What changed in this filing

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Item 1. Financial Statements

2026-04-29 compared with 2025-10-30 · 146 added, 185 removed, 325 unchanged (50% of the section changed)

3 unchanged sentences

(In thousands, except share and per share data)

September 30,

2026 December 31,

15 unchanged sentences

Accrued expenses 26,293 45,459

Income taxes payable — 871

Operating lease liabilities 18,805 18,392

6 unchanged sentences

25,000,000 shares authorized;

no shares issued or outstanding at September 30, 2025 and December 31, 2024

no shares issued or outstanding at March 31, 2026 and December 31, 2025

Common stock, $ 0.001 par value;

125,000,000 shares authorized;

74,496,287 shares issued and 58,279,876 shares outstanding at September 30, 2025, and 73,372,696 shares issued and 57,156,285 shares outstanding at December 31, 2024

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

Additional paid-in capital 2,572,655 2,555,037

Accumulated other comprehensive income (loss) 2,051 ( 1,518 )

Treasury stock, at cost ( 16,216,411 shares at September 30, 2025 and December 31, 2024)

( 802,486 ) ( 802,486 )

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

3 unchanged sentences

Condensed Consolidated Statements of Operations

Three Months Ended September 30, Nine Months Ended September 30,

Three Months Ended March 31,

(In thousands, except share and per share data)

2025 2024 2025 2024

Product revenue, net $ 20,746 $ 8,726

6 unchanged sentences

Loss from operations ( 110,025 ) ( 106,629 )

Gain on sale of contingent payments — 889,136 — 889,136

Milestone payment from gain on sale of oncology business — 200,000 — 200,000

Interest income, net 10,795 16,087

Other income, net 119 1,253

Net (loss) income before taxes ( 103,433 ) 1,001,035 ( 304,742 ) 823,368

Income tax expense — 53,120 — 53,120

Net (loss) income $ ( 103,433 ) $ 947,915 $ ( 304,742 ) $ 770,248

Net (loss) income per share - basic $ ( 1.78 ) $ 16.65 $ ( 5.27 ) $ 13.58

Net (loss) income per share - diluted $ ( 1.78 ) $ 16.22 $ ( 5.27 ) $ 13.38

Weighted-average number of common shares used in computing net (loss) income per share – basic 58,139,277 56,939,403 57,846,173 56,709,318

Weighted-average number of common shares used in computing net (loss) income per share – diluted 58,139,277 58,432,796 57,846,173 57,581,382

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.

AGIOS PHARMACEUTICALS, INC.

Condensed Consolidated Statements of Comprehensive (Loss) Income

Three Months Ended September 30, Nine Months Ended September 30,

Condensed Consolidated Statements of Comprehensive Loss

Three Months Ended March 31,

(In thousands)

2025 2024 2025 2024

Net (loss) income $ ( 103,433 ) $ 947,915 $ ( 304,742 ) $ 770,248

Other comprehensive income

Unrealized gain on available-for-sale securities 1,396 4,465 3,569 3,861

Comprehensive (loss) income $ ( 102,037 ) $ 952,380 $ ( 301,173 ) $ 774,109

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.

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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 gain on available-for-sale securities — — — 1,883 — — — 1,883

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

2 unchanged sentences

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

Unrealized gain on available-for-sale securities — — — 290 — — — 290

Common stock issued under stock incentive plan and ESPP 151,764 — 65 — — — — 65

Stock-based compensation expense — — 14,692 — — — — 14,692

Net loss — — — — ( 112,020 ) — — ( 112,020 )

Balance at June 30, 2025 74,254,956 $ 74 $ 2,521,545 $ 655 $ ( 350,233 ) ( 16,216,411 ) $ ( 802,486 ) $ 1,369,555

Unrealized gain on available-for-sale securities — — — 1,396 — — — 1,396

Common stock issued under stock incentive plan and ESPP 241,331 — 4,435 — — — — 4,435

Stock-based compensation expense — — 12,377 — — — — 12,377

Net loss — — — — ( 103,433 ) — — ( 103,433 )

Balance at September 30, 2025 74,496,287 $ 74 $ 2,538,357 $ 2,051 $ ( 453,666 ) ( 16,216,411 ) $ ( 802,486 ) $ 1,284,330

Common Stock Additional

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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 loss on available-for-sale securities — — — ( 646 ) — — — ( 646 )

Common stock issued under stock incentive plan and ESPP 806,433 1 5,863 — — — — 5,864

Stock-based compensation expense — — 9,234 — — — — 9,234

Net loss — — — — ( 81,549 ) — — ( 81,549 )

Balance at March 31, 2024 72,967,922 $ 73 $ 2,451,620 $ ( 1,087 ) $ ( 904,198 ) ( 16,216,411 ) $ ( 802,486 ) $ 743,922

