Amicus Therapeutics, a
biopharmaceutical company developing small molecule, orally-administered
pharmacological chaperones for the treatment of a range of human genetic
diseases, announced today that it will present positive results from its
recently completed Phase 1 clinical studies of Plicera(TM) (isofagomine
tartrate, AT2101) for Gaucher disease at the American College of Medical
Genetics (ACMG) Annual Meeting on March 21-25 in Nashville, TN. The Phase 1
results show that Plicera was well-tolerated and that oral administration
resulted in a significant elevation of target enzyme levels in healthy
volunteers. Based on these results, Amicus announced today the initiation
of two Phase 2 clinical trials of Plicera for Gaucher disease.

Plicera is designed to selectively bind to and stabilize GCase, the
enzyme deficient in Gaucher disease. This deficiency leads to lysosomal
accumulation of glucocerebroside inside certain cells, which is believed to
cause the various symptoms of Gaucher disease. Plicera facilitates proper
trafficking of the enzyme to the lysosomes, the compartments in the cell
where it is needed to break down glucocerebroside.

Phase 1 Plicera data being presented at ACMG

Two double-blind, placebo-controlled, dose escalation Phase 1 studies
in healthy volunteers were completed. These studies were designed to
evaluate the safety, tolerability and pharmacokinetics of Plicera. In the
first study, 36 subjects received a single dose of one of five dose levels
of Plicera. The second study was a multiple-dose study in which 18 subjects
received one of three dose levels of Plicera once daily for 7 consecutive
days. In both studies, Plicera was safe and well tolerated at all doses.
There were no serious adverse events and no subjects withdrew or
discontinued due to an adverse event. In the multiple-dose study, a
dose-dependent increase in GCase levels was observed in white blood cells
from healthy volunteers receiving Plicera.

Phase 2 Clinical Trials of Plicera in Gaucher Disease

Based on the Phase 1 results, Amicus has initiated two Phase 2 clinical
trials of Plicera for Gaucher disease. One is a 4-week study designed to
evaluate the safety and pharmacodynamic effects of Plicera in Type I
Gaucher patients who will discontinue enzyme replacement therapy for the
duration of the study. The second is a 6-month study designed to evaluate
the safety of Plicera and its effect on parameters that are commonly
abnormal in Gaucher disease. This study will be conducted in Type I Gaucher
patients who have never received enzyme replacement therapy. More
information regarding these studies can be found at clinicaltrials
and amicustherapeutics.

About Gaucher Disease

Gaucher disease, the most commonly diagnosed lysosomal storage
disorder, is caused by inherited genetic mutations in the GBA gene, which
result in deficient activity of the enzyme acid beta-glucosidase, also
known as glucocerebrosidase (GCase). Deficient GCase activity leads to
lysosomal accumulation of glucocerebroside inside certain cells, which is
believed to cause the various symptoms of Gaucher disease, including an
enlarged liver and spleen, abnormally low levels of red blood cells and
platelets and skeletal complications. In some cases there is significant
impairment of the central nervous system. Gaucher disease affects an
estimated 8,000 to 10,000 people worldwide. The U.S. Food and Drug
Administration’s Office of Orphan Products Development has granted orphan
drug designation for the active ingredient in Plicera in the United States.

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(TM) for Fabry disease, and Plicera(TM) for Gaucher
disease. The company is currently conducting Phase 1 trials with AT2220 for
the treatment of Pompe disease.

Amicus Therapeutics
amicustherapeutics