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

Agios announced Phase 3 RISE UP trial results during the EHA 2026 plenary session, highlighting mitapivat’s efficacy in sickle cell disease. The trial demonstrated a statistically significant improvement in hemoglobin response compared to placebo, with rapid and durable effects. New analyses revealed a clinically meaningful reduction in transfusion burden—patients on mitapivat required 41.1% fewer transfusions and 55.9% fewer red blood cell units on average than those on placebo. Mitapivat also showed improvements in key secondary endpoints, including indirect bilirubin levels, a marker of hemolysis. While the trial did not meet statistical significance for reducing sickle cell pain crises (SCPCs) overall, patients who achieved a hemoglobin response on mitapivat experienced meaningful reductions in SCPCs, hospitalizations, and fatigue. These responders also reported better pain management, sleep, and physical function compared to non-responders. Biree Andemariam, M.D., Professor of Medicine at the University of Connecticut Health and a RISE UP investigator, emphasized the trial’s findings. She noted that mitapivat’s anti-hemolytic effects translate to tangible clinical benefits, addressing the relentless physical toll of sickle cell disease. The drug’s safety profile remained consistent with prior trials. Agios will host an investor conference call and webcast on June 10, 2026, at 9:00 a.m. ET to discuss the results further. The topline data from November 2025 confirmed mitapivat’s potential as a new treatment option for patients with sickle cell disease, particularly those with high transfusion needs.