enGene (ENGN), a public Canadian company, presented an update on its bladder cancer gene-therapy program at the Wells Fargo 21st Annual Healthcare Conference on Tuesday, September 8, 2026. CEO Ron Cooper discussed interim results from the LEGEND program for detalimogene voraplasmid, a non-viral gene therapy intended for BCG-unresponsive non-muscle invasive bladder cancer with carcinoma in situ. Wells Fargo biotech analyst Yanan Zhu was identified as the conference host/analyst.
The company said the pivotal Cohort 1 study is an open-label trial enrolling 125 patients, a size it described as one of the largest in this bladder cancer setting. Interim data released in May showed a complete response rate of 54% at any time. Based on Kaplan-Meier estimates, the 12-month landmark complete response rate was projected to be around 25%; 21 complete responders were available for 12-month follow-up at the interim readout. The treatment interruption and discontinuation rate was 2.4%, and disease progression was 3%. About a quarter of LEGEND patients had prior therapy other than BCG.
Cooper said the drug's tolerability continued to hold with additional exposures and that the company believed the safety profile was probably best in class. He also said enGene was the only company offering efficacy, tolerability and ease of use together.
enGene also outlined a surfactant cohort using polidocanol. The safety run-in was completed without reported dose-limiting toxicities. The surfactant reduced dwell time from 60 minutes to 30 minutes, producing about 25 minutes of net patient time savings when installation time is included. Preclinical data showed about a 10-fold increase in IL-12 expression. The cohort could enroll up to 85 patients, a matter still under discussion with the FDA.
The company projected a pre-BLA meeting with the FDA in the fourth quarter of 2024 and a BLA submission by year-end 2024, with FDA approval projected for 2025. enGene said it has obtained Regenerative Medicine Advanced Therapy designation and the CBER Dossier Review Pilot.
Management described the market as split between academic medical centers, which account for about 20% of the patient population, and community urology practices, which account for about 80%. The total annual incident market was estimated at 20,000 to 40,000 patients, depending on definition. A more specific addressable opportunity was identified at about 8,200 patients a year, or 41% of a 20,000-patient baseline, in resource-constrained practices lacking -80°C freezers, BSL-2 hoods and specialized personal protective equipment. The manufacturing module for the BLA is expected to be five to eight times larger than the clinical module.
Financially, enGene reported $266 million in cash. Its stock had declined 79% over the preceding six months and was cited at $1.93 against a 52-week high of $12.25, with a beta of -0.27. Analyst price targets ranged from $2 to $5, and eight analysts had recently revised earnings estimates upward. The company also referenced off-label gemcitabine and ANKTIVA in the treatment context.
