Tebapivat

– Filed for Regulatory Approval of Mitapivat (PYRUKYND®) for the Treatment of Adult Patients with Non-Transfusion-Dependent and Transfusion-Dependent Alpha- or Beta-Thalassemia in U.S., European Union , Kingdom of Saudi Arabia and United Arab Emirates; PDUFA Goal Date of September 7, 2025 – – Completed Enrollment for Phase 3 RISE UP Study of Mitapivat in Sickle Cell Disease; Topline Results Expected in Late 2025, with Potential U.S. Commercial Launch in 2026 – – Early- and Mid-Stage Pipeline Offers Strong Foundation for Innovation and Growth – – PYRUKYND Net Revenue of $10.7 Million in Q4; Cash, Cash Equivalents and Marketable Securities of $1.5 Billion as of December 31, 2024 – CAMBRIDGE, Mass. , Feb. 13, 2025 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in cellular metabolism and pyruvate kinase (PK) activation pioneering therapies for rare diseases, today reported business highlights and financial results for the fourth quarter and year ended December 31, 2024 . “Agios had a transformative year in 2024, continuing to successfully deliver on all priorities. Our PYRUKYND franchise is poised for multi-billion-dollar potential, driven by the key milestones we achieved last year, including filing for regulatory approval in thalassemia across four markets and completing enrollment in our Phase 3 RISE UP study for sickle cell disease,” said Brian Goff, chief executive officer at Agios. “Backed by a strong balance sheet and a highly experienced team, Agios is focused on maximizing the potential PYRUKYND launches in thalassemia and sickle cell disease in 2025 and 2026, respectively, while advancing and diversifying our key pipeline programs and strategically deploying our capital to drive long-term growth. We are well positioned to bring significant value for shareholders, healthcare professionals and patients, as we build towards a breakout year in 2025.” Fourth Quarter 2024 and Recent Highlights PYRUKYND® Revenues: Generated $10.7 million in net revenue for the fourth quarter of 2024, a 20 percent increase from the third quarter of 2024, primarily driven by year-end stocking and adjustments to certain revenue reserves. A total of 223 unique patients have completed prescription enrollment forms, representing an increase of 6 percent over the third quarter of 2024. A total of 130 patients are on PYRUKYND therapy, inclusive of new prescriptions and continued therapy. Thalassemia: Presented positive results from the ENERGIZE-T Phase 3 randomized clinical trial evaluating mitapivat versus placebo in adults with transfusion-dependent alpha- or beta-thalassemia at the 66ᵗʰ American Society of Hematology Annual Meeting and Exposition (ASH 2024) in December 2024. Based on the favorable benefit-risk profile observed in both the ENERGIZE and ENERGIZE-T Phase 3 studies, filed regulatory applications for mitapivat (PYRUKYND) for the treatment of adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia with the U.S., European Union, Kingdom of Saudi Arabia and United Arab Emirates health authorities. The U.S. Food and Drug Administration (FDA) accepted the company’s supplemental New Drug Application (sNDA) for PYRUKYND for the treatment of adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia. The review classification for this application is Standard and the Prescription Drug User Fee Act (PDUFA) goal date is September 7, 2025. Sickle Cell Disease: Completed enrollment of the Phase 3 RISE UP study evaluating mitapivat for the treatment of sickle cell disease patients who are 16 years of age or older. This Phase 3 study enrolled more than 200 patients worldwide. European Commission adopted a positive decision for the designation of mitapivat as an orphan medicinal product for the treatment of sickle cell disease. Presented Phase 1 clinical results on tebapivat (AG-946) in patients with sickle cell disease at ASH 2024. Pediatric Pyruvate Kinase (PK) Deficiency: Reported in a separate press release today, positive topline results from the ACTIVATE-Kids Phase 3 study of mitapivat in children aged 1 to <18 years with PK deficiency who are not regularly transfused. Lower-risk myelodysplastic syndromes (LR-MDS): Initiated patient enrollment in the Phase 2b study of tebapivat in LR-MDS. FDA granted orphan drug designation to tebapivat for the treatment of MDS. Medical Congresses: Presented 16 abstracts highlighting new data on mitapivat and tebapivat at ASH 2024. Corporate: David Schenkein, M.D., has informed the company that he will step down from Agios’ Board of Directors, effective February 28, 2025 , to devote more time to his other commitments. He will continue to serve as a strategic advisor to Agios’ Leadership Team, concentrating on advancing the company’s clinical development programs. Presented positive results from the ENERGIZE-T Phase 3 randomized clinical trial evaluating mitapivat versus placebo in