Cover Page
Cover Page - USD ($) | 12 Months Ended | ||
Dec. 31, 2019 | Feb. 14, 2020 | Jun. 30, 2019 | |
Cover [Abstract] | |||
Document Type | 10-K | ||
Amendment Flag | false | ||
Document Period End Date | Dec. 31, 2019 | ||
Document Fiscal Year Focus | 2019 | ||
Document Fiscal Period Focus | FY | ||
Current Fiscal Year End Date | --12-31 | ||
Entity Registrant Name | Karyopharm Therapeutics Inc. | ||
Trading Symbol | KPTI | ||
Entity Central Index Key | 0001503802 | ||
Entity Filer Category | Accelerated Filer | ||
Entity Small Business | true | ||
Entity Emerging Growth Company | false | ||
Entity Common Stock, Shares Outstanding | 65,548,095 | ||
Entity Public Float | $ 343,517,677 | ||
Entity Well-known Seasoned Issuer | Yes | ||
Entity Current Reporting Status | Yes | ||
EntityInteractiveDataCurrent | Yes | ||
Entity File Number | 001-36167 | ||
Entity Incorporation, State or Country Code | DE | ||
Entity Tax Identification Number | 26-3931704 | ||
Entity Address, Address Line One | 85 Wells Avenue, 2nd Floor | ||
Entity Address, City or Town | Newton | ||
Entity Address, Postal Zip Code | 02459 | ||
City Area Code | 617 | ||
Local Phone Number | 658-0600 | ||
Document Transition Report | false | ||
Document Annual Report | true | ||
Entity Shell Company | false | ||
Entity Voluntary Filers | No | ||
Entity Address, State or Province | MA | ||
Title of 12(b) Security | Common Stock | ||
Security Exchange Name | NASDAQ |
Condensed Consolidated Balance
Condensed Consolidated Balance Sheets - USD ($) $ in Thousands | Dec. 31, 2019 | Dec. 31, 2018 |
Current assets: | ||
Cash and cash equivalents | $ 128,858 | $ 118,021 |
Short-term investments | 133,098 | 210,178 |
Accounts receivable | 7,862 | |
Inventory | 346 | |
Prepaid expenses and other current assets | 7,289 | 6,413 |
Restricted cash | 1,117 | |
Total current assets | 278,570 | 334,612 |
Property and equipment, net | 3,046 | 3,863 |
Operating lease right-of-use assets | 10,617 | |
Long-term investments | 2,016 | 2,001 |
Restricted cash | 714 | 716 |
Total assets | 294,963 | 341,192 |
Current liabilities: | ||
Accounts payable | 985 | 4,332 |
Accrued expenses | 40,878 | 32,493 |
Deferred revenue | 2,341 | 9,362 |
Operating lease liabilities | 1,646 | |
Deferred rent | 390 | |
Other current liabilities | 500 | 327 |
Total current liabilities | 46,350 | 46,904 |
Convertible senior notes | 109,857 | 102,664 |
Deferred royalty obligation | 73,588 | |
Operating lease liabilities, net of current portion | 13,202 | |
Deferred revenue, net of current portion | 2,192 | 4,532 |
Deferred rent, net of current portion | 3,922 | |
Total liabilities | 245,189 | 158,022 |
Commitments and contingencies (Note 9) | ||
Stockholders' equity: | ||
Preferred stock, $0.0001 par value; 5,000,000 shares authorized; none issued and outstanding | ||
Common stock, $0.0001 par value; 200,000,000 shares authorized; 65,370,448 and 60,829,308 shares issued and outstanding at December 31, 2019 and 2018, respectively | 7 | 6 |
Additional paid-in capital | 923,142 | 857,156 |
Accumulated other comprehensive loss | (37) | (244) |
Accumulated deficit | (873,338) | (673,748) |
Total stockholders' equity | 49,774 | 183,170 |
Total liabilities and stockholders' equity | $ 294,963 | $ 341,192 |
Condensed Consolidated Balanc_2
Condensed Consolidated Balance Sheets (Parenthetical) - $ / shares | Dec. 31, 2019 | Dec. 31, 2018 |
Statement of Financial Position [Abstract] | ||
Preferred stock, par value | $ 0.0001 | $ 0.0001 |
Preferred stock, shares authorized | 5,000,000 | 5,000,000 |
Preferred stock, shares issued | 0 | 0 |
Preferred stock, shares outstanding | 0 | 0 |
Common stock, par value | $ 0.0001 | $ 0.0001 |
Common stock, shares authorized | 200,000,000 | 200,000,000 |
Common stock, shares issued | 65,370,448 | 60,829,308 |
Common stock, shares outstanding | 65,370,448 | 60,829,308 |
Condensed Consolidated Statemen
Condensed Consolidated Statements of Operations - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Revenues: | |||
Total revenues | $ 40,893 | $ 30,336 | $ 1,605 |
Operating expenses: | |||
Cost of sales | 2,407 | ||
Research and development | 122,340 | 161,372 | 107,273 |
Selling, general and administrative | 105,421 | 48,847 | 24,870 |
Total operating expenses | 230,168 | 210,219 | 132,143 |
Loss from operations | (189,275) | (179,883) | (130,538) |
Other income (expense): | |||
Interest income | 5,422 | 4,028 | 1,698 |
Interest expense | (15,647) | (2,493) | |
Other expense | (50) | (33) | (81) |
Total other (expense) income, net | (10,275) | 1,502 | 1,617 |
Loss before income taxes | (199,550) | (178,381) | (128,921) |
Income tax provision | (40) | (26) | (63) |
Net loss | $ (199,590) | $ (178,407) | $ (128,984) |
Net loss per share—basic and diluted | $ (3.22) | $ (3.14) | $ (2.81) |
Weighted-average number of common shares outstanding used in net loss per share—basic and diluted | 61,955,420 | 56,799,699 | 45,899,784 |
Product revenue | |||
Revenues: | |||
Total revenues | $ 30,540 | ||
License and other revenue | |||
Revenues: | |||
Total revenues | $ 10,353 | $ 30,336 | $ 1,605 |
Condensed Consolidated Statem_2
Condensed Consolidated Statements of Comprehensive Loss - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Statement of Comprehensive Income [Abstract] | |||
Net loss | $ (199,590) | $ (178,407) | $ (128,984) |
Other comprehensive income (loss): | |||
Unrealized gain (loss) on investments | 207 | 39 | (97) |
Foreign currency translation adjustment | (66) | 154 | |
Comprehensive loss | $ (199,383) | $ (178,434) | $ (128,927) |
Condensed Consolidated Statem_3
Condensed Consolidated Statement of Stockholders' Equity - USD ($) $ in Thousands | Total | Common Stock [Member] | Additional Paid-In Capital [Member] | Accumulated Other Comprehensive Income (loss) [Member] | Accumulated Deficit [Member] |
Beginning balance at Dec. 31, 2016 | $ 162,243 | $ 4 | $ 528,617 | $ (274) | $ (366,104) |
Beginning balance,Shares at Dec. 31, 2016 | 41,887,829 | ||||
Cumulative effect adjustment for adoption of new accounting guidance | 253 | (253) | |||
Vesting of restricted stock (in shares) | 182,496 | ||||
Exercise of stock options and shares issued under the employee stock purchase plan | 858 | 858 | |||
Exercise of stock options and shares issued under the employee stock purchase plan, (shares) | 154,623 | ||||
Stock-based compensation expense | 20,405 | 20,405 | |||
Issuance of common stock | 74,885 | $ 1 | 74,884 | ||
Issuance of common stock (in Shares) | 7,308,202 | ||||
Unrealized gain (loss) on investments | (97) | (97) | |||
Foreign currency translation adjustment | 154 | 154 | |||
Net loss | (128,984) | (128,984) | |||
Ending balance at Dec. 31, 2017 | 129,464 | $ 5 | 625,017 | (217) | (495,341) |
Ending balance,Shares at Dec. 31, 2017 | 49,533,150 | ||||
Vesting of restricted stock (in shares) | 113,800 | ||||
Exercise of stock options and shares issued under the employee stock purchase plan | 3,519 | 3,519 | |||
Exercise of stock options and shares issued under the employee stock purchase plan, (shares) | 656,934 | ||||
Stock-based compensation expense | 17,275 | 17,275 | |||
Issuance of common stock | 145,705 | $ 1 | 145,704 | ||
Issuance of common stock (in Shares) | 10,525,424 | ||||
Equity component of 2025 Notes | 67,850 | 67,850 | |||
Equity component of deferred financing costs for 2025 notes | (2,209) | (2,209) | |||
Unrealized gain (loss) on investments | 39 | 39 | |||
Foreign currency translation adjustment | (66) | (66) | |||
Net loss | (178,407) | (178,407) | |||
Ending balance at Dec. 31, 2018 | 183,170 | $ 6 | 857,156 | (244) | (673,748) |
Ending balance,Shares at Dec. 31, 2018 | 60,829,308 | ||||
Exercise of stock options and shares issued under the employee stock purchase plan | 4,505 | 4,505 | |||
Exercise of stock options and shares issued under the employee stock purchase plan, (shares) | 811,281 | ||||
Stock-based compensation expense | 15,291 | 15,291 | |||
Issuance of common stock | 46,191 | $ 1 | 46,190 | ||
Issuance of common stock (in Shares) | 3,712,359 | ||||
Unrealized gain (loss) on investments | 207 | 207 | |||
Net loss | (199,590) | (199,590) | |||
Ending balance at Dec. 31, 2019 | $ 49,774 | $ 7 | $ 923,142 | $ (37) | $ (873,338) |
Ending balance,Shares at Dec. 31, 2019 | 65,370,448 |
Condensed Consolidated Statem_4
Condensed Consolidated Statement of Stockholders' Equity (Parenthetical) - USD ($) | 12 Months Ended | ||
Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Common Stock [Member] | Controlled Equity Offering Sales Agreement [Member] | |||
Issuance of stock, issuance costs | $ 1,000 | $ 200 | $ 1,100 |
Condensed Consolidated Statem_5
Condensed Consolidated Statements of Cash Flows - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Operating activities | |||
Net loss | $ (199,590) | $ (178,407) | $ (128,984) |
Adjustments to reconcile net loss to net cash used in operating activities: | |||
Depreciation and amortization | 974 | 735 | 713 |
Net amortization of premiums and discounts on investments | (1,382) | 29 | 1,187 |
Stock-based compensation expense | 15,291 | 17,275 | 20,405 |
Amortization of the value of debt discount and issuance costs | 7,193 | 1,420 | |
Change in operating assets and liabilities: | |||
Accounts receivable | (7,862) | ||
Inventory | (346) | ||
Prepaid expenses and other current assets | (868) | (4,663) | 342 |
Operating lease right-of-use assets | 1,094 | ||
Accounts payable | (3,301) | (1,380) | 909 |
Accrued expenses and other liabilities | 8,512 | 11,255 | 10,070 |
Operating lease liabilities | (1,175) | ||
Deferred revenue | (9,362) | (8,027) | 21,921 |
Deferred rent | 2,646 | (280) | |
Net cash used in operating activities | (190,822) | (159,117) | (73,717) |
Investing activities | |||
Purchases of property and equipment | (206) | (2,363) | (62) |
Proceeds from maturities of investments | 257,145 | 137,510 | 115,544 |
Purchases of investments | (178,489) | (242,811) | (98,374) |
Net cash provided by (used in) investing activities | 78,450 | (107,664) | 17,108 |
Financing activities | |||
Proceeds from issuance of convertible senior notes, net of issuance costs | 166,885 | ||
Proceeds from issuance of common stock, net of issuance costs | 46,191 | 145,705 | 74,885 |
Proceeds from the exercise of stock options and shares issued under employee stock purchase plan | 4,505 | 3,519 | 858 |
Proceeds from deferred royalty obligation, net | 73,609 | ||
Net cash provided by financing activities | 124,305 | 316,109 | 75,743 |
Effect of exchange rate on cash, cash equivalents and restricted cash | 19 | (78) | 211 |
Net increase in cash, cash equivalents and restricted cash | 11,952 | 49,250 | 19,345 |
Cash, cash equivalents and restricted cash at beginning of period | 118,737 | 69,487 | 50,142 |
Cash, cash equivalents and restricted cash end of period | 130,689 | 118,737 | 69,487 |
Reconciliation of cash, cash equivalents and restricted cash reported within the consolidated balance sheets | |||
Cash and cash equivalents | 128,858 | 118,021 | 68,997 |
Short-term restricted cash | 1,117 | 200 | |
Long-term restricted cash | 714 | 716 | 290 |
Cash, cash equivalents and restricted cash end of period | 130,689 | $ 118,737 | $ 69,487 |
Supplemental disclosures: | |||
Cash paid for interest on convertible debt | 5,175 | ||
Operating lease right-of-use assets obtained in exchange for operating lease liabilities | 11,711 | ||
Cash paid for amounts included in the measurement of operating lease liabilities | $ 2,889 |
Organization and Operations
Organization and Operations | 12 Months Ended |
Dec. 31, 2019 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
Organization and Operations | 1. Organization and Operations The Company We are an innovation-driven pharmaceutical company focused on the discovery, development and commercialization of novel, first-in-class S I N E SINE In July 2019, the U.S. Food and Drug Administration (“FDA”) approved XPOVIO ® As of December 31, 2019, we had an accumulated deficit of $873.3 million. We have had limited revenues to date from product sales and have financed our operations principally through private placements of our preferred stock, proceeds from our initial public offering and follow-on |
Summary of Significant Accounti
Summary of Significant Accounting Policies | 12 Months Ended |
Dec. 31, 2019 | |
Accounting Policies [Abstract] | |
Summary of Significant Accounting Policies | 2. Summary of Significant Accounting Policies Basis of Presentation The accompanying financial statements have been prepared in accordance with accounting principles generally accepted in the United States of America (“U.S. GAAP”). Segment Information Operating segments are defined as components of an enterprise about which separate discrete information is available for evaluation by the chief operating decision maker, or decision-making group, in deciding how to allocate resources and in assessing performance. We view our operations and manage our business in one operating segment, which is the business of discovering, developing and commercializing drugs to treat cancer and certain other major diseases. All of our revenue to date has been derived in the United States. All of our material long-lived assets reside in the United States. Use of Estimates The preparation of financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities and the disclosure of contingent assets and liabilities at the date of the financial statements and the reported amounts of revenues and expenses during the reporting period. On an ongoing basis, we evaluate our estimates, including estimates related to our net product revenue, clinical trial accruals, stock-based compensation expense, interest expense on our deferred royalty obligation and Principles of Consolidation The consolidated financial statements at December 31, 2019 include the accounts of (i) Karyopharm Therapeutics Inc., (ii) Karyopharm Securities Corp. (“KPSC”, our wholly-owned Massachusetts corporation incorporated in December 2013), (iii) Karyopharm Europe GmbH (our wholly-owned German Limited Liability Company, incorporated in September 2014), (iv) Karyopharm Therapeutics (Bermuda) Ltd. (our limited liability company, registered in Bermuda in March 2015), and (vi) Karyopharm Israel Ltd. (our wholly - Cash and Cash Equivalents Cash and cash equivalents consist primarily of demand deposit accounts and deposits in short-term money market funds. Cash equivalents are stated at cost, which approximates fair value. We consider all highly liquid investments with maturities of three months or less from the date of purchase to be cash equivalents. We do not hold any money market funds with significant liquidity restrictions that would be required to be excluded from cash equivalents. Investments We determine the appropriate classification of our investments in debt securities at the time of purchase. All of our securities are classified as available-for-sale Available-for-sale Concentrations of Credit Risk and Off-Balance Financial instruments which potentially subject us to credit risk consist primarily of cash, cash equivalents and investments. We hold these investments in highly rated financial institutions, and, by policy, limit the amounts of credit exposure to any one financial institution. These amounts at times may exceed federally insured limits. We have not experienced any credit losses in such accounts and do not believe we are exposed to any significant credit risk on these funds. We have no off-balance Fair Value Measurements Financial instruments, including cash, restricted cash, prepaid expenses and other current assets, accounts payable and accrued expenses, are presented at amounts that approximate fair value at December 31, 2019 and 2018. We are required to disclose information on all assets and liabilities reported at fair value that enables an assessment of the inputs used in determining the reported fair values. The fair value hierarchy prioritizes valuation inputs based on the observable nature of those inputs. The fair value hierarchy applies only to the valuation inputs used in determining the reported fair value of the investments and is not a measure of the investment credit quality. The hierarchy defines three levels of valuation inputs: Level 1 inputs Quoted prices in active markets for identical assets or liabilities Level 2 inputs Inputs other than quoted prices included within Level 1 that are observable for the asset or liability, either directly or indirectly Level 3 inputs Unobservable inputs that reflect our own assumptions about the assumptions market participants would use in pricing the asset or liability Our cash equivalents are composed of money market funds. We measure these investments at fair value. The fair value of cash equivalents is determined based on “Level 1” inputs. Items classified as Level 2 within the valuation hierarchy consist of commercial paper, corporate debt securities, U.S. government agency securities and certificates of deposit. We estimate the fair values of these marketable securities by taking into consideration valuations obtained from third-party pricing sources. These pricing sources utilize industry standard valuation models, including both income and market-based approaches, for which all significant inputs are observable, either directly or indirectly, to estimate fair value. These inputs include market pricing based on real-time trade data for the same or similar securities, issuer credit spreads, benchmark yields, and other observable inputs. We validate the prices provided by our third-party pricing sources by understanding the models used, obtaining market values from other pricing sources and analyzing pricing data in certain instances. In certain cases where there is limited activity or less transparency around inputs to valuation, the related assets or liabilities are classified as Level 3. The embedded derivative liability associated with our deferred royalty obligation, as discussed further in Note 1 5 5 The following table presents information about our financial assets and liability that have been measured at fair value at December 31, 2019 and indicates the fair value hierarchy of the valuation inputs utilized to determine such fair value (in thousands): Description Total Quoted Prices Significant Significant Financial assets Cash equivalents: Money market funds $ 71,380 $ 71,380 $ — $ — Investments: Short-term: Corporate debt securities 89,079 — 89,079 — Commercial paper 39,022 — 39,022 — U.S. government and agency securities 4,997 — 4,997 — Long-term: Corporate debt securities (one to two year maturity) 2,016 — 2,016 — $ 206,494 $ 71,380 $ 135,114 $ — Financial liability Embedded derivative liability $ 2,300 $ — $ — $ 2,300 The following table presents information about our financial assets that have been measured at fair value at December 31, 2018 and indicates the fair value hierarchy of the valuation inputs utilized to determine such fair value (in thousands): Description Total Quoted Prices Significant Significant Financial assets Cash equivalents: Money market funds $ 76,881 $ 76,881 $ — $ — Investments: Short-term: Corporate debt securities 143,079 — 143,079 — Commercial paper 43,978 — 43,978 — U.S. government and agency securities 19,124 — 19,124 — Certificate of deposit 3,997 — 3,997 — Long-term: Corporate debt securities (one to two year maturity) 2,001 — 2,001 — $ 289,060 $ 76,881 $ 212,179 $ — The following table sets forth a summary of the changes in the estimated fair value of our embedded derivative liability during the year ended December 31, 2019 (in thousands): Embedded Balance as of December 31, 2018 $ — Addition of derivative related to deferred royalty obligation . 