Investing in our common stock involves a high degree of risk. You
should carefully consider the risks described below, as well as the other information in this Quarterly Report on Form
10-Q,
or Quarterly Report, and in our other public filings before making an investment
decision. Our business, prospects, financial condition, or operating results could be harmed by any of these risks, as well as other risks not currently known to us or that we currently consider immaterial. If any such risks or uncertainties
actually occur, our business, financial condition or operating results could differ materially from the plans, projections and other forward-looking statements included in the section titled Managements Discussion and Analysis of
Financial Condition and Results of Operations and elsewhere in this Quarterly Report and in our other public filings. The trading price of our common stock could decline due to any of these risks, and as a result, you may lose all or part of
your investment.
Risks Related to Product Development, Regulatory Approval and Commercialization
We currently depend almost entirely on the success of one product candidate,
ZGN-1061,
which recently completed
Phase 1 clinical development. We cannot be certain that we will be able to obtain regulatory approval for
ZGN-1061,
or successfully commercialize
ZGN-1061
if approved.
We currently have only one product candidate,
ZGN-1061,
which recently completed Phase 1
clinical development in the Netherlands, and our business currently depends almost entirely on its successful clinical development, regulatory approval and commercialization. We currently have no drug products for sale and may never be able to
develop marketable drug products. In order to conduct clinical trials in the United States we need to file an Investigational New Drug Application, or IND with the U.S. Food and Drug Administration, or FDA. Because our business is almost entirely
dependent upon this one product candidate, any setback in our pursuit of regulatory approval for
ZGN-1061
would have a material adverse effect on our business and prospects. The clinical trials of our product
candidates are, and the manufacturing and marketing of our product candidates will be, subject to extensive and rigorous review and regulation by numerous government authorities in the United States and in other countries where we intend to test
and, if approved, market any product candidate. Before obtaining regulatory approvals for the commercial sale of any product candidate, we must demonstrate through
pre-clinical
testing and clinical trials that
the product candidate is safe and effective for use in each target indication. This process can take many years and will likely include post-marketing studies, or PMS, post-marketing requirements, or PMRs, and surveillance such as Risk Evaluation
and Mitigation Strategies, or REMS, which will require the expenditure of substantial resources beyond the proceeds we currently have on hand.
Furthermore, we are not permitted to market
ZGN-1061
in the United States until we receive approval of
a New Drug Application, or NDA, from the FDA, or in any foreign countries until we receive the requisite marketing approval from such countries. Development of diabetes drugs requires approximately 2,500 subjects randomized to active doses of the
product with 1,300 to 1,500 subjects exposed for a year and 300 to 500 subjects exposed for 18 months in order to estimate the safety of the drug in an NDA. In addition, it is anticipated that the FDA may require that their guidance for assessment
of cardiovascular risk with diabetes products be followed which may require testing of 5,000 to 10,000 subjects. Pursuant to the FDAs draft guidance documents to industry related to the development of weight management drugs, in order to
reasonably estimate the safety of a weight-management drug in an NDA, Phase 3 clinical trials must randomize approximately 3,000 subjects to active doses of the product and 1,500 subjects to placebo in clinical trials of
one-year
duration. Meeting the requirements of the FDA or certain European regulatory authorities may require that we conduct additional pivotal clinical trials. Accordingly, obtaining approval of an NDA or
Marketing Authorization Application, or MAA, is a complex, lengthy, expensive and uncertain process.
The FDA and certain European
regulatory authorities may delay, limit or deny approval of
ZGN-1061
for many reasons, including, among others:
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the FDA may not accept our IND for
ZGN-1061
or may put it on clinical hold;
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we may not be able to demonstrate that
ZGN-1061
is safe and effective to the satisfaction of the FDA and the European Medicines Agency, or EMA;
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the results of our clinical trials may not meet the level of statistical or clinical significance required by the FDA and EMA for marketing approval;
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the FDA and EMA may disagree with the number, design, size, duration, conduct or implementation of our clinical trials;
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the FDA and EMA may require that we conduct additional clinical trials or
pre-clinical
studies;
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the FDA and EMA may not approve the formulation, labeling or specifications of
ZGN-1061;
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the contract research organizations, or CROs, that we retain to conduct our clinical trials may take actions outside of our control that materially adversely impact our clinical trials;
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the FDA and EMA may find the data from
pre-clinical
studies and clinical trials insufficient to demonstrate that
ZGN-1061s
clinical
and other benefits outweigh its safety risks;
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the FDA and EMA may disagree with our interpretation of data from our
pre-clinical
studies and clinical trials;
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the FDA and EMA may not accept data generated at our clinical trial sites;
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if and when our NDA is submitted and reviewed by an FDA advisory committee, the FDA may have difficulties scheduling an advisory committee meeting in a timely manner or the advisory committee may recommend against
approval of our application or may recommend that the FDA require, as a condition of approval, additional
pre-clinical
studies or clinical trials, limitations on approved labeling or distribution and use
restrictions;
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the FDA could require development of a REMS as a condition of approval or post-approval, or may not agree with our proposed REMS, or may impose additional requirements that limit the promotion, advertising,
distribution, or sales of
ZGN-1061;
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the FDA and EMA may find deficiencies with or not approve the manufacturing processes or facilities of third-party manufacturers with which we contract; or
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the FDA and EMA may change their approval policies or adopt new regulations.
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Any of these
factors, many of which are beyond our control, could jeopardize our ability to obtain and/or maintain regulatory approval for and successfully market
ZGN-1061.
Of the large number of drugs in development in
the United States, only a small percentage will successfully complete the FDA regulatory approval process and be commercialized. Accordingly, even if we are able to obtain the requisite financing to continue to fund our development and clinical
trials, we cannot assure you that
ZGN-1061
or any other of our product candidates will be successfully developed or commercialized.
Favorable results from
pre-clinical
studies and Phase 1 clinical trial of
ZGN-1061
to date are not necessarily predictive of the results of longer-term
pre-clinical
studies or clinical trials of
ZGN-1061.
Given the thrombosis findings in humans treated with beloranib, development costs for
ZGN-1061
may be higher and we may be unable to successfully develop,
obtain regulatory approval for and commercialize
ZGN-1061.
Favorable results from our
pre-clinical
studies of
ZGN-1061
may not necessarily be predictive of the results from clinical trials. To date we have shown that
ZGN-1061
has similar potency against the MetAP2 target and similar activity in mouse and rat models of obesity compared to beloranib. Toxicology studies in rats and dogs have shown that
ZGN-1061
is not exhibiting any testicular safety signals or activation of thrombosis-related biochemical markers, and displays an appreciable margin for embryofetal toxicity, compared to beloranib, which showed
testicular toxicity and
pro-thrombotic
effects with a small therapeutic margin and no margin for embryofetal toxicity. However, we can provide no assurance that the results of this
pre-clinical
development program will be replicated in future clinical trials of
ZGN-1061.
Many companies in the pharmaceutical and biotechnology industries have suffered
significant setbacks in clinical trials after achieving positive results in
pre-clinical
and early-stage development. In particular, we have suffered significant setbacks in later-stage clinical trials of our
former lead product candidate, beloranib, after achieving positive results in
pre-clinical
and clinical development, and we cannot be certain that we will not face similar setbacks in our development of
ZGN-1061.
The setbacks in later-stage clinical development have been caused by, among other things,
pre-clinical
findings made while clinical trials were underway or safety or
efficacy observations made in clinical trials, including previously unreported or ununderstood adverse events. Moreover,
pre-clinical
and clinical data are often susceptible to varying interpretations and
analyses, and many companies that believed their product candidates performed satisfactorily in
pre-clinical
studies and clinical trials nonetheless failed to obtain FDA and/or EMA approval. If we fail to
produce positive results in our clinical trials of
ZGN-1061,
the development timeline and regulatory approval and commercialization prospects for our lead product candidate, and, correspondingly, our business
and financial prospects, would be materially adversely affected.
Our product candidates may cause undesirable side effects that could delay or
prevent their regulatory approval, limit the commercial profile of an approved label, or result in significant negative consequences following marketing approval, if any.
Undesirable side effects caused by our product candidates could cause us or regulatory authorities such as the FDA to interrupt, delay or halt
clinical trials and could result in a more restrictive label or the delay or denial of regulatory approval by the FDA or other regulatory authorities. For example, common adverse events observed in patients treated with beloranib versus placebo
included diarrhea, injection site bruising, dizziness, decreased appetite, anxiety and sleep disturbances (insomnia principally manifested as delayed onset of sleep and abnormal dreams), among others. In addition, an imbalance in the number of
thrombotic events observed in
30
patients treated with beloranib as compared to patients on placebo in our clinical trials was observed. We may see similar adverse events with
ZGN-1061
as
we saw with beloranib, and therefore, we will study many of these parameters in
pre-clinical
and clinical development of
ZGN-1061.
In the first small Phase 1 clinical
trial assessment of
ZGN-1061s
safety, the most common side effects were mild gastrointestinal issues, (comparable between
ZGN-1061
and placebo groups), headache
and procedural-related irritation.
Further, if
ZGN-1061
receives marketing approval and we or
others identify undesirable side effects caused by the product (or any other similar product) after the approval, a number of potentially significant negative consequences could result, including:
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regulatory authorities may request that we withdraw the product from the market or may limit their approval of the product through labeling or other means;
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regulatory authorities may require the addition of labeling statements, such as a black box warning or a contraindication or a precaution;
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we may be required to change the way the product is distributed or administered, conduct additional clinical trials or change the labeling of the product;
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we may decide to remove the products from the marketplace;
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we could be sued and held liable for injury caused to individuals exposed to or taking our product candidates; and
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our reputation may suffer.
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Any of these events could prevent us from achieving or maintaining
market acceptance of the affected product candidate and could substantially increase the costs of commercializing our product candidates and significantly impact our ability to successfully commercialize our product candidates and generate revenues.
Failures or delays in the commencement or completion of our planned clinical trials of
ZGN-1061
could
result in increased costs to us and could delay, prevent or limit our ability to generate revenue and continue our business.
ZGN-1061
recently completed Phase 1 clinical development in the Netherlands and will require substantial further clinical development before we can submit an NDA to the FDA or an MAA to the EMA for its marketing
approval.
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Despite the guidance we may receive from the FDA, the EMA, or other applicable regulatory
authorities including Australia and New Zealand, any of these regulatory authorities can change their positions on the acceptability of our clinical trial designs or the clinical endpoints selected, which may require us to complete additional
clinical trials or impose stricter approval conditions than we currently expect. Successful completion of such clinical trials is a prerequisite to submitting an NDA to the FDA and an MAA to the EMA and, consequently, the ultimate approval and
commercial marketing of
ZGN-1061.
