Robust Response by UGN-103 in NMIBC Supports Future NDA Submission

Preliminary findings from the phase 3 UTOPIA trial (NCT06331299) show encouraging 3-month efficacy data for UGN-103, a novel mitomycin-based formulation, in the treatment of recurrent low-grade intermediate-risk non–muscle-invasive bladder cancer (NMIBC).¹

In particular, a complete response (CR) rate of 77.8% (95% CI, 68.3%-85.5%) was achieved at the 3-month mark with UGN-103 treatment. This finding is consistent with the 3-month CR rate (79.6%; 95% CI, 73.9%-84.5%) previously reported with the FDA-approved intravesical mitomycin (Zusduri; UGN-102) in the same indication from the phase 3 ENVISION trial (NCT05243550).

“The robust 77.8% three-month CR rate observed in the UTOPIA trial is highly encouraging and reinforces the potential of UGN-103 to deliver meaningful benefits to patients,” said Liz Barrett, president and CEO of UroGen Pharma, in a news release.¹

The FDA accepted the investigational new drug application for UGN-103 in April 2024. These new positive data position the agent to become a next-generation nonsurgical treatment option for this patient population, and both the sponsor and FDA have aligned on a potential pathway to new drug application (NDA) submission.

“The FDA’s agreement that the UTOPIA trial can support the submission of an NDA for UGN-103 represents a significant regulatory milestone and a strong validation of our clinical strategy. Together, these achievements give us significant momentum and a clear path toward potential approval, positioning UGN-103 as a key growth driver and marking an important advancement…in uro-oncology. We look forward to working closely with the FDA as we complete the UTOPIA trial and prepare for an NDA submission in 2026,” Barrett added.¹

Like UGN-102, UGN-103 is a chemoablative therapy that uses UroGen’s proprietary RTGel technology, a sustained-release hydrogel that allows for prolonged drug exposure with bladder tissue, which enables nonsurgical treatment. UGN-103’s design is specifically intended to streamline manufacturing and simplify preparation.

UTOPIA Trial Design

The phase 3 UTOPIA trial is a single-arm, multicenter study evaluating the efficacy and safety of UGN-103 in adult patients with low-grade NMIBC at intermediate risk of recurrence.² The study’s primary end point is 3-month CR rate, determined based on the presence of detectable disease in the bladder at the 3-month visit. Those who are determined to have a CR enter a follow-up period for response evaluation every 3 months, while those who do not begin standard-of-care treatment.

Secondary end points include duration of response, durable CR rate, incidence of treatment-emergent adverse events, and mitomycin plasma concentrations.

A total of 99 patients have been enrolled to receive 75 mg of UGN-103 once weekly for 6 weeks, and the study is actively recruiting.

REFERENCES

1. UroGen reports 77.8% three-month complete response rate from phase 3 UTOPIA trial of UGN-103 and receives FDA agreement on NDA submission strategy in recurrent LG-IR-NMIBC based on UTOPIA trial. News release. UroGen Pharma. November 6, 2025. Accessed November 6, 2025. https://tinyurl.com/3r77jd65

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 November 6, 2025. https://clinicaltrials.gov/study/NCT06331299