agio-20260331

UNITED STATES

SECURITIES AND EXCHANGE COMMISSION

Washington, D.C. 20549

FORM 10-Q

(Mark One)


☒			QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934

For the quarterly period ended March 31, 2026


☐			TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934

Commission File Number: 001-36014

AGIOS PHARMACEUTICALS, INC.

(Exact Name of Registrant as Specified in Its Charter)


Delaware									26-0662915
(State or Other Jurisdiction of Incorporation or Organization)									(I.R.S. Employer Identification No.)

88 Sidney Street, Cambridge, Massachusetts									'02139
(Address of Principal Executive Offices)									(Zip Code)

(617) 649-8600

(Registrant’s Telephone Number, Including Area Code)

(Former Name, Former Address and Former Fiscal Year, if Changed Since Last Report)

Securities registered pursuant to Section 12(b) of the Act:


Title of each class	Trading symbol(s)	Name of each exchange on which registered
Common Stock, Par Value $0.001 per share	AGIO	Nasdaq Global Select Market

Indicate by check mark whether the registrant (1) has filed all reports required to be filed by Section 13 or 15(d) of the Securities Exchange Act of 1934 during the preceding 12 months (or for such shorter period that the registrant was required to file such reports), and (2) has been subject to such filing requirements for the past 90 days. Yes ☒ No ☐

Indicate by check mark whether the registrant has submitted electronically every Interactive Data File required to be submitted pursuant to Rule 405 of Regulation S-T (§232.405 of this chapter) during the preceding 12 months (or for such shorter period that the registrant was required to submit such files). Yes ☒ No ☐

Indicate by check mark whether the registrant is a large accelerated filer, an accelerated filer, a non-accelerated filer, a smaller reporting company, or an emerging growth company. See the definitions of “large accelerated filer,” “accelerated filer,” “smaller reporting company,” and “emerging growth company” in Rule 12b-2 of the Exchange Act.


Large accelerated filer	☒	Accelerated filer	☐
Non-accelerated filer	☐	Smaller reporting company	☐
		Emerging growth company	☐

If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐

Indicate by check mark whether the registrant is a shell company (as defined in Rule 12b-2 of the Exchange Act). Yes ☐ No ☒

Number of shares of the registrant’s Common Stock, $0.001 par value, outstanding on April 24, 2026: 59,471,609

Table of Contents

Available Information about Agios Pharmaceuticals, Inc.

Our Annual Reports on Form 10-K, Quarterly Reports on Form 10-Q, Current Reports on Form 8-K and any amendments to these reports filed or furnished pursuant to Section 13(a) or 15(d) of the Securities Exchange Act of 1934, as amended, are available free of charge on our website located at www.agios.com as soon as reasonably practicable after they are filed with or furnished to the Securities and Exchange Commission, or SEC. These reports are also available at the SEC’s website at www.sec.gov.

We may also disclose information to the public concerning our business, products and product candidates and other items through a variety of disclosure channels in order to achieve broad, non-exclusionary distribution of information to the public, including press releases, SEC filings and public conference calls and webcasts. Some of the information distributed through these disclosure channels may be considered material information. Investors and others are encouraged to review the information we make public through our disclosure channels.

Investors and others should note that Agios plans to use its website (www.agios.com) as a distribution channel to announce and give notice of Agios’ upcoming events and presentations (including, but not limited to, presentations at medical or healthcare conferences). Such information, which may be deemed material, will be available on the “Investors” section of the Company’s website under the “Events & Presentations” tab. Investors and others are encouraged to monitor the website and review such information Agios makes public through the website. In addition, you may sign up to automatically receive email alerts about Agios’ upcoming events and presentations (“Calendar Alerts”) by visiting the “Email Alerts” option under the “IR Resources” tab of the “Investors” section of the Company’s website and submitting your email address.

The Company’s website, and the contents thereof, are not incorporated by reference into this Quarterly Report on Form 10-Q nor deemed filed with the SEC.

Table of Contents

AGIOS PHARMACEUTICALS, INC.

