Blueprint Medicines Corporation BPMC announced topline results from the registrational Part 2 of the PIONEER trial of Ayvakit (avapritinib) for non-advanced systemic mastocytosis (SM).
- SM is a rare disorder that results in too many mast cells (type of mast cells) building up in the body.
- Blueprint Medicines plans to submit a supplemental marketing application to the FDA for Ayvakitin in Q4 of 2022, with European submission in 2023.
- Related: This Analyst Thinks Blueprint Medicines' Non-Dilutive Funding 'Deflates M&A Speculation'
- The Ayvakit arm had a reduction of 15.6 points in mean symptom severity score at 24 weeks, which continued to deepen to 20.2 points at 48 weeks in patients who rolled over to the Part 3 open-label extension study.
- At 24 weeks, the control arm had a reduction of 9.2 points in the mean score.
- In addition, the PIONEER trial met all key secondary endpoints, including significant improvements across all measures of mast cell burden. More than half of Ayvakit-treated patients had over 50% reduction of serum tryptase, compared to no patients in the control arm.
- Ayvakit had a favorable safety profile compared to the control arm.
- The rate of adverse events was 90.8% in the Ayvakit arm and 93.0% in the control arm.
- Overall, 0.7% of patients in the Ayvakit arm and no patients in the control arm were discontinued due to treatment-related adverse events.
- Price Action: BPMC shares are down 24.1% at $52.04 on the last check Wednesday.
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