AGIO Stock — Agios Pharmaceuticals Inc.: Cash, EV, Pipeline

What they do

We are a commercial-stage biopharmaceutical company dedicated to redefining the future of rare disease treatment. Fueled by connections, we build trusted partnerships with communities, collaborating to develop and deliver innovative medicines with the potential to transform lives.

Source: 10-K FY2025 via SEC EDGAR

Share price

$29.41

Market cap

$1,755.8M

Cash

$907.0M

Enterprise value

$848.8M

Balance sheet

Cash	$907.0M
Debt	$0.0M
Net cash	$907.0M
Enterprise value	$848.8M
Basic shares	59.8M shares
Fully diluted shares	70.2M shares
Fully diluted market cap	$2,064.1M
Cash per share	$15.17
Quarterly burn	$57.3M
Cash runway	16.8 quarters

Cash figures as of 2026-09-30. Database snapshot: 2026-11-07.

Share structure

Common

59.7M

Pre-funded warrants

0.0M

Options

6.6M

RSUs

2.5M

Warrants

0.0M

Converts

0.0M

Conv preferred

0.0M

ESPP reserve

1.4M

FDSO

70.2M

Cap table as of 2026-06-30.

Price history

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Next catalyst

AG-181 · Feb 28 - Apr 10 in 16 mo · Phase 1 · Phenylketonuria

Recently reported

Mitapivat Phase 3 Mixed · 2026-11-04

AG-236

Primary: Hemoglobin improvement (≥1.0 g/dL increase from baseline in average hemoglobin during any two consecutive study visits) in the RISE UP Phase 3 open-label extension in sickle cell disease — 62.7% (n=74) of 118 patients continuing on mitapivat achieved a hemoglobin improvement during the OLE period

· Of 176 patients who completed the double-blind period, 174 (118 from the mitapivat arm and 56 from the placebo arm) enrolled in the OLE period; long-term treatment up to 115 weeks

· Of 63 non-responders during the double-blind period, 39.7% (n=25) achieved a hemoglobin improvement with continued mitapivat treatment during the OLE period

· Of 56 patients who crossed over from placebo to mitapivat, 51.8% (n=29) achieved a hemoglobin improvement during the OLE period

· Post hoc eGFR analysis in 132 patients with hyperfiltration (eGFR >135 mL/min/1.73 m²) at baseline: 14.7 mL/min/1.73 m² decrease in eGFR at Week 52 with mitapivat (n=92) vs 4.9 decrease with placebo (n=40)

· Post hoc cardiac analysis at Week 52: left ventricular mass index decreased 5.9 g/m² among hemoglobin responders in the mitapivat arm (n=40) vs increases of 5.8 in non-responders (n=45) and 4.6 in placebo (n=27)

· AG-236 first Phase 1 data (24 healthy volunteers; 1.0, 3.0 and 9.0 mg/kg): sustained hepcidin increases and decreases in serum iron and transferrin saturation through Month 6 post-dose

Safety: Safety profile in the OLE period was consistent with the double-blind period; AG-236 was well tolerated across all dose levels with no serious adverse events and no dose-limiting toxicities

Source release →

Upcoming catalysts

Expected	Drug	Phase	Indication	Mgmt guide
Feb 28 - Apr 10 in 16 mo	AG-181	Phase 1	Phenylketonuria	—
Apr 12 - May 24 in 30 mo	Mitapivat	Phase 3	Non-Transfusion-dependent Alpha-Thalassemia; Non-Transfusion-dependent Beta-Thalassemia	—
Jul 13 - Aug 24 in 33 mo	Mitapivat	Phase 3	Transfusion-dependent Alpha-Thalassemia; Transfusion-dependent Beta-Thalassemia	—
Sep 12 - Oct 24 in 35 mo	Mitapivat	Phase 3	Sickle Cell Disease	—

Estimated readout windows: registry primary-completion date + 6-12 weeks. Windows already open are shown from "Now". Drug names link to ClinicalTrials.gov.

Full readout calendar →

Pipeline assets

Mitapivat

Approved · Active Modality: small molecule

Aliases PYRUKYND, AQVESME

Target pyruvate kinase (PK)

Partnerships NewBridge Pharmaceuticals: distribution agreement for PYRUKYND in certain MENA territories; Avanzanite: commercialization access in EU/GB/Saudi Arabia/UAE

Indications

PK deficiency (Approved)
Thalassemia (alpha/beta) (Approved)
Sickle cell disease (Stage not disclosed)

Catalysts

— November 1, 2026

Status note Approved in US as AQVESME for anemia in adults with non-transfusion dependent and transfusion-dependent alpha- or beta-thalassemia and as PYRUKYND for hemolytic anemia in adults with PK deficiency; also approved in EU/GB/Saudi Arabia/UAE. Confirmatory REIGNITE phase 3 trial in SCD ongoing under accelerated-approval pathway.

Tebapivat

Phase 2 · Terminated

Target pyruvate kinase (PK)

Indications

Lower-risk myelodysplastic syndromes (Phase 2)
Sickle cell disease (Phase 2)

Status note Development discontinued: May 2026 — will not advance in LR-MDS (phase 2b did not meet predefined threshold); July 2026 — will not continue development in SCD (phase 2 did not establish sufficient differentiation from other PK activators).

