Engene Therapeutics Inc. · NASDAQ:ENGN
Engene Therapeutics — Investor Presentation (July 2026)
35 slides from Engene Therapeutics's investor presentation, Jul 17, 2026
Slide 1: Corporate Presentation: Non-Viral Genetic Medicine
Corporate Presentation Non-Viral Genetic Medicine
Slide 2: Disclaimers: Cautionary Statement Regarding Forward-Looking Statements
Disclaimers Cautionary Statement Regarding Forward-Looking Statements This Presentation contains certain forward-looking statements within the meaning of the federal securities laws and "forward-looking information" within the meaning of Canadian securities laws (collectively, "forward-looking state
Slide 3: Detalimogene: Investigational Next-Generation Therapy for NMIBC
Detalimogene: Investigational Next-Generation Therapy for NMIBC Designed for the Unique Needs of Community Urology Practices Potential Best-in-Class Tolerability Lowest treatment interruption and discontinuation rates based on approved products PIs 54% CR Anytime (Primary Endpoint), With Low Rate of
Slide 4: DDX Platform: Unique Non-Viral Gene Therapy Without the Headaches
DDX Platform: Unique Non-Viral Gene Therapy Without the Headaches First Plasmid Gene Therapy in Development Large Genetic Cargo Redosable Low Cost of Goods/ No Specialized Handling Corporate Presentation July 2026 Detalimogene
Slide 5: Non-Muscle Invasive Bladder Cancer
Non-Muscle Invasive Bladder Cancer
Slide 6: Non-Muscle Invasive Represents 75%-80% of Bladder Cancer Diagnoses
Non-Muscle Invasive Represents 75%-80% of Bladder Cancer Diagnoses High-Risk NMIBC Patient Segmentation CIS +/- papillary tumors Papillary tumors only NMIBC = non-muscle invasive bladder cancer, HR = High-risk. Sources: SEER database; Knowles et al., Nat Rev Cancer 15, 25–41 (2015); Isharwal and Kon
Slide 7: High-Risk NMIBC is a Slowly Progressing Disease but Highly Recurrent
High-Risk NMIBC is a Slowly Progressing Disease but Highly Recurrent 20% of NMIBC Patients Progress to Muscle Invasive Bladder Cancer (MIBC) Over 10 Years Treatment paradigm diagram is not comprehensive and acts only as an illustration. Additional Sources: Schneider, Bryan J., et al. J Clin Onc 39.3
Slide 8: ~80% of NMIBC Treated in Community Urology Practices
~80% of NMIBC Treated in Community Urology Practices Share of NMIBC Patients by Practice Setting Academic Medical Center-Owned Practice Health System / Hospital-Owned Practice Large Urology Group Practice Corporate Presentation July 2026 Sources: Quantitative Market Research (2,390 patient claims).
Slide 9: Community Urologists Balance a Range of Needs in Selecting Treatments
Community Urologists Balance a Range of Needs in Selecting Treatments Sources: Quantitative Market Research (2,390 patient claims), “The State of the Urology Workforce and Practice in the United States, 2024” American Urological Association Education and Research, Inc. Raymond James & Associates “Co
Slide 10: Detalimogene Voraplasmid
Detalimogene Voraplasmid
Slide 11: Detalimogene: Designed for Bladder-Localized Immune Activation
Detalimogene: Designed for Bladder-Localized Immune Activation Synergistic Stimulation of Specific Innate and Adaptive Immune Responses 1. Gao J, et al, inventors; enGene, Inc., assignee. US patent WO 2013/138930 A1. September 26, 2013. 2. Joshi S, et al. Presented at: 2025 ASCO GU Cancers Symposium
Slide 12: Industrial-Scale Manufacturing With Ease of Handling and Low COGS
Industrial-Scale Manufacturing With Ease of Handling and Low COGS Note: CDRP = Chemistry, Manufacturing and Controls Development and Readiness Pilot. enGene is one of nine companies awarded CDRP per year. Plasmid DNA Proprietary DDX sugar polymer PEG-PLE Easy to Store and Transport Long storage time
Slide 13: Designed to Streamline Experience for Patients and Urology Practices
