Amicus Therapeutics Announces Full-Year 2012 Financial Results and Corporate Updates

Amicus Therapeutics Announces Full-Year 2012 Financial Results and Corporate

            CHART Programs Advancing in Lysosomal Storage Diseases

12-Month Results from Ongoing Phase 3 Fabry Disease Monotherapy Study Expected

CRANBURY, N.J., March 12, 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 full-year
ended December 31, 2012. The Company also summarized recent and upcoming
milestones and reiterated full-year 2013 operating expense guidance.

Key Highlights and Upcoming Milestones:

  *Stage 1 (6-month) results from first ongoing Phase 3 Fabry monotherapy
    study (Study 011) – Stage 2 (12-month) data anticipated 3Q13. FDA will
    consider entirety of Stage 1 and Stage 2 data for potential U.S. approval
    of migalastat HCl monotherapy.
  *Positive results from Phase 2 study (Study 010) of AT2220 co-administered
    with ERT (Myozyme®/Lumizyme®) in Pompe patients – repeat-dose clinical
    study on track to begin 3Q13.
  *Results from Phase 2 study (Study 013) of migalastat HCl co-administered
    with ERT (Fabrazyme® and Replagal®) in Fabry patients – IND submission
    planned for chaperone-ERT co-formulated product by year-end 2013 for 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, "During 2012 we announced encouraging 6-month results from our first
ongoing Phase 3 Fabry monotherapy study, or Study 011, and established initial
human proof-of-concept for our Chaperone-Advanced Replacement Therapy, or
CHART, platform. Throughout 2013 our strong financial position will allows us
to advance our CHART programs for lysosomal storage diseases and to work
toward a potential NDA submission for migalastat HCl monotherapy for Fabry
disease. Given our dialogue with the Food and Drug Administration regarding
the pre-specified analysis plan for Study 011, we will remain blinded to the
12-month results until the third quarter of this year. We look forward to our
continued interactions with the agency to support a potential U.S. approval of
migalastat HCl monotherapy. We believe that our pharmacological chaperones, in
particular our CHART platform, have the potential to deliver next-generation
treatments to patients and create significant shareholder value for many years
to come."

Financial Highlights for Full-Year Ended December 31, 2012

  *Cash, cash equivalents, and marketable securities totaled $99.1 million at
    December 31, 2012 compared to $55.7 million at December 31, 2011.
  *Total revenue was $18.4 million compared to $21.4 million for the
    full-year 2011. The year-over-year decrease is attributed to a change in
    revenue recognition accounting under the expanded GlaxoSmithKline (GSK)
  *Total operating expenses were $71.3 million compared to $72.3 million in
    the full-year 2011 due to lower research and development expenses as well
    as a decrease in personnel costs.
  *Cash operating expenses net of cash reimbursements received under the GSK
    collaboration were $40.7 million, within the full-year 2012 guidance range
    of $37-43 million.
  *Net loss was $48.8 million, or $1.07 per share, compared to a net loss of
    $44.4 million, or $1.28 per share, for the full-year 2011.

2013 Financial Guidance

As previously announced, Amicus expects full-year 2013 operating expenses to
total between $52 million and $58 million, net of 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 potentially support a
    U.S. marketing application as well as global registration. Results were
    reported from the 6-month double-blind treatment period (Stage 1) and data
    from the 6-month open-label follow up period (Stage 2) are anticipated in
    the third quarter of 2013. The FDA has indicated that it will consider the
    entirety of the efficacy and safety data from Stage 1 and Stage 2 of Study
    011. Following the 12-month results, a meeting is anticipated with the FDA
    to discuss a U.S. approval pathway for migalastat HCl monotherapy.
  *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 is 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 enzyme in any patient receiving ERT for Fabry disease. Amicus and GSK
completed an open-label Phase 2 study (Study 013) to investigate a single oral
dose of migalastat HCl (150 mg or 450 mg) co-administered prior to ERT
(Fabrazyme® or Replagal®) in males with Fabry disease.

Based on the results from this study, the next chaperone-ERT combination study
for Fabry disease is being designed to investigate intravenous treatment of
migalastat HCl co-formulated with JCR Pharmaceutical Co. Ltd's proprietary
recombinant human alpha-Gal A enzyme (JR-051). Amicus and GSK, in
collaboration with JCR, are conducting IND-enabling studies of this
chaperone-ERT co-formulated product. An IND submission for this chaperone-ERT
co-formulated product is planned by year-end 2013 for entry into the clinic in
early 2014.

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. In
chaperone-advanced replacement therapy programs for Pompe disease, the
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, Myozyme/Lumizyme) in parallel with the
development of a next-generation ERT (AT2220 co-formulated with a proprietary
rhGAA enzyme). These investigational chaperone-advanced replacement therapies
have the potential to increase enzyme activity in muscle and other
disease-relevant tissues, improve glycogen reduction, and mitigate
immunogenicity compared to Myozyme/Lumizyme alone.

