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Amicus Therapeutics Completes Phase 2 Enrollment
or Amigal™ for Fabry Disease
Cranbury, NJ – March 16, 2007
Amicus Therapeutics, a biopharmaceutical company developing
small-molecule, orally administered pharmacological chaperones for the
treatment of human genetic diseases, announced today that enrollment has
been completed for all ongoing Phase 2 clinical trials of Amigal™ (migalastat
hydrochloride, AT1001). Amigal is in development for the treatment of
Fabry disease.
The four open-label, multi-national Phase 2 trials are examining various
dose levels and frequencies of Amigal in men and women with Fabry
disease. The primary objective of the studies is to evaluate the safety
and tolerability of treatment with Amigal. The secondary objective is to
evaluate certain pharmacodynamic measures of treatment, including
effects on a-galactosidase A (a-GAL) and globotriaosylceramide (GL-3)
levels in various cells and tissues of disease. An additional objective
is the preliminary assessment of cardiac, renal and central nervous
system function. The results of these clinical trials are expected to be
available by the end of 2007.
Amigal is designed to selectively bind to and stabilize a-GAL, the
enzyme deficient in Fabry disease. This deficiency leads to lysosomal
accumulation of GL-3, which is believed to cause the various symptoms of
Fabry disease. Amigal facilitates proper trafficking of the enzyme to
the lysosomes, the compartments in the cell where it is needed to break
down GL-3.
About Fabry Disease
Fabry disease is a lysosomal storage disorder caused by inherited
genetic mutations in the GLA gene, which result in deficient activity of
the enzyme a-galactosidase A (a-GAL). Deficient a-GAL activity leads to
lysosomal accumulation of globotriaosylceramide (GL-3), which is
believed to cause the various symptoms of Fabry disease, including pain,
kidney failure and increased risk of heart attack and stroke. Fabry
disease is estimated to affect approximately 5,000 to 10,000 people in
the developed world, but recent evidence suggests that the disease may
be significantly underdiagnosed. The U.S. Food and Drug Administration's
Office of Orphan Products Development has granted orphan designation for
Amigal in the United States, and the European Commission has designated
Amigal as an orphan medicinal product in the European Union.
About Amicus Therapeutics
Amicus Therapeutics is a biopharmaceutical company developing novel,
oral therapeutics known as pharmacological chaperones for the treatment
of a range of human genetic diseases. 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. Amicus is initially targeting
lysosomal storage disorders, which are severe, chronic genetic diseases
with unmet medical needs. Amicus is currently conducting Phase 2
clinical trials for its two lead compounds, Amigal™ for Fabry disease,
and AT2101 for Gaucher disease. The company is currently conducting
Phase 1 trials with AT2220 for the treatment of Pompe disease.
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