Unrealized gain on available-for-sale securities — — — 1,883 — — — 1,883

2 unchanged sentences

Net loss — — — — ( 89,289 ) — — ( 89,289 )

Balance at June 30, 2024 73,091,490 $ 73 $ 2,464,284 $ ( 1,045 ) $ ( 1,000,316 ) ( 16,216,411 ) $ ( 802,486 ) $ 660,510

Unrealized gain on available-for-sale securities — — — 4,465 — — — 4,465

Common stock issued under stock incentive plan and ESPP 146,464 — 2,637 — — — — 2,637

Stock-based compensation expense — — 11,145 — — — — 11,145

Net income — — — — 947,915 — — 947,915

Balance at September 30, 2024 73,237,954 $ 73 $ 2,478,066 $ 3,420 $ ( 52,401 ) ( 16,216,411 ) $ ( 802,486 ) $ 1,626,672

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.

1 unchanged sentence

Condensed Consolidated Statements of Cash Flows

Nine Months Ended

September 30,

Three Months Ended

(In thousands) 2026 2025

Operating activities

Net (loss) income $ ( 304,742 ) $ 770,248

Adjustments to reconcile net (loss) income to net cash used in 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

1 unchanged sentence

Net accretion of discount on marketable securities ( 1,144 ) ( 1,348 )

Loss (gain) on disposal of property and equipment 304 ( 39 )

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

Expense associated with license agreement 10,000 —

Realized gain on investments ( 28 ) ( 2 )

Gain on sale of contingent payments — ( 889,136 )

Milestone payment from gain on sale of oncology business — ( 200,000 )

Changes in operating assets and liabilities:

6 unchanged sentences

Operating lease liabilities ( 4,462 ) ( 4,048 )

Other non-current liabilities ( 790 ) —

Net cash used in operating activities ( 118,920 ) ( 111,489 )

2 unchanged sentences

Proceeds from maturities and sales of marketable securities 290,263 288,256

Payments associated with license agreement ( 10,000 ) —

Proceeds from sale of contingent payments — 889,136

Proceeds from milestone payment from gain on sale of oncology business — 200,000

Purchases of property and equipment ( 769 ) ( 766 )

Proceeds from sale of equipment — 40

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

7 unchanged sentences

Additions to property and equipment in accounts payable $ 668 $ 221

Net cash taxes paid (returned) $ 1,568 $ ( 637 )

Exchanges of marketable securities $ 2,229 $ —

Net cash taxes paid $ 4 $ 188

See accompanying Notes to Condensed Consolidated Financial Statements.

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Overview and Basis of Presentation

References to Agios

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.

We are a commercial-stage biopharmaceutical company dedicated to redefining the future of rare disease treatment.

We champion an inclusive culture that fosters bold innovation and collaboration, both within our teams and with the rare disease communities we serve.

By building deep connections and trusted partnerships with patients, advocates, and healthcare professionals, we advance our understanding of the unique priorities and needs of these communities.

These real-world insights, combined with our biological expertise, allow us to develop and deliver innovative medicines with the potential to transform lives.

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, PYRUKYND® (mitapivat), is an activator of both wild-type and mutant pyruvate kinase, or PK, enzymes for the potential treatment of hemolytic anemias.

PYRUKYND® is approved for use by the U.S.

Food and Drug Administration, or FDA, for the treatment of hemolytic anemia in adults with PK deficiency in the United States and by the European Commission for the treatment of PK deficiency in adult patients in the European Union, or EU.

Additionally, we received marketing authorization in Great Britain for PYRUKYND® for the treatment of PK deficiency in adult patients under the European Commission Decision Reliance Procedure.

In December 2024, we announced that we submitted a supplemental new drug application, or sNDA, to the FDA for PYRUKYND® for the treatment of adult patients with non-transfusion dependent and transfusion-dependent alpha- or beta-thalassemia.

The FDA accepted the sNDA and granted a Prescription Drug User Fee Act, or PDUFA, goal date of September 7, 2025, which the FDA subsequently extended to December 7, 2025 following our submission of a proposed Risk Evaluation and Mitigation Strategy, or REMS, to mitigate the risk of hepatocellular injury that was described in the original sNDA filing.

The submission of the REMS is a major amendment to the PYRUKYND® sNDA, resulting in the three-month review extension.

The extension was not the result of new or additional efficacy or safety data requested by the FDA or submitted by us.

In December 2024, we also announced that we submitted a marketing authorization application, or MAA, to the European Medicines Agency, or EMA, and regulatory applications to the Kingdom of Saudi Arabia and the United Arab Emirates health authorities for PYRUKYND® for the treatment of adult patients with non-transfusion dependent and transfusion-dependent alpha- or beta-thalassemia.

In August 2025, we announced that the Saudi Food and Drug Authority approved PYRUKYND® for the treatment of adult patients with non-transfusion dependent and transfusion-dependent alpha- or beta-thalassemia.

