Krystal Biotech (KRYS -3.2%) initiates a pivotal Phase 3 (GEM-3) trial evaluating topical gene therapy bercolagene telserpavec (B-VEC) (previously KB103) for the treatment of dystrophic epidermolysis bullosa (DEB), a rare inherited disorder characterized by fragile skin that blisters easily.
The trial will evaluate the efficacy and safety of either B-VEC or placebo in ~30 in patients suffering from both recessive and dominant forms of DEB.
The primary endpoint is complete wound healing at weeks 20, 22 and 24 versus placebo.
The company expects to report topline data and file a U.S. marketing application in 2021.
The company also expects that its second GMP facility, ASTRA, will be operational in 1H of 2022.
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