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Protara Therapeutics Inc. (NASDAQ:TARA) on Wednesday announced interim results from its ongoing Phase 2 open-label STARBORN-1 trial assessing intracystic injection of TARA-002.
TARA-002 is the company’s investigational cell-based therapy for pediatric patients with macrocystic and mixed cystic lymphatic malformations (LMs), types of congenital lymphatic system abnormalities classified by the size of the cysts they form.
Lymphatic malformations are benign, congenital growths of abnormal and abnormally dilated lymphatic vessels that can occur anywhere in the body, most commonly in the head and neck.
The interim analysis includes a total of 12 patients. Patients receive up to four injections of TARA-002 spaced approximately six weeks apart.
Of the eight patients who were evaluable, the majority (7/8) achieved clinical success with one or two doses.
Only one patient, who presented with a 1,739 ml macrocystic LM, required all four doses and achieved a complete response.
Among patients who completed therapy, 80% (8/10) achieved clinical success, and all patients who completed the eight-week assessment (100%) (8/8) met the clinical success criteria.
In macrocystic cases, 83% (5 of 6) achieved a complete response with a 90–100% reduction in lymphatic malformation volume, while the remaining patient showed a substantial 60–<90% reduction.
The only mixed cystic patient treated achieved a complete response. Two LMs patients reached the 32-week post-treatment assessment and remain disease-free.
One patient deemed a complete response, was subsequently diagnosed with a ranula (a different type of maxillofacial cyst from LMs).
Two patients withdrew before the eight-week post-treatment assessment:
The majority of adverse events (AEs) were mild to moderate, with no serious AEs reported. The most common AEs were swelling and fatigue. One patient discontinued treatment due to a Grade 2 AE of fatigue.
Price Action: TARA stock was trading lower by 2.88% to $6.555 at last check Wednesday.
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Posted In: TARA