Engene Therapeutics Inc. · NASDAQ:ENGN
Engene Therapeutics — 2024 AUA Annual Meeting
12 slides from Engene Therapeutics's conference presentation, 2024
Slide 1: LEGEND: a Phase 1/2 study of EG-70 (detalimogene voraplasmid)
LEGEND: a Phase 1/2 study of EG-70 (detalimogene voraplasmid), a novel, non-viral intravesical gene therapy for patients with BCG-unresponsive non- muscle invasive bladder cancer with carcinoma in situ (CIS) Susan Kalota, Shreyas Joshi, Matthew Bui, Rian Dickstein, Jen-Jane Liu, Yair Lotan, Scott Jo
Slide 2: Disclosures: Gordon Brown D.O., FACOS
Disclosures: Gordon Brown D.O., FACOS Affiliations • Summit Health-NJU, Jefferson Health • No conflicts of interest Research support • Astellas, Bayer, enGene, Janssen, mdxhealth, Sumitomo Consultancy support • Astellas, Bayer, enGene, Ferring, Janssen, Lantheus, mdxhealth, Novartis, Pfizer, Sumitom
Slide 3: Bladder-sparing therapies for BCG-unresponsive NMIBC address an important unmet need
Bladder-sparing therapies for BCG-unresponsive NMIBC address an important unmet need Bladder cancer Non-muscle invasive bladder In 2023, there In 2023, there cancer (NMIBC)2 80% were more than were over 82,000 16,000 estimated new estimated deaths bladder cancer due to bladder 20% Muscle-invasive ca
Slide 4: What is EG-70 (detalimogene voraplasmid)?
What is EG-70 (detalimogene voraplasmid)? A novel, investigational, non-integrating, non-viral gene therapy designed for ease of use in any urology clinic Simplified preparation & administration No extended vial thaw; no pre-medication required Simple IVI administration of 50 mL volume; 60 minutes
Slide 5: EG-70: Designed to bring genetic medicine to any urology clinic
EG-70: Designed to bring genetic medicine to any urology clinic EG-70 is designed to eradicate tumors via synergistic Innate immune system activation: dual RIG-I agonists stimulation of specific innate and adaptive immune responses localized to the bladder • Combination of NK cell stimulation and su
Slide 6: LEGEND (NCT04752722): Phase 1/2 study
LEGEND (NCT04752722): Phase 1/2 study Phase 1: Primary, secondary endpoints – completed Phase 2: Interim data – mid 2024 N = 24 N = ~100 Patients: High-risk NMIBC failed BCG, with CIS Patients: BCG-unresponsive NMIBC with CIS* EG-70 dosing: 2 or 4 doses over 12-week cycle EG-70 dosing: 4 x 0.8 mg/mL
Slide 7: Phase 1: IL-12 detected in the urine
Phase 1: IL-12 detected in the urine Concentration of IL-12 protein in patient urine during treatment 1000 Dose level • Transgene IL-12 protein detected in a dose– 0.25 mg/mL 0.8 mg/mL responsive manner in the urine of every patient 2.5 mg/mL 100 Concentration (pg/mL) − Urine IL-12 levels at Dose le
Slide 8: Phase 1: Status and patient demographics
Phase 1: Status and patient demographics Status* Baseline demographics/disease characteristics (safety population) • 24 patients had received ≥1 dose of EG-70 by Baseline characteristic N=24 intravesical administration and are evaluable for safety Gender, n (%) • 22 patients are in the efficacy data
Slide 9: Phase 1: Treatment-related adverse events (TRAEs)
Phase 1: Treatment-related adverse events (TRAEs) All TRAEs Grades 4 or 5 Patients with Grade 1 Grade 2 Grade 3 (life-threatening Patients with TRAEs TRAEs, n (%) (Mild) (Moderate) (Severe) or death) n (%) N=24 Hematuria 3 (12.5) 0 (0.0) 0 (0.0) 0 (0.0) Any 13 (54.2) Grade 1 11 (45.8) Urinary tract
Slide 10: LEGEND: Phase 1 efficacy
LEGEND: Phase 1 efficacy % (n/N)* All doses Selected Phase 2 dose Complete Response (n=22) (n=10) Any time 73% (16/22) 70% (7/10) 3 months 68% (15/22) 70% (7/10) 6 months 45% (10/22) 60% (6/10) Duration of response ≥3 months 11/15 (73%) 6/7 (86%) ≥6 months 6/10 (60%) 3/4 (75%) *The efficacy evaluabl
Slide 11: LEGEND study: Conclusions
LEGEND study: Conclusions • EG-70 is an investigational, non-viral, non-integrating, intravesically administered immunotherapy undergoing evaluation for the treatment of patients with NMIBC with CIS • Interim data from the phase 1 portion of the LEGEND study suggest a promising safety, tolerability
Slide 12: Acknowledgements
Acknowledgements • The authors would like to express their gratitude to all the investigators, patients and their families for participating in the LEGEND study