GIST stands for gastrointestinal stromal tumor, a rare mesenchymal neoplasm of the gastrointestinal tract often driven by KIT or PDGFRA activating mutations. It most commonly arises in the stomach or small intestine and has variable malignant potential. Risk stratification includes tumor size, mitotic activity, and location.
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.
Biodexa Pharmaceuticals has licensed MTX240, a Phase 1-ready molecular glue therapy for gastrointestinal stromal tumors from Otsuka. The drug demonstrated preclinical efficacy in resistant tumors and is eligible for orphan drug exclusivity, with clinical trials planned by year-end.
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 |
|---|---|---|---|
| NCT07411118 |
RCT of EFTR Versus STER for GIST Treatment |
RECRUITING | NA |
| NCT07406633 |
KQB198 in Combination With Imatinib in Participants With Advanced/Metastatic GIST in 1st Line Setting |
RECRUITING | PHASE2 |
| NCT07096609 |
Lenvatinib After Progression on Imatinib, Sunitinib, and Regorafenib for GIST Patients |
RECRUITING | PHASE1/PHASE2 |
| NCT06640361 |
A Study of Olverembatinib in SDH-deficient GIST. |
RECRUITING | PHASE3 |
| NCT06441331 |
Phase I Trial to Determine the Dose and Evaluate the PK and Safety of Lutetium Lu 177 Edotreotide Therapy in Pediatric Participants With SSTR-positive Tumors |
RECRUITING | PHASE1 |
| NCT06208748 |
SARC044: A Phase II Trial of Bezuclastinib in Combination With Sunitinib in Patients With GIST |
ACTIVE_NOT_RECRUITING | PHASE2 |
| NCT05957367 |
A Study of Inlexisertib (DCC-3116) in Combination With Anticancer Therapies in Participants With Advanced Malignancies |
RECRUITING | PHASE1/PHASE2 |
| NCT05895942 |
Exploring the Molecular Mechanism Based on KIT Mutation |
UNKNOWN | |
| NCT05804331 |
The Australia and New Zealand Multicentre Upper Gastrointestinal Endoscopic Tissue Resection Study |
RECRUITING | |
| NCT05734105 |
A Study of Ripretinib vs Sunitinib in Patients With Advanced GIST With Specific KIT Exon Mutations Who Were Previously Treated With Imatinib |
ACTIVE_NOT_RECRUITING | PHASE3 |
