On November 11, clinical-stage non-viral genetic medicines company enGene (Nasdaq: ENGN) announced updated results from the pivotal Cohort 1 of its Phase 2 LEGEND trial of detalimogene voraplasmid, which are expected to form the basis for a Biologics License Application (BLA) with the U.S. FDA.
The cohort enrolled 125 patients with high-risk, BCG-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ. As the data matured, the primary endpoint of complete response at any time improved to 56% from 54%, while the key secondary endpoint of the Kaplan-Meier estimate of duration of response of at least 12 months improved to 43% from 25%, and median duration of response improved to 10.1 months from 8.6 months.
On safety, only 2% of patients experienced a serious treatment-related adverse event, with no Grade 4 or Grade 5 events reported. Following a pre-BLA meeting, the company aligned with the FDA on the efficacy-evaluable population and expects to initiate the submission by year-end 2026, with potential approval in 2027.
If approved, detalimogene would be the first and only non-viral plasmid gene therapy suitable for integration into any urology practice setting.

Source: https://investors.engene.com/English/investor/press-releases/news-details/2026/enGene-to-Initiate-a-BLA-Submission-for-Detalimogene-by-Year-End-and-Reports-New-Data-from-LEGEND-Trial-Pivotal-Cohort-1-in-BCG-Unresponsive-NMIBC/default.aspx