Krystal Biotech has announced a strategic shift in its oncology program, prioritizing the development of inhaled KB707 for non-small cell lung cancer (NSCLC) treatment following promising early clinical results. The commercial-stage biotechnology company secured an End of Phase 2 meeting with the FDA scheduled for October to discuss potential registration pathways for the novel immunotherapy.
Promising Efficacy Data Drives Strategic Focus
KB707 represents a redosable immunotherapy designed to drive sustained, localized expression of interleukin-2 (IL-2) and interleukin-12 (IL-12) in the tumor microenvironment, promoting systemic immune-mediated tumor clearance. The company's decision to prioritize the inhaled formulation stems from compelling clinical evidence presented at the 2025 American Society of Clinical Oncology Annual Meeting.
As of the April 15, 2025 data cut-off, the objective response rate in heavily pre-treated NSCLC patients reached 36%. Notably, median duration of response and progression-free survival had not been reached at the time of analysis, suggesting potentially durable therapeutic benefits. The treatment demonstrated monotherapy activity and produced durable responses in this challenging patient population.
"The acceleration of our work on inhaled KB707 is a reflection of both the clear and acute unmet need that exists for new treatments of NSCLC and the promising efficacy profile we have observed to date with inhaled KB707," said Suma Krishnan, President of Research and Development of Krystal Biotech. "We look forward to meeting with the FDA and bringing another urgently needed therapeutic option to patients."
Safety Profile Supports Outpatient Administration
The inhaled formulation of KB707 continued to demonstrate a favorable safety profile throughout clinical testing. No Grade 4 or 5 adverse events were observed, and the treatment remained generally well tolerated. The therapy's safety characteristics make it amenable to administration in an outpatient setting, potentially improving patient convenience and healthcare system efficiency.
Clinical Development Strategy Adjustment
With the prioritization of inhaled KB707, Krystal Biotech has made strategic adjustments to its clinical development portfolio. Enrollment continues in the KYANITE-1 study, a Phase 1/2 open-label, multi-center, dose escalation and expansion study evaluating inhaled KB707 in patients with locally advanced or metastatic solid tumors of the lung. The study can be found at clinicaltrials.gov under NCT identifier NCT06228326.
Conversely, the company has paused enrollment in OPAL-1, a Phase 1/2 open-label, multi-center, dose escalation and expansion study evaluating intratumoral KB707 in patients with locally advanced or metastatic solid tumor malignancies. Existing patients on OPAL-1 continue to be followed, and the company may adjust development plans for intratumoral KB707 based on ongoing safety and efficacy results. The OPAL-1 study details are available under NCT identifier NCT05970497.
Company Portfolio Context
Krystal Biotech operates as a fully integrated, commercial-stage biotechnology company focused on genetic medicines for diseases with high unmet medical needs. The company's first commercial product, VYJUVEK, represents the first-ever redosable gene therapy and the first genetic medicine approved in the United States, Europe, and Japan for treating dystrophic epidermolysis bullosa. The company maintains a robust pipeline of investigational genetic medicines across respiratory, oncology, dermatology, ophthalmology, and aesthetics applications.
