Krystal Biotech, Inc. has announced positive interim results from its ongoing placebo-controlled Phase 1/2 clinical trial of KB103.

Two adult Recessive Dystrophic Epidermolysis Bullosa (“RDEB”), NC1[+] patients aged 35 and 28 years old have completed treatment with topical KB103 to date. The patients were re-dosed during the study. In each patient, two wounds with an approximate surface area of 10 cm²were randomized to receive either topical KB103 or placebo (for a total of 4 wounds evaluated). KB103 was also injected intradermally in both patients to intact skin to evaluate the mechanism of action of KB103 and validate molecular correction. Both KB103-treated wounds and intradermally injected intact skin were biopsied to assess functional COL7 expression and anchoring fibril formation.  KB103-treated wounds were also evaluated for clinically relevant healing compared to placebo-treated wounds and to baseline. They were also monitored for general and recombinant viral infection and autoimmune response at each evaluation.

“Results on 2 patients demonstrate a meaningful clinical benefit and suggest that KB103 can afford a simple, convenient, painless way to administer treatment for patients suffering with this debilitating disease,” says Dr. Peter Marinkovich, MD, Associate Professor of Dermatology, Stanford University and Principal Investigator in the GEM study. “These early data are encouraging and we look forward to continuing the study in pediatric populations.”