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 will now review the CHMP’s opinion, with the final decision expected by early 2026.

In addition, we are currently evaluating PYRUKYND® for the treatment of sickle cell disease, or SCD and in pediatric patients with PK deficiency.

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;

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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.

Alnylam License Agreement

On July 28, 2023, we entered into a license agreement with Alnylam under which we acquired the rights to develop and commercialize Alnylam’s novel preclinical siRNA targeting the TMPRSS6 gene, as a potential disease-modifying treatment for patients with PV.

In accordance with the license agreement, we are responsible to pay up to $ 130.0 million in potential development and regulatory milestones, in addition to sales milestones as well as tiered royalties on annual net sales, if any, of licensed products, which may be subject to specified reductions and offsets.

In the nine months ended September 30, 2025, we achieved a regulatory milestone that triggered a $ 10.0 million payment to Alnylam, which we recorded within research and development expenses in our condensed consolidated statements of operations.

Sale of Oncology Business to Servier and Sale of Contingent Payments

On March 31, 2021, we completed the sale of our oncology business to Servier Pharmaceuticals, LLC, or Servier, which represented a discontinued operation.

The transaction included the sale of our oncology business, including TIBSOVO®, our clinical-stage product candidates vorasidenib, AG-270 and AG-636, and our oncology research programs, for a payment of approximately $ 1.8 billion in cash at the closing, subject to certain adjustments, and a payment of $ 200.0 million in cash, if, prior to January 1, 2027, vorasidenib is granted new drug application, or NDA, approval from the FDA with an approved label that permits vorasidenib’s use as a single agent for the adjuvant treatment of patients with Grade 2 glioma that have an isocitrate dehydrogenase, or IDH, 1 or 2 mutation (and, to the extent required by such approval, the vorasidenib companion diagnostic test is granted an FDA premarket approval), or the Vorasidenib Milestone Payment, as well as a royalty of 5 % of U.S.

net sales of TIBSOVO® from the close of the transaction through loss of exclusivity, and a royalty of 15 % of U.S.

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 Milestone Payment, Vorasidenib Royalty Rights and royalty payments related to TIBSOVO® are referred to as contingent payments and recognized as income when realizable.

Servier also acquired our co-commercialization rights for Bristol Myers Squibb’s IDHIFA® and the right to receive a $ 25.0 million potential milestone payment under our prior collaboration agreement with Celgene Corporation, or Celgene, and following the sale Servier has agreed to conduct certain clinical development activities within the IDHIFA® development program.

In October 2022, we sold our rights to future contingent payments associated with the royalty of 5 % of U.S.

net sales of TIBSOVO® from the close of the transaction through the loss of exclusivity to entities affiliated with Sagard Healthcare Partners, or Sagard, and recognized income of $ 127.9 million in our consolidated statements of operations for the year ended December 31, 2022.

In August 2024, the FDA approved vorasidenib for adult and pediatric patients 12 years and older with Grade 2 astrocytoma or oligodendroglioma with a susceptible IDH1 or IDH2 mutation, following surgery including biopsy, sub-total resection, or gross total resection.

In September 2024, we received the Vorasidenib Milestone Payment from Servier and recognized income of $ 200.0 million within the milestone payment from gain on sale of oncology business line item in our consolidated statements of operations for the three months ended September 30, 2024.

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, for $ 905.0 million in cash, or the Upfront Payment.

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.

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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.

As a result of the sale, we recognized income of $ 889.1 million ($ 905.0 million net of fees of $ 15.9 million) within the gain on sale of contingent payments line item in our consolidated statements of operations for the three months ended September 30, 2024.

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 September 30, 2025, the condensed consolidated statements of operations, comprehensive loss and stockholders' equity for the three and nine months ended September 30, 2025 and 2024, and the condensed consolidated statements of cash flows for the nine months ended September 30, 2025 and 2024 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 September 30, 2025, our results of operations and stockholders' equity for the three and nine months ended September 30, 2025 and 2024, and cash flows for the nine months ended September 30, 2025 and 2024.

The financial data and other financial information disclosed in these notes to the condensed consolidated financial statements related to the three and nine-month periods are also unaudited.

The results of operations for the three and nine months ended

September 30, 2025 are not necessarily indicative of the results to be expected for the year ending December 31, 2025 or for any other future annual or interim period.

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.

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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.

As of September 30, 2025, we had cash, cash equivalents and marketable securities of $ 1.3 billion.

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.

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Recent Accounting Pronouncements

Accounting standards that have been issued by the Financial Accounting Standards Board or other standards-setting bodies that do not require adoption until a future date are not expected to have a material impact on our financial statements upon adoption.

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 statements a disaggregation of certain expense categories included within the expense captions on the face of the income statement.

The standard is effective for annual periods beginning after December 15, 2026 and interim periods beginning after December 15, 2027, with early adoption permitted, and can be applied either prospectively or retrospectively.

