Business Summary
Agios Pharmaceuticals is a commercial-stage biopharmaceutical company focused on developing and commercializing therapies for rare hematologic diseases through its expertise in cellular metabolism. The company's lead product, mitapivat (marketed as PYRUKYND and AQVESME), is a first-in-class oral pyruvate kinase activator approved for pyruvate kinase (PK) deficiency in the US, EU, and Great Britain, and received FDA approval in December 2025 for anemia in adults with alpha- or beta-thalassemia (both non-transfusion dependent and transfusion dependent). Agios is also advancing mitapivat for sickle cell disease, where Phase 3 RISE UP data showed significant hemoglobin response but missed the co-primary endpoint on sickle cell pain crises.
Products & Services
- AQVESME (mitapivat) for thalassemia: FDA approved Dec 2025 for anemia in adults with NTD and TD alpha/beta-thalassemia; only FDA-approved medicine for anemia in both forms; launched late Jan 2026; requires REMS for hepatocellular injury monitoring
- **PYRUKYND (mitapivat) for...
AI-generated summary of Item 1 (Business) from the 10-K filed Feb 12, 2026 — View filing on SEC.gov. May contain errors — refer to the filing for authoritative information.
Across SEC filings
Institutional holders · 13F
Q2 2026| Largest holders | Reported value |
|---|---|
| BlackRock Inc | $239.6M |
| FARALLON CAPITAL MANAGEMENT LLC | $219.6M |
| STATE STREET CORPORATION | $131.1M |
| Lynx1 Capital Management LP | $126.5M |
| D E Shaw & Co Inc | $115.3M |
Annual results · 10-K
Fiscal 2025- Revenue
- $54.0M
- Net loss
- -$412.8M
As reported in the fiscal 2025 annual report.
Funds & ETFs · N-PORT
233 funds| Fund | Value | % of fund |
|---|---|---|
| State Street(R) SPDR(R) S&P(R) Biotech ETF XBI | $74.4M | 0.69% |
| VANGUARD TOTAL STOCK MARKET INDEX FUND VTI | $70.1M | < 0.01% |
| iShares Russell 2000 ETF IWM | $57.1M | 0.07% |
| Nomura Small Cap Core Fund | $57.1M | 1.09% |
| VANGUARD SMALL-CAP INDEX FUND VB | $50.6M | 0.03% |
| VANGUARD SMALL-CAP GROWTH INDEX FUND VBK | $29.3M | 0.06% |
| VANGUARD EXTENDED MARKET INDEX FUND | $25.4M | 0.03% |
| Fidelity Small Cap Index Fund | $19.4M | 0.06% |
| iShares Russell 2000 Value ETF IWN | $19.3M | 0.13% |
| Strategic Advisers U.S. Total Stock Fund | $18.2M | 0.02% |
233 funds reported holding $689.6M in total on their latest public Form N-PORT reports, the most recent dated August 2026. Security record
Insider trades · Form 4
Latest filings
- Sarah Gheuens 2026-10-28 Sell
- Sarah Gheuens 2026-10-23 Sell
- Sarah Gheuens 2026-08-06 Sell
5%+ holders · 13D/G
7 filers| Filer | % of class | Filed |
|---|---|---|
| FARALLON CAPITAL MANAGEMENT LLC 13G | 9.9% | 2026-08-03 |
| D E SHAW & CO L P 13G | 5.1% | 2026-07-02 |
| Lynx1 Capital Management LP 13G | 5.1% | 2026-05-06 |
| VANGUARD CAPITAL MANAGEMENT LLC 13G | 5.3% | 2026-04-29 |
| STATE STREET CORPORATION 13G | 5.3% | 2026-02-09 |
| Farallon Capital Partners L P 13G | 8.8% | 2026-02-09 |
| BlackRock Inc 13G | 7.8% | 2025-07-17 |
Latest Schedule 13D or 13G from each filer; stakes a filer has reported closed are left out.
Federal lobbying · LD-2
Securities
| CUSIP | Security | Type |
|---|---|---|
| 00847X104 | AGIO AGIOS PHARMACEUTICALS INC | Common stock |