What changed in this filing

/stocks — the workspaceLOADING

Item 9A. Controls and Procedures

2026-02-12 compared with 2025-02-13 · 179 added, 169 removed, 891 unchanged (28% of the section changed)

2 unchanged sentences

Our management, with the participation of our principal executive officer and our principal financial officer, evaluated, as of the end of the period covered by this Annual Report on Form 10-K, the effectiveness of our disclosure controls and procedures.

Based on that evaluation of our disclosure controls and procedures as of December 31, 2024, our principal executive officer and

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principal financial officer concluded that our disclosure controls and procedures as of such date are effective at the reasonable assurance level.

Based on that evaluation of our disclosure controls and procedures as of December 31, 2025, our principal executive officer and principal financial officer concluded that our disclosure controls and procedures as of such date are effective at the reasonable assurance level.

The term “disclosure controls and procedures,” as defined in Rules 13a-15(e) and 15d-15(e) under the Securities Exchange Act of 1934, as amended, or the Exchange Act, means controls and other procedures of a company that are designed to ensure that information required to be disclosed by a company in the reports that it files or submits under the Exchange Act are recorded, processed, summarized and reported within the time periods specified in the SEC’s rules and forms.

32 unchanged sentences

Aggregate Number of Securities

Cecilia Jones ( Chief Financial Officer )

Adoption ( December 13, 2024 )

Rule 10b5-1 trading arrangement

Through and including March 17, 2026

Up to 24,779 shares

Tsveta Milanova ( Chief Commercial Officer )

Adoption ( December 13, 2024 )

Rahul Ballal ( Director )

Adoption ( November 3, 2025 )

Rule 10b5-1 trading arrangement

Through and including March 17, 2026

Through and including February 8, 2027

Up to 28,995 shares

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Consolidated Statements of Operations

Consolidated Statements of Comprehensive Income (Loss)

Consolidated Statements of Comprehensive (Loss) Income

Consolidated Statements of Stockholders’ Equity

17 unchanged sentences

Description of Securities Registered Under Section 12 of the Securities Exchange Act of 1934

February 19, 2020

2013 Stock Incentive Plan

5 unchanged sentences

Amended and Restated 2013 Employee Stock Purchase Plan

February 13, 2025 10.4

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25 unchanged sentences

August 1, 2019

10.19 Sublease Agreement, dated July 27, 2021, between the Registrant and Prime Medicine, Inc.

(64 Sidney Street)

February 24, 2022

10.20 Sublease Agreement, dated April 14, 2023, between the Registrant and Watershed Informatics, Inc.

February 15, 2024

Letter Agreement, dated July 8, 2022, between the Registrant and Brian Goff

4 unchanged sentences

August 4, 2022

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10.22#** Form of Inducement Performance Stock Unit Agreement for Brian Goff

2 unchanged sentences

November 3, 2022

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Form of Inducement Stock Option Agreement for Cecilia Jones

14 unchanged sentences

January 3, 2023

10.34# 2023 Stock Incentive Plan

10.32# 2023 Stock Incentive Plan , as amended

333-288151 June 18, 2025 99.1

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10-Q 001-36014 August 1, 2024 10.1

10.37# Letter Agreement, dated January 31, 2025, between the Registrant and Krishnan Viswanadhan

10.38# Form of Inducement Stock Option Agreement for Krishnan Viswanadhan

S-8 333-285585 March 5, 2025 99.1

10.39# Form of Inducement Restricted Stock Unit Agreement for Krishnan Viswanadhan

S-8 333-285585 March 5, 2025 99.2

10.40# Form of Inducement Performance Stock Unit Agreement for Krishnan Viswanadhan

S-8 333-285585 March 5, 2025 99.3

10.41 Sublease Agreement, dated June 5, 2025, between the Registrant and GNS Healthcare, Inc.

d/b/a Aitia (64 Sidney Street)

001-36014 July 31, 2025 10.2

19.1 Insider Trading Policy

001-36014 February 13, 2025 19.1

Subsidiaries of the Registrant

1 unchanged sentence

Certification of principal executive officer pursuant to Rule 13a-14(a)/15d-14(a) of the Securities Exchange Act of 1934, as amended

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Certification of principal financial officer pursuant to Rule 13a-14(a)/15d-14(a) of the Securities Exchange Act of 1934, as amended.

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§1350, as adopted pursuant to Section 906 of the Sarbanes-Oxley Act of 2002.

