Amicus Therapeutics Announces Updated Results From Phase 2 Extension Study of Migalastat HCl for Fabry Disease

Amicus Therapeutics Announces Updated Results From Phase 2 Extension Study of
Migalastat HCl for Fabry Disease

Kidney Interstitial Capillary GL-3 Results for 60-Week Treatment on Migalastat
         HCl Presented at American Society of Nephrology Kidney Week

   Pharmacodynamic Markers Indicate Stable Renal Function in Patients With
                              Amenable Mutations

CRANBURY, N.J., Nov. 5, 2012 (GLOBE NEWSWIRE) -- Amicus Therapeutics
(Nasdaq:FOLD) today announced updated data from an open-label, Phase 2
extension study (Study 205/FAB-CL-205) to investigate the long-term safety,
tolerability and pharmacodynamics of migalastat HCl in 23 patients with Fabry
disease who completed an initial Phase 2 study. Amicus in collaboration with
GlaxoSmithKline (GSK) is developing the investigational pharmacological
chaperone Migalastat HCl for the treatment of Fabry disease. Results^1 from
Study 205 were presented at the American Society of Nephrology (ASN) Kidney
Week 2012.

Pol F. Boudes, MD, Chief Medical Officer of Amicus Therapeutics stated, "Fabry
is a broad spectrum disease that affects males and females resulting in
various symptoms. In our Phase 2 studies, kidney biopsies from both males and
females with amenable mutations have shown decreases in interstitial capillary
GL-3, a key biomarker of disease. Collectively these results support the
ongoing investigation of migalastat HCl monotherapy in Phase 3 studies. We
believe that migalastat HCl has the potential to become an important new
treatment option for Fabry patients with amenable mutations."

Key Study 205 Results Presented at ASN:

  *Enrolled 23 total subjects who completed primary treatment period (12 or
    24 weeks) and treatment extension (24 to 84 weeks) in 4 Phase 2 studies
    (Studies FAB-CL-201-204).
  *No drug-related serious adverse events. The most common adverse events in
    6 out of 23 subjects were arthralgia, fatigue, back pain, and pain in
  *8 patients had evaluable paired kidney biopsies from baseline in primary
    study and follow-up in Study 205 (60-week median treatment duration in
    Study 205 prior to biopsy). Median decrease in interstitial capillary
    globotriaosylceramide (GL-3) was 78% in the 5 patients (2 males and 3
    females) with genetic mutations amenable to migalastat HCl monotherapy in
    a cell-based assay. Median increase in interstitial capillary GL-3 was
    114% in 3 females with non-amenable mutations.
  *Renal function remained stable in patients with amenable mutations as
    demonstrated by estimated glomerular filtration rate (eGFR) and
  *Median treatment duration on migalastat HCl was 5.2 years (4.7 to 6.4
    years) in 17 subjects who completed Study 205 – final data analysis
  *16 out of 17 subjects who completed Study 205 are now enrolled in a
    separate open-label extension study (MGM116041)

GL-3 in Renal Peritubular Capillary Cells (PTCs, or Interstitial Capillaries)

GL-3 is the lipid substrate that accumulates in tissues affected by Fabry
disease, including the kidney. GL-3 inclusions in PTCs - also referred to as
interstitial capillaries - are measured by histology from kidney biopsies.
Reduction of GL-3 in renal PTCs previously supported conditional approval of
enzyme replacement therapy (ERT) for Fabry disease by the U.S. Food and Drug
Administration (FDA).

Previous scientific presentations^2 highlighted changes in interstitial
capillary GL-3 from baseline to various time points in initial Phase 2 studies
(Studies 202-204). Changes in interstitial capillary GL-3 from baseline (in
Studies 202-204) to follow-up (Study 205) were presented for the first time at
ASN 2012. Pathologists blinded to biopsy sequence assessed a total of 8
evaluable paired kidney biopsies by histology using the published,
quantitative Barisoni Lipid Inclusion Scoring System (BLISS)^3 by virtual

Renal Function

Estimated glomerular filtration rate (eGFR) remained stable. In 9 male
evaluable patients, decreases in proteinuria from baseline were observed in 8
patients with amenable mutations, with an increase reported in 1 non-amenable

About The Phase 2 and Phase 2 Extension Studies
Phase 2 Protocols Represented in Study 205 (n = 23)
          Treatment                     Extension            # in  # in Study
Phase 2   Period    Migalastat HCl      Period     Kidney    Study 205 with
Protocol  (Weeks)   Doses Evaluated     (Weeks)    Biopsies* 205   Amenable
Study 201           25, 100 and 250 mg
(n=9      12        twice-daily (BID)   84         No        6     6
males)              then 50 mg
                    once-daily (QD)
Study 202           150 mg,
(n=4      12        every-other-day     36         Yes       3     2
males)              (QOD)***
Study 203
(n=5      24        150 mg QOD***       24         Yes       5     3
Study 204           50, 150***, or 250
(n=9      12        mg QOD              36         Yes       9     5
*Per protocols, no kidney biopsies in Study 201; kidney biopsies in Studies
202-204 at baseline, after primary treatment period, and after extension
**Retrospectively identified as amenable to migalastat HCl in a cell-based
***Dose selected for Phase 3 studies (150 mg QOD)

Study 205 is an open-label extension study to investigate the long-term
safety, tolerability and pharmacodynamics of migalastat HCl that enrolled 23
patients with Fabry disease who completed four open-label Phase 2 studies
(Study 201, -202, -203 and -204) to evaluate the safety and tolerability of
various doses and dose regimens of oral migalastat HCl. In addition to safety
and tolerability, Study 205 is evaluating globotriaosylceramide (GL-3) in
interstitial capillaries, an important biomarker for Fabry disease, as well as
kidney function as measured by estimated glomerular filtration rate (eGFR) and

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 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

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.

About Amicus Therapeutics

Amicus Therapeutics (Nasdaq:FOLD) is a biopharmaceutical company at the
forefront of developing therapies for rare 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.

1. Germain, et al., ASN 2012, Long-Term Effects of Migalastat HCl on Fabry

2. Shiffmann, et al., LDN WORLD 2011, Long-Term Safety, Tolerability, and
Assessments of Renal Function in Adult Fabry Patients Receiving Treatment with
AT1001 (Migalastat Hydrochloride), a Pharmacological Chaperone

Giugliani, et al., LDN WORLD 2012, Oral Migalastat HCL (AT1001/GR181314A) as
an Investigational Therapy Evaluated in Females with Fabry Disease

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

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,"
"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 Annual 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.


CONTACT: Investors/Media:
         Sara Pellegrino
         (609) 662-5044
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