AGIOS PHARMACEUTICALS, INC.
AGIO evidence brief
What we can confirm
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 is a biopharmaceutical company focused on cellular metabolism and rare genetic diseases, with approved products PYRUKYND® and AQVESME™ and a pipeline targeting hemolytic anemias and other indications.
Key risk: Clinical and regulatory risk
BioSniper Score
Based on 6 of 6 signals
Market Cap
$1.99B
Cash Runway
32.5 moLead Asset
AG-946 (Phase 2)Next Catalyst
Phase3_Topline · Dec 1, 2027Financial data as of Jun 30, 2026.
Tracking 3 pipeline candidates · 33 clinical trials · 10 catalysts for AGIOS PHARMACEUTICALS, INC..
Agios Pharmaceuticals is a biopharmaceutical company focused on cellular metabolism and rare genetic diseases, with approved products PYRUKYND® and AQVESME™ and a pipeline targeting hemolytic anemias and other indications.
- Agios has two approved products: PYRUKYND® (mitapivat) for pyruvate kinase deficiency and AQVESME™ for thalassemia [sec_mda · as of 2026-07-30].
- The company is advancing mitapivat in sickle cell disease (SCD) and thalassemia, with multiple Phase 3 trials ongoing [sec · filed 2024-04-26].
- Pipeline includes AG-181 for PKU (Phase 1b) and AG-236 for polycythemia vera (IND cleared June 2025) [sec_mda · as of 2026-07-30].
- As of June 30, 2026, Agios reported cash & equivalents of $99.6M, total revenue of $44.7M, and a net loss of $100.7M [sec].
- The company expects existing cash, product revenue, and interest income to fund the U.S. launch of AQVESME™ and prepare for potential mitapivat launch in SCD [sec_mda · as of 2026-04-29].
What works
- Agios has two commercial-stage products (PYRUKYND® and AQVESME™) generating revenue, providing a foundation for ongoing operations [sec_mda · as of 2026-07-30].
- The pipeline includes multiple Phase 3 trials for mitapivat in SCD and thalassemia, targeting large unmet needs [sec · filed 2024-04-26].
- The company has a license agreement with Alnylam for an siRNA targeting TMPRSS6 (AG-236) and an IND cleared for polycythemia vera, diversifying the pipeline [sec_mda · as of 2026-07-30].
What to weigh
- Agios reported a net loss of $100.7M for the six months ended June 30, 2026, indicating ongoing operating losses [sec].
- The company faces significant commercialization expenses for potential new indications, and future capital requirements depend on uncertain revenue and partnership payments [sec_mda · as of 2026-04-29].
- There is no guarantee that any product candidate will successfully complete clinical development or receive marketing approval [sec · filed 2026-04-24].
From the filings
Quoted directly from source documents.
Agios has approved products PYRUKYND® and AQVESME™ and is commercializing them.
“commercial launches of PYRUKYND® and AQVESME™”
SEC MD&A · July 30, 2026
The company is conducting Phase 3 trials for mitapivat in thalassemia and SCD.
“Advance PYRUKYND®, including progressing pivotal programs in thalassemia and SCD”
SEC filings · April 26, 2024
Agios has an IND cleared for AG-236 for polycythemia vera.
“We filed an IND with the FDA for AG-236 for the treatment of PV, which cleared in June 2025”
SEC MD&A · July 30, 2026
The company expects existing cash and revenue to fund near-term launches.
“We expect that our existing cash, cash equivalents and marketable securities as of March 31, 2026, together with anticipated product revenue and interest income, will provide the financial independence to execute the U.S. commercial launch of AQVESME™ in thalassemia, prepare for the potenti”
SEC MD&A · April 29, 2026
Catalyst timeline
- 2027-12-01 · Phase3_Topline · ivosidenib — Phase 3 Topline: An Open-Label Early Access Phase 3b Study of Ivosidenib in Patients With a Pretreated Locally Advanc — Topline data from a Phase 3b study of ivosidenib in pretreated patients.
