Financial Analysis

14. Commitments and contingencies Under various agreements, the Company will be required to pay royalties and milestone payments upon the successful development and commercialization of products. Pursuant to the Agilis Merger Agreement with Agilis, Agilis equityholders were previously entitled to receive contingent consideration payments from the Company based on (i) the achievement of certain development milestones up to an aggregate maximum amount of $60.0 million, (ii) the achievement of certain regulatory approval milestones together with a milestone payment following the receipt of a priority review voucher up to an aggregate maximum amount of $535.0 million, (iii) the achievement of certain net sales milestones up to an aggregate maximum amount of $150.0 million, and (iv) a percentage of annual net sales for FA and Angelman syndrome during specified terms, ranging from 2%-6%. The Company was required to pay $40.0 million of the development milestone payments upon the passing of the second anniversary of the closing of the Agilis Merger, regardless of whether the applicable milestones have been achieved. As of December 31, 2025, all of the milestones have either been paid or settled, with the exception of the regulatory milestones and net sales milestones related to FA and Angelman syndrome and the net sales milestones related to Upstaza/Kebilidi. In May 2023, as part of the Companys strategic portfolio prioritization, the Company decided to discontinue its preclinical a …

CommitmentsAndContingenciesDisclosureTextBlock · excerpt; the full note is in the filing

Years ended December 31, 2025 2024 2023 United States International Total United States International Total United States International Total Translarna $ 235,299 235,299 $ 321,071 321,071 $ 332,753 332,753 Emflaza 146,361 146,361 207,215 207,215 255,087 255,087 Sephience 95,045 16,108 111,153 Upstaza/Kebilidi 10,779 45,847 56,626 16,913 16,913 18,841 18,841 All other products 37,264 37,264 36,946 36,946 31,496 31,496 Total net product revenue $ 252,185 $ 334,518 $ 586,703 $ 207,215 $ 374,930 $ 582,145 $ 255,087 $ 383,090 $ 638,177 Years ended December 31, 2025 2024 2023 United States $ 252,185 $ 207,215 $ 255,087 Russia 100,445 105,377 85,974 Brazil 65,661 72,081 58,606 All other countries 168,412 197,472 238,510 Total net product revenue $ 586,703 $ 582,145 $ 638,177 …

DisaggregationOfRevenueTableTextBlock · excerpt; the full note is in the filing

10. Stock award plan In May 2013, the Companys Board of Directors and stockholders approved the 2013 Long Term Incentive Plan, which became effective upon the closing of the Companys IPO. The 2013 Long Term Incentive Plan provides for the grant of incentive stock options, nonstatutory stock options, restricted stock awards and other stock-based awards. On June 8, 2022 (the Restatement Effective Date), the Companys stockholders approved the Amended and Restated 2013 Long-Term Incentive Plan (the Amended 2013 LTIP). The Amended 2013 LTIP provides for the grant of incentive stock options, nonstatutory stock options, restricted stock units and other stock-based awards. The number of shares of common stock reserved for issuance under the Amended 2013 LTIP is the sum of (A) the number of shares of the Companys common stock (up to 16,724,212 shares) that is equal to the sum of (1) the number of shares issued under the 2013 Long-Term Incentive Plan prior to the Restatement Effective Date, (2) the number of shares that remain available for issuance under the 2013 Long-Term Incentive Plan immediately prior to the Restatement Effective Date and (3) the number of shares subject to awards granted under the 2013 Long-Term Incentive Plan prior to the Restatement Effective Date that are outstanding as of the Restatement Effective Date, plus (B) from and after the Restatement Effective Date, an additional 8,475,000 shares of Common Stock. As of December 31, 2025, awards for 5,270,151 shares o …

DisclosureOfCompensationRelatedCostsShareBasedPaymentsTextBlock · excerpt; the full note is in the filing

