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Agios Presents Updated PYRUKYND® (mitapivat) Data Highlighting Long-term Safety Profile and Durable Improvement in Hemoglobin and Markers of Hemolysis and Ineffective Erythropoiesis in Non-transfusion-dependent α- and β-Thalassemia at 64th ASH Annual ...
Agios Presents Updated PYRUKYND® (mitapivat) Data Highlighting Long-term Safety Profile and Durable Improvement in Hemoglobin and Markers of Hemolysis and
About this update from Agios Pharmaceuticals, Inc.
[{"type":"text","content":"Agios Presents Updated PYRUKYND® (mitapivat) Data Highlighting Long-term Safety Profile and Durable Improvement in Hemoglobin and Markers of Hemolysis and Ineffective Erythropoiesis in Non-transfusion-dependent α- and β-Thalassemia at 64th ASH Annual Meeting and Exposition \n – 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 – – Agios to Host Live and Webcast Investor Event on Dec. 12, 2022, at 7 a.m. CT – CAMBRIDGE, Mass., Dec. 10, 2022 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism pioneering therapies for rare diseases, today reported new data from the ongoing long-term extension period of the Phase 2 open-label study of PYRUKYND® (mitapivat), a first-in-class, oral, small molecule allosteric activator of wild-type and a variety of mutated pyruvate kinase (PK) enzymes, in adults with non-transfusion dependent α- or β-thalassemia. Data from the study were featured in a poster presentation (abstract #1030) at the 64th American Society of Hematology (ASH) Annual Meeting and Exposition, hosted Dec. 10-13, 2022, in New Orleans. Consistent with previously reported data, durable improvements in hemoglobin concentration and markers of hemolysis and ineffective erythropoiesis were observed for up to 72 weeks of treatment in both α- and β-thalassemia patients. Additionally, markers of iron homeostasis remained stable or improved through Week 72. PYRUKYND® was well tolerated, and the safety profile was consistent with previous studies. “The data presented today continue to underscore the potential of PK activation to address multiple aspects of the complex underlying pathophysiology of α- and β-thalassemia, including hallmarks of the disease: 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, and there are no currently approved treatment options for those with α-thalassemia and options are limited for those with β-thalassemia. These data, along with long-term extension study data from ongoing studies of the treatment in pyruvate kinase deficiency, demonstrate the potential ...