Summary of enGene Holdings Conference Call on DETALIMGENE Company Overview - Company: enGene Holdings (NasdaqCM: ENGN) - Focus: Development of DETALIMGENE, a non-viral gene therapy for high-risk BCG unresponsive non-muscle invasive bladder cancer (NMIBC) Key Points from the Conference Call Industry Context - Target Disease: High-risk BCG unresponsive NMIBC, particularly carcinoma in situ (CIS) - Current Treatment Landscape: Limited options for patients who progress to BCG unresponsive disease, with radical cystectomy being a last resort due to high morbidity and mortality rates [doc id='22'][doc id='23'] Clinical Trial Updates - Enrollment: Completion of enrollment in the pivotal cohort with 125 patients, exceeding the original target by 25% due to investigator demand [doc id='3'][doc id='4'] - Protocol Amendments: Significant changes made to align the trial with standard care, including re-resection requirements for T1 lesions and biopsy confirmations for persistent CIS [doc id='8'][doc id='9'] - Efficacy Data: - Six-month complete response (CR) rate increased to 62% post-amendment, compared to previous rates of 47% [doc id='4'][doc id='12'] - Anytime CR rate reported at 63% [doc id='10][doc id='14'] - High-risk patient characteristics noted, with 42% having concurrent papillary disease [doc id='10][doc id='41'] Safety and Tolerability - Adverse Events: 42% of patients experienced treatment-related adverse events, mostly mild (grade one or two) [doc id='17'] - Discontinuation Rates: Very low treatment discontinuation (0.8%) and interruption (1.6%) rates, indicating a favorable safety profile [doc id='17] Competitive Positioning - Differentiation: DETALIMGENE is positioned as a potentially best-in-class therapy due to its ease of use, tolerability, and competitive efficacy [doc id='5][doc id='30] - Market Readiness: Anticipation of high uptake among providers due to its non-viral nature and reduced logistical burdens compared to existing therapies [doc id='38][doc id='39] Future Outlook - Regulatory Engagement: Plans to engage with the FDA regarding the statistical analysis plan (SAP) and patient inclusion criteria for final analysis [doc id='15][doc id='36] - Long-term Data: Expectation of 12-month data availability in the second half of 2026, with a BLA filing planned for the same period [doc id='30][doc id='29] Insights from Clinical Experts - Real-world Management: Insights shared by Dr. Suzanne Merrill on the challenges of managing NMIBC and the importance of bladder-sparing therapies [doc id='20][doc id='23] - Treatment Selection: Emphasis on the need for individualized treatment strategies based on efficacy, safety, and logistical considerations [doc id='24][doc id='45] Conclusion - Overall Sentiment: Positive outlook on DETALIMGENE's potential to provide a new treatment option for NMIBC patients, with a focus on improving patient quality of life and reducing the burden on healthcare providers [doc id='30][doc id='27]
enGene Holdings (NasdaqCM:ENGN) Update / Briefing Transcript