Welcome to our dedicated page for Agios Pharmaceuticals news (Ticker: AGIO), a resource for investors and traders seeking the latest updates and insights on Agios Pharmaceuticals stock.
Agios Pharmaceuticals Inc. (AGIO) is a leader in developing therapies for rare hematologic and genetic disorders through its groundbreaking work in cellular metabolism. This page provides investors and industry professionals with timely updates on AGIO’s clinical advancements, regulatory milestones, and strategic initiatives.
Discover official press releases covering AGIO’s progress in treating conditions like pyruvate kinase deficiency, thalassemia, and sickle cell disease. The curated news includes updates on clinical trials, FDA communications, research collaborations, and pipeline expansions. Each update is sourced to ensure accuracy and relevance for informed decision-making.
Key areas of coverage include novel therapeutic developments, regulatory submissions, and strategic partnerships that underscore AGIO’s role in advancing precision medicine. Bookmark this page to stay informed about the company’s contributions to transforming rare disease treatment through metabolic science.
Agios (Nasdaq: AGIO) will present new clinical and preclinical mitapivat data at the 67th ASH Annual Meeting, Dec 6–9, 2025 in Orlando.
Key highlights include a subgroup from ENERGIZE-T showing 77.8% (7/9) transfusion-reduction response in alpha-thalassemia versus 0% (0/3) placebo; long-term transfusion-free mean duration of 30.5 weeks (max 84.3 weeks) among 17 patients; and Phase 3 ACTIVATE-Kids meeting its primary hemoglobin endpoint with safety consistent with adult data. Ten Agios-led or collaborator presentations will be shared, plus advisory-council research on patient knowledge and fatigue impacts.
Agios (Nasdaq: AGIO) reported third quarter 2025 results and program updates on Oct 30, 2025. PYRUKYND net product revenue was $12.9M in Q3 2025, up 44% year‑over‑year. The FDA set a PDUFA goal date of Dec 7, 2025 for the PYRUKYND sNDA in thalassemia after requesting a REMS; CHMP adopted a positive opinion and an EC decision is expected by early 2026. Saudi Arabia approved PYRUKYND for thalassemia and commercial launch is underway there.
Topline results from the RISE UP Phase 3 sickle cell trial are expected by year‑end 2025, with a potential U.S. launch in 2026. The Phase 2b tebapivat LR‑MDS trial completed enrollment; topline data expected in early 2026. Cash, cash equivalents and marketable securities were $1.3B as of Sept 30, 2025.
Avanzanite Bioscience reports that partner Agios (NASDAQ: AGIO) received a positive CHMP opinion for PYRUKYND (mitapivat) as a treatment for anemia in adults with transfusion-dependent and non-transfusion-dependent alpha- or beta-thalassemia.
Avanzanite entered an exclusive European commercialization agreement with Agios in June 2025 for PYRUKYND across the EEA, UK, and Switzerland. The CHMP opinion is based on Phase 3 ENERGIZE and ENERGIZE-T trial results. The European Commission will review the opinion, with a final decision expected by early 2026. PYRUKYND is already approved in Europe for adults with PK deficiency; thalassemia would be its second EU indication if authorized.
Agios (Nasdaq: AGIO) announced that the EMA CHMP adopted a positive opinion for PYRUKYND (mitapivat) to treat anemia in adults with transfusion‑dependent and non‑transfusion‑dependent alpha‑ or beta‑thalassemia, based on Phase 3 ENERGIZE‑T and ENERGIZE trials.
The European Commission will now review the opinion, with a final decision expected by early 2026. Agios signed an exclusive commercialization agreement with Avanzanite in June 2025 for the EEA, UK, and Switzerland and will support a potential European launch pending approval. PYRUKYND is approved in Saudi Arabia for thalassemia and has ongoing U.S. review with a PDUFA goal date of December 7, 2025. PYRUKYND is also approved for PK deficiency in the U.S. and Europe.
Agios (Nasdaq: AGIO) will report its third quarter 2025 financial results and business highlights on Thursday, October 30, 2025 at 8:00 a.m. ET.
The company will host a conference call and live webcast accessible from the Investors > Events & Presentations section of its website. A replay will be available on the company website approximately two hours after the event.
Agios Pharmaceuticals (Nasdaq: AGIO) announced that the FDA has extended the PDUFA goal date for PYRUKYND® (mitapivat) by three months, from September 7, 2025, to December 7, 2025. The extension follows Agios's submission of a proposed Risk Evaluation and Mitigation Strategy (REMS) to address hepatocellular injury risk.
The REMS submission is considered a major amendment to the supplemental New Drug Application (sNDA) for PYRUKYND®, which aims to treat adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia. The extension is not related to new safety or efficacy data. The sNDA is supported by results from the ENERGIZE and ENERGIZE-T Phase 3 trials.
Agios Pharmaceuticals (Nasdaq: AGIO) has achieved a significant milestone as its drug PYRUKYND® (mitapivat) received approval from the Saudi Food and Drug Authority (SFDA) for treating adult patients with thalassemia. This marks the first regulatory approval for PYRUKYND in thalassemia globally.
The approval, evaluated under SFDA's Breakthrough Medicines Program, is based on results from the ENERGIZE and ENERGIZE-T Phase 3 trials. The drug is approved for both non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia patients. Agios has partnered with NewBridge Pharmaceuticals to manage PYRUKYND commercialization in the Gulf Region.
Additional regulatory applications for PYRUKYND in thalassemia are under review in the U.S. (PDUFA date: September 7, 2025), United Arab Emirates, and European Union.
Agios Pharmaceuticals (NASDAQ: AGIO) reported Q2 2025 financial results, highlighting $12.5 million in PYRUKYND net revenues, a significant increase from $8.6 million in Q2 2024. The company maintains a strong financial position with $1.3 billion in cash and equivalents.
Key developments include the pending FDA review of PYRUKYND's sNDA for thalassemia with a PDUFA date of September 7, 2025, and anticipated topline results from the RISE UP Phase 3 trial in sickle cell disease by year-end. The company reported a net loss of $112.0 million for Q2 2025, compared to $96.1 million in Q2 2024, with increased R&D expenses of $91.9 million and SG&A expenses of $45.9 million.
Notable operational progress includes 248 unique patients completing prescription enrollment forms and 142 patients currently on PYRUKYND therapy. The company also secured a European distribution agreement with Avanzanite Bioscience and received IND clearance for AG-236 in polycythemia vera treatment.
Agios Pharmaceuticals (Nasdaq: AGIO), a company focused on cellular metabolism and PK activation therapies for rare diseases, will host its second quarter 2025 financial results conference call and webcast on July 31, 2025, at 8:00 a.m. ET.
The presentation will be available through the company's website investor section, with a replay accessible approximately two hours after the event.
Agios Pharmaceuticals (Nasdaq: AGIO), a leader in cellular metabolism and PK activation therapies, has appointed Dr. Jay Backstrom to its Board of Directors. Dr. Backstrom, who will serve on the Science and Technology Committee, brings extensive experience in advancing clinical programs and regulatory approvals, particularly in rare diseases.
Dr. Backstrom's recent roles include serving as President and CEO of Scholar Rock, where he led the company's spinal muscular atrophy program through Phase 3 development. His previous positions include Executive VP of Research and Development at Acceleron Pharma and Chief Medical Officer at Celgene Corporation, where he contributed to regulatory approvals in beta thalassemia and myelodysplastic syndromes.
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