On February 12, 2025 UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to developing and commercializing novel solutions that treat urothelial and specialty cancers, reported results 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, FEB 12, 2025, View Source [SID1234650214]). Among patients from the OLYMPUS trial who achieved a complete response after primary chemoablation with JELMYTO (n=41, 20 of whom entered the long-term follow-up study), the median duration of response was 47.8 months (median follow-up 28.1 months [95% CI 13.1, 57.5]). The study results are published in the March issue of The Journal of Urology.
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"These results provide compelling evidence of the durability of JELMYTO in treating low-grade upper tract urothelial cancer," said Phillip Pierorazio, M.D., Chief, Section of Urology, Penn Presbyterian Medical Center, Professor of Surgery, Hospital of the University of Pennsylvania in Philadelphia, P.A. "The median duration of response of nearly four 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."
Of the 71 patients enrolled in OLYMPUS, 41 achieved a complete response after treatment with JELMYTO and had a median duration of response of 47.8 months (95% CI 13.0, not estimable), with median follow-up of 28.1 months (95% CI 13.1, 57.5).
"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," said Mark Schoenberg, M.D., Chief Medical Officer of UroGen. "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 analysis has certain limitations, including its post-hoc nature and the inherent selection bias of the 20 patients enrolled in the long-term follow-up study.
To further explore the potential of JELMYTO in treating patients with LG-UTUC, investigators are 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.
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 low-grade-UTUC (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 (UTUC)
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.