Amicus Therapeutics Announces Full-Year 2013 Financial Results and Corporate Updates

Amicus Therapeutics Announces Full-Year 2013 Financial Results and Corporate

  Executing 3-in-3 Strategy to Advance 3 Next-Generation ERTs into Clinic in
                                 Next 3 Years

           Reiterating FY14 Cash Spend Guidance of $54-$59 Million

CRANBURY, N.J., March 3, 2014 (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, 2013. The Company also provided program updates and
reiterated full-year 2014 operating expense guidance.

John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics,
Inc., stated, "During 2013 we focused on strengthening our biologics business
strategy to develop next-generation ERTs for patients with lysosomal storage
diseases. Through our purchase of Callidus Biopharma, we have acquired a
proprietary Pompe ERT as well as a peptide tagging technology that is
complementary to our CHART platform. We believe that these technologies
together provide a unique tool set to enhance enzyme activity, increase enzyme
uptake into tissues, and potentially address the tolerability and
immunogenicity associated with current ERTs. During 2014 we are strongly
positioned and well capitalized to execute our 3-in-3 strategy to advance
three next-generation ERTs into the clinical in the next three years, with
lead programs in Fabry, Pompe and MPS I."

Financial Highlights for Full Year Quarter Ended December 31, 2013

  *Cash, cash equivalents, and marketable securities totaled $82.0 million at
    December 31, 2013 compared to $99.1 million at December 31, 2012.
  *Total operating expenses decreased to $64.5 million compared to $71.3
    million for the full-year 2012 primarily due to decreases in clinical
    development costs on the Fabry monotherapy program.
  *Net cash spend was $47.1 million, within the full-year 2013 guidance range
    of $47-53 million.
  *Net loss was $59.6 million, or $1.16 per share, compared to a net loss of
    $48.8 million, or $1.07 per share, for the full-year 2012.

2014 Financial Guidance

Cash, cash equivalents, and marketable securities totaled $82.0 million at
December 31, 2013 compared to $99.1 million at December 31, 2012. The
Company's balance sheet was strengthened in the fourth quarter of 2013 with a
$15.0 million equity financing and a $25.0 million debt financing under which
$15.0 million was drawn and $10.0 million remains available. Amicus continues
to expect full-year 2014 net cash spend between $54 million and $59 million.
The current cash position is projected to fund operations into the second half
of 2015.

Program Updates

Amicus owns exclusive global rights to its next-generation ERTs, as well as
all applications of its Chaperone-Advanced Replacement Therapy (CHART™) and
enzyme targeting technology platforms. 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. Through its
purchase of Callidus Biopharma Amicus has also acquired a differentiated
peptide tagging technology that can be used to uniquely engineer bio-better
ERTs. These platform technologies provide a complementary tool set to design
next-generation therapies for enhanced tissue uptake of active enzyme, greater
lysosomal activity, more reduction of substrate, and potentially address the
tolerability and immunogenicity associated with currently marketed ERTs.

Next-Generation ERT for Pompe Disease

Amicus is advancing a recombinant human acid alpha-glucosidase (rhGAA) for
Pompe disease into late preclinical development. The Company's acquisition of
Callidus Biopharma, brings a differentiated Pompe ERT, designated AT-B200,
with a unique carbohydrate structure. In preclinical studies AT-B200 has shown
superior tissue uptake and activity when compared to current standard of care.
This ERT may be further optimized through co-formulation with Amicus'
pharmacological chaperone AT2220 to improve enzyme stability and tolerability,
and by applying the Company's peptide tagging technology for better targeting.

Next-Generation ERT for Fabry Disease

In combination with ERT, Amicus' pharmacological chaperone migalastat HCl is
designed to bind and stabilize the 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.

Amicus has completed a Phase 2 clinical study (Study 013) of migalastat HCl
co-administered with currently approved ERTs for Fabry disease (Fabrazyme^®
and Replagal^®) as well as preclinical studies of migalastat HCl co-formulated
with a proprietary investigational ERT for Fabry disease (JCR Pharmaceutical
Co Ltd's JR-051). JR-051 is a human recombinant alpha-Gal A enzyme that is
designed to be biosimilar to Fabrazyme. Positive results from these clinical
and preclinical studies demonstrated increased enzyme activity in plasma and
greater enzyme uptake into tissues in the presence of the chaperone compared
to any of these ERTs alone^1,2.

In the first half of 2014 Amicus plans to conduct a Phase 1 study to assess
the pharmacokinetics of an intravenous formulation of migalastat HCl in
healthy volunteers to identify the optimal dose for co-formulation with ERT.
In the second half of 2014, Amicus expects to initiate a Phase 1/2 study to
evaluate migalastat HCl co-formulated with JR-051. Amicus is currently
evaluating its long-term strategy for supplying late-stage clinical and
commercial ERT, which may include developing or in-licensing a recombinant
alpha-Gal A enzyme comparable to JR-051.

