IntraBio’s Levacetylleucine Succeeds in Phase 3 Trial for Ataxia-Telangiectasia, Plans FDA and EMA Approval

IntraBio announced positive topline results from the Phase 3 IB1001-303 trial of levacetylleucine (IB1001) for Ataxia-Telangiectasia (A-T) on January 21, 2026.4

The trial met its primary endpoint with a statistically significant -1.88 point improvement on the Scale for the Assessment and Rating of Ataxia (SARA) compared to placebo (p<0.001).134

Key secondary endpoints were also met, including improvements on the International Cooperative Ataxia Rating Scale (ICARS) (-4.22 vs. -1.69; p=0.003) and Investigator’s Clinical Global Impression of Improvement (CGI-I).34

Levacetylleucine was safe and well-tolerated with no drug-related serious adverse events.134

IntraBio plans immediate regulatory submissions to the FDA, EMA, and other authorities for A-T, a neurodegenerative disease with no approved treatments affecting ~1 in 70,000 people.34

The drug is already approved as Aqneursa for Niemann-Pick Disease Type C in the US and EMA.34

Sources:

1. https://www.neurologylive.com/view/levacetylleucine-meets-primary-and-secondary-end-points-phase-3-ataxia-telangiectasia-study

3. https://www.clinicaltrialsarena.com/news/intrabios-ataxia-telangiectasia-drug-meets-endpoints-in-pivotal-trial/

4. https://www.businesswire.com/news/home/20260121091020/en/IntraBio-Announces-Positive-Pivotal-Trial-Results-of-Levacetylleucine-for-the-Treatment-of-Ataxia-Telangiectasia