Press Release Details

Agios to Present Updated Clinical Proof-of-Concept Data from the Phase 2 Study of Mitapivat in Thalassemia in Oral Presentation at the European Hematology Association Annual Congress

May 14, 2020

– Data Supporting Utility of TIBSOVO® in AML and MDS Also Accepted for Presentation –

– Agios to Host Investor Webcast on June 12 at 7:30 a.m. ET

CAMBRIDGE, Mass., May 14, 2020 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), a leader in the field of cellular metabolism to treat cancer and rare genetic diseases, today announced that mitapivat and ivosidenib clinical data will be presented at the European Hematology Association (EHA) Annual Congress being held virtually June 11-14, 2020.

The accepted abstracts are listed below and are available online on the EHA meeting library website: All presentations can be accessed on demand by registered meeting attendees on the EHA Virtual Congress platform as of Friday, June 12 at 08:30 a.m. CEST / 2:30 a.m. ET and will be accessible until October 15, 2020.

Oral Presentation:

Title: Proof of concept for the oral pyruvate kinase activator mitapivat in adults with non-transfusion-dependent thalassemia: Interim results from an ongoing, phase 2, open-label, multicenter study
Date & Time: Friday, June 12, 2020 at 8:30 a.m. CEST / 2:30 a.m. ET
Oral Abstract Session: New therapeutic approaches for thalassemia
Abstract: S297
Presenter: Kevin H. M. Kuo, M.D., Toronto General Hospital

Poster Presentations:

Title: Mitapivat (AG-348) long-term safety and efficacy in pyruvate kinase deficiency: 3-year results of the Drive PK study
Poster Session: Enzymopathies, membranopathies and other anemias
Abstract: EP1561
Author: Rachael F. Grace, M.D., Boston Children’s Hospital

Title: Ivosidenib improves overall survival relative to standard therapies in relapsed or refractory mutant IDH1 AML: Results from matched comparisons to historical controls
Poster Session: Acute myeloid leukemia - Clinical
Abstract: EP540
Author: Peter Paschka, M.D., Universitätsklinikum Ulm

Title: Ivosidenib (IVO) in patients with IDH1-mutant relapsed/refractory myelodysplastic syndrome (R/R MDS): Updated enrollment of a phase 1 dose escalation and expansion study
Poster Session: Myelodysplastic syndromes - Clinical
Abstract: EP826
Author: Courtney D. DiNardo, M.D., University of Texas MD Anderson Cancer Center

Title: Pharmacokinetic/pharmacodynamic evaluation of ivosidenib or enasidenib combined with intensive induction and consolidation chemotherapy in patients with newly diagnosed IDH1/2-mutant AML
Poster Session: Acute myeloid leukemia - Clinical
Abstract: EP620
Author: Yue Chen, Agios Pharmaceuticals

Title: Ivosidenib (IVO) prior to hematopoietic cell transplant for patients with IDH1-mutant relapsed or refractory acute myeloid leukemia (R/R AML)
Poster Session: Acute myeloid leukemia - Clinical
Abstract: EP577
Author: Courtney D. DiNardo, M.D., University of Texas MD Anderson Cancer Center

Publication Only:

Title: Agile: Phase 3, double-blind, randomized, placebo-controlled study of ivosidenib in combination with azacitidine in adults with newly diagnosed acute myeloid leukemia and an IDH1 mutation
Publication Only (in abstract book): 04. Acute myeloid leukemia - Clinical
Abstract: PB1862
Author: Pau Montesinos, M.D., Ph.D., Hospital Universitari i Politècnic La Fe

Conference Call Information
Agios will host a virtual investor event on June 12, 2020 at 7:30 a.m. ET to review the mitapivat clinical data. 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 The archived webcast will be available on the company's website beginning approximately two hours after the event.

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. For more information, please visit the company's website at

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 TIBSOVO® (ivosidenib) and mitapivat; 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 the two approved oncology precision medicines being commercialized by Agios and its collaborators 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; 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
Director, Investor Relations

Jessica Rennekamp, 857-209-3286
Associate Director, Corporate Communications

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Source: Agios Pharmaceuticals, Inc.