3. Fair value of financial instruments and investments The Company follows the fair value measurement rules, which provide guidance on the use of fair value in accounting and disclosure for assets and liabilities when such accounting and disclosure is called for by other accounting literature. Cash equivalents, marketable securities, and equity investments are reflected in the accompanying financial statements at fair value. The carrying amount of receivables and accounts payable and accrued expenses approximate fair value due to the short-term nature of those instruments. The Company uses the market approach to measure fair value for its marketable securities. The market approach uses prices and other relevant information generated by market transactions involving identical or comparable assets. The Companys marketable securities are classified as Level 2 as they primarily utilize broker quotes in a nonactive market to value these securities. The Company owned common stock in ClearPoint Neuro, Inc. (ClearPoint) (formerly MRI Interventions, Inc.), a publicly traded medical device company. The ClearPoint equity investments (collectively, the ClearPoint Equity Investments) represented financial instruments, and therefore, were recorded at fair value, which was readily determinable. As of December 31, 2025, the Company sold all of its ClearPoint Equity Investments. The ClearPoint Equity Investments were components of prepaids and other current assets as of December 31, 2024 on t …

FairValueDisclosuresTextBlock · excerpt; the full note is in the filing

17. Intangible assets and goodwill Definite-lived intangibles Definite lived intangible assets consisted of the following at December 31, 2025 and 2024: Ending Balance at Foreign Ending Balance at Definite-lived December 31, currency December 31, intangible assets, gross 2024 Additions translation 2025 Waylivra 12,397 4,432 1,769 18,598 Tegsedi 18,249 4,132 2,444 24,825 Kebilidi 10,731 10,731 Upstaza 106,937 106,937 Sephience 283,500 283,500 Total definite-lived intangibles, gross $ 148,314 $ 292,064 $ 4,213 $ 444,591 Ending Balance at Foreign Ending Balance at Definite-lived December 31, currency December 31, intangible assets, accumulated amortization 2024 Amortization translation 2025 Waylivra (5,273) (2,666) (750) (8,689) Tegsedi (5,609) (3,860) (826) (10,295) Kebilidi (112) (894) (1,006) Upstaza (18,526) (8,911) (27,437) Sephience (8,411) (8,411) Total definite-lived intangibles, accumulated amortization $ (29,520) $ (24,742) $ (1,576) $ (55,838) Total definite-lived intangibles, net $ 388,753 Akcea is also entitled to receive royalty payments subject to certain terms set forth in the Tegsedi-Waylivra Agreement related to sales of Waylivra and Tegsedi. In accordance with the guidance for an asset acquisition, the Company records royalty payments when they become payable to Akcea and increase the cost basis for the Waylivra and Tegsedi intangible assets, respectively. For the year ended December 31, 2025, royalties of $4.1 million and $4.4 million for Tegsedi and Waylivra …

GoodwillAndIntangibleAssetsDisclosureTextBlock · excerpt; the full note is in the filing

13. Income taxes The income (loss) from operations before tax (expense) benefit consisted of the following for the years ended December 31, 2025, 2024, and 2023: 2025 2024 2023 Domestic $ 1,249,817 $ (598,807) $ (784,744) Foreign (553,198) 235,688 88,634 Total $ 696,619 $ (363,119) $ (696,110) The Income Tax Provision consisted of the following for the years ended December 31, 2025, 2024 and 2023: 2025 2024 2023 Current: U.S. Federal $ (2,728) $ (26,798) $ U.S. State and Local (4,397) (19,419) 27,226 Foreign (5,870) (8,896) (4,003) Deferred: U.S. Federal (6) 49,511 36,408 U.S. State and Local 6,397 10,521 Foreign (974) (971) (646) Total tax (expense) benefit $ (13,975) $ (176) $ 69,506 The difference between income tax expense and effective tax rate and the amounts resulting from applying the federal statutory rate of 21% to Income before income taxes in 2025 after the adoption of ASU 2023-09 is as follows: December 31, 2025 Amount Percent Federal income tax provision at statutory rate $ (146,290) 21.00 % State and local income tax, net of federal benefit 1 (4,397) 0.63 Foreign Tax Effects Ireland Foreign rate differential (12,858) 1.85 Nontaxable dividend income 57,466 (8.25) Change in valuation allowance (18,821) 2.70 IP & Royalty DTA Adjustment (11,073) 1.59 Other Foreign (17,688) 2.54 Effect of cross-border tax laws (4,056) 0.58 Tax Credits Research and development 42,627 (6.12) Foreign tax credits 1,516 (0.22) Changes in valuation allowance 139,978 (20.09) Nontaxable …

