Amicus Therapeutics Presents Additional 6-Month Results from Phase 3 Fabry Monotherapy Study at LDN World Symposium

Amicus Therapeutics Presents Additional 6-Month Results from Phase 3 Fabry
Monotherapy Study at LDN World Symposium

Conference Call Today at 11:30am ET

CRANBURY, N.J. and ORLANDO, Fla., Feb. 15, 2013 (GLOBE NEWSWIRE) -- Amicus
Therapeutics (Nasdaq:FOLD), today announced additional 6-month (Stage 1)
results from the first ongoing Phase 3 global registration study (Study 011)
of investigational oral migalastat HCl monotherapy (150 mg, every-other-day)
in males and females with Fabry disease who had genetic mutations identified
as amenable to migalastat HCl in a cell-based assay. Stage 1 results were
highlighted in an oral platform presentation^1 at the Lysosomal Disease
Network WORLD Symposium (LDN WORLD) by Dr. Fatih Ezgu, Gazi University.

Study 011 consists of a 6-month, double-blind period (Stage 1) when subjects
received migalastat HCl 150 mg or placebo, a 6-month open label-follow up
period (Stage 2) when all patients received migalastat HCl, and an ongoing
12-month open-label extension.

Initial top-line Stage 1 results previously reported in December, 2012 for the
primary endpoint in Stage 1 did not meet statistical significance. The
pre-specified primary and secondary analyses of the primary endpoint
numerically favored migalastat HCl over placebo. In the primary responder
analysis, 13/32 (41%) in the migalastat HCl group versus 9/32 (28%) in the
placebo group demonstrated a 50% or greater reduction in kidney interstitial
capillary GL-3 from baseline to month 6 (p=0.3). Taken alone the secondary
analysis of the absolute percent change in kidney interstitial capillary GL-3
from baseline to month 6 showed a median reduction of 41% in the migalastat
HCl group versus a median reduction of 6% in the placebo group (p=0.093).

Study 011 entry criteria, particularly elevated urine GL-3, were intended to
enrich for patients with higher interstitial capillary GL-3, and more
measurable disease burden. Although all patients had detectable interstitial
capillary GL-3 at baseline, a number of patients had low levels of GL-3 at
baseline, making it difficult to detect a significant difference in responders
between the 2 treatment groups. Clearance of kidney interstitial capillary
GL-3, a marker of treatment effect, is being measured by histology^2 in
evaluable kidney biopsies from baseline to month 6 (Stage 1) as well as
baseline to month 12 (Stage 2). Stage 2 results remain blinded at this time.

Dr. Ezgu said, "The 6-month data from Study 011 are encouraging, including a
post-hoc subgroup analysis presented today, and we continue to evaluate
patients in this ongoing study. There is still an unmet medical need for Fabry
disease treatments. Migalastat HCl may potentially offer an oral treatment for
Fabry patients with amenable mutations based on these results from Study 011
and earlier clinical studies, and potentially forthcoming data from ongoing

Study 011: Stage 1 (Baseline to Month 6) Updated Data Highlights at LDN WORLD

  *Post-Hoc Subgroup Analysis of Primary Endpoint (modified intent-to-treat
    (mITT), n=60): in a post-hoc sub-group analysis, patients with a higher
    baseline disease burden (0.3 inclusions or more per interstitial
    capillary, n=25) were compared to those with a lower baseline disease
    burden (0.3 or fewer inclusions per interstitial capillary, n=35). In the
    25 patients (14 males and 11 females) with higher baseline disease burden,
    7/11 (64%) on migalastat HCl and 2/14 (14%) on placebo were classified as
    responders. Among the 35 patients (8 males and 27 females) with lower
    baseline disease burden, 6/19 (32%) on migalastat HCl and 7/16 (44%) on
    placebo were classified as responders.
  *Urine GL-3: The observed median reduction in urine GL-3 from baseline was
    17% for placebo and 12% for migalastat. However due to unexpected
    variability in the pre-treatment urine GL-3 data (>1.6 fold difference
    between values at screening and baseline), any potential treatment effects
    on urine GL-3 cannot be determined.
  *Renal Function from Baseline to Month 6: Renal function remained stable
    and changes from baseline were similar in both treatment groups during
    stage 1. Themean (SD) increase in estimated glomerular filtration rate
    (eGFR) was 2.7 (15.1) mL/min/1.73m^2 in the migalastat HCl group compared
    to a mean decrease of 2.4 (10.8) mL/min/1.73m^2 in the placebo group. No
    clinically meaningful changes in proteinuria were observed, and iohexol
    GFR data are currently being analyzed.
  *Safety: During the first 6 months, no drug-related serious adverse events
    have been observed. No subjects discontinued migalastat HCl therapy due to
    a treatment emergent adverse event and the majority of adverse events in
    both treatment groups were mild in nature.

John F. Crowley, Chairman and Chief Executive Officer of Amicus, stated, "The
6-month results presented today at the WORLD Symposium support our commitment
to the ongoing development of migalastat HCl monotherapy. We look forward to
reporting the 12-month results from this study to add to the entirety of the
data that the FDA has indicated would support a potential U.S. conditional

Study 011 results from Stage 2 are anticipated in the second quarter of 2013.
The U.S. Food and Drug Administration (FDA) has indicated that it will
consider the entirety of the Stage 1 and Stage 2 efficacy and safety data from
Study 011. A meeting with the agency is anticipated in mid-2013 to discuss the
U.S. conditional approval pathway for migalastat HCl under subpart H.

A second Phase 3 global registration study (Study 012) is also underway to
compare open-label migalastat HCl to 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.

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, and will be archived for
30 days. The slide presentation for the conference call/webcast will also be
available at 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.

Study 011 Design

Study 011 - also referred to as FACETS - is one of 2 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 considered amenable to chaperone
monotherapy in a cell-based assay. For the 6-month, double-blind period (Stage
1) 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 (Stage 2), 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 in Stage 1, based on a 50% or greater reduction in
interstitial capillary globotriaosylceramide (GL-3) as measured in kidney
biopsies. Pathologists blinded to biopsy sequence are using the published,
quantitative Barisoni Lipid Inclusion Scoring System with virtual microscopy
(BLISS-VM)^2 for the histological evaluation of interstitial capillary GL-3 in
kidney biopsies from baseline to month 6 (Stage 1) and from baseline to month
12 (Stage 2). Secondary endpoints for Study 011 include safety and
tolerability, urine GL-3 and kidney function.

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 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

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.

1. Nicholls, et al., Phase 3 Study of Migalastat HCl for Fabry Disease: Stage
1 Results, LDN WORLD 2013

2. Barisoni, 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 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.


CONTACT: Investors/Media:
         Sara Pellegrino
         (609) 662-5044
         Dan Budwick
         (973) 271-6085
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