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ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934
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TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934
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Delaware
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04-3475813
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(State or Other Jurisdiction of Incorporation or Organization)
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(IRS Employer Identification No.)
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9 Cedarbrook Drive
Cranbury, NJ
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08512
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(Address of Principal Executive Offices)
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(Zip Code)
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Title of each class
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Trading Symbol(s) |
Name of each exchange on which registered
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Common Stock, $0.01 par value
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RCKT
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Nasdaq Global Market
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Large accelerated filer
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☒
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Accelerated filer
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☐
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Non-accelerated filer
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☐
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Smaller reporting company
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Emerging growth company
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☐
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PART I.
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Page
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Item 1.
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5
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Item 1A.
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31
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Item 1B.
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63
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Item 2.
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63
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Item 3.
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63
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Item 4.
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63
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PART II.
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Item 5.
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63
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Item 6.
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64
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Item 7.
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64
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Item 7A.
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73
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Item 8.
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73
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Item 9.
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73 | |
Item 9A.
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73
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Item 9B.
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74
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Item 9C.
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74 | |
PART III
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Item 10.
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74
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Item 11.
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74
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Item 12.
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75
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Item 13.
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75
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Item 14.
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75
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PART IV
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Item 15.
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76
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Item 16.
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78
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79
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•
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federal, state, and non-U.S. regulatory requirements, including regulation of our current or any other future product candidates by the U.S. Food and Drug Administration (“FDA”);
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the timing of and our ability to submit regulatory filings with the FDA and to obtain and maintain FDA or other regulatory authority approval of, or other action with respect to, our product candidates;
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our competitors’ activities, including decisions as to the timing of competing product launches, pricing and discounting;
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whether safety and efficacy results of our clinical trials and other required tests for approval of our product candidates provide data to warrant progression of clinical trials, potential regulatory approval or further development of
any of our product candidates;
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our ability to develop, acquire and advance product candidates into, enroll a sufficient number of patients into, and successfully complete, clinical studies, and our ability to apply for and obtain regulatory approval for such product
candidates, within currently anticipated timeframes, or at all;
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our ability to establish key collaborations and vendor relationships for our product candidates and any other future product candidates;
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our ability to establish key collaborations and vendor relationships for our product candidates and any other future product candidates;
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our ability to acquire additional businesses, form strategic alliances or create joint ventures and our ability to realize the benefit of such acquisitions, alliances, or joint ventures;
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our ability to successfully develop and commercialize any technology that we may in-license or products we may acquire;
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unanticipated delays due to manufacturing difficulties, including the development of our direct manufacturing capabilities for our AAV programs, and any supply constraints or changes in the regulatory environment; our ability to
successfully operate in non-U.S. jurisdictions in which we currently or in the future do business, including compliance with applicable regulatory requirements and laws;
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uncertainties associated with obtaining and enforcing patents to protect our product candidates, and our ability to successfully defend ourselves against unforeseen third-party infringement claims;
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anticipated trends and challenges in our business and the markets in which we operate;
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natural and manmade disasters, including pandemics such as COVID-19, and other force majeures, which could impact our operations, and those of our partners and other participants in the health care industry, and which could adversely
impact our clinical studies, preclinical research activities, and drug supply;
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our estimates regarding our capital requirements; and
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our ability to obtain additional financing and raise capital as necessary to fund operations or pursue business opportunities.
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The outbreak of the novel strain of coronavirus, SARS-CoV-2, which causes COVID-19, has and may continue to adversely impact our business, including our preclinical and clinical studies.
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If we fail to obtain additional funding to conduct our planned research and development efforts, we could be forced to delay, reduce, or eliminate our product development programs or commercial development efforts.
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We have never generated any revenue from product sales and may never be profitable.
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We may encounter substantial delays in commencement, enrollment or completion of our clinical trials or may fail to demonstrate safety and efficacy to the satisfaction of applicable regulatory authorities, which could prevent us from
commercializing our current and future product candidates on a timely basis, if at all.
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If we have difficulty enrolling a sufficient number of patients to conduct our clinical trials as planned, we may need to delay, limit, or terminate planned clinical trials, the occurrence of any of which would harm our business,
financial condition, results of operations and prospects.
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Initial or interim results in our ongoing clinical studies may not be indicative of results obtained when these studies are completed. Furthermore, success in early clinical studies may not be indicative of results obtained in later
studies.
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Even if we successfully complete the necessary preclinical studies and clinical trials, we cannot predict when, or if, we will obtain regulatory approval to commercialize a product candidate and the approval may be for a narrower
indication than we seek.
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We may never obtain approval for any of our product candidates in the United States or the European Union, or other jurisdictions, which would limit our ability to realize our full market potential.
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Our product candidates may cause undesirable and unforeseen side effects or be perceived by the public as unsafe, which could delay or prevent their advancement into clinical trials or regulatory approval, limit the commercial
potential or result in significant negative consequences.
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We could experience production problems that result in delays in our development or commercialization programs, limit the supply of our products or otherwise harm our business.
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We have no experience in manufacturing, and there can be no assurance that we will be able to successfully manufacture products.
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Our ability to successfully develop and commercialize our product candidates will substantially depend upon the availability of reimbursement funds for the costs of the resulting drugs and related treatments.
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Even if approved, we may not successfully commercialize our product candidates.
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We may not be successful in our efforts to expand a pipeline of additional product candidates.
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The success of our research and development activities, clinical testing, and commercialization, upon which we primarily focus, is uncertain.
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We expect to rely on third parties to conduct some or all aspects of our drug product manufacturing, research, and preclinical and clinical testing, and these third parties may not perform satisfactorily.
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Our business could suffer if it loses the services of, or fails to attract, key personnel.
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We may need to expand our organization and may experience difficulties in managing this growth, which could disrupt our operations.
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Our employees, principal investigators, consultants, and commercial partners may engage in misconduct or other improper activities, including non-compliance with regulatory standards and requirements and insider trading which could
harm our business.
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Given our commercial relationships outside of the United States, in particular the European Union, a variety of risks associated with international operations could harm our business.
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If we are unable to obtain and maintain adequate patent protection for products and related technology, our ability to successfully commercialize our products may be harmed.
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If we breach our license agreements, it could have a material adverse effect on our commercialization efforts for our product candidates.
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We may incur substantial costs as a result of litigation or other proceedings relating to patent and other intellectual property rights and we may be unable to protect our rights to, or use, our technology.
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If we are unable to protect the confidentiality of our trade secrets, our business and competitive position may be harmed.
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If we are unable to obtain or protect intellectual property rights related to our product candidates, we may not be able to compete effectively in our markets.
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RTW Investments, LP, our largest stockholder, may have the ability to significantly influence matters submitted to stockholders for approval.
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Item 1. |
Business
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Term
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Definition
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Optimal Ranges
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LVV Therapy (hematopoietic disorders)
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CD34+ cell(s)
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Hematopoietic Stem Cell (most CD34+ cells are not true stem cells, but this continues to be the most clinically useful measure)
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Will depend on underlying disorder, generally > 1 million CD34+ cells/kg.
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Vector copy number (VCN)
[product]
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The average number of gene copies per infused stem cell (as determined by DNA analysis; this is an average ratio, not a precise value)
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0.5 to 2 has been target in some LVV clinical studies (5.0 generally considered maximum)
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Vector copy number (VCN)
[in vivo, post-treatment]
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The average number of gene copies per peripheral blood or bone marrow cell (as determined by DNA analysis; this is an average ratio, not a precise value)
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Will depend on underlying disorder, but many disorders may be correctable with in vivo VCNs << 1.0
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AAV Therapy
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Vector copy number (VCN)
[in vivo, post-treatment]
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The average number of gene copies per cell in the organ of interest (as determined by DNA analysis; this is an average ratio, not a precise value)
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Will depend on underlying disorder, but vivo VCNs << 1.0
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Analysis of the explanted heart revealed significant fibrosis consistent with advanced DD.
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Myocardial tissue from the explanted heart at 5 months post-treatment displayed 100% LAMP2B protein expression by immunohistochemistry throughout non-fibrotic cardiac regions including the ventricles and other essential targeted
areas.
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The ability of HSCT to cure the hematologic component of FA is proof-of-principle that gene therapy will work in FA. If a sufficient number of hematopoietic stem cells (“HSCs”) with a
correct (non-FA) gene are able to engraft in the bone marrow of an FA patient, the blood component of FA can be eradicated, including both the risk of bone marrow failure and of leukemia. We believe that gene therapy with a patient’s
own gene-corrected blood stem cells will work in a similar manner, but likely with fewer side effects than those resulting from an allogeneic transplant and with reduced long-term treatment cost burden.
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Mosaicism in FA patients: this is a condition in which a second mutation enables formation of a functional FA protein and leads to stabilization or correction of blood counts, in some cases enabling
decades of bone-marrow-failure free survival. Mosaicism occurs because gene-corrected FA stem and progenitor cells have a selective advantage over uncorrected FA cells; this phenomenon
has demonstrated that a modest number of gene corrected HSCs can repopulate a patient’s blood and bone marrow with corrected (non-FA) cells. A comprehensive review of all known cases of somatic mosaicism has demonstrated a correlation
with lowered risk of both bone marrow failure and hematologic malignancy. This selective advantage also has been demonstrated by the results of our initial FANCOLEN-I gene therapy study in Madrid, Spain, in which patients received
gene-corrected cells without any chemotherapy conditioning; the percentage of blood and bone marrow cells containing the corrected FA gene has increased progressively over time. These increases have been accompanied by increases in the
percentage of cells that are resistant to DNA-damaging agents, indicating a reversal of the FA phenotype in the blood and bone marrow of these patients.
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Improved vector design, stem cell selection methods, cell harvest and transduction procedures have the potential to substantially improve the quality of autologous gene therapy cell products; many of
these improvements have been included in our programs. As a result of these factors, we believe that there is reliable potential to confer disease correction at levels comparable to
allogeneic transplant, but without the chemotherapy conditioning and additional side effects associated with a transplant. For example, stem cell selection methods utilized by our academic partners have increased both CD34+ cell yield and
purity, while retaining select non-CD34+ populations that may be essential for successful engraftment of gene-corrected cells in the bone marrow.
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completion of non-clinical laboratory tests, animal studies and formulation studies conducted according to Good Laboratory Practice (“GLP”), or other applicable regulations;
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submission of an IND, which allows clinical trials to begin unless FDA objects within 30 days;
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performance of adequate and well-controlled human clinical trials to establish the safety and efficacy of the proposed drug or biologic for its intended use or uses conducted in accordance with FDA regulations
and Good Clinical Practices (“GCP”), which are international ethical and scientific quality standards meant to ensure that the rights, safety and well-being of trial participants are protected, and that the integrity of the data is
maintained;
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preparation and submission to the FDA of a BLA;
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submission of a user fee for FDA review of the BLA;
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review of the product by an FDA advisory committee, where appropriate or if applicable;
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satisfactory completion of pre-approval inspection of manufacturing facilities and clinical trial sites at which the product, or components thereof, are produced to assess compliance with current Good
Manufacturing Practice (“cGMP”) requirements, and if applicable, the FDA’s current Good Tissue Practice (“cGTP”) requirements, and of selected clinical trial sites to assess compliance with GCP requirements; and
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FDA approval of a BLA which must occur before a biologic can be marketed or sold.
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In August 2011, President Obama signed into law the Budget Control Act of 2011, which, among other things, created the Joint Select Committee on Deficit Reduction to recommend to Congress proposals for deficit reduction of at least
$1.2 trillion for the years 2013 through 2021. The Joint Select Committee on Deficit Reduction did not achieve a targeted deficit reduction, which triggered the legislation’s automatic reduction to several government programs. This
includes aggregate reductions to Medicare payments to providers of, up to 2% per fiscal year, and, due to subsequent legislative amendments, will remain in effect through 2030 unless Congress takes additional action. These reductions
went into effect in April 2013 and, due to subsequent legislative amendments to the statute, will remain in effect through 2030 unless additional action is taken by Congress. Pursuant to the Coronavirus Aid, Relief, and Economic
Security Act, also known as the CARES Act, as well as subsequent legislation, these reductions have been suspended from May 1, 2020 through March 31, 2022 due to the COVID-19 pandemic. Then, a 1% payment reduction will occur beginning
April 1, 2022 through June 30, 2022, and the 2% payment reduction will resume on July 1, 2022.
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In January 2013, the American Taxpayer Relief Act of 2012, among other things, reduced Medicare payments to several providers, including hospitals and cancer treatment centers, and increased the statute of limitations period for the
government to recover overpayments to providers from three to five years.
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On April 13, 2017, CMS published a final rule that gives states greater flexibility in setting benchmarks for insurers in the individual and small group marketplaces, which may have the effect of relaxing the essential health benefits
required under the ACA for plans sold through such marketplaces.
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On May 30, 2018, the Right to Try Act, was signed into law. The law, among other things, provides a federal framework for certain patients to access certain investigational new drug products that have completed a Phase 1 clinical trial
and that are undergoing investigation for FDA approval. Under certain circumstances, eligible patients can seek treatment without enrolling in clinical trials and without obtaining FDA permission under the FDA expanded access program.
There is no obligation for a pharmaceutical manufacturer to make its drug products available to eligible patients as a result of the Right to Try Act.
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On May 23, 2019, CMS published a final rule to allow Medicare Advantage Plans the option of using step therapy for Part B drugs beginning January 1, 2020.
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On December 20, 2019, former President Trump signed into law the Further Consolidated Appropriations Act (H.R. 1865), which repealed the Cadillac tax, the health insurance provider tax, and the medical device excise tax. It is
impossible to determine whether similar taxes could be instated in the future.
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a second applicant can establish that its product, although similar to the authorized product, is safer, more effective, or otherwise clinically superior;
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the marketing authorization holder for the authorized product consents to a second orphan medicinal product application; or
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the marketing authorization holder for the authorized product cannot supply enough orphan medicinal product.
