Document and Entity Information
Document and Entity Information - shares | 3 Months Ended | |
Mar. 31, 2019 | May 03, 2019 | |
Document And Entity Information [Abstract] | ||
Document Type | 10-Q | |
Amendment Flag | false | |
Document Period End Date | Mar. 31, 2019 | |
Document Fiscal Year Focus | 2019 | |
Document Fiscal Period Focus | Q1 | |
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 Common Stock, Shares Outstanding | 36,636,825 |
Consolidated Balance Sheets
Consolidated Balance Sheets - USD ($) $ in Thousands | Mar. 31, 2019 | Dec. 31, 2018 |
Current assets | ||
Cash and cash equivalents | $ 55,852 | $ 75,561 |
Marketable securities | 229,373 | 244,200 |
Accounts receivable | 8,372 | 8,587 |
Prepaid expenses | 6,292 | 5,734 |
Other current assets | 3,995 | 3,831 |
Total current assets | 303,884 | 337,913 |
Marketable securities | 159,083 | 150,819 |
Accounts receivable | 22,758 | 23,012 |
Property and equipment, net | 23,140 | 28,702 |
Operating lease right-of-use assets | 6,858 | |
Restricted cash | 1,053 | 1,053 |
Other assets | 2,255 | 2,315 |
Total assets | 519,031 | 543,814 |
Current liabilities | ||
Accounts payable | 4,204 | 4,412 |
Accrued expenses and other current liabilities | 14,189 | 17,164 |
Deferred revenue | 600 | 600 |
Operating lease liabilities | 2,397 | |
Total current liabilities | 21,390 | 22,176 |
Deferred revenue | 3,333 | 3,333 |
Operating lease liabilities | 5,483 | |
Deferred rent | 1,098 | |
Financing lease obligations | 5,854 | |
Other liabilities | 1,772 | 2,505 |
Total liabilities | 31,978 | 34,966 |
Commitments and contingencies (Note 6) | ||
Stockholders’ equity | ||
Preferred stock; $0.0001 par value; 10,000 shares authorized, and no shares issued and outstanding at March 31, 2019 and December 31, 2018 | ||
Common stock; $0.0001 par value; 100,000 shares authorized at March 31, 2019 and December 31, 2018; 36,611 and 36,120 shares issued and outstanding at March 31, 2019 and December 31, 2018, respectively | 4 | 4 |
Additional paid-in capital | 602,425 | 592,580 |
Accumulated other comprehensive loss | (59) | (720) |
Accumulated deficit | (115,317) | (83,016) |
Total stockholders’ equity | 487,053 | 508,848 |
Total liabilities and stockholders’ equity | $ 519,031 | $ 543,814 |
Consolidated Balance Sheets (Pa
Consolidated Balance Sheets (Parenthetical) - $ / shares | Mar. 31, 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 | 36,611,000 | 36,120,000 |
Common stock, shares outstanding | 36,611,000 | 36,120,000 |
Consolidated Statements of Oper
Consolidated Statements of Operations and Comprehensive Income (Loss) - USD ($) shares in Thousands, $ in Thousands | 3 Months Ended | |
Mar. 31, 2019 | Mar. 31, 2018 | |
Revenues | ||
Revenues | $ 884 | $ 132,391 |
Type of Revenue [Extensible List] | us-gaap:LicenseMember | us-gaap:LicenseMember |
Costs of revenues | ||
Costs of revenues | $ 29 | $ 2,408 |
Type of Cost, Good or Service [Extensible List] | us-gaap:LicenseMember | us-gaap:LicenseMember |
Research and development | $ 25,203 | $ 19,550 |
General and administrative | 11,558 | 8,380 |
Other operating expenses | 28 | |
Total operating expenses | 36,790 | 30,366 |
Income (loss) from operations | (35,906) | 102,025 |
Other Income | ||
Interest income from licensing | 613 | 1,355 |
Investment income | 2,995 | 859 |
Total other income | 3,608 | 2,214 |
Income (loss) before income taxes | (32,298) | 104,239 |
Income Tax Benefit | 70 | |
Net income (loss) | (32,228) | 104,239 |
Other Comprehensive Income (Loss) | ||
Unrealized gain (loss) on available-for-sale securities, net of reclassifications and income tax expense | 621 | (188) |
Total other comprehensive income (loss) | 621 | (188) |
Comprehensive income (loss) | (31,607) | 104,051 |
Net income (loss) applicable to common stockholders | $ (32,228) | $ 104,239 |
Net income (loss) per share: | ||
Basic | $ (0.89) | $ 3.30 |
Diluted | $ (0.89) | $ 3.04 |
Weighted-average common shares outstanding: | ||
Basic | 36,366 | 31,632 |
Diluted | 36,366 | 34,275 |
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 | ||||
Adoption of ASU | ASU 2014-09 [Member] | 4,803 | 4,803 | |||
Exercise of stock options | 3,824 | 3,824 | |||
Exercise of stock options, Shares | 586 | ||||
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 | 3,291 | 3,291 | |||
Unrealized gain (loss) on available-for-sale securities, net of reclassifications and income tax expense | (188) | (188) | |||
Net income (loss) | 104,239 | 104,239 | |||
Balances at Mar. 31, 2018 | 299,340 | $ 3 | 378,954 | (903) | (78,714) |
Balances (Shares) at Mar. 31, 2018 | 31,900 | ||||
Balances at Dec. 31, 2018 | 508,848 | $ 4 | 592,580 | (720) | (83,016) |
Balances (Shares) at Dec. 31, 2018 | 36,120 | ||||
