IntraBio is a biopharmaceutical company focused on developing treatments for progressive neurological conditions. This page provides updates on their Levacetylleucine programme.
Levacetylleucine is a chemically modified version of the amino acid L-leucine. Amino acids are the building blocks of proteins. There have been case reports over the years describing the benefits of similar chemically modified derivatives of leucine, and IntraBio has taken this forward initially with a clinical trial in Niemann-Pick Disease Type C (NPC), then Ataxia-Telangiectasia (A-T), and has plans for a clinical trial in other ataxias including CACNA1A and specifically SCA6 and episodic ataxia.
Update 25/09/2024: IntraBio receive FDA approval of Levacetylleucine for the treatment of Niemann-Pick Disease Type C (NPC)
On 25th September 2024, the pharmaceutical company IntraBio announced that the FDA in the US has approved Levacetylleucine for the treatment of neurological symptoms of NPC. NPC is a rare lysosomal storage disorder, in which ataxia can be a presenting symptom. The FDA approval follows positive data from their phase III placebo-controlled clinical trial which showed significant improvement in neurological symptoms and functional benefits in adults and children with NPC taking Levacetylleucine within 12 weeks of administration, including on the Scale for the Assessment and Rating of Ataxia (SARA) and Spinocerebellar Ataxia Functional Index (SCAFI). Read the press release here.
IntraBio’s Chief Development Officer Taylor Fields commented,
“IntraBio has been dedicated to bringing novel treatments to patients with extremely high unmet medical needs like NPC. We remain committed to ensuring that all patients who can benefit from this novel treatment will have the opportunity to do so. Based on our clinical research, we believe that Levacetylleucine may hold potential for treating other rare and common neurodegenerative and neurodevelopmental disorders, and we will continue to rapidly develop Levacetylleucine for these additional indications.”
We previously reported on the positive results from IntraBio’s multinational Phase IIb clinical trials testing Levacetylleucine in NPC and GM2 Gangliosidosis. Levacetylleucine resulted in a clinically meaningful improvement of symptoms, including in the Scale for the Assessment and Rating of Ataxia (SARA) score. The treatment was observed to be safe and well-tolerated, with no drug-related serious adverse events. The trial took place in the USA, European Union and the UK.
A representative of IntraBio has previously attended Ataxia UK’s patient conference to present data on this trial and Ataxia UK has continued to work collaboratively with IntraBio, supporting them through their development programme in a number of ways. For example, Dr Julie Greenfield, Director of Research at Ataxia UK, is co-author of a publication with IntraBio, detailing the development of a protocol to investigate Levacetylleucine.
IntraBio has also explored the potential of this drug in multiple inherited cerebellar ataxias, including Ataxia-Telangiectasia, Spinocerebellar ataxias and episodic ataxias, so it may offer hope in the future.
Update September 2025: IntraBio to conduct phase III trial in SCA6 and Episodic Ataxia Type-2
IntraBio has announced that they plan to trial Levacetylleucine in CACNA1A-related disorders, which includes SCA6 and EA2. They hope to be able to start the trial in 2026 and are exploring potential UK sites. We will share more about this through our communications channels.
Updates 21/01/2026:
IntraBio receive EMA approval of Levacetylleucine for the treatment of Niemann-Pick Disease Type C (NPC)
On 21st January 2025, Levacetylleucine was approved by the European Medicines Association (EMA) for the treatment neurological symptoms of Niemann-Pick Type C (NPC) disease, following a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) in 2025. Read the press release here.
IntraBio announce positive topline Phase III trial results of Levacetylleucine for Ataxia-Telangiectasia (A-T)
In January 2026, IntraBio announced that their phase III trial of Levacetylleucine for the treatment of A-T had met the primary endpoint by demonstrating a statistically significant and clinically meaningful improvement in SARA score compared to placebo after 12 weeks of treatment. The trial also met secondary endpoints. The drug was safe and well-tolerated. IntraBio now plans to pursue regulatory approval by submitting to the FDA and EMA as well as other regulatory authorities. Read the press release here.
Update 24/02/2026: Levacetylleucine received a positive opinion from the EMA recommending it receives Orphan Medicinal Product Designation for CACNA1A disorders
Levacetylleucine received a positive opinion from the EMA recommending it receives Orphan Medicinal Product Designation for CACNA1A disorders which is expected to be granted in the second quarter of 2026. This designation, which is granted to rare, serious or life-threatening conditions, provides companies with regulatory incentives. Read more here.
