On September 8, 2026 enGene Therapeutics Inc. (Nasdaq: ENGN, "enGene" or the "Company"), a clinical-stage, non-viral genetic medicines company, reported its financial results for the third quarter ended July 31, 2026, and provided clinical and corporate updates.

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"As we move through the second half of 2026, our focus is on maturing regulatory endpoints data from LEGEND’s pivotal cohort and our planned engagement with the FDA regarding a BLA filing for detalimogene," said Ron Cooper, President and Chief Executive Officer, enGene. "We believe the clinical profile observed to date, together with detalimogene’s potential best-in-class tolerability and straightforward, office-based administration, supports it becoming an important treatment option for urologists and their patients with BCG-unresponsive NMIBC."

"We are pleased to have cleared the surfactant safety run-in period and are encouraged by the progress of the cohort, which is designed to explore whether we can further enhance efficacy and durability while preserving tolerability and the practical attributes that we believe differentiate detalimogene," added Mr. Cooper. "With a strong balance sheet, we remain focused on disciplined capital allocation to advance the program toward BLA submission and prepare for commercialization."

Recent Clinical and Corporate Updates

Detalimogene without Surfactant Pivotal Cohort 1: Detalimogene previously reported an interim 54% complete response (CR) rate at any time and a low rate of progression to muscle-invasive disease (3.2%) in LEGEND’s pivotal Cohort 1, which is evaluating detalimogene without surfactant in patients with high-risk (HR), Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS). enGene expects to report updated data on key primary and secondary regulatory endpoints (CR at any time and Duration of Response (DOR) 12 months), as well as additional durability datapoints, from Cohort 1 and conduct a pre-BLA meeting with the FDA in 4Q 2026 to discuss its planned initiation of a BLA filing before yearend.

Detalimogene plus Surfactant Key Opinion Leader (KOL) Webinar: The Company is currently enrolling high-risk, BCG-unresponsive NMIBC patients with CIS in an additional LEGEND cohort incorporating a brief surfactant bladder rinse with an FDA-approved surfactant solution (generic name: polidocanol). Surfactants have been shown to enhance the efficacy of other intravesical gene therapies for NMIBC and have subsequently been incorporated into their clinical development. In murine models tested by the Company, pretreatment with polidocanol demonstrated a 10-fold increase in mean IL-12 expression and was able to boost efficacy of a subtherapeutic dose of detalimogene. Findings were validated in a large mammal model where a brief surfactant rinse significantly increased the distribution of detalimogene nanoparticles throughout the bladder and IL-12 expression by over nine-fold. Preclinical data indicates that use of a surfactant bladder rinse not only raises the peak of transgene expression but makes transfection consistent across the bladder surface. In a disease that recurs multifocally, transfection coverage may matter as much as amplitude.

On October 15, 2026, at 10:00 a.m. ET, the Company will host a webinar to provide greater detail on the preclinical data supporting the incorporation of its surfactant bladder rinse into clinical development. In addition, the Company will be joined by two KOLs from Colorado Urology, David Cahn, MD, and Suzanne Merrill, MD, FACS, to review clinical case studies and discuss various treatment considerations.

Please click here to register for the event.

Emerging NMIBC Market Insights KOL Webinar: On August 11, 2026, the Company hosted a virtual KOL event featuring Neal Shore, MD, FACS, Medical Director for START-Carolinas Research, who joined management to discuss the evolving non-muscle invasive bladder cancer (NMIBC) treatment landscape. As a part of its broader pre-commercial preparation efforts, the Company shared highlights from its ongoing market research, including the future treatment paradigm where avoidance of radical cystectomy by sequencing multiple lines of therapy will become the norm, the prevalent population will continue to grow as a result of increased sequencing, and a new market price point has been established by recently launched products.

Dr. Shore emphasized the importance of a product profile that could address the various needs of community urology practices, including practice workflow constraints, while also providing a well-tolerated and effective therapy for patients. A replay of the webinar can be accessed here.

Board Leadership Transition: In July 2026, Michael Heffernan, a member of enGene’s Board of Directors since July 2025, assumed the role of Chairman of the Board, succeeding Dr. Richard Glickman, who had served as Chairman for over 14 years. The transition was made as the Company plans for potential regulatory milestones and to support commercial readiness for detalimogene, if approved.

Anticipated Milestones

Data update on key regulatory endpoints, complete response (CR) at any time and maturing durability, as well as landmark CR rates from LEGEND’s pivotal cohort planned for 4Q 2026
Pre-BLA meeting with the FDA in 4Q 2026
Initiation of BLA filing for detalimogene in 4Q 2026
Potential FDA approval decision for detalimogene and platform designation in 2027
Third Quarter 2026 Financial Results

As of July 31, 2026, cash, cash equivalents and marketable securities were $266.3 million providing significant operational flexibility.

Total operating expenses were $34.0 million for the three months ended July 31, 2026, compared to $29.9 million for the three months ended July 31, 2025. Research and development expenses decreased by $2.5 million, primarily driven by timing of process validation manufacturing activities in preparation to initiate the submission of a planned Biologics License Application with the FDA in the fourth quarter of 2026, partially offset by increased workforce reduction-related costs. General and administrative expenses increased by $6.6 million, primarily driven by workforce reduction-related costs and the annualization of personnel-related costs.

For the three months ended July 31, 2026, net loss attributable to common shareholders was approximately $32.5 million, or $0.47 per share, compared to approximately $29.0 million, or $0.57 per share, for the three months ended July 31, 2025. The increase in net loss is mainly attributed to the increase in operating expenses, partially offset by net interest income earned during the period.

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 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 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, SEP 8, 2026, View Source [SID1234670634])