Document and Entity Information
Document and Entity Information - USD ($) $ in Millions | 12 Months Ended | ||
Dec. 31, 2019 | Feb. 20, 2020 | Jun. 30, 2019 | |
Cover [Abstract] | |||
Entity Registrant Name | Akcea Therapeutics, Inc. | ||
Entity Central Index Key | 0001662524 | ||
Document Type | 10-K | ||
Document Period End Date | Dec. 31, 2019 | ||
Amendment Flag | false | ||
Trading Symbol | AKCA | ||
Current Fiscal Year End Date | --12-31 | ||
Entity Well-known Seasoned Issuer | No | ||
Entity Voluntary Filers | No | ||
Entity Current Reporting Status | Yes | ||
Entity Filer Category | Accelerated Filer | ||
Entity Public Float | $ 218.4 | ||
Entity Common Stock, Shares Outstanding | 101,073,964 | ||
Document Fiscal Year Focus | 2019 | ||
Document Fiscal Period Focus | FY | ||
Entity Small Business | false | ||
Entity Emerging Growth Company | true | ||
Entity Ex Transition Period | true | ||
Entity Shell Company | false | ||
Entity File Number | 001-38137 | ||
Entity Tax Identification Number | 47-2608175 | ||
Entity Address, Address Line One | 22 Boston Wharf Road | ||
Entity Address, Address Line Two | 9th Floor | ||
Entity Address, City or Town | Boston | ||
Entity Address, State or Province | MA | ||
Entity Address, Postal Zip Code | 02210 | ||
City Area Code | 617 | ||
Local Phone Number | 207-0202 | ||
Entity Interactive Data Current | Yes | ||
Title of 12(b) Security | Common Stock, $.001 Par Value | ||
Security Exchange Name | NASDAQ | ||
Entity Incorporation, State or Country Code | DE | ||
Document Annual Report | true | ||
Document Transition Report | false | ||
Documents Incorporated by Reference | DOCUMENTS INCORPORATED BY REFERENCE Portions of the Registrant’s definitive Information Statement with the Securities and Exchange Commission in connection with the Registrant’s annual meeting of stockholders are incorporated by reference into Part III of this Report. Such information statement will be filed with the Securities and Exchange Commission not later than 120 days following the end of the Registrant’s fiscal year ended December 31, 2019. |
CONSOLIDATED BALANCE SHEETS
CONSOLIDATED BALANCE SHEETS - USD ($) $ in Thousands | Dec. 31, 2019 | Dec. 31, 2018 |
Current assets: | ||
Cash and cash equivalents | $ 306,866 | $ 86,454 |
Short-term investments | 156,806 | 166,155 |
Accounts receivable | 10,496 | 4,597 |
Receivable from Ionis Pharmaceuticals, Inc. | 3,231 | |
Inventories | 8,817 | 85 |
Other current assets | 10,689 | 9,944 |
Total current assets | 496,905 | 267,235 |
Property, plant and equipment, net | 5,261 | 5,696 |
Operating lease right-of-use assets | 11,094 | |
Intangible assets, net | 83,051 | 88,914 |
Deposits and other assets | 2,939 | 3,416 |
Total assets | 599,250 | 365,261 |
Current liabilities: | ||
Accounts payable | 10,216 | 12,068 |
Payable to Ionis Pharmaceuticals, Inc. | 18,901 | |
Accrued compensation | 12,793 | 8,583 |
Accrued liabilities | 14,191 | 14,787 |
Current portion of deferred revenue | 2,165 | 25,354 |
Other current liabilities | 2,633 | 968 |
Total current liabilities | 41,998 | 80,661 |
Long-term portion of lease liabilities | 14,248 | 4,442 |
Long-term portion of deferred revenue | 3,434 | |
Total liabilities | 56,246 | 88,537 |
Stockholders’ equity: | ||
Common stock, $0.001 par value; 125,000,000 shares authorized at December 31, 2019 and 2018; 100,993,173 and 89,345,978 shares issued and outstanding at December 31, 2019 and 2018, respectively. | 101 | 89 |
Additional paid-in capital | 1,024,168 | 799,001 |
Accumulated other comprehensive loss | 5 | (324) |
Accumulated deficit | (481,270) | (522,042) |
Total stockholders’ equity | 543,004 | 276,724 |
Total liabilities and stockholders’ equity | $ 599,250 | $ 365,261 |
CONSOLIDATED BALANCE SHEETS (Pa
CONSOLIDATED BALANCE SHEETS (Parenthetical) - $ / shares | Dec. 31, 2019 | Dec. 31, 2018 |
Stockholders’ equity: | ||
Common stock, par value (in dollars per share) | $ 0.001 | $ 0.001 |
Common stock, shares authorized (in shares) | 125,000,000 | 125,000,000 |
Common stock, shares issued (in shares) | 100,993,173 | 89,345,978 |
Common stock, shares outstanding (in shares) | 100,993,173 | 89,345,978 |
CONSOLIDATED STATEMENTS OF OPER
CONSOLIDATED STATEMENTS OF OPERATIONS - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Revenue: | |||
Revenue | $ 488,543 | $ 64,867 | $ 43,401 |
Expenses: | |||
Cost of sales - intangible asset amortization | 5,690 | 2,713 | 0 |
Research and development | 292,852 | 130,340 | 126,890 |
Selling, general and administrative | 181,681 | 153,610 | 36,981 |
Total expenses | 450,469 | 295,683 | 163,871 |
Income (loss) from operations | 38,074 | (230,816) | (120,470) |
Other income (expense): | |||
Investment income | 5,505 | 5,631 | 1,813 |
Interest expense | 0 | 0 | (1,731) |
Other (expense) income | (615) | (189) | 104 |
Income (loss) before income tax expense | 42,964 | (225,374) | (120,284) |
Income tax expense | (2,192) | (447) | (1,275) |
Net income (loss) | 40,772 | (225,821) | (121,559) |
Ionis [Member] | |||
Expenses: | |||
Net loss share from commercial activities under arrangement with Ionis Pharmaceuticals, Inc. | $ (39,723) | $ 0 | 0 |
Preferred Stock [Member] | |||
Other income (expense): | |||
Net income (loss) | $ (28,385) | ||
Net loss per share, basic and diluted (in dollars per share) | $ 0 | $ 0 | $ (1.80) |
Weighted-average shares outstanding, basic and diluted (in shares) | 0 | 0 | 15,748,009 |
Net income (loss) per share of common stock, basic | $ (1.80) | ||
Weighted-average shares of common stock outstanding, basic | 15,748,009 | ||
Common Stock [Member] | Ionis [Member] | |||
Other income (expense): | |||
Net income (loss) | $ 34,073 | $ (163,938) | $ (63,638) |
Net income (loss) per share of common stock, basic | $ 0.49 | $ (2.74) | $ (3.08) |
Weighted-average shares of common stock outstanding, basic | 70,099,576 | 59,812,394 | 20,669,446 |
Net income (loss) per share of common stock, diluted | $ 0.48 | $ (2.74) | $ (3.08) |
Weighted-average shares of common stock outstanding, diluted | 70,099,576 | 59,812,394 | 20,669,446 |
Common Stock [Member] | Others [Member] | |||
Other income (expense): | |||
Net income (loss) per share of common stock, basic | $ 0.29 | $ (2.87) | $ (3.08) |
Weighted-average shares of common stock outstanding, basic | 22,815,682 | 21,553,407 | 9,593,322 |
Net income (loss) per share of common stock, diluted | $ 0.29 | $ (2.87) | $ (3.08) |
Weighted-average shares of common stock outstanding, diluted | 25,282,273 | 21,553,407 | 9,593,322 |
Product [Member] | |||
Revenue: | |||
Revenue | $ 42,253 | $ 2,237 | $ 0 |
Expenses: | |||
Cost of product/license | 4,569 | 1,820 | 0 |
Research and Development Revenue Under Collaborative Agreements [Member] | |||
Revenue: | |||
Revenue | 436,118 | 50,630 | 43,401 |
Licensing [Member] | |||
Revenue: | |||
Revenue | 10,172 | 12,000 | 0 |
Expenses: | |||
Cost of product/license | $ 5,400 | $ 7,200 | $ 0 |
CONSOLIDATED STATEMENTS OF COMP
CONSOLIDATED STATEMENTS OF COMPREHENSIVE INCOME (LOSS) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Statement Of Income And Comprehensive Income [Abstract] | |||
Net income (loss) | $ 40,772 | $ (225,821) | $ (121,559) |
Unrealized gains (losses) on investments, net of tax | 237 | 144 | (337) |
Currency translation adjustment | 92 | (17) | (93) |
Comprehensive income (loss) | $ 41,101 | $ (225,694) | $ (121,989) |
CONSOLIDATED STATEMENTS OF CONV
CONSOLIDATED STATEMENTS OF CONVERTIBLE PREFERRED STOCK AND STOCKHOLDERS' EQUITY (DEFICIT) - USD ($) shares in Thousands, $ in Thousands | Total | Ionis [Member] | Ionis [Member]TEGSEDI [Member] | Ionis [Member]TTR License Agreement [Member] | Convertible Preferred Stock [Member] | Common Stock [Member] | Common Stock [Member]Ionis [Member]TEGSEDI [Member] | Common Stock [Member]Ionis [Member]TTR License Agreement [Member] | Additional Paid In Capital [Member] | Additional Paid In Capital [Member]Ionis [Member] | Additional Paid In Capital [Member]Ionis [Member]TEGSEDI [Member] | Additional Paid In Capital [Member]Ionis [Member]TTR License Agreement [Member] | Accumulated Other Comprehensive Loss [Member] | Accumulated Deficit [Member] |
Balance at Dec. 31, 2016 | $ (17,747) | $ 100,000 | $ 56,936 | $ (21) | $ (174,662) | |||||||||
Balance (in shares) at Dec. 31, 2016 | 28,885 | |||||||||||||
Net income (loss) | (121,559) | (121,559) | ||||||||||||
Change in unrealized gains (losses), net of tax | (337) | (337) | ||||||||||||
Currency translation adjustment | (93) | (93) | ||||||||||||
Conversion of convertible preferred stock to common stock | $ (100,000) | $ 29 | 99,971 | |||||||||||
Conversion of convertible preferred stock to common stock (in shares) | (28,885) | 28,885 | ||||||||||||
Issuance of stock | 132,291 | $ 18 | 132,273 | |||||||||||
Issuance of stock (in shares) | 17,969 | |||||||||||||
Issuance of common stock in connection with the conversion of the line of credit with Ionis Pharmaceuticals Inc. together with accrued interest | 107,731 | $ 14 | 107,717 | |||||||||||
Issuance of common stock in connection with the conversion of the line of credit with Ionis Pharmaceuticals Inc. together with accrued interest (in shares) | 13,438 | |||||||||||||
Issuance of common stock in connection with private placement | 50,000 | $ 6 | 49,994 | |||||||||||
Issuance of common stock in connection with private placement (in shares) | 6,250 | |||||||||||||
Stock-based compensation expense | 17,539 | 17,539 | ||||||||||||
Balance at Dec. 31, 2017 | 167,825 | $ 67 | 464,430 | (451) | (296,221) | |||||||||
Balance (in shares) at Dec. 31, 2017 | 66,542 | |||||||||||||
Net income (loss) | (225,821) | (225,821) | ||||||||||||
Change in unrealized gains (losses), net of tax | 144 | 144 | ||||||||||||
Currency translation adjustment | (17) | (17) | ||||||||||||
Exercise of common stock options | 6,622 | $ 1 | 6,621 | |||||||||||
Exercise of common stock options (in shares) | 831 | |||||||||||||
Issuance of common stock in connection with employee stock purchase plan | 341 | 341 | ||||||||||||
Issuance of common stock in connection with employee stock purchase plan (in shares) | 32 | |||||||||||||
Issuance of restricted common stock (in shares) | 5 | |||||||||||||
Issuance of stock | $ 90,000 | $ 200,112 | $ 3 | $ 18 | $ 89,997 | $ 200,094 | ||||||||
Issuance of stock (in shares) | 3,269 | 18,667 | ||||||||||||
Stock-based compensation expense | 44,282 | 44,282 | ||||||||||||
Distribution to Ionis in connection with the TTR license transaction | (7,792) | (7,792) | ||||||||||||
Capital contribution from Ionis | $ 1,028 | $ 1,028 | ||||||||||||
Balance at Dec. 31, 2018 | 276,724 | $ 89 | 799,001 | (324) | (522,042) | |||||||||
Balance (in shares) at Dec. 31, 2018 | 89,346 | |||||||||||||
Net income (loss) | 40,772 | 40,772 | ||||||||||||
Change in unrealized gains (losses), net of tax | 237 | 237 | ||||||||||||
Currency translation adjustment | 92 | 92 | ||||||||||||
Exercise of common stock options | 10,752 | $ 1 | 10,751 | |||||||||||
Exercise of common stock options (in shares) | 2,489 | |||||||||||||
Issuance of common stock in connection with employee stock purchase plan | 822 | 822 | ||||||||||||
Issuance of common stock in connection with employee stock purchase plan (in shares) | 40 | |||||||||||||
Issuance of restricted common stock (in shares) | 9 | |||||||||||||
Issuance of stock | 200,000 | $ 10 | 199,990 | |||||||||||
Issuance of stock (in shares) | 9,711 | |||||||||||||
Stock-based compensation expense | 37,112 | 37,112 | ||||||||||||
Distribution to Ionis in connection with the TTR license transaction | $ (13,492) | $ (13,492) | ||||||||||||
Payments of tax withholdings related to exercise of employee stock options | (10,015) | $ 1 | (10,016) | |||||||||||
Payments of tax withholdings related to vesting of employee stock options (in shares) | (602) | |||||||||||||
Balance at Dec. 31, 2019 | $ 543,004 | $ 101 | $ 1,024,168 | $ 5 | $ (481,270) | |||||||||
Balance (in shares) at Dec. 31, 2019 | 100,993 |
CONSOLIDATED STATEMENTS OF CASH
CONSOLIDATED STATEMENTS OF CASH FLOWS - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Operating activities: | |||
Net income (loss) | $ 40,772 | $ (225,821) | $ (121,559) |
Adjustments to reconcile net income (loss) to net cash provided by (used in) operating activities: | |||
Depreciation | 789 | 307 | 108 |
Amortization of right-of-use operating lease assets | 942 | ||
Amortization of intangibles | 5,863 | 2,870 | 120 |
Amortization of discount/premium on investment securities, net | (438) | (23) | 499 |
Non-cash interest expense for line of credit with Ionis Pharmaceuticals, Inc. | 1,731 | ||
Non-cash sublicensing expense | 200,000 | 33,394 | |
Stock-based compensation expense | 37,112 | 44,282 | 17,539 |
Changes in operating assets and liabilities: | |||
Accounts receivable | (5,899) | 816 | (5,413) |
Other current and long-term assets | (5,316) | (8,680) | (1,761) |
Inventory | (4,545) | (85) | |
Accounts payable | (610) | 8,444 | 1,905 |
Receivable/payable to Ionis Pharmaceuticals, Inc. | (22,132) | 4,527 | (43,385) |
Long-term income tax receivable | 861 | ||
Accrued compensation | 4,210 | 4,500 | 1,578 |
Accrued liabilities | (1,877) | 8,420 | 6,572 |
Income taxes payable | 716 | (540) | 1,789 |
Deferred revenue | (26,623) | (41,905) | 70,693 |
Net cash provided by (used in) operating activities | 223,825 | (202,888) | (36,190) |
Investing activities: | |||
Purchases of short-term investments | (186,988) | (136,895) | (301,377) |
Proceeds from maturity of short-term investments | 197,012 | 208,559 | 98,778 |
Purchase of property, plant and equipment | (1,596) | (1,119) | (9) |
Net cash provided by (used in) investing activities | 8,428 | 70,545 | (202,608) |
Financing activities: | |||
Proceeds from exercise of common stock options and employee stock purchase plan issuances | 11,574 | 6,963 | |
Payments of tax withholdings related to exercise of employee stock awards | (10,015) | ||
Distribution to Ionis Pharmaceuticals, Inc. | (13,492) | (7,792) | |
Proceeds from issuance of common stock to Ionis Pharmaceuticals in TTR transaction | 163,660 | ||
Proceeds from issuance of common stock, net of underwriters' discount | 135,438 | ||
Proceeds from sale of common stock to Novartis in private placement | 50,000 | ||
Proceeds from line of credit from Ionis Pharmaceuticals, Inc. | 106,000 | ||
Offering costs paid | (2,037) | ||
Net cash (used in) provided by financing activities | (11,933) | 162,831 | 289,401 |
Effect of exchange rates on cash | 92 | (17) | (93) |
Net increase in cash and cash equivalents | 220,412 | 30,471 | 50,510 |
Cash, cash equivalents and restricted cash at beginning of period | 88,838 | 58,367 | 7,857 |
Cash, cash equivalents and restricted cash at end of period | 309,250 | 88,838 | 58,367 |
Supplemental disclosures of non-cash investing and financing activities: | |||
Right-of-use assets obtained in exchange for lease liabilities | 363 | ||
Purchase of property, plant and equipment included in accounts payable | 1,252 | ||
Purchase of property, plant and equipment included in long-term deferred rent liability | 3,555 | ||
Acquisition of research and development licenses and milestone payments | 90,563 | ||
Conversion of preferred stock to common stock upon initial public offering | 100,000 | ||
Conversion of line of credit from Ionis Pharmaceuticals, Inc. into common stock | 107,731 | ||
Cash, cash equivalents and restricted cash reported within the condensed consolidated balance sheets: | |||
Cash and cash equivalents | 306,866 | 86,454 | 58,367 |
Restricted cash included in deposits and other assets | 2,384 | 2,384 | |
Cash, cash equivalents and restricted cash at end of period | $ 309,250 | 88,838 | $ 58,367 |
Ionis [Member] | |||
Supplemental disclosures of non-cash investing and financing activities: | |||
Capital contribution from Ionis Pharmaceuticals, Inc. | $ 1,028 |
Organization and Basis of Prese
Organization and Basis of Presentation | 12 Months Ended |
Dec. 31, 2019 | |
Organization Consolidation And Presentation Of Financial Statements [Abstract] | |
Organization and Basis of Presentation | 1. Organization and Basis of Presentation We were incorporated in Delaware in December 2014. We were organized by Ionis Pharmaceuticals, Inc., or Ionis, to focus on developing and commercializing medicines to treat patients with rare and serious diseases. On July 19, 2017, we completed our initial public offering, or IPO. As of December 31, 2019, Ionis owned approximately 76% of our common stock and is our majority shareholder. Prior to our IPO, we were wholly owned by Ionis. The accompanying consolidated financial statements have been prepared in conformity with accounting principles generally accepted in the United States of America, or U.S. GAAP. Certain amounts in the prior period financial statements have been revised to conform to the presentation of the current period financial statements. The consolidated financial statements include the accounts of Akcea Therapeutics, Inc. ("we," "our," and "us") and our wholly owned subsidiaries. All intercompany transactions and balances were eliminated in consolidation. We included all normal recurring adjustments in the financial statements which we considered necessary for a fair presentation of our financial position and our operating results and cash flows for the years ended December 31, 2019, 2018 and 2017. In accordance with Accounting Standard Codification, or ASC, 205-40, Going Concern |
Summary of Significant Accounti
Summary of Significant Accounting Policies | 12 Months Ended |
Dec. 31, 2019 | |
Accounting Policies [Abstract] | |
Summary of Significant Accounting Policies | 2. Summary of Significant Accounting Policies Use of Estimates The preparation of financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities, the disclosure of contingent assets and liabilities at the date of the financial statements and the reported amounts of revenue and expenses during the reporting periods. Significant estimates and assumptions reflected in these financial statements include, but are not limited to, revenue recognition, the accrual for research and development expenses, stock-based compensation and income taxes. Estimates are periodically reviewed in light of changes in facts, circumstances and experience. Changes in estimates are recorded in the period in which they become known. Actual results could differ from those estimates. Translation of Foreign Currency For our foreign subsidiaries that report in a functional currency other than U.S. dollars, we translate their assets and liabilities into U.S. dollars using the exchange rate at the balance sheet date. We translate revenue and expenses at the monthly average exchange rates for the period. We translate transactions in our capital accounts at the historic exchange rate in effect at the date of the transaction. We include foreign currency translation adjustments in accumulated other comprehensive income (loss) as a component of stockholders’ equity. Concentration of Credit Risk Financial instruments that potentially subject us to concentrations of credit risk consist primarily of cash, cash equivalents, short-term investments and accounts receivable. We place our cash, cash equivalents and short-term investments with reputable financial institutions. We primarily invest our excess cash in commercial paper and debt instruments of the U.S. Treasury, financial institutions, corporations and U.S. government agencies with strong credit ratings and an investment grade rating at or above A-1, P-1 or F-1 by Moody's, Standard & Poor's, or S&P, or Fitch, respectively. We have established guidelines relative to diversification and maturities that maintain safety and liquidity. We periodically review and modify these guidelines to maximize trends in yields and interest rates without compromising safety and liquidity. Cash Equivalents and Short-Term Investments We consider all liquid investments with maturities of three months or less when we purchase them to be cash equivalents. Our short-term investments have initial maturities of greater than three months from the date of purchase. We classify our short-term investments as available-for-sale and we carry them at fair market value based upon prices for identical or similar items on the last day of the fiscal period. We record unrealized gains and losses as a separate component of comprehensive income (loss) and we include net realized gains and losses in investment income on our consolidated statement of operations. We use the specific identification method to determine the cost of securities sold. Accounts Receivable Our accounts receivable balance is comprised of payments due from our partners in connection with our collaborative agreements and from our customers for product sales. We record receivables for product sales net of allowances for prompt payment discounts and other related fees and discounts based on contractual terms with our customers. We have standard payment terms that generally require payment within 30 to 90 days. We do not adjust our receivables for the effects of a significant financing component at contract inception if we expect to collect the receivables in one year or less from the time of sale. We provide reserves against trade receivables for estimated losses that may result from a customer's inability to pay. Amounts determined to be uncollectible are charged or written-off against the reserve. As of December 31, 2019 and 2018, we did not recognize any reserves for uncollectible accounts. Inventory Prior to the regulatory approval of our product candidates, we incur expenses for the manufacturing of drug product that could potentially be available to support the commercial launch of our products. Until the first reporting period when regulatory approval has been received or is otherwise considered probable, we record all such costs as research and development expense. WAYLIVRA inventory-related costs incurred subsequent to April 1, 2019 and TEGSEDI inventory-related costs incurred subsequent to July 1, 2018 are reflected as inventory on our consolidated balance sheet at the lower of cost or net realizable value under the first-in, first-out, or FIFO, basis. We periodically analyze our inventory levels and write down inventory that has become obsolete, inventory that has a cost basis in excess of its estimated realizable value and inventory in excess of expected sales requirements as cost of product sales. The determination of whether inventory costs will be realizable requires estimates by our management and if actual market conditions are less favorable than projected by our management, additional write-downs of inventory may be required which would be recorded as a cost of product sales in the consolidated statement of operations. At December 31, 2018, a majority of our physical inventory for TEGSEDI was produced prior to when we obtained regulatory approval and accordingly had no cost basis as we recorded the related costs as research and development expense in prior periods. We obtained the first regulatory approval for TEGSEDI in July 2018 and for WAYLIVRA in May 2019. At December 31, 2019, our physical inventory for TEGSEDI and WAYLVRA included API that we produced prior to when we obtained regulatory approval. As such, this API has no cost basis as we had previously expensed these costs as R&D expenses. Property and Equipment Property and equipment are recorded at cost and depreciated using the straight-line method over the estimated useful life of each asset. Furniture and fixtures are depreciated over seven years. Computer equipment and software are depreciated over three years. Manufacturing equipment is depreciated over five years. Leasehold improvements are amortized over the shorter of the lease term or the ten-year estimated useful life of the asset. Upon retirement or sale, the cost of assets disposed of and the related accumulated depreciation are removed from the accounts and any resulting gain or loss is credited or charged to income. Repairs and maintenance costs are expensed as incurred. Leases Topic 842 Adoption In February 2016, the Financial Accounting Standards Board, or FASB, issued amended accounting guidance related to lease accounting. This guidance superseded the lease requirements we previously followed in ASC Topic 840, Leases Leases We adopted Topic 842 on January 1, 2019 and adjusted our opening consolidated balance sheet on that date to record our operating lease right-of-use assets and lease liabilities. We adopted Topic 842 using the available practical expedients permitted under the transition guidance within the new standard, which, among other things, allowed us to carry forward historical lease classification of those leases we had in place as of January 1, 2019. Results for the year ended December 31, 2019 are presented under Topic 842. Results for the year ended December 31, 2018 are presented in accordance with our historic accounting under Topic 840. The impact of the adoption of Topic 842 on the accompanying condensed consolidated balance sheet as of January 1, 2019 was as follows (in thousands): December 31, 2018 Adjustment due to adoption of Topic 842 January 1, 2019 Operating lease right-of-use assets — 11,932 11,932 Other current liabilities 968 1,029 1,997 Long-term portion of lease liabilities 4,442 10,915 15,357 We determine if an arrangement contains a lease at inception. We currently only have operating leases. We recognize an operating lease right-of-use asset and associated short and long-term lease liability for operating leases greater than one year on our consolidated balance sheet. We calculate our operating lease right-of-use asset and lease liability based on the present value of the future minimum lease payments we will pay over the lease term. We determine the lease term at the commencement date of the lease and we include renewal options in the lease term if we are reasonably certain that we will exercise the option. As our current leases do not provide an implicit interest rate, we used our incremental borrowing rate in determining the present value of future payments. We estimate the incremental borrowing rate based on the observed interest rates for secured debt issued by companies with similar credit ratings and with similar terms. Our operating lease right-of-use asset also includes any lease payments we made and excludes any tenant improvement allowances we received. We recognize rent expense for the lease components of our operating leases on a straight-line basis over the term of our lease. We recognize non-lease components, such as common area maintenance expenses, in the period we incur the expense. Intangible Assets We obtained exclusive licenses from Ionis for specific patents that Ionis owns and maintains related to our medicine pipeline. We recorded our licenses from Ionis as a capital contribution using the carryover basis of Ionis' historical cost for the related patents. We are amortizing our capitalized licenses over their estimated useful life, which is the term of the underlying individual patents owned by Ionis. In addition, we maintain definite-lived intangible assets related to regulatory milestone payments made to Ionis that are recoverable through future cash flows from approved products, which are capitalized as licensed intangible assets. These assets are amortized over their remaining useful lives, which are generally estimated to be the remaining patent life. If our estimate of the product’s useful life is shorter than the remaining patent life, then the shorter period is used. Intangible assets are amortized using the economic consumption method if anticipated future revenue can be reasonably estimated. The straight-line method is used when future revenue cannot be reasonably estimated. We use the straight-line method and amortization expense is recorded as a component of cost of sales to the extent the underlying license is related to a commercial product or research and development prior to product commercialization in the consolidated statement of operations. We assess our intangible assets for impairment if indicators are present or changes in circumstances suggest that impairment may exist. Events that could result in an impairment or trigger an interim impairment assessment may include actions by regulatory authorities with respect to us or our competitors, the receipt of additional clinical or nonclinical data regarding our medicine or a potentially competitive medicine, changes in the clinical development program for a medicine or new information regarding potential sales for the medicine. If impairment indicators are present or changes in circumstances suggest that impairment may exist, we perform a recoverability test by comparing the sum of the estimated undiscounted cash flows of each intangible asset to its carrying value on the consolidated balance sheet. If the undiscounted cash flows used in the recoverability test are less than the carrying value, we would determine the fair value of the intangible asset and recognize an impairment loss if the carrying value of the intangible asset exceeds its fair value. Fair Value of Financial Instruments We have estimated the fair value of our financial instruments. The amounts reported for cash equivalents, accounts payable and accrued expenses approximate fair value because of their short maturities. We report our investment securities at their estimated fair value based on a three-tier fair value hierarchy to prioritize the inputs used in our fair value measurements. These tiers include: Level 1, defined as observable inputs such as quoted prices in active markets for identical assets, which includes our money market funds and treasury securities classified as available-for-sale securities; Level 2, defined as inputs other than quoted prices in active markets that are either directly or indirectly observable, which includes our fixed income securities and commercial paper classified as available-for-sale securities; and Level 3, defined as unobservable inputs in which little or no market data exists, therefore requiring us to develop our own assumptions. We have not historically held any Level 3 investments. Our securities have been classified as Level 1 or Level 2. We obtain the fair value of our Level 2 investments from our custodian bank and from a professional pricing service. We validate the fair value of our Level 2 investments by understanding the pricing model used by the custodian banks or professional pricing service provider and comparing that fair value to the fair value based on observable market prices. We recognize transfers between levels of the fair value hierarchy on the date of the event or change in circumstances that caused the transfer . No transfers between levels have occurred to date Revenue Recognition Collaboration and License Revenue Effective January 1, 2018, we adopted Accounting Standards Codification, or ASC, Topic 606, Revenue from Contracts with Customers Under Topic 606, an entity recognizes revenue when its customer obtains control of promised goods or services in an amount that reflects the consideration which the entity expects to receive in exchange for those goods or services. To determine revenue recognition for arrangements that an entity determines are within the scope of Topic 606, the entity performs the following five steps: (i) identify the contract(s) with a customer; (ii) identify the performance obligations in the contract; (iii) determine the transaction price; (iv) allocate the transaction price to the performance obligations in the contract; and (v) recognize revenue when (or as) the entity satisfies a performance obligation. At contract inception, once the contract is determined to be within the scope of Topic 606, we assess the goods or services promised within each contract and determine those that are performance obligations, then assess whether each promised good or service is distinct. When we offer options for additional goods or services, such as an option to license a medicine in the future or for additional goods or services to be provided in the future, we evaluate whether such options are material rights that should be treated as additional performance obligations. We typically have concluded that the option to license a medicine or the options for additional goods or services that may be requested in the future under our collaboration agreements are not material rights as the amounts attributable to such options represent standalone selling price, and therefore no consideration is allocated to these items at the inception of an agreement. When a partner exercises its option to license a medicine or requests the additional goods or services, a new performance obligation is created for that item. Once performance obligations are identified, we then recognize as revenue the amount of the transaction price that we allocated to the respective performance obligation when (or as) each performance obligation is satisfied, either at a point in time or over time. If the performance obligation is satisfied over time, we recognize revenue based on the use of an output or input method. As of December 31, 2019, we had four collaboration and license revenue agreements: our strategic collaboration, option and license agreement with Novartis Pharma AG, or Novartis, which we entered into in January 2017; our TTR development, commercialization, collaboration and license agreement with Ionis, which we entered into in April 2018 and pursuant to which we are recognizing commercial product revenue related to TEGSEDI sales subsequent to product launch in the fourth quarter of 2018; our collaboration and license agreement with PTC Therapeutics International Limited, or PTC