Interim data on UGN-103 show promising efficacy in LG-IR-NMIBC

Preliminary results from the phase 3 UTOPIA trial (NCT06331299) show encouraging early efficacy with UGN-103 (mitomycin) for intravesical solution in patients with recurrent low-grade intermediate-risk non–muscle invasive bladder cancer (LG-IR-NMIBC), UroGen Pharma reported in a news release.¹

Specifically, the agent demonstrated a 3-month complete response (CR) rate of 77.8% (95% CI, 68.3 to 85.5) in this patient population. According to UroGen, these results are consistent with the 79.6% (95% CI, 73.9 to 84.5) CR rate observed in the pivotal phase 3 ENVISION trial (NCT05243550), which led to the FDA approval of UGN-102 (mitomycin) for intravesical solution (Zusduri) in June 2025 for recurrent LG-IR-NMIBC.

The FDA has confirmed that CR and durability results from the phase 3 UTOPIA trial can support submission of a new drug application (NDA) for UGN-103 in this same space, as well.

“The robust 77.8% 3-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, in the news release.¹ “In addition, 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 expanding our leadership 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.”

The single-arm UTOPIA trial is assessing the safety and efficacy of UGN-103 in 99 patients across global clinical trial sites.² Patients are eligible for enrollment if they have histologically confirmed LG-NMIBC, intermediate-risk disease, and adequate organ and bone marrow function. Participants are not eligible for enrollment if they had received BCG within the prior year, had a current tumor stage of T1, or received prior treatment with an intravesical chemotherapeutic agent in the past 2 years aside from a single dose of chemotherapy following TURBT.

Patients enrolled in the study will receive intravesical instillation of 75 mg UGN-103 once weekly for 6 weeks.

The primary end point for the study is CR rate at 3 months. Responders will enter into follow-up assessment for up to 12 months to evaluate durability of response. Secondary outcome measures include duration of response, incidence of adverse events, and pharmacokinetic measures.

Final completion of the trial is expected in August 2026.

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 Ltd. November 6, 2025. Accessed November 6, 2025. https://investors.urogen.com/news-releases/news-release-details/urogen-reports-778-three-month-complete-response-rate-phase-3

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. Last updated April 9, 2025. Accessed November 6, 2025. https://clinicaltrials.gov/study/NCT06331299