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Certification of principal financial officer pursuant to 18 U.S.C.

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Fouse Chair of the Board of Directors February 12, 2026

/s/ Jay Backstrom Director February 12, 2026

Jay Backstrom, M.D., M.P.H.

/s/ Rahul Ballal Director February 12, 2026

5 unchanged sentences

Maykin Ho, Ph.D.

/s/ Catherine Owen Director February 13, 2025

Catherine Owen

/s/ Catherine Owen Adams Director February 12, 2026

Catherine Owen Adams

/s/ David Scadden Director February 12, 2026

David Scadden, M.D.

Schenkein Director February 13, 2025

Schenkein, M.D.

/s/ Cynthia Smith Director February 12, 2026

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Consolidated Statements of Operations

Consolidated Statements of Comprehensive Income (Loss)

Consolidated Statements of Comprehensive (Loss) Income

Consolidated Statements of Stockholders’ Equity

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Additional paid-in capital 2,555,037 2,493,811

Accumulated other comprehensive loss ( 1,518 ) ( 441 )

Accumulated other comprehensive income (loss) 2,193 ( 1,518 )

Accumulated deficit ( 561,705 ) ( 148,924 )

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Product revenue, net $ 54,028 $ 36,498 $ 26,823

Milestone revenue — — 2,500

Total revenue 54,028 36,498 26,823

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

Royalty income from gain on sale of oncology business — — 9,851

Interest income, net 56,379 48,083 33,344

Other income, net 1,956 6,487 6,055

Net income (loss) before taxes 717,969 ( 352,088 ) ( 231,801 )

Income tax expense 44,244 — —

Net income (loss) $ 673,725 $ ( 352,088 ) $ ( 231,801 )

Net income (loss) per share - basic $ 11.86 $ ( 6.33 ) $ ( 4.23 )

Net income (loss) per share - diluted $ 11.64 $ ( 6.33 ) $ ( 4.23 )

Weighted-average number of common shares used in computing net income (loss) per share – basic 56,807,415 55,651,487 54,789,435

Weighted-average number of common shares used in computing net income (loss) per share – diluted 57,889,255 55,651,487 54,789,435

Net (loss) income before taxes ( 413,797 ) 717,969 ( 352,088 )

Income tax (benefit) expense ( 1,016 ) 44,244 —

Net (loss) income $ ( 412,781 ) $ 673,725 $ ( 352,088 )

Net (loss) income per share - basic $ ( 7.12 ) $ 11.86 $ ( 6.33 )

Net (loss) income per share - diluted $ ( 7.12 ) $ 11.64 $ ( 6.33 )

Weighted-average number of common shares used in computing net (loss) income per share – basic 57,972,004 56,807,415 55,651,487

Weighted-average number of common shares used in computing net (loss) income per share – diluted 57,972,004 57,889,255 55,651,487

See accompanying Notes to Consolidated Financial Statements.

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Agios Pharmaceuticals, Inc.

Consolidated Statements of Comprehensive Income (Loss)

Consolidated Statements of Comprehensive (Loss) Income

(In thousands) Years Ended December 31:

2025 2024 2023

Net income (loss) $ 673,725 $ ( 352,088 ) $ ( 231,801 )

Net (loss) income $ ( 412,781 ) $ 673,725 $ ( 352,088 )

Other comprehensive (loss) income

Unrealized (loss) gain on available-for-sale securities ( 1,077 ) 12,094 ( 11,337 )

Comprehensive income (loss) $ 672,648 $ ( 339,994 ) $ ( 243,138 )

Unrealized gain (loss) on available-for-sale securities 3,711 ( 1,077 ) 12,094

Comprehensive (loss) income $ ( 409,070 ) $ 672,648 $ ( 339,994 )

See accompanying Notes to Consolidated Financial Statements.

