CLEVELAND, Dec. 15, 2025 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO) today announced the appointment of Mohamad Tabrizi, M.S., M.B.A., as Senior Vice President, Chief Business Officer (CBO). In this role, Mr. Tabrizi will lead the Company’s corporate strategy and business development functions, as well as drive operating efficiency for the Company. “Mohamad brings a wealth of experience in strategic planning and business development,” said Vish Seshadri, Chief Executive Officer of Abeona. “W ...

CLEVELAND, Dec. 11, 2025 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO) today announced activation of The University of Texas Medical Branch (UTMB) in Galveston, Texas, as the newest Qualified Treatment Center (QTC) for the administration of ZEVASKYN (prademagene zamikeracel) gene-modified cellular sheets. ZEVASKYN, a first-of-its-kind, FDA-approved autologous, gene therapy is indicated for the treatment of wounds in adult and pediatric patients with recessive dystrophic epidermolysis bullosa ( ...

Core Insights - Abeona Therapeutics Inc. has announced the first commercial patient treatment with its FDA-approved gene therapy ZEVASKYN, aimed at treating wounds in patients with recessive dystrophic epidermolysis bullosa (RDEB) [1][2] - ZEVASKYN is the first and only autologous cell sheet-based gene therapy for RDEB, which is characterized by severe skin wounds due to a defect in the COL7A1 gene [4][5] - The treatment has shown clinically meaningful wound healing and pain reduction with a single surgical application [4] Company Overview - Abeona Therapeutics is a commercial-stage biopharmaceutical company focused on developing cell and gene therapies for serious diseases, with ZEVASKYN being a key product in its portfolio [8] - The company operates a fully integrated cGMP manufacturing facility in Cleveland, Ohio, which is responsible for the commercial production of ZEVASKYN [8] Treatment Details - ZEVASKYN incorporates the COL7A1 gene into a patient's skin cells to produce functional type VII collagen, essential for skin integrity [4] - The therapy is indicated for both adult and pediatric patients suffering from RDEB [5] - Abeona Assist® is a comprehensive patient support program that helps patients understand insurance benefits and provides logistical assistance [2]

Summary of Abeona Therapeutics Conference Call Company Overview - **Company**: Abeona Therapeutics (NasdaqCM:ABEO) - **Industry**: Biotechnology, specifically focused on cell and gene therapy - **Product**: ZivaSkin, approved for treating recessive dystrophic epidermolysis bullosa (RDEB) [2][80] Key Points and Arguments Product Launch and Demand - ZivaSkin was approved in Q2 2025, and the company is making significant progress towards its U.S. launch [2][80] - Patient demand is strong, with favorable coverage from payers and interest from Centers of Excellence (QTCs) [2][80] - The company has received 12 ZivaSkin Product Order Forms (ZPOFs) from identified patients, indicating a good conversion rate [28][29] Manufacturing and Sterility Testing - A temporary pause was placed on patient biopsy collection to optimize a sterility test after a false positive was detected during the first manufacturing run [3][4] - The FDA required a more robust sterility testing process, which has now been validated, reducing the risk of false positives from 35% to less than 1% [19][20] - The shelf life of the manufactured product is 84 hours, necessitating careful scheduling of surgeries [13][20] Patient Treatment and Logistics - The company is working to schedule surgeries within a tight timeframe, with a focus on maximizing patient logistics [23][24] - The average time from patient identification to treatment is expected to decrease from 3-4 months to 2-3 months as payer policies improve [31][32] Expansion of Treatment Centers - Abeona aims to activate 5-7 QTCs by 2026, with ongoing discussions with hospitals interested in becoming treatment centers [46][47] - The company is strategically considering geographic locations to ensure broad access for patients [47] Reimbursement and Coverage - A permanent J Code has been received, which is expected to streamline the reimbursement process for hospitals [58][60] - The payer mix shows that 60% of patients are commercially insured, with baseline coverage established for all 50 states [59][60] Future Growth and Capacity - The company plans to increase manufacturing capacity from 6 to 10 slots per month by mid-2026, with potential for further expansion [72][73] - The total addressable market (TAM) is estimated at 750 patients, with an expectation that each patient may require an average of two treatments [75][76] Pipeline and Financial Outlook - Abeona has a gene therapy platform with ongoing preclinical assets, including ABO 503, which has been selected for a rare disease pilot program by the FDA [78][79] - The company reported $207.5 million in cash, providing a two-year operating runway, with expectations to become profit-generating in the first half of 2026 [80] Additional Important Information - The company is focused on building relationships with Centers of Excellence to enhance patient access and treatment options [54][55] - There is a positive outlook on the competitive landscape, as ZivaSkin is an approved product, which may favor its utilization over investigational products [54][56]

