UGN-103 Shows Durable Disease Control in LG-IR-NMIBC, Paving Path for FDA Submission

UGN-103 (mitomycin) for intravesical solution achieved a 94.5% durability of response rate at 6 months in the ongoing phase 3 UTOPIA trial (NCT06331299) in patients with recurrent low-grade intermediate-risk non–muscle-invasive bladder cancer (LG-IR-NMIBC), reinforcing UroGen’s plan to submit a new drug application (NDA) to the FDA for what could become a next-generation nonsurgical treatment option in this setting.¹

The 6-month estimated durability of response rate of 94.5% (95% CI, 86.1%-97.9%) was measured among patients who had achieved a complete response (CR) at 3 months.

"These clinical data reinforce the potential of UGN-103 to become a new standard of care for adult patients with recurrent LG-IR-NMIBC," Liz Barrett, president and chief executive officer of UroGen, stated in a news release.¹ "With FDA alignment on our regulatory path, we are advancing with urgency toward NDA submission. We believe UGN-103 represents a significant opportunity to build on our leadership in uro-oncology, expand our commercial portfolio, and drive long-term growth."

Building on the Initial 3-Month UTOPIA Data

Today's 6-month data follow the November 2025 announcement of the UTOPIA trial's primary end point results, in which UGN-103 achieved a 3-month complete response rate of 77.8% (95% CI, 68.3%-85.5%) among 99 enrolled patients across global sites.²

The UTOPIA trial is a single-arm, multicenter study evaluating the efficacy and safety of UGN-103 in patients with histologically confirmed recurrent LG-IR-NMIBC. Patients received 75 mg of UGN-103 administered as an intravesical instillation once weekly for 6 weeks, for a total of 6 doses. The primary end point was 3-month CR rate, with complete responders then entering a follow-up phase of up to 12 months to characterize durability of response. Secondary end points include durability of response, durable CR rate, safety, and mitomycin plasma concentration assessments.³

Eligibility required histological confirmation of LG NMIBC via cold cup biopsy at or within eight weeks of screening, at least one prior NMIBC episode, and intermediate-risk disease defined by no more than two of the following criteria: multiple tumors, a solitary tumor exceeding 3 cm, or early or frequent recurrence. Patients with BCG treatment for urothelial carcinoma within one year of study treatment, a history of high-grade bladder cancer within 2years, or clinically significant urethral stricture were excluded.³

UTOPIA Safety Profile for UGN-103

Safety data from the UTOPIA trial have been consistent with the established profile of mitomycin-based intravesical therapy. Adverse events were predominantly low grade. the The most commonly reported treatment-emergent adverse events occurring in at least 10% of patients treated in trials of UGN-103 have been dysuria, hematuria, urinary tract infection, and other lower urinary tract symptoms. Serious adverse events in UTOPIA were reported in 12% of patients, including urinary retention and urethral stenosis, both of which resolved. One patient experienced a fatal adverse event of cardiac failure considered unrelated to study treatment. Treatment discontinuations due to adverse events remained low, supporting the overall tolerability of the regimen.

Next Steps for UGN-103

UroGen plan an FDA NDA submission for UGN-103 before the end of 2026. The company also plans to pursue expansion of UGN-103 into high-grade NMIBC and the adjuvant intermediate-risk setting, with phase 3 trials in those settings anticipated to be initiated after NDA submission.

REFERENCES

1. UroGen Pharma Ltd. UroGen reports 94.5% six-month duration of response in phase 3 UTOPIA trial, advancing UGN-103 toward potential approval in recurrent low-grade intermediate-risk NMIBC. News release. UroGen Pharma Ltd; May 15, 2026. Accessed May 15, 2026. https://tinyurl.com/4nwfb7ns

2. UroGen Pharma Ltd. 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 May 15, 2026. https://tinyurl.com/34vzdy9y

3. UroGen Pharma Ltd. A phase 3 study of UGN-103 for treatment of patients with low-grade intermediate-risk non-muscle invasive bladder cancer (UTOPIA). ClinicalTrials.gov identifier: NCT06331299. Updated April 9, 2025. Accessed May 15, 2026. https://clinicaltrials.gov/study/NCT06331299