~ Achieved statistically significant, dose-dependent, and durable evidence of potential therapeutic benefit; Patients receiving high-dose AMT-130 showed 80% slowing of disease progression in the composite Unified Huntington's Disease Rating Scale (cUHDRS) at 24 months compared to a propensity score-weighted external control ~ ~ Investor conference call and webcast today at 8:30 a.m. ET ~ "These updated results are exciting and provide compelling evidence of potential therapeutic benefit," stated Victor Sung ...

~ Genezen to manufacture global commercial supply of HEMGENIX® and support uniQure's pipeline programs as a preferred customer ~ ~ uniQure's comprehensive review of operations and options to reduce expenses is ongoing and expected to be complete in the third quarter of 2024 ~ Under the proposed transaction, Genezen will be responsible for manufacturing global commercial supply of HEMGENIX® for CSL and providing development and manufacturing services to support uniQure's investigational gene therapies. uniQu ...

Strategic acquisition will enable Genezen to deliver late-phase and commercial gene therapy development and manufacturing services to customers around the world. BOSTON and INDIANAPOLIS, July 1, 2024 /PRNewswire/ -- Genezen, a best-in-class gene therapy CDMO, today announced an agreement to acquire uniQure's (Nasdaq: QURE) commercial gene therapy operations in Lexington, MA, bringing a strategic manufacturing facility and a world-class team of employees to the company. Under the terms of the agreement, Gene ...

~ Genezen to manufacture global commercial supply of HEMGENIX® and support uniQure's pipeline programs as a preferred customer ~ Genezen agreed to acquire uniQure's commercial-scale gene-therapy manufacturing facility for total consideration of $25 million, comprised of $12.5 million of newly issued Series C Preferred Stock and a $12.5 million convertible note. In addition, Matt Kapusta, chief executive officer of uniQure, will join the Board of Genezen upon the closing of the transaction, which is expected ...

~ Designation based on 24-month interim Phase I/II clinical data for AMT-130 announced in December 2023 ~ LEXINGTON, Mass. and AMSTERDAM, June 03, 2024 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation for its investigational gene therapy AMT-130 for the treatment of Hunti ...

uniQure Announces First Quarter 2024 Financial Results and Highlights Recent Company Progress Exhibit 99.1 ~ On track to initiate FDA interaction regarding AMT-130 in second quarter of 2024 and provide a clinical update from the Phase I/II trials in mid-2024 ~ ~ Clinical trial initiation for Fabry disease on track to begin in second quarter of 2024, followed by refractory mesial temporal lobe epilepsy and SOD1-ALS in third quarter of 2024 ~ ~ Comprehensive review of operations and options to reduce expenses ...

PART I – FINANCIAL INFORMATION [Financial Statements](index=5&type=section&id=Item%201.%20Financial%20Statements) The company reported **$8.5 million** in Q1 2024 revenues and a **$65.6 million** net loss, with **$770.1 million** total assets and **$146.8 million** equity as of March 31, 2024 Consolidated Balance Sheet Summary (Unaudited) | Balance Sheet Items | March 31, 2024 (in thousands) | December 31, 2023 (in thousands) | | :--- | :--- | :--- | | **Assets** | | | | Cash and cash equivalents | $243,062 | $241,360 | | Current investment securities | $312,621 | $376,532 | | Total current assets | $596,003 | $651,853 | | Total assets | $770,050 | $831,689 | | **Liabilities & Equity** | | | | Total current liabilities | $63,473 | $73,675 | | Long-term debt | $102,120 | $101,749 | | Liability from royalty financing agreement | $405,398 | $394,241 | | Total liabilities | $623,296 | $624,019 | | Total shareholders' equity | $146,754 | $207,670 | Consolidated Statement of Operations Summary (Unaudited) | Income Statement Items | Three months ended March 31, 2024 (in thousands) | Three months ended March 31, 2023 (in thousands) | | :--- | :--- | :--- | | Total revenues | $8,485 | $5,325 | | Research and development expenses | ($40,692) | ($60,809) | | Selling, general and administrative expenses | ($13,937) | ($17,848) | | Loss from operations | ($54,228) | ($74,172) | | Net loss | ($65,618) | ($77,227) | | Basic and diluted net loss per ordinary share | ($1.36) | ($1.63) | Consolidated Statement of Cash Flows Summary (Unaudited) | Cash Flow Items | Three months ended March 31, 2024 (in thousands) | Three months ended March 31, 2023 (in thousands) | | :--- | :--- | :--- | | Net cash used in operating activities | ($60,575) | ($78,302) | | Net cash generated from investing activities | $63,985 | $2,988 | | Net cash generated from financing activities | $0 | $131 | | Net increase / (decrease) in cash | $1,685 | ($74,149) | [Management's Discussion and Analysis of Financial Condition and Results of Operations](index=30&type=section&id=Item%202.