Document and Entity Information
Document and Entity Information - USD ($) $ in Millions | 12 Months Ended | ||
Dec. 31, 2017 | Feb. 01, 2018 | Jun. 30, 2017 | |
Document and Entity Information | |||
Entity Registrant Name | INSMED INC | ||
Entity Central Index Key | 1,104,506 | ||
Document Type | 10-K | ||
Document Period End Date | Dec. 31, 2017 | ||
Amendment Flag | false | ||
Current Fiscal Year End Date | --12-31 | ||
Entity Well-known Seasoned Issuer | Yes | ||
Entity Voluntary Filers | No | ||
Entity Current Reporting Status | Yes | ||
Entity Filer Category | Large Accelerated Filer | ||
Entity Public Float | $ 1,057 | ||
Entity Common Stock, Shares Outstanding | 76,617,946 | ||
Document Fiscal Year Focus | 2,017 | ||
Document Fiscal Period Focus | FY |
Consolidated Balance Sheets
Consolidated Balance Sheets - USD ($) $ in Thousands | Dec. 31, 2017 | Dec. 31, 2016 |
Current assets: | ||
Cash and cash equivalents | $ 381,165 | $ 162,591 |
Prepaid expenses and other current assets | 8,279 | 5,816 |
Total current assets | 389,444 | 168,407 |
In-process research and development | 58,200 | 58,200 |
Fixed assets, net | 12,432 | 10,020 |
Other assets | 1,971 | 1,329 |
Total assets | 462,047 | 237,956 |
Current liabilities: | ||
Accounts payable | 14,671 | 10,439 |
Accrued expenses | 29,339 | 16,822 |
Other current liabilities | 646 | 728 |
Total current liabilities | 44,656 | 27,989 |
Debt, long-term | 55,567 | 54,791 |
Other long-term liabilities | 765 | 693 |
Total liabilities | 100,988 | 83,473 |
Shareholders' equity: | ||
Common stock, $0.01 par value; 500,000,000 authorized shares, 76,610,508 and 62,019,889 issued and outstanding shares at December 31, 2017 and December 31, 2016, respectively | 766 | 620 |
Additional paid-in capital | 1,318,181 | 919,164 |
Accumulated deficit | (957,885) | (765,236) |
Accumulated other comprehensive loss | (3) | (65) |
Total shareholders' equity | 361,059 | 154,483 |
Total liabilities and shareholders' equity | $ 462,047 | $ 237,956 |
Consolidated Balance Sheets (Pa
Consolidated Balance Sheets (Parenthetical) - $ / shares | Dec. 31, 2017 | Dec. 31, 2016 |
Statement of Financial Position [Abstract] | ||
Common stock, par value (in dollars per share) | $ 0.01 | $ 0.01 |
Common stock, authorized shares (in shares) | 500,000,000 | 500,000,000 |
Common stock, issued shares (in shares) | 76,610,508 | 62,019,889 |
Common stock, outstanding shares (in shares) | 76,610,508 | 62,019,889 |
Consolidated Statements of Comp
Consolidated Statements of Comprehensive Loss - USD ($) shares in Thousands, $ in Thousands | 12 Months Ended | ||
Dec. 31, 2017 | Dec. 31, 2016 | Dec. 31, 2015 | |
Statement of Comprehensive Income [Abstract] | |||
Revenues | $ 0 | $ 0 | $ 0 |
Operating expenses: | |||
Research and development | 109,749 | 122,721 | 74,277 |
General and administrative | 79,171 | 50,679 | 43,216 |
Total operating expenses | 188,920 | 173,400 | 117,493 |
Operating loss | (188,920) | (173,400) | (117,493) |
Investment income | 1,624 | 604 | 261 |
Interest expense | (5,925) | (3,498) | (2,889) |
Other income (expense), net | 300 | 119 | (33) |
Loss before income taxes | (192,921) | (176,175) | (120,154) |
Income tax (benefit) provision | (272) | 98 | (1,971) |
Net loss | $ (192,649) | $ (176,273) | $ (118,183) |
Basic and diluted net loss per share | $ (2.89) | $ (2.85) | $ (2.02) |
Weighted average basic and diluted common shares outstanding | 66,576 | 61,892 | 58,633 |
Net loss | $ (192,649) | $ (176,273) | $ (118,183) |
Other comprehensive income (loss): | |||
Foreign currency translation gains (losses) | 62 | (65) | 0 |
Total comprehensive loss | $ (192,587) | $ (176,338) | $ (118,183) |
Consolidated Statements of Shar
Consolidated Statements of Shareholders' Equity - USD ($) $ in Thousands | Total | Common Stock | Additional Paid-in Capital | Accumulated Deficit | Accumulated Other Comprehensive Loss |
Balance at Dec. 31, 2014 | $ 186,237 | $ 498 | $ 656,519 | $ (470,780) | $ 0 |
Balance (in shares) at Dec. 31, 2014 | 49,806,000 | ||||
Increase (Decrease) in Shareholders' Equity | |||||
Net loss | $ (118,183) | (118,183) | |||
Exercise of stock options (in shares) | 481,140 | 481,000 | |||
Exercise of stock options | $ 5,112 | $ 5 | 5,107 | ||
Net proceeds from issuance of common stock | 222,942 | $ 115 | 222,827 | ||
Net proceeds from issuance of common stock (in shares) | 11,500,000 | ||||
Issuance of common stock for vesting of RSUs (in shares) | 27,000 | ||||
Stock compensation expense | 15,590 | 15,590 | |||
Balance at Dec. 31, 2015 | 311,698 | $ 618 | 900,043 | (588,963) | 0 |
Balance (in shares) at Dec. 31, 2015 | 61,814,000 | ||||
Increase (Decrease) in Shareholders' Equity | |||||
Net loss | (176,273) | (176,273) | |||
Other comprehensive income (loss) | $ (65) | (65) | |||
Exercise of stock options (in shares) | 162,340 | 162,000 | |||
Exercise of stock options | $ 1,084 | $ 2 | 1,082 | ||
Issuance of common stock for vesting of RSUs (in shares) | 44,000 | ||||
Stock compensation expense | 18,039 | 18,039 | |||
Balance at Dec. 31, 2016 | 154,483 | $ 620 | 919,164 | (765,236) | (65) |
Balance (in shares) at Dec. 31, 2016 | 62,020,000 | ||||
Increase (Decrease) in Shareholders' Equity | |||||
Net loss | (192,649) | (192,649) | |||
Other comprehensive income (loss) | $ 62 | 62 | |||
Exercise of stock options (in shares) | 378,275 | 379,000 | |||
Exercise of stock options | $ 3,433 | $ 4 | 3,429 | ||
Net proceeds from issuance of common stock | 377,656 | $ 141 | 377,515 | ||
Net proceeds from issuance of common stock (in shares) | 14,123,000 | ||||
Issuance of common stock for vesting of RSUs | 1 | $ 1 | |||
Issuance of common stock for vesting of RSUs (in shares) | 89,000 | ||||
Stock compensation expense | 18,073 | 18,073 | |||
Balance at Dec. 31, 2017 | $ 361,059 | $ 766 | $ 1,318,181 | $ (957,885) | $ (3) |
Balance (in shares) at Dec. 31, 2017 | 76,611,000 |
Consolidated Statements of Cash
Consolidated Statements of Cash Flows - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2017 | Dec. 31, 2016 | Dec. 31, 2015 | |
Operating activities | |||
Net loss | $ (192,649) | $ (176,273) | $ (118,183) |
Adjustments to reconcile net loss to net cash used in operating activities: | |||
Depreciation and amortization | 2,901 | 2,438 | 1,982 |
Stock-based compensation expense | 18,073 | 18,039 | 15,590 |
Amortization of debt issuance costs | 118 | 281 | 458 |
Accrual of the end of term charge on the debt | 658 | 171 | 76 |
Changes in operating assets and liabilities: | |||
Prepaid expenses and other assets | (2,783) | 191 | (1,484) |
Accounts payable | 3,604 | 2,767 | (1,781) |
Accrued expenses and other | 10,461 | 5,678 | 2,642 |
Net cash used in operating activities | (159,617) | (146,708) | (100,700) |
Investing activities | |||
Purchase of fixed assets | (3,001) | (4,200) | (3,454) |
Net cash used in investing activities | (3,001) | (4,200) | (3,454) |
Financing activities | |||
Proceeds from issuance of debt | 0 | 30,000 | 0 |
Proceeds from issuance of common stock | 377,656 | 0 | 222,942 |
Proceeds from exercise of stock options | 3,433 | 1,084 | 5,112 |
Payment of debt issuance costs | 0 | (411) | (250) |
Net cash provided by financing activities | 381,089 | 30,673 | 227,804 |
Effect of exchange rates on cash and cash equivalents | 103 | (50) | 0 |
Net increase (decrease) in cash and cash equivalents | 218,574 | (120,285) | 123,650 |
Cash and cash equivalents at beginning of period | 162,591 | 282,876 | 159,226 |
Cash and cash equivalents at end of period | 381,165 | 162,591 | 282,876 |
Supplemental disclosures of cash flow information: | |||
Cash paid for interest | 5,165 | 3,608 | 2,948 |
Cash paid (received) for income taxes, net | $ 166 | $ 85 | $ (3,008) |
Description of Business and Bas
Description of Business and Basis of Presentation | 12 Months Ended |
Dec. 31, 2017 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
Description of Business and Basis of Presentation | Description of Business and Basis of Presentation Description of Business —Insmed is a global biopharmaceutical company focused on the unmet needs of patients with rare diseases. The Company's lead product candidate is amikacin liposome inhalation suspension (ALIS) (formerly known as liposomal amikacin for inhalation), which is in late-stage development for adult patients with treatment refractory nontuberculous mycobacteria (NTM) lung disease caused by Mycobacterium avium complex (MAC), a rare and often chronic infection that can cause irreversible lung damage and can be fatal. The Company's earlier clinical-stage pipeline includes INS1007, a novel oral reversible inhibitor of dipeptidyl peptidase 1, and INS1009, an inhaled nanoparticle formulation of a treprostinil prodrug. In recent years, the Company has funded its operations through public offerings of securities and debt financings. The Company expects to continue to incur losses both in its US and certain international entities, as the Company plans to fund research and development activities and commercial launch activities. The Company may need to raise additional capital to fund its operations, to develop and commercialize ALIS, to develop INS1007 and INS1009, and to develop, acquire, in-license or co-promote other products that address orphan or rare diseases. The Company believes it currently has sufficient funds to meet its financial needs for at least the next 12 months . The Company was incorporated in the Commonwealth of Virginia on November 29, 1999 and its principal executive offices are located in Bridgewater, New Jersey. The Company has legal entities in the United States (US), Ireland, Germany, France, the United Kingdom (UK), the Netherlands and Japan. Basis of Presentation —The consolidated financial statements include the accounts of the Company and its wholly-owned subsidiaries, Insmed Limited, Celtrix Pharmaceuticals, Inc., Insmed Holdings Limited, Insmed Ireland Limited, Insmed France SAS, Insmed Germany GmbH, Insmed Netherlands B.V. and Insmed Godo Kaisha. All intercompany transactions and balances have been eliminated in consolidation. |
Summary of Significant Accounti
Summary of Significant Accounting Policies | 12 Months Ended |
Dec. 31, 2017 | |
Accounting Policies [Abstract] | |
Summary of Significant Accounting Policies | Summary of Significant Accounting Policies Use of Estimates —The preparation of the consolidated financial statements in conformity with accounting principles generally accepted in the United States (GAAP) requires management to make estimates and assumptions that affect the amounts reported in the consolidated financial statements and accompanying notes. The Company bases its estimates and judgments on historical experience and on various other assumptions. The amounts of assets and liabilities reported in the Company's balance sheets and the amounts of expenses reported for each period presented are affected by estimates and assumptions, which are used for, but not limited to, the accounting for stock-based compensation, income taxes, loss contingencies, and accounting for research and development costs. Actual results could differ from those estimates. Investment Income and Interest Expense —Investment income consists of interest and dividend income earned on the Company's cash and cash equivalents. Interest expense consists primarily of interest costs related to the Company's debt. Cash and Cash Equivalents —The Company considers cash equivalents to be highly liquid investments with maturities of three months or less from the date of purchase. Fixed Assets, Net —Fixed assets are recorded at cost and are depreciated on a straight-line basis over the estimated useful lives of the assets. Estimated useful lives of three years to five years are used for computer equipment. Estimated useful lives of seven years are used for laboratory equipment, office equipment, manufacturing equipment and furniture and fixtures. Leasehold improvements are amortized over the shorter of the lease term or the estimated useful life of the asset. Long-lived assets are reviewed for impairment whenever events or changes in circumstances indicate that the carrying amount of an asset may not be recoverable. Recoverability of assets to be held and used is measured by a comparison of the carrying amount of an asset to estimated undiscounted future cash flows expected to be generated by the asset. If the carrying amount of an asset exceeds its estimated future cash flows, then an impairment charge is recognized for the amount by which the carrying value of the asset exceeds the fair value of the asset. Identifiable Intangible Assets —Identifiable intangible assets are measured at their respective fair values and are not amortized until commercialization. Once commercialization occurs, these intangible assets will be amortized over their estimated useful lives. The fair values assigned to the Company's intangible assets are based on reasonable estimates and assumptions given available facts and circumstances. Unanticipated events or circumstances may occur that may require the Company to review the assets for impairment. Events or circumstances that may require an impairment assessment include negative clinical trial results, the non-approval of a new drug application by a regulatory agency, material delays in the Company's development program or a sustained decline in market capitalization. Indefinite-lived intangible assets are not subject to periodic amortization. Rather, indefinite-lived intangibles are reviewed for impairment by applying a fair value based test on an annual basis or more frequently if events or circumstances indicate impairment may have occurred. Events or circumstances that may require an interim impairment assessment are consistent with those described above. The Company performs its annual impairment test as of October 1 of each year. The Company uses the income approach to derive the fair value of in-process research and development assets. This approach calculates fair value by estimating future cash flows attributable to the assets and then discounting these cash flows to a present value using a risk-adjusted discount rate. This approach requires significant management judgment with respect to unobservable inputs such as future volume, revenue and expense growth rates, changes in working capital use, appropriate discount rates and other assumptions and estimates. The estimates and assumptions used are consistent with the Company's business plans. A market based valuation approach was not considered given a lack of revenues and profits for the Company. Debt Issuance Costs —Debt issuance costs are amortized to interest expense using the effective interest rate method over the term of the debt. Debt issuance costs paid to the lender and third parties are reflected as a discount to the debt in the consolidated balance sheets. Unamortized debt issuance costs associated with extinguished debt are expensed in the period of the extinguishment. Fair Value Measurements —The Company categorizes its financial assets and liabilities measured and reported at fair value in the financial statements on a recurring basis based upon the level of judgments associated with the inputs used to measure their fair value. Hierarchical levels, which are directly related to the amount of subjectivity associated with the inputs used to determine the fair value of financial assets and liabilities, are as follows: • Level 1—Inputs are unadjusted, quoted prices in active markets for identical assets or liabilities at the measurement date. • Level 2—Inputs (other than quoted prices included in Level 1) are either directly or indirectly observable for the assets or liability through correlation with market data at the measurement date and for the duration of the instrument's anticipated life. • Level 3—Inputs reflect management's best estimate of what market participants would use in pricing the asset or liability at the measurement date. Consideration is given to the risk inherent in the valuation technique and the risk inherent in the inputs to the model. Each major category of financial assets and liabilities measured at fair value on a recurring basis is categorized based upon the lowest level of significant input to the valuations. The fair value hierarchy also requires an entity to maximize the use of observable inputs and minimize the use of unobservable inputs when measuring fair value. Financial instruments in Level 1 generally include US treasuries and mutual funds listed in active markets. The Company's only assets and liabilities which were measured at fair value as of December 31, 2017 and December 31, 2016 were its cash and cash equivalents of $381.2 million and $162.6 million , respectively. These amounts were measured at Level 1 using quoted prices in active markets for identical assets at the measurement date. The Company's cash and cash equivalents permit daily redemption and the fair values of these investments are based upon the quoted prices in active markets provided by the holding financial institutions. Cash equivalents consist of liquid investments with a maturity of three months or less from the date of purchase and the short-term investments consist of instruments with maturities greater than three months. The Company recognizes transfers between levels within the fair value hierarchy, if any, at the end of each quarter. There were no transfers in or out of Level 1, Level 2 or Level 3 during 2017 and 2016 . As of December 31, 2017 and 2016 , the Company held no securities that were in an unrealized loss or gain position. The Company reviews the status of each security quarterly to determine whether an other-than-temporary impairment has occurred. In making its determination, the Company considers a number of factors, including: (1) the significance of the decline; (2) whether the securities were rated below investment grade; (3) how long the securities have been in an unrealized loss position; and (4) the Company's ability and intent to retain the investment for a sufficient period of time for it to recover. Foreign Currency —The Company has operations in the US, Ireland, Germany, France, the UK and the Netherlands. The results of its non-US dollar based functional currency operations are translated to US dollars at the average exchange rates during the period. Assets