enGene (ENGN) Oppenheimer 36th Annual Healthcare Life Sciences Conference summary

Strategic focus and market outlook

• Emphasis on non-viral genetic medicines, with detalimogene voraplasmid as the lead product targeting non-muscle invasive bladder cancer (NMIBC).

• NMIBC market expected to grow significantly, with projections from $1 billion to over $20 billion as new agents are adopted and sequencing becomes standard.

• Majority of NMIBC patients are treated in community settings, driving demand for therapies that are easy to administer and integrate into existing practices.

• Detalimogene is positioned as a convenient, well-tolerated, and efficacious option, fitting the needs of both patients and community urologists.

• Anticipated data update in the second half of 2024, BLA filing by year-end, and potential product launch in 2027.

Clinical development and efficacy data

• Pivotal trial for detalimogene is fully enrolled with 125 patients, exceeding the initial target.

• Six-month complete response (CR) rate of 62% and anytime CR rate of 63% in a high-risk, heavily pretreated population.

• Early nine-month data show all evaluable patients remained responders, indicating durable efficacy.

• Post-protocol amendment patients demonstrated improved CR rates compared to pre-amendment cohorts.

• Competitive efficacy profile compared to other approved and investigational agents in NMIBC.

Safety, tolerability, and administration advantages

• Most adverse events are mild (Grade 1 or 2), primarily related to catheterization.

• Dose interruptions and discontinuations are very low, supporting strong tolerability.

• Detalimogene can be administered quickly, with minimal chair time and no special handling or decontamination required.

• Product is stable in standard freezers and refrigerators, simplifying logistics for practices.

• Best-in-class convenience and ease of use for both patients and healthcare providers.