enGene Reports First Quarter 2026 Financial Results and Provides Business Update

LEGEND pivotal cohort update planned for a spring 2026 medical conference

12-month complete response data from LEGEND pivotal cohort expected in 2H 2026

Biologics License Application (BLA) submission for detalimogene planned for 2H 2026

Cash and marketable securities of $312.5 million expected to provide cash runway into 2H 2028

Expanded $125 million debt facility with Hercules Capital provides additional financial flexibility to advance detalimogene development and commercialization for bladder cancer

enGene Holdings Inc. (Nasdaq: ENGN, “enGene” or the “Company”), a clinical-stage, non-viral genetic medicines company, today announced its financial results for the first quarter ended January 31, 2026, and provided a business update.

“As data from LEGEND's pivotal cohort in high-risk, BCG-unresponsive NMIBC continues to mature, we look forward to providing an update at a spring medical conference,” said Ron Cooper, President and Chief Executive Officer, enGene. “On the heels of our successful November follow-on offering, we entered 2026 in a position of financial strength and ready to execute. With RMAT and CDRP designations for detalimogene, we are in active dialogue with the FDA to ensure regulatory and manufacturing readiness as we plan for a BLA submission in the second half of this year and potential launch in 2027.”

Recent Corporate Updates

Expanded debt facility: In January 2026, the Company expanded its existing loan and security agreement with Hercules Capital, Inc. (NYSE: HTGC) (“Hercules”), for up to $125 million. Access to the additional non-dilutive capital strengthens enGene’s balance sheet in preparation for its planned Biologics License Application (“BLA”) to the U.S. Food and Drug Administration (“FDA”) for detalimogene voraplasmid as a treatment for high-risk, Bacillus Calmette-Guérin (“BCG”)-unresponsive non-muscle invasive bladder cancer (“NMIBC”) with carcinoma in situ (“CIS”) in the second half of 2026, and the potential commercial launch of detalimogene should it receive FDA approval.

Successful completion of public offering: In November 2025, the Company successfully closed an underwritten public offering of its common shares and pre-funded warrants at an offering price of $8.50 per share and $8.4999 per pre-funded warrant. The offering totaled $140.1 million in net proceeds, following the full exercise of the underwriters’ option to purchase additional shares.

LEGEND pivotal cohort update: In November 2025, enGene reported preliminary data from LEGEND’s pivotal cohort for patients enrolled following a protocol amendment implemented in the fourth quarter of 2024. The preliminary analysis of the post-amendment patients with at least one post-baseline disease assessment included 62 patients at 3 months and 37 patients at 6 months and 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), including 4 patients who 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 total. Data from these patients demonstrated a generally 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.

enGene continues to believe that detalimogene’s emerging profile supports its potential first-line use in patients with high-risk, BCG-unresponsive non-muscle invasive bladder cancer with CIS.

CMC Development and Readiness Pilot (CDRP) Program: In November 2025, the Company was selected as one of nine companies to participate in the FDA’s Chemistry, Manufacturing, and Controls (CMC) Development and Readiness Pilot (CDRP) program. The FDA created the CDRP Program to facilitate CMC development for therapies with compressed clinical development timeframes based on the anticipated clinical benefits of earlier patient access to the therapy. Acceptance into the program further supports enGene’s confidence in detalimogene’s potential and aligns with enGene’s commitment to strong manufacturing practices as clinical development advances.

Anticipated Milestones

• Update from LEGEND pivotal cohort planned for a spring 2026 medical conference.
• 12-month complete response data from LEGEND pivotal cohort expected in 2H 2026.
• Planned BLA filing for LEGEND’s pivotal cohort in the second half of 2026.

First Quarter 2026 Financial Results

As of January 31, 2026, cash, cash equivalents and marketable securities were $312.5 million. The Company expects that its cash, cash equivalents and marketable securities will fund operating expenses, debt obligations and capital expenditures into the second half of 2028.

Total operating expenses were $31.2 million for the three months ended January 31, 2026, compared to $26.6 million for 2025. Research and development expenses increased by $2.3 million, primarily driven by increased personnel and clinical costs related to our LEGEND trial and in preparation for our planned Biologics License Application submission in the second half of 2026. General and administrative expenses increased by $2.3 million, primarily driven by personnel and facilities costs as the Company scales its general and administrative function to support the operation of a public company.

For the three months ended January 31, 2026, net loss attributable to common shareholders was approximately $29.8 million, or $0.44 per share, compared to approximately $24.6 million, or $0.48 per share, for the three months ended January 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 our 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.

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. The RMAT program is intended to expedite the development and review of regenerative medicine therapies for serious or life-threatening conditions, where preliminary clinical evidence suggests potential to address unmet medical needs. Similarly, Fast Track designation is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need. Detalimogene has also been selected to participate in the FDA’s Chemistry, Manufacturing, and Controls (CMC) Development and Readiness Pilot (CDRP) program. The FDA created the CDRP Program 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 three 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); and BCG-unresponsive high-risk NMIBC patients with papillary-only disease (Cohort 3). The LEGEND trial is actively enrolling patients with sites participating in the USA, Canada, Europe, and the Asia-Pacific region.

