Document and Entity Information
Document and Entity Information - shares | 6 Months Ended | |
Jun. 30, 2019 | Aug. 01, 2019 | |
Document And Entity Information [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 | Jun. 30, 2019 | |
Amendment Flag | false | |
Trading Symbol | AKCA | |
Current Fiscal Year End Date | --12-31 | |
Entity Filer Category | Accelerated Filer | |
Entity Common Stock Shares Outstanding | 93,032,386 | |
Document Fiscal Year Focus | 2019 | |
Document Fiscal Period Focus | Q2 | |
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 | MA | |
Document Quarterly Report | true | |
Document Transition Report | false |
CONDENSED CONSOLIDATED BALANCE
CONDENSED CONSOLIDATED BALANCE SHEETS (Unaudited) - USD ($) $ in Thousands | Jun. 30, 2019 | Dec. 31, 2018 |
Current assets: | ||
Cash and cash equivalents | $ 150,234 | $ 86,454 |
Short-term investments | 145,374 | 166,155 |
Accounts receivable | 9,193 | 4,597 |
Receivable from Ionis Pharmaceuticals, Inc. | 7,911 | |
Inventories | 8,286 | 85 |
Other current assets | 6,713 | 9,944 |
Total current assets | 327,711 | 267,235 |
Property, plant and equipment, net | 5,443 | 5,696 |
Operating lease right-of-use assets | 11,534 | |
Intangible assets, net | 86,006 | 88,914 |
Deposits and other assets | 3,426 | 3,416 |
Total assets | 434,120 | 365,261 |
Current liabilities: | ||
Accounts payable | 7,936 | 12,068 |
Payable to Ionis Pharmaceuticals, Inc. | 18,901 | |
Accrued compensation | 7,521 | 8,583 |
Accrued liabilities | 17,569 | 14,787 |
Current portion of deferred revenue | 15,830 | 25,354 |
Other current liabilities | 1,713 | 968 |
Total current liabilities | 50,569 | 80,661 |
Long-term portion of lease liabilities | 14,909 | 4,442 |
Long-term portion of deferred revenue | 3,434 | |
Total liabilities | 65,478 | 88,537 |
Stockholders’ equity: | ||
Common stock, $0.001 par value; 125,000,000 shares authorized at June 30, 2019 and December 31, 2018; 92,921,173 and 89,345,978 shares issued and outstanding at June 30, 2019 and December 31, 2018, respectively. | 93 | 89 |
Additional paid-in capital | 900,837 | 799,001 |
Accumulated other comprehensive loss | (110) | (324) |
Accumulated deficit | (532,178) | (522,042) |
Total stockholders’ equity | 368,642 | 276,724 |
Total liabilities and stockholders’ equity | $ 434,120 | $ 365,261 |
CONDENSED CONSOLIDATED BALANC_2
CONDENSED CONSOLIDATED BALANCE SHEETS (Unaudited) (Parenthetical) - $ / shares | Jun. 30, 2019 | Dec. 31, 2018 |
Stockholders’ equity: | ||
Common stock, par value (in dollars per share) | $ 0.001 | $ 0.001 |
Common stock, shares authorized (in shares) | 125,000,000 | 125,000,000 |
Common stock, shares issued (in shares) | 92,921,173 | 89,345,978 |
Common stock, shares outstanding (in shares) | 92,921,173 | 89,345,978 |
CONDENSED CONSOLIDATED STATEMEN
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS (Unaudited) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2019 | Jun. 30, 2018 | Jun. 30, 2019 | Jun. 30, 2018 | |
Revenue: | ||||
Revenue | $ 26,623 | $ 18,321 | $ 190,439 | $ 35,429 |
Expenses: | ||||
Cost of sales - intangible asset amortization | 1,419 | 2,822 | ||
Research and development | 20,271 | 39,457 | 119,890 | 67,427 |
Selling, general and administrative | 50,740 | 42,287 | 95,342 | 61,752 |
Total expenses | 65,329 | 81,744 | 202,938 | 129,179 |
Loss from operations | (38,706) | (63,423) | (12,499) | (93,750) |
Other income (expense): | ||||
Investment income | 1,571 | 1,546 | 2,795 | 2,414 |
Other income (expense) | (28) | 45 | (140) | (123) |
Loss before income tax expense | (37,163) | (61,832) | (9,844) | (91,459) |
Income tax expense | (160) | (214) | (292) | (214) |
Net loss | (37,323) | (62,046) | (10,136) | (91,673) |
Ionis [Member] | ||||
Expenses: | ||||
Net loss share from commercial activities under arrangement with Ionis Pharmaceuticals, Inc. | (11,465) | (20,521) | ||
Common Stock [Member] | Ionis [Member] | ||||
Other income (expense): | ||||
Net loss | $ (28,244) | $ (43,814) | $ (4,380) | $ (63,198) |
Net loss per share, basic and diluted (in dollars per share) | $ (0.40) | $ (0.72) | $ (0.06) | $ (1.19) |
Weighted-average shares outstanding, basic and diluted (in shares) | 70,221,338 | 60,832,494 | 69,406,181 | 53,182,685 |
Common Stock [Member] | Others [Member] | ||||
Other income (expense): | ||||
Net loss per share, basic and diluted (in dollars per share) | $ (0.40) | $ (0.85) | $ (0.26) | $ (1.33) |
Weighted-average shares outstanding, basic and diluted (in shares) | 22,573,900 | 21,492,157 | 22,351,368 | 21,332,650 |
Product [Member] | ||||
Revenue: | ||||
Revenue | $ 9,865 | $ 16,619 | ||
Expenses: | ||||
Cost of product | 4,364 | 5,405 | ||
Licensing [Member] | ||||
Revenue: | ||||
Revenue | 6,036 | 6,036 | ||
Research and Development and License Revenue Under Collaborative Agreement [Member] | ||||
Revenue: | ||||
Revenue | $ 10,722 | $ 18,321 | $ 167,784 | $ 35,429 |
CONDENSED CONSOLIDATED STATEM_2
CONDENSED CONSOLIDATED STATEMENTS OF COMPREHENSIVE LOSS (Unaudited) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2019 | Jun. 30, 2018 | Jun. 30, 2019 | Jun. 30, 2018 | |
Statement Of Income And Comprehensive Income [Abstract] | ||||
Net loss | $ (37,323) | $ (62,046) | $ (10,136) | $ (91,673) |
Unrealized gains on debt securities, net of tax | 10 | 151 | 222 | 106 |
Currency translation adjustment | (93) | 20 | (8) | 48 |
Comprehensive loss | $ (37,406) | $ (61,875) | $ (9,922) | $ (91,519) |
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 Loss [Member] | Accumulated Other Comprehensive Loss [Member]Ionis [Member]TTR License Agreement [Member] | Accumulated Deficit [Member] | Accumulated Deficit [Member]Ionis [Member]TTR License Agreement [Member] |
Balance at Dec. 31, 2017 | $ 167,825 | $ 67 | $ 464,430 | $ (451) | $ (296,221) | |||||
Balance (in shares) at Dec. 31, 2017 | 66,542 | |||||||||
Net loss | (91,673) | $ 0 | 0 | 0 | (91,673) | |||||
Change in unrealized gains, net of tax | 106 | 0 | 0 | 106 | 0 | |||||
Currency translation adjustment | 48 | 0 | 0 | 48 | 0 | |||||
Exercise of common stock options | 3,174 | $ 0 | 3,174 | 0 | 0 | |||||
Exercise of common stock options (in shares) | 456 | |||||||||
Issuance of stock | $ 200,100 | $ 19 | $ 200,081 | $ 0 | $ 0 | |||||
Issuance of stock (in shares) | 18,667 | |||||||||
Distribution in connection with license transaction | (7,792) | $ 0 | (7,792) | 0 | 0 | |||||
Issuance of common stock in connection with employee stock purchase plan | 107 | $ 0 | 107 | 0 | 0 | |||||
Issuance of common stock in connection with employee stock purchase plan (in shares) | 16 | |||||||||
Stock-based compensation expense | 18,509 | $ 0 | 18,509 | 0 | 0 | |||||
Balance at Jun. 30, 2018 | 290,404 | $ 86 | 678,509 | (297) | (387,894) | |||||
Balance (in shares) at Jun. 30, 2018 | 85,681 | |||||||||
Balance at Mar. 31, 2018 | 146,300 | $ 67 | 472,549 | (468) | (325,848) | |||||
Balance (in shares) at Mar. 31, 2018 | 66,804 | |||||||||
Net loss | (62,046) | $ 0 | 0 | 0 | (62,046) | |||||
Change in unrealized gains, net of tax | 151 | 151 | ||||||||
Currency translation adjustment | 20 | 0 | 0 | 20 | 0 | |||||
Exercise of common stock options | 1,545 | $ 0 | 1,545 | 0 | 0 | |||||
Exercise of common stock options (in shares) | 210 | |||||||||
Issuance of stock | 200,100 | $ 19 | 200,081 | 0 | 0 | |||||
Issuance of stock (in shares) | 18,667 | |||||||||
Distribution in connection with license transaction | (7,792) | $ 0 | (7,792) | $ 0 | $ 0 | |||||
Stock-based compensation expense | 12,126 | $ 0 | 12,126 | 0 | 0 | |||||
Balance at Jun. 30, 2018 | 290,404 | $ 86 | 678,509 | (297) | (387,894) | |||||
Balance (in shares) at Jun. 30, 2018 | 85,681 | |||||||||
Balance at Dec. 31, 2018 | 276,724 | $ 89 | 799,001 | (324) | (522,042) | |||||
Balance (in shares) at Dec. 31, 2018 | 89,346 | |||||||||
Net loss | (10,136) | (10,136) | ||||||||
Change in unrealized gains, net of tax | 222 | 222 | ||||||||
Currency translation adjustment | (8) | (8) | ||||||||
Exercise of common stock options | 7,027 | $ 1 | 7,026 | |||||||
Exercise of common stock options (in shares) | 720 | |||||||||
Issuance of stock | 75,000 | $ 3 | 74,997 | |||||||
Issuance of stock (in shares) | 2,837 | |||||||||
Distribution in connection with license transaction | $ (13,492) | $ (13,492) | ||||||||
Issuance of common stock in connection with employee stock purchase plan | 382 | 382 | ||||||||
Issuance of common stock in connection with employee stock purchase plan (in shares) | 18 | |||||||||
Stock-based compensation expense | 32,923 | 32,923 | ||||||||
Balance at Jun. 30, 2019 | 368,642 | $ 93 | 900,837 | (110) | (532,178) | |||||
Balance (in shares) at Jun. 30, 2019 | 92,921 | |||||||||
Balance at Mar. 31, 2019 | 388,864 | $ 93 | 883,653 | (27) | (494,855) | |||||
Balance (in shares) at Mar. 31, 2019 | 92,635 | |||||||||
Net loss | (37,323) | $ 0 | 0 | 0 | (37,323) | |||||
Change in unrealized gains, net of tax | 10 | 0 | 0 | 10 | 0 | |||||
Currency translation adjustment | (93) | 0 | 0 | (93) | 0 | |||||
Exercise of common stock options | 2,821 | $ 0 | 2,821 | 0 | 0 | |||||
Exercise of common stock options (in shares) | 286 | |||||||||
Stock-based compensation expense | 14,363 | $ 0 | 14,363 | 0 | 0 | |||||
Balance at Jun. 30, 2019 | $ 368,642 | $ 93 | $ 900,837 | $ (110) | $ (532,178) | |||||
Balance (in shares) at Jun. 30, 2019 | 92,921 |
CONDENSED CONSOLIDATED STATEM_4
CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS (Unaudited) - USD ($) $ in Thousands | 6 Months Ended | |
Jun. 30, 2019 | Jun. 30, 2018 | |
Operating activities: | ||
Net loss | $ (10,136) | $ (91,673) |
Adjustments to reconcile net loss to net cash provided by (used in) operating activities: | ||
Depreciation | 400 | 54 |
Amortization of right-of-use operating lease assets | 501 | |
Amortization of intangibles | 2,908 | 70 |
Amortization of discount/premium on investment securities, net | (434) | 208 |
Non-cash sublicensing expense | 75,000 | |
Stock-based compensation expense | 32,923 | 18,509 |
Changes in operating assets and liabilities: | ||
Accounts receivable | (4,596) | 2,118 |
Other current and long-term assets | (966) | (2,275) |
Inventory | (4,014) | |
Accounts payable | (2,890) | 325 |
Payable/receivable to/from Ionis Pharmaceuticals, Inc. | (26,812) | 12,772 |
Accrued compensation | (1,062) | 465 |
Accrued liabilities | 2,128 | 17,976 |
Income taxes payable | (169) | (720) |
Deferred revenue | (12,958) | (29,141) |
Net cash provided by (used in) operating activities | 49,823 | (71,312) |
Investing activities: | ||
Purchases of short-term investments | (78,606) | (22,197) |
Proceeds from maturity of short-term investments | 100,043 | 94,736 |
Purchases of property, plant and equipment | (1,389) | (5) |
Net cash provided by investing activities | 20,048 | 72,534 |
Financing activities: | ||
Proceeds from exercise of common stock options and employee stock purchase plan issuances | 7,409 | 3,281 |
Distribution to Ionis | (13,492) | (7,792) |
Proceeds from issuance of common stock to Ionis in TTR transaction | 164,098 | |
Net cash (used in) provided by financing activities | (6,083) | 159,587 |
Effect of exchange rates on cash | (8) | 48 |
Net increase in cash and cash equivalents | 63,780 | 160,857 |
Cash, cash equivalents and restricted cash at beginning of period | 88,838 | 58,367 |
Cash, cash equivalents and restricted cash at end of period | 152,618 | 219,224 |
Supplemental disclosures of non-cash operating and financing activities: | ||
Right-of-use assets obtained in exchange for lease liabilities | 363 | |
Purchases of property, plant and equipment included in accrued liabilities | 491 | |
Purchases of property, plant and equipment included in long-term portion of deferred rent | 1,004 | |
Acquisition of research and development licenses | 563 | |
Unpaid deferred offering costs | 450 | |
Cash, cash equivalents and restricted cash reported within the condensed consolidated balance sheets: | ||
Cash and cash equivalents | 150,234 | 216,840 |
Restricted cash included in deposits and other assets | 2,384 | 2,384 |
Cash, cash equivalents and restricted cash at end of period | $ 152,618 | $ 219,224 |
Organization and Basis of Prese
Organization and Basis of Presentation | 6 Months Ended |
Jun. 30, 2019 | |
Organization Consolidation And Presentation Of Financial Statements [Abstract] | |
Organization and Basis of Presentation | 1. Organization and Basis of Presentation We were incorporated in Delaware in December 2014. We were organized by Ionis Pharmaceuticals, Inc., or Ionis, to focus on developing and commercializing drugs to treat patients with rare and serious diseases. On July 19, 2017, we completed our initial public offering, or IPO. As of June 30, 2019, Ionis owned approximately 76% of our common stock and is our majority shareholder. Prior to our IPO, we were wholly owned by Ionis. The accompanying 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, which we considered necessary for a fair presentation of our financial position and our operating results and cash flows for the interim periods ended June 30, 2019 and 2018. Certain prior year amounts have been reclassified for consistency with the current year presentation. These reclassifications had no effect on the reported results of operations. In accordance with Accounting Standards Codification, or ASC, 205-40, Going Concern |
Summary of Significant Accounti
Summary of Significant Accounting Policies | 6 Months Ended |
Jun. 30, 2019 | |
Accounting Policies [Abstract] | |
Summary of Significant Accounting Policies | 2. Summary of Significant Accounting Policies The accounting policies followed in the preparation of the 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, 2018. 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 and the accrual for research and development expenses. Estimates are periodically reviewed in light of changes in facts, circumstances and experience. Changes in estimates are recorded in the period in which they become known. Actual results could differ from those estimates. Translation of Foreign Currency For our foreign subsidiaries that report in a functional currency other than U.S. dollars, we translate their assets and liabilities into U.S. dollars using the exchange rate at the balance sheet date. We translate revenue and expenses at the monthly average exchange rates for the period. We translate transactions in our capital accounts at the historic exchange rate in effect at the date of the transaction. We include foreign currency translation adjustments as a component of accumulated other comprehensive loss within the condensed consolidated statements of comprehensive loss. Revenue Recognition Collaboration and License Revenue In May 2014, the Financial Accounting Standards Board, or FASB, issued Accounting Standards Update, or ASU, No. 2014-09, Revenue from Contracts with Customers To determine revenue recognition for arrangements that an entity determines are within the scope of Topic 606, the entity performs the following five steps: (i) identify the contract(s) with a customer; (ii) identify the performance obligations in the contract; (iii) determine the transaction price; (iv) allocate the transaction price to the performance obligations in the contract; and (v) recognize revenue when (or as) the entity satisfies a performance obligation. At contract inception, once the contract is determined to be within the scope of Topic 606, we assess the goods or services promised within each contract and determine those that are performance obligations, then assess whether each promised good or service is distinct. When we offer options for additional goods or services, such as an option to license a drug in the future or for additional goods or services to be provided in the future, we evaluate whether such options are material rights that should be treated as additional performance obligations. We typically have concluded that the option to license a drug or the options for additional goods or services that may be requested in the future under our collaboration agreements are not material rights as the amounts attributable to such options represent standalone selling price, and therefore no consideration is allocated to these items at the inception of an agreement. When a partner exercises its option to license a drug or requests the additional goods or services, a new performance obligation is created for that item. Once performance obligations are identified, we then recognize as revenue the amount of the transaction price that we allocated to the respective performance obligation when (or as) each performance obligation is satisfied, either at a point in time or over time. If the performance obligation is satisfied over time, we recognize revenue based on the use of an output or input method. As of June 30, 2019, we have three revenue agreements: our strategic collaboration, option and license agreement, or collaboration agreement, with Novartis Pharma AG, or Novartis, which we entered into in January 2017, our collaboration and license agreement with PTC Therapeutics International Limited, or PTC Therapeutics, which we entered into in August 2018, and our TTR development, commercialization, collaboration and license agreement with Ionis, under which we are recognizing commercial product revenue related to TEGSEDI sales subsequent to product launch in the fourth quarter of 2018. For a complete discussion of the accounting related to our collaborative