Protara Therapeutics Announces Results from THRIVE-1, a Prospective Observational Study Evaluating the Prevalence of Choline Deficiency and Liver Injury in Patients Dependent on Parenteral Support
- 78% of patients dependent on parenteral support were choline deficient, with 63% of these patients demonstrating liver dysfunction, including steatosis, cholestasis, and hepatobiliary injury
- IV Choline Chloride could fill a significant unmet medical need for this population
- Company expects to dose first patient in pivotal PK trial for IV Choline Chloride in Q1 2025
NEW YORK, Sept. 09, 2024 (GLOBE NEWSWIRE) -- Protara Therapeutics, Inc. (NASDAQ:TARA), a clinical-stage company developing transformative therapies for the treatment of cancer and rare diseases, today announced the results of THRIVE-1, a prospective, observational study evaluating the prevalence of choline deficiency and liver injury in patients dependent on parenteral support (PS). Results from the study were featured during a poster session at the 46th European Society for Clinical Nutrition and Metabolism Congress (ESPEN), from September 7, 2024, to September 10, 2024, in Milan.
The study found 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 intravenous (IV) choline supplementation in this patient population. Protara is developing 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.
"Findings from this study reinforce that there are a significant number of people dependent on PS who may potentially benefit from treatment with IV Choline Chloride," said Palle Bekker Jeppesen M.D., Ph.D., Clinical Professor, Department of Intestinal Failure and Liver Diseases, Rigshospitalet, Copenhagen University Hospital, Copenhagen, Denmark. "Choline is essential for PS dependent patients, as deficiency can lead to hepatic injury, neuropsychological impairment, muscle damage, and thrombotic abnormalities. 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."
"Currently, there are no approved IV choline products for patients dependent on PS globally, despite top professional medical societies in both the U.S. and Europe recommending choline treatment for these patients," 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 trial in the first quarter of 2025."
THRIVE-1 Study and Results
THRIVE-1 was a multi-center, cross-sectional prospective, observational study involving 78 patients to assess the prevalence of choline deficiency and liver injury in adolescents and adult patients with intestinal failure who are dependent on PS.
78% (61/78) were choline deficient, with 63% (38/60) of these patients demonstrating liver dysfunction including steatosis, cholestasis, and signs of hepatobiliary injury.
IV choline is recommended for patients receiving PS by the American Society for Parenteral and Enteral Nutrition (ASPEN) Recommendations for Changes in Commercially Available Parenteral Multivitamin and Multi–Trace Element Products, as well as by ESPEN in its Guideline on Home Parenteral Nutrition.
Based on feedback from the U.S. Food and Drug Administration, Protara intends to assess the safety and efficacy of IV Choline Chloride in THRIVE-3, a seamless registrational Phase 2b/3 trial with dose confirmation followed by a double-blinded, randomized, placebo-controlled trial in adolescents and adults receiving parenteral support.
About IV Choline Chloride
IV Choline Chloride is an investigational, intravenous phospholipid substrate replacement therapy initially in development for patients receiving parenteral support (PS). Choline is a known important substrate for phospholipids that are critical for healthy liver function and also plays 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 80 percent of patients dependent on PS are choline-deficient and have some degree of liver damage, which can lead to hepatic failure. In the U.S. alone, there are approximately 40,000 patients on long-term parenteral support who could benefit from an IV formulation of choline. IV Choline Chloride has the potential to become the first U.S. Food and Drug Administration (FDA) approved IV choline formulation for PS patients. IV Choline Chloride has been granted Orphan Drug Designation by the FDA for the prevention of choline deficiency in PS patients. The Company was issued a U.S. patent claiming a choline composition 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, 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 support who are otherwise unable to meet their choline needs via oral or enteral routes. For more information, visit www.protaratx.com.
Forward-Looking Statements
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Company Contact:
Justine O'Malley
Protara Therapeutics
[email protected]
646-817-2836
Source: Protara Therapeutics
