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GlaxoSmithKline and Amicus to co-develop Amigal drug for Fabry disease

Last updated: 18:07 01 Nov 2010 AEDT, First published: 19:07 01 Nov 2010 AEDT

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GlaxoSmithKline (LON:GSK, NYSE:GSK) and Amicus Therapeutics (Nasdaq:FOLD) said Friday they agreed to jointly develop and commercialize Amicus' Amigal, a drug currently in phase 3 trials for the treatment of Fabry disease, a rare inherited enzyme deficiency disorder.

Under the terms of the deal, GlaxoSmithKline will receive an exclusive worldwide license to develop, manufacture and commercialize the drug, in exchange for Amicus receiving an upfront payment of US$30 million. Amicus is also eligible to receive further payments of approximately US$170 million upon achieving certain milestones, as well as tiered double-digit royalties on global sales of the product.

Both companies have agreed to jointly fund development costs and to advance the clinical studies of Amigal to explore the co-administration of it with enzyme replacement therapy.

In addition, as part of the collaboration, GlaxoSmithKline is purchasing 6.9 million shares of Amicus common stock at a price of US$4.56 per share, for a total value of US$31 million, representing a near 20% stake.

Fabry disease is an inherited lysosomal storage disorder caused by deficiency of an enzyme called alpha-galactosidase A. The role of this enzyme within the body is to break down a complex lipid and reduced or absent levels leads to the accumulation of the lipid in effected tissue, including the central nervous system, heart, kidneys, and skin. Symptoms of the disease include pain, kidney failure, and increased risk of heart attack and stroke.

Amigal is designed to bind to and stabilize the target enzyme, which facilitates proper trafficking of the enzyme to the lysosomes, where it is needed to break down the lipid. Amicus said that results from phase 2 studies have indicated increased levels of the alpha-Gal A enzyme and reduced levels of the complex lipid, as measured from kidney biopsies and in urine, with potential positive impact on renal function.

Currently, the drug is in a phase 3 study, which began in the second quarter of last year, using 60 patients in approximately 40 investigational sites around the world. Subjects being enrolled are Fabry patients who have never received enzyme replacement therapy, or who have not received it for at least six months. Preliminary results are expected to be announced in the second half of 2011.

"With this key strategic alliance with GSK and the added financial strength it provides, Amicus is now uniquely positioned to build shareholder value through our expertise in rare disease drug development," said chairman and CEO of Amicus, John F. Crowley.

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