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SECURITIES AND EXCHANGE COMMISSION
SECURITIES EXCHANGE ACT OF 1934
Delaware | 71-0869350 | |
(State or other jurisdiction of | (IRS Employer | |
incorporation or organization) | Identification No.) |
(Address of principal executive offices)
Telephone: (609) 662-2000
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 filero | Accelerated filero | Non-accelerated filerþ | Smaller Reporting Companyo |
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• | 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 | ||
Biodistribution | Variable tissue distribution | Broad tissue distribution, including brain | ||
Ease of Use | Weekly or every other week intravenous infusion | Oral administration | ||
Manufacturing | Recombinant protein manufacturing | Chemical synthesis |
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Stage of | Worldwide | |||||||||||
Product Candidate | Indication | Development | Commercial Rights | |||||||||
Amigal(migalastat hydrochloride) | Fabry | Phase 2 Complete | Amicus U.S., | |||||||||
Disease | Phase 3 in Planning | Shire ex-U.S. | ||||||||||
Plicera(isofagomine tartrate) | Gaucher | Amicus U.S., | ||||||||||
Disease | Phase 2 Ongoing | Shire ex-U.S. | ||||||||||
AT2220 (1-deoxynojirimycin HCl) | 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 ofa-GAL, and methods for the treatment of Fabry disease using Amigal and other specific competitive inhibitors ofa-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) | ||||||||||||||||||||
Genzyme Corporation | 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 | ||||||||||||||||
Shire | Fabry disease | Replagal® | Enzyme Replacement Therapy | Marketed | $ | 132 | * | |||||||||||||
Gaucher disease | GA-GCB | Enzyme Replacement Therapy | Phase 3 | N/A | ||||||||||||||||
Actelion, Ltd. | Gaucher disease | Zavesca® | Substrate Reduction Therapy | Marketed | $ | 29 | * | |||||||||||||
Protalix Biotherapeutics | 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 product’s 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 distributor’s business strategy may also adversely affect a distributor’s 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 management’s 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 candidate’s benefits outweigh its risks; |
• | our inability to demonstrate that the product candidate is at least as effective as existing therapies; |
• | the FDA’s or comparable regulatory authorities’ disagreement with the manner in which we interpret the data from preclinical studies or clinical trials; |
• | the FDA’s 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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Item 5. MARKET FOR THE REGISTRANT’S COMMON EQUITY, RELATED STOCKHOLDER MATTERS AND ISSUER PURCHASES OF EQUITY SECURITIES. |
High | Low | |||||||
2008 | ||||||||
First Quarter | $ | 11.84 | $ | 9.00 | ||||
Second Quarter | 12.35 | 9.00 | ||||||
Third Quarter | 18.00 | 10.52 | ||||||
Fourth Quarter | 15.78 | 7.16 |
High | Low | |||||||
2007 | ||||||||
Second Quarter (from May 31, 2007) | $ | 16.80 | $ | 10.30 | ||||
Third Quarter | 16.75 | 10.00 | ||||||
Fourth Quarter | 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 | |||||||||||||||||||||||||
Amicus Therapeutics, Inc. | 100 | 80 | 116 | 74 | 74 | 74 | 105 | 55 | ||||||||||||||||||||||||
NASDAQ Composite | 100 | 100 | 104 | 102 | 88 | 88 | 88 | 61 | ||||||||||||||||||||||||
NASDAQ Biotechnology | 100 | 96 | 102 | 100 | 93 | 95 | 100 | 87 |
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(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 | ||||||||||||
October 1, 2008 – October 31, 2008 | 220 | $ | 15.10 | — | 5,295 | |||||||||||
November 1, 2008 – November 31, 2008 | 220 | $ | 10.45 | — | 5,075 | |||||||||||
December 1, 2008 – December 31, 2008 | 220 | $ | 7.98 | — | 4,855 | |||||||||||
Total | 660 | — | ||||||||||||||
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Period from | ||||||||||||||||||||||||
February 4, | ||||||||||||||||||||||||
2002 | ||||||||||||||||||||||||
(inception) to | ||||||||||||||||||||||||
Year Ended December 31 | December 31, | |||||||||||||||||||||||
2004 | 2005 | 2006 | 2007 | 2008 | 2008 | |||||||||||||||||||
Statement of Operations Data: | ||||||||||||||||||||||||
Revenue: | ||||||||||||||||||||||||
Research revenue | $ | — | $ | — | $ | — | $ | 1,375 | $ | 12,189 | $ | 13,564 | ||||||||||||
Collaboration revenue | — | — | — | 409 | 2,778 | 3,187 | ||||||||||||||||||
Total revenue | — | — | — | 1,784 | 14,967 | 16,751 | ||||||||||||||||||
Operating expenses: | ||||||||||||||||||||||||
Research and development | 6,301 | 13,652 | 33,630 | 31,074 | 37,764 | 127,642 | ||||||||||||||||||
General and administrative | 2,081 | 6,877 | 12,277 | 15,278 | 19,666 | 57,736 | ||||||||||||||||||
Impairment of leasehold improvements | — | — | — | — | — | 1,030 | ||||||||||||||||||
Depreciation and amortization | 146 | 303 | 952 | 1,237 | 1,493 | 4,287 | ||||||||||||||||||
In-process research and development | — | — | — | — | — | 418 | ||||||||||||||||||
Total operating expenses | 8,528 | 20,832 | 46,859 | 47,589 | 58,923 | 191,113 | ||||||||||||||||||
Loss from operations | (8,528 | ) | (20,832 | ) | (46,859 | ) | (45,805 | ) | (43,956 | ) | (174,362 | ) | ||||||||||||
Other income (expenses): | ||||||||||||||||||||||||
Interest income | 190 | 610 | 1,990 | 5,135 | 4,819 | 12,760 | ||||||||||||||||||
Interest expense | (550 | ) | (82 | ) | (273 | ) | (348 | ) | (218 | ) | (1,648 | ) | ||||||||||||
Change in fair value of warrant liability | (2 | ) | (280 | ) | (23 | ) | (149 | ) | — | (454 | ) | |||||||||||||
Other expense | — | — | (1,180 | ) | — | — | (1,180 | ) | ||||||||||||||||
Loss before tax benefit | (8,890 | ) | (20,584 | ) | (46,345 | ) | (41,167 | ) | (39,355 | ) | (164,884 | ) | ||||||||||||
Income tax benefit | 83 | 612 | — | — | — | 695 | ||||||||||||||||||
Net loss | (8,807 | ) | (19,972 | ) | (46,345 | ) | (41,167 | ) | (39,355 | ) | (164,189 | ) | ||||||||||||
Deemed dividend | — | — | (19,424 | ) | — | — | (19,424 | ) | ||||||||||||||||
Preferred stock accretion | (126 | ) | (139 | ) | (159 | ) | (351 | ) | — | (802 | ) | |||||||||||||
Net loss attributable to common stockholders | $ | (8,933 | ) | $ | (20,111 | ) | $ | (65,928 | ) | $ | (41,518 | ) | $ | (39,355 | ) | (184,415 | ) | |||||||
Net loss attributable to common stockholders per common share — basic and diluted | $ | (29.05 | ) | $ | (49.02 | ) | $ | (89.58 | ) | $ | (3.14 | ) | $ | (1.75 | ) | |||||||||
Weighted-average common shares outstanding — basic and diluted | 307,539 | 410,220 | 735,967 | 13,235,755 | 22,493,803 | |||||||||||||||||||
