Document and Entity Information
Document and Entity Information - shares | 9 Months Ended | |
Sep. 30, 2018 | Nov. 02, 2018 | |
Document And Entity Information [Abstract] | ||
Document Type | 10-Q | |
Amendment Flag | false | |
Document Period End Date | Sep. 30, 2018 | |
Document Fiscal Year Focus | 2,018 | |
Document Fiscal Period Focus | Q3 | |
Trading Symbol | KPTI | |
Entity Registrant Name | KARYOPHARM THERAPEUTICS INC. | |
Entity Central Index Key | 1,503,802 | |
Current Fiscal Year End Date | --12-31 | |
Entity Filer Category | Accelerated Filer | |
Entity Small Business | false | |
Entity Emerging Growth Company | true | |
Entity Ex Transition Period | true | |
Entity Common Stock, Shares Outstanding | 60,707,876 |
Condensed Consolidated Balance
Condensed Consolidated Balance Sheets - USD ($) $ in Thousands | Sep. 30, 2018 | Dec. 31, 2017 |
Current assets: | ||
Cash and cash equivalents | $ 101,600 | $ 68,997 |
Short-term investments | 105,170 | 77,472 |
Prepaid expenses and other current assets | 4,792 | 1,754 |
Restricted cash | 200 | |
Total current assets | 211,562 | 148,423 |
Property and equipment, net | 2,914 | 2,185 |
Long-term investments | 4,804 | 29,396 |
Restricted cash | 712 | 290 |
Total assets | 219,992 | 180,294 |
Current liabilities: | ||
Accounts payable | 2,238 | 5,665 |
Accrued expenses | 29,155 | 21,445 |
Deferred revenue | 9,362 | 21,921 |
Deferred rent | 256 | 303 |
Other current liabilities | 556 | 133 |
Total current liabilities | 41,567 | 49,467 |
Deferred revenue, net of current portion | 4,532 | |
Deferred rent, net of current portion | 2,815 | 1,363 |
Total liabilities | 48,914 | 50,830 |
Stockholders' equity: | ||
Preferred stock, $0.0001 par value; 5,000,000 shares authorized; none issued and outstanding | ||
Common stock, $0.0001 par value; 100,000,000 shares authorized; 60,664,857 and 49,533,150 shares issued and outstanding at September 30, 2018 and December 31, 2017, respectively | 6 | 5 |
Additional paid-in capital | 786,763 | 625,017 |
Accumulated other comprehensive loss | (153) | (217) |
Accumulated deficit | (615,538) | (495,341) |
Total stockholders' equity | 171,078 | 129,464 |
Total liabilities and stockholders' equity | $ 219,992 | $ 180,294 |
Condensed Consolidated Balanc_2
Condensed Consolidated Balance Sheets (Parenthetical) - $ / shares | Sep. 30, 2018 | Dec. 31, 2017 |
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 | 100,000,000 | 100,000,000 |
Common stock, shares issued | 60,664,857 | 49,533,150 |
Common stock, shares outstanding | 60,664,857 | 49,533,150 |
Condensed Consolidated Statemen
Condensed Consolidated Statements of Operations - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2018 | Sep. 30, 2017 | Sep. 30, 2018 | Sep. 30, 2017 | |
Income Statement [Abstract] | ||||
License and other revenue | $ 239 | $ 30,130 | $ 71 | |
Operating expenses: | ||||
Research and development | 36,427 | $ 25,237 | 122,482 | 72,440 |
General and administrative | 12,966 | 5,818 | 30,076 | 18,717 |
Total operating expenses | 49,393 | 31,055 | 152,558 | 91,157 |
Loss from operations | (49,154) | (31,055) | (122,428) | (91,086) |
Other income (expense): | ||||
Interest income | 1,098 | 454 | 2,260 | 1,266 |
Other expense | (13) | (26) | (20) | (70) |
Total other income, net | 1,085 | 428 | 2,240 | 1,196 |
Loss before income taxes | (48,069) | (30,627) | (120,188) | (89,890) |
Income tax provision | (14) | (13) | (9) | (54) |
Net loss | $ (48,083) | $ (30,640) | $ (120,197) | $ (89,944) |
Net loss per share-basic and diluted | $ (0.79) | $ (0.65) | $ (2.17) | $ (2) |
Weighted-average number of common shares outstanding used in net loss per share-basic and diluted | 60,586,511 | 47,141,146 | 55,465,261 | 44,974,945 |
Condensed Consolidated Statem_2
Condensed Consolidated Statements of Comprehensive Loss - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2018 | Sep. 30, 2017 | Sep. 30, 2018 | Sep. 30, 2017 | |
Statement of Comprehensive Income [Abstract] | ||||
Net loss | $ (48,083) | $ (30,640) | $ (120,197) | $ (89,944) |
Comprehensive income | ||||
Unrealized gain on investments | 110 | 25 | 105 | 50 |
Foreign currency translation adjustments | (3) | 50 | (41) | 134 |
Comprehensive loss | $ (47,976) | $ (30,565) | $ (120,133) | $ (89,760) |
Condensed Consolidated Statem_3
Condensed Consolidated Statements of Cash Flows - USD ($) $ in Thousands | 9 Months Ended | |
Sep. 30, 2018 | Sep. 30, 2017 | |
Operating activities | ||
Net loss | $ (120,197) | $ (89,944) |
Adjustments to reconcile net loss to net cash used in operating activities: | ||
Depreciation and amortization | 541 | 539 |
Net amortization of premiums and discounts on investments | 280 | 903 |
Stock-based compensation expense | 13,378 | 15,906 |
Changes in operating assets and liabilities: | ||
Prepaid expenses and other current assets | (3,039) | 18 |
Accounts payable | (3,425) | (2,867) |
Accrued expenses and other liabilities | 8,139 | 6,423 |
Deferred revenue | (8,027) | 1,050 |
Deferred rent | 1,405 | (207) |
Net cash used in operating activities | (110,945) | (68,179) |
Investing activities | ||
Purchases of property and equipment | (1,270) | (7) |
Proceeds from maturities of investments | 94,378 | 94,033 |
Purchases of investments | (97,662) | (74,017) |
Net cash (used in) provided by investing activities | (4,554) | 20,009 |
Financing activities | ||
Proceeds from the issuance of common stock, net of issuance costs | 145,706 | 52,323 |
Proceeds from the exercise of stock options and shares issued under employee stock purchase plan | 2,627 | 463 |
Net cash provided by financing activities | 148,333 | 52,786 |
Effect of exchange rate on cash, cash equivalents and restricted cash | (9) | 181 |
Net increase in cash, cash equivalents and restricted cash | 32,825 | 4,797 |
Cash, cash equivalents and restricted cash at beginning of period | 69,487 | 50,142 |
Cash, cash equivalents and restricted cash at end of period | 102,312 | 54,939 |
Reconciliation of cash, cash equivalents and restricted cash reported within the condensed consolidated balance sheets | ||
Cash and cash equivalents | 101,600 | 54,450 |
Short-term restricted cash | 200 | |
Long-term restricted cash | 712 | 289 |
Cash, cash equivalents and restricted cash at end of period | $ 102,312 | $ 54,939 |
Summary of Significant Accounti
Summary of Significant Accounting Policies | 9 Months Ended |
Sep. 30, 2018 | |
Accounting Policies [Abstract] | |
Summary of Significant Accounting Policies | 1. Summary of Significant Accounting Policies Basis of Presentation The accompanying unaudited condensed consolidated financial statements of Karyopharm Therapeutics Inc., a Delaware corporation (the “Company”), have been prepared in accordance with accounting principles generally accepted in the United States (“GAAP”) for interim financial reporting and as required by Regulation S-X, Rule 10-01. Form 10-K At September 30, 2018, the Company had $211,574 in cash, cash equivalents and short- and long-term investments. The Company has had recurring losses and incurred a loss of $120,197 for the nine months ended September 30, 2018. Net cash used in operations for the nine months ended September 30, 2018 was $110,945. The Company expects that cash, cash equivalents and short- and long-term investments at September 30, 2018, in addition to the approximately $166,900 of net proceeds the Company raised in the private placement of the Notes (see Note 12) in October 2018, will be sufficient to fund its current operating plans and capital expenditure requirements for at least twelve months from the date of issuance of the financial statements contained in this Form 10-Q Basis of Consolidation The condensed consolidated financial statements at September 30, 2018 include the accounts of (i) the Company, (ii) Karyopharm Securities Corp. (a wholly-owned Massachusetts corporation of the Company incorporated in December 2013), (iii) Karyopharm Europe GmbH (a wholly-owned German Limited Liability Company formed in August 2014), (iv) Karyopharm Therapeutics (Bermuda) Ltd. (a wholly-owned Bermuda subsidiary of the Company formed in March 2015), and (v) Karyopharm Israel Ltd. (a wholly-owned Israeli subsidiary of the Company formed in June 2018). All intercompany balances and transactions have been eliminated in consolidation. The significant accounting policies used in preparation of these condensed consolidated financial statements on Form 10-Q Form 10-K Revenue Recognition The Company adopted Accounting Standards Update (“ASU”) 2014-09, Revenue from Contracts with Customers ( Revenue Recognition 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, an entity recognizes revenue when its customer obtains control of promised goods or services, in an amount that reflects the consideration which the entity expects to receive in exchange for those goods or services. To determine revenue recognition for arrangements that an entity determines are within the scope of ASC 606, the entity performs the following five steps: (i) identify the contract(s) with a customer; (ii) identify the performance obligations in the contract; (iii) determine the transaction price; (iv) allocate the transaction price to the performance obligations in the contract; and (v) recognize revenue when (or as) the entity satisfies a performance obligation. At contract inception, once the contract is determined to be within the scope of ASC 606, the Company assesses the goods or services promised within each contract and determines those that are performance obligations and assesses whether each promised good or service is distinct. The Company then recognizes as revenue the amount of the transaction price that is allocated to the respective performance obligation when (or as) the performance obligation is satisfied. The Company generates revenue from license or similar agreements with pharmaceutical companies for the development and commercialization of certain of its 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 the Company’s intellectual property is determined to be distinct from the other performance obligations identified in the arrangement, the Company recognizes the transaction price allocated to the license as revenue upon transfer of control of the license. The Company evaluates 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 the Company 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. The Company utilizes judgment to determine the transaction price. In connection therewith, the Company evaluates 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 the control of the Company, 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 the Company recognizes revenue as or when the performance obligations under the contract are satisfied. At the end of each reporting period, the Company re-evaluates catch-up The Company then determines 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 is deemed to be the predominant item to which the royalties relate, the Company recognizes 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). |
