- Blueprint Medicines Corporation BPMC announced detailed results from the PIONEER Phase 2 trial of Ayvakit (avapritinib) in patients with indolent systemic mastocytosis (SM).
- As previously reported, Ayvakit achieved statistically significant and clinically meaningful improvements on the primary and all key secondary endpoints.
- New results further highlight the benefits of Ayvakit on pathological mast cell burden, disease symptoms – including total symptom score (TSS), most severe symptom, and all individual symptoms – and quality of life.
- Across clinical measures, improvements continued to deepen over time in patients treated with Ayvakit through 48 weeks.
- Related: Data From Blueprint Medicines’ Blood Disorder Trial Fails To Lift Shares.
- Ayvakit achieved a statistically significant and clinically meaningful improvement in total symptom scores that deepened over time, showing improvements across all individual symptoms.
- Ayvakit had a favorable safety profile compared to placebo, supporting the potential for chronic treatment. 96% of patients in the Ayvakit arm opted to continue treatment in the open-label extension study.
- Blueprint Medicines regained global commercialization and development rights to Gavreto (pralsetinib), excluding Greater China, following a decision by Roche Holdings AG RHHBY to discontinue the collaboration agreement for strategic reasons.
- Price Action: BPMC shares traded 1.92% higher at $38.70 on the last check Monday.
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