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April 24, 2008 Update - Shire Acquires Metazym from Zymenex
Shire HGT has acquired Metazym from Zymenex for $135M. Shire will continue to expedite the testing and market release of Metazym. Shire can and will likely apply significant corporate resources to these later stage tests and an expedited market release.
Metazym is a promising enzyme replacement therapy (ERT) for MLD.
This is exciting for MLD affected families. Shire can and will likely apply significant corporate resources to these later stage tests and an expedited market release. In addition, this external endorsement of Metazyme seems to validate the effectiveness of this ERT in advance of seeing published Phase II European clinical trial results.
Metazym is nearing completion of Phase II clinical trials in Europe. An IND has been granted by the FDA to prepare for Phase II clinical trials in the US. In addition, Orphan Drug Status has been granted in both Europe and the US for Metazym. Additional details on the status of Metazym can be seen on the Zymenex page on this site.
The Shire press release can be seen here.
We will post more details as they come available.
Overview - Shire's Enzyme Replacement Therapy
[Please note this information from 2006 refers to Shire's internally developed ERT. With the Metazym acquisition it is not clear when or how this ERT will be brought to market]
Shire HGT has shown a proof of concept with mice for an enzyme replacement therapy (ERT). They are in the pre-clinical stage of development.
A natural history study and patient registry are currently in the planning stages, with the goal of beginning patient enrollment by the end of 2007. These studies will help scientists to better understand disease progression and pathology in MLD, and will be used to inform the design of future clinical trials.
The MLD Foundation is under non-disclosure agreement with Shire HGT and agreed at a private meeting in March 2007 to work closely with Shire to help facilitate the registry a
nd their pre-clinical efforts, including surveying and contacting MLD affected families. When clinical trial recruitment starts in 2008 we will assist with that as well.
Shire is investigating several methods of dosing, intrathecal and intravenous, so both the central and peripheral nervous systems receive the proper dosing of the enzyme.
Existing FGE Products
Shire has demonstrated successes with its proprietary technologies, including human cell line, gene activation and Formylglycine Generating Enzyme (FGE). During 2006, Shire's HGT division posted revenue growth of over 30%, mainly as a result of the approval and launch in the US of ELAPRASE, a treatment for Hunter Syndrome, and the continued worldwide roll-out of REPLAGAL, their ERT therapy for the treatment of patients with Fabry disease.
Their ERT product pipeline consists of ERT therapies for Gaucher disease, Sanfilippo Syndrome (dog & mouse successes), and Metachromatic Leukodystrophy (mouse proof's of concept). They are adding a new condition every 12 months to the pipeline and expect approximately 18 months for each to be developed. Their GA-GCB enzyme for the treatment of Type 1 Gaucher disease started recruitment for Phase 3 clinical trials commenced in Q1 2007.
HGT ERT for MLD
Shire HGT is developing an investigational ERT to replace the deficient or absent arylsulfatase A (ASA) for the treatment of Metachromatic Leukodystrophy (MLD). The company is using its proprietary FGE technology to create highly productive human cell lines for the manufacture of sulfatases. Co-expression of FGE with a sulfatase leads to significantly increased enzymatic activity, in vitro, as compared to sulfatases produced without this technology. Shire HGT’s MLD program is currently in preclinical development.
Shire is not yet recruiting for clinical trials - they are not scheduled to start until 2008 or later. The natural history and registry project will start recruiting later in 2007. Shire's May 10th 2007 presentation to a UK analyst shows their MLD theraphy contributing to Shire's 2014 revenues as a released product . There is a lot of reserach and numerous trials to be performed int hemeantime.
Please be sure to sign up for the MLD Foundation's Announcement e-mail list to be kept abreast of progress by Shire and other research.
The Shire HGT contact is Carol Cannon (Medical Affairs): ccannon@shire.com
Shire Human Genetic Therapies (Shire HGT), is a business unit of Shire plc, a global specialty pharmaceutical company. Metachromatic leukodystrophy (MLD):
Shire HGT specializes in researching, developing and commercializing therapeutics primarily for the treatment of genetic diseases caused by protein deficiencies. We have a high quality product portfolio and a relatively low risk, well-balanced development pipeline, especially in the area of enzyme replacement therapies. These biologics products generally have significant periods of market exclusivity resulting either from orphan drug designation or patent protection.
Shire HGT is based in Boston, MA, and has 600 employees in 16 countries around the globe.
June 11, 2007
Shire Human Genetic Therapies (Shire HGT), a business unit of Shire plc, a global specialty pharmaceutical company, is focused on developing innovative therapies for life-threatening genetic diseases. Shire HGT applies its proprietary technologies, including human cell line, gene activation and Formylglycine Generating Enzyme (FGE), to the discovery, development and manufacturing of protein therapies to treat genetic diseases. With over 600 employees in 16 countries around the globe, Shire HGT is dedicated to providing hope for patients and their families, for better, healthier lives.
Shire HGT is developing an investigational ERT to replace the deficient or absent arylsulfatase A (ASA) for the treatment of Metachromatic Leukodystrophy (MLD). The company is using its proprietary FGE technology to create highly productive human cell lines for the manufacture of sulfatases. Co-expression of FGE with a sulfatase leads to significantly increased enzymatic activity, in vitro, as compared to sulfatases produced without this technology. Shire HGT’s MLD program is currently in preclinical development. A natural history study and patient registry are currently in the planning stages, with the goal of beginning patient enrollment by the end of 2007. These studies will help scientists to better understand disease progression and pathology in MLD, and will be used to inform the design of future clinical trials.
For more information about the program, please contact Carol Cannon at Shire HGT Medical Affairs: ccannon@shire.com
2006 Shire HGT Financial Report