enGene (NASDAQ:ENGN) outlined its view of the high-risk Bacillus Calmette-Guérin-unresponsive non-muscle invasive bladder cancer, or NMIBC, market, citing market research that it believes supports a larger addressable patient population and greater use of treatment sequencing before radical cystectomy.
The company also reviewed interim data for its investigational non-viral gene therapy, detalimogene, and said it plans to meet with the U.S. Food and Drug Administration and initiate a biologics license application submission by the end of the year. enGene said it expects a potential approval and platform designation in 2027, though the company noted that its comments included forward-looking statements subject to risks and uncertainties.
Detalimogene Data and Development Plans
At the American Urological Association annual meeting in May, enGene reported an anytime complete response rate of 54% for detalimogene without a surfactant. Cooper said 55% of patients experienced treatment-related adverse events, most of which were mild, while 2.4% were associated with dose interruptions or discontinuations.
The company said duration-of-response data remain maturing. Its Kaplan-Meier estimate for complete response at 12 months was 25%, which Cooper said was similar to the profile of approved therapy ADSTILADRIN.
enGene is also evaluating a surfactant bladder rinse with detalimogene. In preclinical murine and nonhuman primate models, the company said the approach increased IL-12 expression by eightfold to 10-fold. Cooper said the rinse could potentially reduce the treatment dwell time from 60 minutes to 30 minutes while improving efficacy without compromising tolerability. The surfactant cohort is currently underway with an initial safety run-in.
Cooper said enGene has completed process-performance qualification batches and received an FDA Chemistry, Manufacturing and Controls Development and Readiness Pilot Program designation, which the company believes supports biologics license application readiness. The company reported having $285 million in funding.
Market Research Points to Broader Use of Sequencing
Amy Pott, enGene’s chief global commercialization officer, said the company conducted six months of qualitative and quantitative research, including more than 65 interviews with urologists and uro-oncologists, 15 payer interviews, surveys of 200 urologists and uro-oncologists, analysis of more than 2,000 patient claims, and over 20 literature reviews.
Pott cited estimates of approximately 90,000 annual new bladder cancer cases, with about 80% classified as NMIBC and roughly 30% of those considered high risk. She said uncertainty remains around the size of the CIS and papillary-only populations, BCG-unresponsive classification, pricing, and market share.
According to Pott, the company’s research indicates that papillary-only patients are clinically managed and reimbursed similarly to CIS patients. The LEGEND study includes a papillary-only cohort, and enGene said it would seek National Comprehensive Cancer Network guideline inclusion for that population if it receives FDA approval based on its pivotal cohort.
All payers interviewed by enGene indicated they would broadly cover BCG-unresponsive therapies with an NCCN recommendation of category 2A or higher, Pott said.
The company also argued that increasing availability of therapies could expand the treated population because physicians and patients may sequence three or four therapies before turning to radical cystectomy. Pott said this dynamic could increase the prevalent pool of treatable patients rather than merely redistribute market share among available products.
Community Practice Adoption Considerations
enGene’s research categorized private community practices as resource-limited, workflow-constrained, economics-focused, or clinically focused. Pott said more than 40% of practices balance multiple concerns involving resources, economics, and efficacy.
She said practices that use buy-and-bill reimbursement—estimated by enGene to account for 80% of urologists outside hospital health systems—consider net cost recovery important when adopting therapies. The company said the annual wholesale acquisition cost benchmark for NMIBC therapies has risen from about $220,000 to roughly $690,000.
Pott said that at pricing comparable with recent market benchmarks, an NMIBC product would need treatment uptake from about 10% of the eligible population, or around 2,000 patients, to generate roughly $1 billion in net sales. Those estimates reflect enGene’s market assumptions rather than reported sales guidance.
Dr. Neal Shore, medical director at START Cancer Research and an investigator for detalimogene, said community physicians increasingly weigh logistical requirements, staffing demands, patient time in clinic, safety profiles, reimbursement, and treatment efficacy when selecting therapy.
“Patients don’t really want to undergo a life-changing surgery” such as radical cystectomy, Shore said, while physicians also seek to avoid progression to muscle-invasive disease. He said appropriately monitored high-risk NMIBC patients may be candidates for multiple treatment approaches before surgery.
Shore said his practice uses available approved therapies and clinical trials rather than reflexively selecting one product. He added that, in his experience, patients who do not respond to one BCG-unresponsive therapy commonly ask about additional options before considering cystectomy.
Cooper concluded that enGene believes detalimogene’s efficacy observed to date, tolerability, and non-viral handling profile could make it suitable for a broad range of community urology practices if approved.
About enGene (NASDAQ:ENGN)
enGene Holdings Inc, through its subsidiary enGene, Inc, operates as a clinical-stage biotechnology company that develops genetic medicines through the delivery of therapeutics to mucosal tissues and other organs. Its lead product candidate is EG-70 (detalimogene voraplasmid), which is a non-viral immunotherapy to treat non-muscle invasive bladder cancer patients with carcinoma-in-situ (Cis), who are unresponsive to treatment with Bacillus Calmette-Guérin. The company was founded in 2023 and is based in Saint-Laurent, Canada.
