Abeona Therapeutics Reports First Quarter Financial Results and Business Updates

First patient treated in ongoing pivotal Phase 3 VIITAL study evaluating EB-101 for RDEB

Additional patients dosed in the Phase 1/2 gene therapy studies for MPS IIIA and MPS IIIB

Positive interim data from MPS III gene therapy programs presented at WORLDSymposium; updated results to be presented at ASGCT 2020

Appointed two industry leaders to Board of Directors and strengthened management team

Company to host investor conference call Thursday, May 7, 2020 at 8:30 a.m. ET

NEW YORK and CLEVELAND, May 06, 2020 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in gene and cell therapy, today announced first quarter 2020 financial results, which will be discussed on a conference call scheduled for Thursday, May 7, 2020 at 8:30 a.m. ET. Interested parties are invited to participate in the call by dialing 844-455-1352 (U.S. toll-free) or 509-844-0155 (international), and reference conference ID 5352629, or via webcast at https://investors.abeonatherapeutics.com/ir-calendar.

“During these challenging times, our priority remains to ensure the safety of our employees and patients, while supporting continuity of our business and clinical operations,” said João Siffert, M.D., Chief Executive Officer of Abeona. “We have made considerable progress in our clinical programs in the first quarter of 2020, and are working with our investigators to minimize the impact of the COVID-19 pandemic. It is our intention to restore full patient access to our clinical programs as soon as possible. Our gene and cell therapies in development aim at addressing urgent unmet needs, and have the potential to provide durable benefit to patients who have no approved treatments.”

First Quarter and Recent Highlights

• First patient treated in pivotal Phase 3 VIITAL study evaluating EB-101 for recessive dystrophic epidermolysis bullosa (RDEB). An additional 10 patients have been prescreened for this study.
• Additional patients treated in dose cohort 3 of the Transpher A study and the Transpher B study.
• Presented positive interim data from the Transpher A study of ABO-102 at WORLDSymposium demonstrating improved neurocognitive skills 18 months to two years post-treatment in MPS IIIA patients younger than 30 months, sustained, dose-related biomarker improvements, and a favorable safety profile.
• Presented positive interim data from the Transpher B study of ABO-101 at WORLDSymposium demonstrating initial improvement in multiple disease-specific biomarkers, denoting clear biologic effects, and a favorable safety profile among MPS IIIB patients.
• Updated interim results from the Transpher A and Transpher B studies to be presented during the American Society of Gene & Cell Therapy (ASGCT) 23rd Annual Meeting, which will take place online May 12-15, 2020.
• Two U.S. patents issued for adeno-associated virus (AAV) capsids exclusively licensed by Abeona from the University of North Carolina (“UNC”), generated using UNC’s AIM vector platform.
• Announced key appointments of industry leaders to its Board of Directors in April. Dr. Brian J. G. Pereira was appointed as Executive Chairman and Ms. Shawn Tomasello as an Independent Board Member. Dr. Pereira is a seasoned biopharmaceutical and healthcare leader with experience in financing and growing companies, including the clinical development and commercialization of innovative drug products. Ms. Tomasello has substantial commercial and strategic experience, including serving as Chief Commercial Officer at cell therapy pioneer Kite Pharma, which was acquired by Gilead Sciences.
• Strengthened its leadership team with the appointments of Gregory Gin as Vice President, Investor Relations and Dr. Dan Rudin as Vice President, Clinical Development, focusing on the EB-101 program. Mr. Gin brings more than 25 years of investor relations, communications, and capital markets experience with small- and mid-cap biotechnology and specialty pharmaceutical companies developing novel treatments for orphan diseases and areas of high unmet medical need. Dr. Rudin has substantial research and development experience gained in industry and academia with focus on rare diseases, including lysosomal storage diseases. He has led several programs through the lifecycle of clinical development supporting multiple product approvals.

Dr. Siffert continued, “We look forward to working with our new Executive Chairman, Dr. Brian Pereira and Independent Board Member, Ms. Shawn Tomasello. Both bring invaluable experience guiding biotech companies from clinical development through commercial launch. In addition, with the appointments of Greg and Dan, we have strengthened our leadership in investor relations and clinical development, respectively.”