- By FYH News Team
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The following discussion and analysis of our financial condition and results of operations should be read in conjunction with our consolidated financial statements and related notes included elsewhere in this Annual Report on Form 10-K, or Annual Report. Some of the information contained in this discussion and analysis or set forth elsewhere in this Annual Report, including information with respect to our plans and strategy for our business, includes forward-looking statements within the meaning of Section 27A of the Securities Act, and Section 21E of the Securities Exchange Act of 1934, as amended, or the Exchange Act, that involve risks and uncertainties. As a result of many factors, including those factors set forth in the risks identified in Part I-Item 1A "Risk Factors" section of this Annual Report and our other filings with theSecurities and Exchange Commission , orSEC , our actual results could differ materially from the results, performance or achievements expressed in or implied by these forward-looking statements.
Overview
We are a global biopharmaceutical company focused on discovering, acquiring, developing and commercializing therapeutic medicines for patients suffering from debilitating diseases with significant unmet medical need. Our portfolio of assets, ARCALYST® (rilonacept), mavrilimumab, vixarelimab and KPL-404, are based on strong biologic rationale or validated mechanisms, target underserved conditions, and offer the potential for differentiation. These assets are designed to modulate immunological pathways across a spectrum of diseases. ARCALYST is an interleukin-1? and interleukin-1? cytokine trap. Cytokines are small proteins that play a role in cell signaling. We licensed ARCALYST from Regeneron, who discovered and initially developed the drug, in 2017. Our exclusive license to ARCALYST from Regeneron includes worldwide rights, excluding theMiddle East andNorth Africa , for all applications other than those in oncology and local administration to the eye or ear. We received FDA approval of ARCALYST for the treatment of recurrent pericarditis and reduction in risk of recurrence in adults and children 12 years and older inMarch 2021 . Recurrent pericarditis is a painful inflammatory cardiovascular disease with an estimatedU.S. prevalent population of approximately 40,000 patients seeking and receiving medical treatment. ARCALYST is commercially available through a distribution network of specialty pharmacies, which provide access acrossthe United States . ARCALYST is also approved inthe United States for the treatment of CAPS in adults and children 12 years and older, and the maintenance of remission in DIRA in adults and children weighing 10 kg or more. We are responsible for sales and distribution of ARCALYST in all approved indications inthe United States , and evenly split profits on sales with Regeneron. In the fourth quarter of 2021, our ARCALYST franchise achieved profitability after three quarters of commercial availability for recurrent pericarditis. InFebruary 2022 , we granted Huadong exclusive rights to develop and commercialize ARCALYST in theAsia Pacific region, excludingJapan . Mavrilimumab is an investigational monoclonal antibody inhibitor targeting GM-CSFR?. In 2017, we licensed exclusive worldwide rights in all indications to mavrilimumab from MedImmune. We plan to focus mavrilimumab development on cardiovascular diseases where the GM-CSF mechanism has been implicated and that have synergies with our existing commercial infrastructure. In parallel, we may also explore the use of mavrilimumab through research collaborations in other development areas. We have also been evaluating mavrilimumab in COVID-19-related ARDS. InDecember 2021 , we announced that the Phase 3 portion of the global double-blind, placebo-controlled Phase 2/3 clinical trial did not meet its primary efficacy endpoint. We subsequently decided to not progress mavrilimumab in the COVID-19-related ARDS indication, although we are continuing the Phase 3 portion of the clinical trial through its completion date. We previously evaluated mavrilimumab in GCA, a chronic inflammatory disease of the medium-to-large arteries with an estimatedU.S. prevalence of approximately 75,000 to 150,000 patients. InOctober 2020 , we announced that our Phase 2 proof-of-concept clinical trial for the study of mavrilimumab in GCA achieved both its primary and secondary efficacy endpoints with statistical significance. InSeptember 2020 , the FDA granted Orphan Drug designation for mavrilimumab for the treatment of GCA. InFebruary 2022 , we announced that we do not plan to initiate a Phase 3 trial of mavrilimumab in GCA. InFebruary 2022 , we granted Huadong exclusive rights to develop and commercialize mavrilimumab in theAsia Pacific region, excludingJapan . Vixarelimab is an investigational monoclonal antibody inhibitor of signaling through OSMR?. We acquired worldwide rights to vixarelimab in all indications from Biogen in 2016. We are evaluating vixarelimab for the potential 122
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treatment of prurigo nodularis, a chronic inflammatory skin condition with an estimatedU.S. prevalence of approximately 300,000 patients. InApril 2020 , we announced that our Phase 2a trial of vixarelimab in prurigo nodularis achieved its primary and secondary efficacy endpoints. InNovember 2020 , the FDA granted Breakthrough Therapy designation for vixarelimab for the treatment of pruritus associated with prurigo nodularis. We are conducting a global, randomized, double-blind, placebo-controlled Phase 2b dose-ranging clinical trial of vixarelimab in prurigo nodularis. This trial is designed to investigate efficacy, safety and pharmacokinetics. We expect to report top-line data from this trial in the second half of 2022. KPL-404 is an investigational monoclonal antibody inhibitor of CD40-CD154 interaction. In 2019, we acquired all of the outstanding securities of Primatope, the company that owned or controlled the intellectual property related to KPL-404. In connection with our acquisition of Primatope, we acquired an exclusive world-wide license to KPL-404 from BIMDC. The CD40-CD154 interaction is a key T-cell co-stimulatory signal critical for B-cell maturation, immunoglobulin class switching and Type 1 immune response. We believe disrupting the CD40-CD154 interaction is an attractive approach to address multiple autoimmune disease pathologies such as RA, Sjogren's syndrome, Graves' disease, systemic lupus erythematosus and solid organ transplant graft rejection. InMay 2021 , we announced positive final data from our Phase 1 clinical trial of KPL-404 in healthy volunteers, which evaluated safety and pharmacokinetics, as well as RO and TDAR. InDecember 2021 , we initiated a Phase 2 proof-of-concept clinical trial of KPL-404 in rheumatoid arthritis, which is designed to enable its potential development in a spectrum of autoimmune diseases believed to be mediated by the CD40-CD154 co-stimulatory interaction. The trial will evaluate pharmacokinetics, safety and efficacy