Boston– enGene Therapeutics Inc. said it ended its fiscal second quarter with $285.2 million in cash, cash equivalents and marketable securities, while announcing clinical updates for its lead bladder cancer program and a roughly 50% workforce reduction to preserve cash.
The clinical-stage, non-viral genetic medicines company said it expects 12-month complete response data from the pivotal Cohort 1 of its LEGEND trial and further engagement with the U.S. Food and Drug Administration in the second half of 2026. The company also plans to initiate a Biologics License Application filing for detalimogene in the same period.
Detalimogene is enGene’s investigational non-viral gene therapy being studied for high-risk, Bacillus Calmette-Guérin-unresponsive non-muscle invasive bladder cancer with carcinoma in situ.
“Feedback from the urology community on the emerging detalimogene without surfactant profile, presented at the AUA meeting in May, supports our plan to await mature durability data in the second half of 2026 and meet with the FDA about initiating a BLA submission for detalimogene before year end,” said Ron Cooper, President and Chief Executive Officer, enGene.
Mr. Cooper continued, “We are also encouraged by the interest in the detalimogene surfactant bladder rinse study from the medical community. With patients already enrolled, we are optimistic about the potential to further enhance efficacy while maintaining the ease of use and tolerability profile that has resonated with urologists.”
“To preserve shareholder capital as we await additional durability data and meetings with the FDA, we made the very difficult decision to downsize the organization to streamline operations. We are sincerely grateful to the enGeneers who have created a high-performance culture and helped advance detalimogene and our mission to provide people living with NMIBC a new treatment option,” added Mr. Cooper.
At the recent American Urological Association meeting, enGene presented interim data from LEGEND’s pivotal Cohort 1, which is studying detalimogene without surfactant in patients with high-risk, BCG-unresponsive non-muscle invasive bladder cancer with carcinoma in situ.
The company reported a 54% complete response rate at any time, or 67 of 124 patients, and a low rate of progression to muscle-invasive or more advanced disease at 3.2%. Treatment-related adverse events leading to treatment interruption and treatment discontinuation were each reported in 2.4% of patients.
As of the April 21 data cutoff, 21 patients were still pending disease assessments at either six, nine or 12 months. enGene said it is awaiting 12-month complete response data from Cohort 1, along with most of the 12-month durability data, and expects to discuss a BLA filing with the FDA in the second half of 2026.
The company also said the first patients have been enrolled in an additional LEGEND cohort evaluating detalimogene with a short surfactant bladder rinse using diluted polidocanol solution. enGene said it may enroll up to 80 patients in the global cohort.
enGene said preclinical testing showed that polidocanol pretreatment increased mean IL-12 expression by 10-fold in murine models and boosted the efficacy of a subtherapeutic dose of detalimogene. In a large mammal model, a surfactant rinse increased distribution of detalimogene nanoparticles throughout the bladder by about 50% and IL-12 expression by nine-fold.
The company said it believes the surfactant approach could further improve efficacy and durability while preserving the treatment’s tolerability and office-based administration profile.
As part of cash conservation efforts, enGene has stopped enrollment in LEGEND Cohorts 2a, 2b and 3 and plans to reevaluate its strategy after FDA discussions in the second half of 2026.
The company said it implemented a plan in June to reduce its workforce by about 50%. It said it retained personnel and resources needed to complete LEGEND Cohort 1, enroll the detalimogene plus surfactant cohort, meet with the FDA, prepare for BLA initiation and support precommercial activities for a potential 2027 launch.
In connection with the restructuring, Chief Financial Officer Ryan Daws, Chief Legal Officer Lee Giguere and Chief Strategy and Operations Officer Alex Nichols will leave the company effective July 15. Chief Scientific Officer Anthony Cheung will remain in his role through Sept. 30 before transitioning to a consulting arrangement.
Hussein Sweiti, M.D., stepped down as Chief Medical Officer effective June 14 to pursue another professional opportunity. William Grossman, M.D., Ph.D., a member of enGene’s board, will serve as interim Chief Medical Officer.
enGene also said Constantine Chinoporos, who joined the company in May as a business development consultant, is expected to serve as interim Chief Business Officer.
For the quarter ended April 30, enGene reported total operating expenses of $32.0 million, compared with $27.1 million for the same period a year earlier. Research and development expenses rose by $2.0 million, mainly due to higher personnel and clinical costs tied to the LEGEND trial, completion of PPQ batch manufacturing and preparation for the planned BLA submission.
General and administrative expenses increased by $2.9 million, primarily due to personnel-related costs and higher facility costs.
Net loss attributable to common shareholders was about $30.2 million, or 43 cents per share, compared with a net loss of about $25.8 million, or 51 cents per share, a year earlier. The company said the higher net loss was mainly due to increased operating expenses, partly offset by net interest income.
enGene said its anticipated milestones include 12-month complete response data for Cohort 1 and a pre-BLA meeting in the second half of 2026, initiation of the BLA filing for detalimogene in the second half of 2026 and a potential FDA approval decision and platform designation in 2027.