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delays or difficulties in enrolling patients in our clinical trials;
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delays or difficulties in clinical site initiation, including difficulties in recruiting clinical site investigators and clinical site staff;
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diversion of healthcare resources from the conduct of clinical trials such as patient follow up visits, the diversion of hospitals ability to serve as our clinical trial sites and hospital staff supporting the conduct of our clinical
trials;
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interruption of clinical trial activities, such as clinical trial site data monitoring, due to limitations on travel imposed or recommended by federal or state governments, employers and others or interruption of clinical trial subject
visits and study procedures (particularly any procedures that may be deemed non-essential), which may impact the integrity of subject data and clinical study endpoints;
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delays or difficulties in the buildout of our in-house manufacturing;
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delays or difficulties in securing manufacturing slots or materials;
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delays or difficulties in advancing preclinical research requiring in-person laboratory work at our facility at academic partners or contract research facilities; and
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interruption or delays in the operations of the FDA and comparable foreign regulatory agencies, which may impact approval timelines.
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the timing of enrollment, commencement, completion, and results of our preclinical studies and clinical trials;
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costs for seeking regulatory approval for our product candidates in the U.S., Europe and other jurisdictions;
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the production of LVV and AAV gene therapy product candidates to support preclinical and clinical needs;
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the results of our preclinical studies for our current product candidates and any subsequent clinical trials;
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the scope, progress, results, and costs of researching and developing our product candidates, and conducting preclinical studies and clinical trials;
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the costs, timing, and outcome of regulatory review of our product candidates;
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the costs of future activities, including product sales, medical affairs, marketing, manufacturing, and distribution, for any of our product candidates for which we receive marketing approval;
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the costs of manufacturing clinical-grade and commercial-grade product to support our clinical trials and commercial launch, if approved;
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the ability to receive additional non-dilutive funding, including grants from organizations and foundations;
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the revenue, if any, received from commercial sale of its products, should any of its product candidates receive marketing approval;
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the costs of preparing, filing and prosecuting patent applications, maintaining and enforcing our intellectual property rights and defending intellectual property-related claims;
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our ability to establish and maintain collaborations on favorable terms, if at all;
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the extent to which we acquire or in-license other product candidates and technologies; and
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the timing, receipt, and amount of sales of, or milestone payments related to our royalties on, current or future product candidates, if any.
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completing research and preclinical and clinical development of our product candidates;
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seeking and obtaining regulatory and marketing approvals for product candidates for which we successfully complete clinical studies;
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developing a sustainable, commercial-scale, reproducible, and transferable manufacturing process for our vectors and product candidates;
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establishing and maintaining supply and manufacturing relationships with third parties that can provide adequate (in amount and quality) products and services to support preclinical and clinical development and the market demand for
our product candidates, if approved;
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launching and commercializing product candidates for which we obtain regulatory and marketing approval, either by collaborating with a partner or, if launched independently, by establishing a sales force, marketing and distribution
infrastructure;
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obtaining sufficient pricing and reimbursement for our product candidates from private and governmental payors;
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obtaining market acceptance of our product candidates and gene therapy as a viable treatment option;
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addressing any competing technological and market developments;
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identifying and validating new gene therapy product candidates;
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negotiating favorable terms in any collaboration, licensing or other arrangements into which we may enter; and
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maintaining, protecting and expanding our portfolio of intellectual property rights, including patents, trade secrets and know-how.
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failure of patients to enroll in the studies at the rate we expect;
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ineffectiveness of our product candidates;
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patients experiencing unexpected side effects or other safety concerns being raised during treatment;
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changes in governmental regulations or administrative actions;
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failure to conduct studies in accordance with required clinical practices;
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inspection of clinical study operations or study sites by the FDA, the EMA or other regulatory authorities, resulting in a clinical hold;
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insufficient financial resources;
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insufficient supplies of drug product to treat patients in our ongoing and planned clinical trials;
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political unrest at domestic or foreign clinical sites;
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a shutdown of the U.S. government, including the FDA;
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public health crises such as pandemics and epidemics; or
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natural disasters at any of our clinical sites.
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difficulty in establishing or managing relationships with CROs and physicians;
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different standards for the conduct of clinical trials;
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absence in some countries of established groups with sufficient regulatory expertise for review of LVV and AAV gene therapy protocols;
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our inability to locate qualified local partners or collaborators for such clinical trials; and
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the potential burden of complying with a variety of foreign laws, medical standards and regulatory requirements, including the regulation of pharmaceutical and biotechnology products and treatment.
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severity of the disease under investigation;
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design of the study protocol;
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size of the patient population;
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eligibility criteria for the study in question;
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perceived risks and benefits of the product candidate under study, including as a result of adverse effects observed in similar or competing therapies;
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proximity and availability of clinical study sites for prospective patients;
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availability of competing therapies and clinical studies;
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efforts to facilitate timely enrollment in clinical studies;
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patient referral practices of physicians; and
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ability to monitor patients adequately during and after treatment.
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regulatory authorities may suspend or withdraw approvals of such product candidate;
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regulatory authorities may require additional warnings on the label;
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we may be required to change the way a product candidate is administered or conduct additional clinical trials; and
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our reputation may suffer.
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the second applicant can establish in its application that its medicinal product, although similar to the orphan medicinal product already authorized, is safer, more effective or otherwise clinically superior;
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the holder of the marketing authorization for the original orphan medicinal product consents to a second orphan medicinal product application; or
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the holder of the marketing authorization for the original orphan medicinal product cannot supply sufficient quantities of orphan medicinal product.
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issue a warning letter asserting that we are in violation of the law;
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seek an injunction or impose civil or criminal penalties or monetary fines;
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suspend any ongoing clinical studies;
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refuse to approve a pending marketing application, such as a BLA or supplements to a BLA submitted by us;
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seize products; or
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refuse to allow us to enter into supply contracts, including government contracts.
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a covered benefit under its health plan;
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safe, effective, and medically necessary;
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appropriate for the specific patient;
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cost-effective; and
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neither experimental nor investigational.
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some or all of our product candidates may be found to be unsafe or ineffective or otherwise fail to meet applicable regulatory standards or receive necessary regulatory clearances; our product candidates, if safe and effective, may
nonetheless not be able to be developed into commercially viable products;
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it may be difficult to manufacture or market our product candidates on a scale that is necessary to ultimately deliver our products to end-users;
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proprietary rights of third parties may preclude us from marketing our product candidates;
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the nature of our indications as rare diseases means that the potential market size may be limited; and
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third parties may market superior or equivalent drugs which could adversely affect the commercial viability and success of our product candidates.
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the efficacy and safety of such product candidates as demonstrated in preclinical studies and clinical trials;
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the potential and perceived advantages of product candidates over alternative treatments;
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the cost of our treatment relative to alternative treatments;
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the clinical indications for which the product candidate is approved by the FDA or the EMA;
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patient awareness of, and willingness to seek, gene therapy;
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the willingness of physicians to prescribe new therapies;
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the willingness of physicians to undergo specialized training with respect to administration of our product candidates;
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the willingness of the target patient population to try new therapies;
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the prevalence and severity of any side effects;
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product labeling or product insert requirements of the FDA, the EMA or other regulatory authorities, including any limitations or warnings contained in a product’s approved labeling;
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relative convenience and ease of administration;
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the strength of marketing and distribution support;
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the timing of market introduction of competitive products;
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publicity concerning our products or competing products and treatments; and
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sufficient third-party payor coverage and reimbursement.
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the inability to negotiate manufacturing agreements with third parties under commercially reasonable terms;
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reduced control as a result of using third-party manufacturers for certain aspects of manufacturing activities;
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the risk that these activities are not conducted in accordance with our study plans and protocols;
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• |
termination or nonrenewal of manufacturing agreements with third parties in a manner or at a time that is costly or damaging to us; and
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• |
disruptions to the operations of our third-party manufacturers or suppliers caused by conditions unrelated to our business or operations, including the bankruptcy of the manufacturer or supplier.
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the scope of rights granted under the license agreement;
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• |
whether and the extent to which our technology and processes infringe on intellectual property of the licensor that is not subject to the licensing agreement;
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• |
our right to sublicense patent and other intellectual property rights to third parties under collaborative development relationships;
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• |
our diligence obligations with respect to the use of the licensed technology in relation to our development and commercialization of is product candidates, and what activities satisfy those diligence obligations;
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• |
the ownership of inventions and know-how resulting from the joint creation or use of intellectual property by our licensors and us and our partners; and
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• |
whether and the extent to which inventors are able to contest the assignment of their rights to our licensors.
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• |
others may be able to make product candidates that are similar to ours but that are not covered by the claims of the patents that we own;
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• |
we, or our license partners or current or future collaborators, might not have been the first to make the inventions covered by the issued patent or pending patent applications that we license or may own in the future;
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• |
we, or our license partners or current or future collaborators, might not have been the first to file patent applications covering certain of our or their inventions;
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• |
others may independently develop similar or alternative technologies or duplicate any of our technologies without infringing our owned or in-licensed intellectual property rights;
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• |
our competitors might conduct research and development activities in countries where we do not have patent rights and then use the information learned from such activities to develop competitive products for sale in our major
commercial markets;
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• |
we cannot ensure that any of our patents, or any of our pending patent applications, if issued, or those of our licensors, will include claims having a scope sufficient to protect our product candidates;
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• |
we cannot ensure that any patents issued to us, or our licensors will provide a basis for an exclusive market for our commercially viable product candidates or will provide us with any competitive advantages;
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• |
we cannot ensure that our commercial activities or product candidates will not infringe upon the patents of others;
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• |
we cannot ensure that we will be able to successfully commercialize our product candidates on a substantial scale, if approved, before the relevant patents that we own, or license expire;
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• |
we may not develop additional proprietary technologies that are patentable;
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• |
the patents or intellectual property rights of others may harm our business; and
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• |
we may choose not to file a patent in order to maintain certain trade secrets or know-how, and a third party may subsequently file a patent covering such intellectual property.
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• |
different regulatory requirements for approval of drugs and biologics in foreign countries;
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• |
reduced protection for intellectual property rights;
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• |
unexpected changes in tariffs, trade barriers and regulatory requirements;
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• |
economic weakness, including inflation, or political instability in particular foreign economies and markets;
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• |
compliance with tax, employment, immigration, and labor laws for employees living or traveling abroad;
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• |
foreign currency fluctuations, which could result in increased operating expenses and reduced revenues, and other obligations incident to doing business in another country;
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• |
workforce uncertainty in countries where labor unrest is more common than in the United States;
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• |
shortages resulting from any events affecting raw material supply or manufacturing capabilities abroad;
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• |
business interruptions resulting from geopolitical actions, including war and terrorism or natural disasters including earthquakes, typhoons, floods and fires, public health crises such as pandemics and epidemics, or from economic or
political instability;
|
• |
compliance with foreign laws, regulations, standards, and regulatory guidance governing the collection, use, disclosure, retention, security and transfer of personal data, including the European Union General Data Privacy Regulation
(“GDPR”); and
|
• |
greater difficulty with enforcing our contracts in jurisdictions outside of the United States.
|
• |
diversion of management time and focus from operating our business to addressing acquisition integration challenges;
|
• |
coordination of R&D efforts;
|
• |
retention of key employees from any acquired company;
|
• |
changes in relationships with strategic partners as a result of any product acquisitions or strategic positioning resulting from the acquisition;
|
• |
cultural challenges associated with integrating employees from any acquired company into our organization or managing a strategic alliance or joint venture;
|
• |
the need to implement or improve controls, procedures, and policies at any acquired business that prior to the acquisition may have lacked sufficiently effective controls, procedures and policies;
|
• |
liability for activities of any acquired company before the acquisition, including intellectual property infringement claims, violation of laws, commercial disputes, tax liabilities, and other known liabilities;
|
• |
unanticipated write-offs or charges; and
|
• |
litigation or other claims in connection with any acquired company, including claims from terminated employees, customers, former stockholders or other third parties
|
• |
results of clinical trials of our product candidates or those of our competitors;
|
• |
the success of competitive products or technologies;
|
• |
commencement or termination of collaborations;
|
• |
regulatory or legal developments in the United States and other countries;
|
• |
developments or disputes concerning patent applications, issued patents or other proprietary rights;
|
• |
the recruitment or departure of key personnel;
|
• |
the level of expenses related to any of our product candidates or clinical development programs;
|
• |
the results of our efforts to discover, develop, acquire or in-license additional product candidates;
|
• |
actual or anticipated changes in estimates as to financial results, development timelines or recommendations by securities analysts;
|
• |
negative publicity around gene therapy in general, or our product candidates;
|
• |
variations in our financial results or those of companies that are perceived to be similar to us;
|
• |
changes in the structure of healthcare payment systems;
|
• |
market conditions in the pharmaceutical and biotechnology sectors; and
|
• |
general economic, industry and market conditions.
|
• |
the requirement that our independent registered public accounting firm attest to the effectiveness of our internal control over financial reporting under Section 404(b) of the Sarbanes-Oxley Act of 2002;
|
• |
compliance with any requirement that may be adopted by the Public Company Accounting Oversight Board regarding mandatory audit firm rotation or a supplement to the auditor’s report providing additional information about the audit and
the financial statements;
|
• |
the requirement that we provide full and more detailed disclosures regarding executive compensation; and
|
• |
the requirement that we hold a non-binding advisory vote on executive compensation and obtain stockholder approval of any golden parachute payments not previously approved.
|
• |
permit only the Board of Directors to establish the number of directors;
|
• |
require super-majority voting to amend some provisions in our restated certificate of incorporation and restated bylaws;
|
• |
prohibit stockholder action by written consent, which requires all stockholder actions to be taken at a meeting of our stockholders; and
|
• |
establish advance notice requirements for nominations for election to our board or for proposing matters that can be acted upon by stockholders at annual stockholder meetings.
|
Item 1B. |
Unresolved SEC Comments
|
Item 2. |
Properties
|
Item 3. |
Legal Proceedings
|
Item 4. |
Mine Safety Disclosures
|
Item 5. |
Market for Registrant’s Common Equity, Related Stockholder Matters and Issuer Purchases of Equity Securities Market Information
|
Item 6. |
Reserved
|
Item 7. |
Management’s Discussion and Analysis of Financial Condition and Results of Operations
|
•
|
expenses incurred under agreements with research institutions that conduct R&D activities including, process development, preclinical, and clinical activities on our behalf;
|
• |
costs related to process development, production of preclinical and clinical materials, including fees paid to contract manufacturers and manufacturing input costs for use in internal manufacturing processes;
|
• |
consultants supporting process development and regulatory activities;
|
• |
patent fees; and
|
• |
costs related to in-licensing of rights to develop and commercialize our product candidate portfolio.
