Krystal Biotech, Inc. (NASDAQ: KRYS) has released early clinical data from its ongoing Kyanite-1 study evaluating inhaled KB707 in patients with solid tumors of the lung. The study has shown promising results in advanced non-small cell lung cancer (NSCLC) patients, with an objective response rate (ORR) of 27% and a disease control rate (DCR) of 73% as of the data cut-off on December 6, 2024.
The study, which began with the first patient dosed on April 17, 2024, has enrolled a total of 37 patients, including 17 with a diagnosis of advanced NSCLC. Patients received inhaled KB707 once weekly for the first three weeks, then once every three weeks. The treatment has been generally well tolerated in this heavily pre-treated patient population with advanced disease, with no grade 4 or 5 adverse events observed.
In the NSCLC patient analysis cohort, the ORR was 27% with three partial responses, and the DCR was 73% with 7 out of 11 patients still on treatment. Additionally, in the same 11 evaluable NSCLC patients, the ORR in target lung lesions specifically was 36%, with three partial responses and one complete response, and the DCR was 82%.
Based on these positive initial results, the company has amended the Kyanite-1 protocol to add two cohorts evaluating inhaled KB707 for the treatment of advanced NSCLC in combination with either anti-programmed cell death protein 1 (PD-1) therapy or anti-PD-1 therapy and chemotherapy. No patients have been enrolled in the combination expansion cohorts to date.
Krystal Biotech, Inc. is a commercial-stage biotechnology company focused on the discovery, development, and commercialization of genetic medicines to treat diseases with high unmet medical needs. Their first commercial product, Vyjuvek®, is the first-ever redosable gene therapy and the first medicine approved by the FDA for the treatment of dystrophic epidermolysis bullosa. The company is also rapidly advancing a robust preclinical and clinical pipeline of investigational genetic medicines in respiratory, oncology, dermatology, ophthalmology, and aesthetics. The market has reacted to these announcements by moving the company's shares -5.5% to a price of $159.8. For more information, read the company's full 8-K submission here.
