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Plicera

Amicus Therapeutics, Inc.

Drug Names(s): HGT 3410, AT2101, isofagomine tartrate, afegostat tartrate

Description: AT2101 is designed to act as a pharmacological chaperone by selectively binding to the misfolded enzyme responsible for Gaucher disease, b-glucocerebrosidase (GCase). After binding to the enzyme, it is thought that AT2101 promotes the proper folding, processing, and trafficking of the enzyme from the endoplasmic reticulum to its final destination, the lysosome, the area of the cell where the enzyme does its work. Once it reaches the lysosome, the pharmacological chaperone is displaced and the enzyme can perform its normal function, which is the breakdown of its natural substrate, glucocerebroside.

Deal Structure: In November 2007, Amicus Therapeutics announced that it entered into a strategic collaboration with Shire Human Genetic Therapies, a subsidiary of Shire plc, to jointly develop Amicus' three pharmacological chaperone compounds for lysosomal storage disorders. Shire will receive rights to commercialize these products outside of the United States. Amicus will retain all rights to commercialize these products in the United States. The collaboration includes Amigal (migalastat hydrochloride), Plicera (isofagomine tartrate), and AT2220 (deoxynojirimycin).

Under the terms of the deal, Amicus will receive an initial, non-refundable licensing payment of US$ 50 million. Joint development costs toward global approval of the three compounds will be shared 50/50 going forward, and Amicus is eligible to receive an additional US$ 150 million if certain clinical and regulatory milestones are met for the three programs through approvals. Amicus is also eligible to receive up to US$ 240 million...See full deal structure in Biomedtracker


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