Gastrointestinal stromal tumors (GISTs) are rare mesenchymal neoplasms of the gastrointestinal tract, commonly driven by activating KIT or PDGFRA mutations and arising from interstitial cells of Cajal. They can occur throughout the GI tract, most often in the stomach and small intestine. Clinical behavior ranges from indolent to malignant, and management depends on tumor risk features and molecular profile.
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.
Researchers are developing personalized treatment approaches for rare and pediatric cancers using genetic profiling, AI-driven dosing, and 3D-printed medications to improve outcomes while reducing long-term toxicity.
New research from the University of Missouri-Columbia reveals that targeted cancer therapies, including tyrosine kinase inhibitors, antibody-drug conjugates, and CAR-T cell therapies, cause distinct patterns of gastrointestinal injury that are often underrecognized.
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 |
|---|---|---|---|
| NCT07454967 |
Development of a Multimodal AI System for GIST Management |
COMPLETED | |
| NCT07171203 |
Neoadjuvant Imatinib and Fampridine in KIT Mutant Gastrointestinal Stromal Tumor |
RECRUITING | PHASE1 |
| NCT06805825 |
A Study of the c-Kit Specific Antibody-Drug Conjugate NN3201 for Advanced and/or Metastatic Solid Tumors Known to Express c-Kit |
RECRUITING | PHASE1 |
| NCT06523582 |
Genetic Bases of Neuroendocrine Neoplasms in Mexican Patients |
RECRUITING | |
| NCT06507683 |
Evaluate the Efficacy and Safety of Radiotherapy Combined With Ripretinib in the Treatment of Unresectable Advanced GIST |
RECRUITING | NA |
| NCT06431451 |
The Correlation Between Ripretinib Exposure and the Efficacy and Safety in Patients with Advanced GISTs |
RECRUITING | |
| NCT06326346 |
GIST Oral Paclitaxel(Liporaxel) |
RECRUITING | PHASE2 |
| NCT06208748 |
SARC044: A Phase II Trial of Bezuclastinib in Combination With Sunitinib in Patients With GIST |
ACTIVE_NOT_RECRUITING | PHASE2 |
| NCT06087263 |
Phase 2 Study to Evaluate the Efficacy of Regorafenib in Specific GIST Mutation Subsets (KIT Exon 17, 18, or 14 Mutation and SDHB Deficient GIST) in the Post-imatinib Second-line Setting. |
ACTIVE_NOT_RECRUITING | PHASE2 |
| NCT05938309 |
A Cohort Study on the Safety of Laparoscopic Resection of 5cm or Larger Gastric Gastrointestinal Stromal Tumors |
RECRUITING |
