IntraBio will head to regulators after its neurological rare disease drug met primary and secondary endpoints in a Phase III trial in paediatric and adult patients with ataxia-telangiectasia (A-T).
In the study (NCT06673056), levacetylleucine, a modified form of the amino acid leucine, demonstrated 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.
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The trial also met secondary endpoints, demonstrating statistically significant and clinically meaningful improvement on the International Cooperative Ataxia Rating Scale (ICARS), with a 4.22-point reduction with patients treated with levacetylleucine compared to a 1.69-point reduction in the placebo cohort.
The drug was found to be safe and well-tolerated, with no drug-related serious adverse events (AEs) observed in the trial.
Based on these results, IntraBio plans to immediately advance regulatory submissions to the US Food and Drug Administration (FDA), the European Medicines Agency (EMA), and additional global regulatory authorities.
The drug has already received EMA and FDA approval in Niemann-Pick Disease Type C (NPC), where it is marketed as Aqneursa.
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By GlobalDataDr Franziska Hoche, Massachusetts General, investigator of the study, said: “This is a breakthrough for patients and families affected by A-T. The results from the 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 patients’ lives.”
A-T is a rare inherited childhood disorder caused by a mutated ATM gene and affects the nervous system, immune system, and other body systems. Symptoms include movement problems, poor coordination, and speech issues. There are no approved treatments, making this trial a notable development for patients.
Brad Margus, founder of the A-T Children’s Project, added: “These results mark a major turning point for the A-T community. 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.”
IntraBio is not the only company looking into therapies for this patient population. Quince is currently running a pivotal, Phase III trial of its drug EryDex, a corticosteroid comprised of dexamethasone sodium phosphate (DSP) encapsulated in a patient’s own red blood cells (autologous erythrocytes), in patients with A-T, evaluating the neurological effects of the drug in paediatric patients. If successful, Quince will submit a New Drug Application (NDA) for the medication to the FDA in 2026.
