IntraBio’s A-T Therapy Meets Phase 3 Endpoints, Will Seek FDA Approval

IntraBio reported topline results from its pivotal phase 3 clinical trial of its experimental therapy levacetylleucine for children and adults with the rare, neurodegenerative disorder ataxia-telangiectasia, showing that the study met its primary and secondary endpoints. Ataxia-telangiectasia is a rare, inherited, progressive neurodegenerative disorder that typically begins in early childhood.

The company reported that treatment with levacetylleucine led to a statistically significant and clinically meaningful improvement on the Scale for the Assessment and Rating of Ataxia (SARA) compared with placebo after 12 weeks of treatment. The trial also met secondary endpoints, demonstrating statistically significant and clinically meaningful improvements on the International Cooperative Ataxia Rating Scale (ICARS), and on the investigator’s Clinical Global Impression of Improvement.

Levacetylleucine was observed to be safe and well-tolerated, with no drug-related serious adverse events, consistent with its established safety profile. Based on these results, IntraBio plans to immediately advance regulatory submissions to the U.S. Food and Drug Administration, the European Medicines Agency, and additional global regulatory authorities.

A-T is characterized by degeneration of the cerebellum, leading to progressive loss of coordination, impaired speech and abnormal eye movements, and eventual wheelchair dependence. Many patients also develop visible blood vessel changes (telangiectasia), immune system deficiencies with recurrent, life-threatening infections, lung disease, and a dramatically increased risk of cancer. There are currently no approved therapies for A-T.

IB1001-303 is a phase 3 clinical study evaluating levacetylleucine for the treatment of A-T, using the SARA score as the primary endpoint. The study includes a randomized, placebo-controlled, double-blind crossover phase, followed by a long-term open-label extension. It was designed in partnership with key opinion leaders and A-T patient organizations to evaluate both symptomatic effects and longer-term clinical outcomes. Levacetylleucine is an investigational drug for A-T and has not yet been approved by the FDA or any other regulatory authority for this indication.

Levacetylleucine is approved in the United States for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing 15 kilograms or more. Separately, IntraBio reported that the European Union approved levacetylleucine for the treatment of neurological manifestations of Niemann-Pick disease type C in adults and children 6 years and older weighing at least 20 kilograms, either in combination with miglustat or as monotherapy in patients who cannot tolerate miglustat.

“These results mark a major turning point for the ataxia-telangiectasia community,” said Brad Margus, founder of the A-T Children’s Project. “This offers real hope that families will soon have access to their first effective and safe treatment approved for A-T.”

Photo: Brad Margus, founder of the A-T Children’s Project