of KPL-404 with subcutaneous administration. InJanuary 2022 , we provided KPL-404 to theUniversity of Maryland School of Medicine , as part of an experimental immunosuppressive regimen in connection with a transplant of a genetically-modified pig heart into an adult human with end-stage heart disease who was not eligible for a standard allogeneic heart transplant. Our future success is dependent on our ability to continue to commercialize ARCALYST and to develop, obtain regulatory approval for and successfully commercialize one or more of our current or future product candidates. Upon approval from the FDA of the commercial marketing of ARCALYST inthe United States for the treatment of recurrent pericarditis and reduction in risk of recurrence in adults and children 12 years and older inMarch 2021 , we assumed the sales and distribution of ARCALYST for the previously approved indications inthe United States and will evenly split profits on ARCALYST sales with Regeneron. However, as a company we have limited experience obtaining marketing approval for product candidates, commercializing a therapeutic, supporting sales, marketing, and distribution activities and maintaining applicable infrastructure for these activities either directly and/or through agreements with third parties; as a result we may not be able to continue to commercialize ARCALYST or successfully commercialize any future approved product candidates, if any, thus potentially impairing the commercial potential of ARCALYST and our other product candidates. We have incurred significant operating losses since inception. Our ability to generate product revenue sufficient to achieve corporate profitability will depend heavily on the continued commercialization of ARCALYST and the development and eventual commercialization of one or more of our current or future product candidates, if approved. In the fourth quarter of 2021, our ARCALYST franchise achieved profitability, though such profits remain small compared to our total net losses. Our net losses were$157.9 million and$161.4 million for the years endedDecember 31, 2021 and 2020, respectively. As ofDecember 31, 2021 , we had an accumulated deficit of$675.4 million . We expect to continue to incur significant operating losses as we advance our product candidates through preclinical and clinical development and, ultimately, seek regulatory approval. In addition, we expect to continue to incur significant commercialization expenses related to product manufacturing, marketing, sales and distribution of ARCALYST. We may also incur expenses in connection with the in-licensing or acquisition of additional product candidates. As a result, until such time as we can generate significant and sustained revenue from product sales of ARCALYST and one or more of our current or future approved product candidates, if ever, we expect to finance our operations through a combination of sales of ARCALYST, raising additional capital such as through debt or equity offerings or through other sources, which may include licensing, collaborations or other strategic transactions or arrangements. We may be unable to raise additional funds or enter into such other transactions or arrangements when needed on favorable terms, or at all. If we fail to raise capital or enter into such transactions or arrangements as and when needed, we may have to significantly delay, scale back or discontinue the development
of
one or more of our
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current or future product candidates, delay our pursuit of potential in-licenses
or acquisitions or scale back on commercialization activities for ARCALYST.
Because of the numerous risks and uncertainties associated with product development, including any impact from the COVID-19 pandemic, we are unable to predict the timing or amount of increased expenses or when or if we will be able to achieve or maintain corporate profitability. Even if we are able to continue to commercialize ARCALYST and generate product sales from one or more of our current or future product candidates, if approved, we may not become profitable. If we fail to become profitable or are unable to sustain profitability on a continuing basis, then we may be unable to continue our operations at planned levels and be forced to reduce or terminate our operations. As ofDecember 31, 2021 , we had cash, cash equivalents and short-term investments of$182.2 million . We believe that our existing cash, cash equivalents and short-term investments will enable us to fund our operating expenses and capital expenditure requirements for at least the next 12 months from the date of issuance of the audited consolidated financial statements included in this Annual Report. We have based this estimate on assumptions that may prove to be wrong, and we could exhaust our available capital resources sooner than we expect. See "- Liquidity and Capital Resources." Our future viability is dependent on our ability to fund our operations through sales of ARCALYST and/or raise additional capital, such as through debt or equity offerings, as needed.
Components of Our Results of Operations
Product revenue, net
Following the FDA approval of ARCALYST inMarch 2021 , we began generating product revenue from sales of ARCALYST inApril 2021 . ARCALYST is sold through a third party logistics provider that distributes primarily through a network of authorized specialty pharmacies and specialty distributors (collectively, "customers"), which deliver the medication to patients by mail. Net revenue from product sales is recognized at the transaction price when the specialty pharmacy or specialty distributors obtains control of our product, which occurs at a point in time, typically upon shipment of the product from the third party logistics provider. Our net revenues represent total revenues adjusted for discounts and allowances, including estimated cash discounts, chargebacks, rebates, returns, copay assistance, and specialty pharmacy and distributor fees. These adjustments represent variable consideration under ASC 606 and are estimated using the expected value method and are recorded when revenue is recognized on the sale of the product. These adjustments are established by management as its best estimate based on available information and will be adjusted to reflect known changes in the factors that impact such allowances. Adjustments for variable consideration are determined based on the contractual terms with customers, historical trends, communications with customers and the levels of inventory remaining in the distribution channel, as well as expectations about the market for the product and anticipated introduction of competitive products.
Operating Expenses
Cost of Goods Sold
Cost of goods sold includes production and distribution costs of ARCALYST, and
amortization of the regulatory milestone, and other miscellaneous product costs
associated with ARCALYST. Cost of goods sold also includes the allocations for
the labor and overhead costs associated with the production of ARCALYST
associated with quality control, quality assurance, and supply chain activities.
