UGN-102 for Recurrent Low-Grade Intermediate-Risk Nonmuscle-Invasive Bladder Cancer: 24-Month Duration of Response Results from the Phase 3 ENVISION Trial.

PURPOSE:We report the duration of response to UGN-102 treatment in patients with recurrent low-grade intermediate-risk nonmuscle-invasive bladder cancer (LG-IR-NMIBC) 24 months after achieving complete response (CR). MATERIALS AND METHODS:ENVISION is an ongoing, multinational, single-arm, phase 3 study in patients with biopsy-proven recurrent LG-IR-NMIBC. Patients received 6 once-weekly intravesical instillations of UGN-102. Patients underwent cystoscopy, urine cytology testing, and for-cause biopsy to determine bladder cancer presence at 3 months. Duration of response (from CR at 3 months to disease recurrence, progression, or death from any cause) at 24 months was estimated using the Kaplan-Meier method. RESULTS:A total of 240 patients were enrolled and received ≥ 1 dose of UGN-102. Most patients were White (98%), male (61%), and aged ≥ 65 years (68%). At 3 months, 191 patients (80%; 95% CI: 74-85) achieved CR. Among these responders, the probability of remaining event-free 24 months after CR was 72% (95% CI: 64-79; Kaplan-Meier estimate). Among patients with CR at 3 months (n = 191), 36 developed low-grade disease recurrence. A treatment-emergent adverse event that occurred in ≥ 10% of enrolled patients (N = 240) was dysuria. CONCLUSIONS:Patients who achieved CR after UGN-102 treatment had a high probability of remaining event-free 24 months later. These data indicate that UGN-102 is a novel, nonsurgical treatment that provides a durable CR for patients with recurrent LG-IR-NMIBC. Study limitations included the single-arm design and the limited racial diversity of the patients. TRIAL REGISTRATION:ClinicalTrials.gov: NCT05243550.