American Society of Hematology Annual Meeting 2022
Building connections, pioneering therapies.
Agios Events and Presentations
ASH 2022 Data Room
View our latest news and presentations from the ASH Annual Meeting
News
December 11, 2022
Agios Presents Updated PYRUKYND® (mitapivat) Long-term Extension Data Demonstrating Sustained Clinical Benefits in Adults with Pyruvate Kinase (PK) Deficiency at 64th ASH Annual Meeting and Exposition
December 10, 2022
Agios Presents Updated PYRUKYND® (mitapivat) Data Highlighting Long-term Safety Profile and Durable Improvement in Hemoglobin and Markers of Hemolysis and Ineffective Erythropoiesis in Non-transfusion-dependent α- and β-Thalassemia at 64th ASH Annual Meet
November 3, 2022
Agios Pharmaceuticals to Present Broad Set of Clinical and Translational Data in Chronic Hemolytic Anemias at 64th ASH Annual Meeting and Exposition
Events
December 12, 2022 | 7:00 AM ET
Join our investor event to learn more about our key clinical oral and poster presentations from this year’s ASH meeting.
Abstracts
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Agios- and Collaborator-Led Presentations
PK Deficiency
Data to be presented highlight that long-term treatment with PYRUKYND® in adults with PK deficiency is associated with sustained clinical benefits, including improvements in hemoglobin, iron overload, transfusion burden and patient-reported outcomes. In addition, Agios is presenting data supporting its pediatric PK deficiency program, including characterization of disease complications and co-morbidities in pediatric patients.
Oral Presentations:
Title: Long-term Improvements in Patient-reported Outcomes in Patients with Pyruvate Kinase Deficiency Treated with Mitapivat
Presentation Time: Sunday, Dec. 11, 2022, at 9:45 a.m. CT
Oral Abstract Session: 904. Outcomes Research—Non-malignant Conditions: Classical Hematology: From Horses to Zebras
Abstract: 506
Presenter: Kevin H. M. Kuo, M.D., Division of Hematology, University of Toronto, Toronto, Canada
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Poster Presentations:
Title: Next Generation Sequencing for the Diagnosis of Hereditary Hemolytic Anemias Including Pyruvate Kinase Deficiency: Report from a No-cost Diagnostic Program
Poster Session: 101. Red Cells and Erythropoiesis, Excluding Iron: Poster I
Session Date and Time: Saturday, Dec. 10, 2022, 5:30-7:30 p.m. CT
Abstract: 1010
Lead Author: Jorune Balciuniene, PerkinElmer Genomics
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Title: Mitapivat Improves Iron Overload in Patients with Pyruvate Kinase Deficiency
Poster Session: 102. Iron Homeostasis and Biology: Poster I
Session Date and Time: Saturday, Dec. 10, 2022, 5:30-7:30 p.m. CT
Abstract: 1021
Lead Author: Kevin H. M. Kuo, M.D., Division of Hematology, University of Toronto, Toronto, Canada
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Title: Long-term Hemoglobin Response and Reduction in Transfusion Burden Are Maintained in Patients with Pyruvate Kinase Deficiency Treated with Mitapivat
Poster Session: 101. Red Cells and Erythropoiesis, Excluding Iron: Poster II
Session Date and Time: Sunday, Dec. 11, 2022, 6-8 p.m. CT
Abstract: 2328
Lead Author: Rachael F. Grace, M.D., MMSc, Dana-Farber/Boston Children’s Cancer and Blood Disorders Center
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Title: Comorbidities and Complications in Pediatric Patients with Pyruvate Kinase Deficiency Enrolled in the Peak Registry / Peak Pediatric Comorbidities
Poster Session: 101. Red Cells and Erythropoiesis, Excluding Iron: Poster II
Session Date and Time: Sunday, Dec. 11, 2022, 6-8 p.m. CT
Abstract: 2329
Lead Author: Rachael F. Grace, M.D., MMSc, Dana-Farber/Boston Children’s Cancer and Blood Disorders Center
