Engene Therapeutics Inc. · NASDAQ:ENGN
Engene Therapeutics — Investor Presentation (September 2025)
28 slides from Engene Therapeutics's investor presentation, Sep 3, 2025
Slide 1: Corporate Presentation September 2025
Non-Viral Genetic Medicine Corporate Presentation September 2025
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 Voraplasmid for NMIBC
Detalimogene Voraplasmid for NMIBC Designed to be a practice-changing therapy requiring no change in practice Transformational Near Term Commercial Highly Differentiated Market Opportunity Opportunity Investigational Product Anticipated Milestones: Pivotal Cohort Update: Q4 2025 Cohort 2 & 3 Updates
Slide 4: NMIBC Represents 75-80% of Bladder Cancer Diagnosis
NMIBC Represents 75-80% of Bladder Cancer Diagnosis Estimated Bladder Cancer US Prevalence: ~730,000 Patients Bladder Cis/Tis Ta T1 Non-Muscle Invasive lumen 80% of newly diagnosed bladder cancer Lamina (~65,000 annual USA cases) propria Inner muscle Muscle-Invasive Outer 20% of newly diagnosed musc
Slide 5: Bladder Cancer- High Incidence, High Prevalence, High Cost to Society
Bladder Cancer- High Incidence, High Prevalence, High Cost to Society Rank by Indication Incidence 1 Breast Bladder Cancer is among the most 2 Prostate expensive cancers to manage per patient: 3 Lung 4 Colorectal 5 Melanoma 6 Bladder 7 Kidney/Renal Estimated >$6.5B Top-10 Cancer 8 Lymphoma (NHL) tot
Slide 6: High-Risk NMIBC: A Multi-year Journey Managed by Community Urologists
High-Risk NMIBC: A Multi-year Journey Managed by Community Urologists Goal: Treat Multiple Recurrences Non-Surgically and Delay Radical Cystectomy Recurrence w/o Progression Diagnosis 1st line: 2nd line and beyond: of high-risk Intravesical BCG Exhaustion of available MIBC Treatment (HR) disease and
Slide 7: Radical Cystectomy: Life-Transforming Surgery of Last Resort
Radical Cystectomy: Life-Transforming Surgery of Last Resort • 10% mortality rate, 6-8 hour inpatient surgery • High morbidity and complication rate: • Sexual dysfunction • Urostomy • Body image • Depression • Anxiety Radical Cystectomy remains standard-of-care for Patient Abdomen BCG-Unresponsive H
Slide 8: Currently Available Non-Surgical Treatments Have Major Limitations
Currently Available Non-Surgical Treatments Have Major Limitations 1st Line Treatment Factors Limiting Use BCG-TICE Chronic global shortage leads to inadequate treatment and rationing Intravesical Chemo (e.g., gemcitabine, Poor efficacy and tolerability; used as BCG backstop mitomycin) 2nd Line Trea
Slide 9: Detalimogene: Non-viral Investigational Therapy Designed To Be the First-Choice
Detalimogene: Non-viral Investigational Therapy Designed To Be the First-Choice • Clinical activity and durability demonstrated: Promising preliminary data from pivotal arm of LEGEND study* • Favorable safety profile observed: LEGEND TRAEs were generally mild, largely instrumentation-related** • Pot
Slide 10: Non-Viral Activation of Both Innate and Adaptive Immunity
Non-Viral Activation of Both Innate and Adaptive Immunity Drug product Bladder lumen (DDX nanoparticle) Intracellular dsRNA agonists Activate intracellular of RIG-I RIG-I receptor Secreted IL-12 Locally stimulates Drug substance incoming T cells (DNA plasmid) Unique mechanism of action uses DNA plas
Slide 11: Industrial-Scale, Well-Characterized Manufacturing Process
Industrial-Scale, Well-Characterized Manufacturing Process 2 1 + + + + + + + + 3 - - -e - - Proprietary Sugar - - - - Polymer PEG-PLE - (DDX) (Excipient) - - - - Plasmid DNA 4 (Drug Substance) e In-line Mixing Bulk Mixing + + + e + • Non-viral, lyophilized drug product + PEGylated DDX/DNA • Establis
Slide 12: Multiple LEGEND Cohorts Demonstrate Detalimogene’s Potential
Multiple LEGEND Cohorts Demonstrate Detalimogene’s Potential Anticipated Milestones Data Update: BCG-Unresponsive NMIBC with Cis Q4 2025 Pivotal Cohort 1 Target Enrollment Achieved n ≈ 100* BLA Filing: H2 2026 Detalimogene Voraplasmid BCG-Naïve NMIBC with Cis Enrolling Cohort Update Q4 2025 (intrave
Slide 13: Pivotal Study
Pivotal Study Target Enrollment Achieved1 Patients: BCG-Unresponsive High-risk NMIBC with CIS Design: Global, single-arm, open label N ≈ 100 Dosing2: • Year 1: 800μg/ml IVe at weeks 1,2,5,6 Q3M • Year 2-3 (Maintenance): 800μg/ml IVe at weeks 1,2 Q3M Primary Endpoints: CR rate at 12-months; safety 1:
Slide 14: LEGEND Pivotal Cohort Patient Demographics
LEGEND Pivotal Cohort Patient Demographics Baseline characteristic N=21 Gender, n (%) Male 15 (71.4) Female 6 (28.6) Age, years Mean (SD) 72.7 (9.4) Median (range) 74 (59, 92) Age categories, n (%) ≤65 6 (28.6) >65 15 (71.4) ECOG, n (%) 0 19 (90.5) 1 2 (9.5) BCG Doses Median 11 Range 8, 33 Tumor Sta
Slide 15: Detalimogene was Generally Well-Tolerated in the LEGEND Study
