Agios Pharmaceuticals (AGIO)
Commercializes mitapivat for rare blood disorders and develops additional hematology therapies.
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Sells mitapivat as PYRUKYND for adult PK deficiency and as AQVESME for U.S. adults with thalassemia, while developing therapies for sickle cell disease and other rare blood disorders. Revenue comes from U.S. specialty-drug sales and overseas distributors, with the business managed as one rare-disease segment.
Commercializes the oral PK activator mitapivat as PYRUKYND for adult PK deficiency and as AQVESME for adult thalassemia in the United States. PYRUKYND also reaches approved European and Gulf markets through distribution partners.
Builds rare-disease medicines around hematology and cellular metabolism. The pipeline extends mitapivat into sickle cell disease and includes AG-236 for polycythemia vera, AG-181 for phenylketonuria, and licensed SYK inhibitor cevidoplenib for immune thrombocytopenia.
Execution combines U.S. specialty-drug commercialization with clinical and regulatory development in a single operating segment. Product uptake depends on physician adoption, coverage, and reimbursement; AQVESME requires a safety-monitoring program. Commercial supply relies on third-party manufacturers, while access outside the United States relies on distribution partners.
Company facts
Target rangeAs of Sep 2, 2026
Latest analyst notes
| Date | Firm | Action | Rating | Prior target | Target |
|---|---|---|---|---|---|
| Jul 30, 2026 | H.C. Wainwright | Target | $52 | $55 | |
| Jul 21, 2026 | H.C. Wainwright | Reiterate | Buy | $54 | $52 |
| Jul 7, 2026 | Truist Financial | Target | $36 | $41 | |
| Jul 7, 2026 | RBC Capital | Reiterate | Sector Perform | $53 | $32 |
| Jun 15, 2026 | H.C. Wainwright | Target | $50 | $54 |
EPSReportedEstimate
RevenueReportedEstimate
Balance sheet, Jun 30, 2026Cash & short-term investments $617.6M · Total debt $31.2M · Total assets $1.11B · Shareholders' equity $1.03B
- Revenue
- $44.7M
- Operating income
- −$110.6M
- Net income
- −$100.7M
No dividends on record.
| Institutions251 holders · as of Jun 30, 2026 · largest filers plus last quarter's tail | ≈ 110.61% | 66.04M shares | |
| Insiders16 holders · as of Aug 6, 2026 | 1.11% | 659.8K shares | |
| Strategic holders1 holder · as of Nov 14, 2019 | 11.93% | 7.12M shares | |
| Adds to | 123.65% |
59.70M company shares as of Sep 3, 2026 · institutions as of Jun 30, 2026 · holders as of their latest filing
This quarter's institutional total failed a plausibility check; shown: the largest filers plus last quarter's remainder.
These groups come from separate filings that can overlap, so they add to 123.65%; nothing is left for Other.
Top shareholders13F · 13D/G · Form 4
| Holder | Stake | Shares | Evidence |
|---|---|---|---|
Celgene CorporationStrategic | 11.93% | 7.12M | 13D/Afiled Nov 14, 2019 ↗ |
BlackRock, Inc.Institution | 10.81% | 6.46M | 13Fas of Jun 30, 2026 |
Farallon Capital Management LLCInstitution | 9.91% | 5.92M | 13Fas of Jun 30, 2026 |
Capital International InvestorsInstitution | 7.79% | 4.65M | 13D/Gfiled Feb 14, 2020 ↗ |
State Street CorpInstitution | 5.92% | 3.53M | 13Fas of Jun 30, 2026 |
| See all 25 → | |||
13F — a manager's quarterly holdings · 13D/13G — an ownership statement filed at 5% or more
Insider tradesForm 4
| Date | Insider | Role | Shares | Avg price |
|---|---|---|---|---|
| Aug 4, 2026 | Sarah Gheuens | officer: Chief Medical Officer | −30,000 | $30.48 |
| Jul 1, 2026 | Sarah Gheuens | officer: Chief Medical Officer | −4,007 | $37.56 |
| Apr 2, 2026 | Brian Goff | director, officer: Chief Executive Officer | −19,068 | $34.71 |
| Apr 2, 2026 | Krishnan Viswanadhan | officer: Chief Corp Dev & Strategy | −2,959 | $34.71 |
| Apr 2, 2026 | Tsveta Milanova | officer: Chief Commercial Officer | −3,262 | $34.71 |
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Compare
| Company | Price | Change | |||||
|---|---|---|---|---|---|---|---|
| Agios Pharmaceuticals | $34.14 | −0.20% | $2.04B | — | — | 20.7 | +259.3% |
| Vertex Pharmaceuticals | $555.40 | −0.23% | $141.31B | 32.4 | 28.6 | 11.2 | +12.5% |
| Bristol-Myers Squibb | $67.64 | −0.02% | $138.21B | 14.9 | 10.6 | 2.8 | +5.7% |
| Novo Nordisk (ADR) | $47.46 | +1.51% | $207.69B | 11.5 | 18.9 | 4.1 | +0.0% |
| CRISPR Therapeutics | $56.94 | +0.08% | $5.49B | — | — | 409.9 | +1,041.4% |
| Beam Therapeutics | $29.57 | +0.89% | $3.01B | — | — | 19.3 | −94.2% |
| Median | 14.9 | 18.9 | 11.2 | +5.7% |