New Real-World Study on Retrograde Instillation of JELMYTO for Treatment of Low-Grade Upper Tract Urothelial Cancer Reports Similar Complete Response Rate and Lower Stricture Rate Compared to OLYMPUS Pivotal Trial

UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to developing and commercializing novel solutions that treat urothelial and specialty cancers, today highlights 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. 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.

“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.