Financial Performance - The net loss for the three months ended September 30, 2024, was $30.269 million, compared to a loss of $11.836 million for the same period in 2023, reflecting a 156% increase in losses[12]. - The net loss for the nine months ended September 30, 2024 and 2023, respectively, was $54.4 million and $37.6 million, with an accumulated deficit of approximately $804.0 million as of September 30, 2024[26]. - The basic and diluted loss per common share for the three months ended September 30, 2024, was $(0.63), compared to $(0.48) for the same period in 2023[12]. - The company has incurred significant operating losses and negative cash flows from operations since inception, which are expected to continue for the foreseeable future[25]. - The company has incurred recurring losses since inception and expects to continue generating operating losses for the foreseeable future[26]. Assets and Liabilities - As of September 30, 2024, total current assets increased to $112.657 million from $55.737 million as of December 31, 2023, representing a 102% increase[9]. - Total liabilities rose to $74.819 million from $49.176 million, marking a 52% increase[10]. - The total stockholders' equity increased significantly to $45.773 million from $14.826 million, representing a 208% increase[11]. - Total assets measured at fair value as of September 30, 2024, were $109,168 million, an increase from $72,305 million as of December 31, 2023[51]. - Total liabilities measured at fair value as of September 30, 2024, were $38,789 million, up from $31,352 million as of December 31, 2023[55]. Cash Flow and Investments - Cash and cash equivalents increased to $15.726 million as of September 30, 2024, from $14.473 million as of December 31, 2023, a rise of 9%[9]. - As of September 30, 2024, the Company had cash, cash equivalents, restricted cash, and short-term investments totaling $110.0 million, expected to fund operations for at least the next 12 months[26]. - The total cash, cash equivalents, and restricted cash at the end of the period is $16,064,000, compared to $5,050,000 at the end of the prior year[20]. - Total available-for-sale short-term investments amounted to $93.975 million as of September 30, 2024, with unrealized losses primarily due to changes in interest rates[42]. Research and Development - Research and development expenses for the nine months ended September 30, 2024, totaled $25.366 million, up from $23.712 million in the same period of 2023, indicating a 7% increase[12]. - The company is developing pz-cel, an autologous cell-based gene therapy for recessive dystrophic epidermolysis bullosa (RDEB)[21]. - The reimbursement of development and transition costs from Ultragenyx is recognized as a reduction of research and development costs[109]. Stock and Equity - The company issued 12,285,056 shares of common stock in connection with an underwritten offering, net of offering costs, during the reporting period[16]. - The Company sold 1,902,376 shares under the ATM Agreement during the nine months ended September 30, 2024, resulting in net proceeds of $10.0 million[84]. - The Company sold 3,284,407 shares and pre-funded warrants for an aggregate purchase price of $25.0 million gross on July 6, 2023[89]. - The Company sold 12,285,056 shares and pre-funded warrants for an aggregate purchase price of $75.0 million gross on May 7, 2024[90]. Revenue and Licensing - The company reported no license and other revenues for the three months ended September 30, 2024, compared to $3.5 million for the same period in 2023[12]. - The Company recognized no revenue from the sublicense agreements for the three and nine months ended September 30, 2024 and 2023[103]. - The transaction price for the sublicense agreement related to CLN1 disease includes $7.0 million of fixed consideration and up to $56.0 million of variable consideration[102]. - Under the Ultragenyx License Agreement, the Company is eligible to receive tiered royalties from mid-single-digit up to 10% on net sales and up to $30.0 million in commercial milestone payments[108]. Risks and Future Outlook - The company has highlighted potential risks including the timing and outcome of the FDA's review of its Biologics License Application for pz-cel, which could materially affect performance[7]. - The Company resubmitted its Biologics License Application (BLA) for pz-cel on October 28, 2024, with a target action date of April 29, 2025, set by the FDA[110]. - The BLA resubmission follows a Type A meeting with the FDA in August 2024, aligning on additional information required for Chemistry Manufacturing and Controls (CMC)[111]. Operating Costs and Expenses - Total operating lease costs for the nine months ended September 30, 2024, were $1,308 million, a decrease from $1,379 million in the same period of 2023[68]. - The Company incurred stock-based compensation expense of $4,673,000 for the nine months ended September 30, 2023, compared to $3,254,000 for the same period in 2022[20]. - Total stock-based compensation expense for the three months ended September 30, 2024 was $1.804 million, compared to $1.557 million for the same period in 2023[95].

Financial Performance - Abeona Therapeutics reported a net loss of $30.3 million for Q3 2024, compared to a net loss of $11.8 million in Q3 2023, reflecting an increase in losses due to higher expenses[8]. - Abeona's total expenses for Q3 2024 were $15.3 million, compared to $11.3 million in Q3 2023, indicating a significant increase in operational costs[12]. - Research and development expenses for Q3 2024 were $8.9 million, up from $7.1 million in the same period of 2023, while general and administrative expenses rose to $6.4 million from $4.2 million[8]. - Total stockholders' equity increased to $45,773 million as of September 30, 2024, compared to $14,826 million as of December 31, 2023[15]. - Accumulated deficit rose to $(803,965) million from $(749,524) million[15]. - Total liabilities and stockholders' equity reached $120,592 million, up from $64,002 million[15]. - Additional paid-in capital increased to $849,388 million from $764,151 million[15]. - Accumulated other comprehensive loss increased to $(84) million from $(66) million[15]. - Total shares issued and outstanding remained at 26,523,878 as of September 30, 2024, unchanged from December 31, 2023[15]. Cash Position - As of September 30, 2024, the company had cash, cash equivalents, short-term investments, and restricted cash totaling $110.0 million, down from $123.0 million as of June 30, 2024[6]. - Abeona's current cash resources are estimated to fund operations into 2026, excluding potential revenue from pz-cel sales[7]. Product Development and Regulatory Progress - The FDA accepted the resubmission of Abeona's Biologics License Application (BLA) for pz-cel, with a target action date set for April 29, 2025[1][4]. - Abeona has made significant progress in commercial readiness for pz-cel, including discussions with payors and onboarding treatment centers[2][4]. - The Centers for Medicare and Medicaid Services (CMS) assigned a product-specific procedure code for pz-cel, facilitating higher reimbursement levels for the therapy[3]. - The company has received new patents related to pz-cel, which are expected to enhance its intellectual property position in the market[3]. Operational Expansion - The company entered a lease agreement for additional manufacturing space in Cleveland, Ohio, to expand its production capacity[3].

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 ...