PART I - FINANCIAL INFORMATION [Item 1. Financial Statements](index=8&type=section&id=Item%201.%20Financial%20Statements) Presents Abeona Therapeutics Inc.'s unaudited condensed consolidated financial statements for Q1 2024, including balance sheets, statements of operations, stockholders' equity, cash flows, and detailed notes [Unaudited Condensed Consolidated Balance Sheets](index=5&type=section&id=Unaudited%20Condensed%20Consolidated%20Balance%20Sheets%20as%20of%20March%2031%2C%202024%20and%20December%2031%2C%202023) Details the company's financial position, including assets, liabilities, and stockholders' equity, as of March 31, 2024, and December 31, 2023 - **Total assets increased from $64.0 million to $74.8 million**, driven by cash, cash equivalents, and short-term investments[9](index=9&type=chunk) - **Total liabilities significantly increased from $49.2 million to $83.7 million**, primarily due to new long-term debt, derivative liabilities, and a substantial rise in warrant liabilities[9](index=9&type=chunk) - **Total stockholders' equity shifted from a positive $14.8 million to a deficit of $(8.9) million**[9](index=9&type=chunk) Condensed Consolidated Balance Sheets (in thousands) | --- | --- | --- | | **ASSETS** | **March 31, 2024 (in thousands)** | **December 31, 2023 (in thousands)** | | Current assets: | | | | Cash and cash equivalents | $17,558 | $14,473 | | Short-term investments | 44,786 | 37,753 | | Restricted cash | 338 | 338 | | Other receivables | 2,232 | 2,444 | | Prepaid expenses and other current assets | 1,811 | 729 | | Total current assets | 66,725 | 55,737 | | Property and equipment, net | 3,767 | 3,533 | | Operating lease right-of-use assets | 4,222 | 4,455 | | Other assets | 114 | 277 | | **Total assets** | **$74,828** | **$64,002** | | **LIABILITIES AND STOCKHOLDERS' (DEFICIT) EQUITY** | | | | Current liabilities: | | | | Accounts payable | $3,362 | $1,858 | | Accrued expenses | 2,791 | 5,985 | | Current portion of operating lease liability | 1,044 | 998 | | Current portion of payable to licensor | 4,691 | 4,580 | | Other current liabilities | — | 1 | | Total current liabilities | 11,889 | 13,422 | | Long-term operating lease liabilities | 4,046 | 4,402 | | Long-term debt | 18,079 | — | | Derivative liabilities | 1,005 | — | | Warrant liabilities | 48,690 | 31,352 | | **Total liabilities** | **83,709** | **49,176** | | Stockholders' (deficit) equity: | | | | Common stock | 276 | 265 | | Additional paid-in capital | 772,129 | 764,151 | | Accumulated deficit | (781,102) | (749,524) | | Accumulated other comprehensive loss | (184) | (66) | | **Total stockholders' (deficit) equity** | **(8,881)** | **14,826** | | **Total liabilities and stockholders' equity** | **$74,828** | **$64,002** | [Unaudited Condensed Consolidated Statements of Operations and Comprehensive Loss](index=6&type=section&id=Unaudited%20Condensed%20Consolidated%20Statements%20of%20Operations%20and%20Comprehensive%20Loss%20for%20the%20three%20months%20ended%20March%2031%2C%202024%20and%202023) Presents the company's financial performance, including revenues, expenses, and net loss, for the three months ended March 31, 2024, and 2023 - **Net loss for Q1 2024 was $(31.6) million**, a significant increase from $(9.1) million in the prior year period[11](index=11&type=chunk) - This was primarily driven by a **$(17.3) million loss from the change in fair value of warrant and derivative liabilities**, increased general and administrative expenses (up **78% to $7.1 million**), and increased interest expense (up **843% to $1.0 million**)[11](index=11&type=chunk)[125](index=125&type=chunk) - **Research and development expenses decreased by 10% to $7.2 million**[11](index=11&type=chunk)[125](index=125&type=chunk) Condensed Consolidated Statements of Operations and Comprehensive Loss (in thousands, except per share amounts) | --- | --- | --- | | | **For the three months ended March 31, 2024 (in thousands)** | **For the three months ended March 31, 2023 (in thousands)** | | Revenues: | | | | License and other revenues | $— | $— | | Expenses: | | | | Research and development | 7,207 | 8,041 | | General and administrative | 7,123 | 3,997 | | Total expenses | 14,330 | 12,038 | | Loss from operations | (14,330) | (12,038) | | Interest income | 843 | 364 | | Interest expense | (952) | (101) | | Change in fair value of warrant and derivative liabilities | (17,301) | 2,265 | | Other income | 162 | 403 | | **Net loss** | **$(31,578)** | **$(9,107)** | | Basic and diluted loss per common share | $(1.16) | $(0.54) | | Weighted average number of common shares outstanding – basic and diluted | 27,315,537 | 16,904,024 | | Other comprehensive income (loss): | | | | Change in unrealized (losses) gains related to available-for-sale debt securities | (118) | 64 | | **Comprehensive loss** | **$(31,696)** | **$(9,043)** | [Unaudited Condensed Consolidated Statements of Stockholders' Equity (Deficit)](index=7&type=section&id=Unaudited%20Condensed%20Consolidated%20Statements%20of%20Stockholders%27%20Equity%20(Deficit)%20for%20the%20three%20months%20ended%20March%2031%2C%202024%20and%202023) Details changes in stockholders' equity (deficit) for the three months ended March 31, 2024, and 2023, reflecting net loss, stock issuances, and compensation - **Total stockholders' equity shifted from a positive $14.8 million to a deficit of $(8.9) million**[9](index=9&type=chunk)[13](index=13&type=chunk) - This change was primarily due to the **net loss of $(31.6) million**, partially offset by proceeds from common stock issuances (**$6.5 million from ATM sales**) and stock-based compensation (**$1.5 million**)[11](index=11&type=chunk)[13](index=13&type=chunk)[84](index=84&type=chunk)[95](index=95&type=chunk) Condensed Consolidated Statements of Stockholders' Equity (Deficit) (in thousands, except share amounts) | --- | --- | --- | --- | --- | --- | --- | | | **Common Shares** | **Common Stock Amount (in thousands)** | **Additional Paid-in Capital (in thousands)** | **Accumulated Deficit (in thousands)** | **Accumulated Other Comprehensive Loss (in thousands)** | **Total Stockholders' Equity (Deficit) (in thousands)** | | **Balance at December 31, 2023** | 26,523,878 | $265 | $764,151 | $(749,524) | $(66) | $14,826 | | Stock-based compensation expense | — | — | 1,546 | — | — | 1,546 | | Issuance of common stock in connection with restricted share awards, net | 137,500 | 2 | (16) | — | — | (14) | | Issuance of common stock, net of offering costs under open market sale agreement (ATM) | 889,315 | 9 | 6,448 | — | — | 6,457 | | Net loss | — | — | — | (31,578) | — | (31,578) | | Other comprehensive income | — | — | — | — | (118) | (118) | | **Balance at March 31, 2024** | **27,550,693** | **$276** | **$772,129** | **$(781,102)** | **$(184)** | **$(8,881)** | | **Balance at December 31, 2022** | 17,719,720 | $177 | $722,049 | $(695,336) | $(129) | $26,761 | | Stock-based compensation expense | — | — | 770 | — | — | 770 | | Issuance of common stock in connection with restricted share awards, net | 111,064 | 1 | (5) | — | — | (4) | | Issuance of common stock, net of offering costs under open market sale agreement (ATM) | 98,560 | 1 | 255 | — | — | 256 | | Net loss | — | — | — | (9,107) | — | (9,107) | | Other comprehensive income | — | — | — | — | 64 | 64 | | **Balance at March 31, 2023** | **17,929,344** | **$179** | **$723,069** | **$(704,443)** | **$(65)** | **$18,740** | [Unaudited Condensed Consolidated Statements of Cash Flows](index=8&type=section&id=Unaudited%20Condensed%20Consolidated%20Statements%20of%20Cash%20Flows%20for%20the%20three%20months%20ended%20March%2031%2C%202024%20and%202023) Summarizes cash inflows and outflows from operating, investing, and financing activities for the three months ended March 31, 2024, and 2023 - **Net cash used in operating activities increased to $(14.5) million** for Q1 2024 from $(11.7) million in the prior year, primarily due to the increased net loss[15](index=15&type=chunk)[136](index=136&type=chunk) - **Net cash used in investing activities was $(7.8) million**, a shift from $2.2 million provided in the prior year, mainly due to increased purchases of short-term investments[15](index=15&type=chunk)[137](index=137&type=chunk) - **Net cash provided by financing activities significantly increased to $25.4 million** from $(4) thousand in the prior year, driven by **$6.4 million from ATM sales** and **$19.0 million from the January 2024 Loan Agreement**[15](index=15&type=chunk)[138](index=138&type=chunk) - Overall, there was a **net increase in cash, cash equivalents, and restricted cash of $3.1 million** for the period, compared to a decrease of $(9.5) million in the prior year[15](index=15&type=chunk) Condensed Consolidated Statements of Cash Flows (in thousands) | --- | --- | --- | | | **For the three months ended March 31, 2024 (in thousands)** | **For the three months ended March 31, 2023 (in thousands)** | | Cash flows from operating activities: | | | | Net loss | $(31,578) | $(9,107) | | Adjustments to reconcile net loss to cash used in operating activities: | | | | Depreciation and amortization | 491 | 661 | | Stock-based compensation expense | 1,546 | 770 | | Change in fair value of warrant and derivative liabilities | 17,301 | (2,265) | | Accretion and interest on short-term investments | (59) | (117) | | Amortization of right-of-use lease assets | 233 | 227 | | Non-cash interest | 345 | 100 | | Change in operating assets and liabilities: | | | | Other receivables | 252 | (75) | | Prepaid expenses and other current assets | (1,232) | (1,199) | | Other assets | 163 | (56) | | Accounts payable and accrued expenses | (1,690) | (376) | | Lease liabilities | (310) | (308) | | Other current liabilities | — | 1 | | **Net cash used in operating activities** | **$(14,538)** | **$(11,744)** | | Cash flows from investing activities: | | | | Capital expenditures | (725) | (218) | | Purchases of short-term investments | (29,343) | (7,964) | | Proceeds from maturities of short-term investments | 22,251 | 10,393 | | **Net cash (used in) provided by investing activities** | **$(7,817)** | **$2,211** | | Cash flows from financing activities: | | | | Proceeds from ATM sales of common stock, net of issuance costs | 6,417 | — | | Payment from net settlement of restricted share awards | (14) | (4) | | Proceeds from issuance of long-term debt | 20,000 | — | | Payment of debt issuance costs | (963) | — | | **Net cash provided by (used in) financing activities** | **$25,440** | **$(4)** | | Net increase (decrease) in cash, cash equivalents and restricted cash | 3,085 | (9,537) | | Cash, cash equivalents and restricted cash at beginning of period | 14,811 | 14,555 | | **Cash, cash equivalents and restricted cash at end of period** | **$17,896** | **$5,018** | | Supplemental cash flow information: | | | | Cash and cash equivalents | $17,558 | $4,680 | | Restricted cash | 338 | 338 | | Total cash, cash equivalents and restricted cash | $17,896 | $5,018 | | Supplemental non-cash flow information: | | | | Derivative and warrant additions associated with loan and security agreement | $1,042 | $— | | Cash paid for interest | $607 | $— | | Cash paid for taxes | $8 | $6 | [Notes to Unaudited Condensed Consolidated Financial Statements](index=9&type=section&id=Notes%20to%20Unaudited%20Condensed%20Consolidated%20Financial%20Statements) Provides detailed explanations and disclosures for the unaudited condensed consolidated financial statements, covering significant accounting policies and specific financial items [NOTE 1 – Nature of Operations and Significant Accounting Policies](index=9&type=section&id=NOTE%201%20%E2%80%93%20NATURE%20OF%20OPERATIONS%20AND%20SIGNIFICANT%20ACCOUNTING%20POLICIES) Outlines Abeona Therapeutics Inc.'s business, its clinical-stage biopharmaceutical focus, and key accounting policies, including its liquidity outlook - Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing cell and gene therapies, with **pz-cel for RDEB as its lead clinical program**[18](index=18&type=chunk) - The Company expects its existing cash resources of **$62.7 million** as of March 31, 2024, plus **$75.0 million gross proceeds from a May 2024 offering**, to fund operations for at least the next 12 months[23](index=23&type=chunk)[25](index=25&type=chunk) Net Loss and Potential Dilutive Securities | Metric | 3 Months Ended March 31, 2024 | 3 Months Ended March 31, 2023 | | :------------------------------------------------------------------ | :---------------------------- | :---------------------------- | | Net Loss (in millions) | $(31.6) | $(9.1) | | Basic and Diluted Loss Per Common Share | $(1.16) | $(0.54) | | Weighted Average Common Shares Outstanding (Basic & Diluted) | 27,315,537 | 16,904,024 | | Potential Dilutive Securities (not included due to anti-dilution): | | | | Shares issuable upon exercise of stock options | 179,001 | 234,697 | | Shares underlying restricted stock | 2,542,619 | 929,946 | | Shares issuable upon exercise of warrants | 9,903,142 | 9,397,879 | | Total Potential Dilutive Securities | 12,624,762 | 10,562,522 | [NOTE 2 – Short-Term Investments](index=12&type=section&id=NOTE%202%20%E2%80%93%20SHORT-TERM%20INVESTMENTS) Details the company's short-term investments, primarily U.S. treasury and federal agency securities, and their fair value measurement - The company's short-term investments, primarily U.S. treasury and federal agency securities, totaled **$44.8 million** as of March 31, 2024, up from $37.8 million at December 31, 2023[37](index=37&type=chunk)[38](index=38&type=chunk) - These available-for-sale securities are carried at fair value, with unrealized losses attributed to interest rate changes, and no significant realized gains or losses were recognized[38](index=38&type=chunk)[39](index=39&type=chunk) Short-Term Investments (Available-for-sale) (in thousands) | Investment Type | March 31, 2024 (in thousands) | December 31, 2023 (in thousands) | | :------------------------------- | :---------------------------- | :------------------------------- | | U.S. treasury securities | $36,832 | $8,393 | | U.S. federal agency securities | $7,954 | $29,360 | | **Total Fair Value** | **$44,786** | **$37,753** | | Gross Unrealized Loss (March 31, 2024) | $(183) | $(66) | [NOTE 3 – Property and Equipment, Net](index=13&type=section&id=NOTE%203%20%E2%80%93%20PROPERTY%20AND%20EQUIPMENT%2C%20NET) Reports