November 11, 2025 — Leads & Copy —
Boston-based enGene Holdings Inc. (Nasdaq: ENGN) has announced additional preliminary data from the pivotal cohort of its ongoing Phase 2 LEGEND trial of detalimogene voraplasmid (EG-70), a non-viral genetic medicine, in high-risk, Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) patients with carcinoma in situ (CIS) with or without concomitant papillary disease.
The company reported an improved 6-month complete response (CR) rate for patients being treated with detalimogene under its amended protocol. Preliminary data to date and differentiated profile continue to support the Company’s planned Biologics License Application (BLA) submission in the second half of 2026.
The preliminary analysis included 62 patients at 3 months and 37 patients at 6 months. All patients included in this analysis were evaluated under LEGEND’s amended protocol, which went into effect in the fourth quarter of 2024, and was designed to more closely align the LEGEND trial with the American Urological Association’s Guidelines and standard of care.
Data from 62 patients enrolled under the amended protocol with at least one post-baseline disease assessment demonstrated:
- 63% complete response (CR) rate at any time (n=62);
- 56% CR rate at 3 months (n=62);
- 62% CR rate at 6 months (n=37), with 4 patients having successfully converted to CR post reinduction; and
- All 5 patients who completed the 9-month assessment had a CR.
The Company completed enrollment of 125 patients in the pivotal cohort, exceeding its target by 25%. Data from these patients demonstrated a favorable tolerability profile:
- 42% of patients experienced a treatment-related adverse event (TRAE);
- 1.6% of patients experienced dose interruptions due to TRAEs; and
- 0.8% of patients experienced dose discontinuations due to TRAEs.
In September 2024, enGene reported preliminary data from 21 patients in the pivotal cohort of LEGEND who were enrolled prior to implementing a protocol amendment in the fourth quarter of 2024. An additional 10 patients were enrolled under the prior protocol for a total of 31 patients.
Following recent discussions with the FDA, the primary endpoint for LEGEND’s pivotal cohort will change to CR rate at any time from a primary endpoint of landmark 12-month CR rate, with its key secondary endpoint becoming duration of response (DOR) for patients in CR. This updated primary endpoint is consistent with other recent programs registered with FDA.
Cohort 2a, evaluating detalimogene in NMIBC patients with CIS who are naïve to treatment with BCG, has enrolled 30 patients.
Cohort 2b, evaluating detalimogene in high-risk NMIBC patients with CIS who have been exposed to BCG but have not received adequate BCG treatment, has enrolled 45 patients.
Cohort 3, evaluating detalimogene in BCG-unresponsive high-risk NMIBC patients with papillary-only disease, has enrolled 36 patients.
enGene expects to provide a data update on the LEGEND trial’s pivotal cohort in the second half of 2026.
The Company continues to expect to file the BLA in the second half of 2026, with a potential FDA approval in 2027.
Of the 125 patients assessed for safety in Cohort 1 as of October 24, 2025, 53 patients (42%) experienced at least one TRAE, which were mainly Grade 1/2 in severity, except for 3 patients with Grade 3 TRAEs. The most common TRAEs were Bladder Spasm (10.4%), Dysuria (12.0%), Fatigue (16.8%), Micturition Urgency (10.4%), and Pollakiuria (10.4%).
enGene will host a conference call and live webcast at 8:00 a.m. ET today, November 11, 2025.
Non-muscle invasive bladder cancer (NMIBC) is a disease that poses a significant burden on both patients and clinics and has a massive economic impact on the healthcare system.
Detalimogene is a novel, investigational, non-viral gene therapy for patients with high-risk, non-muscle invasive bladder cancer (NMIBC), including Bacillus Calmette-Guérin (BCG)-unresponsive disease. It is designed to be instilled in the bladder and elicit a powerful yet localized anti-tumor immune response.
Detalimogene was developed using the Company’s Dually Derivatized Oligochitosan® (DDX) platform, a technology designed to transform how gene therapies are accessed by patients and utilized by clinicians.
Suzanne Merrill, M.D., Senior Physician, Urologic Oncologist and Bladder Cancer Regional Lead, at Colorado Urology, remarked “Careful selection of an appropriate bladder-sparing therapy is of utmost importance in creating a long-term strategy to maintain a patient’s disease control and quality of life, while minimizing the logistical burden on patient and practice,”
Ron Cooper, President and CEO, said LEGEND study supports planned BLA filing and based on the emerging clinical profile and detalimogene’s differentiated ease of use, the company believes there is a substantial commercial opportunity for detalimogene if approved.
Contact:
enGene Investor Relations
investors@engene.com
Source: enGene
