What changed in this filing

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Item 1. Financial Statements

2026-07-30 compared with 2026-04-29 · 132 added, 98 removed, 373 unchanged (38% of the section changed)

9 unchanged sentences

Inventory 30,152 32,920

Contract assets 4,104 —

Prepaid expenses and other current assets 45,724 44,130

17 unchanged sentences

25,000,000 shares authorized;

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

no shares issued or outstanding at June 30, 2026 and December 31, 2025

Common stock, $ 0.001 par value;

125,000,000 shares authorized;

75,557,749 shares issued and 59,341,338 outstanding at March 31, 2026, and 74,665,003 shares issued and 58,448,592 outstanding at December 31, 2025

75,821,241 shares issued and 59,604,830 outstanding at June 30, 2026, and 74,665,003 shares issued and 58,448,592 outstanding at December 31, 2025

Additional paid-in capital 2,591,745 2,555,037

1 unchanged sentence

Accumulated deficit ( 761,519 ) ( 561,705 )

Treasury stock, at cost ( 16,216,411 shares at March 31, 2026 and December 31, 2025)

Treasury stock, at cost ( 16,216,411 shares at June 30, 2026 and December 31, 2025)

( 802,486 ) ( 802,486 )

4 unchanged sentences

Condensed Consolidated Statements of Operations

Three Months Ended March 31,

Three Months Ended June 30, Six Months Ended June 30,

(In thousands, except share and per share data)

2026 2025 2026 2025

Product revenue, net $ 44,745 $ 12,455 $ 65,491 $ 21,181

14 unchanged sentences

Condensed Consolidated Statements of Comprehensive Loss

Three Months Ended March 31,

Three Months Ended June 30, Six Months Ended June 30,

(In thousands)

2026 2025 2026 2025

Net loss $ ( 100,703 ) $ ( 112,020 ) $ ( 199,814 ) $ ( 201,309 )

18 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 loss on available-for-sale securities — — — ( 1,481 ) — — — ( 1,481 )

Common stock issued under stock incentive plan and ESPP 263,492 — 3,149 — — — — 3,149

Stock-based compensation expense — — 15,941 — — — — 15,941

Net loss — — — — ( 100,703 ) — — ( 100,703 )

Balance at June 30, 2026 75,821,241 $ 76 $ 2,591,745 $ ( 1,795 ) $ ( 761,519 ) ( 16,216,411 ) $ ( 802,486 ) $ 1,026,021

Common Stock Additional

11 unchanged sentences

Balance at March 31, 2025 74,103,192 $ 74 $ 2,506,788 $ 365 $ ( 238,213 ) ( 16,216,411 ) $ ( 802,486 ) $ 1,466,528

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

See accompanying Notes to Condensed Consolidated Financial Statements.

1 unchanged sentence

Condensed Consolidated Statements of Cash Flows

Three Months Ended

Six Months Ended

(In thousands) 2026 2025

5 unchanged sentences

Net accretion of discount on marketable securities ( 2,271 ) ( 3,768 )

Loss on disposal of property and equipment — 5

Non-cash operating lease expense 6,579 6,094

Expense associated with license agreement 25,000 10,000

Realized gain on investments ( 95 ) ( 2 )

2 unchanged sentences

Inventory 2,768 ( 3,232 )

Contract assets ( 4,104 ) —

Prepaid expenses and other current and non-current assets ( 7,344 ) ( 3,746 )

3 unchanged sentences

Operating lease liabilities ( 9,036 ) ( 8,228 )

Other non-current liabilities 150 ( 779 )

Net cash used in operating activities ( 176,232 ) ( 188,613 )

2 unchanged sentences

Proceeds from maturities and sales of marketable securities 508,256 508,894

Payments associated with license agreement ( 25,000 ) ( 10,000 )

Purchases of property and equipment ( 1,981 ) ( 1,641 )

17 unchanged sentences

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;

In addition, unless otherwise stated or the context indicates otherwise, all references in this Quarterly Report on Form 10-Q to “AQVESME™ (mitapivat)” or “AQVESME™” refer to our U.S.

Food and Drug Administration, or 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.

4 unchanged sentences

The lead product candidate in our portfolio, mitapivat, is an activator of both wild-type and mutant pyruvate kinase, or PK, enzymes for the potential treatment of hemolytic anemias.

Mitapivat is approved in the United States by the U.S.

Food and Drug Administration, or FDA, under the brand name AQVESME™ for the treatment of anemia in adults with non-transfusion dependent and transfusion-dependent alpha- or beta-thalassemia and in Saudi Arabia and the United Arab Emirates under the brand name PYRUKYND® for the treatment of adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia.