adults with transfusion-dependent alpha- or beta-thalassemia at the 66ᵗʰ American Society of Hematology Annual Meeting and Exposition (ASH 2024) in December 2024. Based on the favorable benefit-risk profile observed in both the ENERGIZE and ENERGIZE-T Phase 3 studies, filed regulatory applications for mitapivat (PYRUKYND) for the treatment of adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia with the U.S., European Union, Kingdom of Saudi Arabia and United Arab Emirates health authorities. The U.S. Food and Drug Administration (FDA) accepted the company’s supplemental New Drug Application (sNDA) for PYRUKYND for the treatment of adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia. The review classification for this application is Standard and the Prescription Drug User Fee Act (PDUFA) goal date is September 7, 2025. Completed enrollment of the Phase 3 RISE UP study evaluating mitapivat for the treatment of sickle cell disease patients who are 16 years of age or older. This Phase 3 study enrolled more than 200 patients worldwide. European Commission adopted a positive decision for the designation of mitapivat as an orphan medicinal product for the treatment of sickle cell disease. Presented Phase 1 clinical results on tebapivat (AG-946) in patients with sickle cell disease at ASH 2024. Reported in a separate press release today, positive topline results from the ACTIVATE-Kids Phase 3 study of mitapivat in children aged 1 to <18 years with PK deficiency who are not regularly transfused. Initiated patient enrollment in the Phase 2b study of tebapivat in LR-MDS. FDA granted orphan drug designation to tebapivat for the treatment of MDS. Key Upcoming Milestones & Priorities Agios expects to achieve the following key milestones in 2025: Thalassemia: Receive FDA regulatory decision for PYRUKYND for the treatment of adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia (PDUFA goal date is September 7, 2025). Sickle Cell Disease: Announce topline results from the Phase 3 RISE UP study of mitapivat in sickle cell disease in late 2025, with a potential U.S. commercial launch in 2026. Additionally, begin patient enrollment for the Phase 2 study of tebapivat in sickle cell disease in mid-2025. LR-MDS: Complete patient enrollment in the Phase 2b study of tebapivat for LR-MDS in late 2025. Early-Stage Pipeline: File an Investigational New Drug Application for AG-236, a siRNA targeting TMPRSS6 intended for the treatment of polycythemia vera, in mid-2025. Fourth Quarter 2024 Financial Results Revenue: Net product revenue from sales of PYRUKYND for the fourth quarter of 2024 was $10.7 million , compared to $7.1 million for the fourth quarter of 2023, and $36.5 million for the year ended December 31, 2024 , compared to $26.8 million for the year ended December 31, 2023 . Cost of Sales: Cost of sales for the fourth quarter of 2024 was $1.3 million and $4.2 million for the full year ended December 31, 2024 . Research and Development (R&D) Expenses: R&D expenses were $82.8 million for the fourth quarter of 2024, compared to $77.5 million for the fourth quarter of 2023, and $301.3 million for the full year ended December 31, 2024 , compared to $295.5 million for the full year ended December 31, 2023 . Selling, General and Administrative (SG&A) Expenses: SG&A expenses were $51.7 million for the fourth quarter of 2024 compared to $35.3 million for the fourth quarter of 2023, and $156.8 million for the full year ended December 31, 2024 , compared to $119.9 million for the full year ended December 31, 2023 . The year-over-year increase was primarily attributable to an increase in commercial-related activities as the company prepares for the potential approval of PYRUKYND in thalassemia. Net Income (Loss): Net loss was $96.5 million for the fourth quarter of 2024 compared to a net loss of $95.9 million for the fourth quarter of 2023, and net income was $673.7 million for the year ended December 31, 2024 , compared to a net loss of $352.1 million for the year ended December 31, 2023 . Cash Position and Guidance: Cash, cash equivalents and marketable securities as of December 31, 2024 , were $1.5 billion compared to $806.4 million as of December 31, 2023 . This increase reflects payments from the royalty monetization for vorasidenib and a milestone payment from Servier for vorasidenib approval received in the third quarter of 2024. Agios expects that its cash, cash equivalents and marketable securities, together with anticipated product revenue and interest income, will provide the financial independence to prepare for potential PYRUKYND launches in thalassemia and sickle cell disease, advance existing programs, and to opportunistically expand its pipeline through both internally and externally discovered assets. Conference Call Information Agios will host a conference call and live webcast today at 8:00 a.m. ET to discuss