Positive results for mitapivat in pyruvate kinase deficiency who are regularly transfused in Phase 3 ACTIVATE-T trial

In the study, 37% of patients treated with mitapivat (small molecule allosteric activator of wild-type and mutated PKR enzymes) achieved ≥33% reduction in transfusion burden vs individual historical transfusion burden standardized to 24 weeks (p=0.0002).

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Biospace Inc.