Therapeutics, which we entered into in August 2018; and our license agreement with Pfizer Inc., or Pfizer, which we entered into in October 2019. For a complete discussion of the accounting related to our license and collaboration agreements, see Note 7, Strategic Collaboration with Novartis, Note 8 , License Agreements and Services Agreement with Ionis , Note 9, Collaboration and License Agreement with PTC Therapeutics and Note 10 , License Agreement with Pfizer . Product Revenue, Net Subsequent to obtaining regulatory approval from the U.S. Food and Drug Administration, or FDA, on October 5, 2018, we began to sell TEGSEDI in the U.S. in the fourth quarter of 2018. The product is distributed through an exclusive distribution agreement with a third-party logistics company, or 3PL, that takes title to the product and represents our sole customer in the U.S. Our U.S. customer distributes TEGSEDI to a specialty pharmacy and a specialty distributor (collectively referred to as “wholesalers”), who then distribute the product to health care providers and patients. On July 11, 2018, we obtained regulatory approval of TEGSEDI in Europe, following which we began to sell TEGSEDI in the E.U. in the fourth quarter of 2018. On May 7, 2019, we obtained regulatory approval of WAYLIVRA in the E.U., following which we began to sell WAYLIVRA in the E.U. in the third quarter of 2019. TEGSEDI and WAYLIVRA are distributed through 3PLs that distribute the product to hospitals and pharmacies in the E.U . Revenue from product sales is recognized when the customer obtains control of our product, which occurs upon transfer of title to the customer. Revenue is recognized at the amount that we expect to be entitled to in exchange for the sale of our product. This amount includes both fixed and variable consideration and excludes amounts that are collected from customers and remitted to governmental authorities. We record shipping and handling costs related to commercial products within cost of goods sold on our consolidated statement of operations. We classify payments to customers or other parties in the distribution channel for services that are distinct and priced at fair value as selling, general and administrative expenses on our consolidated statement of operation. Payments to customers or other parties in the distribution channel that do not meet those criteria are classified as a reduction of revenue, as discussed further below. We have elected not to adjust consideration for the effects of a significant financing component when the period between the transfer of a promised good or service to the customer and when the customer pays for that good or service will be one year or less. Our payment terms are generally between thirty to ninety days . Reserves for Variable Consideration Revenue from product sales is recorded at the net sales price (transaction price), which includes estimates of variable consideration for which reserves are established related to discounts, returns, chargebacks, rebates, co-pay assistance and other allowances that are offered within contracts between us and our customers, wholesalers, health care providers and other indirect customers relating to the sale of TEGSEDI and WAYLIVRA. These reserves are based on the amounts earned or to be claimed on the related sales and are classified as a reduction of accounts receivable or a current liability. Where appropriate, these estimates take into consideration a range of possible outcomes that are probability-weighted for relevant factors such as our historical experience, current contractual and statutory requirements, specific known market events and trends, industry data and forecasted customer buying and payment patterns. Overall, product revenue net of these reserves reflects our best estimate of the amount of consideration to which we are entitled based on the terms of the contract. The amount of variable consideration that is included in the transaction price may be constrained and is included in the net sales price only to the extent that it is considered probable that a significant reversal in the amount of the cumulative revenue recognized will not occur in a future period. Actual amounts of consideration ultimately received may differ from our estimates. If actual results in the future vary from our estimates, we will adjust these estimates, which would affect net product revenue and earnings, in the period such variances become known . The following are the components of variable consideration related to product revenue: Chargebacks: In the U.S., we estimate obligations resulting from contractual commitments with the government and other entities to sell products to qualified healthcare providers at prices lower than the list prices charged to our U.S. customer. Our U.S. customer charges us for the difference between what they pay for the product and the selling price to the qualified healthcare providers. We record reserves and reduce our product revenue for these chargebacks related to product sold to our U.S. customer during the reporting period as well as our estimate of product that remains in the distribution channel at the end of the reporting period that we expect will be sold to qualified healthcare providers in future periods . Our established reserve for chargebacks is included in accrued liabilities on our consolidated balance sheet . Government rebates: We are subject to discount obligations under government programs, including Medicaid and Medicare programs in the U.S., and similar programs in certain countries in Europe. We record reserves for government rebates based on statutory discount rates and estimated utilization in the period in which revenue is recognized. We estimate Medicaid and Medicare rebates based upon estimated payer mix. These reserves are recorded in the same period the related revenue is recognized, resulting in a reduction of product revenue and the establishment of a liability that is included in accrued expenses on our consolidated balance sheet. For Medicare, we also estimate the number of patients in the prescription drug coverage gap for whom we expect we will owe an additional liability under the Medicare Part D program. On a quarterly basis, we update our estimates and record any adjustments in the period that we identify the adjustments . Managed care rebates: We are subject to rebates in connection with a value-based agreement with a certain commercial payer. Rebate accruals are recorded in the same period the related revenue is recognized, resulting in a reduction of product revenue and the establishment of a liability which is included in accrued liabilities on our consolidated balance sheet. This rebate is based on a fixed percentage and includes a price increase limit allowance (price protection). Our estimate for managed care rebates is based upon estimated payer mix and the applicable contractual rebate rate. Trade discounts and allowances: We provide customary invoice discounts on sales to our U.S. customer for prompt payment. The discounts are recorded in the same period the related revenue is recognized, resulting in a reduction of product revenue, and the establishment of a reserve that is offset against our accounts receivable balance on our consolidated balance sheet. Distribution fees: We receive and pay for various distribution services provided by our U.S. and E.U. customers and our U.S. wholesalers. These fees are generally accounted for as a reduction of revenue in the same period the related revenue is recognized, and an establishment of a reserve that is offset against our accounts receivable balance on our consolidated balance sheet. To the extent that the services received are distinct from the sale of products to our customers, we classify these payments as selling, general and administrative expenses. Product Returns: Our U.S. customer has return rights and our wholesalers in the U.S. have limited return rights primarily related to the product’s expiration date. We estimate the amount of product sales that may be returned and record the estimate as a reduction of revenue and a refund liability included in accrued liabilities in our consolidated balance sheet in the period the related product revenue is recognized. Based on our distribution model, contractual inventory limits with our U.S. customer and wholesalers and the price of TEGSEDI, we believe there will be minimal returns in the U.S. Our E.U. customers only take title to TEGSEDI and WAYLIVRA when an order is received and therefore only maintain inventory levels of our products based on demand. Accordingly, there is limited return risk in the E.U. and we have not recorded any return estimate in the transaction price for products sold in the E.U . Other incentives: In the U.S., other incentives include co-payment assistance that we provide to patients with commercial insurance that have coverage and reside in states that allow co-payment assistance. The calculation of the accrual for co-payment assistance is based on an estimate of claims and the cost per claim that we expect to receive associated with product that has been recognized as revenue. Our estimate is recorded in the same period the related revenue is recognized, resulting in a reduction of product revenue and the establishment of a liability which is included in accrued liabilities on our consolidated balance sheet . During the year ended December 31, 2019, we recorded product revenue, net, of $42.3 million, which consisted of $34.6 million of TEGSEDI sales in the U.S., and $7.7 million of TEGSEDI and WAYLIVRA sales in the E.U. This is compared to the year ended December 31, 2018 product revenue, net, of $2.2 million which consisted of $1.2 million of TEGSEDI sales in the U.S., and $1.0 million of TEGSEDI sales in the E.U. The following table summarizes balances and activity in each of the product revenue allowance and reserve categories for the years ended December 31, 2019 and 2018 (in thousands): Chargebacks, discounts and fees Government and other rebates Returns Total Balance at December 31, 2017 $ — $ — $ — $ — Provision related to current period sales 50 293 5 348 Adjustment related to prior period sales — — — — Credit or payments made during the period — — — — Balance at December 31, 2018 50 293 5 348 Provision related to current period sales 1,240 2,604 192 4,036 Adjustment related to prior period sales (4 ) (28 ) — (32 ) Credits or payments made during the period (1,016 ) (1,516 ) — (2,532 ) Balance at December 31, 2019 $ 270 $ 1,353 $ 197 $ 1,820 Cost of Product Sales As a result of receiving marketing authorization, or MA, approval in the E.U. for WAYLIVRA in May 2019 and TEGSEDI in July 2018, we began recording all WAYLIVRA and TEGSEDI expenses related to commercial product as cost of product sales starting in April 2019 and July 2018, respectively. Cost of product sales consists of manufacturing costs, transportation and freight, and indirect overhead costs associated with the commercial manufacturing and distribution of WAYLIVRA and TEGSEDI. Cost of product sales may also include period costs related to certain commercial manufacturing services and inventory adjustment charges. Additionally, we previously expensed a significant portion of the cost of producing WAYLIVRA and TEGSEDI that we used in the commercial launch of these products as research and development expense prior to the regulatory approval of WAYLIVRA and TEGSEDI. Commercial Sublicensing Expenses We incur sublicense expenses under our development, commercialization, collaboration and license agreement, or TTR License Agreement, and our development, commercialization and license agreement, or Cardiometabolic License Agreement, with Ionis related to the medicines we have licensed under these agreements. We include our sublicense fee expenses in cost of license expenses on our consolidated statement of operations for those medicines that are approved for marketing. We recognize sublicense fee expenses in the period they are incurred. Research and Development Expenses Our research and development expenses include wages, benefits, facilities, supplies, external services, clinical study and manufacturing costs and other expenses that are directly related to our research and development activities. We expense research and development costs as we incur them. We do not conduct research activities and no such costs are included in these amounts. If we make payments for research and development services prior to the services being rendered, we record those amounts as prepaid assets on our balance sheet and we expense them as the services are provided. Research and Development Sublicensing Expenses We incur sublicense expenses under our Cardiometabolic Development, Commercialization and License Agreement with Ionis related to the medicines we have licensed under the agreement. We include our sublicense fee expenses in research and development expenses on our consolidated statement of operations since the applicable medicines are not yet approved for marketing. We recognize sublicense fee expenses in the period they are incurred. Estimated Liability for Research and Development Costs We record accrued liabilities related to expenses for which vendors or service providers have not yet billed us. These liabilities are for products or services that we have received and primarily relate to ongoing nonclinical and clinical studies. These costs primarily include third-party clinical management costs, costs for contract research organizations, laboratory and analysis costs, toxicology studies and investigator grants. We have medicines in concurrent nonclinical and clinical studies at several sites throughout the world. To ensure that we have adequately provided for ongoing nonclinical and clinical research and development costs during the period in which we incur such costs, we maintain an accrual to cover these costs. We update our estimate for this accrual on at least a quarterly basis. The assessment of these costs is a subjective process that requires judgment. Upon settlement, these costs may differ materially from the amounts accrued in our consolidated financial statements. Our historical accrual estimates have not been materially different from our actual amounts. Stock-Based Compensation Expense We measure stock-based compensation expense for equity-classified awards, principally related to stock options, restricted stock units, or RSUs, and stock purchase rights under our employee stock purchase plan, or ESPP, based on the estimated fair value of the award on the date of grant. We recognize the value of the portion of the award that we ultimately expect to vest as stock-based compensation expense over the requisite vesting period in our consolidated statement of operations. We reduce stock-based compensation expense for estimated forfeitures at the time of grant and revise the expense in subsequent periods if actual forfeitures differ from those estimates. We value our stock option awards and stock purchase rights under our ESPP using the Black-Scholes model. The determination of the grant date fair value of options using an option pricing model is affected principally by our common stock fair value and requires us to make a number of other assumptions, including: the expected life of the option, the volatility of the underlying stock, the risk-free interest rate and expected dividends. The fair value of RSUs is based on the market price of our common stock on the date of grant. We have granted RSUs with various vesting terms between six months and four years. Prior to December 2015, Ionis granted our employees options to purchase shares of Ionis' common stock, or Ionis options. In December 2015, we granted our employees holding Ionis options additional options to purchase shares of our common stock, or Akcea options. We determined the stock-based compensation expense for the Ionis options at the date of grant and recognized compensation expense over the vesting period of the Ionis options. In December 2015, we accounted for the issuance of the Akcea options as a modification to the original grant of the Ionis options because the grant of the Ionis options and Akcea options essentially represented a single stock award as the exercisability provisions of the Ionis option grants and Akcea option grants were interrelate |
Investments and Fair Value Meas
Investments and Fair Value Measurements | 12 Months Ended |
Dec. 31, 2019 | |
Investments And Fair Value Measurements [Abstract] | |
Investments And Fair Value Measurements | 3. Investments and Fair Value Measurements Investments As of December 31, 2019 and 2018, we primarily invested our excess cash in money market funds and debt instruments of the U.S. Treasury, financial institutions, corporations and U.S. government agencies with strong credit ratings and an investment grade rating at or above A-1, P-1 or F-1 by Moody's, S&P or Fitch, respectively. We have established guidelines relative to diversification and maturities that maintain safety and liquidity. We periodically review and modify these guidelines to maximize trends in yields and interest rates without compromising safety and liquidity. The following is a summary of our investments at December 31, 2019 and 2018 (in thousands): Gross Unrealized Estimated December 31, 2019 Cost Gains Losses Fair Value Available-for-sale securities: Corporate debt securities $ 105,679 $ 40 $ (23 ) $ 105,696 Debt securities issued by U.S. government agencies 38,970 30 (6 ) 38,994 Total securities with a maturity of one year or less $ 144,649 $ 70 $ (29 ) $ 144,690 Corporate debt securities 2,033 5 — 2,038 Debt securities issued by U.S. government agencies 14,079 — (3 ) 14,076 Total securities with a maturity of one to two years 16,112 5 (3 ) 16,114 Total available-for-sale securities $ 160,761 $ 75 $ (32 ) $ 160,804 Gross Unrealized Estimated December 31, 2018 Cost Gains Losses Fair Value Available-for-sale securities: Corporate debt securities $ 81,770 $ — $ (151 ) $ 81,619 Debt securities issued by U.S. government agencies 85,578 — (42 ) 85,536 Total securities with a maturity of one year or less $ 167,348 $ — $ (193 ) $ 167,155 We recorded unrealized gains and losses related to the securities listed above as of December 31, 2019 and 2018. We believe that the unrealized losses associated with the decline in value of these securities is temporary and primarily related to the change in market interest rates since purchase. We believe it is more likely than not that we will be able to hold our debt securities to maturity. Therefore, we anticipate a full recovery of the amortized cost basis of our debt securities at maturity. All of our available-for-sale securities are available to us for use in our current operations. As a result, we categorized all of these securities as current assets even though the stated maturity of some individual securities may be one year or more beyond the balance sheet date. Fair Value Measurements The following tables present the investments we held at December 31, 2019 and 2018 that are regularly measured and carried at fair value. The table segregates our investments by the level within the fair value hierarchy of the valuation techniques we utilized to determine the respective securities' fair value (in thousands): At December 31, 2019 Quoted Prices in Active Markets (Level 1) Significant Other Observable Inputs (Level 2) Money market funds (1) $ 285,510 $ 285,510 $ — Corporate debt securities (2) 107,735 — 107,735 Debt securities issued by U.S. government agencies (3) 53,069 — 53,069 Total $ 446,314 $ 285,510 $ 160,804 At December 31, 2018 Quoted Prices in Active Markets (Level 1) Significant Other Observable Inputs (Level 2) Money market funds (1) $ 82,343 $ 82,343 $ — Corporate debt securities (2) 81,619 — 81,619 Debt securities issued by U.S. government agencies (3) 85,536 — 85,536 Total $ 249,498 $ 82,343 $ 167,155 (1) Included in cash and cash equivalents on our consolidated balance sheet. (2) At December 31, 2019 and 2018, $4.0 million and $1.0 million, respectively, was included in cash and cash equivalents on our consolidated balance sheet, with the difference included in short-term investments on our consolidated balance sheet. ( 3 ) Included in short-term investments on our consolidated balance sheet. |
Property Plant and Equipment
Property Plant and Equipment | 12 Months Ended |
Dec. 31, 2019 | |
Property Plant And Equipment [Abstract] | |
Property, Plant and Equipment | 4 . Property, Plant and Equipment The following table presents property and equipment, at cost, and related accumulated depreciation (in thousands): December 31, 2019 2018 Furniture and fixtures $ 1,611 $ 1,611 Computer equipment and software 289 102 Manufacturing equipment 416 — Leasehold improvements 3,955 4,213 Total property and equipment, at cost 6,271 5,926 Less accumulated depreciation and amortization (1,010 ) (230 ) Total property and equipment, net $ 5,261 $ 5,696 Total depreciation expense amounted to $0.8 million, $0.3 million and $0.1 million for the years ended December 31, 2019, 2018 and 2017, respectively. As part of the operating lease for our corporate headquarters, the landlord provided and we utilized a tenant improvement allowance of $3.6 million which is included in our leasehold improvements. |
Inventory
Inventory | 12 Months Ended |
Dec. 31, 2019 | |
Inventory Disclosure [Abstract] | |
Inventory | 5. Inventory The following table presents inventory (in thousands): Years Ended December 31, 2019 2018 Raw materials $ 6,520 $ — Work in process 2,039 — Finished goods 258 85 Total inventories $ 8,817 $ 85 We recorded inventory write-offs of $0.2 million for the year ended December 31, 2019. We did not record any inventory write-offs for the year ended December 31, 2018. |
Intangible Assets
Intangible Assets | 12 Months Ended |
Dec. 31, 2019 | |
Goodwill And Intangible Assets Disclosure [Abstract] | |
Intangible Assets | 6 . Intangible Assets The following table presents intangible assets (in thousands): December 31, Estimated 2019 2018 useful life Acquired and in-licensed rights $ 2,262 $ 2,262 7 - 21 Years Capitalized regulatory approval milestones 90,000 90,000 16 Years Less accumulated amortization (9,211 ) (3,348 ) Total intangible assets, net $ 83,051 $ 88,914 The capitalized regulatory approval milestones The Company recorded $5.9 million, $2.9 million and $0.1 million in amortization expense related to intangible assets during the years ended December 31, 2019, 2018 and 2017, respectively. We did not recognize any impairment losses for the years ended December 31, 2019, 2018 or 2017. Estimated future amortization expense for intangible assets as of December 31, 2019 is as follows (in thousands): Total 2020 $ 5,879 2021 5,861 2022 5,856 2023 5,843 2024 5,822 Thereafter 53,790 $ 83,051 The weighted average remaining amortizable life of our patents was 10.9 years at December 31, 2019. For additional detail of our license agreements with Ionis see Note 8, License Agreements and Services Agreement with Ionis |
Strategic Collaboration with No
Strategic Collaboration with Novartis | 12 Months Ended |
Dec. 31, 2019 | |
Novartis [Member] | |
Strategic Collaboration [Line Items] | |
Strategic Collaboration | 7 . Background In January 2017, we initiated a strategic collaboration with Novartis for the development and commercialization of AKCEA-APO(a)-L Rx Rx AKCEA-APO(a)-L Rx In February 2019, Novartis exercised its exclusive option to license AKCEA-APO(a)-L Rx AKCEA-APOCIII-L Rx In December 2019, we received written notice from Novartis that they elected not to exercise their option to license AKCEA-APOCIII-L Rx Rx . RX . Rx . Accounting Analysis We received a $75.0 million upfront payment in the first quarter of 2017, of which we retained $60.0 million and paid Ionis $15.0 million as a sublicense fee under our Cardiometabolic Development, Commercialization and License Agreement with Ionis. At commencement of our strategic collaboration, we identified the following four distinct performance obligations: • Development activities for AKCEA-APO(a)-L Rx • Development activities for AKCEA-APOCIII-L Rx • API for AKCEA-APO(a)-L Rx • API for AKCEA-APOCIII-L Rx The development activities and the supply of API were concluded to be distinct because Novartis or another third party could provide these items without our assistance. We determined the transaction price for the Novartis collaboration was $108.4 million, comprised of the following: • $75.0 million from the upfront payment we received; • $28.4 million for the premium paid by Novartis, which represented the excess of the fair value Ionis received from Novartis' purchase of Ionis' stock at a premium in the first quarter of 2017; and • $5.0 million for the premium Novartis would have paid to purchase Ionis' stock if we did not complete our IPO within 15 months of the inception of the agreement. We recognized revenue on the $75.0 million upfront payment plus the premium paid by Novartis from its purchase of Ionis’ stock and the premium associated with Novartis’ obligation to purchase Ionis’ stock if we did not complete our IPO because we were the party providing the services and API under the collaboration agreement with Novartis. None of the options or development or regulatory milestone payments under this agreement were included in the upfront transaction price determined in January 2017 as all payments were fully constrained at that time. As part of our evaluation of the constraint, we considered numerous factors, including the fact that achievement of the milestones is outside of our control and contingent upon the success of our clinical trials, Novartis’ efforts, and the receipt of regulatory approval. We re-evaluate the transaction price, including estimated variable consideration included in the transaction price and all constrained amounts, in each reporting period and as uncertain events are resolved or other changes in circumstances occur. Based on the distinct performance obligations under the Novartis collaboration, we allocated the $108.4 million transaction price based on relative stand-alone selling prices of each of our performance obligations as follows: • $64.0 million for development services for AKCEA-APO(a)-L Rx • $40.1 million for development services for AKCEA-APOCIII-L Rx • $1.5 million for the delivery of AKCEA-APO(a)-L Rx • $2.8 million for the delivery of AKCEA-APOCIII-L Rx We recognized revenue related to each of our performance obligations as follows: • In the second quarter of 2019, we completed the development services performance obligation for AKCEA-APO(a)-L Rx Rx • As a result of Novartis’ election to terminate the strategic collaboration in relation to AKCEA-APOCIII-L Rx Rx Rx • We recognized the amount attributed to the AKCEA-APO(a)-L Rx • We recognized the amount attributed to the AKCEA-APOCIII-L Rx Additionally, we and Ionis entered into a stock purchase agreement, or SPA, with Novartis in 2017. Under the SPA, in July 2017, Novartis purchased $50.0 million of our common stock in a separate private placement concurrent with the completion of our IPO at a price per share equal to the IPO price. On February 22, 2019, Novartis exercised its option to license AKCEA-APO(a)-L Rx Rx in the first quarter of 2019 because Novartis had full use of the license without any continuing involvement from us. Additionally, we did not have any further performance obligations related to the license after Novartis exercised its option to license AKCEA-APO(a)-L Rx Rx Rx w Rx Rx Rx Rx . In the third quarter of 2019, we delivered additional AKCEA-APO(a)- Rx API to AKCEA-APO(a)-L Rx We will earn the next milestone payment of $25.0 million under this collaboration if Novartis reaches a specific level of enrollment related to the Phase 3 study for AKCEA-APO(a)- Rx Rx . During the years ended December 31, 2019, 2018 and 2017, we recorded revenue of $187.4 million, $50.6 million and $43.4 million, respectively, from our strategic collaboration with Novartis. During the year ended December 31, 2019, we recognized $28.8 million of revenue from amounts that were in our beginning deferred revenue balance. There is no remaining deferred revenue related to the Novartis agreement on our consolidated balance sheet as of December 31, 2019. Our deferred revenue balance within our consolidated balance sheet at December 31, 2018 included $28.8 million related to our strategic collaboration with Novartis. |
License Agreements and Services
License Agreements and Services Agreement with Ionis | 12 Months Ended |
Dec. 31, 2019 | |
Related Party Transactions [Abstract] | |
License Agreements and Services Agreement with Ionis | 8 . In December 2015, we entered into a development, commercialization and license agreement related to our cardiometabolic franchise and a services agreement with Ionis. In March 2018, we entered into a new development, commercialization, collaboration and license agreement related to our TTR franchise and amended the services agreement previously in place with Ionis. The following sections summarize these related party agreements with Ionis. Cardiometabolic Development, Commercialization and License Agreement Our Cardiometabolic License Agreement with Ionis granted exclusive rights to us to develop and commercialize WAYLIVRA, AKCEA-APO(a)-L Rx Rx Rx We and Ionis share development responsibilities for the Lipid Drugs, other than the medicines licensed to Novartis and to Pfizer. We pay Ionis for the research and development expenses it incurs on our behalf, which include both external and internal expenses. External research and development expenses include costs for contract research organizations, or CROs, costs to conduct nonclinical and clinical studies on our medicines, costs to acquire and evaluate clinical study data, such as investigator grants, patient screening fees and laboratory work, and fees paid to consultants. Internal research and development expenses include costs for the work that Ionis' research and development employees perform for us. Ionis charges us a full-time equivalent rate that covers personnel-related expenses, including salaries and benefits, plus an allocation of facility-related expenses, including rent, utilities, insurance and property taxes, for those development employees who work either directly or indirectly on the development of our medicines. We also pay Ionis for the API and drug product we use in our nonclinical and clinical studies for all of our medicines. Ionis manufactures the API for us and charges us a price per gram consistent with the price Ionis charges its pharmaceutical partners, which includes the cost for direct materials, direct labor and overhead required to manufacture the API. If we need the API filled in vials or prefilled syringes for our clinical studies and Ionis contracts with a third party to perform this work, Ionis will charge us for the resulting cost. As we commercialize each of the Lipid Drugs other than medicines licensed to Novartis and to Pfizer, we will pay Ionis royalties from the mid-teens to the mid-twenty percent range on sales related to the Lipid Drugs that we sell. If we sell a Lipid Drug for a Rare Disease Indication (defined in the agreement as less than 500,000 patients worldwide or an indication that required a Phase 3 program of less than 1,000 patients and less than two years of treatment), we will pay a higher royalty rate to Ionis than if we sell a Lipid Drug for a Broad Disease Patient Population (defined in the agreement as more than 500,000 patients worldwide or an indication that required a Phase 3 program of 1,000 or more patients and two or more years of treatment). Other than with respect to AKCEA-APO(a)-L Rx Rx We may terminate this agreement if Ionis is in material breach of the agreement. Ionis may terminate this agreement if we are in material breach of the agreement. In each circumstance, the party that is in breach will have an opportunity to cure the breach prior to the other party terminating this agreement. In the first quarter of 2017, we entered into letter agreements with Ionis to reflect the agreed upon payment terms with respect to the upfront option payment that we received from Novartis and to allocate the premium that Novartis paid for Ionis' common stock in connection with our strategic collaboration with Novartis. In the fourth quarter of 2019, we entered into a letter agreement with Ionis to reflect the agreed upon payment terms with respect to the upfront license fee we received from Pfizer in connection with our license agreement with Pfizer. For additional detail regarding our strategic collaboration with Novartis, see Note 7, Strategic Collaboration with Novartis. License Agreement with Pfizer. TTR Development, Commercialization, Collaboration and License Agreement On April 17, 2018, our stockholders, other than Ionis and its affiliates, approved the TTR License Agreement and a stock purchase agreement, or Ionis SPA, with Ionis, which was entered into on March 14, 2018. In addition, in connection with these agreements, we entered into an amended and restated services agreement, or Amended Services Agreement, and an amended and restated investor rights agreement, or Amended Investor Rights Agreement, with Ionis. We determined that the TTR License Agreement and Ionis SPA included provisions that required the approval of our stockholders other than Ionis and its affiliates, which we deemed was not perfunctory in nature; therefore, we concluded that the approved date of the agreements for accounting purposes was April 17, 2018, the date on which such approval was received and the closing of the agreements took place. In accordance with the terms and provisions of the TTR License Agreement, we received rights to: • commercialize TEGSEDI following receipt of regulatory approval and perform certain other non-commercial activities with respect to TEGSEDI, in each case, in accordance with a global strategic plan; • partner on the completion of all pivotal studies of a follow-on medicine to TEGSEDI, AKCEA-TTR-L Rx , Rx • commercialize AKCEA-TTR-L Rx • share in profits and losses with respect to TEGSEDI and AKCEA-TTR-L Rx • manufacture (including through a third party) each product following receipt of regulatory approval for such product; and • sublicense the development and commercialization of either product to third parties or affiliates, with the consent of Ionis. As consideration for the grant of rights under the TTR License Agreement, we paid an upfront licensing fee of $150.0 million, which was paid through the issuance of 8 million shares of our common stock priced by reference to a trading average at the time of execution of the agreements. In addition, we are obligated to make milestone payments to Ionis in connection with the achievement of certain development, regulatory and commercialization events. These milestone payments include up to $110.0 million, if all TEGSEDI regulatory approval milestones are met; up to $145.0 million, if all AKCEA-TTR-L Rx The TTR License Agreement will remain in effect until the expiration of all included payment obligations, unless earlier terminated. The TTR License Agreement can be terminated by mutual consent of us and Ionis, by either us or Ionis upon certain events, by either party upon material breach, or by Akcea for convenience upon providing 90 days written notice to Ionis. Upon termination all rights received under the TTR License Agreement will terminate. To support the commercialization of TEGSEDI and AKCEA-TTR-L Rx In connection with the licensing transaction, we amended our Certificate of Incorporation to increase our authorized shares of common stock from 100,000,000 shares to 125,000,000 shares. We determined that the upfront accounting for the TTR License Agreement should follow the accounting guidance for common control transactions given the nature of the relationship between us and Ionis, including the fact that Ionis maintains a controlling ownership position in us. In addition, we assessed the identifiable assets that were acquired under the terms of the TTR License Agreement, including the licensed rights to TEGSEDI and AKCEA-TTR-L Rx In connection with the transaction, we also purchased $4.7 million of commercial TEGSEDI inventory held by Ionis. In addition, in the first quarter of 2019, we purchased $13.5 million of clinical TEGSEDI material held by Ionis. Prospectively we are responsible for the procurement of all additional inventory. The inventory and clinical material did not have a carrying value on the books of Ionis at the time of purchase. As such, in accordance with the accounting guidance for common control transactions above, we recorded the purchase of this inventory and clinical material as a reduction of additional paid in capital. This amount represented a cash distribution to Ionis; therefore, we have included this distribution as a distribution to Ionis for purposes of income (loss) per share and we have applied the two–class method as discussed in Note 15, Basic and Diluted Net Income (Loss) Per Share . We also determined that the TTR License Agreement represented a collaboration arrangement as defined by ASC 808. Prior to April 1, 2018, Ionis was responsible for all costs associated with TEGSEDI and for the period from April 1, 2018 to December 31, 2018, we were responsible for all costs associated with TEGSEDI. We and Ionis share all costs associated with AKCEA-TTR-L Rx . We recorded $3.1 million paid to Ionis for costs related to the period prior to the closing of the TTR License Agreement to equity, as these amounts were previously expensed in the financial statements of Ionis. This amount also represents a cash distribution to Ionis and was included as an adjustment to the net loss attributable to Ionis for purposes of applying the two-class method for income (loss) per share as discussed in Note 15, Basic and Diluted Net (Loss) Per Share . On July 11, 2018, we received regulatory approval in the E.U. for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis, or hATTR amyloidosis. As a result of the regulatory approval in the E.U., on August 3, 2018, we issued 1,597,571 shares of our common stock to Ionis as payment of the $40.0 million regulatory approval milestone for TEGSEDI. We capitalized this regulatory approval milestone payment as a licensed intangible asset on our consolidated balance sheet as the amount is expected to be recoverable through future cash flows. On October 5, 2018, we received regulatory approval for TEGSEDI from the FDA for the treatment of polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults in the U.S. As a result of the regulatory approval in the U.S., on October 17, 2018, we issued 1,671,849 shares of our common stock to Ionis as payment of the $50.0 million regulatory approval milestone for TEGSEDI. We capitalized this regulatory approval milestone payment as a licensed intangible asset on our consolidated balance sheet as the amount is expected to be recoverable through future cash flows. Both milestone payments are being amortized to cost of sales on a straight-line basis over the licensed asset’s expected useful life of approximately 16 years from the date of the initial regulatory approval milestone achievement. Amortization expense for the TTR milestone payments was $5.7 million and $2.7 million for the year ended December 31, 2019 and 2018, respectively. Profit/(Loss) Share Under the TTR License Agreement, we and Ionis agreed to share TEGSEDI and AKCEA-TTR-L Rx Rx Rx Rx In the first quarter of 2019, the profit sharing provisions for TEGSEDI under the TTR License Agreement with Ionis became effective. As we are the principal for all commercial activities related to the TTR License Agreement, we record all commercial activities related to TEGSEDI on a gross basis in our consolidated statement of operations, including revenues, cost of sales and sales and marketing expenses. The Ionis share of commercialization costs for TEGSEDI is separately presented within operating expenses in our consolidated statement of operations under the caption “Net loss share from commercial activities under arrangement with Ionis Pharmaceuticals, Inc.” As we are a collaborator with Ionis for the execution of TTR development activities, we record all research and development expenses on a net basis representing our proportionate share of total costs incurred by Ionis and us. Accordingly, only our share of total costs incurred related to development activities under the TTR License Agreement is presented within research and development expense in our consolidated statement of operations. A summary of the loss share related to the commercial activities under the TTR Agreement is as follows (in thousands): Year Ended December 31, 2019 Net losses incurred by the collaboration related to the commercial activities under the TTR License Agreement $ (66,210 ) Ionis' share of commercial losses under the TTR License Agreement reflected in our consolidated statements of operations (39,723 ) Akcea's share of commercial losses under the TTR License Agreement reflected in our consolidated statements of operations (26,487 ) A summary of the development expenses related to the TTR License Agreement is as follows (in thousands): Year Ended December 31, 2019 Total development expense incurred by the collaboration related to development activities under the TTR License Agreement $ (66,663 ) Akcea's share of TTR development expense reflected in research and development expense in our consolidated statements of operations (29,444 ) We did not record any commercial or research and development expense related to the profit/(loss) share under the TTR License Agreement for the years ended December 31, 2018 and 2017. Services Agreement We originally entered into a services agreement with Ionis in December 2015 in conjunction with the Cardiometabolic License Agreement. We entered into the Amended Services Agreement with Ionis in April 2018 in conjunction with the TTR License Agreement (collectively, the service agreements). The primary purpose of the Amended Services Agreement was to allow for the expansion of general and administrative services provided to us by Ionis to cover the TEGSEDI and AKCEA-TTR-L Rx Our services agreement with Ionis is designed to be flexible to adjust for our increasing capabilities in various functions. Under the services agreement, Ionis provides us certain services, including, without limitation, general and administrative support services and development support services. Ionis allocated a certain percentage of personnel to perform the services that it provides to us based on its good faith estimate of the required services. We pay Ionis for these allocated costs, which reflect the Ionis full-time equivalent, or FTE, rate for the applicable personnel, plus out-of-pocket expenses, such as occupancy costs, associated with the FTEs allocated to providing us these services. We do not pay a mark-up or profit on the external or internal expenses Ionis bills to us. Ionis invoices us quarterly for all amounts due under the services agreement and payments are due within 30 days of the receipt of an invoice. In addition, as long as Ionis continues to consolidate our financials, we will comply with Ionis' policies and procedures and internal controls. As long as we are consolidated into Ionis' financial statements under U.S. GAAP, we may continue to access the following services from Ionis: • investor relations services, • human resources and personnel services, • risk management and insurance services, • tax related services, • corporate record keeping services, • financial and accounting services, • credit services, and • COO/CFO/CBO oversight. However, if we wanted to provide the foregoing services internally, and doing so would not negatively impact Ionis' internal controls and procedures for financial reporting, we can negotiate in good faith with Ionis for a reduced scope of services related to the aforementioned services. When Ionis determines it should no longer consolidate our financials, we may mutually agree with Ionis in writing to extend the term of this arrangement in six-month increments. We can establish our own benefits programs or continue to use Ionis' benefits, however we must provide Ionis a minimum advance notice to opt-out of using Ionis' benefits. We do not currently plan to establish our own benefits programs at this time or in the near future. Pursuant to our various agreements with Ionis, as of December 31, 2019, Ionis owed us $3.2 million. As of December 31, 2018, we owed Ionis $18.9 million. The following table summarizes the amounts recorded related to transactions with Ionis including amounts related to the TTR License Agreement for the following periods (in thousands) (1): Years Ended December 31, 2019 2018 2017 Operating expenses: Services performed by Ionis $ 4,703 $ 15,404 $ 9,742 Active pharmaceutical ingredient manufactured by Ionis 5,515 5,229 6,012 Pre-commercial inventory manufactured by Ionis — 5,996 — Sublicensing expenses 205,400 7,200 48,394 Out-of-pocket expenses paid by Ionis 4,250 46,162 37,426 Royalty expenses 183 — — Less: commercial share of loss in connection with the TTR License Agreement (39,695 ) — — Plus: R&D share of loss in connection with the TTR License Agreement 3,651 — — Total operating expenses generated by transactions with Ionis 184,007 79,991 101,574 Plus: distributions to Ionis: 13,492 7,792 — Total charges generated by transactions with Ionis 197,499 87,783 101,574 Payable balance to Ionis at the beginning of the period 18,901 14,365 24,355 Less: total amounts received from (paid to) Ionis during the period (19,631 ) (83,247 ) (78,170 ) Less: non-cash sublicensing expenses (200,000 ) — (33,394 ) Total amount (receivable) payable (from) to Ionis at period end $ (3,231 ) $ 18,901 $ 14,365 (1) This table excludes amounts capitalized as license intangible assets on the balance sheet. |
Collaboration and License Agree
Collaboration and License Agreement with PTC Therapeutics | 12 Months Ended |
Dec. 31, 2019 | |
Collaboration And License Agreement [Abstract] | |
Collaboration and License Agreement with PTC Therapeutics | 9 . Collaboration and License Agreement with PTC Therapeutics In August 2018, we entered into a collaboration and license agreement with PTC Therapeutics, or the PTC License Agreement, to commercialize TEGSEDI and WAYLIVRA in Latin America and certain Caribbean countries, or the PTC Territory. We received a $12.0 million upfront payment from PTC Therapeutics related to TEGSEDI in the third quarter of 2018 upon execution of the PTC License Agreement, of which we paid Ionis $7.2 million as a sublicense fee related to the TTR License Agreement and recorded as a cost of license in our consolidated statement of operations. We received a $6.0 million payment from PTC Therapeutics in the second quarter of 2019 as a result of WAYLIVRA regulatory approval in Europe, of which we paid Ionis $3.0 million as a sublicense fee related to the Cardiometabolic License Agreement and recorded as a cost of license in our consolidated statement of operations. In the fourth quarter of 2019, TEGSEDI received approval in Brazil. As a result, we received a $4.0 million regulatory milestone payment from PTC Therapeutics, of which we paid Ionis $2.4 million as a sublicense fee related to the TTR License Agreement and recorded as a cost of license in our consolidated statement of operations. In addition, we are eligible to receive an additional $4.0 million for the achievement of a regulatory milestone and royalties in the mid-twenty percent range on net sales of TEGSEDI and WAYLIVRA in the PTC Territory. PTC Therapeutics’ obligation to pay royalties to us begins on the earlier of 12 months after the first commercial sale of a product in Brazil or the date that PTC Therapeutics recognizes revenue of at least $10.0 million in the PTC Territory. PTC Therapeutics will reduce these royalties upon the expiration of certain patents or if a generic competitor negatively impacts the market share of the product in the PTC Territory. Milestone payments and royalties that we are eligible to receive from PTC Therapeutics for TEGSEDI are split 60% to Ionis and 40% to Akcea. All WAYLIVRA milestone payments and royalties that we are eligible to receive from PTC are split 50/50 with Ionis. PTC Therapeutics is solely responsible for the commercialization of the products in the PTC Territory at its sole cost and expense, including the pursuit and maintenance of applicable regulatory approvals. Unless earlier terminated, the PTC License Agreement will continue in effect until the date on which the royalty term and all payment obligations with respect to all products in all countries in the PTC Territory have expired. At the commencement of the PTC License Agreement, we identified two performance obligations, consisting of the transfer of (1) the license to TEGSEDI and related know-how and (2) the license to WAYLIVRA and related know-how, both of which were satisfied during the third quarter of 2018. In addition, we identified a customer option related to PTC Therapeutics’ option to purchase supply of product from us for its development and commercial needs. We considered the manufacturing capabilities of PTC Therapeutics and the fact that manufacturing services are not proprietary and can be provided by other vendors, to conclude that the licenses have stand-alone functionality and are distinct. Further, the customer options for manufacturing of product are priced similar to other manufacturing options with similar customers and therefore not considered a material right. We determined the transaction price for the PTC License Agreement to be $12.0 million comprised of the upfront payment received. None of the regulatory milestones were included in the transaction price determined in August 2018, as all payments were fully constrained. As there were no remaining unsatisfied performance obligations as of September 30, 2018, the $12.0 million upfront payment was recognized as license revenue upon contract execution in the third quarter of 2018. The option to purchase supply from us is subject to the terms of a supply agreement we entered into with PTC Therapeutics in April 2019. In May 2019, we received $6.0 million of consideration from PTC Therapeutics as a result of regulatory approval of WAYLIVRA in Europe and in October 2019, we received $4.0 million of consideration from PTC Therapeutics as a result of TEGSEDI approval in Brazil. Since the constraints on the regulatory approvals were resolved, we updated the transaction price to include the additional milestone consideration that was deemed probable, and accordingly, we recognized the $6.0 million and $4.0 million as licensing revenue during the second and fourth quarter of 2019, respectively. As part of our evaluation of the constraint as of December 31, 2019, we considered numerous factors, including that regulatory approvals are not within our control and accordingly the remaining milestone is fully constrained and excluded from the arrangement consideration until such regulatory approval is received. We will continue to re-evaluate the transaction price, including estimated variable consideration included in the transaction price and all constrained amounts, in each reporting period and as uncertain events are resolved or other changes in circumstances occur. Any consideration related to sales-based royalties will be recognized when the related sales occur |
License Agreement with Pfizer
License Agreement with Pfizer | 12 Months Ended |
Dec. 31, 2019 | |
Related Party Transactions [Abstract] | |
License Agreement with Pfizer | 10 . License Agreement with Pfizer In October 2019, we entered into a license agreement, or the Pfizer License Agreement, with Pfizer for the development and commercialization of AKCEA-ANGPTL3-L Rx Under the terms of the Pfizer License Agreement, we granted Pfizer an exclusive license, with the right to grant certain sublicenses and to develop, manufacture, commercialize and otherwise exploit AKCEA-ANGPTL3-L Rx worldwide. We are responsible for completing a Phase 2 study and providing quantities of API for AKCEA-ANGPTL3-L Rx . We have the right to exercise an option, to participate in commercialization activities with Pfizer in the U.S. and certain additional markets. Accounting Analysis We received a $250.0 million upfront payment in the fourth quarter of 2019. We issued 6,873,344 shares of our common stock to Ionis as payment of the $125.0 million sublicense fee due under our Cardiometabolic License Agreement with Ionis. At commencement of the Pfizer License Agreement, we identified the following three distinct performance obligations: • License to develop and commercialize AKCEA-ANGPTL3-L Rx • Development activities for AKCEA-ANGPTL3-L Rx • API for AKCEA-ANGPTL3-L Rx We considered the manufacturing capabilities of Pfizer and the fact that manufacturing services are not proprietary and can be provided by another third party to conclude that the license has stand-alone functionality and is distinct. Further, the development activities and the supply of API are distinct because Pfizer or another third party could provide these items without our assistance. We determined the transaction price for the Pfizer License Agreement to be $250.0 million comprised of the upfront payment we received. None of the development or regulatory milestone payments under this agreement were included in the upfront transaction price as all future payments were fully constrained. As part of our evaluation of the constraint, we considered numerous factors, including the fact that achievement of the milestones is outside of our control and contingent upon the success of our clinical trials, Pfizer’s efforts, and the receipt of regulatory approval. We re-evaluate the transaction price, including estimated variable consideration included in the transaction price and all constrained amounts, in each reporting period and as uncertain events are resolved or other changes in circumstances occur. Based on the distinct performance obligations under the Pfizer License Agreement, we allocated the $250 million transaction price based on relative stand-alone selling prices of each of our performance obligations as follows: • $245.6 million for the transfer of the license of AKCEA-ANGPTL3-L Rx • $2.2 million for development services for AKCEA-ANGPTL3-L Rx • $2.2 million for the delivery of AKCEA-ANGPTL3-L Rx We are recognizing revenue related to each of our performance obligations as follows: • We recognized the full amount related to the license and related know-how in the fourth quarter of 2019 because Pfizer, upon the date of closing, had full use of the license and related know-how without any continuing involvement from us. Additionally, we did not have any further performance obligations related to the license after the license was transferred to Pfizer; • We are satisfying the development services performance obligation for AKCEA-ANGPTL3-L Rx Pfizer is responsible for conducting and funding all future development, regulatory and commercialization activities for AKCEA-ANGPTL3-L Rx • We recognized the amount attributed to the AKCEA-ANGPTL3-L Rx We will earn the next milestone payment of $75 million upon Pfizer’s initiation of a Phase 2b or Phase 3 study. In addition, we are eligible to receive up to $1.3 billion in milestone payments, including up to $205.0 million for the achievement of development milestones, up to $250.0 million for the achievement of regulatory milestones and up to $850.0 million for the achievement of commercialization milestones. We are also eligible to receive tiered, double digit royalties in the mid-teens to low twenty percent range on net sales of AKCEA-ANGPTL3-L Rx Rx During the year ended December 31, 2019, we recorded revenue of $248.7 million from our license agreement with Pfizer. Our consolidated balance sheet at December 31, 2019 includes deferred revenue of $1.3 million related to our development services obligation. |
Equity and Stock-Based Compensa
Equity and Stock-Based Compensation | 12 Months Ended |
Dec. 31, 2019 | |
Disclosure Of Compensation Related Costs Sharebased Payments [Abstract] | |
Equity and Stock-Based Compensation | 11 . Equity and Stock-Based Compensation Series A Convertible Preferred Stock In December 2015, we issued and sold to Ionis an aggregate of 28,884,540 shares of Series A convertible preferred stock for a total purchase price of $100.0 million plus the grant of the rights and licenses we received under the Cardiometabolic License Agreement with Ionis. The $100.0 million of proceeds we received was recorded in Series A convertible preferred stock on our consolidated balance sheet. We had 28,884,540 shares of Series A convertible preferred stock authorized, issued and outstanding as of December 31, 2016, of which all was held by Ionis. Conversion Shares of our Series A convertible preferred stock were convertible 1:1 into common stock, subject to certain adjustments for reorganizations, reclassifications, stock splits, stock dividends and dilutive issuances. All shares of Series A convertible preferred stock automatically converted into common stock upon completion of the IPO in July 2017. As of December 31, 2019 and 2018, we had no shares of Series A convertible preferred stock issued or outstanding. Our IPO is discussed in Note 13, Initial Public Offering Preferred Stock In July 2017, our board of directors approved an amendment and restatement of our certificate of incorporation to, among other things, change the authorized shares of our preferred stock to 10,000,000 shares with a par value of $0.001, all of which are undesignated. Our board of directors may establish the rights, preference and privileges of the preferred stock from time to time. The amended and restated certificate of incorporation was approved by our stockholders and became effective upon the completion of our IPO and the filing of the amended and restated certificate of incorporation with the State of Delaware in July 2017. As of December 31, 2019 and 2018, there were no shares of Preferred Stock outstanding. Common Stock We had 125,000,000 shares of common stock authorized at December 31, 2019 and 2018, of which 100,993,173 and 89,345,978 shares were issued and outstanding as of December 31, 2019 and 2018, respectively. In May 2017, our board of directors approved an amendment to our certificate of incorporation to (1) effect a reverse stock split on outstanding shares of our common stock and preferred stock on a one-for-2.555 basis, (2) change the authorized shares of our preferred stock to 40,000,000 and (3) modify the threshold for automatic conversion of our preferred stock into shares of our common stock in connection with an IPO to eliminate the price per share threshold and only require that we raise at least $50.0 million in gross proceeds (collectively, the "Charter Amendment"). The par values of the common stock and preferred stock were not adjusted as a result of the reverse stock split. The amendment to our certificate of incorporation was approved by our stockholder and became effective upon the filing with the State of Delaware in June 2017. All issued and outstanding common stock and preferred stock and related share and per share amounts contained in these consolidated financial statements have been retroactively adjusted to reflect the reverse stock split for all periods presented. Stock Plans 2015 Equity Incentive Plan In December 2015, our board of directors and stockholder adopted and approved our 2015 Equity Incentive Plan, or 2015 EIP. In May 2017 and June 2017, our board of directors and stockholder, respectively, approved an amendment to the 2015 EIP in order to, among other things, increase the number of shares of common stock reserved for issuance thereunder to 8,500,000 shares of common stock in conjunction with the IPO. As of December 31, 2019, the aggregate number of shares of common stock that may be issued pursuant to stock awards under the 2015 EIP was 18,500,000 shares. The 2015 EIP also provides for the grant of non-statutory stock options, or NSOs, incentive stock options, or ISOs, stock appreciation rights, restricted stock awards and restricted stock unit awards. At December 31, 2019, a total of 8,866,474 options were outstanding, of which 3,094,602 were exercisable, 701,252 restricted stock unit awards were outstanding, and 4,541,985 shares were available for future grant under the 2015 EIP. 2017 Employee Stock Purchase Plan In May 2017 and June 2017, our board of directors and stockholder, respectively, approved our ESPP, which became effective upon the completion of our IPO, and the reservation for issuance thereunder of 500,000 shares of common stock. In addition, the number of shares of common stock that may be issued under the ESPP will automatically increase commencing on January 1, 2018 and ending on (and including) January 1, 2027 in an amount equal to the lesser of (i) 1% of the total number of shares of common stock outstanding on December 31st of the preceding calendar year, and (ii) 500,000 shares of common stock. On January 1, 2019, 500,000 shares of common stock were added to the ESPP. Under the ESPP, participating employees can elect to have a portion of their base pay withheld during a consecutive payment period for the purchase of shares of our common stock. At the conclusion of each offering period, participating employees can purchase shares of our common stock at 85% of the lesser of the closing price at the beginning or at the end of the period. As of December 31, 2019, the aggregate number of shares of common stock reserved under the ESPP was 1,500,000 and we had 1,428,725 shares available for future issuance under the ESPP. During the year ended December 31, 2019, 39,724 shares were issued under our ESPP. At December 31, 2019, accrued liabilities included $0.4 million of ESPP contributions for which the related shares are issued in the first quarter of 2020. Stock Option Activity The following table summarizes the stock option activity for the year ended December 31, 2019 (in thousands, except per share and contractual life data) for the 2015 EIP: Number of Shares Weighted Average Exercise Price per Share Average Remaining Contractual Term (Years) Aggregate Intrinsic Value Outstanding at December 31, 2018 11,011 $ 15.00 Granted 4,756 $ 27.04 Exercised (1) (3,541 ) $ 7.99 Cancelled/forfeited/expired (3,360 ) $ 24.13 Outstanding at December 31, 2019 8,866 $ 20.81 7.97 $ 16,627 Exercisable at December 31, 2019 3,095 $ 14.68 6.43 $ 14,183 (1) This amount includes 1,052 shares withheld to cover the exercise price for options which were net exercised The weighted-average estimated fair value of options granted were $27.04, $18.29 and $10.40 for the years ended December 31, 2019, 2018 and 2017, respectively. For the years ended December 31, 2019, 2018 and 2017, there were 3,540,593 stock options, 834,800 stock options and no stock options exercised, respectively. As of December 31, 2019, total unrecognized estimated non-cash stock-based compensation expense related to non-vested stock options was $45.9 million. Our actual expenses may differ from these estimates because we will adjust our unrecognized non-cash stock-based compensation expense for future forfeitures. We expect to recognize the cost of non-cash stock-based compensation expense related to non-vested stock options over a weighted average amortization period of 1.45 years. Restricted Stock Unit Activity The following table summarizes restricted stock unit, or RSU, activity for year ended December 31, 2019 (in thousands, except per share data): Number of Shares Weighted Average Grant Date Fair Value per Share Non-vested at December 31, 2018 38 $ 25.65 Granted 706 $ 21.95 Vested (9 ) $ 25.76 Cancelled/forfeited/expired (34 ) $ 25.01 Non-vested at December 31, 2019 701 $ 21.95 For years ended December 31, 2019, 2018 and 2017, the weighted-average grant date fair value of RSUs granted was $21.95, $27.80 and $23.04 per RSU, respectively. As of December 31, 2019, total unrecognized compensation cost related to RSUs was $12.5 million. We will adjust the total unrecognized compensation cost for future changes in estimated forfeitures. We expect to recognize this cost over a weighted average period of 2.47 years. Stock-based Compensation Expense and Valuation Information The following table summarizes the breakdown of stock-based compensation expense by line item within the statement of operations for the years ended December 31, 2019, 2018 and 2017 (in thousands): Years Ended December 31, 2019 2018 2017 Cost of sales - product $ 437 $ 160 $ — Research and development expenses 11,895 9,435 8,630 Selling, general and administrative expenses 24,780 34,687 8,909 Total $ 37,112 $ 44,282 $ 17,539 Determining Fair Value Valuation. We measure stock-based compensation expense for equity-classified awards related to stock options and stock purchase rights under the ESPP at the grant date based on the estimated fair value of the award and we recognize the expense over the vesting period. We use the Black-Scholes model to estimate the fair value of stock options granted and stock purchase rights under our ESPP. The expected term of stock options granted represents the period of time that we expect them to be outstanding. We estimate the expected term of options granted based on actual and projected exercise patterns. We recognize compensation expense for stock options granted and stock purchase rights under the ESPP using the accelerated multiple-option approach. Under the accelerated multiple-option approach (also known as the graded-vesting method), an entity recognizes compensation expense over the requisite service period for each separately vesting tranche of the award as though the award were in substance multiple awards, which results in the expense being front-loaded over the vesting period. In valuing our options, we make a number of assumptions, including the risk-free interest rate, expected dividend yield, expected volatility, expected term, rate of forfeitures and fair value of common stock. We considered the following factors in applying these assumptions: Risk-Free Interest Rate. We determine the risk-free interest rate assumption based on the yields of U.S. Treasury securities with maturities that correspond to the term of the award. Expected Dividend Yield. We assume a dividend yield of zero as we have not paid dividends in the past and do not expect to pay dividends on our common stock for the foreseeable future. Expected Volatility. We do not have sufficient history to estimate the volatility of our common stock. We calculate expected volatility based on a blend of our historical volatility and reported data from selected publicly traded peer companies for which historical information is available. We plan to continue to use this blend to calculate our volatility until the historical volatility of our common stock is sufficient to measure expected volatility for future option grants. Expected Term. The expected term estimates represent the period of time that we expect the options to be outstanding. As we do not have historical information, we use the simplified method for estimating the expected term. Under the simplified method we calculate the expected term as the average time-to-vesting and the contractual life of the options. As we gain additional historical information, we will transition to calculating our expected term based on our exercise patterns. Rate of Forfeiture. We reduce stock-based compensation expense for estimated forfeitures. We estimate forfeitures at the time of grant and revise, if necessary, in subsequent periods if actual forfeitures differ from those estimates. Prior to the fourth quarter of 2019, we had estimated forfeitures based on Ionis’ historical rates as we did not have sufficient historical forfeiture information regarding our forfeiture activities. Beginning in the fourth quarter of 2019, we estimate forfeitures based on our historical experience as we determined that we have sufficient Akcea history for such an estimate. Fair Value of Common Stock. Prior to our IPO our board of directors estimated the fair value of our common stock considering, among other things, contemporaneous valuations of our common stock prepared by an unrelated third-party valuation firm in accordance with the guidance provided by the American Institute of Certified Public Accountants 2013 Practice Aid, Valuation of Privately-Held-Company Equity Securities Issued as Compensation. Subsequent to the IPO, we use the market closing price for our common stock on the date of grant as reported on Nasdaq to determine the fair value of our common stock on the date of grant. For the years ended December 31, 2019, 2018 and 2017, we used the following weighted-average assumptions in our Black-Scholes calculations for stock option grants under our 2015 EIP and ESPP: Employee Stock Options: Years Ended December 31, 2019 2018 2017 Risk-free interest rate 2.3 % 2.8 % 1.9 % Dividend yield 0.0 % 0.0 % 0.0 % Volatility 75.5 % 77.1 % 79.5 % Expected life 6.08 years 6.08 years 6.06 years Board of Director Stock Options: Years Ended December 31, 2019 2018 2017 Risk-free interest rate 1.8 % 2.9 % 1.9 % Dividend yield 0.0 % 0.0 % 0.0 % Volatility 73.8 % 78.2 % 79.4 % Expected life 6.25 years 6.42 years 6.25 years ESPP: Years Ended December 31, 2019 2018 2017 Risk-free interest rate 2.4 % 1.9 % 1.1 % Dividend yield 0.0 % 0.0 % 0.0 % Volatility 60.0 % 64.2 % 73.3 % Expected life 6 months 6 months 6 months |
