enGene Therapeutics (ENGN) investor relations material

Market opportunity and disease landscape

• NMIBC represents 75–80% of bladder cancer diagnoses, with ~72,000 new US cases annually and a market forecast exceeding $20B globally.

• 80% of NMIBC patients are treated in community urology practices, which face operational strains and high patient volumes.

• Current treatments like BCG face chronic shortages, poor tolerability, and high recurrence rates, highlighting significant unmet needs.

• FDA guidance supports the development of new therapies for long-term bladder preservation, with recent approvals in the space.

Product overview and platform

• Detalimogene is a non-viral gene therapy using the proprietary DDX platform, enabling large genetic cargo, low immunogenicity, and low cost of goods.

• The therapy is designed for easy integration into community and academic urology settings, with simple storage and administration requirements.

• Detalimogene delivers plasmid DNA encoding genes that activate the RIG-I pathway and IL-12, stimulating both innate and adaptive immune responses locally in the bladder.

• Manufacturing leverages low-cost, readily available components, with long storage times and minimal supply shortage risk.

Clinical development and efficacy

• The pivotal LEGEND study enrolled 125 patients with BCG-unresponsive high-risk NMIBC with CIS, using a global, single-arm, open-label design.

• Protocol amendments aligned with guidelines improved patient selection and assessment, resulting in a post-amendment cohort with higher risk and more heavily pre-treated patients.

• Post-amendment, the 6-month complete response (CR) rate reached 62% (CI: 46–76%), with 63% at any time, showing improvement over pre-amendment results.

• CR rates for detalimogene are trending competitively with other approved and investigational therapies, especially in the post-amendment cohort.