- 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.