Krystal Biotech has initiated its Phase 3 IOLITE clinical trial, dosing the first patient in a pivotal study evaluating KB803 for treating and preventing corneal abrasions in patients with Dystrophic Epidermolysis Bullosa (DEB). This investigational gene therapy, administered as an eye drop, is engineered to deliver two copies of the COL7A1 transgene to the corneal epithelium, aiming to restore local production of type VII collagen—a protein deficient in DEB that leads to recurrent, painful corneal abrasions and potential vision loss.

Ocular complications affect over 25% of DEB patients, with over half of those with the recessive form (RDEB) experiencing them. Currently, there are no corrective therapies, only supportive care. The IOLITE study is a randomized, double-blind, placebo-controlled, multicenter trial with a crossover design, enrolling approximately 16 DEB patients aged 6 months or older. Participants, who must complete a 12-week run-in period in an ongoing natural history study to establish baseline corneal abrasion symptoms, will receive either KB803 or placebo once weekly for 12 weeks, then switch treatments for another 12 weeks. The study's primary endpoint is the change in the average number of days per month with corneal abrasion symptoms while on KB803 versus placebo, with safety and secondary efficacy endpoints also being assessed.

This Phase 3 trial builds on compelling prior data, including a compassionate use case where a DEB patient experienced full corneal healing within three months and significant visual acuity improvement within eight months after receiving KB803 eye drops. Krystal Biotech's commitment to comprehensively treating DEB, as highlighted by President of Research & Development Suma Krishnan, underscores the potential of KB803 to address this significant unmet medical need and prevent vision loss in affected individuals. Krystal Biotech is a fully integrated, commercial-stage biotechnology company that recently gained FDA and EMA approval for VYJUVEK®, its first commercial product, also for DEB, and continues to advance a robust pipeline of genetic medicines.