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Agios Announces Data from Investigator-led Studies of Mitapivat in Adults with Sickle Cell Disease Demonstrating Improvements in Anemia, Hemolysis and Sickling Parameters at 63rd ASH Annual Meeting and Exposition
– Data Underscore Potential of Mitapivat to Provide Clinically Meaningful Outcomes for Patients – – Safety Profile Consistent with Previously Reported
About this update from Agios Pharmaceuticals, Inc.
[{"type":"text","content":"– Data Underscore Potential of Mitapivat to Provide Clinically Meaningful Outcomes for Patients – – Safety Profile Consistent with Previously Reported Clinical Data – – Agios Announces Initiation of Phase 2/3 RISE UP Study of Mitapivat in Adults with Sickle Cell Disease – – Agios to Host Investor Webcast on Dec. 14, 2021, at 7:30 a.m. ET – CAMBRIDGE, Mass., Dec. 13, 2021 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism to treat genetically defined diseases, today announced new data from investigator-led studies of mitapivat, a first-in-class, investigational, oral, small molecule allosteric activator of wild-type and a variety of mutated pyruvate kinase (PK) enzymes, in adults with sickle cell disease. The data, reported by the National Institutes of Health (NIH) and University Medical Center (UMC) Utrecht, were featured in two presentations at the American Society of Hematology (ASH) Annual Meeting and Exposition, hosted virtually and in person from Dec. 11-14, 2021, in Atlanta. The data demonstrate the potential of mitapivat to provide clinically meaningful outcomes for patients, including improvements in anemia, hemolysis and red blood cell sickling. Consistent with previously reported proof-of-concept data, mitapivat reduced 2,3-diphosphoglycerate (2,3-DPG) and increased adenosine triphosphate (ATP), and through this mechanism, may reduce hemoglobin S polymerization and red blood cell sickling. The safety profile observed in both studies was also generally consistent with previously published clinical data, including Phase 3 data in adults with pyruvate kinase deficiency. “Results reported from these investigator-led studies provide additional efficacy, safety and translational data that continue to support the clinical development of mitapivat in people with sickle cell disease, a lifelong, debilitating condition with few treatment options,” said Sarah Gheuens, M.D., Ph.D., chief medical officer at Agios. “We’d like to thank our collaborators at the NIH and UMC Utrecht and look forward to building upon their contributions through our recently initiated Phase 2/3 RISE UP trial. In collaboration with the sickle cell disease community, we designed this trial to have a broad global reach, reduce barriers to participation and understand how mitapivat can impact the aspe...