Bezuclastinib is an investigational tyrosine kinase inhibitor targeting KIT mutations and is being evaluated in solid tumors and systemic mastocytosis. It is an oral small molecule with development codes CGT-9486 and PLX-9486. The drug is not yet approved as a marketed therapy.
The FDA granted Breakthrough Therapy Designation to bezuclastinib plus sunitinib for previously treated GIST. The PEAK trial showed median progression-free survival of 16.5 months versus 9.2 months for sunitinib alone.
January and March 2026 drug pipeline updates included Phase 3 results, Fast Track designations and Priority Review decisions across multiple disease areas. Mavacamten, teplizumab-mzwv, anifrolumab, AXS-05, gedatolisib and AFTX-201 were among the programs with notable milestones.
Cogent Biosciences has submitted a New Drug Application for bezuclastinib to treat systemic mastocytosis caused by the KIT D816V mutation, with FDA review underway and strong analyst support.
Cogent Biosciences is preparing parallel FDA submissions for bezuclastinib across three indications following positive pivotal trial results, with potential launches beginning in late 2026 and full commercialization expected by early 2027.
Cogent Biosciences presented additional SUMMIT trial data showing bezuclastinib achieved 56% improvement in symptoms at 48 weeks in NonAdvSM patients, with multiple regulatory submissions advancing toward expected commercial launch in second half of 2026.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT06948955 |
Expanded Access to Bezuclastinib to be Coadministered With Sunitinib for Patients With Gastrointestinal Stromal Tumors |
AVAILABLE | |
| NCT06915766 |
Expanded Access to Bezuclastinib for Patients With NonAdvanced Systemic Mastocytosis or Advanced Systemic Mastocytosis |
AVAILABLE | |
| NCT04996875 |
(Apex) Bezuclastinib in Patients With Advanced Systemic Mastocytosis |
RECRUITING | PHASE2 |
