66th American Society of Hematology Annual Meeting and Exposition (ASH 2024)
Agios ASH 2024 Data Room
Agios is presenting new data on mitapivat and tebapivat (AG-946), the company’s two PK activators, at ASH 2024.
Key presentations and publications will include:
- An oral presentation on results from the Phase 3 ENERGIZE-T study evaluating mitapivat in adults with transfusion-dependent alpha- or beta-thalassemia versus placebo. Alongside the positive results from the Phase 3 ENERGIZE study of mitapivat in non-transfusion-dependent alpha- or beta-thalassemia previously presented at the 2024 European Hematology Association (EHA) Congress, these data support mitapivat’s potential as an oral, disease-modifying therapy across the full range of patients with thalassemia regardless of transfusion status.
- A poster presentation of a Phase 1 study assessing the safety, tolerability, pharmacokinetics and pharmacodynamics of tebapivat in patients with sickle cell disease, providing further evidence that PK activation may have beneficial effects in this patient population.
- A trial-in-progress publication that outlines the Phase 2b study evaluating the efficacy and safety of tebapivat in patients with anemia due to lower-risk myelodysplastic syndromes.
News
Dec 08, 2024
Agios Presents Positive Results from Phase 3 ENERGIZE-T Study of Mitapivat at ASH 2024 and Provides Regulatory Update on Mitapivat
Nov 05, 2024
Agios to Present New Data on Mitapivat and Tebapivat in Rare Blood Disorders at 66th ASH Annual Meeting and Exposition
Investor Event
Monday, December 9
7 a.m. PT
Agios will host a live and webcast investor event with the company’s leadership team and medical experts to discuss key data presented at ASH 2024. The event will take place on Monday, December 9, starting at 7:00 a.m. PT (10:00 a.m. ET).
Agios- and Collaborator-Led Presentations & Publications
Thalassemia
Oral Presentations:
ENERGIZE-T: A Global, Phase 3, Double-Blind, Randomized, Placebo-Controlled Study of Mitapivat in Adults with Transfusion-Dependent Alpha- or Beta-Thalassemia
Abstract: 409
Presenter: Maria Domenica Cappellini, M.D., University of Milan, Italy
Session Name: 112. Thalassemia and Globin Gene Regulation: We’re Going to Catch a Big One: Towards Targeted Therapies in Thalassemia
Session Date: Sunday, December 8, 2024
Session Time: 9:30 – 11:00 a.m. PT
Presentation Time: 9:30 a.m. PT
Location: San Diego Convention Center, Room 31
View presentation >
PKM2 binds to the regulatory regions of Gata-1 and STAT5 in β-thalassemic mouse erythroblasts
Abstract: 410
Presenter: Enrica Federti, Ph.D., University of Verona, Verona, Italy
Session Name: 112. Thalassemia and Globin Gene Regulation: We’re Going to Catch a Big One: Towards Targeted Therapies in Thalassemia
Session Date: Sunday, December 8, 2024
Session Time: 9:30 AM – 11:00 AM
Presentation Time: 9:45 AM
Location: San Diego Convention Center, Room 31
View presentation >
Poster Presentations:
Ex vivo treatment by mitapivat, an allosteric pyruvate kinase activator, reduced hemolysis and reactive oxygen species in red blood cells of non-regularly transfused hemolytic anemic patients with β-thalassemia/Hb E disease
Abstract: 2479
Presenter: Thidarat Suksangpleng, Ph.D., Siriraj Hospital, Mahidol University, Bangkok, Thailand
Session Name: 112. Thalassemia and Globin Gene Regulation: Poster II
Session Date: Sunday, December 8, 2024
Presentation Time: 6:00 PM – 8:00 PM
Location: San Diego Convention Center, Halls G-H
View poster >
Thalassemia scenario in Brazil: A descriptive study
Abstract: 2310
Presenter: Catherine Moura, M.D., MSc, Abrasta – Brazilian Thalassemia Association, São Paulo, SP, Brazil
Session Name: 904. Outcomes Research: Hemoglobinopathies: Poster I
Session Date: Saturday, December 7, 2024
Presentation Time: 5:30 PM – 7:30 PM
Location: San Diego Convention Center, Halls G-H
Sickle Cell Disease
Poster Presentations:
Results From A Phase 1 Study To Assess The Safety, Tolerability, Pharmacokinetics, And Pharmacodynamics Of Tebapivat (AG-946) In Patients With Sickle Cell Disease
Abstract: 2496
