Amicus Therapeutics & Biogen Idec to Collaborate
Amicus Therapeutics recently announced it has entered a collaboration with Biogen Idec (BIIB) to discover, develop, and commercialize novel small molecules for the treatment of Parkinson’s disease. The collaboration will build upon preclinical studies at Amicus and independent published research that suggest increasing activity of the lysosomal enzyme glucocerobrosidase (GCase) in the brain may correct alpha-synuclein pathology and other deficits associated with Parkinson’s disease.
“Our collaboration with Amicus complements our current strategy to identify and develop novel therapies to address Parkinson’s disease,” said Tim Harris, Senior Vice President of Translational Medicine at Biogen Idec. “Amicus has been a pioneer in the discovery of novel small molecules that increase GCase activity in the brain, and we look forward to working together to discover potential treatments for Parkinson’s disease.”
Under terms of the multi-year agreement, Amicus and Biogen Idec will collaborate in the discovery of a new class of small molecules that target the GCase enzyme, for further development and commercialization by Biogen Idec. Biogen Idec will be responsible for funding all discovery, development, and commercialization activities. In addition, Amicus will be reimbursed for all full-time employees working on the project. Amicus is also eligible to receive development and regulatory milestones, as well as modest royalties on global net sales.
“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,” said John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics. “We believe that this collaboration is an important step forward in validating the potential to address neurodegenerative diseases by increasing relevant enzyme activity in the brain.”
Inherited genetic mutations in the GBA1 gene, which encodes the GCase enzyme, have been identified as the most widespread genetic risk factor for Parkinson’s disease and Dementia with Lewy bodies. The accumulation of alpha-synuclein in Lewy bodies in the brain is a hallmark of Parkinson’s disease. Independent research published over the past decade has demonstrated a link between GCase deficiency and alpha-synuclein accumulation, and suggests that improving the lysosomal targeting of GCase and increasing enzyme activity may be a beneficial therapeutic approach for Parkinson’s disease and other synucleinopathies.
Amicus Therapeutics is a biopharmaceutical company at the forefront of therapies for rare and orphan diseases. The company is developing novel, first-in-class treatments for a broad range of human genetic diseases, with a focus on delivering new benefits to individuals with lysosomal storage diseases. For more information, visit www.amicustherapeutics.com.
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