We do not know whether any clinical trials will begin or be completed on schedule, if at all, as the commencement and completion of clinical trials can be delayed or prevented
for a number of reasons, including, among others:
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the FDA, EMA or other governing bodies in Europe or Australia and New Zealand may deny permission to pursue clinical trials and/or indications we want to initiate;
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delays in regulatory filings or receiving regulatory approvals of INDs, or clinical trial authorization applications, or CTAs, that may be required;
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unfavorable results from our
pre-clinical
and /or
non-clinical
studies, the FDA, the EMA or the applicable regulatory authorities in
Australia or New Zealand, may require additional
pre-clinical
and /or
non-clinical
studies;
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delays in reaching or failing to reach agreement on acceptable terms with prospective CROs and clinical trial sites, the terms of which can be subject to extensive negotiation and may vary significantly among different
CROs and trial sites;
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inadequate quantity or quality of a product candidate or other materials necessary to conduct clinical trials, for example delays in the manufacturing of sufficient supply of finished drug product;
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difficulties obtaining Institutional Review Board, or IRB, and/or ethics committee approval to conduct a clinical trial at a prospective site or sites in the United States, the European Union, Australia or New Zealand;
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challenges in recruiting and enrolling patients to participate in clinical trials, including the size and nature of the patient population, the proximity of patients to clinical trial sites, eligibility criteria for the
clinical trial, the nature of the clinical trial protocol, the availability of approved effective treatments for the relevant disease and competition from other clinical trial programs for similar indications;
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severe or unexpected drug-related side effects experienced by patients in a clinical trial, including side effects previously identified in our previous clinical trials for beloranib;
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the FDA, the EMA, or the applicable regulatory authorities in Australia and New Zealand may disagree with our clinical trial design and our interpretation of data from clinical trials, or may change the requirements for
approval even after it has reviewed and commented on the design for our clinical trials;
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difficulties in retaining or recruiting clinical investigators and/or patients in our ongoing or future clinical trials;
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reports from
pre-clinical,
non-clinical
or clinical testing of other weight loss therapies that raise safety or efficacy concerns; and
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difficulties retaining patients who have enrolled in a clinical trial but may be prone to withdraw due to rigors of the clinical trial, lack of efficacy, side effects, screening and monitoring measures, personal issues
or loss of interest.
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Clinical trials may also be delayed or terminated as a result of ambiguous or negative interim
results. In addition, a clinical trial may be suspended or terminated by us, the FDA, other regulatory authorities, the IRBs, or ethics committees, at the sites where the IRBs or ethics committees are overseeing a clinical trial, a data monitoring
committee, or DMC, overseeing the clinical trial at issue or other regulatory authorities due to a number of factors, including, among others:
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failure to conduct the clinical trial in accordance with regulatory requirements or our clinical protocols;
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inspection of the clinical trial operations or trial sites by the FDA, the EMA, or the applicable regulatory authorities in Australia and New Zealand that reveals deficiencies or violations that require us to undertake
corrective action, including the imposition of a partial clinical hold or a full clinical hold;
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unforeseen safety issues, including any that could be identified in our
non-clinical
or
pre-clinical
studies, adverse side effects or lack
of effectiveness;
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changes in government regulations or administrative actions;
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problems with clinical supply materials; and
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lack of adequate funding to continue the clinical trial.
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Changes in regulatory requirements, FDA guidance or guidance from EMA, Australia or New Zealand or
unanticipated events during our clinical trials of
ZGN-1061
may occur, which may result in changes to clinical trial protocols or additional clinical trial requirements, which could result in increased costs
to us and could delay our development timeline.
Changes in regulatory requirements, FDA guidance or guidance from EMA or
unanticipated events during our clinical trials may force us to adjust our clinical program or the FDA, the EMA, or the applicable regulatory authorities in Australia and New Zealand may impose additional clinical trial and/or
pre-clinical
study requirements. For instance, the FDA issued draft guidance on developing products for weight management in February 2007, and issued draft guidance on developing products for the treatment of
diabetes in February 2008 but these guidance documents may be revised at any time. In December 2008, FDA established guidance on evaluating cardiovascular risk of new therapies for the treatment of type 2 diabetes. In March 2012,
the FDAs Endocrinologic and Metabolic Drugs Advisory Committee met to discuss possible changes to how the FDA evaluates the cardiovascular safety of weight-management drugs and although new guidance has not been issued yet it may occur at any
time. Amendments to our clinical trial protocols would require resubmission to the FDA, the EMA, or the applicable regulatory authorities in Australia and New Zealand as well as IRBs and ethics committees for review and approval, which may adversely
impact the cost, timing or successful completion of a clinical trial. If we experience delays completing, or if we terminate, any of our clinical trials, or if we are required to conduct additional clinical trials and/or
pre-clinical
studies, the commercial prospects for
ZGN-1061
may be harmed and our ability to generate product revenue will be delayed.
We rely, and expect that we will continue to rely, on third parties to conduct any future clinical trials for
ZGN-1061.
If these third parties do not successfully carry out their contractual duties or meet expected deadlines, we may not be able to develop and obtain regulatory approval for or commercialize
ZGN-1061
and our business could be substantially harmed.
We enter into agreements with
third-party CROs to provide monitors for and to manage data for our ongoing clinical trials. We rely heavily on these parties for execution of clinical trials for
ZGN-1061
and control only certain aspects of
their activities. As a result, we have less direct control over the conduct, timing and completion of these clinical trials and the management of data developed through the clinical trials than would be the case if we were relying entirely upon our
own staff. Communicating with outside parties can also be challenging, potentially leading to mistakes as well as difficulties in coordinating activities. Outside parties may:
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have staffing difficulties;
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fail to comply with contractual obligations;
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experience regulatory compliance issues;
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undergo changes in priorities or become financially distressed; or
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form relationships with other entities, some of which may be our competitors.
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These factors
may materially adversely affect the willingness or ability of third parties to conduct our clinical trials and may subject us to unexpected cost increases that are beyond our control. Nevertheless, we are responsible for ensuring that each of our
clinical trials is conducted in accordance with the applicable protocol, legal and regulatory requirements and scientific standards, and our reliance on CROs does not relieve us of our regulatory responsibilities. We and our CROs are required to
comply with requirements for Good Clinical Practice, or GCPs, which are legal requirements enforced by the FDA, the Competent Authorities of the Member States of the European Economic Area and comparable foreign regulatory authorities for any
products in clinical development. The FDA enforces these GCP regulations through periodic inspections of clinical trial sponsors, principal investigators and clinical trial sites, IRBs, and other vendors that may be involved in the clinical
development of new products. If we or our investigators or CROs fail to comply with applicable GCPs, the clinical data generated in our clinical trials may be deemed unreliable and the FDA or comparable foreign regulatory authorities may require us
to perform additional clinical trials before approving our marketing applications. We cannot assure you that, upon inspection, the FDA will determine that any of our clinical trials comply with GCPs. In addition, our clinical trials must be
conducted with products produced under current Good Manufacturing Practices, or cGMPs regulations, to assure the identity, strength, quality, and purity of our drug product candidates being used in the clinical trials, as well as the
to-be-marketed
formulation and product. Our failure or the failure of our CROs and/or contract manufacturing organizations, or CMOs, to comply with these regulations may
require us to repeat clinical trials, which would delay the regulatory approval process and could also subject us to enforcement action, up to and including, civil and criminal penalties.
Although we do design our clinical trials for
ZGN-1061,
investigators and CROs conduct all of the
clinical trials. As a result, many important aspects of our drug development programs are outside of our direct control. In addition, the investigators or CROs may not perform all of their obligations under arrangements with us or in compliance with
regulatory requirements, but we remain responsible and are subject to enforcement action that may include civil penalties up to and including criminal prosecution for any violations of FDA laws and regulations during the conduct of our clinical
trials. If the investigators or CROs do not perform clinical trials in a satisfactory manner, breach their obligations to us, or fail to comply with regulatory requirements, the development and commercialization of
ZGN-1061
may be delayed or our development program materially and irreversibly harmed. We cannot control the amount and timing of resources these investigators or CROs devote to our program or
ZGN-1061.
If we are unable to rely on clinical data collected by our investigators or CROs, we could be required to repeat, extend the duration of, or increase the size of our clinical trials and this could
significantly delay commercialization and require significantly greater expenditures.
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If any of our relationships with these third-party investigators or CROs terminate, we may not be
able to enter into arrangements with alternative investigators or CROs in a timely manner, or at all. If investigators or CROs do not successfully carry out their contractual duties or obligations or meet expected deadlines, if they need to be
replaced or if the quality or accuracy of the clinical data they obtain is compromised due to the failure to adhere to our clinical protocols, regulatory requirements or for other reasons, any such clinical trials may be extended, delayed or
terminated, and we may not be able to obtain regulatory approval for or successfully commercialize
ZGN-1061.
As a result, our financial results and the commercial prospects for
ZGN-1061
in the subject indication would be harmed, our costs could increase and our ability to generate revenue could be delayed.
We rely completely on third-party suppliers to manufacture our clinical drug supplies for
ZGN-1061,
and we
intend to rely on third parties to produce commercial supplies of
ZGN-1061
and
pre-clinical,
clinical and commercial supplies of any future product candidate.
We do not currently have, nor do we currently plan to acquire, the infrastructure or capability to internally manufacture our
clinical drug supply of
ZGN-1061,
or any future product candidates, for use in the conduct of our
pre-clinical
studies and clinical trials, and we lack the internal
resources and the capability to manufacture any product candidates on a clinical or commercial scale. The facilities used by our CMOs to manufacture the active drug substance and final drug product must be approved by our quality assurance unit and
inspected by the FDA and other comparable foreign regulatory agencies.
We rely on our CMOs to comply with cGMPs for manufacture of raw
materials, active drug substance and finished drug products. If our CMOs cannot successfully manufacture material that conforms to our specifications and the regulatory requirements of the FDA or applicable foreign regulatory agencies, the CMOs will
not be able to secure and/or maintain regulatory approval for their manufacturing facilities. While we manage our quality expectations through an audit program for our vendors and suppliers, we have no direct control over our CMOs ability to
maintain adequate quality control, quality assurance and qualified personnel. Furthermore, our CMOs are engaged with third party vendors to supply and/or manufacture starting materials or components for them, which exposes our CMOs to regulatory
risks for the production of such materials and components. As a result, failure to satisfy the regulatory requirements for the production of those materials and components may affect the regulatory clearance of our CMOs facilities generally.
If the FDA or an applicable foreign regulatory agency does not approve these facilities for the manufacture of our product candidates or if it withdraws its approval in the future, we may need to find alternative manufacturing facilities, which
would adversely impact our ability to develop, obtain regulatory approval for or market our product candidates.
We rely completely on
third-party suppliers to manufacture our
pre-clinical
and clinical drug supplies for
ZGN-1061.
Currently each batch of
ZGN-1061
is individually contracted under a work order, which is governed by a quality and service agreement. The current drug substance manufacturing process will support
pre-clinical
studies and early clinical trials
and will be further optimized to support advanced clinical development and commercialization. Current drug substance, including key starting material, in inventory, is expected to support Phase 2 clinical trials. A new formulation with
longer shelf life has been developed and manufactured to support Phase 2 clinical development.
Even if we receive marketing approval for
ZGN-1061
in the United States, we may never receive regulatory approval to market
ZGN-1061
outside of the United States.
