uniQure(QURE) - 2025 Q2 - Quarterly Report

PART I – FINANCIAL INFORMATION Item 1. Financial Statements This section presents uniQure N.V.'s unaudited consolidated financial statements, detailing financial position, operations, equity, and cash flows UNAUDITED CONSOLIDATED BALANCE SHEETS This section presents the company's financial position, detailing assets, liabilities, and equity at specific reporting dates Consolidated Balance Sheet Highlights (in thousands) | Item | June 30, 2025 | December 31, 2024 | | :-------------------------------- | :------------ | :---------------- | | Cash and cash equivalents | $253,778 | $158,930 | | Current investment securities | $123,196 | $208,591 | | Total current assets | $408,573 | $390,289 | | Total assets | $584,890 | $556,536 | | Total current liabilities | $40,929 | $40,053 | | Total liabilities | $588,893 | $563,288 | | Total shareholders' (deficit) / equity | $(4,003) | $(6,752) | - Cash and cash equivalents increased significantly from $158.9 million at December 31, 2024, to $253.8 million at June 30, 2025, while current investment securities decreased from $208.6 million to $123.2 million[13] - The company's total shareholders' deficit improved from $(6.8) million at December 31, 2024, to $(4.0) million at June 30, 2025[13] UNAUDITED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS This section details the company's revenues, expenses, and net loss over specific reporting periods Consolidated Statements of Operations and Comprehensive Loss (in thousands) | Item | Three months ended June 30, 2025 | Three months ended June 30, 2024 | Six months ended June 30, 2025 | Six months ended June 30, 2024 | | :-------------------------------- | :------------------------------- | :------------------------------- | :----------------------------- | :----------------------------- | | Total revenues | $5,262 | $11,126 | $6,829 | $19,611 | | Research and development expenses | $(35,383) | $(33,655) | $(71,523) | $(74,347) | | Selling, general and administrative expenses | $(13,500) | $(15,767) | $(24,408) | $(29,704) | | Net loss | $(37,719) | $(56,299) | $(81,356) | $(121,917) | | Basic and diluted net loss per ordinary share | $(0.69) | $(1.16) | $(1.49) | $(2.51) | - Total revenues decreased by 52.7% for the three months ended June 30, 2025, compared to the same period in 2024, primarily due to the absence of contract manufacturing and collaboration revenues following the Lexington Transaction[16] - Net loss improved by 33.0% for the three months ended June 30, 2025, to $(37.7) million from $(56.3) million in the prior year, and by 33.3% for the six months ended June 30, 2025, to $(81.4) million from $(121.9) million[16] UNAUDITED CONSOLIDATED STATEMENTS OF SHAREHOLDERS' (DEFICIT) / EQUITY This section outlines changes in the company's shareholders' equity, including share issuances and accumulated deficit Shareholders' (Deficit) / Equity Movement (in thousands) | Item | June 30, 2025 | December 31, 2024 | | :-------------------------------- | :------------ | :---------------- | | Ordinary shares (No. of shares) | 54,865,673 | 48,988,087 | | Additional paid-in capital | $1,262,971 | $1,173,068 | | Accumulated deficit | $(1,211,321) | $(1,129,965) | | Total shareholders' (deficit) / equity | $(4,003) | $(6,752) | - The number of ordinary shares issued and outstanding increased from 48,988,087 at December 31, 2024, to 54,865,673 at June 30, 2025, primarily due to a follow-on public offering[13][21] - Additional paid-in capital increased by $89.9 million, reflecting proceeds from the public offering and share-based compensation[21] UNAUDITED CONSOLIDATED STATEMENTS OF CASH FLOWS This section presents the company's cash inflows and outflows from operating, investing, and financing activities Consolidated Statements of Cash Flows (in thousands) | Item | Six months ended June 30, 2025 | Six months ended June 30, 2024 | | :-------------------------------- | :----------------------------- | :----------------------------- | | Net cash used in operating activities | $(83,995) | $(93,286) | | Net cash generated from investing activities | $93,172 | $141,922 | | Net cash generated from financing activities | $80,714 | $50 | | Net increase in cash, cash equivalents and restricted cash | $94,943 | $46,495 | | Cash, cash equivalents and restricted cash at end of period | $255,272 | $291,039 | - Net cash used in operating activities decreased to $(84.0) million for the six months ended June 30, 2025, from $(93.3) million in the prior year, primarily due to a lower net loss and changes in working capital[24] - Net cash generated from financing activities significantly increased to $80.7 million in 2025, driven by proceeds from a follow-on public offering, compared to $0.1 million in 2024[24] NOTES TO CONSOLIDATED FINANCIAL STATEMENTS This section provides detailed explanations of the company's accounting policies and specific financial statement line items 1 General business information This section provides an overview of the company's business, incorporation, and stock listing - uniQure N.V. is a gene therapy company incorporated in the Netherlands, focused on developing single treatments for rare and devastating diseases. Its ordinary shares are listed on the Nasdaq Global Select Market under the symbol "QURE"[26][27] 2 Summary of significant accounting policies This section outlines the key accounting principles and policies applied in preparing the financial statements - The unaudited consolidated financial statements are prepared in compliance with U.S. GAAP and SEC interim financial reporting rules, presented in U.S. dollars, and reflect normal recurring adjustments[28][29][30] - There have been no material changes to the Company's significant accounting policies or new accounting pronouncements with a material impact during the six months ended June 30, 2025[34][35] 3 Other investments This section details the company's holdings in convertible promissory notes and non-publicly traded equity securities - The Company holds a convertible promissory note with a nominal amount of $12.5 million, bearing 8.0% interest, with a balance of $14.1 million as of June 30, 2025. Interest income of $0.5 million was recognized for the six months ended June 30, 2025[36] - Investments in non-publicly traded equity securities, valued at cost less impairment, totaled $15.6 million as of June 30, 2025, with no recognized gains, losses, or impairments during the period[37] 4 Investment securities This section describes the company's investment in government debt securities, including their amortized cost and fair value Investment Securities (Government Debt, held-to-maturity, in thousands) | Item | June 30, 2025 | December 31, 2024 | | :-------------------- | :------------ | :---------------- | | Amortized cost | $123,196 | $208,591 | | Estimated fair value | $123,364 | $208,755 | - The Company's current investment securities, primarily short-term U.S. and European government debt, decreased from $208.6 million at December 31, 2024, to $123.2 million at June 30, 2025[38] 5 Fair value measurement This section details the fair value measurement of financial instruments, particularly contingent consideration liabilities - The fair value of contingent consideration related to the uniQure France SAS acquisition increased from $10.9 million at December 31, 2024, to $15.9 million at June 30, 2025, primarily due to an unrealized change in fair value and currency translation effects[48][50] - If AMT-260 were assumed to advance into a Phase III clinical study, the fair value of contingent consideration would increase to $51.4 million[49] 6 Accrued expenses and other current liabilities This section provides a breakdown of the company's accrued expenses and other short-term liabilities Accrued Expenses and Other Current Liabilities (in thousands) | Item | June 30, 2025 | December 31, 2024 | | :-------------------------------------------------------------------------------- | :------------ | :---------------- | | Accruals for goods received from and services provided by vendors-not yet billed | $13,878 | $10,109 | | Personnel related accruals and liabilities | $8,285 | $12,583 | | Liability owed to the Purchaser pursuant to the Royalty Financing Agreement | $5,248 | $4,951 | | Current portion of firm purchase commitment liability | $4,068 | $1,582 | | Total | $31,479 | $29,225 | - Total accrued expenses and other current liabilities increased to $31.5 million at June 30, 2025, from $29.2 million at December 31, 2024, driven by higher accruals for vendor services and firm purchase commitments[53] 7 Long-term debt This section details the company's long-term debt obligations, including principal outstanding and interest expenses - The Company's total principal outstanding under the Hercules debt facility was $50.0 million as of June 30, 2025, following a $50.0 million prepayment in July 2024[55][56] - Interest expense on the Hercules debt facility decreased to $1.8 million for the three months ended June 30, 2025, from $3.7 million in the prior year, primarily due to the principal repayment and lower market interest rates[58][151] - The Company recognized a foreign currency gain of $4.3 million on the facility for the three months ended June 30, 2025, compared to a loss of $0.7 million in the same period in 2024[57] 8 Royalty Financing Agreement This section describes the terms and financial impact of the company's royalty financing agreement - Under the Royalty Financing Agreement, the Company received an upfront payment of $375.0 million in exchange for rights to the lowest royalty tier on HEMGENIX® sales, with the Purchaser receiving 1.85 to 2.25 times the upfront payment depending on the date[61][62] - The liability from the royalty financing agreement increased to $460.5 million as of June 30, 2025, from $439.9 million at December 31, 2024, reflecting $27.1 million in interest expense for the period and $6.5 million in royalty payments[67] - The effective interest rate for the royalty financing agreement is expected to be within a range of 12.0% to 13.5% per annum[65] 9 Shareholders' (deficit) / equity This section details changes in the company's share capital and additional paid-in capital from financing activities - In January and February 2025, the Company completed a follow-on public offering, raising net proceeds of $70.1 million and an additional $10.4 million from underwriters' option exercise, totaling $80.5 million[68][69] 10 Share-based compensation This section outlines the company's share-based compensation expense and related unrecognized costs Share-based Compensation Expense (in thousands) | Item | Three months ended June 30, 2025 | Three months ended June 30, 2024 | Six months ended June 30, 2025 | Six months ended June 30, 2024 | | :-------------------------------- | :------------------------------- | :------------------------------- | :----------------------------- | :----------------------------- | | Research and development | $2,758 | $2,856 | $5,221 | $6,281 | | Selling, general and administrative | $2,326 | $3,334 | $4,273 | $6,755 | | Total | $5,084 | $6,880 | $9,494 | $14,071 | - Total share-based compensation expense decreased by 26.1% to $5.1 million for the three months ended June 30, 2025, and by 32.5% to $9.5 million for the six months ended June 30, 2025, primarily due to the divestment of commercial manufacturing activities and a restructuring in August 2024[72][144][149][171][176] - As of June 30, 2025, unrecognized share-based compensation expense related to unvested awards totaled $38.4 million, with a weighted-average remaining recognition period of 2.34 years[74] 11 Segment Reporting This section describes the company's operating segments and their financial performance - The Company operates as a single operating segment, managing research and development activities for gene therapies due to common infrastructure and resources[82] - Segment operating loss for the three months ended June 30, 2025, was $(32.3) million, consistent