Low rates of treatment-related adverse events (42%) and dose interruptions (1.6%)

enGene Holdings Inc. a clinical-stage, non-viral genetic medicines company, reported additional preliminary data from the pivotal cohort of its ongoing, Phase 2 LEGEND trial of detalimogene voraplasmid (also known as detalimogene and previously EG-70) in high-risk, Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) patients with carcinoma in situ (CIS) with or without concomitant papillary disease. Detalimogene’s preliminary data to date and differentiated profile continue to support the Company’s planned Biologics License Application (BLA) submission in the second half of 2026.

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The preliminary analysis included 62 patients at 3 months and 37 patients at 6 months. All patients included in this analysis were evaluated under LEGEND’s amended protocol, which went into effect in the fourth quarter of 2024, and was designed to more closely align the LEGEND trial with the American Urological Association’s Guidelines and standard of care.

Data from 62 patients enrolled under the amended protocol with at least one post-baseline disease assessment demonstrated:

  • 63% complete response (CR) rate at any time (n=62);
  • 56% CR rate at 3 months (n=62);
  • 62% CR rate at 6 months (n=37), with 4 patients having successfully converted to CR post reinduction; and
  • All 5 patients who completed the 9-month assessment had a CR.

The Company completed enrollment of 125 patients in the pivotal cohort, exceeding its target by 25%. Data from these patients demonstrated a favorable tolerability profile:

  • 42% of patients experienced a treatment-related adverse event (TRAE);
  • 1.6% of patients experienced dose interruptions due to TRAEs; and
  • 0.8% of patients experienced dose discontinuations due to TRAEs.

“We are pleased to report an improved 6-month CR rate for patients being treated with detalimogene under our amended protocol,” said Hussein Sweiti, M.D., MSc, Chief Medical Officer. “With a competitive preliminary efficacy profile and potential for best-in-class tolerability and ease of use, we believe detalimogene could emerge as the first-line therapy for patients with high-risk, BCG-unresponsive NMIBC.”

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“Careful selection of an appropriate bladder-sparing therapy is of utmost importance in creating a long-term strategy to maintain a patient’s disease control and quality of life, while minimizing the logistical burden on patient and practice,” remarked Suzanne Merrill, M.D., Senior Physician, Urologic Oncologist and Bladder Cancer Regional Lead, at Colorado Urology. “I am pleased to see the positive trajectory of detalimogene’s efficacy and tolerability data. Combined with its ease of use, detalimogene would be an attractive option to both patient and a busy urology practice.”

In September 2024, enGene reported preliminary data from 21 patients in the pivotal cohort of LEGEND who were enrolled prior to implementing a protocol amendment in the fourth quarter of 2024. An additional 10 patients were enrolled under the prior protocol for a total of 31 patients. The table below provides summary efficacy from the two separate patient subpopulations.

Preliminary efficacy data for patients enrolled prior to implementing the protocol amendment demonstrated a markedly lower 12-month CR rate than those of U.S. Food and Drug Administration (FDA)-approved products for BCG-unresponsive NMIBC. The Company is encouraged by the strong improvement demonstrated in the preliminary 6-month CR rate of patients enrolled under the amended protocol.

Additional LEGEND trial updates

  • Following recent discussions with the FDA, the primary endpoint for LEGEND’s pivotal cohort will change to CR rate at any time from a primary endpoint of landmark 12-month CR rate, with its key secondary endpoint becoming duration of response (DOR) for patients in CR. This updated primary endpoint is consistent with other recent programs registered with FDA.
  • The Company plans to engage with FDA on a statistical analysis plan (SAP) to determine which patients will be included in the final efficacy evaluable population.
  • Cohort 2a, evaluating detalimogene in NMIBC patients with CIS who are naïve to treatment with BCG, has enrolled 30 patients.
  • Cohort 2b, evaluating detalimogene in high-risk NMIBC patients with CIS who have been exposed to BCG but have not received adequate BCG treatment, has enrolled 45 patients.
  • Cohort 3, evaluating detalimogene in BCG-unresponsive high-risk NMIBC patients with papillary-only disease, has enrolled 36 patients.

“We are highly encouraged with the preliminary data from our LEGEND study, which support our planned BLA filing,” said Ron Cooper, President and CEO. “Based on the emerging clinical profile and detalimogene’s differentiated ease of use, we continue to believe there is a substantial commercial opportunity for detalimogene if approved.”

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Source- businesswire