Moyer on Early Efficacy Data With Nadofaragene Firadenovec in BCG-Unresponsive NMIBC

"At first assessment, 83% of [patients with] CIS had a CR. [CR rates were] was durable:74% at 6 months and 60% at 9 months. [The safety data] were pretty in line with [thoseseen in] the phase 3 [CS-003] trial [NCT02773849], and the overall survival rate was 100%.”

Jacob Moyer, BS, a graduate researcher at Mayo Clinic, discusses real-world data supporting the efficacy and safety of nadofaragene firadenovec-vncg (Adstiladrin) in patients with Bacilllus Calmette-Guérin (BCG)–unresponsive non–muscle-invasive bladder cancer (NMIBC).

Preliminary results from the study were presented at the 2025 Genitourinary Cancers Symposium and showed that at a median follow-up of 8.2 months, all evaluable patients with BCG-unresponsive NMIBC (n = 43) were alive, and the cystectomy-free survival rate was 95%. Among evaluable patients with carcinoma in situ (CIS) with or without papillary disease (n = 24), 79% (95% CI, 68%-92%) achieved a complete response (CR) at 3 months, Moyer reports.

At a median follow-up of 7.3 months, 84% of all evaluable responders maintained CRs, and the median duration of response (DOR) was not reached. The Kaplan-Meier–estimated DOR rates were 100% at 6 months and 75% at 9 months, Moyer continues. Among patients with papillary-only disease (n = 19), 68% were recurrence free at 3 months, and at a median follow-up of 8.9 months, 77% remained recurrence free, he adds. Three patients experienced disease progression: one from Ta to T1 disease, one from CIS to T2 disease, and one from Ta to metastatic disease.

Furthermore, the 6- and 9-month RFS rates in the efficacy-evaluable population were 66% (95% CI, 53%-82%) and 51% (95% CI, 36%-72%), respectively. For patients with CIS with or without high-grade Ta or T1 disease (n = 24), the 3-, 6-, and 9-month RFS rates were 83% (95% CI, 70%-100%), 74% (95% CI, 58%-94%), and 60% (95% CI, 41%-88%), respectively. These respective RFS rates for other key subgroups were as follows: high-grade Ta or T1 alone disease (n = 19; 74% [95% CI, 56%-96%]; 57% [95% CI, 38%-85%]; 40% [95% CI, 22%-75%]), prior receipt of pembrolizumab (Keytruda; n = 11; 73% [95% CI, 51%-100%]; 53% [95% CI, 30%-94%]; 20% [95% CI, 4%-99%]), and prior receipt of intravesical chemotherapy (n = 27; 70% [95% CI, 55%-90%]; 52% [95% CI, 36%-77%]; 36% [95% CI, 19%-69%]).

Regarding safety, adverse effects (AEs) were primarily low-grade and transient, Moyerbegins. No grade 4 or 5 AEs were reported. Grade 3 toxicities included fatigue (4%), fevers (2%), and dizziness (2%). Overall, 83% of patients experienced grade 1 or 2 AEs, including bladder spasm (61%), inability to retain nadofaragene firadenovec for the full target dwell time (33%), micturition urgency (20%), fatigue (13%), dysuria (13%), fevers (9%), hematuria (9%), dizziness (4%), and other AEs (26%).