IncomeTaxDisclosureTextBlock · excerpt; the full note is in the filing

7. Debt Liability for sale of future royalties On July 17, 2020, the Company, RPI Intermediate Finance Trust (RPI), and, for the limited purposes set forth in the agreement, Royalty Pharma PLC, entered into a royalty purchase agreement (the Original Royalty Purchase Agreement). Pursuant to the Original Royalty Purchase Agreement, the Company sold to RPI 42.933% (the Original Assigned Royalty Rights) of the Companys right to receive sales-based royalty payments (the Royalty) on worldwide net sales of Evrysdi and any other product developed pursuant to the License and Collaboration Agreement (the License Agreement), dated as of November 23, 2011, by and among the Company, Roche and, for the limited purposes set forth therein, the SMA Foundation under the SMA program. In consideration for the sale of the Original Assigned Royalty Rights, RPI paid the Company $650.0 million in cash consideration. At the time, the Company retained a 57.067% interest in the Royalty and all economic rights to receive the remaining potential regulatory and sales milestone payments under the License Agreement, which remaining milestone payments equal $150.0 million in the aggregate as of December 31, 2025. The Original Royalty Purchase Agreement was set to terminate 60 days following the earlier of the date on which Roche is no longer obligated to make any payments of the Royalty pursuant to the License Agreement and the date on which RPI has received $1.3 billion in respect of the Original Assigned R …

LongTermDebtTextBlock · excerpt; the full note is in the filing

Recent accounting pronouncements 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. ASU 2024-03 enhances financial reporting by requiring additional information about specific expense categories in the notes to financial statements at interim and annual reporting periods. The guidance is effective for public business entities for annual periods beginning after December 15, 2026, and interim reporting periods beginning after December 15, 2027. Early adoption is permitted. The Company is currently planning to adopt this guidance when effective. The Company is assessing the impact of the adoption on the Companys consolidated financial statements and accompanying footnotes but expects the impact will be enhanced disclosures related to income statement expenses. In July 2025, the FASB issued ASU 2025-05, Financial Instruments-Credit Losses (Topic 326): Measurement of Credit Losses for Accounts Receivable and Contract Assets. ASU 2025-05 addresses challenges encountered when applying the guidance in Topic 326, Financial InstrumentsCredit Losses, to current accounts receivable and current contract assets arising from transactions accounted for under Topic 606, Revenue from Contracts with Customers. The guidance is effective for public business entities for annual periods beginning after December 15, 2025, and interim reporting periods within …

NewAccountingPronouncementsPolicyPolicyTextBlock · excerpt; the full note is in the filing

12. Revenue recognition Net product sales The Company views its operations and manages its business in one operating segment: life science. During the years ended December 31, 2025, 2024, and 2023, net product revenues consisted of the following: Years ended December 31, 2025 2024 2023 United States International Total United States International Total United States International Total Translarna $ 235,299 235,299 $ 321,071 321,071 $ 332,753 332,753 Emflaza 146,361 146,361 207,215 207,215 255,087 255,087 Sephience 95,045 16,108 111,153 Upstaza/Kebilidi 10,779 45,847 56,626 16,913 16,913 18,841 18,841 All other products 37,264 37,264 36,946 36,946 31,496 31,496 Total net product revenue $ 252,185 $ 334,518 $ 586,703 $ 207,215 $ 374,930 $ 582,145 $ 255,087 $ 383,090 $ 638,177 Disaggregated net product revenues by country for the years ended December 31, 2025, 2024, and 2023 are as follows: Years ended December 31, 2025 2024 2023 United States $ 252,185 $ 207,215 $ 255,087 Russia 100,445 105,377 85,974 Brazil 65,661 72,081 58,606 All other countries 168,412 197,472 238,510 Total net product revenue $ 586,703 $ 582,145 $ 638,177 Net product revenue for sales of Translarna in France are not depicted in the net product revenue tables above. Refer to Note 2 for further details. For the year ended December 31, 2025, four of the Companys distributors each accounted for over 10% of the Companys net product sales. For the years ended December 31, 2024 and 2023, two of the Companys distr …

RevenueFromContractWithCustomerTextBlock · excerpt; the full note is in the filing

