Document and Entity Information
Document and Entity Information - shares | 6 Months Ended | |
Jun. 30, 2019 | Aug. 02, 2019 | |
Document And Entity Information [Abstract] | ||
Document Type | 10-Q | |
Amendment Flag | false | |
Document Period End Date | Jun. 30, 2019 | |
Document Fiscal Year Focus | 2019 | |
Document Fiscal Period Focus | Q2 | |
Trading Symbol | RGNX | |
Entity Registrant Name | REGENXBIO Inc. | |
Entity Central Index Key | 0001590877 | |
Current Fiscal Year End Date | --12-31 | |
Entity Filer Category | Large Accelerated Filer | |
Entity Small Business | false | |
Entity Emerging Growth Company | false | |
Entity Current Reporting Status | Yes | |
Entity Shell Company | false | |
Entity File Number | 001-37553 | |
Entity Tax Identification Number | 47-1851754 | |
Entity Address, Address Line One | 9600 Blackwell Road | |
Entity Address, Address Line Two | Suite 210 | |
Entity Address, City or Town | Rockville | |
Entity Address, State or Province | MD | |
Entity Address, Postal Zip Code | 20850 | |
City Area Code | 240 | |
Local Phone Number | 552-8181 | |
Entity Common Stock, Shares Outstanding | 36,806,632 | |
Document Transition Report | false | |
Document Quarterly Report | true | |
Entity Interactive Data Current | Yes | |
Security Exchange Name | NASDAQ | |
Title of 12(b) Security | Common Stock, $0.0001 par value per share | |
Entity Incorporation, State or Country Code | DE |
Consolidated Balance Sheets
Consolidated Balance Sheets - USD ($) $ in Thousands | Jun. 30, 2019 | Dec. 31, 2018 |
Current assets | ||
Cash and cash equivalents | $ 55,142 | $ 75,561 |
Marketable securities | 286,354 | 244,200 |
Accounts receivable | 9,679 | 8,587 |
Prepaid expenses | 6,036 | 5,734 |
Other current assets | 2,281 | 3,831 |
Total current assets | 359,492 | 337,913 |
Marketable securities | 108,194 | 150,819 |
Accounts receivable | 23,955 | 23,012 |
Property and equipment, net | 27,330 | 28,702 |
Operating lease right-of-use assets | 5,904 | |
Restricted cash | 1,053 | 1,053 |
Other assets | 3,211 | 2,315 |
Total assets | 529,139 | 543,814 |
Current liabilities | ||
Accounts payable | 5,851 | 4,412 |
Accrued expenses and other current liabilities | 16,534 | 17,164 |
Deferred revenue | 600 | |
Operating lease liabilities | 2,276 | |
Total current liabilities | 24,661 | 22,176 |
Deferred revenue | 3,333 | 3,333 |
Operating lease liabilities | 4,654 | |
Deferred rent | 1,098 | |
Financing lease obligations | 5,854 | |
Other liabilities | 1,899 | 2,505 |
Total liabilities | 34,547 | 34,966 |
Commitments and contingencies (Note 7) | ||
Stockholders’ equity | ||
Preferred stock; $0.0001 par value; 10,000 shares authorized, and no shares issued and outstanding at June 30, 2019 and December 31, 2018 | ||
Common stock; $0.0001 par value; 100,000 shares authorized at June 30, 2019 and December 31, 2018; 36,752 and 36,120 shares issued and outstanding at June 30, 2019 and December 31, 2018, respectively | 4 | 4 |
Additional paid-in capital | 610,891 | 592,580 |
Accumulated other comprehensive income (loss) | 471 | (720) |
Accumulated deficit | (116,774) | (83,016) |
Total stockholders’ equity | 494,592 | 508,848 |
Total liabilities and stockholders’ equity | $ 529,139 | $ 543,814 |
Consolidated Balance Sheets (Pa
Consolidated Balance Sheets (Parenthetical) - $ / shares | Jun. 30, 2019 | Dec. 31, 2018 |
Statement Of Financial Position [Abstract] | ||
Preferred stock, par value | $ 0.0001 | $ 0.0001 |
Preferred stock, shares authorized | 10,000,000 | 10,000,000 |
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 | 36,752,000 | 36,120,000 |
Common stock, shares outstanding | 36,752,000 | 36,120,000 |
Consolidated Statements of Oper
Consolidated Statements of Operations and Comprehensive Income (Loss) - USD ($) shares in Thousands, $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2019 | Jun. 30, 2018 | Jun. 30, 2019 | Jun. 30, 2018 | |
Revenues | ||||
Revenues | $ 7,881 | $ 40,031 | $ 8,765 | $ 172,422 |
Type of Revenue [Extensible List] | us-gaap:LicenseAndServiceMember | us-gaap:LicenseAndServiceMember | us-gaap:LicenseAndServiceMember | us-gaap:LicenseAndServiceMember |
Operating Expenses | ||||
Cost of revenues | $ 1,927 | $ 3,872 | $ 1,956 | $ 6,280 |
Research and development | 29,483 | 21,486 | 54,686 | 41,036 |
General and administrative | 13,405 | 8,318 | 24,963 | 16,698 |
Total operating expenses | 44,753 | 33,681 | 81,543 | 64,047 |
Other operating expenses (income) | (62) | 5 | (62) | 33 |
Income (loss) from operations | (36,872) | 6,350 | (72,778) | 108,375 |
Other Income | ||||
Interest income from licensing | 762 | 6,898 | 1,375 | 8,253 |
Investment income | 34,524 | 1,196 | 37,519 | 2,055 |
Total other income | 35,286 | 8,094 | 38,894 | 10,308 |
Income (loss) before income taxes | (1,586) | 14,444 | (33,884) | 118,683 |
Income Tax Benefit (Expense) | 129 | (3,850) | 199 | (3,850) |
Net income (loss) | (1,457) | 10,594 | (33,685) | 114,833 |
Other Comprehensive Income (Loss) | ||||
Unrealized gain (loss) on available-for-sale securities, net of reclassifications and income tax expense | 530 | 132 | 1,151 | (56) |
Total other comprehensive income (loss) | 530 | 132 | 1,151 | (56) |
Comprehensive income (loss) | (927) | 10,726 | (32,534) | 114,777 |
Net income (loss) applicable to common stockholders | $ (1,457) | $ 10,594 | $ (33,685) | $ 114,833 |
Net income (loss) per share: | ||||
Basic | $ (0.04) | $ 0.33 | $ (0.92) | $ 3.60 |
Diluted | $ (0.04) | $ 0.30 | $ (0.92) | $ 3.29 |
Weighted-average common shares outstanding: | ||||
Basic | 36,669 | 32,082 | 36,518 | 31,858 |
Diluted | 36,669 | 35,272 | 36,518 | 34,884 |
Consolidated Statements of Stoc
Consolidated Statements of Stockholders' Equity - USD ($) shares in Thousands, $ in Thousands | Total | Common Stock [Member] | Additional paid in capital [Member] | Accumulated Other Comprehensive Loss [Member] | Accumulated Deficit [Member] |
Balances at Dec. 31, 2017 | $ 183,029 | $ 3 | $ 371,497 | $ (715) | $ (187,756) |
Balances (Shares) at Dec. 31, 2017 | 31,295 | ||||
Exercise of stock options | 6,999 | 6,999 | |||
Exercise of stock options, Shares | 961 | ||||
Issuance of common stock under employee stock purchase plan | 342 | 342 | |||
Issuance of common stock under employee stock purchase plan, shares | 20 | ||||
Stock-based compensation expense | 7,272 | 7,272 | |||
Unrealized gain (loss) on available-for-sale securities, net of reclassifications and income tax expense | (56) | (56) | |||
Net income (loss) | 114,833 | 114,833 | |||
Balances at Jun. 30, 2018 | 317,222 | $ 3 | 386,110 | (771) | (68,120) |
Balances (Shares) at Jun. 30, 2018 | 32,275 | ||||
Adoption of ASU | ASU 2014-09 [Member] | 4,803 | 4,803 | |||
Balances at Mar. 31, 2018 | 299,340 | $ 3 | 378,954 | (903) | (78,714) |
Balances (Shares) at Mar. 31, 2018 | 31,900 | ||||
Exercise of stock options | 3,174 | 3,174 | |||
Exercise of stock options, Shares | 375 | ||||
Stock-based compensation expense | 3,982 | 3,982 | |||
Unrealized gain (loss) on available-for-sale securities, net of reclassifications and income tax expense | 132 | 132 | |||
Net income (loss) | 10,594 | 10,594 | |||
Balances at Jun. 30, 2018 | 317,222 | $ 3 | 386,110 | (771) | (68,120) |
Balances (Shares) at Jun. 30, 2018 | 32,275 | ||||
Balances at Dec. 31, 2018 | 508,848 | $ 4 | 592,580 | (720) | (83,016) |
Balances (Shares) at Dec. 31, 2018 | 36,120 | ||||
Exercise of stock options | 5,129 | 5,129 | |||
Exercise of stock options, Shares | 622 | ||||
Issuance of common stock under employee stock purchase plan | 365 | 365 | |||
Issuance of common stock under employee stock purchase plan, shares | 10 | ||||
Stock-based compensation expense | 12,817 | 12,817 | |||
Unrealized gain (loss) on available-for-sale securities, net of reclassifications and income tax expense | 1,151 | 1,151 | |||
Net income (loss) | (33,685) | (33,685) | |||
Balances at Jun. 30, 2019 | 494,592 | $ 4 | 610,891 | 471 | (116,774) |
Balances (Shares) at Jun. 30, 2019 | 36,752 | ||||
Adoption of ASU | ASU 2016-02 [Member] | (33) | (33) | |||
Adoption of ASU | ASU 2018-02 [Member] | 40 | (40) | |||
Balances at Mar. 31, 2019 | 487,053 | $ 4 | 602,425 | (59) | (115,317) |
Balances (Shares) at Mar. 31, 2019 | 36,611 | ||||
Exercise of stock options | 1,367 | 1,367 | |||
Exercise of stock options, Shares | 141 | ||||
Stock-based compensation expense | 7,099 | 7,099 | |||
Unrealized gain (loss) on available-for-sale securities, net of reclassifications and income tax expense | 530 | 530 | |||
Net income (loss) | (1,457) | (1,457) | |||
Balances at Jun. 30, 2019 | $ 494,592 | $ 4 | $ 610,891 | $ 471 | $ (116,774) |
Balances (Shares) at Jun. 30, 2019 | 36,752 |
Consolidated Statements of Cash
Consolidated Statements of Cash Flows - USD ($) $ in Thousands | 6 Months Ended | |
Jun. 30, 2019 | Jun. 30, 2018 | |
Cash flows from operating activities | ||
Net income (loss) | $ (33,685) | $ 114,833 |
Adjustments to reconcile net income (loss) to net cash provided by (used in) operating activities | ||
Stock-based compensation expense | 12,817 | 7,272 |
Net amortization of premiums and accretion of discounts on marketable debt securities | (788) | 718 |
Depreciation and amortization | 3,363 | 1,730 |
Imputed interest income from licensing | (1,375) | (8,253) |
Unrealized gains on marketable equity securities | 31,656 | |
Other non-cash adjustments | 442 | 13 |
Changes in operating assets and liabilities | ||
Accounts receivable | (1,419) | 8,879 |
Prepaid expenses | (534) | 1,644 |
Other current assets | 1,419 | (585) |
Operating lease right-of-use assets | 1,156 | |
Other assets | (1,350) | (652) |
Accounts payable | 1,774 | (340) |
Accrued expenses and other current liabilities | (957) | 2,566 |
Deferred revenue | (600) | 600 |
Operating lease liabilities | (1,171) | |
Deferred rent | 19 | |
Other liabilities | (482) | (99) |
Net cash provided by (used in) operating activities | (53,046) | 128,345 |
Cash flows from investing activities | ||
Purchases of marketable securities | (106,119) | (139,081) |
Maturities of marketable securities | 141,262 | 68,645 |
Purchases of property and equipment | (8,010) | (5,017) |
Net cash provided by (used in) investing activities | 27,133 | (75,453) |
Cash flows from financing activities | ||
Proceeds from exercise of stock options | 5,129 | 6,999 |
Proceeds from issuance of common stock under employee stock purchase plan | 365 | 342 |
Net cash provided by financing activities | 5,494 | 7,341 |
Net increase (decrease) in cash and cash equivalents and restricted cash | (20,419) | 60,233 |
Cash and cash equivalents and restricted cash | ||
Beginning of period | 76,614 | 46,881 |
End of period | $ 56,195 | $ 107,114 |
Nature of Business
Nature of Business | 6 Months Ended |
Jun. 30, 2019 | |
Organization Consolidation And Presentation Of Financial Statements [Abstract] | |
Nature of Business | 1. Nature of Business REGENXBIO Inc. (the Company) is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. The Company’s proprietary adeno-associated virus (AAV) gene delivery platform (NAV Technology Platform) consists of exclusive rights to over 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. The Company’s NAV® Technology Platform is being applied by the Company, as well as by third-party licensees (NAV Technology Licensees), in the development of product candidates for a variety of diseases with unmet needs. The Company was formed in 2008 in the State of Delaware and is headquartered in Rockville, Maryland. Liquidity and Risks As of June 30, 2019, the Company had generated an accumulated deficit of $116.8 million since inception. As the Company has incurred cumulative losses since inception, transition to recurring profitability is dependent upon the successful development, approval and commercialization of its product candidates and achieving a level of revenues adequate to support the Company’s cost structure. The Company may never achieve recurring profitability, and unless and until it does, the Company will continue to need to raise additional capital. As of June 30, 2019, the Company had cash, cash equivalents and marketable securities of $449.7 million, which management believes is sufficient to fund operations for at least the next 12 months from the date these consolidated financial statements were issued. The Company is subject to risks common to companies in the biotechnology industry, including, but not limited to, development by the Company or its competitors of technological innovations, risks of failure of clinical trials, dependence on key personnel, protection of proprietary technology, compliance with government regulations and ability to transition from clinical manufacturing to the commercial production of products. |
Summary of Significant Accounti
Summary of Significant Accounting Policies | 6 Months Ended |
Jun. 30, 2019 | |
Accounting Policies [Abstract] | |
Summary of Significant Accounting Policies | 2. Summary of Significant Accounting Policies Basis of Presentation and Principles of Consolidation The accompanying consolidated financial statements are unaudited and have been prepared in conformity with accounting principles generally accepted in the United States of America (GAAP). The interim unaudited consolidated financial statements have been prepared on the same basis as the annual audited consolidated financial statements as of and for the year ended December 31, 2018 included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2018, which was filed with the SEC on February 27, 2019. Certain information and footnote disclosures required by GAAP which are normally included in the Company’s annual consolidated financial statements have been omitted pursuant to SEC rules and regulations for interim reporting. In the opinion of management, the accompanying consolidated financial statements reflect all adjustments, which are normal and recurring in nature, necessary for fair financial statement presentation. The results of operations for the interim periods are not necessarily indicative of the results of operations to be expected for the full year, any other interim periods, or any future year or period. These interim consolidated financial statements should be read in conjunction with the audited consolidated financial statements as of and for the year ended December 31, 2018, and the notes thereto, which are included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2018. The accompanying consolidated financial statements include the accounts of the Company and its wholly owned subsidiaries. All intercompany balances and transactions have been eliminated in consolidation. Use of Estimates The preparation of financial statements in conformity with GAAP requires management to make estimates and assumptions that affect the reported amounts in the financial statements and accompanying notes. Actual results could differ materially from those estimates. Management considers many factors in selecting appropriate financial accounting policies and controls, and in developing the estimates and assumptions that are used in the preparation of these consolidated financial statements. Management must apply significant judgment in this process. In addition, other factors may affect estimates, including: expected business and operational changes, sensitivity and volatility associated with the assumptions used in developing estimates and whether historical trends are expected to be representative of future trends. The estimation process often may yield a range of potentially reasonable estimates of the ultimate future outcomes and management must select an amount that falls within that range of reasonable estimates. This process may result in actual results differing materially from those estimated amounts used in the preparation of the consolidated financial statements. Significant estimates are used in the following areas, among others: license and royalty revenue, stock-based compensation expense, accrued research and development expenses and other accrued liabilities, income taxes and the fair value of financial instruments. Reclassifications Certain amounts reported in prior periods have been reclassified to conform to current period financial statement presentation. These reclassifications are not material and have no effect on previously reported financial position, results of operations and cash flows. Restricted Cash Restricted cash includes money market mutual funds used to collateralize irrevocable letters of credit as required by the Company’s lease agreements. The following table provides a reconciliation of cash and cash equivalents and restricted cash as reported on the consolidated balance sheets to the total of these amounts as reported at the end of the period in the consolidated statements of cash flows (in thousands): June 30, 2019 June 30, 2018 Cash and cash equivalents $ 55,142 $ 106,889 Restricted cash 1,053 225 Total cash and cash equivalents and restricted cash $ 56,195 $ 107,114 Marketable Securities Marketable securities consist of available-for-sale debt securities and equity securities and are carried at fair value. Marketable debt securities with remaining maturity dates exceeding 12 months which are not intended to be sold prior to maturity for use in current operations are classified as non-current assets. Marketable equity securities are classified as current assets. Unrealized gains and losses on available-for-sale debt securities, net of any related tax effects, are excluded from results of operations and are included in other comprehensive income (loss) and reported as a separate component of stockholders’ equity until realized. The Company uses the aggregate portfolio approach to release the tax effects of unrealized gains and losses on available-for-sale debt securities in accumulated other comprehensive income (loss). Purchase premiums and discounts on marketable debt securities are amortized or accreted into the cost basis over the life of the related security as adjustments to the yield using the effective-interest method. Interest income is recognized when earned. Unrealized gains and losses on marketable equity securities are included in results of operations as investment income. Realized gains and losses from the sale or maturity of marketable securities are based on the specific identification method and are included in results of operations as investment income. A decline in the fair value below cost of available-for-sale debt securities that is deemed other-than-temporary is charged to results of operations, resulting in the establishment of a new cost basis for the security. The Company regularly evaluates whether declines in the fair value of its debt securities below their cost are other-than-temporary. The evaluation includes consideration of the cause of the impairment, including the creditworthiness of the security issuers, the number of securities in an unrealized loss position, the severity and duration of the unrealized losses, whether the Company has the intent to sell the securities and whether it is more likely than not that the Company will be required to sell the securities before the recovery of their amortized cost basis. The Company has not recorded any impairment of available-for-sale debt securities which was deemed to be to be other-than-temporary. Leases Effective January 1, 2019, the Company adopted Accounting Standards Update (ASU) 2016-02, Leases Leases Under Topic 842, the Company classifies its leases as either finance or operating leases based on the principle of whether or not the lease is effectively a financed purchase by the Company. Lease classification is evaluated at the inception of the lease agreement. Regardless of classification, the Company records a right-of-use asset and a lease liability for all leases with a term greater than 12 months. All of the Company’s leases as of June 30, 2019 have been classified as operating leases. Operating lease expense is recognized on a straight-line basis over the term of the lease, with the exception of variable lease expenses which are recognized as incurred. The Company identifies leases in its contracts if the contract conveys the right to control the use of identified property, plant or equipment for a period of time in exchange for consideration. The Company does not allocate lease consideration between lease and nonlease components and records a lease liability equal to the present value of the remaining fixed consideration under the lease. The interest rates implicit in the Company’s leases are generally not readily determinable. Accordingly, the Company uses its estimated incremental borrowing rate at the commencement date of the lease to determine the present value discount of the lease liability. The Company estimates its incremental borrowing rate for each lease based on an evaluation of its expected credit rating and the prevailing market rates for collateralized debt in a similar economic environment with similar payment terms and maturity dates commensurate with the term of the lease. The right-of-use asset for each lease is equal to the lease liability, adjusted for unamortized initial direct costs and lease incentives and prepaid or accrued rent. Initial direct costs of entering into a lease are included in the right-of-use asset and amortized as lease expense over the term of the lease. Lease incentives, such as tenant improvements allowances, are recorded as a reduction of the right-of-use asset and amortized as a reduction of lease expense over