Core Insights - Abeona Therapeutics Inc. reported strong figures for Q3 2025, with a GAAP EPS of -$0.10, surpassing expectations by $0.18, leading to a 22% increase in stock price post-announcement [1] Financial Performance - The company achieved a GAAP EPS of -$0.10 for Q3 2025, which was better than the anticipated figure by $0.18 [1] - The stock price rose by 22% following the announcement of these results [1] Company Background - Abeona Therapeutics is focused on biotechnology, particularly in drug development, with an emphasis on innovative therapies and unique mechanisms of action [1]

Core Viewpoint - Abeona Therapeutics' stock surged over 25% following strong third-quarter earnings and positive updates regarding its treatment for Recessive Dystrophic Epidermolysis Bullosa (RDEB), Zevaskyn [1][7]. Group 1: Treatment Overview - RDEB is a rare inherited skin disorder with an incidence of 3.05 per million births and a prevalence of 1.35 per million, leading to a high mortality rate among sufferers due to complications [2]. - Zevaskyn is a gene therapy derived from a patient's own skin, modified to produce a healthy collagen gene, and applied to wounds [3]. Group 2: Patient and Coverage Updates - Management identified 30 patients at treatment centers, with 12 patients currently scheduling treatments, indicating strong interest in the therapy among RDEB patients [5]. - Major commercial insurers, including UnitedHealthcare, Cigna, Aetna, Anthem, and most Blue Cross Blue Shield plans, have published policies covering Zevaskyn, enhancing treatment accessibility [5]. Group 3: Market Reaction and Future Outlook - The market has largely ignored a one-quarter delay in treatment initiation due to a resolved issue, with Abeona expected to start patient treatments this quarter [6]. - Significant revenue growth is anticipated in 2026 as treatment progresses [6].

Financial Data and Key Metrics Changes - As of September 30, 2025, the company had cash, cash equivalents, restricted cash, and short-term investments totaling $207.5 million, providing significant financial flexibility for the ZEVASKYN commercial launch [15] - Research and development (R&D) spending for Q3 2025 was $4.2 million, a decrease from $8.9 million in Q3 2024, primarily due to costs capitalized into inventory and reclassification of certain costs to selling, general, and administrative expenses (SG&A) [16] - SG&A expenses increased to $19.3 million in Q3 2025 from $6.4 million in Q3 2024, reflecting the reclassification of R&D expenses and increased costs associated with the commercial launch [16] - The net loss for Q3 2025 was $5.2 million, or -$0.10 per share, compared to a net loss of $30.3 million, or -$0.63 per share, in Q3 2024 [16] Business Line Data and Key Metrics Changes - The ZEVASKYN commercial launch is progressing, with growing patient demand and the activation of a third qualified treatment center (QTC), Children's Hospital Colorado [5][12] - The number of identified eligible patients at QTCs has more than doubled to approximately 30, up from over 12 previously reported [11] - The company has received ZEVASKYN Product Order Forms (ZPOFs) for 12 patients, indicating strong interest and movement towards treatment [10] Market Data and Key Metrics Changes - ZEVASKYN has received coverage decisions from all major commercial payers, covering over 80% of commercially insured lives, and has baseline coverage across all 51 state Medicaid programs [13] - A permanent product J-code for ZEVASKYN will be established by CMS effective January 1, 2026, which is expected to simplify claims and reimbursement processing [14] Company Strategy and Development Direction - The company is focused on scaling the ZEVASKYN commercial launch to meet patient demand and expanding its QTC network [5] - The management is actively discussing the onboarding of additional EB centers across the U.S. to further expand ZEVASKYN's geographic footprint [12] - The company aims to achieve profitability in the first half of 2026, despite the delay in the first patient treatment [30] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in achieving 2026 launch goals based on trends in patient demand and market access, despite a temporary delay in the first patient treatment [5][6] - The management highlighted the importance of maintaining high-quality standards in manufacturing personalized drug products [8] - The company is encouraged by the doubling of identified patients and the favorable payer landscape for ZEVASKYN [19] Other Important Information - The company has paused collecting additional patient biopsies to investigate and optimize a release assay that encountered performance issues [9] - The gene therapy program for X-linked retinoschisis, ABO-503, has been selected for the FDA Rare Disease Endpoint Advancement Pilot Program, which may accelerate development [18] Q&A Session Summary Question: Expected timeline for patients to receive treatment - Management indicated that several patients have already been scheduled for biopsy in November and early 2026, with treatment expected to follow if all paperwork is completed [24][26] Question: Impact on profitability timeline due to treatment delays - Management does not foresee a significant impact on the timeline for achieving profitability, maintaining guidance for the first half of 2026 [30] Question: Status of routine maintenance shutdown - The company confirmed a planned shutdown for routine maintenance starting mid-December and lasting about a month [34][35] Question: Biopsy collection during optimization pause - No biopsies were collected during the pause; the company prioritized resolving the assay issue before proceeding [36] Question: Lead time for ZEVASKYN Product Order Forms - Management expects the lead time to decrease as more patients progress through the treatment process and payer policies become established [39][42] Question: Attrition rate among patients with ZPOFs - Management anticipates a high conversion rate for patients with ZPOFs, as these are motivated patients who have expressed interest in treatment [47][49] Question: Prior authorization process for RDEB patients - The prior authorization process involves clinical and financial discussions, with most payers following inclusion/exclusion criteria from clinical trials [52] Question: Revenue recognition timeline - Revenue is recognized when the product is administered to the patient, with cash flow considerations varying by site [58]

