Document and Entity Information
Document and Entity Information - USD ($) $ in Millions | 12 Months Ended | ||
Dec. 31, 2018 | Feb. 20, 2019 | Jun. 30, 2018 | |
Document And Entity Information [Abstract] | |||
Entity Registrant Name | AKCEA THERAPEUTICS, INC. | ||
Entity Central Index Key | 1,662,524 | ||
Document Type | 10-K | ||
Document Period End Date | Dec. 31, 2018 | ||
Amendment Flag | false | ||
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 | $ 209.3 | ||
Entity Common Stock, Shares Outstanding | 89,433,187 | ||
Document Fiscal Year Focus | 2,018 | ||
Document Fiscal Period Focus | FY | ||
Entity Small Business | false | ||
Entity Emerging Growth Company | true | ||
Entity Ex Transition Period | true | ||
Entity Shell Company | false |
CONSOLIDATED BALANCE SHEETS
CONSOLIDATED BALANCE SHEETS - USD ($) $ in Thousands | Dec. 31, 2018 | Dec. 31, 2017 |
Current assets: | ||
Cash and cash equivalents | $ 86,454 | $ 58,367 |
Short-term investments | 166,155 | 201,763 |
Accounts receivable | 4,597 | 5,413 |
Other current assets | 10,029 | 1,302 |
Total current assets | 267,235 | 266,845 |
Property, plant and equipment, net | 5,696 | 77 |
Licenses, net | 88,914 | 1,221 |
Deposits and other assets | 3,416 | 661 |
Total assets | 365,261 | 268,804 |
Current liabilities: | ||
Accounts payable | 12,068 | 2,381 |
Payable to Ionis Pharmaceuticals, Inc. | 18,901 | 14,365 |
Accrued compensation | 8,583 | 4,083 |
Accrued liabilities | 14,787 | 7,570 |
Current portion of deferred revenue | 25,354 | 58,192 |
Other current liabilities | 968 | 1,875 |
Total current liabilities | 80,661 | 88,466 |
Long-term portion of deferred rent | 4,442 | 12 |
Long-term portion of deferred revenue | 3,434 | 12,501 |
Total liabilities | 88,537 | 100,979 |
Stockholders’ equity: | ||
Common stock, $0.001 par value; 125,000,000 and 100,000,000 shares authorized, 89,345,978 and 66,541,629 shares issued and outstanding at December 31, 2018 and 2017, respectively. | 89 | 67 |
Additional paid-in capital | 799,001 | 464,430 |
Accumulated other comprehensive loss | (324) | (451) |
Accumulated deficit | (522,042) | (296,221) |
Total stockholders’ equity | 276,724 | 167,825 |
Total liabilities and stockholders’ equity | $ 365,261 | $ 268,804 |
CONSOLIDATED BALANCE SHEETS (Pa
CONSOLIDATED BALANCE SHEETS (Parenthetical) - $ / shares | Dec. 31, 2018 | Dec. 31, 2017 |
Stockholders’ equity: | ||
Common stock, par value (in dollars per share) | $ 0.001 | $ 0.001 |
Common stock, shares authorized (in shares) | 125,000,000 | 100,000,000 |
Common stock, shares issued (in shares) | 89,345,978 | 66,541,629 |
Common stock, shares outstanding (in shares) | 89,345,978 | 66,541,629 |
CONSOLIDATED STATEMENTS OF OPER
CONSOLIDATED STATEMENTS OF OPERATIONS - USD ($) | 3 Months Ended | 12 Months Ended | |||||||||
Dec. 31, 2018 | Sep. 30, 2018 | Jun. 30, 2018 | Mar. 31, 2018 | Dec. 31, 2017 | Sep. 30, 2017 | Jun. 30, 2017 | Mar. 31, 2017 | Dec. 31, 2018 | Dec. 31, 2017 | Dec. 31, 2016 | |
Revenue: | |||||||||||
Revenue | $ 10,197,000 | $ 19,241,000 | $ 18,321,000 | $ 17,108,000 | $ 21,688,000 | $ 9,906,000 | $ 5,713,000 | $ 6,094,000 | $ 64,867,000 | $ 43,401,000 | $ 0 |
Expenses: | |||||||||||
Cost of sales - intangible asset amortization | 2,012,000 | 701,000 | 2,713,000 | 0 | 0 | ||||||
Research and development | 33,532,000 | 29,381,000 | 39,457,000 | 27,970,000 | 25,968,000 | 17,640,000 | 18,487,000 | 64,794,000 | 130,340,000 | 126,890,000 | 68,459,000 |
Selling, general and administrative | 45,934,000 | 45,924,000 | 42,287,000 | 19,465,000 | 17,018,000 | 8,373,000 | 6,915,000 | 4,676,000 | 153,610,000 | 36,981,000 | 15,053,000 |
Total expenses | 82,255,000 | 84,249,000 | 81,744,000 | 47,435,000 | 42,986,000 | 26,013,000 | 25,402,000 | 69,470,000 | 295,683,000 | 163,871,000 | 83,512,000 |
Loss from operations | (72,058,000) | (65,008,000) | (63,423,000) | (30,327,000) | (21,298,000) | (16,107,000) | (19,689,000) | (63,376,000) | (230,816,000) | (120,470,000) | (83,512,000) |
Other income (expense): | |||||||||||
Investment income | 1,542,000 | 1,675,000 | 1,546,000 | 868,000 | 819,000 | 687,000 | 245,000 | 61,000 | 5,631,000 | 1,813,000 | 295,000 |
Interest expense | (224,000) | (965,000) | (541,000) | 0 | (1,731,000) | 0 | |||||
Other income (expense) | (41,000) | (25,000) | 45,000 | (168,000) | (19,000) | 73,000 | 50,000 | (189,000) | 104,000 | 0 | |
Loss before income tax expense | (70,557,000) | (63,358,000) | (61,832,000) | (29,627,000) | (20,498,000) | (15,571,000) | (20,359,000) | (63,856,000) | (225,374,000) | (120,284,000) | (83,217,000) |
Income tax expense | (233,000) | (214,000) | 791,000 | (2,066,000) | (447,000) | (1,275,000) | 0 | ||||
Net loss | $ (70,557,000) | $ (63,591,000) | $ (62,046,000) | $ (29,627,000) | $ (19,707,000) | (17,637,000) | $ (20,359,000) | $ (63,856,000) | (225,821,000) | (121,559,000) | (83,217,000) |
Preferred Stock [Member] | |||||||||||
Other income (expense): | |||||||||||
Net loss | $ (64,000) | $ 0 | $ (28,385,000) | $ (83,217,000) | |||||||
Net loss per share, basic and diluted (in dollars per share) | $ (0.01) | $ (0.70) | $ (2.21) | $ 0 | $ (1.80) | $ (2.88) | |||||
Weighted-average shares outstanding, basic and diluted (in shares) | 5,651,323 | 28,888,450 | 28,884,540 | 0 | 15,748,009 | 28,884,540 | |||||
Common Stock [Member] | |||||||||||
Other income (expense): | |||||||||||
Net loss | $ (17,573,000) | ||||||||||
Common Stock [Member] | Ionis [Member] | |||||||||||
Other income (expense): | |||||||||||
Net loss | $ (163,938,000) | $ (63,638,000) | $ 0 | ||||||||
Net loss per share, basic and diluted (in dollars per share) | $ (0.79) | $ (0.73) | $ (0.72) | $ (0.44) | $ (0.30) | $ (0.33) | $ (2.74) | $ (3.08) | $ 0 | ||
Weighted-average shares outstanding, basic and diluted (in shares) | 67,129,553 | 65,538,467 | 60,832,494 | 45,447,879 | 45,447,879 | 36,555,903 | 59,812,394 | 20,669,446 | 0 | ||
Common Stock [Member] | Others [Member] | |||||||||||
Other income (expense): | |||||||||||
Net loss per share, basic and diluted (in dollars per share) | $ (0.79) | $ (0.73) | $ (0.85) | $ (0.44) | $ (0.30) | $ (0.33) | $ (2.87) | $ (3.08) | $ 0 | ||
Weighted-average shares outstanding, basic and diluted (in shares) | 21,869,713 | 21,671,415 | 21,492,157 | 21,171,372 | 21,093,750 | 16,966,712 | 21,553,407 | 9,593,322 | 0 | ||
Product [Member] | |||||||||||
Revenue: | |||||||||||
Revenue | $ 2,237,000 | $ 2,237,000 | $ 0 | $ 0 | |||||||
Expenses: | |||||||||||
Cost of product/license | 777,000 | $ 1,043,000 | 1,820,000 | 0 | 0 | ||||||
Licensing [Member] | |||||||||||
Revenue: | |||||||||||
Revenue | 12,000,000 | 12,000,000 | 0 | 0 | |||||||
Expenses: | |||||||||||
Cost of product/license | 7,200,000 | 7,200,000 | 0 | 0 | |||||||
Commercial Revenue [Member] | |||||||||||
Revenue: | |||||||||||
Revenue | 2,237,000 | 12,000,000 | 14,237,000 | 0 | 0 | ||||||
Research and Development Revenue Under Collaborative Agreement [Member] | |||||||||||
Revenue: | |||||||||||
Revenue | $ 7,960,000 | $ 7,241,000 | $ 18,321,000 | $ 17,108,000 | $ 21,688,000 | $ 9,906,000 | $ 5,713,000 | $ 6,094,000 | $ 50,630,000 | $ 43,401,000 | $ 0 |
CONSOLIDATED STATEMENTS OF COMP
CONSOLIDATED STATEMENTS OF COMPREHENSIVE LOSS - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2018 | Dec. 31, 2017 | Dec. 31, 2016 | |
Statement Of Income And Comprehensive Income [Abstract] | |||
Net loss | $ (225,821) | $ (121,559) | $ (83,217) |
Unrealized gains (losses) on investments, net of tax | 144 | (337) | 75 |
Currency translation adjustment | (17) | (93) | (21) |
Comprehensive loss | $ (225,694) | $ (121,989) | $ (83,163) |
CONSOLIDATED STATEMENTS OF STOC
CONSOLIDATED STATEMENTS OF STOCKHOLDERS' EQUITY - USD ($) shares in Thousands, $ in Thousands | Total | Ionis [Member] | Ionis [Member]TEGSEDI [Member] | Ionis [Member]TTR License Agreement [Member] | Convertible Preferred Stock [Member] | Convertible Preferred Stock [Member]Ionis [Member] | Convertible Preferred Stock [Member]Ionis [Member]TEGSEDI [Member] | Convertible Preferred Stock [Member]Ionis [Member]TTR License Agreement [Member] | Common Stock [Member] | Common Stock [Member]Ionis [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 Other Comprehensive Loss [Member]Ionis [Member] | Accumulated Other Comprehensive Loss [Member]Ionis [Member]TEGSEDI [Member] | Accumulated Other Comprehensive Loss [Member]Ionis [Member]TTR License Agreement [Member] | Accumulated Deficit [Member] | Accumulated Deficit [Member]Ionis [Member] | Accumulated Deficit [Member]Ionis [Member]TEGSEDI [Member] | Accumulated Deficit [Member]Ionis [Member]TTR License Agreement [Member] |
Balance at Dec. 31, 2015 | $ 55,267 | $ 100,000 | $ 0 | $ 46,787 | $ (75) | $ (91,445) | ||||||||||||||||||
Balance (in shares) at Dec. 31, 2015 | 28,885 | 0 | ||||||||||||||||||||||
Net loss | (83,217) | $ 0 | $ 0 | 0 | 0 | (83,217) | ||||||||||||||||||
Change in unrealized gains (losses), net of tax | 75 | 0 | 0 | 0 | 75 | 0 | ||||||||||||||||||
Currency translation adjustment | (21) | 0 | 0 | 0 | (21) | 0 | ||||||||||||||||||
Stock-based compensation expense | 10,149 | 0 | 0 | 10,149 | 0 | 0 | ||||||||||||||||||
Balance at Dec. 31, 2016 | (17,747) | $ 100,000 | $ 0 | 56,936 | (21) | (174,662) | ||||||||||||||||||
Balance (in shares) at Dec. 31, 2016 | 28,885 | 0 | ||||||||||||||||||||||
Net loss | (121,559) | $ 0 | $ 0 | 0 | 0 | (121,559) | ||||||||||||||||||
Change in unrealized gains (losses), net of tax | (337) | 0 | 0 | 0 | (337) | 0 | ||||||||||||||||||
Currency translation adjustment | (93) | 0 | 0 | 0 | (93) | 0 | ||||||||||||||||||
Conversion of convertible preferred stock to common stock | 0 | $ (100,000) | $ 29 | 99,971 | 0 | 0 | ||||||||||||||||||
Conversion of convertible preferred stock to common stock (in shares) | (28,885) | 28,885 | ||||||||||||||||||||||
Issuance of stock | 132,291 | $ 0 | $ 18 | 132,273 | 0 | 0 | ||||||||||||||||||
Issuance of stock (in shares) | 0 | 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 | $ 0 | $ 14 | 107,717 | 0 | 0 | ||||||||||||||||||
Issuance of common stock in connection with the conversion of the line of credit with Ionis Pharmaceuticals Inc. together with accrued interest (in shares) | 0 | 13,438 | ||||||||||||||||||||||
Issuance of common stock in connection with private placement | 50,000 | $ 0 | $ 6 | 49,994 | 0 | 0 | ||||||||||||||||||
Issuance of common stock in connection with private placement (in shares) | 0 | 6,250 | ||||||||||||||||||||||
Stock-based compensation expense | 17,539 | $ 0 | $ 0 | 17,539 | 0 | 0 | ||||||||||||||||||
Balance at Dec. 31, 2017 | 167,825 | $ 0 | $ 67 | 464,430 | (451) | (296,221) | ||||||||||||||||||
Balance (in shares) at Dec. 31, 2017 | 0 | 66,542 | ||||||||||||||||||||||
Net loss | (225,821) | $ 0 | $ 0 | 0 | 0 | (225,821) | ||||||||||||||||||
Change in unrealized gains (losses), net of tax | 144 | 0 | 0 | 0 | 144 | 0 | ||||||||||||||||||
Currency translation adjustment | (17) | 0 | 0 | 0 | (17) | 0 | ||||||||||||||||||
Exercise of common stock options | 6,622 | $ 0 | $ 1 | 6,621 | 0 | 0 | ||||||||||||||||||
Exercise of common stock options (in shares) | 0 | 831 | ||||||||||||||||||||||
Issuance of common stock in connection with employee stock purchase plan | 341 | $ 0 | $ 0 | 341 | 0 | 0 | ||||||||||||||||||
Issuance of common stock in connection with employee stock purchase plan (in shares) | 0 | 32 | ||||||||||||||||||||||
Issuance of restricted common stock | 0 | $ 0 | $ 0 | 0 | 0 | 0 | ||||||||||||||||||
Issuance of restricted common stock (in shares) | 0 | 5 | ||||||||||||||||||||||
Issuance of stock | $ 90,000 | $ 200,112 | $ 0 | $ 0 | $ 3 | $ 18 | $ 89,997 | $ 200,094 | $ 0 | $ 0 | $ 0 | $ 0 | ||||||||||||
Issuance of stock (in shares) | 0 | 0 | 3,269 | 18,667 | ||||||||||||||||||||
Stock-based compensation expense | 44,282 | $ 0 | $ 0 | 44,282 | 0 | 0 | ||||||||||||||||||
Distribution to Ionis in connection with the TTR license transaction | $ (7,792) | $ 0 | $ 0 | $ (7,792) | $ 0 | $ 0 | ||||||||||||||||||
Capital contribution from Ionis | $ 1,028 | $ 0 | $ 0 | $ 1,028 | $ 0 | $ 0 | ||||||||||||||||||
Balance at Dec. 31, 2018 | $ 276,724 | $ 0 | $ 89 | $ 799,001 | $ (324) | $ (522,042) | ||||||||||||||||||
Balance (in shares) at Dec. 31, 2018 | 0 | 89,346 |
CONSOLIDATED STATEMENTS OF CASH
CONSOLIDATED STATEMENTS OF CASH FLOWS - USD ($) | 12 Months Ended | ||
Dec. 31, 2018 | Dec. 31, 2017 | Dec. 31, 2016 | |
Operating activities: | |||
Net loss | $ (225,821,000) | $ (121,559,000) | $ (83,217,000) |
Adjustments to reconcile net loss to net cash used in operating activities: | |||
Depreciation | 307,000 | 108,000 | 12,000 |
Amortization of licenses | 2,870,000 | 120,000 | 119,000 |
Amortization of discount/premium on investment securities, net | (23,000) | 499,000 | 170,000 |
Non-cash interest expense for line of credit with Ionis Pharmaceuticals, Inc. | 1,731,000 | ||
Non-cash sublicensing expense | 33,394,000 | ||
Stock-based compensation expense | 44,282,000 | 17,539,000 | 10,149,000 |
Changes in operating assets and liabilities: | |||
Accounts receivable | 816,000 | (5,413,000) | |
Other current and long-term assets | (8,765,000) | (1,761,000) | 64,000 |
Accounts payable | 8,444,000 | 1,905,000 | (54,000) |
Payable to Ionis Pharmaceuticals, Inc. | 4,527,000 | (43,385,000) | 15,157,000 |
Accrued compensation | 4,500,000 | 1,578,000 | 1,582,000 |
Deferred rent | 1,203,000 | (15,000) | 20,000 |
Accrued liabilities | 7,217,000 | 6,587,000 | 637,000 |
Income taxes payable | (540,000) | 1,789,000 | |
Deferred revenue | (41,905,000) | 70,693,000 | |
Net cash used in operating activities | (202,888,000) | (36,190,000) | (55,361,000) |
Investing activities: | |||
Purchases of short-term investments | (136,895,000) | (301,377,000) | (16,638,000) |
Proceeds from sale of short-term investments | 208,559,000 | 98,778,000 | 51,464,000 |
Purchase of property, plant and equipment | (1,119,000) | (9,000) | (179,000) |
Net cash provided by (used in) investing activities | 70,545,000 | (202,608,000) | 34,647,000 |
Financing activities: | |||
Proceeds from exercise of common stock options and employee stock purchase plan issuances | 6,963,000 | ||
Proceeds from issuance of common stock, net of underwriters' discount | 135,438,000 | ||
Proceeds from sale of common stock to Novartis in private placement | 50,000,000 | ||
Proceeds from line of credit from Ionis Pharmaceuticals, Inc. | 106,000,000 | ||
Net proceeds from issuance of common stock to Ionis in connection with TTR License Agreement | 155,868,000 | ||
Offering costs paid | (2,037,000) | (818,000) | |
Net cash provided by (used in) financing activities | 162,831,000 | 289,401,000 | (818,000) |
Effect of exchange rates on cash | (17,000) | (93,000) | |
Net increase (decrease) in cash and cash equivalents | 30,471,000 | 50,510,000 | (21,532,000) |
Cash, cash equivalents and restricted cash at beginning of period | 58,367,000 | 7,857,000 | 29,389,000 |
Cash, cash equivalents and restricted cash at end of period | 88,838,000 | 58,367,000 | 7,857,000 |
Supplemental disclosures of non-cash investing and financing activities: | |||
Unpaid deferred offering costs | 291,000 | ||
Conversion of preferred stock to common stock upon initial public offering | 100,000,000 | ||
Conversion of line of credit from Ionis Pharmaceuticals, Inc. into common stock | 107,731,000 | ||
Purchase of property, plant and equipment included in accounts payable | 1,252,000 | ||
Purchase of property, plant and equipment included in long-term deferred rent liability | 3,555,000 | ||
Acquisition of research and development licenses and milestone payments | 90,563,000 | ||
Cash, cash equivalents and restricted cash reported within the condensed consolidated balance sheets: | |||
Cash and cash equivalents | 86,454,000 | 58,367,000 | 7,857,000 |
Restricted cash included in deposits and other assets | 2,384,000 | ||
Cash, cash equivalents and restricted cash at end of period | 88,838,000 | $ 58,367,000 | $ 7,857,000 |
Ionis [Member] | |||
Supplemental disclosures of non-cash investing and financing activities: | |||
Capital contribution from Ionis | $ 1,028,000 |
Organization and Basis of Prese
Organization and Basis of Presentation | 12 Months Ended |
Dec. 31, 2018 | |
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 drugs to treat patients with rare and serious diseases. On July 19, 2017, we completed our initial public offering, or IPO. As of December 31, 2018, Ionis owned approximately 75% 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. See Note 2, Summary of Significant Accounting Policies 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, 2018, 2017 and 2016. In accordance with Accounting Standard Codification, or ASC, 205-40, Going Concern Rx |
Summary of Significant Accounti
Summary of Significant Accounting Policies | 12 Months Ended |
Dec. 31, 2018 | |
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 and prior to our IPO, the valuation of common stock. 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 as a component of accumulated other comprehensive loss within the consolidated statements of comprehensive loss. 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 receivables. 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 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 (expense) on our consolidated statement of operations. We use the specific identification method to determine the cost of securities sold. 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. For TEGSEDI inventory related costs incurred subsequent to July 1, 2018, we reflected these amounts as inventory on our consolidated balance sheets at the lower of cost or market 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 statements 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. At December 31, 2018 the amount of finished goods recorded in our consolidated balance sheets in other current assets related to our approved product TEGSEDI was $85,000. Property and Equipment Property and equipment are recorded at cost and depreciated over their estimated useful lives using the straight-line method over the estimated useful life of each asset. Furniture and fixtures are depreciated over seven 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. Intangible Assets We obtained exclusive licenses from Ionis for specific patents that Ionis owns and maintains related to our drug 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 license 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. Amortization expense is recorded as a component of cost of sales to the extent the underlying license is commercialized or research and development prior to its commercialization in the consolidated statements of operations. 