Collaboration expenses
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Collaboration expenses consists of Regeneron's share of the profit related to ARCALYST sales under the Regeneron Agreement. We evenly split profits on sales of ARCALYST with Regeneron, where profits are determined after deducting from net sales of ARCALYST certain costs related to the manufacturing and commercialization of ARCALYST. Such costs include but are not limited to (i) our cost of goods sold for product used, sold or otherwise distributed for patient use by us; (ii) customary commercialization expenses, including the cost of our field force, and (iii) our cost to market, advertise and otherwise promote ARCALYST, with such costs identified in subsection (iii) subject to specified limits. In addition, should there be a transfer of technology related to the manufacture of ARCALYST, then, to the extent permitted in accordance with the Regeneron Agreement, the fully-burdened costs of each of us and Regeneron incurred in performing (or having performed) such technology transfer shall also be deducted from net sales of ARCALYST to determine profit. We also evenly split with Regeneron any proceeds received by us from any licensees, sublicensees and distributors in consideration for the sale, license or other disposition of rights with respect to ARCALYST, including upfront payments, milestone payments and royalties.
Research and Development Expenses
Research and development expenses consist primarily of costs incurred in
connection with the research and development of our product candidates. We
expense research and development costs as incurred. These expenses may include:
?expenses incurred to conduct the necessary preclinical studies and clinical
trials required to obtain regulatory approval;
?expenses incurred under agreements with CROs that are primarily engaged in the oversight and conduct of our clinical trials and CMOs that are primarily engaged to provide preclinical and clinical drug substance and product for our research and development programs for our product candidates;
?other costs related to acquiring and manufacturing preclinical and clinical
trial materials, including manufacturing validation batches, as well as
investigative sites and consultants that conduct our clinical trials,
preclinical studies and other scientific development services;
?payments made in cash or equity securities under third-party licensing,
acquisition and other similar agreements;
?employee-related expenses, including salaries and benefits, travel and
share-based compensation expense for employees engaged in research and
development functions;
?costs related to compliance with regulatory requirements; and
?allocated facilities-related costs, which include rent and utilities,
depreciation and other expenses.
We recognize external development costs based on an evaluation of the progress
to completion of specific tasks using information provided to us by our service
providers. This process involves reviewing open contracts and purchase orders,
communicating with our personnel to identify services that have been performed
on our behalf and estimating the level of service performed and the associated
cost incurred for the service when we have not yet been invoiced or otherwise
notified of actual costs. Nonrefundable advance payments for goods or services
to be received in the future for use in research and development activities are
recorded as prepaid expenses. Such amounts are recognized as an expense as the
goods are delivered or the related services are performed, or until it is no
longer expected that the goods will be delivered or the services rendered.
Our direct research and development expenses are tracked on a program-by-program
basis for our product candidates and consist primarily of external costs, such
as fees paid to outside consultants, CROs, CMOs and research laboratories in
connection with our preclinical development, process development, manufacturing
and clinical development activities. Our direct research and development
expenses by program also include fees incurred under
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license, acquisition and other similar agreements. We do not allocate employee costs or facility expenses, including depreciation or other indirect costs, to specific programs because these costs are deployed across multiple programs and, as such, are not separately classified. We use internal resources primarily to conduct our research and discovery activities as well as for managing our preclinical and clinical development, process development and manufacturing activities. Research and development activities are central to our business. Product candidates in later stages of clinical development generally have higher costs than those in earlier stages of clinical development, primarily due to the increased size and duration of later-stage clinical trials. As a result, we expect that our research and development expenses will be substantial over the next several years as we conduct our ongoing and/or planned clinical trials for our product candidates as well as conduct other preclinical and clinical development, and make regulatory filings for our product candidates. We also expect to incur additional expenses related to milestone and royalty payments payable to third parties with whom we have entered into license, acquisition and other similar agreements to acquire the rights to our product candidates. At this time, we cannot reasonably estimate or know the nature, timing and costs of the efforts that will be necessary to complete the clinical development of our current or future product candidates or when, if ever, we will realize significant revenue from product sales or be profitable. This uncertainty is due to the numerous risks and uncertainties, including those described in Part I, Item 1A. "Risk Factors" in this Annual Report.
Selling, General and Administrative Expenses
Selling, general and administrative expenses consist primarily of salaries and benefits, travel and share based compensation expense for personnel in selling, marketing, medical, executive, business development, finance, human resources, legal and support personnel functions. Selling, general and administrative expenses also include insurance and professional fees for legal, patent, consulting, accounting and audit services. Upon approval from the FDA of the commercial marketing of ARCALYST inthe United States for the treatment of recurrent pericarditis and reduction in risk of recurrence in adults and children 12 years and older inMarch 2021 , we assumed the sales and distribution of ARCALYST for the previously approved indications inthe United States . We expect that our selling, general and administrative expenses will continue to increase in the future as we continue to perform commercialization and sales activities. We also anticipate that we will continue to incur significant costs, including accounting, audit, legal, compliance and director and officer insurance costs as well as investor and public relations expenses, and that such costs will increase over time as the company continues to expand. Interest Income
Interest income consists of income recognized from investments in money market
funds and
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Income Taxes
As an exempted company incorporated under the laws of Bermuda , we are
principally subject to taxation in Bermuda . Under the current laws of Bermuda ,
tax on a company's income is assessed at a zero percent tax rate. As a result,
we have not recorded any income tax benefits from our losses incurred in Bermuda
during each reporting period, and no net operating loss carryforwards are
currently available to us for those losses, while our assets remain in Bermuda .
Our wholly owned U.S. subsidiaries, Kiniksa US, and Primatope are subject to
federal and state income taxes in the United States . Our wholly owned subsidiary
Kiniksa UK , and its wholly owned subsidiaries, Kiniksa Pharmaceuticals (Germany)
GmbH , Kiniksa Pharmaceuticals (France ) SARL, and Kiniksa Pharmaceuticals GmbH
are subject to taxation in their respective countries. Our provision for income
taxes relates mainly to U.S. taxable income, generated by our wholly owned
subsidiary Kiniksa US. The Company transferred all of its rights, title and
interest in, among other things, certain contracts (including the Regeneron
Agreement), intellectual property rights, product filings and approvals and
other information, plans and materials owned or controlled by the Company
insofar as they related exclusively or primarily to ARCALYST to Kiniksa UK in
January 2021 pursuant to an asset transfer agreement between the Company and
Kiniksa UK for the consideration described therein.