Recent Accounting Pronouncement
Recent Accounting Pronouncements | 9 Months Ended |
Sep. 30, 2018 | |
Accounting Changes and Error Corrections [Abstract] | |
Recent Accounting Pronouncements | 2. Recent Accounting Pronouncements Recently Adopted Accounting Standards As detailed above, the Company adopted ASC 606 on January 1, 2018. Under the modified retrospective transition method, the Company applied ASC 606 to all contracts within scope as of January 1, 2018. Under the practical expedient concerning contract modifications contained in the transitional provisions of ASC 606, the Company has not retrospectively restated its contracts for modifications prior to the earliest period presented, and instead has reflected the aggregate effect of all modifications when identifying the satisfied and unsatisfied performance obligations, determining the transaction price and allocating the transaction price. Qualitatively, the effect of applying this practical expedient is not material to the periods presented in the consolidated financial statements. As more fully discussed in Note 3, Asset Purchase and License Agreements, only the Company’s arrangement with Ono Pharmaceutical Co., Ltd. was determined to have unsatisfied performance obligations as of the adoption date. However, the pattern of revenue recognition was not affected and, therefore, no transition adjustment was recorded to the opening balance of accumulated deficit on January 1, 2018. All other agreements subject to transition, which only included the Company’s arrangement with Anivive Lifesciences Inc., were unaffected by the adoption of ASC 606 in all periods presented in the consolidated financial statements through application of the modified retrospective transition method. In August 2016, the Financial Accounting Standards Boards (“FASB”) issued ASU 2016-15, Classification of Certain Cash Receipts and Cash Payments (“ASU 2016-15”). ASU 2016-05 In October 2016, the FASB issued ASU No. 2016-16, Accounting for Income Taxes: Intra-Entity Asset Transfers of Assets Other than Inventory (Topic 740) In November 2016, the FASB issued ASU No. 2016-18, Statement of Cash Flows (Topic 230): Restricted Cash beginning-of-period end-of-period In May 2017, the FASB issued ASU 2017-09, Compensation-Stock Compensation (Topic 718) (“ASU 2017-09”) Scope of Modification Accounting ASU 2017-09 non-substantive. On December 22, 2017, the Tax Cuts and Jobs Act (“TCJA”) was enacted and led to significant changes to U.S. tax law. Also on December 22, 2017, the SEC staff issued Staff Accounting Bulletin No. 118 (“SAB 118”), allowing companies to record the effects of the TCJA on a provisional basis during a measurement period not to extend beyond one year of the enactment date. SAB 118 was codified into ASC 740 by ASU 2018-05. Recently Issued Accounting Standards On August 17, 2018, the SEC issued an amendment to Rule 3-04 Regulation S-X, year-to-date Form 10-Q. In June 2018, the FASB issued ASU No. 2018-07, Compensation-Stock Compensation (Topic 718): Improvements to Nonemployee Share-Based Payment Accounting (“ASU 2018-07”). ASU 2018-07 In February 2016, the FASB issued ASU No. 2016-02, Leases (Topic 842) ASU 2018-11, Leases (Topic 842) Targeted Improvements Pursuant to the guidance under ASU No. 2016-02, non-lease In July 2018, the FASB issued ASU No. 2018-09, No. 2018-09 No. 2018-09 In August 2018, the FASB issued ASU No. 2018-13, No. 2018-13 No. 2018-13 No. 2018-13 In August 2018, the FASB issued ASU No. 2018-15, Intangible-Goodwill and Other Internal-Use 350-40) No. 2018-15 No. 2018-15 |
License and Asset Purchase Agre
License and Asset Purchase Agreements | 9 Months Ended |
Sep. 30, 2018 | |
Revenue from Contract with Customer [Abstract] | |
License and Asset Purchase Agreements | 3. License and Asset Purchase Agreements Antengene License Agreement Effective May 23, 2018 (the “Antengene Effective Date”), the Company 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 the Company granted Antengene exclusive rights to develop and commercialize, at its own cost, (i) selinexor, the Company’s lead, novel, oral Selective Inhibitor of Nuclear Export (“SINE”) compound, (ii) eltanexor, the Company’s 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, the Company received an upfront payment of $11,703, and could receive up to $105,000 in milestone payments if certain development goals are achieved and up to $45,000 in milestone payments if certain sales milestones are achieved, as well as a high single-digit to low double-digit royalty based on future net sales of the Antengene Licensed Compounds in the Antengene Territory. In addition, upon Antengene’s election and the parties’ full execution of a manufacturing technology transfer plan and satisfaction of other specified conditions (the “Antengene Manufacturing Election”), the Company will grant to Antengene 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 Until such time as Antengene elects to manufacture its own drug substance, the Company will furnish clinical supplies of drug substance to Antengene for use in Antengene’s development efforts pursuant to a clinical supply agreement to be entered into by the Company and Antengene, and Antengene may elect to have the Company provide commercial supplies of drug product to Antengene pursuant to a commercial supply agreement to be entered into by the Company and Antengene, in each case the costs of which will be borne by Antengene. The Antengene License Agreement will continue in effect on a product-by-product, country-by-country product-by-product product-by-product, country-by-country The Company assessed the Antengene arrangement in accordance with ASC 606 and concluded that the contract counterparty, Antengene, is a customer. The Company 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 the Company 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. The Company also identified immaterial promises under the contract relating to information exchanges and participation on operating committees and other working groups. Separately, the Company also identified certain customer options that would create an obligation for the Company 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 the Company 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, the Company also identified certain other customer options that would create a manufacturing obligation for the Company 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, the Company 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”, “Antengene Combined License Obligation for eltanexor”, “Antengene Combined License Obligation for KPT-9274” KPT-9274, The Company 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 $11,703 up-front re-evaluate Through September 30, 2018, the Company has recognized no revenue under the Antengene License Agreement. Revenue will be recognized for (i) the Antengene Combined License Obligation for selinexor once the initial clinical supply of selinexor is delivered, which is currently expected to occur before December 31, 2018. Revenue will be recognized for (ii) the Antengene Combined License Obligation for eltanexor, (iii) the Antengene Combined License Obligation for KPT-9274, non-current Biogen Asset Purchase Agreement On January 24, 2018, the Company 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, the Company sold to Biogen exclusive worldwide rights to develop and commercialize the Company’s oral SINE compound KPT-350 KPT-350 know-how KPT-350 KPT-350 KPT-350 KPT-350 country-by-country The Company 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 The Company assessed this arrangement in accordance with ASC 606 and concluded that the contract counterparty, Biogen, is a customer. The Company identified the following material promises in the arrangement: the Transfer of IP and the Manufacturing License. The Company also identified immaterial promises under the contract that were not deemed performance obligations. The Company further determined that other promises for Additional Supply and Transition Assistance represented customer options, which would create an obligation for the Company if exercised by Biogen. Since no additional or material consideration is owed to the Company by Biogen upon exercise of the customer options for Additional Supply and Transition Assistance, the Company determined that both are offered at significant and incremental discounts. Accordingly, they were assessed as material rights and, therefore, separate performance obligations in the arrangement. The Company then determined that the Transfer of IP and the Manufacturing License were not distinct from one another and must be combined as a performance obligation (the “Combined Performance Obligation”). This is because Biogen requires the Manufacturing License to derive benefit from the Transfer of IP. Based on these determinations, as well as the considerations noted above with respect to the material rights for Additional Supply and Transition Assistance, the Company identified three distinct performance obligations at the inception of the contract: (i) the Combined Performance Obligation, (ii) the material right for Additional Supply, and (iii) the material right for Transition Assistance. The Company further determined that the up-front Upon execution of the APA, the transaction price included only the $10,000 up-front re-evaluate During the nine months ended September 30, 2018, the Company recognized $10,000 of revenue, as it had satisfied its promises under the Combined Performance Obligation by transferring the underlying promised goods during the first quarter of 2018. Ono License Agreement Effective October 11, 2017 (the “Ono Effective Date”), the Company 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 the Company 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”) (the “Ono Exclusive License”). Pursuant to the terms of the