J&J files intravesical bladder cancer drug TAR-200 in US
Johnson & Johnson has kicked off a marketing application in the US for TAR-200 as a treatment for a form of bladder cancer, using a novel technology that releases the drug intravesically, or directly into the bladder.
The FDA application is for the use of TAR-200 as a treatment for high-risk, non-muscle-invasive bladder cancer (NMIBC) that does not respond to BCG, a live bacterial vaccine that is the standard first-line treatment.
J&J's drug features a silicone-based drug delivery system that allows for the continuous release of gemcitabine chemotherapy and is administered into the bladder via a catheter.
The FDA has accepted the filing under its Real-Time Oncology Review (RTOR) programme, which aims to speed up access to potentially groundbreaking new therapies, based on the results of phase 2b SunRISe-1 study. It has also been granted breakthrough status by the agency.
In the trial, which was presented at last year's ESMO cancer congress, monotherapy with TAR-200 achieved an 83.5% complete response (CR) rate, with 82% of them maintaining that status after nine months.
There are still some questions about the durability of the response, given that at 12 months the CR rate had dipped to 57%. However, the data still looks strong compared to the 41% CR rate seen with MSD's cancer immunotherapy Keytruda (pembrolizumab) at three months in the KeyNote-057 study.
J&J acquired rights to TAR-200 when it bought privately-owned biotech Taris for an undisclosed sum in 2019, and has big sales expectations for the drug, even though it recently failed a second trial in muscle-invasive bladder cancer (MIBC), a potential follow-up indication.
J&J is developing a second Taris-derived candidate called TAR-210, an intravesical formulation of J&J's FGFR kinase inhibitor Balversa (erdafitinib), and has predicted that peak sales of the two drugs could reach $5 billion at peak.
J&J's oncology head, Yusri Elsayed, said that - if approved - TAR-200 will be a "meaningful additional treatment option for certain patients with NMIBC, addressing a critical need for people who have had relatively limited therapeutic alternatives." It could also help some people to avoid surgery to remove the bladder entirely, he suggested.
Options for BCG-unresponsive NMIBC have expanded dramatically in the last few years. While it used to be that options were limited to regular intravesical chemotherapy or surgery, that changed with the approval of Keytruda in 2020 and since then additional options have come to market, including Ferring's intravesical gene therapy Adstiladrin (nadofaragene firadenovec) and ImmunityBio's IL-15 receptor agonist Anktiva (nogapendekin alfa inbakicep), which both launched in the US last year.
Adstiladrin showed a 53% CR rate at three months in its pivotal trial, although, that fell to 24% at 12 months, while Anktiva's efficacy was very close to TAR-200, with an initial rate of 84% that fell to 57% after a year. Another potential rival - CG Oncology's retostimogene grenadenorepvec - is in phase 3 for BCG-unresponsive NMIBC.
J&J is still hopeful of finding a role for TAR-200 in the smaller indication of MIBC, despite the setback in the SunRISe-2 trial, and is also hoping to position the drug as an alternative to BCG in the first-line treatment setting, based on the ongoing SunRISe-3 trial, due to read out next year.
