PITTSBURGH, Pennsylvania, October 15, 2026
Krystal Biotech, Inc. (NASDAQ: KRYS) announced positive preliminary clinical data for KB113, an investigational intratumoral immunotherapy being developed for basal cell carcinomas (BCCs) in patients with Gorlin syndrome. In a three-patient pilot study, more than 80% of assessed landmark lesions regressed, including lesions that were not directly injected with the candidate. At the final assessment, conducted 30 days after the last dose, 69 of 83 assessed lesions (83.1%) showed some clinical regression. All three patients also had an injected lesion with histologically confirmed complete tumor clearance following biopsy. KB113 is designed to promote sustained, localized expression of interleukin-2 (IL-2) and interleukin-12 (IL-12) within the tumor microenvironment to stimulate immune-mediated tumor clearance. Krystal reported that all treatment-related adverse events observed in the pilot study were mild to moderate, transient and resolved during the study. The findings provide preliminary proof of concept for the program, while larger clinical studies will be needed to establish its safety and efficacy. The company is discussing advanced clinical development of KB113 with the U.S. Food and Drug Administration (FDA) and plans to provide an update following alignment with the agency.
Pilot Study Shows Regression in Injected and Non-Injected Lesions
The Gorlin Syndrome Pilot Study was a multicenter, single-arm, open-label study designed to assess the safety and preliminary efficacy of KB113 in adults with Gorlin syndrome. Three patients received six doses of up to 2 mL of KB113 at a concentration of 10⁸ plaque-forming units (PFU)/mL over 14 weeks. The first two doses were administered two weeks apart, followed by dosing every three weeks. Investigators selected landmark lesions for assessment, defined as facial BCCs measuring at least 5 mm or non-facial BCCs measuring at least 9 mm in diameter. Only landmark lesions were eligible for direct injection. Lesions were evaluated using a prespecified, investigator-assessed five-point clearance scale throughout treatment and again 30 days after the final dose. At the final assessment, 29 of 36 injected lesions (80.6%) and 40 of 47 non-injected lesions (85.1%) showed some clinical regression. Overall, 69 of 83 lesions (83.1%) regressed. More than 50% regression was observed in 42 lesions (50.6%), including 15 injected lesions (41.7%) and 27 non-injected lesions (57.4%). Eight lesions (9.6%) achieved at least 90% clinical regression. Responses were observed as early as two weeks after treatment initiation and reportedly increased in breadth and depth over time..
Histological Findings and Safety Results
In a recent publication in Science Advances, Immunocore and the University of Florida reported findings from experiments using live human pancreas tissue slices, including tissue from a donor with recent-onset type 1 diabetes. According to the company, IMC-S118AI selectively bound to HLA-A*02:01-positive beta cells and suppressed autoreactive T-cell activity around pancreatic islets. The experiments also indicated potential protection of beta cells and preservation of insulin secretion. These observations provide preclinical support for the candidate’s tissue-targeted mechanism, but they do not establish that the treatment will be safe or effective in patients. The Phase 1 trial will assess safety, pharmacokinetics and clinical activity as doses are escalated. Immunocore’s Executive Vice President of Clinical Development and Chief Medical Officer, Dr. Mohammed Dar, said the company intends to extend its modular bispecific platform into autoimmune diseases after development in oncology and initial proof of concept in infectious disease indications. The company’s objective is to evaluate whether tissue-targeted immune modulation can protect insulin-producing beta cells and help preserve insulin secretion in people with T1D. The results of the clinical study will be needed to determine whether the preclinical findings translate into a useful therapeutic approach.
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Immunocore Expands Its Autoimmune Development Pipeline
The initiation of the IMC-S118AI Phase 1 trial marks Immunocore’s expansion into tissue-specific autoimmune therapies. The company is also advancing a second ImmTAAI candidate for inflammatory dermatological diseases. Its broader ImmTAX platform, or Immune Mobilizing Monoclonal TCRs Against X Disease, supports the development of T-cell receptor bispecific immunotherapies across oncology, infectious diseases and autoimmune conditions. Immunocore’s most advanced oncology therapy, KIMMTRAK, is approved for eligible HLA-A*02:01-positive adults with unresectable or metastatic uveal melanoma in the United States, European Union, Canada, Australia and the United Kingdom. The company says its pipeline includes nine active clinical and preclinical programs across its therapeutic areas. With the first patient now dosed, IMC-S118AI enters clinical testing to establish an initial understanding of its safety, pharmacokinetic properties and activity in patients with T1D. Further development will depend on the evidence generated through clinical evaluation. The program’s central objective is to investigate whether selectively modulating immune responses at pancreatic beta cells can help preserve insulin production while limiting unwanted effects beyond the targeted tissue.
Source::: Krystal Biotech,press release
