Krystal Biotech has prioritized the inhaled formulation of KB707, its redosable gene therapy for solid tumors, based on early efficacy signals in non-small cell lung cancer (NSCLC). The company reported a 36% objective response rate in a heavily pre-treated NSCLC cohort, with no reported Grade 4 or 5 adverse events. Median duration of response and progression-free survival were not yet reached as of the April 15, 2025, data cutoff.
This decision reflects a strategic shift toward inhaled delivery, driven by the observed efficacy and KB707’s potential to address the significant unmet need in NSCLC. Krystal Biotech has secured an End of Phase 2 meeting with the FDA in October to discuss potential registration pathways. This suggests the company aims to expedite development and potentially pursue an accelerated approval strategy based on the early data.
The prioritization of inhaled KB707 impacts the development trajectory of the intratumoral formulation. While patient follow-up continues in the ongoing OPAL-1 trial for intratumoral KB707, enrollment has been paused. This resource allocation underscores Krystal Biotech’s confidence in the inhaled approach, possibly due to easier administration, improved patient tolerance, or superior distribution within the lung.
This move has several implications for the competitive landscape. If successful, inhaled KB707 could offer a new therapeutic option for NSCLC patients, particularly those who have progressed on existing therapies. The redosable nature of the therapy further differentiates it from other gene therapy approaches. For Krystal Biotech, the focus on inhaled KB707 streamlines its pipeline and concentrates resources on a single, promising indication.
Looking ahead, the FDA meeting will be critical. Regulators will likely scrutinize the durability of responses and seek further data on long-term survival. The ongoing KYANITE-1 trial will be crucial for gathering additional data and validating the early efficacy signals. Manufacturing scalability and commercialization strategy for inhaled delivery will also be key factors in KB707’s eventual success. The company’s ability to demonstrate a clear clinical benefit, coupled with a manageable safety profile, will be essential for securing regulatory approval and ultimately gaining market traction in the competitive NSCLC space.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