2,300 Change in fair value of derivative since issuanc e — Balance as of December 31, 2019 $ 2,300 Our L 5 Property and Equipment, net Property and equipment are recorded at cost, less accumulated depreciation. Depreciation is recorded using the straight-line method over the estimated useful lives of the respective assets, generally three Long-Lived Assets We review the carrying values of our long-lived assets for possible impairment whenever events or changes in circumstances indicate that the carrying amounts of the assets may not be recoverable. Any long-lived assets held for disposal are reported at the lower of their carrying amounts or fair values less costs to sell. We have not recorded an impairment in any period since inception. Deferred Rent Deferred rent consists of rent escalation payment terms, tenant improvement allowances and other incentives received from landlords related to our operating leases under Financial Accounting Standards Board (“FASB”) Accounting Standards Codification (“ASC”) 840. Rent escalation represents the difference between actual operating lease payments due and straight-line rent expense. Tenant improvement allowances and other incentives were also recorded as deferred rent under ASC 840 through December 31, 2018. Deferred rent and lease incentives are recorded as a reduction to our operating lease right-of-use Revenue Recognition We adopted Accounting Standards Update (“ASU”) 2014-09, Revenue from Contracts with Customers ASC 606 applies to all contracts with customers, except for contracts that are within the scope of other standards, such as leases, insurance, collaboration arrangements, and financial instruments. Under ASC 606, we recognize revenue when our customer obtains control of promised goods or services, in an amount that reflects the consideration which we expect to receive in exchange for those goods or services. To determine revenue recognition for arrangements that we determine are within the scope of ASC 606, we perform the following five steps: (i) identify the contract(s) with a customer; (ii) identify the performance obligations in the contract; (iii) determine the transaction price; (iv) allocate the transaction price to the performance obligations in the contract; and (v) recognize revenue when (or as) we satisfy a performance obligation. At contract inception, once the contract is determined to be within the scope of ASC 606, we assess the goods or services promised within each contract and determine those that are performance obligations and assess whether each promised good or service is distinct. We then recognize as revenue the amount of the transaction price that is allocated to the respective performance obligation when (or as) the performance obligation is satisfied. Product Revenue Recognition In the third quarter of 2019, we began to ship XPOVIO in the United States to specialty pharmacies and specialty distributors, collectively referred to as our customers, under a limited number of distribution arrangements with such third parties. Our specialty pharmacy customers resell XPOVIO directly to patients while our specialty distributor customers resell XPOVIO to healthcare entities, who then resell to patients. In connection with negotiating and executing contracts with our customers, our policy is to expense incremental costs of obtaining a contract when incurred, if the expected amortization period of the asset that we would have recognized is one year or less. However, no such costs have been incurred to date. In addition to distribution agreements with our customers, we enter into certain arrangements with group purchasing organizations and/or other payors that provide for government mandated and/or privately negotiated rebates, chargebacks, and discounts with respect to the purchase of our products. In the context of ASC 606, each unit of XPOVIO that is ordered by our customers represents a distinct performance obligation that is completed when control of the product is transferred to the customer. Accordingly, we recognize product revenue when the customer obtains control of our product, which occurs at a point in time, generally upon delivery pursuant to our agreements with our customers. If taxes should be collected from customers relating to product sales and remitted to governmental authorities, they will be excluded from revenue. Revenue from product sales is recorded at the net sales price, which includes estimates of variable consideration for which reserves are reported. These reserves, as detailed below, are based on the amounts earned, or to be claimed on the related sales, and are classified as reductions of accounts receivable (if the amount is payable to the customer) or a current liability (if the amount is payable to a party other than a customer). Certain of the amounts noted are known at the time of sale based on contractual terms and, therefore, are recorded pursuant to the most likely amount method under ASC 606. Other amounts are estimated and take into consideration a range of possible outcomes, which are probability-weighted and recorded in accordance with the expected value method in ASC 606 for relevant factors, such as current contractual and statutory requirements, specific known market events and trends, industry data, and forecasted customer buying and payment patterns. Overall, these reserves reflect our best estimates of the amount of consideration to which we are entitled based on the terms of the respective underlying contracts. The amount of variable consideration that is included in the transaction price may be constrained and is included in the net sales price only to the extent that it is probable that a significant reversal in the amount of the cumulative revenue recognized under the contracts with our customers will not occur in a future period. The following are the components of variable consideration related to product revenue: Cash discounts and distributor fees: Product returns: Based on the distribution model for XPOVIO, contractual inventory limits with our customers, the price of XPOVIO, and limited contractual return rights, we currently believe there will be minimal XPOVIO returns. However, we will update our estimated return liability each reporting period based on actual shipments of XPOVIO subject to contractual return rights, changes in expectations about the amount of estimated and/or actual returns, and other qualitative considerations. Chargebacks: Government rebates Other incentives: co-payment co-payments co-payment co-payment Product revenue reserves and allowances: therefore, the transaction price was not reduced further during the year ended December 31, 2019. Actual amounts of consideration ultimately received may differ from our estimates. If actual results in the future vary from our estimates, we will adjust these estimates, which would affect product revenue, net and earnings in the period in which such variances become known. License and Asset Purchase Agreements We generate revenue from license or similar agreements with pharmaceutical companies for the development and commercialization of certain of our product candidates. Such agreements may include the transfer of intellectual property rights in the form of licenses, transfer of technological know-how, non-refundable If a license to our intellectual property is determined to be distinct from the other performance obligations identified in the arrangement, we recognize the transaction price allocated to the license as revenue upon transfer of control of the license. We evaluate all other promised goods or services in the agreement to determine if they are distinct. If they are not distinct, they are combined with other promised goods or services to create a bundle of promised goods or services that is distinct. Optional future services where any additional consideration paid to us reflects their standalone selling prices do not provide the customer with a material right and, therefore, are not considered performance obligations. If optional future services are priced in a manner which provides the customer with a significant or incremental discount, they are material rights, and are accounted for as performance obligations. We utilize judgment to determine the transaction price. In connection therewith, we evaluate contingent milestones at contract inception to estimate the amount which is not probable of a material reversal to include in the transaction price using the most likely amount method. Milestone payments that are not within our control, such as regulatory approvals, are not considered probable of being achieved until those approvals are received and therefore the variable consideration is constrained. The transaction price is then allocated to each performance obligation on a relative stand-alone selling price basis, for which we recognize revenue as or when the performance obligations under the contract are satisfied. At the end of each reporting period, we re-evaluate catch-up We then determine whether the performance obligations or combined performance obligations are satisfied over time or at a point in time and, if over time, the appropriate method of measuring progress for purposes of recognizing revenue from non-refundable, When consideration is received, or such consideration is unconditionally due, from a customer prior to transferring goods or services to the customer under the terms of a contract, a contract liability is recorded within deferred revenue. Contract liabilities within deferred revenue are recognized as revenue after control of the goods or services is transferred to the customer and all revenue recognition criteria have been met. For arrangements that include sales-based royalties, including sales-based milestone payments, and a license of intellectual property that is deemed to be the predominant item to which the royalties relate, we recognize revenue at the later of when the related sales occur or when the performance obligation to which some or all of the royalty has been allocated has been satisfied (or partially satisfied). Accounts Receivable In general, accounts receivable consists of amounts due from customers, net of customer allowances for cash discounts, product returns, and chargebacks. Our contracts with customers have standard payment terms that generally require payment within 30 days for specialty pharmacy customers and 65 days for specialty distributor customers . We analyze accounts that are past due for collectability, and periodically evaluate the creditworthiness of our customers. As of December 31, 2019, we determined an allowance for doubtful accounts was not required based upon our review of contractual payment terms and individual customer circumstances. Inventory Prior to regulatory approval, we expense costs relating to the production of inventory as research and development expense in the period incurred. We capitalize the costs to manufacture our products incurred after regulatory approval when, based on our judgment, future commercialization is considered probable and the future economic benefit is expected to be realized. Such costs are generally recorded as costs of sales upon shipment. In connection therewith, we value our inventories at the lower of cost or estimated net realizable value. We determine the cost of our inventories, which includes amounts related to materials and manufacturing overhead, on a first-in, first-out Prior to FDA approval of XPOVIO, all costs related to the manufacturing of XPOVIO that could potentially be available to support the commercial launch of our products were charged to research and development expense in the period incurred, as there was no alternative future use. We analyze our inventory levels for recoverability each reporting period. In the period in which there is an impairment identified, we write down inventory that has become obsolete, inventory that has a cost basis in excess of its estimated realizable value, and inventory in excess of expected sales requirements as cost of sales. The determination of whether inventory costs will be realizable is based on our estimates. If actual market conditions are less favorable than projected by us, additional write-downs of inventory may be required, which would be recorded as cost of sales. Cost of Sales Cost of sales includes the cost of producing and distributing inventories that are related to product revenue during the respective period, including salary related and stock-based compensation expense for employees involved with production and distribution, freight, and indirect overhead costs, as well as third-party royalties payable on product revenue, net. In addition, shipping and handling costs for product shipments are recorded in cost of sales as incurred. Finally, cost of sales may also include costs related to excess or obsolete inventory adjustment charges, abnormal costs, unabsorbed manufacturing and overhead costs, and manufacturing variances. Deferred Royalty Obligation We treat the liability related to net revenues, as discussed further in Note 15, as a deferred royalty obligation, amortized under the effective interest rate method over the estimated life of the revenue streams. We recognize interest expense thereon using the effective rate, which is based on our current estimates of future revenues over the life of the arrangement. In connection therewith, we periodically assess our expected revenues using internal projections, impute interest on the carrying value of the deferred royalty obligation, and record interest expense using the imputed effective interest rate. To the extent our estimates of future revenues are greater or less than previous estimates or the estimated timing of such payments is materially different than previous estimates, we will account for any such changes by adjusting the effective interest rate on a prospective basis, with a corresponding impact to the reclassification of our deferred royalty obligation. The assumptions used in determining the expected repayment term of the deferred royalty obligation and amortization period of the issuance costs requires that we make estimates that could impact the short-term and long-term classification of such costs, as well as the period over which such costs will be amortized. Research and Development Expenses Research and development costs are charged to expense as incurred and include, but are not limited to: • employee-related expenses, including salaries, benefits, travel and stock-based compensation expense; • expenses incurred under agreements with contract research organizations, contract manufacturing organizations and consultants that help conduct clinical trials and preclinical studies; • the cost of acquiring, developing and manufacturing clinical trial materials, including comparator drugs; • facility, depreciation and other expenses, which include direct and allocated expenses for rent and maintenance of facilities, insurance and other supplies; and • costs associated with preclinical activities and regulatory operations. Costs for certain development activities, such as clinical trials, are recognized based on an evaluation of the progress to completion of specific tasks using data such as patient enrollment, clinical site activations, or information provided to us by our vendors on their actual costs incurred. Payments for these activities are based on the terms of the individual arrangements, which may differ from the pattern of costs incurred, and are accordingly reflected in the financial statements as prepaid or accrued research and development. Comprehensive Loss Comprehensive loss consists of net loss and changes in equity during a period from transactions and other equity and circumstances generated from non-owner Foreign Currency Transactions The functional currency of our subsidiaries in Germany and Israel are the Euro and Shekel, respectively. Foreign currency transaction gains and losses are recorded in the consolidated statement of operations. Net foreign exchange losses of less than $0.1 million were recorded in other income for the years ended December 31, 2019, 2018 and 2017. Income Taxes We use the liability method of accounting for income taxes. Under this method, deferred tax assets and liabilities are determined based on the difference between the financial reporting and the tax reporting basis of assets and liabilities and are measured using the enacted tax rates and laws that are expected to be in effect when the differences are expected to reverse. We provide a valuation allowance against net deferred tax assets unless, based upon the available evidence, it is more likely than not that the deferred tax assets will be realized. We have evaluated available evidence and concluded that we may not realize the benefit of our deferred tax assets; therefore, a valuation allowance has been established for the full amount of the deferred tax assets. We recognize interest and/or penalties related to income tax matters in income tax expense. Our foreign tax provision pertains to foreign income taxes due at our German subsidiary which operates on a cost plus profit margin basis. The Tax Cuts and Jobs Act of 2017 (“TCJA”) resulted in significant changes to the U.S. corporate income tax system. For additional details regarding this act, see Note 14 Accounting for Stock-Based Compensation We account for our stock-based compensation awards in accordance with FASB ASC Topic 718, Compensation—Stock Compensation non-employees, well as modifications to existing stock options and shares issued under our employee stock purchase plan (“ESPP”), to be recognized in the consolidated statements of operations based on their fair values. We use the Black-Scholes option pricing model to determine the fair value of options granted. Compensation expense related to awards to employees and non-employees Net Loss Per Share Basic and diluted net loss per common share is calculated by dividing net loss by the weighted-average number of common shares outstanding for the period, without consideration for common stock equivalents. Our potential dilutive shares, stock options, unvested restricted stock and restricted stock units are considered to be common stock equivalents and are only included in the calculation of diluted net loss per share when their effect is dilutive. The following potentially dilutive securities were excluded from the calculation of diluted net loss per share due to their anti-dilutive effect at December 31, 2019, 2018 and 2017 (in common stock equivalent shares): December 31, 2019 2018 2017 Outstanding stock options 9,843,094 8,917,084 7,019,083 Unvested restricted stock units 787,320 25,000 253,100 We have the option to settle the conversion obligation for our 3.00% convertible senior notes due 2025 (the “Notes”) in cash, shares or any combination of the two. As the Notes are not convertible as of December 31, 2019, they are not participating securities and they will not have an impact on the calculation of basic earnings or loss per share. Based on our net loss position, there is no impact on the calculation of dilutive loss per share during the year ended December 31, 2019. Recently Adopted Accounting Standards In February 2016, the FASB issued ASU No. 2016-02, Leases (Topic 842) 2016-02”), Leases Leases 2016-02, 2018-10, Codification Improvements to Topic 842, Leases 2018-10”) 2018-11, Leases (Topic 842) Targeted Improvements 2018-11”), right-of-use 2018-11 Pursuant to the guidance under ASU 2016-02, which we elected, including: (i) a policy to not record right-of-use non-lease As summarized in the table below, the standard had a material impact on our condensed consolidated balance sheet as of December 31, 2019, specifically through recognition of right-of-use right-of-use right-of-use Please refer to Note 9, “Commitments and Contingencies” for further information January 1, 2019 ASC 842 January 1, 2019 Consolidated balance sheet data (in thousands): Operating lease and right-of-use $ — $ 11,711 $ 11,711 Deferred rent(2) $ 390 $ (390 ) $ — Deferred rent non-current(2) $ 3,922 $ (3,922 ) $ — Operating lease liabilities(3) $ — $ 1,175 $ 1,175 Non-current $ — $ 14,848 $ 14,848 (1) Represents capitalization of operating lease right-of-use right-of-use (2) Represents reclassification of deferred rent and tenant incentives to operating lease right-of-use (3) Represents recognition of operating lease liabilities. We implemented internal controls to enable the preparation of financial information upon adoption. In June 2018, the FASB issued ASU No. 2018-07, Compensation-Stock Compensation (Topic 718): Improvements to Nonemployee Share-Based Payment Accounting 2018-07”). 2018-07 In November 2018, the FASB issued ASU No. 2018-18, Collaborative Arrangements (Topic 808)—Clarifying the Interaction between Topic 808 and Topic 606 2018-18”). 2018-18 2018-18 2018-18 11 In December 2019, the FASB issued ASU No. 2019-12, Simplifying the Accounting for Income Taxes 2019-12”). 740-20-45-7 operations (the so-called Recently Issued Accounting Standards In June 2016, the FASB issued ASU No. 2016-13, Financial Instruments—Credit Losses (Topic 326): Measurement of Credit Losses on Financial Instruments 2016-13”). 2016-13 available-for-sale 2016-13 2016-13 In August 2018, the FASB issued ASU No. 2018-13, Fair Value Measurement—Disclosure Framework-Changes to the Disclosure Requirement for Fair Value Measurement 2018-13”). 2018-13 2018-13 2018-13 In August 2018, the FASB issued ASU No. 2018-15, Intangible-Goodwill and Other Internal-Use Software (Subtopic 350-40) 2018-15”). 2018-15 2018-15 2018-15 |
Property and Equipment, net
Property and Equipment, net | 12 Months Ended |
Dec. 31, 2019 | |
Property, Plant and Equipment [Abstract] | |
Property and Equipment, net | 3. Property and Equipment, net Property and equipment, net consisted of the following (in thousands): December 31, Estimated Useful 2019 2018 Laboratory equipment 4 $ 593 $ 593 Furniture and fixtures 5 654 607 Office and computer equipment 3 598 559 Leasehold improvements Lesser of useful life or lease term 5,443 5,397 7,288 7,156 Less accumulated depreciation and amortization (4,242 ) (3,293 ) $ 3,046 $ 3,863 Depreciation and amortization expense recorded for the years ended December 31, 2019, 2018, and 2017 was $1.0 million, $0.7 million and $0.7 million, respectively. |
Investments
Investments | 12 Months Ended |
Dec. 31, 2019 | |
Investments, Debt and Equity Securities [Abstract] | |
Investments | 4. Investments The following table summarizes our investments in debt securities, classified as available-for-sale Amortized Cost Gross Unrealized Gross Unrealized Fair Value Short-term: Corporate debt securities $ 89,110 $ 12 $ (43 ) $ 89,079 Commercial paper 39,004 18 — 39,022 U.S. government and agency securities 4,990 7 — 4,997 Long-term: Corporate debt securities (one to two year maturity) 2,017 — (1 ) 2,016 $ 135,121 $ 37 $ (44 ) $ 135,114 The following table summarizes our investments in debt securities, classified as available-for-sale Amortized Cost Gross Unrealized Gross Unrealized Fair Value Short-term: Corporate debt securities $ 143,254 $ 3 $ (178 ) $ 143,079 Commercial paper 44,001 — (23 ) 43,978 U.S. government and agency securities 19,131 10 (17 ) 19,124 Certificates of deposit 4,000 — (3 ) 3,997 Long-term: Corporate debt securities (one to two year maturity) 2,007 — (6 ) 2,001 $ 212,393 $ 13 $ (227 ) $ 212,179 At December 31, 2019 and December 31, 2018, we held 27 and 79 debt securities, respectively, that were in an unrealized loss position. The aggregate fair value of debt securities in an unrealized loss position at December 31, 2019 and 2018 was $63.8 million and $180.6 million, respectively. As of December 31, 2019 we did no As of December 31, 2018, 8 corporate debt securities with a fair value of $14.9 million had been in a continuous unrealized loss position for more than 12 months. The unrealized losses of less than $0.1 million related to these corporate debt securities were included in accumulated other comprehensive loss as of December 31, 2018. At December 31, 2018, we did not intend to sell the securities with an unrealized loss position in accumulated other comprehensive income, and it was not likely that we would be required to sell these securities before recovery of their amortized cost basis. We review investments for other-than-temporary impairment whenever the fair value of an investment is less than the amortized cost and evidence indicates that an investment’s carrying amount is not recoverable within a reasonable period of time. Other-than-temporary impairments of investments are recognized in the consolidated statements of operations if we have experienced a credit loss and have the intent to sell the no |
Inventory
Inventory | 12 Months Ended |
Dec. 31, 2019 | |
Inventory Disclosure [Abstract] | |
Inventory | 5. Inventory The following table presents our inventory of XPOVIO at December 31, 2019 and 2018 (in thousands): December 31, 2019 December 31, 2018 Raw materials and work in process $ 273 $ — Finished goods 73 — Total inventory $ 346 $ — At December 31, 2019, all of our inventory was related to XPOVIO, which was approved by the FDA in July 2019, at which time we began to capitalize costs to manufacture XPOVIO. Prior to FDA approval of XPOVIO, all costs related to the manufacturing of XPOVIO and related material were charged to research and development expense in the period incurred. At December 31, 2019, we have determined that a reserve related to XPOVIO inventory is not required. |
