PTC Therapeutics(PTCT) - 2025 Q2 - Quarterly Report

PART I—FINANCIAL INFORMATION Item 1. Financial Statements (unaudited) This section presents PTC Therapeutics' unaudited consolidated financial statements for Q2 2025, covering balance sheets, operations, comprehensive income, stockholders' deficit, and cash flows, with notes on policies and pipeline Consolidated Balance Sheets (unaudited) This section presents the unaudited consolidated balance sheets, detailing assets, liabilities, and stockholders' deficit for June 30, 2025, and December 31, 2024 Consolidated Balance Sheets | Metric | June 30, 2025 (in thousands) | December 31, 2024 (in thousands) | | :-------------------------------- | :----------------------------- | :------------------------------- | | Total assets | $2,634,155 | $1,705,024 | | Total liabilities | $2,840,701 | $2,803,095 | | Total stockholders' deficit | $(206,546) | $(1,098,071) | - Total assets increased by approximately $929.1 million from December 31, 2024, to June 30, 2025, primarily driven by increases in cash and cash equivalents and marketable securities[18] - The stockholders' deficit significantly decreased from $(1,098.07) million at December 31, 2024, to $(206.55) million at June 30, 2025, indicating an improvement in equity position[18] Consolidated Statements of Operations (unaudited) This section presents the unaudited consolidated statements of operations, detailing revenues, expenses, and net income (loss) for the three and six months ended June 30, 2025 and 2024 Consolidated Statements of Operations | Metric | Three Months Ended June 30, 2025 (in thousands) | Three Months Ended June 30, 2024 (in thousands) | Six Months Ended June 30, 2025 (in thousands) | Six Months Ended June 30, 2024 (in thousands) | | :-------------------------------------------------- | :---------------------------------------------- | :---------------------------------------------- | :-------------------------------------------- | :-------------------------------------------- | | Total revenues | $178,875 | $186,704 | $1,354,971 | $396,822 | | Net product revenue | $118,329 | $133,220 | $271,755 | $310,824 | | Collaboration and license revenue | $2,941 | $0 | $989,172 | $0 | | Royalty revenue | $57,605 | $53,183 | $94,044 | $84,337 | | Total operating expenses | $213,832 | $226,922 | $419,703 | $482,493 | | Net (loss) income attributable to common stockholders | $(64,849) | $(99,179) | $801,713 | $(190,755) | | Net (loss) income per share—basic | $(0.83) | $(1.29) | $10.22 | $(2.49) | | Net (loss) income per share—diluted | $(0.83) | $(1.29) | $9.29 | $(2.49) | - For the six months ended June 30, 2025, total revenues significantly increased by 241.5% to $1,354.97 million, primarily due to $989.17 million in collaboration and license revenue from the Novartis Agreement[19] - The company reported a net income of $801.71 million for the six months ended June 30, 2025, a substantial turnaround from a net loss of $190.76 million in the prior year period[19] Consolidated Statements of Comprehensive Income (Loss) (unaudited) This section presents the unaudited consolidated statements of comprehensive income (loss), including net income (loss) and other comprehensive income (loss) for the periods ended June 30, 2025 and 2024 Consolidated Statements of Comprehensive Income (Loss) | Metric | Three Months Ended June 30, 2025 (in thousands) | Three Months Ended June 30, 2024 (in thousands) | Six Months Ended June 30, 2025 (in thousands) | Six Months Ended June 30, 2024 (in thousands) | | :------------------------------------ | :---------------------------------------------- | :---------------------------------------------- | :-------------------------------------------- | :-------------------------------------------- | | Net (loss) income | $(64,849) | $(99,179) | $801,713 | $(190,755) | | Other comprehensive income (loss) | $21,305 | $(10,938) | $33,242 | $(15,213) | | Comprehensive (loss) income | $(43,544) | $(110,117) | $834,955 | $(205,968) | - Comprehensive income for the six months ended June 30, 2025, was $834.96 million, a significant improvement from a comprehensive loss of $205.97 million in the same period last year, driven by net income and foreign currency translation gains[21] Consolidated Statements of Stockholders' Deficit (unaudited) This section presents the unaudited consolidated statements of stockholders' deficit, detailing changes in equity components for the periods ended June 30, 2025, and December 31, 2024 Consolidated Statements of Stockholders' Deficit | Metric | June 30, 2025 (in thousands) | December 31, 2024 (in thousands) | | :-------------------------- | :----------------------------- | :------------------------------- | | Additional paid-in capital | $2,631,180 | $2,574,611 | | Accumulated deficit | $(2,845,160) | $(3,646,873) | | Total stockholders' deficit | $(206,546) | $(1,098,071) | - The accumulated deficit decreased by $801.71 million from December 31, 2024, to June 30, 2025, primarily due to the net income reported