uniQure(QURE) - 2025 Q1 - Quarterly Report

Corporate Information This section provides essential company details, including filing status, stock listing, and a cautionary notice on forward-looking statements Filing Details uniQure N.V. filed its Quarterly Report on Form 10-Q for the period ended March 31, 2025. The company is an accelerated filer with 54,748,496 ordinary shares outstanding as of May 6, 2025, and its shares trade on the Nasdaq Global Select Market under the symbol 'QURE' - Registrant: uniQure N.V., incorporated in The Netherlands, with headquarters in Amsterdam. Its ordinary shares trade on the Nasdaq Global Select Market under the symbol 'QURE'[2][23] Filing and Company Status | Metric | Value | |:---|:---| | Filing Type | Quarterly Report on Form 10-Q | | Period Ended | March 31, 2025 | | Filer Status | Accelerated filer | | Ordinary Shares Outstanding (as of May 6, 2025) | 54,748,496 | Special Cautionary Notice Regarding Forward-Looking Statements This section provides a cautionary notice regarding forward-looking statements in the report, highlighting that such statements are based on current expectations and involve risks and uncertainties, and actual results may differ materially. Investors are advised not to place undue reliance on these statements and to consider the risk factors discussed in the report - Forward-looking statements are based on current expectations and involve risks and uncertainties, with actual results potentially differing materially from projections[8][9] - Key areas covered by forward-looking statements include funding, financial position, R&D activities, regulatory approvals, product candidate benefits, strategies, collaborations, intellectual property, commercialization, and liquidity[8] - The company claims protection of the safe harbor for forward-looking statements contained in the Private Securities Litigation Reform Act of 1995[11] Part I – Financial Information This part presents unaudited consolidated financial statements, management's discussion and analysis, market risk disclosures, and controls and procedures Item 1. Financial Statements This section presents the unaudited consolidated financial statements for uniQure N.V. for the three months ended March 31, 2025, and comparative periods, including the balance sheets, statements of operations and comprehensive loss, statements of shareholders' equity, and statements of cash flows, along with detailed notes on significant accounting policies, investments, debt, and other financial items Unaudited Consolidated Balance Sheets This subsection presents the company's financial position, detailing assets, liabilities, and equity for the reporting periods Consolidated Balance Sheet Highlights (in thousands) | Metric | March 31, 2025 | December 31, 2024 | Change | |:---|:---|:---|:---|\ | Assets | | | | | Cash and cash equivalents | $217,229 | $158,930 | +$58,299 | | Current investment securities | $191,782 | $208,591 | -$16,809 | | Total current assets | $435,421 | $390,289 | +$45,132 | | Total assets | $605,403 | $556,536 | +$48,867 | | Liabilities & Equity | | | | | Total current liabilities | $36,310 | $40,053 | -$3,743 | | Liability from royalty financing agreement | $446,660 | $434,930 | +$11,730 | | Total liabilities | $571,717 | $563,288 | +$8,429 | | Total shareholders' equity / (deficit) | $33,686 | $(6,752) | +$40,438 | Unaudited Consolidated Statements of Operations and Comprehensive Loss This subsection outlines the company's financial performance, including revenues, expenses, and net loss for the reported periods Consolidated Statements of Operations Highlights (in thousands) | Metric | Three months ended March 31, 2025 | Three months ended March 31, 2024 | Change (YoY) | |:---|:---|:---|:---|\ | Total revenues | $1,567 | $8,485 | -$6,918 | | Research and development expenses | $(36,140) | $(40,692) | +$4,552 | | Selling, general and administrative expenses | $(10,908) | $(13,937) | +$3,029 | | Loss from operations | $(39,331) | $(54,228) | +$14,897 | | Net loss | $(43,637) | $(65,618) | +$21,981 | | Basic and diluted net loss per ordinary share | $(0.82) | $(1.36) | +$0.54 | | Weighted average shares - basic and diluted | 53,110,580 | 48,384,510 | +4,726,070 | Unaudited Consolidated Statements of Shareholders' Equity This subsection details changes in shareholders' equity, including net loss, public offerings, and share-based compensation Shareholders' Equity Movement (in thousands) | Metric | Balance at Dec 31, 2024 | Loss for the period | Other comprehensive loss, net | Follow-on public offering | Exercises of share options | Restricted and performance share units distributed | Share-based compensation expense | Balance at Mar 31, 2025 | |:---|:---|:---|:---|:---|:---|:---|:---|:---|\ | Ordinary shares (Amount) | $2,945 | — | — | $261 | $1 | $33 | — | $3,240 | | Additional paid-in-capital | $1,173,068 | — | — | $80,250 | $158 | $(33) | $4,410 | $1,257,853 | | Accumulated deficit | $(1,129,965) | $(43,637) | — | — | — | — | — | $(1,173,602) | | Total shareholders' equity / (deficit) | $(6,752) | $(43,637) | $(1,005) | $80,511 | $159 | — | $4,410 | $33,686 | Unaudited Consolidated Statements of Cash Flows This subsection presents cash inflows and outflows from operating, investing, and financing activities for the reported periods Consolidated Cash Flow Highlights (in thousands) | Cash Flow Activity | Three months ended March 31, 2025 | Three months ended March 31, 2024 | Change (YoY) | |:---|:---|:---|:---|\ | Net cash used in operating activities | $(44,100) | $(60,575) | +$16,475 | | Net cash generated from investing activities | $20,099 | $63,985 | -$43,886 | | Net cash generated from financing activities | $80,670 | $0 | +$80,670 | | Net increase in cash, cash equivalents and restricted cash | $58,329 | $1,685 | +$56,644 | | Cash, cash equivalents and restricted cash at the end of period | $218,658 | $246,229 | -$27,571 | Notes to Consolidated Financial Statements This subsection provides detailed explanations and disclosures supporting the consolidated financial statements 1. General Business Information This note provides