Pivotal phase 2 LEGEND study of EG-70 in BCG-unresponsive non-muscle invasive bladder cancer (NMIBC) remains on track with interim data anticipated in mid-2024 Closed a $200 million oversubscribed private financing, expected to extend cash runway into 2027 BOSTON and MONTREAL, March 11, 2024 /PRNewswire/ - enGene Holdings Inc. (Nasdaq: ENGN or "enGene" or the "Company"), a clinical-stage genetic medicines company whose non-viral, intravesical lead product candidate, EG-70, is in a pivotal study for BCG-unre ...

Financing included participation from new and existing investors Pro forma cash and cash equivalents expected to fund current operating plan into 2027 BOSTON and MONTREAL, Feb. 14, 2024 /PRNewswire/ - enGene Holdings Inc. (Nasdaq: ENGN or "enGene" or the "Company"), a clinical-stage genetic medicines company whose non-viral lead program EG-70 is in a pivotal study for BCG-unresponsive non-muscle invasive bladder cancer (NMIBC), today announced that it has agreed to sell 20 million of its common shares at a ...

Experienced biotech executive brings public company expertise in legal, corporate governance, and compliance to support enGene during late-stage clinical development and anticipated future commercial launch of its lead program EG-70 BOSTON and MONTREAL, Jan. 30, 2024 /PRNewswire/ - enGene Holdings Inc. ("enGene," (Nasdaq: ENGN), a clinical-stage genetic medicines company whose lead program is in a pivotal study for BCG-unresponsive non-muscle invasive bladder cancer (NMIBC), today announced the appointment ...

Achieved primary and secondary endpoints in Phase 1 portion of LEGEND study in non-muscle invasive bladder cancer (NIMBC); pivotal portion underway with interim data expected mid this year Completed a reverse merger transaction with Forbion European Acquisition Corp (FEAC), launched as a public company on November 1, 2023, and completed an upsized PIPE financing Combined transactions resulted in net proceeds of approximately $109 million Closed an expanded $50M debt facility with Hercules Capital in Decembe ...

