New data from the Phase 3 ENVISION trial reveals promising long-term efficacy for UGN-102 (mitomycin) in patients with low-grade intermediate-risk non-muscle-invasive bladder cancer (LG-IR-NMIBC). Published in the Journal of Urology, the study highlights an 82.3% duration of response (DOR) at 12 months in patients who achieved a complete response (CR) after UGN-102 treatment.
The ENVISION trial, a single-arm, multinational, multicenter study, enrolled approximately 240 patients. Participants received six weekly intravesical instillations of UGN-102. The primary endpoint was the CR rate at three months, while a key secondary endpoint assessed durability over time in patients achieving CR.
High Complete Response and Durable Effect
The study demonstrated a 79.6% complete response rate at three months following the initial UGN-102 instillation (95% CI, 73.9%, 84.5%). Among those who achieved a complete response, the Kaplan-Meier estimate for DOR was 82.3% at 12 months (95% CI, 75.9%, 87.1%). Furthermore, DOR Kaplan-Meier estimates at 15 months and 18 months post-3-month CR were both 80.9% (95% CI, 73.9%, 86.2%).
Expert Commentary
"These data demonstrate that treatment with UGN-102 results in a clinically meaningful CR rate and that the durability of the response in patients with LG-IR-NMIBC is robust," said Sandip Prasad, MD, M.Phil., Director of Genitourinary Surgical Oncology, Morristown Medical Center/Atlantic Health System, NJ and Principal Investigator of the ENVISION trial. "This study adds to the mounting evidence supporting UGN-102 as a potentially valuable treatment option for patients with recurrent LG-IR-NMIBC."
UGN-102: A Potential Non-Surgical Option
UGN-102 is an innovative formulation of mitomycin designed for intravesical use. Utilizing UroGen’s proprietary RTGel technology, it allows for sustained release of the drug, prolonging exposure of bladder tissue to mitomycin. This approach aims to treat tumors non-surgically.
Regulatory Progress and Market Need
UroGen initiated a rolling New Drug Application (NDA) submission to the FDA in January 2024 and completed it in August. The FDA accepted the NDA for UGN-102 with a PDUFA goal date of June 13, 2025. If approved, UGN-102 could offer a significant advancement in treating LG-IR-NMIBC, which affects approximately 22,000 newly diagnosed patients and results in an estimated 60,000 recurrences annually in the U.S.
Safety Profile
The most common treatment-emergent adverse events (TEAEs) in the ENVISION trial included dysuria, hematuria, urinary tract infection, pollakiuria, fatigue, and urinary retention. TEAEs were generally mild to moderate in severity and resolved during the study. The safety profile was consistent with previous UGN-102 studies.
