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Safety, Tolerability, and Preliminary Efficacy of TAR-200 in Patients With Muscle-invasive Bladder Cancer Who Refused
Mark D Tyson1, David Morris2, Juan Palou3
1Mayo Clinic Arizona, Phoenix, Arizona.
Purpose:
Half of patients with muscle-invasive bladder cancer worldwide may not receive curative-intent therapy. Elderly or frail patients are most affected by this unmet need. TAR-200 is a novel, intravesical drug delivery system that provides sustained, local release of gemcitabine into the bladder over a 21-day dosing cycle. The phase 1 TAR-200-103 study evaluated the safety, tolerability, and preliminary efficacy of TAR-200 in patients with muscle-invasive bladder cancer who either refused or were unfit for curative-intent therapy.
Materials And Methods:
Eligible patients had cT2-cT3bN0M0 urothelial carcinoma of the bladder. TAR-200 was inserted for 4 consecutive 21-day cycles over 84 days. The primary end points were safety and tolerability at 84 days. Secondary end points included rates of clinical complete response and partial response as determined by cystoscopy, biopsy, and imaging; duration of response; and overall survival.
Results:
Median age of the 35 enrolled patients was 84 years, and most were male (24/35, 68.6%). Treatment-emergent adverse events related to TAR-200 occurred in 15 patients. Two patients experienced treatment-emergent adverse events leading to removal of TAR-200. At 3 months, complete response and partial response rates were 31.4% (11/35) and 8.6% (3/35), respectively, yielding an overall response rate of 40.0% (14/35; 95% CI 23.9-57.9). Median overall survival and duration of response were 27.3 months (95% CI 10.1-not estimable) and 14 months (95% CI 10.6-22.7), respectively. Progression-free rate at 12 months was 70.5%.
Conclusions:
TAR-200 was generally safe, well tolerated, and had beneficial preliminary efficacy in this elderly and frail cohort with limited treatment options.
Insights
TAR-200, a novel intravesical gemcitabine delivery system, shows promising safety and efficacy in elderly patients with muscle-invasive bladder cancer unfit for standard therapy. The treatment achieved a 40% overall response rate in a Phase 1 study.
Area of Science:
- Oncology
- Urology
- Drug Delivery Systems
Background:
- Muscle-invasive bladder cancer (MIBC) affects many patients, with a significant unmet need for curative-intent therapy in elderly or frail individuals.
- Current treatment options for MIBC are limited for patients who are ineligible for or refuse standard curative therapies.
Purpose of the Study:
- To evaluate the safety, tolerability, and preliminary efficacy of TAR-200, an intravesical drug delivery system, in patients with MIBC.
- TAR-200 provides sustained local release of gemcitabine over 21-day cycles for patients with MIBC who are unfit for or refuse curative treatment.
Main Methods:
- A Phase 1 study (TAR-200-103) enrolled 35 patients with cT2-cT3bN0M0 urothelial carcinoma of the bladder.
- Patients received TAR-200 for 4 consecutive 21-day cycles (84 days), with primary endpoints being safety and tolerability at 84 days.
- Secondary endpoints included clinical response rates, duration of response, and overall survival.
Main Results:
- TAR-200 was generally safe and well-tolerated in this cohort, with treatment-emergent adverse events in 15 patients, and two discontinuations due to adverse events.
- An overall response rate of 40.0% was observed, with a 31.4% complete response rate and an 8.6% partial response rate at 3 months.
- Median overall survival was 27.3 months, median duration of response was 14 months, and the 12-month progression-free rate was 70.5%.
Conclusions:
- TAR-200 demonstrated favorable safety and tolerability in elderly and frail patients with MIBC.
- Preliminary efficacy results, including response rates and survival data, suggest a beneficial role for TAR-200 in this patient population.
- TAR-200 represents a potential new treatment option for patients with MIBC who have limited therapeutic alternatives.
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