background on uniQure N.V., its incorporation, gene therapy focus, and Nasdaq listing - uniQure N.V. was incorporated on January 9, 2012, in the Netherlands, focusing on gene therapy for rare and devastating diseases. It converted to a public company in 2014 and its shares are listed on Nasdaq under 'QURE'[22][23] 2. Summary of Significant Accounting Policies This note outlines key accounting principles and methods used in preparing financial statements - The financial statements are prepared in compliance with U.S. GAAP and SEC interim financial reporting rules, presented in U.S. dollars, and rely on management estimates and assumptions[24][25][26][28] - No material changes to significant accounting policies or new accounting pronouncements materially impacted the consolidated financial statements during the three months ended March 31, 2025[29][30] 3. Other Investments This note details the company's other investments, including a convertible promissory note and equity securities Other Investments (in millions) | Investment Type | March 31, 2025 | December 31, 2024 | |:---|:---|:---|\ | Convertible promissory note | $13.9 | $13.7 | | Non-publicly traded equity securities | $14.4 | $14.4 | - The convertible promissory note bears 8.0% interest per annum, with $0.2 million interest income recognized in Q1 2025[31] 4. Investment Securities This note describes the company's investment in short-term government debt securities Government Debt Securities (Held-to-Maturity, in thousands) | Metric | March 31, 2025 | December 31, 2024 | |:---|:---|:---|\ | Amortized cost | $191,782 | $208,591 | | Estimated fair value | $191,791 | $208,755 | - Investments are in short-term U.S. and European government debt securities with the highest credit rating, measured at amortized cost for maturities 90 days or less[33][34] 5. Fair Value Measurement This note explains methodologies and inputs used to measure financial assets and liabilities at fair value - The company measures certain financial assets and liabilities at fair value, categorizing inputs into Level 1 (quoted prices in active markets), Level 2 (observable inputs for similar assets/liabilities), and Level 3 (unobservable inputs requiring significant judgment)[36][37][38] Fair Value of Contingent Consideration (in thousands) | Metric | March 31, 2025 | December 31, 2024 | |:---|:---|:---|\ | Contingent consideration | $12,560 | $10,860 | | Discount rates | 14.8% to 15.5% | 15.3% to 16.2% | - Contingent consideration relates to the acquisition of uniQure France SAS, with potential payments up to EUR 143.1 million ($154.8 million) upon milestone achievement. A 100% likelihood of AMT-260 Phase III advancement would increase fair value to $40.5 million[42][44] 6. Accrued Expenses and Other Current Liabilities This note provides a breakdown of accrued expenses and other current liabilities Accrued Expenses and Other Current Liabilities (in thousands) | Category | March 31, 2025 | December 31, 2024 | |:---|:---|:---|\ | Personnel related accruals and liabilities | $10,921 | $12,583 | | Accruals for goods/services not yet billed | $11,594 | $10,109 | | Current portion of firm purchase commitment liability | $3,249 | $1,582 | | Liability owed to Purchaser (Royalty Financing Agreement) | $1,580 | $4,951 | | Total | $27,344 | $29,225 | 7. Long-term Debt This note details the company's venture debt loan facility, including principal, maturity, interest, and covenants - The company has a venture debt loan facility with Hercules Capital, Inc., amended in July 2024, with $50.0 million principal outstanding as of March 31, 2025, due January 5, 2027[49][50][51] Long-term Debt Details | Metric | Details | |:---|:---|\ | Principal outstanding (Mar 31, 2025) | $50.0 million | | Maturity Date | January 5, 2027 | | Interest Rate | Greater of (i) 7.95% and (ii) 7.95% + prime rate - 3.25% | | Back-end fees | $2.4 million (Dec 1, 2025), $0.6 million (Maturity Date) | | Interest expense (Q1 2025) | $1.8 million (vs $3.7 million in Q1 2024) | | Foreign currency gain (Q1 2025) | $2.1 million (vs $2.3 million loss in Q1 2024) | - Covenants include minimum cash balances and restrictions on future indebtedness, investments, asset transfers, and dividend payments. The company was in material compliance as of March 31, 2025[54][55] 8. Royalty Financing Agreement This note describes the royalty financing agreement, outlining upfront payment and sales-based obligations - In May 2023, uniQure entered a Royalty Financing Agreement, receiving $375.0 million upfront for rights to the lowest royalty tier on HEMGENIX® sales, with a potential obligation to pay $25.0 million of the first worldwide sales milestone[56] - The Purchaser will receive 1.85 times the upfront payment ($693.8 million) by June 30, 2032, or up to 2.25 times by December 31, 2038. The company expects to satisfy its commitment before the Second Hard Cap Date[57][60] Royalty Financing Agreement Liability Movement (in thousands) | Metric | Amount | |:---|:---|\ | Balance as of Dec 31, 2024 | $439,881 | | Royalty payments to the Purchaser | $(4,951) | | Interest expense for the period | $13,309 | | Balance as of Mar 31, 2025 | $448,239 | 9. Shareholders' Equity This note details changes in shareholders' equity, including proceeds from a public offering - In January 2025, the company completed a follow-on public offering of 4.4 million ordinary shares at $17.00 per share, generating $70.1 million in net proceeds. An additional $10.6 million was raised in February 2025 from underwriters' option exercise[63][64] 10. Share-based Compensation This note provides a breakdown of share-based compensation expense by classification and award type Share-based Compensation Expense by Classification (in thousands) | Classification | Three months ended March 31, 2025 | Three months ended March 31, 2024 | |:---|:---|:---|\ | Research and development | $2,463 | $3,425 | | Selling, general and administrative | $1,947 | $3,421 | | Total | $4,410 | $7,191 | Share-based Compensation Expense by Award Type (in thousands) | Award Type | Three months ended March 31, 2025 | Three months ended March 