- 2028-12-01 · Phase3_Topline · mitapivat — Phase 3 Topline: A Phase 3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study Evaluating the Efficacy a — Topline data from a Phase 3 study of mitapivat evaluating efficacy in an unspecified indication.
- 2029-06-01 · Phase3_Topline — Phase 3 Topline: A Phase 3, Multicenter, Randomized, Double-blind, Placebo-Controlled Study to Evaluate the Efficacy — Topline data from a Phase 3 study evaluating efficacy of an unspecified candidate.
- 2029-06-01 · Phase3_Topline · mitapivat — Phase 3 Topline: A Phase 3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study Evaluating the Efficacy a — Topline data from another Phase 3 study of mitapivat.
- 2030-01-01 · Phase3_Topline — Phase 3 Topline: A Phase 3, Multicenter, Randomized, Double-blind, Placebo-Controlled Study to Evaluate the Efficacy — Topline data from a Phase 3 study evaluating efficacy.
- 2030-02-01 · Phase3_Topline — Phase 3 Topline: A Phase 2/3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study to Evaluate the Efficac — Topline data from a Phase 2/3 study evaluating efficacy.
- 2030-08-01 · Phase3_Topline — Phase 3 Topline: A Phase 3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study to Evaluate the Effect of — Topline data from a Phase 3 study evaluating the effect of an unspecified candidate.
- 2032-03-01 · Phase3_Topline — Phase 3 Topline: A Phase 3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study Evaluating the Efficacy, — Topline data from a Phase 3 study evaluating efficacy.
Risks & what to watch (3)›
Key risks
- Clinical and regulatory risk — No guarantee that any product candidate will complete clinical development or gain approval [sec · filed 2026-04-24].
- Financial sustainability — Net loss of $100.7M in H1 2026; future capital needs depend on uncertain revenue and partnerships [sec].
- Commercial execution risk — Significant expenses for launches of AQVESME™ and potential mitapivat in SCD; revenue may not cover costs [sec_mda · as of 2026-04-29].
What to watch
- Phase 3 topline data for mitapivat in SCD (ENERGIZE study) expected by end of 2027 [upcoming catalysts].
- Phase 3 topline data for mitapivat in thalassemia (ENERGIZE-T study) expected in 2028-2029 [upcoming catalysts].
- Proof-of-mechanism data for AG-181 in PKU expected in second half of 2026 [sec_mda · as of 2026-07-30].
- Commercial uptake of AQVESME™ following U.S. launch in thalassemia [sec_mda · as of 2026-04-29].
Sources: SEC filings · SEC MD&A · Generated August 28, 2026 · How we verify sources →
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Evidence & data
Score breakdownBased on 6 of 6 signals · derived from public data
32.5 months
D/E 0.08 · 0% dilution
Lead: Approved
3 candidates
10 upcoming, next in ~458d
0 buys · 11 sells (180d)
Runway
Catalysts
Pipeline
Regulatory
Financials
Institutional Holdings
Source: SEC Form 13F · as of Mar 2026Institutional holders
234
Total value held
$2.14B
| Holder | Shares | Value |
|---|---|---|
| armistice capital, llc | 5,916,000 | $200.1M |
| blackrock, inc. | 5,754,957 | $194.7M |
| farallon capital management llc | 5,684,000 | $192.3M |
| vanguard capital management llc | 5,264,632 | $178.1M |
| state street corp | 3,364,879 | $113.7M |
| d. e. shaw & co., inc. | 2,681,169 | $90.7M |
| lynx1 capital management lp | 2,611,568 | $88.3M |
| vanguard portfolio management llc | 2,611,035 | $88.3M |
| bnp paribas asset management holding s.a. | 1,580,840 | $53.5M |
| susquehanna international group, llp | 1,556,353 | $52.7M |
Showing top 10 of 234 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 is a biopharmaceutical company focused on cellular metabolism and rare genetic diseases, with approved products PYRUKYND® and AQVESME™ and a pipeline targeting hemolytic anemias and other indications.
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_Topline event — "Phase 3 Topline: An Open-Label Early Access Phase 3b Study of Ivosidenib in Patients With a Pretreated Locally Advanc" — expected December 1, 2027.
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.