%20Management%27s%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses the company's gene therapy pipeline, Q1 2024 financial performance with increased revenues and reduced net loss, and projected cash runway into Q2 2027 - uniQure is a gene therapy company with one approved product, **HEMGENIX®** for **hemophilia B**, and a pipeline including **AMT-130** for **Huntington's disease**, **AMT-162** for **ALS**, **AMT-260** for **refractory MTLE**, and **AMT-191** for **Fabry disease**[87](index=87&type=chunk) - As of March 31, 2024, the company held **$555.7 million** in cash, cash equivalents, and investment securities, projected to fund operations into the **second quarter of 2027**[101](index=101&type=chunk)[139](index=139&type=chunk) Financial Highlights Q1 2024 vs Q1 2023 | Metric | Q1 2024 (in thousands) | Q1 2023 (in thousands) | Change | | :--- | :--- | :--- | :--- | | Total Revenues | $8,485 | $5,325 | $3,160 | | R&D Expenses | ($40,692) | ($60,809) | $20,117 | | SG&A Expenses | ($13,937) | ($17,848) | $3,911 | | Net Loss | ($65,618) | ($77,227) | $11,609 | - R&D expenses decreased by **$20.1 million** to **$40.7 million** in Q1 2024, driven by a **$13.3 million** reduction in direct program spending, especially for **AMT-162 (ALS)**, and lower personnel costs post-2023 restructuring[120](index=120&type=chunk)[121](index=121&type=chunk)[131](index=131&type=chunk) - Interest expense increased to **$16.1 million** in Q1 2024 from **$3.6 million** in Q1 2023, primarily due to **$12.4 million** in interest expense from the May 2023 Royalty Financing Agreement[135](index=135&type=chunk) [Quantitative and Qualitative Disclosures About Market Risk](index=51&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) The company's market risks, including currency, price, interest rate, credit, and liquidity risks, remained materially unchanged in Q1 2024 - The company's market risks, encompassing currency, price, interest rate, credit, and liquidity risks, have not materially changed during Q1 2024[164](index=164&type=chunk)[165](index=165&type=chunk) [Controls and Procedures](index=51&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded the company's disclosure controls and procedures were effective as of March 31, 2024, with no material changes in internal control over financial reporting during the quarter - The CEO and CFO concluded that the company's disclosure controls and procedures were **effective** as of March 31, 2024[166](index=166&type=chunk) - No material changes occurred in internal control over financial reporting during Q1 2024[167](index=167&type=chunk) PART II – OTHER INFORMATION [Legal Proceedings](index=52&type=section&id=Item%201.%20Legal%20Proceedings) The company reported no legal proceedings during the period - The company reports no legal proceedings to disclose for the period[170](index=170&type=chunk) [Risk Factors](index=52&type=section&id=Item%201A.%20Risk%20Factors) The company faces significant risks including dependency on **AMT-130** clinical success, manufacturing complexities, funding needs, regulatory hurdles, and stock price volatility - The company's business is highly dependent on the clinical success of its lead candidate, **AMT-130** for **Huntington's disease**[173](index=173&type=chunk)[177](index=177&type=chunk) - Gene therapies are complex and expensive to manufacture, posing potential production challenges that could delay development and commercialization[173](index=173&type=chunk)[224](index=224&type=chunk) - The company has a history of net losses and requires additional funding to advance product candidates; failure to secure capital could delay or terminate development[173](index=173&type=chunk)[332](index=332&type=chunk) - Reliance on third parties for clinical trials introduces risks of delays or termination due to poor performance, impacting commercial prospects[180](index=180&type=chunk)[279](index=279&type=chunk) - The company's stock price remains volatile, influenced by clinical trial results, regulatory developments, and market conditions[173](index=173&type=chunk)[379](index=379&type=chunk) - The October 2023 restructuring, aimed at portfolio reprioritization and resource conservation, carries risks such as unachieved benefits, employee attrition, and management distraction[375](index=375&type=chunk)[377](index=377&type=chunk) [Unregistered Sales of Equity Securities, Use of Proceeds, and Issuer Purchases of Equity Securities](index=128&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%2C%20Use%20of%20Proceeds%2C%20and%20Issuer%20Purchases%20of%20Equity%20Securities) The company reported no unregistered sales of equity securities, use of proceeds, or issuer purchases during the period - None reported[403](index=403&type=chunk) [Other Information](index=128&type=section&id=Item%205.