As of December 31, | ||||||||||||||||||||
2004 | 2005 | 2006 | 2007 | 2008 | ||||||||||||||||
Balance Sheet Data: | ||||||||||||||||||||
Cash and cash equivalents and marketable securities | $ | 4,336 | $ | 24,418 | $ | 54,699 | $ | 161,527 | $ | 121,124 | ||||||||||
Working capital | 3,569 | 22,267 | 44,814 | 147,247 | 110,209 | |||||||||||||||
Total assets | 5,073 | 28,670 | 59,645 | 167,097 | 128,773 | |||||||||||||||
Total liabilities | 1,346 | 4,031 | 13,071 | 63,800 | 57,730 | |||||||||||||||
Redeemable convertible preferred stock | 20,013 | 60,469 | 124,089 | — | — | |||||||||||||||
Deficit accumulated during the development stage | (17,351 | ) | (37,322 | ) | (83,667 | ) | (124,834 | ) | (164,189 | ) | ||||||||||
Total stockholders’ (deficiency) equity | $ | (16,287 | ) | $ | (35,830 | ) | $ | (77,515 | ) | $ | 103,297 | $ | 71,043 |
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• | 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 | |||||||||||||
Product Candidate | ||||||||||||||||
Third party direct project expenses | ||||||||||||||||
Amigal (Fabry Disease — Ended Phase 2) | $ | 3,361 | $ | 4,648 | $ | 4,410 | $ | 25,440 | ||||||||
Plicera (Gaucher Disease — Phase 2) | 9,905 | 4,378 | 2,796 | 18,904 | ||||||||||||
AT2220 (Pompe Disease — Phase 2) | 4,427 | 3,426 | 2,836 | 11,024 | ||||||||||||
Total third party direct project expenses | 17,693 | 12,452 | 10,042 | 55,368 | ||||||||||||
Other project costs(1) | ||||||||||||||||
Personnel costs | 8,187 | 9,720 | 14,535 | 38,966 | ||||||||||||
Other costs(2) | 7,750 | 8,902 | 13,187 | 33,308 | ||||||||||||
Total other project costs | 15,937 | 18,622 | 27,722 | 72,274 | ||||||||||||
Total research and development costs | $ | 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. |
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• | 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. |
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• | 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 | ||||||||||
Stock compensation expense recognized in: | ||||||||||||
Research and development expense | $ | 1.7 | $ | 1.6 | $ | 2.5 | ||||||
General and administrative expense | 1.6 | 2.4 | 3.9 | |||||||||
Total stock compensation expense | $ | 3.3 | $ | 4.0 | $ | 6.4 | ||||||
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Years Ended December 31, | ||||||||||||
2006 | 2007 | 2008 | ||||||||||
Expected stock price volatility | 74.8 | % | 78.3 | % | 78.2 | % | ||||||
Risk free interest rate | 4.7 | % | 4.5 | % | 3.0 | % | ||||||
Expected life of options (years) | 6.25 | 6.25 | 6.25 | |||||||||
Expected annual dividend per share | $ | 0.00 | $ | 0.00 | $ | 0.00 |
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Years Ended December 31, | ||||||||||||
2006 | 2007 | 2008 | ||||||||||
Historical | ||||||||||||
Numerator: | ||||||||||||
Net loss | $ | (46,345 | ) | $ | (41,167 | ) | $ | (39,355 | ) | |||
Deemed dividend | (19,424 | ) | — | — | ||||||||
Accretion of redeemable convertible preferred stock | (159 | ) | (351 | ) | — | |||||||
Net loss attributable to common stockholders | $ | (65,928 | ) | $ | (41,518 | ) | $ | (39,355 | ) | |||
Denominator: | ||||||||||||
Weighted average common shares outstanding — basic and diluted | 735,967 | 13,235,755 | 22,493,803 | |||||||||
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Approximate | ||||||||||||
Amount(1) | ||||||||||||
Funding(2) | Year | No. Shares | (in thousands) | |||||||||
Series A Redeemable Convertible Preferred Stock | 2002 | 444,443 | $ | 2,500 | ||||||||
Series B Redeemable Convertible Preferred Stock | 2004, 2005, 2006, 2007 | 4,917,853 | 31,189 | |||||||||
Series C Redeemable Convertible Preferred Stock | 2005, 2006 | 5,820,020 | 54,999 | |||||||||
Series D Redeemable Convertible Preferred Stock | 2006, 2007 | 4,930,405 | 60,000 | |||||||||
Common Stock | 2007 | 5,000,000 | 75,000 | |||||||||
Upfront License Fee from Shire | 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. |
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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. |
• | 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. |
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Less than | 1-3 | 3-5 | Over 5 | |||||||||||||||||
Total | 1 Year | Years | Years | Years | ||||||||||||||||
Operating lease obligations | $ | 6,162 | $ | 2,145 | $ | 4,017 | — | — | ||||||||||||
Capital lease obligations | 1,294 | 957 | 337 | — | — | |||||||||||||||
Employment agreement | 2,940 | 2,199 | 741 | — | — | |||||||||||||||
Total fixed contractual obligations (1) | $ | 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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• | 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/ John F. Crowley | /s/ James E. Dentzer |
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Amicus Therapeutics, Inc.
/s/ Ernst & Young LLP |
February 4, 2009
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(a development stage company)
Consolidated Balance Sheets
(in thousands, except share and per share amounts)
December 31, | ||||||||
2007 | 2008 | |||||||
Assets: | ||||||||
Current assets: | ||||||||
Cash and cash equivalents | $ | 44,188 | �� | $ | 28,073 | |||
Investments in marketable securities | 117,339 | 93,051 | ||||||
Prepaid expenses and other current assets | 1,513 | 2,463 | ||||||
Total current assets | 163,040 | 123,587 | ||||||
Property and equipment, less accumulated depreciation and amortization of $2,793 and $4,260 at December 31, 2007 and 2008, respectively | 3,790 | 4,919 | ||||||
Other non-current assets | 267 | 267 | ||||||
Total Assets | $ | 167,097 | $ | 128,773 | ||||
Liabilities and Stockholders’ Equity | ||||||||
Current liabilities: | ||||||||
Accounts payable and accrued expenses | $ | 10,465 | $ | 8,796 | ||||
Current portion of capital lease obligations | 1,527 | 877 | ||||||
Current portion of deferred revenue | 3,801 | 3,705 | ||||||
Total current liabilities | 15,793 | 13,378 | ||||||
Deferred revenue, less current portion | 46,813 | 44,035 | ||||||
Capital lease obligations, less current portion | 1,194 | 317 | ||||||
Commitments and contingencies | ||||||||
Stockholders’ equity: | ||||||||
Common stock, $.01 par value, 50,000,000 shares authorized, 22,408,731 shares issued and outstanding at December 31, 2007, 50,000,000 shares authorized, 22,634,711 shares issued and outstanding at December 31, 2008 | 285 | 287 | ||||||
Additional paid-in capital | 227,438 | 234,412 | ||||||
Accumulated other comprehensive income | 408 | 533 | ||||||
