Protara Therapeutics Announces Encore Presentation of Results from THRIVE-1 Prospective Observational Study Evaluating the Prevalence of Choline Deficiency and Liver Injury in Patients Dependent on Parenteral Support

Rhea-AI Summary

Protara Therapeutics (Nasdaq: TARA) has announced an encore presentation of their THRIVE-1 study results at the ASPEN 2025 Nutrition Science & Practice Conference. The study revealed that 78% of patients dependent on parenteral support (PS) were choline deficient, with 63% of these patients showing liver dysfunction.

The company is developing intravenous (IV) Choline Chloride as a treatment for patients who cannot receive choline through oral or enteral nutrition. The treatment has received Fast Track designation from the FDA. Protara plans to initiate THRIVE-3, a registrational Phase 2b/3 trial, in the first half of 2025, which will include an 8-week dose confirmation phase with 24 patients, followed by a 24-week randomized, placebo-controlled phase with 100 patients.

Positive

• High market need demonstrated: 78% of PS patients are choline deficient
• FDA Fast Track designation received for IV Choline Chloride
• Clear development timeline with Phase 2b/3 trial starting in 1H 2025
• Treatment recommended by both ASPEN and ESPEN guidelines

Negative

• Phase 2b/3 trial results still pending
• No currently approved IV choline formulation available
• Long timeline to potential commercialization

• Data to be featured during oral presentation at the American Society for Parenteral and Enteral Nutrition 2025 Nutrition Science & Practice Conference
• Dosing of first patient in THRIVE-3 registrational trial of IV Choline Chloride in patients dependent on parenteral support expected in 1H 2025

NEW YORK, March 19, 2025 (GLOBE NEWSWIRE) -- Protara Therapeutics, Inc. (Nasdaq: TARA), a clinical-stage company developing transformative therapies for the treatment of cancer and rare diseases, today announced that data from THRIVE-1, a prospective, observational study evaluating the prevalence of choline deficiency and liver injury in patients dependent on parenteral support (PS), will be featured during an oral session at the American Society for Parenteral and Enteral Nutrition (ASPEN) 2025 Nutrition Science & Practice Conference, taking place March 22 through March 25, 2025 in Columbus, Ohio.

Choline is an important substrate for phospholipids that are critical for healthy liver function. Patients on PS are unable to synthesize choline from enteral nutrition sources, and there are currently no available PS formulations containing choline. Protara is developing intravenous (IV) Choline Chloride, an investigational phospholipid substrate replacement therapy, as a source of choline when oral or enteral nutrition is not possible, insufficient, or contraindicated.

THRIVE-1 was conducted to better understand the prevalence of choline deficiency and liver injury in patients on PS. Results demonstrated that 78% of patients who are dependent on PS were choline deficient, and that 63% of choline deficient participants had liver dysfunction, including steatosis, cholestasis, and hepatobiliary injury, underscoring the need for IV choline replacement therapy in this patient population.

“Findings from this study reinforce that there are a significant number of people dependent on PS who have evidence of liver disease and may potentially benefit from treatment with IV Choline Chloride,” said Dejan Micic M.D., Associate Professor and Division Chief of Gastroenterology and Nutrition at Loyola University Medical Center in Maywood, Illinois. “Choline is an essential micronutrient for patients dependent on PS, as deficiency can lead to liver injury. Access to an IV formulation of choline has the potential to meaningfully impact patients for whom oral or enteral choline supplementation is not an option.”

“Results from THRIVE-1 underscore the significant need for choline among patients on PS,” said Jesse Shefferman, Chief Executive Officer of Protara Therapeutics. “We remain committed to bringing the first approved IV formulation of choline to the PS community and look forward to initiating our registrational THRIVE-3 trial of IV Choline Chloride in the first half of 2025.”

