Amicus Therapeutics Announces Third Quarter 2009 Financial Results and Strategic Business Updates
Amicus Reacquires Global Development and Commercialization Rights to Amigal(TM), Plicera(TM) and AT2220 Through a Mutual Termination Agreement with Shire Amicus Identifies Strategic Priorities and Implements Workforce Reduction
CRANBURY, N.J., Oct. 29 /PRNewswire-FirstCall/ --
Amicus Therapeutics (NASDAQ:FOLD)
today announced financial results for the third quarter 2009.
Additionally, Amicus also today announced that it has reacquired
all global development and commercialization rights from its
partner, Shire, for its lead lysosomal
storage disease programs as a result of the parties' mutual
termination of their collaboration. The Company also announced a
workforce reduction of approximately 20 percent and provided cash
guidance into the second half of 2011.
Amicus/Shire Collaboration Mutual Termination:
Amicus and Shire have reached a mutual termination agreement
with respect to their collaboration to jointly develop Amicus'
three lead pharmacological chaperone compounds for the treatment of
lysosomal storage disorders, effective immediately. As a result of
this agreement, Amicus has regained exclusive worldwide rights to
develop and commercialize Amigal(TM)
(migalastat hydrochloride), Plicera
(TM) (afegostat tartrate) and AT2220
(1-deoxynojirimycin HCl) outside of the United
States (U.S.) and now owns worldwide rights for these programs.
Shire will pay Amicus $5.2 million in the fourth quarter as full
and final payment for any amounts that may be due to the Company
under the collaboration, and both parties will be relieved of all
other future obligations, financial or otherwise.
John F. Crowley, President and CEO of Amicus Therapeutics
stated, "Shire has been an excellent partner to work with during
the past two years. However, reacquiring the ex-U.S. commercial
rights to Amigal furthers our desire to enhance the value of that
program for our shareholders. Since Shire and Amicus mutually
concluded that Plicera will not move forward to Phase 3 development
in Gaucher disease, having global rights to Amigal was of critical
importance to Amicus."
Amicus will continue to manage global clinical operations for
Amigal Phase 3 development and expects to initiate more than thirty
investigational sites worldwide. As previously announced, the first
global Phase 3 Amigal study is a 60 patient randomized,
double-blind placebo controlled six month study intended to support
U.S. registration. The primary surrogate endpoint agreed to with
the U.S. Food and Drug Administration (FDA) is a histological
evaluation of the change in GL-3 levels in the interstitial
capillary cells of the kidney. The first patient was randomized
into this study in mid-October. The Company estimates it will
complete enrollment for this trial by the end of 2010 and expects
to have data available in mid-2011. A separate Phase 3 study for
registration in the European Union comparing Amigal with
Fabrazyme® is required by the
European Medicines Agency (EMEA). Amicus expects to provide a
further update on that proposed study for the EMEA in 2010 as the
supply constraints with Fabrazyme become clearer.
Key Strategic Priorities and Workforce Reduction:
As part of its strategic business plan, Amicus will focus its
resources in three key areas:
-- The Phase 3 development of Amigal for Fabry disease;
-- Advancement of its lead pre-clinical programs that utilize its
pharmacological chaperone technology for the treatment of diseases of
neurodegeneration. The Company has an advanced pre-clinical program
in Parkinson's disease, as well as an earlier stage pre-clinical
program in an as yet undisclosed neurodegenerative target disease; and
-- Advancement of pharmacological chaperones used in combination with
enzyme replacement therapies (ERT) for the treatment of lysosomal
storage diseases.
"By focusing our resources on these three value creating centers
within Amicus, we will balance the execution of our global late
stage clinical program in Fabry disease with continued significant
investment with the chaperone platform in diseases of
neurodegeneration, which continue to yield very exciting near-term
and long-term potential value for the Company. The chaperone-ERT
combination approach is also one of potentially significant value
for Amicus shareholders as a further extension of our technology,"
said John F. Crowley.
The Company also announced today a work-force reduction of
approximately 20 percent, or 26 employees, as a part of a corporate
restructuring, with reductions occurring across all levels and
organizations within the Company. In addition, Amicus is
terminating its relationship with approximately 17 contractors
currently working at the Company. Amicus is taking this step to
reduce costs and to align its resources with its key strategic
priorities. The Company estimates that it will record charges of
approximately $0.9 million during the fourth quarter of 2009 for
employment termination costs payable in cash in connection with the
workforce reduction.
