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Krystal Biotech Announces Initiation of Pivotal Phase 3 Study of Beremagene Geperpavec in Patients with Dystrophic Epidermolysis Bullosa
The randomized, double-blind GEM-3 trial will compare repeat dosing of either B-VEC or placebo in approximately 30 dystrophic epidermolysis bullosa (DEB)
About this update from Krystal Biotech, Inc.
[{"type":"text","content":"The randomized, double-blind GEM-3 trial will compare repeat dosing of either B-VEC or placebo in approximately 30 dystrophic epidermolysis bullosa (DEB) patients Top line data and BLA filing are anticipated in 2021; EMA aligned on pivotal study design and an MAA is anticipated shortly after BLA PITTSBURGH, July 28, 2020 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (Nasdaq:KRYS), a fully integrated gene therapy company driven by its proprietary, engineered herpes simplex virus type 1 vector (HSV-1) platform, today announced the initiation of GEM-3 study, a multi-center, placebo-controlled, double-blinded, Phase 3 clinical study of beremagene geperpavec (“B-VEC”, previously “KB103”) for the treatment of dystrophic epidermolysis bullosa (DEB) patients.\n “Despite the logistical challenges associated with COVID-19, Stanford and I are excited to begin this pivotal study,” said Dr. Peter Marinkovich, M.D., associate professor of dermatology and director of the Blistering Disease Clinic at Stanford University, and primary investigator for the trial. “We and the other study sites have worked closely with the Krystal team to ensure safe trial practices in the context of the ongoing pandemic, enabled by the out-patient nature of B-VEC dosing.” Suma Krishnan, founder and chief operating officer of Krystal Biotech added, “The initiation of the pivotal study marks an important milestone toward our goal of a potential painless, convenient, and corrective approach to treat patients suffering from this debilitating disease. We look forward to having a home dosing protocol in place in the upcoming months that, we believe, would further improve the quality of life of our patients and their caregivers.” This clinical progress follows announcement of positive results from the GEM-1 and GEM-2 studies. B-VEC is a topical, re-dosable gene therapy in development for the treatment of both the dominant and recessive forms of DEB, a rare and severe monogenic skin disease for which there is currently no approved treatment. The U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have each granted B-VEC orphan drug designation for the treatment of DEB, and the FDA has granted B-VEC fast track designation and rare pediatric designation for the treatment of DEB. In addition, in 2019, the FDA granted Regenerative Medicine Advanced Ther...