enGene (NASDAQ:ENGN) CEO Ron Cooper said the company’s focus is advancing its DDX platform, a proprietary non-viral gene therapy technology, with its lead program detalimogene being developed for non-muscle invasive bladder cancer (NMIBC). Speaking at a Barclays event moderated by Senior Biotech Analyst Etzer Darout, Cooper described 2026 as an “exciting time” for the company as it runs an open-label pivotal trial and prepares multiple data updates and regulatory steps.
Pipeline overview and upcoming milestones
Cooper said enGene is in the middle of an open-label pivotal study for detalimogene in NMIBC. He laid out several anticipated milestones:
- Spring (Q2): a planned data update at a major scientific conference.
- Second half of the year: planned disclosure of long-term, 12-month pivotal-study data; Cooper characterized this as the final results of the fully enrolled pivotal study.
- Planned BLA filing: Cooper said the company is working toward a filing in the second half of the year.
- Potential approval timing: Cooper said enGene expects an approval in 2027.
Why enGene sees NMIBC as a growing market opportunity
Cooper argued the NMIBC opportunity is underappreciated, emphasizing that bladder cancer is the “number six” cancer and that NMIBC is “around 740,000 in the U.S.” He said the market is shifting from a historical paradigm dominated by BCG and bladder removal following BCG failure.
According to Cooper, NMIBC is increasingly recognized as slow progressing, citing progression of “about 20% over 10 years” or potentially lower. With more treatment options emerging, he said therapies are likely to be used sequentially because patients often recur even if they do not progress. He described recurrence rates as “anywhere from 60%-80%.”
To illustrate how sequencing can expand a market, Cooper compared NMIBC to multiple myeloma, where he said multiple product launches increased market size significantly over time. He added that the bladder cancer market is “about a $2 billion market” and is “projected to be a $20 billion plus” market due to sequencing and evolving treatment patterns.
Detalimogene’s differentiation: non-viral delivery, tolerability, and handling
Cooper positioned detalimogene as differentiated primarily because it is a non-viral gene therapy, which he said confers multiple advantages for community urology practices where most patients are treated. He said community urologists prioritize three areas:
- Efficacy
- Tolerability, particularly given that many patients are “75-80-year-old smoking men with comorbidities”
- Practice fit and logistics, including how easily a product integrates into clinic workflow and resource requirements
Cooper said enGene’s preliminary data indicate a complete response (CR) rate that is “very competitive” and suggested the program has “best-in-class tolerability,” citing “very low discontinuation” and “very low treatment interruptions.” He contrasted detalimogene’s handling requirements with other therapies that may require special handling steps, decontamination, hoods, or procedure rooms.
He said detalimogene can be stored in a “regular freezer for many years” and in a “regular fridge for many months,” and described administration as straightforward: remove from the fridge, mix with water, and instill via a regular catheter in a process taking “five or 10 minutes.”
Commercial planning focused on community urologists
On commercialization, Cooper said enGene believes it will need approximately 40–60 sales representatives in the U.S. He said the company is still evaluating its approach in Europe, framing it as a financial decision between going alone versus partnering; outside those regions, he said the company would partner.
Cooper also discussed targeting within community urology groups, including practices organized into “LUGPAs,” where only a subset of physicians may focus heavily on bladder cancer. He emphasized the importance of “buy and bill” dynamics in urology and said the company expects its product’s ease-of-use to help activate practices that have found earlier-generation technologies “clunky.”
LEGEND pivotal study changes, FDA dialogue, and manufacturing strategy
Cooper said the pivotal program is the LEGEND study, with Cohort 1 serving as the pivotal cohort. After initial data shared in 2024, he said enGene implemented three protocol changes aimed at aligning the trial with standard of care:
- Second resection for T1 patients prior to study entry, which Cooper described as standard practice for higher-risk T1 lesions.
- Allowing resection of small Ta papillary growths at the three-month assessment rather than requiring immediate study discontinuation, while continuing to focus on CIS status.
- Requiring biopsies before removing patients from study to make assessments more objective, noting that the bladder can appear red with immunotherapies.
He said these changes, together with improved execution and follow-up, contributed to results shared “late last year” that he described as “really compelling and much better.” For the spring data update, Cooper said the company expects to focus on the primary endpoint: complete response at any time (including three and six months). He said enGene expects this dataset to be “locked” because it has about 125 patients and most should have reached the six-month timepoint.
Cooper said the FDA has been constructive in dialogue and noted that agency guidance has supported open-label studies of roughly 100 patients for full approval in NMIBC settings. He also said enGene has received FDA designations including Fast Track, RMAT, and CDRP.
Discussing endpoints, Cooper said the shift to “CR anytime” as the primary endpoint followed FDA discussions and alignment with endpoints used for other approved products, with duration of response as a key secondary endpoint.
On benchmarking, Cooper said other products tend to show “50%-70%” CR anytime, and he cited enGene’s preliminary “63% anytime” CR. For 12-month outcomes, he described a “20-40” range and said the company expects to be competitive.
Cooper also highlighted manufacturing as a differentiator, saying the company is “pretty much finished” with PPQ/validation batches and will be writing the CMC module. He described CDRP as a way to engage earlier with FDA on manufacturing plans to reduce the risk of complete response letters tied to CMC issues. He added that because detalimogene is neither viral nor an LNP-based product, the company believes it can achieve relatively low cost of goods and broader applicability across diseases.
Finally, Cooper said enGene has begun additional detalimogene cohorts beyond BCG-unresponsive NMIBC with CIS, including patients who are BCG-naïve, BCG-exposed, and those with papillary-only disease without CIS.
About enGene (NASDAQ:ENGN)
enGene, Inc is a clinical‐stage biopharmaceutical company focused on the development of gene‐based therapeutics for oncology. The company’s core technology is the EnGene Delivery Vehicle (EDV) platform, which employs nonliving, bacterially derived minicells to transport therapeutic payloads directly to tumor cells. By combining targeted delivery with potent payloads, enGene aims to improve the precision and efficacy of cancer treatments while reducing off‐target toxicity.
Through its EDV platform, enGene has advanced multiple therapeutic candidates into preclinical and clinical stages.
