- Updated results from a Phase 1 clinical trial evaluating Blueprint Medicines' (BPMC -1.6%) BLU-285 in patients with advanced gastrointestinal stromal tumors (GIST) showed a treatment effect. The data are being presenting today at the CTOS meeting in Hawaii.
- 67% of patients with heavily pretreated KIT-driven GIST who received once-daily BLU-285 (300 mg - 400 mg) showed radiographic tumor reductions. In this group, the objective response rate (ORR) was 17% with median progression-free survival (PFS) of 11.5 months.
- In patients with PDGFRα-D842-driven GIST, ORR was 71% with an estimated 12-month PFS of 78%.
- BLU-285 was well-tolerated with most adverse events being mild or moderate.
- A global randomized Phase 3 study in third-line GIST should be initiated in H1 2018.
- BLU-285 is a highly selective inhibitor of the abnormally active receptor tyrosine kinase mutants KIT and PDGFRα, including KIT exon 17 and PDGFRα-D842V mutations which have no currently effective treatment options.
- Previously: Blueprint's BLU-285 shows encouraging action in early-stage GIST study (Dec. 1, 2016)
- Now read: Applied Genetic Technologies Corp.: A Forgotten Net-Net With Clear Upside Catalysts
Original article