Krystal Biotech announced positive clinical data from its pilot study of KB113, an immunotherapy for treating basal cell carcinomas (BCCs) in patients with Gorlin syndrome. The study demonstrated broad antitumor responses, with significant regression in both injected and non-injected lesions across all three patients treated.

Key Details

  • Overall Response: 83.1% (69 of 83) of all assessed BCC lesions showed clinical regression. Over half of the lesions (50.6%) demonstrated greater than 50% regression.
  • Systemic Effect: Significant regression was observed in 85.1% of lesions that were not directly injected, providing preliminary evidence of a systemic immune response (abscopal effect).
  • Safety & Next Steps: KB113 was well tolerated, with all reported adverse events being mild to moderate and transient. The company has initiated discussions with the FDA regarding advanced clinical development.