30th European Hematology Association Congress
(EHA 2025)
Fueled by Connections to Transform Rare Diseases
Agios EHA 2025 Data Room
Agios is presenting new data on mitapivat and tebapivat, the company’s two pyruvate kinase (PK) activators, at EHA 2025.
Select presentations and publications at EHA 2025 will include:
- An oral presentation of results from the ACTIVATE-KidsT Phase 3 study of mitapivat in children aged 1 to <18 years with PK deficiency who are regularly transfused. The study showed a clinically meaningful reduction in transfusion burden with mitapivat, with a higher proportion of patients achieving the primary endpoint of transfusion reduction response compared to placebo, though the prespecified statistical criterion was not met. Safety results were consistent with the safety profile for mitapivat previously observed in adults with PK deficiency who are regularly transfused. The ACTIVATE-KidsT study, along with the positive ACTIVATE-Kids Phase 3 trial, represents the successful execution of Agios’ first pediatric clinical program.
- An oral presentation of long-term data from the investigator-led ESTIMATE Phase 2 trial, an open-label study investigating mitapivat in patients with sickle cell disease. In the study, mitapivat showed sustained efficacy and tolerability over three years, including improvements in anemia, hemolysis, painful vaso-occlusive crises, and markers of kidney damage.
- A poster presentation with preclinical data demonstrating that tebapivat reduced red blood cell sickling and adhesion in blood samples from sickle cell disease patients, highlighting its therapeutic potential in this patient population.
- A preclinical publication examining expression patterns of PKM2, an isoform (or variant) of the PK enzyme. The study found that, compared with healthy controls, patients with myelodysplastic syndromes (MDS) have significantly reduced PKM2 expression in CD34+ hematopoietic stem cells, which may be implicated in the development of MDS. These findings further support the ongoing investigation of tebapivat in lower-risk MDS, as it activates PKM2 in addition to PKR (the PK isoform found in red blood cells).
News
May 14, 2025
Agios to Highlight Pyruvate Kinase Activation Portfolio with New Data in Rare Blood Disorders at 30th EHA Congress
Agios- and Collaborator-Led Presentations & Publications
Thalassemia
Poster Presentations:
Overall survival and morbidity among adults with thalassemia in England: A retrospective analysis using routinely collected healthcare data from 2008 to 2020
Abstract: PS2183
Presenter: Khaled M. Musallam, M.D., Ph.D., Burjeel Medical City, Abu Dhabi, United Arab Emirates
Session Date: Saturday, June 14, 2025
Session Time: 6:30 – 7:30 PM CEST
View poster >
Impact of non-transfusion-dependent thalassemia on adult patients’ health-related quality of life and work productivity: A multi-region real-world survey
Abstract: PF1192
Presenter: Khaled M. Musallam, M.D., Ph.D., Burjeel Medical City, Abu Dhabi, United Arab Emirates
Session Date: Friday, June 13, 2025
Session Time: 6:30 – 7:30 PM CEST
View poster >
ENERGIZE-T/ENERGIZE: Roxyscan assesses pyruvate kinase activator’s effect on oxidative stress sensitivity in β-thalassemia patients
Abstract: PS2192
Presenter: Eduard J. van Beers, M.D., Ph.D., University Medical Center of Utrecht, Netherlands
Session Date: Saturday, June 14, 2025
Session Time: 6:30 – 7:30 PM CEST
View poster >
Publication Only:
Understanding health literacy among patients with thalassemia: Initial key learnings from a global patient survey by the Thalassemia Advocacy Advisory Council
Abstract: PB3545
Presenter: Maria Domenica Cappellini, M.D., University of Milan, Italy
Sickle Cell Disease
Oral Presentations:
Three-year safety, efficacy, and renal outcomes of mitapivat treatment in sickle cell disease: Results from a phase 2, open-label study
Abstract: S299
Presenter: Geoffrey Kuppens, University Medical Center Utrecht, Netherlands
Session Date: Thursday, June 12, 2025
Session Time: 5:00 – 6:15 PM CEST
View presentation >
Poster Presentations:
Patient-reported vaso-occlusive events, their associated pain severity, and impact of sickle cell disease on fatigue and quality of life: A real-world survey in the United States
Abstract: PS2179
Presenter: Oladipo Cole, M.D., University of Connecticut Health Center
Session Date: Saturday, June 14, 2025
Session Time: 6:30 – 7:30 PM CEST
View poster >
Optimizing hydroxyurea therapy in sickle cell disease: Insights from metabolite detection, treatment response and clinical outcomes*
Abstract: PF1176
Presenter: Sigrid van der Veen, University Medical Center Utrecht, Netherlands
Session Date: Friday, June 13, 2025
Session Time: 6:30 – 7:30 PM CEST
Ex-vivo activation of pyruvate kinase by tebapivat reduces sickling and red blood cell adhesion in sickle cell disease
Abstract: PS2170
Presenter: Minke Rab, M.D., Ph.D., University Medical Center Utrecht, Netherlands
Session Date: Saturday, June 14, 2025
Session Time: 6:30 – 7:30 PM CEST
View poster >
PK Deficiency
Oral Presentation:
Efficacy and safety of mitapivat in pediatric patients with pyruvate kinase deficiency who are regularly transfused: Results from the phase 3 randomized global placebo-controlled ACTIVATE-KidsT trial
Abstract: S296
Presenter: Rachael F. Grace, M.D., MMSc; Dana-Farber/Boston Children’s Cancer and Blood Disorder Center, Harvard Medical School
Session Date: Thursday, June 12, 2025
Session Time: 5:00 – 6:15 PM CEST
View presentation >
Publication Only:
Cardiac magnetic resonance observations in a patient with pyruvate kinase deficiency and beta-thalassemia trait treated with mitapivat – a case report
Abstract: PB3570
Presenter: Paolo Ricchi, M.D., Ph.D., Center for Rare Red Blood Cell Diseases, AORN A. Cardarelli, Naples, Italy
Myelodysplastic Syndromes
Publication Only:
PKM and PKLR mRNA expression in CD34+ cells derived from patients with myelodysplastic syndromes
Abstract: PB2748
Presenter: Erin Tsai, M.S., Agios Pharmaceuticals
Other
Oral Presentation:
SATISFY: Mitapivat in adults with erythrocyte membranopathies and congenital dyserythropoietic anemia type II: A EuroBloodNet, multicenter, single-arm, phase 2 study
Abstract: S297
Presenter: Thomas Doeven, M.D., University Medical Center Utrecht, Netherlands
Session Date: Thursday, June 12, 2025
Session Time: 5:00 – 6:15 PM CEST
View presentation >
Poster Presentations:
Red blood cell age distribution and metabolic features in hereditary spherocytosis, hereditary xerocytosis and congenital dyserythropoietic anemia – baseline results of exploratory analysis from the SATISFY study
Abstract: PS2199
Presenter: Richard van Wijk, Ph.D., University Medical Center Utrecht, Netherlands
Session Date: Saturday, June 14, 2025
Session Time: 6:30 – 7:30 PM CEST
View poster >
PIEZO1 gain-of-function drives glycolytic imbalance in late-stage erythropoiesis: The potential of mitapivat therapy in dehydrated hereditary stomatocytosis
Abstract: PS2201
Presenter: Barbara Eleni Rosato, Ph.D., University of Naples, Italy
Session Date: Saturday, June 14, 2025
Session Time: 6:30 – 7:30 PM CEST
View poster >
*This investigator-sponsored trial is part of a larger project funded by Agios