15. Segment and geographic information The Company views its operations and manages its business in one operating segment: life science. The table below summarizes the significant expense categories for the life science segment regularly reviewed by the CODM for the years ended December 31, 2025, 2024, and 2023: Years Ended December 31, 2025 2024 2023 Total revenues $ 1,730,655 $ 806,780 $ 937,822 Less: Cost of product, collaboration and license sales 28,427 26,901 27,404 Program spend 201,706 211,230 258,705 Employee costs 283,583 251,184 334,305 Manufacturing costs 67,323 64,120 86,641 Administrative costs 73,805 72,420 70,531 Occupancy costs 29,561 34,913 42,085 Milestones 65,000 30,000 Other segment items (a) 363,606 444,307 714,755 Segment net income (loss) $ 682,644 $ (363,295) $ (626,604) Reconciliation of profit or loss Adjustments and reconciling items Consolidated net income (loss) $ 682,644 $ (363,295) $ (626,604) (a) Other segment items includes the following: Years Ended December 31, 2025 2024 2023 Interest income $ (74,078) $ (47,314) $ (13,325) Interest expense 226,308 214,307 142,505 Income tax expense (benefit) 13,975 176 (69,506) Depreciation 14,196 14,924 13,947 Amortization 24,742 60,738 222,635 All other (b) 158,463 201,476 418,499 Total other segment items $ 363,606 $ 444,307 $ 714,755 (b) All other includes cost of goods sold, royalty, travel and entertainment, distribution costs, bad debt expense, finance costs, contract labor costs, stock compensation …

SegmentReportingDisclosureTextBlock · excerpt; the full note is in the filing

8. Capital structure Common stock In August 2019, the Company entered into an At the Market Offering Sales Agreement (the Sales Agreement) with Cantor Fitzgerald and RBC Capital Markets, LLC (together, the Sales Agents), pursuant to which, the Company may offer and sell shares of its common stock, having an aggregate offering price of up to $125.0 million from time to time through the Sales Agents by any method that is deemed to be an at the market offering as defined in Rule 415(a)(4) promulgated under the Securities Act of 1933, as amended. No shares were sold pursuant to the Sales Agreement during the years ended December 31, 2025, 2024, and 2023. The remaining shares of the Companys common stock available to be issued and sold, under the Sales Agreement, have an aggregate offering price of up to $93.0 million as of December 31, 2025. In February 2023, the Company completed enrollment of its Phase 3 placebo-controlled clinical trial for sepiapterin for PKU. In connection with this event and pursuant to the Censa Merger Agreement, the Company paid a $30.0 million development milestone to the former Censa securityholders during the year ended December 31, 2023. The Company elected to pay this milestone in the form of shares of its common stock, less certain cash payments in accordance with the Censa Merger Agreement. Pursuant to such election, the Company issued 657,462 shares of its common stock and paid $0.4 million to the former Censa securityholders. As of December 31, 2 …

StockholdersEquityNoteDisclosureTextBlock · excerpt; the full note is in the filing

18. Subsequent events The Company has evaluated all subsequent events and transactions through the filing date. There were no material events that impacted the audited consolidated financial statements or disclosures.

SubsequentEventsTextBlock

10. Commitments and contingencies Under various agreements, the Company will be required to pay royalties and milestone payments upon the successful development and commercialization of products. Pursuant to the Agreement and Plan of Merger, dated as of July 19, 2018 by and among the Company, Agilis Biotherapeutics, Inc. (Agilis) and Agility Merger Sub, Inc. (such merger pursuant thereto, the Agilis Merger), Agilis equityholders were previously entitled to receive contingent consideration payments from the Company based on (i) the achievement of certain development milestones up to an aggregate maximum amount of $60.0 million, (ii) the achievement of certain regulatory approval milestones together with a milestone payment following the receipt of a priority review voucher up to an aggregate maximum amount of $535.0 million, (iii) the achievement of certain net sales milestones up to an aggregate maximum amount of $150.0 million, and (iv) a percentage of annual net sales for FA and Angelman syndrome during specified terms, ranging from 2%-6%. The Company was required to pay $40.0 million of the development milestone payments upon the passing of the second anniversary of the closing of the Agilis Merger, regardless of whether the applicable milestones have been achieved. As of June 30, 2026, all of the milestones have either been paid or settled, with the exception of the regulatory milestones and net sales milestones related to FA and Angelman syndrome and the net sales milest …

