The Lancet Oncology Publishes Details of UroGen Pharma’s Pivotal OLYMPUS Trial

  • 59% Complete Response in patients with low-grade upper tract urothelial cancer
  • Kaplan-Meier analysis, based on interim data, estimated 12-month durability at 84%
  • Results supported recent U.S. FDA expedited approval of Jelmyto™ as the first non-surgical treatment for patients with this difficult-to-treat cancer
San Francisco, CA ( -- UroGen Pharma Ltd. announced The Lancet Oncology published results from the pivotal Phase 3 OLYMPUS trial, reporting that 59% of low-grade upper tract urothelial cancer (LG-UTUC) patients treated with UGN-101, now referred to as Jelmyto™ (mitomycin) for pyelocalyceal solution, achieved a Complete Response (CR). Additionally, in the publication, durability at 12 months (based on interim data) was estimated to be 84% by Kaplan-Meier analysis.1

Jelmyto consists of mitomycin, an established chemotherapy, and sterile hydrogel, using UroGen’s proprietary sustained release RTGel™ technology. It has been designed to enable longer exposure of urinary tract tissue to mitomycin, thereby enabling the treatment of tumors by non-surgical means. Jelmyto is indicated for the treatment of adult patients with low-grade upper tract urothelial cancer (LG-UTUC). It is contraindicated in patients with perforation of the bladder or upper urinary tract.

“The current approach to treating low-grade upper tract urothelial cancer includes multiple endoscopic surgeries, high risk of disease recurrence and for many patients, the eventual removal of a kidney, which can present a whole new set of challenges that can adversely impact long-term health,” said Seth Lerner, M.D., FACS, Professor of Urology at Baylor College of Medicine in Houston, TX and Principal Investigator of the OLYMPUS trial. “Data from the OLYMPUS trial suggest UGN-101 is associated with a higher than expected initial complete response rate and strong durability without additional endoscopic surgery, which represents significant progress for patients living with this rare and difficult-to-treat type of cancer.”
LG-UTUC is a rare cancer that develops in the lining of the upper urinary tract, ureters, and kidneys. In the U.S., there are approximately 6,000 - 7,000 new or recurrent LG-UTUC patients annually. It is a challenging condition to treat due to the complex anatomy of the urinary tract system. The current standard of care includes multiple surgeries, and most patients require a radical nephroureterectomy, which includes the removal of the renal pelvis, kidney, ureter, and bladder cuff.2 Treatment is further complicated by the fact that LG-UTUC is most commonly diagnosed in patients over 70 years of age, who may already have compromised kidney function and may suffer further complications as a result of major surgery.

In the pivotal OLYMPUS trial, participants received six once-weekly instillations of Jelmyto via a retrograde catheter in the renal pelvis and calyces. The intent-to-treat (ITT) population included the seventy-one patients who received at least one dose of Jelmyto; 48% of these patients had tumors that were deemed endoscopically unresectable. As reported in The Lancet Oncology publication, Jelmyto achieved a Complete Response (CR) in 59% of the ITT population (a CR was defined as negative 3-month ureteroscopic evaluation, negative cytology and negative for cause biopsy) and durability at 12 months (at the time of the data cutoff) was estimated to be 84% by Kaplan-Meier analysis.

“The positive results of the OLYMPUS trial demonstrate that Jelmyto has the potential to help fulfill a significant unmet need for patients with low-grade upper tract urothelial cancer,” said Dr. Mark P. Schoenberg, Chief Medical Officer at UroGen. “We are committed to addressing treatment challenges for this underserved patient population and improving the standard of care for those who need it most.”

As reported in The Lancet Oncology publication, in the trial, 67 patients (94%) experienced adverse events (AEs) and 60 patients (85%) had AEs that were considered to be related to the study treatment or procedure. No treatment-related deaths occurred. Overall, the most frequently reported AEs were ureteric stenosis in 31 (44%) of 71 patients, urinary tract infection in 23 (32%), haematuria in 22 (31%), flank pain in 21 (30%), and nausea in 17 (24%).

The U.S. Food and Drug Administration (FDA) approved Jelmyto on April 15, 2020. The FDA-approved labeling for Jelmyto reports Complete Response (CR) (primary endpoint) of 58% in the intent-to-treat population. The product labeling also reports that at the 12-month time point for assessment of durability, 19 patients remained in CR, seven had experienced recurrence of disease, nine patients continued to be followed for the 12-month duration of response, and median duration of response was not reached as of the FDA-approval date.

Source: WIRE, BUSINESS. 2020. "The Lancet Oncology Publishes Details Of Urogen Pharma’S Pivotal OLYMPUS Trial". Businesswire.Com.


  1. Lerner, Seth. Primary chemoablation of low-grade upper tract urothelial carcinoma using UGN-101, a mitomycin-containing reverse thermal gel (OLYMPUS): a prospective single-arm phase 3 trial. The Lancet Oncology, 2020
  2. Browne BM, Stensland KD, Moynihan MJ, Canes D. An Analysis of Staging and Treatment Trends for Upper Tract Urothelial Carcinoma in the National Cancer Database. Clin Genitourin Cancer 2018;16:e743-e50.
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Read: Primary Chemoablation of Low-Grade Upper Tract Urothelial Carcinoma Using UGN-101, A Mitomycin-Containing Reverse Thermal Gel (OLYMPUS): An Open-Label, Single-Arm, Phase 3 Trial
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