Phase III GEM-3 study of B VEC shows continued benefits in dystrophic epidermolysis bullosa.
Krystal Biotech presented detailed results from the GEM-3 Phase III study of B VEC (beremagene geperpavec), an investigational, topical gene therapy, for the treatment of dystrophic epidermolysis bullosa (DEB), at the 2022 American Academy of Dermatology Annual Meeting in Boston, Mass.
The data was presented by Peter Marinkovich, M.D., Director of the Blistering Disease Clinic at Stanford Health Care and Associate Professor of Dermatology at the Stanford University School of Medicine and primary investigator of the GEM-3 study, during the late-breaking research session.
The GEM-3 trial was a multicenter, randomized, double-blind, placebo-controlled intra-patient study evaluating the efficacy and safety of B VEC in 31 patients with DEB and following the initial report of the topline results, the latest data lend further support that B VEC can correct this disease at the molecular level. As previously announced, B VEC met the primary and secondary efficacy endpoints in complete wound healing relative to placebo. The proportion of primary wounds with complete wound healing was significantly greater with B VEC than placebo at both 3- and 6-month timepoints.
Results show that 49.7% of B VEC treated wounds compared to 7.1% of placebo treated wounds demonstrated durability of response, defined as complete wound healing at both 3 and 6 months. Nearly half of all B VEC treated wounds demonstrated complete wound healing for three consecutive visits. Of the total wounds closed at 3 months (n=21) 66.7% of B VEC treated wounds were also closed at 6 months, as compared to 33.3% for placebo treated (p=0.02). Treatment response was in favor of B VEC for all gender, age, and wound area/size subgroups, however the individual subgroups were not powered to demonstrate statistical significance. Finally a trend towards decreased pain in B VEC treated versus placebo treated wounds was observed across Weeks 22, 24, and 26; improvement in pain was consistent with wound healing. The majority of AEs were mild; there were no AEs leading to treatment discontinuation or death and one AE, mild erythema, was considered possibly related to study drug as assessed by the investigator. The long-term use of B VEC is being further investigated in an ongoing open-label extension study (NCT04917874), regardless of prior enrollment in GEM-3.
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