Document and Entity Information
Document and Entity Information - shares | 3 Months Ended | |
Mar. 31, 2020 | Apr. 29, 2020 | |
Cover [Abstract] | ||
Entity Registrant Name | Akcea Therapeutics, Inc. | |
Entity Central Index Key | 0001662524 | |
Entity Current Reporting Status | Yes | |
Document Type | 10-Q | |
Document Period End Date | Mar. 31, 2020 | |
Amendment Flag | false | |
Trading Symbol | AKCA | |
Current Fiscal Year End Date | --12-31 | |
Entity Filer Category | Accelerated Filer | |
Entity Common Stock Shares Outstanding | 101,509,223 | |
Document Fiscal Year Focus | 2020 | |
Document Fiscal Period Focus | Q1 | |
Entity Interactive Data Current | Yes | |
Title of 12(b) Security | Common Stock | |
Security Exchange Name | NASDAQ | |
Entity Small Business | false | |
Entity Emerging Growth Company | true | |
Entity Ex Transition Period | true | |
Entity Shell Company | false | |
Entity Address, Address Line One | 22 Boston Wharf Road | |
Entity Address, Address Line Two | 9th Floor | |
Entity Address, City or Town | Boston | |
Entity Address, State or Province | MA | |
Entity Address, Postal Zip Code | 02210 | |
City Area Code | 617 | |
Local Phone Number | 207-0202 | |
Entity File Number | 001-38137 | |
Entity Tax Identification Number | 47-2608175 | |
Entity Incorporation, State or Country Code | DE | |
Document Quarterly Report | true | |
Document Transition Report | false |
CONDENSED CONSOLIDATED BALANCE
CONDENSED CONSOLIDATED BALANCE SHEETS (Unaudited) - USD ($) $ in Thousands | Mar. 31, 2020 | Dec. 31, 2019 |
Current assets: | ||
Cash and cash equivalents | $ 163,320 | $ 306,866 |
Short-term investments | 257,982 | 156,806 |
Accounts receivable | 10,673 | 10,496 |
Receivable from Ionis Pharmaceuticals, Inc. | 2,432 | 3,231 |
Inventories | 13,723 | 8,817 |
Other current assets | 10,813 | 10,689 |
Total current assets | 458,943 | 496,905 |
Property, plant and equipment, net | 5,437 | 5,261 |
Operating lease right-of-use assets | 10,868 | 11,094 |
Intangible assets, net | 81,589 | 83,051 |
Deposits and other assets | 3,084 | 2,939 |
Total assets | 559,921 | 599,250 |
Current liabilities: | ||
Accounts payable | 10,372 | 10,216 |
Accrued compensation | 6,996 | 12,793 |
Accrued liabilities | 14,131 | 14,191 |
Deferred revenue | 832 | 2,165 |
Other current liabilities | 4,001 | 2,633 |
Total current liabilities | 36,332 | 41,998 |
Long-term portion of lease liabilities | 13,898 | 14,248 |
Total liabilities | 50,230 | 56,246 |
Stockholders’ equity: | ||
Common stock, $0.001 par value; 125,000,000 shares authorized at March 31, 2020 and December 31, 2019; 101,348,633 and 100,993,173 shares issued and outstanding at March 31, 2020 and December 31, 2019, respectively. | 101 | 101 |
Additional paid-in capital | 1,033,319 | 1,024,168 |
Accumulated other comprehensive income | 391 | 5 |
Accumulated deficit | (524,120) | (481,270) |
Total stockholders’ equity | 509,691 | 543,004 |
Total liabilities and stockholders’ equity | $ 559,921 | $ 599,250 |
CONDENSED CONSOLIDATED BALANC_2
CONDENSED CONSOLIDATED BALANCE SHEETS (Unaudited) (Parenthetical) - $ / shares | Mar. 31, 2020 | Dec. 31, 2019 |
Stockholders’ equity: | ||
Common stock, par value (in dollars per share) | $ 0.001 | $ 0.001 |
Common stock, shares authorized (in shares) | 125,000,000 | 125,000,000 |
Common stock, shares issued (in shares) | 101,348,633 | 100,993,173 |
Common stock, shares outstanding (in shares) | 101,348,633 | 100,993,173 |
CONDENSED CONSOLIDATED STATEMEN
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS (Unaudited) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2020 | Mar. 31, 2019 | |
Revenue: | ||
Revenue | $ 16,074 | $ 163,816 |
Expenses: | ||
Cost of sales - intangible asset amortization | 1,419 | 1,403 |
Research and development | 17,355 | 99,619 |
Selling, general and administrative | 46,246 | 44,602 |
Total expenses | 61,333 | 137,609 |
(Loss) income from operations | (45,259) | 26,207 |
Other income (expense): | ||
Investment income | 1,771 | 1,224 |
Other expense | (130) | (112) |
(Loss) income before income tax expense | (43,618) | 27,319 |
Income tax benefit (expense) | 768 | (132) |
Net (loss) income | (42,850) | $ 27,187 |
Weighted-average shares of common stock outstanding, diluted | 94,127,942 | |
Ionis [Member] | ||
Expenses: | ||
Net loss share from commercial activities under arrangement with Ionis Pharmaceuticals, Inc. | $ (7,051) | $ (9,056) |
Common Stock [Member] | ||
Other income (expense): | ||
Weighted-average shares of common stock outstanding, basic | 101,105,070 | 90,708,330 |
Common Stock [Member] | Ionis [Member] | ||
Other income (expense): | ||
Net (loss) income | $ (32,674) | $ 23,846 |
Net (loss) income per share of common stock, basic | $ (0.42) | $ 0.35 |
Weighted-average shares of common stock outstanding, basic | 77,094,682 | 68,581,967 |
Net (loss) income per share of common stock, diluted | $ (0.42) | $ 0.34 |
Weighted-average shares of common stock outstanding, diluted | 77,094,682 | 68,581,967 |
Common Stock [Member] | Others [Member] | ||
Other income (expense): | ||
Net (loss) income per share of common stock, basic | $ (0.42) | $ 0.15 |
Weighted-average shares of common stock outstanding, basic | 24,010,388 | 22,126,363 |
Net (loss) income per share of common stock, diluted | $ (0.42) | $ 0.15 |
Weighted-average shares of common stock outstanding, diluted | 24,010,388 | 25,545,975 |
Product [Member] | ||
Revenue: | ||
Revenue | $ 15,159 | $ 6,754 |
Expenses: | ||
Cost of sales | 3,364 | 1,041 |
Research and Development and License Revenue Under Collaborative Agreement [Member] | ||
Revenue: | ||
Revenue | $ 915 | $ 157,062 |
CONDENSED CONSOLIDATED STATEM_2
CONDENSED CONSOLIDATED STATEMENTS OF COMPREHENSIVE (LOSS) INCOME (Unaudited) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2020 | Mar. 31, 2019 | |
Statement Of Income And Comprehensive Income [Abstract] | ||
Net (loss) income | $ (42,850) | $ 27,187 |
Unrealized gains on investments, net of tax | 377 | 212 |
Currency translation adjustment | 9 | 85 |
Comprehensive (loss) income | $ (42,464) | $ 27,484 |
CONDENSED CONSOLIDATED STATEM_3
CONDENSED CONSOLIDATED STATEMENTS OF STOCKHOLDERS' EQUITY (Unaudited) - USD ($) shares in Thousands, $ in Thousands | Total | Ionis [Member]TTR License Agreement [Member] | Common Stock [Member] | Common Stock [Member]Ionis [Member]TTR License Agreement [Member] | Additional Paid In Capital [Member] | Additional Paid In Capital [Member]Ionis [Member]TTR License Agreement [Member] | Accumulated Other Comprehensive Income (Loss) [Member] | Accumulated Deficit [Member] |
Balance at Dec. 31, 2018 | $ 276,724 | $ 89 | $ 799,001 | $ (324) | $ (522,042) | |||
Balance at Dec. 31, 2018 | 89,346 | |||||||
Net (loss) income | 27,187 | 27,187 | ||||||
Change in unrealized gains (losses), net of tax | 212 | 212 | ||||||
Currency translation adjustment | 85 | 85 | ||||||
Issuance of common stock in connection with employee stock plans | 4,588 | $ 1 | 4,587 | |||||
Issuance of common stock in connection with employee stock plans (in shares) | 452 | |||||||
Issuance of common stock in connection with Ionis sublicense fee | $ 75,000 | $ 3 | $ 74,997 | |||||
Issuance of stock (in shares) | 2,837 | |||||||
Distribution to Ionis | $ (13,492) | $ (13,492) | ||||||
Stock-based compensation expense | 18,560 | 18,560 | ||||||
Balance at Mar. 31, 2019 | 388,864 | $ 93 | 883,653 | (27) | (494,855) | |||
Balance at Mar. 31, 2019 | 92,635 | |||||||
Balance at Dec. 31, 2019 | 543,004 | $ 101 | 1,024,168 | 5 | (481,270) | |||
Balance at Dec. 31, 2019 | 100,993 | |||||||
Net (loss) income | (42,850) | (42,850) | ||||||
Change in unrealized gains (losses), net of tax | 377 | 377 | ||||||
Currency translation adjustment | 9 | 9 | ||||||
Issuance of common stock in connection with employee stock plans | 1,945 | 1,945 | ||||||
Issuance of common stock in connection with employee stock plans (in shares) | 360 | |||||||
Stock-based compensation expense | 7,282 | 7,282 | ||||||
Payments of tax withholdings related to exercise of employee stock options | (76) | (76) | ||||||
Payments of tax withholdings related to vesting of employee stock options (in shares) | (4) | |||||||
Balance at Mar. 31, 2020 | $ 509,691 | $ 101 | $ 1,033,319 | $ 391 | $ (524,120) | |||
Balance at Mar. 31, 2020 | 101,349 |
CONDENSED CONSOLIDATED STATEM_4
CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS (Unaudited) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2020 | Mar. 31, 2019 | |
Operating activities: | ||
Net (loss) income | $ (42,850) | $ 27,187 |
Adjustments to reconcile net (loss) income to net cash (used in) provided by operating activities: | ||
Depreciation | 212 | 207 |
Amortization of right-of-use operating lease assets | 226 | 330 |
Amortization of intangibles | 1,462 | 1,446 |
Amortization of premium/discount on investment securities, net | 311 | (229) |
Non-cash sublicensing expense | 75,000 | |
Stock-based compensation expense | 7,282 | 18,560 |
Changes in operating assets and liabilities: | ||
Accounts receivable | (177) | (5,672) |
Other current and long-term assets | (2,664) | (1,327) |
Inventories | (2,382) | (272) |
Accounts payable | 156 | (4,673) |
Receivable from Ionis Pharmaceuticals, Inc. | 620 | (26,165) |
Income tax receivable | (129) | |
Accrued compensation | (5,797) | (2,283) |
Accrued liabilities | 1,729 | 2,063 |
Income taxes payable | (771) | (56) |
Deferred revenue | (1,333) | (5,524) |
Net cash (used in) provided by operating activities | (44,105) | 78,592 |
Investing activities: | ||
Purchases of short-term investments | (154,910) | (13,858) |
Proceeds from maturity of short-term investments | 53,800 | 59,689 |
Purchases of property, plant and equipment | (209) | (1,031) |
Net cash (used in) provided by investing activities | (101,319) | 44,800 |
Financing activities: | ||
Proceeds from exercise of common stock options and employee stock purchase plan issuances | 1,945 | 4,588 |
Distribution to Ionis Pharmaceuticals, Inc. | (13,492) | |
Payments of tax withholdings related to exercise of employee stock awards | (76) | |
Net cash provided by (used in) financing activities | 1,869 | (8,904) |
Effect of exchange rates on cash | 9 | 85 |
Net (decrease) increase in cash and cash equivalents | (143,546) | 114,573 |
Cash, cash equivalents and restricted cash at beginning of period | 309,250 | 88,838 |
Cash, cash equivalents and restricted cash at end of period | 165,704 | 203,411 |
Cash, cash equivalents and restricted cash reported within the condensed consolidated balance sheets: | ||
Cash and cash equivalents | 163,320 | 201,027 |
Restricted cash included in deposits and other assets | 2,384 | 2,384 |
Cash, cash equivalents and restricted cash at end of period | 165,704 | $ 203,411 |
Ionis [Member] | ||
Supplemental disclosures of non-cash financing activities: | ||
Purchase of property, plant and equipment included in payable to Ionis Pharmaceuticals, Inc. | $ 179 |
Organization and Basis of Prese
Organization and Basis of Presentation | 3 Months Ended |
Mar. 31, 2020 | |
Organization Consolidation And Presentation Of Financial Statements [Abstract] | |
Organization and Basis of Presentation | 1. Organization and Basis of Presentation We were incorporated in Delaware in December 2014. We were organized by Ionis Pharmaceuticals, Inc., or Ionis, to focus on developing and commercializing medicines to treat patients with serious and rare diseases. On July 19, 2017, we completed our initial public offering, or IPO. As of March 31, 2020, Ionis owned approximately 76% of our common stock and is our majority shareholder. Prior to our IPO, we were wholly owned by Ionis. The accompanying condensed consolidated financial statements have been prepared in conformity with accounting principles generally accepted in the United States of America, or U.S. GAAP. The condensed consolidated financial statements include the accounts of Akcea Therapeutics, Inc. and our wholly owned subsidiaries ("we," "our," and "us"). All intercompany transactions and balances were eliminated in consolidation. We included all normal recurring adjustments in the financial statements that we considered necessary for a fair presentation of our financial position and our operating results and cash flows for the interim periods ended March 31, 2020 and 2019. Results for the interim periods are not necessarily indicative of the results for the entire year. For more complete financial information, these financial statements, and notes thereto, should be read in conjunction with the audited financial statements included in our Annual Report on Form 10-K for the year ended December 31, 2019. In accordance with Accounting Standards Codification, or ASC, 205-40, Going Concern |
Summary of Significant Accounti
Summary of Significant Accounting Policies | 3 Months Ended |
Mar. 31, 2020 | |
Accounting Policies [Abstract] | |