for the period[22] - Additional paid-in capital increased by $56.57 million, reflecting share-based compensation expense, exercise of options, and employee stock purchase plan issuances[22] Consolidated Statements of Cash Flows (unaudited) This section presents the unaudited consolidated statements of cash flows, detailing cash movements from operating, investing, and financing activities for the six months ended June 30, 2025 and 2024 Consolidated Statements of Cash Flows | Cash Flow Activity | Six Months Ended June 30, 2025 (in thousands) | Six Months Ended June 30, 2024 (in thousands) | | :-------------------------------------- | :-------------------------------------------- | :-------------------------------------------- | | Net cash provided by (used in) operating activities | $811,770 | $(692) | | Net cash used in investing activities | $(606,369) | $(180,855) | | Net cash provided by financing activities | $13,371 | $247,022 | | Net increase in cash and cash equivalents | $237,753 | $58,258 | | Cash and cash equivalents, end of period | $1,033,069 | $668,542 | - Net cash provided by operating activities significantly increased to $811.77 million for the six months ended June 30, 2025, compared to net cash used of $0.69 million in the prior year, primarily due to the $1.0 billion upfront payment from the Novartis Agreement[23][316] - Net cash used in investing activities increased to $606.37 million for the six months ended June 30, 2025, from $180.86 million in the prior year, mainly due to increased purchases of marketable securities and product rights[23][317] 1. The Company (Business Overview & Product Pipeline) This section provides an overview of PTC Therapeutics, its focus on rare disorders, key commercial products, and diversified development pipeline - PTC Therapeutics is a global biopharmaceutical company focused on discovering, developing, and commercializing clinically differentiated medicines for rare disorders, with a diversified pipeline in neurology and metabolism[25] - Key commercial products include Translarna and Emflaza for Duchenne muscular dystrophy (DMD), Sephience for phenylketonuria (PKU), Upstaza/Kebilidi for AADC deficiency, and commercialization rights for Tegsedi and Waylivra in Latin America[26][31][32][33] - The company's pipeline includes votoplam for Huntington's disease (HD) in Phase 2, with a collaboration agreement with Novartis, and vatiquinone for Friedreich's ataxia (FA) with an NDA submitted to the FDA[35][37][38] - Translarna's conditional marketing authorization in the European Economic Area (EEA) was not renewed in March 2025, though individual EU countries may allow continued commercial use[27] - Sephience received FDA approval for PKU in the US on July 28, 2025, and EC marketing authorization in the EEA on June 19, 2025[31][187][198] 2. Summary of significant accounting policies This section outlines the company's significant accounting policies, including financial statement preparation, segment reporting, inventory valuation, revenue recognition, and new accounting standards - The financial statements are unaudited and prepared in accordance with GAAP, with certain information condensed or omitted per SEC rules[42][43][44] - The company operates in one segment: life science, focusing on discovery, development, and commercialization of medicines for rare disorders[49] - Inventory is stated at the lower of cost and net realizable value, with write-downs of $4.3 million for the six months ended June 30, 2025, primarily due to adjustments to reserves and product approaching expiration[55][56] - Revenue recognition for product sales occurs when performance obligations are satisfied, typically upon delivery, net of variable consideration like discounts and rebates[58][59] - The company classifies cash consideration from royalty purchase agreements as debt, amortized using the effective interest method, with the effective interest rate updated quarterly[70] - Goodwill of $82.3 million remains unchanged since the Agilis Merger in August 2018[71][182] - New accounting standards ASU 2023-09 (Income Tax Disclosures) and ASU 2024-03 (Income Statement Expense Disaggregation) are being assessed for impact and planned for adoption when effective[87][88] 3. Leases This section details the company's lease arrangements for office and laboratory spaces, including finance and operating lease liabilities and associated costs - The company leases principal office space in Warren, NJ, laboratory space in Bridgewater, NJ, and other international office spaces[89] Lease Liabilities | Lease Type | June 30, 2025 (in thousands) | December 31, 2024 (in thousands) | | :-------------------------------- | :----------------------------- | :------------------------------- | | Finance lease liabilities-current | $5,358 | $3,000 | | Finance lease liabilities-noncurrent | $13,833 | $15,574 | Operating Lease Cost | Metric | Three Months Ended June 30, 2025 (in thousands) | Three Months Ended June 30, 2024 (in thousands) | Six Months