US-based Protara Therapeutics has commenced dosing in its Phase II STARBORN-1 trial investigating its cell therapy candidate for the treatment of lymphatic malformations in paediatric patients.
Lymphatic malformations are masses of abnormal lymph cells that are benign but grow into cysts.
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The New York-headquartered company’s candidate, TARA-002, will be injected intracystically every six weeks in approximately 30 patients for four total injections each.
The drug was granted rare paediatric disease designation by the US Food and Drug Administration (FDA) in July 2020.
The single-arm, open-label, prospective trial (NCT05871970) will evaluate the safety and efficacy of TARA-002 in treating macrocystic and mixed cystic lymphatic malformations in patients aged six months to 18 years. The primary endpoint is a complete response or substantial response, characterised by a 90%-100% or a 60%-90% reduction from baseline in total lymphatic malformation volume respectively.
TARA-002 is a cell therapy based on the immunopotentiator OK-432, a lyophilised mixture of group A Streptococcus pyogenes that is approved in Japan as an immunotherapeutic agent marketed as Picibanil by Chugai Pharmaceutical.
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By GlobalDataProtara believes TARA-002 works by producing an immune cascade which results in neutrophils, monocytes, and lymphocytes infiltrating the abnormal cells. Various cytokines and interleukins, in addition to tumour necrosis factor (TNF)-alpha, are secreted during the immune response which helps destroy the abnormal cells.
Protara Therapeutics CEO Jesse Shefferman said: “Initiation of the STARBORN-1 trial marks an important step forward for our lymphatic malformation programme and, more importantly, the young patients impacted by this rare condition.”
In May 2023, the company began dosing in the Phase Ib ADVANCED-1EXP expansion trial of TARA-002 to treat high-grade non-muscle invasive bladder cancer in patients with carcinoma in situ (CIS).