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Title: ACTIVATE-KidsT: Mitapivat in Children with Pyruvate Kinase Deficiency Who Are Regularly Transfused
Poster Session: 101. Red Cells and Erythropoiesis, Excluding Iron: Poster II
Session Date and Time: Sunday, Dec. 11, 2022, 6-8 p.m. CT
Abstract: 2330
Lead Author: Rachael F. Grace, M.D., MMSc, Dana-Farber/Boston Children’s Cancer and Blood Disorders Center
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Title: Age of Onset of Complications in Patients with Pyruvate Kinase Deficiency: Analysis from the Peak Registry
Poster Session: 101. Red Cells and Erythropoiesis, Excluding Iron: Poster II
Session Date and Time: Sunday, Dec. 11, 2022, 6-8 p.m. CT
Abstract: 2332
Lead Author: Andreas Glenthøj, M.D., Ph.D., Department of Haematology, Copenhagen University Hospital – Rigshospitalet, Copenhagen, Denmark
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Title: ACTIVATE-Kids: Mitapivat in Children with Pyruvate Kinase Deficiency Who Are Not Regularly Transfused
Poster Session: 101. Red Cells and Erythropoiesis, Excluding Iron: Poster II
Session Date and Time: Sunday, Dec. 11, 2022, 6-8 p.m. CT
Abstract: 2335
Lead Author: Rachael F. Grace, M.D., MMSc, Dana-Farber/Boston Children’s Cancer and Blood Disorders Center
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Title: Mitapivat Improves Markers of Hemolysis and Erythropoiesis in Patients with Pyruvate Kinase Deficiency Irrespective of Hemoglobin Response
Poster Session: 101. Red Cells and Erythropoiesis, Excluding Iron: Poster III
Session Date and Time: Monday, Dec. 12, 2022, 6-8 p.m. CT
Abstract: 3644
Lead Author: Hanny Al-Samkari, M.D., Division of Hematology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, United States
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Publication Only:
Title: The Launch of Two Sub-studies of the Peak Registry, A Global, Longitudinal Study of Pyruvate Kinase Deficiency
Abstract: 4960
Lead Author: Carl Lander, Thrive with Pyruvate Kinase Deficiency Patient Organization
Thalassemia
Long-term Phase 2 data for PYRUKYND® in adults with alpha- or beta-thalassemia treated for up to 72 weeks show sustained hemoglobin response and improvements in hemolysis and ineffective erythropoiesis. Additional data underscore the significant disease burden across both alpha- and beta-thalassemia.
Poster Presentations:
Title: Mitapivat Improves Markers of Erythropoietic Activity in Long-term Study of Adults with Alpha- or Beta-non-transfusion-dependent Thalassemia
Poster Session: 112. Thalassemia and Globin Gene Regulation: Poster I
Session Date and Time: Saturday, Dec. 10, 2022, 5:30-7:30 p.m. CT
Abstract: 1030
Lead Author: Kevin H. M. Kuo, M.D., Division of Hematology, University of Toronto, Toronto, Canada
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Title: Characterizing the Clinical, Health-related Quality of Life and Economic Burden of Alpha-thalassemia: A Systematic Literature Review and Evidence Gaps Assessment
Poster Session: 112. Thalassemia and Globin Gene Regulation: Poster I
Session Date and Time: Saturday, Dec. 10, 2022, 5:30-7:30 p.m. CT
Abstract: 1036
Lead Author: Khaled M. Musallam, M.D., Ph.D., Thalassemia Center, Burjeel Medical City, Abu Dhabi, United Arab Emirates
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Title: Clinical Burden of Alpha- and Beta-thalassemia Compared to Matched Controls in the Real-world Setting
Poster Session: 112. Thalassemia and Globin Gene Regulation: Poster II
Session Date and Time: Sunday, Dec. 11, 2022, 6-8 p.m. CT
Abstract: 2351
Lead Author: Arielle L. Langer, M.D., MPH, Division of Hematology, Brigham & Women’s Hospital
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Sickle Cell Disease
Data to be presented continue to highlight that PK activation may be a promising therapeutic approach for patients with sickle cell disease.
