Non-Viral Genetic Medicine
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enGene (NasdaqCM:ENGN) Earnings Call Presentation
2026-03-09 11:00
Non-Viral Genetic Medicine Corporate Presentation Disclaimers Cautionary Statement Regarding Forward-Looking Statements The lead program described herein is an investigational drug therapy that has not been subject to testing designed to demonstrate that the therapy is effective in humans or to provide a basis to predict in advance whether an adequate level of efficacy in humans will be demonstrated in further testing. Although deemed sufficient to permit further testing, the limited, early Phase 1 testing ...
enGene Holdings (NasdaqCM:ENGN) FY Earnings Call Presentation
2025-11-11 17:30
LEGEND Pivotal Cohort Protocol Amendment - The current LEGEND protocol includes changes such as performing a 2nd resection at the lesion site and restaging, which aligns with the standard of care and AUA Guidelines[8] - Enrollment is complete with 125 patients in the pivotal cohort, with 94 patients (75%) enrolled post-amendment and 31 patients (25%) pre-amendment[10, 11] Efficacy Data - Post-protocol amendment, the Intent-to-Treat (ITT) population (N=62) showed a Complete Response (CR) rate of 63% at any time, 56% at 3 months, and 62% at 6 months[14] - Pre-protocol amendment, the ITT population (N=31) showed a CR rate of 55% at any time, 55% at 3 months, and 41% at 6 months[18] - The 6-month CR rate for detalimogene post-amendment is 62%, which is trending competitively with novel products in the industry[25] Tolerability and Safety - In the entire Cohort 1 (n=125), 42.4% of patients experienced at least one Treatment-Related Adverse Event (TRAE), with zero discontinuations due to adverse events[30] - Detalimogene is trending towards best-in-class tolerability compared to other investigational agents and approved products, with a serious TRAE rate of 1.6%[31] Trial Progress and Enrollment - Enrollment rate was heavily skewed to the last 10 months, delaying insights into long-term efficacy[28, 35] - Additional cohorts are in progress, including BCG-Naïve, BCG-Exposed, and Papillary-Only NMIBC, with varying enrollment statuses[33]
enGene Holdings (NasdaqCM:ENGN) Earnings Call Presentation
2025-11-11 12:00
Detalimogene Voraplasmid & Market Opportunity - The Non-Muscle Invasive Bladder Cancer (NMIBC) market is forecasted to be greater than $20 billion [11] - NMIBC represents 75-80% of bladder cancer diagnoses [14] - Approximately 25,000 US patients per year are diagnosed with High-Risk NMIBC [15] Clinical Trial & Efficacy - The LEGEND study is a global, single-arm, open-label study with a target enrollment of 125 patients with BCG-Unresponsive High-risk NMIBC with CIS [49] - In the post-protocol amendment group (N=62), the Any Time Complete Response (CR) rate was 63% (CI: 51-74) [55, 58] - In the pre-protocol amendment group (N=31), the Any Time Complete Response (CR) rate was 55% (CI: 38-71) [59] Safety & Tolerability - In the Cohort 1 study (n=125), 42.4% of patients experienced at least one Treatment-Related Adverse Event (TRAE) [62] - The most common TRAEs reported in >10% of patients were fatigue (16.8%), dysuria (12.0%), bladder spasm (10.4%), micturition urgency (10.4%), and pollakiuria (10.4%) [62] - Only 1.6% of patients experienced TRAEs leading to dose interruptions, and 0.8% led to dose discontinuations [62] Operational & Financial - Buy+Bill has grown from 5% of revenue in 2008 to 15-20% in 2025 [32] - The company projects its current capital to provide runway into 2027 [78]