We could not find any results for:
Make sure your spelling is correct or try broadening your search.
Share Name | Share Symbol | Market | Type |
---|---|---|---|
Amicus Therapeutics Inc | NASDAQ:FOLD | NASDAQ | Common Stock |
Price Change | % Change | Share Price | Bid Price | Offer Price | High Price | Low Price | Open Price | Shares Traded | Last Trade | |
---|---|---|---|---|---|---|---|---|---|---|
-0.12 | -1.15% | 10.30 | 10.19 | 11.99 | 10.79 | 10.36 | 10.74 | 1,360,422 | 05:00:01 |
|
ý
|
ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934
|
o
|
TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934
|
Delaware
(State or Other Jurisdiction of
Incorporation or Organization)
|
|
71-0869350
(IRS Employer
Identification Number)
|
1 Cedar Brook Drive, Cranbury, NJ
(Address of Principal Executive Offices)
|
|
08512
(Zip Code)
|
(609) 662-2000
(Registrant's Telephone Number, Including Area Code)
|
Title of each class
|
Name of each exchange on which registered
|
Common Stock, par value $0.01 per share
|
The NASDAQ Stock Market LLC
|
Large accelerated filer
ý
|
|
Accelerated filer
o
|
Non-accelerated filer
o
|
|
Smaller reporting company
o
|
|
|
Emerging growth company
o
|
|
•
|
the progress and results of our preclinical and clinical trials of our drug candidates;
|
•
|
the cost of manufacturing drug supply for our clinical and preclinical studies, including the cost of manufacturing Pompe Enzyme Replacement Therapy (“ERT”) and gene therapies;
|
•
|
the scope, progress, results and costs of preclinical development, laboratory testing and clinical trials for our product candidates including those testing the use of pharmacological chaperones co-formulated and co-administered with ERT and for the treatment of lysosomal storage disorders and gene therapies for the treatment of rare genetic metabolic diseases;
|
•
|
the future results of on-going preclinical research and subsequent clinical trials for cyclin-dependent kinase-like 5 (“CDKL5”) deficiency, including our ability to obtain regulatory approvals and commercialize CDKL5 therapies and obtain market acceptance for such therapies;
|
•
|
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;
|
•
|
our ability to successfully commercialize Galafold
®
(“migalastat HCl”);
|
•
|
our ability to manufacture or supply sufficient clinical or commercial products;
|
•
|
our ability to obtain reimbursement for Galafold
®
;
|
•
|
our ability to satisfy post-marketing commitments or requirements for continued regulatory approval of Galafold
®
;
|
•
|
our ability to obtain market acceptance of Galafold
®
;
|
•
|
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;
|
•
|
our ability to successfully integrate our acquired products and technologies into our business, including the possibility that the expected benefits of the transactions will not be fully realized by us or may take longer to realize than expected;
|
•
|
our ability to establish collaborations and obtain milestone, royalty or other payments from any such collaborators;
|
•
|
our ability to adjust to changes in European and United Kingdom markets as the United Kingdom leaves the European Union; and
|
•
|
fluctuations in foreign currency exchange rates; and changes in accounting standards.
|
•
|
Commercial and regulatory success in Fabry disease
. During the year ended
December 31, 2018
, Galafold
®
revenue totaled approximately
$91.2 million
. Revenue has been generated primarily in the EU since May of 2016. In 2018, we received approvals for Galafold
®
in the U.S. and Japan.
|
•
|
Pompe clinical program milestones
. We reported a series of positive data from a Phase 1/2 clinical study to evaluate Pompe disease patients treated with our novel treatment paradigm AT-GAA for up to 18 months. We also initiated a global pivotal study of AT-GAA (ATB200-03, also known as PROPEL) which is expected to enroll approximately 100 participants with late-onset Pompe disease at up to 90 global sites.
|
•
|
Pipeline Growth:
With 14 new gene therapy programs for LSDs, we have established a leading portfolio of medicines for people living with rare metabolic disorders. Through our license with NCH, we acquired worldwide development and commercial rights for ten gene therapy programs in rare, neurologic LSDs with lead programs in CLN6, CLN3, and CLN8 Batten disease. An additional four programs were added to the pipeline through the collaboration
with Penn to pursue research and development of novel gene therapies for Pompe disease, Fabry disease, CDKL5 deficiency disorder (“CDD”) and one additional undisclosed rare metabolic disorder.
|
•
|
Manufacturing
. We successfully scaled up manufacturing of our Pompe biologic to commercial scale (1,000L) for our pivotal PROPEL study and commercial supply. Our supply agreement with WuXi Biologics and current capacity are expected to produce sufficient quantities to serve the entire Pompe population as quickly as possible after receipt of applicable regulatory approvals. Through our collaborations with NCH and Penn, we also gain access to their preclinical manufacturing capabilities, clinical supply and CMO relationships for those gene therapy programs.
|
•
|
Financial strength.
Total cash, cash equivalents and marketable securities of
$504.2 million
at
December 31, 2018
compared to
$358.6 million
at
December 31, 2017
. The current cash position, including expected Galafold
®
revenues, is sufficient to fund ongoing Fabry, Pompe and gene therapy program operations into at least mid-2021. Potential future business development collaborations, pipeline expansion, and investment in manufacturing capabilities could impact our future capital requirements.
|
•
|
We have an exclusive license to a number of issued U.S. patents that cover the use of Galafold
®
to treat Fabry disease, as well as corresponding European, Japanese, and Canadian patents. These exclusively licensed U.S. patents relating to Galafold
®
expired in 2018, while the European, Japanese, and Canadian patents will expire in 2019. The patents include claims covering methods of increasing the activity of and preventing the degradation of alpha-Gal A, and methods for the treatment of Fabry disease using Galafold
®
. In addition, we own two issued U.S. patents directed to dosing regimens with Galafold
®
that expire in 2027 (not including any extensions), as well as a pending application which, if granted, may result in a patent that also expires in 2027 (not including any extensions). Foreign counterpart patents are issued in Australia, Europe, Hong Kong, Mexico, and Japan, and foreign applications are pending in Australia, Canada, Europe, Hong Kong, Japan, and Mexico. Further, we own an issued U.S. patent directed to synthetic steps related to the commercial process for preparing Galafold
®
, which expires in 2026, as well as issued patents in China, Europe, Hong Kong, India, Israel, and Japan. We jointly own issued U.S., European, Hong Kong, and Mexican patents covering a method of determining whether male Fabry disease patients are likely to respond to treatment with Galafold
®
which expires in 2027. We have two issued U.S. patents covering a method of treating a patient diagnosed with Fabry disease with Galafold
®
wherein the Fabry patient has one of several alpha-Gal A variants. These patents will expire in 2029. We also have a pending U.S. application covering a method of determining which alpha-Gal A variants are likely to be amenable to therapy with Galafold
®
which, if granted, will expire in 2029. Foreign counterpart patents have also been issued in Europe, Japan, Canada, Mexico, and Australia; all of which will also expire in 2029. We also have filed a patent application covering a method of treating renal symptoms in a Fabry patient in relevant jurisdictions. As of February 2019, we have allowance of the U.S. counterpart and when granted, the patent would expired in 2038.
|
•
|
We have an exclusive license to pending patent applications covering the co-administration of Galafold
®
with ERT (recombinant alpha-Gal A). Patents covering specific combinations have issued in the U.S., Europe, Canada, China, India, Israel, Hong Kong, Japan, and Mexico. These issued patents will expire in 2024. We also own a U.S. patent application covering specific doses and dosing regimens of Galafold
®
to treat Fabry disease in combination with ERT (recombinant alpha-Gal A) in the U.S. and foreign counterpart applications in Australia, Canada, Europe, Hong Kong, Japan, and issued patents in Australia, Canada, and China. Any patents issuing from these applications will expire in 2032.
|
•
|
We have patents covering a co-formulation of recombinant acid alpha-glucosidase and Galafold
®
in the U.S. and Australia, as well as pending patent applications in the U.S., Australia, Canada, China, Europe, and Hong Kong. If patents issue from these applications, expiration will be in 2033 or 2034.
|
•
|
As part of the Callidus acquisition, we acquired a portfolio of patent applications including an application series covering reagents and methods for coupling targeting peptides to recombinant lysosomal enzymes, including recombinant acid alpha-glucosidase. Patents in this series are issued in the U.S., Canada, China and Europe, and applications are pending in the U.S., Europe, Japan, Brazil, Canada, China, and South Korea. If patents issue from these applications, expiration will be in 2032 to 2034 depending on the specific application.
|
•
|
Another patent application portfolio related to a modified lysosomal enzyme (acid alpha-glucosidase) that binds more effectively to the receptor and more potent than conventional recombinant enzymes. These applications pending in the U.S., Australia, Brazil, Canada, China, Eurasia, Europe, Israel, India, Japan, South Korea, Mexico, Singapore, Taiwan, South Africa, and other countries. If patents issue from this series, they will expire in 2035 to 2038.
|
•
|
As part of the acquisition of MiaMed, we acquired an exclusive worldwide license to certain patent rights held by the Università di Bologna. These patent rights include two issued U.S. patent and a pending U.S. patent application directed to novel CDD fusion proteins, as well as pending counterpart patent applications in several foreign countries. The issued U.S. patent and the patent applications, if issued, will expire in 2035.
|
•
|
From NCH through the acquisition of Celenex, we have an exclusive license to pending patent applications covering ten AAV program in neurodegenerative disorders, including CLN6, CLN3, and CLN8 Batten diseases. These patent rights include one issued European patent and several pending U.S. patent applications pertaining to various aspects of the gene therapy programs, as well as pending counterpart patent applications in several foreign countries. The issued European patent and the patent applications, if issued, will expire in 2033 or 2040.
|
•
|
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.
|
Competitor
|
Indication
|
|
Product
|
|
Class of Product
|
|
Status
|
|
2018 Sales
|
||
|
|
|
|
|
|
|
|
|
(in millions)
|
||
Sanofi Aventis
|
Fabry Disease
|
|
Fabrazyme
®
|
|
ERT
|
|
Marketed
|
|
$
|
742.5
|
|
Pompe Disease
|
|
Myozyme
®
/ Lumizyme
®
|
|
ERT
|
|
Marketed
|
|
$
|
827.5
|
|
|
Fabry Disease
|
|
GZ402671
|
|
Oral GCS Inhibitor
|
|
Phase 2
|
|
N/A
|
|
||
Pompe Disease
|
|
GZ402666 ("neo GAA")
|
|
ERT
|
|
Phase 3
|
|
N/A
|
|
||
Takeda
|
Fabry Disease
|
|
Replagal
®
|
|
ERT
|
|
Marketed
|
|
$
|
498.1
|
|
Protalix Biotherapeutics
|
Fabry Disease
|
|
PRX-102
|
|
ERT
|
|
Phase 2/3
|
|
N/A
|
|
|
Audentes
|
Pompe Disease
|
|
AT845
|
|
Gene Therapy
|
|
Preclinical
|
|
N/A
|
|
|
Sangamo
|
Fabry Disease
|
|
ST-920
|
|
Gene Therapy
|
|
Preclinical
|
|
N/A
|
|
|
Avrobio
|
Fabry Disease
|
|
AVR-RD-01
|
|
Gene Therapy
|
|
Phase 1/2
|
|
N/A
|
|
|
Pompe Disease
|
|
AVR-RD-03
|
|
Gene Therapy
|
|
Preclinical
|
|
N/A
|
|
||
Spark
|
Pompe Disease
|
|
SPK-3006
|
|
Gene Therapy
|
|
Preclinical
|
|
N/A
|
|
|
Abeona
|
CLN3 Batten
|
|
ABO-201
|
|
Gene Therapy
|
|
Preclinical
|
|
N/A
|
|
•
|
obtaining a sufficiently broad label in each territory that would not unduly restrict patient access;
|
•
|
obtaining additional foreign approvals for Galafold
®
;
|
•
|
continuing to build and maintain an infrastructure capable of supporting product sales, marketing, and distribution of Galafold
®
in the EU, U.S., Japan and other territories where we pursue commercialization directly;
|
•
|
maintaining commercial manufacturing arrangements with third party manufacturers;
|
•
|
maintaining commercial distribution agreements with third party distributors;
|
•
|
launching commercial sales of Galafold
®
, where approved, whether alone or in collaboration with others;
|
•
|
acceptance of Galafold
®
, where approved, by patients, the medical community and third party payors;
|
•
|
effectively competing with other therapies, including potential generics and potential gene therapies;
|
•
|
a continued acceptable safety profile of Galafold
®
;
|
•
|
obtaining and maintaining patent and trade secret protection and regulatory exclusivity;
|
•
|
protecting and enforcing our rights in our intellectual property portfolio; and
|
•
|
obtaining a commercially viable price for our products.