31, 2024 | |:---|:---|:---|\ | Share options | $1,637 | $2,913 | | Restricted share units | $2,213 | $4,370 | | Performance share units | $560 | $(99) | | Employee share purchase plan | — | $7 | | Total | $4,410 | $7,191 | - Unrecognized share-based compensation expense totaled $33.3 million as of March 31, 2025, with a weighted-average remaining recognition period of 2.36 years[69] 11. Segment Reporting This note clarifies the company operates as a single segment focused on gene therapy R&D - The company operates as one segment, focusing on gene therapy R&D, with the Chief Operating Decision Maker (CODM) allocating resources based on scientific data and risk-adjusted returns[77][78] Segment Operating Loss (in thousands) | Metric | Three months ended March 31, 2025 | Three months ended March 31, 2024 | |:---|:---|:---|\ | Revenue | $1,567 | $8,485 | | Employee related expenses | $(14,216) | $(24,383) | | Laboratory and development expenses | $(15,967) | $(9,076) | | Segment operating loss | $(34,310) | $(43,020) | 12. CSL Behring Collaboration This note describes the CSL Behring collaboration for HEMGENIX® and the company's post-transaction sales role - uniQure biopharma B.V. granted CSL Behring exclusive global rights to HEMGENIX® in June 2020. Following the Lexington Transaction, uniQure acts as an agent in HEMGENIX® sales to CSL Behring, recognizing costs net of income in Other expense[80][81] Accounts Receivable from Collaboration (in thousands) | Metric | March 31, 2025 | December 31, 2024 | |:---|:---|:---|\ | Accounts receivable | $2,522 | $5,657 | 13. Other Income This note details components of other income, including reagent sales, R&D grants, and sublease income Other Income (in thousands) | Category | Three months ended March 31, 2025 | Three months ended March 31, 2024 | |:---|:---|:---|\ | Sale of critical reagents to Genezen | $6,000 | — | | Research and development grants from Dutch authorities | $1,719 | $1,224 | | Sublease income | $458 | $152 | | Total | $8,306 | $1,376 | - The significant increase in other income was primarily due to a one-time $6.0 million sale of critical reagents to Genezen in Q1 2025[83] 14. Income Taxes This note explains the company's income tax expense and its effective tax rate Income Tax Expense (in thousands) | Metric | Three months ended March 31, 2025 | Three months ended March 31, 2024 | |:---|:---|:---|\ | Income tax expense | $(496) | $(656) | - The effective income tax rate of 1.1% in Q1 2025 (vs 1.0% in Q1 2024) is substantially lower than the Netherlands' enacted rate of 25.8% due to valuation allowances against net deferred tax assets in the Netherlands and France[85] 15. Basic and Diluted Earnings Per Share This note clarifies the computation of basic and diluted loss per share, noting anti-dilutive effects - Due to a net loss, all potentially dilutive ordinary shares (totaling 7,526,322 in Q1 2025) were anti-dilutive and excluded from the computation of loss per share[86][87] 16. Subsequent Events This note confirms no subsequent events requiring disclosure were reported - No subsequent events were reported[88] Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations This section provides management's perspective on the company's financial condition and results of operations for the three months ended March 31, 2025, discussing key business developments, financial performance, liquidity, and capital resources, including details on revenue, expenses, and cash flows Overview This subsection summarizes uniQure N.V.'s gene therapy focus and key clinical candidates - 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), SOD1-ALS (AMT-162), refractory mTLE (AMT-260), and Fabry disease (AMT-191)[90] Business Developments This subsection highlights recent strategic and clinical advancements in gene therapy programs Financing This note details the company's recent capital raising activities, including a public offering - In January and February 2025, uniQure completed a follow-on public offering, raising a total of $80.7 million in net proceeds from the sale of 5.1 million ordinary shares[91] Huntington's Disease Program (AMT-130) This note outlines regulatory designations and clinical trial progress for AMT-130 - AMT-130 received FDA Breakthrough Therapy designation in April 2025, supported by Phase I/II data showing potential for slowing disease progression in Huntington's disease[92] - Previously, AMT-130 was granted Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug, and Fast Track designations by the FDA[93] Regulatory Alignment This note describes FDA discussions on CMC requirements and accelerated approval pathways for AMT-130 - In March 2025, uniQure held a Type B meeting with the FDA to discuss CMC requirements for AMT-130's Biologics License Application (BLA) submission[94] - In December 2024, FDA agreed that Phase I/II data, compared to natural history control, could serve as the primary basis for an accelerated approval BLA for AMT-130, using cUHDRS as an intermediate clinical endpoint and NfL reductions in CSF as supportive evidence[95] Recent Developments of Other Product Candidates This note provides updates on clinical progress and regulatory designations for other gene therapy candidates Temporal Lobe Epilepsy Program (AMT-260) This note details clinical trial progress for AMT-260 in refractory mTLE - The Phase I/IIa GenTLE clinical trial for AMT-260 in refractory mTLE dosed its first patient in November 2024. The FDA approved a protocol amendment in February 2025 to expand inclusion criteria for certain patients[96][97] Fabry Disease Program (AMT-191) This note outlines clinical trial progress and regulatory designations for AMT-191 - AMT-191's Phase I/IIa clinical trial for Fabry disease received a favorable safety recommendation from the IDMC in February 2025, allowing enrollment in the second cohort. The first patient was dosed in August 2024, and it received FDA Orphan Drug and Fast Track Designations in September and October 2024, respectively[98][99][100] Amyotrophic Lateral Sclerosis (AMT-162) This note describes clinical trial progress for AMT-162 in SOD1-ALS - The EPISOD1 Phase I/II trial for AMT-162 in SOD1-ALS dosed its first patient in October 2024. The IDMC recommended proceeding to the second cohort in January 2025, and enrollment for the third cohort is expected in Q3 2025[101][102] Financial Overview This subsection summarizes key financial metrics, including revenues, expenses, net loss, and cash position Financial Overview (in thousands) | Metric | Three months ended March 31, 2025 | Three months ended March 31, 2024 | |:---|:---|:---|\ | Total revenues | $1,567 | $8,485 | | Research and development expenses | $(36,140) | $(40,692) | | Selling, general and administrative expenses | $(10,908) | $(13,937) | | Net loss | $(43,637) | $(65,618) | | Cash, cash equivalents and investment securities | $409,000 (as of Mar 31, 2025) | $367,500 (as of Dec 31, 2024) | | Accumulated deficits | $1,173,600 (as of Mar 31, 2025) | $1,130,000 (as of Dec 31, 2024) | Critical Accounting Policies and Estimates This subsection discusses significant accounting policies and estimates requiring management judgment - The company's financial statements rely on management's assumptions, judgments, and estimates. No material changes to critical accounting policies occurred during Q1 2025[105] Contract Manufacturing This subsection explains changes in contract manufacturing revenue recognition post-Lexington Transaction - Prior to the July 2024 Lexington Transaction, uniQure recognized contract manufacturing revenues and costs for HEMGENIX® for CSL Behring. Post-transaction, these activities ceased for uniQure[106] Research and Development Expenses This subsection describes the nature of R&D expenses and factors influencing their variability - R&D expenses are expensed as incurred, covering employee costs, preclinical/clinical studies, manufacturing process development, technology platforms, collaboration services, and milestone payments to licensors[107][108] - R&D expenses are highly uncertain and can vary significantly based on timing of activities, clinical trial progress, regulatory submissions, and patient enrollment[109] Selling, General and Administrative Expenses This subsection outlines primary components of SG&A expenses, including personnel and professional fees - SG&A expenses primarily include employee, office, consulting, legal, and other professional and administrative costs, as well as expenses associated with operating as a public company and maintaining intellectual property[110] Other Items, Net This subsection details components of other income and expense, including R&D subsidies and supply agreement costs - Other income includes R&D subsidies, sublease income, and a one-time sale of critical reagents in Q1 2025. Other expense primarily consists of costs under the commercial supply agreement with Genezen post-Lexington Transaction[111][112] Results of Operations This subsection provides a detailed analysis of the company's financial performance Revenue This note analyzes changes in total revenue, highlighting the Lexington Transaction's impact Total Revenues (in thousands) | Revenue Type | Three months ended March 31, 2025 | Three months ended March 31, 2024 | Change (YoY) | |:---|:---|:---|:---|\ | License revenues | $1,567 | $1,202 | +$365 | | Contract manufacturing revenues | — | $3,990 | -$3,990 | | Collaboration revenues | — | $3,293 | -$3,293 | | Total revenues | $1,567 | $8,485 | -$6,918 | - The decrease in total revenue was primarily due to the cessation of contract manufacturing and collaboration services for CSL Behring following the Lexington Transaction in July 2024[116][117][118] Cost of Contract Manufacturing This note explains the absence of contract manufacturing costs due to divestment of activities - No contract manufacturing costs were incurred in Q1 2025, a decrease of $9.1 million from Q1 2024, due to the divestment of commercial manufacturing activities in July 2024[119] R&D Expense This note details changes in R&D expenses, attributing variations to employee costs and clinical trial activities Research and Development Expenses (in thousands) | Category | Three months ended March 31, 2025 | Three months ended March 31, 2024 | Change (YoY) | |:---|:---|:---|:---|\ | Total direct R&D expenses | $15,603 | $8,476 | +$7,127 | | Total other R&D expenses | $20,537 | $32,216 | -$11,679 | | Total R&D expenses | $36,140 | $40,692 | -$4,552 | - Total R&D expenses decreased by $4.5 million YoY, primarily due to lower employee and contractor-related expenses, facility expenses, and share-based compensation following the Lexington Transaction and restructuring[121][132] - Direct R&D expenses for AMT-130 increased by $5.8 million due to BLA submission preparation activities. Costs for AMT-191 increased due to Phase I/II clinical trial preparation[123][124][125] Selling, General and Administrative Expenses This note analyzes the decrease in SG&A expenses due to personnel reductions and share-based compensation Selling, General and Administrative Expenses (in thousands) | Category | Three months ended March 31, 2025 | Three months ended March 31, 2024 | Change (YoY) | |:---|:---|:---|:---|\ | Employee and contractor-related expenses | $5,485 | $6,219 | -$734 | | Share-based compensation expense | $1,947 | $3,421 | -$1,474 | | Professional fees | $1,903 | $2,041 | -$138 | | Total SG&A expenses | $10,908 | $13,937 | -$3,029 | - SG&A expenses decreased by $3.0 million YoY, mainly due to reductions in personnel, contractor-related expenses, and share-based compensation following the Lexington Transaction and organizational restructuring[137] Other Items, Net This note explains changes in other income and expense, highlighting a one-time reagent sale Other Items, Net (in thousands) | Category | Three months ended March 31, 2025 | Three months ended March 31, 2024 | Change (YoY) | |:---|:---|:---|:---|\ | Sale of critical reagents to Genezen | $6,000 | — | +$6,000 | | Research and development grants from Dutch authorities | $1,719 | $1,224 | +$495 | | Sublease income | $458 | $152 | +$306 | | Supply of HEMGENIX® to CSL Behring | $(1,525) | — | -$1,525 | | Total other income, net | $6,347 | $1,142 | +$5,205 | - The increase in other income was primarily driven by a one-time $6.0 million sale of critical reagents to Genezen[134] Other Non-Operating Items, Net This note details changes in non-operating items, including interest income, expense, and foreign currency Other Non-Operating Items, Net (in thousands) | Category | Three months ended March 31, 2025 | Three months ended March 31, 2024 | Change (YoY) | |:---|:---|:---|:---|\ | Interest income | $4,127 | $6,508 | -$2,381 | | Interest expense - Royalty Financing Agreement | $(13,309) | $(12,401) | -$908 | | Interest expense - Hercules debt facility | $(1,800) | $(3,696) | +$1,896 | | Foreign currency gains / (losses), net | $7,172 | $(1,145) | +$8,317 | | Total non-operating expense, net | $(3,810) | $(10,734) | +$6,924 | - Interest income decreased due to lower interest earned on investment securities and cash. Interest expense for Hercules debt decreased due to a $50.0 million repayment in July 2024 and lower market interest rates[136][139] - A significant net foreign currency gain of $7.2 million was recognized in Q1 2025, compared to a $1.1 million net loss in Q1 2024[141] Income Tax Expense This note explains the company's income tax expense and its effective tax rate Income Tax Expense (in thousands) | Metric | Three months ended March 31, 2025 | Three months ended March 31, 2024 | |:---|:---|:---|\ | Deferred tax expense | $(496) | $(656) | Financial Position, Liquidity and Capital Resources This subsection assesses the company's financial health, liquidity, and ability to fund operations Debt This note details the company's outstanding principal on its Hercules loan and associated fees - As of March 31, 2025, uniQure had $50.0 million outstanding principal on its Hercules loan, with $14.5 million in future interest and financing fees, of which $8.6 million is due within 12 months. The principal is due in January 2027[144] Leases This note outlines the company's fixed lease payment obligations and Lexington Facility guarantees - Fixed lease payment obligations totaled $22.8 million as of March 31, 2025, with $4.2 million payable within 12 months. The company guarantees $19.6 million in lease payments for the Lexington Facility until May 2029, following its assignment to Genezen[145] Commitments Related to uniQure France SAS Acquisition This note describes potential milestone payments related to the uniQure France SAS acquisition - Remaining commitments include EUR 160.0 million ($166.2 million) in potential milestone payments for AMT-260's Phase III development and approval, expected between 2029 and 2033. Up to 25% of these payments can be settled with ordinary shares[146][147] Commitments Related to Licensors and Financial Advisors This note details obligations for future payments to third parties upon achieving milestones - 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 payments collected under the CSL Behring Agreement[148] Commitments Related to the CSA and the DMSA with Genezen This note outlines minimum purchase commitments to Genezen under supply and manufacturing agreements - Minimum purchase commitments to Genezen under the Commercial Supply Agreement (CSA) for HEMGENIX® total $33.1 million (with $17.0 million due within 12 months) and under the Development and Other Manufacturing Services Agreement (DMSA) total $11.6 million (with $4.5 million due within 12 months), both ending July 2027[149][150] Sources of Liquidity This note describes the company's funding sources and projected liquidity runway - Operations are funded through equity offerings, debt financings, collaborations, and royalty monetization. In January/February 2025, $80.7 million net proceeds were raised from a public offering[151][152] - Existing cash, cash equivalents, and investment securities are expected to fund operations through the second half of 2027, but additional funding may be required for late-stage clinical development of other candidates[143][156] Net Cash Used in Operating Activities This note analyzes changes in net cash used in operating activities due to net loss and non-cash adjustments Net Cash Used in Operating Activities (in thousands) | Metric | Three months ended March 31, 2025 | Three months ended March 31, 2024 | |:---|:---|:---|\ | Net cash used | $(44,100) | $(60,575) | | Net loss | $(43,637) | $(65,618) | | Depreciation and amortization | $4,527 | $2,629 | | Share-based compensation expense | $4,410 | $7,191 | | Royalty financing agreement interest expense, net of interest paid | $8,358 | $10,925 | | Unrealized foreign exchange (gains) / losses, net | $(7,105) | $1,389 | - The decrease in net cash used in operating activities by $16.5 million YoY was primarily due to a lower net loss and favorable non-cash adjustments, including unrealized foreign exchange gains[20][157][158] Net Cash Generated from Investing Activities This note details changes in net cash generated from investing activities Net Cash Generated from Investing Activities (in thousands) | Metric | Three months ended March 31, 2025 | Three months ended March 31, 2024 | |:---|:---|:---|\ | Net cash generated | $20,099 | $63,985 | | Proceeds from maturity of debt securities | $85,000 | $150,107 | | Investment in debt securities | $(64,775) | $(83,778) | | Purchases of property, plant, and equipment | $(126) | $(2,344) | - Net cash generated from investing activities decreased by $43.9 million YoY, mainly due to lower proceeds from maturity of debt securities and reduced investment in debt securities[20][159][160] Net Cash Generated from Financing Activities This note explains the significant increase in net cash from financing activities due to a public offering Net Cash Generated from Financing Activities (in thousands) | Metric | Three months ended March 31, 2025 | Three months ended March 31, 2024 | |:---|:---|:---|\ | Net cash generated | $80,670 | — | | Proceeds from public offering of shares | $80,511 | — | | Proceeds from employee stock option/purchase plans | $159 | — | - Net cash generated from financing activities significantly increased to $80.7 million in Q1 2025, primarily from the