Ended June 30, 2025 (in thousands) | Six Months Ended June 30, 2024 (in thousands) | | :-------------------- | :---------------------------------------------- | :---------------------------------------------- | :-------------------------------------------- | :-------------------------------------------- | | Total operating lease cost | $5,234 | $6,750 | $10,275 | $13,608 | - Operating lease costs decreased by $1.52 million (22.5%) for the three months ended June 30, 2025, and by $3.33 million (24.4%) for the six months ended June 30, 2025, compared to the same periods in 2024[92] 4. Fair value of financial instruments and marketable securities This section describes the fair value measurement of the company's financial instruments and marketable securities, including available-for-sale debt securities and equity investments - The company's marketable securities include available-for-sale debt securities and equity investments, measured at fair value using a three-level hierarchy[53][54][94][95] Marketable Securities | Asset Type | June 30, 2025 (in thousands) | December 31, 2024 (in thousands) | | :-------------------------------- | :----------------------------- | :------------------------------- | | Marketable securities - available for sale | $937,561 | $330,953 | | Marketable securities - equity investments | $34,203 | $29,034 | | ClearPoint Equity Investments | $10,681 | $13,759 | - Available-for-sale debt securities significantly increased to $937.56 million at June 30, 2025, from $330.95 million at December 31, 2024[102][103] - The estimated fair value of the 2026 Convertible Notes was $332.8 million at June 30, 2025, up from $321.3 million at December 31, 2024[107] - The contingent consideration payable balance was written down to zero in Q1 2025 as the probability of triggering remaining milestones was deemed remote, resulting in a $0.8 million gain for the six months ended June 30, 2025[108] 5. Accounts payable and accrued expenses This section details the composition and changes in the company's accounts payable and accrued expenses, including income tax and milestone payables Accounts Payable and Accrued Expenses | Category | June 30, 2025 (in thousands) | December 31, 2024 (in thousands) | | :------------------------------------ | :----------------------------- | :------------------------------- | | Total Accounts payable and accrued expenses | $338,945 | $304,292 | | Income tax payable | $39,923 | $4,701 | | Milestone payable | $25,150 | $11,025 | - Total accounts payable and accrued expenses increased by $34.65 million from December 31, 2024, to June 30, 2025[109] - Income tax payable saw a significant increase from $4.70 million to $39.92 million[109] - Milestone payable increased to $25.15 million, reflecting a $25.0 million regulatory milestone triggered by Sephience's marketing authorization in the EEA[109][156] 6. Capitalization This section outlines the company's capitalization, including its At the Market Offering Sales Agreement for common stock issuance - The company has an At the Market Offering Sales Agreement allowing the sale of common stock up to $125.0 million, with $93.0 million remaining available as of June 30, 2025[110] - No shares were sold under the At the Market Offering during the three and six months ended June 30, 2025 and 2024[110] 7. Net (loss) income per share This section presents the company's basic and diluted net income (loss) per share for the three and six months ended June 30, 2025 and 2024 Net (Loss) Income Per Share | Metric | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | | :-------------------------------- | :------------------------------- | :------------------------------- | :----------------------------- | :----------------------------- | | Net (loss) income, basic | $(64,849) | $(99,179) | $801,713 | $(190,755) | | Weighted-average shares outstanding, basic | 78,151,240 | 76,725,070 | 78,438,830 | 76,610,598 | | Net (loss) income per common share, basic | $(0.83) | $(1.29) | $10.22 | $(2.49) | | Net (loss) income per common share, diluted | $(0.83) | $(1.29) | $9.29 | $(2.49) | - Diluted EPS for the six months ended June 30, 2025, was $9.29, a significant improvement from a diluted loss per share of $(2.49) in the prior year period[112] - The company did not report any dilutive share impact for the three months ended June 30, 2025, and the three and six months ended June 30, 2024, due to net losses[112] 8. Stock award plan This section details the company's stock award plans, including shares available for issuance, stock option activity, restricted stock units, and share-based compensation expense - As of June 30, 2025, 5,104,352 shares are available for issuance under the Amended 2013 LTIP and 1,851,726 shares under the 2020 Inducement Stock Incentive Plan[117][118] Stock Option Activity | Stock Option Activity | Number of options | Weighted-average exercise price | | :-------------------------------- | :---------------- | :------------------------------ | | Outstanding at December 31, 2024 | 8,482,489 | $42.29 | | Granted (6 months ended June 30, 2025) | 822,470 | $46.84 | | Exercised (6 months ended June 30, 2025) | (467,285) | $36.68 | | Outstanding at June 30, 2025 | 8,305,990 | $42.30 | - The weighted average grant date fair value of options granted during the six months ended June 30, 2025, was $25.30 per share[122] Restricted Stock Units | Restricted Stock Units | Number of Shares | Weighted Average Grant Date Fair Value | | :-------------------------------- | :--------------- | :------------------------------------- | | Unvested at December 31, 2024 | 3,527,575 | $33.39 | | Granted (6 months ended June 30, 2025) | 1,576,245 | $46.87 | | Vested (6 months ended June 30, 2025) | (1,134,189) | $37.01 | | Unvested at June 30, 2025 | 3,818,644 | $37.84 | Share-based Compensation Expense | Share-based Compensation Expense | Three Months Ended June 30, 2025 (in thousands) | Three Months Ended June 30, 2024 (in thousands) | Six Months Ended June 30, 2025 (in thousands) | Six Months Ended June 30, 2024 (in thousands) | | :--------------------------------- | :---------------------------------------------- | :---------------------------------------------- | :-------------------------------------------- | :-------------------------------------------- | | Research and development | $9,030 | $9,428 | $17,693 | $18,395 | | Selling, general and administrative | $9,513 | $9,815 | $18,910 | $19,226 | | Total | $18,543 | $19,243 | $36,603 | $37,621 | - Total unrecognized compensation cost related to unvested share-based compensation was approximately $153.3 million as of June 30, 2025, to be recognized over an average of 2.8 years[127] 9. Debt This section describes the company's debt obligations, including royalty purchase agreements and convertible notes, detailing their terms and associated interest expenses - The company sold 90.49% of its royalty rights on Evrysdi net sales to Royalty Pharma for $1.9 billion in upfront cash consideration, which will reduce to 83.33% after Royalty Pharma receives $1.3 billion in aggregate payments[130][136] - The liability for sale of future royalties is amortized using the effective interest method, with an effective interest rate of 9.3% as of June 30, 2025[134][138] 2026 Convertible Notes | Metric | June 30, 2025 (in thousands) | December 31, 2024 (in thousands) | | :-------------------------------- | :----------------------------- | :------------------------------- | | 2026 Convertible Notes Principal | $287,500 | $287,500 | | Less: Debt issuance costs | $(1,487) | $(2,088) | | Net carrying amount | $286,013 | $285,412 | - The 2026 Convertible Notes have a remaining contractual life of approximately 1.2 years as of June 30, 2025, bearing 1.50% annual cash interest[139][147] Interest Expense (2026 Convertible Notes) | Interest Expense (2026 Convertible Notes) | Three Months Ended June 30, 2025 (in thousands) | Three Months Ended June 30, 2024 (in thousands) | Six Months Ended June 30, 2025 (in thousands) | Six Months Ended June 30, 2024 (in thousands) | | :---------------------------------------- | :---------------------------------------------- | :---------------------------------------------- | :-------------------------------------------- | :-------------------------------------------- | | Contractual interest expense | $1,067 | $1,066 | $2,135 | $2,142 | | Amortization of debt issuance costs | $301 | $295 | $601 | $591 | | Total | $1,368 | $1,361 | $2,736 | $2,733 | 10. Commitments and contingencies This section outlines the company's various royalty and milestone payment obligations arising from acquisition agreements and product development - The company has various royalty and milestone payment obligations from agreements like the Agilis Merger, BioElectron Asset Purchase, and Censa Merger[149][150][152][154] - Milestones related to FA and Angelman syndrome from the Agilis Merger are not expected to be achieved due to discontinuation of preclinical programs[151] - A $25.0 million regulatory milestone was triggered in June 2025 for Sephience's marketing authorization in the EEA, payable to former Censa securityholders[156] - Remaining potential sales milestones for Upstaza/Kebilidi are $50.0 million, but the probability of triggering them is considered remote[151] 11. Revenue recognition This section details the company's revenue streams, including net product, collaboration and license, royalty, and manufacturing revenues, and their respective changes Revenue by Type | Revenue Type | Three Months Ended June 30, 2025 (in thousands) | Three Months Ended June 30, 2024 (in thousands) | Six Months Ended June 30, 2025 (in thousands) | Six Months Ended June 30, 2024 (in thousands) | | :-------------------------------- | :---------------------------------------------- | :---------------------------------------------- | :-------------------------------------------- | :-------------------------------------------- | | Net product revenue | $118,329 | $133,220 | $271,755 | $310,824 | | Collaboration and license revenue | $2,941 | $0 | $989,172 | $0 | | Royalty revenue | $57,605 | $53,183 | $94,044 | $84,337 | | Manufacturing revenue | $0 | $301 | $0 | $1,661 | - Net product revenue decreased by $14.9 million (11%) for Q2 2025 and $39.1 million (13%) for YTD 2025, primarily due to decreased sales of Emflaza (generic competition) and Translarna (EC's negative opinion)[271][290] - Collaboration and license revenue increased significantly to $989.17 million for YTD 2025, driven by a $1.0 billion upfront payment from the Novartis Agreement for the votoplam HD program[168][174][291] - Royalty revenue increased by $4.4 million (8%) for Q2 2025 and $9.7 million (12%) for YTD 2025, due to higher Evrysdi sales[167][275][292] - Manufacturing revenue ceased in Q2 2025 following the sale of the gene therapy manufacturing business in June 2024[176][276][293] 12. Intangible assets and goodwill This section details the company's intangible assets and goodwill, including additions, amortization expense, and the impact of milestones on asset valuation Intangible Assets | Intangible Asset | Ending Balance at December 31, 2024 (in thousands) | Additions (in thousands) | Ending Balance at June 30, 2025 (in thousands) | | :----------------------- | :----------------------------------------------- | :----------------------- | :--------------------------------------------- | | Waylivra | $12,397 | $3,293 | $17,395 | | Tegsedi | $18,249 | $2,021 | $22,631 | | Kebilidi | $10,731 | $0 | $10,731 | | Upstaza | $106,937 | $0 | $106,937 | | Sephience | $0 | $25,000 | $25,000 | | Total definite-lived intangibles, gross | $148,314 | $30,314 | $182,694 | - A $25.0 million milestone for Sephience's EEA marketing authorization was recorded as an intangible asset in June 2025[179] - Amortization expense for acquired intangible assets decreased by $46.5 million (86%) for the six months ended June 30, 2025, primarily because the Emflaza rights intangible asset was fully amortized as of February 2024[181][295] - Goodwill remains at $82.3 million as of June 30, 2025, with no changes since the Agilis Merger[182] 13. Segment information This section reports the company's financial performance as a single operating segment, life science, detailing revenues and various expense categories - The company operates as a single operating segment: life science, with performance assessed based on net (loss) income, revenues, R&D, and SG&A expenses[49][183] Segment Financial Performance | Expense Category | Three Months Ended June 30, 2025 (in thousands) | Three Months Ended June 30, 2024 (in thousands) | Six Months Ended June 30, 2025 (in thousands) | Six Months Ended June 30, 2024 (in thousands) | | :-------------------------------- | :---------------------------------------------- | :---------------------------------------------- | :-------------------------------------------- | :-------------------------------------------- | | Total revenues | $178,875 | $186,704 | $1,354,971 | $396,822 | | Cost of product, collaboration and license sales | $8,093 | $4,477 | $15,800 | $9,671 | | Program spend | $49,705 | $52,959 | $97,863 | $101,323 | | Employee costs | $65,593 | $59,558 | $133,292 | $123,555 | | Manufacturing costs | $19,519 | $12,325 | $34,470 | $30,406 | | Administrative costs | $17,896 | $18,623 | $36,310 | $35,495 | | Occupancy costs | $6,643 | $9,588 | $13,825 | $19,481 | | Milestones | $0 | $15,000 | $0 | $15,000 | | Segment net (loss) income | $(64,849) | $(99,179) | $801,713 | $(190,755) | - Segment net income for the six months ended June 30, 2025, was $801.71 million, a significant improvement from a loss of $190.76 million in the prior year[183] 14. Subsequent events This section describes significant events occurring after the reporting period, including regulatory approvals for Sephience and a related Rights Satisfaction Agreement - On July 28, 2025, Sephience was approved by the FDA for PKU in the US, triggering a $32.5 million regulatory milestone payable to former Censa securityholders[187] - On August 5, 2025, the company entered into a Rights Satisfaction Agreement with former Censa securityholders, canceling their rights to receive certain net sales payments for Sephience[188][189] - In exchange, the company paid approximately $225.0 million upfront cash and is obligated to make up to $450.0 million in additional milestone payments based on specified net sales thresholds for Sephience[190][191][192] Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations Management discusses the company's financial condition, operations, and cash flows, covering corporate updates, product performance, pipeline, funding, and the impact of the Novartis collaboration and Translarna's market challenges Our Company This section introduces PTC Therapeutics as a global biopharmaceutical company focused on rare disorders, with a diversified therapeutic portfolio and pipeline - PTC Therapeutics is a global biopharmaceutical company focused on rare disorders, leveraging scientific expertise and commercial infrastructure