the term of the lease. The Company excludes options to extend or terminate leases from the calculation of the lease liability unless it is reasonably certain the option will be exercised. Fair Value of Financial Instruments 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. ASC 820, Fair Value Measurements and Disclosures • Level 1—Valuations based on unadjusted quoted prices in active markets for identical assets or liabilities that the Company has the ability to access at the measurement date. • Level 2—Valuations based on quoted prices for similar assets or liabilities in markets that are not active or for which all significant inputs are observable, either directly or indirectly. • Level 3—Valuations that require inputs that reflect the Company’s own assumptions that are both significant to the fair value measurement and unobservable. To the extent that valuation is based on models or inputs that are less observable or unobservable in the market, the determination of fair value requires more judgment. Accordingly, the degree of judgment exercised by the Company in determining fair value is greatest for instruments categorized in Level 3. A financial instrument’s level within the fair value hierarchy is based on the lowest level of any input that is significant to the fair value measurement. The fair values of the Company’s Level 2 instruments are based on quoted market prices or broker or dealer quotations for similar assets. These investments are initially valued at the transaction price and subsequently valued utilizing third party pricing providers or other market observable data. Please refer to Note 4 for further information on the fair value measurement of the Company’s financial instruments. Revenue Recognition The Company recognizes revenue in accordance with ASC 606, Revenue from Contracts with Customers The Company applies the five-step model to contracts that are within the scope of Topic 606 only when it is probable that the Company will collect the consideration it is entitled to in exchange for the goods or services it transfers to the customer. At contract inception, for contracts within the scope of Topic 606, the Company assesses the goods or services promised within each contract and determine those that are performance obligations and whether each promised good or service is distinct. The Company then recognizes as revenue the amount of the transaction price that is allocated to respective performance obligations when (or as) the respective performance obligations are satisfied. The Company evaluates its contracts with customers for the presence of significant financing components. If a significant financing component is identified in a contract and provides a financing benefit to the customer, the transaction price for the contract is adjusted to account for the financing portion of the arrangement, which is recognized as interest income over the financing term using the effective interest method. In determining the appropriate interest rates for significant financing components, the Company evaluates the credit profile of the customer and prevailing market interest rates and selects an interest rate in which it believes would be charged to the customer in a separate financing arrangement over a similar financing term. License and Royalty Revenue The Company licenses its NAV Technology Platform to other biotechnology and pharmaceutical companies. The terms of the licenses vary, and licenses may be exclusive or non-exclusive and may be sublicensable by the licensee. Licenses may grant intellectual property rights for purposes of internal and preclinical research and development only, or may include the rights, or options to obtain future rights, to commercialize drug therapies for specific diseases using the Company’s NAV Technology Platform. License agreements generally have a term at least equal to the life of the underlying patents, but are terminable at the option of the licensee. Consideration payable to the Company under its license agreements may include: (i) up-front and annual fees, (ii) option fees to acquire additional licenses, (iii) milestone payments based on the achievement of certain development and sales-based milestones by licensees, (iv) sublicense fees and (v) royalties on sales of licensed products. The Company’s license agreements are accounted for as contracts with customers within the scope of Topic 606. At the inception of each license agreement, the Company determines the contract term for purposes of applying the requirements of Topic 606. Licenses are generally terminable at the option of the licensee with advance notice to the Company. For each license, the Company evaluates these termination rights to determine whether a substantive termination penalty would be incurred by the licensee upon termination. If the licensee incurs a substantive termination penalty upon termination, the contract term for revenue recognition purposes is generally equal to the stated term of the license, which is the life of the underlying licensed patents. Alternatively, if the licensee does not incur a substantive termination penalty upon termination, the contract term for revenue recognition purposes may be shorter than the stated term of the license, in which case the termination rights may be accounted for as contract renewal options. The determination of whether a substantive termination penalty is associated with the termination rights requires significant judgment. In making this determination, the Company considers, among other things, the nature of the intellectual property rights that would be returned to the Company upon termination, including the exclusivity of the licensed rights and the stage of development of the licensed products, the payment terms, including the amount and timing of non-refundable or guaranteed payments, and the business purpose of the termination rights granted to the licensee. The Company considers all of the facts and circumstances relevant to each license when making this determination. Performance obligations under the Company’s license agreements may include (i) the delivery of intellectual property licenses and (ii) options granted to licensees to acquire additional licenses to the extent the options represent material rights to the licensee. At the inception of each license agreement which contains options for the licensee to acquire additional licenses, or contract renewal options, the Company evaluates the options to determine whether they provide material rights to the licensee. In making this determination, the Company considers whether the options are priced at a discount to the standalone selling price for the underlying licenses. If an option is priced at a discount to the standalone selling price for the underlying license, the option is considered to be a material right to the licensee and is accounted for as a separate performance obligation under the current license agreement. The Company evaluates the transaction price of its license agreements at the inception of each agreement and at each reporting date. The transaction price includes the fixed consideration payable to the Company during the contract term, as well as any variable consideration to the extent that it is probable that a significant reversal of revenue will not occur in the future. Fixed consideration under the license agreements includes up-front and annual fees payable during the contract term. Variable consideration under the license agreements includes development and sales-based milestone payments, sublicense fees and royalties on sales of licensed products. Consideration contingent upon the exercise of options by a licensee is excluded from the transaction price and not accounted for as part of the license agreement until the option is exercised. The transaction price for each license agreement is allocated to the underlying performance obligations and recognized as revenue when the performance obligations are satisfied. Consideration allocated to performance obligations for the delivery of an intellectual property license is recognized as revenue in full upon the delivery of the license to the licensee. Consideration allocated to performance obligations for license options is recognized as revenue in full upon the earlier of the option exercise or expiration. The exercise of a license option by a licensee is accounted for as a new license for revenue recognition purposes. Up-front and annual licenses fees payable to the Company over the contract term of each license are included in the transaction price, and the portion of this consideration that is allocated to the performance obligation for the delivery of the intellectual property license is recognized as revenue in full upon the delivery of the license to the licensee. If annual license fees are payable to the Company in periods beyond 12 months from the delivery of the license, a significant financing component is deemed to exist which provides a financing benefit to the licensee. If a significant financing component is identified, the Company adjusts the transaction price for the license to include only the present value of the annual license fees payable to the Company over the contract term. The discounted portion of the license fees is recognized as interest income from licensing over the financing period of the license. Development milestone payments are payable to the Company upon the achievement of specified development milestones by licensees. At the inception of each license agreement that contains development milestone payments, the Company evaluates whether the milestones are considered probable of achievement and estimates the amount to be included in the transaction price using the most likely amount method. If it is probable that a significant revenue reversal will not occur in the future, milestone payments are included in the transaction price and recognized as revenue upon the delivery of the license. Milestone payments contingent on the achievement of development milestones that are not within the control of the Company or the licensee, such as regulatory approvals, are not considered probable of being achieved and are excluded from the transaction price until the milestone is achieved. At each reporting date, the Company re-evaluates the probability of achievement of outstanding development milestones and, if necessary, adjusts the transaction price for any milestones for which the probability of achievement has changed due to current facts and circumstances. Any such adjustments are recorded on a cumulative catch-up basis and recognized as revenue in the period of the adjustment. Royalties on sales of licensed products, sales-based milestone payments and sublicense fees based on the receipt of certain fees by licensees from any sublicensees are excluded from the transaction price of each license and recognized as revenue in the period that the related sales or sublicenses occur, provided that the associated license has been delivered to the licensee. Royalty revenue to date consists of royalties on net sales of Zolgensma®, which is marketed by AveXis, Inc. (AveXis), a wholly owned subsidiary of Novartis AG (Novartis). Zolgensma is a licensed product under the Company’s March 2014 license agreement, as amended, with AveXis for the development and commercialization of treatments for spinal muscular atrophy (SMA). The Company recognizes royalty revenue from net sales of Zolgensma in the period in which the underlying products are sold by AveXis, which in certain cases may require the Company to estimate royalty revenue for periods of net sales which have not yet been reported to the Company. Sales-based milestone payments related to net sales of Zolgensma are recognized as royalty revenue in the period in which the milestone is achieved. The Company receives payments from licensees based on the billing schedules established in each license agreement. Amounts recognized as revenue which have not yet been received from licensees, including unbilled royalties, are recorded as accounts receivable when the Company’s rights to the consideration are conditional solely upon the passage of time. Amounts recognized as revenue which have not yet been received from licensees are recorded as contract assets when the Company’s rights to the consideration are not unconditional. Contract assets are recorded as other current assets on the consolidated balance sheets. If a licensee elects to terminate a license prior to the end of the license term, the licensed intellectual property is returned to the Company and any consideration recorded as accounts receivable or contract assets which is not contractually payable by the licensee is charged off as a reduction of license revenue in the period of the termination. Amounts received by the Company prior to the delivery of underlying performance obligations are deferred and recognized as revenue upon the satisfaction of the performance obligations by the Company. Deferred revenue which is not expected to be recognized within 12 months from the reporting date is recorded as non-current on the consolidated balance sheets. Cost of Revenues Cost of revenues consists primarily of sublicense fees, milestone payments and royalties on net sales of licensed products as specified in the Company’s agreements with its licensors. Sublicense fees are based on a percentage of license fees received by the Company from NAV Technology Licensees and are recognized in the period that the underlying license revenue is recognized. Milestone payments are payable to licensors upon the achievement of specified milestones by NAV Technology Licensees and are recognized in the period the milestone is achieved or deemed probable of achievement. Royalties are based on a percentage of net sales of licensed products by NAV Technology Licensees and are recognized in the period that the underlying sales occur. Amounts which are payable to licensors in periods beyond 12 months from the reporting date are recorded as non-current liabilities on the consolidated balance sheets. Net Income (Loss) Per Share Basic net income (loss) per share is calculated by dividing net income (loss) applicable to common stockholders by the weighted-average common shares outstanding during the period, without consideration for common stock equivalents. Diluted net income (loss) per share is calculated by adjusting the weighted-average common shares outstanding for the dilutive effect of common stock equivalents outstanding for the period, determined using the treasury-stock method. Contingently convertible shares in which conversion is based on non-market-priced contingencies are excluded from the calculations of both basic and diluted net income (loss) per share until the contingency has been fully met. For purposes of the diluted net income (loss) per share calculation, common stock equivalents are excluded from the calculation of diluted net income (loss) per share if their effect would be anti-dilutive. Recent Accounting Pronouncements Adoption of ASU 2016-02, Leases In February 2016, the Financial Accounting Standards Board (FASB) issued ASU 2016-02, Leases Leases The Company elected certain practical expedients allowed by Topic 842 for transition purposes, including the package of practical expedients which permitted the Company to not reassess lease identification, classification and initial direct costs under Topic 842 for leases that commenced prior to January 1, 2019. Additionally, the Company elected the practical expedient allowed for transition purposes to use hindsight in determining the terms of leases that commenced prior to January 1, 2019. Upon the adoption of Topic 842, the Company recorded operating lease right-of-use assets of $7.4 million and operating lease liabilities of $8.4 million for its leases which were in effect and had commenced prior to January 1, 2019 and had original lease terms of more than 12 months. The Company also derecognized current and non-current deferred rent liabilities of $1.4 million and prepaid expenses, other current assets and other assets of $0.4 million upon the adoption of Topic 842. Additionally, upon the adoption of Topic 842, the Company derecognized $5.9 million of property and equipment and $5.9 million of financing lease obligations related to construction-in-progress at 9800 Medical Center Drive, as the Company does not control the building during the construction period under the requirements of Topic 842. The lease term for the facility at 9800 Medical Center Drive does not commence until certain construction is completed by the landlord and the building is delivered to the Company. The right-of-use assets and lease liabilities related to the facility at 9800 Medical Center Drive will not be recognized on the Company’s consolidated balance sheets until the commencement date of the lease, which is expected to occur in 2020. The cumulative impact of the adoption of Topic 842 resulted in an increase in accumulated deficit of less than $0.1 million on January 1, 2019. The adoption of Topic 842 did not have a material impact on the Company’s results of operations for the three and six months ended June 30, 2019, nor does the Company believe it will have a material impact on future results of operations based on its current leasing arrangements. Other R ecently A dopted A ccounting P ronouncements In February 2018, the FASB issued ASU 2018-02, Income Statement—Reporting Comprehensive Income (Topic 220): Reclassification of Certain Tax Effects from Accumulated Other Comprehensive Income Tax Cuts and Jobs Act of 2017 (the TCJA) In April 2017, the FASB issued ASU 2017-08, Receivables—Nonrefundable Fees and Other Costs (Subtopic 310-20) Recent Accounting Pronouncements Not Yet Adopted In August 2018, the FASB issued ASU 2018-15, Intangibles—Goodwill and Other—Internal-Use Software (Subtopic 350-40): Customer’s Accounting for Implementation Costs Incurred in a Cloud Computing Arrangement That Is a Service Contract In August 2018, the FASB issued ASU 2018-13, Fair Value Measurement (Topic 820): Disclosure Framework—Changes to the Disclosure Requirements for Fair Value Measurement In June 2016, the FASB issued ASU 2016-13, Financial Instruments—Credit Losses (Topic 326): Measurement of Credit Losses on Financial Instruments |
Marketable Securities
Marketable Securities | 6 Months Ended |
Jun. 30, 2019 | |
Investments Debt And Equity Securities [Abstract] | |
Marketable Securities | 3. Marketable Securities The following tables present a summary of the Company’s marketable securities, which consist of available-for-sale debt securities and equity securities (in thousands): Amortized Cost Unrealized Gains Unrealized Losses Fair Value June 30, 2019 U.S. government and federal agency securities $ 102,825 $ 388 $ — $ 103,213 Certificates of deposit 8,748 67 — 8,815 Corporate bonds 249,377 1,070 (3 ) 250,444 Equity securities 420 31,656 — 32,076 $ 361,370 $ 33,181 $ (3 ) $ 394,548 Amortized Cost Unrealized Gains Unrealized Losses Fair Value December 31, 2018 U.S. government and federal agency securities $ 103,410 $ 93 $ (37 ) $ 103,466 Certificates of deposit 8,992 — — 8,992 Corporate bonds 282,902 36 (377 ) 282,561 $ 395,304 $ 129 $ (414 ) $ 395,019 As of June 30, 2019 and December 31, 2018, no available-for-sale debt securities had remaining maturities greater than three years. The amortized cost of marketable debt securities is adjusted for amortization of premiums and accretion of discounts to maturity, or to the earliest call date for callable debt securities purchased at a premium. As of June 30, 2019 and December 31, 2018, the balance in the Company’s accumulated other comprehensive income (loss) consisted solely of net unrealized gains and losses on available-for-sale debt securities, net of income tax effects and reclassification adjustments for realized gains and losses. During the three and six months ended June 30, 2019, the Company recognized net unrealized gains on available-for-sale debt securities of $0.8 million and $1.8 million, respectively, and income tax expense of $0.3 million and $0.7 million, respectively, in other comprehensive income for the periods. The Company recognized net realized gains of zero and less than $0.1 million, respectively, on the sale or maturity of available-for-sale debt securities during the three and six months ended June 30, 2019, respectively, which were reclassified out of accumulated other comprehensive income (loss) during the periods and are included in investment income in the consolidated statements of operations and comprehensive income (loss). During the three and six months ended June 30, 2018, the Company recognized net unrealized gains (losses) on available-for-sale debt securities of $0.1 million and $(0.1) million, respectively, and income tax expense of zero in other comprehensive income (loss) for the periods. The Company did not recognize any realized gains or losses on the sale or maturity of available-for-sale debt securities during the three and six months ended June 30, 2018. Realized gains and losses from the sale or maturity of available-for-sale debt securities are determined based on the specific identification method. The following tables present the fair values and unrealized losses of available-for-sale debt securities held by the Company in an unrealized loss position for less than 12 months and 12 months or greater (in thousands): Less than 12 Months 12 Months or Greater Total Fair Value Unrealized Losses Fair Value Unrealized Losses Fair Value Unrealized Losses June 30, 2019 Corporate bonds $ 19,176 $ (3 ) $ — $ — $ 19,176 $ (3 ) $ 19,176 $ (3 ) $ — $ — $ 19,176 $ (3 ) Less than 12 Months 12 Months or Greater Total Fair Value Unrealized Losses Fair Value Unrealized Losses Fair Value Unrealized Losses December 31, 2018 U.S. government and federal agency securities $ 53,124 $ (37 ) $ — $ — $ 53,124 $ (37 ) Corporate bonds 245,283 (354 ) 12,424 (23 ) 257,707 (377 ) $ 298,407 $ (391 ) $ 12,424 $ (23 ) $ 310,831 $ (414 ) As of June 30, 2019, available-for-sale debt securities held by the Company which were in an unrealized loss position consisted of 5 investment grade security positions. The Company has the intent and ability to hold such securities until recovery and has determined that none of the securities were other-than-temporarily impaired as of June 30, 2019 or December 31, 2018. Marketable equity securities held by the Company as of June 30, 2019 consist solely of common stock of Prevail Therapeutics Inc. (Prevail). The Company acquired the securities as consideration for a commercial license to the NAV Technology Platform granted to Prevail in August 2017. Prevail completed its initial public offering (IPO) in June 2019. Prior to Prevail’s IPO, the securities were accounted for as non-marketable equity securities without a readily determinable fair value and had a carrying value of $0.4 million. Upon Prevail’s IPO in June 2019, the securities were reclassified to marketable securities and are measured at fair value. The Company recorded an unrealized gain of $31.7 million during the three and six months ended June 30, 2019 related to the marketable equity securities of Prevail, which is included in investment income in the consolidated statements of operations and comprehensive income (loss). Pursuant to a lock-up agreement executed in connection with Prevail’s IPO, the Company is restricted from selling its common stock of Prevail prior to December 2019. As of June 30, 2019, no amounts have been realized by the Company related to its marketable securities of Prevail as there have been no sales of the securities. |