agreements, see Note 7, Strategic Collaboration with Novartis, Collaboration and License Agreement with PTC Therapeutics Product Revenue, Net Subsequent to regulatory approval in Europe on July 11, 2018 and FDA approval in the U.S. on October 5, 2018, in the fourth quarter of 2018, we began to sell TEGSEDI in the U.S. and Europe. In the U.S., the product is distributed through an exclusive distribution agreement with a third-party logistics, or 3PL, company that takes title to the product and represents our sole customer in the U.S. Our U.S. customer distributes TEGSEDI to a specialty pharmacy and a specialty distributor (collectively referred to as “wholesalers”), who then distribute the product to health care providers and patients. In Europe, the product is currently distributed through a non-exclusive distribution model with a 3PL that takes title to the product and currently is our sole customer in Europe. Our customer then distributes TEGSEDI to hospitals and pharmacies in Europe. Revenue from product sales is recognized when the customer obtains control of our product, which occurs upon transfer of title to the customer. We record shipping and handling costs within cost of goods sold on our condensed consolidated statement of operations. We classify payments to customers or other parties in the distribution channel for services that are distinct and priced at fair value as selling, general and administrative expenses in our condensed consolidated statements of operations. Payments to customers or other parties in the distribution channel that do not meet those criteria are classified as a reduction of revenue, as discussed further below. Taxes collected from customers relating to product sales and remitted to governmental authorities are excluded from revenue. We have elected not to adjust consideration for the effects of a significant financing component when the period between the transfer of a promised good or service to the customer and when the customer pays for that good or service will be one year or less. Our payment terms are generally between thirty to ninety days. Reserves for Variable Consideration Revenue from product sales is recorded at the net sales price (transaction price), which includes estimates of variable consideration for which reserves are established and which result from discounts, returns, chargebacks, rebates, co-pay assistance and other allowances that are offered within contracts between us and our customers, wholesalers, health care providers and other indirect customers relating to the sale of TEGSEDI. These reserves are based on the amounts earned or to be claimed on the related sales and are classified as reductions of accounts receivable (if no payments are required of us) or a current liability (if a payment is required by us). Where appropriate, these estimates take into consideration a range of possible outcomes that are probability-weighted for relevant factors such as our historical experience, current contractual and statutory requirements, specific known market events and trends, industry data and forecasted customer buying and payment patterns. Overall, product revenue net of these reserves reflects our best estimates of the amount of consideration to which we are entitled based on the terms of the contract. The amount of variable consideration that is included in the transaction price may be constrained and is included in the net sales price only to the extent that it is considered probable that a significant reversal in the amount of the cumulative revenue recognized will not occur in a future period. Actual amounts of consideration ultimately received may differ from our estimates. If actual results in the future vary from our estimates, we will adjust these estimates, which would affect net product revenue and earnings in the period such variances become known. The following are the components of variable consideration related to product revenue: Chargebacks: In the U.S., we estimate obligations resulting from contractual commitments with the government and other entities to sell products to qualified healthcare providers at prices lower than the list prices charged to our U.S. customer. Our U.S. customer charges us for the difference between what they pay for the product and the selling price to the qualified healthcare providers. We record reserves for these chargebacks related to product sold to our U.S. customer during the reporting period, as well as our estimate of product that remains in the distribution channel at the end of the reporting period that we expect will be sold to qualified healthcare providers in future periods. Government rebates: We are subject to discount obligations under government programs, including Medicaid and Medicare programs in the U.S., and we record reserves for government rebates based on statutory discount rates and estimated utilization in the period in which revenue is recognized. We estimate Medicaid and Medicare rebates based upon estimated payer mix. These reserves are recorded in the same period the related revenue is recognized, resulting in a reduction of product revenue and the establishment of a liability that is included in accrued expenses on our condensed consolidated balance sheet. For Medicare, we also estimate the number of patients in the prescription drug coverage gap for whom we expect we will owe an additional liability under the Medicare Part D program. On a quarterly basis, we update our estimates and record any adjustments in the period that we identify the adjustments. Trade discounts and allowances: We provide customary invoice discounts on TEGSEDI sales to our U.S. customer for prompt payment that are recorded as a reduction of revenue in the period the related product revenue is recognized. Distribution fees: We receive and pay for various distribution services provided by our U.S. and E.U. customers and wholesalers in the U.S. distribution channel and these fees are generally accounted for as a reduction of revenue. To the extent that the services received are distinct from the sale of products to our customers, these payments are accounted for as selling, general and administrative expenses. Product Returns: Our U.S. customer has return rights and the wholesalers have limited return rights primarily related to the product’s expiration date. We estimate the amount of product sales that may be returned and record the estimate as a reduction of revenue and a refund liability in the period the related product revenue is recognized. Based on the distribution model for TEGSEDI, contractual inventory limits with our U.S. customer and wholesalers and the price of TEGSEDI, we believe there will be minimal returns in the U.S. Our E.U. customer only takes title to the product once it receives an order from a hospital or pharmacy and therefore does not maintain any inventory of TEGSEDI. Accordingly, there is limited return risk in the E.U. and we have not recorded any return estimate in the transaction price for TEGSEDI sold in Europe. Other incentives: In the U.S., other incentives include co-payment assistance that we provide to patients with commercial insurance that have coverage and reside in states that allow co-payment assistance. The calculation of the accrual for co-pay assistance is based on an estimate of claims and the cost per claim that we expect to receive associated with product that has been recognized as revenue. The estimate is recorded as a reduction of revenue in the same period that the related revenue is recognized. During the three and six months ended June 30, 2019, we recorded TEGSEDI product revenue, net, of $9.9 million and $16.6 million, respectively. The following table summarizes balances and activity in each of the product revenue allowance and reserve categories for the six months ended June 30, 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 438 774 138 1,350 Adjustment related to prior period sales (4 ) (25 ) — (29 ) Credits or payments made during the period (311 ) (210 ) — (521 ) Balance at June 30, 2019 $ 173 $ 832 $ 143 $ 1,148 Leases Topic 842 Adoption In February 2016, the FASB issued amended accounting guidance related to lease accounting. This guidance supersedes the lease requirements we previously followed in ASC Topic 840, Leases , or Topic 840, and created a new lease accounting standard, ASC Topic 842, Leases , or Topic 842. We adopted Topic 842 on January 1, 2019 and adjusted our opening condensed consolidated balance sheet on that date to record our right-of-use operating lease asset and operating lease liabilities. We adopted Topic 842 using the available practical expedients permitted under the transition guidance within the new standard, which, among other things, allowed us to carry forward historical lease classification of those leases we had in place as of January 1, 2019. Results for the six months ended June 30, 2019 are presented under Topic 842. Results for the six months ended June 30, 2018 are presented in accordance with our historic accounting under Topic 840. The impact of the adoption of Topic 842 on the accompanying condensed consolidated balance sheet as of January 1, 2019 was as follows (in thousands): December 31, 2018 Adjustment due to adoption of Topic 842 January 1, 2019 Operating lease right-of-use-assets — 11,932 11,932 Other current liabilities 968 1,029 1,997 Long-term portion of lease liabilities 4,442 10,915 15,357 Leases We determine if an arrangement contains a lease at inception. We currently only have operating leases. We recognize a right-of-use operating lease asset and associated short and long-term operating lease liability for operating leases greater than one year on our condensed consolidated balance sheet. We calculate our right-of-use operating lease asset and operating lease liability based on the present value of the future minimum lease payments we will pay over the lease term. We determine the lease term at the commencement date of the lease and we include renewal options in the lease term if we are reasonably certain that we will exercise the option. As our current leases do not provide an implicit interest rate, we used our incremental borrowing rate in determining the present value of future payments. We estimate the incremental borrowing rate based on the observed interest rates for secured debt issued by companies with similar credit ratings and with similar terms. Our right-of-use operating lease asset also includes any lease payments we made and excludes any tenant improvement allowances we received. We recognize rent expense for the lease components of our operating leases on a straight-line basis over the term of our lease. We recognize non-lease components, such as common area maintenance expenses, in the period we incur the expense. New Accounting Pronouncements – Recently Issued In June 2016, the FASB issued guidance that changes the measurement of credit losses for most financial assets and certain other instruments. If we have credit losses, this updated guidance requires us to record allowances for these instruments under a new expected credit loss model. This model requires us to estimate the expected credit loss of an instrument over its lifetime, which represents the portion of the amortized cost basis we do not expect to collect. The new guidance requires us to remeasure our allowance in each reporting period we have credit losses. The new standard is effective for annual and interim periods beginning after December 15, 2019. Early adoption is permitted for periods beginning after December 15, 2018. When we adopt the new standard, we will make any adjustments to beginning balances through a cumulative-effect adjustment to accumulated deficit on that date. We plan to adopt this guidance on January 1, 2020. We are currently assessing the effects it could have 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. The updated guidance is effective for fiscal years beginning after December 15, 2019, and interim periods within those fiscal years. We plan to adopt this guidance on January 1, 2020 on a prospective basis. We are currently assessing the effects this updated guidance could have on our condensed consolidated financial statements and disclosures. In August 2018, the FASB updated its disclosure requirements related to Level 1, 2 and 3 fair value measurements. The update included deletion and modification of certain disclosure requirements and additional disclosure related to Level 3 measurements. The guidance is effective for fiscal years beginning after December 15, 2019 and early adoption is permitted. We adopted this updated guidance on January 1, 2019 and it did not have a significant impact on our disclosures. In November 2018, the FASB issued clarifying guidance on the interaction between the collaboration accounting guidance and the new revenue recognition guidance we adopted on January 1, 2018 (Topic 606). Below is the clarifying guidance and how we will implement it (in italics): 1) When a participant is considered a customer in a collaborative arrangement, all of the associated accounting under Topic 606 should be applied. ● We will apply all of the associated accounting under Topic 606 when we determine a participant in a collaborative arrangement is a customer. 2) Adds “unit of account” concept to collaboration accounting guidance to align with Topic 606. The “unit of account” concept is used to determine if revenue is recognized or if a contra expense is recognized from consideration received under a collaboration. ● We will use the “unit of account” concept when we receive consideration under a collaboration agreement to determine when we recognize revenue or a contra expense. 3) The clarifying guidance precludes us from recognizing revenue under Topic 606 when we determine a transaction with a collaborative partner is not a customer and is not directly related to the sales to third parties. ● When we conclude a collaboration partner is not a customer and is not directly related to the sales to third parties, we will not recognize revenue for the transaction. The updated guidance is effective for public entities for fiscal years beginning after December 15, 2019, and interim periods within those fiscal years. Early adoption is permitted. We plan to adopt this guidance on January 1, 2020. We are currently assessing the effects it could have on our condensed consolidated financial statements and disclosures. |
Investments and Fair Value Meas
Investments and Fair Value Measurements | 6 Months Ended |
Jun. 30, 2019 | |
Investments And Fair Value Measurements [Abstract] | |
Investments And Fair Value Measurements | 3. Investments and Fair Value Measurements Investments As of June 30, 2019 and December 31, 2018, we primarily invested our excess cash in money market funds and debt instruments of the U.S. Treasury, financial institutions, corporations and U.S. government agencies with strong credit ratings and an investment grade rating at or above A-1, P-1 or F-1 by Moody’s, S&P or Fitch, respectively. We have established guidelines relative to diversification and maturities that maintain safety and liquidity. We periodically review and modify these guidelines to maximize trends in yields and interest rates without compromising safety and liquidity. The following is a summary of our investments at June 30, 2019 and December 31, 2018 (in thousands): Gross Unrealized Estimated June 30, 2019 Cost Gains Losses Fair Value Available-for-sale securities: Corporate debt securities $ 62,426 $ 25 $ (49 ) $ 62,402 Debt securities issued by U.S. government agencies 60,476 88 — 60,564 Total securities with a maturity of one year or less $ 122,902 $ 113 $ (49 ) $ 122,966 Corporate debt securities 22,443 — (35 ) 22,408 Total securities with a maturity of one to two years 22,443 — (35 ) 22,408 Total available-for-sale securities $ 145,345 $ 113 $ (84 ) $ 145,374 Gross Unrealized Estimated December 31, 2018 Cost Gains Losses Fair Value Available-for-sale securities: Corporate debt securities $ 81,770 $ — $ (151 ) $ 81,619 Debt securities issued by U.S. government agencies 85,578 — (42 ) 85,536 Total securities with a maturity of one year or less $ 167,348 $ — $ (193 ) $ 167,155 We recorded unrealized gains (losses) related to the securities listed above as of June 30, 2019 and December 31, 2018. We believe that the decline in value of some of our securities is temporary and primarily related to the change in market interest rates since purchase. We believe it is more likely than not that we will be able to hold our debt securities to maturity. Therefore, we anticipate a full recovery of the amortized cost basis of our debt securities at maturity. All of our available-for-sale securities are available to us for use in our current operations. As a result, we categorized all of these securities as current assets even though the stated maturity of some individual securities may be one year or more beyond the balance sheet date. Fair Value Measurements The following tables present the investments we held at June 30, 2019 and December 31, 2018 that are regularly measured and carried at fair value. The table segregates each security by the level within the fair value hierarchy of valuation techniques we utilized to determine the respective security’s fair value (in thousands): At June 30, 2019 Quoted Prices in Active Markets (Level 1) Significant Other Observable Inputs (Level 2) Money market funds (1) $ 131,241 $ 131,241 $ — Corporate debt securities (3) 84,811 — 84,811 Debt securities issued by U.S. government agencies (3) 60,563 — 60,563 Total $ 276,615 $ 131,241 $ 145,374 At December 31, 2018 Quoted Prices in Active Markets (Level 1) Significant Other Observable Inputs (Level 2) Money market funds (1) $ 82,343 $ 82,343 $ — Corporate debt securities (2) 81,619 — 81,619 Debt securities issued by U.S. government agencies (3) 85,536 — 85,536 Total $ 249,498 $ 82,343 $ 167,155 (1) Included in cash and cash equivalents on our condensed consolidated balance sheets. (2) At December 31, 2018, $1.0 million 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 sheets. |