the company’s fourth quarter 2024 financial results and recent business highlights. The live webcast will be accessible on the Investors section of the company's website (www.agios.com) under the “Events & Presentations” tab. A replay of the webcast will be available on the company’s website approximately two hours after the event. About Agios Agios is the pioneering leader in PK activation and is dedicated to developing and delivering transformative therapies for patients living with 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 deep scientific expertise in classical hematology and leadership in the field of cellular metabolism and rare hematologic diseases, Agios is advancing a robust clinical pipeline of investigational medicines with programs in alpha- and beta-thalassemia, sickle cell disease, pediatric PK deficiency, myelodysplastic syndromes (MDS)-associated anemia and phenylketonuria (PKU). In addition to its clinical pipeline, Agios is advancing a preclinical TMPRSS6 siRNA as a potential treatment for polycythemia vera. 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 PYRUKYND® (mitapivat), tebapivat (AG-946) , AG -236 and AG-181, Agios’ PAH stabilizer; Agios’ plans, strategies and expectations for its preclinical, clinical and commercial advancement of its drug development, including PYRUKYND®, tebapivat, AG-236 and AG-181; Agios’ use of proceeds from the transaction with Royalty Pharma; potential U.S. net sales of vorasidenib and potential future royalty payments; Agios’ strategic vision and goals, including its key milestones for 2025; and 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 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; 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 establish and maintain key collaborations; uncertainty regarding any royalty payments related to the sale of its oncology business or any milestone or royalty payments related to its in-licensing of AG-236, and the uncertainty of the timing of any such payments; uncertainty of the results and effectiveness of the use of Agios’ cash and cash equivalents; 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. Contacts: Investor Contact Chris Taylor , VP, Investor Relations and Corporate Communications Agios Pharmaceuticals IR@agios.com Media Contact Eamonn Nolan , Senior Director, Corporate Communications Agios Pharmaceuticals media@agios.com Source: Agios Pharmaceuticals, Inc.

– FDA Accepted Agios’ Supplemental New Drug Application for PYRUKYND® (mitapivat) in Adult Patients with Non-Transfusion-Dependent and Transfusion-Dependent Alpha- or Beta-Thalassemia; PDUFA Goal Date is September 7, 2025 – – Topline Results from Phase 3 RISE UP Study of Mitapivat in Sickle Cell Disease to be Announced in Late 2025, with Potential U.S. Commercial Launch in 2026 – – Strong Financial Position Provides Opportunity to Maximize Potential PYRUKYND Commercial Launches, Advance Early- and Mid-Stage Clinical Programs and Expand Pipeline – CAMBRIDGE, Mass. , Jan. 13, 2025 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in cellular metabolism and pyruvate kinase (PK) activation pioneering therapies for rare diseases, today announced its anticipated key 2025 milestones and value-driving catalysts through 2026. The company’s management team will present this information at the 43rd Annual J.P. Morgan Healthcare Conference on Wednesday, January 15, 2025, at 7:30 a.m. PT / 10:30 a.m. ET . “2024 was marked by exceptional progress at Agios. We delivered on all our key priorities, advanced our potential best- and first-in-class rare disease pipeline and further strengthened our financial position. Today, we are entering an era of growth and expansion for the company, building on a strong foundation and focus, and are well-positioned for a sustained trajectory of success,” said Brian Goff, chief executive officer at Agios. “Our blueprint encompasses the potential for two additional commercial launches of PYRUKYND in thalassemia and sickle cell disease in 2025 and 2026, respectively, along with an early- and mid-stage pipeline that offers a strong foundation for innovation and growth, all supported by a highly experienced team with proven executional excellence and a strong balance sheet. Over the next 12 months, our priorities will be to maximize the potential of the PYRUKYND franchise, advance and diversify our key pipeline programs, and strategically focus our capital deployment to sustain our growth. We are excited about the future and the meaningful impact we can have in addressing the critical needs of rare disease patients.” 