As of December 31, 2021 and 2020, we had federal research and development tax
credit carryforwards of approximately $1.5 million and $1.4 million
respectively, available to reduce future tax liabilities, which begin to expire
in 2041. As of December 31, 2021 and 2020, we had state research and development
tax credit carryforwards of approximately $0.3 million and $0.8 million
respectively, available to reduce future tax liabilities.
Results of Operations
Comparison of the Years Ended
The following table summarizes our results of operations for the years ended
2021/2020 2020/2019
Years Ended Comparison Comparison
December 31, Increase/(Decrease) Increase/(Decrease)
2021 2020 2019 $ % $ %
(in thousands) (in thousands, except percentages)
Revenue:
Product revenue, net $ 38,544 $ - $ - $ 38,544 100% $ - 0%
Operating expenses:
Cost of goods sold 9,100 - - 9,100 100% - 0%
Collaboration
expenses 835 - - 835 100% - 0%
Research and
development 99,297 112,042 135,001 (12,745) (11)% (22,959) (17)%
Selling, general and
administrative 85,948 45,321 34,962 40,627 90% 10,359 30%
Total operating
expenses 195,180 157,363 169,963 37,817 24% (12,600) (7)%
Loss from operations (156,636) (157,363) (169,963) 727 0% 12,600 (7)%
Interest income 97 1,134 6,049 (1,037) (91)% (4,915) (81)%
Loss before
(provision) benefit
for income taxes (156,539) (156,229) (163,914) (310) 0% 7,685 (5)%
(Provision) benefit
for income taxes (1,385) (5,152) 2,047 3,767 (73)% (7,199) (352)%
Net loss $ (157,924) $ (161,381) $ (161,867) $ 3,457 (2)% $ 486 0%
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Product Revenue, Net
Following the FDA approval of ARCALYST in March 2021 , we began generating
product revenue from sales of ARCALYST in April 2021 . We recognize product
revenue, net of ARCALYST at the transaction price when the specialty pharmacy or
specialty distributors obtains control of our products, which typically occurs
upon shipment of the product from the third party logistics provider. We
recognized net revenue from the sale of ARCALYST of $38.5 million for the year
ended December 31, 2021 .
Cost of goods sold
Upon the first sale commencing in April 2021 , we began generating cost of goods
sold associated with the sales of ARCALYST. We recognized cost of goods sold
from the sale of ARCALYST of $9.1 million for the year ended December 31, 2021 ,
which includes $0.8 million for the amortization of the regulatory milestone.
During the year ended December 31, 2021 , we sold the remaining relabeled
ARCALYST product previously acquired to support the clinical trials which
carried zero cost.
Research and Development Expenses
2021/2020 2020/2019
Years Ended Comparison Comparison
December 31, Increase/(Decrease) Increase/(Decrease)
2021 2020 2019 $ % $ %
Direct research and
development expenses by
program:
Rilonacept $ 10,842 25,729 $ 25,677 $ (14,887) (58)% $ 52 0%
Mavrilimumab 30,704 25,862 13,840 4,842 19% 12,022 87%
Vixarelimab 10,739 8,796 28,772 1,943 22% (19,976) (69)%
KPL-404 5,316 3,738 22,848 1,578 42% (19,110) (84)%
Unallocated research and
development expenses:
Personnel related
(including share-based
compensation) 27,736 33,489 29,019 (5,753) (17)% 4,470 15%
Other 13,960 14,428 14,845 (468) (3)% (417) (3)%
Total research and
development expenses $ 99,297 $ 112,042 $ 135,001 $ (12,745) (11)% $ (22,959) (17)%
Research and development expenses were $99.3 million for the year ended December
31, 2021 , compared to $112.0 million for the year ended December 31, 2020 , or a
decrease of $12.7 million . Research and development expenses were $112.0 million
for the year ended December 31, 2020 , compared to $135.0 million for the year
ended December 31, 2019 , or a decrease of $23.0 million .
Direct costs for our rilonacept program were $10.8 million , $25.7 million and
$25.7 million for the years ended December 31, 2021 , 2020 and 2019,
respectively. During the year ended December 31, 2021 , the decrease in expenses
incurred related primarily to the completion of RHAPSODY, our global, pivotal
Phase 3 clinical trial in recurrent pericarditis, and transition to the
long-term extension. During the year ended December 31, 2020 , expenses incurred
primarily related to conducting RHAPSODY, a milestone payment of $7.5 million
for the achievement of a specified regulatory milestone event under the
Regeneron Agreement, and supply chain costs. During the year ended December 31,
2019 , expenses incurred primarily related to our clinical research and
development for RHAPSODY, including $6.6 million related to purchases of drug
materials under our clinical supply agreement with Regeneron, as well as for our
open-label Phase 2 proof-of-concept clinical trial.
