Full Press Release Details
– Long-term Data for PYRUKYND® (mitapivat) in Adults with Pyruvate Kinase (PK) Deficiency and Adults with Thalassemia Demonstrate Sustained Improvement in Hemoglobin, Markers of Hemolysis and Other Disease Impacts –
– New Data from Single Ascending Dose (SAD) and Multiple Ascending Dose (MAD) Cohorts of Phase 1 Study of AG-946 in Healthy Volunteers Support Profile as Potent PK Activator and Initiation of Phase 2 Study in Myelodysplastic Syndromes (MDS)-associated Anemia –
– Agios to Host Live and Webcast Investor Event on Dec. 12, 2022, at 7 a.m. ET –
CAMBRIDGE, Mass., Nov. 03, 2022 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism to treat rare and genetically defined diseases, today announced that a broad set of clinical and translational data from its programs in hemolytic anemias, including PK deficiency, thalassemia and sickle cell disease, will be presented at the 64th American Society of Hematology (ASH) Annual Meeting & Exposition, to be held Dec. 10-13, 2022, in New Orleans.
In total, 22 abstracts led by Agios and external collaborators will be presented. The accepted abstracts are listed below and are available online on the ASH conference website at https://www.hematology.org/meetings/annual-meeting/abstracts.
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.
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
Presenter: Kevin H. M. Kuo, M.D., Division of Hematology, University of Toronto, Toronto, Canada
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
Lead Author: Jorune Balciuniene, PerkinElmer Genomics
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
Lead Author: Kevin H. M. Kuo, M.D., Division of Hematology, University of Toronto, Toronto, Canada
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
Lead Author: Rachael F. Grace, M.D., MMSc, Dana-Farber/Boston Children's Cancer and Blood Disorders Center
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
Lead Author: Rachael F. Grace, M.D., MMSc, Dana-Farber/Boston Children's Cancer and Blood Disorders Center
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
Lead Author: Rachael F. Grace, M.D., MMSc, Dana-Farber/Boston Children's Cancer and Blood Disorders Center
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
Lead Author: Andreas Glenthøj, M.D., Ph.D., Department of Haematology, Copenhagen University Hospital - Rigshospitalet, Copenhagen, Denmark
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
Lead Author: Rachael F. Grace, M.D., MMSc, Dana-Farber/Boston Children's Cancer and Blood Disorders Center
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
Lead Author: Hanny Al-Samkari, M.D., Division of Hematology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, United States
Title: The Launch of Two Sub-studies of the Peak Registry, A Global, Longitudinal Study of Pyruvate Kinase Deficiency
Lead Author: Carl Lander, Thrive with Pyruvate Kinase Deficiency Patient Organization
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
Lead Author: Kevin H. M. Kuo, M.D., Division of Hematology, University of Toronto, Toronto, Canada
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
Lead Author: Khaled M. Musallam, M.D., Ph.D., Thalassemia Center, Burjeel Medical City, Abu Dhabi, United Arab Emirates
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
Lead Author: Arielle L. Langer, M.D., MPH, Division of Hematology, Brigham & Women’s Hospital
Data to be presented continue to highlight that PK activation may be a promising therapeutic approach for patients with sickle cell disease.
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
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
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
Presenter: Myrthe J. van Dijk, Van Creveldkliniek, University Medical Center Utrecht, Utrecht University, Utrecht, The Netherlands
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
Lead Author: Rohitash Jamwal, Ph.D., Agios Pharmaceuticals
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
Presenter: Xunde Wang, Ph.D., Sickle Cell Branch, National Heart Lung and Blood Institute, National Institutes of Health
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
Lead Author: Maureen Lundt, NHLBI / NIH, Bethesda, Maryland, United States
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
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
Lead Author: Kang Le, Ph.D., National Institutes of Health
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
Lead Author: Hanny Al-Samkari, M.D., Division of Hematology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, United States
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: Saturday, Dec. 10, 2022, 5:30-7:30 p.m. CT
Lead Author: Xiaoshu Dai, Ph.D., Agios Pharmaceuticals
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