Abeona has announced topline results from its phase 3 VIITAL study of EB-101.
The autologous cell therapy EB-101 (Abeona Therapeutics) significantly improved wound healing and reduced pain in people with recessive dystrophic epidermolysis bullosa (RDEB), according to updated data from the phase 3 VIITAL study (NCT04227106).
The study met both its primary endpoints of achieving over 50% wound healing and achieving a greater magnitude of pain reduction benefit at 6 months after treatment compared to baseline and control wounds. Based off these data, Abeona Therapeutics plans to submit a biologics license application to the FDA in the second quarter of 2023.
“Large chronic RDEB wounds are the toughest to treat and often associated with intense chronic pain that significantly impacts the quality of life of RDEB patients, necessitating frequent use of opioids. In the Phase 3 VIITAL study, EB-101 has been shown to both heal such large chronic wounds and significantly reduce pain. And we continue to see durable clinical benefit of EB-101 with up to 8 years of follow-up in our Phase 1/2a study,” principal investigatorJean Tang, MD, PhD, Professor, Dermatology, Stanford University School of Medicine, said in a statement.
The VIITAL study evaluated EB-101's effect in 11 participants with RDEB with 43 large chronic wound pairs. These wounds were larger than 20 cm2 of surface area and had remained open for at least 6 months but no more than 21 years (mean, 6.2 years). Wounds randomized to be treated with EB-101 had an 81.4% rate of 50% or greater healing at 6 months while untreated control wounds had a 16.3% rate of 50% healing (P <.0001). The proportion of treated wounds achieving 75% or greater healing and complete healing was also statistically significant compared to control wounds.
Pain, associated with wound dressing, was measured by Wong-Baker FACES scale. Treated wounds had a mean 3.07 pain reduction score at 6 months from baseline while control wounds had a mean 0.90 reduction (P = .0002). A post-hoc analysis of more severe wounds (baseline score of at least 6) showed that treated severe wounds (n = 27) had a mean pain reduction of 5.70 compared to a reduction of 3.51 in all treated randomized and later unrandomized and treated wounds.
EB-101 was well-tolerated with no serious treatment-related adverse events (AEs) observed. These safety results are consistent with data from previous clinical studies. Investigators observed no positive replication-competent retrovirus cases, squamous cell carcinoma, or systemic immunologic responses and there were no deaths. Two participants experienced serious AEs unrelated to treatment and 8 participants had infections unrelated to treatment. Four participants had treatment-emergent AEs including procedural pain, muscle spasms, and pruritis.
“I am incredibly enthused to see new clinical evidence of EB-101’s potential to treat the more difficult chronic and large wounds. Our patient community needs options to address not only the healing of wounds but also the chronic pain and the acute treatment related pain of daily wound care associated with these wounds. Today’s standard of care comprises hours of brutal and painful wound care, and EB-101’s promise to be a transformational option for RDEB patients is truly exciting,” Brett Kopelan, Executive Director, debra of America, added to the statement. Kopelan is father to Rafi, a 15-year-old with RDEB.
Abeona will present further results from the study including additional endpoints and week 12 results at a future medical meeting and for publication in a peer-reviewed journal. Week 12 results also achieved statistical significance in pain reduction and wound healing.