Income Taxes
Income Taxes | 12 Months Ended |
Dec. 31, 2019 | |
Income Tax Disclosure [Abstract] | |
Income Taxes | 1 2 . Income Taxes Income (loss) before income taxes is comprised of (in thousands): Years Ended December 31, 2019 2018 2017 United States $ 40,475 $ (218,794 ) $ (108,691 ) Foreign 2,489 (6,580 ) (11,593 ) Income (loss) before income tax expense $ 42,964 $ (225,374 ) $ (120,284 ) The provision (benefit) for income taxes is comprised of (in thousands): Years Ended December 31, 2019 2018 2017 Current: Federal $ 1,692 $ — $ — State 87 73 1,041 Foreign 413 374 234 Total current 2,192 447 1,275 Income tax expense $ 2,192 $ 447 $ 1,275 Our expense (benefit) for income taxes differs from the amount computed by applying the U.S. federal statutory rate to income (loss) before taxes. The sources and tax effects of the differences are as follows (in thousands) : Years Ended December 31, 2019 2018 2017 Pretax income (loss) $ 42,964 $ (225,374 ) $ (120,284 ) Statutory rate 9,023 21.0 % (47,329 ) 21.0 % (42,099 ) 35.0 % State income tax net of federal benefit (3,025 ) (7.0 )% (6,441 ) 2.9 % (2,371 ) 2.0 % Impact of foreign tax rate differential 340 0.8 % 1,735 (0.8 )% 4,072 (3.4 )% Net change in valuation allowance (2,072 ) (4.8 )% 54,173 (24.0 )% (18,917 ) 15.7 % IP Transfer — — (3,947 ) 1.8 % — — Tax credits (1,559 ) (3.6 )% (4,035 ) 1.8 % 4,189 (3.5 )% IPO/Deconsolidation adjustment — — — — 37,911 (31.5 )% Tax rate change 2,178 5.1 % 3,906 (1.7 )% 19,046 (15.8 )% Nondeductible items and other (222 ) (0.7 )% 1,734 (0.9 )% (556 ) 0.5 % Inventory purchase from Ionis (2,833 ) (6.6 )% — — — — Intangible basis difference (2,596 ) (6.0 )% — — — — Foreign derived intangible income benefit (481 ) (1.1 )% — — — — Stock-based compensation 3,439 8.0 % 651 (0.3 )% — — Effective rate $ 2,192 5.1 % $ 447 (0.2 )% $ 1,275 (1.0 )% Deferred income taxes reflect the net tax effects of temporary differences between the carrying amounts of assets and liabilities for financial reporting purposes and the amounts used for income tax purposes. Significant components of our deferred tax assets and liabilities as of December 31, 2019 and 2018 are as follows (in thousands): December 31, 2019 2018 Deferred Tax Assets: Net operating loss carryovers $ 22,045 $ 51,280 Tax credits 27,801 31,768 Stock-based compensation 7,825 11,812 Deferred revenue — 6,876 Intangible and capital assets 99,038 56,984 Other 4,381 1,996 Total deferred tax assets $ 161,090 $ 160,716 Deferred Tax Liabilities: Fixed assets (798 ) (811 ) Other (2,512 ) — Total deferred tax liabilities $ (3,310 ) $ (811 ) Valuation allowance (157,780 ) (159,905 ) Net deferred tax assets and liabilities $ — $ — On December 22, 2017, the U.S. government enacted the Tax Act. The Tax Act created a new requirement on GILTI earned by foreign subsidiaries for tax years beginning on or after January 1, 2018. The GILTI provisions require foreign subsidiary earnings in excess of an allowable return on the foreign subsidiary’s assets to be included in our U.S. income tax return. Under U.S. GAAP, we are permitted to make an accounting policy election to either treat taxes due on future inclusions in U.S. taxable income related to GILTI as a current-period expense when incurred or to factor such amounts into our measurement of deferred taxes. We have made an accounting policy election to treat taxes due on GILTI inclusion as a current period expense. Prior to the completion of our IPO we filed our tax returns on a consolidated and combined basis with Ionis for federal and state income tax purposes, respectively. For financial statement purposes when we are required to file on a consolidated or combined basis, we calculate our income tax amounts, including net operating losses and tax credit carryforwards, using a separate return methodology which determines income taxes as if we were a separate taxpayer from Ionis. Effective July 19, 2017, the date of our IPO, we are no longer included in the consolidated federal income tax return with Ionis. We are still required to file most of our state tax returns on a consolidated or combined basis with Ionis. Therefore, for financial statement purposes we calculated our state income tax amounts using the separate return method. We have excluded from the deferred tax table above state net operating loss carryforwards (and the associated valuation allowance) that have been generated by Akcea on a separate company basis and utilized by Ionis in consolidated state tax return filings as the amounts represent hypothetical deferred tax assets which are not legally eligible to be utilized on tax returns by Akcea in future years. At December 31, 2019, we had federal and state tax net operating loss carry forwards on a separate basis of $99.5 million and $1.4 million, respectively. The federal net operating losses can be carried forward indefinitely. The state tax net operating loss carry forwards will begin to expire in 2031. We also have federal research and development tax credit carry forwards of $33.6 million that will begin to expire in 2034. Utilization of the net operating loss carry forwards and credits may be subject to an annual limitation due to the ownership change limitations provided by the Internal Revenue Code of 1986, as amended, and similar state provisions. The annual limitation may result in the expiration of net operating losses and credits before utilization. We record a valuation allowance to reduce the balance of our net deferred tax assets to the amount we believe is more-likely-than-not to be realized. Due to our historical financial statement losses, we have a full valuation allowance recorded against our net deferred tax assets. We regularly assess the future realization of our net deferred tax assets and will reduce the valuation allowance in any such period in which we determine that all, or a portion, of our deferred tax assets are more-likely-than-not to be realized. Our valuation allowance decreased by $2.1 million from December 31, 2018 to December 31, 2019. The decease relates primarily to the utilization of net operating loss carryforwards in 2019 offset by an increase related to certain costs, which are capitalized and amortized for tax purposes. We analyze our filing positions in all the U.S. federal, state and foreign jurisdictions where we are required to file income tax returns to determine if we have any uncertain tax positions on any income tax returns. We recognize the impact of an uncertain tax position on an income tax return at the largest amount that the relevant taxing authority is more-likely-than not to sustain upon audit. We do not recognize a tax benefit if the position has a less than 50 percent likelihood of being sustained upon examination. The following table summarizes our gross unrecognized tax benefits (in thousands): Years Ended December 31, 2019 2018 2017 Beginning balance of unrecognized tax benefits $ 5,606 $ 5,001 $ 5,012 Additions related to prior year tax positions 155 — — Additions related to the current year 601 691 1,723 Decreases related to prior year tax positions (549 ) (86 ) (1,734 ) Ending balance of unrecognized tax benefits $ 5,813 $ 5,606 $ 5,001 Due to our valuation allowance, there are no unrecognized tax benefits at December 31, 2019 that would impact our effective tax rate, if recognized. We do not foresee any material changes to our gross unrecognized tax benefits within the next twelve months. We recognize interest and/or penalties related to income tax matters in income tax expense. We did not recognize any accrued interest and penalties related to gross unrecognized tax benefits during the years ended December 31, 2019, 2018 or 2017. We are subject to taxation in the United States and various state and foreign jurisdictions. The tax years for 2015 through 2019 are subject to examination by the U.S. federal, state and foreign tax authorities. We do not provide for a U.S. income tax liability and foreign withholding taxes on undistributed foreign earnings of our foreign subsidiaries as we consider those earnings to be permanently reinvested. The amount of unrecognized deferred tax liabilities associated with these earnings is immaterial. |
Initial Public Offering
Initial Public Offering | 12 Months Ended |
Dec. 31, 2019 | |
Initial Public Offering [Abstract] | |
Initial Public Offering | 13 . Initial Public Offering On July 19, 2017, we completed our IPO. Total net proceeds were $182.3 million, including the following: • $132.3 million from the sale of 17,968,750 shares of our common stock in our IPO of which $25.0 million was invested by Ionis; and • $50.0 million from the purchase of 6,250,000 shares by Novartis in a concurrent private placement. In addition, both of the following occurred in connection with the completion of our IPO on July 19, 2017: • the conversion of all outstanding shares of Series A convertible preferred stock into 28,884,540 shares of our common stock; and • the conversion of $106.0 million of outstanding principal plus accrued interest from the line of credit into 13,438,339 shares of common stock. |
Employment Benefits
Employment Benefits | 12 Months Ended |
Dec. 31, 2019 | |
Compensation And Retirement Disclosure [Abstract] | |
Employment Benefits | 1 4 . We have an employee 401(k) salary deferral plan covering all employees. Employees may make contributions by withholding a percentage of their salary up to the IRS annual limit of $19,000 and $25,000 in 2019 for employees under 50 years old and employees 50 years old or over, respectively. We made approximately $2.1 million, $1.6 million and $0.3 million in matching contributions for the years ended December 31, 2019, 2018 and 2017, respectively. |
Basic and Diluted Net Loss Per
Basic and Diluted Net Loss Per Share | 12 Months Ended |
Dec. 31, 2019 | |
Earnings Per Share [Abstract] | |
Basic and Diluted Net Loss Per Share | 1 5 . Basic and Diluted Net Loss Per Share We issued 28,884,540 shares of Series A convertible preferred stock in December 2015. The Series A convertible preferred stock converted into common stock in conjunction with the IPO in July 2017. As a result, there were 66,541,629 shares of common stock issued and outstanding and there were no longer any outstanding shares of Series A convertible preferred stock. We determined that the Series A convertible preferred stock was in substance common stock during the period that it was outstanding because the Series A convertible preferred stock was the lowest form of subordinated equity outstanding during that period and this class of stock would have been required to absorb the losses of the Company. Accordingly, we are using the two-class method for computing EPS. In connection with the TTR License Agreement with Ionis, we made distributions to Ionis representing the consideration to be paid in cash provided to Ionis in excess of the carrying value of the related assets acquired. These distributions are treated as dividends to Ionis; therefore, we have applied the two-class method loss per share to reflect the allocation of these distributions to the participating Ionis common shares. The two-class method is an earnings allocation formula that determines loss per share for each class of common stock and participating security according to dividends declared (or accumulated) and participation rights in undistributed earnings. For the purposes of calculating loss per share under the two-class method, we have allocated the net loss between the Series A convertible preferred stock, common stock owned by Ionis and common stock owned by others. Basic income (loss) per share for each class of stock is computed by dividing total distributable losses applicable to Series A convertible preferred stock, common stock owned by Ionis and common stock owned by others, including the 6% cumulative dividend contractually due to Series A convertible preferred shareholders, by the weighted-average of preferred and common shares outstanding during the requisite period. The cumulative preferred stock dividend was not paid upon completion of the IPO because the IPO was not a liquidation event or a change in control. Prior to the IPO, the 6% cumulative Series A convertible preferred stock dividend was considered as required under the two-class method regardless of whether those dividends were actually distributed. The following table summarizes the distributable income (losses) for the years ended December 31, 2019, 2018 and 2017: Year Ended December 31, 2019 2018 2017 Net income (loss) $ 40,772 $ (225,821 ) $ (121,559 ) Preferred stock dividend — — (20,100 ) Distributions to Ionis (13,492 ) (7,792 ) — Distributable income (losses) $ 27,280 $ (233,613 ) $ (141,659 ) The following table summarizes the reconciliation of weighted-average shares outstanding used in the calculation of basic income (loss) per share for the years ended December 31, 2019, 2018 and 2017: Year Ended December 31, 2019 2018 2017 Determination of shares: Weighted-average preferred shares outstanding — — 15,748,009 Weighted-average common shares outstanding owned by Ionis 70,099,576 59,812,394 20,669,446 Weighted-average common shares outstanding owned by others 22,815,682 21,553,407 9,593,322 The following table summarizes the calculation of basic income (loss) per share for the years ended December 31, 2019, 2018 and 2017 (in thousands, except share and per share amounts): Year Ended December 31, 2019 2018 2017 Losses attributable to preferred shares $ — $ — $ (48,485 ) Less: Assumed dividend to preferred shares — — 20,100 Losses allocated to preferred shares — — (28,385 ) Weighted-average preferred shares outstanding — — 15,748,009 Basic loss per preferred share $ — $ — $ (1.80 ) Income (losses) allocated to Ionis $ 20,581 $ (171,730 ) $ (63,638 ) Plus: Distribution to Ionis 13,492 7,792 — Income (losses) available to Ionis 34,073 (163,938 ) (63,638 ) Weighted-average common shares outstanding owned by Ionis 70,099,576 59,812,394 20,669,446 Basic income (loss) per common share owned by Ionis $ 0.49 $ (2.74 ) $ (3.08 ) Income (losses) allocated to common shares owned by others $ 6,699 $ (61,883 ) $ (29,536 ) Weighted-average common shares outstanding owned by others 22,815,682 21,553,407 9,593,322 Basic income (loss) per common share owned by others $ 0.29 $ (2.87 ) $ (3.08 ) For the years ended December 31, 2018 and 2017, we incurred a net loss; therefore, we did not include dilutive common equivalent shares in the computation of diluted net loss per share because the effect would have been anti-dilutive. Common stock from the following would have had an anti-dilutive effect on net loss per share: • Options to purchase common stock; • Unvested restricted stock units; and • Employee Stock Purchase Plan. For the year ended December 31, 2019, we had net income. As a result, we computed diluted net income per share using the weighted-average number of common shares owned by Ionis, weighted-average number of common shares owned by others and dilutive common equivalent shares outstanding during the period. The following table summarizes the reconciliation of weighted-average shares outstanding and diluted common equivalent shares used in the calculation of diluted income per share for the year ended December 31, 2019: Year Ended December 31, 2019 Determination of shares: Weighted-average common shares outstanding owned by Ionis 70,099,576 Weighted-average common shares outstanding owned by others 22,815,682 Shares issuable upon exercise of stock options 2,413,624 Shares issuable upon restricted stock awards issuance 29,128 Shares issuable related to our ESPP 23,839 Weighted-average shares outstanding owned by others, plus assumed conversions 25,282,273 The following table summarizes the calculation of diluted income per share for the year ended December 31, 2019 (in thousands, except per share amounts): Year Ended December 31, 2019 Income allocated to Ionis $ 20,049 Plus: Distribution to Ionis 13,492 Income available to Ionis 33,541 Weighted-average common shares outstanding owned by Ionis 70,099,576 Diluted income per common share owned by Ionis $ 0.48 Income allocated to common shares owned by others, plus assumed conversions $ 7,231 Weighted-average common shares outstanding owned by others, plus assumed conversions 25,282,273 Diluted income per common share owned by others $ 0.29 |
Contractual Obligations and Com
Contractual Obligations and Commitments | 12 Months Ended |
Dec. 31, 2019 | |
Commitments And Contingencies Disclosure [Abstract] | |
Contractual Obligations and Commitments | 1 6 . Operating Lease Operating Lease On April 5, 2018, we entered into an operating lease agreement for 30,175 square feet of office space located in Boston, Massachusetts for our corporate headquarters. The lease commencement date was August 15, 2018 and we took occupancy in September 2018. We are leasing this space under a non-cancelable operating lease with an initial term of 123 months and an option to extend the lease for an additional five-year term. We did not include the extension option in our right-of-use asset and lease liability calculation as we do not consider it reasonably certain that we would exercise the option. Under the lease agreement, we received a three-month free rent period, which commenced on August 15, 2018, and a tenant improvement allowance up to $3.8 million. We provided the lessor with a letter of credit to secure our obligations under the lease in the initial amount of $2.4 million, to be reduced to $1.8 million on the third anniversary of the rent commencement date and to $1.2 million on the fifth anniversary of the rent commencement date if we meet certain conditions set forth in the lease at each such time. The letter of credit amount is included in deposits and other assets on the accompanying consolidated balance sheet . On November 12, 2018, we entered into an operating lease agreement with Ionis to sublease 4,723 square feet of office space located in Carlsbad, California. The commencement date was March 2018 and the term of the lease is 64 months with a four-month free rent period. There is no extension option for this lease. On May 8, 2019, we entered into an operating lease agreement for office space located in Dublin, Ireland. The lease commenced in May 2019 and the initial term of the lease is 18 months with an extension option. We have included a 12-month extension period in our right-of-use asset and lease liability calculation as we consider it reasonably certain that we will exercise the option to extend the lease for an additional 12 months. Operating lease expense for the years ended December 31, 2019, 2018 and 2017 was $2.1 million, $2.4 million and $0.7 million, respectively. We recognize rent expense on a straight-line basis over the lease term for the lease of our office spaces. Cash paid for amounts included in the measurement of lease liabilities for the year ended December 31, 2019 was $2.4 million and was included in net cash used in operating activities in our consolidated statement of cash flows. Other information related to our operating lease is as follows (dollar amounts in thousands): At December 31, 2019 Operating lease right-of-use assets $ 11,094 Operating lease liabilities (1) 15,551 Weighted average remaining lease term 8.7 years Weighted average discount rate 8 % (1) Current portion of $1.4 million included in other current liabilities and remaining $14.2 million included in long-term portion of lease liabilities in our consolidated balance sheet. Annual maturities of our operating lease liabilities as of December 31, 2019 are as follows (in thousands): Years Ended December 31, Operating Leases 2020 $ 2,501 2021 2,506 2022 2,403 2023 2,400 2024 2,395 Thereafter 9,565 Total minimum lease payments $ 21,770 Less: Imputed interest (6,219 ) Total operating lease liability $ 15,551 Purchase Commitments Purchase commitments include agreements to purchase goods or services that are enforceable and legally binding on us and that specify all significant terms, including, fixed or minimum quantities to be purchased; fixed, minimum or variable price provisions; and the approximate timing of the transaction. Such obligations are related principally to inventory purchase orders based on our current manufacturing needs and require significant lead times to be fulfilled by our vendors. Purchase commitments exclude agreements that are cancelable without penalty. As of December 31, 2019, our purchase commitments for the following 12 months were $4.3 million. |
Restructuring
Restructuring | 12 Months Ended |
Dec. 31, 2019 | |
Restructuring And Related Activities [Abstract] | |
Restructuring | 1 7 . Restructuring On September 6, 2018, we enacted a plan to reorganize our workforce to better align with the immediate needs of our business, or the Reorganization Plan, following the August 27, 2018 announcement of the FDA’s issuance of a Complete Response Letter for our New Drug Application for WAYLIVRA. In connection with the Reorganization Plan, we reduced our workforce by approximately 12%. The Reorganization Plan was approved by our board of directors on September 2, 2018 and affected employees were informed on September 6, 2018. The Reorganization Plan impacted U.S. employees primarily from the WAYLIVRA field team and functions focused principally on WAYLIVRA. For the year ended December 31, 2018, we recorded $1.7 million of restructuring-related costs in operating expense including employee severance, benefits and related costs, net of adjustments for employees who forfeited part of their benefits. In addition, we also recorded $0.4 million of non-cash stock option modifications expenses related to the Reorganization Plan for the year ended December 31, 2018. We did not incur any additional significant costs associated with this reorganization in 2019 and we have no remaining liability associated with the Reorganization Plan as of December 31, 2019. The following table summarizes the restructuring costs by category for the periods indicated (in thousands): Year Ended December 31, 2018 Cash Adjustment Non-Cash Total Research and development $ 327 $ (34 ) $ 209 $ 502 Selling, general and administrative 1,562 (116 ) 200 $ 1,646 Total $ 1,889 $ (150 ) $ 409 $ 2,148 The following table summarizes the restructuring reserve included in accrued compensation for the periods indicated (in thousands): Years Ended December 31, 2019 2018 Restructuring reserve beginning balance $ 43 $ — Restructuring expenses incurred during the period — 1,889 Adjustments during the period — (150 ) Amounts paid during the period (43 ) (1,696 ) Restructuring reserve ending balance $ — $ 43 |
Legal Proceedings
Legal Proceedings | 12 Months Ended |
Dec. 31, 2019 | |
Loss Contingency Information About Litigation Matters [Abstract] | |
Legal Proceedings | 18. Legal Proceedings From time to time, we may be involved in legal proceedings arising in the ordinary course of our business. Periodically, we evaluate the status of each legal matter and assess our potential financial exposure. If the potential loss from any legal proceeding is considered probable and the amount can be reasonably estimated, we accrue a liability for the estimated loss. Significant judgment is required to determine the probability of a loss and whether the amount of the loss is reasonably estimable. The outcome of any proceeding is not determinable in advance. As a result, the assessment of a potential liability and the amount of accruals recorded are based only on the information available to us at the time. As additional information becomes available, we reassess the potential liability related to the legal proceeding and may revise our estimates. On November 11, 2019, a purported Company stockholder filed an action in the Delaware Court of Chancery captioned City of Cambridge Retirement System v. Crooke, et al. L Rx |
Quarterly Financial Data (Unaud
Quarterly Financial Data (Unaudited) | 12 Months Ended |
Dec. 31, 2019 | |
Quarterly Financial Information Disclosure [Abstract] | |
Quarterly Financial Data (Unaudited) | 1 9 . The following financial information reflects all normal recurring adjustments, which are, in the opinion of management, necessary for a fair statement of the results of the interim periods. Summarized quarterly data for the years ended December 31, 2019 and 2018 are as follows (in thousands, except per share data): 2019 Quarters First Quarter Second Quarter Third Quarter Fourth Quarter Revenue: Product revenue, net $ 6,754 $ 9,865 $ 11,944 $ 13,690 Licensing revenue — 6,036 136 4,000 Research and development and license revenue under collaborative agreements 157,062 10,722 8,544 259,790 Total revenue 163,816 26,623 20,624 277,480 Expenses: Cost of sales - product 1,041 1,364 967 1,197 Cost of sales - intangible asset amortization 1,403 1,419 1,434 1,434 Cost of license — 3,000 — 2,400 Research and development 99,619 20,271 24,797 148,165 Selling, general and administrative 44,602 50,740 34,905 51,434 Net loss share from commercial activities under arrangement with Ionis Pharmaceuticals, Inc. (9,056 ) (11,465 ) (8,889 ) (10,313 ) Total expenses 137,609 65,329 53,214 194,317 Income (loss) from operations 26,207 (38,706 ) (32,590 ) 83,163 Other income (expense): Investment income 1,224 1,571 1,487 1,223 Other expense (112 ) (28 ) (136 ) (339 ) Income (loss) from operations 27,319 (37,163 ) (31,239 ) 84,047 Income tax expense (132 ) (160 ) (259 ) (1,641 ) Net income (loss) $ 27,187 $ (37,323 ) $ (31,498 ) $ 82,406 Net income (loss) per share of common stock owned by Ionis, basic $ 0.35 $ (0.40 ) $ (0.34 ) $ 0.87 Weighted-average shares of common stock outstanding owned by Ionis, basic 68,581,967 70,221,338 70,221,338 71,341,992 Net income (loss) per share of common stock owned by others, basic $ 0.15 $ (0.40 ) $ (0.34 ) $ 0.87 Weighted-average shares of common stock outstanding owned by others, basic 22,126,363 22,573,900 22,821,555 23,723,298 Net income (loss) per share of common stock owned by Ionis, diluted $ 0.34 $ (0.40 ) $ (0.34 ) $ 0.86 Weighted-average shares of common stock outstanding owned by Ionis, diluted 68,581,967 70,221,338 70,221,338 71,341,992 Net income (loss) per share of common stock owned by others, diluted $ 0.15 $ (0.40 ) $ (0.34 ) $ 0.86 Weighted-average shares of common stock outstanding owned by others, diluted 25,545,975 22,573,900 22,821,555 24,862,521 2018 Quarters First Quarter Second Quarter Third Quarter Fourth Quarter Revenue: Product revenue, net $ — $ — $ — $ 2,237 Licensing revenue — — 12,000 — Research and development and license revenue under collaborative agreements 17,108 18,321 7,241 7,960 Total revenue 17,108 18,321 19,241 10,197 Expenses: Cost of sales - product — — 1,043 777 Cost of sales - intangible asset amortization — — 701 2,012 Cost of license — — 7,200 — Research and development 27,970 39,457 29,381 33,532 Selling, general and administrative 19,465 42,287 45,924 45,934 Net loss share from commercial activities under arrangement with Ionis Pharmaceuticals, Inc. — — — — Total expenses 47,435 81,744 84,249 82,255 Loss from operations (30,327 ) (63,423 ) (65,008 ) (72,058 ) Other income (expense): Investment income 868 1,546 1,675 1,542 Other (expense) income (168 ) 45 (25 ) (41 ) Loss before income tax expense (29,627 ) (61,832 ) (63,358 ) (70,557 ) Income tax expense — (214 ) (233 ) — Net loss $ (29,627 ) $ (62,046 ) $ (63,591 ) $ (70,557 ) Net loss per share of common stock owned by Ionis, basic $ (0.44 ) $ (0.72 ) $ (0.73 ) $ (0.79 ) Weighted-average shares of common stock outstanding owned by Ionis, basic 45,447,879 60,832,494 65,538,467 67,129,553 Net loss per share of common stock owned by others, basic $ (0.44 ) $ (0.85 ) $ (0.73 ) $ (0.79 ) Weighted-average shares of common stock outstanding owned by others, basic 21,171,372 21,492,157 21,671,415 21,869,713 Net loss per share of common stock owned by Ionis, diluted $ (0.44 ) $ (0.72 ) $ (0.73 ) $ (0.79 ) Weighted-average shares of common stock outstanding owned by Ionis, diluted 45,447,879 60,832,494 65,538,467 67,129,553 Net loss per share of common stock owned by others, diluted $ (0.44 ) $ (0.85 ) $ (0.73 ) $ (0.79 ) Weighted-average shares of common stock outstanding owned by others, diluted 21,171,372 21,492,157 21,671,415 21,869,713 (1) We computed net loss per share independently for each of the quarters presented. Therefore, the sum of the quarterly net loss per share will not necessarily equal the total for the year. ( 2 ) We did not include dilutive common equivalent shares in the computation of diluted net loss per share because the effect would have been antidilutive. We included dilutive common equivalent shares in the computation of diluted net income per share. |
Summary of Significant Accoun_2
Summary of Significant Accounting Policies (Policies) | 12 Months Ended |
Dec. 31, 2019 | |
Accounting Policies [Abstract] | |
Use of Estimates | Use of Estimates The preparation of financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities, the disclosure of contingent assets and liabilities at the date of the financial statements and the reported amounts of revenue and expenses during the reporting periods. Significant estimates and assumptions reflected in these financial statements include, but are not limited to, revenue recognition, the accrual for research and development expenses, stock-based compensation and income taxes. Estimates are periodically reviewed in light of changes in facts, circumstances and experience. Changes in estimates are recorded in the period in which they become known. Actual results could differ from those estimates. |
Translation of Foreign Currency | Translation of Foreign Currency For our foreign subsidiaries that report in a functional currency other than U.S. dollars, we translate their assets and liabilities into U.S. dollars using the exchange rate at the balance sheet date. We translate revenue and expenses at the monthly average exchange rates for the period. We translate transactions in our capital accounts at the historic exchange rate in effect at the date of the transaction. We include foreign currency translation adjustments in accumulated other comprehensive income (loss) as a component of stockholders’ equity. |
Concentration of Credit Risk | Concentration of Credit Risk Financial instruments that potentially subject us to concentrations of credit risk consist primarily of cash, cash equivalents, short-term investments and accounts receivable. We place our cash, cash equivalents and short-term investments with reputable financial institutions. We primarily invest our excess cash in commercial paper and debt instruments of the U.S. Treasury, financial institutions, corporations and U.S. government agencies with strong credit ratings and an investment grade rating at or above A-1, P-1 or F-1 by Moody's, Standard & Poor's, or S&P, or Fitch, respectively. We have established guidelines relative to diversification and maturities that maintain safety and liquidity. We periodically review and modify these guidelines to maximize trends in yields and interest rates without compromising safety and liquidity. |
Cash Equivalents And Short Term Investments | Cash Equivalents and Short-Term Investments We consider all liquid investments with maturities of three months or less when we purchase them to be cash equivalents. Our short-term investments have initial maturities of greater than three months from the date of purchase. We classify our short-term investments as available-for-sale and we carry them at fair market value based upon prices for identical or similar items on the last day of the fiscal period. We record unrealized gains and losses as a separate component of comprehensive income (loss) and we include net realized gains and losses in investment income on our consolidated statement of operations. We use the specific identification method to determine the cost of securities sold. |
