On April 27, 2025 UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to developing and commercializing novel solutions that treat urothelial and specialty cancers, reported a duration of response of nearly four years from a long-term follow-up study with JELMYTO (mitomycin) for pyelocalyceal solution, which is FDA-approved for the treatment of low-grade upper tract urothelial cancer (LG-UTUC) in adult patients (Press release, UroGen Pharma, APR 27, 2025, View Source [SID1234652198]).

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Among patients from the OLYMPUS trial who achieved a complete response after primary chemoablation with JELMYTO (n=41), the median duration of response was 47.8 months, irrespective of whether their cancer was new-onset or recurrent (median follow-up 28.1 months [95% CI 13.1, 57.5]). Of these patients, 21 had new-onset UTUC, and 20 had recurrent UTUC at baseline; there were no significant differences in durability between groups, with 8 patients in each group experiencing recurrence or death not due to treatment. Twenty patients entered long-term follow-up with a median follow-up of 53.3 months (95% CI 27.9, 65.3); median duration of response was not estimable (95% CI 43.5, not estimable) due to the low event rate.

"This sub-analysis highlights new evidence for the impressive long-term benefits of JELMYTO, in both new-onset and recurrent low-grade upper tract urothelial cancer patients," said Brian Hu, MD, Associate Professor of Urology at Loma Linda University Health, CA. "These findings further establish JELMYTO as an effective treatment, providing patients with durable responses and a significant reduction in recurrence rates."

LG-UTUC is a challenging disease often managed through endoscopically guided ablation, but recurrence is common, and patients face a lifetime of surveillance and potential complications. The OLYMPUS Phase 3 trial evaluated JELMYTO, a reverse thermal gel containing mitomycin (4 mg/mL), as a primary treatment for adults with LG-UTUC. While the overall trial demonstrated the potential of JELMYTO to significantly eradicate disease, this sub-analysis focuses specifically on patients with new-onset and recurrent LG-UTUC who achieved a complete response, providing new insights into the durability and long-term effectiveness of the treatment. Limitations of this study include the post-hoc nature of the analysis and potential selection bias regarding entry of patients into long-term follow-up.

"We are encouraged by the long-term outcomes evidence observed in both new-onset and recurrent LG-UTUC patients," said Mark Schoenberg, MD, Chief Medical Officer, UroGen. "The sub-analysis provides further compelling evidence that JELMYTO offers durable disease control and clinically meaningful responses, which could significantly reduce the need for repeated interventions. We look forward to expanding our understanding of JELMYTO through the ongoing uTRACT Registry and further evaluating its potential in real-world settings."

To further explore the potential of JELMYTO in treating patients with LG-UTUC, investigators are currently enrolling participants in the JELMYTO uTRACT Registry (NCT05874921) to gather longitudinal real-world usage data. As of April 2025, 22 sites have been activated with 251 patients enrolled.

About JELMYTO

JELMYTO (mitomycin) for pyelocalyceal solution is a mitomycin-containing reverse thermal gel containing 4 mg mitomycin per mL gel approved for the treatment of adult patients with LG-UTUC. JELMYTO is a viscous liquid when cooled and becomes a semi-solid gel at body temperature. The drug slowly dissolves over four to six hours after instillation and is removed from the urinary tract by normal urine flow and voiding. It is approved for administration in a retrograde manner via ureteral catheter or antegrade through a nephrostomy tube. The delivery system allows the initial liquid to coat and conform to the upper urinary tract anatomy. The eventual semisolid gel allows for chemoablative therapy to remain in the collecting system for four to six hours without immediately being diluted or washed away by urine flow.

About Upper Tract Urothelial Cancer

Urothelial cancer is the ninth most common cancer globally and the eighth most lethal neoplasm in men in the U.S. Between five percent and ten percent of primary urothelial cancers originate in the ureter or renal pelvis and are collectively referred to as UTUC. In the U.S., there are approximately 6,000 – 7,000 new or recurrent LG-UTUC patients annually. Most cases are diagnosed in patients over 70 years old, and these older patients often have multiple comorbidities. There are limited treatment options for UTUC, with the most common being endoscopic surgery or nephroureterectomy (removal of the entire kidney and ureter). Treatment with endoscopic surgery can be associated with a high rate of recurrence and relapse.