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Agios Presents Positive Results from Phase 2 Study of Mitapivat in Non-transfusion-dependent α- and β-Thalassemia at the European Hematology Association Virtual Congress
– Study Met Primary Endpoint; Treatment with Mitapivat Induced Hemoglobin Increase of ≥1.0 g/dL in 16 of 20 (80%) Patients During Weeks 4-12 – – Mitapivat
About this update from Agios Pharmaceuticals, Inc.
[{"type":"text","content":"– Study Met Primary Endpoint; Treatment with Mitapivat Induced Hemoglobin Increase of ≥1.0 g/dL in 16 of 20 (80%) Patients During Weeks 4-12 – – Mitapivat Safety Profile Consistent with Previously Published Studies – – Agios to Host Investor Webcast Today at 7:30 a.m. ET – CAMBRIDGE, Mass., June 11, 2021 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism to treat genetically defined diseases, today announced positive results from its Phase 2, open-label, multicenter study of mitapivat in adults with non-transfusion dependent α- or β-thalassemia. Data from the study will be featured in an oral presentation on Tuesday, June 15, at the European Hematology Association (EHA) Virtual Congress. Consistent with previously announced proof-of-concept data, the study met its primary endpoint, with 16 of the 20 patients (80%) achieving a hemoglobin increase of ≥1.0 g/dL from baseline at one or more assessments during Weeks 4-12. Additionally, a sustained hemoglobin response and improvements in hemolysis and ineffective erythropoiesis were observed in both α- or β-thalassemia patients treated with mitapivat. Mitapivat was well tolerated, and the safety profile was consistent with previous studies. Mitapivat is a first-in-class, investigational, oral, small molecule allosteric activator of wild-type and a variety of mutated pyruvate kinase R (PKR) enzymes. “These data continue to validate the potential of PK activation as an entirely new mechanism for treating thalassemia, a disease for which there have been few medical advancements. In particular, we are excited to see data generated, for the first time, in α-thalassemia, demonstrating an increase in hemoglobin from baseline in all five patients in this subgroup,” said Kevin Kuo, M.D., hematologist at University Health Network, University of Toronto, and an investigator in the study. “The impressive results reported today underscore the potential of mitapivat to meaningfully improve hallmarks of this disease, including hemolysis and ineffective erythropoiesis.” Mitapivat Phase 2 Proof-of-concept StudyThe open-label Phase 2 study evaluated the efficacy, safety, pharmacokinetics and pharmacodynamics of mitapivat treatment in adults with either non-transfusion-dependent α- or β-thalassemia who have a baseline hemoglobin (Hb) concentratio...