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Amicus Therapeutics Advances Chaperone-Enzyme Replacement Therapy (ERT) Combination Platform in Pompe Disease



Amicus Therapeutics Advances Chaperone-Enzyme Replacement Therapy (ERT)
Combination Platform in Pompe Disease

Phase 2 AT2220-ERT Co-Administration and Preclinical AT2220-ERT Co-Formulation
                   Studies Presented at LDN WORLD Symposium

             Next-Generation ERT Advancing in Preclinical Studies

              Conference Call on February 15, 2013 at 11:30am ET

CRANBURY, N.J., and ORLANDO, Fla., Feb. 14, 2013 (GLOBE NEWSWIRE) -- Amicus
Therapeutics (Nasdaq:FOLD) today announced positive results from clinical and
preclinical studies of the pharmacological chaperone AT2220 (duvoglustat HCl)
in combination with ERT for Pompe disease at the Lysosomal Disease Network
WORLD Symposium (LDN WORLD).

John F. Crowley, Chairman and Chief Executive Officer of Amicus stated, "The
advancement of our core platform technology in Pompe and other lysosomal
storage disorders is a continued great step forward for Amicus. Across these
serious genetic diseases, we are leveraging this chaperone-ERT combination
platform to work towards improving currently marketed ERTs and to develop our
own proprietary next-generation ERTs that incorporate our small molecule
chaperones. These chaperone stabilizers have the potential to enhance ERT
activity and tissue uptake while also significantly reducing the
immunogenicity of the ERTs. Through these programs we hope to offer new
benefits and treatment options for patients with lysosomal storage diseases."

Chaperone-ERT Combinations for Pompe Disease

AT2220 Co-Administered with Marketed ERTs

Positive results from a Phase 2 study (Study 010) established human
proof-of-concept that co-administration of AT2220 just prior to infusing ERT
(Myozyme/Lumizyme, or rhGAA enzymes) increases GAA enzyme activity in muscle
tissue compared to ERT alone. These results appear in a poster^1 and will be
featured in an oral platform presentation at LDN WORLD on Friday, February 15
at 9:15 am ET.

Based on these results, Amicus plans to initiate a repeat-dose clinical study
in the third quarter of 2013 to evaluate a novel intravenous formulation of
AT2220 (AT2220-IV) co-administered with Myozyme/Lumizyme. AT2220-IV when
co-administered with ERT is designed to have an improved pharmacokinetic (PK)
profile compared to oral AT2220 for all Pompe patients, many of whom are
unable to swallow an oral small molecule. The upcoming clinical study will
investigate multiple doses of AT2220-IV co-administered with Myozyme/Lumizyme
every two weeks in Pompe patients. Objectives of the study are to characterize
safety and PK for later evaluation of infants and special populations. Key
parameters are expected to include GAA enzyme activity and AT2220 levels in
plasma and muscle, as well as rhGAA antibody titers.

Next-Generation ERT (AT2220 Co-Formulated with a Proprietary Amicus ERT)

Preclinical studies of AT2220 co-formulated with rhGAA enzyme
(Myozyme/Lumizyme) were presented for the first time in a poster^2 at LDN
WORLD. This chaperone-ERT co-formulation resulted in up to 2.5-fold greater
enzyme uptake in multiple disease-relevant tissues and led to greater glycogen
reduction compared to rhGAA alone in GAA knock-out mice. Collectively these
data suggest that AT2220 directly binds to and stabilizes rhGAA, potentially
leading to a larger amount of properly folded, active enzyme available for
uptake into tissue. AT2220 co-formulated with ERT may also mitigate Pompe
ERT-related immunogenicity since properly-folded proteins are less prone to
aggregation and less immunogenic.

Following the completion of these preclinical studies, Amicus entered into a
contract with Laureate Pharmaceuticals for the manufacture of a proprietary
rhGAA enzyme. Amicus is developing AT2220 co-formulated with this proprietary
enzyme as a next-generation ERT for Pompe disease. Through this approach
Amicus believes it has the potential to improve the properties of the rhGAA
enzyme itself while incorporating AT2220 as a small molecule stabilizer to
increase exposure and tissue uptake, and reduce immunogenicity relative to
currently marketed ERTs. Successful development of a more stable ERT may also
enable novel routes of delivery such as subcutaneous administration.

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 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 AT2220 for Pompe Disease

AT2220 is an investigational, orally-administered pharmacological chaperone
owned exclusively by Amicus. In published preclinical studies, AT2220-ERT
co-administration resulted in significant increases in muscle rhGAA levels and
decreases in glycogen levels in a mouse model of Pompe disease. Preclinical
results to date also suggest that AT2220-ERT co-administration may mitigate
ERT-induced immunogenicity by stabilizing the enzyme in its properly folded
and active form. Initial ex vivo studies using T cells derived from blood from
50 healthy donors demonstrated that the addition of AT2220 may significantly
reduce the immunogenicity of Myozyme and Lumizyme.

Pompe disease is a lysosomal storage disease characterized by progressive
skeletal muscle weakness and respiratory insufficiency. It is caused by a
deficiency in GAA activity, which leads to accumulation of glycogen in tissues
affected by the disease (primarily muscle). Pompe disease affects an estimated
5,000 to 10,000 individuals worldwide and is clinically heterogeneous in the
age of onset, the extent of organ involvement, and the rate of progression.

^1 Kishnani, et al., A Phase 2a Study to Investigate Drug-Drug Interactions
between Escalating Doses of AT2220 (Duvoglustat Hydrochloride) and Acid
Alfa-Glucosidase in Subjects with Pompe Disease, LDN WORLD 2013

^2 Khanna, et al., Exploring the Use of a Co-formulated Pharmacological
Chaperone AT2220 with Recombinant Human Acid Alpha-Glucosidase for Pompe
Disease, LDN WORLD 2013

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.

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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