Deficit accumulated during the development stage | (124,834 | ) | (164,189 | ) | ||||
Total stockholders’ equity | 103,297 | 71,043 | ||||||
Total Liabilities and Stockholders’ Equity | $ | 167,097 | $ | 128,773 | ||||
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(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 | |||||||||||||
Revenue: | ||||||||||||||||
Research revenue | $ | — | $ | 1,375 | $ | 12,189 | $ | 13,564 | ||||||||
Collaboration revenue | — | 409 | 2,778 | 3,187 | ||||||||||||
Total revenue | — | 1,784 | 14,967 | 16,751 | ||||||||||||
Operating Expenses: | ||||||||||||||||
Research and development | 33,630 | 31,074 | 37,764 | 127,642 | ||||||||||||
General and administrative | 12,277 | 15,278 | 19,666 | 57,736 | ||||||||||||
Impairment of leasehold improvements | — | — | — | 1,030 | ||||||||||||
Depreciation and amortization | 952 | 1,237 | 1,493 | 4,287 | ||||||||||||
In-process research and development | — | — | — | 418 | ||||||||||||
Total operating expenses | 46,859 | 47,589 | 58,923 | 191,113 | ||||||||||||
Loss from operations | (46,859 | ) | (45,805 | ) | (43,956 | ) | (174,362 | ) | ||||||||
Other income (expenses): | ||||||||||||||||
Interest income | 1,990 | 5,135 | 4,819 | 12,760 | ||||||||||||
Interest expense | (273 | ) | (348 | ) | (218 | ) | (1,648 | ) | ||||||||
Change in fair value of warrant liability | (23 | ) | (149 | ) | — | (454 | ) | |||||||||
Other expense | (1,180 | ) | — | — | (1,180 | ) | ||||||||||
Loss before income tax benefit | (46,345 | ) | (41,167 | ) | (39,355 | ) | (164,884 | ) | ||||||||
Income tax benefit | — | — | — | 695 | ||||||||||||
Net loss | (46,345 | ) | (41,167 | ) | (39,355 | ) | (164,189 | ) | ||||||||
Deemed dividend | (19,424 | ) | — | — | (19,424 | ) | ||||||||||
Preferred stock accretion | (159 | ) | (351 | ) | — | (802 | ) | |||||||||
Net loss attributable to common stockholders | $ | (65,928 | ) | $ | (41,518 | ) | $ | (39,355 | ) | $ | (184,415 | ) | ||||
Net loss attributable to common stockholders per common share — basic and diluted | $ | (89.58 | ) | $ | (3.14 | ) | $ | (1.75 | ) | |||||||
Weighted-average common shares outstanding — basic and diluted | 735,967 | 13,235,755 | 22,493,803 | |||||||||||||
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(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 | ||||||||||||||||||||||
Balance at February 4, 2002 (inception) | — | $ | — | $ | — | $ | — | $ | — | $ | — | $ | — | |||||||||||||||
Issuance of common stock to a consultant | 74,938 | 6 | 78 | — | — | — | 84 | |||||||||||||||||||||
Stock issued for in-process research and development | 232,266 | 17 | 401 | — | — | — | 418 | |||||||||||||||||||||
Deferred compensation | — | — | 209 | — | (209 | ) | — | — | ||||||||||||||||||||
Amortization of deferred compensation | — | — | — | — | 27 | — | 27 | |||||||||||||||||||||
Issuance of warrants with financing arrangements | — | — | 8 | — | — | — | 8 | |||||||||||||||||||||
Accretion of redeemable convertible preferred stock | — | — | (11 | ) | — | — | — | (11 | ) | |||||||||||||||||||
Net loss | — | — | — | — | — | (1,775 | ) | (1,775 | ) | |||||||||||||||||||
Balance at December 31, 2002 | 307,204 | 23 | 685 | — | (182 | ) | (1,775 | ) | (1,249 | ) | ||||||||||||||||||
Stock issued from exercise of stock options | 333 | — | — | — | — | — | — | |||||||||||||||||||||
Deferred compensation | — | — | 14 | — | (14 | ) | — | — | ||||||||||||||||||||
Amortization of deferred compensation | — | — | — | — | 70 | — | 70 | |||||||||||||||||||||
Issuance of stock warrants with convertible notes | — | — | 210 | — | — | — | 210 | |||||||||||||||||||||
Issuance of stock options to consultants | — | — | 4 | — | — | — | 4 | |||||||||||||||||||||
Accretion of redeemable convertible preferred stock | — | — | (17 | ) | — | — | — | (17 | ) | |||||||||||||||||||
Beneficial conversion feature related to bridge financing | — | — | 41 | — | — | — | 41 | |||||||||||||||||||||
Net loss | — | — | — | — | — | (6,768 | ) | (6,768 | ) | |||||||||||||||||||
Balance at December 31, 2003 | 307,537 | 23 | 937 | — | (126 | ) | (8,543 | ) | (7,709 | ) | ||||||||||||||||||
Deferred compensation | — | — | 68 | — | (68 | ) | — | — | ||||||||||||||||||||
Amortization of deferred compensation | — | — | — | — | 60 | — | 60 | |||||||||||||||||||||
Issuance of stock options to consultants | — | — | 16 | — | — | — | 16 | |||||||||||||||||||||
Accretion of redeemable convertible preferred stock | — | — | (126 | ) | — | — | — | (126 | ) | |||||||||||||||||||
Interest waived on converted convertible notes | — | — | 193 | — | — | — | 193 | |||||||||||||||||||||
Beneficial conversion feature related to bridge financing | — | — | 95 | — | — | — | 95 | |||||||||||||||||||||
Comprehensive Loss: | ||||||||||||||||||||||||||||
Unrealized holding loss on available-for-sale securities | — | — | — | (9 | ) | — | — | (9 | ) | |||||||||||||||||||
Net loss | — | — | — | — | — | (8,807 | ) | (8,807 | ) | |||||||||||||||||||
Net total comprehensive loss | — | — | — | — | — | — | (8,816 | ) | ||||||||||||||||||||
Balance at December 31, 2004 | 307,537 | 23 | 1,183 | (9 | ) | (134 | ) | (17,350 | ) | (16,287 | ) | |||||||||||||||||
Stock issued from exercise of stock options | 97,156 | 7 | 17 | — | — | — | 24 | |||||||||||||||||||||
Stock issued from exercise of warrants | 133,332 | 10 | 65 | — | — | — | 75 | |||||||||||||||||||||
Deferred compensation | — | — | 2,778 | — | (2,778 | ) | — | — | ||||||||||||||||||||
Amortization of deferred compensation | — | — | — | — | 365 | — | 365 | |||||||||||||||||||||
Non-cash charge for stock options to consultants | — | — | 112 | — | — | — | 112 | |||||||||||||||||||||
Accretion of redeemable convertible preferred stock | — | — | (139 | ) | — | — | — | (139 | ) | |||||||||||||||||||
Comprehensive Loss: | ||||||||||||||||||||||||||||
Unrealized holding loss on available-for-sale securities | — | — | — | (7 | ) | — | — | (7 | ) | |||||||||||||||||||
Net loss | — | — | — | — | — | (19,972 | ) | (19,972 | ) | |||||||||||||||||||
Net total comprehensive loss | — | — | — | — | — | — | (19,979 | ) | ||||||||||||||||||||
Balance at December 31, 2005 | 538,025 | 40 | 4,016 | (16 | ) | (2,547 | ) | (37,322 | ) | (35,829 | ) |
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(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 | ||||||||||||||||||||||
Balance at December 31, 2005 | 538,025 | 40 | 4,016 | (16 | ) | (2,547 | ) | (37,322 | ) | (35,829 | ) | |||||||||||||||||
Stock issued from exercise of options | 265,801 | 20 | 138 | — | — | — | 158 | |||||||||||||||||||||
Stock issued for license payment | 133,333 | 10 | 1,210 | — | — | — | 1,220 | |||||||||||||||||||||