FORM 10-Q

FOR THE THREE MONTHS ENDED MARCH 31, 2026

TABLE OF CONTENTS


		Page No.
PART I. FINANCIAL INFORMATION
Item 1.	Financial Statements (Unaudited)	1
	Condensed Consolidated Balance Sheets as of March 3 1 , 202 6 and December 31, 2025	1
	Condensed Consolidated Statements of Operations for the Three Months Ended March 3 1 , 202 6 and 2025	2
	Condensed Consolidated Statements of Comprehensive Loss for the Three Months Ended March 3 1 , 202 6 and 2025	3
	Condensed Consolidated Statements of Stockholders' Equity for the Three Months Ended March 3 1 , 202 6 and 2025	4
	Condensed Consolidated Statements of Cash Flows for the Three Months Ended March 3 1 , 202 6 and 2025	5
	Notes to Condensed Consolidated Financial Statements	6
Item 2.	Management’s Discussion and Analysis of Financial Condition and Results of Operations	17
Item 3.	Quantitative and Qualitative Disclosures About Market Risk	27
Item 4.	Controls and Procedures	27
PART II. OTHER INFORMATION		28
Item 1A.	Risk Factors	28
Item 5.	Other Information	57
Item 6.	Exhibits	58
	Signatures	59

Table of Contents

PART I. FINANCIAL INFORMATION

Item 1. Financial Statements (Unaudited)

AGIOS PHARMACEUTICALS, INC.

Condensed Consolidated Balance Sheets

(Unaudited)


(In thousands, except share and per share data)	March 31, 2026		December 31, 2025
Assets
Current assets:
Cash and cash equivalents	$	113,600	$	89,130
Marketable securities	623,438		765,295
Accounts receivable, net	16,132		10,577



Inventory	35,087		32,920
Prepaid expenses and other current assets	46,586		44,130

Total current assets	834,843		942,052
Marketable securities	308,454		310,013
Operating lease assets	27,196		30,454
Property and equipment, net	10,566		10,775
Other non-current assets	3,931		3,931

Total assets	$	1,184,990	$	1,297,225
Liabilities and stockholders’ equity
Current liabilities:

Accounts payable	$	13,747	$	18,355
Accrued expenses	26,293		45,459

Operating lease liabilities	18,805		18,392




Total current liabilities	58,845		82,206

Operating lease liabilities, net of current portion	16,940		21,815
Other non-current liabilities	90		90


Total liabilities	75,875		104,111
Stockholders’ equity:
Preferred stock, $0.001 par value; 25,000,000 shares authorized; no shares issued or outstanding at March 31, 2026 and December 31, 2025	—		—
Common stock, $0.001 par value; 125,000,000 shares authorized; 75,557,749 shares issued and 59,341,338 outstanding at March 31, 2026, and 74,665,003 shares issued and 58,448,592 outstanding at December 31, 2025	76		75
Additional paid-in capital	2,572,655		2,555,037
Accumulated other comprehensive (loss) income	(314)		2,193
Accumulated deficit	(660,816)		(561,705)
Treasury stock, at cost (16,216,411 shares at March 31, 2026 and December 31, 2025)	(802,486)		(802,486)
Total stockholders’ equity	1,109,115		1,193,114
Total liabilities and stockholders’ equity	$	1,184,990	$	1,297,225

See accompanying Notes to Condensed Consolidated Financial Statements.

Table of Contents

AGIOS PHARMACEUTICALS, INC.

Condensed Consolidated Statements of Operations

(Unaudited)


	Three Months Ended March 31,
(In thousands, except share and per share data)	2026		2025
Revenues:
Product revenue, net	$	20,746	$	8,726


Total revenue	20,746		8,726
Operating expenses:
Cost of sales	$	1,319	$	1,085
Research and development	81,148		72,743
Selling, general and administrative	48,304		41,527
Total operating expenses	130,771		115,355
Loss from operations	(110,025)		(106,629)


Interest income, net	10,795		16,087
Other income, net	119		1,253





Net loss	$	(99,111)	$	(89,289)


Net loss per share - basic and diluted	$	(1.69)	$	(1.55)


Weighted-average number of common shares used in computing net loss per share – basic and diluted	58,782,241		57,459,195

See accompanying Notes to Condensed Consolidated Financial Statements.

Table of Contents

AGIOS PHARMACEUTICALS, INC.

Condensed Consolidated Statements of Comprehensive Loss

(Unaudited)


	Three Months Ended March 31,
(In thousands)	2026		2025
Net loss	$	(99,111)	$	(89,289)
Other comprehensive (loss) income
Unrealized (loss) gain on available-for-sale securities	(2,507)		1,883
Comprehensive loss	$	(101,618)	$	(87,406)

See accompanying Notes to Condensed Consolidated Financial Statements.