|
•
|
our failure to demonstrate to the satisfaction of the applicable regulatory authorities that any of our product candidates are safe and effective for a particular indication;
|
•
|
the results of clinical trials may not meet the level of statistical significance or other efficacy or safety parameters required by the applicable regulatory authorities for approval;
|
•
|
the applicable regulatory authority may disagree with the number, design, size, conduct, or implementation of our clinical trials or conclude that the data fail to meet statistical or clinical significance;
|
•
|
the applicable regulatory authority may not find the data from preclinical studies and clinical trials sufficient to demonstrate that the product candidate's clinical and other benefits outweigh its safety risks;
|
•
|
the applicable regulatory authority may disagree with our interpretation of data from preclinical studies or clinical trials, and may reject conclusions from preclinical studies or clinical trials, or determine that primary or secondary endpoints from clinical trials were not met, or reject safety conclusions from such studies or trials;
|
•
|
the applicable regulatory authority may not accept data generated at one or more of our clinical trial sites;
|
•
|
the applicable regulatory authority may determine that we did not properly oversee our clinical trials or follow the regulatory authority's advice or recommendations in designing and conducting our clinical trials;
|
•
|
an advisory committee, if convened by the applicable regulatory authority, may recommend against approval of our application or may recommend that the applicable regulatory authority require, as a condition of approval, additional preclinical studies or clinical trials, limitations on approved labeling or distribution and use restrictions, or even if an advisory committee, if convened, makes a favorable recommendation, the respective regulatory authority may still not approve the product candidate; and
|
•
|
the applicable regulatory authority may identify deficiencies in the chemistry, manufacturing, and control sections of our application, our manufacturing processes, facilities, or analytical methods or those of our third party contract manufacturers, and this may lead to significant delays in the approval of our product candidates or to the rejection of our applications altogether.
|
•
|
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 additional clinical trials are conducted.
|
•
|
our inability to recruit, train and retain adequate numbers of effective sales and marketing personnel;
|
•
|
the inability of sales personnel to obtain access to adequate numbers of physicians to prescribe any future products;
|
•
|
the lack of complementary products to be offered by sales personnel, which may put us at a competitive disadvantage relative to companies with more extensive product lines;
|
•
|
unforeseen costs and expenses associated with creating 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 or product candidates;
|
•
|
our distributors may experience financial difficulties;
|
•
|
our distributors may experience compliance related issues and associated government investigations;
|
•
|
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.
|
•
|
the efficacy and potential advantages compared to alternative treatments, including generics and gene therapies;
|
•
|
the prevalence and severity of any side effects;
|
•
|
the ability to offer our product and product candidates for sale at competitive prices;
|
•
|
convenience and ease of administration compared to alternative treatments;
|
•
|
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 coverage or reimbursement.
|
•
|
different regulatory requirements for maintaining approval of drugs in foreign countries;
|
•
|
reduced protection for contractual and intellectual property rights in some countries;
|
•
|
unexpected changes in tariffs, trade barriers and regulatory requirements;
|
•
|
economic weakness, including inflation, or political instability in particular foreign economies and markets;
|
•
|
compliance with tax, employment, immigration and labor laws for employees living or traveling abroad;
|
•
|
foreign currency fluctuations, which could result in increased operating expenses and reduced revenue, and other obligations incident to doing business in another country;
|
•
|
workforce uncertainty in countries where labor unrest is more common than in the U.S.;
|
•
|
noncompliance with the U.S. Foreign Corrupt Practices Act, the U.K. Bribery Act 2010 and similar anti-bribery and anti-corruption laws in other jurisdictions;
|
•
|
tighter restrictions on privacy and the collection and use of patient data; and
|
•
|
business interruptions resulting from geopolitical actions, including war and terrorism, or natural disasters including earthquakes, typhoons, floods and fires.
|
•
|
restrictions on such products, manufacturers or manufacturing processes;
|
•
|
changes to or restrictions on the labeling or marketing of a product;
|
•
|
restrictions on product distribution or use;
|
•
|
requirements to implement a REMS;
|
•
|
requirements to conduct post-marketing studies or clinical trials;
|
•
|
warning or untitled letters;
|
•
|
withdrawal of the products from the market;
|
•
|
refusal to approve pending applications or supplements to approved applications that we submit;
|
•
|
recall of products;
|
•
|
fines, restitution or disgorgement of profits or revenues;
|
•
|
suspension or withdrawal of marketing approvals;
|
•
|
refusal to permit the import or export of our products;
|
•
|
product seizure;
|
•
|
injunctions; or
|
•
|
the imposition of civil or criminal penalties.
|
•
|
the U.S. federal Anti-Kickback Statute, which prohibits, among other things, knowingly and willfully soliciting, offering, receiving or providing remuneration, directly or indirectly, in cash or in kind, to induce or reward either the referral of an individual for, or the purchase, order or recommendation of, any good or service, for which payment may be made under federal and state healthcare programs such as Medicare and Medicaid. A person or entity does not need to have actual knowledge of the statute or specific intent to violate it in order to have committed a violation. Several other countries, including the United Kingdom, have enacted similar anti-kickback, fraud and abuse, and healthcare laws and regulations;
|
•
|
the U.S. federal False Claims Act, which imposes criminal and civil penalties, including through civil whistleblower or qui tam actions, against individuals or entities for knowingly presenting, or causing to be presented, to the federal government, claims for payment that are false or fraudulent or making a false statement to avoid, decrease or conceal an obligation to pay money to the federal government. In addition, the government may assert that a claim including items and services resulting from a violation of the federal Anti-Kickback Statute constitutes a false or fraudulent claim for purposes of the False Claims Act. There is also a separate false claims provision imposing criminal penalties. Applicable regulations of both the EMA and EU member states also impose liability for failing to comply with fraud and abuse laws or improperly using information obtained in in the course of clinical trials with the EMA or other regulatory authorities;
|
•
|
The U.S. federal Health Insurance Portability and Accountability Act of 1996 ("HIPAA") which imposes criminal and civil liability for executing a scheme to defraud any healthcare benefit program or making false statements relating to healthcare matters. Similar to the federal Anti-Kickback Statute, a person or entity does not need to have actual knowledge of the statute to defraud any healthcare benefit program or specific intent to violate it in order to have committed a violation. This statute also may impose monetary penalties on any offers or transfers of remuneration to Medicare or Medicaid beneficiaries (patients) which is likely to influence the beneficiary's selection of particular supplier of government payable items. Similarly, the collection and use of personal health data in the EU is governed by the EU General Data Protection Regulation (the "GDPR"), with many requirements mandated by the GDPR for the consent of the individuals to whom the personal data relates, the information provided to the individuals, transfer of personal data within and outside of the EU and the security and confidentiality of the personal data. Enforcement of the GDPR began on May 25, 2018, and failure to comply with the requirements of the GDPR may result in substantial fines and other administrative penalties. The GDPR increases our responsibility and liability in relation to personal data that we process and we may be required to put in place additional mechanisms ensuring compliance with the GDPR. This may be onerous and adversely affect our business, financial condition, results of operations and prospects;
|
•
|
HIPAA, as amended by the Health Information Technology for Economic and Clinical Health Act of 2009, and its implementing regulations, which also imposes obligations on certain covered entity healthcare providers, health plans, and healthcare clearinghouses as well as their business associates that perform certain services involving the use or disclosure of individually identifiable health information, including mandatory contractual terms, with respect to safeguarding the privacy, security and transmission of individually identifiable health information;
|
•
|
U.S. federal laws requiring drug manufacturers to report annually information related to certain payments and other transfers of value made to physicians (defined to include doctors, dentists, optometrists, podiatrists and chiropractors) and teaching hospitals, as well as ownership or investment interests held by physicians and their immediate family members, including under the federal Open Payments program, commonly known as the Sunshine Act, as well as other state and foreign laws regulating marketing activities and requiring manufacturers to report marketing expenditures, payments and other transfers of value to physicians and other healthcare providers. Similarly, payments made to physicians in certain EU member states must be publicly disclosed. Moreover, agreements with physicians often must be the subject of prior notification and approval by the physician's employer, his or her competent professional organization and/or the regulatory authorities of the individual EU member states. These requirements are provided in the national laws, industry codes or professional codes of conduct, applicable in the EU member states. In addition, the provision of benefits or advantages to physicians to induce or encourage the prescription, recommendation, endorsement, purchase, supply, order or use of medicinal products is prohibited in the EU. Failure to comply with these requirements could result in reputational risk, public reprimands, administrative penalties, fines or imprisonment;
|
•
|
U.S. federal government price reporting laws, which require us to calculate and report complex pricing metrics to government programs, where such reported prices may be used in the calculation of reimbursement and/or discounts on our marketed drugs. Participation in these programs and compliance with the applicable requirements may subject us to potentially significant discounts on our products, increased infrastructure costs, potential liability for the failure to report such prices in an accurate and timely manner, and potentially limit our ability to offer certain marketplace discounts;
|
•
|
U.S. Foreign Corrupt Practices Act, which prohibit us and third parties working on our behalf from making payments to foreign government officials to assist in obtaining or retaining business. Specifically, the anti-bribery provisions of the FCPA prohibit the willful use of the mails or any means of instrumentality of interstate commerce corruptly in furtherance of any offer, payment, promise to pay, or authorization of the payment of money or anything of value to any person, while knowing that all or a portion of such money or thing of value will be offered, given or promised, directly or indirectly, to a foreign official to influence the foreign official in his or her official capacity, induce the foreign official to do or omit to do an act in violation of his or her lawful duty, or to secure any improper advantage in order to assist in obtaining or retaining business for or with, or directing business to, any person; and
|
•
|
state and foreign equivalents of each of the above laws, including foreign anti-bribery and corruption laws and state anti-kickback and false claims laws, which may apply to sales or marketing arrangements and claims involving healthcare items or services reimbursed by non-governmental payors, including private insurers; state laws which require pharmaceutical companies to comply with the pharmaceutical industry's voluntary compliance guidelines and the relevant compliance guidance promulgated by the federal government or otherwise restricting payments that may be made to healthcare providers; and state and foreign laws governing the privacy and security of health information in certain circumstances, many of which differ from each other in significant ways and often are not preempted by HIPAA, thus complicating compliance efforts.
|
•
|
reduced resources of our management to pursue our business strategy;
|
•
|
decreased demand for any product candidates or products that we may develop;
|
•
|
injury to our reputation and significant negative media attention;
|
•
|
regulatory investigations, prosecutions or enforcement actions that could require costly recalls or product modifications;
|
•
|
withdrawal of clinical trial participants;
|
•
|
significant costs to defend the related litigation;
|
•
|
increased insurance costs, or an inability to maintain appropriate insurance coverage;
|
•
|
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; and
|
•
|
the inability to commercialize any products that we may develop.
|
•
|
choose not to seek regulatory approval in the U.S., EU or other key jurisdictions;
|
•
|
be delayed in obtaining marketing approval for our product candidates;
|
•
|
not obtain marketing approval at all;
|
•
|
obtain approval for indications or patient populations that are not as broad as intended or desired;
|
•
|
obtain approval with labeling that includes significant use or distribution restrictions or safety warnings, including boxed warnings;
|
•
|
be subject to additional post-marketing testing requirements, safety strategies or restrictions, such as a requirement of a risk evaluation and mitigation strategy, or REMS; or
|
•
|
have the product removed from the market after obtaining regulatory approval.
|
•
|
clinical trials of our product candidates may produce negative or inconclusive results, and we may decide, or regulators may require us, to conduct additional clinical trials or abandon product development programs;
|
•
|
the number of patients required for clinical trials of our product candidates may be larger than we anticipate, enrollment in these clinical trials may be slower than we anticipate, or patients may drop out of these clinical trials at a higher rate than we anticipate;
|
•
|
we may be unable to enroll a sufficient number of patients in our trials to ensure adequate statistical power to detect any statistically significant treatment effects;
|
•
|
our third party contractors may fail to comply with regulatory requirements or meet their contractual obligations to us in a timely manner, or at all;
|
•
|
regulators, institutional review boards, or independent ethics committees may not authorize us or our investigators to commence a clinical trial or conduct a clinical trial at a prospective trial site;
|
•
|
we may have delays in reaching or fail to reach agreement on acceptable clinical trial contracts or clinical trial protocols with prospective trial sites;
|
•
|
we may have to suspend or terminate clinical trials of our product candidates for various reasons, including a finding that the participants are being exposed to unacceptable health risks;
|
•
|
regulators, institutional review boards, or independent ethics committees may require that we or our investigators suspend or terminate clinical research for various reasons, including noncompliance with regulatory requirements or a finding that the participants are being exposed to unacceptable health risks;
|
•
|
the cost of clinical trials of our product candidates may be greater than we anticipate;
|
•
|
the supply or quality of our product candidates or other materials necessary to conduct clinical trials of our product candidates may be insufficient or inadequate; or
|
•
|
our product candidates may have undesirable side effects or other unexpected characteristics, causing us or our investigators, regulators, institutional review boards or independent ethics committees to suspend or terminate the trials.
|
•
|
severity of the disease under investigation;
|
•
|
eligibility criteria for the clinical trial in question;
|
•
|
perceived risks and benefits of the product candidate under study;
|
•
|
efforts to facilitate timely enrollment in clinical trials;
|
•
|
patient referral practices of physicians;
|
•
|
the ability to monitor patients adequately during and after treatment; and
|
•
|
proximity and availability of clinical trial sites for prospective patients.
