Protara Therapeutics has announced encouraging interim data from its ongoing Phase II STARBORN-1 study evaluating TARA-002 in paediatric subjects with lymphatic malformations (LMs).
The trial is assessing intracystic injection of TARA-002 in children with macrocystic and mixed cystic LMs, a condition currently lacking approved treatments.
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It enrolled 12 participants who received at least one dose of TARA-002. Of the total participants, eight of them completed the eight-week response assessment and achieved clinical success.
Seven participants responded after just one or two doses. One subject with a 1,739ml macrocystic lymphatic malformation required all four doses and achieved a complete response.
Participants received up to four injections spaced nearly six weeks apart.
In the trial, 83% of those with macrocystic lesions achieved a complete response, defined as a 90% to 100% reduction in total lymphatic malformation volume.
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By GlobalDataOne participant experienced a substantial response, defined as a reduction of 60% to less than 90%. The sole participant with a mixed cystic lesion who received treatment also achieved a complete response.
Two participants completed the 32-week post-treatment evaluation and continue to be free of disease.
Another two subjects withdrew before the assessment period of eight weeks, one was misdiagnosed with cancer, and one dropped out after achieving notable resolution following two doses, with aspiration reduced from 160ml to 10ml between doses.
Protara CEO Jesse Shefferman said: “Treatment with TARA-002 resulted in clinically meaningful responses, with a favourable safety profile observed across all evaluable patients.
“The totality of available clinical data, including data from prior studies with TARA-002’s predecessor compound OK-432, an established treatment for LMs in Japan, underscore our belief in the potential for TARA-002 to emerge as an important intervention for pediatric patients suffering from LMs.”
Most adverse events were mild to moderate, including fatigue and swelling, with no serious adverse events reported. One participant discontinued due to Grade 2 fatigue.
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