Accrued Expenses
Accrued Expenses | 12 Months Ended |
Dec. 31, 2019 | |
Text Block [Abstract] | |
Accrued Expenses | 6. Accrued Expenses Accrued expenses consisted of the following as of December 31, 2019 and 2018 (in thousands): December 31, 2019 2018 Research and development costs $ 13,122 $ 15,903 Payroll and employee-related costs 13,630 10,103 Professional fees 6,172 4,931 I 4,371 — Other 3,583 1,556 $ 40,878 $ 32,493 |
Related Party Transactions
Related Party Transactions | 12 Months Ended |
Dec. 31, 2019 | |
Related Party Transactions [Abstract] | |
Related Party Transactions | 7. Related Party Transactions We paid consulting expenses of $0.2 million for the years ended December 31, 2019 and 2018, for consulting services with certain related parties, including a family member of management and a board member. At December 31, 2019 and 2018, there was less than $0.1 million and $0, respectively, included in accounts payable and accrued expenses due to related parties. |
Stockholders' Equity
Stockholders' Equity | 12 Months Ended |
Dec. 31, 2019 | |
Equity [Abstract] | |
Stockholders' Equity | 8. Stockholders’ Equity Underwritten Offerings On May 7, 2018, we completed a follow-on S-3 333-222726) On April 28, 2017, we completed a follow-on S-3 333-214489) million Controlled Equity Offering Sales Agreement On December 7, 2015, we entered into a Controlled Equity Offering Sales Agreement (as amended on November 7, 2016 and December 1, 2017, the “Sales Agreement”) with Cantor Fitzgerald & Co., as sales agent (“Cantor”), pursuant to which we issued and sold through Cantor an aggregate of 9,172,159 shares of our common stock, for net proceeds of approximately $89.1 million. The Sales Agreement was terminated effective August 12, 2018 “at-the-market” During the year ended December 31, 2018, we did not sell any shares under the Sales Agreement. During the year ended December 31, 2017, we sold an aggregate of 3,405,763 shares under the Sales Agreement for net proceeds of approximately $37.0 million. Open Market Sale Agreement On August 17, 2018, we entered into an Open Market Sale Agreement (the “Open Market Sale Agreement”) with Jefferies LLC, as agent (“Jefferies”), pursuant to which we may issue and sell shares of our common stock having an aggregate offering price of up to $75.0 million (the “Open Market Shares”) from time to time through Jefferies (the “Open Market Offering”). Under the Open Market Sale Agreement, Jefferies may sell the Open Market Shares by methods deemed to be an “at the market offering” as defined in Rule 415(a)(4) promulgated under the Securities Act. We may sell the Open Market Shares in amounts and at times to be determined by us from time to time subject to the terms and conditions of the Open Market Sale Agreement, but we have no obligation to sell any of the Open Market Shares in the Open Market Offering. We or Jefferies may suspend or terminate the offering of Open Market Shares upon notice to the other party and subject to other conditions. We have agreed to pay Jefferies commissions for its services in acting as agent in the sale of the Open Market Shares in the amount of up to 3.0% of gross proceeds from the sale of the Open Market Shares pursuant to the Open Market Sale Agreement. We have also agreed to provide Jefferies with customary indemnification and contribution rights. During the year ended December 31, 2019, we sold an aggregate of 3,712,359 Open Market Shares under the Open Market Sale Agreement, for net proceeds of approximately $46.2 million. |
Commitments and Contingencies
Commitments and Contingencies | 12 Months Ended |
Dec. 31, 2019 | |
Commitments and Contingencies Disclosure [Abstract] | |
Commitments and Contingencies | 9. Commitments and Contingencies Operating Leases We are party to an operating lease of 98,502 square feet of office and research space in Newton, Massachusetts with a term through September 30, 2025 (the “Newton, MA Lease”). Pursuant to the Newton, MA Lease, we have provided a security deposit in the form of a cash-collateralized letter of credit in the amount of $0.6 million. The amount is classified within long-term restricted cash. Upon the adoption of ASU 2016-02, right-of-use 8 right-of-use 2016-02 The Newton, MA Lease provides for increases in future minimum annual rental payments, as defined in the lease agreement. The Newton, MA Lease also includes real estate taxes and common area maintenance (“CAM”) charges in the annual rental payments. As these charges were included in minimum annual rental payments as part of our accounting for the Newton, MA Lease under ASC 840 through December 31, 2018, we have included such amounts in the calculation of the operating lease liability, consistent with ASC 842 and our accounting policy elections thereunder, as specified in Note 2, “Recent Accounting Pronouncements.” The operating lease cost for the Newton, MA Lease for the year ended December 31, 2019 was $2.8 million, of which approximately $0.9 million was charges for CAM. In addition, we are party to short-term leases having a term of twelve months or less at the commencement date. We recognize short-term lease expense on a straight-line basis and do not record a related right-of Lease Commitments As of December 31, 2019, future minimum lease payments under non-cancellable right-of-use Years ending December 31, Future 2020 $ 3,200 2021 3,277 2022 3,447 2023 3,718 2024 and thereafter 6,735 Total minimum lease payments $ 20,377 Less: present value adjustment (5,529 ) Present value of minimum lease payments $ 14,848 As of December 31, 2019, the remaining lease term on the Newton, MA Lease was 5.8 years. The lease has a renewal option for an additional five years, although there is no economic penalty for failure to exercise the option. However, because we did not elect the use of hindsight in estimating the lease term for leases subject to transition to the new standard, and the renewal option was not previously considered in our assessment of the lease term for the Newton, MA Lease before adoption of ASC 842, the renewal option was not considered as part of the lease term in calculating the operating lease right-of-use As a discount rate was not directly observable for our Newton, MA Lease, the discount rate used to calculate the net present value of future payments was our incremental borrowing rate calculated at transition based on the remaining lease term. Upon adoption and through December 31, 2019, the discount rate used to calculate the operating lease liability was 11.0%. The incremental borrowing rate is the rate of interest that we would expect to pay to borrow, on a collateralized basis, over a similar term, an amount equal to the lease payments in a similar economic environment. In determining the incremental borrowing rate, we considered (i) our estimated public credit rating, (ii) our observable debt yields, as well as other bonds in the market issued by other companies with similar credit ratings as us, and (iii) adjustments necessary for collateral, lease term, and inflation or foreign currency. Litigation From time to time we may face legal claims or actions in the normal course of business. We have been named as a defendant in securities class action litigation in the U.S. District Court for the District of Massachusetts. A complaint was filed on July 23, 2019, by the Allegheny County Employees’ Retirement System, against us and certain of our current and former executive officers and directors as well as the underwriters of our public offerings of common stock conducted in April 2017 and May 2018. A second complaint was filed by Heather Mehdi on September 17, 2019, against the same defendants with the exception of the underwriters. The two complaints are related and we expect them to be consolidated by the court. Both complaints allege violations of federal securities laws based on our disclosures related to the results from the Phase 2 SOPRA study and Part 2 of the Phase 2b STORM study, and seek unspecified compensatory damages, including interest; reasonable costs and expenses, including attorneys’ and expert fees; unspecified recessionary damages; and such equitable/injunctive relief or other relief as the court may deem just and proper. We have reviewed the allegations and believe they are without merit. We intend to defend vigorously against this litigation. |
Product Revenue
Product Revenue | 12 Months Ended |
Dec. 31, 2019 | |
Product Revenue [Member] | |
Product Revenue | 10. Product Revenue To date, our only source of product revenue has been from the U.S. sales of XPOVIO, which we began shipping to our customers in July 2019. The following table summarizes activity in each of the product revenue allowance and reserve categories from the date of approval by the FDA through December 31, 2019 (in thousands): Discounts and Fees, Rebates, Returns Total Short-term: Beginning balance at July 3 $ — $ — $ — $ — Provision related to sales in the current year 2,657 2,318 234 5,209 Credit and payments made (1,655 ) (499 ) — (2,154 ) Ending balance at December 31, 2019 $ 1,002 $ 1,819 $ 234 $ 3,055 Discounts, chargebacks, and returns are recorded as reductions of accounts receivable, and fees, rebates, and other incentives are recorded as a component of accrued expenses. |
License and Asset Purchase Agre
License and Asset Purchase Agreements | 12 Months Ended |
Dec. 31, 2019 | |
License and Asset Purchase Agreements [Member] | |
License and Asset Purchase Agreements | 11. License and Asset Purchase Agreements Antengene License Agreement Effective May 23, 2018 (the “Antengene Effective Date”), we entered into a License Agreement (“Antengene License Agreement”) with Antengene Therapeutics Limited, a corporation organized and existing under the laws of Hong Kong (“Antengene”) and a subsidiary of Antengene Corporation Co. Ltd., a corporation organized and existing under the laws of the People’s Republic of China, pursuant to which we granted Antengene exclusive rights to develop and commercialize, at its own cost, (i) selinexor, our lead, novel, oral Selective Inhibitor of Nuclear Export (“SINE”) compound, (ii) eltanexor, our second-generation oral SINE compound, and (iii) KPT-9274, first-in-class non-competitive non-oncology “Non-Oncology KPT-9274 Non-Oncology Pursuant to the terms of the Antengene License Agreement, we received an upfront payment of $11.7 million, and could receive up to $105.0 million in milestone payments if certain development and regulatory non-exclusive As part of the Antengene License Agreement, Antengene will also have the right to participate in global clinical studies of the Antengene Licensed Compounds and will bear the cost and expense for patients enrolled in clinical studies in the Antengene Territory. Antengene is responsible for seeking regulatory and marketing approvals for the Antengene Licensed Compounds in the Antengene Territory, as well as any development of the products specifically necessary to obtain such approvals. Antengene is also responsible for the commercialization of the Antengene Licensed Compounds in the Oncology Field and Non-Oncology The Antengene License Agreement will continue in effect on a product-by-product, country-by-country We assessed the Antengene arrangement in accordance with ASC 606 and concluded that the contract counterparty, Antengene, is a customer. We identified the following material promises under the contract: (i) exclusive licenses for each Antengene Licensed Compound, (ii) initial data transfers for each Antengene Licensed Compound, which consisted of regulatory data compiled by us for the Antengene Licensed Compounds as of the Antengene Effective Date, and (iii) obligations to stand-ready to provide an initial clinical supply for each Antengene Licensed Compound. We also identified immaterial promises under the contract relating to information exchanges and participation on operating committees and other working groups. Separately, we also identified certain customer options that would create an obligation for us if exercised by Antengene, including (i) additional data transfers for each Antengene Licensed Compound, which would consist of the transfer of additional regulatory data compiled by us for each Antengene Licensed Compound after the Antengene Effective Date, (ii) obligations to provide additional clinical supply and related substance supply for each Antengene Licensed Compound upon request by Antengene, (iii) manufacturing technology transfers and licenses for each Antengene Licensed Compound under the Antengene Manufacturing Election, as detailed above, and (iv) options for a backup compound, which represents Antengene’s option to select a replacement compound in the event it elects to discontinue the development of the Antengene Licensed Compounds (the “Antengene Transfer Options”). The Antengene Transfer Options individually represent material rights, as they were offered at a significant and incremental discount. Therefore, they were further assessed as performance obligations under the Antengene License Agreement. Finally, we also identified certain other customer options that would create a manufacturing obligation for us if exercised by Antengene, including for commercial supply. These options do not represent a material right, as they are not offered at a significant and incremental discount. In further evaluating the promises detailed above, we determined that the exclusive licenses, initial data transfers, and stand-ready obligation to provide initial clinical supply for each Antengene Licensed Compound were not distinct from one another, and must be combined as four separate performance obligations (the “Antengene Combined License Obligation for selinexor , , KPT-9274” KPT-9274, We further determined that the up-front KPT-9274 KPT-9274, Upon execution of the Antengene License Agreement, the only fixed component of the transaction price included the $11.7 million up-front and regulatory are achieved and up to $45.0 million in milestone payments if certain sales milestones are achieved, as well as a high single-digit to low double-digit royalty on future net sales of the Antengene Licensed Compounds in the Antengene Territory. In addition, we would receive cost reimbursement in connection with Antengene’s election to receive additional clinical supply for the Antengene Licensed Compounds in the future. We expect to receive payment The future development a nd re-evaluate Through the year ended December 31, 2019, we recognized $9.4 million in revenue under the Antengene License Agreement, as the Antengene Combined License Obligation for selinexor was satisfied when the initial clinical supply of selinexor was delivered during the second quarter of 2019. Revenue will be recognized for the Antengene Combined License Obligation for eltanexor, the Antengene Combined License Obligation for KPT-9274, KPT-9274 Biogen Asset Purchase Agreement On January 24, 2018, we entered into an Asset Purchase Agreement (the “APA”) and Letter Agreement with Biogen MA Inc., a Massachusetts corporation and subsidiary of Biogen, Inc. (“Biogen”). Under the terms of the APA and Letter Agreement, we sold to Biogen exclusive worldwide rights to develop and commercialize our oral SINE compound KPT-350 KPT-350 know-how KPT-350 KPT-350 KPT-350 KPT-350 and regulatory country-by-country We and Biogen have made customary representations and warranties and agreed to customary covenants in the APA, including covenants requiring Biogen to use commercially reasonable efforts to develop KPT-350 We assessed this arrangement in accordance with ASC 606 and concluded that the contract counterparty, Biogen, is a customer. We identified the following material promises in the arrangement: the Transfer of IP and the Manufacturing License. We also identified immaterial promises under the contract that were not deemed performance obligations. We further determined that other promises for Additional Supply and Transition up-front 10.0 10.0 Upon execution of the APA, the transaction price included only the $10.0 million up-front We expect to receive the next milestone payment under this agreement, which is $2.0 million, when the fifth patient in a Phase 1 Multiple Ascending Dose Trial in the United States of a Product in amyotrophic lateral sclerosis is dosed. The future development and regulatory milestones, which represent variable consideration, were evaluated under the most likely amount method, and were not included in the transaction price, because the amounts were fully constrained as of December 31, 2019. As part of our evaluation of the constraint, we considered numerous factors, including that receipt of such milestones is outside our control. Separately, any consideration related to sales-based milestones, as well as royalties on net sales upon commercialization by Biogen, will be recognized when the related sales occur, as they were determined to relate predominantly to the intellectual property and, therefore, have also been excluded from the transaction price in accordance with the sales-based royalty exception, as well as our accounting policy. We will re-evaluate We recognized $10.0 million of revenue during the first quarter of 2018, which was when we had satisfied our promises under the Combined Performance Obligation by transferring the underlying promised goods. Ono License Agreement Effective October 11, 2017 (the “Ono Effective Date”), we entered into a license agreement (the “Ono License Agreement”) with Ono Pharmaceutical Co., Ltd., a corporation organized and existing under the laws of Japan (“Ono”), pursuant to which we granted Ono exclusive rights to develop and commercialize, at its own cost, selinexor and eltanexor, for the diagnosis, treatment and/or prevention of all human oncology indications (the “Ono Field”) in Japan, Republic of Korea, Republic of China (Taiwan) and Hong Kong, as well as in the ten Southeast Asian countries currently comprising the Association of Southeast Asian Nations (the “Ono Territory”) and regulatory non-exclusive As part of the Ono License Agreement, Ono will also have the right to participate in global clinical studies of selinexor and eltanexor and will bear the cost and expense for patients enrolled in clinical studies in the Ono Territory. Ono is responsible for seeking regulatory and marketing approvals for selinexor and eltanexor in the Ono Territory, as well as any development of the products specifically necessary to obtain such approvals. Ono is also responsible for the commercialization of products containing selinexor or eltanexor in the Ono Field in the Ono Territory at its own cost and expense. Subject to the Ono Manufacturing Election, we will furnish clinical supplies of drug substance to Ono for use in Ono’s development efforts pursuant to a clinical supply agreement between us and Ono, and Ono may elect to have us provide commercial supplies of drug product to Ono pursuant to a commercial supply agreement between us and Ono, in each case the costs of which will be borne by Ono. The Ono License Agreement will continue in effect on a product-by-product, country-by-country the Ono License Agreement may be terminated earlier by (i) either party for breach of the Ono License Agreement by the other party or in the event of the insolvency or bankruptcy of the other party, product-by-product product-by-product, country-by-country We assessed this arrangement in accordance with ASC 606 and concluded that the contract counterparty, Ono, is a customer. We identified the following material promises under the contract: (i) the Ono Exclusive License for selinexor and eltanexor, (ii) initial data transfer for selinexor and eltanexor, which consisted of regulatory data compiled by us for the licensed compounds and products as of the Ono Effective Date, (iii) initial clinical supply for selinexor, which consisted of units of clinical supply for Ono to conduct its Phase I Trial, and (iv) an obligation to stand-ready to provide initial clinical supply for eltanexor. We also identified immaterial promises under the contract relating to information exchanges, and participation on operating committees and other working groups. Separately, we also identified certain customer options that would create an obligation for us if exercised by Ono, including the (i) additional data transfer for selinexor and eltanexor, which would consist of the transfer of additional regulatory data compiled by us for the licensed compounds and products after the Ono Effective Date, (ii) additional clinical supply and related substance supply for selinexor and eltanexor, which would consist of supplying Ono with units and substance of selinexor and eltanexor incremental to the initial clinical supply for selinexor and the obligation to stand-ready to provide initial clinical supply for eltanexor, as noted above, (iii) manufacturing technology transfer and license for selinexor and eltanexor under the Ono Manufacturing Election, as detailed above, and (iv) options for a backup compound, which represents Ono’s option to select a replacement compound in the event it elects to discontinue the development of either of the licensed compounds (the “Ono Transfer Options”). The Ono Transfer Options individually represent material rights, as they were offered at a significant and incremental discount. Therefore, they were further assessed as performance obligations under the Ono License Agreement. We also identified certain other customer options that would create a manufacturing obligation for us if exercised by Ono, including commercial supply. This option is referred to herein as the “Ono Manufacturing Option.” The Ono Manufacturing Option does not represent a material right, as it is not offered at a significant and incremental discount. In further evaluating the promises detailed above, we determined that the (i) Ono Exclusive License, initial data transfer, and initial clinical supply for selinexor and (ii) Ono Exclusive License, initial data transfer, and obligation to stand-ready to provide initial clinical supply of eltanexor were not distinct from one another, and must be combined as two separate performance obligations (the “Ono Combined License Obligation for selinexor” and the “Ono Combined License Obligation for eltanexor”). This is because, for both selinexor and eltanexor, Ono requires the initial data transfer and clinical supply to derive benefit from the Ono Exclusive License since we did not grant manufacturing licenses for selinexor and eltanexor at contract inception. We also determined that each of the Ono Transfer Options represents a distinct performance obligation. Based on these determinations, we identified six distinct performance obligations at the inception of the Ono License Agreement, including (i) the Ono Combined License Obligation for selinexor, (ii) the Ono Combined License Obligation for eltanexor, and the four components of the Ono Transfer Options, including (iii) the material right for additional data transfer, (iv) the material right for additional clinical supply and related substance supply, (iv) the material right for manufacturing technology transfer and license, and (vi) the material right for the option for a backup compound. We further determined that the up-front stand-alone selling prices for any of the identified performance obligations would not have a significant effect on the allocation of the underlying transaction price to the performance obligations. Upon execution of the Ono License Agreement, the transaction price included only the ¥2.5 billion (US$21.9 million on the date received) up-front and regulatory expect to receive milestone payment under this agreement upon the first licensed product in Multiple , of ¥ billion (approximately US$ million), in Japan. The future development and regulatory milestones and cost reimbursement for providing initial clinical supply of eltanexor, both of which represent variable consideration, were evaluated under the most likely amount method, and were not included in the transaction price, because the amounts were fully constrained as of December 31, 2019. As part of our evaluation of the constraint, we considered numerous factors, including that receipt of such amounts is outside our control. Separately, any consideration related to sales-based milestones, as well as royalties on net sales upon commercialization by Ono, will be recognized when the related sales occur, as they were determined to relate predominantly to the intellectual property granted to Ono and, therefore, have also been excluded from the transaction price in accordance with the sales-based royalty exception, as well as our accounting policy. We will re-evaluate As the initial clinical supply of selinexor was delivered in April 2018, the Ono Combined License Obligation for selinexor was determined to be fulfilled and revenue of $19.7 million was recognized during the quarter ended June 30, 2018. The transaction price allocated to the Ono Combined License Obligation for eltanexor will be recognized as revenue once our stand-ready promise to provide initial clinical supply of eltanexor in the future is fulfilled, which is the last remaining undelivered promise associated with the Ono Combined License Obligation for eltanexor. As of December 31, 2019, $2.2 million of the Ono License Agreement upfront payment is included in deferred revenue and is classified as a non-current |
