On January 22, 2025 UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers, reported results from a study on the durability of response from the first and largest post-commercialization study of JELMYTO (mitomycin) for pyelocalyceal solution (Press release, UroGen Pharma, JAN 22, 2025, View Source [SID1234649829]). The long-term study evaluated 56 patients who achieved complete response after treatment with JELMYTO from 15 high-volume academic and community centers and helps characterize how urologists are now using JELMYTO in their practices. This long-term study titled, "Durability of Response of UGN-101: Longitudinal Follow-Up of Multicenter Study," is published online in Urologic Oncology: Seminars and Investigations.
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"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 Solomon L. Woldu, MD, Assistant Professor of Urology, UT Southwestern Medical Center, Dallas, Texas, and study investigator. "Notably, we found that recurrence-free survival 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."
In this study on durability of response, 68% of patients with LG-UTUC who initially responded to JELMYTO had no evidence of disease recurrence at 3 years, as evaluated via endoscopy. The median follow-up was 23.5 months. RFS did not vary significantly based on use of JELMYTO for chemoablative versus adjuvant intent, tumor location (pyelocalyceal versus ureteral), tumor size before induction, single versus multiple tumors, or JELMYTO administration route (antegrade versus retrograde). The administration of maintenance treatment did appear to be associated with significantly better RFS, however, only 15 patients received maintenance therapy and, according to the authors, further study is required to determine the value of maintenance treatments.
"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, M.D., Chief Medical Officer, UroGen. "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."
The limitations of this study include the retrospective design, lack of a control group, and the lack of a centralized pathology review. Further study is needed to better understand the long-term outcomes of JELMYTO and the risks/benefits of maintenance therapy in this setting. In the phase 3 OLYMPUS study, the safety and efficacy of JELMYTO was not investigated in the adjuvant setting (although tumor debulking was permitted prior to study entry), patients with ureteral tumors and tumors larger than 15 mm were excluded, administration was limited to the retrograde technique, and complete response to treatment was assessed via urine cytology, ureteroscopy and biopsy (when warranted). Due to the risks associated with JELMYTO treatment following endoscopic ablation of UTUC or following placement of a nephrostomy tube for JELMYTO instillation (antegrade administration), an appropriate time interval consistent with institutional guidelines and standard medical practice should precede treatment with JELMYTO.
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.