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 . Revenue Recognition Collaboration and License Revenue In May 2014, the Financial Accounting Standards Board, or FASB, issued Accounting Standards Update, or ASU, No. 2014-09, which amended the guidance for accounting for revenue from contracts with customers. This ASU superseded the revenue recognition requirements in ASC Topic 605, Revenue Recognition Revenue from Contracts with Customers 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 drug 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 not concluded that the option to license a drug or the options for additional goods or services that may be requested in the future under our collaboration agreement are 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 drug 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, 2018, we have three revenue streams: our strategic collaboration, option and license agreement, or collaboration agreement, with Novartis Pharma AG, or Novartis, which we entered into in January 2017, our collaboration and license agreement with PTC Therapeutics International Limited, or PTC Therapeutics, which we entered into in August 2018 and commercial product revenue related TEGESDI sales subsequent to product launch in the fourth quarter of 2018. For a complete discussion of the accounting for our revenue streams, see Note 6, Strategic Collaboration with Novartis, Collaboration and License Agreement with PTC Therapeutics Summary of Significant Accounting Policies Effective January 1, 2018, we adopted Topic 606 using the full retrospective transition method. Under this method, we revised our consolidated financial statements for prior period amounts including the periods included in this Report on Form 10-K, as if Topic 606 had been effective for such periods. The references “as revised” used herein refer to revisions of amounts originally reported for the year ended December 31, 2017 and as of December 31, 2017 as Impact of Adoption As a result of adopting Topic 606 on January 1, 2018, we revised our comparative financial statements for the prior years as if Topic 606 had been effective for that period. On September 18, 2018, we filed a Current Report on Form 8-K to present recast consolidated financial statements for each of the three years ended December 31, 2015, 2016 and 2017, to reflect our adoption of the new accounting standard for revenue recognition set forth in Topic 606. The financial information recast in the Form 8-K was originally filed with the SEC in our Annual Report on Form 10-K for the fiscal year ended December 31, 2017, which was filed with the SEC on February 28, 2018. Under Topic 605, we recognized revenue from our collaboration with Novartis over time on a straight-line basis. Under Topic 606, we recognize revenue from our collaboration with Novartis using the input method based on the total cost of performing services over time. As a result, the following financial statement line items for fiscal year 2017 were affected. Consolidated Balance Sheets December 31, 2017 (in thousands) As Revised Under Topic 606 As Originally Reported Under Topic 605 Effect of Change Current portion of deferred revenue $ 58,192 $ 50,579 $ 7,613 Long-term portion of deferred revenue 12,501 8,306 4,195 Accumulated deficit $ (296,221 ) $ (284,413 ) $ (11,808 ) Consolidated Statements of Operations and Comprehensive Loss Year Ended December 31, 2017 (in thousands, except per share data) As Revised Under Topic 606 As Originally Reported Under Topic 605 Effect of Change Research and development revenue under collaborative Agreement $ 43,401 $ 55,209 $ (11,808 ) Loss from operations (120,470 ) (108,662 ) (11,808 ) Net loss (121,559 ) (109,751 ) (11,808 ) Net loss per share of preferred stock, basic and diluted (1.80 ) (1.55 ) (0.25 ) Net loss per share of common stock owned by Ionis, basic and diluted (3.08 ) (2.82 ) (0.26 ) Net loss per share of common stock owned by others, basic and diluted $ (3.08 ) (2.82 ) $ (0.26 ) Consolidated Statement of Cash Flows Years Ended December 31, 2017 (in thousands) As Revised Under Topic 606 As Originally Reported Under Topic 605 Effect of Change Net loss $ (121,559 ) $ (109,751 ) $ (11,808 ) Adjustments to reconcile net loss to net cash used in operating activities: Deferred revenue 70,693 58,885 11,808 Cash, cash equivalents and restricted cash at beginning of period 7,857 7,857 — Cash, cash equivalents and restricted at end of period $ 58,367 $ 58,367 $ — Product Revenue, Net Subsequent to regulatory approval in Europe on July 11, 2018 and FDA approval in the U.S. on October 5, 2018, in the fourth quarter of 2018 we began to sell TEGSEDI in the U.S. and Germany. In the U.S., the product is distributed through an exclusive distribution agreement with a third-party logistics (3PL) company 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. In Germany, the product is distributed through a non-exclusive distribution model with a 3PL that takes title to the product and currently represents our sole customer in Germany. Our customer in Germany then distributes TEGSEDI to hospitals and pharmacies in Germany. Revenue from product sales are recognized when the customer obtains control of our product, which occurs at a point in time, upon transfer of title to the customer. We record shipping and handling costs 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 in our consolidated statements of operations. Otherwise 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. Taxes collected from customers relating to product sales and remitted to governmental authorities are excluded from revenue. 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. We expense incremental costs of obtaining a contract as and when incurred since the expected amortization period of the asset that we would have recognized is one year or less. Reserves for Variable Consideration Revenues from product sales are recorded at the net sales price (transaction price), which includes estimates of variable consideration for which reserves are established and which result from 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. These reserves are based on the amounts earned or to be claimed on the related sales and are classified as reductions of accounts receivable (if the amount is payable to the customer) or a current liability (if the amount is payable to a party other than a customer). 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, these reserves reflect our best estimates 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 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. Government rebates: We are subject to discount obligations under government programs, including Medicaid programs and Medicare in the United States. We estimate Medicaid and Medicare rebates based upon a range of possible outcomes that are probability-weighted for the 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 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. In Germany, pharmaceutical companies must grant a specified rebate percentage to the German government. We have included this rebate as a reduction of revenue in the period the related product revenue is recognized. Trade discounts and allowances: We provide customary invoice discounts on TEGSEDI sales to our U.S. customer for prompt payment that are recorded as a reduction of revenue in the period the related product revenue is recognized. In addition, we receive and pay for various distribution services from our U.S. customer and wholesalers in the U.S. distribution channel. For services that are either not distinct from the sale of our product or for which we cannot reasonably estimate the fair value, such fees are classified as a reduction of product revenue. Product Returns: Our U.S. customer has return rights and the wholesalers 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 in the period the related product revenue is recognized. Based on the distribution model for TEGSEDI, contractual inventory limits with our customer and wholesalers and the price of TEGSEDI, we believe there will be minimal returns. Our customer in Germany only takes title to the product once it receives an order from a hospital or pharmacy and therefore does not maintain any inventory of TEGSEDI. Therefore, there is limited return risk and the Company has not recorded any return estimate in the transaction price for TEGSEDI sold in Germany. Other incentives: In the U.S., other incentives include co-payment assistance 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-pay 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. The estimate is recorded as a reduction of revenue in the same period the related revenue is recognized. During the year ended December 31, 2018, we recorded product revenue, net, of $2.2 million, which consist of $1.2 million of TEGSEDI sales in the U.S., and $1.0 million of TEGSEDI sales in Germany. The following table summarizes balances and activity in each of the product revenue allowance and reserve categories for the year ended December 31, 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 Cost of Product Sales As a result of receiving marketing authorization, or MA, approval for TEGSEDI from the European Commission, or EC, in July 2018, we began recording all TEGSEDI related expenses as cost of product sales starting in July 2018. Cost of product sales consists of manufacturing costs, transportation and freight, and indirect overhead costs associated with the manufacturing and distribution of TEGSEDI. Cost of product sales may also include period costs related to certain manufacturing services and inventory adjustment charges. Additionally, we expensed a significant portion of the cost of producing TEGSEDI that we will use in the commercial launch as research and development expense prior to the regulatory approval of TEGSEDI. Commercial Sublicensing Expenses We incur sublicense expenses under our TTR Development, Commercialization, Collaboration and License Agreement with Ionis related to the drugs we have licensed under the agreement. We include our sublicense fee expenses in our cost of license expenses on our consolidated statements of operations for those drugs 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 and services agreement with Ionis related to the drugs we have licensed under the agreement. We include our sublicense fee expenses in our research and development expenses on our consolidated statements of operations since the applicable drugs 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, laboratory and analysis costs, toxicology studies and investigator grants. We have drugs 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 service period in our consolidated statements 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 estimated 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. The RSUs we have granted vest annually over a four-year period. 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 options and Akcea options 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. In 2017, we no longer concurrently granted 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 will continue to recognize expense based on the valuation that was determined upon the grant date for options issued in 2016 or 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 9, Equity and Stock-based Compensation, Accumulated Other Comprehensive Loss Accumulated other comprehensive 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 loss for the years ended December 31, 2018, 2017 and 2016 (in thousands): Years Ended December 31, 2018 2017 2016 Beginning balance accumulated other comprehensive loss $ (451 ) $ (21 ) $ (75 ) Unrealized gains (losses) on investments, net of tax (1) 144 (337 ) 75 Currency translation adjustment (17 ) (93 ) (21 ) Net other comprehensive income (loss) 127 (430 ) 54 Ending balance accumulated other comprehensive loss $ (324 ) $ (451 ) $ (21 ) (1) There was no tax benefit for other comprehensive income (loss) for the years ended December 31, 2018, 2017 and 2016. Income Taxes On December 22, 2017, the United States enacted H.R.1., known as the Tax Cuts and Jobs Act, which represented a substantial change to tax laws in the United States. The SEC staff subsequently issued Staff Accounting Bulletin No. 118, Income Tax Accounting Implications of the Tax Cuts and Jobs Act 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 n |
Investments and Fair Value Meas
Investments and Fair Value Measurements | 12 Months Ended |
Dec. 31, 2018 | |
Investments And Fair Value Measurements [Abstract] | |
Investments And Fair Value Measurements | 3. Investments and Fair Value Measurements Investments As of December 31, 2018 and 2017, we primarily invested our excess cash in 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, 2018 and 2017 (in thousands): 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 Gross Unrealized Estimated December 31, 2017 Cost Gains Losses Fair Value Available-for-sale securities: Corporate debt securities $ 132,434 $ — $ (206 ) $ 132,228 Debt securities issued by U.S. government agencies 38,135 — (59 ) 38,076 Total securities with a maturity of one year or less 170,569 — (265 ) 170,304 Corporate debt securities 8,267 — (35 ) 8,232 Debt securities issued by U.S. government agencies 23,264 — (37 ) 23,227 Total securities with a maturity of one to two years 31,531 — (72 ) 31,459 Total available-for-sale securities $ 202,100 $ — $ (337 ) $ 201,763 We recorded unrealized losses related to the securities listed above as of December 31, 2018 and 2017. We believe that 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, 2018 and 2017 that are regularly measured and carried at fair value. The table segregates each security 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, 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 At December 31, 2017 Quoted Prices in Active Markets (Level 1) Significant Other Observable Inputs (Level 2) Money market funds (1) $ 48,430 $ 48,430 $ — Corporate debt securities (3) 140,460 — 140,460 Debt securities issued by U.S. government agencies (3) 61,303 — 61,303 Total $ 250,193 $ 48,430 $ 201,763 (1) Included in cash and cash equivalents on our consolidated balance sheets. (2) At December 31, 2018, $1.0 million 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 sheets. |
Property Plant and Equipment
Property Plant and Equipment | 12 Months Ended |
Dec. 31, 2018 | |
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, 2018 2017 Furniture and fixtures $ 1,611 $ 183 Computer equipment and software 102 15 Leasehold improvements 4,213 — Total property and equipment, at cost 5,926 198 Less accumulated depreciation and amortization (230 ) (121 ) Total property and equipment, net $ 5,696 $ 77 Total depreciation expense amounted to $307,000, $108,000 and $12,000 for the years ended December 31, 2018, 2017 and 2016, respectively. As part of the operating lease for our new corporate headquarters, the landlord has provided a tenant improvement allowance of $3.6 million which is included in our leasehold improvements. |
Intangible Assets
Intangible Assets | 12 Months Ended |
Dec. 31, 2018 | |
Goodwill And Intangible Assets Disclosure [Abstract] | |
Intangible Assets | 5 . Intangible Assets The following table presents intangible assets (in thousands): December 31, Estimated 2018 2017 useful life Acquired and in-licensed rights $ 2,262 $ 1,699 7 - 21 Years Capitalized regulatory approval milestones 90,000 — 16 Years Less accumulated amortization (3,348 ) (478 ) Total intangible assets, net $ 88,914 $ 1,221 The increase in capitalized regulatory milestones The Company recorded $2.9 million, $0.1 million and $0.1 million, respectively, in amortization expense related to intangible assets during the years ended December 31, 2018, 2017 and 2016. Estimated future amortization expense for intangible assets as of December 31, 2018 is as follows (in thousands): Total 2019 $ 5,863 2020 5,879 2021 5,861 2022 5,856 2023 5,843 Thereafter 59,613 $ 88,914 The weighted average remaining amortizable life of our patents was 12.15 years at December 31, 2018. For additional detail of Akcea's license agreements with Ionis see Note 7, License Agreements and Services Agreement with Ionis |
Strategic Collaboration with No
Strategic Collaboration with Novartis | 12 Months Ended |
Dec. 31, 2018 | |
Strategic Collaboration With Novartis [Abstract] | |
Strategic Collaboration with Novartis | 6 . In January 2017, we initiated a strategic collaboration with Novartis for the development and commercialization of AKCEA-APO(a)-L Rx Rx Rx We received a $75.0 million upfront payment in the first quarter of 2017, of which we retained $60.0 million and we paid Ionis $15.0 million as a sublicense fee under our Cardiometabolic License Agreement with Ionis. If Novartis exercises its option for a drug, Novartis will pay us a license fee equal to $150.0 million for each drug licensed by Novartis. In addition, for w Rx Rx Rx 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 are 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 represents 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 are recognizing 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 are the party providing the services and API under the collaboration agreement. None of the options or development or regulatory milestone payments under this agreement have been included in the transaction price as all payments are 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, Novartis’ efforts, and the receipt of regulatory approval. We will 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 are recognizing revenue related to each of our performance obligations as follows: • We will satisfy the development services performance obligation for AKCEA-APO(a)-L Rx • We will satisfy the development services performance obligation for AKCEA-APOCIII-L 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. 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. Our IPO is discussed in Note 11, Initial Public Offering During the years ended December 31, 2018 and 2017 (as revised), we earned revenue of $50.6 million and $43.4 million, respectively, from our strategic collaboration with Novartis, representing 100% of our research and development revenue. We did not earn research and development revenue during the year ended December 31, 2016. During the year ended December 31, 2018 we recognized $42.2 million of revenue from amounts that were in our beginning deferred revenue balance. |
License Agreements and Services
License Agreements and Services Agreement with Ionis | 12 Months Ended |
Dec. 31, 2018 | |
Related Party Transactions [Abstract] | |
License Agreements and Services Agreement with Ionis | 7 . 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 development, commercialization and license agreement, or 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. 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 drugs, 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 drugs. We also pay Ionis for the API and drug product we use in our nonclinical and clinical studies for all of our drugs. 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 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, 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 the drugs licensed to Novartis under the collaboration agreement, if our annual sales reach $500.0 million, $1.0 billion and $2.0 billion, we will be obligated to pay Ionis sales milestones in the amount of $50.0 million for each sales milestone reached by each Lipid Drug. If and when triggered, we will pay Ionis each of these sales milestones over the subsequent 12 quarters in equal payments. 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. For additional detail regarding our strategic collaboration with Novartis, see Note 6, Strategic Collaboration with Novartis. TTR Development, Commercialization, Collaboration and License Agreement On April 17, 2018, our stockholders, other than Ionis and its affiliates, approved the development, commercialization, collaboration and license agreement, or TTR License Agreement, and a stock purchase agreement, or Ionis SPA, with Ionis, our majority shareholder 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 which required the approval of the agreements by 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 drug 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 Under the TTR License Agreement, w e and Ionis also agreed to share TEGSEDI and AKCEA-TTR-L Rx Rx Rx Rx The TTR License Agreement will remain in effect until the expiration of all included payment obligations, or 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. Prospectively we will be responsible for the procurement of all additional inventory. The inventory did not have a carrying value on the books of Ionis at the time of the acquisition. As such, in accordance with the accounting guidance for common control transactions above, we recorded the purchase of this inventory 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 loss per share and we have applied the two–class method as discussed in Note 13, Basic and Diluted Net 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 are 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 loss per share as discussed in Note 13, Basic and Diluted Net Loss Per Share . During the year ended December 31, 2018, we recorded $26.0 million, as a component of research and development expense related to the TTR License Agreement. We did not record any research and development expense related to the TTR License Agreement in the years ended December 31, 2017 and 2016. In addition, on July 11, 2018, we received marketing authorization, or MA, approval for TEGSEDI from the European Commission, or EC, for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis, or hATTR amyloidosis, in the European Union, or EU. As a result of the MA approval in the EU, 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. As a result of the marketing authorization in the EU, we capitalized this regulatory approval milestone payment as a license intangible asset on our consolidated balance sheets as the amount in expected to be recoverable through future cash flows. In addition, 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 United States. As a result of the regulatory approval in the United States, 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. As a result of the FDA approval in the U.S., we capitalized this regulatory approval milestone payment as a license intangible asset on our consolidated balance sheets as the amount in 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 assets 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 $2.7 million for the year ended December 31, 2018. We did not record any amortization expense for the TTR milestone payments for the years ended December 31, 2017 and 2016. 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. As of December 31, 2018 and 2017, we owed Ionis $18.9 million and $14.4 million, respectively. The following table summarizes the amounts recorded related to transactions with Ionis including amounts related to the TTR licensing transaction for the following periods (in thousands): Years Ended December 31, 2018 2017 2016 Operating expenses: Services performed by Ionis $ 15,404 $ 9,742 $ 8,599 Active pharmaceutical ingredient manufactured by Ionis 5,229 6,012 12,648 Commercial inventory manufactured by Ionis 1,288 — — Sublicensing expenses 7,200 48,394 — Out-of-pocket expenses paid by Ionis 50,870 37,426 42,367 Total operating expenses generated by transactions with Ionis 79,991 101,574 63,614 Distributions to Ionis: Commercial inventory manufactured by Ionis 4,707 — — Distribution to Ionis in connection with the TTR license transaction 3,085 — — Total distribution to Ionis 7,792 — — Total charges generated by transactions with Ionis 87,783 101,574 63,614 Payable balance to Ionis at the beginning of the period 14,365 24,355 9,198 Less: total amounts paid to Ionis during the period (83,247 ) (78,170 ) (48,457 ) Less: non-cash sublicensing expenses — (33,394 ) — Total amount payable to Ionis at period end $ 18,901 $ 14,365 $ 24,355 |