Fair Value of Financial Instrum
Fair Value of Financial Instruments | 6 Months Ended |
Jun. 30, 2019 | |
Fair Value Disclosures [Abstract] | |
Fair Value of Financial Instruments | 4. Fair Value of Financial Instruments Financial instruments reported at fair value on a recurring basis include cash equivalents and marketable securities. The following tables present the fair value of cash equivalents and marketable securities in accordance with the hierarchy discussed in Note 2 (in thousands): Quoted Significant prices other Significant in active observable unobservable markets inputs inputs (Level 1) (Level 2) (Level 3) Total June 30, 2019 Cash equivalents: Money market mutual funds $ — $ 55,126 $ — $ 55,126 Total cash equivalents — 55,126 — 55,126 Marketable securities: U.S. government and federal agency securities — 103,213 — 103,213 Certificates of deposit — 8,815 — 8,815 Corporate bonds — 250,444 — 250,444 Equity securities 32,076 — — 32,076 Total marketable securities 32,076 362,472 — 394,548 Total cash equivalents and marketable securities $ 32,076 $ 417,598 $ — $ 449,674 Quoted Significant prices other Significant in active observable unobservable markets inputs inputs (Level 1) (Level 2) (Level 3) Total December 31, 2018 Cash equivalents: Money market mutual funds $ — $ 75,542 $ — $ 75,542 Total cash equivalents — 75,542 — 75,542 Marketable securities: U.S. government and federal agency securities — 103,466 — 103,466 Certificates of deposit — 8,992 — 8,992 Corporate bonds — 282,561 — 282,561 Total marketable securities — 395,019 — 395,019 Total cash equivalents and marketable securities $ — $ 470,561 $ — $ 470,561 There were no transfers of financial instruments between levels of the fair value hierarchy during the six months ended June 30, 2019. Management estimates that the carrying amounts of its current accounts receivable, accounts payable and accrued expenses and other current liabilities approximate fair value due to the short-term nature of those instruments. Accounts receivable which contain non-current portions are recorded at their present values using a discount rate that is based on prevailing market rates and the credit profile of the licensee on the date the amounts are initially recorded. Management does not believe there have been any significant changes in market conditions or credit quality that would cause the discount rates initially used to be significantly different from those that would be used as of June 30, 2019 to determine the present value of the receivables. Accordingly, management estimates that the carrying value of its non-current accounts receivable approximates the fair value of those instruments. Non-marketable equity securities are measured at cost less impairment, adjusted for observable price changes for identical or similar investments of the same issuer. As of December 31, 2018, non-marketable equity securities had a carrying value of $0.4 million and were included in other assets on the consolidated balance sheets. The Company did not identify any observable price changes or changes in circumstances that would have had an adverse effect on the fair value of the securities as of December 31, 2018. In June 2019, all of the Company’s non-marketable equity securities were reclassified to marketable securities and as of June 30, 2019, the Company did not hold any non-marketable equity securities. No remeasurements or impairment losses were recorded on non-marketable equity securities during the three and six months ended June 30, 2019 and 2018. |
Property and Equipment, Net
Property and Equipment, Net | 6 Months Ended |
Jun. 30, 2019 | |
Property Plant And Equipment [Abstract] | |
Property and Equipment, Net | 5. Property and Equipment, Net Property and equipment, net consists of the following (in thousands): June 30, 2019 December 31, 2018 Lab equipment $ 16,533 $ 14,417 Computer equipment and software 2,342 2,002 Furniture and fixtures 1,946 1,915 Leasehold improvements 17,092 11,751 Construction-in-progress — 5,854 Total property and equipment 37,913 35,939 Accumulated depreciation and amortization (10,583 ) (7,237 ) Property and equipment, net $ 27,330 $ 28,702 Construction-in-progress reported in the table above as of December 31, 2018 consisted of certain costs incurred and reported by the Company’s landlord at 9800 Medical Center Drive. Upon the adoption of Topic 842 on January 1, 2019, the Company derecognized the cumulative amount of construction costs incurred by the landlord of $5.9 million. Please refer to Note 2 for further information on the Company’s adoption of Topic 842 and Note 6 for further information on the Company’s lease at 9800 Medical Center Drive. |
Leases
Leases | 6 Months Ended |
Jun. 30, 2019 | |
Leases [Abstract] | |
Leases | 6. Leases 9800 Medical Center Drive In November 2018, the Company entered into a lease agreement, as amended in April 2019, for approximately 139,000 square feet of office and laboratory facilities in a new building to be constructed at 9800 Medical Center Drive in Rockville, Maryland (the 9800 Medical Center Drive Lease). The initial construction of the building is being conducted by the landlord and is expected to be completed in 2020, after which the leased premises will be delivered to the Company to make additional improvements to the building. Pursuant to the amended lease agreement, the Company will receive a $15.3 million tenant improvement allowance from the landlord to construct additional improvements to the leased premises. The lease expires approximately 16 years from delivery of the leased premises to the Company, subject to certain extension and termination options held by the Company. The Company has the option to extend the term of the lease for up to 10 additional years and the option to terminate the lease after 12 years from the delivery of the leased premises to the Company. If the Company elects to terminate the lease, it will be subject to a termination fee equal to the unamortized tenant improvement allowance, rent abatement and landlord commissions as of the termination date, bearing interest at 5% per annum, plus four months of base rent and operating expenses. Additionally, after the delivery of the leased premises under the 9800 Medical Center Drive Lease, the Company will have the option to terminate its lease at 9712 Medical Center Drive with six months’ notice. Monthly payments under the 9800 Medical Center Drive Lease begin approximately 12 months from the delivery of the leased premises to the Company and escalate annually in accordance with the lease agreement. As required by the lease agreement, the Company has provided the landlord with an irrevocable letter of credit of $0.8 million which the landlord may draw upon in the event of any uncured default by the Company under the terms of the lease. The Company is involved in the construction project for the leased premises at 9800 Medical Center Drive, including having the responsibility to pay for a portion of the costs of non-normal tenant improvements such as finish work, mechanical, electrical and plumbing elements of the building, among other items. As of December 31, 2018, under the requirements of Topic 840, the Company was deemed the owner of the leased premises during the construction period for accounting purposes and certain estimated construction costs incurred and reported by the landlord were recorded as property and equipment, with a corresponding financing lease obligation, on the consolidated balance sheet. The Company has determined that it does not control the building during the construction period under the requirements of Topic 842. Accordingly, upon the adoption of Topic 842 on January 1, 2019, the Company derecognized property and equipment of $5.9 million for the cumulative costs of construction incurred by the landlord as well as the associated $5.9 million financing lease obligation. As of June 30, 2019, the Company had recorded $4.8 million of costs related to construction-in-progress at 9800 Medical Center Drive, which have been recorded as leasehold improvements within property and equipment on the consolidated balance sheets. As of June 30, 2019, the right-of-use assets and lease liabilities related to the 9800 Medical Center Drive Lease have not been recorded on the Company’s consolidated balance sheets and will be measured and recognized on the commencement date of the lease, which is expected to occur in 2020 when the landlord delivers the newly constructed building to the Company. Other Leases In March 2015, the Company entered into an operating lease for office space at 9712 Medical Center Drive in Rockville, Maryland (the 9712 Medical Center Drive Lease). The lease term commenced in April 2015. Monthly payments under the lease began in October 2015 and escalate annually in accordance with the lease agreement. In September 2015, November 2015, July 2017 and April 2018, the Company amended the 9712 Medical Center Drive Lease to include additional office and laboratory space at 9714 Medical Center Drive, and ultimately extend the term of the lease to September 2021. The Company has options to extend the term of the 9712 Medical Center Drive Lease for up to six additional years. Additionally, upon the commencement of the 9800 Medical Center Drive Lease, the Company will have the option to terminate the 9712 Medical Center Drive Lease with six months’ notice. The Company’s extension and termination options under the 9712 Medical Center Drive Lease have been excluded from the measurement of the right-of-use assets and lease liabilities for the lease as they are not reasonably certain of exercise. The Company received a $0.4 million tenant improvement allowance from the landlord which has been recorded as a reduction of the right-of-use assets for the lease and is amortized on a straight-line basis as a reduction of rent expense over the term of the lease. In January 2016, the Company entered into an operating lease for its corporate headquarters at 9600 Blackwell Road in Rockville, Maryland (the Blackwell Road Lease). The lease commenced in February 2016 and expires in September 2023. In November 2017, the Blackwell Road Lease was amended to include additional office space for the remainder of the lease term. Monthly payments under the lease began in September 2016 and escalate annually in accordance with the lease agreement. The Company has an option to extend the term of the Blackwell Road Lease for up to five additional years and the option to terminate the lease after 67 months from the lease commencement date. If the Company elects to terminate the lease, it will be subject to a termination fee equal to the unamortized tenant improvement allowance, rent abatement and landlord costs and commissions as of the termination date, bearing interest at 8% per annum. The Company’s extension and termination options under the Blackwell Road Lease have been excluded from the measurement of the right-of-use assets and lease liabilities for the lease as they are not reasonably certain of exercise. The Company received a $0.8 million tenant improvement allowance from the landlord which has been recorded as a reduction of the right-of-use assets for the lease and is amortized on a straight-line basis as a reduction of rent expense over the term of the lease. In May 2016, the Company entered into an operating lease for office space at 400 Madison Avenue in New York, New York (the 400 Madison Lease). The lease commenced in July 2016 and monthly payments under the lease began in October 2016 and escalate annually in accordance with the lease agreement. As required by the lease agreement, the Company has provided the landlord with an irrevocable letter of credit of $0.2 million which the landlord may draw upon in the event of any uncured default by the Company under the terms of the lease. In May 2019, the 400 Madison Lease was amended to include additional office space and extend the term of the lease. Prior to the amendment, the lease was to expire in October 2020. Pursuant to the amendment, the Company will vacate the original leased premises in the third quarter of 2019, upon which the landlord will perform demolition of the original premises as well as the additional premises under the amended lease. Upon completion of the demolition, the landlord will deliver the entire expanded premises to the Company to make tenant improvements. The amended lease will expire approximately 7.5 years from the date the expanded premises are delivered to the Company, which is expected to occur in 2019. The Company will receive a $0.7 million tenant improvement allowance from the landlord to construct improvements to the leased premises. As a result of the amendment in May 2019, the expiration of the lease term for the original premises under the 400 Madison Lease was adjusted from October 2020 to August 2019. Accordingly, the right-of-use assets and lease liabilities for the original premises were reduced by $0.4 million to account for the modification of the lease term. As of June 30, 2019, the Company does not have control of the additional leased premises under the amended lease and the right-of-use assets and lease liabilities have not been recorded on the Company’s consolidated balance sheets. The right-of-use assets and lease liabilities related to the expanded premises for the extended lease term will be measured and recognized upon the delivery of the expanded premises to the Company to make tenant improvements. The Company leases additional office and laboratory facilities in Rockville, Maryland, as well as laboratory and other equipment, under operating leases with various expiration dates through 2022. Operating Lease Information All of the Company’s leases are classified as operating leases. The following table summarizes the Company’s lease costs and supplemental cash flow information related to operating leases (in thousands): Three Months Ended Six Months Ended June 30, 2019 June 30, 2019 Operating lease cost $ 697 $ 1,398 Variable lease cost 138 298 Total lease cost $ 835 $ 1,696 Cash paid for amounts included in operating lease liabilities $ 684 $ 1,395 Right-of-use assets acquired through operating lease liabilities $ (371 ) $ (335 ) Right-of-use assets acquired through operating lease liabilities for the three and six months ended June 30, 2019 includes a reduction of $0.4 million related to the April 2019 amendment to the 400 Madison Lease for the adjustment of the lease term. Short-term lease expense for the three and six months ended June 30, 2019 was not material and is included in operating lease cost in the table above. Variable lease cost under the Company’s operating leases includes items such as common area maintenance, utilities, taxes and other charges. The weighted-average remaining lease term and weighted-average discount rate of the Company’s operating leases were as follows: As of June 30, 2019 Weighted-average remaining lease term (years) 2.9 Weighted-average discount rate 5.6 % The following table presents a reconciliation of the undiscounted future minimum lease payments remaining under leases that have not yet commenced and other operating leases to the amounts reported as operating lease liabilities on the consolidated balance sheet as of June 30, 2019 (in thousands): Leases Other Total Not Yet Operating Minimum Lease Commenced (a) Leases Payments Undiscounted future minimum lease payments: 2019 (remainder of year) $ 98 $ 1,200 $ 1,298 2020 460 2,813 3,273 2021 2,308 2,412 4,720 2022 5,422 623 6,045 2023 6,376 479 6,855 Thereafter 83,650 — 83,650 Total undiscounted future minimum lease payments $ 98,314 $ 7,527 $ 105,841 Amount representing imputed interest (597 ) Total operating lease liabilities 6,930 Current portion of operating lease liabilities (2,276 ) Operating lease liabilities, non-current $ 4,654 (a) Includes undiscounted future minimum lease payments under the 9800 Medical Center Drive Lease and 400 Madison Lease which are not included in the lease liabilities reported on the consolidated balance sheet as of June 30, 2019. The actual timing and amounts of these payments are subject to adjustment based on the commencement dates and actual square footage of the leased premises. Accordingly, these amounts were estimates as of June 30, 2019. As of December 31, 2018, future minimum lease payments under Topic 840 for the 9800 Medical Center Drive Lease and other operating leases were as follows (in thousands): 9800 Medical Other Total Center Drive Operating Minimum Lease Lease (a) Leases Payments 2019 $ — $ 2,798 $ 2,798 2020 — 3,054 3,054 2021 1,329 2,391 3,720 2022 4,289 621 4,910 2023 5,156 479 5,635 Thereafter 76,420 — 76,420 Total minimum lease payments $ 87,194 $ 9,343 $ 96,537 (a) Includes all future minimum lease payments under the 9800 Medical Center Drive Lease, including amounts recorded as financing lease obligations on the consolidated balance sheet . The actual timing and amounts of payments under the 9800 Medical Center Drive Lease are subject to adjustment based on the commencement date and actual square footage of the leased premises. Accordingly, these amounts were estimates as of December 31, 2018. |
Commitments and Contingencies
Commitments and Contingencies | 6 Months Ended |
Jun. 30, 2019 | |
Commitments And Contingencies Disclosure [Abstract] | |
Commitments and Contingencies | 7. Commitments and Contingencies License from The Trustees of the University of Pennsylvania In February 2009, the Company entered into a license agreement, which has been amended from time to time, with The Trustees of the University of Pennsylvania (together with the University of Pennsylvania, Penn) for exclusive, worldwide rights to certain patents owned by Penn underlying the Company’s NAV Technology Platform, as well as exclusive rights to certain data, results and other information generated in connection with the clinical trial for RGX-501, the Company’s product candidate for the treatment of homozygous familial hypercholesterolemia (HoFH). Pursuant to the license agreement, the Company and is obligated to pay Penn royalties on net sales of licensed products and sublicense fees. Additionally, the Company is obligated to reimburse Penn for certain costs incurred related to the maintenance of the licensed patents. In April 2019, the Company amended its license from Penn to include exclusive license rights to certain know-how, including research data and other information, relating to the treatment of late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease. In consideration for the additional licensed rights, and in addition to any consideration owed under the license prior to the amendment, the Company paid Penn an upfront fee and is obligated to pay milestone fees of up to $20.5 million upon the achievement of various development and sales-based milestones and additional royalties on net sales of licensed products for the treatment of CLN2 disease. Additionally, the amendment modifies the percentage of sublicense fees the Company is obligated to pay Penn on amounts the Company receives from third parties for the sublicensing of the licensed rights for the treatment of CLN2 disease. European Patent Office Proceeding In June 2017, a third party filed an opposition with the European Patent Office (EPO) challenging the validity of a European patent owned by Penn for the AAV8 vector, which the Company has exclusively licensed (EU AAV8 Patent). The EPO conducted oral proceedings in October 2018 and upheld the validity of the EU AAV8 Patent subject to certain amendments made during the proceeding. Each party to the proceeding has appealed the EPO’s ruling. As of June 30, 2019 and December 31, 2018, the Company had not recorded any liabilities related to this matter nor does the Company believe this matter will have a material adverse impact on its business. |
