Protara’s lead candidate, TARA-002, has demonstrated durable complete response (CR) rates in a single-arm Phase II trial in Bacillus Calmette-Guérin (BCG)-naïve non-muscle invasive bladder cancer (NMIBC) patients.
During the open-label ADVANCED-2 (NCT05951179) study, the cell therapy triggered a complete response (CR) rate of 72% at any time in 29 patients.
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At the six-month mark, CR was achieved in 18 out of 26 (69%) of the evaluable patients. This effect was also maintained at 12 months in 50% of the evaluable patients (7 out of 14).
While five patients did not initially respond to treatment with TARA-002, four were able to achieve a CR at six months following re-induction therapy. CR was maintained in all four of these patients after 12-months.
The cell therapy also demonstrated a favourable tolerability profile, with a majority of treatment-emergent adverse events (TEAEs) observed classifying as Grade 1. There were no TEAEs rated Grade 3 or higher during the study period.
Protara’s stock value grew 9% after the news debuted, from $6.22 at market close on 2 December to $6.80 at close on 3 December.
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By GlobalDataTARA-002 acts through the TLR2/NOD2 pathway, which the company claims can activate both the innate and adaptive immune pathways within the bladder wall – allowing the therapy to both directly and indirectly kill tumour cells.
Protara originally pursued a study involving BCG-naïve patients after the US Food and Drug Administration (FDA) offered written feedback recommending a registrational path forward for TARA-002 in this population.
Alongside the ADVANCED-2 study, Protara is assessing the effectiveness of TARA-002 in BCG-unresponsive patients. Interim results from this registrational cohort are expected to debut in Q1 2026, while enrolment of BCG-unresponsive patients is set to finish in H2 2026.
Protara’s next steps for TARA-002
Following the positive results of the Phase II study, Protara’s CEO Jesse Shefferman said the New York-based biotech will now look to “finalise a regulatory pathway for TARA-002 in BCG-naïve patients”.
If the FDA were to approve TARA-002 for use in BCG-naïve NIMBC, it would become the first drug to obtain regulatory approval in this setting since the debut of BCG itself.
Analysts at GlobalData currently forecast that TARA-002 will launch in 2027, making $62m in its first year on the market. Looking to 2031, the drug is expected to make $452m for Protara.
While the BCG vaccine was originally created as a preventive measure for tuberculosis (TB), researchers uncovered its role in reducing the recurrence of NMIBC in the 1980s. Since then, the jab has been the standard of care (SoC) as first line treatment for this cancer due to its strong efficacy and safety profile.
Though targeted therapies have been approved to treat BCG-unresponsive patients – including MSD’s best-selling oncology asset, Keytruda (pembrolizumab) – nothing has yet obtained regulatory approval in the BCG-naïve setting.
There are companies, along with Protara, that are exploring the potential of their therapies in this patient population.
This includes enGene, which is currently assessing its gene therapy, detalimogene voraplasmid, in a Phase II study involving both BCG-naïve and unresponsive patients. The drug has already touted a CR rate of 63% across BCG-unresponsive patients, which has prompted the company to seek FDA approval in this setting.
Johnson & Johnson (J&J) released data from the Phase III CREST trial which combined sasanlimab, a PD-1 inhibitor, with BCG as induction therapy with or without maintenance in patients with BCG-naïve, high-risk NMIBC. The trial met its endpoints, however the big pharma company has not announced its regulatory plans for the combination.
J&J is also looking into the potential of Inlexzo (gemcitabine hydrochloride) in the SunRISe-3 study. Through this Phase III trial, the US-based pharma is comparing the efficacy of Inlexzo to the BCG vaccine, both as a monotherapy and alongside cetrelimab.
In 2031, detalimogene voraplasmid and sasanlimab, if approved, are expected to generate $435m and $924m, respectively. Inlexzo is expected to make $4.13bn in the same year.
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Cell & Gene therapy coverage on Clinical Trials Arena is supported by Cytiva.
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