Press Releases


September 18, 2003 at 12:00 AM EDT

New Brunswick, NJ - Amicus Therapeutics, Inc., an emerging drug development company focused on the development of a novel therapeutic approach to the treatment of human genetic disorders, with an initial focus on lysosomal storage diseases, announced today the issuance of U.S. Patent number 6,599,919. This marks Amicus' fourth U.S. Patent for its novel method of restoring normal function to mutant proteins with therapeutic pharmacological chaperones. This patent serves to consolidate the areas of coverage provided by the Company's existing patent portfolio, which includes claims directed to treating both mutant and non-mutant enzymes, as well as broad intellectual property related to treating all lysosomal storage diseases.

"With the issuance of this broad patent we have secured our commercial position and our ability to dominate the field of pharmacological chaperone therapy to rescue any and all mutant enzymes." said Norman Hardman, Ph.D., Chief Executive Officer of Amicus Therapeutics, Inc. "Amicus was founded on the belief that pharmacological chaperones present a superior small molecule approach to the treatment of genetic disorders that result from protein misfolding, and provide an effective alternative to enzyme replacement therapy. Our therapeutic products have the ability to expand the market by offering treatment to a broader range of patients because of their oral delivery, high selectivity for disease protein, and greater suitability for long term-chronic therapy. With our consolidated patent portfolio we are optimally positioned to navigate a wealth of drug development and collaboration opportunities."

The newly issued patent covers the method for rescuing the activity of all mutant enzymes with pharmacological chaperones, completing a portfolio of patents that includes U.S. Patent 6,583,158, which covers the method for enhancing mutant enzyme activities in lysosomal storage disorders, U.S. Patent 6,589,964, covering claims for the enhancement of wild-type enzymes, and Patent 6,274,597, describing methods for enhancing lysosomal alpha-Galactosidase A. Amicus' intellectual property estate includes these four issued U.S. patents, and a related family of five pending applications, all of which are licensed exclusively from the Mount Sinai School of Medicine.

About Amicus Therapeutics

Amicus was established in April 2002 with seed financing provided by CHL Medical Partners II, a venture capital fund managed by Collinson, Howe & Lennox, and is focused on the development of orally-active, small molecule drugs capable of restoring normal function to mutant proteins. Amicus was founded to capitalize on the discovery that many diseases of genetic origin are caused by missense mutations and other rescuable mutations that result in the misfolding of a protein/enzyme. These misfolded mutant proteins become targeted for degradation before reaching their normal site of action, leading to the disease phenotype. Pharmacological Chaperones are designed to help the mutant protein fold correctly into its normal 3-dimensional conformation, restoring the normal processing and transport of the protein and rescuing its intrinsic biological activity and function.

Amicus technology is based on research conducted by Jian-Qiang Fan, Ph.D., Assistant Professor, Department of Human Genetics at Mount Sinai School of Medicine, and a founder of Amicus. Amicus' pharmacological chaperone approach has the potential to be applied to a wide range of genetic disorders. The Company's initial focus is on lysosomal storage disorders and its first compound, AT1001 for Fabry disease, is in late-stage preclinical development. The Company anticipates filing an IND for AT1001 by the first quarter of 2004. Amicus currently has 10 employees and is headquartered at the New Jersey Technology Center in North Brunswick, New Jersey. Additional information about the Company can be found at