Estimated DOR of 2 years is seen with UGN-102 in LG-IR-NMIBC

Treatment of low-grade, intermediate-risk non–muscle invasive bladder cancer (NMIBC) with reverse thermal gel containing mitomycin (UGN-102) was associated with an estimated duration of response (DOR) of approximately 2 years, investigators reported at the 2025 American Urological Association (AUA) Annual Meeting in Las Vegas, Nevada.¹

Results from the phase 2b OPTIMA II study (NCT03558503) were previously reported in the Journal of Urology. In that study, 41 (65%) patients treated with UGN-102 achieved a complete response (CR) at 3 months, “of whom 39 (95%), 30 (73%) and 25 (61%) remained disease-free at 6, 9 and 12 months after treatment initiation, respectively,” the authors wrote.²

Neal D. Shore, MD, FACS

In the study presented at AUA 2025, investigators led by Neal D. Shore, MD, FACS, director of the Carolina Urologic Research Center in Myrtle Beach, South Carolina, reported on 4-year follow-up data. A total of 63 patients were enrolled in the study. Median age was 68 years (range, 33-96 years). Thirty-eight (60.3%) patients were male, and 25 (39.7%) were female. Fifty-five (87.3%) patients were Caucasian. A total of 49 (77.8%) patients had recurrent low-grade NMIBC. Mean number of prior transurethral resection of the bladder tumor (TURBT) procedures was 4.0 (SD: 3.3), and median number of prior TURBTs was 3.0 (range, 0-13).

Patients received 6 once-weekly instillations; a total of 57 patients received all doses. For the long-term follow-up study, 17 patients who remained in CR at 12 months were followed with standard of care management for 4 years until disease recurrence, progression, or death.

The investigators reported that the median Kaplan-Meier estimate of DOR was 24.2 months (95% CI: 9.72-47.18). Median follow-up was 33.6 months (95% CI: 10.78-42.94). A total of 20 (48.8%) patients had recurrence of low-grade disease, 1 patient progressed to high-grade disease, and 1 patient died as a result of a cardiac disorder.

“Five patients remained disease-free at the time of the 4-year data,” the investigators wrote in their poster.

“These results demonstrate that treatment with UGN-102 results in a clinically meaningful duration of response in patients with newly diagnosed or recurrent [low-grade intermediate-risk] NMIBC. UGN-102 may represent a durable and safe non-surgical alternative to TURBT, the current standard of care, for treating these patients,” the investigators concluded.

A new drug application (NDA) for UGN-102 was submitted to the FDA in August 2024, seeking approval of the therapy for patients with low-grade, intermediate-risk NMIBC. The FDA accepted the NDA in October 2024, issuing a target action date of June 13, 2025.³ According to UroGen Pharma, the developer of the therapy, UGN-102 has the potential to become the first chemoablative, non-surgical treatment option for patients with LG-IR-NMIBC if approved.

"As the burden of LG-IR-NMIBC persists, the need for long-lasting treatment options becomes increasingly urgent," said Mark Schoenberg, Chief Medical Officer, UroGen, in a news release.⁴ "The growing body of evidence supporting UGN-102 underscores its potential to address this unmet need. These results emphasize UGN-102’s potential to deliver meaningful and sustained responses, offering hope to patients who have long struggled with recurrence and limited treatment options."

REFERENCES

1. Shore ND, Chevli KK, Trainer A, et al. Treatment of low-grade intermediate-risk non-muscle invasive bladder cancer with UGN-102: long-term outcomes of the OPTIMA II study. J Urol. 2025;213(5S):e497. doi:10.1097/01.JU.0001109876.32161.cb.05

2. Chevli KK, Shore ND, Trainer A, et al. Primary chemoablation of low-grade intermediate-risk nonmuscle-invasive bladder cancer using UGN-102, a mitomycin-containing reverse thermal gel (Optima II): A phase 2b, open-label, single-arm trial. J Urol. 2022;207(1):61-69. doi:10.1097/JU.0000000000002186

3. UroGen announces FDA acceptance of its new drug application for UGN-102. News release. UroGen Pharma Ltd. October 15, 2024. Accessed October 28, 2024. https://investors.urogen.com/news-releases/news-release-details/urogen-announces-fda-acceptance-its-new-drug-application-ugn-102

4. New long-term follow-up data from OPTIMA II study of UGN-102 demonstrates median duration of response of two years in patients with LG-IR-NMIBC. News release. UroGen Pharma. April 27, 2025. Accessed April 27, 2025. https://investors.urogen.com/news-releases/news-release-details/new-long-term-follow-data-optima-ii-study-ugn-102-demonstrates