Abeona Therapeutics (ABEO) risk factors, FY2024 10-K

Risks related to the discovery and development of our product candidates

1. 01We may encounter substantial delays in our clinical studies, such as clinical holds, or we may fail to demonstrate safety and efficacy to the satisfaction of applicable regulatory authorities
2. 02We may find it difficult to enroll patients in our clinical studies, which could delay or prevent clinical studies of our product candidates
3. 03Our products or product candidates may cause undesirable side effects or have other properties that could delay or prevent their regulatory approval or commercialization
4. 04Even if we complete the necessary preclinical and clinical studies, we cannot predict when or if we will obtain regulatory approval to commercialize a product candidate or the approval may be for a narrower indication than we expect
5. 05Certain of our product candidates have received orphan drug designation from the FDA, there is no guarantee that we will be able to maintain this designation, receive this designation for any of our other product candidates, or receive or maintain any corresponding benefits, including periods of exclusivity
6. 06Even if we obtain regulatory approval for a product candidate, our products will remain subject to regulatory scrutiny
7. 07Any government investigation of alleged violations of law could require us to expend significant time and resources in response and could generate negative publicity. The occurrence of any event or penalty described above may inhibit our ability to commercialize our product candidates and generate revenues
8. 08The Complete Response Letter related to our Biologics License Application for pz-cel for the treatment of patients with recessive dystrophic epidermolysis bullosa may impair our ability to successfully commercialize pz-cel

Risks related to manufacturing

1. 09We could experience production problems in our manufacturing facilities that result in delays in our development or commercialization programs or otherwise adversely affect our business
2. 10We may rely on third parties to conduct aspects of our product manufacturing, and these third parties may not perform satisfactorily. We may rely on third parties to produce certain materials for our product candidates and, therefore, we can control only certain aspects of their activities
3. 11If we, our collaborators, or any third-party manufacturers we engage fail to comply with environmental, health and safety laws and regulations, we could become subject to fines or penalties or incur costs that could harm our business
4. 12The widespread outbreak of an illness, communicable disease, or any other public health crisis could adversely affect our business, results of operations and financial condition

Risks related to our reliance on third-parties

1. 13We expect to rely on third parties to conduct some or all aspects of our viral vector production, drug product manufacturing, research and preclinical, and clinical testing, and these third parties may not perform satisfactorily
2. 14Our reliance on third parties requires us to share our trade secrets, which increases the possibility that a competitor will discover them or that our trade secrets will be misappropriated or disclosed

Risks related to commercializing our product candidates

1. 15If we do not obtain the necessary U.S. or worldwide regulatory approvals to commercialize pz-cel, we will not be able to sell pz-cel
2. 16Even if we receive regulatory approval for pz-cel, our lead drug candidate, we may not be able to successfully commercialize the product and the revenue that we generate from its sales, if any, may be limited
3. 17Our drug candidates are subject to the risks of failure inherent in the development of pharmaceutical products based on new technologies, and our failure to develop safe and commercially viable drugs would severely limit our ability to become profitable or to achieve significant revenues
4. 18We are subject to extensive governmental regulation, which increases our cost of doing business and may affect our ability to commercialize any new products that we may develop
5. 19We may incur substantial product liability expenses due to the use or misuse of our products for which we may be unable to obtain insurance coverage
6. 20Intense competition may limit our ability to successfully develop and market commercial products
7. 21Our products and product candidates may face competition sooner than anticipated
8. 22Our ability to successfully develop and commercialize our drug candidates will substantially depend upon the availability of reimbursement funds for the costs of the resulting drugs and related treatments
9. 23The market may not accept any pharmaceutical products that we develop, thereby materially impairing our ability to generate revenue from such products
10. 24Adverse public perception of gene therapy products may negatively affect demand for, or regulatory approval of, our product candidates
11. 25Healthcare reform measures could hinder or prevent our product candidates’ commercial success
12. 26We may be subject, directly or indirectly, to federal, state, and foreign healthcare laws and regulations, including fraud and abuse laws, false claims laws and health information privacy and security laws. If we are unable to comply, or have not fully complied, with such laws, we could face substantial penalties
13. 27We are subject to extensive laws and regulations related to data privacy, and our failure to comply with these laws and regulations could harm our business
14. 28Trends toward managed health care, health technology assessment, and downward price pressures on medical products and services may limit our ability to profitably sell any drugs that we may develop

Risks related to our intellectual property

1. 29Our rights to develop and commercialize our product candidates are subject to, in part, the terms and conditions of licenses granted to us by others
2. 30If any dispute over in-licensed intellectual property prevents or impairs our ability to maintain our current licensing arrangements on acceptable terms, we may be unable to successfully develop and commercialize the affected product candidates
3. 31Our intellectual property licenses with third parties may be subject to disagreements over contract interpretation, which could narrow the scope of our rights to the relevant intellectual property or technology or increase our financial or other obligations to our licensors
4. 32We may not be successful in obtaining necessary rights to our product candidates through acquisitions and in-licenses
5. 33Issued patents covering our product candidates could be found invalid or unenforceable if challenged in court. We may not be able to protect our trade secrets in court
6. 34Third-parties may initiate legal proceedings alleging that we are infringing their intellectual property rights, the outcome of which would be uncertain and could harm our business
7. 35We may be subject to claims asserting that our employees, consultants, or advisors have wrongfully used or disclosed alleged trade secrets of their current or former employers or claims asserting ownership of what we regard as our own intellectual property
8. 36If we do not obtain patent term extension and data exclusivity for our product candidates, our business may be harmed

Risks related to our financial condition and capital requirements

1. 37We have experienced a history of losses; we expect to incur future losses and we may be unable to obtain necessary additional capital to fund operations in the future
2. 38We do not have significant operating revenue and may never achieve profitability
3. 39Failure to achieve and maintain effective internal controls could have a material adverse effect on our business
4. 40We may need to raise additional capital to operate our business, and our failure to obtain funding when needed or on terms that are favorable to us may force us to delay, reduce or eliminate our development programs or commercialization efforts

Risks related to our common stock

1. 41The market price of our common stock may be volatile and adversely affected by several factors
2. 42Raising additional funds by issuing securities or through licensing or lending arrangements or through our at-the-market sale agreement may cause dilution to our existing stockholders, restrict our operations, or require us to relinquish proprietary rights
3. 43Our ability to use our net operating loss carry forwards may be subject to limitation

Risks related to cybersecurity

1. 44Significant disruptions of information technology (“IT”) systems, breaches of data security, or unauthorized disclosures of personal information (including sensitive personal information) could adversely affect our business and could subject us to liability or reputational damage