Summary of Significant Accounting Policies | 2. Summary of Significant Accounting Policies The accounting policies followed in the preparation of these interim condensed consolidated financial statements are consistent in all material respects with those presented in Note 2 to our financial statements included in our Annual Report on Form 10-K for the year ended December 31, 2019. Use of Estimates The preparation of financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities, the disclosure of contingent assets and liabilities at the date of the financial statements and the reported amounts of revenue and expenses during the reporting periods. Significant estimates and assumptions reflected in these financial statements include, but are not limited to, revenue recognition, the accrual for research and development expenses, stock-based compensation and income taxes. Estimates are periodically reviewed in light of changes in facts, circumstances and experience. Changes in estimates are recorded in the period in which they become known. Actual results could differ from those estimates. Concentration of Credit Risk Financial instruments that potentially subject us to concentrations of credit risk consist primarily of cash, cash equivalents, short-term investments and accounts receivable. We place our cash, cash equivalents and short-term investments with reputable financial institutions. We primarily invest our excess cash in commercial paper and debt instruments of the U.S. Treasury, financial institutions, corporations and U.S. government agencies with strong credit ratings and an investment grade rating at or above A-1, P-1 or F-1 by Moody's, Standard & Poor's, or S&P, or Fitch, respectively. We have established guidelines relative to diversification and maturities that maintain safety and liquidity. We periodically review and modify these guidelines to maximize trends in yields and interest rates without compromising safety and liquidity. New Accounting Pronouncements – Recently Issued In June 2016, the Financial Accounting Standards Board, or FASB, issued guidance that changes the measurement of credit losses for most financial assets and certain other instruments. This standard requires us to estimate the expected credit loss of an instrument over its lifetime using an expected credit loss model, which represents the portion of the amortized cost basis we do not expect to collect. The new guidance requires us to remeasure our allowance in each reporting period we have credit losses. We adopted this guidance on January 1, 2020 and it did not have a significant impact on our condensed consolidated financial statements and disclosures. In August 2018, the FASB issued clarifying guidance on how to account for implementation costs related to cloud-servicing arrangements. The guidance states that if these fees qualify to be capitalized and amortized over the service period, they need to be expensed in the same line item as the service expense and recognized in the same balance sheet category. The update can be applied either retrospectively or prospectively to all implementation costs incurred after the date of adoption. We adopted this guidance on January 1, 2020 on a prospective basis and it did not have a significant impact on our condensed consolidated financial statements and disclosures. In November 2018, the FASB issued clarifying guidance on the interaction between the collaboration accounting guidance and the new revenue recognition guidance we adopted on January 1, 2018 (Topic 606). Below is the clarifying guidance and how we implemented it (in italics): 1) When a participant is considered a customer in a collaborative arrangement, all of the associated accounting under Topic 606 should be applied. ● We apply all of the associated accounting under Topic 606 when we determine a participant in a collaborative arrangement is a customer. 2) Adds “unit of account” concept to collaboration accounting guidance to align with Topic 606. The “unit of account” concept is used to determine if revenue is recognized or if a contra expense is recognized from consideration received under a collaboration. ● We use the “unit of account” concept when we receive consideration under a collaboration agreement to determine when we recognize revenue or a contra expense. 3) The clarifying guidance precludes us from recognizing revenue under Topic 606 when we determine a transaction with a collaborative partner is not a customer and is not directly related to the sales to third parties. ● When we conclude a collaboration partner is not a customer and is not directly related to sales to third parties, we do not recognize revenue for the transaction. We adopted this guidance on January 1, 2020 and it did not have a significant impact on our condensed consolidated financial statements and disclosures . |
Net Product Revenue
Net Product Revenue | 3 Months Ended |
Mar. 31, 2020 | |
Revenue From Contract With Customer [Abstract] | |
Net Product Revenue | 3. Net Product Revenue Net product revenue consists of sales of TEGSEDI and WAYLIVRA. TEGSEDI is sold in the U.S. and E.U. On May 7, 2019, we obtained regulatory approval of WAYLIVRA in Europe, following which we began to sell WAYLIVRA in the E.U. During the three months ended March 31, 2020 and 2019, we recorded product revenue, net, of $15.2 million and $6.8 million, respectively. The following table summarizes balances and activity in each of our product revenue allowance and reserve categories for the three months ended March 31, 2020 and 2019 (in thousands): Chargebacks, discounts and fees Government and other rebates Returns Total Balance at December 31, 2018 $ 50 $ 293 $ 5 $ 348 Provision related to current period sales 155 375 52 582 Adjustment related to prior period sales — (30 ) — (30 ) Credits or payments made during the period (104 ) — — (104 ) Balance at March 31, 2019 $ 101 $ 638 $ 57 $ 796 Balance at December 31, 2019 $ 270 $ 1,353 $ 197 $ 1,820 Provision related to current period sales 470 1,621 58 2,149 Adjustment related to prior period sales (66 ) 69 — 3 Credits or payments made during the period (498 ) (554 ) — (1,052 ) Balance at March 31, 2020 $ 176 $ 2,489 $ 255 $ 2,920 The following table presents the balance of our receivables and contract liabilities related to net product revenue (in thousands): March 31, 2020 December 31, 2019 Receivables included in "Accounts receivable" $ 10,330 $ 6,946 Contract liabilities included in "Accrued liabilities" 2,645 1,550 |
Revenue from Collaboration and
Revenue from Collaboration and License Agreements | 3 Months Ended |
Mar. 31, 2020 | |
Revenue From Contract With Customer [Abstract] | |
Revenue from Collaboration and License Agreements | 4. Revenue from Collaboration and License Agreements Net revenue from our partners in connection with our collaboration and license agreements consisted of the following (in thousands): Three Months Ended March 31, 2020 2019 Strategic Collaboration with Novartis $ 311 $ 157,062 Vupanorsen (AKCEA-ANGPTL3-L Rx) 604 — Total net revenue from partners $ 915 $ 157,062 The following table presents the balance of our receivables and contract liabilities related to our collaboration and license agreements associated with our partners as of March 31, 2020 and December 31, 2019 (in thousands): March 31, 2020 December 31, 2019 Receivables included in "Accounts receivable" $ 717 $ 3,550 Contract liabilities included in "Deferred revenue" 718 1,322 During the three months ended March 31, 2020, we recognized the following revenue as a result of the change in the contract liability balances related to our collaboration and license agreements associated with our partners (in thousands): Revenue recognized in current period from: Three Months Ended March 31, 2020 Amounts included in deferred revenue at the beginning of the period $ 604 Strategic collaboration with Novartis In January 2017, we initiated a strategic collaboration with Novartis Pharma AG, or Novartis, for the development and commercialization of AKCEA-APO(a)-L Rx Rx . AKCEA-APO(a)-L Rx In February 2019, Novartis exercised its exclusive option to license AKCEA-APO(a)-L Rx Rx in full in February 2019 upon exercise In the second quarter of 2019, we completed the development services performance obligation for AKCEA-APO(a)-L Rx We are eligible to receive up to $675.0 million in additional milestone payments, including $25.0 million for the achievement of a development milestone, up to $290.0 million for the achievement of regulatory milestones and up to $360.0 million for the achievement of commercialization milestones. In connection with Novartis’ exercise of its option to exclusively license AKCEA-APO(a)-L Rx Rx Rx Rx We will earn the next milestone payment of $25.0 million under this collaboration if Novartis reaches a specific level of enrollment related to the Phase 3 study for AKCEA-APO(a)- Rx Rx . Rx AKCEA-APOCIII-L Rx In December 2019, we received written notice from Novartis electing not to exercise its option to license AKCEA-APOCIII-L Rx Rx . Rx As a result of Novartis’ election not to exercise its option, we retain the rights to further develop and commercialize AKCEA-APOCIII-L Rx Rx We did not recognize any revenue relating to AKCEA-APO(a)-L Rx Rx during the three months ended March 31, 2020, compared to $156.2 million of revenue recognized during the three months ended March 31, 2019. As of March 31, 2020 and December 31, 2019, we did not have any remaining deferred revenue related to the strategic collaboration with Novartis on our condensed consolidated balance sheet. 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. In addition, in April 2019, we entered into a supply agreement with PTC Therapeutics providing them the option to purchase product from us subject to terms as described in the agreement. During the three months ended March 31, 2020, we recognized revenue of $0.4 million relating to the sale of TEGSEDI to PTC Therapeutics. This amount is included in net product revenue on our condensed consolidated statement of operations. In May 2019, we received $6.0 million from PTC Therapeutics as a result of regulatory approval of WAYLIVRA in Europe. We paid Ionis a $3.0 million sublicense fee under our Cardiometabolic License Agreement and recorded it as a cost of license in our condensed consolidated statement of operations. In October 2019, we received $4.0 million from PTC Therapeutics as a result of regulatory approval of TEGSEDI in Brazil. We paid Ionis a $2.4 million sublicense fee under our development, commercialization, collaboration and license agreement with Ionis dated March 2018, or the TTR License Agreement, and recorded it as a cost of license in our condensed consolidated statement of operations. After receiving regulatory approval for WAYLIVRA and TEGSEDI, we deemed the milestone consideration probable, therefore we updated the transaction price to include these payments and accordingly, we recognized the $6.0 million and $4.0 million as license revenue during the second and fourth quarter of 2019, respectively. Prior to receiving regulatory approvals, we fully constrained these payments because regulatory approvals are not within our control. We are eligible to receive an additional $4.0 million for the achievement of a regulatory milestone and royalties in the mid-twenty percent range on net sales of TEGSEDI and WAYLIVRA in the PTC Territory. PTC Therapeutics’ obligation to pay royalties to us begins on the earlier of 12 months after the first commercial sale of a product in Brazil or the date that PTC Therapeutics recognizes revenue of at least $10.0 million in the PTC Territory. PTC Therapeutics will reduce these royalties upon the expiration of certain patents or if a generic competitor negatively impacts the market share of the product in the PTC Territory. Milestone payments and royalties that we are eligible to receive from PTC Therapeutics for TEGSEDI are split 60% to Ionis and 40% to Akcea in accordance with our TTR License Agreement. All WAYLIVRA milestone payments and royalties that we are eligible to receive from PTC Therapeutics are split equally with Ionis in accordance with our Cardiometabolic License Agreement. 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. Pfizer Vupanorsen License Agreement In October 2019, we entered into a license agreement with Pfizer, or the Pfizer Vupanorsen License Agreement, for the development and commercialization of vupanorsen (previously referred to as AKCEA-ANGPTL3-L Rx Under the terms of the Pfizer License Agreement, we granted Pfizer an exclusive license to further develop, manufacture and commercialize worldwide, subject to our potential participation in co-commercialization. We are responsible for completing a Phase 2 study and providing quantities of API for . We received a $250.0 million upfront payment in the fourth quarter of 2019 from Pfizer. We issued 6,873,344 shares of our common stock to Ionis as payment of the $125.0 million sublicense fee due under our Cardiometabolic License Agreement with Ionis. At commencement of the Pfizer Vupanorsen • License to develop and commercialize vupanorsen • Development activities for vupanorsen • API for vupanorsen We considered the manufacturing capabilities of Pfizer and the fact that manufacturing services are not proprietary and can be provided by another third party to conclude that the license has stand-alone functionality and is distinct. Further, the development activities and the supply of API are distinct because Pfizer or another third party could provide these items without our assistance. We determined the transaction price for the Pfizer Vupanorsen Based on the distinct performance obligations under the Pfizer Vupanorsen • $245.6 million for the transfer of the license of vupanorsen • $2.2 million for the development services for vupanorsen • $2.2 million for the delivery of vupanorsen We are recognizing revenue related to each of our performance obligations as follows: • We recognized the full amount related to the license and related know-how in the fourth quarter of 2019 because Pfizer, upon the date of closing, had full use of the license and related know-how without any continuing involvement from us. Additionally, we did not have any further performance obligations related to the license after the license was transferred to Pfizer; • We are satisfying the development services performance obligation for vupanorsen Pfizer is responsible for conducting and funding all future development, regulatory and commercialization activities for vupanorsen once we complete the Phase 2 program; • We recognized the amount attributed to the vupanorsen In addition, we are eligible to receive up to $1.3 billion in milestone payments, including up to $205.0 million for the achievement of development milestones, up to $250.0 million for the achievement of regulatory milestones and up to $850.0 million for the achievement of commercialization milestones. We will achieve the next milestone payment of $75.0 million when Pfizer advances vupanorsen . royalties in the mid-teens to low twenty percent range on net sales vupanorsen During the three months ended March 31, 2020, we recognized $0.6 million of revenue that was in our beginning deferred revenue balance. Our condensed consolidated balance sheet at March 31, 2020 and December 31, 2019 included deferred revenue of $0.7 million and $1.3 million, respectively, related to our development services obligation under the Pfizer Vupanorsen License Agreement. |
Investments and Fair Value Meas
Investments and Fair Value Measurements | 3 Months Ended |
Mar. 31, 2020 | |
Investments And Fair Value Measurements [Abstract] | |