AG-181

Phase 1 · Active

Target phenylalanine hydroxylase (PAH)

Indications

Phenylketonuria (PKU) (Phase 1)

Status note IND filed December 2023; phase 1 trial in healthy volunteers initiated Q1 2024, multiple ascending dose portion initiated Q2 2024.

AG-236

Preclinical · Active Modality: siRNA

Target TMPRSS6

Partnerships Alnylam Pharmaceuticals: license agreement for development and commercialization of the TMPRSS6 siRNA

Indications

Polycythemia vera (Preclinical)

Status note In-licensed from Alnylam under July 28, 2023 license agreement ($17.5M upfront IP R&D recognized in 2023).

Cevidoplenib

Stage not disclosed · Active Modality: small molecule

Target SYK (spleen tyrosine kinase)

Partnerships Oscotec Inc.: June 1, 2026 license agreement for worldwide rights (Oscotec retains opt-in for South Korea); $25M upfront IP R&D paid; up to $140M in development/regulatory milestones

Indications

Immune thrombocytopenia (ITP) (Stage not disclosed)
Other autoimmune indications (Stage not disclosed)

Catalysts

— first half of 2028

Status note Oral SYK inhibitor in-licensed from Oscotec; Agios will account for the deal as an asset acquisition.

Per-asset detail extracted from the 10-Q filed 2026-07-30. Fields the filing does not state are marked "not disclosed" rather than filled from other sources.

Pipeline

Lead drug	Mitapivat
Lead indication	Pyruvate Kinase Deficiency
Lead phase	Phase 4
Active trials	15
Collaborator trials	2

Active clinical trials

Trial	Phase	Status	Role	Sponsor	BP	Condition(s)	Mgmt guide
NCT07517133	Phase 3	Recruiting	Sponsor	AGIO	—	Non-Transfusion-dependent Alpha-Thalassemia; Non-Transfusion-dependent Beta-Thalassemia	—
NCT07506863	Phase 3	Recruiting	Sponsor	AGIO	—	Transfusion-dependent Alpha-Thalassemia; Transfusion-dependent Beta-Thalassemia	—
NCT07656415	Phase 3	Recruiting	Sponsor	AGIO	—	Sickle Cell Disease	—
NCT04770753	Phase 3	Active, not recruiting	Sponsor	AGIO	—	Non-Transfusion-dependent Alpha-Thalassemia; Non-Transfusion-dependent Beta-Thalassemia	—
NCT04770779	Phase 3	Active, not recruiting	Sponsor	AGIO	—	Transfusion-dependent Alpha-Thalassemia; Transfusion-dependent Beta-Thalassemia	—
NCT05144256	Phase 3	Active, not recruiting	Sponsor	AGIO	—	Pediatric Pyruvate Kinase Deficiency; Pediatric Hemolytic Anemia	—
NCT05175105	Phase 3	Active, not recruiting	Sponsor	AGIO	—	Pediatric Pyruvate Kinase Deficiency; Pediatric Hemolytic Anemia	—
NCT05031780	Phase 3	Active, not recruiting	Sponsor	AGIO	—	Sickle Cell Disease	—
NCT03692052	Phase 2	Active, not recruiting	Sponsor	AGIO	—	Thalassemia	—
NCT05490446	Phase 2	Active, not recruiting	Sponsor	AGIO	—	Myelodysplastic Syndromes	—
NCT06924970	Phase 2	Active, not recruiting	Sponsor	AGIO	—	Sickle Cell Disease	—
NCT07241234	Phase 1	Recruiting	Sponsor	AGIO	—	Phenylketonuria	—

Sponsor-matched active trials from ClinicalTrials.gov (15 as sponsor, 2 as collaborator); "Sponsor" = company is the lead sponsor, "Collaborator" = company is a collaborator. The BP column flags big-pharma involvement as lead sponsor or collaborator (excluding the company itself). The Mgmt guide column shows what management guided for readout timing when a guided program matches the trial (hover for the quote, click for the source). Trial listings are not a company pipeline slide.

View all 17 active trials for Agios Pharmaceuticals, Inc. on ClinicalTrials.gov

Specialist fund holders

Tracked specialist funds: ADAR1 Capital Management, Aisling Capital, Ally Bridge Group, Avoro Capital Advisors, BVF Partners, Baker Bros. Advisors, Boxer Capital, Braidwell LP, Casdin Capital, Cormorant Asset Management, DAFNA Capital, Deerfield Management, EcoR1 Capital, Frazier Life Sciences, Great Point Partners, Ikarian Capital, Logos Global Management, OrbiMed Advisors, Perceptive Advisors, RA Capital Management, RTW Investments, Sphera Biotech, Stonepine Capital, Tang Capital Partners, Vivo Capital.

Fund	Shares	Q/Q change	% of portfolio	Filing
ADAR1 Capital Management	1,288,053	+1,147,381	2.25%	2026-08-14
Frazier Life Sciences	0	-1,256,080	0.00%	2026-08-14
Tang Capital Partners	0	-100,000	0.00%	2026-08-14

Specialist-fund positions from 13F filings. Q/Q change is vs the prior quarter's filing; % of portfolio is the fund's own reported portfolio weight.

Latest news

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AGIO — Agios Pharmaceuticals Inc. ☆

What they do

Balance sheet

Share structure

Price history

Next catalyst

Recently reported

Upcoming catalysts

Pipeline assets

Pipeline

Active clinical trials

Specialist fund holders

Latest news

AGIO — Agios Pharmaceuticals Inc.