Mitomycin intravesical solution demonstrates long-term durability in LG-IR-NMIBC

Treatment with mitomycin for intravesical solution (Zusduri, formerly UGN-102) was associated with durable, long-term complete responses (CRs) in patients with low-grade, intermediate-risk non–muscle invasive bladder cancer (LG-IR-NMIBC) who achieved an initial CR to treatment, according to data from a 5-year long-term extension study of the phase 2b OPTIMA II trial (NCT03558503).¹

Mitomycin intravesical solution was granted FDA approval in June 2025 for the treatment of adult patients with recurrent LG-IR-NMIBC.

Neal D. Shore, MD, FACS

“Low-grade intermediate-risk bladder cancer is a chronic, recurring disease that often requires repeated surgical intervention,” said lead author Neal D. Shore, MD, FACS, medical director for the START Carolinas/Carolina Urologic Research Center, in a news release on the findings.² “The long-term data from the extension study of OPTIMA II highlight ZUSDURI’s ability to deliver sustained responses in an outpatient setting, which may be especially valuable for recurrent patients and thus for physicians who prefer a different, non-surgical treatment option.”

The initial OPTIMA II trial enrolled 63 patients, of whom 41 achieved a complete response (CR) at 3-months following treatment. Among those who achieved a CR, 25 remained in CR at 12 months and 17 entered into the long-term follow-up study.

The median follow-up for the 63 patients initially enrolled in the trial was 38.77 months (95% CI, 12.62 to 63.47). Per Kaplan-Meier estimate, the disease-free survival was 11.27 months (95% CI, 7.85 to 25.92).

The median duration of response (DOR) was 24.2 months (95% CI, 9.7 to 42.1) among the 41 patients who achieved an initial CR, with a median follow-up of 35.8 months. The 12-month response rate was 43.8% (25 of 57).

Among the 17 patients in the long-term follow-up study, the median DOR was 42.1 months (95% CI, 24.2 to NE), and the median follow-up was 50.4 months. According to the authors, “Eight patients experienced recurrence of LG disease, 1 had progression to [high-grade] disease, 1 patient died (not due to bladder cancer), 5 discontinued the study, and 2 were still in CR at the end of the study period).”

In total, the OPTIMA II trial included 63 patients who received at least 1 dose of mitomycin intravesical solution and 57 patients who received all 6 doses. To be eligible for enrollment, patients needed to have recurrent or newly diagnosed LG-IR-NMIBC diagnosed using a cold cup biopsy and negative voiding cytology for high-grade disease.

The median age of patients was 68 years in the parent OPTIMA II trial and 70 years in the long-term follow-up study. Overall, 78% of patients in the parent study and 94% of patients in the follow-up study had recurrent disease.

Safety data were not collected during the long-term follow-up study.

Among all patients initially enrolled in the trial (n = 63), 91% of patients experienced an adverse event (AE) and 63% experienced at least 1 treatment-emergent adverse event (TEAE). The most common TEAEs included dysuria (41%), pollakiuria (21%), hematuria (16%), urgency or urinary tract infection (both, 14%), and fatigue (11%).

“These results reflect our continued commitment to bringing forward innovative treatments that give patients and physicians more options,” concluded Mark Schoenberg, MD, chief medical officer for UroGen, in the news release.² “For recurrent patients facing repeated surgeries, it offers a non-surgical approach that can empower patients and providers to choose a path that best fits individual needs and preferences.”

REFERENCES

1. Shore ND, Chevli KK, Saltzstein D, et al. Treatment of low-grade intermediate-risk non-muscle invasive bladder cancer with UGN-102: Outcomes from the 5-year long-term extension study of the single-arm, phase 2b Optima II study. Clinical Genitourinary Cancer. 2025. doi:10.1016/j.clgc.2025.102392

2. Long-term extension study of the OPTIMA II trial demonstrates long-term durability of response to ZUSDURI in patients with low-grade intermediate-risk non-muscle invasive bladder cancer. News release. UroGen Pharma Ltd. July 21, 2025. Accessed July 21, 2025. https://investors.urogen.com/news-releases/news-release-details/urogen-announces-five-year-long-term-extension-study-optima-ii