Property Plant and Equipment
Property Plant and Equipment | 6 Months Ended |
Jun. 30, 2019 | |
Property Plant And Equipment [Abstract] | |
Property, Plant and Equipment | 4. Property, Plant and Equipment The following table presents property and equipment, at cost, and related accumulated depreciation (in thousands): June 30, December 31, 2019 2018 Furniture and fixtures $ 1,611 $ 1,611 Computer equipment and software 140 102 Manufacturing equipment 357 — Leasehold improvements 3,956 4,213 Total property and equipment, at cost 6,064 5,926 Less accumulated depreciation and amortization (621 ) (230 ) Total property and equipment, net $ 5,443 $ 5,696 Total depreciation expense amounted to $0.2 million and $0.4 million for the three and six months ended June 30, 2019, respectively. Total depreciation expense was nominal for each of the three and six months ended June 30, 2018. As part of the operating lease for our new corporate headquarters, the landlord provided a tenant improvement allowance of $3.6 million, which we utilized to construct leasehold improvements. |
Inventory
Inventory | 6 Months Ended |
Jun. 30, 2019 | |
Inventory Disclosure [Abstract] | |
Inventory | 5. Inventory Prior to the regulatory approval of our product candidates, we incur expenses for the manufacturing of drug product that could potentially be available to support the commercial launch of our products. Until the first reporting period when regulatory approval has been received or is otherwise considered probable, we record all such costs as research and development expense. For TEGSEDI inventory related costs incurred subsequent to July 1, 2018, we reflected these amounts as inventory on our condensed consolidated balance sheets at the lower of cost or net realizable value under the first-in, first-out, or FIFO, basis. We periodically analyze our inventory levels and write down inventory that has become obsolete, inventory that has a cost basis in excess of its estimated realizable value and inventory in excess of expected sales requirements as cost of product sales. The determination of whether inventory costs will be realizable requires estimates by our management and if actual market conditions are less favorable than projected by our management, additional write-downs of inventory may be required which would be recorded as a cost of product sales in the condensed consolidated statements of operations. We did not record any material inventory write-offs for the six months ended June 30, 2019. Inventory consists of the following (in thousands): June 30, December 31, 2019 2018 Raw materials $ 8,034 $ — Work in process 192 — Finished goods 60 85 Total inventories $ 8,286 $ 85 |
Intangible Assets, Net
Intangible Assets, Net | 6 Months Ended |
Jun. 30, 2019 | |
Goodwill And Intangible Assets Disclosure [Abstract] | |
Intangible Assets, Net | 6 . Intangible Assets, net The following table presents intangible assets (in thousands): June 30, December 31, Estimated 2019 2018 useful life Acquired and in-licensed rights $ 2,262 $ 2,262 7 - 21 Years Capitalized regulatory approval milestones 90,000 90,000 16 Years Less accumulated amortization (6,256 ) (3,348 ) Total intangible assets, net $ 86,006 $ 88,914 We recorded $1.5 million and $2.9 million, respectively, in amortization expense related to intangible assets during the three and six months ended June 30, 2019. For each of the same periods in 2018, we recorded $0.1 million. Estimated future amortization expense for intangible assets as of June 30, 2019 is as follows (in thousands): Total 2019 $ 2,955 2020 5,879 2021 5,861 2022 5,856 2023 5,843 Thereafter 59,612 $ 86,006 The weighted average remaining amortizable life of our patents was 11.23 years at June 30, 2019. For additional detail of Akcea's license agreements with Ionis see Note 8, License Agreements and Services Agreement with Ionis |
Strategic Collaboration with No
Strategic Collaboration with Novartis | 6 Months Ended |
Jun. 30, 2019 | |
Strategic Collaboration With Novartis [Abstract] | |
Strategic Collaboration with Novartis | 7 . Background In January 2017, we initiated a strategic collaboration with Novartis for the development and commercialization of AKCEA-APO(a)-L Rx Rx AKCEA-APO(a)-L Rx In December 2018, we conducted an end-of-Phase 2 meeting with the FDA. In February 2019, Novartis exercised its exclusive option to further develop and commercialize AKCEA-APO(a)-L Rx Rx AKCEA-APOCIII-L Rx We are responsible for completing a Phase 2 program, conducting an end-of-Phase 2 meeting with the FDA and providing initial quantities of API for AKCEA-APOCIII-L Rx . Accounting Analysis We received a $75.0 million upfront payment in the first quarter of 2017, of which we retained $60.0 million and paid Ionis $15.0 million as a sublicense fee under our Cardiometabolic License Agreement with Ionis. At commencement of our strategic collaboration, we identified the following four distinct performance obligations: • Development activities for AKCEA-APO(a)-L Rx • Development activities for AKCEA-APOCIII-L Rx • API for AKCEA-APO(a)-L Rx • API for AKCEA-APOCIII-L Rx The development activities and the supply of API are distinct because Novartis or another third party could provide these items without our assistance. We determined the transaction price for the Novartis collaboration was $108.4 million, comprised of the following: • $75.0 million from the upfront payment we received; • $28.4 million for the premium paid by Novartis, which represents the excess of the fair value Ionis received from Novartis' purchase of Ionis' stock at a premium in the first quarter of 2017; and • $5.0 million for the premium Novartis would have paid to purchase Ionis' stock if we did not complete our IPO within 15 months of the inception of the agreement. We are recognizing the $75.0 million upfront payment plus the premium paid by Novartis from its purchase of Ionis’ stock and the premium associated with Novartis’ obligation to purchase Ionis’ stock if we did not complete our IPO because we are the party providing the services and API under the collaboration agreement. None of the options or development or regulatory milestone payments under this agreement were included in the upfront transaction price determined in January 2017 as all payments were fully constrained at that time. As part of our evaluation of the constraint, we considered numerous factors, including the fact that achievement of the milestones is outside of our control and contingent upon the success of our clinical trials, Novartis’ efforts, and the receipt of regulatory approval. We re-evaluate the transaction price, including estimated variable consideration included in the transaction price and all constrained amounts, in each reporting period and as uncertain events are resolved or other changes in circumstances occur. Based on the distinct performance obligations under the Novartis collaboration, we allocated the $108.4 million transaction price based on relative stand-alone selling prices of each of our performance obligations as follows: • $64.0 million for development services for AKCEA-APO(a)-L Rx • $40.1 million for development services for AKCEA-APOCIII-L Rx • $1.5 million for the delivery of AKCEA-APO(a)-L Rx • $2.8 million for the delivery of AKCEA-APOCIII-L Rx We are recognizing revenue related to each of our performance obligations as follows: • We have completed the development services performance obligation for AKCEA-APO(a)-L Rx Rx • We are satisfying the development services performance obligation for AKCEA-APOCIII-L Rx • We recognized the amount attributed to the AKCEA-APO(a)-L Rx • We recognized the amount attributed to the AKCEA-APOCIII-L Rx Additionally, we and Ionis entered into a stock purchase agreement, or SPA, with Novartis. Under the SPA, in July 2017, Novartis purchased $50.0 million of our common stock in a separate private placement concurrent with the completion of our IPO at a price per share equal to the IPO price. On February 22, 2019, Novartis exercised its option to license AKCEA-APO(a)-L Rx Rx in the first quarter of 2019 because Novartis had full use of the license without any continuing involvement from us. Additionally, we did not have any further performance obligations related to the license after Novartis exercised its option to license AKCEA-APO(a)-L Rx Rx Rx w Rx Rx Rx Rx . If Novartis exercises its option for AKCEA-APOCIII-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 either drug. We are also eligible to receive tiered royalties in the mid-teens to low twenty percent range on net sales of AKCEA-APO(a)-L Rx Rx During the three and six months ended June 30, 2019, we earned revenue of $10.7 million and $167.8 million, respectively, from our strategic collaboration with Novartis, representing 100% of our research and development and license revenue. In comparison, we earned revenue of $18.3 million and $35.4 million for the same periods in 2018. During the three and six months ended June 30, 2019, we recognized $10.2 million and $15.7 million, respectively, of revenue from amounts that were in our beginning deferred revenue balance. In comparison, we recognized $12.6 million and $32.9 million for the same periods in 2018. |
License Agreements and Services
License Agreements and Services Agreement with Ionis | 6 Months Ended |
Jun. 30, 2019 | |
Related Party Transactions [Abstract] | |
License Agreements and Services Agreement with Ionis | 8 . In December 2015, we entered into a development, commercialization and license agreement related to our cardiometabolic franchise and a services agreement with Ionis. In March 2018, we entered into a new development, commercialization, collaboration and license agreement related to our TTR franchise and amended the services agreement previously in place with Ionis. The following sections summarize these related party agreements with Ionis. Cardiometabolic Development, Commercialization and License Agreement Our development, commercialization and license agreement, or Cardiometabolic License Agreement, with Ionis granted exclusive rights to us to develop and commercialize WAYLIVRA, AKCEA-APO(a)-L Rx Rx Rx We and Ionis share development responsibilities for the Lipid Drugs, other than drugs licensed to Novartis. We pay Ionis for the research and development expenses it incurs on our behalf, which include both external and internal expenses. External research and development expenses include costs for contract research organizations, or CROs, costs to conduct nonclinical and clinical studies on our drugs, costs to acquire and evaluate clinical study data, such as investigator grants, patient screening fees and laboratory work, and fees paid to consultants. Internal research and development expenses include costs for the work that Ionis' research and development employees perform for us. Ionis charges us a full-time equivalent rate that covers personnel-related expenses, including salaries and benefits, plus an allocation of facility-related expenses, including rent, utilities, insurance and property taxes, for those development employees who work either directly or indirectly on the development of our drugs. We also pay Ionis for the API and drug product we use in our nonclinical and clinical studies for all of our drugs. Ionis manufactures the API for us and charges us a price per gram consistent with the price Ionis charges its pharmaceutical partners, which includes the cost for direct materials, direct labor and overhead required to manufacture the API. If we need the API filled in vials for our clinical studies and Ionis contracts with a third party to perform this work, Ionis will charge us for the resulting cost. As we commercialize each of the Lipid Drugs, other than drugs licensed to Novartis, we will pay Ionis royalties from the mid-teens to the mid-twenty percent range on sales related to the Lipid Drugs that we sell. If we sell a Lipid Drug for a Rare Disease Indication (defined in the agreement as less than 500,000 patients worldwide or an indication that required a Phase 3 program of less than 1,000 patients and less than two years of treatment), we will pay a higher royalty rate to Ionis than if we sell a Lipid Drug for a Broad Disease Patient Population (defined in the agreement as more than 500,000 patients worldwide or an indication that required a Phase 3 program of 1,000 or more patients and two or more years of treatment). Other than with respect to the drugs licensed to Novartis under the collaboration agreement, if our annual sales reach $500.0 million, $1.0 billion and $2.0 billion, we will be obligated to pay Ionis sales milestones in the amount of $50.0 million for each sales milestone reached by each Lipid Drug. If and when triggered, we will pay Ionis each of these sales milestones over the subsequent 12 quarters in equal payments. We share 50% of payments we receive from Novartis with Ionis. We may terminate this agreement if Ionis is in material breach of the agreement. Ionis may terminate this agreement if we are in material breach of the agreement. In each circumstance the party that is in breach will have an opportunity to cure the breach prior to the other party terminating this agreement. In the first quarter of 2017, we entered into letter agreements with Ionis to reflect the agreed upon payment terms with respect to the upfront option payment that we received from Novartis and to allocate the premium that Novartis paid for Ionis' common stock in connection with our strategic collaboration with Novartis. For additional detail regarding our strategic collaboration with Novartis, see Note 7, Strategic Collaboration with Novartis. TTR Development, Commercialization, Collaboration and License Agreement On April 17, 2018, our stockholders, other than Ionis and its affiliates, approved the development, commercialization, collaboration and license agreement, or TTR License Agreement, and a stock purchase agreement, or Ionis SPA, with Ionis, which was entered into on March 14, 2018. In addition, in connection with these agreements, we entered into an amended and restated services agreement, or Amended Services Agreement, and an amended and restated investor rights agreement, or Amended Investor Rights Agreement, with Ionis. We determined that the TTR License Agreement and Ionis SPA included provisions which required the approval of the agreements by our stockholders other than Ionis and its affiliates, which we deemed was not perfunctory in nature, therefore, we concluded that the approved date of the agreements for accounting purposes was April 17, 2018, the date on which such approval was received and the closing of the agreements took place. In accordance with the terms and provisions of the TTR License Agreement, we received rights to: • commercialize TEGSEDI following receipt of regulatory approval and perform certain other non-commercial activities with respect to TEGSEDI, in each case, in accordance with a global strategic plan; • partner on the completion of all pivotal studies of a follow-on drug to TEGSEDI, AKCEA-TTR-L Rx Rx • commercialize AKCEA-TTR-L Rx • share in profits and losses with respect to TEGSEDI and AKCEA-TTR-L Rx • manufacture (including through a third party) each product following receipt of regulatory approval for such product; and • sublicense the development and commercialization of either product to third parties or affiliates, with the consent of Ionis. As consideration for the grant of rights under the TTR License Agreement, we paid an upfront licensing fee of $150.0 Rx The TTR License Agreement will remain in effect until the expiration of all included payment obligations, unless earlier terminated. The TTR License Agreement can be terminated by mutual consent of us and Ionis, by either us or Ionis upon certain events, by either party upon material breach, or by Akcea for convenience upon providing 90 days written notice to Ionis. Upon termination, all rights received under the TTR License Agreement will terminate. To support the commercialization of TEGSEDI and AKCEA-TTR-L Rx In connection with the licensing transaction, we amended our Certificate of Incorporation to increase our authorized shares of common stock from 100,000,000 shares to 125,000,000 shares. We determined that the upfront accounting for the TTR License Agreement should follow the accounting guidance for common control transactions given the nature of the relationship between us and Ionis, including the fact that Ionis maintains a controlling ownership position in us. In addition, we assessed the identifiable assets that were acquired under the terms of the TTR License Agreement, including the licensed rights to TEGSEDI and AKCEA-TTR-L Rx In connection with the transaction, we also