Protara Announces Promising Phase 2 Results for TARA-002 in High-Risk NMIBC

Protara Therapeutics has announced encouraging results from the ongoing phase 2 ADVANCED-2 trial, showcasing the potential of its investigational cell-based therapy, TARA-002, in treating high-risk non-muscle invasive bladder cancer (NMIBC). The study evaluates TARA-002 as an intravesical treatment for NMIBC patients with carcinoma in situ (CIS) with or without Ta/T1 disease who are either Bacillus Calmette-Guérin (BCG)-unresponsive or BCG-naïve. The results were presented at the SUO 25^th Annual Meeting.

The trial reported a six-month complete response (CR) rate of 72% across BCG exposures, with a CR rate of 70% observed at any time point. In the cohort of BCG-unresponsive patients, the six-month CR rate was 100%, with 80% achieving CR at any time. The proof-of-concept cohort of BCG-naïve patients demonstrated a six-month CR rate of 64%, with a CR rate of 67% at any time. Notably, 100% of patients who achieved a CR at three months maintained their response through six months, and two of three patients maintained a CR at nine months.

TARA-002 demonstrated a favorable safety profile, with no grade 2 or higher treatment-related adverse events reported. Most adverse events were grade 1 and transient, including flu-like symptoms and bladder-related irritations such as spasms and burning sensations, which resolved within hours to days post-treatment. No patients discontinued therapy due to adverse events.

The ADVANCED-2 trial includes 20 patients evaluable at three months, 18 at six months, and three at nine months, with data collected as of November 19, 2024. The study’s positive outcomes across both BCG-naïve and BCG-unresponsive cohorts underscore TARA-002’s promise as a bladder-preserving therapy with manageable safety considerations and low procedural burden for physicians.

Dr. Brian Mazzarella, a study investigator, noted that TARA-002’s activity across BCG exposures and ease of use positions it as a compelling therapeutic option for NMIBC. Jesse Shefferman, CEO of Protara Therapeutics, expressed optimism about the international expansion of the trial and the upcoming release of 12-month data in mid-2025.

The findings from the ADVANCED-2 trial highlight TARA-002’s potential to address significant unmet needs in NMIBC treatment, particularly in patients who have limited options due to BCG resistance or unavailability. The promising efficacy, durability of response, and favorable safety profile set the stage for further clinical development.