Financial Data and Key Metrics Changes - As of September 30, 2025, the company had cash, cash equivalents, restricted cash, and short-term investments totaling $207.5 million, providing significant financial flexibility for the ZEVASKYN commercial launch [15] - Research and development (R&D) spending for Q3 2025 was $4.2 million, a decrease from $8.9 million in Q3 2024, primarily due to costs capitalized into inventory and reclassification of certain costs to selling, general, and administrative expenses (SG&A) [16] - SG&A expenses increased to $19.3 million in Q3 2025 from $6.4 million in Q3 2024, reflecting the reclassification of R&D expenses and increased costs associated with the commercial launch [16] - The net loss for Q3 2025 was $5.2 million, or negative $0.10 per share, compared to a net loss of $30.3 million, or negative $0.63 per share, in Q3 2024 [16] Business Line Data and Key Metrics Changes - The ZEVASKYN commercial launch is progressing, with the first patient treatment delayed to Q4 2025 due to optimization of a product release assay, but demand is growing [5][6] - The number of identified eligible patients at qualified treatment centers (QTCs) has more than doubled to approximately 30, up from over 12 previously reported [11] - The activation of Children's Hospital Colorado as a QTC brings the total to three, alongside Lurie Children's Hospital of Chicago and Lucille Packard Children's Hospital Stanford [12] Market Data and Key Metrics Changes - ZEVASKYN has received positive coverage decisions from major commercial payers, covering over 80% of commercially insured lives, and has baseline coverage across all 51 state Medicaid programs effective October 1, 2025 [13] - A permanent product J-code for ZEVASKYN will be established by CMS effective January 1, 2026, simplifying claims and reimbursement processing [14] Company Strategy and Development Direction - The company is focused on scaling the ZEVASKYN commercial launch to meet patient demand and expanding its QTC network [5][6] - The management team has been strengthened with the appointment of Dr. James A. Gao as Senior Vice President, Head of Clinical Development and Medical Affairs, to advance the pipeline [18] Management's Comments on Operating Environment and Future Outlook - Management remains confident in achieving 2026 launch goals despite the delay in the first patient treatment, citing trends in patient demand and market access [5][6] - The company expects to maintain a profitable business in the first half of 2026, with no significant impact from the treatment delay [29] Other Important Information - The company paused collecting additional patient biopsies to investigate a performance issue with a release assay, resuming in November 2023 [9] - The company plans to participate in the Stifel 2025 Healthcare Conference following the earnings call [16] Q&A Session Summary Question: Expected timeline for patients to receive treatment - Management indicated that several patients have already received prior authorizations and are scheduled for biopsies in November and early 2026 [24] Question: Impact on profitability timeline due to treatment delay - Management does not foresee a significant impact on the timeline to profitability, maintaining guidance for the first half of 2026 [29] Question: Routine maintenance shutdown of the plant - A shutdown for routine maintenance is scheduled for mid-December to early January, as mandated by the FDA [32] Question: Current lead time for ZEVASKYN Product Order Forms - The current lead time is about three months, but management expects this to decrease as more patients progress through the process [36] Question: Attrition rate among patients with ZEVASKYN Product Order Forms - Management expects a high conversion rate among patients who have received ZPOFs, as these are motivated patients [44] Question: Prior authorization process for RDEB patients - The prior authorization process involves clinical and financial discussions, with most payers following inclusion/exclusion criteria from clinical trials [50] Question: Biopsies and sterility release criteria - Management confirmed that they have resumed biopsies and are not guiding on the number of biopsies completed yet [54] Question: Patients on background Vyjuvek or Filsuvez - Management does not have visibility into the exact number of patients currently on these treatments but expects many to be [58] Question: UMass not listed as a QTC - Different sites have various reasons for not being onboarded, including financial constraints and trade policy differences [62]