purchased $4.7 million of commercial TEGSEDI inventory held by Ionis. In addition, during the six months ended June 30, 2019 we purchased $13.5 million of clinical TEGSEDI material held by Ionis. Prospectively we are responsible for the procurement of all additional inventory. The inventory and clinical material did not have a carrying value on the books of Ionis at the time of purchase. As such, in accordance with the accounting guidance for common control transactions above, we recorded the purchase of this inventory and clinical material as a reduction of additional paid in capital. This amount represented a cash distribution to Ionis; therefore, we have included this distribution as a distribution to Ionis for purposes of loss per share and we have applied the two–class method as discussed in Note 11, Basic and Diluted Net Loss Per Share . We also determined that the TTR License Agreement represented a collaboration arrangement as defined by ASC 808. Prior to April 1, 2018, Ionis was responsible for all costs associated with TEGSEDI and for the period from April 1, 2018 to December 31, 2018, we were responsible for all costs associated with TEGSEDI. We and Ionis share all costs associated with AKCEA-TTR-L Rx . We recorded $3.1 million paid to Ionis for costs related to the period prior to the closing of the TTR License Agreement to equity, as these amounts were previously expensed in the financial statements of Ionis. This amount also represents a cash distribution to Ionis and was included as an adjustment to the net loss attributable to Ionis for purposes of applying the two-class method for loss per share as discussed in Note 11, Basic and Diluted Net Loss Per Share . In addition, on July 11, 2018, we received marketing authorization, or MA, approval for TEGSEDI from the European Commission, or EC, for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis, or hATTR amyloidosis, in the E.U. As a result of the MA 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. As a result of the marketing authorization in the E.U., we capitalized this regulatory approval milestone payment as a licensed intangible asset on our condensed consolidated balance sheets as the amount is expected to be recoverable through future cash flows. In addition, on October 5, 2018, we received regulatory approval for TEGSEDI from the FDA for the treatment of polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults in the 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. As a result of the FDA approval in the U.S., we capitalized this regulatory approval milestone payment as a licensed intangible asset on our condensed consolidated balance sheets 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 milestone payments was $1.4 million and $2.8 million, respectively, for the three and six months ended June 30, 2019. We did not record any amortization expense for the TTR milestone payments for the three and six months ended June 30, 2018. 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 revenues, cost of sales and sales and marketing expenses. The Ionis share of commercialization costs for TEGSEDI is separately presented within operating expenses in our 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 Agreement is as follows: Three Months Ended June 30, Six Months Ended June 30, 2019 2019 Net losses incurred by the collaboration related to the commercial activities under the TTR Agreement $ (19,109 ) $ (34,207 ) Ionis' share of commercial losses under the TTR Agreement reflected in our condensed consolidated statements of operations (11,465 ) (20,521 ) Akcea's share of commercial losses under the TTR Agreement reflected in our condensed consolidated statements of operations (7,644 ) (13,686 ) A summary of the development expenses related to the TTR Agreement is as follows: Three Months Ended June 30, Six Months Ended June 30, 2019 2019 Total development expense incurred by the collaboration related to development activities under the TTR Agreement $ (12,032 ) $ (26,412 ) Akcea's share of TTR development expense reflected in research and development expense in our condensed consolidated statements of operations $ (5,265 ) $ (11,398 ) We did not record any commercial or research and development expense related to the profit/(loss) share under the TTR License Agreement during the three and six months ended June 30, 2018. Services Agreement We originally entered into a services agreement with Ionis in December 2015 in conjunction with the Cardiometabolic License Agreement. We entered into the Amended Services Agreement with Ionis in April 2018 in conjunction with the TTR License Agreement (collectively, the service agreements). The primary purpose of the Amended Services Agreement was to allow for the expansion of general and administrative services provided to us by Ionis to cover the TEGSEDI and AKCEA-TTR-L Rx Our services agreement with Ionis is designed to be flexible to adjust for our increasing capabilities in various functions. Under the services agreement, Ionis provides us certain services, including, without limitation, general and administrative support services and development support services. Ionis allocated a certain percentage of personnel to perform the services that it provides to us based on its good faith estimate of the required services. We pay Ionis for these allocated costs, which reflect the Ionis full-time equivalent, or FTE, rate for the applicable personnel, plus out-of-pocket expenses, such as occupancy costs, associated with the FTEs allocated to providing us these services. We do not pay a mark-up or profit on the external or internal expenses Ionis bills to us. Ionis invoices us quarterly for all amounts due under the services agreement and payments are due within 30 days of the receipt of an invoice. In addition, as long as Ionis continues to consolidate our financials, we will comply with Ionis' policies and procedures and internal controls. As long as we are consolidated into Ionis' financial statements under U.S. GAAP, we may continue to access the following services from Ionis: • investor relations services, • human resources and personnel services, • risk management and insurance services, • tax related services, • corporate record keeping services, • financial and accounting services, • credit services, and • COO/CFO/CBO oversight. However, if we wanted to provide the foregoing services internally, and doing so would not negatively impact Ionis' internal controls and procedures for financial reporting, we can negotiate in good faith with Ionis for a reduced scope of services related to the aforementioned services. When Ionis determines it should no longer consolidate our financials, we may mutually agree with Ionis in writing to extend the term of this arrangement in six-month increments. We can establish our own benefits programs or continue to use Ionis' benefits, however we must provide Ionis a minimum advance notice to opt-out of using Ionis' benefits. We do not currently plan to establish our own benefits programs at this time or in the near future. Pursuant to our various agreements with Ionis, as of June 30, 2019, Ionis owed us $7.9 million. As of December 31, 2018, we owed Ionis $18.9 million. The following table summarizes the amounts recorded related to transactions with Ionis including amounts related to the TTR licensing transaction for the following periods (in thousands): Three Months Ended June 30, Six Months Ended June 30, 2019 2018 2019 2018 Operating expenses: Services performed by Ionis $ 1,075 $ 6,045 $ 2,150 $ 7,990 Active pharmaceutical ingredient manufactured by Ionis — — — 5,229 Pre-commercial inventory manufactured by Ionis — 5,996 — 5,996 Sublicensing expenses 3,000 — 78,000 — Out-of-pocket expenses paid by Ionis 1,275 16,029 2,846 15,224 Less: commercial share of loss in connection with the TTR license transaction (11,465 ) — (20,521 ) — Less: R&D share of loss in connection with the TTR license transaction 1,204 — 408 — Total operating expenses generated by transactions with Ionis (4,911 ) 28,070 62,883 34,439 Plus: distribution to Ionis — — 13,492 7,792 Total charges generated by transactions with Ionis (4,911 ) 28,070 76,375 42,231 (Receivable) payable balance (from) to Ionis at the beginning of the period (7,206 ) 27,737 18,901 14,365 Less: total amounts received from (paid to) Ionis during the period 4,206 (27,737 ) (28,187 ) (28,526 ) Less: receivable from Ionis — (933 ) — (933 ) Less: non-cash sublicensing expenses — — (75,000 ) — Total amount (receivable) payable (from) to Ionis at period end $ (7,911 ) $ 27,137 $ (7,911 ) $ 27,137 |
Collaboration and License Agree
Collaboration and License Agreement with PTC Therapeutics | 6 Months Ended |
Jun. 30, 2019 | |
Collaboration And License Agreement [Abstract] | |
Collaboration and License Agreement with PTC Therapeutics | 9 . Collaboration and License Agreement with PTC Therapeutics In August 2018, we entered into a collaboration and license agreement with PTC Therapeutics, or the PTC License Agreement, to commercialize TEGSEDI and WAYLIVRA in Latin America and certain Caribbean countries, or the PTC Territory. We received a $12.0 million upfront payment from PTC Therapeutics related to TEGSEDI in the third quarter of 2018 upon execution of the PTC License Agreement, of which we paid Ionis $7.2 million as a sublicense fee related to the TTR License Agreement. We received a $6.0 million payment from PTC Therapeutics in the second quarter of 2019 as a result of WAYLIVRA approval by the EC, of which we paid Ionis $3.0 million as a sublicense fee related to the cardiometabolic license agreement and was recorded as product cost of sales in the condensed consolidated statement of operations. In addition, we are eligible to receive up to $8.0 million for the achievement of regulatory milestones and royalties in the mid-twenty percent range on net sales of TEGSEDI and WAYLIVRA in the PTC Territory. PTC Therapeutics’ obligation to pay royalties to us begins on the earlier of 12 months after the first commercial sale of a product in Brazil or the date that PTC Therapeutics recognized revenue of at least $10.0 million in the PTC Territory. PTC Therapeutics will reduce these royalties upon the expiration of certain patents or if a generic competitor negatively impacts the market share of the product in the PTC Territory. Milestone payments and royalties that we are eligible to receive from PTC Therapeutics for TEGSEDI will be split 60% to Ionis and 40% to Akcea. All WAYLIVRA milestone payments and royalties that we are eligible to receive from PTC will be split 50/50 with Ionis. PTC Therapeutics is solely responsible for the commercialization of the products in the PTC Territory at its sole cost and expense, including the pursuit and maintenance of applicable regulatory approvals. Unless earlier terminated, the PTC License Agreement will continue in effect until the date on which the royalty term and all payment obligations with respect to all products in all countries in the PTC Territory have expired. At the commencement of the PTC License Agreement, we identified two performance obligations, consisting of the transfer of (1) the license to TEGSEDI and related know-how and (2) the license to WAYLIVRA and related know-how, both of which were satisfied during the third quarter of 2018. In addition, we identified a customer option related to PTC Therapeutics’ option to purchase supply of product from us for its development and commercial needs. We considered the manufacturing capabilities of PTC Therapeutics and the fact that manufacturing services are not proprietary and can be provided by other vendors to conclude that the licenses have stand-alone functionality and are distinct. Further, the customer options for manufacturing of product is priced similar to other manufacturing options with similar customers and is therefore not considered a material right. As there were no remaining unsatisfied performance obligations as of September 30, 2018, the $12.0 million upfront payment was recognized as licensing revenue upon contract execution in the third quarter of 2018. The option to purchase supply from us is subject to the terms of a supply agreement we entered into with PTC Therapeutics in April 2019. None of the regulatory milestones were included in the upfront transaction price determined in August 2018, as all payments were fully constrained. In May 2019, we received $6.0 million of consideration from PTC Therapeutics as a result of WAYLIVRA approval by the EC and we paid $3.0 million as a sublicense fee. Since the constraint on the regulatory approval was resolved, we updated the transaction price to include this consideration, and accordingly, we recognized the $6.0 million as licensing revenue during the second quarter of 2019. We will continue to re-evaluate the transaction price, including estimated variable consideration included in the transaction price and all constrained amounts, in each reporting period and as uncertain events are resolved or other changes in circumstances occur. Any consideration related to sales-based royalties will be recognized when the related sales occur |
Stock-Based Compensation
Stock-Based Compensation | 6 Months Ended |
Jun. 30, 2019 | |
Disclosure Of Compensation Related Costs Sharebased Payments [Abstract] | |
Stock-Based Compensation | 10 . Stock Plans 2015 Equity Incentive Plan As of June 30, 2019, 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. At June 30, 2019, a total of 13,068,974 options were outstanding, of which 5,382,040 were exercisable, 32,669 restricted stock unit awards were outstanding, and 3,824,563 shares were available for future grant under the 2015 Plan. 2017 Employee Stock Purchase Plan On January 1, 2019, 500,000 shares of common stock were added to the 2017 Employee Stock Purchase, 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 increased on January 1 st Stock-based Compensation Expense We measure stock-based compensation expense for equity-classified awards, principally related to stock options, restricted stock units, or RSUs, and stock purchase rights under our 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 statements 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 six months ended June 30, 2019 and 2018, we used the following weighted-average assumptions in our Black-Scholes calculations: Employee Stock Options: Six Months Ended June 30, 2019 2018 Risk-free interest rate 2.5 % 2.8 % Dividend yield 0.0 % 0.0 % Volatility 76.3 % 77.7 % Expected life 6.1 years 6.1 years Board of Directors Stock Options: Six Months Ended June 30, 2019 2018 Risk-free interest rate 1.9 % 2.9 % Dividend yield 0.0 % 0.0 % Volatility 74.3 % 78.2 % Expected life 6.3 years 6.4 years 2017 ESPP: Six Months Ended June 30, 2019 2018 Risk-free interest rate 2.5 % 1.6 % Dividend yield 0.0 % 0.0 % Volatility 64.1 % 62.3 % Expected life 6 months 6 months The following table summarizes stock-based compensation expense for the three and six months ended June 30, 2019 and 2018 (in thousands): Three Months Ended June 30, Six Months Ended June 30, 2019 2018 2019 2018 Cost of sales - product $ 137 $ — $ 255 $ — Research and development expenses 2,563 2,242 6,484 4,555 Selling, general and administrative expenses 11,663 9,884 26,184 13,954 Total $ 14,363 $ 12,126 $ 32,923 $ 18,509 As of June 30, 2019, total unrecognized, estimated non-cash stock-based compensation expense related to non-vested stock options and RSUs was $82.1 million and $0.9 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 1.46 years and 1.45 years, respectively. |
Basic and Diluted Net Loss Per
Basic and Diluted Net Loss Per Share | 6 Months Ended |
Jun. 30, 2019 | |
Earnings Per Share [Abstract] | |
