uniQure(US:QURE)2024-05-07 11:57PART I – FINANCIAL INFORMATION Financial Statements 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 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 disease87 - 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 2027101139 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 restructuring120121131 - 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 Agreement135 Quantitative and Qualitative Disclosures About Market Risk 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 2024164165 Controls and Procedures 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, 2024166 - No material changes occurred in internal control over financial reporting during Q1 2024167 PART II – OTHER INFORMATION Legal Proceedings The company reported no legal proceedings during the period - The company reports no legal proceedings to disclose for the period170 Risk Factors 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 disease173177 - Gene therapies are complex and expensive to manufacture, posing potential production challenges that could delay development and commercialization173224 - The company has a history of net losses and requires additional funding to advance product candidates; failure to secure capital could delay or terminate development173332 - Reliance on third parties for clinical trials introduces risks of delays or termination due to poor performance, impacting commercial prospects180279 - The company's stock price remains volatile, influenced by clinical trial results, regulatory developments, and market conditions173379 - The October 2023 restructuring, aimed at portfolio reprioritization and resource conservation, carries risks such as unachieved benefits, employee attrition, and management distraction375377 Unregistered Sales of Equity Securities, Use of Proceeds, and Issuer Purchases of Equity Securities The company reported no unregistered sales of equity securities, use of proceeds, or issuer purchases during the period - None reported403 Other Information 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 2024406 Exhibits 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 exhibits410