Delaware | 71-0869350 | |
(State or other jurisdiction of | (IRS Employer | |
incorporation or organization) | Identification No.) |
Title of each class | Name of each exchange on which registered | |
Common Stock, par value $0.01 per share | The NASDAQ Stock Market LLC |
Large accelerated filer o | Accelerated filer o | Non-accelerated filer þ | Smaller Reporting Company o |
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Exhibit 23.1 | ||||||||
Exhibit 31.1 | ||||||||
Exhibit 31.2 | ||||||||
Exhibit 32.1 | ||||||||
Exhibit 32.2 |
| our plans to develop, seek regulatory approval for and commercialize Amigal, Plicera and AT2220; | ||
| our ongoing and planned discovery programs, preclinical studies and clinical trials; | ||
| our ability to enter into selective collaboration arrangements; | ||
| the timing of and our ability to obtain agreement with the regulatory agencies on the design of our Phase 3 program for Amigal; | ||
| the timing of and our ability to obtain and maintain regulatory approvals for our product candidates; | ||
| the rate and degree of market acceptance and clinical utility of our products; | ||
| our ability to quickly and efficiently identify and develop product candidates; | ||
| the extent to which our scientific approach may potentially address a broad range of diseases across multiple therapeutic areas; | ||
| our commercialization, marketing and manufacturing capabilities and strategy; | ||
| our intellectual property position; | ||
| our estimates regarding expenses, future revenues, capital requirements and needs for additional financing; | ||
| our belief about our ability to fund our operating expenses; and | ||
| our eligibility to receive milestone payments under our collaboration agreement with Shire Pharmaceuticals Ireland Ltd. |
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Amigal for Fabry disease.
We are developing Amigal for the treatment of Fabry disease.
As previously disclosed in August 2008, Amicus completed an End of Phase 2 meeting for
Amigal with the U.S. Food and Drug Administration (FDA). Following this End of Phase 2
meeting, we continued discussions with the FDA in the second half of 2008. The FDA
indicated that it supports a Phase 3 clinical trial comparing Amigal to placebo based on a
surrogate primary endpoint of the change in the amount of kidney GL-3, the substrate that
accumulates in the cells of Fabry patients. We plan to continue discussions with the FDA
through a Special Protocol Assessment (SPA) procedure that commenced in the fourth quarter
of 2008 in order to finalize how the primary endpoint will be measured. We expect to
complete the final protocol in the second quarter of 2009. Based on discussions with the
European Medicines Agency (EMEA), we also plan to initiate a separate clinical study
designed to evaluate the safety and efficacy of Amigal versus ERT in Fabry patients. We
and our partner, Shire, plan additional discussions with the EMEA in the first half of 2009
to finalize the design of this study. In parallel with the regulatory process, 23 of the
original 26 patients who participated in the Phase 2 clinical trial of Amigal continue to
be treated with Amigal in the voluntary Phase 2
extension study to characterize the long term safety and efficacy and evaluate additional
doses and dose regimens. Data from this extension study are expected to be available in the
first quarter of 2009 and the results will be used to finalize the dose and regimen for the
Phase 3 program.
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Plicera for Gaucher disease.
We are developing Plicera for the treatment of Gaucher
disease. A Phase 2 clinical trial of Plicera in Gaucher patients naïve to ERT is ongoing.
This 6-month study is designed to evaluate safety as well as to demonstrate trends of
efficacy as measured by the standard endpoints in Gaucher disease. Target enrollment of 16
patients for this study was surpassed and the results are expected to be available in the
third quarter of 2009. In addition, we are working closely with our partner, Shire, to
prepare for Phase 3 development of Plicera pending the results of the ongoing Phase 2
trial.
AT2220 for Pompe disease.
We are developing AT2220 for the treatment of Pompe disease.
A Phase 2 clinical trial of AT2220 (1-deoxynojirimycin HCl) in adult Pompe patients is
ongoing. The trial includes an 11-week treatment period with an optional extension study.
The objectives of the trial include the evaluation of the safety and pharmacodynamics of
multiple doses and regimens of AT2220. The results of this study are expected to be
available in the second half of 2009. In addition, we are continuing to conduct
preclinical animal studies to evaluate the effects of administering AT2220 in combination
with ERT. Encouraging preliminary results were announced at the American Society of Human
Genetics conference in November 2008. These results indicated that AT2220 in combination
with ERT increases the stability and tissue uptake of ERT. We are conducting additional
preclinical proof-of-concept studies to determine the feasibility of a combination that may
be appropriate for Pompe patients who are not amenable to chaperone monotherapy. We expect
to announce additional results in the first quarter of 2009.
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Product Characteristic
Enzyme Replacement Therapy
Pharmacological Chaperone Therapy
Variable tissue distribution
Broad tissue distribution,
including brain
Weekly or every other week
intravenous infusion
Oral administration
Recombinant protein manufacturing
Chemical synthesis
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Stage of
Worldwide
Product Candidate
Indication
Development
Commercial Rights
Fabry
Phase 2 Complete
Amicus U.S.,
Disease
Phase 3 in Planning
Shire ex-U.S.
Gaucher
Amicus U.S.,
Disease
Phase 2 Ongoing
Shire ex-U.S.
Pompe
Amicus U.S.,
Disease
Phase 2 Ongoing
Shire ex-U.S.
Amigal was generally safe and well-tolerated at all doses evaluated and no drug-related
serious adverse events were reported.
Amigal increased the level of the enzyme deficient in Fabry patients in 24 of 26 study
subjects.
Amigal was shown to reduce the accumulated substrate in a majority of study subjects.
Renal and cardiac function results were encouraging, including those seen in patients
treated for nearly two years.
Responses in patients with different Fabry mutations were consistent with the results of
in vitro testing, thus confirming the ability to use pharmacogenetics to select likely
responders for future studies.
Twenty-three of the 26 patients continue to receive Amigal in a voluntary extension
study.
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Type I Chronic Nonneuronopathic Gaucher Disease.
Type I Gaucher disease is the most
common subtype affecting more than 90% of patients and symptoms usually first appear in
adulthood. Type I Gaucher disease is characterized by the occurrence of an enlarged spleen
and liver, anemia, low platelet counts and fractures and bone pain. Patients with Type I
Gaucher disease do not experience the neurological features associated with Types II
and III Gaucher disease. The clinical severity of Type I Gaucher disease is extremely
variable with some patients experiencing the full range of symptoms, while others are
asymptomatic throughout most of their lives.
Type II Acute Neuronopathic Gaucher Disease.
Type II Gaucher disease symptoms
typically appear in infancy with an average age of onset of about three months. Type II
Gaucher disease involves rapid neurodegeneration with extensive visceral involvement that
usually results in death before two years of age, typically due to respiratory
complications. The clinical presentation in Type II Gaucher disease is typically more
uniform than Type I Gaucher disease.
Type III Subacute Neuronopathic Gaucher Disease.
Type III Gaucher disease
symptoms typically first appear in infancy or early childhood and involve some
neurological symptoms, along with visceral and bone complications. Age of onset and
disease severity can vary widely. Disease progression in Type III Gaucher disease is
typically slower than in Type II Gaucher disease.
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We have an exclusive license to five issued U.S. patents and several pending
U.S. applications that cover use of Amigal, as well as corresponding foreign applications.
U.S. patents relating to Amigal expire in 2018 (not including the Hatch-Waxman statutory
extension, which is described below), while the foreign counterpart patents, if granted,
would expire in 2019 (not including the Supplemental Protection Certificates or SPC
extensions, which are described below). The patents and the pending applications include
claims covering methods of increasing the activity of and preventing the degradation of
a
-GAL, and methods for the treatment of Fabry disease using Amigal and other specific
competitive inhibitors of
a
-GAL. In addition, we own pending U.S. applications directed to
specific treatment and monitoring regimens with Amigal as well as to dosing regimens with
Amigal, which, if granted, may result in patents that expire in 2028. Further, we have
several pending U.S. applications directed to synthetic steps related to the commercial
process for preparing Amigal, which may result in patents that expire in 2026. Lastly, we
jointly own one pending U.S. application covering methods of diagnosing Fabry disease and
determining whether Fabry patients will respond to treatment with Amigal, which, if
granted, will expire in 2027. We have filed, or plan to file, foreign counterparts of
these applications, where appropriate, by the applicable deadlines.
We have an exclusive license to seven U.S. patents and two pending U.S. applications,
and five foreign patents and several pending foreign applications that cover Plicera or its
use. Two of the U.S. patents relating to Plicera compositions of matter expire in 2015 and
2016 (not including the Hatch-Waxman statutory extension, which is described below); the
five composition of matter foreign patents and one pending foreign application, if granted,
expire in 2015 (not including the SPC extensions, which are described below). The other
five U.S. patents and two pending applications, which claim methods of increasing the
activity of and preventing the degradation of GCase, and methods for the treatment of
Gaucher disease using Plicera and other specific competitive inhibitors of GCase, expire in
2018. We own a pending U.S. application directed to the particular form of the active
agent in Plicera, which, if granted, will expire in 2027. We own one pending
U.S. application directed to dosing regimens for Plicera, which if granted, will expire in
2028. We own one pending U.S. application directed to specific treatment and monitoring
regimens with Plicera. If granted, this also will expire in 2028. Lastly, we own one
pending U.S. application directed to methods of synthesis of Plicera, which if granted,
will expire in 2028. We have filed, or plan to file, foreign counterparts of these
applications, where appropriate, by the applicable deadlines.
We have an exclusive license to three U.S. patents that cover use of AT2220, two pending
U.S. applications, as well as corresponding foreign applications. The U.S. patents
relating to AT2220 expire in 2018 (not including the Hatch-Waxman statutory extension,
which is described below), while the foreign counterpart patents, if granted, would expire
in 2019 (not including the SPC extensions, which are described below). The patents and the
pending applications include claims covering methods of increasing the activity of and
preventing the degradation of GAA, and methods for the treatment of Pompe disease using
AT2220 and other specific competitive inhibitors of GAA.
the longer of 17 years from the issue date or 20 years from the earliest effective
filing date, if the patent application was filed prior to June 8, 1995; and
20 years from the earliest effective filing date, if the patent application was filed on
or after June 8, 1995.
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Mt. Sinai School of Medicine
We have acquired exclusive worldwide patent rights to
develop and commercialize Amigal, Plicera and AT2220 and other pharmacological chaperones
for the prevention or treatment of human diseases or clinical conditions by increasing the
activity of wild-type and mutant enzymes pursuant to a license agreement with Mt. Sinai
School of Medicine (MSSM) of New York University. In connection with this agreement, we
issued 232,266 shares of our common stock to MSSM in April 2002. In October 2006 we issued
MSSM an additional 133,333 shares of common stock and made a payment of $1.0 million in
consideration of an expanded field of use under that license. Under this agreement, to
date we have paid no upfront or annual license fees and we have no milestone or future
payments other than royalties on net sales. However, on October 31, 2008, we amended and
restated this license agreement to, among other items, provide us with the sole right to
control the prosecution of patent rights under such agreement and to clarify the portion of
royalties and milestone payments we receive from Shire that are payable to MSSM. In
connection therewith, we agreed to pay MSSM $2.6 million in connection with the $50 million
upfront payment that we received in November 2007 from Shire, which was already accrued for
at year-end 2007, and an additional $2.6 million for the sole right to and control over the
prosecution of patent rights. This agreement expires upon expiration of the last of the
licensed patent rights, which will be in 2019 if a foreign patent is granted and 2018
otherwise, or later subject to any patent term extension that may be granted.
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University of Maryland, Baltimore County
We have acquired exclusive U.S. patent rights
to develop and commercialize Plicera for the treatment of Gaucher disease from the
University of Maryland, Baltimore County. Under this agreement, to date we have paid
aggregate upfront and annual license fees of $30 thousand. We are required to make a
milestone payment upon the demonstration of safety and efficacy of Plicera for the
treatment of Gaucher disease in a Phase 2 study, and another payment upon receiving FDA
approval for Plicera for the treatment of Gaucher disease. We are also required to pay
royalties on net sales. Upon satisfaction of both milestones, we could be required to make
up to $0.2 million in aggregate payments. This agreement expires upon expiration of the
last of the licensed patent rights in 2015.
Novo Nordisk A/S
We have acquired exclusive patent rights to develop and commercialize
Plicera for all human indications. Under this agreement, to date we have paid an aggregate
of $0.4 million in license fees. We are also required to make milestone payments based on
clinical progress of Plicera, with a payment due after initiation of a Phase 2 clinical
trial for Plicera for the treatment of Gaucher disease, and a payment due upon each filing
for regulatory approval of Plicera for the treatment of Gaucher disease in any of the U.S.,
Europe or Japan. An additional payment is due upon approval of Plicera for the treatment
of Gaucher disease in the U.S. and a payment is also due upon each approval of Plicera for
the treatment of Gaucher disease in either of Europe or Japan. Assuming successful
development of Plicera for the treatment of Gaucher disease in the U.S., Europe and Japan,
total milestone payments would be $7.8 million. We are also required to pay royalties on
net sales. This license will terminate in 2016.
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Competitor
Indication
Product
Class of Product
Status
2008 Sales
(in millions)
Fabry disease
Fabrazyme
®
Enzyme Replacement
Therapy
Marketed
$
494
Gaucher disease
Cerezyme
®
Enzyme Replacement
Therapy
Marketed
$
1,238
Pompe disease
Myozyme
®
Enzyme Replacement
Therapy
Marketed
$
296
Gaucher disease
Genz-112638
Substrate Reduction
Therapy
Phase 2
N/A
Fabry disease
Replagal
®
Enzyme Replacement
Therapy
Marketed
$
132
*
Gaucher disease
GA-GCB
Enzyme Replacement
Therapy
Phase 3
N/A
Gaucher disease
Zavesca
®
Substrate Reduction
Therapy
Marketed
$
29
*
Gaucher disease
prGCD
Enzyme Replacement
Phase 3
N/A
Therapy
*
Nine Months Sales through September 30, 2008
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continue our ongoing Phase 2 extension study of Amigal (migalastat hydrochloride)
for the treatment of Fabry disease and initiate a Phase 3 clinical trial of Amigal;
continue our ongoing Phase 2 clinical trials of Plicera (isofagomine tartrate) for
the treatment of Gaucher disease and potentially conduct additional Phase 2 and
later-stage clinical trials of Plicera;
continue our ongoing Phase 2 clinical trial of AT2220 for the treatment of Pompe
disease and potentially conduct later-stage clinical trials of AT2220;
continue our preclinical studies on the combination use of pharmacological
chaperones and enzyme replacement therapy in Pompe disease;
continue the research and development of additional product candidates;
seek regulatory approvals for our product candidates that successfully complete
clinical trials;
establish a sales and marketing infrastructure to commercialize products for which
we may obtain regulatory approval; and
add operational, financial and management information systems and personnel,
including personnel to support our product development efforts and our obligations as a
public company.
