Advanced systemic mastocytosis is a severe form within systemic mastocytosis characterized by aggressive disease behavior and poorer outcomes than indolent forms. It involves pathologic mast cell accumulation in bone marrow and other organs and may include aggressive systemic mastocytosis or mast cell leukemia phenotypes.
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 |
|---|---|---|---|
| NCT05609942 |
Study of Elenestinib (BLU-263) in Advanced Systemic Mastocytosis (AdvSM) and and Other KIT Altered Hematologic Malignancies |
TERMINATED | PHASE1 |
| NCT04695431 |
Retrospective Study Assessing the Effect of Avapritinib Versus Best Available Therapy in Patients With AdvSM |
COMPLETED | |
| NCT03580655 |
(PATHFINDER) Study to Evaluate Efficacy and Safety of Avapritinib (BLU-285), A Selective KIT Mutation-targeted Tyrosine Kinase Inhibitor, in Patients With Advanced Systemic Mastocytosis |
COMPLETED | PHASE2 |
| NCT02571036 |
A Safety, Tolerability and PK Study of DCC-2618 in Patients With Advanced Malignancies |
COMPLETED | PHASE1 |
