enGene (ENGN) Corporate presentation summary

Market opportunity and unmet need

• NMIBC represents 75-80% of bladder cancer diagnoses, with a US prevalence of ~740,000 and incidence of ~90,000/year.

• NMIBC is highly recurrent, slow progressing, and costly to manage, with >$6.5B/year in total costs.

• Current therapies like BCG face chronic shortages and poor tolerability, while cystectomy is a last resort with high morbidity.

• FDA guidance highlights the need for new therapies that preserve the bladder long-term.

• The NMIBC market is forecasted to exceed $20B, indicating a significant commercial opportunity.

Product profile and platform

• Detalimogene voraplasmid is a novel, non-viral gene therapy designed for seamless integration in urology practices.

• The DDX platform enables delivery of large genetic cargo, is non-immunogenic, and requires no specialized handling.

• The therapy activates both innate and adaptive immune pathways via plasmid-encoded immune-stimulatory genes.

• Manufacturing is industrial-scale, non-viral, lyophilized, and does not require special delivery or storage.

• Designed for logistical simplicity, with short administration, no decontamination, and minimal post-treatment precautions.

Clinical development and efficacy

• The pivotal LEGEND study enrolled 125 patients with BCG-unresponsive high-risk NMIBC with CIS.

• Protocol amendments in Q4 2024 aligned with standard of care and improved patient management.

• Post-amendment, the anytime CR rate was 63% (CI: 51-74%), with a 6-month CR rate of 62% (CI: 46-76%).

• Pre-amendment, the anytime CR rate was 55% (CI: 38-71%), and 6-month CR rate was 41% (CI: 25-59%).

• Efficacy is competitive with other novel products, trending toward best-in-class for 6-month CR rates.