Financial Data and Key Metrics Changes - As of September 30, 2025, the company reported cash, cash equivalents, restricted cash, and short-term investments totaling $207.5 million, providing significant financial flexibility for operations over the next two years [13][14] - Research and development (R&D) spending for Q3 2025 was $4.2 million, a decrease from $8.9 million in Q3 2024, primarily due to costs capitalized into inventory [14] - Selling, general and administrative (SG&A) expenses increased to $19.3 million in Q3 2025 from $6.4 million in Q3 2024, reflecting the reclassification of R&D expenses and increased costs associated with the commercial launch [15] - The net loss for Q3 2025 was $5.2 million, or negative $0.10 per share, compared to a net loss of $30.3 million, or negative $0.63 per share, in Q3 2024 [15] Business Line Data and Key Metrics Changes - The company is focused on the commercial launch of ZEVASKYN, the first autologous cell-based gene therapy for recessive dystrophic epidermolysis bullosa (RDEB), with growing patient demand and an expanding network of qualified treatment centers (QTCs) [4][10] - The number of identified eligible patients at QTCs has more than doubled to approximately 30, up from over 12 previously reported [10] - The activation of Children's Hospital Colorado as a QTC brings the total to three, alongside Lurie Children's Hospital of Chicago and Lucille Packard Children's Hospital Stanford [11] Market Data and Key Metrics Changes - ZEVASKYN has received coverage decisions from all major commercial payers, covering over 80% of commercially insured lives, and has baseline coverage across all 51 state Medicaid programs effective October 1, 2025 [12] - A permanent product J-code for ZEVASKYN will be established by CMS effective January 1, 2026, simplifying claims and reimbursement processing [12] Company Strategy and Development Direction - The company aims to achieve its 2026 launch goals for ZEVASKYN despite a temporary delay in the first patient treatment, focusing on expanding treatment center networks and ensuring market access [4][5] - The company is also advancing its pipeline, with a gene therapy program for X-linked retinoschisis selected for the FDA Rare Disease Endpoint Advancement pilot program [17] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the ability to meet 2026 launch goals based on trends in patient demand and treatment center expansion, despite the delay in the first patient treatment [4][5] - The management team highlighted the importance of maintaining high-quality standards in manufacturing and the proactive measures taken to optimize release assays [6][7] Other Important Information - The company has strengthened its management team with the appointment of Dr. James A. Gao as Senior Vice President, Head of Clinical Development and Medical Affairs, bringing over 20 years of industry experience [17] Q&A Session Summary Question: Expected timeline for patients to receive treatment after ZEVASKYN Product Order Forms - Management indicated that many patients have already received prior authorizations and are scheduled for biopsies in November and early 2026, with treatment expected to follow if all paperwork is completed [20][22] Question: Impact of treatment delays on profitability timeline - Management stated that the timeline for achieving profitability remains unchanged, with expectations for profitability in the first half of 2026 [26][28] Question: Routine maintenance shutdown of the plant - Management confirmed a mandated FDA-required shutdown for general maintenance from mid-December to early January [29][30] Question: Status of biopsies collected before optimization pause - Management clarified that no biopsies were collected during the pause to ensure patient safety and product quality [31] Question: Lead time for ZEVASKYN Product Order Forms - Management expects the lead time to decrease as more patients progress through the treatment process, currently estimated at about three months [34] Question: Attrition rate among patients with ZEVASKYN Product Order Forms - Management expressed confidence in a high conversion rate for patients with ZPOFs, as these are motivated patients eager to proceed with treatment [40][41] Question: Prior authorization process for RDEB patients - Management described the prior authorization process as involving clinical and financial discussions, with most payers following inclusion/exclusion criteria from clinical trials [44][46]

