Engene Therapeutics Inc. · NASDAQ:ENGN
Engene Therapeutics — Investor Presentation (September 2026)
37 slides from Engene Therapeutics's investor presentation, September 2026
Slide 1: Corporate Presentation: Non-Viral Genetic Medicine
Non-Viral Genetic Medicine Corporate Presentation
Slide 2: Disclaimers
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 NMIBC Market is Growing Unique Non-Viral Offering • More treatment options are expected to increase • Simplified storage and handling designed to sequencing which will expand treatable population support broad community adoption • Marke
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 Detalimogene Redosable Low Cost of Goods/ No Specialized Handling 4 Corporate Presentation September 2026
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 US Prevalent Bladder Cancer ~740,000 High-Risk NMIBC Patient Segmentation • CIS +/- papillary tumors US Incident Bladder Cancer • Papillary tumors only ~90,000/year Incident NMIBC ~72,000/year Incident HR NMIBC ~22,000/year NMIBC = n
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 HR NMIBC Diagnosis Standard of care, chronic supply shortage BCG Poor long-term tolerability 50%: 2-year recurrence New Used as BCG backstop
Slide 8: ~75% of NMIBC Treated in Community Urology Practices
~75% of NMIBC Treated in Community Urology Practices Community Practices • Operations may be strained due 29% 47% to high patient volume, staff Health System / Hospital-Owned shortages, and equipment Practice limitations Private Practice • Community urologists manage higher patient volumes on 24% av
Slide 9: Community Urologists Balance a Range of Needs in Selecting Treatments
Community Urologists Balance a Range of Needs in Selecting Treatments Community Urologist Treatment Considerations: Tolerability, Efficacy & Ease of Use Tolerability Private Practice Academic Medical 12-month CR Rate Center-Owned Practice 12-month DOR Rate 47% Lack of Freezer Storage & 24% Thaw Time
Slide 10: ~40% of Patients Are in Practices Balancing Efficacy, Safety, Resources and Economics
~40% of Patients Are in Practices that Consider the Balance Between Efficacy, Safety, Available Resources and Practice Economics Urology Segment NMIBC Patients Considerations Resource-LimitedPractices Practices 19% Upfront acquisition cost is the gating factor; Resource-Limited practices cannot admi
Slide 11: Detalimogene Voraplasmid
Detalimogene Voraplasmid
Slide 12: Detalimogene: Designed for Bladder-Localized Immune Activation
Detalimogene: Designed for Bladder-Localized Immune Activation Synergistic Stimulation of Specific Innate and Adaptive Immune Responses Detalimogene Voraplasmid VA1 (RIG-I agonist dsRNA) Detalimogene Plasmid IL-12 eRNA11a nanoparticle (RIG-I agonist dsRNA) • Detalimogene delivers plasmid DNA encodin
Slide 13: Industrial-Scale Manufacturing With Ease of Handling and Low COGS
Industrial-Scale Manufacturing With Ease of Handling and Low COGS Readily Available Low-Cost Components Easy to Store and Transport • Long storage time: − Freezer for years − Fridge for months • No special delivery, biosafety or Plasmid DNA PPE requirements Proprietary DDX sugar PEG-PLE polymer Low
Slide 14: 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 Detalimogene Patients Practice Fewer doses per 12-week cycle than Storage at BCG induction 2-8°C* Minimal office time Administered by nurses with no thaw or pre
Slide 15: Developed for Logistical and Post-Treatment Protocol Simplicity
Developed for Logistical and Post-Treatment Protocol Simplicity Non-Viral Gene Viral Gene Chemo-Device BCG-Cytokine Checkpoint Therapy Therapy Combination Combo Therapy Inhibitor No Complex Shipping & Storage ✓ ✓ ✓ ✓ Short Administration/Procedure ✓ ✓ ✓ ✓ No Viral/Bacterium Decontamination or HC
Slide 16: Multiple LEGEND Cohorts Maximize Detalimogene's Potential
Multiple LEGEND Cohorts Maximize Detalimogene’s Potential Cohort / NMIBC Indication Pre-Clinical Phase 1 Pivotal Status Enrollment Complete: Pivotal Cohort 1: 12-Month Data Update BCG-Unresponsive /w CIS Expected 2H 2026 Surfactant Bladder Rinse Cohort: First Patients Enrolled: BCG-Unresponsive NMIB
Slide 17: Detalimogene Without Surfactant
Detalimogene Without Surfactant Pivotal Cohort 1 Interim Analysis Presented at AUA Plenary Session May 2026
Slide 18: Pivotal Cohort Study
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: K
Slide 19: LEGEND Cohort 1: Baseline Demographics and High-Risk Disease Characteristics
LEGEND Cohort 1: Baseline Demographics and High-Risk Disease Characteristics 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 cystectomy,
Slide 20: Clinically Meaningful Activity, Low Progression
Detalimogene Without Surfactant Clinically Meaningful Activity, Low Progression 100% CR Landmark* CR Rate, % (95% CI) CR by KM (Est %) Any Time Complete 6 month 43.0% Response* (n=121) (CI: 34.0%, 52.3%) 39.4% 80% Overall CR Rate, % 54% 12 month (n=98) 13.3% (CI: 7.3%, 21.6%) 24.5% (CI: 44.9%, 63.0%
Slide 21: 54% CR at Any Time; In Range of Approved Products
