Krystal Biotech doses first patient in phase 3 trial IOLITE

Krystal Biotech has dosed the first patient in IOLITE, its phase 3 clinical trial evaluating KB803 for the treatment and prevention of corneal abrasions in dystrophic epidermolysis bullosa (DEB) patients.

KB803 is a redosable eye drop gene therapy designed to deliver 2 copies of the COL7A1 transgene to the epithelial cells in a patient’s eye for the treatment and prevention of corneal abrasions in DEB patients. The company states the aim of the treatment is to address “the fundamental disease-causing mechanism at the molecular level.”

IOLITE (NCT07016750) is an intra-patient, double-blind, placebo-controlled, multicenter Phase 3 study with a crossover design to evaluate KB803, administered as an eye drop in DEB patients 6 months of age or older.

The company states that approximately 16 patients will be enrolled in the trial. Enrolled patients will initially receive a single eye drop of either placebo or KB803, at a concentration of 10⁹ PFU/mL, to each eye once weekly for 12 weeks. At the conclusion of the first 12 weeks, patients will be switched from placebo to KB803, or vice versa, and continue with once weekly administration for a second 12 week period. Drug administration for the trial will occur at the patient’s home by a healthcare provider.

The primary endpoint of IOLITE is the change in the average number of days per month with corneal abrasion symptoms while receiving KB803 versus placebo. Statistical significance will be analyzed via intra-patient paired measurements to account for potentially expected high inter-patient variability. Patients will complete weekly symptom diaries and monthly questionnaires to document corneal abrasion symptoms and frequency and gauge overall subject (or parent/legal guardian) impressions of disease severity and disease/symptom changes during the study.

References:

1. Krystal Biotech announces first patient dosed in phase 3 clinical trial of KB803 for the treatment and prevention of corneal abrasions in patients with dystrophic epidermolysis bullosa. Published June 24, 2025. Accessed June 25, 2025. https://ir.krystalbio.com/news-releases/news-release-details/krystal-biotech-announces-first-patient-dosed-phase-3-clinical

2. A Study Assessing B-VEC Compared to Matching Placebo for the Treatment and Prevention of Corneal Abrasions in Dystrophic Epidermolysis Bullosa (IOLITE). Accessed June 25, 2025. https://clinicaltrials.gov/study/NCT07016750?tab=table