Amicus Therapeutics Announces Second Quarter 2013 Financial Results

Amicus Therapeutics Announces Second Quarter 2013 Financial Results

Full-Year 2013 Net Cash Spend Guidance Reduced to $47 Million to $53 Million –
              Current Cash Expected to Fund Operations into 4Q14

Multiple Next-Generation Enzyme Replacement Therapies Advancing for Lysosomal
                               Storage Diseases

CRANBURY, N.J., Aug. 7, 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 second
quarter ended June 30, 2013. The Company also summarized recent and upcoming
milestones and reduced full-year 2013 net cash spend guidance.

Key Program Highlights and Upcoming Milestones:

  *Phase 2 chaperone-ERT co-administration study for Pompe disease expected
    to begin 2H13
  *Fabry chaperone-ERT co-formulated product anticipated to enter clinic in
    1H14
  *Next-generation ERTs for Pompe disease and Mucopolysaccharidosis Type I
    (MPS I) advancing in preclinical studies
  *12-month (Stage 2) data from Phase 3 Study 011 of migalastat HCl
    monotherapy for Fabry disease anticipated 4Q13 and top-line data from
    Study 012 expected 2H14 - U.S. new drug application (NDA) submission
    expected to include data from all clinical studies

John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics
stated, "During the second quarter we continued to build out our CHART
platform of chaperone-ERT combinations across the lysosomal storage diseases.
We expect to initiate our next Phase 2 Pompe co-administration study in the
second half of this year, as well as initial clinical studies of our
chaperone-ERT co-formulated product for Fabry disease in 2014. Our long-term
vision is to move multiple next-generation ERTs into the clinic over the next
several years. We also remain committed to advancing migalastat HCl
monotherapy toward a potential U.S. approval for Fabry patients who have
amenable mutations."

Financial Highlights for Second Quarter Ended June 30, 2013

  *Cash, cash equivalents, and marketable securities totaled $74.2 million at
    June 30, 2013 compared to $99.1 million at December 31, 2012.
  *Cash reimbursements received from GlaxoSmithKline (GSK) for shared
    development of migalastat HCl totaled $1.3 million compared to $4.3
    million in the second quarter 2012.
  *No revenue was reported due to a change in revenue recognition accounting
    under the expanded GSK collaboration. Total revenue of $10.6 million was
    recognized in the second quarter 2012.
  *Total operating expenses decreased to $16.0 million from $20.0 million in
    the second quarter 2012 due to lower expenses in research and development
    as well as general and administrative.
  *Net loss was $15.3 million, or $0.31 per share, compared to a net loss of
    $9.3 million, or $0.20 per share, for the second quarter 2012.

2013 Financial Guidance

Amicus expects full-year 2013 net cash spend to total between $47 million and
$53 million, compared to previous net cash spend guidance of $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 projects that the current
cash position and anticipated Fabry program reimbursements from GSK are
sufficient to fund operations into the fourth quarter of 2014.

William D. Baird, Chief Financial Officer of Amicus Therapeutics said, "We are
reducing our full-year 2013 net cash spend guidance to reflect
lower-than-anticipated cash spend during the first half of 2013 as well as
some adjustments to our budget for the remainder of the year. We are committed
to carefully managing our cash position to efficiently advance our programs."

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. Based on feedback from the FDA during a Type C
    meeting in the second quarter of 2013, Amicus expects to submit a new drug
    application (NDA) after data are available from both Study 011 and Study
    012. The 6-month results from Study 011 have been reported, 12-month data
    from Study 011 are anticipated in the fourth quarter of 2013, and results
    from Study 012 are anticipated in the second half of 2014.
    
  *Migalastat HCl in Combination with ERT: 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). 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. Amicus believes this approach
    has the potential to benefit all patients with Fabry disease. This
    chaperone-ERT co-formulated product is expected to enter the clinic in the
    first half of 2014.

CHART™ Programs for Pompe Disease and Mucopolysaccharidosis Type I (MPS I)

Outside the collaboration agreement with GSK, Amicus owns exclusive rights to
the rest of its pipeline and applications of its chaperone-advanced
replacement therapy (CHART™) platform technology. In each CHART program, a
unique pharmacological chaperone is designed to bind to and stabilize a
specific therapeutic enzyme in its properly folded and active form. These
next-generation therapies have the potential to allow for enhanced tissue
uptake of active enzyme, greater lysosomal activity, more reduction of
substrate, and lower immunogenicity compared to current standard of care ERTs.

  *AT2220-IV Co-Administered with Marketed ERTs for Pompe Disease: Amicus
    plans to conduct a Phase 2 repeat-dose clinical study to evaluate a novel
    intravenous formulation of AT2220 (AT2220-IV) co-administered with
    currently marketed recombinant human GAA (Myozyme/Lumizyme) in Pompe
    patients.The study is designed to build upon positive results from a
    single-dose Phase 2 co-administration study (Study 010). Amicus expects to
    initiate the repeat-dose study in the second half of 2013 and to report
    initial results in the first half of 2014.
    
