Protara reveals choline deficiency in parenteral support patients

NEW YORK - Clinical-stage company Protara Therapeutics, Inc. (NASDAQ: TARA) has announced significant findings from its THRIVE-1 study, which will be presented at the upcoming American Society for Parenteral and Enteral Nutrition (ASPEN) 2025 Nutrition Science & Practice Conference in Columbus, Ohio. The study highlights a prevalent choline deficiency and associated liver injury in patients dependent on parenteral support (PS).

Choline, a crucial nutrient for liver function, is lacking in current PS formulations. The study found that 78% of PS-dependent patients were choline deficient, with 63% of these individuals experiencing liver dysfunction, including steatosis, cholestasis, and hepatobiliary injury.

Protara is developing intravenous (IV) Choline Chloride, a phospholipid substrate replacement therapy, to address this unmet need for a choline source in PS patients. The company plans to initiate its THRIVE-3 registrational Phase 3 clinical trial in the first half of 2025, aiming to assess the efficacy and safety of IV Choline Chloride in adolescents and adults on long-term PS. InvestingPro analysis reveals strong analyst confidence in the company’s potential, with price targets ranging from $22 to $30 per share. Subscribers can access 8 additional exclusive ProTips and comprehensive financial metrics to better understand the company’s growth trajectory.

IV Choline Chloride has been recognized by both ASPEN and the European Society for Clinical Nutrition and Metabolism (ESPEN) as a recommended nutrient for patients receiving PS. It has received Fast Track designation by the U.S. Food and Drug Administration (FDA) and could potentially become the first FDA-approved IV choline formulation for PS patients.

The need for choline supplementation in the PS community is underscored by the high prevalence of liver disease among these patients. Protara’s CEO, Jesse Shefferman, expressed the company’s commitment to advancing IV Choline Chloride to benefit those who cannot obtain choline through oral or enteral means.

The THRIVE-1 study’s findings will be detailed in an oral presentation titled "THRIVE-1: A Multi-Center, Cross-Sectional, Observational Study to Assess the Prevalence of Choline Deficiency in Patients Dependent on Parenteral Support" on Sunday, March 23, 2025.

This news is based on a press release statement from Protara Therapeutics, which continues to focus on transformative therapies for cancer and rare diseases, including its lead candidate, TARA-002, for bladder cancer and lymphatic malformations. The company’s next earnings report is scheduled for May 1, 2025, with InvestingPro data showing the stock has demonstrated strong momentum with a 120% price return over the past six months, despite analysts not anticipating profitability this year.

In other recent news, Protara Therapeutics reported a narrower-than-expected loss for the fourth quarter of 2024, with an adjusted loss of $0.48 per share, surpassing analyst estimates of a $0.59 loss per share. The company did not report any revenue for the quarter, as it continues in the clinical development phase for its drug candidates. Research and development expenses rose to $9.5 million, up from $6.4 million the previous year, due to increased clinical trial activities for TARA-002 and IV Choline Chloride. Protara ended the year with a robust cash position of $170.3 million, supported by a $100 million public offering in December, which is expected to fund operations into 2027.

Additionally, H.C. Wainwright maintained its Buy rating on Protara with a $23 price target, highlighting the potential of TARA-002 in treating high-risk non-muscle invasive bladder cancer (HR-NMIBC). The Phase II ADVANCED-2 trial showed promising results, with an 80% complete response rate at any time and a 100% rate at six months for certain patients. Cantor Fitzgerald also initiated coverage with an Overweight rating, pointing to the significant potential of Protara’s IV Choline, which could reach $400-500 million in peak sales. This assessment reflects confidence in the company’s ability to deliver value through its late-stage pipeline assets. Protara plans to release further data updates on its key products, including the 12-month data from the Phase 2 ADVANCED-2 trial by mid-2025.

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