UGN-103 under development for urothelial cancers

UroGen Pharma has entered into a licensing agreement with medac GmbH to develop UGN-103, a next-generation novel mitomycin-based formulation for the treatment of patients with urothelial cancers, according to a news release from UroGen Pharma.¹

UroGen plans to submit a New Drug Application in 2024 and anticipates an FDA decision in 2025 for UGN-102.

UGN-103 combines UroGen’s RTGel technology with medac GmbH’s mitomycin. The formulation, if approved, is expected to provide advantages in regard to production, cost, supply, and product convenience. A phase 3 study is expected to initiate in 2024 to explore the safety and efficacy of UGN-103 for the treatment of patients with low-grade intermediate-risk non–muscle-invasive bladder cancer (LG-IR-NMIBC).

"The strategic alliance with medac fortifies our commitment to continuously innovate novel, non-surgical treatments for patients with urothelial cancers, including LG-IR-NMIBC,” said Liz Barrett, president and CEO of UroGen, in the news release.¹ "The 80 mg formulation of mitomycin from medac is specifically designed to be mixed with our RTGel technology, which may provide advantages for patients looking for non-surgical treatments for urothelial cancer. With medac's intellectual property protection for this next-generation mitomycin formulation lasting until June 2035 and our pending U.S. patent applications, we foresee potential intellectual property protection until December 2041."

Development of UGN-103 is anticipated to initiate following the approval and launch of UGN-102, according to UroGen.

Data on UGN-102

UGN-102 (mitomycin) for intravesical solution is a hydrogel-based formulation being investigated for use in patients with LG-IR-NMIBC. The treatment is currently being assessed in the phase 3 ENVISION trial (NCT05243550).

According to findings presented at the 2023 Society of Urologic Oncology Annual Meeting, interim data from the trial showed a complete response rate of 79.2% (95% CI, 73.5-84.1) with UGN-102 at 3-month follow-up.² In the remaining patients, 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.

Overall, the study included 240 patients with LG-IR-NMIBC. Patients received primary chemoablative therapy with UGN-102 as 6 once-weekly intravesical instillations administered using a traditional urethral catheter. Nearly all patients (95%) received the full 6 instillations of UGN-102.

The study’s primary end point was the complete response rate at 3 months following the initial treatment instillation. Secondary outcome measures include the duration and durability of response, as well as disease-free survival.³

UroGen plans to submit a New Drug Application in 2024 and anticipates an FDA decision in 2025 for UGN-102 pending positive results on the durability of response end point from the ENVISION trial.

References

1. UroGen secures exclusive license from medac GmbH to develop a next-generation novel mitomycin-based formulation for urothelial cancers. News release. UroGen Pharma. Published online and accessed January 17, 2024. https://www.biospace.com/article/releases/urogen-secures-exclusive-license-from-medac-gmbh-to-develop-a-next-generation-novel-mitomycin-based-formulation-for-urothelial-cancers/?s=86

2. 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

3. A phase 3 single-arm study of UGN-102 for treatment of low grade intermediate risk non-muscle-invasive bladder cancer (ENVISION). ClinicalTrials.gov. Last updated September 29, 2023. Accessed January 17, 2024. https://clinicaltrials.gov/study/NCT05243550