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]
enGene(ENGN) - 2025 FY - Earnings Call Transcript