2024 Highlights: Thalassemia: Presented positive results from the ENERGIZE and ENERGIZE-T Phase 3 trials evaluating mitapivat versus placebo in adults with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia, respectively. The ENERGIZE randomized clinical trial results were presented at the European Hematology Association 2024 Hybrid Congress in June 2024 , and the ENERGIZE-T randomized clinical trial results were presented at the 66th American Society of Hematology Annual Meeting and Exposition in December 2024 . Agios filed regulatory applications for mitapivat (PYRUKYND) for the treatment of adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia with the U.S., European Union, Kingdom of Saudi Arabia and United Arab Emirates health authorities. Sickle Cell Disease: Completed enrollment of the Phase 3 RISE UP study that is evaluating mitapivat in sickle cell disease patients who are 16 years of age or older. This Phase 3 study enrolled more than 200 patients worldwide. Pediatric Pyruvate Kinase (PK) Deficiency: Reported topline results from the Phase 3 ACTIVATE-KidsT trial of mitapivat in children with PK deficiency who are regularly transfused. Further, completed enrollment of the Phase 3 ACTIVATE-Kids study of mitapivat in children with PK deficiency who are not regularly transfused. Lower-Risk Myelodysplastic Syndromes (LR-MDS): Initiated patient enrollment in the Phase 2b study of tebapivat (AG-946) in LR-MDS. Additionally, the U.S. Food and Drug Administration (FDA) granted orphan drug designation to tebapivat for the treatment of MDS. Early-Stage Pipeline: Dosed the first healthy volunteer participants in the Phase 1 study of AG-181, a PAH stabilizer, in phenylketonuria. Corporate Development: Announced a $905 million purchase agreement with Royalty Pharma for Agios’ rights to its vorasidenib royalty. Under the agreement, Agios received a payment of $905 million following the approval of vorasidenib by the FDA. Royalty Pharma will receive the entirety of the 15% royalty on annual U.S. net sales of vorasidenib up to $1 billion, and a 12% royalty on annual U.S. net sales greater than $1 billion. Agios retains a 3% royalty on annual U.S. net sales greater than $1 billion. Agios also received a $200 million milestone payment from Servier following the FDA approval of vorasidenib. Altogether, Agios received a total of $1.1 billion in milestone payments as part of this purchase agreement. Entered into a distribution agreement with NewBridge Pharmaceuticals to advance commercialization of PYRUKYND in the Gulf Cooperation Council (GCC) region. NewBridge, a leading specialty company headquartered in Dubai, will commercialize PYRUKYND in Bahrain, Kuwait, Oman, Qatar, Saudi Arabia and the United Arab Emirates. Announced a $905 million purchase agreement with Royalty Pharma for Agios’ rights to its vorasidenib royalty. Under the agreement, Agios received a payment of $905 million following the approval of vorasidenib by the FDA. Royalty Pharma will receive the entirety of the 15% royalty on annual U.S. net sales of vorasidenib up to $1 billion, and a 12% royalty on annual U.S. net sales greater than $1 billion. Agios retains a 3% royalty on annual U.S. net sales greater than $1 billion. Agios also received a $200 million milestone payment from Servier following the FDA approval of vorasidenib. Altogether, Agios received a total of $1.1 billion in milestone payments as part of this purchase agreement. Entered into a distribution agreement with NewBridge Pharmaceuticals to advance commercialization of PYRUKYND in the Gulf Cooperation Council (GCC) region. NewBridge, a leading specialty company headquartered in Dubai, will commercialize PYRUKYND in Bahrain, Kuwait, Oman, Qatar, Saudi Arabia and the United Arab Emirates. Anticipated 2025 Milestones: Thalassemia: Receive FDA regulatory decision for PYRUKYND for the treatment of adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia. The review classification for the company’s supplemental New Drug Application is Standard and the Prescription Drug User Fee Act (PDUFA) goal date is September 7, 2025. Sickle Cell Disease: Announce topline results from the Phase 3 RISE UP study of mitapivat in sickle cell disease in late 2025, with a potential U.S. commercial launch in 2026. Additionally, begin patient enrollment for the Phase 2 study of tebapivat in sickle cell disease in mid-2025. Pediatric Pyruvate Kinase (PK) Deficiency: Announce topline results from the Phase 3 ACTIVATE-Kids study of mitapivat in children with PK deficiency who are not regularly transfused in early 2025. Lower-Risk Myelodysplastic Syndromes (LR-MDS): Complete patient enrollment in the Phase 2b study of tebapivat for LR-MDS in late 2025. Early-Stage Pipeline: File an Investigational New Drug Application for AG-236, a siRNA targeting TMPRSS6 intended for the treatment of polycythemia vera, in mid-2025. Presentation at 43rd Annual J.P. Morgan Healthcare Conference Agios’ management team will present at the 43rd Annual J.P. Morgan Healthcare Conference on Wednesday, January 15, 2025 , at 7:30 a.m. PT / 10:30 a.m. ET . The live webcast will be accessible on the Investors section