Agios Announces Publication of Phase 3 ACTIVATE Study in New England Journal of Medicine Demonstrating Benefits of PYRUKYND® (mitapivat) for Adults with Pyruvate Kinase Deficiency

– In Adults with Pyruvate Kinase (PK) Deficiency Who Are Not Regularly Transfused, PYRUKYND® Significantly Increased Hemoglobin Level, Decreased Hemolysis and Improved Patient-Reported Outcomes –

– Following FDA Approval in February, PYRUKYND® Is the First and Only Disease-Modifying Treatment for Adults with PK Deficiency –

CAMBRIDGE, Mass., April 14, 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 pivotal Phase 3 ACTIVATE study of PYRUKYND® (mitapivat) in adults with pyruvate kinase (PK) deficiency who do not receive regular transfusions were published on April 14, 2022 in the New England Journal of Medicine. Data from this study were previously presented at the European Hematology Association (EHA) Virtual Congress held in June 2021. PYRUKYND® is a first-in-class, oral PK activator and the first and only approved disease-modifying treatment for this rare, debilitating, lifelong hemolytic anemia.

The publication can be accessed at the following link: https://www.nejm.org/doi/full/10.1056/NEJMoa2116634

“The results of the ACTIVATE study confirm that mitapivat, through its novel mechanism of activating pyruvate kinase and thus increasing ATP levels in red blood cells, successfully addressed the underlying cause of chronic hemolytic anemia in adults with PK deficiency with a wide array of genotypes,” said Hanny Al-Samkari, M.D., hematologist and clinical investigator at the Mass General Cancer Center and Harvard Medical School, an investigator in the pivotal ACTIVATE Phase 3 study and first author of this publication. “Improvements in the PK deficiency–specific patient-reported outcome measures further support the clinical efficacy of mitapivat and its benefits on health-related quality of life and reduction in symptom severity.”

“We have been pioneering the science of PK activation for more than a decade, and are pleased to have developed the first approved product for people with PK deficiency who previously had no disease-modifying treatment options,” said Sarah Gheuens, M.D., Ph.D., chief medical officer at Agios. “The results of the ACTIVATE study underscore the clinical value of PYRUKYND® and support our current efforts to deliver this medicine to as many patients as possible who may benefit from it.”

As reported in the publication, the ACTIVATE study met its primary endpoint, with 40 percent of patients randomized to PYRUKYND® achieving a hemoglobin response, defined as a ≥1.5 g/dL increase in hemoglobin concentration from baseline sustained at two or more scheduled assessments at Weeks 16, 20 and 24 during the fixed-dose period, compared to 0 patients randomized to placebo (2-sided p