Collaboration and License Agree
Collaboration and License Agreement with PTC Therapeutics | 12 Months Ended |
Dec. 31, 2018 | |
Collaboration And License Agreement [Abstract] | |
Collaboration and License Agreement with PTC Therapeutics | 8 . 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 sub-license royalty related to the TTR License Agreement. If WAYLIVRA is approved by the FDA or EMA, PTC Therapeutics will pay us $6.0 million. In addition, we are eligible to receive up to $8.0 million for the achievement of regulatory milestones 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 recognized 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 will be split 60% to Ionis and 40% to Akcea. All WAYLIVRA milestone payments and royalties that we are eligible to receive from PTC will be 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. The option to purchase supply from us is subject to a supply agreement we are negotiating with PTC. 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 is expected to be priced similar to other manufacturing options with similar customers and is therefore not considered a material right. 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. None of the regulatory milestones have been included in the transaction price, as all milestones were fully constrained. As part of our evaluation of the constraint, we considered numerous factors, including that regulatory approvals are not within our control and accordingly the related milestones are fully constrained and excluded from the arrangement consideration until such regulatory approvals are received. Any consideration related to sales-based royalties will be recognized when the related sales occur |
Equity and Stock-Based Compensa
Equity and Stock-Based Compensation | 12 Months Ended |
Dec. 31, 2018 | |
Disclosure Of Compensation Related Costs Sharebased Payments [Abstract] | |
Equity and Stock-Based Compensation | 9 . 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 development, commercialization and 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, 2018 and 2017, we had no shares of Series A convertible preferred stock issued or outstanding. Our IPO is discussed in Note 11, 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, 2018 and 2017, there were no shares of Preferred Stock outstanding. Common Stock At December 31, 2018 and 2017, we had 125,000,000 and 100,000,000, respectively shares of common stock authorized, of which 89,345,978 and 66,541,629 were issued and outstanding as of December 31, 2018 and 2017, 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 the 2015 Plan. In May 2017 and June 2017, our board of directors and stockholder, respectively, approved an amendment to our 2015 Equity Incentive Plan 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, 2018, the aggregate number of shares of common stock that may be issued pursuant to stock awards under the 2015 Plan was 18,500,000 shares. The 2015 Plan also provides for the grant of nonstatutory stock options, or NSOs, incentive stock options, or ISOs, stock appreciation rights, restricted stock awards and restricted stock unit awards. At December 31, 2018, a total of 11,010,828 options were outstanding, of which 4,305,172 were exercisable, 38,134 restricted stock unit awards were outstanding, and 1,610,490 shares were available for future grant under the 2015 Plan. 2017 Employee Stock Purchase Plan In May 2017 and June 2017, our board of directors and stockholder, respectively, approved our 2017 Employee Stock Purchase Plan, or 2017 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, 2018, 500,000 shares of common stock were added to the ESPP. As of December 31, 2018, the aggregate number of shares of common stock reserved under the 2017 ESPP was 1,000,000 and we had 968,449 shares available for future issuance under the 2017 ESPP. During the year ended December 31, 2018, 31,551 shares were issued under our 2017 ESPP. At December 31, 2018, accrued liabilities included $0.4 million of ESPP contributions for which the related shares were issued on January 1, 2019. Stock Option Activity The following table summarizes the stock option activity for the year ended December 31, 2018 (in thousands, except per share and contractual life data) for the 2015 Plan: Number of Shares Weighted Average Exercise Price per Share Average Remaining Contractual Term (Years) Aggregate Intrinsic Value Outstanding at December 31, 2017 7,905 $ 9.64 Granted 4,833 $ 22.86 Exercised (835 ) $ 7.98 Cancelled/forfeited/expired (892 ) $ 16.53 Outstanding at December 31, 2018 11,011 $ 15.00 8.07 $ 167,184 Exercisable at December 31, 2018 4,305 $ 8.04 7.11 $ 95,124 The weighted-average estimated fair value of options granted were $18.29, $10.4 and $4.13 for the years ended December 31, 2018, 2017 and 2016, respectively. For the year ended December 31, 2017, no stock options were exercised. For the year ended December 31, 2018, 834,800 stock options were exercised. As of December 31, 2018, total unrecognized estimated non-cash stock-based compensation expense related to non-vested stock options was $60.5 million. We will adjust total unrecognized compensation cost 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 1.32 years. Stock-based Compensation Expense and Valuation Information The following table summarizes stock-based compensation expense for the years ended December 31, 2018, 2017 and 2016 (in thousands): Years Ended December 31, 2018 2017 2016 Cost of sales - product $ 160 $ — $ — Research and development expenses 9,435 8,630 4,576 Selling, general and administrative expenses 34,687 8,909 5,573 Total $ 44,282 $ 17,539 $ 10,149 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 employee's requisite service 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 estimate forfeitures based on Ionis' historical rates of forfeiture as we do not have similar historical information for ourselves. We and Ionis are engaged in similar businesses and we believe this is a good estimate of expected forfeitures. As we gain additional historical information, we will transition to using our historical forfeiture rate. 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, 2018, 2017 and 2016, we used the following weighted-average assumptions in our Black-Scholes calculations for stock option grants under our 2015 Equity Incentive Plan: Employee Stock Options: Years Ended December 31, 2018 2017 2016 Risk-free interest rate 2.8 % 1.9 % 1.6 % Dividend yield 0.0 % 0.0 % 0.0 % Volatility 77.1 % 79.5 % 71.4 % Expected life 6.08 years 6.06 years 6.08 years Board of Director Stock Options: Years Ended December 31, 2018 2017 2016 Risk-free interest rate 2.9 % 1.9 % 2.0 % Dividend yield 0.0 % 0.0 % 0.0 % Volatility 78.2 % 79.4 % 79.6 % Expected life 6.42 years 6.25 years 6.08 years |
Income Taxes
Income Taxes | 12 Months Ended |
Dec. 31, 2018 | |
Income Tax Disclosure [Abstract] | |
Income Taxes | 1 0 . Income Taxes Loss before income taxes is comprised of (in thousands): Years Ended December 31, 2018 2017 2016 (as revised) United States $ (218,794 ) $ (108,691 ) $ (83,217 ) Foreign (6,580 ) (11,593 ) — Loss before income tax expense $ (225,374 ) $ (120,284 ) $ (83,217 ) The provision (benefit) for income taxes is comprised of (in thousands): Years Ended December 31, 2018 2017 2016 Current: Federal $ — $ — $ — State 73 1,041 — Foreign 374 234 — Total current 447 1,275 — Deferred: Federal — — — State — — — Foreign — — — Total deferred — — — Income tax expense $ 447 $ 1,275 $ — We recorded income tax expense of $0.4 million for the year ended December 31, 2018, which primarily consists of foreign income tax. We recorded income tax expense of $1.3 million for the year ended December 31, 2017, which primarily consists of state and foreign income tax. There is no provision for income taxes for the year ended December 31, 2016 because we have historically incurred net operating losses and we maintain a full valuation allowance against our net deferred tax assets. The reconciliation between our effective tax rate on loss from continuing operations and the statutory U.S. tax rate is as follows (in thousands): Years Ended December 31, 2018 2017 2016 (as revised) Pre-tax loss $ (225,374 ) $ (120,284 ) $ (83,217 ) Statutory rate (47,329 ) 21.0 % (42,099 ) 35.0 % (29,126 ) 35.0 % State income tax net of federal benefit (6,441 ) 2.9 % (2,371 ) 2.0 % (4,099 ) 4.9 % Impact of foreign tax rate differential 1,735 (0.8 )% 4,072 (3.4 )% — — Net change in valuation allowance 54,173 (24.0 )% (18,917 ) 15.7 % 43,438 (52.1 )% IP Transfer (3,947 ) 1.8 % Tax credits (4,035 ) 1.8 % 4,189 (3.5 )% (11,007 ) 13.2 % IPO/Deconsolidation adjustment — — 37,911 (31.5 )% — — Tax rate change 3,906 (1.7 )% 19,046 (15.8 )% — — Nondeductible items and other 1,734 (0.9 )% (556 ) 0.5 % 794 (1.0 )% Stock-based compensation 651 (0.3 )% — — — — Effective rate $ 447 (0.2 )% $ 1,275 (1.0 )% $ — — Significant components of our deferred tax assets and liabilities as of December 31, 2017 and 2016 are as follows (in thousands): December 31, 2018 2017 (as revised) Deferred Tax Assets: Net operating loss carryovers $ 51,280 $ 1,157 Tax credits 31,768 29,334 Stock-based compensation 11,812 7,515 Deferred revenue 6,876 29,256 Intangible and capital assets 56,984 — Other 1,996 240 Total deferred tax assets $ 160,716 $ 67,502 Deferred Tax Liabilities: Fixed assets (811 ) (125 ) Total deferred tax liabilities $ (811 ) $ (125 ) Valuation allowance (159,905 ) (67,377 ) Net deferred tax assets and liabilities $ — $ — On December 22, 2017, the U.S. government enacted comprehensive tax legislation commonly referred to as the Tax Cuts and Jobs Act of 2017 (the "Tax Act"). The Tax Act created a new requirement on global intangible low-taxed income (“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 the election to account for GILTI as a component of current taxes incurred rather than as a component of deferred taxes. 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, 2018, we had federal and state tax net operating loss carry forwards on a separate basis of $238.7 million and $4.2 million, respectively. $4.3 million of the federal net operating loss carry forwards will expire in 2034 and the remaining $234.4 million can be carried forward indefinitely. The state tax net operating loss carry forwards will begin to expire in 2029. We also have federal research and development tax credit carry forwards of $37.4 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. We have incurred financial statement losses since inception and as a result 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 increased by $92.5 million from December 31, 2017 to December 31, 2018. The increase relates primarily to the net operating loss carryforward generated in 2018 and certain costs associated with the Inotersen transaction with Ionis which are capitalized and amortized for tax purposes. Pursuant to the SEC Staff Accounting Bulletin No. 118, Income Tax Accounting Implications of the Tax Cuts and Jobs Act (“SAB 118”), a company may select between one of three scenarios to determine a reasonable estimate arising from the Tax Act. Those scenarios are (i) a final estimate which effectively closes the measurement window; (ii) a reasonable estimate leaving the measurement window open for future revisions; and (iii) no estimate as the law is still being analyzed. The Company was able to provide a reasonable estimate for the provisional revaluation of deferred taxes and the effects of the transition tax on undistributed foreign earnings and profits for the period ended December 31, 2017. During the quarter ended December 31, 2018 the Company completed its accounting for the impacts of the Tax Act and identified no material changes from its original analysis. In February 2018, the FASB issued Accounting Standards Update No. 2018-02, Income Statement—Reporting Comprehensive Income (Topic 220): Reclassification of Certain Tax Effects from Accumulated Other Comprehensive Income (ASU 2018-02), which allows companies to reclassify stranded tax effects resulting from the Tax Act, from accumulated other comprehensive income to retained earnings. The new standard is effective for us beginning January 1, 2019, with early adoption permitted. We elected to early adopt the new standard at the beginning of the fourth quarter of 2018 using the aggregate portfolio approach. We elected not to reclassify the income tax effects of the Tax Act from AOCI to retained earnings. 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, 2018 2017 2016 Beginning balance of unrecognized tax benefits $ 5,001 $ 5,012 $ 1,766 Additions related to the current year 691 1,723 3,246 Decreases related to prior year tax positions (86 ) (1,734 ) — Ending balance of unrecognized tax benefits $ 5,606 $ 5,001 $ 5,012 We have unrecognized tax benefits of $5.6 million and $5.0 million for the years ended December 31, 2018 and 2017, respectively. Due to our valuation allowance, there are no unrecognized tax benefits at December 31, 2018 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 year ended December 31, 2018, 2017 or 2016. We are subject to taxation in the United States and various state and foreign jurisdictions. The tax years for 2014 through 2018 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, 2018 | |
Initial Public Offering [Abstract] | |
Initial Public Offering | 1 1 . 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, 2018 | |
Compensation And Retirement Disclosure [Abstract] | |
Employment Benefits | 1 2 . 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 $18,500 and $24,500 in 2018 for employees under 50 years old and employees 50 years old or over, respectively. We made approximately $1.6 million, $0.3 million and $0.2 million in matching contributions for the years ended December 31, 2018, 2017 and 2016, respectively. |
Basic and Diluted Net Loss Per
Basic and Diluted Net Loss Per Share | 12 Months Ended |
Dec. 31, 2018 | |
Earnings Per Share [Abstract] | |
Basic and Diluted Net Loss Per Share | 1 3 . 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 completed on April 17, 2018 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 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 losses for the years ended December 31, 2018, 2017 and 2016: Year Ended December 31, 2018 2017 2016 Net loss $ (225,821 ) $ (121,559 ) $ (83,217 ) Preferred stock dividend — (20,100 ) — Distributions to Ionis (7,792 ) — — Distributable losses $ (233,613 ) $ (141,659 ) $ (83,217 ) The following table summarizes the reconciliation of weighted-average shares outstanding used in the calculation of basic loss per share for the years ended December 31, 2018, 2017 and 2016: Year Ended December 31, Determination of shares: 2018 2017 2016 Weighted-average preferred shares outstanding — 15,748,009 28,884,540 Weighted-average common shares outstanding owned by Ionis 59,812,394 20,669,446 — Weighted-average common shares outstanding owned by others 21,553,407 9,593,322 — Total weighted-average shares outstanding 81,365,801 46,010,777 28,884,540 The following table summarizes the calculation of basic loss per share for the years ended December 31, 2018, 2017 and 2016 (in thousands, except per share amounts): Year Ended December 31, 2018 2017 2016 Losses attributable to preferred shares $ — $ (48,485 ) $ (83,217 ) Less: Assumed dividend to preferred shares — 20,100 — Income (losses) allocated to preferred shares — (28,385 ) (83,217 ) Weighted-average preferred shares outstanding — 15,748,009 28,884,540 Basic loss per preferred share $ — $ (1.80 ) $ (2.88 ) Losses allocated to Ionis $ (171,730 ) $ (63,638 ) $ — Plus: Distribution to Ionis 7,792 — — Losses available to Ionis (163,938 ) (63,638 ) - Weighted-average common shares outstanding owned by Ionis 59,812,394 20,669,446 — Basic loss per common share owned by Ionis $ (2.74 ) $ (3.08 ) $ — Losses allocated to common shares owned by others $ (61,883 ) $ (29,536 ) $ — Weighted-average common shares outstanding owned by others 21,553,407 9,593,322 — Basic loss per common share owned by others $ (2.87 ) $ (3.08 ) $ — For the years ended December 31, 2018, 2017 and 2016, 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. |
Contractual Obligations and Com
Contractual Obligations and Commitments | 12 Months Ended |
Dec. 31, 2018 | |
Commitments And Contingencies Disclosure [Abstract] | |
Contractual Obligations and Commitments | 1 4. The following table summarizes the contractual obligations as of December 31, 2018 (in thousands): Payments due by period Year Ending December 31, Total Less than 1 year 1 to 3 years 3 to 5 years More than 5 years Operating lease obligations $ 23,688 $ 2,308 $ 4,711 $ 4,805 $ 11,864 Purchase commitments 5,033 5,033 - - - Total $ 28,721 $ 7,341 $ 4,711 $ 4,805 $ 11,864 Operating Lease On April 5, 2018, we entered into an operating lease agreement with MEPT Seaport 13 Stillings LLC, or MEPT, for 30,175 square feet of office space located in Boston, Massachusetts for our new corporate headquarters. The commencement date of the lease was August 2018 and the initial term of the lease is 123 months with one five-year renewal option. We took occupancy of the office space in Boston, Massachusetts in September 2018. On November 12, 2018, we entered into an operating lease agreement with Ionis Pharmaceuticals 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. Rent expense for the year ended December 31, 2018, 2017 and 2016 was $2.4 million, $0.7 million and $0.4 million, respectively. We recognize rent expense on a straight-line basis over the lease term for the lease of our office spaces, which resulted in a deferred rent balance of $4.8 million and $39,000 at December 31, 2018 and 2017, respectively. 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. |