We intend to pursue marketing approval of
ZGN-1061
in the United States, the European Union and in
other countries worldwide. In order to market any product outside of the United States, we must establish and comply with the numerous and varying safety, efficacy and other regulatory requirements of other countries, including potential additional
clinical trials and/or
pre-clinical
studies. Approval procedures vary among countries and can involve additional product candidate testing and additional administrative review periods. The time required to
obtain approvals in other countries might differ from that required to obtain FDA approval. The marketing approval processes in other countries may implicate all of the risks detailed above regarding FDA approval in the United States as well as
other risks. In particular, in many countries outside of the United States, products must receive pricing and reimbursement approval before the product can be commercialized. Obtaining this approval can result in substantial delays in bringing
products to market in such countries. In addition, on March 20, 2017, the United Kingdom government started the process to leave the European Union by April 2019, or Brexit. The effects of Brexit will depend on any agreements the United Kingdom
makes to retain access to European Union markets either during a transitional period or more permanently. Brexit could lead to legal uncertainty and potentially divergent national laws and regulation as the United Kingdom determines which European
Union laws to replace or replicate. Marketing approval in one country does not necessarily ensure marketing approval in another, but a failure or delay in obtaining marketing approval in one country may have a negative effect on the regulatory
process or commercial activities in others. Failure to obtain marketing approval in other countries or any delay or other setback in obtaining such approval would impair our ability to market
ZGN-1061
in such
foreign markets. Any such impairment would reduce the size of our potential market, which could have a material adverse impact on our business, results of operations and prospects.
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Even if we receive marketing approval for
ZGN-1061,
it may not
achieve broad market acceptance, which would limit the revenue that we generate from its sales.
The commercial success of
ZGN-1061,
if developed and approved for marketing by the FDA or EMA or other applicable regulatory authorities, will depend upon the awareness and acceptance of
ZGN-1061
among
the medical community, including physicians, patients, advocacy groups and healthcare payors. Market acceptance of
ZGN-1061,
if approved, will depend on a number of factors, including, among others:
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the relative convenience and ease of subcutaneous injections as the necessary method of administration of
ZGN-1061;
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the prevalence and severity of any adverse side effects associated with
ZGN-1061;
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limitations or warnings contained in the labeling approved for
ZGN-1061
by the FDA, EMA, or other regulatory authorities, such as a black box warning;
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availability of alternative treatments, including a number of competitive type 2 diabetes or obesity therapies already approved or expected to be commercially launched in the near future;
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the willingness of the target patient population to try new therapies and of physicians to prescribe these therapies;
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the strength of marketing and distribution support and timing of market introduction of competitive products;
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publicity concerning our products or competing products and treatments;
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the effectiveness of our sales and marketing strategies;
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our ability to increase awareness of
ZGN-1061
through marketing efforts;
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our ability to obtain sufficient third-party coverage or reimbursement;
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the willingness of patients to pay
out-of-pocket
in the absence of third-party coverage; and
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the likelihood that the FDA may require development of a REMS, as a condition of approval or post-approval or may not agree with our proposed REMS or may impose additional requirements that limit the promotion,
advertising, distribution or sales of
ZGN-1061.
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If
ZGN-1061
is approved but does not achieve an adequate level of acceptance by patients, advocacy groups, physicians and payors, we may not generate sufficient revenue from
ZGN-1061
to become or remain profitable. Before granting reimbursement approval, healthcare payors may require us to demonstrate that, in addition to treating type 2 diabetes or obesity in patients,
ZGN-1061
also provides incremental health benefits to patients. Our efforts to educate the medical community and third-party payors about the benefits of
ZGN-1061
may require
significant resources and may never be successful.
If we are unable to establish sales and marketing capabilities or enter into agreements with
third parties to market and sell
ZGN-1061,
we may not be able to generate any revenue.
We
do not currently have an established infrastructure for the sales, marketing and distribution of pharmaceutical products. In order to market
ZGN-1061,
if approved by the FDA or any other regulatory body, we
must build our sales, marketing, managerial and other
non-technical
capabilities or make arrangements with third parties to perform these services. If we are unable to establish adequate sales, marketing and
distribution capabilities, whether independently or with third parties, or if we are unable to do so on commercially reasonable terms, our business, results of operations, financial condition and prospects will be materially adversely affected.
Even if we receive marketing approval for
ZGN-1061,
we may still face future development and regulatory
difficulties.
Even if we receive marketing approval for
ZGN-1061,
regulatory authorities
may still impose significant restrictions on
ZGN-1061s
indicated uses or marketing or impose ongoing requirements for potentially costly post-approval studies.
ZGN-1061
will also be subject to ongoing FDA and EMA requirements governing the labeling, packaging, storage and promotion of the product and recordkeeping and submission of safety and other post-market
information. The FDA has significant post-market authority, including, for example, the authority to require labeling changes based on new safety information and to require post-market studies or clinical trials to evaluate serious safety risks
related to the use of a drug. The FDA also has the authority to require, as part of an NDA or post-approval, the submission of a REMS. Any REMS required by the FDA may lead to increased costs to assure compliance with new post-approval regulatory
requirements and potential requirements or restrictions on the sale of approved products, all of which could lead to lower sales volume and revenue. Additionally, the FDA may require a PMS and/or PMRs, that could represent and result in additional
restrictions and/or limitations for the product.
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Manufacturers of drug products and their facilities are subject to continual review and periodic
inspections by the FDA and other regulatory authorities for compliance with cGMPs and other regulations. If we or a regulatory agency discover problems with
ZGN-1061,
such as adverse events of unanticipated
severity or frequency, or problems with the facility where
ZGN-1061
is manufactured, a regulatory agency may impose restrictions on
ZGN-1061,
the manufacturer or us,
including requiring withdrawal of
ZGN-1061
from the market or suspension of manufacturing. If we or the manufacturing facilities for
ZGN-1061
fail to comply with
applicable regulatory requirements, a regulatory agency may, among other things:
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issue warning letters or untitled letters;
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seek an injunction or impose civil or criminal penalties or monetary fines;
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suspend or withdraw marketing approval;
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suspend any ongoing clinical trials;
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refuse to approve pending applications or supplements to applications submitted by us;
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suspend or impose restrictions on operations, including costly new manufacturing requirements; or
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seize or detain products, refuse to permit the import or export of products, or request that we initiate a product recall.
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Competing technologies could emerge, including devices and surgical procedures, adversely affecting our opportunity to generate revenue from the sale of
ZGN-1061.
The biotechnology and pharmaceutical industries are intensely competitive and
subject to rapid and significant technological change. We have competitors in a number of jurisdictions, many of which have substantially greater name recognition, commercial infrastructures and financial, technical and personnel resources than we
have. Established competitors may invest heavily to quickly discover and develop novel compounds that could make
ZGN-1061
obsolete or uneconomical. Any new product that competes with an approved product may
need to demonstrate compelling advantages in efficacy, convenience, tolerability and safety to be commercially successful. Other competitive factors, including generic competition, could force us to lower prices or could result in reduced sales. In
addition, new products developed by others could emerge as competitors to
ZGN-1061.
If we are not able to compete effectively against our current and future competitors, our business will not grow and our
financial condition and operations will suffer.
Our future growth depends, in part, on our ability to penetrate foreign markets, where we would be
subject to additional regulatory burdens and other risks and uncertainties.
Our future profitability will depend, in part, on our
ability to commercialize
ZGN-1061
in foreign markets for which we may rely on collaborations with third parties. If we commercialize
ZGN-1061
in foreign markets, we
would be subject to additional risks and uncertainties, including:
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our customers ability to obtain reimbursement for
ZGN-1061
in foreign markets;
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our inability to directly control commercial activities because we are relying on third parties;
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the burden of complying with complex and changing foreign regulatory, tax, accounting and legal requirements;
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different medical practices and customs in foreign countries affecting acceptance in the marketplace;
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import or export licensing requirements;
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longer accounts receivable collection times;
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longer lead times for shipping;
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language barriers for technical training;
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reduced protection of intellectual property rights in some foreign countries;
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foreign currency exchange rate fluctuations; and
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the interpretation of contractual provisions governed by foreign laws in the event of a contract dispute.
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Foreign sales of
ZGN-1061
could also be adversely affected by the imposition of governmental controls,
political and economic instability, trade restrictions and changes in tariffs.
36
We are subject to healthcare laws and regulations, which could expose us to criminal sanctions, civil
penalties, contractual damages, reputational harm and diminished profits and future earnings.
Healthcare providers, physicians and
others will play a primary role in the recommendation and prescription of
ZGN-1061,
if approved. Our future arrangements with third-party payors will expose us to broadly applicable fraud and abuse and other
healthcare laws and regulations that may constrain the business or financial arrangements and relationships through which we market, sell and distribute
ZGN-1061,
if we obtain marketing approval. Restrictions
under applicable federal and state healthcare laws and regulations include the following:
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The federal Anti-Kickback Statute prohibits, among other things, persons from knowingly and willfully soliciting, offering, receiving or paying 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 healthcare programs such as Medicare and Medicaid.
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The federal false claims laws impose criminal and civil penalties, including those from civil whistleblower or qui tam actions pursuant to the federal False Claims Act, 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.
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The federal Health Insurance Portability and Accountability Act of 1996, as amended by the Health Information Technology for Economic and Clinical Health Act, imposes criminal and civil liability for executing a scheme
to defraud any healthcare benefit program and also imposes obligations, including mandatory contractual terms, with respect to safeguarding the privacy, security and transmission of individually identifiable health information.
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The federal false statements statute prohibits knowingly and willfully falsifying, concealing or covering up a material fact or making any materially false statement in connection with the delivery of or payment for
healthcare benefits, items or services.
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The federal transparency requirements, sometimes referred to as the Sunshine Act, under the Patient Protection and Affordable Care Act, require manufacturers of drugs, devices, biologics, and medical
supplies to report to the Department of Health and Human Services information related to physician payments and other transfers of value and physician ownership and investment interests.
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Analogous state laws and regulations, such as state anti-kickback and false claims laws and transparency laws, may apply to sales or marketing arrangements and claims involving healthcare items or services reimbursed by
non-governmental
third-party payors, including private insurers, and some state laws require pharmaceutical companies to comply with the pharmaceutical industrys voluntary compliance guidelines and the
relevant compliance guidance promulgated by the federal government in addition to requiring drug manufacturers to report information related to payments to physicians and other healthcare providers or marketing expenditures and drug pricing.
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Ensuring that our future business arrangements with third parties comply with applicable healthcare laws and regulations
could be costly. It is possible that governmental authorities will conclude that our business practices do not comply with current or future statutes, regulations or case law involving applicable fraud and abuse or other healthcare laws and
regulations. If our operations, including anticipated activities to be conducted by our sales team, were found to be in violation of any of these laws or any other governmental regulations that may apply to us, we may be subject to significant
civil, criminal and administrative penalties, damages, fines and exclusion from government funded healthcare programs, such as Medicare and Medicaid, any of which could substantially disrupt our operations. If any of the physicians or other
providers or entities with whom we expect to do business is found not to be in compliance with applicable laws, they may be subject to criminal, civil or administrative sanctions, including exclusions from government funded healthcare programs.
The FDA and other regulatory agencies actively enforce the laws and regulations prohibiting the promotion of
off-label
uses. If we are found to have improperly promoted
off-label
uses, we may become subject to significant liability.
