UroGen Pharma Ltd. (NASDAQ:URGN) on Sunday highlighted a duration of response of nearly four years from a long-term follow-up study with Jelmyto (mitomycin) for pyelocalyceal solution.
The U.S. Food And Drug Administration approved Jelmyto in 2020 for low-grade upper tract urothelial cancer (LG-UTUC) in adults.
Among patients from the OLYMPUS trial who achieved a complete response after primary chemoablation with Jelmyto (n = 41), the median duration of response was 47.8 months, regardless of whether their cancer was new onset or recurrent (median follow-up, 28.1 months).
Of these patients, 21 had new-onset UTUC, and 20 had recurrent UTUC at baseline. There were no significant differences in durability between the groups, with eight patients in each group experiencing recurrence or death not due to treatment.
20 patients entered long-term follow-up.
To further explore Jelmyto’s potential in treating patients with LG-UTUC, investigators are now enrolling participants in the Jelmyto uTRACT Registry to gather longitudinal real-world usage data. As of April 2025, 22 sites have been activated and 251 patients have been enrolled.
On Sunday, UroGen Pharma also announced new data from the OPTIMA II Phase 2b study of UGN‑102 (mitomycin) for an intravesical solution.
The data demonstrated a clinically meaningful two-year duration of response (24.2 months).
UGN-102 is UroGen’s sustained-release formulation of mitomycin, being developed for recurrent low-grade, intermediate-risk, non-muscle-invasive bladder cancer (LG-IR-NMIBC).
Among the 41 patients achieving a complete response (CR) at three months, 25 remained in CR at 12 months, and 17 of these patients entered long-term follow-up.
The median duration of response for the 41 patients who achieved CR was 24.2 months, with a median follow-up time of 33.6 months. Twenty patients (48.8%) experienced recurrence of low-grade disease.
One patient progressed to a high-grade disease, and one patient died due to a cardiac disorder. Five patients remained disease-free at the time of the four-year data analysis.
UroGen completed the submission of the UGN-102 rolling new drug application (NDA) to the FDA for UGN-102 in August 2024. The FDA accepted the NDA for UGN-102 with a Prescription Drug User Free Act (PDUFA) goal date of June 13, 2025.
UroGen Pharma also shared an updated 18-month DOR of 80.6% from the Phase 3 ENVISION trial of UGN-102 (mitomycin) for the intravesical solution, an investigational treatment for recurrent LG-IR-NMIBC.
On Friday, UroGen Pharma shared safety data from the Phase 1 dose-escalation study for UGN-301 (zalifrelimab) intravesical solution for recurrent non-muscle invasive bladder cancer.
The study included up to 30 patients per arm and aimed to assess safety and determine the recommended Phase 2 dose of UGN-301 as monotherapy and in combination with other agents.
In the monotherapy arm, dose escalation continued to the maximum feasible dose. No dose-limiting toxicities or treatment-emergent adverse events leading to treatment discontinuation were observed.
Among evaluable patients who received UGN-301, 46% of those with Ta/T1 disease and 33% with carcinoma in situ (CIS) ± Ta/T1 disease were recurrence-free or had achieved a complete response at week 12.
At the 15-month disease assessment, 60% of patients with Ta/T1 disease treated with 300 mg remained recurrence-free, including one with high-grade T1 disease.
In the 500 mg cohort, 25% of patients with CIS disease and 33% of patients with Ta/T1 disease remained disease-free at six months; both are still active participants in the study.
D.Boral Capital analyst Jason Kolbert writes, “These data position UGN-301 as a promising candidate for a population in urgent need of more localized, immunomodulatory therapies…”
Price Action: URGN stock is up 7.65% at $10.63 at the last check Monday.
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