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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.
• 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.
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