and liabilities are translated at the exchange rate prevailing at the balance sheet date. Equity is translated at the prevailing exchange rate at the date of the equity transaction. Translation adjustments are included in shareholders' equity, as a component of other comprehensive loss. The Company realizes foreign currency transaction gains (losses) in the normal course of business based on movements in the applicable exchange rates. These gains (losses) are included as a component of other income (expense), net. Concentration of Credit Risk —Financial instruments that potentially subject the Company to concentrations of credit risk consist primarily of cash and cash equivalents. The Company places its cash equivalents with high credit-quality financial institutions and may invest its short-term investments in US treasury securities, mutual funds and government agency bonds. The Company has established guidelines relative to credit ratings and maturities that seek to maintain safety and liquidity. The Company relies on third-party manufacturers and suppliers for manufacturing and supply of its products. The inability of the suppliers or manufacturers to fulfill supply requirements of the Company could materially impact future operating results. A change in the relationship with the suppliers or manufacturer, or an adverse change in their business, could materially impact future operating results. Other Income —In 2015, the French National Agency for Medicines and Health Products Safety (ANSM) granted ALIS a Temporary Authorizations for Use (Autorisation Temporaire d'Utilisation or ATU). Pursuant to this program, the Company shipped product to pharmacies after receiving requests from physicians for patients in France. For the years ended December 31, 2017 , 2016 and 2015 , the revenue recorded was immaterial and is included as a component of other income (expense), net. The Company is initiating expanded access programs (EAPs) in other select territories in Europe, some of which may be fully reimbursed. EAPs are intended to make products available on a named patient basis before they are commercially available in accordance with local regulations. Research and Development —Research and development expenses consist primarily of salaries, benefits and other related costs, including stock-based compensation, for personnel serving in the Company's research and development functions, and other internal operating expenses, the cost of manufacturing a drug candidate, including the medical devices for drug delivery, for clinical study, the cost of conducting clinical studies, and the cost of conducting preclinical and research activities. In addition, research and development expenses include payments to third parties for the license rights to products in development (prior to marketing approval). The Company's expenses related to manufacturing its drug candidate and medical devices for clinical study are primarily related to activities at contract manufacturing organizations that manufacture ALIS, INS1007, and INS1009 and the medical devices for the Company's use. The Company's expenses related to clinical trials are primarily related to activities at contract research organizations that conduct and manage clinical trials on the Company's behalf. These contracts set forth the scope of work to be completed at a fixed fee or amount per patient enrolled. Payments under these contracts primarily depend on performance criteria such as the successful enrollment of patients or the completion of clinical trial milestones as well as time-based fees. Expenses are accrued based on contracted amounts applied to the level of patient enrollment and to activity according to the clinical trial protocol. Nonrefundable advance payments for goods or services that will be used or rendered for future research and development activities are deferred and capitalized. Such amounts are then recognized as an expense as the related goods are delivered or the services are performed, or when the goods or services are no longer expected to be provided. Stock-Based Compensation —The Company recognizes stock-based compensation expense for awards of equity instruments to employees and directors based on the grant-date fair value of those awards. The grant-date fair value of the award is recognized as compensation expense ratably over the requisite service period, which generally equals the vesting period of the award, and if applicable, is adjusted for expected forfeitures. The Company also grants performance-based stock options to employees. The grant-date fair value of the performance-based stock options is recognized as compensation expense over the implicit service period using the accelerated attribution method once it is probable that the performance condition will be achieved. Stock-based compensation expense is included in both research and development expenses and general and administrative expenses in the Consolidated Statements of Comprehensive Loss. Income Taxes —The Company accounts for income taxes under the asset and liability method. Deferred tax assets and liabilities are recognized for the future tax consequences attributable to differences between the financial statement carrying amounts of existing assets and liabilities and their respective tax bases and operating loss carry forwards. Deferred tax assets and liabilities are measured using enacted tax rates expected to apply to taxable income in the years in which those temporary differences are expected to be recovered or settled. The effect on deferred tax assets and liabilities of a change in tax rates is recognized in income in the period that includes the enactment date. A valuation allowance is recorded to reduce the deferred tax assets to the amount that is expected to be realized. In evaluating the need for a valuation allowance, the Company takes into account various factors, including the expected level of future taxable income and available tax planning strategies. If actual results differ from the assumptions made in the evaluation of a valuation allowance, the Company records a change in valuation allowance through income tax expense in the period such determination is made. The Company uses a comprehensive model for how it measures, presents and discloses an uncertain tax position taken or expected to be taken in a tax return. The Company may recognize the tax benefit from an uncertain tax position only if it is more likely than not that the tax position will be sustained on examination by taxing authorities, based solely on the technical merits of the position. The tax benefits recognized in the financial statements from such a position should be measured based on the largest benefit that has a greater than 50% likelihood to be sustained upon ultimate settlement. The Company had no uncertain tax positions as of December 31, 2017 and 2016 that qualified for either recognition or disclosure in the consolidated financial statements. The Company's policy for interest and penalties related to income tax exposures is to recognize interest and penalties as a component of the income tax (benefit) provision in the Consolidated Statements of Comprehensive Loss. Tax Cuts and Jobs Act On December 22, 2017, the US government enacted comprehensive tax legislation, referred to as the Tax Cuts and Jobs Act (the Tax Act). The Tax Act significantly revises US tax law by, among other provisions, lowering the US federal statutory income tax rate from 35% to 21%, imposing a mandatory one-time transition tax on previously deferred foreign earnings, and eliminating or reducing certain income tax deductions. ASC 740, Income Taxes requires the effects of changes in tax laws to be recognized in the period in which the legislation is enacted. However, due to the complexity and significance of the Tax Act’s provisions, the SEC staff issued Staff Accounting Bulletin No. 118 (SAB 118), which allows companies to record the tax effects of the Tax Act on a provisional basis based on a reasonable estimate, and then, if necessary, subsequently adjust such amounts during a limited measurement period as more information becomes available. The measurement period ends when a company has obtained, prepared, and analyzed the information necessary to finalize its accounting, but cannot extend beyond one year from enactment. The provisional amounts recorded for the Tax Act did not have a material impact on the Company's financial statements as of December 31, 2017, because its deferred temporary differences are fully offset by a valuation allowance and the Company does not have any significant offshore earnings from which to record the mandatory transition tax. However, given the significant complexity of the Tax Act, anticipated guidance from the US Treasury about implementing the Tax Act, and the potential for additional guidance from the SEC or the FASB related to the Tax Act, these estimates may be adjusted during the measurement period. The provisional amounts disclosed in our footnotes were based on the Company’s present interpretations of the Tax Act and current available information, including assumptions and expectations about future events, such as its projected financial performance, and are subject to further refinement as additional information becomes available (including the Company’s actual full Fiscal 2018 results of operations, as well as potential new or interpretative guidance issued by the FASB or the Internal Revenue Service and other tax agencies) and further analyses are completed. The Company continues to analyze the changes in certain income tax deductions, assess calculations of earnings and profits in certain foreign subsidiaries, including if those earnings are held in cash or other assets and gather additional data to compute the full impacts on the Company’s deferred and current tax assets and liabilities. Net Loss Per Common Share —Basic net loss per common share is computed by dividing net loss attributable to common shareholders by the weighted average number of common shares outstanding during the period. Diluted net loss per common share is computed by dividing net loss by the weighted average number of common shares and other dilutive securities outstanding during the period. Potentially dilutive securities from stock options and restricted stock units would be anti-dilutive as the Company incurred a net loss in all periods presented. Potentially dilutive common shares resulting from the assumed exercise of outstanding stock options are determined based on the treasury stock method. The following table sets forth the reconciliation of the weighted average number of shares used to compute basic and diluted net loss per share for the years ended December 31, 2017 , 2016 and 2015 . Years Ended December 31, 2017 2016 2015 (in thousands, except per share amounts) Numerator: Net loss $ (192,649 ) $ (176,273 ) $ (118,183 ) Denominator: Weighted average common shares used in calculation of basic net loss per share: 66,576 61,892 58,633 Effect of dilutive securities: Common stock options — — — Restricted stock and restricted stock units — — — Weighted average common shares outstanding used in calculation of diluted net loss per share 66,576 61,892 58,633 Net loss per share: Basic and Diluted $ (2.89 ) $ (2.85 ) $ (2.02 ) The following potentially dilutive securities have been excluded from the computations of diluted weighted average common shares outstanding as of December 31, 2017 , 2016 and 2015 as their effect would have been anti-dilutive (in thousands). 2017 2016 2015 Stock options to purchase common stock 8,609 7,117 5,274 Restricted stock and restricted stock units 47 89 44 Segment Information —The Company currently operates in one business segment, which is the development and commercialization of therapies for patients with rare diseases. A single management team that reports to the Chief Executive Officer comprehensively manages the entire business. The Company does not operate separate lines of business with respect to its products or product candidates. Accordingly, the Company does not have separate reportable segments. New Accounting Pronouncements (Adopted) —In August 2014, the Financial Accounting Standards Board (FASB) issued Accounting Standards Update (ASU) No. 2014-15, Presentation of Financial Statements—Going Concern: Disclosure of Uncertainties about an Entity's Ability to Continue as a Going Concern, which requires management to evaluate whether there is substantial doubt about the entity's ability to continue as a going concern and, if so, provide certain footnote disclosures. This ASU was effective for the annual period ended December 31, 2016, and interim reporting periods thereafter. The adoption of this standard did not have an impact on the Company's consolidated financial statements and related footnote disclosures. In March 2016, the FASB issued ASU 2016-9, Improvements to Employee Share-Based Payment Accounting , which amends ASC Topic 718, Compensation—Stock Compensation . ASU 2016-9 simplifies several aspects of the accounting for share-based payment transactions, including the income tax consequences, classification of awards as either equity or liabilities, and classification on the statement of cash flows. ASU 2016-9 is effective for fiscal years beginning after December 15, 2016, and interim periods within those fiscal years. The Company adopted ASU 2016-9 in the first quarter of 2017. ASU 2016-9 did not have a material impact on its consolidated financial statements. New Accounting Pronouncements (Not Yet Adopted) —In May 2014, the FASB issued ASU 2014-9, Revenue from Contracts with Customers (Topic 606) which amended the existing accounting standards for revenue recognition. ASU 2014-9 establishes principles for recognizing revenue upon the transfer of promised goods or services to customers, in an amount that reflects the expected consideration received in exchange for those goods or services. In July 2015, the FASB deferred the effective date for annual reporting periods beginning after December 15, 2017. The Company will adopt ASU 2014-9 in the first quarter of 2018 and expects the impact of adoption will not be material to its consolidated financial statements. In February 2016, the FASB issued ASU 2016-2, Leases (Topic 842) in order to increase transparency and comparability among organizations by recognizing lease assets and lease liabilities on the balance sheet for those leases classified as operating leases under previous GAAP. ASU 2016-2 requires that a lessee should recognize a liability to make lease payments (the lease liability) and a right-of-use asset representing its right to use the underlying asset for the lease term on the balance sheet. ASU 2016-2 is effective for fiscal years beginning after December 15, 2018 (including interim periods within those periods) using a modified retrospective approach and early adoption is permitted. The Company expects to adopt ASU 2016-2 in the first quarter of 2019 and is in the process of evaluating the impact of adoption on its consolidated financial statements. |
Accrued Expenses
Accrued Expenses | 12 Months Ended |
Dec. 31, 2017 | |
Payables and Accruals [Abstract] | |
Accrued Expenses | Accrued Expenses Accrued expenses consist of the following: As of December 31, 2017 2016 (in thousands) Accrued clinical trial expenses $ 7,837 $ 6,683 Accrued compensation 12,197 6,937 Accrued professional fees 4,500 1,992 Accrued technical operation expenses 2,182 591 Accrued interest payable 423 438 Accrued construction costs 1,719 — Other accrued expenses 481 181 $ 29,339 $ 16,822 |
Identifiable Intangible Assets
Identifiable Intangible Assets | 12 Months Ended |
Dec. 31, 2017 | |
Goodwill and Intangible Assets Disclosure [Abstract] | |
Identifiable Intangible Assets | Identifiable Intangible Assets The Company's only identifiable intangible asset was in-process research and development (IPRD) related to ALIS as of December 31, 2017 and 2016 . The total intangible IPRD asset was $58.2 million as of December 31, 2017 and 2016 , which resulted from the initial amount recorded at the time of the Company's merger with Transave in 2010 and subsequent adjustments in the value. The Company uses the income approach to derive the fair value of in-process research and development assets. This approach calculates fair value by estimating future cash flows attributable to the assets and then discounting these cash flows to a present value using a risk-adjusted discount rate. Identifiable intangible assets are measured at their respective fair values and will not be amortized until commercialization. If commercialization occurs, intangible assets will be amortized over their estimated useful lives. As of December 31, 2017 , the Company did not identify any indicators of impairment of its in-process research and development intangible assets and the implied value of the intangible assets was more than 100% greater than the book value. |
Fixed Assets, net
Fixed Assets, net | 12 Months Ended |
Dec. 31, 2017 | |
Property, Plant and Equipment [Abstract] | |
Fixed Assets, net | Fixed Assets, net Fixed assets are stated at cost and depreciated using the straight-line method, based on useful lives as follows: Estimated Useful Life (years) As of December 31, Asset Description 2017 2016 (in thousands) Lab equipment 7 $ 7,055 $ 5,662 Furniture and fixtures 7 1,937 1,903 Computer hardware and software 3 - 5 2,325 2,251 Office equipment 7 65 65 Manufacturing equipment 7 1,436 1,148 Leasehold improvements lease term 6,939 6,735 Construction in Progress (CIP) — 3,320 — 23,077 17,764 Less accumulated depreciation (10,645 ) (7,744 ) Fixed assets, net $ 12,432 $ 10,020 Depreciation expense was $2.9 million , $2.4 million and $2.0 million for the years ended December 31, 2017 , 2016 and 2015 , respectively. |
Debt
Debt | 12 Months Ended |
Dec. 31, 2017 | |
Debt Disclosure [Abstract] | |
