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.
Latest Press Releases
- Shire delivers strong quarter driven by 3m of new product sales
- Jerini receives european commission approval for FIRAZYR® (Icatibant) in HAE treatment
- Shire to add new orphan drug to its HGT portfolio - EU launch imminnent
- Graham Hetherington appointed Chief Financial Officer and Board Director at Shire
- First and Only Treatment for Hunter Syndrome Now Approved in 40 Countries Worldwide
- Results of Court Meeting and Extraordinary General Meeting - Shareholders Approve Scheme of Arrangement and New Share Plans
- Shire welcomes the Court of Appeal's decision that NICE review of Alzheimer's drugs was procedurally unfair
- Publication of Supplementary Prospectus
- Shire enhances its orphan drug pipeline with the acquisition of a new clinical candidate for Metachromatic Leukodystrophy
- FDA Approves VYVANSE (TM) (lisdexamfetamine dimesylate), the First and Only Once-Daily Prodrug Stimulant to Treat ADHD in Adults