"After extensive review of our business plan, we have made some
very difficult decisions to restructure our workforce to best
enable us to align the Company around the three key strategic
priorities for Amicus going forward," said John F. Crowley.
"Specifically, we expect that these changes will enable our current
cash to last into the second half of 2011. It is exceptionally
difficult to implement these employee changes as so many remarkably
dedicated and talented persons, including Jim Dentzer, our Chief
Financial Officer, will be leaving Amicus. All of them have
contributed significantly to the Company, and I cannot thank them
enough for all of their tireless efforts, passion and
contributions."
"We are highly confident that with this restructuring and
strategic repositioning, we are well poised to maximize shareholder
value while ensuring that we deliver on our mission to provide
innovative and high quality therapies to people afflicted with
these diseases. We remain fully committed to the success of
Amicus," concluded John F. Crowley.
Financial Guidance:
Based on current projections of net operating expense, the
Company reiterates its expectation to end 2009 with approximately
$70-$80 million in cash, cash equivalents and marketable
securities. Additionally, the Company expects that its current
cash, cash equivalents and marketable securities together with the
fourth quarter $5.2 million payment from Shire will be sufficient
to fund operations and capital expenditure requirements into the
second half of 2011.
Third Quarter 2009 Results Summary
On a reported basis calculated in accordance with U.S. Generally
Accepted Accounting Principles (GAAP), Amicus announced a net loss
attributable to common stockholders of $0.59 per share ($0.51 per
share on a non-GAAP basis) for the three months ended September 30,
2009. As of September 30, 2009, cash, cash equivalents and
marketable securities totaled $89.3 million.
Clinical Program Updates Amigal(TM) (migalastat hydrochloride) for the treatment of Fabry disease
In the second quarter of 2009, Amicus announced that the Company
reached agreement with the FDA on the key protocol design elements
of its pivotal trial, including the use of the surrogate primary
endpoint of the change in the amount of kidney interstitial
capillary GL-3, the substrate that accumulates in the cells of
Fabry patients. In addition, the FDA is in agreement that the
Company is eligible to seek Accelerated Approval for Amigal
according to Subpart H regulations. Amicus began submitting the
Phase 3 protocol to investigational sites worldwide in June 2009
and patient enrollment and dosing are now underway.
Furthermore, Amicus previously reported that it completed a
series of discussions with the EMEA regarding the clinical study
required for Amigal registration in Europe. The Company will
provide an update on the timing of the initiation of this study in
2010.
Plicera(TM) (afegostat tartrate) for the treatment of Gaucher
Disease
The Company previously reported preliminary results from its
Phase 2 randomized, open-label study to assess the safety,
tolerability and preliminary efficacy of its investigational drug,
Plicera, in treatment-naive adult patients with type 1 Gaucher
disease. Two dose regimens of Plicera (225 mg three days on/four
days off and seven days on/seven days off) were studied during this
six month trial. While all patients enrolled experienced an
increase in the level of the target enzyme (GCase) as measured in
white blood cells, clinically meaningful improvements in key
measures of disease were observed in just one of the eighteen
patients who completed the study. The preliminary results suggest
that treatment with Plicera was generally well tolerated, with no
serious adverse events (SAEs) reported. Nineteen subjects were
enrolled and 18 subjects completed the study. One subject
discontinued treatment because of an adverse event
(conjunctivitis-related symptoms).
The Company plans to further analyze and evaluate the results of
this Phase 2 study. However, Amicus no longer plans to advance
Plicera into Phase 3 development.
AT2220 (1-deoxynojirimycin HCl) for the treatment of Pompe
Disease
Late in the third quarter, the Company announced its plans to
initiate a Phase 1 study of AT2220 (1-deoxynojirimycin HCl), its
investigational drug in development for the treatment of Pompe
Disease. The primary objective of this study is to evaluate the
pharmacokinetics of AT2220 in muscle tissue in healthy adult
subjects. The FDA agreed to Amicus' proposal for the Phase 1 study
and subsequently converted the clinical hold of AT2220 to a partial
hold to allow the conduct of this study.