We plan to adopt the standard in our 2027 annual period and are currently assessing its effect on our financial statement disclosures.

In September 2025, the FASB issued ASU No.

2025-07 Derivatives Scope Refinements and Scope Clarification for Share-Based Noncash Consideration from a Customer in a Revenue Contract .

The amendments provide for a new scope exception to the derivatives guidance for underlyings based on the operations or activities specific to one of the parties to the contract, and also clarifies that share-based noncash consideration received from a customer as consideration for the transfer of goods or services in a revenue contract is subject to the revenue guidance and not the financial instruments guidance unless and until the company’s right to receive or retain the share-based noncash consideration is unconditional as defined in the ASU.

The amendments are effective for annual reporting periods beginning after December 15, 2026, including interim periods within those fiscal years, with early adoption permitted.

We adopted the standard prospectively in the first quarter of 2026 with no material impact to our business, financial condition or results of operations.

Fair Value Measurements

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Level 3 – Unobservable inputs that reflect our own assumptions about the assumptions market participants would use in pricing the asset or liability in which there is little, if any, market activity for the asset or liability at the measurement date.

The following table summarizes our cash equivalents and marketable securities measured at fair value and by level on a recurring basis as of September 30, 2025:

The following table summarizes our cash equivalents and marketable securities measured at fair value and by level on a recurring basis as of March 31, 2026:

(In thousands) Level 1 Level 2 Level 3 Total

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The pricing services utilize industry standard valuation models, including both income and market-based approaches, and observable market inputs to determine value.

After completing our validation procedures, we did not adjust or override any fair value measurements provided by the pricing services as of September 30, 2025.

There have been no changes to the valuation methods during the nine months ended September 30, 2025, and we had no financial assets or liabilities that were classified as Level 3 at any point during the nine months ended September 30, 2025.

After completing our validation procedures, we did not adjust or override any fair value measurements provided by the pricing services as of March 31, 2026.

There have been no changes to the valuation methods during the three months ended March 31, 2026, and we had no financial assets or liabilities that were classified as Level 3 at any point during the three months ended March 31, 2026.

Marketable Securities

Our marketable securities are classified as available-for-sale pursuant to ASC 320, Investments – Debt and Equity Securities , and are recorded at fair value.

Unrealized gains and losses are included as a component of accumulated other comprehensive income (loss) in the condensed consolidated balance sheets and statements of stockholders’ equity, and a component of total comprehensive income (loss) in the condensed consolidated statements of comprehensive (loss) income, until realized.

Unrealized gains and losses are included as a component of accumulated other comprehensive (loss) income in the condensed consolidated balance sheets and statements of stockholders’ equity, and a component of total comprehensive loss in the condensed consolidated statements of comprehensive loss, until realized.

Unrealized losses are evaluated for impairment under ASC 326, Financial Instruments - Credit Losses , to determine if the impairment is credit-related or noncredit-related.

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Realized gains and losses are included in investment income on a specific-identification basis.

There were no material realized gains or losses on marketable securities for the three and nine months ended September 30, 2025 or 2024.

Marketable securities at September 30, 2025 consisted of the following:

There were no material realized gains or losses on marketable securities for the three months ended March 31, 2026 or 2025.

Marketable securities at March 31, 2026 consisted of the following:

(In thousands) Amortized

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Total marketable securities $ 1,073,115 $ 2,254 $ ( 61 ) $ 1,075,308

As of September 30, 2025 and December 31, 2024, we held both current and non-current investments.

As of March 31, 2026 and December 31, 2025, we held both current and non-current investments.

Investments classified as current have maturities of less than one year.

Investments classified as non-current are those that:

(i) have a maturity of greater than one year, and (ii) we do not intend to liquidate within the next twelve months, although these funds are available for use and, therefore, are classified as available-for-sale.

As of September 30, 2025 and December 31, 2024, we held 58 and 213 debt securities, respectively, that were in an unrealized loss position for less than one year.

We did no t record an allowance for credit losses as of September 30, 2025 and December 31, 2024 related to these securities.

The aggregate fair value of debt securities in an unrealized loss position at September 30, 2025 and December 31, 2024 was $ 288.9 million and $ 768.1 million, respectively.

There were no individual securities that were in a significant unrealized loss position as of September 30, 2025 and December 31, 2024.

(i) have a maturity of greater

than one year, and (ii) we do not intend to liquidate within the next twelve months, although these funds are available for use and, therefore, are classified as available-for-sale.

As of March 31, 2026 and December 31, 2025, we held 112 and 37 debt securities, respectively, that were in an unrealized loss position for less than one year.

We did no t record an allowance for credit losses as of March 31, 2026 and December 31, 2025 related to these securities.

The aggregate fair value of debt securities in an unrealized loss position at March 31, 2026 and December 31, 2025 was $ 461.4 million and $ 136.2 million, respectively.

