On August 14, 2024 UroGen Pharma Ltd. (Nasdaq: URGN), a leading biotech company specializing in novel therapies for urothelial and specialty cancers, reported the successful completion of its New Drug Application (NDA) submission for investigational drug UGN-102, (mitomycin) for intravesical solution, a significant step forward in potentially addressing the urgent need for innovative treatments for low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC) (Press release, UroGen Pharma, AUG 14, 2024, View Source [SID1234645930]). UroGen anticipates potential FDA approval in early 2025, if the NDA is accepted for filing by the FDA and priority review is granted.

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"The completion of the NDA submission for UGN-102 marks a crucial milestone for UroGen and underscores our dedication to advancing this groundbreaking treatment for patients with LG-IR-NMIBC," said Liz Barrett, President and CEO of UroGen. "By providing a viable alternative to repeated surgeries, if approved UGN-102 may offer patients quality of life benefits and clinically meaningful recurrence-free intervals. The high recurrence rates associated with LG-IR-NMIBC make the need for innovative therapies like UGN-102 urgent. UGN-102 could become a valuable new option for managing this challenging disease."

The NDA is supported by the clinical program for UGN-102, including the long-term durability results from the Phase 3 ENVISION study. The ENVISION trial met its primary endpoint by demonstrating that patients treated with UGN-102 had a 79.6% complete response (CR) rate at three months following the first instillation. UGN-102 demonstrated an 82.3% 12-month duration of response (DOR) by Kaplan-Meier estimate (n=108) in patients who achieved a CR at three months. The DOR estimates at 15 (n=43) and 18 (n=9) months after 3-month CR were both 80.9%.

The most common treatment-emergent adverse events (TEAEs) in the ENVISION trial were dysuria, hematuria, urinary tract infection, pollakiuria, fatigue, and urinary retention. TEAEs were typically mild-to-moderate in severity and resolved or resolving. The ENVISION trial demonstrated a similar safety profile to that observed in other studies of UGN-102.

For more information and perspective on the ENVISION study results and the unmet need for patients with LG-IR-NMIBC click here to view the webcast: UGN-102 12-Month Durability of Response Data Event.

About UGN-102

UGN-102 (mitomycin) for intravesical solution is an innovative drug formulation of mitomycin, currently in Phase 3 development for the treatment of LG-IR-NMIBC. Utilizing UroGen’s proprietary RTGel technology, a sustained release, hydrogel-based formulation, UGN-102 is designed to enable longer exposure of bladder tissue to mitomycin, thereby enabling the treatment of tumors by non-surgical means. UGN-102 is delivered to patients using a standard urinary catheter in an outpatient setting by a trained healthcare professional.

About Non-Muscle Invasive Bladder Cancer (NMIBC)

In the U.S. bladder cancer is the second most common urologic cancer in men. LG-IR-NMIBC represents approximately 22,000 newly diagnosed bladder cancer patients each year and an estimated 60,000 recurrences annually among patients diagnosed from previous years. Bladder cancer primarily affects older populations with the median age of diagnosis 73 years and an increased risk of comorbidities. Guideline recommendations for the management of NMIBC include TURBT as the standard of care. Up to 70 percent of NMIBC patients experience at least one recurrence and LG-IR-NMIBC patients are even more likely to recur and face repeated TURBT procedures.

About ENVISION

The Phase 3 ENVISION trial is a single-arm, 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 Phase 3 ENVISION trial completed target enrollment with approximately 240 patients across 56 sites. Study participants received six once-weekly intravesical instillations of UGN-102. The primary endpoint evaluated the CR rate at the three-month assessment after the first instillation, and the key secondary endpoint evaluated durability over time in patients who achieved a CR at the three-month assessment. Learn more about the Phase 3 ENVISION trial at www.clinicaltrials.gov (NCT05243550).