with the prior year, and $(66.6) million for the six months, an improvement from $(75.4) million in 2024[84] 12 CSL Behring collaboration This section details the financial impact and accounting treatment of the collaboration with CSL Behring - Following the divestiture of commercial manufacturing activities in July 2024, the Company now recognizes costs related to HEMGENIX® purchase from Genezen net of sales income to CSL Behring as 'Other expense'[86] - Accounts receivable related to collaboration services and royalty revenue decreased slightly from $5.88 million at December 31, 2024, to $5.61 million at June 30, 2025[87] 13 Other income This section provides a breakdown of the company's other income sources, including grants and reagent sales Other Income (in thousands) | Item | Three months ended June 30, 2025 | Three months ended June 30, 2024 | Six months ended June 30, 2025 | Six months ended June 30, 2024 | | :-------------------------------- | :------------------------------- | :------------------------------- | :----------------------------- | :----------------------------- | | Sale of critical reagents to Genezen | $0 | $0 | $6,000 | $0 | | Research and development grants | $1,392 | $1,662 | $3,111 | $2,886 | | Sublease income | $801 | $173 | $1,259 | $325 | | Total | $2,597 | $1,983 | $10,903 | $3,359 | - Total other income increased significantly to $10.9 million for the six months ended June 30, 2025, primarily due to a one-time $6.0 million sale of critical reagents to Genezen[88] 14 Income taxes This section details the company's income tax expense and effective tax rate - The Company recorded deferred tax expense of $0.4 million for the three months and $0.9 million for the six months ended June 30, 2025, related to U.S. operations[89] - The effective income tax rate was 1.1% for both the three and six months ended June 30, 2025, substantially lower than the Netherlands' enacted rate of 25.8% due to valuation allowances against deferred tax assets[90] 15 Basic and diluted earnings per share This section explains the calculation of basic and diluted loss per share, including anti-dilutive items - Due to net losses, all potentially dilutive ordinary shares (stock options, RSUs, PSUs) were anti-dilutive and excluded from the computation of loss per share for the three and six months ended June 30, 2025[91] Anti-dilutive Ordinary Share Equivalents | Item | Three months ended June 30, 2025 | Six months ended June 30, 2025 | | :-------------------------------- | :------------------------------- | :----------------------------- | | Stock options under 2014 Plans | 5,321,862 | 5,321,862 | | Non-vested RSUs and PSUs | 2,442,969 | 2,442,969 | | Total anti-dilutive ordinary share equivalents | 7,764,831 | 7,764,831 | 16 Subsequent events This section reports on events occurring after the reporting period that may require disclosure - No subsequent events requiring disclosure were reported[93] Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations This section provides management's perspective on uniQure N.V.'s financial condition and results of operations, highlighting business developments, financial performance, critical accounting policies, and liquidity. It covers the company's gene therapy pipeline, regulatory progress for AMT-130, and financial results for the three and six months ended June 30, 2025, compared to 2024 Overview This section provides a high-level introduction to the company's gene therapy focus and clinical pipeline - uniQure N.V. is a gene therapy leader focused on single treatments for rare and devastating diseases, advancing clinical candidates for Huntington's disease (AMT-130), refractory MTLE (AMT-260), Fabry disease (AMT-191), and SOD1-ALS (AMT-162)[96] Business Developments This section highlights recent strategic and operational advancements across the company's programs Huntington's disease program (AMT-130) This section details the clinical and regulatory progress of the AMT-130 program for Huntington's disease - AMT-130 received Breakthrough Therapy designation from the FDA in April 2025, supported by Phase I/II clinical data showing potential for meaningful slowing of disease progression in Huntington's disease[97] - The FDA also granted RMAT designation in June 2024, and previously Orphan Drug and Fast Track designations for AMT-130[98] - The company expects to present 3-year follow-up data from Phase I/II studies in Q3 2025 to support a potential Biologics License Application (BLA) submission[100] Regulatory Alignment This section outlines key agreements and designations received from regulatory authorities for AMT-130 - In December 2024, the FDA agreed that Phase I/II data for AMT-130, compared to natural history external control, may serve as the primary basis for an Accelerated Approval BLA submission, using cUHDRS as an intermediate clinical endpoint and NfL reductions as supportive evidence[102] - In June 2025, the FDA agreed on using ENROLL-HD as the external control dataset for the primary efficacy analysis of 3-year cUHDRS change in high-dose AMT-130 patients[103] - The FDA also agreed that AMT-130 manufacturing process validation could leverage HEMGENIX® experience, complemented by additional GMP batches and a single process performance qualification batch[104] Financing This section details recent capital raising activities, including public offerings - In January and February 2025, the Company completed a follow-on public offering, raising net proceeds of $70.1 million and an additional $10.4 million, respectively, totaling $80.5 million[106] Organization This section reports on key organizational changes and executive appointments - Kylie O'Keefe was appointed Chief Customer and Strategy Officer in June 2025, responsible for leading commercial and medical affairs[107] Recent Developments of other Product Candidates This section provides updates on the clinical progress of other product candidates in the pipeline Temporal lobe epilepsy program (AMT-260) This