on the company's property and equipment, net, including additions and depreciation, for the reported periods - **Property and equipment, net, increased to $3.8 million** as of March 31, 2024, from $3.5 million at December 31, 2023, reflecting additions to various assets[41](index=41&type=chunk) - **Depreciation and amortization on property and equipment decreased to $0.5 million** for Q1 2024 from $0.7 million for the same period in 2023[41](index=41&type=chunk) Property and Equipment, Net (in thousands) | Category | March 31, 2024 (in thousands) | December 31, 2023 (in thousands) | | :------------------------------------- | :---------------------------- | :------------------------------- | | Laboratory equipment | $7,498 | $6,935 | | Furniture, software and office equipment | $1,045 | $986 | | Leasehold improvements | $8,706 | $8,603 | | Subtotal | $17,249 | $16,524 | | Less: accumulated depreciation | $(13,482) | $(12,991) | | **Total property and equipment, net** | **$3,767** | **$3,533** | [NOTE 4 – Fair Value Measurements](index=13&type=section&id=NOTE%204%20%E2%80%93%20FAIR%20VALUE%20MEASUREMENTS) Discusses the fair value measurements of financial assets and liabilities, categorizing them into Level 1, 2, and 3 inputs - As of March 31, 2024, **total assets measured at fair value were $62.3 million**, primarily in Level 1 and Level 2 categories[47](index=47&type=chunk) - **Total liabilities measured at fair value significantly increased to $54.4 million**, all classified as Level 3 due to unobservable inputs in valuation models[47](index=47&type=chunk) Financial Assets and Liabilities Measured at Fair Value (in thousands) | Description | Fair Value at March 31, 2024 (in thousands) | Fair Value at December 31, 2023 (in thousands) | Level 1 (2024) (in thousands) | Level 2 (2024) (in thousands) | Level 3 (2024) (in thousands) | | :----------------------------------------- | :---------------------------- | :------------------------------- | :------------- | :------------- | :------------- | | **Recurring Assets:** | | | | | | | Cash equivalents (Money market fund) | $17,558 | $1,034 | $17,558 | $— | $— | | Short-term investments (U.S. treasury) | $36,832 | $8,393 | $36,832 | $— | $— | | Short-term investments (U.S. federal agency) | $7,954 | $29,360 | $— | $7,954 | $— | | **Total Assets Measured at Fair Value** | **$62,344** | **$38,787** | **$54,390** | **$7,954** | **$—** | | **Liabilities:** | | | | | | | Payable to licensor | $4,691 | $4,580 | $— | $— | $4,691 | | Derivative liabilities | $1,005 | $— | $— | $— | $1,005 | | Warrant liabilities | $48,690 | $31,352 | $— | $— | $48,690 | | **Total Liabilities Measured at Fair Value** | **$54,386** | **$35,932** | **$—** | **$—** | **$54,386** | Warrant Liabilities Activity (in thousands) | Activity | Amount (in thousands) | | :-------------------------------------------------------------------- | :-------------------- | | Warrant liabilities as of December 31, 2023 | $31,352 | | Fair value of warrants issued in connection with Loan Agreement | $200 | | Loss recognized in earnings from change in fair value | $17,138 | | **Warrant liabilities as of March 31, 2024** | **$48,690** | Derivative Liabilities Activity (in thousands) | Activity | Amount (in thousands) | | :-------------------------------------------------------------------- | :-------------------- | | Derivative liabilities as of December 31, 2023 | $— | | Fair value of derivatives issued in connection with Loan Agreement | $800 | | Loss recognized in earnings from change in fair value | $205 | | **Derivative liabilities as of March 31, 2024** | **$1,005** | [NOTE 5 – Settlement Liability](index=17&type=section&id=NOTE%205%20%E2%80%93%20SETTLEMENT%20LIABILITY) Details the company's settlement agreement with REGENXBIO Inc., including past payments and the remaining present value due - The Company entered into a settlement agreement with REGENXBIO Inc. for **$30.0 million**, with **$20.0 million paid in November 2021** and **$5.0 million paid in November 2022**[58](index=58&type=chunk) - A remaining payment of **$5.0 million is due by November 2024** or upon a Strategic Transaction's closing[58](index=58&type=chunk) - As of March 31, 2024, the **present value of the amount due was $4.7 million**, calculated using a **9.6% effective interest rate**[59](index=59&type=chunk) [NOTE 6 – Accrued Expenses](index=17&type=section&id=NOTE%206%20%E2%80%93%20ACCRUED%20EXPENSES) Reports on the components and changes in accrued expenses, highlighting the primary drivers of reduction - **Total accrued expenses decreased to $2.8 million** as of March 31, 2024, from $6.0 million at December 31, 2023[61](index=61&type=chunk) - This reduction was primarily driven by a decrease in accrued employee compensation and accrued contracted services and other expenses[61](index=61&type=chunk) Components of Accrued Expenses (in thousands) | Component | March 31, 2024 (in thousands) | December 31, 2023 (in thousands) | | :------------------------------ | :---------------------------- | :------------------------------- | | Accrued employee compensation | $1,328 | $3,688 | | Accrued contracted services and other | $1,463 | $2,297 | | **Total accrued expenses** | **$2,791** | **$5,985** | [NOTE 7 – Leases](index=17&type=section&id=NOTE%207%20%E2%80%93%20LEASES) Provides details on operating lease liabilities, costs, and expected sublease income for the reported periods - **Total operating lease liabilities decreased to $5.1 million** as of March 31, 2024, from $5.4 million at December 31, 2023[63](index=63&type=chunk) - **Operating lease costs for Q1 2024 were $0.4 million**, a decrease from $0.47 million in the prior year[65](index=65&type=chunk) - The Company expects to receive **$1.0 million in future sublease income through September 2025** from two sublease agreements[62](index=62&type=chunk) Operating Lease Liabilities (in thousands) | Category | March 31, 2024 (in thousands) | December 31, 2023 (in thousands) | | :------------------------------ | :---------------------------- | :------------------------------- | | Current operating lease liability | $1,044 | $998 | | Non-current operating lease liability | $4,046 | $4,402 | | **Total operating lease liability** | **$5,090** | **$5,400** | Operating Lease Costs (in thousands) | Component | 3 Months Ended March 31, 2024 (in thousands) | 3 Months Ended March 31, 2023 (in thousands) | | :------------------------ | :------------------------------------------- | :------------------------------------------- | | Operating lease cost | $334 | $415 | | Variable lease cost | $74 | $39 | | Short-term lease cost | $23 | $18 | | **Total operating lease costs** | **$431** | **$472** | [NOTE 8 – Debt](index=19&type=section&id=NOTE%208%20%E2%80%93%20DEBT) Outlines the terms of the January 2024 Loan and Security Agreement, including principal, interest rates, and associated derivative liabilities - In January 2024, the company entered into a **$50 million Loan and Security Agreement**, with a committed **Tranche 1 of $20 million funded at closing**[73](index=73&type=chunk) - The loans bear interest at **13.50%** as of March 31, 2024, and