Prademagene zamikeracel

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 (GLOBE NEWSWIRE) -- 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 the recent financing, which has extended our cash runway into 2026, well beyond anticipated regulatory milestones,” said Vish Seshadri, Chief Executive Officer of Abeona. “We now remain focused on working with the FDA to address the CMC deficiencies noted in the CRL and making the BLA resubmission to bring pz-cel to RDEB patients as soon as possible.” First Quarter and Recent Progress Corporate highlights On May 7, 2024, Abeona closed a $75 million underwritten securities offering with participation from both new and existing investors. In January 2024, Abeona entered into a $50 million credit facility and received the first tranche of $20 million. Pz-cel for RDEB In April 2024, Abeona received a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) regarding the Company's Biologics License Application (BLA) for prademagene zamikeracel (pz-cel) for recessive dystrophic epidermolysis bullosa (RDEB), based on the need for additional Chemistry Manufacturing and Controls (CMC) information. In the CRL, the FDA noted that certain additional information needed to satisfy CMC requirements must be resolved before the application can be approved. The information needed to satisfy the CMC requests in the CRL pertains to validation requirements for certain manufacturing and release testing methods. The CRL did not identify any deficiencies related to the clinical efficacy or clinical safety data in the BLA, and the FDA did not request any new clinical trials or clinical data to support the approval of pz-cel. The Company anticipates completing the BLA resubmission in the second half of 2024. New pz-cel long-term safety data with up to 11 years of follow-up has been accepted as a late-breaking presentation at the Society for Investigative Dermatology (SID) Annual Meeting, being held on May 15-18, 2024. U.S. commercial launch preparations for pz-cel Abeona continues to advance key commercial activities in preparation for a potential U.S. launch for pz-cel, including onboarding discussions with epidermolysis bullosa treatment sites, conducting medical and payer engagement, as well as building supply chain and enterprise capabilities to support the Company’s transition to a commercial stage company. First Quarter Financial Results and Cash Runway Guidance Cash, cash equivalents, restricted cash and short-term investments totaled $62.7 million as of March 31, 2024, compared to $52.6 million as of December 31, 2023. Net cash used in operating activities was $14.5 million for the three months ended March 31, 2024. Abeona estimates that its current cash and cash equivalents, restricted cash and short-term investments, as well as the credit facility, combined with the net proceeds from the underwritten securities offering, are sufficient resources to fund operations into 2026, before accounting for any potential revenue from commercial sales of pz-cel, if approved, or proceeds from the sale of a Priority Review Voucher or PRV, if awarded by the FDA. Research and development expenses for the three months ended March 31, 2024 were $7.2 million, compared to $8.0 million for the same period of 2023. General and administrative expenses were $7.1 million for the three months ended March 31, 2024, compared to $4.0 million for the same period of 2023. Net loss was $31.6 million for the first quarter of 2024, or $1.16 loss per common share, including a change in the fair value of warrant liabilities due to remeasurement of the Company’s issued stock purchase warrants. Net loss in the first quarter of 2023 was $9.1 million, or $0.54 loss per common share. Conference Call Details The Company will host a conference call and webcast on Wednesday, May 15, 2024, at 8:30 a.m. ET, to discuss the first quarter results. To access the call, dial 888-506-0062 (U.S. toll-free) or 973-528-0011 (international) and Entry Code: 496484 five minutes prior to the start of the call. A live, listen-only webcast and archived replay of the call can be accessed on the Investors & Media section of Abeona’s website at . The archived webcast replay will be available for 30 days following the call. About Abeona Therapeutics Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing cell and gene therapies for serious diseases. Prademagene zamikeracel (pz-cel) is Abeona’s investigational autologous, COL7A1 gene-corrected epidermal sheets currently in development for recessive dystrophic epidermolysis bullosa. The Company’s fully integrated cell and gene therapy cGMP manufacturing facility served as the manufacturing site for pz-cel used in its Phase 3 VIITAL™ trial, and is capable of supporting commercial production of pz-cel upon FDA approval. The Company’s development portfolio also features AAV-based gene therapies for ophthalmic diseases with high unmet medical need. Abeona’s novel, next-generation AAV capsids are being evaluated to improve tropism profiles for a variety of devastating diseases. For more information, visit . Forward-Looking Statements This press release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties. We have attempted to identify forward-looking statements by such terminology as “may,” “will,” “believe,” “anticipate,” “expect,” “intend,” “potential,” and similar words and expressions (as well as other words or expressions referencing future events, conditions or circumstances), which constitute and are intended to identify forward-looking statements. