Agios presents results from Phase 2 portion of RISE UP pivotal study

Agios Pharmaceuticals presented detailed results from the Phase 2 portion of the global RISE UP study of mitapivat in sickle cell disease in an oral presentation at the 65th American Society of Hematology Annual Meeting & Exposition, which is being hosted Dec. 9-12, 2023, in San Diego. During the Phase 2 double-blind, placebo-controlled study, treatment with mitapivat demonstrated statistically significant improvement in hemoglobin response across both mitapivat dose levels, compared to placebo. The safety profile for mitapivat observed in the study was generally consistent with previously reported data in other studies of sickle cell disease and other hemolytic anemias. Improvements were observed in annualized rates of sickle cell pain crises, and markers of hemolysis and erythropoiesis for both mitapivat treatment arms compared to placebo. Improvement in patient-reported fatigue scores was observed with mitapivat 50 mg BID compared to placebo. Based on the Phase 2 data, Agios selected 100 mg BID as the Phase 3 dose and is currently enrolling patients in this portion of the RISE UP study. The operationally seamless Phase 2/3 study design allows Agios to leverage and create efficiencies in the start and conduct of the Phase 3 portion of RISE UP, with a goal of reporting the Phase 3 data in 2025 and potentially receiving U.S. approval in 2026.