Investments And Fair Value Measurements | 5 . Investments and Fair Value Measurements Investments As of March 31, 2020 and December 31, 2019, we primarily invested our excess cash in money market funds and debt instruments of the U.S. Treasury, financial institutions, corporations and U.S. government agencies with strong credit ratings and an investment grade rating at or above A-1, P-1 or F-1 by Moody’s, S&P or Fitch, respectively. We have established guidelines relative to diversification and maturities that maintain safety and liquidity. We periodically review and modify these guidelines to maximize trends in yields and interest rates without compromising safety and liquidity. The following is a summary of our investments at March 31, 2020 and December 31, 2019 (in thousands): Gross Unrealized Estimated March 31, 2020 Cost Gains Losses Fair Value Available-for-sale securities: Corporate debt securities $ 112,967 $ 14 $ (414 ) $ 112,567 Debt securities issued by U.S. government agencies 85,776 558 — 86,334 Total securities with a maturity of one year or less $ 198,743 $ 572 $ (414 ) $ 198,901 Corporate debt securities $ 40,202 $ 45 $ (175 ) $ 40,072 Debt securities issued by U.S. government agencies 38,596 417 (4 ) 39,009 Total securities with a maturity of one to two years 78,798 462 (179 ) 79,081 Total available-for-sale securities $ 277,541 $ 1,034 $ (593 ) $ 277,982 Gross Unrealized Estimated December 31, 2019 Cost Gains Losses Fair Value Available-for-sale securities: Corporate debt securities $ 105,679 $ 40 $ (23 ) $ 105,696 Debt securities issued by U.S. government agencies 38,970 30 (6 ) 38,994 Total securities with a maturity of one year or less $ 144,649 $ 70 $ (29 ) $ 144,690 Corporate debt securities $ 2,033 $ 5 $ - $ 2,038 Debt securities issued by U.S. government agencies 14,079 — (3 ) 14,076 Total securities with a maturity of one to two years 16,112 5 (3 ) 16,114 Total available-for-sale securities $ 160,761 $ 75 $ (32 ) $ 160,804 We recorded unrealized gains and losses related to the securities listed above as of March 31, 2020 and December 31, 2019. We believe that the decline in value of certain 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 these securities to maturity. Therefore, we anticipate a full recovery of the amortized cost basis of these 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 March 31, 2020 and December 31, 2019 that are regularly measured and carried at fair value. The table segregates our investments by level within the fair value hierarchy of valuation techniques used to determine their fair value (in thousands): At March 31, 2020 Quoted Prices in Active Markets (Level 1) Significant Other Observable Inputs (Level 2) Money market funds (1) $ 119,699 $ 119,699 $ — Corporate debt securities (3) 152,639 — 152,639 Debt securities issued by U.S. government agencies (2) 125,343 — 125,343 Total $ 397,681 $ 119,699 $ 277,982 At December 31, 2019 Quoted Prices in Active Markets (Level 1) Significant Other Observable Inputs (Level 2) Money market funds (1) $ 285,510 $ 285,510 $ — Corporate debt securities (2) 107,735 — 107,735 Debt securities issued by U.S. government agencies (3) 53,069 — 53,069 Total $ 446,314 $ 285,510 $ 160,804 (1) Included in cash and cash equivalents on our condensed consolidated balance sheet. (2) At March 31, 2020 and December 31, 2019, $20.0 million and $4.0 million, respectively, was included in cash and cash equivalents on our condensed consolidated balance sheet, with the difference included in short-term investments on our condensed consolidated balance sheet. (3) Included in short-term investments on our condensed consolidated balance sheet. |
Property Plant and Equipment
Property Plant and Equipment | 3 Months Ended |
Mar. 31, 2020 | |
Property Plant And Equipment [Abstract] | |
Property, Plant and Equipment | 6 . Property, Plant and Equipment The following table presents property and equipment, at cost, and related accumulated depreciation (in thousands): March 31, December 31, 2020 2019 Furniture and fixtures $ 1,611 $ 1,611 Computer equipment and software 345 289 Manufacturing equipment 439 416 Leasehold improvements 4,264 3,955 Total property and equipment, at cost 6,659 6,271 Less accumulated depreciation and amortization (1,222 ) (1,010 ) Total property and equipment, net $ 5,437 $ 5,261 We recorded depreciation expense of $0.2 million for each of the three months ended March 31, 2020 and 2019. As part of the operating lease for our corporate headquarters, the landlord provided a tenant improvement allowance of $3.6 million, which is included in leasehold improvements. |
Inventories
Inventories | 3 Months Ended |
Mar. 31, 2020 | |
Inventory Disclosure [Abstract] | |
Inventories | 7 . Inventories Prior to the regulatory approval of our medicines, we incur expenses for the manufacturing of drug product that could potentially be available to support the commercial launch of our products. We record all such costs as research and development expenses until the first reporting period when regulatory approval has been received or is otherwise considered probable. WAYLIVRA inventory-related costs incurred subsequent to April 1, 2019 and TEGSEDI inventory-related costs incurred subsequent to July 1, 2018 are reflected as inventories on our condensed consolidated balance sheet at the lower of cost or net realizable value under the first-in, first-out, or FIFO, basis. At March 31, 2020, a majority of our physical inventory for WAYLIVRA and a portion of our physical inventory for TEGSEDI included API that we produced prior to regulatory approval. As such, there is no cost basis for this API as we previously expensed the related costs as research and development expenses. 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 condensed consolidated statement of operations. The following table presents inventories (in thousands): March 31, December 31, 2020 2019 Raw materials $ 10,476 $ 6,520 Work in process 2,646 2,039 Finished goods 601 258 Total inventories $ 13,723 $ 8,817 |
Intangible Assets, Net
Intangible Assets, Net | 3 Months Ended |
Mar. 31, 2020 | |
Goodwill And Intangible Assets Disclosure [Abstract] | |
Intangible Assets, Net | 8 . Intangible Assets, net The following table presents intangible assets (in thousands): March 31, December 31, Estimated 2020 2019 useful life Acquired and in-licensed rights $ 2,262 $ 2,262 7 - 21 Years Capitalized regulatory approval milestones 90,000 90,000 16 Years Less accumulated amortization (10,673 ) (9,211 ) Total intangible assets, net $ 81,589 $ 83,051 We recorded $1.5 million and $1.4 million in amortization expense related to intangible assets during the three months ended March 31, 2020 and 2019, respectively. Estimated future amortization expense for intangible assets as of March 31, 2020 is as follows (in thousands): Years ended December 31, Intangible Assets Remainder of 2020 $ 4,417 2021 5,861 2022 5,856 2023 5,843 2024 5,822 Thereafter 53,790 $ 81,589 The weighted average remaining amortizable life of our patents was 10.7 years at March 31, 2020. For additional detail of our license agreements with Ionis, see Note 9, License Agreements and Services Agreement with Ionis |
License Agreements and Services
License Agreements and Services Agreement with Ionis | 3 Months Ended |
Mar. 31, 2020 | |
Related Party Transactions [Abstract] | |
License Agreements and Services Agreement with Ionis | 9 . In December 2015, we entered into a development, commercialization and license agreement related to our cardiometabolic franchise and a services agreement with Ionis. In March 2018, we entered into a new development, commercialization, collaboration and license agreement related to our TTR franchise and amended the services agreement previously in place with Ionis. The following sections summarize these related party agreements with Ionis. Cardiometabolic Development, Commercialization and License Agreement Our Cardiometabolic License Agreement with Ionis granted exclusive rights to us to develop and commercialize WAYLIVRA, AKCEA-APO(a)-L Rx Rx We and Ionis share development responsibilities for the Lipid Medicines, other than the medicines licensed to Novartis and to Pfizer. We pay Ionis for the research and development expenses it incurs on our behalf, which include both external and internal expenses. External research and development expenses include costs for contract research organizations, or CROs, costs to conduct nonclinical and clinical studies on our medicines, costs to acquire and evaluate clinical study data, such as investigator grants, patient screening fees and laboratory work, and fees paid to consultants. Internal research and development expenses include costs for the work that Ionis' research and development employees perform for us. Ionis charges us a full-time equivalent, or FTE, rate that covers personnel-related expenses, including salaries and benefits, plus an allocation of facility-related expenses, including rent, utilities, insurance and property taxes, for those development employees who work either directly or indirectly on the development of our medicines. We also pay Ionis for the API and drug product we use in our nonclinical and clinical studies for all of our medicines. Ionis manufactures the API for us and charges us a price per gram consistent with the price Ionis charges its pharmaceutical partners, which includes the cost for direct materials, direct labor and overhead required to manufacture the API. If we need the API filled in vials or prefilled syringes for our clinical studies and Ionis contracts with a third party to perform this work, Ionis charges us for the resulting cost. As we commercialize each of the Lipid Medicines, other than medicines licensed to Novartis and to Pfizer, we pay Ionis royalties in the mid-teens to the mid-twenty percent range on sales related to the Lipid Medicines that we sell. If we sell a Lipid Medicine 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 Medicine for a Broad Disease Patient Population (defined in the agreement as more than 500,000 patients worldwide or an indication that required a Phase 3 program of 1,000 or more patients and two or more years of treatment). Other than with respect to AKCEA-APO(a)-L Rx We may terminate this agreement if Ionis is in material breach of the agreement. Ionis may terminate this agreement if we are in material breach of the agreement. In each circumstance the party that is in breach will have an opportunity to cure the breach prior to the other party terminating this agreement. In the first quarter of 2017, we entered into letter agreements with Ionis to reflect the agreed upon payment terms with respect to the upfront option payment that we received from Novartis and to allocate the premium that Novartis paid for Ionis' common stock in connection with our strategic collaboration with Novartis. In the fourth quarter of 2019, we entered into a letter agreement with Ionis to reflect the agreed upon payment terms with respect to the upfront license fee we received from Pfizer in connection with our license agreement with Pfizer. For additional detail regarding our strategic collaboration with Novartis and license agreement with Pfizer, see Note 4, Revenue from Collaboration and License Agreements. TTR Development, Commercialization, Collaboration and License Agreement On April 17, 2018, our stockholders, other than Ionis and its affiliates, approved the TTR License Agreement and a stock purchase agreement, or Ionis SPA, with Ionis, which was entered into on March 14, 2018. In addition, in connection with these agreements, we entered into an amended and restated services agreement, or Amended Services Agreement, and an amended and restated investor rights agreement, or Amended Investor Rights Agreement, with Ionis. We determined that the TTR License Agreement and Ionis SPA included provisions that required the approval of our stockholders other than Ionis and its affiliates, which we deemed was not perfunctory in nature, therefore, we concluded that the approved date of the agreements for accounting purposes was April 17, 2018, the date on which such approval was received and the closing of the agreements took place. In accordance with the terms and provisions of the TTR License Agreement, we received rights to: • commercialize TEGSEDI following receipt of regulatory approval and perform certain other non-commercial activities with respect to TEGSEDI, in each case, in accordance with a global strategic plan; • partner on the completion of all pivotal studies of a follow-on medicine to TEGSEDI, AKCEA-TTR-L Rx Rx • commercialize AKCEA-TTR-L Rx • share in profits and losses with respect to TEGSEDI and AKCEA-TTR-L Rx • manufacture (including through a third party) each product following receipt of regulatory approval for such product; and • sublicense the development and commercialization of either product to third parties or affiliates, with the consent of Ionis. As consideration for the grant of rights under the TTR License Agreement, we paid an upfront license fee of $150.0 Rx The TTR License Agreement will remain in effect until the expiration of all included payment obligations, unless earlier terminated. The TTR License Agreement can be terminated by mutual consent of us and Ionis, by either us or Ionis upon certain events, by either party upon material breach, or by us 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 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 purchased $4.7 million of commercial TEGSEDI inventory held by Ionis. In addition, in the first quarter of 2019 we purchased $13.5 million of clinical TEGSEDI material held by Ionis. Prospectively we are responsible for the procurement of all additional inventory. The inventory and clinical material did not have a carrying value on the books of Ionis at the time of purchase. As such, in accordance with the accounting guidance for common control transactions above, we recorded the purchase of this commercial inventory and clinical material as a reduction of additional paid in capital as t his amount represented a cash distribution to Ionis. Accordingly, we included this amount as a distribution to Ionis for purposes of net (loss) income per share and we applied the two–class method as discussed in Note 11, Basic and Diluted Net (Loss) Income Per Share . We also determined that the TTR License Agreement represented a collaboration arrangement as defined by ASC 808. Prior to April 1, 2018, Ionis was responsible for all costs associated with TEGSEDI and for the period from April 1, 2018 to December 31, 2018, we were responsible for all costs associated with TEGSEDI. We and Ionis share all costs associated with AKCEA-TTR-L Rx We recorded $3.1 million paid to Ionis for costs related to the period prior to the closing of the TTR License Agreement to equity, as these amounts were previously expensed in the financial statements of Ionis. On July 11, 2018, we received regulatory approval for TEGSEDI in the E.U. for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis, or hATTR amyloidosis. As a result of the regulatory approval in the E.U., on August 3, 2018 we issued 1,597,571 shares of our common stock to Ionis as payment of the $40.0 million regulatory approval milestone for TEGSEDI. We capitalized this regulatory approval milestone payment as a licensed intangible asset on our condensed consolidated balance sheet as the amount is expected to be recoverable through future cash flows. On October 5, 2018, we received regulatory approval for TEGSEDI from the FDA for the treatment of polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults in the U.S. As a result of the regulatory approval in the U.S., on October 17, 2018 we issued 1,671,849 shares of our common stock to Ionis as payment of the $50.0 million regulatory approval milestone for TEGSEDI. We capitalized this regulatory approval milestone payment as a licensed intangible asset on our condensed consolidated balance sheet as the amount is expected to be recoverable through future cash flows. Both milestone payments are being amortized to cost of sales on a straight-line basis over the licensed assets’ expected useful life of approximately 16 years from the date of the initial regulatory approval milestone achievement. Amortization expense for the TTR intangible assets was $1.4 million for each of the three months ended March 31, 2020 and 2019. Profit/(Loss) Share Under the TTR License Agreement, w e and Ionis agreed to share TEGSEDI and AKCEA-TTR-L Rx Rx Rx Rx In the first quarter of 2019, the profit sharing provisions for TEGSEDI under the TTR License Agreement with Ionis became effective. As we are the principal for all commercial activities related to the TTR License Agreement, we record all commercial activities related to TEGSEDI on a gross basis in our condensed consolidated statement of operations, including revenue, cost of sales and sales and marketing expenses. The Ionis share of commercialization costs for TEGSEDI is separately presented within operating expenses in our condensed consolidated statement of operations under the caption “Net loss share from commercial activities under arrangement with Ionis Pharmaceuticals, Inc.” As we are a collaborator with Ionis for the execution of TTR development activities, we record all research and development expenses on a net basis representing our proportionate share of total costs incurred by Ionis and us. Accordingly, only our share of total costs incurred related to development activities under the TTR License Agreement is presented within research and development expense in our condensed consolidated statement of operations. A summary of the loss share related to the commercial activities under the TTR License Agreement is as follows (in thousands): Three Months Ended March 31, Three Months Ended March 31, 2020 2019 Net losses incurred by the collaboration related to the commercial activities under the TTR License Agreement $ (11,802 ) $ (15,098 ) Ionis' share of commercial losses under the TTR License Agreement reflected in our condensed consolidated statement of operations (7,051 ) (9,056 ) Akcea's share of commercial losses under the TTR License Agreement reflected in our condensed consolidated statement of operations (4,751 ) (6,042 ) A summary of the development expenses related to the TTR Agreement is as follows (in thousands): Three Months Ended March 31, Three Months Ended March 31, 2020 2019 Total development expense incurred by the collaboration related to development activities under the TTR License Agreement $ (18,331 ) $ (14,380 ) Akcea's share of TTR development expense reflected in research and development expense in our condensed consolidated statement of operations $ (8,190 ) $ (6,133 ) 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 FTE rate for the applicable personnel, plus out-of-pocket expenses, such as occupancy costs, associated with the FTEs allocated to providing us these services. We do not pay a mark-up or profit on the external or internal expenses Ionis bills to us. Ionis invoices us quarterly for all amounts due under the services agreement and payments are due within 30 days of the receipt of an invoice. In addition, as long as Ionis continues to consolidate our financials, we will comply with Ionis' policies and procedures and internal controls. As long as we are consolidated into Ionis' financial statements under U.S. GAAP, we may continue to access the following services from Ionis: • investor relations services, • human resources and personnel services, • risk management and insurance services, • tax related services, • corporate record keeping services, • financial and accounting services, • credit services, and • COO/CFO/CBO oversight. However, if we wanted to provide the foregoing services internally, and doing so would not negatively impact Ionis' internal controls and procedures for financial reporting, we can negotiate in good faith with Ionis for a reduced scope of services related to the aforementioned services. When Ionis determines it should no longer consolidate our financials, we may mutually agree with Ionis in writing to extend the term of this arrangement in six-month increments. We can establish our own benefits programs or continue to use Ionis' benefits, however we must provide Ionis a minimum advance notice to opt-out of using Ionis' benefits. We do not currently plan to establish our own benefits programs at this time or in the near future. Pursuant to our various agreements with Ionis, at March 31, 2020 and December 31, 2019, Ionis owed us $2.4 million and $3.2 million, respectively. The following table summarizes the amounts recorded related to transactions with Ionis including amounts related to the TTR License Agreement for the following periods (in thousands): Three Months Ended March 31, 2020 2019 Operating expenses: Services performed by Ionis $ 1,903 $ 1,075 Sublicensing expenses — 75,000 Out-of-pocket expenses paid by Ionis 585 1,571 Royalty expense 804 — Less: commercial share of loss in connection with the TTR license transaction (6,898 ) (9,056 ) Less: R&D share of loss (income) in connection with the TTR license transaction 1,174 (796 ) Total operating (income) expenses generated by transactions with Ionis (2,432 ) 67,794 Plus: distribution to Ionis — 13,492 Total net charges generated by transactions with Ionis (2,432 ) 81,286 (Receivable) payable balance (from) to Ionis at the beginning of the period (3,231 ) 18,901 Less: total amounts received from (paid to) Ionis during the period 3,231 (32,393 ) Less: non-cash sublicensing expenses — (75,000 ) Total amount (receivable) from Ionis at period end $ (2,432 ) $ (7,206 ) |
Stock-Based Compensation
Stock-Based Compensation | 3 Months Ended |
Mar. 31, 2020 | |
Disclosure Of Compensation Related Costs Sharebased Payments [Abstract] | |
Stock-Based Compensation | 1 0 . Stock Plans 2015 Equity Incentive Plan As of March 31, 2020, the aggregate number of shares of common stock that may be issued pursuant to stock awards under the 2015 Equity Incentive Plan, or 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, or RSUs. At March 31, 2020, a total of 10,645,227 stock options were outstanding, of which 3,463,767 were exercisable, 1,535,512 RSU awards were outstanding, and 1,479,402 shares were available for future grant under the 2015 Plan. 2017 Employee Stock Purchase Plan On January 1, 2020, 500,000 shares of common stock were added to the 2017 Employee Stock Purchase Plan, or 2017 ESPP. In accordance with the provisions of our 2017 ESPP, the number of shares of our common stock reserved for issuance under the 2017 ESPP automatically increases on January 1 st Stock-based Compensation Expense We measure stock-based compensation expense for equity-classified awards related to stock options, RSUs and stock purchase rights under our 2017 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 condensed consolidated statement of operations. We reduce stock-based compensation expense for estimated forfeitures at the time of grant and revise in subsequent periods if actual forfeitures differ from those estimates. We use the Black-Scholes model to estimate the fair value of stock options granted and stock purchase rights under our 2017 ESPP. The expected term of stock options granted represents the period of time that we expect them to be outstanding. As we do not have sufficient 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. For the three months ended March 31, 2020 and 2019, we used the following weighted-average assumptions in our Black-Scholes calculations: Employee Stock Options: Three Months Ended March 31, 2020 2019 Risk-free interest rate 1.5 % 2.5 % Dividend yield 0.0 % 0.0 % Volatility 73.6 % 76.4 % Expected life 6.1 years 6.1 years Board of Directors Stock Options (1): Three Months Ended March 31, 2020 Risk-free interest rate 1.5 % Dividend yield 0.0 % Volatility 73.3 % Expected life 6.3 years (1) We did not grant stock options to our Board of Directors in the first quarter of 2019. 2017 ESPP: Three Months Ended March 31, 2020 2019 Risk-free interest rate 1.0 % 2.5 % Dividend yield 0.0 % 0.0 % Volatility 71.9 % 64.1 % Expected life 6 months 6 months The following table summarizes stock-based compensation expense for the three months ended March 31, 2020 and 2019 (in thousands): Three Months Ended March 31, 2020 2019 Cost of sales - product $ 237 $ 118 Research and development expenses 1,349 3,921 Selling, general and administrative expenses 5,696 14,521 Total $ 7,282 $ 18,560 As of March 31, 2020, total unrecognized, estimated non-cash stock-based compensation expense related to non-vested stock options and RSUs was $42.9 million and $18.9 million, respectively. 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 and RSUs over a weighted average amortization period of 1.6 years and 2.8 years, respectively. |
Basic and Diluted Net (Loss) In
Basic and Diluted Net (Loss) Income Per Share | 3 Months Ended |
Mar. 31, 2020 | |
Earnings Per Share [Abstract] | |
Basic and Diluted Net (Loss) Income Per Share | 1 1 . Basic and Diluted Net (Loss) Income Per Share In the first quarter of 2019, we purchased inventory from Ionis and the amount paid to Ionis was in excess of Ionis’ carrying value of the related assets acquired. In accordance with accounting guidance for common control transactions, this distribution was treated as a dividend to Ionis; therefore, we have applied the two-class method of net (loss) income per share to reflect the allocation of this distribution to the participating Ionis common shares. The two-class method is an earnings allocation formula that determines net (loss) income 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 net (loss) income per share under the two-class method, we have allocated the net (loss) income between common stock owned by Ionis and common stock owned by others. Basic net (loss) income per share for each class of stock is computed by dividing total distributable (losses) income applicable to common stock owned by Ionis and common stock owned by others by the weighted-average number of common shares outstanding during the requisite period. The following table summarizes the distributable (losses) income for the three months ended March 31, 2020 and 2019 (in thousands): Three Months Ended March 31, 2020 2019 Net (loss) income $ (42,850 ) $ 27,187 Distributions to Ionis — (13,492 ) Distributable (losses) income $ (42,850 ) $ 13,695 The following table summarizes the reconciliation of weighted-average shares outstanding used in the calculation of basic (loss) income per share for the three months ended March 31, 2020 and 2019: Three Months Ended March 31, 2020 2019 Determination of shares: Weighted-average common shares outstanding owned by Ionis 77,094,682 68,581,967 Weighted-average common shares outstanding owned by others 24,010,388 22,126,363 Total weighted-average common shares outstanding 101,105,070 90,708,330 The following table summarizes the calculation of basic (loss) income per share for the three months ended March 31, 2020 and 2019 (in thousands, except share and per share amounts): Three Months Ended March 31, 2020 2019 (Losses) income allocated to Ionis $ (32,674 ) $ 10,354 Plus: Distribution to Ionis — 13,492 (Losses) income available to Ionis $ (32,674 ) $ 23,846 Weighted-average common shares outstanding owned by Ionis 77,094,682 68,581,967 Basic (loss) income per common share owned by Ionis $ (0.42 ) $ 0.35 (Losses) income allocated to common shares owned by others $ (10,176 ) $ 3,341 Weighted-average common shares outstanding owned by others 24,010,388 22,126,363 Basic loss per common share owned by others $ (0.42 ) $ 0.15 For the three months ended March 31, 2020 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 RSUs; and • Employee Stock Purchase Plan. For the three months ended March 31, 2019, we had net income available to our common shareholders. As a result, we computed diluted net income per share using the weighted-average number of common shares owned by Ionis, weighted-average number of common shares owned by others and dilutive common equivalent shares outstanding during the period. The following table summarizes the reconciliation of weighted-average shares outstanding and diluted common equivalent shares used in the calculation of diluted income per share for the three months ended March 31, 2019: Three Months Ended March 31, 2019 Determination of shares: Weighted-average common shares outstanding owned by Ionis 68,581,967 Weighted-average common shares outstanding owned by others 22,126,363 Shares issuable upon exercise of stock options 3,370,817 Shares issuable upon restricted stock awards issuance 14,813 Shares issuable related to our ESPP 33,982 Weighted-average shares outstanding owned by others, plus assumed conversions 25,545,975 Total weighted-average shares outstanding 94,127,942 The following table summarizes the calculation of diluted income per share for the three months ended March 31, 2019 (in thousands, except per share amounts): Three Months Ended March 31, 2019 Income allocated to Ionis $ 9,978 Plus: Distribution to Ionis 13,492 Income available to Ionis $ 23,470 Weighted-average common shares outstanding owned by Ionis 68,581,967 Diluted income per common share owned by Ionis $ 0.34 Income allocated to common shares owned by others, plus assumed conversions $ 3,717 Weighted-average common shares outstanding owned by others, plus assumed conversions 25,545,975 Diluted income per common share owned by others $ 0.15 |
Contractual Obligations and Com
Contractual Obligations and Commitments | 3 Months Ended |
Mar. 31, 2020 | |
Commitments And Contingencies Disclosure [Abstract] | |