Table of Contents

AGIOS PHARMACEUTICALS, INC.

Condensed Consolidated Statements of Stockholders' Equity

(Unaudited)


	Common Stock			Additional Paid-In Capital		Accumulated Other Comprehensive Income (Loss)		Accumulated Deficit		Treasury Stock			Total Stockholders’ Equity
(in thousands, except share amounts)	Shares	Amount								Shares	Amount
Balance at December 31, 2025	74,665,003	$	75	$	2,555,037	$	2,193	$	(561,705)	(16,216,411)	$	(802,486)	$	1,193,114

Unrealized loss on available-for-sale securities	—	—		—		(2,507)		—		—	—		(2,507)
Common stock issued under stock incentive plan and ESPP	892,746	1		2,077		—		—		—	—		2,078
Stock-based compensation expense	—	—		15,541		—		—		—	—		15,541

Net loss	—	—		—		—		(99,111)		—	—		(99,111)

Balance at March 31, 2026	75,557,749	$	76	$	2,572,655	$	(314)	$	(660,816)	(16,216,411)	$	(802,486)	$	1,109,115


	Common Stock			Additional Paid-In Capital		Accumulated Other Comprehensive Income (Loss)		Accumulated Deficit		Treasury Stock			Total Stockholders’ Equity
(in thousands, except share amounts)	Shares	Amount								Shares	Amount
Balance at December 31, 2024	73,372,696	$	73	$	2,493,811	$	(1,518)	$	(148,924)	(16,216,411)	$	(802,486)	$	1,540,956
Unrealized gain on available-for-sale securities	—	—		—		1,883		—		—	—		1,883

Common stock issued under stock incentive plan and ESPP	730,496	1		1,618		—		—		—	—		1,619
Stock-based compensation expense	—	—		11,359		—		—		—	—		11,359
Net loss	—	—		—		—		(89,289)		—	—		(89,289)
Balance at March 31, 2025	74,103,192	$	74	$	2,506,788	$	365	$	(238,213)	(16,216,411)	$	(802,486)	$	1,466,528

See accompanying Notes to Condensed Consolidated Financial Statements.

Table of Contents

AGIOS PHARMACEUTICALS, INC.

Condensed Consolidated Statements of Cash Flows
(Unaudited)


	Three Months Ended March 31,
(In thousands)	2026		2025
Operating activities
Net loss	$	(99,111)	$	(89,289)


Adjustments to reconcile net loss from operations to net cash used in operating activities:
Depreciation and amortization	1,363		1,278
Stock-based compensation expense	15,541		11,359
Net accretion of discount on marketable securities	(1,144)		(1,348)

Non-cash operating lease expense	3,258		3,018

Realized gain on investments	(28)		(2)



Changes in operating assets and liabilities:
Accounts receivable, net	(5,555)		765



Inventory	(2,167)		(1,989)


Prepaid expenses and other current and non-current assets	(2,456)		(2,124)
Accounts payable	(4,993)		(2,821)
Accrued expenses and other current liabilities	(19,166)		(26,100)

Income taxes payable	—		(188)
Operating lease liabilities	(4,462)		(4,048)




Net cash used in operating activities	(118,920)		(111,489)
Investing activities
Purchases of marketable securities	(148,182)		(174,903)
Proceeds from maturities and sales of marketable securities	290,263		288,256



Purchases of property and equipment	(769)		(766)



Net cash provided by investing activities	141,312		112,587
Financing activities



Net proceeds from stock option exercises and employee stock purchase plan	2,078		1,619


Net cash provided by financing activities	2,078		1,619
Net change in cash and cash equivalents	24,470		2,717
Cash and cash equivalents at beginning of the period	89,130		76,247
Cash and cash equivalents at end of the period	$	113,600	$	78,964


Supplemental disclosure of non-cash investing and financing transactions
Additions to property and equipment in accounts payable	$	668	$	221
Exchanges of marketable securities	$	2,229	$	—



Net cash taxes paid	$	4	$	188

See accompanying Notes to Condensed Consolidated Financial Statements.

Table of Contents

AGIOS PHARMACEUTICALS, INC.

Notes to Condensed Consolidated Financial Statements

(Unaudited)

1. Overview and Basis of Presentation

Note Regarding Certain References in this Quarterly Report on Form 10-Q

Throughout this Quarterly Report on Form 10-Q,“Agios,” “the Company,” “we,” “us,” and “our,” and similar expressions, except where the context requires otherwise, refer to Agios Pharmaceuticals, Inc. and its consolidated subsidiaries, and “our Board of Directors” refers to the board of directors of Agios Pharmaceuticals, Inc.

