Retrospective Study Finds JELMYTO Use Effective Following Partial Ablation or Biopsy in Larger Volume Low-Grade Upper Tract Urothelial Tumors that Impede Kidney Preservation

UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers, today highlights the results of a sub-analysis from the first post-commercial utilization review of JELMYTO (mitomycin) for pyelocalyceal solution. This study assessed the efficacy of JELMYTO in managing patients with larger volume disease where initial tumor burden may not be amenable to complete mechanical ablation or renal preservation. Findings from the study titled, The Ablative Effect of Mitomycin Reverse Thermal Gel: Expanding the Role for Nephron Preservation Therapy in Low Grade Upper Tract Urothelial Carcinoma, are published in Urologic Oncology: Seminars and Original Investigations online.

The goal of this study was to explore alternatives to nephroureterectomy for patients with higher-volume low-grade disease to preserve kidney function and minimize complications. This study aimed to determine if higher volume, low-grade upper tract urothelial tumors (LG-UTUC) patients could achieve disease-free status by undergoing partial ablation or biopsy only before treatment with JELMYTO during the initial ureteroscopy (URS) procedure.

In this study of 116 patients from 15 centers no significant differences were found in the rates of rendered disease free (RDF) among those who underwent complete ablation (78.6%), partial ablation (57.6%), or biopsy only (66.7%) during the initial URS procedure (p=0.15). RDF was defined as patients with absence of any tumor or an almost complete response requiring only minimal mechanical ablation to render the upper tract clear of visible disease at first URS after JELMYTO.

The analysis also showed that tumor size prior to JELMYTO induction did not have a significant impact on RDF rates (p=0.09). Tumor size was <1 cm for completely ablated, 1-3 cm for partial ablation or >3 cm for biopsy only. Among the 112 renal units evaluated, 50 underwent complete ablation, 42 underwent partial ablation and 20 underwent biopsy only.

In the Olympus trial, patients were required to have a tumor size of 5 to 15 mm prior to enrollment, and lesions larger than 15 mm were eligible for endoscopic downsizing prior to primary treatment with JELMYTO. Thirty-seven percent (37%) of patients who underwent endoscopic downsizing before enrollment and 58% of Olympus patients achieved a complete response with JELMYTO treatment, defined as the complete absence of tumor three months after initiation of JELMYTO treatment.