Ono License Agreement, the Company received an upfront payment of ¥2.5 billion (US$21,916 on the date received), and could receive up to ¥10.15 billion (approximately US$90,500 at the exchange rate as of the Ono Effective Date) in milestone payments if certain development goals are achieved and up to ¥9.0 billion (approximately US$80,200 at the exchange rate as of the Ono Effective Date) in milestone payments if certain sales milestones are achieved, as well as a low double-digit royalty based on future net sales of selinexor and eltanexor in the Ono Territory. In addition, upon Ono’s election and the parties’ full execution of a manufacturing technology transfer plan and satisfaction of other specified conditions (the “Ono Manufacturing Election”), the Company will grant to Ono 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, the Company will furnish clinical supplies of drug substance to Ono for use in Ono’s development efforts pursuant to a clinical supply agreement to be entered into by the Company and Ono, and Ono may elect to have the Company provide commercial supplies of drug product to Ono pursuant to a commercial supply agreement to be entered into by the Company 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 product-by-product product-by-product, country-by-country The Company assessed this arrangement in accordance with ASC 606 and concluded that the contract counterparty, Ono, is a customer. The Company 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 the Company 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. The Company also identified immaterial promises under the contract relating to information exchanges, and participation on operating committees and other working groups. Separately, the Company also identified certain customer options that would create an obligation for the Company 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 the Company 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. The Company also identified certain other customer options that would create a manufacturing obligation for the Company 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, the Company 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 the Company did not grant manufacturing licenses for selinexor and eltanexor at contract inception. The Company also determined that each of the Ono Transfer Options represents a distinct performance obligation. Based on these determinations, the Company 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. The Company further determined that the up-front Upon execution of the Ono License Agreement, the transaction price included only the ¥2.5 billion (US$21,916 on the date received) up-front 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 $19,724 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 the Company’s 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 September 30, 2018, $2,192 of the Ono License Agreement upfront payment is included in deferred revenue and is classified as a non-current Given the determination that the license rights conveyed to Ono lacked standalone value from the initial clinical supply of product required for Ono to obtain benefit from the rights granted and the fact that no initial clinical supply had been provided to Ono as of December 31, 2017, the Company concluded that no revenue should be recognized under ASC 605. Arrangement consideration at the inception of the arrangement included the ¥2.5 billion (US$21,916 on the date received) upfront payment. All other forms of consideration such as milestones and royalties, were considered contingent consideration, with no amount allocable to deliverables at the inception of the arrangement. The Company concluded that the contingent consideration would be recognized when the underlying contingencies have been resolved, assuming all other revenue recognition criteria are met. As the accounting treatment for this agreement did not materially differ under ASC 605 and ASC 606, and no revenue was recognized under the Company’s previous accounting policy through December 31, 2017, no transition adjustment was recorded to the opening balance of accumulated deficit as of January 1, 2018. Accordingly, the upfront payment of ¥2.5 billion (US$21,916 on the date received), which represents a contract liability, was also included in deferred revenue as of December 31, 2017. MMRF Research Agreement The Company is a party to a research agreement with the Multiple Myeloma Research Foundation (“MMRF”). Under this research agreement, the Company is obligated to make certain payments to MMRF, including if the Company out-licenses KPT-9274 mid-single-digit Anivive License Agreement On April 28, 2017 (the “Anivive Effective Date”), the Company entered into a license agreement (the “Anivive Agreement”) with Anivive Lifesciences, Inc. (“Anivive”), a biopharmaceutical company engaged in the research, development and commercialization of animal health medicines, pursuant to which the Company has granted Anivive an exclusive, worldwide license to develop and commercialize verdinexor (KPT-335) The Company assessed this arrangement in accordance with ASC 606 and concluded that the contract counterparty, Anivive, is a customer. The Company identified the following material promises under the contract, the Anivive Exclusive License and the technology transfer, which consisted of regulatory data compiled by the Company for the licensed compound and product as of the Anivive Effective Date. The Company also identified immaterial promises under the contract that were not deemed performance obligations, including participating on a product advisory committee and sharing regulatory matter information. The Company further determined that other promises for (i) transfer of additional technology in the future, if developed by the Company, and (ii) facilitating manufacturing and supply relationships with the Company’s third-party contract manufacturers represented customer options, would create an obligation for the Company if exercised by Anivive. Since no additional or immaterial consideration is owed to the Company by Anivive upon exercise of the customer options noted, the Company determined that both are offered at significant and incremental discounts. Accordingly, they were assessed as material rights and, therefore, separate performance obligations in the arrangement. In further evaluating the promises detailed above, the Company determined that the Anivive Exclusive License and the technology transfer were not distinct from one another and must be combined as a performance obligation (the “Anivive Combined License Obligation”). This is because Anivive requires the technology transfer to derive benefit from the Anivive Exclusive License. Based on these determinations, the Company identified three distinct performance obligations at the inception of the contract: (i) the Anivive Combined License Obligation, (ii) the material right for transfer of additional technology in the future, if developed by the Company, and (iii) the material right for facilitating manufacturing and supply relationships with the Company’s third-party contract manufacturers. The Company further determined that the up-front As referenced above, the up-front re-evaluate To date, the Company recognized $1,250 of revenue associated with the Anivive Agreement. Revenue for the upfront payment and technology transfer milestone was recognized upon completion of the technology transfer in October 2017, as all promises under the Anivive Combined License Obligation had been fulfilled. The Company reached similar conclusions when evaluating this agreement under its previous accounting policy, which was based on legacy guidance within ASC 605. When evaluating this agreement under ASC 605, the Company concluded that the licenses to verdinexor and technology transfer concerning the licensed product are essential to Anivive’s intended use of the license to develop and commercialize the licensed compound and represented a single unit of accounting. Other potential contractual obligations were evaluated and determined not to be deliverables at inception of the arrangement or were evaluated and determined to be immaterial to the arrangement and, therefore, not evaluated further in the Company’s analysis. Arrangement consideration at the inception of the arrangement included the $1,250 in upfront payments, which includes the milestone fee upon completion of the technology transfer. All other forms of consideration, such as milestones and royalties, were considered contingent consideration, with no amount allocable to deliverables at the inception of the arrangement. The Company concluded that the contingent consideration would be recognized when the underlying contingencies have been resolved, assuming all other revenue recognition criteria are met. Given the single unit of accounting and that the technology transfer would be the last item to be delivered within the unit of accounting, the Company concluded that revenue would be recognized upon the completion of delivery of the technology transfer assuming all other general revenue recognition criteria would be met as of that date. As the accounting treatment for this agreement did not materially differ under ASC 605 and ASC 606, and the upfront payment and technology transfer fee, totaling $1,250, was recognized as revenue during the year ended December 31, 2017 in accordance with the Company’s previous accounting policy, and would have also been recognized during the year ended December 31, 2017 in accordance with the Company’s accounting policy under ASC 606, no transition adjustment was recorded to the opening balance of accumulated deficit as of January 1, 2018. |
Fair Value of Financial Instrum
Fair Value of Financial Instruments | 9 Months Ended |
Sep. 30, 2018 | |
Fair Value Disclosures [Abstract] | |
