A Phase 2 Study of Vesimune®, intravesical imiquimod, for the Treatment of Carcinoma in Situ Bladder Cancer

AUA poster presentation, Saturday, May 7, 2016 10:30 AM-11:30 AM

A Phase 2 Study of Vesimune®, intravesical imiquimod, for the Treatment of Carcinoma in Situ Bladder Cancer
Nicholas Donin*, Karim Chamie, Los Angeles, CA, Madhu Reddy, Philadelpia, PA, Dana Kivlin, Philadelphia, PA, Johanna Holldack, Raffaella Pozzi, Bioggio, Switzerland, Gil Hakim, Ra’anana, Israel, Stuart Holden, Los Angeles, CA, Lawrence Karsh, Denver, CO, Donald Lamm, Phoenix, AZ, Laurence Belkoff, Philadelphia, PA, Arie Belldegrun, Los Angeles, CA, Neal Shore, Myrtle Beach, NC

Abstract: LB-S&T-21
Introduction and Objectives
Imiquimod is a toll like receptor agonist with proven anti-tumor activity as a topical treatment for skin cancer. Vesimune® 0.4% is a novel liquid formulation of imiquimod optimized for intravesical delivery. The agent demonstrated safety as an intravesical treatment for non-muscle-invasive bladder cancer in a phase 1 clinical trial. Herein we report the results of a phase 2 clinical trial assessing the safety and activity of intravesical Vesimune®.

We performed a phase 2 multicenter prospective clinical trial in patients with bladder carcinoma in situ. Patients with concomitant papillary disease were allowed to enroll if all papillary lesions had been completely resected. Enrolled patients received 6-weekly intravesical administrations of 200mg/50mL Vesimune® 0.4%. The primary study endpoint was response rate at 6 weeks following the last instillation, determined by post-treatment biopsies and urine cytology. Secondary endpoints included the rate of adverse events, and changes in urinary cytokine levels pre- and post-treatment.

12 patients were enrolled, with 10 available for efficacy analysis. The majority were male (92%) and caucasian (83%). Median duration of CIS since diagnosis was 7.4 months (range 0.4–109.5), with median number of 2 previous TURBTs (range 0-10). 67% (8/12) patients had received prior BCG, with 50% (6/12) having received ≥2 courses. Four patients were free from disease at 6 weeks post-instillation based on local cytology and biopsy results. In the safety cohort 92% (11/12) experienced a total of 51 adverse events, all but 1 of which were mild or moderate. All 12 patients enrolled received 6 doses of Vesimune® per protocol. No patient required a dose reduction during the study. Significant increases were seen in levels of several urinary cytokines following treatment, including IL-6 and IL-18.

Vesimune® is safe and well-tolerated. Clinical activity was suggested by the clinical responses and the increase in post-treatment urinary cytokines. Further prospective investigation of Vesimune ®is warranted given the positive signal demonstrated herein.

Date & Time: May 7-10, 2016 10:30 AM-11:30 AM
Session Title: Late-Breaking Science & Technology Poster
Sources of Funding: The research leading to these results received funding from the European Community Seventh Framework Programme – FP7-2007-2013 – under grand agreement nHEALTH-F4-2011-281608 (TIMER). Telormedix supported this work.


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