Investigators highlight real-world data on nadofaragene firadenovec in NMIBC

In this video, Mark D. Tyson II, MD, MPH, and Jacob A. Moyer, BS, highlight the background and key findings from the study, “Real-world outcomes of nadofaragene firadenovec in BCG-unresponsive non-muscle invasive bladder cancer,” which were presented by Moyer at the 2025 American Society of Clinical Oncology Genitourinary (ASCO GU) Cancers Symposium in San Francisco, California. Tyson is a urologic oncologist at the Mayo Clinic in Arizona, and Moyer is a research intern at Mayo Clinic in Arizona.

Video Transcript:

Could you describe the background/rationale for this study?

Moyer: Since nadofaragene firadenovec was approved in December 2022, there hasn't been any published real-world data. Since, of course, real-world outcomes and clinical trial outcomes can vary at times due to screening criteria and differences between a clinical trial population and a real-world population, we conducted a real-world evaluation of patients treated with nadofaragene firadenovec at all 3 Mayo Clinic sites in Arizona, Rochester, and Florida. We looked at patients from the first dose given outside of a clinical trial in November 2023 up through December 2024 before we put the data together. All of our patients met the FDA-defined BCG-unresponsive criteria for non–muscle invasive bladder cancer.

What were the key findings?

Moyer: There were 46 patients treated during this time frame. Of those 46 patients, 3 had pending post-treatment cystoscopy. So, while all 46 were evaluated for safety outcomes, 43 were efficacy evaluable. Of those 43 patients, 24 had carcinoma in situ with or without papillary disease. At 3 months, those patients had a 79% complete response rate, which was durable in 74% at 6 months and 60% at 9 months. In the papillary only cohort, which had 19 patients, the high-grade recurrence-free survival was 68% at 3 months, which was durable at 57% at 6 months and 40% at 9 months. In terms of safety, 100% overall survival at a median follow-up of about 8 months.

What we did see was a lot of low-grade, transient and local side effects, usually during treatment, which resolved. Less commonly, we had 9% [of patients] with grade 3 adverse events, which included fatigue, fever, and dizziness. Generally, the safety profile was in-line with the clinical trial, which was low-grade and tolerable, although frequent.

Tyson: I would just add that the 3- and 6-month KM estimates are in the intention-to-treat population, and not in the complete response population. So, it wouldn't be 74% of 79% that responded. It would be 74% of the intention-to-treat population who were in complete response at 6 months, and then 60% at 9 months.

Moyer: Thanks for clarifying that. In fact, within the 79% of CIS patients with complete response at 3 months, 84% of those patients remained in complete response at the end of our 8 months of follow-up.

This transcript was AI generated and edited by human editors for clarity.