Engene Therapeutics Inc. · NASDAQ:ENGN
Engene Therapeutics — Corporate Update (May 2026)
20 slides from Engene Therapeutics's corporate update, May 7, 2026
Slide 1: LEGEND Interim Data Update
Non-Viral Genetic Medicine LEGEND Interim Data Update May 7, 2026
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: Introductory Remarks
Introductory Remarks
Slide 4: LEGEND Phase 2 Cohort 1: CONSORT Diagram
LEGEND Phase 2 Cohort 1: CONSORT Diagram Enrolled 125 Patients 1 Discontinued 1 no disease evaluation (censored*) 3-month 124 Completed Evaluation 19 Discontinued • 18 non-CR • 1 CR (censored*) 103 Completed Evaluation 6-month • 2 pending evaluation at 6-mos 51 Discontinued • 51 non-CR 44 Completed
Slide 5: LEGEND Phase 2 Cohort 1: Baseline Demographics and Disease Characteristics
LEGEND Phase 2 Cohort 1: Baseline Demographics and 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 6: 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 Most TRAEs were localized, brief, low-grade events Participants with events, n (%) (N=125) • 69 patients (55.2%) had any grade TRAEs ≥ 1 Trea
Slide 7: LEGEND Cohort 1 Interim Efficacy Data
LEGEND Cohort 1 Interim Efficacy Data Any Time 6 Month 9 Month 12 Month (N=124) (N=121) (N=113) (N=98) CR Rate* 54.0% 43.0% 32.7% 13.3% Total Population (CI: 44.9%, 63.0%) (CI: 34.0%, 52.3%) (CI: 24.2%, 42.2%) (CI: 7.3%, 21.6%) N=125 KM Estimate 39.4% 31.6% 24.5% • Re-induction success rate of 14.0%
Slide 8: Patients Post October 2025 Analysis Underperformed Earlier Subgroups
Patients Post October 2025 Analysis Underperformed Earlier Subgroups Pre-Protocol Amendment Post-Protocol Amendment Newly Assessed Patients October 2025 Analysis* October 2025 Analysis* Since October 2025** N=31 N=62 N=32 Any time CR Rate 55% 63% 39% 6 Month CR Rate 41% 62% 32% • Preliminary subgrou
Slide 9: Bladder Rinse Opportunity
Bladder Rinse Opportunity
Slide 10: Surfactant Bladder Rinse Could Increase Transgene Expression
Surfactant Bladder Rinse Could Increase Transgene Expression • Bladder lumen contains protective mucosal • Surfactant bladder rinse designed to disrupt bladder boundary layer mucosal barrier and further increase DDX permeability • DDX designed to spontaneously penetrate • Potentially improves DDX pe
Slide 11: Detalimogene and Surfactant Bladder Rinse (Polidocanol)
Detalimogene and Surfactant Bladder Rinse (Polidocanol) Designed to Improve Gene Expression, Shorten Dwell Time, and Improve Efficacy Increases Magnitude & Shortens Bladder Dwell Time Improves Efficacy Duration of Gene Expression 1000 No pre-Tx 1000 Curing Ratep< 0.03 0.25% polidocanol (5 min) 50 pg
Slide 12: Detalimogene and Surfactant Bladder Rinse LEGEND Cohort Underway
Detalimogene and Surfactant Bladder Rinse LEGEND Cohort Underway • 20 trial sites activated in US and Canada and actively recruiting patients • Simple 5-min pre-treatment of the bladder with 50 mL of 0.25% polidocanol solution prior to detalimogene administration • Total detalimogene dwell time shor
Slide 13: Market Research Insights
Market Research Insights
Slide 14: Community Urologists: Higher Volume with Fewer Resources and Staff
Community Urologists: Higher Volume with Fewer Resources and Staff Average # of NMIBC Patients per Procedure Room / Urologist Patients per Procedure Room Patients per Urologist 30 14 12 Patients per Procedure Room 25 24 12 Patients per Urologist 10 20 19 8 8 8 15 6 11 6 10 10 4 5 2 0 0 S L Health HS
Slide 15: Less Than Half of Urologists Prescribe Branded Therapies to BCG-UR Patients
Less Than Half of Urologists Prescribe Branded Therapies to BCG-UR Patients Next-Step Following BCG-Unresponsive CIS Diagnosis Branded Therapy Other “I prescribe [Product X] and [Product Y]. I just cannot administer them in my Small Private Practice S 38% 61% office. I have to go to a hospital I hav
Slide 16: Low Adoption of Branded Therapies Driven by Logistics and Cost to Acquire
Low Adoption of Branded Therapies Driven by Logistics and Cost to Acquire Reasons for Limited Use of Branded Therapies Rank Top 3 Small Private Large Urology Hospital System Academic 100% 80% 81% 79% 80% 75% 76% 76% 66% 67% 63% 60% 57% 57% 51% 48% 46% 38% 40% 33% 33% 33% 29% 21% 21% 21% 18% 20% 10%
Slide 17: Market Research Findings: Opportunities for Detalimogene
Market Research Findings: Opportunities for Detalimogene Challenge Detalimogene Opportunity Accessible in lower-resource settings: Limited Resources Nurse-administered without specialized procedure- room or BSL-2 hood requirements Minimal logistical burden: Burdensome No vial thaw, bench-top prepara
Slide 18: Preliminary Efficacy and Durability Estimates
Preliminary Efficacy and Durability Estimates CR Any Time (95% Confidence Intervals) 3 Product 12 Months Landmark Adstiladrin 2 51.0% (41.0, 61.0) Adstiladrin 24% (n=98) 1 Detalimogene 54.0% (44.9, 63.0) Detalimogene 25% est** 0 (n=125) 40% 45% 50% 55% 60% 65% ** KM estimate Percent Median Duration
Slide 19: Closing Remarks and Q&A
Closing Remarks and Q&A
Slide 20: Non-Viral Genetic Medicine
—s i enGene Non-Viral Genetic Medicine