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Agios Pharmaceuticals Says Topline Results From RISE UP Phase 3 Trial Of Mitapivat For Sickle Cell Disease Met Its Primary Endpoint Of Hemoglobin Response, While Mitapivat Did Not Achieve Statistical Significance On The Annualized Rate Of Sickle Cell Pain Crises Compared To Placebo

Benzinga·11/19/2025 12:06:10
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  • Trial met primary endpoint of hemoglobin response and key secondary endpoints of change from baseline in hemoglobin concentration and indirect bilirubin
  • Trial showed trend favoring mitapivat but did not meet statistical significance in primary endpoint of annualized rate of SCPCs (pain crises), and the key secondary endpoint of change from baseline in PROMIS Fatigue was not met
  • Patients in the mitapivat arm who achieved hemoglobin response had clinically meaningful benefits in SCPC-related endpoints and PROMIS Fatigue
  • Favorable safety profile observed in RISE UP Phase 3 trial was consistent with that observed in prior mitapivat sickle cell disease trials
  • Company will share the comprehensive results from the RISE UP clinical program with the FDA and intends to submit for potential U.S. regulatory approval