About enGene

enGene is a clinical-stage biotechnology company mainstreaming gene therapy through the delivery of therapeutics to mucosal tissues and other organs, with the goal of creating new ways to address diseases with high clinical needs. enGene’s lead program is detalimogene voraplasmid (also known as detalimogene, and previously EG-70) for patients with Non-Muscle Invasive Bladder Cancer (NMIBC), a disease with a high clinical burden. Detalimogene is being evaluated in the ongoing multi-cohort LEGEND Phase 2 trial, which includes a pivotal cohort studying detalimogene in high-risk, Bacillus Calmette-Guérin (BCG)-unresponsive patients with carcinoma in situ (CIS) with or without concomitant papillary disease. Detalimogene was developed using enGene’s proprietary Dually Derivatized Oligochitosan (DDX) platform, which enables penetration of mucosal tissues and delivery of a wide range of sizes and types of cargo, including DNA and various forms of RNA.

To learn more, please visit enGene.com and follow us on LinkedIn , X and BlueSky .

Forward-Looking Statements

Certain statements contained in this press release may constitute “forward-looking statements” within the meaning of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, and “forward-looking information” within the meaning of Canadian securities laws (collectively, “forward-looking statements”). enGene’s forward-looking statements include, but are not limited to, statements regarding enGene’s management team’s expectations, hopes, beliefs, intentions, goals, or strategies regarding the future. In addition, any statements that refer to projections, forecasts or other characterizations of future events or circumstances, including any underlying assumptions, are forward-looking statements. The words “anticipate”, “appear”, “approximate”, “believe”, “continue”, “could”, “estimate”, “expect”, “foresee”, “goal”, “intends”, “may”, “might”, “plan”, “possible”, “potential”, “predict”, “project”, “seek”, “should”, “would”, and similar expressions (or the negative version of such words or expressions) may identify forward-looking statements, but the absence of these words does not mean that a statement is not forward-looking. Forward-looking statements may include, for example, statements about: the Company’s plans and expectations regarding regulatory interactions and potential BLA submission for detalimogene and potential commercial launch of detalimogene, the expected period over which enGene estimates its cash and marketable securities will be sufficient to fund its current operating plan, the Company’s expectations as to the timing and anticipated results of the LEGEND study, including the timing of preliminary data or other updates, the CDRP program and its potential benefits, the RMAT designation for detalimongene and its potential benefits, the future development of detalimogene, the potential benefits of detalimogene, including its ability to become a first line therapy in BCG-unresponsive NMIBC and its attractiveness to urologists, additional loan proceeds being available to the Company under the Hercules debt facility subject to satisfaction of certain terms and conditions, and the potential benefits of medicines developed with the DDX platform. Such statements are subject to numerous important factors, risks and uncertainties, many of which are beyond enGene's control, that may cause actual events or results to differ materially from enGene's current expectations. For example, there can be no guarantee that detalimogene will successfully complete necessary clinical development phases, including achieving positive results in the pivotal cohort of the LEGEND study, or that those results or any feedback from regulatory authorities will ultimately lead to BLA submission for, and the approval of, detalimogene.

Management's expectations and, therefore, any forward-looking statements in this press release could also be affected by risks, uncertainties and assumptions relating to a number of other factors, which could cause the Company’s actual results, performance or achievements to differ materially from those expressed or implied by the forward-looking statements, including, without limitation, the inability of preliminary clinical data to predict the final results of the trial, changes in the results from enGene’s clinical trials, including due to new data collected from the ongoing LEGEND study or future studies, subsequent analysis of existing data, and audit and verification procedures; the content and timing of decisions made by the FDA and other regulatory authorities; the Company’s ability to recruit and retain qualified scientific and management personnel, establish clinical trial sites and enroll patients in its clinical trials, execute on the Company’s clinical development plans; and ability to secure regulatory approval on anticipated timelines, and other risks and uncertainties detailed in filings with Canadian securities regulators on SEDAR+ and with the U.S. Securities and Exchange Commission (“SEC”) on EDGAR, including those described in the “Risk Factors” section of the Company’s Annual Report on Form 10-K for the fiscal year ended October 31, 2025 (copies of which may be obtained at www.sedarplus.ca or www.sec.gov).

You should not place undue reliance on any forward-looking statements, which speak only as of the date on which they are made. enGene anticipates that subsequent events and developments will cause enGene’s assessments to change. While enGene may elect to update these forward-looking statements at some point in the future, enGene specifically disclaims any obligation to do so, unless required by applicable law. Nothing in this press release should be regarded as a representation by any person that the forward-looking statements set forth herein will be achieved or that any of the contemplated results of such forward-looking statements will be achieved.

View source version on businesswire.com: https://www.businesswire.com/news/home/20260309831252/en/

Media Contact:
Media@engene.com

Investor Contact:
Investors@engene.com