IntraBio's Levacetylleucine Achieves Primary Endpoint in Pivotal Phase III Trial for Ataxia-Telangiectasia
Key Insights
IntraBio's pivotal Phase III trial IB1001-303 successfully met its primary endpoint, demonstrating a statistically significant -1.88 point improvement in SARA scores compared to placebo (p<0.001) for levacetylleucine in Ataxia-Telangiectasia (search) patients.
The trial also achieved key secondary endpoints with significant improvements on the ICARS scale (-4.22 vs -1.69 on placebo; p=0.003) and Clinical Global Impression of Improvement scores.
Levacetylleucine showed favorable safety and tolerability with no drug-related serious adverse events, marking a potential breakthrough for A-T patients who currently have no approved treatments.
IntraBio Inc. (search) announced positive topline results from its pivotal Phase III IB1001-303 clinical trial evaluating levacetylleucine (N-acetyl-L-leucine) in pediatric and adult patients with Ataxia-Telangiectasia (search) (A-T), a rare neurodegenerative disorder affecting approximately 1 in 70,000 people with no currently approved treatments.
Trial Results Demonstrate Statistical Significance
The randomized, placebo-controlled, double-blind crossover study successfully achieved its primary endpoint, demonstrating a statistically significant and clinically meaningful -1.88 point improvement on the Scale for the Assessment and Rating of Ataxia (SARA) compared to placebo after 12 weeks of treatment (-1.92 on levacetylleucine vs -0.14 on placebo, p<0.001).
The trial also met key secondary endpoints with statistically significant improvements on the International Cooperative Ataxia Rating Scale (ICARS) (-4.22 on levacetylleucine vs. -1.69 on placebo; p=0.003) and the Investigator's Clinical Global Impression of Improvement (CGI-I) (-0.6 on levacetylleucine vs. -0.2 on placebo; p=0.02).
Safety Profile Remains Favorable
Levacetylleucine demonstrated favorable safety and tolerability with no drug-related serious adverse events, consistent with its established safety profile. The drug is already approved in the United States as AQNEURSA® for treating neurological manifestations of Niemann-Pick disease type C (search) (NPC) in adults and pediatric patients weighing ≥15 kg, with flatulence being the most commonly reported adverse reaction.
Clinical Impact for Underserved Population
"This is a breakthrough for patients and families affected by Ataxia-Telangiectasia (search)," said Dr. Franziska Hoche, Massachusetts General (search), Investigator of 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."
Brad Margus, Founder of the A-T Children's Project (search), emphasized the significance for the patient community: "These results mark a major turning point for the Ataxia-Telangiectasia (search) community. This offers real hope that families will soon have access to their first effective and safe treatment approved for A-T."
Regulatory Strategy and Development Pipeline
Based on these results, IntraBio plans to immediately advance regulatory submissions to the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA), along with additional global regulatory authorities. The positive results build on prior clinical experience with levacetylleucine across multiple neurological, neurodevelopmental, and mitochondrial disorders.
Levacetylleucine is also in late-stage development in the United States and Europe for CACNA1A-related disorders (search), a group of rare neurological conditions affecting approximately 1 to 2 in 10,000 people.
Understanding Ataxia-Telangiectasia
Ataxia-Telangiectasia (search) is a rare, inherited, progressive neurodegenerative disorder that typically begins in early childhood. The condition 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 IB1001-303 study was designed in partnership with key opinion leaders and A-T patient organizations to evaluate both symptomatic effects and longer-term clinical outcomes, incorporating a randomized, placebo-controlled, double-blind crossover phase followed by a long-term open-label extension.