On November 11, 2026 enGene Therapeutics Inc. (Nasdaq: ENGN, "enGene" or the "Company"), a clinical-stage, non-viral genetic medicines company, reported updated results from the pivotal Cohort 1 of its ongoing, Phase 2 LEGEND trial of detalimogene voraplasmid (also known as detalimogene). Data from Cohort 1, which enrolled 125 patients with high-risk, Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without concomitant papillary disease, are expected to form the basis for a Biologics License Application (BLA) with the U.S. Food and Drug Administration (FDA).

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"For patients with high-risk BCG-unresponsive NMIBC, we need therapies that provide meaningful and durable disease control while minimizing treatment-related toxicity and burden," said Ashish M. Kamat, MD, Professor of Urologic Oncology at The University of Texas MD Anderson Cancer Center and Primary Investigator of the LEGEND trial. "The updated LEGEND data are encouraging, demonstrating clinically meaningful antitumor activity with a favorable tolerability profile. Combined with its straightforward administration and handling requirements, detalimogene has the potential to become a valuable addition to our bladder-preserving treatment options, particularly in community urology practices where ease of administration and practical considerations can influence treatment selection."

"The updated results from Cohort 1 demonstrate encouraging improvements across all key efficacy measures as the data have matured, bringing detalimogene’s efficacy profile to date within range of approved products for this indication," said Ron Cooper, President and Chief Executive Officer, enGene. "Importantly, our recent interactions with the FDA provide a clear path to initiate a BLA submission before year-end 2026, representing a significant step toward bringing detalimogene to patients, with potential approval in 2027."

Mr. Cooper continued, "In parallel, we are preparing for commercialization, with a focus on positioning detalimogene for broad adoption across community urology practices, where more than 75% of high-risk, BCG-unresponsive NMIBC patients receive care. We believe detalimogene’s differentiated ease of use and handling could facilitate integration into existing treatment settings, helping expand access to a new bladder-preserving treatment option for patients."

LEGEND Pivotal Cohort Update

The following efficacy data are based on 120 efficacy-evaluable patients enrolled in Cohort 1 as of the data cutoff date, October 29, 2026.

Efficacy Overview:

CR at any time of 56% (CI:47%, 65%) (n=120)
Landmark 12-month CR rate is 24% (CI: 16%, 33%) (n=110)
1 patient is pending the 12-month landmark disease assessment
Median follow-up for responders increased to 12.0 months from 9.2 months since April 2026 data analysis
Preliminary Kaplan-Meier Estimate of DoR ≥12 months is 43% (CI: 30%, 55%)
9 patients are pending the DoR ≥12 months disease assessment
8 patients currently in maintenance are pending a durability assessment
1 patient is pending the 12-month landmark disease assessment
100% of patients who entered maintenance and had been assessed for durability have recorded durable responses
Median duration of complete response is 10.1 months (CI: 7.5, Not Estimable)
97% of all efficacy evaluable patients were free from progression to muscle-invasive or metastatic bladder cancer, defined as T2 or greater disease
100% of responders were free from progression to T2 or greater disease in the bladder
86% of all efficacy evaluable patients remain free from cystectomy
96% of responders remain free from cystectomy
Safety Overview:

The overall tolerability profile associated with detalimogene has been favorable. Of the 125 patients assessed for safety in Cohort 1, 59% experienced at least one TRAE, which were mainly Grade 1/2 in severity. The most common TRAEs were fatigue (21%), dysuria (16%), pollakiuria (16%), and bladder spasm (13%), micturition urgency (13%), and urinary tract infection (10%). Only 4% of patients experienced Grade 3 TRAE, with just 2% of patients experiencing a serious TRAE. No Grade 4 or Grade 5 TRAEs were reported. TRAEs leading to dose discontinuation (1%) or dose interruption (4%) were rare.

Regulatory Update and Anticipated Milestones:

Following the Company’s pre-BLA meeting with the FDA, enGene reached alignment on key components of the planned BLA for detalimogene, including the efficacy evaluable patient population. The Company expects to initiate the submission by year-end 2026 and plans to complete the BLA submission in the first half of 2027, with potential FDA approval in 2027. In addition, the Company expects to provide an update on the detalimogene plus surfactant bladder rinse cohort mid- 2027.

Conference Call and Webcast

enGene will host a conference call and webcast on November 11, 2026, at 4:30 p.m. Eastern Time to discuss the updated LEGEND data and provide an overview of the regulatory path for detalimogene. Details for the conference call and webcast are included below.

Conference Call Dial-in Numbers:

North America Toll-Free: +1 833-461-5787
International Toll: +1 585-542-9983
Additional International Dial-Ins can be found here

Meeting ID: 485 327 198

Webcast Link: View Source

A link to the webcast will also be available on the Investors section of enGene’s website. A replay of the webcast will be available at this same link within two hours of the event’s conclusion.

About Non-Muscle Invasive Bladder Cancer (NMIBC)

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. NMIBC occurs when cancer cells grow in the tissues that line the interior of the bladder, but the cancer has not yet penetrated the muscle of the bladder wall. NMIBC can present as papillary outgrowths from the bladder wall, which are typically resected, or as carcinoma in situ (CIS), which consists of flat, multifocal lesions that cannot generally be resected. The two forms can also co-occur. About 75%-80% of new bladder cancer diagnoses are NMIBC. Patients suffering from high-risk NMIBC who are unresponsive to the standard of care, Bacillus Calmette-Guérin (BCG), face high rates of disease recurrence (50%-70%) and are potentially subject to full removal of the bladder (cystectomy) as a curative but life-altering next step.

About Detalimogene Voraplasmid

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. Medicines developed with the DDX platform can potentially overcome the limitations of viral-based gene therapies, reduce complexities related to safe handling and cold storage, and streamline both manufacturing processes and administration paradigms.

Regenerative Medicine Advanced Therapy (RMAT) and Fast Track Designations

Detalimogene has received Regenerative Medicine Advanced Therapy (RMAT) and Fast Track designations from the U.S. Food and Drug Administration (FDA) based on its potential to address the high unmet medical need for patients with BCG-unresponsive carcinoma in situ (CIS) NMIBC with or without resected papillary tumors who are unable or refuse to undergo cystectomy. These designations are intended to expedite the development and review of drugs intended to treat serious or life-threatening conditions and fill an unmet medical need. Detalimogene has also been selected for the FDA’s Chemistry, Manufacturing, and Controls (CMC) Development and Readiness Pilot (CDRP) program, designed to facilitate CMC development for therapies with compressed clinical development timeframes based on the anticipated clinical benefits of earlier patient access to the therapy.

About the LEGEND Trial

Detalimogene is being evaluated in the ongoing, open-label, multi-cohort, Phase 2 LEGEND trial to establish its safety and efficacy in high-risk NMIBC. LEGEND’s pivotal cohort (Cohort 1) consists of 125 patients with high-risk, BCG-unresponsive NMIBC with CIS (with or without papillary disease) and is designed to serve as the basis of the Company’s planned Biologics License Application (BLA) filing. In addition to this pivotal cohort, LEGEND includes four additional cohorts, including NMIBC patients with CIS who are naïve to treatment with BCG (Cohort 2a); NMIBC patients with CIS who have been exposed to BCG but have not received adequate BCG treatment (Cohort 2b); BCG-unresponsive high-risk NMIBC patients with papillary-only disease (Cohort 3); and BCG-unresponsive high-risk NMIBC patients with CIS who receive polidocanol plus detalimogene.

(Press release, enGene Therapeutics, NOV 11, 2026, View Source [SID1234674612])