Protara Therapeutics Granted FDA Fast Track Designation for Intravenous Choline Chloride for Patients Receiving Parenteral Support
Protara Therapeutics (Nasdaq: TARA) has received Fast Track designation from the FDA for its Intravenous (IV) Choline Chloride therapy. This investigational treatment is designed for adult and adolescent patients on parenteral support (PS) who cannot receive sufficient nutrition orally or enterally. The designation highlights the urgent need for such treatment, as approximately 80% of PS-dependent patients experience choline deficiency, which can lead to severe health complications.
Protara plans to initiate the registrational THRIVE-3 clinical trial in Q1 2025. This seamless Phase 2b/3 trial will assess the safety and efficacy of IV Choline Chloride in a double-blinded, randomized, placebo-controlled study. The primary endpoint will measure the change in plasma choline concentration from baseline compared to placebo. Currently, there are no FDA-approved IV choline products for PS-dependent patients globally, despite recommendations from major nutrition societies.
Protara Therapeutics (Nasdaq: TARA) ha ricevuto la designazione Fast Track dalla FDA per la sua terapia a base di Cloruro di Colina Intravenoso (IV). Questo trattamento sperimentale è stato progettato per pazienti adulti e adolescenti che necessitano di supporto parenterale (PS) e non possono ricevere un'alimentazione sufficiente per via orale o enterale. La designazione evidenzia l'urgenza di tale trattamento, considerando che circa l'80% dei pazienti dipendenti dal PS presenta carenza di colina, che può portare a gravi complicazioni sanitarie.
Protara prevede di avviare il trial clinico registrativo THRIVE-3 nel primo trimestre del 2025. Questo studio di fase 2b/3 senza soluzione di continuità valuterà la sicurezza e l'efficacia del Cloruro di Colina IV in uno studio randomizzato, controllato con placebo e in doppio cieco. L'obiettivo principale sarà misurare il cambiamento nella concentrazione plasmatiche di colina rispetto al basale rispetto al placebo. Attualmente, non ci sono prodotti di colina IV approvati dalla FDA per pazienti dipendenti dal PS a livello globale, nonostante le raccomandazioni delle principali società di nutrizione.
Protara Therapeutics (Nasdaq: TARA) ha recibido la designación Fast Track de la FDA para su terapia de Cloruro de Colina Intravenoso (IV). Este tratamiento en investigación está diseñado para pacientes adultos y adolescentes en soporte parenteral (PS) que no pueden recibir suficiente nutrición por vía oral o enteral. La designación destaca la urgente necesidad de tal tratamiento, ya que aproximadamente el 80% de los pacientes dependientes de PS experimentan deficiencia de colina, lo que puede conducir a graves complicaciones de salud.
Protara planea iniciar el ensayo clínico registrativo THRIVE-3 en el primer trimestre de 2025. Este ensayo de Fase 2b/3 sin fisuras evaluará la seguridad y eficacia del Cloruro de Colina IV en un estudio aleatorizado, controlado con placebo y doble ciego. El objetivo principal medirá el cambio en la concentración plasmática de colina desde el inicio en comparación con el placebo. Actualmente, no hay productos de colina IV aprobados por la FDA para pacientes dependientes de PS a nivel mundial, a pesar de las recomendaciones de las principales sociedades de nutrición.
Protara Therapeutics (Nasdaq: TARA)는 FDA로부터 패스트 트랙 지정을 받았습니다. 이는 정맥용 콜린 염화물(IV) 치료법에 대한 것입니다. 이 실험적 치료는 경장 영양을 받을 수 없는 성인 및 청소년 환자들을 위해 설계되었습니다. 이 지정은 PS 의존 환자의 약 80%가 콜린 결핍을 경험하고 있으며, 이는 심각한 건강 문제로 이어질 수 있다는 긴급한 필요성을 강조합니다.
Protara는 2025년 1분기에 등록 임상 시험 THRIVE-3을 시작할 계획입니다. 이 연속적인 2b/3 단계 임상 시험은 이중 맹검 및 무작위 배정을 통해 IV 콜린 염화물의 안전성과 효능을 평가할 것입니다. 주요 목표는 기준선과 비교하여 플라시보와의 혈장 콜린 농도 변화를 측정하는 것입니다. 현재 PS 의존 환자를 위한 FDA 승인 IV 콜린 제품이 전 세계적으로 없으며, 이는 주요 영양학회에서 권장하고 있습니다.
