Krystal Biotech, Inc. (NASDAQ: KRYS) announced clinical data from its Gorlin Syndrome Pilot Study on October 15, 2026, regarding the use of KB113, an intratumoral immunotherapy. The study evaluated KB113 for the treatment of basal cell carcinomas (BCCs) in patients with Gorlin syndrome. The data will be presented by study investigator Dr. Shireen V. Guide at the 14th Pediatric Dermatology Research Alliance (PeDRA) Annual Conference in Alexandria, Virginia, scheduled for October 15–17, 2026.

The Gorlin Syndrome Pilot Study was a multicenter, single-arm, open label study designed to assess safety and preliminary efficacy. Three adult patients with Gorlin syndrome were enrolled and received six doses of KB113 over a 14-week period. The dosing schedule began with two doses spaced two weeks apart, followed by one dose every three weeks. The treatment involved injecting landmark lesions, defined as facial BCCs at least 5 mm in diameter or non-facial BCCs at least 9 mm in diameter.

Results indicated broad antitumor responses across the three patients. Over 80% of all assessed landmark lesions regressed in size. Responses were observed in both injected and non-injected lesions, suggesting a systemic immune-mediated effect. Specifically, 69 out of 83 total lesions (83.1%) showed any clinical regression, with 42 lesions (50.6%) demonstrating greater than 50% regression. Biopsies of injected lesions that achieved at least 90% regression confirmed complete histological clearance in all three patients.

The press release notes that Gorlin syndrome is a genetic disease characterized by a greatly increased risk of developing BCCs, with potential prevalence in the United States exceeding 10,000 patients. The study found KB113 to be well tolerated; all reported adverse events were mild to moderate in severity and transient.

Krystal Biotech stated that it has initiated discussions with the United States Food and Drug Administration (FDA) regarding the advanced clinical development of KB113 for this indication. The company indicated it will provide an update on its clinical development plans following alignment with the FDA.