AGIOS PHARMACEUTICALS INC (AGIO)
Health Care · Rare disease biopharmaceuticals · NASDAQ
A rare-disease biopharmaceutical company commercializing mitapivat (AQVESME™/PYRUKYND®), a pyruvate kinase activator for hemolytic anemias including thalassemia and sickle cell disease.
What AGIOS PHARMACEUTICALS INC does
Agios is a commercial-stage biopharmaceutical company focused on rare disease treatment, particularly hemolytic anemias. The company's flagship product mitapivat (marketed as AQVESME™ in the US and PYRUKYND® in other regions) is an activator of pyruvate kinase enzymes, approved for treating anemia in thalassemia patients and PK deficiency, with applications being pursued in sickle cell disease. Agios is also developing a pipeline including tebapivat for myelodysplastic syndromes and sickle cell disease, AG-181 for phenylketonuria, and AG-236 (an siRNA licensed from Alnylam) for polycythemia vera.
Themes: ["Rare hemolytic anemias / thalassemia","Sickle cell disease","Myelodysplastic syndromes","Rare metabolic disorders / phenylketonuria","RNAi therapeutics / gene targeting"]
Fundamentals
- Price$42.79 as of 2026-07-09 close
- Market cap$2.5B as of 2026-07-10
- 1-year return+9.5% as of 2026-07-09 close
- Net margin-639.8% as of 2026-07-10
- Gross margin+90.0% as of 2026-07-10
- ROE-34.1% as of 2026-07-10
- Debt / equity0.00 as of 2026-07-10
- Revenue growth (YoY)+78.3% as of 2026-07-10
- Revenue CAGR (3y)+56.0% SEC XBRL
- Beta0.58 as of 2026-07-10
Key risks (from latest filing)
["Mitapivat commercialization execution risk across multiple international markets and rare disease indications with limited patient populations","Early-stage pipeline programs (tebapivat, AG-181, AG-236) carry clinical and regulatory development risk with unproven efficacy","Substantial net losses and negative cash flow require access to capital markets; company is unprofitable with -639.84% net margin"]
Competitors & peers
- Vertex Pharmaceuticals (VERX)
- Bluebird Bio
- Sangamo Therapeutics
- CRISPR Therapeutics
- Sesen Bio
Fundamentals: Finnhub, as of 2026-07-10. Filings: SEC EDGAR. Prices are delayed daily-close data.
Last updated 2026-07-09.
Informational only — NOT financial advice. All figures are delayed daily-close data from SEC EDGAR & Finnhub, shown with their as-of date.