Mitapivat

CAMBRIDGE, Mass. , Dec. 08, 2025 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a commercial-stage biopharmaceutical company focused on delivering innovative medicines for patients with rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has not yet issued a regulatory decision on the supplemental New Drug Application (sNDA) for mitapivat for the treatment of adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia and that the sNDA remains under active review. The Prescription Drug User Fee Act (PDUFA) goal date issued by the FDA was December 7, 2025 . Agios is collaborating closely with the FDA to finalize the labeling documents and Risk Evaluation and Mitigation Strategy (REMS) materials. The FDA has not requested, and the company has not submitted, any new or additional efficacy or safety data. The FDA has not provided a timeline for its regulatory decision. Agios continues to work expeditiously with the FDA to conclude the review of the sNDA. About Agios: Fueled by Connections to Transform Rare Diseases™ At Agios, our vision is to redefine the future of rare disease treatment. Fueled by connections, we build trusted partnerships with communities – collaborating to develop and deliver innovative medicines that have the potential to transform lives. With a foundation in hematology, we combine biological expertise with real-world insights to advance a growing pipeline of rare disease medicines that reflect the priorities of the people we serve. Agios is a commercial-stage biopharmaceutical company headquartered in Cambridge, Massachusetts . To learn more, visit www.agios.com and follow us on LinkedIn and X. 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’ products, including mitapivat, its interactions with regulators, including the FDA, and its 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: 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’ results of clinical trials and preclinical studies, including subsequent analysis of existing data and new data received from ongoing and future studies; 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’ 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 Morgan Sanford , Vice President, Investor Relations Agios Pharmaceuticals morgan.sanford@agios.com Media Contact Eamonn Nolan , Senior Director, Corporate Communications Agios Pharmaceuticals eamonn.nolan@agios.com Source: Agios Pharmaceuticals, Inc.

Agios' Pyrukynd missed one of its two primary endpoints in a phase 3 sickle cell disease trial, but the company is still plotting an FDA bid next year. In its mission to grow the reach of its pyruvate kinase (PK) activator Pyrukynd (mitapivat), Agios Pharmaceuticals has come up short of producing an unequivocal win in the key indication of sickle cell disease (SCD).Attempting to capture a “broad assessment” of the potential benefits of the drug across “multiple aspects of the disease,” the company ran the 52-week Rise Up study, with primary endpoints assessing hemoglobin responses and the annualized rate of sickle cell pain crises (SCPCs) compared to placebo. The study further examined five secondary endpoints, including other biomarker responses, patient fatigue and the annualized rate of hospitalizations for SCPCs.Rise Up met one primary endpoint by demonstrating an improved hemoglobin response, Agios said on Wednesday, with 40.6% of patients on the drug meeting hemoglobin response criteria, versus 2.9% on placebo. On the other primary measure, however, Pyrukynd showed a “reduction” in SCPCs but did not ultimately achieve statistical significance.On secondary endpoints, the drug showed certain improved biomarker outcomes but failed to show an average change from baseline on a patient-reported fatigue scoring system. Two other secondary measures couldn't be determined, Agios said.Agios plans to “engage with the FDA to discuss these findings and our goal of bringing this innovative medicine to patients with sickle cell disease,” Sarah Gheuens, M.D., Ph.D., chief medical officer, said in the company’s release.“The RISE UP Phase 3 results further support mitapivat’s strong anti-hemolytic profile, as demonstrated in other rare blood disease trials,” Gheuens said. “These effects can help address debilitating features of sickle cell disease that can profoundly worsen quality of life and lead to early mortality.”The company aims to submit its bid for Pyrukynd in SCD after a meeting with the FDA in the first quarter of 2026, the company said.It's tough to get a sense of the drug's outlook in SCD in the wake of the readout, but investors aren't thrilled. The company’s stock price crashed by more than 49% on Wednesday morning, reaching $22.91 by midday, down from its previous close of $45.49.For another indication of how the company is digesting the news, Agios said it's planning to take "proactive steps to reduce operating expenses" to support its financial position and support another potential launch. Pyrukynd won approval in 2022 as the first therapy indicated for hemolytic anemia in adults with PK deficiency. The company’s ambitions to grow beyond its existing patient population have so far faced ups and downs, with last year’s study of Pyrukynd in children with PK deficiency who receive regular transfusions missing its prespecified statistical criterion.Meanwhile, an FDA approval decision in another potential area of expansion, thalassemia, is expected next month after the agency extended its review by three months.Troubles in SCD trialsSickle cell disease challenges aren’t unique to Agios. Besides the 2023 FDA approvals of multimillion-dollar genetic medicines from Genetix Biotherapeutics (formerly known as bluebird bio), Vertex Pharmaceuticals and CRISPR Therapeutics, the field has seen several setbacks lately.Pfizer, for one, last year cut one of its two phase 3 studies evaluating its sickle cell asset, inclacumab and ultimately reported that the remaining study missed its primary vaso-occlusive crises (VOCs) endpoint in August. That setback came after another major stumble for Pfizer’s SCD portfolio—and the oral SCD treatment market as a whole—with the withdrawal of its oral Oxbryta.Oxbryta formed the centerpiece of Pfizer’s $5.4 billion acquisition of Global Blood Therapeutics in 2022, from which the company also got inclacumab. Upon its accelerated FDA approval in 2019, Oxbryta was touted as the first medicine to target the root cause of sickle cell disease. However, that approval was based on a phase 3 study that, like Agios', used a measure of hemoglobin response as its primary efficacy indicator.Years later, when Pfizer pulled the drug from global markets, it cited “the totality of clinical data that now indicates the overall benefit of Oxbryta no longer outweighs the risk” in SCD patients, pointing to data that suggested an “imbalance in vaso-occlusive crises and fatal events.”A February article published in the journal HemaSphere explored the drug development hurdles in SCD. According to the authors, the Oxbryta situation exemplified that although “laboratory endpoints, such as increased hemoglobin levels, are easier to achieve, it is still important to demonstrate some meaningful clinical benefit, such as reduced episodes of pain or improved quality of life.”