WALTHAM, Mass. and BOULDER, Colo. — September 15, 2026
Cogent Biosciences, Inc. announced that the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for bezuclastinib in patients with Advanced Systemic Mastocytosis (AdvSM). The FDA assigned a PDUFA target action date of June 29, 2027, marking a major regulatory milestone for Cogent’s precision therapy program. The agency also communicated that it currently does not plan to convene an advisory committee and has not identified potential review issues. The NDA is supported by results from the APEX pivotal trial, which demonstrated clinically meaningful responses and durable clinical activity in patients with AdvSM. Cogent said the regulatory milestone brings bezuclastinib closer to potential availability for patients with a serious KIT-driven disease while advancing the company’s broader strategy of developing precision therapies for genetically defined conditions.
APEX Trial Demonstrates Responses and Durable Disease Control
The APEX pivotal trial provided the clinical evidence supporting the AdvSM NDA, with results most recently presented at the 2026 European Hematology Association Congress. As of the March 31, 2026 data cutoff, bezuclastinib achieved a 65% overall response rate according to mIWG-MRT-ECNM criteria, including 57% of patients achieving complete response (CR), CR with partial hematologic recovery (CRh) or partial response (PR) as their best response. Using pure pathological response criteria, the response rate was 81%. The treatment also demonstrated durable activity, with a 79% 12-month progression-free survival rate and 87% 12-month overall survival rate. Median progression-free survival and overall survival had not yet matured at the data cutoff. Bezuclastinib is designed to selectively inhibit KIT D816V and other KIT exon 17 mutations, which are key drivers of systemic mastocytosis and are also relevant to other KIT-dependent diseases.
Cogent Builds Multi-Indication Bezuclastinib Strategy
The AdvSM filing is one of three regulatory programs Cogent is advancing for bezuclastinib, supporting a broader strategy across KIT-driven diseases. The company’s NDA for NonAdvanced Systemic Mastocytosis (NonAdvSM) remains under FDA review with a PDUFA target action date of December 30, 2026, while its NDA for gastrointestinal stromal tumor (GIST) has a November 30, 2026 PDUFA target action date. The three programs could expand the potential application of bezuclastinib across different patient populations affected by KIT signaling abnormalities. Cogent has also established Expanded Access Programs in the United States for eligible patients with systemic mastocytosis or GIST who may benefit from bezuclastinib or bezuclastinib combined with sunitinib. With regulatory reviews progressing in parallel, the company is preparing its clinical, medical and commercial infrastructure for potential launches across multiple indications.
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Bezuclastinib Strengthens Cogent’s Precision Oncology Pipeline
Bezuclastinib is the lead clinical program within Cogent Biosciences’ precision-therapy pipeline, which is focused on genetically defined diseases. The selective tyrosine kinase inhibitor is being developed to target the molecular drivers of systemic mastocytosis and KIT-dependent GIST, while Cogent is also researching targeted therapies directed against mutations involving ErbB2, PI3Kα, KRAS and JAK2. The company said its medical and commercial teams are already in place as it prepares for potential launches associated with the ongoing regulatory reviews. The FDA acceptance of the AdvSM NDA therefore adds an important regulatory step to a multi-indication development program. With three bezuclastinib NDAs progressing through FDA review and the AdvSM application now carrying a June 2027 PDUFA date, Cogent is moving toward a potential commercial-stage expansion of its precision medicine strategy across KIT-driven diseases.
Source:Cogent Biosciences press release