There were no individual securities that were in a significant unrealized loss position as of March 31, 2026 and December 31, 2025.

We regularly review the securities in an unrealized loss position and evaluate the current expected credit loss by considering factors such as historical experience, market data, issuer-specific factors, and current economic conditions.

We do not consider these marketable securities to be impaired as of September 30, 2025 and December 31, 2024.

Inventory, which consists of commercial supply of PYRUKYND®, consisted of the following:

(In thousands) September 30,

We do not consider these marketable securities to be impaired as of March 31, 2026 and December 31, 2025.

Inventory, which consists of commercial supply of PYRUKYND® and AQVESME™, consisted of the following:

(In thousands) March 31,

2026 December 31,

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The components of lease expense and other information related to leases were as follows:

Three Months Ended September 30, Nine Months Ended September 30,

Three Months Ended March 31,

(In thousands) 2026 2025

1 unchanged sentence

Cash paid for amounts included in the measurement of operating lease liabilities 5,012 4,837

We have not entered into any material short-term leases or financing leases as of September 30, 2025.

In arriving at the operating lease liabilities as of September 30, 2025 and December 31, 2024, we applied the weighted-average incremental borrowing rate of 5.7 % for both periods over a weighted-average remaining lease term of 2.4 and 3.2 years, respectively.

As of September 30, 2025, undiscounted minimum rental commitments under non-cancelable leases were as follows:

We have not entered into any material short-term leases or financing leases as of March 31, 2026.

In arriving at the operating lease liabilities as of March 31, 2026 and December 31, 2025, we applied the weighted-average incremental borrowing rate of 5.7 % for both periods over a weighted-average remaining lease term of 1.9 and 2.2 years, respectively.

As of March 31, 2026, undiscounted minimum rental commitments under non-cancelable leases were as follows:

(In thousands)

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We provided our landlord a security deposit of $ 2.9 million as security for our leases, which is included within other non-current assets on our condensed consolidated balance sheet.

In August 2021, we entered into a long-term sublease agreement for 13,000 square feet of the office space at 38 Sidney Street, Cambridge, Massachusetts, which expired on December 31, 2024.

In April 2022, we entered into a long-term sublease agreement for 27,000 square feet of the office space at 64 Sidney Street, Cambridge, Massachusetts, which expired on April 30, 2025.

In May 2023, we entered into a long-term sublease agreement for 7,407 square feet of office space on the first floor of 64 Sidney Street, Cambridge, Massachusetts, which expired on July 31, 2025.

In July 2025, we entered into a long-term sublease agreement with a new tenant for the same space, which will begin in November 2025 with the term of the lease running through February 2028.

We recorded operating sublease income of less than $ 0.1 million and $ 1.6 million for the three months ended September 30, 2025 and 2024, respectively, and $ 1.8 million and $ 4.8 million for the nine months ended September 30, 2025 and 2024, respectively, in other income, net in the condensed consolidated statements of operations.

In July 2025, we entered into a long-term sublease agreement with a new tenant for the same space, which began in November 2025 with the term of the lease running through February 2028.

We recorded operating sublease income of $ 0.1 million and $ 1.3 million for the three months ended March 31, 2026 and 2025, respectively, in other income, net in the condensed consolidated statements of operations.

We hold security deposits from our sublessee of approximately $ 0.1 million which is recorded within other non-current assets on our condensed consolidated balance sheet.

As of September 30, 2025, the future minimum lease payments to be received under the long-term sublease agreements were as follows:

As of March 31, 2026, the future minimum lease payments to be received under the long-term sublease agreements were as follows:

(In thousands)

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Accrued expenses consisted of the following:

(In thousands) September 30,

(In thousands) March 31,

2026 December 31,

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Product Revenue

We generate product revenue from sales of PYRUKYND® to a limited number of specialty distributors and specialty pharmacy providers, or collectively, the Customers.

These Customers subsequently resell PYRUKYND® to pharmacies or dispense PYRUKYND® directly to patients.

In addition to distribution agreements with Customers, we enter into arrangements with healthcare providers and payors that provide for government-mandated and/or privately-negotiated rebates, chargebacks and discounts with respect to the purchase of PYRUKYND®.

The performance obligation related to the sale of PYRUKYND® is satisfied and revenue is recognized when the Customer obtains control of the product, which occurs at a point in time, typically upon delivery to the Customer.

Product revenue, net, were as follows:

Three Months Ended September 30, Nine Months Ended September 30,

We generate product revenue from sales of PYRUKYND® and AQVESME™ in the United States to a limited number of specialty distributors and specialty pharmacy providers, and from sales of PYRUKYND® to Avanzanite and NewBridge outside of the United States, or collectively, the Customers.

These Customers subsequently resell PYRUKYND® and AQVESME™ to pharmacies or dispense PYRUKYND® and AQVESME™ directly to patients.