In addition, unless otherwise stated or the context indicates otherwise, all references in this Annual Report on Form 10-K to “AQVESME™ (mitapivat)” or “AQVESME™” refer to our FDA-approved medicine for the treatment of anemia in adults with non-transfusion dependent and transfusion-dependent alpha- or beta-thalassemia in the United States; references to “PYRUKYND® (mitapivat)”, “PYRUKYND®”, or “mitapivat” refer to all other commercially available mitapivat products or mitapivat product candidates for which we are exploring further applications and indications, as the context requires.

Overview

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 that have the potential to transform lives. With a foundation in hematology, we combine biological expertise with real-world insights to advance a growing pipeline of rare disease medicines that reflect the priorities of the people we serve. We are located in Cambridge, Massachusetts.

The lead product candidate in our portfolio, mitapivat, is an activator of both wild-type and mutant pyruvate kinase, or PK, enzymes for the potential treatment of hemolytic anemias. Mitapivat is approved in the United States by the U.S. Food and Drug Administration, or FDA, under the brand name AQVESME™ for the treatment of anemia in adults with non-transfusion dependent and transfusion-dependent alpha- or beta-thalassemia and in Saudi Arabia and the United Arab Emirates under the brand name PYRUKYND® for the treatment of adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia. Mitapivat is also approved under the brand name PYRUKYND® in the United States by the FDA for the treatment of hemolytic anemia in adults with PK deficiency and in the European Union, or EU, and Great Britain for the treatment of PK deficiency in adults.

In December 2024, we announced that we submitted a marketing authorization application, or MAA, to the European Medicines Agency, or EMA for PYRUKYND® for the treatment of adult patients with non-transfusion dependent and transfusion-dependent alpha- or beta-thalassemia. In October 2025, we announced that the Committee for Medicinal Products for Human Use, or the CHMP, of the EMA adopted a positive opinion for the new indication for PYRUKYND® in adults for the treatment of anemia associated with transfusion-dependent and non-transfusion-dependent alpha- or beta-thalassemia. The European Commission is reviewing the CHMP’s opinion, with the final decision expected in the first half of 2026.

In the first quarter of 2026, we presented data from the phase 2 and phase 3 portions of the RISE UP study of PYRUKYND® in adults with sickle cell disease, or SCD, in a pre-supplemental New Drug Application meeting with the FDA. Based on the FDA’s recommendation, we submitted our proposal to the FDA for a confirmatory clinical trial to support U.S. accelerated approval of mitapivat in SCD. The proposal incorporates a primary endpoint that is different from the endpoints in the RISE UP studies and is informed by both analyses of RISE UP data and discussions with the FDA. We intend to submit a supplemental New Drug Application, or sNDA, for mitapivat for SCD in the second quarter of 2026.

In addition, we are evaluating mitapivat for the treatment of pediatric patients with PK deficiency. We are also developing (i) tebapivat, a novel PK activator, for the potential treatment of lower-risk myelodysplastic syndromes, or LR MDS, and SCD; (ii) AG-181, our phenylalanine hydroxylase, or PAH, stabilizer for the potential treatment of phenylketonuria, or PKU; and (iii) AG-236, an siRNA in-licensed from Alnylam Pharmaceuticals, Inc., or Alnylam, targeting the transmembrane serine protease 6, or TMPRSS6, gene for the potential treatment of polycythemia vera, or PV.

We are subject to risks common to companies in our industry including, but not limited to, uncertainties relating to conducting preclinical and clinical research and development, the manufacture and supply of products for clinical and commercial use, obtaining and maintaining regulatory approvals and pricing and reimbursement for our products, market acceptance, managing global growth and operating expenses, availability of additional capital, competition, obtaining and enforcing patents, stock price volatility, dependence on collaborative relationships and third-party service providers, dependence on key personnel, potential litigation, potential product liability claims and potential government investigations.

Table of Contents

Sale of Oncology Business to Servier and Sale of Contingent Payments

In March 2021, we completed the sale of our oncology business to Servier Pharmaceuticals, LLC, or Servier. The transaction included the sale of our entire oncology business, including our clinical-stage product candidate vorasidenib, and included a royalty of 15% of U.S. net sales of vorasidenib from the first commercial sale of vorasidenib through loss of exclusivity, or the Vorasidenib Royalty Rights. The Vorasidenib Royalty Rights are referred to as contingent payments and recognized as income when realizable.

