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Amicus Therapeutics Presents Positive Data Update From Phase 2 Extension Study of Amigal(TM) for Fabry Disease
Date:2/11/2010

e successful.  Our focus is the completion of enrollment in Study 011 this year and the commencement of Study 012.  We are also very encouraged by the results of our preclinical studies of the chaperone-ERT combination approach as well as the advancements with our Parkinson's program.  We look forward to moving these applications of our technology forward in 2010."

Preclinical Chaperone-ERT Combination Therapy Data

Today Amicus presented data from preclinical studies that evaluated the combination of migalastat HCl and ERT and AT2220 and ERT in mouse models of Fabry and Pompe disease, respectively.  Studies of both combinations demonstrated that co-administration of the chaperone with ERT resulted in prolonged half-life of ERT in the circulation, increased enzyme activity in cells and greater substrate reduction in target tissues compared to that seen with ERT alone.  

Preclinical Parkinson's Disease Data

Amicus also presented data from preclinical studies that evaluated the chaperone AT2101 in mouse models of Parkinson's disease. The studies demonstrated that treatment with AT2101 increased the activity of beta-glucocerebrosidase (GCase), prevented accumulation of alpha-synuclein in the brain and improved motor function as assessed in various behavioral tests.  The Company also reported that new compounds have been identified that improve on the properties of AT2101 and expand the range of doses and regimens that show motor improvement in mouse models of the disease.  

About Fabry Disease

Fabry disease is a lysosomal storage disorder caused by inherited genetic mutations in the GLA gene, which results in deficient activity of the enzyme alpha-galactosidase A (alpha-Gal A). Deficient alpha-Gal A activity leads to lysosomal accumulation of globotriaosylceramide (GL-3), which is believed to cause the various symptoms of Fabry di
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Related medicine technology :

1. Amicus Therapeutics Commences Phase 3 Trial Evaluating Amigal(TM) for the Treatment of Fabry Disease
2. Amicus Therapeutics Announces Issuance of New Plicera(TM) Composition of Matter Patent
3. Amicus Therapeutics Suspends Enrollment for Phase 2 Clinical Trial of AT2220 for Pompe Disease
4. Amicus Therapeutics Announces Positive Progress of Three Lead Clinical Programs and General Outlook for 2009
5. Amicus Therapeutics Announces Second Quarter 2008 Financial Results
6. Amicus Therapeutics Announces Successful Completion of End of Phase 2 Meeting With FDA for Amigal in Fabry Disease
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10. Amicus Therapeutics Announces Positive Results From Two Phase 1 Clinical Studies of AT2220 for Pompe Disease
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