License and Royalty Revenue
License and Royalty Revenue | 6 Months Ended |
Jun. 30, 2019 | |
License Agreement Revenue Recognition [Abstract] | |
License and Royalty Revenue | 8. License and Royalty Revenue As of June 30, 2019, the Company’s NAV Technology Platform was being applied in the development of more than 20 partnered product candidates by nine NAV Technology Licensees. Consideration to the Company under its license agreements may include: (i) up-front and annual fees, (ii) option fees to acquire additional licenses, (iii) milestone payments based on the achievement of certain development and sales-based milestones by licensees, (iv) sublicense fees and (v) royalties on sales of licensed products. Sublicense fees vary by license and range from a mid-single digit percentage to a low-double digit percentage of license fees received by licensees as a result of sublicenses. Royalties on net sales of commercialized products vary by license and range from a mid-single digit percentage to a low double-digit percentage of net sales by licensees. Development milestone payments are only included in the transaction price of each license and recognized as revenue to the extent they are considered probable of achievement. Sales-based milestones are excluded from the transaction price of each license agreement and recognized as revenue in the period of achievement. As of June 30, 2019, the Company’s license agreements, excluding additional licenses that could be granted upon the exercise of options by licensees, contained unachieved milestones which could result in aggregate milestone payments to the Company of up to $18.6 million upon the commencement of various stages of clinical trials, $32.5 million upon the submission of regulatory approval filings, $81.0 million upon the approval of commercial products by regulatory agencies and $187.0 million upon the achievement of specified sales targets for licensed products. To the extent the milestone payments are realized by the Company, the Company will be obligated to pay sublicense fees to licensors based on a specified percentage of the fees earned by the Company. The achievement of milestones by licensees is highly dependent on the successful development and commercialization of licensed products and it is at least reasonably possible that some or all of the milestone fees will not be realized by the Company. The following tables present changes in the balances of the Company’s receivables, contract assets and contract liabilities during the periods presented (in thousands): Balance at Beginning Net Additions Balance at of Period (Deductions) End of Period Three Months Ended June 30, 2019 Receivables and contract assets: Accounts receivable, current and non-current $ 31,130 $ 2,504 $ 33,634 Contract assets $ 1,000 $ (1,000 ) $ — Contract liabilities: Deferred revenue, current and non-current $ 3,933 $ (600 ) $ 3,333 Balance at Beginning Net Additions Balance at of Period (Deductions) End of Period Six Months Ended June 30, 2019 Receivables and contract assets: Accounts receivable, current and non-current $ 31,599 $ 2,035 $ 33,634 Contract assets $ 750 $ (750 ) $ — Contract liabilities: Deferred revenue, current and non-current $ 3,933 $ (600 ) $ 3,333 Balance at Beginning Net Additions Balance at of Period (Deductions) End of Period Three Months Ended June 30, 2018 Receivables and contract assets: Accounts receivable, current and non-current $ 58,621 $ (53,397 ) $ 5,224 Contract assets $ 250 $ (250 ) $ — Contract liabilities: Deferred revenue, current and non-current $ — $ 600 $ 600 Balance at Beginning Net Additions Balance at of Period (Deductions) End of Period Six Months Ended June 30, 2018 Receivables and contract assets: Accounts receivable, current and non-current $ 5,850 $ (626 ) $ 5,224 Contract assets $ 350 $ (350 ) $ — Contract liabilities: Deferred revenue, current and non-current $ — $ 600 $ 600 The net increases in accounts receivable during the three and six months ended June 30, 2019 were primarily attributable to additional licenses granted by the Company during the periods as a result of license options exercised by licensees. The net decrease in accounts receivable during the three months ended June 30, 2018 was primarily attributable to the acceleration and collection of $100.0 million in license fees under our amended March 2014 license agreement with AveXis as a result of their acquisition by Novartis in May 2018. Of the $100.0 million received, $53.3 million was previously recorded as accounts receivable at the beginning of the period. As of June 30, 2019, the Company had recorded deferred revenue, current and non-current, of $3.3 million which represents consideration received from licensees for performance obligations that have not yet been satisfied by the Company. Unsatisfied performance obligations consist of options granted to licensees that provide material rights to the licensee to acquire additional licenses from the Company. These performance obligations will be satisfied, and underlying revenue will be recognized, upon the exercise or expiration of the options. During the three and six months ended June 30, 2019, the Company recognized $0.6 million of license revenue that was previously included in deferred revenue at the beginning of the period as a result of options exercised by licensees during the period. The Company did not recognize any license revenue during the three and six months ended June 30, 2018 that was included in deferred revenue at the beginning of the period. During the three and six months ended June 30, 2019, the Company recognized license revenue of $3.2 million and $4.0 million, respectively, from licenses delivered to licensees in prior periods as a result of changes in the transaction prices of its license agreements. Changes in the transaction prices during the three and six months ended June 30, 2019 were primarily attributable to development milestones achieved or deemed probable of achievement during the period that were previously not considered probable of achievement. During the three and six months ended June 30, 2018, the Company recognized license revenue of $39.9 million and $0.2 million, respectively, from licenses delivered to licensees in prior periods as a result of changes in the transaction prices of its license agreements. Changes in the transaction prices during the three and six months ended June 30 2018 were primarily attributable to development milestones achieved or deemed probable of achievement during the period that were previously not considered probable of achievement, as well as the acquisition of AveXis by Novartis in May 2018 resulting in the accelerated payment of $40.0 million of sales-based milestones which were previously excluded from the transaction price of the license. As of June 30, 2019, the Company had recorded total current and non-current accounts receivable of $33.6 million, of which $0.2 million had been billed to customers and $33.4 million was billable to customers in future periods. As of December 31, 2018, the Company had recorded total current and non-current accounts receivable of $31.6 million, of which $0.4 million had been billed to customers and $31.2 million was billable to customers in future periods. Accounts receivable, current and non-current, as of June 30, 2019 and December 31, 2018 included $27.3 million and $26.0 million, respectively, related to the November 2018 license agreement with Abeona Therapeutics Inc. for the development and commercialization of treatments for various diseases. The Company believes that it is not exposed to significant credit risk related to accounts receivable due to the credit quality and history of collections from its significant customers. The Company is unaware of any concentrations of credit risk related to accounts receivable from significant customers with deteriorated credit quality. As of June 30, 2019 and December 31, 2018, the Company had not recognized any impairment losses on its receivables or contract assets from contracts with customers and no allowance for doubtful accounts was recorded. AveXis March 2014 License and January 2018 Amendment In March 2014, the Company entered into an exclusive license agreement (the March 2014 License) with AveXis. Under the license, the Company granted AveXis an exclusive, worldwide commercial license, with rights to sublicense, to the NAV AAV9 vector for the treatment of SMA in humans by in vivo gene therapy. mid-single to low double-digit royalties on net sales of licensed products, In January 2018, the Company and AveXis amended the March 2014 License (the January 2018 Amendment). Under the January 2018 Amendment, the licensed intellectual property was expanded to include, in addition to the NAV AAV9 vector previously licensed, sublicenses to other third-party patents exclusively licensed by the Company as well as any other recombinant AAV vector in the Company’s intellectual property portfolio during a period of 14 years from the effective date of the January 2018 Amendment, for the treatment of SMA in humans by in vivo The January 2018 Amendment also modified the assignment provision of the March 2014 License. Under the amended assignment provision, AveXis was permitted to transfer the March 2014 License, as amended, without the Company’s consent in connection with a change of control of AveXis, subject to certain conditions. Under the original March 2014 License, any assignment by AveXis without the Company’s prior written consent had been prohibited. In consideration for the additional rights granted under the January 2018 Amendment, and in addition to any consideration owed under the original March 2014 License, AveXis paid to the Company a fee of $80.0 million upon entry into the January 2018 Amendment. In addition, AveXis was obligated to pay the Company (i) $30.0 million on the first anniversary of the effective date of the January 2018 Amendment, (ii) $30.0 million on the second anniversary of the effective date of the January 2018 Amendment and (iii) potential sales-based milestone payments of up to $120.0 million. In the event of a change of control of AveXis, to the extent that any fee described in (i) or (ii) above, or the first $40.0 million of sales-based milestone payments described in (iii) above, had not yet been paid to the Company, AveXis was required to pay any such unpaid fee to the Company upon the change of control. For any product developed for the treatment of SMA using the NAV AAV9 vector, AveXis will continue to be obligated to pay to the Company mid-single to low double-digit royalties on net sales as required by the March 2014 License, and for any product developed for the treatment of SMA using a licensed vector other than NAV AAV9, the Company will receive a low double-digit royalty on net sales. In May 2018, AveXis was acquired by Novartis, which qualified as a change of control of AveXis under the January 2018 Amendment. Pursuant to the January 2018 Amendment, AveXis paid the Company $100.0 million in accelerated license payments as a result of the change of control. In May 2019, the U.S. Food and Drug Administration (the FDA) approved Zolgensma for marketing in the United States, which is a licensed product under the March 2014 License, as amended, with AveXis. Upon its commercial launch in the second quarter of 2019, the Company began recognizing royalty revenue on net sales of Zolgensma. Accounting Analysis The January 2018 Amendment was accounted for under Topic 606 as a modification of the license agreement resulting in a new and separate contract from the original March 2014 License for revenue recognition purposes. The Company determined that a substantive termination penalty is associated with AveXis’ termination rights under the amended license agreement, and therefore the contract term for revenue recognition purposes is equal to the stated term of the license. The only material performance obligation of the Company under the January 2018 Amendment is for the delivery of the modified license, which occurred upon the execution of the amendment in January 2018. As of June 30, 2019, the transaction price of the original March 2014 License was $11.0 million. The transaction price includes (i) the up-front payment in March 2014 of $2.0 million, (ii) the present value of aggregate annual fees payable to the Company over the term of the license and (iii) payments for development milestones achieved to date or which are deemed probable of achievement. The discounted portion of the annual fees represents the financing benefit provided to AveXis and is recognized as interest income from licensing over the term of the license. Variable consideration under the original March 2014 License, which has been excluded from the transaction price, includes $3.5 million in payments for remaining development milestones that had not yet been achieved and were not considered probable of achievement, as well as any potential sublicense fees or royalties on sales of licensed products, which will be recognized in the period of the underlying sales or sublicenses. The transaction price of the original March 2014 License increased by $3.5 million during the three and six months ended June 30, 2019 as a result of development milestones achieved during the period which were previously excluded from the transaction price. Upon its execution, the transaction price of the January 2018 Amendment was $132.1 million, which was fully recognized as license revenue upon the delivery of the modified license in January 2018. In May 2018, as a result of the acquisition of AveXis by Novartis, the transaction price was increased by $40.0 million to account for the acceleration of the sale-based milestone which was previously excluded from the transaction price. The $40.0 million increase in the transaction price was recognized as license revenue upon the completion of the change of control in May 2018 since the amended license had been fully delivered to AveXis. Additionally, due to the acceleration of the two $30.0 million payments originally due in January 2019 and January 2020, the Company recognized $6.1 million of interest income from licensing upon the completion of the change of control of AveXis, which represents the remaining present value discount on such payments as of the date of the change of control. As of June 30, 2019, the transaction price of the January 2018 Amendment was $172.1 million, which includes: (i) the $80.0 million payment in January 2018, (ii) the present value, as of the date of the January 2018 Amendment, of the two $30.0 million payments originally due in January 2019 and January 2020 and (iii) the $40.0 million sales-based milestone which was accelerated upon the change of control in May 2018. Variable consideration under the January 2018 Amendment, which has been excluded from the transaction price, includes the remaining sales-based milestone payment of $80.0 million, as well as any potential sublicense fees or royalties on sales of licensed products, which will be recognized in the period of the underlying sales or sublicenses. There were no increases in the transaction price of the January 2018 Amendment during the three and six months ended June 30, 2019. The Company recognized the following amounts under the March 2014 License with AveXis, as amended, which include amounts from both the original March 2014 License and the January 2018 Amendment (in thousands): Three Months Ended June 30, Six Months Ended June 30, 2019 2018 2019 2018 License and royalty revenue $ 4,424 $ 40,000 $ 4,424 $ 172,066 Interest income from licensing $ 7 $ 6,752 $ 15 $ 7,950 As of June 30, 2019, the Company had recorded $1.1 million of accounts receivable from AveXis under the March 2014 License, as amended, of which $0.9 million were included in current assets and $0.2 million were included in non-current assets. As of December 31, 2018, the Company had recorded $0.2 million of accounts receivable from AveXis under the March 2014 License, as amended, of which less than $0.1 million were included in current assets and $0.2 million were included in non-current assets. |
Stock-based Compensation
Stock-based Compensation | 6 Months Ended |
Jun. 30, 2019 | |
Disclosure Of Compensation Related Costs Sharebased Payments [Abstract] | |
Stock-based Compensation | 9. Stock-based Compensation In January 2019, an additional 1,444,808 shares became available for issuance under the 2015 Equity Incentive Plan (the 2015 Plan). As of June 30, 2019, the total number of shares of common stock authorized for issuance under the 2015 Plan and the 2014 Stock Plan (the 2014 Plan) was 10,933,221, of which 2,327,843 remained available for future grants under the 2015 Plan. Stock-based Compensation Expense The Company’s stock-based compensation expense by award type was as follows (in thousands): Three Months Ended June 30, Six Months Ended June 30, 2019 2018 2019 2018 Stock options $ 6,844 $ 3,818 $ 12,296 $ 6,939 Restricted stock units 69 69 136 136 Employee stock purchase plan 186 95 385 197 $ 7,099 $ 3,982 $ 12,817 $ 7,272 As of June 30, 2019, the Company had $70.4 million of unrecognized stock-based compensation expense related to stock options, restricted stock units and the 2015 Employee Stock Purchase Plan (the 2015 ESPP), which is expected to be recognized over a weighted-average period of 3.0 years. The Company has recorded aggregate stock-based compensation expense in the consolidated statements of operations and comprehensive income (loss) as follows (in thousands): Three Months Ended June 30, Six Months Ended June 30, 2019 2018 2019 2018 Research and development $ 3,526 $ 1,895 $ 5,873 $ 3,424 General and administrative 3,573 2,087 6,944 3,848 $ 7,099 $ 3,982 $ 12,817 $ 7,272 Stock Options The following table summarizes stock option activity under the 2014 Plan and 2015 Plan (in thousands, except per share data): Weighted- average Weighted- Remaining average Contractual Aggregate Exercise Life Intrinsic Shares Price (Years) Value (a) Outstanding at December 31, 2018 4,855 $ 19.31 7.6 $ 118,185 Granted 1,410 $ 48.48 Exercised (621 ) $ 8.24 Cancelled or forfeited (190 ) $ 32.92 Outstanding at June 30, 2019 5,454 $ 27.64 7.8 $ 136,336 Exercisable at June 30, 2019 2,630 $ 13.65 6.7 $ 99,341 Vested and expected to vest at June 30, 2019 5,454 $ 27.64 7.8 $ 136,336 (a) The aggregate intrinsic value is calculated as the difference between the exercise price of the underlying options and the fair value of the common stock for the options that were in the money at the dates reported. The weighted-average grant date fair value per share of options granted during the six months ended June 30, 2019 was $32.24. During the six months ended June 30, 2019, the total number of stock options exercised was 621,415, resulting in total proceeds of $5.1 million. The total intrinsic value of options exercised during the six months ended June 30, 2019 was $25.5 million. Restricted Stock Units The following table summarizes restricted stock unit activity under the 2015 Plan (in thousands, except per share data): Weighted- average Grant Date Shares Fair Value Unvested balance at December 31, 2018 40 $ 20.90 Granted — $ — Vested — $ — Forfeited — $ — Unvested balance at June 30, 2019 40 $ 20.90 Employee Stock Purchase Plan As of June 30, 2019, the total number of shares of common stock authorized for issuance under the 2015 ESPP was 254,000, of which 159,339 remained available for future issuance. During the six months ended June 30, 2019, 10,241 shares of common stock were issued under the 2015 ESPP. |
Income Taxes
Income Taxes | 6 Months Ended |
Jun. 30, 2019 | |
Income Tax Disclosure [Abstract] | |
Income Taxes | 10. Income Taxes The Company has evaluated the positive and negative evidence bearing upon the realizability of its deferred tax assets. Based on the Company’s history of operating losses, including a three-year cumulative loss position as of June 30, 2019 and December 31, 2018, the Company has concluded that it is more likely than not that the benefit of its deferred tax assets will not be realized. Accordingly, the Company has provided a full valuation allowance for its net deferred tax assets as of June 30, 2019 and December 31, 2018. During the three and six months ended June 30, 2019, the Company recognized income tax benefit of $0.1 million and $0.2 million, respectively, and income tax expense in other comprehensive income of $0.3 million and $0.7 million, respectively, related to net unrealized gains on available-for-sale debt securities. As of June 30, 2019, the Company had accrued $0.5 million related to this tax benefit, which is expected to be generated from losses in continuing operations in 2019 and is included in accrued expenses and other current liabilities on the consolidated balance sheet. |
Related Party Transactions
Related Party Transactions | 6 Months Ended |
Jun. 30, 2019 | |
