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 [5][9] - The company is focusing on pivotal cohort 1 while also initiating additional cohorts to expand knowledge and data for treating urologists [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 is confident in its manufacturing capabilities, which are crucial given the challenges faced in the industry [15] Company Strategy and Development Direction - The company aims to provide incremental data updates as more patients are enrolled under the new protocol, which is expected to improve the overall profile of the treatment [1][2] - There is a focus on aligning the treatment protocol with standard care to enhance the drug's marketability and acceptance among urologists [9][10] Management's Comments on Operating Environment and Future Outlook - Management expressed excitement about the upcoming data and the potential for the treatment to meet regulatory standards, emphasizing the importance of the new protocol [1][14] - The management highlighted the challenges in the industry related to manufacturing but expressed confidence in their current capabilities and readiness for the BLA submission [15] Other Important Information - The company is actively enrolling patients in three new cohorts to gather more data, which will help in understanding the treatment's efficacy across different patient profiles [17] - The management cautioned against making direct comparisons between different studies due to variations in protocols and patient populations [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 achieved [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? - Management advised caution in comparing CR rates across studies due to differences in protocols and patient heterogeneity [12][13] Question: Can you touch on BLA filing and confidence to be on track? - The company is on track for a BLA submission in the second half of 2026, with many modules already prepared [14][15] Question: What are the new cohorts and their opportunities? - The new cohorts aim to expand knowledge for treating urologists, with a focus on pivotal cohort 1 for generating critical data [17]
enGene(ENGN) - 2025 FY - Earnings Call Transcript