UroGen Pharma Ltd. has initiated a Phase 3 clinical trial for UGN-103 (mitomycin), an investigational intravesical solution designed to treat low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC). The first patient has been dosed in the UTOPIA study, a single-arm, multicenter trial evaluating the efficacy and safety of UGN-103.
Liz Barrett, President and Chief Executive Officer of UroGen, stated that UGN-103 represents a significant advancement, potentially improving manufacturing, convenience, and cost. The trial aims to enroll 87 patients with LG-IR-NMIBC.
Trial Design and Endpoints
The UTOPIA study involves weekly intravesical instillations of 75 mg of UGN-103 for six weeks. The primary efficacy endpoint is the complete response rate at the three-month visit, defined as the absence of detectable disease in the bladder. Patients achieving a complete response will enter a follow-up period with evaluations every three months to monitor for disease recurrence or progression. The trial will continue until disease recurrence, progression, death, or the last patient completes 12 months of follow-up.
UGN-103: Next-Generation Mitomycin
UGN-103 is a novel formulation of mitomycin that leverages UroGen's proprietary RTGel technology. This reverse-thermal hydrogel is designed for sustained release, allowing for extended exposure of bladder tissue to mitomycin. UroGen received a Notice of Allowance from the USPTO for UGN-103, with a potential patent expiration in December 2041.
Current Landscape of NMIBC Treatment
In the U.S., bladder cancer is the second most common urologic cancer in men. LG-IR-NMIBC affects approximately 22,000 newly diagnosed patients each year, with an estimated 60,000 recurrences annually. Current guidelines recommend transurethral resection of bladder tumor (TURBT) as the standard of care, but recurrence rates are high, with up to 70% of NMIBC patients experiencing at least one recurrence.
UroGen's Pipeline: UGN-102
UroGen is also developing UGN-102 (mitomycin) for intravesical solution, another innovative formulation for LG-IR-NMIBC. The New Drug Application (NDA) for UGN-102 was submitted to the FDA in August 2024, with a potential FDA decision expected in early 2025 if priority review is granted. Phase 3 data for UGN-102 demonstrated a 79.6% complete response rate at 3 months, with a 12-month duration of response of 82.3% among responders.
