ENGN

54% complete response (CR) rate at any time; 43% six‑month CR rate Low rate of progression to muscle-invasive or higher disease (3.2%) Low percentage of patients experienced treatment-related adverse events (TRAEs) (55%), nearly all of which were mild Low percentage of patients experienced TRAEs leading to treatment interruption (2.4%) or discontinuation (2.4%) Kaplan-Meier estimate of 12-month CR rate is 25% enGene to host webcast to discuss results today, May 7, 2026, at 8:00 a.m. ET BOSTON & MONTREAL, May 07, 2026--(BUSINESS WIRE)--enGene Therapeutics Inc. (Nasdaq: ENGN or "enGene" or the "Company"), a clinical-stage, non-viral genetic medicines company, today reported additional interim results from the pivotal cohort of its ongoing, Phase 2 LEGEND trial of detalimogene voraplasmid (also known as detalimogene) 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, which enrolled 125 patients. These data, as of April 21, 2026, demonstrated that patients treated with detalimogene achieved a 54% complete response (CR) at any time and a 43% CR rate at six months. The progression rate to muscle-invasive or advanced disease was low, at 3.2%. Detalimogene was generally well tolerated with 55% of patients having experienced a treatment-related adverse event, mostly mild (Grade 1 and 2). "These updated data continue to reinforce the favorable safety and tolerability profile of detalimogene and its clinical activity in a heavily pretreated, high-risk NMIBC patient population with limited therapeutic options. Importantly, the low rate of progression to muscle-invasive disease leaves patients eligible for other bladder-sparing therapies," said Ron Cooper, President and Chief Executive Officer, enGene. "While durability outcomes to date are not what we hoped, these data are preliminary. We are focused on evaluating the totality of the data as it evolves and plan to continue to engage with the FDA and the medical community." LEGEND Pivotal Cohort Data Update The interim analysis is based on the 125 patients enrolled in Cohort 1. Patients who discontinued without any disease evaluation or who discontinued after a CR were considered not evaluable for subsequent landmark CR analysis. Efficacy overview: 54% (95% CI: 45%, 63%) CR at any time (6...

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 BOSTON & MONTREAL, March 09, 2026--(BUSINESS WIRE)--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, followin...

EnGene (ENGN) reported a fiscal 2025 net loss late Monday of $2.29 per diluted share, widening from

Completed enrollment in LEGEND’s pivotal cohort with 125 patients in high-risk, BCG-unresponsive NMIBC with carcinoma in situ Reported preliminary 6-month complete response rate (62%) in pivotal cohort patients enrolled under amended LEGEND protocol enGene selected for FDA’s CDRP Program to support CMC readiness Biologic License Application (BLA) submission for detalimogene planned for 2H 2026 Cash and marketable securities plus proceeds from November financing expected to provide cash runway into 2H 2028 BOSTON & MONTREAL, December 22, 2025--(BUSINESS WIRE)--enGene Holdings Inc. (Nasdaq: ENGN, "enGene" or the "Company"), a clinical-stage, non-viral genetic medicines company, today announced its financial results for the full year ended October 31, 2025, and provided a business update. "We are closing 2025 in a position of strength: enrollment in LEGEND's pivotal cohort is complete and the recent data update from the LEGEND pivotal cohort supports an emerging, competitive profile for the use of detalimogene as the first choice therapy in BCG-unresponsive NMIBC if approved," said Ron Cooper, President and Chief Executive Officer. "With recent acceptance into FDA’s CDRP program and a cash position of $342.4 million, extending our runway into the second half of 2028, we are entering the new year laser-focused on scaling our organization in preparation for our planned BLA filing in the second half of 2026 and a potential approval in 2027." Recent Corporate Updates LEGEND pivotal cohort update: In November, the Company reported updated preliminary data from LEGEND’s pivotal cohort for patients who were enrolled following a protocol amendment that was implemented in the fourth quarter of 2024 ("post-amendment patients"). 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. Data from these patients 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 the pivotal cohort, exceeding its target by 25%. Data from these patients demonstrated a generally favorable tolerability profile: 42% of pat...

enGene Holdings Inc. recently reported additional positive preliminary data from its Phase 2 LEGEND trial, highlighting a 62% complete response rate at six months for detalimogene voraplasmid in patients with high-risk, BCG-unresponsive non-muscle invasive bladder cancer. The updated trial enrolled 125 patients, surpassing original targets, and enGene plans to file for regulatory approval in the second half of 2026 for this new gene therapy approach. We'll explore how these encouraging clinical results, especially the high response rates, contribute to enGene Holdings' evolving investment narrative. These 13 companies survived and thrived after COVID and have the right ingredients to survive Trump's tariffs. Discover why before your portfolio feels the trade war pinch. For enGene Holdings, the central case for owning shares hinges on the belief that its gene therapy candidate, detalimogene voraplasmid, can transform care for high-risk, BCG-unresponsive non-muscle invasive bladder cancer. The company’s recent announcement of robust Phase 2 clinical results, a 62% complete response rate at six months, appears to have boosted investor enthusiasm, as reflected by the stock’s rapid surge and a raft of analyst price target upgrades. This milestone potentially shifts the near-term catalyst from a lengthy clinical wait to anticipation around the planned FDA filing in late 2026. However, the follow-on equity offering adds short-term dilution risk, and with enGene still unprofitable and burning cash, uncertainty around future funding and execution remains significant. The latest data may improve the risk/reward profile but does not erase the fundamental business challenges facing pre-revenue biotech firms. However, the funding and dilution risks are still very real concerns investors should be aware of. enGene Holdings' shares have been on the rise but are still potentially undervalued. Find out how large the opportunity might be. Only one fair value estimate from the Simply Wall St Community pegs enGene at US$23.70, a figure well above current prices. Against a backdrop of clinical milestones and capital raises, your outlook could vary sharply, so it’s worth exploring a range of investor viewpoints before making up your mind. Explore another fair value estimate on enGene Holdings - why the stock might be worth just $23.70! Disagree with this assessment? Create your o...

