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FDA accepts IND application for UGN-103 in NMIBC


The IND will initiate the launch of a phase 3 study to explore the safety and efficacy of UGN-103 in patients with low-grade, intermediate-risk NMIBC.

The FDA has accepted an investigational new drug (IND) application for UGN-103, a mitomycin-based formulation for patients with low-grade intermediate-risk non–muscle-invasive bladder cancer (LG-IR-NMIBC), according to a news release from UroGen Pharma, the developer of the therapy.1

UGN-103 is planned to follow the anticipated FDA approval and launch of UGN-102, according to the company.

UGN-103 is planned to follow the anticipated FDA approval and launch of UGN-102, according to the company.

The IND will initiate the launch of a phase 3 study to explore the safety and efficacy of UGN-103 in patients with LG-IR-NMIBC. UroGen plans to begin the study in 2024.

“We are delighted by the FDA's acceptance of our IND for UGN-103, marking a significant step forward in our mission. We eagerly anticipate commencing a clinical trial with UGN-103 this year, as we strive to continually advance and develop treatments for patients with high unmet need,” said Liz Barrett, president and CEO of UroGen, in the news release.1

UGN-103 for intravesical solution utilizes UroGen’s RTgel technology to enable sustained release of mitomycin for longer exposure of the therapy to the bladder tissue. The treatment builds on the company’s UGN-102 therapy, which is currently in phase 3 development for the treatment of patients with LG-IR-NMIBC.

According to the news release, the anticipated advantages of UGN-103 include the new 80-mg mitomycin dosage strength, which may shorten the manufacturing process, simplify the reconstitution procedure, and potentially extend intellectual property protection until as late as December 2041.

UGN-103 is planned to follow the anticipated FDA approval and launch of UGN-102, according to the company. UroGen initiated the start of a rolling new drug application (NDA) for UGN-102 in January 2024.2 The company plans to complete the rolling NDA in September 2024 assuming positive findings from the durability of response end point in the phase 3 ENVISION study. This could result in an FDA decision potentially as early as the first quarter of 2025.

Data from the ENVISION trial

In July 2023, UroGen reported that the phase 3 ENVISION trial (NCT05243550) met its primary end point by demonstrating a complete response rate of 79.2% among patients with LG-IR-NMIBC at 3 months following initial treatment with UGN-102.3

Data were also presented at the 2023 Society of Urologic Oncology Annual Meeting,4 showing that in the remaining patients without a complete response, 14.6% had residual disease, 2.5% progressed to high-grade disease, 1.7% had an indeterminate response, and 2.1% did not have data.

Regarding safety, nearly all treatment-emergent adverse events (TEAEs) were mild or moderate. TEAEs occurring in at least 5% of patients included dysuria (17.9% mild; 3.8% moderate), hematuria (6.3% mild; 2.1% moderate), pollakiuria (5.4% mild; 0.8% moderate), urinary tract infection (1.7% mild; 4.2% moderate), and fatigue (3.8% mild; 1.7% moderate). One patient had severe dysuria.

In total, the single-arm, multinational study enrolled approximately 240 patients across 56 clinical trial sites to assess the safety and efficacy of UGN-102 for intravesical solution as primary chemoablative therapy in patients with LG-IR-NMIBC. Patients included in the study received 6 once-weekly intravesical instillations of UGN-102 administered using a traditional urethral catheter. Nearly all patients (95%) received the full 6 instillations of UGN-102.

The primary end point for the study is the complete response rate at 3 months following initial therapy, and the key secondary end point is the duration of complete response, as assessed up to 63 months. Other secondary end points include disease-free survival and the incidence of TEAEs.

The trial remains ongoing to assess the study’s key secondary end point of duration of response.


1. UroGen announces FDA acceptance of Investigational New Drug Application for UGN-103, a next generation mitomycin-based formulation for low-grade intermediate-risk non-muscle invasive bladder cancer. News release. UroGen Pharma Ltd. Published online and accessed April 15, 2024. https://investors.urogen.com/news-releases/news-release-details/urogen-announces-fda-acceptance-investigational-new-drug

2. UroGen initiates submission of a rolling NDA to the FDA for UGN-102. News release. January 24, 2024. Accessed April 15, 2024. https://investors.urogen.com/news-releases/news-release-details/urogen-initiates-submission-rolling-nda-fda-ugn-102

3. UGN-102, in development as the potential first non-surgical therapy for LG-IR-NMIBC, met primary endpoints in both phase 3 ATLAS and ENVISION clinical trials. News release. July 27, 2023. Accessed April 15, 2024. https://investors.urogen.com/news-releases/news-release-details/ugn-102-development-potential-first-non-surgical-therapy-lg-ir

4. Prasad S, Mladenov B, Shishkov D, et al. Primary chemoablation for recurrent low grade intermediate risk (LG IR) NMIBC: the ENVISION trial. Presented at: 2023 Society of Urologic Oncology Annual Meeting. November 28 – December 1, 2023; Washington, DC. LBA 3389

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