IntraBio Announces Positive Pivotal Trial Results of Levacetylleucine for the Treatment of Ataxia-Telangiectasia

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AUSTIN, Texas--(BUSINESS WIRE)--Jan 21, 2026--

IntraBio Inc. today announced positive topline results from its pivotal Phase III IB1001-303 clinical trial, “Effects of N-Acetyl-L-Leucine on Ataxia-Telangiectasia (A-T): A Randomized, Placebo-Controlled, Double-Blind, Crossover Study” (NCT06673056) evaluating N-acetyl-L-leucine (levacetylleucine) in pediatric and adult patients with Ataxia-Telangiectasia (A-T).

The primary endpoint of the trial evaluated the effect of treatment with levacetylleucine on the Scale for the Assessment and Rating of Ataxia (SARA) compared to placebo after 12 weeks of treatment. Treatment with levacetylleucine demonstrated a statistically significant and clinically meaningful -1.88 point improvement of the SARA score compared to placebo (-1.92 on levacetylleucine vs -0.14 on placebo, p<0.001).

The trial also met secondary endpoints, demonstrating statistically significant and clinically meaningful improvement 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). 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 US Food and Drug Administration (FDA) and the European Medicines Agency (EMA), and additional global regulatory authorities.

“This is a breakthrough for patients and families affected by Ataxia-Telangiectasia,” said Dr. Franziska Hoche, Massachusetts General, 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, and provide compelling evidence that levacetylleucine has a meaningful impact on A-T patients’ lives.”

“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. 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.”

The positive results from IB1001-303 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, a group of rare neurological conditions affecting approximately 1 to 2 in 10,000 people.

About IB1001-303

IB1001-303 is a Phase III clinical study evaluating levacetylleucine for the treatment of Ataxia-Telangiectasia (A-T), using the Scale for the Assessment and Rating of Ataxia (SARA) 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.

About Ataxia-Telangiectasia

Ataxia-Telangiectasia (A-T) is a rare, inherited, progressive neurodegenerative disorder that typically begins in early childhood. A-T 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. There are currently no approved treatments for the treatment of A-T.

AboutAQNEURSA ®

AQNEURSA ® (levacetylleucine) is approved in the United States, indicated for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing ≥15 kg.

Today, IntraBio also separately announced the approval of AQNEURSA ® in the European Union for the treatment of neurological manifestations of Niemann-Pick disease Type C in adults and children aged 6 years and older weighing at least 20 kg, either in combination with miglustat or as monotherapy in patients who cannot tolerate miglustat. The most commonly reported adverse reaction with AQNEURSA ® was flatulence.

About IntraBio

IntraBio Inc. is an Austin, Texas-based global biopharmaceutical company that develops and commercializes targeted therapies for rare and common neurological, neurodevelopmental, and mitochondrial diseases. IntraBio's platform technologies result from decades of research and collaboration with universities and institutions worldwide, and leverage the expertise of its scientific founders from the University of Oxford and the University of Munich.

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CONTACT: For further information, please contact:

Cass Fields

Vice-President of External Affairs

[email protected]

www.intrabio.com

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SOURCE: IntraBio Inc.

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PUB: 01/21/2026 08:00 AM/DISC: 01/21/2026 08:00 AM

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