Amicus Therapeutics Announces Third Quarter 2012 Financial Results & Corporate Updates

Amicus Therapeutics Announces Third Quarter 2012 Financial Results & Corporate
Updates

6-Month Results Expected 4Q12 and 12-Month Results Anticipated 1H13 From Phase
                        3 Fabry Monotherapy Study 011

     Additional Human Proof-of-Concept Demonstrated for Chaperone-Enzyme
                Replacement Therapy (ERT) Combination Platform

CRANBURY, N.J., Nov. 5, 2012 (GLOBE NEWSWIRE) -- Amicus Therapeutics
(Nasdaq:FOLD), a biopharmaceutical company at the forefront of developing
therapies for rare and orphan diseases, today announced financial results for
the third quarter ended September 30, 2012. The Company also summarized recent
and upcoming milestones and reiterated full-year 2012 operating expense
guidance.

Key Highlights and Upcoming Milestones

  *6-month primary endpoint results expected 4Q12 and 12-month data
    anticipated 1H13 from first Phase 3 Fabry monotherapy study (Study 011)
    
  *Achieved target enrollment in Second Phase 3 Fabry monotherapy study
    (Study 012)
    
  *Announced updated results from Phase 2 extension study (Study 205) to
    evaluate investigational migalastat HCl monotherapy
    
  *Completed enrollment in Phase 2 Study 013 to investigate migalastat HCl
    co-administered with ERT - updated results to be presented at American
    Society of Human Genetics (ASHG) on November 8, 2012
    
  *Announced positive Pompe program updates - additional preliminary results
    for AT2220-ERT co-administration from Phase 2 Study 010 and ex vivo
    studies on ERT-related immunogenicity – Study 010 results from all 4
    cohorts anticipated 4Q12

John F. Crowley, Chairman and Chief Executive Officer of Amicus, stated, "The
third quarter 2012 was excellent for Amicus. We continued to demonstrate our
operational capabilities in global clinical research in rare and orphan
diseases through our advancement of multiple Phase 2 and Phase 3 studies in
Fabry and Pompe disease. We are especially pleased with the recruitment
success in our second Phase 3 Fabry Study 012. Finally, further clinical and
preclinical proof-of-concept evaluating the direct combination of
pharmacological chaperones with ERTs to both enhance ERT stability and reduce
ERT immunogenicity will be a cornerstone of our pipeline and shareholder value
going forward."

3Q12 Financial Results

The Company's third quarter 2012 financial statements reflect updated revenue
recognition accounting in conjunction with the expanded GSK collaboration
(entered in July 2012). Payments received from GSK under the agreement are now
being recorded in the "deferred reimbursements" account on the balance sheet.
On September 30, 2012, the deferred reimbursements balance was $27.2 million.
This amount included $4.5 million in cash reimbursed for shared global
development costs of migalastat HCl during the third quarter 2012, and the
$22.7 million unrecognized balance of the upfront license payment and premium
on equity. Prior to July 1, 2012, quarterly cash reimbursements were
recognized as "research revenue" and the upfront payment was amortized each
quarter in "collaboration revenue."

For the three months ended September 30, 2011, Amicus reported total revenue
of $5.8 million, including $4.1 million in research revenue and $1.7 million
in collaboration revenue. More information on the updated revenue recognition
accounting, which does not impact cash, will be contained in the Company's
Form 10-Q for the quarter ended September 30, 2012.

Total operating expenses for the third quarter 2012 totaled $16.9 million
compared to $18.9 million in the year-ago period on lower research and
development expenses. Net loss attributable to common stockholders in the
third quarter 2012 was $16.3 million, or $0.34 per share, compared to a net
loss of $9.8 million, or $0.28 per share, in the year-ago period. The wider
net loss versus the year-ago period is attributed to the change in revenue
recognition under the expanded GSK collaboration.

Financial Guidance

Cash, cash equivalents, and marketable securities totaled $106.2 million at
September 30, 2012 compared to $95.8 million at June 30, 2012 and $55.7
million at December 31, 2011. The current cash position reflects an $18.6
million equity investment from GSK related to the expanded collaboration and a
$3.5 million cash milestone payment received from GSK in the third quarter
2012. Amicus expects to end 2012 with at least $90 million in cash, cash
equivalents and marketable securities which is expected to fund its current
operating plan beyond 2013. This projection includes quarterly reimbursement
from GSK for shared development costs for migalastat HCl.

