SAN FRANCISCO—Nadofaragene firadenovec, a novel intravesical gene-mediated therapy, is a promising option for patients with high-grade nonmuscle-invasive bladder cancer (NMIBC) that fails to respond to bacillus Calmette-Guérin (BCG) therapy, an investigator reported at the 2020 Genitourinary Cancers Symposium.

In an open-label phase 3 trial that enrolled 157 patients with BCG-unresponsive high-grade NMIBC, most patients experienced a complete response (CR) within 3 months of starting treatment, lead investigator Stephen A. Boorjian, MD, of Mayo Clinic in Rochester, Minnesota, told attendees. In many patients, the CR remained durable to 1 year. Serious adverse events occurred in only 3 patients (1.9%): 1 case each of sepsis, syncope, and hematuria. “The drug was well tolerated with a highly acceptable safety profile,” he said.

Dr Boorjian and his collaborators evaluated safety of nadofaragene firadenovec in all 157 patients and efficacy in 151 patients (103 with carcinoma in situ (CIS) with or without stage Ta/T2 and 48 with papillary tumors). Investigators administered 75 mL of the drug once every 3 months for up to 4 doses in the initial 12 months, with additional dosing at investigators’ discretion. The primary outcome was CR at any time in the patients with CIS.

Among the 103 patients with CIS in the efficacy population, 55 (53.4%) achieved a CR, all by month 3 after treatment. Of these 55 patients, 25 (45.5%) remained free of high-grade recurrence at month 12, which was confirmed on protocol-mandated biopsy. Of the 48 patients with high-grade papillary tumors in the efficacy population, 35 (72.9%) and 21 (43.8%) were free from recurrence at 3 and 12 months, respectively.


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Most treatment-emergency adverse events (TEAEs) were transient, and included instillation site discharge (33.1%), fatigue (23.6%), bladder spasm (19.7%), urinary urgency (17.8%), and hematuria (16.6%).

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Two grade 4 TEAEs (sepsis and anaphylactic reaction) occurred, but neither was related to study drug. No grade 5 TEAEs developed.

Disclosure: FKD Therapies Oy provided research funding.

Reference

Boorjian SA, Dinney CPN. Safety and efficacy of intravesical nadofaragene firadenovec for patients with high-grade, BCG unresponsive nonmuscle invasive bladder cancer (NMIBC): Results from a phase III trial. Presented at the 2020 Genitourinary Cancers Symposium held February 13 to 15 in San Francisco. Abstract 442.

This article originally appeared on Renal and Urology News