Executives from enGene NASDAQ: ENGN provided an update on the company’s detalimogene program in non-muscle invasive bladder cancer (NMIBC), discussing recent protocol changes, regulatory interactions, and commercial planning as the study advances.
Protocol amendments and response-rate improvement
In discussing modifications to the NMIBC trial protocol, the company highlighted three changes: T1 re-resection, TA reinduction, and a shift toward biopsy confirmation before removing a patient from the study. Management characterized these as “standard of care” steps intended to align the trial more closely with typical urologist practice.
When asked which amendment contributed most to the improvement observed in six-month complete response (CR) rate, the company emphasized that the outcome likely reflected a combination of factors rather than a single change. Management noted the relatively small size of NMIBC datasets (typically 70 to 100 patients), where results can be meaningfully affected by a small number of patients. In addition to protocol adjustments, the company pointed to changes in clinical trial execution, including different oversight and operational approach, as contributing to improved response rates.
Regarding the switch from visual impression to biopsy, the company said allowing investigators to remove patients based on visual assessment alone could be confounded by bladder inflammation or redness, which can occur with immunotherapy. The company said biopsy confirmation better reflects standard practice and has had a positive effect on efficacy rates.
Reinduction approach and follow-up timing
Management also discussed reinduction in the post-amendment cohort, where four patients converted from non-response at three months to CR at six months following reinduction. The company said all patients are assessed at three months and then receive treatment at six months. Patients who are responders at three months receive treatment at six months, while non-responders at three months are reinduced at six months; after that point, only patients with a CR continue therapy.
On upcoming longer-term readouts, the company reiterated prior guidance that 12-month data are expected in the second half of this year. Management also said the company plans to provide an update at a scientific/medical conference in the second quarter, though details—such as whether it will include nine-month or 12-month data—will depend on coordination with conference organizers. The company suggested it would likely present an intent-to-treat (ITT) population view reflecting the “totality of the data” available at that time.
Pivotal cohort focus and endpoint alignment with FDA
The company said its primary focus remains on the pivotal cohort, noting it exceeded enrollment objectives and grew to roughly 125 patients, which management described as the largest program in the space. Other cohorts (including BCG-naïve or BCG-exposed populations) were described as ongoing but lower priority, intended to provide physicians information over time.
Management also addressed an endpoint change made after FDA feedback: shifting the primary endpoint from CR at six months to CR at any time. The company said this adjustment was intended to align with endpoints used in prior FDA approvals in the NMIBC setting. Management said the company’s interim data show a “competitive” CR at any time rate, described as being “in the 60s,” and compared that to other approved agents that are “roughly” around two-thirds.
Regulatory discussions, SAP process, and manufacturing programs
On the pre- and post-amendment populations, management said there were 31 pre-amendment patients and 94 post-amendment patients, and characterized the pre-amendment group as having minimal impact given the larger post-amendment dataset. The company said it is in ongoing discussions with the FDA on the statistical analysis plan (SAP), describing it as a standard process after full enrollment and before filing. Management said some patient exclusions are common during this process and anticipated potential back-and-forth with the agency.
Executives also discussed FDA engagement tools. Management said the company has received RMAT and CDRP designations, describing RMAT as similar in spirit to breakthrough designation in that it enables more frequent dialogue and earlier feedback on submission plans. On CDRP (CMC Development and Readiness Pilot), management said it is a pilot program involving “one of nine companies” and is intended to support chemistry, manufacturing, and controls (CMC) readiness—an area the company noted has increasingly driven complete response letters (CRLs) in drug reviews. Management said the company is nearing completion of its PPQ/validation batches and expects to begin writing Module 3 soon.
Commercial positioning, practice workflow, and financial runway
From a commercial perspective, the company framed the core prescribing audience as community urologists, stating they represent over 80% of urologists and see the majority of patients. Management said physician market research suggests that efficacy, tolerability, and non-clinical benefits such as ease of use and practice flow are valued at nearly equal levels.
The company outlined workflow and handling attributes it believes differentiate detalimogene, including that it is a non-viral gene therapy and can be stored in a standard freezer for years and in a refrigerator for months. Management emphasized reduced handling complexity, noting it would not require specialized nursing, a hood, or decontamination procedures. It also described administration as potentially taking about five minutes in real-world use, with patients able to void at home.
Management said the company expects its label would not restrict administration to a urologist, suggesting an advanced practice provider or staff member could administer it, which it contrasted with other products that require physician administration. The company also highlighted the attractiveness of a buy-and-bill model for urology practices and suggested private equity ownership trends are increasing focus on practice economics.
On pricing, management said it does not view the market as “winner-take-all,” arguing that with recurrence rates in approved products cited as 60% to 80% and continued desire to avoid bladder removal, therapies are likely to be sequenced and used in combination over time. The company said its manufacturing approach—described as using “simple ingredients” in a non-viral platform—could provide flexibility to price at different levels, with pricing decisions typically finalized late in the process.
Looking ahead to commercialization, management said CMC and clinical spending are currently the largest cash uses, but expects both to decline as the company transitions, partially offset by increases in G&A and commercial spending. The company said it has cash runway into the second half of 2028 and expects a potential approval in 2027.
About enGene NASDAQ: ENGN
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.
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