Jelmyto Demonstrates Significant Recurrence-Free Survival Rate at Three Years in Patients with Low-Grade Upper Tract Urothelial Cancer

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A majority of patients with low-grade upper tract urothelial cancer (LG-UTUC) who were treated with UroGen Pharma’s Jelmyto (mitomycin) experienced durable recurrence-free survival (RFS) three years after treatment. According to the company, the Durability of Response of UGN-101: Longitudinal Follow-Up of Multicenter Study represents the first and largest post-commercialization evaluation of the treatment’s long-term efficacy. Full results were published in Urologic Oncology: Seminars and Investigations.¹

"The three-year durability data from this study further validate the potential of Jelmyto in providing long-term disease control for patients with low-grade upper tract urothelial cancer,” said study investigator Solomon L. Woldu, MD, assistant professor of urology, UT Southwestern Medical Center, Dallas, Texas, in a press release. “Notably, we found that [RFS] was not influenced by factors like tumor size or location, highlighting the broad applicability of this treatment. The potential benefits of maintenance therapy are encouraging, and further research will be key in confirming its role in improving outcomes for these patients.”

Investigators conducted a retrospective review of patients treated with Jelmyto across 15 academic and community centers, identifying Patients with a negative endoscopic evaluation following either adjuvant or chemoablative Jelmyto. Additionally, data on recurrence and progression were reviewed.²

Results show that 68% of patients demonstrated RFS at three years. Notably, RFS outcomes were consistent regardless of tumor size, location, or intent of treatment; however, maintenance therapy was associated with improved outcomes. The authors of the study determined that moving forward, additional research will be required to determine the worth of maintenance treatments, as only 15 patients required them.

UroGen stated that the limitations of the study included the retrospective design, lack of a control group, and the lack of a centralized pathology review. The authors of the study stated that further research of Jelmyto is required in order to evaluate the treatment’s long-term effects. In the Phase III OLYMPUS trial, safety and efficacy were not explored in the neoadjuvant setting. Additionally, participants with ureteral tumors and tumors larger than 15 mm were excluded.¹

According to the Bladder Cancer Advocacy Network, urothelial cancer most commonly occurs in the renal pelvis or ureter. An estimated 5% to 7% of cases can occur in the inner lining of the kidney. It can also develop in both ureters. Over half of patients with urothelial cancer have the non-invasive form, in which cancer stays in the urothelial cells that line the renal pelvis or ureters. Less people have the invasive form, which grows beyond the urothelial cells and could spread to other parts of the body.³

Cleveland Clinic states that urothelial cancer accounts for 90% of all bladder cancers and 7% of all kidney cancers, with both having similar symptoms. Bladder cancer is currently the sixth most common form of cancer in the United States while kidney cancer is the eighth.⁴

"We are excited by the three-year results showing the durability of Jelmyto in treating low-grade upper tract urothelial cancer, with 68% of patients remaining recurrence-free,” said Mark Schoenberg, MD, chief medical officer, UroGen, in the press release. “These findings underscore the promising long-term potential of Jelmyto in managing this challenging disease. We are committed to improving the lives of patients with urothelial cancer and advancing our mission to deliver breakthrough therapies that transform the standard of care for patients with complex urological conditions."

References

1. New Real-World Durability of Response Data for JELMYTO Reports 68% Recurrence-Free Survival Rate (RFS) at Three Years Across a Broad Patient Population with Low-Grade Upper Tract Urothelial Cancer (LG-UTUC). BusinessWire. January 22, 2025. Accessed January 23, 2025. https://www.businesswire.com/news/home/20250122030643/en

2. Durability of response of UGN-101: Longitudinal follow up of multicenter study. ScienceDirect. Accessed January 23, 2025. https://www.sciencedirect.com/science/article/abs/pii/S1078143924010597?dgcid=author

3. Upper Tract Urothelial Carcinoma (UTUC). BCAN. Accessed January 23, 2025. https://bcan.org/what-is-upper-tract-urothelial-carcinoma-utuc/?gad_source=1&gclid=Cj0KCQiA7se8BhCAARIsAKnF3rywgoZ_QlXqYZUjtN_WW1UAJXn7wNdhxbUuwH9_X3q1stIOmX5WPsMaAtGkEALw_wcB

4. Urothelial Carcinoma (Transitional Carcinoma). Cleveland Clinic. Accessed January 23, 2025. https://my.clevelandclinic.org/health/diseases/6239-transitional-cell-cancer