Protara Therapeutics (Nasdaq: TARA) a reçu la désignation Fast Track de la FDA pour sa thérapie à base de chlorure de choline intraveineux (IV). Ce traitement expérimental est conçu pour les patients adultes et adolescents nécessitant un soutien parenteral (PS) qui ne peuvent pas recevoir une nutrition suffisante par voie orale ou entérale. Cette désignation met en évidence le besoin urgent de ce type de traitement, car environ 80 % des patients dépendants du PS souffrent d'une carence en choline, ce qui peut entraîner de graves complications de santé.
Protara prévoit de lancer l'essai clinique THRIVE-3 au premier trimestre 2025. Cet essai de phase 2b/3 sans interruption évaluera la sécurité et l'efficacité du chlorure de choline IV dans le cadre d'une étude randomisée, contrôlée par placebo et en double aveugle. L'objectif principal mesurera le changement de concentration plasmatique de choline par rapport à la ligne de base en comparaison au placebo. Actuellement, il n'existe pas de produits de choline IV approuvés par la FDA pour les patients dépendants du PS dans le monde, malgré les recommandations des principales sociétés de nutrition.
Protara Therapeutics (Nasdaq: TARA) hat von der FDA die Fast Track-Bezeichnung für seine intravenöse Cholinchlorid-Therapie (IV) erhalten. Diese experimentelle Behandlung zielt auf erwachsene und jugendliche Patienten ab, die auf parenterale Unterstützung (PS) angewiesen sind und nicht ausreichend Nahrung oral oder enteral aufnehmen können. Die Bezeichnung unterstreicht den dringenden Bedarf an solch einer Behandlung, da etwa 80 % der PS-abhängigen Patienten an Cholinmangel leiden, was zu schwerwiegenden Gesundheitskomplikationen führen kann.
Protara plant, im ersten Quartal 2025 die registrierende THRIVE-3-Studie zu starten. Diese nahtlose Phase 2b/3-Studie wird die Sicherheit und Wirksamkeit von IV Cholinchlorid in einer doppelblinden, randomisierten, placebokontrollierten Studie bewerten. Der primäre Endpunkt wird die Veränderung der Plasmakonzentration von Cholin im Vergleich zur Ausgangswerte und zum Placebo messen. Derzeit gibt es weltweit keine von der FDA genehmigten IV-Cholinprodukte für PS-abhängige Patienten, trotz der Empfehlungen führender Ernährungsgesellschaften.
- FDA granted Fast Track designation for IV Choline Chloride
- Potential to be the first FDA-approved IV choline therapy for PS-dependent patients
- Addressing an unmet medical need for approximately 40,000 long-term PS patients in the U.S.
- On track to initiate registrational THRIVE-3 trial in Q1 2025
- Registrational trial not starting until Q1 2025, indicating a long timeline to potential approval
Insights
The FDA's Fast Track designation for Protara Therapeutics' Intravenous Choline Chloride is a significant development. This designation highlights the unmet medical need for patients on parenteral support (PS) who cannot receive adequate nutrition orally or enterally. With approximately 40,000 patients on long-term PS in the U.S. and 80% experiencing choline deficiency, this therapy addresses a critical gap in treatment options.
The potential impact on patient outcomes is substantial. Choline deficiency can lead to severe complications, including hepatic injury and neuropsychological impairment. As the first FDA-approved IV choline therapy for PS-dependent patients, if successful, this could significantly improve patient care and reduce long-term health risks.
The planned THRIVE-3 trial, set to begin in Q1 2025, will be important for assessing the safety and efficacy of IV Choline Chloride. The seamless Phase 2b/3 design could potentially accelerate the development timeline. Investors should monitor the trial's progress closely, as positive results could lead to rapid market entry and potential revenue growth for Protara Therapeutics.
