CAMBRIDGE - Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism to treat cancer and rare genetic diseases, today reported interim data from its ongoing Phase 2 study evaluating single agent mitapivat in non-transfusion-dependent and thalassemia.

Data from the study were featured in an oral presentation at the 25th European Hematology Association Annual Congress, which is being held virtually. Mitapivat is an investigational, first-in-class, oral, small molecule allosteric activator of wild-type and a variety of mutated pyruvate kinase-R (PKR) enzymes.

'These data are exciting and further validate the potential of PKR activation as an entirely new mechanism for treating thalassemia, including -thalassemia, for which there have been few medical advancements,' said Kevin Kuo, M.D., hematologist at University Health Network, University of Toronto, and an investigator in the study. 'Findings from the study indicate that activation of wild-type PKR by mitapivat, an oral treatment option, improved hemoglobin and associated markers of hemolysis and erythropoiesis in patients with and thalassemia. In addition, the safety profile was consistent with previously published data for mitapivat.'

'We are pleased to share the impressive interim results from our clinical study of mitapivat in and thalassemia, as the data validate pre-clinical work conducted in our laboratories and with academic collaborators and demonstrate the potential for PKR activators in hemoglobinopathies such as thalassemia and sickle cell disease,' said Chris Bowden, chief medical officer at Agios. 'Our focus now is to advance the development of mitapivat for these patients as quickly and efficiently as possible. By the end of the year, we expect to finalize a robust pivotal development plan that spans both and thalassemia, as well as transfusion dependent and non-transfusion dependent patients, with a goal of initiating a pivotal program in 2021.'

About Agios

Agios is focused on discovering and developing novel investigational medicines to treat malignant hematology, solid tumors and rare genetic diseases through scientific leadership in the field of cellular metabolism. In addition to an active research and discovery pipeline across these three therapeutic areas, Agios has two approved oncology precision medicines and multiple first-in-class investigational therapies in clinical and/or preclinical development.

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; Agios' plans for the further clinical development of mitapivat and Agios' strategic plans and prospects. The words 'anticipate,' 'believe,' 'estimate,' 'expect,' 'intend,' 'may,' 'plan,' 'predict,' 'project,' 'would,' 'could,' 'potential,' 'possible,' 'hope' 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; that positive safety and efficacy findings observed in early stage clinical trials will be replicated in later stage trials; 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 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 and 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 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.

Contact:

Holly Manning

Tel: 617-844-6630

Email: Holly.Manning@agios.com

(C) 2020 Electronic News Publishing, source ENP Newswire