Financial Performance - Net loss for the three months ended September 30, 2025, was $5.16 million, a decrease from a loss of $30.27 million in the same period of 2024[16] - For the nine months ended September 30, 2025, the net income was $91,643 thousand, compared to a net loss of $54,441 thousand for the same period in 2024[23] - As of September 30, 2025, the Company reported a net loss of $5.161 million for the three months ended, compared to a net loss of $30.269 million for the same period in 2024[39] - The basic net loss per share for the three months ended September 30, 2025, was $(0.10), while for the nine months ended, it was $1.76, compared to $(1.41) in 2024[39] - The Company has not generated any product revenue to date and continues to incur significant expenses and operating losses[114] Assets and Liabilities - Total current assets increased to $216.13 million as of September 30, 2025, compared to $100.85 million as of December 31, 2024, representing a 114% increase[14] - Total liabilities decreased to $59.86 million as of September 30, 2025, from $64.90 million as of December 31, 2024, reflecting an 8% decline[14] - Cash and cash equivalents rose significantly to $82.88 million as of September 30, 2025, compared to $23.36 million as of December 31, 2024, marking a 255% increase[14] - The total stockholders' equity increased to $171.23 million as of September 30, 2025, from $44.03 million as of December 31, 2024, representing a 288% increase[14] - Cash and cash equivalents at the end of the period were $82,884 thousand, up from $15,726 thousand at the end of the same period in 2024[23] Revenue and Income - The company reported license and other revenues of $400,000 for the nine months ended September 30, 2025, compared to no revenues in the same period of 2024[16] - The company recognized $0.4 million in revenue from a licensing agreement during the nine months ended September 30, 2025, with no revenue recognized in the same period of 2024[103] - The Company has not recognized any sales-based or royalty revenue from its licensing arrangements[109][111] Research and Development - Research and development expenses for the nine months ended September 30, 2025, were $20.10 million, down from $25.37 million in the same period of 2024, indicating a 21% reduction[16] - Total research and development costs for the three months ended September 30, 2025 were $4.2 million, a decrease from $8.9 million in the same period of 2024[115] Cash Flow and Financing - The company reported a net cash used in operating activities of $58,373 thousand for the nine months ended September 30, 2025[23] - The company generated $152,366 thousand from the sale of a priority review voucher, significantly impacting cash flows[23] - The company reported a net cash provided by financing activities of $22,426 thousand for the nine months ended September 30, 2025[23] Stock and Equity - The weighted average number of common shares outstanding increased to 54,242,507 for the three months ended September 30, 2025, compared to 48,081,758 for the same period in 2024, indicating a 13% increase[16] - The company has a total of 13,792,969 potential dilutive securities that could affect future earnings per share calculations[40] - The company had 3,280,169 shares available to be granted under the 2023 Equity Incentive Plan as of September 30, 2025[10] - The total fair value of restricted stock awards that vested during the nine months ended September 30, 2025, was $6.5 million, compared to $4.5 million in 2024[94] Expenses - Total selling, general and administrative costs for the three months ended September 30, 2025 were $19.3 million, compared to $6.4 million in the same period of 2024[115] - Total stock-based compensation expense for the nine months ended September 30, 2025, was $8,220 thousand, an increase from $4,673 thousand in 2024[23] - The company recorded total operating lease costs of $520,000 for the three months ended September 30, 2025, compared to $416,000 for the same period in 2024[63] Future Outlook - The company expects its capital resources to be sufficient to fund ongoing operations for the next 12 months[27] - The Company is currently assessing potential limitations on the utilization of its net operating loss carryforwards due to multiple ownership changes[119] Regulatory and Compliance - The company capitalized inventory costs related to ZEVASKYN following FDA approval in April 2025, marking a shift in accounting treatment[32] - The Company entered into a sublicense agreement with Taysha for a gene therapy for Rett syndrome, with a transaction price including $3.0 million fixed consideration and up to $56.5 million in variable consideration[109] - Under the exclusive license agreement with Ultragenyx for ABO-102, 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[111]