The pharmaceutical company received Regenerative Medicine Advanced Therapy designation from FDA for KB103

Krystal Biotech, Inc., a gene therapy company recently announced positive results from a Phase 2 clinical trial of KB103 (the GEM 2 study) and an update on results from Phase 1 trial – the GEM 1 study.

Reportedly, in the Phase 2 clinical trial, two patients of ages 22 & 19 and two pediatric patients of ages 14 & 15 with severe generalized RDEB (recessive dystrophic epidermolysis bullosa) were enrolled in December, last year. Prior to dosing, 3 wounds of size up to 20 cm² were selected on each patient for the trial & then randomized to receive placebo or KB103 in a ratio of 2:1. In the trial results, a total of two KB103 treated chronic wounds and four KB103 treated recurring wounds were included.

Having confirmed the presence of COL7 (type VII collagen) & anchoring fibrils in the Phase 1 part of the trial and acknowledged by Food & Drug Administration, the endpoints of the Phase 2 part were revised towards a focus on clinical improvement.

As per sources close to the matter, 1 of the 4 patients dropped out of the trial voluntarily. The dropout was unrelated to any safety issues with KB103. The Phase 2 analysis was conducted on remaining 3 patients (three placebo treated wounds and six KB103 treated wounds). Out of the 6 wounds that were KB103-treated in patients enrolled for the duration of the Phase 2 trial, 4 were categorized as recurring and 2 were categorized as chronic, based on patient reporting. Recurring wounds open and close spontaneously while chronic wounds remain open for greater than 12 weeks.

Earlier, in the Phase 1 trial, 2 adult patients with severe generalized recessive dystrophic epidermolysis bullosa were evaluated. In each patient, 2 wounds with a surface area of 10 cm² were randomized to receive placebo or topical KB103.

Source credits: http://ir.krystalbio.com/news-releases/news-release-details/krystal-biotech-announces-positive-results-phase-2-clinical