are repayable in equal monthly installments beginning April 8, 2025[74](index=74&type=chunk) - The agreement includes a Conversion Right for Avenue to convert up to **$3 million of principal into common stock**, classified as a derivative liability, and warrants were issued to lenders[78](index=78&type=chunk)[80](index=80&type=chunk) Debt, Net of Issuance Costs and Discounts (in thousands) | Component | March 31, 2024 (in thousands) | December 31, 2023 (in thousands) | | :-------------------------------------- | :---------------------------- | :------------------------------- | | Loan Agreement principal | $20,000 | $— | | Accreted final payment fee | $74 | $— | | Unamortized debt issuance costs and discounts | $(1,995) | $— | | **Total debt** | **$18,079** | **$—** | [NOTE 9 – Equity](index=20&type=section&id=NOTE%209%20%E2%80%93%20EQUITY) Details common stock issuances, warrant classifications, and pre-funded warrants, impacting the company's equity structure - The Company sold **889,315 shares of common stock** under its ATM Agreement during Q1 2024, generating **net proceeds of $6.5 million**[84](index=84&type=chunk) - Warrants from the 2021 Public Offering (**1,788,000 shares**), 2022 Private Placement (**7,609,879 shares**), and 2024 Loan Agreement (**505,263 shares**) are classified as liabilities and re-measured at fair value[49](index=49&type=chunk)[51](index=51&type=chunk)[82](index=82&type=chunk)[86](index=86&type=chunk)[89](index=89&type=chunk) - The 2023 Direct Placement Offering included **2,919,140 pre-funded warrants**, classified as equity and considered outstanding shares for basic EPS calculation[88](index=88&type=chunk) [NOTE 10 – Stock-Based Compensation](index=22&type=section&id=NOTE%2010%20%E2%80%93%20STOCK-BASED%20COMPENSATION) Reports on stock-based compensation expense, its allocation, and unrecognized compensation costs for options and restricted stock awards - **Stock-based compensation expense for Q1 2024 increased to $1.5 million** from $0.8 million in the prior year, with a significant portion allocated to general and administrative expenses[95](index=95&type=chunk) - As of March 31, 2024, **total unrecognized compensation cost for non-vested option awards was approximately $1.0 million**, with a weighted average remaining vesting period of 1.1 years[99](index=99&type=chunk) - As of March 31, 2024, there was **$7.4 million of total unrecognized compensation expense** related to unvested restricted stock awards, expected to be recognized over a weighted average vesting period of 2.2 years[101](index=101&type=chunk) Stock-Based Compensation Expense (in thousands) | Category | 3 Months Ended March 31, 2024 (in thousands) | 3 Months Ended March 31, 2023 (in thousands) | | :--------------------------- | :------------------------------------------- | :------------------------------------------- | | Research and development | $346 | $584 | | General and administrative | $1,220 | $186 | | **Total stock-based compensation expense** | **$1,546** | **$770** | [NOTE 11 – License/Supplier Agreement](index=24&type=section&id=NOTE%2011%20%E2%80%93%20LICENSE%2FSUPPLIER%20AGREEMENT) Details sublicense and exclusive license agreements, including potential milestone payments and royalties, and revenue recognition policies - The Company has sublicense agreements with Taysha Gene Therapies for CLN1 disease and Rett syndrome, with potential event-based milestone payments up to **$26.0 million and $26.5 million**, and sales-based milestone payments up to **$30.0 million each**, plus royalties[104](index=104&type=chunk)[109](index=109&type=chunk) - An exclusive license agreement for ABO-102 (MPS IIIA) includes eligibility for **tiered royalties (mid-single-digit up to 10%)** on net sales and up to **$30.0 million in commercial milestone payments**[111](index=111&type=chunk) - No revenue was recognized from these licensing arrangements for Q1 2024 or 2023, as milestone payments are constrained until probable of not resulting in significant cumulative revenue reversal[105](index=105&type=chunk)[110](index=110&type=chunk) [NOTE 12 – Subsequent Events](index=25&type=section&id=NOTE%2012%20%E2%80%93%20SUBSEQUENT%20EVENTS) Reports on significant events occurring after the reporting period, specifically a May 2024 underwritten public offering and its net proceeds - On May 7, 2024, the Company closed an underwritten offering of **12,285,056 shares of common stock** and **6,142,656 pre-funded warrants** at **$4.07 per share**[113](index=113&type=chunk) - The estimated **net proceeds from this offering are approximately $70.2 million**, after deducting underwriting discounts, commissions, and estimated offering expenses[113](index=113&type=chunk) [Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=26&type=section&id=Item%202.%20Management%27s%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management's perspective on Q1 2024 financial condition and results, covering strategic overview, performance drivers, liquidity, and critical accounting estimates [Overview](index=26&type=section&id=OVERVIEW) Summarizes Abeona's strategic focus, lead clinical program pz-cel's FDA status, preclinical pipeline, and recent financing activities - Abeona's lead clinical program, **pz-cel for RDEB**, received a **Complete Response Letter (CRL) from the FDA in April 2024** due to Chemistry Manufacturing and Controls (CMC) requirements[116](index=116&type=chunk)[117](index=117&type=chunk)[118](index=118&type=chunk) - The company is preparing its cGMP commercial facility in Cleveland, Ohio, for pz-cel manufacturing and engaging with healthcare stakeholders for market access and pricing[119](index=119&type=chunk) - Preclinical pipeline includes AAV-based gene therapies for Stargardt disease (ABO-504), X-linked retinoschisis (ABO-503), and autosomal dominant optic atrophy (ABO-505)[120](index=120&type=chunk)[121](index=121&type=chunk) - Recent financing activities include a **$50 million credit facility in January 2024** and a **May 2024 underwritten offering yielding approximately $70.2 million in net proceeds**[122](index=122&type=chunk)[123](index=123&type=chunk) [Results of Operations](index=27&type=section&id=RESULTS%20OF%20OPERATIONS) Analyzes the company's financial results for Q1 2024 compared to Q1 2023, focusing on key drivers of net loss and expense changes - For Q1 2024, Abeona reported a **net loss of $(31.6) million**, a significant increase from $(9.1) million in the prior year[125](index=125&type=chunk) - This was primarily driven by a **$(17.3) million loss from the change in fair value of warrant and derivative liabilities**, a **78% increase in general and administrative expenses to $7.1 million**, and an **843% increase in interest expense to $1.0 million**[125](index=125&type=chunk)[128](index=128&type=chunk)[131](index=131&type=chunk) - **Research and development expenses decreased by 10% to $7.2 million**, mainly due to reduced clinical and development work[125](index=125&type=chunk)[126](index=126&type=chunk) Consolidated Statements of Operations Comparison (Q1 2024 vs. Q1 2023) (in thousands) | Metric | Q1 2024 (in thousands) | Q1 2023 (in thousands) | Change ($) (in