FDA grants RMAT designation to enGene’s bladder cancer therapy

BOSTON/MONTREAL - The U.S. Food and Drug Administration has granted Regenerative Medicine Advanced Therapy (RMAT) designation to enGene Holdings Inc.’s (NASDAQ:ENGN) lead therapy for treating high-risk bladder cancer, the company announced Wednesday. The clinical-stage biotech company, currently valued at $152.8 million, appears undervalued according to InvestingPro analysis.

The designation applies to detalimogene voraplasmid, an investigational treatment for high-risk, BCG-unresponsive, non-muscle invasive bladder cancer with carcinoma in situ.

RMAT designation provides enGene with regulatory advantages including early FDA engagement, potential for rolling review, and priority review. The program is designed to expedite development and review of regenerative medicine therapies for serious conditions where preliminary clinical evidence suggests potential to address unmet medical needs.

The FDA based its decision on preliminary results from enGene’s ongoing pivotal LEGEND study, which showed clinical activity and favorable tolerability in patients with BCG-unresponsive bladder cancer with carcinoma in situ.

"Receiving the RMAT designation highlights the promising profile of detalimogene and its potential to address the high unmet need in NMIBC," said Ron Cooper, Chief Executive Officer of enGene, according to the press release. The company maintains a strong financial position with a healthy current ratio of 12.66 and minimal debt-to-equity of 0.11. InvestingPro analysts have set price targets ranging from $7 to $30, suggesting significant upside potential.

Detalimogene is designed for administration in urology clinics, including community practices where approximately 70% of urologists provide care. The therapy aims to treat bladder cancer by stimulating a local immune response in the bladder without using viral vectors.

Non-muscle invasive bladder cancer accounts for 75-80% of new bladder cancer diagnoses. Patients with high-risk NMIBC who don’t respond to standard BCG treatment face high recurrence rates and may require complete bladder removal.

The LEGEND trial is currently enrolling patients across multiple cohorts in the United States, Canada, Europe, and the Asia-Pacific region. While the stock has experienced a 63% decline over the past year, InvestingPro subscribers can access detailed analysis of enGene’s financial health, which currently rates as ’FAIR’ with particularly strong cash flow metrics.

In other recent news, enGene Holdings Inc. reported its second-quarter 2025 financial results, revealing a net loss of $25.8 million and earnings per share of $(0.51), which was better than estimates by Raymond James. The company ended the quarter with approximately $252 million in cash, expected to fund operations into 2027. Analysts at H.C. Wainwright and JMP Securities reiterated their positive outlooks on enGene, maintaining buy and market outperform ratings with price targets of $25.00 and $18.00, respectively. These ratings are based on enGene’s progress in its pivotal LEGEND trial for high-risk non-muscle invasive bladder cancer and the potential for regulatory approval in Europe.

Additionally, enGene announced the adoption of its 2025 Employee Stock Purchase Plan, allowing employees to acquire company shares. The plan has reserved 2,000,000 common shares for issuance and aims to align employee and shareholder interests. In management news, Chief Medical Officer Dr. Raj Pruthi will step down in mid-June, with plans in place to ensure a smooth transition. The company continues to focus on advancing its lead program, detalimogene voraplasmid, in the LEGEND Phase 2 study. Lastly, enGene’s recent Annual General Meeting saw the re-election of its directors and the approval of its auditor, reflecting strong shareholder support.

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