Stock-based Compensation
Stock-based Compensation | 12 Months Ended |
Dec. 31, 2019 | |
Disclosure of Compensation Related Costs, Share-based Payments [Abstract] | |
Stock-based Compensation | 12. Stock-based Compensation During 2010, we established the 2010 Stock Incentive Plan (the “Plan” or the “2010 Plan”). Under the terms of the Plan, we granted options to our employees, officers, directors, consultants and advisors. The exercise price of each stock option is the fair market value as determined in good faith by the Board of Directors (the Board) at the time each option is granted. We granted service-based options under the Plan, which generally vest as follows: 25% of the shares vest one calendar year from the vesting start date, 2.083% of the shares vest on the first day of each month for the three years thereafter. The options granted under the Plan generally expire in 10 years from the date of grant. We will grant no further stock options or other awards under the 2010 Plan. In October 2013, the Board adopted and our stockholders approved the 2013 Stock Incentive Plan (the “2013 Plan”). The 2013 Plan became effective immediately prior to the closing of the IPO and provides for the grant of incentive stock options, nonstatutory stock options, stock appreciation rights, restricted stock awards, restricted stock unit awards and other stock-based awards. The number of shares of common stock reserved for issuance under the 2013 Plan is equal to the sum of (1) 969,696 shares plus (2) the number of shares (up to 2,126,377 shares) equal to the sum of the number of shares of common stock then available for issuance under the 2010 Plan and the number of shares of common stock subject to outstanding awards under the 2010 Plan that expire, terminate or are otherwise surrendered, cancelled, forfeited or repurchased by us at their original issuance price pursuant to a contractual repurchase right plus (3) an annual increase, to be added on the first day of each fiscal year, beginning with the fiscal year ending December 31, 2014 and continuing until, and including, the fiscal year ending December 31, 2023, equal to the lesser of (A) 1,939,393 shares of common stock, (B) 4% of the number of shares of common stock outstanding on the first day of such fiscal year, or (C) an amount determined by the Board. In January 2019, 2018 and 2017, the number of shares available for issuance under the 2013 Plan was increased by 1,939,393, 1,939,393 and 1,675,513 shares of common stock, respectively. As of December 31, 2019, we had 2,373,779 shares available for issuance under the 2013 Plan. In connection with all share-based payment awards, total stock-based compensation expense recognized was as follows (in thousands): Year Ended December 31, 2019 2018 2017 Research and development $ 6,457 $ 8,686 $ 11,208 Selling, general and administrative 8,834 8,589 9,197 Total $ 15,291 $ 17,275 $ 20,405 Stock Options The total stock-based compensation non-employee The following table summarizes stock option activity for employees and nonemployees: Options Weighted- Weighted- Aggregate (in Options outstanding at December 31, 2018 8,917,084 $ 13.78 7.4 $ 8,197 Granted 3,325,390 8.67 Exercised (394,358 ) 7.18 Forfeited (2,005,022 ) 13.36 Options outstanding at December 31, 2019 9, 843,094 $ 12.40 7.0 $ 82,134 Options exercisable at December 31, 2019 5,391,153 $ 14.65 5.6 $ 39,879 The total intrinsic value of stock options exercised for the years ended December 31, 2019, 2018 and 2017 was $2.2 million, $6.0 million and $0.4 million, respectively. The fair value of each stock option granted to employees is estimated on the date of grant and for non-employees Years Ended December 31, 2019 2018 2017 Volatility 79%-81% 79%-81% 79%-85% Expected term (in years) 5.5-6.0 5.5-9.8 5.5-9.6 Risk-free interest rate 1.42%-2.58% 2.50%-3.05% 1.76%-2.29% Dividend —% —% —% We use the simplified method as prescribed by the Securities and Exchange Commission Staff Accounting Bulletin No. 107, Share-Based Payment non-employees. ours including enterprise value, risk profiles, position within the industry, and with historical share price information sufficient to meet the expected term of the options. The risk-free interest rate is based on a treasury instrument whose term is consistent with the expected term of the stock options. We account for forfeitures as they occur. Using the Black-Scholes option-pricing model, the weighted-average grant date fair values of options granted during the years ended December 31, 2019, 2018 and 2017 was $6.01, $8.91 and $7.22 per share, respectively. At December 31, 2019, the total unrecognized compensation related to unvested employee and non-employee Restricted Stock Units A restricted stock unit (“RSU”) represents the right to receive one share of our common stock upon vesting of the RSU. The fair value of each RSU is based on the closing price of our common stock on the date of grant. We grant RSUs with service conditions that vest in or equal annual installments provided that the employee remains employed with us. During the year ended December 31, 2019, we granted 1,065,970 Number of Weighted-Average Unvested at December 31, 2018 25,000 $ 9.87 Granted 1,065,970 9.25 Forfeited (286,150 ) 9.24 Vested (17,500 ) 8.96 Unvested at December 31, 2019 787,320 $ 9.28 The total stock-based compensation expense related to RSUs for the years ended December 31, 2019, 2018 and 2017 was $1.6 million, $0.4 million and $ As of December 31, 2019, there was $5.8 million of unrecognized compensation costs related to unvested RSUs, which are expected to be recognized over a weighted average period of 3.12 years. Employee Stock Purchase Plan We have an Employee Stock Purchase Plan (“ESPP”) that permits eligible employees to enroll in six-month six-month In 2013, our shareholders approved an increase in the number of shares of common stock authorized for issuance pursuant to the ESPP to 242,424 shares of common stock, plus an annual increase to be added on the first day of each fiscal year, commencing on January 1, 2015 and ending on December 31, 2023, equal to the lesser of 484,848 shares of our common stock, 1% of the number of outstanding shares on such date, or an amount determined by the Board. During the years ended December 31, 2019, 2018 and 2017, $1.7 million, $0.9 million and 0.4 million, respectively, was withheld from employees, on an after-tax 57,582 shares of our common stock, respectively. For the years ended December 31, 2019, 2018 and 2017, we recorded stock-based compensation expense related to the ESPP our Years Ended 2019 2018 2017 Volatility 61 % 48%-61% 48%-78% Expected term (in years) 0.5 0.5 0.5 Risk-free interest rate 2.4 % 1.3%-2.1% 0.5%-1.3% Dividend —% —% —% |
401(k) Plan
401(k) Plan | 12 Months Ended |
Dec. 31, 2019 | |
Retirement Benefits [Abstract] | |
Pension and Other Postretirement Benefits Disclosure [Text Block] | 13. 401(k) Plan We have a 401(k) retirement and profit-sharing plan (the “401(k) Plan”) covering all qualified employees. The 401(k) Plan allows each participant to contribute a portion of their base wages up to an amount not to exceed an annual statutory maximum. Effective January 1, 2011, we adopted a Safe Harbor Plan that provides a Company match up to 4% of salary. We contributed a match of $1.7 million, $1.1 million and $0.6 million to the 401(k) Plan for the years ended December 31, 2019, 2018 and 2017, respectively. |
Income Taxes
Income Taxes | 12 Months Ended |
Dec. 31, 2019 | |
Income Tax Disclosure [Abstract] | |
Income Taxes | 14. Income Taxes New Tax Legislation On December 22, 2017, the President of the United States signed into law the Tax Cuts and Jobs Act (“TCJA”). This legislation reduced the U.S. corporate tax rate from the current rate of 34% to 21% for tax years beginning after December 31, 2017. As a result of the enacted law, we were required to revalue deferred tax assets and liabilities existing as of December 31, 2017 from the 34% federal rate in effect through the end of 2017, to the new 21% rate. We have recognized the impact of the TCJA in these consolidated financial statements and related disclosures. In the year ended December 31, 2018, we recorded an impact of $0.5 million related to the return to provision items for federal rate change, which is offset by a full valuation allowance. The impact of the remeasurement of our U.S. deferred tax assets and liabilities to 21% resulted in the reduction of deferred tax assets of approximately $42.7 million, which is offset by a full valuation allowance. There was no impact to our income statement due to the reduction in the U.S. corporate tax rate. Income Taxes For the years ended December 31, 2019, 2018 and 2017, we recorded an income tax expense of less than $ million for our operations in Germany. Our foreign tax provision pertains to foreign income taxes due at our German subsidiary which operates on a cost-plus profit margin. The components of loss before income taxes were as follows (in thousands): Year Ended 2019 2018 2017 Foreign $ (23,350 ) $ (28,689 ) $ (35,680 ) U.S. (176,200 ) (149,692 ) (93,241 ) Totals $ (199,550 ) $ (178,381 ) $ (128,921 ) Deferred taxes are recognized for temporary differences between the basis of assets and liabilities for financial statement and income tax purposes. The significant components of our deferred tax assets are comprised of the following (in thousands): Year Ended 2019 2018 Deferred tax assets: U.S. and state net operating loss carryforwards $ 150,909 $ 116,236 Stock-based compensation 11,914 11,228 Accruals and other temporary differences 4,212 3,605 Research and development credits 62,374 49,307 Capitalized research and development 1,021 1,388 Fixed assets and intangibles 5,893 6,422 Deferred revenue 1,051 527 Foreign net operating loss carryforwards 4 72 Lease liability 3,443 — Deferred royalty embedded derivative 533 — Valuation allowance (224,943 ) (173,247 ) Total deferred tax assets 16,411 15,538 Deferred tax liabilities: Convertible debt amortization (13,431 ) (15,538 ) Right-of-use asset (2,462 ) — Deferred royalty obligation (518 ) — Total deferred tax liabilities (16,411 ) (15,538 ) Net deferred tax assets $ — $ — We have evaluated the positive and negative evidence bearing upon the realizability of our deferred tax assets. Based on our history of operating losses, we have concluded that it is more likely than not that the benefit of our deferred tax assets will not be realized. Accordingly, we have provided a full valuation allowance for deferred tax assets as of December 31, 2019 and 2018. The valuation allowance increased approximately $51.7 million during the year ended December 31, 2019 to $224.9 million, from $173.2 million during the year ended December 31, 2018, primarily due to the generation of net operating losses. A reconciliation of income tax expense computed at the statutory federal income tax rate to income taxes as reflected in the financial statements is as follows: Year Ended 2019 2018 2017 Federal income tax expense at statutory rate 21.0 % 21.0 % 34.0 % State income tax, net of federal benefit 1.7 % 0.7 % 5.9 % Permanent differences (0.5 )% — % (3.9 )% Research and development credit 6.0 % 7.8 % 8.4 % Foreign rate differential (2.5 )% (3.4 )% (9.4 )% Change in valuation allowance (25.9 )% (28.2 )% (1.4 )% Provision to return adjustments 0.6 % 2.4 % 1.0 % Other (0.4 )% (0.6 )% (1.4 )% Federal rate change — % 0.3 % (33.2 )% Effective income tax rate — % — % — % As of December 31, 2019 and 2018, we had U.S. federal net operating loss carryforwards of approximately $576.5 million and $427.0 million, respectively, which may be able to offset future income tax liabilities. Of the $ million carryforward as of December 31, 2019, $ million of the carryforward has an indefinite life and $ million will expire at various dates through 2037. As of December 31, 2019 and 2018, we had U.S. state net operating loss carryforwards of approximately $502.3 million and $ million, respectively, which may be available to offset future state income tax liabilities and expire at various dates through 203 9 $ million, which have an indefinite li fe and As of December 31, 2019 and 2018, we had federal research and development tax credit carryforwards of approximately $ 58.5 million and $ million, respectively, available to reduce future tax liabilities, which expire at various dates through 203 9 million and $ million, respectively, available to reduce future tax liabilities, which expire at various dates through 203 4 Under the provisions of the Internal Revenue Code, the net operating loss and tax credit carryforwards are subject to review and possible adjustment by the Internal Revenue Service and state tax authorities. Net operating loss and tax credit carryforwards may become subject to an annual limitation in the event of certain cumulative changes in the ownership interest of significant shareholders over a three which will reduce our deferred tax assets for tax attributes we believe will expire unused due to the change in control limitations. In October 2016 the FASB issued ASU 2016-16. We will recognize interest and penalties related to uncertain tax positions in income tax expense. As of December 31, 2019 and 2018, we had no accrued interest or penalties related to uncertain tax positions and no such amounts have been recognized. We or one of our subsidiaries file income tax returns in the United States, and various state and foreign jurisdictions. The federal, state and foreign income tax returns are generally subject to tax examinations for the tax years ended December 31, 2016 through December 31, 2019. To the extent we have tax attribute carryforwards , the tax years in which the attribute was generated may still be adjusted upon examination by the Internal Revenue Service, state or foreign tax authorities to the extent utilized in a future period. |
Long-Term Obligations
Long-Term Obligations | 12 Months Ended |
Dec. 31, 2019 | |
3% Convertible Senior Notes Due 2025 [Member] | |
Long-Term Obligations | 15. Long-term obligations 3.00% Convertible Senior Notes due 2025 On October 16, 2018, we completed an offering of $150.0 million aggregate principal amount of our 3.00% convertible senior notes due 2025 (the “Notes”). In addition, on October 26, 2018, we issued an additional $22.5 million aggregate principal amount of the Notes pursuant to the full exercise of the option to purchase additional Notes granted to the initial purchasers in the offering. The Notes were sold in a private offering to qualified institutional buyers in reliance on Rule 144A under the Securities Act. In accordance with accounting guidance for debt with conversion and other options, we separately accounted for the liability component (“Liability Component”) and the embedded conversion option (“Equity Component”) of the Notes by allocating the proceeds between the Liability Component and the Equity Component, due to our ability to settle the Notes in cash, shares of our common stock or a combination of cash and shares of our common stock, at our option. In connection with the issuance of the Notes, we incurred approximately $5.6 million of debt issuance costs, which primarily consisted of underwriting, legal and other professional fees, and allocated these costs between the Liability Component and the Equity Component based on the allocation of the proceeds. Of the total debt issuance costs, $2.2 million was allocated to the Equity Component and recorded as a reduction to additional paid-in The Notes are our senior unsecured obligations and bear interest at a rate of 3.00% per year payable semiannually in arrears on April 15 and October 15 of each year, beginning on April 15, 2019. Upon conversion, the Notes will be convertible into cash, shares of our common stock or a combination of cash and shares of our common stock, at our election. (1) during any calendar quarter commencing after the calendar quarter ending on December 31, 2018 (and only during such calendar quarter), if the last reported sale price of our common stock for at least 20 trading days (whether or not consecutive) during the period of 30 consecutive trading days ending on, and including, the last trading day of the immediately preceding calendar quarter is greater than or equal to 130% of the conversion price for the Notes on each applicable trading day; (2) during the five business day period immediately after any five consecutive trading day (3) if we call the Notes for redemption, until the close of business on the business day immediately preceding the redemption date; or (4) upon the occurrence of specified corporate events as described within the indenture governing the Notes. We may not redeem the Notes prior to October 15, 2022. On or after October 15, 2022, we may redeem for cash all or part of the Notes at our option if the last reported sale price of our common stock equals or exceeds 130% of the conversion price then in effect for at least 20 trading days (whether or not consecutive) during any 30 consecutive trading day period ending within five trading days prior to the date on which we send any notice of redemption. The redemption price will be 100% of the principal amount of the Notes to be redeemed, plus accrued and unpaid interest, if any. In addition, calling any convertible note for redemption will constitute a make-whole fundamental change with respect to that convertible note, in which case the conversion rate applicable to the conversion of that convertible note, if it is converted in connection with the redemption, will be increased in certain circumstances. The initial carrying amount of the Liability Component of $101.2 million was calculated by measuring the fair value of a similar liability that does not have an associated convertible feature. The allocation was performed in a manner that reflected our non-convertible The outstanding balances of the Notes as of December 31, 2019 consisted of the following (in thousands): Liability component: Principal $ 172,500 Less: debt discount and issuance costs, net (62,643 ) Net carrying amount $ 109,857 Equity component: $ 65,641 We determined the expected life of the Notes was equal to its seven-year term. The effective interest rate on the Liability Component of the Notes was 11.85%. As of December 31, 2019, the “if-converted exceed the remaining principal amount of the Notes. The fair value of the Notes was determined based on data points other than quoted prices that are observable, either directly or indirectly, and has been classified as Level 2 within the fair value hierarchy. The fair value of the Notes, which differs from their carrying value, is influenced by market interest rates, our stock price and stock price volatility. The estimated fair value of the Notes as of December 31, 2019 was approximately $251.0 million. The following table sets forth total interest expense recognized related to the Notes during the year ended December 31, 2019 (in thousands): Year Ended Contractual interest expense $ 5,175 Amortization of debt discount 6,849 Amortization of debt issuance costs 344 Total interest expense $ 12,368 Future minimum payments on the Notes as of December 31, 2019 were as follows (in thousands): Years