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
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.
Protara Therapeutics (Nasdaq: TARA) ha annunciato una presentazione straordinaria dei risultati del loro studio THRIVE-1 alla Conferenza sulla Scienza e Pratica della Nutrizione ASPEN 2025. Lo studio ha rivelato che il 78% dei pazienti dipendenti dal supporto parenterale (PS era carente di colina, con il 63% di questi pazienti che mostrava disfunzione epatica.
L'azienda sta sviluppando Cloruro di Colina endovenoso (IV) come trattamento per i pazienti che non possono ricevere colina attraverso nutrizione orale o enterale. Il trattamento ha ricevuto la designazione Fast Track dalla FDA. Protara prevede di avviare THRIVE-3, uno studio registrativo di Fase 2b/3, nella prima metà del 2025, che includerà una fase di conferma della dose di 8 settimane con 24 pazienti, seguita da una fase randomizzata e controllata con placebo di 24 settimane con 100 pazienti.
Protara Therapeutics (Nasdaq: TARA) ha anunciado una presentación adicional de los resultados de su estudio THRIVE-1 en la Conferencia de Ciencia y Práctica de Nutrición ASPEN 2025. El estudio reveló que el 78% de los pacientes dependientes del soporte parenteral (PS) eran deficientes en colina, con el 63% de estos pacientes mostrando disfunción hepática.
La empresa está desarrollando Cloruro de Colina intravenoso (IV) como tratamiento para pacientes que no pueden recibir colina a través de nutrición oral o enteral. El tratamiento ha recibido la designación de Vía Rápida por parte de la FDA. Protara planea iniciar THRIVE-3, un ensayo registrativo de Fase 2b/3, en la primera mitad de 2025, que incluirá una fase de confirmación de dosis de 8 semanas con 24 pacientes, seguida de una fase aleatorizada y controlada con placebo de 24 semanas con 100 pacientes.
프로타라 테라퓨틱스 (Nasdaq: TARA)는 ASPEN 2025 영양 과학 및 실천 회의에서 THRIVE-1 연구 결과의 앙코르 발표를 발표했습니다. 연구에 따르면 부모 영양 지원(PS)에 의존하는 환자의 78%가 콜린 결핍이었다, 이들 환자 중 63%가 간 기능 장애를 보였다.
회사는 정맥 주사용 콜린 클로라이드 (IV)를 경구 또는 장내 영양을 통해 콜린을 받을 수 없는 환자 치료제로 개발하고 있습니다. 이 치료법은 FDA로부터 신속 심사 지정을 받았습니다. 프로타라는 2025년 상반기에 24명의 환자를 대상으로 8주 용량 확인 단계와 100명의 환자를 포함한 24주 무작위 위약 대조 단계가 포함된 THRIVE-3, 등록 Phase 2b/3 시험을 시작할 계획입니다.
Protara Therapeutics (Nasdaq: TARA) a annoncé une présentation supplémentaire des résultats de leur étude THRIVE-1 lors de la Conférence sur la Science et la Pratique de la Nutrition ASPEN 2025. L'étude a révélé que 78 % des patients dépendants du soutien parentéral (PS) étaient déficients en choline, avec 63 % de ces patients présentant une dysfonction hépatique.
La société développe Chlorure de Choline intraveineux (IV) comme traitement pour les patients qui ne peuvent pas recevoir de choline par voie orale ou entérale. Ce traitement a reçu la désignation Fast Track de la FDA. Protara prévoit de lancer THRIVE-3, un essai de Phase 2b/3 d'enregistrement, dans la première moitié de 2025, qui comprendra une phase de confirmation de dose de 8 semaines avec 24 patients, suivie d'une phase randomisée contrôlée par placebo de 24 semaines avec 100 patients.
Protara Therapeutics (Nasdaq: TARA) hat eine zusätzliche Präsentation der Ergebnisse ihrer THRIVE-1-Studie auf der ASPEN 2025 Konferenz für Ernährungswissenschaft und -praxis angekündigt. Die Studie ergab, dass 78% der auf parenterale Unterstützung (PS) angewiesenen Patienten an Cholinmangel litten, wobei 63% dieser Patienten eine Leberfunktionsstörung aufwiesen.
Das Unternehmen entwickelt intravenöses Cholinchlorid (IV) als Behandlung für Patienten, die Cholin nicht über orale oder enterale Ernährung erhalten können. Die Behandlung erhielt von der FDA die Fast-Track-Zulassung. Protara plant, in der ersten Hälfte des Jahres 2025 THRIVE-3, eine registrierende Phase 2b/3-Studie, zu starten, die eine 8-wöchige Dosisbestätigungsphase mit 24 Patienten und eine 24-wöchige randomisierte, placebo-kontrollierte Phase mit 100 Patienten umfasst.
- 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
- Phase 2b/3 trial results still pending
- No currently approved IV choline formulation available
- Long timeline to potential commercialization
Insights
Protara's announcement highlights important clinical progress for their IV Choline Chloride program targeting patients dependent on parenteral support (PS). The THRIVE-1 observational study revealed 78% of PS-dependent patients are choline deficient, with
The company is advancing to THRIVE-3, a registrational Phase 2b/3 trial scheduled to begin in 1H 2025. This represents a significant milestone as it moves their lead asset toward potential approval. The seamless trial design with an initial dose confirmation phase followed by a randomized controlled phase demonstrates a well-considered regulatory strategy.
The FDA's Fast Track designation for IV Choline Chloride acknowledges both the unmet medical need and the therapy's potential, potentially accelerating the regulatory pathway. Recommendations from both American (ASPEN) and European (ESPEN) nutrition societies further validate the medical necessity of IV choline supplementation for this patient population.
While the announcement represents positive progression of Protara's clinical program, investors should recognize that significant development milestones—including patient enrollment, interim analyses, and ultimate trial results—still lie ahead before potential commercialization. The clearly defined patient population with documented need provides a focused market opportunity, though the exact size of this opportunity remains to be quantified.
Protara's presentation of THRIVE-1 data represents incremental progress in their IV Choline Chloride development program. The data demonstrating
From a drug development perspective, the upcoming THRIVE-3 trial employs a pragmatic design. The seamless Phase 2b/3 approach with an initial dose confirmation component followed by a larger randomized controlled phase balances efficiency with regulatory rigor. This design potentially streamlines the development timeline while still generating the robust efficacy and safety data required for approval.
The therapeutic sits in an advantageous regulatory position with Fast Track designation, which can provide benefits including more frequent FDA interactions, potential for accelerated approval, and priority review. The endorsements from major clinical societies (ASPEN and ESPEN) further strengthen the clinical case and potential market adoption.
For a rare disease-focused company like Protara (market cap:
- 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
“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
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?
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What regulatory designation has TARA received for IV Choline Chloride?
What percentage of choline deficient patients in TARA's study showed liver dysfunction?