The FDA and other regulatory agencies strictly regulate the promotional claims that may be made about prescription products, such as
ZGN-1061,
if approved. In particular, a product may not be promoted for uses that are not approved by the FDA or such other regulatory agencies as reflected in the products approved labeling. If we receive
marketing approval for
ZGN-1061,
physicians may nevertheless prescribe
ZGN-1061
to their patients in a manner that is inconsistent with the approved label. If we are
found to have promoted such
off-label
uses, we may become subject to significant liability. The federal government has levied large civil and criminal fines against companies for alleged improper promotion and
has enjoined several companies from engaging in
off-label
promotion and required that they enter into corporate integrity agreements with the Office of Inspector General of the Department of Health and Human
Services, or OIG. The FDA has also requested that companies enter into consent decrees or permanent injunctions under which specified promotional conduct is changed or curtailed. If we cannot successfully manage the promotion of
ZGN-1061,
if approved, we could become subject to significant liability, which would materially adversely affect our business and financial condition.
37
Even if approved, reimbursement policies could limit our ability to sell
ZGN-1061.
If approved by regulatory authorities, market acceptance and sales of
ZGN-1061
will depend on reimbursement policies and may be affected by healthcare reform measures. Government authorities and third-party payors, such as private health insurers and health maintenance organizations,
decide which medications they will pay for and establish reimbursement levels for those medications. Cost containment is a primary concern in the U.S. healthcare industry and elsewhere. Government authorities and these third-party payors have
attempted to control costs by limiting coverage and the amount of reimbursement for particular medications. We cannot be sure that reimbursement will be available for
ZGN-1061
and, if reimbursement is
available, the level of such reimbursement. Reimbursement may impact the demand for, or the price of,
ZGN-1061.
If reimbursement is not available or is available only at limited levels, we may not be able to
successfully commercialize
ZGN-1061.
In some foreign countries, particularly in Canada and
European countries, the pricing of prescription pharmaceuticals is subject to strict governmental control. In these countries, pricing negotiations with governmental authorities can take six to 12 months or longer after the receipt of regulatory
approval and product launch. To obtain favorable reimbursement for the indications sought or pricing approval in some countries, we may be required to conduct a clinical trial that compares the cost-effectiveness of
ZGN-1061
with other available therapies. If reimbursement for
ZGN-1061
is unavailable in any country in which we seek reimbursement, if it is limited in scope or amount,
if it is conditioned upon our completion of additional clinical trials, or if pricing is set at unsatisfactory levels, our operating results could be materially adversely affected.
Our development programs for our product candidates, which are primarily related to
ZGN-1061,
may require
substantial financial resources and may ultimately be unsuccessful.
Our lead product candidate
ZGN-1061
recently completed Phase 1 clinical development, and there are a number of FDA and certain European regulatory requirements that we must satisfy before we can commence late-stage clinical trials of
ZGN-1061.
Satisfaction of these requirements will entail substantial time, effort and financial resources. We may never satisfy these requirements. We believe that our cash, cash equivalents and marketable
securities will be sufficient to fund operations for a period of at least one year from the issuance date of this Quarterly Report, but we may need to raise more funds to continue development and commercialization of
ZGN-1061
and our other product candidates, which may not be easily available. Furthermore, any time, effort and financial resources we expend on our other early-stage development programs may adversely affect
our ability to continue development and commercialization of
ZGN-1061,
and we may never commence clinical trials of such development programs despite expending significant resources in pursuit of their
development. If we do commence clinical trials of our other potential product candidates, such product candidates may never be approved by the FDA or other regulatory authorities.
Risks Relating to Our Intellectual Property Rights
If we are unable to adequately protect our proprietary technology or maintain issued patents which are sufficient to protect
ZGN-1061
or future product candidates, others could compete against us more directly, which would have a material adverse impact on our business, results of operations, financial condition and prospects.
Our commercial success will depend in part on our success in obtaining and maintaining issued patents and other intellectual
property rights in the United States and elsewhere and protecting our proprietary technology. If we do not adequately protect our intellectual property and proprietary technology, competitors may be able to use our technologies and erode or negate
any competitive advantage we may have, which could harm our business and ability to achieve profitability. Our owned patent application relates to compositions of matter and methods of use of
ZGN-1061.
As of July 31, 2017, we own one issued U.S. patent, one pending U.S. patent application, one pending Patent Cooperation Treaty, or
PCT, patent application, and two pending U.S. provisional patent applications that relate to
ZGN-1061.
As of July 31, 2017, we own 15 issued U.S. patents, and 9 pending U.S. patent applications with pending foreign counterpart applications,
all of which relate to our internal efforts to discover novel MetAP2 inhibitors.
We cannot provide any assurances that any of our pending
patent applications that mature into issued patents will include claims with a scope sufficient to protect
ZGN-1061
and our other product candidates. Other parties have developed technologies that may be
related or competitive to our approach, and may have filed or may file patent applications and may have received or may receive patents that may overlap or conflict with our patent applications, either by claiming the same methods or formulations or
by claiming subject matter that could dominate our patent position. The patent positions of biotechnology and pharmaceutical companies, including our patent position, involve complex legal and factual questions, and, therefore, the issuance, scope,
validity and enforceability of any patent claims that we may obtain cannot be predicted with certainty. Patents, if issued, may be challenged, deemed unenforceable, invalidated, or circumvented. U.S. patents and patent applications may also be
subject to interference proceedings,
ex parte
reexamination, or
inter partes
review proceedings, supplemental examination and challenges in district court. Patents may be subjected to opposition, post-grant review, or comparable
proceedings lodged in various foreign, both national and
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regional, patent offices. These proceedings could result in either loss of the patent or denial of the patent application or loss or reduction in the scope of one or more of the claims of the
patent or patent application. In addition, such proceedings may be costly. Thus, any patents that we may own or exclusively license may not provide any protection against competitors. Furthermore, an adverse decision in an interference proceeding
can result in a third party receiving the patent right sought by us, which in turn could affect our ability to develop, market or otherwise commercialize
ZGN-1061
and our other product candidates.
Furthermore, though an issued patent is presumed valid and enforceable, its issuance is not conclusive as to its validity or its
enforceability and it may not provide us with adequate proprietary protection or competitive advantages against competitors with similar products. Competitors may also be able to design around our patents. Other parties may develop and obtain patent
protection for more effective technologies, designs or methods. The laws of some foreign countries do not protect our proprietary rights to the same extent as the laws of the United States, and we may encounter significant problems in protecting our
proprietary rights in these countries. If these developments were to occur, they could have a material adverse effect on our potential future sales.
Our ability to enforce our patent rights depends on our ability to detect infringement. It is difficult to detect infringers who do not
advertise the components that are used in their products. Moreover, it may be difficult or impossible to obtain evidence of infringement in a competitors or potential competitors product. Any litigation to enforce or defend our patent
rights, even if we were to prevail, could be costly and time-consuming and would divert the attention of our management and key personnel from our business operations. We may not prevail in any lawsuits that we initiate and the damages or other
remedies awarded if we were to prevail may not be commercially meaningful.
In addition, proceedings to enforce or defend our patents
could put our patents at risk of being invalidated, held unenforceable, or interpreted narrowly. Such proceedings could also provoke third parties to assert claims against us, including that some or all of the claims in one or more of our patents
are invalid or otherwise unenforceable. If any of our patents covering
ZGN-1061
are invalidated or found unenforceable, our financial position and results of operations would be materially and adversely
impacted. In addition, if a court found that valid, enforceable patents held by third parties covered
ZGN-1061,
our financial position and results of operations would also be materially and adversely impacted.
The degree of future protection for our proprietary rights is uncertain, and we cannot ensure that:
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any of our patents, or any of our pending patent applications, if issued, will include claims having a scope sufficient to protect
ZGN-1061
or any other products or product
candidates;
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any of our pending patent applications will issue as patents;
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we will be able to successfully develop and commercialize
ZGN-1061,
if approved, before our relevant patents expire;
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we were the first to make the inventions covered by each of our patents and pending patent applications;
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we were the first to file patent applications for these inventions;
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others will not develop similar or alternative technologies that do not infringe our patents;
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any of our patents will be found to ultimately be valid and enforceable;
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any patents issued to us will provide a basis for an exclusive market for our commercially viable products, will provide us with any competitive advantages or will not be challenged by third parties;
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we will develop additional proprietary technologies or product candidates that are separately patentable; or
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that our commercial activities or products will not infringe upon the patents of others.
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We
rely upon unpatented trade secrets, unpatented
know-how
and continuing technological innovation to develop and maintain our competitive position, which we seek to protect, in part, by confidentiality
agreements with our employees and our collaborators and consultants. We also have agreements with our employees and selected consultants that obligate them to assign their inventions to us and have
non-compete
agreements with some, but not all, of our consultants. It is possible that technology relevant to our business will be independently developed by a person that is not a party to such an agreement. Furthermore, if the employees and consultants who
are parties to these agreements breach or violate the terms of these agreements, we may not have adequate remedies for any such breach or violation, and we could lose our trade secrets through such breaches or violations. Further, our trade secrets
could otherwise become known or be independently discovered by our competitors.
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We may infringe the intellectual property rights of others, which may prevent or delay our product
development efforts and stop us from commercializing or increase the costs of commercializing
ZGN-1061,
if approved.
Our success will depend in part on our ability to operate without infringing the intellectual property and proprietary rights of third parties.
We cannot assure you that our business, products and methods do not or will not infringe the patents or other intellectual property rights of third parties.
The pharmaceutical industry is characterized by extensive litigation regarding patents and other intellectual property rights. Other parties
may allege that
ZGN-1061
or the use of our technologies infringes patent claims or other intellectual property rights held by them or that we are employing their proprietary technology without authorization.
Patent and other types of intellectual property litigation can involve complex factual and legal questions, and their outcome is uncertain. Any claim relating to intellectual property infringement that is successfully asserted against us may require
us to pay substantial damages, including treble damages and attorneys fees if we are found to be willfully infringing another partys patents, for past use of the asserted intellectual property and royalties and other consideration going
forward if we are forced to take a license. In addition, if any such claim were successfully asserted against us and we could not obtain such a license, we may be forced to stop or delay developing, manufacturing, selling or otherwise
commercializing
ZGN-1061.
Even if we are successful in these proceedings, we may incur
substantial costs and divert management time and attention in pursuing these proceedings, which could have a material adverse effect on us. If we are unable to avoid infringing the patent rights of others, we may be required to seek a license,
defend an infringement action or challenge the validity of the patents in court, or redesign our products. Patent litigation is costly and time consuming. We may not have sufficient resources to bring these actions to a successful conclusion. In
addition, intellectual property litigation or claims could force us to do one or more of the following:
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cease developing, selling or otherwise commercializing
ZGN-1061;
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pay substantial damages for past use of the asserted intellectual property;
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obtain a license from the holder of the asserted intellectual property, which license may not be available on reasonable terms, if at all; and
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in the case of trademark claims, redesign or rename the trademarks or trade names of our product candidates to avoid infringing the intellectual property rights of third parties, which may not be possible and, even if
possible, could be costly and time-consuming.
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Any of these risks coming to fruition could have a material adverse effect on
our business, results of operations, financial condition and prospects.
We may be subject to claims challenging the inventorship or ownership of
our patents and other intellectual property.
We may also be subject to claims that former employees, collaborators or other third
parties have an ownership interest in our patents or other intellectual property. Litigation may be necessary to defend against these and other claims challenging inventorship or ownership. If we fail in defending any such claims, in addition to
paying monetary damages, we may lose valuable intellectual property rights, such as exclusive ownership of, or right to use, valuable intellectual property. Such an outcome could have a material adverse effect on our business. Even if we are
successful in defending against such claims, litigation could result in substantial costs and be a distraction to management and other employees.
