Clearance given to clinical trial with potential to change course of brain disease

August 24, 2022 - 3 minutes
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A trial to look at gene therapy, BV-01, for Huntington’s Disease has been given the go ahead to take place in France and has ‘the potential to change the course of the devastating disease.  

Asklepios BioPharmaceutical, Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG, received clearance for the phase 1/2 trial today (August 24).

The authorization was provided by the National Agency for Safety of Medicines and Health Products (ANSM), the country’s governing drug authority, alongside approval of the trial protocol by the Ethics Committee in charge. It enables Asklepios BioPharmaceutical to begin recruiting participants.

The trial will be undertaken by BrainVectis, a subsidiary of AskBio.

Nathalie Cartier-Lacave is a physician researcher, gene therapy pioneer and founder of BrainVectis.

Mutant huntingtin

She said: “Unlike other attempts to treat Huntington’s Disease, BV-101 aims to restore cholesterol metabolism, reduce mutant huntingtin and to improve neuronal function. Importantly, BV-101 does not affect the levels of normal huntingtin protein in cells.

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“If this proves successful, we have the potential to change the course of a devastating disease that causes severe functional and cognitive decline.”

BV-101 is a novel, exclusively designed adeno-associated virus (AAV) gene therapy vector that simultaneously addresses the metabolic dysfunction of diseased neurons as well as contributes to the clearance of the mutant huntingtin protein.

Basal structures

BV-101 is administered through MRI-guided neurosurgical techniques directed to target tissues in the basal structures of the brain. In preclinical studies in mice, BV-101 demonstrated the ability to repair the essential cholesterol pathway, provide neuroprotection, and restore physical performance by delivering CYP46A1, a crucial enzyme in the brain, which is reduced in people with Huntington’s Disease. BV-101 was granted orphan drug designation in the European Union in 2019 by the European Medicines Agency.

Currently there are no approved disease modifying therapies for HD, a rare inherited neurodegenerative disease that, based on information from the Committee for Orphan Medicinal Products (COMP), affects approximately 62,000 people in the European Union.

Protein aggregates

The Hutington’s disease is caused by anomalous repeating mutations in the huntingtin gene leading to abnormal protein aggregates in nerve cells. This results in a range of progressive symptoms, leading to complete physical and mental deterioration, with symptoms usually beginning in adults ages 30 to 50, but which can also occur at an earlier age.

Sheila Mikhail, co-founder of AskBio, said: “The approval of this trial in France marks a major milestone to potentially treat one of the world’s most devastating genetic diseases.

“If successful, this novel approach for treating Huntington’s Disease may impact how we treat many other neurodegenerative diseases in the future.”

The BV-101 clinical trial will be an open-label, dose-escalation study to assess the safety, tolerability, and preliminary efficacy of administration of BV-101 in adult subjects with early-stage Huntington’s Disease (HD). The trial will include 12-18 participants and is expected to begin in Paris in the fourth quarter of this year (2022).

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