section details the clinical progress of the AMT-260 program for refractory MTLE - The Phase I/IIa GenTLE clinical trial for AMT-260 in refractory MTLE dosed its first patient in November 2024[110] - Initial safety and exploratory efficacy data from the first patient showed a 92% reduction in seizure frequency over five months with no serious safety events[110] Fabry disease program (AMT-191) This section outlines the clinical and regulatory progress of the AMT-191 program for Fabry disease - The Phase I/IIa clinical trial for AMT-191 in Fabry disease dosed its first patient in August 2024, and received Orphan Drug and Fast Track Designations from the FDA in September and October 2024, respectively[112] - An independent data monitoring committee recommended proceeding with enrollment in the second cohort in February 2025, following a favorable safety review of the first two patients[113] Amyotrophic Lateral Sclerosis (AMT-162) This section details the clinical progress of the AMT-162 program for SOD1-ALS - The Phase I/II EPISOD1 trial for AMT-162 in SOD1-ALS dosed its first patient in October 2024, with the IDMC recommending proceeding to the second cohort in January 2025 after reviewing safety data[116] - Initial data from the EPISOD1 study is expected in the first half of 2026[116] Financial Overview This section provides a summary of the company's key financial performance indicators and position Key Financials (in thousands) | Item | Three months ended June 30, 2025 | Three months ended June 30, 2024 | Six months ended June 30, 2025 | Six months ended June 30, 2024 | | :-------------------------------- | :------------------------------- | :------------------------------- | :----------------------------- | :----------------------------- | | Total revenues | $5,262 | $11,126 | $6,829 | $19,611 | | Research and development expenses | $(35,383) | $(33,655) | $(71,523) | $(74,347) | | Selling, general and administrative expenses | $(13,500) | $(15,767) | $(24,408) | $(29,704) | | Net loss | $(37,719) | $(56,299) | $(81,356) | $(121,917) | - Cash and cash equivalents and investment securities increased to $377.0 million as of June 30, 2025, from $367.5 million at December 31, 2024[118] - The Company's net loss improved to $(37.7) million for Q2 2025 and $(81.4) million for H1 2025, compared to $(56.3) million and $(121.9) million for the respective periods in 2024[118] Critical Accounting Policies and Estimates This section discusses the company's significant accounting policies and estimates that require management judgment - There were no material changes to the Company's critical accounting policies during the six months ended June 30, 2025[120] - Research and development expenses are expensed as incurred and include costs for preclinical and clinical trials, manufacturing development, and changes in fair value of contingent consideration[122][123] - Following the Lexington Transaction, contract manufacturing revenues are no longer recognized, and other expenses primarily consist of costs incurred under the commercial supply agreement with Genezen[121][127] Results of Operations This section analyzes the company's financial performance for the reported periods, comparing revenues and expenses Comparison of the three months ended June 30, 2025 and 2024 This section compares the company's financial results for the three months ended June 30, 2025, and 2024 Revenue (3 months) This section analyzes the company's revenue streams and their changes over the reporting period Revenue (in thousands) | Item | Three months ended June 30, 2025 | Three months ended June 30, 2024 | Change | | :-------------------------- | :------------------------------- | :------------------------------- | :----- | | License revenues | $5,262 | $1,869 | $3,393 | | Contract manufacturing revenues | $0 | $2,124 | $(2,124) | | Collaboration revenues | $0 | $7,133 | $(7,133) | | Total revenues | $5,262 | $11,126 | $(5,864) | - Total revenues decreased by $5.9 million (52.7%) due to the absence of contract manufacturing and collaboration revenues following the Lexington Transaction[130][132][133] - License revenues increased by $3.4 million (181.5%) to $5.3 million, driven by higher royalty payments on HEMGENIX® sales[131] Cost of contract manufacturing (3 months) This section details the costs associated with contract manufacturing activities - Cost of contract manufacturing was nil for the three months ended June 30, 2025, compared to $7.2 million in the prior year, due to the Lexington Transaction in July 2024[135] R&D expense (3 months) This section analyzes the company's research and development expenses and their drivers R&D Expenses (in thousands) | Item | Three months ended June 30, 2025 | Three months ended June 30, 2024 | Change | | :--------------------------------------- | :------------------------------- | :------------------------------- | :----- | | Huntington's disease (AMT-130) | $10,517 | $2,894 | $7,623 | | Employee and contractor-related expenses | $9,523 | $13,035 | $(3,512) | | Fair value changes related to contingent consideration | $2,254 | $(1,786) | $4,040 | | Total research and development expenses | $35,383 | $33,655 | $1,728 | - Total R&D expenses increased by $1.7 million (5.1%) to $35.4 million, primarily driven by a $7.6 million increase in external costs for AMT-130 BLA preparation[136][139] - Personnel and contractor-related expenses decreased by $3.5 million due to the Lexington Transaction and August 2024 restructuring[144] - A $2.3 million loss was recognized from an increase in the fair value of contingent consideration for uniQure France SAS, compared to a $1.8 million gain in the prior year[144] Selling, general and administrative expenses (3 months) This section details the company's selling, general, and administrative expenses and their changes SG&A Expenses (in thousands) | Item | Three months ended June 30, 2025 | Three months ended June 30, 2024 | Change | | :--------------------------------------- | :------------------------------- | :------------------------------- | :----- | | Employee and contractor-related expenses | $5,397 | $5,669 | $(272) | | Professional fees | $3,428 | $4,053 | $(625) | | Share-based compensation expense | $2,326 | $3,334 | $(1,008) | | Total selling, general and administrative expenses | $13,500 | $15,767 | $(2,267) | - SG&A expenses decreased by $2.3 million (14.4%) to $13.5 million, primarily due to reductions in personnel, contractor-related expenses, and share-based compensation following the Lexington Transaction and restructuring[146][149] Other items, net (3 months) This section analyzes other income and expenses not directly related to core operations Other Items, Net (in thousands) | Item | Three months ended June 30, 2025 | Three months ended June 30, 2024 | Change | | :--------------------------------------- | :------------------------------- | :------------------------------- | :----- | | Research and development grants | $1,392 | $1,662 | $(270) | | Sublease income / (expense), net | $347 | $(63) | $410 | | Supply of HEMGENIX® to CSL Behring | $(1,731) | $0 | $(1,731) | | Total other income, net | $412 | $1,747 | $(1,335) | - Net other items decreased by $1.3 million, primarily due to $1.7 million in net other expenses related to the purchase of HEMGENIX® from Genezen, net of sales to CSL Behring, following the Lexington Transaction[148] Other non-operating items, net (3 months) This section details non-operating income and expenses, including interest and foreign currency effects Other Non-Operating Items, Net (in thousands) | Item | Three months ended June 30, 2025 | Three months ended June 30, 2024 | Change | | :--------------------------------------- | :------------------------------- | :------------------------------- | :----- | | Interest income | $3,524 | $5,805 | $(2,281) | | Interest expense - Royalty Financing Agreement | $(13,770) | $(12,438) | $(1,332) | | Interest expense - Hercules debt facility | $(1,821) | $(3,719) | $1,898 | | Foreign currency gains, net | $18,638 | $(989) | $19,627 | | Total non-operating expense, net | $6,571 | $(11,341) | $17,912 | - Net non-operating income significantly improved by $17.9 million, primarily driven by a $19.6 million increase in net foreign currency gains[150][153] - Interest income decreased by $2.3 million due to lower cash and investment securities balances, while Hercules debt interest expense decreased by $1.9 million due to principal repayment and lower market rates[150][151] Income tax expense (3 months) This section details the company's income tax expense for the reporting period - Income tax expense decreased to $0.4 million for the three months ended June 30, 2025, from $0.9 million in the prior year[154] Comparison of the six months ended June 30, 2025 and 2024 This section compares the company's financial results for the six months ended June 30, 2025, and 2024 Revenue (6 months) This section analyzes the company's revenue streams and their changes over the reporting period Revenue (in thousands) | Item | Six months ended June 30, 2025 | Six months ended June 30, 2024 | Change | | :-------------------------- | :----------------------------- | :----------------------------- | :----- | | License revenues | $6,829 | $3,071 | $3,758 | | Contract manufacturing revenues | $0 | $6,114 | $(6,114) | | Collaboration revenues | $0 | $10,426 | $(10,426) | | Total revenues | $6,829 | $19,611 | $(12,782) | - Total revenues decreased by $12.8 million (65.2%) due to the absence of contract manufacturing and collaboration revenues following the Lexington Transaction[157][159][161] - License revenues increased by $3.8 million (122.4%) to $6.8 million, driven by higher royalty payments on HEMGENIX® sales[158] Cost of contract manufacturing (6 months) This section details the costs associated with contract manufacturing activities - Cost of contract manufacturing was nil for the six months ended June 30, 2025, compared to $16.3 million in the prior year, due to the Lexington Transaction[162] R&D expense (6 months) This section analyzes the company's research and development expenses and their drivers R&D Expenses (in thousands) | Item | Six months ended June 30, 2025 | Six months ended June 30, 2024 | Change | | :--------------------------------------- | :----------------------------- | :----------------------------- | :----- | | Huntington's disease (AMT-130) | $18,747 | $5,320 | $13,427 | | Employee and contractor-related expenses | $19,682 | $29,958 | $(10,276) | | Fair value changes related to contingent consideration | $3,470 | $(1,621) | $5,091 | | Total research and development expenses | $71,523 | $74,347 | $(2,824) | - Total R&D expenses decreased by $2.8 million (3.8%) to $71.5 million, primarily due to a $10.3 million reduction in personnel and contractor-related expenses following the Lexington Transaction and restructuring[163][171] - Direct R&D expenses for AMT-130 increased by $13.4 million, driven by BLA submission preparation activities[166] - A $3.5 million loss was recognized from an increase in the fair value of contingent consideration for uniQure France SAS, compared to a $1.6 million gain in the prior year[171] Selling, general and administrative expenses (6 months) This section details the company's selling, general, and administrative expenses and their changes SG&A Expenses (in thousands) | Item | Six months ended June 30, 2025 | Six months ended June 30, 2024 | Change | | :--------------------------------------- | :----------------------------- | :----------------------------- | :----- | | Employee and contractor-related expenses | $10,882 | $11,888 | $(1,006) | | Professional fees | $5,331 | $6,094 | $(763) | | Share-based compensation expense | $4,273 | $6,755 | $(2,482) | | Total selling, general and administrative expenses | $24,408 | $29,704 | $(5,296) | - SG&A expenses decreased by $5.3 million (17.8%) to $24.4 million, primarily due to reductions in personnel, contractor-related expenses, and share-based compensation following