Contractual Obligations and Commitments | 1 2 . Operating Lease On April 5, 2018, we entered into an operating lease agreement for 30,175 square feet of office space located in Boston, Massachusetts for our corporate headquarters. The lease commencement date was August 15, 2018 and we took occupancy in September 2018. We are leasing this space under a non-cancelable operating lease with an initial term of 123 months and an option to extend the lease for an additional five-year term. We did not include the extension option in our right-of-use asset and lease liability calculation as we do not consider it reasonably certain that we would exercise the option. Under the lease agreement, we received a three-month free rent period, which commenced on August 15, 2018, and a tenant improvement allowance of $3.6 million. We provided the lessor with a letter of credit to secure our obligations under the lease in the initial amount of $2.4 million, to be reduced to $1.8 million on the third anniversary of the rent commencement date and to $1.2 million on the fifth anniversary of the rent commencement date if we meet certain conditions set forth in the lease at each such date. The letter of credit amount is included in deposits and other assets on the accompanying condensed consolidated balance sheet. On November 12, 2018, we entered into an operating lease agreement with Ionis to sublease 4,723 square feet of office space located in Carlsbad, California. The commencement date was March 2018 and the term of the lease is 64 months with a four-month free rent period. There is no extension option for this lease. On May 8, 2019, we entered into an operating lease agreement for office space located in Dublin, Ireland. The lease commenced in May 2019 and the initial term of the lease is 18 months with an extension option. We have included a 12-month extension period in our right-of-use asset and lease liability calculation as we consider it reasonably certain that we will exercise the option to extend the lease for an additional 12 months. The following table summarizes the contractual lease obligations as of March 31, 2020 (in thousands): Years ended December 31, Operating Leases Remainder of 2020 $ 1,877 2021 2,504 2022 2,403 2023 2,400 2024 2,395 Thereafter 9,565 Total minimum lease payments $ 21,144 Purchase Commitments Purchase commitments include agreements to purchase goods or services that are enforceable and legally binding on us and that specify all significant terms, including fixed or minimum quantities to be purchased, fixed, minimum or variable price provisions, and the approximate timing of the transaction. Such obligations are related principally to inventory purchase orders based on our current manufacturing needs and require significant lead times to be fulfilled by our vendors. Purchase commitments exclude agreements that are cancelable without penalty. As of March 31, 2020, our purchase commitments for the following 12 months were $0.9 million. |
Income Taxes
Income Taxes | 3 Months Ended |
Mar. 31, 2020 | |
Income Tax Disclosure [Abstract] | |
Income Taxes | 13. Income Taxes We recorded an income tax benefit of $0.8 million for the three months ended March 31, 2020, compared to income tax expense of $0.1 million for the same period in 2019. The decrease in our income tax expense was primarily due to a tax benefit of $1.7 million recorded in the period related to the Coronavirus, Aid, Relief and Economic Security Act, or CARES Act, signed into law on March 27, 2020, which offsets income tax expense related to taxable income earned in certain foreign jurisdictions. Under the Tax Cut and Jobs Act of 2017, or the Tax Act, federal net operating losses incurred in 2018 and in future years could be carried forward indefinitely, but the deductibility of such federal net operating losses was limited. The CARES Act temporarily removes the limitation on net operating loss deductions, resulting in the $1.7 million tax benefit recorded for the period. The limitation on the deductibility of federal net operating losses is reinstated for tax years beginning after 2020. |
Legal Proceedings
Legal Proceedings | 3 Months Ended |
Mar. 31, 2020 | |
Loss Contingency Information About Litigation Matters [Abstract] | |
Legal Proceedings | 14. From time to time, we may be involved in legal proceedings arising in the ordinary course of our business. Periodically, we evaluate the status of each legal matter and assess our potential financial exposure. If the potential loss from any legal proceeding is considered probable and the amount can be reasonably estimated, we accrue a liability for the estimated loss. Significant judgment is required to determine the probability of a loss and whether the amount of the loss is reasonably estimable. The outcome of any proceeding is not determinable in advance. As a result, the assessment of a potential liability and the amount of accruals recorded are based only on the information available to us at the time. As additional information becomes available, we reassess the potential liability related to the legal proceeding and may revise our estimates. On November 11, 2019, a purported Company stockholder filed the Delaware Action in the Delaware Court of Chancery captioned City of Cambridge Retirement System v. Crooke, et al. L Rx . That dismissal was with prejudice only as to the individual stockholder that filed the Delaware Action. It is therefore possible that a similar action could be filed at a later date prior to the expiration of the applicable statute of limitations. |
Summary of Significant Accoun_2
Summary of Significant Accounting Policies (Policies) | 3 Months Ended |
Mar. 31, 2020 | |
Accounting Policies [Abstract] | |
Use of Estimates | Use of Estimates The preparation of financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities, the disclosure of contingent assets and liabilities at the date of the financial statements and the reported amounts of revenue and expenses during the reporting periods. Significant estimates and assumptions reflected in these financial statements include, but are not limited to, revenue recognition, the accrual for research and development expenses, stock-based compensation and income taxes. Estimates are periodically reviewed in light of changes in facts, circumstances and experience. Changes in estimates are recorded in the period in which they become known. Actual results could differ from those estimates. |
Concentration of Credit Risk | Concentration of Credit Risk Financial instruments that potentially subject us to concentrations of credit risk consist primarily of cash, cash equivalents, short-term investments and accounts receivable. We place our cash, cash equivalents and short-term investments with reputable financial institutions. We primarily invest our excess cash in commercial paper and debt instruments of the U.S. Treasury, financial institutions, corporations and U.S. government agencies with strong credit ratings and an investment grade rating at or above A-1, P-1 or F-1 by Moody's, Standard & Poor's, or S&P, or Fitch, respectively. We have established guidelines relative to diversification and maturities that maintain safety and liquidity. We periodically review and modify these guidelines to maximize trends in yields and interest rates without compromising safety and liquidity. |
New Accounting Pronouncements - Recently Issued | New Accounting Pronouncements – Recently Issued In June 2016, the Financial Accounting Standards Board, or FASB, issued guidance that changes the measurement of credit losses for most financial assets and certain other instruments. This standard requires us to estimate the expected credit loss of an instrument over its lifetime using an expected credit loss model, which represents the portion of the amortized cost basis we do not expect to collect. The new guidance requires us to remeasure our allowance in each reporting period we have credit losses. We adopted this guidance on January 1, 2020 and it did not have a significant impact on our condensed consolidated financial statements and disclosures. In August 2018, the FASB issued clarifying guidance on how to account for implementation costs related to cloud-servicing arrangements. The guidance states that if these fees qualify to be capitalized and amortized over the service period, they need to be expensed in the same line item as the service expense and recognized in the same balance sheet category. The update can be applied either retrospectively or prospectively to all implementation costs incurred after the date of adoption. We adopted this guidance on January 1, 2020 on a prospective basis and it did not have a significant impact on our condensed consolidated financial statements and disclosures. In November 2018, the FASB issued clarifying guidance on the interaction between the collaboration accounting guidance and the new revenue recognition guidance we adopted on January 1, 2018 (Topic 606). Below is the clarifying guidance and how we implemented it (in italics): 1) When a participant is considered a customer in a collaborative arrangement, all of the associated accounting under Topic 606 should be applied. ● We apply all of the associated accounting under Topic 606 when we determine a participant in a collaborative arrangement is a customer. 2) Adds “unit of account” concept to collaboration accounting guidance to align with Topic 606. The “unit of account” concept is used to determine if revenue is recognized or if a contra expense is recognized from consideration received under a collaboration. ● We use the “unit of account” concept when we receive consideration under a collaboration agreement to determine when we recognize revenue or a contra expense. 3) The clarifying guidance precludes us from recognizing revenue under Topic 606 when we determine a transaction with a collaborative partner is not a customer and is not directly related to the sales to third parties. ● When we conclude a collaboration partner is not a customer and is not directly related to sales to third parties, we do not recognize revenue for the transaction. We adopted this guidance on January 1, 2020 and it did not have a significant impact on our condensed consolidated financial statements and disclosures . |
Net Product Revenue (Tables)
Net Product Revenue (Tables) | 3 Months Ended |
Mar. 31, 2020 | |
Revenue From Contract With Customer [Abstract] | |
Summary of Balances and Activity in Each of Product Revenue Allowance and Reserve Categories | The following table summarizes balances and activity in each of our product revenue allowance and reserve categories for the three months ended March 31, 2020 and 2019 (in thousands): Chargebacks, discounts and fees Government and other rebates Returns Total Balance at December 31, 2018 $ 50 $ 293 $ 5 $ 348 Provision related to current period sales 155 375 52 582 Adjustment related to prior period sales — (30 ) — (30 ) Credits or payments made during the period (104 ) — — (104 ) Balance at March 31, 2019 $ 101 $ 638 $ 57 $ 796 Balance at December 31, 2019 $ 270 $ 1,353 $ 197 $ 1,820 Provision related to current period sales 470 1,621 58 2,149 Adjustment related to prior period sales (66 ) 69 — 3 Credits or payments made during the period (498 ) (554 ) — (1,052 ) Balance at March 31, 2020 $ 176 $ 2,489 $ 255 $ 2,920 |
Summary of Balance of Receivables and Contract Liabilities Related to Net Product Revenue | The following table presents the balance of our receivables and contract liabilities related to net product revenue (in thousands): March 31, 2020 December 31, 2019 Receivables included in "Accounts receivable" $ 10,330 $ 6,946 Contract liabilities included in "Accrued liabilities" 2,645 1,550 |
Revenue from Collaboration an_2
Revenue from Collaboration and License Agreements (Tables) | 3 Months Ended |
Mar. 31, 2020 | |
Summary of Net Revenue from Partners in Connection with Collaboration and License Agreements | Net revenue from our partners in connection with our collaboration and license agreements consisted of the following (in thousands): Three Months Ended March 31, 2020 2019 Strategic Collaboration with Novartis $ 311 $ 157,062 Vupanorsen (AKCEA-ANGPTL3-L Rx) 604 — Total net revenue from partners $ 915 $ 157,062 |
Summary of Balance of Receivables and Contract Liabilities Related to Net Product Revenue | The following table presents the balance of our receivables and contract liabilities related to net product revenue (in thousands): March 31, 2020 December 31, 2019 Receivables included in "Accounts receivable" $ 10,330 $ 6,946 Contract liabilities included in "Accrued liabilities" 2,645 1,550 |
Summary of Revenue Recognized of Change in Contract Liability Balances Related to Collaboration and License Agreements Associated with Partners | During the three months ended March 31, 2020, we recognized the following revenue as a result of the change in the contract liability balances related to our collaboration and license agreements associated with our partners (in thousands): Revenue recognized in current period from: Three Months Ended March 31, 2020 Amounts included in deferred revenue at the beginning of the period $ 604 |
Collaboration and License Agreements [Member] | |
Summary of Balance of Receivables and Contract Liabilities Related to Net Product Revenue | The following table presents the balance of our receivables and contract liabilities related to our collaboration and license agreements associated with our partners as of March 31, 2020 and December 31, 2019 (in thousands): March 31, 2020 December 31, 2019 Receivables included in "Accounts receivable" $ 717 $ 3,550 Contract liabilities included in "Deferred revenue" 718 1,322 |
Investments and Fair Value Me_2
Investments and Fair Value Measurements (Tables) | 3 Months Ended |
Mar. 31, 2020 | |
Investments And Fair Value Measurements [Abstract] | |
Summary of Investments | The following is a summary of our investments at March 31, 2020 and December 31, 2019 (in thousands): Gross Unrealized Estimated March 31, 2020 Cost Gains Losses Fair Value Available-for-sale securities: Corporate debt securities $ 112,967 $ 14 $ (414 ) $ 112,567 Debt securities issued by U.S. government agencies 85,776 558 — 86,334 Total securities with a maturity of one year or less $ 198,743 $ 572 $ (414 ) $ 198,901 Corporate debt securities $ 40,202 $ 45 $ (175 ) $ 40,072 Debt securities issued by U.S. government agencies 38,596 417 (4 ) 39,009 Total securities with a maturity of one to two years 78,798 462 (179 ) 79,081 Total available-for-sale securities $ 277,541 $ 1,034 $ (593 ) $ 277,982 Gross Unrealized Estimated December 31, 2019 Cost Gains Losses Fair Value Available-for-sale securities: Corporate debt securities $ 105,679 $ 40 $ (23 ) $ 105,696 Debt securities issued by U.S. government agencies 38,970 30 (6 ) 38,994 Total securities with a maturity of one year or less $ 144,649 $ 70 $ (29 ) $ 144,690 Corporate debt securities $ 2,033 $ 5 $ - $ 2,038 Debt securities issued by U.S. government agencies 14,079 — (3 ) 14,076 Total securities with a maturity of one to two years 16,112 5 (3 ) 16,114 Total available-for-sale securities $ 160,761 $ 75 $ (32 ) $ 160,804 |