|
•
|
continue our development and commercialization of, and seek regulatory approvals for, product candidates in the U.S., the EU, Japan and other foreign countries, as applicable;
|
•
|
conduct additional clinical trials to support the full approval of Galafold
®
in the U.S. and post-approval commitments or trials;
|
•
|
continue communicating with the EMA, as necessary, regarding post-marketing requirements and clinical trials for Galafold
®
;
|
•
|
continue to or initiate the regulatory submission process for marketing approval of Galafold
®
outside of the U.S. and EU, as applicable;
|
•
|
build and maintain our commercial infrastructure so that it is capable of supporting product sales, marketing and distribution of Galafold
®
and our other product candidates in the EU, Japan and the U.S. or other territories in which we may receive regulatory approval;
|
•
|
continue wind-down of our Phase 3 clinical trial of SD-101 for the treatment of EB;
|
•
|
continue our preclinical studies and clinical trials on the use of AT-GAA for Pompe disease and our gene therapies for Fabry, Pompe, Batten’s and other LSDs; and
|
•
|
continue our preclinical studies of and potentially conduct clinical studies of ERT and gene therapy for CDD.
|
•
|
successfully complete development activities and obtain additional regulatory and pricing and reimbursement approvals for, and successfully commercialize, Galafold
®
;
|
•
|
develop and maintain a commercial organization capable of sales, marketing, and distribution for Galafold
®
and any product candidates we intend to market, in the countries where we have chosen to commercialize the product candidates ourselves including the U.S. and Japan;
|
•
|
manufacture commercial quantities of our products at acceptable cost levels;
|
•
|
obtain a commercially viable price for our products;
|
•
|
obtain coverage and adequate reimbursement from third-parties, including government payors;
|
•
|
successfully satisfy post-marketing requirements that the FDA, EMA, or other foreign regulatory authorities may impose for migalastat HCl
or any of our other product candidates that may receive regulatory approval, including pediatric trials and patient registries;
|
•
|
successfully complete development activities, including the necessary preclinical studies and clinical trials, with respect to product candidates, including AT-GAA and our gene therapies;
|
•
|
complete and submit regulatory submissions to the FDA and obtain regulatory approval for our product candidates including AT-GAA and our gene therapies; and
|
•
|
complete and submit applications to, and obtain regulatory approval from, foreign regulatory authorities.
|
•
|
significantly delay, scale back, or discontinue the development or the commercialization of our product or product candidates or one or more of our other research and development initiatives;
|
•
|
seek collaborators for Galafold
®
or one or more of our current or future product candidates at an earlier stage than otherwise would be desirable, or on terms that are less favorable than might otherwise be available;
|
•
|
relinquish or license on unfavorable terms our rights to our technologies, product or product candidates that we otherwise would seek to develop or commercialize ourselves; or
|
•
|
significantly curtail operations.
|
•
|
the costs of commercialization activities, including maintaining sales, marketing, and distribution capabilities for Galafold
®
and any product candidates for which we may receive regulatory approval in regions where we choose to commercialize our products on our own;
|
•
|
the scope, progress, results, and costs of preclinical development, laboratory testing, and clinical trials for our product candidates and any other product candidates that we may in-license or acquire;
|
•
|
the cost of manufacturing drug supply for our preclinical studies and clinical trials, including the significant cost of manufacturing AT-GAA and our gene therapies;
|
•
|
the cost of transferring manufacturing technologies for our gene therapies to CMOs;
|
•
|
the outcome, timing, and cost of the regulatory approval process by the FDA, EMA, PMDA and other foreign regulatory authorities, including the potential for regulatory authorities to require that we perform more studies than those that we currently anticipate for our product and product candidates;
|
•
|
the cost of filing, prosecuting, defending, and enforcing any patent claims and other intellectual property rights;
|
•
|
the cost and timing of completion of existing or expanded commercial-scale outsourced manufacturing activities;
|
•
|
the cost of defending any claims asserted against us;
|
•
|
the emergence of competing technologies and other adverse market developments;
|
•
|
the extent to which we acquire or invest in additional businesses, products, and technologies.
|
•
|
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;
|
•
|
licenses from other third parties will not be required to commercialize patented products;
|
•
|
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;
|
•
|
the patents of others will not have a negative effect on our ability to do business; or
|
•
|
patent authorities will not identify deficiencies in our patent applications and refuse to grant our patents.
|
•
|
We do not hold composition of matter patents covering Galafold
®
and we have method of manufacturing patent applications allowed for ATB200 as well as method of treatment patents allowed for migalastat HCl. There can be no assurance that the allowed applications will be issued or that the scope of such patents, if they issue, will be sufficient to protect our product. 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.
|
•
|
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;
|
•
|
inability to demonstrate comparability to GMP commercial scale product for biologic products;
|
•
|
inability to manufacture batches that meet specifications and quality standards;
|
•
|
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;
|
•
|
the possible misappropriation of our proprietary information, including our trade secrets and know-how; and
|
•
|
the possible termination or nonrenewal of the agreement by the third party at a time that is costly or inconvenient for us.
|
•
|
collaborators have significant discretion in determining the efforts and resources that they will apply to these collaborations;
|
•
|
collaborators may not pursue development and commercialization of our product or product candidates or may elect not to continue or renew development or commercialization programs, based on clinical trial results, changes in the collaborators' strategic focus or available funding, or external factors such as an acquisition that diverts resources or creates competing priorities;
|
•
|
collaborators may delay clinical trials, provide insufficient funding for a clinical trial program, stop a clinical trial or abandon a product candidate, repeat or conduct new clinical trials or require a new formulation of a product candidate for clinical testing;
|
•
|
collaborators could independently develop, or develop with third parties, products that compete directly or indirectly with our products or product candidates if the collaborators believe that competitive products are more likely to be successfully developed or can be commercialized under terms that are more economically attractive than ours;
|
•
|
a collaborator with marketing and distribution rights to one or more products may not commit sufficient resources to the marketing and distribution of such product or products;
|
•
|
collaborators may not properly maintain or defend our intellectual property rights or may use our proprietary information in such a way as to invite litigation that could jeopardize or invalidate our intellectual property or proprietary information or expose us to potential liability;
|
•
|
collaborators may infringe the intellectual property rights of third parties, which may expose us to litigation and potential liability;
|
•
|
disputes may arise between the collaborator and us as to the ownership of intellectual property arising during the collaboration;
|
•
|
we may grant exclusive rights to our collaborators, which would prevent us from collaborating with others;
|
•
|
disputes may arise between the collaborators and us that result in the delay or termination of the research, development or commercialization of our products or product candidates or that result in costly litigation or arbitration that diverts management attention and resources; and
|
•
|
collaborations may be terminated and, if terminated, may result in a need for additional capital to pursue further development or commercialization of the applicable product candidates.
|
•
|
managing the development and commercialization of any product candidates approved for marketing;
|
•
|
overseeing our ongoing preclinical studies and clinical trials effectively;
|
•
|
identifying, recruiting, maintaining, motivating and integrating additional employees, including any sales and marketing personnel engaged in connection with the commercialization of any approved product;
|
•
|
managing our internal development efforts effectively while complying with our contractual obligations to licensors, licensees, contractors and other third parties;
|
•
|
improving our managerial, development, operational and financial systems and procedures;
|
•
|
developing our compliance infrastructure and processes to ensure compliance with regulations applicable to public companies;
|
•
|
developing biologics and gene therapy manufacturing expertise; and
|
•
|
expanding our facilities.
|
•
|
FDA or similar regulations of foreign regulatory authorities, including those laws requiring the reporting of true, complete and accurate information to such authorities;
|
•
|
manufacturing standards;
|
•
|
federal and state healthcare fraud and abuse laws and regulations, anti-bribery and corruption laws, and similar laws and regulations established and enforced by foreign regulatory authorities; or
|
•
|
laws that require the reporting of financial information or data accurately.
|
•
|
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 outstanding voting stock to amend or repeal certain provisions of our charter or bylaws.
|
•
|
the success of competitive products or technologies;
|
•
|
regulatory actions with respect to our product or product candidates or our competitors' products or product candidates;
|
•
|
actual or anticipated changes in our growth rate relative to our competitors;
|
•
|
the outcome of any patent infringement or other litigation that may be brought against us;
|
•
|
announcements by us or our competitors of significant acquisitions, strategic collaborations, joint ventures, collaborations or capital commitments;
|
•
|
results of clinical trials of our product candidates or those of our competitors;
|
•
|
regulatory or legal developments in the EU, U.S. and other countries;
|
•
|
the impact of Brexit on our operations, supply chain, regulatory approvals and personnel;
|
•
|
developments or disputes concerning patent applications, issued patents or other proprietary rights;
|
•
|
the recruitment or departure of key personnel;
|
•
|
the level of expenses related to our product or any of our product candidates or clinical development programs;
|
•
|
actual or anticipated variations in our quarterly operating results;
|
•
|
the number and characteristics of our efforts to in-license or acquire additional product candidates or products;
|
•
|
introduction of new products or services by us or our competitors;
|
•
|
failure to meet the estimates and projections of the investment community or that we may otherwise provide to the public;
|
•
|
actual or anticipated changes in estimates as to financial results, development timelines or recommendations by securities analysts;
|
•
|
variations in our financial results or those of companies that are perceived to be similar to us;
|
•
|
fluctuations in the valuation of companies perceived by investors to be comparable to us;
|
•
|
share price and volume fluctuations attributable to inconsistent trading volume levels of our shares;
|
•
|
announcement or expectation of additional financing efforts;
|
•
|
sales of our common stock by us, our insiders or our other stockholders;
|
•
|
changes in accounting practices;
|
•
|
lawsuits and other claims asserted against us;
|
•
|
changes in the structure of healthcare payment systems;
|
•
|
market conditions in the pharmaceutical and biotechnology sectors;
|
•
|
general economic, industry and market conditions;
|
•
|
publication of research reports about us, our competitors or our industry, or positive or negative recommendations or withdrawal of research coverage by securities or industry analysts;
|
•
|
other events or factors, many of which are beyond our control; and
|
•
|
the other factors described in this "Risk Factors" section.
|
•
|
a limited availability of market quotations for our securities;
|
•
|
reduced liquidity with respect to our securities;
|
•
|
a determination that our shares are a "penny stock," which will require brokers trading in our shares to adhere to more stringent rules, possibly resulting in a reduced level of trading activity in the secondary trading market for our shares;
|
•
|
a limited amount of news and analyst coverage for our company; and
|
•
|
a decreased ability to issue additional securities or obtain additional financing in the future.
