Cogent Biosciences filed a New Drug Application with the U.S. Food and Drug Administration on December 30, 2025 for its selective KIT inhibitor, bezuclastinib, targeting Non‑Advanced Systemic Mastocytosis (NonAdvSM). The filing follows the company’s October 2025 Breakthrough Therapy Designation for the same indication and is supported by the SUMMIT pivotal trial, which showed significant symptom improvement and biomarker reductions in patients with NonAdvSM.
The approval of bezuclastinib would address a critical unmet need in a rare disease where no targeted therapies exist for the NonAdvSM patient population. Cogent plans to submit two additional NDAs in 2026—one for gastrointestinal stromal tumor (GIST) and another for Advanced Systemic Mastocytosis (AdvSM)—based on data from the PEAK and APEX trials, positioning bezuclastinib as a multi‑indication platform drug.
Cogent’s financial position underpins its ability to pursue this aggressive pipeline. The company’s cash and marketable securities are sufficient to fund operations through late 2026, providing runway for continued research and development. The NDA filing is a key milestone that could unlock a new revenue stream and strengthen the company’s portfolio of precision therapies for genetically defined diseases.
Andrew Robbins, Cogent’s President and CEO, said the filing “moves us closer to delivering a disease‑modifying therapy to patients with NonAdvSM.” He added that this is the first of three planned submissions for bezuclastinib, underscoring the company’s confidence in the drug’s clinical and commercial potential.
As of December 30, 2025, Cogent’s stock traded around $37.92. Analysts have generally maintained a positive outlook, with consensus ratings of “Buy” or “Strong Buy” and average price targets suggesting upside. The market reaction has been driven by the positive SUMMIT data, the Breakthrough Therapy Designation, and the company’s strong cash position, all of which reinforce investor confidence in the drug’s regulatory trajectory.
Looking ahead, Cogent’s strategy hinges on the successful approval of bezuclastinib in NonAdvSM, followed by the planned GIST and AdvSM NDAs. The company’s focus on precision therapies for rare diseases, combined with its robust financial footing, positions it to capitalize on a growing market for targeted treatments in oncology and hematology.
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