Company is paying an initial in-licensing fee of $50 million for one early-stage and two mid-stage compounds.

Shire is expanding its pipeline of therapeutics for rare genetic diseases through a licensing deal with Amicus Therapeutics that could be worth up to $440 million. The three Amicus candidates are based on chaperone technology, which will augment Shire’s enzyme-replacement therapies.

Shire will pay $50 million upfront for three compounds in markets outside of the United States: Phase II Amigal™ for Fabry disease, Phase II Plicera™ for Gaucher disease, and Phase I AT2220 for Pompe disease.

Phase II data for Amigal and preliminary Phase II results for Plicera will be available by year end, according to Matt Patterson, COO at Amicus.  For AT2220, the firm plans to start a Phase II study early in 2008, he adds. 

Based on development achievements through to approval of these compounds, Patterson says that Amicus is eligible to receive $150 million. The company may also get another $240 million in sales milestones. Shire will also make tiered, double-digit royalty payments on net sales.

The companies will jointly pursue a development program toward market approval in the U.S. and Europe and share expenses equally.

The pharmacological chaperone technology, which the firms expect to make available as oral therapies, has been applied to various enzymes that are defective as a result of improper folding. In contrast to the traditional enzyme-replacement approach, pharmacological chaperone technology involves the use of small molecules that selectively bind to and stabilize proteins in cells. This reportedly leads to improved protein folding and trafficking as well as increased activity.

“Amicus’ pharmacological chaperone compounds have the potential to be an excellent addition to our current enzyme-replacement therapy business,” states Shire CEO, Matthew Emmens. Shire’s pipeline includes enzyme-replacement treatments Replagal™ for Fabry disease and GA-GCB in Phase III development for Gaucher disease. “In addition,” continues Emmens, “it provides an opportunity for Shire to enter the market for Pompe disease.”

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