Topline Results: Phase 3 Energize Study of Pyrukynd in Thalassemia

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Agios

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Healthcare

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2024

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#1#2#33 Forward-looking statements This presentation and various remarks we make during this presentation contain 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 future clinical development of mitapivat in alpha-and-beta thalassemia; 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 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 any medicines ultimately commercialized by Agios 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 presentation and various remarks we make during this presentation 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 pandemics or other public health emergencies 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; Agios' ability to obtain, maintain and enforce patent and other intellectual property protection for any product candidates it is developing; Agios' ability to establish and maintain key collaborations; uncertainty regarding any milestone or royalty payments related to the sale of Agios' oncology business or its in-licensing of TMPRSS6 siRNA, and the uncertainty of the timing of any such payments; uncertainty of the results and effectiveness of the use of proceeds from the transaction with Servier; competitive factors; 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 presentation and various remarks we make during this presentation 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.#4#5agios Data Highlights from Phase 3 ENERGIZE Study Jeremie Estepp, M.D. Medical Director at Agios#66 There are no approved oral treatments for non-transfusion-dependent thalassemia (NTDT) ● ● ● ● Thalassemia is a group of genetic disorders impacting a- and/or B-globin genes, resulting in an imbalance of globin production ¹,2 ■ Excess globin chains precipitate and are toxic to red blood cells (RBCs), directly leading to in effective erythropoiesis and hemolysis² Thalassemic RBCs lack sufficient levels of ATP to meet the increased energy demands associated with degradation of globin chain precipitates and cellular oxidative stress responses 3,4 Although patients with non-transfusion-dependent thalassemia (NTDT) do not require regular blood transfusions for survival, they can experience chronic anemia and serious complications 1,2 Treatment options for NTDT are supportive only, highlighting an unmet need for disease-modifying therapies 5 Mitapivat is an investigational, first-in-class, oral, small-molecule allosteric activator of pyruvate kinase (PK) in RBCs, a key enzyme that regulates ATP production6 ■ PYRUKYND® is approved in the U.S., EU, and Great Britain for adult PK deficiency and is under investigation for pediatric PK deficiency, thalassemia, and sickle cell disease. ATP, adenosine triphosphate; NTDT, non-transfusion-dependent thalassemia; PK, pyruvate kinase; RBC, red blood cell; 1. Taher AT et al. Lancet 2018;391:155-67; 2. Galanello R et al. Orphanet J Rare Dis 2010;5:11; 3. Khandros E et al. Blood 2012;119:5265-75; 4. Shaeffer JR. J Biol Chem 1988;263:13663-9; 5. Musallam KM et al. Haematologica 2021;106:2489-92; 6. Kung C et al. Blood 2017;130:1347-56#7#8#9#10#11#12#13#14#15#16#17#18#19#20

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