Amicus Therapeutics and GlaxoSmithKline Announce Top Line 6-Month Primary Treatment Period Results From First Phase 3 Fabry

Amicus Therapeutics and GlaxoSmithKline Announce Top Line 6-Month Primary
Treatment Period Results From First Phase 3 Fabry Monotherapy Study

CRANBURY, N.J. and LONDON, Dec. 19, 2012 (GLOBE NEWSWIRE) -- Amicus
Therapeutics (Nasdaq:FOLD) and GlaxoSmithKline plc (GSK) today announced the
6-month primary treatment period results from the first Phase 3 global
registration study (Study 011) of investigational oral migalastat HCl
monotherapy in males and females with Fabry disease who had genetic mutations
identified as amenable to migalastat HCl in a cell-based assay. Study 011
randomized a total of 67 patients to receive oral migalastat HCl 150 mg or
placebo on an every-other-day (QOD) dosing schedule during a 6-month,
double-blind primary treatment period.

Reduction of GL-3 Substrate in Kidney Interstitial Capillaries:

Globotriaosylceramide (GL-3) is the lipid substrate that accumulates in
tissues of patients with Fabry disease, most notably in the kidney. GL-3
clearance from the kidney interstitial capillaries has been used as a marker
of treatment effect in Fabry disease. The pre-designated primary endpoint of
Study 011 was a responder analysis evaluating the number of patients who
demonstrated a 50% or greater reduction in kidney interstitial capillary GL-3
after 6 months of treatment with migalastat HCl compared to placebo. During a
6-month open label follow-up period all patients received migalastat HCl. The
FDA has also indicated that it will consider the 12-month efficacy and safety
data from Study 011. The paired kidney biopsies from baseline and month 6 were
assessed by histological scoring using the published, quantitative Barisoni
Lipid Inclusion Scoring System with Virtual Microscopy (BLISS-VM).^1 This
methodology will also be utilized for the evaluation of the kidney biopsies at
month 12. Amicus and GSK remain blinded to the 12-month data.

In Study 011 patients with evaluable baseline biopsies, 13/32 (41%) in the
migalastat HCl treatment group demonstrated a 50% or greater reduction in
kidney interstitial capillary GL-3 after 6 months of study treatment versus
9/32 (28%) in the placebo group. This difference did not achieve statistical
significance (p=0.3) according to the pre-specified primary endpoint analysis.

In addition to the binary responder analysis reported above, a pre-specified
secondary analysis assessing the absolute percent change in kidney
interstitial capillary GL-3 from baseline to month 6 was performed. Taken
alone this analysis showed a median reduction of 41% in the migalastat HCl
group versus a median reduction of 6% in the placebo group (p=0.093).

To date, no drug-related serious adverse events have been observed. The most
common treatment emergent adverse events occurring in 10% or more of subjects
were (migalastat; placebo, respectively): headache (35%; 21%); fatigue (12%;
12%); nausea (12%; 9%); nasopharyngitis, or inflammation of the nose and
throat (15%; 6%); and parasthesia, or tingling sensation of the skin (9%;
12%). The 4 dropouts in this portion of the study were deemed by the
investigators to be unrelated to study medication.

The 6-month secondary endpoints in Study 011 continue to be analyzed and will
be presented at the Lysosomal Disease Network WORLD Symposium (LDN WORLD), to
be held February 12-15, 2013, in Orlando, Florida. Secondary endpoints include
urine GL-3 and renal function (iohexol GFR, eGFR and 24-hour urine protein).

John F. Crowley, Chairman and Chief Executive Officer of Amicus, stated
"Consistent with our Phase 2 experience, the 6-month results from Study 011
demonstrate notable trends in kidney interstitial capillary GL-3 reduction in
favor of migalastat HCl monotherapy compared to placebo. We look forward to
announcing additional 6-month results at the WORLD Symposium in February,
including a presentation of important secondary and tertiary endpoints in this
study. We also anticipate 12-month results from this study in the first half
of 2013. Once we have the 12-month data, we intend to meet with FDA to discuss
a U.S. approval pathway. We continue to believe that migalastat HCl may become
an important treatment option as an oral monotherapy drug for both men and
women with Fabry disease who have amenable mutations."

Marc Dunoyer, Global Head of GSK Rare Diseases added, "GSK and Amicus are
committed to advancing migalastat HCl as a monotherapy in Fabry patients with
amenable mutations. While these 6-month data are encouraging, there is
additional work to be done. We continue to analyze the 6-month results and
look forward to receiving the 12-month results from this study. In addition
the results of Study 012, our second Phase 3 Fabry monotherapy study, will add
to the totality of our data and give us a more complete picture of the
clinical effect of migalastat HCl. This study, an 18-month comparison of
migalastat to ERT, with iohexol GFR as the primary endpoint, is fully
recruited and due to report in 2014."