Reversal of deferred compensation upon adoption of FAS 123(R) | — | — | (2,547 | ) | — | 2,547 | — | — | ||||||||||||||||||||
Stock-based compensation | 53,333 | — | 2,816 | — | — | — | 2,816 | |||||||||||||||||||||
Issuance of stock options to consultants | — | — | 476 | — | — | — | 476 | |||||||||||||||||||||
Accretion of redeemable convertible preferred stock | — | — | (159 | ) | — | — | — | (159 | ) | |||||||||||||||||||
Reclassification of warrant liability upon exercise of Series B redeemable convertible preferred stock warrants | — | — | 117 | — | — | — | 117 | |||||||||||||||||||||
Beneficial conversion on issuance of Series C redeemable convertible preferred stock | — | — | 19,424 | — | — | — | 19,424 | |||||||||||||||||||||
Beneficial conversion charge (deemed dividend) on issuance of Series C redeemable convertible preferred stock | — | — | (19,424 | ) | — | — | — | (19,424 | ) | |||||||||||||||||||
Comprehensive (Loss)/ Income: | ||||||||||||||||||||||||||||
Unrealized holding gain on available-for-sale securities | — | — | — | 31 | — | — | 31 | |||||||||||||||||||||
Net loss | — | — | — | — | — | (46,345 | ) | (46,345 | ) | |||||||||||||||||||
Net total comprehensive loss | — | — | — | — | — | — | (46,314 | ) | ||||||||||||||||||||
Balance at December 31, 2006 | 990,492 | 70 | 6,067 | 15 | — | (83,667 | ) | (77,515 | ) | |||||||||||||||||||
Stock issued from initial public offering | 5,000,000 | 50 | 68,095 | — | — | — | 68,145 | |||||||||||||||||||||
Stock issued from conversion of preferred shares | 16,112,721 | 162 | 148,429 | — | — | — | 148,591 | |||||||||||||||||||||
Stock issued from exercise of stock options, net | 305,518 | 3 | 455 | — | — | — | 458 | |||||||||||||||||||||
Stock based compensation | — | — | 3,823 | — | — | — | 3,823 | |||||||||||||||||||||
Issuance of stock options to consultants | — | — | 162 | — | — | — | 162 | |||||||||||||||||||||
Accretion of redeemable convertible preferred stock | — | — | (351 | ) | — | — | — | (351 | ) | |||||||||||||||||||
Charge for warrant liability | — | — | 758 | — | — | — | 758 | |||||||||||||||||||||
Comprehensive (Loss)/ Income: | ||||||||||||||||||||||||||||
Unrealized holding gain on available-for-sale securities | — | — | — | 393 | — | — | 393 | |||||||||||||||||||||
Net loss | — | — | — | — | — | (41,167 | ) | (41,167 | ) | |||||||||||||||||||
Net total comprehensive loss | — | — | — | — | — | — | (40,774 | ) | ||||||||||||||||||||
Balance at December 31, 2007 | 22,408,731 | 285 | 227,438 | 408 | — | (124,834 | ) | 103,297 | ||||||||||||||||||||
Stock issued from exercise of stock options, net | 225,980 | 2 | 528 | — | — | — | 530 | |||||||||||||||||||||
Stock based compensation | — | — | 6,446 | — | — | — | 6,446 | |||||||||||||||||||||
Comprehensive (Loss)/ Income: | ||||||||||||||||||||||||||||
Unrealized holding gain on available-for-sale securities | — | — | — | 125 | — | — | 125 | |||||||||||||||||||||
Net loss | — | — | — | — | — | (39,355 | ) | (39,355 | ) | |||||||||||||||||||
Net total comprehensive loss | — | — | — | — | — | — | (39,230 | ) | ||||||||||||||||||||
Balance at December 31, 2008 | 22,634,711 | $ | 287 | $ | 234,412 | $ | 533 | $ | — | $ | (164,189 | ) | $ | 71,043 | ||||||||||||||
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Consolidated Statements of Cash Flows
(in thousands)
Period from | ||||||||||||||||
February 4, | ||||||||||||||||
2002 | ||||||||||||||||
(Inception) to | ||||||||||||||||
Years Ended December 31, | December 31, | |||||||||||||||
2006 | 2007 | 2008 | 2008 | |||||||||||||
Operating activities | ||||||||||||||||
Net loss | $ | (46,345 | ) | $ | (41,167 | ) | $ | (39,355 | ) | $ | (164,189 | ) | ||||
Adjustments to reconcile net loss to net cash (used in)/provided by operating activities: | ||||||||||||||||
Non-cash interest expense | — | — | — | 525 | ||||||||||||
Depreciation and amortization | 952 | 1,237 | 1,493 | 4,285 | ||||||||||||
Amortization of non-cash compensation | — | — | — | 522 | ||||||||||||
Stock-based compensation | 2,816 | 3,823 | 6,446 | 13,085 | ||||||||||||
Non-cash charge for stock based compensation issued to consultants | 475 | 162 | — | 853 | ||||||||||||
Change in fair value of warrant liability | 22 | 149 | — | 454 | ||||||||||||
Loss on disposal of asset | — | — | 44 | 44 | ||||||||||||
Stock-based license payment | 1,220 | — | — | 1,220 | ||||||||||||
Impairment of leasehold improvements | — | — | — | 1,030 | ||||||||||||
Non-cash charge for in process research and development | — | — | — | 418 | ||||||||||||
Beneficial conversion feature related to bridge financing | — | — | — | 135 | ||||||||||||
Changes in operating assets and liabilities: | ||||||||||||||||
Prepaid expenses and other current assets | 120 | (1,192 | ) | (949 | ) | (2,463 | ) | |||||||||
Other non-current assets | 265 | — | — | (288 | ) | |||||||||||
Account payable and accrued expenses | 6,586 | 1,566 | (1,669 | ) | 8,796 | |||||||||||
Deferred revenue | — | 50,614 | (2,873 | ) | 47,741 | |||||||||||
Net cash (used in)/provided by operating activities | (33,889 | ) | 15,192 | (36,863 | ) | (87,832 | ) | |||||||||
Investing activities | ||||||||||||||||
Sale and redemption of marketable securities | 37,441 | 126,370 | 178,100 | 347,166 | ||||||||||||
Purchases of marketable securities | (62,013 | ) | (200,743 | ) | (153,687 | ) | (439,801 | ) | ||||||||
Purchases of property and equipment | (2,031 | ) | (669 | ) | (2,667 | ) | (10,278 | ) | ||||||||
Net cash (used in)/provided by investing activities | (26,603 | ) | (75,042 | ) | 21,746 | (102,913 | ) | |||||||||
Financing activities | ||||||||||||||||
Proceeds from the issuance of preferred stock, net of issuance costs | 63,371 | 24,053 | — | 143,022 | ||||||||||||
Proceeds from issuance of common stock, net of issuance costs | — | 68,093 | — | 68,093 | ||||||||||||
Proceeds from the issuance of convertible notes | — | — | — | 5,000 | ||||||||||||
Payments of capital lease obligations | (881 | ) | (1,388 | ) | (1,528 | ) | (4,393 | ) | ||||||||
Payments from exercise of stock options | 158 | 510 | 530 | 1,221 | ||||||||||||
Proceeds from exercise of warrants (common and preferred) | 91 | 97 | — | 264 | ||||||||||||
Proceeds from capital asset financing arrangement | 3,431 | 546 | — | 5,611 | ||||||||||||
Net cash provided by/(used in) financing activities | 66,170 | 91,911 | (998 | ) | 218,818 | |||||||||||
Net increase/(decrease) in cash and cash equivalents | 5,678 | 32,061 | (16,115 | ) | 28,073 | |||||||||||
Cash and cash equivalents at beginning of year/ period | 6,449 | 12,127 | 44,188 | — | ||||||||||||