|
• |
salaries and personnel-related costs, including benefits, travel, and stock-based compensation, for our scientific personnel performing R&D activities;
|
• |
facilities and other expenses, which include expenses for rent and maintenance of facilities, and depreciation expense; and
|
• |
laboratory supplies and equipment used for internal R&D activities.
|
• |
the scope, rate of progress, and expense of ongoing as well as any clinical studies and other R&D activities that we undertake;
|
• |
future clinical study results;
|
• |
uncertainties in clinical study enrollment rates;
|
• |
changing standards for regulatory approval; and
|
• |
the timing and receipt of any regulatory approvals.
|
• |
the scope, progress, outcome and costs of our clinical trials and other R&D activities;
|
• |
the efficacy and potential advantages of our product candidates compared to alternative treatments, including any standard of care;
|
• |
the market acceptance of our product candidates;
|
• |
obtaining, maintaining, defending, and enforcing patent claims and other intellectual property rights;
|
• |
significant and changing government regulation; and
|
• |
the timing, receipt, and terms of any marketing approvals.
|
For the Years Ended December 31,
|
||||||||||||
2021
|
2020
|
Change
|
||||||||||
(in thousands)
|
||||||||||||
Operating expenses:
|
||||||||||||
Research and development
|
$
|
125,476
|
$
|
105,438
|
$
|
20,038
|
||||||
General and administrative
|
41,772
|
28,865
|
12,907
|
|||||||||
Total operating expenses
|
167,248
|
134,303
|
32,945
|
|||||||||
Loss from operations
|
(167,248
|
)
|
(134,303
|
)
|
(32,945
|
)
|
||||||
Research and development incentives
|
1,000
|
-
|
1,000
|
|||||||||
Interest expense
|
(2,977
|
)
|
(6,967
|
)
|
3,990
|
|||||||
Interest and other income, net
|
3,068
|
2,150
|
918
|
|||||||||
(Amortization of premium) accretion of discount on investments - net
|
(2,912
|
)
|
(580
|
)
|
(2,332
|
)
|
||||||
Total other expense, net
|
(1,821
|
)
|
(5,397
|
)
|
3,576
|
|||||||
Net loss
|
$
|
(169,069
|
)
|
$
|
(139,700
|
)
|
$
|
(29,369
|
)
|
For the Years Ended December 31,
|
||||||||||||
2020
|
2019
|
Change
|
||||||||||
(in thousands)
|
||||||||||||
Operating expenses:
|
||||||||||||
Research and development
|
$
|
105,438
|
$
|
57,907
|
$
|
47,531
|
||||||
General and administrative
|
28,865
|
18,244
|
10,621
|
|||||||||
Total operating expenses
|
134,303
|
76,151
|
58,152
|
|||||||||
Loss from operations
|
(134,303
|
)
|
(76,151
|
)
|
(58,152
|
)
|
||||||
Research and development incentives
|
-
|
250
|
(250
|
)
|
||||||||
Interest expense
|
(6,967
|
)
|
(5,958
|
)
|
(1,009
|
)
|
||||||
Interest and other income, net
|
2,150
|
3,414
|
(1,264
|
)
|
||||||||
(Amortization of premium) accretion of discount on investments - net
|
(580
|
)
|
1,175
|
(1,755
|
)
|
|||||||
Total other expense, net
|
(5,397
|
)
|
(1,119
|
)
|
(4,278
|
)
|
||||||
Net loss
|
$
|
(139,700
|
)
|
$
|
(77,270
|
)
|
$
|
(62,430
|
)
|
For the Years Ended December 31,
|
||||||||||||
2021
|
2020
|
2019
|
||||||||||
Cash used in operating activities
|
$
|
(121,163
|
)
|
$
|
(74,640
|
)
|
$
|
(64,663
|
)
|
|||
Cash provided by (used in) investing activities
|
18,853
|
(96,591
|
)
|
(39,011
|
)
|
|||||||
Cash provided by financing activities
|
37,681
|
282,989
|
177,791
|
|||||||||
Net change in cash, cash equivalents and restricted cash
|
$
|
(64,629
|
)
|
$
|
111,758
|
$
|
74,117
|
• |
CROs in connection with performing R&D services on our behalf;
|
• |
investigative sites or other providers in connection with clinical trials;
|
• |
vendors in connection with non-clinical development activities; and
|
• |
vendors related to product manufacturing, development and distribution of clinical supplies.
|
• |
Pertain to the maintenance of records that in reasonable detail accurately and fairly reflect the transactions and dispositions of our assets;
|
• |
Provide reasonable assurance that transactions are recorded as necessary to permit preparation of financial statements in accordance with generally accepted accounting principles, and that our receipts and expenditures are being made
only in accordance with authorizations of our management and directors; and
|
• |
Provide reasonable assurance regarding prevention or timely detection of unauthorized acquisition, use or disposition of our assets that could have a material effect on the financial statements.
|
Item 12.
|
Security Ownership of Certain Beneficial Owners and Management and Related Stockholder Matters
|
Reports of Independent Registered Public Accounting Firm
|
F-2
|
Consolidated Balance Sheets as of December 31, 2021 and 2020
|
F-4
|
Consolidated Statements of Operations for the Years Ended December 31, 2021, 2020 and 2019
|
F-5
|
Consolidated Statements of Comprehensive Loss for the Years Ended December 31, 2021, 2020 and 2019
|
F-6
|
Consolidated Statements of Changes in Stockholders’ Equity for the Years Ended December 31, 2021, 2020 and 2019
|
F-7
|
Consolidated Statements of Cash Flows for the Years Ended December 31, 2021, 2020 and 2019
|
F-8
|
Notes to Consolidated Financial Statements
|
F-9
|
Exhibit
Number |
Exhibit Index
Description of Exhibit |
Agreement and Plan of Merger and Reorganization, dated as of September 12, 2017, by and among Inotek Pharmaceuticals Corporation, Rocket Pharmaceuticals, Ltd. and Rome Merger Sub (Filed as Exhibit 2.1
to the Company’s Current Report on Form 8-K (001-36829), filed with the SEC on September 13, 2017, and incorporated herein by reference)
|
|
Seventh Amended and Restated Certificate of Incorporation of Rocket Pharmaceuticals, Inc., effective as of February 23, 2015 (Filed as Exhibit 3.1 to the Company’s Annual Report on Form 10-K
(001-36829), filed with the SEC on March 31, 2015, and incorporated herein by reference)
|
|
Certificate of Amendment (Reverse Stock Split) to the Seventh Amended and Restated Certificate of Incorporation of the Registrant, effective as of January 4, 2018 (Filed as Exhibit 3.1 to the Company’s
Current Report on Form 8-K (001-36829), filed with the SEC on January 5, 2018, and incorporated herein by reference)
|
|
Certificate of Amendment (Name Change) to the Seventh Amended and Restated Certificate of Incorporation of the Registrant, effective January 4, 2018 (Filed as Exhibit 3.2 to the Company’s Current Report
on Form 8-K (001-36829), filed with the SEC on January 5, 2018, and incorporated herein by reference)
|
|
Certificate of Amendment to the Seventh Amended and Restated Certificate of Incorporation of the Registrant, effective June 25, 2018 (Filed as Exhibit 3.1 to the Company’s Current Report on Form 8-K
(001-36829), filed with the SEC on June 25, 2019, and incorporated herein by reference)
|
|
Amended and Restated By-Laws of Rocket Pharmaceuticals, Inc., effective as of March 29, 2018 (Filed as Exhibit 3.4 to the Company’s registration statement on Form 8-A/A, as amended (001-36829), filed
with the SEC on January 11, 2018, and incorporated herein by reference)
|
|
Form of Common Stock Certificate of Rocket Pharmaceuticals, Inc. (Filed as Exhibit 4.1 to the Company’s Current Report on Form 8-K (001-36829), filed with the SEC on January 5, 2018, and incorporated
herein by reference)
|
|
Description of Securities (Filed as Exhibit 4.8 to the Company’s Annual Report on Form 10-K (001-36829), filed with the SEC on March 1, 2021, and incorporated herein by reference)
|
|
2004 Stock Option and Incentive Plan (Filed as Exhibit 10.1 to the Company’s Registration Statement on Form S-1 (333-199859), filed with the SEC on November 5, 2014, as amended, and incorporated herein
by reference)
|
|
Rocket Pharmaceuticals, Inc. Second Amended and Restated 2014 Stock Option and Incentive Plan (Filed as Exhibit 10.1 to the Company’s Current Report on Form 8-K
(001-36829), filed with the SEC on June 25, 2018, and incorporated herein by reference)
|
|
Form of Incentive Stock Option Agreement (Employees) (Filed as Exhibit 10.2 to the Company’s Quarterly Report on Form 10-Q (001-36829), filed with the SEC on August 14, 2018, and incorporated herein by
reference)
|
|
Form of Non-Qualified Stock Option Agreement (Employees) (Filed as Exhibit 10.3 to the Company’s Quarterly Report on Form 10-Q (001-36829), filed with the SEC on August 14, 2018, and incorporated herein
by reference)
|
|
Form of Non-Qualified Stock Option Agreement (Non-Employee Directors) (Filed as Exhibit 10.4 to the Company’s Quarterly Report on Form 10-Q (001-36829), filed with the SEC on August 14, 2018, and
incorporated herein by reference)
|
|
Form of Non-Qualified Stock Option Agreement (Consultants) (Filed as Exhibit 10.5 to the Company’s Quarterly Report on Form 10-Q (001-36829), filed with the SEC on August 14, 2018, and incorporated
herein by reference)
|
|
Form of Restricted Stock Unit Award Agreement (Filed as Exhibit 10.6.1 to the Company’s Annual Report on Form 10-K (001-36829), filed with the SEC on March 1, 2021, and incorporated herein by reference)
|
|
Rocket Pharmaceuticals, Ltd. 2015 Share Option Plan (Filed as Exhibit 10.3 to the Company’s Annual Report on Form 10-K (001-36829), filed with the SEC on March 7, 2018, and incorporated herein by
reference)
|
|
Letter Agreement, dated as of July 28, 2014, by and between the Registrant and David P. Southwell (Filed as Exhibit 10.3 to the Company’s Registration Statement on Form S-1 (333-199859), filed with the
SEC on November 5, 2014, as amended, and incorporated herein by reference)
|
|
Amendment to Offer Letter, effective as of September 1, 2017, by and between Inotek and David Southwell (Filed as Exhibit 10.2 to the Company’s Current Report on Form 8-K
(001-36829), filed with the SEC on September 1, 2017, and incorporated herein by reference)
|
|
Offer Letter, dated September 29, 2017, by and between the Registrant and Raj Prabhakar.
|
|
Offer Letter, dated November 25, 2020, by and between the registrant and Carlos Garcia-Parada. (Filed as Exhibit 10.11 to the Company’s Annual Report on Form 10-K (001-36829), filed with the SEC on
March 1, 2021, and incorporated herein by reference)
|
|
Offer Letter, dated April 29, 2021, by and between the registrant and Isabel Carmona.
|
|
Amended and Restated Lease Agreement, dated as of June 26, 2019, by and between Rocket Pharmaceuticals, Inc. and Cedar Brook 12 Corporate Center, L.P. (Filed as Exhibit 10.1 to the Company’s Quarterly
Report on Form 10-Q (001-36829), filed with the SEC on August 8, 2018, and incorporated herein by reference)
|
|
Rocket Pharmaceuticals, Inc. Amended and Restated 2014 Employee Stock Purchase Plan (Filed as Exhibit 10.10 to the Company’s Annual Report on Form 8-K (001-36829), filed with the SEC on March 7, 2018,
and incorporated herein by reference)
|
|
Form of Indemnification Agreement, to be entered into between the Registrant and its directors (Filed as Exhibit 10.1 to the Company’s Current Report on Form 8-K (001-36829), filed with the SEC on
January 5, 2018, and incorporated herein by reference)
|
|
Form of Indemnification Agreement, to be entered into between the Registrant and its officers (Filed as Exhibit 10.2 to the Company’s Current Report on Form 8-K (001-36829), filed with the SEC on
January 5, 2018, and incorporated herein by reference)
|
|
Form of Severance and Change of Control Agreements, to be entered into between the Registrant and certain of its officers (Filed as Exhibit 10.16 to the Company’s Annual Report on Form 10-K (001-36829),
filed with the SEC on March 1, 2021, and incorporated herein by reference)
|
Agreement of Lease, dated as of June 6, 2018, by and between Rocket Pharmaceuticals, Inc. and ESRT Empire State Building, L.L.C., (Filed as Exhibit 10.6 to the Company’s Quarterly Report on Form 10-Q
(001-36829), filed with the SEC on August 14, 2018, and incorporated herein by reference)
|
|
License Agreement, dated as of November 19, 2018, by and between Rocket Pharmaceuticals, Ltd. and REGENXBIO Inc. (Filed as Exhibit 10.26 to the Company’s Annual Report on Form 10-K (001-36829), filed
with the SEC on March 8, 2019, and incorporated herein by reference)
|
|
Warrant to Purchase Shares of Common Stock, dated as of December 21, 2020, by and between the Registrant and Neptune Consulting, LLC. (Filed as Exhibit 10.23 to the Company’s Annual Report on Form 10-K
(001-36829), filed with the SEC on March 1, 2021, and incorporated herein by reference)
|
|
Warrant to Purchase Shares of Series Preferred Stock, dated as of June 28, 2013, by and between Inotek Pharmaceuticals Corporation and Horizon Technology Finance Corporation (Filed as Exhibit 10.24 to
the Company’s Annual Report on Form 10-K (001-36829), filed with the SEC on March 1, 2021, and incorporated herein by reference)
|
|
Warrant to Purchase Shares of Series Preferred Stock dated as of June 28, 2013, by and between Inotek Pharmaceuticals Corporation and Fortress Credit Co LLC (Filed as Exhibit 10.25 to the Company’s
Annual Report on Form 10-K (001-36829), filed with the SEC on March 1, 2021, and incorporated herein by reference)
|
|
Warrant to Purchase Shares of Common Stock, dated as of December 17, 2021, by and between the Registrant and Neptune Consulting, LLC. (First Indication)
|
|
Warrant to Purchase Shares of Common Stock, dated as of December 17, 2021, by and between the Registrant and Neptune Consulting, LLC. (Second Indication)
|
|
Securities Purchase Agreement, dated as of August 27, 2021, by and among Rocket Pharmaceuticals, Inc., and each of those persons listed as a Purchaser on the Schedule of Purchasers attached as Schedule
I thereto (Filed as Exhibit 10.1 to the Company’s Current Report on Form 8-K (001-36829), filed with the SEC on August 30, 2021, and incorporated herein by reference)
|
|
Registration Rights Agreement, dated as of August 27, 2021, by and among Rocket Pharmaceuticals, Inc., and each of those persons listed as an Investor on the Schedule of Inventors attached as Schedule A
thereto (Filed as Exhibit 10.2 to the Company’s Current Report on Form 8-K (001-36829), filed with the SEC on August 30, 2021, and incorporated herein by reference).