Restructuring
Restructuring | 12 Months Ended |
Dec. 31, 2018 | |
Restructuring And Related Activities [Abstract] | |
Restructuring | 1 5 . Restructuring On September 6, 2018, we enacted a plan to reorganize our workforce to better align with the immediate needs of our business, 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. team members 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 do not expect to incur any additional significant costs associated with this reorganization. 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): Year Ended December 31, 2018 Restructuring reserve beginning balance $ — Restructuring expenses incurred during the period 1,889 Adjustments during the period (150 ) Amounts paid during the period (1,696 ) Restructuring reserve ending balance $ 43 |
Quarterly Financial Data (Unaud
Quarterly Financial Data (Unaudited) | 12 Months Ended |
Dec. 31, 2018 | |
Quarterly Financial Information Disclosure [Abstract] | |
Quarterly Financial Data (Unaudited) | 1 6 . 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, 2018 and 2017 are as follows (in thousands, except per share data): 2018 Quarters First Quarter Second Quarter Third Quarter Fourth Quarter Revenue: Commercial revenue: Product revenue $ — $ — $ — $ 2,237 Licensing revenue — — 12,000 — Total commercial revenue — — 12,000 2,237 Research and development revenue under collaborative agreement 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 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 Interest expense — — — — Other income (expense) (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 preferred stock, basic and diluted $ — $ — $ — $ — Weighted-average shares of preferred stock outstanding, basic and diluted — — — — Net loss per share of common stock owned by Ionis, basic and diluted $ (0.44 ) $ (0.72 ) $ (0.73 ) $ (0.79 ) Weighted-average shares of common stock outstanding owned by Ionis, basic and diluted 45,447,879 60,832,494 65,538,467 67,129,553 Net loss per share of common stock owned by others, basic and diluted $ (0.44 ) $ (0.85 ) $ (0.73 ) $ (0.79 ) Weighted-average shares of common stock outstanding owned by others, basic and diluted 21,171,372 21,492,157 21,671,415 21,869,713 2017 Quarters First Quarter Second Quarter Third Quarter Fourth Quarter (as revised) Revenue: Commercial revenue: Product revenue $ — $ — $ — $ — Licensing revenue — — — — Total commercial revenue — — — — Research and development revenue under collaborative agreement 6,094 5,713 9,906 21,688 Total revenue 6,094 5,713 9,906 21,688 Expenses: Cost of sales - product — — — — Cost of sales - intangible asset amortization — — — — Cost of license — — — — Research and development 64,794 18,487 17,640 25,968 Selling, general and administrative 4,676 6,915 8,373 17,018 Total expenses 69,470 25,402 26,013 42,986 Loss from operations (63,376 ) (19,689 ) (16,107 ) (21,298 ) Other income (expense): Investment income 61 245 687 819 Interest expense (541 ) (965 ) (224 ) — Other income (expense) — 50 73 (19 ) Loss before income tax expense (63,856 ) (20,359 ) (15,571 ) (20,498 ) Income tax expense — — (2,066 ) 791 Net loss $ (63,856 ) $ (20,359 ) $ (17,637 ) $ (19,707 ) Net loss per share of preferred stock, basic and diluted $ (2.21 ) $ (0.70 ) $ (0.01 ) $ — Weighted-average shares of preferred stock outstanding, basic and diluted 28,884,540 28,888,450 5,651,323 — Net loss per share of common stock owned by Ionis, basic and diluted $ — $ — $ (0.33 ) $ (0.30 ) Weighted-average shares of common stock outstanding owned by Ionis, basic and diluted — — 36,555,903 45,447,879 Net loss per share of common stock owned by others, basic and diluted $ — $ — $ (0.33 ) $ (0.30 ) Weighted-average shares of common stock outstanding owned by others, basic and diluted — — 16,966,712 21,093,750 (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) For the purposes of calculating EPS under the two-class method since our IPO in July 2017, we have allocated the net loss between the common stock and the Series A convertible preferred stock for the three-month period ended September 30, 2017. We determined it was appropriate to allocate losses to the Series A convertible preferred stock because it was the lowest form of subordinated equity during such period and because Ionis, the sole holder of the Series A convertible preferred stock, was absorbing our losses during such period. Basic EPS for each class of stock is computed by dividing total distributable losses applicable to preferred and common stock, 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 losses for the quarter ended September 30, 2017 (in thousands): September 30, 2017 (as revised) Net loss $ (17,637 ) Preferred stock dividend (1,791 ) Distributable losses $ (19,428 ) The following table summarizes the reconciliation of weighted-average shares outstanding used in the calculation of basic EPS for the quarter ended September 30, 2017: September 30, 2017 Determination of shares: Weighted-average preferred shares outstanding 5,651,323 Weighted-average common shares outstanding 53,522,615 Total weighted-average shares outstanding 59,173,938 The following table summarizes the calculation of basic EPS for the quarter ended September 30, 2017 (in thousands, except per share amounts): September 30, 2017 (as revised) Losses attributable to preferred shares $ (1,855 ) Less: Assumed dividend to preferred shares 1,791 Income (losses) allocated to preferred shares $ (64 ) Weighted-average preferred shares outstanding 5,651,323 Basic income (loss) per preferred share $ 0.01 Losses allocated to common shares $ (17,573 ) Weighted-average common shares outstanding 53,522,615 Basic loss per common share $ (0.33 ) ( 3) We did not include dilutive common equivalent shares in the computation of diluted net loss per share because the effect would have been antidilutive. |
Subsequent Events
Subsequent Events | 12 Months Ended |
Dec. 31, 2018 | |
Subsequent Events [Abstract] | |
Subsequent Events | 1 7 . Subsequent Events AKCEA-APO(a)-L Rx On February 22, 2019, Novartis exercised its option to license AKCEA-APO(a)-L Rx Strategic Collaboration with Novartis . As a result we earned a license fee of $150.0 million of which we will pay $75.0 million to Ionis as a sublicense fee. We will issue 2,837,373 shares of our common stock to Ionis as payment of the $75.0 million sublicense fee. |
Summary of Significant Accoun_2
Summary of Significant Accounting Policies (Policies) | 12 Months Ended |
Dec. 31, 2018 | |
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 and prior to our IPO, the valuation of common stock. 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 as a component of accumulated other comprehensive loss within the consolidated statements of comprehensive loss. |
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 receivables. 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 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 (expense) on our consolidated statement of operations. We use the specific identification method to determine the cost of securities sold. |
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. For TEGSEDI inventory related costs incurred subsequent to July 1, 2018, we reflected these amounts as inventory on our consolidated balance sheets at the lower of cost or market 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 statements 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. At December 31, 2018 the amount of finished goods recorded in our consolidated balance sheets in other current assets related to our approved product TEGSEDI was $85,000. |
Property, Plant and Equipment | Property and Equipment Property and equipment are recorded at cost and depreciated over their estimated useful lives using the straight-line method over the estimated useful life of each asset. Furniture and fixtures are depreciated over seven 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. |
Intangible Assets | Intangible Assets We obtained exclusive licenses from Ionis for specific patents that Ionis owns and maintains related to our drug 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 license 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. Amortization expense is recorded as a component of cost of sales to the extent the underlying license is commercialized or research and development prior to its commercialization in the consolidated statements of operations. |
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 . |
Revenue Recognition | Revenue Recognition Collaboration and License Revenue In May 2014, the Financial Accounting Standards Board, or FASB, issued Accounting Standards Update, or ASU, No. 2014-09, which amended the guidance for accounting for revenue from contracts with customers. This ASU superseded the revenue recognition requirements in ASC Topic 605, Revenue Recognition Revenue from Contracts with Customers 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 drug 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 not concluded that the option to license a drug or the options for additional goods or services that may be requested in the future under our collaboration agreement are 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 drug 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, 2018, we have three revenue streams: our strategic collaboration, option and license agreement, or collaboration agreement, with Novartis Pharma AG, or Novartis, which we entered into in January 2017, our collaboration and license agreement with PTC Therapeutics International Limited, or PTC Therapeutics, which we entered into in August 2018 and commercial product revenue related TEGESDI sales subsequent to product launch in the fourth quarter of 2018. For a complete discussion of the accounting for our revenue streams, see Note 6, Strategic Collaboration with Novartis, Collaboration and License Agreement with PTC Therapeutics Summary of Significant Accounting Policies Effective January 1, 2018, we adopted Topic 606 using the full retrospective transition method. Under this method, we revised our consolidated financial statements for prior period amounts including the periods included in this Report on Form 10-K, as if Topic 606 had been effective for such periods. The references “as revised” used herein refer to revisions of amounts originally reported for the year ended December 31, 2017 and as of December 31, 2017 as Impact of Adoption As a result of adopting Topic 606 on January 1, 2018, we revised our comparative financial statements for the prior years as if Topic 606 had been effective for that period. On September 18, 2018, we filed a Current Report on Form 8-K to present recast consolidated financial statements for each of the three years ended December 31, 2015, 2016 and 2017, to reflect our adoption of the new accounting standard for revenue recognition set forth in Topic 606. The financial information recast in the Form 8-K was originally filed with the SEC in our Annual Report on Form 10-K for the fiscal year ended December 31, 2017, which was filed with the SEC on February 28, 2018. Under Topic 605, we recognized revenue from our collaboration with Novartis over time on a straight-line basis. Under Topic 606, we recognize revenue from our collaboration with Novartis using the input method based on the total cost of performing services over time. As a result, the following financial statement line items for fiscal year 2017 were affected. Consolidated Balance Sheets December 31, 2017 (in thousands) As Revised Under Topic 606 As Originally Reported Under Topic 605 Effect of Change Current portion of deferred revenue $ 58,192 $ 50,579 $ 7,613 Long-term portion of deferred revenue 12,501 8,306 4,195 Accumulated deficit $ (296,221 ) $ (284,413 ) $ (11,808 ) Consolidated Statements of Operations and Comprehensive Loss Year Ended December 31, 2017 (in thousands, except per share data) As Revised Under Topic 606 As Originally Reported Under Topic 605 Effect of Change Research and development revenue under collaborative Agreement $ 43,401 $ 55,209 $ (11,808 ) Loss from operations (120,470 ) (108,662 ) (11,808 ) Net loss (121,559 ) (109,751 ) (11,808 ) Net loss per share of preferred stock, basic and diluted (1.80 ) (1.55 ) (0.25 ) Net loss per share of common stock owned by Ionis, basic and diluted (3.08 ) (2.82 ) (0.26 ) Net loss per share of common stock owned by others, basic and diluted $ (3.08 ) (2.82 ) $ (0.26 ) Consolidated Statement of Cash Flows Years Ended December 31, 2017 (in thousands) As Revised Under Topic 606 As Originally Reported Under Topic 605 Effect of Change Net loss $ (121,559 ) $ (109,751 ) $ (11,808 ) Adjustments to reconcile net loss to net cash used in operating activities: Deferred revenue 70,693 58,885 11,808 Cash, cash equivalents and restricted cash at beginning of period 7,857 7,857 — Cash, cash equivalents and restricted at end of period $ 58,367 $ 58,367 $ — |
Product Revenue, Net | Product Revenue, Net Subsequent to regulatory approval in Europe on July 11, 2018 and FDA approval in the U.S. on October 5, 2018, in the fourth quarter of 2018 we began to sell TEGSEDI in the U.S. and Germany. In the U.S., the product is distributed through an exclusive distribution agreement with a third-party logistics (3PL) company 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. In Germany, the product is distributed through a non-exclusive distribution model with a 3PL that takes title to the product and currently represents our sole customer in Germany. Our customer in Germany then distributes TEGSEDI to hospitals and pharmacies in Germany. Revenue from product sales are recognized when the customer obtains control of our product, which occurs at a point in time, upon transfer of title to the customer. We record shipping and handling costs 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 in our consolidated statements of operations. Otherwise 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. Taxes collected from customers relating to product sales and remitted to governmental authorities are excluded from revenue. 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. We expense incremental costs of obtaining a contract as and when incurred since the expected amortization period of the asset that we would have recognized is one year or less. Reserves for Variable Consideration Revenues from product sales are recorded at the net sales price (transaction price), which includes estimates of variable consideration for which reserves are established and which result from 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. These reserves are based on the amounts earned or to be claimed on the related sales and are classified as reductions of accounts receivable (if the amount is payable to the customer) or a current liability (if the amount is payable to a party other than a customer). 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, these reserves reflect our best estimates 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 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. Government rebates: We are subject to discount obligations under government programs, including Medicaid programs and Medicare in the United States. We estimate Medicaid and Medicare rebates based upon a range of possible outcomes that are probability-weighted for the 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 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. In Germany, pharmaceutical companies must grant a specified rebate percentage to the German government. We have included this rebate as a reduction of revenue in the period the related product revenue is recognized. Trade discounts and allowances: We provide customary invoice discounts on TEGSEDI sales to our U.S. customer for prompt payment that are recorded as a reduction of revenue in the period the related product revenue is recognized. In addition, we receive and pay for various distribution services from our U.S. customer and wholesalers in the U.S. distribution channel. For services that are either not distinct from the sale of our product or for which we cannot reasonably estimate the fair value, such fees are classified as a reduction of product revenue. Product Returns: Our U.S. customer has return rights and the wholesalers 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 in the period the related product revenue is recognized. Based on the distribution model for TEGSEDI, contractual inventory limits with our customer and wholesalers and the price of TEGSEDI, we believe there will be minimal returns. Our customer in Germany only takes title to the product once it receives an order from a hospital or pharmacy and therefore does not maintain any inventory of TEGSEDI. Therefore, there is limited return risk and the Company has not recorded any return estimate in the transaction price for TEGSEDI sold in Germany. Other incentives: In the U.S., other incentives include co-payment assistance 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-pay 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. The estimate is recorded as a reduction of revenue in the same period the related revenue is recognized. During the year ended December 31, 2018, we recorded product revenue, net, of $2.2 million, which consist of $1.2 million of TEGSEDI sales in the U.S., and $1.0 million of TEGSEDI sales in Germany. The following table summarizes balances and activity in each of the product revenue allowance and reserve categories for the year ended December 31, 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 |
Cost of Product Sales | Cost of Product Sales As a result of receiving marketing authorization, or MA, approval for TEGSEDI from the European Commission, or EC, in July 2018, we began recording all TEGSEDI related expenses as cost of product sales starting in July 2018. Cost of product sales consists of manufacturing costs, transportation and freight, and indirect overhead costs associated with the manufacturing and distribution of TEGSEDI. Cost of product sales may also include period costs related to certain manufacturing services and inventory adjustment charges. Additionally, we expensed a significant portion of the cost of producing TEGSEDI that we will use in the commercial launch as research and development expense prior to the regulatory approval of TEGSEDI. |
Commercial Sublicensing Expenses | Commercial Sublicensing Expenses We incur sublicense expenses under our TTR Development, Commercialization, Collaboration and License Agreement with Ionis related to the drugs we have licensed under the agreement. We include our sublicense fee expenses in our cost of license expenses on our consolidated statements of operations for those drugs 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 and services agreement with Ionis related to the drugs we have licensed under the agreement. We include our sublicense fee expenses in our research and development expenses on our consolidated statements of operations since the applicable drugs 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, laboratory and analysis costs, toxicology studies and investigator grants. We have drugs 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 service period in our consolidated statements 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 estimated 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. The RSUs we have granted vest annually over a four-year period. 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 options and Akcea options 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. In 2017, we no longer concurrently granted 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 will continue to recognize expense based on the valuation that was determined upon the grant date for options issued in 2016 or 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 9, Equity and Stock-based Compensation, |