The performance obligation related to the sale of PYRUKYND® and AQVESME™ is satisfied and revenue is recognized when the Customer obtains control of the product, which occurs at a point in time, typically upon delivery to the Customer.

Product revenue, net, was as follows:

Three Months Ended March 31,

(In thousands) 2026 2025

Product revenue, net

United States $ 18,851 $ 8,726

Rest of world 1,895 —

Total product revenue, net $ 20,746 $ 8,726

Reserves for Variable Consideration

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We currently estimate product return and replacement liabilities using the expected value method, based on available industry data, including our visibility into the inventory remaining in the distribution channel.

The following table summarizes balances and activity in each of the product revenue allowance and reserve categories for the nine months ended September 30, 2025:

The following table summarizes balances and activity in each of the product revenue allowance and reserve categories for the three months ended March 31, 2026:

(In thousands) Contractual Adjustments Government Rebates Returns/ Replacement Total

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Current provisions relating to sales in the current year 759 1,216 210 2,185

Adjustments relating to prior years ( 51 ) ( 84 ) — ( 135 )

Payments/returns relating to sales in the current year ( 311 ) — — ( 311 )

Payments/returns relating to sales in the prior years ( 166 ) ( 833 ) ( 16 ) ( 1,015 )

Balance at September 30, 2025 $ 111 $ 1,898 $ 880 $ 2,889

Balance at March 31, 2026 $ 464 $ 1,657 $ 1,234 $ 3,355

Total revenue-related reserves above, included in our condensed consolidated balance sheets, are summarized as follows:

(In thousands) September 30, 2025 December 31, 2024

(In thousands) March 31, 2026 December 31, 2025

Reduction of accounts receivable $ 208 $ 174

1 unchanged sentence

Total revenue-related reserves $ 3,355 $ 2,496

The following table presents changes in our contract assets during the nine months ended September 30, 2025:

(In thousands) December 31, 2024 Additions Deductions September 30, 2025

The following table presents changes in our contract assets during the three months ended March 31, 2026:

(In thousands) December 31, 2025 Additions Deductions March 31, 2026

Contract assets (1)

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In connection with the start of employment of our Chief Executive Officer and Chief Financial Officer in 2022, our Chief Commercial Officer in 2023, and our Chief Corporate Development and Strategy Officer in March 2025, our Board of Directors granted each of them equity awards in the form of stock options, RSUs and PSUs, which awards were made outside our equity incentive plans as inducements material to their respective entry into employment with us in accordance with Nasdaq Listing Rule 5635(c)(4).

As of September 30, 2025, the maximum number of shares reserved under the 2013 Plan, the 2023 Plan and the inducement grants described above was 12,501,715 , and we had 3,697,640 shares available for future issuance under the 2023 Plan.

As of March 31, 2026, the maximum number of shares reserved under the 2013 Plan, the 2023 Plan and the inducement grants described above was 11,498,895 , and we had 1,977,151 shares available for future issuance under the 2023 Plan.

Stock options

The following table presents stock option activity for the nine months ended September 30, 2025:

The following table presents stock option activity for the three months ended March 31, 2026:

Stock Options Weighted-Average

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Expired ( 90,190 ) 39.77

Outstanding at September 30, 2025 6,271,947 $ 39.58

Exercisable at September 30, 2025 4,232,483 $ 43.19

Vested and expected to vest at September 30, 2025 6,271,947 $ 39.58

At September 30, 2025, there was approximately $ 32.6 million of total unrecognized compensation expense related to unvested stock option awards, which we expect to recognize over a weighted-average period of approximately 2.46 years.

Outstanding at March 31, 2026 6,646,266 $ 38.45

Exercisable at March 31, 2026 4,515,842 $ 41.76

Vested and expected to vest at March 31, 2026 6,646,266 $ 38.45

At March 31, 2026, there was approximately $ 34.5 million of total unrecognized compensation expense related to unvested stock option awards, which we expect to recognize over a weighted-average period of approximately 2.85 years.

Restricted stock units

The following table presents RSU activity for the nine months ended September 30, 2025:

The following table presents RSU activity for the three months ended March 31, 2026:

Stock Units Weighted-Average

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Forfeited ( 105,231 ) 33.29

Unvested shares at September 30, 2025 2,055,122 $ 31.98

As of September 30, 2025, there was approximately $ 47.0 million of total unrecognized compensation expense related to RSUs, which we expect to recognize over a weighted-average period of approximately 1.94 years.

Unvested shares at March 31, 2026 2,283,500 $ 31.25

As of March 31, 2026, there was approximately $ 63.5 million of total unrecognized compensation expense related to RSUs, which we expect to recognize over a weighted-average period of approximately 2.23 years.