  *AT2220-IV Co-Administered with Marketed ERTs: Based on positive results
    from a Phase 2 co-administration study (Study 010) in Pompe patients,
    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. The upcoming clinical study will
    investigate multiple doses of AT2220-IV co-administered with
    Myozyme/Lumizyme every 2 weeks in treatment-naïve and ERT-experienced
    Pompe patients to characterize safety, PK, and anti-rhGAA antibody titers.
  *Next-Generation ERT (AT2220 Co-Formulated with a Proprietary Amicus ERT):
    Amicus entered into a contract with Laureate Pharmaceuticals for the
    manufacture of a proprietary rhGAA enzyme, which is being co-formulated
    with AT2220 as a next-generation ERT for Pompe disease. Through this
    investigational chaperone-advanced replacement therapy, 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.

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
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 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). Amicus is leveraging the CHART platform to
improve currently marketed ERTs through co-administration of a pharmacological
chaperone prior to ERT infusion, and to develop next-generation ERTs that
consist of a proprietary lysosomal enzyme therapy co-formulated with a
pharmacological chaperone. In a chaperone-advanced replacement therapy, a
unique pharmacological chaperone binds to a specific therapeutic enzyme,
stabilizing the enzyme in its properly folded and active form. This proposed
CHART mechanism may allow for enhanced tissue uptake, 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 ERTs.

About Migalastat HCl for Fabry Disease

Migalastat HCl is an investigational pharmacological chaperone migalastat 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 form.

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 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.

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 Section21E
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,
                      Three Months          Twelve Months         To
                      Ended December 31,    Ended December 31,    Dec.31,
                      2011       2012       2011       2012       2012
Research revenue       $3,970     $--       $14,794    $11,591    $57,493
Collaborationand     1,660      --         6,640      6,820      64,382
milestone revenue
Total revenue          5,630      --        21,434     18,411     121,875
Operating Expenses:                                            
Research and           14,401     11,047     50,856     50,273     315,893
General and            3,917      4,455      19,880     19,364     132,613
Restructuring charges  --         --         --         --         1,522
Impairment of          --         --         --         --         1,030
leasehold improvements
Depreciation and       342        421        1,585      1,705      11,768
In-process research    --         --         --         --         418
and development
Total operating        18,660     15,923     72,321     71,342     463,244
Loss from operations   (13,030)   (15,923)   (50,887)   (52,931)   (341,369)
Other income                                                   
Interest income        24         81         160        316        14,389
Interest expense       (27)       (12)       (148)      (89)       (2,422)
Change in fair value   742        2,594      2,764      653        1,553
of warrant liability
Other income           --         --         70         21         252
Loss before tax        (12,291)   (13,260)   (48,041)   (52,030)   (327,597)
Benefit from income    3,629      3,245      3,629      3,245      8,708
Net loss               (8,662)    (10,015)   (44,412)   (48,785)   (318,889)
Deemed dividend        --         --         --         --         (19,424)
Preferred stock        --         --         --         --         (802)
Net loss attributable  $(8,662)   $(10,015)  $(44,412)  $(48,785)  $(339,115)
to common stockholders
Net loss attributable
to commonstockholders $(0.25)    $(0.20)    $(1.28)    $(1.07)    
per common share –
basic and diluted
common shares          34,643,722 49,477,596 34,569,642 45,565,217 
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, December 31,
                                                    2011         2012
Current assets:                                                  
Cash and cash equivalents                            $25,668     $33,971
Investments in marketable securities                 30,034       65,151
Receivable due from GSK                              5,043        3,225
Prepaid expenses and other current assets            5,903        2,270
Total current assets                                 66,648       104,617
Property and equipment, less accumulated
depreciation and amortization of $9,507 and          2,438        5,029
$8,501at December 31, 2011 and 2012, respectively
Other non-current assets                             709          442
Total Assets                                         $69,795     $110,088
Liabilities and Stockholders' Equity                             
Current liabilities:                                             
Accounts payable and accrued expenses                $9,708      $8,845
Current portion of deferred reimbursements           8,504        --
Current portion of secured loan                      1,044        398
Total current liabilities                            19,256       9,243
Deferredreimbursements, less current portion        18,999       30,418
Warrant liability                                    1,948        908
Secured loan, less current portion                   --           299
Commitments and contingencies                                    
Stockholders' equity:                                            
Common stock, $.01 par value, 125,000,000 shares
authorized, 34,654,206 shares issued and outstanding 407          556
at December 31, 2011,49,631,672 shares issued and
outstanding at December 31, 2012
Additional paid-in capital                           299,285      387,539
Accumulated other comprehensive income               4            14
Deficit accumulated during the development stage     (270,104)    (318,889)
Total stockholders' equity                           29,592       69,220
Total Liabilities and Stockholders' Equity           $69,795     $110,088


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