Engene Therapeutics Inc. · NASDAQ:ENGN
Engene Therapeutics — Investor Presentation (October 2024)
28 slides from Engene Therapeutics's investor presentation, Oct 25, 2024
Slide 1: Non-Viral Genetic Medicine Corporate Presentation
Non-Viral Genetic Medicine Corporate Presentation October 2024 Exhibit 99.2
Slide 2: Cautionary Statement Regarding Forward-Looking Statements
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 statements"). For
Slide 3: Detalimogene Voraplasmid for NMIBC
Detalimogene Voraplasmid for NMIBC Designed to be a practice-changing therapy requiring no change in practice Detalimogene voraplasmid was formerly referred to as EG-70 NMIBC = Non-muscle invasive bladder cancer NMIBC Market Forecast based on internal estimates utilizing multiple source data Highly
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 Non-Muscle Invasive 80% of newly diagnosed bladder cancer (~65,000 annual USA cases) Muscle-Invasive 20% of newly diagnosed bladder cancer (~20,000 annual USA cases) Metastatic Papillary tum
Slide 5: Bladder Cancer - High Incidence, High Prevalence, High Cost to Society
Bladder Cancer- High Incidence, High Prevalence, High Cost to Society Bladder Cancer is among the most expensive cancers to manage per patient: Estimated >$6.5B total annual cost US Top-10 Cancer by US Incidence Rank by Incidence Indication 1 Breast 2 Prostate 3 Lung 4 Colorectal 5 Melanoma 6 Bladde
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 Diagnosis of high-risk (HR) disease 1st line: Intravesical BCG and/or Chemo 2nd line and beyond: Exhaustion of available non-surgical options <20%: Progr
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 treating High-Risk (HR) NMIBC with Cis Image of
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., valrubicin, gemcitabine, mitomycin) Poor efficacy and tolerability; used as BCG backstop 2
Slide 9: Detalimogene: Potential first-choice agent for urologists
Detalimogene: Potential first-choice agent for urologists in a multi-year, multi-drug treatment journey, if approved Clinical activity and durability demonstrated: Promising preliminary data from pivotal arm of LEGEND study Favorable safety profile observed: LEGEND TRAEs were generally mild, largely
Slide 10: Non-Viral Activation of Both Innate and Adaptive Immunity
Non-Viral Activation of Both Innate and Adaptive Immunity Unique mechanism of action uses DNA plasmid to encode large payload of three genes Bladder lumen Intracellular Drug product (DDX nanoparticle) Drug substance (DNA plasmid) dsRNA agonists of RIG-I Secreted IL-12 Activate intracellular RIG-I re
Slide 11: Industrial-Scale, Well-Characterized Manufacturing Process
e e e e e - - - - - (Excipient) PEG-PLE Bulk Mixing In-line Mixing PEGylated DDX/DNA Nanoparticle (Non-Viral Drug Product) DDX/DNA Nanoparticle + + + + + + + Non-viral, lyophilized drug product Established at-scale manufacturing No special delivery or handling + + + + + + + Proprietary Sugar Polymer
Slide 12: Multiple LEGEND Cohorts Demonstrate Detalimogene's Potential
Multiple LEGEND Cohorts Demonstrate Detalimogene’s Potential All NMIBC cohorts refer to high-risk NMIBC unless otherwise specified Expected data update, timelines, enrollment targets, and anticipated milestones reflect enGene management's current estimate and are subject to change Anticipated Milest
Slide 13: Pivotal Study Currently Enrolling Patients
Pivotal Study Currently Enrolling Patients: BCG-Unresponsive High-risk NMIBC with CIS Design: Global, single-arm, open label N ≈ 100 Dosing: 800μg/ml intravesical at weeks 1,2,5,6 Q3M Endpoints: 1° - CR rate at 12-months; 2° - safety and durability
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%)
Slide 16: Detalimogene Demonstrated a Generally Favorable Tolerability Profile
Detalimogene Demonstrated a Generally Favorable Tolerability Profile 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 Gr
