On May 4, 2024 UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to developing and commercializing novel solutions that treat urothelial and specialty cancers, reported the results of a sub-analysis from a real-world patient cohort review of JELMYTO (mitomycin) for pyelocalyceal solution presented at the American Urological Association Meeting 2024 in San Antonio, TX (Press release, UroGen Pharma, MAY 4, 2024, View Source [SID1234642644]). The study includes a stepwise approach to retrograde administration of JELMYTO and reports that retrograde administration of JELMYTO in the clinic (n=20) produced a 60% complete response rate in patients and ureteral stents were placed in 25% of patients, which is lower than the rate reported in the pivotal OLYMPUS study.

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"The retrograde instillation of JELMYTO for the clinical management of low-grade upper tract urothelial cancer is FDA-approved and offers a safe and efficacious mode of administration, and this study in a real-world population adds to the growing body of evidence supporting retrograde administration in appropriate patients," said Khurshid Ridwan Ghani, MBChB, MS, FRCS, Professor of Urology, Director, Michigan Urological Surgery Improvement Collaborative. "The stepwise approach outlined in this study offers valuable insights into achieving positive efficacy and safety outcomes, in the clinic with the procedure done under local anesthesia, with notable durability of response observed."

In the OLYMPUS study all enrolled patients (n=71) received retrograde administration of JELMYTO. In the OLYMPUS study 58% (n=41) of patients achieved a complete response, defined as the absence of tumor lesions 3 months after initiation of JELMYTO treatment assessed by urine cytology and ureteroscopy. Ureteric obstruction* was reported in 58% (n=41) of patients treated with JELMYTO and 88% (n=36) of that subgroup went on to receive ureteral stent placement. For additional information about the retrograde administration of JELMYTO, consult the JELMYTO Instructions for Administration accompanying the Full Prescribing Information.

In this retrospective real-world study, 20 patients with a mean tumor burden of 1.67 cm received at least one dose of JELMYTO via the retrograde mode of administration, of which 16 (80%) completed six instillations. Twelve patients (60%) had a complete response and seven patients (35%) received at least one dose of monthly maintenance therapy, of which five demonstrated durability of response (4 patients remained tumor-free at 14.25-month follow-up and 1 patient at 24 months). Six patients (30%) had an adverse event related to the urinary system. Ureteral stents were placed in five patients for stenosis (25%); of which four were transient, with no subsequent obstruction. Only one patient (5%) required permanent stenting. Three patients who were unable to tolerate the retrograde approach had antegrade administration of JELMYTO via a nephrostomy tube.

"These findings emphasize the versatility of JELMYTO administration and also underscore the significance of continuous research and innovation in elevating treatment standards for urothelial cancers," remarked Mark Schoenberg, M.D., Chief Medical Officer at UroGen.

The limitations of this sub-analysis include the small sample size, the retrospective design, lack of a control group, and the lack of a centralized pathology review and standardized clinicopathologic assessment.

To further explore the full potential of JELMYTO for the treatment of patients with upper tract urothelial cancers (UTUC), investigators are in the process of enrolling the prospective and retrospective uTRACT Registry to capture data in a large-scale, standardized manner to report further on patient outcomes following JELMYTO treatment including longitudinal follow-up.

*Includes hydronephrosis, obstructive uropathy, pelvic-ureteric obstruction, ureteric obstruction, ureteric stenosis, and urinary tract obstruction.

About JELMYTO

JELMYTO (mitomycin) for pyelocalyceal solution is a mitomycin-containing reverse thermal gel containing 4 mg mitomycin per mL gel indicated 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 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 face comorbidities. There are limited treatment options for UTUC, with the most common being endoscopic surgery or nephroureterectomy (removal of the entire kidney and ureter). These treatments can lead to a high rate of recurrence and relapse.