Basic and Diluted Net Loss Per Share | 1 1 . Basic and Diluted Net Loss Per Share In connection with the TTR License Agreement completed on April 17, 2018 with Ionis, we made distributions to Ionis representing the consideration to be paid in cash provided to Ionis in excess of the carrying value of the related assets acquired. These distributions are treated as dividends to Ionis; therefore, we have applied the two-class method of loss per share to reflect the allocation of these distributions to the participating Ionis common shares. The two-class method is an earnings allocation formula that determines loss per share for each class of common stock and participating security according to dividends declared (or accumulated) and participation rights in undistributed earnings. For the purposes of calculating loss per share under the two-class method, we have allocated the net loss between common stock owned by Ionis and common stock owned by others. Basic loss per share for each class of stock is computed by dividing total distributable losses applicable to common stock owned by Ionis and common stock owned by others by the weighted-average of common shares outstanding during the requisite period. The following table summarizes the distributable losses for the three and six months ended June 30, 2019 and 2018 (in thousands): Three Months Ended June 30, Six Months Ended June 30, 2019 2018 2019 2018 Net loss $ (37,323 ) $ (62,046 ) $ (10,136 ) $ (91,673 ) Distributions to Ionis — (7,792 ) (13,492 ) (7,792 ) Distributable losses $ (37,323 ) $ (69,838 ) $ (23,628 ) $ (99,465 ) The following table summarizes the reconciliation of weighted-average shares outstanding used in the calculation of basic loss per share for the three and six months ended June 30, 2019 and 2018: Three Months Ended June 30, Six Months Ended June 30, Determination of shares: 2019 2018 2019 2018 Weighted-average common shares outstanding owned by Ionis 70,221,338 60,832,494 69,406,181 53,182,685 Weighted-average common shares outstanding owned by others 22,573,900 21,492,157 22,351,368 21,332,650 Total weighted-average shares outstanding 92,795,238 82,324,651 91,757,549 74,515,335 The following table summarizes the calculation of basic loss per share for the three and six months ended June 30, 2019 and 2018 (in thousands, except per share and share amounts): Three Months Ended June 30, Six Months Ended June 30, 2019 2018 2019 2018 Losses allocated to Ionis $ (28,244 ) $ (51,606 ) $ (17,872 ) $ (70,990 ) Plus: Distribution to Ionis — 7,792 13,492 7,792 Losses available to Ionis $ (28,244 ) $ (43,814 ) $ (4,380 ) $ (63,198 ) Weighted-average common shares outstanding owned by Ionis 70,221,338 60,832,494 69,406,181 53,182,685 Basic loss per common share owned by Ionis $ (0.40 ) $ (0.72 ) $ (0.06 ) $ (1.19 ) Losses allocated to common shares owned by others $ (9,079 ) $ (18,232 ) $ (5,756 ) $ (28,475 ) Weighted-average common shares outstanding owned by others 22,573,900 21,492,157 22,351,368 21,332,650 Basic loss per common share owned by others $ (0.40 ) $ (0.85 ) $ (0.26 ) $ (1.33 ) For the three and six months ended June 30, 2019 and June 30, 2018, we incurred a net loss; therefore, we did not include dilutive common equivalent shares in the computation of diluted net loss per share because the effect would have been anti-dilutive. Common stock from the following would have had an anti-dilutive effect on net loss per share: • Options to purchase common stock; • Unvested restricted stock units; and • Employee Stock Purchase Plan. |
Contractual Obligations and Com
Contractual Obligations and Commitments | 6 Months Ended |
Jun. 30, 2019 | |
Commitments And Contingencies Disclosure [Abstract] | |
Contractual Obligations and Commitments | 1 2 . Operating Lease Corporate Headquarters 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 ending after 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 did not consider it reasonably certain that we would exercise the option. Under the lease agreement, we received a three-month free rent period, which commenced on August 15, 2018, and a tenant improvement allowance up to $3.8 million. We provided the lessor with a letter of credit to secure its obligations under the lease in the initial amount of $2.4 million, to be reduced to $1.8 million on the third anniversary of the rent commencement date and to $1.2 million on the fifth anniversary of the rent commencement date if we meet certain conditions set forth in the lease at each such time. The letter of credit amount is included in deposits and other assets on the accompanying condensed consolidated balance sheets. Ionis Sublease On November 12, 2018, we entered into an operating lease agreement with Ionis Pharmaceuticals to sublease 4,723 square feet of office space located in Carlsbad, California. The commencement date was March 2018 and the term of the lease is 64 months with a four-month free rent period. There is no extension option with this lease. Cambridge Lease We leased office space in a building in Cambridge, Massachusetts under an operating lease that commenced in April 2015 and was subsequently amended and expanded in February 2016 and March 2017. The lease was scheduled to expire in April 2020. We have subsequently terminated the lease effective April 2019. Ireland 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 for an additional 12 months. We have included the 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. Other Operating Lease Information Other information related to our operating leases is as follows (dollar amounts in thousands): At June 30, 2019 Operating lease right-of-use assets $ 11,534 Operating lease liabilities (1) $ 16,178 Weighted average remaining lease term 9.2 years Weighted average discount rate 8 % (1) Current portion of $1.3 million included in other current liabilities and the difference of $14.9 million included in long-term portion of lease liabilities on our condensed consolidated balance sheet. Annual maturities of our operating lease liabilities as of June 30, 2019 are as follows (in thousands): Years ended December 31, Operating Leases Remainder of 2019 $ 1,256 2020 2,504 2021 2,507 2022 2,403 2023 2,400 Thereafter 11,961 Total minimum lease payments $ 23,031 Less: Imputed interest (6,853 ) Total operating lease liability $ 16,178 Operating lease expense was $0.5 million and $1.1 million for the three and six months ended June 30, 2019, respectively. In comparison, operating lease expense was $0.7 million and $0.9 million for the same periods in 2018. Cash paid for amounts included in the measurement of lease liabilities for the six months ended June 30, 2019 was $1.3 million and was included in net cash used in operating activities in our condensed consolidated statements of cash flows. 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 June 30, 2019 our purchase commitments for the following 12 months were $6.6 million. |
Summary of Significant Accoun_2
Summary of Significant Accounting Policies (Policies) | 6 Months Ended |
Jun. 30, 2019 | |
Accounting Policies [Abstract] | |
Use of Estimates | Use of Estimates The preparation of financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities, the disclosure of contingent assets and liabilities at the date of the financial statements and the reported amounts of revenue and expenses during the reporting periods. Significant estimates and assumptions reflected in these financial statements include, but are not limited to, revenue recognition and the accrual for research and development expenses. Estimates are periodically reviewed in light of changes in facts, circumstances and experience. Changes in estimates are recorded in the period in which they become known. Actual results could differ from those estimates. |
Translation of Foreign Currency | Translation of Foreign Currency For our foreign subsidiaries that report in a functional currency other than U.S. dollars, we translate their assets and liabilities into U.S. dollars using the exchange rate at the balance sheet date. We translate revenue and expenses at the monthly average exchange rates for the period. We translate transactions in our capital accounts at the historic exchange rate in effect at the date of the transaction. We include foreign currency translation adjustments as a component of accumulated other comprehensive loss within the condensed consolidated statements of comprehensive loss. |
Revenue Recognition | Revenue Recognition Collaboration and License Revenue In May 2014, the Financial Accounting Standards Board, or FASB, issued Accounting Standards Update, or ASU, No. 2014-09, Revenue from Contracts with Customers To determine revenue recognition for arrangements that an entity determines are within the scope of Topic 606, the entity performs the following five steps: (i) identify the contract(s) with a customer; (ii) identify the performance obligations in the contract; (iii) determine the transaction price; (iv) allocate the transaction price to the performance obligations in the contract; and (v) recognize revenue when (or as) the entity satisfies a performance obligation. At contract inception, once the contract is determined to be within the scope of Topic 606, we assess the goods or services promised within each contract and determine those that are performance obligations, then assess whether each promised good or service is distinct. When we offer options for additional goods or services, such as an option to license a drug in the future or for additional goods or services to be provided in the future, we evaluate whether such options are material rights that should be treated as additional performance obligations. We typically have concluded that the option to license a drug or the options for additional goods or services that may be requested in the future under our collaboration agreements are not material rights as the amounts attributable to such options represent standalone selling price, and therefore no consideration is allocated to these items at the inception of an agreement. When a partner exercises its option to license a drug or requests the additional goods or services, a new performance obligation is created for that item. Once performance obligations are identified, we then recognize as revenue the amount of the transaction price that we allocated to the respective performance obligation when (or as) each performance obligation is satisfied, either at a point in time or over time. If the performance obligation is satisfied over time, we recognize revenue based on the use of an output or input method. As of June 30, 2019, we have three revenue agreements: our strategic collaboration, option and license agreement, or collaboration agreement, with Novartis Pharma AG, or Novartis, which we entered into in January 2017, our collaboration and license agreement with PTC Therapeutics International Limited, or PTC Therapeutics, which we entered into in August 2018, and our TTR development, commercialization, collaboration and license agreement with Ionis, under which we are recognizing commercial product revenue related to TEGSEDI sales subsequent to product launch in the fourth quarter of 2018. For a complete discussion of the accounting related to our collaborative agreements, see Note 7, Strategic Collaboration with Novartis, Collaboration and License Agreement with PTC Therapeutics |
Product Revenue Net | Product Revenue, Net Subsequent to regulatory approval in Europe on July 11, 2018 and FDA approval in the U.S. on October 5, 2018, in the fourth quarter of 2018, we began to sell TEGSEDI in the U.S. and Europe. In the U.S., the product is distributed through an exclusive distribution agreement with a third-party logistics, or 3PL, company that takes title to the product and represents our sole customer in the U.S. Our U.S. customer distributes TEGSEDI to a specialty pharmacy and a specialty distributor (collectively referred to as “wholesalers”), who then distribute the product to health care providers and patients. In Europe, the product is currently distributed through a non-exclusive distribution model with a 3PL that takes title to the product and currently is our sole customer in Europe. Our customer then distributes TEGSEDI to hospitals and pharmacies in Europe. Revenue from product sales is recognized when the customer obtains control of our product, which occurs upon transfer of title to the customer. We record shipping and handling costs within cost of goods sold on our condensed consolidated statement of operations. We classify payments to customers or other parties in the distribution channel for services that are distinct and priced at fair value as selling, general and administrative expenses in our condensed consolidated statements of operations. Payments to customers or other parties in the distribution channel that do not meet those criteria are classified as a reduction of revenue, as discussed further below. Taxes collected from customers relating to product sales and remitted to governmental authorities are excluded from revenue. We have elected not to adjust consideration for the effects of a significant financing component when the period between the transfer of a promised good or service to the customer and when the customer pays for that good or service will be one year or less. Our payment terms are generally between thirty to ninety days. Reserves for Variable Consideration Revenue from product sales is recorded at the net sales price (transaction price), which includes estimates of variable consideration for which reserves are established and which result from discounts, returns, chargebacks, rebates, co-pay assistance and other allowances that are offered within contracts between us and our customers, wholesalers, health care providers and other indirect customers relating to the sale of TEGSEDI. These reserves are based on the amounts earned or to be claimed on the related sales and are classified as reductions of accounts receivable (if no payments are required of us) or a current liability (if a payment is required by us). Where appropriate, these estimates take into consideration a range of possible outcomes that are probability-weighted for relevant factors such as our historical experience, current contractual and statutory requirements, specific known market events and trends, industry data and forecasted customer buying and payment patterns. Overall, product revenue net of these reserves reflects our best estimates of the amount of consideration to which we are entitled based on the terms of the contract. The amount of variable consideration that is included in the transaction price may be constrained and is included in the net sales price only to the extent that it is considered probable that a significant reversal in the amount of the cumulative revenue recognized will not occur in a future period. Actual amounts of consideration ultimately received may differ from our estimates. If actual results in the future vary from our estimates, we will adjust these estimates, which would affect net product revenue and earnings in the period such variances become known. The following are the components of variable consideration related to product revenue: Chargebacks: In the U.S., we estimate obligations resulting from contractual commitments with the government and other entities to sell products to qualified healthcare providers at prices lower than the list prices charged to our U.S. customer. Our U.S. customer charges us for the difference between what they pay for the product and the selling price to the qualified healthcare providers. We record reserves for these chargebacks related to product sold to our U.S. customer during the reporting period, as well as our estimate of product that remains in the distribution channel at the end of the reporting period that we expect will be sold to qualified healthcare providers in future periods. Government rebates: We are subject to discount obligations under government programs, including Medicaid and Medicare programs in the U.S., and we record reserves for government rebates based on statutory discount rates and estimated utilization in the period in which revenue is recognized. We estimate Medicaid and Medicare rebates based upon estimated payer mix. These reserves are recorded in the same period the related revenue is recognized, resulting in a reduction of product revenue and the establishment of a liability that is included in accrued expenses on our condensed consolidated balance sheet. For Medicare, we also estimate the number of patients in the prescription drug coverage gap for whom we expect we will owe an additional liability under the Medicare Part D program. On a quarterly basis, we update our estimates and record any adjustments in the period that we identify the adjustments. Trade discounts and allowances: We provide customary invoice discounts on TEGSEDI sales to our U.S. customer for prompt payment that are recorded as a reduction of revenue in the period the related product revenue is recognized. Distribution fees: We receive and pay for various distribution services provided by our U.S. and E.U. customers and wholesalers in the U.S. distribution channel and these fees are generally accounted for as a reduction of revenue. To the extent that the services received are distinct from the sale of products to our customers, these payments are accounted for as selling, general and administrative expenses. Product Returns: Our U.S. customer has return rights and the wholesalers have limited return rights primarily related to the product’s expiration date. We estimate the amount of product sales that may be returned and record the estimate as a reduction of revenue and a refund liability in the period the related product revenue is recognized. Based on the distribution model for TEGSEDI, contractual inventory limits with our U.S. customer and wholesalers and the price of TEGSEDI, we believe there will be minimal returns in the U.S. Our E.U. customer only takes title to the product once it receives an order from a hospital or pharmacy and therefore does not maintain any inventory of TEGSEDI. Accordingly, there is limited return risk in the E.U. and we have not recorded any return estimate in the transaction price for TEGSEDI sold in Europe. Other incentives: In the U.S., other incentives include co-payment assistance that we provide to patients with commercial insurance that have coverage and reside in states that allow co-payment assistance. The calculation of the accrual for co-pay assistance is based on an estimate of claims and the cost per claim that we expect to receive associated with product that has been recognized as revenue. The estimate is recorded as a reduction of revenue in the same period that the related revenue is recognized. During the three and six months ended June 30, 2019, we recorded TEGSEDI product revenue, net, of $9.9 million and $16.6 million, respectively. The following table summarizes balances and activity in each of the product revenue allowance and reserve categories for the six months ended June 30, 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 438 774 138 1,350 Adjustment related to prior period sales (4 ) (25 ) — (29 ) Credits or payments made during the period (311 ) (210 ) — (521 ) Balance at June 30, 2019 $ 173 $ 832 $ 143 $ 1,148 |
