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Amicus Therapeutics Announces Fourth Quarter and Full Year 2007 Financial Results & Program Advancements
Date:1/29/2008

r of 2007, Amicus successfully completed a series of Phase I clinical studies of AT2220 in healthy volunteers. AT2220 is an orally available small-molecule pharmacological chaperone targeting the enzyme deficient in people living with Pompe disease. These studies demonstrated that AT2220 was generally safe and well-tolerated at all doses tested and that there were no drug-related serious adverse events. Amicus expects to report on these data at the ACMG conference in March along with the results of an additional clinical ex vivo response study designed to test the responsiveness of various Pompe mutations to AT2220.

"The data from these Phase 2 trials of Amigal and Plicera are part of an expanding body of scientific and clinical evidence that we are building through rigorous studies to demonstrate that many patients with Fabry, Gaucher and Pompe disease may be successfully switched from ERT to a pharmacological chaperone," noted John Crowley.

Other Protein Folding and Stabilization Programs:

In 2008, Amicus expects to accelerate its investments in research and development to better understand the potential for using pharmacological chaperones to treat a range of human genetic diseases that could benefit from improved protein folding and stabilization.

The company's advanced pre-clinical program in Parkinson's disease, funded in part by a grant from the Michael J. Fox Foundation, will continue with the potential to initiate clinical studies in 2008. In the fourth quarter of 2007, the company announced pre-clinical results which further support a biochemical link between the GCase enzyme and the accumulation of alpha- synuclein, a protein believed to play a key role in Parkinson's disease. Furthermore, in an animal model Amicus showed preliminary pre-clinical results that for the first time demonstrated that administration of a pharmacological chaperone can lead to reduction of alpha-synuclein aggregates in the brain. These results were fir
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SOURCE Amicus Therapeutics
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