Accounts Receivable | Accounts Receivable Our accounts receivable balance is comprised of payments due from our partners in connection with our collaborative agreements and from our customers for product sales. We record receivables for product sales net of allowances for prompt payment discounts and other related fees and discounts based on contractual terms with our customers. We have standard payment terms that generally require payment within 30 to 90 days. We do not adjust our receivables for the effects of a significant financing component at contract inception if we expect to collect the receivables in one year or less from the time of sale. We provide reserves against trade receivables for estimated losses that may result from a customer's inability to pay. Amounts determined to be uncollectible are charged or written-off against the reserve. As of December 31, 2019 and 2018, we did not recognize any reserves for uncollectible accounts. |
Inventory | Inventory Prior to the regulatory approval of our product candidates, we incur expenses for the manufacturing of drug product that could potentially be available to support the commercial launch of our products. Until the first reporting period when regulatory approval has been received or is otherwise considered probable, we record all such costs as research and development expense. WAYLIVRA inventory-related costs incurred subsequent to April 1, 2019 and TEGSEDI inventory-related costs incurred subsequent to July 1, 2018 are reflected as inventory on our consolidated balance sheet at the lower of cost or net realizable value under the first-in, first-out, or FIFO, basis. We periodically analyze our inventory levels and write down inventory that has become obsolete, inventory that has a cost basis in excess of its estimated realizable value and inventory in excess of expected sales requirements as cost of product sales. The determination of whether inventory costs will be realizable requires estimates by our management and if actual market conditions are less favorable than projected by our management, additional write-downs of inventory may be required which would be recorded as a cost of product sales in the consolidated statement of operations. At December 31, 2018, a majority of our physical inventory for TEGSEDI was produced prior to when we obtained regulatory approval and accordingly had no cost basis as we recorded the related costs as research and development expense in prior periods. We obtained the first regulatory approval for TEGSEDI in July 2018 and for WAYLIVRA in May 2019. At December 31, 2019, our physical inventory for TEGSEDI and WAYLVRA included API that we produced prior to when we obtained regulatory approval. As such, this API has no cost basis as we had previously expensed these costs as R&D expenses. |
Property, Plant and Equipment | Property and Equipment Property and equipment are recorded at cost and depreciated using the straight-line method over the estimated useful life of each asset. Furniture and fixtures are depreciated over seven years. Computer equipment and software are depreciated over three years. Manufacturing equipment is depreciated over five years. Leasehold improvements are amortized over the shorter of the lease term or the ten-year estimated useful life of the asset. Upon retirement or sale, the cost of assets disposed of and the related accumulated depreciation are removed from the accounts and any resulting gain or loss is credited or charged to income. Repairs and maintenance costs are expensed as incurred. |
Leases | Leases Topic 842 Adoption In February 2016, the Financial Accounting Standards Board, or FASB, issued amended accounting guidance related to lease accounting. This guidance superseded the lease requirements we previously followed in ASC Topic 840, Leases Leases We adopted Topic 842 on January 1, 2019 and adjusted our opening consolidated balance sheet on that date to record our operating lease right-of-use assets and lease liabilities. We adopted Topic 842 using the available practical expedients permitted under the transition guidance within the new standard, which, among other things, allowed us to carry forward historical lease classification of those leases we had in place as of January 1, 2019. Results for the year ended December 31, 2019 are presented under Topic 842. Results for the year ended December 31, 2018 are presented in accordance with our historic accounting under Topic 840. The impact of the adoption of Topic 842 on the accompanying condensed consolidated balance sheet as of January 1, 2019 was as follows (in thousands): December 31, 2018 Adjustment due to adoption of Topic 842 January 1, 2019 Operating lease right-of-use assets — 11,932 11,932 Other current liabilities 968 1,029 1,997 Long-term portion of lease liabilities 4,442 10,915 15,357 We determine if an arrangement contains a lease at inception. We currently only have operating leases. We recognize an operating lease right-of-use asset and associated short and long-term lease liability for operating leases greater than one year on our consolidated balance sheet. We calculate our operating lease right-of-use asset and lease liability based on the present value of the future minimum lease payments we will pay over the lease term. We determine the lease term at the commencement date of the lease and we include renewal options in the lease term if we are reasonably certain that we will exercise the option. As our current leases do not provide an implicit interest rate, we used our incremental borrowing rate in determining the present value of future payments. We estimate the incremental borrowing rate based on the observed interest rates for secured debt issued by companies with similar credit ratings and with similar terms. Our operating lease right-of-use asset also includes any lease payments we made and excludes any tenant improvement allowances we received. We recognize rent expense for the lease components of our operating leases on a straight-line basis over the term of our lease. We recognize non-lease components, such as common area maintenance expenses, in the period we incur the expense. |
Intangible Assets | Intangible Assets We obtained exclusive licenses from Ionis for specific patents that Ionis owns and maintains related to our medicine pipeline. We recorded our licenses from Ionis as a capital contribution using the carryover basis of Ionis' historical cost for the related patents. We are amortizing our capitalized licenses over their estimated useful life, which is the term of the underlying individual patents owned by Ionis. In addition, we maintain definite-lived intangible assets related to regulatory milestone payments made to Ionis that are recoverable through future cash flows from approved products, which are capitalized as licensed intangible assets. These assets are amortized over their remaining useful lives, which are generally estimated to be the remaining patent life. If our estimate of the product’s useful life is shorter than the remaining patent life, then the shorter period is used. Intangible assets are amortized using the economic consumption method if anticipated future revenue can be reasonably estimated. The straight-line method is used when future revenue cannot be reasonably estimated. We use the straight-line method and amortization expense is recorded as a component of cost of sales to the extent the underlying license is related to a commercial product or research and development prior to product commercialization in the consolidated statement of operations. We assess our intangible assets for impairment if indicators are present or changes in circumstances suggest that impairment may exist. Events that could result in an impairment or trigger an interim impairment assessment may include actions by regulatory authorities with respect to us or our competitors, the receipt of additional clinical or nonclinical data regarding our medicine or a potentially competitive medicine, changes in the clinical development program for a medicine or new information regarding potential sales for the medicine. If impairment indicators are present or changes in circumstances suggest that impairment may exist, we perform a recoverability test by comparing the sum of the estimated undiscounted cash flows of each intangible asset to its carrying value on the consolidated balance sheet. If the undiscounted cash flows used in the recoverability test are less than the carrying value, we would determine the fair value of the intangible asset and recognize an impairment loss if the carrying value of the intangible asset exceeds its fair value. |
Fair Value of Financial Instruments | Fair Value of Financial Instruments We have estimated the fair value of our financial instruments. The amounts reported for cash equivalents, accounts payable and accrued expenses approximate fair value because of their short maturities. We report our investment securities at their estimated fair value based on a three-tier fair value hierarchy to prioritize the inputs used in our fair value measurements. These tiers include: Level 1, defined as observable inputs such as quoted prices in active markets for identical assets, which includes our money market funds and treasury securities classified as available-for-sale securities; Level 2, defined as inputs other than quoted prices in active markets that are either directly or indirectly observable, which includes our fixed income securities and commercial paper classified as available-for-sale securities; and Level 3, defined as unobservable inputs in which little or no market data exists, therefore requiring us to develop our own assumptions. We have not historically held any Level 3 investments. Our securities have been classified as Level 1 or Level 2. We obtain the fair value of our Level 2 investments from our custodian bank and from a professional pricing service. We validate the fair value of our Level 2 investments by understanding the pricing model used by the custodian banks or professional pricing service provider and comparing that fair value to the fair value based on observable market prices. We recognize transfers between levels of the fair value hierarchy on the date of the event or change in circumstances that caused the transfer . No transfers between levels have occurred to date |
Revenue Recognition | Revenue Recognition Collaboration and License Revenue Effective January 1, 2018, we adopted Accounting Standards Codification, or ASC, Topic 606, Revenue from Contracts with Customers Under Topic 606, an entity recognizes revenue when its customer obtains control of promised goods or services in an amount that reflects the consideration which the entity expects to receive in exchange for those goods or services. To determine revenue recognition for arrangements that an entity determines are within the scope of Topic 606, the entity performs the following five steps: (i) identify the contract(s) with a customer; (ii) identify the performance obligations in the contract; (iii) determine the transaction price; (iv) allocate the transaction price to the performance obligations in the contract; and (v) recognize revenue when (or as) the entity satisfies a performance obligation. At contract inception, once the contract is determined to be within the scope of Topic 606, we assess the goods or services promised within each contract and determine those that are performance obligations, then assess whether each promised good or service is distinct. When we offer options for additional goods or services, such as an option to license a medicine in the future or for additional goods or services to be provided in the future, we evaluate whether such options are material rights that should be treated as additional performance obligations. We typically have concluded that the option to license a medicine or the options for additional goods or services that may be requested in the future under our collaboration agreements are not material rights as the amounts attributable to such options represent standalone selling price, and therefore no consideration is allocated to these items at the inception of an agreement. When a partner exercises its option to license a medicine or requests the additional goods or services, a new performance obligation is created for that item. Once performance obligations are identified, we then recognize as revenue the amount of the transaction price that we allocated to the respective performance obligation when (or as) each performance obligation is satisfied, either at a point in time or over time. If the performance obligation is satisfied over time, we recognize revenue based on the use of an output or input method. As of December 31, 2019, we had four collaboration and license revenue agreements: our strategic collaboration, option and license agreement with Novartis Pharma AG, or Novartis, which we entered into in January 2017; our TTR development, commercialization, collaboration and license agreement with Ionis, which we entered into in April 2018 and pursuant to which we are recognizing commercial product revenue related to TEGSEDI sales subsequent to product launch in the fourth quarter of 2018; our collaboration and license agreement with PTC Therapeutics International Limited, or PTC Therapeutics, which we entered into in August 2018; and our license agreement with Pfizer Inc., or Pfizer, which we entered into in October 2019. For a complete discussion of the accounting related to our license and collaboration agreements, see Note 7, Strategic Collaboration with Novartis, Note 8 , License Agreements and Services Agreement with Ionis , Note 9, Collaboration and License Agreement with PTC Therapeutics and Note 10 , License Agreement with Pfizer . |
Product Revenue, Net | Product Revenue, Net Subsequent to obtaining regulatory approval from the U.S. Food and Drug Administration, or FDA, on October 5, 2018, we began to sell TEGSEDI in the U.S. in the fourth quarter of 2018. The product is distributed through an exclusive distribution agreement with a third-party logistics company, or 3PL, that takes title to the product and represents our sole customer in the U.S. Our U.S. customer distributes TEGSEDI to a specialty pharmacy and a specialty distributor (collectively referred to as “wholesalers”), who then distribute the product to health care providers and patients. On July 11, 2018, we obtained regulatory approval of TEGSEDI in Europe, following which we began to sell TEGSEDI in the E.U. in the fourth quarter of 2018. On May 7, 2019, we obtained regulatory approval of WAYLIVRA in the E.U., following which we began to sell WAYLIVRA in the E.U. in the third quarter of 2019. TEGSEDI and WAYLIVRA are distributed through 3PLs that distribute the product to hospitals and pharmacies in the E.U . Revenue from product sales is recognized when the customer obtains control of our product, which occurs upon transfer of title to the customer. Revenue is recognized at the amount that we expect to be entitled to in exchange for the sale of our product. This amount includes both fixed and variable consideration and excludes amounts that are collected from customers and remitted to governmental authorities. We record shipping and handling costs related to commercial products within cost of goods sold on our consolidated statement of operations. We classify payments to customers or other parties in the distribution channel for services that are distinct and priced at fair value as selling, general and administrative expenses on our consolidated statement of operation. Payments to customers or other parties in the distribution channel that do not meet those criteria are classified as a reduction of revenue, as discussed further below. We have elected not to adjust consideration for the effects of a significant financing component when the period between the transfer of a promised good or service to the customer and when the customer pays for that good or service will be one year or less. Our payment terms are generally between thirty to ninety days . Reserves for Variable Consideration Revenue from product sales is recorded at the net sales price (transaction price), which includes estimates of variable consideration for which reserves are established related to discounts, returns, chargebacks, rebates, co-pay assistance and other allowances that are offered within contracts between us and our customers, wholesalers, health care providers and other indirect customers relating to the sale of TEGSEDI and WAYLIVRA. These reserves are based on the amounts earned or to be claimed on the related sales and are classified as a reduction of accounts receivable or a current liability. Where appropriate, these estimates take into consideration a range of possible outcomes that are probability-weighted for relevant factors such as our historical experience, current contractual and statutory requirements, specific known market events and trends, industry data and forecasted customer buying and payment patterns. Overall, product revenue net of these reserves reflects our best estimate of the amount of consideration to which we are entitled based on the terms of the contract. The amount of variable consideration that is included in the transaction price may be constrained and is included in the net sales price only to the extent that it is considered probable that a significant reversal in the amount of the cumulative revenue recognized will not occur in a future period. Actual amounts of consideration ultimately received may differ from our estimates. If actual results in the future vary from our estimates, we will adjust these estimates, which would affect net product revenue and earnings, in the period such variances become known . The following are the components of variable consideration related to product revenue: Chargebacks: In the U.S., we estimate obligations resulting from contractual commitments with the government and other entities to sell products to qualified healthcare providers at prices lower than the list prices charged to our U.S. customer. Our U.S. customer charges us for the difference between what they pay for the product and the selling price to the qualified healthcare providers. We record reserves and reduce our product revenue for these chargebacks related to product sold to our U.S. customer during the reporting period as well as our estimate of product that remains in the distribution channel at the end of the reporting period that we expect will be sold to qualified healthcare providers in future periods . Our established reserve for chargebacks is included in accrued liabilities on our consolidated balance sheet . Government rebates: We are subject to discount obligations under government programs, including Medicaid and Medicare programs in the U.S., and similar programs in certain countries in Europe. We record reserves for government rebates based on statutory discount rates and estimated utilization in the period in which revenue is recognized. We estimate Medicaid and Medicare rebates based upon estimated payer mix. These reserves are recorded in the same period the related revenue is recognized, resulting in a reduction of product revenue and the establishment of a liability that is included in accrued expenses on our consolidated balance sheet. For Medicare, we also estimate the number of patients in the prescription drug coverage gap for whom we expect we will owe an additional liability under the Medicare Part D program. On a quarterly basis, we update our estimates and record any adjustments in the period that we identify the adjustments . Managed care rebates: We are subject to rebates in connection with a value-based agreement with a certain commercial payer. Rebate accruals are recorded in the same period the related revenue is recognized, resulting in a reduction of product revenue and the establishment of a liability which is included in accrued liabilities on our consolidated balance sheet. This rebate is based on a fixed percentage and includes a price increase limit allowance (price protection). Our estimate for managed care rebates is based upon estimated payer mix and the applicable contractual rebate rate. Trade discounts and allowances: We provide customary invoice discounts on sales to our U.S. customer for prompt payment. The discounts are recorded in the same period the related revenue is recognized, resulting in a reduction of product revenue, and the establishment of a reserve that is offset against our accounts receivable balance on our consolidated balance sheet. Distribution fees: We receive and pay for various distribution services provided by our U.S. and E.U. customers and our U.S. wholesalers. These fees are generally accounted for as a reduction of revenue in the same period the related revenue is recognized, and an establishment of a reserve that is offset against our accounts receivable balance on our consolidated balance sheet. To the extent that the services received are distinct from the sale of products to our customers, we classify these payments as selling, general and administrative expenses. Product Returns: Our U.S. customer has return rights and our wholesalers in the U.S. have limited return rights primarily related to the product’s expiration date. We estimate the amount of product sales that may be returned and record the estimate as a reduction of revenue and a refund liability included in accrued liabilities in our consolidated balance sheet in the period the related product revenue is recognized. Based on our distribution model, contractual inventory limits with our U.S. customer and wholesalers and the price of TEGSEDI, we believe there will be minimal returns in the U.S. Our E.U. customers only take title to TEGSEDI and WAYLIVRA when an order is received and therefore only maintain inventory levels of our products based on demand. Accordingly, there is limited return risk in the E.U. and we have not recorded any return estimate in the transaction price for products sold in the E.U . Other incentives: In the U.S., other incentives include co-payment assistance that we provide to patients with commercial insurance that have coverage and reside in states that allow co-payment assistance. The calculation of the accrual for co-payment assistance is based on an estimate of claims and the cost per claim that we expect to receive associated with product that has been recognized as revenue. Our estimate is recorded in the same period the related revenue is recognized, resulting in a reduction of product revenue and the establishment of a liability which is included in accrued liabilities on our consolidated balance sheet . During the year ended December 31, 2019, we recorded product revenue, net, of $42.3 million, which consisted of $34.6 million of TEGSEDI sales in the U.S., and $7.7 million of TEGSEDI and WAYLIVRA sales in the E.U. This is compared to the year ended December 31, 2018 product revenue, net, of $2.2 million which consisted of $1.2 million of TEGSEDI sales in the U.S., and $1.0 million of TEGSEDI sales in the E.U. The following table summarizes balances and activity in each of the product revenue allowance and reserve categories for the years ended December 31, 2019 and 2018 (in thousands): Chargebacks, discounts and fees Government and other rebates Returns Total Balance at December 31, 2017 $ — $ — $ — $ — Provision related to current period sales 50 293 5 348 Adjustment related to prior period sales — — — — Credit or payments made during the period — — — — Balance at December 31, 2018 50 293 5 348 Provision related to current period sales 1,240 2,604 192 4,036 Adjustment related to prior period sales (4 ) (28 ) — (32 ) Credits or payments made during the period (1,016 ) (1,516 ) — (2,532 ) Balance at December 31, 2019 $ 270 $ 1,353 $ 197 $ 1,820 |
Cost of Product Sales | Cost of Product Sales As a result of receiving marketing authorization, or MA, approval in the E.U. for WAYLIVRA in May 2019 and TEGSEDI in July 2018, we began recording all WAYLIVRA and TEGSEDI expenses related to commercial product as cost of product sales starting in April 2019 and July 2018, respectively. Cost of product sales consists of manufacturing costs, transportation and freight, and indirect overhead costs associated with the commercial manufacturing and distribution of WAYLIVRA and TEGSEDI. Cost of product sales may also include period costs related to certain commercial manufacturing services and inventory adjustment charges. Additionally, we previously expensed a significant portion of the cost of producing WAYLIVRA and TEGSEDI that we used in the commercial launch of these products as research and development expense prior to the regulatory approval of WAYLIVRA and TEGSEDI. |
Commercial Sublicensing Expenses | Commercial Sublicensing Expenses We incur sublicense expenses under our development, commercialization, collaboration and license agreement, or TTR License Agreement, and our development, commercialization and license agreement, or Cardiometabolic License Agreement, with Ionis related to the medicines we have licensed under these agreements. We include our sublicense fee expenses in cost of license expenses on our consolidated statement of operations for those medicines that are approved for marketing. We recognize sublicense fee expenses in the period they are incurred. |
Research and Development Expense | Research and Development Expenses Our research and development expenses include wages, benefits, facilities, supplies, external services, clinical study and manufacturing costs and other expenses that are directly related to our research and development activities. We expense research and development costs as we incur them. We do not conduct research activities and no such costs are included in these amounts. If we make payments for research and development services prior to the services being rendered, we record those amounts as prepaid assets on our balance sheet and we expense them as the services are provided. Research and Development Sublicensing Expenses We incur sublicense expenses under our Cardiometabolic Development, Commercialization and License Agreement with Ionis related to the medicines we have licensed under the agreement. We include our sublicense fee expenses in research and development expenses on our consolidated statement of operations since the applicable medicines are not yet approved for marketing. We recognize sublicense fee expenses in the period they are incurred. |
Estimated Liability for Research and Development Costs | Estimated Liability for Research and Development Costs We record accrued liabilities related to expenses for which vendors or service providers have not yet billed us. These liabilities are for products or services that we have received and primarily relate to ongoing nonclinical and clinical studies. These costs primarily include third-party clinical management costs, costs for contract research organizations, laboratory and analysis costs, toxicology studies and investigator grants. We have medicines in concurrent nonclinical and clinical studies at several sites throughout the world. To ensure that we have adequately provided for ongoing nonclinical and clinical research and development costs during the period in which we incur such costs, we maintain an accrual to cover these costs. We update our estimate for this accrual on at least a quarterly basis. The assessment of these costs is a subjective process that requires judgment. Upon settlement, these costs may differ materially from the amounts accrued in our consolidated financial statements. Our historical accrual estimates have not been materially different from our actual amounts. |
Stock-Based Compensation Expense | Stock-Based Compensation Expense We measure stock-based compensation expense for equity-classified awards, principally related to stock options, restricted stock units, or RSUs, and stock purchase rights under our employee stock purchase plan, or ESPP, based on the estimated fair value of the award on the date of grant. We recognize the value of the portion of the award that we ultimately expect to vest as stock-based compensation expense over the requisite vesting period in our consolidated statement of operations. We reduce stock-based compensation expense for estimated forfeitures at the time of grant and revise the expense in subsequent periods if actual forfeitures differ from those estimates. We value our stock option awards and stock purchase rights under our ESPP using the Black-Scholes model. The determination of the grant date fair value of options using an option pricing model is affected principally by our common stock fair value and requires us to make a number of other assumptions, including: the expected life of the option, the volatility of the underlying stock, the risk-free interest rate and expected dividends. The fair value of RSUs is based on the market price of our common stock on the date of grant. We have granted RSUs with various vesting terms between six months and four years. Prior to December 2015, Ionis granted our employees options to purchase shares of Ionis' common stock, or Ionis options. In December 2015, we granted our employees holding Ionis options additional options to purchase shares of our common stock, or Akcea options. We determined the stock-based compensation expense for the Ionis options at the date of grant and recognized compensation expense over the vesting period of the Ionis options. In December 2015, we accounted for the issuance of the Akcea options as a modification to the original grant of the Ionis options because the grant of the Ionis options and Akcea options essentially represented a single stock award as the exercisability provisions of the Ionis option grants and Akcea option grants were interrelated and mutually exclusive. The total compensation expense measured on the modification date was the sum of the grant date fair value of the Ionis options plus any incremental compensation cost resulting from the grant of the Akcea options. In 2016, we began concurrently granting Ionis options and Akcea options to our employees. Because the exercisability provisions of the awards are interrelated and mutually exclusive as described above, the fair values of the Ionis options and the Akcea options were determined on the date of grant and the option with the greater fair value was recognized over the vesting period of the awards. Following our IPO in 2017, we no longer concurrently grant Ionis and Akcea options. Our board of directors only receive grants under the Akcea option plan. Following our IPO, we no longer grant Ionis options to our employees. Under the terms of the Ionis options, when we completed our IPO, the Ionis options our employees were holding were terminated. The termination of the Ionis options was determined not to be a modification, as the options were terminated based upon the existing contractual terms of the option agreements. As such, we continue to recognize expense based on the valuation that was determined on the grant date for options issued in 2016 or on the modification date for options issued in 2015 and 2017. The fair value of stock options granted under our 2015 Equity Incentive Plan is based on the fair value of our common stock on the date of grant. The fair value of stock options granted under the Ionis 2011 Equity Incentive Plan is based on the fair value of Ionis' common stock on the date of grant. Options granted to employees vest over a four-year period, with 25 percent exercisable at the end of one year from the date of the grant and the balance vesting ratably, on a monthly basis, thereafter and have a term of ten years. Options granted to directors vest annually over a four-year period and have a term of ten years. See Note 11, Equity and Stock-based Compensation, |
Accumulated Other Comprehensive Income (Loss) | Accumulated Other Comprehensive Income (Loss) Accumulated other comprehensive income (loss) is comprised of unrealized gains and losses on investments, net of taxes and currency translation adjustments. The following table summarizes changes in accumulated other comprehensive income (loss) for the years ended December 31, 2019, 2018 and 2017 (in thousands): Years Ended December 31, 2019 2018 2017 Beginning balance accumulated other comprehensive loss $ (324 ) $ (451 ) $ (21 ) Unrealized gains (losses) on investments, net of tax (1) 237 144 (337 ) Currency translation adjustment 92 (17 ) (93 ) Net other comprehensive income (loss) 329 127 (430 ) Ending balance accumulated other comprehensive income (loss) $ 5 $ (324 ) $ (451 ) (1) There was no tax benefit for other comprehensive income (loss) for the years ended December 31, 2019, 2018 and 2017. |