of the company's website (www.agios.com) under the “Events & Presentations” tab. A replay of the webcast will be archived on the company’s website for at least two weeks following the presentation. About PYRUKYND® (mitapivat) U.S. INDICATIONPYRUKYND is a pyruvate kinase activator indicated for the treatment of hemolytic anemia in adults with pyruvate kinase (PK) deficiency. U.S. IMPORTANT SAFETY INFORMATIONAcute Hemolysis: Acute hemolysis with subsequent anemia has been observed following abrupt interruption or discontinuation of PYRUKYND in a dose-ranging study. Avoid abruptly discontinuing PYRUKYND. Gradually taper the dose of PYRUKYND to discontinue treatment if possible. When discontinuing treatment, monitor patients for signs of acute hemolysis and anemia including jaundice, scleral icterus, dark urine, dizziness, confusion, fatigue, or shortness of breath. Hepatocellular Injury in Another Condition: In patients with another condition treated with PYRUKYND at a higher dose than that recommended for patients with PK deficiency, liver injury has been observed. These events were characterized by a time to onset within the first 6 months of treatment with peak elevations of alanine aminotransferase of >5× upper limit of normal (ULN) with or without jaundice. All patients discontinued treatment with PYRUKYND, and these events improved upon treatment discontinuation. Obtain liver tests prior to the initiation of PYRUKYND and monthly thereafter for the first 6 months and as clinically indicated. Interrupt PYRUKYND if clinically significant increases in liver tests are observed or alanine aminotransferase is >5x ULN. Discontinue PYRUKYND if hepatic injury due to PYRUKYND is suspected. Adverse Reactions: The most common adverse reactions including laboratory abnormalities (≥10%) in patients with PK deficiency were estrone decreased (males), increased urate, back pain, estradiol decreased (males), and arthralgia. Drug Interactions: Strong CYP3A Inhibitors and Inducers: Avoid concomitant use. Moderate CYP3A Inhibitors: Do not titrate PYRUKYND beyond 20 mg twice daily. Moderate CYP3A Inducers: Consider alternatives that are not moderate inducers. If there are no alternatives, adjust PYRUKYND dosage. Sensitive CYP3A, CYP2B6, CYP2C Substrates Including Hormonal Contraceptives: Avoid concomitant use with substrates that have narrow therapeutic index. UGT1A1 Substrates: Avoid concomitant use with substrates that have narrow therapeutic index. P-gp Substrates: Avoid concomitant use with substrates that have narrow therapeutic index. Hepatic Impairment: Avoid use of PYRUKYND in patients with moderate and severe hepatic impairment. Please see full Prescribing Information for PYRUKYND. About AgiosAgios is the pioneering leader in PK activation and is dedicated to developing and delivering transformative therapies for patients living with 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 deep scientific expertise in classical hematology and leadership in the field of cellular metabolism and rare hematologic diseases, Agios is advancing a robust clinical pipeline of investigational medicines with programs in alpha- and beta-thalassemia, sickle cell disease, pediatric PK deficiency, myelodysplastic syndrome (MDS)-associated anemia and phenylketonuria (PKU). In addition to its clinical pipeline, Agios is advancing a preclinical TMPRSS6 siRNA as a potential treatment for polycythemia vera. 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 PYRUKYND® (mitapivat), tebapivat (AG-946) , AG -236 and AG-181, Agios’ PAH stabilizer; Agios’ plans, strategies and expectations for its preclinical, clinical and commercial advancement of its drug development, including PYRUKYND®, tebapivat, AG-181 and AG-236; the submission of PYRUKYND® to regulators for approval in alpha-and-beta thalassemia; Agios’ strategic vision and goals, including its key milestones for 2025; and the potential benefits of Agios’ strategic plans and focus. The words “anticipate”, “expect”, “goal”, “hope”, “milestone”, “opportunity”, “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 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; 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 establish and maintain key collaborations; uncertainty regarding any royalty payments related to the sale of its oncology business or any milestone or royalty payments related to its in-licensing of AG-236, and the uncertainty of the timing of any such payments; uncertainty of the results and effectiveness of the use of Agios’ cash and cash equivalents; 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. Contacts: Investor Contact Chris Taylor , VP, Investor Relations and Corporate Communications Agios Pharmaceuticals IR@agios.com Media Contact Eamonn Nolan , Senior Director, Corporate Communications Agios Pharmaceuticals Media@agios.com Source: Agios Pharmaceuticals, Inc.