Mitapivat is also approved under the brand name PYRUKYND® in the United States by the FDA for the treatment of hemolytic anemia in adults with PK deficiency and in the European Union, or EU, and Great Britain for the treatment of PK deficiency in adults.

In December 2024, we announced that we submitted a marketing authorization application, or MAA, to the European Medicines Agency, or EMA for PYRUKYND® for the treatment of adult patients with non-transfusion dependent and transfusion-dependent alpha- or beta-thalassemia.

In October 2025, we announced that the Committee for Medicinal Products for Human Use, or the CHMP, of the EMA adopted a positive opinion for the new indication for PYRUKYND® in adults for the treatment of anemia associated with transfusion-dependent and non-transfusion-dependent alpha- or beta-thalassemia.

The European Commission is reviewing the CHMP’s opinion, with the final decision expected in the first half of 2026.

In the first quarter of 2026, we presented data from the phase 2 and phase 3 portions of the RISE UP study of PYRUKYND® in adults with sickle cell disease, or SCD, in a pre-supplemental New Drug Application meeting with the FDA.

Based on the FDA’s recommendation, we submitted our proposal to the FDA for a confirmatory clinical trial to support U.S.

accelerated approval of mitapivat in SCD.

The proposal incorporates a primary endpoint that is different from the endpoints in the RISE UP studies and is informed by both analyses of RISE UP data and discussions with the FDA.

We intend to submit a supplemental New Drug Application, or sNDA, for mitapivat for SCD in the second quarter of 2026.

In addition, we are evaluating mitapivat for the treatment of pediatric patients with PK deficiency.

We are also developing (i) tebapivat, a novel PK activator, for the potential treatment of lower-risk myelodysplastic syndromes, or LR MDS, and SCD;

(ii) AG-181, our phenylalanine hydroxylase, or PAH, stabilizer for the potential treatment of phenylketonuria, or PKU;

and (iii) AG-236, an siRNA in-licensed from Alnylam Pharmaceuticals, Inc., or Alnylam, targeting the transmembrane serine protease 6, or TMPRSS6, gene for the potential treatment of polycythemia vera, or PV.

Mitapivat is approved under the brand name AQVESME™ in the United States for the treatment of anemia in adults with non-transfusion dependent and transfusion-dependent alpha- or beta-thalassemia.

Mitapivat is approved under the brand name PYRUKYND®:

in the United States for the treatment of hemolytic anemia in adults with PK deficiency;

in the European Union, or EU, for the treatment of PK deficiency and for the treatment of non-transfusion dependent and transfusion dependent alpha- or beta-thalassemia in adult patients;

in the Kingdom of Saudi Arabia for the treatment of adults with non-transfusion dependent and transfusion-dependent alpha- or beta-thalassemia;

in the United Arab Emirates for the treatment of non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia in adult patients;

and in Great Britain for the treatment of PK deficiency in adult patients.

We submitted a supplemental New Drug Application, or sNDA, for the U.S.

accelerated approval of mitapivat in sickle cell disease, or SCD, in May 2026, which was accepted with priority review by the FDA and granted a Prescription Drug User Fee Act, or PDUFA, goal date of November 1, 2026.

To support this accelerated approval pathway, we initiated REIGNITE, a phase 3 confirmatory trial designed to demonstrate the clinical benefit of mitapivat on reducing transfusion burden in patients with SCD aged 12 years or older.

Additionally, in the second quarter of 2026 we filed for regulatory approval for SCD in the Kingdom of Saudi Arabia.

We are also developing (i) AG-181, our phenylalanine hydroxylase, or PAH, stabilizer for the potential treatment of phenylketonuria, or PKU;

(ii) AG-236, an siRNA in-licensed from Alnylam Pharmaceuticals, Inc., or Alnylam, targeting the transmembrane serine protease 6, or TMPRSS6, gene for the potential treatment of polycythemia vera, or PV;

and (iii) cevidoplenib, an oral spleen tyrosine kinase, or SYK, inhibitor in-licensed from Oscotec Inc., or Oscotec, for the potential treatment of immune thrombocytopenia, or ITP, and other autoimmune indications.

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.

