Amicus Therapeutics Presents Additional Results From Phase 2 Chaperone-Enzyme Replacement Therapy (ERT) Study for Fabry Disease

Amicus Therapeutics Presents Additional Results From Phase 2 Chaperone-Enzyme
Replacement Therapy (ERT) Study for Fabry Disease at LDN World Symposium

Migalastat HCl Co-Administered With ERT Increased Fabry Enzyme (Alpha-Gal A)
Levels

CRANBURY, N.J. and ORLANDO, Fla., Feb. 13, 2013 (GLOBE NEWSWIRE) -- Amicus
Therapeutics (Nasdaq:FOLD), today announced further results from an open-label
Phase 2 drug-drug interaction study (Study 013) to evaluate a single oral dose
of migalastat HCl (150 mg or 450 mg) co-administered prior to enzyme
replacement therapy (ERT) in males with Fabry disease. Preliminary results
were announced during 2012. Results for the migalastat HCl 450 mg dose group
are being presented for the first time in a poster^1 at the Lysosomal Disease
Network WORLD Symposium (LDN WORLD).

Dr. David G. Warnock, University of Alabama-Birmingham, stated, "When
co-administered with ERT, both doses of migalastat HCl appeared to increase
enzyme activity compared to Fabrazyme and Replagal alone in this study. We
believe that these results support further clinical studies in Fabry patients
to investigate the use of a pharmacological chaperone to maintain infused
alpha Gal-A enzymes in optimally active form through this chaperone-ERT
combination approach."

Amicus, in collaboration with GlaxoSmithKline (GSK), is developing the
investigational pharmacological chaperone migalastat HCl as a monotherapy and
in combination with ERT for the treatment of Fabry disease. When
co-administered with ERT, migalastat HCl binds to and stabilizes infused
enzyme in the circulation.

Migalastat HCl (150 mg and 450 mg) Co-Administered with ERT (Fabrazyme and
Replagal)

Each patient in Study 013 received their current dose and regimen of ERT
(Fabrazyme® or Replagal®, infused alpha-Gal A enzymes) at one infusion. A
single oral dose of migalastat HCl (150 mg or 450 mg) was co-administered 2
hours prior to the next infusion of the same ERT at the same dose and regimen.
Among 20 total males who enrolled, 3 opted to participate in the migalastat
150 mg and then again in the 450 mg treatment arms.

  *Safety: finalized safety data are expected in 2Q13. As currently
    evaluated, 2 serious adverse events (SAEs) occurred in one patient
    (transient ischemic attack and hospitalization for acute pain and
    acroparesthesia due to Fabry disease) and were considered by the
    investigator to be unrelated to migalastat HCl when co-administered with
    ERT. Of the remaining treatment emergent adverse events the majority were
    unrelated to study drug.
  *Active enzyme in plasma: active alpha-Gal A enzyme levels were measured in
    plasma (total area under the curve, or AUC) during and after each
    infusion. Following co-administration, active enzyme levels in plasma
    increased in 22 out of 23 instances and were unchanged in 1 instance
    compared to ERT alone. These PK results demonstrated a drug-drug
    interaction between both doses of migalastat HCl and both ERTs.
  *Enzyme uptake into skin: alpha-Gal A enzyme levels were also measured in
    skin following each infusion. Increased levels of alpha-Gal A enzyme
    measured in skin biopsy samples on day 2 and, to a lesser extent, on day
    7, showed increased enzyme uptake into tissue compared to ERT alone. This
    exploratory assessment showed that the greatest increases in active
    alpha-Gal A enzyme levels were observed following migalastat HCl 450 mg
    co-administered with ERT.

