Press Release Details

Initial Data to be Presented from AG-348 Phase 2 DRIVE PK Study in PK Deficiency and AG-519 Phase 1 Healthy Volunteer Study 

Company to Host Conference Call and Webcast on Saturday, June 11, 2016

CAMBRIDGE, Mass., May 19, 2016 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), a leader in the fields of cancer metabolism and rare genetic metabolic disorders, today announced that new clinical and preclinical data for AG-348 and AG-519 will be presented at the 21^stCongress of the European Hematology Association (EHA) taking place June 9-12, 2016 in Copenhagen. 

In total, five abstracts led by Agios and collaborators describing new clinical and preclinical data from the company's rare genetic metabolic disorders program, including three oral presentations, have been accepted for presentation at EHA. AG-348 is in an ongoing Phase 2 study (DRIVE PK) in adults with pyruvate kinase (PK) deficiency. AG-519 is in an ongoing Phase 1 study in healthy volunteers. 

The accepted abstracts are listed below and are available online on the EHA conference website: http://learningcenter.ehaweb.org/eha/#!*menu=16*browseby=2*sortby=1*media=3*label=9759

Oral presentations by Agios and/or its collaborators:

Date & Time: Friday, June 10, 2016 at 12:15 p.m. CET
Title: The pyruvate kinase activator AG-348 improves murine β-thalassemic anemia and corrects ineffective erythropoiesis
Session: Red Blood Cells and Iron - Biology
Abstract Code: S135
Location: Hall C15
Presenter: Lucia De Franceschi, M.D., University of Verona-AOUI Verona

Date & Time: Saturday, June 11, 2016 at 12:30 p.m. CET
Title: Effects of AG-348, a pyruvate kinase activator, on anemia and hemolysis in patients with pyruvate kinase deficiency: early data from the DRIVE PK study
Session: Red Blood Cells and Iron - Clinical  
Abstract Code: S466
Location: Hall C14
Presenter: Rachael Grace, M.D., Dana-Farber Boston Children's Cancer and Blood Disorder Center

Date & Time: Sunday, June 12, 2016 at 8:30 a.m. CET
Title: Preclinical pharmacokinetics and pharmacodynamics of AG-519, an allosteric pyruvate kinase-R activator
Session: Non-malignant Hematopoietic Disorders
Abstract Code: S830
Location: Room H5
Presenter: Hua Yang, Ph.D., Agios 

Poster presentations by Agios: 

Date & Time: Saturday, June 11, 2016 at 5:30 p.m. CET
Title: A phase 1, single and multiple ascending dose study of safety, tolerability, pharmacokinetics and pharmacodynamics of AG-519, an allosteric activator of pyruvate kinase-R in healthy subjects
Session: Red Blood Cells and Iron - Clinical 
Abstract Code: P752
Location: Poster area (Hall H)
Presenter: Ann Barbier, M.D., Ph.D., Agios

Title: AG-519 is a potent activator of mutant pyruvate kinase associated with hemolytic anemia
Abstract Code: P742
Session: Red Blood Cells and Iron 

• Poster Pitch Presentation Date & Time: Friday, June 10, 2016 at 12:00 p.m. CET
• Location: Hall C15
• Presenter: Charles Kung D., Agios 

• Poster Walk Date & Time: Saturday, June 11, 2016 at 5:30 p.m. CET
• Location: Poster area (Hall H)
• Presenter: Jeffrey Hixon, Agios

Conference Call Information
Agios will host a conference call and webcast from the congress to review the data on Saturday, June 11, 2016 beginning at 9:30 a.m. ET (3:30 p.m. CET). Conference call dial-in information will be provided in a separate release prior to the meeting. 

About Agios
Agios is focused on discovering and developing novel investigational medicines to treat cancer and rare genetic metabolic disorders through scientific leadership in the field of cellular metabolism. In addition to an active research and discovery pipeline across both therapeutic areas, Agios has multiple first-in-class investigational medicines in clinical and/or preclinical development. All Agios programs focus on genetically identified patient populations, leveraging our knowledge of metabolism, biology and genomics. 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 Agios' product candidates targeting pyruvate kinase-R mutations, including AG-348 and AG-519; its plans and timelines for the clinical development of AG-348 and AG-519; its plans regarding future data presentations; and the benefit of its strategic plans and focus. 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, 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: 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, such as its agreements with Celgene; and general economic and market conditions.  These and other risks are described in greater detail under the caption “Risk Factors” included in Agios’ Quarterly Report on Form 10-Q for the quarter ended March 31, 2016, and other filings that Agios may make with the Securities and Exchange Commission in the future. 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.

Contact:
Renee Leck, Senior Manager, Investor & Public Relations
Renee.Leck@agios.com
617-649-8299