CAMBRIDGE, Mass., May 14, 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 that new data on the company’s PK activators, mitapivat and tebapivat, will be featured in oral and poster presentations during the 30^th European Hematology Association (EHA) Congress (EHA 2025) in Milan, Italy, June 12-15, 2025.

“The clinical results and scientific insights being presented at EHA add to the robust body of efficacy and safety data demonstrating the promise of PK activation in treating both adults and children with rare blood disorders,” said Sarah Gheuens, M.D., Ph.D., Chief Medical Officer and Head of R&D, Agios. “The presentations span serious conditions with limited or no treatment options, including sickle cell disease, thalassemia, PK deficiency, and myelodysplastic syndromes, offering meaningful results that highlight the therapeutic potential of mitapivat and tebapivat. We look forward to this important opportunity to share these new data and strengthen our collaboration with the global hematology community at EHA.”

Select presentations and publications at EHA 2025 will include:

In total, 14 presentations and publications led by Agios and external collaborators will be shared at EHA 2025.

Overall survival and morbidity among adults with thalassemia in England: A retrospective analysis using routinely collected healthcare data from 2008 to 2020	PS2183	Saturday, June 14, 2025, 6:30 - 7:30 PM CEST	Khaled M. Musallam, M.D., Ph.D., Burjeel Medical City, Abu Dhabi, United Arab Emirates	Poster
Impact of non-transfusion-dependent thalassemia on adult patients’ health-related quality of life and work productivity: A multi-region real-world survey	PF1192	Friday, June 13, 2025, 6:30 - 7:30 PM CEST	Khaled M. Musallam, M.D., Ph.D., Burjeel Medical City, Abu Dhabi, United Arab Emirates	Poster
ENERGIZE-T/ENERGIZE: Roxyscan assesses pyruvate kinase activator's effect on oxidative stress sensitivity in β-thalassemia patients	PS2192	Saturday, June 14, 2025, 6:30 - 7:30 PM CEST	Eduard J. van Beers, M.D., Ph.D., University Medical Center of Utrecht, Netherlands	Poster
Understanding health literacy among patients with thalassemia: Initial key learnings from a global patient survey by the Thalassemia Advocacy Advisory Council	PB3545	N/A	Maria Domenica Cappellini, M.D., University of Milan, Italy	Publication
Three-year safety, efficacy, and renal outcomes of mitapivat treatment in sickle cell disease: Results from a phase 2, open-label study	S299	Thursday, June 12, 2025, 5:00 - 6:15 PM CEST	Geoffrey Kuppens, University Medical Center Utrecht, Netherlands	Oral
Patient-reported vaso-occlusive events, their associated pain severity, and impact of sickle cell disease on fatigue and quality of life: A real-world survey in the United States	PS2179	Saturday, June 14, 2025, 6:30 - 7:30 PM CEST	Oladipo Cole, M.D., University of Connecticut Health Center	Poster
Optimizing hydroxyurea therapy in sickle cell disease: Insights from metabolite detection, treatment response and clinical outcomes*	PF1176	Friday, June 13, 2025, 6:30 - 7:30 PM CEST	Sigrid van der Veen, University Medical Center Utrecht, Netherlands	Poster
Ex-vivo activation of pyruvate kinase by tebapivat reduces sickling and red blood cell adhesion in sickle cell disease	PS2170	Saturday, June 14, 2025, 6:30 - 7:30 PM CEST	Minke Rab, M.D., Ph.D., University Medical Center Utrecht, Netherlands	Poster
Efficacy and safety of mitapivat in pediatric patients with pyruvate kinase deficiency who are regularly transfused: Results from the phase 3 randomized global placebo-controlled ACTIVATE-KidsT trial	S296	Thursday, June 12, 2025, 5:00 - 6:15 PM CEST	Rachael F. Grace, M.D., MMSc; Dana-Farber/Boston Children’s Cancer and Blood Disorder Center, Harvard Medical School	Oral
Cardiac magnetic resonance observations in a patient with pyruvate kinase deficiency and beta-thalassemia trait treated with mitapivat – a case report	PB3570	N/A	Paolo Ricchi, M.D., Ph.D., Center for Rare Red Blood Cell Diseases, AORN A. Cardarelli, Naples, Italy	Publication
PKM and PKLR mRNA expression in CD34+ cells derived from patients with myelodysplastic syndromes	PB2748	N/A	Erin Tsai, M.S., Agios Pharmaceuticals	Publication
SATISFY: Mitapivat in adults with erythrocyte membranopathies and congenital dyserythropoietic anemia type II: A EuroBloodNet, multicenter, single-arm, phase 2 study	S297	Thursday, June 12, 2025, 5:00 - 6:15 PM CEST	Thomas Doeven, M.D., University Medical Center Utrecht, Netherlands	Oral
Red blood cell age distribution and metabolic features in hereditary spherocytosis, hereditary xerocytosis and congenital dyserythropoietic anemia – baseline results of exploratory analysis from the SATISFY study	PS2199	Saturday, June 14, 2025, 6:30 - 7:30 PM CEST	Richard van Wijk, Ph.D., University Medical Center Utrecht, Netherlands	Poster
PIEZO1 gain-of-function drives glycolytic imbalance in late-stage erythropoiesis: The potential of mitapivat therapy in dehydrated hereditary stomatocytosis	PS2201	Saturday, June 14, 2025, 6:30 - 7:30 PM CEST	Barbara Eleni Rosato, Ph.D., University of Naples, Italy	Poster

*This investigator-sponsored trial is part of a larger project funded by Agios

Please refer to the EHA 2025 online program for full session details and data presentation listings, and visit the Agios booth (#C04) onsite.

PYRUKYND is a pyruvate kinase activator indicated for the treatment of hemolytic anemia in adults with pyruvate kinase (PK) deficiency.

Acute hemolysis with subsequent anemia has been observed following abrupt interruption or discontinuation of PYRUKYND in a dose-ranging study. Avoid abruptly discontinuing PYRUKYND. Gradually taper the dose of PYRUKYND to discontinue treatment if possible. When discontinuing treatment, monitor patients for signs of acute hemolysis and anemia including jaundice, scleral icterus, dark urine, dizziness, confusion, fatigue, or shortness of breath.

In patients with another condition treated with PYRUKYND at a higher dose than that recommended for patients with PK deficiency, liver injury has been observed. These events were characterized by a time to onset within the first 6 months of treatment with peak elevations of alanine aminotransferase of >5× upper limit of normal (ULN) with or without jaundice. All patients discontinued treatment with PYRUKYND, and these events improved upon treatment discontinuation.

Obtain liver tests prior to the initiation of PYRUKYND and monthly thereafter for the first 6 months and as clinically indicated. Interrupt PYRUKYND if clinically significant increases in liver tests are observed or alanine aminotransferase is >5x ULN. Discontinue PYRUKYND if hepatic injury due to PYRUKYND is suspected.

The most common adverse reactions including laboratory abnormalities (≥10%) in patients with PK deficiency were estrone decreased (males), increased urate, back pain, estradiol decreased (males), and arthralgia.

Avoid use of PYRUKYND in patients with moderate and severe hepatic impairment.

full Prescribing Information for PYRUKYND.

About Agios
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, 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 potential benefits of PYRUKYND^® (mitapivat) and tebapivat; Agios’ plans regarding future data presentations; and the potential benefits of Agios’ strategic plans and focus. 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. 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. 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 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
[email protected] 

Media Contact
Eamonn Nolan, Senior Director, Corporate Communications
Agios Pharmaceuticals
[email protected]