KRYS Up 4% on Initiating Dosing in Rare Eye Disease Phase III Study

Company Overview - Krystal Biotech's shares increased by 4.2% following the announcement of dosing the first patient in a late-stage study for KB803, aimed at treating and preventing corneal abrasions in dystrophic epidermolysis bullosa (DEB) [1][5] - The company's only marketed product, Vyjuvek, is FDA-approved for DEB patients aged six months or older, known as B-VEC outside the U.S. [2] Disease Context - DEB is a rare, severe monogenic disease affecting skin and mucosal tissues, with over 25% of patients experiencing eye complications. There are no corrective treatments available, leading to reliance on wound management and occasional surgery. Approximately 750 individuals in the U.S. and 2,000 globally are affected [3][6]. Study Details - The phase III IOLITE study will enroll 16 DEB patients, administering either placebo or KB803 eye drops for 12 weeks, followed by a switch for another 12 weeks. The study is decentralized, allowing home dosing by healthcare professionals [7][8]. - The primary endpoint is to measure the change in average monthly days with corneal abrasion symptoms in patients receiving KB803 compared to placebo, with safety and secondary efficacy data collected throughout the 24-week study [8]. Previous Efficacy Evidence - KB803 (B-VEC) previously demonstrated efficacy in a compassionate use case, leading to full corneal healing and significant visual acuity improvement in a DEB patient after eye surgery [9].