thousands) | Change (%) | | :------------------------------------------- | :--------------------- | :--------------------- | :--------- | :--------- | | License and other revenues | $— | $— | $— | N/A | | Research and development expenses | $7,207 | $8,041 | $(834) | (10)% | | General and administrative expenses | $7,123 | $3,997 | $3,126 | 78% | | Total expenses | $14,330 | $12,038 | $2,292 | 19% | | Loss from operations | $(14,330) | $(12,038) | $(2,292) | 19% | | Interest income | $843 | $364 | $479 | 132% | | Interest expense | $(952) | $(101) | $(851) | 843% | | Change in fair value of warrant and derivative liabilities | $(17,301) | $2,265 | $(19,566) | (864)% | | Other income | $162 | $403 | $(241) | (60)% | | **Net loss** | **$(31,578)** | **$(9,107)** | **$(22,471)** | **247%** | [Liquidity and Capital Resources](index=29&type=section&id=LIQUIDITY%20AND%20CAPITAL%20RESOURCES) Discusses the company's cash resources, financing activities, cash flow trends, and future funding requirements - As of March 31, 2024, **cash resources were $62.7 million**, and with **$75.0 million gross proceeds from the May 2024 offering**, funds are sufficient for at least the next 12 months[23](index=23&type=chunk)[140](index=140&type=chunk) - **Net cash provided by financing activities was $25.4 million** for Q1 2024, driven by **$6.4 million from ATM sales** and **$19.0 million from the January 2024 Loan Agreement**[138](index=138&type=chunk) - **Net cash used in operating activities was $14.5 million**, and **net cash used in investing activities was $7.8 million** for Q1 2024[136](index=136&type=chunk)[137](index=137&type=chunk) - The Company expects to incur losses for several years and will require additional funding through equity offerings, debt financings, or collaborations for product development and commercialization[142](index=142&type=chunk)[143](index=143&type=chunk) Cash Flows Summary (Q1 2024 vs. Q1 2023) (in thousands) | Activity | Q1 2024 (in thousands) | Q1 2023 (in thousands) | | :-------------------------------------------------------------------------- | :--------------------- | :--------------------- | | Net cash used in operating activities | $(14,538) | $(11,744) | | Net cash (used in) provided by investing activities | $(7,817) | $2,211 | | Net cash provided by (used in) financing activities | $25,440 | $(4) | | **Net increase (decrease) in cash, cash equivalents and restricted cash** | **$3,085** | **$(9,537)** | [Critical Accounting Estimates](index=32&type=section&id=Critical%20Accounting%20Estimates) Highlights key accounting estimates, specifically the fair value measurement of the derivative liability associated with the loan agreement - The fair value of the conversion right embedded within the loan agreement is accounted for as a derivative liability, measured using a Monte Carlo simulation model[149](index=149&type=chunk) [Recently Issued Accounting Standards Not Yet Effective or Adopted](index=32&type=section&id=Recently%20Issued%20Accounting%20Standards%20Not%20Yet%20Effective%20or%20Adopted) Identifies recently issued accounting pronouncements, ASU No. 2023-09 and ASU 2023-07, and their effective dates - The Company is assessing the impact of recently issued accounting pronouncements, **ASU No. 2023-09 (Income Taxes)** and **ASU 2023-07 (Segment Reporting)**[34](index=34&type=chunk)[35](index=35&type=chunk)[150](index=150&type=chunk) [Item 3. Quantitative and Qualitative Disclosures About Market Risk](index=32&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) States that no quantitative and qualitative disclosures about market risk are applicable for the reported period [Item 4. Controls and Procedures](index=32&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded that disclosure controls and procedures were effective as of March 31, 2024, with no material changes in internal control over financial reporting - The Chief Executive Officer and Chief Financial Officer concluded that the Company's Disclosure Controls and Procedures were effective as of March 31, 2024[151](index=151&type=chunk) - There were no material changes in the Company's internal control over financial reporting during Q1 2024[152](index=152&type=chunk) PART II - OTHER INFORMATION [Item 1. Legal Proceedings](index=33&type=section&id=Item%201.%20Legal%20Proceedings) The company reported no legal proceedings for the quarter ended March 31, 2024 [Item 1A. Risk Factors](index=33&type=section&id=Item%201A.%20Risk%20Factors) Highlights the new material risk factor of the FDA's Complete Response Letter (CRL) for pz-cel's BLA due to CMC requirements, potentially delaying commercialization - The FDA issued a **Complete Response Letter (CRL) for pz-cel's Biologics License Application (BLA)** due to Chemistry Manufacturing and Controls (CMC) requirements, posing a new material risk factor[155](index=155&type=chunk) - Failure to satisfy CRL requirements or delays could shorten exclusive commercialization rights, allow competitors to enter, and materially harm the company's business and prospects[155](index=155&type=chunk) [Item 2. Unregistered Sale of Equity Securities and Use of Proceeds](index=33&type=section&id=Item%202.%20Unregistered%20Sale%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) The company reported no unregistered sales of equity securities or purchases of registered equity securities for the quarter ended March 31, 2024 [Item 5. Other Information](index=33&type=section&id=Item%205.%20Other%20Information) Details Rule 10b5-1 trading arrangements adopted by the President and CEO and CFO during Q1 2024 for common stock and restricted stock unit sales - President and CEO, Vishwas Seshadri, adopted a **Rule 10b5-1 trading arrangement on February 9, 2024**, for the sale of up to **20,000 shares of common stock** and restricted stock unit settlements, effective until October 30, 2025[159](index=159&type=chunk) - CFO, Joseph Vazzano, adopted two **Rule 10b5-1 trading arrangements on February 9 and February 12, 2024**, for the sale of up to **14,979 shares** and **47,302 shares from restricted stock units**, and an additional **5,500 shares**, effective until December 31, 2024[160](index=160&type=chunk)[161](index=161&type=chunk) [Item 6. Exhibits](index=35&type=section&id=Item%206.%20Exhibits) Lists the exhibits filed as part of the Form 10-Q, including warrants, the Loan and Security Agreement, certifications, and XBRL data SIGNATURES - The report is duly signed on behalf of Abeona Therapeutics Inc. by its President and Chief Executive Officer, Vishwas Seshadri, and Chief Financial Officer, Joseph Vazzano, on May 15, 2024[167](index=167&type=chunk)[168](index=168&type=chunk)

Exhibit 99.1 Abeona Therapeutics Reports First Quarter 2024 Financial Results and Recent Corporate Progress BLA resubmission anticipated in second half of 2024 Closed $75 million underwritten offering in May, extending expected cash runway into 2026 CLEVELAND, May 15, 2024 – Abeona Therapeutics Inc. (Nasdaq: ABEO) today reported financial results for the first quarter of 2024 and recent corporate progress. “We are grateful to our existing as well as new investors who have demonstrated their support through ...

