– 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 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.libproxy1.nus.edu.sg/meetings/annual-meeting/abstracts. 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. 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
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
Title: Improvements in Markers of Hemolysis and Liver Iron Concentration in Mitapivat-Treated Adult Patients with a Delayed Hemoglobin Response 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
Lead Author: Paola Bianchi, BSc, PhD; Hematology Unit, Pathophysiology of Anemia Unit, Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Milan, Italy 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. 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
Lead Author: Alessandro Matte, PhD; Department of Medicine, University of Verona and AOUI Verona, Verona, Italy 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
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
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 Lead Author: Sujit Sheth, MD; Joan and Sanford I Weill Medical College of Cornell University, New York, NY 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. 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
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
Presenter: Swee Lay Thein, MBBS, DSc, FRCP, FRCPath, MRCP, MRCPath; Sickle Cell Branch, National Heart, Lung & Blood Institute, NIH, Bethesda, MD 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
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, Dec. 9, 2023, 5:30-7:30 p.m. PT
Session Date and Time: Sunday, Dec. 10, 2023, 6:00-8:00 p.m. PT
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 Title: Functional and Multi-omics Signatures of Mitapivat Efficacy Upon Activation of Pyruvate Kinase in Red Blood Cells from Patients with Sickle Cell Disease Session Date and Time: Sunday, Dec. 10, 2023, 6:00-8:00 p.m. PT
Title: Longitudinal Characterization of Hemodynamic Changes with Multimodal Optical Techniques in Patients with Sickle Cell Disease Treated with Mitapivat Session Date and Time: Sunday, Dec. 10, 2023, 6:00-8:00 p.m. PT
New data from preclinical studies of Agios’ AG-946 continue to support its novel mechanism of action and PK activation as a promising potential treatment option for anemia associated with lower-risk myelodysplastic syndromes. Session Date and Time: Saturday, Dec. 9, 2023, 5:30-7:30 p.m. PT
Session Date and Time: Sunday, Dec. 10, 2023, 6:00-8:00 p.m. PT
Lead Author: Bruno Fattizzo, MD; Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, University of Milan, Milan, Italy 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. 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
Title: Setting Industry Standards for Patient Engagement, Partnership, Allyship and Care: The Patient Vision Project
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
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
Lead Author: Andreas Glenthøj, MD, PhD; Department of Haematology, Copenhagen University Hospital - Rigshospitalet, Copenhagen, Denmark
Conference Call Information
Agios will host a live investor event on Dec. 11, 2023, at 7:00 a.m. PT in San Diego to review the key clinical oral and poster presentations from this year’s ASH meeting. The event will be webcast live and can be accessed under “Events & Presentations” in the Investors and Media section of the company's website at www.agios.com. The archived webcast will be available on the company's website beginning approximately two hours after the event. Agios is the pioneering leader in PK activation and is dedicated to developing and delivering transformative therapies for patients living with rare diseases. In the U.S., Agios markets a first-in-class pyruvate kinase (PK) activator for adults with PK deficiency, the first disease-modifying therapy for this rare, lifelong, debilitating hemolytic anemia. Building on the company's deep scientific expertise in classical hematology and leadership in the field of cellular metabolism and rare hematologic diseases, Agios is advancing a robust clinical pipeline of investigational medicines with programs in alpha- and beta-thalassemia, sickle cell disease, pediatric PK deficiency and MDS-associated anemia. In addition to its clinical pipeline, Agios is advancing a preclinical TMPRSS6 siRNA as a potential treatment for polycythemia vera, and a preclinical PAH stabilizer as a potential treatment for phenylketonuria (PKU). For more information, please visit the company’s website at www.agios.com. Cautionary Note Regarding Forward-Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Such forward-looking statements include those regarding the potential benefits of PYRUKYND® (mitapivat) and AG-946; Agios’ plans regarding future data presentations; and the potential benefit of its strategic plans and focus. The words “anticipate,” “expect,” “intend,” “potential,” “milestone,” “goal,” “will,” “on track,” “upcoming,” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Such statements are subject to numerous important factors, risks and uncertainties that may cause actual events or results to differ materially from Agios' current expectations and beliefs. For example, there can be no guarantee that any product candidate Agios is developing will successfully commence or complete necessary preclinical and clinical development phases, or that development of any of Agios' product candidates will successfully continue. Moreover, there can be no guarantee that any medicines ultimately commercialized by Agios will receive commercial acceptance. There can be no guarantee that any positive developments in Agios' business will result in stock price appreciation. Management's expectations and, therefore, any forward-looking statements in this press release could also be affected by risks and uncertainties relating to a number of other important factors, including, without limitation: risks and uncertainties related to the impact of the COVID-19 pandemic or other public health emergencies to Agios’ business, operations, strategy, goals and anticipated milestones, including its ongoing and planned research activities, ability to conduct ongoing and planned clinical trials, clinical supply of current or future drug candidates, commercial supply of current or future approved products, and launching, marketing and selling current or future approved products; Agios’ results of clinical trials and preclinical studies, including subsequent analysis of existing data and new data received from ongoing and future studies; the content and timing of decisions made by the U.S. FDA, the EMA or other regulatory authorities, investigational review boards at clinical trial sites and publication review bodies; Agios’ ability to obtain and maintain requisite regulatory approvals and to enroll patients in its planned clinical trials; unplanned cash requirements and expenditures; Agios' ability to obtain, maintain and enforce patent and other intellectual property protection for any product candidates it is developing; Agios’ ability to maintain key collaborations; uncertainty regarding any milestone or royalty payments related to the sale of its oncology business or its in-licensing of TMPRSS6 siRNA, and the uncertainty of the timing of any such payments; uncertainty of the results and effectiveness of the use of proceeds from the transaction with Servier; competitive factors; and general economic and market conditions. These and other risks are described in greater detail under the caption "Risk Factors" included in Agios’ public filings with the Securities and Exchange Commission. Any forward-looking statements contained in this press release speak only as of the date hereof, and Agios expressly disclaims any obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law. Chris Taylor, VP, Investor Relations and Corporate Communications