Next-Generation ERT for MPS I

Amicus is leveraging its CHART platform to develop a proprietary human
recombinant alpha-L-iduronidase (rhIDUA) enzyme for MPS I. In support of its
development of this next-generation ERT, Amicus has received funding of up to
$250,000 from a private U.S.-based donor that provides medical research grants
to find better treatments and cures for rare genetic disorders, including
lysosomal storage diseases.

Migalastat HCl Monotherapy for Fabry Disease

Migalastat HCl monotherapy is being investigated in two ongoing Phase 3
studies for Fabry patients with amenable mutations. Interim 6-month data from
the first ongoing Phase 3 study (Study 011) have been reported, and 12- and
24-month data from this study are anticipated in the second quarter of 2014.
Top-line, 18-month clinical data from the second ongoing Phase 3 study (Study
012) are expected in the second half of 2014.

Novel Small Molecules for Parkinson's Disease

In September 2013 Amicus and Biogen Idec entered a multi-year collaboration to
discover of a new class of small molecules that target the glucocerobrosidase
(GCase) enzyme for further development and commercialization by Biogen Idec.
Biogen Idec is responsible for funding all discovery, development, and
commercialization activities. Amicus will be reimbursed for all full-time
employees working on the project. In addition Amicus is eligible to receive
development and regulatory milestones, as well as modest royalties on global
net sales.

Conference Call and Webcast

Amicus Therapeutics will host a conference call and audio webcast today, March
3, 2014 at 5:00 p.m. ET to discuss full-year 2013 financial results and
program updates. 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, 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

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 proprietary next-generation
therapies that consist of lysosomal enzymes co-formulated with pharmacological

^1Bichet, et al., American Society of Human Genetics, November 2012

^2Benjamin, et al., Molecular Therapy, April 2012

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 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,
                 Three Months            Twelve Months           To
                 Ended December 31,      Ended December 31,      Dec.31,
                 2012        2013        2012        2013        2013
Research revenue  $--        $324        $11,591     $363        $57,856
Collaborationand --         --         6,820      --         64,382
milestone revenue
Total revenue     --         324        18,411     363        122,238
Research and      11,047     9,120      50,273     41,944     357,837
General and       4,455      4,605      19,364     18,893     151,506
Restructuring     --         1,988      --         1,988      3,510
Impairment of
leasehold         --         --         --         --         1,030
Depreciation and  421        401        1,705      1,719      13,487
research and      --         --         --         --         418
Total operating   15,923     16,114     71,342     64,544     527,788
Loss from         (15,923)   (15,790)   (52,931)   (64,181)   (405,550)
Other income                                                  
Interest income   81         27         316        174        14,563
Interest expense  (12)       (20)       (89)       (46)       (2,468)
Change in fair
value of warrant  2,594      34         653        908        2,461
Other income      --         --         21         --         252
Loss before tax   (13,260)   (15,749)   (52,030)   (63,145)   (390,742)
Benefit from      3,245      3,512      3,245      3,512      12,220
income taxes
Net loss          (10,015)   (12,237)   (48,785)   (59,633)   (378,522)
Deemed dividend   --         --         --         --         (19,424)
Preferred stock   --         --         --         --         (802)
Net loss
attributable to   $(10,015) $(12,237) $(48,785) $(59,633) $(398,748)
Net loss
attributable to
common            $(0.20)   $(0.22)   $(1.07)   $(1.16)   
stockholders per
common share –
basic and diluted
common shares     49,477,596  56,173,260  45,565,217  51,286,059  
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,
                                                    2012         2013
Current assets:                                                  
Cash and cash equivalents                            $33,971      $43,640
Investments in marketable securities                 65,151       38,360
Receivable due from GSK                              3,225        759
Prepaid expenses and other current assets            2,270        5,519
Total current assets                                 104,617      88,278
Property and equipment, less accumulated
depreciation and amortization of $8,501 and          5,029        4,120
$9,973at December 31, 2012 and 2013, respectively
In-process research & development                    —            23,000
Goodwill                                             —            11,613
Other non-current assets                             442          552
Total Assets                                         $110,088     $127,563
Liabilities and Stockholders' Equity                             
Current liabilities:                                             
Accounts payable and accrued expenses                $8,845       $10,162
Current portion of secured loan                      398          299
Total current liabilities                            19,256       9,243
Deferredreimbursements, less current portion        30,418       36,677
Warrant liability                                    908          —
Secured loan, less current portion                   299          14,174
Contingent consideration payable                     —            10,600
Deferred tax liability                               —            9,186
Other non-current liability                          —            714
Commitments and contingencies                                    
Stockholders' equity:                                            
Common stock, $.01 par value, 125,000,000 shares
authorized, 49,631,672 shares issued and outstanding 556          679
at December 31, 2012,61,975,416 shares issued and
outstanding at December 31, 2013
Additional paid-in capital                           387,539      423,593
Accumulated other comprehensive income               14           1
Deficit accumulated during the development stage     (318,889)   (378,522)
Total stockholders' equity                           69,220       45,751
Total Liabilities and Stockholders' Equity           $110,088     $127,563


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