ended December 31, Future Minimum Payments 2020 $ 5,175 2021 5,175 2022 5,175 2023 5,175 2024 and thereafter 182,850 Total minimum payments $ 203,550 Less: interest (31,050 ) Less: unamortized discount (62,643 ) Less: current portion — Convertible senior notes $ 109,857 Deferred Royalty Obligation In September 2019, we entered into a Revenue Interest Financing Agreement (”deferred royalty obligation”) with HealthCare Royalty Partners III, L.P. and HealthCare Royalty Partners IV, L.P. (“HCR”) whereby HCR will receive payments from us at a tiered percentage (the “Applicable Tiered Percentage”) of future net revenues of XPOVIO and any of our other future products, including worldwide net product sales and upfront payments, milestones, and royalties. We received $75.0 million upon closing (the “First Investment Amount”) and have the right to receive an additional $75.0 million (the “Second Investment Amount” and together with the First Investment Amount, the “Investment Amount”) upon the achievement of future regulatory and commercial milestones and subject to the approval of both parties and customary closing conditions. In exchange for the First Investment Amount, HCR will receive a tiered royalty in the mid-single If HCR has not received 65% of the Investment Amount by December 31, 2022 or 100% of the Investment Amount by December 31, 2024, we must make a cash payment sufficient to gross HCR up to such minimum amounts. As the repayment of the funded amount is contingent upon worldwide net product sales and upfront payments, milestones, and royalties, the repayment term may be shortened or extended depending on actual worldwide net product sales and upfront payments, milestones, and royalties. The repayment period commenced on October 1, 2019 and expires on the earlier of (i) the date in which HCR has received cash payments totaling an aggregate of 185% of the Investment Amount or (ii) the legal maturity date of October 1, 2031. If HCR has not received payments equal to 185% of the Investment Amount by the twelve-year anniversary of the initial closing date, we shall pay an amount equal to the Investment Amount plus a specific annual rate of return less payments previously received by HCR. In the event of a change of control, we are obligated to pay HCR an amount equal to 185% of the Investment Amount less payments previously received by HCR. In addition, upon the occurrence of an event of default, including, among others, our failure to pay any amounts due to HCR under the deferred royalty obligation, insolvency, our failure to pay indebtedness when due, the revocation of regulatory approval of XPOVIO in the United States or our breach of any covenant contained in the Revenue Interest Financing Agreement and our failure to cure the breach within the prescribed time frame, we are obligated to pay HCR an amount equal to 185% of the Investment Amount less payments previously received by HCR. In addition, upon an event of default, HCR may exercise all other rights and remedies available under the Revenue Interest Financing Agreement, including foreclosing on the collateral that was pledged to HCR, which consists of all of our present and future assets relating to XPOVIO. We have evaluated the terms of the deferred royalty obligation and concluded that the features of the Investment Amount are similar to those of a debt instrument. Accordingly, we have accounted for the transaction as long-term debt. We have further evaluated the terms of the debt and determined that the repayment of 185% of the Investment Amount, less any payments made to date, upon a change of control is an embedded derivative that requires bifurcation from the debt instrument and fair value recognition. We determined the fair value of the derivative using an option pricing Monte Carlo simulation model taking into account the probability of change of control occurring and potential repayment amounts and timing of such payments that would result under various scenarios, as further described in Note 2. The aggregate fair value of the embedded derivative at issuance date is included in deferred royalty obligation. We will remeasure the embedded derivative to fair value each reporting period until the time the features lapse and/or termination of the deferred royalty obligation. The effective interest rate as of December 31, 2019 was 18.4 %. In connection with the deferred royalty obligation, we incurred debt issuance costs totaling $ million. Debt issuance costs have been netted against the debt as of December 31, 2019 and are being amortized over the estimated term of the debt using the effective interest method, adjusted on a prospective basis for changes in the underlying assumptions and inputs. The assumptions used in determining the expected repayment term of the debt and amortization period of the issuance costs requires that we make estimates that could impact the short and long-term classification of these costs, as well as the period over which these costs will be amortized. The carrying value of the deferred royalty obligation at December 31, 2019 was $ million based on $ million of proceeds, net of fair value of the bifurcated embedded derivative liability and debt issuance costs incurred. The carrying value of the deferred royalty obligation approximates fair value at December 31, 2019 and was measured using Level 3 inputs. The estimated fair market value was calculated using an option pricing Monte Carlo simulation model with inputs consistent with those used in determining the embedded derivative values as described in Note 2. |
Summary of Significant Accoun_2
Summary of Significant Accounting Policies (Policies) | 12 Months Ended |
Dec. 31, 2019 | |
Accounting Policies [Abstract] | |
Basis of Presentation | Basis of Presentation The accompanying financial statements have been prepared in accordance with accounting principles generally accepted in the United States of America (“U.S. GAAP”). |
Segment Information | Segment Information Operating segments are defined as components of an enterprise about which separate discrete information is available for evaluation by the chief operating decision maker, or decision-making group, in deciding how to allocate resources and in assessing performance. We view our operations and manage our business in one operating segment, which is the business of discovering, developing and commercializing drugs to treat cancer and certain other major diseases. All of our revenue to date has been derived in the United States. All of our material long-lived assets reside in the United States. |
Use of Estimates | Use of Estimates The preparation of financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities and the disclosure of contingent assets and liabilities at the date of the financial statements and the reported amounts of revenues and expenses during the reporting period. On an ongoing basis, we evaluate our estimates, including estimates related to our net product revenue, clinical trial accruals, stock-based compensation expense, interest expense on our deferred royalty obligation and |
Principles of Consolidation | Principles of Consolidation The consolidated financial statements at December 31, 2019 include the accounts of (i) Karyopharm Therapeutics Inc., (ii) Karyopharm Securities Corp. (“KPSC”, our wholly-owned Massachusetts corporation incorporated in December 2013), (iii) Karyopharm Europe GmbH (our wholly-owned German Limited Liability Company, incorporated in September 2014), (iv) Karyopharm Therapeutics (Bermuda) Ltd. (our limited liability company, registered in Bermuda in March 2015), and (vi) Karyopharm Israel Ltd. (our wholly - |
Cash and Cash Equivalents | Cash and Cash Equivalents Cash and cash equivalents consist primarily of demand deposit accounts and deposits in short-term money market funds. Cash equivalents are stated at cost, which approximates fair value. We consider all highly liquid investments with maturities of three months or less from the date of purchase to be cash equivalents. We do not hold any money market funds with significant liquidity restrictions that would be required to be excluded from cash equivalents. |
Investments | Investments We determine the appropriate classification of our investments in debt securities at the time of purchase. All of our securities are classified as available-for-sale Available-for-sale |
Concentrations of Credit Risk and Off-Balance Sheet Risk | Concentrations of Credit Risk and Off-Balance Financial instruments which potentially subject us to credit risk consist primarily of cash, cash equivalents and investments. We hold these investments in highly rated financial institutions, and, by policy, limit the amounts of credit exposure to any one financial institution. These amounts at times may exceed federally insured limits. We have not experienced any credit losses in such accounts and do not believe we are exposed to any significant credit risk on these funds. We have no off-balance |
Fair Value Measurements | Fair Value Measurements Financial instruments, including cash, restricted cash, prepaid expenses and other current assets, accounts payable and accrued expenses, are presented at amounts that approximate fair value at December 31, 2019 and 2018. We are required to disclose information on all assets and liabilities reported at fair value that enables an assessment of the inputs used in determining the reported fair values. The fair value hierarchy prioritizes valuation inputs based on the observable nature of those inputs. The fair value hierarchy applies only to the valuation inputs used in determining the reported fair value of the investments and is not a measure of the investment credit quality. The hierarchy defines three levels of valuation inputs: Level 1 inputs Quoted prices in active markets for identical assets or liabilities Level 2 inputs Inputs other than quoted prices included within Level 1 that are observable for the asset or liability, either directly or indirectly Level 3 inputs Unobservable inputs that reflect our own assumptions about the assumptions market participants would use in pricing the asset or liability Our cash equivalents are composed of money market funds. We measure these investments at fair value. The fair value of cash equivalents is determined based on “Level 1” inputs. Items classified as Level 2 within the valuation hierarchy consist of commercial paper, corporate debt securities, U.S. government agency securities and certificates of deposit. We estimate the fair values of these marketable securities by taking into consideration valuations obtained from third-party pricing sources. These pricing sources utilize industry standard valuation models, including both income and market-based approaches, for which all significant inputs are observable, either directly or indirectly, to estimate fair value. These inputs include market pricing based on real-time trade data for the same or similar securities, issuer credit spreads, benchmark yields, and other observable inputs. We validate the prices provided by our third-party pricing sources by understanding the models used, obtaining market values from other pricing sources and analyzing pricing data in certain instances. In certain cases where there is limited activity or less transparency around inputs to valuation, the related assets or liabilities are classified as Level 3. The embedded derivative liability associated with our deferred royalty obligation, as discussed further in Note 1 5 5 The following table presents information about our financial assets and liability that have been measured at fair value at December 31, 2019 and indicates the fair value hierarchy of the valuation inputs utilized to determine such fair value (in thousands): Description Total Quoted Prices Significant Significant Financial assets Cash equivalents: Money market funds $ 71,380 $ 71,380 $ — $ — Investments: Short-term: Corporate debt securities 89,079 — 89,079 — Commercial paper 39,022 — 39,022 — U.S. government and agency securities 4,997 — 4,997 — Long-term: Corporate debt securities (one to two year maturity) 2,016 — 2,016 — $ 206,494 $ 71,380 $ 135,114 $ — Financial liability Embedded derivative liability $ 2,300 $ — $ — $ 2,300 The following table presents information about our financial assets that have been measured at fair value at December 31, 2018 and indicates the fair value hierarchy of the valuation inputs utilized to determine such fair value (in thousands): Description Total Quoted Prices Significant Significant Financial assets Cash equivalents: Money market funds $ 76,881 $ 76,881 $ — $ — Investments: Short-term: Corporate debt securities 143,079 — 143,079 — Commercial paper 43,978 — 43,978 — U.S. government and agency securities 19,124 — 19,124 — Certificate of deposit 3,997 — 3,997 — Long-term: Corporate debt securities (one to two year maturity) 2,001 — 2,001 — $ 289,060 $ 76,881 $ 212,179 $ — The following table sets forth a summary of the changes in the estimated fair value of our embedded derivative liability during the year ended December 31, 2019 (in thousands): Embedded Balance as of December 31, 2018 $ — Addition of derivative related to deferred royalty obligation . 2,300 Change in fair value of derivative since issuanc e — Balance as of December 31, 2019 $ 2,300 Our L 5 |
Property and Equipment, net | Property and Equipment, net Property and equipment are recorded at cost, less accumulated depreciation. Depreciation is recorded using the straight-line method over the estimated useful lives of the respective assets, generally three |
Long-Lived Assets | Long-Lived Assets We review the carrying values of our long-lived assets for possible impairment whenever events or changes in circumstances indicate that the carrying amounts of the assets may not be recoverable. Any long-lived assets held for disposal are reported at the lower of their carrying amounts or fair values less costs to sell. We have not recorded an impairment in any period since inception. |
Deferred Rent | Deferred Rent Deferred rent consists of rent escalation payment terms, tenant improvement allowances and other incentives received from landlords related to our operating leases under Financial Accounting Standards Board (“FASB”) Accounting Standards Codification (“ASC”) 840. Rent escalation represents the difference between actual operating lease payments due and straight-line rent expense. Tenant improvement allowances and other incentives were also recorded as deferred rent under ASC 840 through December 31, 2018. Deferred rent and lease incentives are recorded as a reduction to our operating lease right-of-use |
Revenue Recognition | Revenue Recognition We adopted Accounting Standards Update (“ASU”) 2014-09, Revenue from Contracts with Customers ASC 606 applies to all contracts with customers, except for contracts that are within the scope of other standards, such as leases, insurance, collaboration arrangements, and financial instruments. Under ASC 606, we recognize revenue when our customer obtains control of promised goods or services, in an amount that reflects the consideration which we expect to receive in exchange for those goods or services. To determine revenue recognition for arrangements that we determine are within the scope of ASC 606, we perform the following five steps: (i) identify the contract(s) with a customer; (ii) identify the performance obligations in the contract; (iii) determine the transaction price; (iv) allocate the transaction price to the performance obligations in the contract; and (v) recognize revenue when (or as) we satisfy a performance obligation. At contract inception, once the contract is determined to be within the scope of ASC 606, we assess the goods or services promised within each contract and determine those that are performance obligations and assess whether each promised good or service is distinct. We then recognize as revenue the amount of the transaction price that is allocated to the respective performance obligation when (or as) the performance obligation is satisfied. |
Product Revenue Recognition | Product Revenue Recognition In the third quarter of 2019, we began to ship XPOVIO in the United States to specialty pharmacies and specialty distributors, collectively referred to as our customers, under a limited number of distribution arrangements with such third parties. Our specialty pharmacy customers resell XPOVIO directly to patients while our specialty distributor customers resell XPOVIO to healthcare entities, who then resell to patients. In connection with negotiating and executing contracts with our customers, our policy is to expense incremental costs of obtaining a contract when incurred, if the expected amortization period of the asset that we would have recognized is one year or less. However, no such costs have been incurred to date. In addition to distribution agreements with our customers, we enter into certain arrangements with group purchasing organizations and/or other payors that provide for government mandated and/or privately negotiated rebates, chargebacks, and discounts with respect to the purchase of our products. In the context of ASC 606, each unit of XPOVIO that is ordered by our customers represents a distinct performance obligation that is completed when control of the product is transferred to the customer. Accordingly, we recognize product revenue when the customer obtains control of our product, which occurs at a point in time, generally upon delivery pursuant to our agreements with our customers. If taxes should be collected from customers relating to product sales and remitted to governmental authorities, they will be excluded from revenue. Revenue from product sales is recorded at the net sales price, which includes estimates of variable consideration for which reserves are reported. These reserves, as detailed below, are based on the amounts earned, or to be claimed on the related sales, and are classified as reductions of accounts receivable (if the amount is payable to the customer) or a current liability (if the amount is payable to a party other than a customer). Certain of the amounts noted are known at the time of sale based on contractual terms and, therefore, are recorded pursuant to the most likely amount method under ASC 606. Other amounts are estimated and take into consideration a range of possible outcomes, which are probability-weighted and recorded in accordance with the expected value method in ASC 606 for relevant factors, such as current contractual and statutory requirements, specific known market events and trends, industry data, and forecasted customer buying and payment patterns. Overall, these reserves reflect our best estimates of the amount of consideration to which we are entitled based on the terms of the respective underlying contracts. The amount of variable consideration that is included in the transaction price may be constrained and is included in the net sales price only to the extent that it is probable that a significant reversal in the amount of the cumulative revenue recognized under the contracts with our customers will not occur in a future period. The following are the components of variable consideration related to product revenue: Cash discounts and distributor fees: Product returns: Based on the distribution model for XPOVIO, contractual inventory limits with our customers, the price of XPOVIO, and limited contractual return rights, we currently believe there will be minimal XPOVIO returns. However, we will update our estimated return liability each reporting period based on actual shipments of XPOVIO subject to contractual return rights, changes in expectations about the amount of estimated and/or actual returns, and other qualitative considerations. Chargebacks: Government rebates Other incentives: co-payment co-payments co-payment co-payment Product revenue reserves and allowances: therefore, the transaction price was not reduced further during the year ended December 31, 2019. Actual amounts of consideration ultimately received may differ from our estimates. If actual results in the future vary from our estimates, we will adjust these estimates, which would affect product revenue, net and earnings in the period in which such variances become known. License and Asset Purchase Agreements We generate revenue from license or similar agreements with pharmaceutical companies for the development and commercialization of certain of our product candidates. Such agreements may include the transfer of intellectual property rights in the form of licenses, transfer of technological know-how, non-refundable If a license to our intellectual property is determined to be distinct from the other performance obligations identified in the arrangement, we recognize the transaction price allocated to the license as revenue upon transfer of control of the license. We evaluate all other promised goods or services in the agreement to determine if they are distinct. If they are not distinct, they are combined with other promised goods or services to create a bundle of promised goods or services that is distinct. Optional future services where any additional consideration paid to us reflects their standalone selling prices do not provide the customer with a material right and, therefore, are not considered performance obligations. If optional future services are priced in a manner which provides the customer with a significant or incremental discount, they are material rights, and are accounted for as performance obligations. We utilize judgment to determine the transaction price. In connection therewith, we evaluate contingent milestones at contract inception to estimate the amount which is not probable of a material reversal to include in the transaction price using the most likely amount method. Milestone payments that are not within our control, such as regulatory approvals, are not considered probable of being achieved until those approvals are received and therefore the variable consideration is constrained. The transaction price is then allocated to each performance obligation on a relative stand-alone selling price basis, for which we recognize revenue as or when the performance obligations under the contract are satisfied. At the end of each reporting period, we re-evaluate catch-up We then determine whether the performance obligations or combined performance obligations are satisfied over time or at a point in time and, if over time, the appropriate method of measuring progress for purposes of recognizing revenue from non-refundable, When consideration is received, or such consideration is unconditionally due, from a customer prior to transferring goods or services to the customer under the terms of a contract, a contract liability is recorded within deferred revenue. Contract liabilities within deferred revenue are recognized as revenue after control of the goods or services is transferred to the customer and all revenue recognition criteria have been met. For arrangements that include sales-based royalties, including sales-based milestone payments, and a license of intellectual property that is deemed to be the predominant item to which the royalties relate, we recognize revenue at the later of when the related sales occur or when the performance obligation to which some or all of the royalty has been allocated has been satisfied (or partially satisfied). |