The 6-month primary treatment period in Study 011 was completed in June 2012
in 63 out of 67 randomized patients. All 63 of these patients entered the
6-month open-label follow-up period in Study 011, to continue to receive
migalastat HCl or to switch from placebo to migalastat HCl. In December 2012,
a total of 59 patients completed this treatment period and received an
additional kidney biopsy at month 12. In addition, 57 out of 59 patients who
completed Study 011 continue to receive migalastat HCl in both ongoing
open-label extension studies. The results from the 6-12 month period of study
011 are expected in the first half of 2013 and will include 12-month data in
the migalastat HCl group and 6-month data in the placebo crossover group.

A second Phase 3 global registration study (Study 012) is also underway to
compare open-label migalastat HCl to enzyme replacement therapy (ERT) to
primarily support global registration. Study 012 (The ATTRACT, or
FAB-AT1001-012 Study) is a randomized, open-label 18-month Phase 3 study
investigating the safety and efficacy of oral migalastat HCl 150 mg QOD
compared to standard-of-care infused therapy using ERTs (Fabrazyme® and
Replagal®). This study achieved final enrollment of 60 total patients in
December 2012.

Study 011 Design

Study 011 - also referred to as FACETS - is one of two ongoing Phase 3 studies
of migalastat HCl monotherapy being conducted by Amicus and GlaxoSmithKline
(GSK). This study was designed based on feedback from the U.S. Food and Drug
Administration (FDA), and is primarily intended to support U.S. registration.
Study 011 randomized 67 patients (24 males and 43 females) diagnosed with
Fabry disease who had genetic mutations amenable to chaperone monotherapy in a
cell-based assay. For the 6-month, double-blind primary treatment period
patients were randomized to migalastat HCl 150 mg or placebo on an
every-other-day (QOD) oral dosing schedule. During a 6-month open-label follow
up period, patients continued treatment with migalastat HCl or switched from
placebo to migalastat HCl.

The primary analysis compared the number of responders in the migalastat HCl
versus placebo groups, based on a 50% or greater reduction in interstitial
capillary globotriaosylceramide (GL-3) during the 6-month, double-blind
treatment period. GL-3 – also referred to as peritubular capillary (PTC)
inclusions – is measured in kidney biopsies. Pathologists blinded to biopsy
sequence used the published, quantitative Barisoni Lipid Inclusion Scoring
System with virtual microscopy (BLISS-VM) for the histological evaluation of
interstitial capillary GL-3 in Study 011. BLISS is a more sensitive scoring
system to measure GL-3 inclusions in PTCs compared to the semi-quantitative
methodology used in previous pivotal studies of enzyme replacement therapy
(ERT) for Fabry disease. Secondary endpoints for Study 011 include safety and
tolerability, urine GL-3 and kidney function.

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 those patients with genetic mutations that are amenable to this
chaperone in a cell-based assay. Oral migalastat HCl monotherapy is in Phase 3
development (Study 011 and Study 012) for Fabry patients with amenable
mutations. Study 011 is a placebo-controlled study intended primarily to
support U.S. registration, and Study 012 is comparing open-label 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. 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. Migalastat HCl is subject to evaluation by regulatory authorities
before being made available as a treatment for patients.

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
manifestations 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.

About Amicus Therapeutics

Amicus Therapeutics (Nasdaq:FOLD) is a biopharmaceutical company at the
forefront of developing 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 GlaxoSmithKline

GlaxoSmithKline - one of the world's leading research-based pharmaceutical and
healthcare companies - is committed to improving the quality of human life by
enabling people to do more, feel better and live longer. For further
information please visit www.gsk.com.

1. Barisoni L., et al., Archives of Pathology & Laboratory Medicine: July
2012, Vol. 136, No. 7, pp. 816-824.

Amicus Forward-Looking Statements

This press release contains, and the accompanying conference call will
contain, "forward-looking statements" within the meaning of the Private
Securities Litigation Reform Act of 1995 relating to clinical development of
Amicus' candidate drug products, the timing and reporting of results from
preclinical studies and 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," "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 Section 21E of the Private Securities Litigation
Reform Act of 1995.

GlaxoSmithKline cautionary statement regarding forward-looking statements

Under the safe harbor provisions of the U.S. Private Securities Litigation
Reform Act of 1995, GSK cautions investors that any forward-looking statements
or projections made by GSK, including those made in this announcement, are
subject to risks and uncertainties that may cause actual results to differ
materially from those projected. Factors that may affect GSK's operations are
described under 'Risk Factors' in the 'Financial review & risk section' in the
company's Annual Report 2011 included as exhibit 15.2 to the company's Annual
Report on Form 20-F for 2011.

Amicus Contact:
Investors/Media: Sara Pellegrino (609) 662-5044 / spellegrino@amicusrx.com

Media: Dan Budwick (973) 271-6085 / dan@purecommunicationsinc.com


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