|
|
List of Subsidiaries
|
|
Consent of EisnerAmper LLP
|
|
Power of Attorney (included in the signature page)
|
|
Certification of Principal Executive Officer pursuant to Rule 13a-14(a) or Rule 15d-14(a) of the Securities Exchange Act of 1934, as adopted pursuant to Section 302 of the Sarbanes-Oxley Act of 2002
|
|
Certification of Principal Financial Officer pursuant to Rule 13a-14(a) or Rule 15d-14(a) of the Securities Exchange Act of 1934, as adopted pursuant to Section 302 of the Sarbanes-Oxley Act of 2002
|
|
Certification of Principal Executive Officer and Principal Financial Officer pursuant to 18 U.S.C. Section 1350, as adopted pursuant to Section 906 of the Sarbanes-Oxley Act of 2002
|
|
101.INS
|
Inline XBRL Instance Document.
|
101.SCH
|
Inline XBRL Taxonomy Extension Schema Document.
|
101.CAL
|
Inline XBRL Taxonomy Extension Calculation Document.
|
101.DEF
|
Inline XBRL Taxonomy Extension Definition Linkbase Document.
|
101.LAB
|
Inline XBRL Taxonomy Extension Labels Linkbase Document.
|
101.PRE
|
Inline XBRL Taxonomy Extension Presentation Link Document.
|
104
|
Cover Page Interactive Data File (formatted as inline XBRL and contained in exhibit 101)
|
*
|
Filed herewith.
|
#
|
Indicates management contract or compensatory plan.
|
†
|
Confidential treatment has been granted with respect to certain portions of this exhibit. Omitted portions have been filed separately with the SEC.
|
**
|
Certain portions of this exhibit have been excluded because they are both not material and would likely cause competitive harm to the Company if publicly disclosed.
|
† |
The certification attached as Exhibit 32.1 accompanying this Annual Report on Form 10- K is not deemed filed with the Securities and Exchange Commission and is not to be incorporated by reference into any filing of Rocket
Pharmaceuticals, Inc. under the Securities Act of 1933, as amended, or the Securities Exchange Act of 1934, as amended, whether made before or after the date of this Annual Report on Form 10-K, irrespective of any general incorporation
language contained in such filing.
|
|
Rocket Pharmaceuticals, Inc.
|
|
|
By: |
/s/ Gaurav Shah, MD
|
|
Gaurav Shah, MD
|
|
President and Chief Executive Officer
|
Name |
Title
|
Date
|
||
/s/ Gaurav Shah, MD
|
Chief Executive Officer and Director
|
February 28, 2022
|
||
Gaurav Shah, MD
|
(Principal Executive Officer)
|
|||
/s/ Carlos Garcia-Parada, MBA
|
Chief Financial Officer
|
February 28, 2022
|
||
Carlos Garcia-Parada
|
(Principal Financial Officer)
|
|||
/s/ John C. Militello
|
VP, Finance, Senior Controller & Treasurer
|
February 28, 2022
|
||
John C. Militello
|
(Principal Accounting Officer)
|
|||
/s/ Carsten Boess
|
Director
|
February 28, 2022
|
||
Carsten Boess
|
||||
/s/ Pedro Granadillo
|
Director
|
February 28, 2022
|
||
Pedro Granadillo
|
||||
/s/ Gotham Makker, MD
|
Director
|
February 28, 2022
|
||
Gotham Makker, MD
|
||||
/s/ David P. Southwell
|
Director
|
February 28, 2022
|
||
David P. Southwell
|
||||
/s/ Roderick Wong, MD
|
Director
|
February 28, 2022
|
||
Roderick Wong, MD
|
||||
/s/ Naveen Yalamanchi, MD
|
Director
|
February 28, 2022
|
||
Naveen Yalamanchi, MD
|
||||
/s/ Elisabeth Björk
|
Director
|
February 28, 2022
|
||
Elisabeth Björk
|
Reports of Independent Registered Public Accounting Firm (PCAOB ID
) |
F-2
|
Consolidated Balance Sheets as of December 31, 2021 and 2020
|
F-4
|
Consolidated Statements of Operations for the Years Ended December 31, 2021, 2020 and 2019
|
F-5
|
Consolidated Statements of Comprehensive Loss for the Years Ended December 31, 2021, 2020 and 2019
|
F-6
|
Consolidated Statements of Changes in Stockholders’ Equity for the Years Ended December 31, 2021, 2020 and 2019
|
F-7
|
Consolidated Statements of Cash Flows for the Years Ended December 31, 2021, 2020 and 2019
|
F-8
|
Notes to Consolidated Financial Statements
|
F-9
|
December 31,
2021
|
December 31,
2020
|
|||||||
Assets
|
||||||||
Current assets:
|
||||||||
Cash and cash equivalents
|
$
|
232,694
|
$
|
297,098
|
||||
Investments
|
156,046
|
185,621
|
||||||
Prepaid expenses and other current assets
|
3,319
|
4,626
|
||||||
Total current assets
|
392,059
|
487,345
|
||||||
Property and equipment, net
|
22,299
|
19,206
|
||||||
Goodwill
|
30,815
|
30,815
|
||||||
Restricted cash
|
1,343
|
1,568
|
||||||
Deposits
|
455
|
455
|
||||||
Operating lease right-of-use assets
|
1,569
|
914
|
||||||
Finance lease right-of-use asset
|
48,480
|
50,521
|
||||||
Total assets
|
$
|
497,020
|
$
|
590,824
|
||||
Liabilities and stockholders’ equity
|
||||||||
Current liabilities:
|
||||||||
Accounts payable and accrued expenses
|
$
|
19,615
|
$
|
25,472
|
||||
Convertible notes, net of unamortized discount, current
|
-
|
4,875
|
||||||
Operating lease liabilities, current
|
863
|
626
|
||||||
Finance lease liability, current
|
1,689
|
1,644
|
||||||
Total current liabilities
|
22,167
|
32,617
|
||||||
Convertible notes, net of unamortized discount, non-current
|
-
|
35,066
|
||||||
Operating lease liabilities, non-current
|
905
|
498
|
||||||
Finance lease liability, non-current
|
19,144
|
18,988
|
||||||
Other liabilities
|
80
|
136
|
||||||
Total liabilities
|
42,296
|
87,305
|
||||||
Commitments and contingencies (Note 12)
|
||||||||
Stockholders’ equity:
|
||||||||
Preferred stock, $0.01 par value, authorized 5,000,000 shares:
|
||||||||
Series A convertible preferred stock; 300,000 shares designated as Series A; 0 shares
issued and outstanding
|
-
|
-
|
||||||
Series B convertible preferred stock; 300,000 shares designated as Series B; 0 shares
issued and outstanding
|
-
|
-
|
||||||
Common stock, $0.01 par value, 120,000,000 shares authorized; 64,505,889 and 60,996,367 shares issued and
outstanding at December 31, 2021 and 2020, respectively
|
645
|
610
|
||||||
Additional paid-in capital
|
946,152
|
825,794
|
||||||
Accumulated other comprehensive loss
|
(161
|
)
|
(42
|
)
|
||||
Accumulated deficit
|
(491,912
|
)
|
(322,843
|
)
|
||||
Total stockholders’ equity
|
454,724
|
503,519
|
||||||
Total liabilities and stockholders’ equity
|
$
|
497,020
|
$
|
590,824
|
For the Years Ended December 31,
|
||||||||||||
2021
|
2020
|
2019
|
||||||||||
Revenue
|
$
|
-
|
$
|
-
|
$
|
-
|
||||||
Operating expenses:
|
||||||||||||
Research and development
|
125,476
|
105,438
|
57,907
|
|||||||||
General and administrative
|
41,772
|
28,865
|
18,244
|
|||||||||
Total operating expenses
|
167,248
|
134,303
|
76,151
|
|||||||||
Loss from operations
|
(167,248
|
)
|
(134,303
|
)
|
(76,151
|
)
|
||||||
Research and development incentives
|
1,000
|
-
|
250
|
|||||||||
Interest expense
|
(2,977
|
)
|
(6,967
|
)
|
(5,958
|
)
|
||||||
Interest and other income, net
|
3,068
|
2,150
|
3,414
|
|||||||||
(Amortization of premium) accretion of discount on investments - net
|
(2,912
|
)
|
(580
|
)
|
1,175
|
|||||||
Net loss
|
$
|
(169,069
|
)
|
$
|
(139,700
|
)
|
$
|
(77,270
|
)
|
|||
Net loss per share attributable to common stockholders - basic and diluted
|
$
|
(2.67
|
)
|
$
|
(2.52
|
)
|
$
|
(1.58
|
)
|
|||
Weighted-average common shares outstanding - basic and diluted
|
63,235,417
|
55,380,740
|
49,010,358
|
For the Years Ended December 31,
|
||||||||||||
2021
|
2020
|
2019
|
||||||||||
Net loss
|
$
|
(169,069
|
)
|
$
|
(139,700
|
)
|
$
|
(77,270
|
)
|
|||
Other comprehensive loss
|
||||||||||||
Net unrealized (loss) gain on investments
|
(119
|
)
|
(62
|
)
|
147
|
|||||||
Total comprehensive loss
|
$
|
(169,188
|
)
|
$
|
(139,762
|
)
|
$
|
(77,123
|
)
|
Common Stock
|
Additional
|
Accumulated
Other
|
Total
|
|||||||||||||||||||||||||
Shares
|
Amount
|
Treasury
Stock
|
Paid-In
Capital
|
Comprehensive
Income/(Loss)
|
Accumulated
Deficit
|
Stockholders’
Equity
|
||||||||||||||||||||||
Balance at December 31, 2018
|
45,194,736
|
$ |
452
|
$ |
(668
|
)
|
$ |
300,253
|
$ |
(127
|
)
|
$ |
(105,873
|
)
|
$ |
194,037
|
||||||||||||
Issuance of common stock, net of issuance costs
|
9,568,000
|
96
|
-
|
177,664
|
-
|
-
|
177,760
|
|||||||||||||||||||||
Issuance of common stock pursuant to exercise of stock options
|
110,325
|
1
|
-
|
30
|
-
|
-
|
31
|
|||||||||||||||||||||
Treasury stock purchases
|
-
|
-
|
(725
|
)
|
(1
|
)
|
-
|
-
|
(726
|
)
|
||||||||||||||||||
Issuance of treasury stock pursuant to exercise of stock options
|
-
|
-
|
(397
|
)
|
-
|
-
|
-
|
(397
|
)
|
|||||||||||||||||||
Retirement of treasury stock
|
(100,000
|
)
|
(1
|
)
|
1,393
|
(1,392
|
)
|
-
|
-
|
-
|
||||||||||||||||||
Sale of treasury stock
|
-
|
-
|
344
|
-
|
-
|
-
|
344
|
|||||||||||||||||||||
Unrealized comprehensive gain on investments
|
-
|
-
|
-
|
-
|
147
|
-
|
147
|
|||||||||||||||||||||
Share-based compensation
|
-
|
-
|
-
|
13,371
|
-
|
-
|
13,371
|
|||||||||||||||||||||
Net loss
|
-
|
-
|
-
|
-
|
-
|
(77,270
|
)
|
(77,270
|
)
|
|||||||||||||||||||
Balance at December 31, 2019
|
54,773,061
|
548
|
(53
|
)
|
489,925
|
20
|
(183,143
|
)
|
307,297
|
|||||||||||||||||||
Issuance of common stock, net of issuance costs
|
5,339,286
|
53
|
-
|
280,710
|
-
|
-
|
280,763
|
|||||||||||||||||||||
Issuance of common stock pursuant to exercise of stock options
|
586,857
|
6
|
-
|
2,552
|
-
|
-
|
2,558
|
|||||||||||||||||||||
Issuance of common stock pursuant to exercise of warrant
|
1,601
|
-
|
-
|
-
|
-
|
-
|
-
|
|||||||||||||||||||||
Issuance of common stock pursuant to conversion of notes
|
298,562
|
3
|
-
|
7,626
|
-
|
-
|
7,629
|
|||||||||||||||||||||
Issuance of warrants
|
-
|
-
|
-
|
26,562
|
-
|
-
|
26,562
|
|||||||||||||||||||||
Stock repurchase
|
(3,000
|
)
|
-
|
-
|
(72
|
)
|
-
|
-
|
(72
|
)
|
||||||||||||||||||
Sale of treasury stock
|
-
|
-
|
667
|
(76
|
)
|
-
|
-
|
591
|
||||||||||||||||||||
Issuance of treasury stock pursuant to exercise of stock options
|
-
|
-
|
(614
|
)
|
-
|
-
|
-
|
(614
|
)
|
|||||||||||||||||||
Unrealized comprehensive loss on marketable securities
|
-
|
-
|
-
|
-
|
(62
|
)
|
-
|
(62
|
)
|
|||||||||||||||||||
Share-based compensation
|
-
|
-
|
-
|
18,567
|
-
|
- |
18,567
|
|||||||||||||||||||||
Net loss
|
-
|
-
|
-
|
-
|
-
|
(139,700
|
)
|
(139,700
|
)
|
|||||||||||||||||||
Balance at December 31, 2020
|
60,996,367
|
|
610
|
|
-
|
|
825,794
|
|
(42
|
)
|
|
(322,843
|
)
|
|
503,519
|
|||||||||||||
Issuance of common stock pursuant to exercise of stock options
|
1,209,960 | 12 | - | 11,315 | - | - | 11,327 | |||||||||||||||||||||
Issuance of common stock pursuant to conversion of notes
|
1,487,046 | 15 | - | 40,679 | - | - | 40,694 | |||||||||||||||||||||
Issuance of common stock, net of issuance costs
|
812,516 | 8 | - | 26,346 | - | - | 26,354 | |||||||||||||||||||||
Issuance of warrants
|
- | - | - | 12,781 | - | - | 12,781 | |||||||||||||||||||||
Unrealized comprehensive loss on investments
|
- | - | - | - | (119 | ) | - | (119 | ) | |||||||||||||||||||
Share-based compensation
|
- | - | - | 29,237 | - | - | 29,237 | |||||||||||||||||||||
Net loss
|
- | - | - | - | - | (169,069 | ) | (169,069 | ) | |||||||||||||||||||
Balance at December 31, 2021
|
64,505,889 | $ | 645 | $ | - | $ | 946,152 | $ | (161 | ) | $ | (491,912 | ) | $ | 454,724 |
For the Years Ended December 31,
|
||||||||||||
2021
|
2020
|
2019
|
||||||||||
Operating Activities:
|
||||||||||||
Net loss
|
$
|
(169,069
|
)
|
$
|
(139,700
|
)
|
$
|
(77,270
|
)
|
|||
Adjustments to reconcile net loss to net cash used in operating activities:
|
||||||||||||
Accretion of discount on convertible notes
|
753
|
2,758
|
3,602
|
|||||||||
Depreciation and amortization of property and equipment
|
3,240
|
1,145
|
426
|
|||||||||
Amortization of right of use asset
|
2,133 | 5,105 | - | |||||||||
