Long-Term Efficacy and Safety of URO-902 (PVAX/HSLO) in Women With Overactive Bladder and Urge Urinary Incontinence: Final Results of a Phase 2A Trial

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Conference Proceeding

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Journal of Urology


INTRODUCTION AND OBJECTIVE: URO-902 is an investigational gene therapy for overactive bladder (OAB) consisting of a plasmid vector expressing the large-conductance Ca2þ-activatedKþchannelasubunit. URO-902 showed improved efficacy and quality of life (QoL) vs placebo in a 12-week interim analysis of a phase 2atrial. Here we report final safety and efficacy results from the trial. METHODS: This 48-week multicenter, double-blind, placebo-controlled, dose-escalation trial (NCT04211831) randomized women40e79 years old with OAB, urge urinary incontinence (UUI), and inadequate pharmacologic OAB management to single-dose URO-902 24 or 48 mg or placebo administered by intradetrusor injection via cystoscopy under local anesthesia. Patients could request additional OAB treatments starting at week 24, after which they were not included in efficacy analyses. Exploratory efficacy endpoints were change from baseline (CFB) in mean daily micturitions, urgency episodes, and UUI episodes. Safety was assessed by adverse events(AEs) and postvoid residual (PVR) urine volume. RESULTS: Of 80 patients randomized, 74 were treated (intent-to-treat exposed [ITT-E] population), and 67 completed the trial. In the ITT-E population, mean age was 64.7 years; 13.5% had prior onabotulinumtoxinA treatment. URO-902 24 and 48 mg were associated with reductions from baseline starting at week 2 in daily micturitions, urgency episodes, and UUI episodes; reductions were durable through week 48. Overall, 45.5%, 53.8%, and 53.8% of patients receiving URO-902 24 mg, URO-902 48 mg, and placebo, respectively, experienced ≥1 treatment-emergent AE; the most commonly occurring (24 mg/48 mg/placebo) were urinary tract infection (0%/15.4%/3.8%) and hematuria (4.5%/7.7%/7.7%). One patient receiving URO-902 48 mg had asymptomatic elevated PVR urine volume, which resolved spontaneously without catheterization. CONCLUSIONS: URO-902 24 or 48 mg showed durable, meaningful improvements from baseline in micturitions, urgency episodes (dose dependent), and UUI episodes, consistent with previously reported QoL improvements at 12 weeks, and was safe and well tolerated in this phase 2a trial. Interpretation of efficacy vs placebo was limited by use of additional OAB treatments after week 24





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American Urological Association Annual Meeting, April 28 - May 1, 2023, Chicago, IL