the Lexington Transaction and restructuring[172][176] Other items, net (6 months) This section analyzes other income and expenses not directly related to core operations Other Items, Net (in thousands) | Item | Six months ended June 30, 2025 | Six months ended June 30, 2024 | Change | | :--------------------------------------- | :----------------------------- | :----------------------------- | :----- | | Sale of critical reagents to Genezen | $6,000 | $0 | $6,000 | | Research and development grants | $3,111 | $2,886 | $225 | | Supply of HEMGENIX® to CSL Behring | $(3,256) | $0 | $(3,256) | | Total other income, net | $6,759 | $2,889 | $3,870 | - Net other items increased by $3.9 million, primarily due to a one-time $6.0 million income from the sale of critical reagents to Genezen, partially offset by $3.3 million in net other expenses related to HEMGENIX® supply[174][175] Other non-operating items, net (6 months) This section details non-operating income and expenses, including interest and foreign currency effects Other Non-Operating Items, Net (in thousands) | Item | Six months ended June 30, 2025 | Six months ended June 30, 2024 | Change | | :--------------------------------------- | :----------------------------- | :----------------------------- | :----- | | Interest income | $7,651 | $12,313 | $(4,662) | | Interest expense - Royalty Financing Agreement | $(27,079) | $(24,839) | $(2,240) | | Interest expense - Hercules debt facility | $(3,621) | $(7,415) | $3,794 | | Foreign currency gains / (losses), net | $25,810 | $(2,134) | $27,944 | | Total non-operating expense, net | $2,761 | $(22,075) | $24,836 | - Net non-operating income significantly improved by $24.8 million, primarily driven by a $27.9 million increase in net foreign currency gains[178][181] - Interest income decreased by $4.7 million due to lower cash and investment securities balances, while Hercules debt interest expense decreased by $3.8 million due to principal repayment and lower market rates[178][179] Income tax expense (6 months) This section details the company's income tax expense for the reporting period - Income tax expense decreased to $0.9 million for the six months ended June 30, 2025, from $1.6 million in the prior year[182] Financial Position, Liquidity and Capital Resources This section discusses the company's financial health, cash management, and funding capabilities - As of June 30, 2025, the Company had $378.5 million in cash, cash equivalents, restricted cash, and investment securities[183] - Existing cash resources are expected to fund operations into the second half of 2027, but additional funding will be required for AMT-130 commercialization and other late-stage clinical development[183] - The Company incurred a net loss of $81.4 million for the six months ended June 30, 2025, and had an accumulated deficit of $1,211.3 million[191] Debt This section details the company's outstanding debt obligations and repayment schedules - As of June 30, 2025, the Company had $50.0 million in outstanding principal under the Hercules debt facility, with $12.9 million in future interest and financing fees, $8.6 million of which is payable within 12 months[184] - The $50.0 million principal balance is due in full by January 2027[184] Leases This section outlines the company's lease payment obligations and guarantees - Fixed lease payment obligations totaled $23.2 million as of June 30, 2025, with $4.4 million payable within 12 months[185] - The Company guarantees $18.5 million in fixed lease payments for the Lexington Facility until May 2029, despite assigning the lease to Genezen[185] Commitments related to uniQure France SAS acquisition (nominal amounts) This section details potential milestone payments related to the uniQure France SAS acquisition - Remaining commitments include EUR 160.0 million ($187.9 million) in potential milestone payments for AMT-260's Phase III development and approval, expected between 2029 and 2033[186] - Up to 25% of these milestone payments can be settled with ordinary shares[186] Commitments related to licensors and financial advisors This section outlines future payment obligations to licensors and financial advisors - The Company has obligations for future payments to third parties upon achievement of development, regulatory, and commercial milestones, and to a financial advisor related to CSL Behring Agreement payments[187] Commitments related to the CSA and the DMSA with Genezen This section details minimum purchase commitments under agreements with Genezen - Remaining minimum purchase commitments to Genezen under the CSA for HEMGENIX® manufacturing services total $25.2 million, with $13.6 million payable within 12 months[188] - Minimum purchase commitments under the DMSA for development services total $9.8 million, with $3.8 million payable within 12 months[189] Sources of liquidity This section describes the primary sources of funding for the company's operations - Operations have been funded primarily through equity and convertible debt sales, venture loans, collaboration upfront payments, and $370.1 million from royalty monetization in 2023[192] - In January and February 2025, the Company raised $80.5 million in net proceeds from a follow-on public offering[193] - The Hercules debt facility was amended in July 2024, with a $50.0 million principal repayment, leaving $50.0 million outstanding due in January 2027[194] Net cash used in operating activities This section analyzes cash flows from the company's core business operations - Net cash used in operating activities was $84.0 million for the six months ended June 30, 2025, an improvement from $93.3 million in 2024[197][198] - Adjustments to net loss included $8.2 million in depreciation and amortization, $9.5 million in share-based compensation, and $20.6 million in royalty financing agreement interest expense (net of paid interest)[197] Net cash generated from investing activities This section details cash flows related to the company's investment activities