US-FDA grants breakthrough therapy designation to Johnson & Johnson’s TAR-200 to treat high-risk NMIBC

Johnson & Johnson announced that the US Food and Drug Administration (FDA) has granted TAR-200 Breakthrough Therapy Designation (BTD) for the potential future treatment of patients with Bacillus Calmette-Guérin (BCG)-unresponsive high-risk non-muscle-invasive bladder cancer (HR-NMIBC), who are ineligible for or elected not to undergo radical cystectomy (surgical removal of the bladder). TAR-200 is a novel investigational targeted releasing system designed to provide sustained local release of gemcitabine into the bladder. Today’s BTD marks Johnson & Johnson’s 13th such designation in oncology.

“TAR-200 represents a novel interventional approach for the treatment of localized bladder cancer where today, unfortunately, options are limited and include antiquated BCG therapy or radical cystectomy,” said Kiran Patel, M.D., vice president, clinical development, Solid Tumours, Johnson & Johnson Innovative Medicine. “This Breakthrough Therapy Designation recognizes TAR-200 as a promising advancement and marks an important step forward in our innovative focus to transform the treatment of bladder cancer.”

The BTD is supported by data from SunRISe-1 (NCT04640623), an open-label phase 2b clinical study evaluating the safety and efficacy of TAR-200 in combination with cetrelimab, TAR-200 alone, or cetrelimab alone for BCG-unresponsive HR-NMIBC carcinoma in situ (CIS) patients, who are ineligible for or elected not to undergo radical cystectomy. Data from the SunRISe-1 study were featured during the 2023 European Society for Medical Oncology Annual Congress as a late-breaking mini-oral presentation (Abstract #LBA105) and interim results were presented at the 2023 American Urological Association Annual Meeting (Abstract #LBA02-03).

A US FDA BTD is granted to expedite the development and regulatory review of a medicine that is intended to treat a serious or life-threatening condition and is based on preliminary clinical evidence that demonstrates the drug may have substantial improvement over available therapies on a clinically significant endpoint(s).

SunRISe-1 (NCT04640623) is an open-label phase 2b clinical study evaluating the safety and efficacy of TAR-200 in combination with cetrelimab, TAR-200 alone, or cetrelimab alone for BCG-unresponsive HR-NMIBC carcinoma in situ (CIS) patients, who are ineligible for or elected not to undergo radical cystectomy. Participants were randomized to one of three cohorts: treatment with TAR-200 in combination with cetrelimab (Cohort 1), TAR-200 alone (Cohort 2), or cetrelimab alone (Cohort 3). The primary endpoint is Complete Response rate at any time point. Secondary endpoints include duration of response, overall survival, pharmacokinetics, quality of life, safety, and tolerability. Cohorts 1 and 3 were closed to further enrollment effective June 1, 2023.

TAR-200 is an investigational targeted releasing system enabling controlled release of gemcitabine into the bladder, sustaining local drug exposure for weeks at a time. The safety and efficacy of TAR-200 are being evaluated in phase 2 and phase 3 studies in patients with muscle-invasive bladder cancer in SunRISe-2 and SunRISe-4 and NMIBC in SunRISe-1 and SunRISe-3.

Administered intravenously, cetrelimab is an investigational programmed cell death receptor-1 (PD-1) monoclonal antibody being studied for the treatment of bladder cancer, prostate cancer, melanoma, and multiple myeloma as part of a combination regimen.

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- and intermediate risk NMIBC. HR-NMIBC makes up 15–44 percent of patients with NMIBC and is characterized by a high-grade, large tumour size, presence of multiple tumours and CIS. Radical cystectomy is currently recommended for NMIBC patients who are unresponsive to BCG therapy, with over 90 per cent cancer-specific survival if performed before muscle-invasive progression. Given that NMIBC typically affects older patients, many may be unwilling or unable to undergo radical cystectomy. The high rates of recurrence and progression can pose significant morbidity and distress for patients with NMIBC.

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