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the progress and results of our clinical trials of Amigal, Plicera and AT2220;
the scope, progress, results and costs of preclinical development, laboratory
testing and clinical trials for our other product candidates;
our achievement of milestone payments under our collaboration agreement with Shire;
the costs, timing and outcome of regulatory review of our product candidates;
the number and development requirements of other product candidates that we pursue;
the costs of commercialization activities, including product marketing, sales and
distribution;
the emergence of competing technologies and other adverse market developments;
the costs of preparing, filing and prosecuting patent applications and maintaining,
enforcing and defending intellectual property related claims;
the extent to which we acquire or invest in businesses, products and technologies;
and
our ability to establish collaborations and obtain milestone, royalty or other
payments from any such collaborators.
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obtaining supplies of Amigal, Plicera and AT2220 for completion of our clinical
trials on a timely basis;
successful enrollment of patients in our clinical trials on a timely basis;
successful completion of preclinical studies and clinical trials;
obtaining regulatory agreement in the structure and design of our Phase 3 clinical
programs;
obtaining marketing approvals from the United States Food and Drug Administration
(FDA), and similar regulatory authorities outside the U.S.;
establishing commercial-scale manufacturing arrangements with third party
manufacturers whose manufacturing facilities are operated in compliance with current
good manufacturing practice (cGMP) regulations;
launching commercial sales of the product, whether alone or in collaboration with
others;
acceptance of the product by patients, the medical community and third party payors;
competition from other companies and their therapies;
successful protection of our intellectual property rights from competing products in
the U.S. and abroad; and
a continued acceptable safety and efficacy profile of our product candidates
following approval.
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our preclinical tests or clinical trials may produce negative or inconclusive
results, and we may decide, or regulators may require us, to conduct additional
preclinical testing or clinical trials or we may abandon projects that we expect to be
promising;
regulators or institutional review boards may not authorize us to commence a
clinical trial or conduct a clinical trial at a prospective trial site;
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conditions imposed on us by the FDA or any non-U.S. regulatory authority regarding
the scope or design of our clinical trials or may require us to resubmit our clinical
trial protocols to institutional review boards for re-inspection due to changes in the
regulatory environment;
the number of patients required for our clinical trials may be larger than we
anticipate or participants may drop out of our clinical trials at a higher rate than we
anticipate;
our third party contractors or clinical investigators may fail to comply with
regulatory requirements or fail to meet their contractual obligations to us in a timely
manner;
we might have to suspend or terminate one or more of our clinical trials if we, the
regulators or the institutional review boards determine that the participants are being
exposed to unacceptable health risks;
regulators or institutional review boards may require that we hold, suspend or
terminate clinical research for various reasons, including noncompliance with
regulatory requirements;
the cost of our clinical trials may be greater than we anticipate;
the supply or quality of our product candidates or other materials necessary to
conduct our clinical trials may be insufficient or inadequate or we may not be able to
reach agreements on acceptable terms with prospective clinical research organizations;
and
the effects of our product candidates may not be the desired effects or may include
undesirable side effects or the product candidates may have other unexpected
characteristics.
be delayed in obtaining, or may not be able to obtain, marketing approval for one or
more of our product candidates;
obtain approval for indications that are not as broad as intended or entirely
different than those indications for which we sought approval; or
have the product removed from the market after obtaining marketing approval.
the prevalence and severity of any side effects, including any limitations or
warnings contained in a products approved labeling;
the efficacy and potential advantages over alternative treatments;
the pricing of our product candidates;
relative convenience and ease of administration;
the willingness of the target patient population to try new therapies and of
physicians to prescribe these therapies;
the strength of marketing and distribution support and timing of market introduction
of competitive products;
publicity concerning our products or competing products and treatments; and
sufficient third party insurance coverage or reimbursement.
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a covered benefit under its health plan;
safe, effective and medically necessary;
appropriate for the specific patient;
cost-effective; and
neither experimental nor investigational.
our inability to recruit and retain adequate numbers of effective sales and
marketing personnel;
the inability of sales personnel to obtain access to or persuade adequate numbers of
physicians to prescribe our products;
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the lack of complementary products to be offered by our sales personnel, which may
put us at a competitive disadvantage against companies with broader product lines;
unforeseen costs associated with creating our own sales and marketing team or with
entering into a partnering agreement with an independent sales and marketing
organization; and
efforts by our competitors to commercialize products at or about the time when our
product candidates would be coming to market.
we may not be able to control the amount and timing of resources that our
distributors may devote to the commercialization of our product candidates;
our distributors may experience financial difficulties;
business combinations or significant changes in a distributors business strategy
may also adversely affect a distributors willingness or ability to complete its
obligations under any arrangement; and
these arrangements are often terminated or allowed to expire, which could interrupt
the marketing and sales of a product and decrease our revenue.
decreased demand for any product candidates or products that we may develop;
damage to our reputation;
regulatory investigations that could require costly recalls or product
modifications;
withdrawal of clinical trial participants;
costs to defend the related litigation;
substantial monetary awards to trial participants or patients, including awards that
substantially exceed our product liability insurance, which we would then be required
to pay from other sources, if available, and would damage our ability to obtain
liability insurance at reasonable costs, or at all, in the future;
loss of revenue;
the diversion of managements attention from managing our business; and
the inability to commercialize any products that we may develop.
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reliance on the third party for regulatory compliance and quality assurance;
limitations on supply availability resulting from capacity and scheduling
constraints of the third parties;
impact on our reputation in the marketplace if manufacturers of our products, once
commercialized, fail to meet the demands of our customers;
the possible breach of the manufacturing agreement by the third party because of
factors beyond our control; and
the possible termination or non-renewal of the agreement by the third party, based
on its own business priorities, at a time that is costly or inconvenient for us.
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our collaboration agreements are likely to be for fixed terms and subject to
termination by our collaborators in the event of a material breach or lack of
scientific progress by us;
our collaborators are likely to have the first right to maintain or defend our
intellectual property rights and, although we would likely have the right to assume
the maintenance and defense of our intellectual property rights if our collaborators
do not, our ability to do so may be compromised by our collaborators acts or
omissions; and
our collaborators may utilize our intellectual property rights in such a way as
to invite litigation that could jeopardize or invalidate our intellectual property
rights or expose us to potential liability.
we or our licensors were the first to make the inventions covered by each of our
pending patent applications;
we or our licensors were the first to file patent applications for these inventions;
others will not independently develop similar or alternative technologies or
duplicate any of our technologies;
any patents issued to us or our licensors will provide a basis for commercially
viable products, will provide us with any competitive advantages or will not be
challenged by third parties;
we will develop additional proprietary technologies that are patentable;
we will file patent applications for new proprietary technologies promptly or at
all;
our patents will not expire prior to or shortly after commencing commercialization
of a product; or
the patents of others will not have a negative effect on our ability to do business.
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We do not hold composition of matter patents covering Amigal and AT2220, two of our
three lead product candidates. Composition of matter patents can provide protection
for pharmaceutical products to the extent that the specifically covered compositions
are important. For our product candidates for which we do not hold composition of
matter patents, competitors who obtain the requisite regulatory approval can offer
products with the same composition as our products so long as the competitors do not
infringe any method of use patents that we may hold.
For some of our product candidates, the principal patent protection that covers or
those we expect will cover, our product candidate is a method of use patent. This type
of patent only protects the product when used or sold for the specified method.
However, this type of patent does not limit a competitor from making and marketing a
product that is identical to our product that is labeled for an indication that is
outside of the patented method, or for which there is a substantial use in commerce
outside the patented method.
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our failure to demonstrate to the satisfaction of the FDA or comparable regulatory
authorities that a product candidate is safe and effective for a particular indication;
the results of clinical trials may not meet the level of statistical significance
required by the FDA or comparable regulatory authorities for approval;
our inability to demonstrate that a product candidates benefits outweigh its risks;
our inability to demonstrate that the product candidate is at least as effective as
existing therapies;
the FDAs or comparable regulatory authorities disagreement with the manner in
which we interpret the data from preclinical studies or clinical trials;
the FDAs or comparable regulatory authorities failure to approve the manufacturing
processes, quality procedures or manufacturing facilities of third party manufacturers
with which we contract for clinical or commercial supplies; and
a change in the approval policies or regulations of the FDA or comparable regulatory
authorities or a change in the laws governing the approval process.
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regulatory authorities may require the addition of restrictive labeling statements;
regulatory authorities may withdraw their approval of the product; and
we may be required to change the way the product is administered or conduct
additional clinical trials.
restrictions on such products, manufacturers or manufacturing processes;
warning letters;
withdrawal of the products from the market;
refusal to approve pending applications or supplements to approved applications that
we submit;
voluntary or mandatory recall;
fines;
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suspension or withdrawal of regulatory approvals or refusal to approve pending
applications or supplements to approved applications that we submit;
refusal to permit the import or export of our products;
product seizure or detentions;
injunctions or the imposition of civil or criminal penalties; and
adverse publicity.
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establish a classified board of directors, and, as a result, not all directors are
elected at one time;
allow the authorized number of our directors to be changed only by resolution of our
board of directors;
limit the manner in which stockholders can remove directors from our board of
directors;
establish advance notice requirements for stockholder proposals that can be acted on
at stockholder meetings and nominations to our board of directors;
require that stockholder actions must be effected at a duly called stockholder
meeting and prohibit actions by our stockholders by written consent;
limit who may call stockholder meetings;
authorize our board of directors to issue preferred stock, without stockholder
approval, which could be used to institute a poison pill that would work to dilute
the stock ownership of a potential hostile acquirer, effectively preventing
acquisitions that have not been approved by our board of directors; and
require the approval of the holders of at least 67% of the votes that all our
stockholders would be entitled to cast to amend or repeal certain provisions of our
charter or bylaws.
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results of clinical trials of our product candidates or those of our competitors;
our entry into or the loss of a significant collaboration;
regulatory or legal developments in the U.S. and other countries, including changes
in the health care payment systems;
variations in our financial results or those of companies that are perceived to be
similar to us;
changes in the structure of healthcare payment systems;
market conditions in the pharmaceutical and biotechnology sectors and issuance of
new or changed securities analysts reports or recommendations;
general economic, industry and market conditions;
results of clinical trials conducted by others on drugs that would compete with our
product candidates;
developments or disputes concerning patents or other proprietary rights;
public concern over our product candidates or any products approved in the future;
litigation;
future sales or anticipated sales of our common stock by us or our stockholders; and
the other factors described in this Risk Factors section.
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- 44 -
- 45 -
- 46 -
- 47 -
- 48 -
- 49 -
- 50 -
- 51 -
- 52 -
- 53 -
- 54 -
- 55 -
- 56 -
- 57 -
- 58 -
- 59 -
- 60 -
- 61 -
- 62 -
- 63 -
- 64 -
- 65 -
- 66 -
- 67 -
- 68 -
- 69 -
- 70 -
- 71 -
- 72 -
- 73 -
- 74 -
- 75 -
- 76 -
- 77 -
- 78 -
- 79 -
- 80 -
- 81 -
- 82 -
- 83 -
- 84 -
Item 5. MARKET FOR THE REGISTRANTS COMMON EQUITY, RELATED STOCKHOLDER MATTERS AND ISSUER PURCHASES
OF EQUITY SECURITIES.
High
Low
$
11.84
$
9.00
12.35
9.00
18.00
10.52
15.78
7.16
High
Low
$
16.80
$
10.30
16.75
10.00
18.22
9.20
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*
$100 invested on May 31, 2007 in Amicus Therapeutics, Inc. stock or in index-including
reinvestment of dividends.
May-07
Jun-07
Sep-07
Dec-07
Mar-08
Jun-08
Sep-08
Dec-08
100
80
116
74
74
74
105
55
100
100
104
102
88
88
88
61
100
96
102
100
93
95
100
87
Table of Contents
(c) Total number of
(d) Maximum number of
shares purchased as
shares that may yet be
(a) Total number
(b) Average
part of publicly
purchased under
of shares
Price Paid
announced plans or
the plans or
Period
purchased
per Share
programs
programs
220
$
15.10
5,295
220
$
10.45
5,075
220
$
7.98
4,855
660
Table of Contents
Period from
February 4,
2002
(inception) to
Year Ended December 31
December 31,
2004
2005
2006
2007
2008
2008
$
$
$
$
1,375
$
12,189
$
13,564
409
2,778
3,187
1,784
14,967
16,751
6,301
13,652
33,630
31,074
37,764
127,642
2,081
6,877
12,277
15,278
19,666
57,736
1,030
146
303
952
1,237
1,493
4,287
418
8,528
20,832
46,859
47,589
58,923
191,113
(8,528
)
(20,832
)
(46,859
)
(45,805
)
(43,956
)
(174,362
)
190
610
1,990
5,135
4,819
12,760
(550
)
(82
)
(273
)
(348
)
(218
)
(1,648
)
(2
)
(280
)
(23
)
(149
)
(454
)
(1,180
)
(1,180
)
(8,890
)
(20,584
)
(46,345
)
(41,167
)
(39,355
)
(164,884
)
83
612
695
(8,807
)
(19,972
)
(46,345
)
(41,167
)
(39,355
)
(164,189
)
(19,424
)
(19,424
)
(126
)
(139
)
(159
)
(351
)
(802
)
$
(8,933
)
$
(20,111
)
$
(65,928
)
$
(41,518
)
$
(39,355
)
(184,415
)
$
(29.05
)
$
(49.02
)
$
(89.58
)
$
(3.14
)
$
(1.75
)
307,539
410,220
735,967
13,235,755
22,493,803
As of December 31,
2004
2005
2006
2007
2008
$
4,336
$
24,418
$
54,699
$
161,527
$
121,124
3,569
22,267
44,814
147,247
110,209
5,073
28,670
59,645
167,097
128,773
1,346
4,031
13,071
63,800
57,730
20,013
60,469
124,089
(17,351
)
(37,322
)
(83,667
)
(124,834
)
(164,189
)
$
(16,287
)
$
(35,830
)
$
(77,515
)
$
103,297
$
71,043
Table of Contents
Table of Contents
internal costs associated with our research activities;
payments we make to third party contract research organizations, contract manufacturers,
investigative sites, and consultants;
technology license costs;
manufacturing development costs;
personnel related expenses, including salaries, benefits, travel, and related costs for
the personnel involved in drug discovery and development;
activities relating to regulatory filings and the advancement of our product candidates
through preclinical studies and clinical trials; and
facilities and other allocated expenses, which include direct and allocated expenses for
rent, facility maintenance, as well as laboratory and other supplies.
