Real-World Study on JELMYTO® for Low-Grade Upper Tract Urothelial Cancer Shows Similar Response and Lower Stricture Rate to OLYMPUS Trial

UroGen Pharma Ltd., a biotechnology company focused on developing innovative treatments for urothelial and specialty cancers, recently presented significant findings from a real-world patient cohort review of JELMYTO (mitomycin) for pyelocalyceal solution. The data was shared at the American Urological Association Meeting 2024 in San Antonio, Texas.

The study utilized a stepwise approach to retrograde administration of JELMYTO, which involved 20 patients. These patients showed a 60% complete response rate, with ureteral stents placed in only 25% of them—a lower rate compared to the pivotal OLYMPUS study. The OLYMPUS study had enrolled 71 patients, with 58% achieving a complete response, defined as the absence of tumor lesions three months post-treatment. Notably, 58% of the patients in the OLYMPUS study experienced ureteric obstruction, leading to stent placements in 88% of that subgroup.

Dr. Khurshid Ridwan Ghani, a Professor of Urology and Director at the Michigan Urological Surgery Improvement Collaborative, emphasized that the retrograde instillation of JELMYTO is FDA-approved and offers a safe and effective treatment option for low-grade upper tract urothelial cancer. He highlighted the valuable insights provided by the stepwise approach, which demonstrated positive efficacy and safety outcomes under local anesthesia, along with notable durability in response.

The real-world study reviewed 20 patients with an average tumor size of 1.67 cm who received at least one dose of JELMYTO. Out of these, 16 patients completed six instillations. Twelve patients, representing 60%, achieved a complete response. Seven patients received at least one dose of monthly maintenance therapy and showed durability of response, with four patients remaining tumor-free at a 14.25-month follow-up and one patient at 24 months.

Adverse events related to the urinary system were reported in 30% of the patients, with ureteral stents placed in five patients due to stenosis. Four of these cases were transient, requiring no further intervention, while only one patient needed permanent stenting. Three patients who couldn’t tolerate the retrograde approach were administered JELMYTO via a nephrostomy tube.

Dr. Mark Schoenberg, Chief Medical Officer at UroGen, remarked on the versatility of JELMYTO administration and stressed the need for ongoing research to elevate treatment standards for urothelial cancers. He acknowledged that the sub-analysis had limitations, including a small sample size, retrospective design, lack of a control group, and absence of a standardized clinicopathologic assessment.

To further understand JELMYTO's potential in treating upper tract urothelial cancers (UTUC), investigators are enrolling patients in the uTRACT Registry. This registry aims to capture large-scale, standardized data to monitor patient outcomes and provide longitudinal follow-up.

JELMYTO is a mitomycin-containing gel specifically designed for the treatment of adult patients with low-grade upper tract urothelial cancer (LG-UTUC). It is unique in that it transitions from a liquid to a semi-solid gel at body temperature, allowing it to remain in the urinary tract for four to six hours after administration. This prolonged presence helps in the chemoablative treatment of tumors.

UTUC is a subset of urothelial cancer, which is the ninth most common cancer worldwide. In the U.S., approximately 6,000-7,000 new or recurrent cases of LG-UTUC are diagnosed annually, primarily in patients over 70 years old who often have other health conditions. Current treatment options are limited and frequently result in high recurrence rates.

UroGen Pharma Ltd. is committed to developing and commercializing cutting-edge solutions for treating urothelial and specialty cancers. Their proprietary RTGel technology aims to enhance the effectiveness of existing drugs by ensuring longer exposure of the urinary tract tissue to the medication. UroGen is headquartered in Princeton, NJ, with additional operations in Israel.