Krystal Biotech Announces Positive Results from Phase 2 Clinical Trial

Krystal Biotech (:NASDAQ:KRYS) has announced positive results from its Phase 2 clinical trial of KB103 (GEM-2).

As quoted in the press release:

In the Phase 2 trial, four additional patients (two adults (ages 22 and 19) and two pediatric (ages 14 and 15)) with severe generalized recessive dystrophic epidermolysis bullosa (“RDEB”) were enrolled in December 2018. Prior to dosing, three wounds of size up to 20cm² were selected on each patient for the trial and subsequently randomized to receive either KB103 or placebo in a 2:1 (KB103: placebo) ratio. A total of four KB103-treated recurring wounds and two KB103-treated chronic wounds were included in the trial results. Having established the presence of type VII collagen (COL7) and anchoring fibrils in the Phase 1 portion of the trial and acknowledged by the FDA, the endpoints of the Phase 2 portion were revised toward a focus on clinical improvement.

One of the four patients (aged 19) voluntarily dropped out of the trial after 30 days due to an inability to travel to the clinical trial site. The dropout was unrelated to any safety or efficacy issues with KB103. Data from this patient was excluded from the overall analysis. The Phase 2 analysis was conducted on remaining three patients (six KB103 treated wounds and three placebo treated wounds) in the Phase 2 trial. With respect to the six KB103 treated wounds on patients enrolled for the duration of the Phase 2 trial, two were categorized as chronic and four as recurring based on patient reporting. Chronic wounds remain open for greater than 12 weeks while recurring wounds open and close spontaneously.

Earlier in the Phase 1 trial, two adult patients with severe generalized RDEB were evaluated. In each patient, two wounds with an approximate surface area of 10 cm² were randomized to receive either topical KB103 or placebo.

Click here to read the full press release.