Period from
February 4,
2002
(Inception) to
Years Ended December 31,
December 31,
2006
2007
2008
2008
$
3,361
$
4,648
$
4,410
$
25,440
9,905
4,378
2,796
18,904
4,427
3,426
2,836
11,024
17,693
12,452
10,042
55,368
8,187
9,720
14,535
38,966
7,750
8,902
13,187
33,308
15,937
18,622
27,722
72,274
$
33,630
$
31,074
$
37,764
$
127,642
(1)
Other project costs are leveraged across multiple clinical and pre-clinical projects.
(2)
Other costs include facility, supply, overhead, and licensing costs that support multiple
clinical and preclinical projects.
Table of Contents
the number of clinical sites included in the trials;
the length of time required to enroll suitable patients;
the number of patients that ultimately participate in the trials;
the results of our clinical trials; and
any mandate by the U.S. Food and Drug Administration (FDA) or other regulatory authority
to conduct clinical trials beyond those currently anticipated.
Table of Contents
fees owed to contract research organizations in connection with preclinical and
toxicology studies and clinical trials;
fees owed to investigative sites in connection with clinical trials;
fees owed to contract manufacturers in connection with the production of clinical trial
materials;
fees owed for professional services, and
unpaid salaries, wages and benefits.
Years Ended December 31,
2006
2007
2008
$
1.7
$
1.6
$
2.5
1.6
2.4
3.9
$
3.3
$
4.0
$
6.4
Table of Contents
Years Ended December 31,
2006
2007
2008
74.8
%
78.3
%
78.2
%
4.7
%
4.5
%
3.0
%
6.25
6.25
6.25
$
0.00
$
0.00
$
0.00
Table of Contents
Years Ended December 31,
2006
2007
2008
$
(46,345
)
$
(41,167
)
$
(39,355
)
(19,424
)
(159
)
(351
)
$
(65,928
)
$
(41,518
)
$
(39,355
)
735,967
13,235,755
22,493,803
Table of Contents
Approximate
Amount
(1)
Funding
(2)
Year
No. Shares
(in thousands)
2002
444,443
$
2,500
2004, 2005, 2006, 2007
4,917,853
31,189
2005, 2006
5,820,020
54,999
2006, 2007
4,930,405
60,000
2007
5,000,000
75,000
2007
50,000
21,112,721
$
273,688
(1)
Represents gross proceeds.
(2)
The Series A, B, C and D Redeemable Convertible Preferred Stock was converted to common
stock upon the effectiveness of our IPO.
Table of Contents
the progress and results of our clinical trials of Amigal, Plicera and AT2220;
the scope, progress, results and costs of preclinical development, laboratory
testing and clinical trials for our other product candidates;
our achievement of milestone payments under our collaboration agreement with Shire;
the costs, timing and outcome of regulatory review of our product candidates;
the number and development requirements of other product candidates that we pursue;
the costs of commercialization activities, including product marketing, sales and
distribution;
the emergence of competing technologies and other adverse market developments;
the costs of preparing, filing and prosecuting patent applications and maintaining,
enforcing and defending intellectual property related claims;
the extent to which we acquire or invest in businesses, products and technologies;
and
our ability to establish collaborations and obtain milestone, royalty or other payments
from any such collaborators.
completion of Phase 2 clinical trials;
commencement of Phase 3 clinical trials;
submission of a new drug application (NDA) to the FDA or foreign equivalents; and
receipt of marketing approval from the FDA or foreign equivalents.
Table of Contents
Less than
1-3
3-5
Over 5
Total
1 Year
Years
Years
Years
$
6,162
$
2,145
$
4,017
1,294
957
337
2,940
2,199
741
$
10,396
$
5,301
$
5,095
(1)
This table does not include (a) any milestone payments which may
become payable to third parties under license agreements as the timing
and likelihood of such payments are not known, (b) any royalty
payments to third parties as the amounts of such payments, timing
and/or the likelihood of such payments are not known, (c) amounts, if
any, that may be committed in the future to construct additional
facilities, and (d) contracts that are entered into in the ordinary
course of business which are not material in the aggregate in any
period presented above.
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Table of Contents
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pertain to the maintenance of records that in reasonable detail accurately and fairly
reflect the transactions and dispositions of the assets of Amicus Therapeutics, Inc.;
provide reasonable assurance that transactions are recorded as necessary to permit
preparation of financial statements in accordance with generally accepted accounting
principles, and that receipts and expenditures of Amicus therapeutics, Inc. are being made
only in accordance with authorizations of management and directors of Amicus therapeutics,
Inc.; and
provide reasonable assurance regarding prevention or timely detection of unauthorized
acquisition, use or disposition of the assets of Amicus Therapeutics, Inc. that could have
a material effect on the financial statements.
/s/ James E. Dentzer
Table of Contents
Amicus Therapeutics, Inc.
/s/ Ernst & Young LLP
February 4, 2009
Table of Contents
(a development stage company)
Consolidated Balance Sheets
(in thousands, except share and per share amounts)
December 31,
2007
2008
$
44,188
$
28,073
117,339
93,051
1,513
2,463
163,040
123,587
3,790
4,919
267
267
$
167,097
$
128,773
$
10,465
$
8,796
1,527
877
3,801
3,705
15,793
13,378
46,813
44,035
1,194
317
285
287
227,438
234,412
408
533
(124,834
)
(164,189
)
103,297
71,043
$
167,097
$
128,773
Table of Contents
(a development stage company)
Consolidated Statements of Operations
(in thousands, except share and per share amounts)
Period from
February 4,
2002
(Inception) to
Years Ended December 31,
December 31,
2006
2007
2008
2008
$
$
1,375
$
12,189
$
13,564
409
2,778
3,187
1,784
14,967
16,751
33,630
31,074
37,764
127,642
12,277
15,278
19,666
57,736
1,030
952
1,237
1,493
4,287
418
46,859
47,589
58,923
191,113
(46,859
)
(45,805
)
(43,956
)
(174,362
)
1,990
5,135
4,819
12,760
(273
)
(348
)
(218
)
(1,648
)
(23
)
(149
)
(454
)
(1,180
)
(1,180
)
(46,345
)
(41,167
)
(39,355
)
(164,884
)
695
(46,345
)
(41,167
)
(39,355
)
(164,189
)
(19,424
)
(19,424
)
(159
)
(351
)
(802
)
$
(65,928
)
$
(41,518
)
$
(39,355
)
$
(184,415
)
$
(89.58
)
$
(3.14
)
$
(1.75
)
735,967
13,235,755
22,493,803
Table of Contents
(a development stage company)
Consolidated Statements of Changes in Stockholders (Deficiency)/Equity
Period from February 4, 2002 (inception) to December 31, 2002,
and the six year period ended December 31, 2008
(in thousands, except share amounts)
Deficit
Accumulated
Additional
Other
During the
Total
Common Stock
Paid-In
Comprehensive
Deferred
Development
Stockholders
Shares
Amount
Capital
Gain/ (Loss)
Compensation
Stage
(Deficiency) Equity
$
$
$
$
$
$
74,938
6
78
84
232,266
17
401
418
209
(209
)
27
27
8
8
(11
)
(11
)
(1,775
)
(1,775
)
307,204
23
685
(182
)
(1,775
)
(1,249
)
333
14
(14
)
70
70
210
210
4
4
(17
)
(17
)
41
41
(6,768
)
(6,768
)
307,537
23
937
(126
)
(8,543
)
(7,709
)
68
(68
)
60
60
16
16
(126
)
(126
)
193
193
95
95
(9
)
(9
)
(8,807
)
(8,807
)
(8,816
)
307,537
23
1,183
(9
)
(134
)
(17,350
)
(16,287
)
97,156
7
17
24
133,332
10
65
75
2,778
(2,778
)
365
365
112
112
(139
)
(139
)
(7
)
(7
)
(19,972
)
(19,972
)
(19,979
)
538,025
40
4,016
(16
)
(2,547
)
(37,322
)
(35,829
)
Table of Contents
(a development stage company)
Consolidated Statements of Changes in Stockholders (Deficiency) Equity
Period from February 4, 2002 (inception) to December 31, 2002,
and the six year period ended December 31, 2008
(in thousands, except share amounts)
Deficit
Accumulated
Additional
Other
During the
Total
Common Stock
Paid-In
Comprehensive
Deferred
Development
Stockholders
Shares
Amount
Capital
Gain/ (Loss)
Compensation
Stage
(Deficiency) Equity
538,025
40
4,016
(16
)
(2,547
)
(37,322
)
(35,829
)
265,801
20
138
158
133,333
10
1,210
1,220
(2,547
)
2,547
53,333
2,816
2,816
476
476
(159
)
(159
)
117
117
19,424
19,424
(19,424
)
(19,424
)
31
31
(46,345
)
(46,345
)
(46,314
)
990,492
70
6,067
15
(83,667
)
(77,515
)
5,000,000
50
68,095
68,145
16,112,721
162
148,429
148,591
305,518
3
455
458
3,823
3,823
162
162
(351
)
(351
)
758
758
393
393
(41,167
)
(41,167
)
(40,774
)
22,408,731
285
227,438
408
(124,834
)
103,297
225,980
2
528
530
6,446
6,446
125
125
(39,355
)
(39,355
)
(39,230
)
22,634,711
$
287
$
234,412
$
533
$
$
(164,189
)
$
71,043
Table of Contents
(a development stage company)
Consolidated Statements of Cash Flows
(in thousands)
Period from
February 4,
2002
(Inception) to
Years Ended December 31,
December 31,
2006
2007
2008
2008
$
(46,345
)
$
(41,167
)
$
(39,355
)
$
(164,189
)
525
952
1,237
1,493
4,285
522
2,816
3,823
6,446
13,085
475
162
853
22
149
454
44
44
1,220
1,220
1,030
418
135
120
(1,192
)
(949
)
(2,463
)
265
(288
)
6,586
1,566
(1,669
)
8,796
50,614
(2,873
)
47,741
(33,889
)
15,192
(36,863
)
(87,832
)
37,441
126,370
178,100
347,166
(62,013
)
(200,743
)
(153,687
)
(439,801
)
(2,031
)
(669
)
(2,667
)
(10,278
)
(26,603
)
(75,042
)
21,746
(102,913
)
63,371
24,053
143,022
68,093
68,093
5,000
(881
)
(1,388
)
(1,528
)
(4,393
)
158
510
530
1,221
91
97
264
3,431
546
5,611
66,170
91,911
(998
)
218,818
5,678
32,061
(16,115
)
28,073
6,449
12,127
44,188
$
12,127
$
44,188
$
28,073
$
28,073
$
273
$
348
$
218
$
1,354
$
$
148,591
$
148,951
$
$
$
$
5,000
$
159
$
351
$
$
802
$
19,424
$
$
$
19,424
Table of Contents
(a development stage company)
Notes To Consolidated Financial Statements
Table of Contents
(a development stage company)
Notes To Consolidated Financial Statements (Continued)
Table of Contents
(a development stage company)
Notes To Consolidated Financial Statements (Continued)
Table of Contents
(a development stage company)
Notes To Consolidated Financial Statements (Continued)
Table of Contents
(a development stage company)
Notes To Consolidated Financial Statements (Continued)
Years Ended December 31,
2006
2007
2008
$
1.7
$
1.6
$
2.5
1.6
2.4
3.9
$
3.3
$
4.0
$
6.4
Table of Contents
(a development stage company)
Notes To Consolidated Financial Statements (Continued)
Years Ended December 31,
2006
2007
2008
$
(46,345
)
$
(41,167
)
$
(39,355
)
(19,424
)
(159
)
(351
)
$
(65,928
)
$
(41,518
)
$
(39,355
)
735,967
13,235,755
22,493,803
Table of Contents
(a development stage company)
Notes To Consolidated Financial Statements (Continued)
Table of Contents
(a development stage company)
Notes To Consolidated Financial Statements (Continued)
Table of Contents
(a development stage company)
Notes To Consolidated Financial Statements (Continued)
Fair Value Measurements at
Reporting Date using
Quoted Prices
In Active
Significant
Markets for
Other
Significant
Identical
Observable
Unobservable
December 31,
Assets
Inputs
Inputs
2008
(Level 1)
(Level 2)
(Level 3)
$
22,447
$
$
22,447
$
7,217
7,217
58,790
58,790
4,597
4,597
24,626
24,626
$
117,677
$
24,626
$
93,051
$
December 31,
2007
2008
$
694
$
1,322
167
996
3,089
4,096
579
657
2,054
2,108
6,583
9,179
(2,793
)
(4,260
)
$
3,790
$
4,919
Table of Contents