Debt | Debt On June 29, 2012, the Company and its domestic subsidiaries, as co-borrowers, entered into a Loan and Security Agreement with Hercules Technology Growth Capital, Inc. (as subsequently amended, the Prior Loan Agreement) under which the Company borrowed an aggregate of $25.0 million at an interest rate of 9.25% . The Company was required to pay an "end of term" charge of $390,000 in January of 2016, which was charged to interest expense (and accreted to the debt) using the effective interest method over the life of the Prior Loan Agreement. On September 30, 2016, the Company and its domestic subsidiaries, as co-borrowers, entered into an Amended and Restated Loan and Security Agreement (the A&R Loan Agreement) with Hercules Capital, Inc. (Hercules). The A&R Loan Agreement included a total commitment from Hercules of up to $55.0 million , of which $25.0 million was previously outstanding. The amount of borrowings was increased by $10.0 million to an aggregate total of $35.0 million on September 30, 2016. An additional $20.0 million was available at the Company's option through June 30, 2017 subject to certain conditions, including the payment of a facility fee of 0.375% . The Company exercised this option in early October 2016 and borrowed an additional $20.0 million in connection with its upfront payment obligation under the License Agreement with AstraZeneca (see Note 10 ). The interest rate for the term is floating and is defined as the greater of (i) 9.25% or (ii) 9.25% plus the sum of the US prime rate minus 4.50% , along with a backend fee of 4.15% of the aggregate principal amount outstanding and an aggregate facility fee of $337,500 . The maturity date of the loan facility was also extended to October 1, 2020. In connection with the Company generating and announcing top-line data from the CONVERT study on September 5, 2017 that supports the filing of a New Drug Application (NDA), along with the completion of the equity financing, the interest-only period was automatically extended through May 1, 2019 and the Company's requirement to have a consolidated minimum cash liquidity in an amount no less than $25.0 million was eliminated. In connection with the A&R Loan Agreement, the Company granted Hercules a first position lien on all of the Company's assets, excluding intellectual property. Prepayment of the loans made pursuant to the A&R Loan Agreement is subject to penalty. The backend fee of 4.15% on the aggregate outstanding principal balance will be charged to interest expense (and accreted to the debt) using the effective interest method over the original life of the A&R Loan Agreement. Debt issuance fees paid to Hercules were recorded as a discount on the debt and are being amortized to interest expense using the effective interest method over the life of the A&R Loan Agreement. The A&R Loan Agreement also contains representations and warranties by the Company and Hercules and indemnification provisions in favor of Hercules and customary covenants (including limitations on other indebtedness, liens, acquisitions, investments and dividends, and a minimum liquidity covenant), and events of default (including payment defaults, breaches of covenants following any applicable cure period, a material impairment in the perfection or priority of the lender's security interest or in the collateral, and events relating to bankruptcy or insolvency). Upon the occurrence of an event of default, a default interest rate of an additional 5% may be applied to the outstanding loan balances, and the lender may terminate its lending commitment, declare all outstanding obligations immediately due and payable, and take such other actions as set forth in the A&R Loan Agreement. The following table presents the components of the Company's debt balance as of December 31, 2017 (in thousands): Debt: Note payable under A&R Loan Agreement $ 55,000 Accretion of end of term charge 828 Issuance fees paid to lender (261 ) Current portion of long-term debt — Long-term debt $ 55,567 Future principal repayments of the Company's long-term debt are as follows (in thousands): Year Ending in December 31: 2018 $ — 2019 13,399 2020 41,601 $ 55,000 The estimated fair value of the debt (categorized as a Level 2 liability for fair value measurement purposes) is determined using current market factors and the ability of the Company to obtain debt at comparable terms to those that are currently in place. As of December 31, 2017 and 2016 , the fair value of the Company's debt approximates the carrying amount. In February 2018, the Company notified Hercules that it will repay the A&R Loan Agreement in full on February 28, 2018. The total aggregate cash payable to Hercules for the early prepayment of debt, inclusive of accrued interest, the backend fee and an early payment penalty will be approximately $58.0 million . |
Shareholders' Equity
Shareholders' Equity | 12 Months Ended |
Dec. 31, 2017 | |
Stockholders' Equity Note [Abstract] | |
Shareholders' Equity | Shareholders' Equity Common Stock —As of December 31, 2017 , the Company had 500,000,000 shares of common stock authorized with a par value of $0.01 and 76,610,508 shares of common stock issued and outstanding. In addition, as of December 31, 2017 , the Company had reserved 8,608,921 shares of common stock for issuance upon the exercise of outstanding common stock options and 46,914 shares of common stock for issuance upon the vesting of restricted stock units. In September 2017, the Company completed an underwritten public offering of 14,123,150 shares of the Company’s common stock, which included the underwriter’s exercise in full of its over-allotment option of 1,842,150 shares, at a price to the public of $28.50 per share. The Company’s net proceeds from the sale of the shares, after deducting underwriting discounts and offering expenses of $24.8 million , were approximately $377.7 million . In April 2015, the Company completed an underwritten public offering of 11,500,000 shares of the Company's common stock, which included the underwriter's exercise in full of its over-allotment option of 1,500,000 shares, at a price to the public of $20.65 per share. The Company's net proceeds from the sale of the shares, after deducting underwriting discounts and offering expenses of $14.5 million , were approximately $222.9 million . Preferred Stock —As of December 31, 2017 and 2016 , the Company had 200,000,000 shares of preferred stock authorized with a par value of $0.01 and no shares of preferred stock were issued and outstanding. |
Stock-Based Compensation
Stock-Based Compensation | 12 Months Ended |
Dec. 31, 2017 | |
Share-based Compensation [Abstract] | |
Stock-Based Compensation | Stock-Based Compensation The Company’s current equity compensation plan, the 2017 Incentive Plan, was approved by shareholders at the Company’s Annual Meeting of Shareholders on May 18, 2017. The 2017 Incentive Plan is administered by the Compensation Committee and the Board of Directors of the Company. Under the terms of the 2017 Incentive Plan, the Company is authorized to grant a variety of incentive awards based on its common stock, including stock options (both incentive stock options and non-qualified stock options), RSUs, performance options/shares and other stock awards, as well as pay incentive bonuses to eligible employees and non-employee directors. On May 18, 2017, upon the approval of the 2017 Incentive Plan by shareholders, 5,000,000 shares were authorized for issuance thereunder, plus any shares subject to then-outstanding awards under the 2015 Incentive Plan and the 2013 Incentive Plan that subsequently were canceled, terminated unearned, expired, were forfeited, lapsed for any reason or were settled in cash without the delivery of shares. As of December 31, 2017 , 4,910,002 shares remained for future issuance under the 2017 Incentive Plan. The 2017 Incentive Plan will terminate on April 3, 2027 unless it is extended or terminated earlier pursuant to its terms. In addition, from time to time, the Company makes inducement grants of stock options. These awards are made pursuant to the Nasdaq inducement grant exception as a component of new hires’ employment compensation in connection with the Company’s equity grant program. During the twelve months ended December 31, 2017 and 2016 , the Company granted inducement stock options covering 266,230 and 88,060 , respectively, shares of the Company's common stock to new employees. Stock Options —The Company calculates the fair value of stock options granted using the Black-Scholes valuation model. The following table summarizes the grant date fair value and assumptions used in determining the fair value of all stock options granted, including grants of inducement options, during the years ended December 31, 2017 , 2016 and 2015 . 2017 2016 2015 Volatility 71% - 79% 74% - 77% 78% - 82% Risk-free interest rate 1.73% - 2.13% 1.00% - 1.90% 1.31% - 1.75% Dividend yield 0.0% 0.0% 0.0% Expected option term (in years) 6.25 6.25 6.25 Weighted average fair value of stock options granted $10.52 $8.77 $14.20 For the years ended December 31, 2017 , 2016 and 2015 , the volatility factor was based on the Company’s historical volatility during the expected option term. Estimated forfeitures were based on the actual percentage of option forfeitures since the closing of the Company’s merger with Transave, Inc. in December 2010 for the years ended December 31, 2016 and 2015 . Beginning with the year ended December 31, 2017 , estimated forfeitures were based on the actual percentage of option forfeitures over the expected option term. From time to time, the Company grants performance-condition options to certain employees. Vesting of these options is subject to the Company achieving certain performance criteria established at the date of grant and the individuals fulfilling a service condition (continued employment). As a result of the Marketing Authorization Application (MAA) acceptance for ALIS, which was received from the European Medicines Agency (EMA) in February 2015, the vesting of performance options totaling $1.5 million were recorded as non-cash compensation expense in the first quarter of 2015 . As of December 31, 2017 , the Company had performance options totaling 133,334 shares outstanding. The following table summarizes stock option activity for stock options granted for the years ended December 31, 2017 , 2016 and 2015 as follows: Number of Shares Weighted Average Exercise Price Weighted Average Remaining Contractual Life in Years Aggregate Intrinsic Value (in '000) Options outstanding at January 1, 2015 4,400,106 $ 10.59 Granted 1,902,850 20.45 Exercised (481,140 ) 10.62 Forfeited and expired (548,094 ) 15.43 Options outstanding at December 31, 2015 5,273,722 $ 13.64 Vested and expected to vest at December 31, 2015 5,059,645 13.46 Exercisable at December 31, 2015 1,991,141 8.70 Options outstanding at December 31, 2015 5,273,722 $ 13.64 Granted 2,532,675 12.96 Exercised (162,340 ) 6.68 Forfeited and expired (527,351 ) 17.08 Options outstanding at December 31, 2016 7,116,706 $ 13.30 Vested and expected to vest at December 31, 2016 6,850,658 13.25 Exercisable at December 31, 2016 3,113,998 11.28 Options outstanding at December 31, 2016 7,116,706 $ 13.30 Granted 2,284,710 15.92 Exercised (378,275 ) 9.08 Forfeited and expired (414,220 ) 15.50 Options outstanding at December 31, 2017 8,608,921 $ 14.08 7.4 $ 147,260 Vested and expected to vest at December 31, 2017 8,325,255 $ 14.03 7.4 $ 142,783 Exercisable at December 31, 2017 4,229,478 $ 12.71 6.3 $ 78,138 The total intrinsic value of stock options exercised during the years ended December 31, 2017 , 2016 and 2015 was $4.3 million , $1.0 million and $4.7 million , respectively. As of December 31, 2017 , there was $29.7 million of unrecognized compensation expense related to unvested stock options, which is expected to be recognized over a weighted average period of 2.6 years . Included above in unrecognized compensation expense was $1.1 million related to outstanding performance-based options. The following table summarizes the range of exercise prices and the number of stock options outstanding and exercisable as of December 31, 2017 : Outstanding as of December 31, 2017 Exercisable as of Range of Exercise Prices Number of Options Weighted Average Remaining Contractual Term (in years) Weighted Average Exercise Price Number of Options Weighted Average Exercise Price $ 3.03 $ 4.55 973,195 4.65 $ 3.60 973,195 $ 3.60 $ 6.90 $ 6.90 137,577 5.22 $ 6.90 100,077 $ 6.90 $ 6.96 $ 10.85 1,077,621 8.29 $ 10.76 420,374 $ 10.62 $ 11.14 $ 12.58 1,088,035 6.39 $ 12.17 781,991 $ 12.18 $ 12.66 $ 13.67 1,043,273 8.74 $ 13.59 101,126 $ 13.26 $ 13.94 $ 15.91 862,300 7.76 $ 14.97 406,441 $ 14.53 $ 16.07 $ 16.16 1,008,750 7.69 $ 16.13 463,629 $ 16.12 $ 16.35 $ 17.24 868,650 9.24 $ 17.13 23,999 $ 16.85 $ 17.36 $ 22.76 1,415,700 7.14 $ 21.18 927,334 $ 21.23 $ 22.84 $ 31.78 133,820 8.86 $ 27.47 31,312 $ 23.74 Restricted Stock and Restricted Stock Units —The Company may grant Restricted Stock (RS) and Restricted Stock Units (RSUs) to employees and non-employee directors. Each share of RS vests upon and each RSU represents a right to receive one share of the Company's common stock upon the completion of a specific period of continued service or achievement of a certain milestone. RS and RSU awards granted are valued at the market price of the Company's common stock on the date of grant. The Company recognizes noncash compensation expense for the fair values of these RS and RSUs on a straight-line basis over the requisite service period of these awards. The following table summarizes RSU awards granted during the years ended December 31, 2017 , 2016 and 2015 : Number of RSUs Weighted Average Grant Price Outstanding at January 1, 2015 20,502 $ 19.47 Granted 49,776 16.07 Released (26,724 ) (18.68 ) Forfeited — — Outstanding at December 31, 2015 43,554 $ 19.47 Granted 89,194 10.85 Released (43,554 ) (16.07 ) Forfeited — — Outstanding at December 31, 2016 89,194 $ 10.85 Granted 46,914 17.16 Released (89,194 ) (10.85 ) Forfeited — — Outstanding at December 31, 2017 46,914 $ 17.16 The following table summarizes the stock-based compensation recorded in the Consolidated Statements of Comprehensive Loss related to stock options and RSUs during the years ended December 31, 2017 , 2016 and 2015 : 2017 2016 2015 (in millions) Research and development expenses $ 6.5 $ 6.2 $ 4.0 General and administrative expenses 11.6 11.8 11.6 Total (1) $ 18.1 $ 18.0 $ 15.6 _________________________________ (1) Includes $0.0 million , $1.7 million and $2.3 million for the years ended December 31, 2017 , 2016 and 2015 , respectively, for the remeasurement of certain stock options and RSUs that occurred during May 2013. |
Income Taxes
Income Taxes | 12 Months Ended |
Dec. 31, 2017 | |
Income Tax Disclosure [Abstract] | |
Income Taxes | Income Taxes The income tax (benefit) provision was $(0.3) million , $0.1 million and $(2.0) million and the effective rates were approximately 0% , 0% and 2% for the years ended December 31, 2017 , 2016 and 2015 , respectively. The income tax (benefit) for the year ended December 31, 2017 reflects the reversal of the valuation allowance related to alternative minimum tax (AMT) that the Company paid in 2009. As a result of the Tax Act, the Company recorded a noncurrent receivable to reflect the refund due to the Company in future periods relating to the previously paid AMT. In addition, the income tax (benefit) provision for the years ended December 31, 2017 and 2016 reflected current income tax expense recorded as a result of the taxable income in certain of the Company's subsidiaries in Europe. The income tax benefit recorded and the effective tax rates for the year ended December 31, 2015 primarily reflected the reversal of valuation allowances previously recorded against the Company's New Jersey State net operating losses (NOLs) that resulted from the Company's sale of $24.3 million of its New Jersey State NOLs under the State of New Jersey's Technology Business Tax Certificate Transfer Program (the Program) for cash of $2.0 million , respectively, net of commissions. The Program allows qualified technology and biotechnology businesses in New Jersey to sell unused amounts of NOLs and defined research and development tax credits for cash. In 2015, the Company reached the lifetime maximum cap of NOLs that can be sold to the State of New Jersey. The Company is subject to US federal and state income taxes and the statute of limitations for tax audit is open for the federal tax returns for the years ended 2014 and later, and is generally open for certain states for the years 2013 and later. The Company has incurred net operating losses since inception, except for the year ended December 31, 2009. Such loss carryforwards would be subject to audit in any tax year in which those losses are utilized, notwithstanding the year of origin. The Company's policy is to recognize interest accrued related to unrecognized tax benefits and penalties in income tax expense. The Company has recorded no such expense. As of December 31, 2017 and 2016 , the Company has recorded no reserves for unrecognized income tax benefits, nor has it recorded any accrued interest or penalties related to uncertain tax positions. The Company does not anticipate any material changes in the amount of unrecognized tax positions over the next 12 months. For the years ended December 31, 2017 and 2016 , the Company was also subject to foreign income taxes as a result of legal entities established for activities in Europe. The Company's loss before income taxes in the US and globally was as follows (in thousands): Years Ended December 31, 2017 2016 2015 US $ (136,682 ) $ (140,354 ) $ (100,278 ) Foreign (56,239 ) (35,821 ) (19,876 ) Total $ (192,921 ) $ (176,175 ) $ (120,154 ) The Company's income tax (benefit) provision consisted of the following (in thousands): Years Ended December 31, 2017 2016 2015 Current: Federal $ — $ — $ — State 3 3 (2,015 ) Foreign 142 95 44 145 98 (1,971 ) Deferred: Federal (417 ) — — State — — — Foreign — — — (417 ) — — Total $ (272 ) $ 98 $ (1,971 ) The reconciliation between the federal statutory tax rate of 34% and the Company's effective tax rate is as follows: Years Ended December 31, 2017 2016 2015 Statutory federal tax rate 34 % 34 % 34 % Permanent items (3 )% (3 )% (4 )% State income taxes, net of federal benefit 4 % 4 % 4 % R&D and other tax credits 8 % 8 % 12 % Foreign income taxes (6 )% (4 )% (1 )% Impact of 2017 Tax Act (49 )% — % — % Change in valuation allowance 12 % (39 )% (43 )% Other — % — % — % Effective tax rate — % — % 2 % Deferred tax assets and liabilities are determined