enGene Therapeutics (ENGN) investor relations material

Market opportunity and disease landscape

• Non-muscle invasive bladder cancer (NMIBC) represents 75–80% of bladder cancer diagnoses, with ~72,000 new NMIBC cases annually in the US and a high recurrence rate for high-risk patients.

• About 80% of NMIBC patients are treated in community urology practices, which face operational challenges such as high patient volume and limited resources.

• The total addressable market for NMIBC therapies is projected to exceed $20B, with future potential for detalimogene to address over 120,000 patients across risk categories.

Product profile and differentiation

• Detalimogene is a non-viral, plasmid-based gene therapy designed for localized immune activation in the bladder, delivering three genes to stimulate both innate and adaptive immunity.

• The therapy is engineered for ease of use, requiring no specialized handling, and can be administered by nurses or medical assistants in standard exam rooms.

• Industrial-scale manufacturing uses low-cost, readily available components, with long storage times and minimal supply shortage risk.

Clinical data and efficacy

• In the pivotal Phase 2 LEGEND study, detalimogene achieved a 54% complete response (CR) rate at any time, with 91% of CRs occurring at the first assessment and 96.8% of responders free from progression to muscle-invasive disease.

• Six-month CR rate was 43%, and 12-month CR rate data is pending; efficacy is within the range of approved NMIBC therapies.

• The pivotal cohort enrolled 125 patients with high-risk, BCG-unresponsive NMIBC, with a median age of 71 and a majority having received prior BCG therapy.