Direct costs of our mavrilimumab program were
respectively. During the year ended
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related to our Phase 2/3 clinical trial in COVID-19 related ARDS. During the year endedDecember 31, 2020 , expenses incurred primarily related to conducting our global Phase 2 clinical trial in GCA, including manufacturing costs for our clinical drug supply, and initiation of our Phase 2/3 clinical trial in COVID-19 related ARDS. During the year endedDecember 31, 2019 , expenses incurred primarily related to costs related to our global Phase 2 clinical trial in GCA. Direct costs for our vixarelimab program were$10.7 million ,$8.8 million and$28.8 million for the year endedDecember 31, 2021 , 2020 and 2019, respectively. During the year endedDecember 31, 2021 , expenses incurred related primarily to the initiation of our Phase 2b clinical trial in prurigo nodularis. During the year endedDecember 31, 2020 , expenses incurred related primarily to conducting our Phase 2a clinical trial in prurigo nodularis and our exploratory Phase 2 clinical trial in diseases characterized by chronic pruritus, which concluded earlier in the year. During the year endedDecember 31, 2019 , expenses incurred primarily related to a milestone payment of$10.0 million under the Biogen Agreement associated with the achievement of a specified clinical milestone event as well as expenses incurred for our Phase 2a clinical trial in prurigo nodularis, our exploratory Phase 2 clinical trial in diseases characterized by chronic pruritus and our Phase 1b clinical trial, and approximately$2.1 million of manufacturing and development costs for our clinical drug supply. Direct costs for our KPL-404 program were$5.3 million ,$3.7 million and$22.8 million for the years endedDecember 31, 2021 , 2020 and 2019, respectively. During the year endedDecember 31, 2021 , expenses incurred primarily related to manufacturing of drug product supply for our anticipated Phase 2 trial in rheumatoid arthritis. During the year endedDecember 31, 2020 , expenses incurred primarily related to preclinical and clinical trial costs for our Phase 1 trial of KPL-404 in healthy volunteers, including toxicology costs. During the year endedDecember 31, 2019 , expenses incurred primarily related to the acquisition of all of the outstanding securities of Primatope for aggregate upfront and contingent payments of$18.0 million paid in a combination of cash and Class A common shares in accordance with the Primatope Agreement. The Primatope acquisition was accounted for as an asset acquisition in 2019 as it did not meet the definition of a business. We recorded the upfront payment, milestone payments and the accrued milestone as research and development expense because the acquired technology represented in-process research and development and had no alternative future use. Unallocated research and development expenses were$41.7 million ,$47.9 million and$43.9 million for the years endedDecember 31, 2021 , 2020 and 2019, respectively. The decrease of$6.2 million in unallocated research and development expense in 2021 from 2020 was due to a decrease in cost associated with ARCALYST including quality control and supply chain of$4.9 million included in costs of goods sold beginning with the approval of ARCALYST. The increase of$4.0 million in unallocated research and development expenses in 2020 from 2019 was due to increases of$2.9 million in operating costs of our laboratory and$4.5 million in personnel-related costs primarily related to share-based compensation and additional personnel in our research and development functions to support our ongoing clinical trials, partially offset by decreases of$2.0 million related to the cessation of our development of a pre-clinical product candidate and$1.4 million in other operating expenses including costs related to our internal lab and costs related to preclinical studies and discovery research. The increase in personnel-related costs was primarily due to additional personnel in our research and development functions to support our ongoing clinical trials, including development and manufacture of clinical supply and regulatory filings. Personnel-related costs for the years endedDecember 31, 2021 , 2020 and 2019 included share-based compensation of$8.5 million ,$8.9 million and$5.7 million , respectively.
Selling, General and Administrative Expenses
Selling, general and administrative expenses were$85.9 million ,$45.3 million and$35.0 million for the years endedDecember 31, 2021 , 2020 and 2019. The increase of$40.6 million was primarily due to an increase of$22.0 million in personnel-related costs related to the build out of our commercial function, including hiring of a sales force and$8.9 million in marketing expenses associated with the commercial launch of ARCALYST. The increase of$10.3 million in 2020 from 2019 was primarily due to increases of$5.2 million in personnel-related costs and$6.7 million in marketing costs associated with the pre-commercialization activities to support our future launch of ARCALYST partially offset by a decrease$1.6 million of other expenses primarily due to a decrease in travel costs due to the travel restrictions associated with the COVID-19 pandemic and other miscellaneous professional fees. Personnel-related costs for the years endedDecember 30, 2021 , 2020 and 2019 included share-based compensation of$16.5 million ,$12.0 million and$9.3 million , respectively. 129 Table of Contents Collaboration Expenses Collaboration expenses was$0.8 million for the year endedDecember 31, 2021 . Our collaboration with Regeneron achieved profitability in the fourth quarter of 2021, following three quarters of commercial availability for recurrent pericarditis. We expect to continue to incur collaboration expenses associated with sales of ARCALYST. Interest Income Interest income was$0.1 million for the year endedDecember 31, 2021 , compared to interest income of$1.1 million for the year endedDecember 31, 2020 . The decrease was due primarily to lower interest rates onU.S. Treasury notes and a lower average balance in short term investments. Interest income was$1.1 million for the year endedDecember 31, 2020 , compared to$6.0 million for the year endedDecember 31, 2019 . The decrease was primarily due to lower interest rates onU.S. Treasury notes.
(Provision) Benefit for Income Taxes
For the year endedDecember 31, 2021 , we recorded a provision for income taxes of$1.4 million relating primarily toU.S. current taxes from the cost-plus arrangement less amounts related to the impact of Foreign Derived Intangible Income, or FDII deduction andU.S. federal and state research credits. As ofDecember 31, 2021 we maintained a full valuation allowance against our deferred tax assets of$127.9 million because we believe, more likely than not, it is not realizable. For the year endedDecember 31, 2020 , we recorded a provision for income taxes of$5.2 million relating primarily to the recognition of the valuation allowance and the current year tax expense. For the year endedDecember 31, 2019 , we recorded a$2.0 million benefit for income taxes relating primarily to the impact of the FDII deduction, andU.S. federal and state research and development tax credits.
Liquidity and Capital Resources
As of
equivalents and short-term investments, which totaled
losses were
ended
operating losses for the foreseeable future.
