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 Phase 3 RISE UP trial results for mitapivat, an oral pyruvate kinase activator for sickle cell disease. The drug demonstrated statistically significant hemoglobin response improvement versus placebo with rapid onset and durable effects, clinically meaningful reduction in transfusion burden (41.1% relative reduction in patients requiring transfusions), and improvements in pain crises, fatigue, and quality of life measures among hemoglobin responders. Mitapivat was well-tolerated with a safety profile consistent with previous trials.