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Balance at December 31, 2022 71,256,118 $ 71 $ 2,386,325 $ ( 12,535 ) $ ( 470,561 ) ( 16,216,411 ) $ ( 802,486 ) $ 1,100,814

Unrealized loss on available-for-sale securities — — — ( 11,337 ) — — — ( 11,337 )

Net loss — — — — ( 231,801 ) — — ( 231,801 )

Stock-based compensation expense — — 49,296 — — — — 49,296

Common stock issued under stock incentive plan and ESPP 705,487 — 2,681 — — — — 2,681

Balance at December 31, 2022 71,256,118 $ 71 $ 2,386,325 $ ( 12,535 ) $ ( 470,561 ) ( 16,216,411 ) $ ( 802,486 ) $ 1,100,814

Unrealized gain on available-for-sale securities — — — 12,094 — — — 12,094

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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 gain on available-for-sale securities — — — 3,711 — — — 3,711

Net loss — — — — ( 412,781 ) — — ( 412,781 )

Stock-based compensation expense — — 52,552 — — — — 52,552

Common stock issued under stock incentive plan and ESPP 1,292,307 2 8,674 — — — — 8,676

Balance at December 31, 2025 74,665,003 $ 75 $ 2,555,037 $ 2,193 $ ( 561,705 ) ( 16,216,411 ) $ ( 802,486 ) $ 1,193,114

See accompanying Notes to Consolidated Financial Statements.

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Operating activities

Net income (loss) $ 673,725 $ ( 352,088 ) $ ( 231,801 )

Adjustments to reconcile net income (loss) to net cash used in operating activities:

Net (loss) income $ ( 412,781 ) $ 673,725 $ ( 352,088 )

Adjustments to reconcile net (loss) income to net cash used in operating activities:

Depreciation and amortization 5,177 5,653 6,623

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Inventory ( 5,304 ) ( 8,540 ) ( 10,584 )

Other receivables — — 447

Prepaid expenses and other current and non-current assets ( 3,172 ) ( 5,811 ) 3,833

15 unchanged sentences

Financing activities

Payments on financing lease obligations — — ( 331 )

Net proceeds from stock option exercises and employee stock purchase plan 8,676 14,442 5,433

11 unchanged sentences

Nature of Business

References to Agios

Throughout this Annual Report on Form 10-K, “the Company,” “Agios,” “we,” “us,” and “our,” and similar expressions, except where the context requires otherwise, refer to Agios Pharmaceuticals, Inc.

Note Regarding Certain References in this Annual Report on Form 10-K

Throughout this Annual Report on Form 10-K, “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.

We are a biopharmaceutical company committed to transforming patients’ lives through leadership in the field of cellular metabolism, with the goal of creating differentiated medicines for rare diseases, with a focus on classical hematology.

With a history of focused study on cellular metabolism, we have a deep and mature understanding of this biology, which is involved in the healthy functioning of nearly every system in the body.

Building on this expertise, these learnings can be rapidly applied to our clinical trials with the goal of developing medicines that can have a significant impact for patients.

We accelerate the impact of our portfolio by cultivating connections with patient communities, healthcare professionals, partners and colleagues to discover, develop and deliver potential therapies for rare diseases.

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.

Fueled by connections, we build trusted partnerships with communities, collaborating to develop and deliver innovative medicines with 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, which was accepted with standard review by the FDA and granted a Prescription Drug User Fee Act, or PDUFA, goal date of September 7, 2025.

Also in December 2024, we 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 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 addition, we are currently evaluating PYRUKYND® in Phase 3 clinical trials for the treatment of sickle cell disease, or SCD, and in 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 hemolytic anemias;

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 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, and a regulatory application to 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 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 early 2026.

We will have a pre-supplemental New Drug Application, or sNDA, meeting with the FDA in the first quarter of 2026 and intend to submit a U.S.

marketing application for mitapivat in sickle cell disease, or SCD, following that engagement.

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;

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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.

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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.

Because the acquired assets do not meet the definition of a business in accordance with Accounting Standards Codification, or ASC, 805, Business Combinations , we accounted for the agreement as an asset acquisition.

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, which we refer to as AG-236, as a potential disease-modifying treatment for patients with PV.

In accordance with the license agreement, in the year ended December 31, 2023, we made an up-front payment to Alnylam and recognized in-process research and development of $ 17.5 million which was recorded in research and development expense within our consolidated statements of operations and classified as investing activities within our consolidated statements of cash flows.

We will also pay Alnylam for certain expenses associated with the development of AG-236, an siRNA targeting the TMPRSS6 gene, and these will be recorded in our consolidated statements of operations as incurred.

Additionally, we are responsible to pay up to $ 130.0 million in potential development and regulatory milestones, in addition to sales milestones as

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well as tiered royalties on annual net sales, if any, of licensed products, which may be subject to specified reductions and offsets.

We will also pay Alnylam for certain expenses associated with the development of AG-236, and these will be recorded in our consolidated statements of operations as incurred.