Fair Value of Financial Instruments | 4. Fair Value of Financial Instruments Financial instruments, including cash, restricted cash, prepaid expenses and other current assets, accounts payable and accrued expenses are presented in the condensed consolidated financial statements at amounts that approximate fair value at September 30, 2018 and December 31, 2017. The Company is 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 the Company’s own assumptions about the assumptions market participants would use in pricing the asset or liability 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. The Company estimates 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. The Company validates the prices provided by its third-party pricing sources by understanding the models used, obtaining market values from other pricing sources and analyzing pricing data in certain instances. The following table presents information about the Company’s financial assets that have been measured at fair value at September 30, 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 $ 70,876 $ 70,876 $ — $ — Investments: Current: Corporate debt securities 87,205 — 87,205 — Commercial paper 8,988 — 8,988 — U.S. government and agency securities 2,477 — 2,477 — Certificates of deposit 6,500 — 6,500 — Non-current: Corporate debt securities (one to two year maturity) 4,804 — 4,804 — $ 180,850 $ 70,876 $ 109,974 $ — The following table presents information about the Company’s financial assets that have been measured at fair value at December 31, 2017 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 $ 41,805 $ 41,805 $ — $ — Investments: Current: Corporate debt securities 66,253 — 66,253 — Commercial paper 6,720 — 6,720 — Certificates of deposit 2,500 — 2,500 — U.S. government and agency securities 1,999 — 1,999 — Non-current: — Corporate debt securities (one to two year maturity) 26,916 — 26,916 — U.S. government securities and agency securities 2,480 — 2,480 — $ 148,673 $ 41,805 $ 106,868 $ — |
Investments
Investments | 9 Months Ended |
Sep. 30, 2018 | |
Investments, Debt and Equity Securities [Abstract] | |
Investments | 5. Investments The following table summarizes the Company’s investments in debt securities, classified as available-for-sale, Amortized Cost Gross Unrealized Gross Unrealized Fair Value Current: Corporate debt securities $ 87,317 $ 6 $ (118 ) $ 87,205 Commercial paper 8,988 — — 8,988 U.S. government and agency securities 2,500 — (23 ) 2,477 Certificates of deposit 6,500 — — 6,500 Non-current: Corporate debt securities (one to two year maturity) 4,817 — (13 ) 4,804 $ 110,122 $ 6 $ (154 ) $ 109,974 The following table summarizes the Company’s investments in debt securities, classified as available-for-sale, Amortized Cost Gross Unrealized Gross Unrealized Fair Value Current: Corporate debt securities $ 66,384 $ — $ (131 ) $ 66,253 Commercial paper 6,719 1 — 6,720 Certificates of deposit 2,500 — — 2,500 U.S. government and agency securities 2,000 — (1 ) 1,999 Non-current: Corporate debt securities (one to two year maturity) 27,018 2 (104 ) 26,916 U.S. government and agency securities 2,500 — (20 ) 2,480 $ 107,121 $ 3 $ (256 ) $ 106,868 At September 30, 2018 and December 31, 2017, the Company held 41 and 54 debt securities, respectively, that were in an unrealized loss position. The aggregate fair value of debt securities in an unrealized loss position at September 30, 2018 and December 31, 2017 was $81,222 and $96,623, respectively. As of September 30, 2018, 6 corporate debt securities with a fair value of $12,246 had been in a continuous unrealized loss position for more than 12 months. The unrealized losses of $35 related to these corporate debt securities are included in accumulated other comprehensive loss as of September 30, 2018. As of December 31, 2017, no securities had been in a continuous unrealized loss position for more than 12 months. The Company reviews 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 condensed consolidated statements of operations if the Company has experienced a credit loss and has the intent to sell the investment or if it is more likely than not that the Company will be required to sell the investment before recovery of the amortized cost basis. Evidence considered in this assessment includes reasons for the impairment, compliance with the Company’s investment policy, the severity and the duration of the impairment and changes in value subsequent to the end of the period. The unrealized losses at September 30, 2018 and December 31, 2017 are attributable to changes in interest rates and the Company does not believe any unrealized losses represent other-than-temporary impairments. |
Property and Equipment, net
Property and Equipment, net | 9 Months Ended |
Sep. 30, 2018 | |
Property, Plant and Equipment [Abstract] | |
Property and Equipment, net | 6. Property and Equipment, net Property and equipment, net consisted of the following (in thousands): Estimated Useful September 30, December 31, Laboratory equipment 4 $ 593 $ 593 Furniture and fixtures 5 601 381 Office and computer equipment 3 408 378 Construction in process N/A 491 — Leasehold improvements Lesser of useful life or lease term 3,920 3,391 6,013 4,743 Less accumulated depreciation and amortization (3,099 ) (2,558 ) $ 2,914 $ 2,185 |
Accrued Expenses
Accrued Expenses | 9 Months Ended |
Sep. 30, 2018 | |
Text Block [Abstract] | |
Accrued Expenses | 7. Accrued Expenses Accrued expenses consisted of the following (in thousands): September 30, December 31, Research and development costs $ 18,118 $ 16,198 Payroll and employee-related costs 7,349 3,982 Professional fees 3,443 972 Other 245 293 $ 29,155 $ 21,445 |
Net Loss Per Share
Net Loss Per Share | 9 Months Ended |
Sep. 30, 2018 | |
Earnings Per Share [Abstract] | |
Net Loss Per Share | 8. 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 during the period, without consideration for common stock equivalents. The Company’s potentially dilutive shares, which include outstanding stock options and 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: Three and Nine Months Ended 2018 2017 Outstanding stock options 8,962,643 7,049,007 Unvested restricted stock units 25,000 425,850 |
Stock-based Compensation
Stock-based Compensation | 9 Months Ended |
Sep. 30, 2018 | |
Disclosure of Compensation Related Costs, Share-based Payments [Abstract] | |
Stock-based Compensation | 9. Stock-based Compensation Stock Options A summary of the Company’s stock option activity and related information follows: Shares Weighted- Weighted- Aggregate Outstanding at December 31, 2017 7,019,083 $ 13.77 7.4 $ 11,897 Granted 3,232,350 $13.11 Exercised (458,989 ) $4.91 Canceled (829,801 ) $ 15.12 Outstanding at September 30, 2018 8,962,643 $ 13.86 7.5 $ 50,973 Exercisable at September 30, 2018 4,329,028 $ 15.52 6.0 $ 27,085 Total stock-based compensation expense related to stock options for the nine months ended September 30, 2018 and 2017 was $12,779 and $13,140, respectively. As of September 30, 2018, there was $36,766 of total unrecognized stock-based compensation expense related to stock options. The expense is expected to be recognized over a weighted-average period of 2.9 years. Restricted Stock Units A restricted stock unit (“RSU”) represents the right to receive one share of the Company’s common stock upon vesting of the RSU. The fair value of each RSU is based on the closing price of the Company’s common stock on the date of grant. During the year ended December 31, 2017, the Company granted RSUs with service conditions that vest provided that the employee remains employed with the Company (“Time-Based RSUs”). The following is a summary of Time-Based RSU activity under the 2013 Stock Incentive Plan for the nine months ended September 30, 2018: Number of Weighted- Unvested at December 31, 2017 30,000 $ 10.52 Granted — — Forfeited — — Vested (5,000 ) 10.27 Unvested at September 30, 2018 25,000 $ 10.57 The total stock-based compensation expense related to Time-Based RSUs for the nine months ended September 30, 2018 and 2017 was $118 and $2,243, respectively. As of September 30, 2018, there was $134 of unrecognized compensation costs related to unvested Time-Based RSUs, which are expected to be recognized over a weighted-average period of 1.0 years. Separately, and during the year ended December 31, 2017, the Company granted performance-based RSUs, which vest upon the achievement of certain performance goals subject to the employee’s continued employment (“Performance-Based RSUs”). During the nine months ended September 30, 2018, the Company recognized $180 of stock-based compensation expense related to a portion of the Performance-Based RSUs when the associated performance goal became probable of achievement in the first quarter and was achieved in the second quarter. The remaining 98,800 Performance-Based RSUs were forfeited in July 2018 when the performance goal was not achieved. Employee Stock Purchase Plan The Company has an Employee Stock Purchase Plan (“ESPP”) that permits eligible employees to enroll in six-month six-month For the nine months ended September 30, 2018 and 2017, the Company recorded stock-based compensation expense related to the ESPP of $301 and $152, respectively. As of September 30, 2018, 390,107 shares of the Company’s common stock remained available for issuance under the ESPP. As of September 30, 2018, there was $48 of total unrecognized stock-based compensation expense related to the ESPP. The expense is expected to be recognized over a period of one month. |
Commitments and Contingencies
Commitments and Contingencies | 9 Months Ended |
Sep. 30, 2018 | |
Commitments and Contingencies Disclosure [Abstract] | |
