AGIOS PHARMACEUTICALS, INC.
AGIO evidence brief
What we can confirm
- BioSniper Score:
- 7.1 / 10
- Market Cap:
- $2.00B
- Lead Asset:
- AG-946 (Phase 2)
What it means
What to watch
Prepared research question
What matters most for AGIO—catalysts, financial risk and supporting evidence?
Traceable citations · Unknowns marked
BioSniper AI · AGIO
Agios Pharmaceuticals, Inc. (AGIO) is a commercial-stage biopharmaceutical company focused on developing and delivering innovative medicines for patients with rare diseases, with a core franchise built around the pyruvate kinase activator mitapivat (PYRUKYND®).
Key risk: Operating losses continue
BioSniper Score
Based on 5 of 6 signals
Financial data as of Jun 30, 2026.
Tracking 3 pipeline candidates · 34 clinical trials · 2 catalysts for AGIOS PHARMACEUTICALS, INC..
Agios Pharmaceuticals, Inc. (AGIO) is a commercial-stage biopharmaceutical company focused on developing and delivering innovative medicines for patients with rare diseases, with a core franchise built around the pyruvate kinase activator mitapivat (PYRUKYND®).
- Cash & equivalents of $99.6M, total revenue of $44.7M, and net income of -$100.7M as of 2026-06-30 [sec].
- Mitapivat (PYRUKYND®) is FDA-approved for hemolytic anemia in adults with PK deficiency and is being evaluated for thalassemia, sickle cell disease (SCD), and pediatric PK deficiency [pipeline].
- Agios submitted an sNDA to the FDA for U.S. accelerated approval of mitapivat in sickle cell disease in May 2026 [news · published 2026-05-12].
- A Phase 2/3 study of AG-236 in subjects with polycythemia vera is set to start on 2026-11-01 [catalyst].
- The company is expanding mitapivat into multiple rare disease indications, including PK deficiency, thalassemia, and SCD, to build a rare disease franchise [news · published 2026-08-20].
What works
- Mitapivat is already FDA-approved (PYRUKYND®) for PK deficiency, providing a commercial revenue stream and a foundation for label expansion [pipeline].
- Agios has a clear regulatory pathway for mitapivat in sickle cell disease, having submitted an sNDA for accelerated approval after a pre-sNDA meeting with the FDA [news · published 2026-03-31].
- The company is advancing a broad pipeline of pyruvate kinase activators (mitapivat, AG-946, tebapivat) across multiple rare disease indications, creating potential for a diversified rare disease franchise [pipeline].
What to weigh
- The company reported a net loss of -$100.7M for the period ending 2026-06-30, indicating ongoing operating losses despite commercial revenue [sec].
- Mitapivat faces a competitive landscape with at least 18 competitors in sickle cell disease and myelodysplastic syndromes, including programs from Novartis and Rigel [pipeline].
- The company's growth story is heavily dependent on mitapivat's commercial expansion, making it vulnerable to pipeline setbacks or regulatory delays in new indications [news · published 2026-10-02].
From the filings
Quoted directly from source documents.
Mitapivat is FDA-approved for hemolytic anemia in adults with PK deficiency.
“PYRUKYND® Generic Name: mitapivat Indication: Hemolytic anemia in adults with PK deficiency; being evaluated for thalassemia, sickle cell disease (SCD), and pediatric patients with PK deficiency Phase: Approved”
Agios submitted an sNDA for accelerated approval of mitapivat in sickle cell disease.
“Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a commercial-stage biopharmaceutical company focused on delivering innovative medicines for patients with rare diseases, today announced the submission of its sup”
News · May 12, 2026
Agios is expanding mitapivat into multiple rare disease indications.
“Agios is leveraging mitapivat across PK deficiency, thalassemia, and sickle cell disease to build a rare disease franchise.”
News · August 20, 2026
Catalyst timeline
- 2026-11-01 · Phase3_Start: A Phase 2/3, Double-blind, Randomized, Placebo-Controlled, Multicenter, Efficacy, Safety, and Pharmacokinetics Study of AG-236 in Subjects With Polycythemia Vera — Initiation of a Phase 2/3 study for AG-236 in polycythemia vera, a new indication for the company.
- 2041-07-31 · Patent_Expiry: NDA216196 — Patent expiry for mitapivat sulfate (PYRUKYND®), far in the future but a long-term exclusivity consideration.