based on the difference between financial statement and tax bases using enacted tax rates in effect for the year in which the differences are expected to reverse. The components of the deferred tax assets and liabilities consist of the following: As of December 31, 2017 2016 Deferred tax assets: Net operating loss carryforwards $ 186,342 $ 228,729 General business credits 66,371 50,648 Product license 7,730 11,783 Alternative minimum tax (AMT) credit — 418 Other 17,217 16,265 Gross deferred tax assets $ 277,660 $ 307,843 Deferred tax liabilities: In-process research and development $ (16,360 ) $ (23,245 ) Deferred tax liabilities $ (16,360 ) $ (23,245 ) Net deferred tax assets $ 261,300 $ 284,598 Valuation allowance (261,300 ) (284,598 ) Net deferred tax assets $ — $ — The net deferred tax assets (prior to applying the valuation allowance) of $261.3 million and $284.6 million at December 31, 2017 and 2016 , respectively, primarily consist of net operating loss carryforwards for income tax purposes. Due to the Company's history of operating losses, the Company recorded a full valuation allowance on its net deferred tax assets by decreasing the valuation allowance by $23.3 million in 2017 and increasing by $68.4 million in 2016 , respectively, as it was more likely than not that such tax benefits will not be realized. As of December 31, 2017, the Company's gross deferred tax assets were also impacted by the Tax Act which required the change to a 21% US tax rate (see below for further discussion on the Tax Act). At December 31, 2017 , the Company had federal net operating loss carryforwards for income tax purposes of approximately $721.8 million . Due to the limitation on NOLs as more fully discussed below, $544.0 million of the NOLs are available to offset future taxable income, if any. The NOL carryovers and general business tax credits expire in various years beginning in 2018. For state tax purposes, the Company has approximately $296.0 million of New Jersey NOLs available to offset against future taxable income. The Company also has California and Virginia NOLs that are entirely limited due to Section 382 (as discussed below), in addition to changing state apportionment allocations, as the Company is now 100% resident in New Jersey. During 2014, the Company completed an Internal Revenue Code Section 382 (Section 382) analysis in order to determine the amount of losses that are currently available for potential offset against future taxable income, if any. It was determined that the utilization of the Company's NOL and general business tax credit carryforwards generated in tax periods up to and including December 2010 were subject to substantial limitations under Section 382 due to ownership changes that occurred at various points from the Company's original organization through December 2010. In general, an ownership change, as defined by Section 382, results from transactions increasing the ownership of shareholders that own, directly or indirectly, 5% or more of a corporation's stock, in the stock of a corporation by more than 50 percentage points over a testing period (usually 3 years). Since the Company's formation, it has raised capital through the issuance of common stock on several occasions which, combined with the purchasing shareholders' subsequent disposition of those shares, resulted in multiple changes in ownership, as defined by Section 382 since the Company's formation in 1999. These ownership changes resulted in substantial limitations on the use of the Company's NOLs and general business tax credit carryforwards up to and including December 2010. The Company continues to track all of its NOLs and tax credit carryforwards but has provided a full valuation allowance to offset those amounts. On December 22, 2017, the US government enacted the Tax Act. The Tax Act significantly revises US tax law by, among other provisions, lowering the US federal statutory income tax rate from 35% to 21%, imposing a mandatory one-time transition tax on previously deferred foreign earnings, and eliminating or reducing certain income tax deductions. The Tax Act ASC 740, Income Taxes requires the effects of changes in tax laws to be recognized in the period in which the legislation is enacted. However, due to the complexity and significance of the Tax Act’s provisions, the SEC staff issued SAB 118, which allows companies to record the tax effects of the Tax Act on a provisional basis based on a reasonable estimate, and then, if necessary, subsequently adjust such amounts during a limited measurement period as more information becomes available. The measurement period ends when a company has obtained, prepared, and analyzed the information necessary to finalize its accounting, but cannot extend beyond one year from enactment. The Tax Act did not have a material impact on the Company's financial statements because its deferred temporary differences are fully offset by a valuation allowance and the Company does not have any significant offshore earnings from which to record the mandatory transition tax. However, given the significant complexity of the Tax Act, anticipated guidance from the US Treasury about implementing the Tax Act, and the potential for additional guidance from the SEC or the FASB related to the Tax Act, these estimates may be adjusted during the measurement period. The provisional amounts disclosed in our footnotes were based on the Company’s present interpretations of the Tax Act and current available information, including assumptions and expectations about future events, such as its projected financial performance, and are subject to further refinement as additional information becomes available (including the Company’s actual full year 2018 results of operations, as well as potential new or interpretative guidance issued by the FASB or the Internal Revenue Service and other tax agencies) and further analyses are completed. The Company continues to analyze the changes in certain income tax deductions, assess calculations of earnings and profits in certain foreign subsidiaries, including if those earnings are held in cash or other assets, and gather additional data to compute the full impacts on the Company’s deferred and current tax assets and liabilities. |
License and Other Agreements
License and Other Agreements | 12 Months Ended |
Dec. 31, 2017 | |
License and Other Agreements | |
License and Other Agreements | License and Other Agreements In-License Agreements PARI Pharma GmbH —In April 2008, the Company entered into a licensing agreement with PARI Pharma GmbH (PARI) for use of the optimized eFlow Nebulizer System for delivery of ALIS in treating patients with NTM lung infections, CF and bronchiectasis. Under the licensing agreement, the Company has rights under several US and foreign issued patents and patent applications involving improvements to the optimized eFlow Nebulizer System, to exploit such system with ALIS for the treatment of such indications, but the Company cannot manufacture such nebulizers except as permitted under the Commercialization Agreement with PARI. Under the licensing agreement, the Company paid PARI an upfront license fee and PARI is entitled to receive milestone payments up to an aggregate of €4.3 million either in cash, qualified stock or a combination of both, at PARI's discretion, based on achievement of certain future milestone events including first acceptance of MAA submission (or equivalent) in the US of ALIS and the device, first receipt of marketing approval in the US for ALIS and the device, and first receipt of marketing approval in a major EU country for ALIS and the device. In addition, PARI is entitled to receive royalty payments in the mid-single digits on the annual global net sales of ALIS, pursuant to the licensing agreement, subject to certain specified annual minimum royalties. In October 2017, the Company exercised an option to buy-down the future royalties that will be payable to PARI on ALIS net sales, if approved. The royalty buy-down will reduce the Company's future royalty payments due to PARI. The payment to PARI was included as a component of general and administrative expenses in the fourth quarter of 2017. See below for information related to the commercialization agreement with PARI. Other Agreements Cystic Fibrosis Foundation Therapeutics, Inc. —In 2004 and 2009, the Company entered into research funding agreements with Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT) whereby it received $1.7 million and $2.2 million for each respective agreement in research funding for the development of ALIS. If ALIS becomes an approved product for CF in the US, the Company will owe a payment to CFFT of up to $13.4 million that is payable over a three -year period after approval as a commercialized drug in the US. Furthermore, if certain global sales milestones are met within five years of the drug's commercialization, the Company would owe an additional payment of $3.9 million . Since there is significant development and regulatory risk associated with ALIS, including with respect to the CF indication, the Company has not accrued these obligations. Therapure Biopharma Inc. —In February 2014, the Company entered into a contract manufacturing agreement with Therapure Biopharma Inc. (Therapure) for the manufacture of ALIS at a 200 kg scale. Pursuant to the agreement, the Company and Therapure collaborated to construct a production area for the manufacture of ALIS in Therapure's existing manufacturing facility in Canada. Therapure manufactures ALIS for the Company on a non-exclusive basis. The agreement has an initial term of five years from the first date on which Therapure delivers ALIS to the Company after it obtains permits related to the manufacture of ALIS, and will renew automatically for successive periods of two years each, unless terminated by either party by providing the required two years' prior written notice to the other party. Notwithstanding the foregoing, the parties have rights and obligations under the agreement prior to the commencement of the initial term. Under the agreement, the Company is obligated to pay certain minimum amounts for the batches of ALIS produced each calendar year. Costs incurred under this agreement will be recorded as a component of research and development expense until such time as the Company receives regulatory approvals for ALIS. PARI Pharma GmbH —In July 2014, the Company entered into a Commercialization Agreement with PARI for the manufacture and supply of eFlow nebulizer systems and related accessories (the Device) as optimized for use with ALIS. Under the Commercialization Agreement, PARI manufactures the Device except in the case of certain defined supply failures, when the Company will have the right to make the Device and have it made by third parties (but not certain third parties deemed under the Commercialization Agreement to compete with PARI). The Commercialization Agreement has an initial term of fifteen years from the first commercial sale of ALIS pursuant to the licensing agreement (the Initial Term). The term of the agreement may be extended by the Company for an additional five years by providing written notice to PARI at least one year prior to the expiration of the Initial Term. Notwithstanding the foregoing, the parties have certain rights and obligations under the agreement prior to the commencement of the Initial Term. SynteractHCR, Inc. —In December 2014, the Company entered into a services agreement with SynteractHCR, Inc. (Synteract) pursuant to which the Company retained Synteract to perform implementation and management services in connection with the 212 study. The Company anticipates that aggregate costs relating to all work orders for the 212 study will be approximately $45 million over the period of the study. In April 2015, the Company entered into a work order with Synteract to perform implementation and management services for the 312 study. The Company anticipates that aggregate costs relating to all work orders for the 312 study will be approximately $25 million over the period of the study. Ajinomoto Althea, Inc. —In September 2015, the Company entered into a Commercial Fill/Finish Services Agreement (the Fill/Finish Agreement) with Ajinomoto Althea, Inc., a Delaware corporation (Althea), for Althea to produce, on a non-exclusive basis, ALIS in finished dosage form at a 50 kg scale. Under the Fill/Finish Agreement, the Company is obligated to pay a minimum of $2.7 million for the batches of ALIS produced by Althea each calendar year during the term of the Fill/Finish Agreement. The Fill/Finish Agreement became effective as of January 1, 2015, and had an initial term that was to end on December 31, 2017. In 2016, the Company signed an extension of the Fill/Finish Agreement through December 31, 2019, and it may be extended for additional two year periods upon mutual written agreement of the Company and Althea at least one year prior to the expiration of its then-current term. The Company has expensed at least the required minimum in each year of the contract. AstraZeneca —In October 2016, the Company entered into a license agreement (AZ License Agreement) with AstraZeneca AB, a Swedish corporation (AstraZeneca). Pursuant to the terms of the AZ License Agreement, AstraZeneca granted the Company exclusive global rights for the purpose of developing and commercializing AZD7986 (renamed INS 1007). In consideration of the licenses and other rights granted by AstraZeneca, the Company made an upfront payment of $30.0 million , which was included as research and development expense in the fourth quarter of 2016. The Company is also obligated to make a series of contingent milestone payments totaling up to an additional $85.0 million upon the achievement of clinical development and regulatory filing milestones. If the Company elects to develop INS1007 for a second indication, the Company will be obligated to make an additional series of contingent milestone payments to AstraZeneca totaling up to $42.5 million . The Company is not obligated to make any additional milestone payments for additional indications. In addition, the Company will pay AstraZeneca tiered royalties ranging from a high single-digit to mid-teen on net sales of any approved product based on INS1007 and one additional payment of $35.0 million upon the first achievement of $1.0 billion in annual net sales. The AZ License Agreement provides AstraZeneca with the option to negotiate a future agreement with the Company for commercialization of INS1007 in chronic obstructive pulmonary disease or asthma. Patheon UK Limited —In October 2017, the Company entered into certain agreements with Patheon UK Limited (Patheon) related to increasing its long-term production capacity for ALIS commercial inventory. The agreements provide for Patheon to manufacture and supply ALIS for its anticipated commercial needs. Under these agreements, the Company is required to deliver to Patheon the required raw materials, including active pharmaceutical ingredients, and certain fixed assets needed to manufacture ALIS. Patheon's supply obligations will commence once certain technology transfer and construction services are completed. The Company's manufacturing and supply agreement with Patheon will remain in effect for a fixed initial term, after which it will continue for successive renewal terms unless either we or Patheon have given written notice of termination. The technology transfer agreement will expire when the parties agree that the technology transfer services have been completed. The agreements may also be terminated under certain other circumstances, including by either party due to a material uncured breach of the other party or the other party’s insolvency. These early termination clauses may reduce the amounts due to the relevant parties. The investment in our long-term production capacity build-out, including under the Patheon agreements and related agreements or purchase orders with third parties for raw materials and fixed assets, is estimated to be approximately $60.0 million and will be incurred over the next three to four years. |
Commitments and Contingencies
Commitments and Contingencies | 12 Months Ended |
Dec. 31, 2017 | |
Commitments and Contingencies Disclosure [Abstract] | |
Commitments and Contingencies | Commitments and Contingencies Commitments The Company has an operating lease for office and laboratory space located in Bridgewater, NJ for which the initial lease term expires in November 2019. Future minimum rental payments under this lease are $2.0 million . In July 2016, the Company signed an operating lease for additional laboratory space located in Bridgewater, NJ for which the initial lease term expires in September 2021. Future minimum rental payments under this lease are $1.9 million . Rent expense charged to operations was $1.5 million , $1.2 million , and $0.8 million for the years ended December 31, 2017 , 2016 and 2015 , respectively. Rent expense is recorded on a straight-line basis over the term of the applicable leases. Future minimum rental cash payments required under the Company's operating leases as of December 31, 2017 are as follows (in thousands): Year Ending on December 31: 2018 $ 1,521 2019 1,421 2020 477 2021 498 2022 — $ 3,917 Legal Proceedings On July 15, 2016, a lawsuit captioned Hoey v. Insmed Incorporated, et al, No. 3:16-cv-04323-FLW-TJB (D.N.J. July 15, 2016) was filed in the US District Court for the District of New Jersey on behalf of a putative class of investors who purchased the Company’s common stock from March 18, 2013 through June 8, 2016. The complaint alleged that the Company and certain of its executives violated Sections 10(b) and 20(a) of the Securities Exchange Act of 1934 (Exchange Act) by misrepresenting and/or omitting the likelihood of the European Medicines Agency (EMA) approving the Company’s European marketing authorization application (MAA) for use of ALIS in the treatment of NTM lung disease and the likelihood of commercialization of ALIS in Europe. On October 25, 2016, the Court issued an order appointing Bucks County Employees Retirement Fund as lead plaintiff for the putative class. On December 15, 2016, the lead plaintiff filed an amended complaint that shortens the putative class period for the Exchange Act claims to March 26, 2014 through June 8, 2016 and adds claims under Sections 11, 12, and 15 of the Securities Act of 1933 (Securities Act) on behalf of a putative class of investors who purchased common stock in or traceable to the Company’s March 31, 2015 public offering. The amended complaint names as defendants in the Securities Act claims the Company, certain directors and officers, and the investment banks who served as underwriters in connection with the secondary offering. The amended complaint alleges defendants violated the Securities Act by using a purportedly misleading definition of “culture conversion” and supposedly failing to disclose in the offering materials purported flaws in its Phase 2 study that made the secondary offering risky or speculative. The amended complaint seeks damages in an unspecified amount. The Company moved to dismiss the amended complaint on March 1, 2017. The lead plaintiff opposed the motion on May 17, 2017 and the Company provided its reply brief on July 11, 2017. On July 20, 2017, the plaintiff asked for leave to file a sur-reply in further opposition to the Company’s motion to dismiss the amended complaint, which the Company had opposed. On February 15, 2018, the Court issued a decision granting the Company’s motion and dismissing the amended complaint without prejudice. The lead plaintiff has until March 19, 2018 to file a second amended complaint. If a second amended complaint is filed, the Company intends to continue to defend the lawsuit vigorously; however, there can be no assurance regarding the ultimate outcome of the lawsuit. From time to time, the Company is a party to various other lawsuits, claims and other legal proceedings that arise in the ordinary course of business. While the outcomes of these matters are uncertain, management does not expect that the ultimate costs to resolve these matters will have a material adverse effect on the Company’s consolidated financial position, results of operations or cash flows. |