(a development stage company)
Notes To Consolidated Financial Statements (Continued)
December 31,
2007
2008
$
530
$
1,987
979
283
6,035
2,985
2,279
2,846
364
281
278
414
$
10,465
$
8,796
Table of Contents
(a development stage company)
Notes To Consolidated Financial Statements (Continued)
Series A
Series B
Series C
Series D
Shares
Amount
Shares
Amount
Shares
Amount
Shares
Amount
(in thousands)
(in thousands)
(in thousands)
(in thousands)
$
$
$
$
444,443
2,500
(95
)
10
444,443
2,415
17
444,443
2,432
2,823,523
18,000
(122
)
(422
)
17
109
444,443
2,449
2,823,523
17,565
2,039,211
13,000
(6
)
2,910,010
27,500
(178
)
17
110
12
444,443
2,466
4,862,734
30,669
2,910,010
27,334
Table of Contents
(a development stage company)
Notes To Consolidated Financial Statements (Continued)
Series A
Series B
Series C
Series D
Shares
Amount
Shares
Amount
Shares
Amount
Shares
Amount
(in thousands)
(in thousands)
(in thousands)
(in thousands)
444,443
2,466
4,862,734
30,669
2,910,010
27,334
14,322
91
2,910,010
27,500
2,953,878
35,947
(76
)
10
108
35
5
444,443
2,476
4,877,056
30,868
5,820,020
54,869
2,953,878
35,876
1,976,527
24,053
40,797
98
24
126
130
71
(444,443
)
(2,500
)
(4,917,853
)
(31,092
)
(5,820,020
)
(54,999
)
(4,930,405
)
(60,000
)
$
$
$
$
Table of Contents
(a development stage company)
Notes To Consolidated Financial Statements (Continued)
Years Ended December 31,
2006
2007
2008
$
1.7
$
1.6
$
2.5
1.6
2.4
3.9
$
3.3
$
4.0
$
6.4
Table of Contents
(a development stage company)
Notes To Consolidated Financial Statements (Continued)
Years Ended December 31,
2006
2007
2008
74.8
%
78.3
%
78.2
%
4.7
%
4.5
%
3.0
%
6.25
6.25
6.25
$
0.00
$
0.00
$
0.00
Weighted
Weighted
Average
Average
Remaining
Number of
Exercise
Contractual
Aggregate
Shares
Price
Life
Intrinsic Value
(in thousands)
(in millions)
1,237.1
$
2.10
1,005.1
$
6.00
(265.8
)
$
0.60
(108.0
)
$
2.20
1,868.4
$
4.27
1,035.6
$
13.16
(308.6
)
$
1.80
(152.2
)
$
8.94
2,443.2
$
8.08
965.2
$
10.49
(225.1
)
$
2.48
(106.0
)
$
9.69
3,077.3
$
9.19
7.9 years
$
3.5
2,914.8
$
9.09
7.9 years
$
3.5
1,266.3
$
7.36
7.0 years
$
2.8
Table of Contents
(a development stage company)
Notes To Consolidated Financial Statements (Continued)
Restricted Stock
Weighted
Number of
Average Grant
Shares
Date Fair Value
(in thousands)
51.1
$
8.94
$
(16.1
)
$
8.88
$
35.0
$
8.96
$
(14.4
)
$
8.85
$
20.6
$
9.04
Table of Contents
(a development stage company)
Notes To Consolidated Financial Statements (Continued)
$
2,145
1,922
1,839
256
$
6,162
$
957
295
42
1,294
(100
)
1,194
(877
)
$
317
Table of Contents
(a development stage company)
Notes To Consolidated Financial Statements (Continued)
For Years Ended December 31,
2006
2007
2008
$
246
$
283
$
1,560
1,309
1,232
141
1,555
1,515
1,701
1,289
1,129
2,682
3,611
5,403
7,294
27,257
42,282
36,196
19,096
121
478
518
33,833
50,807
67,487
33,833
50,807
67,487
(33,833
)
(50,807
)
(67,487
)
$
$
$
Table of Contents
(a development stage company)
Notes To Consolidated Financial Statements (Continued)
Years Ended December 31,
2006
2007
2008
(34
)%
(34
)%
(34
)%
(6
)
(5
)
(5
)
1
3
2
(4
)
(4
)
(5
)
2
(1
)
41
41
42
0
%
0
%
0
%
Table of Contents
(a development stage company)
Notes To Consolidated Financial Statements (Continued)
Table of Contents
(a development stage company)
Notes To Consolidated Financial Statements (Continued)
Quarters Ended
March 31
June 30
September 30
December 31
$
(9,695
)
$
(9,396
)
$
(10,303
)
$
(11,773
)
(9,736
)
(9,706
)
(10,303
)
(11,773
)
(10.21
)
(1.37
)
(0.46
)
(0.53
)
(7,731
)
(9,294
)
(8,180
)
(14,150
)
(7,731
)
(9,294
)
(8,180
)
(14,150
)
(0.34
)
(0.41
)
(0.36
)
(0.63
)
(1)
Per common share amounts for the quarters and full years have been
calculated separately. Accordingly, quarterly amounts do not add to
the annual amounts because of differences on the weighted-average
common shares outstanding during each period principally due to the
effect of the Companys issuing shares of its common stock during the
year.
Table of Contents
Table of Contents
- 85 -
- 86 -
- 87 -
Table of Contents
Table of Contents
Item 12. SECURITY OWNERSHIP OF CERTAIN BENEFICIAL OWNERS AND MANAGEMENT AND RELATED STOCKHOLDER
MATTERS.
Table of Contents
- 88 -
- 89 -
(a)
1.
Consolidated Financial Statements
Exhibit
Incorporated by Reference to SEC Filing
Filed with this
No.
Filed Exhibit Description
Form
Date
Exhibit No.
Form 10-K
3.1
S-1 (333-141700)
5/17/07
3.2
3.2
S-1/A (333-141700)
4/27/07
3.4
4.1
S-1 (333-141700)
3/30/07
4.1
4.2
S-1 (333-141700)
3/30/07
4.3
10.1
S-1/A (333-141700)
4/27/07
10.1
10.2
S-1/A (333-141700)
5/17/07
10.2
++10.3
X
+10.4
S-1 (333-141700)
3/30/07
10.4
+10.5
S-1 (333-141700)
3/30/07
10.5
10.6
S-1 (333-141700)
3/30/07
10.6
10.7
Form 8-K
Current Report
12/31/08
10.1
10.8
S-1 (333-141700)
3/30/07
10.8
10.9
S-1 (333-141700)
3/30/07
10.9
10.10
S-1 (333-141700)
3/30/07
10.10
10.11
S-1 (333-141700)
3/30/07
10.15
10.12
S-1 (333-141700)
3/30/07
10.17
10.13
S-1/A (333-141700)
4/27/07
10.19
10.14
S-1/A (333-141700)
4/27/07
10.20
10.15
S-1/A (333-141700)
4/27/07
10.21
10.16
S-1/A (333-141700)
4/27/07
10.22
Table of Contents
Exhibit
Incorporated by Reference to SEC Filing
Filed with this
No.
Filed Exhibit Description
Form
Date
Exhibit No.
Form 10-K
10.17
S-1/A (333-141700)
5/17/07
10.23
10.18
S-1/A (333-141700)
5/17/07
10.24
10.19
Form 8-K
Current Report
6/12/08
10.1
10.20
Form 8-K
Current Report
9/15/08
10.1
+10.21
Form 10-K
Annual Report
2/08/08
10.20
10.22
Form 8-K
Current Report
12/31/08
10.4
10.23
Form 8-K
Current Report
12/31/08
10.2
10.24
Form 8-K
Current Report
12/31/08
10.3
10.25
X
10.26
X
10.27
Form 8-K
Current Report
12/31/08
10.5
10.28
X
10.29
X
10.30
X
10.31
X
23.1
X
31.1
X
31.2
X
32.1
X
32.2
X
+
Confidential treated has been granted as to certain portions of the
document, which portions have been omitted and filed separately with
the Securities and Exchange Commission.
++
Confidential treated has been requested as to certain portions of the
document, which portions have been omitted and filed separately with
the Securities and Exchange Commission.
Table of Contents
- 90 -
- 91 -
- 92 -
AMICUS THERAPEUTICS, INC.
By:
/s/ John F. Crowley
John F. Crowley
Chief Executive Officer
Signature
Title
Date
/s/ John F. Crowley
(Principal Executive Officer)
February 4, 2009
/s/ James E. Dentzer
(Principal Financial and Accounting Officer)
February 4, 2009
/s/ Donald J. Hayden
February 4, 2009
/s/ Alexander E. Barkas, Ph.D.
February 4, 2009
/s/ Stephen Bloch, M.D.
February 4, 2009
/s/ P. Sherrill Neff
February 4, 2009
/s/ Michael G. Raab
February 4, 2009
/s/ Glenn Sblendorio
February 4, 2009
/s/ James N. Topper, M.D., Ph.D.
February 4, 2009
/s/ Sol J. Barer, Ph.D.
February 4, 2009
Table of Contents
Exhibit
Incorporated by Reference to SEC Filing
Filed with this
No.
Filed Exhibit Description
Form
Date
Exhibit No.
Form 10-K
3.1
S-1 (333-141700)
5/17/07
3.2
3.2
S-1/A (333-141700)
4/27/07
3.4
4.1
S-1 (333-141700)
3/30/07
4.1
4.2
S-1 (333-141700)
3/30/07
4.3
10.1
S-1/A (333-141700)
4/27/07
10.1
10.2
S-1/A (333-141700)
5/17/07
10.2
++10.3
X
+10.4
S-1 (333-141700)
3/30/07
10.4
+10.5
S-1 (333-141700)
3/30/07
10.5
10.6
S-1 (333-141700)
3/30/07
10.6
10.7
Form 8-K
Current Report
12/31/08
10.1
10.8
S-1 (333-141700)
3/30/07
10.8
10.9
S-1 (333-141700)
3/30/07
10.9
10.10
S-1 (333-141700)
3/30/07
10.10
10.11
S-1 (333-141700)
3/30/07
10.15
10.12
S-1 (333-141700)
3/30/07
10.17
10.13
S-1/A (333-141700)
4/27/07
10.19
10.14
S-1/A (333-141700)
4/27/07
10.20
10.15
S-1/A (333-141700)
4/27/07
10.21
10.16
S-1/A (333-141700)
4/27/07
10.22
10.17
S-1/A (333-141700)
5/17/07
10.23
10.18
S-1/A (333-141700)
5/17/07
10.24
10.19
Form 8-K
Current Report
6/12/08
10.1
10.20
Form 8-K
Current Report
9/15/08
10.1
+10.21
Form 10-K
Annual Report
2/08/08
10.20
10.22
Form 8-K
Current Report
12/31/08
10.4
10.23
Form 8-K
Current Report
12/31/08
10.2
Table of Contents
Exhibit
Incorporated by Reference to SEC Filing
Filed with this
No.
Filed Exhibit Description
Form
Date
Exhibit No.
Form 10-K
10.24
Form 8-K
Current Report
12/31/08
10.3
10.25
X
10.26
X
10.27
Form 8-K
Current Report
12/31/08
10.5
10.28
X
10.29
X
10.30
X
10.31
X
23.1
X
31.1
X
31.2
X
32.1
X
32.2
X
+
Confidential treated has been granted as to certain portions of the
document, which portions have been omitted and filed separately with
the Securities and Exchange Commission.
++
Confidential treated has been requested as to certain portions of the
document, which portions have been omitted and filed separately with
the Securities and Exchange Commission.
[***] |
INDICATES MATERIAL THAT HAS BEEN OMITTED AND FOR WHICH CONFIDENTIAL TREATMENT HAS BEEN REQUESTED.
ALL SUCH OMITTED MATERIAL HAS BEEN FILED WITH THE SECURITIES AND EXCHANGE COMMISSION PURSUANT TO RULE 24B-2
PROMULGATED UNDER THE SECURITIES EXCHANGE ACT OF 1934, AS AMENDED
|
[***] |
INDICATES MATERIAL THAT HAS BEEN OMITTED AND FOR WHICH CONFIDENTIAL TREATMENT HAS BEEN REQUESTED.
ALL SUCH OMITTED MATERIAL HAS BEEN FILED WITH THE SECURITIES AND EXCHANGE COMMISSION PURSUANT TO RULE 24B-2
PROMULGATED UNDER THE SECURITIES EXCHANGE ACT OF 1934, AS AMENDED
|
2
1. |
Definitions.
|
a. |
Affiliate shall mean any corporation, firm, limited liability company,
partnership or other entity that directly or indirectly controls or is controlled by or
is under common control with a party to this Agreement. Control means ownership,
directly or through one or more Affiliates, of 50 percent or more of the shares of
stock entitled to vote for the election of directors, in the case of a corporation, or
50 percent or more of the equity interests in the case of any other type of legal
entity, status as a general partner in any partnership, or any other arrangement
whereby a party controls or has the right to control the board of directors or
equivalent governing body of a corporation or other entity.
|
b. |
Calendar Year shall mean any consecutive period of twelve months commencing
on the first day of January of any year.
|
c. |
[Intentionally Left Blank]
|
d. |
Conformational Diseases shall mean any inherited or acquired human disease in
which affected individuals have at least one mutant allele that results in impaired
protein folding, stability, degradation, or sorting of the encoded mutant protein.
|
e. |
Field shall mean the discovery, validation, development, application,
production or sale of Licensed Products for the prevention, diagnosis and treatment of
all human indications, diseases and conditions.
|
f. |
License shall mean the license under the Patent Rights to develop,
manufacture, have manufactured, use, offer for sale and sell the Licensed Products as
provided in Article 2, below.
|
g. |
Licensed Product shall mean any product or part thereof, the manufacture,
use, or sale of which is: (i) covered by one or more Valid Claims of any Patent Rights,
or (ii) which could not be developed, manufactured, used, sold, comprised or delivered
without the Patent Rights.
|
[***] |
INDICATES MATERIAL THAT HAS BEEN OMITTED AND FOR WHICH CONFIDENTIAL TREATMENT HAS BEEN REQUESTED.
