Agios Showcases RISE UP Phase 3 Results at EHA 2026 Plenary Session Reinforcing Strong Anti-Hemolytic Profile of Mitapivat in Sickle Cell Disease

Agios presented detailed 52-week results from the RISE UP Phase 3 trial of mitapivat in adults and adolescents with sickle cell disease, showcased at EHA 2026 in Stockholm. The study demonstrated a statistically significant improvement in hemoglobin response versus placebo, with rapid onset and durability. New analyses highlighted reductions in transfusion burden and, among hemoglobin responders, meaningful decreases in sickle cell pain crises, fatigue, and related outcomes. Mitapivat was generally well tolerated, aligning with prior trials’ safety profile. Although the primary endpoint of reducing pain crises did not reach statistical significance overall, the data suggest that patients achieving hemoglobin responses experience tangible clinical benefits, underscoring mitapivat’s potential for treating sickle cell disease.