Obtaining and maintaining our patent protection depends on compliance with various procedural, document submission, fee payment and other requirements
imposed by governmental patent agencies, and our patent protection could be reduced or eliminated for
non-compliance
with these requirements.
The U.S. Patent and Trademark Office, or U.S. PTO, and various foreign governmental patent agencies require compliance with a number of
procedural, documentary, fee payment and other provisions during the patent process. There are situations in which noncompliance can result in abandonment or lapse of a patent or patent application, resulting in partial or complete loss of patent
rights in the relevant jurisdiction. In such an event, competitors might be able to enter the market earlier than would otherwise have been the case.
We may be involved in lawsuits to protect or enforce our patents or the patents of our licensors, which could be expensive, time-consuming and
unsuccessful.
Competitors may infringe our patents or the patents of our licensors. To counter infringement or unauthorized use,
we may be required to file infringement claims, which can be expensive and time-consuming. In addition, in an infringement proceeding, a court may decide that a patent of ours or our licensors is not valid, is unenforceable and/or is not infringed,
or may refuse to stop the other party from using the technology at issue on the grounds that our patents do not cover the technology in question. An adverse result in any litigation or defense proceedings could put one or more of our patents at risk
of being invalidated or interpreted narrowly and could put our patent applications at risk of not issuing.
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Interference proceedings provoked by third parties or brought by us may be necessary to determine
the priority of inventions with respect to our patents or patent applications or those of our licensors. An unfavorable outcome could require us to cease using the related technology or to attempt to license rights to it from the prevailing party.
Our business could be harmed if the prevailing party does not offer us a license on commercially reasonable terms. Our defense of litigation or interference proceedings may fail and, even if successful, may result in substantial costs and distract
our management and other employees. We may not be able to prevent, alone or with our licensors, misappropriation of our intellectual property rights, particularly in countries where the laws may not protect those rights as fully as in the United
States.
Furthermore, because of the substantial amount of discovery required in connection with intellectual property litigation, there
is a risk that some of our confidential information could be compromised by disclosure during this type of litigation. There could also be public announcements of the results of hearings, motions or other interim proceedings or developments. If
securities analysts or investors perceive these results to be negative, it could have a material adverse effect on the price of our common stock.
Issued patents covering our product candidates could be found invalid or unenforceable if challenged in court.
If we or one of our licensing partners initiated legal proceedings against a third party to enforce a patent covering our product candidate,
the defendant could counterclaim that the patent covering our product candidate is invalid and/or unenforceable. In patent litigation in the United States, defendant counterclaims alleging invalidity and/or unenforceability are commonplace. Grounds
for a validity challenge include alleged failures to meet any of several statutory requirements, including lack of novelty, obviousness or
non-enablement.
Grounds for unenforceability assertions include
allegations that someone connected with prosecution of the patent withheld relevant information from the U.S. PTO, or made a misleading statement, during prosecution. Third parties may also raise similar claims before administrative bodies in the
United States or abroad, even outside the context of litigation. Such mechanisms include
re-examination,
post grant review and equivalent proceedings in foreign jurisdictions, e.g., opposition proceedings.
Such proceedings could result in revocation or amendment of our patents in such a way that they no longer cover our product candidates or competitive products. The outcome following legal assertions of invalidity and unenforceability is
unpredictable. With respect to validity, for example, we cannot be certain that there is no invalidating prior act, of which we and the patent examiner were unaware during prosecution. If a defendant were to prevail on a legal assertion of
invalidity and/or unenforceability, we would lose at least part, and perhaps all, of the patent protection on our product candidates. Such a loss of patent protection would have a material adverse impact on our business.
We do not seek to protect our intellectual property rights in all jurisdictions throughout the world and we may not be able to adequately enforce our
intellectual property rights even in the jurisdictions where we seek protection.
Filing, prosecuting and defending patents on
product candidates in all countries and jurisdictions throughout the world would be prohibitively expensive, and our intellectual property rights in some countries outside the United States could be less extensive than those in the United States. In
addition, the laws of some foreign countries do not protect intellectual property rights to the same extent as federal and state laws in the United States. Consequently, we may not be able to prevent third parties from practicing our inventions in
all countries outside the United States, or from selling or importing products made using our inventions in and into the United States or other jurisdictions. Competitors may use our technologies in jurisdictions where we have not obtained patent
protection to develop their own products and further, may export otherwise infringing products to territories where we have patent protection, but enforcement is not as strong as that in the United States. These products may compete with our
products and our patents or other intellectual property rights may not be effective or sufficient to prevent them from competing.
Many
companies have encountered significant problems in protecting and defending intellectual property rights in foreign jurisdictions. The legal systems of certain countries, particularly certain developing countries, do not favor the enforcement of
patents and other intellectual property protection, particularly those relating to biopharmaceuticals, which could make it difficult for us to stop the infringement of our patents or marketing of competing products in violation of our proprietary
rights generally. For example, an April 2014 report from the Office of the United States Trade Representative identified a number of countries, including India and China, where challenges to the procurement and enforcement of patent rights have
been reported. Several countries, including India and China, have been listed in the report every year since 1989. Proceedings to enforce our patent rights in foreign jurisdictions could result in substantial costs and divert our efforts and
attention from other aspects of our business, could put our patents at risk of being invalidated or interpreted narrowly, could put our patent applications at risk of not issuing and could provoke third parties to assert claims against us. We may
not prevail in any lawsuits that we initiate and the damages or other remedies awarded, if any, may not be commercially meaningful. Accordingly, our efforts to enforce our intellectual property rights around the world may be inadequate to obtain a
significant commercial advantage from the intellectual property that we develop or license.
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We are dependent on licensed intellectual property for certain early-stage product candidates. If we were
to lose our rights to licensed intellectual property, we may not be able to continue developing or commercializing such product candidates, if approved.
We have an exclusive license with Childrens Medical Center Corporation, pursuant to which we exclusively licensed certain patent rights
relating to decreasing the growth of fat tissue on a worldwide basis. We may enter into additional licenses to third-party intellectual property that are necessary or useful to our business. Current or future licensors may also allege that we have
breached our license agreement and may accordingly seek to terminate our license with them. In addition, current or future licensors may decide to terminate our license at will. If successful, this could result in our loss of the right to use the
licensed intellectual property, which could materially adversely affect our ability to develop and commercialize a product candidate or product, if approved, as well as harm our competitive business position and our business prospects.
We have not yet registered trademarks for a commercial trade name for
ZGN-1061
and failure to secure such
registrations could adversely affect our business.
We have not yet registered trademarks for a commercial trade name for
ZGN-1061.
Any future trademark applications may be rejected during trademark registration proceedings. Although we would be given an opportunity to respond to those rejections, we may be unable to overcome such
rejections. In addition, in the U.S. PTO and in comparable agencies in many foreign jurisdictions, third parties are given an opportunity to oppose pending trademark applications and to seek to cancel registered trademarks. Opposition or
cancellation proceedings may be filed against our trademarks, and our trademarks may not survive such proceedings. Moreover, any name we propose to use with our product candidates in the United States must be approved by the FDA, regardless of
whether we have registered it, or applied to register it, as a trademark. The FDA typically conducts a review of proposed product names, including an evaluation of potential for confusion with other product names. If the FDA objects to any of our
proposed proprietary product names, we may be required to expend significant additional resources in an effort to identify a suitable substitute name that would qualify under applicable trademark laws, not infringe the existing rights of third
parties and be acceptable to the FDA.
If we do not obtain additional protection under the Hatch-Waxman Amendments and similar foreign legislation
by extending the patent terms and obtaining data exclusivity for
ZGN-1061,
our business may be materially harmed.
Depending upon the timing, duration and specifics of development and FDA marketing approval of
ZGN-1061,
one or more of our U.S. patents may be eligible for limited patent term restoration under the Drug Price Competition and Patent Term Restoration Act of 1984, referred to as the Hatch-Waxman
Amendments. The Hatch-Waxman Amendments permit a patent restoration term of up to five years as compensation for patent term lost during product development and the FDA regulatory review process. However, we may not be granted an extension because
of, for example, failing to apply within applicable deadlines, failing to apply prior to expiration of relevant patents or otherwise failing to satisfy applicable requirements. Moreover, the applicable time period or the scope of patent protection
afforded could be less than we request. If we are unable to obtain patent term extension or restoration or the term of any such extension is less than we request, our competitors may obtain approval of competing products following our patent
expiration, and our ability to generate revenues could be materially adversely affected.
Changes in U.S. patent law could diminish the value of
patents in general, thereby impairing our ability to protect our products.
The United States has recently enacted and is currently
implementing the America Invents Act of 2011, which is wide-ranging patent reform legislation. Further, the U.S. Supreme Court has ruled on several patent cases in recent years, either narrowing the scope of patent protection available in certain
circumstances or weakening the rights of patent owners in certain situations. In addition to increasing uncertainty with regard to our ability to obtain future patents, this combination of events has created uncertainty with respect to the value of
patents, once obtained. Depending on decisions by the U.S. Congress, the federal courts and the U.S. PTO, the laws and regulations governing patents could change in unpredictable ways that would weaken our ability to obtain new patents or to enforce
our existing patents or future patents.
We may be subject to damages resulting from claims that we or our employees have wrongfully used or
disclosed alleged trade secrets of their former employers.
Our employees have been previously employed at other biotechnology or
pharmaceutical companies, including our competitors or potential competitors. Although we are not aware of any claims currently pending against us, we may be subject to claims that these employees or we have inadvertently or otherwise used or
disclosed trade secrets or other proprietary information of the former employers of our employees. Litigation may be necessary to defend against these claims. Even if we are successful in defending against these claims, litigation could result in
substantial costs and be a distraction to management. If we fail in defending such claims, in addition to paying money claims, we may lose valuable intellectual property rights or personnel. A loss of key personnel or their work product could hamper
or prevent our ability to develop and commercialize
ZGN-1061
and other product candidates, which would materially adversely affect our business, financial condition and results of operations.
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General Company-Related Risks
We have recently reduced the size of our organization, and we may encounter difficulties in managing this development and restructuring, which could
disrupt our operations. In addition, we may not achieve anticipated benefits and savings from the reduction.
In July 2016,
our board of directors approved the suspension of further development of beloranib and a restructuring plan, pursuant to which our workforce was reduced by approximately 31% as of December 2016. The workforce reduction resulted in the loss of
longer-term employees, the loss of institutional knowledge and expertise and the reallocation and combination of certain roles and responsibilities across the organization, all of which could adversely affect our operations. Given the
complexity of our business, we must continue to implement and improve our managerial, operational and financial systems, manage our facilities and continue to recruit and retain qualified personnel. This will be made more challenging given the
workforce reduction described above. As a result, our management may need to divert a disproportionate amount of its attention away from our
day-to-day
activities,
and devote a substantial amount of time to managing these activities. Further, the restructuring and possible additional cost containment measures may yield unintended consequences, such as attrition beyond our intended workforce
reduction and reduced employee morale. In addition, we may not achieve anticipated benefits from the workforce reduction. Due to our limited resources, we may not be able to effectively manage our operations or recruit and retain qualified
personnel, which may result in weaknesses in our infrastructure and operations, risks that we may not be able to comply with legal and regulatory requirements, and loss of employees and reduced productivity among remaining employees. For
example, the workforce reduction may negatively impact our clinical and regulatory functions, which would have a negative impact on our ability to successfully develop, and ultimately, commercialize ZGN-1061. If our management is unable to
effectively manage this transition and workforce reduction and additional cost containment measures, our expenses may be more than expected and we may not be able to implement our business strategy. As a result, our future financial performance and
our ability to commercialize
ZGN-1061
successfully would be negatively affected.
