enGene Therapeutics (ENGN) investor relations material

Company overview and financial position

• Focuses on non-viral gene therapies, with lead asset detalimogene voraplasmid for BCG-resistant non-muscle invasive bladder cancer (NMIBC).

• LEGEND program pivotal cohort includes 125 patients, with data maturing by year-end and FDA filing planned by year-end, targeting approval next year.

• Holds $266 million in cash, sufficient to fund upcoming milestones and growth.

Clinical data and efficacy

• Interim data showed a 54% complete response (CR) rate at any time, aligning with approved products.

• 12-month landmark CR projected at 25%, with durability data maturing in Q4.

• Tolerability is strong, with a low 2.4% discontinuation rate and best-in-class profile.

• Final Q4 data will provide more mature durability and insights from pre-BLA FDA meeting.

Market positioning and commercial opportunity

• Detalimogene is positioned for strong uptake in community urology practices, which represent 80% of the market.

• Product offers efficacy, best-in-class tolerability, and ease of use, making it attractive for resource-constrained practices.

• Estimated incident population is 20,000–40,000 annually, with 41% as prime targets for detalimogene.

• Community practices are expected to drive the majority of adoption, with later-line use in academic centers.