Blueprint Medicines Highlights Detailed Data From Lead Product In Blood Cancer Patients

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.