Our future success depends
on our ability to retain our executive officers, and particularly our current President and Chief Executive Officer, and to attract, retain and motivate qualified personnel.
We are highly dependent on Dr. Thomas E. Hughes, our President and Chief Executive Officer. We have entered into a severance and
change in control agreement with Dr. Hughes, but he may terminate his employment with us at any time. Although we do not have any reason to believe that we will lose the services of Dr. Hughes in the foreseeable future, the loss of his
services might impede the achievement of our research, development and commercialization objectives. We also do not have any
key-man
life insurance on Dr. Hughes.
Our success also depends upon the principal members of our executive, medical and development teams. We have entered into a severance and
change in control agreement with our executive officers and department vice president level employees, but they may terminate their employment with us at any time. The loss of the services of any of these persons might impede the achievement of our
development and commercialization objectives.
With any change in leadership, there is also a risk to retention of employees, as well as
the potential for disruption to business operations, initiatives, plans and strategies.
We rely on consultants and advisors, including
scientific and clinical advisors, to assist us in formulating our development and commercialization strategy. Our consultants and advisors may be employed by employers other than us and may have commitments under consulting or advisory contracts
with other entities that may limit their availability to us and may not be subject to our standard
non-compete
agreements. Recruiting and retaining qualified scientific personnel and sales and marketing
personnel will also be critical to our success. We may not be able to attract and retain these personnel on acceptable terms given the workforce reduction and competition among numerous pharmaceutical and biotechnology companies for similar
personnel. We also experience competition for the hiring of scientific personnel from universities and research institutions. Failure to succeed in clinical trials may make it more challenging to recruit and retain qualified scientific personnel.
Our employees may engage in misconduct or other improper activities, including violating applicable regulatory standards and requirements or
engaging in insider trading, which could significantly harm our business.
We are exposed to the risk of employee fraud or other
misconduct. Misconduct by employees could include intentional failures to comply with the regulations of the FDA and applicable
non-U.S.
regulators, provide accurate information to the FDA and applicable
non-U.S.
regulators, comply with healthcare fraud and abuse laws and regulations in the United States and abroad, report financial information or data accurately or disclose unauthorized activities to us. In
particular, sales, marketing and business arrangements in the healthcare industry are subject to extensive laws and regulations intended to prevent fraud, misconduct, kickbacks, self-dealing and other abusive practices. These laws and regulations
restrict or prohibit a wide range of pricing, discounting, marketing and promotion, sales commission, customer incentive programs and other business arrangements. Employee misconduct could also involve the improper use of, including trading on,
information obtained in the course of clinical trials, which
43
could result in regulatory sanctions and serious harm to our reputation. We have adopted an insider trading policy and a code of conduct, but it is not always possible to identify and deter
employee misconduct, and the precautions we take to detect and prevent this activity may be ineffective in controlling unknown or unmanaged risks or losses or in protecting us from governmental investigations or other actions or lawsuits stemming
from a failure to comply with these laws or regulations. If any such actions are instituted against us, and we are not successful in defending ourselves or asserting our rights, those actions could have a significant impact on our business,
including the imposition of significant fines or other sanctions.
We face potential product liability exposure, and, if claims are brought against
us, we may incur substantial liability.
The use of beloranib and
ZGN-1061
in clinical
trials and the sale of
ZGN-1061,
if developed and approved, exposes us to the risk of product liability claims. Product liability claims might be brought against us by patients, healthcare providers or others
selling or otherwise coming into contact with
ZGN-1061.
For example, we may be sued if any product we develop allegedly causes injury or death or is found to be otherwise unsuitable during product testing,
manufacturing, marketing or sale. Any such product liability claims may include allegations of defects in manufacturing, defects in design, a failure to warn of dangers inherent in the product, including as a result of interactions with alcohol or
other drugs, negligence, strict liability and a breach of warranties. Claims could also be asserted under state consumer protection acts. If we become subject to product liability claims and cannot successfully defend ourselves against them, we
could incur substantial liabilities. In addition, regardless of merit or eventual outcome, product liability claims may result in, among other things:
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withdrawal of patients from our clinical trials;
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substantial monetary awards to patients or other claimants;
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decreased demand for
ZGN-1061
or any future product candidates following marketing approval, if obtained;
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damage to our reputation and exposure to adverse publicity;
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increased FDA warnings on product labels;
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distraction of managements attention from our primary business;
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the inability to successfully commercialize
ZGN-1061
or any future product candidates, if approved.
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We maintain product liability insurance coverage for our clinical trials with a $10.0 million annual aggregate coverage limit.
Nevertheless, our insurance coverage may be insufficient to reimburse us for any expenses or losses we may suffer. Moreover, in the future, we may not be able to maintain insurance coverage at a reasonable cost or in sufficient amounts to protect us
against losses, including if insurance coverage becomes increasingly expensive. If and when we obtain marketing approval for
ZGN-1061,
we intend to expand our insurance coverage to include the sale of
commercial products; however, we may not be able to obtain this product liability insurance on commercially reasonable terms. Large judgments have been awarded in class action lawsuits based on drugs that had unanticipated side effects. The cost of
any product liability litigation or other proceedings, even if resolved in our favor, could be substantial, particularly in light of the size of our business and financial resources. A product liability claim or series of claims brought against us
could cause our stock price to decline and, if we are unsuccessful in defending such a claim or claims and the resulting judgments exceed our insurance coverage, our financial condition, business and prospects could be materially adversely affected.
We must maintain effective internal control over financial reporting, and if we are unable to do so, the accuracy and timeliness of our financial
reporting may be adversely affected, which could have a material adverse effect on our business and stock price.
We currently are
an emerging growth company, as defined in the Jumpstart our Business Startups Act of 2012, or the JOBS Act, and we have taken advantage of certain exemptions from various reporting requirements that are applicable to other public
companies that are not emerging growth companies including not being required to comply with the auditor attestation requirements of Section 404(b) of the Sarbanes-Oxley Act.
We must maintain effective internal control over financial reporting in order to accurately and timely report our results of operations and
financial condition. In addition, as a public company, the Sarbanes-Oxley Act of 2002, or the Sarbanes-Oxley Act, requires, among other things, that we assess the effectiveness of our disclosure controls and procedures quarterly and the
effectiveness of our internal control over financial reporting at the end of each fiscal year.
The rules governing the standards that
must be met for our management to assess our internal control over financial reporting pursuant to Section 404 of the Sarbanes-Oxley Act are complex and require significant documentation, testing and possible remediation. These stringent
standards require that our audit committee be advised and regularly updated on managements review of internal control over financial reporting. Our management may not be able to effectively and timely implement controls and
44
procedures that adequately respond to the increased regulatory compliance and reporting requirements that are applicable to us as a public company. If we fail to staff our accounting and finance
function adequately or maintain internal control over financial reporting adequate to meet the demands that will be placed upon us as a public company, including the requirements of the Sarbanes-Oxley Act, our business and reputation may be harmed
and our stock price may decline. Furthermore, investor perceptions of us may be adversely affected, which could cause a decline in the market price of our common stock.
Our ability to use our net operating loss carryforwards and certain tax credit carryforwards may be subject to limitation.
Since our inception in 2005, we have not recorded any U.S. federal or state income tax benefits for the net losses we have incurred in each
year or our earned tax credits, due to our uncertainty of realizing a benefit from those items. As of December 31, 2016, we had net operating loss carryforwards for federal and state income tax purposes of $49.1 million and
$35.9 million, respectively, which begin to expire in 2026 and 2030, respectively. As of December 31, 2016, we did not record deferred tax assets of $12.8 million (gross) that were attributable to stock option exercises which will be
recorded as an increase in additional paid in capital once they are realized in accordance with accounting for stock-based compensation awards. These deductions are not reflected in the federal and state net operating loss carryforwards and the
capitalized research and development expense deferred tax assets in the amounts of $9.4 million, $7.2 million, and $3.4 million, respectively. As of December 31, 2016, we also had available tax credit carryforwards for federal
and state income tax purposes of $13.1 million and $1.9 million, respectively, which begin to expire in 2026 and 2021, respectively. Under Section 382 of the Internal Revenue Code of 1986, as amended, or the Code, changes in our
ownership may limit the amount of our net operating loss carryforwards and tax credit carryforwards that could be utilized annually to offset our future taxable income, if any. This limitation would generally apply in the event of a cumulative
change in ownership of our company of more than 50% within a three-year period. Any such limitation may significantly reduce our ability to utilize our net operating loss carryforwards and tax credit carryforwards before they expire. Our
follow-on
public offering, initial public offering, or IPO, private placements and other transactions that have occurred since our inception, may trigger such an ownership change pursuant to Section 382. Any
such limitation, whether as the result of our recent
follow-on
public offering, IPO, prior private placements, sales of our common stock by our existing stockholders or additional sales of our common stock by
us, could have a material adverse effect on our results of operations in future years. We have not completed a study to assess whether an ownership change for purposes of Section 382 has occurred, or whether there have been multiple ownership
changes since our inception, due to the significant costs and complexities associated with such study.
Unfavorable global economic conditions could
adversely affect our business, financial condition or results of operations.
Our results of operations could be adversely affected
by general conditions in the global economy and in the global financial markets. A severe or prolonged economic downturn could result in a variety of risks to our business, including, weakened demand for our product candidates and our ability to
raise additional capital when needed on acceptable terms, if at all. A weak or declining economy could also strain our suppliers, possibly resulting in supply disruption, or cause our customers to delay making payments for our services. Any of the
foregoing could harm our business and we cannot anticipate all of the ways in which the current economic climate and financial market conditions could adversely impact our business.
Our internal computer systems, or those of our third-party CROs or other contractors or consultants, may fail or suffer security breaches, which could
result in a material disruption of our
ZGN-1061
or other product candidate development programs.
Despite the implementation of security measures, our internal computer systems and those of our third-party CROs and other contractors and
consultants are vulnerable to damage from computer viruses, unauthorized access, natural disasters, terrorism, war and telecommunication and electrical failures. While we have not experienced any such system failure or accident, if such an event
were to occur and cause interruptions in our operations, it could result in a material disruption of our programs. For example, the loss of clinical trial data for
ZGN-1061
could result in delays in our
regulatory approval efforts and significantly increase our costs to recover or reproduce the data. To the extent that any disruption or security breach results in a loss of or damage to our data or applications or other data or applications relating
to our technology or product candidates, or inappropriate disclosure of confidential or proprietary information, we could incur liabilities and the further development of
ZGN-1061
could be delayed.
We may not be successful in our efforts to identify or discover additional product candidates.
The success of our business depends primarily upon our ability to identify, develop and commercialize products based on our MetAP2 platform.