Additionally, 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 year ended December 31, 2025, we achieved a regulatory milestone that triggered a $ 10.0 million payment to Alnylam, which was recorded in research and development expense within our consolidated statements of operations and classified as investing activities within our consolidated statements of cash flows.

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 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.

On March 31, 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, 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, and 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 within the gain on sale of contingent payments line item in our consolidated statements of operations for the year ended December 31, 2023.

The Vorasidenib Milestone Payment and Vorasidenib Royalty Rights are referred to as contingent payments and recognized as income when realizable.

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.

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Royalty income related to the Retained Earn-Out Rights, if any, will be recognized in the period when realizable.

We recorded income from royalties of $ 9.9 million on U.S.

net sales of TIBSOVO® by Servier in the royalty income from gain on sale of oncology business line item within the consolidated statements of operation s for the year ended December 31, 2022.

As of December 31, 2025, we had cash, cash equivalents and marketable securities of $ 1.2 billion.

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Principles of consolidation

The consolidated financial statements include our accounts and the accounts of our wholly owned subsidiaries, Agios Securities Corporation, Agios International Sarl (GmbH), Agios Germany GmbH, Agios Netherlands B.V., Agios Italy S.R.L., Agios France SARL, and Agios Limited.

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France SARL, and Agios Limited.

All intercompany transactions have been eliminated in consolidation.

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generally accepted accounting principles, or U.S.

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Use of estimates

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Our trade accounts receivable arise from product sales and represent amounts due from specialty distributors and specialty pharmacy providers in the U.S.

and our distribution partners outside the U.S.

We monitor the financial performance and creditworthiness of our customers so that we can properly assess and respond to changes in their credit profile.

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Upon approval of our wholly owned product, PYRUKYND®, by the FDA on February 17, 2022 for the treatment of hemolytic anemia in adults with PK deficiency in the United States, we began to capitalize inventories of PYRUKYND®.

Additionally, upon approval of our wholly owned product, AQVESME™, by the FDA on December 23, 2025 for the treatment of anemia in adults with non-transfusion dependent and transfusion-dependent alpha- or beta-thalassemia in the United States, we began to capitalize inventories of AQVESME™.

Revenue recognition

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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.

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

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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.

Revenues from product sales are recorded at the net sales price, or transaction price, which includes estimates of variable consideration for which reserves are established and result from contractual adjustments, government rebates, returns and other allowances that are offered within the contracts with our Customers, healthcare providers, payors and other indirect customers relating to the sale of our products.

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Contractual Adjustments .

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These reserves are recorded in the same period the related revenue is recognized, resulting in a reduction of product revenue.

For Medicare, we also estimate the number of patients in the prescription drug coverage gap for whom we will owe an additional liability under the Medicare Part D program.

We estimate the amount of product sales that may be returned by Customers and record this estimate as a reduction of revenue in the period the related product revenue is recognized.

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Marketable securities

Marketable securities at December 31, 2024 and 2023 consisted of investments in U.S.

Treasuries, government securities, corporate debt securities and certificates of deposit.

Marketable securities at December 31, 2025 and 2024 consisted of investments in corporate debt securities, U.S.

Treasuries, government securities, and certificates of deposit.

We determine the appropriate classification of the securities at the time they are acquired and evaluate the appropriateness of such classifications at each balance sheet date.

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Marketable securities are recorded at fair value.

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

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

Realized gains and losses are included in interest income, net on a specific-identification basis.

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Investments classified as current are those that:

(i) have a maturity of less than one year , or (ii) have a maturity of one to two years but we intend to liquidate within the next twelve months.

(i) have a maturity of less than one year , or (ii) have a maturity of greater than one year but we intend to liquidate within the next twelve months.

Investments classified as non-current are those that:

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

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

We review marketable securities for impairment whenever the fair value of a marketable security is less than the amortized cost and evidence indicates that a marketable security’s carrying amount is not recoverable.

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

Unrealized losses are evaluated for

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

Credit-related impairment is recognized as an allowance on the balance sheet with a corresponding adjustment to earnings, and noncredit-related impairment is recognized in other comprehensive (loss) income, net of taxes.

Evidence considered in this assessment includes reasons for the impairment, compliance with our investment policy, the severity of the impairment, collectability of the security, and any adverse conditions specifically related to the security, an industry, or geographic area.

