Vignesh Packiam, MD, on the MOA, dosing of detalimogene voraplasmid

Detalimogene voraplasmid (formerly EG-70) is an investigational, non-viral gene-based immunotherapy designed to be instilled in the bladder to elicit an anti-tumor immune response. The agent was recently granted a Regenerative Medicine Advanced Therapy (RMAT) designation by the FDA for the treatment of patients with high-risk BCG-unresponsive non–muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS).¹

In a recent interview with Urology Times®, Vignesh T. Packiam, MD, outlined the mechanism of action (MOA) for the agent as well as potential benefits in terms of storage and dosing. Packiam is an associate professor of surgery at Rutgers Robert Wood Johnson Medical School as well as the director of clinical and translational research in urologic oncology at Rutgers Cancer Institute in New Jersey.

On the MOA for detalimogene, Packiam explained that the agent involves the use of a DDX nanoparticle, which enables penetration of mucosal tissues. He noted, “This is unique in that it does not require the use of an adenovirus or a different type of virus in order to cross the cell barrier.”

The agent also stimulates production of IL-12, which activates the adaptive immune system, plus 2 double stranded RNA products, which activate the innate immune system.

Packiam then walked through a couple of the potential benefits of detalimogene when used in clinical practice. For example, the storage, he says, is convenient for practices because the agent does not need to be stored at extremely low temperatures.

Further, he explains that the dosing regimen for detalimogene is consistent with other intravesical therapies, with the exception of 2 off weeks on weeks 3 and 4. Thus, the dosing schedule for detalimogene includes 4 doses over 6 weeks, given on weeks 1 and 2, off weeks on weeks 3 and 4, and then dosed again on weeks 5 and 6.

REFERENCE

1. FDA grants RMAT Designation for enGene’s detalimogene, enabling potential for expedited review in high-risk, non-muscle invasive bladder cancer. News release. enGene. June 25, 2025. Accessed July 2, 2025. https://www.businesswire.com/news/home/20250625944717/en/FDA-Grants-RMAT-Designation-for-enGenes-Detalimogene-Enabling-Potential-for-Expedited-Review-in-High-Risk-Non-Muscle-Invasive-Bladder-Cancer