Summary of enGene Holdings FY Conference Call Company Overview - **Company**: enGene Holdings (NasdaqCM: ENGN) - **Lead Product Candidate**: datolimogene hayemab - **Focus Area**: Bladder cancer, specifically non-muscle invasive bladder cancer (NMIBC) Key Points and Arguments Market Opportunity - The bladder cancer treatment market is expected to grow significantly due to new FDA guidance and the introduction of new agents, moving away from traditional treatments like BCG and radical cystectomy [4][6] - The target demographic primarily consists of elderly patients with comorbidities who prefer less invasive treatment options [4] Product Value Proposition - **Efficacy**: Datolimogene hayemab shows a competitive emerging efficacy profile, with a reported 62% complete response (CR) rate at six months, which is comparable to other agents in the market [12][20] - **Tolerability**: The product has a low treatment-related adverse event (AE) rate of 42% and a very low treatment interruption rate of 1.8%, indicating a favorable tolerability profile [21][23] - **Ease of Use**: The non-viral gene therapy approach simplifies handling for both doctors and patients, with no pre-washes or post-treatment requirements [5][6] Clinical Trial Updates - Enrollment in the Legend Pivotal cohort was completed with 125 patients, exceeding the target by 25% [11] - Protocol amendments were made to align with standard care and AUA guidelines, which are expected to enhance efficacy [14][16] - The primary endpoint has been aligned with other approved products, focusing on the CR rate at any time [19] Regulatory and Manufacturing Insights - The company is nearing completion of FDA validation runs for its manufacturing process, which utilizes a simple non-viral approach, allowing for cost-effective production [28][29] - The company holds RMAT designation, which may reduce the risk of receiving a Complete Response Letter (CRL) from the FDA [28] Commercialization Strategy - enGene plans to commercialize datolimogene hayemab independently in the U.S. with a manageable sales force of 40-50 representatives [34] - The company is exploring options for commercialization in Europe and plans to partner for markets outside the U.S. [34] Financial Position - enGene reported a cash position of approximately $225 million, which is expected to sustain operations into 2027 [37] Future Directions - The company is studying datolimogene hayemab in various patient cohorts, including naive and BCG-exposed patients, to gather more data on its efficacy across different populations [33] - Long-term data updates and FDA discussions are anticipated in the second half of the next year [26] Additional Important Insights - The competitive landscape for NMIBC treatments is evolving, with new products priced between $200 million to $700 million, indicating a large market potential [31] - The company is conducting extensive market research to shape its go-to-market strategy and understand the competitive environment better [35]

Financial Data and Key Metrics Changes - The company reported a complete response (CR) rate of 71% in the September 2024 update, indicating a strong efficacy profile for detalimogene voraplasmid [8][9] - The target enrollment for the study has been reached at 100 patients, with expectations to exceed this number, which is a significant increase from the previous cohort of 21 patients [3][5] Business Line Data and Key Metrics Changes - The majority of patients enrolled in the study will be under the new protocol, which aligns with standard care practices, potentially enhancing the profile of detalimogene voraplasmid [3][5] - The company is focused on generating data from the pivotal cohort (cohort 1) while also initiating additional cohorts to expand knowledge in the community [17] Market Data and Key Metrics Changes - The company is preparing for a Biologics License Application (BLA) submission in the second half of 2026, indicating a strategic timeline for regulatory approval [14][15] - The company has noted that the manufacturing process is already at scale, which is a competitive advantage in an industry facing manufacturing challenges [15] Company Strategy and Development Direction - The company aims to provide as much data as possible to treating urologists, indicating a commitment to transparency and education within the medical community [17] - The strategic focus is on aligning the protocol with standard care to improve the treatment profile and increase the likelihood of regulatory approval [9][14] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the efficacy of detalimogene voraplasmid, especially after protocol changes that align with standard care [9][10] - The management team is optimistic about the upcoming data releases and the potential for a successful BLA filing, highlighting the strength of their regulatory and clinical teams [14][15] Other Important Information - The company is cautious about making cross-study comparisons due to the small sample sizes and variability in patient populations across studies [12][13] - The management emphasized the importance of understanding the heterogeneity of patients in clinical trials, which can affect treatment outcomes [12][13] Q&A Session Summary Question: How many patients from the new protocol? - The majority of patients will be under the new protocol, with a target enrollment of 100 patients, likely to exceed this number [3][5] Question: Could you talk about the bar for the CR rate? - The FDA has approved agents with CR rates between 20% to 40%, while the company reported a 71% CR rate, indicating strong efficacy [8][9] Question: What are the caveats for cross-file comparisons? - The management cautioned against comparisons due to differences in study protocols and patient heterogeneity, emphasizing the need for careful interpretation of CR rates [12][13] Question: Can you touch on BLA filing and confidence? - The company is on track for a BLA submission in the second half of 2026, with many modules already prepared, indicating strong readiness [14][15] Question: What are the new cohorts and their opportunities? - The company initiated three new cohorts to expand knowledge, focusing primarily on the pivotal cohort while also enrolling patients in the others [17]