Assets Measured at Fair Value on a Recurring Basis | The following tables present the investments we held at March 31, 2020 and December 31, 2019 that are regularly measured and carried at fair value. The table segregates our investments by level within the fair value hierarchy of valuation techniques used to determine their fair value (in thousands): At March 31, 2020 Quoted Prices in Active Markets (Level 1) Significant Other Observable Inputs (Level 2) Money market funds (1) $ 119,699 $ 119,699 $ — Corporate debt securities (3) 152,639 — 152,639 Debt securities issued by U.S. government agencies (2) 125,343 — 125,343 Total $ 397,681 $ 119,699 $ 277,982 At December 31, 2019 Quoted Prices in Active Markets (Level 1) Significant Other Observable Inputs (Level 2) Money market funds (1) $ 285,510 $ 285,510 $ — Corporate debt securities (2) 107,735 — 107,735 Debt securities issued by U.S. government agencies (3) 53,069 — 53,069 Total $ 446,314 $ 285,510 $ 160,804 (1) Included in cash and cash equivalents on our condensed consolidated balance sheet. (2) At March 31, 2020 and December 31, 2019, $20.0 million and $4.0 million, respectively, was included in cash and cash equivalents on our condensed consolidated balance sheet, with the difference included in short-term investments on our condensed consolidated balance sheet. (3) Included in short-term investments on our condensed consolidated balance sheet. |
Property, Plant and Equipment (
Property, Plant and Equipment (Table) | 3 Months Ended |
Mar. 31, 2020 | |
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): March 31, December 31, 2020 2019 Furniture and fixtures $ 1,611 $ 1,611 Computer equipment and software 345 289 Manufacturing equipment 439 416 Leasehold improvements 4,264 3,955 Total property and equipment, at cost 6,659 6,271 Less accumulated depreciation and amortization (1,222 ) (1,010 ) Total property and equipment, net $ 5,437 $ 5,261 |
Inventories (Tables)
Inventories (Tables) | 3 Months Ended |
Mar. 31, 2020 | |
Inventory Disclosure [Abstract] | |
Schedule of Inventories | The following table presents inventories (in thousands): March 31, December 31, 2020 2019 Raw materials $ 10,476 $ 6,520 Work in process 2,646 2,039 Finished goods 601 258 Total inventories $ 13,723 $ 8,817 |
Intangible Assets, Net (Tables)
Intangible Assets, Net (Tables) | 3 Months Ended |
Mar. 31, 2020 | |
Goodwill And Intangible Assets Disclosure [Abstract] | |
Schedule of Intangible Assets, Net | The following table presents intangible assets (in thousands): March 31, December 31, Estimated 2020 2019 useful life Acquired and in-licensed rights $ 2,262 $ 2,262 7 - 21 Years Capitalized regulatory approval milestones 90,000 90,000 16 Years Less accumulated amortization (10,673 ) (9,211 ) Total intangible assets, net $ 81,589 $ 83,051 |
Schedule of Estimated Future Amortization Expenses for Intangible Assets | Estimated future amortization expense for intangible assets as of March 31, 2020 is as follows (in thousands): Years ended December 31, Intangible Assets Remainder of 2020 $ 4,417 2021 5,861 2022 5,856 2023 5,843 2024 5,822 Thereafter 53,790 $ 81,589 |
License Agreements and Servic_2
License Agreements and Services Agreement with Ionis (Tables) | 3 Months Ended |
Mar. 31, 2020 | |
Related Party Transactions [Abstract] | |
Schedule Of Net Loss Share Related to Commercial Activities Under TTR Agreement | A summary of the loss share related to the commercial activities under the TTR License Agreement is as follows (in thousands): Three Months Ended March 31, Three Months Ended March 31, 2020 2019 Net losses incurred by the collaboration related to the commercial activities under the TTR License Agreement $ (11,802 ) $ (15,098 ) Ionis' share of commercial losses under the TTR License Agreement reflected in our condensed consolidated statement of operations (7,051 ) (9,056 ) Akcea's share of commercial losses under the TTR License Agreement reflected in our condensed consolidated statement of operations (4,751 ) (6,042 ) |
Schedule Of Development Expenses Related to TTR Agreement | A summary of the development expenses related to the TTR Agreement is as follows (in thousands): Three Months Ended March 31, Three Months Ended March 31, 2020 2019 Total development expense incurred by the collaboration related to development activities under the TTR License Agreement $ (18,331 ) $ (14,380 ) Akcea's share of TTR development expense reflected in research and development expense in our condensed consolidated statement of operations $ (8,190 ) $ (6,133 ) |
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 License Agreement for the following periods (in thousands): Three Months Ended March 31, 2020 2019 Operating expenses: Services performed by Ionis $ 1,903 $ 1,075 Sublicensing expenses — 75,000 Out-of-pocket expenses paid by Ionis 585 1,571 Royalty expense 804 — Less: commercial share of loss in connection with the TTR license transaction (6,898 ) (9,056 ) Less: R&D share of loss (income) in connection with the TTR license transaction 1,174 (796 ) Total operating (income) expenses generated by transactions with Ionis (2,432 ) 67,794 Plus: distribution to Ionis — 13,492 Total net charges generated by transactions with Ionis (2,432 ) 81,286 (Receivable) payable balance (from) to Ionis at the beginning of the period (3,231 ) 18,901 Less: total amounts received from (paid to) Ionis during the period 3,231 (32,393 ) Less: non-cash sublicensing expenses — (75,000 ) Total amount (receivable) from Ionis at period end $ (2,432 ) $ (7,206 ) |
Stock-Based Compensation (Table
Stock-Based Compensation (Tables) | 3 Months Ended |
Mar. 31, 2020 | |
Stockholders Equity [Abstract] | |
Weighted-Average Assumptions for Stock Options | For the three months ended March 31, 2020 and 2019, we used the following weighted-average assumptions in our Black-Scholes calculations: Employee Stock Options: Three Months Ended March 31, 2020 2019 Risk-free interest rate 1.5 % 2.5 % Dividend yield 0.0 % 0.0 % Volatility 73.6 % 76.4 % Expected life 6.1 years 6.1 years Board of Directors Stock Options (1): Three Months Ended March 31, 2020 Risk-free interest rate 1.5 % Dividend yield 0.0 % Volatility 73.3 % Expected life 6.3 years (1) We did not grant stock options to our Board of Directors in the first quarter of 2019. 2017 ESPP: Three Months Ended March 31, 2020 2019 Risk-free interest rate 1.0 % 2.5 % Dividend yield 0.0 % 0.0 % Volatility 71.9 % 64.1 % Expected life 6 months 6 months |
Stock-Based Compensation Expense | The following table summarizes stock-based compensation expense for the three months ended March 31, 2020 and 2019 (in thousands): Three Months Ended March 31, 2020 2019 Cost of sales - product $ 237 $ 118 Research and development expenses 1,349 3,921 Selling, general and administrative expenses 5,696 14,521 Total $ 7,282 $ 18,560 |
Basic and Diluted Net (Loss) _2
Basic and Diluted Net (Loss) Income Per Share (Tables) | 3 Months Ended |
Mar. 31, 2020 | |
Earnings Per Share [Abstract] | |
Basic Loss Per Share | The following table summarizes the distributable (losses) income for the three months ended March 31, 2020 and 2019 (in thousands): Three Months Ended March 31, 2020 2019 Net (loss) income $ (42,850 ) $ 27,187 Distributions to Ionis — (13,492 ) Distributable (losses) income $ (42,850 ) $ 13,695 The following table summarizes the reconciliation of weighted-average shares outstanding used in the calculation of basic (loss) income per share for the three months ended March 31, 2020 and 2019: Three Months Ended March 31, 2020 2019 Determination of shares: Weighted-average common shares outstanding owned by Ionis 77,094,682 68,581,967 Weighted-average common shares outstanding owned by others 24,010,388 22,126,363 Total weighted-average common shares outstanding 101,105,070 90,708,330 The following table summarizes the calculation of basic (loss) income per share for the three months ended March 31, 2020 and 2019 (in thousands, except share and per share amounts): Three Months Ended March 31, 2020 2019 (Losses) income allocated to Ionis $ (32,674 ) $ 10,354 Plus: Distribution to Ionis — 13,492 (Losses) income available to Ionis $ (32,674 ) $ 23,846 Weighted-average common shares outstanding owned by Ionis 77,094,682 68,581,967 Basic (loss) income per common share owned by Ionis $ (0.42 ) $ 0.35 (Losses) income allocated to common shares owned by others $ (10,176 ) $ 3,341 Weighted-average common shares outstanding owned by others 24,010,388 22,126,363 Basic loss per common share owned by others $ (0.42 ) $ 0.15 |
Diluted Loss Per Share | The following table summarizes the reconciliation of weighted-average shares outstanding and diluted common equivalent shares used in the calculation of diluted income per share for the three months ended March 31, 2019: Three Months Ended March 31, 2019 Determination of shares: Weighted-average common shares outstanding owned by Ionis 68,581,967 Weighted-average common shares outstanding owned by others 22,126,363 Shares issuable upon exercise of stock options 3,370,817 Shares issuable upon restricted stock awards issuance 14,813 Shares issuable related to our ESPP 33,982 Weighted-average shares outstanding owned by others, plus assumed conversions 25,545,975 Total weighted-average shares outstanding 94,127,942 The following table summarizes the calculation of diluted income per share for the three months ended March 31, 2019 (in thousands, except per share amounts): Three Months Ended March 31, 2019 Income allocated to Ionis $ 9,978 Plus: Distribution to Ionis 13,492 Income available to Ionis $ 23,470 Weighted-average common shares outstanding owned by Ionis 68,581,967 Diluted income per common share owned by Ionis $ 0.34 Income allocated to common shares owned by others, plus assumed conversions $ 3,717 Weighted-average common shares outstanding owned by others, plus assumed conversions 25,545,975 Diluted income per common share owned by others $ 0.15 |
Contractual Obligations and C_2
Contractual Obligations and Commitments (Tables) | 3 Months Ended |
Mar. 31, 2020 | |
Commitments And Contingencies Disclosure [Abstract] | |
Summary of the Contractual Lease Obligations | The following table summarizes the contractual lease obligations as of March 31, 2020 (in thousands): Years ended December 31, Operating Leases Remainder of 2020 $ 1,877 2021 2,504 2022 2,403 2023 2,400 2024 2,395 Thereafter 9,565 Total minimum lease payments $ 21,144 |
Organization and Basis of Pre_2
Organization and Basis of Presentation (Details) - USD ($) $ in Thousands | 3 Months Ended | ||
Mar. 31, 2020 | Mar. 31, 2019 | Dec. 31, 2019 | |
Basis of Presentation and Organization [Abstract] | |||
Accumulated deficit | $ 524,120 | $ 481,270 | |
Net loss | 42,850 | $ (27,187) | |
Net cash used in operating activities | (44,105) | $ 78,592 | |
Cash, cash equivalents and short-term investments | $ 421,300 | ||
Ionis [Member] | Akcea [Member] | |||
Basis of Presentation and Organization [Abstract] | |||
Ownership percentage by Ionis | 76.00% |
Net Product Revenue - Narrative
Net Product Revenue - Narratives (Details) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2020 | Mar. 31, 2019 | |
Disaggregation Of Revenue [Line Items] | ||
Product revenue, net | $ 16,074 | $ 163,816 |
Product [Member] | ||
Disaggregation Of Revenue [Line Items] | ||
Product revenue, net | $ 15,159 | $ 6,754 |
Net Product Revenue - Summary o
Net Product Revenue - Summary of Balances and Activity in Each of Product Revenue Allowance and Reserve Categories (Details) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2020 | Mar. 31, 2019 | |
Valuation And Qualifying Accounts Disclosure [Line Items] | ||
Balance at December 31, 2018 | $ 1,820 | $ 348 |
Provision related to current period sales | 2,149 | 582 |
Adjustment related to prior period sales | 3 | (30) |
Credits or payments made during the period | (1,052) | (104) |
Balance at March 31, 2019 | 2,920 | 796 |
Chargebacks, Discounts and Fees [Member] | ||
Valuation And Qualifying Accounts Disclosure [Line Items] | ||
Balance at December 31, 2018 | 270 | 50 |
Provision related to current period sales | 470 | 155 |
Adjustment related to prior period sales | (66) | 0 |
Credits or payments made during the period | (498) | (104) |
Balance at March 31, 2019 | 176 | 101 |
Government and Other Rebates [Member] | ||
Valuation And Qualifying Accounts Disclosure [Line Items] | ||
Balance at December 31, 2018 | 1,353 | 293 |
Provision related to current period sales | 1,621 | 375 |
Adjustment related to prior period sales | 69 | (30) |
Credits or payments made during the period | (554) | 0 |
Balance at March 31, 2019 | 2,489 | 638 |
Returns [Member] | ||
Valuation And Qualifying Accounts Disclosure [Line Items] | ||
Balance at December 31, 2018 | 197 | 5 |
Provision related to current period sales | 58 | 52 |
Adjustment related to prior period sales | 0 | 0 |
Credits or payments made during the period | 0 | 0 |
Balance at March 31, 2019 | $ 255 | $ 57 |
Net Product Revenue - Summary_2
Net Product Revenue - Summary of Balance of Receivables and Contract Liabilities Related to Net Product Revenue (Details) - USD ($) $ in Thousands | Mar. 31, 2020 | Dec. 31, 2019 |
Disaggregation Of Revenue [Line Items] | ||
Accounts receivable | $ 10,673 | $ 10,496 |
Contract liabilities included in "Accrued liabilities" | 832 | 2,165 |
Accounts Receivable [Member] | ||
Disaggregation Of Revenue [Line Items] | ||
Accounts receivable | 10,330 | 6,946 |
Accrued Liabilities [Member] | ||
Disaggregation Of Revenue [Line Items] | ||
Contract liabilities included in "Accrued liabilities" | $ 2,645 | $ 1,550 |
Revenue from Collaboration an_3
Revenue from Collaboration and License Agreements - Summary of Net Revenue from Partners in Connection with Collaboration and License Agreements (Details) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2020 | Mar. 31, 2019 | |
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||
Revenue | $ 16,074 | $ 163,816 |
Licensing Revenue and Research and Development and License Revenue Under Collaborative Agreement [Member] | ||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||
Revenue | 915 | 157,062 |
Licensing Revenue and Research and Development and License Revenue Under Collaborative Agreement [Member] | Strategic Collaboration with Novartis [Member] | ||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||
Revenue | 311 | $ 157,062 |
Licensing Revenue and Research and Development and License Revenue Under Collaborative Agreement [Member] | Vupanorsen (AKCEA-ANGPTL3-LRx) License Agreement with Pfizer [Member] | ||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||
Revenue | $ 604 |
Revenue from Collaboration an_4
Revenue from Collaboration and License Agreements - Summary of Receivables and Contract Liabilities Related to Collaboration and License Agreements Associated with Partners (Details) - USD ($) $ in Thousands | Mar. 31, 2020 | Dec. 31, 2019 |
