Systemic mastocytosis is a blood disorder characterized by abnormal accumulation of mast cells in bone marrow and other organs, producing multisystem symptoms. It includes several subtypes ranging from indolent to aggressive disease.
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.
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 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 |
|---|---|---|---|
| NCT07562542 |
Peripheral Blood KIT-D816V Mutation in Adult Systemic Mastocytosis |
RECRUITING | |
| NCT07142473 |
Mastocytosis From Pediatric Age to Adulthood: Local Registry of Cutaneous and Systemic Mastocytosis |
RECRUITING | |
| NCT04681105 |
Flotetuzumab for the Treatment of Relapsed or Refractory Advanced CD123-Positive Hematological Malignancies |
COMPLETED | PHASE1 |
| NCT03739606 |
Flotetuzumab in Treating Patients With Recurrent or Refractory CD123 Positive Blood Cancer |
WITHDRAWN | PHASE2 |
| NCT03401060 |
Interest of Denosumab Treatment in Osteoporosis Associated to Systemic Mastocytosis |
COMPLETED | PHASE3 |
| NCT03214666 |
GTB-3550 Tri-Specific Killer Engager (TriKE®) for High Risk Hematological Malignancies |
TERMINATED | PHASE1/PHASE2 |
| NCT02478957 |
Treatment of Indolent Systemic Mastocytosis With PA101 |
COMPLETED | PHASE2 |
| NCT02441166 |
Diagnostic Value of Bone Marrow Tryptase in Systemic Mastocytosis |
COMPLETED | NA |
| NCT02415608 |
Ibrutinib in Treating Patients With Advanced Systemic Mastocytosis |
TERMINATED | PHASE2 |
| NCT01807598 |
Brentuximab Vedotin in Treating Patients With Advanced Systemic Mastocytosis or Mast Cell Leukemia |
COMPLETED | PHASE2 |
