Core Viewpoint - The FDA has accepted the Biologics License Application (BLA) resubmission for pz-cel, targeting a PDUFA action date of April 29, 2025, indicating significant progress towards potential commercialization of the therapy for recessive dystrophic epidermolysis bullosa (RDEB) [1][2][3]. Company Progress - Abeona Therapeutics has made strides in commercial readiness for pz-cel, including discussions with payors and onboarding treatment centers [2][3]. - The company completed a Type A meeting with the FDA in August 2024, aligning on the BLA resubmission content and addressing Chemistry Manufacturing and Controls (CMC) issues noted in a previous Complete Response Letter [3]. - The Centers for Medicare and Medicaid Services (CMS) granted a product-specific procedure code for pz-cel and assigned it to a high reimbursement category, facilitating hospital billing and patient access [3]. - Abeona has expanded its manufacturing capacity with a new facility lease in Cleveland, Ohio, and received new patents related to pz-cel, with the '504 Patent expiring on January 3, 2037 [3][4]. Financial Results - As of September 30, 2024, the company reported cash and cash equivalents totaling $110.0 million, down from $123.0 million as of June 30, 2024 [4]. - Research and development expenses for Q3 2024 were $8.9 million, an increase from $7.1 million in Q3 2023, while general and administrative expenses rose to $6.4 million from $4.2 million in the same period [6]. - The net loss for Q3 2024 was $30.3 million, compared to a net loss of $11.8 million in Q3 2023 [6][10]. Pipeline and Partnerships - In July 2024, Abeona entered a non-exclusive agreement with Beacon Therapeutics to evaluate its AAV204 capsid for potential use in ophthalmology gene therapies [3]. - Ultragenyx participated in a pre-BLA meeting with the FDA regarding its partnered program UX111 AAV gene therapy for Sanfilippo syndrome type A, expected to be filed by the end of 2024 [3].

Core Viewpoint - Abeona Therapeutics Inc. has announced the acceptance of its Biologics License Application (BLA) resubmission for prademagene zamikeracel (pz-cel) by the FDA, targeting a decision date of April 29, 2025, for its investigational gene therapy for recessive dystrophic epidermolysis bullosa (RDEB) [1][2] Company Overview - Abeona Therapeutics is a clinical-stage biopharmaceutical company focused on developing cell and gene therapies for serious diseases, with pz-cel being a key investigational product for RDEB [5] - The company operates a fully integrated cGMP manufacturing facility capable of supporting commercial production of pz-cel upon FDA approval [5] Product Details - Prademagene zamikeracel (pz-cel) is an autologous, COL7A1 gene therapy designed to treat RDEB, a rare genetic skin disease caused by mutations in the COL7A1 gene, leading to a lack of functional collagen VII [4] - Pz-cel is derived from patients' own skin cells, which are genetically corrected to express collagen VII, and expanded into keratinocyte sheets for wound coverage in a single surgical application [4] - The BLA resubmission is backed by clinical efficacy and safety data from the pivotal Phase 3 VIITAL™ study and a Phase 1/2a study, with follow-up data extending up to 8 years [2] Regulatory Status - The FDA had previously accepted the BLA for pz-cel for Priority Review, and the company may be eligible for a Priority Review Voucher if pz-cel is approved [3]

Company Overview - Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company focused on developing cell and gene therapies for serious diseases [3] - The company is developing an investigational autologous, COL7A1 gene-corrected epidermal sheet, prademagene zamikeracel (pz-cel), for recessive dystrophic epidermolysis bullosa [3] - Abeona has a fully integrated cell and gene therapy cGMP manufacturing facility that supports the production of pz-cel for its Phase 3 VIITAL™ trial and is capable of commercial production upon FDA approval [3] - The company is also working on AAV-based gene therapies for ophthalmic diseases with high unmet medical needs, utilizing next-generation AAV capsids to improve tropism profiles for various diseases [3] Upcoming Events - Members of Abeona's management team will participate in the Truist Securities BioPharma Symposium on November 7, 2024, for investor meetings [1] - A fireside chat at the Stifel 2024 Healthcare Conference is scheduled for November 18, 2024, at 4:10 p.m. ET, along with additional investor meetings [1][2] - A live webcast of the fireside chat will be available on the Investors section of the Abeona website, with a replay accessible for a limited time [2]

Core Viewpoint - Abeona Therapeutics has resubmitted its Biologics License Application (BLA) for prademagene zamikeracel (pz-cel) to the FDA as a potential treatment for recessive dystrophic epidermolysis bullosa (RDEB) [1][2] Group 1: BLA Resubmission Details - The resubmission follows a Type A meeting with the FDA in August 2024, where the company aligned on the content required for the resubmission [2] - The FDA's Complete Response Letter (CRL) from April 2024 did not identify deficiencies related to clinical efficacy or safety data, nor did it request new clinical trials [2][3] - The BLA resubmission is supported by clinical efficacy and safety data from the pivotal Phase 3 VIITAL™ study and a Phase 1/2a study [2] Group 2: Regulatory Process and Expectations - Upon acceptance of the BLA resubmission, the FDA will set a new Prescription Drug User Fee Act (PDUFA) target action date for pz-cel [3] - The review period for a resubmitted BLA can be either two or six months, depending on the classification as Class 1 or Class 2 [3] Group 3: Product Information - Prademagene zamikeracel (pz-cel) is an investigational autologous gene therapy targeting the COL7A1 gene, aimed at treating RDEB, a rare genetic skin disease [4] - The therapy involves genetically correcting patients' skin cells to express collagen VII, which is essential for skin integrity [4] - Pz-cel has received multiple designations from the FDA, including Regenerative Medicine Advanced Therapy and Breakthrough Therapy [4] Group 4: Company Overview - Abeona Therapeutics is a clinical-stage biopharmaceutical company focused on developing cell and gene therapies for serious diseases [5] - The company has a fully integrated cGMP manufacturing facility capable of supporting commercial production of pz-cel upon FDA approval [5] - Abeona's development portfolio also includes AAV-based gene therapies for ophthalmic diseases [5]

