Data from the phase 3 ENVISION trial, published in the Journal of Urology, highlight the potential of UGN-102 (mitomycin) intravesical solution for patients with low-grade, intermediate-risk non-muscle-invasive bladder cancer (LG-IR-NMIBC). The study supports the new drug application (NDA) submitted to the FDA, which is currently under review with a target action date of June 13, 2025.
Key Findings from the ENVISION Trial
The ENVISION trial demonstrated a 79.6% complete response rate at 3 months following the first instillation of UGN-102 in patients with recurrent LG-IR-NMIBC. Updated data revealed that the 12-month duration of response (DOR) was 82.3% (95% CI, 75.9% to 87.1%) among patients who achieved a complete response at 3 months (n = 108), per Kaplan-Meier estimate.
Further analysis showed a DOR of 80.9% (95% CI, 73.9%–86.2%) at both 15 (n = 43) and 18 (n = 9) months, according to Kaplan-Meier estimates. With a median follow-up of 13.9 months, the median DOR was not estimable. At 15 months, 61% of patients (n = 146; 95% CI, 54 to 67) maintained a complete response following treatment, and disease-free survival at 15 months was 76% (95% CI: 69.7, 81.1) per Kaplan-Meier estimate.
According to principal investigator Dr. Sandip Prasad, MD, MPhil, “These data demonstrate that treatment with UGN-102 results in a clinically meaningful complete response rate and that the durability of the response in patients with LG-IR-NMIBC is robust. This study adds to the mounting evidence supporting UGN-102 as a potentially valuable treatment option for patients with recurrent LG-IR-NMIBC.”
Safety and Tolerability
The safety profile of UGN-102 in the ENVISION trial was consistent with previous reports. The most common treatment-emergent adverse events (TEAEs) included dysuria (22%), hematuria (8.3%), urinary tract infection (7.1%), pollakiuria (6.7%), fatigue (5.4%), and urinary retention (5.0%). The authors noted that TEAEs were generally mild to moderate in severity. Serious AEs occurred in 12.1% of patients, with only two being related to treatment (urinary retention/urethral stenosis), both of which were resolved.
Trial Design and Patient Population
The single-arm, multinational, multicenter ENVISION trial enrolled 240 adult patients across 56 sites in the United States and Europe to evaluate the safety and efficacy of UGN-102 as a primary chemoablative therapy in patients with LG-IR-NMIBC. Eligible patients had a negative voiding cytology for high-grade disease within 8 weeks before screening, adequate organ and bone marrow function, and an anticipated life expectancy of at least the duration of the trial. Participants received six once-weekly intravesical instillations of UGN-102, with 95% (228) receiving all six planned doses.
The primary endpoint was the complete response rate at 3 months following the first instillation. Secondary outcome measures include DOR, durable complete response rate, disease-free survival, and safety outcomes, assessed for up to 63 months. The trial is ongoing, with final study completion anticipated for 2028.
If approved, UGN-102, developed by UroGen Pharma, has the potential to become the first chemoablative, non-surgical treatment option for patients with LG-IR-NMIBC.