Designed to Streamline Experience for Patients and Urology Practices Designed to be Simpler to Use than BCG for Both Patients and Practices Patients Practice Fewer doses per 12-week cycle than BCG induction No urine bleaching or close contact precautions with loved ones Storage at 2-8°C* Administere
Slide 14: Designed for Logistical and Post-Treatment Protocol Simplicity
Designed for Logistical and Post-Treatment Protocol Simplicity Non-Viral Gene Therapy Viral Gene Therapy Chemo-Device Combination BCG-Cytokine Combo Therapy Checkpoint Inhibitor No Complex Shipping & Storage Short Administration/Procedure No Viral/Bacterium Decontamination or HCP
Slide 15: Multiple LEGEND Cohorts Maximize Detalimogene's Potential
Multiple LEGEND Cohorts Maximize Detalimogene’s Potential Comprehensive IP Portfolio Through at Least 2040 *These additional non-pivotal cohorts of the LEGEND study provide further opportunity to demonstrate the potential use of detalimogene in clinical settings with unmet need. Progress as of Octob
Slide 16: Detalimogene Without Surfactant Pivotal Cohort Interim Analysis Presented at AUA
Detalimogene Without Surfactant Pivotal Cohort 1 Interim Analysis Presented at AUA Plenary Session May 2026
Slide 17: Pivotal Cohort Study Target Enrollment Achieved
Pivotal Cohort Study Target Enrollment Achieved Patients: BCG-Unresponsive High-risk NMIBC with CIS Design: Global, single-arm, open label N = 125 (fully enrolled) Dosing1: Year 1: 0.8mg/mL in 50mL IVI at weeks 1,2,5,6 Q3M Year 2-3 (Maintenance): 0.8mg/mL IVI at weeks 1,2 Q3M Primary Endpoint: Any t
Slide 18: LEGEND Phase Cohort 1: Baseline Demographics and Disease Characteristics
LEGEND Phase 2 Cohort 1: Baseline Demographics and High-Risk Disease Characteristics Characteristics Pivotal Cohort 1 (N=125) Age, years Median (Range) 71.0 (35.0-90.0) Sex, n (%) Female 24 (19.2%) Male 101 (80.8%) Race n (%) White 114 (91.2%) Asian 4 (3.2%) Black or African American 2 (1.6%) Not Re
Slide 19: 91.0% of CRs Occurred at First Assessment
91.0% of CRs occurred at first assessment 96.8% were free from progression to ≥T2 disease 90.4% of participants remain free from cystectomy 21 additional patients could achieve a CR at 12 months 19 are responders at various time points Any Time Complete Response* 54% (CI: 44.9%, 63.0%) CR Landmark*
Slide 20: Primary Endpoint 54% CR at Any Time; In Range of Approved Products
Primary Endpoint 54% CR at Any Time; In Range of Approved Products These data are presented for informational purposes and are not based on any head-to-head or comparator clinical studies. Cross-trial comparisons are inherently limited and may suggest misleading similarities or differences. Accordin
Slide 21: Detalimogene Demonstrated Favorable Safety and Tolerability Profile
Detalimogene Demonstrated Favorable Safety and Tolerability Profile TRAEs were Generally Mild and Reversible, Consistent with Catheterization Corporate Presentation July 2026 TRAE= treatment-related adverse event. *One patient had a treatment-related pyelonephritis/sepsis that led to a dose interrup
Slide 22: Trending to Potential Best-in-Class Tolerability for Detalimogene
Trending to Potential Best-in-Class Tolerability for Detalimogene Investigational Agents based on TRAEs*** Detalimogene n=125 Creto n = 112 Any Grade 55% 63% Serious 1.6% 1.8% Fatal 0 0 Dose Interruptions 2.4% Not reported Dose Discontinuations 2.4% 0 Source AUA P2 Presentation* CG Investor Deck** A
Slide 23: Detalimogene With Surfactant: Potential to Enhance Efficacy and Durability
Detalimogene With Surfactant Potential to Enhance Efficacy and Durability
Slide 24: Goal: Enhance Detalimogene Profile While Maintaining Differentiated Ease of Use
