Krystal Biotech Reports Over 80% Lesion Reduction in Rare Skin Cancer Trial

Krystal Biotech Inc. (NASDAQ:KRYS) on Thursday released a clinical update for its intratumoral immunotherapy, KB113.

The treatment demonstrated initial proof of concept by reducing basal cell carcinomas (BCCs) in patients suffering from Gorlin syndrome.

This targeted therapy functions by driving sustained, localized expression of interleukin-2 and interleukin-12 within the tumor microenvironment. By doing so, it promotes systemic, immune-mediated tumor clearance.

Gorlin syndrome (nevoid basal cell carcinoma syndrome) is a rare genetic disorder that increases the risk of basal cell carcinoma (a type of skin cancer). It can also cause benign growths.

William Blair wrote, “While only a small dataset, we believe these results are encouraging, demonstrate initial proof-of-concept, and support further development in the indication.”

Promising Tumor Reduction And Trial Results

The multicenter, single-arm pilot study evaluated three adult patients with Gorlin syndrome.

Each participant received six doses of the immunotherapy over a 14-week period. The initial two doses occurred two weeks apart, followed by doses every three weeks. Investigators assessed landmark lesions throughout the treatment and 30 days after the final dose.

The data highlighted broad antitumor responses across all participants. Over 80% of assessed BCC landmark lesions regressed in size.

More than half of all evaluated lesions showed greater than 50% clinical regression, and a subset achieved at least 90% regression.

Evidence Of Abscopal Effect And Complete Clearance

Notably, the KB113 therapy triggered a distinct abscopal effect. Researchers observed substantial regression in non-injected lesions. Out of 47 non-injected tumors, 40 demonstrated measurable clinical regression.

Furthermore, investigators biopsied one injected lesion from each patient exhibiting at least 90% regression. Each biopsy confirmed a complete histological response, showing no evidence of residual tumor tissue.

Easing Gorlin Syndrome Burden And Next Steps

Gorlin syndrome is a genetic disease that increases a patient’s risk of developing BCCs, sometimes beginning in infancy. Patients endure frequent and disfiguring surgeries, causing permanent tissue loss. With an estimated prevalence exceeding 10,000 individuals in the U.S., non-surgical treatments remain a critical need.

All three patients tolerated the KB113 regimen well. Reported adverse events proved mild or moderate and resolved during the study.

Krystal Biotech recently initiated discussions with the U.S. Food and Drug Administration (FDA) regarding advanced clinical development for this treatment. The company will announce further plans following FDA alignment.

Analyst Lachlan Hanbury-Brown said Krystal has begun discussions with the FDA to advance KB113 for Gorlin syndrome, with a clinical development update expected once the company aligns with the agency.

He sees the program as a meaningful market opportunity that could leverage Krystal’s existing expertise and infrastructure in rare genetic skin disorders.

KRYS Stock Price Activity: Krystal Biotech shares are trading higher by 0.72% at $333.06 at the time of publication on Thursday, according to Benzinga Pro data.

Image via Shutterstock/ Fuss Sergey