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Aprea Therapeutics(APRE) - 2024 Q4 - Annual Report

Financial Condition - The company incurred a net loss of $14.3 million for the year ended December 31, 2023, and an accumulated deficit of $321.0 million as of the same date [271]. - The company has not generated any commercial revenue to date and expects to continue incurring significant operating losses for the foreseeable future [271]. - The company has approximately $22.8 million in cash and cash equivalents as of December 31, 2024, raising substantial doubt about its ability to continue as a going concern [276]. - The company has identified conditions that raise substantial doubt regarding its ability to continue as a going concern within one year after the filing of its Annual Report [276]. - The company has incurred significant losses since inception and may never achieve or maintain profitability [271]. - The company expects expenses to increase due to planned operations and will finance cash needs through equity offerings, debt financings, collaborations, and strategic alliances [286]. - Future capital requirements will depend on various factors, including the costs and outcomes of clinical trials and regulatory reviews [285]. - The company may need to seek additional funds sooner than planned due to changing circumstances and potential faster capital consumption [285]. - The company faces risks from global economic uncertainty, including geopolitical conflicts and regulatory changes, which could adversely affect its financial condition [288]. Product Development and Clinical Trials - The company is substantially dependent on the success of its product candidates ATRN-119 and APR-1051, which are currently in clinical development [264]. - The company has never obtained marketing approval for any product candidate and may face challenges in doing so in the future [268]. - The company is in the early stages of testing product candidates ATRN-119 and APR-1051 in Phase 1 clinical trials, with no later phase trials conducted yet [308]. - The company has filed and obtained INDs for ATRN-119 and APR-1051, but may face delays in filing INDs for other candidates due to manufacturing or study delays [309]. - The success of ATRN-119 and APR-1051 will depend on timely patient enrollment and completion of clinical trials, as well as obtaining marketing approvals [306]. - Patient enrollment in clinical trials is critical, and difficulties in enrollment could delay necessary marketing approvals [315]. - Adverse events during clinical trials could lead to interruptions or halts in trials, affecting the approval process by the FDA [321]. - The company has limited experience in conducting clinical trials, which may hinder the ability to complete pivotal trials [312]. - The company may need to conduct additional clinical trials or testing, which could lead to unplanned costs and delays in obtaining marketing approval [335]. - There is a high failure rate for product candidates in clinical trials, and setbacks could materially harm the company's business and financial condition [308]. - Clinical trials are lengthy and costly, with no assurance that they will demonstrate safety and efficacy to the satisfaction of regulatory authorities, potentially leading to additional funding needs [331]. Competition and Market Risks - The company may face substantial competition from larger pharmaceutical and biotechnology companies with greater resources and expertise [347]. - The commercial opportunity could be diminished if competitors develop safer or more effective drugs that gain market approval faster [348]. - Market acceptance of approved products is uncertain and may depend on factors such as efficacy, safety, and the willingness of healthcare providers to prescribe them [344]. - The company may need to develop companion diagnostic tests for its product candidates, which could present additional challenges and delays [339]. - The company is developing ATRN-119, an orally bioavailable small molecule targeting Ataxia ATR protein, which may face significant competition from other late-stage clinical drugs [352]. - APR-1051, another product candidate, is an orally bioavailable small molecule inhibitor of WEE1, with potential competition from various other companies' products in clinical development [353]. Regulatory and Compliance Challenges - The company must comply with stringent regulatory requirements for clinical trials and manufacturing, and failure to do so could result in significant sanctions [390]. - The FDA placed a partial clinical hold on the clinical trials of eprenetapopt in combination with azacitidine, and a clinical hold on trials evaluating eprenetapopt with acalabrutinib or with venetoclax and rituximab [374]. - Regulatory agencies may require extensive additional clinical trials post-approval, which could be expensive and time-consuming, potentially limiting or withdrawing product sales [363]. - The company may be subject to unfavorable pricing regulations and reimbursement policies that could negatively impact revenue generation from approved products [366]. - Coverage and reimbursement from government authorities and private insurers are uncertain, which may affect the demand and pricing of commercialized products [367]. Intellectual Property Risks - The company faces risks related to intellectual property, including potential challenges to the validity and enforceability of its patents, which could adversely affect its business and financial condition [399]. - The patent portfolio for eprenetapopt consists of method-of-use and formulation patent claims, but does not include composition-of-matter patents, which may limit competitive protection [406]. - The company may need to cease development or commercialization of product candidates if it cannot obtain necessary licenses from third parties involved in patent disputes [413]. - The company may face significant harm if it fails to obtain patent term extensions or data exclusivity for its product candidates, which could allow competitors to launch similar products after patent expiration [416]. - The company may incur substantial costs and time in defending its patents against infringement claims, which could distract from its core business operations [424]. Sales and Marketing Challenges - The company lacks a sales and marketing infrastructure and has no experience in selling or marketing pharmaceutical drugs, which may hinder commercialization efforts [357]. - Establishing a sales and marketing organization or outsourcing these functions could incur significant costs and delays in product launches [358]. - The company may face adverse effects on sales if the FDA approves generic versions of its product candidates, as generic drugs typically capture a significant percentage of sales from branded products [361]. - The lengthy approval process and unpredictability of clinical trial results may hinder the marketing approval of ATRN-119, significantly harming the company's business and prospects [451]. Miscellaneous Risks - The company may encounter unforeseen expenses and difficulties as it transitions from a research-focused entity to one capable of supporting commercial activities [282]. - The company may have to relinquish valuable rights to technology or revenue streams if it raises additional funds through collaborations or licensing arrangements [287]. - The company is dependent on a single third-party manufacturer for the active pharmaceutical ingredient, increasing the risk of supply issues [385]. - The company currently holds clinical trial liability insurance coverage for up to $5.0 million, which may not be adequate to cover all potential liabilities [370]. - The consequences of the U.K.'s withdrawal from the EU remain largely unknown, creating uncertainty regarding macro-economic and financial implications [455].