AUSTIN, Texas — IntraBio Inc. on Tuesday reported positive topline results from its pivotal Phase III clinical trial evaluating levacetylleucine in pediatric and adult patients with Ataxia-Telangiectasia, a rare and progressive neurodegenerative disorder with no approved treatments.
The IB1001-303 study, a randomized, placebo-controlled, double-blind crossover trial, met its primary endpoint by demonstrating a statistically significant and clinically meaningful improvement in neurological function compared with placebo. The primary endpoint measured changes on the Scale for the Assessment and Rating of Ataxia after 12 weeks of treatment.
Patients treated with levacetylleucine showed a 1.88-point improvement in SARA scores compared with placebo, with scores declining by 1.92 points on active treatment versus 0.14 points on placebo. The difference was statistically significant, with a p-value of less than 0.001.
The trial also met key secondary endpoints. Patients receiving levacetylleucine demonstrated greater improvement on the International Cooperative Ataxia Rating Scale, with a 4.22-point reduction compared with a 1.69-point reduction on placebo. Investigators also reported improvement on the Clinical Global Impression of Improvement scale, with scores improving by 0.6 on levacetylleucine versus 0.2 on placebo. The drug was reported to be safe and well tolerated, with no drug-related serious adverse events, consistent with its established safety profile.
Based on the results, IntraBio said it plans to advance regulatory submissions to the U.S. Food and Drug Administration, the European Medicines Agency, and other regulatory authorities worldwide.
“This is a breakthrough for patients and families affected by Ataxia-Telangiectasia,” said Dr. Franziska Hoche of Massachusetts General, an investigator in the IB1001-303 study. “A-T is a rare and devastating disorder with no approved treatments. The results from the IB1001-303 trial, demonstrating levacetylleucine significantly improved patients’ neurological symptoms and everyday function, represent a major scientific and clinical milestone, and provide compelling evidence that levacetylleucine has a meaningful impact on A-T patients’ lives.”
Brad Margus, Founder of the A-T Children’s Project, said the findings offer renewed hope for the patient community. “These results mark a major turning point for the Ataxia-Telangiectasia community,” he said. “This offers real hope that families will soon have access to their first effective and safe treatment approved for A-T. 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.”
IntraBio said the results build on prior clinical experience with levacetylleucine across a range of neurological, neurodevelopmental, and mitochondrial disorders. The compound is also in late-stage development in the United States and Europe for CACNA1A-related disorders, a group of rare neurological conditions affecting an estimated one to two people per 10,000.
Ataxia-Telangiectasia is a rare inherited disorder that typically begins in early childhood and is marked by progressive loss of coordination, impaired speech and eye movements, immune deficiencies, lung disease, and a substantially increased risk of cancer. The condition affects approximately one in 70,000 people worldwide and currently has no approved treatments.
