Mitapivat raises hemoglobin levels in Phase 3 sickle cell disease trial

Agios Pharmaceuticals’ medication mitapivat rapidly increased levels of the oxygen-carrying protein hemoglobin in patients ages 16 and older with sickle cell disease (SCD), while also being linked to a lower blood transfusion burden over 52 weeks.

These are new data from the Phase 3 portion of the global Phase 2/3 RISE UP clinical trial (NCT05031780), which tested mitapivat oral tablets, taken twice daily, against a placebo. The trial’s two main goals were to assess hemoglobin response and annualized rates of sickle cell pain crises, also called vaso-occlusive crises (VOCs).