Blueprint hits goal in midphase rare disease trial, teeing up pivotal study

A phase 2 trial has linked Blueprint Medicines’ avapritinib to statistically significant improvements in the symptoms of patients with indolent systemic mastocytosis (SM). The results tee Blueprint up to move the kinase inhibitor into the pivotal efficacy part of the midphase rare disease clinical trial.

Blueprint hits goal in midphase rare disease trial, teeing up pivotal study
A phase 2 trial has linked Blueprint Medicines’ avapritinib to statistically significant improvements in the symptoms of patients with indolent systemic mastocytosis (SM). The results tee Blueprint up to move the kinase inhibitor into the pivotal efficacy part of the midphase rare disease clinical trial.