Goal: Enhance Detalimogene Profile While Maintaining Differentiated Ease of Use, Tolerability, and Patient Experience Corporate Presentation July 2026 Design for Detalimogene without Surfactant Bladder Rinse Design for Detalimogene + Surfactant Bladder Rinse Simple logistics and administration Diffe
Slide 25: Surfactants Increase Transfection of Gene Therapies
Surfactants Increase Transfection of Gene Therapies Preclinical Evaluation Before and After Surfactant Incorporation Corporate Presentation July 2026 Cretostimogene1 Adstiladrin3 1: Cretostimogene developed with a pre-wash surfactant, DDM, before administration of drug. 2: Ramesh et al. Molecular Th
Slide 26: Detalimogene + Surfactant: Broad, Deep Urothelial Nanoparticle Distribution
Detalimogene + Surfactant: Broad, Deep Urothelial Nanoparticle Distribution Increased Gene Expression >10X and Improved Efficacy in Preclinical Murine Models Detalimogene Only Detalimogene + Surfactant Significant Efficacy Increase deta + surfactant >10× Increase IL-12 expression deta + surfactant C
Slide 27: IL-12 Expression Increases: >9X Magnitude and >50% Distribution
IL-12 Expression Increases: >9X Magnitude and >50% Distribution Murine Model Findings Validated in Non-Human Primate Model Detalimogene Only Surfactant + Detalimogene Corporate Presentation July 2026 No Pretreatment Detalimogene 60m 5-min Pretreatment Detalimogene 15m Increase Mean hIL-12 expression
Slide 28: Polidocanol Surfactant: Commercially Available, No Systemic Exposure
Polidocanol Surfactant: Commercially Available, No Systemic Exposure Preclinical Evaluation in Murine Models EG-70 (15’) EG-70 (60’) mIL-12 in Plasma pDNA in Plasma Corporate Presentation July 2026 Clinical Development of a Surfactant Bladder Rinse: 0.25% polidocanol Established Clinical and Safety
Slide 29: LEGEND Detalimogene + Polidocanol Surfactant Cohort First Patients Enrolled
LEGEND Detalimogene + Polidocanol Surfactant Cohort First Patients Enrolled Study Design Target Population: Patients with BCG-unresponsive HR NMIBC with CIS +/- Papillary Enrollment: Up to 80 patients 1w 2w 3w 4w 5w 6w 7w 8w 9w 10w 11w 12w Cycle 2-4 x4 Corporate Presentation July 2026 50 mL bladder
Slide 30: Future Market Dynamics
Future Market Dynamics
Slide 31: Logical Sequencing - Critical Future Consideration for Long-Term Treatment
Logical Sequencing - Critical Future Consideration for Long-Term Treatment Detalimogene ±Surfactant Would Provide a Viable Option for Community Urologists Referral Future Community Practice Sequencing Academic Practice Sequencing HR NMIBC Diagnosis BCG Chemotherapy, CPI & Clinical Trials Radical Cys
Slide 32: Sizeable Future Market Opportunity for Detalimogene
Sizeable Future Market Opportunity for Detalimogene Corporate Presentation July 2026 40 - 75K 25 - 30K 50 - 80K Future Potential Total Addressable Market*of 120K+ High Risk BCG-Unresponsive / Exposed High Risk BCG-Naive Intermediate Risk Total Population 120K+ *Detalimogene pivotal study is for high
Slide 33: Experience Developing and Commercializing Highly Successful Medicines
Experience Developing and Commercializing Highly Successful Medicines RON COOPER Chief Executive Officer Chief Executive Officer, Albireo Pharma President, Europe, Bristol Myers Squibb ANTHONY CHEUNG, Ph.D. Chief Scientific Officer Co-founder, enGene Co-inventor All Key enGene Patents JOAN CONNOLLY
Slide 34: Detalimogene: Next Gen NMIBC Treatment Designed for Community Use
Detalimogene: Next Gen NMIBC Treatment Designed for Community Use Large Total Addressable Market Differentiated Total Product Profile Enhancing Detalimogene Near-Term Planned Catalysts $285M Cash Expect to Provide Operating Flexibility through Potential BLA Filing and Approval Corporate Presentation
Slide 35: July 2026
July 2026
enGene Therapeutics — Investor Presentation (July 2026) on 6ix