OnFebruary 4, 2019 , we completed a follow-on offering of 2,654,984 Class A common shares at a public offering price of$18.26 per share and concurrent private placement of 2,000,000 Class A1 common shares at an offering price of$18.26 per share for aggregate gross proceeds of$85.0 million . In addition, onMarch 1, 2019 , we completed the sale of 161,126 Class A common shares to the underwriters of the follow-on offering following the exercise in part of their option to purchase additional shares at a public offering price of$18.26 per share for gross proceeds of$2.9 million . The aggregate net proceeds to us from the follow-on offering and concurrent private placement, inclusive of the option exercise, was$83.0 million after deducting underwriting discounts and commissions, placement agent fees and other offering costs. OnMay 18, 2020 , we completed a follow-on offering of 2,760,000 Class A common shares, inclusive of the exercise of the underwriters' overallotment option at a public offering price of$18.25 per share and a concurrent private placement of 1,600,000 Class A1 common shares at an offering price of$18.25 per share for aggregate gross proceeds of$79.6 million . The aggregate net proceeds to us from the follow-on offering and concurrent private placement, inclusive of the option exercise, was$74.5 million after deducting underwriting discounts and commissions, placement agent fees and other offering costs. OnJuly 24, 2020 , we completed a follow-on offering of 5,952,381 Class A common shares, at a public offering price of$21.00 and a concurrent private placement of 1,428,572 Class A1 common shares at an offering price of$21.00 per share for aggregate gross proceeds of$155.0 million . The estimated aggregate net proceeds to us from the follow-on offering and concurrent private placement was$146.0 million after deducting underwriting discounts and commissions, placement agent fees and other offering costs. 130
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Under various agreements with third parties, we have agreed to make milestone payments, pay royalties, annual maintenance fees and to meet due diligence requirements based upon specified milestones. Under our license agreement with Regeneron, we have entered into supply agreements to provide both clinical and commercial product. We have committed to minimum payments to Regeneron of$43.6 million , of which$30.6 million are due within one year. We have entered into lease agreements for office and laboratory space, and vehicles, with total future lease payments of$6.3 million , of which$3.6 million are due within one year. These agreements impact our short-term and long-term liquidity and capital needs.
As of
investments of
Cash Flows
The following table summarizes our cash flows for each of the periods presented:
Years Ended
December 31,
2021 2020 2019
(in thousands)
Net cash used in operating activities $ (126,298) $ (136,532) $ (158,369)
Net cash (used in) provided by investing
activities 128,635 (23,444) 49,214
Net cash provided by financing activities 5,885 227,086 84,107
Net increase (decrease) in cash and cash
equivalents and restricted cash $ 8,222 $ 67,110 $ (25,048)
Operating Activities
During the year ended December 31, 2021 , operating activities used $126.3
million of cash, primarily resulting from our net loss of $157.9 million ,
partially offset by non-cash charges of $30.9 million . Net cash used in our
operating assets and liabilities for the year ended December 31, 2021 consisted
of a $10.3 million increase in accrued expenses and other liabilities primarily
due to increases in the accrued costs for our clinical trials and accrual of the
2021 employee bonus, a $3.9 million increase in accounts receivable due to the
start of sales for ARCALYST, and a $3.7 million increase in inventory.
During the year ended December 31, 2020 , operating activities used $136.5
million of cash, primarily resulting from our net loss of $161.4 million and net
cash used in our operating assets and liabilities of $4.3 million , partially
offset by non-cash charges of $29.2 million . Net cash used in our operating
assets and liabilities for the year ended December 31, 2020 consisted of a $5.0
million decrease in accounts payable primarily due to the timing of vendor
invoicing and payments, a $8.8 million increase in accrued expenses and other
liabilities primarily due to increases in the accrued costs for our clinical
trials and pre-commercialization activities for ARCALYST, a $0.5 million
increase in other long-term liabilities, a $1.7 million decrease in operating
lease liabilities due to monthly payments for our right-of-use assets, a $5.6
million increase in other long term assets due to payments associated with
minimum balance requirements of our clinical trials which are not expected to be
completed within a year, and $1.3 million increase in prepaid expenses and other
current assets.
During the year ended December 31, 2019 , operating activities used $158.4
million of cash, primarily resulting from our net loss of $161.9 million and net
cash used by changes in our operating assets and liabilities of $17.0 million
partially offset by non-cash charges of $20.5 million . Net cash used by changes
in our operating assets and liabilities for the year ended December 31, 2019 ,
consisted of a $15.0 million decrease in accrued milestones, a $4.7 million
decrease in accounts payable, and a $1.3 million decrease in operating lease
liabilities partially offset by a $1.0 million increase in prepaid expenses and
other current assets, a $0.3 million increase in other long-term liabilities and
a $4.6 million increase in accrued expenses. The decrease in accrued milestones
resulted from the payment of outstanding milestones for which the expense was
recognized in prior years. The decrease in accounts payable was primarily due to
the timing of vendor invoicing and payments. The increase in prepaid expenses
and other current assets was due to increases in
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prepaid insurance expenses and prepaid expenses to CROs related to our clinical trials. The decrease in operating lease liabilities is due to monthly payments for our right-of-use assets. Investing Activities During the year endedDecember 31, 2021 , investing activities provided$128.6 million of cash, consisting of$306.3 million from proceeds of maturities of short-term investments, offset by$157.3 million of purchases of short-term investments and$20.0 million related to the payment of a regulatory milestone incurred under the Regeneron Agreement. During the year endedDecember 31, 2020 , investing activities used$23.4 million of cash, consisting of$430.2 million of purchases of short-term investments and$0.3 million of purchases of property and equipment, partially offset by$407.1 million from proceeds of maturities of short-term investments. During the year endedDecember 31, 2019 , investing activities provided$49.2 million of cash, consisting of$541.2 million from proceeds of maturities of short-term investments partially offset by$488.8 million of purchases of short-term investments and$3.2 million of purchases of property and equipment.
Financing Activities
During the year ended
activities was
employee share options and our ESPP.
During the year endedDecember 31, 2020 , net cash provided by financing activities was$227.1 million , consisting of net proceeds of$220.5 million in aggregate from our issuance and sale of Class A common shares in two follow-on public offerings, inclusive of the exercise of the underwriters' option to purchase additional Class A common shares, as applicable, and the concurrent issuances and sales of Class A1 common shares in two private placements, after the deduction of underwriting discounts and commissions, placement agent fees and other offering costs, and$6.6 million of proceeds primarily from the exercise of share options and our employee share purchase plan. During the year endedDecember 31, 2019 , net cash provided by financing activities was$84.1 million , consisting of net proceeds of$83.0 million from our issuance and sale of Class A common shares in a follow-on public offering, inclusive of the concurrent issuance and sale of Class A1 common shares in a private placement, and exercise in part of the underwriters' option to purchase additional Class A common shares after the deduction of underwriting discounts and commissions, placement agent fees and other offering costs.