Leases | Leases Topic 842 Adoption In February 2016, the FASB issued amended accounting guidance related to lease accounting. This guidance supersedes the lease requirements we previously followed in ASC Topic 840, Leases , or Topic 840, and created a new lease accounting standard, ASC Topic 842, Leases , or Topic 842. We adopted Topic 842 on January 1, 2019 and adjusted our opening condensed consolidated balance sheet on that date to record our right-of-use operating lease asset and operating lease liabilities. We adopted Topic 842 using the available practical expedients permitted under the transition guidance within the new standard, which, among other things, allowed us to carry forward historical lease classification of those leases we had in place as of January 1, 2019. Results for the six months ended June 30, 2019 are presented under Topic 842. Results for the six months ended June 30, 2018 are presented in accordance with our historic accounting under Topic 840. The impact of the adoption of Topic 842 on the accompanying condensed consolidated balance sheet as of January 1, 2019 was as follows (in thousands): December 31, 2018 Adjustment due to adoption of Topic 842 January 1, 2019 Operating lease right-of-use-assets — 11,932 11,932 Other current liabilities 968 1,029 1,997 Long-term portion of lease liabilities 4,442 10,915 15,357 Leases We determine if an arrangement contains a lease at inception. We currently only have operating leases. We recognize a right-of-use operating lease asset and associated short and long-term operating lease liability for operating leases greater than one year on our condensed consolidated balance sheet. We calculate our right-of-use operating lease asset and operating lease liability based on the present value of the future minimum lease payments we will pay over the lease term. We determine the lease term at the commencement date of the lease and we include renewal options in the lease term if we are reasonably certain that we will exercise the option. As our current leases do not provide an implicit interest rate, we used our incremental borrowing rate in determining the present value of future payments. We estimate the incremental borrowing rate based on the observed interest rates for secured debt issued by companies with similar credit ratings and with similar terms. Our right-of-use operating lease asset also includes any lease payments we made and excludes any tenant improvement allowances we received. We recognize rent expense for the lease components of our operating leases on a straight-line basis over the term of our lease. We recognize non-lease components, such as common area maintenance expenses, in the period we incur the expense. |
New Accounting Pronouncements - Recently Issued | New Accounting Pronouncements – Recently Issued In June 2016, the FASB issued guidance that changes the measurement of credit losses for most financial assets and certain other instruments. If we have credit losses, this updated guidance requires us to record allowances for these instruments under a new expected credit loss model. This model requires us to estimate the expected credit loss of an instrument over its lifetime, which represents the portion of the amortized cost basis we do not expect to collect. The new guidance requires us to remeasure our allowance in each reporting period we have credit losses. The new standard is effective for annual and interim periods beginning after December 15, 2019. Early adoption is permitted for periods beginning after December 15, 2018. When we adopt the new standard, we will make any adjustments to beginning balances through a cumulative-effect adjustment to accumulated deficit on that date. We plan to adopt this guidance on January 1, 2020. We are currently assessing the effects it could have 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. The updated guidance is effective for fiscal years beginning after December 15, 2019, and interim periods within those fiscal years. We plan to adopt this guidance on January 1, 2020 on a prospective basis. We are currently assessing the effects this updated guidance could have on our condensed consolidated financial statements and disclosures. In August 2018, the FASB updated its disclosure requirements related to Level 1, 2 and 3 fair value measurements. The update included deletion and modification of certain disclosure requirements and additional disclosure related to Level 3 measurements. The guidance is effective for fiscal years beginning after December 15, 2019 and early adoption is permitted. We adopted this updated guidance on January 1, 2019 and it did not have a significant impact on our disclosures. In November 2018, the FASB issued clarifying guidance on the interaction between the collaboration accounting guidance and the new revenue recognition guidance we adopted on January 1, 2018 (Topic 606). Below is the clarifying guidance and how we will implement it (in italics): 1) When a participant is considered a customer in a collaborative arrangement, all of the associated accounting under Topic 606 should be applied. ● We will apply all of the associated accounting under Topic 606 when we determine a participant in a collaborative arrangement is a customer. 2) Adds “unit of account” concept to collaboration accounting guidance to align with Topic 606. The “unit of account” concept is used to determine if revenue is recognized or if a contra expense is recognized from consideration received under a collaboration. ● We will use the “unit of account” concept when we receive consideration under a collaboration agreement to determine when we recognize revenue or a contra expense. 3) The clarifying guidance precludes us from recognizing revenue under Topic 606 when we determine a transaction with a collaborative partner is not a customer and is not directly related to the sales to third parties. ● When we conclude a collaboration partner is not a customer and is not directly related to the sales to third parties, we will not recognize revenue for the transaction. The updated guidance is effective for public entities for fiscal years beginning after December 15, 2019, and interim periods within those fiscal years. Early adoption is permitted. We plan to adopt this guidance on January 1, 2020. We are currently assessing the effects it could have on our condensed consolidated financial statements and disclosures. |
Summary of Significant Accoun_3
Summary of Significant Accounting Policies (Tables) | 6 Months Ended |
Jun. 30, 2019 | |
Accounting Policies [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 the product revenue allowance and reserve categories for the six months ended June 30, 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 438 774 138 1,350 Adjustment related to prior period sales (4 ) (25 ) — (29 ) Credits or payments made during the period (311 ) (210 ) — (521 ) Balance at June 30, 2019 $ 173 $ 832 $ 143 $ 1,148 |
Summary of Impact of Adoption of Topic 842 | The impact of the adoption of Topic 842 on the accompanying condensed consolidated balance sheet as of January 1, 2019 was as follows (in thousands): December 31, 2018 Adjustment due to adoption of Topic 842 January 1, 2019 Operating lease right-of-use-assets — 11,932 11,932 Other current liabilities 968 1,029 1,997 Long-term portion of lease liabilities 4,442 10,915 15,357 |
Investments and Fair Value Me_2
Investments and Fair Value Measurements (Tables) | 6 Months Ended |
Jun. 30, 2019 | |
Investments And Fair Value Measurements [Abstract] | |
Summary of Investments | The following is a summary of our investments at June 30, 2019 and December 31, 2018 (in thousands): Gross Unrealized Estimated June 30, 2019 Cost Gains Losses Fair Value Available-for-sale securities: Corporate debt securities $ 62,426 $ 25 $ (49 ) $ 62,402 Debt securities issued by U.S. government agencies 60,476 88 — 60,564 Total securities with a maturity of one year or less $ 122,902 $ 113 $ (49 ) $ 122,966 Corporate debt securities 22,443 — (35 ) 22,408 Total securities with a maturity of one to two years 22,443 — (35 ) 22,408 Total available-for-sale securities $ 145,345 $ 113 $ (84 ) $ 145,374 Gross Unrealized Estimated December 31, 2018 Cost Gains Losses Fair Value Available-for-sale securities: Corporate debt securities $ 81,770 $ — $ (151 ) $ 81,619 Debt securities issued by U.S. government agencies 85,578 — (42 ) 85,536 Total securities with a maturity of one year or less $ 167,348 $ — $ (193 ) $ 167,155 |
Assets Measured at Fair Value on a Recurring Basis | The following tables present the investments we held at June 30, 2019 and December 31, 2018 that are regularly measured and carried at fair value. The table segregates each security by the level within the fair value hierarchy of valuation techniques we utilized to determine the respective security’s fair value (in thousands): At June 30, 2019 Quoted Prices in Active Markets (Level 1) Significant Other Observable Inputs (Level 2) Money market funds (1) $ 131,241 $ 131,241 $ — Corporate debt securities (3) 84,811 — 84,811 Debt securities issued by U.S. government agencies (3) 60,563 — 60,563 Total $ 276,615 $ 131,241 $ 145,374 At December 31, 2018 Quoted Prices in Active Markets (Level 1) Significant Other Observable Inputs (Level 2) Money market funds (1) $ 82,343 $ 82,343 $ — Corporate debt securities (2) 81,619 — 81,619 Debt securities issued by U.S. government agencies (3) 85,536 — 85,536 Total $ 249,498 $ 82,343 $ 167,155 (1) Included in cash and cash equivalents on our condensed consolidated balance sheets. (2) At December 31, 2018, $1.0 million 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 sheets. |
Property, Plant and Equipment (
Property, Plant and Equipment (Table) | 6 Months Ended |
Jun. 30, 2019 | |
Property Plant And Equipment [Abstract] | |
Summary of Property and Equipment at Cost and Related Accumulated Depreciation | The following table presents property and equipment, at cost, and related accumulated depreciation (in thousands): June 30, December 31, 2019 2018 Furniture and fixtures $ 1,611 $ 1,611 Computer equipment and software 140 102 Manufacturing equipment 357 — Leasehold improvements 3,956 4,213 Total property and equipment, at cost 6,064 5,926 Less accumulated depreciation and amortization (621 ) (230 ) Total property and equipment, net $ 5,443 $ 5,696 |
Inventory (Tables)
Inventory (Tables) | 6 Months Ended |
Jun. 30, 2019 | |
Inventory Disclosure [Abstract] | |
Schedule of Inventory | Inventory consists of the following (in thousands): June 30, December 31, 2019 2018 Raw materials $ 8,034 $ — Work in process 192 — Finished goods 60 85 Total inventories $ 8,286 $ 85 |
Intangible Assets, Net (Tables)
Intangible Assets, Net (Tables) | 6 Months Ended |
Jun. 30, 2019 | |
Goodwill And Intangible Assets Disclosure [Abstract] | |
Schedule of Intangible Assets, Net | The following table presents intangible assets (in thousands): June 30, December 31, Estimated 2019 2018 useful life Acquired and in-licensed rights $ 2,262 $ 2,262 7 - 21 Years Capitalized regulatory approval milestones 90,000 90,000 16 Years Less accumulated amortization (6,256 ) (3,348 ) Total intangible assets, net $ 86,006 $ 88,914 |
Schedule of Estimated Future Amortization Expenses for Intangible Assets | Estimated future amortization expense for intangible assets as of June 30, 2019 is as follows (in thousands): Total 2019 $ 2,955 2020 5,879 2021 5,861 2022 5,856 2023 5,843 Thereafter 59,612 $ 86,006 |
License Agreements and Servic_2
License Agreements and Services Agreement with Ionis (Tables) | 6 Months Ended |
Jun. 30, 2019 | |
Related Party Transactions [Abstract] | |
Schedule Of Net Loss Share Related to Commercial Activities Under TTR Agreement | A summary of the loss share related to the commercial activities under the TTR Agreement is as follows: Three Months Ended June 30, Six Months Ended June 30, 2019 2019 Net losses incurred by the collaboration related to the commercial activities under the TTR Agreement $ (19,109 ) $ (34,207 ) Ionis' share of commercial losses under the TTR Agreement reflected in our condensed consolidated statements of operations (11,465 ) (20,521 ) Akcea's share of commercial losses under the TTR Agreement reflected in our condensed consolidated statements of operations (7,644 ) (13,686 ) |
Schedule Of Development Expenses Related to TTR Agreement | A summary of the development expenses related to the TTR Agreement is as follows: Three Months Ended June 30, Six Months Ended June 30, 2019 2019 Total development expense incurred by the collaboration related to development activities under the TTR Agreement $ (12,032 ) $ (26,412 ) Akcea's share of TTR development expense reflected in research and development expense in our condensed consolidated statements of operations $ (5,265 ) $ (11,398 ) |
Amounts Recorded Related to Transactions with Ionis Including Amounts Related to TTR Licensing Transaction | The following table summarizes the amounts recorded related to transactions with Ionis including amounts related to the TTR licensing transaction for the following periods (in thousands): Three Months Ended June 30, Six Months Ended June 30, 2019 2018 2019 2018 Operating expenses: Services performed by Ionis $ 1,075 $ 6,045 $ 2,150 $ 7,990 Active pharmaceutical ingredient manufactured by Ionis — — — 5,229 Pre-commercial inventory manufactured by Ionis — 5,996 — 5,996 Sublicensing expenses 3,000 — 78,000 — Out-of-pocket expenses paid by Ionis 1,275 16,029 2,846 15,224 Less: commercial share of loss in connection with the TTR license transaction (11,465 ) — (20,521 ) — Less: R&D share of loss in connection with the TTR license transaction 1,204 — 408 — Total operating expenses generated by transactions with Ionis (4,911 ) 28,070 62,883 34,439 Plus: distribution to Ionis — — 13,492 7,792 Total charges generated by transactions with Ionis (4,911 ) 28,070 76,375 42,231 (Receivable) payable balance (from) to Ionis at the beginning of the period (7,206 ) 27,737 18,901 14,365 Less: total amounts received from (paid to) Ionis during the period 4,206 (27,737 ) (28,187 ) (28,526 ) Less: receivable from Ionis — (933 ) — (933 ) Less: non-cash sublicensing expenses — — (75,000 ) — Total amount (receivable) payable (from) to Ionis at period end $ (7,911 ) $ 27,137 $ (7,911 ) $ 27,137 |
Equity and Stock-Based Compensa
Equity and Stock-Based Compensation (Tables) | 6 Months Ended |
Jun. 30, 2019 | |
Stockholders Equity [Abstract] | |
Weighted-Average Assumptions for Stock Options | For the six months ended June 30, 2019 and 2018, we used the following weighted-average assumptions in our Black-Scholes calculations: Employee Stock Options: Six Months Ended June 30, 2019 2018 Risk-free interest rate 2.5 % 2.8 % Dividend yield 0.0 % 0.0 % Volatility 76.3 % 77.7 % Expected life 6.1 years 6.1 years Board of Directors Stock Options: Six Months Ended June 30, 2019 2018 Risk-free interest rate 1.9 % 2.9 % Dividend yield 0.0 % 0.0 % Volatility 74.3 % 78.2 % Expected life 6.3 years 6.4 years 2017 ESPP: Six Months Ended June 30, 2019 2018 Risk-free interest rate 2.5 % 1.6 % Dividend yield 0.0 % 0.0 % Volatility 64.1 % 62.3 % Expected life 6 months 6 months |
Stock-Based Compensation (Table
Stock-Based Compensation (Tables) | 6 Months Ended |
Jun. 30, 2019 | |
Stockholders Equity [Abstract] | |
Stock-Based Compensation Expense | The following table summarizes stock-based compensation expense for the three and six months ended June 30, 2019 and 2018 (in thousands): Three Months Ended June 30, Six Months Ended June 30, 2019 2018 2019 2018 Cost of sales - product $ 137 $ — $ 255 $ — Research and development expenses 2,563 2,242 6,484 4,555 Selling, general and administrative expenses 11,663 9,884 26,184 13,954 Total $ 14,363 $ 12,126 $ 32,923 $ 18,509 |
Basic and Diluted Net Loss Pe_2
Basic and Diluted Net Loss Per Share (Tables) | 6 Months Ended |
Jun. 30, 2019 | |
Earnings Per Share [Abstract] | |
Basic Loss Per Share | The following table summarizes the distributable losses for the three and six months ended June 30, 2019 and 2018 (in thousands): Three Months Ended June 30, Six Months Ended June 30, 2019 2018 2019 2018 Net loss $ (37,323 ) $ (62,046 ) $ (10,136 ) $ (91,673 ) Distributions to Ionis — (7,792 ) (13,492 ) (7,792 ) Distributable losses $ (37,323 ) $ (69,838 ) $ (23,628 ) $ (99,465 ) The following table summarizes the reconciliation of weighted-average shares outstanding used in the calculation of basic loss per share for the three and six months ended June 30, 2019 and 2018: Three Months Ended June 30, Six Months Ended June 30, Determination of shares: 2019 2018 2019 2018 Weighted-average common shares outstanding owned by Ionis 70,221,338 60,832,494 69,406,181 53,182,685 Weighted-average common shares outstanding owned by others 22,573,900 21,492,157 22,351,368 21,332,650 Total weighted-average shares outstanding 92,795,238 82,324,651 91,757,549 74,515,335 The following table summarizes the calculation of basic loss per share for the three and six months ended June 30, 2019 and 2018 (in thousands, except per share and share amounts): Three Months Ended June 30, Six Months Ended June 30, 2019 2018 2019 2018 Losses allocated to Ionis $ (28,244 ) $ (51,606 ) $ (17,872 ) $ (70,990 ) Plus: Distribution to Ionis — 7,792 13,492 7,792 Losses available to Ionis $ (28,244 ) $ (43,814 ) $ (4,380 ) $ (63,198 ) Weighted-average common shares outstanding owned by Ionis 70,221,338 60,832,494 69,406,181 53,182,685 Basic loss per common share owned by Ionis $ (0.40 ) $ (0.72 ) $ (0.06 ) $ (1.19 ) Losses allocated to common shares owned by others $ (9,079 ) $ (18,232 ) $ (5,756 ) $ (28,475 ) Weighted-average common shares outstanding owned by others 22,573,900 21,492,157 22,351,368 21,332,650 Basic loss per common share owned by others $ (0.40 ) $ (0.85 ) $ (0.26 ) $ (1.33 ) |