Income Taxes | Income Taxes On December 22, 2017, the U.S. government enacted comprehensive tax legislation commonly referred to as the Tax Cut and Jobs Act of 2017, or the Tax Act. The Tax Act made broad and complex changes to the U.S. tax code, including, but not limited to, (i) reducing the U.S. federal statutory tax rate from 35% to 21%; (ii) requiring companies to pay a one-time transition tax on certain unrepatriated earnings of foreign subsidiaries; (iii) generally eliminating U.S federal income taxes on dividends from foreign subsidiaries; (iv) requiring a current inclusion in U.S. federal taxable income of certain earnings of controlled foreign corporations; (v) eliminating the corporate alternative minimum tax (AMT) and changing how existing AMT credits can be realized; (vi) subjecting certain foreign earnings to U.S. taxation through base erosion anti-abuse tax (BEAT) and global intangible low-taxed income (GILTI); (vii) changing rules related to uses and limitations of net operating loss carryforwards created in tax years beginning after December 31, 2017, and (vii) modifying the officer’s compensation limitation. Our accounting for the elements of the Tax Act is complete. We have made an accounting policy election to treat taxes due on the GILTI inclusion as a current period expense. Prior to the completion of our IPO we filed our tax returns on a consolidated and combined basis with Ionis for federal and state income tax purposes, respectively. For financial statement purposes, when we are required to file on a consolidated or combined basis, we calculate our income tax amounts, including net operating losses and tax credit carryforwards, using a separate return methodology which determines income taxes as if we were a separate taxpayer from Ionis. Effective July 19, 2017, the date of our IPO, we are no longer included in the consolidated federal income tax return with Ionis. We determined the amount of federal tax attributes, primarily net operating losses and tax credit carryforwards, that transferred to us upon deconsolidation from Ionis. We are still required to file most of our state tax returns on a consolidated or combined basis with Ionis. Therefore, for financial statement purposes we calculated our state income tax amounts using the separate return method. We account for income taxes using the asset and liability method, which requires the recognition of deferred tax assets and liabilities for the expected future tax consequences of events that have been recognized in our financial statements or tax returns. In addition, deferred tax assets are recorded for the future benefit of utilizing net operating losses and research and development credit carry forwards. Valuation allowances are provided when necessary to reduce deferred tax assets to the amount expected to be realized. We apply the authoritative accounting guidance prescribing a threshold and measurement attribute for the financial recognition and measurement of a tax position taken or expected to be taken in a tax return. We recognize liabilities for uncertain tax positions based on a two-step process. The first step is to evaluate the tax position for recognition by determining if the weight of available evidence indicates that it is more likely than not that the position will be sustained on audit, including resolution of related appeals or litigation settlement. The second step is to estimate and measure the tax benefit as the largest amount that is more than 50% likely to be realized upon ultimate settlement. We recognize interest and penalties related to unrecognized tax benefits within the income tax expense line in our consolidated statement of operations. Accrued interest and penalties are included within other long-term liabilities on the consolidated balance sheet. Significant judgment is required in evaluating our uncertain tax positions and determining our provision for income taxes. Although we believe our reserves are reasonable, no assurance can be given that the final tax outcome of these matters will not be different from that which is reflected in our historical income tax provisions and accruals. We adjust these reserves for changing facts and circumstances, such as the closing of a tax audit or the refinement of an estimate. To the extent that the final tax outcome of these matters is different than the amounts recorded, such differences may impact the provision for income taxes in the period in which such determination is made. Significant judgment is also required in determining any valuation allowance recorded against deferred tax assets. In assessing the need for a valuation allowance, we consider all available evidence, including scheduled reversal of deferred tax liabilities, past operating results, the feasibility of tax planning strategies and estimates of future taxable income. Estimates of future taxable income are based on assumptions that are consistent with our plans. Assumptions represent management's best estimates and involve inherent uncertainties and the application of management's judgment. Should actual amounts differ from our estimates, the amount of our tax expense and liabilities could be materially impacted. We do not provide for a U.S. income tax liability and foreign withholding taxes on undistributed foreign earnings of our foreign subsidiaries. The earnings of non-U.S. subsidiaries are currently expected to be indefinitely reinvested in non-U.S. operations. |
New Accounting Pronouncements - Recently Issued | New Accounting Pronouncements - Recently Issued In June 2016, the FASB issued guidance that changes the measurement of credit losses for most financial assets and certain other instruments. If we have credit losses, this updated guidance requires us to record allowances for these instruments under a new expected credit loss model. This model requires us to estimate the expected credit loss of an instrument over its lifetime, which represents the portion of the amortized cost basis we do not expect to collect. The new guidance requires us to remeasure our allowance in each reporting period we have credit losses. We adopted this guidance on January 1, 2020. This guidance is not anticipated to have a significant impact on our consolidated financial statements and disclosures . In August 2018, the FASB issued clarifying guidance on how to account for implementation costs related to cloud-servicing arrangements. The guidance states that if these fees qualify to be capitalized and amortized over the service period, they need to be expensed in the same line item as the service expense and recognized in the same balance sheet category. The update can be applied either retrospectively or prospectively to all implementation costs incurred after the date of adoption. We adopted this guidance on January 1, 2020 on a prospective basis. This guidance is not anticipated to have a significant impact on our consolidated financial statements and disclosures . In August 2018, the FASB updated its disclosure requirements related to Level 1, 2 and 3 fair value measurements. The update included deletion and modification of certain disclosure requirements and additional disclosure related to Level 3 measurements. We adopted this updated guidance on January 1, 2019 and it did not have a significant impact on our disclosures. In November 2018, the FASB issued clarifying guidance on the interaction between the collaboration accounting guidance and the new revenue recognition guidance we adopted on January 1, 2018 (Topic 606). Below is the clarifying guidance and how we will implement it (in italics): 1) When a participant is considered a customer in a collaborative arrangement, all of the associated accounting under Topic 606 should be applied. • We will apply all of the associated accounting under Topic 606 when we determine a participant in a collaborative arrangement is a customer. 2) Adds “unit of account” concept to collaboration accounting guidance to align with Topic 606. The “unit of account” concept is used to determine if revenue is recognized or if a contra expense is recognized from consideration received under a collaboration. • We will use the “unit of account” concept when we receive consideration under a collaboration agreement to determine when we recognize revenue or a contra expense. 3) The clarifying guidance precludes us from recognizing revenue under Topic 606 when we determine a transaction with a collaborative partner is not a customer and is not directly related to the sales to third parties. • When we conclude a collaboration partner is not a customer and is not directly related to the sales to third parties, we will not recognize revenue for the transaction. We adopted this guidance on January 1, 2020. . In December 2019, the FASB issued guidance to simplify the accounting for income taxes. The update includes removing several exceptions to existing guidance and includes several simplification updates, all of which do not apply to our current accounting for income taxes. We adopted this updated guidance in the fourth quarter of 2019. This guidance did not have a significant impact on our consolidated financial statements and disclosures. |
Summary of Significant Accoun_3
Summary of Significant Accounting Policies (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Accounting Policies [Abstract] | |
Summary of Impact of Adoption of Topic 842 | The impact of the adoption of Topic 842 on the accompanying condensed consolidated balance sheet as of January 1, 2019 was as follows (in thousands): December 31, 2018 Adjustment due to adoption of Topic 842 January 1, 2019 Operating lease right-of-use assets — 11,932 11,932 Other current liabilities 968 1,029 1,997 Long-term portion of lease liabilities 4,442 10,915 15,357 |
Summary of Balances and Activity in Each of Product Revenue Allowance and Reserve Categories | The following table summarizes balances and activity in each of the product revenue allowance and reserve categories for the years ended December 31, 2019 and 2018 (in thousands): Chargebacks, discounts and fees Government and other rebates Returns Total Balance at December 31, 2017 $ — $ — $ — $ — Provision related to current period sales 50 293 5 348 Adjustment related to prior period sales — — — — Credit or payments made during the period — — — — Balance at December 31, 2018 50 293 5 348 Provision related to current period sales 1,240 2,604 192 4,036 Adjustment related to prior period sales (4 ) (28 ) — (32 ) Credits or payments made during the period (1,016 ) (1,516 ) — (2,532 ) Balance at December 31, 2019 $ 270 $ 1,353 $ 197 $ 1,820 |
Changes in Accumulated Other Comprehensive Loss | Accumulated other comprehensive income (loss) is comprised of unrealized gains and losses on investments, net of taxes and currency translation adjustments. The following table summarizes changes in accumulated other comprehensive income (loss) for the years ended December 31, 2019, 2018 and 2017 (in thousands): Years Ended December 31, 2019 2018 2017 Beginning balance accumulated other comprehensive loss $ (324 ) $ (451 ) $ (21 ) Unrealized gains (losses) on investments, net of tax (1) 237 144 (337 ) Currency translation adjustment 92 (17 ) (93 ) Net other comprehensive income (loss) 329 127 (430 ) Ending balance accumulated other comprehensive income (loss) $ 5 $ (324 ) $ (451 ) (1) There was no tax benefit for other comprehensive income (loss) for the years ended December 31, 2019, 2018 and 2017. |
Investments and Fair Value Me_2
Investments and Fair Value Measurements (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Investments And Fair Value Measurements [Abstract] | |
Summary of Investments | The following is a summary of our investments at December 31, 2019 and 2018 (in thousands): Gross Unrealized Estimated December 31, 2019 Cost Gains Losses Fair Value Available-for-sale securities: Corporate debt securities $ 105,679 $ 40 $ (23 ) $ 105,696 Debt securities issued by U.S. government agencies 38,970 30 (6 ) 38,994 Total securities with a maturity of one year or less $ 144,649 $ 70 $ (29 ) $ 144,690 Corporate debt securities 2,033 5 — 2,038 Debt securities issued by U.S. government agencies 14,079 — (3 ) 14,076 Total securities with a maturity of one to two years 16,112 5 (3 ) 16,114 Total available-for-sale securities $ 160,761 $ 75 $ (32 ) $ 160,804 Gross Unrealized Estimated December 31, 2018 Cost Gains Losses Fair Value Available-for-sale securities: Corporate debt securities $ 81,770 $ — $ (151 ) $ 81,619 Debt securities issued by U.S. government agencies 85,578 — (42 ) 85,536 Total securities with a maturity of one year or less $ 167,348 $ — $ (193 ) $ 167,155 |
Assets Measured at Fair Value on a Recurring Basis | The following tables present the investments we held at December 31, 2019 and 2018 that are regularly measured and carried at fair value. The table segregates our investments by the level within the fair value hierarchy of the valuation techniques we utilized to determine the respective securities' fair value (in thousands): At December 31, 2019 Quoted Prices in Active Markets (Level 1) Significant Other Observable Inputs (Level 2) Money market funds (1) $ 285,510 $ 285,510 $ — Corporate debt securities (2) 107,735 — 107,735 Debt securities issued by U.S. government agencies (3) 53,069 — 53,069 Total $ 446,314 $ 285,510 $ 160,804 At December 31, 2018 Quoted Prices in Active Markets (Level 1) Significant Other Observable Inputs (Level 2) Money market funds (1) $ 82,343 $ 82,343 $ — Corporate debt securities (2) 81,619 — 81,619 Debt securities issued by U.S. government agencies (3) 85,536 — 85,536 Total $ 249,498 $ 82,343 $ 167,155 (1) Included in cash and cash equivalents on our consolidated balance sheet. (2) At December 31, 2019 and 2018, $4.0 million and $1.0 million, respectively, was included in cash and cash equivalents on our consolidated balance sheet, with the difference included in short-term investments on our consolidated balance sheet. ( 3 ) Included in short-term investments on our consolidated balance sheet. |
Property, Plant and Equipment (
Property, Plant and Equipment (Table) | 12 Months Ended |
Dec. 31, 2019 | |
Property Plant And Equipment [Abstract] | |
Summary of Property and Equipment at Cost and Related Accumulated Depreciation | The following table presents property and equipment, at cost, and related accumulated depreciation (in thousands): December 31, 2019 2018 Furniture and fixtures $ 1,611 $ 1,611 Computer equipment and software 289 102 Manufacturing equipment 416 — Leasehold improvements 3,955 4,213 Total property and equipment, at cost 6,271 5,926 Less accumulated depreciation and amortization (1,010 ) (230 ) Total property and equipment, net $ 5,261 $ 5,696 |
Inventory (Tables)
Inventory (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Inventory Disclosure [Abstract] | |
Schedule of Inventory | The following table presents inventory (in thousands): Years Ended December 31, 2019 2018 Raw materials $ 6,520 $ — Work in process 2,039 — Finished goods 258 85 Total inventories $ 8,817 $ 85 |
Intangible Assets (Tables)
Intangible Assets (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Goodwill And Intangible Assets Disclosure [Abstract] | |
Schedule of Intangible Assets | The following table presents intangible assets (in thousands): December 31, Estimated 2019 2018 useful life Acquired and in-licensed rights $ 2,262 $ 2,262 7 - 21 Years Capitalized regulatory approval milestones 90,000 90,000 16 Years Less accumulated amortization (9,211 ) (3,348 ) Total intangible assets, net $ 83,051 $ 88,914 |
Schedule of Estimated Future Amortization Expenses for Intangible Assets | Estimated future amortization expense for intangible assets as of December 31, 2019 is as follows (in thousands): Total 2020 $ 5,879 2021 5,861 2022 5,856 2023 5,843 2024 5,822 Thereafter 53,790 $ 83,051 |
License Agreements and Servic_2
License Agreements and Services Agreement with Ionis (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Related Party Transactions [Abstract] | |
Schedule Of Net Loss Share Related to Commercial Activities Under TTR Agreement | A summary of the loss share related to the commercial activities under the TTR Agreement is as follows (in thousands): Year Ended December 31, 2019 Net losses incurred by the collaboration related to the commercial activities under the TTR License Agreement $ (66,210 ) Ionis' share of commercial losses under the TTR License Agreement reflected in our consolidated statements of operations (39,723 ) Akcea's share of commercial losses under the TTR License Agreement reflected in our consolidated statements of operations (26,487 ) |
Schedule Of Development Expenses Related to TTR License Agreement | A summary of the development expenses related to the TTR License Agreement is as follows (in thousands): Year Ended December 31, 2019 Total development expense incurred by the collaboration related to development activities under the TTR License Agreement $ (66,663 ) Akcea's share of TTR development expense reflected in research and development expense in our consolidated statements of operations (29,444 ) |
Amounts Recorded Related to Transactions with Ionis Including Amounts Related to TTR License Agreement | The following table summarizes the amounts recorded related to transactions with Ionis including amounts related to the TTR License Agreement for the following periods (in thousands) (1): Years Ended December 31, 2019 2018 2017 Operating expenses: Services performed by Ionis $ 4,703 $ 15,404 $ 9,742 Active pharmaceutical ingredient manufactured by Ionis 5,515 5,229 6,012 Pre-commercial inventory manufactured by Ionis — 5,996 — Sublicensing expenses 205,400 7,200 48,394 Out-of-pocket expenses paid by Ionis 4,250 46,162 37,426 Royalty expenses 183 — — Less: commercial share of loss in connection with the TTR License Agreement (39,695 ) — — Plus: R&D share of loss in connection with the TTR License Agreement 3,651 — — Total operating expenses generated by transactions with Ionis 184,007 79,991 101,574 Plus: distributions to Ionis: 13,492 7,792 — Total charges generated by transactions with Ionis 197,499 87,783 101,574 Payable balance to Ionis at the beginning of the period 18,901 14,365 24,355 Less: total amounts received from (paid to) Ionis during the period (19,631 ) (83,247 ) (78,170 ) Less: non-cash sublicensing expenses (200,000 ) — (33,394 ) Total amount (receivable) payable (from) to Ionis at period end $ (3,231 ) $ 18,901 $ 14,365 (1) This table excludes amounts capitalized as license intangible assets on the balance sheet. |
Equity and Stock-Based Compen_2
Equity and Stock-Based Compensation (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Restricted Stock Unit Activity | The following table summarizes restricted stock unit, or RSU, activity for year ended December 31, 2019 (in thousands, except per share data): Number of Shares Weighted Average Grant Date Fair Value per Share Non-vested at December 31, 2018 38 $ 25.65 Granted 706 $ 21.95 Vested (9 ) $ 25.76 Cancelled/forfeited/expired (34 ) $ 25.01 Non-vested at December 31, 2019 701 $ 21.95 |
Breakdown of Stock-Based Compensation Expense | The following table summarizes the breakdown of stock-based compensation expense by line item within the statement of operations for the years ended December 31, 2019, 2018 and 2017 (in thousands): Years Ended December 31, 2019 2018 2017 Cost of sales - product $ 437 $ 160 $ — Research and development expenses 11,895 9,435 8,630 Selling, general and administrative expenses 24,780 34,687 8,909 Total $ 37,112 $ 44,282 $ 17,539 |
2015 Equity Incentive Plan [Member] | |
Stock Options Activity | The following table summarizes the stock option activity for the year ended December 31, 2019 (in thousands, except per share and contractual life data) for the 2015 EIP: Number of Shares Weighted Average Exercise Price per Share Average Remaining Contractual Term (Years) Aggregate Intrinsic Value Outstanding at December 31, 2018 11,011 $ 15.00 Granted 4,756 $ 27.04 Exercised (1) (3,541 ) $ 7.99 Cancelled/forfeited/expired (3,360 ) $ 24.13 Outstanding at December 31, 2019 8,866 $ 20.81 7.97 $ 16,627 Exercisable at December 31, 2019 3,095 $ 14.68 6.43 $ 14,183 |
2015 Equity Incentive Plan and Employee Stock Purchase Plan [Member] | |
Weighted-Average Assumptions for Stock Options | For the years ended December 31, 2019, 2018 and 2017, we used the following weighted-average assumptions in our Black-Scholes calculations for stock option grants under our 2015 EIP and ESPP: Employee Stock Options: Years Ended December 31, 2019 2018 2017 Risk-free interest rate 2.3 % 2.8 % 1.9 % Dividend yield 0.0 % 0.0 % 0.0 % Volatility 75.5 % 77.1 % 79.5 % Expected life 6.08 years 6.08 years 6.06 years Board of Director Stock Options: Years Ended December 31, 2019 2018 2017 Risk-free interest rate 1.8 % 2.9 % 1.9 % Dividend yield 0.0 % 0.0 % 0.0 % Volatility 73.8 % 78.2 % 79.4 % Expected life 6.25 years 6.42 years 6.25 years ESPP: Years Ended December 31, 2019 2018 2017 Risk-free interest rate 2.4 % 1.9 % 1.1 % Dividend yield 0.0 % 0.0 % 0.0 % Volatility 60.0 % 64.2 % 73.3 % Expected life 6 months 6 months 6 months |
Income Taxes (Tables)
Income Taxes (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Income Tax Disclosure [Abstract] | |
Income (Loss) Before Income Taxes | Income (loss) before income taxes is comprised of (in thousands): Years Ended December 31, 2019 2018 2017 United States $ 40,475 $ (218,794 ) $ (108,691 ) Foreign 2,489 (6,580 ) (11,593 ) Income (loss) before income tax expense $ 42,964 $ (225,374 ) $ (120,284 ) |
Provision for Income Taxes | The provision (benefit) for income taxes is comprised of (in thousands): Years Ended December 31, 2019 2018 2017 Current: Federal $ 1,692 $ — $ — State 87 73 1,041 Foreign 413 374 234 Total current 2,192 447 1,275 Income tax expense $ 2,192 $ 447 $ 1,275 |
Reconciliation of Statutory to Effective Tax Rate | Our expense (benefit) for income taxes differs from the amount computed by applying the U.S. federal statutory rate to income (loss) before taxes. The sources and tax effects of the differences are as follows (in thousands) : Years Ended December 31, 2019 2018 2017 Pretax income (loss) $ 42,964 $ (225,374 ) $ (120,284 ) Statutory rate 9,023 21.0 % (47,329 ) 21.0 % (42,099 ) 35.0 % State income tax net of federal benefit (3,025 ) (7.0 )% (6,441 ) 2.9 % (2,371 ) 2.0 % Impact of foreign tax rate differential 340 0.8 % 1,735 (0.8 )% 4,072 (3.4 )% Net change in valuation allowance (2,072 ) (4.8 )% 54,173 (24.0 )% (18,917 ) 15.7 % IP Transfer — — (3,947 ) 1.8 % — — Tax credits (1,559 ) (3.6 )% (4,035 ) 1.8 % 4,189 (3.5 )% IPO/Deconsolidation adjustment — — — — 37,911 (31.5 )% Tax rate change 2,178 5.1 % 3,906 (1.7 )% 19,046 (15.8 )% Nondeductible items and other (222 ) (0.7 )% 1,734 (0.9 )% (556 ) 0.5 % Inventory purchase from Ionis (2,833 ) (6.6 )% — — — — Intangible basis difference (2,596 ) (6.0 )% — — — — Foreign derived intangible income benefit (481 ) (1.1 )% — — — — Stock-based compensation 3,439 8.0 % 651 (0.3 )% — — Effective rate $ 2,192 5.1 % $ 447 (0.2 )% $ 1,275 (1.0 )% |
Deferred Tax Assets and Liabilities | Significant components of our deferred tax assets and liabilities as of December 31, 2019 and 2018 are as follows (in thousands): December 31, 2019 2018 Deferred Tax Assets: Net operating loss carryovers $ 22,045 $ 51,280 Tax credits 27,801 31,768 Stock-based compensation 7,825 11,812 Deferred revenue — 6,876 Intangible and capital assets 99,038 56,984 Other 4,381 1,996 Total deferred tax assets $ 161,090 $ 160,716 Deferred Tax Liabilities: Fixed assets (798 ) (811 ) Other (2,512 ) — Total deferred tax liabilities $ (3,310 ) $ (811 ) Valuation allowance (157,780 ) (159,905 ) Net deferred tax assets and liabilities $ — $ — |
Gross Unrecognized Tax Benefits | The following table summarizes our gross unrecognized tax benefits (in thousands): Years Ended December 31, 2019 2018 2017 Beginning balance of unrecognized tax benefits $ 5,606 $ 5,001 $ 5,012 Additions related to prior year tax positions 155 — — Additions related to the current year 601 691 1,723 Decreases related to prior year tax positions (549 ) (86 ) (1,734 ) Ending balance of unrecognized tax benefits $ 5,813 $ 5,606 $ 5,001 |
Basic and Diluted Net Loss Pe_2
Basic and Diluted Net Loss Per Share (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Earnings Per Share [Abstract] | |
Basic Loss Per Share | The following table summarizes the distributable income (losses) for the years ended December 31, 2019, 2018 and 2017: Year Ended December 31, 2019 2018 2017 Net income (loss) $ 40,772 $ (225,821 ) $ (121,559 ) Preferred stock dividend — — (20,100 ) Distributions to Ionis (13,492 ) (7,792 ) — Distributable income (losses) $ 27,280 $ (233,613 ) $ (141,659 ) The following table summarizes the reconciliation of weighted-average shares outstanding used in the calculation of basic income (loss) per share for the years ended December 31, 2019, 2018 and 2017: Year Ended December 31, 2019 2018 2017 Determination of shares: Weighted-average preferred shares outstanding — — 15,748,009 Weighted-average common shares outstanding owned by Ionis 70,099,576 59,812,394 20,669,446 Weighted-average common shares outstanding owned by others 22,815,682 21,553,407 9,593,322 The following table summarizes the calculation of basic income (loss) per share for the years ended December 31, 2019, 2018 and 2017 (in thousands, except share and per share amounts): Year Ended December 31, 2019 2018 2017 Losses attributable to preferred shares $ — $ — $ (48,485 ) Less: Assumed dividend to preferred shares — — 20,100 Losses allocated to preferred shares — — (28,385 ) Weighted-average preferred shares outstanding — — 15,748,009 Basic loss per preferred share $ — $ — $ (1.80 ) Income (losses) allocated to Ionis $ 20,581 $ (171,730 ) $ (63,638 ) Plus: Distribution to Ionis 13,492 7,792 — Income (losses) available to Ionis 34,073 (163,938 ) (63,638 ) Weighted-average common shares outstanding owned by Ionis 70,099,576 59,812,394 20,669,446 Basic income (loss) per common share owned by Ionis $ 0.49 $ (2.74 ) $ (3.08 ) Income (losses) allocated to common shares owned by others $ 6,699 $ (61,883 ) $ (29,536 ) Weighted-average common shares outstanding owned by others 22,815,682 21,553,407 9,593,322 Basic income (loss) per common share owned by others $ 0.29 $ (2.87 ) $ (3.08 ) |
Diluted Loss Per Share | The following table summarizes the reconciliation of weighted-average shares outstanding and diluted common equivalent shares used in the calculation of diluted income per share for the year ended December 31, 2019: Year Ended December 31, 2019 Determination of shares: Weighted-average common shares outstanding owned by Ionis 70,099,576 Weighted-average common shares outstanding owned by others 22,815,682 Shares issuable upon exercise of stock options 2,413,624 Shares issuable upon restricted stock awards issuance 29,128 Shares issuable related to our ESPP 23,839 Weighted-average shares outstanding owned by others, plus assumed conversions 25,282,273 The following table summarizes the calculation of diluted income per share for the year ended December 31, 2019 (in thousands, except per share amounts): Year Ended December 31, 2019 Income allocated to Ionis $ 20,049 Plus: Distribution to Ionis 13,492 Income available to Ionis 33,541 Weighted-average common shares outstanding owned by Ionis 70,099,576 Diluted income per common share owned by Ionis $ 0.48 Income allocated to common shares owned by others, plus assumed conversions $ 7,231 Weighted-average common shares outstanding owned by others, plus assumed conversions 25,282,273 Diluted income per common share owned by others $ 0.29 |
Contractual Obligations and C_2
Contractual Obligations and Commitments (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Commitments And Contingencies Disclosure [Abstract] | |
Lessee, Operating Lease, Disclosure | Other information related to our operating lease is as follows (dollar amounts in thousands): At December 31, 2019 Operating lease right-of-use assets $ 11,094 Operating lease liabilities (1) 15,551 Weighted average remaining lease term 8.7 years Weighted average discount rate 8 % (1) Current portion of $1.4 million included in other current liabilities and remaining $14.2 million included in long-term portion of lease liabilities in our consolidated balance sheet. |
Lessee, Operating Lease, Liability, Maturity | Annual maturities of our operating lease liabilities as of December 31, 2019 are as follows (in thousands): Years Ended December 31, Operating Leases 2020 $ 2,501 2021 2,506 2022 2,403 2023 2,400 2024 2,395 Thereafter 9,565 Total minimum lease payments $ 21,770 Less: Imputed interest (6,219 ) Total operating lease liability $ 15,551 |
Restructuring (Tables)
Restructuring (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Restructuring And Related Activities [Abstract] | |
Summary of Restructuring Costs By Category | The following table summarizes the restructuring costs by category for the periods indicated (in thousands): Year Ended December 31, 2018 Cash Adjustment Non-Cash Total Research and development $ 327 $ (34 ) $ 209 $ 502 Selling, general and administrative 1,562 (116 ) 200 $ 1,646 Total $ 1,889 $ (150 ) $ 409 $ 2,148 |
Summary of Restructuring Reserve Included in Accrued Compensation | The following table summarizes the restructuring reserve included in accrued compensation for the periods indicated (in thousands): Years Ended December 31, 2019 2018 Restructuring reserve beginning balance $ 43 $ — Restructuring expenses incurred during the period — 1,889 Adjustments during the period — (150 ) Amounts paid during the period (43 ) (1,696 ) Restructuring reserve ending balance $ — $ 43 |
Quarterly Financial Data (Una_2
Quarterly Financial Data (Unaudited) (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Quarterly Financial Information Disclosure [Abstract] | |
Quarterly Financial Data | The following financial information reflects all normal recurring adjustments, which are, in the opinion of management, necessary for a fair statement of the results of the interim periods. Summarized quarterly data for the years ended December 31, 2019 and 2018 are as follows (in thousands, except per share data): 2019 Quarters First Quarter Second Quarter Third Quarter Fourth Quarter Revenue: Product revenue, net $ 6,754 $ 9,865 $ 11,944 $ 13,690 Licensing revenue — 6,036 136 4,000 Research and development and license revenue under collaborative agreements 157,062 10,722 8,544 259,790 Total revenue 163,816 26,623 20,624 277,480 Expenses: Cost of sales - product 1,041 1,364 967 1,197 Cost of sales - intangible asset amortization 1,403 1,419 1,434 1,434 Cost of license — 3,000 — 2,400 Research and development 99,619 20,271 24,797 148,165 Selling, general and administrative 44,602 50,740 34,905 51,434 Net loss share from commercial activities under arrangement with Ionis Pharmaceuticals, Inc. (9,056 ) (11,465 ) (8,889 ) (10,313 ) Total expenses 137,609 65,329 53,214 194,317 Income (loss) from operations 26,207 (38,706 ) (32,590 ) 83,163 Other income (expense): Investment income 1,224 1,571 1,487 1,223 Other expense (112 ) (28 ) (136 ) (339 ) Income (loss) from operations 27,319 (37,163 ) (31,239 ) 84,047 Income tax expense (132 ) (160 ) (259 ) (1,641 ) Net income (loss) $ 27,187 $ (37,323 ) $ (31,498 ) $ 82,406 Net income (loss) per share of common stock owned by Ionis, basic $ 0.35 $ (0.40 ) $ (0.34 ) $ 0.87 Weighted-average shares of common stock outstanding owned by Ionis, basic 68,581,967 70,221,338 70,221,338 71,341,992 Net income (loss) per share of common stock owned by others, basic $ 0.15 $ (0.40 ) $ (0.34 ) $ 0.87 Weighted-average shares of common stock outstanding owned by others, basic 22,126,363 22,573,900 22,821,555 23,723,298 Net income (loss) per share of common stock owned by Ionis, diluted $ 0.34 $ (0.40 ) $ (0.34 ) $ 0.86 Weighted-average shares of common stock outstanding owned by Ionis, diluted 68,581,967 70,221,338 70,221,338 71,341,992 Net income (loss) per share of common stock owned by others, diluted $ 0.15 $ (0.40 ) $ (0.34 ) $ 0.86 Weighted-average shares of common stock outstanding owned by others, diluted 25,545,975 22,573,900 22,821,555 24,862,521 2018 Quarters First Quarter Second Quarter Third Quarter Fourth Quarter Revenue: Product revenue, net $ — $ — $ — $ 2,237 Licensing revenue — — 12,000 — Research and development and license revenue under collaborative agreements 17,108 18,321 7,241 7,960 Total revenue 17,108 18,321 19,241 10,197 Expenses: Cost of sales - product — — 1,043 777 Cost of sales - intangible asset amortization — — 701 2,012 Cost of license — — 7,200 — Research and development 27,970 39,457 29,381 33,532 Selling, general and administrative 19,465 42,287 45,924 45,934 Net loss share from commercial activities under arrangement with Ionis Pharmaceuticals, Inc. — — — — Total expenses 47,435 81,744 84,249 82,255 Loss from operations (30,327 ) (63,423 ) (65,008 ) (72,058 ) Other income (expense): Investment income 868 1,546 1,675 1,542 Other (expense) income (168 ) 45 (25 ) (41 ) Loss before income tax expense (29,627 ) (61,832 ) (63,358 ) (70,557 ) Income tax expense — (214 ) (233 ) — Net loss $ (29,627 ) $ (62,046 ) $ (63,591 ) $ (70,557 ) Net loss per share of common stock owned by Ionis, basic $ (0.44 ) $ (0.72 ) $ (0.73 ) $ (0.79 ) Weighted-average shares of common stock outstanding owned by Ionis, basic 45,447,879 60,832,494 65,538,467 67,129,553 Net loss per share of common stock owned by others, basic $ (0.44 ) $ (0.85 ) $ (0.73 ) $ (0.79 ) Weighted-average shares of common stock outstanding owned by others, basic 21,171,372 21,492,157 21,671,415 21,869,713 Net loss per share of common stock owned by Ionis, diluted $ (0.44 ) $ (0.72 ) $ (0.73 ) $ (0.79 ) Weighted-average shares of common stock outstanding owned by Ionis, diluted 45,447,879 60,832,494 65,538,467 67,129,553 Net loss per share of common stock owned by others, diluted $ (0.44 ) $ (0.85 ) $ (0.73 ) $ (0.79 ) Weighted-average shares of common stock outstanding owned by others, diluted 21,171,372 21,492,157 21,671,415 21,869,713 (1) We computed net loss per share independently for each of the quarters presented. Therefore, the sum of the quarterly net loss per share will not necessarily equal the total for the year. ( 2 ) We did not include dilutive common equivalent shares in the computation of diluted net loss per share because the effect would have been antidilutive. We included dilutive common equivalent shares in the computation of diluted net income per share. |