to provide treatments for unmet medical needs[197] - The company has a diversified therapeutic portfolio with commercial products and product candidates in various development stages, focusing on neurology and metabolism[197] Corporate Updates This section provides recent corporate developments, including regulatory approvals for Sephience and a significant Rights Satisfaction Agreement with former Censa securityholders - Sephience (sepiapterin) received FDA approval for PKU in the US on July 28, 2025, and EC marketing authorization in the EEA on June 19, 2025[198] - The company made regulatory submissions for Sephience in Brazil (Q3 2024) and Japan (Q4 2024), with decisions expected in H2 2025 and Q4 2025, respectively[198] - A Rights Satisfaction Agreement was entered into on August 5, 2025, with former Censa securityholders, canceling their rights to certain net sales payments for Sephience in exchange for an upfront cash payment of approximately $225.0 million and potential additional milestone payments up to $450.0 million[199][200][202][203][204] Global Commercial Footprint This section details the commercial performance and market status of key products like Translarna, Emflaza, Upstaza/Kebilidi, Tegsedi, Waylivra, and Evrysdi - Translarna net sales were $59.5 million for Q2 2025, but its EEA marketing authorization was not renewed in March 2025, posing a substantial risk to future revenue in the region[208][211] - Emflaza net sales were $36.4 million for Q2 2025, facing a 23% decrease due to additional generic competition following the expiration of its orphan drug exclusivity for patients five years and older in February 2024[210][213] - Upstaza/Kebilidi, a gene therapy for AADC deficiency, received FDA accelerated approval in November 2024 (marketed as Kebilidi in the US) and EC approval in July 2022 (Upstaza in EEA)[215] - The company holds commercialization rights for Tegsedi and Waylivra in Latin America and the Caribbean, with both products having received marketing authorizations in various regions for rare diseases[216] - Evrysdi, for spinal muscular atrophy (SMA), has received marketing authorization in over 100 countries, with label expansions approved by the FDA (May 2022) and EC (August 2023) to include infants under two months old[217] Diversified Development Pipeline This section highlights the company's development pipeline, focusing on votoplam for Huntington's disease and vatiquinone for Friedreich's Ataxia, including clinical trial results and regulatory status - Votoplam (PTC518) for Huntington's disease (HD) met primary endpoints of blood HTT lowering and safety in its Phase 2 study, showing dose-dependent mHTT lowering in blood and CSF, and favorable clinical trends[218][219] - The company entered a License and Collaboration Agreement with Novartis in November 2024 for the votoplam HD program, which closed in January 2025[220] - Vatiquinone for Friedreich's Ataxia (FA) demonstrated significant benefit on key disease subscales in its Phase 3 MOVE-FA trial, and durable treatment benefit in long-term extension studies[221] - An NDA for vatiquinone for FA was submitted to the FDA in December 2024, granted priority review in February 2025, with a target action date of August 19, 2025[221] Funding This section discusses the company's funding sources, financial position, and future funding requirements for operations, clinical trials, and potential acquisitions - The company's operations are primarily financed through convertible senior notes, stock offerings, royalty purchase agreements, collaborations (e.g., Roche, Novartis), and product sales[224][226] - As of June 30, 2025, the company had an accumulated deficit of $2,845.2 million, but reported a net income of $801.7 million for the six months ended June 30, 2025[230] - Cash flows from product sales, Roche royalties, and Novartis milestones, along with existing cash, cash equivalents, and marketable securities, are expected to fund operations for at least the next twelve months[40] - Future funding requirements include significant expenses for commercialization, ongoing clinical trials, potential acquisitions, and milestone payments for Sephience ($25.0 million for EC, $32.5 million for FDA) and vatiquinone ($75.0 million potential in 2026)[231][233][235][236] Financial operations overview This section provides an overview of the company's financial operations, including net product revenues, collaboration and license revenue, royalty revenue, and research and development expenses - Net product revenues are primarily from Translarna (outside US) and Emflaza (US), recognized upon customer control of product, net of variable consideration[239][240] Net Product Revenue by Product | Product | Three Months Ended June 30, 2025 (in thousands) | Three Months Ended June 30, 2024 (in thousands) | Six Months Ended June 30, 2025 (in thousands) | Six Months Ended June 30, 2024 (in