member] | Fair Value, Inputs, Level 1 [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Cash equivalents fair value | 16,694 | 28,590 |
Fair Value, Recurring [Member] | Money Market Funds [Member] | Fair Value, Inputs, Level 2 [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Cash equivalents fair value | ||
Fair Value, Recurring [Member] | Money Market Funds [Member] | Fair Value, Inputs, Level 3 [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Cash equivalents fair value | ||
Fair Value, Recurring [Member] | US Treasury Securities [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Short-term investments fair value | 10,989 | 12,086 |
Fair Value, Recurring [Member] | US Treasury Securities [Member] | Fair Value, Inputs, Level 1 [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Short-term investments fair value | ||
Fair Value, Recurring [Member] | US Treasury Securities [Member] | Fair Value, Inputs, Level 2 [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Short-term investments fair value | 10,989 | 12,086 |
Fair Value, Recurring [Member] | US Treasury Securities [Member] | Fair Value, Inputs, Level 3 [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Short-term investments fair value | ||
Fair Value, Nonrecurring [Member] | Licensed Technology Net [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Licensed technology, net | 1,384 | |
Fair Value, Nonrecurring [Member] | Licensed Technology Net [Member] | Fair Value, Inputs, Level 1 [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Licensed technology, net | ||
Fair Value, Nonrecurring [Member] | Licensed Technology Net [Member] | Fair Value, Inputs, Level 2 [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Licensed technology, net | ||
Fair Value, Nonrecurring [Member] | Licensed Technology Net [Member] | Fair Value, Inputs, Level 3 [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Licensed technology, net | $ 1,384 |
SCHEDULE OF ACCRUED EXPENSES (D
SCHEDULE OF ACCRUED EXPENSES (Details) - USD ($) $ in Thousands | Mar. 31, 2022 | Dec. 31, 2021 |
Accrued Expenses | ||
Accrued employee compensation | $ 745 | $ 1,794 |
Accrued contracted services and other | 3,461 | 3,091 |
Accrued sublicense fee owed to licensor | 700 | |
Accrued expenses | $ 4,206 | $ 5,585 |
SCHEDULE OF COMPONENTS OF LEASE
SCHEDULE OF COMPONENTS OF LEASE COST (Details) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2022 | Mar. 31, 2021 | |
Leases | ||
Operating lease cost | $ 472 | $ 434 |
Variable lease cost | 96 | 135 |
Short-term lease cost | $ 21 | $ 5 |
SCHEDULE OF MATURITIES OF OPERA
SCHEDULE OF MATURITIES OF OPERATING LEASE LIABILITIES (Details) $ in Thousands | Mar. 31, 2022USD ($) |
Leases | |
2022, remainder | $ 1,364 |
2023 | 1,834 |
2024 | 1,879 |
2025 | 1,896 |
2026 | 871 |
Thereafter | 3,662 |
Total undiscounted operating lease payments | 11,506 |
Less: imputed interest | 2,411 |
Present value of operating lease liabilities | $ 9,095 |
LEASES (Details Narrative)
LEASES (Details Narrative) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2022 | Mar. 31, 2021 | |
Leases | ||
Lease impairment charge | $ 1,561 | |
Weighted-average remaining lease term for operating leases | 84 months | |
Weighted-average discount rate for operating leases | 7.30% |
SCHEDULE OF STOCK BASED COMPENS
SCHEDULE OF STOCK BASED COMPENSATION (Details) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2022 | Mar. 31, 2021 | |
Share-Based Payment Arrangement, Expensed and Capitalized, Amount [Line Items] | ||
Stock based compensation expense | $ 862 | $ 1,950 |
Research and Development Expense [Member] | ||
Share-Based Payment Arrangement, Expensed and Capitalized, Amount [Line Items] | ||
Stock based compensation expense | 372 | 1,155 |
General and Administrative Expense [Member] | ||
Share-Based Payment Arrangement, Expensed and Capitalized, Amount [Line Items] | ||
Stock based compensation expense | $ 490 | $ 795 |
SCHEDULE OF WEIGHTED-AVERAGE AS
SCHEDULE OF WEIGHTED-AVERAGE ASSUMPTIONS TO ESTIMATE THE FAIR VALUE OF THE OPTIONS GRANTED (Details) | 3 Months Ended | |
Mar. 31, 2022 | Mar. 31, 2021 | |
Share-Based Payment Arrangement [Abstract] | ||
Expected volatility | 95.00% | 99.00% |
Expected term | 6 years 29 days | 6 years 29 days |
Risk-free interest rate | 1.73% | 1.00% |
Expected dividend yield | 0.00% | 0.00% |
SCHEDULE OF STOCK OPTIONS ACTIV
SCHEDULE OF STOCK OPTIONS ACTIVITY (Details) $ / shares in Units, $ in Thousands | 3 Months Ended |
Mar. 31, 2022USD ($)$ / sharesshares | |
2015 Equity Incentive Plan [Member] | |
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items] | |
Number of Options, Beginning balance | shares | 7,854,851 |
Weighted- average exercise price, Beginning balance | $ / shares | $ 1.54 |
Weighted-average Remaining Contractual Term (years), Outstanding | 7 years 7 months 17 days |
Beginning balance, Options, Aggregate Intrinsic Value | $ | |
Number of Options, Granted | shares | 104,000 |
Weighted- average exercise price, Granted | $ / shares | $ 0.26 |
Number of Options, Cancelled/forfeited | shares | (937,048) |
Weighted- average exercise price, Cancelled/forfeited | $ / shares | $ 1.40 |
Number of Options, Exercised | shares | |
