Engene Therapeutics Inc. · NASDAQ:ENGN
Engene Therapeutics — Investor Presentation (June 2024)
21 slides from Engene Therapeutics's investor presentation, Jun 14, 2024
Slide 1: Corporate Presentation June 2024
Corporate Presentation June 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: enGene: moving genetic medicines into the mainstream
enGene: moving genetic medicines into the mainstream
Slide 4: Highly differentiated program designed for community urologists
Highly differentiated program designed for community urologists: Single-step administration, more patient-friendly, no onerous viral containment / handling requirements Profile supportive of expansive applications across bladder cancer: Product candidate attributes conducive to broad physician adopt
Slide 5: Investigational genetic medicine designed for urologists
Investigational genetic medicine designed for urologists: EG-70 combines promising efficacy with a unique, fit-for-purpose product profile * CR = Complete Response. 73% complete response rate represents the overall CR rate at any time during the Ph1 LEGEND study in BCG-unresponsive NMIBC with Cis. M
Slide 6: EG-70 highlights favorable attributes of DDX platform
EG-70 highlights favorable attributes of DDX platform, potentially enabling treatment of other mucosal organs via direct instillation Dually Derivatized Oligochitosan (DDX) Functionalized oligomeric chitosan with embedded DNA plasmid Easily reconstituted in water on open benchtops Proprietary to enG
Slide 7: EG-70 (detalimogene voraplasmid) program and anticipated milestones
EG-70 (detalimogene voraplasmid) Intravesical Phase 2 interim efficacy (Q3 2024) BLA (Mid 2026) Enrollment to commence Q4 2024 HR BCG-unresponsive NMIBC w/ carcinoma in situ (Cis) Multiple billion-dollar opportunity for EG-70 supported by ongoing bladder cancer clinical studies ONCOLOGY Therapeutic
Slide 8: EG-70: addressing unmet needs in NMIBC
EG-70 (detalimogene voraplasmid): addressing unmet needs in NMIBC
Slide 9: Potential multi-billion dollar market opportunity addressing unmet needs in NMIBC
Potential multi-billion dollar market opportunity addressing the unmet needs in NMIBC 1SEER database, 2023 figure; 2Mossanen and Gore, Curr Opin Urol 2014 >82K new diagnoses of bladder cancer per year in USA,1 of which an estimated 21K-24K are high-risk NMIBC Highest cost of all cancers to treat2 BC
Slide 10: EG-70: Designed to eradicate tumors via synergistic immune stimulation
EG-70: Designed to eradicate tumors via synergistic stimulation of bladder-specific innate and adaptive immune responses Innate immune system activation: Dual RIG-I agonists NK cell stimulation and suppressor cell attenuation promotes tumor killing Historical clinical data w/ innate immune activator
Slide 11: Patient / clinician-friendly profile ideal for community urology clinics
Patient / clinician-friendly profile ideal for community urology clinics Product Attribute EG-70 cretostimogene Adstiladrin TAR-200 Does not require repeated burdensome placement and removal of indwelling intravesical device X Active ingredient not used in earlier lines of therapy X Comm
Slide 12: EG-70 is designed to streamline administration for patients and clinicians
EG-70 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 immunode
Slide 13: FDA guidance: single-arm, open-label design appropriate for BCG-unresponsive NMIBC
FDA guidance: Use of a single-arm, open-label design is appropriate for approval for BCG-unresponsive NMIBC Clearly defined patient population and entry criteria for BCG-unresponsive NMIBC provided by guidance Approval based on complete response rate, duration of response, and safety Pivotal Phase 2
Slide 14: Phase 1/2 combined registrational study design and fast track designation
Phase 1/2 combined registrational study design and fast track designation support BLA submission in mid-2026 Phase 1: Primary, Secondary Endpoints - Completed Phase 2, Cohort 1: Interim Data – Mid 2024 N = ~100 *RP2D is Recommended Phase 2 Dose; ** CR is complete response Expected timelines and anti
Slide 15: Data from ongoing Phase 1: 73% complete response rate at any time
Data from ongoing Phase 1: 73% complete response (CR) rate at any time 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
Slide 16: Urine and plasma analysis demonstrate organ-localized production of IL-12
Urine and plasma analysis demonstrate organ-localized production of IL-12 and support Phase 2 dose selection Treatment Day 1 3 8 10 15 43 64 0.1 1 10 100 1000 Dose Level 1 Dose Level 2 Dose Level 3 Urine IL-12 in Phase 1 dose-escalation 1st dose 2nd dose *Slide reflects all data available as of Nove
Slide 17: No DLTs observed in Phase 1: EG-70 safety profile
No DLTs observed in Phase 1: EG-70’s safety profile allowed rational Ph2 dose selection based on efficacy & biomarkers, not toxicity1 Confidential ©2024 1. Kalota S, et al. AUA Annual Meeting 2024 Promising safety and tolerability profile No safety-related patient discontinuations Reversible and lar
Slide 18: EG-70 selected Phase 2 dose demonstrates promising efficacy and durability
EG-70’s selected Phase 2 dose demonstrates a potentially promising efficacy and durability profile EG-70 cretostimogene* Adstiladrin** Mechanism of Action Non-viral, non-integrating gene therapy Viral Oncolytic immunotherapy Viral gene therapy secreting IFN Trial LEGEND BOND-003 CS-003 Data Summariz
Slide 19: LEGEND seeks to establish EG-70 as cornerstone of a non-viral bladder cancer franchise
LEGEND seeks to establish EG-70 as the cornerstone of a non-viral bladder cancer franchise HR BCG Naïve & Exposed NMIBC Other bladder applications (Example: IR NMIBC, MIBC) Other GU cancers Papillary-only expansion of LEGEND study designed to increase potential EG-70 use across entire HR NMIBC 21K-2
Slide 20: Summary: Moving genetic medicines into the mainstream
NMIBC = Non-muscle invasive bladder cancer; BCG = Bacillus Calmette-Guerin; BLA = Biologics License Application; COGS = Cost of Goods Sold Expected timelines and anticipated milestones reflect enGene management's current estimate and are subject to change. Summary: Moving genetic medicines into the
Slide 21: Corporate Presentation June 2024
Corporate Presentation June 2024