Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in gene and cell therapy, today announced that it has received Institutional Review Board (IRB) approval from Stanford University to commence the VIITAL study, the Companys pivotal Phase 3 clinical trial evaluating EB-101 for the treatment of recessive dystrophic epidermolysis bullosa (RDEB). The majority of patients targeted for enrollment have completed the pre-screening process at Stanford, and the Company expects the first patient in the VIITAL study to be treated in the first quarter of 2020.
We expect 2020 to be a transformational year at Abeona, and we are proud to start it with the initiation of our pivotal Phase 3 study evaluating EB-101 in RDEB, said Joo Siffert, M.D., Chief Executive Officer. We look forward to the first patient receiving EB-101 this quarter, setting in motion the final stages of this important program. We have devoted significant effort to establish and validate our independent, fully-functional GMP facility that will produce EB-101 for the VIITAL study and has capacity to support commercial launch. EB-101 has the potential to be the first approved therapy for RDEB and the only durable treatment to address large chronic wounds, which are the most painful and debilitating.
The VIITAL Phase 3 study is a multi-center, randomized clinical trial assessing EB-101 in up to 15 RDEB patients, with approximately 30 large, chronic wound sites treated in total. The primary study endpoint is the proportion of wounds with greater than 50% healing at three months, comparing treated with untreated wound sites on the same patient. Secondary endpoints include the patients global impression of change from baseline in pain as well as other patient reported outcomes assessing pain during dressing changes, pain impact and physical function. Investigators at Stanford University Medical Center are currently enrolling eligible patients into the VIITAL study, with additional study sites expected to be added in the coming months.
Abeona will produce EB-101 for the pivotal VIITAL study at the Elisa Linton Center for Rare Disease Therapies, its fully-functional gene and cell therapy manufacturing facility, centrally-located in Cleveland, OH. The center is a 26,000 ft2 facility housing large-scale cGMP capacity and state-of-the-art laboratories to support CMC development for process and analytics, all of which is validated and governed by comprehensive quality systems and overseen by experienced staff. The facility is also capable of clinical production of the Companys AAV gene therapies. 2019 Global Data Point.