Press Release

Shire expands its Human Genetic Therapies pipeline through in-licensing agreement with Amicus Therapeutics

08 Nov 2007

  • Acquires ex-US rights for AMIGAL™, PLICERA™ and AT2220
  • Oral therapies based on novel chaperone technology for Lysosomal Storage Disorders

Basingstoke, UK and Cambridge, MA – November 8, 2007 – Shire plc (LSE: SHP, NASDAQ: SHPGY, TSX: SHQ), the global specialty biopharmaceutical company, announced today that it has licensed from Amicus Therapeutics, Inc. (NASDAQ: FOLD) the rights to three compounds in markets outside of the United States: AMIGAL™ (migalastat hydrochloride) for Fabry disease (phase 2), PLICERA™ (isofagomine tartrate) for Gaucher disease (phase 2) and AT2220 (deoxynojirimycin) for Pompe disease (phase 1).

Matthew Emmens, Chief Executive of Shire comments:

“Amicus’ pharmacological chaperone compounds have the potential to be an excellent addition to our current enzyme replacement therapy business, which includes REPLAGAL™ for Fabry disease, ELAPRASE™ for Hunter syndrome and GA-GCB in phase 3 development for Gaucher disease. In addition, it provides an opportunity for Shire to enter the market for Pompe disease. This technology should provide significant benefit to patients with these serious genetic diseases.”

Sylvie Gregoire, President of Shire Human Genetic Therapies adds:

"We are excited about this opportunity to add to our already well established therapeutic platform and we look forward to working closely with Amicus on the development of these new therapies."

John F. Crowley, Amicus’ President & CEO said:

“We are immensely pleased to enter into this partnership with Shire, which leverages both companies’ unique experience and expertise in developing therapies for lysosomal storage disorders. The combination of Amicus’ strong science foundation in pharmacological chaperones and Shire’s proven track record in drug development and commercialization will greatly enhance our efforts to bring these novel therapies to patients.”

The pharmacological chaperone technology, which will be available as an oral therapy, 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, leading to improved protein folding and trafficking, and increased activity.

Financial terms of the license are geared to the successful development and commercialization of the products. Shire will pay Amicus an upfront license fee of million, and development and sales-based milestones totalling up to 0MM. Shire will also pay royalties on net sales of the products, with tiered, double digit royalty rates. The companies will pursue a joint development program toward market approval in the U.S. and Europe; expenses for this program will be shared 50:50.

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