Accumulated Other Comprehensive Loss | Accumulated Other Comprehensive Loss Accumulated other comprehensive 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 loss for the years ended December 31, 2018, 2017 and 2016 (in thousands): Years Ended December 31, 2018 2017 2016 Beginning balance accumulated other comprehensive loss $ (451 ) $ (21 ) $ (75 ) Unrealized gains (losses) on investments, net of tax (1) 144 (337 ) 75 Currency translation adjustment (17 ) (93 ) (21 ) Net other comprehensive income (loss) 127 (430 ) 54 Ending balance accumulated other comprehensive loss $ (324 ) $ (451 ) $ (21 ) (1) There was no tax benefit for other comprehensive income (loss) for the years ended December 31, 2018, 2017 and 2016. |
Income Taxes | Income Taxes On December 22, 2017, the United States enacted H.R.1., known as the Tax Cuts and Jobs Act, which represented a substantial change to tax laws in the United States. The SEC staff subsequently issued Staff Accounting Bulletin No. 118, Income Tax Accounting Implications of the Tax Cuts and Jobs Act 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 the accompanying consolidated statements of operations. Accrued interest and penalties are included within other long-term liabilities in the consolidated balance sheets. 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 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. We have incurred financial statement losses since inception and as a result 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. 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 February 2016, the FASB issued amended accounting guidance related to lease accounting, which will require us to record all leases with a term longer than one year on our balance sheet. When we record leases on our balance sheet under the new guidance, we will record a liability with a value equal to the present value of payments we will make over the life of the lease (lease liability) and an asset representing the underlying leased asset (right of use asset). The new accounting guidance requires us to determine if our leases are operating or financing leases. We will record expense for operating leases on a straight-line basis as an operating expense. If we determine a lease is a financing lease, we will record both interest and amortization expense and generally the expense will be higher in the earlier periods of the lease. We adopted this guidance on January 1, 2019 and adjusted our opening balance sheet on that date. We elected the available practical expedients. The most significant impact was the recognition of right of use assets and lease liabilities for our operating leases. We are in the process of finalizing the impact of the adoption. The adoption will not have an impact in our consolidated statement of operations or statement of cash flows. In February 2018, the FASB issued updated guidance for reclassification of tax effects from accumulated other comprehensive income (loss). The updated guidance gives entities an option to reclassify the stranded tax effects resulting from changes due to the Tax Act from accumulated other comprehensive income (loss) to retained earnings. The updated guidance is effective for all entities for fiscal years beginning after December 31, 2018, and interim periods within those fiscal years. Early adoption is permitted and adoption is optional. We are currently assessing the impact this updated guidance could have on our consolidated financial statements and the timing of potential adoption. In June 2018, the FASB issued updated guidance to simplify the accounting for stock-based compensation expense for non-employees. We adopted this guidance in the second quarter of 2018. We have not granted stock options to non-employees as of December 31, 2018 and therefore this new guidance has no impact on our consolidated financial statements. 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. The guidance is effective for fiscal years beginning after December 15, 2019 and early adoption is permitted. We anticipate we will adopt this updated guidance on January 1, 2019 and we do not expect it to have a significant impact on our disclosures. In August 2018, the FASB issued clarifying guidance on how to account for implementation costs related to hosted cloud-servicing arrangements. The primary change is to include any arrangement in which the customer accesses or uses software but does not take possession of the software when assessing for the capitalization of implementation costs of internal use software. Qualified costs are to be capitalized and amortized over the service period and they need to be expensed in the same line item as the service expense and recognized on the balance sheet in the same category as amounts prepaid for the hosted cloud-servicing arrangements generally as another asset. Cash flows related to the capitalized implementation costs should be presented consistent with the presentation of cash flows for the fees related to hosted cloud-servicing arrangements. The update can be applied either retrospectively or prospectively to all implementation costs incurred after the date of adoption. The updated guidance is effective for fiscal years beginning after December 15, 2019, and interim periods within those fiscal years. Early adoption is permitted in any interim period. We are currently assessing the effects this updated guidance could have on our consolidated financial statements and timing of potential adoption. In November 2018, the FASB issued clarifying guidance of the interaction between the collaboration accounting guidance and the new revenue recognition guidance we adopted on January 1, 2018 (Topic 606). The clarifying guidance included the following: 1) When a participant is considered a customer in a collaborative arrangement, all of the associated accounting under Topic 606 should be applied; 2) Adds “unit of account” concept to collaboration accounting guidance to align with Topic 606. This is used to determine if revenue is recognized or if a contra expense is recognized from consideration received under a collaboration; and 3) Precludes revenue from being recognized under Topic 606 when a transaction with a collaborative partner is determined not be a customer and is not directly related to the sales to third parties. The updated guidance is effective for public entities for fiscal years beginning after December 15, 2019, and interim periods within those fiscal years. Early adoption is permitted. We plan to adopt this guidance on January 1, 2020. We are currently assessing the effects it will have on our consolidated financial statements and disclosures. |
Summary of Significant Accoun_3
Summary of Significant Accounting Policies (Tables) | 12 Months Ended |
Dec. 31, 2018 | |
Accounting Policies [Abstract] | |
Impact of Adoption of Topic 606 | As a result, the following financial statement line items for fiscal year 2017 were affected. Consolidated Balance Sheets December 31, 2017 (in thousands) As Revised Under Topic 606 As Originally Reported Under Topic 605 Effect of Change Current portion of deferred revenue $ 58,192 $ 50,579 $ 7,613 Long-term portion of deferred revenue 12,501 8,306 4,195 Accumulated deficit $ (296,221 ) $ (284,413 ) $ (11,808 ) Consolidated Statements of Operations and Comprehensive Loss Year Ended December 31, 2017 (in thousands, except per share data) As Revised Under Topic 606 As Originally Reported Under Topic 605 Effect of Change Research and development revenue under collaborative Agreement $ 43,401 $ 55,209 $ (11,808 ) Loss from operations (120,470 ) (108,662 ) (11,808 ) Net loss (121,559 ) (109,751 ) (11,808 ) Net loss per share of preferred stock, basic and diluted (1.80 ) (1.55 ) (0.25 ) Net loss per share of common stock owned by Ionis, basic and diluted (3.08 ) (2.82 ) (0.26 ) Net loss per share of common stock owned by others, basic and diluted $ (3.08 ) (2.82 ) $ (0.26 ) Consolidated Statement of Cash Flows Years Ended December 31, 2017 (in thousands) As Revised Under Topic 606 As Originally Reported Under Topic 605 Effect of Change Net loss $ (121,559 ) $ (109,751 ) $ (11,808 ) Adjustments to reconcile net loss to net cash used in operating activities: Deferred revenue 70,693 58,885 11,808 Cash, cash equivalents and restricted cash at beginning of period 7,857 7,857 — Cash, cash equivalents and restricted at end of period $ 58,367 $ 58,367 $ — |
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 year ended December 31, 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 |
Changes in Accumulated Other Comprehensive Loss | Accumulated other comprehensive 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 loss for the years ended December 31, 2018, 2017 and 2016 (in thousands): Years Ended December 31, 2018 2017 2016 Beginning balance accumulated other comprehensive loss $ (451 ) $ (21 ) $ (75 ) Unrealized gains (losses) on investments, net of tax (1) 144 (337 ) 75 Currency translation adjustment (17 ) (93 ) (21 ) Net other comprehensive income (loss) 127 (430 ) 54 Ending balance accumulated other comprehensive loss $ (324 ) $ (451 ) $ (21 ) (1) There was no tax benefit for other comprehensive income (loss) for the years ended December 31, 2018, 2017 and 2016. |
Investments and Fair Value Me_2
Investments and Fair Value Measurements (Tables) | 12 Months Ended |
Dec. 31, 2018 | |
Investments And Fair Value Measurements [Abstract] | |
Summary of Investments | The following is a summary of our investments at December 31, 2018 and 2017 (in thousands): 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 Gross Unrealized Estimated December 31, 2017 Cost Gains Losses Fair Value Available-for-sale securities: Corporate debt securities $ 132,434 $ — $ (206 ) $ 132,228 Debt securities issued by U.S. government agencies 38,135 — (59 ) 38,076 Total securities with a maturity of one year or less 170,569 — (265 ) 170,304 Corporate debt securities 8,267 — (35 ) 8,232 Debt securities issued by U.S. government agencies 23,264 — (37 ) 23,227 Total securities with a maturity of one to two years 31,531 — (72 ) 31,459 Total available-for-sale securities $ 202,100 $ — $ (337 ) $ 201,763 |
Assets Measured at Fair Value on a Recurring Basis | The following tables present the investments we held at December 31, 2018 and 2017 that are regularly measured and carried at fair value. The table segregates each security 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, 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 At December 31, 2017 Quoted Prices in Active Markets (Level 1) Significant Other Observable Inputs (Level 2) Money market funds (1) $ 48,430 $ 48,430 $ — Corporate debt securities (3) 140,460 — 140,460 Debt securities issued by U.S. government agencies (3) 61,303 — 61,303 Total $ 250,193 $ 48,430 $ 201,763 (1) Included in cash and cash equivalents on our consolidated balance sheets. (2) At December 31, 2018, $1.0 million 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 sheets. |
Property, Plant and Equipment (
Property, Plant and Equipment (Table) | 12 Months Ended |
Dec. 31, 2018 | |
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, 2018 2017 Furniture and fixtures $ 1,611 $ 183 Computer equipment and software 102 15 Leasehold improvements 4,213 — Total property and equipment, at cost 5,926 198 Less accumulated depreciation and amortization (230 ) (121 ) Total property and equipment, net $ 5,696 $ 77 |
Intangible Assets (Tables)
Intangible Assets (Tables) | 12 Months Ended |
Dec. 31, 2018 | |
Goodwill And Intangible Assets Disclosure [Abstract] | |
Schedule of Intangible Assets | The following table presents intangible assets (in thousands): December 31, Estimated 2018 2017 useful life Acquired and in-licensed rights $ 2,262 $ 1,699 7 - 21 Years Capitalized regulatory approval milestones 90,000 — 16 Years Less accumulated amortization (3,348 ) (478 ) Total intangible assets, net $ 88,914 $ 1,221 |
Schedule of Estimated Future Amortization Expenses for Intangible Assets | Estimated future amortization expense for intangible assets as of December 31, 2018 is as follows (in thousands): Total 2019 $ 5,863 2020 5,879 2021 5,861 2022 5,856 2023 5,843 Thereafter 59,613 $ 88,914 |
License Agreements and Servic_2
License Agreements and Services Agreement with Ionis (Tables) | 12 Months Ended |
Dec. 31, 2018 | |
Related Party Transactions [Abstract] | |
Amounts Recorded Related to Transactions with Ionis Including Amounts Related to TTR Licensing Transaction | The following table summarizes the amounts recorded related to transactions with Ionis including amounts related to the TTR licensing transaction for the following periods (in thousands): Years Ended December 31, 2018 2017 2016 Operating expenses: Services performed by Ionis $ 15,404 $ 9,742 $ 8,599 Active pharmaceutical ingredient manufactured by Ionis 5,229 6,012 12,648 Commercial inventory manufactured by Ionis 1,288 — — Sublicensing expenses 7,200 48,394 — Out-of-pocket expenses paid by Ionis 50,870 37,426 42,367 Total operating expenses generated by transactions with Ionis 79,991 101,574 63,614 Distributions to Ionis: Commercial inventory manufactured by Ionis 4,707 — — Distribution to Ionis in connection with the TTR license transaction 3,085 — — Total distribution to Ionis 7,792 — — Total charges generated by transactions with Ionis 87,783 101,574 63,614 Payable balance to Ionis at the beginning of the period 14,365 24,355 9,198 Less: total amounts paid to Ionis during the period (83,247 ) (78,170 ) (48,457 ) Less: non-cash sublicensing expenses — (33,394 ) — Total amount payable to Ionis at period end $ 18,901 $ 14,365 $ 24,355 |
Equity and Stock-Based Compen_2
Equity and Stock-Based Compensation (Tables) | 12 Months Ended |
Dec. 31, 2018 | |
Stock-Based Compensation Expense | The following table summarizes stock-based compensation expense for the years ended December 31, 2018, 2017 and 2016 (in thousands): Years Ended December 31, 2018 2017 2016 Cost of sales - product $ 160 $ — $ — Research and development expenses 9,435 8,630 4,576 Selling, general and administrative expenses 34,687 8,909 5,573 Total $ 44,282 $ 17,539 $ 10,149 |
2015 Equity Incentive Plan [Member] | |
Stock Options Activity | The following table summarizes the stock option activity for the year ended December 31, 2018 (in thousands, except per share and contractual life data) for the 2015 Plan: Number of Shares Weighted Average Exercise Price per Share Average Remaining Contractual Term (Years) Aggregate Intrinsic Value Outstanding at December 31, 2017 7,905 $ 9.64 Granted 4,833 $ 22.86 Exercised (835 ) $ 7.98 Cancelled/forfeited/expired (892 ) $ 16.53 Outstanding at December 31, 2018 11,011 $ 15.00 8.07 $ 167,184 Exercisable at December 31, 2018 4,305 $ 8.04 7.11 $ 95,124 |
Weighted-Average Assumptions for Stock Options | For the years ended December 31, 2018, 2017 and 2016, we used the following weighted-average assumptions in our Black-Scholes calculations for stock option grants under our 2015 Equity Incentive Plan: Employee Stock Options: Years Ended December 31, 2018 2017 2016 Risk-free interest rate 2.8 % 1.9 % 1.6 % Dividend yield 0.0 % 0.0 % 0.0 % Volatility 77.1 % 79.5 % 71.4 % Expected life 6.08 years 6.06 years 6.08 years Board of Director Stock Options: Years Ended December 31, 2018 2017 2016 Risk-free interest rate 2.9 % 1.9 % 2.0 % Dividend yield 0.0 % 0.0 % 0.0 % Volatility 78.2 % 79.4 % 79.6 % Expected life 6.42 years 6.25 years 6.08 years |
Income Taxes (Tables)
Income Taxes (Tables) | 12 Months Ended |
Dec. 31, 2018 | |
Income Tax Disclosure [Abstract] | |
Loss Before Income Taxes | Loss before income taxes is comprised of (in thousands): Years Ended December 31, 2018 2017 2016 (as revised) United States $ (218,794 ) $ (108,691 ) $ (83,217 ) Foreign (6,580 ) (11,593 ) — Loss before income tax expense $ (225,374 ) $ (120,284 ) $ (83,217 ) |
Provision for Income Taxes | The provision (benefit) for income taxes is comprised of (in thousands): Years Ended December 31, 2018 2017 2016 Current: Federal $ — $ — $ — State 73 1,041 — Foreign 374 234 — Total current 447 1,275 — Deferred: Federal — — — State — — — Foreign — — — Total deferred — — — Income tax expense $ 447 $ 1,275 $ — |
Reconciliation of Statutory to Effective Tax Rate | The reconciliation between our effective tax rate on loss from continuing operations and the statutory U.S. tax rate is as follows (in thousands): Years Ended December 31, 2018 2017 2016 (as revised) Pre-tax loss $ (225,374 ) $ (120,284 ) $ (83,217 ) Statutory rate (47,329 ) 21.0 % (42,099 ) 35.0 % (29,126 ) 35.0 % State income tax net of federal benefit (6,441 ) 2.9 % (2,371 ) 2.0 % (4,099 ) 4.9 % Impact of foreign tax rate differential 1,735 (0.8 )% 4,072 (3.4 )% — — Net change in valuation allowance 54,173 (24.0 )% (18,917 ) 15.7 % 43,438 (52.1 )% IP Transfer (3,947 ) 1.8 % Tax credits (4,035 ) 1.8 % 4,189 (3.5 )% (11,007 ) 13.2 % IPO/Deconsolidation adjustment — — 37,911 (31.5 )% — — Tax rate change 3,906 (1.7 )% 19,046 (15.8 )% — — Nondeductible items and other 1,734 (0.9 )% (556 ) 0.5 % 794 (1.0 )% Stock-based compensation 651 (0.3 )% — — — — Effective rate $ 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, 2017 and 2016 are as follows (in thousands): December 31, 2018 2017 (as revised) Deferred Tax Assets: Net operating loss carryovers $ 51,280 $ 1,157 Tax credits 31,768 29,334 Stock-based compensation 11,812 7,515 Deferred revenue 6,876 29,256 Intangible and capital assets 56,984 — Other 1,996 240 Total deferred tax assets $ 160,716 $ 67,502 Deferred Tax Liabilities: Fixed assets (811 ) (125 ) Total deferred tax liabilities $ (811 ) $ (125 ) Valuation allowance (159,905 ) (67,377 ) 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, 2018 2017 2016 Beginning balance of unrecognized tax benefits $ 5,001 $ 5,012 $ 1,766 Additions related to the current year 691 1,723 3,246 Decreases related to prior year tax positions (86 ) (1,734 ) — Ending balance of unrecognized tax benefits $ 5,606 $ 5,001 $ 5,012 |
Basic and Diluted Net Loss Pe_2
Basic and Diluted Net Loss Per Share (Tables) | 12 Months Ended |
Dec. 31, 2018 | |
Earnings Per Share [Abstract] | |
Basic Loss Per Share | The following table summarizes the distributable losses for the years ended December 31, 2018, 2017 and 2016: Year Ended December 31, 2018 2017 2016 Net loss $ (225,821 ) $ (121,559 ) $ (83,217 ) Preferred stock dividend — (20,100 ) — Distributions to Ionis (7,792 ) — — Distributable losses $ (233,613 ) $ (141,659 ) $ (83,217 ) The following table summarizes the reconciliation of weighted-average shares outstanding used in the calculation of basic loss per share for the years ended December 31, 2018, 2017 and 2016: Year Ended December 31, Determination of shares: 2018 2017 2016 Weighted-average preferred shares outstanding — 15,748,009 28,884,540 Weighted-average common shares outstanding owned by Ionis 59,812,394 20,669,446 — Weighted-average common shares outstanding owned by others 21,553,407 9,593,322 — Total weighted-average shares outstanding 81,365,801 46,010,777 28,884,540 The following table summarizes the calculation of basic loss per share for the years ended December 31, 2018, 2017 and 2016 (in thousands, except per share amounts): Year Ended December 31, 2018 2017 2016 Losses attributable to preferred shares $ — $ (48,485 ) $ (83,217 ) Less: Assumed dividend to preferred shares — 20,100 — Income (losses) allocated to preferred shares — (28,385 ) (83,217 ) Weighted-average preferred shares outstanding — 15,748,009 28,884,540 Basic loss per preferred share $ — $ (1.80 ) $ (2.88 ) Losses allocated to Ionis $ (171,730 ) $ (63,638 ) $ — Plus: Distribution to Ionis 7,792 — — Losses available to Ionis (163,938 ) (63,638 ) - Weighted-average common shares outstanding owned by Ionis 59,812,394 20,669,446 — Basic loss per common share owned by Ionis $ (2.74 ) $ (3.08 ) $ — Losses allocated to common shares owned by others $ (61,883 ) $ (29,536 ) $ — Weighted-average common shares outstanding owned by others 21,553,407 9,593,322 — Basic loss per common share owned by others $ (2.87 ) $ (3.08 ) $ — |