This open label, single dose Phase 1 study was initiated in
early October and the Company expects to announce results from the
trial in the first half of 2010.
Additionally, Amicus continues to be encouraged by the results
of preclinical studies designed to evaluate the use of AT2220 in
combination with enzyme replacement therapy (ERT). The Company
expects to report additional data from these studies at scientific
conferences in 2010.
Preclinical Chaperone Programs
Amicus continues to invest in research to assess the potential
for applying its versatile chaperone technology platform to the
treatment of a broad range of human genetic diseases. As part of
this effort, Amicus continues to conduct preclinical studies in
Parkinson's disease and is investing in new research aimed at
evaluating disease targets for other neurodegenerative and genetic
disorders.
Additional Financial Results & Notes
On a reported basis, the net loss attributable to common
stockholders for the three months ended September 30, 2009, was
$13.4 million as compared to $8.2 million for the same period in
2008. On a non-GAAP basis, the net loss for the three months ended
September 30, 2009, was $11.5 million as compared to $6.6 million
in the same period in 2008.
Amicus recorded revenue during the third quarter of 2009
representing two different revenue streams from the Shire
collaboration agreement. Upon signing the collaboration agreement,
Amicus received an upfront payment of $50 million that had been
recognized as revenue on a straight-line basis over 18 years from
the date of the agreement. In the third quarter of 2009, Amicus
recognized $0.7 million of the Shire upfront payment and $4.2
million of research revenue on reimbursed research and development
costs.
The differences between U.S. GAAP and non U.S. GAAP financial
results are itemized in Tables 2 and 3 and are primarily due to
pre-tax stock compensation expense.
Use of Non-GAAP Financial Measures
Amicus' "non-GAAP net loss" and "non-GAAP diluted net loss per
common share" financial measures are defined as reported, or GAAP,
net loss and diluted net loss per common share excluding certain
items further discussed below. Amicus' management uses these
non-GAAP financial measures to establish financial goals and to
gain an understanding of the comparative financial performance of
Amicus from year to year and quarter to quarter. Accordingly,
Amicus believes investors' understanding of Amicus' financial
performance is enhanced as a result of disclosing these non-GAAP
financial measures. Non-GAAP net loss and diluted net loss per
common share should not be viewed in isolation or as a substitute
for reported, or GAAP net loss and diluted net loss per common
share.
(1) Stock option expense -- Non-GAAP net loss and diluted net
loss per common share exclude the impact of the stock option
expense recorded in accordance with SFAS No. 123R. Amicus believes
that excluding the impact of expensing stock options better
reflects the recurring economic characteristics of its
business.
Conference Call and Webcast
Amicus will host a conference call to discuss financial and
operational results for the third quarter 2009, today, October 29,
2009 at 5:30 p.m. EDT. Interested participants and investors may
access the conference call by dialing 877-440-5807 (U.S./Canada) or
719-325-4915 (international). A telephonic replay of the call will
be available for seven days beginning at 8 p.m. EDT. Access numbers
for this replay are 888-203-1112 (U.S./Canada) and 719-457-0820
(international); participant code 6603470.
An audio webcast can also be accessed via the investor section
of the Amicus Therapeutics Web site at www.amicustherapeutics.com
under Investors: Events and Presentations. 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.
After the live webcast, an audio webcast replay will remain
available in the Investors section of the Amicus Therapeutics Web
site for 30 days.
Amicus' press releases are available at www.amicustherapeutics.com. About Amicus Therapeutics
Amicus Therapeutics is a biopharmaceutical company developing
novel, oral therapeutics known as pharmacological chaperones for
the treatment of a range of human genetic diseases. Pharmacological
chaperone technology involves the use of small molecules that
selectively bind to and stabilize proteins in cells, leading to
improved protein folding and trafficking, and increased activity.
Amicus is initially targeting lysosomal storage disorders, which
are severe, chronic genetic diseases with unmet medical
needs.
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 trial 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 ongoing
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 preclinical and clinical studies could be delayed
because we identify serious side effects or other safety issues;
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.