  *Next-Generation ERTs for Pompe Disease and Mucopolysaccharidosis Type I
    (MPS I): Amicus is currently conducting preclinical studies to evaluate IV
    and subcutaneous administration of a proprietary recombinant human GAA
    enzyme co-formulated with AT2220 as a next-generation therapy for Pompe
    disease. In addition, a proprietary recombinant human alpha-L-iduronidase
    (rhIDUA) enzyme co-formulated with a novel pharmacological chaperone is in
    preclinical development for MPS I. In support of its development of a
    proprietary rhIDUA enzyme, Amicus has received a grant of up to $250,000
    from a private U.S.-based donor.

Conference Call and Webcast

Amicus Therapeutics will host a conference call and audio webcast today,
August 7, 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 webcast can also be accessed via the Investors section of the Amicus
Therapeutics corporate web site at http://www.amicusrx.com, 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. 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
26879608.

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.

Current CHART programs in development include the pharmacological chaperone
AT2220 co-administered with currently marketed ERTs in Phase 2 for Pompe
disease, as well as preclinical-stage next-generation ERTs for Pompe disease
and Mucopolysaccharidosis Type I (MPS I).

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
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.

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,"
"may," "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
(Unaudited)
(In thousands, except share and per share amounts)
                                                              
                                                              Period from
                                                              February 4,
                                                              2002
                                                              (inception)
                      Three Months         Six Months           To
                      Ended June 30,        Ended June 30,        June30,
                      2012       2013       2012       2013       2013
Revenue:                                                       
Research revenue       $5,477     $--       $11,591    $--       $57,493
Collaborationand      5,160      --         6,820      --         64,382
milestone revenue
Total revenue          10,637     $--       18,411     $--       121,875
                                                              
Operating Expenses:                                            
Research and           13,723     10,725     27,727     22,714     338,607
development
General and            5,819      4,830      9,914      9,653      142,266
administrative
Restructuring charges  --         --         --         --         1,522
Impairment of          --         --         --         --         1,030
leasehold improvements
Depreciation and       442        450        862        889        12,657
amortization
In-process research    --         --         --         --         418
and development
Total operating        19,984     16,005     38,503     33,256     496,500
expenses
Loss from operations   (9,347)    (16,005)   (20,092)   (33,256)   (374,625)
Other income                                                   
(expenses):
Interest income        116        46         143        111        14,500
Interest expense       (15)       (9)        (58)       (19)       (2,441)
Change in fair value   (118)      619        (2,494)    357        1,910
of warrant liability
Other income           21         --         21         --         252
Loss before tax        (9,343)    (15,349)   (22,480)   (32,807)   (360,404)
benefit
Income tax benefit     --         --         --         --         8,708
Net loss               (9,343)    (15,349)   (22,480)   (32,807)   (351,696)
Deemed dividend        --         --         --         --         (19,424)
Preferred stock        --         --         --         --         (802)
accretion
Net loss attributable  ($9,343)   ($15,349)  ($22,480)  ($32,807)  ($371,922)
to common stockholders
Net loss attributable
to common stockholders ($0.20)    ($0.31)    ($0.53)    ($0.66)    
per common share –
basic and diluted
                                                              
Weighted-average
common shares          46,870,067 49,621,188 42,103,642 49,621,188 
outstanding – basic
and diluted

                                                                   
Table 2                                                             
Amicus Therapeutics, Inc.
(a development stage company)
Consolidated Balance Sheets
(Unaudited)
(in thousands, except share and per share amounts)
                                                                   
                                                                   
                                                                   
                                                                   
                                                       December 31, June 30,
                                                       2012         2013
Assets:                                                             
Current assets:                                                     
Cash and cash equivalents                               $33,971      $23,476
Investments in marketable securities                    65,151       50,700
Receivable due from GSK                                 3,225        1,024
Prepaid expenses and other current assets               2,270        1,978
Total current assets                                    104,617      77,178
                                                                   
Property and equipment, less accumulated depreciation
and amortization of $8,501 and $9,321at December 31,   5,029        4,715
2012 and June 30, 2013, respectively
Other non-current assets                                442          442
Total Assets                                            $110,088     $82,335
                                                                   
Liabilities and Stockholders' Equity                                
Current liabilities:                                                
Accounts payable and accrued expenses                   $8,845       $9,018
Current portion of secured loan                         398          398
Warrant liability                                       --           551
Total current liabilities                               9,243        9,967
                                                                   
Deferredreimbursements                                 30,418       32,709
Warrant liability, non-current                          908          --
Secured loan, less current portion                      299          100
                                                                   
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 June 30, 2013
Additional paid-in capital                              387,539      390,696
Accumulated other comprehensive income                  14           3
Deficit accumulated during the development stage        (318,889)    (351,696)
Total stockholders' equity                              69,220       39,559
Total Liabilities and Stockholders' Equity              $110,088     $82,335

FOLD-G

CONTACT: Investors/Media:
         Sara Pellegrino
         spellegrino@amicusrx.com
         (609) 662-5044