Contractual Obligations and C_2
Contractual Obligations and Commitments (Tables) | 12 Months Ended |
Dec. 31, 2018 | |
Commitments And Contingencies Disclosure [Abstract] | |
Contractual Obligation | The following table summarizes the contractual obligations as of December 31, 2018 (in thousands): Payments due by period Year Ending December 31, Total Less than 1 year 1 to 3 years 3 to 5 years More than 5 years Operating lease obligations $ 23,688 $ 2,308 $ 4,711 $ 4,805 $ 11,864 Purchase commitments 5,033 5,033 - - - Total $ 28,721 $ 7,341 $ 4,711 $ 4,805 $ 11,864 |
Restructuring (Tables)
Restructuring (Tables) | 12 Months Ended |
Dec. 31, 2018 | |
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): Year Ended December 31, 2018 Restructuring reserve beginning balance $ — Restructuring expenses incurred during the period 1,889 Adjustments during the period (150 ) Amounts paid during the period (1,696 ) Restructuring reserve ending balance $ 43 |
Quarterly Financial Data (Una_2
Quarterly Financial Data (Unaudited) (Tables) | 12 Months Ended |
Dec. 31, 2018 | |
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, 2018 and 2017 are as follows (in thousands, except per share data): 2018 Quarters First Quarter Second Quarter Third Quarter Fourth Quarter Revenue: Commercial revenue: Product revenue $ — $ — $ — $ 2,237 Licensing revenue — — 12,000 — Total commercial revenue — — 12,000 2,237 Research and development revenue under collaborative agreement 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 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 Interest expense — — — — Other income (expense) (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 preferred stock, basic and diluted $ — $ — $ — $ — Weighted-average shares of preferred stock outstanding, basic and diluted — — — — Net loss per share of common stock owned by Ionis, basic and diluted $ (0.44 ) $ (0.72 ) $ (0.73 ) $ (0.79 ) Weighted-average shares of common stock outstanding owned by Ionis, basic and diluted 45,447,879 60,832,494 65,538,467 67,129,553 Net loss per share of common stock owned by others, basic and diluted $ (0.44 ) $ (0.85 ) $ (0.73 ) $ (0.79 ) Weighted-average shares of common stock outstanding owned by others, basic and diluted 21,171,372 21,492,157 21,671,415 21,869,713 2017 Quarters First Quarter Second Quarter Third Quarter Fourth Quarter (as revised) Revenue: Commercial revenue: Product revenue $ — $ — $ — $ — Licensing revenue — — — — Total commercial revenue — — — — Research and development revenue under collaborative agreement 6,094 5,713 9,906 21,688 Total revenue 6,094 5,713 9,906 21,688 Expenses: Cost of sales - product — — — — Cost of sales - intangible asset amortization — — — — Cost of license — — — — Research and development 64,794 18,487 17,640 25,968 Selling, general and administrative 4,676 6,915 8,373 17,018 Total expenses 69,470 25,402 26,013 42,986 Loss from operations (63,376 ) (19,689 ) (16,107 ) (21,298 ) Other income (expense): Investment income 61 245 687 819 Interest expense (541 ) (965 ) (224 ) — Other income (expense) — 50 73 (19 ) Loss before income tax expense (63,856 ) (20,359 ) (15,571 ) (20,498 ) Income tax expense — — (2,066 ) 791 Net loss $ (63,856 ) $ (20,359 ) $ (17,637 ) $ (19,707 ) Net loss per share of preferred stock, basic and diluted $ (2.21 ) $ (0.70 ) $ (0.01 ) $ — Weighted-average shares of preferred stock outstanding, basic and diluted 28,884,540 28,888,450 5,651,323 — Net loss per share of common stock owned by Ionis, basic and diluted $ — $ — $ (0.33 ) $ (0.30 ) Weighted-average shares of common stock outstanding owned by Ionis, basic and diluted — — 36,555,903 45,447,879 Net loss per share of common stock owned by others, basic and diluted $ — $ — $ (0.33 ) $ (0.30 ) Weighted-average shares of common stock outstanding owned by others, basic and diluted — — 16,966,712 21,093,750 (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) For the purposes of calculating EPS under the two-class method since our IPO in July 2017, we have allocated the net loss between the common stock and the Series A convertible preferred stock for the three-month period ended September 30, 2017. We determined it was appropriate to allocate losses to the Series A convertible preferred stock because it was the lowest form of subordinated equity during such period and because Ionis, the sole holder of the Series A convertible preferred stock, was absorbing our losses during such period. Basic EPS for each class of stock is computed by dividing total distributable losses applicable to preferred and common stock, 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 losses for the quarter ended September 30, 2017 (in thousands): September 30, 2017 (as revised) Net loss $ (17,637 ) Preferred stock dividend (1,791 ) Distributable losses $ (19,428 ) The following table summarizes the reconciliation of weighted-average shares outstanding used in the calculation of basic EPS for the quarter ended September 30, 2017: September 30, 2017 Determination of shares: Weighted-average preferred shares outstanding 5,651,323 Weighted-average common shares outstanding 53,522,615 Total weighted-average shares outstanding 59,173,938 The following table summarizes the calculation of basic EPS for the quarter ended September 30, 2017 (in thousands, except per share amounts): September 30, 2017 (as revised) Losses attributable to preferred shares $ (1,855 ) Less: Assumed dividend to preferred shares 1,791 Income (losses) allocated to preferred shares $ (64 ) Weighted-average preferred shares outstanding 5,651,323 Basic income (loss) per preferred share $ 0.01 Losses allocated to common shares $ (17,573 ) Weighted-average common shares outstanding 53,522,615 Basic loss per common share $ (0.33 ) |
Organization and Basis of Pre_2
Organization and Basis of Presentation (Details) - USD ($) $ in Thousands | 3 Months Ended | 12 Months Ended | |||||||||
Dec. 31, 2018 | Sep. 30, 2018 | Jun. 30, 2018 | Mar. 31, 2018 | Dec. 31, 2017 | Sep. 30, 2017 | Jun. 30, 2017 | Mar. 31, 2017 | Dec. 31, 2018 | Dec. 31, 2017 | Dec. 31, 2016 | |
Basis of Presentation and Organization [Abstract] | |||||||||||
Accumulated deficit | $ 522,042 | $ 296,221 | $ 522,042 | $ 296,221 | |||||||
Net loss | 70,557 | $ 63,591 | $ 62,046 | $ 29,627 | $ 19,707 | $ 17,637 | $ 20,359 | $ 63,856 | 225,821 | 121,559 | $ 83,217 |
Net cash used in operating activities | 202,888 | $ 36,190 | $ 55,361 | ||||||||
Cash, cash equivalents and short-term investments | 252,600 | 252,600 | |||||||||
License fee receivable | $ 150,000 | $ 150,000 | |||||||||
Ionis [Member] | Akcea [Member] | |||||||||||
Basis of Presentation and Organization [Abstract] | |||||||||||
Ownership percentage by Ionis | 75.00% | 75.00% |
Summary of Significant Accoun_4
Summary of Significant Accounting Policies - Narratives (Details) | Dec. 31, 2018USD ($) |
Inventory [Abstract] | |
Finished goods | $ 85,000 |
Summary of Significant Accoun_5
Summary of Significant Accounting Policies, Property, Plant and Equipment (Details) | 12 Months Ended |
Dec. 31, 2018 | |
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 |
Summary of Significant Accoun_6
Summary of Significant Accounting Policies - Condensed Consolidated Balance Sheets (Details) - USD ($) $ in Thousands | Dec. 31, 2018 | Dec. 31, 2017 |
Condensed Consolidated Balance Sheets [Abstract] | ||
Current portion of deferred revenue | $ 25,354 | $ 58,192 |
Long-term portion of deferred revenue | 3,434 | 12,501 |
Accumulated deficit | $ (522,042) | (296,221) |
As Originally Reported Under Topic 605 [Member] | ASU 2014-09 [Member] | ||
Condensed Consolidated Balance Sheets [Abstract] | ||
Current portion of deferred revenue | 50,579 | |
Long-term portion of deferred revenue | 8,306 | |
Accumulated deficit | (284,413) | |
Effect of Change [Member] | ASU 2014-09 [Member] | ||
Condensed Consolidated Balance Sheets [Abstract] | ||
Current portion of deferred revenue | 7,613 | |
Long-term portion of deferred revenue | 4,195 | |
Accumulated deficit | $ (11,808) |
Summary of Significant Accoun_7
Summary of Significant Accounting Policies - Condensed Consolidated Statements of Operations and Comprehensive Loss (Details) - USD ($) $ / shares in Units, $ in Thousands | 3 Months Ended | 12 Months Ended | |||||||||
Dec. 31, 2018 | Sep. 30, 2018 | Jun. 30, 2018 | Mar. 31, 2018 | Dec. 31, 2017 | Sep. 30, 2017 | Jun. 30, 2017 | Mar. 31, 2017 | Dec. 31, 2018 | Dec. 31, 2017 | Dec. 31, 2016 | |
Condensed Consolidated Statements of Operations and Comprehensive Loss [Abstract] | |||||||||||
Revenue | $ 10,197 | $ 19,241 | $ 18,321 | $ 17,108 | $ 21,688 | $ 9,906 | $ 5,713 | $ 6,094 | $ 64,867 | $ 43,401 | $ 0 |
Loss from operations | (72,058) | (65,008) | (63,423) | (30,327) | (21,298) | (16,107) | (19,689) | (63,376) | (230,816) | (120,470) | (83,512) |
Net loss | $ (70,557) | $ (63,591) | $ (62,046) | $ (29,627) | $ (19,707) | (17,637) | $ (20,359) | $ (63,856) | (225,821) | (121,559) | (83,217) |
Preferred Stock [Member] | |||||||||||
Condensed Consolidated Statements of Operations and Comprehensive Loss [Abstract] | |||||||||||
Net loss | $ (64) | $ 0 | $ (28,385) | $ (83,217) | |||||||
Net loss per share, basic and diluted (in dollars per share) | $ (0.01) | $ (0.70) | $ (2.21) | $ 0 | $ (1.80) | $ (2.88) | |||||
Common Stock [Member] | |||||||||||
Condensed Consolidated Statements of Operations and Comprehensive Loss [Abstract] | |||||||||||
Net loss | $ (17,573) | ||||||||||
Common Stock [Member] | Ionis [Member] | |||||||||||
Condensed Consolidated Statements of Operations and Comprehensive Loss [Abstract] | |||||||||||
Net loss | $ (163,938) | $ (63,638) | $ 0 | ||||||||
Net loss per share, basic and diluted (in dollars per share) | $ (0.79) | $ (0.73) | $ (0.72) | $ (0.44) | $ (0.30) | $ (0.33) | $ (2.74) | $ (3.08) | $ 0 | ||
Common Stock [Member] | Others [Member] | |||||||||||
Condensed Consolidated Statements of Operations and Comprehensive Loss [Abstract] | |||||||||||
Net loss per share, basic and diluted (in dollars per share) | $ (0.79) | $ (0.73) | $ (0.85) | $ (0.44) | $ (0.30) | $ (0.33) | $ (2.87) | $ (3.08) | $ 0 | ||
Research and Development Revenue Under Collaborative Agreement [Member] | |||||||||||
Condensed Consolidated Statements of Operations and Comprehensive Loss [Abstract] | |||||||||||
Revenue | $ 7,960 | $ 7,241 | $ 18,321 | $ 17,108 | $ 21,688 | $ 9,906 | $ 5,713 | $ 6,094 | $ 50,630 | $ 43,401 | $ 0 |
As Originally Reported Under Topic 605 [Member] | ASU 2014-09 [Member] | |||||||||||
Condensed Consolidated Statements of Operations and Comprehensive Loss [Abstract] | |||||||||||
Loss from operations | (108,662) | ||||||||||
Net loss | $ (109,751) | ||||||||||
As Originally Reported Under Topic 605 [Member] | ASU 2014-09 [Member] | Preferred Stock [Member] | |||||||||||
Condensed Consolidated Statements of Operations and Comprehensive Loss [Abstract] | |||||||||||
Net loss per share, basic and diluted (in dollars per share) | $ (1.55) | ||||||||||
As Originally Reported Under Topic 605 [Member] | ASU 2014-09 [Member] | Common Stock [Member] | Ionis [Member] | |||||||||||
Condensed Consolidated Statements of Operations and Comprehensive Loss [Abstract] | |||||||||||
Net loss per share, basic and diluted (in dollars per share) | (2.82) | ||||||||||
As Originally Reported Under Topic 605 [Member] | ASU 2014-09 [Member] | Common Stock [Member] | Others [Member] | |||||||||||
Condensed Consolidated Statements of Operations and Comprehensive Loss [Abstract] | |||||||||||
Net loss per share, basic and diluted (in dollars per share) | $ (2.82) | ||||||||||
As Originally Reported Under Topic 605 [Member] | Research and Development Revenue Under Collaborative Agreement [Member] | ASU 2014-09 [Member] | |||||||||||
Condensed Consolidated Statements of Operations and Comprehensive Loss [Abstract] | |||||||||||
Revenue | $ 55,209 | ||||||||||
Effect of Change [Member] | ASU 2014-09 [Member] | |||||||||||
Condensed Consolidated Statements of Operations and Comprehensive Loss [Abstract] | |||||||||||
Loss from operations | (11,808) | ||||||||||
Net loss | $ (11,808) | ||||||||||
Effect of Change [Member] | ASU 2014-09 [Member] | Preferred Stock [Member] | |||||||||||
Condensed Consolidated Statements of Operations and Comprehensive Loss [Abstract] | |||||||||||
Net loss per share, basic and diluted (in dollars per share) | $ (0.25) | ||||||||||
Effect of Change [Member] | ASU 2014-09 [Member] | Common Stock [Member] | Ionis [Member] | |||||||||||
Condensed Consolidated Statements of Operations and Comprehensive Loss [Abstract] | |||||||||||
Net loss per share, basic and diluted (in dollars per share) | (0.26) | ||||||||||
Effect of Change [Member] | ASU 2014-09 [Member] | Common Stock [Member] | Others [Member] | |||||||||||
Condensed Consolidated Statements of Operations and Comprehensive Loss [Abstract] | |||||||||||
Net loss per share, basic and diluted (in dollars per share) | $ (0.26) | ||||||||||
Effect of Change [Member] | Research and Development Revenue Under Collaborative Agreement [Member] | ASU 2014-09 [Member] | |||||||||||
Condensed Consolidated Statements of Operations and Comprehensive Loss [Abstract] | |||||||||||
Revenue | $ (11,808) |
Summary of Significant Accoun_8
Summary of Significant Accounting Policies - Condensed Consolidated Statement of Cash Flows (Details) - USD ($) $ in Thousands | 3 Months Ended | 12 Months Ended | |||||||||
Dec. 31, 2018 | Sep. 30, 2018 | Jun. 30, 2018 | Mar. 31, 2018 | Dec. 31, 2017 | Sep. 30, 2017 | Jun. 30, 2017 | Mar. 31, 2017 | Dec. 31, 2018 | Dec. 31, 2017 | Dec. 31, 2016 | |
Condensed Consolidated Statement of Cash Flows [Abstract] | |||||||||||
Net loss | $ (70,557) | $ (63,591) | $ (62,046) | $ (29,627) | $ (19,707) | $ (17,637) | $ (20,359) | $ (63,856) | $ (225,821) | $ (121,559) | $ (83,217) |
Adjustments to reconcile net loss to net cash used in operating activities: | |||||||||||
Deferred revenue | (41,905) | 70,693 | |||||||||
Cash, cash equivalents and restricted cash at beginning of period | 58,367 | 7,857 | 58,367 | 7,857 | |||||||
Cash, cash equivalents and restricted at end of period | $ 86,454 | 58,367 | 86,454 | 58,367 | 7,857 | ||||||
As Originally Reported Under Topic 605 [Member] | ASU 2014-09 [Member] | |||||||||||
Condensed Consolidated Statement of Cash Flows [Abstract] | |||||||||||
Net loss | (109,751) | ||||||||||
Adjustments to reconcile net loss to net cash used in operating activities: | |||||||||||
Deferred revenue | 58,885 | ||||||||||
Cash, cash equivalents and restricted cash at beginning of period | 58,367 | 7,857 | 58,367 | 7,857 | |||||||
Cash, cash equivalents and restricted at end of period | 58,367 | 58,367 | 7,857 | ||||||||
Effect of Change [Member] | ASU 2014-09 [Member] | |||||||||||
Condensed Consolidated Statement of Cash Flows [Abstract] | |||||||||||
Net loss | (11,808) | ||||||||||
Adjustments to reconcile net loss to net cash used in operating activities: | |||||||||||
Deferred revenue | 11,808 | ||||||||||
Cash, cash equivalents and restricted cash at beginning of period | $ 0 | $ 0 | $ 0 | 0 | |||||||
Cash, cash equivalents and restricted at end of period | $ 0 | $ 0 | $ 0 |
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, 2018 | Sep. 30, 2018 | Jun. 30, 2018 | Mar. 31, 2018 | Dec. 31, 2017 | Sep. 30, 2017 | Jun. 30, 2017 | Mar. 31, 2017 | Dec. 31, 2018 | Dec. 31, 2017 | Dec. 31, 2016 | |
Disaggregation of Revenue [Abstract] | |||||||||||
Revenue, net | $ 10,197 | $ 19,241 | $ 18,321 | $ 17,108 | $ 21,688 | $ 9,906 | $ 5,713 | $ 6,094 | $ 64,867 | $ 43,401 | $ 0 |
TEGSEDI [Member] | |||||||||||
Disaggregation of Revenue [Abstract] | |||||||||||
Revenue, net | 2,200 | ||||||||||
TEGSEDI [Member] | U.S. [Member] | |||||||||||
Disaggregation of Revenue [Abstract] | |||||||||||
Revenue, net | 1,200 | ||||||||||
TEGSEDI [Member] | Germany [Member] | |||||||||||
Disaggregation of Revenue [Abstract] | |||||||||||
Revenue, net | $ 1,000 | ||||||||||
Maximum [Member] | |||||||||||
Summary Of Significant Accounting Policies [Line Items] | |||||||||||
Period of time after billing when payment is received | 1 year | ||||||||||
Payment terms | 90 days | ||||||||||
Amortization period of assets incremental cost of obtaining contract recognized as expense | 1 year | ||||||||||
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) $ in Thousands | 12 Months Ended |
Dec. 31, 2018USD ($) | |
Revenue Allowance and Reserves Balance [Abstract] | |
Provision related to current period sales | $ 348 |
Balance at December 31, 2018 | 348 |
Chargebacks, Discounts and Fees [Member] | |
Revenue Allowance and Reserves Balance [Abstract] | |
Provision related to current period sales | 50 |
Balance at December 31, 2018 | 50 |
Government and Other Rebates [Member] | |
Revenue Allowance and Reserves Balance [Abstract] | |
Provision related to current period sales | 293 |
Balance at December 31, 2018 | 293 |
Returns [Member] | |
Revenue Allowance and Reserves Balance [Abstract] | |
Provision related to current period sales | 5 |
Balance at December 31, 2018 | $ 5 |
Summary of Significant Accou_11
Summary of Significant Accounting Policies, Stock-Based Compensation Expense (Details) | 12 Months Ended |
Dec. 31, 2018 | |
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] | Employee Stock Options [Member] | One Year from Date of Grant [Member] | |
Stock-Based Compensation [Abstract] | |
Vesting percentage | 25.00% |
2015 Equity Incentive Plan [Member] | Board of Director Stock Options [Member] | |
Stock-Based Compensation [Abstract] | |
Vesting period | 4 years |
Award term | 10 years |
Summary of Significant Accou_12
Summary of Significant Accounting Policies, Accumulated Other Comprehensive Loss (Details) - USD ($) $ in Thousands | 12 Months Ended | |||
Dec. 31, 2018 | Dec. 31, 2017 | Dec. 31, 2016 | ||
Accumulated Other Comprehensive Loss [Roll Forward] | ||||
Beginning balance | $ 167,825 | |||
Net other comprehensive income (loss) | 127 | $ (430) | $ 54 | |
Ending balance | 276,724 | 167,825 | ||
Tax benefit included in other comprehensive income (loss) | 0 | 0 | 0 | |
Accumulated Other Comprehensive Loss [Member] | ||||
Accumulated Other Comprehensive Loss [Roll Forward] | ||||
Beginning balance | (451) | (21) | (75) | |
Ending balance | (324) | (451) | (21) | |
Unrealized Gains (Losses) on Investments [Member] | ||||
Accumulated Other Comprehensive Loss [Roll Forward] | ||||
Unrealized gains (losses) on investments, net of tax | [1] | 144 | (337) | 75 |
Currency Translation Adjustment [Member] | ||||
Accumulated Other Comprehensive Loss [Roll Forward] | ||||
Unrealized gains (losses) on investments, net of tax | $ (17) | $ (93) | $ (21) | |
[1] | There was no tax benefit for other comprehensive income (loss) for the years ended December 31, 2018, 2017 and 2016. |
Summary of Significant Accou_13
Summary of Significant Accounting Policies, Income Taxes (Details) | 12 Months Ended |
Dec. 31, 2018 | |
Income Taxes [Abstract] | |
Tax cuts and jobs act of 2017 measurement period | 1 year |
Investments and Fair Value Me_3
Investments and Fair Value Measurements - Summary of Investments (Details) - USD ($) $ in Thousands | Dec. 31, 2018 | Dec. 31, 2017 |
Summary of Investments [Abstract] | ||
Cost | $ 202,100 | |
Gross unrealized gains | 0 | |
Gross unrealized losses | (337) | |
Estimated fair value | 201,763 | |
Securities with Maturity of One Year or Less [Member] | ||
Summary of Investments [Abstract] | ||
Cost | $ 167,348 | 170,569 |
Gross unrealized gains | 0 | |
Gross unrealized losses | (193) | (265) |
Estimated fair value | 167,155 | 170,304 |
Securities with Maturity of One to Two Years [Member] | ||
Summary of Investments [Abstract] | ||
Cost | 31,531 | |
Gross unrealized gains | 0 | |
Gross unrealized losses | (72) | |
Estimated fair value | 31,459 | |
Corporate Debt Securities [Member] | Securities with Maturity of One Year or Less [Member] | ||
Summary of Investments [Abstract] | ||
Cost | 81,770 | 132,434 |
Gross unrealized gains | 0 | |