Organization and Basis of Pre_2
Organization and Basis of Presentation (Details) - USD ($) $ in Thousands | 3 Months Ended | 12 Months Ended | |||||||||
Dec. 31, 2019 | Sep. 30, 2019 | Jun. 30, 2019 | Mar. 31, 2019 | Dec. 31, 2018 | Sep. 30, 2018 | Jun. 30, 2018 | Mar. 31, 2018 | Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Basis of Presentation and Organization [Abstract] | |||||||||||
Accumulated deficit | $ 481,270 | $ 522,042 | $ 481,270 | $ 522,042 | |||||||
Net income (loss) | 82,406 | $ (31,498) | $ (37,323) | $ 27,187 | $ (70,557) | $ (63,591) | $ (62,046) | $ (29,627) | 40,772 | (225,821) | $ (121,559) |
Net cash used from operating activities | 223,825 | $ (202,888) | $ (36,190) | ||||||||
Cash, cash equivalents and short-term investments | $ 463,700 | $ 463,700 | |||||||||
Ionis [Member] | Akcea [Member] | |||||||||||
Basis of Presentation and Organization [Abstract] | |||||||||||
Ownership percentage by Ionis | 76.00% | 76.00% |
Summary of Significant Accoun_4
Summary of Significant Accounting Policies - Account Receivable (Details) - USD ($) | 12 Months Ended | |
Dec. 31, 2019 | Dec. 31, 2018 | |
Summary Of Significant Accounting Policies [Line Items] | ||
Uncollectible accounts reserves | $ 0 | $ 0 |
Maximum [Member] | ||
Summary Of Significant Accounting Policies [Line Items] | ||
Period of time after billing when payment is received | 1 year | |
Payment terms | 90 days | |
Minimum [Member] | ||
Summary Of Significant Accounting Policies [Line Items] | ||
Payment terms | 30 days |
Summary of Significant Accoun_5
Summary of Significant Accounting Policies, Property, Plant and Equipment (Details) | 12 Months Ended |
Dec. 31, 2019 | |
Furniture And Fixtures [Member] | |
Property, Plant and Equipment [Abstract] | |
Estimated useful lives | 7 years |
Leasehold Improvements [Member] | |
Property, Plant and Equipment [Abstract] | |
Estimated useful lives | 10 years |
Estimated useful lives, description | over the shorter of the lease term or the ten-year |
Computer Equipment and Software [Member] | |
Property, Plant and Equipment [Abstract] | |
Estimated useful lives | 3 years |
Manufacturing Equipment [Member] | |
Property, Plant and Equipment [Abstract] | |
Estimated useful lives | 5 years |
Summary of Significant Accoun_6
Summary of Significant Accounting Policies - Summary of Impact of Adoption of Topic 842 (Details) - USD ($) $ in Thousands | Dec. 31, 2019 | Jan. 01, 2019 | Dec. 31, 2018 |
New Accounting Pronouncements Or Change In Accounting Principle [Line Items] | |||
Operating lease right-of-use assets | $ 11,094 | ||
Other current liabilities | 2,633 | $ 968 | |
Long-term portion of lease liabilities | $ 14,248 | $ 4,442 | |
ASU 2016-02 [Member] | |||
New Accounting Pronouncements Or Change In Accounting Principle [Line Items] | |||
Operating lease right-of-use assets | $ 11,932 | ||
Other current liabilities | 1,997 | ||
Long-term portion of lease liabilities | 15,357 | ||
Restatement Adjustment [Member] | ASU 2016-02 [Member] | |||
New Accounting Pronouncements Or Change In Accounting Principle [Line Items] | |||
Operating lease right-of-use assets | 11,932 | ||
Other current liabilities | 1,029 | ||
Long-term portion of lease liabilities | $ 10,915 |
Summary of Significant Accoun_7
Summary of Significant Accounting Policies - Fair Value of Financial Instruments (Details) | Dec. 31, 2019USD ($) |
Fair Value Measurements [Abstract] | |
Transfers from Level 1 to Level 2 | $ 0 |
Transfers from Level 2 to Level 1 | $ 0 |
Summary of Significant Accoun_8
Summary of Significant Accounting Policies - Narratives (Details) | Dec. 31, 2019RevenueStream |
Summary of Significant Accounting Policies [Abstract] | |
Number of revenue agents | 4 |
Summary of Significant Accoun_9
Summary of Significant Accounting Policies - Product Revenue, Net (Details) - USD ($) $ in Thousands | 3 Months Ended | 12 Months Ended | |||||||||
Dec. 31, 2019 | Sep. 30, 2019 | Jun. 30, 2019 | Mar. 31, 2019 | Dec. 31, 2018 | Sep. 30, 2018 | Jun. 30, 2018 | Mar. 31, 2018 | Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Summary Of Significant Accounting Policies [Line Items] | |||||||||||
Payments for good or service | true | ||||||||||
Amortization period | true | ||||||||||
Disaggregation of Revenue [Abstract] | |||||||||||
Revenue, net | $ 277,480 | $ 20,624 | $ 26,623 | $ 163,816 | $ 10,197 | $ 19,241 | $ 18,321 | $ 17,108 | $ 488,543 | $ 64,867 | $ 43,401 |
TEGSEDI [Member] | |||||||||||
Disaggregation of Revenue [Abstract] | |||||||||||
Revenue, net | 2,200 | ||||||||||
TEGSEDI [Member] | U.S. [Member] | |||||||||||
Disaggregation of Revenue [Abstract] | |||||||||||
Revenue, net | 34,600 | 1,200 | |||||||||
TEGSEDI [Member] | E.U. [Member] | |||||||||||
Disaggregation of Revenue [Abstract] | |||||||||||
Revenue, net | $ 1,000 | ||||||||||
TEGSEDI and WAYLIVRA [Member] | |||||||||||
Disaggregation of Revenue [Abstract] | |||||||||||
Revenue, net | 42,300 | ||||||||||
TEGSEDI and WAYLIVRA [Member] | E.U. [Member] | |||||||||||
Disaggregation of Revenue [Abstract] | |||||||||||
Revenue, net | $ 7,700 | ||||||||||
Maximum [Member] | |||||||||||
Summary Of Significant Accounting Policies [Line Items] | |||||||||||
Period of time after billing when payment is received | 1 year | ||||||||||
Payment terms | 90 days | ||||||||||
Minimum [Member] | |||||||||||
Summary Of Significant Accounting Policies [Line Items] | |||||||||||
Payment terms | 30 days |
Summary of Significant Accou_10
Summary of Significant Accounting Policies - Summary of Balances and Activity in Each of Product Revenue Allowance and Reserve Categories (Details) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2019 | Dec. 31, 2018 | |
Valuation And Qualifying Accounts Disclosure [Line Items] | ||
Balance at December 31, 2017 | $ 348 | |
Provision related to current period sales | 4,036 | $ 348 |
Adjustment related to prior period sales | (32) | |
Credit or payments made during the period | (2,532) | |
Balance at December 31, 2018 | 1,820 | 348 |
Chargebacks, Discounts and Fees [Member] | ||
Valuation And Qualifying Accounts Disclosure [Line Items] | ||
Balance at December 31, 2017 | 50 | |
Provision related to current period sales | 1,240 | 50 |
Adjustment related to prior period sales | (4) | |
Credit or payments made during the period | (1,016) | |
Balance at December 31, 2018 | 270 | 50 |
Government and Other Rebates [Member] | ||
Valuation And Qualifying Accounts Disclosure [Line Items] | ||
Balance at December 31, 2017 | 293 | |
Provision related to current period sales | 2,604 | 293 |
Adjustment related to prior period sales | (28) | |
Credit or payments made during the period | (1,516) | |
Balance at December 31, 2018 | 1,353 | 293 |
Returns [Member] | ||
Valuation And Qualifying Accounts Disclosure [Line Items] | ||
Balance at December 31, 2017 | 5 | |
Provision related to current period sales | 192 | 5 |
Balance at December 31, 2018 | $ 197 | $ 5 |
Summary of Significant Accou_11
Summary of Significant Accounting Policies, Stock-Based Compensation Expense (Details) | 12 Months Ended |
Dec. 31, 2019 | |
Vesting Period 1 | RSUs [Member] | |
Stock-Based Compensation [Abstract] | |
Vesting period | 6 months |
Vesting Period 2 | RSUs [Member] | |
Stock-Based Compensation [Abstract] | |
Vesting period | 4 years |
2015 Equity Incentive Plan [Member] | Employee Stock Options [Member] | |
Stock-Based Compensation [Abstract] | |
Vesting period | 4 years |
Award term | 10 years |
2015 Equity Incentive Plan [Member] | Board of Director Stock Options [Member] | |
Stock-Based Compensation [Abstract] | |
Vesting period | 4 years |
Award term | 10 years |
2015 Equity Incentive Plan [Member] | Vesting Period 1 | Employee Stock Options [Member] | |
Stock-Based Compensation [Abstract] | |
Vesting percentage | 25.00% |
Summary of Significant Accou_12
Summary of Significant Accounting Policies, Accumulated Other Comprehensive Income (Loss ) (Details) - USD ($) $ in Thousands | 12 Months Ended | |||
Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | ||
Accumulated Other Comprehensive Loss [Roll Forward] | ||||
Beginning balance | $ 276,724 | |||
Net other comprehensive income (loss) | 329 | $ 127 | $ (430) | |
Ending balance | 543,004 | 276,724 | ||
Accumulated Other Comprehensive Loss [Member] | ||||
Accumulated Other Comprehensive Loss [Roll Forward] | ||||
Beginning balance | (324) | (451) | (21) | |
Ending balance | 5 | (324) | (451) | |
Unrealized Gains (Losses) on Investments [Member] | ||||
Accumulated Other Comprehensive Loss [Roll Forward] | ||||
Unrealized gains (losses) on investments, net of tax | [1] | 237 | 144 | (337) |
Currency Translation Adjustment [Member] | ||||
Accumulated Other Comprehensive Loss [Roll Forward] | ||||
Unrealized gains (losses) on investments, net of tax | $ 92 | $ (17) | $ (93) | |
[1] | There was no tax benefit for other comprehensive income (loss) for the years ended December 31, 2019, 2018 and 2017. |
Summary of Significant Accou_13
Summary of Significant Accounting Policies, Disclosure - Summary of Significant Accounting Policies, Accumulated Other Comprehensive Income (Loss ) (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Accumulated Other Comprehensive Loss [Roll Forward] | |||
Tax benefit included in other comprehensive income (loss) | $ 0 | $ 0 | $ 0 |
Summary of Significant Accou_14
Summary of Significant Accounting Policies, Income Taxes (Details) | 12 Months Ended | ||
Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Income Taxes [Abstract] | |||
U.S. federal statutory tax rate | 21.00% | 21.00% | 35.00% |
Investments and Fair Value Me_3
Investments and Fair Value Measurements - Summary of Investments (Details) - USD ($) $ in Thousands | Dec. 31, 2019 | Dec. 31, 2018 |
Summary of Investments [Abstract] | ||
Cost | $ 160,761 | |
Gross unrealized gains | 75 | |
Gross unrealized losses | (32) | |
Estimated fair value | 160,804 | |
Securities with Maturity of One Year or Less [Member] | ||
Summary of Investments [Abstract] | ||
Cost | 144,649 | $ 167,348 |
Gross unrealized gains | 70 | |
Gross unrealized losses | (29) | (193) |
Estimated fair value | 144,690 | 167,155 |
Securities with Maturity of More than One Year [Member] | ||
Summary of Investments [Abstract] | ||
Cost | 16,112 | |
Gross unrealized gains | 5 | |
Gross unrealized losses | (3) | |
Estimated fair value | 16,114 | |
Corporate Debt Securities [Member] | Securities with Maturity of One Year or Less [Member] | ||
Summary of Investments [Abstract] | ||
Cost | 105,679 | 81,770 |
Gross unrealized gains | 40 | |
Gross unrealized losses | (23) | (151) |
Estimated fair value | 105,696 | 81,619 |
Corporate Debt Securities [Member] | Securities with Maturity of More than One Year [Member] | ||
Summary of Investments [Abstract] | ||
Cost | 2,033 | |
Gross unrealized gains | 5 | |
Estimated fair value | 2,038 | |
Debt Securities Issued by U.S. Government Agencies [Member] | Securities with Maturity of One Year or Less [Member] | ||
Summary of Investments [Abstract] | ||
Cost | 38,970 | 85,578 |
Gross unrealized gains | 30 | |
Gross unrealized losses | (6) | (42) |
Estimated fair value | 38,994 | $ 85,536 |
Debt Securities Issued by U.S. Government Agencies [Member] | Securities with Maturity of More than One Year [Member] | ||
Summary of Investments [Abstract] | ||
Cost | 14,079 | |
Gross unrealized losses | (3) | |
Estimated fair value | $ 14,076 |
Investments and Fair Value Me_4
Investments and Fair Value Measurements - Fair Value Measurements (Details) - USD ($) $ in Thousands | Dec. 31, 2019 | Dec. 31, 2018 | |
Fair Value Measurements [Abstract] | |||
Available-for-sale securities | $ 160,804 | ||
Recurring Basis [Member] | |||
Fair Value Measurements [Abstract] | |||
Money market funds | [1] | 285,510 | $ 82,343 |
Total | 446,314 | 249,498 | |
Recurring Basis [Member] | Corporate Debt Securities [Member] | |||
Fair Value Measurements [Abstract] | |||
Available-for-sale securities | [2] | 107,735 | 81,619 |
Recurring Basis [Member] | Corporate Debt Securities [Member] | Cash and Cash Equivalents [Member] | |||
Fair Value Measurements [Abstract] | |||
Available-for-sale securities | 4,000 | 1,000 | |
Recurring Basis [Member] | Debt Securities Issued by U.S. Government Agencies [Member] | |||
Fair Value Measurements [Abstract] | |||
Available-for-sale securities | [3] | 53,069 | 85,536 |
Recurring Basis [Member] | Quoted Prices in Active Markets (Level 1) [Member] | |||
Fair Value Measurements [Abstract] | |||
Money market funds | [1] | 285,510 | 82,343 |
Total | 285,510 | 82,343 | |
Recurring Basis [Member] | Significant Other Observable Inputs (Level 2) [Member] | |||
Fair Value Measurements [Abstract] | |||
Total | 160,804 | 167,155 | |
Recurring Basis [Member] | Significant Other Observable Inputs (Level 2) [Member] | Corporate Debt Securities [Member] | |||
Fair Value Measurements [Abstract] | |||
Available-for-sale securities | [2] | 107,735 | 81,619 |
Recurring Basis [Member] | Significant Other Observable Inputs (Level 2) [Member] | Debt Securities Issued by U.S. Government Agencies [Member] | |||
Fair Value Measurements [Abstract] | |||
Available-for-sale securities | [3] | $ 53,069 | $ 85,536 |
[1] | Included in cash and cash equivalents on our consolidated balance sheet. | ||
[2] | At December 31, 2019 and 2018, $4.0 million and $1.0 million, respectively, was included in cash and cash equivalents on our consolidated balance sheet, with the difference included in short-term investments on our consolidated balance sheet. | ||
[3] | Included in short-term investments on our consolidated balance sheet. |
Property Plant and Equipment -
Property Plant and Equipment - Summary of Property and Equipment at Cost and Related Accumulated Depreciation (Details) - USD ($) $ in Thousands | Dec. 31, 2019 | Dec. 31, 2018 |
Property Plant And Equipment [Line Items] | ||
Property and equipment, at cost | $ 6,271 | $ 5,926 |
Less accumulated depreciation and amortization | (1,010) | (230) |
Total property and equipment, net | 5,261 | 5,696 |
Furniture And Fixtures [Member] | ||
Property Plant And Equipment [Line Items] | ||
Property and equipment, at cost | 1,611 | 1,611 |
Computer Equipment And Software [Member] | ||
Property Plant And Equipment [Line Items] | ||
Property and equipment, at cost | 289 | 102 |
Manufacturing Equipment [Member] | ||
Property Plant And Equipment [Line Items] | ||
Property and equipment, at cost | 416 | |
Leasehold Improvements [Member] | ||
Property Plant And Equipment [Line Items] | ||
Property and equipment, at cost | $ 3,955 | $ 4,213 |
Property Plant Equipment - Narr
Property Plant Equipment - Narratives (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Property Plant And Equipment [Abstract] | |||
Depreciation | $ 789 | $ 307 | $ 108 |
Tenant improvement allowance utilized | $ 3,600 |
Inventory - Schedule of Invento
Inventory - Schedule of Inventory (Details) - USD ($) $ in Thousands | Dec. 31, 2019 | Dec. 31, 2018 |
Inventory Disclosure [Abstract] | ||
Raw materials | $ 6,520 | |
Work in process | 2,039 | |
Finished goods | 258 | $ 85 |
Total inventories | $ 8,817 | $ 85 |
Inventory - Narratives (Details
Inventory - Narratives (Details) - USD ($) | 12 Months Ended | |
Dec. 31, 2019 | Dec. 31, 2018 | |
Inventory Disclosure [Abstract] | ||
Inventory write-offs | $ 200,000 | $ 0 |
Intangible Assets - Schedule of
Intangible Assets - Schedule of Intangible Assets (Details) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2019 | Dec. 31, 2018 | |
Finite Lived Intangible Assets [Line Items] | ||
Less accumulated amortization | $ (9,211) | $ (3,348) |
Total intangible assets, net | 83,051 | 88,914 |
Acquired and In-licensed Rights [Member] | ||
Finite Lived Intangible Assets [Line Items] | ||
Intangible assets, gross | $ 2,262 | 2,262 |
Acquired and In-licensed Rights [Member] | Minimum [Member] | ||
Finite Lived Intangible Assets [Line Items] | ||
Estimated useful life | 7 years | |
Acquired and In-licensed Rights [Member] | Maximum [Member] | ||
Finite Lived Intangible Assets [Line Items] | ||
Estimated useful life | 21 years | |
Capitalized Regulatory Approval Milestones [Member] | ||
Finite Lived Intangible Assets [Line Items] | ||
Intangible assets, gross | $ 90,000 | $ 90,000 |
Estimated useful life | 16 years |
Intangible Assets - Additional
Intangible Assets - Additional Information (Details) - USD ($) | Oct. 05, 2018 | Jul. 11, 2018 | Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 |
Finite Lived Intangible Assets [Line Items] | |||||
Amortization of intangibles | $ 5,863,000 | $ 2,870,000 | $ 120,000 | ||
Impairment losses | $ 0 | $ 0 | $ 0 | ||
Patents [Member] | |||||
Finite Lived Intangible Assets [Line Items] | |||||
Weighted average remaining amortizable life | 10 years 10 months 24 days | ||||
Ionis [Member] | Capitalized Regulatory Milestones [Member] | Inotersen [Member] | |||||
Finite Lived Intangible Assets [Line Items] | |||||
Milestone paid | $ 50,000,000 | $ 40,000,000 |
Intangible Assets - Schedule _2
Intangible Assets - Schedule of Estimated Future Amortization Expenses for Intangible Assets (Details) - USD ($) $ in Thousands | Dec. 31, 2019 | Dec. 31, 2018 |
Goodwill And Intangible Assets Disclosure [Abstract] | ||
2020 | $ 5,879 | |
2021 | 5,861 | |
2022 | 5,856 | |
2023 | 5,843 | |
2024 | 5,822 | |
Thereafter | 53,790 | |
Total intangible assets, net | $ 83,051 | $ 88,914 |
Strategic Collaboration with _2
Strategic Collaboration with Novartis (Details) $ in Thousands | Jul. 19, 2017USD ($) | Jul. 31, 2017USD ($) | Dec. 31, 2019USD ($) | Sep. 30, 2019USD ($) | Jun. 30, 2019USD ($) | Mar. 31, 2019USD ($)shares | Dec. 31, 2018USD ($) | Sep. 30, 2018USD ($) | Jun. 30, 2018USD ($) | Mar. 31, 2018USD ($) | Mar. 31, 2017USD ($) | Dec. 31, 2019USD ($) | Dec. 31, 2018USD ($) | Dec. 31, 2017USD ($)shares | Jan. 31, 2017PerformanceObligation |
Strategic Collaboration with Novartis [Abstract] | |||||||||||||||
Proceeds from sale of common stock to Novartis in a private placement | $ 50,000 | $ 50,000 | |||||||||||||
Revenue | $ 277,480 | $ 20,624 | $ 26,623 | $ 163,816 | $ 10,197 | $ 19,241 | $ 18,321 | $ 17,108 | $ 488,543 | $ 64,867 | $ 43,401 | ||||
Revenue recognized from deferred revenue | 28,800 | ||||||||||||||
Common Stock [Member] | |||||||||||||||
Strategic Collaboration with Novartis [Abstract] | |||||||||||||||
Issuance of stock (in shares) | shares | 17,969,000 | ||||||||||||||
Ionis [Member] | Akcea [Member] | |||||||||||||||
Strategic Collaboration with Novartis [Abstract] | |||||||||||||||
Sublicense fee payable | $ 75,000 | ||||||||||||||
Ionis [Member] | Common Stock [Member] | Akcea [Member] | |||||||||||||||
Strategic Collaboration with Novartis [Abstract] | |||||||||||||||
Issuance of stock (in shares) | shares | 2,837,373 | ||||||||||||||
Development Services for AKCEA-APO(a)-L [Member] | |||||||||||||||
Strategic Collaboration with Novartis [Abstract] | |||||||||||||||
Transaction price | $ 64,000 | ||||||||||||||
Development Services for AKCEA-APOCIII-L [Member] | |||||||||||||||
Strategic Collaboration with Novartis [Abstract] | |||||||||||||||
Transaction price | 40,100 | ||||||||||||||
Delivery of AKCEA-APO(a)-L [Member] | |||||||||||||||
Strategic Collaboration with Novartis [Abstract] | |||||||||||||||
Transaction price | 1,500 | ||||||||||||||
Delivery of AKCEA-APOCIII-L [Member] | |||||||||||||||
Strategic Collaboration with Novartis [Abstract] | |||||||||||||||
Transaction price | 2,800 | ||||||||||||||
Licensing [Member] | |||||||||||||||
Strategic Collaboration with Novartis [Abstract] | |||||||||||||||
Revenue | 4,000 | 136 | $ 6,036 | $ 12,000 | 10,172 | 12,000 | $ 0 | ||||||||
Licensing [Member] | Akcea [Member] | |||||||||||||||
Strategic Collaboration with Novartis [Abstract] | |||||||||||||||
Revenue | $ 150,000 | ||||||||||||||
AKCEA-APO(a)-L [Member] | |||||||||||||||
Strategic Collaboration with Novartis [Abstract] | |||||||||||||||
Maximum amount of payments receivable for milestones | 675,000 | 675,000 | |||||||||||||
Maximum amount of payments receivable for development milestones | 25,000 | 25,000 | |||||||||||||
Maximum amount of payments receivable for regulatory milestones | 290,000 | 290,000 | |||||||||||||
Maximum amount of payments receivable for commercialization milestones | 360,000 | $ 360,000 | |||||||||||||
Royalty percentage received on sales of drug | 20.00% | ||||||||||||||
Novartis [Member] | |||||||||||||||
Strategic Collaboration with Novartis [Abstract] | |||||||||||||||
Upfront payment received | 75,000 | ||||||||||||||
Portion of upfront payment retained | 60,000 | ||||||||||||||
Portion of upfront payment paid as sublicense fee to Ionis | 15,000 | ||||||||||||||
Number of separate performance obligations | PerformanceObligation | 4 | ||||||||||||||
Transaction price | 108,400 | ||||||||||||||
Premium received on shares issued by lonis | 28,400 | ||||||||||||||
Potential premium received if Ionis common stock is purchased in the future | $ 5,000 | ||||||||||||||
Number of months from inception of agreement for IPO to be completed | 15 months | ||||||||||||||
Proceeds from sale of common stock to Novartis in a private placement | $ 50,000 | ||||||||||||||
Revenue | $ 187,400 | 50,600 | $ 43,400 | ||||||||||||
Deferred revenue | 0 | $ 28,800 | 0 | $ 28,800 | |||||||||||
Novartis [Member] | AKCEA-APO(a)-L [Member] | |||||||||||||||
Strategic Collaboration with Novartis [Abstract] | |||||||||||||||
Next prospective milestone | $ 25,000 | $ 25,000 | |||||||||||||
Novartis [Member] | Delivery of AKCEA-APO(a)-L [Member] | |||||||||||||||
Strategic Collaboration with Novartis [Abstract] | |||||||||||||||
Revenue | $ 5,500 |
License Agreements and Servic_3
License Agreements and Services Agreement with Ionis, Cardiometabolic Development, Commercialization and License Agreement (Details) - Ionis [Member] - Cardiometabolic Development, Commercialization and License Agreement [Member] $ in Millions | 12 Months Ended |
Dec. 31, 2019USD ($)Patientqtr | |
Development, Commercialization and License Agreement with Ionis [Abstract] | |
Royalty percentage paid on sales of lipid drug | 20.00% |
First annual sales threshold | $ | $ 500 |
Second annual sales threshold | $ | 1,000 |
Third annual sales threshold | $ | 2,000 |
Sales milestone payment | $ | $ 50 |
Number of quarters in which equal payments are made | qtr | 12 |
Percentage of license fees, milestone payments and royalties paid as sublicense fee to Ionis | 50.00% |
Lipid Drug for Rare Disease Indication [Member] | Maximum [Member] | |
Development, Commercialization and License Agreement with Ionis [Abstract] | |
Number of patients worldwide | Patient | 500,000 |
Number of patients for Phase 3 program | Patient | 1,000 |
Number of years of treatment | 2 years |
Lipid Drug for a Broad Disease Patient Population [Member] | Minimum [Member] | |
Development, Commercialization and License Agreement with Ionis [Abstract] | |
Number of patients worldwide | Patient | 500,000 |
Number of patients for Phase 3 program | Patient | 1,000 |
Number of years of treatment | 2 years |
License Agreements and Servic_4
License Agreements and Services Agreement with Ionis, TTR Development, Commercialization, Collaboration and License Agreement (Details) $ in Thousands | Oct. 17, 2018USD ($)shares | Aug. 03, 2018USD ($)shares | Apr. 17, 2018USD ($)Milestoneshares | Dec. 31, 2019USD ($)shares | Sep. 30, 2019USD ($) | Jun. 30, 2019USD ($) | Mar. 31, 2019USD ($) | Dec. 31, 2018USD ($)shares | Sep. 30, 2018USD ($) | Dec. 31, 2019USD ($)shares | Dec. 31, 2018USD ($)shares | Dec. 31, 2017USD ($) | Apr. 16, 2018shares |
TTR Development, Commercialization, Collaboration and License Agreement [Abstract] | |||||||||||||
Stock purchased by Ionis | $ 135,438 | ||||||||||||
Common stock, shares authorized (in shares) | shares | 125,000,000 | 125,000,000 | 125,000,000 | 125,000,000 | |||||||||
Intangible assets, net | $ 83,051 | $ 88,914 | $ 83,051 | $ 88,914 | |||||||||
Amortization expense of milestone payment | 1,434 | $ 1,434 | $ 1,419 | $ 1,403 | $ 2,012 | $ 701 | $ 5,690 | 2,713 | $ 0 | ||||
Ionis [Member] | |||||||||||||
TTR Development, Commercialization, Collaboration and License Agreement [Abstract] | |||||||||||||
Costs incurred by Ionis prior to closing of TTR Agreement | $ 3,100 | ||||||||||||
Ionis [Member] | TTR License Agreement [Member] | |||||||||||||
TTR Development, Commercialization, Collaboration and License Agreement [Abstract] | |||||||||||||
Upfront licensing fee paid to Ionis | $ 150,000 | ||||||||||||
Shares issued in consideration of upfront licensing fee (in shares) | shares | 8,000,000 | ||||||||||||
Number of sales milestones | Milestone | 7 | ||||||||||||
Annual worldwide net sales required for subsequent milestone payments to be paid in cash | $ 750,000 | ||||||||||||
Notice period for termination of agreement | 90 days | ||||||||||||
Common stock, shares authorized (in shares) | shares | 125,000,000 | 100,000,000 | |||||||||||
Intangible assets, net | $ 600 | ||||||||||||
Commercial Inotersen inventory acquired from Ionis | $ 4,700 | ||||||||||||
Clinical material acquired from Ionis | $ 13,500 | ||||||||||||
Percentage of profits and losses from development and commercialization of Inotersen paid to Ionis, tier 1 | 60.00% | ||||||||||||
Percentage of profits and losses from development and commercialization of Inotersen retained, tier 1 | 40.00% | ||||||||||||
Percentage of profits and losses from development and commercialization of Inotersen paid to Ionis, tier 2 | 50.00% | ||||||||||||
Percentage of profits and losses from development and commercialization of Inotersen retained, tier 2 | 50.00% | ||||||||||||
Percentage of profits and losses from development and commercialization of AKCEA-TTR-L paid to Ionis | 50.00% | ||||||||||||
Percentage of profits and losses from development and commercialization of AKCEA-TTR-L retained | 50.00% | ||||||||||||
Ionis [Member] | TTR License Agreement [Member] | Ionis Stock Purchase Agreement [Member] | |||||||||||||
TTR Development, Commercialization, Collaboration and License Agreement [Abstract] | |||||||||||||
Common stock shares issued to Ionis | shares | 10,700,000 | ||||||||||||
Stock purchased by Ionis | $ 200,000 | ||||||||||||
Ionis [Member] | TTR License Agreement [Member] | Inotersen [Member] | |||||||||||||
TTR Development, Commercialization, Collaboration and License Agreement [Abstract] | |||||||||||||
Maximum amount of payments payable for milestones | 110,000 | $ 110,000 | |||||||||||
Ionis [Member] | TTR License Agreement [Member] | AKCEA-TTR-L [Member] | |||||||||||||
TTR Development, Commercialization, Collaboration and License Agreement [Abstract] | |||||||||||||
Maximum amount of payments payable for milestones | 145,000 | 145,000 | |||||||||||
Ionis [Member] | TTR License Agreement [Member] | Inotersen and AKCEA-TTR-L [Member] | |||||||||||||
TTR Development, Commercialization, Collaboration and License Agreement [Abstract] | |||||||||||||
Maximum amount of payments payable for milestones | $ 1,300,000 | 1,300,000 | |||||||||||
Ionis [Member] | TTR License Agreement [Member] | TEGSEDI [Member] | |||||||||||||
TTR Development, Commercialization, Collaboration and License Agreement [Abstract] | |||||||||||||
Common stock shares issued to Ionis | shares | 1,671,849 | 1,597,571 | |||||||||||
Amount of regulatory approval milestone paid by issuing common stock | $ 50,000 | $ 40,000 | |||||||||||
Amortization expense of milestone payment | $ 5,700 | $ 2,700 | |||||||||||
Ionis [Member] | TTR License Agreement [Member] | TEGSEDI [Member] | Milestone Payment [Member] | |||||||||||||
TTR Development, Commercialization, Collaboration and License Agreement [Abstract] | |||||||||||||
Useful life of milestone payment amortized to cost of sales | 16 years |
License Agreements and Servic_5
License Agreements and Services Agreement with Ionis - Summary of Loss Share Related to Commercial Activities Under TTR Agreement (Details) - USD ($) $ in Thousands | 3 Months Ended | 12 Months Ended | |||||
Dec. 31, 2019 | Sep. 30, 2019 | Jun. 30, 2019 | Mar. 31, 2019 | Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Related Party Transaction [Line Items] | |||||||
Net losses incurred by the collaboration related to the commercial activities under the TTR License Agreement | $ (66,210) | ||||||
Akcea [Member] | |||||||
Related Party Transaction [Line Items] | |||||||
Akcea's share of commercial losses under the TTR License Agreement reflected in our consolidated statements of operations | (26,487) | ||||||
Ionis [Member] | |||||||
Related Party Transaction [Line Items] | |||||||
Net loss share from commercial activities under arrangement with Ionis Pharmaceuticals, Inc. | $ (10,313) | $ (8,889) | $ (11,465) | $ (9,056) | $ (39,723) | $ 0 | $ 0 |
License Agreements and Servic_6
License Agreements and Services Agreement with Ionis - Summary of Development Expenses Related to TTR License Agreement (Details) $ in Thousands | 12 Months Ended |
Dec. 31, 2019USD ($) | |
Related Party Transaction [Line Items] | |
Total development expense incurred by the collaboration related to development activities under the TTR License Agreement | $ (66,663) |
Akcea [Member] | |
Related Party Transaction [Line Items] | |
Akcea's share of TTR development expense reflected in research and development expense in our consolidated statements of operations | $ (29,444) |
License Agreements and Servic_7
License Agreements and Services Agreement with Ionis, Services Agreement (Details) - USD ($) $ in Thousands | 12 Months Ended | |||
Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | ||
Services Agreement with Ionis [Abstract] | ||||
Payment term after receipt of invoice | 30 days | |||
Term of extension for services agreement | 6 months | |||
Ionis [Member] | Services Agreement [Member] | ||||
Operating expenses: | ||||
Services performed by Ionis | [1] | $ 4,703 | $ 15,404 | $ 9,742 |
Active pharmaceutical ingredient manufactured by Ionis | [1] | 5,515 | 5,229 | 6,012 |
Pre-commercial inventory manufactured by Ionis | [1] | 5,996 | ||
Sublicensing expenses | [1] | 205,400 | 7,200 | 48,394 |
Out-of-pocket expenses paid by Ionis | [1] | 4,250 | 46,162 | 37,426 |
Royalty expenses | [1] | 183 | ||
Less: commercial share of loss in connection with the TTR license transaction | [1] | (39,695) | ||
Plus: R&D share of loss in connection with the TTR license transaction | [1] | 3,651 | ||
Total operating expenses generated by transactions with Ionis | [1] | 184,007 | 79,991 | 101,574 |
Plus: distributions to Ionis: | [1] | 13,492 | 7,792 | |
(Receivable) payable balance (from) to Ionis at the beginning of the period | [1] | 18,901 | 14,365 | 24,355 |
Total charges generated by transactions with Ionis | [1] | 197,499 | 87,783 | 101,574 |
Less: total amounts received from (paid to) Ionis during the period | [1] | (19,631) | (83,247) | (78,170) |
Less: non-cash sublicensing expenses | [1] | (200,000) | (33,394) | |
Total amount (receivable) payable (from) to Ionis at period end | [1] | $ (3,231) | $ 18,901 | $ 14,365 |
[1] | This table excludes amounts capitalized as license intangible assets on the balance sheet. |
Collaboration and License Agr_2
Collaboration and License Agreement with PTC Therapeutics - Additional Information (Details) | Aug. 01, 2018USD ($) | Dec. 31, 2019USD ($)PerformanceObligation | Sep. 30, 2019USD ($) | Jun. 30, 2019USD ($) | Mar. 31, 2019USD ($) | Dec. 31, 2018USD ($) | Sep. 30, 2018USD ($) | Jun. 30, 2018USD ($) | Mar. 31, 2018USD ($) | Dec. 31, 2019USD ($)PerformanceObligation | Dec. 31, 2018USD ($) | Dec. 31, 2017USD ($) | Oct. 31, 2019USD ($) | May 31, 2019USD ($) |
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||
Revenue | $ 277,480,000 | $ 20,624,000 | $ 26,623,000 | $ 163,816,000 | $ 10,197,000 | $ 19,241,000 | $ 18,321,000 | $ 17,108,000 | $ 488,543,000 | $ 64,867,000 | $ 43,401,000 | |||
Cardiometabolic License Agreement [Member] | ||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||
Sublicense fee paid | $ 3,000,000 | |||||||||||||
TTR License Agreement [Member] | ||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||
Sublicense fee paid | 2,400,000 | |||||||||||||
TEGSEDI [Member] | ||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||
Revenue | $ 2,200,000 | |||||||||||||
PTC Therapeutics [Member] | ||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||
Upfront payment received | $ 12,000,000 | |||||||||||||
Revenue | $ 4,000,000 | $ 6,000,000 | 12,000,000 | |||||||||||
Deferred revenue | $ 0 | |||||||||||||
Number of separate performance obligations | PerformanceObligation | 2 | 2 | ||||||||||||
Consideration amount received | $ 4,000,000 | $ 6,000,000 | ||||||||||||
PTC Therapeutics [Member] | Akcea [Member] | ||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||
Upfront payment received | 12,000,000 | |||||||||||||
Sublicense fee paid | 7,200,000 | |||||||||||||
Payment received | 6,000,000 | |||||||||||||
Regulatory milestone payment received | $ 4,000,000 | |||||||||||||
Additional amount of payments receivable per drug for regulatory milestone | $ 4,000,000 | |||||||||||||
Period before PTC pays royalties on net sales of product after first commercial sale in Brazil | 12 months | |||||||||||||
Minimum revenue recognized in Latin America by PTC before paying royalties | $ 10,000,000 | |||||||||||||
PTC Therapeutics [Member] | TEGSEDI [Member] | Akcea [Member] | ||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||
Milestone payments and royalties retained percentage | 40.00% | |||||||||||||
PTC Therapeutics [Member] | TEGSEDI [Member] | Ionis [Member] | ||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||
