Summary of enGene Conference Call Company Overview - **Company Name**: enGene - **Ticker**: ENGN - **Focus**: Non-viral genetic medicines, specifically developing a product called detalimogene voraplasmid for non-muscle invasive bladder cancer (NMIBC) [2][3] Core Points and Arguments - **Market Potential**: The NMIBC market is expected to grow significantly, with the introduction of new agents improving patient outcomes and physician practices [3] - **Product Profile**: Detalimogene is characterized as a novel class of gene therapy with a large cargo capacity, allowing for redosing and competitive cost of goods due to its non-viral nature [4][5] - **Patient Demographics**: Over 740,000 bladder cancer patients in the U.S., with 27,500 classified as high-risk NMIBC. The majority of patients are treated in community settings rather than academic centers [5][9] - **Current Treatment Landscape**: Existing treatments like BCG are in short supply and can be toxic at higher doses, leading to high rates of bladder removal surgeries with significant morbidity and mortality [6][7] - **Clinical Development**: enGene plans to provide data updates in the second half of the year, file a Biologics License Application (BLA) by year-end, and expects a potential product launch in 2027 [4][23] Clinical Data and Efficacy - **Pivotal Study**: The pivotal study has enrolled 125 patients, exceeding the target of 100. The primary endpoint is the Complete Response (CR) rate, with a reported 62% CR rate at six months [11][15] - **Tolerability**: Most adverse events are Grade 1 and 2, primarily related to catheterization, with low rates of dose interruptions and discontinuations compared to other agents [17] - **Convenience**: Detalimogene is designed for ease of use, requiring minimal special handling and storage, which is advantageous for community practices [18] Competitive Landscape - **Emerging Competitive Profile**: Detalimogene is positioned as having a competitive efficacy and tolerability profile compared to existing therapies, with a focus on community urologists who prefer products that integrate easily into their practices [19][20] - **Combination Therapy Potential**: The non-viral nature of detalimogene allows for potential combination therapies with other agents, which could enhance treatment efficacy [25] Market Dynamics - **Market Growth**: The NMIBC market is projected to grow from approximately $1 billion to over $20 billion, driven by the adoption of new therapies and changing treatment paradigms [22] - **Economic Impact**: The introduction of detalimogene is expected to benefit community practices economically, improving patient satisfaction and practice revenue through a buy-and-bill model [20][21] Additional Insights - **Pricing Strategy**: enGene's low cost of goods provides a competitive advantage in pricing, allowing flexibility in response to market dynamics [28] - **Future Data**: Anticipated updates on 12-month response data from the LEGEND study are expected in the second half of the year [27] This summary encapsulates the key points discussed during the enGene conference call, highlighting the company's strategic focus, product development, market potential, and competitive positioning in the NMIBC treatment landscape.

Company Overview - enGene Holdings Inc. (NASDAQ:ENGN) is focused on developing non-viral gene therapies for mucosal diseases, particularly in the bladder cancer treatment sector [4] - The company is headquartered in Saint-Laurent, Quebec, and was founded in 1999 [4] Clinical Development - enGene is advancing its lead candidate, detalimogene voraplasmid, into late-stage development for high-risk non-muscle invasive bladder cancer (NMIBC) [3] - A pivotal study is currently underway, with a key data update expected in the second half of 2026 [2] Financial Position - On January 21, enGene expanded its debt facility with Hercules Capital to up to $125 million, which includes $25 million immediately available for refinancing existing debt [3] - The additional financing of up to $100 million is tied to clinical, regulatory, and commercial milestones, enhancing the company's financial flexibility as it transitions to a potential commercial-stage entity [3] Investment Potential - enGene is viewed as a high-risk, high-reward investment opportunity due to its innovative approach and the potential for significant upside in the oncology market [4] - The company's strengthened liquidity and upcoming pivotal data position it favorably within a competitive landscape [4]

Core Insights - enGene Holdings Inc. is positioned strongly at the end of 2025, with completed enrollment in the LEGEND pivotal cohort and a cash position of $342.4 million, extending its financial runway into the second half of 2028 [2][9] - The company aims to file a Biologics License Application (BLA) in the second half of 2026, with potential approval anticipated in 2027 [2][14] Financial Performance - For the full year ended October 31, 2025, total operating expenses were $123.2 million, a significant increase from $62.3 million in 2024, primarily due to higher research and development costs [10][24] - The net loss attributable to common shareholders was approximately $117.3 million, or $2.29 per share, compared to a net loss of $55.1 million, or $1.46 per share, for the previous year [11][25] - As of October 31, 2025, cash, cash equivalents, and marketable securities totaled $202.3 million, with an additional $140.1 million raised from a public offering in November [9][26] Clinical Development Updates - The LEGEND pivotal cohort has completed enrollment of 125 patients, exceeding the target by 25%, and has shown a generally favorable tolerability profile [3][4] - Recent data from the LEGEND trial indicated a 63% complete response (CR) rate at any time, with 56% at 3 months and 62% at 6 months for post-baseline assessments [6][17] - The company is also participating in the FDA's Chemistry, Manufacturing, and Controls (CMC) Development and Readiness Pilot (CDRP) program, which aims to facilitate the development of therapies with compressed clinical timelines [5][16] Corporate Developments - enGene appointed Hussein Sweiti, M.D., MSc, as Chief Medical Officer, bringing over 15 years of experience in oncology clinical research and drug development [8] - The company successfully closed a public offering of common shares and pre-funded warrants, raising $140.1 million in net proceeds [4]