Accounts Receivable | Accounts Receivable In general, accounts receivable consists of amounts due from customers, net of customer allowances for cash discounts, product returns, and chargebacks. Our contracts with customers have standard payment terms that generally require payment within 30 days for specialty pharmacy customers and 65 days for specialty distributor customers . We analyze accounts that are past due for collectability, and periodically evaluate the creditworthiness of our customers. As of December 31, 2019, we determined an allowance for doubtful accounts was not required based upon our review of contractual payment terms and individual customer circumstances. |
Inventory | Inventory Prior to regulatory approval, we expense costs relating to the production of inventory as research and development expense in the period incurred. We capitalize the costs to manufacture our products incurred after regulatory approval when, based on our judgment, future commercialization is considered probable and the future economic benefit is expected to be realized. Such costs are generally recorded as costs of sales upon shipment. In connection therewith, we value our inventories at the lower of cost or estimated net realizable value. We determine the cost of our inventories, which includes amounts related to materials and manufacturing overhead, on a first-in, first-out Prior to FDA approval of XPOVIO, all costs related to the manufacturing of XPOVIO that could potentially be available to support the commercial launch of our products were charged to research and development expense in the period incurred, as there was no alternative future use. We analyze our inventory levels for recoverability each reporting period. In the period in which there is an impairment identified, we write down inventory that has become obsolete, inventory that has a cost basis in excess of its estimated realizable value, and inventory in excess of expected sales requirements as cost of sales. The determination of whether inventory costs will be realizable is based on our estimates. If actual market conditions are less favorable than projected by us, additional write-downs of inventory may be required, which would be recorded as cost of sales. |
Cost of Sales | Cost of Sales Cost of sales includes the cost of producing and distributing inventories that are related to product revenue during the respective period, including salary related and stock-based compensation expense for employees involved with production and distribution, freight, and indirect overhead costs, as well as third-party royalties payable on product revenue, net. In addition, shipping and handling costs for product shipments are recorded in cost of sales as incurred. Finally, cost of sales may also include costs related to excess or obsolete inventory adjustment charges, abnormal costs, unabsorbed manufacturing and overhead costs, and manufacturing variances. |
Deferred Royalty Obligation | Deferred Royalty Obligation We treat the liability related to net revenues, as discussed further in Note 15, as a deferred royalty obligation, amortized under the effective interest rate method over the estimated life of the revenue streams. We recognize interest expense thereon using the effective rate, which is based on our current estimates of future revenues over the life of the arrangement. In connection therewith, we periodically assess our expected revenues using internal projections, impute interest on the carrying value of the deferred royalty obligation, and record interest expense using the imputed effective interest rate. To the extent our estimates of future revenues are greater or less than previous estimates or the estimated timing of such payments is materially different than previous estimates, we will account for any such changes by adjusting the effective interest rate on a prospective basis, with a corresponding impact to the reclassification of our deferred royalty obligation. The assumptions used in determining the expected repayment term of the deferred royalty obligation and amortization period of the issuance costs requires that we make estimates that could impact the short-term and long-term classification of such costs, as well as the period over which such costs will be amortized. |
Research and Development Expenses | Research and Development Expenses Research and development costs are charged to expense as incurred and include, but are not limited to: • employee-related expenses, including salaries, benefits, travel and stock-based compensation expense; • expenses incurred under agreements with contract research organizations, contract manufacturing organizations and consultants that help conduct clinical trials and preclinical studies; • the cost of acquiring, developing and manufacturing clinical trial materials, including comparator drugs; • facility, depreciation and other expenses, which include direct and allocated expenses for rent and maintenance of facilities, insurance and other supplies; and • costs associated with preclinical activities and regulatory operations. Costs for certain development activities, such as clinical trials, are recognized based on an evaluation of the progress to completion of specific tasks using data such as patient enrollment, clinical site activations, or information provided to us by our vendors on their actual costs incurred. Payments for these activities are based on the terms of the individual arrangements, which may differ from the pattern of costs incurred, and are accordingly reflected in the financial statements as prepaid or accrued research and development. |
Comprehensive Loss | Comprehensive Loss Comprehensive loss consists of net loss and changes in equity during a period from transactions and other equity and circumstances generated from non-owner |
Foreign Currency Transactions | Foreign Currency Transactions The functional currency of our subsidiaries in Germany and Israel are the Euro and Shekel, respectively. Foreign currency transaction gains and losses are recorded in the consolidated statement of operations. Net foreign exchange losses of less than $0.1 million were recorded in other income for the years ended December 31, 2019, 2018 and 2017. |
Income Taxes | Income Taxes We use the liability method of accounting for income taxes. Under this method, deferred tax assets and liabilities are determined based on the difference between the financial reporting and the tax reporting basis of assets and liabilities and are measured using the enacted tax rates and laws that are expected to be in effect when the differences are expected to reverse. We provide a valuation allowance against net deferred tax assets unless, based upon the available evidence, it is more likely than not that the deferred tax assets will be realized. We have evaluated available evidence and concluded that we may not realize the benefit of our deferred tax assets; therefore, a valuation allowance has been established for the full amount of the deferred tax assets. We recognize interest and/or penalties related to income tax matters in income tax expense. Our foreign tax provision pertains to foreign income taxes due at our German subsidiary which operates on a cost plus profit margin basis. The Tax Cuts and Jobs Act of 2017 (“TCJA”) resulted in significant changes to the U.S. corporate income tax system. For additional details regarding this act, see Note 14 |
Accounting for Stock-Based Compensation | Accounting for Stock-Based Compensation We account for our stock-based compensation awards in accordance with FASB ASC Topic 718, Compensation—Stock Compensation non-employees, well as modifications to existing stock options and shares issued under our employee stock purchase plan (“ESPP”), to be recognized in the consolidated statements of operations based on their fair values. We use the Black-Scholes option pricing model to determine the fair value of options granted. Compensation expense related to awards to employees and non-employees |
Net Loss Per Share | Net Loss Per Share Basic and diluted net loss per common share is calculated by dividing net loss by the weighted-average number of common shares outstanding for the period, without consideration for common stock equivalents. Our potential dilutive shares, stock options, unvested restricted stock and restricted stock units are considered to be common stock equivalents and are only included in the calculation of diluted net loss per share when their effect is dilutive. The following potentially dilutive securities were excluded from the calculation of diluted net loss per share due to their anti-dilutive effect at December 31, 2019, 2018 and 2017 (in common stock equivalent shares): December 31, 2019 2018 2017 Outstanding stock options 9,843,094 8,917,084 7,019,083 Unvested restricted stock units 787,320 25,000 253,100 We have the option to settle the conversion obligation for our 3.00% convertible senior notes due 2025 (the “Notes”) in cash, shares or any combination of the two. As the Notes are not convertible as of December 31, 2019, they are not participating securities and they will not have an impact on the calculation of basic earnings or loss per share. Based on our net loss position, there is no impact on the calculation of dilutive loss per share during the year ended December 31, 2019. |
Recently Adopted Accounting Standards | Recently Adopted Accounting Standards In February 2016, the FASB issued ASU No. 2016-02, Leases (Topic 842) 2016-02”), Leases Leases 2016-02, 2018-10, Codification Improvements to Topic 842, Leases 2018-10”) 2018-11, Leases (Topic 842) Targeted Improvements 2018-11”), right-of-use 2018-11 Pursuant to the guidance under ASU 2016-02, which we elected, including: (i) a policy to not record right-of-use non-lease As summarized in the table below, the standard had a material impact on our condensed consolidated balance sheet as of December 31, 2019, specifically through recognition of right-of-use right-of-use right-of-use Please refer to Note 9, “Commitments and Contingencies” for further information January 1, 2019 ASC 842 January 1, 2019 Consolidated balance sheet data (in thousands): Operating lease and right-of-use $ — $ 11,711 $ 11,711 Deferred rent(2) $ 390 $ (390 ) $ — Deferred rent non-current(2) $ 3,922 $ (3,922 ) $ — Operating lease liabilities(3) $ — $ 1,175 $ 1,175 Non-current $ — $ 14,848 $ 14,848 (1) Represents capitalization of operating lease right-of-use right-of-use (2) Represents reclassification of deferred rent and tenant incentives to operating lease right-of-use (3) Represents recognition of operating lease liabilities. We implemented internal controls to enable the preparation of financial information upon adoption. In June 2018, the FASB issued ASU No. 2018-07, Compensation-Stock Compensation (Topic 718): Improvements to Nonemployee Share-Based Payment Accounting 2018-07”). 2018-07 In November 2018, the FASB issued ASU No. 2018-18, Collaborative Arrangements (Topic 808)—Clarifying the Interaction between Topic 808 and Topic 606 2018-18”). 2018-18 2018-18 2018-18 11 In December 2019, the FASB issued ASU No. 2019-12, Simplifying the Accounting for Income Taxes 2019-12”). 740-20-45-7 operations (the so-called Recently Issued Accounting Standards In June 2016, the FASB issued ASU No. 2016-13, Financial Instruments—Credit Losses (Topic 326): Measurement of Credit Losses on Financial Instruments 2016-13”). 2016-13 available-for-sale 2016-13 2016-13 In August 2018, the FASB issued ASU No. 2018-13, Fair Value Measurement—Disclosure Framework-Changes to the Disclosure Requirement for Fair Value Measurement 2018-13”). 2018-13 2018-13 2018-13 In August 2018, the FASB issued ASU No. 2018-15, Intangible-Goodwill and Other Internal-Use Software (Subtopic 350-40) 2018-15”). 2018-15 2018-15 2018-15 |
Summary of Significant Accoun_3
Summary of Significant Accounting Policies (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Accounting Policies [Abstract] | |
Schedule of Financial Assets That Have Been Measured at Fair Value | The following table presents information about our financial assets and liability that have been measured at fair value at December 31, 2019 and indicates the fair value hierarchy of the valuation inputs utilized to determine such fair value (in thousands): Description Total Quoted Prices Significant Significant Financial assets Cash equivalents: Money market funds $ 71,380 $ 71,380 $ — $ — Investments: Short-term: Corporate debt securities 89,079 — 89,079 — Commercial paper 39,022 — 39,022 — U.S. government and agency securities 4,997 — 4,997 — Long-term: Corporate debt securities (one to two year maturity) 2,016 — 2,016 — $ 206,494 $ 71,380 $ 135,114 $ — Financial liability Embedded derivative liability $ 2,300 $ — $ — $ 2,300 The following table presents information about our financial assets that have been measured at fair value at December 31, 2018 and indicates the fair value hierarchy of the valuation inputs utilized to determine such fair value (in thousands): Description Total Quoted Prices Significant Significant Financial assets Cash equivalents: Money market funds $ 76,881 $ 76,881 $ — $ — Investments: Short-term: Corporate debt securities 143,079 — 143,079 — Commercial paper 43,978 — 43,978 — U.S. government and agency securities 19,124 — 19,124 — Certificate of deposit 3,997 — 3,997 — Long-term: Corporate debt securities (one to two year maturity) 2,001 — 2,001 — $ 289,060 $ 76,881 $ 212,179 $ — |
Schedule of Changes in the Estimated Fair Value of Our Embedded Derivative Liability | The following table sets forth a summary of the changes in the estimated fair value of our embedded derivative liability during the year ended December 31, 2019 (in thousands): Embedded Balance as of December 31, 2018 $ — Addition of derivative related to deferred royalty obligation . 2,300 Change in fair value of derivative since issuanc e — Balance as of December 31, 2019 $ 2,300 |
Schedule of Potentially Dilutive Securities Were Excluded From The Calculation of Diluted Net Loss Per Share Due to Their Anti-Dilutive Effect | The following potentially dilutive securities were excluded from the calculation of diluted net loss per share due to their anti-dilutive effect at December 31, 2019, 2018 and 2017 (in common stock equivalent shares): December 31, 2019 2018 2017 Outstanding stock options 9,843,094 8,917,084 7,019,083 Unvested restricted stock units 787,320 25,000 253,100 |
Schedule of Adjustments in Balance Sheet due to Adoption of ASC 842 | January 1, 2019 ASC 842 January 1, 2019 Consolidated balance sheet data (in thousands): Operating lease and right-of-use $ — $ 11,711 $ 11,711 Deferred rent(2) $ 390 $ (390 ) $ — Deferred rent non-current(2) $ 3,922 $ (3,922 ) $ — Operating lease liabilities(3) $ — $ 1,175 $ 1,175 Non-current $ — $ 14,848 $ 14,848 (1) Represents capitalization of operating lease right-of-use right-of-use (2) Represents reclassification of deferred rent and tenant incentives to operating lease right-of-use (3) Represents recognition of operating lease liabilities. |
Property and Equipment, net (Ta
Property and Equipment, net (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Property, Plant and Equipment [Abstract] | |
Schedule of Property and Equipment, Net | Property and equipment, net consisted of the following (in thousands): December 31, Estimated Useful 2019 2018 Laboratory equipment 4 $ 593 $ 593 Furniture and fixtures 5 654 607 Office and computer equipment 3 598 559 Leasehold improvements Lesser of useful life or lease term 5,443 5,397 7,288 7,156 Less accumulated depreciation and amortization (4,242 ) (3,293 ) $ 3,046 $ 3,863 |
Investments (Tables)
Investments (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Investments, Debt and Equity Securities [Abstract] | |
Summary of Investments, Classified as Available-for-Sale | The following table summarizes our investments in debt securities, classified as available-for-sale Amortized Cost Gross Unrealized Gross Unrealized Fair Value Short-term: Corporate debt securities $ 89,110 $ 12 $ (43 ) $ 89,079 Commercial paper 39,004 18 — 39,022 U.S. government and agency securities 4,990 7 — 4,997 Long-term: Corporate debt securities (one to two year maturity) 2,017 — (1 ) 2,016 $ 135,121 $ 37 $ (44 ) $ 135,114 The following table summarizes our investments in debt securities, classified as available-for-sale Amortized Cost Gross Unrealized Gross Unrealized Fair Value Short-term: Corporate debt securities $ 143,254 $ 3 $ (178 ) $ 143,079 Commercial paper 44,001 — (23 ) 43,978 U.S. government and agency securities 19,131 10 (17 ) 19,124 Certificates of deposit 4,000 — (3 ) 3,997 Long-term: Corporate debt securities (one to two year maturity) 2,007 — (6 ) 2,001 $ 212,393 $ 13 $ (227 ) $ 212,179 |
Inventory (Tables)
Inventory (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Inventory Disclosure [Abstract] | |
Schedule of Inventory, Current | The following table presents our inventory of XPOVIO at December 31, 2019 and 2018 (in thousands): December 31, 2019 December 31, 2018 Raw materials and work in process $ 273 $ — Finished goods 73 — Total inventory $ 346 $ — |
Accrued Expenses (Tables)
Accrued Expenses (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Text Block [Abstract] | |
Schedule of Accrued Expenses | Accrued expenses consisted of the following as of December 31, 2019 and 2018 (in thousands): December 31, 2019 2018 Research and development costs $ 13,122 $ 15,903 Payroll and employee-related costs 13,630 10,103 Professional fees 6,172 4,931 I 4,371 — Other 3,583 1,556 $ 40,878 $ 32,493 |
Commitments and Contingencies
Commitments and Contingencies (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Leases [Abstract] | |
Schedule of Minimum Future Rent Payments Under Lease Agreement | As of December 31, 2019, future minimum lease payments under non-cancellable right-of-use Years ending December 31, Future 2020 $ 3,200 2021 3,277 2022 3,447 2023 3,718 2024 and thereafter 6,735 Total minimum lease payments $ 20,377 Less: present value adjustment (5,529 ) Present value of minimum lease payments $ 14,848 |
Product Revenue (Tables)
Product Revenue (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
XPOVIO [Member] | |
Schedule of Product Discounts Returns Allowances Reconciliation | The following table summarizes activity in each of the product revenue allowance and reserve categories from the date of approval by the FDA through December 31, 2019 (in thousands): Discounts and Fees, Rebates, Returns Total Short-term: Beginning balance at July 3 $ — $ — $ — $ — Provision related to sales in the current year 2,657 2,318 234 5,209 Credit and payments made (1,655 ) (499 ) — (2,154 ) Ending balance at December 31, 2019 $ 1,002 $ 1,819 $ 234 $ 3,055 |
Stock-based Compensation (Table
Stock-based Compensation (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Schedule of Total Stock-based Compensation Expense Recognized in Connection with All Share-based Payment Awards | In connection with all share-based payment awards, total stock-based compensation expense recognized was as follows (in thousands): Year Ended December 31, 2019 2018 2017 Research and development $ 6,457 $ 8,686 $ 11,208 Selling, general and administrative 8,834 8,589 9,197 Total $ 15,291 $ 17,275 $ 20,405 |
Summary of Stock Option Activity for Employees and Nonemployees | The following table summarizes stock option activity for employees and nonemployees: Options Weighted- Weighted- Aggregate (in Options outstanding at December 31, 2018 8,917,084 $ 13.78 7.4 $ 8,197 Granted 3,325,390 8.67 Exercised (394,358 ) 7.18 Forfeited (2,005,022 ) 13.36 Options outstanding at December 31, 2019 9, 843,094 $ 12.40 7.0 $ 82,134 Options exercisable at December 31, 2019 5,391,153 $ 14.65 5.6 $ 39,879 |
Summary of RSU Activity | During the year ended December 31, 2019, we granted 1,065,970 Number of Weighted-Average Unvested at December 31, 2018 25,000 $ 9.87 Granted 1,065,970 9.25 Forfeited (286,150 ) 9.24 Vested (17,500 ) 8.96 Unvested at December 31, 2019 787,320 $ 9.28 |
ESPP [Member] | |
Schedule of Assumptions Used to Estimate Fair Value on Grant Date Using Black Scholes Option Pricing Model | The fair value of the option component of the shares purchased under the ESPP was estimated using the Black-Scholes option-pricing model with the following weighted-average assumptions: Years Ended 2019 2018 2017 Volatility 61 % 48%-61% 48%-78% Expected term (in years) 0.5 0.5 0.5 Risk-free interest rate 2.4 % 1.3%-2.1% 0.5%-1.3% Dividend —% —% —% |
Employee and Nonemployee Stock Option [Member] | |
Schedule of Assumptions Used to Estimate Fair Value on Grant Date Using Black Scholes Option Pricing Model | The fair value of each stock option granted to employees is estimated on the date of grant and for non-employees Years Ended December 31, 2019 2018 2017 Volatility 79%-81% 79%-81% 79%-85% Expected term (in years) 5.5-6.0 5.5-9.8 5.5-9.6 Risk-free interest rate 1.42%-2.58% 2.50%-3.05% 1.76%-2.29% Dividend —% —% —% |
Income Taxes (Table)
Income Taxes (Table) | 12 Months Ended |
Dec. 31, 2019 | |
Income Tax Disclosure [Abstract] | |
Schedule of Components of Income (Loss) Before Income Taxes | The components of loss before income taxes were as follows (in thousands): Year Ended 2019 2018 2017 Foreign $ (23,350 ) $ (28,689 ) $ (35,680 ) U.S. (176,200 ) (149,692 ) (93,241 ) Totals $ (199,550 ) $ (178,381 ) $ (128,921 ) |