|
Location
|
Approximate
Square Feet
|
|
Use
|
|
Lease expiry date
|
|
Cranbury, New Jersey, USA
|
90,000
|
|
|
Office and laboratory
|
|
September 2025
|
United Kingdom
|
46,617
|
|
|
Office
|
|
August 2028
|
Princeton, New Jersey, USA
|
21,922
|
|
|
Office
|
|
January 2022
|
|
High
|
|
Low
|
||||
2018
|
|
|
|
||||
First Quarter
|
$
|
17.12
|
|
|
$
|
13.76
|
|
Second Quarter
|
$
|
17.09
|
|
|
$
|
13.13
|
|
Third Quarter
|
$
|
16.54
|
|
|
$
|
11.60
|
|
Fourth Quarter
|
$
|
13.44
|
|
|
$
|
8.38
|
|
|
High
|
|
Low
|
||||
2017
|
|
|
|
||||
First Quarter
|
$
|
7.79
|
|
|
$
|
5.13
|
|
Second Quarter
|
$
|
10.44
|
|
|
$
|
6.65
|
|
Third Quarter
|
$
|
15.78
|
|
|
$
|
10.08
|
|
Fourth Quarter
|
$
|
16.24
|
|
|
$
|
12.51
|
|
|
2018
|
|
2017
|
|
2016
|
|
2015
|
|
2014
|
||||||||||
Revenue:
|
|
|
|
|
|
|
|
|
|
||||||||||
Net product sales
|
$
|
91,245
|
|
|
$
|
36,930
|
|
|
$
|
4,958
|
|
|
—
|
|
|
—
|
|
||
Research revenue
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
1,224
|
|
|||||
Total revenue
|
91,245
|
|
|
36,930
|
|
|
4,958
|
|
|
—
|
|
|
1,224
|
|
|||||
Total cost of goods sold
|
14,404
|
|
|
6,236
|
|
|
833
|
|
|
—
|
|
|
—
|
|
|||||
Gross profit
|
76,841
|
|
|
30,694
|
|
|
4,125
|
|
|
—
|
|
|
1,224
|
|
|||||
Operating expenses:
|
|
|
|
|
|
|
|
|
|
||||||||||
Research and development
|
270,902
|
|
|
149,310
|
|
|
104,793
|
|
|
76,943
|
|
|
47,624
|
|
|||||
Selling, general and administrative
|
127,200
|
|
|
88,671
|
|
|
71,151
|
|
|
47,269
|
|
|
20,717
|
|
|||||
Changes in fair value of contingent consideration payable
|
3,300
|
|
|
(234,322
|
)
|
|
6,760
|
|
|
4,377
|
|
|
100
|
|
|||||
Loss on impairment of assets
|
—
|
|
|
465,427
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|||||
Restructuring charges
|
—
|
|
|
—
|
|
|
69
|
|
|
15
|
|
|
(63
|
)
|
|||||
Depreciation and amortization
|
4,216
|
|
|
3,593
|
|
|
3,242
|
|
|
1,833
|
|
|
1,547
|
|
|||||
Total operating expenses
|
405,618
|
|
|
472,679
|
|
|
186,015
|
|
|
130,437
|
|
|
69,925
|
|
|||||
Loss from operations
|
(328,777
|
)
|
|
(441,985
|
)
|
|
(181,890
|
)
|
|
(130,437
|
)
|
|
(68,701
|
)
|
|||||
Other income (expense):
|
|
|
|
|
|
|
|
|
|
|
|
|
|
||||||
Interest income
|
10,461
|
|
|
4,096
|
|
|
1,602
|
|
|
929
|
|
|
223
|
|
|||||
Interest expense
|
(22,402
|
)
|
|
(17,240
|
)
|
|
(5,398
|
)
|
|
(1,578
|
)
|
|
(1,484
|
)
|
|||||
Change in fair value of derivatives
|
(2,739
|
)
|
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|||||
Loss on extinguishment of debt
|
—
|
|
|
—
|
|
|
(13,302
|
)
|
|
(952
|
)
|
|
—
|
|
|||||
Other income (expense)
|
(5,632
|
)
|
|
6,008
|
|
|
(4,793
|
)
|
|
(80
|
)
|
|
(77
|
)
|
|||||
Loss before income tax
|
(349,089
|
)
|
|
(449,121
|
)
|
|
(203,781
|
)
|
|
(132,118
|
)
|
|
(70,039
|
)
|
|||||
Income tax benefit
|
94
|
|
|
165,119
|
|
|
3,739
|
|
|
—
|
|
|
1,113
|
|
|||||
Net loss attributable to common stockholders
|
$
|
(348,995
|
)
|
|
$
|
(284,002
|
)
|
|
$
|
(200,042
|
)
|
|
$
|
(132,118
|
)
|
|
$
|
(68,926
|
)
|
Net loss attributable to common stockholders per common share — basic and diluted
|
$
|
(1.88
|
)
|
|
$
|
(1.85
|
)
|
|
$
|
(1.49
|
)
|
|
$
|
(1.20
|
)
|
|
$
|
(0.93
|
)
|
Weighted-average common shares outstanding — basic and diluted
|
185,790
|
|
|
153,355
|
|
|
134,402
|
|
|
109,924
|
|
|
74,444
|
|
|
As of December 31,
|
||||||||||||||||||
|
2018
|
|
2017
|
|
2016
|
|
2015
|
|
2014
|
||||||||||
Cash and cash equivalents and marketable securities
|
$
|
504,152
|
|
|
$
|
358,562
|
|
|
$
|
330,351
|
|
|
$
|
214,033
|
|
|
$
|
169,139
|
|
Working capital
|
464,971
|
|
|
321,925
|
|
|
229,105
|
|
|
142,985
|
|
|
134,392
|
|
|||||
Total assets
|
789,951
|
|
|
627,024
|
|
|
1,036,845
|
|
|
908,384
|
|
|
209,967
|
|
|||||
Total liabilities
|
447,039
|
|
|
274,174
|
|
|
676,694
|
|
|
560,550
|
|
|
87,789
|
|
|||||
Accumulated deficit
|
(1,412,222
|
)
|
|
(1,063,610
|
)
|
|
(779,608
|
)
|
|
(579,566
|
)
|
|
(447,448
|
)
|
|||||
Total stockholders' equity
|
342,912
|
|
|
352,850
|
|
|
360,151
|
|
|
347,834
|
|
|
122,178
|
|
|
|
Years Ended December 31,
|
||||||||||
(in thousands)
|
|
2018
|
|
2017
|
|
Change
|
||||||
Net product sales
|
|
$
|
91,245
|
|
|
$
|
36,930
|
|
|
$
|
54,315
|
|
Cost of goods sold
|
|
14,404
|
|
|
6,236
|
|
|
8,168
|
|
|||
Cost of goods sold as a percentage of net product sales
|
|
15.8
|
%
|
|
16.9
|
%
|
|
(1.1
|
)%
|
|||
Operating expenses:
|
|
|
|
|
|
|
||||||
Research and development
|
|
270,902
|
|
|
149,310
|
|
|
121,592
|
|
|||
Selling, general and administrative
|
|
127,200
|
|
|
88,671
|
|
|
38,529
|
|
|||
Changes in fair value of contingent consideration payable
|
|
3,300
|
|
|
(234,322
|
)
|
|
237,622
|
|
|||
Loss on impairment of asset
|
|
—
|
|
|
465,427
|
|
|
(465,427
|
)
|
|||
Depreciation
|
|
4,216
|
|
|
3,593
|
|
|
623
|
|
|||
Other income (expense):
|
|
|
|
|
|
|
||||||
Interest income
|
|
10,461
|
|
|
4,096
|
|
|
6,365
|
|
|||
Interest expense
|
|
(22,402
|
)
|
|
(17,240
|
)
|
|
(5,162
|
)
|
|||
Change in fair value of derivatives
|
|
(2,739
|
)
|
|
—
|
|
|
(2,739
|
)
|
|||
Other (expense) income
|
|
(5,632
|
)
|
|
6,008
|
|
|
(11,640
|
)
|
|||
Income tax benefit
|
|
94
|
|
|
165,119
|
|
|
(165,025
|
)
|
|||
Net loss attributable to common stockholders
|
|
$
|
(348,995
|
)
|
|
$
|
(284,002
|
)
|
|
$
|
(64,993
|
)
|
(in thousands)
|
|
Years Ended December 31,
|
||||||
Projects
|
|
2018
|
|
2017
|
||||
Third party direct project expenses
|
|
|
|
|
|
|
||
Migalastat (Fabry Disease)
|
|
$
|
12,665
|
|
|
$
|
11,107
|
|
AT-GAA (Pompe Disease)
|
|
55,919
|
|
|
49,890
|
|
||
SD-101 (EB-Epidermolysis Bullosa)
|
|
337
|
|
|
15,424
|
|
||
Gene therapy programs
|
|
137
|
|
|
—
|
|
||
Pre-clinical programs
|
|
1,225
|
|
|
539
|
|
||
Total third party direct project expenses
|
|
70,283
|
|
|
76,960
|
|
||
Other project costs
|
|
|
|
|
|
|
||
Personnel costs
|
|
62,999
|
|
|
50,095
|
|
||
Other costs
|
|
30,620
|
|
|
22,255
|
|
||
Total other project costs
|
|
93,619
|
|
|
72,350
|
|
||
Business development transactions
|
|
107,000
|
|
|
—
|
|
||
Total research and development costs
|
|
$
|
270,902
|
|
|
$
|
149,310
|
|
|
|
Years Ended December 31,
|
|||||||||
(in thousands)
|
|
2017
|
|
2016
|
|
Change
|
|||||
Net product sales
|
|
$
|
36,930
|
|
|
4,958
|
|
|
$
|
31,972
|
|
Cost of goods sold
|
|
6,236
|
|
|
833
|
|
|
5,403
|
|
||
Cost of goods sold as a percentage of net product sales
|
|
16.9
|
%
|
|
16.8
|
%
|
|
0.1
|
%
|
||
Operating expenses:
|
|
|
|
|
|
|
|||||
Research and development
|
|
149,310
|
|
|
104,793
|
|
|
44,517
|
|
||
Selling, general and administrative
|
|
88,671
|
|
|
71,151
|
|
|
17,520
|
|
||
Changes in fair value of contingent consideration payable
|
|
(234,322
|
)
|
|
6,760
|
|
|
(241,082
|
)
|
||
Loss on impairment of asset
|
|
465,427
|
|
|
—
|
|
|
465,427
|
|
||
Restructuring charges
|
|
—
|
|
|
69
|
|
|
(69
|
)
|
||
Depreciation
|
|
3,593
|
|
|
3,242
|
|
|
351
|
|
||
Other income (expense):
|
|
|
|
|
|
|
|||||
Interest income
|
|
4,096
|
|
|
1,602
|
|
|
2,494
|
|
||
Interest expense
|
|
(17,240
|
)
|
|
(5,398
|
)
|
|
(11,842
|
)
|
||
Change in fair value of derivatives
|
|
—
|
|
|
(13,302
|
)
|
|
13,302
|
|
||
Other income (expense)
|
|
6,008
|
|
|
(4,793
|
)
|
|
10,801
|
|
||
Income tax benefit
|
|
165,119
|
|
|
3,739
|
|
|
161,380
|
|
||
Net loss attributable to common stockholders
|
|
(284,002
|
)
|
|
(200,042
|
)
|
|
(83,960
|
)
|
(in thousands)
|
|
Years Ended December 31,
|
||||||
Projects
|
|
2017
|
|
2016
|
||||
Third party direct project expenses
|
|
|
|
|
|
|
||
Migalastat (Fabry Disease)
|
|
$
|
11,107
|
|
|
$
|
14,055
|
|
AT-GAA (Pompe Disease)
|
|
49,890
|
|
|
20,548
|
|
||
SD-101 (EB-Epidermolysis Bullosa)
|
|
15,424
|
|
|
9,530
|
|
||
Pre-clinical programs
|
|
539
|
|
|
6,939
|
|
||
Total third party direct project expenses
|
|
76,960
|
|
|
51,072
|
|
||
Other project costs
|
|
|
|
|
||||
Personnel costs
|
|
50,095
|
|
|
36,624
|
|
||
Other costs
|
|
22,255
|
|
|
17,097
|
|
||
Total other project costs
|
|
72,350
|
|
|
53,721
|
|
||
Total research and development costs
|
|
$
|
149,310
|
|
|
$
|
104,793
|
|
•
|
internal costs associated with our research and clinical development 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.
|
•
|
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 FDA or other regulatory authority to conduct clinical trials beyond those currently anticipated.
|
•
|
the feasibility and timing of achievement of development, regulatory and commercial milestones;
|
•
|
expected costs to develop the in-process research and development into commercially viable products; and
|
•
|
future expected cash flows from product sales.
|
•
|
fees owed to contract research organizations in connection with preclinical, 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.
|
•
|
the progress and results of our preclinical and clinical trials of our drug candidates;
|
•
|
the cost of manufacturing drug supply for our clinical and preclinical studies, including the significant cost of manufacturing Pompe ERT and gene therapies;
|
•
|
the scope, progress, results and costs of preclinical development, laboratory testing and clinical trials for our product candidates including those testing the use of pharmacological chaperones co-formulated and co-administered with ERT and for the treatment of LSDs and gene therapies for the treatment of rare genetic metabolic diseases;
|
•
|
the future results of on-going preclinical research and subsequent clinical trials for CDD, including our ability to obtain regulatory approvals and commercialize CDKL5 therapies and obtain market acceptance for such therapies;
|
•
|
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;
|
•
|
our ability to successfully commercialize Galafold
®
(“migalastat HCl”);
|
•
|
our ability to manufacture or supply sufficient clinical or commercial products;
|
•
|
our ability to obtain reimbursement for Galafold
®
;
|
•
|
our ability to satisfy post-marketing commitments or requirements for continued regulatory approval of Galafold
®
;
|
•
|
our ability to obtain market acceptance of Galafold
®
;
|
•
|
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;
|
•
|
our ability to successfully integrate our acquired products and technologies into our business, including the possibility that the expected benefits of the transactions will not be fully realized by us or may take longer to realize than expected;
|
•
|
our ability to establish collaborations and obtain milestone, royalty or other payments from any such collaborators;
|
•
|
our ability to adjust to changes in European and United Kingdom markets as the United Kingdom leaves the European Union; and
|
•
|
fluctuations in foreign currency exchange rates; and changes in accounting standards.
|
(in thousands)
|
Total
|
|
Less than 1 year
|
|
1-3 years
|
|
3-5 years
|
|
Over 5 years
|
||||||||||
Operating lease obligations
(2)
|
$
|
30,887
|
|
|
$
|
6,244
|
|
|
$
|
7,623
|
|
|
$
|
6,982
|
|
|
$
|
10,038
|
|
Capital lease obligations, including interest
(3)
|
331
|
|
|
194
|
|
|
107
|
|
|
30
|
|
|
—
|
|
|||||
Debt obligations, including interest
(4)
|
508,067
|
|
|
22,430
|
|
|
45,149
|
|
|
440,488
|
|
|
—
|
|
|||||
Purchase obligations
(5)
|
47,287
|
|
|
47,287
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|||||
Total fixed contractual obligations
(1)
|
$
|
586,572
|
|
|
$
|
76,155
|
|
|
$
|
52,879
|
|
|
$
|
447,500
|
|
|
$
|
10,038
|
|
(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, (d) agreements with clinical research organizations and other outside contractors who are partially responsible for conducting and monitoring our clinical trials for our drug candidates including Galafold
®
. These contractual obligations are not reflected in the table above because we may terminate them without penalty, and (e) contracts that are entered into in the ordinary course of business which are not material in the aggregate in any period presented above.
|
(2)
|
Represents the future payments on operating leases for properties, equipment and vehicles at the United States and international locations. For more details, refer to "— Note 13. Leases," in our Notes to Consolidated Financial Statements.
|
(3)
|
Represents the future payments of principal and interest to be made on our capital leases. For more details, refer to "— Note 13. Leases," in our Notes to Consolidated Financial Statements.
|
(4)
|
Represents the future payments of principal and interest to be made on our $250 million 3% unsecured Convertible Notes due 2023 (the "Convertible Notes") and our $150 million Secured Senior Term Loan due 2023 (“Senior Secured Term Loan”). The Convertible Notes bear interest at a fixed rate of 3.00% per year, payable semiannually on June 15 and December 15 of each year, beginning on June 15, 2017 and will mature on December 15, 2023. The Senior Secured Term Loan bears interest at a rate equal to the 3-month LIBOR plus 7.5% per year, payable quarterly of each year, beginning on December 31, 2019 and will mature on September 28, 2023. In the first quarter of 2019, we converted a portion of the Convertible Notes into equity. For more details, refer to "— Note 12. Debt," in our Notes to Consolidated Financial Statements.
