Core Insights - Agios Pharmaceuticals reported financial results for Q3 2025, highlighting a focus on key milestones for its drug PYRUKYND in treating thalassemia and sickle cell disease [1][2]. Financial Performance - PYRUKYND generated $12.9 million in net revenues for Q3 2025, a 44% increase from $9.0 million in Q3 2024 and a 3% increase from $12.5 million in Q2 2025 [5][6]. - The company reported a net loss of $103.4 million for Q3 2025, compared to a net income of $947.9 million in Q3 2024, which was primarily due to a milestone payment and sale of royalty rights recorded in the previous year [7][17]. Product Development and Regulatory Updates - The PDUFA goal date for PYRUKYND's supplemental New Drug Application (sNDA) for thalassemia has been extended to December 7, 2025, due to a request for a Risk Evaluation and Mitigation Strategy (REMS) [6][11]. - The European Medicines Agency's CHMP has adopted a positive opinion for PYRUKYND in thalassemia, with a final decision expected by early 2026 [5][6]. - The RISE UP Phase 3 trial for sickle cell disease is expected to provide topline results by year-end 2025, potentially leading to a U.S. commercial launch in 2026 [5][11]. Research and Development - The Phase 2b trial for tebapivat in lower-risk myelodysplastic syndromes (MDS) has completed enrollment, with topline results anticipated in early 2026 [5][11]. - Research and Development expenses for Q3 2025 were $86.8 million, an increase of $14.3 million compared to Q3 2024, driven by higher clinical trial costs [11][17]. Cash Position - As of September 30, 2025, Agios had $1.3 billion in cash, cash equivalents, and marketable securities, down from $1.5 billion at the end of 2024 [5][11].

AMSTERDAM--(BUSINESS WIRE)-- #biopharma--Avanzanite's Partner Agios Pharmaceuticals Announces Regulatory Update. ...

CAMBRIDGE, Mass., Oct. 17, 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 Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion for the new indication for PYRUKYND® (mitapivat), an oral pyruvate kinase (PK) activator, in adults for the treatment of anemia associated with tr ...

CAMBRIDGE, Mass., Oct. 16, 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 the company will host a conference call and live webcast on Thursday, October 30, 2025, at 8:00 a.m. ET to report its third quarter 2025 financial results and business highlights. The live webcast will be accessible on the Investors section of the company’s website (www.agios.co ...

Core Viewpoint - The Schall Law Firm is investigating Agios Pharmaceuticals, Inc. for potential violations of securities laws related to misleading statements and undisclosed information affecting investors [2]. Group 1: Investigation Details - The investigation centers on whether Agios issued false or misleading statements or failed to disclose critical information to investors [2]. - On September 4, 2025, Agios announced that the FDA extended the PDUFA goal date for the supplemental New Drug Application of PYRUKYND® by three months to December 7, 2025 [2]. - Following this announcement, Agios shares dropped by over 11% the next day [2]. Group 2: Legal Representation - The Schall Law Firm is representing investors globally and specializes in securities class action lawsuits and shareholder rights litigation [3]. - Shareholders who suffered losses are encouraged to contact the firm for a free discussion of their rights [3].

Group 1 - Agios Pharmaceuticals, Inc. is currently under investigation for potential fraud, providing an opportunity for investors to join the legal proceedings with the Schall Law Firm [1] - The investigation may involve allegations of misleading statements or omissions that could have affected the stock price and investor decisions [1] - Investors are encouraged to consider their legal options in light of the ongoing investigation [1]

Core Viewpoint - Pomerantz LLP is investigating potential securities fraud or unlawful business practices by Agios Pharmaceuticals and its officers or directors, following a significant stock price drop due to a delayed FDA review of a key drug [1][3]. Group 1: Company Overview - Agios Pharmaceuticals, Inc. is under investigation for possible securities fraud related to its business practices [1]. - The company’s stock price fell by $4.48, or 11.03%, closing at $36.13 per share on September 4, 2025, after the announcement of a three-month extension for the FDA review of its drug PYRUKYND® [3]. Group 2: Regulatory Developments - The U.S. FDA extended the Prescription Drug User Fee Act (PDUFA) goal date for the supplemental New Drug Application (sNDA) of PYRUKYND® to December 7, 2025, impacting investor sentiment and stock performance [3].

Core Viewpoint - Pomerantz LLP is investigating potential securities fraud or unlawful business practices by Agios Pharmaceuticals following a delay in FDA review of its drug PYRUKYND® [1][2]. Group 1: Company Overview - Agios Pharmaceuticals, Inc. is under investigation for possible securities fraud related to its business practices [1]. - The company’s stock price dropped by $4.48, or 11.03%, closing at $36.13 per share after the announcement of the FDA's extension of the review date for its drug [2]. Group 2: Regulatory Update - On September 4, 2025, Agios announced that the FDA extended the PDUFA goal date for the supplemental New Drug Application of PYRUKYND® by three months to December 7, 2025 [2].

Company Overview - Agios Pharmaceuticals (NASDAQ: AGIO) has experienced a stock price increase of approximately 29% since April, following Liberation Day [1] - The company is currently facing uncertainty with the upcoming PDUFA decision date, which was originally set for September 7 but has been postponed [1] Analyst Background - The analyst has a Master's degree in Cell Biology and extensive experience in drug discovery, including cell culture, assay development, and therapeutic research [1] - The analyst has been active in the investment space for five years, focusing on biotechnology companies that are innovating through unique mechanisms of action and first-in-class therapies [1] - The approach combines scientific expertise with financial and market analysis to deliver technically sound and investment-driven research [1] Investment Focus - The analyst plans to cover a range of biotechnology companies at various stages of development, from early clinical pipelines to commercial-stage biotechs [1] - Emphasis will be placed on evaluating the science behind drug candidates, the competitive landscape, clinical trial design, and potential market opportunities, while also considering financial fundamentals and valuation [1]

Core Viewpoint - Shares of Agios Pharmaceuticals (AGIO) fell 11% after the FDA extended the review timeline for the supplemental new drug application (sNDA) for Pyrukynd in thalassemia by three months, with a new decision date set for December 7, 2025 [1][7]. Company Developments - The sNDA for Pyrukynd is based on data from two phase III studies, ENERGIZE and ENERGIZE-T, aiming for approval in treating non-transfusion-dependent and transfusion-dependent thalassemia in adults [2]. - Agios submitted a proposed Risk Evaluation and Mitigation Strategy (REMS) to address liver injury risks, which led to the three-month extension of the review timeline [3]. - The FDA had previously expected a decision on September 7, 2025, and the delay may impact the commercial launch timeline, which was initially anticipated before the end of 2025 [8]. Financial Performance - Year-to-date, Agios shares have increased by 10%, outperforming the industry average rise of 4.1% [4]. - Pyrukynd generated sales of $21.2 million in the first half of 2025, marking a 26% year-over-year increase [9]. Ongoing Research - Agios is also conducting late-stage studies, ACTIVATE-Kids and ACTIVATE-KidsT, to evaluate Pyrukynd for treating PK deficiency in pediatric patients [10].