enGene Holdings FY Conference Summary Company Overview - **Company**: enGene Holdings (NasdaqCM: ENGN) - **Industry**: Biotechnology, specifically focusing on non-viral genetic therapies - **Lead Product**: Detalimogene voraplasmid for non-muscle invasive bladder cancer (NMIBC) [4][3] Core Points and Arguments - **Unique Technology Platform**: enGene has developed a non-viral genetic therapy platform that addresses common limitations of traditional gene therapies, such as immunogenicity and manufacturing difficulties [5][4] - **Market Opportunity**: NMIBC is a prevalent disease in the U.S. with approximately 730,000 cases annually, including 25,000 high-risk cases. The management of NMIBC is costly, exceeding $6.5 billion per year [6][7] - **Patient Journey**: Current treatment options for NMIBC are limited, with BCG therapy being in chronic short supply. Patients often progress to chemotherapy or radical cystectomy, which significantly impacts their quality of life [8][7] - **FDA Guidance**: The FDA has streamlined development for therapies targeting carcinoma in situ, emphasizing the need for new treatment options [9][9] - **Community Practice**: 67% of urologists practice in community settings, where ease of use and tolerability of therapies are critical for adoption [10][9] Product Profile - **Detalimogene Voraplasmid**: A non-viral gene therapy designed to stimulate both innate and adaptive immune responses, promoting tumor clearance [13][12] - **Manufacturing Advantage**: The manufacturing process is straightforward, allowing for cost-effective production and ease of use in clinical settings [14][12] - **Clinical Study**: The LEGEND study is a registrational open-label study focusing on BCG unresponsive patients, with a primary endpoint of complete response rate [15][14] Clinical Data - **Response Rates**: The six-month complete response rate is 62%, significantly higher than the 41% rate observed under the old protocol [17][16] - **Durability of Response**: The durability of responses in the new protocol shows promising results, with a favorable tolerability profile compared to other therapies [18][17] - **Competitive Landscape**: Detalimogene's response rates and tolerability compare favorably against existing and developing therapies in the market [19][18] Future Outlook - **Market Positioning**: enGene aims for detalimogene to be a first-line therapy for high-risk NMIBC, particularly in community settings, with the potential for patients to progress to more advanced therapies if needed [20][19] - **Financial Position**: Recent fundraising has extended the company's cash runway into 2028, providing a solid foundation for ongoing development [21][20] Additional Important Information - **Regulatory Designations**: enGene has received CDRP designation and RMAT designation from the FDA, which may facilitate the review process for their therapies [14][13] - **Team Experience**: The company boasts a strong team with prior experience in developing similar products, enhancing confidence in their ability to bring detalimogene to market [21][20]

Summary of enGene Holdings Conference Call Company Overview - **Company**: enGene Holdings (NasdaqCM: ENGN) - **Focus**: Non-viral gene therapy for various diseases, specifically targeting non-muscle invasive bladder cancer (NMIBC) with its lead asset, detalimogene voraplasmid [1][2] Core Product Insights - **Lead Asset**: Detalimogene voraplasmid - **Indication**: Non-muscle invasive bladder cancer (NMIBC) - **Market Potential**: Projected to grow to over $20 billion due to a shift from an incident-based to a prevalent-based disease model [2] - **Efficacy and Tolerability**: Demonstrated promising efficacy and tolerability, with a focus on ease of use for both community and academic urologists [2][3] Market Dynamics - **Current NMIBC Statistics**: Approximately 730,000 cases in the U.S., with 65,000 new cases annually [3] - **Treatment Landscape**: Current management involves BCG therapy, which has been in chronic backorder and presents challenges in administration [4][5] - **Unmet Needs**: Significant unmet needs in the NMIBC market, particularly in community settings where most urologists practice [5][6] Competitive Advantages - **Non-Viral Approach**: Overcomes limitations of viral gene therapies, such as genetic payload size, redosing issues, and high manufacturing costs [3] - **Ease of Administration**: Detalimogene can be administered intravesically with minimal logistical requirements, making it suitable for community practices [12][14] - **Cost-Effectiveness**: Competitive cost of goods due to simple manufacturing processes [12][18] Clinical Development - **Pivotal Program**: Fully enrolled pivotal program for NMIBC patients unresponsive to BCG, with 125 patients enrolled [9] - **Efficacy Data**: Six-month complete response (CR) rate of 62%, showing improvement from pre-protocol rates [11][13] - **Tolerability Profile**: Mostly grade 1 or 2 adverse events, with low rates of dose interruptions (1.6%) and discontinuations (0.8%) [11][14] Future Outlook - **Regulatory Plans**: Anticipated filing with the FDA in the second half of next year, with potential approval in 2027 [2][19] - **Market Growth**: NMIBC market currently valued at approximately $2 billion, expected to grow significantly [18] - **Strategic Positioning**: Detalimogene is positioned to integrate into community urologist practices without disrupting existing workflows, offering a more patient-friendly treatment option [18][19] Conclusion - **Company's Financial Health**: Well-capitalized with over $100 million raised recently, providing a strong foundation for future growth and development [17][18] - **Commitment to Innovation**: enGene is focused on delivering effective and accessible treatments for NMIBC, aiming to improve patient outcomes and streamline urologist practices [16][19]

