NEW YORK - Protara Therapeutics, Inc. (NASDAQ: TARA), a clinical-stage biotech company whose stock has surged over 150% in the past year according to InvestingPro data, today revealed encouraging results from its Phase 2 ADVANCED-2 trial of TARA-002, an investigational cell-based therapy for high-risk Non-Muscle Invasive Bladder Cancer (NMIBC) patients. The trial demonstrated a 72% complete response rate at six months and a 70% rate at any time across different Bacillus Calmette-Guérin (BCG) exposures.
The study, which included patients unresponsive or naïve to BCG—a common treatment for NMIBC—showed particularly strong outcomes in BCG-Unresponsive patients, with a 100% complete response rate at six months. BCG-Naïve patients exhibited a 64% response rate at six months. The therapy's safety profile was also favorable, with no treatment-related adverse events above Grade 1 reported and no discontinuations due to adverse events.
These results will be presented at the 25th Annual Meeting of the Society of Urologic Oncology in Dallas, Texas. The data cutoff for the trial was November 19, 2024, and included 20 patients at three months, 18 patients at six months, and three patients at nine months. With analyst price targets ranging from $20 to $50, significantly above the current trading price of $3.54, InvestingPro subscribers can access 13 additional key insights about Protara's financial health and market position. The trial's pivotal cohort aims to be registrational in alignment with the FDA's guidance for BCG-Unresponsive NMIBC.
Dr. Brian Mazzarella, Vice President of Research for Urology America and an investigator in the ADVANCED-2 study, commented on the therapy's meaningful activity and its potential to become a new treatment option due to its ease of use and low procedural burden for physicians.
Jesse Shefferman, CEO of Protara Therapeutics, expressed optimism about the six-month data and the therapy's potential in NMIBC. He indicated that these positive results, along with the expansion of international trial sites, could accelerate patient enrollment, with initial data from 12-month evaluable patients expected in mid-2025.
TARA-002 is being developed based on a master cell bank of genetically distinct group A Streptococcus pyogenes, similar to the immunopotentiator OK-432, marketed in Japan. The therapy works by activating immune cells to produce a pro-inflammatory response and directly killing tumor cells to enhance the antitumor immune response. While the company maintains a strong financial position with more cash than debt and a healthy current ratio of 9.85, InvestingPro analysis indicates the company is currently burning through cash rapidly, a common characteristic of clinical-stage biotech companies.
The trial's results are based on a press release statement from Protara Therapeutics. The company will host a conference call and webcast to discuss the data today at 8:30 am ET. The webcast will be available on the company's website for a limited time after the presentation. Protara is committed to developing therapies for cancer and rare diseases, with TARA-002 as its lead candidate for NMIBC and lymphatic malformations.
In other recent news, Protara Therapeutics has been making significant strides in the biopharmaceutical landscape. The U.S. Food and Drug Administration (FDA) has granted Fast Track designation to Protara's investigational therapy, Intravenous (IV) Choline Chloride, aimed at treating patients requiring parenteral support. The company is set to advance this therapy into a pivotal study in the first quarter of 2025.
According to Protara, around 80% of patients on parenteral support suffer from choline deficiency, underscoring the urgent need for treatment options. The company's TARA-002 has also shown promising results in early-stage trials for patients with non-muscle invasive bladder cancer.
In addition, Protara has reported positive results from its LM registrational trial, where two out of three patients showed a complete response after a single dose of TARA-002. These developments have led analyst firms Oppenheimer and TD Cowen to maintain an Outperform and Buy rating on Protara shares, respectively. These are recent developments in Protara's ongoing efforts to address unmet medical needs through innovative therapies.
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