Cogent Biosciences has submitted a New Drug Application to the U.S. Food and Drug Administration (FDA) for bezuclastinib in patients with nonadvanced systemic mastocytosis (SM), the company shared in a recent press release.
The submission comes following positive results from the SUMMIT clinical trial, which met all of its primary and secondary endpoints. The study found that bezuclastinib significantly reduced serum tryptase levels and 11 different patient-reported symptoms compared with a placebo. These benefits extended through 48 weeks, with favorable safety and tolerability profiles.
What is the difference between advanced and nonadvanced SM?
Nonadvanced SM comprises the indolent SM and smoldering SM subtypes. Advanced SM includes aggressive SM, SM with an associated hematologic neoplasm and mast cell leukemia.
“Building on the exceptional results from the SUMMIT trial, this filing moves us closer to delivering an important disease-modifying therapy to patients with [non-advanced SM],” said Andrew Robbins, president and chief executive officer at Cogent Biosciences. If approved, Cogent will be permitted to sell and market the drug in the U.S.
Read more about SM therapies
In October 2025, bezuclastinib received Breakthrough Therapy Designation from the FDA for patients with nonadvanced SM who previously received avapritinib and patients with smoldering SM. The designation helps accelerate the development and review processes for therapies aimed to treat serious or life-threatening diseases.
Cogent Biosciences also plans to submit New Drug Applications for bezuclastinib in gastrointestinal stromal tumor and advanced SM during the first half of 2026. The company reported promising clinical trial results for bezuclastinib in both of these diseases in late 2025.
A targeted tyrosine kinase inhibitor, bezuclastinib is an oral pill that works by blocking mutations in the KIT gene, including the KIT D816V mutation. By doing so, the drug aims to prevent the accumulation of mast cells throughout the body and associated symptoms.
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