Summary of enGene Holdings Conference Call Company Overview - Company: enGene Holdings (NasdaqCM: ENGN) - Focus: Development of non-viral gene therapies for bladder cancer, specifically targeting non-muscle invasive bladder cancer (NMIBC) Industry Context - Disease Prevalence: NMIBC affects approximately 730,000 patients in the US, with about 85,000 new cases annually [1][2] - Market Size: The management of NMIBC is estimated to cost the healthcare system $6.5 billion per year [3] - Current Treatment Landscape: Traditional treatments include BCG therapy, IV chemotherapy, and radical cystectomy, which are often associated with high morbidity and mortality [4][5] Core Product Insights - Product: Detalimogene voraplasmid, a non-viral gene therapy designed for intravesical administration [7] - Mechanism: Combines plasmid DNA with RIG-I agonists and IL-12 to activate both innate and adaptive immune systems [7] - Manufacturing: Utilizes four simple ingredients, allowing for competitive cost of goods and easier handling [8] Clinical Development - LEGEND Program: Focused on BCG-unresponsive NMIBC patients, with 125 patients enrolled in the pivotal cohort [8][10] - Efficacy Data: - 63% complete response (CR) rate at any time for post-amendment patients [11] - 62% CR rate at six months, showing improvement over previous data [11][13] - Tolerability: 42% treatment-related adverse event rate, primarily mild and associated with catheterization [14][15] Competitive Landscape - Comparison with Other Products: Detalimogene's efficacy and tolerability are competitive with other approved products, such as those from J&J [18][19] - Market Positioning: Positioned as a first-line treatment option for busy community urologists, emphasizing ease of use and integration into practice [21][27] Regulatory and Future Outlook - FDA Engagement: Ongoing discussions with the FDA regarding clinical endpoints and potential approval timelines, with a target for filing in the second half of next year and potential approval in 2027 [21] - Financial Position: Company has sufficient cash reserves to support operations through 2027 [21] Additional Insights - Community Urologist Feedback: There is enthusiasm for new products, including J&J's TAR-200, which is expected to integrate well into existing treatment workflows [22][23] - Importance of Durability: While CR at any time is a primary endpoint, durability of response is also critical for treatment decisions, especially for older patients with multiple options [25][26] Conclusion - enGene Holdings is positioned to address significant unmet needs in the NMIBC market with its innovative non-viral gene therapy, which offers a competitive profile in terms of efficacy, tolerability, and ease of use for healthcare providers. The company is on track for potential regulatory approval and is well-prepared to capitalize on emerging market opportunities.
enGene Holdings (NasdaqCM:ENGN) 2025 Conference Transcript