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 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 –

Excerpt from the Press Release:

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.

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