Schedule of Significant Components of Company's Deferred Tax Assets | The significant components of our deferred tax assets are comprised of the following (in thousands): Year Ended 2019 2018 Deferred tax assets: U.S. and state net operating loss carryforwards $ 150,909 $ 116,236 Stock-based compensation 11,914 11,228 Accruals and other temporary differences 4,212 3,605 Research and development credits 62,374 49,307 Capitalized research and development 1,021 1,388 Fixed assets and intangibles 5,893 6,422 Deferred revenue 1,051 527 Foreign net operating loss carryforwards 4 72 Lease liability 3,443 — Deferred royalty embedded derivative 533 — Valuation allowance (224,943 ) (173,247 ) Total deferred tax assets 16,411 15,538 Deferred tax liabilities: Convertible debt amortization (13,431 ) (15,538 ) Right-of-use asset (2,462 ) — Deferred royalty obligation (518 ) — Total deferred tax liabilities (16,411 ) (15,538 ) Net deferred tax assets $ — $ — |
Schedule of Reconciliation of Income Tax Expense Computed at Statutory Federal Income Tax Rate to Income Taxes | A reconciliation of income tax expense computed at the statutory federal income tax rate to income taxes as reflected in the financial statements is as follows: Year Ended 2019 2018 2017 Federal income tax expense at statutory rate 21.0 % 21.0 % 34.0 % State income tax, net of federal benefit 1.7 % 0.7 % 5.9 % Permanent differences (0.5 )% — % (3.9 )% Research and development credit 6.0 % 7.8 % 8.4 % Foreign rate differential (2.5 )% (3.4 )% (9.4 )% Change in valuation allowance (25.9 )% (28.2 )% (1.4 )% Provision to return adjustments 0.6 % 2.4 % 1.0 % Other (0.4 )% (0.6 )% (1.4 )% Federal rate change — % 0.3 % (33.2 )% Effective income tax rate — % — % — % |
Long-Term Obligations (Tables)
Long-Term Obligations (Tables) - 3% Convertible Senior Notes Due 2025 [Member] | 12 Months Ended |
Dec. 31, 2019 | |
Summary of Outstanding Balances of Convertible Notes | The outstanding balances of the Notes as of December 31, 2019 consisted of the following (in thousands): Liability component: Principal $ 172,500 Less: debt discount and issuance costs, net (62,643 ) Net carrying amount $ 109,857 Equity component: $ 65,641 |
Schedule of Interest Expense Recognized Related to Convertible Notes | The following table sets forth total interest expense recognized related to the Notes during the year ended December 31, 2019 (in thousands): Year Ended Contractual interest expense $ 5,175 Amortization of debt discount 6,849 Amortization of debt issuance costs 344 Total interest expense $ 12,368 |
Summary of Future Minimum Payments on Convertible Notes | Future minimum payments on the Notes as of December 31, 2019 were as follows (in thousands): Years ended December 31, Future Minimum Payments 2020 $ 5,175 2021 5,175 2022 5,175 2023 5,175 2024 and thereafter 182,850 Total minimum payments $ 203,550 Less: interest (31,050 ) Less: unamortized discount (62,643 ) Less: current portion — Convertible senior notes $ 109,857 |
Organization and Operations - A
Organization and Operations - Additional Information (Detail) - USD ($) $ in Thousands | Dec. 31, 2019 | Dec. 31, 2018 |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | ||
Accumulated deficit | $ (873,338) | $ (673,748) |
Summary of Significant Accoun_4
Summary of Significant Accounting Policies - Additional Information (Detail) $ in Thousands | 12 Months Ended | ||||
Dec. 31, 2019USD ($)Segments | Dec. 31, 2018USD ($) | Dec. 31, 2017USD ($) | Jan. 01, 2019USD ($) | ||
Accounting Policies And General Information [Line Items] | |||||
Number of operating segments | Segments | 1 | ||||
Foreign exchange losses | $ 100 | $ 100 | $ 100 | ||
Operating lease right-of-use asset | 10,617 | $ 11,711 | [1] | ||
Lease liability | 14,848 | 1,175 | [2] | ||
Accounting Standards Update 2016-02 [Member] | |||||
Accounting Policies And General Information [Line Items] | |||||
Operating lease right-of-use asset | $ 11,700 | ||||
Lease liability | $ 16,000 | ||||
Minimum [Member] | |||||
Accounting Policies And General Information [Line Items] | |||||
Estimated useful life | 3 years | ||||
Maximum [Member] | |||||
Accounting Policies And General Information [Line Items] | |||||
Estimated useful life | 5 years | ||||
[1] | Represents capitalization of operating lease right-of-use assets, offset by reclassification of deferred rent and tenant incentives to operating lease right-of-use assets. | ||||
[2] | Represents recognition of operating lease liabilities. |
Summary of Significant Accoun_5
Summary of Significant Accounting Policies - Schedule of Financial Assets That Have Been Measured at Fair Value (Details) - USD ($) $ in Thousands | Dec. 31, 2019 | Dec. 31, 2018 |
Financial assets | ||
Total | $ 206,494 | $ 289,060 |
Financial liability | ||
Embedded derivative liability | 2,300 | |
Short-term [Member] | Corporate Debt Securities [Member] | ||
Financial assets | ||
Investments | 89,079 | 143,079 |
Short-term [Member] | Commercial Paper [Member] | ||
Financial assets | ||
Investments | 39,022 | 43,978 |
Short-term [Member] | US Government and Agency Securities [Member] | ||
Financial assets | ||
Investments | 4,997 | 19,124 |
Long-term [Member] | Corporate Debt Securities [Member] | ||
Financial assets | ||
Investments | 2,001 | |
Money Market Funds [Member] | ||
Financial assets | ||
Cash equivalents | 71,380 | 76,881 |
Certificates of Deposit [Member] | Short-term [Member] | ||
Financial assets | ||
Investments | 3,997 | |
Certificates of Deposit [Member] | Long-term [Member] | ||
Financial assets | ||
Investments | 2,016 | |
Level 1 [Member] | ||
Financial assets | ||
Total | 71,380 | 76,881 |
Level 1 [Member] | Money Market Funds [Member] | ||
Financial assets | ||
Cash equivalents | 71,380 | 76,881 |
Level 2 [Member] | ||
Financial assets | ||
Total | 135,114 | 212,179 |
Level 2 [Member] | Short-term [Member] | Corporate Debt Securities [Member] | ||
Financial assets | ||
Investments | 89,079 | 143,079 |
Level 2 [Member] | Short-term [Member] | Commercial Paper [Member] | ||
Financial assets | ||
Investments | 39,022 | 43,978 |
Level 2 [Member] | Short-term [Member] | US Government and Agency Securities [Member] | ||
Financial assets | ||
Investments | 4,997 | 19,124 |
Level 2 [Member] | Long-term [Member] | Corporate Debt Securities [Member] | ||
Financial assets | ||
Investments | 2,001 | |
Level 2 [Member] | Certificates of Deposit [Member] | Short-term [Member] | ||
Financial assets | ||
Investments | $ 3,997 | |
Level 2 [Member] | Certificates of Deposit [Member] | Long-term [Member] | ||
Financial assets | ||
Investments | 2,016 | |
Level 3 [Member] | ||
Financial liability | ||
Embedded derivative liability | $ 2,300 |
Summary of Significant Accoun_6
Summary of Significant Accounting Policies - The Changes in the Estimated Fair Value of Our Embedded Derivative Liability - (Details) - Embedded Derivative Financial Instruments [Member] $ in Thousands | 12 Months Ended |
Dec. 31, 2019USD ($) | |
Fair Value, Liabilities Measured on Recurring Basis, Unobservable Input Reconciliation [Line Items] | |
Beginning balance | $ 0 |
Addition of derivative related to deferred royalty obligation | 2,300 |
Change in fair value of derivative since issuance | 0 |
Ending balance | $ 2,300 |
Summary of Significant Accoun_7
Summary of Significant Accounting Policies - Schedule of Potentially Dilutive Securities Were Excluded From The Calculation of Diluted Net Loss Per Share Due to Their Anti-Dilutive Effect (Detail) - shares | 12 Months Ended | ||
Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Outstanding Stock Options [Member] | |||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | |||
Dilutive securities excluded from the calculation of diluted net loss per share due to anti-dilutive effect (in shares) | 9,843,094 | 8,917,084 | 7,019,083 |
Unvested Restricted Stock Units [Member] | |||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | |||
Dilutive securities excluded from the calculation of diluted net loss per share due to anti-dilutive effect (in shares) | 787,320 | 25,000 | 253,100 |
Summary of Significant Accoun_8
Summary of Significant Accounting Policies- Schedule of Adjustments in Balance Sheet due to Adoption of ASC 842 (Detail) - USD ($) $ in Thousands | Dec. 31, 2019 | Jan. 01, 2019 | Dec. 31, 2018 | ||
Adjustments For Change In Accounting Principle [Line Items] | |||||
Operating lease and right-of-use assets | $ 10,617 | $ 11,711 | [1] | ||
Deferred rent | $ 390 | ||||
Deferred rent non-current | $ 3,922 | ||||
Operating lease liabilities | 14,848 | 1,175 | [2] | ||
Non-current operating lease liabilities | $ 13,202 | 14,848 | [2] | ||
Previously Reported [Member] | |||||
Adjustments For Change In Accounting Principle [Line Items] | |||||
Deferred rent | [3] | 390 | |||
Deferred rent non-current | [3] | 3,922 | |||
Restatement Adjustment [Member] | |||||
Adjustments For Change In Accounting Principle [Line Items] | |||||
Operating lease and right-of-use assets | [1] | 11,711 | |||
Deferred rent | [3] | (390) | |||
Deferred rent non-current | [3] | (3,922) | |||
Operating lease liabilities | [2] | 1,175 | |||
Non-current operating lease liabilities | [2] | $ 14,848 | |||
[1] | Represents capitalization of operating lease right-of-use assets, offset by reclassification of deferred rent and tenant incentives to operating lease right-of-use assets. | ||||
[2] | Represents recognition of operating lease liabilities. | ||||
[3] | Represents reclassification of deferred rent and tenant incentives to operating lease right-of-use assets. |
Property and Equipment, net - S
Property and Equipment, net - Schedule of Property and Equipment, Net (Detail) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2019 | Dec. 31, 2018 | |
Property, Plant and Equipment [Line Items] | ||
Property and equipment, gross | $ 7,288 | $ 7,156 |
Less accumulated depreciation and amortization | (4,242) | (3,293) |
Property and equipment, net | $ 3,046 | 3,863 |
Laboratory Equipment [Member] | ||
Property, Plant and Equipment [Line Items] | ||
Estimated useful life | 4 years | |
Property and equipment, gross | $ 593 | 593 |
Furniture and Fixtures [Member] | ||
Property, Plant and Equipment [Line Items] | ||
Estimated useful life | 5 years | |
Property and equipment, gross | $ 654 | 607 |
Office Equipment [Member] | ||
Property, Plant and Equipment [Line Items] | ||
Estimated useful life | 3 years | |
Property and equipment, gross | $ 598 | 559 |
Leasehold Improvements [Member] | ||
Property, Plant and Equipment [Line Items] | ||
Estimated useful life | Lesser of useful life or lease term | |
Property and equipment, gross | $ 5,443 | $ 5,397 |
Property and Equipment, net - A
Property and Equipment, net - Additional Information (Detail) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Property, Plant and Equipment [Abstract] | |||
Depreciation and amortization expense | $ 974 | $ 735 | $ 713 |
Investments - Summary of Invest
Investments - Summary of Investments, Classified as Available-for-Sale (Detail) - USD ($) $ in Thousands | Dec. 31, 2019 | Dec. 31, 2018 |
Debt Securities, Available-for-sale [Line Items] | ||
Amortized Cost | $ 135,121 | $ 212,393 |
Gross Unrealized Gains | 37 | 13 |
Gross Unrealized Loss | (44) | (227) |
Fair Value | 135,114 | 212,179 |
Short-term [Member] | Certificates of Deposit [Member] | ||
Debt Securities, Available-for-sale [Line Items] | ||
Amortized Cost | 4,000 | |
Gross Unrealized Loss | (3) | |
Fair Value | 3,997 | |
Short-term [Member] | Corporate Debt Securities [Member] | ||
Debt Securities, Available-for-sale [Line Items] | ||
Amortized Cost | 89,110 | 143,254 |
Gross Unrealized Gains | 12 | 3 |
Gross Unrealized Loss | (43) | (178) |
Fair Value | 89,079 | 143,079 |
Short-term [Member] | Commercial Paper [Member] | ||
Debt Securities, Available-for-sale [Line Items] | ||
Amortized Cost | 39,004 | 44,001 |
Gross Unrealized Gains | 18 | |
Gross Unrealized Loss | (23) | |
Fair Value | 39,022 | 43,978 |
Short-term [Member] | US Government and Agency Securities [Member] | ||
Debt Securities, Available-for-sale [Line Items] | ||
Amortized Cost | 4,990 | 19,131 |
Gross Unrealized Gains | 7 | 10 |
Gross Unrealized Loss | (17) | |
Fair Value | 4,997 | 19,124 |
Long-term [Member] | Corporate Debt Securities [Member] | ||
Debt Securities, Available-for-sale [Line Items] | ||
Amortized Cost | 2,017 | 2,007 |
Gross Unrealized Loss | (1) | (6) |
Fair Value | $ 2,016 | $ 2,001 |
Investments - Additional Inform
Investments - Additional Information (Detail) | Dec. 31, 2019USD ($)Securities | Dec. 31, 2018USD ($)Securities |
Debt Securities, Available-for-sale [Line Items] | ||
Number of debt securities with unrealized loss position for less than one year | Securities | 27 | 79 |
Aggregate fair value of debt securities with unrealized loss position for less than one year | $ 63,800,000 | $ 180,600,000 |
Unrealized losses , included in accumulated other comprehensive loss | 44,000 | 227,000 |
Unrealized losses, other-than-temporary impairments | $ 0 | $ 0 |
Corporate Debt Securities [Member] | ||
Debt Securities, Available-for-sale [Line Items] | ||
Number of corporate debt securities with continuous unrealized loss position for more than 12 months | Securities | 0 | 8 |
Aggregate fair value of corporate debt securities with continuous unrealized loss position for more than 12 months | $ 14,900,000 | |
Unrealized losses , included in accumulated other comprehensive loss | $ 100,000 |
Inventory - Schedule of Invento
Inventory - Schedule of Inventory Current (Detail) - USD ($) $ in Thousands | Dec. 31, 2019 | Dec. 31, 2018 |
Inventory Disclosure [Abstract] | ||
Raw materials and work in process | $ 273 | $ 0 |
Finished goods | 73 | |
Total inventory | $ 346 |
Accrued Expenses - Schedule of
Accrued Expenses - Schedule of Accrued Expenses (Detail) - USD ($) $ in Thousands | Dec. 31, 2019 | Dec. 31, 2018 |
Accrued Liabilities, Current [Abstract] | ||
Research and development costs | $ 13,122 | $ 15,903 |
Payroll and employee-related costs | 13,630 | 10,103 |
Professional fees | 6,172 | 4,931 |
Interest | 4,371 | |
Other | 3,583 | 1,556 |
Total Accrued Expenses | $ 40,878 | $ 32,493 |
Related Party Transactions - Ad
Related Party Transactions - Additional Information (Detail) - USD ($) $ in Millions | 12 Months Ended | |
Dec. 31, 2019 | Dec. 31, 2018 | |
Consulting and Histopathology Services [Member] | ||
Related Party Transaction [Line Items] | ||
Expenses for consulting and histopathology services | $ 0.2 | $ 0.2 |
Consulting and Contract Research Services [Member] | ||
Related Party Transaction [Line Items] | ||
Accounts payable or accrued expenses | $ 0.1 | $ 0 |
Stockholders' Equity - Controll
Stockholders' Equity - Controlled Equity Offering Sales Agreement - Additional Information (Detail) - USD ($) | Oct. 02, 2018 | May 07, 2018 | Apr. 28, 2017 | Dec. 07, 2015 | Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | Aug. 07, 2018 |
Equity Offering [Line Items] | ||||||||
Stock Issued During Period, Value, New Issues | $ 46,191,000 | $ 145,705,000 | $ 74,885,000 | |||||
Sales agreement term date | Aug. 12, 2018 | |||||||
Common Stock [Member] | ||||||||
Equity Offering [Line Items] | ||||||||
Issuance of common stock, net of issuance costs | 10,525,424 | 3,902,439 | 3,712,359 | 10,525,424 | 7,308,202 | |||
Public offering price of common shares | $ 14.75 | $ 10.25 | ||||||
Net proceeds after deducting underwriting discounts, commissions and offering expenses | $ 145,700,000 | $ 37,900 | ||||||
Stock Issued During Period, Value, New Issues | $ 1,000 | $ 1,000 | $ 1,000 | |||||
Jefferies LLC [Member] | Open Market Sale Agreement [Member] | Common Stock [Member] | ||||||||
Equity Offering [Line Items] | ||||||||
Issuance of common stock, net of issuance costs | 3,712,359 | |||||||
Net proceeds from sale of common stock | $ 46,200,000 | |||||||
Cantor Fitzgerald & Co. [Member] | Controlled Equity Offering Sales Agreement [Member] | Common Stock [Member] | ||||||||
Equity Offering [Line Items] | ||||||||
Issuance of common stock, net of issuance costs | 9,172,159 | 3,405,763 | ||||||
Stock Issued During Period, Value, New Issues | $ 89,100,000 | $ 37,000,000 | ||||||
Maximum [Member] | Jefferies LLC [Member] | Open Market Sale Agreement [Member] | ||||||||
Equity Offering [Line Items] | ||||||||
Aggregate offering price | $ 75,000,000 | |||||||
Percentage of commission of gross proceeds from the sale of Shares | 3.00% | |||||||
Maximum [Member] | Cantor Fitzgerald & Co. [Member] | Controlled Equity Offering Sales Agreement [Member] | ||||||||
Equity Offering [Line Items] | ||||||||
Percentage of commission of gross proceeds from the sale of Shares | 3.00% |
Commitments and Contingencies -
Commitments and Contingencies - Additional Information (Detail) $ in Thousands | 12 Months Ended | |
Dec. 31, 2019USD ($)ft² | Dec. 31, 2018USD ($) | |
Leases [Line Items] | ||
Deferred rent | $ 3,922 | |
Operating lease cost | $ 2,800 | |
Lease term | 5 years 9 months 18 days | |
Operating lease discount rate | 11.00% | |
Common Area Maintenance [Member] | ||
Leases [Line Items] | ||
Operating lease cost | $ 900 | |
Accounting Standards Update 2016-02 [Member] | ||
Leases [Line Items] | ||
Allowance for improvements | 11,700 | 1,700 |
Deferred rent | $ 16,000 | $ 2,600 |
Office and Research Space Lease [Member] | ||
Leases [Line Items] | ||
Office and laboratory space leased | ft² | 98,502 | |
Security deposit in the form of a letter of credit | $ 600 |
Commitments and Contingencies_2
Commitments and Contingencies - Schedule of Minimum Future Rent Payments Under Lease Agreement (Detail) - USD ($) $ in Thousands | Dec. 31, 2019 | Jan. 01, 2019 | [1] |
Commitments and Contingencies Disclosure [Abstract] | |||
2020 | $ 3,200 | ||
2021 | 3,277 | ||
2022 | 3,447 | ||
2023 | 3,718 | ||
2024 and thereafter | 6,735 | ||
Total minimum lease payments | 20,377 | ||
Less: present value adjustment | (5,529) | ||
Present value of minimum lease payments | $ 14,848 | $ 1,175 | |
[1] | Represents recognition of operating lease liabilities. |
Product Revenue - Schedule of
Product Revenue - Schedule of Product Discounts Returns Allowances Reconciliation (Detail) $ in Thousands | 12 Months Ended |
Dec. 31, 2019USD ($) | |
Revenue from Contract with Customer [Abstract] | |
Beginning balance at July 3, 2019 | |
Provision related to sales in the current year | 2,657 |
Credit and payments made | (1,655) |
Ending balance at December 31, 2019 | 1,002 |
Provision related to sales in the current year | 2,318 |
Credit and payments made | (499) |
Ending balance at December 31, 2019 | 1,819 |
Provision related to sales in the current year | 234 |
Ending balance at December 31, 2019 | 234 |
Provision related to sales in the current year | 5,209 |
Credit and payments made | (2,154) |
Ending balance at December 31, 2019 | $ 3,055 |
License and Asset Purchase Ag_2
License and Asset Purchase Agreements - Additional Information - Antengene License Agreement (Detail) - Antengene Therapeutics Limited [Member] - USD ($) $ in Millions | May 23, 2018 | Dec. 31, 2019 |
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||
Revenues | $ 9.4 | |
Government Research Grant Agreement [Member] | ||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||
License agreement termination prior notice period | 180 days | |
License agreement termination description | License Agreement may be terminated earlier by (i) either party for breach of the Antengene License Agreement by the other party or in the event of the insolvency or bankruptcy of the other party, (ii) Antengene on a product-by-product basis for certain safety reasons or on a product-by-product, country-by-country basis for any reason with 180 days’ prior notice or (iii) us in the event Antengene challenges or assists with a challenge to certain of our patent rights. | |
Milestone payments receivable | $ 5 | |
Government Research Grant Agreement [Member] | Development Milestone [Member] | Maximum [Member] | ||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||
Milestone payments receivable | $ 105 | |
Government Research Grant Agreement [Member] | Sales Milestone Events [Member] | Maximum [Member] | ||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||
Milestone payments receivable | 45 | |
Commercial milestone payments receivable | 45 | |
Government Research Grant Agreement [Member] | Development Goals [Member] | Maximum [Member] | ||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||
Milestone payments receivable | 105 | |
Selinexor [Member] | Government Research Grant Agreement [Member] | ||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||
Performance obligations | 9.4 | |
Eltanexor [Member] | Government Research Grant Agreement [Member] | ||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||
Performance obligations | 1 | |
KPT-9274 [Member] | Government Research Grant Agreement [Member] | ||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||
Performance obligations | 1.1 | |
Verdinexor [Member] | Government Research Grant Agreement [Member] | ||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||
Performance obligations | 0.2 | |
Up-front Payment Arrangement [Member] | Government Research Grant Agreement [Member] | ||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||
Upfront payments | $ 11.7 | $ 2.3 |
License and Asset Purchase Ag_3
License and Asset Purchase Agreements - Additional Information - Biogen Asset Purchase Agreement (Detail) - Biogen MA Inc [Member] - USD ($) $ in Millions | Jan. 24, 2018 | Mar. 31, 2018 | Dec. 31, 2019 |
Research and Development Arrangement, Contract to Perform for Others [Line Items] | |||
Revenues | $ 10 | ||
Asset Purchase Agreement [Member] | |||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | |||
Upfront payment received | $ 10 | $ 2 | |
Commercial milestone payments receivable | 65 | ||
Performance obligations | 10 | ||
Asset Purchase Agreement [Member] | Scenario, Plan [Member] | Maximum [Member] | Sales Milestone [Member] | |||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | |||
Milestone payments receivable | $ 142 |
License and Asset Purchase Ag_4
License and Asset Purchase Agreements - Additional Information - Ono License Agreement (Detail) - Ono Pharmaceutical Co Ltd [Member] | Oct. 11, 2017USD ($) | Oct. 11, 2017JPY (¥) | Jun. 30, 2019USD ($) | Dec. 31, 2019USD ($) | Dec. 31, 2019JPY (¥) | Sep. 30, 2019USD ($) | Sep. 30, 2019JPY (¥) | Oct. 11, 2017JPY (¥) |
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||||
Revenues | $ 19,700,000 | |||||||
Deferred Revenue, non-current | $ 2,200,000 | |||||||
