Krystal Biotech Initiates Phase 3 Trial for Rare Eye Disease

Krystal Biotech has initiated dosing in its Phase 3 IOLITE clinical trial to evaluate KB803, an eye drop treatment aimed at preventing and treating corneal abrasions in patients with dystrophic epidermolysis bullosa (DEB). The double-blind, placebo-controlled, multicenter trial with a crossover design will enroll approximately 16 patients aged six months and older, who must first complete a 12-week natural history run-in phase. KB803 delivers the COL7A1 transgene to the corneal epithelium to promote local type VII collagen production, with the primary endpoint measuring changes in monthly corneal abrasion days. Prior compassionate use cases showed promising results, including full corneal healing within three months and significant visual acuity improvement, indicating KB803's potential efficacy. The decentralized trial design allows home administration by healthcare providers, facilitating patient participation in this rare disease population. This trial represents a significant milestone for Krystal Biotech, addressing an unmet medical need with no current corrective therapies for DEB-related ocular complications, while also presenting financial opportunities and inherent clinical risks.