Related Party Transactions [Abstract] | |
Related Party Transactions | 11. Related Party Transactions FOXKISER LLP Since 2016, the Company has been party to professional services agreements with FOXKISER LLP (FOXKISER), an affiliate of certain stockholders of the Company and an affiliate of a member of the Company’s Board of Directors, pursuant to which the Company pays a fixed monthly fee in consideration for certain strategic services provided by FOXKISER. Effective January 2019, the Company entered into a new professional services agreement with FOXKISER with similar terms and conditions as the previous agreements. The agreement was amended effective June 2019 to expand the services provided and increase the monthly fee, and the amended agreement expires in December 2020. Expenses incurred under the agreements with FOXKISER for the three and six months ended June 30, 2019 were $0.9 million and $1.7 million, respectively. Expenses incurred under the agreements with FOXKISER for the three and six months ended June 30, 2018 were $0.5 million and $1.1 million, respectively. Expenses incurred under the agreements with FOXKISER are recorded as research and development expenses in the consolidated statements of operations and comprehensive income (loss). As of June 30, 2019, the Company had accrued $0.2 million in expenses payable to FOXKISER under the agreement, which are included in accrued expenses and other current liabilities on the consolidated balance sheet. |
Net Income (Loss) Per Share
Net Income (Loss) Per Share | 6 Months Ended |
Jun. 30, 2019 | |
Earnings Per Share [Abstract] | |
Net Income (Loss) Per Share | 12. Net Income (Loss) Per Share The computations of basic and diluted net income (loss) per share are as follows (in thousands, except per share data): Three Months Ended June 30, Six Months Ended June 30, 2019 2018 2019 2018 Basic net income (loss) per share: Net income (loss) applicable to common stockholders $ (1,457 ) $ 10,594 $ (33,685 ) $ 114,833 Shares used in computation: Weighted-average common shares outstanding 36,669 32,082 36,518 31,858 Basic net income (loss) per share $ (0.04 ) $ 0.33 $ (0.92 ) $ 3.60 Diluted net income (loss) per share: Net income (loss) applicable to common stockholders $ (1,457 ) $ 10,594 $ (33,685 ) $ 114,833 Shares used in computation: Weighted-average common shares outstanding 36,669 32,082 36,518 31,858 Stock options — 3,153 — 2,995 Restricted stock units — 30 — 27 Employee stock purchase plan — 7 — 4 Weighted-average diluted common shares 36,669 35,272 36,518 34,884 Diluted net income (loss) per share $ (0.04 ) $ 0.30 $ (0.92 ) $ 3.29 For periods in which the Company incurred net losses applicable to common stockholders, common stock equivalents are excluded from the calculation of diluted net loss per share as their effect would be anti-dilutive, and accordingly, basic and diluted net loss per share are the same for such periods. Outstanding stock options with exercise prices greater than the average market price of common stock are excluded from the calculation of diluted net income (loss) per share as their effect would be anti-dilutive. The following potentially dilutive common stock equivalents outstanding at the end of the period were excluded from the computations of weighted-average diluted common shares for the periods indicated as their effects would be anti-dilutive (in thousands): Three Months Ended June 30, Six Months Ended June 30, 2019 2018 2019 2018 Stock options issued and outstanding 5,454 804 5,454 1,236 Unvested restricted stock units outstanding 40 — 40 — Employee stock purchase plan 26 — 26 — 5,520 804 5,520 1,236 |
Supplemental Disclosures
Supplemental Disclosures | 6 Months Ended |
Jun. 30, 2019 | |
Payables And Accruals [Abstract] | |
Supplemental Disclosures | 13. Supplemental Disclosures Accrued expenses and other current liabilities consists of the following (in thousands): June 30, 2019 December 31, 2018 Accrued personnel costs $ 6,692 $ 9,484 Accrued external research and development expenses 4,925 4,274 Accrued sublicense fees and royalties 1,514 1,617 Accrued external general and administrative expenses 1,498 773 Accrued income taxes payable 564 726 Accrued purchases of property and equipment 397 221 Other accrued expenses and current liabilities 944 69 $ 16,534 $ 17,164 Other liabilities of $1.9 million and $2.5 million reported as of June 30, 2019 and December 31, 2018, respectively, consist of accrued sublicense fees payable to licensors in periods beyond 12 months from the reporting date. |
Summary of Significant Accoun_2
Summary of Significant Accounting Policies (Policies) | 6 Months Ended |
Jun. 30, 2019 | |
Accounting Policies [Abstract] | |
Basis of Presentation and Principles of Consolidation | Basis of Presentation and Principles of Consolidation The accompanying consolidated financial statements are unaudited and have been prepared in conformity with accounting principles generally accepted in the United States of America (GAAP). The interim unaudited consolidated financial statements have been prepared on the same basis as the annual audited consolidated financial statements as of and for the year ended December 31, 2018 included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2018, which was filed with the SEC on February 27, 2019. Certain information and footnote disclosures required by GAAP which are normally included in the Company’s annual consolidated financial statements have been omitted pursuant to SEC rules and regulations for interim reporting. In the opinion of management, the accompanying consolidated financial statements reflect all adjustments, which are normal and recurring in nature, necessary for fair financial statement presentation. The results of operations for the interim periods are not necessarily indicative of the results of operations to be expected for the full year, any other interim periods, or any future year or period. These interim consolidated financial statements should be read in conjunction with the audited consolidated financial statements as of and for the year ended December 31, 2018, and the notes thereto, which are included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2018. The accompanying consolidated financial statements include the accounts of the Company and its wholly owned subsidiaries. All intercompany balances and transactions have been eliminated in consolidation. |
Use of Estimates | Use of Estimates The preparation of financial statements in conformity with GAAP requires management to make estimates and assumptions that affect the reported amounts in the financial statements and accompanying notes. Actual results could differ materially from those estimates. Management considers many factors in selecting appropriate financial accounting policies and controls, and in developing the estimates and assumptions that are used in the preparation of these consolidated financial statements. Management must apply significant judgment in this process. In addition, other factors may affect estimates, including: expected business and operational changes, sensitivity and volatility associated with the assumptions used in developing estimates and whether historical trends are expected to be representative of future trends. The estimation process often may yield a range of potentially reasonable estimates of the ultimate future outcomes and management must select an amount that falls within that range of reasonable estimates. This process may result in actual results differing materially from those estimated amounts used in the preparation of the consolidated financial statements. Significant estimates are used in the following areas, among others: license and royalty revenue, stock-based compensation expense, accrued research and development expenses and other accrued liabilities, income taxes and the fair value of financial instruments. |
Reclassifications | Reclassifications Certain amounts reported in prior periods have been reclassified to conform to current period financial statement presentation. These reclassifications are not material and have no effect on previously reported financial position, results of operations and cash flows. |
Restricted Cash | Restricted Cash Restricted cash includes money market mutual funds used to collateralize irrevocable letters of credit as required by the Company’s lease agreements. The following table provides a reconciliation of cash and cash equivalents and restricted cash as reported on the consolidated balance sheets to the total of these amounts as reported at the end of the period in the consolidated statements of cash flows (in thousands): June 30, 2019 June 30, 2018 Cash and cash equivalents $ 55,142 $ 106,889 Restricted cash 1,053 225 Total cash and cash equivalents and restricted cash $ 56,195 $ 107,114 |
Marketable Securities | Marketable Securities Marketable securities consist of available-for-sale debt securities and equity securities and are carried at fair value. Marketable debt securities with remaining maturity dates exceeding 12 months which are not intended to be sold prior to maturity for use in current operations are classified as non-current assets. Marketable equity securities are classified as current assets. Unrealized gains and losses on available-for-sale debt securities, net of any related tax effects, are excluded from results of operations and are included in other comprehensive income (loss) and reported as a separate component of stockholders’ equity until realized. The Company uses the aggregate portfolio approach to release the tax effects of unrealized gains and losses on available-for-sale debt securities in accumulated other comprehensive income (loss). Purchase premiums and discounts on marketable debt securities are amortized or accreted into the cost basis over the life of the related security as adjustments to the yield using the effective-interest method. Interest income is recognized when earned. Unrealized gains and losses on marketable equity securities are included in results of operations as investment income. Realized gains and losses from the sale or maturity of marketable securities are based on the specific identification method and are included in results of operations as investment income. A decline in the fair value below cost of available-for-sale debt securities that is deemed other-than-temporary is charged to results of operations, resulting in the establishment of a new cost basis for the security. The Company regularly evaluates whether declines in the fair value of its debt securities below their cost are other-than-temporary. The evaluation includes consideration of the cause of the impairment, including the creditworthiness of the security issuers, the number of securities in an unrealized loss position, the severity and duration of the unrealized losses, whether the Company has the intent to sell the securities and whether it is more likely than not that the Company will be required to sell the securities before the recovery of their amortized cost basis. The Company has not recorded any impairment of available-for-sale debt securities which was deemed to be to be other-than-temporary. |
Leases | Leases Effective January 1, 2019, the Company adopted Accounting Standards Update (ASU) 2016-02, Leases Leases Under Topic 842, the Company classifies its leases as either finance or operating leases based on the principle of whether or not the lease is effectively a financed purchase by the Company. Lease classification is evaluated at the inception of the lease agreement. Regardless of classification, the Company records a right-of-use asset and a lease liability for all leases with a term greater than 12 months. All of the Company’s leases as of June 30, 2019 have been classified as operating leases. Operating lease expense is recognized on a straight-line basis over the term of the lease, with the exception of variable lease expenses which are recognized as incurred. The Company identifies leases in its contracts if the contract conveys the right to control the use of identified property, plant or equipment for a period of time in exchange for consideration. The Company does not allocate lease consideration between lease and nonlease components and records a lease liability equal to the present value of the remaining fixed consideration under the lease. The interest rates implicit in the Company’s leases are generally not readily determinable. Accordingly, the Company uses its estimated incremental borrowing rate at the commencement date of the lease to determine the present value discount of the lease liability. The Company estimates its incremental borrowing rate for each lease based on an evaluation of its expected credit rating and the prevailing market rates for collateralized debt in a similar economic environment with similar payment terms and maturity dates commensurate with the term of the lease. The right-of-use asset for each lease is equal to the lease liability, adjusted for unamortized initial direct costs and lease incentives and prepaid or accrued rent. Initial direct costs of entering into a lease are included in the right-of-use asset and amortized as lease expense over the term of the lease. Lease incentives, such as tenant improvements allowances, are recorded as a reduction of the right-of-use asset and amortized as a reduction of lease expense over the term of the lease. The Company excludes options to extend or terminate leases from the calculation of the lease liability unless it is reasonably certain the option will be exercised. |
Fair Value of Financial Instruments | Fair Value of Financial Instruments 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. ASC 820, Fair Value Measurements and Disclosures • Level 1—Valuations based on unadjusted quoted prices in active markets for identical assets or liabilities that the Company has the ability to access at the measurement date. • Level 2—Valuations based on quoted prices for similar assets or liabilities in markets that are not active or for which all significant inputs are observable, either directly or indirectly. • Level 3—Valuations that require inputs that reflect the Company’s own assumptions that are both significant to the fair value measurement and unobservable. To the extent that valuation is based on models or inputs that are less observable or unobservable in the market, the determination of fair value requires more judgment. Accordingly, the degree of judgment exercised by the Company in determining fair value is greatest for instruments categorized in Level 3. A financial instrument’s level within the fair value hierarchy is based on the lowest level of any input that is significant to the fair value measurement. The fair values of the Company’s Level 2 instruments are based on quoted market prices or broker or dealer quotations for similar assets. These investments are initially valued at the transaction price and subsequently valued utilizing third party pricing providers or other market observable data. Please refer to Note 4 for further information on the fair value measurement of the Company’s financial instruments. |
Revenue Recognition | Revenue Recognition The Company recognizes revenue in accordance with ASC 606, Revenue from Contracts with Customers The Company applies the five-step model to contracts that are within the scope of Topic 606 only when it is probable that the Company will collect the consideration it is entitled to in exchange for the goods or services it transfers to the customer. At contract inception, for contracts within the scope of Topic 606, the Company assesses the goods or services promised within each contract and determine those that are performance obligations and whether each promised good or service is distinct. The Company then recognizes as revenue the amount of the transaction price that is allocated to respective performance obligations when (or as) the respective performance obligations are satisfied. The Company evaluates its contracts with customers for the presence of significant financing components. If a significant financing component is identified in a contract and provides a financing benefit to the customer, the transaction price for the contract is adjusted to account for the financing portion of the arrangement, which is recognized as interest income over the financing term using the effective interest method. In determining the appropriate interest rates for significant financing components, the Company evaluates the credit profile of the customer and prevailing market interest rates and selects an interest rate in which it believes would be charged to the customer in a separate financing arrangement over a similar financing term. License and Royalty Revenue The Company licenses its NAV Technology Platform to other biotechnology and pharmaceutical companies. The terms of the licenses vary, and licenses may be exclusive or non-exclusive and may be sublicensable by the licensee. Licenses may grant intellectual property rights for purposes of internal and preclinical research and development only, or may include the rights, or options to obtain future rights, to commercialize drug therapies for specific diseases using the Company’s NAV Technology Platform. License agreements generally have a term at least equal to the life of the underlying patents, but are terminable at the option of the licensee. Consideration payable to the Company under its license agreements may include: (i) up-front and annual fees, (ii) option fees to acquire additional licenses, (iii) milestone payments based on the achievement of certain development and sales-based milestones by licensees, (iv) sublicense fees and (v) royalties on sales of licensed products. The Company’s license agreements are accounted for as contracts with customers within the scope of Topic 606. At the inception of each license agreement, the Company determines the contract term for purposes of applying the requirements of Topic 606. Licenses are generally terminable at the option of the licensee with advance notice to the Company. For each license, the Company evaluates these termination rights to determine whether a substantive termination penalty would be incurred by the licensee upon termination. If the licensee incurs a substantive termination penalty upon termination, the contract term for revenue recognition purposes is generally equal to the stated term of the license, which is the life of the underlying licensed patents. Alternatively, if the licensee does not incur a substantive termination penalty upon termination, the contract term for revenue recognition purposes may be shorter than the stated term of the license, in which case the termination rights may be accounted for as contract renewal options. The determination of whether a substantive termination penalty is associated with the termination rights requires significant judgment. In making this determination, the Company considers, among other things, the nature of the intellectual property rights that would be returned to the Company upon termination, including the exclusivity of the licensed rights and the stage of development of the licensed products, the payment terms, including the amount and timing of non-refundable or guaranteed payments, and the business purpose of the termination rights granted to the licensee. The Company considers all of the facts and circumstances relevant to each license when making this determination. Performance obligations under the Company’s license agreements may include (i) the delivery of intellectual property licenses and (ii) options granted to licensees to acquire additional licenses to the extent the options represent material rights to the licensee. At the inception of each license agreement which contains options for the licensee to acquire additional licenses, or contract renewal options, the Company evaluates the options to determine whether they provide material rights to the licensee. In making this determination, the Company considers whether the options are priced at a discount to the standalone selling price for the underlying licenses. If an option is priced at a discount to the standalone selling price for the underlying license, the option is considered to be a material right to the licensee and is accounted for as a separate performance obligation under the current license agreement. The Company evaluates the transaction price of its license agreements at the inception of each agreement and at each reporting date. The transaction price includes the fixed consideration payable to the Company during the contract term, as well as any variable consideration to the extent that it is probable that a significant reversal of revenue will not occur in the future. Fixed consideration under the license agreements includes up-front and annual fees payable during the contract term. Variable consideration under the license agreements includes development and sales-based milestone payments, sublicense fees and royalties on sales of licensed products. Consideration contingent upon the exercise of options by a licensee is excluded from the transaction price and not accounted for as part of the license agreement until the option is exercised. The transaction price for each license agreement is allocated to the underlying performance obligations and recognized as revenue when the performance obligations are satisfied. Consideration allocated to performance obligations