Core Viewpoint - Abeona Therapeutics Inc. is focused on addressing FDA's Chemistry Manufacturing and Controls (CMC) deficiencies to resubmit its Biologics License Application (BLA) for prademagene zamikeracel (pz-cel) for recessive dystrophic epidermolysis bullosa (RDEB) in the second half of 2024, following a successful $75 million financing that extends its cash runway into 2026 [1][3][5]. Corporate Highlights - On May 7, 2024, the company closed a $75 million underwritten securities offering with participation from both new and existing investors [2]. - In January 2024, Abeona entered into a $50 million credit facility and received the first tranche of $20 million [2]. Product Development - The FDA issued a Complete Response Letter (CRL) in April 2024 regarding the BLA for pz-cel, citing the need for additional CMC information, but did not request new clinical trials or data [3]. - The company anticipates completing the BLA resubmission in the second half of 2024 and has accepted new long-term safety data for pz-cel for presentation at the Society for Investigative Dermatology Annual Meeting [3]. Commercial Launch Preparations - Abeona is advancing key commercial activities in preparation for a potential U.S. launch of pz-cel, including discussions with treatment sites and building supply chain capabilities [4]. Financial Results - As of March 31, 2024, the company reported cash and cash equivalents totaling $62.7 million, an increase from $52.6 million as of December 31, 2023 [5]. - The net loss for the first quarter of 2024 was $31.6 million, or $1.16 loss per common share, compared to a net loss of $9.1 million, or $0.54 loss per common share, in the same period of 2023 [6][7]. Research and Development Expenses - Research and development expenses for the first quarter of 2024 were $7.2 million, down from $8.0 million in the same period of 2023 [6]. - General and administrative expenses increased to $7.1 million in the first quarter of 2024, compared to $4.0 million in the same period of 2023 [6]. Balance Sheet Overview - Total assets as of March 31, 2024, were $74.8 million, compared to $64.0 million as of December 31, 2023 [17]. - Total liabilities increased to $83.7 million as of March 31, 2024, from $49.2 million as of December 31, 2023 [18].

Core Viewpoint - Abeona Therapeutics Inc. has announced an underwritten offering of common stock and pre-funded warrants, aiming to raise approximately $75 million to support its product candidate pz-cel and other corporate purposes [1][2]. Group 1: Offering Details - The offering consists of 12,285,056 shares of common stock and pre-funded warrants for 6,142,656 shares, priced at $4.07 per share, equal to the closing price on May 2, 2024 [1]. - The pre-funded warrants are immediately exercisable at a nominal price of $0.0001 per share and can be exercised at any time until fully exercised [1]. - The closing of the offering is expected around May 7, 2024, pending customary closing conditions [1]. Group 2: Investor Participation - The offering has attracted both new and existing investors, including notable firms such as Adage Capital Partners, L.P., Janus Henderson Investors, and Nantahala Capital [2]. - Stifel is serving as the sole bookrunner for this offering [2]. Group 3: Use of Proceeds - Abeona plans to utilize the net proceeds primarily for the resubmission of its Biologics License Application (BLA) for pz-cel, commercialization efforts, working capital, and general corporate purposes [2]. Group 4: Company Overview - Abeona Therapeutics is a clinical-stage biopharmaceutical company focused on developing cell and gene therapies for serious diseases, with pz-cel being an investigational treatment for recessive dystrophic epidermolysis bullosa [5]. - The company has a fully integrated cGMP manufacturing facility capable of supporting commercial production of pz-cel upon FDA approval [5]. - Abeona is also developing AAV-based gene therapies for ophthalmic diseases, utilizing next-generation AAV capsids to enhance treatment efficacy [5].

chemicalbilly/E+ via Getty Images Abeona Therapeutics Inc. (NASDAQ:ABEO) dropped by more than half on Tuesday after the company received a Complete Response Letter from the FDA in response to the Company’s Biologics License Application for pz-cel. In one day, ABEO erased nearly a year's worth of gains that had been building on positive speculation that pz-cel would gain approval at next month's PDUFA. Those hopes were dashed as the FDA identified manufacturing issues that the company must take the next ...

Core Viewpoint - Abeona Therapeutics received a Complete Response Letter (CRL) from the FDA regarding its Biologics License Application (BLA) for prademagene zamikeracel (pz-cel), indicating the need for additional Chemistry Manufacturing and Controls (CMC) information, but did not identify any deficiencies related to clinical efficacy or safety data [1][2][3] Group 1: Regulatory Update - The FDA's CRL was issued following a Late Cycle Review Meeting in March 2024, where it was noted that additional CMC information is required before the application can be approved [1][2] - Abeona plans to complete and submit the requested CMC information by the third quarter of 2024 [1][3] - A conference call is scheduled for April 23, 2024, to provide details on the requested CMC information [4] Group 2: Product Information - Prademagene zamikeracel (pz-cel) is an investigational treatment for recessive dystrophic epidermolysis bullosa (RDEB), utilizing gene-corrected epidermal sheets to address a defect in the COL7A1 gene [5][6] - The pivotal Phase 3 VIITAL™ study demonstrated that a single application of pz-cel can lead to sustained wound healing and pain reduction [3][5] - Pz-cel has received multiple designations from the FDA, including Regenerative Medicine Advanced Therapy and Breakthrough Therapy [6] Group 3: Company Overview - Abeona Therapeutics is a clinical-stage biopharmaceutical company focused on developing cell and gene therapies for serious diseases [7] - The company operates a fully integrated gene and cell therapy manufacturing facility in Cleveland, Ohio, capable of supporting commercial production upon FDA approval [7]

[Company Announcement & Highlights](index=1&type=section&id=Company%20Announcement%20%26%20Highlights) Abeona Therapeutics reported 2023 financial results and completed key FDA inspections for its pz-cel BLA, including manufacturing and clinical sites [Full Year 2023 Financial Results and FDA Inspections Completion](index=1&type=section&id=Full%20Year%202023%20Financial%20Results%20and%20FDA%20Inspections%20Completion) Abeona Therapeutics reported its full year 2023 financial results and announced the completion of key FDA inspections for its pz-cel Biologics License Application (BLA), including a Pre-License Inspection of its Cleveland manufacturing facility and clinical trial site inspections - Abeona Therapeutics reported **full year 2023 financial results**[1](index=1&type=chunk) - FDA completed a **Pre-License Inspection (PLI)** of Abeona's Cleveland manufacturing facility for pz-cel BLA[2](index=2&type=chunk) - FDA also completed clinical study site inspections for the pivotal Phase 3 VIITAL™ study, with **no Form 483 observations**[2](index=2&type=chunk) [Regulatory & Commercial Progress](index=1&type=section&id=Regulatory%20%26%20Commercial%20Progress) Abeona advanced pz-cel's FDA review with priority status, prepared for U.S. commercial launch, and secured a $50 million credit facility [Pz-cel for RDEB Regulatory Updates](index=1&type=section&id=Pz-cel%20for%20RDEB%20Regulatory%20Updates) The FDA has made significant progress in its review of Abeona's pz-cel BLA for recessive dystrophic epidermolysis bullosa (RDEB), including granting Priority Review, completing various inspections, and reaffirming a target PDUFA date of May 25, 2024, without plans for an Advisory Committee - FDA completed a Pre-License Inspection (PLI) of the Cleveland manufacturing facility, issuing a **Form 483 with observations** related to process controls, to which Abeona submitted a response[2](index=2&type=chunk) - FDA accepted and granted **Priority Review** for the pz-cel BLA in November 2023, with a target PDUFA date of **May 25, 2024**[3](index=3&type=chunk) - FDA completed a Bioresearch Monitoring (BIMO) inspection and Mid-Cycle Meeting in January 2024, with **no Form 483s issued** from the BIMO inspection and **no Risk Evaluation and Mitigation Strategies (REMS) anticipated** for the BLA[4](index=4&type=chunk) [U.S. Commercial Launch Preparations for pz-cel](index=2&type=section&id=U.S.