Detalimogene was Generally Well-Tolerated in the LEGEND Study (n = 42*) Any Grade Grade 1 Grade 2 Grade 3 Grade 4/5 Patients with ≥ 1 TRAE 20 (47.6%) 17 (40.5%) 9 (21.4%) 0 (0.0%) 0 (0) TRAE Reported in >10% patients: Bladder Spasm 8 (19%) 4 (9.5%) 4 (9.5%) 0 (0) 0 (0) Dysuria 9 (21.4%) 8 (19.0%) 1
Slide 16: Treatment Related AEs of Approved and Investigational NMIBC Agents
Treatment Related AEs of Approved and Investigational NMIBC Agents Detalimogene Cretostimogene TAR-2003 Keytruda4 Adstiladrin5 Anktiva + BCG7 Trial LEGEND BOND-0021 BOND-0032 SunRISe-1 KEYNOTE-057 NCT02773849 QUILT 3.032 Sample Size N = 42 N = 67 N = 112 N = 85 N = 101 N = 157 N = 88 TRAEs (Any Grad
Slide 17: Detalimogene: Preliminary Pivotal Efficacy Results
Detalimogene: Preliminary Pivotal Efficacy Results Any Time 3 Month (n=21) 6 Month (n=17) 6 Month KM Estimate 71% CR Rate 67% CR Rate 47% CR Rate 51% CR Rate Data relate to 3m and 6m efficacy-evaluable patients available as of September 13, 2024 17 KM = Kaplan-Meier Analysis; CR = Complete Response
Slide 18: LEGEND Protocol: Revisions Aligned with Treatment Standards
LEGEND Protocol: Revisions Aligned with Treatment Standards Focus Area Prior LEGEND Protocol Current LEGEND Protocol* T1 disease at • Surgically resect lesion via TURBT • Perform 2nd resection at lesion site and restage pre-enrollment screen • Enroll patient • If T1 disease present, patient ineligib
Slide 19: Detalimogene: Preliminary LEGEND Pivotal Data Show Promising Clinical Activity
Detalimogene: Preliminary LEGEND Pivotal Data Show Promising Clinical Activity Detalimogene Cretostimogene TAR-200 Keytruda Adstiladrin Anktiva + BCG Trial LEGEND BOND-0021 BOND-0032 SunRISe-13 KEYNOTE-0574 NCT027738495 QUILT 3.0326 CR Rate, Anytime 71% 65% 76% 82% 41% 51% 62% CR Rate, 6m 47% 44% 63
Slide 20: Previous Single-Arm, Open-Label Studies Have Sufficed for FDA Approval
Previous Single-Arm, Open-Label Studies Have Sufficed for FDA Approval* Pembrolizumab Nadofaragene firadenovec Nogapendekin alfa inbakicep (Keytruda) (Adstiladrin) (Anktiva) + BCG Approval Date January 2020 December 2022 April 2024 Single Arm, Single Arm, Single Arm, Study Design Open label Open lab
Slide 21: Detalimogene: Designed to be the Urologist’s and Patient’s First Choice
Detalimogene: Designed to be the Urologist’s and Patient’s First Choice Practical and Clinical Activity Generally Ease of Use for Differentiated Therapy and Durability Favorable AE Urologists and Designed for Busy Clinics Observed* Profile Observed* Patients 21 *LEGEND phase 1 reported data and Sept
Slide 22: Urologist-Friendly Design: Ideal For Use Early in Treatment Sequence
Urologist-Friendly Design: Ideal For Use Early in Treatment Sequence Detalimogene • Clinical activity observed† • Generally well-tolerated † • “Off-the-shelf” ease of use Cretostimogene* TAR-200 Adstiladrin** • Complex process • Procedure room and cystoscopy likely • Clinic logistics of vial thaw •
Slide 23: Patient Friendly Design That Makes for Ease of Experience
Patient Friendly Design That Makes for Ease of Experience Detalimogene • One hour of chair time total • No need to bleach urine • No induction period Cretostimogene* TAR-200 Adstiladrin** • Virus exposure precautions likely • Potential foreign body sensation when • Virus exposure precautions • Deter
Slide 24: Complementary Investigational Therapies Expected to Transform NMIBC Market
Complementary Investigational Therapies Expected to Transform NMIBC Market and Allow for Sequential Treatments Non-viral Recombinant Viral Gene Drug/Device genetic Immunotherapy Therapies Combos medicine and CPIs + Chemo 2nd Line and Beyond: Pembrolizumab Nadofaragene Gemcitabine/ Exhaustion of non-
Slide 25: Experience Developing and Commercializing Highly Successful Medicines
Experience Developing and Commercializing Highly Successful Medicines RON COOPER ANTHONY CHEUNG, Ph.D. AMY POTT RYAN DAWS ALEX NICHOLS, Ph.D. Chief Executive Officer Chief Scientific Officer Chief Global Commercialization Chief Financial Officer Chief Strategy & Operations Chief Executive Officer, A
Slide 26: Detalimogene Voraplasmid for NMIBC
Detalimogene Voraplasmid for NMIBC Designed to be a practice-changing therapy requiring no change in practice Transformational Near Term Commercial Highly Differentiated Market Opportunity Opportunity Investigational Product Anticipated Milestones: Pivotal Cohort Update: Q4 2025 Cohort 2 & 3 Updates
Slide 27: Additional Slides
Non-Viral Genetic Medicine Additional Slides
Slide 28: Detalimogene is Designed to Streamline Administration and Ease the Experience for Patients and Clinicians
Detalimogene is Designed to Streamline Administration and Ease the Experience for Patients and Clinicians Detalimogene: As designed, no vial thaw, simple preparation; no pre/post treatment protocol Preparation: can be conducted on open Instillation (No post-treatment urine bleaching required) table
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