Contractual Obligations and C_2
Contractual Obligations and Commitments (Tables) | 6 Months Ended |
Jun. 30, 2019 | |
Commitments And Contingencies Disclosure [Abstract] | |
Summary of Other Information related to Operating Leases | Other information related to our operating leases is as follows (dollar amounts in thousands): At June 30, 2019 Operating lease right-of-use assets $ 11,534 Operating lease liabilities (1) $ 16,178 Weighted average remaining lease term 9.2 years Weighted average discount rate 8 % (1) Current portion of $1.3 million included in other current liabilities and the difference of $14.9 million included in long-term portion of lease liabilities on our condensed consolidated balance sheet. |
Summary of Annual Maturities of Operating Lease Liabilities | Annual maturities of our operating lease liabilities as of June 30, 2019 are as follows (in thousands): Years ended December 31, Operating Leases Remainder of 2019 $ 1,256 2020 2,504 2021 2,507 2022 2,403 2023 2,400 Thereafter 11,961 Total minimum lease payments $ 23,031 Less: Imputed interest (6,853 ) Total operating lease liability $ 16,178 |
Organization and Basis of Pre_2
Organization and Basis of Presentation (Details) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | |||
Jun. 30, 2019 | Jun. 30, 2018 | Jun. 30, 2019 | Jun. 30, 2018 | Dec. 31, 2018 | |
Basis of Presentation and Organization [Abstract] | |||||
Accumulated deficit | $ 532,178 | $ 532,178 | $ 522,042 | ||
Net loss | 37,323 | $ 62,046 | 10,136 | $ 91,673 | |
Net cash provided in operating activities | 49,823 | $ (71,312) | |||
Cash, cash equivalents and short-term investments | $ 295,600 | $ 295,600 | |||
Ionis [Member] | Akcea [Member] | |||||
Basis of Presentation and Organization [Abstract] | |||||
Ownership percentage by Ionis | 76.00% | 76.00% |
Summary of Significant Accoun_4
Summary of Significant Accounting Policies - Narratives (Details) | Jun. 30, 2019RevenueStream |
Summary of Significant Accounting Policies [Abstract] | |
Number of revenue agents | 3 |
Summary of Significant Accoun_5
Summary of Significant Accounting Policies - Product Revenue, Net (Details) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2019 | Jun. 30, 2018 | Jun. 30, 2019 | Jun. 30, 2018 | |
Disaggregation of Revenue [Abstract] | ||||
Revenue, net | $ 26,623 | $ 18,321 | $ 190,439 | $ 35,429 |
TEGSEDI [Member] | ||||
Disaggregation of Revenue [Abstract] | ||||
Revenue, net | $ 9,900 | $ 16,600 | ||
Maximum [Member] | ||||
Summary Of Significant Accounting Policies [Line Items] | ||||
Period of time after billing when payment is received | 1 year | |||
Payment terms | 90 days | |||
Minimum [Member] | ||||
Summary Of Significant Accounting Policies [Line Items] | ||||
Payment terms | 30 days |
Summary of Significant Accoun_6
Summary of Significant Accounting Policies - Summary of Balances and Activity in Each of Product Revenue Allowance and Reserve Categories (Details) $ in Thousands | 6 Months Ended |
Jun. 30, 2019USD ($) | |
Revenue Allowance and Reserves Balance [Abstract] | |
Balance at December 31, 2018 | $ 348 |
Provision related to current period sales | 1,350 |
Adjustment related to prior period sales | (29) |
Credits or payments made during the period | (521) |
Balance at June 30, 2019 | 1,148 |
Chargebacks, Discounts and Fees [Member] | |
Revenue Allowance and Reserves Balance [Abstract] | |
Balance at December 31, 2018 | 50 |
Provision related to current period sales | 438 |
Adjustment related to prior period sales | (4) |
Credits or payments made during the period | (311) |
Balance at June 30, 2019 | 173 |
Government and Other Rebates [Member] | |
Revenue Allowance and Reserves Balance [Abstract] | |
Balance at December 31, 2018 | 293 |
Provision related to current period sales | 774 |
Adjustment related to prior period sales | (25) |
Credits or payments made during the period | (210) |
Balance at June 30, 2019 | 832 |
Returns [Member] | |
Revenue Allowance and Reserves Balance [Abstract] | |
Balance at December 31, 2018 | 5 |
Provision related to current period sales | 138 |
Adjustment related to prior period sales | 0 |
Balance at June 30, 2019 | $ 143 |
Summary of Significant Accoun_7
Summary of Significant Accounting Policies - Summary of Impact of Adoption of Topic 842 (Details) - USD ($) $ in Thousands | Jun. 30, 2019 | Jan. 01, 2019 | Dec. 31, 2018 |
New Accounting Pronouncements Or Change In Accounting Principle [Line Items] | |||
Operating lease right-of-use assets | $ 11,534 | ||
Other current liabilities | 1,713 | $ 968 | |
Long-term portion of lease liabilities | $ 14,909 | $ 4,442 | |
ASU 2016-02 [Member] | |||
New Accounting Pronouncements Or Change In Accounting Principle [Line Items] | |||
Operating lease right-of-use assets | $ 11,932 | ||
Other current liabilities | 1,997 | ||
Long-term portion of lease liabilities | 15,357 | ||
Restatement Adjustment [Member] | ASU 2016-02 [Member] | |||
New Accounting Pronouncements Or Change In Accounting Principle [Line Items] | |||
Operating lease right-of-use assets | 11,932 | ||
Other current liabilities | 1,029 | ||
Long-term portion of lease liabilities | $ 10,915 |
Investments and Fair Value Me_3
Investments and Fair Value Measurements - Summary of Investments (Details) - USD ($) $ in Thousands | Jun. 30, 2019 | Dec. 31, 2018 |
Summary of Investments [Abstract] | ||
Cost | $ 145,345 | |
Gross unrealized gains | 113 | |
Gross unrealized losses | (84) | |
Estimated fair value | 145,374 | |
Securities with Maturity of One Year or Less [Member] | ||
Summary of Investments [Abstract] | ||
Cost | 122,902 | $ 167,348 |
Gross unrealized gains | 113 | |
Gross unrealized losses | (49) | (193) |
Estimated fair value | 122,966 | 167,155 |
Securities with Maturity of More than One Year [Member] | ||
Summary of Investments [Abstract] | ||
Cost | 22,443 | |
Gross unrealized losses | (35) | |
Estimated fair value | 22,408 | |
Corporate Debt Securities [Member] | Securities with Maturity of One Year or Less [Member] | ||
Summary of Investments [Abstract] | ||
Cost | 62,426 | 81,770 |
Gross unrealized gains | 25 | |
Gross unrealized losses | (49) | (151) |
Estimated fair value | 62,402 | 81,619 |
Corporate Debt Securities [Member] | Securities with Maturity of More than One Year [Member] | ||
Summary of Investments [Abstract] | ||
Cost | 22,443 | |
Gross unrealized losses | (35) | |
Estimated fair value | 22,408 | |
Debt Securities Issued by U.S. Government Agencies [Member] | Securities with Maturity of One Year or Less [Member] | ||
Summary of Investments [Abstract] | ||
Cost | 60,476 | 85,578 |
Gross unrealized gains | 88 | |
Gross unrealized losses | (42) | |
Estimated fair value | $ 60,564 | $ 85,536 |
Investments and Fair Value Me_4
Investments and Fair Value Measurements - Fair Value Measurements (Details) - Recurring Basis [Member] - USD ($) $ in Thousands | Jun. 30, 2019 | Dec. 31, 2018 | ||
Fair Value Measurements [Abstract] | ||||
Money market funds | [1] | $ 131,241 | $ 82,343 | |
Total | 276,615 | 249,498 | ||
Corporate Debt Securities [Member] | ||||
Fair Value Measurements [Abstract] | ||||
Available-for-sale securities | 84,811 | 81,619 | [2] | |
Corporate Debt Securities [Member] | Cash and Cash Equivalents [Member] | ||||
Fair Value Measurements [Abstract] | ||||
Available-for-sale securities | 1,000 | |||
Debt Securities Issued by U.S. Government Agencies [Member] | ||||
Fair Value Measurements [Abstract] | ||||
Available-for-sale securities | [3] | 60,563 | 85,536 | |
Quoted Prices in Active Markets (Level 1) [Member] | ||||
Fair Value Measurements [Abstract] | ||||
Money market funds | [1] | 131,241 | 82,343 | |
Total | 131,241 | 82,343 | ||
Significant Other Observable Inputs (Level 2) [Member] | ||||
Fair Value Measurements [Abstract] | ||||
Total | 145,374 | 167,155 | ||
Significant Other Observable Inputs (Level 2) [Member] | Corporate Debt Securities [Member] | ||||
Fair Value Measurements [Abstract] | ||||
Available-for-sale securities | 84,811 | 81,619 | [2] | |
Significant Other Observable Inputs (Level 2) [Member] | Debt Securities Issued by U.S. Government Agencies [Member] | ||||
Fair Value Measurements [Abstract] | ||||
Available-for-sale securities | [3] | $ 60,563 | $ 85,536 | |
[1] | Included in cash and cash equivalents on our condensed consolidated balance sheets | |||
[2] | At December 31, 2018, $1.0 million was included in cash and cash equivalents on our condensed consolidated balance sheet, with the difference included in short-term investments on our condensed consolidated balance sheet | |||
[3] | Included in short-term investments on our condensed consolidated balance sheets |
Property Plant and Equipment -
Property Plant and Equipment - Summary of Property and Equipment at Cost and Related Accumulated Depreciation (Details) - USD ($) $ in Thousands | Jun. 30, 2019 | Dec. 31, 2018 |
Property Plant And Equipment [Line Items] | ||
Property and equipment, at cost | $ 6,064 | $ 5,926 |
Less accumulated depreciation and amortization | (621) | (230) |
Total property and equipment, net | 5,443 | 5,696 |
Furniture And Fixtures [Member] | ||
Property Plant And Equipment [Line Items] | ||
Property and equipment, at cost | 1,611 | 1,611 |
Computer Equipment And Software [Member] | ||
Property Plant And Equipment [Line Items] | ||
Property and equipment, at cost | 140 | 102 |
Manufacturing Equipment [Member] | ||
Property Plant And Equipment [Line Items] | ||
Property and equipment, at cost | 357 | |
Leasehold Improvements [Member] | ||
Property Plant And Equipment [Line Items] | ||
Property and equipment, at cost | $ 3,956 | $ 4,213 |
Property Plant Equipment - Narr
Property Plant Equipment - Narratives (Details) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | |
Jun. 30, 2019 | Jun. 30, 2019 | Jun. 30, 2018 | |
Property Plant And Equipment [Abstract] | |||
Depreciation | $ 200 | $ 400 | $ 54 |
Tenant improvement allowance | $ 3,600 |
Inventory - Schedule of Invento
Inventory - Schedule of Inventory (Details) - USD ($) $ in Thousands | Jun. 30, 2019 | Dec. 31, 2018 |
Inventory Disclosure [Abstract] | ||
Raw materials | $ 8,034 | |
Work in process | 192 | |
Finished goods | 60 | $ 85 |
Total inventories | $ 8,286 | $ 85 |
Intangible Assets, Net - Schedu
Intangible Assets, Net - Schedule of Intangible Assets (Details) - USD ($) $ in Thousands | 6 Months Ended | |
Jun. 30, 2019 | Dec. 31, 2018 | |
Finite Lived Intangible Assets [Line Items] | ||
Less accumulated amortization | $ (6,256) | $ (3,348) |
Total intangible assets, net | 86,006 | 88,914 |
Acquired and In-licensed Rights [Member] | ||
Finite Lived Intangible Assets [Line Items] | ||
Intangible assets, gross | $ 2,262 | 2,262 |
Acquired and In-licensed Rights [Member] | Minimum [Member] | ||
Finite Lived Intangible Assets [Line Items] | ||
Estimated useful life | 7 years | |
Acquired and In-licensed Rights [Member] | Maximum [Member] | ||
Finite Lived Intangible Assets [Line Items] | ||
Estimated useful life | 21 years | |
Capitalized Regulatory Approval Milestones [Member] | ||
Finite Lived Intangible Assets [Line Items] | ||
Intangible assets, gross | $ 90,000 | $ 90,000 |
Estimated useful life | 16 years |
Intangible Assets, Net - Additi
Intangible Assets, Net - Additional Information (Details) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2019 | Jun. 30, 2018 | Jun. 30, 2019 | Jun. 30, 2018 | |
Finite Lived Intangible Assets [Line Items] | ||||
Amortization of intangibles | $ 1,500 | $ 100 | $ 2,908 | $ 70 |
Patents [Member] | ||||
Finite Lived Intangible Assets [Line Items] | ||||
Weighted average remaining amortizable life | 11 years 2 months 23 days |
Intangible Assets, Net - Sche_2
Intangible Assets, Net - Schedule of Estimated Future Amortization Expenses for Intangible Assets (Details) - USD ($) $ in Thousands | Jun. 30, 2019 | Dec. 31, 2018 |
Goodwill And Intangible Assets Disclosure [Abstract] | ||
2019 | $ 2,955 | |
2020 | 5,879 | |
2021 | 5,861 | |
2022 | 5,856 | |
2023 | 5,843 | |
Thereafter | 59,612 | |
Total intangible assets, net | $ 86,006 | $ 88,914 |
Strategic Collaboration with _2
Strategic Collaboration with Novartis (Details) $ in Thousands | Feb. 22, 2019USD ($) | Jul. 31, 2017USD ($) | Jun. 30, 2019USD ($)shares | Jun. 30, 2018USD ($) | Mar. 31, 2018 | Mar. 31, 2017USD ($) | Jun. 30, 2019USD ($)shares | Jun. 30, 2018USD ($) | Jan. 31, 2017PerformanceObligation |
Strategic Collaboration with Novartis [Abstract] | |||||||||
Revenue | $ 26,623 | $ 18,321 | $ 190,439 | $ 35,429 | |||||
Revenue recognized from deferred revenue | $ 10,200 | 12,600 | $ 15,700 | 32,900 | |||||
Ionis [Member] | Akcea [Member] | |||||||||
Strategic Collaboration with Novartis [Abstract] | |||||||||
Common stock shares to be issued | shares | 2,837,373 | 2,837,373 | |||||||
Sublicense fee payable | $ 75,000 | $ 75,000 | |||||||
Development Services for AKCEA-APO(a)-L [Member] | |||||||||
Strategic Collaboration with Novartis [Abstract] | |||||||||
Transaction price | $ 64,000 | ||||||||
Development Services for AKCEA-APOCIII-L [Member] | |||||||||
Strategic Collaboration with Novartis [Abstract] | |||||||||
Transaction price | 40,100 | ||||||||
Delivery of AKCEA-APO(a)-L [Member] | |||||||||
Strategic Collaboration with Novartis [Abstract] | |||||||||
Transaction price | 1,500 | ||||||||
Delivery of AKCEA-APOCIII-L [Member] | |||||||||
Strategic Collaboration with Novartis [Abstract] | |||||||||
Transaction price | 2,800 | ||||||||
Licensing [Member] | |||||||||
Strategic Collaboration with Novartis [Abstract] | |||||||||
Revenue | 6,036 | 6,036 | |||||||
Licensing [Member] | Akcea [Member] | |||||||||
Strategic Collaboration with Novartis [Abstract] | |||||||||
Revenue | $ 150,000 | ||||||||
AKCEA-APO(a)-L [Member] | |||||||||
Strategic Collaboration with Novartis [Abstract] | |||||||||
Maximum amount of payments receivable for milestones | 675,000 | 675,000 | |||||||
Maximum amount of payments receivable for development milestones | 25,000 | 25,000 | |||||||
Maximum amount of payments receivable for regulatory milestones | 290,000 | 290,000 | |||||||
Maximum amount of payments receivable for commercialization milestones | 360,000 | $ 360,000 | |||||||
Royalty percentage received on sales of drug | 20.00% | ||||||||
AKCEA-APOCIII-L [Member] | |||||||||
Strategic Collaboration with Novartis [Abstract] | |||||||||
Sublicense fee payable | 75,000 | $ 75,000 | |||||||
Maximum amount of payments receivable for milestones | 530,000 | 530,000 | |||||||
Maximum amount of payments receivable for development milestones | 25,000 | 25,000 | |||||||
Maximum amount of payments receivable for regulatory milestones | 240,000 | 240,000 | |||||||
Maximum amount of payments receivable for commercialization milestones | 265,000 | $ 265,000 | |||||||
Royalty percentage received on sales of drug | 20.00% | ||||||||
Research and Development and License Revenue Under Collaborative Agreement [Member] | |||||||||
Strategic Collaboration with Novartis [Abstract] | |||||||||
Revenue | 10,722 | 18,321 | $ 167,784 | 35,429 | |||||
Novartis [Member] | |||||||||
Strategic Collaboration with Novartis [Abstract] | |||||||||
Upfront payment received | 75,000 | ||||||||
Portion of upfront payment retained | 60,000 | ||||||||
Portion of upfront payment paid as sublicense fee to Ionis | 15,000 | ||||||||
Number of separate performance obligations | PerformanceObligation | 4 | ||||||||
Transaction price | 108,400 | ||||||||
Premium received on shares issued by lonis | 28,400 | ||||||||
Revenue | 10,700 | 18,300 | 167,800 | 35,400 | |||||
Next prospective milestone | 25,000 | $ 25,000 | |||||||
Percentage of license fees, milestone payments and royalties paid as sublicense fee to lonis | 50.00% | ||||||||
Deferred revenue | 15,800 | $ 28,800 | $ 15,800 | $ 28,800 | |||||
Potential premium received if Ionis common stock is purchased in the future | $ 5,000 | ||||||||
Number of months from inception of agreement for IPO to be completed | 15 months | ||||||||
Proceeds from sale of common stock to Novartis in a private placement | $ 50,000 | ||||||||
Novartis [Member] | AKCEA-APOCIII-L [Member] | |||||||||
Strategic Collaboration with Novartis [Abstract] | |||||||||
License fee receivable per drug | $ 150,000 | $ 150,000 | |||||||
Novartis [Member] | Research and Development and License Revenue Under Collaborative Agreement [Member] | Revenue [Member] | Strategic Partner [Member] | |||||||||
Strategic Collaboration with Novartis [Abstract] | |||||||||
Concentration percentage | 100.00% |
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 | 6 Months Ended |
Jun. 30, 2019USD ($)Patientqtr | |
Development, Commercialization and License Agreement with Ionis [Abstract] | |
Royalty percentage paid on sales of lipid drug | 20.00% |
First annual sales threshold | $ | $ 500 |
Second annual sales threshold | $ | 1,000 |
