Trial met primary endpoint of hemoglobin response and key secondary endpoints of change from baseline in hemoglobin concentration and indirect bilirubin Trial showed trend favoring mitapivat but did not meet statistical significance in primary endpoint of annualized rate of SCPCs (pain crises), and the key secondary endpoint of change from baseline in PROMIS Fatigue was not met Patients in the mitapivat arm who achieved hemoglobin response had clinically meaningful benefits in SCPC-related endpoints and PRO ...

CAMBRIDGE, Mass., Nov. 03, 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 new data on mitapivat, an oral pyruvate kinase (PK) activator, will be featured in oral and poster presentations during the 67th American Society of Hematology (ASH) Annual Meeting and Exposition (ASH 2025) in Orlando, Florida, December 6-9, 2025. “This year’s presentations ...

Financial Performance - Q3 2025 net revenues reached $12.9 million, a 44% increase compared to $9.0 million in Q3 2024 [12] - Cash on hand remains strong at $1.3 billion, intended for investment in U S launches and pipeline development [10, 12, 15] - Net loss for Q3 2025 was $103.4 million, compared to a net income of $947.9 million in Q3 2024 [15] PYRUKYND® Commercial Progress - PYRUKYND net sales were $12.9 million in Q3 2025, compared to $12.5 million in Q2 2025 and $9.0 million in Q3 2024 [15, 20] - In the U S, 262 unique PK deficiency patients have completed prescription enrollment forms since launch [20] - There are 149 net patients on PYRUKYND treatment in the U S, including new prescriptions and treatment continuations [20] - PYRUKYND has been prescribed by 227 unique prescribers in the U S [20] Regulatory and Pipeline Advancements - PYRUKYND received SFDA approval in Saudi Arabia for thalassemia [12, 27, 28] - A positive CHMP opinion in Europe was received for PYRUKYND in thalassemia [12, 27, 28] - Enrollment is complete in the tebapivat Phase 2b trial for lower-risk MDS, with top-line data anticipated in early 2026 [12, 27]

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 ...

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].

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].

Financial Performance - PYRUKYND net revenues reached $12.5 million in Q2 2025, a 44% increase compared to $8.7 million in Q1 2025 and a 45% increase compared to $8.6 million in Q2 2024[10] - The company maintains a strong financial position with $1.3 billion in cash on hand[9, 10, 14] - Net loss for Q2 2025 was $112.0 million, compared to a net loss of $96.1 million for Q2 2024[14] Pipeline Development - First patient dosed in Phase 2 Sickle Cell Disease trial for AG-236 after receiving IND clearance[11] - RISE UP Phase 3 trial in sickle cell disease is expected to have topline data by the end of 2025[37] - Tebapivat Phase 2b trial for LR-MDS is ongoing, with topline results expected in early 2026[9, 45] Commercialization - 248 unique PK deficiency patients completed prescription enrollment forms since launch in the U S [21] - 142 patients are on treatment in the U S , including new prescriptions and treatment continuations, with 215 unique prescribers[22] - The company anticipates a potential U S launch of PYRUKYND for thalassemia in Q3 2025, with a PDUFA goal date of September 7, 2025[11, 23, 24] Strategic Partnerships - Announced a commercialization and distribution partnership with Avanzanite Bioscience in Europe[10] - A commercialization and distribution agreement is in place with NewBridge for the GCC region, with anticipated first regulatory approval in the coming months[30]

Business Overview - Agios views PYRUKYND® as having a multi-billion dollar market opportunity [9] - Agios aims to maximize the potential of the PYRUKYND® franchise and progress key pipeline programs in 2025 [11] - Agios anticipates several clinical and regulatory catalysts in 2025, including a potential FDA approval for PYRUKYND® in thalassemia with a PDUFA goal date of September 7, 2025 [13] Clinical Development - Approximately 67% of thalassemia patients are non-transfusion dependent, representing about 4,000 adults in the U S [19] - Approximately 33% of thalassemia patients are transfusion dependent, representing about 2,000 adults in the U S [19] - Agios's PYRUKYND® has the potential to be the first and only approved therapy for all subtypes of thalassemia [23] - The Phase 3 RISE UP study of PYRUKYND® in sickle cell disease completed enrollment in October 2024, with topline data expected in late 2025 [31] - Tebapivat's Phase 2b study in lower-risk MDS expects patient enrollment completion in late 2025 [34, 41] Commercial Performance & Financials - PYRUKYND® net revenue was $8 7 million for Q1 2025, compared to $8 2 million in Q1 2024 [68] - Agios had $1 4 billion in cash, cash equivalents, and marketable securities as of March 31, 2025 [68, 74]