Although
ZGN-1061
is currently in clinical development, our research programs may fail to identify other potential product candidates for clinical development for a number of reasons. Our research methodology
may be unsuccessful in identifying potential product candidates or our potential product candidates may be shown to have harmful side effects or may have other characteristics that may make the products unmarketable or unlikely to receive marketing
approval.
If any of these events occur, we may be forced to abandon our development efforts for a program or programs, which would have a
material adverse effect on our business and could potentially cause us to cease operations. Research programs to identify new product candidates require substantial technical, financial and human resources. We may focus our efforts and resources on
potential programs or product candidates that ultimately prove to be unsuccessful.
45
We may seek to establish collaborations and, if we are not able to establish them on commercially
reasonable terms, we may have to alter our development and commercialization plans or expand our internal efforts and growth.
Our
drug development programs and the potential commercialization of our product candidates will require substantial additional cash to fund expenses. For some of our product candidates, we may decide to collaborate with pharmaceutical and biotechnology
companies for the development and potential commercialization of those product candidates in some or all markets.
We face significant
competition in seeking appropriate collaborators. Whether we reach a definitive agreement for a collaboration will depend, among other things, upon our assessment of the collaborators resources and expertise, the terms and conditions of the
proposed collaboration and the proposed collaborators evaluation of a number of factors. Those factors may include the design or results of clinical trials, the likelihood of approval by the FDA or similar regulatory authorities outside the
United States , the potential market for the applicable product candidate, the costs and complexities of manufacturing and delivering such product candidate to patients, the potential of competing products, the existence of uncertainty with respect
to our ownership of technology, which can exist if there is a challenge to such ownership without regard to the merits of the challenge and industry and market conditions generally. The collaborator may also consider alternative product candidates
or technologies for similar indications that may be available to collaborate on and whether such collaboration could be more attractive than the one with us for our product candidate. The terms of any collaboration or other arrangements that we may
establish may not be favorable to us.
We may also be restricted under existing license agreements from entering into future agreements on
certain terms with potential collaborators. Collaborations are complex and time-consuming to negotiate and document. In addition, there have been a significant number of recent business combinations among large pharmaceutical companies that have
resulted in a reduced number of potential future collaborators.
We may not be able to negotiate collaborations on a timely basis, on
acceptable terms, or at all. If we are unable or unwilling to do so, we may have to curtail the development of the product candidate for which we are seeking to collaborate, reduce or delay its development program or one or more of our other
development programs, delay its potential commercialization in some or all markets or reduce the scope of any sales or marketing activities, or increase our expenditures and undertake development or commercialization activities at our own expense,
including potentially increasing our infrastructure and investment outside the United States. If we elect to increase our expenditures to fund development or commercialization activities on our own, we may need to obtain additional capital, which
may not be available to us on acceptable terms or at all. If we do not have sufficient funds, we may not be able to further develop our product candidates or bring them to market and generate product revenue. In addition such efforts may
require diversion of a disproportionate amount of our attention away from other
day-to-day
activities, and require devotion of a substantial amount of our time to
managing these expansion activities.
In addition, any future collaborations that we enter into may not be successful. The success of our
collaboration arrangements will depend heavily on the efforts and activities of our collaborators. Collaborators generally have significant discretion in determining the efforts and resources that they will apply to these collaborations.
Disagreements between parties to a collaboration arrangement regarding clinical development and commercialization matters can lead to delays in the development process or commercializing the applicable product candidate and, in some cases,
termination of the collaboration arrangement. These disagreements can be difficult to resolve if neither of the parties has final decision-making authority. Collaborations with pharmaceutical or biotechnology companies and other third parties often
are terminated or allowed to expire by the other party. Any such termination or expiration would adversely affect us financially and could harm our business reputation.
We may acquire businesses or products, or form strategic alliances, in the future, and we may not realize the benefits of such acquisitions or
alliances.
We may acquire additional businesses or products, form strategic alliances or create joint ventures with third parties
that we believe will complement or augment our existing business. If we acquire businesses with promising markets or technologies, we may not be able to realize the benefit of such transactions if we are unable to successfully integrate such
businesses with our existing operations and company culture. We may encounter numerous difficulties in developing, manufacturing and marketing any new products resulting from a strategic alliance or acquisition that delay or prevent us from
realizing their expected benefits or enhancing our business. We cannot assure you that, following any such transaction, we will achieve the expected synergies to justify the transaction.
46
Risks Related to Our Financial Position and Need for Capital
We have not generated any revenue from product sales. We have incurred significant operating losses since our inception, and anticipate that we will
incur continued losses for the foreseeable future.
Biopharmaceutical product development is a highly speculative undertaking and
involves a substantial degree of risk. Our operations to date have been limited primarily to organizing and staffing our company and conducting research and development activities for beloranib,
ZGN-1061
and
ZGN-839
and early research activities. We have never generated any revenue from product sales. We have not obtained regulatory approvals for any of our product candidates.
Since our inception and until recently, we focused substantially all of our efforts and financial resources on developing beloranib, which was
in Phase 3 clinical development for our lead indication of the treatment of hyperphagia and obesity in patients with Prader-Willi Syndrome, or PWS, and Phase 2 clinical development for the treatment of obesity in patients with hypothalamic
injury-associated obesity, or HIAO. In December 2015, the FDA put the beloranib IND on full clinical hold. Due to the uncertainties, costs and risks associated with the development of beloranib, in July 2016, we suspended further
development of beloranib and directed our efforts and financial resources to developing
ZGN-1061.
In October 2016, we suspended our development of
ZGN-839
in order
to focus all of our resources to developing
ZGN-1061
and the discovery of novel and highly differentiated MetAP2 inhibitors.
We have funded our operations to date through proceeds from sales of redeemable convertible preferred stock, convertible debt and proceeds
from our IPO and
follow-on
public offering, and have incurred losses in each year since our inception. Our net losses were $26.4 million for the six months ended June 30, 2017 and $57.9 million
for the year ended December 31, 2016. As of June 30, 2017, we had an accumulated deficit of $263.9 million. Substantially all of our operating losses resulted from costs incurred in connection with our development programs for
beloranib,
ZGN-1061
and
ZGN-839,
early research activities, licensing milestone fees and from general and administrative costs associated with our operations. We expect
to incur increasing levels of operating losses over the next several years and for the foreseeable future. Our prior losses, combined with expected future losses, have had and will continue to have an adverse effect on our stockholders equity
and working capital. We expect our research and development expenses will increase over time in connection with our clinical trials of
ZGN-1061,
and of any other product candidates we may choose to pursue. In
addition, if and when we obtain marketing approval for
ZGN-1061,
we will incur significant sales, marketing and outsourced manufacturing expenses. We will continue to incur additional costs associated with
operating as a public company. As a result, we expect to continue to incur significant operating losses that would increase over time for the foreseeable future. Because of the numerous risks and uncertainties associated with developing
pharmaceutical products, we are unable to predict the extent of any future losses or when we will become profitable, if at all. Even if we do become profitable, we may not be able to sustain or increase our profitability on a quarterly or annual
basis.
Our ability to become profitable depends upon our ability to generate revenue. To date, we have not generated any revenue from any
of our product candidates, and we do not know when, or if, we will generate any revenue. We do not expect to generate significant revenue unless and until we obtain marketing approval of, and begin to sell,
ZGN-1061.
Our ability to generate revenue depends on a number of factors, including, but not limited to, our ability to:
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initiate and successfully complete clinical trials that meet their clinical endpoints;
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initiate and successfully complete all safety studies required to obtain U.S. and foreign marketing approval for
ZGN-1061
in the indications we are pursuing;
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commercialize
ZGN-1061,
if developed and approved, by developing a sales force or entering into collaborations with third parties; and
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achieve market acceptance of
ZGN-1061
in the medical community and with third-party payors.
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Absent our entering into a collaboration or partnership agreement, we expect to incur significant sales and marketing costs when we prepare to
commercialize
ZGN-1061.
Even if we initiate and successfully complete our clinical trials of
ZGN-1061,
and
ZGN-1061
is approved
for commercial sale, and despite expending these costs,
ZGN-1061
may not be a commercially successful drug. We may not achieve profitability soon after generating product sales, if ever. If we are unable to
generate sufficient product revenue, we will not become profitable and may be unable to continue operations without continued funding.
We will need
to raise additional funding, which may not be available on acceptable terms, or at all. Failure to obtain this necessary capital when needed may force us to delay, limit or terminate our product development efforts or other operations.
Developing small molecule products is expensive, and we expect our research and development expenses to increase substantially in connection
with our ongoing activities, particularly as we advance
ZGN-1061
into later stage clinical trials. Depending on the status of regulatory approval or, if approved, commercialization of
ZGN-1061
or any of our other product candidates, as well as the progress we make in selling
ZGN-1061
or any of our other product candidates, we may require additional capital
to fund operating needs thereafter. We may also need to raise additional funds sooner if we choose to pursue additional indications and/or geographies for
ZGN-1061
or our other product candidates or otherwise
expand more rapidly than we presently anticipate.
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As of June 30, 2017, our cash, cash equivalents and marketable securities were
$106.0 million. We expect that our cash, cash equivalents and marketable securities will be sufficient to fund our current operations for a period of at least one year from the issuance date of this Quarterly Report. However, our operating plan
may change as a result of many factors currently unknown to us, and we may need to seek additional funds sooner than planned, through public or private equity or debt financings, government or other third-party funding, marketing and distribution
arrangements and other collaborations, strategic alliances and licensing arrangements or a combination of these approaches. In any event, we will require additional capital to obtain regulatory approval for, and to commercialize, our product
candidates. Raising funds in the current economic environment may present additional challenges. Even if we believe we have sufficient funds for our current or future operating plans, we may seek additional capital if market conditions are favorable
or if we have specific strategic considerations.
Any additional fundraising efforts may divert our management from their
day-to-day
activities, which may adversely affect our ability to develop and commercialize our product candidates. In addition, we cannot guarantee that future financing will
be available in sufficient amounts or on terms acceptable to us, if at all. Moreover, the terms of any financing may adversely affect the holdings or the rights of our stockholders and the issuance of additional securities, whether equity or debt,
by us, or the possibility of such issuance, may cause the market price of our shares to decline. The sale of additional equity or convertible securities would dilute all of our stockholders. The incurrence of indebtedness would result in increased
fixed payment obligations and we may be required to agree to certain restrictive covenants, such as limitations on our ability to incur additional debt, limitations on our ability to acquire, sell or license intellectual property rights and other
operating restrictions that could adversely impact our ability to conduct our business. We could also be required to seek funds through arrangements with collaborative partners or otherwise at an earlier stage than otherwise would be desirable and
we may be required to relinquish rights to some of our technologies or product candidate or otherwise agree to terms unfavorable to us, any of which may have a material adverse effect on our business, operating results and prospects.
If we are unable to obtain funding on a timely basis, we may be required to significantly curtail, delay or discontinue one or more of our
research or development programs or the commercialization of any product candidate or be unable to expand our operations or otherwise capitalize on our business opportunities, as desired, which could materially affect our business, financial
condition and results of operations.
Raising additional capital may cause dilution to our existing stockholders, restrict our operations or require
us to relinquish rights.
