There have been strong early results from Protara Therapeutics ongoing phase 2 STARBORN-1 trial evaluating TARA-002 in paediatric patients with lymphatic malformations (LMs), a rare and often debilitating condition with no approved therapies.

The interim analysis included 12 patients who received at least one dose of TARA-002.
Out of the eight patients who completed the eight-week response assessment, all achieved clinical success.

Seven of these patients responded after just one or two doses. One patient with a large 1,739ml macrocystic LM required all four doses and achieved a complete response.

TARA-002 was well tolerated, with no serious adverse events reported. Most side effects were mild to moderate, including swelling and fatigue.

One patient discontinued due to a grade 2 fatigue event.

Jesse Shefferman, Chief Executive Officer of Protara Therapeutics, said: “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.” He added: “Treatment with TARA-002 resulted in clinically meaningful responses, with a favorable safety profile observed across all evaluable patients.”

Jesse G.A. Jones, Associate Professor at the University of Alabama at Birmingham and study investigator, said: “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. I am encouraged by the positive interim safety and efficacy data from TARA-002 and believe this promising candidate has the potential to help the many patients in need of FDA-approved therapeutic approaches for LMs.”