enGene Holdings Inc. (ENGN) announced Wednesday that the U.S. Food and Drug Administration or FDA has granted Regenerative Medicine Advanced Therapy or RMAT designation to Detalimogene voraplasmid, the company's lead investigational gene-based immunotherapy for high-risk, BCG-unresponsive non-muscle invasive bladder cancer or NMIBC with carcinoma in situ or CIS.
Detalimogene is a non-viral therapy delivered directly into the bladder to stimulate a localized immune response.
It is currently being evaluated in the pivotal LEGEND Phase 2 trial, enrolling approximately 100 patients with BCG-unresponsive NMIBC.
According to enGene, RMAT designation was granted based on preliminary clinical results demonstrating meaningful anti-tumor activity and a favorable safety profile.
The designation provides access to accelerated development tools, including rolling submission and priority review, and facilitates early, frequent interaction with the FDA.
Detalimogene is administered in urology clinics, including community settings, and is designed as a bladder-sparing alternative for patients who otherwise face radical cystectomy.
The therapy was developed using enGene's proprietary Dually Derivatized Oligochitosan or DDX platform, which supports efficient gene delivery without the use of viruses. It also holds Fast Track designation from the FDA.
The ongoing LEGEND study includes additional cohorts exploring detalimogene in other NMIBC populations and is active across North America, Europe, and the Asia-Pacific region.
CEO Ron Cooper stated that RMAT recognition supports the potential for detalimogene to become a first-in-class treatment option for bladder cancer patients with few alternatives.
Currently, ENGN is trading at $3.00, up by 0.33 percent on the Nasdaq.
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