– Actively initiating the Phase 3 ENERGIZE and ENERGIZE-T studies evaluating PYRUKYND^® in adults with non-transfusion-dependent or transfusion-dependent α- or β-thalassemia –

– Agios to Host Live and Webcast Investor Event on December 12, 2022 at 7am CT –

CAMBRIDGE, Mass., December 10, 2022 (GLOBE NEWSWIRE) — Agios Pharmaceuticals, Inc. AGIOa leader in breakthrough therapies for cellular metabolism in rare diseases, today announced new data from the ongoing long-term extension of its open-label Phase 2 study of PYRUKYND^® (Mitapivat), a first-in-class oral small molecule allosteric activator of wild-type and a variety of mutant pyruvate kinase (PK) enzymes, in adults with nontransfusion-dependent α- or β-thalassemia. Data from the study were presented in a poster presentation (Abstract #1030) at the 64th^th American Society of Hematology (ASH) Annual Meeting and Exhibition to be held December 10-13, 2022 in New Orleans.

In harmony with previously reported datasustained improvements in hemoglobin concentration and markers of hemolysis and ineffective erythropoiesis were observed in both α- and β-thalassemia patients for up to 72 weeks of treatment. In addition, markers of iron homeostasis remained stable or improved through week 72. PYRUCYND^® was well tolerated and the safety profile was consistent with previous studies.

“The data presented today further 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, MD, 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 currently no approved treatment options for patients with α-thalassemia and options are limited for patients with β-thalassemia. These dates, along with the long-term extension…