Agios Pharmaceuticals announces new data on mitapivat

Agios Pharmaceuticals (AGIO) announced that new data on mitapivat, an oral pyruvate kinase activator, will be featured in oral and poster presentations during the 67th American Society of Hematology, ASH, Annual Meeting and Exposition in Orlando, Florida, December 6-9, 2025. Select presentations and publications at ASH 2025 will include: Two poster presentations on results from the ENERGIZE-T Phase 3 trial of mitapivat in adults. The first is a subgroup analysis of patients with alpha-thalassemia, showing that 77.8% of individuals in the mitapivat arm achieved the primary endpoint of transfusion reduction response, compared to 0% (0 of 3) in the placebo arm. The second highlights long-term results from the 17 patients who achieved transfusion independence with mitapivat during the double-blind phase of ENERGIZE-T, showing that the mean duration of their longest transfusion-free period was 30.5 weeks, with a maximum of 84.3 weeks, across the double-blind and ongoing open-label extension periods. An oral presentation with preclinical data from an investigator-led study demonstrating that mitapivat protects against cardiomyopathy in a mouse model of beta-thalassemia, with this mechanism potentially linked to its activation of the PKM2 isoform of the PK enzyme in the heart. A poster presentation with positive findings from the ACTIVATE-Kids Phase 3 trial of mitapivat in children aged 1 to less than18 years with PK deficiency who are not regularly transfused. The trial met its primary endpoint, with the mitapivat arm showing a higher hemoglobin response rate compared to the placebo arm. Research from two Agios-supported advisory councils, each comprised of patients, caregivers, advocates, and physicians, that builds on clinical and preclinical findings to help advance the scientific understanding of rare blood disorders.