Oscotec License Agreement

On June 1, 2026, we entered into a license agreement with Oscotec, a South Korean biopharmaceutical company, under which Agios acquired the worldwide rights (subject to Oscotec’s right to opt-in to develop and commercialize cevidoplenib in South Korea) to develop, manufacture, and commercialize cevidoplenib as a potential treatment for ITP and other autoimmune

Because the acquired assets do not meet the definition of a business in accordance with ASC 805, Business Combinations , Agios will account for the agreement as an asset acquisition.

In accordance with the agreement, in the three months ended June 30, 2026, Agios made an up-front payment to Oscotec and recognized in-process research and development of $ 25.0 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.

In addition, Agios is responsible to pay up to $ 140.0 million in potential development and regulatory milestones in the U.S.

and Europe, in addition to sales milestones as well as tiered royalties on future net sales, if any, of licensed products, which may be subject to specified reductions and offsets.

Sale of Oncology Business to Servier and Sale of Contingent Payments

11 unchanged sentences

Basis of Presentation

The condensed consolidated balance sheet as of March 31, 2026, the condensed consolidated statements of operations, comprehensive loss and stockholders' equity for the three months ended March 31, 2026 and 2025, and the condensed consolidated statements of cash flows for the three months ended March 31, 2026 and 2025 are unaudited.

The unaudited condensed consolidated financial statements have been prepared on the same basis as the annual financial statements and, in the opinion of our management, reflect all adjustments, which include only normal recurring adjustments, necessary to fairly state our financial position as of March 31, 2026, our results of operations and stockholders' equity for the three months ended March 31, 2026 and 2025, and cash flows for the three months ended March 31, 2026 and 2025.

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

The results of operations for the three months ended March 31, 2026 are not necessarily indicative of the results to be expected for the year ending December 31, 2026 or for any other future annual or interim period.

The condensed consolidated balance sheet as of June 30, 2026, the condensed consolidated statements of operations, comprehensive loss and stockholders' equity for the three and six months ended June 30, 2026 and 2025, and the condensed consolidated statements of cash flows for the six months ended June 30, 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 June 30, 2026, our results of operations and stockholders' equity for the three and six months ended June 30, 2026 and 2025, and cash flows for the six months ended June 30, 2026 and 2025.

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

The results of operations for the three and six months ended June 30, 2026 are not necessarily indicative of the results to be expected for the year ending December 31, 2026 or for any other future annual or interim period.

The condensed consolidated balance sheet data as of December 31, 2025 was derived from our audited financial statements, but does not include all disclosures required by U.S.

generally accepted accounting principles, or U.S.

The condensed consolidated interim financial statements should be read in conjunction with the audited consolidated financial statements and notes thereto included in our Annual Report on Form 10-K for the year ended December 31, 2025 that was filed with the Securities and Exchange Commission, or SEC, on February 12, 2026.

Our condensed consolidated financial statements include our accounts and the accounts of our wholly owned subsidiaries.

5 unchanged sentences

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 March 31, 2026, we had cash, cash equivalents and marketable securities of $ 1.0 billion.

As of June 30, 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.

3 unchanged sentences

Recent Accounting Pronouncements

In November 2024, the Financial Accounting Standards Board, or FASB, issued Accounting Standards Update, or ASU, 2024-03, Income Statement – Reporting Comprehensive Income – Expense Disaggregation Disclosures (Subtopic 220-40):

In November 2024, the Financial Accounting Standards Board 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.

2 unchanged sentences

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

5 unchanged sentences

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 March 31, 2026:

The following table summarizes our cash equivalents and marketable securities measured at fair value and by level on a recurring basis as of June 30, 2026:

(In thousands) Level 1 Level 2 Level 3 Total

2 unchanged sentences

Marketable securities:

Certificates of deposit — 751 — 751

Treasuries — 251,421 — 251,421

5 unchanged sentences

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 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.

After completing our validation procedures, we did not adjust or override any fair value measurements provided by the pricing services as of June 30, 2026.

There have been no changes to the valuation methods during the six months ended June 30, 2026, and we had no financial assets or liabilities that were classified as Level 3 at any point during the six months ended June 30, 2026.

Marketable Securities

4 unchanged sentences

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 months ended March 31, 2026 or 2025.

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

There were no material realized gains or losses on marketable securities for the three and six months ended June 30, 2026 or 2025.

Marketable securities at June 30, 2026 consisted of the following:

(In thousands) Amortized

1 unchanged sentence

Gains Unrealized

Certificates of deposit $ 750 $ 1 $ — $ 751

Treasuries $ 96,509 $ 102 $ ( 8 ) $ 96,603

21 unchanged sentences

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

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

As of June 30, 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 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.

