AGIO Agios Pharmaceuticals Inc

Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in the field of cellular metabolism pioneering therapies for rare diseases, today announced the launch of a multi-stakeholder advocacy advisory council for hemolytic anemias including PK deficiency, thalassemia and sickle cell disease. The council will bring together patients, caregivers and physicians from these communities, which share commonalities in underlying disease pathology, difficulties in transitioning from pediatric to adult care and immense quality of life concerns, to generate published evidence and meaningful solutions that are relevant across disease areas and to create a platform to raise awareness about the needs and experiences of people touched by these diseases.

“The Red Cell Revolution advisory council is designed to build connections among PK deficiency, thalassemia and sickle cell disease advocates and thought leaders, and to generate insights to raise disease awareness, inform Agios’ mission and contribute to shaping the broader hemolytic anemia field through evidence-based patient advocacy,” said Sarah Gheuens, M.D., Ph.D., chief medical officer and head of R&D at Agios. “On Rare Disease Day, we celebrate the power of various rare disease communities coming together, finding common ground and amplifying each other’s voices; our hope is that the Red Cell Revolution can accomplish similar goals and have an important impact for rare blood disorder communities.”

“The Red Cell Revolution is a real opportunity to bring together great minds and passionate individuals, including people who live every day with these conditions that have more similarities than differences,” said Biree Andemariam, M.D., hematologist and founding director of the New England Sickle Cell Institute (NESCI) at the University of Connecticut Health Center, and council member of the Red Cell Revolution. “Together, we can be a powerful, unified voice to drive change not only in the U.S., but also throughout the world, and to strive for these underserved conditions to get the same prioritization, innovation, access and compassionate care that more common illnesses get. I’m grateful to Agios for spearheading this initiative and facilitating these connections which can fuel positive changes for people with sickle cell disease, thalassemia and PK deficiency.”

To learn more about the Red Cell Revolution, watch this video or listen to this podcast episode, which debuted yesterday across three Agios-sponsored podcasts, including Just Listen: Voices of PK Deficiency, Thal Pals: The Alpha Beta Revolution and Cheat Codes: A Sickle Cell Podcast.

The Agios team is also supporting rare disease communities throughout the month of February as a sponsor of the following initiatives:

• Uplifting Athletes: Health Equity Initiative, which supports rare disease researchers from underrepresented backgrounds
• MassBio: Rare Disease Day 2023
• National Organization for Rare Disorders: Rare Disease Day 2023

About AgiosAgios is a biopharmaceutical company that is fueled by connections. The Agios team cultivates strong bonds with patient communities, healthcare professionals, partners and colleagues to discover, develop and deliver therapies for 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 leadership in the field of cellular metabolism, Agios is advancing a robust clinical pipeline of investigational medicines with programs in alpha- and beta-thalassemia, sickle cell disease, pediatric PK deficiency and MDS-associated anemia. In addition to its clinical pipeline, Agios has a PAH stabilizer in preclinical development as a potential treatment for phenylketonuria (PKU) and deep scientific expertise in classical hematology. For more information, please visit the company’s website at www.agios.com.

Cautionary Note Regarding Forward-Looking StatementsThis 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’ 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 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; the failure of Agios to receive milestone or royalty payments related to the sale of its oncology business, the uncertainty of the timing of any receipt of any such payments, and the uncertainty of the results and effectiveness of the use of proceeds from the transaction with Servier; 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.

Investor & Media Contact

Jessica RennekampSenior Director, Corporate CommunicationsJessica.Rennekamp@agios.com