SAN ANTONIO, May 5, 2024 – Johnson & Johnson announced today updated results from an open-label, multicenter, multi-cohort Phase 1 study of the safety and efficacy of TAR-210, an intravesical targeted releasing system designed to provide sustained, local release of erdafitinib into the bladder, in patients with non–muscle-invasive bladder cancer (NMIBC) with select FGFR alterations. These data were featured today in an Oral Presentation Session (Abstract # PD48-02) at the 2024 American Urological Association (AUA) Annual Meeting taking place May 3-6, 2024, in San Antonio, Texas.
Results featured updated data from Cohort 1 (C1), patients with recurrent, Bacillus Calmette-Guérin (BCG)–unresponsive high-risk (HR) NMIBC (high-grade Ta/T1; papillary only) who refused or were ineligible for radical cystectomy and Cohort 3 (C3), patients with recurrent, intermediate-risk NMIBC (Ta/T1) low-grade papillary disease left in situ as tumor marker lesions. First results were featured at the European Society for Medical Oncology 2023 Congress, with interim results presented at the European Association of Urology (EAU) 2024 Annual Congress.
“Advancement in the treatment landscape of high- or intermediate-risk non–muscle-invasive bladder cancer has remained stagnant for more than 50 years,” said Antoni Vilaseca,* M.D., Ph.D., of the Hospital Clínic de Barcelona, presenting author of the Phase 1 TAR-210 study. “Results presented today further underscore that TAR-210 for the localized treatment of bladder cancer may offer a promising alternative for patients with limited treatment options.”
At the data cutoff of March 22, 2024, 64 patients had been treated with TAR-210 across the 2 cohorts. Of the 21 patients in C1 with HR-NMIBC, the 12-month recurrence-free (RF) survival rate was 90%. In C3, 31 patients were efficacy evaluable with a complete response (CR) rate of 90%.1
The most common treatment emergent adverse events (TEAEs) were Grade 1/2 lower urinary tract events. There were no dose-limiting toxicities and no deaths. Two patients (3%) discontinued the study due to TEAEs of low-grade urinary symptoms and two patients had serious TEAEs with pyelonephritis and sepsis or UTI and sepsis, respectively.1
“FGFR genetic alterations are most common in NMIBC,” said Sabine Brookman-May, M.D., Vice President, Late Development Oncology, Johnson & Johnson Innovative Medicine. “These results further support the potential of TAR-210 with quarterly administration as a bladder-sparing and BCG-free treatment option, underscoring our deep commitment to pioneering novel therapies for patients who face limited treatment avenues.”
TAR-210 is an investigational targeted releasing system designed to provide sustained local release of erdafitinib. Oral erdafitinib was approved by the U.S. Food and Drug Administration (FDA) as BALVERSA® (erdafitinib) for patients with locally advanced or metastatic urothelial carcinoma (mUC) with susceptible FGFR3 genetic alterations that have progressed on or after at least one line of prior systemic therapy. BALVERSA® is not recommended for the treatment of patients who are eligible for and have not received prior PD-1 or PD-L1 inhibitor therapy.2
Bladder cancer ranks as the sixth most prevalent cancer in the U.S., with over 83,000 individuals receiving diagnoses annually.3 NMIBC constitutes approximately 75-85% of these cases.4 Currently, adjuvant intravesical immunotherapy with BCG or intravesical chemotherapy is the standard of care for patients with intermediate- and high-risk NMIBC.5 Between 30 and 40% of patients do not respond to BCG, facing disease recurrence or progression.6 In such scenarios of HR-NMIBC, radical cystectomy (removal of the bladder) emerges as the primary treatment option. This major abdominal procedure requires a urinary diversion to be created to collect and store urine.7
About TAR-210
TAR-210 is an investigational erdafitinib intravesical targeted releasing system. The safety and efficacy of TAR-210 is being evaluated in a Phase 1 study (NCT05316155) in patients with muscle-invasive bladder cancer (MIBC) and NMIBC. The study categorizes patients into 4 cohorts based on their disease presentation. Cohort 1 includes patients with recurrent, BCG-unresponsive high-risk NMIBC with concomitant high-grade papillary disease who have refused or are ineligible for radical cystectomy (RC). Cohort 2 includes patients with the same presentation, but who are scheduled for RC. Cohort 3 includes patients with recurrent, intermediate-risk NMIBC with a history of low-grade papillary disease. To be eligible for C3, the presence of visible tumor(s) is required. Cohort 4 includes patients with MIBC. The primary endpoint of the study is safety (adverse events, including dose-limiting toxicity). Secondary endpoints include pharmacokinetics, RF survival in patients in C1 and C2, CR rate and duration of CR in patients in C3 and pathologic CR rate in C4.8
About BALVERSA®
BALVERSA® (erdafitinib) is a once-daily, oral FGFR kinase inhibitor indicated for the treatment of adult patients with locally advanced or mUC with susceptible fibroblast growth factor receptor 3 (FGFR3) genetic alterations whose disease has progressed on or after at least one line of prior systemic therapy. BALVERSA ® is not recommended for the treatment of patients who are eligible for and have not received prior PD-1 or PD-(L)1 inhibitor therapy.2 Patients are selected for therapy based on an FDA-approved companion diagnostic for BALVERSA®. Information on FDA-approved tests for the detection of FGFR genetic alterations in urothelial cancer is available at: http://www.fda.gov/CompanionDiagnostics.
