Amicus Therapeutics Announces First Quarter 2014 Financial Results and Corporate Updates

Amicus Therapeutics Announces First Quarter 2014 Financial Results and
Corporate Updates

  Positive 12- and 24-Month Data From First Phase 3 Fabry Monotherapy Study
(Study 011) – Results from Second Phase 3 Fabry Monotherapy Study (Study 012)
                                Expected 3Q14

  3-in-3 Strategy to Advance 3 Next-Generation Enzyme Replacement Therapies
                      (ERTs) Into Clinic in Next 3 Years

CRANBURY, N.J., May 5, 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 first
quarter ended March 31, 2014. 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, "Last week was a momentous occasion for Amicus as we announced
very positive 12- and 24-month data from our first Phase 3 Fabry monotherapy
study. The feedback from investigators and, most especially, Fabry patients,
has been so very enthusiastic. Moving migalastat forward toward
commercialization is an important objective for this company and will have our
relentless focus in the months ahead. During the first quarter we also made
great progress with the execution of our 3-in-3 strategy to advance 3
next-generation ERTs into the clinic over the next three years, with lead
programs in Fabry, Pompe, and MPS I. We believe that the strength of our
programs and the breadth of our technology platforms will create significant
value for our shareholders throughout 2014 and hopefully for many years to

Financial Highlights for First Quarter Ended March 31, 2014

  *Cash, cash equivalents, and marketable securities totaled $71.6 million at
    March 31, 2014 compared to $82.0 million at December 31, 2013.
  *Total operating expenses decreased to $16.1 million compared to $17.3
    million for the first quarter 2013 due to decreases in personnel and
    contract research costs.
  *Net cash spend was $10.4 million, compared to $14.4 million for the first
    quarter 2013.
  *Net loss was $15.9 million, or $0.25 per share, compared to a net loss of
    $17.5 million, or $0.35 per share, for the first quarter 2013.

2014 Financial Guidance

Cash, cash equivalents, and marketable securities totaled $71.6 million at
March 31, 2014 compared to $82.0 million at December 31, 2013. 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

Migalastat Monotherapy and Next-Generation ERT for Fabry Disease

Amicus controls global development and commercialization of its
pharmacological chaperone migalastat HCl ("migalastat") monotherapy and its
next-generation ERT (migalastat co-formulated with ERT) for Fabry disease. As
a monotherapy, migalastat is designed to bind and stabilize alpha-Gal A enzyme
in patients with amenable mutations. In combination with ERT, migalastat is
designed to bind and stabilize infused alpha-Gal A enzyme, independent of a
patient's genetic mutation. Between these approaches Amicus believes
migalastat has the potential to benefit all patients with Fabry disease.

Migalastat Monotherapy

Migalastat monotherapy is being investigated in two Phase 3 registration
studies (Study 011 and Study 012) and an open-label extension study (Study
041) in Fabry patients with amenable mutations. Interim 6-month data and
positive 12- and 24-month data from Study 011 have been reported. Migalastat
demonstrated a statistically significant (p=0.013) and durable reduction in
kidney interstitial capillary GL-3 on the 12-month pre-specified primary
analysis in Fabry patients with amenable mutations in a GLP-validated human
embryonic kidney (HEK) cell-based in vitro assay ("GLP HEK amenable"). Kidney
function, measured by glomerular filtration rate (GFR), also remained stable
following up to 24 months of treatment with migalastat in patients with GLP
HEK amenable mutations.

In Study 012, the primary analysis will evaluate GFR over 18 months of
treatment with migalastat compared to enzyme replacement therapy (ERT), the
current standard of care for Fabry disease. Top-line data from Study 012 are
on track to be reported in the third quarter of 2014. Pending positive data
from Study 012, Amicus expects to meet with regulatory authorities to discuss
data from both studies and determine the fastest registration pathway for
migalastat monotherapy.

