Rare Disease Report

Phase 3 OLYMPUS Trial Interim Analysis for UTUC Shows Efficacy

MAY 22, 2018
Krista Rossi
Yesterday, UroGen Pharma Ltd released data from its interim analysis of the ongoing pivotal Phase 3 OLYMPUS clinical trial of UGN-101 (MitoGel), an investigational mitomycin formulation for the non-surgical treatment of low-grade upper tract urothelial cancer (UTUC).

Seth Paul Lerner, MD, FACS, Principal Investigator of the OLYMPUS trial and Professor of Urology at Baylor College of Medicine in Houston presented the results orally during a plenary session at the 113th American Urological Association’s (AUA) Annual Meeting in San Francisco.

The international, multi-center trial displayed a complete response (CR) rate of 59% in 34 patients who were evaluated for primary disease evaluation (PDE, or the primary endpoint) in the interim analysis. The UGN-101 treatment was administered once weekly for 6 weeks, and the PDE is conducted 4 to 6 weeks post completion of UGN-101 treatment.

“These interim data from the OLYMPUS trial are promising and demonstrate the potential of UGN-101 to become the first drug ever approved for low-grade UTUC. We are pleased to see that the results to date from this trial improve upon the 44 percent complete response rate on an intent to treat basis observed in our similarly designed Compassionate Use program of UGN-101,” stated Mark Schoenberg, MD, Chief Medical Officer of UroGen in a. recent statement.

“Currently, the thousands of patients living with UTUC do not have many therapeutic options to manage their disease. With UGN-101, we hope to change the treatment paradigm for low-grade UTUC by potentially enabling the treatment of these tumors by non-surgical means, sparing patients from the risks and complications associated with repetitive surgical procedures and potential kidney removal.”
UGN-101 is an investigational drug formulation of mitomycin. By utilizing its proprietary sustained release, hydrogel-based formulation, UGN-101 enables longer exposure of mitomycin C to the urinary tract tissue, which potentially enables the treatment of tumors by non-surgical means. Standard intravesical catheters are used to deliver UGN-101.


According to the data, 20 of the interim analysis intent to treat the population of 34 patients (59%) achieved a CR, defined as a negative ureteroscopic evaluation and a negative wash cytology. Five of 34 patients (15%) experienced a partial response, and 39%, roughly, of tumors treated were categorized as unresectable by surgery at baseline.

To date, the CRs have been durable, and 13 of the 20 subjects who experienced a CR have reached the 3-month follow-up and are all remaining in CR. Of these 13 patients, 4 have reached six-month follow-up, and 1 of the 13 patients has reached the 9-month follow-up. All subjects are currently remaining in CR. 

UGN-101 was generally well tolerated. Most treatment-emergent adverse events, which included urinary tract infection, flank pain, ureteral narrowing and hydronephrosis and time-limited creatinine elevation, were defined as mild or moderate and transient.
“We are excited about the interim analysis from our pivotal Phase 3 trial of UGN-101 presented today. This is an important step toward our anticipated Q1 2019 submission of a New Drug Application to the US Food and Drug Administration,” added Ron Bentsur, Chief Executive Officer of UroGen. “This interim analysis also demonstrates the potential of our proprietary RTGel sustained release platform, which we believe has the potential to transform existing treatment paradigms across a number of urologic conditions. We look forward to continuing to advance our pipeline towards this end.”

The OLYMPUS trial is continuing to enroll patients, in the second half of 2018 expects top-line results.
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