BALVERSA® received Breakthrough Therapy Designation from the U.S. FDA in 2018 and received accelerated approval in 2019 for the treatment of adults with locally advanced or mUC that has susceptible FGFR3 or fibroblast growth factor receptor 2 (FGFR2) genetic alterations and who have progressed during or following at least one line of prior platinum-containing chemotherapy, including within 12 months of neoadjuvant or adjuvant platinum-containing chemotherapy.2
The Company submitted a marketing authorization application to the European Medicines Agency in September 2023 for BALVERSA ® as a treatment for adult patients with FGFR3-altered, locally advanced unresectable or mUC that has progressed following therapy with a PD-(L)1 inhibitor.
In 2008, Janssen Pharmaceuticals entered into an exclusive worldwide license and collaboration agreement with Astex Pharmaceuticals to develop and commercialize BALVERSA®.
For more information, visit www.BALVERSA.com.
About High-Risk Non–Muscle-Invasive Bladder Cancer
High-risk non-muscle-invasive bladder cancer (HR-NMIBC) is a type of non-invasive bladder cancer that is more likely to recur or spread beyond the lining of the bladder, called the urothelium, and progress to invasive bladder cancer compared to low-risk NMIBC. HR-NMIBC makes up 15-44% of patients with NMIBC and is characterized by a combination of high-grade, large tumor size, presence of multiple tumors, and carcinoma in situ. Radical cystectomy (RC) is currently recommended for NMIBC patients who fail BCG therapy, with over 90% cancer-specific survival if performed before muscle-invasive progression. Given that NMIBC typically affects older patients, many may be unwilling or unfit to undergo RC. The high rates of recurrence and progression can pose significant morbidity and distress for these patients.9
About Johnson & Johnson
At Johnson & Johnson, we believe health is everything. Our strength in healthcare innovation empowers us to build a world where complex diseases are prevented, treated, and cured, where treatments are smarter and less invasive, and solutions are personal. Through our expertise in Innovative Medicine and MedTech, we are uniquely positioned to innovate across the full spectrum of healthcare solutions today to deliver the breakthroughs of tomorrow, and profoundly impact health for humanity. Learn more at https://www.jnj.com/ or at www.janssen.com/johnson-johnson-innovative-medicine. Follow us at @JanssenUS and @JNJInnovMed. Janssen Research & Development, LLC, and Janssen Biotech, Inc., are both Johnson & Johnson companies.
*Dr. Vilaseca has not been paid for any media work.
1.Vilaseca A, Jayram G, Raventos C, Shore ND, Zainfeld D, Kang TW, et al. PD48-02 first safety and efficacy results of the TAR-210 erdafitinib intravesical delivery system in patients with non–muscle-invasive bladder cancer with select FGFR alterations. Journal of Urology. 2024;211.
2.BALVERSA® [Prescribing Information]. Horsham, PA: Janssen Biotech, Inc.
3.American Cancer Society. Cancer facts & figures 2024. Atlanta: American Cancer Society; 2024.
4.Deng S, Meng F, Wang L, et al. Global research trends in non–muscle invasive bladder cancer: Bibliometric and visualized analysis. Front Oncol. 2022;12:1044830. Published 2022 Nov 17. Doi:10.3389/fonc.2022.1044830
5.Laukhtina E, Abufaraj M, Al-Ani A, et al; European Association of Urology-Young Academic Urologists (EAU-YAU): Urothelial carcinoma working group. Intravesical therapy in patients with intermediate-risk non–muscle-invasive bladder cancer: A systematic review and network meta-analysis of disease recurrence. Eur Urol Focus. 2022;8(2):447-456. doi: 10.1016/j.euf.2021.03.016. Epub 2021 Mar 21. PMID: 33762203
6.Zlotta AR, Fleshner NE, Jewett MA. The management of BCG failure in non–muscle-invasive bladder cancer: an update. Can Urol Assoc J. 2013;3(6-S4):199.
7.Bladder removal surgery: What is a radical cystectomy? Bladder Cancer Advocacy Network. Accessed April 1, 2024. https://bcan.org/bladder-removal-surgery/.
8.Vilaseca A, Guerrero F, Zainfeld D, Shore ND, Rodriguez Faba O, Meijer RP, et al. Safety and efficacy of the erdafitinib (erda) intravesical delivery system, TAR-210, in patients (pts) with non–muscle-invasive bladder cancer (NMIBC) or muscle-invasive bladder cancer (MIBC) harboring select FGFR mutations or fusions: Phase 1 first-in-human study. Journal of Clinical Oncology. 2023;41.
9.Brooks NA, O’Donnell MA. Treatment options in non–muscle-invasive bladder cancer after BCG failure. Indian J Urol. 2015;31(4):312-319. Accessed March 20, 2024. doi:10.4103/0970-1591.166475