Core Insights - enGene Holdings Inc. reported additional preliminary data from the pivotal cohort of its ongoing Phase 2 LEGEND trial for detalimogene voraplasmid in high-risk, BCG-unresponsive non-muscle invasive bladder cancer patients [1] Group 1: Company Overview - enGene Holdings Inc. is a clinical-stage, non-viral genetic medicines company [1] Group 2: Clinical Trial Information - The LEGEND trial is focused on patients with carcinoma in situ (CIS) with or without concomitant conditions [1]

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]

Core Insights - enGene Holdings Inc. has been recognized by BioSpace as one of the 2026 Best Places to Work in the life sciences industry, highlighting its commitment to a positive work environment [1] Company Recognition - enGene is among 50 U.S. operating employers acknowledged for being desirable workplaces in the life sciences sector [1] - The recognition reflects enGene's dedication to fostering a supportive and engaging workplace culture [1]

Core Viewpoint - enGene Holdings Inc. has appointed Dr. Hussein Sweiti as Chief Medical Officer, effective September 29, 2025, bringing over 15 years of experience in oncology and drug development [1]. Company Summary - enGene Holdings Inc. is a clinical-stage, non-viral genetic medicine company [1]. - The new Chief Medical Officer, Dr. Sweiti, has a background as a surgical oncologist and physician-scientist [1]. - Dr. Sweiti's expertise includes clinical practice, oncology clinical research, global drug development, regulatory submissions, and medical affairs [1].

Company Overview - enGene specializes in nonviral gene therapy, which addresses some of the common shortcomings associated with traditional gene therapies [2] - The company utilizes a unique Special DDX platform to deliver gene therapy through mucosal routes, enhancing its effectiveness [2] Product Features - enGene's technology allows for the delivery of large genetic cargo without limitations, making it versatile for various applications [3] - The products are redoseable, enabling repeated use as necessary, which is a significant advantage in treatment [3] - The nonviral nature of the therapy simplifies manufacturing processes, leading to competitive cost of goods and broader applicability across diseases [3]

Summary of enGene Holdings FY Conference Call Company Overview - **Company**: enGene Holdings (NasdaqCM: ENGN) - **Focus**: Non-viral gene therapy utilizing the Dually Derivatized Oligochitosan (DDX) platform - **Lead Asset**: Detalimogene voraplasmid, targeting non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) resistant to Bacillus Calmette-Guérin (BCG) [2][3] Industry Context - **Bladder Cancer Statistics**: - Sixth most common cancer in the U.S. - Approximately 780,000 patients currently, with 82,000 new cases annually [5] - **Current Treatment Landscape**: - BCG is the standard treatment but is often on backorder, leading to radical cystectomy in many cases [5] Core Points and Arguments - **Efficacy and Tolerability**: - Detalimogene voraplasmid shows a 71% complete response (CR) rate with good tolerability [7][10] - Compared to existing products with CR rates of 20% to 40% [8] - **Ease of Administration**: - No pre-washes, special handling, or co-medications required, making it more suitable for busy community urology practices [9][10] - **Protocol Changes in LEGEND Study**: - Implemented three protocol amendments to enhance efficacy, including stricter enrollment criteria for T1 patients and allowing resection of TA [13][16] - **Regulatory Confidence**: - High confidence in the registrational path, supported by FDA engagement and recent designations [20] Competitive Landscape - **Differentiation from Competitors**: - Detalimogene voraplasmid is non-viral, contrasting with newer products that may require complex handling [11][12] - **Market Positioning**: - Targeting community urologists who may not be tracking new medications closely, focusing on the product's efficacy and ease of use [22] Future Outlook - **Clinical Development**: - Anticipated data updates in Q4 of the current year, with a BLA filing expected in mid-next year and potential approval in 2027 [28] - **Cash Position**: - Over $250 million in cash, sufficient to fund operations into 2027 [28] - **Expansion Beyond Bladder Cancer**: - Potential applications of the DDX platform in other cancers and mucosal delivery systems [26][27] Additional Insights - **AI Integration**: - enGene is exploring AI's potential to enhance drug development and commercialization processes [33][34] - **Regulatory Environment**: - Positive interactions with the FDA, indicating a supportive regulatory landscape for enGene's products [35][36] Conclusion - enGene Holdings is positioned to address significant unmet needs in bladder cancer treatment with its innovative non-viral gene therapy platform, demonstrating strong efficacy, tolerability, and ease of use, while maintaining a solid financial position and regulatory support for future growth.

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]

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]

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]

Core Insights - enGene Holdings Inc. (NASDAQ: ENGN) has shown promising early data, leading to a "Strong Buy" sentiment from analysts [1] Company Analysis - The company is involved in the biotech sector, focusing on clinical trials and innovative therapies [1] - The analyst has a PhD in biochemistry and extensive experience in analyzing biotech companies, indicating a strong foundation for evaluating enGene's potential [1] Industry Context - The biotech industry is characterized by high volatility and significant investment risks, necessitating thorough due diligence by investors [1]