Oral Presentations:
Title: Effects of Pyruvate Kinase Activators on Red Blood Cell Rheology, Sickling and Senescence in Sickle Cell Disease
Presentation Time: Saturday, Dec. 10, 2022, at 9:45 a.m. CT
Oral Abstract Session: 114. Hemoglobinopathies, Excluding Thalassemia: Clinical and Epidemiological: Novel Therapies
Abstract: 8
Presenter: Philippe Joly, Ph.D., Laboratory of Biochemistry and Molecular Biology, UF Biochemistry of Red Blood Cell Diseases, Est Center of Biology and Pathology, Hospices Civils de Lyon, Lyon France
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Title: Untargeted Metabolomics in Dried Blood Spots of Patients with Sickle Cell Disease Treated with the Pyruvate Kinase Activator Mitapivat
Presentation Time: Saturday, Dec. 10, 2022, at 10 a.m. CT
Oral Abstract Session: 114. Hemoglobinopathies, Excluding Thalassemia: Clinical and Epidemiological: Novel Therapies
Abstract: 9
Presenter: Myrthe J. van Dijk, Van Creveldkliniek, University Medical Center Utrecht, Utrecht University, Utrecht, The Netherlands
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Title: AG-946 Normalizes Glycolysis and Improves Red Cell Indices in a Humanized Sickle Cell Mouse Model
Presentation Time: Sunday, Dec. 11, 2022, at 9:30 a.m. CT
Oral Abstract Session: 113. Hemoglobinopathies, Excluding Thalassemia: Basic and Translational: Targeting the Red Blood Cell: Novel Therapeutic Approaches in Sickle Cell Disease
Abstract: 391
Lead Author: Rohitash Jamwal, Ph.D., Agios Pharmaceuticals
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Title: PKLR Variants Associated with Acute Pain in Sickle Cell Disease Influence ATP Concentrations in Red Blood Cells
Presentation Time: Sunday, Dec. 11, 2022, at 10 a.m. CT
Oral Abstract Session: 113. Hemoglobinopathies, Excluding Thalassemia: Basic and Translational: Targeting the Red Blood Cell: Novel Therapeutic Approaches in Sickle Cell Disease
Abstract: 393
Presenter: Xunde Wang, Ph.D., Sickle Cell Branch, National Heart Lung and Blood Institute, National Institutes of Health
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Poster Presentations:
Title: The Pyruvate Kinase Activator Mitapivat Improves Red Blood Cell Deformability and Sickling Kinetics in Adult Patients with Sickle Cell Disease
Poster Session: 113. Hemoglobinopathies, Excluding Thalassemia: Basic and Translational: Poster I
Session Date and Time: Saturday, Dec. 10, 2022, 5:30-7:30 p.m. CT
Abstract: 1044
Lead Author: Maureen Lundt, NHLBI / NIH, Bethesda, Maryland, United States
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Title: Feasibility of Near-infrared Spectroscopy for Monitoring Hemodynamic Changes in Sickle Cell Disease Patients Treated with Mitapivat
Poster Session: 113. Hemoglobinopathies, Excluding Thalassemia: Basic and Translational: Poster I
Session Date and Time: Saturday, Dec. 10, 2022, 5:30-7:30 p.m. CT
Abstract: 1053
Lead Author: Timothy Quang, National Institute of Child Health and Human Development
Title: Activating Pyruvate Kinase Improves Red Blood Cell Integrity by Reducing Band3 Tyrosine Phosphorylation
Poster Session: 113. Hemoglobinopathies, Excluding Thalassemia: Basic and Translational: Poster II
Session Date and Time: Sunday, Dec. 11, 2022, 6-8 p.m. CT
Abstract: 2367
Lead Author: Kang Le, Ph.D., National Institutes of Health
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AG-946
New clinical data from the Phase 1 healthy volunteers study of Agios’ novel PK activator, AG-946, support the initiation of a Phase 2a/2b study in lower-risk myelodysplastic syndromes.
Poster Presentations:
Title: A Phase 2a/2b Multicenter Study of AG-946 in Patients with Anemia Due to Lower-risk Myelodysplastic Syndromes
Poster Session: 637. Myelodysplastic Syndromes – Clinical and Epidemiological: Poster I
Session Date and Time: Saturday, Dec. 10, 2022, 5:30-7:30 p.m. CT
Abstract: 1773
Lead Author: Hanny Al-Samkari, M.D., Division of Hematology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, United States
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Title: Results from the Single and Multiple Ascending Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-946 in Healthy Volunteers
Poster Session: 114. Hemoglobinopathies, Excluding Thalassemia: Clinical and Epidemiological: Poster II
Session Date and Time: Sunday, Dec. 11, 2022, 6:00-8:00 p.m. CT
Abstract: 2383
Lead Author: Xiaoshu Dai, Ph.D., Agios Pharmaceuticals
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