On May 11, 2023 Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in the field of cellular metabolism pioneering therapies for rare diseases, reported that the company and its collaborators will present a broad range of clinical and translational data at the European Hematology Association (EHA) (Free EHA Whitepaper) Hybrid Congress, hosted virtually and in person in Frankfurt, Germany, June 8-11, 2023, and continuing virtually on the Congress platform June 14-15, 2023 (Press release, Agios Pharmaceuticals, MAY 11, 2023, View Source [SID1234631478]).
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The accepted abstracts are listed below and are available online on the EHA (Free EHA Whitepaper) meeting library website. All posters will be presented during the poster session on Friday, June 9 at 16:30-17:45 CEST. All presentations can be accessed on demand by registered meeting attendees on the EHA (Free EHA Whitepaper) Virtual Congress platform until Aug. 15, 2023.
Agios-led Abstracts:
Poster Presentations
Title: Healthcare Resource Use, Economic Burden and In-Patient Mortality in Patients with Alpha- and Beta-Thalassemia Compared to Matched Controls in the Real-World Setting
Abstract: P1463
Presenting Author: Louise Lombard, M Nutr, Agios Pharmaceuticals, Cambridge, MA, United States
Title: Clinically Relevant Hemoglobin Response in Adults with Pyruvate Kinase Deficiency Treated with Mitapivat – A Sub-Analysis of the ACTIVATE Trial
Abstract: P1473
Presenting Author: Hanny Al-Samkari, M.D., Division of Hematology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, United States
Title: The Clinical Characteristics and Overall Survival of Pyruvate Kinase Deficiency Patients in the UK: A Real-World Study
Abstract: P1476
Presenting Author: Sarah Higa, PharmD, M.S., Agios Pharmaceuticals, Cambridge, MA, United States
Title: Mitapivat Efficacy in Adults with Pyruvate Kinase Deficiency and Baseline Hemoglobin Levels >10 g/dL
Abstract: P1477
Presenting Author: Rachael F. Grace, M.D., Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, Harvard Medical School, Boston, MA, United States
Title: Comorbidities and Complications in Adults with Pyruvate Kinase Deficiency According to Hemoglobin Strata – A Descriptive Analysis from the Peak Registry
Abstract: P1479
Presenting Author: Dagmar Pospíšilová, M.D., Ph.D., Department of Pediatrics, Palacky University and University Hospital, Olomouc, Czech Republic
Title: PKM And PKR Expression During Hematopoiesis and Erythropoiesis
Abstract: P1485
Presenting Author: Erin Tsai, M.S., Agios Pharmaceuticals, Cambridge, MA, United States
Title: Mitapivat Improves Iron Overload in Patients with Pyruvate Kinase Deficiency Who Are Regularly Transfused
Abstract: P1497
Presenting Author: Eduard J. van Beers, M.D., Center for Benign Haematology, Thrombosis and Haemostasis, Van Creveldkliniek, University Medical Center Utrecht, Utrecht University, Utrecht, The Netherlands
Publication Only
Title: Estimating Utility Values for Health States in Pyruvate Kinase Deficiency
Abstract: PB2547
Lead Author: Sarah Higa, PharmD, M.S., Agios Pharmaceuticals, Cambridge, MA, United States
Collaborator-led Abstracts:
Poster Presentations
Title: AG946, A Pyruvate Kinase (PK) Activator Improves PK Properties and Red Blood Cell (RBC) Metabolism Upon Ex Vivo Treatment of RBCs from Patients with Myelodysplastic Syndromes
Abstract: P717
Presenting Author: Jonathan de Wilde, MSc, University Medical Center Utrecht, Utrecht University, Utrecht, Netherlands
Title: One-Year Follow-Up of a Phase 2 Study of Mitapivat, an Oral Pyruvate Kinase Activator, for the Treatment of Sickle Cell Disease
Abstract: P1424
Presenting Author: Myrthe J. van Dijk, Ph.D., Division Laboratories, Pharmacy and Biomedical Genetics, CDL en Van Creveldkliniek , University Medical Center Utrecht, Utrecht University, Utrecht, Netherlands
Title: Rare Anaemia Disorders European Epidemiological Platform (RADeep): Distribution of Patients Affected by RADs in Europe
Abstract: P1427
Presenting Author: Maria Del Mar Mañú Pereira, Ph.D., Vall d’Hebron University Hospital, Group of Translational Research in Cancer and Blood disorders in Children, Barcelona, Spain
Title: Mitapivat Ameliorates In Vitro Human β Thalassemic Erythroid Maturation Index and Modulates the Expression of Peroxiredoxin-2
Abstract: P1458
Presenting Author: Lucia De Franceschi, M.D., Department of Medicine, University of Verona, and Azienda Ospedaliera Universitaria Verona, Policlinico GB Rossi, Verona, Italy
Publication Only
Title: Glycolytic Activity and Effect of Ex-Vivo Treatment with the Pyruvate Kinase (PK) Activator AG-946 in Red Blood Cells (RBC) From Low-Risk Myelodysplastic Syndromes (LR-MDS) Patients: A Proof-Of-Concept Study
Abstract: PB1990
Lead Author: Bruno Fattizzo, Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, University of Milan, Milan, Italy
Title: A Pilot Study of the International Hemoglobinopathy Research Network (INHERENT)
Abstract: PB2515
Lead Author: Petros Kountouris, Ph.D., The Cyprus Institute of Neurology and Genetics, Nicosia, Cyprus