for the delivery of an intellectual property license is recognized as revenue in full upon the delivery of the license to the licensee. Consideration allocated to performance obligations for license options is recognized as revenue in full upon the earlier of the option exercise or expiration. The exercise of a license option by a licensee is accounted for as a new license for revenue recognition purposes. Up-front and annual licenses fees payable to the Company over the contract term of each license are included in the transaction price, and the portion of this consideration that is allocated to the performance obligation for the delivery of the intellectual property license is recognized as revenue in full upon the delivery of the license to the licensee. If annual license fees are payable to the Company in periods beyond 12 months from the delivery of the license, a significant financing component is deemed to exist which provides a financing benefit to the licensee. If a significant financing component is identified, the Company adjusts the transaction price for the license to include only the present value of the annual license fees payable to the Company over the contract term. The discounted portion of the license fees is recognized as interest income from licensing over the financing period of the license. Development milestone payments are payable to the Company upon the achievement of specified development milestones by licensees. At the inception of each license agreement that contains development milestone payments, the Company evaluates whether the milestones are considered probable of achievement and estimates the amount to be included in the transaction price using the most likely amount method. If it is probable that a significant revenue reversal will not occur in the future, milestone payments are included in the transaction price and recognized as revenue upon the delivery of the license. Milestone payments contingent on the achievement of development milestones that are not within the control of the Company or the licensee, such as regulatory approvals, are not considered probable of being achieved and are excluded from the transaction price until the milestone is achieved. At each reporting date, the Company re-evaluates the probability of achievement of outstanding development milestones and, if necessary, adjusts the transaction price for any milestones for which the probability of achievement has changed due to current facts and circumstances. Any such adjustments are recorded on a cumulative catch-up basis and recognized as revenue in the period of the adjustment. Royalties on sales of licensed products, sales-based milestone payments and sublicense fees based on the receipt of certain fees by licensees from any sublicensees are excluded from the transaction price of each license and recognized as revenue in the period that the related sales or sublicenses occur, provided that the associated license has been delivered to the licensee. Royalty revenue to date consists of royalties on net sales of Zolgensma®, which is marketed by AveXis, Inc. (AveXis), a wholly owned subsidiary of Novartis AG (Novartis). Zolgensma is a licensed product under the Company’s March 2014 license agreement, as amended, with AveXis for the development and commercialization of treatments for spinal muscular atrophy (SMA). The Company recognizes royalty revenue from net sales of Zolgensma in the period in which the underlying products are sold by AveXis, which in certain cases may require the Company to estimate royalty revenue for periods of net sales which have not yet been reported to the Company. Sales-based milestone payments related to net sales of Zolgensma are recognized as royalty revenue in the period in which the milestone is achieved. The Company receives payments from licensees based on the billing schedules established in each license agreement. Amounts recognized as revenue which have not yet been received from licensees, including unbilled royalties, are recorded as accounts receivable when the Company’s rights to the consideration are conditional solely upon the passage of time. Amounts recognized as revenue which have not yet been received from licensees are recorded as contract assets when the Company’s rights to the consideration are not unconditional. Contract assets are recorded as other current assets on the consolidated balance sheets. If a licensee elects to terminate a license prior to the end of the license term, the licensed intellectual property is returned to the Company and any consideration recorded as accounts receivable or contract assets which is not contractually payable by the licensee is charged off as a reduction of license revenue in the period of the termination. Amounts received by the Company prior to the delivery of underlying performance obligations are deferred and recognized as revenue upon the satisfaction of the performance obligations by the Company. Deferred revenue which is not expected to be recognized within 12 months from the reporting date is recorded as non-current on the consolidated balance sheets. |
Cost of Revenues | Cost of Revenues Cost of revenues consists primarily of sublicense fees, milestone payments and royalties on net sales of licensed products as specified in the Company’s agreements with its licensors. Sublicense fees are based on a percentage of license fees received by the Company from NAV Technology Licensees and are recognized in the period that the underlying license revenue is recognized. Milestone payments are payable to licensors upon the achievement of specified milestones by NAV Technology Licensees and are recognized in the period the milestone is achieved or deemed probable of achievement. Royalties are based on a percentage of net sales of licensed products by NAV Technology Licensees and are recognized in the period that the underlying sales occur. Amounts which are payable to licensors in periods beyond 12 months from the reporting date are recorded as non-current liabilities on the consolidated balance sheets. |
Net Income (Loss) Per Share | Net Income (Loss) Per Share Basic net income (loss) per share is calculated by dividing net income (loss) applicable to common stockholders by the weighted-average common shares outstanding during the period, without consideration for common stock equivalents. Diluted net income (loss) per share is calculated by adjusting the weighted-average common shares outstanding for the dilutive effect of common stock equivalents outstanding for the period, determined using the treasury-stock method. Contingently convertible shares in which conversion is based on non-market-priced contingencies are excluded from the calculations of both basic and diluted net income (loss) per share until the contingency has been fully met. For purposes of the diluted net income (loss) per share calculation, common stock equivalents are excluded from the calculation of diluted net income (loss) per share if their effect would be anti-dilutive. |
Recent Accounting Pronouncements | Recent Accounting Pronouncements Adoption of ASU 2016-02, Leases In February 2016, the Financial Accounting Standards Board (FASB) issued ASU 2016-02, Leases Leases The Company elected certain practical expedients allowed by Topic 842 for transition purposes, including the package of practical expedients which permitted the Company to not reassess lease identification, classification and initial direct costs under Topic 842 for leases that commenced prior to January 1, 2019. Additionally, the Company elected the practical expedient allowed for transition purposes to use hindsight in determining the terms of leases that commenced prior to January 1, 2019. Upon the adoption of Topic 842, the Company recorded operating lease right-of-use assets of $7.4 million and operating lease liabilities of $8.4 million for its leases which were in effect and had commenced prior to January 1, 2019 and had original lease terms of more than 12 months. The Company also derecognized current and non-current deferred rent liabilities of $1.4 million and prepaid expenses, other current assets and other assets of $0.4 million upon the adoption of Topic 842. Additionally, upon the adoption of Topic 842, the Company derecognized $5.9 million of property and equipment and $5.9 million of financing lease obligations related to construction-in-progress at 9800 Medical Center Drive, as the Company does not control the building during the construction period under the requirements of Topic 842. The lease term for the facility at 9800 Medical Center Drive does not commence until certain construction is completed by the landlord and the building is delivered to the Company. The right-of-use assets and lease liabilities related to the facility at 9800 Medical Center Drive will not be recognized on the Company’s consolidated balance sheets until the commencement date of the lease, which is expected to occur in 2020. The cumulative impact of the adoption of Topic 842 resulted in an increase in accumulated deficit of less than $0.1 million on January 1, 2019. The adoption of Topic 842 did not have a material impact on the Company’s results of operations for the three and six months ended June 30, 2019, nor does the Company believe it will have a material impact on future results of operations based on its current leasing arrangements. Other R ecently A dopted A ccounting P ronouncements In February 2018, the FASB issued ASU 2018-02, Income Statement—Reporting Comprehensive Income (Topic 220): Reclassification of Certain Tax Effects from Accumulated Other Comprehensive Income Tax Cuts and Jobs Act of 2017 (the TCJA) In April 2017, the FASB issued ASU 2017-08, Receivables—Nonrefundable Fees and Other Costs (Subtopic 310-20) Recent Accounting Pronouncements Not Yet Adopted In August 2018, the FASB issued ASU 2018-15, Intangibles—Goodwill and Other—Internal-Use Software (Subtopic 350-40): Customer’s Accounting for Implementation Costs Incurred in a Cloud Computing Arrangement That Is a Service Contract In August 2018, the FASB issued ASU 2018-13, Fair Value Measurement (Topic 820): Disclosure Framework—Changes to the Disclosure Requirements for Fair Value Measurement In June 2016, the FASB issued ASU 2016-13, Financial Instruments—Credit Losses (Topic 326): Measurement of Credit Losses on Financial Instruments |
Summary of Significant Accoun_3
Summary of Significant Accounting Policies (Tables) | 6 Months Ended |
Jun. 30, 2019 | |
Accounting Policies [Abstract] | |
Summary of Reconciliation of Cash and Cash Equivalents and Restricted Cash | The following table provides a reconciliation of cash and cash equivalents and restricted cash as reported on the consolidated balance sheets to the total of these amounts as reported at the end of the period in the consolidated statements of cash flows (in thousands): June 30, 2019 June 30, 2018 Cash and cash equivalents $ 55,142 $ 106,889 Restricted cash 1,053 225 Total cash and cash equivalents and restricted cash $ 56,195 $ 107,114 |
Marketable Securities (Tables)
Marketable Securities (Tables) | 6 Months Ended |
Jun. 30, 2019 | |
Investments Debt And Equity Securities [Abstract] | |
Summary of Company Marketable Securities | The following tables present a summary of the Company’s marketable securities, which consist of available-for-sale debt securities and equity securities (in thousands): Amortized Cost Unrealized Gains Unrealized Losses Fair Value June 30, 2019 U.S. government and federal agency securities $ 102,825 $ 388 $ — $ 103,213 Certificates of deposit 8,748 67 — 8,815 Corporate bonds 249,377 1,070 (3 ) 250,444 Equity securities 420 31,656 — 32,076 $ 361,370 $ 33,181 $ (3 ) $ 394,548 Amortized Cost Unrealized Gains Unrealized Losses Fair Value December 31, 2018 U.S. government and federal agency securities $ 103,410 $ 93 $ (37 ) $ 103,466 Certificates of deposit 8,992 — — 8,992 Corporate bonds 282,902 36 (377 ) 282,561 $ 395,304 $ 129 $ (414 ) $ 395,019 |
Summary of Fair Values and Unrealized Losses of Marketable Securities Held by the Company in an Unrealized Loss Position for Less Than 12 months and 12 Months or Greater | The following tables present the fair values and unrealized losses of available-for-sale debt securities held by the Company in an unrealized loss position for less than 12 months and 12 months or greater (in thousands): Less than 12 Months 12 Months or Greater Total Fair Value Unrealized Losses Fair Value Unrealized Losses Fair Value Unrealized Losses June 30, 2019 Corporate bonds $ 19,176 $ (3 ) $ — $ — $ 19,176 $ (3 ) $ 19,176 $ (3 ) $ — $ — $ 19,176 $ (3 ) Less than 12 Months 12 Months or Greater Total Fair Value Unrealized Losses Fair Value Unrealized Losses Fair Value Unrealized Losses December 31, 2018 U.S. government and federal agency securities $ 53,124 $ (37 ) $ — $ — $ 53,124 $ (37 ) Corporate bonds 245,283 (354 ) 12,424 (23 ) 257,707 (377 ) $ 298,407 $ (391 ) $ 12,424 $ (23 ) $ 310,831 $ (414 ) |
Fair Value of Financial Instr_2
Fair Value of Financial Instruments (Tables) | 6 Months Ended |
Jun. 30, 2019 | |
Fair Value Disclosures [Abstract] | |
Schedule of Fair Value of Cash Equivalents and Marketable Securities | The following tables present the fair value of cash equivalents and marketable securities in accordance with the hierarchy discussed in Note 2 (in thousands): Quoted Significant prices other Significant in active observable unobservable markets inputs inputs (Level 1) (Level 2) (Level 3) Total June 30, 2019 Cash equivalents: Money market mutual funds $ — $ 55,126 $ — $ 55,126 Total cash equivalents — 55,126 — 55,126 Marketable securities: U.S. government and federal agency securities — 103,213 — 103,213 Certificates of deposit — 8,815 — 8,815 Corporate bonds — 250,444 — 250,444 Equity securities 32,076 — — 32,076 Total marketable securities 32,076 362,472 — 394,548 Total cash equivalents and marketable securities $ 32,076 $ 417,598 $ — $ 449,674 Quoted Significant prices other Significant in active observable unobservable markets inputs inputs (Level 1) (Level 2) (Level 3) Total December 31, 2018 Cash equivalents: Money market mutual funds $ — $ 75,542 $ — $ 75,542 Total cash equivalents — 75,542 — 75,542 Marketable securities: U.S. government and federal agency securities — 103,466 — 103,466 Certificates of deposit — 8,992 — 8,992 Corporate bonds — 282,561 — 282,561 Total marketable securities — 395,019 — 395,019 Total cash equivalents and marketable securities $ — $ 470,561 $ — $ 470,561 |
Property and Equipment, Net (Ta
Property and Equipment, Net (Tables) | 6 Months Ended |
Jun. 30, 2019 | |
Property Plant And Equipment [Abstract] | |
Schedule of Property and Equipment | Property and equipment, net consists of the following (in thousands): June 30, 2019 December 31, 2018 Lab equipment $ 16,533 $ 14,417 Computer equipment and software 2,342 2,002 Furniture and fixtures 1,946 1,915 Leasehold improvements 17,092 11,751 Construction-in-progress — 5,854 Total property and equipment 37,913 35,939 Accumulated depreciation and amortization (10,583 ) (7,237 ) Property and equipment, net $ 27,330 $ 28,702 |
Leases (Tables)
Leases (Tables) | 6 Months Ended |
Jun. 30, 2019 | |
Leases [Abstract] | |
Summary of Lease Costs and Supplemental Cash Flow Information of Operating Leases | The following table summarizes the Company’s lease costs and supplemental cash flow information related to operating leases (in thousands): Three Months Ended Six Months Ended June 30, 2019 June 30, 2019 Operating lease cost $ 697 $ 1,398 Variable lease cost 138 298 Total lease cost $ 835 $ 1,696 Cash paid for amounts included in operating lease liabilities $ 684 $ 1,395 Right-of-use assets acquired through operating lease liabilities $ (371 ) $ (335 ) |
Weighted-Average Remaining Lease Term and Weighted-Average Discount Rate of Operating Leases | The weighted-average remaining lease term and weighted-average discount rate of the Company’s operating leases were as follows: As of June 30, 2019 Weighted-average remaining lease term (years) 2.9 Weighted-average discount rate 5.6 % |
Reconciliation of Undiscounted Future Minimum Lease Payments Remaining Under Leases that Have Not Yet Commenced and Other Operating Leases | The following table presents a reconciliation of the undiscounted future minimum lease payments remaining under leases that have not yet commenced and other operating leases to the amounts reported as operating lease liabilities on the consolidated balance sheet as of June 30, 2019 (in thousands): Leases Other Total Not Yet Operating Minimum Lease Commenced (a) Leases Payments Undiscounted future minimum lease payments: 2019 (remainder of year) $ 98 $ 1,200 $ 1,298 2020 460 2,813 3,273 2021 2,308 2,412 4,720 2022 5,422 623 6,045 2023 6,376 479 6,855 Thereafter 83,650 — 83,650 Total undiscounted future minimum lease payments $ 98,314 $ 7,527 $ 105,841 Amount representing imputed interest (597 ) Total operating lease liabilities 6,930 Current portion of operating lease liabilities (2,276 ) Operating lease liabilities, non-current $ 4,654 (a) Includes undiscounted future minimum lease payments under the 9800 Medical Center Drive Lease and 400 Madison Lease which are not included in the lease liabilities reported on the consolidated balance sheet as of June 30, 2019. The actual timing and amounts of these payments are subject to adjustment based on the commencement dates and actual square footage of the leased premises. Accordingly, these amounts were estimates as of June 30, 2019. |
Schedule of Future Minimum Lease Payments Under Topic 840 for 9800 Medical Center Drive Lease and Other Operating Leases | As of December 31, 2018, future minimum lease payments under Topic 840 for the 9800 Medical Center Drive Lease and other operating leases were as follows (in thousands): 9800 Medical Other Total Center Drive Operating Minimum Lease Lease (a) Leases Payments 2019 $ — $ 2,798 $ 2,798 2020 — 3,054 3,054 2021 1,329 2,391 3,720 2022 4,289 621 4,910 2023 5,156 479 5,635 Thereafter 76,420 — 76,420 Total minimum lease payments $ 87,194 $ 9,343 $ 96,537 (a) Includes all future minimum lease payments under the 9800 Medical Center Drive Lease, including amounts recorded as financing lease obligations on the consolidated balance sheet . The actual timing and amounts of payments under the 9800 Medical Center Drive Lease are subject to adjustment based on the commencement date and actual square footage of the leased premises. Accordingly, these amounts were estimates as of December 31, 2018. |
License and Royalty Revenue (Ta
License and Royalty Revenue (Tables) | 6 Months Ended |
Jun. 30, 2019 | |
License Agreement Revenue Recognition [Abstract] | |
Summary of Changes in Balances of Receivables, Contract Assets and Contract Liabilities | The following tables present changes in the balances of the Company’s receivables, contract assets and contract liabilities during the periods presented (in thousands): Balance at Beginning Net Additions Balance at of Period (Deductions) End of Period Three Months Ended June 30, 2019 Receivables and contract assets: Accounts receivable, current and non-current $ 31,130 $ 2,504 $ 33,634 Contract assets $ 1,000 $ (1,000 ) $ — Contract liabilities: Deferred revenue, current and non-current $ 3,933 $ (600 ) $ 3,333 Balance at Beginning Net Additions Balance at of Period (Deductions) End of Period Six Months Ended June 30, 2019 Receivables and contract assets: Accounts receivable, current and non-current $ 31,599 $ 2,035 $ 33,634 Contract assets $ 750 $ (750 ) $ — Contract liabilities: Deferred revenue, current and non-current $ 3,933 $ (600 ) $ 3,333 Balance at Beginning Net Additions Balance at of Period (Deductions) End of Period Three Months Ended June 30, 2018 Receivables and contract assets: Accounts receivable, current and non-current $ 58,621 $ (53,397 ) $ 5,224 Contract assets $ 250 $ (250 ) $ — Contract liabilities: Deferred revenue, current and non-current $ — $ 600 $ 600 Balance at Beginning Net Additions Balance at of Period (Deductions) End of Period Six Months Ended June 30, 2018 Receivables and contract assets: Accounts receivable, current and non-current $ 5,850 $ (626 ) $ 5,224 Contract assets $ 350 $ (350 ) $ — Contract liabilities: Deferred revenue, current and non-current $ — $ 600 $ 600 |
Schedule of License Revenue | The Company recognized the following amounts under the March 2014 License with AveXis, as amended, which include amounts from both the original March 2014 License and the January 2018 Amendment (in thousands): Three Months Ended June 30, Six Months Ended June 30, 2019 2018 2019 2018 License and royalty revenue $ 4,424 $ 40,000 $ 4,424 $ 172,066 Interest income from licensing $ 7 $ 6,752 $ 15 $ 7,950 |
Stock-based Compensation (Table
Stock-based Compensation (Tables) | 6 Months Ended |
Jun. 30, 2019 | |
Stock-Based Compensation Expense by Award Type | The Company’s stock-based compensation expense by award type was as follows (in thousands): Three Months Ended June 30, Six Months Ended June 30, 2019 2018 2019 2018 Stock options $ 6,844 $ 3,818 $ 12,296 $ 6,939 Restricted stock units 69 69 136 136 Employee stock purchase plan 186 95 385 197 $ 7,099 $ 3,982 $ 12,817 $ 7,272 |
Stock-Based Compensation Expense | The Company has recorded aggregate stock-based compensation expense in the consolidated statements of operations and comprehensive income (loss) as follows (in thousands): Three Months Ended June 30, Six Months Ended June 30, 2019 2018 2019 2018 Research and development $ 3,526 $ 1,895 $ 5,873 $ 3,424 General and administrative 3,573 2,087 6,944 3,848 $ 7,099 $ 3,982 $ 12,817 $ 7,272 |
