Amicus Therapeutics Announces First Quarter 2013 Financial Results and Corporate Updates

Amicus Therapeutics Announces First Quarter 2013 Financial Results and
Corporate Updates

    12-Month Results From Ongoing Phase 3 Fabry Disease Monotherapy Study
                               Anticipated 3Q13

        Phase 2b Pompe Co-Administration Study on Track to Begin 3Q13

CRANBURY, N.J., May 9, 2013 (GLOBE NEWSWIRE) -- Amicus Therapeutics
(Nasdaq:FOLD), a biopharmaceutical company at the forefront of therapies for
rare and orphan diseases, today announced financial results for the first
quarter ended March 31, 2013. The Company also summarized recent and upcoming
milestones and reiterated full-year 2013 operating expense guidance.

Key Highlights and Upcoming Milestones:

  *12-month results from first ongoing Phase 3 Fabry monotherapy study (Study
    011) anticipated 3Q13. FDA will consider entirety of 6- and 12-month data
    for potential U.S. approval of migalastat HCl monotherapy.
  *Phase 2b repeat-dose clinical study of AT2220 (duvoglustat HCl)
    co-administered with ERT (Myozyme®/Lumizyme®) for Pompe disease expected
    to begin 3Q13.
  *IND submission for Fabry chaperone-ERT co-formulated product planned by
    year-end 2013 for potential entry into clinic in early 2014.
  *Next-generation ERTs for Pompe disease and other LSDs advancing in
    preclinical studies.

John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics
stated, "Amicus had a productive first quarter that was largely focused on the
execution of clinical and regulatory activities for migalastat HCl monotherapy
for Fabry disease, our upcoming clinical studies in Fabry and Pompe, and the
build out of our CHART platform. A Type C meeting with the FDA will take place
during the second quarter to discuss the statistical analysis plan for Study
011 while the 12-month data are still blinded. We remain on track to unblind
and announce these 12-month results in the third quarter. We are also
finalizing the design of our repeat-dose Phase 2b co-administration study for
Pompe disease, which we expect to begin in the third quarter. Our strong
financial position will continue to support the advancement of these programs
as we approach several key milestones throughout the remainder of 2013."

Financial Highlights for First Quarter Ended March 31, 2013

  *Cash, cash equivalents, and marketable securities totaled $84.8 million at
    March 31, 2013 compared to $99.1 million at December 31, 2012.
  *Cash reimbursements received from GlaxoSmithKline (GSK) for shared
    development of migalastat HCl totaled $3.2 million compared to $5.0
    million in the first quarter 2012.
  *No revenue was reported due to a change in revenue recognition accounting
    under the expanded GSK collaboration. Total revenue of $7.8 million was
    recognized in the first quarter 2012.
  *Total operating expenses decreased to $17.3 million from $18.5 million in
    the first quarter 2012 due to lower research and development expenses as
    well as a decrease in personnel-related costs.
  *Net loss was $17.5 million, or $0.35 per share, compared to a net loss of
    $13.1 million, or $0.35 per share, for the first quarter 2012.

2013 Financial Guidance

As previously announced, Amicus expects full-year 2013 net cash spend to total
between $52 million and $58 million, including cash reimbursements received
from GSK. Amicus and GSK are responsible for 40% and 60% of global development
costs for migalastat HCl, respectively, in 2013 and beyond. The Company
continues to project that the current cash position and anticipated Fabry
program reimbursements from GSK are sufficient to fund operations into the
second half of 2014.

Program Updates

Migalastat HCl for Fabry Disease

Amicus in collaboration with 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.

Migalastat HCl Monotherapy

Migalastat HCl monotherapy (150 mg, every-other-day) is being investigated in
two ongoing randomized Phase 3 studies for Fabry disease (Study 011 and Study
012) in patients with genetic mutations identified as amenable to this
pharmacological chaperone in a cell-based assay.

  *Study 011 is comparing migalastat HCl to placebo to support a potential
    U.S. marketing application. The FDA has indicated that it will consider
    the entirety of the efficacy and safety data from the 6-month double-blind
    treatment period (Stage 1) and 6-month follow-up period (Stage 2) of Study
    011. The Stage 1 results have been reported while Stage 2 data are
    anticipated in the third quarter of 2013. A Type C meeting with the FDA
    has been scheduled during the second quarter to review the statistical
    analysis plan. Both Amicus and GSK will remain blinded to the Stage 2
    results until after this Type C meeting has occurred. Following the
    announcement of 12-month results, an additional meeting is anticipated
    with the FDA to discuss a U.S. marketing application for migalastat HCl
  *Study 012 is comparing open-label migalastat HCl to current standard of
    care ERTs (Fabrazyme and Replagal) to support global registration. A total
    of 60 patients were randomized 1.5:1 to switch from ERT to migalastat HCl
    or remain on ERT. Data are anticipated in the second half of 2014 on the
    primary outcome measure, which is renal function assessed by iohexol
    Glomerular Filtration Rate (GFR) at 18 months.