Amicus continues to expect full-year 2012 operating expenses within the higher
end of the previously disclosed guidance range of $37 million to $43 million,
net of anticipated cost sharing under the expanded Fabry disease collaboration
with GSK. Amicus and GSK are funding 25% and 75% of the development costs,
respectively, for migalastat HCl monotherapy and co-administration for
full-year 2012. During the second half of 2012, Amicus and GSK will be
responsible for 40% and 60% of the preclinical development costs,
respectively, for the co-formulated chaperone-ERT product. Amicus will be
responsible for all U.S. commercial activities for migalastat HCl upon
approval, including pricing, marketing, patient access and reimbursement.

Program Updates

Amicus and GSK are co-developing all formulations of migalastat HCl under a
global Fabry collaboration. 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. Outside the GSK collaboration, Amicus owns exclusive rights
to the rest of its pipeline and applications of its platform technology.

Migalastat HCl Monotherapy for Fabry Disease

Migalastat HCl monotherapy is in Phase 3 development (Study 011 and Study 012)
for Fabry patients with genetic mutations that are amenable to this chaperone
monotherapy, as determined by a cell-based assay. Both studies are
investigating oral migalastat HCl monotherapy 150 mg, dosed every-other-day
(QOD).

Study 011 is a randomized, placebo-controlled study. During the fourth quarter
2012, Amicus and GSK expect to announce the primary endpoint analysis, based
on interstitial capillary globotriaosylceramide (GL-3) as measured in kidney
biopsy at 6 months. Results from the 6-month follow-up period in Study 011 are
expected in the first half of 2013, which will include 12 months on migalastat
HCl in the treatment group and 6 months on migalastat HCl in the
placebo-crossover group.

Study 012 is the first clinical study to compare oral migalastat HCl to
standard-of-care ERTs (Fabrazyme^® and Replagal^®). The open-label study
randomized males and females with Fabry disease, who either to switch to
migalastat HCl or remain on ERT. Amicus and GSK achieved target enrollment in
Study 012 ahead of schedule. The primary outcome measure is renal function
assessed by Glomerular Filtration Rate (GFR) at 18 months.

Amicus and GSK also continue to evaluate patients who completed a previous
Phase 2 study in an ongoing Phase 2 extension study (Study 205) of migalastat
HCl. Updated results from Study 205 were presented at the American Society of
Nephrology (ASN) Kidney Week 2012. A total of 17 patients completed Study 205
after a median treatment duration of 5.2 years (4.7 to 6.4 years) with
migalastat HCl in the Phase 2 and Phase 2 extension studies. 16 of these
patients are currently enrolled in a separate open-label extension study
(MGM116041).

Migalastat HCl in Combination with ERT for Fabry Disease

During the third quarter 2012, Amicus and GSK completed enrollment in an
open-label Phase 2 study (Study 013) to investigate the effects of a single
oral dose of migalastat HCl (150 mg or 450 mg) co-administered prior to ERT
(Fabrazyme or Replagal) in 23 males with Fabry disease. Positive preliminary
results were announced in the first quarter 2012 and updated data will be
presented at the American Society of Human Genetics (ASHG). Amicus and GSK,
along with GSK's collaborator JCR Pharmaceutical Co. Ltd, also continue to
advance preclinical studies of migalastat HCl co-formulated with JCR
Pharmaceutical Co. Ltd's proprietary investigational ERT (JR-051, recombinant
human alpha-Gal A enzyme).

AT2220-ERT Co-Administration for Pompe Disease

In October 2012, Amicus announced additional positive preliminary results from
a Phase 2 open-label study (Study 010) to investigate four ascending dose
cohorts of the pharmacological chaperone AT2220 co-administered with ERT
(Myozyme^® and Lumizyme^®) for Pompe disease. In the first 3 dose cohorts,
AT2220-ERT co-administration increased Pompe enzyme (recombinant GAA)
activity. Study 010 is fully enrolled and results from Cohort 4 are
anticipated by year-end. In parallel with Study 010, initial ex vivo studies
completed using T cells from 50 healthy donor blood samples demonstrated that
the addition of AT2220 may reduce the immunogenicity of Myozyme and Lumizyme.
Results from these studies may help guide further investigation of the effects
of AT2220 on immune response to ERT in future clinical studies.

Conference Call and Webcast

Amicus Therapeutics will host a conference call and audio/visual webcast
today, November 5, 2012 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 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.

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 http://www.amicusrx.com. 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 64424986.

