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 [3][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][15] 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, enhancing the profile of detalimogene voraplasmid [3][5] - The company is focusing on pivotal cohort 1 while also initiating additional cohorts to expand knowledge and data for treating urologists [19] 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 [15][16] - The company is confident in its manufacturing capabilities, which are crucial given the challenges faced in the industry [16] Company Strategy and Development Direction - The company aims to provide incremental data updates to enhance the understanding of detalimogene voraplasmid's efficacy and safety profile [3][9] - There is a strong emphasis on aligning the study protocols with standard care to improve the treatment profile and increase the likelihood of regulatory approval [10][15] Management's Comments on Operating Environment and Future Outlook - Management expressed excitement about reaching target enrollment and the potential for sharing more data in the future, which is expected to strengthen the company's position in the market [10][21] - The management highlighted the importance of understanding the heterogeneous nature of NMIBC patients when interpreting efficacy data [14] Other Important Information - The company has initiated three new cohorts to gather more data, focusing on different patient profiles, which will help in expanding the knowledge base for the treatment [19] - The management is cautious about making cross-study comparisons due to the small sample sizes and variability in patient characteristics [13][14] Q&A Session Summary Question: How many patients from the new protocol? - The vast majority of patients will be under the new protocol, with a target enrollment of 100 patients, likely to exceed this number [3] Question: Could you discuss the CR rate expectations? - The FDA has approved agents with CR rates between 20% to 40%, while the company reported a 71% CR rate, indicating confidence in the drug's efficacy [9] Question: What is the timeline for BLA filing? - The company updated its guidance for BLA submission to the second half of 2026, with strong regulatory and clinical teams ready to proceed [15][16] Question: Can you elaborate on the new cohorts? - The new cohorts aim to expand knowledge for treating urologists, with a focus on pivotal cohort 1 being the highest priority [19]
enGene(ENGN) - 2025 FY - Earnings Call Transcript