UroGen Pharma is one step closer to potentially revolutionizing the treatment landscape for low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC) with its investigational drug, UGN-102 (mitomycin) for intravesical solution. The U.S. Food and Drug Administration (FDA) has accepted the New Drug Application (NDA) for UGN-102, setting a Prescription Drug User Fee Act (PDUFA) goal date of June 13, 2025. If approved, UGN-102 would be the first FDA-approved medicine for this indication.
ENVISION Trial Data
The NDA is supported by data from the Phase 3 ENVISION trial, a single-arm, multinational, multicenter study evaluating the efficacy and safety of UGN-102 as primary chemoablative therapy in patients with LG-IR-NMIBC. The trial met its primary endpoint, demonstrating a 79.6% complete response (CR) rate at three months after the first instillation of UGN-102. Key secondary endpoint data revealed an 82.3% 12-month duration of response (DOR) by Kaplan-Meier estimate in patients who achieved a CR at three months. Kaplan-Meier estimates for DOR at 15 months and 18 months following the three-month CR were both 80.9%.
Dr. Mark Schoenberg, Chief Medical Officer of UroGen, emphasized the significance of these findings: "The NDA for UGN-102 is backed by a robust data set demonstrating impressive durability of response across three clinical trials and a favorable safety profile... We believe that, if approved, UGN-102’s ability to achieve durable complete responses and potentially reduce recurrence rates while extending treatment-free intervals will represent a significant advance in managing LG-IR-NMIBC."
The most common treatment-emergent adverse events in the ENVISION trial were dysuria, hematuria, urinary tract infection, pollakiuria, fatigue, and urinary retention. These adverse events were generally mild to moderate in severity and either resolved or were resolving.
UGN-102: A Novel Approach
UGN-102 is an innovative formulation of mitomycin utilizing UroGen’s proprietary RTGel technology. This reverse-thermal hydrogel-based platform allows for sustained release, enabling longer exposure of bladder tissue to mitomycin. The drug is administered via standard urinary catheter in an outpatient setting by trained healthcare professionals, offering a non-surgical approach to tumor ablation.
Market Opportunity and Future Prospects
The U.S. market for LG-IR-NMIBC that UGN-102 can address, if approved, is valued at approximately $5 billion. Approximately 22,000 newly diagnosed bladder cancer patients each year and an estimated 60,000 recurrences annually among patients diagnosed from previous years. With high recurrence rates following standard of care transurethral resection of bladder tumor (TURBT), UGN-102 presents a potential solution to reduce the need for repeated surgical procedures.
UroGen is also advancing its pipeline with UGN-103 and UGN-104, next-generation mitomycin-based formulations. The company has received a new U.S. patent allowance for these products and has commenced the Phase 3 UTOPIA trial for UGN-103, targeting recurrent LG-IR-NMIBC patients.
Financial Position
As of September 30, 2024, UroGen Pharma reported cash and cash equivalents of $254.2 million, demonstrating a strong financial position to support pre-commercial and launch activities for UGN-102, pending FDA approval.