Performance-based stock units

The following table presents PSU activity for the nine months ended September 30, 2025:

The following table presents PSU activity for the three months ended March 31, 2026:

Stock Units Weighted-Average

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Granted 83,500 29.20

Vested ( 83,027 ) 26.35

Unvested shares at September 30, 2025 477,006 $ 31.47

Stock-based compensation expense associated with these PSUs is recognized if the underlying performance condition is considered probable of achievement using our management’s best estimates.

As of September 30, 2025, there was no unrecognized compensation expense related to PSUs with performance-based vesting criteria that are considered probable of achievement, and $ 15.0 million of total unrecognized compensation expense related to PSUs with performance-based vesting criteria that are considered not probable of achievement.

2013 Employee Stock Purchase Plan

In June 2013, our Board of Directors adopted, and in July 2013 our stockholders approved, the 2013 Employee Stock Purchase Plan, or the 2013 ESPP.

We issued and sold 133,283 and 102,805 shares of common stock during the nine months ended September 30, 2025 and 2024, respectively, under the 2013 ESPP.

Unvested shares at March 31, 2026 460,478 $ 31.95

Included in unvested shares were 103,128 shares with performance-based vesting criteria that were considered probable of achievement at March 31, 2026 and vested in April 2026.

Stock-based compensation expense associated with performance-based PSUs is recognized if the underlying performance condition is considered probable of achievement using our management’s best estimates.

As of March 31, 2026, there was no unrecognized compensation expense related to PSUs with performance-based vesting criteria that are considered probable of achievement, and $ 11.4 million of total unrecognized compensation expense related to PSUs with performance-based vesting criteria that are considered not probable of achievement.

Market-based stock units

We have issued certain equity awards that contain market based vesting conditions, in which shares of stock are earned at vesting based on total shareholder return compared to an industry stock index.

The fair value of market-based stock units, or MSUs, is estimated using a Monte Carlo simulation model.

Assumptions and estimates utilized in the model include the risk-free interest rate, dividend yield, expected stock volatility and the service period.

The following table presents MSU activity for the three months ended March 31, 2026:

Stock Units Weighted-Average

Grant Date Fair

Unvested shares at December 31, 2025 — $ —

Granted 131,500 49.34

Unvested shares at March 31, 2026 131,500 $ 49.34

As of March 31, 2026, there was $ 6.3 million of unrecognized compensation expense related to MSUs with market-based vesting criteria, which we expect to recognize over a weighted-average period of approximately 2.75 years.

Amended and Restated 2013 Employee Stock Purchase Plan

In June 2013, our Board of Directors adopted, and in July 2013 our stockholders approved, the 2013 ESPP, which was further amended and restated by our Board of Directors in December 2024.

We issued and sold 65,809 and 52,092 shares of common stock during the three months ended March 31, 2026 and 2025, respectively, under the 2013 ESPP.

The 2013 ESPP provides participating employees with the opportunity to purchase up to an aggregate of 2,363,636 shares of our common stock.

As of September 30, 2025, we had 1,449,951 shares of common stock available for future issuance under the 2013 ESPP.

As of March 31, 2026, we had 1,384,142 shares of common stock available for future issuance under the 2013 ESPP.

Stock-based compensation expense

1 unchanged sentence

Three Months Ended

September 30, Nine Months Ended

September 30,

(In thousands) 2026 2025

2 unchanged sentences

Performance-based stock units 3,268 —

Market-based stock units 194 —

Employee stock purchase plan 380 275

1 unchanged sentence

Expenses related to stock options and stock-based awards were allocated as follows in the condensed consolidated statements of operations:

Three Months Ended September 30, Nine Months Ended September 30,

Three Months Ended March 31,

(In thousands) 2026 2025

2 unchanged sentences

Total stock-based compensation expense $ 15,541 $ 11,359

Net (Loss) Income per Share

Basic net (loss) income per share is calculated by dividing net (loss) income by the weighted-average shares outstanding during the period, without consideration for common stock equivalents.

Diluted net (loss) income per share is calculated by adjusting the weighted-average shares outstanding for the dilutive effect of common stock equivalents outstanding for the period, determined using the treasury stock method.

For purposes of the dilutive net (loss) income per share calculation, stock options, RSUs and PSUs for which the performance and market vesting conditions, respectively, have been deemed probable, and 2013 ESPP shares are considered to be common stock equivalents, while PSUs with performance and market vesting conditions, respectively, that were not deemed probable as of September 30, 2025 are not considered to be common stock equivalents.

Net Loss per Share

Basic net loss per share is calculated by dividing net loss by the weighted-average shares outstanding during the period, without consideration for common stock equivalents.

Diluted net loss per share is calculated by adjusting the weighted-average shares outstanding for the dilutive effect of common stock equivalents outstanding for the period, determined using the treasury stock method.