Government Research Grant Agreement [Member] | ||||||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||||
License agreement termination prior notice period | 180 days | |||||||
License agreement termination description | the Ono License Agreement may be terminated earlier by (i) either party for breach of the Ono License Agreement by the other party or in the event of the insolvency or bankruptcy of the other party, (ii) Ono on a product-by-product basis for certain safety reasons or on a product-by-product, country-by-country basis for any reason with 180 days' prior notice or (iii) us in the event Ono challenges or assists with a challenge to certain of our patent rights. | |||||||
Commercial milestone payments receivable | $ 80,200,000 | ¥ 9,000,000,000 | ||||||
Government Research Grant Agreement [Member] | Development Goals [Member] | Maximum [Member] | ||||||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||||
Milestone payments receivable | $ 90,500,000 | ¥ 10,150,000,000 | ||||||
Government Research Grant Agreement [Member] | Sales Milestone Events [Member] | Maximum [Member] | ||||||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||||
Milestone payments receivable | 80,200,000 | ¥ 9,000,000,000 | ||||||
Government Research Grant Agreement [Member] | Scenario, Plan [Member] | Sales Milestone Events [Member] | Maximum [Member] | ||||||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||||
Milestone payments receivable | $ 1,500,000 | ¥ 1,500,000,000 | ||||||
Government Research Grant Agreement [Member] | Scenario, Plan [Member] | Clinical Development and Regulatory Milestone [Member] | Maximum [Member] | ||||||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||||
Milestone payments receivable | 90,500,000 | ¥ 10,150,000,000 | ||||||
Selinexor [Member] | Government Research Grant Agreement [Member] | ||||||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||||
Performance obligations | 19,700,000 | |||||||
Eltanexor [Member] | Government Research Grant Agreement [Member] | ||||||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||||
Performance obligations | $ 2,200,000 | |||||||
Up-front Payment Arrangement [Member] | Government Research Grant Agreement [Member] | ||||||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||||
Upfront payments | $ 21,900,000 | ¥ 2,500,000,000 |
Net Loss Per Share - Additional
Net Loss Per Share - Additional Information (Detail) | Oct. 16, 2018 | Dec. 31, 2019 |
3% Convertible Senior Notes Due 2025 [Member] | ||
Notes, interest rate | 3.00% | 11.85% |
Stock-based Compensation - Addi
Stock-based Compensation - Additional Information (Detail) $ / shares in Units, $ in Thousands | 1 Months Ended | 12 Months Ended | ||
Oct. 31, 2013shares | Dec. 31, 2019USD ($)Installment$ / sharesshares | Dec. 31, 2018USD ($)$ / sharesshares | Dec. 31, 2017USD ($)$ / sharesshares | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Total stock-based compensation expense | $ | $ 15,291 | $ 17,275 | $ 20,405 | |
Employee And Non Employee Stock Option [Member] | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Total stock-based compensation expense | $ | 12,600 | 16,400 | 16,700 | |
Total intrinsic value of stock options exercised | $ | $ | $ 2,200 | $ 6,000 | $ 400 | |
Weighted-average grant date fair values of options granted (in dollars per share) | $ / shares | $ / shares | $ 6.01 | $ 8.91 | $ 7.22 | |
Period for recognition of unrecognized expense | 2 years 7 months 28 days | |||
Total unrecognized stock-based compensation expense related to unvested stock options including estimated forfeitures | $ | $ | $ 26,700 | |||
Unvested Restricted Stock [Member] | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Number of Shares Underlying RSUs, Granted | 1,065,970 | |||
Unvested Restricted Stock Units [Member] | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Total stock-based compensation expense | $ | $ 1,600 | $ 400 | $ 3,400 | |
Period for recognition of unrecognized expense | 3 years 1 month 13 days | |||
Total unrecognized stock-based compensation expense related to unvested stock options including estimated forfeitures | $ | $ | $ 5,800 | |||
Rights Received For Each Restricted Share Unit | Installment | 2 | |||
Service condition that vest in equal annual installment | Installment | 4 | |||
Number of Shares Underlying RSUs, Granted | 1,065,970 | |||
2010 Plan [Member] | Employee And Non Employee Stock Option [Member] | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Expiration period | 10 years | |||
2010 Plan [Member] | Employee And Non Employee Stock Option [Member] | Percent Vesting One Calendar Year from Vesting Start Date [Member] | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Vesting percentage | 25.00% | |||
2010 Plan [Member] | Employee And Non Employee Stock Option [Member] | Percentage Vesting on the First Day of each Month after One Calendar Year [Member] | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Vesting percentage | 2.083% | |||
2010 Plan [Member] | Minimum [Member] | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Common stock shares available for issuance under ESPP | 484,848 | |||
2013 Plan [Member] | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Number of shares of common stock authorized | 969,696 | 1,939,393 | 1,939,393 | 1,675,513 |
Annual increase in number of additional shares authorized under the Plan through December 31, 2023 | 1,939,393 | |||
Percentage applied to the outstanding shares as annual increase in the number of shares authorized for issuance | 4.00% | |||
Number of additional shares available under the 2013 plan | 2,373,779 | |||
2013 Plan [Member] | Maximum [Member] | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Number of additional shares authorized under the 2010 Plan | 2,126,377 | |||
ESPP [Member] | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Number of shares of common stock authorized | 242,424 | |||
Total stock-based compensation expense | $ | $ 1,100 | $ 400 | $ 200 | |
Period for recognition of unrecognized expense | 4 months | |||
Offering period | 6 months | |||
Purchase price of common stock | 85.00% | |||
Common stock shares available for issuance under ESPP | 404,332 | |||
Percentage of shares of common stock available for issuance | 1.00% | |||
Total unrecognized stock-based compensation expense | $ | $ 400 | |||
Employee stock purchase plan, description | In 2013, our shareholders approved an increase in the number of shares of common stock authorized for issuance pursuant to the ESPP to 242,424 shares of common stock, plus an annual increase to be added on the first day of each fiscal year, commencing on January 1, 2015 and ending on December 31, 2023, equal to the lesser of 484,848 shares of our common stock, 1% of the number of outstanding shares on such date, or an amount determined by the Board. | |||
Employee stock purchase plan, amount withheld from employees | $ | $ | $ 1,700 | $ 900 | $ 400 | |
Common stock available for payment related to tax withholding | 415,257 | 98,770 | 57,582 |
Stock-based Compensation - Sche
Stock-based Compensation - Schedule of Total Stock-based Compensation Expense Recognized in Connection with All Share-based Payment Awards (Detail) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Share-based Compensation Arrangement by Share-based Payment Award, Compensation Cost [Line Items] | |||
Total stock-based compensation expense | $ 15,291 | $ 17,275 | $ 20,405 |
Research and Development [Member] | |||
Share-based Compensation Arrangement by Share-based Payment Award, Compensation Cost [Line Items] | |||
Total stock-based compensation expense | 6,457 | 8,686 | 11,208 |
Selling,General and Administrative Expenses [Member] | |||
Share-based Compensation Arrangement by Share-based Payment Award, Compensation Cost [Line Items] | |||
Total stock-based compensation expense | $ 8,834 | $ 8,589 | $ 9,197 |
Stock-based Compensation - Summ
Stock-based Compensation - Summary of Stock Option Activity for Employees and Nonemployees (Detail) - Employee and Nonemployee Stock Option [Member] - USD ($) $ / shares in Units, $ in Thousands | 12 Months Ended | |
Dec. 31, 2019 | Dec. 31, 2018 | |
Aggregate Intrinsic Value,Granted | ||
Shares, Outstanding, Beginning balance | 8,917,084 | |
Shares, Granted | 3,325,390 | |
Shares, Exercised | (394,358) | |
Shares, Forfeited | (2,005,022) | |
Shares, Outstanding, Ending balance | 9,843,094 | 8,917,084 |
Shares, Exercisable | 5,391,153 | |
Weighted-Average Exercise Price Per Share, Outstanding, Beginning balance | $ 13.78 | |
Weighted-Average Exercise Price Per Share, Granted | 8.67 | |
Weighted-Average Exercise Price Per Share, Exercised | 7.18 | |
Weighted-Average Exercise Price Per Share, Forfeited | 13.36 | |
Weighted-Average Exercise Price Per Share, Outstanding, Ending balance | 12.40 | $ 13.78 |
Weighted-Average Exercise Price Per Share, Exercisable | $ 14.65 | |
Weighted-Average Remaining Contractual Term (years), Outstanding | 7 years | 7 years 4 months 24 days |
Weighted-Average Remaining Contractual Term (years), Exercisable | 5 years 7 months 6 days | |
Aggregate Intrinsic Value, Outstanding | $ 82,134 | $ 8,197 |
Aggregate Intrinsic Value, Exercisable | $ 39,879 |
Stock-based Compensation - Sc_2
Stock-based Compensation - Schedule of Assumptions Used to Estimate Fair Value of Each Employee and Non-employee Stock Award on Grant Date Using Black Scholes Option Pricing Model (Detail) - Employee and Nonemployee Stock Option [Member] | 12 Months Ended | ||
Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Dividend | 0.00% | 0.00% | 0.00% |
Minimum [Member] | |||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Volatility | 79.00% | 79.00% | 79.00% |
Expected term (in years) | 5 years 6 months | 5 years 6 months | 5 years 6 months |
Risk-free interest rate | 1.42% | 2.50% | 1.76% |
Maximum [Member] | |||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Volatility | 81.00% | 81.00% | 85.00% |
Expected term (in years) | 6 years | 9 years 9 months 18 days | 9 years 7 months 6 days |
Risk-free interest rate | 2.58% | 3.05% | 2.29% |
Stock-based Compensation - Su_2
Stock-based Compensation - Summary of RSU Activity (Detail) - Unvested Restricted Stock Units [Member] | 12 Months Ended |
Dec. 31, 2019$ / sharesshares | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |
Number of Shares Underlying RSUs, Unvested beginning balance | shares | 25,000 |
Number of Shares Underlying RSUs, Granted | shares | 1,065,970 |
Number of Shares Underlying RSUs, Forfeited | shares | (286,150) |
Number of Shares Underlying RSUs, Vested | shares | (17,500) |
Number of Shares Underlying RSUs, Unvested ending balance | shares | 787,320 |
Weighted-Average Grant Date Fair Value, Unvested beginning balance | $ / shares | $ 9.87 |
Weighted-Average Grant Date Fair Value, Granted | $ / shares | 9.25 |
Weighted-Average Grant Date Fair Value, Forfeited | $ / shares | 9.24 |
Weighted-Average Grant Date Fair Value, Vested | $ / shares | 8.96 |
Weighted-Average Grant Date Fair Value, Unvested ending balance | $ / shares | $ 9.28 |
Stock-based Compensation - Sc_3
Stock-based Compensation - Schedule of Assumptions Used to Estimate Fair Value on Grant Date Using Black Scholes Option Pricing Model (Detail) - ESPP [Member] | 12 Months Ended | ||
Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Expected term (in years) | 6 months | 6 months | 6 months |
Dividend | 0.00% | 0.00% | 0.00% |
Minimum [Member] | |||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Volatility | 61.00% | 48.00% | 48.00% |
Risk-free interest rate | 2.40% | 1.30% | 0.50% |
Maximum [Member] | |||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Volatility | 104.00% | 61.00% | 78.00% |
Risk-free interest rate | 2.50% | 2.10% | 1.30% |
401(k) Plan - Additional Inform
401(k) Plan - Additional Information (Detail) - USD ($) $ in Millions | 12 Months Ended | ||
Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Retirement Benefits [Abstract] | |||
Defined contribution plan, matching percentage | 4.00% | ||
Defined contribution plan, matching amount | $ 1.7 | $ 1.1 | $ 0.6 |
Income Taxes - Additional Infor
Income Taxes - Additional Information (Detail) - USD ($) | 12 Months Ended | |||
Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | Jan. 01, 2018 | |
Income Tax Disclosure [Line Items] | ||||
Federal statutory rate | 21.00% | 21.00% | 34.00% | |
Impact related to provision items for federal rate change | $ 500,000 | |||
Reduction in deferred tax asset | 42,700,000 | |||
Income tax expense | $ 40,000 | 26,000 | $ 63,000 | |
Increase in valuation allowance | 51,700,000 | |||
Net operating loss carryforwards foreign | $ 4,000 | 72,000 | ||
Period over which certain cumulative changes in the ownership interest of significant shareholders will be considered, subject to an annual limitation of net operating loss and tax credit carryforwards | 3 days | |||
Operating Loss And Tax Credit Carryforwards Limitations Threshold Percentage Of Cumulative Changes In Ownership Interest Of Significant Shareholders Considered | 50 | |||
Deferred tax assets, net of valuation allowance | $ 0 | 0 | ||
Interest or penalties related to uncertain tax positions recognized in statements of operations and comprehensive loss | 0 | 0 | ||
Deferred Tax Assets, Valuation Allowance | 224,943,000 | 173,247,000 | ||
Maximum [Member] | ||||
Income Tax Disclosure [Line Items] | ||||
Income tax expense | 100,000 | |||
Germany [Member] | Maximum [Member] | ||||
Income Tax Disclosure [Line Items] | ||||
Income tax expense | 100,000 | |||
Accounting Standards Update 2016-16 [Member] | ||||
Income Tax Disclosure [Line Items] | ||||
Deferred tax assets, net of valuation allowance | $ 19,200,000 | |||
Internal Revenue Service (IRS) [Member] | ||||
Income Tax Disclosure [Line Items] | ||||
Net operating loss carryforwards | 576,500,000 | 427,000,000 | ||
Net operating loss carryforwards indefinite life | 576,500,000 | |||
Internal Revenue Service (IRS) [Member] | Research Tax Credit Carryforward [Member] | ||||
Income Tax Disclosure [Line Items] | ||||
Tax credit carryforwards | 58,500,000 | 46,900,000 | ||
State and Local Jurisdiction [Member] | ||||
Income Tax Disclosure [Line Items] | ||||
Net operating loss carryforwards | 502,300,000 | 414,800,000 | ||
Net operating loss carryforwards indefinite life | 283,600,000 | |||
Net operating loss carryforwards indefinite life remaining | 292,900,000 | |||
State and Local Jurisdiction [Member] | Maximum [Member] | ||||
Income Tax Disclosure [Line Items] | ||||
Net operating loss carryforwards foreign | 100,000 | |||
State and Local Jurisdiction [Member] | Research Tax Credit Carryforward [Member] | ||||
Income Tax Disclosure [Line Items] | ||||
Tax credit carryforwards | $ 4,900,000 | $ 3,000,000 |
Income Taxes - Schedule of Comp
Income Taxes - Schedule of Components of Income (Loss) Before Income Taxes (Detail) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Components of Income Tax Expense (Benefit), Continuing Operations [Abstract] | |||
Foreign | $ (23,350) | $ (28,689) | $ (35,680) |
U.S. | (176,200) | (149,692) | (93,241) |
Totals | $ (199,550) | $ (178,381) | $ (128,921) |
Income Taxes - Schedule of Sign
Income Taxes - Schedule of Significant Components of Company's Deferred Tax Assets (Detail) - USD ($) $ in Thousands | Dec. 31, 2019 | Dec. 31, 2018 |
Deferred tax assets: | ||
U.S. and state net operating loss carryforwards | $ 150,909 | $ 116,236 |
Stock-based compensation | 11,914 | 11,228 |
Accruals and other temporary differences | 4,212 | 3,605 |
Research and development credits | 62,374 | 49,307 |
Capitalized research and development | 1,021 | 1,388 |
Fixed Assets and Intangibles | 5,893 | 6,422 |
Deferred revenue | 1,051 | 527 |
Foreign net operating loss carryforwards | 4 | 72 |
Lease liability | 3,443 | |
Deferred Tax Assets Royalty Embaded Derivatives | 533 | |
Valuation allowance | (224,943) | (173,247) |
Total deferred tax assets | 16,411 | 15,538 |
Deferred tax liabilities: | ||
Convertible debt amortization | (13,431) | (15,538) |
Right-of-use asset | (2,462) | |
Deferred royalty obligation | (518) | |
Total deferred tax liabilities | (16,411) | (15,538) |
Net deferred tax assets | $ 0 | $ 0 |
Income Taxes - Schedule of Reco
Income Taxes - Schedule of Reconciliation of Income Tax Expense Computed at Statutory Federal Income Tax Rate to Income Taxes (Detail) | 12 Months Ended | ||
Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Effective Income Tax Rate Reconciliation, Percent [Abstract] | |||
Federal income tax expense at statutory rate | 21.00% | 21.00% | 34.00% |
State income tax, net of federal benefit | 1.70% | 0.70% | 5.90% |
Permanent differences | (0.50%) | 0.00% | (3.90%) |
Research and development credit | 6.00% | 7.80% | 8.40% |
Foreign rate differential | (2.50%) | (3.40%) | (9.40%) |
Change in valuation allowance | (25.90%) | (28.20%) | (1.40%) |
Provision to return adjustments | 0.60% | 2.40% | 1.00% |
Other | (0.40%) | (0.60%) | (1.40%) |
Federal rate change | 0.00% | 0.30% | (33.20%) |
Effective income tax rate | 0.00% | 0.00% | 0.00% |
Long-Term Obligations - Additio
Long-Term Obligations - Additional Information (Detail) | Oct. 15, 2022d | Oct. 16, 2018USD ($)$ / sharesshares | Sep. 30, 2019USD ($) | Dec. 31, 2019USD ($)d | Dec. 31, 2018USD ($) | Oct. 26, 2018USD ($) |
Subsequent Event [Line Items] | ||||||
Debt issuance costs allocated to equity component and recorded as a reduction to additional paid-in capital | $ 67,850,000 | |||||
Notes, maturity date | Oct. 15, 2025 | |||||
Debt instrument convertible threshold consecutive trading days | d | 5 | |||||
Description of debt instrument convertible period | during the five business day period immediately after any five consecutive trading day | |||||
Principal amount of notes used in conversion rate | $ 1,000 | |||||
Debt instrument convertible threshold maximum percentage of product of last reported sale price of common stock | 98.00% | |||||
Equity component of convertible notes recognized as debt discount | $ 67,900,000 | |||||
Proceeds from issuance of convertible notes | 172,500,000 | |||||
Fair value of liability of convertible notes | 104,700,000 | |||||
Estimated fair value of convertible notes | $ 251,000,000 | |||||
Expected life of convertible notes | 7 years | |||||
Deferred Royalty Obligation [Member] | Level 3 [Member] | ||||||
Subsequent Event [Line Items] | ||||||
Deferred royalty obligation at fair value | $ 71,300,000 | |||||
Revenue Interest Financing Agreement [Member] | ||||||
Subsequent Event [Line Items] | ||||||
Debt issuance costs | 1,400,000 | |||||
First investment amount | $ 75,000,000 | $ 75,000,000 | ||||
Second investment amount | $ 75,000,000 | |||||
Debt Instrument Interest Rate | 18.40% | |||||
Revenue Interest Financing Agreement [Member] | HealthCare Royalty Partners IV LP [Member] | ||||||
Subsequent Event [Line Items] | ||||||
Aggregate Royalties Percentage | 185.00% | |||||
Revenue Interest Financing Agreement [Member] | HealthCare Royalty Partners IV LP [Member] | Royalty Due On December 31, 2022 [Member] | ||||||
Subsequent Event [Line Items] | ||||||
Royalty Payable Percentage | 65.00% | |||||
Revenue Interest Financing Agreement [Member] | HealthCare Royalty Partners IV LP [Member] | Royalty Due On December 31, 2024 [Member] | ||||||
Subsequent Event [Line Items] | ||||||
Royalty Payable Percentage | 100.00% | |||||
Convertible Note Offering [Member] | ||||||
Subsequent Event [Line Items] | ||||||
Notes converted in to common stock, amount | shares | 63.0731 | |||||
Notes converted in to common stock, shares | $ 1 | |||||
Notes, conversion price per share | $ / shares | $ 15.85 | |||||
3% Convertible Senior Notes Due 2025 [Member] | ||||||
Subsequent Event [Line Items] | ||||||
Aggregate principal amount | $ 150,000,000 | $ 22,500,000 | ||||
Debt issuance costs | $ 5,600,000 | |||||
Debt issuance costs allocated to equity component and recorded as a reduction to additional paid-in capital | 2,200,000 | |||||
Debt issuance costs allocated to liability component and recorded as a reduction of convertible notes | $ 3,400,000 | |||||
Debt discount and issuance costs amortized to interest expense, amortization period | 7 years | |||||
Notes, interest rate | 3.00% | 11.85% | ||||
Notes, maturity date | Oct. 15, 2025 | |||||
Principal amount of notes used in conversion rate | $ 1 | |||||
Debt instrument, convertible latest date | Jun. 15, 2025 | |||||
Notes conversion price, percentage | 130.00% | |||||
Notes instrument, trading days | d | 20 | |||||
Debt instrument convertible threshold consecutive trading days | d | 30 | |||||
Initial amount of liability component | $ 101,200,000 | |||||
Equity component of convertible notes recognized as debt discount | $ 65,641,000 | |||||
3% Convertible Senior Notes Due 2025 [Member] | Scenario, Forecast [Member] | ||||||
Subsequent Event [Line Items] | ||||||
Notes conversion price, percentage | 130.00% | |||||
Notes instrument, trading days | d | 20 | |||||
Debt instrument convertible threshold consecutive trading days | d | 30 | |||||
Notes, repurchase price | 100.00% |
Long-Term Obligations - Summary
Long-Term Obligations - Summary of Outstanding Balances of Convertible Notes (Detail) $ in Thousands | Dec. 31, 2019USD ($) |
Liability component: | |
Equity component | $ 67,900 |
3% Convertible Senior Notes Due 2025 [Member] | |
Liability component: | |
Principal | 172,500 |
Less: debt discount and issuance costs, net | (62,643) |
Net carrying amount | 109,857 |
Equity component | $ 65,641 |
Long-Term Obligations - Schedul
Long-Term Obligations - Schedule of Interest Expense Recognized Related to Convertible Notes (Detail) - 3% Convertible Senior Notes Due 2025 [Member] $ in Thousands | 12 Months Ended |
Dec. 31, 2019USD ($) | |
Debt Instrument [Line Items] | |
Contractual interest expense | $ 5,175 |
Amortization of debt discount | 6,849 |
Amortization of debt issuance costs | 344 |
Total interest expense | $ 12,368 |
Long-Term Obligations - Summa_2
Long-Term Obligations - Summary of Future Minimum Payments on Convertible Notes (Detail) - 3% Convertible Senior Notes Due 2025 [Member] $ in Thousands | Dec. 31, 2019USD ($) |
Debt Instrument [Line Items] | |
2020 | $ 5,175 |
2021 | 5,175 |
2022 | 5,175 |
2023 | 5,175 |
2024 and thereafter | 182,850 |
Total minimum payments | 203,550 |
Less: interest | (31,050) |
Less: unamortized discount | (62,643) |
Less: current portion | |
Convertible senior notes | $ 109,857 |