follow-on public offering of shares[20][161][162] Funding Requirements This note outlines factors influencing future capital requirements, including BLA submission and clinical costs - Future capital requirements depend on factors including BLA submission for AMT-130, commercialization efforts, earnout payments, clinical trial scope and costs, and intellectual property maintenance[163] Item 3. Quantitative and Qualitative Disclosures About Market Risk This section describes the company's exposure to various financial risks, including market risk (currency, price, and interest rate), credit risk, and liquidity risk, noting no material changes from previous disclosures - The company is exposed to market risks including currency, price, and interest rate risk, as well as credit and liquidity risk. No material changes in market risks were noted compared to the Annual Report[166][167] Item 4. Controls and Procedures This section confirms that management, with the CEO and CFO, evaluated the effectiveness of disclosure controls and procedures as of March 31, 2025, concluding they were effective. No material changes in internal control over financial reporting occurred during the period - Management, with CEO and CFO participation, concluded that disclosure controls and procedures were effective as of March 31, 2025. No material changes in internal control over financial reporting occurred during the period[168][169] Part II – Other Information This part provides additional information, including legal proceedings, risk factors, equity sales, and exhibits Item 1. Legal Proceedings This section states that there are no legal proceedings to report - No legal proceedings were reported[171] Item 1A. Risk Factors This section outlines significant risks associated with an investment in uniQure's ordinary shares, covering business and product development, regulatory approval, commercialization, manufacturing, intellectual property, financial position, legal compliance, and general market risks Summary Risk Factors This subsection provides a high-level overview of critical risks, including product development, regulatory, and financial uncertainties - Key risks include dependence on AMT-130 success, potential clinical trial delays/failures, challenges in commercialization, market acceptance issues, regulatory uncertainties (especially for gene therapies), manufacturing complexities, need for additional funding, competition, and volatility of share price[174][179] Risks Related to Our Business and the Development of Our Product Candidates This subsection details risks associated with product candidate success, clinical trial outcomes, and drug development uncertainties - The company's success is highly dependent on AMT-130 for Huntington's disease, which has received Breakthrough Therapy and RMAT designations, but faces risks including safety issues, insufficient efficacy, and regulatory challenges for BLA submission[176][177][178][180][182] - Drug development is expensive and uncertain, with potential for delays or failures in clinical trials due to adverse events, patient recruitment issues, regulatory disagreements, or manufacturing interruptions[183][186][190] - Early-stage clinical trial results may not predict long-term efficacy, and interim data are subject to change and regulatory audit, potentially leading to different final conclusions or regulatory disagreement on statistical analyses[193][197][198][199][200] - Reliance on unvalidated exploratory biomarkers (e.g., NfL in CSF for AMT-130) carries risks of inaccuracy and inefficient resource allocation if the understanding or use of these biomarkers is flawed[205][206] - The company may not successfully expand its product pipeline or secure partnerships, potentially impacting future product revenues and financial position[207][208][209][210][211][212] Risks Related to Regulatory Approval of Our Products This subsection outlines risks concerning marketing approvals, including regulatory policies, specialized pathways, and market exclusivity - Obtaining marketing approval is expensive, time-consuming, and uncertain, with potential for delays or rejections due to evolving regulatory policies, staffing limitations, or insufficient data, especially for novel gene therapies[226][227][228][229][231][232] - Leveraging specialized regulatory pathways (e.g., accelerated approval, RMAT, Breakthrough Therapy) does not guarantee faster development or approval, and benefits may not be maintained if conditions are not met or if regulatory scrutiny increases[233][238][239][240] - Failure to obtain or maintain orphan product exclusivity could limit commercial opportunities, and competitors obtaining exclusivity first could block market entry for significant periods[241][243][244] - Uncertainty exists regarding insurance coverage, pricing, and reimbursement for HEMGENIX® and other product candidates, with government and third-party payers exerting pressure to control costs, potentially impacting commercial viability[249][250][251][254][256] Risks Related to Commercialization This subsection addresses challenges in commercializing products, including market acceptance, pricing, and reimbursement - Successful commercialization depends on obtaining and maintaining marketing approvals, launching sales, market acceptance by physicians and patients, effective competition, optimal pricing, and adequate reimbursement, among other factors[269][270] - Market acceptance of gene therapies is uncertain due to factors like efficacy compared to alternatives, cost-effectiveness, ease of administration, side effects, and patient/physician willingness to adopt new treatments[272][275] - Addressable markets for orphan indications may be smaller than projected due to inaccurate prevalence estimates, neutralizing antibodies, or difficulties in patient identification, adversely affecting product revenues[274][276][277][278] - Ethical, legal, and social issues surrounding genetic testing may reduce demand for gene therapies, and commercializing products outside the U.S. faces additional risks like differing regulatory requirements, intellectual