CommitmentsAndContingenciesDisclosureTextBlock · excerpt; the full note is in the filing

Three Months Ended June 30, 2026 2025 (in thousands) United States International Total United States International Total Sephience $ 127,550 $ 23,760 $ 151,310 $ $ $ Translarna 42,217 42,217 59,470 59,470 Emflaza 24,634 24,634 36,353 36,353 Upstaza/Kebilidi 11,163 11,163 11,889 11,889 All other products 9,495 9,495 10,617 10,617 Total net product revenue $ 152,184 $ 86,635 $ 238,819 $ 36,353 $ 81,976 $ 118,329 Six Months Ended June 30, 2026 2025 United States International Total United States International Total Sephience $ 239,590 $ 36,271 $ 275,861 $ $ $ Translarna 101,193 101,193 145,624 145,624 Emflaza 46,112 46,112 84,142 84,142 Upstaza/Kebilidi 2,998 17,759 20,757 20,547 20,547 All other products 20,469 20,469 21,442 21,442 Total net product revenue $ 288,700 $ 175,692 $ 464,392 $ 84,142 $ 187,613 $ 271,755 Three Months Ended June 30, Six Months Ended June 30, (in thousands) 2026 2025 2026 2025 United States $ 152,184 $ 36,353 $ 288,700 $ 84,142 Russia 25,581 18,124 28,830 56,638 Brazil 8,210 40,951 49,422 50,456 All other countries 52,844 22,901 97,440 80,519 Total net product revenue $ 238,819 $ 118,329 $ 464,392 $ 271,755 …

DisaggregationOfRevenueTableTextBlock · excerpt; the full note is in the filing

8. Stock award plan In May 2013, the Companys Board of Directors and stockholders approved the 2013 Long-Term Incentive Plan, which became effective upon the closing of the Companys initial public offering. On June 8, 2022 (the Restatement Effective Date), the Companys stockholders approved the Amended and Restated 2013 Long-Term Incentive Plan (the Amended 2013 LTIP). The Amended 2013 LTIP provides for the grant of incentive stock options, nonstatutory stock options, restricted stock units and other stock-based awards. The number of shares of common stock reserved for issuance under the Amended 2013 LTIP is the sum of (A) the number of shares of the Companys common stock (up to 16,724,212 shares) that is equal to the sum of (1) the number of shares issued under the 2013 Long-Term Incentive Plan prior to the Restatement Effective Date, (2) the number of shares that remain available for issuance under the 2013 Long-Term Incentive Plan immediately prior to the Restatement Effective Date and (3) the number of shares subject to awards granted under the 2013 Long-Term Incentive Plan prior to the Restatement Effective Date that are outstanding as of the Restatement Effective Date, plus (B) from and after the Restatement Effective Date, an additional 8,475,000 shares of Common Stock. As of June 30, 2026, awards for 3,408,235 shares of common stock are available for issuance under the Amended 2013 LTIP. In January 2020, the Companys Board of Directors approved the 2020 Inducement Sto …

DisclosureOfCompensationRelatedCostsShareBasedPaymentsTextBlock · excerpt; the full note is in the filing

4. Fair value of financial instruments and marketable securities The Company follows the fair value measurement rules, which provide guidance on the use of fair value in accounting and disclosure for assets and liabilities when such accounting and disclosure is called for by other accounting literature. These rules establish a fair value hierarchy for inputs to be used to measure fair value of financial assets and liabilities. This hierarchy prioritizes the inputs to valuation techniques used to measure fair value into three levels: Level 1 (highest priority), Level 2, and Level 3 (lowest priority). Level 1Unadjusted quoted prices in active markets for identical assets or liabilities that the Company has the ability to access at the balance sheet date. Level 2Inputs other than quoted prices included within Level 1 that are observable for the asset or liability, either directly or indirectly. Level 2 inputs include quoted prices for similar assets and liabilities in active markets, quoted prices for identical or similar assets or liabilities in markets that are not active, inputs other than quoted prices that are observable for the asset or liability (i.e., interest rates, yield curves, etc.), and inputs that are derived principally from or corroborated by observable market data by correlation or other means (market corroborated inputs). Level 3Inputs are unobservable and reflect the Companys assumptions as to what market participants would use in pricing the asset or liabilit …