For purposes of the dilutive net loss per share calculation, stock options, RSUs and PSUs for which the performance and market vesting conditions, respectively, have been deemed probable, and 2013 ESPP shares are considered to be common stock equivalents, while PSUs with performance and market vesting conditions, respectively, that were not deemed probable as of March 31, 2026 are not considered to be common stock equivalents.

We utilize the control number concept in the computation of diluted earnings per share to determine whether potential common stock equivalents are dilutive.

The control number used is net (loss) income from continuing operations.

The control number used is net loss from continuing operations.

The control number concept requires that the same number of potentially dilutive securities applied in computing diluted earnings per share from continuing operations be applied to all other categories of income or loss, regardless of their anti-dilutive effect on such categories.

Since we had a net loss for the three and nine months ended September 30, 2025, no dilutive effect was recognized in the calculation of loss per share and basic and diluted net loss per share was the same for those periods.

The following is a reconciliation of basic weighted-average number of common shares used in computing net (loss) income per share to diluted weighted-average number of common shares used in computing net (loss) income per share for the periods indicated:

Three Months Ended September 30, Nine Months Ended September 30,

2025 2024 2025 2024

Basic shares 58,139,277 56,939,403 57,846,173 56,709,318

Effect of dilutive securities

Since we had a net loss for all period presented, no dilutive effect was recognized in the calculation of loss per share and basic and diluted net loss per share was the same for those periods.

The following common stock equivalents were excluded from the calculation of diluted net loss per share applicable to common stockholders for the periods indicated because including them would have had an anti-dilutive effect:

Three Months Ended March 31,

Stock options 6,646,266 6,378,183

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Performance-based stock units 103,128 —

Employee stock purchase plan shares — 8,944 — 2,981

Diluted shares 58,139,277 58,432,796 57,846,173 57,581,382

The following common stock equivalents were excluded from the calculation of diluted net (loss) income per share applicable to common stockholders for the periods indicated because including them would have had an anti-dilutive effect:

Three Months Ended September 30, Nine Months Ended September 30,

2025 2024 2025 2024

Stock options 6,271,947 3,463,344 6,271,947 5,176,610

Restricted stock units 2,055,122 — 2,055,122 90,480

Employee stock purchase plan shares 81,191 3,139 81,191 4,393

Total common stock equivalents 9,032,894 8,549,710

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Our CODM is our CEO.

Our CEO views our operations and manages our business as one operating segment, which derives its revenues from the development and commercialization of therapies for patients with rare diseases.

Our CEO views our operations and manages our

business as one operating segment, which derives its revenues from the development and commercialization of therapies for patients with rare diseases.

Our CEO manages and allocates resources to the operations of our company on a total company basis by assessing the overall level of resources available and how to best deploy these resources across functions and research and development projects that are in line with our long-term company-wide strategic goals.

In making these decisions, our CEO uses consolidated financial information for purposes of evaluating performance, forecasting future period financial results, allocating resources and setting incentive targets.

The CODM performs this assessment based on our consolidated net income (loss).

Through this analysis, the CODM assesses performance by comparing actual consolidated net income (loss) versus the budget, and then decides how to allocate resources to invest in our research and development programs.

The CODM performs this assessment based on our consolidated net (loss) income.

Through this analysis, the CODM assesses performance by comparing actual consolidated net (loss) income versus the budget, and then decides how to allocate resources to invest in our research and development programs.

The measure of segment assets is reported on the condensed consolidated balance sheets as total assets.

The following table contains additional information on our consolidated revenue and net loss, including significant segment expenses:

Three Months Ended September 30, Nine Months Ended September 30,

Three Months Ended March 31,

(In thousands) 2026 2025

Product revenue, net $ 12,880 $ 8,964 $ 34,061 $ 25,768

PK activator (PYRUKYND®) direct expenses - research and development ( 30,533 ) ( 26,829 ) ( 81,663 ) ( 80,833 )

Product revenue, net - U.S.

$ 18,851 $ 8,726

Product revenue, net - Rest of world 1,895 —

PK activator (PYRUKYND®/AQVESME™) direct expenses - research and development ( 23,905 ) ( 21,321 )

Compensation and related expenses - research and development ( 36,789 ) ( 32,990 )

Total selling, general and administrative expenses ( 48,304 ) ( 41,527 )

Gain on sale of contingent payments — 889,136 — 889,136

Milestone payment from gain on sale of oncology business — 200,000 — 200,000

Other segment items* ( 10,859 ) ( 2,177 )

Net (loss) income $ ( 103,433 ) $ 947,915 $ ( 304,742 ) $ 770,248

*Other segment items primarily include cost of sales, other research and development expenses, interest income, and income tax expense.

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

*Other segment items primarily include cost of sales, other research and development expenses, and interest income.

Compared sentence by sentence after normalising whitespace, quotation marks, case and digits, so re-formatting and restated figures do not read as changed language. Wording changes appear as one removal and one addition. The current filing and the prior one are authoritative.

What changed 10-Q

Item 1. Financial Statements