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Amicus Therapeutics

Company Profile

Amicus Therapeutics is a biopharmaceutical company focused on the discovery, development and commercialization of novel small molecule, orally administered drugs known as pharmacological chaperones, for the treatment of a range of human genetic diseases. 

Amicus is focused on three key strategic priorities:

  • The Phase 3 development of its lead program, AT1001 (migalastat HCl) for the treatment of Fabry disease;
  • The advancement of pharmacological chaperones used in combination with enzyme replacement therapies (ERT) for the treatment of lysosomal storage diseases; and
  • The advancement of its lead pre-clinical programs that utilize its pharmacological chaperone technology for the treatment of diseases of neurodegeneration.

A Phase 3 study intended to support approval of migalastat HCl in the United States (Study 011) commenced in the second quarter of 2009 and treatment of the first patient began in the fourth quarter of 2009.  The Company expects to complete enrollment by the end of 2010 and to have preliminary results from this study in mid-2011.   Additionally, Amicus expects to commence a separate Phase 3 study (Study 012) intended to support approval in the European Union before year end.

Amicus continues to advance its program evaluating the use of pharmacological chaperones in combination with enzyme replacement therapy (ERT) as an expansion of the chaperone technology platform. The Company has selected migalastat HCl as its first clinical candidate and plans to initiate a Phase 2 study with migalastat HCl in combination with ERT before the end of 2010.  Additionally, the Company is evaluating options to advance chaperone-ERT combination therapy programs for Pompe disease and Gaucher disease.   

Amicus has an advanced pre-clinical program utilizing its pharmacological chaperone technology for the treatment of Parkinson’s disease with unique molecules, a unique target and what has the potential to be a very ground breaking approach to treating this devastating disease.  The Company has established initial proof-of-concept for the use of its pharmacological chaperones in animal models of Parkinson’s disease and continues to advance this preclinical program.  Amicus’ second lead preclinical program using the pharmacological chaperone approach for diseases of neurodegeneration is for the treatment of Alzheimer’s disease.  The Company expects to complete initial proof-of-concept studies during 2010 and report data in the second half of 2010.

The Company has broad, issued intellectual property around the methods, use, and composition of pharmacological chaperones to treat genetic diseases and diseases of neurodegeneration. In addition to the currently issued patents, Amicus has several patent applications pending.

Since it’s inception in 2002, Amicus has assembled a world-class leadership team with proven expertise in drug discovery, development and commercialization.   The Company completed an Initial Public Offering in May 2007 and trades on the NASDAQ National Market under the Ticker Symbol:  FOLD.   Amicus’ headquarters is located in Cranbury, NJ and has a research facility in San Diego, CA.