The Company plans to initiate THRIVE-3, a registrational Phase 3 clinical trial, in the first half of 2025. THRIVE-3 is a seamless Phase 2b/3 trial with an 8-week, dose confirmation phase (n=24) followed by a 24-week double-blinded, randomized, placebo-controlled phase to assess the efficacy and safety of IV Choline Chloride in adolescents and adults on long-term PS (n=100). IV Choline Chloride was previously granted Fast Track designation by the U.S. Food and Drug Administration.

IV choline is recommended for patients receiving PS by ASPEN in its Recommendations for Changes in Commercially Available Parenteral Multivitamin and Multi–Trace Element Products, as well as by the European Society for Clinical Nutrition and Metabolism (ESPEN) in its Guideline on Home Parenteral Nutrition.

Details of the oral presentation are as follows:

• Abstract Title: THRIVE-1: A Multi-Center, Cross-Sectional, Observational Study to Assess the Prevalence of Choline Deficiency in Patients Dependent on Parenteral Support
• Session Title: Nutrition and Metabolism Research Paper Session: Parenteral Nutrition Therapy
• Session Date and Time: Sunday, March 23, 2025, 2:00 p.m. – 3:30 p.m. ET
  

About IV Choline Chloride

IV Choline Chloride is an investigational, intravenous phospholipid substrate replacement therapy in development for patients receiving parenteral support (PS). Choline is a known important substrate for phospholipids that are critical for healthy liver function that also play an important role in modulating gene expression, cell membrane signaling, brain development and neurotransmission, muscle function, and bone health. PS patients are unable to synthesize choline from enteral nutrition sources, and there are currently no available PS formulations containing choline. Approximately 78% of patients dependent on PS are choline-deficient and of those approximately 63% have some degree of liver dysfunction, which can lead to hepatic failure. Every year in the U.S. there are approximately 90,000 people who require PS at home and of those approximately 30,000 are on long-term PS. IV Choline Chloride has the potential to become the first U.S. Food and Drug Administration (FDA) approved IV choline formulation for PS patients. It has been granted Orphan Drug Designation by the FDA for the prevention and/or treatment of choline deficiency in patients on long-term PN and been granted Fast Track Designation as a source of choline when oral or enteral nutrition is not possible, insufficient, or contraindicated. The U.S. Patent and Trademark Office has issued us a U.S. patent claiming a choline composition and a U.S. patent claiming a method for treating choline deficiency with a choline composition, each with a term expiring in 2041.

About Protara Therapeutics, Inc.

Protara is a clinical-stage biotechnology company committed to advancing transformative therapies for people with cancer and rare diseases. Protara’s portfolio includes its lead candidate, TARA-002, an investigational cell-based therapy in development for the treatment of non-muscle invasive bladder cancer (NMIBC) and lymphatic malformations (LMs). The Company is evaluating TARA-002 in an ongoing Phase 2 trial in NMIBC patients with carcinoma in situ (CIS) who are unresponsive or naïve to treatment with Bacillus Calmette-Guérin (BCG), as well as a Phase 2 trial in pediatric patients with LMs. Additionally, Protara is developing IV Choline Chloride, an investigational phospholipid substrate replacement for patients on parenteral nutrition who are otherwise unable to meet their choline needs via oral or enteral routes. For more information, visit www.protaratx.com.

Company Contact:

Justine O'Malley
Protara Therapeutics
Justine.OMalley@protaratx.com
646-817-2836

FAQ

What percentage of patients showed choline deficiency in TARA's THRIVE-1 study?

78% of patients dependent on parenteral support were found to be choline deficient.

When will Protara Therapeutics (TARA) begin the THRIVE-3 clinical trial?

Protara plans to initiate the THRIVE-3 registrational trial in the first half of 2025.

How many patients will be enrolled in TARA's THRIVE-3 trial?

The trial will include 24 patients in the dose confirmation phase and 100 patients in the randomized phase.

What regulatory designation has TARA received for IV Choline Chloride?

IV Choline Chloride has received Fast Track designation from the U.S. FDA.

What percentage of choline deficient patients in TARA's study showed liver dysfunction?

63% of choline deficient participants demonstrated liver dysfunction.