Write down of property and equipment, net
|
261
|
419
|
-
|
|||||||||
Stock-based compensation
|
29,237
|
18,567
|
13,371
|
|||||||||
Expense in connection with warrant issuances
|
12,781
|
26,562
|
-
|
|||||||||
Accretion of discount (amortization of premium) on investments, net
|
2,887
|
580
|
(1,066
|
)
|
||||||||
Changes in operating assets and liabilities:
|
||||||||||||
Prepaid expenses and other assets
|
1,307
|
(1,517
|
)
|
(917
|
)
|
|||||||
Accounts payable and accrued expenses
|
(4,827
|
)
|
11,015
|
(2,722
|
)
|
|||||||
Operating lease liabilities
|
(11
|
)
|
(139
|
)
|
(87
|
)
|
||||||
Finance lease liability
|
201
|
452
|
-
|
|||||||||
Other long term liabilities
|
(56
|
)
|
113
|
-
|
||||||||
Net cash used in operating activities
|
(121,163
|
)
|
(74,640
|
)
|
(64,663
|
)
|
||||||
Investing activities:
|
||||||||||||
Purchases of investments
|
(245,875
|
)
|
(209,343
|
)
|
(184,298
|
)
|
||||||
Proceeds from maturities of investments
|
272,443
|
141,811
|
168,556
|
|||||||||
Payments made to acquire right of use asset
|
(95
|
)
|
(8,452
|
)
|
-
|
|||||||
Purchases of property and equipment
|
(7,620
|
)
|
(20,607
|
)
|
(23,269
|
)
|
||||||
Net cash provided by (used in) investing activities
|
18,853
|
(96,591
|
)
|
(39,011
|
)
|
|||||||
Financing activities:
|
||||||||||||
Issuance of common stock, net of issuance costs
|
26,354
|
280,763
|
177,760
|
|||||||||
Issuance of common stock, pursuant to exercise of stock options
|
11,327
|
2,558
|
31
|
|||||||||
Common stock repurchase
|
-
|
(72
|
)
|
-
|
||||||||
Proceeds from sale of treasury stock
|
-
|
591
|
344
|
|||||||||
Payment of withholding tax on option exercises
|
-
|
(614
|
)
|
(344
|
)
|
|||||||
Convertible notes refinancing costs to the lender
|
-
|
(237
|
)
|
-
|
||||||||
Net cash provided by financing activities
|
37,681
|
282,989
|
177,791
|
|||||||||
Net change in cash, cash equivalents and restricted cash
|
(64,629
|
)
|
111,758
|
74,117
|
||||||||
Cash, cash equivalents and restricted cash at beginning of period
|
298,666
|
186,908
|
112,791
|
|||||||||
Cash, cash equivalents and restricted cash at end of period
|
$
|
234,037
|
$
|
298,666
|
$
|
186,908
|
||||||
Supplemental disclosure of non-cash financing and investing activities:
|
||||||||||||
Accrued purchases of property and equipment
|
$
|
728
|
$
|
1,756
|
$
|
4,650
|
||||||
Retirement of treasury stock
|
$
|
-
|
$
|
-
|
$
|
1,393
|
||||||
Treasury stock purchases paid in prior year
|
$
|
-
|
$
|
-
|
$
|
726
|
||||||
Withholding tax payable on shares withheld in treasury stock
|
$
|
-
|
$
|
-
|
$
|
53
|
||||||
Unrealized (loss) gain on investments
|
$
|
(119
|
)
|
$
|
(62
|
)
|
$
|
147
|
||||
Conversion of 2021 and 2022 convertible notes into common stock |
$ | 40,694 | $ | 7,629 | $ | - | ||||||
Finance lease right of use asset and lease liability
|
$
|
-
|
$
|
20,179
|
$
|
-
|
||||||
Reclassification of construction in process to finance right of use asset
|
$
|
98
|
$
|
26,465
|
$
|
-
|
||||||
Supplemental cash flow information:
|
||||||||||||
Cash paid for interest
|
$
|
148
|
$
|
2,960
|
$
|
2,990
|
||||||
Cash paid for income taxes
|
$
|
-
|
$
|
-
|
$
|
26
|
1. |
Nature of Business and Basis of Presentation
|
2. |
Risks and Liquidity
|
3. |
Summary of Significant Accounting Policies
|
December 31,
2021
|
December 31,
2020
|
|||||||
Cash and cash equivalents
|
$
|
232,694
|
$
|
297,098
|
||||
Restricted cash
|
1,343
|
1,568
|
||||||
$
|
234,037
|
$
|
298,666
|
4. |
Fair Value of Financial Instruments
|
Fair Value Measurements as of
December 31, 2021
Using:
|
||||||||||||||||
Level 1
|
Level 2
|
Level 3
|
Total
|
|||||||||||||
Assets:
|
||||||||||||||||
Cash equivalents:
|
||||||||||||||||
Money market mutual funds
|
$
|
179,900
|
$
|
-
|
$
|
-
|
$
|
179,900
|
||||||||
179,900
|
-
|
-
|
179,900
|
|||||||||||||
Investments:
|
||||||||||||||||
United States Treasury securities
|
44,045
|
-
|
-
|
44,045
|
||||||||||||
Corporate Bonds
|
-
|
96,696
|
-
|
96,696
|
||||||||||||
Municipal Bonds
|
-
|
6,000
|
-
|
6,000
|
||||||||||||
Agency Bonds
|
-
|
9,305
|
-
|
9,305
|
||||||||||||
44,045
|
112,001
|
-
|
156,046
|
|||||||||||||
$
|
223,945
|
$
|
112,001
|
$
|
-
|
$
|
335,946
|
Fair Value Measurements as of
December 31, 2020
Using:
|
||||||||||||||||
Level 1
|
Level 2
|
Level 3
|
Total
|
|||||||||||||
Assets:
|
||||||||||||||||
Cash equivalents:
|
||||||||||||||||
Money market mutual funds
|
$
|
193,312
|
$
|
-
|
$
|
-
|
$
|
193,312
|
||||||||
United States Treasury securities
|
62,497 | - | - | 62,497 | ||||||||||||
Corporate Bonds
|
- | 501 | - | 501 | ||||||||||||
Agency Bonds
|
- | 8,015 | - | 8,015 | ||||||||||||
255,809
|
8,516
|
-
|
264,325
|
|||||||||||||
Investments:
|
||||||||||||||||
United States Treasury securities
|
112,328
|
-
|
-
|
112,328
|
||||||||||||
Corporate Bonds
|
-
|
63,710
|
-
|
63,710
|
||||||||||||
Municipal Bonds
|
-
|
6,000
|
-
|
6,000
|
||||||||||||
Agency Bonds
|
-
|
3,583
|
-
|
3,583
|
||||||||||||
112,328
|
73,293
|
-
|
185,621
|
|||||||||||||
$
|
368,137
|
$
|
81,809
|
$
|
-
|
$
|
449,946
|
5. |
Property and Equipment, Net
|
December 31,
2021
|
December 31,
2020
|
|||||||
Laboratory equipment
|
$
|
12,600
|
$
|
7,807
|
||||
Machinery and equipment
|
10,432
|
9,933
|
||||||
Computer equipment
|
218
|
218
|
||||||
Furniture and fixtures
|
1,963
|
1,880
|
||||||
Leasehold improvements
|
407
|
29
|
||||||
Internal use software
|
1,902
|
1,385
|
||||||
27,522
|
21,252
|
|||||||
Less: accumulated depreciation and amortization
|
(5,223
|
)
|
(2,046
|
)
|
||||
$
|
22,299
|
$
|
19,206
|
6. |
Accounts Payable and Accrued Expenses
|
December 31,
2021
|
December 31,
2020
|
|||||||
Research and development
|
$
|
12,082
|
$
|
14,962
|
||||
Property and equipment
|
725
|
1,756
|
||||||
Employee compensation
|
4,533
|
4,875
|
||||||
Accrued interest
|
-
|
1,122
|
||||||
Government grant payable
|
597
|
590
|
||||||
Professional fees
|
1,196
|
1,332
|
||||||
Other
|
482
|
835
|
||||||
$
|
19,615
|
$
|
25,472
|
7. |
Convertible Notes Payable
|
2021 Notes |
2022 Notes |
|||||||||||||||
December 31,
2021
|
December 31,
2020
|
December 31,
2021
|
December 31,
2020
|
|||||||||||||
Principal amount
|
$
|
-
|
$
|
5,150
|
$ | - | $ | 38,350 | ||||||||
Discount
|
-
|
(275
|
)
|
- | (3,284 | ) | ||||||||||
Carrying value
|
$
|
-
|
$
|
4,875
|
$ | - | $ | 35,066 |
8. |
Stockholders’ Equity
|
9. |
Stock-Based Awards
|
Years Ended December 31,
|
||||||||||||
2021
|
2020
|
2019
|
||||||||||
Risk-free interest rate
|
0.83
|
%
|
1.00
|
%
|
2.26
|
%
|
||||||
Expected term (in years)
|
5.84
|
5.84
|
5.81
|
|||||||||
Expected volatility
|
69.27
|
%
|
76.98
|
%
|
75.71
|
%
|
||||||
Expected dividend yield
|
0.00
|
%
|
0.00
|
%
|
0.00
|
%
|
||||||
Exercise price
|
$
|
51.20
|
$
|
22.96
|
$
|
14.47
|
||||||
Fair value of common stock
|
$
|
51.20
|
$
|
22.96
|
$
|
14.47
|
Number of
Shares
|
Weighted
Average
Exercise
Price
|
Weighted
Average
Contractual
Term (Years)
|
Aggregate
Intrinsic
Value
|
|||||||||||||
Outstanding as of December 31, 2019
|
9,763,541
|
$
|
5.95
|
6.96
|
$
|
164,021
|
||||||||||
Granted
|
2,193,546
|
22.96
|
8.97
|
|||||||||||||
Exercised
|
(586,857
|
)
|
4.82
|
13,494
|
||||||||||||
Cancelled
|
(319,299
|
)
|
15.82
|
|||||||||||||
Outstanding as of December 31, 2020
|
11,050,931
|
$
|
9.10
|
6.55
|
$
|
504,079
|
||||||||||
Granted
|
1,671,759
|
51.20
|
8.58
|
|||||||||||||
Exercised
|
(1,209,960
|
)
|
9.32
|
54,487
|
||||||||||||
Cancelled
|
(368,969
|
)
|
35.87
|
|||||||||||||
Outstanding as of December 31, 2021
|
11,143,761
|
$
|
14.51
|
5.95
|
$
|
128,817
|
||||||||||
Options vested and exercisable as of December 31, 2021
|
8,692,898
|
$
|
7.31
|
5.11
|
$
|
127,571
|
||||||||||
Options unvested as of December 31, 2021
|
2,450,863
|
$
|
40.01
|
8.93
|
Years Ended December 31,
|
||||||||||||
2021
|
2020
|
2019
|
||||||||||
Stock options
|
$
|
28,811
|
$
|
18,527
|
$
|
13,371
|
||||||
Restricted stock units
|
426
|
40
|
-
|
|||||||||
Total share based compensation expense
|
$
|
29,237
|
$
|
18,567
|
$
|
13,371
|
Years Ended December 31,
|
||||||||||||
2021
|
2020
|
2019
|
||||||||||
Research and development
|
$
|
11,954
|
$
|
7,355
|
$
|
6,153
|
||||||
General and administrative
|
17,283
|
11,212
|
7,218
|
|||||||||
Total share based compensation expense
|
$
|
29,237
|
$
|
18,567
|
$
|
13,371
|
Number of
Shares
|
Weighted
Average
Grant Date
Fair Value
|
|||||||
Unvested as of December 31, 2019
|
-
|
$ |
-
|
|||||
Granted
|
20,000
|
|
25.06
|
|||||
Exercised
|
- | |||||||
Forfeited
|
- | |||||||
Unvested as of December 31, 2020
|
20,000
|
25.06 | ||||||
Granted
|
3,500 | 62.32 | ||||||
Exercised
|
- | |||||||
Forfeited
|
- | |||||||
Unvested as of December 31, 2021 | 23,500 | 30.61 |
Price
|
Outstanding
|
Grant Date
|
Expiration Date
|
|||
24.42
|
7,051
|
June 28, 2013
|
June 28, 2023
|
|||
57.11
|
603,386
|
December 21, 2020
|
December 21, 2030
|
|||
33.63
|
301,291
|
August 9, 2021
|
August 9, 2031
|
|||
22.51
|
153,155
|
December 17, 2021
|
December 17, 2031
|
|||
22.51
|
153,155
|
December 17, 2021
|
December 17, 2031
|
|||
Total
|
1,218,038
|
|
Number of
Warrant Shares
Outstanding
and Exercisable |
Exercise
Price per
Share
|
||||||
Balance as of December 31, 2019
|
|
14,102
|
$
|
24.42
|
||||
Granted
|
603,386
|
$
|
57.11
|
|||||
Exercised
|
(7,051
|
)
|
$
|
-
|
||||
Balance as of December 31, 2020
|
610,437
|
|||||||
Granted August 2021
|
301,291
|
$
|
33.63
|
|||||
Granted December 2021
|
306,310
|
$
|
22.51
|
|||||
Exercised
|
-
|
$
|
-
|
|||||
Balance as of December 31, 2021
|
1,218,038
|
Years Ended December 31,
|
||||||||
2021
|
2020
|
|||||||
Risk-free interest rate
|
1.37
|
%
|
0.95
|
%
|
||||
Expected term (in years)
|
10.00
|
10.00
|
||||||
Expected volatility
|
70.25
|
%
|
74.20
|
%
|
||||
Expected dividend yield
|
0.00
|
%
|
0.00
|
%
|
||||
Exercise price
|
$
|
28.07
|
$
|
57.11
|
||||
Fair value of common stock
|
$
|
28.07
|
$
|
57.11
|
10. |
Net Loss Per Share
|
For the Years Ended December 31,
|
||||||||||||
2021
|
2020
|
2019
|
||||||||||
Numerator:
|
||||||||||||
Net loss attributable to common stockholders
|
$
|
(169,069
|
)
|
$
|
(139,700
|
)
|
$
|
(77,270
|
)
|
|||
Denominator:
|
||||||||||||
Weighted-average common shares outstanding - basic and diluted
|
63,235,417
|
55,380,740
|
49,010,358
|
|||||||||
Net loss per share attributable to common stockholders - basic and diluted
|
$
|
(2.67
|
)
|
$
|
(2.52
|
)
|
$
|
(1.58
|
)
|
For the Years Ended December 31,
|
||||||||||||
2021
|
2020
|
2019
|
||||||||||
Shares issuable upon conversion of the 2021 Convertible Notes
|
-
|
160,536
|
1,620,948
|
|||||||||
Shares issuable upon conversion of the 2022 Convertible Notes
|
-
|
1,195,449
|
-
|
|||||||||
Warrants exercisable for common shares
|
1,218,038
|
610,437
|
14,102
|
|||||||||
Restricted stock units exercisable for common shares |
23,500 | - | - | |||||||||
Options to purchase common shares
|
11,143,761
|
11,050,931
|
9,608,537
|
|||||||||
12,385,299
|
13,017,353
|
11,243,587
|
11. |
Income Taxes
|
For the years ended December 31,
|
||||||||||||
2021
|
2020
|
2019
|
||||||||||
U.S. federal tax at statutory rate
|
21.0
|
%
|
21.0
|
%
|
21.0
|
%
|
||||||
Foreign rate differential |
(13.0 | )% | 0.0 | % | 0.0 | % | ||||||
Change in state tax apportionment
|
0.1
|
%
|
0.1
|
%
|
(17.4
|
)%
|
||||||
Stock compensation
|
1.5
|
%
|
0.9
|
%
|
(0.7
|
)%
|
||||||
Transfer pricing adjustments
|
(22.8
|
)%
|
0.0
|
%
|
0.0
|
%
|
||||||
Valuation allowance
|
4.6
|
%
|
(35.0
|
)%
|
(5.1
|
)%
|
||||||
Tax credits
|
8.7
|
%
|
13.5
|
%
|
0.0
|
%
|
||||||
Other
|
(0.2
|
)%
|
(0.5
|
)%
|
2.1
