UroGen Pharma presented positive data from an 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).
These results were presented during an oral session at the 113th American Urological Association’s (AUA) Annual Meeting in San Francisco bySeth Paul Lerner, MD, FACS. The interim analysis from this international, multi-center trial showed a complete response (CR) rate of 59% in 34 patients who were evaluated for primary disease evaluation (PDE, or the primary endpoint). PDE is conducted four to six weeks after completion of UGN-101 treatment, which was administered once weekly for six weeks. Results showed that 20 of the interim analysis intent to treat population of 34 patients (59%) achieved a CR, defined as a negative ureteroscopic evaluation and a negative wash cytology. In addition, five of 34 patients (15%) achieved a partial response. Approximately 39% of tumors treated were categorized as unresectable by surgery at baseline. The CRs to date have been durable. Of the 20 patients who achieved a CR, 13 patients have reached three-month follow-up and all remain in CR. Four of these 13 patients have reached six-month follow-up and one of the 13 patients has reached nine-month follow-up. All remain in CR.
UGN-101 appears to be well-tolerated with most treatment-emergent adverse events characterized as mild or moderate and transient. These included urinary tract infection, flank pain, ureteral narrowing and hydronephrosis and time-limited creatinine elevation.
“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,” said Mark Schoenberg, M.D., Chief Medical Officer of UroGen. “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.”
The OLYMPUS trial continues to enroll patients, and top-line results are expected in the second half of 2018.