enGene CEO Talks Detalimogene for Community Urology as LEGEND Study Hits Full Enrollment

enGene (NASDAQ:ENGN) CEO Ron Cooper said the company’s lead product candidate, detalimogene, is being developed with an eye toward the practical needs of urologists treating high-risk non-muscle invasive bladder cancer (HR-NMIBC), particularly in the community setting where the majority of patients are managed.

Positioning in a community-driven market

In a fireside chat moderated by Guggenheim Senior Biotech Analyst Michael Schmidt, Cooper emphasized that community urologists face increasing workflow and throughput pressures, noting that many practices are now private-equity owned. Based on enGene’s market research, Cooper said community physicians place roughly equal weight on efficacy, tolerability, and non-clinical factors such as ease of use, fit within practice flow, and economics.

Cooper cited claims-based market research suggesting that more than 80% of NMIBC patients are treated in community settings, compared with earlier data suggesting closer to two-thirds. He described the typical diagnosis pathway as patients—often men in their mid-70s—being referred from primary care to a local urologist after symptoms such as blood in the urine.

Cooper argued detalimogene is well-suited for this environment due to:

• Competitive efficacy relative to other agents in the space, based on interim data the company has shared.
• Low treatment discontinuation and interruptions, which he said reflect strong tolerability.
• Non-viral handling advantages, including expectations of extended storage (potentially years in a standard freezer and multiple months refrigerated), which he said could simplify clinic operations.

Viewing the competitive landscape and the TAR-200 launch

Cooper said he was surprised by the historical HR-NMIBC standard of care, citing backordered BCG and the possibility of bladder removal among the options. He described recent approvals and innovation as “a new dawn” for patients and highlighted interest in Johnson & Johnson’s TAR-200 launch.

He said enGene is watching TAR-200 for two main reasons. First, Cooper noted that available therapies in the space generally show 12-month complete response (CR) rates in the “20%–40-ish%” range, implying high recurrence rates but not necessarily progression; he said recurrence can allow patients to move to other medicines. In that context, he said a strong launch could help “build the prevalent population.”

Second, he said TAR-200 established a “new price point” that is “significantly different” relative to other agents, and enGene is watching how pricing and reimbursement play out. Cooper said detalimogene may have flexibility because, as an immunotherapy with “four simple ingredients,” it could have comparatively low cost of goods. He added that success at that higher benchmark could affect enGene’s valuation, in his view.

LEGEND pivotal study: enrollment, interim efficacy, and tolerability

Cooper said enGene has fully enrolled the LEGEND pivotal study. While the initial target was 100 patients, he said demand was strong and enrollment ultimately reached 125 patients.

Discussing interim results presented in November, Cooper highlighted a six-month complete response rate of 62%, which he characterized as in line with other agents. He also stressed tolerability as a key differentiator, stating that adverse events occurred in 44% of patients “at any time” and were mostly associated with catheterization. He contrasted this with other therapies that he said typically show higher adverse event rates.

Cooper further pointed to low rates of treatment interruption and discontinuation—“in 1% or 2% range,” according to his comments—arguing these measures indicate patients are not having difficulty staying on therapy.

Durability signals and endpoint alignment

Schmidt asked about durability beyond the six-month CR rate. Cooper said enGene’s preclinical models suggest good durability and argued that immunotherapies tend to be durable. From the interim update, he noted that only five patients were evaluable at nine months, and “five of five of those were in complete response.” He also referenced the typical “decay curve” seen across other agents between six, nine, and 12 months, saying he remains confident the product will be “highly durable.”

Cooper also confirmed that the study’s primary endpoint was aligned with prior FDA precedent used for other agents. Instead of a 12-month landmark analysis, he said the primary endpoint is now complete response rate “at any time.”

Regulatory path, manufacturing readiness, and commercial plans

Cooper said enGene has had an active dialogue with the FDA, including guidance indicating that a single, open-label study of around 100 patients could support a full approval. He said this approach has held up in the space, pointing to TAR-200 as an example of approval based on that framework. He added that the company has received RMAT designation and was included in the CDRP pilot program, which he said enables earlier and deeper dialogue with FDA on manufacturing.

On manufacturing, Cooper described the company as “in very good shape,” citing the non-viral approach as a competitive advantage and saying enGene has been manufacturing at scale for some time. He outlined a more complex validation approach involving three drug substances and 12 validation batches, and said the company is “almost done” with FDA validation batches.

Cooper said top-line data from LEGEND are expected in the second half of the year, and he stated the company is on track to file in the second half as well. He noted that 94 of the 125 enrolled patients are post-amendment and 31 are pre-amendment, adding that results are expected to be largely driven by the post-amendment cohort and that the company plans to discuss the statistical analysis plan with FDA.

On commercialization, Cooper said enGene plans to go alone in the U.S., evaluate Europe as a financial decision, and pursue partnerships in the rest of the world. He said U.S. preparations include a head of commercialization, medical affairs personnel already on the ground, and an expected salesforce of roughly 40 to 60 representatives.

Regarding pricing, Cooper said TAR-200 has moved the benchmark higher and described detailed pricing decisions—particularly in buy-and-bill models—as something best finalized late in the process. He said low cost of goods could allow enGene to “thread the needle” on a price that works for practices while remaining consistent with the value of the therapy.

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.