Amicus Therapeutics is pioneering a paradigm-shifting approach to treating a range of human genetic diseases in which crucial proteins are misfolded, thus reducing or eliminating their functionality.

Amicus develops pharmacological chaperones - oral therapies that bind to and stabilize the affected proteins, restoring their shape, proper trafficking, and biological activity. This novel, next-generation approach offers the potential to improve treatment options for patients with genetic diseases. (Information from www.amicustherapeutics.com).

Plicera(tm)
Amicus Therapeutics Announces Preliminary Results of Phase 2 Study with Plicera(tm) for Gaucher Disease.

CRANBURY, N.J., Oct 02, 2009 /PRNewswire-FirstCall via COMTEX News Network/ -- Amicus Therapeutics (Nasdaq: FOLD) today announced preliminary results from its Phase 2 randomized, open-label study to assess the safety, tolerability and preliminary efficacy of its investigational drug, Plicera(TM) (afegostat tartrate), in treatment-naive adult patients with type 1 Gaucher disease. Two dose regimens of Plicera (225 mg three days on/four days off and seven days on/seven days off) were studied during this six month trial. While all patients enrolled experienced an increase in the level of the target enzyme (GCase) as measured in white blood cells, clinically meaningful improvements in key measures of disease were observed in just one of the eighteen patients who completed the study. The preliminary results suggest that treatment with Plicera was generally well tolerated, with no serious adverse events (SAEs) reported. Nineteen subjects were enrolled and 18 subjects completed the study. One subject discontinued treatment because of an adverse event (conjunctivitis-related symptoms). Once the data are final, the Company plans to further analyze and evaluate the results in collaboration with its partner, Shire Human Genetic Therapies, Inc. (Shire HGT), and, based on this work, will determine the appropriate next steps for the Plicera program. However, based on these preliminary results, the Company does not expect to advance Plicera into Phase 3 development at this time.

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For information on clinical trials, go to www.amicustherapeutics.com or www.clinicaltrials.gov