Benzinga | Nov 17, 2020 08:06AM EST

UroGen Pharma Reports Final Topline Results From OPTIMA II Phase 2b Trial Evaluating Efficacy, Safety Of UGN-102 For Intravesical Solution In Patients With Low-Grade Intermediate Risk Non-Muscle Invasive Bladder Cancer

UroGen Pharma Ltd. (NASDAQ:URGN) today announced final topline results from the single-arm, open-label OPTIMA II Phase 2b trial evaluating the efficacy and safety of investigational UGN-102 (mitomycin) for intravesical solution in patients with low-grade intermediate risk non-muscle invasive bladder cancer (LG IR-NMIBC). As previously reported, 65% (41/63) of patients receiving UGN-102 achieved a complete response (CR) three months after the start of therapy. In this subset of patients, duration of response at nine months (12-months from start of therapy) was estimated by Kaplan-Meier analysis to be 72.5%. Median duration of response was not reached. The Company expects to initiate a Phase 3 study evaluating UGN-102 versus current standard of care by the end of the year.

Treatment with UGN-102 was generally well tolerated and the safety profile was consistent with previously reported results. In the OPTIMA II trial, the majority of the most common adverse events (? 10%) were reported as mild to moderate in severity and include dysuria, urinary frequency, hematuria, urinary urgency, urinary tract infection and fatigue. No treatment related serious adverse events were reported.

"The current approach to treating patients diagnosed with low-grade intermediate risk non-muscle invasive bladder cancer is surgery. In most cases, the cancer comes back and repetitive surgical intervention is required. This puts a tremendous burden on patients and their families and can even be life-threatening," said Andrea Maddox-Smith, Chief Executive Officer, Bladder Cancer Advocacy Network. "Being able to provide patients with an alternative, non-surgical treatment option that is effective, well-tolerated and durable, would greatly benefit those in the patient community."

Patients with LG IR-NMIBC are chronically relapsing and currently, their only treatment option is repeated transurethral resection of bladder tumors (TURBT). Some patients require multiple TURBT surgeries per year, which can lead to increased morbidity and risks associated with repetitive anesthesia. It is estimated that 80,000 people in the U.S. are treated annually for LG IR-NMIBC. This includes newly diagnosed patients and patients who have a recurrence after surgery.

"We are extremely encouraged by the OPTIMA II data and believe UGN-102 has the potential to provide a safe, durable, outpatient treatment alternative for low-grade intermediate risk non-muscle invasive bladder cancer," said Dr. Mark Schoenberg, Chief Medical Officer at UroGen. "We look forward to the expected initiation of our Phase 3 trial this year and further exploring the potential of our innovative technology in advancing new treatments for specialty cancers and urologic diseases."

The Company anticipates submitting the data for presentation at an upcoming medical meeting as well as potential publication in a peer-reviewed journal.