Milestone payments and royalties payable percentage | 60.00% | |||||||||||||
PTC Therapeutics [Member] | WAYLIVRA [Member] | Ionis [Member] | ||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||
Milestone payments and royalties payable percentage | 50.00% |
License Agreement with Pfizer -
License Agreement with Pfizer - Narratives (Details) $ in Thousands | 1 Months Ended | 3 Months Ended | 12 Months Ended | |||||||||
Oct. 31, 2019USD ($)PerformanceObligation | Dec. 31, 2019USD ($)shares | Sep. 30, 2019USD ($) | Jun. 30, 2019USD ($) | Mar. 31, 2019USD ($) | Dec. 31, 2018USD ($) | Sep. 30, 2018USD ($) | Jun. 30, 2018USD ($) | Mar. 31, 2018USD ($) | Dec. 31, 2019USD ($) | Dec. 31, 2018USD ($) | Dec. 31, 2017USD ($) | |
License Agreement with Pfizer [Abstract] | ||||||||||||
Revenue | $ 277,480 | $ 20,624 | $ 26,623 | $ 163,816 | $ 10,197 | $ 19,241 | $ 18,321 | $ 17,108 | $ 488,543 | $ 64,867 | $ 43,401 | |
Pfizer License Agreement [Member] | ||||||||||||
License Agreement with Pfizer [Abstract] | ||||||||||||
Upfront license fee receivable | $ 250,000 | |||||||||||
Issuance of stock (in shares) | shares | 6,873,344 | |||||||||||
Sublicense fee payable | $ 125,000 | 125,000 | ||||||||||
Number of separate performance obligations | PerformanceObligation | 3 | |||||||||||
Upfront payment received | 250,000 | |||||||||||
Transaction price | 250,000 | 250,000 | ||||||||||
Revenue | 248,700 | |||||||||||
Deferred revenue | 1,300 | 1,300 | ||||||||||
Pfizer License Agreement [Member] | Transfer of License of AKCEA-ANGPTL3-LRx [Member] | ||||||||||||
License Agreement with Pfizer [Abstract] | ||||||||||||
Transaction price | 245,600 | 245,600 | ||||||||||
Pfizer License Agreement [Member] | Development services for AKCEA-ANGPTL3-LRx [Member] | ||||||||||||
License Agreement with Pfizer [Abstract] | ||||||||||||
Transaction price | 2,200 | 2,200 | ||||||||||
Pfizer License Agreement [Member] | Delivery of AKCEA-ANGPTL3-LRx API [Member] | ||||||||||||
License Agreement with Pfizer [Abstract] | ||||||||||||
Transaction price | $ 2,200 | $ 2,200 | ||||||||||
Pfizer License Agreement [Member] | AKCEA-ANGPTL3-LRx [Member] | ||||||||||||
License Agreement with Pfizer [Abstract] | ||||||||||||
Next prospective milestone | $ 75,000 | |||||||||||
Maximum amount of payments receivable for milestones | 1,300,000 | |||||||||||
Maximum amount of payments receivable for development milestones | 205,000 | |||||||||||
Maximum amount of payments receivable for regulatory milestones | 250,000 | |||||||||||
Maximum amount of payments receivable for commercialization milestones | $ 850,000 | |||||||||||
Royalty percentage received on sales of drug | 20.00% | |||||||||||
Percentage of license fees, milestone payments and royalties paid as sublicense fee to Ionis | 50.00% |
Equity and Stock-Based Compen_3
Equity and Stock-Based Compensation - Series A Convertible Preferred Stock (Details) - USD ($) $ in Millions | 1 Months Ended | 12 Months Ended | ||||
Dec. 31, 2015 | Dec. 31, 2019 | Dec. 31, 2018 | Jul. 31, 2017 | May 31, 2017 | Dec. 31, 2016 | |
Series A Convertible Preferred Stock [Abstract] | ||||||
Preferred stock, shares authorized (in shares) | 10,000,000 | 40,000,000 | ||||
Preferred stock, shares outstanding (in shares) | 0 | 0 | ||||
Preferred Stock [Member] | ||||||
Series A Convertible Preferred Stock [Abstract] | ||||||
Shares issued (in shares) | 28,884,540 | |||||
Proceeds from sale of stock | $ 100 | |||||
Preferred stock, shares authorized (in shares) | 28,884,540 | |||||
Preferred stock, shares issued (in shares) | 0 | 0 | 28,884,540 | |||
Preferred stock, shares outstanding (in shares) | 0 | 0 | 28,884,540 | |||
Conversion ratio | convertible 1:1 into common stock |
Equity and Stock-Based Compen_4
Equity and Stock-Based Compensation - Preferred Stock (Details) - $ / shares | Dec. 31, 2019 | Dec. 31, 2018 | Jul. 31, 2017 | May 31, 2017 |
Stockholders' Equity (Deficit) [Abstract] | ||||
Preferred stock, shares authorized (in shares) | 10,000,000 | 40,000,000 | ||
Preferred Stock, Par or Stated Value Per Share | $ 0.001 | |||
Preferred stock, shares outstanding (in shares) | 0 | 0 |
Equity and Stock-Based Compen_5
Equity and Stock-Based Compensation - Common Stock (Details) $ in Millions | 1 Months Ended | |||
May 31, 2017USD ($)shares | Dec. 31, 2019shares | Dec. 31, 2018shares | Jul. 31, 2017shares | |
Stockholders' Equity (Deficit) [Abstract] | ||||
Common stock, shares authorized (in shares) | 125,000,000 | 125,000,000 | ||
Common stock, shares issued (in shares) | 100,993,173 | 89,345,978 | 66,541,629 | |
Common stock, shares outstanding (in shares) | 100,993,173 | 89,345,978 | 66,541,629 | |
Stock split ratio | 0.3914 | |||
Preferred stock, shares authorized (in shares) | 40,000,000 | 10,000,000 | ||
Minimum Threshold For Gross Proceeds From IPO Required Under Charter Amendment | $ | $ 50 |
Equity and Stock-Based Compen_6
Equity and Stock-Based Compensation - Stock Plans (Details) - USD ($) $ in Millions | 12 Months Ended | ||||
Dec. 31, 2019 | Jan. 01, 2019 | Dec. 31, 2018 | Jul. 31, 2017 | Jun. 30, 2017 | |
Stock Plans [Abstract] | |||||
Shares of common stock added | 100,993,173 | 89,345,978 | 66,541,629 | ||
RSUs [Member] | |||||
Stock Plans [Abstract] | |||||
Restricted stock units outstanding (in shares) | 701,000 | 38,000 | |||
2015 Equity Incentive Plan [Member] | |||||
Stock Plans [Abstract] | |||||
Number of shares of common stock reserved for issuance (in shares) | 8,500,000 | ||||
Number of shares authorized for issuance pursuant to stock awards (in shares) | 18,500,000 | ||||
Number of shares available for future grant (in shares) | 4,541,985 | ||||
2015 Equity Incentive Plan [Member] | Stock Options [Member] | |||||
Stock Plans [Abstract] | |||||
Options outstanding (in shares) | 8,866,474 | 11,011,000 | |||
Options exercisable (in shares) | 3,094,602 | ||||
2015 Equity Incentive Plan [Member] | RSUs [Member] | |||||
Stock Plans [Abstract] | |||||
Restricted stock units outstanding (in shares) | 701,252 | ||||
2017 Employee Stock Purchase Plan [Member] | |||||
Stock Plans [Abstract] | |||||
Number of shares of common stock reserved for issuance (in shares) | 1,500,000 | 500,000 | |||
Number of shares available for future grant (in shares) | 1,428,725 | ||||
Percentage of shares outstanding used to calculate annual increase in number of shares that can be issued under ESPP | 1.00% | ||||
Annual maximum increase in number of shares that can be issued under ESPP (in shares) | 500,000 | ||||
Shares of common stock added | 500,000 | ||||
Purchase price of common stock, percent | 85.00% | ||||
Shares issued under ESPP (in shares) | 39,724 | ||||
Accrued liability for contributions to employee stock purchase plan | $ 0.4 |
Equity and Stock-Based Compen_7
Equity and Stock-Based Compensation - Stock Option Activity (Details) - 2015 Equity Incentive Plan [Member] - USD ($) $ / shares in Units, $ in Thousands | 12 Months Ended | ||||
Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |||
Unrecognized Compensation Expense [Abstract] | |||||
Unrecognized compensation expense related to non-vested stock options | $ 45,900 | ||||
Stock Options [Member] | |||||
Number of Shares [Abstract] | |||||
Outstanding at beginning of period (in shares) | 11,011,000 | ||||
Granted (in shares) | 4,756,000 | ||||
Exercised (in shares) | (3,540,593) | [1] | (834,800) | 0 | |
Cancelled/forfeited/expired (in shares) | (3,360,000) | ||||
Outstanding at end of period (in shares) | 8,866,474 | 11,011,000 | |||
Options exercisable (in shares) | 3,094,602 | ||||
Weighted Average Exercise Price Per Share [Abstract] | |||||
Outstanding at beginning of period (in dollars per share) | $ 15 | ||||
Granted (in dollars per share) | 27.04 | ||||
Exercised (in dollars per share) | [1] | 7.99 | |||
Cancelled/forfeited/expired (in dollars per share) | 24.13 | ||||
Outstanding at end of period (in dollars per share) | 20.81 | $ 15 | |||
Exercisable at end of period (in dollars per share) | $ 14.68 | ||||
Average Remaining Contractual Term, Aggregate Intrinsic Value and Other [Abstract] | |||||
Average remaining contractual term, Outstanding | 7 years 11 months 19 days | ||||
Average remaining contractual term, Exercisable | 6 years 5 months 4 days | ||||
Aggregate intrinsic value, Outstanding | $ 16,627 | ||||
Aggregate intrinsic value, Exercisable | $ 14,183 | ||||
Unrecognized Compensation Expense [Abstract] | |||||
Weighted average fair value of options granted (in dollars per share) | $ 27.04 | $ 18.29 | $ 10.40 | ||
Exercise of common stock options (in shares) | 3,540,593 | [1] | 834,800 | 0 | |
Non-vested Stock Options [Member] | |||||
Unrecognized Compensation Expense [Abstract] | |||||
Weighted average period for recognition | 1 year 5 months 12 days | ||||
[1] | This amount includes 1,052 shares withheld to cover the exercise price for options which were net exercised |
Equity and Stock-Based Compen_8
Equity and Stock-Based Compensation - Stock Option Activity (Parenthetical) (Details) | 12 Months Ended |
Dec. 31, 2019shares | |
2015 Equity Incentive Plan [Member] | Stock Options [Member] | |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |
Shares withheld to cover the exercise price for options which were net exercised | 1,052 |
Equity and Stock-Based Compen_9
Equity and Stock-Based Compensation - Restricted Stock Unit Activity (Details) - RSUs [Member] - USD ($) $ / shares in Units, shares in Thousands, $ in Millions | 12 Months Ended | ||
Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Weighted Average Grant Date Fair Value | |||
Non-vested, Weighted Average Grant Date Fair Value, Beginning Balance (in dollar per share) | $ 25.65 | ||
Granted (in dollar per share) | 21.95 | $ 27.80 | $ 23.04 |
Vested (in dollar per share) | 25.76 | ||
Cancelled/forfeited/expired (in dollar per share) | 25.01 | ||
Non-vested, Weighted Average Grant Date Fair Value, Ending Balance (in dollar per share) | $ 21.95 | $ 25.65 | |
Number of Shares | |||
Non-vested shares, Beginning Balance | 38 | ||
Granted (In shares) | 706 | ||
Vested (In shares) | (9) | ||
Cancelled/forfeited/expired (In shares) | (34) | ||
Non-vested shares, Ending Balance | 701 | 38 | |
Granted (in dollar per share) | $ 21.95 | $ 27.80 | $ 23.04 |
Unrecognized compensation cost related to RSUs | $ 12.5 | ||
Weighted average period for recognition | 2 years 5 months 19 days |
Equity and Stock-Based Compe_10
Equity and Stock-Based Compensation - Breakdown of Stock-Based Compensation Expense (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Stock-Based Compensation [Abstract] | |||
Stock-based compensation expense | $ 37,112 | $ 44,282 | $ 17,539 |
Cost of Sales - Product [Member] | |||
Stock-Based Compensation [Abstract] | |||
Stock-based compensation expense | 437 | 160 | |
Research and Development Expenses [Member] | |||
Stock-Based Compensation [Abstract] | |||
Stock-based compensation expense | 11,895 | 9,435 | 8,630 |
Selling, General and Administrative Expenses [Member] | |||
Stock-Based Compensation [Abstract] | |||
Stock-based compensation expense | $ 24,780 | $ 34,687 | $ 8,909 |
Equity and Stock-Based Compe_11
Equity and Stock-Based Compensation - Stock-based Valuation Information (Details) | 12 Months Ended | ||
Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Employee Stock Option [Member] | 2015 Equity Incentive Plan [Member] | |||
Weighted-Average Assumptions [Abstract] | |||
Risk-free interest rate | 2.30% | 2.80% | 1.90% |
Dividend yield | 0.00% | 0.00% | 0.00% |
Volatility | 75.50% | 77.10% | 79.50% |
Expected life | 6 years 29 days | 6 years 29 days | 6 years 21 days |
Board of Director Stock Options [Member] | 2015 Equity Incentive Plan [Member] | |||
Weighted-Average Assumptions [Abstract] | |||
Risk-free interest rate | 1.80% | 2.90% | 1.90% |
Dividend yield | 0.00% | 0.00% | 0.00% |
Volatility | 73.80% | 78.20% | 79.40% |
Expected life | 6 years 3 months | 6 years 5 months 1 day | 6 years 3 months |
ESPP [Member] | |||
Weighted-Average Assumptions [Abstract] | |||
Risk-free interest rate | 2.40% | 1.90% | 1.10% |
Dividend yield | 0.00% | 0.00% | 0.00% |
Volatility | 60.00% | 64.20% | 73.30% |
Expected life | 6 months | 6 months | 6 months |
Income Taxes, Income (Loss) Bef
Income Taxes, Income (Loss) Before Income Taxes (Details) - USD ($) $ in Thousands | 3 Months Ended | 12 Months Ended | |||||||||
Dec. 31, 2019 | Sep. 30, 2019 | Jun. 30, 2019 | Mar. 31, 2019 | Dec. 31, 2018 | Sep. 30, 2018 | Jun. 30, 2018 | Mar. 31, 2018 | Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Income Tax Disclosure [Abstract] | |||||||||||
United States | $ 40,475 | $ (218,794) | $ (108,691) | ||||||||
Foreign | 2,489 | (6,580) | (11,593) | ||||||||
Income (loss) before income tax expense | $ 84,047 | $ (31,239) | $ (37,163) | $ 27,319 | $ (70,557) | $ (63,358) | $ (61,832) | $ (29,627) | $ 42,964 | $ (225,374) | $ (120,284) |
Income Taxes, Provision for Inc
Income Taxes, Provision for Income Taxes (Details) - USD ($) $ in Thousands | 3 Months Ended | 12 Months Ended | |||||||
Dec. 31, 2019 | Sep. 30, 2019 | Jun. 30, 2019 | Mar. 31, 2019 | Sep. 30, 2018 | Jun. 30, 2018 | Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Current: | |||||||||
Federal | $ 1,692 | ||||||||
State | 87 | $ 73 | $ 1,041 | ||||||
Foreign | 413 | 374 | 234 | ||||||
Total current | 2,192 | 447 | 1,275 | ||||||
Income tax expense | $ 1,641 | $ 259 | $ 160 | $ 132 | $ 233 | $ 214 | $ 2,192 | $ 447 | $ 1,275 |
Income Taxes, Reconciliation of
Income Taxes, Reconciliation of Statutory to Effective Tax Rate (Details) - USD ($) $ in Thousands | 3 Months Ended | 12 Months Ended | |||||||||
Dec. 31, 2019 | Sep. 30, 2019 | Jun. 30, 2019 | Mar. 31, 2019 | Dec. 31, 2018 | Sep. 30, 2018 | Jun. 30, 2018 | Mar. 31, 2018 | Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Reconciliation Between Effective and Statutory Tax Rate [Abstract] | |||||||||||
Pretax income (loss) | $ 84,047 | $ (31,239) | $ (37,163) | $ 27,319 | $ (70,557) | $ (63,358) | $ (61,832) | $ (29,627) | $ 42,964 | $ (225,374) | $ (120,284) |
Statutory rate | 9,023 | (47,329) | (42,099) | ||||||||
State income tax net of federal benefit | (3,025) | (6,441) | (2,371) | ||||||||
Impact of foreign tax rate differential | 340 | 1,735 | 4,072 | ||||||||
Net change in valuation allowance | (2,072) | 54,173 | (18,917) | ||||||||
IP Transfer | (3,947) | ||||||||||
Tax credits | (1,559) | (4,035) | 4,189 | ||||||||
IPO/Deconsolidation adjustment | 37,911 | ||||||||||
Tax rate change | 2,178 | 3,906 | 19,046 | ||||||||
Nondeductible items and other | (222) | 1,734 | (556) | ||||||||
Inventory purchase from Ionis | (2,833) | ||||||||||
Intangible basis difference | (2,596) | ||||||||||
Foreign derived intangible income benefit | (481) | ||||||||||
Stock-based compensation | 3,439 | 651 | |||||||||
Income tax expense | $ 1,641 | $ 259 | $ 160 | $ 132 | $ 233 | $ 214 | $ 2,192 | $ 447 | $ 1,275 | ||
Income Taxes [Abstract] | |||||||||||
Statutory rate | 21.00% | 21.00% | 35.00% | ||||||||
State income tax net of federal benefit | (7.00%) | 2.90% | 2.00% | ||||||||
Impact of foreign tax rate differential | 0.80% | (0.80%) | (3.40%) | ||||||||
Net change in valuation allowance | (4.80%) | (24.00%) | 15.70% | ||||||||
IP Transfer | 1.80% | ||||||||||
Tax credits | (3.60%) | 1.80% | (3.50%) | ||||||||
IPO/Deconsolidation adjustment | (31.50%) | ||||||||||
Tax rate change | 5.10% | (1.70%) | (15.80%) | ||||||||
Nondeductible items and other | (0.70%) | (0.90%) | 0.50% | ||||||||
Inventory purchase from Ionis | (6.60%) | ||||||||||
Intangible basis difference | (6.00%) | ||||||||||
Foreign derived intangible income benefit | (1.10%) | ||||||||||
Stock-based compensation | 8.00% | (0.30%) | |||||||||
Effective rate | 5.10% | (0.20%) | (1.00%) |
Income Taxes, Deferred Tax Asse
Income Taxes, Deferred Tax Assets and Liabilities (Details) - USD ($) $ in Thousands | Dec. 31, 2019 | Dec. 31, 2018 |
Deferred Tax Assets [Abstract] | ||
Net operating loss carryovers | $ 22,045 | $ 51,280 |
Tax credits | 27,801 | 31,768 |
Stock-based compensation | 7,825 | 11,812 |
Deferred revenue | 6,876 | |
Intangible and capital assets | 99,038 | 56,984 |
Other | 4,381 | 1,996 |
Total deferred tax assets | 161,090 | 160,716 |
Deferred Tax Liabilities [Abstract] | ||
Fixed assets | (798) | (811) |
Other | (2,512) | |
Total deferred tax liabilities | (3,310) | (811) |
Valuation allowance | $ (157,780) | $ (159,905) |
Income Taxes - Narrative (Detai
Income Taxes - Narrative (Details) - USD ($) | 12 Months Ended | ||
Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Investments Owned Federal Income Tax Note [Line Items] | |||
Unrecognized tax benefits that would impact effective tax rate, if recognized | $ 0 | ||
Interest and penalties on unrecognized tax benefits | 0 | $ 0 | $ 0 |
Deferred Tax Assets [Member] | |||
Investments Owned Federal Income Tax Note [Line Items] | |||
Decrease in valuation allowance | 2,100,000 | $ 2,100,000 | |
State [Member] | |||
Investments Owned Federal Income Tax Note [Line Items] | |||
Net operating loss carryforwards | 1,400,000 | ||
Federal [Member] | |||
Investments Owned Federal Income Tax Note [Line Items] | |||
Net operating loss carryforwards | 99,500,000 | ||
Federal [Member] | Research and Development [Member] | |||
Investments Owned Federal Income Tax Note [Line Items] | |||
Tax credit carryforwards | $ 33,600,000 |
Income Taxes, Gross Unrecognize
Income Taxes, Gross Unrecognized Tax Benefits (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Gross Unrecognized Tax Benefits [Roll Forward] | |||
Beginning balance of unrecognized tax benefits | $ 5,606 | $ 5,001 | $ 5,012 |
Additions related to prior year tax positions | 155 | ||
Additions related to the current year | 601 | 691 | 1,723 |
Decreases related to prior year tax positions | (549) | (86) | (1,734) |
Ending balance of unrecognized tax benefits | $ 5,813 | $ 5,606 | $ 5,001 |
Initial Public Offering (Detail
Initial Public Offering (Details) - USD ($) $ in Thousands | Jul. 19, 2017 | Dec. 31, 2017 |
Initial Public Offering [Abstract] | ||
Net proceeds from sale of common stock in IPO | $ 182,300 | |
Gross proceeds from sale of common stock in IPO | 132,300 | |
Investment by Ionis | 25,000 | |
Proceeds from sale of common stock to Novartis in a private placement | $ 50,000 | $ 50,000 |
Shares issued upon conversion of Series A Convertible Preferred Stock (in shares) | 28,884,540 | |
Principal and accrued interest from line of credit converted | $ 106,000 | |
Shares issued upon conversion of line of credit (in shares) | 13,438,339 | |
IPO [Member] | ||
Initial Public Offering [Abstract] | ||
Shares issued (in shares) | 17,968,750 | |
Private Placement [Member] | ||
Initial Public Offering [Abstract] | ||
Shares issued (in shares) | 6,250,000 |
Employment Benefits (Details)
Employment Benefits (Details) - USD ($) | 12 Months Ended | ||
Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Compensation And Retirement Disclosure [Abstract] | |||
Employee contribution limit per calendar year for employees under 50 years of age | $ 19,000 | ||
Employee contribution limit per calendar year for employees over 50 years of age | 25,000 | ||
Matching contributions | $ 2,100,000 | $ 1,600,000 | $ 300,000 |
Basic and Diluted Net Loss Pe_3
Basic and Diluted Net Loss Per Share - Narratives (Details) - shares | 12 Months Ended | |||
Dec. 31, 2019 | Dec. 31, 2018 | Jul. 31, 2017 | Dec. 31, 2015 | |
Earnings Per Share Basic [Line Items] | ||||
Common stock, shares issued (in shares) | 100,993,173 | 89,345,978 | 66,541,629 | |
Common stock, shares outstanding (in shares) | 100,993,173 | 89,345,978 | 66,541,629 | |
Series A Convertible Preferred Stock [Member] | ||||
Earnings Per Share Basic [Line Items] | ||||
Shares of Series A convertible preferred stock issued | 28,884,540 | |||
Shares of Series A convertible preferred stock outstanding | 0 | |||
Dividend rate | 6.00% |
Basic and Diluted Net Loss Pe_4
Basic and Diluted Net Loss Per Share (Details) - USD ($) $ / shares in Units, $ in Thousands | 3 Months Ended | 12 Months Ended | |||||||||
Dec. 31, 2019 | Sep. 30, 2019 | Jun. 30, 2019 | Mar. 31, 2019 | Dec. 31, 2018 | Sep. 30, 2018 | Jun. 30, 2018 | Mar. 31, 2018 | Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Basic and Diluted Net Loss Per Share [Abstract] | |||||||||||
Net income (loss) | $ 82,406 | $ (31,498) | $ (37,323) | $ 27,187 | $ (70,557) | $ (63,591) | $ (62,046) | $ (29,627) | $ 40,772 | $ (225,821) | $ (121,559) |
Preferred stock dividend | (20,100) | ||||||||||
Distribution to Ionis | (13,492) | (7,792) | |||||||||
Distributable income (losses) | $ 27,280 | (233,613) | (141,659) | ||||||||
Shares issuable related to our ESPP | 23,839 | ||||||||||
Weighted-average shares outstanding owned by others, plus assumed conversions | 25,282,273 | ||||||||||
Employee Stock Options [Member] | |||||||||||
Basic and Diluted Net Loss Per Share [Abstract] | |||||||||||
Weighted-average shares issuable | 2,413,624 | ||||||||||
Restricted Stock [Member] | |||||||||||
Basic and Diluted Net Loss Per Share [Abstract] | |||||||||||
Weighted-average shares issuable | 29,128 | ||||||||||
Preferred Stock [Member] | |||||||||||
Basic and Diluted Net Loss Per Share [Abstract] | |||||||||||
Net income (loss) | (28,385) | ||||||||||
Preferred stock dividend | $ (20,100) | ||||||||||
Weighted-average shares of common stock outstanding, basic | 15,748,009 | ||||||||||
Losses attributable to preferred shares | $ (48,485) | ||||||||||
Net income (loss) per share of common stock, basic | $ (1.80) | ||||||||||
Ionis [Member] | Common Stock [Member] | |||||||||||
Basic and Diluted Net Loss Per Share [Abstract] | |||||||||||
Net income (loss) | $ 34,073 | (163,938) | $ (63,638) | ||||||||
Distribution to Ionis | 13,492 | 7,792 | |||||||||
Distributable income (losses) | $ 20,581 | $ (171,730) | $ (63,638) | ||||||||
Weighted-average shares of common stock outstanding, basic | 71,341,992 | 70,221,338 | 70,221,338 | 68,581,967 | 67,129,553 | 65,538,467 | 60,832,494 | 45,447,879 | 70,099,576 | 59,812,394 | 20,669,446 |
Net income (loss) per share of common stock, basic | $ 0.87 | $ (0.34) | $ (0.40) | $ 0.35 | $ (0.79) | $ (0.73) | $ (0.72) | $ (0.44) | $ 0.49 | $ (2.74) | $ (3.08) |
Weighted-average shares of common stock outstanding, diluted | 71,341,992 | 70,221,338 | 70,221,338 | 68,581,967 | 67,129,553 | 65,538,467 | 60,832,494 | 45,447,879 | 70,099,576 | 59,812,394 | 20,669,446 |
Income allocated to Ionis | $ 20,049 | ||||||||||
Income available to Ionis | $ 33,541 | ||||||||||
Net income (loss) per share of common stock, diluted | $ 0.86 | $ (0.34) | $ (0.40) | $ 0.34 | $ (0.79) | $ (0.73) | $ (0.72) | $ (0.44) | $ 0.48 | $ (2.74) | $ (3.08) |
Others [Member] | Common Stock [Member] | |||||||||||
Basic and Diluted Net Loss Per Share [Abstract] | |||||||||||
Distributable income (losses) | $ 6,699 | $ (61,883) | $ (29,536) | ||||||||
Weighted-average shares of common stock outstanding, basic | 23,723,298 | 22,821,555 | 22,573,900 | 22,126,363 | 21,869,713 | 21,671,415 | 21,492,157 | 21,171,372 | 22,815,682 | 21,553,407 | 9,593,322 |
Net income (loss) per share of common stock, basic | $ 0.87 | $ (0.34) | $ (0.40) | $ 0.15 | $ (0.79) | $ (0.73) | $ (0.85) | $ (0.44) | $ 0.29 | $ (2.87) | $ (3.08) |
Weighted-average shares of common stock outstanding, diluted | 24,862,521 | 22,821,555 | 22,573,900 | 25,545,975 | 21,869,713 | 21,671,415 | 21,492,157 | 21,171,372 | 25,282,273 | 21,553,407 | 9,593,322 |
Weighted-average shares outstanding owned by others, plus assumed conversions | 25,282,273 | ||||||||||
Income allocated to Ionis | $ 7,231 | ||||||||||
Net income (loss) per share of common stock, diluted | $ 0.86 | $ (0.34) | $ (0.40) | $ 0.15 | $ (0.79) | $ (0.73) | $ (0.85) | $ (0.44) | $ 0.29 | $ (2.87) | $ (3.08) |
Contractual Obligations and C_3
Contractual Obligations and Commitments - Narratives (Details) $ in Millions | May 08, 2019 | Nov. 12, 2018ft² | Aug. 15, 2018USD ($) | Apr. 05, 2018USD ($)ft² | Dec. 31, 2019USD ($) | Dec. 31, 2018USD ($) | Dec. 31, 2017USD ($) |
Operating Leased Assets [Line Items] | |||||||
Operating lease expense | $ 2.1 | $ 2.4 | $ 0.7 | ||||
Agreement to purchase goods and services | $ 4.3 | ||||||
Dublin, Ireland [Member] | |||||||
Operating Leased Assets [Line Items] | |||||||
Lessee operating lease, lease commencement date | May 31, 2019 | ||||||
Initial term of lease | 18 months | ||||||
Operating lease, option to extend | a 12-month extension period in our right-of-use asset and lease liability | ||||||
Lessee, Operating Lease, Existence of Option to Extend | true | ||||||
Office Space for Corporate Headquarters [Member] | |||||||
Operating Leased Assets [Line Items] | |||||||
Lessee operating lease, lease commencement date | Apr. 5, 2018 | ||||||
Area of office space under lease agreement | ft² | 30,175 | ||||||
Initial term of lease | 123 months | ||||||
Operating lease, option to extend | option to extend the lease for an additional five-year term | ||||||
Term of lease extension | 5 years | ||||||
Period of free rent | 3 months | ||||||
Tenant improvement allowance | $ 3.8 | ||||||
Initial letter of credit to secure lease obligation | $ 2.4 | ||||||
Letter of credit to secure lease obligation on third anniversary of rent commencement date | 1.8 | ||||||
Letter of credit to secure lease obligation on fifth anniversary of rent commencement date | $ 1.2 | ||||||
Ionis Sublease [Member] | |||||||
Operating Leased Assets [Line Items] | |||||||
Area of office space under lease agreement | ft² | 4,723 | ||||||
Initial term of lease | 64 months | ||||||
Period of free rent | 4 months | ||||||
Lessee, Operating Lease, Existence of Option to Extend | false |
Contractual Obligations and C_4
Contractual Obligations and Commitments - Summary of Other Information related to Operating Leases (Details) $ in Thousands | Dec. 31, 2019USD ($) | |
Commitments And Contingencies Disclosure [Abstract] | ||
Operating lease right-of-use assets | $ 11,094 | |
Operating lease liabilities | $ 15,551 | [1] |
Weighted average remaining lease term | 8 years 8 months 12 days | |
Weighted average discount rate | 8.00% | |
[1] | Current portion of $1.4 million included in other current liabilities and remaining $14.2 million included in long-term portion of lease liabilities in our consolidated balance sheet. |
Contractual Obligations and C_5
Contractual Obligations and Commitments - Summary of Other Information related to Operating Leases (Parenthetical) (Details) - USD ($) $ in Thousands | Dec. 31, 2019 | Dec. 31, 2018 |
Commitments And Contingencies Disclosure [Abstract] | ||
Current portion of operating lease liabilities | $ 1,400 | |
Operating Lease, Liability, Current, Statement of Financial Position [Extensible List] | us-gaap:OtherLiabilitiesCurrent | |
Long-term portion of lease liabilities | $ 14,248 | $ 4,442 |
Contractual Obligations and C_6
Contractual Obligations and Commitments - Summary of Annual Maturities of Operating Lease Liabilities (Details) $ in Thousands | Dec. 31, 2019USD ($) | |
Commitments And Contingencies Disclosure [Abstract] | ||
2020 | $ 2,501 | |
2021 | 2,506 | |
2022 | 2,403 | |
2023 | 2,400 | |
2024 | 2,395 | |
Thereafter | 9,565 | |
Total minimum lease payments | 21,770 | |
Less: Imputed interest | (6,219) | |
Operating lease liabilities | $ 15,551 | [1] |
[1] | Current portion of $1.4 million included in other current liabilities and remaining $14.2 million included in long-term portion of lease liabilities in our consolidated balance sheet. |
Restructuring - Narratives (Det
Restructuring - Narratives (Details) - USD ($) | Sep. 06, 2018 | Dec. 31, 2018 | Dec. 31, 2019 |
Restructuring Costs [Abstract] | |||
Percentage of reduction in workforce | 12.00% | ||
Restructuring-related costs | $ 1,700,000 | ||
Non-cash stock option modification expenses | 400,000 | ||
Remaining restructuring liability | $ 43,000 | ||
Reorganization Plan [Member] | |||
Restructuring Costs [Abstract] | |||
Remaining restructuring liability | $ 0 |
Restructuring - Summary of Rest
Restructuring - Summary of Restructuring Costs By Category (Details) $ in Thousands | 12 Months Ended |
Dec. 31, 2018USD ($) | |
Restructuring Costs [Abstract] | |
Restructuring charges, Cash | $ 1,889 |
Restructuring charges, Adjustment | (150) |
Restructuring charges, Non-cash | 409 |
Restructuring charges, Total | 2,148 |
Research and Development Expenses [Member] | |
Restructuring Costs [Abstract] | |
Restructuring charges, Cash | 327 |
Restructuring charges, Adjustment | (34) |
Restructuring charges, Non-cash | 209 |
Restructuring charges, Total | 502 |
Selling, General and Administrative Expenses [Member] | |
Restructuring Costs [Abstract] | |
Restructuring charges, Cash | 1,562 |
Restructuring charges, Adjustment | (116) |
Restructuring charges, Non-cash | 200 |
Restructuring charges, Total | $ 1,646 |
Restructuring - Summary of Re_2
Restructuring - Summary of Restructuring Reserve Included in Accrued Compensation (Details) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2019 | Dec. 31, 2018 | |
Restructuring Reserve [Abstract] | ||
Restructuring reserve beginning balance | $ 43 | |
Restructuring-related costs | $ 1,889 | |
Adjustments during the period | (150) | |
Amounts paid during the period | $ (43) | (1,696) |
Restructuring reserve ending balance | $ 43 |
Quarterly Financial Data (Una_3
Quarterly Financial Data (Unaudited) (Details) - USD ($) $ / shares in Units, $ in Thousands | 3 Months Ended | 12 Months Ended | |||||||||
Dec. 31, 2019 | Sep. 30, 2019 | Jun. 30, 2019 | Mar. 31, 2019 | Dec. 31, 2018 | Sep. 30, 2018 | Jun. 30, 2018 | Mar. 31, 2018 | Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Revenue: | |||||||||||
Revenue | $ 277,480 | $ 20,624 | $ 26,623 | $ 163,816 | $ 10,197 | $ 19,241 | $ 18,321 | $ 17,108 | $ 488,543 | $ 64,867 | $ 43,401 |
Expenses: | |||||||||||
Cost of sales - intangible asset amortization | 1,434 | 1,434 | 1,419 | 1,403 | 2,012 | 701 | 5,690 | 2,713 | 0 | ||
Research and development | 148,165 | 24,797 | 20,271 | 99,619 | 33,532 | 29,381 | 39,457 | 27,970 | 292,852 | 130,340 | 126,890 |
Selling, general and administrative | 51,434 | 34,905 | 50,740 | 44,602 | 45,934 | 45,924 | 42,287 | 19,465 | 181,681 | 153,610 | 36,981 |
Total expenses | 194,317 | 53,214 | 65,329 | 137,609 | 82,255 | 84,249 | 81,744 | 47,435 | 450,469 | 295,683 | 163,871 |
Income (loss) from operations | 83,163 | (32,590) | (38,706) | 26,207 | (72,058) | (65,008) | (63,423) | (30,327) | 38,074 | (230,816) | (120,470) |
Other income (expense): | |||||||||||
Investment income | 1,223 | 1,487 | 1,571 | 1,224 | 1,542 | 1,675 | 1,546 | 868 | 5,505 | 5,631 | 1,813 |
Other (expense) income | (339) | (136) | (28) | (112) | (41) | (25) | 45 | (168) | (615) | (189) | 104 |
Income (loss) before income tax expense | 84,047 | (31,239) | (37,163) | 27,319 | (70,557) | (63,358) | (61,832) | (29,627) | 42,964 | (225,374) | (120,284) |
Income tax expense | (1,641) | (259) | (160) | (132) | (233) | (214) | (2,192) | (447) | (1,275) | ||
Net income (loss) | 82,406 | (31,498) | (37,323) | 27,187 | $ (70,557) | $ (63,591) | $ (62,046) | $ (29,627) | 40,772 | (225,821) | (121,559) |
Ionis [Member] | |||||||||||
Expenses: | |||||||||||
Net loss share from commercial activities under arrangement with Ionis Pharmaceuticals, Inc. | $ (10,313) | $ (8,889) | $ (11,465) | $ (9,056) | (39,723) | 0 | 0 | ||||
Common Stock [Member] | Ionis [Member] | |||||||||||
Other income (expense): | |||||||||||
Net income (loss) | $ 34,073 | $ (163,938) | $ (63,638) | ||||||||
Net income (loss) per share of common stock, basic | $ 0.87 | $ (0.34) | $ (0.40) | $ 0.35 | $ (0.79) | $ (0.73) | $ (0.72) | $ (0.44) | $ 0.49 | $ (2.74) | $ (3.08) |
Weighted-average shares of common stock outstanding, basic | 71,341,992 | 70,221,338 | 70,221,338 | 68,581,967 | 67,129,553 | 65,538,467 | 60,832,494 | 45,447,879 | 70,099,576 | 59,812,394 | 20,669,446 |
Net income (loss) per share of common stock, diluted | $ 0.86 | $ (0.34) | $ (0.40) | $ 0.34 | $ (0.79) | $ (0.73) | $ (0.72) | $ (0.44) | $ 0.48 | $ (2.74) | $ (3.08) |
Weighted-average shares of common stock outstanding, diluted | 71,341,992 | 70,221,338 | 70,221,338 | 68,581,967 | 67,129,553 | 65,538,467 | 60,832,494 | 45,447,879 | 70,099,576 | 59,812,394 | 20,669,446 |
Common Stock [Member] | Others [Member] | |||||||||||
Other income (expense): | |||||||||||
Net income (loss) per share of common stock, basic | $ 0.87 | $ (0.34) | $ (0.40) | $ 0.15 | $ (0.79) | $ (0.73) | $ (0.85) | $ (0.44) | $ 0.29 | $ (2.87) | $ (3.08) |
Weighted-average shares of common stock outstanding, basic | 23,723,298 | 22,821,555 | 22,573,900 | 22,126,363 | 21,869,713 | 21,671,415 | 21,492,157 | 21,171,372 | 22,815,682 | 21,553,407 | 9,593,322 |
Net income (loss) per share of common stock, diluted | $ 0.86 | $ (0.34) | $ (0.40) | $ 0.15 | $ (0.79) | $ (0.73) | $ (0.85) | $ (0.44) | $ 0.29 | $ (2.87) | $ (3.08) |
Weighted-average shares of common stock outstanding, diluted | 24,862,521 | 22,821,555 | 22,573,900 | 25,545,975 | 21,869,713 | 21,671,415 | 21,492,157 | 21,171,372 | 25,282,273 | 21,553,407 | 9,593,322 |
Product [Member] | |||||||||||
Revenue: | |||||||||||
Revenue | $ 13,690 | $ 11,944 | $ 9,865 | $ 6,754 | $ 2,237 | $ 42,253 | $ 2,237 | $ 0 | |||
Expenses: | |||||||||||
Cost of product/license | 1,197 | 967 | 1,364 | 1,041 | 777 | $ 1,043 | 4,569 | 1,820 | 0 | ||
Licensing [Member] | |||||||||||
Revenue: | |||||||||||
Revenue | 4,000 | 136 | 6,036 | 12,000 | 10,172 | 12,000 | 0 | ||||
Expenses: | |||||||||||
Cost of product/license | 2,400 | 3,000 | 7,200 | 5,400 | 7,200 | 0 | |||||
Research and Development Revenue Under Collaborative Agreements [Member] | |||||||||||
Revenue: | |||||||||||
Revenue | $ 259,790 | $ 8,544 | $ 10,722 | $ 157,062 | $ 7,960 | $ 7,241 | $ 18,321 | $ 17,108 | $ 436,118 | $ 50,630 | $ 43,401 |