Krystal Biotech Reports Shrinkage in 83.1% of Lesions in Small Gorlin Syndrome Study
Key Facts
Krystal Biotech announced on 15 October 2026 that 69 of 83 basal cell carcinoma lesions shrank in patients with Gorlin syndrome who received its experimental treatment KB113, representing 83.1% of evaluable lesions. The study enrolled three adult patients, according to a company statement, which described the results as preliminary proof of concept. The trial was conducted at multiple centers to assess the treatment's safety and preliminary efficacy in patients with the rare genetic disorder. The study was open-label and single-arm, meaning participants received the experimental treatment in a single treatment group. The announcement follows the company's expansion of the program to include Gorlin patients and sets the stage for discussions with regulators on the clinical development pathway.
According to the results released by the company, 29 of 36 lesions injected directly with the treatment shrank, representing 80.6%. Meanwhile, 40 of 47 uninjected lesions decreased in size, equivalent to 85.1% of that group. Shrinkage began to emerge two weeks after treatment started, with responses broadening and deepening as follow-up continued. The final assessment was conducted 30 days after participants received their last dose. Histological examination confirmed complete tumor clearance in three injected lesions selected for biopsy at the end of the study, one from each participant. The company described the response in uninjected lesions as preliminary evidence consistent with a therapeutic effect beyond the injection site.
Participants received six intralesional doses over three months, according to the company's scientific poster. The treatment is designed to deliver the genes for interleukin 12 and interleukin 2 to the tumor microenvironment to stimulate antitumor immunity. This design aims to activate the immune response around the tumor through direct injection into lesions. On safety, the study recorded 22 treatment-related adverse events, all classified as mild or moderate. These events resolved during the study, according to the scientific poster. The company said in the poster: "No serious adverse events were reported."
Gorlin syndrome is a rare genetic disorder that affects multiple organs and tissues in the body, according to the U.S. National Cancer Institute. The disorder increases the risk of basal cell carcinoma, the disease targeted by Krystal Biotech's trial. The U.S. National Library of Medicine estimates the syndrome's prevalence at one in 31,000 people. Those affected typically begin developing basal cell carcinomas during adolescence or early adulthood, according to the library. In its second-quarter results disclosure, published in August, the company said it had expanded the intratumoral treatment study to assess dose safety and efficacy in Gorlin patients. The expansion followed early efficacy signals in patients with basal cell carcinoma.
The program's development comes as the company recorded revenue growth for its product VYJUVEK in the second quarter of 2026. Global net revenue for the product reached $119.2 million, according to Krystal Biotech's financial results disclosure. That represents a 24% increase from the same quarter a year earlier. The company's total cash, cash equivalents and investments stood at $1.1 billion at the end of the second quarter. The financial disclosure of these revenues and balances coincided with the announcement that the intratumoral treatment study had been expanded to include patients with Gorlin syndrome.
The company's statement identified the Pediatric Dermatology Research Conference, held in Alexandria, Virginia, between 15 and 17 October 2026, as the venue for presenting the study results. On the regulatory front, Krystal Biotech confirmed that its experimental treatment had not received approval from the U.S. Food and Drug Administration. It had also not received approval from any other regulatory authority, according to the statement. The company said it had begun discussions with the U.S. agency on late-stage clinical development of the program. In its announcement on 15 October 2026, it added that it would disclose the development plan after reaching alignment with the agency.