Primary Endpoint 54% CR at Any Time; In Range of Approved Products Approved Products Detalimogene Keytruda Adstiladrin Anktiva + BCG Inlexzo Cretostimogene Drug Class Non-Viral Gene Therapy CPI Viral Gene Therapy Bacterial IO Chemo-Device Viral Gene Therapy Product Label USPI Sample Size TBD N = 96
Slide 22: Detalimogene Demonstrated Favorable Safety and Tolerability Profile
Detalimogene Demonstrated Favorable Safety and Tolerability Profile TRAEs were Generally Mild and Reversible, Consistent with Catheterization Pivotal Cohort 1 Participants with events n, (%) (N=125) ≥ 1 Treatment-related adverse event 55.2% Most frequent TRAEs (≥10%) Fatigue Dysuria 21.6% 13.6% Low
Slide 23: Trending to Potential Best-in-Class Tolerability for Detalimogene
Trending to Potential Best-in-Class Tolerability for Detalimogene Investigational Agents based on Approved Products based on ADRs*** TRAEs*** Detalimogene Creto Keytruda Adstiladrin Anktiva + BCG Inlexzo n=125 n = 112 n=148 n=157 n=88 n=85 Any Grade 55% 63% 66% Not disclosed Not disclosed 84% Seriou
Slide 24: Detalimogene With Surfactant
Detalimogene With Surfactant Potential to Enhance Efficacy and Durability
Slide 25: Goal: Enhance Detalimogene Profile
Goal: Enhance Detalimogene Profile While Maintaining Differentiated Ease of Use, Tolerability, and Patient Experience Design for Detalimogene without Design for Detalimogene + Surfactant Bladder Rinse Surfactant Bladder Rinse • Simple logistics and administration • Simple logistics and administratio
Slide 26: Surfactants Increase Transfection of Gene Therapies
Surfactants Increase Transfection of Gene Therapies Preclinical Evaluation Before and After Surfactant Incorporation Cretostimogene1 Adstiladrin3 Cretostimogene-like2 virus: <5% transfection Adstiladrin-like virus4: Negligible transfection Bladders Harvested Bladder Surfactant (DDM) + Cretostimogene
Slide 27: 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 >10× Increase IL-12 expression deta + surfactant Detalimogene + Surfactant Significant Efficacy Increase deta + surfactant D
Slide 28: IL-12 Expression Increases: >9X Magnitude and >50% Distribution
IL-12 Expression Increases: >9X Magnitude and >50% Distribution Murine Model F indings Validated in Non-Human Primate Model Surfactant + Detalimogene Detalimogene Only No Pretreatment 5-min Pretreatment Increase Detalimogene 60m Detalimogene 15m Mean hIL-12 expression 0.294 2.725 9X Magnitude (pg/mg
Slide 29: Polidocanol Surfactant: Commercially Available, No Systemic Exposure
Polidocanol Surfactant: Commercially Available, No Systemic Exposure Preclinical Evaluation in Murine and Non-Human Primate Models EG-70 (60’) EG-70 (15’) mIL-12 pDNApDNA in Plasmain Plasma Concentration mIL-12 mIL-12 Protein in Plasma in Plasma Concentration Polidocanol in Plasma* 150 Polidocanol i
Slide 30: LEGEND Detalimogene + Polidocanol Surfactant Cohort
LEGEND Detalimogene + Polidocanol Surfactant Cohort First Patients Enrolled • Single-catheterization 5 min bladder rinse with 50 mL of 0.25% polidocanol prior to detalimogene administration • Intravesical dwell time for detalimogene reduced from 60 to 30 minutes • 29 active enrolling sites; expandin
Slide 31: Future Market Dynamics
Future Market Dynamics
Slide 32: Eligible Patient Pool Exceeds the Annual BCG-Unresponsive Incidence
Eligible Patient Pool Exceeds the Annual BCG-Unresponsive Incidence Patients will Sequence Therapies Creating a Compounding Effect on the Eligible Pool Presents at Community All interviewed urologists will 89% patients would try HR NMIBC Diagnosis sequence patients through 3-4L of anything to avoid
Slide 33: NCCN Guideline 2A Dictates Payer Coverage At New Price Point
NCCN Guideline 2A Dictates Payer Coverage At New Price Point Interviewed payers will cover therapies at parity assuming NCCN guideline category 2A New Price Benchmark $800 Annual WAC ($ USD) $600 $400 $200 $0 2019 2020 2021 2022 2023 2024 2025 2026 Launch Year $1 Billion Net Sales with ≈2,000 Patien
Slide 34: Sizeable Future Market Opportunity for Detalimogene
Sizeable Future Market Opportunity for Detalimogene Future Potential Total Addressable Market*of 120K+ 180 150 120 50 - 80K 90 25 - 30K 120K+ 60 30 40 - 75K 0 High Risk High Risk High Risk Risk Intermediate Total High Intermediate Total BCG-Unresponsive BCG-Unresponsive / BCG-Naïve Risk BCG-Naive Ri
Slide 35: Experience Developing and Commercializing Highly Successful Medicines
Experience Developing and Commercializing Highly Successful Medicines RON COOPER William Grossman, M.D., ANTHONY CHEUNG, Ph.D. AMY POTT Chief Executive Officer Ph.D Chief Scientific Officer Chief Global Commercialization Chief Executive Officer, Albireo Interim Chief Medical Officer Co-founder, enGe
Slide 36: Detalimogene: Next Gen NMIBC Treatment Designed for Community Use
Detalimogene: Next Gen NMIBC Treatment Designed for Community Use Large Total NMIBC market forecasted to be >$20B Addressable Market Differentiated Total Demonstrated Clinical Activity, Favorable Safety, and a Streamlined Patient and Practice Experience Product Profile Enhancing Surfactant Bladder R
Slide 37: Thank You
Z enGene Thank You September 2026