Quarterly Financial Data (Unaud
Quarterly Financial Data (Unaudited) | 12 Months Ended |
Dec. 31, 2017 | |
Quarterly Financial Information Disclosure [Abstract] | |
Quarterly Financial Data (Unaudited) | Quarterly Financial Data (Unaudited) The following table summarizes unaudited quarterly financial data for the years ended December 31, 2017 and 2016 (in thousands, except per share data). 2017 First Quarter Second Quarter Third Quarter Fourth Quarter* Total* Revenues $ — $ — $ — $ — $ — Operating loss $ (35,969 ) $ (43,515 ) $ (44,083 ) $ (65,353 ) $ (188,920 ) Net loss $ (37,414 ) $ (44,672 ) $ (45,179 ) $ (65,384 ) $ (192,649 ) Basic and diluted net loss per share $ (0.60 ) $ (0.72 ) $ (0.69 ) $ (0.85 ) $ (2.89 ) 2016 First Quarter Second Quarter Third Quarter Fourth Quarter** Total** Revenues $ — $ — $ — $ — $ — Operating loss $ (33,067 ) $ (36,133 ) $ (37,149 ) $ (67,051 ) $ (173,400 ) Net loss $ (33,532 ) $ (36,579 ) $ (37,760 ) $ (68,402 ) $ (176,273 ) Basic and diluted net loss per share $ (0.54 ) $ (0.59 ) $ (0.61 ) $ (1.10 ) $ (2.85 ) ________________ * Includes a one-time payment in October 2017 related to the buy-down of future royalties payable to PARI on the global net sales of ALIS, if approved. ** Includes a $30.0 million upfront payment to AstraZeneca under the AZ License Agreement related to INS1007, which was included as a component of research and development expense. Basic and diluted net loss per share amounts included in the above table were computed independently for each of the quarters presented. Accordingly, the sum of the quarterly basic and diluted net loss per share amounts may not agree to the total for the year. |
Retirement Plan
Retirement Plan | 12 Months Ended |
Dec. 31, 2017 | |
Retirement Benefits [Abstract] | |
Retirement Plan | Retirement Plan The Company has a 401(k) defined contribution plan for the benefit for all US employees and permits voluntary contributions by employees subject to IRS-imposed limitations. Beginning in April 2015, the Company matched 100% of eligible employee contributions on the first 3% of employee salary (up to the IRS maximum). Employer contributions for the year ended December 31, 2017 , 2016 and 2015 were $0.8 million , $0.6 million and $0.4 million , respectively. Effective January 1, 2018, the Company is matching 100% of eligible employee contributions on the first 4% of employee salary (up to the IRS maximum). |
Subsequent Event
Subsequent Event | 12 Months Ended |
Dec. 31, 2017 | |
Subsequent Events [Abstract] | |
Subsequent Event | Subsequent Event In January 2018, the Company completed an underwritten public offering of 1.75% convertible senior notes due 2025 (the Convertible Notes) pursuant to an indenture dated as of January 26, 2018, between the Company and Wells Fargo Bank, National Association (Wells Fargo), as trustee, as supplemented by the first supplemental indenture, dated January 26, 2018 between the Company and Wells Fargo (as supplemented, the Indenture). The Company sold $450.0 million aggregate principal amount of Convertible Notes, including the exercise in full of the underwriters' option to purchase additional Convertible Notes of $50.0 million . The Company's net proceeds from the offering, after deducting underwriting discounts and commissions and other offering expenses of $14.2 million , were approximately $435.8 million . The Company is currently evaluating the accounting for the debt and equity components of the offering. The Convertible Notes bear interest payable semiannually in arrears on January 15 and July 15 of each year, beginning on July 15, 2018. The Convertible Notes mature on January 15, 2025, unless earlier converted, redeemed, or repurchased. The Convertible Notes are convertible into common shares of the Company under certain circumstances described in the Indenture. The initial conversion rate is 25.5384 shares of common stock per $1,000 principal amount of Convertible Notes (equivalent to an initial conversion price of approximately $39.16 per share of common stock). The conversion rate will be subject to adjustment in some events but will not be adjusted for any accrued and unpaid interest. |
Summary of Significant Accoun21
Summary of Significant Accounting Policies (Policies) | 12 Months Ended |
Dec. 31, 2017 | |
Accounting Policies [Abstract] | |
Basis of Presentation | Basis of Presentation —The consolidated financial statements include the accounts of the Company and its wholly-owned subsidiaries, Insmed Limited, Celtrix Pharmaceuticals, Inc., Insmed Holdings Limited, Insmed Ireland Limited, Insmed France SAS, Insmed Germany GmbH, Insmed Netherlands B.V. and Insmed Godo Kaisha. All intercompany transactions and balances have been eliminated in consolidation. |
Use of Estimates | Use of Estimates —The preparation of the consolidated financial statements in conformity with accounting principles generally accepted in the United States (GAAP) requires management to make estimates and assumptions that affect the amounts reported in the consolidated financial statements and accompanying notes. The Company bases its estimates and judgments on historical experience and on various other assumptions. The amounts of assets and liabilities reported in the Company's balance sheets and the amounts of expenses reported for each period presented are affected by estimates and assumptions, which are used for, but not limited to, the accounting for stock-based compensation, income taxes, loss contingencies, and accounting for research and development costs. Actual results could differ from those estimates. |
Investment Income and Interest Expense | Investment Income and Interest Expense —Investment income consists of interest and dividend income earned on the Company's cash and cash equivalents. Interest expense consists primarily of interest costs related to the Company's debt. |
Cash and Cash Equivalents | Cash and Cash Equivalents —The Company considers cash equivalents to be highly liquid investments with maturities of three months or less from the date of purchase. |
Fixed Assets, Net | Fixed Assets, Net —Fixed assets are recorded at cost and are depreciated on a straight-line basis over the estimated useful lives of the assets. Estimated useful lives of three years to five years are used for computer equipment. Estimated useful lives of seven years are used for laboratory equipment, office equipment, manufacturing equipment and furniture and fixtures. Leasehold improvements are amortized over the shorter of the lease term or the estimated useful life of the asset. Long-lived assets are reviewed for impairment whenever events or changes in circumstances indicate that the carrying amount of an asset may not be recoverable. Recoverability of assets to be held and used is measured by a comparison of the carrying amount of an asset to estimated undiscounted future cash flows expected to be generated by the asset. If the carrying amount of an asset exceeds its estimated future cash flows, then an impairment charge is recognized for the amount by which the carrying value of the asset exceeds the fair value of the asset. |
Identifiable Intangible Assets | Identifiable Intangible Assets —Identifiable intangible assets are measured at their respective fair values and are not amortized until commercialization. Once commercialization occurs, these intangible assets will be amortized over their estimated useful lives. The fair values assigned to the Company's intangible assets are based on reasonable estimates and assumptions given available facts and circumstances. Unanticipated events or circumstances may occur that may require the Company to review the assets for impairment. Events or circumstances that may require an impairment assessment include negative clinical trial results, the non-approval of a new drug application by a regulatory agency, material delays in the Company's development program or a sustained decline in market capitalization. Indefinite-lived intangible assets are not subject to periodic amortization. Rather, indefinite-lived intangibles are reviewed for impairment by applying a fair value based test on an annual basis or more frequently if events or circumstances indicate impairment may have occurred. Events or circumstances that may require an interim impairment assessment are consistent with those described above. The Company performs its annual impairment test as of October 1 of each year. The Company uses the income approach to derive the fair value of in-process research and development assets. This approach calculates fair value by estimating future cash flows attributable to the assets and then discounting these cash flows to a present value using a risk-adjusted discount rate. This approach requires significant management judgment with respect to unobservable inputs such as future volume, revenue and expense growth rates, changes in working capital use, appropriate discount rates and other assumptions and estimates. The estimates and assumptions used are consistent with the Company's business plans. A market based valuation approach was not considered given a lack of revenues and profits for the Company. |
Debt Issuance Costs | Debt Issuance Costs —Debt issuance costs are amortized to interest expense using the effective interest rate method over the term of the debt. Debt issuance costs paid to the lender and third parties are reflected as a discount to the debt in the consolidated balance sheets. Unamortized debt issuance costs associated with extinguished debt are expensed in the period of the extinguishment. |
Fair Value Measurements | Fair Value Measurements —The Company categorizes its financial assets and liabilities measured and reported at fair value in the financial statements on a recurring basis based upon the level of judgments associated with the inputs used to measure their fair value. Hierarchical levels, which are directly related to the amount of subjectivity associated with the inputs used to determine the fair value of financial assets and liabilities, are as follows: • Level 1—Inputs are unadjusted, quoted prices in active markets for identical assets or liabilities at the measurement date. • Level 2—Inputs (other than quoted prices included in Level 1) are either directly or indirectly observable for the assets or liability through correlation with market data at the measurement date and for the duration of the instrument's anticipated life. • Level 3—Inputs reflect management's best estimate of what market participants would use in pricing the asset or liability at the measurement date. Consideration is given to the risk inherent in the valuation technique and the risk inherent in the inputs to the model. Each major category of financial assets and liabilities measured at fair value on a recurring basis is categorized based upon the lowest level of significant input to the valuations. The fair value hierarchy also requires an entity to maximize the use of observable inputs and minimize the use of unobservable inputs when measuring fair value. Financial instruments in Level 1 generally include US treasuries and mutual funds listed in active markets. The Company's only assets and liabilities which were measured at fair value as of December 31, 2017 and December 31, 2016 were its cash and cash equivalents of $381.2 million and $162.6 million , respectively. These amounts were measured at Level 1 using quoted prices in active markets for identical assets at the measurement date. The Company's cash and cash equivalents permit daily redemption and the fair values of these investments are based upon the quoted prices in active markets provided by the holding financial institutions. Cash equivalents consist of liquid investments with a maturity of three months or less from the date of purchase and the short-term investments consist of instruments with maturities greater than three months. The Company recognizes transfers between levels within the fair value hierarchy, if any, at the end of each quarter. There were no transfers in or out of Level 1, Level 2 or Level 3 during 2017 and 2016 . As of December 31, 2017 and 2016 , the Company held no securities that were in an unrealized loss or gain position. The Company reviews the status of each security quarterly to determine whether an other-than-temporary impairment has occurred. In making its determination, the Company considers a number of factors, including: (1) the significance of the decline; (2) whether the securities were rated below investment grade; (3) how long the securities have been in an unrealized loss position; and (4) the Company's ability and intent to retain the investment for a sufficient period of time for it to recover. |
Foreign currency | Foreign Currency —The Company has operations in the US, Ireland, Germany, France, the UK and the Netherlands. The results of its non-US dollar based functional currency operations are translated to US dollars at the average exchange rates during the period. Assets and liabilities are translated at the exchange rate prevailing at the balance sheet date. Equity is translated at the prevailing exchange rate at the date of the equity transaction. Translation adjustments are included in shareholders' equity, as a component of other comprehensive loss. The Company realizes foreign currency transaction gains (losses) in the normal course of business based on movements in the applicable exchange rates. These gains (losses) are included as a component of other income (expense), net. |
Concentration of Credit Risk | Concentration of Credit Risk —Financial instruments that potentially subject the Company to concentrations of credit risk consist primarily of cash and cash equivalents. The Company places its cash equivalents with high credit-quality financial institutions and may invest its short-term investments in US treasury securities, mutual funds and government agency bonds. The Company has established guidelines relative to credit ratings and maturities that seek to maintain safety and liquidity. The Company relies on third-party manufacturers and suppliers for manufacturing and supply of its products. The inability of the suppliers or manufacturers to fulfill supply requirements of the Company could materially impact future operating results. A change in the relationship with the suppliers or manufacturer, or an adverse change in their business, could materially impact future operating results. |
Other Income | Other Income —In 2015, the French National Agency for Medicines and Health Products Safety (ANSM) granted ALIS a Temporary Authorizations for Use (Autorisation Temporaire d'Utilisation or ATU). Pursuant to this program, the Company shipped product to pharmacies after receiving requests from physicians for patients in France. For the years ended December 31, 2017 , 2016 and 2015 , the revenue recorded was immaterial and is included as a component of other income (expense), net. The Company is initiating expanded access programs (EAPs) in other select territories in Europe, some of which may be fully reimbursed. EAPs are intended to make products available on a named patient basis before they are commercially available in accordance with local regulations. |
Research and Development | Research and Development —Research and development expenses consist primarily of salaries, benefits and other related costs, including stock-based compensation, for personnel serving in the Company's research and development functions, and other internal operating expenses, the cost of manufacturing a drug candidate, including the medical devices for drug delivery, for clinical study, the cost of conducting clinical studies, and the cost of conducting preclinical and research activities. In addition, research and development expenses include payments to third parties for the license rights to products in development (prior to marketing approval). The Company's expenses related to manufacturing its drug candidate and medical devices for clinical study are primarily related to activities at contract manufacturing organizations that manufacture ALIS, INS1007, and INS1009 and the medical devices for the Company's use. The Company's expenses related to clinical trials are primarily related to activities at contract research organizations that conduct and manage clinical trials on the Company's behalf. These contracts set forth the scope of work to be completed at a fixed fee or amount per patient enrolled. Payments under these contracts primarily depend on performance criteria such as the successful enrollment of patients or the completion of clinical trial milestones as well as time-based fees. Expenses are accrued based on contracted amounts applied to the level of patient enrollment and to activity according to the clinical trial protocol. Nonrefundable advance payments for goods or services that will be used or rendered for future research and development activities are deferred and capitalized. Such amounts are then recognized as an expense as the related goods are delivered or the services are performed, or when the goods or services are no longer expected to be provided. |