Gross unrealized losses | (151) | (206) |
Estimated fair value | 81,619 | 132,228 |
Corporate Debt Securities [Member] | Securities with Maturity of One to Two Years [Member] | ||
Summary of Investments [Abstract] | ||
Cost | 8,267 | |
Gross unrealized gains | 0 | |
Gross unrealized losses | (35) | |
Estimated fair value | 8,232 | |
Debt Securities Issued by U.S. Government Agencies [Member] | Securities with Maturity of One Year or Less [Member] | ||
Summary of Investments [Abstract] | ||
Cost | 85,578 | 38,135 |
Gross unrealized gains | 0 | |
Gross unrealized losses | (42) | (59) |
Estimated fair value | $ 85,536 | 38,076 |
Debt Securities Issued by U.S. Government Agencies [Member] | Securities with Maturity of One to Two Years [Member] | ||
Summary of Investments [Abstract] | ||
Cost | 23,264 | |
Gross unrealized gains | 0 | |
Gross unrealized losses | (37) | |
Estimated fair value | $ 23,227 |
Investments and Fair Value Me_4
Investments and Fair Value Measurements - Fair Value Measurements (Details) - Recurring Basis [Member] - USD ($) $ in Thousands | Dec. 31, 2018 | Dec. 31, 2017 | |
Fair Value Measurements [Abstract] | |||
Money market funds | [1] | $ 82,343 | $ 48,430 |
Total | 249,498 | 250,193 | |
Corporate Debt Securities [Member] | |||
Fair Value Measurements [Abstract] | |||
Available-for-sale securities | [2] | 81,619 | 140,460 |
Corporate Debt Securities [Member] | Cash and Cash Equivalents [Member] | |||
Fair Value Measurements [Abstract] | |||
Available-for-sale securities | 1,000 | ||
Debt Securities Issued by U.S. Government Agencies [Member] | |||
Fair Value Measurements [Abstract] | |||
Available-for-sale securities | [3] | 85,536 | 61,303 |
Quoted Prices in Active Markets (Level 1) [Member] | |||
Fair Value Measurements [Abstract] | |||
Money market funds | [1] | 82,343 | 48,430 |
Total | 82,343 | 48,430 | |
Quoted Prices in Active Markets (Level 1) [Member] | Corporate Debt Securities [Member] | |||
Fair Value Measurements [Abstract] | |||
Available-for-sale securities | [2] | 0 | |
Quoted Prices in Active Markets (Level 1) [Member] | Debt Securities Issued by U.S. Government Agencies [Member] | |||
Fair Value Measurements [Abstract] | |||
Available-for-sale securities | [3] | 0 | |
Significant Other Observable Inputs (Level 2) [Member] | |||
Fair Value Measurements [Abstract] | |||
Money market funds | [1] | 0 | |
Total | 167,155 | 201,763 | |
Significant Other Observable Inputs (Level 2) [Member] | Corporate Debt Securities [Member] | |||
Fair Value Measurements [Abstract] | |||
Available-for-sale securities | [2] | 81,619 | 140,460 |
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] | $ 85,536 | $ 61,303 |
[1] | Included in cash and cash equivalents on our consolidated balance sheets. | ||
[2] | At December 31, 2018, $1.0 million was included in cash and cash equivalents on our consolidated balance sheet, with the difference included in short-term investments on our consolidated balance sheet. | ||
[3] | Included in short-term investments on our consolidated balance sheets. |
Property Plant and Equipment -
Property Plant and Equipment - Summary of Property and Equipment at Cost and Related Accumulated Depreciation (Details) - USD ($) $ in Thousands | Dec. 31, 2018 | Dec. 31, 2017 |
Property Plant And Equipment [Line Items] | ||
Property and equipment, at cost | $ 5,926 | $ 198 |
Less accumulated depreciation and amortization | (230) | (121) |
Total property and equipment, net | 5,696 | 77 |
Furniture And Fixtures [Member] | ||
Property Plant And Equipment [Line Items] | ||
Property and equipment, at cost | 1,611 | 183 |
Computer Equipment And Software [Member] | ||
Property Plant And Equipment [Line Items] | ||
Property and equipment, at cost | 102 | $ 15 |
Leasehold Improvements [Member] | ||
Property Plant And Equipment [Line Items] | ||
Property and equipment, at cost | $ 4,213 |
Property Plant Equipment - Narr
Property Plant Equipment - Narratives (Details) - USD ($) | 12 Months Ended | ||
Dec. 31, 2018 | Dec. 31, 2017 | Dec. 31, 2016 | |
Property Plant And Equipment [Abstract] | |||
Depreciation | $ 307,000 | $ 108,000 | $ 12,000 |
Tenant improvement allowance | $ 3,600,000 |
Intangible Assets - Schedule of
Intangible Assets - Schedule of Intangible Assets (Details) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2018 | Dec. 31, 2017 | |
Finite Lived Intangible Assets [Line Items] | ||
Less accumulated amortization | $ (3,348) | $ (478) |
Total intangible assets, net | 88,914 | 1,221 |
Acquired and In-licensed Rights [Member] | ||
Finite Lived Intangible Assets [Line Items] | ||
Intangible assets, gross | $ 2,262 | $ 1,699 |
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 | |
Estimated useful life | 16 years |
Intangible Assets - Additional
Intangible Assets - Additional Information (Details) - USD ($) $ in Thousands | Oct. 05, 2018 | Jul. 11, 2018 | Dec. 31, 2018 | Dec. 31, 2017 | Dec. 31, 2016 |
Finite Lived Intangible Assets [Line Items] | |||||
Amortization of licenses | $ 2,870 | $ 120 | $ 119 | ||
Patents [Member] | |||||
Finite Lived Intangible Assets [Line Items] | |||||
Weighted average remaining amortizable life | 12 years 1 month 24 days | ||||
Ionis [Member] | Capitalized Regulatory Milestones [Member] | Inotersen [Member] | |||||
Finite Lived Intangible Assets [Line Items] | |||||
Milestone paid | $ 50,000 | $ 40,000 |
Intangible Assets - Schedule _2
Intangible Assets - Schedule of Estimated Future Amortization Expenses for Intangible Assets (Details) - USD ($) $ in Thousands | Dec. 31, 2018 | Dec. 31, 2017 |
Goodwill And Intangible Assets Disclosure [Abstract] | ||
2,019 | $ 5,863 | |
2,020 | 5,879 | |
2,021 | 5,861 | |
2,022 | 5,856 | |
2,023 | 5,843 | |
Thereafter | 59,613 | |
Total intangible assets, net | $ 88,914 | $ 1,221 |
Strategic Collaboration with _2
Strategic Collaboration with Novartis (Details) | Jul. 19, 2017USD ($) | Jul. 31, 2017USD ($) | Jan. 31, 2017DrugPerformanceObligation | Dec. 31, 2018USD ($) | Sep. 30, 2018USD ($) | Jun. 30, 2018USD ($) | Mar. 31, 2018USD ($) | Dec. 31, 2017USD ($) | Sep. 30, 2017USD ($) | Jun. 30, 2017USD ($) | Mar. 31, 2017USD ($) | Dec. 31, 2018USD ($) | Dec. 31, 2017USD ($) | Dec. 31, 2016USD ($) |
Strategic Collaboration with Novartis [Abstract] | ||||||||||||||
Proceeds from sale of common stock to Novartis in a private placement | $ 50,000,000 | $ 50,000,000 | ||||||||||||
Revenue | $ 10,197,000 | $ 19,241,000 | $ 18,321,000 | $ 17,108,000 | $ 21,688,000 | $ 9,906,000 | $ 5,713,000 | $ 6,094,000 | $ 64,867,000 | 43,401,000 | $ 0 | |||
Revenue recognized from deferred revenue | 42,200,000 | |||||||||||||
AKCEA-APO(a)-L [Member] | ||||||||||||||
Strategic Collaboration with Novartis [Abstract] | ||||||||||||||
Maximum amount of payments receivable for milestones | 675,000,000 | 675,000,000 | ||||||||||||
Maximum amount of payments receivable for development milestones | 25,000,000 | 25,000,000 | ||||||||||||
Maximum amount of payments receivable for regulatory milestones | 290,000,000 | 290,000,000 | ||||||||||||
Maximum amount of payments receivable for commercialization milestones | 360,000,000 | $ 360,000,000 | ||||||||||||
Royalty percentage received on sales of drug | 20.00% | |||||||||||||
AKCEA-APOCIII-L [Member] | ||||||||||||||
Strategic Collaboration with Novartis [Abstract] | ||||||||||||||
Maximum amount of payments receivable for milestones | 530,000,000 | $ 530,000,000 | ||||||||||||
Maximum amount of payments receivable for development milestones | 25,000,000 | 25,000,000 | ||||||||||||
Maximum amount of payments receivable for regulatory milestones | 240,000,000 | 240,000,000 | ||||||||||||
Maximum amount of payments receivable for commercialization milestones | 265,000,000 | $ 265,000,000 | ||||||||||||
Royalty percentage received on sales of drug | 20.00% | |||||||||||||
Development Services for AKCEA-APO(a)-L [Member] | ||||||||||||||
Strategic Collaboration with Novartis [Abstract] | ||||||||||||||
Transaction price | 64,000,000 | |||||||||||||
Development Services for AKCEA-APOCIII-L [Member] | ||||||||||||||
Strategic Collaboration with Novartis [Abstract] | ||||||||||||||
Transaction price | 40,100,000 | |||||||||||||
Delivery of AKCEA-APO(a)-L [Member] | ||||||||||||||
Strategic Collaboration with Novartis [Abstract] | ||||||||||||||
Transaction price | 1,500,000 | |||||||||||||
Delivery of AKCEA-APOCIII-L [Member] | ||||||||||||||
Strategic Collaboration with Novartis [Abstract] | ||||||||||||||
Transaction price | 2,800,000 | |||||||||||||
Research and Development Revenue Under Collaborative Agreement [Member] | ||||||||||||||
Strategic Collaboration with Novartis [Abstract] | ||||||||||||||
Revenue | 7,960,000 | $ 7,241,000 | $ 18,321,000 | $ 17,108,000 | 21,688,000 | $ 9,906,000 | $ 5,713,000 | 6,094,000 | $ 50,630,000 | 43,401,000 | 0 | |||
Novartis [Member] | ||||||||||||||
Strategic Collaboration with Novartis [Abstract] | ||||||||||||||
Upfront payment received | 75,000,000 | |||||||||||||
Portion of upfront payment retained | 60,000,000 | |||||||||||||
Portion of upfront payment paid as sublicense fee to Ionis | 15,000,000 | |||||||||||||
License fee receivable per drug | 150,000,000 | 150,000,000 | ||||||||||||
Next prospective milestone | 25,000,000 | $ 25,000,000 | ||||||||||||
Percentage of license fees, milestone payments and royalties paid as sublicense fee to lonis | 50.00% | |||||||||||||
Transaction price | 108,400,000 | |||||||||||||
Premium received on shares issued by lonis | 28,400,000 | |||||||||||||
Potential premium received if Ionis common stock is purchased in the future | $ 5,000,000 | |||||||||||||
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 | $ 50,000,000 | |||||||||||||
Deferred revenue | $ 28,800,000 | $ 70,700,000 | $ 28,800,000 | 70,700,000 | ||||||||||
Number of separate performance obligations | PerformanceObligation | 4 | |||||||||||||
Novartis [Member] | Research and Development Revenue Under Collaborative Agreement [Member] | ||||||||||||||
Strategic Collaboration with Novartis [Abstract] | ||||||||||||||
Revenue | $ 50,600,000 | $ 43,400,000 | $ 0 | |||||||||||
Novartis [Member] | Research and Development Revenue Under Collaborative Agreement [Member] | Revenue [Member] | Strategic Partner [Member] | ||||||||||||||
Strategic Collaboration with Novartis [Abstract] | ||||||||||||||
Concentration percentage | 100.00% | 100.00% | ||||||||||||
Novartis [Member] | Minimum [Member] | ||||||||||||||
Strategic Collaboration with Novartis [Abstract] | ||||||||||||||
Number of drugs with exclusive option that could be exercised | Drug | 1 |
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, 2018USD ($)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 |
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) | Oct. 17, 2018USD ($)shares | Aug. 03, 2018USD ($)shares | Apr. 17, 2018USD ($)Milestoneshares | Dec. 31, 2018USD ($)shares | Sep. 30, 2018USD ($) | Jun. 30, 2018USD ($) | Mar. 31, 2018USD ($) | Dec. 31, 2017USD ($)shares | Sep. 30, 2017USD ($) | Jun. 30, 2017USD ($) | Mar. 31, 2017USD ($) | Dec. 31, 2018USD ($)shares | Dec. 31, 2017USD ($)shares | Dec. 31, 2016USD ($) | Apr. 16, 2018shares |
TTR Development, Commercialization, Collaboration and License Agreement [Abstract] | |||||||||||||||
Stock purchased by Ionis | $ 135,438,000 | ||||||||||||||
Common stock, shares authorized (in shares) | shares | 125,000,000 | 100,000,000 | 125,000,000 | 100,000,000 | |||||||||||
Licenses, net | $ 88,914,000 | $ 1,221,000 | $ 88,914,000 | $ 1,221,000 | |||||||||||
Research and development expense | 33,532,000 | $ 29,381,000 | $ 39,457,000 | $ 27,970,000 | $ 25,968,000 | $ 17,640,000 | $ 18,487,000 | $ 64,794,000 | 130,340,000 | 126,890,000 | $ 68,459,000 | ||||
Amortization expense of milestone payment | 2,012,000 | $ 701,000 | 2,713,000 | 0 | 0 | ||||||||||
TTR License Agreement [Member] | |||||||||||||||
TTR Development, Commercialization, Collaboration and License Agreement [Abstract] | |||||||||||||||
Research and development expense | $ 26,000,000 | 0 | 0 | ||||||||||||
Ionis [Member] | |||||||||||||||
TTR Development, Commercialization, Collaboration and License Agreement [Abstract] | |||||||||||||||
Costs incurred by Ionis prior to closing of TTR Agreement | $ 3,100,000 | ||||||||||||||
Ionis [Member] | TTR License Agreement [Member] | |||||||||||||||
TTR Development, Commercialization, Collaboration and License Agreement [Abstract] | |||||||||||||||
Upfront licensing fee paid to Ionis | $ 150,000,000 | ||||||||||||||
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 | $ 750,000,000 | ||||||||||||||
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% | ||||||||||||||
Notice period for termination of agreement | 90 days | ||||||||||||||
Common stock shares issued to Ionis | shares | 10,700,000 | ||||||||||||||
Stock purchased by Ionis | $ 200,000,000 | ||||||||||||||
Common stock, shares authorized (in shares) | shares | 125,000,000 | 100,000,000 | |||||||||||||
Licenses, net | $ 600,000 | ||||||||||||||
Commercial inotersen inventory acquired from Ionis | $ 4,700,000 | ||||||||||||||
Ionis [Member] | TTR License Agreement [Member] | Inotersen [Member] | |||||||||||||||
TTR Development, Commercialization, Collaboration and License Agreement [Abstract] | |||||||||||||||
Maximum amount of payments payable for milestones | 110,000,000 | $ 110,000,000 | |||||||||||||
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 | 145,000,000 | 145,000,000 | |||||||||||||
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 | $ 1,300,000,000 | 1,300,000,000 | |||||||||||||
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 | $ 50,000,000 | $ 40,000,000 | |||||||||||||
Amortization expense of milestone payment | $ 2,700,000 | $ 0 | $ 0 | ||||||||||||
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, Services Agreement (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2018 | Dec. 31, 2017 | Dec. 31, 2016 | |
Services Agreement with Ionis [Abstract] | |||
Payment term after receipt of invoice | 30 days | ||
Term of extension for services agreement | 6 months | ||
Distributions to Ionis: | |||
Total distribution to Ionis | $ 7,792 | $ 0 | $ 0 |
Payable balance to Ionis at the beginning of the period | 14,365 | ||
Total amount payable to Ionis at period end | 18,901 | 14,365 | |
Ionis [Member] | Services Agreement [Member] | |||
Expenses: | |||
Services performed by Ionis | 15,404 | 9,742 | 8,599 |
Active pharmaceutical ingredient manufactured by Ionis | 5,229 | 6,012 | 12,648 |
Commercial inventory manufactured by Ionis | 1,288 | ||
Sublicensing expenses | 7,200 | 48,394 | |
Out-of-pocket expenses paid by Ionis | 50,870 | 37,426 | 42,367 |
Total operating expenses generated by transactions with Ionis | 79,991 | 101,574 | 63,614 |
Distributions to Ionis: | |||
Commercial inventory manufactured by Ionis | 4,707 | ||
Distribution to Ionis in connection with the TTR license transaction | 3,085 | ||
Total distribution to Ionis | 7,792 | ||
Total charges generated by transactions with Ionis | 87,783 | 101,574 | 63,614 |
Payable balance to Ionis at the beginning of the period | 14,365 | 24,355 | 9,198 |
Less: total amounts paid to Ionis during the period | (83,247) | (78,170) | (48,457) |
Less: non-cash sublicensing expenses | (33,394) | ||
Total amount payable to Ionis at period end | $ 18,901 | $ 14,365 | $ 24,355 |
Collaboration and License Agr_2
Collaboration and License Agreement with PTC Therapeutics - Additional Information (Details) | Aug. 01, 2018USD ($) | Dec. 31, 2018USD ($)PerformanceObligation | Sep. 30, 2018USD ($) | Jun. 30, 2018USD ($) | Mar. 31, 2018USD ($) | Dec. 31, 2017USD ($) | Sep. 30, 2017USD ($) | Jun. 30, 2017USD ($) | Mar. 31, 2017USD ($) | Dec. 31, 2018USD ($)PerformanceObligation | Dec. 31, 2017USD ($) | Dec. 31, 2016USD ($) |
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||
Revenue | $ 10,197,000 | $ 19,241,000 | $ 18,321,000 | $ 17,108,000 | $ 21,688,000 | $ 9,906,000 | $ 5,713,000 | $ 6,094,000 | $ 64,867,000 | $ 43,401,000 | $ 0 | |
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] | ||||||||||||
Revenue | 12,000,000 | |||||||||||
Deferred revenue | $ 0 | |||||||||||
Number of separate performance obligations | PerformanceObligation | 2 | 2 | ||||||||||
PTC Therapeutics [Member] | Akcea [Member] | ||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||
Upfront payment received | $ 12,000,000 | |||||||||||
Payment for sub-license royalty | 7,200,000 | |||||||||||
Upfront payment to be paid on earlier of FDA or EMA approval of WAYLIVRA | 6,000,000 | |||||||||||
Maximum amount of payments receivable per drug for regulatory milestones | $ 8,000,000 | |||||||||||
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% |
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, 2018 | Dec. 31, 2017 | 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, 2018 | Dec. 31, 2017 | 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, 2018shares | Dec. 31, 2017shares | Jul. 31, 2017shares | |
Stockholders' Equity (Deficit) [Abstract] | ||||
Common stock, shares authorized (in shares) | 125,000,000 | 100,000,000 | ||
Common stock, shares issued (in shares) | 89,345,978 | 66,541,629 | 66,541,629 | |
Common stock, shares outstanding (in shares) | 89,345,978 | 66,541,629 | 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, 2018 | Jan. 01, 2019 | Dec. 31, 2017 | Jul. 31, 2017 | Jun. 30, 2017 | |
Stock Plans [Abstract] | |||||
Shares of common stock added | 89,345,978 | 66,541,629 | 66,541,629 | ||
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) | 1,610,490 | ||||
2015 Equity Incentive Plan [Member] | Stock Options [Member] | |||||
Stock Plans [Abstract] | |||||
Options outstanding (in shares) | 11,010,828 | 7,905,000 | |||
Options exercisable (in shares) | 4,305,172 | ||||
2015 Equity Incentive Plan [Member] | RSUs [Member] | |||||
Stock Plans [Abstract] | |||||
Restricted stock units outstanding (in shares) | 38,134 | ||||
2017 Employee Stock Purchase Plan [Member] | |||||
Stock Plans [Abstract] | |||||
Number of shares of common stock reserved for issuance (in shares) | 1,000,000 | 500,000 | |||
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 | ||||
Shares issued under ESPP (in shares) | 31,551 | ||||
Accrued liability for contributions to employee stock purchase plan | $ 0.4 | ||||
2017 Employee Stock Purchase Plan [Member] | Subsequent Event [Member] | |||||
Stock Plans [Abstract] | |||||
Number of shares of common stock reserved for issuance (in shares) | 968,449 |
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, 2018 | Dec. 31, 2017 | Dec. 31, 2016 | |
Unrecognized Compensation Expense [Abstract] | |||
Unrecognized compensation expense related to non-vested stock options | $ 60,500 | ||
Weighted average period for recognition | 1 year 3 months 25 days | ||
Stock Options [Member] | |||
Number of Shares [Abstract] | |||
Outstanding at beginning of period (in shares) | 7,905,000 | ||
Granted (in shares) | 4,833,000 | ||
Exercised (in shares) | (834,800) | 0 | |
Cancelled/forfeited/expired (in shares) | (892,000) | ||
Outstanding at end of period (in shares) | 11,010,828 | 7,905,000 | |
Options exercisable (in shares) | 4,305,172 | ||
Weighted Average Exercise Price Per Share [Abstract] | |||
Outstanding at beginning of period (in dollars per share) | $ 9.64 | ||
Granted (in dollars per share) | 22.86 | ||
Exercised (in dollars per share) | 7.98 | ||
Cancelled/forfeited/expired (in dollars per share) | 16.53 | ||
Outstanding at end of period (in dollars per share) | 15 | $ 9.64 | |
Exercisable at end of period (in dollars per share) | $ 8.04 | ||
Average Remaining Contractual Term, Aggregate Intrinsic Value and Other [Abstract] | |||
Average remaining contractual term, Outstanding | 8 years 25 days | ||
Average remaining contractual term, Exercisable | 7 years 1 month 9 days | ||
Aggregate intrinsic value, Outstanding | $ 167,184 | ||
Aggregate intrinsic value, Exercisable | $ 95,124 | ||
Unrecognized Compensation Expense [Abstract] | |||
Weighted average fair value of options granted (in dollars per share) | $ 18.29 | $ 10.4 | $ 4.13 |
Exercise of common stock options (in shares) | 834,800 | 0 |