Third annual sales threshold | $ | 2,000 |
Sales milestone payment | $ | $ 50 |
Number of quarters in which equal payments are made | qtr | 12 |
Percentage of license fees, milestone payments and royalties paid as sublicense fee to lonis | 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) | Oct. 17, 2018USD ($)shares | Aug. 03, 2018USD ($)shares | Apr. 17, 2018USD ($)Milestoneshares | Jun. 30, 2019USD ($)shares | Jun. 30, 2018USD ($) | Jun. 30, 2019USD ($)shares | Jun. 30, 2018USD ($) | Dec. 31, 2018USD ($)shares | Apr. 16, 2018shares |
TTR Development, Commercialization, Collaboration and License Agreement [Abstract] | |||||||||
Common stock, shares authorized (in shares) | shares | 125,000,000 | 125,000,000 | 125,000,000 | ||||||
Intangible assets, net | $ 86,006,000 | $ 86,006,000 | $ 88,914,000 | ||||||
Amortization expense of milestone payment | 1,419,000 | 2,822,000 | |||||||
Ionis [Member] | |||||||||
TTR Development, Commercialization, Collaboration and License Agreement [Abstract] | |||||||||
Costs incurred by Ionis prior to closing of TTR Agreement | $ 3,100,000 | ||||||||
Ionis [Member] | TTR License Agreement [Member] | |||||||||
TTR Development, Commercialization, Collaboration and License Agreement [Abstract] | |||||||||
Upfront licensing fee paid to Ionis | $ 150,000 | ||||||||
Shares issued in consideration of upfront licensing fee (in shares) | shares | 8,000,000 | ||||||||
Number of sales milestones | Milestone | 7 | ||||||||
Annual worldwide net sales required for subsequent milestone payments to be paid in cash | $ 750,000,000 | ||||||||
Notice period for termination of agreement | 90 days | ||||||||
Common stock shares issued to Ionis | shares | 10,700,000 | ||||||||
Stock purchased by Ionis | $ 200,000,000 | ||||||||
Common stock, shares authorized (in shares) | shares | 125,000,000 | 100,000,000 | |||||||
Intangible assets, net | $ 600,000 | ||||||||
Commercial inotersen inventory acquired from Ionis | $ 4,700,000 | ||||||||
Clinical material acquired from Ionis | 13,500,000 | $ 13,500,000 | |||||||
Percentage of profits and losses from development and commercialization of inotersen paid to Ionis, tier 1 | 60.00% | ||||||||
Percentage of profits and losses from development and commercialization of inotersen retained, tier 1 | 40.00% | ||||||||
Percentage of profits and losses from development and commercialization of inotersen paid to Ionis, tier 2 | 50.00% | ||||||||
Percentage of profits and losses from development and commercialization of inotersen retained, tier 2 | 50.00% | ||||||||
Percentage of profits and losses from development and commercialization of AKCEA-TTR-L paid to Ionis | 50.00% | ||||||||
Percentage of profits and losses from development and commercialization of AKCEA-TTR-L retained | 50.00% | ||||||||
Ionis [Member] | TTR License Agreement [Member] | Inotersen [Member] | |||||||||
TTR Development, Commercialization, Collaboration and License Agreement [Abstract] | |||||||||
Maximum amount of payments payable for milestones | 110,000,000 | 110,000,000 | |||||||
Ionis [Member] | TTR License Agreement [Member] | AKCEA-TTR-L [Member] | |||||||||
TTR Development, Commercialization, Collaboration and License Agreement [Abstract] | |||||||||
Maximum amount of payments payable for milestones | 145,000,000 | 145,000,000 | |||||||
Ionis [Member] | TTR License Agreement [Member] | Inotersen and AKCEA-TTR-L [Member] | |||||||||
TTR Development, Commercialization, Collaboration and License Agreement [Abstract] | |||||||||
Maximum amount of payments payable for milestones | 1,300,000,000 | 1,300,000,000 | |||||||
Ionis [Member] | TTR License Agreement [Member] | TEGSEDI [Member] | |||||||||
TTR Development, Commercialization, Collaboration and License Agreement [Abstract] | |||||||||
Common stock shares issued to Ionis | shares | 1,671,849 | 1,597,571 | |||||||
Amount of regulatory approval milestone paid by issuing common stock | $ 50,000,000 | $ 40,000,000 | |||||||
Amortization expense of milestone payment | $ 1,400,000 | $ 0 | $ 2,800,000 | $ 0 | |||||
Ionis [Member] | TTR License Agreement [Member] | TEGSEDI [Member] | Milestone Payment [Member] | |||||||||
TTR Development, Commercialization, Collaboration and License Agreement [Abstract] | |||||||||
Useful life of milestone payment amortized to cost of sales | 16 years |
License Agreements and Servic_5
License Agreements and Services Agreement with Ionis - Summary of Loss Share Related to Commercial Activities Under TTR Agreement (Details) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended |
Jun. 30, 2019 | Jun. 30, 2019 | |
Related Party Transaction [Line Items] | ||
Net losses incurred by the collaboration related to the commercial activities under the TTR Agreement | $ (19,109) | $ (34,207) |
Akcea [Member] | ||
Related Party Transaction [Line Items] | ||
Akcea's share of commercial losses under the TTR Agreement reflected in our condensed consolidated statements of operations | (7,644) | (13,686) |
Ionis [Member] | ||
Related Party Transaction [Line Items] | ||
Net loss share from commercial activities under arrangement with Ionis Pharmaceuticals, Inc. | $ (11,465) | $ (20,521) |
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 | 6 Months Ended |
Jun. 30, 2019 | Jun. 30, 2019 | |
Related Party Transaction [Line Items] | ||
Total development expense incurred by the collaboration related to development activities under the TTR Agreement | $ (12,032) | $ (26,412) |
Akcea [Member] | ||
Related Party Transaction [Line Items] | ||
Akcea's share of TTR development expense reflected in research and development expense in our condensed consolidated statements of operations | $ (5,265) | $ (11,398) |
License Agreements and Servic_7
License Agreements and Services Agreement with Ionis, Services Agreement (Details) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2019 | Jun. 30, 2018 | Jun. 30, 2019 | Jun. 30, 2018 | |
Services Agreement with Ionis [Abstract] | ||||
Payment term after receipt of invoice | 30 days | |||
Term of extension for services agreement | 6 months | |||
Operating expenses: | ||||
Distribution to Ionis | $ (7,792) | $ (13,492) | $ (7,792) | |
Ionis [Member] | Services Agreement [Member] | ||||
Operating expenses: | ||||
Services performed by Ionis | $ 1,075 | 6,045 | 2,150 | 7,990 |
Active pharmaceutical ingredient manufactured by Ionis | 5,229 | |||
Pre-commercial inventory manufactured by Ionis | 5,996 | 5,996 | ||
Sublicensing expenses | 3,000 | 78,000 | ||
Out-of-pocket expenses paid by Ionis | 1,275 | 16,029 | 2,846 | 15,224 |
Less: commercial share of loss in connection with the TTR license transaction | (11,465) | (20,521) | ||
Less: R&D share of loss in connection with the TTR license transaction | 1,204 | 408 | ||
Total operating expenses generated by transactions with Ionis | (4,911) | 28,070 | 62,883 | 34,439 |
Distribution to Ionis | 13,492 | 7,792 | ||
Total charges generated by transactions with Ionis | (4,911) | 28,070 | 76,375 | 42,231 |
(Receivable) payable balance (from) to Ionis at the beginning of the period | (7,206) | 27,737 | 18,901 | 14,365 |
Less: total amounts received from (paid to) Ionis during the period | 4,206 | (27,737) | (28,187) | (28,526) |
Less: receivable from Ionis | (933) | (933) | ||
Less: non-cash sublicensing expenses | (75,000) | |||
Total amount (receivable) payable (from) to Ionis at period end | $ (7,911) | $ 27,137 | $ (7,911) | $ 27,137 |
Collaboration and License Agr_2
Collaboration and License Agreement with PTC Therapeutics - Additional Information (Details) | Aug. 01, 2018USD ($) | Jun. 30, 2019USD ($)PerformanceObligation | Sep. 30, 2018USD ($) | Jun. 30, 2018USD ($) | Jun. 30, 2019USD ($)PerformanceObligation | Jun. 30, 2018USD ($) |
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||
Revenue | $ 26,623,000 | $ 18,321,000 | $ 190,439,000 | $ 35,429,000 | ||
Cardiometabolic License Agreement [Member] | ||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||
Sublicense fee paid | $ 3,000,000 | |||||
PTC Therapeutics [Member] | ||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||
Revenue | $ 12,000,000 | $ 6,000,000 | ||||
Deferred revenue | $ 0 | |||||
Number of separate performance obligations | PerformanceObligation | 2 | 2 | ||||
Consideration amount received | $ 6,000,000 | $ 6,000,000 | ||||
Payments on sublicense fee | 3,000,000 | |||||
PTC Therapeutics [Member] | Akcea [Member] | ||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||
Upfront payment received | 12,000,000 | |||||
Payment for sublicense royalty | 7,200,000 | |||||
Payment received | 6,000,000 | |||||
Maximum amount of payments receivable per drug for regulatory milestones | $ 8,000,000 | |||||
Period before PTC pays royalties on net sales of product after first commercial sale in Brazil | 12 months | |||||
Minimum revenue recognized in Latin America by PTC before paying royalties | $ 10,000,000 | |||||
TEGSEDI [Member] | ||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||
Revenue | $ 9,900,000 | $ 16,600,000 | ||||
TEGSEDI [Member] | PTC Therapeutics [Member] | Akcea [Member] | ||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||
Milestone payments and royalties retained percentage | 50.00% | |||||
TEGSEDI [Member] | PTC Therapeutics [Member] | Ionis [Member] | ||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||
Milestone payments and royalties payable percentage | 60.00% | |||||
WAYLIVRA [Member] | PTC Therapeutics [Member] | Ionis [Member] | ||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||
Milestone payments and royalties payable percentage | 40.00% |
Stock-Based Compensation - Stoc
Stock-Based Compensation - Stock Plans (Details) - USD ($) $ in Millions | 3 Months Ended | 6 Months Ended | |||
Jun. 30, 2019 | Jun. 30, 2019 | Jul. 01, 2019 | Jan. 01, 2019 | Dec. 31, 2018 | |
Stock Plans [Abstract] | |||||
Shares of common stock added | 92,921,173 | 92,921,173 | 89,345,978 | ||
Unrecognized compensation expense related to non-vested stock options | $ 82.1 | $ 82.1 | |||
Weighted average amortization period | 1 year 5 months 15 days | ||||
RSUs [Member] | |||||
Stock Plans [Abstract] | |||||
Unrecognized compensation expense related to non-vested stock options | $ 0.9 | $ 0.9 | |||
Weighted average amortization period | 1 year 5 months 12 days | ||||
2015 Equity Incentive Plan [Member] | |||||
Stock Plans [Abstract] | |||||
Number of shares authorized for issuance pursuant to stock awards (in shares) | 18,500,000 | 18,500,000 | |||
Number of shares available for future grant (in shares) | 3,824,563 | 3,824,563 | |||
2015 Equity Incentive Plan [Member] | Stock Options [Member] | |||||
Stock Plans [Abstract] | |||||
Options outstanding (in shares) | 13,068,974 | 13,068,974 | |||
Options exercisable (in shares) | 5,382,040 | 5,382,040 | |||
2015 Equity Incentive Plan [Member] | RSUs [Member] | |||||
Stock Plans [Abstract] | |||||
Restricted stock units outstanding (in shares) | 32,669 | 32,669 | |||
2017 Employee Stock Purchase Plan [Member] | |||||
Stock Plans [Abstract] | |||||
Shares of common stock added | 500,000 | ||||
Number of shares of common stock reserved for issuance (in shares) | 1,500,000 | 1,500,000 | |||
Shares issued under ESPP (in shares) | 0 | 17,740 | |||
Accrued liability for contributions to employee stock purchase plan | $ 0.5 | $ 0.5 | |||
2017 Employee Stock Purchase Plan [Member] | Subsequent Event [Member] | |||||
Stock Plans [Abstract] | |||||
Number of shares of common stock reserved for issuance (in shares) | 1,450,709 |
Equity and Stock-Based Compen_2
Equity and Stock-Based Compensation - Stock-based Valuation Information (Details) | 6 Months Ended | |
Jun. 30, 2019 | Jun. 30, 2018 | |
Employee Stock Option [Member] | ||
Weighted-Average Assumptions [Abstract] | ||
Risk-free interest rate | 2.50% | 2.80% |
Dividend yield | 0.00% | 0.00% |
Volatility | 76.30% | 77.70% |
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 | 1.90% | 2.90% |
Dividend yield | 0.00% | 0.00% |
Volatility | 74.30% | 78.20% |
Expected life | 6 years 3 months 18 days | 6 years 4 months 24 days |
2017 ESPP | ||
Weighted-Average Assumptions [Abstract] | ||
Risk-free interest rate | 2.50% | 1.60% |
Dividend yield | 0.00% | 0.00% |
Volatility | 64.10% | 62.30% |
Expected life | 6 months | 6 months |
Stock-Based Compensation - St_2
Stock-Based Compensation - Stock-Based Compensation Expense (Details) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2019 | Jun. 30, 2018 | Jun. 30, 2019 | Jun. 30, 2018 | |
Stock-Based Compensation [Abstract] | ||||
Stock-based compensation expense | $ 14,363 | $ 12,126 | $ 32,923 | $ 18,509 |
Cost of Sales - Product [Member] | ||||
Stock-Based Compensation [Abstract] | ||||
Stock-based compensation expense | 137 | 255 | ||
Research and Development Expenses [Member] | ||||
Stock-Based Compensation [Abstract] | ||||
Stock-based compensation expense | 2,563 | 2,242 | 6,484 | 4,555 |
Selling, General and Administrative Expenses [Member] | ||||
Stock-Based Compensation [Abstract] | ||||
Stock-based compensation expense | $ 11,663 | $ 9,884 | $ 26,184 | $ 13,954 |
Basic and Diluted Net Loss Pe_3
Basic and Diluted Net Loss Per Share (Details) - USD ($) $ / shares in Units, $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2019 | Jun. 30, 2018 | Jun. 30, 2019 | Jun. 30, 2018 | |
Basic Net Loss Per Share [Abstract] | ||||
Net loss | $ (37,323) | $ (62,046) | $ (10,136) | $ (91,673) |
Distribution to Ionis | (7,792) | (13,492) | (7,792) | |
Distributable losses | $ (37,323) | $ (69,838) | $ (23,628) | $ (99,465) |
Common Stock [Member] | ||||
Basic Net Loss Per Share [Abstract] | ||||
Weighted-average shares outstanding owned | 92,795,238 | 82,324,651 | 91,757,549 | 74,515,335 |
Common Stock [Member] | Ionis [Member] | ||||
Basic Net Loss Per Share [Abstract] | ||||
Net loss | $ (28,244) | $ (43,814) | $ (4,380) | $ (63,198) |
Distributable losses | $ (28,244) | $ (51,606) | $ (17,872) | $ (70,990) |
Weighted-average shares outstanding owned | 70,221,338 | 60,832,494 | 69,406,181 | 53,182,685 |
Basic loss per common share owned by Ionis | $ (0.40) | $ (0.72) | $ (0.06) | $ (1.19) |
Common Stock [Member] | Others [Member] | ||||
Basic Net Loss Per Share [Abstract] | ||||
Distributable losses | $ (9,079) | $ (18,232) | $ (5,756) | $ (28,475) |
Weighted-average shares outstanding owned | 22,573,900 | 21,492,157 | 22,351,368 | 21,332,650 |
Basic loss per common share owned by Ionis | $ (0.40) | $ (0.85) | $ (0.26) | $ (1.33) |
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 | Jun. 30, 2019USD ($) | Jun. 30, 2018USD ($) | Jun. 30, 2019USD ($) | Jun. 30, 2018USD ($) |
Operating Leased Assets [Line Items] | ||||||||
Operating lease expense | $ 0.5 | $ 0.7 | $ 1.1 | $ 0.9 | ||||
Operating lease payments paid | 1.3 | |||||||
Agreement to purchase goods and services | $ 6.6 | $ 6.6 | ||||||
Cambridge, Massachusetts [Member] | ||||||||
Operating Leased Assets [Line Items] | ||||||||
Lessee operating lease, lease commencement date | Apr. 30, 2015 | |||||||
Operating lease, option to extend | subsequently amended and expanded in February 2016 and March 2017 | |||||||
Lessee, Operating Lease, Existence of Option to Extend | false | |||||||
Operating lease expiration date | Apr. 30, 2020 | |||||||
Operating lease, lease termination period | 2019-04 | |||||||
Dublin, Ireland [Member] | ||||||||
Operating Leased Assets [Line Items] | ||||||||
Lessee operating lease, lease commencement date | May 31, 2019 | |||||||
Initial term of lease | 18 months | 18 months | ||||||
Operating lease, option to extend | the 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 | $ 3.8 | |||||||
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 Other Information related to Operating Leases (Details) $ in Thousands | Jun. 30, 2019USD ($) | |
Commitments And Contingencies Disclosure [Abstract] | ||
Operating lease right-of-use assets | $ 11,534 | |
Operating lease liabilities | $ 16,178 | [1] |
Weighted average remaining lease term | 9 years 2 months 12 days | |
Weighted average discount rate | 8.00% | |
[1] | Current portion of $1.3 million included in other current liabilities and the difference of $14.9 million included in long-term portion of lease liabilities on our condensed consolidated balance sheet. |
Contractual Obligations and C_5
Contractual Obligations and Commitments - Summary of Other Information related to Operating Leases (Parenthetical) (Details) - USD ($) $ in Thousands | Jun. 30, 2019 | Dec. 31, 2018 |
Commitments And Contingencies Disclosure [Abstract] | ||
Current portion of operating lease liabilities | $ 1,300 | |
Operating Lease, Liability, Current, Statement of Financial Position [Extensible List] | us-gaap:OtherLiabilitiesCurrent | |
Long-term portion of lease liabilities | $ 14,909 | $ 4,442 |
Contractual Obligations and C_6
Contractual Obligations and Commitments - Summary of Annual Maturities of Operating Lease Liabilities (Details) $ in Thousands | Jun. 30, 2019USD ($) | |
Commitments And Contingencies Disclosure [Abstract] | ||
Remainder of 2019 | $ 1,256 | |
2020 | 2,504 | |
2021 | 2,507 | |
2022 | 2,403 | |
2023 | 2,400 | |
Thereafter | 11,961 | |
Total minimum lease payments | 23,031 | |
Less: Imputed interest | (6,853) | |
Operating lease liabilities | $ 16,178 | [1] |
[1] | Current portion of $1.3 million included in other current liabilities and the difference of $14.9 million included in long-term portion of lease liabilities on our condensed consolidated balance sheet. |