The OPTIMA II phase 2b study (NCT03558503) treated patients with low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC) with UGN-102, a reverse thermal hydrogel containing mitomycin. Efficacy and safety results have been reported for the 12-month parent study; here, we report 5-year follow-up data.
Patients who participated in the OPTIMA II study and achieved a complete response (CR) after 6 weekly doses of UGN-102 were followed for up to 9 months after initial CR. Those with CR at study completion were eligible to enroll in a further long-term follow-up (LTFU) study, during which there were no protocol-specified interventions/treatments, protocol-specified visits, or evaluations. Supervising physicians provided semiannual updates on patients' disease status. Duration of response (DoR) was calculated using the Kaplan-Meier method.
Of the 41 patients achieving a CR at 3 months, 25 remained in CR at 12 months and 17 entered LTFU. For the 41 patients achieving a CR at 3 months the median Kaplan-Meier estimate of DoR was 24.2 months (95% confidence interval [CI], 9.72-42.09), with a median follow-up time of 35.8 months (95% CI, 10.78-60.98). For the 17 patients in the LTFU study the median DoR was 42.1 months (95% CI, 24.18-not estimable [NE]), with a median follow-up of 50.40 months (95% CI, 26.97-NE), CONCLUSION: These results demonstrate that treatment with UGN-102 results in clinically meaningful, and highly durable response in patients with LG-IR-NMIBC. UGN-102 may offer a promising non-surgical alternative to transurethral resection of bladder cancer (TURBT) for LG-IR-NMIBC patients.
Clinical genitourinary cancer. 2025 Jun 25 [Epub ahead of print]
Neal D Shore, K Kent Chevli, Daniel Saltzstein, Yaron Ehrlich, Jay D Raman, Boris Friedman, Alexander Sankin, Douglas Scherr, William C Huang, Michael J Louie, Sunil Raju, Brent Burger, Andrew Meads, Mark Schoenberg, OPTIMA II Study
START Carolinas Research, AUC Urology Specialists, Myrtle Beach, SC. Electronic address: ., Jacobs School of Medicine and Biomedical Sciences, University of Buffalo (State University of New York), Buffalo, NY., Division of Urology, Urology San Antonio, San Antonio, TX., Urology Unit, Rabin Medical Center, Petah-Tikva, Israel., Department of Urology, Penn State Health Milton S. Hershey Medical Center, Hershey, PA., Lady Davis Carmel Medical Center, Technion - Israel Institute of Technology, Haifa, Israel., Department of Urology, Albert Einstein College of Medicine, Bronx, NY., Department of Urology, Weill Cornell Medical College of Cornell University, New York, NY., Department of Urology, NYU Grossman School of Medicine, New York, NY., UroGen Pharma, Princeton, NJ., Department of Urology, Albert Einstein College of Medicine, Bronx, NY; UroGen Pharma, Princeton, NJ.