%20Other%20Information) No director or officer adopted or terminated a Rule 10b5-1 or non-Rule 10b5-1 trading arrangement in Q1 2024 - No director or officer adopted or terminated a Rule 10b5-1 or non-Rule 10b5-1 trading arrangement in Q1 2024[406](index=406&type=chunk) [Exhibits](index=128&type=section&id=Item%206.%20Exhibits) This section lists exhibits filed with the Form 10-Q, including CEO and CFO certifications and iXBRL financial statements - The report includes required certifications from the CEO and CFO as exhibits[410](index=410&type=chunk)

Exhibit 99.1 uniQure Announces 2023 Financial Results and Highlights Recent Company Progress ~ Presented promising clinical update from U.S. and European Phase I/II trials of AMT-130 in Huntington's disease; Up to three years of follow-up data to be presented in mid-2024; Regulatory interactions and clarity on potential strategies for clinical development expected in 2024 ~ ~ Announced FDA clearance of two Investigational New Drug (IND) applications; Initiation of Phase I/II clinical trials in mesial tempor ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington D.C. 20549 FORM 10-K (Mark One) ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2023 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission file number: 001-36294 uniQure N.V. (Exact name of Registrant as specified in its charter) The Netherlands (Jurisdiction of incorpor ...

[PART I – FINANCIAL INFORMATION](index=5&type=section&id=PART%20I%20%E2%80%93%20FINANCIAL%20INFORMATION) [Financial Statements](index=5&type=section&id=Item%201.%20Financial%20Statements) The unaudited consolidated financial statements for uniQure N.V. as of September 30, 2023, show increased total assets and a widened net loss due to higher expenses and a new royalty financing liability Consolidated Balance Sheet Highlights (in thousands) | Balance Sheet Items | Sep 30, 2023 (in thousands) | Dec 31, 2022 (in thousands) | | :--- | :--- | :--- | | Cash and cash equivalents | $229,484 | $228,012 | | Total current assets | $693,626 | $476,774 | | Total assets | $871,550 | $704,964 | | Total current liabilities | $68,607 | $75,919 | | Liability from royalty financing agreement | $383,711 | - | | Total liabilities | $602,766 | $228,955 | | Total shareholders' equity | $268,784 | $476,009 | Consolidated Statements of Operations Highlights (in thousands) | Income Statement Items | Nine months ended Sep 30, 2023 (in thousands) | Nine months ended Sep 30, 2022 (in thousands) | | :--- | :--- | :--- | | Total revenues | $9,154 | $3,738 | | Research and development expenses | ($172,245) | ($139,263) | | Selling, general and administrative expenses | ($57,103) | ($36,802) | | Loss from operations | ($221,123) | ($169,660) | | Interest expense | ($25,846) | ($8,279) | | Net loss | ($235,272) | ($133,596) | | Net loss per ordinary share | ($4.94) | ($2.86) | Consolidated Statements of Cash Flows Highlights (in thousands) | Cash Flow Items | Nine months ended Sep 30, 2023 (in thousands) | Nine months ended Sep 30, 2022 (in thousands) | | :--- | :--- | :--- | | Net cash used in operating activities | ($96,388) | ($90,380) | | Net cash used in investing activities | ($265,248) | ($14,522) | | Net cash generated from financing activities | $362,675 | $844 | | Net increase / (decrease) in cash | $1,463 | ($115,934) | - In May 2023, the company entered into a **Royalty Financing Agreement**, selling certain royalty rights on **HEMGENIX®** net sales to HemB SPV for an upfront payment, accounted for as debt[36](index=36&type=chunk) - On October 5, 2023, the company announced a reorganization plan to reduce its global workforce by approximately **20%** and discontinue over half of its research and technology projects, with an estimated cost of approximately **$2.3 million** in employee severance[86](index=86&type=chunk) [Management's Discussion and Analysis of Financial Condition and Results of Operations](index=31&type=section&id=Item%202.