                         Alpha-Gal A Levels of Active Enzyme
                          Mean-Fold Increase
                          in                 Mean-Fold        Mean-Fold
                         Plasma Area Under  Increase in Skin Increase in Skin
                          Curve (AUC) vs.    at Day 2 vs. ERT at Day 7 vs. ERT
                          ERT                Alone (Range)    Alone (Range)
                          Alone (Range)
Migalastat HCl 150 mg +
Replagal 0.2 mg/kg (n =   4.3 (3.2 to 5.0)   1.8 (1.4 to 2.3) 1.4 (1.1 to 1.8)
4)
Migalastat HCl 150 mg +
Fabrazyme 0.5 mg/kg (n =  3.0 (2.0 to 4.2)   2.6 (1.1 to 3.9) 1.4 (0.7 to 2.8)
5)*
Migalastat HCl 150 mg +
Fabrazyme 1.0 mg/kg (n =  2.0 (1.6 to 2.2)   1.9 (1.6 to 2.1) 1.4 (1.2 to 1.7)
3)
Migalastat HCl 450 mg +
Replagal 0.2 mg/kg (n =   3.1 (2.3 to 5.0)   2.3 (1.6 to 3.7) 2.1 (1.0 to 2.8)
4)
Migalastat HCl 450 mg +
Fabrazyme 0.5 mg/kg (n =  2.4                3.7              1.9
1)*
Migalastat HCl 450 mg +
Fabrazyme 1.0 mg/kg (n =  2.0 (1.0 to 3.2)   1.8 (1.0 to 2.5) 1.5 (0.6 to 3.3)
6)

* Due to Fabrazyme supply difficulties during enrollment, subjects had been
receiving 0.5 mg/kg Fabrazyme infused every two weeks

John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics
said, "Study 013 was the first clinical study to investigate the chaperone-ERT
combination approach. This study has been a major catalyst for our further
development of chaperone-ERT combinations across the lysosomal storage
diseases, including chaperones co-administered with marketed ERTs as well as
proprietary chaperone-ERT co-formulated products in preclinical development as
next-generation ERTs."

In addition to chaperone-ERT co-administration in Fabry disease, Amicus and
GSK are developing migalastat HCl co-formulated with JCR Pharmaceutical Co.
Ltd's proprietary investigational ERT (JR-051, recombinant human alpha-Gal A
enzyme). This chaperone-ERT co-formulated product has the potential to enter
the clinic in late-2013 or early 2014.

Conference Call and Webcast

John F. Crowley, Chairman and Chief Executive Officer, and members of the
Amicus executive team will host a conference call and live audio/visual
webcast on Friday, February 15, 2013 at 11:30am ET to discuss the data from
several programs presented at LDN WORLD. Interested participants and investors
may access the conference call at 11:30 a.m. ET by dialing 877-303-5859
(U.S./Canada) or 678-224-7784 (international). A live audio webcast can also
be accessed via the Investors section of the Amicus Therapeutics corporate web
site at http://ir.amicustherapeutics.com/events.cfm, and will be archived for
30 days. The slide presentation for the conference call/webcast will also be
available at http://ir.amicustherapeutics.com/events.cfm. Web participants are
encouraged to go to the web site 15 minutes prior to the start of the call to
register, download and install any necessary software. A telephonic replay of
the call will be available for seven days beginning at 2:30 p.m. ET on
February 15, 2013. Access numbers for this replay are 855-859-2056
(U.S./Canada) and 404-537-3406 (international); participant code 97505816.

About Amicus Therapeutics

Amicus Therapeutics (Nasdaq:FOLD) is a biopharmaceutical company at the
forefront of therapies for rare and orphan diseases. The Company is developing
orally-administered, small molecule drugs called pharmacological chaperones, a
novel, first-in-class approach to treating a broad range of human genetic
diseases. Amicus' late-stage programs for lysosomal storage disorders include
migalastat HCl monotherapy in Phase 3 for Fabry disease; migalastat HCl
co-administered with enzyme replacement therapy (ERT) in Phase 2 for Fabry
disease; and AT2220 co-administered with ERT in Phase 2 for Pompe disease.

About Migalastat HCl

Amicus in collaboration with GlaxoSmithKline (GSK) is developing the
investigational pharmacological chaperone migalastat HCl for the treatment of
Fabry disease. Amicus has commercial rights to all Fabry products in the
United States and GSK has commercial rights to all of these products in the
rest of world.

As a monotherapy, migalastat HCl is designed to bind to and stabilize, or
"chaperone" a patient's own alpha-galactosidase A (alpha-Gal A) enzyme in
patients with genetic mutations that are amenable to this chaperone in a
cell-based assay. Migalastat HCl monotherapy is in Phase 3 development (Study
011 and Study 012) for Fabry patients with genetic mutations that are amenable
to this chaperone monotherapy in a cell-based assay. Study 011 is a
placebo-controlled study intended primarily to support U.S. registration, and
Study 012 compares migalastat HCl to ERT to primarily support global
registration.