The Fast Track designation for Protara Therapeutics' IV Choline Chloride is a positive catalyst for the company's stock. This designation can lead to several benefits, including:
- More frequent meetings with the FDA
- Eligibility for Accelerated Approval and Priority Review
- Rolling Review of the New Drug Application
These advantages could potentially reduce the time to market and associated costs, which is important for a company with a
Investors should note that the THRIVE-3 trial is not set to begin until Q1 2025, indicating a long runway to potential commercialization. This timeline suggests that near-term revenue impact will be and the company may require additional funding to reach key milestones. However, the Fast Track status and the unmet medical need could attract partnership opportunities or increase the company's appeal as an acquisition target.
- Remain on track to initiate registrational THRIVE-3 trial in 1Q’ 2025
NEW YORK, Oct. 21, 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 that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to Intravenous (IV) Choline Chloride, the Company’s investigational IV phospholipid substrate replacement therapy, as a source of choline for adult and adolescent patients on parenteral support (PS) for whom oral or enteral nutrition is not possible, insufficient, or contraindicated. In the U.S. alone, there are approximately 40,000 patients on long-term parenteral support.
“Receipt of Fast Track designation underscores the urgent need in these patients and our belief that IV Choline Chloride has the potential to serve as the first FDA-approved IV choline therapy for patients dependent on PS,” said Jesse Shefferman, Chief Executive Officer of Protara Therapeutics. “Approximately
IV choline is recommended for patients receiving PS by the American Society for Parenteral and Enteral Nutrition 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 Congress in its Guideline on Home Parenteral Nutrition.
Based on feedback from the FDA, 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. The primary endpoint of the trial is the change in plasma choline concentration from baseline compared to placebo.
About FDA Fast Track Designation
The FDA’s Fast Track program facilitates the development and expedites the review of drugs that treat serious conditions and have the potential to address an unmet medical need. Programs with Fast Track designation may benefit from early and frequent interactions with the FDA over the course of drug development. In addition, the Fast Track designation program allows for the eligibility for accelerated approval and priority review if relevant criteria are met and enables a company to submit individual sections of a New Drug Application for review on a rolling-submission basis.
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
Protara recently presented results from THRIVE-1, a prospective, observational study evaluating the prevalence of choline deficiency and liver injury in patients dependent on PS, which found that
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 patients with NMIBC 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 through oral or enteral routes. For more information, visit www.protaratx.com.
Forward-Looking Statements
Statements contained in this press release regarding matters that are not historical facts are "forward looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Protara may, in some cases, use terms such as “predicts,” “believes,” “potential,” “proposed,” “continue,” “designed,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should” or other words or expressions referencing future events, conditions or circumstances that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such forward-looking statements include but are not limited to, statements regarding Protara’s intentions, beliefs, projections, outlook, analyses or current expectations concerning, among other things: Protara’s business strategy, including its development plans for its product candidates and plans regarding the timing or outcome of existing or future clinical trials; statements related to expectations regarding interactions with the FDA; Protara’s financial position; statements regarding the anticipated safety or efficacy of Protara’s product candidates; and Protara’s outlook for the remainder of the year. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Factors that contribute to the uncertain nature of the forward-looking statements include: risks that Protara’s financial guidance may not be as expected, as well as risks and uncertainties associated with: Protara’s development programs, including the initiation and completion of non-clinical studies and clinical trials and the timing of required filings with the FDA and other regulatory agencies; general market conditions; changes in the competitive landscape; changes in Protara’s strategic and development and commercial plans; Protara’s ability to obtain sufficient financing to fund its strategic plans and commercialization efforts; having to use cash in ways or on timing other than expected; the impact of market volatility on cash reserves; failure to attract and retain management and key personnel; the impact of general U.S. and foreign, economic, industry, market, regulatory, political or public health conditions; and the risks and uncertainties associated with Protara’s business and financial condition in general, including the risks and uncertainties described more fully under the caption “Risk Factors” and elsewhere in Protara's filings and reports with the United States Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made and are based on management's assumptions and estimates as of such date. Protara undertakes no obligation to update any forward-looking statements, whether as a result of the receipt of new information, the occurrence of future events or otherwise, except as required by law.
Company Contact:
Justine O'Malley
Protara Therapeutics
Justine.OMalley@protaratx.com
646-817-2836
Source: Protara Therapeutics
FAQ
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