Agios Pharmaceuticals, Inc. reports developments as a commercial-stage biopharmaceutical company focused on rare diseases and hematology. Its recurring updates center on mitapivat, an oral pyruvate kinase activator marketed as PYRUKYND® and AQVESME™, including product revenue, U.S. and ex-U.S. commercialization, thalassemia approvals, and activity in pyruvate kinase deficiency, sickle cell disease, and other hemolytic anemias.
Company news also covers FDA interactions, supplemental application activity, conference presentations, and pipeline progress for tebapivat, a next-generation PK activator, and other rare disease programs. International commercialization themes include regulatory approvals and distribution activity in Gulf Cooperation Council markets through NewBridge Pharmaceuticals.
Agios (Nasdaq: AGIO) reported Q4 2025 results and business updates on Feb 12, 2026. Key highlights include AQVESME FDA approval and U.S. availability for thalassemia, PYRUKYND Q4 net revenue $20.0M ($16.0M U.S., $4.0M ex-U.S.), full-year PYRUKYND revenue $54.0M, and $1.2B cash as of Dec 31, 2025.
The company recorded a $108.0M Q4 net loss, plans a pre-sNDA FDA meeting for mitapivat in sickle cell disease in Q1 2026, and expects tebapivat Phase 2 topline data in H2 2026.
Agios (Nasdaq: AGIO) will host a conference call and live webcast on Thursday, February 12, 2026 at 8:00 a.m. ET to report fourth quarter and full year 2025 financial results and business highlights. The live webcast will be available on the company’s Investors website under the "Events & Presentations" tab. A replay will be posted on the company website approximately two hours after the event.
Agios (Nasdaq: AGIO) outlined 2026 strategic priorities focused on commercializing AQVESME (mitapivat) in thalassemia and advancing a pipeline of rare-disease programs.
Key points: AQVESME received U.S. FDA approval in December 2025 for anemia in adults with alpha- or beta-thalassemia and is expected to be available in the U.S. in late January 2026 after REMS implementation. Agios plans a pre-sNDA FDA meeting for mitapivat in sickle cell disease in Q1 2026 with a planned U.S. regulatory submission to follow. Multiple topline readouts are expected across 2026 for tebapivat, AG-236, and AG-181. The company projects a clear path to profitability and cites potential for over $1 billion in peak global sales from its PK activation franchise.
Agios (Nasdaq: AGIO) announced its management will present at the 44th Annual J.P. Morgan Healthcare Conference on January 14, 2026 at 8:15 a.m. PST / 11:15 a.m. EST. The presentation will be available via a live webcast on the company’s Investors site under “Events & Presentations.” A replay will be archived on the company website for at least two weeks after the presentation.
Agios (Nasdaq: AGIO) announced U.S. FDA approval of AQVESME™ (mitapivat) for treatment of anemia in adults with alpha- or beta-thalassemia, covering both non-transfusion-dependent and transfusion-dependent patients.
The approval is based on the randomized ENERGIZE and ENERGIZE-T Phase 3 trials (total 452 patients) that met primary and key secondary endpoints, showing improvements in hemoglobin, fatigue, and transfusion burden. AQVESME will be available in the U.S. in late January 2026 after implementation of an FDA REMS requiring liver tests and education. Five patients experienced adverse reactions suggestive of hepatocellular injury, with two hospitalizations; liver tests improved after discontinuation.
Agios (Nasdaq: AGIO) announced that the U.S. FDA has not issued a regulatory decision on the supplemental NDA for mitapivat in adult non‑transfusion‑dependent and transfusion‑dependent alpha‑ or beta‑thalassemia; the application remains under active review.
The FDA's PDUFA goal date was December 7, 2025. Agios said the agency has not requested, and the company has not submitted, any new efficacy or safety data, and that the companies are collaborating to finalize labeling and REMS materials. The FDA has not provided a timeline for its decision and Agios continues to work expeditiously to conclude the review.
Agios (Nasdaq: AGIO) reported topline 52-week RISE UP Phase 3 results for mitapivat in patients ≥16 with sickle cell disease. The trial met the primary hemoglobin response endpoint: 40.6% responders on mitapivat vs 2.9% on placebo (2-sided p<0.0001). Statistically significant improvements were also seen in average hemoglobin concentration (+7.69 g/L vs +0.26 g/L) and indirect bilirubin (-16.03 µmol/L vs +0.88 µmol/L) (both p<0.0001). The trial did not achieve statistical significance for annualized SCPC rate (2.62 vs 3.05, p=0.1213) or PROMIS Fatigue (p=0.7112). Safety was consistent with prior studies. Agios plans FDA engagement and intends an sNDA submission after a Q1 2026 pre-sNDA meeting.
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.