Biogen Idec has signed a deal with Amicus Therapeutics to develop treatments for Parkinson’s disease that target a genetic mutation associated with the neurological condition.
The companies are looking at inherited genetic mutations in the GBA1 gene, which has been identified as the most widespread genetic risk factor for Parkinson’s disease and dementia.
This gene encodes the enzyme glucocerobrosidase (GCase) and if the gene is mutated there can be a deficient level of this enzyme, leading to an accumulation of alpha-synuclein – a protein that is a hallmark of Parkinson’s disease.
Amicus, which usually works in rare diseases, has been involved in preclinical studies of small molecules targeting the GCase enzyme, with early trials suggesting that increasing the activity of the enzyme in the brain may correct alpha-synuclein pathology and other deficits associated with Parkinson’s disease.
Full financial terms of the deal were not disclosed, although Biogen Idec will be responsible for funding all discovery, development, and commercialisation activities.
In addition Amicus will be reimbursed for all full-time employees working on the project and the company is also eligible to receive development and regulatory milestones and royalties.
The deal fits with Biogen Idec’s wider plans in neurological medicine, which most recently involved the expansion of an existing R&D collaboration with Isis to develop treatments using the company’s antisense technology.
Biogen Idec also has an existing major presence in the field of neurodegenerative conditions through its huge selling multiple sclerosis treatment Tysabri (natalizumab), as well as new oral treatment Tecfidera (dimethyl fumarate) for relapsing-remitting multiple sclerosis.
John Crowley, chairman and CEO of Amicus, said this experience was one of the reasons for the deal between the two companies.
“This partnership combines Biogen Idec’s leadership in neurodegenerative diseases with our internal expertise in discovering small molecules that enhance the activity of lysosomal enzymes.”