Texas-based company IntraBio plans to expand the indications for its drug Aqneursa (levacetylleucine), which is currently approved to treat Niemann-Pick disease type C (NPC).
The pharmaceutical manufacturer intends to “immediately” advance regulatory submissions to the U.S. Food and Drug Administration (FDA), the European Medicines Agency (EMA), and other global authorities for the use of Aqneursa in treating Ataxia-Telangiectasia (A-T). This neurodegenerative disease affects approximately 1 in 70,000 people and currently has no approved treatments, according to an IntraBio press release.
IntraBio’s optimism is based on the results of a Phase III clinical trial, in which 12 weeks of treatment with levacetylleucine led to a “statistically significant and clinically meaningful improvement” of -1.88 points (-1.92 for the drug vs. -0.14 for placebo) on the Scale for the Assessment and Rating of Ataxia (SARA). The trial also met its secondary endpoints based on positive results in other clinical measures of ataxia.
“Levacetylleucine was observed to be safe and well-tolerated, with no drug-related serious adverse events, consistent with its established safety profile,” the company emphasized.
“This offers real hope that families will soon have access to their first effective and safe treatment approved for A-T,” said Brad Margus, founder of the non-profit A-T Children’s Project. “We look forward to continuing to collaborate with IntraBio to help ensure levacetylleucine is rapidly approved by the FDA and made available for patients in our community, given their urgent need for effective, approved treatments”.
Ataxia-Telangiectasia (A-T) is a rare, inherited, progressive disorder that typically begins in early childhood. The disease is characterized by degeneration of the cerebellum, leading to worsening loss of coordination, impaired speech and eye movements, and 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.
The positive trial results build on prior clinical experience with levacetylleucine across various neurological, neurodevelopmental, and mitochondrial disorders, the company noted. The drug is also in late-stage development in the U.S. and Europe for CACNA1A-related disorders — a group of rare neurological conditions affecting approximately 1 to 2 in 10,000 people.