Commitments and Contingencies | 10. Commitments and Contingencies In 2014, the Company entered into an operating lease and subsequent amendment to lease approximately 46,167 square feet of office and research space in Newton, Massachusetts with a term through September 30, 2022. The lease provided the Company with an allowance for improvements of $1,616 which was incurred in the first quarter of 2015. In February 2018, the lease was amended to extend the term of the lease to September 30, 2025 and expand the leased premises to approximately 62,143 square feet. In June 2018, the lease was further amended to expand the premise to a total of approximately 98,502 square feet with no change in lease term. The 2018 lease amendments provided the Company with an allowance for improvements of $2,131, of which $1,014 was incurred during the nine months ended September 30, 2018. The Company evaluated the lease amendments and determined that the classification of the lease as an operating lease had not changed, and that the amendments did not constitute a new lease. As such, the unamortized balances of the existing deferred rent and tenant improvement allowances, along with the additions to deferred rent and tenant improvement allowances, will be amortized through September 30, 2025. All improvements were deemed normal tenant improvements, were recorded as leasehold improvements and deferred rent and will be recorded as a reduction to rent expense ratably over the lease term. The Company is recording rent expense on a straight-line basis through the end of the lease term, inclusive of the period in which there are no scheduled rent payments. The Company has recorded deferred rent on the condensed consolidated balance sheets at September 30, 2018 and December 31, 2017, accordingly. Finally, the Company has provided a security deposit in the form of a cash-collateralized letter of credit in the amount of $550. The amount is classified within non-current In November 2014, the Company signed a five-year operating lease agreement in Munich, Germany for approximately 3,681 square feet of office space. The lease is for the period February 2015 through January 2020. Pursuant to the lease agreement, the Company is obligated to make aggregate rent payments of €374 (approximately US$434) through January 31, 2020. The Company is recording rent expense on a straight-line basis through the end of the lease term, inclusive of the period in which there are no scheduled rent payments. The Company recorded rent expense totaling $895 and $296 for the three months ended September 30, 2018 and 2017, respectively, and $1,700 and $894 for the nine months ended September 30, 2018 and 2017, respectively. |
Equity
Equity | 9 Months Ended |
Sep. 30, 2018 | |
Equity [Abstract] | |
Equity | 11. Equity Underwritten Offerings On May 7, 2018, the Company completed a follow-on Form S-3 No. 333-222726) On April 28, 2017, the Company completed a follow-on Form S-3 No. 333-214489) Controlled Equity Offering Sales Agreement On December 7, 2015, the Company 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 the Company issued and sold through Cantor an aggregate of 9,172,159 shares of the Company’s common stock, for net proceeds of approximately $89,053. On August 2, 2018, the Company delivered written notice to Cantor terminating the Sales Agreement effective August 12, 2018. Under the Sales Agreement, Cantor sold shares of the Company’s common stock by methods deemed to be an “at-the-market” Open Market Sale Agreement On August 17, 2018, the Company entered into an Open Market Sale Agreement (the “Open Market Sale Agreement”) with Jefferies LLC, as agent (“Jefferies”), pursuant to which the Company may issue and sell shares of its common stock having an aggregate offering price of up to $75,000 (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. The Company may sell the Open Market Shares in amounts and at times to be determined by the Company from time to time subject to the terms and conditions of the Open Market Sale Agreement, but it has no obligation to sell any of the Open Market Shares in the Open Market Offering. The Company or Jefferies may suspend or terminate the offering of Open Market Shares upon notice to the other party and subject to other conditions. The Company has 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. The Company has also agreed to provide Jefferies with customary indemnification and contribution rights. The Company has not sold any shares to date under the Open Market Sale Agreement. |
Subsequent Event
Subsequent Event | 9 Months Ended |
Sep. 30, 2018 | |
Subsequent Events [Abstract] | |
Subsequent Event | 12. Subsequent Event On October 16, 2018 the Company completed an offering of $150,000 aggregate principal amount of the Company’s 3.00% convertible senior notes due 2025 (the “Notes”). In addition, on October 26, 2018, the Company issued an additional $22,500 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. The net proceeds from the sale of the Notes was approximately $166,900, after deducting the initial purchasers’ discounts and commissions and estimated offering expenses payable by the Company. The Notes are governed by the terms of an indenture (the “Indenture”) between the Company, as issuer, and Wilmington Trust, National Association, as trustee (the “Trustee”). The Notes are senior unsecured obligations of the Company 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. The Notes will mature on October 15, 2025, unless earlier converted, redeemed or repurchased in accordance with their terms. Prior to October 15, 2022, the Company may not redeem the Notes. On or after October 15, 2022, the Company may redeem for cash all or a portion of the Notes if the last reported sale price of the Company’s 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 the Company provides notice of redemption. The redemption price will be equal to the principal amount of the Notes to be redeemed, plus accrued and unpaid interest, if any, to, but excluding, the redemption date. No sinking fund is provided for the Notes. The Notes are convertible into shares of the Company’s common stock at an initial conversion rate of 63.0731 shares per $1 principal amount of Notes (equivalent to an initial conversion price of approximately $15.85 per share of common stock). The conversion rate will be subject to adjustment upon the occurrence of certain events as provided in the Indenture but will not be adjusted for any accrued and unpaid interest. Upon conversion of the Notes, the Company will pay or deliver, as the case may be, cash, shares of the Company’s common stock or a combination of cash and shares of common stock, at the Company’s election (subject to, and in accordance with, the settlement provisions of the Indenture). Holders of the Notes may convert their Notes at their option at any time prior to the close of business on the business day immediately preceding July 15, 2025 only upon the occurrence of certain circumstances. On or after July 15, 2025 until the close of business on the second scheduled trading day immediately preceding the maturity date of the Notes, holders may convert the Notes at any time. The conversion rate is subject to customary anti-dilution adjustments. In addition, if certain corporate events described in the Indenture occur prior to the maturity date, the Company will increase the conversion rate for a holder that elects to convert its Notes in connection with such corporate event in certain circumstances. Furthermore, calling any Note for redemption will result in an increase in the conversion rate applicable to the conversion of that Note, if it is converted in connection with the redemption in certain circumstances. If the Company undergoes a “fundamental change,” as defined in the Indenture, holders may require the Company to repurchase for cash all or any portion of their Notes at a fundamental change repurchase price equal to 100% of the principal amount of the Notes to be repurchased, plus any accrued and unpaid interest, if any, to, but excluding, the fundamental change repurchase date. The Indenture contains customary terms and covenants, including that upon certain events of default occurring and continuing, either the Trustee or the holders of at least 25% in principal amount of the outstanding Notes may declare the principal amount of the Notes to be immediately due and payable. |
Summary of Significant Accoun_2
Summary of Significant Accounting Policies (Policies) | 9 Months Ended |
Sep. 30, 2018 | |
Accounting Policies [Abstract] | |
Basis of Presentation | Basis of Presentation The accompanying unaudited condensed consolidated financial statements of Karyopharm Therapeutics Inc., a Delaware corporation (the “Company”), have been prepared in accordance with accounting principles generally accepted in the United States (“GAAP”) for interim financial reporting and as required by Regulation S-X, Rule 10-01. Form 10-K At September 30, 2018, the Company had $211,574 in cash, cash equivalents and short- and long-term investments. The Company has had recurring losses and incurred a loss of $120,197 for the nine months ended September 30, 2018. Net cash used in operations for the nine months ended September 30, 2018 was $110,945. The Company expects that cash, cash equivalents and short- and long-term investments at September 30, 2018, in addition to the approximately $166,900 of net proceeds the Company raised in the private placement of the Notes (see Note 12) in October 2018, will be sufficient to fund its current operating plans and capital expenditure requirements for at least twelve months from the date of issuance of the financial statements contained in this Form 10-Q |
Basis of Consolidation | Basis of Consolidation The condensed consolidated financial statements at September 30, 2018 include the accounts of (i) the Company, (ii) Karyopharm Securities Corp. (a wholly-owned Massachusetts corporation of the Company incorporated in December 2013), (iii) Karyopharm Europe GmbH (a wholly-owned German Limited Liability Company formed in August 2014), (iv) Karyopharm Therapeutics (Bermuda) Ltd. (a wholly-owned Bermuda subsidiary of the Company formed in March 2015), and (v) Karyopharm Israel Ltd. (a wholly-owned Israeli subsidiary of the Company formed in June 2018). All intercompany balances and transactions have been eliminated in consolidation. The significant accounting policies used in preparation of these condensed consolidated financial statements on Form 10-Q Form 10-K |