|
(5)
|
Represent minimum purchase commitments due to third parties. Contracts for which our commitment is variable, based on volumes, with no fixed minimum quantities, and contracts that can be canceled without payment penalties have been excluded. The purchase obligations included above are in addition to amounts included in total recorded on our December 31, 2018 consolidated balance sheet.
|
•
|
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/ DAPHNE QUIMI
|
Chairman and Chief Executive Officer
|
|
Chief Financial Officer
|
/s/ Ernst & Young LLP
|
/s/ Ernst & Young LLP
|
|
December 31,
|
||||||
|
2018
|
|
2017
|
||||
Assets
|
|
|
|
||||
Current assets:
|
|
|
|
||||
Cash and cash equivalents
|
$
|
79,749
|
|
|
$
|
49,060
|
|
Investments in marketable securities
|
424,403
|
|
|
309,502
|
|
||
Accounts receivable
|
21,962
|
|
|
9,464
|
|
||
Inventories
|
8,390
|
|
|
4,623
|
|
||
Prepaid expenses and other current assets
|
16,592
|
|
|
19,316
|
|
||
Total current assets
|
551,096
|
|
|
391,965
|
|
||
Property and equipment, less accumulated depreciation of $15,671 and $12,515 at December 31, 2018 and 2017, respectively
|
11,375
|
|
|
9,062
|
|
||
In-process research & development
|
23,000
|
|
|
23,000
|
|
||
Goodwill
|
197,797
|
|
|
197,797
|
|
||
Other non-current assets
|
6,683
|
|
|
5,200
|
|
||
Total Assets
|
$
|
789,951
|
|
|
$
|
627,024
|
|
Liabilities and Stockholders' Equity
|
|
|
|
||||
Current liabilities:
|
|
|
|
||||
Accounts payable, accrued expenses, and other current liabilities
|
$
|
80,625
|
|
|
$
|
53,890
|
|
Deferred reimbursements
|
5,500
|
|
|
7,750
|
|
||
Contingent consideration payable
|
—
|
|
|
8,400
|
|
||
Total current liabilities
|
86,125
|
|
|
70,040
|
|
||
Deferred reimbursements
|
10,156
|
|
|
14,156
|
|
||
Convertible notes
|
175,006
|
|
|
164,167
|
|
||
Senior secured term loan
|
146,734
|
|
|
—
|
|
||
Contingent consideration payable
|
19,700
|
|
|
17,000
|
|
||
Deferred income taxes
|
6,465
|
|
|
6,465
|
|
||
Other non-current liabilities
|
2,853
|
|
|
2,346
|
|
||
Total Liabilities
|
447,039
|
|
|
274,174
|
|
||
Commitments and contingencies
|
|
|
|
||||
Stockholders' equity:
|
|
|
|
||||
Common stock, $.01 par value, 500,000,000 shares authorized, 189,383,924 shares issued and outstanding at December 31, 2018 Common stock, $.01 par value, 250,000,000 shares authorized, 166,989,790 shares issued and outstanding at December 31, 2017
|
1,942
|
|
|
1,721
|
|
||
Additional paid-in capital
|
1,740,061
|
|
|
1,400,758
|
|
||
Accumulated other comprehensive loss:
|
|
|
|
||||
Foreign currency translation adjustment
|
495
|
|
|
(1,659
|
)
|
||
Unrealized loss on available-for securities
|
(427
|
)
|
|
(436
|
)
|
||
Warrants
|
13,063
|
|
|
16,076
|
|
||
Accumulated deficit
|
(1,412,222
|
)
|
|
(1,063,610
|
)
|
||
Total stockholders' equity
|
342,912
|
|
|
352,850
|
|
||
Total Liabilities and Stockholders' Equity
|
$
|
789,951
|
|
|
$
|
627,024
|
|
|
Years Ended December 31,
|
||||||||||
|
2018
|
|
2017
|
|
2016
|
||||||
Revenue:
|
|
|
|
|
|
||||||
Net product sales
|
$
|
91,245
|
|
|
$
|
36,930
|
|
|
$
|
4,958
|
|
Cost of goods sold
|
14,404
|
|
|
6,236
|
|
|
833
|
|
|||
Gross profit
|
76,841
|
|
|
30,694
|
|
|
4,125
|
|
|||
Operating expenses:
|
|
|
|
|
|
||||||
Research and development
|
270,902
|
|
|
149,310
|
|
|
104,793
|
|
|||
Selling, general and administrative
|
127,200
|
|
|
88,671
|
|
|
71,151
|
|
|||
Changes in fair value of contingent consideration payable
|
3,300
|
|
|
(234,322
|
)
|
|
6,760
|
|
|||
Loss on impairment of assets
|
—
|
|
|
465,427
|
|
|
—
|
|
|||
Restructuring charges
|
—
|
|
|
—
|
|
|
69
|
|
|||
Depreciation
|
4,216
|
|
|
3,593
|
|
|
3,242
|
|
|||
Total operating expenses
|
405,618
|
|
|
472,679
|
|
|
186,015
|
|
|||
Loss from operations
|
(328,777
|
)
|
|
(441,985
|
)
|
|
(181,890
|
)
|
|||
Other income (expenses):
|
|
|
|
|
|
||||||
Interest income
|
10,461
|
|
|
4,096
|
|
|
1,602
|
|
|||
Interest expense
|
(22,402
|
)
|
|
(17,240
|
)
|
|
(5,398
|
)
|
|||
Change in fair value of derivatives
|
(2,739
|
)
|
|
—
|
|
|
—
|
|
|||
Loss on extinguishment of debt
|
—
|
|
|
—
|
|
|
(13,302
|
)
|
|||
Other income (expense)
|
(5,632
|
)
|
|
6,008
|
|
|
(4,793
|
)
|
|||
Loss before income tax
|
(349,089
|
)
|
|
(449,121
|
)
|
|
(203,781
|
)
|
|||
Income tax benefit
|
94
|
|
|
165,119
|
|
|
3,739
|
|
|||
Net loss attributable to common stockholders
|
$
|
(348,995
|
)
|
|
$
|
(284,002
|
)
|
|
$
|
(200,042
|
)
|
Net loss attributable to common stockholders per common share — basic and diluted
|
$
|
(1.88
|
)
|
|
$
|
(1.85
|
)
|
|
$
|
(1.49
|
)
|
Weighted-average common shares outstanding — basic and diluted
|
185,790,021
|
|
|
153,355,144
|
|
|
134,401,588
|
|
|
Years Ended December 31,
|
||||||||||
|
2018
|
|
2017
|
|
2016
|
||||||
Net loss
|
$
|
(348,995
|
)
|
|
$
|
(284,002
|
)
|
|
$
|
(200,042
|
)
|
Other comprehensive gain (loss):
|
|
|
|
|
|
||||||
Foreign currency translation adjustment gain (loss), net of tax impact of $0, $0, $0, respectively
|
2,537
|
|
|
(3,604
|
)
|
|
1,945
|
|
|||
Unrealized gain (loss) on available-for-sale securities
|
9
|
|
|
(538
|
)
|
|
217
|
|
|||
Other comprehensive income (loss)
|
2,546
|
|
|
(4,142
|
)
|
|
2,162
|
|
|||
Comprehensive loss
|
$
|
(346,449
|
)
|
|
$
|
(288,144
|
)
|
|
$
|
(197,880
|
)
|
|
Common Stock
|
|
Additional
Paid-In Capital |
|
Warrants
|
|
Other
Comprehensive Gain (Loss) |
|
Accumulated
Deficit |
|
Total
Stockholders' Equity |
|||||||||||||||
|
Shares
|
|
Amount
|
|
|
|
|
|
||||||||||||||||||
Balance at December 31, 2015
|
125,027,034
|
|
|
1,306
|
|
|
917,454
|
|
|
8,755
|
|
|
(115
|
)
|
|
(579,566
|
)
|
|
347,834
|
|
||||||
Stock issued from exercise of stock options, net
|
723,102
|
|
|
7
|
|
|
3,029
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
3,036
|
|
||||||
Stock issued from ATM transactions
|
14,989,027
|
|
|
150
|
|
|
96,918
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
97,068
|
|
||||||
Stock issued for MiaMed acquisition
|
825,603
|
|
|
8
|
|
|
4,599
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
4,607
|
|
||||||
Restricted stock tax vesting
|
268,425
|
|
|
—
|
|
|
(1,282
|
)
|
|
—
|
|
|
—
|
|
|
—
|
|
|
(1,282
|
)
|
||||||
Stock issued for contingent consideration
|
858,795
|
|
|
9
|
|
|
6,106
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
6,115
|
|
||||||
Receivable from investor
|
—
|
|
|
—
|
|
|
932
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
932
|
|
||||||
Warrants issued in debt financing
|
—
|
|
|
—
|
|
|
—
|
|
|
7,321
|
|
|
—
|
|
|
—
|
|
|
7,321
|
|
||||||
Equity component of the Convertible Notes issuance, net of issuance costs of $2,709
|
—
|
|
|
—
|
|
|
88,346
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
88,346
|
|
||||||
Premium paid for Capped Call Confirmations
|
|
|
|
|
(13,450
|
)
|
|
—
|
|
|
|
|
|
|
(13,450
|
)
|
||||||||||
Stock-based compensation
|
—
|
|
|
—
|
|
|
17,504
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
17,504
|
|
||||||
Unrealized holding gain on available-for-sale securities
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
217
|
|
|
—
|
|
|
217
|
|
||||||
Foreign currency translation adjustment
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
1,945
|
|
|
—
|
|
|
1,945
|
|
||||||
Net loss
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
(200,042
|
)
|
|
(200,042
|
)
|
||||||
Balance at December 31, 2016
|
142,691,986
|
|
|
1,480
|
|
|
1,120,156
|
|
|
16,076
|
|
|
2,047
|
|
|
(779,608
|
)
|
|
360,151
|
|
||||||
Stock issued from exercise of stock options, net
|
2,878,681
|
|
|
29
|
|
|
16,272
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
16,301
|
|
||||||
Stock issued from equity financing
|
21,122,449
|
|
|
212
|
|
|
242,825
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
243,037
|
|
||||||
Restricted stock tax vesting
|
296,674
|
|
|
—
|
|
|
(1,596
|
)
|
|
—
|
|
|
—
|
|
|
—
|
|
|
(1,596
|
)
|
||||||
Stock-based compensation
|
—
|
|
|
—
|
|
|
23,101
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
23,101
|
|
||||||
Unrealized holding gain on available-for-sale securities
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
(538
|
)
|
|
—
|
|
|
(538
|
)
|
||||||
Foreign currency translation adjustment
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
(3,604
|
)
|
|
—
|
|
|
(3,604
|
)
|
||||||
Net loss
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
(284,002
|
)
|
|
(284,002
|
)
|
||||||
Balance at December 31, 2017
|
166,989,790
|
|
|
1,721
|
|
|
1,400,758
|
|
|
16,076
|
|
|
(2,095
|
)
|
|
(1,063,610
|
)
|
|
352,850
|
|
||||||
Stock issued from exercise of stock options, net
|
1,397,908
|
|
|
14
|
|
|
9,130
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
9,144
|
|
||||||
Stock issued from equity financing
|
20,239,839
|
|
|
202
|
|
|
294,381
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
294,583
|
|
||||||
Restricted stock tax vesting
|
303,173
|
|
|
—
|
|
|
(2,832
|
)
|
|
—
|
|
|
—
|
|
|
—
|
|
|
(2,832
|
)
|
||||||
Stock-based compensation
|
—
|
|
|
—
|
|
|
29,260
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
29,260
|
|
||||||
Reclassification upon ASU 2018-02 adoption
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
(383
|
)
|
|
383
|
|
|
—
|
|
||||||
Warrants exercised
|
453,214
|
|
|
5
|
|
|
6,625
|
|
|
(3,013
|
)
|
|
—
|
|
|
—
|
|
|
3,617
|
|
||||||
Change in fair value of derivatives
|
—
|
|
|
—
|
|
|
2,739
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
