Updated ENVISION data show sustained efficacy of mitomycin intravesical solution for LG-IR-NMIBC

Updated data from the phase 3 ENVISION trial (NCT05243550) point to sustained efficacy of mitomycin for intravesical solution (Zusduri; formerly UGN-102) in patients with recurrent low-grade, intermediate-risk non–muscle invasive bladder cancer (LG-IR-NMIBC). Specifically, treatment with mitomycin intravesical solution led to a 24-month duration of response (DOR) of 72.2% (95% CI, 64.1 to 78.8) among patients who achieved an initial complete response to treatment at 3-month follow-up, UroGen reported in a news release.¹

Mitomycin for intravesical solution was granted FDA approval in June 2025 for adult patients with recurrent LG-IR-NMIBC based on initial data from the ENVISION trial.^2,3

“This latest update from the pivotal ENVISION trial of ZUSDURI showed that, among patients who achieved a complete response at 3 months, the probability of remaining event-free 24 months after CR was 72.2%,” said principal investigator Sandip Prasad, MD, MPhil, director of genitourinary surgical oncology and vice chair of urology at Morristown Medical Center/Atlantic Health System, NJ, in the news release.¹ “Among patients who achieved a complete response, the event rate over time has remained stable. This DOR result highlights the sustained efficacy of the therapy and reinforces its potential as a durable treatment option.”

The median follow-up time after a 3-month CR in the current analysis was 23.7 months. At the time of data report, the median DOR has not been reached.

The most common adverse events (AEs) in the trial included dysuria, increased potassium, increased creatinine, decreased hemoglobin, increased eosinophils, increased aspartate aminotransferase, increased alanine aminotransferase, decreased lymphocytes, urinary tract infection, decreased neutrophils, and hematuria. According to UroGen, AEs were generally mild to moderate in severity.

Serious AEs were reported in 12% of patients and included urinary retention (0.8%) and urethral stenosis (0.4%).

Additional data from ENVISION

The ENVISION trial initially met its primary end point by demonstrating a 78% (95% CI, 72 to 83) complete response rate in patients with LG-IR-NMIBC at 3 months following the first instillation of the therapy.² Overall, 79% of responders remained in CR at 12 months.

Data presented at the 2025 American Society of Clinical Oncology Annual Meeting in Chicago, Illinois,⁴ additionally showed an 18-month CR rate of 80.6% (95% CI, 74.0 to 85.7).

Overall, the single-arm, multinational ENVISION trial evaluated the safety and efficacy of mitomycin as a primary chemoablative therapy in patients with LG-IR-NMIBC. In total, the study enrolled 240 adult patients across 56 sites in the US and Europe.

Patients were eligible for enrollment in the trial if they had a negative voiding cytology for high-grade disease within 8 weeks before screening, adequate organ and bone marrow function, and an anticipated life expectancy of at least the duration of the trial.⁵ Those included in the study received 6 once-weekly intravesical instillations of mitomycin.

The primary end point for the study was the complete response rate at 3 months following the first instillation of the therapy. Secondary outcome measures included DOR, durable complete response rate, disease-free survival, and safety.

“For the first time, adult patients with recurrent LG-IR-NMIBC have an FDA-approved therapy,” Prasad added in the news release.¹ “This marks a potentially transformative step forward in how we manage this chronic, highly recurrent, and disruptive cancer. ZUSDURI is a new, outpatient treatment option for patients that can help provide a clinically meaningful recurrence-free interval, which is compelling given the highly recurrent nature of LG-IR-NMIBC.”

The ENVISION trial is ongoing to assess the long-term safety and efficacy of mitomycin for intravesical solution as part of a post-marketing commitment with the FDA, UroGen previously reported.³

REFERENCES

1. UroGen announces 24-month duration of response of 72.2% from the pivotal phase 3 ENVISION trial of ZUSDURI, the first and only FDA-approved medicine for recurrent low-grade intermediate-risk non-muscle invasive bladder cancer. News release. UroGen Pharma, Ltd. August 5, 2025. Accessed August 5, 2025. https://investors.urogen.com/news-releases/news-release-details/urogen-announces-24-month-duration-response-722-pivotal-phase-3

2. FDA approves mitomycin intravesical solution for recurrent low-grade intermediate-risk non-muscle invasive bladder cancer. News release. US Food & Drug Administration. June 12, 2025. Accessed August 5, 2025. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-mitomycin-intravesical-solution-recurrent-low-grade-intermediate-risk-non-muscle

3. U.S. FDA approves UroGen’s ZUSDURI (mitomycin) for intravesical solution as the first and only medication for recurrent low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC). News release. UroGen Pharma, Ltd. June 12, 2025. Accessed August 5, 2025. https://investors.urogen.com/news-releases/news-release-details/us-fda-approves-urogens-zusduritm-mitomycin-intravesical

4. Prasad SM, Mihaylov NV, Khuskivadze A, et al. Duration of response (DoR) following treatment with UGN-102 in patients with recurrent, low-grade, intermediate-risk, non-muscle invasive, bladder cancer: 18-month DoR data from the phase 3 ENVISION trial. J Clin Oncol. 2025;43 (suppl 16). https://doi.org/10.1200/JCO.2025.43.16_suppl.4598

5. A phase 3 single-arm study of UGN-102 for treatment of low grade intermediate risk non-muscle-invasive bladder cancer (ENVISION). ClinicalTrials.gov. Last updated November 4, 2024. Accessed August 5, 2025. https://clinicaltrials.gov/study/NCT05243550