enGene Holdings (ENGN) investor relations material

Technology platform and product overview

• Non-viral genetic therapy platform enables delivery of large genetic cargoes, is non-immunogenic, and offers favorable manufacturing costs and ease of use.

• Lead product, detalimogene voraplasmid, targets non-muscle invasive bladder cancer (NMIBC) and is in a registrational study.

• Product is a lyophilized nanoparticle, easily prepared and administered intravesically, designed to stimulate both innate and adaptive immune responses.

• Manufacturing process is streamlined, and recent CDRP and RMAT designations from the FDA are expected to further facilitate regulatory review.

Disease landscape and market opportunity

• NMIBC is highly prevalent and costly, with 25,000 high-risk cases diagnosed annually in the US and over $6.5 billion in annual management costs.

• Current standard of care, BCG therapy, faces chronic shortages and limited long-term efficacy, often leading to radical cystectomy as a last resort.

• FDA guidance since 2018, reaffirmed in 2024, supports streamlined development for new CIS therapies, highlighting unmet needs.

• Community urologists, who treat most patients, prioritize tolerability, efficacy, ease of use, and cost in therapy selection.

Clinical development and data highlights

• LEGEND study is a global, single-arm, open-label trial in BCG-unresponsive NMIBC, recently fully enrolled with 125 patients.

• Protocol amendments in September 2024 improved consistency and outcomes, with post-amendment six-month complete response rate at 62% versus 41% pre-amendment.

• Durability and tolerability are favorable, with most patients able to continue therapy and low rates of adverse events.

• Product compares favorably to competitors in efficacy, safety, and ease of use, with no need for complex handling or decontamination.