uniQure(QURE) - 2024 Q4 - Annual Report

Financial Performance - The company reported net losses for the years ended December 31, 2024 and 2023, with significant losses in previous years and expects to incur losses in the future[16] - The company reported net losses for the years ended December 31, 2024 and 2023, and anticipates continued losses in the future, indicating a risk of never achieving profitability[16] - The company has incurred significant losses in previous years and will need to raise additional funding to advance product development, which may not be available on acceptable terms[18] Product Development and Clinical Trials - The lead product candidate, AMT-130, is critical for the treatment of Huntington's disease, and any failure in its clinical development could adversely affect the company's business[15] - The company may face delays in clinical trials and challenges in demonstrating the safety and efficacy of its product candidates, which could impact future operations[15] - The company may face delays in clinical trials and challenges in demonstrating safety and efficacy of its product candidates, which could materially harm its business[15] - The company has encountered risks associated with the marketing approval process for gene therapies, which may affect the commercialization of its product candidates[15] Regulatory and Manufacturing Risks - The company has encountered risks related to the manufacturing of gene therapies, which are complex and expensive, potentially leading to delays in development or commercialization[15] - The company is dependent on third-party manufacturers for its product candidates, which may not perform satisfactorily or comply with regulatory requirements[15] - The company relies on third parties for manufacturing its product candidates, which may not perform satisfactorily or comply with regulations[15] - The company plans to leverage specialized regulatory pathways, such as the FDA's accelerated approval pathway, but may struggle to maintain the benefits associated with these designations[15] - The company may leverage specialized regulatory pathways for its product candidates, but there is no guarantee of obtaining or maintaining the associated benefits[15] Market and Competitive Landscape - The market price of the company's ordinary shares has been volatile and may fluctuate substantially in the future[18] - The company faces substantial competition in the industry, which may impact its ability to successfully commercialize its products[18] - The company faces substantial competition and risks that competitors may develop or commercialize competing products more successfully[18] Business Development and Strategic Initiatives - The company’s business development initiatives, including acquisitions and partnerships, may not achieve their intended benefits, posing additional risks[15] - The Lexington Transaction may not yield expected benefits and could introduce additional risks related to manufacturing and supply requirements[15]