AgiosPharma is gearing up for topline data from its Phase 3 RISE UP trial (NCT05031780), evaluating mitapivat for sickle cell disease (SCD), by year-end. The oral PK activator aims to improve red blood cell health by boosting energy production and reducing harmful 2,3-DPG levels, potentially easing symptoms like pain crises. Phase 2 results showed promise, with mitapivat increasing hemoglobin levels and lowering annual pain crisis rates. The ongoing Phase 3 study, fully enrolled with 200+ participants, tests a 100 mg dose over 52 weeks. EU’s recent orphan drug designation underscores its potential for underserved SCD communities.

OxyDial applauds Agios’ commitment to advancing innovative SCD treatments. With high unmet needs in SCD, diversifying treatment approaches is critical to improving outcomes worldwide.

Read more: https://sicklecellanemianews.com/news/data-expected-2025-phase-3-trial-testing-mitapivat-scd/

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