American Society of Hematology Annual Meeting 2023
Building connections, pioneering therapies.
Agios Events and Presentations
ASH 2023 Data Room
View our latest news and presentations from the ASH Annual Meeting
News
December 9, 2023
Agios Presents Positive Results from Phase 2 Portion of the RISE UP Pivotal Study in Sickle Cell Disease at 65th ASH Annual Meeting and Exposition
November 2, 2023
Agios Pharmaceuticals to Present Broad Set of Clinical and Translational Data in Rare Blood Disorders at 65th ASH Annual Meeting and Exposition
Events
December 11, 2023 | 7:00 AM PT
Join our investor event to learn more about our key clinical oral and poster presentations from this year’s ASH meeting.
Abstracts
Access All ASH Abstracts
Agios- and Collaborator-Led Presentations
Sickle Cell Disease
Presentations of safety and efficacy data from the Phase 2 portion of the Agios RISE UP study will be presented. Agios will showcase its patient-centric approach to sickle cell disease clinical trials, including the incorporation of patient insights and decision making into the clinical trial design and campaign.
Oral Presentations:
Title: A Phase 2/3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study of Mitapivat in Patients With Sickle Cell Disease: RISE UP Phase 2 Results
Presentation Time: Saturday, Dec. 9, 2023, at 4:00 p.m. PT
Oral Abstract Session: 114. Sickle Cell Disease, Sickle Cell Trait and Other Hemoglobinopathies, Excluding Thalassemias: Clinical and Epidemiological: Building on Momentum in Disease-Modifying Therapeutics for Sickle Cell Disease
Abstract: 271
Presenter: Modupe Idowu, MD; McGovern Medical School, UT Health, Houston, TX
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Title: Long-term Safety and Efficacy of Mitapivat, an Oral Pyruvate Kinase Activator, in Adults with Sickle Cell Disease: Extension of a Phase 1 Dose Escalation Study
Presentation Time: Saturday, Dec. 9, 2023, at 4:30 p.m. PT
Oral Abstract Session: 114. Sickle Cell Disease, Sickle Cell Trait and Other Hemoglobinopathies, Excluding Thalassemias: Clinical and Epidemiological: Building on Momentum in Disease-Modifying Therapeutics for Sickle Cell Disease
Abstract: 273
Presenter: Swee Lay Thein, MBBS, DSc, FRCP, FRCPath, MRCP, MRCPath; Sickle Cell Branch, National Heart, Lung & Blood Institute, NIH, Bethesda, MD
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Title: Pyruvate Kinase Thermostability Is Associated with Red Blood Cell Adhesion, Deformability and Oxygen Affinity in Patients with Sickle Cell Disease
Presentation Time: Sunday, Dec. 10, 2023, at 5:00 p.m. PT
Oral Abstract Session: 113. Sickle Cell Disease, Sickle Cell Trait and Other Hemoglobinopathies, Excluding Thalassemias: Basic and Translational: Pathophysiology of Sickle Hemoglobinopathies: From Mice to Humans
Abstract: 561
Lead Author: Marissa J.M. Traets, PhD Candidate; Department of Central Diagnostic Laboratory – Research, University Medical Center Utrecht, Utrecht University, Utrecht, The Netherlands
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Poster Presentations:
Title: One-Year Safety and Efficacy of Mitapivat in Sickle Cell Disease: Follow-Up Results of a Phase 2, Open-Label Study
Poster Session: 114. Sickle cell Disease, Sickle Cell Trait and Other Hemoglobinopathies, Excluding Thalassemias: Clinical and Epidemiological: Poster II
Session Date and Time: Sunday, Dec. 10, 2023, 6:00-8:00 p.m. PT
Abstract: 2515
Lead Author: Myrthe J. van Dijk, PhD; Division Laboratories, Pharmacy and Biomedical Genetics, CDL en Van Creveldkliniek, University Medical Center Utrecht, Utrecht University, Utrecht, Netherlands
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Title: Functional and Multi-omics Signatures of Mitapivat Efficacy Upon Activation of Pyruvate Kinase in Red Blood Cells from Patients with Sickle Cell Disease
Poster Session: 113. Sickle Cell Disease, Sickle Cell Trait and Other Hemoglobinopathies, Excluding Thalassemias: Basic and Translational: Poster II
Session Date and Time: Sunday, Dec. 10, 2023, 6:00-8:00 p.m. PT
Abstract: 2485
Lead Author: Angelo D’Alessandro, PhD; Biochemistry and Molecular Genetics, University of Colorado Anschutz Medical Campus, Aurora, CO
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Title: Longitudinal Characterization of Hemodynamic Changes with Multimodal Optical Techniques in Patients with Sickle Cell Disease Treated with Mitapivat
Poster Session: 113. Sickle Cell Disease, Sickle Cell Trait and Other Hemoglobinopathies, Excluding Thalassemias: Basic and Translational: Poster II
Session Date and Time: Sunday, Dec. 10, 2023, 6:00-8:00 p.m. PT
Abstract: 2492
Lead Author: Timothy Quang, PhD; Section on Biomedical Optics, National Institute of Child Health and Human Development, NIH, Bethesda, MD
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Publication Only:
Title: Outpatient Costs of Patients with Sickle Cell Disease With or Without Hydroxyurea at an Institution in Rio de Janeiro, Brazil
Abstract: 7325
Lead Author: Tarun Aurora, MD; Department of Global Pediatric Medicine, St Jude Children’s Research Hospital, Memphis, TN
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PK Deficiency
Presentation of long term safety and efficacy data from trials of mitapivat in adults with PK deficiency.
