Protara Therapeutics Announces Completion of First Cohort in Phase 2 STARBORN-1 Trial of TARA-002 in Pediatric Patients with Lymphatic Malformations
Protara Therapeutics (Nasdaq: TARA) has completed the first cohort of its Phase 2 STARBORN-1 trial for TARA-002, an investigational cell-based immunopotentiator for treating pediatric lymphatic malformations (LMs). The initial results are promising, with two out of three patients achieving complete responses after a single dose. The safety profile was consistent with historical data, showing mild to moderate, resolvable adverse events.
The trial aims to enroll about 30 patients aged 6 months to 18 years, with initial results from the next cohort expected in 1H'25. The primary endpoint is the proportion of participants demonstrating clinical success, defined as a substantial or complete reduction in LM volume. This development is significant as there are currently no FDA-approved treatments for LMs, a rare condition primarily affecting children.
Protara Therapeutics (Nasdaq: TARA) ha completato il primo gruppo del suo studio di fase 2 STARBORN-1 per TARA-002, un immunopotenziante cellulare in fase investigativa per il trattamento delle malformazioni linfatiche pediatriche (LM). I risultati iniziali sono promettenti, con due pazienti su tre che hanno ottenuto risposte complete dopo una singola dose. Il profilo di sicurezza è coerente con i dati storici, mostrando eventi avversi lievi a moderati, risolvibili.
Lo studio mira a arruolare circa 30 pazienti di età compresa tra 6 mesi e 18 anni, con i risultati iniziali del prossimo gruppo attesi nella prima metà del 2025. L'obiettivo primario è la proporzione di partecipanti che dimostrano successo clinico, definito come una riduzione sostanziale o completa del volume della LM. Questo sviluppo è significativo poiché attualmente non ci sono trattamenti approvati dalla FDA per le LM, una condizione rara che colpisce principalmente i bambini.
Protara Therapeutics (Nasdaq: TARA) ha completado la primera cohorte de su ensayo clínico de fase 2 STARBORN-1 para TARA-002, un inmunopotenciador celular en investigación para tratar malformaciones linfáticas pediátricas (LM). Los resultados iniciales son prometedores, con dos de tres pacientes logrando respuestas completas después de una sola dosis. El perfil de seguridad fue consistente con datos históricos, mostrando eventos adversos leves a moderados, resolubles.
El ensayo tiene como objetivo inscribir a aproximadamente 30 pacientes de entre 6 meses y 18 años, con resultados iniciales de la próxima cohorte esperados en la primera mitad de 2025. El objetivo primario es la proporción de participantes que demuestran éxito clínico, definido como una reducción sustancial o completa en el volumen de LM. Este desarrollo es significativo ya que actualmente no hay tratamientos aprobados por la FDA para las LM, una condición rara que afecta principalmente a los niños.
Protara Therapeutics (Nasdaq: TARA)는 소아 림프형성 이상(LM) 치료를 위한 연구 중인 세포 기반 면역 증강제 TARA-002의 2상 STARBORN-1 임상시험의 첫 번째 코호트를 완료했습니다. 초기 결과는 유망하며, 3명의 환자 중 2명이 단일 투여 후 완전한 반응을 보였습니다. 안전성 프로필은 역사적 데이터와 일치하며, 경미한에서 중간 정도의 해결 가능한 이상 반응을 보였습니다.
이 임상시험은 6개월에서 18세 사이의 약 30명의 환자를 등록할 계획이며, 다음 코호트의 초기 결과는 2025년 1분기에 예상됩니다. 주요 목표는 LM 볼륨의 실질적 또는 완전한 감소를 보여주는 참가자의 비율입니다. 현재 FDA에서 승인된 LM 치료제가 없기 때문에 이 개발은 의미가 큽니다. LM은 주로 어린이에게 영향을 미치는 드문 질환입니다.
