Abeona Therapeutics Inc.

What they do

Abeona manufactures ZEVASKYN (prademagene zamikeracel) at its cGMP facility in Cleveland, Ohio and makes it available through a network of ZEVASKYN qualified treatment centers. The FDA approved ZEVASKYN on April 28, 2025 as the first and only autologous cell-based gene therapy for RDEB wounds in adults and pediatric patients. The company's preclinical portfolio uses novel AIM capsids in AAV gene therapies, including ABO-504 for Stargardt disease, ABO-503 for X-linked retinoschisis and ABO-505 for autosomal dominant optic atrophy. As of August 2026, the ZEVASKYN qualified treatment center network includes seven sites nationwide.

Revenue drivers

• ZEVASKYN product revenue — Net product revenue from commercial sales of ZEVASKYN was $2.4 million for full-year 2025 and $11.4 million in Q2 2026, up 31% from $8.7 million in Q1 2026. This is now the company's principal revenue source.
• License and other revenues — License and other revenue was $3.4 million in 2025, primarily a clinical development milestone under an October 2020 Taysha sublicense for an investigational AAV gene therapy for Rett syndrome, plus $0.4 million from a third party option exercise on certain AAV capsids. There was no license or other revenue in Q2 2026.
• Priority review voucher gain — The 2025 results include a $152.4 million net gain from sale of a priority review voucher, a non-recurring item that drove reported net income, not product sales.

Recent performance

Q2 2026 net ZEVASKYN revenue was $11.4 million, up 31% from $8.7 million in Q1 2026. Five patients were treated in Q2 2026 and revenue was recognized for four treatments because one batch yielded fewer than the threshold number of sheets for revenue recognition. Net loss was $(20.2) million, or $(0.35) per basic and diluted share, compared to a net loss of $(17.1) million in Q1 2026. Total costs and expenses were $25.0 million in Q2 2026, including $5.0 million of R&D and $15.8 million of SG&A. Cash, cash equivalents and short-term investments were $146.8 million as of June 30, 2026, down from $191.4 million at December 31, 2025.

Strategy

Management's stated priorities are commercializing ZEVASKYN, advancing and commercializing its cell and gene therapy programs, and developing in-vivo gene therapies using AIM capsid technology. Abeona is expanding the ZEVASKYN qualified treatment center network, which reached seven active sites, and is progressing patients through biopsy collection and treatment. The company in-licensed ABO-701 (PSMA-SIR-T), an engineered T-cell therapy targeting PSMA that it describes as potentially first-in-class, incurring a one-time $7.0 million upfront cost in Q1 2026. Abeona intends to commercialize ZEVASKYN itself and may form strategic partnerships for ZEVASKYN and other assets subject to FDA approval.

Risks

• Commercial execution risk — The company states it may not successfully manufacture or commercialize ZEVASKYN and that revenue from its sales may be limited; Q2 2026 revenue was recognized for only four of five treated patients due to manufacturing yield and lot release issues.
• Dependence on a single product — Abeona's financial performance depends on the commercial success of ZEVASKYN, and the company has limited experience as a commercial-stage company.
• Qualified treatment center network — The company may encounter challenges engaging or coordinating with the qualified treatment centers needed for ongoing ZEVASKYN commercialization; as of the 10-K it had 4 activated sites, growing to 7 by mid-2026.
• Pipeline development risk — Abeona's cell and gene therapy candidates are based on proprietary methodologies, the regulatory requirements for such products have evolved and may continue to change, and clinical studies may be delayed or fail to demonstrate safety and efficacy.

Outlook

Management says it expects early real-world experience with ZEVASKYN to drive broader adoption and long-term growth as more activated sites mature. The company expects R&D activities to increase as it advances pipeline programs. ZEVASKYN received CMS New Technology Add-On Payment status effective October 1, 2026, which management expects to support hospital adoption and access for Medicare patients, who it estimates represent approximately 10% of RDEB patients.