property protection, and economic instability[279][283] Risks Related to Manufacturing and Our Dependence on Third Parties This subsection details risks associated with manufacturing processes, third-party reliance, and supply chain disruptions - The Lexington Transaction (divestiture of manufacturing activities) may not yield expected cost reductions and introduces risks related to Genezen's manufacturing capabilities, supply chain, and contractual obligations, potentially impacting HEMGENIX® supply and other programs[280][281][282] - Gene therapies are complex and expensive to manufacture, with risks of production interruptions, insufficient yield, product deficiencies, and difficulties in scaling up processes, which could delay development or commercialization[284][285][286][287][288] - Reliance on third-party manufacturers (like Genezen) for product candidates subjects the company to their compliance with cGMP and regulatory requirements, with any failure potentially leading to regulatory enforcement, delays, or inability to obtain/maintain approvals[289][290][291][294] - The use of hazardous materials in development and manufacturing exposes the company and its contractors to regulatory compliance and significant potential liabilities from accidents or non-compliance[295] - Reliance on third parties for preclinical and clinical trials reduces control over these activities, with risks of non-performance, delays, non-compliance with regulatory standards (GCPs, GLPs), and potential data unreliability, which could harm development and commercialization[297][299][300][301][302][303] - Collaborations with third parties carry risks including discretion over efforts, limited control over trial design, potential for non-performance, and disagreements over proprietary rights, which could delay or terminate development programs[304][305] Risks Related to Our Intellectual Property This subsection covers risks concerning intellectual property, including licenses, patent protection, and litigation - Reliance on third-party intellectual property licenses means rights may be inadequate, subject to multiple interpretations, or unavailable on reasonable terms, and licensors may fail to maintain patent protection[308][309][310][311] - Failure to comply with license obligations could lead to loss of important rights, impacting development, manufacturing, or marketing of products[312] - Inability to obtain or maintain broad patent protection for technology and products could impair commercialization, as patent prosecution is expensive, uncertain, and subject to challenges, potentially leading to narrowed, invalidated, or unenforceable claims[313][314][315][316] - Involvement in intellectual property lawsuits, either to protect patents or defend against infringement claims, is expensive, time-consuming, and could divert resources, lead to adverse outcomes, or require costly licenses[317][319][320][321][322] - Failure to protect the confidentiality of proprietary information and know-how, including trade secrets shared with collaborators, could adversely affect technology value and competitive position[323][324][325][326] - Intellectual property rights have limitations and may not address all competitive threats, as competitors might develop similar products without infringement or patents may expire before commercialization[327][328] Risks Related to Our Financial Position and Need for Additional Capital This subsection highlights financial risks, including accumulated losses, funding needs, debt, and strategic initiative effectiveness - The company has incurred significant net losses ($43.6 million in Q1 2025, $239.6 million in FY2024) and accumulated deficits ($1,173.6 million as of March 31, 2025), expecting continued losses due to substantial R&D and commercialization costs[329] - Additional funding is required to advance product candidates and support AMT-130's commercial launch. While existing cash is projected to fund operations through the second half of 2027, this estimate is based on assumptions that may prove incorrect, and capital may be exhausted sooner[331][332][334] - Inability to secure additional capital on acceptable terms (due to debt covenants, shareholder approvals, or market conditions) could force delays, reductions, or termination of R&D programs or commercialization efforts[335][336] - Strategic initiatives, including divestitures like the Lexington Transaction, acquisitions, and collaborations, may not achieve intended benefits, could be complex and expensive, divert management attention, and expose the company to operational challenges and additional costs[337][338][339] - Restructuring efforts (e.g., October 2023, August 2024 workforce reduction of ~65%) aim to streamline operations and reduce costs but may not be as effective as anticipated, could incur near-term costs, and disrupt business through employee attrition or morale issues[341][342] - Existing and future indebtedness (e.g., $50.0 million Hercules loan due Jan 2027) could adversely affect operations by dedicating cash to debt service, increasing vulnerability to economic changes, imposing restrictive covenants, and limiting financial flexibility. Variable interest rates could also increase debt service obligations[343][344][345] Risks Related to Other Legal Compliance Matters This subsection addresses risks related to compliance with healthcare laws, data protection, and product liability - Relationships with employees, customers, and third parties are subject to anti-bribery, fraud and abuse, and other healthcare laws and regulations. Non-compliance could lead to significant civil, criminal, and administrative penalties, and operational restructuring[347][348] - Compliance with labor and employment laws is costly, and non-compliance could expose the company to significant liabilities, including claims of discrimination or retaliation[349] - The company is subject to complex data protection laws (e.g., GDPR), with non-compliance potentially leading to substantial penalties (up to EUR 20.0 million or 4% of annual worldwide gross revenue) and private