Poster Presentations:
Title: Mitapivat Treatment Reduces Levels of Interference in Work/School Activity for Adult Patients with Pyruvate Kinase Deficiency
Poster Session: 904. Outcomes Research – Non-Malignant Conditions: Poster I
Session Date and Time: Saturday, Dec. 9, 2023, 5:30-7:30 p.m. PT
Abstract: 2365
Lead Author: Jennifer A. Rothman, MD, Duke University Medical Center, Durham, NC
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Publication Only:
Title: Improvements in Markers of Hemolysis and Liver Iron Concentration in Mitapivat-Treated Adult Patients with a Delayed Hemoglobin Response
Abstract: 5266
Lead Author: Eduard J. van Beers, MD, PhD; Center for Benign Haematology, Thrombosis and Haemostasis, Van Creveldkliniek, University Medical Center Utrecht, Utrecht University, Utrecht, The Netherlands
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Title: Regional Genetic Heterogeneity Among Patients with Pyruvate Kinase Deficiency
Abstract: 5203
Lead Author: Paola Bianchi, BSc, PhD; Hematology Unit, Pathophysiology of Anemia Unit, Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Milan, Italy
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Thalassemia
Presentation of data for mitapivat in adults with beta-thalassemia, as well as a qualitative study of disease burden in both alpha- and beta- thalassemia.
Poster Presentations:
Title: Mitapivat Treatment Increases β-thalassemic Erythroblasts Energy Production and Responsiveness to Oxidative Stress
Poster Session: 112. Thalassemia and Globin Gene Regulation: Poster III
Session Date and Time: Monday, Dec. 11, 2023, 6:00-8:00 p.m. PT
Abstract: 3850
Lead Author: Alessandro Matte, PhD; Department of Medicine, University of Verona and AOUI Verona, Verona, Italy
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Publication Only:
Title: Association of Hemoglobin Levels With Healthcare Resource Utilization and Costs in Non–Transfusion-Dependent α- and β-Thalassemia: A Retrospective Observational Study Using Real-World Data
Abstract: 5244
Lead Author: Arielle L. Langer, MD; MPH, Division of Hematology, Brigham & Women’s Hospital, Harvard Medical School, Boston MA
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Title: Burden of Illness of Alpha- and Beta-Thalassemia: A Qualitative Study
Abstract: 7329
Lead Author: Sujit Sheth, MD; Joan and Sanford I Weill Medical College of Cornell University, New York, NY
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Myelodysplastic Syndromes
Presentation of data from preclinical studies of Agios’ AG-946 as a treatment for lower-risk myelodysplastic syndromes.
Poster Presentations:
Title: AG-946, An Activator of Pyruvate Kinase, Improves Ineffective Erythropoiesis in the Bone Marrow of Mouse Models of Myelodysplastic Syndromes
Poster Session: 636. Myelodysplastic Syndromes – Basic and Translational: Poster I
Session Date and Time: Saturday, Dec. 9, 2023, 5:30-7:30 p.m. PT
Abstract: 1854
Lead Author: Megan Wind-Rotolo, PhD, Agios Pharmaceuticals, Cambridge, MA
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Title: The Pyruvate Kinase (PK) Activator AG-946 Improves PK Properties and Red Blood Cell (RBC) Characteristics upon Ex Vivo Treatment of RBCs from Patients with Myelodysplastic Syndromes
Poster Session: 636. Myelodysplastic Syndromes—Basic and Translational: Poster II
Session Date and Time: Sunday, Dec. 10, 2023, 6:00-8:00 p.m. PT
Abstract: 3222
Lead Author: Jonathan R.A. de Wilde, PhD Candidate; Red Blood Cell Research Group, Central Diagnostic Laboratory-Research, University Medical Center Utrecht, Utrecht University, Utrecht, the Netherlands
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Publication Only:
Title: Biochemical and Metabolomic Analysis of Glycolytic Activity in Red Blood Cells (RBC) from Low-risk Myelodysplastic Syndromes (LR-MDS) Patients and In-vitro Effect of the Pyruvate Kinase (PK) Activator AG-946
Abstract: 6456
Lead Author: Bruno Fattizzo, MD; Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, University of Milan, Milan, Italy
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Other Genetically Defined Diseases
Agios’ collaborators present new data evaluating mitapivat as a potential treatment for other rare blood diseases.