%20Management%27s%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses the company's gene therapy leadership, strategic reorganization, key clinical program advancements, and financial performance, including a widened net loss and extended cash runway [Business Developments](index=31&type=section&id=Business%20Developments) The company underwent a major reorganization to prioritize clinical programs like AMT-130, AMT-260, AMT-162, and AMT-191, secured $375 million from a royalty financing agreement, and received FDA clearance for AMT-260 IND - The company announced a reorganization to eliminate **~20%** of its workforce, close a research lab, and discontinue over half of its research and technology projects to prioritize advancing clinical-stage programs, with expected costs of **~$2.3 million** for severance[91](index=91&type=chunk)[92](index=92&type=chunk) - The FDA cleared the Investigational New Drug (IND) application for **AMT-260**, a gene therapy candidate for refractory mesial temporal lobe epilepsy (MTLE), in **August 2023**[89](index=89&type=chunk)[95](index=95&type=chunk) - Interim data from the Phase I/II trial of **AMT-130** for Huntington's disease showed the treatment was generally well-tolerated with a manageable safety profile, and clinical function was generally preserved at **24 months** (low-dose) and **12 months** (high-dose) compared to baseline[100](index=100&type=chunk)[101](index=101&type=chunk)[103](index=103&type=chunk) - The company entered into a royalty financing agreement, receiving an upfront payment of **$375.0 million** in exchange for rights to the lowest royalty tier on **HEMGENIX®** worldwide net sales[114](index=114&type=chunk) [Results of Operations](index=43&type=section&id=Results%20of%20Operations) Total revenues increased to $9.2 million, but net loss widened to $235.3 million due to higher R&D expenses, increased SG&A costs, and a surge in interest expense from the new royalty financing agreement Comparison of Operations (Nine Months Ended Sep 30, in thousands) | Line Item | 2023 (in thousands) | 2022 (in thousands) | Change (in thousands) | | :--- | :--- | :--- | :--- | | Total revenues | $9,154 | $3,738 | $5,416 | | R&D expenses | ($172,245) | ($139,263) | ($32,982) | | SG&A expenses | ($57,103) | ($36,802) | ($20,301) | | Loss from operations | ($221,123) | ($169,660) | ($51,463) | | Net loss | ($235,272) | ($133,596) | ($101,676) | Revenue Breakdown (Nine Months Ended Sep 30, in thousands) | Revenue Source | 2023 (in thousands) | 2022 (in thousands) | Change (in thousands) | | :--- | :--- | :--- | :--- | | License revenues | $1,290 | $0 | $1,290 | | Contract manufacturing revenues | $6,596 | $0 | $6,596 | | Collaboration revenues | $1,268 | $3,738 | ($2,470) | | **Total revenues** | **$9,154** | **$3,738** | **$5,416** | Direct R&D Expenses by Program (Nine Months Ended Sep 30, in thousands) | Program | 2023 (in thousands) | 2022 (in thousands) | Change (in thousands) | | :--- | :--- | :--- | :--- | | Amyotrophic lateral sclerosis (AMT-162) | $13,048 | $0 | $13,048 | | Temporal lobe epilepsy (AMT-260) | $11,970 | $11,394 | $576 | | Huntington's disease (AMT-130) | $11,093 | $15,721 | ($4,628) | | Fabry disease (AMT-191) | $1,846 | $1,664 | $182 | [Financial Position, Liquidity and Capital Resources](index=58&type=section&id=Financial%20Position%2C%20Liquidity%20and%20Capital%20Resources) As of September 30, 2023, the company held $662.1 million in liquid assets, projecting funding into Q2 2027, supported by royalty financing proceeds and a milestone payment, while managing significant debt and lease obligations - The company believes its cash, cash equivalents, and investment securities of **$662.1 million** as of September 30, 2023, will fund operations into the **second quarter of 2027**[187](index=187&type=chunk) - Net cash used in operating activities was **$96.4 million** for the nine months ended Sep 30, 2023, compared to **$90.4 million** in the prior year period[202](index=202&type=chunk) - Net cash generated from financing activities was **$362.7 million**, primarily due to **$370.1 million** in net proceeds from the **Royalty Financing Agreement**[208](index=208&type=chunk)[209](index=209&type=chunk) - Key contractual obligations include **$100.0 million** in principal debt to Hercules due in **2027**, the royalty financing agreement liability, **$54.8 million** in lease payments, and up to **EUR 190.0 million** in potential milestone payments related to the uniQure France SAS acquisition[188](index=188&type=chunk)[189](index=189&type=chunk)[191](index=191&type=chunk)[192](index=192&type=chunk) [Quantitative and Qualitative