Agios Highlights 2021 Milestones to Accelerate and Expand Its Genetically Defined Disease Portfolio and Drive Near- and Long-Term Value Creation

– Company Expects to File for Regulatory Approval for Mitapivat for the Treatment of Adults with Pyruvate Kinase (PK) Deficiency in the U.S. in Q2 2021 and in the EU in Mid-2021 –

– Pivotal Trials for Mitapivat in Thalassemia on Track for Initiation in 2H 2021 and in Sickle Cell Disease by Year-End –

– Sale of Oncology Portfolio to Servier Expected to Close in Q2 2021, Enabling Agios to Focus on Genetically Defined Diseases –

– Company’s 2025 Strategic Vision Includes Commercialization of Mitapivat in 3 Indications, 5+ Molecules in Clinical Development across 10+ Indications, an Investigational New Drug Every 12-24 Months and a Cash-Flow Positive Business –

CAMBRIDGE, Mass., Jan. 11, 2021 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism to treat cancer and genetically defined diseases, today announced its key 2021 milestones that will drive its recently announced strategic pivot to focus on developing and commercializing innovative treatments for genetically defined diseases, as well as its five-year vision for the company. Agios will present at the virtual 39th Annual J.P. Morgan Healthcare Conference on Monday, January 11 at 10:50 a.m. ET, and a live webcast will be available at investor.agios.com.

“Agios is at an exciting inflection point as we prepare to move forward with a singular focus on genetically defined diseases,” said Jackie Fouse, Ph.D., chief executive officer of Agios. “2021 will be a year of significant momentum and further evidence of our potential to meaningfully impact the lives of patients with unmet needs in pyruvate kinase deficiency, thalassemia, sickle cell disease and other genetically defined diseases. This year, we expect to file for the approval of mitapivat in pyruvate kinase deficiency, which currently has no disease-modifying treatment options, and to initiate pivotal development programs in thalassemia and sickle cell disease. We also expect to further unlock the potential of PK activation across a range of genetically defined diseases by advancing our rich and sustainable research pipeline. As we reimagine the future of Agios, we look forward to building on our core values and pioneering leadership in cellular metabolism to expand and accelerate our work on behalf of patients.”