Company Overview - Abeona Therapeutics Inc. (NASDAQ: ABEO) specializes in developing gene and cell therapies aimed at treating serious genetic disorders [1] - The company utilizes its AIM platform, which employs adeno-associated virus (AAV) vectors for the targeted delivery of genetic material, facilitating the correction of functions across multiple body organs [1] Technology and Innovation - The AIM platform represents a significant technological advancement in the field of gene therapy, allowing for precise targeting and delivery of therapeutic genes [1]

Group 1 - Abeona Therapeutics Inc. will participate in several investor conferences in September 2024, including the Wells Fargo Healthcare Conference, H.C. Wainwright Annual Global Investment Conference, and Cantor Global Healthcare Conference [1] - The company will present its investigational autologous, COL7A1 gene-corrected epidermal sheets, known as prademagene zamikeracel (pz-cel), which is currently in development for recessive dystrophic epidermolysis bullosa [2] - Abeona's manufacturing facility is fully integrated and capable of supporting commercial production of pz-cel upon FDA approval, and it has a portfolio of AAV-based gene therapies for ophthalmic diseases [2]

Core Insights - Abeona Therapeutics Inc. has appointed Bernhardt G. Zeiher, MD, FCCP, FACP, and Eric Crombez, MD as new independent members of its Board of Directors, enhancing its leadership team during a critical period for the company [1][2] Group 1: Board Appointments - Dr. Zeiher has over 20 years of drug development experience and has overseen the approval of 15 new treatments for serious diseases with limited treatment options [1][2] - Dr. Crombez is currently the Chief Medical Officer at Ultragenyx Pharmaceutical Inc. and has extensive expertise in clinical development for rare genetic disorders [1][3] Group 2: Leadership Background - Dr. Zeiher previously held multiple roles at Astellas Pharma, culminating in his position as Chief Medical Officer, where he led drug development and regulatory affairs [2] - Dr. Crombez has a background in academia as an assistant professor at UCLA and has led clinical development efforts for gene therapy programs at Dimension Therapeutics [3] Group 3: Company Overview - Abeona Therapeutics is a clinical-stage biopharmaceutical company focused on developing cell and gene therapies for serious diseases, including its investigational therapy pz-cel for recessive dystrophic epidermolysis bullosa [4] - The company operates a fully integrated cGMP manufacturing facility capable of supporting commercial production of pz-cel upon FDA approval [4]

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, DC 20549 FORM 10-Q (Mark one) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended June 30, 2024 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) (State or other jurisdiction of incorporation ...

Financial Performance and Cash Position - The company reported cash, cash equivalents, short-term investments, and restricted cash totaling $123.0 million as of June 30, 2024, up from $62.7 million as of March 31, 2024[5] - Net cash used in operating activities was $12.7 million for the three months ended June 30, 2024[5] - Net income for Q2 2024 was $7.4 million, including a $24.9 million gain from the remeasurement of warrant liabilities, compared to a net loss of $16.7 million in Q2 2023[6] - Cash and cash equivalents increased to $34.426 million from $14.473 million, a 138% increase[14] - Short-term investments rose to $88.282 million from $37.753 million, a 134% increase[14] - Total current assets grew to $125.904 million from $55.737 million, a 126% increase[14] - Total assets increased to $134.003 million from $64.002 million, a 109% increase[14] - Total stockholders' equity rose to $73.241 million from $14.826 million, a 394% increase[14] - Additional paid-in capital grew to $846.654 million from $764.151 million, an 11% increase[14] - Accumulated deficit increased to $(773.696) million from $(749.524) million, a 3% increase[14] Expenses and Investments - Research and development expenses increased to $9.2 million in Q2 2024, compared to $8.5 million in the same period of 2023[6] - General and administrative expenses rose to $8.6 million in Q2 2024, primarily due to commercial and launch preparation costs, up from $5.0 million in Q2 2023[6] - Abeona closed a $75 million underwritten securities offering in May 2024, with participation from new and existing investors[4] - The company estimates its current cash resources, including a $50 million credit facility, are sufficient to fund operations into 2026[5] Product Development and Regulatory Updates - Abeona Therapeutics expects to resubmit the Biologics License Application (BLA) for pz-cel in the second half of 2024, with FDA alignment on CMC items[1][2] - Long-term safety data for pz-cel with up to 11 years of follow-up were presented at the Society for Investigative Dermatology Annual Meeting in May 2024[3] - Abeona entered a non-exclusive agreement with Beacon Therapeutics in July 2024 to evaluate its AAV204 capsid for ophthalmology gene therapies[4] Debt and Liabilities - Long-term debt was reported at $16.133 million, up from $0 in the previous period[14] - Warrant liabilities decreased to $24.100 million from $31.352 million, a 23% decrease[14] Shareholder Equity and Stock Issuance - Common stock shares issued and outstanding increased to 41,661,993 from 26,523,878, a 57% increase[14]

Significant progress addressing CMC items noted in CRL BLA resubmission remains on track for 2H 2024 CLEVELAND, Aug. 12, 2024 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO) today reported financial results for the second quarter of 2024 and recent corporate progress. “Having completed data generation for nearly all of the Chemistry Manufacturing and Controls deliverables outlined in the Complete Response Letter that we received in April 2024, we are on track to resubmit our Biologics License A ...