UroGen Submits UGN-102 NDA for FDA Approval to Treat Low-Grade Intermediate-Risk Bladder Cancer

PRINCETON, NJ, USA I August 14, 2024 I UroGen Pharma Ltd. (Nasdaq: URGN), a prominent biotechnology company focusing on novel therapies for urothelial and specialty cancers, has successfully completed its New Drug Application (NDA) submission for the investigational drug UGN-102 (mitomycin) for intravesical solution. This marks a critical step toward providing innovative treatments for low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC). If the NDA is accepted and given priority review by the FDA, UroGen anticipates potential approval in early 2025.

Liz Barrett, President and CEO of UroGen, expressed that the NDA submission for UGN-102 is a pivotal milestone for the company. She highlighted that the approval of UGN-102 could offer patients a significant alternative to repeated surgeries, potentially enhancing their quality of life and providing clinically meaningful recurrence-free intervals. LG-IR-NMIBC has high recurrence rates, making the need for innovative therapies like UGN-102 urgent. Barrett emphasized that UGN-102 could become an essential option for managing this challenging disease.

The NDA is backed by the clinical program for UGN-102, which includes long-term durability results from the Phase 3 ENVISION study. The ENVISION trial achieved its primary endpoint, showing a 79.6% complete response (CR) rate in patients three months after the first instillation of UGN-102. Furthermore, UGN-102 demonstrated an 82.3% 12-month duration of response (DOR) by Kaplan-Meier estimate in patients who achieved a CR at three months. DOR estimates at 15 and 18 months after the initial three-month CR were both 80.9%.

The most common treatment-emergent adverse events (TEAEs) reported in the ENVISION trial included dysuria, hematuria, urinary tract infections, pollakiuria, fatigue, and urinary retention. These TEAEs were generally mild to moderate in severity and were resolving or had resolved. The safety profile observed in the ENVISION trial was consistent with that seen in other studies of UGN-102.

UGN-102 (mitomycin) for intravesical solution is a novel drug formulation of mitomycin, currently in Phase 3 development for treating LG-IR-NMIBC. Utilizing UroGen’s proprietary RTGel® technology, a sustained-release, hydrogel-based formulation, UGN-102 is designed to enable prolonged exposure of bladder tissue to mitomycin, thereby treating tumors non-surgically. UGN-102 is administered to patients via a standard urinary catheter in an outpatient setting by trained healthcare professionals.

In the United States, bladder cancer is the second most common urologic cancer among men. LG-IR-NMIBC represents about 22,000 new bladder cancer diagnoses each year, with an estimated 60,000 recurrences annually among previously diagnosed patients. Bladder cancer predominantly affects older populations, with a median age of diagnosis at 73 years and an increased risk of comorbidities. Current guideline recommendations for managing NMIBC include TURBT as the standard of care, but up to 70 percent of NMIBC patients experience at least one recurrence, with LG-IR-NMIBC patients being even more likely to recur and require repeated TURBT procedures.

The Phase 3 ENVISION trial is a multinational, multicenter study evaluating the efficacy and safety of UGN-102 (mitomycin) for intravesical solution as chemoablative therapy in patients with LG-IR-NMIBC. The trial completed target enrollment with approximately 240 patients across 56 sites. Participants received six once-weekly intravesical instillations of UGN-102. The primary endpoint was the CR rate at the three-month assessment following the first instillation, and the key secondary endpoint evaluated durability over time in patients who achieved a CR at three months.

UroGen Pharma Ltd. is dedicated to developing and commercializing innovative solutions for treating urothelial and specialty cancers. They have developed RTGel® reverse-thermal hydrogel, a proprietary sustained-release hydrogel technology that has the potential to enhance the therapeutic profiles of existing drugs. UroGen aims to improve local therapy for urinary tract treatments, making it a potentially more effective option. The company is headquartered in Princeton, NJ, with operations in Israel.