NEW YORK - A recent study by Protara Therapeutics, Inc. (NASDAQ:TARA) has highlighted a significant prevalence of choline deficiency among patients dependent on parenteral support (PS), with potential liver dysfunction implications. The findings, presented at the European Society for Clinical Nutrition and Metabolism Congress in Milan, showed that 78% of PS-dependent patients were choline deficient, and 63% of these individuals exhibited liver dysfunction, including conditions such as steatosis, cholestasis, and hepatobiliary injury.
The study, known as THRIVE-1, was a prospective, observational study involving 78 patients. Its results underscore the need for intravenous (IV) choline supplementation for patients who cannot meet their choline needs through oral or enteral nutrition. Protara is developing IV Choline Chloride, a phospholipid substrate replacement therapy, to address this unmet medical need. The company plans to begin a pivotal pharmacokinetic (PK) trial for IV Choline Chloride in the first quarter of 2025.
Choline is crucial for maintaining healthy liver function and is involved in various bodily processes, including brain development and muscle function. PS patients are often unable to synthesize choline adequately, leading to deficiency and potential liver damage, which can progress to hepatic failure. Currently, no approved IV choline products exist for PS patients, despite recommendations from medical societies in the U.S. and Europe.
Protara's IV Choline Chloride has received Orphan Drug Designation from the FDA for the prevention of choline deficiency in PS patients. The company also holds a U.S. patent for a choline composition with a term expiring in 2041.
This research is part of Protara's broader mission to develop transformative therapies for cancer and rare diseases. Alongside IV Choline Chloride, Protara is also working on TARA-002, an investigational cell-based therapy for the treatment of bladder cancer and lymphatic malformations.
The information presented in this article is based on a press release statement from Protara Therapeutics.
In other recent news, Protara Therapeutics has been making strides in the biopharmaceutical landscape, particularly with its TARA-002 investigational therapy. The therapy has demonstrated encouraging results in early-stage trials for patients with non-muscle invasive bladder cancer (NMIBC) who have not responded to or previously experienced Bacillus Calmette-Guerin (BCG) treatment. TARA-002 has successfully completed its Phase Ib ADVANCED-1EXP and Phase Ib/II ADVANCED-2 trials, achieving proof of concept efficacy with 3-month results.
These trials have shown promise in treating NMIBC patients, a demographic with significant unmet medical needs, especially for those seeking alternatives to cystectomy, a surgical procedure to remove the bladder. The safety profile of TARA-002 has also been reported as favorable in clinical trials, adding to the optimism surrounding the therapy's potential.
TD Cowen, an analyst firm, has maintained a "Buy" rating on Protara Therapeutics, signaling confidence in the company's strategic direction and the therapeutic promise of its clinical pipeline. The firm's positive stance comes as Protara continues to show clinical progress, especially with TARA-002. These are recent developments that investors should keep an eye on as Protara continues to develop its portfolio and impact the treatment landscape for NMIBC.
InvestingPro Insights
In light of Protara Therapeutics' (NASDAQ:TARA) recent study and its ongoing efforts to address choline deficiency in patients, investors may be interested in the company's financial health and market performance. The company's market capitalization stands at $38.27 million, indicating a relatively small-cap status which often comes with higher volatility in stock price movements. Indeed, Protara's stock has experienced significant volatility, with a six-month price total return of -51.18%, reflecting the challenges and uncertainties inherent in the biotech industry.
From an operational standpoint, Protara holds more cash than debt on its balance sheet, which is a positive sign for financial stability. However, the company is not currently profitable, with a negative P/E ratio of -0.65, and analysts do not anticipate profitability this year. Protara has also been quickly burning through cash, which is a critical factor for investors to consider given the company's stage of development and the capital-intensive nature of the biopharmaceutical industry. Furthermore, the InvestingPro Tips reveal that Protara suffers from weak gross profit margins and the valuation implies a poor free cash flow yield.
For those seeking additional insights, there are 10 more InvestingPro Tips available for Protara Therapeutics, which can provide a deeper analysis of the company's financial health and market potential. These tips could be particularly valuable for investors looking to understand the risks and opportunities associated with Protara as it advances its clinical programs and strives to bring new therapies to market.
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