PRINCETON, N.J.– UroGen Pharma Ltd. (Nasdaq: URGN) today announced The Journal of Urology® published results from the Phase 2b OPTIMA II trial, a Phase 2b, open-label, single-arm trial studying UGN-102 (mitomycin) for intravesical solution as primary non-surgical therapy for low-grade intermediate risk non-muscle invasive bladder cancer (LG IR-NMIBC). The study was published online and will be in the January print edition of The Journal of Urology®.
Approximately 50-60% of the 81,000 estimated new cases of non-muscle invasive bladder cancer (NMIBC) diagnosed in the U.S. in 2020 were low-grade. Patients with LG IR-NMIBC are chronically relapsing, and their only treatment option is repeated transurethral resection of bladder tumor (TURBT) with or without adjuvant chemotherapy. Some patients require multiple TURBT surgeries per year, which may lead to post-operative and long-term morbidity for this patient population.
The OPTIMA II Phase 2b results showed a significant treatment response with sustained durability in non-surgical chemoablation of LG IR-NMIBC. As previously reported, 65% (41/63) of patients receiving UGN-102 achieved a complete response (CR) three months after the start of therapy, of whom 39 (95%), 30 (73%), and 25 (61%) remained disease-free at 6, 9, and 12 months after treatment initiation, respectively. The probability of durable response nine months after CR (12 months after treatment initiation) was estimated to be 72.5% by Kaplan-Meier analysis. Thirteen patients had documented recurrences. 57 of 63 (90%) patients completed all 6 instillations of UGN-102 according to study protocol.
“The data from the OPTIMA II trial represent a significant advancement in the development of UGN-102 for the treatment of LG IR-NMIBC and further support the contention that non-surgical therapy for this disease is both technically feasible and clinically meaningful,” said Dr. Mark Schoenberg, Chief Medical Officer at UroGen. “With UGN-102, our goal is to change the treatment paradigm in LG IR-NMIBC by enabling non-surgical chemoablation. These results provide an important validation of our approach to treating low-grade disease, and our rationale for the continued evaluation of UGN-102 in the ongoing Phase 3 ATLAS study.”
Treatment with UGN-102 was generally well tolerated and the safety profile was consistent with previously reported results. The most common adverse events (≥ 10%) were generally reported as mild to moderate and included dysuria, urinary frequency, hematuria, micturition urgency, urinary tract infection, and fatigue.
“The significant treatment response and sustained durability observed in this trial indicate that UGN-102 has the potential to become a non-surgical alternative for these chronically relapsing patients, who typically undergo repetitive surgeries,” said William C. Huang, M.D., FACS, Professor of Urology and Radiology and Vice Chair of Urology at NYU Langone Health and Principal Investigator of the OPTIMA II trial. “Even more encouraging is that 12 months after treatment was initiated, approximately 73% achieved durable response.”