Presenter: Julia Xu, M.D., MScGH, Vascular Medical Institute, University of Pittsburgh, Pittsburg, PA
Session Name: 114. Sickle Cell Disease, Sickle Cell Trait, and Other Hemoglobinopathies, Excluding Thalassemias: Clinical and Epidemiological: Poster II
Session Date: Sunday, December 8, 2024
Presentation Time: 6:00 PM – 8:00 PM
Location: San Diego Convention Center, Halls G-H
View poster >
Dual Activation of PKR and PKM2 Reduced the Development of Fibrosis and Iron Deposition in a Sickle Cell Disease Nephropathy Mouse Model
Abstract: 1107
Presenter: Trang Nguyen, ScB, Agios Pharmaceuticals, Inc., Cambridge, MA
Session Name: 113. Sickle Cell Disease, Sickle Cell Trait, and Other Hemoglobinopathies, Excluding Thalassemias: Basic and Translational: Poster I
Session Date: Saturday, December 7, 2024
Presentation Time: 5:30 PM – 7:30 PM
Location: San Diego Convention Center, Halls G-H
View poster >
Mitapivat-Induced Improvements in RBC Deformability and Membrane Integrity in Patients with Sickle Cell Disease are Sustained During Extended Therapy
Abstract: 2491
Presenter: Xunde Wang, Ph.D., National Institute of Health, Bethesda, MD
Session Name: 113. Sickle Cell Disease, Sickle Cell Trait, and Other Hemoglobinopathies, Excluding Thalassemias: Basic and Translational: Poster II
Session Date: Sunday, December 8, 2024
Presentation Time: 6:00 PM – 8:00 PM
Location: San Diego Convention Center, Halls G-H
View poster >
Myelodysplastic Syndromes
Publication Only:
A Phase 2B, Open-Label Multicenter Study of Tebapivat (AG-946), a Potent Pyruvate Kinase Activator, in Patients with Anemia due to Lower-Risk Myelodysplastic Syndromes
Published in: Blood Nov. 2024 supplementary issue
Lead Author: Amer M Zeidan, Yale University School of Medicine and Yale Comprehensive Cancer Center, New Haven, CT
PK Deficiency
Poster Presentation:
Clinical Monitoring Practices Among Adult Patients with Pyruvate Kinase Deficiency Who Have Never Been Transfused
Abstract: 3696
Presenter: Stefan W. Eber, M.D., Ph.D., M1 Private Clinic Munich, Munich, Germany
Session Name: 905. Outcomes Research: Non-Malignant Conditions Excluding Hemoglobinopathies: Poster II
Session Date: Sunday, December 8, 2024
Presentation Time: 6:00 PM – 8:00 PM
Location: San Diego Convention Center, Halls G-H
View poster >
Other
Oral Presentation:
uRADAR: European Patients Referral Frame to Improve Access to New Drugs and Therapies in Ultra-Rare Anemia Disorders and Severe Hereditary Spherocytosis
Abstract: 794
Presenter: Mar Manu Pereira, Ph.D., Vall d’Hebron Barcelona Hospital, Barcelona, Spain
Session Name: 905. Outcomes Research: Non-Malignant Conditions Excluding Hemoglobinopathies: Innovative Approaches to Improve Care for Understudied Non-Malignant Hematologic Diseases
Session Date: Monday, December 9, 2024
Session Time: 10:30 AM – 12:00 PM
Presentation Time: 10:45 AM
Location: San Diego Convention Center, Room 6B
View presentation >
Poster Presentations:
PIEZO1 gain-of-function variants lead to alterations in late-stage erythropoiesis by enhancing enucleation rate
Abstract: 3837
Presenter: Barbara Eleni Rosato, Ph.D., University of Naples Federico II, Naples, Italy
Session Name: 101. Red Cells and Erythropoiesis, Excluding Iron: Poster III
Session Date: Monday, December 9, 2024
Presentation Time: 6:00 PM – 8:00 PM
Location: San Diego Convention Center, Halls G-H
View poster >
A Multicenter, Single-Arm Phase 2 Trial of Mitapivat in Adult Patients with Erythrocyte Membranopathies and Congenital Dyserythropoietic Anemia Type II – Results from the 8-Week Dose-Escalation Period
Abstract: 3831
Presenter: Thomas Doeven, M.D., Center for Benign Hematology, Thrombosis and Hemostasis – Van Creveldkliniek, University Medical Center Utrecht, Utrecht University, Utrecht, Netherlands
Session Name: 101. Red Cells and Erythropoiesis, Excluding Iron: Poster III
Session Date: Monday, December 9, 2024
Presentation Time: 6:00 PM – 8:00 PM
Location: San Diego Convention Center, Halls G-H
View poster >
Follow along with Agios at #ASH24
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