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||
Receivables included in "Accounts receivable" | $ 10,673 | $ 10,496 |
Contract liabilities included in "Deferred revenue" | 832 | 2,165 |
Accounts Receivable [Member] | ||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||
Receivables included in "Accounts receivable" | 10,330 | 6,946 |
Deferred Revenue [Member] | ||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||
Contract liabilities included in "Deferred revenue" | 2,645 | 1,550 |
Collaboration and License Agreements [Member] | Accounts Receivable [Member] | ||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||
Receivables included in "Accounts receivable" | 717 | 3,550 |
Collaboration and License Agreements [Member] | Deferred Revenue [Member] | ||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||
Contract liabilities included in "Deferred revenue" | $ 718 | $ 1,322 |
Revenue from Collaboration an_5
Revenue from Collaboration and License Agreements - Summary of Revenue Recognized of Change in Contract Liability Balances Related to Collaboration and License Agreements Associated with Partners (Details) $ in Thousands | 3 Months Ended |
Mar. 31, 2020USD ($) | |
Collaboration and License Agreements [Member] | |
Revenue recognized in current period from: | |
Amounts included in deferred revenue at the beginning of the period | $ 604 |
Revenue from Collaboration an_6
Revenue from Collaboration and License Agreements - Narratives (Details) | Aug. 01, 2018USD ($) | Oct. 31, 2019USD ($)PerformanceObligation | May 31, 2019USD ($) | Feb. 28, 2019USD ($) | Mar. 31, 2020USD ($) | Dec. 31, 2019USD ($)shares | Jun. 30, 2019USD ($) | Mar. 31, 2019USD ($)shares |
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||
Revenue | $ 16,074,000 | $ 163,816,000 | ||||||
Cardiometabolic License Agreement [Member] | ||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||
Sublicense fee paid | $ 3,000,000 | |||||||
TTR License Agreement [Member] | ||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||
Sublicense fee paid | 2,400,000 | |||||||
Ionis [Member] | Akcea [Member] | ||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||
Sublicense fee payable | $ 75,000,000 | |||||||
Ionis [Member] | Common Stock [Member] | Akcea [Member] | ||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||
Issuance of stock (in shares) | shares | 2,837,373 | |||||||
Licensing [Member] | Akcea [Member] | ||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||
Revenue | $ 150,000,000 | |||||||
AKCEA-APO(a)-L [Member] | ||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||
Maximum amount of payments receivable for milestones | 675,000,000 | |||||||
Maximum amount of payments receivable for development milestones | 25,000,000 | |||||||
Maximum amount of payments receivable for regulatory milestones | 290,000,000 | |||||||
Maximum amount of payments receivable for commercialization milestones | $ 360,000,000 | |||||||
Royalty percentage received on sales of drug | 20.00% | |||||||
AKCEA-APOCIII-LRx [Member] | ||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||
Revenue recognized from deferred revenue | $ 0 | $ 156,200,000 | ||||||
Novartis [Member] | ||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||
Revenue recognized from deferred revenue | 300,000 | $ 900,000 | ||||||
Next prospective milestone | 25,000,000 | |||||||
Deferred revenue | 0 | $ 0 | ||||||
PTC Therapeutics [Member] | ||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||
Revenue | 4,000,000 | $ 6,000,000 | ||||||
Consideration amount received | $ 4,000,000 | $ 6,000,000 | ||||||
PTC Therapeutics [Member] | Akcea [Member] | ||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||
Additional amount of payments receivable per drug for regulatory milestone | $ 4,000,000 | |||||||
Period before PTC pays royalties on net sales of product after first commercial sale in Brazil | 12 months | |||||||
Minimum revenue recognized in Latin America by PTC before paying royalties | $ 10,000,000 | |||||||
PTC Therapeutics [Member] | TEGSEDI [Member] | ||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||
Revenue | 400,000 | |||||||
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% | |||||||
Pfizer Vupanorsen License Agreement [Member] | ||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||
Revenue recognized from deferred revenue | 600,000 | |||||||
Sublicense fee payable | $ 125,000,000 | |||||||
Issuance of stock (in shares) | shares | 6,873,344 | |||||||
Deferred revenue | $ 700,000 | $ 1,300,000 | ||||||
Upfront license fee receivable | 250,000,000 | |||||||
Number of separate performance obligations | PerformanceObligation | 3 | |||||||
Upfront payment received | 250,000,000 | |||||||
Transaction price | 250,000,000 | |||||||
Pfizer Vupanorsen License Agreement [Member] | Transfer of License of AKCEA-ANGPTL3-LRx [Member] | ||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||
Transaction price | 245,600,000 | |||||||
Pfizer Vupanorsen License Agreement [Member] | Development services for AKCEA-ANGPTL3-LRx [Member] | ||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||
Transaction price | 2,200,000 | |||||||
Pfizer Vupanorsen License Agreement [Member] | Delivery of AKCEA-ANGPTL3-LRx API [Member] | ||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||
Transaction price | $ 2,200,000 | |||||||
Pfizer Vupanorsen License Agreement [Member] | AKCEA-ANGPTL3-LRx [Member] | ||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||
Maximum amount of payments receivable for milestones | $ 1,300,000,000 | |||||||
Maximum amount of payments receivable for development milestones | 205,000,000 | |||||||
Maximum amount of payments receivable for regulatory milestones | 250,000,000 | |||||||
Maximum amount of payments receivable for commercialization milestones | 850,000,000 | |||||||
Next prospective milestone | $ 75,000,000 | |||||||
Royalty percentage received on sales of drug | 20.00% |
Investments and Fair Value Me_3
Investments and Fair Value Measurements - Summary of Investments (Details) - USD ($) $ in Thousands | Mar. 31, 2020 | Dec. 31, 2019 |
Summary of Investments [Abstract] | ||
Cost | $ 277,541 | $ 160,761 |
Gross unrealized gains | 1,034 | 75 |
Gross unrealized losses | (593) | (32) |
Estimated fair value | 277,982 | 160,804 |
Securities with Maturity of One Year or Less [Member] | ||
Summary of Investments [Abstract] | ||
Cost | 198,743 | 144,649 |
Gross unrealized gains | 572 | 70 |
Gross unrealized losses | (414) | (29) |
Estimated fair value | 198,901 | 144,690 |
Securities with Maturity of More than One Year [Member] | ||
Summary of Investments [Abstract] | ||
Cost | 78,798 | 16,112 |
Gross unrealized gains | 462 | 5 |
Gross unrealized losses | (179) | (3) |
Estimated fair value | 79,081 | 16,114 |
Corporate Debt Securities [Member] | Securities with Maturity of One Year or Less [Member] | ||
Summary of Investments [Abstract] | ||
Cost | 112,967 | 105,679 |
Gross unrealized gains | 14 | 40 |
Gross unrealized losses | (414) | (23) |
Estimated fair value | 112,567 | 105,696 |
Corporate Debt Securities [Member] | Securities with Maturity of More than One Year [Member] | ||
Summary of Investments [Abstract] | ||
Cost | 40,202 | 2,033 |
Gross unrealized gains | 45 | 5 |
Gross unrealized losses | (175) | |
Estimated fair value | 40,072 | 2,038 |
Debt Securities Issued by U.S. Government Agencies [Member] | Securities with Maturity of One Year or Less [Member] | ||
Summary of Investments [Abstract] | ||
Cost | 85,776 | 38,970 |
Gross unrealized gains | 558 | 30 |
Gross unrealized losses | (6) | |
Estimated fair value | 86,334 | 38,994 |
Debt Securities Issued by U.S. Government Agencies [Member] | Securities with Maturity of More than One Year [Member] | ||
Summary of Investments [Abstract] | ||
Cost | 38,596 | 14,079 |
Gross unrealized gains | 417 | |
Gross unrealized losses | (4) | (3) |
Estimated fair value | $ 39,009 | $ 14,076 |
Investments and Fair Value Me_4
Investments and Fair Value Measurements - Fair Value Measurements (Details) - USD ($) $ in Thousands | Mar. 31, 2020 | Dec. 31, 2019 | |||
Fair Value Measurements [Abstract] | |||||
Available-for-sale securities | $ 277,982 | $ 160,804 | |||
Recurring Basis [Member] | |||||
Fair Value Measurements [Abstract] | |||||
Money market funds | [1] | 119,699 | 285,510 | ||
Total | 397,681 | 446,314 | |||
Recurring Basis [Member] | Corporate Debt Securities [Member] | |||||
Fair Value Measurements [Abstract] | |||||
Available-for-sale securities | 152,639 | [2] | 107,735 | [3] | |
Recurring Basis [Member] | Corporate Debt Securities [Member] | Cash and Cash Equivalents [Member] | |||||
Fair Value Measurements [Abstract] | |||||
Available-for-sale securities | 20,000 | 4,000 | |||
Recurring Basis [Member] | Debt Securities Issued by U.S. Government Agencies [Member] | |||||
Fair Value Measurements [Abstract] | |||||
Available-for-sale securities | [2] | 125,343 | 53,069 | ||
Recurring Basis [Member] | Quoted Prices in Active Markets (Level 1) [Member] | |||||
Fair Value Measurements [Abstract] | |||||
Money market funds | [1] | 119,699 | 285,510 | ||
Total | 119,699 | 285,510 | |||
Recurring Basis [Member] | Significant Other Observable Inputs (Level 2) [Member] | |||||
Fair Value Measurements [Abstract] | |||||
Total | 277,982 | 160,804 | |||
Recurring Basis [Member] | Significant Other Observable Inputs (Level 2) [Member] | Corporate Debt Securities [Member] | |||||
Fair Value Measurements [Abstract] | |||||
Available-for-sale securities | 152,639 | [2] | 107,735 | [3] | |
Recurring Basis [Member] | Significant Other Observable Inputs (Level 2) [Member] | Debt Securities Issued by U.S. Government Agencies [Member] | |||||
Fair Value Measurements [Abstract] | |||||
Available-for-sale securities | [2] | $ 125,343 | $ 53,069 | ||
[1] | Included in cash and cash equivalents on our condensed consolidated balance sheet | ||||
[2] | Included in short-term investments on our condensed consolidated balance sheet | ||||
[3] | At March 31, 2020 and December 31, 2019, $20.0 million and $4.0 million, respectively, was included in cash and cash equivalents on our condensed consolidated balance sheet, with the difference included in short-term investments on our condensed consolidated balance sheet |
Property Plant and Equipment -
Property Plant and Equipment - Summary of Property and Equipment at Cost and Related Accumulated Depreciation (Details) - USD ($) $ in Thousands | Mar. 31, 2020 | Dec. 31, 2019 |
Property Plant And Equipment [Line Items] | ||
Property and equipment, at cost | $ 6,659 | $ 6,271 |
Less accumulated depreciation and amortization | (1,222) | (1,010) |
Total property and equipment, net | 5,437 | 5,261 |
Furniture And Fixtures [Member] | ||
Property Plant And Equipment [Line Items] | ||
Property and equipment, at cost | 1,611 | 1,611 |
Computer Equipment And Software [Member] | ||
Property Plant And Equipment [Line Items] | ||
Property and equipment, at cost | 345 | 289 |
Manufacturing Equipment [Member] | ||
Property Plant And Equipment [Line Items] | ||
Property and equipment, at cost | 439 | 416 |
Leasehold Improvements [Member] | ||
Property Plant And Equipment [Line Items] | ||
Property and equipment, at cost | $ 4,264 | $ 3,955 |
Property Plant Equipment - Narr
Property Plant Equipment - Narratives (Details) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2020 | Mar. 31, 2019 | |
Property Plant And Equipment [Abstract] | ||
Depreciation | $ 212 | $ 207 |
Tenant improvement allowance included | $ 3,600 |
Inventories - Schedule of Inven
Inventories - Schedule of Inventories (Details) - USD ($) $ in Thousands | Mar. 31, 2020 | Dec. 31, 2019 |
Inventory Disclosure [Abstract] | ||
Raw materials | $ 10,476 | $ 6,520 |
Work in process | 2,646 | 2,039 |
Finished goods | 601 | 258 |
Total inventories | $ 13,723 | $ 8,817 |
Intangible Assets, Net - Schedu
Intangible Assets, Net - Schedule of Intangible Assets (Details) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2020 | Dec. 31, 2019 | |
Finite Lived Intangible Assets [Line Items] | ||
Less accumulated amortization | $ (10,673) | $ (9,211) |
Total intangible assets, net | 81,589 | 83,051 |
Acquired and In-licensed Rights [Member] | ||
Finite Lived Intangible Assets [Line Items] | ||
Intangible assets, gross | $ 2,262 | 2,262 |
Acquired and In-licensed Rights [Member] | Minimum [Member] | ||
Finite Lived Intangible Assets [Line Items] | ||
Estimated useful life | 7 years | |
Acquired and In-licensed Rights [Member] | Maximum [Member] | ||
Finite Lived Intangible Assets [Line Items] | ||
Estimated useful life | 21 years | |
Capitalized Regulatory Approval Milestones [Member] | ||
Finite Lived Intangible Assets [Line Items] | ||
Intangible assets, gross | $ 90,000 | $ 90,000 |
Estimated useful life | 16 years |
Intangible Assets, Net - Additi
Intangible Assets, Net - Additional Information (Details) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2020 | Mar. 31, 2019 | |
Finite Lived Intangible Assets [Line Items] | ||
Amortization of intangibles | $ 1,462 | $ 1,446 |
Patents [Member] | ||
Finite Lived Intangible Assets [Line Items] | ||
Weighted average remaining amortizable life | 10 years 8 months 12 days |
Intangible Assets, Net - Sche_2
Intangible Assets, Net - Schedule of Estimated Future Amortization Expenses for Intangible Assets (Details) - USD ($) $ in Thousands | Mar. 31, 2020 | Dec. 31, 2019 |
Goodwill And Intangible Assets Disclosure [Abstract] | ||
Remainder of 2020 | $ 4,417 | |
2021 | 5,861 | |
2022 | 5,856 | |
2023 | 5,843 | |
2024 | 5,822 | |
Thereafter | 53,790 | |
Total intangible assets, net | $ 81,589 | $ 83,051 |
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 | 3 Months Ended |
Mar. 31, 2020USD ($)Patientqtr | |
Development, Commercialization and License Agreement with Ionis [Abstract] | |
Royalty percentage paid on sales of lipid medicines | 20.00% |
First annual sales threshold | $ | $ 500 |
Second annual sales threshold | $ | 1,000 |
Third annual sales threshold | $ | 2,000 |
Sales milestone payment | $ | $ 50 |
Number of quarters in which equal payments are made | qtr | 12 |
Percentage of license fees, milestone payments and royalties paid as sublicense fee to Ionis | 50.00% |
Lipid Drug for Rare Disease Indication [Member] | Maximum [Member] | |
Development, Commercialization and License Agreement with Ionis [Abstract] | |
Number of patients worldwide | Patient | 500,000 |
Number of patients for Phase 3 program | Patient | 1,000 |
Number of years of treatment | 2 years |
Lipid Drug for a Broad Disease Patient Population [Member] | Minimum [Member] | |
