IntraBio’s Neurological Rare Disease Drug Shows Positive Phase III Results in Ataxia-Telangiectasia

IntraBio announced that its drug levacetylleucine met primary and secondary endpoints in a Phase III trial (NCT06673056) for pediatric and adult patients with ataxia-telangiectasia (A-T), a rare genetic disorder affecting coordination, movement, and the immune system. After 12 weeks, patients treated with levacetylleucine showed a -1.88 point improvement on the Scale for the Assessment and Rating of Ataxia (SARA) versus placebo. Secondary endpoints, including the International Cooperative Ataxia Rating Scale (ICARS), also demonstrated significant improvement.

The drug was well-tolerated with no drug-related serious adverse events. Based on these results, IntraBio plans to submit regulatory filings to the FDA, EMA, and other authorities. Levacetylleucine is already approved in Niemann-Pick Disease Type C under the brand Aqneursa. Investigators and patient advocates called the results a breakthrough, offering hope for the first approved therapy for A-T. Quince is also pursuing A-T therapies with its EryDex program.

26-01-2026