BOSTON & MONTREAL--(BUSINESS WIRE)--enGene Holdings Inc. (Nasdaq: ENGN, "enGene†or the "Company†), a clinical-stage, non-viral genetic medicine company, today announced that Hussein Sweiti, M.D., MSc, was appointed Chief Medical Officer, effective September 29, 2025. Dr. Sweiti is a surgical oncologist and physician-scientist with more than 15 years of experience spanning clinical practice, oncology clinical research, global drug development, regulatory submissions, and medical affairs. He mos. ...

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]

Summary of NGIN Conference Call Company Overview - NGIN is a non-viral genetic medicines company focused on developing treatments for non-muscle invasive bladder cancer (NMIBC) [1] Industry Insights - The management of NMIBC is undergoing a significant transformation, moving from an incident-based market to a prevalent-based market, similar to the evolution seen in the multiple myeloma market [2][3] - The NMIBC market is currently valued at approximately $1 billion and is expected to grow significantly with the introduction of new therapies [2][3] Product Development - NGIN is developing a product called Dental Imaging, specifically designed for urologists, which combines efficacy, tolerability, and ease of use [3][12] - The company anticipates providing an update on pivotal cohort data later this year, with top-line data and a BLA filing expected in mid-2026, followed by potential approval and launch in 2027 [4][45] Market Dynamics - NMIBC is a highly prevalent disease in the U.S., with around 730,000 patients and 65,000 new cases annually [5] - Bladder cancer is the sixth most common cancer and one of the most costly to manage [6][7] - The majority of NMIBC patients are managed by community urologists, who currently face challenges with existing treatment options, including a shortage of BCG [8][9] Treatment Challenges - Current treatments, including BCG and other agents, have limitations such as availability issues, complex administration, and significant side effects [9][10][11] - Dental Imaging aims to address these challenges by offering a well-tolerated treatment option that does not require significant changes to existing urology practices [12][13] Clinical Efficacy and Tolerability - Dental Imaging has demonstrated a 71% complete response (CR) rate at any time, with a 67% CR rate at three months and 47% at six months [23] - The treatment has a favorable tolerability profile, with adverse events primarily being grade two or below [22][23] Regulatory Landscape - NGIN is in ongoing dialogue with the FDA regarding approval pathways, with a focus on addressing the high unmet medical need in the NMIBC patient population [31][32] Competitive Advantage - Dental Imaging is positioned as a best-in-class product for the urology community, offering competitive efficacy, tolerability, and ease of use compared to other agents [32][41] - The product's administration process is designed to be less burdensome for both patients and urologists, facilitating its integration into busy clinical practices [38][39] Future Outlook - NGIN is optimistic about the future of Dental Imaging and its potential to significantly impact the NMIBC treatment landscape [44] - The company has sufficient capital to support its milestones through 2027, indicating a strong financial position for upcoming developments [45]

Summary of enGene Holdings (ENGN) Conference Call Company Overview - **Company Name**: enGene Holdings (ENGN) - **Headquarters**: Montreal, Canada; Corporate Offices in Boston, USA - **Industry**: Biotechnology, specifically focused on genetic medicines - **Ticker Symbol**: Listed on NASDAQ Core Points and Arguments 1. **Market Opportunity**: The company is developing ditalimogene voraplasmin for non-muscle invasive bladder cancer (NMIBC), a market expected to grow significantly due to the introduction of new agents, similar to the multiple myeloma market which expanded from $1 billion to over $20 billion with new treatments [6][5][4] 2. **Clinical Development Timeline**: Enrollment for the pivotal study will be completed this year, with top-line data expected next year and a BLA filing anticipated for 2027 [7][9] 3. **Product Advantages**: Ditalimogene voraplasmin is a genetic medicine that has shown good efficacy (71% complete response rate) and excellent tolerability, with a unique formulation that allows for easy handling and storage [8][18][32] 4. **Cash Position**: The company has a strong cash position of approximately $275 million, which is expected to last until 2027, providing a buffer against financial risks [9] 5. **Unmet Medical Need**: NMIBC has a significant unmet medical need, with current treatments like BCG in short supply and associated with severe side effects [15][16][18] 6. **Regulatory Clarity**: The company believes it has a clear regulatory path, with existing clinical data and a well-characterized manufacturing process, which is crucial for biotech success [50][51] Additional Important Content 1. **Patient Demographics**: NMIBC predominantly affects older males, with an average age in their mid-seventies, and about 730,000 individuals in the U.S. are affected [11][30] 2. **Treatment Landscape**: Current treatments are limited, with BCG being the first-line therapy but facing supply issues. Other approved agents have limitations in efficacy and adverse events [16][17][18] 3. **Manufacturing Advantages**: enGene has already established a scalable manufacturing process, which is expected to provide a commercial advantage due to lower costs [21][24] 4. **Development Program**: The Legend program includes multiple cohorts, targeting BCG-unresponsive patients and exploring other applications for the platform [25][26] 5. **Efficacy Comparison**: The company anticipates that protocol changes will improve efficacy rates, which are currently competitive with existing treatments [39][40][41] 6. **Team Experience**: The management team has extensive experience in biotech and pharmaceuticals, enhancing the company's credibility and operational capability [49] This summary encapsulates the key points discussed during the enGene Holdings conference call, highlighting the company's strategic positioning within the biotechnology sector and its focus on addressing significant medical needs in bladder cancer treatment.

Company Overview - enGene is a small Canadian company focused on developing genetic medicines specifically for bladder cancer [1] - The company entered the market through a SPAC (Special Purpose Acquisition Company) and has established a platform for its operations [1] Industry Context - The genetic medicine sector is gaining traction, particularly in oncology, as companies explore innovative treatments for various types of cancer [1]