– 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. 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
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
Poster Session: 102. Iron Homeostasis and Biology: Poster I
Session Date and Time: Saturday, Dec. 10, 2022, 5:30-7:30 p.m. CT
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 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 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 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 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 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. 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
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
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
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
Presentation Time: Sunday, Dec. 11, 2022, at 10 a.m. CT
Title: The Pyruvate Kinase Activator Mitapivat Improves Red Blood Cell Deformability and Sickling Kinetics in Adult Patients with Sickle Cell Disease 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
Session Date and Time: Saturday, Dec. 10, 2022, 5:30-7:30 p.m. CT
Title: Activating Pyruvate Kinase Improves Red Blood Cell Integrity by Reducing Band3 Tyrosine Phosphorylation
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. 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 Session Date and Time: Saturday, Dec. 10, 2022, 5:30-7:30 p.m. CT
Agios will host a live investor event on Dec. 12, 2022, at 7:00 a.m. ET in New Orleans 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 a biopharmaceutical company that is fueled by connections. The Agios team cultivates strong bonds with patient communities, healthcare professionals, partners and colleagues to discover, develop and deliver therapies for rare and genetically defined 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 leadership in the field of cellular metabolism, 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 has multiple investigational therapies in preclinical development and an industry-leading research team with unmatched expertise in cellular metabolism and genetics. 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 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 or its collaborators 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 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; the failure of Agios to receive milestone or royalty payments related to the sale of its oncology business, the uncertainty of the timing of any receipt of any such payments, and the uncertainty of the results and effectiveness of the use of proceeds from the transaction with Servier ;competitive factors; 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; 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. Holly Manning, 617-844-6630
Jessica Rennekamp, 857-209-3286
Jessica.Rennekamp@agios.com