Disclosures About Market Risk](index=67&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) The company's market risks, including currency, price, and interest rate fluctuations, remain consistent with prior disclosures, with a focus on capital preservation and minimizing adverse financial market effects - The company's market risks, including currency, price, and interest rate risk, have **not materially changed** during the nine months ended September 30, 2023, from what was disclosed in the Annual Report[218](index=218&type=chunk) [Controls and Procedures](index=67&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded that disclosure controls and procedures were effective as of September 30, 2023, with no material changes to internal control over financial reporting during the quarter - Based on an evaluation as of September 30, 2023, the CEO and CFO concluded that the company's disclosure controls and procedures were **effective**[219](index=219&type=chunk) - There were **no changes** in the company's internal control over financial reporting during the quarter that materially affected, or are reasonably likely to materially affect, internal controls[220](index=220&type=chunk) [PART II – OTHER INFORMATION](index=68&type=section&id=PART%20II%20%E2%80%93%20OTHER%20INFORMATION) [Legal Proceedings](index=68&type=section&id=Item%201.%20Legal%20Proceedings) The company reported no legal proceedings during the period - None[223](index=223&type=chunk) [Risk Factors](index=68&type=section&id=Item%201A.%20Risk%20Factors) The company faces significant risks including clinical trial delays, financial funding needs, operational dependencies, strategic reorganization effectiveness, and intellectual property protection challenges - Clinical Trial Risks: The company has encountered and may continue to encounter **delays** in clinical trials or **fail to demonstrate the safety and efficacy** of its product candidates[226](index=226&type=chunk) - Financial Risks: The company will likely need to raise **additional funding**, which may **not be available**, and the amount required depends on future payments from CSL Behring for **HEMGENIX®**[226](index=226&type=chunk) - Operational Risks: The company's manufacturing facility is subject to **significant government regulations**, and it also **relies on third parties** for preclinical studies and clinical trials, who may **not perform satisfactorily**[226](index=226&type=chunk) - Strategic Risks: Actions taken to restructure the business, including the recent Reorganization, may **not be as effective as anticipated**, and the company may also **not be successful** in its efforts to build a pipeline of additional product candidates[226](index=226&type=chunk)[227](index=227&type=chunk) - Intellectual Property Risks: The company's ability to commercialize products may be **impaired** if it is **unable to obtain and maintain patent protection** for its technology and products[226](index=226&type=chunk) [Unregistered Sales of Equity Securities, Use of Proceeds, and Issuer Purchases of Equity Securities](index=131&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%2C%20Use%20of%20Proceeds%2C%20and%20Issuer%20Purchases%20of%20Equity%20Securities) The company reported no unregistered sales of equity securities, use of proceeds, or issuer purchases of equity securities during the period - None[434](index=434&type=chunk) [Defaults Upon Senior Securities](index=131&type=section&id=Item%203.%20Defaults%20Upon%20Senior%20Securities) The company reported no defaults upon senior securities - None[435](index=435&type=chunk) [Mine Safety Disclosures](index=131&type=section&id=Item%204.%20Mine%20Safety%20Disclosures) This item is not applicable to the company - Not applicable[436](index=436&type=chunk) [Other Information](index=131&type=section&id=Item%205.%20Other%20Information) No director or officer adopted or terminated a Rule 10b5-1 or non-Rule 10b5-1 trading arrangement during the third quarter of 2023 - **No director or officer adopted or terminated** a Rule 10b5-1 trading arrangement or non-Rule 10b5-1 trading arrangement during the three months ended September 30, 2023[437](index=437&type=chunk) [Exhibits](index=131&type=section&id=Item%206.%20Exhibits) This section lists filed exhibits, including the Termination and Consulting Agreement with Dr. Ricardo Dolmetsch, CEO and CFO certifications, and iXBRL data files - Filed exhibits include **CEO and CFO certifications** (31.1, 31.2, 32.1) and the **Termination and Consulting Agreement** for Ricardo Dolmetsch, Ph.D. (10.1)[441](index=441&type=chunk)