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Saturday, June 6, 2020

Blueprint Medicines avapritinib continues effect in mastocytosis study

Blueprint Medicines (NASDAQ:BPMC) announces updated data from part 1 of a Phase 2 clinical trial, PIONEER, evaluating Ayvakit (avapritinib) in patients with indolent (slow-growing) systemic mastocytosis. The results were virtually presented at the European Academy of Allergy and Clinical Immunology Congress (EAACI).
Week 24 data showed a deepening of symptom reductions in the 25 mg once-daily treatment group compared to week 16, reported in March. Specifically, the mean change from baseline in a scale called ISM-SAF was -35% compared to -4% in the control group. At week 16, the mean reduction was -30% in the 25 mg arm. The response rate was 60% versus 0% for placebo (defined as at least a 30% reduction in ISM-SAF total symptom score).
The company has selected the response rate at week 24 as the primary endpoint for registration-enabling part 2 of the study. Recruitment (planned enrollment of 200 subjects) will commence this month.
No new safety signals were observed.
Systemic mastocytosis is a rare disorder characterized by the uncontrolled proliferation and activation of mast cells in the body which, when triggered, leads to a range of symptoms like rapid heartbeat, lightheadedness and facial flushing. It is driven by a mutation in the KIT gene called D816V.
Avapritinib is a highly selective inhibitor of D816V-mutant KIT (protein called receptor tyrosine kinase).
https://seekingalpha.com/news/3580954-blueprint-medicines-avapritinib-continues-to-shows-effect-in-mastocytosis-study

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