13 November 2017 - - Cambridge, Massachusetts-based targeted kinase medicine developer Blueprint Medicines Corp. (NASDAQ: BPMC) has released new Phase 1 clinical data for BLU-285, a potent and highly selective KIT and PDGFRα inhibitor in development as a potential treatment for patients with advanced gastrointestinal stromal tumors (GUST), the company said.
The data confirm and build upon data previously presented for BLU-285 in patients with advanced GIST, demonstrating robust clinical activity and a favorable safety profile.
The data showed 67% of patients with heavily pretreated KIT-driven GIST treated with 300 to 400 mg of BLU-285 once daily had radiographic tumor reductions. In this population, the data also showed an objective response rate of 17% and median progression free survival of 11.5 months.
In patients with PDGFRα-D842 driven GIST, the data showed an ORR of 71% and an estimated 12-month PFS of 78%. BLU-285 was well-tolerated, and most adverse events reported by investigators were Grade 1 or 2.
The Phase 1 clinical trial of BLU-285 is designed to evaluate the safety and tolerability of BLU-285 in adults with advanced GIST. The trial consists of two parts, a dose-escalation portion and an expansion portion.
The dose-escalation portion is complete, and the MTD was determined to be 400 mg QD. Blueprint Medicines has selected 300 mg QD as the RP2D for the dose expansion portion of the trial.
The expansion portion, designed to enroll a total of approximately 200 patients at multiple sites in the United States, United Kingdom and European Union, is actively enrolling patients in three defined cohorts at the RP2D consisting of (1) a cohort of patients with a PDGFRα D842V mutation, regardless of line of therapy, (2) a cohort of patients who have received imatinib and at least one other KIT-directed TKI and (3) a cohort of patients who have received imatinib and no other TKI.
Blueprint Medicines is initially developing BLU-285, an investigational medicine, for the treatment of patients with advanced GIST and advanced systemic mastocytosis.
Blueprint Medicines is developing a new generation of targeted and potent kinase medicines to improve the lives of patients with genomically defined diseases.
The company is advancing four programmes in clinical development for subsets of patients with gastrointestinal stromal tumors, hepatocellular carcinoma, systemic mastocytosis, non-small cell lung cancer, medullary thyroid cancer and other advanced solid tumors, as well as multiple programs in research and preclinical development.