Data From Blueprint Medicines' Blood Disorder Trial Fails To Lift Shares

Data From Blueprint Medicines' Blood Disorder Trial Fails To Lift Shares

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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