Next-Generation Fabry ERT

Amicus has completed a Phase 2 clinical study (Study 013) of migalastat
co-administered with currently approved ERTs for Fabry disease (Fabrazyme^®
and Replagal^®) as well as preclinical studies of migalastat 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 quarter of 2014 Amicus initiated a Phase 1 study to assess the
pharmacokinetics of an intravenous formulation of migalastat 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 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 ERTs for Pompe and MPS I

Amicus also owns exclusive global rights to its next-generation ERTs for Pompe
and MPS I, 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 of create next-generation therapies that are designed to enhance
tissue uptake of active enzyme, improve lysosomal activity and substrate
reduction, 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. This differentiated Pompe
ERT, designated AT-B200, has a unique carbohydrate structure and may be
further optimized through co-formulation with a pharmacological chaperone to
improve enzyme stability and tolerability, and by applying the Company's
peptide tagging technology for better targeting. In preclinical studies
AT-B200 has shown superior tissue uptake and activity when compared to current
standard of care. Longer term preclinical proof-of-concept studies are
currently underway to evaluate AT-B200. Manufacturing scale up activities are
also on track to provide sufficient supply of AT-B200 for IND-enabling
toxicology studies as well as a Phase 1/2 clinical study that is expected to
begin in 2015.

Next-Generation ERT for MPS I

Amicus is also developing 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

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 is being 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, May
5, 2014 at 5:00 p.m. ET to discuss first quarter 2014 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 as a monotherapy and in combination with enzyme
replacement therapy (ERT) for Fabry disease; and AT2220 (duvoglustat) 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, 2013. 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            To
                                         Ended March 31,         March 31,
                                         2013        2014        2014
Research revenue                          $--       $456        $58,312
Collaboration and milestone revenue       --        --          64,382
Total revenue                             --        456         122,694
Operating Expenses:                                             
Research and development                  11,989      9,992       367,829
General and administrative                4,823       5,176       156,682
Changes in contingent consideration       --         505         505
Restructuring charges                     --          (8)         3,502
Impairment of leasehold improvements      --          --          1,030
Depreciation and amortization             439         412         13,899
In-process research and development       --          --          418
Total operating expenses                  17,251      16,077      543,865
Loss from operations                      (17,251)    (15,621)    (421,171)
Other income (expenses):                                        
Interest income                           65          42          14,605
Interest expense                          (10)        (355)       (2,823)
Change in fair value of warrant liability (262)       --          2,461
Other (expense)/income                    --          (9)         243
Loss before tax benefit                   (17,458)    (15,943)    (406,685)
Income tax benefit                        --          --          12,220
Net loss                                  (17,458)    (15,943)    (394,465)
Deemed dividend                           --          --          (19,424)
Preferred stock accretion                 --          --          (802)
Net loss attributable to common           $(17,458) $(15,943) $(414,691)
Net loss attributable to common
stockholders per common share – basic and $(0.35)   $(0.25)   
Weighted-average common shares            49,621,188  64,353,952  
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,
                                                       2013         2014
Current assets:                                                     
Cash and cash equivalents                               $43,640      $27,533
Investments in marketable securities                    38,360       44,121
Receivable due from GSK                                 759          38
Prepaid expenses and other current assets               5,519        1,758
Total current assets                                    88,278       73,450
Property and equipment, less accumulated depreciation
and amortization of$9,973 and $10,385at December 31,  4,120        3,748
2013 and March 31, 2014, respectively
In-process research & development                       23,000       23,000
Goodwill                                                11,613       11,613
Other non-current assets                                552          546
Total Assets                                            $127,563     $112,357
Liabilities and Stockholders' Equity                                
Current liabilities:                                                
Accounts payable and accrued expenses                   $10,162      $9,167
Current portion of secured loan                         299          199
Total current liabilities                               10,461       9,366
Deferredreimbursements                                 36,677       36,677
Secured loan, less current portion                      14,174       14,216
Contingent consideration payable                        10,600       11,105
Deferred tax liability                                  9,186        9,186
Other non-current liability                             714          723
Commitments and contingencies                                       
Stockholders' equity:                                               
Common stock, $.01 par value, 125,000,000 shares
authorized, 61,975,416 shares issued and outstanding at
December 31, 2013, 125,000,000 shares authorized,       679          703
64,340,259 shares issued and outstanding at March 31,
Additional paid-in capital                              423,593      424,844
Accumulated other comprehensive income                  1            2
Deficit accumulated during the development stage        (378,522)    (394,465)
Total stockholders' equity                             45,751       31,084
Total Liabilities and Stockholders' Equity              $127,563     $112,357


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