UGN-102 (intravesical mitomycin) has demonstrated durable responses in patients with low-grade, intermediate-risk non-muscle-invasive bladder cancer (LG-IR-NMIBC), according to data from the phase 3 ENVISION trial. The trial, which enrolled 240 patients, showed a 79.6% complete response (CR) rate at 3 months, meeting the primary endpoint. These results suggest that UGN-102 could offer a clinically meaningful and non-surgical treatment option for this patient population.
High Complete Response and Durability
The ENVISION trial data, published in the Journal of Urology, revealed that 61% of patients maintained a CR for 15 months after starting treatment. The Kaplan-Meier estimate for 12-month duration of response (DOR) was 82.3% in patients who achieved a CR at 3 months after the first instillation of UGN-102. At a median follow-up of 13.9 months, the median DOR was not estimable due to the lack of recurrence during the follow-up period. The Kaplan-Meier estimated disease-free survival rate was 76% at 15 months.
Expert Commentary
"These data from the ENVISION trial provide compelling evidence that treatment with UGN-102 achieves a clinically meaningful CR rate and also demonstrates remarkable durability in patients with LG-IR-NMIBC," said Sandip Prasad, MD, MPhil, director of Genitourinary Surgical Oncology at Morristown Medical Center/Atlantic Health System in New Jersey and principal investigator of the ENVISION trial. "The long-term results further strengthen UGN-102’s potential as a nonsurgical, effective treatment for patients facing the recurrent and challenging nature of LG-IR-NMIBC."
Safety and Tolerability
The most common treatment-emergent adverse events (TEAEs) observed in the ENVISION trial were dysuria, hematuria, urinary tract infection, pollakiuria, fatigue, and urinary retention. These TEAEs were generally mild-to-moderate in severity and either resolved or were resolving. The safety profile of UGN-102 in the ENVISION trial was consistent with that observed in other studies of the agent.
Potential Impact on Treatment Landscape
Mark Schoenberg, MD, chief medical officer of UroGen, stated, "The impressive DOR data from the ENVISION trial further highlight UGN-102’s potential to transform the treatment landscape for patients with LG-IR-NMIBC. Many of these patients are elderly and face the burden of repeated surgeries under general anesthesia, so there is a critical need for innovative treatment options for this patient population. We believe that, if approved, UGN-102’s ability to achieve durable CRs and potentially reduce recurrence rates while extending treatment-free intervals will represent a significant advance in managing LG-IR-NMIBC."
About the ENVISION Trial
UGN-102 is administered via catheter in an outpatient setting and utilizes a sustained-release hydrogel-based formulation to prolong drug exposure to bladder tissue without surgery. The ENVISION trial is an ongoing, multinational, single-arm trial evaluating the safety and efficacy of chemoablation with UGN-102 in patients with biopsy-proven recurrence of treatment-naive LG-IR-NMIBC. The study enrolled 240 patients across 56 global sites.
Regulatory Status
In October 2024, the FDA cleared the submission of a new drug application for UGN-102 in LG-IR-NMIBC, setting a Prescription Drug User Fee Act (PDUFA) goal date of June 13, 2025.