About Amicus Therapeutics

Amicus Therapeutics (Nasdaq:FOLD) is a biopharmaceutical company at the
forefront of developing therapies for rare diseases. The Company is developing
orally-administered, 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 Migalastat HCl for Fabry Disease

Amicus and GSK are co-developing all formulations of migalastat HCl under a
global Fabry collaboration. 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. As a monotherapy, migalastat HCl is designed to bind to and
stabilize, or "chaperone" alpha-galactosidase A (alpha-Gal A) enzyme in
patients 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 disease that is currently
estimated to affect approximately 5,000 to 10,000 people worldwide. Fabry
Disease is caused by deficiency of an enzyme called alpha-galactosidase A
(alpha-Gal A). The role of alpha-Gal A within the body is to break down a
complex lipid called globotriaosylceramide (GL-3). Reduced or absent levels of
alpha-Gal A activity leads to the accumulation of GL-3 in the affected
tissues, including the central nervous system, heart, kidneys, and skin. This
accumulation of GL-3 is believed to cause the various symptoms of Fabry
disease, including pain, kidney failure, and increased risk of heart disorders
and stroke.

About AT2220 for Pompe Disease

AT2220 is an investigational, orally-administered pharmacological chaperone
owned exclusively by Amicus. In published preclinical studies, AT2220-ERT
co-administration resulted in significant increases in muscle rhGAA levels and
decreases in glycogen levels in a mouse model of Pompe disease. Preclinical
results to date also suggest that AT2220-ERT co-administration may mitigate
ERT-induced immunogenicity by stabilizing the enzyme in its properly folded
and active form.

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," "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 Quarterly Report on Form
10-Q for the quarter ended June 30, 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          Nine Months          To
                      Ended September 30,   Ended September 30,   Sept.30,
                      2011       2012       2011       2012       2012
Revenue:                                                       
Research revenue       $4,138   $--       $10,824   $11,591   $57,493
Collaborationand      1,660     --         4,980      6,820      64,382
milestone revenue
Total revenue          $ 5,798   $--       15,804    18,411     121,875
                                                              
Operating Expenses:                                            
Research and           13,711     11,499     36,455    39,226    304,846
development
General and            4,841      4,995      15,963     14,909     128,158
administrative
Restructuring charges  --         --         --         --         1,522
Impairment of          --        --        --        --        1,030
leasehold improvements
Depreciation and       380        422        1,243      1,284      11,347
amortization
In-process research    --        --        --        --        418
and development
Total operating        18,932     16,916     53,661     55,419     447,321
expenses
Loss from operations   (13,134)   (16,916)   (37,857)   (37,008)   (325,446)
Other income                                                   
(expenses):
Interest income        31         92         136        235        14,308
Interest expense       (32)       (19)       (121)      (77)       (2,410)
Change in fair value   3,376     553       2,022     (1,941)   (1,041)
of warrant liability
Other income           --         --         70        21         252
Loss before tax        (9,759)    (16,290)   (35,750)   (38,770)   (314,337)
benefit
Benefit from income    --         --         --         --         5,463
taxes
Net loss               (9,759)    (16,290)   (35,750)   (38,770)   (308,874)
Deemed dividend        --         --         --         --         (19,424)
Preferred stock        --         --         --         --         (802)
accretion
Net loss attributable  $ (9,759)  $ (16,290) $(35,750) $(38,770) $(329,100)
to common stockholders
Net loss attributable
to commonstockholders $(0.28)  $(0.34)  $(1.03)   $(0.88)   
per common share –
basic and diluted
Weighted-average
common shares          34,979,702 48,513,647 34,544,768 44,255,885 
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, September 30,
                                                   2011         2012
Assets:                                                         
Current assets:                                                 
Cash and cash equivalents                           $25,668     $24,273
Investments in marketable securities                30,034       81,942
Receivable due from GSK                             5,043        3,184
Prepaid expenses and other current assets           5,903        3,077
Total current assets                                66,648       112,476
                                                               
Property and equipment, less accumulated
depreciation and amortization of $9,507 and         2,438        5,293
$8,080at December 31, 2011 and September 30, 2012,
respectively
Other non-current assets                            709          442
Total Assets                                        $69,795     $118,211
                                                               
Liabilities and Stockholders' Equity                            
Current liabilities:                                            
Accounts payable and accrued expenses               $9,708      $9,428
Current portion of deferred reimbursements          8,504        —
Current portion of secured loan                     1,044        502
Total current liabilities                           19,256       9,930
                                                               
Deferredreimbursements, less current portion       18,999       27,235
Warrant liability                                   1,948        3,889
Secured loan, less current portion                  —            398
                                                               
Commitments and contingencies                                   
                                                               
Stockholders' equity:                                           
Common stock, $.01 par value, 125,000,000 shares
authorized, 34,654,206 shares issued and                        
outstanding at December 31, 2011, 125,000,000
shares
authorized, 49,360,659 shares issued and           407          554
outstanding at September 30, 2012
Additional paid-in capital                          299,285      385,042
Accumulated other comprehensive income              4            37
Deficit accumulated during the development stage    (270,104)    (308,874)
Total stockholders' equity                         29,592       76,759
Total Liabilities and Stockholders' Equity          $69,795     $118,211

FOLD–G

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