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Fair value measurements

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We evaluate transfers between levels at the end of each reporting period.

The carrying amounts of other receivables, prepaid expenses and other current assets, accounts payable, and accrued expenses approximate their fair values due to their short-term maturities.

The carrying amounts of prepaid expenses and other current assets, accounts payable, and accrued expenses approximate their fair values due to their short-term maturities.

Concentrations of credit risk

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Leasehold improvements are amortized over the lesser of the remaining lease term or the estimated useful life of the improvement.

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Impairment of long-lived assets

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Potential impairment is assessed when there is evidence that events or changes in circumstances indicate that the carrying amount of an asset may not be recovered.

Recoverability of these assets is assessed based on the undiscounted expected future cash flows from the assets, considering a number of factors, including past operating results, budgets and

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economic projections, market trends and product development cycles.

Recoverability of these assets is assessed based on the undiscounted expected future cash flows from the assets, considering a number of factors, including past operating results, budgets and economic projections, market trends and product development cycles.

If impairments are identified, assets are written down to their estimated fair value.

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Short-term leases, or leases that have a lease term of 12 months or less at commencement date, are excluded from this treatment and are recognized on a straight-line basis over the term of the lease.

We have not entered into any material short-term leases or financing leases as of December 31, 2024.

Research and development costs

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Income taxes are recorded in accordance with ASC 740, Accounting for Income Taxes , or ASC 740, which provides for deferred taxes using an asset and liability approach.

We recognize deferred tax assets and liabilities for the expected future tax consequences of events that have been included in our financial statements or tax returns.

We determine our deferred tax assets and liabilities based on differences between the financial reporting and tax bases of assets and liabilities, which are measured using the enacted tax rates and laws that will be in effect when the differences are expected to reverse.

Valuation allowances are

We recognize deferred tax assets and liabilities for the expected future tax

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provided, if based upon the weight of available evidence, it is more likely than not that some or all of the deferred tax assets will not be realized.

consequences of events that have been included in our financial statements or tax returns.

Valuation allowances are provided, if based upon the weight of available evidence, it is more likely than not that some or all of the deferred tax assets will not be realized.

We also account for uncertain tax positions in accordance with the provisions of ASC 740.

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The determination as to whether the tax benefit will more likely than not be realized is based upon the technical merits of the tax position as well as consideration of the available facts and circumstances.

Comprehensive income (loss)

Comprehensive income (loss) is defined as the change in equity of a business enterprise during a period from transactions, and other events and circumstances, and currently consists of net income (loss) and unrealized gains and losses on available-for-sale securities.

Accumulated other comprehensive loss consists entirely of unrealized gains and losses from available-for-sale securities as of December 31, 2024 and 2023.

Net income (loss) per share

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

Diluted net income (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 income (loss) per share calculation, stock options, restricted stock units, or RSUs, and performance-based stock units, or PSUs, for which the performance and market vesting conditions, respectively, have been deemed probable, and 2013 Employee Stock Purchase Plan, or 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 December 31, 2024 are not considered to be common stock equivalents.

Comprehensive (loss) income

Comprehensive (loss) income is defined as the change in equity of a business enterprise during a period from transactions, and other events and circumstances, and currently consists of net (loss) income and unrealized gains and losses on available-for-sale securities.

Accumulated other comprehensive income (loss) consists entirely of unrealized gains and losses from available-for-sale securities as of December 31, 2025 and 2024.

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, restricted stock units, or RSUs, and performance-based stock units, or PSUs, for which the performance and market vesting conditions, respectively, have been deemed probable, and the Amended and Restated 2013 Employee Stock Purchase Plan shares are considered to be common stock equivalents, while PSUs with performance and market vesting conditions, respectively, that were not deemed probable as of December 31, 2025 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 income (loss) from continuing operations.

The control number used is net (loss) income 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.

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Treasury stock purchases are accounted for under the cost method whereby the entire cost of the acquired stock is recorded as treasury stock.

Repurchased shares are held as treasury stock until they are retired or re-issued.

Treasury stock purchases are accounted for under the cost method whereby the entire cost of the acquired stock is recorded as treasury stock.

Repurchases of our common stock are accounted for as of the settlement date.

There were no repurchases, retirements or re-issuances of treasury stock during the year ended December 31, 2024.

Repurchases of our common stock are accounted for as of the settlement date, and are held as treasury stock until they are retired or re-issued.

