IntraBio Announces Publication of Pivotal Phase III Data with Levacetylleucine for Ataxia-Telangiectasia in The Lancet Neurology

AUSTIN, Texas--(BUSINESS WIRE)--IntraBio Inc. today announced that results from its pivotal Phase III clinical trial evaluating levacetylleucine for the treatment of Ataxia-Telangiectasia (A-T) have been published in The Lancet Neurology.

The peer-reviewed publication, titled “Safety and efficacy of levacetylleucine in ataxia telangiectasia: a phase 3, randomised, double-blind, placebo-controlled crossover trial” reports findings from IntraBio’s pivotal randomized, double-blind placebo-controlled Phase III study of levacetylleucine in patients with A-T, a rare, inherited, progressive neurological disease.

This pivotal study met its primary endpoint, demonstrating statistically significant improvement in neurological symptoms and function compared with placebo, as measured by the Scale for the Assessment and Rating of Ataxia. Treatment with levacetylleucine resulted in a mean change in SARA score of -1.92 compared with -0.14 for placebo, representing a treatment difference of -1.88 points (p<0.001). Levacetylleucine also demonstrated statistically significant improvements on key secondary endpoints, including the International Cooperative Ataxia Rating Scale and Investigator Clinical Global Impression. Levacetylleucine was generally well tolerated, with no drug-related serious adverse events reported.

“These findings provide important clinical evidence for levacetylleucine in Ataxia-Telangiectasia, a rare and progressive disease that can profoundly impact movement, coordination, speech, swallowing, and daily functioning,” said PD Dr. med. Tatiana Brémovà-Ertl, PhD, Principal Investigator from the University of Bern. “The results published in The Lancet Neurology represent an important contribution to the understanding of A-T and the potential role of levacetylleucine in addressing the neurological manifestations of the disease.”

The full publication is available at: https://www.thelancet.com/journals/laneur/article/PIIS1474-4422(26)00158-4/fulltext

A supplementary New Drug Application (sNDA) with Levacetylleucine is currently under review by the U.S. Food and Drug Administration for the treatment of Ataxia-Telangiectasia, with a Prescription Drug User Fee Act target action date of September 19, 2026. A variation application has been submitted to the European Medicines Agency to expand the approved marketing authorization for levacetylleucine to include the treatment of Ataxia-Telangiectasia. Levacetylleucine is an experimental therapy for A-T and has not been approved by any jurisdiction for this indication.

ABOUT ATAXIA-TELANGIECTASIA (A-T)

Ataxia-Telangiectasia is a rare, inherited, progressive neurodegenerative disorder that typically manifests in early childhood. A-T is estimated to affect 1 in 40,000-100,000 people. It is characterized by cerebellar degeneration leading to worsening loss of coordination, impaired speech, abnormal eye movements, and eventual wheelchair dependence. Many patients also develop telangiectasia, immune deficiency with recurrent and potentially life-threatening infections, pulmonary disease, and a significantly elevated risk of cancer. There are currently no FDA-approved therapies for A-T.

ABOUT INTRABIO

IntraBio Inc. is a global biopharmaceutical company headquartered in Austin, Texas, focused on developing and commercializing targeted therapies for rare and common neurological, neurodevelopmental, and mitochondrial diseases. IntraBio’s platform technologies are built on decades of scientific research and collaboration with leading institutions worldwide, including the University of Oxford and the University of Munich.