|
%
|
||||||
Effective tax rate
|
0
|
%
|
0
|
%
|
0
|
%
|
At December 31,
|
||||||||||||
2021
|
2020
|
2019
|
||||||||||
Deferred income tax assets (liabilities)
|
||||||||||||
R&D Credits
|
$
|
35,766
|
$
|
42,613
|
$
|
18,471
|
||||||
Net operating losses ("NOL") and credit carryforwards
|
26,789
|
21,359
|
2,517
|
|||||||||
Capitalized research and development costs
|
19,753
|
23,179
|
27,652
|
|||||||||
Stock-based compensation
|
11,552
|
7,406
|
3,896
|
|||||||||
Debt discount
|
-
|
(748
|
)
|
(1,460
|
)
|
|||||||
Warrants
|
8,382
|
5,585
|
-
|
|||||||||
Other
|
(8,881
|
)
|
1,189
|
643
|
||||||||
Valuation allowance
|
(93,361
|
)
|
(100,583
|
)
|
(51,719
|
)
|
||||||
Net deferred income tax asset
|
$
|
-
|
$
|
-
|
$
|
-
|
12. |
Commitments and Contingencies
|
Lease cost
|
December 31, 2021
|
|||
Operating lease cost
|
$
|
645
|
||
Finance lease cost
|
||||
Amortization of right of use assets
|
2,140
|
|||
Interest on lease liablities
|
1,845
|
|||
Total lease cost
|
$
|
4,630
|
Maturity of operating lease liabilities
|
December 31, 2021
|
|||
2022
|
917
|
|||
2023
|
548
|
|||
2024
|
269
|
|||
2025 | 64 | |||
2026 | 54 | |||
Total lease payments
|
$
|
1,852
|
||
Less: interest
|
(84
|
)
|
||
Total operating lease liabilities
|
$
|
1,768
|
Maturity of finance lease liability
|
December 31, 2021
|
|||
2022
|
1,689
|
|||
2023
|
1,736
|
|||
2024
|
1,791
|
|||
2025
|
1,856
|
|||
2026 | 1,912 | |||
Thereafter
|
45,000
|
|||
Total lease payments
|
$
|
53,984
|
||
Less: interest
|
(33,151
|
)
|
||
Total finance lease liability
|
$
|
20,833
|
Leases
|
December 31, 2021
|
|||
Operating right-of-use assets
|
$
|
1,569
|
||
Operating current lease liabilities
|
863
|
|||
Operating noncurrent lease liabilities
|
905
|
|||
Total operating lease liabilities
|
$
|
1,768
|
||
Finance right-of-use assets
|
$
|
48,480
|
||
Finance current lease liability
|
1,689
|
|||
Finance noncurrent lease liability
|
19,144
|
|||
Total finance lease liability
|
$
|
20,833
|
13. |
Agreements Related to Intellectual Property
|
14. |
CIRM Grants
|
15. |
Related Party Transactions
|
16. |
401(k) Savings Plan
|
1.
|
Position.
|
2.
|
Compensation.
|
3.
|
Stock Options Grant.
|
4.
|
At-Will Employment & Termination.
|
•
|
In the event your employment is terminated by the Company without Cause on or after the first-year
anniversary of your Start Date then in addition to the Options that shall have vested as of the date of termination, the Options that would have vested on the last day of the calendar quarter in which such termination occurs shall vest.
|
• |
In the event your employment is terminated by the Company without Cause or you terminate your employment for
Good Reason on or after the first-year anniversary of your Start Date, then the Company shall pay you your base salary for the six months following such termination.
|
5.
|
Additional Information.
|
Sincerely,
|
||
Gaurav Shah, M.D.
|
||
Chief Executive Officer of Rocket Pharmaceuticals LTD.
|
||
Agreed and accepted:
|
||
Raj Prabhakar
|
Agreed and accepted:
|
|||
Date:
|
|||
[New Employee]
|
1. |
Purchase of Shares. Subject to the terms and conditions hereinafter set forth, the holder of
this Warrant is entitled, upon surrender of this Warrant at the principal office of the Company (or at such other place as the Company shall notify the holder hereof in writing), to purchase from the Company up to 153,155 shares, as
adjusted pursuant to Section 7 below (the “Shares”), of the Company’s common stock, par value
|
|
$0.01 per share (the “Common Stock”), at the Exercise Price (as defined below). Any future warrants granted pursuant to the Consulting Agreement may provide for more or less shares.
|
2. |
Definitions.
|
a. |
Change of Control. The term “Change of Control” shall mean (i) the acquisition of the
Company by another entity by means of any transaction or series of related transactions with the Company (including, without limitation, any stock purchase, reorganization, merger or consolidation but, excluding any merger effected
exclusively for the purpose of changing the domicile of the Company); or (ii) a sale of all or substantially all of the assets of the Company, unless the Company’s stockholders of record as constituted immediately prior to such
acquisition or sale will, immediately after such acquisition or sale (solely by virtue of securities issued as consideration for the Company’s acquisition or sale or otherwise) hold at least 50% of the voting power of the surviving or
acquiring entity.
|
b. |
Exercise Price. The exercise price for the Shares shall be $22.51 per Share, as adjusted for any
stock splits, dividends, combinations and the like as provided in Section 7 below (such price, as adjusted from time to time, is herein referred to as the “Exercise Price”).
|
3. |
Change of Control. In the event of a Change of Control, the holder of this Warrant shall have
the right thereafter to receive, upon exercise of this Warrant, the same amount and kind of securities, cash or property as it would have been entitled to receive upon the occurrence of such Change of Control if it had been, immediately
prior to such Change of Control, the holder of the number of Warrant Shares then issuable upon exercise in full of this Warrant without regard to any limitations on exercise contained herein, and the holder of this Warrant shall no longer
have the right to receive Warrant Shares upon exercise of this Warrant.
|
4. |
Exercise.
|
a. |
Method of Exercise. While this Warrant remains outstanding and exercisable, the holder may
exercise, in whole or in part, the purchase rights evidenced hereby. Such exercise shall be effected by:
|
i. |
the surrender of the Warrant, together with a notice of exercise to the Secretary of the Company at its principal offices; and
|
ii. |
the payment to the Company of an amount equal to the aggregate Exercise Price for the number of Shares being purchased.
|
iii. |
In lieu of exercising this Warrant as specified in this Section 4(a)(ii), the holder may convert this Warrant, in whole or in part, into a number of Shares determined by dividing (a) the aggregate fair market value of the Shares issuable
upon exercise of this Warrant (or lesser number of shares in the case of a partial exercise) minus the aggregate Exercise Price of such Shares by (b) the fair market value of one Share. The fair market value of the Shares shall be the
closing price of the Shares reported for the business day immediately before the holder delivers its notice of exercise to the Company
|
b. |
Exercise Period. This Warrant shall be immediately exercisable upon the Effective Date in the
amount of 153,155 Shares. This Warrant shall cease to be exercisable upon the expiration of this Warrant pursuant to Section 16 hereof.
|
5. |
Certificates for Shares. Upon the exercise of the purchase rights evidenced by this Warrant, one
or more certificates or book-entry notations for the number of Shares so purchased and, if this Warrant has not been fully exercised or converted and has not expired, a new Warrant representing the Shares not so acquired, shall be issued
as soon as practicable thereafter.
|
6. |
Issuance of Shares. The Company covenants that the Shares, when issued pursuant to the exercise
of this Warrant, will be duly and validly issued, fully paid and nonassessable.
|
7. |
Adjustment of Exercise Price and Number of Shares. The number of and kind of securities
purchasable upon exercise of this Warrant and the Exercise Price shall be subject to adjustment from time to time as follows:
|
a. |
Subdivisions, Combinations and Other Issuances. If the Company declares or pays a dividend or
distribution on the shares of Common Stock payable in common stock or other securities or property (other than cash), then upon exercise of this Warrant, for each Share acquired, the holder shall receive, without additional cost to the
holder, the total number and kind of securities and property which holder would have received had the holder owned the Shares of record as of the date the dividend or distribution occurred. If the Company subdivides the outstanding shares
of Common Stock by reclassification or otherwise into a greater number of shares, the number of Shares purchasable hereunder shall be proportionately increased and the Exercise Price shall be proportionately decreased. If the outstanding
shares of Common Stock are combined or consolidated, by reclassification or otherwise, into a lesser number of shares, the Exercise Price shall be proportionately increased and the number of Shares shall be proportionately decreased. Any
adjustment under this Section 7(a) shall become effective at the close of business on the date the subdivision or combination becomes effective, or as of the record date of such dividend, or in the event that no record date is fixed, upon
the making of such dividend.
|
b. |
Reclassification, Reorganization and Consolidation. Upon any event whereby all of the
outstanding shares of Common Stock are reclassified, exchanged, combined, substituted, or replaced for, into, with or by Company securities of a different class and/or series, then from and after the consummation of such event, this
Warrant will be exercisable for the number, class and series of Company securities that holder would have received had the Shares been outstanding on and as of the consummation of such event, and subject to further adjustment thereafter
from time to time in accordance with the provisions of this Warrant.
|
c. |
Notice of Adjustment and Certain Events. The Company shall provide the holder with not less than
5 days prior written notice of, including a description of the material facts surrounding, any of the following events: (a) declaration of any dividend or distribution upon its Common Stock, whether in cash, property, stock, or other
securities and whether or not a regular cash dividend; (b) offering for subscription pro rata to the holders of any class or series of its stock any additional shares of stock of any class or series or other rights; (c) effecting any
reclassification or recapitalization of Common Stock; (d) the merger or consolidation with or into any other corporation, or sale, lease, license, or conveyance of all or substantially all of its assets, or liquidation, dissolution or
winding up; or (e) when any other adjustment is required to be made in the number or kind of shares purchasable upon exercise of this Warrant, or in the Exercise Price. When any adjustment is required to be made in the number or kind of
shares purchaseable upon exercise of this Warrant, or in the Exercise Price, the Company shall promptly compute such adjustment or readjustment in accordance with the provisions hereof and prepare a certificate showing such adjustment or
readjustment, and shall mail such certificate to the holder of this Warrant in accordance with the notice provisions of Section 17. The certificate shall set forth such adjustment or readjustment and indicate the number of shares of
Common Stock and the Exercise Price in effect after such adjustment or readjustment. The provisions of this Section 7(c) shall similarly apply to successive reclassifications, exchanges, combinations substitutions, replacements or other
similar events.
|
8. |
No Fractional Shares or Scrip. No fractional shares or scrip representing fractional shares
shall be issued upon the exercise of this Warrant, but in lieu of such fractional shares the Company shall make a cash payment therefor on the basis of the Exercise Price then in effect.