Summary of Unvested RSUs Activity Under 2015 Plan | The following table summarizes restricted stock unit activity under the 2015 Plan (in thousands, except per share data): Weighted- average Grant Date Shares Fair Value Unvested balance at December 31, 2018 40 $ 20.90 Granted — $ — Vested — $ — Forfeited — $ — Unvested balance at June 30, 2019 40 $ 20.90 |
2014 and 2015 Equity Incentive Plan [Member] | |
Summary of Stock Option Activity | The following table summarizes stock option activity under the 2014 Plan and 2015 Plan (in thousands, except per share data): Weighted- average Weighted- Remaining average Contractual Aggregate Exercise Life Intrinsic Shares Price (Years) Value (a) Outstanding at December 31, 2018 4,855 $ 19.31 7.6 $ 118,185 Granted 1,410 $ 48.48 Exercised (621 ) $ 8.24 Cancelled or forfeited (190 ) $ 32.92 Outstanding at June 30, 2019 5,454 $ 27.64 7.8 $ 136,336 Exercisable at June 30, 2019 2,630 $ 13.65 6.7 $ 99,341 Vested and expected to vest at June 30, 2019 5,454 $ 27.64 7.8 $ 136,336 (a) The aggregate intrinsic value is calculated as the difference between the exercise price of the underlying options and the fair value of the common stock for the options that were in the money at the dates reported. |
Net Income (Loss) Per Share (Ta
Net Income (Loss) Per Share (Tables) | 6 Months Ended |
Jun. 30, 2019 | |
Earnings Per Share [Abstract] | |
Computations of Basic and Diluted Net Income (Loss) Per Share | The computations of basic and diluted net income (loss) per share are as follows (in thousands, except per share data): Three Months Ended June 30, Six Months Ended June 30, 2019 2018 2019 2018 Basic net income (loss) per share: Net income (loss) applicable to common stockholders $ (1,457 ) $ 10,594 $ (33,685 ) $ 114,833 Shares used in computation: Weighted-average common shares outstanding 36,669 32,082 36,518 31,858 Basic net income (loss) per share $ (0.04 ) $ 0.33 $ (0.92 ) $ 3.60 Diluted net income (loss) per share: Net income (loss) applicable to common stockholders $ (1,457 ) $ 10,594 $ (33,685 ) $ 114,833 Shares used in computation: Weighted-average common shares outstanding 36,669 32,082 36,518 31,858 Stock options — 3,153 — 2,995 Restricted stock units — 30 — 27 Employee stock purchase plan — 7 — 4 Weighted-average diluted common shares 36,669 35,272 36,518 34,884 Diluted net income (loss) per share $ (0.04 ) $ 0.30 $ (0.92 ) $ 3.29 |
Schedules for Computation of Diluted Weighted-Average Shares Outstanding | The following potentially dilutive common stock equivalents outstanding at the end of the period were excluded from the computations of weighted-average diluted common shares for the periods indicated as their effects would be anti-dilutive (in thousands): Three Months Ended June 30, Six Months Ended June 30, 2019 2018 2019 2018 Stock options issued and outstanding 5,454 804 5,454 1,236 Unvested restricted stock units outstanding 40 — 40 — Employee stock purchase plan 26 — 26 — 5,520 804 5,520 1,236 |
Supplemental Disclosures (Table
Supplemental Disclosures (Tables) | 6 Months Ended |
Jun. 30, 2019 | |
Payables And Accruals [Abstract] | |
Schedules of Accrued Expenses and Other Current Liabilities | Accrued expenses and other current liabilities consists of the following (in thousands): June 30, 2019 December 31, 2018 Accrued personnel costs $ 6,692 $ 9,484 Accrued external research and development expenses 4,925 4,274 Accrued sublicense fees and royalties 1,514 1,617 Accrued external general and administrative expenses 1,498 773 Accrued income taxes payable 564 726 Accrued purchases of property and equipment 397 221 Other accrued expenses and current liabilities 944 69 $ 16,534 $ 17,164 |
Nature of Business - Additional
Nature of Business - Additional Information (Detail) - USD ($) $ in Thousands | Jun. 30, 2019 | Dec. 31, 2018 |
Organization Consolidation And Presentation Of Financial Statements [Abstract] | ||
Accumulated deficit | $ (116,774) | $ (83,016) |
Cash, cash equivalents and marketable securities | $ 449,700 |
Summary of Significant Accoun_4
Summary of Significant Accounting Policies - Summary of Reconciliation of Cash and Cash Equivalents and Restricted Cash (Detail) - USD ($) $ in Thousands | Jun. 30, 2019 | Dec. 31, 2018 | Jun. 30, 2018 |
Accounting Policies [Abstract] | |||
Cash and cash equivalents | $ 55,142 | $ 75,561 | $ 106,889 |
Restricted cash | 1,053 | $ 1,053 | 225 |
Total cash and cash equivalents and restricted cash | $ 56,195 | $ 107,114 |
Summary of Significant Accoun_5
Summary of Significant Accounting Policies - Additional Information (Detail) - USD ($) | Jan. 01, 2019 | Jun. 30, 2019 | Dec. 31, 2018 |
Significant Accounting Policies [Line Items] | |||
Impairment of available-for-sale securities deemed to be other-than-temporary | $ 0 | ||
Operating lease right-of-use assets | 5,904,000 | ||
Property and equipment, net | 27,330,000 | $ 28,702,000 | |
Accumulated deficit | $ (116,774,000) | $ (83,016,000) | |
ASU 2016-02 [Member] | |||
Significant Accounting Policies [Line Items] | |||
Operating lease right-of-use assets | $ 7,400,000 | ||
Operating lease liabilities | 8,400,000 | ||
ASU 2016-02 [Member] | Maximum [Member] | |||
Significant Accounting Policies [Line Items] | |||
Accumulated deficit | 100,000 | ||
ASU 2016-02 [Member] | Restatement Adjustment [Member] | |||
Significant Accounting Policies [Line Items] | |||
Current and noncurrent deferred rent liabilities | 1,400,000 | ||
Prepaid expenses, other current assets and other assets | 400,000 | ||
ASU 2016-02 [Member] | Restatement Adjustment [Member] | 9800 Medical Center Drive Lease [Member] | |||
Significant Accounting Policies [Line Items] | |||
Property and equipment, net | (5,900,000) | ||
Financing lease obligation | (5,900,000) | ||
ASU 2018-02 [Member] | Maximum [Member] | |||
Significant Accounting Policies [Line Items] | |||
Reclassification of stranded tax effects of unrealized gains and losses on available-for-sale securities from accumulated other comprehensive income (loss) to accumulated deficit | $ 100,000 |
Marketable Securities - Summary
Marketable Securities - Summary of Company Marketable Securities (Detail) - USD ($) $ in Thousands | Jun. 30, 2019 | Dec. 31, 2018 |
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost | $ 361,370 | $ 395,304 |
Unrealized Gains | 33,181 | 129 |
Unrealized Losses | (3) | (414) |
Fair Value | 394,548 | 395,019 |
U.S. Government and Federal Agency Securities [Member] | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost | 102,825 | 103,410 |
Unrealized Gains | 388 | 93 |
Unrealized Losses | (37) | |
Fair Value | 103,213 | 103,466 |
Certificates of Deposit [Member] | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost | 8,748 | 8,992 |
Unrealized Gains | 67 | |
Fair Value | 8,815 | 8,992 |
Corporate Bonds [Member] | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost | 249,377 | 282,902 |
Unrealized Gains | 1,070 | 36 |
Unrealized Losses | (3) | (377) |
Fair Value | 250,444 | $ 282,561 |
Equity Securities [Member] | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost | 420 | |
Unrealized Gains | 31,656 | |
Fair Value | $ 32,076 |
Marketable Securities - Additio
Marketable Securities - Additional Information (Detail) | 3 Months Ended | 6 Months Ended | 12 Months Ended | ||
Jun. 30, 2019USD ($)Security | Jun. 30, 2018USD ($) | Jun. 30, 2019USD ($)Security | Jun. 30, 2018USD ($) | Dec. 31, 2018USD ($) | |
Schedule Of Available For Sale Securities [Line Items] | |||||
Available for sale debt securities remaining maturities greater than three years | $ 0 | $ 0 | $ 0 | ||
Unrealized gains (losses) on available-for-sale debt securities, before tax | 800,000 | $ 100,000 | 1,800,000 | $ (100,000) | |
Unrealized gains (losses) on available-for-sale debt securities, income tax expense | $ 300,000 | 0 | $ 700,000 | 0 | |
Number of investment grade fixed income debt security | Security | 5 | 5 | |||
Other-than-temporary impaired | $ 0 | $ 0 | |||
Non-marketable equity securities, carrying value | $ 400,000 | 400,000 | |||
Marketable securities realized | 0 | ||||
Equity Securities [Member] | |||||
Schedule Of Available For Sale Securities [Line Items] | |||||
Marketable securities unrealized gain (loss) | 31,700,000 | 31,700,000 | |||
Investment Income [Member] | Maximum [Member] | |||||
Schedule Of Available For Sale Securities [Line Items] | |||||
Net realized gains or losses available for sale debt securities | $ 0 | $ 0 | $ 100,000 | $ 0 |
Marketable Securities - Summa_2
Marketable Securities - Summary of Fair Values and Unrealized Losses of Marketable Securities Held by the Company in an Unrealized Loss Position for Less Than 12 months and 12 Months or Greater (Detail) - USD ($) $ in Thousands | Jun. 30, 2019 | Dec. 31, 2018 |
Schedule Of Available For Sale Securities [Line Items] | ||
Less than 12 Months, Fair Value | $ 19,176 | |
Less than 12 Months, Unrealized Losses | (3) | $ (391) |
12 Months or Greater, Unrealized Losses | (23) | |
Total, Fair Value | 19,176 | |
Total, Unrealized Losses | (3) | (414) |
Less than 12 Months, Fair Value | 298,407 | |
12 Months or Greater, Fair Value | 12,424 | |
Total, Fair Value | 310,831 | |
Corporate Bonds [Member] | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Less than 12 Months, Fair Value | 19,176 | |
Less than 12 Months, Unrealized Losses | (3) | (354) |
12 Months or Greater, Unrealized Losses | (23) | |
Total, Fair Value | 19,176 | |
Total, Unrealized Losses | $ (3) | (377) |
Less than 12 Months, Fair Value | 245,283 | |
12 Months or Greater, Fair Value | 12,424 | |
Total, Fair Value | 257,707 | |
U.S. Government and Federal Agency Securities [Member] | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Less than 12 Months, Unrealized Losses | (37) | |
Total, Unrealized Losses | (37) | |
Less than 12 Months, Fair Value | 53,124 | |
Total, Fair Value | $ 53,124 |
Fair Value of Financial Instr_3
Fair Value of Financial Instruments - Schedule of Fair Value of Cash Equivalents and Marketable Securities (Detail) - USD ($) $ in Thousands | Jun. 30, 2019 | Dec. 31, 2018 |
Marketable securities: | ||
Fair Value | $ 394,548 | $ 395,019 |
Cash Equivalents and Marketable Securities [Member] | ||
Marketable securities: | ||
Total cash equivalents and marketable securities | 449,674 | 470,561 |
Corporate Bonds [Member] | ||
Marketable securities: | ||
Fair Value | 250,444 | 282,561 |
Cash Equivalents [Member] | ||
Cash equivalents: | ||
Total cash equivalents | 55,126 | 75,542 |
U.S. Government and Federal Agency Securities [Member] | ||
Marketable securities: | ||
Fair Value | 103,213 | 103,466 |
Certificates of Deposit [Member] | ||
Marketable securities: | ||
Fair Value | 8,815 | 8,992 |
Equity Securities [Member] | ||
Marketable securities: | ||
Fair Value | 32,076 | |
Money Market Mutual Funds [Member] | Cash Equivalents [Member] | ||
Cash equivalents: | ||
Total cash equivalents | 55,126 | 75,542 |
Quoted Prices in Active Markets (Level 1) [Member] | ||
Marketable securities: | ||
Fair Value | 32,076 | |
Quoted Prices in Active Markets (Level 1) [Member] | Cash Equivalents and Marketable Securities [Member] | ||
Marketable securities: | ||
Total cash equivalents and marketable securities | 32,076 | |
Quoted Prices in Active Markets (Level 1) [Member] | Equity Securities [Member] | ||
Marketable securities: | ||
Fair Value | 32,076 | |
Significant Other Observable Inputs (Level 2) [Member] | ||
Marketable securities: | ||
Fair Value | 362,472 | 395,019 |
Significant Other Observable Inputs (Level 2) [Member] | Cash Equivalents and Marketable Securities [Member] | ||
Marketable securities: | ||
Total cash equivalents and marketable securities | 417,598 | 470,561 |
Significant Other Observable Inputs (Level 2) [Member] | Corporate Bonds [Member] | ||
Marketable securities: | ||
Fair Value | 250,444 | 282,561 |
Significant Other Observable Inputs (Level 2) [Member] | Cash Equivalents [Member] | ||
Cash equivalents: | ||
Total cash equivalents | 55,126 | 75,542 |
Significant Other Observable Inputs (Level 2) [Member] | U.S. Government and Federal Agency Securities [Member] | ||
Marketable securities: | ||
Fair Value | 103,213 | 103,466 |
Significant Other Observable Inputs (Level 2) [Member] | Certificates of Deposit [Member] | ||
Marketable securities: | ||
Fair Value | 8,815 | 8,992 |
Significant Other Observable Inputs (Level 2) [Member] | Money Market Mutual Funds [Member] | Cash Equivalents [Member] | ||
Cash equivalents: | ||
Total cash equivalents | $ 55,126 | $ 75,542 |
Fair Value of Financial Instr_4
Fair Value of Financial Instruments - Additional Information (Detail) - USD ($) | 3 Months Ended | 6 Months Ended | |||
Jun. 30, 2019 | Jun. 30, 2018 | Jun. 30, 2019 | Jun. 30, 2018 | Dec. 31, 2018 | |
Fair Value Disclosures [Abstract] | |||||
Fair value hierarchy level 1 to level 2 transfers amount | $ 0 | $ 0 | |||
Fair value hierarchy level 2 to level 1 transfers amount | 0 | 0 | |||
Non-marketable equity securities | $ 400,000 | ||||
Remeasurements or impairment losses on non-marketable equity securities | $ 0 | $ 0 | $ 0 | $ 0 |
Property and Equipment Net - Sc
Property and Equipment Net - Schedule of Property and Equipment (Detail) - USD ($) $ in Thousands | Jun. 30, 2019 | Dec. 31, 2018 |
Property Plant And Equipment [Line Items] | ||
Total property and equipment | $ 37,913 | $ 35,939 |
Accumulated depreciation and amortization | (10,583) | (7,237) |
Property and equipment, net | 27,330 | 28,702 |
Lab Equipment [Member] | ||
Property Plant And Equipment [Line Items] | ||
Total property and equipment | 16,533 | 14,417 |
Computer Equipment and Software [Member] | ||
Property Plant And Equipment [Line Items] | ||
Total property and equipment | 2,342 | 2,002 |
Furniture and Fixtures [Member] | ||
Property Plant And Equipment [Line Items] | ||
Total property and equipment | 1,946 | 1,915 |
Leasehold Improvements [Member] | ||
Property Plant And Equipment [Line Items] | ||
Total property and equipment | $ 17,092 | 11,751 |
Construction in Progress [Member] | ||
Property Plant And Equipment [Line Items] | ||
Total property and equipment | $ 5,854 |
Property and Equipment Net - Ad
Property and Equipment Net - Additional Information (Detail) - USD ($) $ in Thousands | Jun. 30, 2019 | Jan. 01, 2019 | Dec. 31, 2018 |
Property Plant And Equipment [Line Items] | |||
Property and equipment, net | $ 27,330 | $ 28,702 | |
ASU 2016-02 [Member] | Restatement Adjustment [Member] | 9800 Medical Center Drive Lease [Member] | |||
Property Plant And Equipment [Line Items] | |||
Property and equipment, net | $ (5,900) |
Leases - Additional Information
Leases - Additional Information (Detail) | 1 Months Ended | 3 Months Ended | 6 Months Ended | |||||||
May 31, 2019USD ($) | Nov. 30, 2018USD ($)ft² | Apr. 30, 2018USD ($) | May 31, 2016USD ($) | Jan. 31, 2016USD ($) | Mar. 31, 2015 | Jun. 30, 2019USD ($) | Jun. 30, 2019USD ($) | Jan. 01, 2019USD ($) | Dec. 31, 2018USD ($) | |
Other Commitments [Line Items] | ||||||||||
Property and equipment, net | $ 27,330,000 | $ 27,330,000 | $ 28,702,000 | |||||||
Financing lease obligation | $ 5,854,000 | |||||||||
Operating lease liabilities | (1,171,000) | |||||||||
Decrease in right-of-use assets | $ (1,156,000) | |||||||||
Rockville, Maryland [Member] | ||||||||||
Other Commitments [Line Items] | ||||||||||
Operating leases expiration year | 2022 | |||||||||
9800 Medical Center Drive Lease [Member] | Leasehold Improvements [Member] | ||||||||||
Other Commitments [Line Items] | ||||||||||
Property and equipment, net | 4,800,000 | $ 4,800,000 | ||||||||
9800 Medical Center Drive Lease [Member] | ASU 2016-02 [Member] | Restatement Adjustment [Member] | ||||||||||
Other Commitments [Line Items] | ||||||||||
Property and equipment, net | $ (5,900,000) | |||||||||
Financing lease obligation | $ (5,900,000) | |||||||||
9800 Medical Center Drive Lease [Member] | Rockville, Maryland [Member] | ||||||||||
Other Commitments [Line Items] | ||||||||||
Lease Expiration Term | 16 years | |||||||||
Construction completion, description | The initial construction of the building is being conducted by the landlord and is expected to be completed in 2020 | |||||||||
Tenant improvement allowance to be received | $ 15,300,000 | |||||||||
Lease not yet commenced, option to extend the lease | true | |||||||||
Lease not yet commenced, additional lease term | 10 years | |||||||||
Lease not yet commenced, option to terminate the lease | true | |||||||||
Lease not yet commenced, option to terminate the lease, description | the option to terminate the lease after 12 years from the delivery of the leased premises to the Company. If the Company elects to terminate the lease, it will be subject to a termination fee equal to the unamortized tenant improvement allowance, rent abatement and landlord commissions as of the termination date, bearing interest at 5% per annum, plus four months of base rent and operating expenses. | |||||||||
Minimum release term for option to terminate | 12 years | |||||||||
Percentage of early termination fee | 5.00% | |||||||||
Term of required base rent to be paid as termination fee | 4 months | |||||||||
Frequency of periodic payment | Monthly payments under the 9800 Medical Center Drive Lease begin approximately 12 months from the delivery of the leased premises to the Company and escalate annually in accordance with the lease agreement. | |||||||||
Periodic payment term, from the delivery of leased premises | 12 months | |||||||||
Letters of credit | $ 800,000 | |||||||||
Number of square feet to be constructed | ft² | 139,000 | |||||||||
9712 Medical Center Drive [Member] | Rockville, Maryland [Member] | ||||||||||
Other Commitments [Line Items] | ||||||||||
Lease term commenced date | 2015-04 | |||||||||
Lease term | In September 2015, November 2015, July 2017 and April 2018, the Company amended the 9712 Medical Center Drive Lease to include additional office and laboratory space at 9714 Medical Center Drive, and ultimately extend the term of the lease to September 2021. | |||||||||
Operating lease, existence of option to extend the lease | true | |||||||||
Additional lease term under option to extend | 6 years | |||||||||
Tenant improvement allowance received | $ 400,000 | |||||||||
9600 Blackwell Road [Member] | Rockville, Maryland [Member] | ||||||||||
Other Commitments [Line Items] | ||||||||||
Percentage of early termination fee | 8.00% | |||||||||
Lease term commenced date | 2016-02 | |||||||||
Operating lease, existence of option to extend the lease | true | |||||||||
Additional lease term under option to extend | 5 years | |||||||||
Tenant improvement allowance received | $ 800,000 | |||||||||
Lease expiration date | Sep. 30, 2023 | |||||||||
Required period to terminate lease after commencement period | 67 months | |||||||||
400 Madison Avenue [Member] | 400 Madison Lease [Member] | ||||||||||
Other Commitments [Line Items] | ||||||||||
Letters of credit | $ 200,000 | |||||||||
Operating leases commencement year | 2016-07 | |||||||||
Operating lease expiration year | 2020-10 | |||||||||
400 Madison Avenue [Member] | 400 Madison Lease [Member] | Lease Amendment [Member] | ||||||||||
Other Commitments [Line Items] | ||||||||||
Tenant improvement allowance to be received | $ 700,000 | |||||||||
Operating lease expiration year | 2019-08 | |||||||||
Operating leases expiration term | 7 years 6 months | |||||||||
Operating lease liabilities | 400,000 | $ 400,000 | ||||||||
Decrease in right-of-use assets | $ 400,000 | $ 400,000 |
Leases - Summary of Lease Costs
Leases - Summary of Lease Costs and Supplemental Cash Flow Information of Operating Leases (Detail) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended |
Jun. 30, 2019 | Jun. 30, 2019 | |
Leases [Abstract] | ||
Operating lease cost | $ 697 | $ 1,398 |
Variable lease cost | 138 | 298 |
Total lease cost | 835 | 1,696 |
Cash paid for amounts included in operating lease liabilities | 684 | 1,395 |
Right-of-use assets acquired through operating lease liabilities | $ (371) | $ (335) |
Leases - Weighted-Average Remai
Leases - Weighted-Average Remaining Lease Term and Weighted-Average Discount Rate of Operating Leases (Detail) | Jun. 30, 2019 |
Leases [Abstract] | |
Weighted-average remaining lease term (years) | 2 years 10 months 24 days |
Weighted-average discount rate | 5.60% |
Leases - Reconciliation of Undi
Leases - Reconciliation of Undiscounted Future Minimum Lease Payments Remaining Under Leases that Have Not Yet Commenced and Other Operating Leases (Detail) $ in Thousands | Jun. 30, 2019USD ($) |
Undiscounted future minimum lease payments: | |
2019 (remainder of year) | $ 1,298 |
2020 | 3,273 |
2021 | 4,720 |
2022 | 6,045 |
2023 | 6,855 |
Thereafter | 83,650 |
Total undiscounted future minimum lease payments | 105,841 |
Current portion of operating lease liabilities | (2,276) |
Operating lease liabilities, non-current | 4,654 |
Leases Not Yet Commenced [Member] | |
Undiscounted future minimum lease payments: | |
2019 (remainder of year) | 98 |
2020 | 460 |
2021 | 2,308 |
2022 | 5,422 |
2023 | 6,376 |
Thereafter | 83,650 |
Total undiscounted future minimum lease payments | 98,314 |
Other Operating leases [Member] | |
Undiscounted future minimum lease payments: | |
2019 (remainder of year) | 1,200 |
2020 | 2,813 |
2021 | 2,412 |
2022 | 623 |
2023 | 479 |
Total undiscounted future minimum lease payments | 7,527 |