Revenue Recognition | Revenue Recognition The Company adopted Accounting Standards Update (“ASU”) 2014-09, Revenue from Contracts with Customers ( Revenue Recognition 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, an entity recognizes revenue when its customer obtains control of promised goods or services, in an amount that reflects the consideration which the entity expects to receive in exchange for those goods or services. To determine revenue recognition for arrangements that an entity determines are within the scope of ASC 606, the entity performs the following five steps: (i) identify the contract(s) with a customer; (ii) identify the performance obligations in the contract; (iii) determine the transaction price; (iv) allocate the transaction price to the performance obligations in the contract; and (v) recognize revenue when (or as) the entity satisfies a performance obligation. At contract inception, once the contract is determined to be within the scope of ASC 606, the Company assesses the goods or services promised within each contract and determines those that are performance obligations and assesses whether each promised good or service is distinct. The Company then recognizes as revenue the amount of the transaction price that is allocated to the respective performance obligation when (or as) the performance obligation is satisfied. The Company generates revenue from license or similar agreements with pharmaceutical companies for the development and commercialization of certain of its 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 the Company’s intellectual property is determined to be distinct from the other performance obligations identified in the arrangement, the Company recognizes the transaction price allocated to the license as revenue upon transfer of control of the license. The Company evaluates 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 the Company 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. The Company utilizes judgment to determine the transaction price. In connection therewith, the Company evaluates 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 the control of the Company, 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 the Company recognizes revenue as or when the performance obligations under the contract are satisfied. At the end of each reporting period, the Company re-evaluates catch-up The Company then determines 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 is deemed to be the predominant item to which the royalties relate, the Company recognizes 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). |
Recent Accounting Pronouncements | Recently Adopted Accounting Standards As detailed above, the Company adopted ASC 606 on January 1, 2018. Under the modified retrospective transition method, the Company applied ASC 606 to all contracts within scope as of January 1, 2018. Under the practical expedient concerning contract modifications contained in the transitional provisions of ASC 606, the Company has not retrospectively restated its contracts for modifications prior to the earliest period presented, and instead has reflected the aggregate effect of all modifications when identifying the satisfied and unsatisfied performance obligations, determining the transaction price and allocating the transaction price. Qualitatively, the effect of applying this practical expedient is not material to the periods presented in the consolidated financial statements. As more fully discussed in Note 3, Asset Purchase and License Agreements, only the Company’s arrangement with Ono Pharmaceutical Co., Ltd. was determined to have unsatisfied performance obligations as of the adoption date. However, the pattern of revenue recognition was not affected and, therefore, no transition adjustment was recorded to the opening balance of accumulated deficit on January 1, 2018. All other agreements subject to transition, which only included the Company’s arrangement with Anivive Lifesciences Inc., were unaffected by the adoption of ASC 606 in all periods presented in the consolidated financial statements through application of the modified retrospective transition method. In August 2016, the Financial Accounting Standards Boards (“FASB”) issued ASU 2016-15, Classification of Certain Cash Receipts and Cash Payments (“ASU 2016-15”). ASU 2016-05 In October 2016, the FASB issued ASU No. 2016-16, Accounting for Income Taxes: Intra-Entity Asset Transfers of Assets Other than Inventory (Topic 740) In November 2016, the FASB issued ASU No. 2016-18, Statement of Cash Flows (Topic 230): Restricted Cash beginning-of-period end-of-period In May 2017, the FASB issued ASU 2017-09, Compensation-Stock Compensation (Topic 718) (“ASU 2017-09”) Scope of Modification Accounting ASU 2017-09 non-substantive. On December 22, 2017, the Tax Cuts and Jobs Act (“TCJA”) was enacted and led to significant changes to U.S. tax law. Also on December 22, 2017, the SEC staff issued Staff Accounting Bulletin No. 118 (“SAB 118”), allowing companies to record the effects of the TCJA on a provisional basis during a measurement period not to extend beyond one year of the enactment date. SAB 118 was codified into ASC 740 by ASU 2018-05. Recently Issued Accounting Standards On August 17, 2018, the SEC issued an amendment to Rule 3-04 Regulation S-X, year-to-date Form 10-Q. In June 2018, the FASB issued ASU No. 2018-07, Compensation-Stock Compensation (Topic 718): Improvements to Nonemployee Share-Based Payment Accounting (“ASU 2018-07”). ASU 2018-07 In February 2016, the FASB issued ASU No. 2016-02, Leases (Topic 842) ASU 2018-11, Leases (Topic 842) Targeted Improvements Pursuant to the guidance under ASU No. 2016-02, non-lease In July 2018, the FASB issued ASU No. 2018-09, No. 2018-09 No. 2018-09 In August 2018, the FASB issued ASU No. 2018-13, No. 2018-13 No. 2018-13 No. 2018-13 In August 2018, the FASB issued ASU No. 2018-15, Intangible-Goodwill and Other Internal-Use 350-40) No. 2018-15 No. 2018-15 |
Fair Value of Financial Instr_2
Fair Value of Financial Instruments (Tables) | 9 Months Ended |
Sep. 30, 2018 | |
Fair Value Disclosures [Abstract] | |
Schedule of Financial Assets That Have Been Measured at Fair Value | The following table presents information about the Company’s financial assets that have been measured at fair value at September 30, 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 $ 70,876 $ 70,876 $ — $ — Investments: Current: Corporate debt securities 87,205 — 87,205 — Commercial paper 8,988 — 8,988 — U.S. government and agency securities 2,477 — 2,477 — Certificates of deposit 6,500 — 6,500 — Non-current: Corporate debt securities (one to two year maturity) 4,804 — 4,804 — $ 180,850 $ 70,876 $ 109,974 $ — The following table presents information about the Company’s financial assets that have been measured at fair value at December 31, 2017 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 $ 41,805 $ 41,805 $ — $ — Investments: Current: Corporate debt securities 66,253 — 66,253 — Commercial paper 6,720 — 6,720 — Certificates of deposit 2,500 — 2,500 — U.S. government and agency securities 1,999 — 1,999 — Non-current: — Corporate debt securities (one to two year maturity) 26,916 — 26,916 — U.S. government securities and agency securities 2,480 — 2,480 — $ 148,673 $ 41,805 $ 106,868 $ — |
Investments (Tables)
Investments (Tables) | 9 Months Ended |
Sep. 30, 2018 | |
Investments, Debt and Equity Securities [Abstract] | |
Summary of Investments, Classified as Available-for-Sale | The following table summarizes the Company’s investments in debt securities, classified as available-for-sale, Amortized Cost Gross Unrealized Gross Unrealized Fair Value Current: Corporate debt securities $ 87,317 $ 6 $ (118 ) $ 87,205 Commercial paper 8,988 — — 8,988 U.S. government and agency securities 2,500 — (23 ) 2,477 Certificates of deposit 6,500 — — 6,500 Non-current: Corporate debt securities (one to two year maturity) 4,817 — (13 ) 4,804 $ 110,122 $ 6 $ (154 ) $ 109,974 The following table summarizes the Company’s investments in debt securities, classified as available-for-sale, Amortized Cost Gross Unrealized Gross Unrealized Fair Value Current: Corporate debt securities $ 66,384 $ — $ (131 ) $ 66,253 Commercial paper 6,719 1 — 6,720 Certificates of deposit 2,500 — — 2,500 U.S. government and agency securities 2,000 — (1 ) 1,999 Non-current: Corporate debt securities (one to two year maturity) 27,018 2 (104 ) 26,916 U.S. government and agency securities 2,500 — (20 ) 2,480 $ 107,121 $ 3 $ (256 ) $ 106,868 |
Property and Equipment, net (Ta
Property and Equipment, net (Tables) | 9 Months Ended |
Sep. 30, 2018 | |
Property, Plant and Equipment [Abstract] | |
Schedule of Property and Equipment, Net | Property and equipment, net consisted of the following (in thousands): Estimated Useful September 30, December 31, Laboratory equipment 4 $ 593 $ 593 Furniture and fixtures 5 601 381 Office and computer equipment 3 408 378 Construction in process N/A 491 — Leasehold improvements Lesser of useful life or lease term 3,920 3,391 6,013 4,743 Less accumulated depreciation and amortization (3,099 ) (2,558 ) $ 2,914 $ 2,185 |
Accrued Expenses (Tables)
Accrued Expenses (Tables) | 9 Months Ended |
Sep. 30, 2018 | |
Text Block [Abstract] | |
Schedule of Accrued Expenses | Accrued expenses consisted of the following (in thousands): September 30, December 31, Research and development costs $ 18,118 $ 16,198 Payroll and employee-related costs 7,349 3,982 Professional fees 3,443 972 Other 245 293 $ 29,155 $ 21,445 |
Net Loss Per Share (Tables)
Net Loss Per Share (Tables) | 9 Months Ended |
Sep. 30, 2018 | |
Earnings Per Share [Abstract] | |
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: Three and Nine Months Ended 2018 2017 Outstanding stock options 8,962,643 7,049,007 Unvested restricted stock units 25,000 425,850 |
Stock-based Compensation (Table
Stock-based Compensation (Tables) | 9 Months Ended |
Sep. 30, 2018 | |
Disclosure of Compensation Related Costs, Share-based Payments [Abstract] | |
Summary of Stock Option Activity for Employees and Nonemployees | A summary of the Company’s stock option activity and related information follows: Shares Weighted- Weighted- Aggregate Outstanding at December 31, 2017 7,019,083 $ 13.77 7.4 $ 11,897 Granted 3,232,350 $13.11 Exercised (458,989 ) $4.91 Canceled (829,801 ) $ 15.12 Outstanding at September 30, 2018 8,962,643 $ 13.86 7.5 $ 50,973 Exercisable at September 30, 2018 4,329,028 $ 15.52 6.0 $ 27,085 |
Summary of RSU Activity | The following is a summary of Time-Based RSU activity under the 2013 Stock Incentive Plan for the nine months ended September 30, 2018: Number of Weighted- Unvested at December 31, 2017 30,000 $ 10.52 Granted — — Forfeited — — Vested (5,000 ) 10.27 Unvested at September 30, 2018 25,000 $ 10.57 |
Summary of Significant Accoun_3
Summary of Significant Accounting Policies - Additional Information (Detail) - USD ($) $ in Thousands | 1 Months Ended | 3 Months Ended | 9 Months Ended | ||
Oct. 31, 2018 | Sep. 30, 2018 | Sep. 30, 2017 | Sep. 30, 2018 | Sep. 30, 2017 | |
Accounting Policies and General Information [Line Items] | |||||
Cash, cash equivalents and short and long-term investments | $ 211,574 | $ 211,574 | |||
Net loss | $ 48,083 | $ 30,640 | 120,197 | $ 89,944 | |
Net cash used in operations | $ 110,945 | $ 68,179 | |||
Subsequent Event [Member] | |||||
Accounting Policies and General Information [Line Items] | |||||
Net proceeds after deducting underwriting discounts, commissions and offering expenses | $ 166,900 |
Recent Accounting Pronounceme_2
Recent Accounting Pronouncements - Additional Information (Detail) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2017 | Sep. 30, 2018 | Sep. 30, 2017 | |
