enGene (NASDAQ:ENGN) Chief Executive Officer Ron Cooper outlined the company’s near-term regulatory and clinical plans for its lead program, detalimogene voraplasmid, during a presentation at Oppenheimer’s 36th Annual Healthcare Life Sciences Conference. Cooper said the company expects to provide an update from its pivotal study in the second half of 2026, submit a biologics license application (BLA) by year-end 2026, and target a potential commercial launch in 2027.
Focus on non-viral genetic medicines
Cooper described enGene as focused on non-viral genetic medicines and highlighted several perceived advantages of a non-viral approach. He said the platform is not constrained by the “small packet size” of viral gene therapies, can be re-dosed due to a lack of immunogenicity, and should enable competitive manufacturing economics because it uses “simple ingredients” that are readily available and do not require special handling.
Targeting high-risk NMIBC
He described the current care pathway as typically beginning with bacillus Calmette-Guérin (BCG) therapy, noting longstanding supply constraints and increased toxicity with additional dosing. According to Cooper, patients may then move to newer therapies that can be “clunky to use,” intravenous chemotherapy with limited efficacy data, or ultimately cystectomy (bladder removal), which he characterized as a major surgery with meaningful morbidity and mortality, citing 5% to 15% mortality.
Cooper also said NMIBC is characterized by recurrence and relatively slow progression, with about 20% progressing to muscle-invasive disease over 10 years. He noted the FDA has issued guidance enabling full approval based on an open-label study of roughly 100 patients, using complete response (CR) as a key endpoint, and said multiple agents have been approved under this pathway.
Mechanism and administration
Cooper said detalimogene is designed to activate both innate and adaptive immune pathways. He described it as a nanoparticle containing two RIG-I agonists intended to stimulate the innate immune system, along with IL-12 to stimulate adaptive immunity. The therapy is administered intravesically, with the solution bathing the bladder.
He emphasized attributes he believes align with community practice needs, including tolerability, ease of administration, and practice workflow integration. Cooper said enGene anticipates administration could take about five minutes and that patients could void at home without special precautions such as bleaching. He also said the product is expected to have straightforward storage requirements, including stability for years in a standard freezer and for many months in a standard refrigerator, without special rooms, specialized staff, or decontamination procedures.
Pivotal LEGEND study enrollment and interim results
Cooper said the pivotal cohort has completed enrollment at 125 patients versus a target of 100. He explained that 94 patients were enrolled after “significant protocol changes,” while 31 were enrolled prior to those amendments, and that the final analysis will be “heavily driven” by the post-amendment group.
Sharing an update previously provided in late 2025 from 62 of the 94 post-amendment patients, Cooper said the study achieved roughly a 62% six-month complete response rate and a 63% CR rate at any time. He added that four patients who did not respond at three months achieved response by six months, which he said was consistent with an immunotherapy-like pattern. He also noted the dataset was not yet mature for long-term follow-up but said the first five patients evaluable at nine months were all responders.
Cooper characterized the post-amendment results as a “material change” in six-month CR relative to the pre-amendment group and said the emerging efficacy profile compares favorably with other agents, which he described as generally delivering CR rates around two-thirds in small datasets. He emphasized that enGene’s study is among the larger datasets, with 125 patients enrolled.
On tolerability, Cooper said most adverse events were Grade 1 or Grade 2 and largely associated with catheterization. He highlighted low rates of dose interruptions (less than 2%) and dose discontinuations (under 1%), and contrasted that with higher interruption and discontinuation rates he said are seen with other agents in the space.
Manufacturing status and regulatory support
Cooper said manufacturing is a competitive advantage for enGene, citing readily sourced inputs and progress toward scale production, including approaching FDA validation batches. He also said the FDA granted the company a CDRP designation as part of a pilot program, which he said is intended to help through the manufacturing process and reduce the risk of a complete response letter by ensuring commercial-level manufacturing readiness.
Commercial positioning, combinations, and outlook
In a question-and-answer session, Cooper said the non-viral approach and low cost of goods could support combination strategies with other therapies. He noted that 12-month CR rates across available treatments are generally in the 20% to 40% range—implying high recurrence—and suggested combinations could improve outcomes as more agents are approved.
Discussing the BCG-naïve opportunity, Cooper said BCG remains entrenched due to habit and cost, but added that supply limitations, restrictions, and toxicity with repeated dosing may create room for alternatives like detalimogene.
On timing, Cooper said enGene intends to provide a second-half 2026 update when most LEGEND patients have reached the 12-month mark. On pricing, he said low cost of goods provides flexibility and that the company will watch how pricing and reimbursement evolve for other agents before maximizing the opportunity for detalimogene.
Cooper also said enGene has sufficient cash to move past key inflection points, with runway into the second half of 2028.
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.
