uniQure (QURE) risk factors, FY2024 10-K

Risks Related to Our Business and the Development of Our Product Candidates

1. 01We have encountered and may encounter future delays in and impediments to the progress of our clinical trials or fail to demonstrate the safety and efficacy of our product candidates
2. 02Our progress in early-stage clinical trials may not be predictive of long-term efficacy in late-stage clinical trials, and our progress in trials for one product candidate may not be predictive of progress in trials for other product candidates
3. 03Interim or preliminary results from our clinical trials may change as more data become available, as such data are subject to regulatory audit and verification procedures, and regulatory review, which could result in material changes in the final results and conclusions
4. 04Data analyses conducted on a post-hoc basis and using external, historical controls may not be accepted as a basis for regulatory approval
5. 05We are making use of exploratory biomarkers and other data that are not scientifically validated, and our reliance on these data may lead us to direct our resources inefficiently
6. 06We may not be successful in our efforts to use our gene therapy technology platform to build a pipeline of additional product candidates or otherwise leverage our research and technology to remain competitive
7. 07We may not be successful in obtaining rights from external parties to new product candidates and key technologies, or in securing partnerships to support the development or commercialization of our product candidates
8. 08Negative public opinion and increased regulatory scrutiny of gene therapy and genetic research may damage the public perception of our product candidates or adversely affect our ability to conduct our business or obtain marketing approvals for our product candidates
9. 09We face substantial competition, and others may discover, develop, or commercialize competing products before or more successfully than we do

Risks Related to Regulatory Approval of Our Products

1. 10We cannot predict when or if we will obtain marketing approval to commercialize our product candidates
2. 11The risks associated with the marketing approval process are heightened by the status of our products as gene therapies
3. 12As appropriate, we intend to seek available periods of regulatory exclusivity for our product candidates. However, there is no guarantee that we will be granted these periods of regulatory exclusivity or that we will be able to maintain these periods of exclusivity
4. 13We and our commercial partner face uncertainty related to insurance coverage of, and pricing and reimbursement for, HEMGENIX® and other product candidates for which we may receive marketing approval
5. 14Due to the generally limited addressable market for our target orphan indications and the potential for our therapies to offer therapeutic benefit in a single administration, we face uncertainty related to our product candidates
6. 15If any of our product candidates receive regulatory approval, we and/or our partners will be subject to extensive regulatory requirements. Failure to fulfill and comply with the applicable regulatory requirements could result in regulatory enforcement actions that would be detrimental to our business

Risks Related to Commercialization

1. 16If we are unable to successfully commercialize our product candidates or experience significant delays in doing so, our business could be materially harmed
2. 17Any approved gene therapy we seek to commercialize may fail to achieve the degree of market acceptance by physicians, patients, third party payers and others in the medical community necessary for commercial success
3. 18The affected populations for our gene therapies may be smaller than we or third parties currently project, which may affect the size of our addressable markets
4. 19If the market opportunities for our product candidates are smaller than we believe they are, our product revenues may be adversely affected, and our business may suffer
5. 20Ethical, legal, and social issues associated with genetic testing may reduce demand for any gene therapy products for which we obtain marketing approval
6. 21If we obtain approval to commercialize any of our product candidates outside of the U.S., a variety of risks associated with international operations could materially adversely affect our business

Risks Related to Manufacturing and Our Dependence on Third Parties

1. 22The Lexington Transaction may not yield the benefits that we expect and may result in additional risks to our business, including those related to Genezen’s ability to manufacture HEMGENIX in accordance with regulatory requirements and to meet CSL Behring’s supply requirements for commercial product
2. 23Gene therapies are complex, expensive and difficult to manufacture. Genezen or any third-party manufacturer that we engage could experience capacity, production or technology transfer challenges that could result in delays in our development or commercialization schedules or otherwise adversely affect our business
3. 24Our use of viruses, chemicals and other potentially hazardous materials requires us and our contract manufacturers to comply with regulatory requirements and exposes us to significant potential liabilities
4. 25Our business may be adversely affected if we or third-party manufacturers are unable to validate our manufacturing processes and methods or develop new processes and methods to meet our product supply needs and obligations
5. 26We rely on third parties for important aspects of our development programs. If these parties do not perform successfully or if we are unable to enter into or maintain key collaborations or other contractual arrangements, our business could be adversely affected