In May 2024, we entered into a purchase and sale agreement to sell the Vorasidenib Royalty Rights to Royalty Pharma Investments 2019 ICAV, or Royalty Pharma. The sale was contingent upon FDA approval of vorasidenib and other customary closing conditions.

Upon consummation of the sale in August 2024, Royalty Pharma acquired 100% of the Vorasidenib Royalty Rights payments made by Servier on account of up to $1.0 billion in U.S. net sales for each calendar year. In addition, any such Vorasidenib Royalty Rights payments made by Servier on account of U.S. net sales in each calendar year in excess of $1.0 billion will be split, with Royalty Pharma having the rights to a 12% earn-out on those excess payments and Agios retaining the rights to a 3% earn-out on those excess payments, or the Retained Earn-Out Rights. Royalty income related to the Retained Earn-Out Rights, if any, will be recognized in the period when realizable.

Basis of Presentation

The condensed consolidated balance sheet as of March 31, 2026, the condensed consolidated statements of operations, comprehensive loss and stockholders' equity for the three months ended March 31, 2026 and 2025, and the condensed consolidated statements of cash flows for the three months ended March 31, 2026 and 2025 are unaudited. The unaudited condensed consolidated financial statements have been prepared on the same basis as the annual financial statements and, in the opinion of our management, reflect all adjustments, which include only normal recurring adjustments, necessary to fairly state our financial position as of March 31, 2026, our results of operations and stockholders' equity for the three months ended March 31, 2026 and 2025, and cash flows for the three months ended March 31, 2026 and 2025. The financial data and other financial information disclosed in these notes to the condensed consolidated financial statements related to the three-month periods are also unaudited. The results of operations for the three months ended March 31, 2026 are not necessarily indicative of the results to be expected for the year ending December 31, 2026 or for any other future annual or interim period. The condensed consolidated balance sheet data as of December 31, 2025 was derived from our audited financial statements, but does not include all disclosures required by U.S. generally accepted accounting principles, or U.S. GAAP. The condensed consolidated interim financial statements should be read in conjunction with the audited consolidated financial statements and notes thereto included in our Annual Report on Form 10-K for the year ended December 31, 2025 that was filed with the Securities and Exchange Commission, or SEC, on February 12, 2026.

Our condensed consolidated financial statements include our accounts and the accounts of our wholly owned subsidiaries. All intercompany transactions have been eliminated in consolidation. The condensed consolidated financial statements have been prepared in conformity with U.S. GAAP.

Use of Estimates

The preparation of our condensed consolidated financial statements requires us to make estimates, judgments and assumptions that may affect the reported amounts of assets, liabilities, equity, revenues and expenses and related disclosure of contingent assets and liabilities. On an ongoing basis we evaluate our estimates, judgments and methodologies. We base our estimates on historical experience and on various other assumptions that we believe are reasonable, the results of which form the basis for making judgments about the carrying values of assets, liabilities and equity and the amount of revenues and expenses.

Liquidity

As of March 31, 2026, we had cash, cash equivalents and marketable securities of $1.0 billion. Although we have incurred recurring losses and expect to continue to incur losses for the foreseeable future, we expect our cash, cash equivalents and marketable securities to be sufficient to fund current operations for at least the next twelve months from the issuance of the financial statements. If we are unable to raise additional funds through equity or debt financings, we may be required to delay, limit, reduce or terminate product development or future commercialization efforts, or grant rights to develop and market products or product candidates that we would otherwise prefer to develop and market ourselves.

Table of Contents

2. Summary of Significant Accounting Policies

There have been no material changes to the significant accounting policies previously disclosed in our Annual Report on Form 10-K for the year ended December 31, 2025.

Recent Accounting Pronouncements

In November 2024, the Financial Accounting Standards Board, or FASB, issued Accounting Standards Update, or ASU, 2024-03, Income Statement – Reporting Comprehensive Income – Expense Disaggregation Disclosures (Subtopic 220-40): Disaggregation of Income Statement Expenses, to improve disclosures around an entity’s expenses. Upon adoption, companies will be required to disclose in the notes to the financial statement

SEC Form 10-Q filed by Agios Pharmaceuticals Inc.