New Accounting Pronouncements or Change in Accounting Principle [Line Items] | |||
Reduction in deferred tax asset | $ 42,763 | ||
ASU 2016-18 [Member] | |||
New Accounting Pronouncements or Change in Accounting Principle [Line Items] | |||
Restricted cash and cash equivalents | $ 490 | $ 479 |
License and Asset Purchase Ag_2
License and Asset Purchase Agreements - Additional Information - Antengene License Agreement (Detail) - Antengene Therapeutics Limited [Member] - USD ($) | May 23, 2018 | Sep. 30, 2018 |
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||
Revenues | $ 0 | |
Deferred Revenue, current | 9,363,000 | |
Deferred Revenue, non-current | $ 2,340,000 | |
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 | 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) the Company in the event Antengene challenges or assists with a challenge to certain of the Company’s patent rights. | |
Government Research Grant Agreement [Member] | Development Goals [Member] | Maximum [Member] | ||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||
Milestone payments receivable | $ 105,000,000 | |
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 | 45,000,000 | |
Commercial milestone payments receivable | 45,000,000 | |
Government Research Grant Agreement [Member] | Development Milestone [Member] | Maximum [Member] | Scenario, Plan [Member] | ||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||
Milestone payments receivable | 105,000,000 | |
Selinexor [Member] | Government Research Grant Agreement [Member] | ||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||
Performance obligations | $ 9,363,000 | |
Eltanexor [Member] | Government Research Grant Agreement [Member] | ||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||
Performance obligations | 1,053,000 | |
KPT-9274 [Member] | Government Research Grant Agreement [Member] | ||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||
Performance obligations | 1,053,000 | |
Verdinexor [Member] | Government Research Grant Agreement [Member] | ||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||
Performance obligations | $ 234,000 | |
Up-front Payment Arrangement [Member] | Government Research Grant Agreement [Member] | ||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||
Upfront payments | $ 11,703,000 |
License and Asset Purchase Ag_3
License and Asset Purchase Agreements - Additional Information - Biogen Asset Purchase Agreement (Detail) - Biogen MA Inc [Member] - USD ($) $ in Thousands | Jan. 24, 2018 | Sep. 30, 2018 |
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||
Revenues | $ 10,000 | |
Asset Purchase Agreement [Member] | ||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||
Upfront payment received | $ 10,000 | |
Commercial milestone payments receivable | 65,000 | |
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 | $ 142,000 |
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 ($) | Oct. 11, 2017JPY (¥) | Jun. 30, 2018USD ($) | Sep. 30, 2018USD ($) | Sep. 30, 2018JPY (¥) | Oct. 11, 2017JPY (¥) |
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||
Revenues | $ 19,724,000 | |||||
Deferred Revenue, non-current | $ 2,192,000 | |||||
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) the Company in the event Ono challenges or assists with a challenge to certain of the Company’s patent rights | |||||
Commercial milestone payments receivable | ¥ | ¥ 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] | Clinical Development and Regulatory Milestone [Member] | Maximum [Member] | ||||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||
Milestone payments receivable | ¥ | ¥ 10,150,000,000 | |||||
Selinexor [Member] | Government Research Grant Agreement [Member] | ||||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||
Performance obligations | $ 19,724,000 | |||||
Eltanexor [Member] | Government Research Grant Agreement [Member] | ||||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||
Performance obligations | $ 2,192,000 | |||||
Up-front Payment Arrangement [Member] | Government Research Grant Agreement [Member] | ||||||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||||||
Upfront payments | $ 21,916,000 | ¥ 2,500,000,000 |
License and Asset Purchase Ag_5
License and Asset Purchase Agreements - Additional Information - MMRF Research Agreement (Detail) - Research Agreement [Member] - Multiple Myeloma Research Foundation [Member] - USD ($) | Sep. 30, 2018 | Dec. 31, 2017 |
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||
Estimated upfront cash payment obligation | $ 1,972,000 | |
Maximum estimated payment obligation | $ 3,750,000 | |
Expensed cash payment obligation | $ 278,000 |
License and Asset Purchase Ag_6
License and Asset Purchase Agreements - Additional Information - Anivive License Agreement (Detail) - Government Research Grant Agreement [Member] - Anivive Lifesciences, Inc. [Member] - USD ($) | Apr. 28, 2017 | Sep. 30, 2018 |
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||
Performance obligations | $ 1,250,000 | |
Maximum [Member] | ||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||
Milestone payments receivable | $ 43,250,000 | |
Technology Transfer [Member] | ||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||
Milestone payments receivable | 250,000 | |
Clinical Development and Regulatory Milestone [Member] | ||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||
Milestone payments receivable | 5,750,000 | |
Sales Milestone Events [Member] | ||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||
Milestone payments receivable | 37,500,000 | |
Up-front Payment Arrangement [Member] | ||
Research and Development Arrangement, Contract to Perform for Others [Line Items] | ||
Upfront payments | $ 1,000,000 | $ 1,000,000 |
Fair Value of Financial Instr_3
Fair Value of Financial Instruments - Schedule of Financial Assets That Have Been Measured at Fair Value (Detail) - USD ($) $ in Thousands | Sep. 30, 2018 | Dec. 31, 2017 |
Financial assets | ||
Investments | $ 109,974 | $ 106,868 |
Total | 180,850 | 148,673 |
Current [Member] | Corporate Debt Securities [Member] | ||
Financial assets | ||
Investments | 87,205 | 66,253 |
Current [Member] | Commercial Paper [Member] | ||
Financial assets | ||
Investments | 8,988 | 6,720 |
Current [Member] | US Government and Agency Securities [Member] | ||
Financial assets | ||
Investments | 2,477 | 1,999 |
Non-current [Member] | Corporate Debt Securities [Member] | ||
Financial assets | ||
Investments | 4,804 | 26,916 |
Non-current [Member] | US Government and Agency Securities [Member] | ||
Financial assets | ||
Investments | 2,480 | |
Money Market Funds [Member] | ||
Financial assets | ||
Cash equivalents | 70,876 | 41,805 |
Certificates of Deposit [Member] | Current [Member] | ||
Financial assets | ||
Investments | 6,500 | 2,500 |
Level 1 [Member] | ||
Financial assets | ||
Total | 70,876 | 41,805 |
Level 1 [Member] | Money Market Funds [Member] | ||
Financial assets | ||
Cash equivalents | 70,876 | 41,805 |
Level 2 [Member] | ||
Financial assets | ||
Total | 109,974 | 106,868 |
Level 2 [Member] | Current [Member] | Corporate Debt Securities [Member] | ||
Financial assets | ||
Investments | 87,205 | 66,253 |
Level 2 [Member] | Current [Member] | Commercial Paper [Member] | ||
Financial assets | ||
Investments | 8,988 | 6,720 |
Level 2 [Member] | Current [Member] | US Government and Agency Securities [Member] | ||
Financial assets | ||
Investments | 2,477 | 1,999 |
Level 2 [Member] | Non-current [Member] | Corporate Debt Securities [Member] | ||
Financial assets | ||
Investments | 4,804 | 26,916 |
Level 2 [Member] | Non-current [Member] | US Government and Agency Securities [Member] | ||
Financial assets | ||
Investments | 2,480 | |
Level 2 [Member] | Certificates of Deposit [Member] | Current [Member] | ||
Financial assets | ||
Investments | $ 6,500 | $ 2,500 |
Investments - Summary of Invest
Investments - Summary of Investments, Classified as Available-for-Sale (Detail) - USD ($) $ in Thousands | Sep. 30, 2018 | Dec. 31, 2017 |
Debt Securities, Available-for-sale [Line Items] | ||
Amortized Cost | $ 110,122 | $ 107,121 |
Gross Unrealized Gains | 6 | 3 |
Gross Unrealized Loss | (154) | (256) |
Fair Value | 109,974 | 106,868 |
Non-current [Member] | Corporate Debt Securities [Member] | ||
Debt Securities, Available-for-sale [Line Items] | ||
Amortized Cost | 4,817 | 27,018 |
Gross Unrealized Gains | 2 | |
Gross Unrealized Loss | (13) | (104) |
Fair Value | 4,804 | 26,916 |
Non-current [Member] | US Government and Agency Securities [Member] | ||
Debt Securities, Available-for-sale [Line Items] | ||
Amortized Cost | 2,500 | |
Gross Unrealized Loss | (20) | |
Fair Value | 2,480 | |
Current [Member] | Certificates of Deposit [Member] | ||
Debt Securities, Available-for-sale [Line Items] | ||
Amortized Cost | 6,500 | 2,500 |
Fair Value | 6,500 | 2,500 |
Current [Member] | Corporate Debt Securities [Member] | ||
Debt Securities, Available-for-sale [Line Items] | ||
Amortized Cost | 87,317 | 66,384 |
Gross Unrealized Gains | 6 | |
Gross Unrealized Loss | (118) | (131) |
Fair Value | 87,205 | 66,253 |
Current [Member] | Commercial Paper [Member] | ||
Debt Securities, Available-for-sale [Line Items] | ||
Amortized Cost | 8,988 | 6,719 |
Gross Unrealized Gains | 1 | |
Fair Value | 8,988 | 6,720 |
Current [Member] | US Government and Agency Securities [Member] | ||
Debt Securities, Available-for-sale [Line Items] | ||
Amortized Cost | 2,500 | 2,000 |
Gross Unrealized Loss | (23) | (1) |
Fair Value | $ 2,477 | $ 1,999 |
Investments - Summary of Inve_2
Investments - Summary of Investments, Classified as Available-for-Sale (Parenthetical) (Detail) - Corporate Debt Securities [Member] - Non-current [Member] | 9 Months Ended | 12 Months Ended |
Sep. 30, 2018 | Dec. 31, 2017 | |
Minimum [Member] | ||
Debt Securities, Available-for-sale [Line Items] | ||
Investments, maturity period | 1 year | 1 year |
Maximum [Member] | ||
Debt Securities, Available-for-sale [Line Items] | ||
Investments, maturity period | 2 years | 2 years |
Investments - Additional Inform
Investments - Additional Information (Detail) | Sep. 30, 2018USD ($)Securities | Dec. 31, 2017USD ($)Securities |
Debt Securities, Available-for-sale [Line Items] | ||
Number of debt securities with unrealized loss position for less than one year | Securities | 41 | 54 |
Aggregate fair value of debt securities with unrealized loss position for less than one year | $ 81,222,000 | $ 96,623,000 |
