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Amicus Therapeutics Awarded $500,000 Grant from The Michael J. Fox Foundation for Development of Next-Generation Pharmacological Chaperone to Treat Parkinson's Disease

CRANBURY, N.J., Dec. 8, 2010 /PRNewswire/ -- Amicus Therapeutics (Nasdaq: FOLD), a biopharmaceutical company focused on developing treatments for rare diseases, today announced that it has been awarded a second grant from The Michael J. Fox Foundation for Parkinson's Research (MJFF) for the development of a novel, next-generation pharmacological chaperone for the treatment of Parkinson's disease.  The $500,000 award will be distributed over a period of 24 months. Amicus' studies will be conducted in collaboration with Marie-Francoise Chesselet, M.D., Ph.D. the Charles H. Markham Professor of Neurology and Chair, Department of Neurobiology, David Geffen School of Medicine at UCLA.  

"We are honored to receive this grant from the MJFF, which will help us further develop our novel treatment for Parkinson's disease," said David J. Lockhart, Ph.D., Amicus Therapeutics' Chief Scientific Officer.  "Currently available treatments for Parkinson's provide symptomatic relief only. Our therapy is designed to address a deficiency that is inherited in a subset of the Parkinson's population, and to actually modify the course of the disease.  The targeted compounds that we have discovered have shown promising effects in initial animal studies, and we are delighted to be working together with the MJFF and Dr. Chesselet to advance our lead compound into advanced preclinical studies."

The studies will focus on the Parkinson's-implicated protein alpha-synuclein. Accumulation of alpha-synuclein in the nervous system is a hallmark of Parkinson's disease. Amicus is working on a strategy to reduce levels of synuclein in the brain, in the hope that this would lessen the changes in the brain seen in Parkinson's disease -- such as loss of the neurotransmitter dopamine -- and resulting behavioral changes.    

"I am pleased to continue working with Amicus on their exciting pharmacological chaperone approach to modify the progression of Parkinson's disease," said Dr. Chesselet. "Amicus' earlier compound partially reversed the motor deficits in our alpha-synuclein overexpression mouse model, and we are eager to test the improved compounds."

John F. Crowley, Chairman and CEO of Amicus Therapeutics stated, "We appreciate MJFF's backing of our pharmacological chaperone technology and believe this grant further demonstrates the strong scientific capabilities at Amicus.  We hope that these continued discoveries will translate into therapies for people living with Parkinson's disease."

About Pharmacological Chaperones

Pharmacological chaperones are orally-available small molecules that selectively bind and stabilize target proteins to facilitate proper folding, reduce premature degradation, and increase the efficiency of protein trafficking through the cell.  The use of pharmacological chaperones may be broadly applicable to diseases where an increase in the activity of a specific protein may provide therapeutic benefit.  

About Pharmacological Chaperones for Parkinson's Disease

Mutations in the GBA1 gene are the most common genetic risk factor known for Parkinson's disease.  Those at increased risk include individuals who inherit only one mutant copy of GBA1, while the other copy is normal.  GBA1 mutations reduce the activity of the lysosomal enzyme glucocerebrosidase (GCase), which leads to Gaucher disease when two mutant copies of the gene are inherited.  Increasing the activity of GCase in the brain with a small-molecule pharmacological chaperone may reduce the negative consequences of carrying GBA1 mutations.  Amicus and collaborators have previously  demonstrated that administration of the pharmacological chaperone AT2101 to mice that were genetically engineered to over-produce -synuclein increased the activity of brain GCase, prevented accumulation of α-synuclein in the brain, and improved motor function as assessed in various behavioral tests.  Through a significant medicinal chemistry effort, Amicus has identified new pharmacological chaperones that improve significantly on the properties of AT2101 and that have better potential for clinical development. 

About Parkinson's Disease

Parkinson's disease is a progressive neurological disease that affects over 1 million people in the US.  It is most commonly associated with changes in motor function such as tremor, rigidity, slowness of movement, and postural instability, but also affects cognition and peripheral functions.  The prevalence of Parkinson's increases above 60 years of age and thus the patient population is expected to grow in the next several decades.

About the Michael J. Fox Foundation for Parkinson's Research

Founded in 2000, MJFF is dedicated to finding a cure for Parkinson's disease through an aggressively funded research agenda and to ensuring the development of improved therapies for those living with Parkinson's today. The Foundation has funded more than $221 million in research to date. For more information, please visit

About Amicus Therapeutics

Amicus Therapeutics is developing orally administered, small molecule drugs called pharmacological chaperones, a novel, first-in-class approach to treating a broad range of diseases including lysosomal storage disorders and neurodegenerative diseases.  Amicus' lead program is in Phase 3 for Fabry disease. For more information about Amicus please visit

Forward-Looking Statements

This press release contains, and the accompanying conference call will contain, "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 relating to preclinical development of Amicus' candidate drug products.  Words such as, but not limited to, "look forward to," "believe," "expect," "anticipate," "estimate," "intend," "plan," "targets," "likely," "will," "would," "should" and "could," and similar expressions or words identify forward-looking statements. Such forward-looking statements are based upon current expectations that involve risks, changes in circumstances, assumptions and uncertainties. The inclusion of forward-looking statements should not be regarded as a representation by Amicus that any of its plans will be achieved. Any or all of the forward-looking statements in this press release may turn out to be wrong. They can be affected by inaccurate assumptions Amicus might make or by known or unknown risks and uncertainties. For example, with respect to statements regarding the potential goals, progress, timing and results of preclinical studies, actual results may differ materially from those set forth in this release due to the risks and uncertainties inherent in the business of Amicus, including, without limitation: the potential that results of pre-clinical studies indicate that the product candidates are unsafe or ineffective; the potential that preclinical studies could be delayed because we identify serious side effects or other safety issues; the potential that we will need additional funding to complete all of our studies and, our dependence on third parties in the conduct of our clinical studies. Further, the results of earlier preclinical studies and/or clinical trials may not be predictive of future results. In addition, all forward looking statements are subject to other risks detailed in our Annual Report on Form 10-K for the year ended December 31, 2009. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement, and Amicus undertakes no obligation to revise or update this news release to reflect events or circumstances after the date hereof. This caution is made under the safe harbor provisions of Section 21E of the Private Securities Litigation Reform Act of 1995.




SOURCE Amicus Therapeutics
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