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

Share price

$33.07

Market cap

$1,974.3M

Cash

$617.6M

Enterprise value

$1,356.7M

Balance sheet

Cash	$617.6M
Debt	$0.0M
Net cash	$617.6M
Enterprise value	$1,356.7M
Basic shares	59.6M shares
Fully diluted shares	101.7M shares
Fully diluted market cap	$3,364.7M
Cash per share	$10.36

Cash figures as of 2026-06-30. Database snapshot: 2026-09-21.

Price history

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Upcoming catalysts

Expected	Drug	Phase	Indication	Mgmt guide
Feb 28 - Apr 10	AG-181	Phase 1	Phenylketonuria	—
Apr 12 - May 24	Mitapivat	Phase 3	Non-Transfusion-dependent Alpha-Thalassemia; Non-Transfusion-dependent Beta-Thalassemia	—
Jul 13 - Aug 24	Mitapivat	Phase 3	Transfusion-dependent Alpha-Thalassemia; Transfusion-dependent Beta-Thalassemia	—
Sep 12 - Oct 24	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	Non-Transfusion-dependent Alpha-Thalassemia
Lead phase	Phase 3
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

Institutional holders

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

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

AGIO — Agios Pharmaceuticals Inc.

Balance sheet

Price history

Upcoming catalysts

Pipeline assets

Pipeline

Active clinical trials

Institutional holders