On May 5, 2019 UroGen Pharma Ltd. (Nasdaq: URGN), a clinical-stage biopharmaceutical company developing treatments to address unmet needs in the field of urology, reported findings from a secondary analysis from the pivotal Phase 3 OLYMPUS trial which showed that UGN-101 (mitomycin gel) for instillation, an investigational mitomycin formulation, demonstrated a 59 percent complete response rate in a subset of patients with endoscopically unresectable low-grade upper tract urothelial cancer (UTUC) (Press release, UroGen Pharma, MAY 5, 2019, View Source [SID1234535721]). Findings were presented by Seth Paul Lerner, M.D., FACS, Professor of Urology at Baylor College of Medicine, in an oral presentation during the plenary session at the 114th American Urological Association (AUA) Annual Meeting in Chicago.

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The analysis showed that in the OLYMPUS intent-to-treat population, 71 patients had undergone PDE at the time of the analysis and 42 of the 71 patients (59 percent) achieved a CR. Forty-one patients entered follow-up. Of the evaluated complete responses to date, 27 patients have undergone a six-month evaluation, and 24 out of 27 patients (89 percent) have remained disease free at six months. Overall, 5 of 41 patients who achieved a CR have relapsed at any time during the study.

Of these 71 patients, 34 were initially characterized by the treating physician as having endoscopically unresectable tumor at baseline, and 20 of 34 of these patients (59 percent) achieved a CR at the PDE.

*Forty-one patients entered follow-up. At the time of the analysis, 66 percent (27/41) of patients have completed a six-month evaluation.

The most common adverse events observed were urinary tract infection, ureteral narrowing and stricture formation. The majority of ureteral events were reported as mild to moderate and have resolved.

"The results from the OLYMPUS trial continue to be compelling for new and recurrent LG UTUC, as well as for those who have unresectable tumors and would be immediate candidates for kidney removal. For this typically elderly patient population, kidney preservation is paramount, and these findings provide evidence-based support for the concept of chemoablation with UGN-101 as an initial kidney-sparing treatment option for low-grade UTUC," said Mark P. Schoenberg, MD, Chief Medical Officer at UroGen. "The analysis also advances our understanding of durability of response, which we are pleased to see has remained consistent as the number of patients who reach the six-month follow-up timepoint increases."

The Company initiated its rolling submission of the UGN-101 New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) in December 2018. The FDA previously granted Orphan Drug, Fast Track, and Breakthrough Therapy Designations to UGN-101 for the treatment of UTUC. If approved, UGN-101 would be the first drug approved for the non-surgical treatment of LG UTUC.

About The Phase 3 OLYMPUS Trial

OLYMPUS (Optimized DeLivery of Mitomycin for Primary UTUC Study) is a pivotal, open-label, single-arm Phase 3 clinical trial of UGN-101 (mitomycin gel) for instillation to evaluate the safety, tolerability and tumor ablative effect of UGN-101 in patients with low-grade UTUC. The trial enrolled 71 patients at clinical sites across the United States and Israel. Study participants were treated with six weekly instillations of UGN-101 administered via a standard catheter. Four to six weeks following the last instillation, patients underwent a primary disease evaluation (PDE) to determine response, the primary endpoint of the study. PDE involved a ureteroscopy and wash cytology, a standard microscopic test of cells obtained from the urine to detect cancer. Patients who achieved a complete response at the PDE timepoint were then followed for up to 12 months to determine the durability of disease control with UGN-101.

About UGN-101

UGN-101 (mitomycin gel) for instillation is an investigational drug formulation of mitomycin in Phase 3 development for the treatment of low-grade upper tract urothelial cancer (LG UTUC). Utilizing the RTGel technology platform, UroGen’s proprietary sustained release, hydrogel-based formulation, UGN-101 is designed to enable longer exposure of urinary tract tissue to mitomycin, thereby enabling the treatment of tumors by non-surgical means. UGN-101 is delivered to patients using standard ureteral catheters. The Company initiated its rolling submission of the UGN-101 New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) in December 2018. The FDA previously granted Orphan Drug, Fast Track, and Breakthrough Therapy Designations to UGN-101 for the treatment of UTUC. If approved, UGN-101 would be the first drug approved for the non-surgical treatment of LG UTUC.