Unrealized losses , included in accumulated other comprehensive loss | 154,000 | 256,000 |
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 | 6 | 0 |
Aggregate fair value of corporate debt securities with continuous unrealized loss position for more than 12 months | $ 12,246,000 | $ 0 |
Unrealized losses , included in accumulated other comprehensive loss | $ 35,000 |
Property and Equipment, net - S
Property and Equipment, net - Schedule of Property and Equipment, Net (Detail) - USD ($) $ in Thousands | 9 Months Ended | |
Sep. 30, 2018 | Dec. 31, 2017 | |
Property, Plant and Equipment [Line Items] | ||
Property and equipment, gross | $ 6,013 | $ 4,743 |
Less accumulated depreciation and amortization | (3,099) | (2,558) |
Property and equipment, net | $ 2,914 | 2,185 |
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 | $ 601 | 381 |
Office and Computer Equipment [Member] | ||
Property, Plant and Equipment [Line Items] | ||
Estimated useful life | 3 years | |
Property and equipment, gross | $ 408 | 378 |
Construction in Progress [Member] | ||
Property, Plant and Equipment [Line Items] | ||
Property and equipment, gross | $ 491 | |
Leasehold Improvements [Member] | ||
Property, Plant and Equipment [Line Items] | ||
Estimated useful life | Lesser of useful life or lease term | |
Property and equipment, gross | $ 3,920 | $ 3,391 |
Accrued Expenses - Schedule of
Accrued Expenses - Schedule of Accrued Expenses (Detail) - USD ($) $ in Thousands | Sep. 30, 2018 | Dec. 31, 2017 |
Accrued Liabilities, Current [Abstract] | ||
Research and development costs | $ 18,118 | $ 16,198 |
Payroll and employee-related costs | 7,349 | 3,982 |
Professional fees | 3,443 | 972 |
Other | 245 | 293 |
Total Accrued Expenses | $ 29,155 | $ 21,445 |
Net Loss Per Share - Schedule o
Net Loss Per Share - Schedule of Potentially Dilutive Securities Were Excluded From The Calculation of Diluted Net Loss Per Share Due to Their Anti-Dilutive Effect (Detail) - shares | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2018 | Sep. 30, 2017 | Sep. 30, 2018 | Sep. 30, 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) | 8,962,643 | 7,049,007 | 8,962,643 | 7,049,007 |
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) | 25,000 | 425,850 | 25,000 | 425,850 |
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 | 9 Months Ended | 12 Months Ended |
Sep. 30, 2018 | Dec. 31, 2017 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Shares, Outstanding, Beginning balance | 7,019,083 | |
Shares, Granted | 3,232,350 | |
Shares, Exercised | (458,989) | |
Shares, Canceled | (829,801) | |
Shares, Outstanding, Ending balance | 8,962,643 | 7,019,083 |
Shares, Exercisable | 4,329,028 | |
Weighted-Average Exercise Price Per Share, Outstanding, Beginning balance | $ 13.77 | |
Weighted-Average Exercise Price Per Share, Granted | 13.11 | |
Weighted-Average Exercise Price Per Share, Exercised | 4.91 | |
Weighted-Average Exercise Price Per Share, Canceled | 15.12 | |
Weighted-Average Exercise Price Per Share, Outstanding, Ending balance | 13.86 | $ 13.77 |
Weighted-Average Exercise Price Per Share, Exercisable | $ 15.52 | |
Weighted-Average Remaining Contractual Term (years), Outstanding | 7 years 6 months | 7 years 4 months 24 days |
Weighted-Average Remaining Contractual Term (years), Exercisable | 6 years | |
Aggregate Intrinsic Value, Outstanding | $ 50,973 | $ 11,897 |
Aggregate Intrinsic Value, Exercisable | $ 27,085 |
Stock-based Compensation - Addi
Stock-based Compensation - Additional Information (Detail) $ in Thousands | 1 Months Ended | 9 Months Ended | |
Jul. 31, 2018shares | Sep. 30, 2018USD ($)shares | Sep. 30, 2017USD ($)Right | |
Stock Option [Member] | |||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Stock-based compensation expense | $ 12,779 | $ 13,140 | |
Total unrecognized stock-based compensation expense | $ 36,766 | ||
Period for recognition of unrecognized expense | 2 years 10 months 24 days | ||
Unvested Restricted Stock Units [Member] | |||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Stock-based compensation expense | $ 118 | $ 2,243 | |
Period for recognition of unrecognized expense | 1 year | ||
Right to received shares of common stock (shares) | Right | 1 | ||
Total unrecognized stock-based compensation expense | $ 134 | ||
Number of Performance-Based RSUs forfeited | shares | 0 | ||
Performance-Based RSUs [Member] | |||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Stock-based compensation expense | $ 180 | ||
Number of Performance-Based RSUs forfeited | shares | 98,800 | ||
ESPP [Member] | |||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Stock-based compensation expense | $ 301 | $ 152 | |
Period for recognition of unrecognized expense | 1 month | ||
Total unrecognized stock-based compensation expense | $ 48 | ||
Offering period | 6 months | ||
Purchase price of common stock | 85.00% | ||
Number of shares of common stock authorized | shares | 242,424 | ||
Common stock shares available for issuance under ESPP | shares | 390,107 | ||
Percentage of shares of common stock available for issuance | 1.00% | ||
Employee stock purchase plan, description | In 2013, the Company’s stockholders approved the reservation of 242,424 shares of the Company’s common stock for issuance under the ESPP, 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 the Company’s common stock, 1% of the number of outstanding shares on such date, or an amount determined by the board of directors. | ||
ESPP [Member] | Minimum [Member] | |||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Common stock shares available for issuance under ESPP | shares | 484,848 |
Stock-based Compensation - Su_2
Stock-based Compensation - Summary of RSU Activity (Detail) - Unvested Restricted Stock Units [Member] | 9 Months Ended |
Sep. 30, 2018$ / sharesshares | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |
Number of Shares Underlying RSUs, Unvested beginning balance | shares | 30,000 |
Number of Shares Underlying RSUs, Granted | shares | 0 |
Number of Shares Underlying RSUs, Forfeited | shares | 0 |
Number of Shares Underlying RSUs, Vested | shares | (5,000) |
Number of Shares Underlying RSUs, Unvested ending balance | shares | 25,000 |
Weighted-Average Grant Date Fair Value, Unvested beginning balance | $ / shares | $ 10.52 |
Weighted-Average Grant Date Fair Value, Granted | $ / shares | 0 |
Weighted-Average Grant Date Fair Value, Forfeited | $ / shares | 0 |
Weighted-Average Grant Date Fair Value, Vested | $ / shares | 10.27 |
Weighted-Average Grant Date Fair Value, Unvested ending balance | $ / shares | $ 10.57 |
Commitments and Contingencies -
Commitments and Contingencies - Additional Information (Detail) € in Thousands, $ in Thousands | 3 Months Ended | 9 Months Ended | ||||||||
Sep. 30, 2018USD ($)ft² | Sep. 30, 2017USD ($) | Sep. 30, 2018USD ($)ft² | Sep. 30, 2017USD ($) | Dec. 31, 2018USD ($) | Feb. 28, 2018ft² | Mar. 31, 2015USD ($) | Dec. 31, 2014ft² | Nov. 30, 2014EUR (€)ft² | Nov. 30, 2014USD ($)ft² | |
Summary Of Commitments And Contingent Liabilities [Line Items] | ||||||||||
Total rent expense | $ 895 | $ 296 | $ 1,700 | $ 894 | ||||||
Office Space Lease [Member] | ||||||||||
Summary Of Commitments And Contingent Liabilities [Line Items] | ||||||||||
Office and laboratory space leased | ft² | 3,681 | 3,681 | ||||||||
Operating lease agreement term | 5 years | 5 years | ||||||||
Scheduled rent payments due | € 374 | $ 434 | ||||||||
Office and Research Space Lease [Member] | ||||||||||
Summary Of Commitments And Contingent Liabilities [Line Items] | ||||||||||
Office and laboratory space leased | ft² | 98,502 | 98,502 | 62,143 | 46,167 | ||||||
Allowance for improvements | $ 1,014 | $ 1,014 | $ 1,616 | |||||||
Office and Research Space Lease [Member] | Scenario, Forecast [Member] | ||||||||||
Summary Of Commitments And Contingent Liabilities [Line Items] | ||||||||||
Allowance for improvements | $ 2,131 | |||||||||
Security deposit in the form of a letter of credit | $ 550 |
Equity - Controlled Equity Offe
Equity - Controlled Equity Offering Sales Agreement - Additional Information (Detail) - USD ($) $ / shares in Units, $ in Thousands | Aug. 02, 2018 | May 07, 2018 | Apr. 28, 2017 | Dec. 07, 2015 | Sep. 30, 2018 | Aug. 17, 2018 |
Common Stock [Member] | ||||||
Equity Offering [Line Items] | ||||||
Issuance of common stock, net of issuance costs | 10,525,424 | 3,902,439 | ||||
Public offering price of common shares | $ 14.75 | $ 10.25 | ||||
Net proceeds after deducting underwriting discounts, commissions and offering expenses | $ 145,720 | $ 37,900 | ||||
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 | |||||
Net proceeds from sale of common stock | $ 89,053 | |||||
Sales agreement term date | Aug. 12, 2018 | |||||
Maximum [Member] | Jefferies LLC [Member] | Open Market Sale Agreement [Member] | ||||||
Equity Offering [Line Items] | ||||||
Percentage of commission of gross proceeds from the sale of Shares | 3.00% | |||||
Aggregate offering price | $ 75,000 | |||||
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% |
Subsequent Event - Additional I
Subsequent Event - Additional Information (Detail) | Oct. 15, 2022d | Oct. 16, 2018USD ($)$ / sharesshares | Oct. 31, 2018USD ($) | Oct. 16, 2018USD ($)$ / shares | Oct. 26, 2018USD ($) |
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 | ||||
Notes instrument, consecutive trading days | d | 30 | ||||
Subsequent Event [Member] | |||||
Subsequent Event [Line Items] | |||||
Net proceeds after deducting underwriting discounts, commissions and offering expenses | $ 166,900,000 | ||||
Subsequent Event [Member] | 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 | $ 1,000 | ||||
Notes, conversion price per share | $ / shares | $ 15.85 | $ 15.85 | |||
Subsequent Event [Member] | 3% Convertible Senior Notes Due 2025 [Member] | |||||
Subsequent Event [Line Items] | |||||
Aggregate principal amount | $ 150,000,000 | $ 150,000,000 | $ 22,500,000 | ||
Net proceeds after deducting underwriting discounts, commissions and offering expenses | $ 166,900,000 | ||||
Notes, interest rate | 3.00% | ||||
Notes, maturity date | Oct. 15, 2025 | ||||
Notes, repurchase price | 100.00% | ||||
Subsequent Event [Member] | 3% Convertible Senior Notes Due 2025 [Member] | Minimum [Member] | |||||
Subsequent Event [Line Items] | |||||
Percentage of principal amount of outstanding notes to be immediately due and payable | 25.00% |