Cash and cash equivalents at end of year/period | $ | 12,127 | $ | 44,188 | $ | 28,073 | $ | 28,073 | ||||||||
Supplemental disclosures of cash flow information | ||||||||||||||||
Cash paid during the period for interest | $ | 273 | $ | 348 | $ | 218 | $ | 1,354 | ||||||||
Non-cash activities | ||||||||||||||||
Conversion of preferred stock to common stock | $ | — | $ | 148,591 | $ | — | 148,951 | |||||||||
Conversion of notes payable to Series B redeemable convertible preferred stock | $ | — | $ | — | $ | — | $ | 5,000 | ||||||||
Accretion of redeemable convertible preferred stock | $ | 159 | $ | 351 | $ | — | $ | 802 | ||||||||
Beneficial conversion feature related to issuance of the additional issuance of Series C redeemable convertible preferred stock | $ | 19,424 | $ | — | $ | — | $ | 19,424 |
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Notes To Consolidated Financial Statements
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Notes To Consolidated Financial Statements — (Continued)
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Notes To Consolidated Financial Statements — (Continued)
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Notes To Consolidated Financial Statements — (Continued)
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Notes To Consolidated Financial Statements — (Continued)
Years Ended December 31, | ||||||||||||
2006 | 2007 | 2008 | ||||||||||
Stock compensation expense recognized in: | ||||||||||||
Research and development expense | $ | 1.7 | $ | 1.6 | $ | 2.5 | ||||||
General and administrative expense | 1.6 | 2.4 | 3.9 | |||||||||
Total stock compensation expense | $ | 3.3 | $ | 4.0 | $ | 6.4 | ||||||
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Notes To Consolidated Financial Statements — (Continued)
Years Ended December 31, | ||||||||||||
2006 | 2007 | 2008 | ||||||||||
Historical | ||||||||||||
Numerator: | ||||||||||||
Net loss | $ | (46,345 | ) | $ | (41,167 | ) | $ | (39,355 | ) | |||
Deemed dividend | (19,424 | ) | — | — | ||||||||
Accretion of redeemable convertible preferred stock | (159 | ) | (351 | ) | — | |||||||
Net loss attributable to common stockholders | $ | (65,928 | ) | $ | (41,518 | ) | $ | (39,355 | ) | |||
Denominator: | ||||||||||||
Weighted average common shares outstanding — basic and diluted | 735,967 | 13,235,755 | 22,493,803 | |||||||||
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Notes To Consolidated Financial Statements — (Continued)
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Notes To Consolidated Financial Statements — (Continued)
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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) | |||||||||||||
Commercial paper | $ | 22,447 | $ | — | $ | 22,447 | $ | — | ||||||||
Asset-backed securities | 7,217 | — | 7,217 | — | ||||||||||||
U.S. government agency securities | 58,790 | — | 58,790 | — | ||||||||||||
Corporate debt securities | 4,597 | — | 4,597 | — | ||||||||||||
Money market fund (cash equivalents) | 24,626 | 24,626 | — | |||||||||||||
$ | 117,677 | $ | 24,626 | $ | 93,051 | $ | — | |||||||||
December 31, | ||||||||
2007 | 2008 | |||||||
Property and equipment consist of the following: | ||||||||
Computer equipment | $ | 694 | $ | 1,322 | ||||
Computer software | 167 | 996 | ||||||
Research equipment | 3,089 | 4,096 | ||||||
Furniture and fixtures | 579 | 657 | ||||||
Leasehold improvements | 2,054 | 2,108 | ||||||
6,583 | 9,179 | |||||||
Less accumulated depreciation and amortization | (2,793 | ) | (4,260 | ) | ||||
$ | 3,790 | $ | 4,919 | |||||
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Notes To Consolidated Financial Statements — (Continued)
December 31, | ||||||||
2007 | 2008 | |||||||
Accounts payable | $ | 530 | $ | 1,987 | ||||
Accrued professional fees | 979 | 283 | ||||||
Accrued contract manufacturing & contract research costs | 6,035 | 2,985 | ||||||
Accrued compensation and benefits | 2,279 | 2,846 | ||||||
Accrued facility costs | 364 | 281 | ||||||
Accrued other | 278 | 414 | ||||||
$ | 10,465 | $ | 8,796 | |||||
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(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) | |||||||||||||||||||||||||||||
Balance at February 4, 2002 (inception) | — | $ | — | — | $ | — | — | $ | — | — | $ | — | ||||||||||||||||||||
Issuance of Series A at $5.63 per share | 444,443 | 2,500 | — | — | — | — | — | — | ||||||||||||||||||||||||
Issuance costs | — | (95 | ) | — | — | — | — | — | — | |||||||||||||||||||||||
Accretion | — | 10 | — | — | — | — | — | — | ||||||||||||||||||||||||
Balance at December 31, 2002 | 444,443 | 2,415 | — | — | — | — | — | — | ||||||||||||||||||||||||
Accretion | — | 17 | — | — | — | — | — | — | ||||||||||||||||||||||||
Balance at December 31, 2003 | 444,443 | 2,432 | — | — | — | — | — | — | ||||||||||||||||||||||||
Issuance of Series B at $6.38 per share | — | — | 2,823,523 | 18,000 | — | — | — | — | ||||||||||||||||||||||||
Issuance cost | — | — | — | (122 | ) | — | — | — | — | |||||||||||||||||||||||
Issuance of warrants with Series B | — | — | — | (422 | ) | — | — | — | — | |||||||||||||||||||||||
Accretion | — | 17 | — | 109 | — | — | — | — | ||||||||||||||||||||||||
Balance at December 31, 2004 | 444,443 | 2,449 | 2,823,523 | 17,565 | — | — | — | — | ||||||||||||||||||||||||
Issuance of Series B at $6.38 per share | — | — | 2,039,211 | 13,000 | — | — | — | — | ||||||||||||||||||||||||
Issuance cost | — | — | — | (6 | ) | — | — | — | — | |||||||||||||||||||||||
Issuance of Series C at $9.45 per share | — | — | — | — | 2,910,010 | 27,500 | — | — | ||||||||||||||||||||||||
Issuance cost | — | — | — | — | — | (178 | ) | — | — | |||||||||||||||||||||||
Accretion | — | 17 | — | 110 | — | 12 | — | — | ||||||||||||||||||||||||
Balance at December 31, 2005 | 444,443 | 2,466 | 4,862,734 | 30,669 | 2,910,010 | 27,334 | — | — |
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(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) | |||||||||||||||||||||||||||||
Balance at December 31, 2005 | 444,443 | 2,466 | 4,862,734 | 30,669 | 2,910,010 | 27,334 | — | — | ||||||||||||||||||||||||
Exercise of warrants with Series B at $6.38 | — | — | 14,322 | 91 | — | — | — | — | ||||||||||||||||||||||||
Issuance of Series C at $9.45 per share | — | — | — | — | 2,910,010 | 27,500 | — | — | ||||||||||||||||||||||||
Issuance of Series D at $12.15 per share | — | — | — | — | — | — | 2,953,878 | 35,947 | ||||||||||||||||||||||||
Issuance cost | — | — | — | — | — | — | — | (76 | ) | |||||||||||||||||||||||
Accretion to redemption value | — | 10 | — | 108 | — | 35 | — | 5 | ||||||||||||||||||||||||