There were no repurchases, retirements or re-issuances of treasury stock during the years ended December 31, 2025 and 2024.

Recent accounting pronouncements

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The standard is effective for annual periods beginning after December 15, 2024, and can be applied either prospectively or retrospectively.

We plan to adopt the standard in our 2025 annual period and are currently assessing its effect on our financial statement disclosures.

We adopted the standard during our 2025 annual period and have included required disclosures in Note 11 , Income Taxes .

In November 2024, the FASB issued 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.

Disaggregation of Income Statement Expenses , to improve disclosures around

Table of Content s

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 the standard on our financial statement disclosures.

an entity’s expenses.

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 plan to adopt the standard in the first quarter of 2026 and are currently assessing its effect on our consolidated financial statements.

Subsequent events

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Total cash equivalents and marketable securities $ 47,496 $ 1,078,655 $ — $ 1,126,151

There were no transfers between Level 1 and Level 2 and we had no financial assets or liabilities that were classified as Level 3 at any point during the year ended December 31, 2024.

We had no financial assets or liabilities that were classified as Level 3 at any point during the year ended December 31, 2025.

Marketable Securities

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(In thousands) Amortized

Certificates of deposit $ 10,374 $ 11 $ — $ 10,385

Treasuries 173,465 153 ( 27 ) 173,591

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

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

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

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

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Our building leases are comprised of office and laboratory space under non-cancelable operating leases.

These lease agreements have remaining lease terms of three years and contain various clauses for renewal at our option.

These lease agreements have remaining lease terms of two years and contain various clauses for renewal at our option.

The renewal options were not included in the calculation of the operating lease assets and the operating lease liabilities as the renewal options are not reasonably certain of being exercised.

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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, with the term of the lease running through April 2025.

At the end of the initial sublease period, the subtenant has the option to extend the lease for one additional 6 -month period.

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, with the term of the lease running through April 2025.

At the end of the initial sublease period, the subtenant has the option to extend the lease for one additional year, followed by a second extension option for twenty-two additional months.

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 began in November 2025 with the term of the lease running through February 2028.

We recorded operating sublease income of $ 1.9 million and $ 6.4 million for the years ended December 31, 2025 and December 31, 2024, respectively, in other income, net in the consolidated statements of operations.

We hold security deposits from our sublessees of approximately $ 0.9 million which is recorded within other non-current assets on our consolidated balance sheet.

As of December 31, 2024, the future minimum lease payments to be received under the long-term sublease agreements were as follows:

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

As of December 31, 2025, the future minimum lease payments to be received under the long-term sublease agreement was as follows:

(In thousands)

Total $ 1,310

Accrued Expenses

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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.

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Product revenue, net

United States $ 49,170 $ 36,395 $ 26,823

Rest of world 4,858 103 —

Total product revenue, net $ 54,028 $ 36,498 $ 26,823

One Customer accounted for 89 %, 95 % and 96 % of our consolidated revenues for the years ended December 31, 2025, 2024 and 2023, respectively, and 55 % and 92 % of accounts receivable from product sales for the years ended December 31, 2025 and 2024, respectively.

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There are no outstanding equity awards under the 2007 Plan.

In connection with the start of employment of our Chief Executive Officer and Chief Financial Officer in 2022, and our Chief Commercial Officer in 2023, 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).

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 December 31, 2025, the maximum number of shares reserved under the 2013 Plan, the 2023 Plan and the inducement grants described above was 12,332,999 , and we had 3,835,495 shares available for future issuance under the 2023 Plan.

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Vested ( 160,055 ) 26.40

Forfeited ( 23,000 ) 30.38

Unvested shares at December 31, 2025 376,978 $ 32.55

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As of December 31, 2025, there was no unrecognized compensation expense related to PSUs with performance-based vesting criteria that are considered probable of achievement that we expect to recognize.

There was $ 11.0 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 stock price performance.

The fair value of market-based stock units, or MSUs, are 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 estimated period to achievement of the market condition.

The following table presents MSU activity for the year ended December 31, 2024:

Stock Units Weighted-Average

Unvested shares at December 31, 2023 42,695 $ 41.50

Expired ( 42,695 ) 41.50

Unvested shares at December 31, 2024 — $ —

As of December 31, 2024, there was no remaining unrecognized compensation expense related to MSUs.

There was $ 12.3 million of total unrecognized compensation expense related to PSUs with performance-based vesting criteria that are considered not probable of achievement.

