Agios Pharmaceuticals Inc Stock Price, News & Analysis

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.

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.