TAR-200 Monotherapy Yields Over 80% Disease-Free Survival in BCG-Unresponsive High-Risk Papillary NMIBC

Johnson & Johnson has unveiled promising preliminary outcomes from the Phase 2b SunRISe-1 study, specifically Cohort 4, focusing on TAR-200, a novel treatment for patients with high-risk non-muscle-invasive bladder cancer (HR-NMIBC) who do not respond to Bacillus Calmette-Guérin (BCG) therapy. The study results, presented at the 2025 American Urological Association Annual Meeting, demonstrate significant potential for TAR-200 to serve as an alternative to radical cystectomy, the current standard but life-altering treatment for this patient group.

The study monitored patients with high-grade papillary bladder tumors and found that TAR-200 could achieve an impressive 94% bladder preservation rate, indicating the possibility of avoiding radical cystectomy for the majority of patients. Additionally, a progression-free survival rate of 95% at nine months underscores TAR-200's promise in managing this high-risk cancer type.

TAR-200 works by delivering sustained doses of gemcitabine directly into the bladder, which differs significantly from traditional treatments that often necessitate systemic administration. This targeted approach allows patients to maintain normal activities without the invasive nature of surgery. The device is inserted during a brief outpatient procedure and remains in the bladder, continuously releasing treatment over a sustained period.

Data from the trial’s interim analysis revealed disease-free survival (DFS) rates of 85.3% and 81.1% at six and nine months, respectively. Among patients with specific disease classifications, notably high-grade Ta and T1 tumors, the DFS rates were similarly robust, affirming TAR-200's efficacy across different tumor invasions. The study also highlights that 94.2% of participants could avoid undergoing radical cystectomy after a median follow-up of 12.8 months.

The safety profile of TAR-200 was consistent with previous findings, showing that most treatment-related adverse events (TRAEs) were mild and resolved quickly, typically within a few weeks. Common adverse effects included dysuria, frequent urination, and urgency, with serious TRAEs occurring in only a small fraction of patients.

Bladder cancer ranks among the top ten most prevalent cancers globally, affecting around a million people annually. Despite recent advances, the standard treatment for HR-NMIBC has remained largely unchanged for several decades, with radical cystectomy presenting significant challenges, especially for older patients often unwilling or unable to undergo such a drastic procedure.

The ongoing development and evaluation in the Phase 3 SunRISe-5 study aim to compare TAR-200 directly with chemotherapy in patients who have undergone BCG treatment, potentially setting the stage for a paradigm shift in bladder cancer treatment. TAR-200's ability to deliver medication effectively within the bladder represents a novel approach that could substantially alter treatment pathways for early-stage bladder cancer.

Johnson & Johnson's commitment to advancing TAR-200 is further underscored by its recent regulatory progress. The company has initiated a new drug application with the FDA under the real-time oncology review program, and TAR-200 has already received Breakthrough Therapy Designation for treating certain bladder cancer patients. With these developments, TAR-200 stands poised to become a transformative treatment option for patients with limited alternatives.