- Mitapivat demonstrated statistically significant improvement in hemoglobin response compared with placebo, with rapid onset and durable effects
- New analyses showed patients in mitapivat arm had clinically meaningful reduction in transfusion burden compared with placebo
- Patients in mitapivat arm who achieved hemoglobin response had clinically meaningful 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 of mitapivat in sickle cell disease
- Company to host investor conference call and webcast today at 9:00 a.m. ET (3:00 p.m. CEST)
CAMBRIDGE, Mass., June 13, 2026 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a commercial-stage biopharmaceutical company focused on delivering innovative medicines for patients with rare diseases, today presented detailed results from the 52-week double-blind period of the global RISE UP Phase 3 trial of mitapivat, an oral pyruvate kinase (PK) activator, in patients aged 16 years or older with sickle cell disease. These efficacy and safety results, which include new transfusion burden and hemoglobin responder analyses reinforcing the strong anti-hemolytic profile of mitapivat, were presented during the distinguished Plenary Abstracts Session at the 31st European Hematology Association (EHA) Congress (EHA 2026) in Stockholm, Sweden.