Weighted- average exercise price, Exercised | $ / shares | |
Number of Options, Ending balance | shares | 7,021,803 |
Weighted- average exercise price, Ending balance | $ / shares | $ 1.54 |
Share-Based Compensation Arrangement by Share-Based Payment Award, Options, Outstanding, Weighted Average Remaining Contractual Term | 7 years 2 months 26 days |
Ending balance, Options, Aggregate Intrinsic Value | $ | $ 6 |
Number of Options, Exerciasble | shares | 3,516,716 |
Weighted- average exercise price, Exercisable | $ / shares | $ 1.51 |
Weighted-average Remaining Contractual Term (years), Exercisable | 5 years 5 months 26 days |
Aggregate Intrinsic Value, Exercisable | $ | |
Number of Options, Unvested options | shares | 3,505,087 |
Weighted- average exercise price, Unvested options | $ / shares | $ 1.57 |
Weighted-average Remaining Contractual Term (years), Unvested | 8 years 11 months 23 days |
Aggregate Intrinsic Value, Unvested | $ | $ 6 |
2005 Equity Incentive Plan [Member] | |
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items] | |
Number of Options, Beginning balance | shares | 80,000 |
Weighted- average exercise price, Beginning balance | $ / shares | $ 1.28 |
Weighted-average Remaining Contractual Term (years), Outstanding | 1 year 9 months 18 days |
Beginning balance, Options, Aggregate Intrinsic Value | $ | |
Number of Options, Cancelled/forfeited | shares | |
Weighted- average exercise price, Cancelled/forfeited | $ / shares | |
Number of Options, Exercised | shares | |
Weighted- average exercise price, Exercised | $ / shares | |
Number of Options, Ending balance | shares | 80,000 |
Weighted- average exercise price, Ending balance | $ / shares | $ 1.28 |
Share-Based Compensation Arrangement by Share-Based Payment Award, Options, Outstanding, Weighted Average Remaining Contractual Term | 1 year 6 months 14 days |
Ending balance, Options, Aggregate Intrinsic Value | $ | |
Number of Options, Exerciasble | shares | 80,000 |
Weighted- average exercise price, Exercisable | $ / shares | $ 1.28 |
Weighted-average Remaining Contractual Term (years), Exercisable | 1 year 6 months 14 days |
Aggregate Intrinsic Value, Exercisable | $ | |
Number of Options, Unvested options | shares | |
Weighted- average exercise price, Unvested options | $ / shares | |
Aggregate Intrinsic Value, Unvested | $ |
SCHEDULE OF RESTRICTED STOCK AW
SCHEDULE OF RESTRICTED STOCK AWARD ACTIVITY (Details) - Restricted Stock [Member] | 3 Months Ended |
Mar. 31, 2022$ / sharesshares | |
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items] | |
Restricted common stock awards, Beginning balance | shares | 2,431,515 |
Weighted-average grant date fair value, Beginning balance | $ / shares | $ 1.86 |
Restricted common stock awards, Granted | shares | 252,000 |
Weighted-average grant date fair value, Granted | $ / shares | $ 0.28 |
Restricted common stock awards, Cancelled/forfeited | shares | (377,523) |
Weighted-average grant date fair value, Forfeited | $ / shares | $ 1.58 |
Restricted common stock awards, Vested | shares | (357,658) |
Weighted-average grant date fair value, Vested | $ / shares | $ 2.31 |
Restricted common stock awards, Ending balance | shares | 1,948,334 |
Weighted-average grant date fair value, Ending balance | $ / shares | $ 1.63 |
STOCK-BASED COMPENSATION (Detai
STOCK-BASED COMPENSATION (Details Narrative) $ in Millions | 3 Months Ended |
Mar. 31, 2022USD ($) | |
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items] | |
Compensation cost related to restricted common stock not recognized | $ 2.8 |
Weighted average compensation costs related to restricted common stock | 2 years 8 months 12 days |
2015 Equity Incentive Plan [Member] | |
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items] | |
Compensation cost related to non-vested options not recognized | $ 5 |
Weighted average period total compensation costs related to non-vested options | 2 years 7 months 6 days |
SUBSEQUENT EVENTS (Details Narr
SUBSEQUENT EVENTS (Details Narrative) - USD ($) $ / shares in Units, $ in Millions | Apr. 29, 2022 | Mar. 31, 2022 | Dec. 31, 2021 |
Subsequent Event [Line Items] | |||
Stated value | $ 0.01 | $ 0.01 | |
Common stock par value | $ 0.01 | $ 0.01 | |
Subsequent Event [Member] | Securities Purchase Agreement [Member] | Private Placement [Member] | Preferred Stock [Member] | |||
Subsequent Event [Line Items] | |||
Offering price per share | $ 19 | ||
Original issue discount percentage | 5.00% | ||
Stated value | $ 20 | ||
Proceeds from private placement offering | $ 25 | ||
Conversion price | $ 0.45 | ||
Subsequent Event [Member] | Securities Purchase Agreement [Member] | Private Placement [Member] | Common Stock [Member] | |||
Subsequent Event [Line Items] | |||
Common stock par value | $ 0.01 | ||
Subsequent Event [Member] | Securities Purchase Agreement [Member] | Private Placement [Member] | Series A Preferred Stock [Member] | |||
Subsequent Event [Line Items] | |||
Shares issued | 1,000,006 | ||
Shares issued, per share price | $ 0.01 | ||
Subsequent Event [Member] | Securities Purchase Agreement [Member] | Private Placement [Member] | Series B Preferred Stock [Member] | |||
Subsequent Event [Line Items] | |||
Shares issued | 250,005 | ||
Shares issued, per share price | $ 0.01 |