Agios to share new insights reinforcing mitapivat’s potential across multiple rare hematologic diseases at ASH 2025

Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a commercial-stage biopharmaceutical company focused on delivering innovative medicines for patients with rare diseases, today announced that new data on mitapivat, an oral pyruvate kinase (PK) activator, will be featured in oral and poster presentations during the 67^th American Society of Hematology (ASH) Annual Meeting and Exposition (ASH 2025) in Orlando, Florida, December 6-9, 2025.

“This year’s presentations at ASH highlight the growing momentum of our PK activation franchise, featuring new clinical and preclinical data that reinforce the therapeutic potential of mitapivat for patients with thalassemia, sickle cell disease, and PK deficiency – debilitating and life-threatening rare blood disorders with few or no treatment options,” said Sarah Gheuens, M.D., Ph.D., Chief Medical Officer and Head of R&D, Agios. “Building on these findings, we are also sharing research led by two of our advisory councils, each comprised of patients, caregivers, advocates, and physicians, that deepens our understanding of these rare diseases and helps guide the development of critical studies and resources tailored to patient needs. ASH provides a vital platform to showcase this important body of data and strengthen our connections with the hematology and rare disease communities.”

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 with transfusion-dependent alpha- or beta-thalassemia.

• The first is a subgroup analysis of patients with alpha-thalassemia, showing that 77.8% (7 of 9) of individuals in the mitapivat arm achieved the primary endpoint of transfusion reduction response, compared to 0% (0 of 3) in the placebo arm. Additionally, reductions in transfusion burden were observed in the mitapivat arm versus none in the placebo arm for all key secondary endpoints.
• 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 (heart muscle disease) in a mouse model of beta-thalassemia, with this mechanism potentially linked to its activation of the PKM2 isoform (or variant) of the PK enzyme in the heart. Cardiomyopathy is a leading cause of morbidity and mortality in patients with hemolytic anemias.
• A poster presentation with positive findings from the ACTIVATE-Kids Phase 3 trial of mitapivat in children aged 1 to <18 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. Additionally, the mitapivat arm showed improvements in changes from baseline for hemoglobin concentration and markers of hemolysis (indirect bilirubin and lactate dehydrogenase) compared to the placebo arm. The safety results were consistent with the safety profile for mitapivat previously observed for adult patients with PK deficiency who are not regularly transfused.
• 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.

• The first is a poster from the Thalassemia Advocacy Advisory Council, which showcases a global patient survey that identified key knowledge gaps about thalassemia, including awareness of complication risks at certain hemoglobin levels and the importance of regular monitoring in non-transfusion-dependent patients.
• The second is a publication-only study from the Red Cell Revolution, which highlights interim results of a qualitative survey assessing the physical, mental, and emotional impact of fatigue across patients with thalassemia, sickle cell disease, and PK deficiency, with cognitive impairment reported as the most bothersome manifestation of fatigue.

In total, 10 presentations and publications led by Agios and external collaborators will be shared at ASH 2025.

View ASH 2025 Accepted Abstracts HERE.