enGene’s gene therapy for high-risk, Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) has touted a 63% complete response (CR) rate in a pivotal Phase II trial.
Preliminary data from the LEGEND study (NCT04752722) of detalimogene voraplasmid is from 62 patients at three months, 37 patients at six months and five patients at nine months.
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At the three-month period, there was a 56% CR rate, which rose to 62% at six months, with four patients having successfully converted to CR post reinduction. All five patients who completed the nine-month assessment had a CR.
Investors were impressed with the readout, with the company’s stock rising 42% to $8.55 per share at market open on 11 November compared to a 10 November close of $6.01. enGene has a market cap of $412.5m.
Chief medical officer at enGene, Dr Hussein Sweiti, said: “We are pleased to report an improved six-month CR rate for patients being treated with detalimogene under our amended protocol. 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.”
The data released on 11 November follows earlier preliminary data from 21 patients released in September 2024, who were enrolled before a protocol amendment. An additional 10 patients were enrolled under the prior protocol for a total of 31 patients. In these patients, there was a CR rate of 55% at any time.
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By GlobalDataThe new preliminary data continues to support the company’s planned Biologics License Application (BLA) submission in H2 2026, with a data update from the pivotal cohort also expected in the same period. enGene will engage with the FDA on a statistical analysis plan (SAP) to determine which patients will be included in the final efficacy evaluable population.
Safety data from the whole patient population showed that 42% experienced a treatment-related adverse event (TRAE), with 1.6% of patients experiencing dose interruptions and 0.8% discontinuing dosing due to TRAEs. Most TRAEs were Grade 1/2 in severity, except for three patients who suffered Grade 3 TRAEs.
The trial has now completed enrolment of 125 patients, which includes 30 NMIBC patients with CIS who are naïve to treatment with BCG, 45 high-risk NMIBC patients with CIS who have been exposed to BCG but have not received adequate BCG treatment, and 36 BCG-unresponsive high-risk NMIBC patients with papillary-only disease.
If approved, GlobalData predicts a patient-based forecast of $731m for detalimogene voraplasmid in 2033.
There have been a lot of developments in the NMIBC space, with UroGen recently gaining approval for Zusduri (mitomycin) in low-grade intermediate-risk NMIBC. As well as this, there are a number of drugs, including MSD’s Keytruda which are in Phase III trials, paving the way for a much broader treatment landscape.
This comes amid anticipated growth in the bladder cancer market across the eight major markets (8MM: US, France, Germany, Italy, Spain, UK, China, and Japan). The market is projected to grow from $3bn in 2023 to $16bn in 2033, at a compound annual growth rate (CAGR) of 18%, across both NMIBC and muscle-invasive bladder cancer (MIBC).
GlobalData is the parent company of Clinical Trials Arena.
Cell & Gene therapy coverage on Clinical Trials Arena is supported by Cytiva.
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