(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 June 30, 2026 and December 31, 2025, we held 134 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 June 30, 2026 and December 31, 2025 related to these securities.

The aggregate fair value of debt securities in an unrealized loss position at June 30, 2026 and

December 31, 2025 was $ 572.7 million and $ 136.2 million, respectively.

There were no individual securities that were in a significant unrealized loss position as of June 30, 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 March 31, 2026 and December 31, 2025.

We do not consider these marketable securities to be impaired as of June 30, 2026 and December 31, 2025.

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

(In thousands) March 31,

(In thousands) June 30,

2026 December 31,

3 unchanged sentences

Total inventory $ 35,752 $ 32,920

Balance Sheet Classification:

Inventory $ 30,152 $ 32,920

Other non-current assets 5,600 —

Total inventory $ 35,752 $ 32,920

Long-term inventory is included in other non-current assets within our condensed consolidated balance sheets.

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

3 unchanged sentences

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

Three Months Ended March 31,

Three Months Ended June 30, Six Months Ended June 30,

(In thousands) 2026 2025 2026 2025

1 unchanged sentence

Cash paid for amounts included in the measurement of operating lease liabilities 5,058 4,911 10,070 9,748

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:

We have not entered into any material short-term leases or financing leases as of June 30, 2026.

In arriving at the operating lease liabilities as of June 30, 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 1.7 and 2.2 years, respectively.

As of June 30, 2026, undiscounted minimum rental commitments under non-cancelable leases were as follows:

(In thousands)

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

In June 2026, we entered into a long-term sublease agreement for 12,995 square feet of the office space at 38 Sidney Street, Cambridge, Massachusetts, which begins in July 2026 with the term of the lease running through February 2028.

We recorded operating sublease income of $ 0.1 million and $ 0.5 million for the three months ended June 30, 2026 and 2025, respectively, and $ 0.2 million and $ 1.8 million for the six months ended June 30, 2026 and 2025, respectively, in other income, net in the condensed consolidated statements of operations.

We hold security deposits from our sublessees of approximately $ 0.2 million which is recorded within other non-current assets on our condensed consolidated balance sheet.

As of June 30, 2026, the future minimum lease payments to be received under the long-term sublease agreements were as follows:

(In thousands)

Remaining 2026 $ 465

Total $ 1,697

Accrued Expenses

Accrued expenses consisted of the following:

(In thousands) March 31,

(In thousands) June 30,

2026 December 31,

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Product revenue, net, was as follows:

Three Months Ended March 31,

Three Months Ended June 30, Six Months Ended June 30,

(In thousands) 2026 2025 2026 2025

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For Medicare, we also estimate the number of patients for whom we will owe an additional liability under the Medicare Part D Manufacturer Discount Program.

Returns / Replacement

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

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 three months ended March 31, 2026:

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

Returns / Replacement / Other Allowances

We estimate the amount of product sales that may be returned by Customers or replaced by Agios, or other allowances for revenue not expected to be sold by Customers, and record these estimates as a reduction of revenue in the period the related product revenue is recognized.

We currently estimate product return and replacement liabilities, and the revenue reserve related to other allowances 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 six months ended June 30, 2026:

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

Balance at December 31, 2025 $ 182 $ 1,274 $ 1,040 $ 2,496

Current provisions relating to sales in the current year 1,817 3,738 4,519 10,074

Adjustments relating to prior years — ( 113 ) ( 370 ) ( 483 )

Payments/returns relating to sales in the current year ( 1,459 ) ( 1,016 ) — ( 2,475 )

Payments/returns relating to sales in the prior years ( 174 ) ( 895 ) ( 16 ) ( 1,085 )

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

Balance at June 30, 2026 $ 366 $ 2,988 $ 5,173 $ 8,527

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

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

(In thousands) June 30, 2026 December 31, 2025

Reduction of accounts receivable $ 369 $ 174

Reduction of contract assets 3,865 —

Component of accrued expenses 4,293 2,322

Total revenue-related reserves $ 8,527 $ 2,496

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)

The following table presents changes in our contract assets during the six months ended June 30, 2026:

(In thousands) December 31, 2025 Additions Deductions June 30, 2026

Accounts receivable, net (1)

(1) Additions to contract assets relate to amounts billed to Customers for product sales and deductions to contract assets primarily relate to collection of receivables during the reporting period.

$ 10,577 $ 67,126 $ ( 57,591 ) $ 20,112

Contract assets (2)

— 4,104 — 4,104

Total contract assets $ 10,577 $ 71,230 $ ( 57,591 ) $ 24,216

(1) Additions to accounts receivable, net relate to amounts billed to Customers for product sales and deductions from accounts receivable, net primarily relate to collection of receivables during the reporting period.

