Engene Therapeutics Inc. · NASDAQ:ENGN

Engene Therapeutics — American Urological Association Annual Meeting 2026

12 slides from Engene Therapeutics's conference presentation, May 2026

  1. Slide 1: Pivotal Phase 2 Interim Results of Detalimogene Voraplasmid

    A Non-Viral Intravesical Gene Therapy for BCG-Unresponsive NMIBC With CIS, With or Without Papillary Disease: Pivotal Phase 2 Interim Results of Detalimogene Voraplasmid Ashish M. Kamat,1 Mark Tyson,2 Ricardo Rendon,3 Félix Guerrero-Ramos,4 Jen-Jane Liu,5 Shreyas Joshi,6 Paras H. Shah,7 Grégoire Rob

  2. Slide 2: Ashish M. Kamat, MD: Disclosures

    Ashish M. Kamat, MD: Disclosures Dr Kamat discloses the following: Speaker for a pharmaceutical company: None Support from a pharmaceutical company for participation in a national or international congress: None Consultant/Scientific Advisory Board: Astra Zeneca; CG Oncology; enGene Therapeutics Inc

  3. Slide 3: Patients With BCG-Unresponsive NMIBC Have a Persistent Need for Novel Bladder-Sparing Treatment Options

    Patients With BCG-Unresponsive NMIBC Have a Persistent Need for Novel Bladder-Sparing Treatment Options • Global burden: ~656,000 new bladder cancer cases worldwide in 2025, including ~87,000 in the United States1,2 • Despite recent approvals, there is still a significant need for novel therapies th

  4. Slide 4: As a Non-Viral Gene Therapy, Investigational Detalimogene Is Easy to Administer

    As a Non-Viral Gene Therapy, Investigational Detalimogene Is Easy to Administer and Overcomes Several Limitations of Viral Therapies PATIENTS PRACTICE Fewer doses per Storage in a VA1 (RIG-I agonist dsRNA) 12-week cycle standard freezer Administered by No thaw or pre- medical assistants IL-12 wash s

  5. Slide 5: LEGEND: A Single-Arm, Open-Label Study of Detalimogene In High-Risk NMIBC

    LEGEND: A Single-Arm, Open-Label Study of Detalimogene In High-Risk NMIBC PHASE 2: ONGOING Pivotal Cohort Treatment Schedule BCG-unresponsive NMIBC with CISa Detalimogene 0.8 mg/mL (50 mL) IVE via catheter with 60-min dwell timeb (n=125) • CIS ± HG Ta/T1 with persistent or recurrent Re- Year 1 (indu

  6. Slide 6: LEGEND Phase 2 Cohort 1: Baseline Demographics and High-Risk Disease Characteristics

    LEGEND Phase 2 Cohort 1: Baseline Demographics and High-Risk Disease Characteristicsa Pivotal cohort 1 Pivotal cohort 1 Characteristics Characteristics (N=125) (N=125) Tumor stage, n (%) Age, years CIS with Ta/T1 disease 49 (39.2%) Median (range) 71.0 (35.0-90.0) CIS only 76 (60.8%) Reason for no cy

  7. Slide 7: LEGEND Phase 2 Cohort 1: CONSORT Diagram

    LEGEND Phase 2 Cohort 1: CONSORT Diagrama Enrolled 125 Participants 1 Discontinued 1 no disease evaluation (censoredc) 3 monthsb 124 Completed Evaluation 19 Discontinued • 18 non-CR • 1 CR (censoredc) 103 Completed Evaluation 6 monthsb • 2 pending evaluation at 6 months 51 Discontinued • 51 non-CR 9

  8. Slide 8: LEGEND Phase 2 Cohort 1: Complete Response Rates

    LEGEND Phase 2 Cohort 1: Complete Response Ratesa 100% CR Landmark CR Rate, % (95% CI) CR by KM (Est %) Any Time Complete 6 month 43.0% 39.4% Response (n=121) (CI: 34.0%, 52.3%) 80% Overall CR Rate, % 54% 12 month (n=98) 13.3% (CI: 7.3%, 21.6%) 24.5% (CI: 44.9%, 63.0%) 60% • Median follow-up was 5.5

  9. Slide 9: LEGEND Phase 2 Cohort 1: Preliminary Durability in Complete Responders

    LEGEND Phase 2 Cohort 1: Preliminary Durability in Complete Respondersa • Kaplan-Meier 12-month CR: 25% (95% CI 11-41%) • 46.3% of responders have an ongoing CRb • Of the responders: • 84% remain in CR at the 9-month assessment Pre-assessment Complete response • 59% remain in CR at the 12- Non-compl

  10. Slide 10: Detalimogene Demonstrates Favorable Safety and Tolerability Profile

    Detalimogene Demonstrates Favorable Safety and Tolerability Profile TRAEs Were Generally Mild and Reversible, Consistent With Catheterizationa Most treatment-related adverse events (TRAEs) were localized, brief, low-grade events Pivotal cohort 1 • 69 patients (55.2%) had any grade TRAEs Participants

  11. Slide 11: Conclusions

    Conclusionsa Detalimogene is a next-generation non-viral gene Detalimogene showed encouraging activity in therapy in development for HR NMIBC, designed to: heavily pre-treated participants with BCG- unresponsive NMIBC • Reduce patient burden (fewer visits, procedures, pre- or post-treatment precauti

  12. Slide 12: Ongoing Phase 2 targets enrollment of n≈350 patients across all cohorts

    Ongoing phase 2 targets United Kingdom 1 site enrollment of n≈350 patients across all cohorts from sites Germany in the USA, Canada, Europe, 6 sites and the Asia-Pacific Region Canada Italy ClinicalTrials.gov NCT04752722 6 sites 6 sites France 7 sites United States Spain Republic of Korea 52 sites 1

enGene Therapeutics — American Urological Association Annual Meeting 2026 on 6ix