Krystal Biotech, Inc., presented more detailed results from the GEM-3 Phase 3 study of beremagene geperpavec (B-VEC), an investigational, topical gene therapy for the treatment of dystrophic epidermolysis bullosa (DEB), at the 2022 American Academy of Dermatology Annual Meeting in Boston.

The data was presented by Peter Marinkovich, MD, 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. 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 three- and six-month timepoints (p <0.005). 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.

“We are very pleased to share this data at such a highly regarded dermatology conference,” says Suma Krishnan, President, Research & Development, Krystal Biotech. “Following the initial report of the topline results, the latest data lend further strong support for our belief that B-VEC could correct this devastating disease at the molecular level and fulfill our founding mission to bring a convenient, non-invasive treatment to EB patients and their families.”