FairValueDisclosuresTextBlock · excerpt; the full note is in the filing

12. Intangible assets and goodwill Definite-lived intangibles Definite-lived intangible assets consisted of the following at June 30, 2026 and December 31, 2025: Ending Balance at Foreign Ending Balance at Definite-lived December 31, currency June 30, intangible assets, gross 2025 Additions translation 2026 Waylivra 18,598 2,933 (578) 20,953 Tegsedi 24,825 3,186 (757) 27,254 Kebilidi 10,731 10,731 Upstaza 106,937 106,937 Sephience 283,500 32,190 315,690 Total definite-lived intangibles, gross $ 444,591 $ 38,309 $ (1,335) $ 481,565 Ending Balance at Foreign Ending Balance at Definite-lived December 31, currency June 30, intangible assets, accumulated amortization 2025 Amortization translation 2026 Waylivra (8,689) (1,853) 284 (10,258) Tegsedi (10,295) (2,455) 337 (12,413) Kebilidi (1,006) (972) (1,978) Upstaza (27,437) (7,950) (35,387) Sephience (8,411) (10,192) (18,603) Total definite-lived intangibles, accumulated amortization $ (55,838) $ (23,422) $ 621 $ (78,639) Total definite-lived intangibles, net $ 402,926 Akcea is entitled to receive royalty payments subject to certain terms set forth in the Tegsedi-Waylivra Agreement related to sales of Waylivra and Tegsedi. In accordance with the guidance for an asset acquisition, the Company records royalty payments when they become payable to Akcea and increase the cost basis for the Waylivra and Tegsedi intangible assets. For the six months ended June 30, 2026, royalties of $3.2 million and $2.9 million related to Tegsedi and Way …

GoodwillAndIntangibleAssetsDisclosureTextBlock · excerpt; the full note is in the filing

9. Debt Liability for sale of future royalties The Company has a royalty purchase agreement, (the A&R Royalty Purchase Agreement), with Royalty Pharma. Under the A&R Royalty Purchase Agreement, Royalty Pharma has provided funding to the Company totaling $2.1 billion. In exchange for these fundings, the Company sold Royalty Pharma 100% of the Company's rights to receive sales-based royalty payments on worldwide net sales of Roches Evrysdi (risdiplam) product and any other product developed pursuant to the SMA collaboration with the Company, Roche, and the SMA Foundation. Pursuant to A&R Royalty Purchase Agreement, the Company is entitled to three potential additional purchase price payments of $20.0 million each conditioned upon receipt by Royalty Pharma of more than $347.0 million of Assigned Royalty Payments (as defined in the A&R Royalty Purchase Agreement) in respect of Calendar Year Net Sales (as defined in the A&R Royalty Purchase Agreement) arising in 2027, $363.0 million of Assigned Royalty Payments in respect of Calendar Year Net Sales arising in 2028, and $379.0 million of Assigned Royalty Payments in respect of Calendar Year Net Sales arising in 2029, respectively. Pursuant to the guidance in ASC 470-10-25-2, the Company determined that these fundings should be classified as debt and are recorded as liability for sale of future royalties-current and liability for sale of future royalties-noncurrent on the Companys consolidated balance sheet based …

LongTermDebtTextBlock · excerpt; the full note is in the filing

Recently issued accounting standards 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. ASU 2024-03 enhances financial reporting by requiring additional information about specific expense categories in the notes to financial statements at interim and annual reporting periods. The guidance is effective for public business entities for annual periods beginning after December 15, 2026, and interim reporting periods beginning after December 15, 2027. Early adoption is permitted. The Company is currently planning to adopt this guidance when effective. The Company is assessing the impact of the adoption on the Companys consolidated financial statements and accompanying footnotes but expects the impact will be enhanced disclosures related to income statement expenses. In September 2025, the FASB issued ASU 2025-06, IntangiblesGoodwill and Other Internal-Use Software (Subtopic 350-40): Targeted Improvements to the Accounting for Internal-Use Software. ASU 2025-06 enhances accounting for software costs that are accounted for under Subtopic 350-40, IntangiblesGoodwill and OtherInternal-Use Software (referred to as internal-use software). The guidance is effective for public business entities for annual periods beginning after December 15, 2027, and interim reporting periods within those annual reporting periods. Entities have three transition o …