Summary of enGene Holdings Conference Call Company Overview - **Company**: enGene Holdings (NasdaqCM: ENGN) - **Focus**: Development of non-viral gene therapies for bladder cancer, specifically targeting non-muscle invasive bladder cancer (NMIBC) Industry Context - **Disease Prevalence**: NMIBC affects approximately 730,000 patients in the US, with about 85,000 new cases annually [1][2] - **Market Size**: The management of NMIBC is estimated to cost the healthcare system $6.5 billion per year [3] - **Current Treatment Landscape**: Traditional treatments include BCG therapy, IV chemotherapy, and radical cystectomy, which are often associated with high morbidity and mortality [4][5] Core Product Insights - **Product**: Detalimogene voraplasmid, a non-viral gene therapy designed for intravesical administration [7] - **Mechanism**: Combines plasmid DNA with RIG-I agonists and IL-12 to activate both innate and adaptive immune systems [7] - **Manufacturing**: Utilizes four simple ingredients, allowing for competitive cost of goods and easier handling [8] Clinical Development - **LEGEND Program**: Focused on BCG-unresponsive NMIBC patients, with 125 patients enrolled in the pivotal cohort [8][10] - **Efficacy Data**: - 63% complete response (CR) rate at any time for post-amendment patients [11] - 62% CR rate at six months, showing improvement over previous data [11][13] - **Tolerability**: 42% treatment-related adverse event rate, primarily mild and associated with catheterization [14][15] Competitive Landscape - **Comparison with Other Products**: Detalimogene's efficacy and tolerability are competitive with other approved products, such as those from J&J [18][19] - **Market Positioning**: Positioned as a first-line treatment option for busy community urologists, emphasizing ease of use and integration into practice [21][27] Regulatory and Future Outlook - **FDA Engagement**: Ongoing discussions with the FDA regarding clinical endpoints and potential approval timelines, with a target for filing in the second half of next year and potential approval in 2027 [21] - **Financial Position**: Company has sufficient cash reserves to support operations through 2027 [21] Additional Insights - **Community Urologist Feedback**: There is enthusiasm for new products, including J&J's TAR-200, which is expected to integrate well into existing treatment workflows [22][23] - **Importance of Durability**: While CR at any time is a primary endpoint, durability of response is also critical for treatment decisions, especially for older patients with multiple options [25][26] Conclusion - enGene Holdings is positioned to address significant unmet needs in the NMIBC market with its innovative non-viral gene therapy, which offers a competitive profile in terms of efficacy, tolerability, and ease of use for healthcare providers. The company is on track for potential regulatory approval and is well-prepared to capitalize on emerging market opportunities.

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]

Core Viewpoint - enGene Holdings Inc. is experiencing a significant increase in stock price following the release of preliminary data from its Phase 2 LEGEND trial for detalimogene voraplasmid in high-risk bladder cancer patients [2][6]. Group 1: Trial Data and Results - The LEGEND trial included 99 patients with BCG-unresponsive non-muscle invasive bladder cancer (NMIBC) [2]. - Preliminary analysis showed a 63% complete response (CR) rate at any time for 62 patients, with a 56% CR rate at 3 months and a 62% CR rate at 6 months for 37 patients [8]. - The company exceeded its enrollment target by 25%, completing enrollment of 125 patients in the pivotal cohort [3][4]. Group 2: Future Plans and Expectations - The company plans to file a Biologics License Application (BLA) in the second half of 2026, with potential FDA approval expected in 2027 [5][6]. - enGene anticipates providing a data update on the LEGEND trial's pivotal cohort in the second half of 2026 [5]. Group 3: Market Performance - enGene Holdings shares rose by 64.73% to $9.90, reaching a new 52-week high [6].

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]