Amount representing imputed interest | (597) |
Total operating lease liabilities | 6,930 |
Current portion of operating lease liabilities | (2,276) |
Operating lease liabilities, non-current | $ 4,654 |
Leases - Schedule of Future Min
Leases - Schedule of Future Minimum Lease Payments Under Topic 840 for 9800 Medical Center Drive Lease and Other Operating Leases (Detail) $ in Thousands | Dec. 31, 2018USD ($) |
Lessee Lease Description [Line Items] | |
2019 | $ 2,798 |
2020 | 3,054 |
2021 | 3,720 |
2022 | 4,910 |
2023 | 5,635 |
Thereafter | 76,420 |
Total minimum lease payments | 96,537 |
9800 Medical Center Drive Lease [Member] | |
Lessee Lease Description [Line Items] | |
2021 | 1,329 |
2022 | 4,289 |
2023 | 5,156 |
Thereafter | 76,420 |
Total minimum lease payments | 87,194 |
Other Operating leases [Member] | |
Lessee Lease Description [Line Items] | |
2019 | 2,798 |
2020 | 3,054 |
2021 | 2,391 |
2022 | 621 |
2023 | 479 |
Total minimum lease payments | $ 9,343 |
Commitments and Contingencies -
Commitments and Contingencies - Additional Information (Detail) - USD ($) | Jun. 30, 2019 | Apr. 30, 2019 |
European Patent Office Proceeding [Member] | ||
Other Commitments [Line Items] | ||
Liability related to third party opposition | $ 0 | |
University of Pennsylvania [Member] | Maximum [Member] | ||
Other Commitments [Line Items] | ||
Upfront fee, milestone fees | $ 20,500,000 |
License and Royalty Revenue - A
License and Royalty Revenue - Additional Information (Detail) | 1 Months Ended | 3 Months Ended | 6 Months Ended | ||||||||
May 31, 2018USD ($) | Jan. 31, 2018USD ($) | Mar. 31, 2014USD ($) | Jun. 30, 2019USD ($)ProductCandidateTechnologyLicensee | Jun. 30, 2018USD ($) | Jun. 30, 2019USD ($)ProductCandidateTechnologyLicensee | Jun. 30, 2018USD ($) | Mar. 31, 2019USD ($) | Dec. 31, 2018USD ($) | Mar. 31, 2018USD ($) | Dec. 31, 2017USD ($) | |
License Revenue [Line Items] | |||||||||||
Milestone payment upon commencement of clinical trials in humans | $ 18,600,000 | ||||||||||
Milestone payment upon submission of regulatory approval filings | 32,500,000 | ||||||||||
Milestone payment upon approval of commercial products by regulatory agencies | 81,000,000 | ||||||||||
Milestone payment upon achievement of specified sales targets for licensed products | 187,000,000 | ||||||||||
Accounts receivable | $ 33,634,000 | $ 5,224,000 | 33,634,000 | $ 5,224,000 | $ 31,130,000 | $ 31,599,000 | $ 58,621,000 | $ 5,850,000 | |||
Deferred revenue, current and non-current | 3,333,000 | 600,000 | 3,333,000 | 600,000 | 3,933,000 | 3,933,000 | |||||
Licenses revenue | 7,881,000 | 40,031,000 | 8,765,000 | 172,422,000 | |||||||
Impirment losses on receivables or contract assets | 0 | ||||||||||
Billed to customers | 200,000 | 200,000 | 400,000 | ||||||||
Billable to customers in future periods | 33,400,000 | 33,400,000 | 31,200,000 | ||||||||
Allowance for doubtful accounts | 0 | $ 0 | 0 | ||||||||
Transaction price description | Upon its execution, the transaction price of the January 2018 Amendment was $132.1 million, which was fully recognized as license revenue upon the delivery of the modified license in January 2018. In May 2018, as a result of the acquisition of AveXis by Novartis, the transaction price was increased by $40.0 million to account for the acceleration of the sale-based milestone which was previously excluded from the transaction price. The $40.0 million increase in the transaction price was recognized as license revenue upon the completion of the change of control in May 2018 since the amended license had been fully delivered to AveXis. Additionally, due to the acceleration of the two $30.0 million payments originally due in January 2019 and January 2020, the Company recognized $6.1 million of interest income from licensing upon the completion of the change of control of AveXis, which represents the remaining present value discount on such payments as of the date of the change of control. As of June 30, 2019, the transaction price of the January 2018 Amendment was $172.1 million, which includes: (i) the $80.0 million payment in January 2018, (ii) the present value, as of the date of the January 2018 Amendment, of the two $30.0 million payments originally due in January 2019 and January 2020 and (iii) the $40.0 million sales-based milestone which was accelerated upon the change of control in May 2018. Variable consideration under the January 2018 Amendment, which has been excluded from the transaction price, includes the remaining sales-based milestone payment of $80.0 million, as well as any potential sublicense fees or royalties on sales of licensed products, which will be recognized in the period of the underlying sales or sublicenses. | ||||||||||
Interest income from licensing | 762,000 | 6,898,000 | $ 1,375,000 | 8,253,000 | |||||||
Accounts receivable, current | 9,679,000 | 9,679,000 | 8,587,000 | ||||||||
AveXis, Inc. [Member] | |||||||||||
License Revenue [Line Items] | |||||||||||
Interest income from licensing | 7,000 | 6,752,000 | 15,000 | 7,950,000 | |||||||
License [Member] | |||||||||||
License Revenue [Line Items] | |||||||||||
Licenses revenue | 600,000 | 0 | 600,000 | ||||||||
License [Member] | AveXis, Inc. [Member] | |||||||||||
License Revenue [Line Items] | |||||||||||
Licenses revenue | $ 4,424,000 | 40,000,000 | $ 4,424,000 | 172,066,000 | |||||||
NAV Technology Licensees [Member] | NAV Technology Platform [Member] | |||||||||||
License Revenue [Line Items] | |||||||||||
Number of technology licensees | TechnologyLicensee | 9 | 9 | |||||||||
NAV Technology Licensees [Member] | NAV Technology Platform [Member] | Minimum [Member] | |||||||||||
License Revenue [Line Items] | |||||||||||
Number of development partnered product candidates | ProductCandidate | 20 | 20 | |||||||||
March 2014 Licence Agreement [Member] | AveXis, Inc. [Member] | |||||||||||
License Revenue [Line Items] | |||||||||||
License fees received | 100,000,000 | ||||||||||
Accounts receivable | $ 53,300,000 | ||||||||||
Accounts receivable | $ 1,100,000 | $ 1,100,000 | 200,000 | ||||||||
Up-front fee paid | $ 2,000,000 | 2,000,000 | |||||||||
Milestone fee payments upon achievement of various development and commercialization | $ 12,300,000 | ||||||||||
License agreement amendment date | 2018-01 | ||||||||||
Transaction price of license | 11,000,000 | 11,000,000 | |||||||||
Payments for remaining development milestones | 3,500,000 | 3,500,000 | |||||||||
Increase in transaction price of license | 3,500,000 | 3,500,000 | |||||||||
Accounts receivable, current | 900,000 | 900,000 | |||||||||
Accounts receivable, non -current | 200,000 | 200,000 | |||||||||
March 2014 Licence Agreement [Member] | Maximum [Member] | AveXis, Inc. [Member] | |||||||||||
License Revenue [Line Items] | |||||||||||
Accounts receivable, current | 100,000 | ||||||||||
Accounts receivable, non -current | 200,000 | ||||||||||
License Agreement [Member] | License [Member] | |||||||||||
License Revenue [Line Items] | |||||||||||
License revenue | 3,200,000 | $ 39,900,000 | 4,000,000 | $ 200,000 | |||||||
Proceeds from milestone license revenue | $ 40,000,000 | ||||||||||
November 2018 License Agreement [Member] | Abeona Therapeutics Inc. [Member] | |||||||||||
License Revenue [Line Items] | |||||||||||
Accounts receivable | 27,300,000 | 27,300,000 | $ 26,000,000 | ||||||||
January 2018 Amendment Agreement [Member] | AveXis, Inc. [Member] | |||||||||||
License Revenue [Line Items] | |||||||||||
License fees received | 100,000,000 | ||||||||||
License agreement period | 14 years | ||||||||||
License fee | $ 80,000,000 | ||||||||||
Sales-based milestone payment unpaid in the event of change of control | 40,000,000 | ||||||||||
Transaction price of license | 132,100,000 | $ 172,100,000 | 172,100,000 | ||||||||
Increase in transaction price of license | 0 | ||||||||||
Interest income from licensing | 6,100,000 | ||||||||||
Sales-based milestone payment exclude transaction price | $ 80,000,000 | ||||||||||
Payments due from related party | $ 30,000,000 | ||||||||||
Consideration payment due period one | 2019-01 | ||||||||||
Consideration payment due period two | 2020-01 | ||||||||||
January 2018 Amendment Agreement [Member] | AveXis, Inc. [Member] | Share-based Milestone [Member] | |||||||||||
License Revenue [Line Items] | |||||||||||
Increase in transaction price of license | 40,000,000 | ||||||||||
January 2018 Amendment Agreement [Member] | AveXis, Inc. [Member] | First Anniversary [Member] | |||||||||||
License Revenue [Line Items] | |||||||||||
License fee | 30,000,000 | ||||||||||
January 2018 Amendment Agreement [Member] | AveXis, Inc. [Member] | Second Anniversary [Member] | |||||||||||
License Revenue [Line Items] | |||||||||||
License fee | 30,000,000 | ||||||||||
January 2018 Amendment Agreement [Member] | Minimum [Member] | AveXis, Inc. [Member] | |||||||||||
License Revenue [Line Items] | |||||||||||
Milestone payment | $ 120,000,000 | ||||||||||
January 2018 Amendment Agreement [Member] | License [Member] | AveXis, Inc. [Member] | |||||||||||
License Revenue [Line Items] | |||||||||||
Increase in transaction price of license | $ 40,000,000 |
License and Royalty Revenue - S
License and Royalty Revenue - Summary of Changes in Balances of Receivables, Contract Assets and Contract Liabilities (Detail) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2019 | Jun. 30, 2018 | Jun. 30, 2019 | Jun. 30, 2018 | |
Receivables and contract assets: | ||||
Accounts receivable, current and non-current, Balance at Beginning of Period | $ 31,130 | $ 58,621 | $ 31,599 | $ 5,850 |
Accounts receivable, current and non-current, Net Additions (Deductions) | 2,504 | (53,397) | 2,035 | (626) |
Accounts receivable, current and non-current, Balance at End of Period | 33,634 | 5,224 | 33,634 | 5,224 |
Contract assets, Balance at Beginning of Period | 1,000 | 250 | 750 | 350 |
Contract assets, Net Additions (Deductions) | (1,000) | (250) | (750) | (350) |
Contract liabilities: | ||||
Deferred revenue, current and non-current, Balance at Beginning of Period | 3,933 | 3,933 | ||
Deferred revenue, Net Additions (Deductions) | (600) | 600 | (600) | 600 |
Deferred revenue, current and non-current, Balance at End of Period | $ 3,333 | $ 600 | $ 3,333 | $ 600 |
License and Royalty Revenue -_2
License and Royalty Revenue - Additional Information (Detail1) | Jun. 30, 2019 |
January 2018 Amendment Agreement [Member] | AveXis, Inc. [Member] | Revenue, Remaining Performance Obligation, Expected Timing of Satisfaction, Start Date: 2018-01-01 | |
License Revenue [Line Items] | |
Revenue, remaining performance obligation, expected timing of satisfaction, period | 1 year |
License and Royalty Revenue -_3
License and Royalty Revenue - Schedule Of License Revenue (Detail) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2019 | Jun. 30, 2018 | Jun. 30, 2019 | Jun. 30, 2018 | |
License Revenue [Line Items] | ||||
Licenses revenue | $ 7,881 | $ 40,031 | $ 8,765 | $ 172,422 |
Interest income from licensing | 762 | 6,898 | 1,375 | 8,253 |
License [Member] | ||||
License Revenue [Line Items] | ||||
Licenses revenue | 600 | 0 | 600 | |
AveXis, Inc. [Member] | ||||
License Revenue [Line Items] | ||||
Interest income from licensing | 7 | 6,752 | 15 | 7,950 |
AveXis, Inc. [Member] | License [Member] | ||||
License Revenue [Line Items] | ||||
Licenses revenue | $ 4,424 | $ 40,000 | $ 4,424 | $ 172,066 |
Stock-based Compensation - Addi
Stock-based Compensation - Additional Information (Detail) - USD ($) $ / shares in Units, $ in Thousands | 1 Months Ended | 6 Months Ended | |
Jan. 31, 2019 | Jun. 30, 2019 | Jun. 30, 2018 | |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||
Unrecognized stock-based compensation expense | $ 70,400 | ||
Unrecognized stock-based compensation, weighted-average period | 3 years | ||
Proceeds from exercise of stock options | $ 5,129 | $ 6,999 | |
2015 Equity Incentive Plan [Member] | |||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||
Additional shares to be issued | 1,444,808 | ||
Common stock shares authorized for issuance | 10,933,221 | ||
Shares available for future grants | 2,327,843 | ||
Weighted-average fair values of options granted | $ 32.24 | ||
Exercise of stock options, Shares | 621,415 | ||
Total intrinsic value of options exercised | $ 25,500 | ||
2015 Employee Stock Purchase Plan [Member] | |||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||
Common stock shares authorized for issuance | 254,000 | ||
Shares available for future grants | 159,339 | ||
Common stock shares issued to participants | 10,241 |
Stock-based Compensation - Stoc
Stock-based Compensation - Stock-Based Compensation Expense by Award Type (Detail) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2019 | Jun. 30, 2018 | Jun. 30, 2019 | Jun. 30, 2018 | |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||
Stock-based compensation expense | $ 7,099 | $ 3,982 | $ 12,817 | $ 7,272 |
Stock Option [Member] | ||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||
Stock-based compensation expense | 6,844 | 3,818 | 12,296 | 6,939 |
Restricted Stock Units [Member] | ||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||
Stock-based compensation expense | 69 | 69 | 136 | 136 |
Employee Stock Purchase Plan [Member] | ||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||
Stock-based compensation expense | $ 186 | $ 95 | $ 385 | $ 197 |
Stock-based Compensation - St_2
Stock-based Compensation - Stock-Based Compensation Expense (Detail) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2019 | Jun. 30, 2018 | Jun. 30, 2019 | Jun. 30, 2018 | |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||
Stock-based compensation expense | $ 7,099 | $ 3,982 | $ 12,817 | $ 7,272 |
Research and Development Costs [Member] | ||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||
Stock-based compensation expense | 3,526 | 1,895 | 5,873 | 3,424 |
General and Administrative [Member] | ||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||
Stock-based compensation expense | $ 3,573 | $ 2,087 | $ 6,944 | $ 3,848 |
Stock-based Compensation - Summ
Stock-based Compensation - Summary of Stock Option Activity (Detail) - 2014 and 2015 Equity Incentive Plan [Member] $ / shares in Units, shares in Thousands, $ in Thousands | 6 Months Ended | 12 Months Ended |
Jun. 30, 2019USD ($)$ / sharesshares | Dec. 31, 2018USD ($)$ / sharesshares | |
Share-based Compensation Arrangement by Share-based Payment Award, Options, Nonvested, Number of Shares [Roll Forward] | ||
Shares Outstanding, Beginning Balance | shares | 4,855 | |
Shares, Granted | shares | 1,410 | |
Shares, Exercised | shares | (621) | |
Shares, Cancelled or forfeited | shares | (190) | |
Shares Outstanding, Ending Balance | shares | 5,454 | 4,855 |
Shares, Exercisable | shares | 2,630 | |
Shares, Vested and expected to vest | shares | 5,454 | |
Share-based Compensation Arrangement by Share-based Payment Award, Options, Outstanding, Weighted Average Exercise Price [Abstract] | ||
Weighted-average Exercise Price Outstanding, Beginning Balance | $ / shares | $ 19.31 | |
Weighted-average Exercise Price, Granted | $ / shares | 48.48 | |
Weighted-average Exercise Price, Exercised | $ / shares | 8.24 | |
Weighted-average Exercise Price, Cancelled or forfeited | $ / shares | 32.92 | |
Weighted-average Exercise Price, Outstanding, Ending Balance | $ / shares | 27.64 | $ 19.31 |
Weighted-average Exercise Price, Exercisable | $ / shares | 13.65 | |
Weighted-average Exercise Price, Vested and expected to vest | $ / shares | $ 27.64 | |
Share-based Compensation Arrangement by Share-based Payment Award, Options, Additional Disclosures [Abstract] | ||
Weighted-average Remaining Contractual Life (Years) Outstanding | 7 years 9 months 18 days | 7 years 7 months 6 days |
Weighted-average Remaining Contractual Life (Years), Exercisable | 6 years 8 months 12 days | |
Weighted-average Remaining Contractual Life (Years), Vested and expected to vest | 7 years 9 months 18 days | |
Aggregate Intrinsic Value Outstanding | $ | $ 136,336 | $ 118,185 |
Aggregate Intrinsic Value, Exercisable | $ | 99,341 | |
Aggregate Intrinsic Value, Vested and expected to vest | $ | $ 136,336 |
Stock-based Compensation - Su_2
Stock-based Compensation - Summary of Unvested RSUs Activity Under 2015 Plan (Detail) - Restricted Stock Units [Member] shares in Thousands | 6 Months Ended |
Jun. 30, 2019$ / sharesshares | |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |
Shares Unvested, Beginning Balance | 40 |
Shares, Granted | 0 |
Shares, Vested | 0 |
Shares, Forfeited | 0 |
Shares Unvested, Ending Balance | 40 |
Weighted-average Grant Date Fair Value, Unvested Beginning Balance | $ / shares | $ 20.90 |
Weighted-average Grant Date Fair Value, Unvested Ending Balance | $ / shares | $ 20.90 |
Income Taxes - Additional Infor
Income Taxes - Additional Information (Detail) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | |||
Jun. 30, 2019 | Jun. 30, 2018 | Jun. 30, 2019 | Jun. 30, 2018 | Dec. 31, 2018 | |
Income Tax Disclosure [Line Items] | |||||
Income tax benefit | $ 129 | $ (3,850) | $ 199 | $ (3,850) | |
Unrealized gains (losses) on available-for-sale debt securities, income tax expense | 300 | $ 0 | 700 | $ 0 | |
Accrued income tax provision | 564 | 564 | $ 726 | ||
Accrued Expenses and Other Current Liabilities [Member] | |||||
Income Tax Disclosure [Line Items] | |||||
Accrued income tax provision | $ 500 | $ 500 |
Related Party Transactions - Ad
Related Party Transactions - Additional Information (Detail) - FOXKISER,LLP [Member] - Service Agreements [Member] - USD ($) $ in Millions | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2019 | Jun. 30, 2018 | Jun. 30, 2019 | Jun. 30, 2018 | |
Accrued Expenses and Other Current Liabilities [Member] | ||||
Related Party Transaction [Line Items] | ||||
Accounts payable due to related party | $ 0.2 | $ 0.2 | ||
Research and Development Costs [Member] | ||||
Related Party Transaction [Line Items] | ||||
Related party transaction expense | $ 0.9 | $ 0.5 | $ 1.7 | $ 1.1 |
Net Income (Loss) Per Share - C
Net Income (Loss) Per Share - Computations of Basic and Diluted Net Income (Loss) Per Share (Detail) - USD ($) $ / shares in Units, shares in Thousands, $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2019 | Jun. 30, 2018 | Jun. 30, 2019 | Jun. 30, 2018 | |
Basic net income (loss) per share: | ||||
Net income (loss) applicable to common stockholders | $ (1,457) | $ 10,594 | $ (33,685) | $ 114,833 |
Shares used in computation: | ||||
Weighted-average common shares outstanding | 36,669 | 32,082 | 36,518 | 31,858 |
Basic net income (loss) per share | $ (0.04) | $ 0.33 | $ (0.92) | $ 3.60 |
Diluted net income (loss) per share: | ||||
Net income (loss) applicable to common stockholders | $ (1,457) | $ 10,594 | $ (33,685) | $ 114,833 |
Shares used in computation: | ||||
Weighted-average common shares outstanding | 36,669 | 32,082 | 36,518 | 31,858 |
Weighted-average diluted common shares | 36,669 | 35,272 | 36,518 | 34,884 |
Diluted net income (loss) per share | $ (0.04) | $ 0.30 | $ (0.92) | $ 3.29 |
Stock Option [Member] | ||||
Shares used in computation: | ||||
Weighted-average diluted common shares | 3,153 | 2,995 | ||
Restricted Stock Units [Member] | ||||
Shares used in computation: | ||||
Weighted-average diluted common shares | 30 | 27 | ||
Employee Stock Purchase Plan [Member] | ||||
Shares used in computation: | ||||
Weighted-average diluted common shares | 7 | 4 |
Net Income (Loss) Per Share - S
Net Income (Loss) Per Share - Schedules for Computation of Diluted Weighted-Average Shares Outstanding (Detail) - shares shares in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2019 | Jun. 30, 2018 | Jun. 30, 2019 | Jun. 30, 2018 | |
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | ||||
Anti-dilutive securities excluded from computation of diluted weighted average shares outstanding | 5,520 | 804 | 5,520 | 1,236 |
Stock Options Issued and Outstanding [Member] | ||||
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | ||||
Anti-dilutive securities excluded from computation of diluted weighted average shares outstanding | 5,454 | 804 | 5,454 | 1,236 |
Unvested Restricted Stock Units Outstanding [Member] | ||||
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | ||||
Anti-dilutive securities excluded from computation of diluted weighted average shares outstanding | 40 | 40 | ||
Employee Stock Purchase Plan [Member] | ||||
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | ||||
Anti-dilutive securities excluded from computation of diluted weighted average shares outstanding | 26 | 26 |
Supplemental Disclosures - Sche
Supplemental Disclosures - Schedules of Accrued Expenses and Other Current Liabilities (Detail) - USD ($) $ in Thousands | Jun. 30, 2019 | Dec. 31, 2018 |
Payables And Accruals [Abstract] | ||
Accrued personnel costs | $ 6,692 | $ 9,484 |
Accrued external research and development expenses | 4,925 | 4,274 |
Accrued sublicense fees and royalties | 1,514 | 1,617 |
Accrued external general and administrative expenses | 1,498 | 773 |
Accrued income taxes payable | 564 | 726 |
Accrued purchases of property and equipment | 397 | 221 |
Other accrued expenses and current liabilities | 944 | 69 |
Accrued expenses and other current liabilities | $ 16,534 | $ 17,164 |
Supplemental Disclosures - Addi
Supplemental Disclosures - Additional Information (Detail) - USD ($) $ in Thousands | Jun. 30, 2019 | Dec. 31, 2018 |
Payables And Accruals [Abstract] | ||
Other liabilities | $ 1,899 | $ 2,505 |