NewAccountingPronouncementsPolicyPolicyTextBlock · excerpt; the full note is in the filing

11. Revenue recognition The Company views its operations and manages its business in one operating segment: life science. The life science segment is focused on the discovery, development and commercialization of the Companys clinically differentiated medicines that provide benefits to patients with rare disorders. The Company derives its revenues through its worldwide net product sales, collaboration and license agreements, and royalty revenues. Net product sales During the three and six months ended June 30, 2026 and 2025, net product revenues consisted of the following: Three Months Ended June 30, 2026 2025 (in thousands) United States International Total United States International Total Sephience $ 127,550 $ 23,760 $ 151,310 $ $ $ Translarna 42,217 42,217 59,470 59,470 Emflaza 24,634 24,634 36,353 36,353 Upstaza/Kebilidi 11,163 11,163 11,889 11,889 All other products 9,495 9,495 10,617 10,617 Total net product revenue $ 152,184 $ 86,635 $ 238,819 $ 36,353 $ 81,976 $ 118,329 Six Months Ended June 30, 2026 2025 United States International Total United States International Total Sephience $ 239,590 $ 36,271 $ 275,861 $ $ $ Translarna 101,193 101,193 145,624 145,624 Emflaza 46,112 46,112 84,142 84,142 Upstaza/Kebilidi 2,998 17,759 20,757 20,547 20,547 All other products 20,469 20,469 21,442 21,442 Total net product revenue $ 288,700 $ 175,692 $ 464,392 $ 84,142 $ 187,613 $ 271,755 Disaggregated net product revenues by country for the three and six months ended June 30, 2026 …

RevenueFromContractWithCustomerTextBlock · excerpt; the full note is in the filing

13. Segment information The Company views its operations and manages its business in one operating segment: life science. The Company expects to continue to incur significant expenses as it advances product candidates through all stages of development and clinical trials and, ultimately, seek regulatory approval. The table below summarizes the significant expense categories for the life science segment regularly reviewed by the chief operating decision maker for the three and six months ended June 30, 2026 and 2025: Three months ended June 30, Six months ended June 30, 2026 2025 2026 2025 Total revenues $ 360,519 $ 178,875 $ 633,070 $ 1,354,971 Less: Cost of product, collaboration and license sales 10,933 8,093 30,611 15,800 Program spend 42,580 49,705 80,125 97,863 Employee costs 68,348 65,593 144,469 133,292 Manufacturing costs 6,232 19,519 15,317 34,470 Administrative costs 16,547 17,896 33,236 36,310 Occupancy costs 7,493 6,643 15,848 13,825 Other segment items (a) 124,890 76,275 232,777 221,698 Segment net income (loss) $ 83,496 $ (64,849) $ 80,687 $ 801,713 Reconciliation of profit or loss Adjustments and reconciling items Consolidated net income (loss) $ 83,496 $ (64,849) $ 80,687 $ 801,713 (a) Other segment items includes the following: Three months ended June 30, Six months ended June 30, 2026 2025 2026 2025 Interest income $ (18,119) $ (20,991) $ (36,104) $ (38,236) Interest expense 66,600 51,349 133,615 102,686 Income tax expense (benefit) 14,049 (6,203) 14,396 57, …

SegmentReportingDisclosureTextBlock · excerpt; the full note is in the filing

6. Capitalization In August 2019, the Company entered into an At the Market Offering Sales Agreement (the Sales Agreement) with Cantor Fitzgerald and RBC Capital Markets, LLC (together, the Sales Agents), pursuant to which, the Company may offer and sell shares of its common stock, having an aggregate offering price of up to $125.0 million from time to time through the Sales Agents by any method that is deemed to be an at the market offering as defined in Rule 415(a)(4) promulgated under the Securities Act of 1933, as amended. No shares were sold during the three and six months ended June 30, 2026 and 2025. The remaining shares of the Companys common stock available to be issued and sold, under the At the Market Offering, have an aggregate offering price of up to $93.0 million as of June 30, 2026. …

StockholdersEquityNoteDisclosureTextBlock · excerpt; the full note is in the filing

14. Subsequent events The Company has evaluated subsequent events and transactions through the filing date. There were no material events that impacted the consolidated financial statements or disclosures.

SubsequentEventsTextBlock