ALL SUCH OMITTED MATERIAL HAS BEEN FILED WITH THE SECURITIES AND EXCHANGE COMMISSION PURSUANT TO RULE 24B-2
PROMULGATED UNDER THE SECURITIES EXCHANGE ACT OF 1934, AS AMENDED
|
3
h. |
Net Sales shall mean the total amount invoiced by AMICUS or by any AMICUS
Affiliate or sub-licensee of AMICUS in connection with sales to any purchaser of the
Licensed Products that is not an Affiliate or a sub-licensee of AMICUS or an AMICUS
Affiliate, after deduction of all the following to the extent applicable to such sales;
|
i) |
trade, cash and quantity credits, discounts, refunds or
rebates;
|
||
ii) |
allowances or credits for returns;
|
||
iii) |
sales commissions;
|
||
iv) |
sales taxes (including value added tax), and
|
||
v) |
freight and insurance charges borne by the seller.
|
i. |
Patent Rights shall mean any issued patent or or any patent application owned
by MSSM, listed in this subclause
1(i)-(xiii),
together with any continuations in whole
or in part, divisional, or substitute patents, any reissues or re-examinations of any
such application or patents, any foreign counterparts of any such application or
patents, and any extension of the term of any such patent in the Field. The issued
patents and patent applications referred to in the preceding sentence are:
|
i) |
U.S. Pat. No. 6,274,597
Method of Enhancing Lysosomal
Alpha-Galactosidase A
|
ii) |
U.S. Pat. No. 6,583,158 (continuation-in-part)- Method of
Enhancing Mutant Enzyme Activities in Lysosomal Storage Disorders
|
iii) |
U.S. Pat. No. 6,744,135 (continuation of the 597 patent)-
Method of Enhancing Lysosomal
Alpha-Galactosidase A
|
iv) |
U.S. Pat. No. 6,599,919 (continuation of the 053 CIP
application)- Method of Enhancing Mutant Enzyme Activities in Lysosomal Storage
Diseases
|
v) |
U.S. Pat. No. 6,589,964 (continuation of the 919 patent)-
Method of Enhancing Mutant Enzyme Activities in Lysosomal Storage Diseases
|
vi) |
U.S. Pat. No. 6,916,829 (continuation of the 919 patent)-
Method of Enhancing Mutant Enzyme Activity in Gaucher Disease
|
vii) |
U.S. Pat. No. 10/868,133 (continuation of 135 patent)- Method
of Enhancing Lysosomal AlphaGalA
|
viii) |
Allowed U.S. Pat. Applic. No. 10/989,258 (continuation of the
829 patent)- Method of Enhancing Mutant Enzyme Activities in Gaucher Disease
|
ix) |
U.S. Pat. Applic. No. 11/264,672 (continuation of the 258
application)-Method for Enhancing Mutant Protein Activity
|
[***] |
INDICATES MATERIAL THAT HAS BEEN OMITTED AND FOR WHICH CONFIDENTIAL TREATMENT HAS BEEN REQUESTED.
ALL SUCH OMITTED MATERIAL HAS BEEN FILED WITH THE SECURITIES AND EXCHANGE COMMISSION PURSUANT TO RULE 24B-2
PROMULGATED UNDER THE SECURITIES EXCHANGE ACT OF 1934, AS AMENDED
|
4
x) |
U.S. Pat. Applic. No. 10/377,179- Screen for Active
Site-Specific Chaperones for Enhancing Protein Folding of Mutant Proteins
|
xi) |
U.S. Pat. Applic. No. 10/781,356- Combination Therapy for
Treating Protein Deficiencies
|
xii) |
U.S. Pat. Applic. No. 11/317,404- Stable Formulations of
Purified Protein
|
j. |
Valid Claim shall mean a claim of (i) an issued patent included in the Patent
Rights which has not been declared invalid in a final, unappealable decision of a court
of appropriate jurisdiction, or (ii) a pending patent application included in the
Patent Rights which is being diligently prosecuted by AMICUS and has not been formally
terminated or abandoned without issuance of a patent.
|
2. |
The License.
|
a. |
Subject to the terms and conditions hereinafter set forth, MSSM hereby grants
to AMICUS and AMICUS hereby accepts from MSSM the world-wide right under the Patent
Rights to develop Licensed Products for use in the Field and to manufacture, use, sell
and offer for sale the Licensed Products for use in the Field. Except as set forth in
Section 2.e and 8f the License shall be exclusive as to all rights of MSSM in and to
the Patent Rights. During the term of this Agreement, MSSM shall make no further grant
of rights in and to the Patent Rights inconsistent with the rights of AMICUS herein.
|
b. |
AMICUS shall be entitled to grant sub-licenses under the License on terms and
conditions not inconsistent with this Agreement (except that the rate of royalty may be
at higher rates than those set forth in this Agreement): (i) to an Affiliate, and (ii)
to other third parties for consideration and in arms-length transactions.
|
c. |
All sub-licenses shall only be granted by AMICUS pursuant to a written
agreement, a true and complete copy of which shall be submitted by AMICUS to MSSM as
soon as practicable after the signing thereof. Each sub-license granted by AMICUS
hereunder shall be subject and subordinate to the terms and conditions of this License
Agreement and shall contain, inter alia, the following provisions:
|
i) |
the sub-license shall expire automatically on the termination
of the License;
|
ii) |
the sub-license shall not be assignable, in whole or in part;
provided, however, that the sublicensee may, with written notice to MSSM,
assign the sub-license in connection with a merger or acquisition of the
sub-licensee or the sale by the sublicensee of substantially all of its assets;
|
[***] |
INDICATES MATERIAL THAT HAS BEEN OMITTED AND FOR WHICH CONFIDENTIAL TREATMENT HAS BEEN REQUESTED.
ALL SUCH OMITTED MATERIAL HAS BEEN FILED WITH THE SECURITIES AND EXCHANGE COMMISSION PURSUANT TO RULE 24B-2
PROMULGATED UNDER THE SECURITIES EXCHANGE ACT OF 1934, AS AMENDED
|
5
iii) |
the sub-licensee shall be entitled to grant further
sub-licensees, provided that the sub-licensee complies with the obligations of
AMICUS under this
Section 2c, Section 2d and all other provisions of this Agreement relating
to sub-licenses by AMICUS; and
|
iv) |
both during the term of the sub-license and thereafter the
sub-licensee shall be bound by a secrecy obligation similar to that imposed on
AMICUS in Section 6 below, and that the sub-licensee shall bind its employees
and agents, both during the terms of their employment and thereafter, with a
similar undertaking of secrecy.
|
d. |
The sub-license agreement shall also include the text of Sections 6, 9 and 10
of this Agreement and shall state that MSSM is an intended third party beneficiary of
such sub-license agreement for purposes of enforcing such indemnification and insurance
provisions.
|
e. |
The License shall be subject to (i) a non-exclusive license in favor of the
U.S. Government to the extent required by Title 35 U.S.C.A. § 200 et seq., or as
otherwise required by virtue of use of federal funding in support of inventions claimed
within the Patent Rights and (ii) a right and license retained by MSSM on behalf of
itself and its faculty, students and academic collaborators to practice the Patent
Rights for its own bona fide research, including sponsored research and collaborations.
The retained rights granted in this Section 2e shall not give MSSM the right to offer
or grant rights in the Field under the Patent Rights to third parties.
|
f. |
Except for the License expressly provided in this Section 2, neither party
hereto will, as a result of this Agreement, obtain any ownership interest in, or any
other right or license to, any existing technology, patents, or Confidential
Information, as defined in Section 6, below, of the other party.
|
3. |
Royalty.
|
a. |
In consideration of the grant of the License hereunder, subject to the
provisions of the Section 3b and Section 3f, AMICUS shall pay to MSSM a royalty of (a)
[***] percent [***] on Net Sales of Core Licensed Products and (b) [***] percent [***]
on Net Sales of Non-Core Licensed Products. If AMICUS grants sublicenses with respect
to a Licensed Product pursuant to which AMICUS receives any Non-Royalty Remuneration,
then AMICUS shall pay to MSSM:
|
i) |
[***] of Non-Royalty Remuneration in connection with such Core
Licensed Product;
|
ii) |
[***] of Non-Royalty Remuneration in connection with a Non-Core
Licensed Product which related to any of the conditions, indications or disease
listed on Schedule I attached to this Agreement;
|
[***] |
INDICATES MATERIAL THAT HAS BEEN OMITTED AND FOR WHICH CONFIDENTIAL TREATMENT HAS BEEN REQUESTED.
ALL SUCH OMITTED MATERIAL HAS BEEN FILED WITH THE SECURITIES AND EXCHANGE COMMISSION PURSUANT TO RULE 24B-2
PROMULGATED UNDER THE SECURITIES EXCHANGE ACT OF 1934, AS AMENDED
|
6
iii) |
[***] of Non-Royalty Remuneration in connection with a Non-Core
Licensed Product (other than (ii) above) where the pertinent sublicense occurs
after three (3) years from October 25, 2006; and
|
iv) |
[***] of Non-Royalty Remuneration in connection with a Non-Core
Licensed Product (other than (ii) above) where the pertinent sublicense occurs
within three (3) years from October 25, 2006.
|
[***] |
INDICATES MATERIAL THAT HAS BEEN OMITTED AND FOR WHICH CONFIDENTIAL TREATMENT HAS BEEN REQUESTED.
ALL SUCH OMITTED MATERIAL HAS BEEN FILED WITH THE SECURITIES AND EXCHANGE COMMISSION PURSUANT TO RULE 24B-2
PROMULGATED UNDER THE SECURITIES EXCHANGE ACT OF 1934, AS AMENDED
|
7
(i) |
payment or reimbursement for patent expenses incurred by
AMICUS;
|
(ii) |
payment or reimbursement for the costs of research or
development conducted by AMICUS that is sponsored by third parties; or
|
(iii) |
purchases by third parties of AMICUS securities;
|
b. |
If AMICUS is required to acquire one or more licenses from third parties to
make, use or sell a Licensed Product such that aggregate royalties payable by AMICUS on
Net Sales (including the royalty due to MSSM pursuant to Section 3.a.) exceeds [***]
percent [***], then AMICUS shall be entitled to a credit against the royalty payments
due to MSSM pursuant to Section 3.a equal to [***] percent [***] of the amount of such
excess; provided, however, that in no event shall the amount otherwise payable to MSSM
be reduced to less than [***] percent [***] of Net Sales.
|
c. |
AMICUS shall notify MSSM of the date of the first commercial sale of a Licensed
Product as soon as practicable after the making of such commercial sale.
|
[***] |
INDICATES MATERIAL THAT HAS BEEN OMITTED AND FOR WHICH CONFIDENTIAL TREATMENT HAS BEEN REQUESTED.
ALL SUCH OMITTED MATERIAL HAS BEEN FILED WITH THE SECURITIES AND EXCHANGE COMMISSION PURSUANT TO RULE 24B-2
PROMULGATED UNDER THE SECURITIES EXCHANGE ACT OF 1934, AS AMENDED
|
8
d. |
Commencing on the date of first commercial sale of a License Product, AMICUS
shall, within 90 days from the last day of each June and December in each Calendar Year
during the term of the License, submit to MSSM a full and detailed report of royalties
or payments due MSSM under the terms of this Agreement for the preceding half year (the
Semi-Annual Report), setting forth the Net Sales and lump sum payments and all other
payments or consideration from sub-licensees upon which such royalties are computed and
including, on a Licensed Product-by-Licensed Product basis at least:
|
i) |
the quantity of Licensed Products used, sold, transferred or
otherwise disposed of,
|
||
ii) |
the selling price of each Licensed Product,
|
iii) |
the deductions permitted to arrive at Net Sales,
|
iv) |
the royalty computations and deductions therefrom based on
royalty payments to third parties.
|
e. |
At the request and expense of MSSM, AMICUS shall permit (and shall require its
sub-licensees to permit) an independent certified or chartered public accountant
appointed by MSSM, at reasonable times during normal business hours and upon reasonable
notice, but in any event no more than once per calendar year, to examine the records of
AMICUS (and its sub-licensees) to the extent necessary to verify royalty calculations
made hereunder; provided, however, that such examination shall be at the expense of
AMICUS if it reveals a discrepancy in the amount of royalties to be paid in MSSMs
favor of more than five percent. Results of such examination shall be made available to
both AMICUS and MSSM.
|
f. |
In connection with the Shire Agreement, in lieu of payments that might
otherwise be called for by Section 3.a above, and, as to royalties, subject to the
provisions of Section 3.b above, AMICUS shall pay to MSSM:
|
(1) |
Upfront Payment
: $2.635 million as MSSMs share of the
upfront payment received by AMICUS from Shire;
|
||
(2) |
Development and Regulatory
Milestone Payments
:
|
(a). |
[***] of the milestone payments AMICUS receives
from Shire in connection with Section 7.2.1 of the Shire Agreement;
|
||
(b). |
Provided however, in the event there is no
Valid Claim in the Shire Territory covering Amigal, then MSSM shall
receive [***] of the milestone payments relating to Amigal; and further
provided that in the event there is no Valid Claim in the Shire
Territory covering Plicera, then MSSM shall receive [***] of the
milestone payments relating to Plicera; and further provided that in
the event there is no Valid Claim in the Shire Territory covering
AT2220, then MSSM shall receive [***] of the milestone payments
relating to AT2220.
|
[***] |
INDICATES MATERIAL THAT HAS BEEN OMITTED AND FOR WHICH CONFIDENTIAL TREATMENT HAS BEEN REQUESTED.
ALL SUCH OMITTED MATERIAL HAS BEEN FILED WITH THE SECURITIES AND EXCHANGE COMMISSION PURSUANT TO RULE 24B-2
PROMULGATED UNDER THE SECURITIES EXCHANGE ACT OF 1934, AS AMENDED
|
9
(3) |
Sales Milestones
:
|
(a). |
[***] of the milestone payments AMICUS receives
in connection with Section 7.2.2 of the Shire Agreement (Sales
Milestones) if only one of the Shire Licensed Products comprising the
Aggregate Annual Net Sales (as used in such Section 7.2.2) is covered
by a Valid Claim in any of the countries in the Shire Territory;
|
(b). |
[***] of the Sales Milestones if only two of
the Shire Licensed Products comprising the Aggregate Annual Net Sales
(as used in such Section 7.2.2) are covered by a Valid Claim in any of
the countries in the Shire Territory; or
|
(c). |
[***] of the Sales Milestones if there is a
Valid Claim covering all three of the Shire Licensed Products in any of
the countries in the Shire Territory.
|
(4) |
Royalties
:
|
(a). |
[***] of Net Sales of a Shire Licensed Product
in any country in the Shire Territory for which there is a Valid Claim
covering the Shire Licensed Product in that country; and
|
(b). |
[***] of Net Sales of a Shire Licensed Product
sold in any country in the Shire Territory for which there is no Valid
Claim covering the Shire Licensed Product in that country.
|
4. |
Method of Payment.
|
a. |
Royalties and any other payments due to MSSM hereunder shall be paid to MSSM in
United States dollars.
|
b. |
AMICUS shall be responsible for prompt payment to MSSM of all royalties due on
sale, transfer or disposition of Licensed Products by the sub-licensees of AMICUS.
|
c. |
As to sales occurring in currencies other than U.S. Dollars, Net Sales shall
first be calculated in the currency in which sale occurred and then converted to U.S.
