Reported Saturday, Agios Presents RISE UP Phase 3 Data At EHA 2026 Showing Mitapivat Achieves 40.6% Hemoglobin Response Rate And 41.1% Reduction In Transfusion Burden Versus Placebo In Sickle Cell Disease

Agios Pharmaceuticals, Inc.

Agios Pharmaceuticals, Inc.

AGIO

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  • 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)
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