An investigational gene therapy may be effective in treating patients with high-grade nonmuscle-invasive bladder cancer (NMIBC) that does not respond to therapy with bacillus Calmette-Guérin (BCG), according to data from a pivotal phase 3 trial presented at the 20th annual meeting of the Society of Urologic Oncology in Washington, DC.

The 33-center trial of nadofaragene firadenovec, which included 157 patients, met its primary end point, with 53% of patients with carcinoma in situ (CIS) with or without concomitant high-grade Ta or T1 papillary disease achieving a complete response (CR) at 3 months and 24% continuing to show a CR at 12 months, Colin P. Dinney, MD, of The University of Texas MD Anderson Cancer Center in Houston, and colleagues reported. In addition, results revealed a 73% and 44% high-grade recurrence-free survival rate at 3 months and 12 months, respectively, among patients with papillary disease.

Common adverse events (AEs) associated with the treatment included fatigue, bladder spasm, micturition urgency, chills, fever, headache, discharge around the catheter, diarrhea, painful urination, and urinary tract infection. The investigators observed no grade 4 or 5 treatment-related AEs.

In a news release, Dr Dinney commented that the results are highly promising for patients, adding, “It would be gratifying to provide an alternative that addresses the critical unmet need for effective second-line therapy for patients facing radical cystectomy.”


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“As a practicing urologist and trial investigator, it’s encouraging to see these types of efficacy and safety results in patients with high-grade NMIBC, an area that’s been in need of new innovative treatment options for 20 years,” Neal Shore, MD, Medical Director, Carolina Urologic Research Center, Myrtle Beach, South Carolina, said in the same release. “These robust clinical results further demonstrate the potential of nadofaragene firadenovec as a valuable treatment options for NMIBC patients.”

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Nadofargene firadenovec is an adenovirus vector-based gene therapy containing the gene interferon alfa-2b. It is administered via catheter into the bladder every 3 months. The vector enters the cells of the bladder wall where it breaks down and releases the active gene.

The FDA has granted Fast Track and Breakthrough Therapy designation for the treatment.

The treatment is under development by FerGene, a company formed by Ferring Pharmaceuticals and Blackstone Life Sciences.