Protara Therapeutics (Nasdaq: TARA) a terminé la première cohorte de son essai clinique de phase 2 STARBORN-1 pour TARA-002, un immunopotentiateur à base de cellules en cours d'investigation pour traiter les malformations lymphatiques pédiatriques (LM). Les résultats initiaux sont prometteurs, avec deux patients sur trois ayant obtenu des réponses complètes après une seule dose. Le profil de sécurité était cohérent avec les données historiques, montrant des effets indésirables légers à modérés, résolvables.
L'essai vise à recruter environ 30 patients âgés de 6 mois à 18 ans, avec des résultats initiaux de la prochaine cohorte attendus dans la première moitié de 2025. L'objectif principal est la proportion de participants montrant un succès clinique, défini comme une réduction substantielle ou complète du volume de LM. Ce développement est significatif car il n'existe actuellement aucun traitement approuvé par la FDA pour les LM, une condition rare touchant principalement les enfants.
Protara Therapeutics (Nasdaq: TARA) hat die erste Kohorte seiner Phase 2 STARBORN-1-Studie für TARA-002, einen investigativen zellbasierten Immunpotentiator zur Behandlung von pädiatrischen lymphatischen Malformationen (LM), abgeschlossen. Die ersten Ergebnisse sind vielversprechend, da zwei von drei Patienten nach einer einzigen Dosis vollständige Reaktionen zeigten. Das Sicherheitsprofil war konsistent mit historischen Daten und zeigte milde bis moderate, lösbare Nebenwirkungen.
Die Studie zielt darauf ab, etwa 30 Patienten im Alter von 6 Monaten bis 18 Jahren zu rekrutieren, wobei die ersten Ergebnisse der nächsten Kohorte in der ersten Hälfte von 2025 erwartet werden. Das primäre Ziel ist der Anteil der Teilnehmer, die klinischen Erfolg zeigen, definiert als eine wesentliche oder vollständige Reduktion des LM-Volumens. Diese Entwicklung ist von Bedeutung, da derzeit keine von der FDA zugelassenen Behandlungen für LMs existieren, eine seltene Erkrankung, die hauptsächlich Kinder betrifft.
- Two out of three patients in the first cohort achieved complete response after one dose of TARA-002
- Safety and tolerability profile consistent with historical experience
- Enrollment in additional cohorts underway with positive investigator feedback
- Strong patient interest with waiting lists for subsequent cohorts
- Initial results to only three patients in the first cohort
- Full trial results not expected until first half of 2025
Insights
The completion of the first cohort in Protara's Phase 2 STARBORN-1 trial for TARA-002 marks a significant milestone. The initial efficacy data is particularly noteworthy, with two out of three patients achieving complete response after just one dose. This suggests
The safety profile aligns with historical data from OK-432, TARA-002's predecessor. While the sample size is small, the consistency in safety is reassuring. The mild to moderate and resolvable nature of the TEAEs is encouraging for pediatric use. However, we'll need to closely monitor larger cohorts for a more comprehensive safety assessment.
The waiting lists for subsequent cohorts indicate high interest from both physicians and patients, potentially signaling a smoother path for recruitment and future market adoption if approved.
Protara Therapeutics' progress with TARA-002 could significantly impact its financial outlook. The positive initial results may attract investor attention and potentially increase the company's valuation. However, it's important to note that full results aren't expected until 1H'25, which means a considerable waiting period for more comprehensive data.
The unmet medical need in pediatric lymphatic malformations presents a valuable market opportunity. If TARA-002 maintains its efficacy in larger cohorts, it could become a first-in-class FDA-approved treatment, potentially leading to
Investors should monitor cash burn rate and potential partnership or licensing deals as the trial progresses.
The initial results from the STARBORN-1 trial are promising for pediatric patients with lymphatic malformations. The complete response in two out of three patients, especially after a single dose, is remarkable. This could potentially reduce treatment burden on young patients and their families.
The inclusion of different age groups in the trial design is crucial. LMs often present in early childhood, so data on safety and efficacy across various age ranges will be valuable for clinical decision-making. The planned enrollment of patients as young as six months is particularly important.
