UroGen Pharma Ltd. (NASDAQ:URGN) today announced the presentation of positive interim data from the Phase 2b OPTIMA II trial evaluating the safety and efficacy of investigational agent UGN-102 (mitomycin) for intravesical solution in patients with low-grade intermediate risk non-muscle invasive bladder cancer (LG IR-NMIBC). The study was accepted for the 2020 American Urological Association (AUA) Annual Meeting, published as a supplement to the April 2020 issue of The Journal of Urology® and presented as part of the AUA Virtual Experience. The presentation can be accessed via the AUA website here.
Trial results showed that 65% (41/63) of patients treated with UGN-102 achieved a complete response (CR) three months after the start of therapy. In this subset of patients, 97% (35/36) of patients (95% Confidence Interval), 86% (24/28) of patients and 85% (11/13) of patients who were present for evaluation at each timepoint, remained disease free at six, nine and 12 months following treatment initiation, respectively. Follow-up will continue until all patients have reached the 12-month time point.
“With UGN-102, we have an opportunity to fundamentally change the way low-grade intermediate risk non-muscle invasive bladder cancer is treated and help patients avoid recurrence of their cancer and repetitive surgeries,” said Dr. Mark Schoenberg, Chief Medical Officer at UroGen. “The positive data presented today, coupled with previously presented data in low-grade upper tract urothelial cancer, continue to validate our technology platform and our hypothesis that increased dwell time significantly improves the effectiveness of intravesical therapy.”
Non-Muscle Invasive Bladder Cancer (NMIBC) is stratified into three risk categories: low, intermediate, and high risk.1 Patients with low risk disease require relatively modest intervention, while those with high risk disease are at risk for disease progression necessitating organ removal. Patients with intermediate risk disease have a clinical course that is best characterized as chronically recurrent. These patients often undergo repetitive transurethral resection of bladder tumors (TURBT), which can lead to increased morbidity and risks of repetitive anesthesia. Some patients require more than one or two surgical procedures per year.2 It is estimated that 80,000 Americans suffer from intermediate risk NMIBC.3,4,5
“The current approach to treating patients with low-grade non-muscle invasive bladder cancer is transurethral surgery. Unfortunately, there is a high rate of recurrence and these patients often need repetitive surgeries, which can result in an increased risk of complications,” said William C. Huang, M.D., FACS, Associate Professor and Vice Chair of Urology at NYU Langone Health and Principal Investigator of the OPTIMA II trial. “The interim results from OPTIMA II indicate that UGN-102, if approved, may provide an effective, non-surgical treatment option for these patients that is both well-tolerated and durable.”
In OPTIMA II the most common adverse events (≥ 10%) were reported as mild to moderate and include dysuria, hematuria, urinary frequency, fatigue, urgency and urinary tract infection. Discussions for the planned Phase 3 clinical trial protocol for the study of UGN-102 in patients with LG IR-NMIBC are underway with the U.S. Food and Drug Administration.