The Company completed enrollment of 125 patients in the pivotal cohort, exceeding its target by 25%. Data from these patients demonstrated a generally favorable tolerability profile: The Company continues to believe that detalimogene's emerging profile supports its potential first line use in patients with high-risk, BCG-unresponsive non-muscle invasive bladder cancer with carcinoma in situ (CIS). BOSTON & MONTREAL--(BUSINESS WIRE)--enGene Holdings Inc. (Nasdaq: ENGN, "enGene†or the "Company†), a clinical ...

Group 1 - enGene Holdings Inc. (NASDAQ:ENGN) has gained over 31% since reporting positive results from its Phase 2 LEGEND trial on November 11 [1] - The company reported a complete response rate of 62% at 6 months for its treatment of high-risk, BCG-unresponsive non-muscle invasive bladder cancer, up from 56% at 3 months among 37 patients [3] - Management is on track for a planned Biologics License Application submission in the second half of 2026 [3] Group 2 - Wall Street analysts maintain a positive outlook with a 12-month price target of $23, indicating more than 190% upside from the current level [2] - David Dai from UBS reiterated a Hold rating with a $10 price target, while Judah Frommer from Morgan Stanley reiterated a Buy rating with an $18 price target [2] - enGene Holdings is a clinical-stage biotechnology company focused on developing non-viral genetic medicines for therapeutic delivery to mucosal tissues and other organs [4]

enGene Holdings (NasdaqCM:ENGN) FY Conference December 02, 2025 12:10 PM ET Company ParticipantsAlexander Nichols - Chief Strategy and Operations OfficerConference Call ParticipantsAshleigh Acker - Biotech AnalystAshleigh AckerWelcome, everyone, to the Piper Sandler Healthcare Conference. My name is Ashleigh Acker. I'm a Biotech Analyst on Allison Bratzel's team here at Piper Sandler. It's my pleasure to introduce our next presenting company, enGene Holdings' Chief Strategy and Operations Officer, Alexander ...

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]

Core Viewpoint - enGene Holdings Inc. has announced the pricing of its underwritten public offering, which includes common shares and pre-funded warrants, indicating a strategic move to raise capital for its operations [1] Group 1: Offering Details - The company is offering 12,558,823 common shares at a public offering price of $8.50 per share [1] - Additionally, pre-funded warrants to purchase 2,735,295 shares are being offered at an offering price of $8.4999 per warrant [1] - The pricing is set before underwriting discounts and commissions, highlighting the financial structure of the offering [1]

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.

Core Insights - The article expresses optimism about enGene Holdings Inc. (ENGN) in the context of bladder cancer research and investment potential [1] Company Analysis - enGene Holdings Inc. is focused on developing therapies for bladder cancer, indicating a specialized niche within the biotech sector [1] - The author has a PhD in biochemistry and extensive experience in analyzing clinical trials and biotech companies, which adds credibility to the insights provided [1] Investment Thesis - The investment thesis for enGene Holdings Inc. is based on its market valuation relative to its potential in the bladder cancer treatment space, suggesting that the company may be undervalued [1]

Core Insights - enGene is discussing preliminary LEGEND data during the conference call [1] Group 1 - The conference call is hosted by enGene's Executive Director of Investor Relations, Lauren Hopfer [1]

Summary of enGene Holdings FY Conference Call Company Overview - **Company**: enGene Holdings (NasdaqCM: ENGN) - **Lead Product Candidate**: datolimogene hayemab - **Focus Area**: Bladder cancer, specifically non-muscle invasive bladder cancer (NMIBC) Key Points and Arguments Market Opportunity - The bladder cancer treatment market is expected to grow significantly due to new FDA guidance and the introduction of new agents, moving away from traditional treatments like BCG and radical cystectomy [4][6] - The target demographic primarily consists of elderly patients with comorbidities who prefer less invasive treatment options [4] Product Value Proposition - **Efficacy**: Datolimogene hayemab shows a competitive emerging efficacy profile, with a reported 62% complete response (CR) rate at six months, which is comparable to other agents in the market [12][20] - **Tolerability**: The product has a low treatment-related adverse event (AE) rate of 42% and a very low treatment interruption rate of 1.8%, indicating a favorable tolerability profile [21][23] - **Ease of Use**: The non-viral gene therapy approach simplifies handling for both doctors and patients, with no pre-washes or post-treatment requirements [5][6] Clinical Trial Updates - Enrollment in the Legend Pivotal cohort was completed with 125 patients, exceeding the target by 25% [11] - Protocol amendments were made to align with standard care and AUA guidelines, which are expected to enhance efficacy [14][16] - The primary endpoint has been aligned with other approved products, focusing on the CR rate at any time [19] Regulatory and Manufacturing Insights - The company is nearing completion of FDA validation runs for its manufacturing process, which utilizes a simple non-viral approach, allowing for cost-effective production [28][29] - The company holds RMAT designation, which may reduce the risk of receiving a Complete Response Letter (CRL) from the FDA [28] Commercialization Strategy - enGene plans to commercialize datolimogene hayemab independently in the U.S. with a manageable sales force of 40-50 representatives [34] - The company is exploring options for commercialization in Europe and plans to partner for markets outside the U.S. [34] Financial Position - enGene reported a cash position of approximately $225 million, which is expected to sustain operations into 2027 [37] Future Directions - The company is studying datolimogene hayemab in various patient cohorts, including naive and BCG-exposed patients, to gather more data on its efficacy across different populations [33] - Long-term data updates and FDA discussions are anticipated in the second half of the next year [26] Additional Important Insights - The competitive landscape for NMIBC treatments is evolving, with new products priced between $200 million to $700 million, indicating a large market potential [31] - The company is conducting extensive market research to shape its go-to-market strategy and understand the competitive environment better [35]

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]