Dollars at the buying rate for such currency calculated as the average of the closing
buying rate for the first and last business day of the six month period for which
royalties are due, as set forth in the Wall Street Journal for such dates.
|
[***] |
INDICATES MATERIAL THAT HAS BEEN OMITTED AND FOR WHICH CONFIDENTIAL TREATMENT HAS BEEN REQUESTED.
ALL SUCH OMITTED MATERIAL HAS BEEN FILED WITH THE SECURITIES AND EXCHANGE COMMISSION PURSUANT TO RULE 24B-2
PROMULGATED UNDER THE SECURITIES EXCHANGE ACT OF 1934, AS AMENDED
|
10
5. |
Development and
Commercialization.
|
a. |
AMICUS shall use its commercially reasonable efforts to bring one or more
Licensed Products to market through a thorough, vigorous and diligent program for
exploitation of the Patent Rights in the Field. AMICUS shall not, however, be
required to pursue the development of more than one Licensed Product at a time, nor
shall AMICUS be required to pursue every possible Licensed Product.
|
b. |
Attached as Appendix A to this Agreement is the current development plan of
AMICUS for the forthcoming period of twelve months (such plan, as updated from time to
time as described in clause (c) below, the Plan). As and when appropriate, future
Plans will incorporate efficacy, pharmaceutical safety, toxicological and/ or clinical
tests or any other activities necessary in order to obtain the approval of the FDA and
counterpart foreign regulatory agencies for the production, use and sale of Licensed
Products, as well as marketing plans to commercialize Licensed Products that have
obtained such approvals.
|
c. |
On the earlier of thirty (30) days prior to the first anniversary of the
Effective Date or the end of AMICUSs first fiscal year, and thereafter on each
successive anniversary of such date, AMICUS shall deliver to MSSM a report setting
forth in reasonable detail progress and problems with the implementation of the Plan
and, providing an update on its efforts to commercialize Licensed Products, including a
forecast and schedule of major events required to market the Licensed Products. Such
report shall also include any amendments proposed by AMICUS to the Plan based upon the
progress made and then current scientific, regulatory and commercial exigencies
relating to Licensed Products. Within forty-five (45) days following the delivery of
such a report (a Diligence Report) representatives of MSSM may request a meeting with
AMICUS to review the Diligence Report, the status of the efforts of AMICUS under the
Plan and any proposed amendments to the Plan. Any such proposed amendments to the Plan
shall be subject to approval by MSSM, which approval shall not be unreasonably withheld
or delayed. Upon approval of any such amendments, they shall be deemed amendments to
the Plan, added to Appendix A and deemed incorporated into this Agreement.
|
d. |
AMICUS will use its commercially reasonable efforts to accomplish the
milestones described in the Plan.
|
e. |
Provided that applicable laws, rules and regulations so require, the
manufacture of Licensed Products shall be carried out by AMICUS or its agents in
accordance with FDA Good Laboratory Practices and FDA Good Manufacturing Practice
(GMP) procedures in a facility which has been certified by the FDA and the
performance of the tests, trials, studies and other activities specified in the Plan
shall be so performed by AMICUS or its agents in accordance with FDA clinical trial
procedures. MSSM shall have no responsibility for the actual production, distribution,
sale or use of any Licensed Product.
|
[***] |
INDICATES MATERIAL THAT HAS BEEN OMITTED AND FOR WHICH CONFIDENTIAL TREATMENT HAS BEEN REQUESTED.
ALL SUCH OMITTED MATERIAL HAS BEEN FILED WITH THE SECURITIES AND EXCHANGE COMMISSION PURSUANT TO RULE 24B-2
PROMULGATED UNDER THE SECURITIES EXCHANGE ACT OF 1934, AS AMENDED
|
11
f. |
If at any time AMICUS abandons or suspends its efforts to commercialize all
Licensed Products for a period exceeding ninety (90) days, AMICUS shall immediately
notify MSSM giving reasons and a statement of its intended actions.
MSSM shall be entitled to terminate this Agreement for Cause in accordance with
Section 11 upon any such abandonment.
|
g. |
MSSM shall also be entitled to terminate this Agreement for Cause in
accordance with Section 11 if AMICUS shall fail to deliver any Diligence Report on a
timely basis, or fail to use commercially reasonable efforts to implement the Plan, and
such failure is not cured within the sixty (60) day period set by the notice provided
pursuant to Section 11, unless such failure is excused by:
|
i) |
causes beyond AMICUSs direct control; or
|
||
ii) |
MSSMs failure to meet its obligations hereunder; or
|
iii) |
inaction of any federal or state agency whose approval is
required for commercial sales of Licensed Products.
|
h. |
Provided that applicable laws, rules and regulations so require, the
performance of the tests, trials, studies and other activities specified in subsection
b, above, shall be carried out in accordance with FDA Good Laboratory Practices and FDA
Good Manufacturing Practice (GMP) procedures in a facility which has been certified
by the FDA as complying with GMP. MSSM shall have no responsibility for the actual
production, distribution, sale or use of any Licensed Product.
|
6. |
Confidential Information.
|
a. |
In the course of research to be performed under this Agreement, it will be
necessary for each party to disclose Confidential Information to the other. For
purposes of this Agreement, Confidential Information is defined as all information,
data and know-how disclosed by one party (the Disclosing Party) to the other (the
Receiving Party), either embodied in tangible materials (including writings,
drawings, graphs, charts, photographs, recordings, structures, technical and other
information) marked Confidential or, if initially disclosed orally, which is reduced
to writing marked Confidential within 21 days after initial oral disclosure, other
than that information which is:
|
i) |
known by the Receiving Party at the time of its receipt, and
not through a prior disclosure by the Disclosing Party, as documented by the
Receiving Partys business records; or
|
ii) |
at the time of disclosure, or thereafter becomes, published or
otherwise part of the public domain without breach of this Agreement by the
Receiving Party; or
|
iii) |
obtained from a third party who has the legal right to make
such disclosure and without any confidentiality obligation to the Disclosing
Party; or
|
[***] |
INDICATES MATERIAL THAT HAS BEEN OMITTED AND FOR WHICH CONFIDENTIAL TREATMENT HAS BEEN REQUESTED.
ALL SUCH OMITTED MATERIAL HAS BEEN FILED WITH THE SECURITIES AND EXCHANGE COMMISSION PURSUANT TO RULE 24B-2
PROMULGATED UNDER THE SECURITIES EXCHANGE ACT OF 1934, AS AMENDED
|
12
iv) |
independently developed by the Receiving Party without the use
of Confidential Information received from the Disclosing Party and such
independent development can be documented by the Receiving Party; or
|
||
v) |
disclosed to governmental or other regulatory agencies in order
to obtain patents, provided that such disclosure may be made only to the extent
reasonably necessary to obtain such patents or authorizations, and further
provided that any such patent applications shall be filed in accordance with
the terms of this Agreement; or
|
||
vi) |
required by law, regulation, rule, act or order of any
governmental authority to be disclosed.
|
b. |
The Receiving Party agrees that at all times and notwithstanding any
termination, expiration, or cancellation hereunder, it will hold the Confidential
Information of the Disclosing Party in strict confidence, will use all reasonable
safeguards to prevent unauthorized disclosure by its employees and agents.
Notwithstanding the foregoing, the parties recognize that industry standards with
respect to the treatment of Confidential Information may not be appropriate in an
academic setting. However, MSSM agrees to retain Confidential Information of AMICUS in
the same manner and with the same level of confidentiality as MSSM retains its own
Confidential Information.
|
||
c. |
The Receiving Party will maintain reasonable procedures to prevent accidental
or other loss, including unauthorized publication of any Confidential Information of
the Disclosing Party. The Receiving Party will promptly notify the Disclosing Party in
the event of any loss or unauthorized disclosure of the Confidential Information.
|
||
d. |
Upon termination or expiration of this Agreement, and upon written request, the
Receiving Party will promptly return to the Disclosing Party all documents or other
tangible materials representing Confidential Information and all copies thereof.
|
||
e. |
The Receiving Party will immediately notify the Disclosing Party in writing, if
it is requested by a court order, a governmental agency, or any other entity to
disclose Confidential Information in the Receiving Partys possession. The Disclosing
Party will have an opportunity to intervene by seeking a protective order or other
similar order, in order to limit or prevent disclosure of the Confidential Information.
The Receiving Party will disclose only the minimum Confidential Information required to
be disclosed in order to comply, whether or not a protective order or other similar
order is obtained by the Disclosing Party.
|
[***] |
INDICATES MATERIAL THAT HAS BEEN OMITTED AND FOR WHICH CONFIDENTIAL TREATMENT HAS BEEN REQUESTED.
ALL SUCH OMITTED MATERIAL HAS BEEN FILED WITH THE SECURITIES AND EXCHANGE COMMISSION PURSUANT TO RULE 24B-2
PROMULGATED UNDER THE SECURITIES EXCHANGE ACT OF 1934, AS AMENDED
|
13
7. |
Patent Rights.
|
a. |
If either party to this Agreement acquires information that a third party is
infringing one or more of the Patent Rights, the party acquiring such information shall
promptly notify the other party to Agreement in writing of such infringement.
|
||
b. |
In the event of infringement of the Patent Rights, AMICUS shall have the right,
but not the obligation, to bring suit against the infringer. Should AMICUS elect to
bring suit against an infringer, AMICUS shall be entitled to retain counsel of its own
choosing, and shall have the right to join MSSM as party plaintiff in any such suit.
Except as otherwise provided herein, the expenses of such suit or suits that AMICUS
elects to bring, shall be paid for entirely by AMICUS and AMICUS shall hold MSSM free,
clear and harmless from and against any and all costs of such litigation, including
attorneys fees. AMICUS shall not compromise or settle such litigation without the
prior written consent of MSSM which shall not be unreasonably withheld.
|
||
c. |
If AMICUS shall undertake the enforcement or defense of the Patent Rights by
litigation, AMICUS may withhold royalties otherwise thereafter due MSSM hereunder and
apply the same toward reimbursement of up to half of AMICUSs expenses, including
reasonable attorneys fees, in connection therewith, provided however that the maximum
amount that can be withheld each year shall not exceed 50% of royalties due to MSSM in
that year.
|
||
d. |
If AMICUS exercises its right to sue, it shall first reimburse itself out of
any sums recovered in such suit or in settlement thereof for all costs and expenses of
every kind and character, including reasonable attorneys fees, necessarily involved in
the prosecution of any such suit, and if after such reimbursement, any funds shall
remain from said recovery, the amount of said funds shall be added to the amount of Net
Sales for the calendar quarter in which such recovery was made.
|
||
e. |
If AMICUS does not bring suit against said infringer pursuant to subsection b,
above, or has not commenced negotiations with said infringer for discontinuance of said
infringement, within 90 days after receipt of such notice, MSSM shall have the right,
but not the obligation, to bring suit for such infringement and to join AMICUS as a
party plaintiff, in which event MSSM shall hold AMICUS free, clear and harmless from
and against any and all costs and expenses of such litigation, including attorneys
fees. In the event MSSM brings suit for infringement of the Patent Rights, MSSM shall
have the right to first reimburse itself out of any sums recovered in such suit or
settlement thereof for all costs and expenses of every kind and character, including
reasonable attorneys fees necessarily involved in the prosecution of such suit, and if
after such reimbursement, any funds shall remain from said recovery, MSSM shall
promptly pay to AMICUS an amount equal to 50 percent of such remainder and MSSM shall be
entitled to receive and retain the balance of the remainder of such recovery.
|
[***] |
INDICATES MATERIAL THAT HAS BEEN OMITTED AND FOR WHICH CONFIDENTIAL TREATMENT HAS BEEN REQUESTED.
ALL SUCH OMITTED MATERIAL HAS BEEN FILED WITH THE SECURITIES AND EXCHANGE COMMISSION PURSUANT TO RULE 24B-2
PROMULGATED UNDER THE SECURITIES EXCHANGE ACT OF 1934, AS AMENDED
|
14
f. |
Each party shall have the right to be represented by counsel of its own
selection, at its sole expense, in any suit for infringement of the Patent Rights
instituted by the other party to this Agreement under the terms hereof.
|
||
g. |
AMICUS shall cooperate fully with MSSM at the request of MSSM, including, by
giving testimony and producing documents lawfully requested in the course of a suit
prosecuted by MSSM for infringement of the Patent Rights; provided MSSM shall pay all
reasonable expenses (including attorneys fees) incurred by AMICUS in connection with
such cooperation. MSSM shall cooperate with AMICUS in the prosecution of a suit by
AMICUS for infringement of the Patent Rights, provided that, except as otherwise
provided in Section 7.f., AMICUS shall pay all reasonable expenses (including
attorneys fees) involved in such cooperation.
|
||
h. |
AMICUS has reimbursed MSSM for all of the reasonable and customary fees and
expenses incurred by MSSM as of the Effective Date, in the prosecution and maintenance
of the Patent Rights. In addition, AMICUS reimbursed MSSM for $100,000 in total
payments to Jian-Qiang Fan and Satoshi Ishii pursuant to the letter of agreement dated
March 24, 2000 between MSSM and Jian-Qiang Fan and Satoshi Ishii.
|
8. |
Patent Prosecution.
|
a. |
MSSM is the owner of the Patent Rights. AMICUS shall have the sole right and
responsibility, and have the sole right to retain legal counsel of its choice to assist
AMICUS, to prepare, file, prosecute, and maintain the pending patent applications and
issued patents comprising the Patent Rights.
|
||
b. |
AMICUS shall keep MSSM informed of its actions on all patent prosecution and
patent maintenance matters related to the Patent Rights. AMICUS shall send to MSSM:
|
i) |
Copies of any document pertaining to the ongoing prosecution of
the Patent Rights received from the U.S. Patent and Trademark Office; and
|
||
ii) |
Copies of any documents submitted to the U.S. Patent and
Trademark Office (or any other patent granting authority) in any such patents
or applications.
|
[***] |
INDICATES MATERIAL THAT HAS BEEN OMITTED AND FOR WHICH CONFIDENTIAL TREATMENT HAS BEEN REQUESTED.
