BPMC - Blueprint Medicines' avapritinib shows clinical benefit in late-stage systemic mastocytosis studies

Blueprint Medicines (BPMC) +3.8% in premarket, has announced top-line data from the Phase 1 EXPLORER and Phase 2 PATHFINDER clinical trials of AYVAKIT (avapritinib) in patients with advanced systemic mastocytosis ((SM)).In the EXPLORER trial, 53 patients were response evaluable, with a median follow-up of 27.3 months. overall response rate ((ORR)) was 76%, and 36% of patients had a complete remission with full or partial recovery of peripheral blood counts (CR/CRh). Median duration of response ((DOR)) was 38.3 months.In a pre-specified interim analysis from the PATHFINDER trial, 32 patients were response evaluable, with a median follow-up of 10.4 months. ORR was 75% and 19% of patients had a CR/CRh.In a pooled efficacy analysis from the 200 mg once daily dose group, 44 patients were response evaluable, with a median follow-up of 10.4 months, with ORR of 68%, and 18% of patients had a CR/CRh.AYVAKIT was generally well-tolerated with most adverse events (AEs) reported as mild to moderate. In both

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Blueprint Medicines' avapritinib shows clinical benefit in late-stage systemic mastocytosis studies