Risks & what to watch (3)›
Key risks
- Operating losses continue — Net loss of -$100.7M as of 2026-06-30, indicating ongoing cash burn despite commercial revenue [sec].
- Heavy reliance on mitapivat — Growth story hinges on mitapivat's commercial expansion; pipeline setbacks could significantly impact the company [news · published 2026-10-02].
- Competitive pressure in SCD — At least 18 competitors in sickle cell disease and MDS, including Novartis and Rigel programs [pipeline].
What to watch
- FDA decision on the mitapivat sNDA for accelerated approval in sickle cell disease, submitted May 2026 [news · published 2026-05-12].
- Initiation of the Phase 2/3 study of AG-236 in polycythemia vera, expected to start 2026-11-01 [catalyst].
- Commercial uptake of mitapivat (PYRUKYND®) in PK deficiency and potential label expansion into thalassemia and pediatric patients [pipeline].
- Financial results for Q2 2026, to be reported on July 30, 2026 [catalyst].
Sources: Catalysts · News · Pipeline · Generated October 11, 2026 · How we verify sources →
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Evidence & data
Score breakdownBased on 5 of 6 signals · derived from public data
Cash runway data unavailable
D/E 0.08 · 0% dilution
Lead: Approved
3 candidates
2 upcoming, next in ~18d
0 buys · 2 sells (180d)
Catalysts
Pipeline
Regulatory
Financials
Institutional Holdings
Source: SEC Form 13F · as of Mar 2026Institutional holders
232
Total value held
$2.00B
| Holder | Shares | Value |
|---|---|---|
| blackrock, inc. | 5,754,957 | $194.7M |
| farallon capital management llc | 5,684,000 | $192.3M |
| state street corp | 3,364,879 | $113.7M |
| armistice capital, llc | 2,716,000 | $91.9M |
| d. e. shaw & co., inc. | 2,681,169 | $90.7M |
| vanguard capital management llc | 2,632,316 | $89.1M |
| lynx1 capital management lp | 2,611,568 | $88.3M |
| vanguard portfolio management llc | 2,611,035 | $88.3M |
| erste asset management gmbh | 2,704,900 | $80.1M |
| bnp paribas asset management holding s.a. | 1,580,840 | $53.5M |
Showing top 10 of 232 institutional holders of AGIOS PHARMACEUTICALS, INC.. Open the full 13F history after sign-in.
Short Interest
as of Apr 19, 2026- % of float
- 11.4%
- Days to cover
- 7.2
- Shares short
- 6,606,016
- Daily short volume
- -
Short percent of float and days-to-cover reflect reported short positioning. Daily short volume is the exchange-reported short share of a single day's trading volume and is noisier. See the most-shorted biotech ranking →
AGIO short interestGovernance
Frequently asked questions
What is the AI investment thesis for AGIOS PHARMACEUTICALS, INC.?
Agios Pharmaceuticals, Inc. (AGIO) is a commercial-stage biopharmaceutical company focused on developing and delivering innovative medicines for patients with rare diseases, with a core franchise built around the pyruvate kinase activator mitapivat (PYRUKYND®).
What is AGIOS PHARMACEUTICALS, INC.'s lead drug candidate?
AGIOS PHARMACEUTICALS, INC.'s most advanced tracked candidate is AG-946 (Phase 2) for Potential treatment of lower-risk myelodysplastic syndrome (LR MDS) and hemolytic anemias.
What is AGIOS PHARMACEUTICALS, INC.'s next catalyst?
AGIOS PHARMACEUTICALS, INC.'s next tracked catalyst is a Phase3_Start event — "Phase3_Start: A Phase 2/3, Double-blind, Randomized, Placebo-Controlled, Multicenter, Efficacy, Safety, and Pharmacokinetics Study of AG-236 in Subjects With Polycythemia Vera" — expected November 1, 2026.
Who is the CEO of AGIOS PHARMACEUTICALS, INC.?
Mr. Brian M. Goff M.B.A. is the chief executive officer of AGIOS PHARMACEUTICALS, INC. (AGIO).
Where is AGIOS PHARMACEUTICALS, INC. headquartered?
AGIOS PHARMACEUTICALS, INC. (AGIO) is headquartered in Cambridge, MA, United States.
The AI overview and BioSniper Score are neutral, data-derived signals built from public SEC filings, ClinicalTrials.gov and FDA records — not investment advice.