Cogent Biosciences is set to present new data on bezuclastinib, a selective tyrosine kinase inhibitor, at the 2025 American Academy of Allergy Asthma & Immunology (AAAAI) Annual Meeting, taking place in San Diego from February 28 to March 3. The poster presentation will focus on the long-term symptomatic benefits observed in patients with non-advanced systemic mastocytosis (NonAdvSM) who have been treated with 100mg of bezuclastinib for at least 48 weeks.
The presentation, titled "Efficacy and Safety Results of Adult Patients with NonAdvanced Systemic Mastocytosis Receiving Bezuclastinib 100 mg in the Ongoing Summit Trial: A Randomized, Double-Blind, Placebo Controlled Phase 2 Clinical Trial of Bezuclastinib," will take place on March 1, 2025, from 9:45am to 10:45am PT in Hall A of the San Diego Convention Center.
Bezuclastinib's Clinical Development
Bezuclastinib is designed to selectively inhibit the KIT D816V mutation, a primary driver of systemic mastocytosis, as well as other mutations in KIT exon 17. Systemic mastocytosis is a rare and debilitating disease characterized by the excessive accumulation of mast cells in various organs. The KIT D816V mutation is present in approximately 95% of patients with systemic mastocytosis.
Cogent Biosciences is currently conducting the SUMMIT trial, a registration-directed, global, randomized, placebo-controlled trial of bezuclastinib in patients with NonAdvSM. Top-line results from the SUMMIT trial are expected in July 2025. The company also anticipates reporting top-line results from the APEX trial, an open-label study in advanced systemic mastocytosis (AdvSM), in the second half of 2025. If the results are favorable, Cogent plans to submit its first New Drug Application (NDA) for bezuclastinib by the end of 2025.
Expanded Access Programs
In addition to the ongoing clinical trials, Cogent Biosciences plans to initiate Expanded Access Programs (EAP) in the U.S. during the first quarter of 2025. These programs will allow eligible patients with systemic mastocytosis and gastrointestinal stromal tumors (GIST) to receive bezuclastinib prior to potential regulatory approval.
Broader Pipeline Development
Beyond bezuclastinib, Cogent Biosciences is also advancing a pipeline of novel small molecule programs. This includes an ongoing Phase 1 study of CGT4859, a selective FGFR2 inhibitor, in patients with documented FGFR mutations, including advanced cholangiocarcinoma. The company also plans to submit Investigational New Drug (IND) applications for its ErbB2 and PI3Kα programs in 2025.
Andrew Robbins, President and CEO of Cogent Biosciences, stated that 2025 is expected to be a transformational year for the company, with the potential to change the lives of thousands of patients fighting SM and GIST. He also highlighted bezuclastinib's potential to be the first potent, CNS-sparing, selective KIT mutant inhibitor.