The primary endpoint focusing on volume reduction is clinically relevant, as it often correlates with symptom improvement. However, future studies should also consider quality of life measures and long-term outcomes to fully assess the impact of TARA-002 on pediatric patients' overall well-being.
- TARA-002 demonstrated compelling efficacy and was generally well-tolerated
- Enrollment in additional cohorts underway with initial results expected in 1H’25
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 completion of the first cohort of the Phase 2 STARBORN-1 trial evaluating TARA-002, an investigational cell-based immunopotentiator, for the treatment of pediatric patients with lymphatic malformations (LMs). Enrollment is now underway in additional cohorts.
“The initial data is compelling and reflective of the significant benefit observed in previous studies with OK-432, the predecessor of TARA-002,” said Nancy Bauman, MD, Children's National Medical Center: Children's Research Institute, Washington DC, and investigator for the STARBORN-1 trial. “There is a pressing need for an effective therapeutic option for LMs, a rare condition mainly affecting children for which there are currently no U.S. FDA approved agents. I remain excited about the potential for TARA-002 to play a meaningful role in the treatment of these patients and look forward to future learnings from this important study.”
Of three patients treated in the first cohort, which enrolled individuals six years to less than 18 years of age, two patients treated with TARA-002 achieved a complete response after receiving one dose of TARA-002; the responses were seen in a patient with a macrocystic lymphatic malformation and a patient with a ranula. The safety and tolerability seen in this cohort was consistent with that of the historical experience with OK-432 and included treatment emergent adverse events (TEAEs) of pain, swelling, fatigue, and body temperature increases. All TEAEs were mild to moderate and resolved.
“We are pleased with the progress of our Phase 2 STARBORN-1 trial and the encouraging results we have seen thus far,” said Jesse Shefferman, Chief Executive Officer of Protara Therapeutics. “We have received positive feedback from our investigators and already have a number of patients on waiting lists for our subsequent cohorts, and expect to share initial results from our next cohort in the first half of 2025.”
STARBORN-1 is a Phase 2 single-arm, open-label, prospective clinical trial evaluating the safety and efficacy of intracystic injection of TARA-002 for the treatment of macrocystic and mixed cystic LMs (≥
The primary endpoint of the trial is the proportion of participants with macrocystic and mixed cystic LMs who demonstrate clinical success, defined as having either a complete response (
About TARA-002 in LMs
TARA-002 is an investigational cell therapy based on the broad immunopotentiator, OK-432, which was originally granted marketing approval by the Japanese Ministry of Health and Welfare as an immunopotentiating cancer therapeutic agent. This cell therapy is currently approved in Japan and Taiwan for LMs and has been used to successfully treat thousands of pediatric patients with this rare condition. In addition, OK-432 was studied in the largest ever conducted Phase 2 trials in LMs, in which the therapy was administered via a now-closed compassionate use program led by the University of Iowa.
TARA-002 has been granted Rare Pediatric Disease designation by the U.S. Food and Drug Administration (FDA) for the treatment of LMs.
About Lymphatic Malformations
Lymphatic malformations (LMs) are rare, congenital malformations of lymphatic vessels resulting in the failure of these structures to connect or drain into the venous system. Most LMs are present in the head and neck region and are diagnosed in early childhood during the period of active lymphatic growth, with more than
About Protara Therapeutics, Inc.
Protara is committed to advancing transformative therapies for people with cancer and rare diseases. Protara’s portfolio includes its lead program, TARA-002, an investigational cell-based therapy being developed for the treatment of non-muscle invasive bladder cancer and lymphatic malformations, and IV Choline Chloride, an investigational phospholipid substrate replacement for patients dependent on parenteral support. For more information, visit www.protaratx.com.
Forward-Looking Statements
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Company Contact:
Justine O'Malley
Protara Therapeutics
Justine.OMalley@protaratx.com
646-817-2836
FAQ
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