“BLU-285 has been a wonderful success in terms of getting a majority of patients in complete and partial remissions,” he said. “So, as this evolves, having better targeted agents, will improve the outcome. The second thing that it does, once you have successful therapeutic options for patients, then you can pick up and diagnose more patients. It is impressive how many newly diagnosed patients were seen that were misdiagnosed previously.”
Based on safety profile, PK, and anti-tumor activity, the researchers selected 300 mg as the RP2D. As of Nov. 27, 2017, 7 patients were enrolled in part 2 of the study, designed to evaluate dose expansion in additional patients, including 15 with ASM, 15 with SM-AHN, and 5 with MCL.
The researchers are currently planning a phase II study designed to assess the effectiveness of a once-daily dose of 300 mg BLU-285 in patients with ASM. In addition, DeAngelo said researchers are evaluating the agent for the treatment of indolent SM and smoldering SM.
DeAngelo DJ, Quiery AT, Radia D, et al Clinical activity in a phase I study of blu-285, a potent, highly-selective inhibitor of kit D816V in advanced systemic mastocytosis (AdvSM). Presented at: ASH Annual Meeting and Exposition; Dec. 9-12, 2017; Atlanta. Abstract 2.