Cogent Biosciences Inc

Cogent Bioscience has filed a New Drug Application with the U.S. FDA for bezuclastinib as a treatment for Advanced Systemic Mastocytosis. The submission is supported by clinical results from the APEX pivotal trial. President and CEO Andrew Robbins said the company is engaging closely with the FDA on three submissions and expects initial approvals by the end of 2026. Bezuclastinib is a selective tyrosine kinase inhibitor targeting KIT receptor mutations, and the company also recently presented preclinical data on a JAK2 V617F mutation-specific inhibitor at the European Hematology Association Congress 2026.

Cogent Biosciences has submitted a new drug application to the U.S. Food and Drug Administration for bezuclastinib as a treatment for advanced systemic mastocytosis. The submission is supported by data from the pivotal Phase 3 APEX trial, which enrolled 81 patients and showed that bezuclastinib achieved at least a 50% reduction in bone marrow mast cell concentration and normalized serum tryptase levels in 89% of patients. The overall response rate was 81%, with 12-month data indicating a 79% progression-free survival rate and an 87% overall survival rate. Bezuclastinib is a selective KIT inhibitor targeting mutations common in advanced systemic mastocytosis, a rare blood neoplasm causing excess mast cell proliferation.

Cogent Biosciences presented early-stage preclinical data showing its experimental drug CGT1145 was more than 100-fold selective for the JAK2 V617F mutation over normal JAK2 and other isoforms. The mutation is associated with rare blood cancers including myelofibrosis, polycythemia vera, and essential thrombocythemia. The company designed CGT1145 as a mutant-selective JAK2 inhibitor that may avoid the side effects of existing treatments, which hit both mutated and normal versions of the gene and can cause low blood counts. The data also suggested the drug could eliminate cancer-driving cells at the root level and support molecular remission, while demonstrating strong oral bioavailability. President and CEO Andrew Robbins said Cogent is on track to file an Investigational New Drug application with the FDA later this year.