ALL SUCH OMITTED MATERIAL HAS BEEN FILED WITH THE SECURITIES AND EXCHANGE COMMISSION PURSUANT TO RULE 24B-2
PROMULGATED UNDER THE SECURITIES EXCHANGE ACT OF 1934, AS AMENDED
|
15
c. |
AMICUS shall have the sole right to determine to what extent each patent
application falling within the Patent Rights and covered by this Agreement should
be prosecuted worldwide. If at any time during the term of this Agreement AMICUS
decides that it is undesirable, as to one or more countries, to prosecute any patent
applications within the Patent Rights, it shall give prompt written notice thereof
to MSSM, and upon receipt of such notice AMICUS shall be released from its
obligations to bear all of the expenses to be incurred thereafter as to such
countries in conjunction with such patent application(s).
|
i) |
Notify MSSM of its intention to abandon such patent or U.S.
patent application at least twenty (20) days prior to the last date for taking
action to preserve such patent;
|
||
ii) |
Permit MSSM to maintain such patent(s) or U.S. patent
application(s) at MSSMs sole expense;
|
||
iii) |
Should MSSM decide to maintain such patent(s) or U.S. patent
application(s), the litigation rights outlined in Section 7 will revert to MSSM
and AMICUS shall be released from its obligations to bear all or any of the
expenses, including but not limited to prosecution, maintenance or litigation
expensed, incurred thereafter as to such patent(s) or patent application(s).
|
d. |
Except as otherwise expressly provided herein, AMICUS shall bear all costs and
fees incurred during the term of this Agreement in connection with the filing,
maintenance, prosecution, protection and the like of the Patent Rights.
|
||
e. |
Nothing herein contained shall be deemed to be a warranty by MSSM that the
manufacture, use, or sale of any element of the Patent Rights or any Licensed Product
will not infringe any patent(s) of a third party.
|
9. |
Liability and Indemnification.
|
a. |
AMICUS shall indemnify, defend and hold harmless MSSM and its trustees,
officers, directors, medical and professional staff, employees, students and agents and
their respective successors, heirs and assigns (the Indemnitees), against any
liability, damage, loss or expense (including reasonable attorneys fees and expenses
of litigation) incurred by or imposed upon the Indemnitees or any one of them in
connection with any claims, suits, actions, demands or judgments: (i) arising out of
the production, manufacture, sale, use in commerce or in human clinical trials, lease,
or promotion by AMICUS or by a licensee, Affiliate or agent of AMICUS of any Licensed
Product, process or service relating to, or developed pursuant to, this Agreement, or
(ii) arising out of any other activities to be carried out pursuant to this Agreement.
|
[***] |
INDICATES MATERIAL THAT HAS BEEN OMITTED AND FOR WHICH CONFIDENTIAL TREATMENT HAS BEEN REQUESTED.
ALL SUCH OMITTED MATERIAL HAS BEEN FILED WITH THE SECURITIES AND EXCHANGE COMMISSION PURSUANT TO RULE 24B-2
PROMULGATED UNDER THE SECURITIES EXCHANGE ACT OF 1934, AS AMENDED
|
16
b. |
AMICUSs indemnification under subsection a(i), above, shall apply to any
liability, damage, loss or expense whether or not it is attributable to the negligent
activities of the Indemnitees. AMICUSs indemnification under subsection a (ii), above,
shall not apply to any liability, damage, loss or expense to the extent that it is
attributable to the negligence, gross negligence or intentional misconduct of the
Indemnitees.
|
||
c. |
AMICUS shall, at its own expense, provide attorneys reasonably acceptable to
MSSM to defend against any actions brought or filed against any party indemnified
hereunder with respect to the subject of indemnity contained herein, whether or not
such actions are rightfully brought.
|
||
d. |
EXCEPT AS PROVIDED IN THIS SECTION 9, NEITHER PARTY SHALL BE LIABLE TO THE
OTHER FOR INCIDENTAL, CONSEQUENTIAL, SPECIAL, EXEMPLARY OR PUNITIVE DAMAGES.
|
10. |
Security for Indemnification.
|
a. |
At such time as any Licensed Product is being commercially distributed or sold
(other than for the purpose of obtaining regulatory approvals) by AMICUS or by a
sub-licensee, Affiliate or agent of AMICUS and to the extent that it is available on
commercially reasonable terms, AMICUS shall at its sole cost and expense, procure and
maintain policies of comprehensive general liability insurance in amounts not less than
[***] per incident and [***] annual aggregate and naming the indemnitees as additional
insureds. Such comprehensive general liability insurance shall provide (i) product
liability coverage and (ii) broad form contractual liability coverage for AMICUSs
indemnification under Section 9 of this Agreement. The minimum amounts of insurance
coverage required under this Section 10 shall not be construed as a limit of AMICUSs
liability with respect to its indemnification under Section 9 of this Agreement.
|
||
b. |
AMICUS shall provide MSSM with written evidence of such insurance upon request
of MSSM. AMICUS shall provide MSSM with written notice at least 60 days prior to the
cancellation, non-renewal or material change in such insurance; if AMICUS does not
obtain replacement insurance providing comparable coverage within such 60 day period
effective immediately upon notice to AMICUS, MSSM shall have the right to terminate
this Agreement effective at the end of such 60 day period without notice or any
additional waiting periods.
|
||
c. |
AMICUS shall maintain such comprehensive general liability insurance beyond the
expiration or termination of this Agreement during: (i) the period that any product,
process or service, relating to, or developed pursuant to, this Agreement is being
commercially distributed or sold (other than for the purpose of obtaining regulatory
approvals) by AMICUS or by a licensee, Affiliate or agent of
AMICUS and (ii) a reasonable period after the period referred to in (c)(i) above
which in no event shall be less than seven years.
|
[***] |
INDICATES MATERIAL THAT HAS BEEN OMITTED AND FOR WHICH CONFIDENTIAL TREATMENT HAS BEEN REQUESTED.
ALL SUCH OMITTED MATERIAL HAS BEEN FILED WITH THE SECURITIES AND EXCHANGE COMMISSION PURSUANT TO RULE 24B-2
PROMULGATED UNDER THE SECURITIES EXCHANGE ACT OF 1934, AS AMENDED
|
17
11. |
Term and Termination.
|
a. |
This Agreement shall come into force as of the Effective Date. Unless sooner
terminated as provided herein, this Agreement shall expire on the expiration of the
last to expire of the Patent Rights.
|
||
b. |
At any time prior to expiration of the term of this Agreement either party may
terminate this Agreement forthwith for cause upon notice to the other party. Cause
for termination of this Agreement shall be deemed to exist if either MSSM or AMICUS
materially breaches or defaults in the performance or observance of any of the
provisions of this Agreement and such breach or default is not cured within 60 days or,
in the case of failure to pay any amounts due hereunder, 30 days (unless otherwise
specified herein) after the giving of notice by the other party specifying such breach
or default, or if either MSSM or AMICUS discontinues its business or becomes insolvent
or bankrupt.
|
||
c. |
Any amount payable hereunder by one of the parties to the other, which has not
been paid by its due date of payment shall bear interest from its due date of payment
until the date of actual payment, at the rate of two percent per annum in excess of the
Prime Rate prevailing at the Citibank, Inc., New York, New York, during the period of
arrears and such amount and the interest thereon may be set off against any amount due,
whether in terms of this Agreement or otherwise, to the party in default by any
non-defaulting party.
|
||
d. |
Upon termination of this Agreement for any reason, all rights in and to the
Patent Rights shall revert to MSSM.
|
||
e. |
Termination of this Agreement shall not relieve the parties of any obligation
occurring prior to such termination.
|
||
f. |
Sections 2e., 3e., 6, 9, 10 and 14 hereof shall survive and remain in full
force and effect after any termination, cancellation or expiration of this Agreement.
|
12. |
Representation, Covenants and Acknowledgements.
|
a. |
MSSM hereby represents, warrants, and covenants to AMICUS that it is a
corporation duly organized and validly existing under the laws of the state or other
jurisdiction of its incorporation or formation;
|
||
b. |
AMICUS hereby represents, warrants and covenants to the other party hereto that
it is a corporation duly organized and validly existing under the laws of the state or
other jurisdiction of its incorporation or formation;
|
[***] |
INDICATES MATERIAL THAT HAS BEEN OMITTED AND FOR WHICH CONFIDENTIAL TREATMENT HAS BEEN REQUESTED.
ALL SUCH OMITTED MATERIAL HAS BEEN FILED WITH THE SECURITIES AND EXCHANGE COMMISSION PURSUANT TO RULE 24B-2
PROMULGATED UNDER THE SECURITIES EXCHANGE ACT OF 1934, AS AMENDED
|
18
c. |
Each of MSSM and AMICUS hereby represents, warrants and covenants to the other
party hereto as follows:
|
i) |
the execution, delivery and performance of this Agreement by
such party has been duly authorized by all requisite corporate action;
|
||
ii) |
it has the power and authority to execute and deliver this
Agreement and to perform its obligations hereunder;
|
||
iii) |
the execution, delivery and performance by such party of this
Agreement and its compliance with the terms and provisions hereof is not
prohibited and does not and will result in a breach of any of the terms and
provisions of, or constitute a default under, (i) a loan agreement, guaranty,
financing agreement, agreement affecting a product, or other agreement or
instrument binding or affecting it or its property; (ii) the provisions of its
charter documents or bylaws; or (iii) any order, writ, injunction or decree of
any court or governmental authority entered against it or by which any of its
property is bound;
|
||
iv) |
the execution, delivery and performance of this Agreement by
such party does not require the consent, approval, or authorization of, or
notice, declaration, filing or registration with, any governmental or
regulatory authority, and the execution, delivery or performance of this
Agreement will not violate any law, rule or regulation applicable to such
party;
|
||
v) |
this Agreement has been duly authorized, executed and delivered
and constitutes such partys legal, valid and binding obligation enforceable
against it in accordance with its terms subject, as to enforcement, to
bankruptcy, insolvency, reorganization and other laws of general applicability
relating to or affecting creditors rights and to the availability of
particular remedies under general equity principles; and
|
||
vi) |
it shall comply with all applicable material laws and
regulations relating to its activities under this Agreement.
|
||
vii) |
Each party represents that performance of all the terms of this
Agreement will not breach any agreement to keep in confidence proprietary
information acquired by a party prior to the execution of this Agreement.
|
[***] |
INDICATES MATERIAL THAT HAS BEEN OMITTED AND FOR WHICH CONFIDENTIAL TREATMENT HAS BEEN REQUESTED.
ALL SUCH OMITTED MATERIAL HAS BEEN FILED WITH THE SECURITIES AND EXCHANGE COMMISSION PURSUANT TO RULE 24B-2
PROMULGATED UNDER THE SECURITIES EXCHANGE ACT OF 1934, AS AMENDED
|
19
d. |
Except as otherwise expressly provided herein, MSSM hereby represents, warrants
and covenants to AMICUS that:
|
i) |
MSSM has the full right, power and authority to grant all of
the right, title and interest in the License; and
|
||
ii) |
there are no judgments or settlements against or owed by MSSM,
or any pending or threatened claims or litigation relating to MSSMs interest
in the Patent Rights; and
|
||
iii) |
MSSM has not granted to any other party any rights that would
conflict with the rights granted in this Agreement.
|
e. |
In connection with the issuance of the Shares to MSSM, MSSM acknowledges and
agrees as follows:
|
i) |
Limited Transferability
. MSSM acknowledges that the
Shares issued to it under this Agreement have not been registered under the
Securities Act of 1933, as amended (the 1933 Act), and have been offered and
sold pursuant to applicable exemptions from such registration and will be
issued as restricted securities as defined by Rule 144 promulgated pursuant
to the 1933 Act. MSSM acknowledges that the Shares may not be resold in the
absence of an effective registration thereof under the 1933 Act and applicable
state securities laws or unless, in the opinion of counsel of AMICUS, an
applicable exemption from registration is available. MSSM acknowledges that the
certificate evidencing the Shares issued pursuant to this Agreement will bear a
restrictive legend with respect to the foregoing.
|
||
ii) |
Suitability; Accredited Investor
. MSSM represents that
it has such knowledge in business and financial matters that it is capable of
evaluating the risks and merits of an investment in the Shares. MSSM further
represents that it is an accredited investor as defined in Rule 501 of
Regulation D promulgated by the Securities and Exchange Commission under the
1933 Act.
|
||
iii) |
Acquisition for Investment
. MSSM represents that it has
acquired the Shares for its own account, for investment purposes only and not
with a view to, or for sale in connection with, a distribution, as that term is
used in Section 2(11) of the 1933 Act thereof in a manner which would require
registration under the 1933 Act of any state securities laws.
|
||
iv) |
Access to Information
. In the decision to acquire the
Shares, MSSM relied upon independent investigations made by it or its
representatives and it and/or such representatives have been given the
opportunity to examine all relevant documents concerning AMICUS and to ask
questions of, and to receive answers from, AMICUS or any person(s) acting on
its behalf.
|
[***] |
INDICATES MATERIAL THAT HAS BEEN OMITTED AND FOR WHICH CONFIDENTIAL TREATMENT HAS BEEN REQUESTED.
