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Agios Presents Positive Results from Phase 2 Portion of the RISE UP Pivotal Study in Sickle Cell Disease at 65th ASH Annual Meeting and Exposition
– Treatment with Mitapivat Demonstrated Statistically Significant Improvement in Hemoglobin Response Compared to Placebo – – Improvements Observed in
About this update from Agios Pharmaceuticals, Inc.
[{"type":"text","content":"– Treatment with Mitapivat Demonstrated Statistically Significant Improvement in Hemoglobin Response Compared to Placebo – – Improvements Observed in Annualized Rates of Sickle Cell Pain Crises, Markers of Hemolysis and Erythropoiesis in Participants Treated with Mitapivat – – Agios to Host Live and Webcast Investor Event on Dec. 11, 2023, at 7:00 a.m. Pacific Time – CAMBRIDGE, Mass., Dec. 09, 2023 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in the field of cellular metabolism pioneering therapies for rare diseases, today presented detailed results from the Phase 2 portion of the global RISE UP study of mitapivat in sickle cell disease in an oral presentation (abstract #271) at the 65th American Society of Hematology (ASH) Annual Meeting & Exposition, which is being hosted Dec. 9-12, 2023, in San Diego. During the Phase 2 double-blind, placebo-controlled study, treatment with mitapivat demonstrated statistically significant improvement in hemoglobin response across both mitapivat dose levels (50 mg and 100 mg BID), compared to placebo. The safety profile for mitapivat observed in the study was generally consistent with previously reported data in other studies of sickle cell disease and other hemolytic anemias. Improvements were observed in annualized rates of sickle cell pain crises, and markers of hemolysis and erythropoiesis for both mitapivat treatment arms compared to placebo. Improvement in patient-reported fatigue scores was observed with mitapivat 50 mg BID compared to placebo. “We are extremely pleased with the results of the Phase 2 portion of the RISE UP study and firmly believe that mitapivat has the potential to be a meaningful therapy for the sickle cell disease community,” said Sarah Gheuens, M.D., Ph.D., chief medical officer and head of R&D. “The data further add to the compelling and growing data set supporting the therapeutic potential of mitapivat, which has demonstrated consistent clinical and mechanistic improvements across three distinct hemolytic anemias. We look forward to building on these data through the Phase 3 portion of RISE UP and continuing to enroll patients around the world in this study.” “Sickle cell disease is a tremendously burdensome disease that impacts all aspects of patients’ and families’ lives. New therapeutic options – particularly those with convenient a...