Migalastat HCl in Combination with ERT

In combination with ERT, migalastat HCl is designed to bind to and stabilize
infused alpha-Gal A enzyme, independent of a patient's genetic mutation.
Therefore Amicus believes this approach has the potential to benefit any
individual with Fabry disease. Amicus and GSK, in collaboration with JCR
Pharmaceutical Co. Ltd, are currently developing intravenous migalastat HCl
co-formulated with a proprietary recombinant human alpha-Gal A enzyme (JCR's
JR-051). An IND submission is planned by year-end 2013 for potential entry
into the clinic in early 2014.

The results from an open-label Phase 2 study (Study 013) of migalastat HCl
co-administered with ERT (Fabrazyme® or Replagal®) have built confidence
around the concept that migalastat HCl in combination with ERT can increase
levels of active enzyme in plasma and tissues and support the rationale for
the first repeat-dose clinical study with the chaperone ERT co-formulated

CHART Programs for Pompe Disease

Outside the collaboration agreement with GSK, Amicus owns exclusive rights to
the rest of its pipeline and applications of its CHART platform technology.
The current CHART programs for Pompe disease are investigating the
pharmacological chaperone AT2220 in combination with human recombinant GAA
(rhGAA) enzymes. These next-generation therapies have the potential to
increase enzyme activity in muscle and other disease-relevant tissues, improve
glycogen reduction, and mitigate immunogenicity compared to rhGAA alone.

  *AT2220-IV Co-Administered with Marketed ERTs: Amicus plans to initiate a
    repeat-dose Phase 2b clinical study in the third quarter of 2013 to
    evaluate a novel intravenous formulation of AT2220 (AT2220-IV)
    co-administered with currently marketed rhGAA (Myozyme/Lumizyme) in Pompe
    patients.To build upon positive results from a Phase 2 co-administration
    study (Study 010),Amicus plans to evaluate AT2220-IV co-administered with
    Myozyme/Lumizyme every 2 weeks to characterize safety, PK, and anti-rhGAA
    antibody titers. The study is expected to include both treatment-naïve and
    ERT-experienced individuals at leading Pompe centers.
  *Next-Generation ERT (AT2220 Co-Formulated with a Proprietary rhGAA
    Enzyme): Amicus is also developing a proprietary rhGAA enzyme
    co-formulated with AT2220 as a next-generation therapy for Pompe disease.
    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 of active enzyme, reduce
    immunogenicity and potentially enable novel routes of delivery such as
    subcutaneous administration.

Conference Call and Webcast

Amicus Therapeutics will host a conference call and audio/visual webcast
today, May 9, 2013 at 5:00 p.m. ET to review financial results and provide a
corporate update. Interested participants and investors may access the
conference call at 5:00 p.m. ET by dialing 877-303-5859 (U.S./Canada) or
678-224-7784 (international).

An audio/visual webcast can also be accessed via the Investors section of the
Amicus Therapeutics corporate web site at, and will be
archived for 30 days. 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.

The slide presentation for today's conference call and webcast is also
available in the Investors section of the Amicus Therapeutics corporate web
site at A telephonic replay of the call will be
available for seven days beginning at 8:00 p.m. ET today. Access numbers for
this replay are 855-859-2056 (U.S./Canada) and 404-537-3406 (international);
participant code 64638686.

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
novel, first-in-class treatments for a broad range of human genetic diseases,
with a focus on delivering new benefits to individuals with lysosomal storage
diseases. Amicus' lead programs include the small molecule pharmacological
chaperones migalastat HCl as a monotherapy and in combination with enzyme
replacement therapy (ERT) for Fabry disease; and AT2220 (duvoglustat HCl) in
combination with ERT for Pompe disease.

About Chaperone-Advanced Replacement Therapy (CHART)

The Chaperone-Advanced Replacement Therapy (CHART™) platform combines unique
pharmacological chaperones with enzyme replacement therapies (ERTs) for
lysosomal storage diseases (LSDs). In a chaperone-advanced replacement
therapy, a unique pharmacological chaperone is designed to bind to and
stabilize a specific therapeutic enzyme in its properly folded and active
form. This proposed CHART mechanism may allow for enhanced tissue uptake of
active enzyme, greater lysosomal activity, more reduction of substrate, and
lower immunogenicity compared to ERT alone. Improvements in enzyme stability
may also enable more convenient delivery of next-generation therapies. Amicus
is leveraging the CHART platform to develop pharmacological chaperones
co-administered with currently marketed ERTs as well as proprietary
next-generation therapies that consist of lysosomal enzymes co-formulated with
pharmacological chaperones.