Cash Flows from Investing Activities (in thousands) | Item | Six months ended June 30, 2025 | Six months ended June 30, 2024 | | :-------------------------------- | :----------------------------- | :----------------------------- | | Proceeds from maturity of debt securities | $213,763 | $297,806 | | Investment in debt securities | $(120,205) | $(152,936) | | Net cash generated from investing activities | $93,172 | $141,922 | - Net cash generated from investing activities decreased to $93.2 million for the six months ended June 30, 2025, from $141.9 million in 2024[199][200] Net cash generated from financing activities This section analyzes cash flows from the company's financing activities, including equity offerings Cash Flows from Financing Activities (in thousands) | Item | Six months ended June 30, 2025 | Six months ended June 30, 2024 | | :-------------------------------- | :----------------------------- | :----------------------------- | | Proceeds from public offering of shares | $80,511 | $0 | | Proceeds from issuance of ordinary shares related to employee plans | $203 | $50 | | Net cash generated from financing activities | $80,714 | $50 | - Net cash generated from financing activities significantly increased to $80.7 million for the six months ended June 30, 2025, from $0.1 million in 2024, primarily due to the follow-on public offering[201][202] Funding requirements This section discusses the company's future capital needs and factors influencing them - Future capital requirements depend on factors such as AMT-130 commercialization, clinical trial progress, regulatory approvals, and intellectual property maintenance[206] - Additional funding will be needed to support AMT-130 commercialization and advance other clinical product candidates into late-stage development[183] Item 3. Quantitative and Qualitative Disclosures About Market Risk This section states that the Company's market risks and exposures, including currency, price, interest rate, credit, and liquidity risks, have not materially changed during the six months ended June 30, 2025, compared to the disclosures in its most recent Annual Report - The Company's market risks and exposures, including currency, price, and interest rate risk, credit risk, and liquidity risk, have not materially changed during the six months ended June 30, 2025[203][204] Item 4. Controls and Procedures This section details the evaluation of the Company's disclosure controls and procedures and reports on any changes in internal control over financial reporting. Management concluded that disclosure controls and procedures were effective as of June 30, 2025, with no material changes to internal control over financial reporting during the period Evaluation of Disclosure Controls and Procedures This section reports on the effectiveness of the company's disclosure controls and procedures - As of June 30, 2025, the CEO and CFO concluded that the Company's disclosure controls and procedures were effective[205] - Disclosure controls are designed to ensure timely recording, processing, summarizing, and reporting of information required under the Exchange Act[205] Changes in Internal Control over Financial Reporting This section reports on any material changes to the company's internal control over financial reporting - No changes in internal control over financial reporting that materially affected, or are reasonably likely to materially affect, the Company's internal control over financial reporting during the period[208] PART II – OTHER INFORMATION Item 1. Legal Proceedings This section states that there are no legal proceedings to report - No legal proceedings were reported for the period[210] Item 1A. Risk Factors This section outlines significant risks associated with an investment in uniQure N.V.'s ordinary shares, covering business operations, product development, regulatory approvals, commercialization, manufacturing, intellectual property, financial position, legal compliance, and market volatility. Key risks include dependence on AMT-130, need for additional funding, potential clinical trial delays, and intense competition in gene therapy Summary Risk Factors This section provides a concise overview of the most critical risks facing the company - The Company is highly dependent on the success of its lead product candidate, AMT-130, for Huntington's disease, with potential failures in clinical development, regulatory approval, manufacturing, or commercialization posing significant risks[213][215] - Additional funding will be required to advance product candidates and support the commercial launch of AMT-130, which may not be available on acceptable terms[213] - Risks include potential delays in clinical trials, failure to demonstrate safety and efficacy, challenges in commercialization, market acceptance issues, and negative public opinion regarding gene therapy[213] Risks Related to Our Business and the Development of Our Product Candidates This section details risks associated with the company's core business and product development pipeline - The Company's business is highly dependent on the success of AMT-130 for Huntington's disease, with risks including potential patient safety issues, insufficient clinical efficacy, and challenges in regulatory alignment or BLA submission[215][219] - Drug development is expensive and uncertain, with significant risks of failure or delay in clinical trials for all product candidates, including AMT-130, AMT-260, AMT-191, and AMT-162[223] - Interim or preliminary clinical trial results, especially from open-label or post-hoc analyses, may not be predictive of long-term efficacy or final outcomes and are subject to regulatory audit and verification[237][238][239] Risks Related to Regulatory Approval of Our Products This section outlines risks concerning the regulatory approval process for the company's product candidates - Obtaining marketing approval for product candidates is expensive, time-consuming, and uncertain, with potential delays or rejections