enGene (ENGN) investor relations material

Market landscape and unmet need

• Non-muscle invasive bladder cancer (NMIBC) patients have historically faced limited and extreme treatment options, such as BCG and radical cystectomy, which carries high mortality and morbidity rates.

• Most NMIBC patients are elderly men with comorbidities, and the disease progresses slowly, making radical surgery particularly undesirable.

• Community urologists manage the majority (83%) of NMIBC cases, but face logistical and resource challenges with current therapies.

• Newer therapies like Adstiladrin and J&J's TAR-200 have improved outcomes but are difficult to integrate into community practice due to handling and workflow issues.

Product differentiation and clinical data

• Detalimogene offers a 6-month complete response rate of 62%, comparable to other leading agents, with a favorable tolerability profile and low discontinuation rates.

• Most adverse events are mild and related to catheterization, and treatment interruptions are in the low single digits, much lower than competitors.

• The product requires no special handling, is stable in standard refrigeration, and fits easily into existing clinic workflows, unlike many alternatives.

• Patient visits are shorter and less frequent, reducing burden on both patients and clinics.

Forward-looking statements and development plans

• A key data update is planned for Q2, providing final anytime complete response rates, with a 12-month durability update expected in the second half of the year.

• Regulatory filing is targeted for after the 12-month data, with potential approval anticipated in 2027.

• Market research indicates strong interest from community urologists in a product with high efficacy, tolerability, and ease of integration.

• Additional LEGEND study cohorts (2A, 2B, papillary only) are being deprioritized for now, but may provide supplementary data for future prescribing and guidelines.