Balance at December 31, 2006 | 444,443 | 2,476 | 4,877,056 | 30,868 | 5,820,020 | 54,869 | 2,953,878 | 35,876 | ||||||||||||||||||||||||
Issuance of Series D at $12.15 per share | — | — | — | — | — | — | 1,976,527 | 24,053 | ||||||||||||||||||||||||
Series B warrant exercise | — | — | 40,797 | 98 | — | — | — | — | ||||||||||||||||||||||||
Accretion to redemption value | — | 24 | — | 126 | — | 130 | — | 71 | ||||||||||||||||||||||||
Conversion of preferred stock to common | (444,443 | ) | (2,500 | ) | (4,917,853 | ) | (31,092 | ) | (5,820,020 | ) | (54,999 | ) | (4,930,405 | ) | (60,000 | ) | ||||||||||||||||
Balance at December 31, 2007 | — | $ | — | — | $ | — | — | $ | — | — | $ | — | ||||||||||||||||||||
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(a development stage company)
Notes To Consolidated Financial Statements — (Continued)
Years Ended December 31, | ||||||||||||
2006 | 2007 | 2008 | ||||||||||
Stock compensation expense recognized in: | ||||||||||||
Research and development expense | $ | 1.7 | $ | 1.6 | $ | 2.5 | ||||||
General and administrative expense | 1.6 | 2.4 | 3.9 | |||||||||
Total stock compensation expense | $ | 3.3 | $ | 4.0 | $ | 6.4 | ||||||
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Notes To Consolidated Financial Statements — (Continued)
Years Ended December 31, | ||||||||||||
2006 | 2007 | 2008 | ||||||||||
Expected stock price volatility | 74.8 | % | 78.3 | % | 78.2 | % | ||||||
Risk free interest rate | 4.7 | % | 4.5 | % | 3.0 | % | ||||||
Expected life of options (years) | 6.25 | 6.25 | 6.25 | |||||||||
Expected annual dividend per share | $ | 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) | |||||||||||||||
Options outstanding, December 31, 2005 | 1,237.1 | $ | 2.10 | |||||||||||||
Granted | 1,005.1 | $ | 6.00 | |||||||||||||
Exercised | (265.8 | ) | $ | 0.60 | ||||||||||||
Forfeited | (108.0 | ) | $ | 2.20 | ||||||||||||
Options outstanding, December 31, 2006 | 1,868.4 | $ | 4.27 | |||||||||||||
Granted | 1,035.6 | $ | 13.16 | |||||||||||||
Exercised | (308.6 | ) | $ | 1.80 | ||||||||||||
Forfeited | (152.2 | ) | $ | 8.94 | ||||||||||||
Options outstanding, December 31, 2007 | 2,443.2 | $ | 8.08 | |||||||||||||
Granted | 965.2 | $ | 10.49 | |||||||||||||
Exercised | (225.1 | ) | $ | 2.48 | ||||||||||||
Forfeited | (106.0 | ) | $ | 9.69 | ||||||||||||
Options outstanding, December 31, 2008 | 3,077.3 | $ | 9.19 | 7.9 years | $ | 3.5 | ||||||||||
Vested and unvested expected to vest, December 31, 2008 | 2,914.8 | $ | 9.09 | 7.9 years | $ | 3.5 | ||||||||||
Exercisable at December 31, 2008 | 1,266.3 | $ | 7.36 | 7.0 years | $ | 2.8 |
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Notes To Consolidated Financial Statements — (Continued)
Restricted Stock | ||||||||
Weighted | ||||||||
Number of | Average Grant | |||||||
Shares | Date Fair Value | |||||||
(in thousands) | ||||||||
Unvested at December 31, 2006 | 51.1 | $ | 8.94 | |||||
Granted | — | $ | — | |||||
Vested | (16.1 | ) | $ | 8.88 | ||||
Forfeited | — | $ | — | |||||
Unvested at December 31, 2007 | 35.0 | $ | 8.96 | |||||
Granted | — | $ | — | |||||
Vested | (14.4 | ) | $ | 8.85 | ||||
Forfeited | — | $ | — | |||||
Unvested at December 31, 2008 | 20.6 | $ | 9.04 | |||||
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Notes To Consolidated Financial Statements — (Continued)
Operating Leases | ||||
Years ending December 31: | ||||
2009 | $ | 2,145 | ||
2010 | 1,922 | |||
2011 | 1,839 | |||
2012 | 256 | |||
2013 | — | |||
$ | 6,162 | |||
Capital Leases | ||||
Years ending December 31: | ||||
2009 | $ | 957 | ||
2010 | 295 | |||
2011 | 42 | |||
2012 | — | |||
1,294 | ||||
Less payments for interest | (100 | ) | ||
Total principal obligation | 1,194 | |||
Less short-term portion | (877 | ) | ||
Long-term portion | $ | 317 | ||
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Notes To Consolidated Financial Statements — (Continued)
For Years Ended December 31, | ||||||||||||
2006 | 2007 | 2008 | ||||||||||
Current deferred tax asset | ||||||||||||
Non-cash stock issue | $ | 246 | $ | 283 | $ | 1,560 | ||||||
Others | 1,309 | 1,232 | 141 | |||||||||
1,555 | 1,515 | 1,701 | ||||||||||
Non-current deferred tax assets | ||||||||||||
Amortization/depreciation | 1,289 | 1,129 | 2,682 | |||||||||
Research tax credit | 3,611 | 5,403 | 7,294 | |||||||||
Net operating loss carry forwards | 27,257 | 42,282 | 36,196 | |||||||||
Deferred revenue | — | — | 19,096 | |||||||||
Others | 121 | 478 | 518 | |||||||||
Total deferred tax asset | 33,833 | 50,807 | 67,487 | |||||||||
Non-current deferred tax liability | — | — | — | |||||||||
Total net deferred tax asset | 33,833 | 50,807 | 67,487 | |||||||||
Less valuation allowance | (33,833 | ) | (50,807 | ) | (67,487 | ) | ||||||
Net deferred tax asset | $ | — | $ | — | $ | — | ||||||
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Notes To Consolidated Financial Statements — (Continued)
Years Ended December 31, | ||||||||||||
2006 | 2007 | 2008 | ||||||||||
Statutory rate | (34 | )% | (34 | )% | (34 | )% | ||||||
State taxes, net of federal benefit | (6 | ) | (5 | ) | (5 | ) | ||||||
Permanent adjustments | 1 | 3 | 2 | |||||||||
R&D credit | (4 | ) | (4 | ) | (5 | ) | ||||||
Other | 2 | (1 | ) | — | ||||||||
Valuation allowance | 41 | 41 | 42 | |||||||||
Net | 0 | % | 0 | % | 0 | % | ||||||
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Notes To Consolidated Financial Statements — (Continued)
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(a development stage company)
Notes To Consolidated Financial Statements — (Continued)
Quarters Ended | ||||||||||||||||
March 31 | June 30 | September 30 | December 31 | |||||||||||||
2007 | ||||||||||||||||
Net loss | $ | (9,695 | ) | $ | (9,396 | ) | $ | (10,303 | ) | $ | (11,773 | ) | ||||
Net loss attributable to common stockholders | (9,736 | ) | (9,706 | ) | (10,303 | ) | (11,773 | ) | ||||||||
Basic and diluted net loss per common share (1) | (10.21 | ) | (1.37 | ) | (0.46 | ) | (0.53 | ) | ||||||||
2008 | ||||||||||||||||
Net loss | (7,731 | ) | (9,294 | ) | (8,180 | ) | (14,150 | ) | ||||||||
Net loss attributable to common stockholders | (7,731 | ) | (9,294 | ) | (8,180 | ) | (14,150 | ) | ||||||||
Basic and diluted net loss per common share (1) | (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 Company’s issuing shares of its common stock during the year. |
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Item 12. SECURITY OWNERSHIP OF CERTAIN BENEFICIAL OWNERS AND MANAGEMENT AND RELATED STOCKHOLDER MATTERS. |
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(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 | Restated Certificate of Incorporation of the Registrant. | S-1 (333-141700) | 5/17/07 | 3.2 | ||||||||||
