67th American Society of Hematology Annual Meeting (ASH 2025)
Fueled by Connections to Transform Rare Diseases™
Agios ASH 2025 Data Room
Agios is presenting new preclinical and clinical data on mitapivat, the company’s first-in-class pyruvate kinase (PK) activator, along with research from two Agios-supported advisory councils at ASH 2025.
Select presentations and publications at ASH 2025 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.
News
Nov 03, 2025
Agios to Present New Mitapivat Data in Rare Blood Disorders at 67th ASH Annual Meeting and Exposition
Agios- and Collaborator-Led Presentations & Publications
Thalassemia
Poster Presentations:
Efficacy of Mitapivat in Patients with Transfusion-Dependent Alpha-Thalassemia: Subgroup Analysis from the ENERGIZE-T Trial
Abstract: 4699
Presenter: Ashutosh Lal, M.D., MBBS, University of California San Francisco Benioff Children’s Hospital Oakland
Session Date: Monday, December 8, 2025
Presentation Time: 6:00 – 8:00 p.m. EST
View presentation >
Long-Term Transfusion-Free Duration and Impact on Transfusion-Related Burdens: Results from the Ongoing ENERGIZE-T Open-Label Extension Study of Mitapivat in Transfusion-Dependent Alpha- or Beta-Thalassemia
Abstract: 4697
Presenter: Sujit Sheth, M.D., Weill Cornell Medicine
Session Date: Monday, December 8, 2025
Presentation Time: 6:00 – 8:00 p.m. EST
View poster >
Ex Vivo Treatment by Mitapivat, an Allosteric Pyruvate Kinase Activator, Reduced Oxidative Stress to Support Terminal Erythropoiesis of Non-Transfusion Dependent Thalassemia Patients Due to β-Thalassemia/Hb E Disease
Abstract: 2916
Presenter: Thidarat Suksangpleng, Ph.D., Siriraj Hospital, Siriraj-Thalassemia Center, Mahidol University, Bangkok, Thailand
Session Date: Sunday, December 7, 2025
Presentation Time: 6:00 – 8:00 p.m. EST
View poster >
Oral Presentations:
Long-Term Mitapivat Treatment Improves Inflammatory Pro-Fibrotic Cardiomyopathy in a Murine Model of β-Thalassemia
Abstract: 727
Presenter: Enrica Federti, Ph.D., University of Verona, Italy
Session Date: Monday, December 8, 2025
Presentation Time: 10:30 – 10:45 a.m. EST
Sickle Cell Disease
Poster Presentations:
Mitapivat Improves Red Blood Cell Integrity by Reducing Membrane Ubiquitination Accumulation
Abstract: 1146
Presenter: Kang Le, Ph.D., National Heart, Lung, and Blood Institute, National Institutes of Health
Session Date: Saturday, December 6, 2025
Presentation Time: 5:30 – 7:30 p.m. EST
View poster >
Pyruvate Kinase Deficiency
Poster Presentations:
Efficacy and Safety of Mitapivat in Pediatric Patients with Pyruvate Kinase Deficiency Who Are Not Regularly Transfused: Results from the Phase 3, Global, Randomized, Double-Blind, Placebo-Controlled ACTIVATE-Kids Trial
Abstract: 4654
Presenter: Satheesh Chonat, M.D., Emory University School of Medicine and Aflac Cancer and Blood Disorders Center, Children’s Healthcare of Atlanta
Session Date: Monday, December 8, 2025
Presentation Time: 6:00 – 8:00 p.m. EST
View poster >
Disease Monitoring and Management Among Pediatric Patients with Pyruvate Kinase Deficiency: Real-World Practices from Pyruvate Kinase Deficiency Registries Prior to 2024 International Expert Guidelines
Abstract: 4454
Presenter: Sule Unal, M.D., Hacettepe University, Ankara, Turkey
Session Date: Sunday, December 7, 2025
Presentation Time: 6:00 – 8:00 p.m. EST
View poster >
Other
Poster Presentations:
Understanding Health Literacy Among Patients with Thalassemia: Results from a Global Patient Survey by the Thalassemia Advocacy Advisory Council
Abstract: 6421
Presenter: Sujit Sheth, M.D., Weill Cornell Medical College
Session Date: Monday, December 8, 2025
Presentation Time: 6:00 – 8:00 p.m. EST
View poster >
Activation of Pyruvate Kinase by Mitapivat Potentially Rescues Ineffective Erythropoiesis in Models of Diamond Blackfan Anemia
Abstract: 1121
Presenter: Jonathan de Wilde, M.D., Feinstein Institutes for Medical Research, Northwell Health
Session Date: Saturday, December 6, 2025
Presentation Time: 5:30 – 7:30 p.m. EST
View poster >
Publication Only:
Qualitative Interviews Exploring the Patient Experience of Fatigue in Individuals with Sickle Cell Disease (SCD), Thalassemia, and Pyruvate Kinase (PK) Deficiency
Abstract: 7971
Corresponding Author: Biree Andemariam, M.D., University of Connecticut Health