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]

Market Overview - U.S. stocks showed mixed performance with the Dow Jones index increasing by 0.64% to 47,673.48, while the NASDAQ decreased by 0.79% to 23,340.59 and the S&P 500 fell by 0.23% to 6,817.03 [1] - Energy shares experienced a rise of 1.2%, while information technology stocks saw a decline of 0.9% [1] Company Performance - RealReal Inc (NASDAQ:REAL) shares surged after reporting better-than-expected third-quarter results and providing fourth-quarter sales guidance above estimates, along with an increase in FY25 sales guidance [2] - enGene Holdings Inc (NASDAQ:ENGN) shares increased by 72% to $10.34 following positive Phase 2 LEGEND study data showing a 63% complete response rate [9] - Energy Vault Holdings Inc (NYSE:NRGV) shares rose by 30% to $4.62 after releasing third-quarter results [9] - Surmodics Inc (NASDAQ:SRDX) shares gained 49% to $40.80 after a court denied the FTC's attempt to block GTCR's acquisition [9] - Vor Biopharma Inc (NASDAQ:VOR) shares dropped by 50% to $9.32 due to the announcement of a $100 million public offering [9] - Salarius Pharmaceuticals Inc (NASDAQ:SLRX) shares fell by 50% to $1.00 after announcing a $7 million underwritten public offering [9] - Outset Medical Inc (NASDAQ:OM) shares decreased by 50% to $6.07 following worse-than-expected third-quarter results and a cut in FY25 sales guidance [9] Commodity Market - Oil prices increased by 1.5% to $61.00, while gold rose by 0.3% to $4,134.70 [5] - Silver traded up by 0.8% to $50.730, whereas copper fell by 0.4% to $5.0845 [5] European Market - European shares showed positive movement with the eurozone's STOXX 600 rising by 1.05%, Spain's IBEX 35 Index gaining 1.05%, and London's FTSE 100 increasing by 0.94% [6] Asian Market - Asian markets closed mixed, with Japan's Nikkei 225 down by 0.14%, Hong Kong's Hang Seng up by 0.18%, China's Shanghai Composite down by 0.39%, and India's BSE Sensex up by 0.40% [7]

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].

Summary of enGene Holdings Conference Call on DETALIMGENE Company Overview - **Company**: enGene Holdings (NasdaqCM: ENGN) - **Focus**: Development of DETALIMGENE, a non-viral gene therapy for high-risk BCG unresponsive non-muscle invasive bladder cancer (NMIBC) Key Points from the Conference Call Industry Context - **Target Disease**: High-risk BCG unresponsive NMIBC, particularly carcinoma in situ (CIS) - **Current Treatment Landscape**: Limited options for patients who progress to BCG unresponsive disease, with radical cystectomy being a last resort due to high morbidity and mortality rates [doc id='22'][doc id='23'] Clinical Trial Updates - **Enrollment**: Completion of enrollment in the pivotal cohort with 125 patients, exceeding the original target by 25% due to investigator demand [doc id='3'][doc id='4'] - **Protocol Amendments**: Significant changes made to align the trial with standard care, including re-resection requirements for T1 lesions and biopsy confirmations for persistent CIS [doc id='8'][doc id='9'] - **Efficacy Data**: - Six-month complete response (CR) rate increased to 62% post-amendment, compared to previous rates of 47% [doc id='4'][doc id='12'] - Anytime CR rate reported at 63% [doc id='10][doc id='14'] - High-risk patient characteristics noted, with 42% having concurrent papillary disease [doc id='10][doc id='41'] Safety and Tolerability - **Adverse Events**: 42% of patients experienced treatment-related adverse events, mostly mild (grade one or two) [doc id='17'] - **Discontinuation Rates**: Very low treatment discontinuation (0.8%) and interruption (1.6%) rates, indicating a favorable safety profile [doc id='17] Competitive Positioning - **Differentiation**: DETALIMGENE is positioned as a potentially best-in-class therapy due to its ease of use, tolerability, and competitive efficacy [doc id='5][doc id='30] - **Market Readiness**: Anticipation of high uptake among providers due to its non-viral nature and reduced logistical burdens compared to existing therapies [doc id='38][doc id='39] Future Outlook - **Regulatory Engagement**: Plans to engage with the FDA regarding the statistical analysis plan (SAP) and patient inclusion criteria for final analysis [doc id='15][doc id='36] - **Long-term Data**: Expectation of 12-month data availability in the second half of 2026, with a BLA filing planned for the same period [doc id='30][doc id='29] Insights from Clinical Experts - **Real-world Management**: Insights shared by Dr. Suzanne Merrill on the challenges of managing NMIBC and the importance of bladder-sparing therapies [doc id='20][doc id='23] - **Treatment Selection**: Emphasis on the need for individualized treatment strategies based on efficacy, safety, and logistical considerations [doc id='24][doc id='45] Conclusion - **Overall Sentiment**: Positive outlook on DETALIMGENE's potential to provide a new treatment option for NMIBC patients, with a focus on improving patient quality of life and reducing the burden on healthcare providers [doc id='30][doc id='27]

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]