Enrollment complete for phase 3 UTOPIA trial in LG-IR-NMIBC

Patient enrollment has been completed for the phase 3 UTOPIA trial (NCT06331299) evaluating UGN-103 (mitomycin) for intravesical solution for the treatment of recurrent low-grade intermediate risk non–muscle invasive bladder cancer (NMIBC), UroGen Pharma reported.¹

The trial will include a total of 99 patients at multiple centers, the company said in a news release.²

UTOPIA is a single-arm, multicenter study that aims to evaluate the efficacy and safety of UGN-103. Eligibility criteria include diagnosis of low-grade NMIBC (Ta disease) that is histologically confirmed via cold cup biopsy at screening or within 8 weeks prior to screening. Intermediate risk was defined as having 1 or 2 of the following criteria: presence of multiple tumors, solitary tumor larger than 3 cm, and early or frequent recurrence.Among the exclusion criteria for the study were receipt of BCG within the previous year, a history of high-grade bladder cancer (papillary or carcinoma in situ) in the past 2 years, past or current muscle-invasive bladder cancer, current T1 tumor stage, and concurrent upper tract urothelial carcinoma.²

Eligible patients will receive UGN-103 75 mg intravesically once per week for 6 weeks. The primary end point of the study is complete response (CR) at 3 months.¹ If patients have achieved a CR at 3 months, they will enter the follow-up period of the study, during which they will visit their clinic every 3 weeks to have their response evaluated.

“Patients will remain on study until disease recurrence, disease progression, death, or the last patient completes 12 months of follow-up (ie, 15 months after the first instillation), whichever occurs first,” according to the study’s ClinicalTrials.gov page.²

“Completing enrollment in the UTOPIA trial marks a significant milestone in our efforts to advance UGN-103 as a next-generation treatment option for patients with recurrent LG-IR-NMIBC,” said Michael Louie, MD, EVP, Medical Affairs and Clinical Development, UroGen. “UGN-103 represents a pivotal advancement in our pipeline, aiming to deliver the same non-surgical benefits to patients as Zusduri, while also enhancing operational efficiency and convenience for providers.”

The announcement of the UTOPIA enrollment comes less than a month after the FDA’s approval of mitomycin for intravesical solution (Zusduri; formerly UGN-102) for the treatment of adult patients with recurrent low-grade intermediate-risk NMIBC.³ That approval is primarily supported by findings from the phase 3 ENVISION trial (NCT05243550), which met its primary end point by demonstrating a 78% (95% CI, 72 to 83) complete response rate in patients with low-grade intermediate-risk NMIBC at 3 months following the first instillation of the therapy. The duration of response ranged from 0 months to over 25 months. Overall, 79% of responders remained in CR at 12 months.

The approval of mitomycin for intravesical solution marks the first FDA-approved medication in this disease setting, UroGen reported.⁴

REFERENCES

1. UroGen announces completion of enrollment in the phase 3 UTOPIA clinical trial of UGN-103 for the treatment of recurrent low-grade intermediate-risk non-muscle invasive bladder cancer. News release. UroGen Pharma. July 7, 2025. Accessed July 8, 2025. https://tinyurl.com/3cx2m95t

2. A phase 3 study of UGN-103 for treatment of patients with low-grade intermediate-risk non-muscle invasive Bladder cancer (UTOPIA). ClinicalTrials.gov. Updated April 9, 2025. Accessed July 8, 2025. https://clinicaltrials.gov/study/NCT06331299?term=NCT06331299&rank=1

3. FDA approves mitomycin intravesical solution for recurrent low-grade intermediate-risk non-muscle invasive bladder cancer. News release. US Food & Drug Administration. Published online June 12, 2025. Accessed July 8, 2025. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-mitomycin-intravesical-solution-recurrent-low-grade-intermediate-risk-non-muscle

4. 2. 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. Published online June 12, 2025. Accessed July 8, 2025. https://investors.urogen.com/news-releases/news-release-details/us-fda-approves-urogens-zusduritm-mitomycin-intravesical