UGN-101 associated with favorable long-term durability in low-grade UTUC

Treatment with UGN-101 (Jelmyto) for pyelocalyceal solution was associated with a median duration of response of nearly 4 years among patients with low-grade upper tract urothelial carcinoma (UTUC) who achieved a complete response in the OLYMPUS trial (NCT02793128), according to long-term data published in the Journal of Urology.¹

The BL007 study is still ongoing.

UGN-101 was approved by the FDA in April 2020 based on data from the phase 3 OLYMPUS trial, which showed favorable efficacy of the therapy in UTUC.

"These results provide compelling evidence of the durability of Jelmyto in treating low-grade upper tract urothelial cancer," said Phillip Pierorazio, MD, Chief of the section of urology at Penn Presbyterian Medical Center and professor of surgery at the Hospital of the University of Pennsylvania in Philadelphia.² "The median duration of response of nearly 4 years in patients who achieved a complete response underscores the potential of Jelmyto to offer sustained control of the disease. These data reinforce the value of Jelmyto as an important treatment option for patients with LG-UTUC.”

In total, the OLYMPUS trial enrolled 71 patients (median age, 71 years), of whom 42 achieved a complete response at 4 to 6 weeks following at least 1 instillation of the therapy. Of those, 41 patients were entered quarterly follow-up after their initial complete response. The median follow-up for these patients was 28.1 months (95% CI, 13.1-57.5 months).

Among the 41 participants who achieved a complete response during the OLYMPUS trial, the median duration of response was 47.8 months (95% CI, 13.0-not estimable), per Kaplan-Meier estimate. Among all patients, 29 (70.7%) received at least 1 dose of maintenance therapy. There were a total of 10 (24.4%) disease recurrence events and 6 (14.6%) deaths. No deaths were attributed to study treatment.

The investigators then conducted a long-term follow-up trial (BL007) to assess outcomes among the patients who maintained a complete response by the end of the OLYMPUS trial at 12 months (n = 23). Of these, 20 were enrolled in the long-term follow-up study. During the follow-up study, eligible patients received standard of care treatment, with data collection conducted for up to 5 years. The median follow-up was 53.3 months (95% CI, 27.9-65.3 months).

Among the 20 patients included in the follow-up study, the median duration of response was not estimable (95% CI, 43.5-not estimable). In total, 16 patients (80%) had received maintenance therapy. There were 2 (10%) disease recurrence events and 3 (15.0%) deaths. Six patients had an ongoing complete response at data report.

"Prior research has emphasized that the main treatment objectives for LG-UTUC should focus on preventing relapse and preserving organ function, given the low likelihood of disease progression," explained Mark Schoenberg, MD, Chief Medical Officer of UroGen, in the news release.² "We are pleased with the study's results, which offer strong evidence supporting the long-term effectiveness of JELMYTO as a primary treatment for LG-UTUC, with increasing data showing extended response duration."

The BL007 study is still ongoing. In addition to that follow-up, UGN-101 is being further assessed in a prospective registry that is collecting longitudinal real-world usage data in patients with UTUC. As of January 27, 2025, the JELMYTO uTRACT Registry (NCT05874921) had enrolled 228 participants across 21 clinical sites, with a total enrollment goal of 400 patients.³

References

1. Pierorazio PM, Kleinmann N, Shabsigh A, et al. Long-term outcomes of primary chemoablation of low-grade upper tract urothelial carcinoma with UGN-101, a mitomycin reverse thermal gel. J Urol. 2025;213(3):313-322. doi:10.1097/JU.0000000000004331

2. Data from a long-term follow-up study to the OLYMPUS trial that shows a median duration of response of nearly four years in patients achieving complete response with JELMYTO published in The Journal of Urology. News release. UroGen Pharma Ltd. Published online and accessed February 12, 2025. https://www.businesswire.com/news/home/20250212280620/en/Data-from-a-Long-Term-Follow-up-Study-to-the-OLYMPUS-Trial-that-Shows-a-Median-Duration-of-Response-of-Nearly-Four-Years-in-Patients-Achieving-Complete-Response-with-JELMYTO-Published-in-The-Journal-of-Urology

3. uTRACT Jelmyto Registry: A registry of patients with upper tract urothelial cancer (UTUC) treated with Jelmyto (uTRACT). ClinicalTrials.gov. Last updated December 5, 2023. Accessed February 12, 2025. https://clinicaltrials.gov/study/NCT05874921