For patients currently receiving ERT for Fabry disease, migalastat HCl in
combination with ERT may improve ERT outcomes by keeping the infused alpha-Gal
A enzyme in its properly folded and active form thereby allowing more active
enzyme to reach tissues.^2 Migalastat HCl co-administered with ERT is in Phase
2 (Study 013) and migalastat HCl co-formulated with JCR Pharmaceutical Co.
Ltd's proprietary investigational ERT (JR-051, recombinant human alpha-Gal A
enzyme) is in preclinical development.

About Fabry Disease

Fabry disease is an inherited lysosomal storage disorder caused by deficiency
of an enzyme called alpha-galactosidase A (alpha-Gal A). The role of alpha-Gal
A within the body is to break down specific lipids in lysosomes, including
globotriaosylceramide (GL-3, also known as Gb3). Lipids that can be degraded
by the action of α-Gal are called "substrates" of the enzyme. Reduced or
absent levels of alpha-Gal A activity leads to the accumulation of GL-3 in the
affected tissues, including the kidneys, heart, central nervous system, and
skin. This accumulation of GL-3 is believed to cause the various symptoms of
Fabry disease, including pain, kidney failure, and increased risk of heart
attack and stroke.

It is currently estimated that Fabry disease affects approximately 5,000 to
10,000 people worldwide. However, several literature reports suggest that
Fabry disease may be significantly under diagnosed, and the prevalence of the
disease may be much higher.

1. Bichet, et al., A Phase 2a Study to Investigate the Effect of a Single Dose
of Migalastat HCl, a Pharmacological Chaperone, on Agalsidase Activity in
Subjects with Fabry Disease, LDN WORLD 2012

2. Benjamin, et al., Molecular Therapy: April 2012, Vol. 20, No. 4, pp.
717–726.

Forward-Looking Statements

This press release contains "forward-looking statements" within the meaning of
the Private Securities Litigation Reform Act of 1995 relating to clinical
development of Amicus' candidate drug products and the timing and reporting of
results from clinical trials evaluating Amicus' candidate drug products. Words
such as, but not limited to, "look forward to," "believe," "expect,"
"anticipate," "estimate," "intend," "plan," "targets," "likely," "will,"
"may," "would," "should" and "could," and similar expressions or words
identify forward-looking statements. Such forward-looking statements are based
upon current expectations that involve risks, changes in circumstances,
assumptions and uncertainties. The inclusion of forward-looking statements
should not be regarded as a representation by Amicus that any of its plans
will be achieved. Any or all of the forward-looking statements in this press
release may turn out to be wrong. They can be affected by inaccurate
assumptions Amicus might make or by known or unknown risks and uncertainties.
For example, with respect to statements regarding the goals, progress, timing
and outcomes of discussions with regulatory authorities and the potential
goals, progress, timing and results of clinical trials, actual results may
differ materially from those set forth in this release due to the risks and
uncertainties inherent in the business of Amicus, including, without
limitation: the potential that results of clinical or pre-clinical studies
indicate that the product candidates are unsafe or ineffective; the potential
that it may be difficult to enroll patients in our clinical trials; the
potential that regulatory authorities may not grant or may delay approval for
our product candidates; the potential that preclinical and clinical studies
could be delayed because we identify serious side effects or other safety
issues; the potential that we will need additional funding to complete all of
our studies and, our dependence on third parties in the conduct of our
clinical studies. Further, the results of earlier preclinical studies and/or
clinical trials may not be predictive of future results. In addition, all
forward looking statements are subject to other risks detailed in our
Quarterly Report on Form 10-Q for the quarter ended June 30, 2012. You are
cautioned not to place undue reliance on these forward-looking statements,
which speak only as of the date hereof. All forward-looking statements are
qualified in their entirety by this cautionary statement, and Amicus
undertakes no obligation to revise or update this news release to reflect
events or circumstances after the date hereof. This caution is made under the
safe harbor provisions of Section21E of the Private Securities Litigation
Reform Act of 1995.

FOLD–G

CONTACT: Investors/Media:
         Sara Pellegrino
         spellegrino@amicusrx.com
         (609) 662-5044
        
         Media:
         Dan Budwick
         (973) 271-6085
         dan@purecommunicationsinc.com
 
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