- Earlier today, Blueprint Medicines Corporation BPMC released the long-awaited pivotal PIONEER dataset in Indolent Systemic Mastocytosis (ISM).
- The topline data indicates that the trial met all primary and key secondary endpoints with statistical significance and that there were no intracranial bleeds.
- SVB Leerink thinks that though these data are approvable, they represent a deterioration in the absolute improvement in the symptom score (TSS), which was 15.6 points in this trial versus 19.7 in Phase 1.
- Conversely, the control arm of best supportive care (BSC) showed a larger reduction in TSS from a baseline of 9.2 points versus 3.2 in Phase 1.
- This represents a meaningful deterioration in the treatment effect for Ayvakit over BSC, now 6.4 from 16.5, a key metric that clinicians and payors are likely to weigh, given the anticipated cost.
- The analyst believes that the data will further magnify the commercial debate, as investors question the size of the addressable ISM population for a chronically used therapy with a limited treatment effect that could cost over $350,000 per year.
- Price Action: BPMC shares are down 7.19% at $63.67 on the last check Wednesday.
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