Mitapivat

CAMBRIDGE, Mass. , July 08, 2025 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in cellular metabolism and pyruvate kinase (PK) activation pioneering therapies for rare diseases, today announced the appointment of Jay Backstrom , M.D., M.P.H., to its Board of Directors, effective immediately. Dr. Backstrom is an accomplished healthcare executive with a proven track record of advancing clinical programs across a range of therapeutic areas, including rare diseases, to regulatory approval. “We are thrilled to welcome Jay to the Agios Board of Directors,” said Brian Goff , Chief Executive Officer, Agios. “His deep scientific insight, extensive regulatory expertise, and strategic business leadership come at a pivotal time for Agios, as we work to maximize the full potential of PYRUKYND (mitapivat), our first-in-class PK activator, and advance our growing pipeline of rare disease medicines.” Dr. Backstrom brings decades of leadership experience spanning large pharmaceutical companies, mid-sized biotech firms, and early-stage biopharma organizations. Most recently, as President and Chief Executive Officer of Scholar Rock, he led a strategic pipeline review and advanced the company’s lead spinal muscular atrophy program through Phase 3 development and regulatory filings in both the U.S. and European Union . Previously, he served as Executive Vice President of Research and Development at Acceleron Pharma , which was acquired by Merck in 2021, and as Chief Medical Officer and Head of Global Regulatory Affairs at Celgene Corporation . At Celgene, Dr. Backstrom led key efforts to expand the pipeline and played a pivotal role in advancing clinical programs that led to regulatory approvals in beta thalassemia and myelodysplastic syndromes. “It is an honor to join Agios’ Board of Directors and work alongside such a talented group as the company enters its next phase of commercial growth and innovation,” said Dr. Backstrom . “Agios is laser-focused on redefining the future of rare disease treatment, driven by a maturing and expanding pipeline of promising innovative medicines. Having followed the company’s progress and collaborated with several members of the leadership team and Board in the past, I am eager to contribute to advancing its inspiring mission.” Dr. Backstrom will serve on Agios’ Science and Technology Committee . About AgiosAgios 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, myelodysplastic syndromes (MDS)-associated anemia and phenylketonuria (PKU). In addition to its clinical pipeline, Agios is advancing a preclinical TMPRSS6 siRNA as a potential treatment for polycythemia vera. 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 expected benefits of Dr. Backstrom’s service on the board of directors of Agios and the potential benefits of the company’s preclinical, clinical and commercial development plans. The words “anticipate,” “expect,” “goal,” “hope,” “milestone,” “plan,” “potential,” “possible,” “strategy,” “will,” “vision,” 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. 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 pandemics 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; 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 establish and maintain key collaborations; uncertainty regarding any royalty payments related to the sale of its oncology business or any milestone or royalty payments related to 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 Agios’ cash and cash equivalents; 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. Contacts: Investor Contact Morgan Sanford , Vice President, Investor Relations Agios Pharmaceuticals morgan.sanford@agios.com Media Contact Eamonn Nolan , Senior Director, Corporate Communications Agios Pharmaceuticals eamonn.nolan@agios.com Source: Agios Pharmaceuticals, Inc.