Krystal Biotech Reports Positive Clinical Data for KB113 in Basal Cell Carcinomas in Gorlin Syndrome Patients

Krystal Biotech, Inc. has announced positive clinical data from its Gorlin Syndrome Pilot Study, demonstrating initial proof of concept for KB113, an intratumoral immunotherapy. The study showed broad antitumor responses in all three Gorlin syndrome patients treated, with over 80% of assessed basal cell carcinoma (BCC) lesions regressing. Notably, responses were observed in both injected and non-injected lesions, indicating a systemic immune-mediated effect. Complete histological clearance was confirmed in biopsied lesions. KB113 was well-tolerated, with only mild to moderate and transient adverse events. The company is in discussions with the FDA regarding advanced clinical development for this therapy, which could offer a new treatment option for patients with Gorlin syndrome who frequently develop BCCs.