2,739
|
|
||||||
Unrealized holding gain on available-for-sale securities
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
9
|
|
|
—
|
|
|
9
|
|
||||||
Foreign currency translation adjustment
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
2,537
|
|
|
—
|
|
|
2,537
|
|
||||||
Net loss
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
—
|
|
|
(348,995
|
)
|
|
(348,995
|
)
|
||||||
Balance at December 31, 2018
|
189,383,924
|
|
|
$
|
1,942
|
|
|
$
|
1,740,061
|
|
|
$
|
13,063
|
|
|
$
|
68
|
|
|
$
|
(1,412,222
|
)
|
|
$
|
342,912
|
|
|
Years Ended December 31,
|
||||||||||
|
2018
|
|
2017
|
|
2016
|
||||||
Operating activities
|
|
|
|
|
|
||||||
Net loss
|
$
|
(348,995
|
)
|
|
$
|
(284,002
|
)
|
|
$
|
(200,042
|
)
|
Adjustments to reconcile net loss to net cash used in operating activities:
|
|
|
|
|
|
||||||
Amortization of debt discount and deferred financing
|
10,976
|
|
|
9,703
|
|
|
2,689
|
|
|||
Depreciation
|
4,216
|
|
|
3,593
|
|
|
3,242
|
|
|||
Stock-based compensation
|
29,260
|
|
|
23,101
|
|
|
17,504
|
|
|||
Restructuring charges
|
—
|
|
|
—
|
|
|
69
|
|
|||
Change in fair value of derivatives
|
2,739
|
|
|
(265
|
)
|
|
265
|
|
|||
Non-cash changes in the fair value of contingent consideration payable
|
3,300
|
|
|
(234,322
|
)
|
|
6,760
|
|
|||
Charges to research expense for stock issued in asset acquisition
|
—
|
|
|
—
|
|
|
4,607
|
|
|||
Loss on extinguishment of debt
|
—
|
|
|
—
|
|
|
13,302
|
|
|||
Foreign currency remeasurement (gain) loss
|
3,217
|
|
|
(5,620
|
)
|
|
3,660
|
|
|||
Non-cash deferred taxes
|
—
|
|
|
(167,305
|
)
|
|
(3,742
|
)
|
|||
(Gain) loss on disposal of assets
|
59
|
|
|
(8
|
)
|
|
17
|
|
|||
Loss on impairment
|
—
|
|
|
465,427
|
|
|
—
|
|
|||
Changes in operating assets and liabilities:
|
|
|
|
|
|
||||||
Accounts receivable
|
(13,294
|
)
|
|
(7,725
|
)
|
|
(1,419
|
)
|
|||
Inventories
|
(4,205
|
)
|
|
(897
|
)
|
|
(3,651
|
)
|
|||
Prepaid expenses and other current assets
|
2,488
|
|
|
(15,329
|
)
|
|
(394
|
)
|
|||
Other non-current assets
|
(1,039
|
)
|
|
(729
|
)
|
|
(970
|
)
|
|||
Account payable and accrued expenses
|
17,115
|
|
|
12,563
|
|
|
7,131
|
|
|||
Non-current liabilities
|
458
|
|
|
720
|
|
|
825
|
|
|||
Deferred reimbursements
|
(6,250
|
)
|
|
(12,600
|
)
|
|
—
|
|
|||
Net cash used in operating activities
|
(299,955
|
)
|
|
(213,695
|
)
|
|
(150,147
|
)
|
|||
Investing activities
|
|
|
|
|
|
||||||
Sale and redemption of marketable securities
|
463,502
|
|
|
323,753
|
|
|
221,374
|
|
|||
Purchases of marketable securities
|
(578,394
|
)
|
|
(490,468
|
)
|
|
(219,932
|
)
|
|||
Capital expenditures
|
(6,308
|
)
|
|
(4,526
|
)
|
|
(5,951
|
)
|
|||
Net cash used in investing activities
|
(121,200
|
)
|
|
(171,241
|
)
|
|
(4,509
|
)
|
|||
Financing activities
|
|
|
|
|
|
||||||
Proceeds from issuance of common stock and warrants, net of issuance costs
|
294,584
|
|
|
243,037
|
|
|
97,068
|
|
|||
Payments of secured loan agreement
|
—
|
|
|
—
|
|
|
(80,000
|
)
|
|||
Payment of capital leases
|
(334
|
)
|
|
(308
|
)
|
|
(193
|
)
|
|||
Purchase of vested restricted stock units
|
(2,832
|
)
|
|
(1,596
|
)
|
|
(1,282
|
)
|
|||
Proceeds from exercise of stock options
|
9,144
|
|
|
16,301
|
|
|
3,036
|
|
|||
Proceeds from exercise of warrants
|
3,617
|
|
|
—
|
|
|
—
|
|
|||
Payment of contingent consideration
|
—
|
|
|
(10,000
|
)
|
|
(5,000
|
)
|
|||
Proceeds from issuance of convertible notes, net of issuance costs
|
—
|
|
|
—
|
|
|
242,536
|
|
|||
Premiums paid for Capped Call Confirmations
|
—
|
|
|
—
|
|
|
(13,450
|
)
|
|||
Proceeds from loan agreements, net of issuance costs
|
146,596
|
|
|
—
|
|
|
30,000
|
|
|||
Net cash provided by financing activities
|
450,775
|
|
|
247,434
|
|
|
272,715
|
|
Effect of exchange rate changes on cash, cash equivalents and restricted cash
|
1,518
|
|
|
1,326
|
|
|
(131
|
)
|
|||
Net increase (decrease) in cash and cash equivalents and restricted cash
|
31,138
|
|
|
(136,176
|
)
|
|
117,928
|
|
|||
Cash and cash equivalents and restricted cash at beginning of year/period
|
51,237
|
|
|
187,413
|
|
|
69,485
|
|
|||
Cash and cash equivalents and restricted cash at end of year/period
|
$
|
82,375
|
|
|
$
|
51,237
|
|
|
$
|
187,413
|
|
Supplemental disclosures of cash flow information
|
|
|
|
|
|
||||||
Cash paid during the period for interest
|
$
|
7,500
|
|
|
$
|
7,424
|
|
|
$
|
2,990
|
|
Contingent consideration paid in shares
|
$
|
—
|
|
|
$
|
—
|
|
|
$
|
6,115
|
|
Capital expenditures unpaid at the end of period
|
$
|
106
|
|
|
$
|
—
|
|
|
$
|
—
|
|
Capital expenditures funded by capital lease borrowings
|
$
|
208
|
|
|
$
|
—
|
|
|
$
|
944
|
|
|
As of December 31, 2018
|
||||||||||||||
(in thousands)
|
Cost
|
|
Unrealized
Gain
|
|
Unrealized
Loss
|
|
Fair
Value
|
||||||||
Cash and cash equivalents
|
$
|
79,749
|
|
|
$
|
—
|
|
|
$
|
—
|
|
|
$
|
79,749
|
|
Corporate debt securities, current portion
|
240,969
|
|
|
7
|
|
|
(250
|
)
|
|
240,726
|
|
||||
Commercial paper
|
115,245
|
|
|
—
|
|
|
(104
|
)
|
|
115,141
|
|
||||
Asset-backed securities
|
68,215
|
|
|
4
|
|
|
(84
|
)
|
|
68,135
|
|
||||
Money market
|
350
|
|
|
—
|
|
|
—
|
|
|
350
|
|
||||
Certificate of deposit
|
51
|
|
|
—
|
|
|
—
|
|
|
51
|
|
||||
|
$
|
504,579
|
|
|
$
|
11
|
|
|
$
|
(438
|
)
|
|
$
|
504,152
|
|
Included in cash and cash equivalents
|
$
|
79,749
|
|
|
$
|
—
|
|
|
$
|
—
|
|
|
$
|
79,749
|
|
Included in marketable securities, current
|
424,830
|
|
|
11
|
|
|
(438
|
)
|
|
424,403
|
|
||||
Total cash, cash equivalents and marketable securities
|
$
|
504,579
|
|
|
$
|
11
|
|
|
$
|
(438
|
)
|
|
$
|
504,152
|
|
|
As of December 31, 2017
|
||||||||||||||
(in thousands)
|
Cost
|
|
Unrealized
Gain
|
|
Unrealized
Loss
|
|
Fair
Value
|
||||||||
Cash and cash equivalents
|
$
|
49,060
|
|
|
$
|
—
|
|
|
$
|
—
|
|
|
$
|
49,060
|
|
Corporate debt securities, current portion
|
199,314
|
|
|
1
|
|
|
(303
|
)
|
|
199,012
|
|
||||
Commercial paper
|
79,878
|
|
|
—
|
|
|
(75
|
)
|
|
79,803
|
|
||||
Asset-backed securities
|
30,346
|
|
|
—
|
|
|
(59
|
)
|
|
30,287
|
|
||||
Money market
|
350
|
|
|
—
|
|
|
—
|
|
|
350
|
|
||||
Certificate of deposit
|
50
|
|
|
—
|
|
|
—
|
|
|
50
|
|
||||
|
$
|
358,998
|
|
|
$
|
1
|
|
|
$
|
(437
|
)
|
|
$
|
358,562
|
|
Included in cash and cash equivalents
|
$
|
49,060
|
|
|
$
|
—
|
|
|
$
|
—
|
|
|
$
|
49,060
|
|
Included in marketable securities, current
|
309,938
|
|
|
1
|
|
|
(437
|
)
|
|
309,502
|
|
||||
Total cash, cash equivalents and marketable securities
|
$
|
358,998
|
|
|
$
|
1
|
|
|
$
|
(437
|
)
|
|
$
|
358,562
|
|
(in thousands)
|
|
December 31, 2018
|
|
December 31, 2017
|
|
December 31, 2016
|
||||||
Cash and cash equivalents
|
|
$
|
79,749
|
|
|
$
|
49,060
|
|
|
$
|
187,026
|
|
Restricted cash
|
|
2,626
|
|
|
2,177
|
|
|
387
|
|
|||
Cash and cash equivalents and restricted cash shown in the statement of cash flows
|
|
$
|
82,375
|
|
|
$
|
51,237
|
|
|
$
|
187,413
|
|
(in thousands)
|
December 31, 2018
|
|
December 31, 2017
|
||||
Raw materials
|
$
|
1,291
|
|
|
$
|
2,393
|
|
Work-in-process
|
3,485
|
|
|
1,450
|
|
||
Finished goods
|
3,614
|
|
|
780
|
|
||
Total inventories
|
$
|
8,390
|
|
|
$
|
4,623
|
|
|
December 31,
|
||||||
(in thousands)
|
2018
|
|
2017
|
||||
Property and equipment consist of the following:
|
|
|
|
||||
Computer equipment
|
$
|
4,691
|
|
|
$
|
3,746
|
|
Computer software
|
1,298
|
|
|
1,236
|
|
||
Research equipment
|
8,445
|
|
|
6,379
|
|
||
Furniture and fixtures
|
4,876
|
|
|
2,992
|
|
||
Leasehold improvements
|
7,425
|
|
|
7,193
|
|
||
Vehicles
|
209
|
|
|
—
|
|
||
Construction in progress
|
102
|
|
|
31
|
|
||
Gross property and equipment
|
27,046
|
|
|
21,577
|
|
||
Less accumulated depreciation
|
(15,671
|
)
|
|
(12,515
|
)
|
||
Net property and equipment
|
$
|
11,375
|
|
|
$
|
9,062
|
|
|
December 31,
|
||||||
(in thousands)
|
2018
|
|
2017
|
||||
Accounts payable
|
$
|
6,606
|
|
|
$
|
7,867
|
|
Accrued professional fees
|
2,276
|
|
|
5,845
|
|
||
Accrued contract manufacturing & contract research costs
|
5,890
|
|
|
4,632
|
|
||
Accrued compensation and benefits
|
21,731
|
|
|
19,620
|
|
||
Accrued facility costs
|
2,102
|
|
|
1,665
|
|
||
Accrued program fees
|
16,674
|
|
|
5,707
|
|
||
Royalties payable
|
4,463
|
|
|
2,529
|
|
||
Accrued interest
|
4,189
|
|
|
313
|
|
||
Milestone payments
|
9,000
|
|
|
—
|
|
||
Accrued sales rebates and discounts
|
3,636
|
|
|
1,957
|
|
||
Other
|
4,058
|
|
|
3,755
|
|
||
|
$
|
80,625
|
|
|
$
|
53,890
|
|
|
Years Ended
December 31,
|
||||||||||
|
2018
|
|
2017
|
|
2016
|
||||||
Expected stock price volatility
|
78.6
|
%
|
|
82.8
|
%
|
|
81.3
|
%
|
|||
Risk free interest rate
|
2.4
|
%
|
|
2.0
|
%
|
|
1.5
|
%
|
|||
Expected life of options (years)
|
5.62
|
|
|
6.18
|
|
|
6.25
|
|
|||
Expected annual dividend per share
|
$
|
0.00
|
|
|
$
|
0.00
|
|
|
$
|
0.00
|
|
|
Number of
Shares
|
|
Weighted
Average
Exercise
Price
|
|
Weighted
Average
Remaining
Contractual
Life
|
|
Aggregate
Intrinsic
Value
|
|||||
|
(in thousands)
|
|
|
|
|
|
(in millions)
|
|||||
Options outstanding, December 31, 2015
|
11,729.2
|
|
|
$
|
7.11
|
|
|
|
|
|
||
Granted
|
5,114.1
|
|
|
$
|
7.67
|
|
|
|
|
|
||
Exercised
|
(723.1
|
)
|
|
$
|
4.20
|
|
|
|
|
|
||
Forfeited
|
(622.7
|
)
|
|
$
|
8.62
|
|
|
|
|
|
||
Options outstanding, December 31, 2016
|
15,497.5
|
|
|
$
|
7.37
|
|
|
|
|
|
||
Granted
|
3,695.3
|
|
|
$
|
7.17
|
|
|
|
|
|
||
Exercised
|
(2,878.7
|
)
|
|
$
|
5.67