Funding Requirements
We expect to incur significant expenses in connection with our ongoing and planned activities as we continue to commercialize ARCALYST and advance our current and future product candidates through preclinical and clinical development, seek regulatory approval and commercialize one or more of our current or future product candidates, if approved. In addition, if we obtain marketing approval for any of our current or future product candidates, we expect to incur significant additional commercialization expenses related to such activities. We may also incur expenses in connection with the in-licensing or acquisition of additional product candidates. As a result, we expect to incur additional expenses related to milestone, royalty and other payments payable to third parties with whom we have entered into license, acquisition and other similar agreements to acquire the rights to our product candidates. Additionally, we expect to continue to incur costs associated with operating as a public company, including significant legal, accounting, investor relations and other expenses. We expect to incur expenses as we:
? conduct our current and planned clinical trials for our current and future
product candidates;
increase clinical and commercial manufacturing capabilities or make
? arrangements with additional third party manufacturers to successfully
manufacture our products and product candidates;
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? develop and timely deliver clinical grade and commercial grade product
formulations that can be used in our clinical trials and for commercial sale;
? seek regulatory approvals for any product candidates that successfully complete
clinical trials;
maintain, establish, and/or expand a sales, marketing, medical affairs and
? distribution infrastructure to commercialize ARCALYST or any of our current or
future product candidates for which we may obtain marketing approval and intend
to commercialize on our own;
? launch commercial sales of any of our current or future product candidates, if
and when approved, whether alone or in collaboration with others;
? make milestone or other payments under any current or future license,
acquisition, collaboration or other strategic transaction agreements;
expand our operational, financial and management systems and increase personnel
? globally to support our clinical development, manufacturing and
commercialization efforts and our operations as a public company;
? maintain, expand and protect our intellectual property portfolio; and
? in-license or acquire other product candidates and technologies or their
related businesses, if we determine to do so.
We believe that our existing cash, cash equivalents and short-term investments will enable us to fund our operating expenses and capital expenditure requirements for at least the next 12 months. The future viability of the Company is dependent on its ability to fund its operations through sales of ARCALYST and/or raise additional capital, such as through debt or equity offerings, as needed. We have based these estimates on assumptions that may prove to be wrong, and we could utilize our available capital resources sooner than we expect. We anticipate that we may require additional capital if we choose to pursue in-licenses or acquisitions of other product candidates and technologies or their related businesses. We expect to continue to incur significant expenses related to product manufacturing, sales, marketing and distribution of ARCALYST. In addition, if we obtain regulatory approval for any of our current or future product candidates, pursue additional indications for our products or any of our current or future product candidates, we expect to incur significant expenses related to product development and manufacturing, sales, marketing and distribution, depending on where we choose to commercialize.
Because of the numerous risks and uncertainties associated with research,
development and commercialization of biologic products, we are unable to
estimate the exact amount of our working capital requirements. Our future
funding requirements may be impacted by a number of factors, including those
described in Part I, Item 1A. “Risk Factors” in this Annual Report.
Until such time, if ever, as we can generate substantial and sustained product
revenue, we expect to finance our cash needs through a combination of public or
private equity offerings, debt financings, or other sources, including,
licensing, collaboration, marketing, distribution or other strategic
transactions or arrangements with third parties. To the extent that we raise
additional capital through the sale of equity or convertible debt securities,
our shareholders' ownership interest may be materially diluted, and the terms of
such securities could include liquidation or other preferences that adversely
affect our shareholders' rights as a common shareholder. Debt financing and
preferred equity financing, if available, may involve agreements that include
restrictive covenants that limit our ability to take specified actions, such as
incurring additional debt, making capital expenditures or declaring dividends.
In addition, debt financing would result in fixed payment obligations.
If we raise funds through licensing, collaboration, marketing, distribution or
other strategic transactions or arrangements with third parties, we may have to
relinquish valuable rights to our technologies, product candidates or
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future revenue streams, or otherwise agree to terms that may not be favorable to us. If we are unable to obtain funding, we could be forced to delay, reduce or eliminate some or all of our research and development programs for product candidates, product portfolio expansion or commercialization efforts, which could adversely affect our business prospects, or we may be unable to continue operations.
Critical Accounting Policies and Significant Judgments and Estimates
Our consolidated financial statements are prepared in accordance with generally accepted accounting principles inthe United States . The preparation of our consolidated financial statements and related disclosures requires us to make estimates and judgments that affect the reported amounts of assets, liabilities, revenue, costs and expenses, and the disclosure of contingent assets and liabilities in our financial statements. We base our estimates on historical experience, known trends and events and various other factors that we believe are reasonable under the circumstances, the results of which form the basis for making judgments about the carrying values of assets and liabilities that are not readily apparent from other sources. We evaluate our estimates and assumptions on an ongoing basis. Our actual results may differ from these estimates under different assumptions or conditions.
While our significant accounting policies are described in more detail in Note 2
to our consolidated financial statements included elsewhere in this Annual
Report, we believe that the following accounting policies are those most
critical to the judgments and estimates used in the preparation of our
consolidated financial statements.
Revenue Recognition
ASC 606 outlines a five-step process for recognizing revenue from contracts with customers: (i) identify the contract with the customer, (ii) identify the performance obligations in the contract, (iii) determine the transaction price, (iv) allocate the transaction price to the separate performance obligations in the contract, and (v) recognize revenue associated with the performance obligations as they are satisfied. We only apply the five-step model to contracts when it is probable that we will collect the consideration we are entitled to in exchange for the goods or services we transfer to the customer. Once a contract is determined to be within the scope of ASC 606, we determine the performance obligations that are distinct. We recognize as revenues the amount of the transaction price that is allocated to each respective performance obligation when the performance obligation is satisfied or as it is satisfied. Generally, our performance obligations are transferred to customers at a point in time, typically upon receipt of the product by the customer. ASC 606 requires entities to record a contract asset when a performance obligation has been satisfied or partially satisfied, but the amount of consideration has not yet been received because the receipt of the consideration is conditioned on something other than the passage of time. ASC 606 also requires an entity to present a revenue contract as a contract liability in instances when a customer pays consideration, or an entity has a right to an amount of consideration that is unconditional (e.g. receivable), before the entity transfers a good or service to the customer.
