Blueprint's endpoint change irks markets

10 June 2022

Blueprint Medicines (Nasdaq: BPMC) has suffered in the stock market after announcing plans to update the primary endpoint of the registrational PIONEER trial of Ayvakit (avapritinib) in indolent systemic mastocytosis (SM).

The change is based on a recommendation from the US Food and Drug Administration (FDA) and means that the mean absolute change in total symptom score (TSS), previously a key secondary endpoint, will be the primary endpoint.

Meanwhile, the proportion of patients with a 30% or greater decrease in TSS, previously the primary endpoint, will be a key secondary endpoint.

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