Development, Commercialization and License Agreement with Ionis [Abstract] | |
Number of patients worldwide | Patient | 500,000 |
Number of patients for Phase 3 program | Patient | 1,000 |
Number of years of treatment | 2 years |
License Agreements and Servic_4
License Agreements and Services Agreement with Ionis, TTR Development, Commercialization, Collaboration and License Agreement (Details) $ in Thousands | Oct. 17, 2018USD ($)shares | Aug. 03, 2018USD ($)shares | Apr. 17, 2018USD ($)Milestoneshares | Mar. 31, 2020USD ($) | Mar. 31, 2019USD ($) | Dec. 31, 2018USD ($)shares | Dec. 31, 2019USD ($) |
TTR Development, Commercialization, Collaboration and License Agreement [Abstract] | |||||||
Intangible assets, net | $ 81,589 | $ 83,051 | |||||
Amortization expense of intangible assets | 1,419 | $ 1,403 | |||||
Ionis [Member] | |||||||
TTR Development, Commercialization, Collaboration and License Agreement [Abstract] | |||||||
Costs incurred by Ionis prior to closing of TTR Agreement | $ 3,100 | ||||||
Ionis [Member] | TTR License Agreement [Member] | |||||||
TTR Development, Commercialization, Collaboration and License Agreement [Abstract] | |||||||
Upfront license fee paid to Ionis | $ 150,000 | ||||||
Shares issued in consideration of upfront licensing fee (in shares) | shares | 8,000,000 | ||||||
Number of sales milestones | Milestone | 7 | ||||||
Annual worldwide net sales required for subsequent milestone payments to be paid in cash | $ 750,000 | ||||||
Notice period for termination of agreement | 90 days | ||||||
Intangible assets, net | 600 | ||||||
Commercial Inotersen inventory acquired from Ionis | $ 4,700 | ||||||
Clinical material acquired from Ionis | 13,500 | ||||||
Percentage of profits and losses from development and commercialization of Inotersen paid to Ionis, tier 1 | 60.00% | ||||||
Percentage of profits and losses from development and commercialization of Inotersen retained, tier 1 | 40.00% | ||||||
Percentage of profits and losses from development and commercialization of Inotersen paid to Ionis, tier 2 | 50.00% | ||||||
Percentage of profits and losses from development and commercialization of Inotersen retained, tier 2 | 50.00% | ||||||
Percentage of profits and losses from development and commercialization of AKCEA-TTR-L paid to Ionis | 50.00% | ||||||
Percentage of profits and losses from development and commercialization of AKCEA-TTR-L retained | 50.00% | ||||||
Ionis [Member] | TTR License Agreement [Member] | Ionis Stock Purchase Agreement [Member] | |||||||
TTR Development, Commercialization, Collaboration and License Agreement [Abstract] | |||||||
Common stock shares issued to Ionis | shares | 10,700,000 | ||||||
Stock purchased by Ionis | $ 200,000 | ||||||
Ionis [Member] | TTR License Agreement [Member] | Inotersen [Member] | |||||||
TTR Development, Commercialization, Collaboration and License Agreement [Abstract] | |||||||
Maximum amount of payments payable for milestones | $ 110,000 | ||||||
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 | ||||||
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 | ||||||
Ionis [Member] | TTR License Agreement [Member] | TEGSEDI [Member] | |||||||
TTR Development, Commercialization, Collaboration and License Agreement [Abstract] | |||||||
Common stock shares issued to Ionis | shares | 1,671,849 | 1,597,571 | |||||
Amount of regulatory approval milestone paid by issuing common stock | $ 50,000 | $ 40,000 | |||||
Amortization expense of intangible assets | $ 1,400 | $ 1,400 | |||||
Ionis [Member] | TTR License Agreement [Member] | TEGSEDI [Member] | Milestone Payment [Member] | |||||||
TTR Development, Commercialization, Collaboration and License Agreement [Abstract] | |||||||
Useful life of milestone payment amortized to cost of sales | 16 years |
License Agreements and Servic_5
License Agreements and Services Agreement with Ionis - Summary of Loss Share Related to Commercial Activities Under TTR Agreement (Details) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2020 | Mar. 31, 2019 | |
Related Party Transaction [Line Items] | ||
Net losses incurred by the collaboration related to the commercial activities under the TTR License Agreement | $ (11,802) | $ (15,098) |
Akcea [Member] | ||
Related Party Transaction [Line Items] | ||
Akcea's share of commercial losses under the TTR License Agreement reflected in our condensed consolidated statement of operations | (4,751) | (6,042) |
Ionis [Member] | ||
Related Party Transaction [Line Items] | ||
Net loss share from commercial activities under arrangement with Ionis Pharmaceuticals, Inc. | $ (7,051) | $ (9,056) |
License Agreements and Servic_6
License Agreements and Services Agreement with Ionis - Summary of Development Expenses Related to TTR Agreement (Details) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2020 | Mar. 31, 2019 | |
Related Party Transaction [Line Items] | ||
Total development expense incurred by the collaboration related to development activities under the TTR License Agreement | $ (18,331) | $ (14,380) |
Akcea [Member] | ||
Related Party Transaction [Line Items] | ||
Akcea's share of TTR development expense reflected in research and development expense in our condensed consolidated statement of operations | $ (8,190) | $ (6,133) |
License Agreements and Servic_7
License Agreements and Services Agreement with Ionis, Services Agreement (Details) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2020 | Mar. 31, 2019 | |
Services Agreement with Ionis [Abstract] | ||
Payment term after receipt of invoice | 30 days | |
Term of extension for services agreement | 6 months | |
Ionis [Member] | Services Agreement [Member] | ||
Operating expenses: | ||
Services performed by Ionis | $ 1,903 | $ 1,075 |
Sublicensing expenses | 75,000 | |
Out-of-pocket expenses paid by Ionis | 585 | 1,571 |
Royalty expenses | 804 | |
Less: commercial share of loss in connection with the TTR license transaction | (6,898) | (9,056) |
Less: R&D share of loss (income) in connection with the TTR license transaction | 1,174 | (796) |
Total operating (income) expenses generated by transactions with ions | (2,432) | 67,794 |
Plus: distribution to Ionis | 13,492 | |
Total net charges generated by transactions with Ionis | (2,432) | 81,286 |
(Receivable) payable balance (from) to Ionis at the beginning of the period | (3,231) | 18,901 |
Less: total amounts received from (paid to) Ionis during the period | 3,231 | (32,393) |
Less: non-cash sublicensing expenses | (75,000) | |
Total amount (receivable) payable (from) to Ionis at period end | $ (2,432) | $ (7,206) |
Stock-Based Compensation - Stoc
Stock-Based Compensation - Stock Plans (Details) - USD ($) $ in Millions | 3 Months Ended | ||
Mar. 31, 2020 | Jan. 01, 2020 | Dec. 31, 2019 | |
Stock Plans [Abstract] | |||
Shares of common stock added | 101,348,633 | 100,993,173 | |
Unrecognized compensation expense related to non-vested stock options | $ 42.9 | ||
RSUs [Member] | |||
Stock Plans [Abstract] | |||
Unrecognized compensation expense related to RSUs | $ 18.9 | ||
Weighted average amortization period | 2 years 9 months 18 days | ||
Non-vested Stock Options [Member] | |||
Stock Plans [Abstract] | |||
Weighted average amortization period | 1 year 7 months 6 days | ||
2015 Equity Incentive Plan [Member] | |||
Stock Plans [Abstract] | |||
Number of shares authorized for issuance pursuant to stock awards (in shares) | 18,500,000 | ||
Number of shares available for future grant (in shares) | 1,479,402 | ||
2015 Equity Incentive Plan [Member] | Stock Options [Member] | |||
Stock Plans [Abstract] | |||
Options outstanding (in shares) | 10,645,227 | ||
Options exercisable (in shares) | 3,463,767 | ||
2015 Equity Incentive Plan [Member] | RSUs [Member] | |||
Stock Plans [Abstract] | |||
Restricted stock units outstanding (in shares) | 1,535,512 | ||
2017 Employee Stock Purchase Plan [Member] | |||
Stock Plans [Abstract] | |||
Number of shares available for future grant (in shares) | 1,893,951 | ||
Shares of common stock added | 500,000 | ||
Purchase price of common stock, percent | 85.00% | ||
Number of shares of common stock reserved for issuance (in shares) | 2,000,000 | ||
Shares issued under ESPP (in shares) | 34,774 | ||
Accrued compensation for contributions to employee stock purchase plan | $ 0.1 |
Stock-Based Compensation - St_2
Stock-Based Compensation - Stock-based Valuation Information (Details) | 3 Months Ended | |
Mar. 31, 2020 | Mar. 31, 2019 | |
Employee Stock Option [Member] | ||
Weighted-Average Assumptions [Abstract] | ||
Risk-free interest rate | 1.50% | 2.50% |
Dividend yield | 0.00% | 0.00% |
Volatility | 73.60% | 76.40% |
Expected life | 6 years 1 month 6 days | 6 years 1 month 6 days |
Board of Director Stock Options [Member] | ||
Weighted-Average Assumptions [Abstract] | ||
Risk-free interest rate | 1.50% | |
Dividend yield | 0.00% | |
Volatility | 73.30% | |
Expected life | 6 years 3 months 18 days | |
2017 ESPP | ||
Weighted-Average Assumptions [Abstract] | ||
Risk-free interest rate | 1.00% | 2.50% |
Dividend yield | 0.00% | 0.00% |
Volatility | 71.90% | 64.10% |
Expected life | 6 months | 6 months |
Stock-Based Compensation - St_3
Stock-Based Compensation - Stock-based Valuation Information (Parenthetical) (Details) | 3 Months Ended |
Mar. 31, 2020shares | |
Board of Director Stock Options [Member] | |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |
Stock options granted | 0 |
Stock-Based Compensation - St_4
Stock-Based Compensation - Stock-Based Compensation Expense (Details) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2020 | Mar. 31, 2019 | |
Stock-Based Compensation [Abstract] | ||
Stock-based compensation expense | $ 7,282 | $ 18,560 |
Cost of Sales - Product [Member] | ||
Stock-Based Compensation [Abstract] | ||
Stock-based compensation expense | 237 | 118 |
Research and Development Expenses [Member] | ||
Stock-Based Compensation [Abstract] | ||
Stock-based compensation expense | 1,349 | 3,921 |
Selling, General and Administrative Expenses [Member] | ||
Stock-Based Compensation [Abstract] | ||
Stock-based compensation expense | $ 5,696 | $ 14,521 |
Basic and Diluted Net (Loss) _3
Basic and Diluted Net (Loss) Income Per Share (Details) - USD ($) $ / shares in Units, $ in Thousands | 3 Months Ended | |
Mar. 31, 2020 | Mar. 31, 2019 | |
Basic and Diluted Net (Loss) Income Per Share [Abstract] | ||
Net (loss) income | $ (42,850) | $ 27,187 |
Distributions to Ionis | (13,492) | |
Distributable (losses) income | $ (42,850) | $ 13,695 |
Weighted-average shares outstanding owned | 94,127,942 | |
Shares issuable related to our ESPP | 33,982 | |
Weighted-average shares outstanding owned by others, plus assumed conversions | 25,545,975 | |
Stock Options [Member] | ||
Basic and Diluted Net (Loss) Income Per Share [Abstract] | ||
Weighted-average shares issuable | 3,370,817 | |
Restricted Stock [Member] | ||
Basic and Diluted Net (Loss) Income Per Share [Abstract] | ||
Weighted-average shares issuable | 14,813 | |
Common Stock [Member] | ||
Basic and Diluted Net (Loss) Income Per Share [Abstract] | ||
Weighted-average common shares outstanding owned | 101,105,070 | 90,708,330 |
Ionis [Member] | Common Stock [Member] | ||
Basic and Diluted Net (Loss) Income Per Share [Abstract] | ||
Net (loss) income | $ (32,674) | $ 23,846 |
Distributions to Ionis | 13,492 | |
Distributable (losses) income | $ (32,674) | $ 10,354 |
Weighted-average common shares outstanding owned | 77,094,682 | 68,581,967 |
Basic (loss) income per common share | $ (0.42) | $ 0.35 |
Weighted-average shares outstanding owned | 77,094,682 | 68,581,967 |
Income allocated to Ionis | $ 9,978 | |
Income available to Ionis | $ 23,470 | |
Diluted income per common share | $ (0.42) | $ 0.34 |
Others [Member] | Common Stock [Member] | ||
Basic and Diluted Net (Loss) Income Per Share [Abstract] | ||
Distributable (losses) income | $ (10,176) | $ 3,341 |
Weighted-average common shares outstanding owned | 24,010,388 | 22,126,363 |
Basic (loss) income per common share | $ (0.42) | $ 0.15 |
Weighted-average shares outstanding owned | 24,010,388 | 25,545,975 |
Weighted-average shares outstanding owned by others, plus assumed conversions | 25,545,975 | |
Income allocated to Ionis | $ 3,717 | |
Diluted income per common share | $ (0.42) | $ 0.15 |
Contractual Obligations and C_3
Contractual Obligations and Commitments - Narratives (Details) $ in Millions | May 08, 2019 | Nov. 12, 2018ft² | Aug. 15, 2018USD ($) | Apr. 05, 2018USD ($)ft² | Mar. 31, 2020USD ($) |
Operating Leased Assets [Line Items] | |||||
Agreement to purchase goods and services | $ 0.9 | ||||
Dublin, Ireland [Member] | |||||
Operating Leased Assets [Line Items] | |||||
Lessee operating lease, lease commencement date | May 31, 2019 | ||||
Initial term of lease | 18 months | ||||
Operating lease, option to extend | a 12-month extension period in our right-of-use asset and lease liability | ||||
Lessee, Operating Lease, Existence of Option to Extend | true | ||||
Office Space for Corporate Headquarters [Member] | |||||
Operating Leased Assets [Line Items] | |||||
Lessee operating lease, lease commencement date | Aug. 15, 2018 | ||||
Area of office space under lease agreement | ft² | 30,175 | ||||
Initial term of lease | 123 months | ||||
Operating lease, option to extend | option to extend the lease for an additional five-year term | ||||
Term of lease extension | 5 years | ||||
Tenant improvement allowance | $ 3.6 | ||||
Period of free rent | 3 months | ||||
Initial letter of credit to secure lease obligation | $ 2.4 | ||||
Letter of credit to secure lease obligation on third anniversary of rent commencement date | 1.8 | ||||
Letter of credit to secure lease obligation on fifth anniversary of rent commencement date | $ 1.2 | ||||
Ionis Sublease | |||||
Operating Leased Assets [Line Items] | |||||
Area of office space under lease agreement | ft² | 4,723 | ||||
Initial term of lease | 64 months | ||||
Period of free rent | 4 months | ||||
Lessee, Operating Lease, Existence of Option to Extend | false |
Contractual Obligations and C_4
Contractual Obligations and Commitments - Summary of the Contractual Lease Obligations (Details) $ in Thousands | Mar. 31, 2020USD ($) |
Commitments And Contingencies Disclosure [Abstract] | |
Remainder of 2020 | $ 1,877 |
2021 | 2,504 |
2022 | 2,403 |
2023 | 2,400 |
2024 | 2,395 |
Thereafter | 9,565 |
Total minimum lease payments | $ 21,144 |
Income Taxes - Additional Infor
Income Taxes - Additional Information (Details) - USD ($) $ in Thousands | Mar. 27, 2020 | Mar. 31, 2020 | Mar. 31, 2019 |
Income Tax Disclosure [Abstract] | |||
Income tax expense (benefit) | $ (768) | $ 132 | |
Tax benefit related to Coronavirus, Aid, Relief and Economic Security Act, or CARES Act | $ 1,700 |