Product Revenue, Net
Net revenue from product sales is recognized at the transaction price when the
specialty pharmacy or specialty distributors obtains control of our products,
which occurs at a point in time, typically upon shipment of the product from the
third party logistics provider.
Our net revenues represent total revenues adjusted for discounts and allowances,
including estimated cash discounts, chargebacks, rebates, returns, copay
assistance, and specialty pharmacy and distributor fees. These adjustments
represent variable consideration under ASC 606 and are estimated using the
expected value method and are recorded when revenue is recognized on the sale of
the product. These adjustments are established by us as our best estimate based
on available information and will be adjusted to reflect known changes in the
factors that impact such allowances. Adjustments for variable consideration are
determined based on the contractual terms with customers, historical trends,
communications with customers and the levels of inventory remaining in the
distribution channel, as well as expectations about the market for the product
and anticipated introduction of competitive products.
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As of
reserve would not result in a material change in our net revenue.
As part of the process of preparing our consolidated financial statements, we are required to estimate our accrued research and development expenses. This process involves reviewing open contracts and purchase orders, communicating with our personnel to identify services that have been performed on our behalf and estimating the level of service performed and the associated cost incurred for the service when we have not yet been invoiced or otherwise notified of actual costs. The majority of our service providers invoice us in arrears for services performed, on a pre-determined schedule or when contractual milestones are met; however, some require advanced payments. We make estimates of our accrued expenses as of each balance sheet date in the consolidated financial statements based on facts and circumstances known to us at that time. We periodically confirm the accuracy of these estimates with the service providers and make adjustments if necessary. Examples of estimated accrued research and development expenses include fees paid to:
? vendors, including research laboratories, in connection with preclinical
development activities;
? CROs and investigative sites in connection with preclinical studies and
clinical trials;
? third parties in the connection with the achievement of milestones due under
license acquisition and other similar agreements; and
? CMOs in connection with drug substance and drug product formulation and
manufacturing of materials.
We base our expenses related to preclinical studies and clinical trials on our estimates of the services received and efforts expended pursuant to quotes and contracts with multiple research institutions and CROs that conduct and manage preclinical studies and clinical trials on our behalf. The financial terms of these agreements are subject to negotiation, vary from contract to contract and may result in uneven payment flows. There may be instances in which payments made to our vendors will exceed the level of services provided and result in a prepayment of the expense. Payments under some of these contracts depend on factors such as the successful enrollment of patients and the completion of clinical trial milestones. Non-refundable prepayments determined to be used within one year for goods or services that will be used or rendered for future research and development activities are recorded as prepaid expenses. Non-refundable prepayments or minimum balance requirements associated to clinical trials determined to not be used within one year are classified as other long-term assets. In accruing service fees, we estimate the time period over which services will be performed and the level of effort to be expended in each period. If the actual timing of the performance of services or the level of effort varies from the estimate, we adjust the accrual or the amount of prepaid expenses accordingly. Although we do not expect our estimates to be materially different from amounts actually incurred, our understanding of the status and timing of services performed relative to the actual status and timing of services performed may vary and may result in reporting amounts that are too high or too low in any particular period. To date, there have not been any material adjustments to our prior estimates of accrued research and development expenses.
As of
development expenses.
Share-Based Compensation
We measure all share-based awards granted to employees and directors based on
their fair value on the date of the grant and recognize compensation expense for
those awards over the requisite service period. Forfeitures are accounted for as
they occur. We issue share-based awards with both service-based vesting
conditions and performance-based vesting conditions. Expense for awards with
service-based vesting is recorded using the straight-line method, and expense
for awards with performance-based vesting conditions is recognized using the
accelerated-attribution method.
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For share-based awards granted to consultants and non-employees, compensation expense is recognized over the vesting period of the awards, which is generally the period during which services are rendered by such consultants and non-employees until completed.
We classified share-based compensation expense in the consolidated statements of
operations and comprehensive loss in the same manner in which the award
recipient’s payroll costs are classified or in which the award recipient’s
service payments are classified.
The fair value of each restricted share award was estimated on the date of grant
based on the fair value of the Company’s Class A common shares on that same
date.
The fair value of each option grant is estimated on the date of grant using the Black-Scholes option-pricing model, which requires inputs based on certain subjective assumptions, including the expected share price volatility, the expected term of the award, the risk-free interest rate, and expected dividends (see Note 11 to in our annual consolidated financial statements included elsewhere in this annual report). Prior toMay 2018 , we were a private company and, accordingly, lacked company-specific historical and implied volatility information. Therefore, we estimate our expected share price volatility based on a blend of our historical volatility and the historical volatility of publicly traded peer companies and expect to continue to do so until such time as we have adequate historical data regarding the volatility of our traded share price. The expected term of our share options has been determined utilizing the "simplified" method for awards that qualify as "plain-vanilla" share options. The expected term of share options granted to non-employees is equal to the contractual term of the share option award. The risk-free interest rate is determined by reference to theU.S. Treasury yield curve in effect at the time of grant of the award for time periods approximately equal to the expected term of the award. Expected dividend yield is based on the fact that we have never paid cash dividends and do not expect to pay any cash dividends in the foreseeable future. The fair value of each restricted share unit award is based on the closing price of our Class A common shares on the date of grant. Restricted share unit awards with an associated performance condition are evaluated on a regular basis for probability of achievement, to determine the timing of recording share-based compensation expense to include in our consolidated statements of operations and comprehensive loss.
Recently Issued Accounting Pronouncements
A description of recently issued accounting pronouncements that may potentially impact our financial position and results of operations is disclosed in Note 2 to our annual consolidated financial statements included elsewhere in this Annual Report.
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