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Agios Announces Publication of Phase 2 Data in The Lancet Demonstrating Safety and Efficacy of PYRUKYND® (mitapivat) in Non-transfusion-dependent α- and β-Thalassemia
– In Adults with Non-transfusion-dependent α- or β-Thalassemia, PYRUKYND® Induced ≥1.0 g/dL Hemoglobin Increase from Baseline in 16 of 20 (80%) Patients
About this update from Agios Pharmaceuticals, Inc.
[{"type":"text","content":"– In Adults with Non-transfusion-dependent α- or β-Thalassemia, PYRUKYND® Induced ≥1.0 g/dL Hemoglobin Increase from Baseline in 16 of 20 (80%) Patients Between Weeks 4-12 – – PYRUKYND® Safety Profile Consistent with Label for FDA-approved Indication in Pyruvate Kinase Deficiency – – Actively Enrolling Phase 3 ENERGIZE and ENERGIZE-T Studies Evaluating PYRUKYND® in Adults with Non-transfusion-dependent and Transfusion-dependent α- or β-Thalassemia, Respectively – CAMBRIDGE, Mass., Aug. 11, 2022 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism pioneering therapies for genetically defined diseases, today announced that data from the core period of the open-label, Phase 2 study of PYRUKYND® (mitapivat) in adults with non-transfusion-dependent α- or β-thalassemia were published on August 11, 2022, in The Lancet. Data from this study were previously presented at the 2021 European Hematology Association (EHA) Annual Congress. PYRUKYND® is a first-in-class, investigational, oral, small molecule allosteric activator of wild-type and a variety of mutated pyruvate kinase (PK) enzymes. The publication can be accessed at the following link: https://www.thelancet.com/journals/lancet/article/PIIS0140-6736(22)01337-X/fulltext “Consistent with previously reported findings, the data reported today underscore the potential of PK activation to improve hallmarks of α- and β-thalassemia, including hemolysis and ineffective erythropoiesis,” said Kevin Kuo, M.D., hematologist at University of Toronto, Toronto General Hospital, and an investigator in the study. “Thalassemia is a rare, debilitating lifelong blood disorder characterized by severe complications, with no treatment options for those with α-thalassemia and limited options for those with β-thalassemia. These data demonstrate the potential clinical benefits of mitapivat for a broad spectrum of thalassemia patients and support its continued investigation in pivotal trials.” “We are encouraged by The Lancet’s publication of our Phase 2 thalassemia data for PYRUKYND®, which has the potential to serve as several important firsts for patients: the first oral therapy for thalassemia, and first treatment for all types of thalassemia – α and β, transfusion-dependent and non-transfusion-dependent,” said Sarah Gheuens, M.D., Ph.D., head of R&D ...