About Migalastat HCl for Fabry Disease

Migalastat HCl is an investigational pharmacological chaperone in development
as a monotherapy and in combination with enzyme replacement therapy (ERT) for
the treatment of Fabry disease. 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 with genetic mutations that are amenable to this
chaperone in a cell-based assay. 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

Fabry disease is an inherited lysosomal storage disorder caused by deficiency
of the alpha-Gal A enzyme. 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 alpha-Gal A 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 CHART for Pompe Disease

In chaperone-advanced replacement therapy programs for Pompe disease, the
small molecule pharmacological chaperone AT2220 is designed to bind to and
stabilize human recombinant GAA (rhGAA) enzyme. Amicus is developing AT2220
co-administered with currently marketed ERTs (rhGAA enzymes, Myozyme/Lumizyme)
in parallel with the development of a next-generation ERT (AT2220
co-formulated with a proprietary rhGAA enzyme). Positive results from a Phase
2 study (Study 010) established human proof-of-concept that oral
administration of AT2220 just prior to infusing Myozyme/Lumizyme increases
enzyme activity in muscle compared to ERT alone. In preclinical studies of
AT2220 co-administered and co-formulated with Myozyme/Lumizyme, greater enzyme
uptake in disease-relevant tissues led to greater glycogen reduction compared
to Myozyme/Lumizyme alone. These chaperone-advanced replacement therapies also
have the potential to mitigate Pompe ERT-related immunogenicity because
properly folded proteins are generally less prone to aggregation and less

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 the
heart, muscle, and other tissues affected by the disease. 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.

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 preclinical and clinical
development of Amicus' candidate drug products, the timing and reporting of
results from preclinical studies and clinical trials evaluating Amicus'
candidate drug products, and the projected cash position for the Company.
Words such as, but not limited to, "look forward to," "believe," "expect,"
"anticipate," "estimate," "intend," "potential," "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 preclinical studies and 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. With
respect to statements regarding projections of the Company's cash position,
actual results may differ based on market factors and the Company's ability to
execute its operational and budget plans. In addition, all forward looking
statements are subject to other risks detailed in our Annual Report on Form
10-K for the year ended December 31, 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.

Table 1
Amicus Therapeutics, Inc.
(a development stage company)
Consolidated Statements of Operations
(In thousands, except share and per share amounts)
                                                           Period from
                                                              February 4, 2002
                                     Three Months            To
                                     Ended March 31,         March 31,
                                     2012        2013        2013
Research revenue                      $6,114    $--      $57,493
Collaboration and milestone revenue   1,660       --          64,382
Total revenue                         7,774       --          121,875
Operating Expenses:                                         
Research and development              14,004      11,989      327,882
General and administrative            4,095       4,823       137,436
Restructuring charges                 --          --          1,522
Impairment of leasehold improvements  --          --          1,030
Depreciation and amortization         420         439         12,207
In-process research and development   --          --          418
Total operating expenses              18,519      17,251      480,495
Loss from operations                  (10,745)    (17,251)    (358,620)
Other income (expenses):                                    
Interest income                       27          65          14,454
Interest expense                      (43)        (10)        (2,432)
Change in fair value of warrant       (2,376)     (262)       1,291
Other income                          --          --          252
Loss before tax benefit               (13,137)    (17,458)    (345,055)
Benefit from income taxes            --          --          8,708
Net loss                              (13,137)    (17,458)    (336,347)
Deemed dividend                       --          --          (19,424)
Preferred stock accretion             --          --          (802)
Net loss attributable to common       $(13,137) $(17,458) $(356,573)
Net loss attributable to common
stockholders per common share – basic $(0.35)   $(0.35)   
and diluted
Weighted-average common shares        37,887,520  49,621,188  
outstanding – basic and diluted

Table 2
Amicus Therapeutics, Inc.
(a development stage company)
Consolidated Balance Sheets
(in thousands, except share and per share amounts)
                                                       December 31, March 31,
                                                       2012         2013
Current assets:                                                     
Cash and cash equivalents                               $33,971      $26,736
Investments in marketable securities                    65,151       58,015
Receivable due from GSK                                 3,225        1,308
Prepaid expenses and other current assets               2,270        1,753
Total current assets                                    104,617      87,812
Property and equipment, less accumulated depreciation
and amortization of$8,501 and $8,872at December 31,   5,029        4,962
2012 and March 31, 2013, respectively
Other non-current assets                                442          442
Total Assets                                            $110,088     $93,216
Liabilities and Stockholders' Equity                                
Current liabilities:                                                
Accounts payable and accrued expenses                   8,845        6,427
Current portion of secured loan                         398          398
Warrant liability                                       --           1,170
Total current liabilities                               9,243        7,995
Deferredreimbursements                                 30,418       31,685
Warrant liability, non-current                          908          --
Secured loan, less current portion                      299          199
Commitments and contingencies                                       
Stockholders' equity:                                               
Common stock, $.01 par value, 125,000,000 shares
authorized, 49,631,672 shares issued and outstanding at 556          556
December 31, 2012, 49,631,672 shares issued and
outstanding at March 31, 2013
Additional paid-in capital                              387,539      389,113
Accumulated other comprehensive income                  14           15
Deficit accumulated during the development stage        (318,889)    (336,347)
Total stockholders' equity                             69,220       53,337
Total Liabilities and Stockholders' Equity              $110,088     $93,216


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