TAR-210 Yields 90% Recurrence-Free Survival and Complete Response in High-Risk and Intermediate-Risk NMIBC

Updated results have highlighted the potential of TAR-210, a targeted delivery system for treating non–muscle-invasive bladder cancer (NMIBC) with fibroblast growth factor receptor (FGFR) alterations. Johnson & Johnson announced these findings from an ongoing open-label, multicenter Phase 1 study at the 2024 American Urological Association (AUA) Annual Meeting in San Antonio, Texas.

The study evaluates the safety and efficacy of TAR-210, which delivers the drug erdafitinib directly into the bladder via an intravesical system. Erdafitinib is already FDA-approved as BALVERSA® for advanced urothelial carcinoma with FGFR3 genetic alterations. The current study involves patients with high-risk (HR) and intermediate-risk NMIBC who exhibit specific FGFR changes.

The results focused on two cohorts: Cohort 1 (C1) includes patients with recurrent, Bacillus Calmette-Guérin (BCG)-unresponsive HR-NMIBC who declined or were ineligible for radical cystectomy, and Cohort 3 (C3) includes patients with recurrent, intermediate-risk NMIBC. In C1, the 12-month recurrence-free survival rate reached 90%, while in C3, 90% of patients achieved a complete response.

Dr. Antoni Vilaseca from the Hospital Clínic de Barcelona, the study's presenting author, emphasized that these findings suggest TAR-210 could offer a promising alternative for patients with limited treatment options. By March 22, 2024, 64 patients had been treated with TAR-210, showing no dose-limiting toxicities or deaths. However, some patients experienced Grade 1/2 lower urinary tract events, and two patients discontinued the study due to adverse events.

Dr. Sabine Brookman-May, Vice President of Late Development Oncology at Johnson & Johnson, remarked that the presence of FGFR genetic alterations in NMIBC underpins the potential of TAR-210 as a bladder-sparing, BCG-free treatment option. This aligns with Johnson & Johnson's commitment to developing innovative therapies for patients with limited treatment avenues.

Bladder cancer is the sixth most common cancer in the U.S., with over 83,000 new cases annually. NMIBC accounts for about 75-85% of these cases. Standard treatments include BCG immunotherapy or intravesical chemotherapy, but 30-40% of patients do not respond to BCG, often leading to disease recurrence or progression. In such instances, radical cystectomy is a primary treatment option, though it involves significant surgical intervention and potential complications.

TAR-210, still under investigation, aims to provide sustained local release of erdafitinib, potentially transforming the treatment landscape for NMIBC patients with FGFR alterations. The Phase 1 study also includes patients with muscle-invasive bladder cancer (MIBC) and categorizes them into four cohorts based on disease presentation. Primary endpoints are safety and adverse events, while secondary endpoints include recurrence-free survival and complete response rates.

Erdafitinib as BALVERSA® is approved for advanced urothelial carcinoma but comes with notable safety considerations, including risks of ocular disorders and hyperphosphatemia. BALVERSA® received Breakthrough Therapy Designation from the FDA in 2018 and accelerated approval in 2019 for treating advanced urothelial carcinoma with susceptible FGFR genetic alterations.

Johnson & Johnson continues to explore new therapeutic frontiers, seeking to improve patient outcomes through innovative treatments like TAR-210 and BALVERSA® for bladder cancer. The ongoing research and promising results reinforce the company's dedication to addressing unmet medical needs and advancing cancer care.