|
|
|
|
|
|
||
Forfeited
|
(1,133.0
|
)
|
|
$
|
9.55
|
|
|
|
|
|
||
Options outstanding, December 31, 2017
|
15,181.1
|
|
|
$
|
7.48
|
|
|
|
|
|
||
Granted
|
2,348.0
|
|
|
$
|
14.96
|
|
|
|
|
|
||
Exercised
|
(1,398.0
|
)
|
|
$
|
6.54
|
|
|
|
|
|
||
Forfeited
|
(313.1
|
)
|
|
$
|
9.55
|
|
|
|
|
|
||
Expired
|
(8.0
|
)
|
|
10.76
|
|
|
|
|
||||
Options outstanding, December 31, 2018
|
15,810.0
|
|
|
$
|
8.63
|
|
|
6.7 years
|
|
$
|
37.6
|
|
Vested and unvested expected to vest, December 31, 2018
|
15,152.6
|
|
|
$
|
8.51
|
|
|
6.6 years
|
|
$
|
36.8
|
|
Exercisable at December 31, 2018
|
9,977.0
|
|
|
$
|
7.43
|
|
|
5.8 years
|
|
$
|
28.8
|
|
|
Number of
Share
|
|
Weighted
Average Grant
Date Fair
Value
|
|
Weighted
Average
Remaining
Years
|
|
Aggregate
Intrinsic
Value
|
|||||
|
(in thousands)
|
|
|
|
|
|
(in millions)
|
|||||
Non-vested units as of December 31, 2016
|
744.4
|
|
|
$
|
7.86
|
|
|
|
|
|
||
Granted
|
2,348.7
|
|
|
$
|
5.69
|
|
|
|
|
|
||
Vested
|
(318.2
|
)
|
|
$
|
9.23
|
|
|
|
|
|
||
Forfeited
|
(199.8
|
)
|
|
$
|
6.24
|
|
|
|
|
|
||
Non-vested units as of December 31, 2017
|
2,575.1
|
|
|
$
|
5.85
|
|
|
|
|
|
||
Granted
|
1,811.9
|
|
|
$
|
16.11
|
|
|
|
|
|
||
Vested
|
(530.0
|
)
|
|
$
|
6.01
|
|
|
|
|
|
||
Forfeited
|
(145.0
|
)
|
|
$
|
9.65
|
|
|
|
|
|
||
Non-vested units as of December 31, 2018
|
3,712.0
|
|
|
$
|
10.59
|
|
|
2.57
|
|
$
|
36.3
|
|
|
Years Ended December 31,
|
||||||||||
|
2018
|
|
2017
|
|
2016
|
||||||
Equity compensation expense recognized in:
|
|
|
|
|
|
||||||
Research and development expense
|
$
|
11,740
|
|
|
$
|
10,328
|
|
|
$
|
8,071
|
|
Selling, general and administrative expense
|
17,520
|
|
|
12,773
|
|
|
9,433
|
|
|||
Total equity compensation expense
|
$
|
29,260
|
|
|
$
|
23,101
|
|
|
$
|
17,504
|
|
(in thousands)
|
Level 2
|
|
Total
|
||||
Assets:
|
|
|
|
||||
Commercial paper
|
$
|
115,141
|
|
|
$
|
115,141
|
|
Asset-back securities
|
68,135
|
|
|
68,135
|
|
||
Corporate debt securities
|
240,726
|
|
|
240,726
|
|
||
Money market funds
|
3,082
|
|
|
3,082
|
|
||
|
$
|
427,084
|
|
|
$
|
427,084
|
|
|
Level 2
|
|
Level 3
|
|
Total
|
||||||
Liabilities:
|
|
|
|
|
|
||||||
Contingent consideration payable
|
$
|
—
|
|
|
$
|
19,700
|
|
|
$
|
19,700
|
|
Deferred compensation plan liability
|
2,732
|
|
|
—
|
|
|
2,732
|
|
|||
|
$
|
2,732
|
|
|
$
|
19,700
|
|
|
$
|
22,432
|
|
(in thousands)
|
Level 2
|
|
Total
|
||||
Assets:
|
|
|
|
||||
Commercial paper
|
$
|
79,803
|
|
|
$
|
79,803
|
|
Asset-back securities
|
30,287
|
|
|
30,287
|
|
||
Corporate debt securities
|
199,012
|
|
|
199,012
|
|
||
Money market funds
|
2,598
|
|
|
2,598
|
|
||
|
$
|
311,700
|
|
|
$
|
311,700
|
|
|
Level 2
|
|
Level 3
|
|
Total
|
||||||
Liabilities:
|
|
|
|
|
|
||||||
Contingent consideration payable
|
$
|
—
|
|
|
$
|
25,400
|
|
|
$
|
25,400
|
|
Deferred compensation plan liability
|
2,258
|
|
|
—
|
|
|
2,258
|
|
|||
|
$
|
2,258
|
|
|
$
|
25,400
|
|
|
$
|
27,658
|
|
Contingent Consideration Liability
|
|
Fair value as of
December 31, 2018 |
|
Valuation Technique
|
|
Unobservable Input
|
|
Range
|
|
|
|
|
|
|
Discount rate
|
|
10%
|
Clinical and regulatory milestones
|
|
$19.2 million
|
|
Probability weighted discounted cash flow
|
|
Probability of achievement of milestones
|
|
71.0% - 100.0%
|
|
|
|
|
|
|
Projected year of payments
|
|
2021 - 2022
|
|
Year ended December 31,
|
||||||
(in thousands)
|
2018
|
|
2017
|
||||
Balance, beginning of the period
|
$
|
25,400
|
|
|
$
|
269,722
|
|
Payment of contingent consideration in cash
|
—
|
|
|
(10,000
|
)
|
||
Milestone payable, included in accrued expenses
|
(9,000
|
)
|
|
—
|
|
||
Unrealized change in fair value change during the period, included in Statement of Operations
|
3,300
|
|
|
(234,322
|
)
|
||
Balance, end of the period
|
$
|
19,700
|
|
|
$
|
25,400
|
|
Liability component (in thousands)
|
2018
|
|
2017
|
||||
Principal
|
$
|
400,000
|
|
|
$
|
250,000
|
|
Less: debt discount
(1)
|
(74,145
|
)
|
|
(81,566
|
)
|
||
Less: deferred financing
(1)
|
(4,115
|
)
|
|
(4,267
|
)
|
||
Net carrying value of the debt
|
$
|
321,740
|
|
|
$
|
164,167
|
|
(1)
|
Included in the consolidated balance sheets within convertible notes and senior secured term loan and amortized to interest expense over the remaining life of the Convertible Notes and Senior Secured Term Loan using the effective interest rate method.
|
Components (in thousands)
|
2018
|
|
2017
|
||||
Contractual interest expense
|
$
|
11,426
|
|
|
$
|
7,528
|
|
Amortization of deferred financing
|
555
|
|
|
470
|
|
||
Amortization of debt discount
|
10,421
|
|
|
9,241
|
|
||
Total
|
$
|
22,402
|
|
|
$
|
17,239
|
|
Effective interest rate of the liability component, convertible debt
|
10.85
|
%
|
|
10.85
|
%
|
||
Effective interest rate of the liability component, senior secured term loan
|
10.48
|
%
|
|
—
|
%
|
Location
|
Approximate
Square Feet
|
|
Use
|
|
Lease expiry date
|
|
Cranbury, New Jersey
|
90,000
|
|
|
Office and laboratory
|
|
September 2025
|
United Kingdom
|
46,617
|
|
|
Office
|
|
August 2028
|
Princeton, New Jersey
|
21,922
|
|
|
Office
|
|
January 2022
|
(in thousands)
|
2019
|
|
2020
|
|
2021
|
|
2022
|
|
2023 and
beyond |
|
Total
|
||||||||||||
Minimum lease payments
|
$
|
6,244
|
|
|
$
|
4,063
|
|
|
$
|
3,560
|
|
|
$
|
3,371
|
|
|
$
|
13,649
|
|
|
$
|
30,887
|
|
Years ending December 31:
|
|
||
2019
|
$
|
194
|
|
2020
|
60
|
|
|
2021
|
47
|
|
|
2022
|
30
|
|
|
2023 and beyond
|
—
|
|
|
Total principal obligation
|
$
|
331
|
|
(in thousands)
|
2018
|
|
2017
|
|
2016
|
||||||
Current
|
|
|
|
|
|
||||||
Federal
|
$
|
—
|
|
|
$
|
—
|
|
|
$
|
—
|
|
State
|
6
|
|
|
9
|
|
|
7
|
|
|||
Foreign
|
(100
|
)
|
|
2,276
|
|
|
—
|
|
|||
Deferred
|
|
|
|
|
|
||||||
Federal
|
—
|
|
|
(150,015
|
)
|
|
(1,101
|
)
|
|||
State
|
—
|
|
|
(17,389
|
)
|
|
(2,645
|
)
|
|||
Foreign
|
—
|
|
|
—
|
|
|
—
|
|
|||
Total
|
$
|
(94
|
)
|
|
$
|
(165,119
|
)
|
|
$
|
(3,739
|
)
|
|
Years Ended
December 31,
|
|||||||
|
2018
|
|
2017
|
|
2016
|
|||
Statutory rate
|
(21
|
)%
|
|
(34
|
)%
|
|
(34
|
)%
|
State taxes, net of federal benefit
|
(4
|
)
|
|
(5
|
)
|
|
(5
|
)
|
Nondeductible IPR&D
|
6
|
|
|
(1
|
)
|
|
3
|
|
Contingent consideration
|
1
|
|
|
(18
|
)
|
|
—
|
|
Tax credits
|
(10
|
)
|
|
(2
|
)
|
|
(3
|
)
|
Foreign income tax rate differential
|
2
|
|
|
5
|
|
|
2
|
|
Impact of 2017 Act
|
—
|
|
|
27
|
|
|
—
|
|
Other
|
—
|
|
|
5
|
|
|
(1
|
)
|
Valuation allowance
|
26
|
|
|
(14
|
)
|
|
36
|
|
Net
|
—
|
%
|
|
(37
|
)%
|
|
(2
|
)%
|
|
For Years Ended
December 31,
|
||||||
(in thousands)
|
2018
|
|
2017
|
||||
Deferred tax assets
|
|
|
|
||||
Intellectual property
|
$
|
48,339
|
|
|
$
|
44,573
|
|
Amortization/depreciation
|
3,732
|
|
|
3,082
|
|
||
Research tax credit
|
96,509
|
|
|
43,382
|
|
||
Net operating loss carry forwards
|
248,398
|
|
|
221,912
|
|
||
Deferred revenue
|
4,401
|
|
|
6,158
|
|
||
Non-cash stock issue
|
16,850
|
|
|
10,751
|
|
||
Interest carry forward limitation
|
1,032
|
|
|
—
|
|
||
Others
|
10,852
|
|
|
7,328
|
|
||
Gross deferred tax assets
|
430,113
|
|
|
337,186
|
|
||
Deferred tax liabilities
|
|
|
|
||||
Business acquisition
|
(6,465
|
)
|
|
(6,465
|
)
|
||
Royalty payable
|
(48,339
|
)
|
|
(44,573
|
)
|
||
Convertible notes
|
(16,666
|
)
|
|
(18,991
|
)
|
||
Advanced R&D payments
|
(2,103
|
)
|
|
(3,069
|
)
|
||
Total net deferred tax assets
|
356,540
|
|
|
264,088
|
|
||
Less: valuation allowance
|
(363,005
|
)
|
|
(270,553
|
)
|
||
Net deferred tax liability
|
$
|
(6,465
|
)
|
|
$
|
(6,465
|
)
|
•
|
a payment for an identifiable benefit, and
|
•
|
the identifiable benefit is separable from the existing relationship between the Company and GSK, and
|
•
|
the identifiable benefit can be obtained from a party other than GSK, and
|
•
|
the Company can reasonably estimate the fair value of the identifiable benefit,
|
(in thousands, except per share amounts)
|
Years Ended December 31,
|
||||||||||
Historical
|
2018
|
|
2017
|
|
2016
|
||||||
Numerator:
|
|
|
|
|
|
||||||
Net loss attributable to common stockholders
|
$
|
(348,995
|
)
|
|
$
|
(284,002
|
)
|
|
$
|
(200,042
|
)
|
Denominator:
|
|
|
|
|
|
||||||
Weighted average common shares outstanding — basic and diluted
|
185,790,021
|
|
|
153,355,144
|
|
|
134,401,588
|
|
|
Year ended December 31,
|
|||||||
(in thousands)
|
2018
|
|
2017
|
|
2016
|
|||
Options to purchase common stock
|
15,810
|
|
|
15,181
|
|
|
15,528
|
|
Convertible debt
|
40,850
|
|
|
40,850
|
|
|
40,850
|
|
Outstanding warrants, convertible to common stock
|
2,657
|
|
|
3,110
|
|
|
3,110
|
|
Unvested restricted stock units
|
3,712
|
|
|
2,575
|
|
|
744
|
|
Vested restricted stock units, unissued
|
91
|
|
|
50
|
|
|
—
|
|
Total number of potentially issuable shares
|
63,120
|
|
|
61,766
|
|
|
60,232
|
|
|
Quarters Ended
|
||||||||||||||
|
March 31
|
|
June 30
|
|
September 30
|
|
December 31
|
||||||||
2018
|
|
|
|
|
|
|
|
||||||||
Net loss
|
$
|
(49,916
|
)
|
|
$
|
(61,833
|
)
|
|
$
|
(159,163
|
)
|
|
$
|
(78,083
|
)
|
Basic and diluted net loss per common share (1)
|
$
|
(0.28
|
)
|
|
$
|
(0.33
|
)
|
|
$
|
(0.84
|
)
|
|
$
|
(0.43
|
)
|
2017
|
|
|
|
|
|
|
|
||||||||
Net loss
|
$
|
(54,992
|
)
|
|
$
|
(48,136
|
)
|
|
$
|
(111,666
|
)
|
|
$
|
(69,208
|
)
|
Basic and diluted net loss per common share (1)
|
(0.39
|
)
|
|
(0.34
|
)
|
|
(0.69
|
)
|
|
(0.42
|
)
|
(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 issuing shares of its common stock during the year.
|
(a)
|
1.
