SUMMARY

• TAR-200-103 (NCT03404791) was a global, phase 1, open-label, multicenter study that evaluated the preliminary efficacy, safety, and tolerability of TAR-200 in patients with muscle-invasive bladder cancer (MIBC) who were unfit for curative intent therapy.^1-3
  • Safety/tolerability at 84 days was the primary endpoint of the study.
  • At a data cut-off of September 18, 2020¹:
    • Treatment-emergent adverse events (TEAEs) related to TAR-200 occurred in 15 (42.9%) patients and were mostly grade ≤2.
    • Median overall survival (OS) was 20.1 months. Overall response rate (ORR) at 3 months was 40%, and progression-free rate at 12 months was 67.7%.
  • At a data cut-off of February 25, 2022³:
    • The safety profile was consistent with that of the previous analysis.
    • Median OS was 27.3 months. ORR at 3 months was 40%, and progression-free rate at 12 months was 70.5%.

BACKGROUND

• TAR-200 (JNJ-17000139) is an investigational intravesical system that is designed to provide local release of gemcitabine in the bladder. TAR-200 contains gemcitabine and urea mini tablets within a dual-lumen silicone tube for gradual release of gemcitabine by an osmotic delivery mechanism throughout the prescribed indwelling period.^1,4-8  
• TAR-200 is inserted intravesically via urinary placement catheter, after which it self-coils into a bi-oval shape. Removal of TAR-200 can be achieved using grasping forceps and flexible cystoscopy.^5,8

CLINICAL DATA

TAR-200-103 Study

Study Design/Methods

• Phase 1, open-label, multicenter study designed to assess the preliminary efficacy, safety, and tolerability of TAR-200 in patients with MIBC who were unfit for curative intent therapy (N=35).^1-3
• The study design is shown in Figure: TAR-200-103 Study Design.

Patient Characteristics

• A total of 35 patients were enrolled in the study at data cutoff (September 18, 2020).¹
• The baseline demographics and disease characteristics are described in Table: Patient Baseline Characteristics.

Efficacy

• At a data cut-off of September 18, 2020¹:
  • At 3 months, rates of clinical complete response (cCR) and clinical partial response (cPR) were 31.4% and 8.6%, respectively, with an ORR of 40% (95% CI, 23.957.9).
  • Median OS was 20.1 months (95% CI, 10.1-not estimable [NE]) with 12 patients still in follow-up at the time of data cutoff.
• At a data cut-off of February 25, 2022³:
  • Median OS was 27.3 months (95% CI 10.1-NE).
  • Median PFS was 9.5 months (95% CI, 4.1-15.6).
• Efficacy outcomes are described in Table: Efficacy Outcomes.

Safety

• At a data cut-off of September 18, 2020¹:
  • TAR-200-related TEAEs occurred in 15 (42.9%) patients and were mostly grade ≤2.¹
  • Overall, 2 (5.7%) patients experienced TEAEs that led to removal of TAR-200.¹
  • None of the TEAEs that led to study discontinuation (17.1%) were treatment-related.¹
  • Clot evacuation and blood transfusion were required in 1 patient each.¹
• At a data cut-off of February 25, 2022³:
  • Safety profile was consistent with that of the previous analysis.
  • Overall, 1 patient experienced a treatment-related TEAE (urinary incontinence) that led to the removal of TAR-200.
• A summary of TAR-200-related TEAEs is shown in Table: TAR-200-Related TreatmentEmergent Adverse Events.

Literature Search

A literature search of MEDLINE^®, Embase^®, BIOSIS Previews^®, and Derwent Drug File (and/or other resources, including internal/external databases) was conducted on 23 January 2025.