Slide 17: Detalimogene: Preliminary Pivotal Efficacy Results
Detalimogene: Preliminary Pivotal Efficacy Results Data relate to 3m and 6m efficacy-evaluable patients available as of September 13, 2024 KM = Kaplan-Meier Analysis; CR = Complete Response 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
Slide 18: Planned LEGEND Protocol Revisions to Align with Treatment Standards
Planned LEGEND Protocol Revisions to Align with Treatment Standards Focus Area Current LEGEND Protocol Proposed Revision T1 disease at pre-enrollment screen Surgically resect lesion via TURBT Enroll patient Perform 2nd resection at lesion site and restage If T1 disease present, patient ineligible Ta
Slide 19: 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% 75% 84% 41% 51% 62% CR Rate, 6m 47% 44% 65
Slide 20: Previous Single-Arm, Open-Label Studies Have Sufficed for FDA Approval
Pembrolizumab (Keytruda) Nadofaragene firadenovec (Adstiladrin) Nogapendekin alfa inbakicep (Anktiva) + BCG Approval Date January 2020 December 2022 April 2024 Study Design Single Arm, Open label Single Arm, Open label Single Arm, Open label Efficacy Evaluable Patients (N)** 96 98 77 12 Month Comple
Slide 21: Practical and Differentiated Therapy Designed for Busy Clinics
Clinical Activity and Durability Observed* Ease of Use for Urologists and Patients Generally Favorable AE Profile Observed* Practical and Differentiated Therapy Designed for Busy Clinics Detalimogene: Designed to be the Urologist’s and Patient’s First Choice *LEGEND phase 1 and preliminary pivotal r
Slide 22: Urologist-Friendly Design: Ideal For Use Early in Treatment Sequence
Adstiladrin** Clinic logistics of vial thaw Risk of financial loss due to prepared product expiry Premedication, biosafety precautions, urine bleaching TAR-200 Procedure room and cystoscopy likely required to place and remove ≥Grade 3 AEs Detalimogene Clinical activity observed† Generally well-toler
Slide 23: Patient Friendly Design That Makes for Ease of Experience
Adstiladrin** Virus exposure precautions Extended clinic visit time 48 hours of urine bleaching TAR-200 Potential foreign body sensation when urinating Risk of additional discomfort or complications due to AE profile Cretostimogene* Virus exposure precautions likely Prewashes extend chair time Possi
Slide 24: Complementary Investigational Therapies Expected to Transform NMIBC Market
Drug/Device Combos + Chemo Gemcitabine/TAR-200 (not approved) Burdensome placement Higher incidence of ≥Gr3 AEs (e.g., urosepsis) Recombinant Immunotherapy and CPIs Pembrolizumab (Keytruda): Risk of severe, systemic AEs Requires IV infusion Nogapendekin alfa inbakicep + BCG (Anktiva) BCG co-administ
Slide 25: Experience Developing and Commercializing Highly Successful Medicines
Experience Developing and Commercializing Highly Successful Medicines RAJ PRUTHI, MD MHA FACS Chief Medical Officer Global Medical Affairs Lead, TAR-210 Bladder Cancer Program, Johnson&Johnson Professor and Chair, Dept of Urology, UCSF Member, AUA/SUO NMIBC Guidelines RON COOPER Chief Executive Offi
Slide 26: Detalimogene Voraplasmid for NMIBC
Detalimogene Voraplasmid for NMIBC Designed to be a practice-changing therapy requiring no change in practice Detalimogene voraplasmid was formerly referred to as EG-70 NMIBC = Non-muscle invasive bladder cancer NMIBC Market Forecast based on internal estimates utilizing multiple source data Highly
Slide 27: Non-Viral Genetic Medicine Additional Slides
Non-Viral Genetic Medicine Additional Slides
Slide 28: Detalimogene is Designed to Streamline Administration and Ease the Experience
Detalimogene is Designed to Streamline Administration and Ease the Experience for Patients and Clinicians †† Based on USPI. * Based on USPI. Note: Once thawed, Adstiladrin vial must be used within 24 hours. Adstiladrin prescribing information also advises that “persons who are immunocompromised or i
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