Cogent Biosciences Announces FDA Breakthrough Therapy Designation for Bezuclastinib

Core Insights - Cogent Biosciences, Inc. announced that the FDA granted Breakthrough Therapy Designation for bezuclastinib in treating NonAdvanced Systemic Mastocytosis (NonAdvSM) and Smoldering Systemic Mastocytosis, highlighting the unmet medical need in these patient populations [1][2] - The company plans to submit a New Drug Application (NDA) for bezuclastinib by the end of 2025, following positive results from the SUMMIT trial [1][2] - The Breakthrough Therapy Designation allows for Priority Review and supports the company's planned commercial launch of bezuclastinib [2][3] Company Overview - Cogent Biosciences focuses on developing precision therapies for genetically defined diseases, with bezuclastinib being a selective tyrosine kinase inhibitor targeting the KIT D816V mutation [5] - The company is also developing therapies targeting mutations in FGFR2, ErbB2, PI3Ka, and KRAS, indicating a broad pipeline aimed at serious, genetically driven diseases [5] Upcoming Milestones - Cogent plans to report top-line results from the Phase 3 PEAK trial in Gastrointestinal Stromal Tumors (GIST) patients in November 2025 and from the APEX trial in Advanced Systemic Mastocytosis (AdvSM) patients in December 2025 [4]