Engene Therapeutics Inc. · NASDAQ:ENGN
Engene Therapeutics — Investor Presentation (September 2024)
25 slides from Engene Therapeutics's investor presentation, Sep 5, 2024
Slide 1: Non-Viral Genetic Medicine Corporate Presentation
Non-Viral Genetic Medicine Corporate Presentation September 2024
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 cases in USA) Muscle-Invasive 20% of newly diagnosed bladder cancer (~20,000 cases in USA) Metastatic Papillary tumors: non
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 due to: Ongoing cystoscopy visits, imaging visits, and urine testing every 3-6m Progression to muscle invasive disease (MIBC) Costly, complex treatments for
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: Avoid Radical Cystectomy and Tumor Progression Beyond the Bladder Diagnosis of high-risk (HR) disease 1st line: Intravesical BCG and/or Chemo 2nd line and beyond: Exhaustion of additional non-surgical options Disease Progres
Slide 7: Radical Cystectomy: Life-Transforming Surgery of Last Resort
Radical Cystectomy: Life-Transforming Surgery of Last Resort 6-8 hour inpatient surgery 10% mortality rate 75% complication rate 43% readmission rate Significant (and often lifelong) impact on sexual function, body image, depression, anxiety, and QoL (e.g., urostomy required) Radical Cystectomy rema
Slide 8: Non-Surgical Treatment Alternatives Have Major Limitations
Non-Surgical Treatment Alternatives 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 2nd Line
Slide 9: Detalimogene: Designed To Be the First-Choice Therapy
Detalimogene: “first choice agent” in a multi-year, multi-drug treatment journey, if approved Clinical activity and durability demonstrated: Promising results from Ph1 LEGEND study Potential best-in-class ease-of-use: Non-viral genetic medicine; no specialized handling or cold-chain storage Favorabl
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 10 Bladder lumen Intracellular Drug product (DDX nanoparticle) Drug substance (DNA plasmid) dsRNA agonists of RIG-I Secreted IL-12 Activate intracellular RIG-I
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 of all key components No special delivery or handling requirements + +
Slide 12: Phase 1 Demonstrated Promising Clinical Profile
Phase 1 Demonstrated Promising Clinical Profile Phase 1: Study Design Phase 1 Results Patients: BCG-Unresponsive, High-risk NMIBC with Cis Dosing: 2 or 4 doses in 12-week cycle Cohorts: 3+3 dose escalation (4 dose levels) Endpoints: 1° - Safety; 2° - Efficacy All Dose Groups (N = 22) CR Rate (%) Any
Slide 13: Detalimogene Was Well Tolerated in Ph 1, No Grade 4/5 TRAEs Observed
Detalimogene Was Well Tolerated in Ph 1, No Grade 4/5 TRAEs Observed TRAE = Treatment-related adverse event *Patient had a history of renal failure and recurrent obstructive uropathy with presence of bilateral hydroureteronephrosis at screening – enrollment criteria later modified and excludes patie
Slide 14: Single-Arm, Open-Label Study Has Been Sufficient 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 15: Registrational Study
Registrational Study Patients: BCG-Unresponsive High-risk NMIBC with Cis Design: Global, single-arm, open label N = 100 Dosing: 4x800ug intravesical at weeks 1,2,5,6 Q3M Endpoints: 1° - CR rate at 12-months; 2° - safety and durability Status: Enrolling, BLA filing planned for mid’26 15
Slide 16: Additional LEGEND Cohorts Expand Detalimogene Potential
Additional LEGEND Cohorts Expand Detalimogene 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 Milestones
Slide 17: Detalimogene: 'Practical Therapy'
Detalimogene: ‘Practical Therapy’ Ease of Administration for Patients Clinical Activity and Durability Observed* Ease of Administration For Clinicians Ease of Manufacture and Storage Favorable AE Profile Observed* Detalimogene is Uniquely Designed for Busy Urology Practices 17 *LEGEND Phase 1 report
Slide 18: Urologist-Friendly Design: Detalimogene Ideal For Early Use
Adstiladrin 3-10 hr vial thaw Use w/in 24hrs once thawed Anticholinergic premedication, biosafety precautions, urine bleaching TAR-200 Procedure room and cystoscopy likely required to place and remove ≥Grade 3 AEs Cretostimogene* Complex process Cold chain storage required Oncolytic virus biosafety
Slide 19: Patient Friendly Design Makes for Ease of Experience
Adstiladrin** Virus exposure precautions Extended clinic visit time, 48 hours of urine bleaching TAR-200 Device insertion and cystoscopic removal Potential foreign body sensation when urinating Cretostimogene* Virus exposure precautions likely Prewashes extend chair time Possible contact restrictio
Slide 20: Complementary Investigational Therapies Should Transform and Expand the Market
Viral Gene Therapies Nadofaragene firadenovec (Adstiladrin) Cold-chain storage required Burdensome handling requirements Post-treatment biocontainment Similar limitations expected for cretostimogene grenadenorepvec Bacterial Immunotherapy Bacille Calmette-Guerin (BCG) Global supply chain challenges
Slide 21: 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 22: 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 23: Non-Viral Genetic Medicine Additional Slides
Non-Viral Genetic Medicine Additional Slides
Slide 24: First-in-Human Ph1 LEGEND Demonstrated Clinical Activity
Data cutoff 8.22.2023; **E means ‘expansion’; †, Patient or physician declined to continue on investigational agent to pursue other modes of treatment (e.g., surgery) † †, One additional patient was dosed in this group but later deemed by the independent DSMB to be ineligible and excluded from ‘effi
Slide 25: 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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