thousands) | | :---------------- | :---------------------------------------------- | :---------------------------------------------- | :-------------------------------------------- | :-------------------------------------------- | | Translarna | $59,470 | $70,350 | $145,624 | $173,934 | | Emflaza | $36,353 | $47,296 | $84,142 | $104,777 | | Upstaza/Kebilidi | $11,889 | $1,589 | $41,989 | $32,113 | | All other products | $10,617 | $13,985 | $0 | $0 | | Total net product revenue | $118,329 | $133,220 | $271,755 | $310,824 | - Collaboration and license revenue for YTD 2025 was $989.17 million, primarily from the Novartis Agreement's $1.0 billion upfront payment, partially offset by a $3.5 million refund for a prior collaboration[248] - Royalty revenue from Evrysdi sales increased to $94.0 million for YTD 2025, up from $84.3 million in YTD 2024[247] R&D Expense by Category | R&D Expense Category | Three Months Ended June 30, 2025 (in thousands) | Three Months Ended June 30, 2024 (in thousands) | Six Months Ended June 30, 2025 (in thousands) | Six Months Ended June 30, 2024 (in thousands) | | :------------------------------------ | :---------------------------------------------- | :---------------------------------------------- | :-------------------------------------------- | :-------------------------------------------- | | Development | $51,150 | $49,931 | $98,999 | $103,355 | | Research | $15,691 | $18,204 | $29,538 | $31,703 | | Milestones | $0 | $15,000 | $0 | $15,000 | | Payroll, benefits, and share-based stock compensation | $36,995 | $37,302 | $75,229 | $76,654 | | Facilities and other | $9,154 | $11,732 | $18,197 | $21,586 | | Total research and development | $112,990 | $132,169 | $221,963 | $248,298 | - Total R&D expense decreased by $19.2 million (15%) for Q2 2025 and $26.3 million (11%) for YTD 2025, primarily due to a $15.0 million regulatory milestone in Q2 2024 not recurring and decreases in program spend and facility-based expenses[259][262][279][296] - Selling, general and administrative expense increased by $15.8 million (23%) for Q2 2025 and $23.5 million (16%) for YTD 2025, reflecting continued investment in commercial activities and an expanding commercial portfolio[280][298] Results of operations This section analyzes the company's financial performance, detailing changes in revenues, expenses, and net income (loss) for the three and six months ended June 30, 2025 and 2024 Results of Operations (Three Months Ended June 30) | Metric | Three Months Ended June 30, 2025 (in thousands) | Three Months Ended June 30, 2024 (in thousands) | Change (2025 vs 2024) | | :-------------------------------------------------- | :---------------------------------------------- | :---------------------------------------------- | :-------------------- | | Net product revenue | $118,329 | $133,220 | $(14,891) | | Collaboration and license revenue | $2,941 | $0 | $2,941 | | Royalty revenue | $57,605 | $53,183 | $4,422 | | Manufacturing revenue | $0 | $301 | $(301) | | Cost of product, collaboration and license sales (excl. amortization) | $11,420 | $15,527 | $(4,107) | | Amortization of acquired intangible assets | $4,061 | $2,865 | $1,196 | | Research and development expense | $112,990 | $132,169 | $(19,179) | | Selling, general and administrative expense | $85,262 | $69,500 | $15,762 | | Change in the fair value of contingent consideration | $0 | $5,100 | $(5,100) | | Tangible asset impairment and losses (gains) on transactions, net | $99 | $1,761 | $(1,662) | | Interest expense, net | $(30,358) | $(43,490) | $13,132 | | Other expense, net | $(5,737) | $(2,025) | $(3,712) | | Income tax benefit (expense) | $6,203 | $(13,446) | $19,649 | Results of Operations (Six Months Ended June 30) | Metric | Six Months Ended June 30, 2025 (in thousands) | Six Months Ended June 30, 2024 (in thousands) | Change (2025 vs 2024) | | :-------------------------------------------------- | :-------------------------------------------- | :-------------------------------------------- | :-------------------- | | Net product revenue | $271,755 | $310,824 | $(39,069) | | Collaboration and license revenue | $989,172 | $0 | $989,172 | | Royalty revenue | $94,044 | $84,337 | $9,707 | | Manufacturing revenue | $0 | $1,661 | $(1,661) | | Cost of product, collaboration and license sales (excl. amortization) | $24,282 | $30,267 | $(5,985) | | Amortization of acquired intangible assets | $7,859 | $54,395 | $(46,536) | | Research and development expense | $221,963 | $248,298 | $(26,335) | | Selling, general and administrative expense | $166,223 | $142,772 | $23,451 | | Change in the fair value of contingent consideration | $(800) | $5,000 | $(5,800) | | Tangible asset impairment and losses (gains) on transactions, net | $176 | $1,761 | $(1,585) | | Interest expense, net | $(64,450) | $(84,324) | $19,874 | | Other expense, net | $(12,042) | $(434) | $(11,608) | | Income tax expense | $(57,063) | $(20,326) | $(36,737) | - Interest expense, net decreased by $13.1 million (30%) for Q2 2025 and $19.9 million (24%) for YTD 2025, primarily due to increased investment income from marketable securities[284][301] - Income