enGene Therapeutics (ENGN) Corporate presentation summary

Market overview and unmet needs

• Non-muscle invasive bladder cancer (NMIBC) represents 75–80% of bladder cancer diagnoses, with ~72,000 new NMIBC cases annually in the US.

• High-risk NMIBC is highly recurrent, with 20% progressing to muscle-invasive disease over 10 years and current treatments facing supply and tolerability issues.

• About 75% of NMIBC patients are treated in community urology practices, which often face operational constraints and prioritize ease of use, tolerability, and cost.

• Patient sequencing through multiple lines of therapy is common, expanding the eligible pool for new treatments.

• The total addressable market for NMIBC therapies is projected to exceed 120,000 patients, with a market value forecasted above $20B.

Product profile and differentiation

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

• The therapy is redosable, uses low-cost components, and requires no specialized handling, supporting broad adoption in community settings.

• Storage is simple (refrigeration for months, freezer for years), with no special biosafety or decontamination requirements.

• Administration is streamlined, with fewer doses per cycle, minimal office time, and no need for specialized staff or post-treatment precautions.

• Surfactant bladder rinse is being developed to further enhance efficacy and reduce dwell time, maintaining ease of use.

Clinical data and safety

• In the pivotal LEGEND cohort (N=125), Detalimogene achieved a 54% complete response (CR) rate at any time, within the range of approved products.

• Six-month CR rate was 43%, and 91% of CRs occurred at the first assessment; 96.8% were free from progression to muscle-invasive disease.

• Safety profile is favorable, with 55% experiencing any grade treatment-related adverse events (TRAEs), mostly mild and reversible; serious TRAEs were rare (1.6%), and dose interruptions/discontinuations were low (2.4%).

• Compared to approved products, Detalimogene trends toward best-in-class tolerability, with lower rates of serious adverse events and discontinuations.