ALL SUCH OMITTED MATERIAL HAS BEEN FILED WITH THE SECURITIES AND EXCHANGE COMMISSION PURSUANT TO RULE 24B-2
PROMULGATED UNDER THE SECURITIES EXCHANGE ACT OF 1934, AS AMENDED
|
20
13. |
Assignment.
|
14. |
Use of Name.
|
15. |
Miscellaneous.
|
a. |
In carrying out this Agreement the parties shall comply with all local, state
and federal laws and regulations including but not limited to, the provisions of Title
35 § 200 et seq. and 15 CFR § 368 et seq.
|
||
b. |
If any provision of this Agreement is determined to be invalid or void, the
remaining provisions shall remain in effect.
|
||
c. |
This Agreement shall be deemed to have been made in the State of New York and
shall be governed and interpreted in all respects under the laws of the State of New
York. Any and all disputes hereunder shall be brought and resolved solely in the courts
of the State of New York in and for the Borough of Manhattan.
|
||
d. |
All payments or notices required or permitted to be given under this agreement
shall be given in writing and shall be effective when either personally delivered or
deposited, postage prepaid, in the United States registered or certified mail,
addressed as follows:
|
|
To MSSM: | Mount Sinai School of Medicine of New York University | ||
|
Attention: W. Patrick McGrath, Ph.D. | |||
|
One Gustave L. Levy Place | |||
|
New York, New York 10029-6574 | |||
|
||||
|
Copy to: | General Counsel (at the same address) | ||
|
||||
|
To AMICUS: | Amicus Therapuetics, Inc | ||
|
6 Cedar Brook Drive | |||
|
Cranbury, NJ 08512 | |||
|
Attention: John F. Crowley | |||
|
||||
|
Copy to: | General Counsel (at the same address) |
[***] |
INDICATES MATERIAL THAT HAS BEEN OMITTED AND FOR WHICH CONFIDENTIAL TREATMENT HAS BEEN REQUESTED.
ALL SUCH OMITTED MATERIAL HAS BEEN FILED WITH THE SECURITIES AND EXCHANGE COMMISSION PURSUANT TO RULE 24B-2
PROMULGATED UNDER THE SECURITIES EXCHANGE ACT OF 1934, AS AMENDED
|
21
e. |
This Agreement and the exhibits attached hereto constitute the entire Agreement
between the parties with respect to the subject matter hereof and no variations,
modification or waiver of any of the terms or conditions hereof shall be deemed valid
unless made in writing and signed by both parties hereto. This Agreement supersedes any
and all prior agreements or understandings, whether oral or written, between AMICUS and
MSSM.
|
||
f. |
No waiver by either party of any non-performance or violation by the other
party of any of the covenants, obligations or agreements of such other party hereunder
shall be deemed to be a waiver of any subsequent violation or non-performance of the
same or any other covenant, agreement or obligation, nor shall forbearance by any party
be deemed to be a waiver by such party of its rights or remedies with respect to such
violation or non-performance.
|
||
g. |
The descriptive headings contained in this Agreement are included for
convenience and reference only and shall not be held to expand, modify or aid in the
interpretation, construction or meaning of this Agreement.
|
||
h. |
It is not the intent of the parties to create a partnership or joint venture or
to assume partnership responsibility or liability. The obligations of the parties shall
be limited to those set out herein and such obligations shall be several and not joint.
|
MOUNT SINAI SCHOOL OF MEDICINE | AMICUS THERAPEUTICS, INC | |||||||
OF NEW YORK UNIVERSITY | ||||||||
|
||||||||
By:
|
/s/ Dennis S. Charney, M.D. | By: | /s/ John F. Crowley | |||||
|
Name: Dennis S. Charney, M.D. | Name: John F. Crowley | ||||||
|
Title: Dean
|
Title: President and CEO | ||||||
Date:
|
Date: | |||||||
|
|
|
[***] |
INDICATES MATERIAL THAT HAS BEEN OMITTED AND FOR WHICH CONFIDENTIAL TREATMENT HAS BEEN REQUESTED.
ALL SUCH OMITTED MATERIAL HAS BEEN FILED WITH THE SECURITIES AND EXCHANGE COMMISSION PURSUANT TO RULE 24B-2
PROMULGATED UNDER THE SECURITIES EXCHANGE ACT OF 1934, AS AMENDED
|
22
i. |
Neurological disorders:
|
|
Neurological disorders and their corresponding wild-types protein targets:
|
|
Parkinsons disease and glucocerebrosidase:
|
|
Niemann-Pick Type C Disease and glucocerebrosidase;
|
||
|
Alzheimers disease and
ά
-secretase; and
|
||
|
Alzheimers Disease and Pinl.
|
ii. |
Cancer
|
|
Cancer and Phosphotase and Tensin Homolog (PTEN).
|
iii. |
Cardiovascular disease:
|
|
Hyperlipidemia and lipoprotein lipase.
|
a. |
Hyperlipidemia includes (i) any pre-condition
in which there is the potential for hyperlipidemia to develop; (ii) any
condition in which hyperlipidemia is manifest.
|
[***] |
INDICATES MATERIAL THAT HAS BEEN OMITTED AND FOR WHICH CONFIDENTIAL TREATMENT HAS BEEN REQUESTED.
ALL SUCH OMITTED MATERIAL HAS BEEN FILED WITH THE SECURITIES AND EXCHANGE COMMISSION PURSUANT TO RULE 24B-2
PROMULGATED UNDER THE SECURITIES EXCHANGE ACT OF 1934, AS AMENDED
|
23
a. |
Individuals having no mutated proteins but in whom it would be beneficial to increase the
activity of specific wile-type proteins which are already expressed.
|
i. |
e.g. glucocerebrosidase in Parkinsons patients who do not have any mutant
alleles
|
||
ii. |
glucocerebrosidase in Neimann-Pick Type C in patients who do not have any
mutant alleles
|
b. |
Individuals having heterozygous, non-conformational mutations (i.e., on one allele),
resulting in decreased levels of a protein, who would benefit by increasing activity of the
protein expressed from the wild-type allele.
|
i. |
e.g., Parkinsons in patients have a mutation in GCase on one allele, but which
mutation is not a conformational mutation and where the patients do not have Gaucher
disease
|
[***] |
INDICATES MATERIAL THAT HAS BEEN OMITTED AND FOR WHICH CONFIDENTIAL TREATMENT HAS BEEN REQUESTED.
ALL SUCH OMITTED MATERIAL HAS BEEN FILED WITH THE SECURITIES AND EXCHANGE COMMISSION PURSUANT TO RULE 24B-2
PROMULGATED UNDER THE SECURITIES EXCHANGE ACT OF 1934, AS AMENDED
|
24
1. |
six (6) months salary continuation to be paid in accordance
with the Companys payroll practices;
|
2. |
an additional six (6) months of option vesting;
|
3. |
in the event that your termination occurs after June
30
th
of the calendar year, you will be entitled to a payment of a
bonus equal to the bonus earned in the preceding year pro-rated for the number
of months actually worked in the year of termination, payable on the date of
termination; and
|
4. |
you will be entitled to a continuation of your health benefit
coverage under COBRA, premiums to be paid by the Company, for a period of
twelve (12) months, which shall commence on the date of termination and run
concurrently with the period of salary continuation.
|
2
3
4
Amicus Therapeutics, Inc.
|
||||
By: | /s/ John F. Crowley | |||
John F. Crowley | ||||
President and Chief Executive Officer |
Accepted and Agreed:
|
||||
|
||||
By:
|
/s/ David Palling | |||
|
|
5
1. |
six (6) months salary continuation to be paid in accordance
with the Companys payroll practices;
|
2. |
an additional six (6) months of option vesting;
|
3. |
in the event that your termination occurs after June
30
th
of the calendar year, you will be entitled to a payment of a
bonus equal to the bonus earned in the preceding year pro-rated for the number
of months actually worked in the year of termination, payable on the date of
termination; and
|
4. |
you will be entitled to a continuation of your health benefit
coverage under COBRA, premiums to be paid by the Company, for a period of
twelve (12) months, which shall commence on the date of termination and run
concurrently with the period of salary continuation.
|
2
3
4
Amicus Therapeutics, Inc.
|
||||
By: | /s/ John F. Crowley | |||
John F. Crowley | ||||
President and Chief Executive Officer |
Accepted and Agreed: | ||||
|
||||
By:
|
/s/ Bradley Campbell | |||
|
|
5
1. |
six (6) months salary continuation to be paid in accordance
with the Companys payroll practices;
|
||
2. |
an additional six (6) months of option vesting;
|
||
3. |
in the event that your termination occurs after June
30
th
of the calendar year, you will be entitled to a payment of a
bonus equal to the bonus earned in the preceding year pro-rated for the number
of months actually worked in the year of termination, payable on the date of
termination; and
|
4. |
you will be entitled to a continuation of your health benefit
coverage under COBRA, premiums to be paid by the Company, for a period of
twelve (12) months, which shall commence on the date of termination and run
concurrently with the period of salary continuation.
|
2
3
4
Amicus Therapeutics, Inc.
|
||||
By: | /s/ John F. Crowley | |||
John F. Crowley | ||||
President and Chief Executive Officer |
Accepted and Agreed: | ||||
|
||||
By:
|
/s/ S. Nicole Schaeffer | |||
|
|
5
1. |
six (6) months salary continuation to be paid in accordance
with the Companys payroll practices;
|
2. |
an additional six (6) months of option vesting;
|
3. |
in the event that your termination occurs after June
30
th
of the calendar year, you will be entitled to a payment of a
bonus equal to the bonus earned in the preceding year pro-rated for the number
of months actually worked in the year of termination, payable on the date of
termination; and
|
4. |
you will be entitled to a continuation of your health benefit
coverage under COBRA, premiums to be paid by the Company, for a period of
twelve (12) months, which shall commence on the date of termination and run
concurrently with the period of salary continuation.
|
2
3
4
Amicus Therapeutics, Inc.
|
||||
By: | /s/ John F. Crowley | |||
John F. Crowley | ||||
President and Chief Executive Officer |
By:
|
/s/ John Kirk | |||
|
|
5
1. |
six (6) months salary continuation to be paid in accordance
with the Companys payroll practices;
|
2. |
an additional six (6) months of option vesting;
|
3. |
in the event that your termination occurs after June
30
th
of the calendar year, you will be entitled to a payment of a
bonus equal to the bonus earned in the preceding year pro-rated for the number
of months actually worked in the year of termination, payable on the date of
termination; and
|
4. |
you will be entitled to a continuation of your health benefit
coverage under COBRA, premiums to be paid by the Company, for a period of
twelve (12) months, which shall commence on the date of termination and run
concurrently with the period of salary continuation.
|
2
3
4
Amicus Therapeutics, Inc.
|
||||
By: | /s/ John F. Crowley | |||
John F. Crowley | ||||
President and Chief Executive Officer |
By:
|
/s/ Andrew Shenker | |||
|
|
5
1. |
six (6) months salary continuation to be paid in accordance
with the Companys payroll practices;
|
2. |
an additional six (6) months of option vesting;
|
3. |
in the event that your termination occurs after June
30
th
of the calendar year, you will be entitled to a payment of a
bonus equal to the bonus earned in the preceding year pro-rated for the number
of months actually worked in the year of termination, payable on the date of
termination; and
|
4. |
you will be entitled to a continuation of your health benefit
coverage under COBRA, premiums to be paid by the Company, for a period of
twelve (12) months, which shall commence on the date of termination and run
concurrently with the period of salary continuation.
|
2
3
4
Amicus Therapeutics, Inc.
|
||||
By: | /s/ John F. Crowley | |||
John F. Crowley | ||||
President and Chief Executive Officer |
Accepted and Agreed: | ||||
|
||||
By:
|
/s/ Geoffrey P. Gilmore | |||
|
||||
|
Geoffrey P. Gilmore |
5
|
/s/ ERNST & YOUNG LLP |
a. | designed such disclosure controls and procedures, or caused such disclosure controls and procedures to be designed under our supervision, to ensure that material information relating to the registrant, including its consolidated subsidiaries, is made known to us by others within those entities, particularly during the period in which this annual report is being prepared; |
b. | designed such internal control over financial reporting, or caused such internal control over financial reporting to be designed under our supervision, to provide reasonable assurance regarding the reliability of financial reporting and the preparation of financial statements for external purposes in accordance with generally accepted accounting principles; |
c. | evaluated the effectiveness of the registrants disclosure controls and procedures and presented in this report our conclusions about the effectiveness of the disclosure controls and procedures, as of the end of the period covered by this report based on such evaluation; and |
d. | disclosed in this report any change in the registrants internal control over financial reporting that occurred during the registrants most recent fiscal quarter (the registrants fourth fiscal quarter in the case of an annual report) that has materially affected, or is reasonably likely to materially affect, the registrants internal control over financial reporting; |
a. | all significant deficiencies and material weaknesses in the design or operation of internal control over financial reporting which are reasonably likely to adversely affect the registrants ability to record, process, summarize and report financial information; and |
b. | any fraud, whether or not material, that involves management or other employees who have a significant role in the registrants internal control over financial reporting. |
/s/ John F. Crowley
|
||
John F. Crowley
|
||
Chief Executive Officer
|
a. | designed such disclosure controls and procedures, or caused such disclosure controls and procedures to be designed under our supervision, to ensure that material information relating to the registrant, including its consolidated subsidiaries, is made known to us by others within those entities, particularly during the period in which this annual report is being prepared; |
b. | designed such internal control over financial reporting, or caused such internal control over financial reporting to be designed under our supervision, to provide reasonable assurance regarding the reliability of financial reporting and the preparation of financial statements for external purposes in accordance with generally accepted accounting principles; |
c. | evaluated the effectiveness of the registrants disclosure controls and procedures and presented in this report our conclusions about the effectiveness of the disclosure controls and procedures, as of the end of the period covered by this report based on such evaluation; and |
d. | disclosed in this report any change in the registrants internal control over financial reporting that occurred during the registrants most recent fiscal quarter (the registrants fourth fiscal quarter in the case of an annual report) that has materially affected, or is reasonably likely to materially affect, the registrants internal control over financial reporting; |
a. | all significant deficiencies and material weaknesses in the design or operation of internal control over financial reporting which are reasonably likely to adversely affect the registrants ability to record, process, summarize and report financial information; and |
b. | any fraud, whether or not material, that involves management or other employees who have a significant role in the registrants internal control over financial reporting. |
/s/ James E. Dentzer
|
||
James E. Dentzer
|
||
Chief Financial Officer
|
/s/ John F. Crowley
|
||
John F. Crowley
|
||
Chief Executive Officer
|
||
February 4, 2009
|
/s/ James E. Dentzer
|
||
James E. Dentzer
|
||
Chief Financial Officer
|
||
February 4, 2009
|