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, numerous risks and uncertainties, including but not limited to, the timing and results of ongoing testing and other corrective actions being performed in response to the FDA’s identified deficiencies, which could delay the Company’s BLA resubmission; the timing and outcome of the FDA’s review of our resubmission; the FDA’s grant of a Priority Review Voucher upon approval; continued interest in our rare disease portfolio; our ability to enroll patients in clinical trials; the outcome of future meetings with the FDA or other regulatory agencies, including those relating to preclinical programs; the ability to achieve or obtain necessary regulatory approvals; the impact of any changes in the financial markets and global economic conditions; risks associated with data analysis and reporting; and other risks disclosed in the Company’s most recent Annual Report on Form 10-K and subsequent periodic reports filed with the Securities and Exchange Commission. The Company undertakes no obligation to revise the forward-looking statements or to update them to reflect events or circumstances occurring after the date of this press release, whether as a result of new information, future developments or otherwise, except as required by the federal securities laws. ABEONA THERAPEUTICS INC. AND SUBSIDIARIES Condensed Consolidated Statements of Operations and Comprehensive Loss (In thousands, except share and per share amounts) (Unaudited) For the three months ended March 31, 2024 2023 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 (expense) 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 gains (losses) related to available-for-sale debt securities (118 ) 64 Comprehensive loss $ (31,696 ) $ (9,043 ) ABEONA THERAPEUTICS INC. AND SUBSIDIARIES Condensed Consolidated Balance Sheets (In thousands, except share and per share amounts) (Unaudited) March 31, 2024 December 31, 2023 ASSETS 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 payable to licensor 4,691 4,580 Other current liabilities 1 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 Commitments and contingencies Stockholders' (deficit) equity: Preferred stock - $0.01 par value; authorized 2,000,000 shares; No shares issued and outstanding as of March 31, 2024 and December 31, 2023, respectively — — Common stock - $0.01 par value; authorized 200,000,000 shares; 27,550,693 and 26,523,878 shares issued and outstanding as of March 31, 2024 and December 31, 2023, respectively 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' (deficit) equity $ 74,828 $ 64,002 Investor and Media Contact: Greg Gin VP, Investor Relations and Corporate Communications Abeona Therapeutics ir@abeonatherapeutics.com

The FDA has rejected Abeona's topical gene therapy pz-cel because of manufacturing issues. Dating to a 2019 clinical hold of a phase 3 trial, getting its gene therapy rademagene zamikeracel (pz-cel) across the FDA finish line has been a challenge for troubled Abeona Therapeutics. Now the wait continues as the FDA has rejected the treatment, sending the Cleveland-based company a complete response letter (CRL), specifying chemistry, manufacturing and controls (CMC) issues that must be addressed. The topical treatment was targeted for approval on May 25 of this year for patients with recessive dystrophic epidermolysis bullosa (RDEP), a rare genetic condition that causes severe blistering of the skin. “While we are surprised and disappointed by this outcome—especially given the positive results of our two clinical studies—I want to say that we believe the CRL provides a clear roadmap to bring this important product to patients as soon as possible,” Abeona CEO Vishwas Seshadri, Ph.D., said during a conference call Tuesday morning. Seshadri, who became the company’s fifth CEO in a span of less than four years when he took over in 2021, was quick to point out that there were no efficacy or safety issues identified in the CRL about pz-cel. “And that is huge,” Seshadri said, adding that the company expects to submit CMC information to the FDA in the third quarter of this year, with the best-case scenario for approval being in the first quarter of 2025. After revealing the CRL in a press release on Monday afternoon, Abeona’s share price dropped by 47% by mid-morning on Tuesday. Abeona had hoped that a meeting with FDA on March 21 and commitments to provide CMC data before the PDUFA date and post-approval validation reports would keep pz-cel on track. But the FDA said in the CRL that the proposed timing of the data submission would not allow sufficient time for FDA to complete its review. “The necessary CMC information requested in the CRL pertains to certain validation requirements for manufacturing and release testing and includes observations that arose in the pre-license inspection,” Seshadri said. That inspection took place in February and resulted in a Form 483 which included observations related to process controls. After going into surprising detail on four CMC issues that need to be addressed, Seshadri said that with the exception of one assay “everything is standard, routine practice that is applicable to most, if not all, biological products and those assays need to be adapted to our matrix.” Less than a year ago, Krystal Biotech became the first company to win approval for a DEP treatment, scoring with Vyjuvek. The approval covers a more mild form of the disease. Abeona's effort with pz-cel is to treat the more severe recessive form of DEP.