- New development pathway significantly expands patient population for IV choline chloride
- IV choline chloride may become the first FDA-approved choline IV formulation for the 40,000 PN patients in the United States.
- Approximately 80% of PN patients have choline deficiency, which can lead to liver damage and liver failure. ASPEN recommends choline supplementation for PN patients
- The company plans to begin a registration study in the first half of 2025 to support FDA approval of IV choline chloride for PN patients
NEW YORK, April 5, 2024 (GLOBE NEWSWIRE) — Protara Therapeutics, Inc. (Nasdaq: TARA), a clinical-stage company developing innovative therapies for the treatment of cancer and rare diseases, today announced that it has filed an application for registration with the U.S. Food and Drug Administration for intravenous (IV) therapy. announced that it had reached an agreement with the FDA. Choline chloride, a phospholipid substrate alternative under investigation. The company had previously pursued indications for intestinal failure-associated liver disease (IFALD), but following feedback from the FDA, patients receiving oral nutrition (PN) or an inability to synthesize choline orally or orally We plan to pursue broader indications for sexually transmitted parenteral nutrition (PN) patients. Source of enteral nutrition. The company plans to advance the development of his IV choline chloride as a choline source for adult and adolescent patients with long-term PN. The FDA has granted IV choline chloride orphan drug designation for the prevention of choline deficiency in patients with PN.
“Currently, there are no IV formulations of choline available or in development for patients with PN. They have helped us define an efficient regulatory path to provide a much-needed source of IV choline,” said Jesse Shefferman, Chief Executive Officer of Protara Therapeutics. “We look forward to advancing the clinical development of IV choline chloride, which we believe has the potential to be the first FDA-approved IV choline therapy for patients dependent on PN. As such, we remain diligent in the development of our lead product candidate, TARA-002, for patients with non-muscle invasive bladder cancer and lymphatic malformations.”
“About 80 percent of PN-dependent patients have choline deficiency and some degree of liver damage, which can be reversed with effective supplements,” says Palle Becker Jeppesen, MD, Clinical Professor and Head of the Department of Enterology. said. Failure and Liver Disease, Rigshospitalet, Copenhagen University Hospital, Copenhagen, Denmark. “Top professional societies in the United States and Europe recognize the effects of long-term choline deficiency on liver health, particularly the development of liver disease with progressive steatosis, fibrosis, and ultimately end-stage cirrhosis. , recommending treatment with choline. Access to IV preparations of choline could have a meaningful impact on patients with intestinal failure for whom oral or enteral choline supplementation is not an option.”
Choline is recommended for patients with PN by the American Society for Parenteral and Enteral Nutrition (ASPEN) Recommendations for Modification of Commercial Parenteral Multivitamin and Multitrace Element Products and by the European Society of Clinical Nutrition and Metabolism (ESPEN). Guidelines for home parenteral nutrition.
The FDA indicated that a single study with the endpoint of restoring choline levels in PN patients could form the basis of a regulatory filing for IV choline chloride. Based on this feedback, the Company announced that the planned seamless Phase 2b/3, double-blind, randomized, placebo-controlled THRIVE-3 study in adolescents and adults receiving oral long-term PN. , will evaluate the safety and efficacy of IV choline chloride. or enteral feeding is not possible, inadequate, or contraindicated. The Phase 2b portion of this study will seek to establish the pharmacokinetic (PK) profile of IV choline chloride and will enroll approximately 24 patients and will He will be given one dose every 24 weeks. In the randomized, double-blind Phase 3 portion of the study, approximately 100 patients will receive either a high dose of IV choline chloride, a low dose of choline chloride, or a placebo for 24 weeks. The primary endpoint of this part of the study aims to prove that IV choline chloride is a durable source of choline. Secondary endpoints will assess the impact of choline supplementation on liver function. All patients are eligible to participate in the open-label extension. We plan to launch this study in the first half of 2025.
Previous studies have shown that treatment with IV choline chloride successfully increases plasma choline concentrations in PN patients and also improves steatosis, an important marker of liver damage.
About IV Choline Chloride
IV choline chloride is an intravenous (IV) phospholipid substrate replacement therapy originally developed for patients receiving parenteral nutrition (PN). Choline is known as an important substrate for phospholipids that are essential for healthy liver function and also plays an important role in regulating gene expression, cell membrane signaling, brain development and neurotransmission, muscle function, and bone health. I am fulfilling my role. PN patients are unable to synthesize choline from enteral nutritional sources, and there are currently no available PN formulations that contain choline. Approximately 80 percent of PN-dependent patients have choline deficiency and some degree of liver damage that can lead to liver failure. Currently, there are no PN formulations available that contain choline. In the United States alone, there are approximately 40,000 patients undergoing long-term parenteral nutrition therapy who benefit from his IV formulation of Choline. IV choline chloride may be the first FDA-approved IV choline formulation for PN patients. IV choline chloride has been granted orphan drug designation by the FDA for the prevention of choline deficiency in patients with PN. The company has been issued a U.S. patent claiming the choline composition, which expires in 2041.
About Protara Therapeutics
Protara is a clinical-stage biotechnology company dedicated to advancing innovative treatments for people with cancer and rare diseases. Protara’s portfolio includes lead candidate TARA-002, an investigational cell-based therapy in development for the treatment of non-muscle invasive bladder cancer (NMIBC) and lymphatic malformations (LM); It contains. The company is conducting an ongoing Phase 2 study in patients with carcinoma in situ (CIS) NMIBC unresponsive or previously untreated with Bacillus Calmette-Guérin (BCG), and a Phase 2 study in pediatric patients with: TARA-002 is being evaluated in a phase 2 study. LMs. In addition, Protara is developing IV choline chloride, an investigational phospholipid substrate replacement for patients receiving parenteral nutritional therapy whose choline requirements cannot be met by oral or enteral routes. . For more information, please visit www.protaratx.com.
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Company contact:
Justin O’Malley
Protara Therapeutics
Justine.OMalley@protaratx.com
646-817-2836