Equity and Stock-Based Compen_8
Equity and Stock-Based Compensation - Stock-Based Compensation Expense (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2018 | Dec. 31, 2017 | Dec. 31, 2016 | |
Stock-Based Compensation [Abstract] | |||
Stock-based compensation expense | $ 44,282 | $ 17,539 | $ 10,149 |
Cost of Sales Product [Member] | |||
Stock-Based Compensation [Abstract] | |||
Stock-based compensation expense | 160 | ||
Research and Development Expenses [Member] | |||
Stock-Based Compensation [Abstract] | |||
Stock-based compensation expense | 9,435 | 8,630 | 4,576 |
General and Administrative Expenses [Member] | |||
Stock-Based Compensation [Abstract] | |||
Stock-based compensation expense | $ 34,687 | $ 8,909 | $ 5,573 |
Equity and Stock-Based Compen_9
Equity and Stock-Based Compensation - Stock-based Valuation Information (Details) - 2015 Equity Incentive Plan [Member] | 12 Months Ended | ||
Dec. 31, 2018 | Dec. 31, 2017 | Dec. 31, 2016 | |
Employee Stock Options [Member] | |||
Weighted-Average Assumptions [Abstract] | |||
Risk-free interest rate | 2.80% | 1.90% | 1.60% |
Dividend yield | 0.00% | 0.00% | 0.00% |
Volatility | 77.10% | 79.50% | 71.40% |
Expected life | 6 years 29 days | 6 years 21 days | 6 years 29 days |
Board of Director Stock Options [Member] | |||
Weighted-Average Assumptions [Abstract] | |||
Risk-free interest rate | 2.90% | 1.90% | 2.00% |
Dividend yield | 0.00% | 0.00% | 0.00% |
Volatility | 78.20% | 79.40% | 79.60% |
Expected life | 6 years 5 months 1 day | 6 years 3 months | 6 years 29 days |
Income Taxes, Loss Before Incom
Income Taxes, Loss Before Income Taxes (Details) - USD ($) $ in Thousands | 3 Months Ended | 12 Months Ended | |||||||||
Dec. 31, 2018 | Sep. 30, 2018 | Jun. 30, 2018 | Mar. 31, 2018 | Dec. 31, 2017 | Sep. 30, 2017 | Jun. 30, 2017 | Mar. 31, 2017 | Dec. 31, 2018 | Dec. 31, 2017 | Dec. 31, 2016 | |
Income Tax Disclosure [Abstract] | |||||||||||
United States | $ (218,794) | $ (108,691) | $ (83,217) | ||||||||
Foreign | (6,580) | (11,593) | 0 | ||||||||
Loss before income tax expense | $ (70,557) | $ (63,358) | $ (61,832) | $ (29,627) | $ (20,498) | $ (15,571) | $ (20,359) | $ (63,856) | $ (225,374) | $ (120,284) | $ (83,217) |
Income Taxes, Provision for Inc
Income Taxes, Provision for Income Taxes (Details) - USD ($) | 3 Months Ended | 12 Months Ended | |||||
Sep. 30, 2018 | Jun. 30, 2018 | Dec. 31, 2017 | Sep. 30, 2017 | Dec. 31, 2018 | Dec. 31, 2017 | Dec. 31, 2016 | |
Current: | |||||||
Federal | $ 0 | $ 0 | $ 0 | ||||
State | 73,000 | 1,041,000 | 0 | ||||
Foreign | 374,000 | 234,000 | 0 | ||||
Total current | 447,000 | 1,275,000 | 0 | ||||
Deferred: | |||||||
Federal | 0 | 0 | 0 | ||||
State | 0 | 0 | 0 | ||||
Foreign | 0 | 0 | 0 | ||||
Total deferred | 0 | 0 | 0 | ||||
Income tax expense | $ 233,000 | $ 214,000 | $ (791,000) | $ 2,066,000 | $ 447,000 | $ 1,275,000 | $ 0 |
Income Taxes - Narrative (Detai
Income Taxes - Narrative (Details) - USD ($) | 3 Months Ended | 12 Months Ended | ||||||
Sep. 30, 2018 | Jun. 30, 2018 | Dec. 31, 2017 | Sep. 30, 2017 | Dec. 31, 2018 | Dec. 31, 2017 | Dec. 31, 2016 | Dec. 31, 2015 | |
Income Taxes [Line Items] | ||||||||
Provision for income taxes | $ 233,000 | $ 214,000 | $ (791,000) | $ 2,066,000 | $ 447,000 | $ 1,275,000 | $ 0 | |
Unrecognized tax benefits | $ 5,001,000 | 5,606,000 | 5,001,000 | 5,012,000 | $ 1,766,000 | |||
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] | ||||||||
Income Taxes [Line Items] | ||||||||
Increase in valuation allowance | 92,500,000 | $ 92,500,000 | ||||||
Federal [Member] | ||||||||
Income Taxes [Line Items] | ||||||||
Net operating loss carryforwards | 238,700,000 | |||||||
Federal [Member] | Expire in 2034 [Member] | ||||||||
Income Taxes [Line Items] | ||||||||
Net operating loss carryforwards | 4,300,000 | |||||||
Federal [Member] | Indefinite Expiration Period [Member] | ||||||||
Income Taxes [Line Items] | ||||||||
Net operating loss carryforwards | 234,400,000 | |||||||
State [Member] | ||||||||
Income Taxes [Line Items] | ||||||||
Net operating loss carryforwards | 4,200,000 | |||||||
Federal [Member] | Research and Development [Member] | ||||||||
Income Taxes [Line Items] | ||||||||
Tax credit carryforwards | $ 37,400,000 |
Income Taxes, Reconciliation of
Income Taxes, Reconciliation of Statutory to Effective Tax Rate (Details) - USD ($) | 3 Months Ended | 12 Months Ended | |||||||||
Dec. 31, 2018 | Sep. 30, 2018 | Jun. 30, 2018 | Mar. 31, 2018 | Dec. 31, 2017 | Sep. 30, 2017 | Jun. 30, 2017 | Mar. 31, 2017 | Dec. 31, 2018 | Dec. 31, 2017 | Dec. 31, 2016 | |
Reconciliation Between Effective and Statutory Tax Rate [Abstract] | |||||||||||
Pre-tax loss | $ (70,557,000) | $ (63,358,000) | $ (61,832,000) | $ (29,627,000) | $ (20,498,000) | $ (15,571,000) | $ (20,359,000) | $ (63,856,000) | $ (225,374,000) | $ (120,284,000) | $ (83,217,000) |
Statutory rate | (47,329,000) | (42,099,000) | (29,126,000) | ||||||||
State income tax net of federal benefit | (6,441,000) | (2,371,000) | (4,099,000) | ||||||||
Impact of foreign tax rate differential | 1,735,000 | 4,072,000 | 0 | ||||||||
Net change in valuation allowance | 54,173,000 | (18,917,000) | 43,438,000 | ||||||||
IP Transfer | (3,947,000) | ||||||||||
Tax credits | (4,035,000) | 4,189,000 | (11,007,000) | ||||||||
IPO/Deconsolidation adjustment | 37,911,000 | 0 | |||||||||
Tax rate change | 3,906,000 | 19,046,000 | |||||||||
Nondeductible items and other | 1,734,000 | (556,000) | 794,000 | ||||||||
Stock-based compensation | 651,000 | ||||||||||
Income tax expense | $ 233,000 | $ 214,000 | $ (791,000) | $ 2,066,000 | $ 447,000 | $ 1,275,000 | $ 0 | ||||
Income Taxes [Abstract] | |||||||||||
Statutory rate | 21.00% | 35.00% | 35.00% | ||||||||
State income tax net of federal benefit | 2.90% | 2.00% | 4.90% | ||||||||
Impact of foreign tax rate differential | (0.80%) | (3.40%) | |||||||||
Net change in valuation allowance | (24.00%) | 15.70% | (52.10%) | ||||||||
IP Transfer | 1.80% | ||||||||||
Tax credits | 1.80% | (3.50%) | 13.20% | ||||||||
IPO/Deconsolidation adjustment | (31.50%) | ||||||||||
Tax rate change | (1.70%) | (15.80%) | |||||||||
Nondeductible items and other | (0.90%) | 0.50% | (1.00%) | ||||||||
Stock-based compensation | (0.30%) | ||||||||||
Effective rate | (0.20%) | (1.00%) |
Income Taxes, Deferred Tax Asse
Income Taxes, Deferred Tax Assets and Liabilities (Details) - USD ($) $ in Thousands | Dec. 31, 2018 | Dec. 31, 2017 |
Deferred Tax Assets [Abstract] | ||
Net operating loss carryovers | $ 51,280 | $ 1,157 |
Tax credits | 31,768 | 29,334 |
Stock-based compensation | 11,812 | 7,515 |
Deferred revenue | 6,876 | 29,256 |
Intangible and capital assets | 56,984 | |
Other | 1,996 | 240 |
Total deferred tax assets | 160,716 | 67,502 |
Deferred Tax Liabilities [Abstract] | ||
Fixed assets | (811) | (125) |
Total deferred tax liabilities | (811) | (125) |
Valuation allowance | $ (159,905) | (67,377) |
Net deferred tax assets and liabilities | $ 0 |
Income Taxes, Gross Unrecognize
Income Taxes, Gross Unrecognized Tax Benefits (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2018 | Dec. 31, 2017 | Dec. 31, 2016 | |
Gross Unrecognized Tax Benefits [Roll Forward] | |||
Beginning balance of unrecognized tax benefits | $ 5,001 | $ 5,012 | $ 1,766 |
Additions related to the current year | 691 | 1,723 | 3,246 |
Decreases related to prior year tax positions | (86) | (1,734) | 0 |
Ending balance of unrecognized tax benefits | $ 5,606 | $ 5,001 | $ 5,012 |
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, 2018 | Dec. 31, 2017 | Dec. 31, 2016 | |
Compensation And Retirement Disclosure [Abstract] | |||
Employee contribution limit per calendar year for employees under 50 years of age | $ 18,500 | ||
Employee contribution limit per calendar year for employees over 50 years of age | 24,500 | ||
Matching contributions | $ 1,600,000 | $ 300,000 | $ 200,000 |
Basic and Diluted Net Loss Pe_3
Basic and Diluted Net Loss Per Share - Narratives (Details) - shares | 12 Months Ended | |||
Dec. 31, 2018 | Dec. 31, 2017 | Jul. 31, 2017 | Dec. 31, 2015 | |
Earnings Per Share Basic [Line Items] | ||||
Common stock, shares issued (in shares) | 89,345,978 | 66,541,629 | 66,541,629 | |
Common stock, shares outstanding (in shares) | 89,345,978 | 66,541,629 | 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, 2018 | Sep. 30, 2018 | Jun. 30, 2018 | Mar. 31, 2018 | Dec. 31, 2017 | Sep. 30, 2017 | Jun. 30, 2017 | Mar. 31, 2017 | Dec. 31, 2018 | Dec. 31, 2017 | Dec. 31, 2016 | |
Basic and Diluted Net Loss Per Share [Abstract] | |||||||||||
Net loss | $ (70,557) | $ (63,591) | $ (62,046) | $ (29,627) | $ (19,707) | $ (17,637) | $ (20,359) | $ (63,856) | $ (225,821) | $ (121,559) | $ (83,217) |
Preferred stock dividend | (1,791) | 0 | (20,100) | 0 | |||||||
Distributions to Ionis | (7,792) | 0 | 0 | ||||||||
Distributable losses | $ (19,428) | (233,613) | (141,659) | (83,217) | |||||||
Weighted-average shares outstanding owned | 59,173,938 | ||||||||||
Preferred Stock [Member] | |||||||||||
Basic and Diluted Net Loss Per Share [Abstract] | |||||||||||
Net loss | $ (64) | 0 | (28,385) | (83,217) | |||||||
Preferred stock dividend | (1,791) | $ 0 | $ (20,100) | $ 0 | |||||||
Distributable losses | $ (1,855) | ||||||||||
Weighted-average shares outstanding owned | 5,651,323 | 0 | 15,748,009 | 28,884,540 | |||||||
Losses attributable to preferred shares | $ 0 | $ (48,485) | $ (83,217) | ||||||||
Basic loss per common share owned | $ 0.01 | $ 0 | $ (1.80) | $ (2.88) | |||||||
Common Stock [Member] | |||||||||||
Basic and Diluted Net Loss Per Share [Abstract] | |||||||||||
Net loss | $ (17,573) | ||||||||||
Weighted-average shares outstanding owned | 53,522,615 | 81,365,801 | 46,010,777 | 28,884,540 | |||||||
Basic loss per common share owned | $ (0.33) | ||||||||||
Common Stock [Member] | Ionis [Member] | |||||||||||
Basic and Diluted Net Loss Per Share [Abstract] | |||||||||||
Net loss | $ (163,938) | $ (63,638) | $ 0 | ||||||||
Distributable losses | $ (171,730) | $ (63,638) | $ 0 | ||||||||
Weighted-average shares outstanding owned | 59,812,394 | 20,669,446 | 0 | ||||||||
Basic loss per common share owned | $ (2.74) | $ (3.08) | $ 0 | ||||||||
Common Stock [Member] | Others [Member] | |||||||||||
Basic and Diluted Net Loss Per Share [Abstract] | |||||||||||
Distributable losses | $ (61,883) | $ (29,536) | $ 0 | ||||||||
Weighted-average shares outstanding owned | 21,553,407 | 9,593,322 | 0 | ||||||||
Basic loss per common share owned | $ (2.87) | $ (3.08) | $ 0 |
Contractual Obligations and C_3
Contractual Obligations and Commitments - Contractual Obligation (Details) $ in Thousands | Dec. 31, 2018USD ($) |
Commitments And Contingencies Disclosure [Abstract] | |
Total | $ 23,688 |
Operating lease obligations, Less than 1 year | 2,308 |
Operating lease obligations, 1 to 3 years | 4,711 |
Operating lease obligations, 3 to 5 years | 4,805 |
Operating lease obligations, More than 5 years | 11,864 |
Total | 5,033 |
Purchase commitments, Less than 1 year | 5,033 |
Purchase commitments, 1 to 3 years | 0 |
Purchase commitments, 3 to 5 years | 0 |
Purchase commitments, More than 5 years | 0 |
Total | 28,721 |
Contractual obligations, Less than 1 year | 7,341 |
Contractual obligations,1 to 3 years | 4,711 |
Contractual obligations, 3 to 5 years | 4,805 |
Contractual obligations, More than 5 years | $ 11,864 |
Contractual Obligations and C_4
Contractual Obligations and Commitments (Details) $ in Thousands | Aug. 15, 2018USD ($) | Mar. 31, 2018 | Dec. 31, 2018USD ($) | Dec. 31, 2017USD ($) | Dec. 31, 2016USD ($) | Nov. 12, 2018ft² | Apr. 05, 2018USD ($)ft²Option |
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 | ||||||
Tenant improvement allowance | $ 3,600 | ||||||
Office Space in Boston and Carlsbad [Member] | |||||||
Operating Leased Assets [Line Items] | |||||||
Rent expense | 2,400 | $ 700 | $ 400 | ||||
Deferred rent balance | $ 4,800 | $ 39 | |||||
Office Space for Corporate Headquarters [Member] | |||||||
Operating Leased Assets [Line Items] | |||||||
Area of office space under lease agreement | ft² | 30,175 | ||||||
Initial term of lease | 123 months | ||||||
Number of renewal options | Option | 1 | ||||||
Term of renewal period | 5 years | ||||||
Period of free rent | 3 months | ||||||
Tenant improvement allowance | $ 3,800 | ||||||
Initial letter of credit to secure lease obligation | $ 2,400 | ||||||
Letter of credit to secure lease obligation on third anniversary of rent commencement date | 1,800 | ||||||
Letter of credit to secure lease obligation on fifth anniversary of rent commencement date | $ 1,200 |
Restructuring - Narratives (Det
Restructuring - Narratives (Details) - USD ($) $ in Millions | Sep. 06, 2018 | Dec. 31, 2018 |
Restructuring Costs [Abstract] | ||
Percentage of reduction in workforce | 12.00% | |
Restructuring-related costs | $ 1.7 | |
Non-cash stock option modification expenses | $ 0.4 |
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) $ in Thousands | 12 Months Ended |
Dec. 31, 2018USD ($) | |
Restructuring Reserve [Abstract] | |
Restructuring-related costs | $ 1,889 |
Adjustments during the period | (150) |
Amounts paid during the period | (1,696) |
Restructuring reserve ending balance | $ 43 |
Quarterly Financial Data (Una_3
Quarterly Financial Data (Unaudited) (Details) - USD ($) | 3 Months Ended | 12 Months Ended | |||||||||
Dec. 31, 2018 | Sep. 30, 2018 | Jun. 30, 2018 | Mar. 31, 2018 | Dec. 31, 2017 | Sep. 30, 2017 | Jun. 30, 2017 | Mar. 31, 2017 | Dec. 31, 2018 | Dec. 31, 2017 | Dec. 31, 2016 | |
Revenue: | |||||||||||
Revenue | $ 10,197,000 | $ 19,241,000 | $ 18,321,000 | $ 17,108,000 | $ 21,688,000 | $ 9,906,000 | $ 5,713,000 | $ 6,094,000 | $ 64,867,000 | $ 43,401,000 | $ 0 |
Expenses: | |||||||||||
Cost of sales - intangible asset amortization | 2,012,000 | 701,000 | 2,713,000 | 0 | 0 | ||||||
Research and development | 33,532,000 | 29,381,000 | 39,457,000 | 27,970,000 | 25,968,000 | 17,640,000 | 18,487,000 | 64,794,000 | 130,340,000 | 126,890,000 | 68,459,000 |
Selling, general and administrative | 45,934,000 | 45,924,000 | 42,287,000 | 19,465,000 | 17,018,000 | 8,373,000 | 6,915,000 | 4,676,000 | 153,610,000 | 36,981,000 | 15,053,000 |
Total expenses | 82,255,000 | 84,249,000 | 81,744,000 | 47,435,000 | 42,986,000 | 26,013,000 | 25,402,000 | 69,470,000 | 295,683,000 | 163,871,000 | 83,512,000 |
Loss from operations | (72,058,000) | (65,008,000) | (63,423,000) | (30,327,000) | (21,298,000) | (16,107,000) | (19,689,000) | (63,376,000) | (230,816,000) | (120,470,000) | (83,512,000) |
Other income (expense): | |||||||||||
Investment income | 1,542,000 | 1,675,000 | 1,546,000 | 868,000 | 819,000 | 687,000 | 245,000 | 61,000 | 5,631,000 | 1,813,000 | 295,000 |
Interest expense | (224,000) | (965,000) | (541,000) | 0 | (1,731,000) | 0 | |||||
Other income (expense) | (41,000) | (25,000) | 45,000 | (168,000) | (19,000) | 73,000 | 50,000 | (189,000) | 104,000 | 0 | |
Loss before income tax expense | (70,557,000) | (63,358,000) | (61,832,000) | (29,627,000) | (20,498,000) | (15,571,000) | (20,359,000) | (63,856,000) | (225,374,000) | (120,284,000) | (83,217,000) |
Income tax expense | (233,000) | (214,000) | 791,000 | (2,066,000) | (447,000) | (1,275,000) | 0 | ||||
Net loss | (70,557,000) | (63,591,000) | (62,046,000) | (29,627,000) | (19,707,000) | (17,637,000) | (20,359,000) | (63,856,000) | (225,821,000) | (121,559,000) | (83,217,000) |
Preferred stock dividend | (1,791,000) | 0 | (20,100,000) | 0 | |||||||
Distributable losses | $ (19,428,000) | (233,613,000) | (141,659,000) | (83,217,000) | |||||||
Weighted-average shares outstanding owned | 59,173,938 | ||||||||||
Net loss | $ (70,557,000) | $ (63,591,000) | $ (62,046,000) | $ (29,627,000) | $ (19,707,000) | $ (17,637,000) | $ (20,359,000) | $ (63,856,000) | (225,821,000) | (121,559,000) | (83,217,000) |
Preferred Stock [Member] | |||||||||||
Other income (expense): | |||||||||||
Net loss | $ (64,000) | $ 0 | $ (28,385,000) | $ (83,217,000) | |||||||
Net loss per share, basic and diluted (in dollars per share) | $ (0.01) | $ (0.70) | $ (2.21) | $ 0 | $ (1.80) | $ (2.88) | |||||
Weighted-average shares outstanding, basic and diluted (in shares) | 5,651,323 | 28,888,450 | 28,884,540 | 0 | 15,748,009 | 28,884,540 | |||||
Preferred stock dividend | $ (1,791,000) | $ 0 | $ (20,100,000) | $ 0 | |||||||
Distributable losses | $ (1,855,000) | ||||||||||
Weighted-average shares outstanding owned | 5,651,323 | 0 | 15,748,009 | 28,884,540 | |||||||
Basic loss per common share owned | $ 0.01 | $ 0 | $ (1.80) | $ (2.88) | |||||||
Net loss | $ (64,000) | $ 0 | $ (28,385,000) | $ (83,217,000) | |||||||
Common Stock [Member] | |||||||||||
Other income (expense): | |||||||||||
Net loss | $ (17,573,000) | ||||||||||
Weighted-average shares outstanding owned | 53,522,615 | 81,365,801 | 46,010,777 | 28,884,540 | |||||||
Basic loss per common share owned | $ (0.33) | ||||||||||
Net loss | $ (17,573,000) | ||||||||||
Common Stock [Member] | Ionis [Member] | |||||||||||
Other income (expense): | |||||||||||
Net loss | $ (163,938,000) | $ (63,638,000) | $ 0 | ||||||||
Net loss per share, basic and diluted (in dollars per share) | $ (0.79) | $ (0.73) | $ (0.72) | $ (0.44) | $ (0.30) | $ (0.33) | $ (2.74) | $ (3.08) | $ 0 | ||
Weighted-average shares outstanding, basic and diluted (in shares) | 67,129,553 | 65,538,467 | 60,832,494 | 45,447,879 | 45,447,879 | 36,555,903 | 59,812,394 | 20,669,446 | 0 | ||
Distributable losses | $ (171,730,000) | $ (63,638,000) | $ 0 | ||||||||
Weighted-average shares outstanding owned | 59,812,394 | 20,669,446 | 0 | ||||||||
Basic loss per common share owned | $ (2.74) | $ (3.08) | $ 0 | ||||||||
Net loss | $ (163,938,000) | $ (63,638,000) | $ 0 | ||||||||
Common Stock [Member] | Others [Member] | |||||||||||
Other income (expense): | |||||||||||
Net loss per share, basic and diluted (in dollars per share) | $ (0.79) | $ (0.73) | $ (0.85) | $ (0.44) | $ (0.30) | $ (0.33) | $ (2.87) | $ (3.08) | $ 0 | ||
Weighted-average shares outstanding, basic and diluted (in shares) | 21,869,713 | 21,671,415 | 21,492,157 | 21,171,372 | 21,093,750 | 16,966,712 | 21,553,407 | 9,593,322 | 0 | ||
Distributable losses | $ (61,883,000) | $ (29,536,000) | $ 0 | ||||||||
Weighted-average shares outstanding owned | 21,553,407 | 9,593,322 | 0 | ||||||||
Basic loss per common share owned | $ (2.87) | $ (3.08) | $ 0 | ||||||||
Product [Member] | |||||||||||
Revenue: | |||||||||||
Revenue | $ 2,237,000 | $ 2,237,000 | $ 0 | $ 0 | |||||||
Expenses: | |||||||||||
Cost of product/license | 777,000 | $ 1,043,000 | 1,820,000 | 0 | 0 | ||||||
Licensing [Member] | |||||||||||
Revenue: | |||||||||||
Revenue | 12,000,000 | 12,000,000 | 0 | 0 | |||||||
Expenses: | |||||||||||
Cost of product/license | 7,200,000 | 7,200,000 | 0 | 0 | |||||||
Commercial Revenue [Member] | |||||||||||
Revenue: | |||||||||||
Revenue | 2,237,000 | 12,000,000 | 14,237,000 | 0 | 0 | ||||||
Research and Development Revenue Under Collaborative Agreement [Member] | |||||||||||
Revenue: | |||||||||||
Revenue | $ 7,960,000 | $ 7,241,000 | $ 18,321,000 | $ 17,108,000 | $ 21,688,000 | $ 9,906,000 | $ 5,713,000 | $ 6,094,000 | $ 50,630,000 | $ 43,401,000 | $ 0 |
Subsequent Events - Narratives
Subsequent Events - Narratives (Details) - USD ($) $ in Thousands | Feb. 22, 2019 | Dec. 31, 2018 | Sep. 30, 2018 | Jun. 30, 2018 | Mar. 31, 2018 | Dec. 31, 2017 | Sep. 30, 2017 | Jun. 30, 2017 | Mar. 31, 2017 | Dec. 31, 2018 | Dec. 31, 2017 | Dec. 31, 2016 |
Subsequent Event [Line Items] | ||||||||||||
Revenue | $ 10,197 | $ 19,241 | $ 18,321 | $ 17,108 | $ 21,688 | $ 9,906 | $ 5,713 | $ 6,094 | $ 64,867 | $ 43,401 | $ 0 | |
Subsequent Event [Member] | Ionis [Member] | Akcea [Member] | ||||||||||||
Subsequent Event [Line Items] | ||||||||||||
Sublicense fee payable | $ 75,000 | |||||||||||
Number of shares of common stock reserved for issuance (in shares) | 2,837,373 | |||||||||||
Licensing [Member] | ||||||||||||
Subsequent Event [Line Items] | ||||||||||||
Revenue | $ 12,000 | $ 12,000 | $ 0 | $ 0 | ||||||||
Licensing [Member] | Subsequent Event [Member] | Akcea [Member] | ||||||||||||
Subsequent Event [Line Items] | ||||||||||||
Revenue | $ 150,000 |