Poster Presentations:
Title: Ex Vivo Treatment by Mitapivat, an Allosteric Pyruvate Kinase Activator, Reduced Oxidative Stress and Promoted Terminal Erythropoiesis in a Severe Hemolytic Anemia Patients Due to Krϋppel-like Factor 1 Mutations
Poster Session: 101. Red Cells and Erythropoiesis, Excluding Iron: Poster I
Session Date and Time: Saturday, Dec. 9, 2023, 5:30-7:30 p.m. PT
Abstract: 1071
Lead Author: Thidarat Suksangpleng, PhD.; Siriraj-Thalassemia Center, Faculty of Medicine Siriraj Hospital, Mahidol University, Bangkok, Thailand
Title: Safety and Efficacy of Mitapivat Sulfate in Adult Patients with Erythrocyte Membranopathies (SATISFY)
Poster Session: 101. Red Cells and Erythropoiesis, Excluding Iron: Poster I
Session Date and Time: Saturday, Dec. 9, 2023, 5:30-7:30 p.m. PT
Abstract: 1085
Lead Author: Andreas Glenthøj, MD, PhD; Department of Haematology, Copenhagen University Hospital – Rigshospitalet, Copenhagen, Denmark
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Patient Advocacy
Agios is fueled by connections, with patient and KOL engagement at the core of our mission. Information presented provides an overview of the formation and vision of the Red Cell Revolution – a multi-stakeholder, patient-advocacy data collection approach to understanding the unmet needs of the patients, caregivers and healthcare professionals for PK deficiency, sickle cell disease and thalassemia.
Poster Presentations:
Title: A Patient-Centric Approach to Sickle Cell Disease Clinical Trials: Integrating Patient Perspectives in the RISE UP Phase 2/3 Trial of Mitapivat for Informed Protocol Design and Associated Patient Community Benefit
Poster Session: 904. Outcomes Research – Non-Malignant Conditions: Poster I
Session Date and Time: Saturday, December 9, 2023; 5:30-7:30pm
Abstract: 2376
Lead Author: Charles Jonassaint, Blaze Eppinger, Dominique Friend, Golie Green, Mia Robinson, Teonna Woolford, DeMitrious Wyant, Janie Davis, Abdulafeez Oluyadi, Ahmar U. Zaidi, Holly John, and Wally R. Smith
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Title: Cross-community Collaboration and Data Collection to Optimize Patient Care in Hemolytic Anemias
Poster Session: 901. Health Services and Quality Improvement – Non-Malignant Conditions: Poster II
Session Date and Time: Sunday, Dec. 10, 2023, 6:00-8:00 p.m. PT
Abstract: 3692
Lead Author: Biree Andemariam, MD; New England Sickle Cell Institute, University of Connecticut Health, Farmington, CT
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Title: Understanding the Physical and Psychosocial Impacts of Pyruvate Kinase Deficiency: Patient-Led Development of the Pyruvate Kinase Deficiency Life Phase Model
Poster Session: 901. Health Services and Quality Improvement – Non-Malignant Conditions: Poster II
Session Date and Time: Sunday, December 10, 2023; 6:00 PM – 8:00 PM
Abstract: 3691
Lead Author: Rachael Grace, Lily Cannon, Maaike Eijgenraam, Carl Lander, LauraMiller-D’Angelo, Susan Morris, Janie Davis, Parija Patel, Tamara Schryver, Alejandra Watson, and Wilma Barcellini
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Publication Only:
Title: Setting Industry Standards for Patient Engagement, Partnership, Allyship and Care: The Patient Vision Project
Abstract: 7233
Lead Author: Biree Andemariam, MD; New England Sickle Cell Institute, University of Connecticut Health, Farmington, CT
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Title: Investigating Health Literacy in Thalassemia: Founding aPatient-Led Research Approach
Abstract: 5251
Lead Author: Lily Cannon, Ralph Colasanti, Jill Deitrick, Holly John,Keely Gilroy, Louise Lombard, Tobias Larkin, Laurice Levine, Eleni Michalaki, Dina Steagall, and Maria Domenica Cappellini
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Follow along with Agios at #ASH2023
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We were excited to host a Care Walk at Agios as a part of our company fall gathering! Inspired by the work of the @CooleysAnemia Foundation, we were grateful for the opportunity to raise awareness and funds to support the #thalassemia community.
It’s #HispanicHeritageMonth! We are proud to celebrate the incredible contributions of the Hispanic community through our Latinx ERG, which is focused on enhancing understanding and knowledge of LatinX cultures. Hear from Cecilia Jones of our LatinX ERG on what this month means…
Our Vice President & Head Of Medical Affairs Eva Gallagher will be participating in a panel next week at the Operationalize: Expanded Access Programs Summit in London to discuss access strategies for #raredisease treatments. Learn more about the event here:…
