Johnson & Johnson (J&J) has announced promising new data supporting the efficacy of TAR-200, its innovative intravesical drug release system, in patients with Bacillus Calmette-Guérin (BCG)-unresponsive, high-risk non-muscle-invasive bladder cancer (HR-NMIBC).
The latest results, presented at the American Urological Association (AUA) Annual Meeting 2025 in Las Vegas, showed impressive response rates in two cohorts from the Phase IIb SunRISe-1 trial, further strengthening the company's New Drug Application (NDA) submitted to the FDA earlier this year.
Strong Efficacy Data Across Multiple Patient Populations
In cohort two, which enrolled 85 BCG-unresponsive NMIBC patients with carcinoma in situ, 82.4% achieved a complete response (CR). More importantly, this translated into sustained disease control, with 52.9% of responders maintaining CR at one year. The median duration of response (DOR) reached 25.8 months, and at 12 months, 86.6% of patients who responded to treatment remained cystectomy-free.
Cohort four, which included 52 HR-NMIBC patients with papillary disease-only, demonstrated similarly impressive results with 85.3% and 81.1% disease-free survival (DFS) rates at six and nine months, respectively. Notably, 94.2% of patients avoided cystectomy at a median follow-up of 12.8 months. The progression-free survival (PFS) and overall survival (OS) rates at nine months were 95.6% and 98%, respectively.
Dr. Christopher Cutie, disease area leader of bladder cancer for J&J Innovative Medicine, emphasized the significance of these findings: "Bladder cancer is one of the ten most common cancers worldwide, yet treatment options have remained largely unchanged for over 40 years, leaving patients with few choices if initial BCG therapy does not work. TAR-200 is designed to allow for sustained delivery of medication directly into the bladder through a brief and routine procedure, which benefits patients. These data now show patients can remain cancer-free for a meaningful period of time, marking a significant step forward for those facing this challenging disease."
Innovative Drug Delivery Technology
TAR-200, trademarked as GemRIS, represents a novel approach to bladder cancer treatment. The drug-device combination product continuously delivers gemcitabine chemotherapy directly to the bladder over a week. A key advantage is that it can be inserted without the need for anesthesia or sedation, potentially improving patient comfort and treatment adherence.
J&J acquired TAR-200 and the underlying drug delivery technology during its purchase of Taris Biomedical in 2019, highlighting the company's strategic investment in innovative treatment modalities for difficult-to-treat cancers.
Safety Profile and Patient Tolerability
Across both cohorts, TAR-200 demonstrated a favorable safety profile. Most adverse events (AEs) were mild urinary symptoms and generally manageable. Between both cohorts, only seven patients discontinued treatment due to treatment-related AEs, and there were no treatment-related deaths reported.
This safety profile is particularly important for bladder cancer patients, who often face challenging treatment regimens with significant side effects.
Regulatory Pathway and Future Development
J&J filed an NDA to the US Food and Drug Administration (FDA) for TAR-200 in January 2025 based on previous data from the SunRISe-1 trial. The application specifically targets NMIBC patients with carcinoma in situ who are BCG-unresponsive, with or without papillary tumors.
The submission is being reviewed through the FDA's Real-Time Oncology Review (RTOR) program, which allows the agency to review data before the complete application is formally submitted, potentially expediting the approval process.
While TAR-200 has shown promise in NMIBC, it's worth noting that the treatment did not demonstrate benefit in a Phase III trial (SunRISe-2) in patients with muscle-invasive bladder cancer (MIBC) not undergoing radical cystectomy. This study was terminated by J&J after an independent data monitoring committee indicated the trial was unlikely to meet its primary endpoint of bladder intact-event free survival.
Nevertheless, the SunRISe-1 trial continues, and TAR-200 is being investigated in NMIBC in two additional Phase III studies: SunRISe-3 (NCT05714202) and SunRISe-5 (NCT06211764).
Competitive Landscape in Bladder Cancer Treatment
The bladder cancer treatment landscape is evolving, with other companies also reporting progress. At the same AUA Annual Meeting, Pfizer presented positive data from a Phase III trial of sasanlimab and BCG, with the combination therapy improving event-free survival in BCG-naïve high-risk NMIBC patients compared to subjects who received BCG alone.
These developments collectively suggest that patients with bladder cancer may soon have access to more effective treatment options, addressing a significant unmet medical need in this patient population.
Clinical Implications
The strong efficacy and safety data from the SunRISe-1 trial position TAR-200 as a potentially important addition to the treatment armamentarium for BCG-unresponsive HR-NMIBC. The high complete response rates and durable responses are particularly encouraging for a patient population with limited therapeutic options.
If approved, TAR-200 could offer patients a non-surgical alternative that preserves bladder function while effectively controlling cancer, potentially improving both survival outcomes and quality of life for those affected by this challenging disease.
