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Amicus Therapeutics Presents Data From Preclinical and Phase 1,Studies of Amigal for Fabry Disease

Studies in Mice Show Reduction of Enzyme Substrate

CRANBURY, N.J., March 19, 2007 /PRNewswire/ -- Amicus Therapeutics, a biopharmaceutical company developing small-molecule, orally administered pharmacological chaperones for the treatment of human genetic diseases, announced today that the Company will present results from studies of Amigal(TM) (migalastat hydrochloride, AT1001), Amicus' compound in development for the treatment of Fabry disease, at the of Medical Genetics (ACMG) Annual Meeting from March 21-25 in Nashville, TN. This presentation will include the first data from the Company demonstrating the reduction of globotriaosylceramide (GL-3), the lipid substrate that accumulates in Fabry disease, after oral administration of a pharmacological chaperone. These data were not available at the time of abstract submission but will be included in the presentation at the meeting.

Amigal is designed to selectively bind to and stabilize alpha- galactosidase A (alpha-GAL), the enzyme deficient in Fabry disease. This deficiency leads to lysosomal accumulation of GL-3, which is believed to cause the various symptoms of Fabry disease. Amigal facilitates proper trafficking of the enzyme to the lysosomes, the compartments in the cell where it is needed to break down GL-3.

At the ACMG meeting, Amicus scientists will present data from several studies that examined the in vitro and in vivo effects of Amigal in cell lines, mice and healthy volunteers. Among the key findings:

    - In vitro exposure to Amigal increased the level of alpha-GAL in cells

      derived from healthy volunteers and from Fabry patients.


    - Oral administration of Amigal resulted in a dose-dependent increase in

      alpha-GAL levels in various tissues of normal mice and Fabry mice

      genetically modified to produce alpha-GAL with a human missense

      mutation.


    - Oral administration of Amigal to healthy vo
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