Risks Related to Our Intellectual Property

1. 27If we fail to comply with our obligations in our intellectual property licenses with third parties, we could lose rights that are important to our business
2. 28If we are unable to obtain and maintain patent protection for our technology and products, or if the scope of the patent protection is not sufficiently broad, our ability to successfully commercialize our products may be impaired
3. 29We may become involved in lawsuits to protect or enforce our patents or other intellectual property, or third parties may assert their intellectual property rights against us, which could be expensive, time consuming and unsuccessful
4. 30If we are unable to protect the confidentiality of our proprietary information and know-how, the value of our technology and products could be adversely affected
5. 31Our reliance on third parties may require us to share our trade secrets, which could increase the possibility that a competitor will discover them or that our trade secrets will be misappropriated or disclosed
6. 32Intellectual property rights do not necessarily address all potential threats to our competitive advantage

Risks Related to Our Financial Position and Need for Additional Capital

1. 33We had net losses in the years ended December 31, 2024 and 2023, have incurred significant losses in previous years and expect to incur losses in the future, and may never achieve or maintain profitability
2. 34Our business development and strategic initiatives, including divestitures such as the Lexington Transaction, acquisitions, partnerships, collaborations or other transactions, may not achieve their intended benefits or goals and may result in additional risks to our business
3. 35Actions that we have taken or may take in the future to restructure our business in alignment with our strategic priorities may not be as effective as anticipated, may not result in cost savings to us and could disrupt our business
4. 36Our existing and any future indebtedness could adversely affect our ability to operate our business

Risks Related to Other Legal Compliance Matters

1. 37Our relationships with employees, customers and third parties are subject to applicable laws and regulations, the non-compliance of any of which could have a material adverse effect on our business, financial condition, and results of operations
2. 38Product liability lawsuits could cause us to incur substantial liabilities and to limit commercialization of our therapies
3. 39Healthcare legislative and regulatory reform measures may have a material adverse effect on our financial operations
4. 40Our future growth may depend, in part, on our ability to penetrate markets outside of the U.S. and Europe where we would be subject to additional regulatory burdens and other risks and uncertainties
5. 41There may be future changes in legal and regulatory requirements that may materially impact our results of operations

Risks Related to Our Ordinary Shares

1. 42The market price of our ordinary shares has been and may in the future be volatile and fluctuate substantially
2. 43Our directors, executive officers, and major shareholders, if they choose to act together, will continue to have a significant degree of control with respect to matters submitted to shareholders for approval
3. 44The rights and responsibilities of our shareholders and directors are governed by Dutch law and differ in some important respects from the rights and responsibilities of shareholders under U.S. law
4. 45Provisions of our articles of association or Dutch corporate law might deter acquisition bids for us that might otherwise be considered favorable and could make it more difficult to replace our board, ensuring continuity and stability in corporate governance in line with Dutch law and case law requirements. t
5. 46We do not expect to pay dividends in the foreseeable future. Accordingly, any returns on an investment in our ordinary shares will likely depend entirely upon any future appreciation in the price of our ordinary shares, which is uncertain
6. 47Any U.S. or other foreign judgments may be difficult to enforce against us in the Netherlands
7. 48We are not obligated to, and do not, comply with all best practice provisions of the Dutch Corporate Governance Code

General Risks

1. 49Our future success depends on our ability to retain key executives, technical staff, and other employees and to attract, retain and motivate qualified personnel
2. 50We may be adversely affected by unstable market and economic conditions and potential macroeconomic effects, such as inflation, new or increased tariffs and higher interest rates, which may negatively impact our business, financial condition and share price
3. 51If we fail to maintain an effective system of internal controls, we may be unable to accurately report our results of operations or prevent fraud or fail to meet our reporting obligations, and investor confidence and the market price of our ordinary shares may be materially and adversely affected
4. 52Our internal computer systems, or those of our collaborators, third-party vendors, contractors or consultants, may fail or suffer security breaches, which could result in a material disruption of our business and development programs
5. 53If securities or industry analysts do not publish, cease to publish or publish inaccurate or unfavorable research about our business, our share price and trading volume could decline
6. 54If we do not achieve our projected development and financial goals in the timeframes we announce and expect, the commercialization of our product candidates may be delayed and, as a result, our share price may decline
7. 55Environmental sustainability and social initiatives that we undertake may impose additional costs on our business and expose us to new risks