Abeona Therapeutics Reinitiates Enrollment in EB-101 Pivotal Phase 3 VIITAL Study in RDEB after COVID-19 Related Pause and Announces Progress in Patient Enrollment in MPS III Studies

NEW YORK and CLEVELAND, July 08, 2020 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in gene and cell therapy, today announced recent updates on its clinical programs, highlighted by the restart of patient enrollment in the Company’s pivotal Phase 3 VIITAL study of EB-101, its autologous, gene-corrected cell therapy, for the treatment of recessive dystrophic epidermolysis bullosa (RDEB). Enrollment in the VIITAL study at Stanford University Medical Center was paused in March 2020 in order to redirect healthcare resources to COVID-19 patients and to ensure the safety of study participants and site staff.

“RDEB is a debilitating and life-threatening rare genetic disorder without an approved treatment option. Reinitiating enrollment in the VIITAL study is important for patients with RDEB and brings us a step closer toward concluding the clinical development of EB-101,” said João Siffert, M.D., Chief Executive Officer of Abeona. “The unprecedented COVID-19 global health crisis has had a broad impact across our industry, and we will continue to monitor the pandemic to ensure the safety of trial participants, healthcare professionals and our employees.”

Abeona’s MPS III Phase 1/2 Clinical Trials Continue Despite COVID-19 Pandemic

Abeona also announced that two ongoing MPS III Phase 1/2 clinical trials of its investigational adeno-associated virus (AAV)-based gene therapies, ABO-102 and ABO-101, have continued to treat patients, with additional enrollment expected in the programs in the coming weeks. In June 2020, the 10^th patient was dosed in cohort 3 of the ABO-102 Transpher A study for MPS IIIA (Sanfilippo syndrome type A), bringing the total to 16 patients. The Company previously reported in May 2020 that additional patients were treated in dose cohort 3 of the Transpher A study and the ABO-101 Transpher B study for MPS IIIB (Sanfilippo syndrome type B), bringing the total enrollment in the Transpher B study to 9 children to date.

Dr. Siffert added, “We have worked closely with sites in our MPS III clinical studies to continue to enroll patients given the urgent need to treat children with Sanfilippo syndrome as early as possible. We are encouraged by the number of new pre-screened patients that may meet enrollment criteria and could allow dosing in the coming weeks. We also hope to resume on-site follow-up study visits that include neurocognitive assessments of already enrolled patients.”