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Krystal Biotech Highlights Broad Antitumor Responses With KB113 In Gorlin Syndrome

Benzinga·10/15/2026 11:05:34
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Over 80% of assessed basal cell carcinoma lesions regressed across three Gorlin syndrome patients treated with KB113

Substantial regression was observed in lesions not directly injected with KB113, consistent with abscopal effect

KB113 injections were well tolerated by Gorlin syndrome patients; all reported adverse events were mild to moderate in severity and transient in nature

Data will be presented by study investigator Dr. Shireen Guide at the 14th PeDRA Annual Conference, October 15-17, 2026

PITTSBURGH, Oct. 15, 2026 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (the "Company") (NASDAQ:KRYS) announced today a positive clinical update supporting initial proof of concept for KB113, an intratumoral immunotherapy designed to drive sustained, localized expression of interleukin-2 (IL-2) and interleukin-12 (IL-12) in the tumor microenvironment and promote systemic immune-mediated tumor clearance, in the treatment of basal cell carcinomas (BCCs) in patients with Gorlin syndrome:

  • Broad antitumor responses were detected in all three Gorlin syndrome patients treated with KB113, with over 80% of assessed BCC lesions reducing in size;
  • Responses were observed in both injected and non-injected lesions, including substantial regression in lesions that were not directly injected with KB113, consistent with an abscopal effect; and
  • Complete clearance of an injected lesion was also confirmed histologically for each of the three patients treated with KB113.
     

Gorlin syndrome is a genetic disease characterized by a greatly increased risk of developing BCCs. Patients with Gorlin syndrome can develop BCCs as early as infancy and may have hundreds of BCCs over their lifetimes requiring frequent and potentially disfiguring surgical procedures. The psychological and physical toll of Gorlin syndrome builds over time, as patients are subjected to repeated cycles of surveillance, surgery, and recovery to manage emergent BCCs, with each surgery adding to the cumulative risk of scarring and permanent tissue loss. Prevalence data for Gorlin syndrome is limited but available data suggest the number of patients with Gorlin syndrome in the United States could exceed 10,000.