Phase 2 trial of ponatinib in patients with metastatic gastrointestinal stromal tumor following failure or intolerance of prior therapy with imatinib (POETIG trial)

The POETIG trial was a phase 2 study evaluating lower-dosed ponatinib (30 mg) in patients with metastatic gastrointestinal stromal tumors resistant to prior therapies like imatinib. The trial's primary endpoint (16-week clinical benefit rate) was not met. However, ponatinib showed clinical benefit in a subset of patients, particularly those with exon 11 and/or T670I mutations. Common side effects included hypertension and gastrointestinal issues, with cardiovascular events in 4 of 46 patients.