NEW YORK - Protara Therapeutics, Inc. (NASDAQ:TARA), a clinical-stage biotechnology firm, has announced its plan to initiate a registrational trial for IV Choline Chloride, targeting parenteral nutrition (PN) patients, slated for the first half of 2025. This follows a strategic shift after discussions with the U.S. Food and Drug Administration (FDA), expanding the drug's potential use to a broader patient population.
The company, which focuses on therapies for cancer and rare diseases, previously sought an indication for intestinal failure-associated liver disease (IFALD). However, the new development pathway aims at PN patients who cannot synthesize choline due to the lack of oral or enteral nutrition sources.
The FDA has acknowledged the absence of an IV choline formulation for PN patients as a significant unmet medical need and has granted Orphan Drug Designation for the prevention of choline deficiency in this group.
PN-dependent patients often suffer from choline deficiency, which is linked to liver damage and can progress to hepatic failure. Approximately 80% of the 40,000 PN patients in the U.S. are estimated to be choline deficient. The American Society for Parenteral and Enteral Nutrition (ASPEN) and the European Society for Clinical Nutrition and Metabolism (ESPEN) both recommend choline supplementation for these patients.
Protara's upcoming THRIVE-3 study will be a seamless Phase 2b/3 trial designed to evaluate the safety and efficacy of IV Choline Chloride. The study will consist of a double-blinded, randomized, placebo-controlled format, with the primary endpoint being the restoration of choline levels in PN patients.
The Phase 2b segment will establish the pharmacokinetics profile of the drug across 24 patients, while the Phase 3 segment will involve around 100 patients, assessing the drug's impact on liver function among other secondary endpoints.
IV Choline Chloride has shown promise in previous studies by increasing plasma choline concentrations and improving steatosis, a marker of liver injury, in PN patients. This investigational therapy could potentially become the first FDA-approved IV choline treatment for PN patients.
This announcement is based on a press release statement from Protara Therapeutics, which is also developing TARA-002 for non-muscle invasive bladder cancer and lymphatic malformations. The company holds a U.S. patent for a choline composition that expires in 2041.
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