3.2 | Restated By-laws of the Registrant. | S-1/A (333-141700) | 4/27/07 | 3.4 | ||||||||||
4.1 | Specimen Stock Certificate evidencing shares of common stock | S-1 (333-141700) | 3/30/07 | 4.1 | ||||||||||
4.2 | Third Amended and Restated Investor Rights Agreement, dated as of September 13, 2006, as amended | S-1 (333-141700) | 3/30/07 | 4.3 | ||||||||||
10.1 | 2002 Equity Incentive Plan, as amended, and forms of option agreements thereunder | S-1/A (333-141700) | 4/27/07 | 10.1 | ||||||||||
10.2 | 2007 Equity Incentive Plan and forms of option agreements | S-1/A (333-141700) | 5/17/07 | 10.2 | ||||||||||
++10.3 | Amended and Restated License Agreement, dated October, 31, 2008, by and between the Registrant and Mount Sinai School of Medicine of New York University | X | ||||||||||||
+10.4 | License Agreement, dated as of June 26, 2003, by and between the Registrant and University of Maryland, Baltimore County, as amended | S-1 (333-141700) | 3/30/07 | 10.4 | ||||||||||
+10.5 | Exclusive License Agreement, dated as of June 8, 2005, by and between the Registrant and Novo Nordisk, A/S | S-1 (333-141700) | 3/30/07 | 10.5 | ||||||||||
10.6 | Sublease Agreement, dated as of May 12, 2005, by and between the Registrant and Purdue Pharma, L.P. | S-1 (333-141700) | 3/30/07 | 10.6 | ||||||||||
10.7 | Amended and Restated Employment Agreement, dated as of December 30, 2008, by and between the Registrant and John F. Crowley | Form 8-K Current Report | 12/31/08 | 10.1 | ||||||||||
10.8 | Letter Agreement, dated as of November 9, 2004, by and between the Registrant and Matthew R. Patterson | S-1 (333-141700) | 3/30/07 | 10.8 | ||||||||||
10.9 | Letter Agreement, dated as of July 27, 2006, by and between the Registrant and James E. Dentzer | S-1 (333-141700) | 3/30/07 | 10.9 | ||||||||||
10.10 | Letter Agreement, dated as of December 19, 2005, by and between the Registrant and David Lockhart, Ph.D. | S-1 (333-141700) | 3/30/07 | 10.10 | ||||||||||
10.11 | Consulting Agreement, dated as of February 28, 2006, by and between the Registrant and Donald J. Hayden, Jr. | S-1 (333-141700) | 3/30/07 | 10.15 | ||||||||||
10.12 | Form of Director and Officer Indemnification Agreement | S-1 (333-141700) | 3/30/07 | 10.17 | ||||||||||
10.13 | Employment Agreement, dated as of September 11, 2006, by and between the Registrant and Donald J. Hayden, Jr. | S-1/A (333-141700) | 4/27/07 | 10.19 | ||||||||||
10.14 | Restricted Stock Agreement, dated as of March 8, 2007, by and between the Registrant and James E. Dentzer | S-1/A (333-141700) | 4/27/07 | 10.20 | ||||||||||
10.15 | Restricted Stock Agreement, dated as of March 8, 2007, by and between the Registrant and Glenn P. Sblendorio | S-1/A (333-141700) | 4/27/07 | 10.21 | ||||||||||
10.16 | Lease Agreement, dated as of July 31, 2006, by and between the Registrant and Cedar Brook II Corporate Center, L.P. | S-1/A (333-141700) | 4/27/07 | 10.22 |
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Exhibit | Incorporated by Reference to SEC Filing | Filed with this | ||||||||||||
No. | Filed Exhibit Description | Form | Date | Exhibit No. | Form 10-K | |||||||||
10.17 | 2007 Director Option Plan and form of option agreement | S-1/A (333-141700) | 5/17/07 | 10.23 | ||||||||||
10.18 | 2007 Employee Stock Purchase Plan | S-1/A (333-141700) | 5/17/07 | 10.24 | ||||||||||
10.19 | Amicus Therapeutics, Inc. 2007 Amended and Restated Equity Incentive Plan | Form 8-K Current Report | 6/12/08 | 10.1 | ||||||||||
10.20 | Lease Agreement dated as of September 11, 2008 by and between the Registrant and A/G Touchstone, TP, LLC. | Form 8-K Current Report | 9/15/08 | 10.1 | ||||||||||
+10.21 | License and Collaboration Agreement, dated as of November 7, 2007, by and between the Registrant and Shire Pharmaceuticals Ireland, Ltd. | Form 10-K Annual Report | 2/08/08 | 10.20 | ||||||||||
10.22 | Letter Agreement, dated as of December 30, 2008, by and between the Registrant and David Lockhart, Ph.D. | Form 8-K Current Report | 12/31/08 | 10.4 | ||||||||||
10.23 | Letter Agreement, dated as of December 30, 2008, by and between the Registrant and James E. Dentzer | Form 8-K Current Report | 12/31/08 | 10.2 | ||||||||||
10.24 | Letter Agreement, dated as of December 30, 2008, by and between the Registrant and Matthew R. Patterson | Form 8-K Current Report | 12/31/08 | 10.3 | ||||||||||
10.25 | Letter Agreement, dated as of December 30, 2008, by and between the Registrant and David Palling, Ph.D. | X | ||||||||||||
10.26 | Letter Agreement, dated as of December 30, 2008, by and between the Registrant and Bradley L. Campbell | X | ||||||||||||
10.27 | Letter Agreement, dated as of December 30, 2008, by and between the Registrant and Gregory P. Licholai, M.D. | Form 8-K Current Report | 12/31/08 | 10.5 | ||||||||||
10.28 | Letter Agreement, dated as of December 30, 2008, by and between the Registrant and S. Nicole Schaeffer | X | ||||||||||||
10.29 | Letter Agreement, dated as of December 30, 2008, by and between the Registrant and John R. Kirk | X | ||||||||||||
10.30 | Letter Agreement, dated as of December 30, 2008, by and between the Registrant and Andrew Shenker, M.D., Ph.D. | X | ||||||||||||
10.31 | Letter Agreement, dated as of December 30, 2008, by and between the Registrant and Geoffrey P. Gilmore | X | ||||||||||||
23.1 | Consent of Independent Registered Public Accounting Firm. | X | ||||||||||||
31.1 | Certification of Principal Executive Officer Pursuant to Rule 13a-14(a) of the Securities Exchange Act of 1934. | X | ||||||||||||
31.2 | Certification of Principal Financial Officer Pursuant to Rule 13a-14(a) of the Securities Exchange Act of 1934. | X | ||||||||||||
32.1 | Certificate of Principal Executive Officer pursuant to 18 U.S.C. Section 1350 and Section 906 of the Sarbanes-Oxley Act of 2002. | X | ||||||||||||
32.2 | Certificate of Principal Financial Officer pursuant to 18 U.S.C. Section 1350 and Section 906 of the Sarbanes-Oxley Act of 2002. | 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. |
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Table of Contents
AMICUS THERAPEUTICS, INC. (Registrant) | ||||
By: | /s/ John F. Crowley | |||
John F. Crowley | ||||
Chief Executive Officer |
Signature | Title | Date | ||
/s/ John F. Crowley | Chief Executive Officer (Principal Executive Officer) | February 4, 2009 | ||
/s/ James E. Dentzer | Chief Financial Officer (Principal Financial and Accounting Officer) | February 4, 2009 | ||
/s/ Donald J. Hayden | Chairman of the Board | February 4, 2009 | ||
/s/ Alexander E. Barkas, Ph.D. | Director | February 4, 2009 | ||