Stock-Based Compensation | Stock-Based Compensation —The Company recognizes stock-based compensation expense for awards of equity instruments to employees and directors based on the grant-date fair value of those awards. The grant-date fair value of the award is recognized as compensation expense ratably over the requisite service period, which generally equals the vesting period of the award, and if applicable, is adjusted for expected forfeitures. The Company also grants performance-based stock options to employees. The grant-date fair value of the performance-based stock options is recognized as compensation expense over the implicit service period using the accelerated attribution method once it is probable that the performance condition will be achieved. Stock-based compensation expense is included in both research and development expenses and general and administrative expenses in the Consolidated Statements of Comprehensive Loss. |
Income Taxes | Income Taxes —The Company accounts for income taxes under the asset and liability method. Deferred tax assets and liabilities are recognized for the future tax consequences attributable to differences between the financial statement carrying amounts of existing assets and liabilities and their respective tax bases and operating loss carry forwards. Deferred tax assets and liabilities are measured using enacted tax rates expected to apply to taxable income in the years in which those temporary differences are expected to be recovered or settled. The effect on deferred tax assets and liabilities of a change in tax rates is recognized in income in the period that includes the enactment date. A valuation allowance is recorded to reduce the deferred tax assets to the amount that is expected to be realized. In evaluating the need for a valuation allowance, the Company takes into account various factors, including the expected level of future taxable income and available tax planning strategies. If actual results differ from the assumptions made in the evaluation of a valuation allowance, the Company records a change in valuation allowance through income tax expense in the period such determination is made. The Company uses a comprehensive model for how it measures, presents and discloses an uncertain tax position taken or expected to be taken in a tax return. The Company may recognize the tax benefit from an uncertain tax position only if it is more likely than not that the tax position will be sustained on examination by taxing authorities, based solely on the technical merits of the position. The tax benefits recognized in the financial statements from such a position should be measured based on the largest benefit that has a greater than 50% likelihood to be sustained upon ultimate settlement. The Company had no uncertain tax positions as of December 31, 2017 and 2016 that qualified for either recognition or disclosure in the consolidated financial statements. The Company's policy for interest and penalties related to income tax exposures is to recognize interest and penalties as a component of the income tax (benefit) provision in the Consolidated Statements of Comprehensive Loss. Tax Cuts and Jobs Act On December 22, 2017, the US government enacted comprehensive tax legislation, referred to as the Tax Cuts and Jobs Act (the Tax Act). The Tax Act significantly revises US tax law by, among other provisions, lowering the US federal statutory income tax rate from 35% to 21%, imposing a mandatory one-time transition tax on previously deferred foreign earnings, and eliminating or reducing certain income tax deductions. ASC 740, Income Taxes requires the effects of changes in tax laws to be recognized in the period in which the legislation is enacted. However, due to the complexity and significance of the Tax Act’s provisions, the SEC staff issued Staff Accounting Bulletin No. 118 (SAB 118), which allows companies to record the tax effects of the Tax Act on a provisional basis based on a reasonable estimate, and then, if necessary, subsequently adjust such amounts during a limited measurement period as more information becomes available. The measurement period ends when a company has obtained, prepared, and analyzed the information necessary to finalize its accounting, but cannot extend beyond one year from enactment. The provisional amounts recorded for the Tax Act did not have a material impact on the Company's financial statements as of December 31, 2017, because its deferred temporary differences are fully offset by a valuation allowance and the Company does not have any significant offshore earnings from which to record the mandatory transition tax. However, given the significant complexity of the Tax Act, anticipated guidance from the US Treasury about implementing the Tax Act, and the potential for additional guidance from the SEC or the FASB related to the Tax Act, these estimates may be adjusted during the measurement period. The provisional amounts disclosed in our footnotes were based on the Company’s present interpretations of the Tax Act and current available information, including assumptions and expectations about future events, such as its projected financial performance, and are subject to further refinement as additional information becomes available (including the Company’s actual full Fiscal 2018 results of operations, as well as potential new or interpretative guidance issued by the FASB or the Internal Revenue Service and other tax agencies) and further analyses are completed. The Company continues to analyze the changes in certain income tax deductions, assess calculations of earnings and profits in certain foreign subsidiaries, including if those earnings are held in cash or other assets and gather additional data to compute the full impacts on the Company’s deferred and current tax assets and liabilities. |
Net Loss Per Common Share | Net Loss Per Common Share —Basic net loss per common share is computed by dividing net loss attributable to common shareholders by the weighted average number of common shares outstanding during the period. Diluted net loss per common share is computed by dividing net loss by the weighted average number of common shares and other dilutive securities outstanding during the period. Potentially dilutive securities from stock options and restricted stock units would be anti-dilutive as the Company incurred a net loss in all periods presented. Potentially dilutive common shares resulting from the assumed exercise of outstanding stock options are determined based on the treasury stock method. The following table sets forth the reconciliation of the weighted average number of shares used to compute basic and diluted net loss per share for the years ended December 31, 2017 , 2016 and 2015 . Years Ended December 31, 2017 2016 2015 (in thousands, except per share amounts) Numerator: Net loss $ (192,649 ) $ (176,273 ) $ (118,183 ) Denominator: Weighted average common shares used in calculation of basic net loss per share: 66,576 61,892 58,633 Effect of dilutive securities: Common stock options — — — Restricted stock and restricted stock units — — — Weighted average common shares outstanding used in calculation of diluted net loss per share 66,576 61,892 58,633 Net loss per share: Basic and Diluted $ (2.89 ) $ (2.85 ) $ (2.02 ) The following potentially dilutive securities have been excluded from the computations of diluted weighted average common shares outstanding as of December 31, 2017 , 2016 and 2015 as their effect would have been anti-dilutive (in thousands). 2017 2016 2015 Stock options to purchase common stock 8,609 7,117 5,274 Restricted stock and restricted stock units 47 89 44 |
Segment Information | Segment Information —The Company currently operates in one business segment, which is the development and commercialization of therapies for patients with rare diseases. A single management team that reports to the Chief Executive Officer comprehensively manages the entire business. The Company does not operate separate lines of business with respect to its products or product candidates. Accordingly, the Company does not have separate reportable segments. |
New Accounting Pronouncements | New Accounting Pronouncements (Adopted) —In August 2014, the Financial Accounting Standards Board (FASB) issued Accounting Standards Update (ASU) No. 2014-15, Presentation of Financial Statements—Going Concern: Disclosure of Uncertainties about an Entity's Ability to Continue as a Going Concern, which requires management to evaluate whether there is substantial doubt about the entity's ability to continue as a going concern and, if so, provide certain footnote disclosures. This ASU was effective for the annual period ended December 31, 2016, and interim reporting periods thereafter. The adoption of this standard did not have an impact on the Company's consolidated financial statements and related footnote disclosures. In March 2016, the FASB issued ASU 2016-9, Improvements to Employee Share-Based Payment Accounting , which amends ASC Topic 718, Compensation—Stock Compensation . ASU 2016-9 simplifies several aspects of the accounting for share-based payment transactions, including the income tax consequences, classification of awards as either equity or liabilities, and classification on the statement of cash flows. ASU 2016-9 is effective for fiscal years beginning after December 15, 2016, and interim periods within those fiscal years. The Company adopted ASU 2016-9 in the first quarter of 2017. ASU 2016-9 did not have a material impact on its consolidated financial statements. New Accounting Pronouncements (Not Yet Adopted) —In May 2014, the FASB issued ASU 2014-9, Revenue from Contracts with Customers (Topic 606) which amended the existing accounting standards for revenue recognition. ASU 2014-9 establishes principles for recognizing revenue upon the transfer of promised goods or services to customers, in an amount that reflects the expected consideration received in exchange for those goods or services. In July 2015, the FASB deferred the effective date for annual reporting periods beginning after December 15, 2017. The Company will adopt ASU 2014-9 in the first quarter of 2018 and expects the impact of adoption will not be material to its consolidated financial statements. In February 2016, the FASB issued ASU 2016-2, Leases (Topic 842) in order to increase transparency and comparability among organizations by recognizing lease assets and lease liabilities on the balance sheet for those leases classified as operating leases under previous GAAP. ASU 2016-2 requires that a lessee should recognize a liability to make lease payments (the lease liability) and a right-of-use asset representing its right to use the underlying asset for the lease term on the balance sheet. ASU 2016-2 is effective for fiscal years beginning after December 15, 2018 (including interim periods within those periods) using a modified retrospective approach and early adoption is permitted. The Company expects to adopt ASU 2016-2 in the first quarter of 2019 and is in the process of evaluating the impact of adoption on its consolidated financial statements. |
Summary of Significant Accoun22
Summary of Significant Accounting Policies (Tables) | 12 Months Ended |
Dec. 31, 2017 | |
Accounting Policies [Abstract] | |
Reconciliation of the Weighted Average Number of Shares Used to Compute Basic and Diluted Net Loss per Share | The following table sets forth the reconciliation of the weighted average number of shares used to compute basic and diluted net loss per share for the years ended December 31, 2017 , 2016 and 2015 . Years Ended December 31, 2017 2016 2015 (in thousands, except per share amounts) Numerator: Net loss $ (192,649 ) $ (176,273 ) $ (118,183 ) Denominator: Weighted average common shares used in calculation of basic net loss per share: 66,576 61,892 58,633 Effect of dilutive securities: Common stock options — — — Restricted stock and restricted stock units — — — Weighted average common shares outstanding used in calculation of diluted net loss per share 66,576 61,892 58,633 Net loss per share: Basic and Diluted $ (2.89 ) $ (2.85 ) $ (2.02 ) |
Potentially Dilutive Securities Excluded from Computations of Diluted Weighted Average Common Shares Outstanding | The following potentially dilutive securities have been excluded from the computations of diluted weighted average common shares outstanding as of December 31, 2017 , 2016 and 2015 as their effect would have been anti-dilutive (in thousands). 2017 2016 2015 Stock options to purchase common stock 8,609 7,117 5,274 Restricted stock and restricted stock units 47 89 44 |
Accrued Expenses (Tables)
Accrued Expenses (Tables) | 12 Months Ended |
Dec. 31, 2017 | |
Payables and Accruals [Abstract] | |
Accrued Expenses | Accrued expenses consist of the following: As of December 31, 2017 2016 (in thousands) Accrued clinical trial expenses $ 7,837 $ 6,683 Accrued compensation 12,197 6,937 Accrued professional fees 4,500 1,992 Accrued technical operation expenses 2,182 591 Accrued interest payable 423 438 Accrued construction costs 1,719 — Other accrued expenses 481 181 $ 29,339 $ 16,822 |
Fixed Assets, net (Tables)
Fixed Assets, net (Tables) | 12 Months Ended |
Dec. 31, 2017 | |
Property, Plant and Equipment [Abstract] | |
Schedule of Fixed Assets | Fixed assets are stated at cost and depreciated using the straight-line method, based on useful lives as follows: Estimated Useful Life (years) As of December 31, Asset Description 2017 2016 (in thousands) Lab equipment 7 $ 7,055 $ 5,662 Furniture and fixtures 7 1,937 1,903 Computer hardware and software 3 - 5 2,325 2,251 Office equipment 7 65 65 Manufacturing equipment 7 1,436 1,148 Leasehold improvements lease term 6,939 6,735 Construction in Progress (CIP) — 3,320 — 23,077 17,764 Less accumulated depreciation (10,645 ) (7,744 ) Fixed assets, net $ 12,432 $ 10,020 |
Debt (Tables)
Debt (Tables) | 12 Months Ended |
Dec. 31, 2017 | |
Debt Disclosure [Abstract] | |
Components of Debt Balance | The following table presents the components of the Company's debt balance as of December 31, 2017 (in thousands): Debt: Note payable under A&R Loan Agreement $ 55,000 Accretion of end of term charge 828 Issuance fees paid to lender (261 ) Current portion of long-term debt — Long-term debt $ 55,567 |
Schedule of Future Principal Repayments of Debt | Future principal repayments of the Company's long-term debt are as follows (in thousands): Year Ending in December 31: 2018 $ — 2019 13,399 2020 41,601 $ 55,000 |
Stock-Based Compensation (Table
Stock-Based Compensation (Tables) | 12 Months Ended |
Dec. 31, 2017 | |
Share-based Compensation [Abstract] | |
Summary of Fair Value Assumptions for Stock Options | The following table summarizes the grant date fair value and assumptions used in determining the fair value of all stock options granted, including grants of inducement options, during the years ended December 31, 2017 , 2016 and 2015 . 2017 2016 2015 Volatility 71% - 79% 74% - 77% 78% - 82% Risk-free interest rate 1.73% - 2.13% 1.00% - 1.90% 1.31% - 1.75% Dividend yield 0.0% 0.0% 0.0% Expected option term (in years) 6.25 6.25 6.25 Weighted average fair value of stock options granted $10.52 $8.77 $14.20 |
Summary of Stock Option Activity | The following table summarizes stock option activity for stock options granted for the years ended December 31, 2017 , 2016 and 2015 as follows: Number of Shares Weighted Average Exercise Price Weighted Average Remaining Contractual Life in Years Aggregate Intrinsic Value (in '000) Options outstanding at January 1, 2015 4,400,106 $ 10.59 Granted 1,902,850 20.45 Exercised (481,140 ) 10.62 Forfeited and expired (548,094 ) 15.43 Options outstanding at December 31, 2015 5,273,722 $ 13.64 Vested and expected to vest at December 31, 2015 5,059,645 13.46 Exercisable at December 31, 2015 1,991,141 8.70 Options outstanding at December 31, 2015 5,273,722 $ 13.64 Granted 2,532,675 12.96 Exercised (162,340 ) 6.68 Forfeited and expired (527,351 ) 17.08 Options outstanding at December 31, 2016 7,116,706 $ 13.30 Vested and expected to vest at December 31, 2016 6,850,658 13.25 Exercisable at December 31, 2016 3,113,998 11.28 Options outstanding at December 31, 2016 7,116,706 $ 13.30 Granted 2,284,710 15.92 Exercised (378,275 ) 9.08 Forfeited and expired (414,220 ) 15.50 Options outstanding at December 31, 2017 8,608,921 $ 14.08 7.4 $ 147,260 Vested and expected to vest at December 31, 2017 8,325,255 $ 14.03 7.4 $ 142,783 Exercisable at December 31, 2017 4,229,478 $ 12.71 6.3 $ 78,138 |
Summary of Exercise Price and Number of Stock Options Exercisable | The following table summarizes the range of exercise prices and the number of stock options outstanding and exercisable as of December 31, 2017 : Outstanding as of December 31, 2017 Exercisable as of Range of Exercise Prices Number of Options Weighted Average Remaining Contractual Term (in years) Weighted Average Exercise Price Number of Options Weighted Average Exercise Price $ 3.03 $ 4.55 973,195 4.65 $ 3.60 973,195 $ 3.60 $ 6.90 $ 6.90 137,577 5.22 $ 6.90 100,077 $ 6.90 $ 6.96 $ 10.85 1,077,621 8.29 $ 10.76 420,374 $ 10.62 $ 11.14 $ 12.58 1,088,035 6.39 $ 12.17 781,991 $ 12.18 $ 12.66 $ 13.67 1,043,273 8.74 $ 13.59 101,126 $ 13.26 $ 13.94 $ 15.91 862,300 7.76 $ 14.97 406,441 $ 14.53 $ 16.07 $ 16.16 1,008,750 7.69 $ 16.13 463,629 $ 16.12 $ 16.35 $ 17.24 868,650 9.24 $ 17.13 23,999 $ 16.85 $ 17.36 $ 22.76 1,415,700 7.14 $ 21.18 927,334 $ 21.23 $ 22.84 $ 31.78 133,820 8.86 $ 27.47 31,312 $ 23.74 |