(2) Additions to contract assets relate to amounts unbilled to Customers for net product sales, and deductions from contract assets relate to amounts billed to Customers for product sales.

Share-Based Payments

1 unchanged sentence

In June 2023, our stockholders approved the 2023 Stock Incentive Plan, or the 2023 Plan.

The 2023 Plan provides for the grant of incentive stock options, nonstatutory stock options, stock appreciation rights, restricted stock awards, restricted stock units, or RSUs, performance-based share units, or PSUs, and other stock-based awards to employees, advisors, consultants and non-employee directors.

The 2023 Plan provides for the grant of incentive stock options, nonstatutory stock options, stock appreciation rights, restricted stock awards, restricted stock units, or RSUs, performance-based stock units, or PSUs, and other stock-based awards to employees, advisors, consultants and non-employee directors.

Following the adoption of the 2023 Plan, we ceased granting equity awards under the 2013 Stock Incentive Plan, or the 2013 Plan.

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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 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.

As of June 30, 2026, the maximum number of shares reserved under the 2013 Plan, the 2023 Plan and the inducement grants described above was 13,242,570 , and we had 4,014,743 shares available for future issuance under the 2023 Plan.

Stock options

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

The following table presents stock option activity for the six months ended June 30, 2026:

Stock Options Weighted-Average

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Expired ( 128,627 ) 42.25

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.

Outstanding at June 30, 2026 6,552,073 $ 38.33

Exercisable at June 30, 2026 4,629,494 $ 41.19

Vested and expected to vest at June 30, 2026 6,552,073 $ 38.33

At June 30, 2026, there was approximately $ 32.2 million of total unrecognized compensation expense related to unvested stock option awards, which we expect to recognize over a weighted-average period of approximately 2.60 years.

Restricted stock units

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

The following table presents RSU activity for the six months ended June 30, 2026:

Stock Units Weighted-Average

4 unchanged sentences

Forfeited ( 207,202 ) 32.19

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.

Unvested shares at June 30, 2026 2,186,904 $ 31.22

As of June 30, 2026, there was approximately $ 54.1 million of total unrecognized compensation expense related to RSUs, which we expect to recognize over a weighted-average period of approximately 2.04 years.

Performance-based stock units

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

The following table presents PSU activity for the six months ended June 30, 2026:

Stock Units Weighted-Average

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Granted 83,500 29.20

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.

Vested ( 103,128 ) 31.69

Unvested shares at June 30, 2026 357,350 $ 32.02

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

For PSUs that also have a service period requirement, stock-based compensation expense is recognized over the service period once the underlying performance condition is considered probable of achievement using our management's best estimates.

Included in unvested shares were 61,812 shares with performance conditions that were considered probable of achievement at June 30, 2026, but which also have a service period requirement until December 31, 2027.

As of June 30, 2026, there was approximately $ 1.1 million of total unrecognized compensation expense related to these PSUs.

As of June 30, 2026, there was $ 9.4 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

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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:

The following table presents MSU activity for the six months ended June 30, 2026:

Stock Units Weighted-Average

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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.

Unvested shares at June 30, 2026 131,500 $ 49.34

As of June 30, 2026, there was $ 5.7 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 2.50 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.

In June 2013, our Board of Directors adopted, and in July 2013 our stockholders approved, the 2013 Employee Stock Purchase Plan, or 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 six months ended June 30, 2026 and 2025, respectively, under the 2013

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

As of June 30, 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

June 30, Six Months Ended

(In thousands) 2026 2025 2026 2025

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

Three Months Ended March 31,

Three Months Ended June 30, Six Months Ended June 30,

(In thousands) 2026 2025 2026 2025

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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.

For purposes of the dilutive net loss per share calculation, stock options, RSUs, PSUs and MSUs 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 and MSUs with performance and market vesting conditions, respectively, that were not deemed probable as of June 30, 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.

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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 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.

Since we had a net loss for all periods 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,

Three and Six Months Ended June 30,

Stock options 6,552,073 6,424,575

1 unchanged sentence

Performance-based stock units 61,812 —

Employee stock purchase plan shares 35,327 45,571

Total common stock equivalents 8,836,116 8,566,269

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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.

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

Three Months Ended March 31,

Three Months Ended June 30, Six Months Ended June 30,

(In thousands) 2026 2025 2026 2025

9 unchanged sentences

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