Additionally, 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. Additionally, all forward looking statements are
subject to other risks detailed in our Annual Report on Form 10-K
for the year ended December 31, 2008, and our other public filings
with the Securities and Exchange Commission. 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
(Unaudited)
(In thousands, except share and per share amounts)
Period from
February
Three Months Nine Months 4, 2002
Ended Ended (inception) to
September 30, September 30, September 30,
------------- ------------ -------------
2008 2009 2008 2009 2009
---- ---- ---- ---- ----
Revenue:
Research revenue $2,959 $4,219 $8,539 $12,799 $26,362
Collaboration
revenue 694 694 2,083 2,083 5,270
--- --- ----- ----- -----
Total revenue 3,653 4,913 10,622 14,882 31,632
----- ----- ------ ------ ------
Operating Expenses:
Research and
development 8,200 12,609 23,989 37,954 165,596
General and
administrative 4,371 5,217 14,676 15,635 73,371
Impairment of
leasehold
improvements - - - - 1,030
Depreciation and
amortization 382 561 1,036 1,585 5,872
In-process research
and development - - - - 418
--- --- --- --- ---
Total operating
expenses 12,953 18,387 39,701 55,174 246,287
------ ------ ------ ------ -------
Loss from operations (9,300) (13,474) (29,079) (40,292) (214,655)
Other income
(expenses):
Interest income 1,019 129 4,053 924 13,684
Interest expense (49) (84) (179) (155) (1,802)
Change in fair value
of warrant liability - - - - (454)
Other expense - - - - (1,180)
--- --- --- --- -----
Loss before tax
benefit (8,330) (13,429) (25,205) (39,523) (204,407)
Benefit from income
taxes 150 - - - 695
--- --- --- --- ---
Net loss (8,180) (13,429) (25,205) (39,523) (203,712)
Deemed dividend - - - - (19,424)
Preferred stock
accretion - - - - (802)
--- --- --- --- ---
Net loss attributable
to common
stockholders $(8,180) $(13,429) $(25,205) $(39,523) $(223,938)
======= ======== ======== ======== =========
Net loss attributable
to common stockholders
per common share -
basic and diluted $(0.36) $(0.59) $(1.12) $(1.75)
====== ====== ====== ======
Weighted-average
common shares
outstanding -
basic and
diluted 22,517,431 22,621,513 22,465,981 22,617,808
========== ========== ========== ==========
Table 2
Amicus Therapeutics, Inc.
Reconciliation of GAAP to non-GAAP Measures for the
Statement of Operations Information for Three Months Ended September
30, 2009
(Unaudited)
(In thousands, except share and per share amounts)
Stock GAAP as
Non-GAAP Compensation Reported
-------- -------------- ------------
Income Statement
Classifications:
Revenue $4,913 $4,913
Research and development (11,780) $(829) (12,609)
General and administrative (4,075) (1,142) (5,217)
Depreciation and amortization (561) (561)
Interest income 129 129
Interest expense (84) (84)
--- ------- ---
Summary:
Net loss: $(11,458) $(1,971) $(13,429)
-------- ------- --------
Net loss per share -
basic and diluted: $(0.51) $(0.09) $(0.59)
====== ====== ======
Weighted average
number of shares
outstanding: 22,621,513 22,621,513
========== ==========
Table 3
Amicus Therapeutics, Inc.
Reconciliation of GAAP to non-GAAP Measures for the
Statement of Operations Information for Three Months Ended September
30, 2008
(Unaudited)
(In thousands, except share and per share amounts)
Stock GAAP as
Non-GAAP Compensation Reported
-------- -------------- ------------
Income Statement
Classifications:
Revenue $3,653 $3,653
Research and development (7,597) $(603) (8,200)
General and administrative (3,358) (1,013) (4,371)
Depreciation and amortization (382) (382)
Interest income 1,019 1,019
Interest expense (49) (49)
(Provision for)/benefit from
income taxes 150 150
--- ------- ---
Summary:
Net loss: $(6,564) $(1,616) $(8,180)
------- ------- -------
Net loss per share -
basic and diluted: $(0.29) $(0.07) $(0.36)
====== ====== ======
Weighted average
number of shares
outstanding: 22,517,431 22,517,431
========== ==========
FOLD -G
Source: Amicus Therapeutics
CONTACT: Jenene Thomas, Director, Investor Relations of
Amicus
Therapeutics, +1-609-662-5084
Web Site: http://www.amicustherapeutics.com/
Posted: October 2009
