enGene Therapeutics (ENGN) Barclays 28th Annual Global Healthcare Conference summary

Company overview and technology

• Proprietary DDX platform enables non-viral gene therapy, with lead asset detalimogene targeting non-muscle invasive bladder cancer (NMIBC).

• Detalimogene is in a pivotal open-label study, with data updates planned for spring and late 2024.

• Cash runway extends into the second half of 2028, supporting ongoing development and commercialization plans.

Market landscape and opportunity

• NMIBC affects about 740,000 people in the U.S., making it a significant market with high unmet need.

• Treatment paradigm is shifting from bladder removal and BCG to sequencing multiple therapies due to high recurrence rates (60%-80%).

• Market projected to grow from $2 billion to over $20 billion as new therapies are sequenced.

Product differentiation and clinical strategy

• Detalimogene is the only non-viral gene therapy in the space, offering best-in-class tolerability and ease of use.

• Product can be stored in standard refrigeration, requires no special handling, and fits well into community urology practices.

• Preliminary data show competitive complete response (CR) rates and low discontinuation.