Blueprint Medicines reports new Phase 1 data of BLU-285
Blueprint Medicines announced new Phase 1 clinical data for BLU-285, a highly selective KIT and PDGFRalpha inhibitor in development as a potential treatment for patients with advanced gastrointestinal stromal tumors, or GIST. The data confirm and build upon data previously presented for BLU-285 in patients with advanced GIST. 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 PDGFRalpha-D842 driven GIST, the data showed an ORR of 71% and an estimated 12-month PFS of 78 percent. BLU-285 was well-tolerated. Blueprint Medicines plans to pursue expedited development of BLU-285 in patients with PDGFRalpha D842V-driven GIST, and the company is on track to initiate a global, randomized Phase 3 clinical trial of BLU-285 in third-line GIST in 1H18, with the goal of supporting registration of BLU-285 in a broader GIST patient population.