Amended and Restated 2013 Employee Stock Purchase Plan

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We account for forfeitures as they occur and, therefore, do not estimate forfeitures.

Net Income (Loss) per Share

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

For purposes of the dilutive net income (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 December 31, 2024 are not considered to be common stock equivalents.

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.

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 December 31, 2025 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 income (loss) from continuing operations.

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

Since we had a net loss for the years ended December 31, 2025 and 2023, 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 income (loss) per share to diluted weighted-average number of common shares used in computing net income (loss) per share for the periods indicated:

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:

Years ended December 31,

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Diluted shares 57,972,004 57,889,255 55,651,487

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

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:

Years ended December 31,

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Total 8,171,844 4,025,996 6,804,118

The domestic and foreign components of income (loss) before income taxes are as follows:

The domestic and foreign components of (loss) income before income taxes are as follows:

(In thousands) 2025 2024 2023

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Total $ ( 413,797 ) $ 717,969 $ ( 352,088 )

We did not have any provision for income taxes for the years ended December 31, 2023 and 2022.

A reconciliation of the expected income tax expense (benefit) computed using the federal statutory income tax rate to our effective income tax rate is as follows for the years ended December 31, 2024, 2023 and 2022:

2024 2023 2022

As further described in Note 2, Summary of Significant Accounting Policies , we have elected to prospectively adopt the guidance in ASU 2023-09.

The following table is a reconciliation of our effective income tax rate to the statutory federal income tax rate for the year ended December 31, 2025 in accordance with the guidance in ASU 2023-09:

(In thousands, except percentages) Year Ended December 31, 2025

federal statutory rate 21.0 % $ ( 86,897 )

State taxes, net of federal effect — % ( 153 )

Research and development costs 1.5 % ( 6,225 )

Orphan drug credit 2.3 % ( 9,654 )

Changes in valuation allowances ( 23.0 ) % 95,035

Nontaxable or nondeductible items

Stock compensation ( 1.7 ) % 7,149

Other items ( 0.1 ) % 592

Other adjustments

Other items 0.2 % ( 863 )

Effective income tax rate 0.2 % $ ( 1,016 )

The following table is a reconciliation of our effective income tax rate to the statutory federal income tax rate for the years ended December 31, 2024 and 2023 in accordance with the guidance prior to the adoption of ASU 2023-09:

Federal statutory tax rate 21.0 % 21.0 %

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Net deferred taxes $ — $ —

The Tax Cuts and Jobs Act, or TCJA, requires taxpayers to capitalize and amortize research and experimental expenditures under Internal Revenue Code section 174 for tax years beginning after December 31, 2021.

We capitalized research and experimental costs of $ 244.9 million and $ 232.7 million for the years ended December 31, 2024 and December 31, 2023, respectively.

We will amortize these costs for tax purposes over 5 years if the research and experimentation was performed in the U.S.

and over 15 years if the research and experimentation was performed outside the U.S.

As of December 31, 2024, we had net operating loss carryforwards, or NOLs, available to reduce state and foreign income taxes of approximately $ 320.0 million and $ 65.2 million, respectively.

On July 4, 2025, the One Big Beautiful Bill Act, or OBBBA, was enacted.

The legislation includes several changes to the U.S.

federal corporate income tax law, among other things, reinstating 100% bonus depreciation on qualified fixed assets, immediate expensing of domestic research and development expenditures, and favorable rules for determining the limitation on business interest expense.

These changes were retroactively enacted for tax years beginning after December 31, 2024 with certain provisions effective after January 19, 2025 and were reflected in the income tax provision for the year ended December 31, 2025.

The provisions of the OBBBA did not have a material impact on the effective income tax rate.

As of December 31, 2025, we had net operating loss carryforwards, or NOLs, available to reduce federal, state and foreign income taxes of approximately $ 515.8 million, $ 394.1 million and $ 38.0 million, respectively.

The federal NOLs will be carried forward indefinitely and could be used to offset up to 80% of taxable income in all other future years.

State NOLs will begin expiring in varying amounts through 2045 unless utilized.

At December 31, 2025, we also had available research and development tax credits for federal and state income tax purposes of approximately $ 32.1 million and $ 31.2 million, respectively.

If not utilized, the credits begin to expire in 2040 and 2028 for federal and state income tax purposes, respectively.

We engaged in clinical testing activities and incurred expenses that qualify for the federal orphan drug tax credit.

