UroGen Pharma's Jelmyto (mitomycin) demonstrates promising long-term efficacy in treating low-grade upper tract urothelial cancer (LG-UTUC), with a significant proportion of patients experiencing durable recurrence-free survival three years after treatment. The findings from the Durability of Response of UGN-101: Longitudinal Follow-Up of Multicenter Study, published in Urologic Oncology: Seminars and Investigations, represent the first and largest post-commercialization evaluation of Jelmyto's long-term efficacy.
Long-Term Efficacy of Jelmyto
The study, a retrospective review across 15 academic and community centers, assessed patients treated with Jelmyto who achieved a negative endoscopic evaluation following adjuvant or chemoablative therapy. Results indicated that 68% of patients demonstrated recurrence-free survival (RFS) at three years. Notably, RFS outcomes remained consistent irrespective of tumor size, location, or treatment intent. However, the use of maintenance therapy appeared to correlate with improved outcomes, though this observation requires further investigation due to the small number of patients (15) who received it.
Clinical Significance and Expert Commentary
Solomon L. Woldu, MD, assistant professor of urology at UT Southwestern Medical Center, emphasized the potential of Jelmyto for long-term disease control in LG-UTUC patients. He noted that the consistent RFS across various tumor characteristics highlights the broad applicability of Jelmyto. Mark Schoenberg, MD, chief medical officer at UroGen, expressed excitement about the three-year results, underscoring Jelmyto's promising long-term potential in managing this challenging disease.
Study Details and Limitations
The study enrolled 71 patients in the OLYMPUS trial, with 41 achieving a complete response after Jelmyto treatment. The median duration of response was 47.8 months, with a median follow-up of 28.1 months. Jelmyto, a pyelocalyceal solution, is administered via ureteral catheter or nephrostomy tube, transforming from a liquid to a semi-solid gel at body temperature to allow sustained release of mitomycin. Limitations of the study include its retrospective design, the absence of a control group, and the lack of centralized pathology review.
Ongoing Research and Future Directions
UroGen Pharma is currently enrolling participants in the Jelmyto uTRACT Registry to gather longitudinal real-world usage data. As of January 27, 2025, 21 sites have been activated with 228 patients enrolled. Further research is required to determine the value of maintenance treatments and to evaluate the long-term effects of Jelmyto, including its safety and efficacy in the neoadjuvant setting, as well as in patients with ureteral tumors and tumors larger than 15 mm, who were excluded from the Phase III OLYMPUS trial.
Disease Context
Urothelial cancer, commonly occurring in the renal pelvis or ureter, accounts for 90% of all bladder cancers and 7% of all kidney cancers. Over half of patients with urothelial cancer have the non-invasive form, where cancer remains in the urothelial cells lining the renal pelvis or ureters.
