Krystal Biotech Announces First Patient Dosed in Phase 1/2 Trial of KB801 for the Treatment of Neurotrophic Keratitis

Core Viewpoint - Krystal Biotech, Inc. has initiated its Phase 1/2 clinical trial, EMERALD-1, for KB801, a redosable eye drop gene therapy aimed at treating neurotrophic keratitis (NK), a rare degenerative corneal disease [1][2][3] Company Overview - Krystal Biotech, Inc. is a global biotechnology company focused on developing genetic medicines for diseases with high unmet medical needs, with its first commercial product being VYJUVEK, the first redosable gene therapy approved by the FDA and EMA [8] Clinical Trial Details - The EMERALD-1 trial is a randomized, double-masked, multicenter, placebo-controlled study involving up to 27 adults with Stage 2 or Stage 3 NK, where participants will receive either KB801 or a placebo twice weekly for 8 weeks [3][4] - The primary objective of the trial is to evaluate the safety and tolerability of KB801, while the secondary objective focuses on the efficacy measured by the proportion of patients achieving complete durable healing of corneal epithelium at 8 weeks [4] Product Information - KB801 is designed to enable sustained expression of nerve growth factor (NGF) in the front of the eye, potentially reducing the treatment burden for NK patients compared to current therapies that require frequent administration [6] - The estimated prevalence of NK is between 10 to 50 cases per 100,000, with claims data indicating a significant increase in awareness and diagnosis rates in the U.S., from 31,000 patients in 2020 to approximately 68,000 in 2024, representing an increase of over 115% [2][6]