Summary of RSU Activity | The following table summarizes RSU awards granted during the years ended December 31, 2017 , 2016 and 2015 : Number of RSUs Weighted Average Grant Price Outstanding at January 1, 2015 20,502 $ 19.47 Granted 49,776 16.07 Released (26,724 ) (18.68 ) Forfeited — — Outstanding at December 31, 2015 43,554 $ 19.47 Granted 89,194 10.85 Released (43,554 ) (16.07 ) Forfeited — — Outstanding at December 31, 2016 89,194 $ 10.85 Granted 46,914 17.16 Released (89,194 ) (10.85 ) Forfeited — — Outstanding at December 31, 2017 46,914 $ 17.16 |
Summary of Allocation of Employee Stock-Based Compensation | The following table summarizes the stock-based compensation recorded in the Consolidated Statements of Comprehensive Loss related to stock options and RSUs during the years ended December 31, 2017 , 2016 and 2015 : 2017 2016 2015 (in millions) Research and development expenses $ 6.5 $ 6.2 $ 4.0 General and administrative expenses 11.6 11.8 11.6 Total (1) $ 18.1 $ 18.0 $ 15.6 _________________________________ (1) Includes $0.0 million , $1.7 million and $2.3 million for the years ended December 31, 2017 , 2016 and 2015 , respectively, for the remeasurement of certain stock options and RSUs that occurred during May 2013. |
Income Taxes (Tables)
Income Taxes (Tables) | 12 Months Ended |
Dec. 31, 2017 | |
Income Tax Disclosure [Abstract] | |
Schedule of Loss Before Income Taxes in the US and Globally | The Company's loss before income taxes in the US and globally was as follows (in thousands): Years Ended December 31, 2017 2016 2015 US $ (136,682 ) $ (140,354 ) $ (100,278 ) Foreign (56,239 ) (35,821 ) (19,876 ) Total $ (192,921 ) $ (176,175 ) $ (120,154 ) |
Schedule of Components Income Tax (Benefit) Provision | The Company's income tax (benefit) provision consisted of the following (in thousands): Years Ended December 31, 2017 2016 2015 Current: Federal $ — $ — $ — State 3 3 (2,015 ) Foreign 142 95 44 145 98 (1,971 ) Deferred: Federal (417 ) — — State — — — Foreign — — — (417 ) — — Total $ (272 ) $ 98 $ (1,971 ) |
Reconciliation Between Federal Statutory Tax Rate and Effective Tax Rate | The reconciliation between the federal statutory tax rate of 34% and the Company's effective tax rate is as follows: Years Ended December 31, 2017 2016 2015 Statutory federal tax rate 34 % 34 % 34 % Permanent items (3 )% (3 )% (4 )% State income taxes, net of federal benefit 4 % 4 % 4 % R&D and other tax credits 8 % 8 % 12 % Foreign income taxes (6 )% (4 )% (1 )% Impact of 2017 Tax Act (49 )% — % — % Change in valuation allowance 12 % (39 )% (43 )% Other — % — % — % Effective tax rate — % — % 2 % |
Schedule of Components of Deferred Tax Assets and Liabilities | The components of the deferred tax assets and liabilities consist of the following: As of December 31, 2017 2016 Deferred tax assets: Net operating loss carryforwards $ 186,342 $ 228,729 General business credits 66,371 50,648 Product license 7,730 11,783 Alternative minimum tax (AMT) credit — 418 Other 17,217 16,265 Gross deferred tax assets $ 277,660 $ 307,843 Deferred tax liabilities: In-process research and development $ (16,360 ) $ (23,245 ) Deferred tax liabilities $ (16,360 ) $ (23,245 ) Net deferred tax assets $ 261,300 $ 284,598 Valuation allowance (261,300 ) (284,598 ) Net deferred tax assets $ — $ — |
Commitments and Contingencies (
Commitments and Contingencies (Tables) | 12 Months Ended |
Dec. 31, 2017 | |
Commitments and Contingencies Disclosure [Abstract] | |
Schedule of Future Minimum Rental Payments Required Under Operating Leases | Future minimum rental cash payments required under the Company's operating leases as of December 31, 2017 are as follows (in thousands): Year Ending on December 31: 2018 $ 1,521 2019 1,421 2020 477 2021 498 2022 — $ 3,917 |
Quarterly Financial Data (Una29
Quarterly Financial Data (Unaudited) (Tables) | 12 Months Ended |
Dec. 31, 2017 | |
Quarterly Financial Information Disclosure [Abstract] | |
Summary of Quarterly Financial Data (Unaudited) | The following table summarizes unaudited quarterly financial data for the years ended December 31, 2017 and 2016 (in thousands, except per share data). 2017 First Quarter Second Quarter Third Quarter Fourth Quarter* Total* Revenues $ — $ — $ — $ — $ — Operating loss $ (35,969 ) $ (43,515 ) $ (44,083 ) $ (65,353 ) $ (188,920 ) Net loss $ (37,414 ) $ (44,672 ) $ (45,179 ) $ (65,384 ) $ (192,649 ) Basic and diluted net loss per share $ (0.60 ) $ (0.72 ) $ (0.69 ) $ (0.85 ) $ (2.89 ) 2016 First Quarter Second Quarter Third Quarter Fourth Quarter** Total** Revenues $ — $ — $ — $ — $ — Operating loss $ (33,067 ) $ (36,133 ) $ (37,149 ) $ (67,051 ) $ (173,400 ) Net loss $ (33,532 ) $ (36,579 ) $ (37,760 ) $ (68,402 ) $ (176,273 ) Basic and diluted net loss per share $ (0.54 ) $ (0.59 ) $ (0.61 ) $ (1.10 ) $ (2.85 ) ________________ * Includes a one-time payment in October 2017 related to the buy-down of future royalties payable to PARI on the global net sales of ALIS, if approved. ** Includes a $30.0 million upfront payment to AstraZeneca under the AZ License Agreement related to INS1007, which was included as a component of research and development expense. |
Summary of Significant Accoun30
Summary of Significant Accounting Policies - Fixed Assets, Net (Details) | 12 Months Ended |
Dec. 31, 2017 | |
Computer equipment | Minimum | |
Fixed Assets, Net | |
Estimated useful live of fixed assets | 3 years |
Computer equipment | Maximum | |
Fixed Assets, Net | |
Estimated useful live of fixed assets | 5 years |
Laboratory equipment, office equipment, manufacturing equipment and furniture and fixtures | |
Fixed Assets, Net | |
Estimated useful live of fixed assets | 7 years |
Summary of Significant Accoun31
Summary of Significant Accounting Policies - Fair Value Measurements (Details) $ in Millions | Dec. 31, 2017USD ($)security | Dec. 31, 2016USD ($)security |
Accounting Policies [Abstract] | ||
Cash and cash equivalents | $ | $ 381.2 | $ 162.6 |
Securities in an unrealized gain or loss position | security | 0 | 0 |
Summary of Significant Accoun32
Summary of Significant Accounting Policies - Income Taxes (Details) - USD ($) | Dec. 31, 2017 | Dec. 31, 2016 |
Accounting Policies [Abstract] | ||
Uncertain tax positions | $ 0 | $ 0 |
Summary of Significant Accoun33
Summary of Significant Accounting Policies - Net Loss Per Common Share (Details) - USD ($) $ / shares in Units, shares in Thousands, $ in Thousands | 3 Months Ended | 12 Months Ended | |||||||||
Dec. 31, 2017 | Sep. 30, 2017 | Jun. 30, 2017 | Mar. 31, 2017 | Dec. 31, 2016 | Sep. 30, 2016 | Jun. 30, 2016 | Mar. 31, 2016 | Dec. 31, 2017 | Dec. 31, 2016 | Dec. 31, 2015 | |
Numerator: | |||||||||||
Net loss | $ (65,384) | $ (45,179) | $ (44,672) | $ (37,414) | $ (68,402) | $ (37,760) | $ (36,579) | $ (33,532) | $ (192,649) | $ (176,273) | $ (118,183) |
Denominator: | |||||||||||
Weighted average common shares used in calculation of basic net loss per share (in shares) | 66,576 | 61,892 | 58,633 | ||||||||
Effect of dilutive securities: | |||||||||||
Weighted average common shares outstanding used in calculation of diluted net loss per share (in shares) | 66,576 | 61,892 | 58,633 | ||||||||
Net loss per share: | |||||||||||
Basic and Diluted (in dollars per share) | $ (0.85) | $ (0.69) | $ (0.72) | $ (0.60) | $ (1.10) | $ (0.61) | $ (0.59) | $ (0.54) | $ (2.89) | $ (2.85) | $ (2.02) |
Common stock options | |||||||||||
Effect of dilutive securities: | |||||||||||
Dilutive securities (in shares) | 0 | 0 | 0 | ||||||||
Restricted stock and restricted stock units | |||||||||||
Effect of dilutive securities: | |||||||||||
Dilutive securities (in shares) | 0 | 0 | 0 |
Summary of Significant Accoun34
Summary of Significant Accounting Policies - Anti-dilutive Securities (Details) - shares shares in Thousands | 12 Months Ended | ||
Dec. 31, 2017 | Dec. 31, 2016 | Dec. 31, 2015 | |
Stock options to purchase common stock | |||
Anti-dilutive securities excluded from computations of diluted weighted-average common shares outstanding | |||
Potentially dilutive securities excluded from computations of diluted weighted-average shares outstanding (in shares) | 8,609 | 7,117 | 5,274 |
Restricted stock and restricted stock units | |||
Anti-dilutive securities excluded from computations of diluted weighted-average common shares outstanding | |||
Potentially dilutive securities excluded from computations of diluted weighted-average shares outstanding (in shares) | 47 | 89 | 44 |
Summary of Significant Accoun35
Summary of Significant Accounting Policies - Segment Information (Details) | 12 Months Ended |
Dec. 31, 2017segment | |
Accounting Policies [Abstract] | |
Number of operating segments | 1 |
Number of reportable segments | 1 |
Accrued Expenses (Details)
Accrued Expenses (Details) - USD ($) $ in Thousands | Dec. 31, 2017 | Dec. 31, 2016 |
Payables and Accruals [Abstract] | ||
Accrued clinical trial expenses | $ 7,837 | $ 6,683 |
Accrued compensation | 12,197 | 6,937 |
Accrued professional fees | 4,500 | 1,992 |
Accrued technical operation expenses | 2,182 | 591 |
Accrued interest payable | 423 | 438 |
Accrued construction costs | 1,719 | 0 |
Other accrued expenses | 481 | 181 |
Total accrued expenses | $ 29,339 | $ 16,822 |
Identifiable Intangible Assets
Identifiable Intangible Assets (Details) - USD ($) $ in Millions | Dec. 31, 2017 | Dec. 31, 2016 |
IPRD | ||
Identifiable Intangible Assets | ||
Total intangible asset | $ 58.2 | $ 58.2 |
Fixed Assets, net (Details)
Fixed Assets, net (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2017 | Dec. 31, 2016 | Dec. 31, 2015 | |
Property, Plant and Equipment | |||
Fixed assets, gross | $ 23,077 | $ 17,764 | |
Less accumulated depreciation | (10,645) | (7,744) | |
Fixed assets, net | 12,432 | 10,020 | |
Depreciation expense | 2,901 | 2,438 | $ 1,982 |
Lab equipment | |||
Property, Plant and Equipment | |||
Fixed assets, gross | $ 7,055 | 5,662 | |
Estimated Useful Life (years) | 7 years | ||
Furniture and fixtures | |||
Property, Plant and Equipment | |||
Fixed assets, gross | $ 1,937 | 1,903 | |
Estimated Useful Life (years) | 7 years | ||
Computer hardware and software | |||
Property, Plant and Equipment | |||
Fixed assets, gross | $ 2,325 | 2,251 | |
Computer hardware and software | Minimum | |||
Property, Plant and Equipment | |||
Estimated Useful Life (years) | 3 years | ||
Computer hardware and software | Maximum | |||
Property, Plant and Equipment | |||
Estimated Useful Life (years) | 5 years | ||
Office equipment | |||
Property, Plant and Equipment | |||
Fixed assets, gross | $ 65 | 65 | |
Estimated Useful Life (years) | 7 years | ||
Manufacturing equipment | |||
Property, Plant and Equipment | |||
Fixed assets, gross | $ 1,436 | 1,148 | |
Estimated Useful Life (years) | 7 years | ||
Leasehold improvements | |||
Property, Plant and Equipment | |||
Fixed assets, gross | $ 6,939 | 6,735 | |
Construction in Progress (CIP) | |||
Property, Plant and Equipment | |||
Fixed assets, gross | $ 3,320 | $ 0 |
Debt - Narrative (Details)
Debt - Narrative (Details) - USD ($) | Feb. 28, 2018 | Sep. 30, 2016 | Oct. 31, 2016 | Jan. 31, 2016 | Dec. 31, 2017 | Sep. 29, 2016 | Jun. 29, 2012 |
Debt | |||||||
Payment of end of term charge | $ 390,000 | ||||||
Hercules | |||||||
Debt | |||||||
Maximum borrowing capacity of term loan | $ 25,000,000 | ||||||
Interest rate (as a percent) | 9.25% | ||||||
A&R Loan Agreement | |||||||
Debt | |||||||
Maximum borrowing capacity of term loan | $ 55,000,000 | ||||||
Interest rate (as a percent) | 9.25% | ||||||
Amount outstanding | 35,000,000 | $ 25,000,000 | |||||
Amount of borrowings during the period | $ 10,000,000 | $ 20,000,000 | |||||
Facility fee paid (as a percent) | 0.375% | ||||||
Backend fee (as a percent) | 4.15% | ||||||
Facility fee | $ 337,500 | ||||||
Minimum cash liquidity requirement eliminated | $ 25,000,000 | ||||||
Additional interest rate in event of default (as a percent) | 5.00% | ||||||
A&R Loan Agreement | License Agreement with AstraZeneca | |||||||
Debt | |||||||
Amount outstanding | $ 20,000,000 | ||||||
A&R Loan Agreement | Prime Rate | |||||||
Debt | |||||||
Basis spread (as a percent) | (4.50%) | ||||||
Forecast | A&R Loan Agreement | |||||||
Debt | |||||||
Prepayment of debt | $ 58,000,000 |
Debt - Components of Debt (Deta
Debt - Components of Debt (Details) - USD ($) $ in Thousands | Dec. 31, 2017 | Dec. 31, 2016 |
Debt: | ||
Note payable under A&R Loan Agreement | $ 55,000 | |
Accretion of end of term charge | 828 | |
Issuance fees paid to lender | (261) | |
Current portion of long-term debt | 0 | |
Long-term debt | $ 55,567 | $ 54,791 |
Debt - Future Principal Repayme
Debt - Future Principal Repayments of Debt (Details) $ in Thousands | Dec. 31, 2017USD ($) |
Debt Disclosure [Abstract] | |
2,018 | $ 0 |
2,019 | 13,399 |
2,020 | 41,601 |
Total | $ 55,000 |
Shareholders' Equity (Details)
Shareholders' Equity (Details) - USD ($) $ / shares in Units, $ in Thousands | 1 Months Ended | 12 Months Ended | |||
Sep. 30, 2017 | Apr. 30, 2015 | Dec. 31, 2017 | Dec. 31, 2016 | Dec. 31, 2015 | |
Class of Stock [Line Items] | |||||
Common stock, authorized shares (in shares) | 500,000,000 | 500,000,000 | |||
Common stock, par value (in dollars per share) | $ 0.01 | $ 0.01 | |||
Common stock, issued shares (in shares) | 76,610,508 | 62,019,889 | |||
Common stock, outstanding shares (in shares) | 76,610,508 | 62,019,889 | |||
Shares issued under underwritten public offering (in shares) | 14,123,150 | 11,500,000 | |||
Shares exercised in full of over-allotment option by underwriter (in shares) | 1,842,150 | 1,500,000 | |||
Shares price (in dollars per share) | $ 28.50 | $ 20.65 | |||
Underwriter's discount and offering expenses | $ 24,800 | $ 14,500 | |||
Net proceeds from the sale of shares | $ 377,700 | $ 222,900 | $ 377,656 | $ 0 | $ 222,942 |
Preferred stock, authorized (in shares) | 200,000,000 | 200,000,000 | |||
Preferred stock, par value (in shares) | $ 0.01 | $ 0.01 | |||
Preferred stock, shares issued (in shares) | 0 | 0 | |||
Preferred stock, shares outstanding (in shares) | 0 | 0 | |||
Stock options to purchase common stock | |||||
Class of Stock [Line Items] | |||||
Common stock shares reserved for issuance (in shares) | 8,608,921 | ||||
Restricted stock and restricted stock units | |||||
Class of Stock [Line Items] | |||||
Common stock shares reserved for issuance (in shares) | 46,914 |
Stock-Based Compensation - Narr
Stock-Based Compensation - Narrative (Details) - shares | 12 Months Ended | ||
Dec. 31, 2017 | Dec. 31, 2016 | May 18, 2017 | |
Share-based Compensation [Abstract] | |||
Shares of common stock, maximum authorized for issuance (in shares) | 5,000,000 | ||
Shares available for grant (in shares) | 4,910,002 | ||
Inducement stock option granted to new employees (in shares) | 266,230 | 88,060 |
Stock-Based Compensation - Stoc
Stock-Based Compensation - Stock Options (Details) - USD ($) $ / shares in Units, $ in Thousands | 3 Months Ended | 12 Months Ended | ||
Mar. 31, 2015 | Dec. 31, 2017 | Dec. 31, 2016 | Dec. 31, 2015 | |
Number of Shares | ||||
Options outstanding beginning of the year (in shares) | 4,400,106 | 7,116,706 | 5,273,722 | 4,400,106 |
Granted (in shares) | 2,284,710 | 2,532,675 | 1,902,850 | |
Exercised (in shares) | (378,275) | (162,340) | (481,140) | |
Forfeited and expired (in shares) | (414,220) | (527,351) | (548,094) | |
Options outstanding end of the year (in shares) | 8,608,921 | 7,116,706 | 5,273,722 | |
Vested and expected to vest (in shares) | 8,325,255 | 6,850,658 | 5,059,645 | |
Exercisable (in shares) | 4,229,478 | 3,113,998 | 1,991,141 | |
Weighted Average Exercise Price | ||||
Options outstanding beginning of the year (in dollars per share) | $ 10.59 | $ 13.30 | $ 13.64 | $ 10.59 |
Granted (in dollars per share) | 15.92 | 12.96 | 20.45 | |
Exercised (in dollars per share) | 9.08 | 6.68 | 10.62 | |
Forfeited and expired (in dollars per share) | 15.50 | 17.08 | 15.43 | |
Options outstanding end of the year (in dollars per share) | 14.08 | 13.30 | 13.64 | |
Vested and expected to vest (in dollars per share) | 14.03 | 13.25 | 13.46 | |
Exercisable (in dollars per share) | $ 12.71 | $ 11.28 | $ 8.70 | |
Weighted Average Remaining Contractual Life in Years | ||||
Options outstanding | 7 years 5 months 4 days | |||
Vested and expected to vest | 7 years 4 months 22 days | |||
Exercisable | 6 years 3 months 23 days | |||
Aggregate Intrinsic Value | ||||
Options outstanding | $ 147,260 | |||
Vested and expected to vest | 142,783 | |||
Exercisable | $ 78,138 | |||
Stock options to purchase common stock | ||||
Fair value and assumptions used in determining fair value of stock options | ||||
Dividend yield | 0.00% | 0.00% | 0.00% | |
Expected option term (in years) | 6 years 3 months | 6 years 3 months | 6 years 3 months | |
Weighted average fair value of stock options granted (in dollars per share) | $ 10.52 | $ 8.77 | $ 14.20 | |
Stock options to purchase common stock | Minimum | ||||
Fair value and assumptions used in determining fair value of stock options | ||||
Volatility | 71.00% | 74.00% | 78.00% | |
Risk-free interest rate | 1.73% | 1.00% | 1.31% | |
Stock options to purchase common stock | Maximum | ||||
Fair value and assumptions used in determining fair value of stock options | ||||
Volatility | 79.00% | 77.00% | 82.00% | |
Risk-free interest rate | 2.13% | 1.90% | 1.75% | |
Performance-condition options | ||||
Stock Option disclosures | ||||
Non-cash compensation expense | $ 1,500 | |||
Number of Shares | ||||
Options outstanding end of the year (in shares) | 133,334 |
Stock-Based Compensation - Rang
Stock-Based Compensation - Range of Exercise Prices (Details) - USD ($) $ / shares in Units, $ in Millions | 12 Months Ended | ||
Dec. 31, 2017 | Dec. 31, 2016 | Dec. 31, 2015 | |
Stock options to purchase common stock | |||
Share-based Compensation, Shares Authorized under Stock Option Plans, Exercise Price Range | |||
Total intrinsic value of stock options exercised during the period | $ 4.3 | $ 1 | $ 4.7 |
Unrecognized compensation expense related to unvested stock options | $ 29.7 | ||
Expected weighted average period for recognizing unrecognized compensation expense | 2 years 6 months 25 days | ||
Performance-condition options | |||
Share-based Compensation, Shares Authorized under Stock Option Plans, Exercise Price Range | |||
