Krystal Biotech Prioritizes Inhaled KB707 for NSCLC Treatment Following Promising Clinical Results

Krystal Biotech has prioritized inhaled KB707 for non-small cell lung cancer treatment after promising clinical results showed a 36% objective response rate and a favorable safety profile with no Grade 4 or 5 adverse events. The FDA has granted an End of Phase 2 meeting to discuss registration pathways. Enrollment in the OPAL-1 study for intratumoral delivery has been paused to focus on the inhaled formulation. The company is continuing the KYANITE-1 study to evaluate inhaled KB707 in patients with locally advanced or metastatic lung tumors.

Krystal Biotech, Inc. (NASDAQ: KRYS) has announced a strategic update to its oncology program, KB707, focusing on the inhaled delivery method for the treatment of non-small cell lung cancer (NSCLC). The company has been granted an End of Phase 2 meeting with the United States Food and Drug Administration (FDA) in October to discuss potential registration pathways for inhaled KB707 [1].

The decision follows promising clinical results presented at the 2025 American Society of Clinical Oncology Annual Meeting, showing an objective response rate of 36% in heavily pre-treated NSCLC patients. The treatment demonstrated durability with median duration of response and progression-free survival not yet reached. Importantly, inhaled KB707 showed a favorable safety profile with no Grade 4 or 5 adverse events [1].

As part of this prioritization, Krystal Biotech has paused enrollment in the OPAL-1 study for intratumoral KB707 while continuing the KYANITE-1 study for inhaled KB707 in lung tumors. The company's decision to focus resources on the inhaled formulation indicates strong internal confidence in this approach and suggests a strategic prioritization of its pipeline based on clinical evidence [1].

The inhaled KB707 immunotherapy aims to deliver interleukin-2 (IL-2) and interleukin-12 (IL-12) directly to lung tumors via inhalation, potentially achieving higher local concentration of these powerful immune stimulators while minimizing systemic toxicity—a common limitation of systemic immunotherapies. This localized approach could represent a significant advancement in the treatment paradigm for NSCLC patients who have limited options after failing standard therapies [2].

Krystal Biotech's strategic update reflects both the clear and acute unmet need for new treatments of NSCLC and the promising efficacy profile observed with inhaled KB707. The company is continuing enrollment in the KYANITE-1 study, which is evaluating inhaled KB707 in patients with locally advanced or metastatic solid tumors of the lung [1].

The company's decision to pause enrollment in the OPAL-1 study and focus on inhaled KB707 demonstrates a commitment to advancing the most promising treatment options for NSCLC patients. The upcoming FDA meeting will be crucial in determining the potential registration pathways for inhaled KB707, which could bring a new therapeutic option to patients in need [1].

References:
[1] https://www.globenewswire.com/news-release/2025/08/21/3137361/0/en/Krystal-Biotech-Announces-Update-on-Development-Plans-for-Oncology-Program-KB707-and-Prioritization-of-Inhaled-KB707-for-the-Treatment-of-Non-Small-Cell-Lung-Cancer.html
[2] https://www.stocktitan.net/news/KRYS/krystal-biotech-announces-update-on-development-plans-for-oncology-0he2506ngi71.html