due to changing regulatory policies, staffing limitations, or insufficient data[265][266] - The regulatory approval process is heightened for gene therapies due to limited FDA experience, ethical concerns, and evolving guidance, potentially leading to increased costs and delays[270][271][274] - Leveraging specialized regulatory pathways (e.g., Breakthrough Therapy, RMAT, Accelerated Approval) does not guarantee faster development or approval, and failure to meet post-marketing requirements could lead to withdrawal of approval[280][282] Risks Related to Commercialization This section details risks associated with the market acceptance and commercial success of approved products - Successful commercialization of approved product candidates depends on market acceptance by physicians, patients, and third-party payers, which is uncertain due to factors like efficacy, cost-effectiveness, ease of administration, and potential side effects[311][314] - The addressable market for orphan indications may be smaller than projected, impacted by factors like neutralizing antibodies, diagnosis criteria, and patient access, which could adversely affect product revenues[316][318][319] - The potential for long-term therapeutic benefit from a single administration of gene therapies presents challenges for pricing and reimbursement, as payers may be reluctant to provide significant upfront reimbursement[301] Risks Related to Manufacturing and Our Dependence on Third Parties This section outlines risks related to product manufacturing and reliance on external partners - The Lexington Transaction may not yield expected benefits and could expose the Company to additional costs and risks, including Genezen's ability to manufacture HEMGENIX® and other product candidates according to regulatory and supply requirements[322][324] - Gene therapies are complex and expensive to manufacture, and third-party manufacturers (like Genezen) may face capacity, production, or technology transfer challenges, leading to delays or supply failures[326] - Reliance on third parties for manufacturing and clinical trials reduces control over these activities, and their failure to perform satisfactorily or comply with regulations could lead to delays, regulatory enforcement, or inability to obtain approvals[331][341][342] Risks Related to Our Intellectual Property This section details risks concerning the company's intellectual property rights and protection - The Company relies heavily on in-licensed intellectual property, which may not provide adequate rights, be subject to multiple interpretations, or be unavailable on commercially reasonable terms in the future[350][351][353] - Failure to obtain and maintain broad patent protection for technology and products, or successful challenges to existing patents, could impair the ability to commercialize products and prevent competitors from using similar technologies[355][356][358] - Involvement in intellectual property lawsuits, whether to protect patents or defend against infringement claims, can be expensive, time-consuming, and divert management resources, potentially leading to significant liabilities or loss of rights[359][361][362] Risks Related to Our Financial Position and Need for Additional Capital This section outlines financial risks, including historical losses and the need for future funding - The Company has incurred significant net losses and accumulated deficits, expecting future losses as it funds late-stage development, commercialization preparations for AMT-130, and other product candidates[371][375] - Additional substantial funding will be required beyond existing cash resources, and failure to obtain capital on acceptable terms could force delays or termination of development and commercialization efforts[373][378] - Strategic initiatives, including divestitures like the Lexington Transaction, may not achieve intended benefits, could result in unanticipated problems, expenses, or liabilities, and may divert management attention[379][381] Risks Related to Other Legal Compliance Matters This section details risks associated with legal and regulatory compliance, including healthcare and data protection laws - Relationships with employees, customers, and third parties are subject to anti-bribery, fraud and abuse, and other healthcare laws, with non-compliance potentially leading to significant penalties, damages, or operational restructuring[389][390] - The Company is subject to complex data protection laws like GDPR, with non-compliance risking substantial penalties, regulatory enforcement actions, and private litigation[393][395][396] - Product liability lawsuits related to clinical trials or approved products could result in substantial liabilities, decreased demand, reputational damage, and diversion of resources[397] Risks Related to Our Ordinary Shares This section outlines risks concerning the company's stock performance and shareholder matters - The market price of the Company's ordinary shares has been and may remain volatile, influenced by clinical trial results, regulatory developments, competition, and general market conditions[407][408] - Directors, executive officers, and major shareholders collectively own approximately 33.8% of outstanding shares, potentially influencing matters submitted for shareholder approval[411] - The Company does not expect to pay dividends in the foreseeable future, meaning returns on investment will depend solely on future share price appreciation[419][420] General Risks This section covers broader risks affecting the company, including economic conditions and operational controls - Future success depends on the ability to attract, retain, and motivate key executives, technical staff, and other qualified employees in a highly competitive pharmaceutical field[430][431] - Unstable market and economic conditions, including inflation, higher interest rates, and geopolitical issues, may adversely affect the business, financial condition, and share price[433] - **Failure