%20Commercial%20Launch%20Preparations%20for%20pz-cel) Abeona is actively preparing for the potential U.S. commercial launch of pz-cel, engaging with treatment sites, payers, and conducting market research to support reimbursement coverage - Abeona is **advancing key commercial activities** for pz-cel launch, including onboarding discussions with EB treatment sites, payer engagement, and hiring key commercial roles[5](index=5&type=chunk) - Payer market research supports **reimbursement coverage** of pz-cel at a price commensurate with the value of approved gene therapies[5](index=5&type=chunk) [Corporate Financing Highlights](index=2&type=section&id=Corporate%20Financing%20Highlights) Abeona secured a $50 million credit facility in January 2024, with an initial $20 million tranche funded, to support ongoing launch and commercialization preparations for pz-cel - Abeona entered a **$50 million credit facility** with Avenue Venture Opportunities Fund, L.P. in January 2024[5](index=5&type=chunk) - The credit agreement includes a **first tranche of $20 million**, funded in January 2024, a second tranche of $10 million, and an option for an additional $20 million[5](index=5&type=chunk) - Proceeds from the facility are intended to **support pz-cel launch and commercialization** preparations[5](index=5&type=chunk) [Financial Performance](index=2&type=section&id=Financial%20Performance) Abeona's 2023 financials show stable cash, increased licensing revenue, higher operating expenses, and an increased net loss [Full Year 2023 Financial Results and Cash Runway Guidance](index=2&type=section&id=Full%20Year%202023%20Financial%20Results%20and%20Cash%20Runway%20Guidance) Abeona maintained a stable cash position at the end of 2023 and significantly reduced net cash used in operating activities. With recent financing, the company projects its cash runway to extend into the first quarter of 2025 Cash and Investments (in millions) | Metric | Dec 31, 2023 | Dec 31, 2022 | | :----------------------------------------- | :----------- | :----------- | | Cash, cash equivalents, restricted cash and short-term investments | $52.6 | $52.5 | Net Cash Used in Operating Activities (in millions) | Period | 2023 | 2022 | | :----------- | :---- | :---- | | Net cash used in operating activities | $37.0 | $43.5 | - Current cash and credit facility are **sufficient to fund operations into Q1 2025**, excluding potential pz-cel revenue or Priority Review Voucher proceeds[6](index=6&type=chunk) [Revenue Analysis](index=2&type=section&id=Revenue%20Analysis) License and other revenues increased significantly in 2023, primarily driven by clinical milestone payments from a licensing agreement for an AAV-based gene therapy License and Other Revenues (in millions) | Metric | 2023 | 2022 | YoY Change | | :------------------------- | :---- | :---- | :--------- | | License and other revenues | $3.5 | $1.4 | +150% | - Revenues primarily represent **clinical milestone payments** under a licensing agreement with Taysha Gene Therapies for investigational AAV-based gene therapy for Rett syndrome[7](index=7&type=chunk) [Operating Expenses](index=2&type=section&id=Operating%20Expenses) Both research and development (R&D) and general and administrative (G&A) expenses increased in 2023, primarily due to increased headcount supporting BLA activities and pz-cel launch preparations Operating Expenses (in millions) | Metric | 2023 | 2022 | YoY Change | | :------------------------------- | :---- | :---- | :--------- | | Research and development expenses | $31.1 | $29.0 | +7.2% | | General and administrative expenses | $19.0 | $17.3 | +9.8% | - Increase in R&D expenses was primarily due to **increased headcount related to BLA activities**[8](index=8&type=chunk) - Increase in G&A expenses was primarily due to **increased headcount for the potential launch of pz-cel**[8](index=8&type=chunk) [Net Loss and EPS](index=2&type=section&id=Net%20Loss%20and%20EPS) Abeona reported an increased net loss attributable to common shareholders in 2023, with a corresponding decrease in loss per common share due to a higher weighted average number of shares outstanding Net Loss and EPS (in millions, except per share amounts) | Metric | 2023 | 2022 | YoY Change | | :----------------------------------------- | :---------- | :---------- | :--------- | | Net loss attributable to common shareholders | $(54.2) million | $(43.5) million | +24.6% | | Loss per common share | $(2.53) | $(5.53) | -54.3% | [Company Information](index=3&type=section&id=Company%20Information) Abeona Therapeutics is a clinical-stage biopharmaceutical company developing cell and gene therapies, with pz-cel for RDEB nearing FDA approval [About Abeona Therapeutics](index=3&type=section&id=About%20Abeona%20Therapeutics) Abeona Therapeutics is a clinical-stage biopharmaceutical company focused on developing cell and gene therapies for serious diseases, with pz-cel for RDEB as its lead investigational product nearing potential FDA approval, supported by its integrated cGMP manufacturing facility - Abeona Therapeutics is a **clinical-stage biopharmaceutical company** developing cell and gene therapies for serious diseases[10](index=10&type=chunk) - The U.S. FDA has granted **Priority Review** for the BLA for pz-cel (prademagene zamikeracel) for recessive dystrophic epidermolysis bullosa (RDEB), with a PDUFA target action date of **May 25, 2024**[10](index=10&type=chunk) - The company's development portfolio also includes **AAV-based gene therapies** for ophthalmic diseases and **novel, next-generation AAV capsids**[10](index=10&type=chunk) [Financial Statements](index=4&type=section&id=Financial%20Statements) This section details Abeona Therapeutics' consolidated statements of operations, comprehensive loss, and balance sheets for 2023 and 2022 [Consolidated Statements of Operations and Comprehensive Loss](index=4&type=section&id=Consolidated%20Statements%20of%20Operations%20and%20Comprehensive%20Loss) The consolidated statements of operations and comprehensive loss provide a detailed breakdown of Abeona Therapeutics' financial performance for the years ended December 31, 2023, and 2022, including revenues, expenses, and net loss Consolidated Statements of Operations and Comprehensive Loss (in thousands, except share and per share amounts) | Metric | 2023 | 2022 | | :--------------------------------------------------------------------------------------------------------- | :----------- | :----------- | | Revenues: | | | | License and other revenues | $3,500 | $1,414 | | Expenses: | | | | Royalties | $1,605 | $450 | | Research and development | $31,091 | $28,965 | | General and administrative | $19,004 | $17,256 | | Impairment of licensed technology | — | $1,355 | | Loss/(gain) on operating lease right-of-use assets | $(1,065) | $2,511 | | Impairment of construction-in-progress | — | $1,792 | | Total expenses | $50,635 | $52,329 | | Loss from operations | $(47,135) | $(50,915) | | Interest income | $2,117 | $431 | | Interest expense | $(418) | $(736) | | Change in fair value of warrant liabilities | $(11,695) | $11,383 | | Other income | $2,943 | $141 | | Net loss | $(54,188) | $(39,696) | | Deemed dividends related to Series A and Series B Convertible Redeemable Preferred Stock | — | $(3,782) | | Net loss attributable to Common Shareholders | $(54,188) | $(43,478) | | Basic and diluted loss per common share | $(2.53) | $(5.53) | | Weighted average number of common shares outstanding – basic and diluted | 21,380,476 | 7,861,515 | | Other comprehensive income (loss): | | | | Change in unrealized gains (losses) related to available-for-sale debt securities | $34 | $(99) | | Foreign currency translation adjustments | $29 | $(3) | | Comprehensive loss | $(54,125) | $(43,580) | [Consolidated Balance Sheets](index=5&type=section&id=Consolidated%20Balance%20Sheets) The consolidated balance sheets present Abeona Therapeutics' financial position as of December 31, 2023, and 2022, detailing assets, liabilities, and stockholders' equity Consolidated Balance Sheets (in thousands, except share and per share amounts) | Metric | December 31, 2023 | December 31, 2022 | | :--------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------- | :------------------ | :------------------ | | ASSETS | | | | Current assets: | | | | Cash and cash equivalents | $14,473 | $14,217 | | Short-term investments | $37,753 | $37,932 | | Restricted cash | $338 | $338 | | Other receivables | $2,444 | $188 | | Prepaid expenses and other current assets | $729 | $424 | | Total current assets | $55,737 | $53,099 | | Property and equipment, net | $3,533 | $5,741 | | Operating lease right-of-use assets | $4,455 | $5,331 | | Other assets | $277 | $43 | | Total assets | $64,002 | $64,214 | | LIABILITIES AND STOCKHOLDERS' EQUITY | | | | Current liabilities: | | | | Accounts payable | $1,858 | $1,811 | | Accrued expenses | $5,985 | $3,991 | | Current portion of operating lease liability | $998 | $1,773 | | Current portion of payable to licensor | $4,580 | — | | Other current liabilities | $1 | $204 | | Total current liabilities | $13,422 | $7,779 | | Payable to licensor | — | $4,163 | | Long-term operating lease liabilities | $4,402 | $5,854 | | Warrant liabilities | $31,352 | $19,657 | | Total liabilities | $49,176 | $37,453 | | Commitments and contingencies | | | | Stockholders' equity: | | | | Preferred stock - $0.01 par value; authorized 2,000,000 shares; No shares issued and outstanding as of December 31, 2023 and December 31, 2022, respectively | — | — | | Common stock - $0.01 par value; authorized 200,000,000 shares; 26,523,878 and 17,719,720 shares issued and outstanding as of December 31, 2023 and December 31, 2022, respectively | 265 | 177 | | Additional paid-in capital | $764,151 | $722,049 | | Accumulated deficit | $(749,524) | $(695,336) | | Accumulated other comprehensive loss | $(66) | $(129) | | Total stockholders' equity | $14,826 | $26,761 | | Total liabilities and stockholders' equity | $64,002 | $64,214 | [Disclosures & Contacts](index=2&type=section&id=Disclosures%20%26%20Contacts) This section provides conference call details, forward-looking statement disclaimers, and investor contact information [Conference Call Details](index=2&type=section&id=Conference%20Call%20Details) Abeona Therapeutics hosted a conference call and webcast on March 18, 2024, to discuss the financial results and corporate updates, with replay access available online - Abeona Therapeutics hosted a conference call and webcast on **March 18, 2024, at 8:30 a.m. ET**[9](index=9&type=chunk) - Access details: Dial 888-506-0062 (U.S. toll-free) or 973-528-0011 (international) with Entry Code: 428606[9](index=9&type=chunk) - A live webcast and archived replay are available at https://investors.abeonatherapeutics.com/events for **30 days**[9](index=9&type=chunk) [Forward-Looking Statements](index=3&type=section&id=Forward-Looking%20Statements) This section contains forward-looking statements subject to various risks and uncertainties, including those related to FDA approvals, clinical trials, and financial market conditions, and the company disclaims any obligation to update these statements - The press release contains **forward-looking statements** as defined by Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934[11](index=11&type=chunk) - Actual results may **differ materially due to various factors**, including the timing and outcome of the pz-cel BLA submission, FDA inspections, regulatory approvals, and global economic conditions[11](index=11&type=chunk) - The Company undertakes **no obligation to revise or update** forward-looking statements, except as required by federal securities laws[11](index=11&type=chunk) [Investor and Media Contact](index=3&type=section&id=Investor%20and%20Media%20Contact) Contact information for investor relations and corporate communications is provided for inquiries - Investor and Media Contact: **Greg Gin, VP, Investor Relations and Corporate Communications**, Abeona Therapeutics[12](index=12&type=chunk) - Email: **ir@abeonatherapeutics.com**[12](index=12&type=chunk)

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 FORM 10-K (Mark One) ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2023 Or ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from _______ to _______ Commission file number 001-15771 ABEONA THERAPEUTICS INC. (Exact name of registrant as specified in its charter) Delaware 83-0221517 (State or Oth ...

CLEVELAND, March 18, 2024 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO) today reported financial results for the full year of 2023 and provided an update on progress toward achieving key corporate objectives. Abeona also announced today that the U.S. Food and Drug Administration (FDA) has completed a Pre-License Inspection (PLI) of its Cleveland, Ohio manufacturing facility related to the Company's Biologics License Application (BLA) for pz-cel (prademagene zamikeracel) for recessive dystrophic ...

Group 1 - Abeona Therapeutics Inc. has granted equity awards to new non-executive employees, totaling up to 25,500 restricted shares of common stock, as a material inducement to employment [1] - The restricted stock awards will vest one-third yearly on each anniversary of the Grant Date, fully vesting on the third anniversary, contingent on continued employment [1] Group 2 - Abeona Therapeutics is a clinical-stage biopharmaceutical company focused on developing cell and gene therapies for serious diseases [2] - The U.S. FDA has accepted and granted Priority Review for the Biologics License Application for pz-cel, with a target action date of May 25, 2024 [2] - The company has a fully integrated cGMP manufacturing facility capable of supporting commercial production of pz-cel upon FDA approval [2] - Abeona's development portfolio includes AAV-based gene therapies for ophthalmic diseases, utilizing next-generation AAV capsids to improve tropism profiles [2]