Funding and Financial Obligations - The company does not have committed external funding sources for development efforts and may need to rely on public or private equity offerings, debt financing, or strategic collaborations to meet cash requirements [186]. - The company is subject to fixed payment obligations under the Amended Loan Agreement, which limits its ability to incur additional debt or make capital expenditures [186]. - The company may need to relinquish rights to product candidates or technologies if it cannot raise sufficient capital, which could adversely affect its business [189]. - The company estimates that the market opportunities for its product candidates may be limited to a small group of patients, potentially impacting revenue projections [259]. - The company has not lost cash or cash equivalents due to the collapse of Silicon Valley Bank, but ongoing liquidity concerns in the financial services industry may affect operations and stock price [372]. Competition and Market Dynamics - The company faces significant competition from larger pharmaceutical companies and specialty biotechnology firms, which may hinder its ability to develop and commercialize products effectively [190]. - The lead product candidate, EG-70, is aimed at treating BCG-unresponsive non-muscle invasive bladder cancer (NMIBC) and faces competition from numerous companies [191]. - Market acceptance of product candidates is crucial for commercial success, and failure to achieve this could result in insufficient revenue generation [222]. - The lead product candidate, EG-70, targets approximately 60,000 new patients globally each year with BCG-unresponsive NMIBC, indicating a high unmet medical need [260]. Research and Development Challenges - The company is focusing its research and development on its gene therapy platform, which is still at an early stage and carries significant development risks [195]. - The regulatory approval process for novel product candidates is expected to be lengthy and expensive, with uncertain outcomes [202]. - The company has not previously conducted any IND-enabling studies or clinical trials, which may limit its ability to develop promising product candidates [196]. - The company may experience delays in clinical trials and commercialization due to challenges in establishing manufacturing processes and regulatory approvals [200]. - The company’s gene therapy technologies are largely unproven, making it difficult to predict development timelines and costs [198]. - Clinical trials may face delays due to various factors, including difficulties in patient enrollment and regulatory discussions, which could impact the ability to receive marketing approval [206]. - The timely completion of clinical trials depends on enrolling a sufficient number of patients, and challenges in this area could lead to increased costs and affect trial outcomes [210]. - The company may face competition for patient enrollment from other clinical trials, which could limit the number of available participants [213]. - Adverse side effects or safety risks associated with product candidates could lead to delays or suspensions in clinical trials, impacting commercial prospects [215]. - The company is focusing on developing its initial product candidate, EG-70, but acknowledges the risk of this being a wrong choice due to limited resources [231]. - The gene therapy platform aims to generate multiple product candidates, but there is uncertainty in identifying viable candidates for clinical development [232]. Manufacturing and Supply Chain Risks - The reliance on third-party manufacturers for production increases the risk of supply shortages and delays in clinical trials [234]. - Manufacturing processes for product candidates are complex and may lead to higher costs and production failures [240]. - The company lacks experience in developing its own manufacturing facility, which could hinder its ability to control supply and manage costs effectively [251]. - There is a risk of increased manufacturing costs if processes or standards change, impacting profitability [246]. - The company may face challenges in scaling manufacturing processes for advanced clinical trials or commercialization [244]. - Regulatory compliance and quality assurance are critical, and any failure in these areas could delay product approvals [245]. - The company is susceptible to operational disruptions due to reliance on third-party suppliers and manufacturers [241]. Regulatory Environment and Approval Processes - The regulatory approval process for product candidates is lengthy and unpredictable, which could delay commercialization efforts [274]. - Regulatory authorities may impose significant limitations on approved product candidates, including narrow indications and post-marketing clinical trial requirements [288]. - The approval process for product candidates is expensive and may take many years, with a small percentage successfully completing the FDA approval process [291]. - The company may face challenges in obtaining regulatory approval due to the novel nature of its product candidates [293]. - Approval of genetic or biomarker diagnostic tests may be necessary for advancing some product candidates to clinical trials, complicating the regulatory pathway [296]. - Delays in obtaining regulatory approval could materially harm the commercial prospects and revenue generation capabilities of the company [298]. - The FDA may require additional studies or disagree with the design of clinical trials, impacting the approval process [298]. - The company has not received approval to market any of its product candidates from regulatory authorities in any jurisdiction [290]. - The company does not have any product candidates approved for sale in any jurisdiction, including international markets [300]. - Regulatory approval processes vary significantly across jurisdictions, potentially leading to delays and increased costs for the company [300]. - The FDA may grant priority review designation for product candidates, which could shorten the review period from ten months to six months, but this is not guaranteed [301]. - Ongoing regulatory obligations and reporting requirements may result in significant additional expenses for the company if product candidates are approved [302]. - The company relies on contract manufacturers who must comply with extensive regulatory requirements, and any failure in compliance could adversely affect product availability and safety [314]. Legal and Compliance Risks - The company is subject to various healthcare laws and regulations, including the Anti-Kickback Statute (AKS) and the False Claims Act (FCA), which could lead to criminal sanctions and civil penalties [360]. - Violations of the AKS could result in exclusion from federal healthcare programs and substantial civil and criminal penalties, impacting future earnings [360]. - The company faces increased scrutiny from federal and state enforcement bodies regarding interactions with healthcare providers, which could lead to investigations and increased costs [363]. - Compliance with international anti-corruption and trade laws is critical, as violations could result in substantial fines and reputational harm [367]. - The company may incur significant legal expenses and management distraction if investigations into marketing practices occur [364]. Data Privacy and Cybersecurity - Compliance with stringent privacy laws and regulations is essential, as violations could lead to significant penalties and reputational harm [341]. - Evolving data privacy laws may require ongoing modifications to company policies and systems, incurring additional costs [344]. - The California Consumer Privacy Act (CCPA) imposes significant requirements on businesses processing personal information of California residents, including the right to access and request erasure of personal data [345]. - The GDPR, effective since May 2018, imposes strict obligations on companies regarding personal data processing and cross-border data transfers, with potential fines of up to €20 million or 4% of total worldwide annual turnover for non-compliance [346]. - Cybersecurity threats have increased in frequency and sophistication, posing risks to the integrity and confidentiality of sensitive data, which could disrupt business operations [352]. - Any cyberattack leading to unauthorized access or data breaches could result in significant legal and financial exposure, including potential fines and reputational damage [357]. - The company does not maintain separate cyber liability insurance, which may leave it vulnerable to significant losses from security incidents [358]. Economic and Geopolitical Factors - Global economic uncertainty, including the conflict between Russia and Ukraine, could adversely affect the company's business and results of operations [370]. - Increased inflation has raised operating costs, which may impact demand, pricing, and manufacturing costs for the company's product candidates [371]. - The company faces significant challenges in accurately forecasting and planning future business activities due to various known and unknown economic and geopolitical factors, which could adversely affect its business and financial condition [373]. - Research and development activities may be conducted in non-U.S. jurisdictions, such as China, and disruptions in these operations could materially impact the company's business and results of operations [374]. Patent and Intellectual Property Risks - The company owns certain granted patents and pending patent applications related to its gene therapy platform, but the ability to secure and maintain patent protection is uncertain and could adversely affect its business [380]. - The patent prosecution process is expensive and complex, and the company may not be able to obtain or maintain necessary patent protections, impacting its competitive position [383]. - The uncertainty surrounding patent rights in the biotechnology and pharmaceutical sectors may hinder the company's ability to effectively protect its technology and product candidates [385].