|
9. |
Reservation of Shares. During the period between the Effective Date and the Expiration Date, the
Company shall at all times reserve and keep available out of its authorized but unissued shares of Common Stock or other securities constituting Shares, solely for the purpose of issuance upon the exercise of this Warrant, the maximum
number of Shares issuable upon the exercise of this Warrant, and the par value per Share shall at all times be less than or equal to the applicable Exercise Price.
|
10. |
Representations of the Company. The Company represents that all corporate actions on the part of
the Company, its officers, directors and stockholders necessary for the sale and issuance of this Warrant have been taken. The Company will pay all original issue and transfer taxes, if any, with respect to the issue and delivery of the
Shares pursuant hereto and all other fees and expenses necessarily incurred by the Company in connection herewith.
|
11. |
Representations and Warranties by the Holder. The holder of this Warrant represents and warrants
to the Company as follows:
|
a. |
This Warrant and the Shares issuable upon exercise thereof are being acquired for its own account, for investment and not with a view to, or for resale in connection with, any distribution or public offering thereof within the meaning of
the Securities Act of 1933, as amended (the "Act"). Upon exercise of this Warrant, the holder of this Warrant shall, if so requested by the Company, confirm in writing, in a form satisfactory to the Company, that the securities
issuable upon exercise of this Warrant are being acquired for investment and not with a view toward distribution or resale.
|
b. |
The holder of this Warrant understands that this Warrant and the Shares have not been registered under the Act by reason of their issuance in a transaction exempt from the registration and prospectus delivery requirements of the Act
pursuant to Section 4(a)(2) thereof, and that they must be held by the holder indefinitely, and that the holder must therefore bear the economic risk of such investment indefinitely, unless a subsequent disposition thereof is registered
under the Act or is exempted from such registration.
|
c. |
The holder of this Warrant has such knowledge and experience in financial and business matters that it is capable of evaluating the merits and risks of the purchase of this Warrant and the Shares purchasable pursuant to the terms of this
Warrant and of protecting its interests in connection therewith.
|
d. |
The holder of this Warrant is aware of the Company’s business affairs and financial condition and has received or has had full access to all the information it considers necessary or appropriate to make an informed investment decision
with respect to the acquisition of this Warrant and its underlying securities. The holder of this Warrant further has had an opportunity to ask questions and receive answers from the Company regarding the terms and conditions of the
offering of this Warrant and its underlying securities and to obtain additional information (to the extent the Company possessed such information or could acquire it without unreasonable effort or expense) necessary to verify any
information furnished to the holder of this Warrant or to which the holder has access.
|
e. |
The holder of this Warrant is able to bear the economic risk of the purchase of the Shares pursuant to the terms of this Warrant.
|
f. |
The holder of this Warrant is an “accredited investor” as such term is defined in Rule 501 of Regulation D promulgated under the Act.
|
12. |
Restrictive Legend. The Shares shall be stamped or imprinted with a legend in substantially the
following form:
|
13. |
Warrants Transferable.
|
a. |
This Warrant, and any Shares issued hereunder, may not be sold, assigned or otherwise transferred by the holder hereof until six months after the Effective Date, except for an assignment or transfer of this Warrant, in whole or in part,
by Neptune to an affiliate of Neptune, excluding any affiliate of the Company that is a natural person. Thereafter, this Warrant and the Shares may be offered, sold or otherwise transferred or disposed, in whole or in part, by the holder.
Any sale, transfer or assignment of this Warrant or the Shares issued hereunder shall be subject to compliance with the terms and conditions of Section 13(b) and applicable federal and state securities laws.
|
b. |
Any transfer of this Warrant shall require surrender of this Warrant properly endorsed or accompanied by written instructions of transfer. With respect to any offer, sale or other disposition of this Warrant or the Shares, the holder
hereof agrees to give written notice to the Company prior thereto, describing briefly the manner thereof, together with a written opinion of the holder’s counsel, or other evidence, if requested by the Company, to the effect that such
offer, sale or other disposition may be effected without registration or qualification (under the Act as then in effect or any federal or state securities law then in effect) of this Warrant or the Shares and indicating whether or not under
the Act certificates for this Warrant or the Shares to be sold or otherwise disposed of require any restrictive legend as to applicable restrictions on transferability in order to ensure compliance with such law; provided, however, that (i)
the Company shall not require the holder to provide an opinion of counsel if the transfer is to any affiliate of the holder, provided that any such transferee is an “accredited investor” as defined in Regulation D promulgated under the Act,
and additionally, (ii) the Company shall also not require an opinion of counsel if there is no material question as to the availability of Rule 144 promulgated under the Act. Upon receiving such written notice and reasonably satisfactory
opinion or other evidence, if so requested, the Company, as promptly as practicable, shall notify the holder that the holder may sell or otherwise dispose of this Warrant or the Shares, all in accordance with the terms of the notice
delivered to the Company. If a determination has been made pursuant to this Section 13 that the opinion of counsel for the holder or other evidence is not reasonably satisfactory to the Company, the Company shall so notify the holder
promptly with details thereof after such determination has been made. Each certificate representing this Warrant or Shares transferred in accordance with this Section 13 shall bear a legend as to the applicable restrictions on
transferability in order to ensure compliance with such laws, unless in the aforesaid opinion of counsel for the holder, such legend is not required in order to ensure compliance with such laws. The Company may issue stop transfer
instructions to its transfer agent in connection with such restrictions.
|
14. |
Reserved.
|
15. |
No Rights as Stockholder. The holder of this Warrant, as a holder of this Warrant, will not have
any voting rights or other rights of a stockholder of the Company until exercise of this Warrant.
|
16. |
Expiration of Warrant. This Warrant shall be exercisable in whole or in part, at any time and
from time to time, until ten years from the original issue date of this Warrant (the “Expiration Date”). If this Warrant has not been exercised prior to the Expiration Date, this Warrant shall be deemed to have been automatically
exercised on the Expiration Date by “cashless” conversion pursuant to Section 4(a)(iii).
|
17. |
Notices. Any notice, consent, claim, demand, waiver, or other communication under this Warrant
have legal effect only if in writing and addressed to a party to the address set forth below (or to such other address or such other person that a party may designate for itself from time to time in accordance with this Section). Notices
sent in accordance with this Section will be deemed effectively given: (a) when received, if delivered by hand, with signed confirmation of receipt; (b) when received, if sent by a nationally recognized overnight courier, signature
required; (c) when sent, if by email, (in each case, with confirmation of transmission), if sent during the addressee's normal business hours, and on the next business day, if sent after the addressee's normal business hours; and (d) on
the third day after the date mailed by certified or registered mail, return receipt requested, postage prepaid.
|
18. |
Governing Law. This Warrant and all actions arising out of or in connection with this Agreement
shall be governed by and construed in accordance with the laws of the State of New York, without regard to the conflicts of law provisions of the State of New York or of any other state.
|
19. |
Rights and Obligations Survive Exercise of Warrant. Unless otherwise provided herein, the rights
and obligations of the Company, of the holder of this Warrant and of the holder of the Shares issued upon exercise of this Warrant, shall survive the exercise of this Warrant.
|
ROCKET PHARMACEUTICALS, INC.
|
|||
By: |
|
||
Name: Gaurav Shah
|
|||
Title: President & CEO |
1.
|
The undersigned hereby elects to purchase Shares pursuant to the terms of the attached Warrant.
|
2. |
Method of Exercise (Please initial the applicable blank):
|
☐
|
The undersigned elects to exercise the attached Warrant by means of a cash payment, and tenders herewith payment in full for the purchase price of the shares being purchased, together with all applicable transfer taxes, if any.
|
☐
|
The undersigned elects to exercise the attached Warrant by means of the net exercise provisions of Section 4(a)(iii) of the Warrant.
|
3. |
Please issue a certificate or certificates representing said Shares in the name of the undersigned or in such other name as is specified below:
|
4. |
The undersigned hereby represents and warrants that all representations and warranties of the undersigned set forth in Section 11 of the attached Warrant (including Section 11(f) thereof) are true and correct as of the date hereof.
|
|
|
1. |
Purchase of Shares. Subject to the terms and conditions hereinafter set forth, the holder of
this Warrant is entitled, upon surrender of this Warrant at the principal office of the Company (or at such other place as the Company shall notify the holder hereof in writing), to purchase from the Company up to 153,155 shares, as
adjusted pursuant to Section 7 below (the “Shares”), of the Company’s common stock, par value
$0.01 per share (the “Common Stock”), at the Exercise Price (as defined below). Any future
warrants granted pursuant to the Consulting Agreement may provide for more or less shares.
|
2. |
Definitions.
|
a. |
Change of Control. The term “Change of Control” shall mean (i) the acquisition of the
Company by another entity by means of any transaction or series of related transactions with the Company (including, without limitation, any stock purchase, reorganization, merger or consolidation but, excluding any merger effected
exclusively for the purpose of changing the domicile of the Company); or (ii) a sale of all or substantially all of the assets of the Company, unless the Company’s stockholders of record as constituted immediately prior to such
acquisition or sale will, immediately after such acquisition or sale (solely by virtue of securities issued as consideration for the Company’s acquisition or sale or otherwise) hold at least 50% of the voting power of the surviving or
acquiring entity.
|
b. |
Exercise Price. The exercise price for the Shares shall be $22.51 per Share, as adjusted for any
stock splits, dividends, combinations and the like as provided in Section 7 below (such price, as adjusted from time to time, is herein referred to as the “Exercise Price”).
|
3. |
Change of Control. In the event of a Change of Control, the holder of this Warrant shall have
the right thereafter to receive, upon exercise of this Warrant, the same amount and kind of securities, cash or property as it would have been entitled to receive upon the occurrence of such Change of Control if it had been, immediately
prior to such Change of Control, the holder of the number of Warrant Shares then issuable upon exercise in full of this Warrant without regard to any limitations on exercise contained herein, and the holder of this Warrant shall no longer
have the right to receive Warrant Shares upon exercise of this Warrant.
|
4. |
Exercise.
|
a. |
Method of Exercise. While this Warrant remains outstanding and exercisable, the holder may
exercise, in whole or in part, the purchase rights evidenced hereby. Such exercise shall be effected by:
|
i. |
the surrender of the Warrant, together with a notice of exercise to the Secretary of the Company at its principal offices; and
|
ii. |
the payment to the Company of an amount equal to the aggregate Exercise Price for the number of Shares being purchased.
|
iii. |
In lieu of exercising this Warrant as specified in this Section 4(a)(ii), the holder may convert this Warrant, in whole or in part, into a number of Shares determined by dividing (a) the aggregate fair market value of the Shares issuable
upon exercise of this Warrant (or lesser number of shares in the case of a partial exercise) minus the aggregate Exercise Price of such Shares by (b) the fair market value of one Share. The fair market value of the Shares shall be the
closing price of the Shares reported for the business day immediately before the holder delivers its notice of exercise to the Company
|
b. |
Exercise Period. This Warrant shall be immediately exercisable upon the Effective Date in the
amount of 153,155 Shares. This Warrant shall cease to be exercisable upon the expiration of this Warrant pursuant to Section 16 hereof.
|
5. |
Certificates for Shares. Upon the exercise of the purchase rights evidenced by this Warrant, one
or more certificates or book-entry notations for the number of Shares so purchased and, if this Warrant has not been fully exercised or converted and has not expired, a new Warrant representing the Shares not so acquired, shall be issued
as soon as practicable thereafter.
|
6. |
Issuance of Shares. The Company covenants that the Shares, when issued pursuant to the exercise
of this Warrant, will be duly and validly issued, fully paid and nonassessable.
|
7. |
Adjustment of Exercise Price and Number of Shares. The number of and kind of securities
purchasable upon exercise of this Warrant and the Exercise Price shall be subject to adjustment from time to time as follows:
|
a. |
Subdivisions, Combinations and Other Issuances. If the Company declares or pays a dividend or
distribution on the shares of Common Stock payable in common stock or other securities or property (other than cash), then upon exercise of this Warrant, for each Share acquired, the holder shall receive, without additional cost to the
holder, the total number and kind of securities and property which holder would have received had the holder owned the Shares of record as of the date the dividend or distribution occurred. If the Company subdivides the outstanding shares
of Common Stock by reclassification or otherwise into a greater number of shares, the number of Shares purchasable hereunder shall be proportionately increased and the Exercise Price shall be proportionately decreased. If the outstanding
shares of Common Stock are combined or consolidated, by reclassification or otherwise, into a lesser number of shares, the Exercise Price shall be proportionately increased and the number of Shares shall be proportionately decreased. Any
adjustment under this Section 7(a) shall become effective at the close of business on the date the subdivision or combination becomes effective, or as of the record date of such dividend, or in the event that no record date is fixed, upon
the making of such dividend.
|
b. |
Reclassification, Reorganization and Consolidation. Upon any event whereby all of the
outstanding shares of Common Stock are reclassified, exchanged, combined, substituted, or replaced for, into, with or by Company securities of a different class and/or series, then from and after the consummation of such event, this
Warrant will be exercisable for the number, class and series of Company securities that holder would have received had the Shares been outstanding on and as of the consummation of such event, and subject to further adjustment thereafter
from time to time in accordance with the provisions of this Warrant.
|
c. |
Notice of Adjustment and Certain Events. The Company shall provide the holder with not less than
5 days prior written notice of, including a description of the material facts surrounding, any of the following events: (a) declaration of any dividend or distribution upon its Common Stock, whether in cash, property, stock, or other
securities and whether or not a regular cash dividend; (b) offering for subscription pro rata to the holders of any class or series of its stock any additional shares of stock of any class or series or other rights; (c) effecting any
reclassification or recapitalization of Common Stock; (d) the merger or consolidation with or into any other corporation, or sale, lease, license, or conveyance of all or substantially all of its assets, or liquidation, dissolution or
winding up; or (e) when any other adjustment is required to be made in the number or kind of shares purchasable upon exercise of this Warrant, or in the Exercise Price. When any adjustment is required to be made in the number or kind of
shares purchaseable upon exercise of this Warrant, or in the Exercise Price, the Company shall promptly compute such adjustment or readjustment in accordance with the provisions hereof and prepare a certificate showing such adjustment or
readjustment, and shall mail such certificate to the holder of this Warrant in accordance with the notice provisions of Section 17. The certificate shall set forth such adjustment or readjustment and indicate the number of shares of
Common Stock and the Exercise Price in effect after such adjustment or readjustment. The provisions of this Section 7(c) shall similarly apply to successive reclassifications, exchanges, combinations substitutions, replacements or other
similar events.
