Agios highlights the milestones and priorities planned for 2022 to realize the strategic vision of the company in the field of genetically defined diseases

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– Company expects to receive FDA regulatory decision for Mitapivat as a potential treatment for adults with PK deficiency in February –

– Five pivotal clinical trials are planned and underway on thalassemia, sickle cell anemia and pediatric PK deficiency –

– Agios expands its clinical portfolio with the trials of the Novel PK Activator AG-946 –

– A strong cash position should allow the execution of a robust operating plan for positive cash flow –

CAMBRIDGE, Mass., Jan. 10, 2022 (GLOBE NEWSWIRE) – Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in pioneering cell metabolism therapies for genetically defined diseases, today announced its planned key milestones for 2022 and priorities that will lead to its transition to a commercial-stage genetically defined disease company and the expansion of its strong clinical and research portfolio. Agios will be presenting at JP Morgan’s 40th Annual Virtual Healthcare Conference on Wednesday, January 12 at 7:30 a.m. ET, and a live webcast will be available at

“We are starting an exciting and defining year for Agios as we prepare for our first product launch against genetically defined diseases, execute five pivotal clinical trials, continue to expand our clinical PK activation portfolio and promote our search engine innovative, ”said Jackie Fouse, Ph.D., CEO of Agios. “We are already making great progress with mitapivat being evaluated in the US FDA’s Priority Review as a potential treatment for adults with PK deficiency and are running our Phase 3 studies ENERGIZE and ENERGIZE-T in thalassemia and the phase 2/3 RISE UP study in sickle cell disease. In 2022, we plan to continue our strategy of expanding PK activation by launching our pivotal pediatric PK deficiency trials, completing recruitment in the Phase 2 portion of the RISE UP study and advancing our new PK activator AG-946 in sickle cell anemia and weak to intermediate risk myelodysplastic syndrome. At Agios, we are fueled by connections – with patient communities, a world-class network of healthcare providers and researchers, and with each other – and these collaborative partnerships will determine our key priorities and move forward. care for people with genetically defined diseases.

Milestones and priorities expected for 2022

Agios plans to execute the following key milestones and priorities in 2022:

Pyruvate kinase (PK) deficiency

  • Receive US Food and Drug Administration (FDA) regulatory decision for mitapivat in adults with first trimester PK deficiency
  • Receive the regulatory decision from the European Medicines Agency (EMA) for mitapivat in adults with PK deficiency by year-end
  • Initiate the phase 3 ACTIVATE-kids and ACTIVATE-kidsT studies on mitapivat in pediatric patients without regular transfusions and regularly transfused with PK deficiency, respectively, by mid-2022


  • Enroll a significant portion of patients in the phase 3 ENERGIZE and ENERGIZE-T studies with mitapivat in non-transfused and regularly transfused adults with thalassemia, respectively, by the end of the year

Sickle cell anemia

  • Complete the recruitment in the phase 2 part of the RISE UP study on mitapivat in sickle cell disease by the end of the year
  • Launch the sickle cell cohort of the phase 1 study of the new PK AG-946 activator in the first half of 2022

Expansion and acceleration of the PK activation portfolio

  • Launch phase 2a study of AG-946 in adults with low-to-intermediate risk myelodysplastic syndrome (MDS) by year-end
  • To continue to publish clinical and translational data supporting the utility of PK activators in key areas of disease and to elucidate the disease burden for PK deficiency, thalassemia and sickle cell anemia

Agios five-year strategic visionAgios’ five-year strategic vision reflects the expected evolution of the company as a leader in the development of therapies against genetically defined diseases based on its scientific expertise in cell metabolism. As part of this vision, Agios plans to achieve the following milestones by the end of 2026:

  • Receive regulatory approvals globally for mitapivat in three initial indications: adult PK deficiency, thalassemia and sickle cell disease
  • Advance a large clinical pipeline of at least 5 molecules exploring at least 10 indications
  • Foster a strong research pipeline ready to deliver an Investigational New Drug (IND) every 12-24 months
  • Achieve positive cash flow

Presentation at 40e JP Morgan Annual Healthcare ConferenceAgios will webcast its corporate presentation from JP Morgan Healthcare’s 40th Annual Virtual Conference on Wednesday, January 12 at 7:30 a.m.ET. A live webcast of the presentation is available under “Events and Presentations” in the Investors section of the Company’s website at A replay of the webcast will be archived on the Agios website for at least two weeks after the presentation.