tax benefit for Q2 2025 was $6.2 million, a $19.6 million change from an expense of $13.4 million in Q2 2024, driven by projected utilization of additional tax attributes[286] - Income tax expense for YTD 2025 increased by $36.7 million (over 100%) to $57.1 million, attributable to the recognition of revenue from the Novartis Agreement[303] Liquidity and capital resources This section assesses the company's liquidity and capital resources, including cash position, cash flow activities, and future funding needs - As of June 30, 2025, the company had $1.99 billion in cash, cash equivalents, and marketable securities[313] - Net cash provided by operating activities was $811.8 million for YTD 2025, a significant increase from net cash used of $0.7 million in YTD 2024, primarily due to the $1.0 billion upfront payment from the Novartis Agreement[316] - Net cash used in investing activities increased to $606.4 million for YTD 2025, from $180.9 million in YTD 2024, mainly due to purchases of marketable securities and product rights[317] - Net cash provided by financing activities decreased to $13.4 million for YTD 2025, from $247.0 million in YTD 2024, as prior year included proceeds from sales of future royalties[318] - The company expects to incur significant expenses for commercialization, R&D, and potential acquisitions, and will need to generate substantial revenues or obtain additional funding to achieve and sustain profitability[319][320][330] Item 3. Quantitative and Qualitative Disclosures About Market Risk There were no material changes in the company's market risk or its management during the period ended June 30, 2025, compared to disclosures in the 2024 Annual Report - No material changes in market risk or its management occurred during the quarter ended June 30, 2025[332] Item 4. Controls and Procedures Management, including the CEO and CFO, evaluated the effectiveness of disclosure controls and procedures as of June 30, 2025, concluding they were effective at a reasonable assurance level. No material changes in internal control over financial reporting occurred during the quarter - Disclosure controls and procedures were evaluated as effective at the reasonable assurance level as of June 30, 2025[333] - No material changes in internal control over financial reporting occurred during the quarter ended June 30, 2025[334] PART II—OTHER INFORMATION Item 1. Legal Proceedings The company is not currently aware of any material legal proceedings against it, though it is subject to claims, legal proceedings, and disputes in the ordinary course of business - The company is not currently aware of any material legal proceedings against it[336] Item 1A. Risk Factors This section updates risk factors, highlighting international operations, pricing regulations, third-party reimbursement, and reliance on manufacturers and collaborators, with concerns over Translarna's non-renewal and Emflaza's generic competition - International operations expose the company to risks including political/regulatory developments, financial risks (currency fluctuations, payment cycles), and challenges in staffing and management[338] - The company faces substantial risk of losing significant revenue from Translarna sales in the EEA due to the EC's decision not to renew its marketing authorization[211][365] - Unfavorable pricing regulations, third-party reimbursement practices, and healthcare reform initiatives could harm the business, particularly for orphan drugs like Emflaza, which faces generic competition[345][358][307] - Reliance on third-party manufacturers and distributors for products like Sephience, Translarna, Emflaza, and Upstaza/Kebilidi increases risks of insufficient quantities, quality issues, and supply chain disruptions[375][376][388] - Dependence on collaborations (e.g., Roche for Evrysdi) means limited control over resource allocation and potential delays or terminations if collaborations are unsuccessful[389][390][391] - Future changes in legal and regulatory requirements, including those from a new federal administration or Supreme Court decisions, could introduce new risks and impact product approval and market access[394][397] Item 5. Other Information This section details director and officer trading arrangements, specifically a Rule 10b5-1 trading arrangement adopted by the Chief Business Officer for the sale of up to 48,550 shares - Eric Pauwels (Chief Business Officer) adopted a Rule 10b5-1 trading arrangement on June 4, 2025, for the sale of up to 48,550 shares, effective until June 3, 2026[400] Item 6. Exhibits This section lists the exhibits filed with the Quarterly Report on Form 10-Q, including certifications from the Principal Executive Officer and Principal Financial Officer, and Inline XBRL documents - The report includes certifications from the Principal Executive Officer and Principal Financial Officer (Exhibits 31.1, 31.2, 32.1, 32.2) and Inline XBRL documents (Exhibits 101.INS, 101.SCH, 101.CAL, 101.LAB, 101.PRE, 101.DEF, 104)[401]