UGN-101 shows durable responses in low-grade upper tract urothelial carcinoma

UGN-101 (JELMYTO [mitomycin] for pyelocalyceal solution) was associated with sustained disease control through extended follow-up among patients with low-grade upper tract urothelial carcinoma (LG-UTUC) who achieved a complete response (CR) following treatment in the phase 3 OLYMPUS trial (NCT02793128), according to findings from a 5-year observational follow-up study published in Therapeutic Advances in Urology.¹

Among 20 patients who enrolled in the long-term follow-up study, 75% (15 of 20) remained event-free as of March 2025, and no progression to high-grade disease was observed.

"These five-year findings further demonstrate the long-term durability of response achieved with JELMYTO in adults with low-grade upper tract urothelial cancer," said lead author Phillip Pierorazio, MD, chief of urology at Penn Presbyterian Medical Center and professor of surgery at the Hospital of the University of Pennsylvania, in a news release on the results.² "The sustained disease control observed during extended follow-up provides additional evidence supporting the long-term benefits of JELMYTO."

JELMYTO received FDA approval in April 2020 for the treatment of adults with LG-UTUC. The approval was based on findings from OLYMPUS, an open-label, single-arm, multicenter phase 3 study evaluating UGN-101 as primary chemoablation.

Long-term outcomes from OLYMPUS

The BL007 study was a noninterventional, observational follow-up of patients who participated in OLYMPUS and achieved a CR following UGN-101 treatment. Patients were eligible for BL007 if they maintained an event-free status for 12 months after achieving CR at the 3-month evaluation in OLYMPUS. Participants were followed for up to 5 years or until disease recurrence, progression, or death. According to the authors, baseline characteristics for the 20 patients in BL007 were generally consistent with the overall OLYMPUS trial population (n = 71).

Among the 41 patients who achieved a CR at the 3-month evaluation in OLYMPUS, the estimated median duration of response (DOR) was 47.8 months, with a median follow-up of 28.1 months (95% CI, 13.1 to 60.1). Twenty of these patients subsequently entered BL007, representing approximately 49% of the original CR population.

In the BL007 cohort, the median follow-up was 53.3 months. Median DOR was not estimable (95% CI, 43.5 to not estimable), reflecting the low event rate. At the March 21, 2025, data cutoff, 15 of 20 patients (75%) remained event-free, including 5 patients with a known ongoing CR. Among the 5 patients who experienced an event during study follow-up, there were 2 recurrences (10%) and 3 deaths unrelated to study treatment (15%).

Per Kaplan-Meier estimates, the probability of remaining event-free at 24, 36, and 72 months was 94% (95% CI, 66.6 to 99.2), 88% (95% CI, 60.2 to 96.9), and 59% (95% CI, 25.3 to 81.4), respectively. No patients experienced progression to high-grade disease during the extended follow-up.

The study's findings extend previously reported OLYMPUS results, which established the initial efficacy of UGN-101 as a primary chemoablative treatment for LG-UTUC. According to the authors, these long-term follow-up study results continue to support UGN-101 as a kidney-sparing treatment option in this setting.

The uTRACT registry (NCT05874921) is currently enrolling patients with UTUC who have received UGN-101 to provide further insights into the efficacy, safety, and utilization of the agent in a real-world setting.

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REFERENCES

1. Pierorazio PM, Hu B, Raman JD, et al. Five-year outcomes following primary chemoablation with UGN-101 in low-grade upper tract urothelial carcinoma: Evidence of durable complete response from an observational long-term follow-up of the phase 3 Olympus trial. Therapeutic Advances in Urology. 2026. doi:10.1177/17562872261493697

2. UroGen’s Final Results from Olympus Five-Year Follow-Up Study Reinforces Durable Response with JELMYTO in Patients with Low-Grade Upper Tract Urothelial Cancer. News release. UroGen. October 28, 2026. Accessed November 3, 2026. https://www.globenewswire.com/news-release/2026/10/28/3390117/0/en/urogen-s-final-results-from-olympus-five-year-follow-up-study-reinforces-durable-response-with-jelmyto-in-patients-with-low-grade-upper-tract-urothelial-cancer.html