litigation[351][352][353][354] - Product liability lawsuits related to clinical trials or approved products could result in substantial liabilities, decreased demand, reputational damage, and significant costs, potentially exceeding insurance coverage[355][356][357] - Healthcare legislative and regulatory reforms (e.g., PPACA, Inflation Reduction Act of 2022) may increase costs, reduce reimbursement, and impose pricing pressures, materially affecting financial operations and commercialization efforts[358][359][360] General Risks This subsection covers broader risks, including international markets, regulatory changes, share price volatility, and economic conditions - Future growth depends on penetrating international markets, which involves additional regulatory burdens and risks like differing requirements, reduced intellectual property protection, and economic instability[361][283] - Future changes in legal and regulatory requirements, including those from new administrations or Supreme Court decisions, may introduce new risks, require additional resources for compliance, or impact FDA approval standards and marketing strategies[363] - The market price of ordinary shares has been and may remain volatile due to factors like clinical trial results, regulatory developments, competition, financing activities, and general market conditions, potentially leading to securities litigation or shareholder activism[365][366][368] - Directors, executive officers, and major shareholders collectively own ~22.7% of shares, potentially influencing shareholder approvals and management decisions, with interests that may differ from other shareholders[369] - The company's governance is subject to Dutch law, which differs from U.S. law, requiring directors to act in the long-term interest of the company and potentially deterring acquisition bids or making board replacement more difficult[370][371][375][376][377] - The company does not expect to pay dividends in the foreseeable future, meaning returns on investment will depend solely on share price appreciation[378][379] - The company may qualify as a Passive Foreign Investment Company (PFIC) for U.S. federal income tax purposes, which could result in adverse tax consequences for U.S. holders, and the company does not intend to provide information for certain tax elections[382][383] - Enforcing U.S. or other foreign judgments against the company in the Netherlands may be difficult due to differences in legal systems and lack of automatic recognition treaties, potentially limiting recourse for shareholders[384][385][386][387][388] - Failure to comply with all best practice provisions of the Dutch Corporate Governance Code may affect shareholder rights and protection levels compared to companies that fully comply[389] - Future success depends on retaining key executives, technical staff, and other qualified employees, as competition for talent is intense, and loss of key personnel could impede R&D objectives and business strategy[390][391][392] - Unstable market and economic conditions, including inflation, higher interest rates, and geopolitical issues, may adversely affect liquidity, cost structure, financing efforts, and share price[393][394] - Failure to maintain effective internal controls could lead to inaccurate financial reporting, fraud, non-compliance with reporting obligations, and a decline in investor confidence and share price[395] - Internal computer systems or those of third parties are vulnerable to security breaches and cyber-attacks, which could disrupt business operations, lead to loss of proprietary information, and result in liabilities under data privacy laws[396][397] - If securities analysts cease coverage or publish inaccurate/unfavorable research, the share price and trading volume could decline[399] - Failure to achieve projected development and financial goals in announced timeframes could delay product commercialization and lead to a decline in share price[400] - Environmental sustainability and social initiatives may impose additional costs and risks due to new legislative/regulatory requirements, public scrutiny, and potential non-compliance, affecting reputation and financial results[401][402] Item 2. Unregistered Sales of Equity Securities, Use of Proceeds, and Issuer Purchases of Equity Securities This section reports that there were no unregistered sales of equity securities, use of proceeds, or issuer purchases of equity securities during the period - No unregistered sales of equity securities, use of proceeds, or issuer purchases of equity securities were reported[403] Item 3. Defaults Upon Senior Securities This section states that there were no defaults upon senior securities - No defaults upon senior securities were reported[404] Item 4. Mine Safety Disclosures This section indicates that mine safety disclosures are not applicable to the company - Mine safety disclosures are not applicable[405] Item 5. Other Information This section discloses Rule 10b5-1 trading arrangements adopted by the CEO and CFO during the three months ended March 31, 2025, for future sales of ordinary shares Rule 10b5-1 Trading Arrangements Adopted (Q1 2025) | Name & Title | Date Adopted | Aggregate Number of Ordinary Shares to be Purchased or Sold | Expiration Date | |:---|:---|:---|:---|\ | Matthew Kapusta, CEO | January 28, 2025 | 226,316 | June 30, 2026 | | Christian Klemt, CFO | January 13, 2025 | 124,750 | December 31, 2026 | - These arrangements are intended to satisfy the affirmative defense of Rule 10b5-1(c) under the Exchange Act, with sales beginning after the adoption date and subject to certain conditions[406][407][408] Item 6. Exhibits This section provides an index of exhibits filed or furnished with the Quarterly Report on Form 10-Q, including amendments to employment agreements, the employee stock purchase plan, certifications, and financial information in iXBRL format - The exhibit index lists documents such as employment agreement amendments, the Amended and Restated uniQure N.V. Employee Stock Purchase Plan, CEO/CFO certifications (302 and 906), and financial information in iXBRL format[410][413]