/s/ Stephen Bloch, M.D. | Director | February 4, 2009 | ||
/s/ P. Sherrill Neff | Director | February 4, 2009 | ||
/s/ Michael G. Raab | Director | February 4, 2009 | ||
/s/ Glenn Sblendorio | Director | February 4, 2009 | ||
/s/ James N. Topper, M.D., Ph.D. | Director | February 4, 2009 | ||
/s/ Sol J. Barer, Ph.D. | Director | February 4, 2009 |
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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 | Restated Certificate of Incorporation of the Registrant. | S-1 (333-141700) | 5/17/07 | 3.2 | ||||||||||
3.2 | Restated By-laws of the Registrant. | S-1/A (333-141700) | 4/27/07 | 3.4 | ||||||||||
4.1 | Specimen Stock Certificate evidencing shares of common stock | S-1 (333-141700) | 3/30/07 | 4.1 | ||||||||||
4.2 | Third Amended and Restated Investor Rights Agreement, dated as of September 13, 2006, as amended | S-1 (333-141700) | 3/30/07 | 4.3 | ||||||||||
10.1 | 2002 Equity Incentive Plan, as amended, and forms of option agreements thereunder | S-1/A (333-141700) | 4/27/07 | 10.1 | ||||||||||
10.2 | 2007 Equity Incentive Plan and forms of option agreements | S-1/A (333-141700) | 5/17/07 | 10.2 | ||||||||||
++10.3 | Amended and Restated License Agreement, dated October, 31, 2008, by and between the Registrant and Mount Sinai School of Medicine of New York University | X | ||||||||||||
+10.4 | License Agreement, dated as of June 26, 2003, by and between the Registrant and University of Maryland, Baltimore County, as amended | S-1 (333-141700) | 3/30/07 | 10.4 | ||||||||||
+10.5 | Exclusive License Agreement, dated as of June 8, 2005, by and between the Registrant and Novo Nordisk, A/S | S-1 (333-141700) | 3/30/07 | 10.5 | ||||||||||
10.6 | Sublease Agreement, dated as of May 12, 2005, by and between the Registrant and Purdue Pharma, L.P. | S-1 (333-141700) | 3/30/07 | 10.6 | ||||||||||
10.7 | Amended and Restated Employment Agreement, dated as of December 30, 2008, by and between the Registrant and John F. Crowley | Form 8-K Current Report | 12/31/08 | 10.1 | ||||||||||
10.8 | Letter Agreement, dated as of November 9, 2004, by and between the Registrant and Matthew R. Patterson | S-1 (333-141700) | 3/30/07 | 10.8 | ||||||||||
10.9 | Letter Agreement, dated as of July 27, 2006, by and between the Registrant and James E. Dentzer | S-1 (333-141700) | 3/30/07 | 10.9 | ||||||||||
10.10 | Letter Agreement, dated as of December 19, 2005, by and between the Registrant and David Lockhart, Ph.D. | S-1 (333-141700) | 3/30/07 | 10.10 | ||||||||||
10.11 | Consulting Agreement, dated as of February 28, 2006, by and between the Registrant and Donald J. Hayden, Jr. | S-1 (333-141700) | 3/30/07 | 10.15 | ||||||||||
10.12 | Form of Director and Officer Indemnification Agreement | S-1 (333-141700) | 3/30/07 | 10.17 | ||||||||||
10.13 | Employment Agreement, dated as of September 11, 2006, by and between the Registrant and Donald J. Hayden, Jr. | S-1/A (333-141700) | 4/27/07 | 10.19 | ||||||||||
10.14 | Restricted Stock Agreement, dated as of March 8, 2007, by and between the Registrant and James E. Dentzer | S-1/A (333-141700) | 4/27/07 | 10.20 | ||||||||||
10.15 | Restricted Stock Agreement, dated as of March 8, 2007, by and between the Registrant and Glenn P. Sblendorio | S-1/A (333-141700) | 4/27/07 | 10.21 | ||||||||||
10.16 | Lease Agreement, dated as of July 31, 2006, by and between the Registrant and Cedar Brook II Corporate Center, L.P. | S-1/A (333-141700) | 4/27/07 | 10.22 | ||||||||||
10.17 | 2007 Director Option Plan and form of option agreement | S-1/A (333-141700) | 5/17/07 | 10.23 | ||||||||||
10.18 | 2007 Employee Stock Purchase Plan | S-1/A (333-141700) | 5/17/07 | 10.24 | ||||||||||
10.19 | Amicus Therapeutics, Inc. 2007 Amended and Restated Equity Incentive Plan | Form 8-K Current Report | 6/12/08 | 10.1 | ||||||||||
10.20 | Lease Agreement dated as of September 11, 2008 by and between the Registrant and A/G Touchstone, TP, LLC. | Form 8-K Current Report | 9/15/08 | 10.1 | ||||||||||
+10.21 | License and Collaboration Agreement, dated as of November 7, 2007, by and between the Registrant and Shire Pharmaceuticals Ireland, Ltd. | Form 10-K Annual Report | 2/08/08 | 10.20 | ||||||||||
10.22 | Letter Agreement, dated as of December 30, 2008, by and between the Registrant and David Lockhart, Ph.D. | Form 8-K Current Report | 12/31/08 | 10.4 | ||||||||||
10.23 | Letter Agreement, dated as of December 30, 2008, by and between the Registrant and James E. Dentzer | Form 8-K Current Report | 12/31/08 | 10.2 |
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Exhibit | Incorporated by Reference to SEC Filing | Filed with this | ||||||||||||
No. | Filed Exhibit Description | Form | Date | Exhibit No. | Form 10-K | |||||||||
10.24 | Letter Agreement, dated as of December 30, 2008, by and between the Registrant and Matthew R. Patterson | Form 8-K Current Report | 12/31/08 | 10.3 | ||||||||||
10.25 | Letter Agreement, dated as of December 30, 2008, by and between the Registrant and David Palling, Ph.D. | X | ||||||||||||
10.26 | Letter Agreement, dated as of December 30, 2008, by and between the Registrant and Bradley L. Campbell | X | ||||||||||||
10.27 | Letter Agreement, dated as of December 30, 2008, by and between the Registrant and Gregory P. Licholai, M.D. | Form 8-K Current Report | 12/31/08 | 10.5 | ||||||||||
10.28 | Letter Agreement, dated as of December 30, 2008, by and between the Registrant and S. Nicole Schaeffer | X | ||||||||||||
10.29 | Letter Agreement, dated as of December 30, 2008, by and between the Registrant and John R. Kirk | X | ||||||||||||
10.30 | Letter Agreement, dated as of December 30, 2008, by and between the Registrant and Andrew Shenker, M.D., Ph.D. | X | ||||||||||||
10.31 | Letter Agreement, dated as of December 30, 2008, by and between the Registrant and Geoffrey P. Gilmore | X | ||||||||||||
23.1 | Consent of Independent Registered Public Accounting Firm. | X | ||||||||||||
31.1 | Certification of Principal Executive Officer Pursuant to Rule 13a-14(a) of the Securities Exchange Act of 1934. | X | ||||||||||||
31.2 | Certification of Principal Financial Officer Pursuant to Rule 13a-14(a) of the Securities Exchange Act of 1934. | X | ||||||||||||
32.1 | Certificate of Principal Executive Officer pursuant to 18 U.S.C. Section 1350 and Section 906 of the Sarbanes-Oxley Act of 2002. | X | ||||||||||||
32.2 | Certificate of Principal Financial Officer pursuant to 18 U.S.C. Section 1350 and Section 906 of the Sarbanes-Oxley Act of 2002. | 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. |
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