Unrecognized compensation expense related to unvested stock options | $ 1.1 | ||
$3.03 to $4.55 | |||
Share-based Compensation, Shares Authorized under Stock Option Plans, Exercise Price Range | |||
Range of Exercise Prices lower range limit (in dollars per share) | $ 3.03 | ||
Range of Exercise Prices upper range limit (in dollars per share) | $ 4.55 | ||
Number of Options outstanding (in shares) | 973,195 | ||
Weighted Average Remaining Contractual Term (in years) | 4 years 7 months 24 days | ||
Outstanding Options, Weighted Average Exercise Price (in dollars per share) | $ 3.60 | ||
Number of Options exercisable (in shares) | 973,195 | ||
Exercisable Options, Weighted Average Exercise Price (in dollars per share) | $ 3.60 | ||
$6.90 to $6.90 | |||
Share-based Compensation, Shares Authorized under Stock Option Plans, Exercise Price Range | |||
Range of Exercise Prices lower range limit (in dollars per share) | 6.90 | ||
Range of Exercise Prices upper range limit (in dollars per share) | $ 6.90 | ||
Number of Options outstanding (in shares) | 137,577 | ||
Weighted Average Remaining Contractual Term (in years) | 5 years 2 months 19 days | ||
Outstanding Options, Weighted Average Exercise Price (in dollars per share) | $ 6.90 | ||
Number of Options exercisable (in shares) | 100,077 | ||
Exercisable Options, Weighted Average Exercise Price (in dollars per share) | $ 6.90 | ||
$3.60 to $10.85 | |||
Share-based Compensation, Shares Authorized under Stock Option Plans, Exercise Price Range | |||
Range of Exercise Prices lower range limit (in dollars per share) | 6.96 | ||
Range of Exercise Prices upper range limit (in dollars per share) | $ 10.85 | ||
Number of Options outstanding (in shares) | 1,077,621 | ||
Weighted Average Remaining Contractual Term (in years) | 8 years 3 months 14 days | ||
Outstanding Options, Weighted Average Exercise Price (in dollars per share) | $ 10.76 | ||
Number of Options exercisable (in shares) | 420,374 | ||
Exercisable Options, Weighted Average Exercise Price (in dollars per share) | $ 10.62 | ||
$11.14 to $12.58 | |||
Share-based Compensation, Shares Authorized under Stock Option Plans, Exercise Price Range | |||
Range of Exercise Prices lower range limit (in dollars per share) | 11.14 | ||
Range of Exercise Prices upper range limit (in dollars per share) | $ 12.58 | ||
Number of Options outstanding (in shares) | 1,088,035 | ||
Weighted Average Remaining Contractual Term (in years) | 6 years 4 months 20 days | ||
Outstanding Options, Weighted Average Exercise Price (in dollars per share) | $ 12.17 | ||
Number of Options exercisable (in shares) | 781,991 | ||
Exercisable Options, Weighted Average Exercise Price (in dollars per share) | $ 12.18 | ||
$12.66 to $13.67 | |||
Share-based Compensation, Shares Authorized under Stock Option Plans, Exercise Price Range | |||
Range of Exercise Prices lower range limit (in dollars per share) | 12.66 | ||
Range of Exercise Prices upper range limit (in dollars per share) | $ 13.67 | ||
Number of Options outstanding (in shares) | 1,043,273 | ||
Weighted Average Remaining Contractual Term (in years) | 8 years 8 months 26 days | ||
Outstanding Options, Weighted Average Exercise Price (in dollars per share) | $ 13.59 | ||
Number of Options exercisable (in shares) | 101,126 | ||
Exercisable Options, Weighted Average Exercise Price (in dollars per share) | $ 13.26 | ||
$13.94 to $15.91 | |||
Share-based Compensation, Shares Authorized under Stock Option Plans, Exercise Price Range | |||
Range of Exercise Prices lower range limit (in dollars per share) | 13.94 | ||
Range of Exercise Prices upper range limit (in dollars per share) | $ 15.91 | ||
Number of Options outstanding (in shares) | 862,300 | ||
Weighted Average Remaining Contractual Term (in years) | 7 years 9 months 4 days | ||
Outstanding Options, Weighted Average Exercise Price (in dollars per share) | $ 14.97 | ||
Number of Options exercisable (in shares) | 406,441 | ||
Exercisable Options, Weighted Average Exercise Price (in dollars per share) | $ 14.53 | ||
$16.07 to $16.16 | |||
Share-based Compensation, Shares Authorized under Stock Option Plans, Exercise Price Range | |||
Range of Exercise Prices lower range limit (in dollars per share) | 16.07 | ||
Range of Exercise Prices upper range limit (in dollars per share) | $ 16.16 | ||
Number of Options outstanding (in shares) | 1,008,750 | ||
Weighted Average Remaining Contractual Term (in years) | 7 years 8 months 8 days | ||
Outstanding Options, Weighted Average Exercise Price (in dollars per share) | $ 16.13 | ||
Number of Options exercisable (in shares) | 463,629 | ||
Exercisable Options, Weighted Average Exercise Price (in dollars per share) | $ 16.12 | ||
$16.35 to $17.24 | |||
Share-based Compensation, Shares Authorized under Stock Option Plans, Exercise Price Range | |||
Range of Exercise Prices lower range limit (in dollars per share) | 16.35 | ||
Range of Exercise Prices upper range limit (in dollars per share) | $ 17.24 | ||
Number of Options outstanding (in shares) | 868,650 | ||
Weighted Average Remaining Contractual Term (in years) | 9 years 2 months 26 days | ||
Outstanding Options, Weighted Average Exercise Price (in dollars per share) | $ 17.13 | ||
Number of Options exercisable (in shares) | 23,999 | ||
Exercisable Options, Weighted Average Exercise Price (in dollars per share) | $ 16.85 | ||
$17.36 to $22.76 | |||
Share-based Compensation, Shares Authorized under Stock Option Plans, Exercise Price Range | |||
Range of Exercise Prices lower range limit (in dollars per share) | 17.36 | ||
Range of Exercise Prices upper range limit (in dollars per share) | $ 22.76 | ||
Number of Options outstanding (in shares) | 1,415,700 | ||
Weighted Average Remaining Contractual Term (in years) | 7 years 1 month 20 days | ||
Outstanding Options, Weighted Average Exercise Price (in dollars per share) | $ 21.18 | ||
Number of Options exercisable (in shares) | 927,334 | ||
Exercisable Options, Weighted Average Exercise Price (in dollars per share) | $ 21.23 | ||
$22.84 to $31.78 | |||
Share-based Compensation, Shares Authorized under Stock Option Plans, Exercise Price Range | |||
Range of Exercise Prices lower range limit (in dollars per share) | 22.84 | ||
Range of Exercise Prices upper range limit (in dollars per share) | $ 31.78 | ||
Number of Options outstanding (in shares) | 133,820 | ||
Weighted Average Remaining Contractual Term (in years) | 8 years 10 months 10 days | ||
Outstanding Options, Weighted Average Exercise Price (in dollars per share) | $ 27.47 | ||
Number of Options exercisable (in shares) | 31,312 | ||
Exercisable Options, Weighted Average Exercise Price (in dollars per share) | $ 23.74 |
Stock-Based Compensation - Rest
Stock-Based Compensation - Restricted Stock and Restricted Stock Units (Details) - USD ($) $ / shares in Units, $ in Millions | 12 Months Ended | ||
Dec. 31, 2017 | Dec. 31, 2016 | Dec. 31, 2015 | |
Stock options and RSUs | |||
Weighted Average Grant Price | |||
Allocated share-based compensation expense | $ 18.1 | $ 18 | $ 15.6 |
Stock-based compensation expense that resulted from the remeasurement of awards | 0 | 1.7 | 2.3 |
Stock options and RSUs | Research and development expenses | |||
Weighted Average Grant Price | |||
Allocated share-based compensation expense | 6.5 | 6.2 | 4 |
Stock options and RSUs | General and administrative expenses | |||
Weighted Average Grant Price | |||
Allocated share-based compensation expense | $ 11.6 | $ 11.8 | $ 11.6 |
Restricted stock and restricted stock units | |||
Number of RSUs | |||
Outstanding, beginning balance (in shares) | 89,194 | 43,554 | 20,502 |
Granted (in shares) | 46,914 | 89,194 | 49,776 |
Released (in shares) | (89,194) | (43,554) | (26,724) |
Forfeited (in shares) | 0 | 0 | 0 |
Outstanding, ending balance (in shares) | 46,914 | 89,194 | 43,554 |
Weighted Average Grant Price | |||
Outstanding Weighted Average Grant Price (in dollars per share) | $ 10.85 | $ 19.47 | $ 19.47 |
Granted (in dollars per share) | 17.16 | 10.85 | 16.07 |
Released (in dollars per share) | (10.85) | (16.07) | (18.68) |
Forfeited (in dollars per share) | 0 | 0 | 0 |
Outstanding Weighted Average Grant Price (in dollars per share) | $ 17.16 | $ 10.85 | $ 19.47 |
RSUs | |||
Stock Based Compensation disclosures | |||
Number of common shares each award holder is entitled to receive | 1 |
Income Taxes - Narrative (Detai
Income Taxes - Narrative (Details) | 12 Months Ended | ||
Dec. 31, 2017USD ($) | Dec. 31, 2016USD ($) | Dec. 31, 2015USD ($) | |
NOL carryforwards | |||
Income tax provision (benefit) | $ (272,000) | $ 98,000 | $ (1,971,000) |
Effective tax rate | 0.00% | 0.00% | 2.00% |
Unrecognized tax benefits, interest and penalties accrued | $ 0 | $ 0 | |
Net deferred tax assets (prior to applying the valuation allowance) | 261,300,000 | 284,598,000 | |
Increase (decrease) in valuation allowance | (23,300,000) | $ 68,400,000 | |
New Jersey | |||
NOL carryforwards | |||
State NOLs sold under the State of New Jersey's Technology Business Tax Certificate Transfer Program | $ 24,300,000 | ||
Cash proceeds from sale of NOLs, net of commissions | $ 2,000,000 | ||
Net operating loss carryforwards available to offset future taxable income | $ 296,000,000 | ||
Percentage Company is resident of NJ | 1 | ||
Federal | |||
NOL carryforwards | |||
Net operating loss carryforwards for income tax purposes | $ 721,800,000 | ||
Net operating loss carryforwards available to offset future taxable income | $ 544,000,000 |
Income Taxes - Loss Before Inco
Income Taxes - Loss Before Income Taxes in the US and Globally (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2017 | Dec. 31, 2016 | Dec. 31, 2015 | |
Income Tax Disclosure [Abstract] | |||
US | $ (136,682) | $ (140,354) | $ (100,278) |
Foreign | (56,239) | (35,821) | (19,876) |
Loss before income taxes | $ (192,921) | $ (176,175) | $ (120,154) |
Income Taxes - Income Tax (Bene
Income Taxes - Income Tax (Benefit) Provision (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2017 | Dec. 31, 2016 | Dec. 31, 2015 | |
Current: | |||
Federal | $ 0 | $ 0 | $ 0 |
State | 3 | 3 | (2,015) |
Foreign | 142 | 95 | 44 |
Current income tax provision (benefit) | 145 | 98 | (1,971) |
Deferred: | |||
Federal | (417) | 0 | 0 |
State | 0 | 0 | 0 |
Foreign | 0 | 0 | 0 |
Deferred income tax provision (benefit) | (417) | 0 | 0 |
Total | $ (272) | $ 98 | $ (1,971) |
Income Taxes - Reconciliation B
Income Taxes - Reconciliation Between Federal Statutory Tax Rate and Effective Tax Rate (Details) | 12 Months Ended | ||
Dec. 31, 2017 | Dec. 31, 2016 | Dec. 31, 2015 | |
Income Tax Disclosure [Abstract] | |||
Statutory federal tax rate | 34.00% | 34.00% | 34.00% |
Permanent items | (3.00%) | (3.00%) | (4.00%) |
State income taxes, net of federal benefit | 4.00% | 4.00% | 4.00% |
R&D and other tax credits | 8.00% | 8.00% | 12.00% |
Foreign income taxes | (6.00%) | (4.00%) | (1.00%) |
Impact of 2017 Tax Act | (49.00%) | 0.00% | 0.00% |
Change in valuation allowance | 12.00% | (39.00%) | (43.00%) |
Other | 0.00% | 0.00% | 0.00% |
Effective tax rate | 0.00% | 0.00% | 2.00% |
Income Taxes - Components of De
Income Taxes - Components of Deferred Tax Assets and Liabilities (Details) - USD ($) $ in Thousands | Dec. 31, 2017 | Dec. 31, 2016 |
Deferred tax assets: | ||
Net operating loss carryforwards | $ 186,342 | $ 228,729 |
General business credits | 66,371 | 50,648 |
Product license | 7,730 | 11,783 |
Alternative minimum tax (AMT) credit | 0 | 418 |
Other | 17,217 | 16,265 |
Gross deferred tax assets | 277,660 | 307,843 |
Deferred tax liabilities: | ||
In-process research and development | (16,360) | (23,245) |
Deferred tax liabilities | (16,360) | (23,245) |
Net deferred tax assets | 261,300 | 284,598 |
Valuation allowance | (261,300) | (284,598) |
Net deferred tax assets | $ 0 | $ 0 |
License and Other Agreements (D
License and Other Agreements (Details) € in Millions, $ in Millions | 1 Months Ended | 3 Months Ended | 12 Months Ended | ||||||||||
Oct. 31, 2017USD ($) | Oct. 31, 2016USD ($) | Apr. 30, 2015USD ($) | Dec. 31, 2014USD ($) | Jul. 31, 2014 | Feb. 28, 2014 | Apr. 30, 2008EUR (€) | Dec. 31, 2016USD ($) | Dec. 31, 2017USD ($) | Dec. 31, 2016 | Dec. 31, 2009USD ($) | Dec. 31, 2004USD ($) | Sep. 30, 2015USD ($) | |
PARI Agreement | |||||||||||||
Collaboration Agreements | |||||||||||||
Initial term of contract manufacturing agreement | 15 years | ||||||||||||
Additional term of contract manufacturing agreement | 5 years | ||||||||||||
PARI Agreement | Minimum | |||||||||||||
Collaboration Agreements | |||||||||||||
Written notice period required for termination of contract manufacturing agreement | 1 year | ||||||||||||
PARI Agreement | Maximum | |||||||||||||
Collaboration Agreements | |||||||||||||
Upfront payment | € | € 4.3 | ||||||||||||
Agreement with SynteractHCR, Inc. for 212 Study | |||||||||||||
Collaboration Agreements | |||||||||||||
Commitment To Pay Aggregate Costs From Agreement | $ 45 | ||||||||||||
Agreement with SynteractHCR, Inc. for 312 Study | |||||||||||||
Collaboration Agreements | |||||||||||||
Commitment To Pay Aggregate Costs From Agreement | $ 25 | ||||||||||||
CFFT | |||||||||||||
Collaboration Agreements | |||||||||||||
Compensation earned under research funding agreements with CFFT | $ 2.2 | $ 1.7 | |||||||||||
Royalty payable on approval of ARIKAYCE as commercialized drug | $ 13.4 | ||||||||||||
Royalty payment period | 3 years | ||||||||||||
Period for meeting sales milestones for additional royalty payments | 5 years | ||||||||||||
Royalty payable on meeting certain sales milestones | $ 3.9 | ||||||||||||
Therapure | |||||||||||||
Collaboration Agreements | |||||||||||||
Initial term of contract manufacturing agreement | 5 years | ||||||||||||
Period of each automatic renewal of contract manufacturing agreement | 2 years | ||||||||||||
Written notice period required for termination of contract manufacturing agreement | 2 years | ||||||||||||
Fill/Finish Agreement | |||||||||||||
Collaboration Agreements | |||||||||||||
Minimum obligation | $ 2.7 | ||||||||||||
Extension period | 2 years | ||||||||||||
Period prior to expiration for extension | 1 year | ||||||||||||
License Agreement with AstraZeneca | |||||||||||||
Collaboration Agreements | |||||||||||||
Royalty payment | $ 35 | ||||||||||||
Annual net sales | 1,000 | ||||||||||||
License Agreement with AstraZeneca | Research and development expenses | |||||||||||||
Collaboration Agreements | |||||||||||||
Upfront payment | $ 30 | ||||||||||||
License Agreement with AstraZeneca | Maximum | |||||||||||||
Collaboration Agreements | |||||||||||||
Aggregate payment upon the achievement of certain clinical milestones | 85 | ||||||||||||
Additional contingent payments upon second indication to develop INS1007 | $ 42.5 | ||||||||||||
Supply Agreement with Patheon UK Limited | |||||||||||||
Collaboration Agreements | |||||||||||||
Agreement to purchase raw materials and fixed assets | $ 60 | ||||||||||||
Supply Agreement with Patheon UK Limited | Minimum | |||||||||||||
Collaboration Agreements | |||||||||||||
Term of agreement to purchase raw materials and fixed assets | 3 years | ||||||||||||
Supply Agreement with Patheon UK Limited | Maximum | |||||||||||||
Collaboration Agreements | |||||||||||||
Term of agreement to purchase raw materials and fixed assets | 4 years |
Commitments and Contingencies -
Commitments and Contingencies - Narrative (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2017 | Dec. 31, 2016 | Dec. 31, 2015 | |
Commitments | |||
Future minimum rental payments under leases | $ 3,917 | ||
Rent expense charged to operations | 1,500 | $ 1,200 | $ 800 |
Bridgewater, NJ Facility | |||
Commitments | |||
Future minimum rental payments under leases | 2,000 | ||
Future minimum rental payments for additional laboratory space | $ 1,900 |
Commitments and Contingencies54
Commitments and Contingencies - Minimum Rental Payments (Details) $ in Thousands | Dec. 31, 2017USD ($) |
Commitments and Contingencies Disclosure [Abstract] | |
2,018 | $ 1,521 |
2,019 | 1,421 |
2,020 | 477 |
2,021 | 498 |
2,022 | 0 |
Total | $ 3,917 |
Quarterly Financial Data (Una55
Quarterly Financial Data (Unaudited) (Details) - USD ($) $ / shares in Units, $ in Thousands | 3 Months Ended | 12 Months Ended | |||||||||
Dec. 31, 2017 | Sep. 30, 2017 | Jun. 30, 2017 | Mar. 31, 2017 | Dec. 31, 2016 | Sep. 30, 2016 | Jun. 30, 2016 | Mar. 31, 2016 | Dec. 31, 2017 | Dec. 31, 2016 | Dec. 31, 2015 | |
Collaboration Agreements | |||||||||||
Revenues | $ 0 | $ 0 | $ 0 | $ 0 | $ 0 | $ 0 | $ 0 | $ 0 | $ 0 | $ 0 | $ 0 |
Operating loss | (65,353) | (44,083) | (43,515) | (35,969) | (67,051) | (37,149) | (36,133) | (33,067) | (188,920) | (173,400) | (117,493) |
Net loss | $ (65,384) | $ (45,179) | $ (44,672) | $ (37,414) | $ (68,402) | $ (37,760) | $ (36,579) | $ (33,532) | $ (192,649) | $ (176,273) | $ (118,183) |
Basic and diluted net loss per share | $ (0.85) | $ (0.69) | $ (0.72) | $ (0.60) | $ (1.10) | $ (0.61) | $ (0.59) | $ (0.54) | $ (2.89) | $ (2.85) | $ (2.02) |
License Agreement with AstraZeneca | Research and development expenses | |||||||||||
Collaboration Agreements | |||||||||||
Upfront payment | $ 30,000 |
Retirement Plan (Details)
Retirement Plan (Details) - USD ($) $ in Millions | 12 Months Ended | |||
Dec. 31, 2018 | Dec. 31, 2017 | Dec. 31, 2016 | Dec. 31, 2015 | |
Defined Benefit Plan Disclosure [Line Items] | ||||
Employer matching contribution, percent of match | 100.00% | |||
Employer matching contribution, percent of employees' gross pay | 3.00% | |||
Employer contributions | $ 0.8 | $ 0.6 | $ 0.4 | |
Forecast | ||||
Defined Benefit Plan Disclosure [Line Items] | ||||
Employer matching contribution, percent of employees' gross pay | 4.00% |
Subsequent Event (Details)
Subsequent Event (Details) - 1.75% convertible senior notes due 2025 - Subsequent event | 1 Months Ended |
Jan. 31, 2018USD ($)$ / shares | |
Subsequent Event [Line Items] | |
Net proceeds | $ 435,800,000 |
Initial conversion rate | 0.0255384 |
Initial conversion price (in dollars per share) | $ / shares | $ 39.16 |
Convertible senior notes | |
Subsequent Event [Line Items] | |
Interest rate (as a percent) | 1.75% |
Aggregate principal amount | $ 450,000,000 |
Option to purchase additional debt | 50,000,000 |
Underwriting discounts and commissions and other offering expenses | $ 14,200,000 |