Consolidated Financial Statements
|
|
|
|
|
Incorporated by Reference
to SEC Filing
|
|
|
|||||
Exhibit
No.
|
|
Filed Exhibit Description
|
|
Form
|
|
Date
|
|
Exhibit No.
|
|
Filed with this
Form 10-K
|
|
2.1
|
|
|
Form 8-K
|
|
2/12/2014
|
|
2.1
|
|
|
|
|
2.2
|
|
|
Form 8-K
|
|
9/30/2015
|
|
2.2
|
|
|
|
|
+2.3
|
|
|
Form 8-K
|
|
7/6/2016
|
|
2.1
|
|
|
|
|
+2.4
|
|
|
Form 8-K
|
|
9/25/2018
|
|
2.1
|
|
|
|
|
3.1
|
|
|
Form 10-K
|
|
2/28/2012
|
|
3.1
|
|
|
|
|
3.2
|
|
|
S-1/A (333-141700)
|
|
4/27/2007
|
|
3.4
|
|
|
|
|
3.3
|
|
|
Form 8-K
|
|
6/10/2015
|
|
3.1
|
|
|
|
|
3.4
|
|
|
Form 8-K
|
|
6/8/2018
|
|
3.1
|
|
|
|
|
4.1
|
|
|
S-1 (333-141700)
|
|
3/30/2007
|
|
4.1
|
|
|
|
|
4.2
|
|
|
S-1 (333-141700)
|
|
3/30/2007
|
|
4.2
|
|
|
|
|
4.3
|
|
|
Form 8-K
|
|
10/1/2015
|
|
4.1
|
|
|
|
|
4.4
|
|
|
Form 8-K
|
|
2/22/2016
|
|
4.1
|
|
|
|
|
|
|
|
Incorporated by Reference
to SEC Filing
|
|
|
|||||
Exhibit
No.
|
|
Filed Exhibit Description
|
|
Form
|
|
Date
|
|
Exhibit No.
|
|
Filed with this
Form 10-K
|
|
4.5
|
|
|
Form 8-K
|
|
7/1/2016
|
|
4.1
|
|
|
|
|
4.6
|
|
|
Form S-3ASR
|
|
4/29/2016
|
|
4.7
|
|
|
|
|
4.7
|
|
|
Form 8-K
|
|
12/21/2016
|
|
4.1
|
|
|
|
|
*10.1
|
|
|
S-1/A (333-141700)
|
|
4/27/2007
|
|
10.1
|
|
|
|
|
+10.2
|
|
|
Form 10-K
|
|
2/6/2009
|
|
10.3
|
|
|
|
|
+10.3
|
|
|
S-1 (333-141700)
|
|
3/30/2007
|
|
10.5
|
|
|
|
|
10.4
|
|
|
S-1 (333-141700)
|
|
3/30/2007
|
|
10.17
|
|
|
|
|
*10.5
|
|
|
Form 8-K
|
|
6/18/2010
|
|
10.2
|
|
|
|
|
*10.6
|
|
|
S-1/A (333-141700)
|
|
5/17/2007
|
|
10.24
|
|
|
|
|
*10.7
|
|
|
Form 8-K
|
|
6/9/2016
|
|
10.1
|
|
|
|
|
10.8
|
|
|
Form 8-K
|
|
8/16/2011
|
|
10.1
|
|
|
|
|
|
|
|
Incorporated by Reference
to SEC Filing
|
|
|
|||||
Exhibit
No.
|
|
Filed Exhibit Description
|
|
Form
|
|
Date
|
|
Exhibit No.
|
|
Filed with this
Form 10-K
|
|
10.9
|
|
|
Form 8-K
|
|
11/20/2013
|
|
10.1
|
|
|
|
|
10.10
|
|
|
Form 8-K
|
|
12/30/2013
|
|
10.1
|
|
|
|
|
+10.11
|
|
|
Form 10-K
|
|
3/3/2014
|
|
10.46
|
|
|
|
|
*10.12
|
|
|
Form 8-K
|
|
7/2/2014
|
|
10.1
|
|
|
|
|
10.13
|
|
|
Form 8-K
|
|
10/16/2014
|
|
10.1
|
|
|
|
|
*10.14
|
|
|
Form 8-K
|
|
4/25/2014
|
|
10.1
|
|
|
|
|
*10.15
|
|
|
Form 8-K
|
|
4/25/2014
|
|
10.2
|
|
|
|
|
*10.16
|
|
|
Form 8-K
|
|
4/25/2014
|
|
10.3
|
|
|
|
|
*10.17
|
|
|
Form 10-Q
|
|
5/5/2014
|
|
10.6
|
|
|
|
|
*10.18
|
|
|
Form 10-Q
|
|
5/5/2014
|
|
10.8
|
|
|
|
|
*10.19
|
|
|
Form 8-K
|
|
6/13/2016
|
|
10.1
|
|
|
|
|
*10.20
|
|
|
Form 8-K
|
|
10/28/2016
|
|
10.1
|
|
|
|
|
*10.21
|
|
|
Form 10-K
|
|
2/29/2016
|
|
10.37
|
|
|
|
|
|
|
|
Incorporated by Reference
to SEC Filing
|
|
|
|||||
Exhibit
No.
|
|
Filed Exhibit Description
|
|
Form
|
|
Date
|
|
Exhibit No.
|
|
Filed with this
Form 10-K
|
|
*10.22
|
|
|
Form 8-K
|
|
10/16/2014
|
|
10.1
|
|
|
|
|
10.23
|
|
|
Form 8-K
|
|
4/28/2015
|
|
10.1
|
|
|
|
|
10.24
|
|
|
Form 8-K
|
|
10/1/2015
|
|
10.1
|
|
|
|
|
10.25
|
|
|
Form 8-K
|
|
9/14/2015
|
|
10.1
|
|
|
|
|
*10.26
|
|
|
Form 8-K
|
|
3/15/2016
|
|
10.1
|
|
|
|
|
10.27
|
|
|
Form 8-K
|
|
2/22/2016
|
|
10.1
|
|
|
|
|
10.28
|
|
|
Form 8-K
|
|
7/1/2016
|
|
10.2
|
|
|
|
|
*10.29
|
|
|
Form 8-K
|
|
7/29/2016
|
|
10.1
|
|
|
|
|
|
|
|
Incorporated by Reference
to SEC Filing
|
|
|
|||||
Exhibit
No.
|
|
Filed Exhibit Description
|
|
Form
|
|
Date
|
|
Exhibit No.
|
|
Filed with this
Form 10-K
|
|
*10.30
|
|
|
Form 8-K
|
|
8/23/2016
|
|
10.1
|
|
|
|
|
10.31
|
|
|
Form 8-K
|
|
12/21/2016
|
|
10.1
|
|
|
|
|
10.32
|
|
|
Form 8-K
|
|
12/21/2016
|
|
10.2
|
|
|
|
|
10.33
|
|
|
Form 8-K
|
|
12/21/2016
|
|
10.3
|
|
|
|
|
10.34
|
|
|
Form 8-K
|
|
12/21/2016
|
|
10.4
|
|
|
|
|
10.35
|
|
|
Form 8-K
|
|
12/21/2016
|
|
10.5
|
|
|
|
|
10.36
|
|
|
Form 8-K
|
|
12/21/2016
|
|
10.6
|
|
|
|
|
10.37
|
|
|
Form 8-K
|
|
12/21/2016
|
|
10.7
|
|
|
|
|
10.38
|
|
|
Form 8-K
|
|
12/21/2016
|
|
10.8
|
|
|
|
|
10.39
|
|
|
Form 8-K
|
|
12/21/2016
|
|
10.9
|
|
|
|
|
|
|
|
Incorporated by Reference
to SEC Filing
|
|
|
|||||
Exhibit
No.
|
|
Filed Exhibit Description
|
|
Form
|
|
Date
|
|
Exhibit No.
|
|
Filed with this
Form 10-K
|
|
10.40
|
|
|
Form 8-K
|
|
12/21/2016
|
|
10.10
|
|
|
|
|
10.41
|
|
|
Form 8-K
|
|
12/21/2016
|
|
10.11
|
|
|
|
|
10.42
|
|
|
Form 8-K
|
|
12/21/2016
|
|
10.12
|
|
|
|
|
*10.43
|
|
|
Form 8-K
|
|
12/30/2016
|
|
10.1
|
|
|
|
|
10.44
|
|
|
Form 8-K
|
|
9/25/18
|
|
10.1
|
|
|
|
|
10.45
|
|
|
Form 8-K
|
|
1/24/19
|
|
10.1
|
|
|
|
|
10.46
|
|
|
Form 8-K
|
|
2/8/19
|
|
10.1
|
|
|
|
|
10.47
|
|
|
Form 8-K
|
|
12/26/18
|
|
10.1
|
|
|
|
|
++10.48
|
|
|
|
|
|
|
|
|
X
|
||
10.49
|
|
|
|
|
|
|
|
|
X
|
||
21
|
|
|
|
|
|
|
|
|
|
X
|
|
23.1
|
|
|
|
|
|
|
|
|
|
X
|
|
31.1
|
|
|
|
|
|
|
|
|
|
X
|
|
31.2
|
|
|
|
|
|
|
|
|
|
X
|
|
32.1
|
|
|
|
|
|
|
|
|
|
X
|
|
32.2
|
|
|
|
|
|
|
|
|
|
X
|
|
|
|
|
Incorporated by Reference
to SEC Filing
|
|
|
||||
Exhibit
No.
|
|
Filed Exhibit Description
|
|
Form
|
|
Date
|
|
Exhibit No.
|
|
Filed with this
Form 10-K
|
101
|
|
The following financial information from this Annual Report on Form 10-K for the year ended December 31, 2018, formatted in XBRL (Extensible Business Reporting Language) and filed electronically herewith: (i) the Consolidated Balance Sheets; (ii) the Consolidated Statements of Operations; (iii) the Consolidated Statements of Comprehensive Loss; (iv) the Consolidated Statements of Cash Flows; (v) and the Notes to the Consolidated Financial Statements.
|
|
|
|
|
|
|
|
X
|
+
|
Confidential treatment has been granted as to certain portions of the document, which portions have been omitted and filed separately with the Securities and Exchange Commission.
|
++
|
Subject to confidential treatment request.
|
*
|
Indicates management contract or compensatory plan.
|
AMICUS THERAPEUTICS, INC.
(Registrant)
|
|
By:
|
/s/ John F. Crowley
|
|
John F. Crowley
Chief Executive Officer
|
Signature
|
|
Title
|
|
Date
|
|
|
|
|
|
/s/ John F. Crowley
|
|
Chairman and Chief Executive Officer
(Principal Executive Officer)
|
|
February 28, 2019
|
(John F. Crowley)
|
|
|
|
|
|
|
|
|
|
/s/ Daphne Quimi
|
|
Chief Financial Officer
(Principal Financial Officer) |
|
February 28, 2019
|
(Daphne Quimi)
|
|
|
|
|
|
|
|
|
|
/s/ Samantha Prout
|
|
Global Controller
(Principal Accounting Officer) |
|
February 28, 2019
|
(Samantha Prout)
|
|
|
|
|
|
|
|
|
|
/s/ Robert Essner
|
|
Director
|
|
February 28, 2019
|
(Robert Essner)
|
|
|
|
|
|
|
|
|
|
/s/ Ted W. Love, M.D.
|
|
Director
|
|
February 28, 2019
|
(Ted W. Love, M.D.)
|
|
|
|
|
|
|
|
|
|
/s/ Margaret G. McGlynn, R.Ph.
|
|
Director
|
|
February 28, 2019
|
(Margaret G. McGlynn, R.Ph.)
|
|
|
|
Signature
|
|
Title
|
|
Date
|
|
|
|
|
|
/s/ Michael G. Raab
|
|
Director
|
|
February 28, 2019
|
(Michael G. Raab)
|
|
|
||
|
|
|
|
|
/s/ Glenn Sblendorio
|
|
Director
|
|
February 28, 2019
|
(Glenn Sblendorio)
|
|
|
||
|
|
|
|
|
/s/ Craig Wheeler
|
|
Director
|
|
February 28, 2019
|
(Craig Wheeler)
|
|
|
||
|
|
|
|
|
/s/ Lynn Bleil
|
|
Director
|
|
February 28, 2019
|
(Lynn Bleil)
|
|
|
1 Year Amicus Therapeutics Chart |
1 Month Amicus Therapeutics Chart |
It looks like you are not logged in. Click the button below to log in and keep track of your recent history.
Support: +44 (0) 203 8794 460 | support@advfn.com
By accessing the services available at ADVFN you are agreeing to be bound by ADVFN's Terms & Conditions