Protara Therapeutics Reports Positive Interim Results in Phase 2 ADVANCED-2 Trial of TARA-002 for NMIBC

Protara Therapeutics, Inc., a company focused on developing innovative treatments for cancer and rare diseases, has announced promising interim results from its Phase 2 ADVANCED-2 trial. This open-label study is evaluating the efficacy of TARA-002, the company's investigational cell-based therapy, for patients with high-risk Non-Muscle Invasive Bladder Cancer (NMIBC) who either have not responded to Bacillus Calmette-Guérin (BCG) treatment or have yet to receive it.

TARA-002 has shown significant results, particularly in BCG-Unresponsive patients, achieving a complete response rate of 100% at any point throughout the study, as well as a 67% complete response rate at the 12-month mark. This success is noteworthy given the limited treatment options available for this group of patients. BCG-Unresponsive patients typically face the challenging option of radical cystectomy, which is difficult to endure and often associated with considerable risks.

In the BCG-Naïve group, TARA-002 demonstrated a 76% complete response rate at any time and a 43% complete response rate at 12 months. The trial's interim results encompass 21 participants, with a significant number reaching the six-month, nine-month, and 12-month evaluation points.

The safety profile of TARA-002 has also been favorable. Most adverse events reported were mild and transient, with no treatment-related adverse events of Grade 3 or higher. Common side effects included flu-like symptoms and urinary tract-related issues, all of which resolved shortly after treatment.

Tom Jayram, M.D., a study investigator and Director of the Advanced Therapeutics Center at Urology Associates, expressed optimism about TARA-002's potential to integrate seamlessly into clinical practice. The therapy's efficacy and ease of use are seen as key advantages that could significantly impact the treatment landscape for high-risk NMIBC patients.

Jesse Shefferman, Chief Executive Officer of Protara Therapeutics, highlighted the impressive 12-month complete response rates in both the BCG-Unresponsive and BCG-Naïve cohorts. He emphasized the company's commitment to advancing this trial with the aim of providing transformative therapies to patients in need.

As the trial progresses, Protara plans to present further interim data from approximately 25 BCG-Unresponsive patients by the end of 2025. This continued research underscores the company's dedication to addressing the needs of NMIBC patients with innovative therapeutic approaches.

TARA-002, developed from the same cell bank as the immunopotentiator OK-432, has shown potential not only in NMIBC but also in treating lymphatic malformations, for which it has been granted a Rare Pediatric Disease Designation by the U.S. Food and Drug Administration. The investigational therapy works by activating immune cells and inducing a pro-inflammatory response, which helps to eliminate tumor cells and boost the host's immune response against cancer.

Bladder cancer remains a prevalent health concern in the United States, with NMIBC accounting for about 80% of cases. Annually, approximately 65,000 individuals are diagnosed with NMIBC, which affects the bladder's inner lining but does not infiltrate the muscle layer.

Protara Therapeutics continues to focus on bringing innovative treatments to market for challenging conditions such as NMIBC. Alongside TARA-002, the company is also developing IV Choline Chloride, aiming to support patients who have specific nutritional needs not met through traditional dietary intake.