We may seek additional capital through a combination of private and public equity offerings, debt
financings, collaborations and strategic and licensing arrangements. To the extent that we raise additional capital through the sale of common stock or securities convertible or exchangeable into common stock, a stockholders ownership interest
in our company will be diluted. In addition, the terms of any such securities may include liquidation or other preferences that materially adversely affect the rights of our stockholders. Debt financing, if available, would increase our fixed
payment obligations and may involve agreements that include covenants limiting or restricting our ability to take specific actions, such as incurring additional debt, making capital expenditures or declaring dividends. If we raise additional funds
through collaboration, strategic partnerships and licensing arrangements with third parties, we may have to relinquish valuable rights to
ZGN-1061
or other product candidates, our intellectual property, future
revenue streams or grant licenses on terms that are not favorable to us.
Risks Related to Our Common Stock
We expect that our stock price may fluctuate significantly.
The market price of shares of our common stock, similar to the market price of shares of common stock of other biopharmaceutical companies, is
subject to wide fluctuations. From January 1, 2017 to June 30, 2017 the daily closing price of our common stock on the NASDAQ Global Market ranged from a high of $5.08 to a low of $3.24 and will continue to be subject to wide fluctuations
in response to many risk factors listed in this section, and others beyond our control, including:
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plans for, progress of, or results from
pre-clinical
studies and clinical trials of
ZGN-1061
and/or other product candidates;
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the failure of the FDA to accept our IND for
ZGN-1061;
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the failure of the FDA or the EMA to approve
ZGN-1061;
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our ability to establish an adequate safety margin and profile for
ZGN-1061,
including risk of serious thromboembolic events;
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announcements of new products, technologies, commercial relationships, acquisitions or other events by us or our competitors;
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the success or failure of other type 2 diabetes or weight loss therapies;
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regulatory or legal developments in the United States and other countries;
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failure of
ZGN-1061,
if successfully developed and approved, to achieve commercial success;
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fluctuations in stock market prices and trading volumes of similar companies;
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general market conditions and overall fluctuations in U.S. equity markets;
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variations in our quarterly operating results;
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changes in our financial guidance or securities analysts estimates of our financial performance;
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changes in accounting principles;
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our ability to raise additional capital and the terms on which we can raise it;
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sales of large blocks of our common stock, including sales by our executive officers, directors and significant stockholders;
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additions or departures of key personnel;
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discussion of us or our stock price by the press and by online investor communities; and
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other risks and uncertainties described in these risk factors.
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These and other market and
industry factors may cause the market price and demand for our common stock to fluctuate substantially, regardless of our actual operating performance, which may limit or prevent investors from readily selling their shares of common stock and may
otherwise negatively affect the liquidity of our common stock. In addition, the stock market in general, and NASDAQ listed and biopharmaceutical companies in particular, have experienced extreme price and volume fluctuations that have often been
unrelated or disproportionate to the operating performance of these companies. In the past, when the market price of a stock has been volatile, holders of that stock have instituted securities class action litigation against the company that issued
the stock.
On October 21, 2015, a purported stockholder of the Company filed a putative class action lawsuit in the U.S. District
Court for the District of Massachusetts, against the Company and Thomas E. Hughes, captioned Aviad Bessler v. Zafgen, Inc. and Thomas E. Hughes,
No. 1:15-cv-13618.
An amended complaint was filed on February 22, 2016. The amended complaint alleges violations of Sections 10(b) and 20(a) of the Securities Exchange Act of 1934 and SEC Rule
10b-5
based on allegedly false
and misleading statements and omissions regarding our clinical trials for beloranib. On August 9, 2016, the District Court granted the motion to dismiss and dismissed the amended complaint with prejudice. On August 12, 2016, plaintiffs
filed a notice of appeal to the First Circuit Court of Appeals and, on April 7, 2017 the dismissal with prejudice was affirmed.
Our executive
officers, directors, and principal stockholders exercise significant control over our company.
As of July 31, 2017, the
existing holdings of our executive officers, directors, principal stockholders and their affiliates, including investment funds affiliated with Atlas Ventures, or Atlas, and entities affiliated with Fidelity Investment (FMR LLC), or Fidelity,
represent beneficial ownership, in the aggregate, of approximately 36.8% of our common stock. As a result, these stockholders, if they act together, are able to influence our management and affairs and control the outcome of matters submitted to our
stockholders for approval, including the election of directors and any sale, merger, consolidation, or sale of all or substantially all of our assets. The concentration of voting power among these stockholders may have an adverse effect on the price
of our common stock. In addition, this concentration of ownership might adversely affect the market price of our common stock by:
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delaying, deferring or preventing a change of control of us;
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impeding a merger, consolidation, takeover or other business combination involving us; or
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discouraging a potential acquirer from making a tender offer or otherwise attempting to obtain control of us.
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Future sales of our common stock may cause our stock price to decline.
Sales of a substantial number of shares of our common stock in the public market or the perception that these sales might occur could
significantly reduce the market price of our common stock and impair our ability to raise adequate capital through the sale of additional equity securities.
We have broad discretion in how we use the proceeds of our
follow-on
public offering. We may not use these
proceeds effectively, which could affect our results of operations and cause our stock price to decline.
We have considerable
discretion in the application of the net proceeds of our
follow-on
public offering. We intend to use the net proceeds to advance the clinical development of
ZGN-1061
and
to fund new and ongoing research and development activities,
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working capital and other general corporate purposes, which may include funding for the hiring of personnel, capital expenditures, early commercialization activities, the costs of operating as a
public company and potential business development activities. As a result, investors will be relying upon managements judgment with only limited information about our specific intentions for the use of the balance of the net proceeds. We may
use the net proceeds for purposes that do not yield a significant return or any return at all for our stockholders. In addition, pending their use, we may invest the net proceeds in a manner that does not produce income or that loses value.
We may be at an increased risk of securities class action litigation.
Historically, securities class action litigation has often been brought against a company following a decline in the market price of its
securities. This risk is especially relevant for us because biotechnology and pharmaceutical companies have experienced significant stock price volatility in recent years.
On October 21, 2015, a purported stockholder of the Company filed a putative class action lawsuit in the U.S. District Court for the
District of Massachusetts, against the Company and Thomas E. Hughes, captioned Aviad Bessler v. Zafgen, Inc. and Thomas E. Hughes,
No. 1:15-cv-13618.
An amended
complaint was filed on February 22, 2016. The amended complaint alleges violations of Sections 10(b) and 20(a) of the Securities Exchange Act of 1934 and SEC Rule
10b-5
based on allegedly false and
misleading statements and omissions regarding our clinical trials for beloranib. On August 9, 2016, the District Court granted the motion to dismiss and dismissed the amended complaint with prejudice. On August 12, 2016, plaintiffs filed a
notice of appeal to the First Circuit Court of Appeals and, on April 7, 2017 the dismissal with prejudice was affirmed.
We are an
emerging growth company and have availed ourselves of reduced disclosure requirements applicable to emerging growth companies, which could make our common stock less attractive to investors.
We are an emerging growth company, as defined in the JOBS Act, and we have taken advantage of certain exemptions from various
reporting requirements that are applicable to other public companies that are not emerging growth companies including not being required to comply with the auditor attestation requirements of Section 404(b) of the Sarbanes-Oxley
Act, reduced disclosure obligations regarding executive compensation in our periodic reports and proxy statements, and exemptions from the requirements of holding a nonbinding advisory vote on executive compensation and shareholder approval of any
golden parachute payments not previously approved. In addition, Section 107 of the JOBS Act also provides that an emerging growth company can take advantage of the extended transition period provided in Section 7(a)(2)(B) of the Securities
Act, for complying with new or revised accounting standards. In other words, an emerging growth company can delay the adoption of certain accounting standards until those standards would otherwise apply to private companies. However, we are electing
not to take advantage of such extended transition period, and as a result we will comply with new or revised accounting standards on the relevant dates on which adoption of such standards is required for
non-emerging
growth companies. Section 107 of the JOBS Act provides that our decision to not take advantage of the extended transition period for complying with new or revised accounting standards is
irrevocable. We cannot predict if investors will find our common stock less attractive because we may rely on any of the exemptions available under the JOBS Act. If some investors find our common stock less attractive as a result, there may be a
less active trading market for our common stock and our stock price may be more volatile. We may take advantage of these reporting exemptions until we are no longer an emerging growth company. We will remain an emerging growth company until the
earliest of (i) the last day of the fiscal year in which we have total annual gross revenue of $1.0 billion or more; (ii) December 31, 2019; (iii) the date on which we have issued more than $1.0 billion in
nonconvertible debt during the previous three years; and (iv) the date on which we are deemed to be a large accelerated filer under the rules of the SEC.
We have never paid dividends on our common stock and we do not anticipate paying any dividends in the foreseeable future. Consequently, any gains from
an investment in our common stock will likely depend on whether the price of our common stock increases.
We have not paid
dividends on any of our common stock to date and we currently intend to retain all of our future earnings, if any, to fund the development and growth of our business. As a result, capital appreciation, if any, of our common stock will be the sole
source of gains for our common stockholders for the foreseeable future. Consequently, in the foreseeable future, our common stockholders will likely only experience a gain from their investment in our common stock if the price of our common stock
increases.
If equity research analysts do not continue to publish research or reports about our business or if they issue unfavorable commentary or
downgrade our common stock, the price of our common stock could decline.
The trading market for our common stock relies in part on
the research and reports that equity research analysts publish about us and our business. We do not control these analysts. The price of our common stock could decline if one or more equity analysts downgrade our common stock or if analysts issue
other unfavorable commentary or cease publishing reports about us or our business.
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Anti-takeover provisions contained in our amended and restated certificate of incorporation and amended and
restated bylaws, as well as provisions of Delaware law, could impair a takeover attempt.
Our amended and restated certificate of
incorporation, amended and restated bylaws and Delaware law contain provisions which could have the effect of rendering more difficult, delaying or preventing an acquisition deemed undesirable by our board of directors. Our corporate governance
documents include provisions:
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creating a classified board of directors whose members serve staggered three-year terms;
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authorizing blank check preferred stock, which could be issued by our board of directors without stockholder approval and may contain voting, liquidation, dividend, and other rights superior to our common
stock;
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limiting the liability of, and providing indemnification to, our directors and officers;
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limiting the ability of our stockholders to call and bring business before special meetings;
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requiring advance notice of stockholder proposals for business to be conducted at meetings of our stockholders and for nominations of candidates for election to our board of directors;
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controlling the procedures for the conduct and scheduling of board of directors and stockholder meetings; and
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providing our board of directors with the express power to postpone previously scheduled annual meetings and to cancel previously scheduled special meetings.
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These provisions, alone or together, could delay or prevent hostile takeovers and changes in control or changes in our management.
As a Delaware corporation, we are also subject to provisions of Delaware law, including Section 203 of the Delaware General Corporation
law, which prevents some stockholders holding more than 15% of our outstanding common stock from engaging in certain business combinations without approval of the holders of substantially all of our outstanding common stock.
Any provision of our amended and restated certificate of incorporation, amended and restated bylaws or Delaware law that has the effect of
delaying or deterring a change in control could limit the opportunity for our stockholders to receive a premium for their shares of our common stock, and could also affect the price that some investors are willing to pay for our common stock.