|
8. |
No Fractional Shares or Scrip. No fractional shares or scrip representing fractional shares
shall be issued upon the exercise of this Warrant, but in lieu of such fractional shares the Company shall make a cash payment therefor on the basis of the Exercise Price then in effect.
|
9. |
Reservation of Shares. During the period between the Effective Date and the Expiration Date, the
Company shall at all times reserve and keep available out of its authorized but unissued shares of Common Stock or other securities constituting Shares, solely for the purpose of issuance upon the exercise of this Warrant, the maximum
number of Shares issuable upon the exercise of this Warrant, and the par value per Share shall at all times be less than or equal to the applicable Exercise Price.
|
10. |
Representations of the Company. The Company represents that all corporate actions on the part of
the Company, its officers, directors and stockholders necessary for the sale and issuance of this Warrant have been taken. The Company will pay all original issue and transfer taxes, if any, with respect to the issue and delivery of the
Shares pursuant hereto and all other fees and expenses necessarily incurred by the Company in connection herewith.
|
11. |
Representations and Warranties by the Holder. The holder of this Warrant represents and warrants
to the Company as follows:
|
a. |
This Warrant and the Shares issuable upon exercise thereof are being acquired for its own account, for investment and not with a view to, or for resale in connection with, any distribution or public offering thereof within the meaning of
the Securities Act of 1933, as amended (the "Act"). Upon exercise of this Warrant, the holder of this Warrant shall, if so requested by the Company, confirm in writing, in a form satisfactory to the Company, that the securities
issuable upon exercise of this Warrant are being acquired for investment and not with a view toward distribution or resale.
|
b. |
The holder of this Warrant understands that this Warrant and the Shares have not been registered under the Act by reason of their issuance in a transaction exempt from the registration and prospectus delivery requirements of the Act
pursuant to Section 4(a)(2) thereof, and that they must be held by the holder indefinitely, and that the holder must therefore bear the economic risk of such investment indefinitely, unless a subsequent disposition thereof is registered
under the Act or is exempted from such registration.
|
c. |
The holder of this Warrant has such knowledge and experience in financial and business matters that it is capable of evaluating the merits and risks of the purchase of this Warrant and the Shares purchasable pursuant to the terms of this
Warrant and of protecting its interests in connection therewith.
|
d. |
The holder of this Warrant is aware of the Company’s business affairs and financial condition and has received or has had full access to all the information it considers necessary or appropriate to make an informed investment decision
with respect to the acquisition of this Warrant and its underlying securities. The holder of this Warrant further has had an opportunity to ask questions and receive answers from the Company regarding the terms and conditions of the
offering of this Warrant and its underlying securities and to obtain additional information (to the extent the Company possessed such information or could acquire it without unreasonable effort or expense) necessary to verify any
information furnished to the holder of this Warrant or to which the holder has access.
|
e. |
The holder of this Warrant is able to bear the economic risk of the purchase of the Shares pursuant to the terms of this Warrant.
|
f. |
The holder of this Warrant is an “accredited investor” as such term is defined in Rule 501 of Regulation D promulgated under the Act.
|
12. |
Restrictive Legend. The Shares shall be stamped or imprinted with a legend in substantially the
following form:
|
13. |
Warrants Transferable.
|
a. |
This Warrant, and any Shares issued hereunder, may not be sold, assigned or otherwise transferred by the holder hereof until six months after the Effective Date, except for an assignment or transfer of this Warrant, in whole or in part,
by Neptune to an affiliate of Neptune, excluding any affiliate of the Company that is a natural person. Thereafter, this Warrant and the Shares may be offered, sold or otherwise transferred or disposed, in whole or in part, by the holder.
Any sale, transfer or assignment of this Warrant or the Shares issued hereunder shall be subject to compliance with the terms and conditions of Section 13(b) and applicable federal and state securities laws.
|
b. |
Any transfer of this Warrant shall require surrender of this Warrant properly endorsed or accompanied by written instructions of transfer. With respect to any offer, sale or other disposition of this Warrant or the Shares, the holder
hereof agrees to give written notice to the Company prior thereto, describing briefly the manner thereof, together with a written opinion of the holder’s counsel, or other evidence, if requested by the Company, to the effect that such
offer, sale or other disposition may be effected without registration or qualification (under the Act as then in effect or any federal or state securities law then in effect) of this Warrant or the Shares and indicating whether or not under
the Act certificates for this Warrant or the Shares to be sold or otherwise disposed of require any restrictive legend as to applicable restrictions on transferability in order to ensure compliance with such law; provided, however, that (i)
the Company shall not require the holder to provide an opinion of counsel if the transfer is to any affiliate of the holder, provided that any such transferee is an “accredited investor” as defined in Regulation D promulgated under the Act,
and additionally, (ii) the Company shall also not require an opinion of counsel if there is no material question as to the availability of Rule 144 promulgated under the Act. Upon receiving such written notice and reasonably satisfactory
opinion or other evidence, if so requested, the Company, as promptly as practicable, shall notify the holder that the holder may sell or otherwise dispose of this Warrant or the Shares, all in accordance with the terms of the notice
delivered to the Company. If a determination has been made pursuant to this Section 13 that the opinion of counsel for the holder or other evidence is not reasonably satisfactory to the Company, the Company shall so notify the holder
promptly with details thereof after such determination has been made. Each certificate representing this Warrant or Shares transferred in accordance with this Section 13 shall bear a legend as to the applicable restrictions on
transferability in order to ensure compliance with such laws, unless in the aforesaid opinion of counsel for the holder, such legend is not required in order to ensure compliance with such laws. The Company may issue stop transfer
instructions to its transfer agent in connection with such restrictions.
|
14. |
Reserved.
|
15. |
No Rights as Stockholder. The holder of this Warrant, as a holder of this Warrant, will not have
any voting rights or other rights of a stockholder of the Company until exercise of this Warrant.
|
16. |
Expiration of Warrant. This Warrant shall be exercisable in whole or in part, at any time and
from time to time, until ten years from the original issue date of this Warrant (the “Expiration Date”). If this Warrant has not been exercised prior to the Expiration Date, this Warrant shall be deemed to have been automatically
exercised on the Expiration Date by “cashless” conversion pursuant to Section 4(a)(iii).
|
17. |
Notices. Any notice, consent, claim, demand, waiver, or other communication under this Warrant
have legal effect only if in writing and addressed to a party to the address set forth below (or to such other address or such other person that a party may designate for itself from time to time in accordance with this Section). Notices
sent in accordance with this Section will be deemed effectively given: (a) when received, if delivered by hand, with signed confirmation of receipt; (b) when received, if sent by a nationally recognized overnight courier, signature
required; (c) when sent, if by email, (in each case, with confirmation of transmission), if sent during the addressee's normal business hours, and on the next business day, if sent after the addressee's normal business hours; and (d) on
the third day after the date mailed by certified or registered mail, return receipt requested, postage prepaid.
|
18. |
Governing Law. This Warrant and all actions arising out of or in connection with this Agreement
shall be governed by and construed in accordance with the laws of the State of New York, without regard to the conflicts of law provisions of the State of New York or of any other state.
|
19. |
Rights and Obligations Survive Exercise of Warrant. Unless otherwise provided herein, the rights
and obligations of the Company, of the holder of this Warrant and of the holder of the Shares issued upon exercise of this Warrant, shall survive the exercise of this Warrant.
|
ROCKET PHARMACEUTICALS, INC.
|
|||
By:
|
|||
Name:
|
Gaurav Shah
|
||
Title:
|
President & CEO
|
1.
|
The undersigned hereby elects to purchase Shares pursuant to the terms of the attached Warrant.
|
2. |
Method of Exercise (Please initial the applicable blank):
|
☐
|
The undersigned elects to exercise the attached Warrant by means of a cash payment, and tenders herewith payment in full for the purchase price of the shares being purchased, together with all applicable
transfer taxes, if any.
|
☐
|
The undersigned elects to exercise the attached Warrant by means of the net exercise provisions of Section 4(a)(iii) of the Warrant.
|
3. |
Please issue a certificate or certificates representing said Shares in the name of the undersigned or in such other name as is specified below:
|
4. |
The undersigned hereby represents and warrants that all representations and warranties of the undersigned set forth in Section 11 of the attached Warrant (including Section 11(f) thereof) are true and correct as of the date hereof.
|
|
|
1.
|
I have reviewed this annual report on Form 10-K of Rocket Pharmaceuticals, Inc.;
|
2.
|
Based on my knowledge, this report does not contain any untrue statement of a material fact or omit to state a material fact necessary to make the statements made, in light of the
circumstances under which such statements were made, not misleading with respect to the period covered by this report;
|
3.
|
Based on my knowledge, the financial statements, and other financial information included in this report, fairly present in all material respects the financial condition, results of
operations and cash flows of the registrant as of, and for, the periods presented in this report;
|
4.
|
The registrant’s other certifying officer and I are responsible for establishing and maintaining disclosure controls and procedures (as defined in Exchange Act Rules 13a-15(e) and
15d-15(e)) and internal control over financial reporting (as defined in Exchange Act Rules 13a-15(f) and 15d-15(f)) for the registrant and have:
|
a.
|
Designed such disclosure controls and procedures, or caused such disclosure controls and procedures to be designed under our supervision, to ensure that material information
relating to the registrant, including its consolidated subsidiaries, is made known to us by others within those entities, particularly during the period in which this report is being prepared;
|
b.
|
Designed such internal control over financial reporting, or caused such internal control over financial reporting to be designed under our supervision, to provide reasonable
assurance regarding the reliability of financial reporting and the preparation of financial statements for external purposes in accordance with generally accepted accounting principles;
|
c.
|
Evaluated the effectiveness of the registrant’s disclosure controls and procedures and presented in this report our conclusions about the effectiveness of the disclosure controls
and procedures, as of the end of the period covered by this report based on such evaluation; and
|
d.
|
Disclosed in this report any change in the registrant’s internal control over financial reporting that occurred during the registrant’s most recent fiscal quarter (the registrant’s
fourth fiscal quarter in the case of an annual report) that has materially affected, or is reasonably likely to materially affect, the registrant’s internal control over financial reporting; and
|
5.
|
The registrant’s other certifying officer and I have disclosed, based on our most recent evaluation of internal control over financial reporting, to the registrant’s auditors and
the audit committee of the registrant’s Board of Directors (or persons performing the equivalent functions):
|
a.
|
All significant deficiencies and material weaknesses in the design or operation of internal control over financial reporting which are reasonably likely to adversely affect the
registrant’s ability to record, process, summarize and report financial information; and
|
b.
|
Any fraud, whether or not material, that involves management or other employees who have a significant role in the registrant’s internal control over financial reporting.
|
Date: February 28, 2022
|
/s/ Gaurav Shah, MD
|
|
Gaurav Shah, MD
|
|
President, Chief Executive Officer and Director
|
|
(Principal Executive Officer)
|
1.
|
I have reviewed this annual report on Form 10-K of Rocket Pharmaceuticals, Inc.;
|
2.
|
Based on my knowledge, this report does not contain any untrue statement of a material fact or omit to state a material fact necessary to make the statements made, in light of the
circumstances under which such statements were made, not misleading with respect to the period covered by this report;
|
3.
|
Based on my knowledge, the financial statements, and other financial information included in this report, fairly present in all material respects the financial condition, results of
operations and cash flows of the registrant as of, and for, the periods presented in this report;
|
4.
|
The registrant’s other certifying officer and I are responsible for establishing and maintaining disclosure controls and procedures (as defined in Exchange Act Rules 13a-15(e) and
15d-15(e)) and internal control over financial reporting (as defined in Exchange Act Rules 13a-15(f) and 15d-15(f)) for the registrant and have:
|
a.
|
Designed such disclosure controls and procedures, or caused such disclosure controls and procedures to be designed under our supervision, to ensure that material information
relating to the registrant, including its consolidated subsidiaries, is made known to us by others within those entities, particularly during the period in which this report is being prepared;
|
b.
|
Designed such internal control over financial reporting, or caused such internal control over financial reporting to be designed under our supervision, to provide reasonable
assurance regarding the reliability of financial reporting and the preparation of financial statements for external purposes in accordance with generally accepted accounting principles;
|
c.
|
Evaluated the effectiveness of the registrant’s disclosure controls and procedures and presented in this report our conclusions about the effectiveness of the disclosure controls
and procedures, as of the end of the period covered by this report based on such evaluation; and
|
d.
|
Disclosed in this report any change in the registrant’s internal control over financial reporting that occurred during the registrant’s most recent fiscal quarter (the registrant’s
fourth fiscal quarter in the case of an annual report) that has materially affected, or is reasonably likely to materially affect, the registrant’s internal control over financial reporting; and
|
5.
|
The registrant’s other certifying officer and I have disclosed, based on our most recent evaluation of internal control over financial reporting, to the registrant’s auditors and
the audit committee of the registrant’s Board of Directors (or persons performing the equivalent functions):
|
a.
|
All significant deficiencies and material weaknesses in the design or operation of internal control over financial reporting which are reasonably likely to adversely affect the
registrant’s ability to record, process, summarize and report financial information; and
|
b.
|
Any fraud, whether or not material, that involves management or other employees who have a significant role in the registrant’s internal control over financial reporting.
|
Date: February 28, 2022
|
/s/ Carlos Garcia-Parada |
|
Carlos Garcia-Parada
|
|
Chief Financial Officer
|
|
(Principal Financial Officer)
|
Date: February 28, 2022
|
/s/ Gaurav Shah, MD
|
|
Gaurav Shah, MD
|
|
President, Chief Executive Officer and Director
|
|
(Principal Executive Officer)
|
Date: February 28, 2022
|
/s/ Carlos Garcia-Parada
|
|
Carlos Garcia-Parada
|
|
Chief Financial Officer
|
|
(Principal Financial Officer)
|