Achieved target enrollment milestone for LEGEND trial pivotal cohort in high-risk BCG unresponsive NMIBC with carcinoma in situ LEGEND’s pivotal cohort data update planned for 4Q 2025 Detalimogene granted Regenerative Medicine Advanced Therapy (RMAT) designation by FDA Biologic License Application (BLA) submission planned for 2H 2026 Cash and marketable securities of $224.9 million expected to provide runway into 2027 BOSTON & MONTREAL, September 11, 2025--(BUSINESS WIRE)--enGene Holdings Inc. (Nasdaq: ENGN, "enGene" or the "Company"), a clinical-stage, non-viral gene therapy company, today announced its financial results for the third quarter ended July 31, 2025, and provided a business update. "Reaching target enrollment in LEGEND’s pivotal Cohort 1 and securing RMAT designation are important milestones that mark our continued momentum," said Ron Cooper, Chief Executive Officer of enGene. "We look forward to providing a data update from the LEGEND pivotal cohort later this year. These advances bring us closer to our planned BLA filing in 2026 with the ultimate goal of delivering detalimogene to patients as a therapy designed for efficacy, safety, and ease-of-use." Recent Corporate Updates LEGEND study enrollment update: The Company announced that it achieved its target enrollment milestone of 100 patients with high-risk, BCG-unresponsive NMIBC carcinoma in-situ (CIS) with or without concomitant papillary disease for the pivotal cohort of its ongoing, open-label, multi-cohort Phase 2 LEGEND trial of detalimogene. Patients in the screening process remain eligible for potential enrollment in Cohort 1. Detalimogene granted RMAT designation: The Company announced that the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to detalimogene voraplasmid for the treatment of high-risk (HR), Bacillus Calmette-Guérin (BCG)-unresponsive, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) based on previously disclosed data from the ongoing LEGEND trial. This designation provides enGene with several regulatory advantages, including early and frequent engagement with the FDA and the potential for rolling submission and priority review. Detalimogene was also granted Fast Track designation from the FDA in November 2020. Key board and management appointments: The Company announced the appointment of...

Additional preliminary data from ongoing LEGEND study of detalimogene in pivotal cohort of BCG-unresponsive NMIBC with CIS anticipated in 2H 2025 Biologics License Application (BLA) filing planned for mid-2026 remains on track EMA Scientific Advice completed; Indicated data could be suitable for a Contingent Marketing Authorization Application Cash and marketable securities of $251.5 million expected to provide runway into 2027 BOSTON & MONTREAL, June 12, 2025--(BUSINESS WIRE)--enGene Holdings Inc. (Nasdaq: ENGN, or "enGene" or the "Company"), a clinical-stage, non-viral genetic medicines company, today announced its financial results for the second quarter ended April 30, 2025, and provided a business update. "We have seen strong enrollment in the pivotal cohort of our LEGEND study," said Ron Cooper, Chief Executive Officer of enGene. "This positions us to stay on track for our planned trial updates across all cohorts in the second half of 2025 and a potential BLA filing in mid-2026, advancing our goal to introduce a novel, non-viral therapy that could redefine treatment for patients with high-risk non-muscle invasive bladder cancer." Recent Corporate Updates Key executive hires and management appointments: In May 2025, the Company announced the appointment of Amy Pott as Chief Global Commercialization Officer. Ms. Pott joined enGene from Astellas Pharma, where she most recently served as Senior Vice President, Strategic Brand Marketing, Ophthalmics and Rare Diseases, and previously as Head of Commercial, Gene Therapies. She will serve as the Company’s first dedicated executive for commercialization planning and execution. LEGEND study enrollment update: Over the course of the first and second quarters of 2025, the Company expanded its clinical footprint for the LEGEND study with the addition of trial sites in Europe and Asia. The pivotal cohort evaluating detalimogene voraplasmid (also known as detalimogene, and previously EG-70) in high-risk, Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) remains on track for our planned BLA in mid-2026. The Company expects to provide an update from LEGEND’s pivotal cohort in the second half of 2025. European Medicines Agency (EMA) Scientific Advice: The Company shared detalimogene preclinical and clinical data through the EMA Scientific Advice process....