TARA-002 Achieves 100% Clinical Success Rate in Phase 2 Trial for Pediatric Lymphatic Malformations
核心洞察
TARA-002 demonstrated 100% clinical success in all eight pediatric patients who completed the eight-week response assessment in the ongoing Phase 2 STARBORN-1 trial for lymphatic malformations (搜索).
The investigational cell-based therapy achieved clinical success with just one or two doses in 88% of patients, with 83% of macrocystic patients achieving complete response.
TARA-002 showed a favorable safety profile with no serious adverse events reported, addressing a significant unmet medical need as no approved therapies currently exist for lymphatic malformations (搜索).
Protara Therapeutics announced positive interim results from its Phase 2 STARBORN-1 trial of TARA-002, an investigational cell-based therapy for pediatric patients with lymphatic malformations (搜索) (LMs). The data revealed that 100% of patients who completed the eight-week response assessment achieved clinical success, representing a significant breakthrough for a rare condition with no approved treatments.
Strong Efficacy Results Across Patient Population
The interim analysis included 12 patients who received at least one dose of TARA-002 as of the November 12, 2025 data cutoff. Of the eight patients evaluable at the eight-week post-treatment assessment, all achieved clinical success. Notably, 88% of patients (7 out of 8) achieved clinical success with just one or two doses of the therapy.
Among patients who completed treatment, 80% achieved clinical success. The results were particularly impressive in macrocystic patients, where 83% (5 out of 6) achieved a complete response, defined as 90% to 100% reduction in total LM volume. One additional macrocystic patient achieved a substantial response (60% to less than 90% reduction). The single mixed cystic patient treated also achieved a complete response.
"We are pleased to report these robust results from the STARBORN-1 trial that demonstrate TARA-002's expected significant clinical benefit in treating patients with macrocystic and mixed cystic LMs," said Jesse Shefferman, Chief Executive Officer of Protara Therapeutics. "Treatment with TARA-002 resulted in clinically meaningful responses, with a favorable safety profile observed across all evaluable patients."
Addressing Critical Unmet Medical Need
The results are particularly significant given the current treatment landscape for lymphatic malformations (搜索). "There are currently no approved therapies for LMs, with many patients turning to invasive surgical procedures that carry high rates of complication and recurrence, or off-label use of chemotherapies and chemicals, which can have challenging side effects, especially for pediatric patients," explained Jesse G.A. Jones, M.D., Associate Professor at the University of Alabama at Birmingham and STARBORN-1 study investigator.
Two patients reached the 32-week post-treatment assessment and remained disease-free, suggesting potential durability of response. One notable case involved a patient who dropped out after achieving notable resolution of their macrocystic LM following two doses, with aspiration volume decreasing from 160 ml at the first dose to 10 ml at the second dose.
Favorable Safety Profile
TARA-002 demonstrated a favorable safety and tolerability profile with no serious adverse events reported. The majority of adverse events were mild to moderate, with the most common being swelling and fatigue. Only one patient discontinued treatment due to a Grade 2 adverse event of fatigue.
About TARA-002 and Trial Design
TARA-002 is an investigational, genetically distinct strain of streptococcus pyogenes that is inactivated while retaining its immune-stimulating properties. The therapy was developed from the same master cell bank as OK-432, which has been the standard of care for LMs in Japan for 30 years and was studied in a large Phase 2 trial involving over 500 patients.
STARBORN-1 is a Phase 2 single-arm, open-label trial evaluating intracystic injection of TARA-002 in 29 participants aged six months to less than 18 years with macrocystic and mixed cystic LMs. Patients receive up to four injections spaced approximately six weeks apart. TARA-002 has been granted Rare Pediatric Disease designation by the FDA for the treatment of LMs.
Lymphatic malformations (搜索) are rare, congenital malformations of lymphatic vessels that fail to connect or drain into the venous system. Most occur in the head and neck region, with more than 50% detected at birth and 90% diagnosed before age three. The condition can cause serious complications including airway obstruction, bleeding, and functional disabilities.