Clinical TrialJun 13, 2026, 09:01 AM
Agios Presents Positive Mitapivat Phase 3 Sickle Cell Data at EHA
AI Summary
Agios Pharmaceuticals presented detailed 52-week results from the global RISE UP Phase 3 trial of mitapivat in sickle cell disease at the 31st European Hematology Association (EHA) Congress. The data demonstrated statistically significant improvement in hemoglobin response and clinically meaningful reductions in transfusion burden. Patients who achieved a hemoglobin response also experienced significant benefits across measures of sickle cell pain crises, fatigue, and other patient-reported outcomes. Mitapivat was well-tolerated, with a safety profile consistent with previous trials, reinforcing its potential as a differentiated anti-hemolytic approach for this underserved patient population.
Key Highlights
- Mitapivat achieved statistically significant hemoglobin response (40.6% vs. 2.9% for placebo, p<0.0001).
- Mitapivat arm showed a 41.1% relative reduction in patients requiring blood transfusions vs. placebo.
- Mitapivat arm had a 55.9% relative reduction in average red blood cell units transfused per patient vs. placebo.
- Hemoglobin responders in mitapivat arm experienced a 26% reduction in annualized sickle cell pain crises.
- Responders also saw a 34% reduction in related hospitalizations and 53% fewer ER visits for SCPCs.
- Clinically meaningful improvements in fatigue scores were reported by hemoglobin responders (-5.19 vs. -2.55).
- Mitapivat's safety profile was consistent with previous trials, with similar adverse event rates (97.1% vs. 98.6%).
- Agios submitted a supplemental New Drug Application (sNDA) to the US FDA in May 2026 for accelerated approval.
Price Impact
More from AGIO