Additionally, we engaged in clinical testing activities and incurred expenses that qualify for the federal orphan drug tax credit.

At December 31, 2025, we had available orphan drug tax credits for federal purposes only of approximately $ 47.1 million.

If not utilized, the orphan drug credits begin to expire in 2040.

If not utilized, these orphan drug credits begin to expire in 2040.

As provided by Section 382 of the Internal Revenue Code of 1986, or Section 382, and similar state provisions, utilization of NOLs and tax credit carryforwards may be subject to substantial annual limitations due to ownership change limitations that have previously occurred or that could occur in the future.

Ownership changes may limit the amount of NOLs and tax credit carryforwards that can be utilized annually to offset future taxable income and tax, respectively.

In general, an ownership change, as defined by Section 382, results from transactions that increase the ownership of five percent stockholders in the stock of a corporation by more than 50 percent in the aggregate over a three year period.

In general, an ownership change, as defined by Section 382, results from transactions that increase the ownership of five percent stockholders in the

stock of a corporation by more than 50 percent in the aggregate over a three year period.

We completed a review of our changes in ownership through December 31, 2025 and determined that transactions have resulted in no ownership changes during the year ended December 31, 2025, as defined by Section 382.

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Based on the weight of available evidence, both positive and negative, we recorded a valuation allowance of $ 365.6 million and $ 275.6 million as of December 31, 2025 and December 31, 2024, respectively, because we have determined that it is more likely than not that these assets will not be fully realized.

The valuation allowance decreased by $ 104.1 million for the year ended December 31, 2024 and increased by $ 83.7 million for the year ended December 31, 2023.

The decrease for the year ended December 31, 2024 relates primarily to the utilization of tax attributes to offset taxable income, and the increase for the year ended December 31, 2023 primarily due to the Section 174 R&D expense capitalization.

The valuation allowance increased by $ 90.1 million for the year ended December 31, 2025 and decreased by $ 104.1 million for the year ended December 31, 2024.

The increase for the year ended December 31, 2025 is primarily due to Section 174 R&D domestic expense and the decrease for the year ended December 31, 2024 relates primarily to the utilization of tax attributes to offset taxable income.

The following table presents our change in valuation allowance for the years ended December 31, 2025 and, 2024:

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Gross increases - current period tax positions 2,871 3,013

Gross increases - prior period tax positions 1,327 —

Unrecognized tax benefits at the end of the year $ 35,789 $ 31,591

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There are currently no federal, state or foreign audits in progress.

As of December 31, 2024 we had an income tax payable of $ 0.9 million and as of December 31, 2023 we had an income tax receivable of $ 1.1 million, which is recorded within prepaid expenses and other current assets in our consolidated balance sheets.

As of December 31, 2025 we had an income tax receivable of $ 0.2 million, which is recorded within prepaid expenses and other current assets in our consolidated balance sheets, and as of December 31, 2024 we had an income tax payable of $ 0.9 million.

Property and Equipment, net

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Participants may contribute a percentage of their annual compensation to this plan, subject to statutory limitations.

We will make matching contributions equal to 100 % of the employee’s contributions, subject to a maximum of 4 % of eligible compensation.

We make matching contributions equal to 100 % of the employee’s contributions, subject to a maximum of 4 % of eligible compensation.

Segment Information

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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 the Company’s 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 the Company’s 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 consolidated balance sheets as total assets.

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

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

(In thousands) Years Ended December 31:

2025 2024 2023

Product revenue, net $ 36,498 $ 26,823 $ 11,740

PK activator (PYRUKYND®) direct expenses - research and development ( 112,720 ) ( 101,322 ) ( 83,271 )

Product revenue, net - U.S.

$ 49,170 $ 36,395 $ 26,823

Product revenue, net - Rest of world 4,858 103 —

PK activator (PYRUKYND®/AQVESME™) direct expenses - research and development ( 108,087 ) ( 112,720 ) ( 101,322 )

Compensation and related expenses - research and development ( 121,645 ) ( 114,618 ) ( 108,484 )

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Other segment items* ( 56,797 ) ( 67,787 ) ( 49,202 )

Net income (loss) $ 673,725 $ ( 352,088 ) $ ( 231,801 )

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

Net (loss) income $ ( 412,781 ) $ 673,725 $ ( 352,088 )

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

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-K

Item 9A. Controls and Procedures