BASINGSTOKE, England and CAMBRIDGE, Massachusetts, April 24
- Shire plc (LSE: SHP, NASDAQ: SHPGY), the global
specialty biopharmaceutical company, announces the acquisition of
arylsulfatase -A (ASA) an Enzyme Replacement Therapy (ERT) in Phase 1-2
clinical trials for Metachromatic Leukodystrophy (MLD) from the Danish
company Zymenex A/S (Zymenex).
MLD is a serious, life-limiting disease in which patients experience
progressive irreversible neurological damage. MLD is caused by a deficiency
in the enzyme ASA which causes an excess concentration of sulphatide in
cells and an ensuing breakdown of myelin. There are approximately 2,000 MLD
patients in developed world markets(1).
The newly acquired ASA product, currently known as METAZYM(TM), has
completed a Phase Ib clinical trial in 12 MLD patients in Europe and an
extension to this study is ongoing. The product has received Food and Drug
Administration (FDA) approval for its Investigational New Drug (IND)
application to initiate a phase 2 clinical trial and has been granted
Orphan Drug Designation in the United States and in the European Union.
Sylvie Gregoire, President of Shire’s Human Genetic Therapies business,
commented:
“This product fits very well with Shire’s ERT portfolio of treatments
for Lysosomal Storage Disorders (LSD). Shire HGT has been committed to MLD
and by acquiring this mid-stage clinical program we hope to bring a MLD
treatment to patients two years earlier than anticipated.”
Shire is making a payment to Zymenex of US$135 million for the
acquisition of global rights to the product upon completion of the
transaction, which is conditional upon the receipt of customary consents.
Zymenex is also providing certain transition services, including know-how
transfer, for up to six months after completion. The transaction includes
no royalties or other downstream payment obligations.
(1) Scriver et al 1995
Notes to editors
Disease Background
Metachromatic Leukodystrophy (MLD) is in the family of lysosomal
storage diseases (LSD’s). MLD is an autosomal recessive disease caused by a
deficiency of the lysosomal enzyme arylsulfatase A (ASA) which results in
an increased concentration of sulphatide in cells of the brains and in
non-neural tissue, such as the kidneys and gallbladder. When these sulfated
glycolipids accumulate in the brain, they cause a breakdown of myelin, a
substance that protects the nerves in the brains and in the rest of the
body. This breakdown is what makes MLD a progressive, neurodegenerative
disease.
Symptoms and Patient Outlook
Sulfatide accumulation in the central and peripheral nervous system
leads to destruction of the myelin sheath (demyelination). MLD has a
spectrum of disease which can arise in infants and young children with a
range of symptoms, though most are related to motor and cognitive decline.
Most MLD patients are normal at birth but often die before age 20, with
some patients within the first few years of life. During the final stages
all patients reach a decerebrate, vegetative state. The majority of cases
are late infantile or juvenile patients. The number of adult patients
exhibiting mild forms of MLD is unknown, as adult onset MLD can present
with symptoms similar to psychosis.
SHIRE PLC
Shire’s strategic goal is to become the leading specialty
biopharmaceutical company that focuses on meeting the needs of the
specialist physician. Shire focuses its business on attention deficit and
hyperactivity disorder (ADHD), human genetic therapies (HGT),
gastrointestinal (GI) and renal diseases. The structure is sufficiently
flexible to allow Shire to target new therapeutic areas to the extent
opportunities arise through acquisitions. Shire’s in-licensing, merger and
acquisition efforts are focused on products in niche markets with strong
intellectual property protection either in the US or Europe. Shire believes
that a carefully selected portfolio of products with strategically aligned
and relatively small-scale sales forces will deliver strong results.
For further information on Shire, please visit the Company’s website:
http://www.shire.com.
“SAFE HARBOR” STATEMENT UNDER THE PRIVATE SECURITIES LITIGATION REFORM
ACT OF 1995
Statements included herein that are not historical facts are
forward-looking statements. Such forward-looking statements involve a
number of risks and uncertainties and are subject to change at any time. In
the event such risks or uncertainties materialize, Shire’s results could be
materially affected. The risks and uncertainties include, but are not
limited to, risks associated with: the inherent uncertainty of
pharmaceutical research, product development including, but not limited to
the successful development of JUVISTA(R) (Human TGFbeta3) and veleglucerase
alfa (GA-GCB); manufacturing and commercialization including, but not
limited to, the establishment in the market of VYVANSE(TM)
(lisdexamfetamine dimesylate) (Attention Deficit and Hyperactivity Disorder
(“ADHD”)); the impact of competitive products, including, but not limited
to, the impact of those on Shire’s ADHD franchise; patents, including but
not limited to, legal challenges relating to Shire’s ADHD franchise;
government regulation and approval, including but not limited to the
expected product approval date of INTUNIV(TM) (guanfacine extended release)
(ADHD); Shire’s ability to secure new products for commercialization and/or
development; and other risks and uncertainties detailed from time to time
in Shire plc’s filings with the Securities and Exchange Commission,
including Shire plc’s Annual Report on Form 10-K for the year ended
December 31, 2007.
SOURCE Shire PLC