AGIO - Agios Pharmaceuticals to Present Broad Set of Clinical and Translational Data in Rare Blood Disorders at 65th ASH Annual Meeting and Exposition | Benzinga
– Both Mitapivat Dose Arms Achieved Statistically Significant Hemoglobin Response in Phase 2 Portion of RISE UP Pivotal Study in Sickle Cell Disease, Compared to the Placebo Arm; Data Support Advancement to Phase 3 Portion of RISE UP –
– Agios to Highlight Additional Data on the Burden of Disease in Pyruvate Kinase (PK) Deficiency and Alpha- and Beta-Thalassemia –
– New Preclinical Data for Novel PK Activator AG-946 Suggest Improvements in Ineffective Erythropoiesis in Model of Myelodysplastic Syndromes (MDS) –
– Agios to Host Live and Webcast Investor Event on Dec. 11, 2023, at 7:00 a.m. Pacific Time –
CAMBRIDGE, Mass., Nov. 02, 2023 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), a leader in the field of cellular metabolism pioneering therapies for rare diseases, today announced that a broad set of clinical and translational data from its programs will be presented at the 65th American Society of Hematology (ASH) Annual Meeting & Exposition, to be held Dec. 9-12, 2023, in San Diego. The presentations will focus on rare blood disorders, including PK deficiency, thalassemia, sickle cell disease and anemia associated with lower-risk myelodysplastic syndromes (LR-MDS).
In total, 23 abstracts led by Agios and external collaborators will be presented or published. The accepted abstracts are listed below and are available online on the ASH conference website at https://www.hematology.org/meetings/annual-meeting/abstracts.
PK Deficiency
Data to be presented highlight that long-term treatment with PYRUKYND® (mitapivat) in adults with PK deficiency is associated with sustained clinical benefits, including improvements in hemoglobin, iron overload, and decreased burden of disease on work and school activities.
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
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, Dec. 10, 2023, 6:00-8:00 p.m. PT
Abstract: 3691
Lead Author: Rachael F. Grace, MD, MMSc, Dana-Farber/Boston Children's Cancer and Blood Disorder Center, Harvard Medical School, Boston, MA
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
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
Thalassemia
Data for PYRUKYND® in adults with beta-thalassemia show evidence of reduced oxidative stress in red blood cells, as well as enhanced PK activity and metabolic reprogramming. Additionally, qualitative data demonstrate the equally common burden of disease across alpha- and beta-thalassemia patients, regardless of transfusion dependency.
Poster Presentations:
Title: Mitapivat Treatment Increases ?-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
Publication Only:
Title: Association of Hemoglobin Levels With Healthcare Resource Utilization and Costs in Non–Transfusion-Dependent ?- and ?-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
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
Title: Investigating Health Literacy in Thalassemia: Founding a Patient-Led Research Approach
Abstract: 5251
Lead Author: Sujit Sheth, MD; Joan and Sanford I Weill Medical College of Cornell University, New York, NY
Sickle Cell Disease
Results from the Phase 2 portion of Agios' RISE UP study will highlight improvements in hemoglobin response rates, markers of hemolysis and erythropoiesis and reductions in annualized rates of pain crises for mitapivat compared to placebo, supporting advancement into the Phase 3 portion of RISE UP. Agios will showcase its patient-centric approach to sickle cell disease clinic 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