About AgiosAgios is a biopharmaceutical company that thrives on connections. The Agios team cultivates strong relationships with patient communities, healthcare professionals, partners and colleagues to discover, develop and deliver therapies for genetically defined diseases. Building on the company’s leadership in cell metabolism, Agios is developing a strong portfolio of investigational drugs, including a first-class pyruvate kinase (PK) activator that is being evaluated for the treatment of three Distinct hemolytic anemias – pyruvate kinase deficiency, alpha- and beta-thalassemia and sickle cell anemia. In addition to its active late-stage clinical pipeline, Agios has several new experimental therapies in clinical and preclinical development. For more information, please visit the company’s website at

Caution regarding forward-looking statementsThis press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements include those concerning Agios’ plans, strategies and expectations for the preclinical, clinical and commercial advancement of its drug development programs. , including mitapivat and AG-946; the potential benefits of Agios’ products and product candidates; The key stages and orientations of Agios for 2022 and its strategic vision for 2026; its financial guidelines regarding the period during which it will have capital to finance its operations; and the potential benefits of Agios’ strategic plans and direction. The words “anticipate”, “expect”, “objective”, “hope”, “,” are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Such statements are subject to many important factors, risks and uncertainties which may cause actual events or results to differ materially from Agios’ current expectations and beliefs. Management’s expectations and, accordingly, all forward-looking statements contained in this press release could also be affected by risks and uncertainties relating to a number of other important factors, including, without limitation, the risks and uncertainties associated with: Agios’ failure to receive milestones or royalty payments related to the sale of its oncology business, the uncertainty of the timing of any receipt of such payments, and the uncertainty the results and effectiveness of the use of the proceeds of the transaction with Servier; the impact of the COVID-19 pandemic on Agios’ business, operations, strategy, goals and planned milestones, including its ongoing and planned research activities, its ability to conduct ongoing clinical trials and planned, the clinical supply of current or future drug candidates, the commercial supply of future approved products, and the launch, marketing and sale of future approved products; the results of clinical trials and preclinical studies of Agios, including subsequent analysis of existing data and new data received from ongoing and future studies; the content and timing of decisions made by the US FDA, EMA or other regulatory authorities, including with respect to regulatory submissions for mitapivat, investigative review panels at sites clinical trials and publication review bodies; Agios’ ability to obtain and maintain required regulatory approvals and enroll patients in its planned clinical trials; unforeseen cash needs and expenses and competitive factors; Agios’ ability to obtain, maintain and enforce patents and other intellectual property protections for all product candidates it develops; Agios’ ability to establish and maintain collaborations; and general economic and market conditions. These and other risks are described in more detail under the heading “Risk Factors” included in Agios’ public filings with the Securities and Exchange Commission, or SEC, including the risks and uncertainties set forth under the heading “Risk Factors”. risk in our documents filed with the SEC. . Although the list of factors presented here is considered representative, this list should not be taken as a complete statement of all potential risks and uncertainties. All forward-looking statements contained in this communication are made only as of the date hereof, and we assume no obligation to update any forward-looking statements to reflect developments or information obtained after the date hereof and disclaim any obligation. to do so other than what may be required by law.


Investors:Holly Manning, 617-844-6630 Senior Director, Investor Relations [email protected]

Media:Jessica Rennekamp, ​​857-209-3286 Director, Corporate [email protected]

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Source: Agios Pharmaceuticals, Inc.

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