Adoption of ASU | ASU 2016-02 [Member] | (33) | (33) | |||
Adoption of ASU | ASU 2018-02 [Member] | 40 | (40) | |||
Exercise of stock options | 3,762 | 3,762 | |||
Exercise of stock options, Shares | 481 | ||||
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 | 5,718 | 5,718 | |||
Unrealized gain (loss) on available-for-sale securities, net of reclassifications and income tax expense | 621 | 621 | |||
Net income (loss) | (32,228) | (32,228) | |||
Balances at Mar. 31, 2019 | $ 487,053 | $ 4 | $ 602,425 | $ (59) | $ (115,317) |
Balances (Shares) at Mar. 31, 2019 | 36,611 |
Consolidated Statements of Cash
Consolidated Statements of Cash Flows - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2019 | Mar. 31, 2018 | |
Cash flows from operating activities | ||
Net income (loss) | $ (32,228) | $ 104,239 |
Adjustments to reconcile net income (loss) to net cash provided by (used in) operating activities | ||
Stock-based compensation expense | 5,718 | 3,291 |
Net amortization of premiums and accretion of discounts on marketable debt securities | (368) | 378 |
Depreciation and amortization | 1,614 | 834 |
Imputed interest income from licensing | (613) | (1,355) |
Other non-cash adjustments | 327 | 10 |
Changes in operating assets and liabilities | ||
Accounts receivable | 591 | (51,414) |
Prepaid expenses | (790) | 667 |
Other current assets | (295) | (446) |
Operating lease right-of-use assets | 573 | |
Other assets | 26 | (21) |
Accounts payable | 316 | 477 |
Accrued expenses and other current liabilities | (2,932) | 490 |
Operating lease liabilities | (592) | |
Deferred rent | 27 | |
Other liabilities | (644) | 957 |
Net cash provided by (used in) operating activities | (29,297) | 58,134 |
Cash flows from investing activities | ||
Purchases of marketable securities | (79,249) | (54,267) |
Maturities of marketable securities | 87,165 | 19,525 |
Purchases of property and equipment | (2,455) | (2,344) |
Net cash provided by (used in) investing activities | 5,461 | (37,086) |
Cash flows from financing activities | ||
Proceeds from exercise of stock options | 3,762 | 3,824 |
Proceeds from issuance of common stock under employee stock purchase plan | 365 | 342 |
Net cash provided by financing activities | 4,127 | 4,166 |
Net increase (decrease) in cash and cash equivalents and restricted cash | (19,709) | 25,214 |
Cash and cash equivalents and restricted cash | ||
Beginning of period | 76,614 | 46,881 |
End of period | $ 56,905 | $ 72,095 |
Nature of Business
Nature of Business | 3 Months Ended |
Mar. 31, 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 March 31, 2019, the Company had generated an accumulated deficit of $115.3 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 March 31, 2019, the Company had cash, cash equivalents and marketable securities of $444.3 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 | 3 Months Ended |
Mar. 31, 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 include all normal and recurring adjustments necessary for the fair statement of the Company’s financial position as of March 31, 2019, and the results of its operations and its cash flows for the interim periods ended March 31, 2019 and 2018. 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: revenue, stock-based compensation expense, accrued research and development expenses and other accrued liabilities, income taxes and the fair value of financial instruments. 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): March 31, 2019 March 31, 2018 Cash and cash equivalents $ 55,852 $ 71,870 Restricted cash 1,053 225 Total cash and cash equivalents and restricted cash $ 56,905 $ 72,095 Marketable Securities Marketable securities consist of debt securities and are classified as available-for-sale and carried at fair value. Marketable 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. Unrealized gains and losses, 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 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. 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. A decline in the fair value below cost of available-for-sale 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 investments 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 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 applies a dual approach to all leases in which it is a lessee and classifies 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 March 31, 2019 have been classified as operating leases. Operating lease expense is recognized on a straight-line basis over the term of the lease. Variable lease expense is 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. 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. 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 months ended March 31, 2019, nor does the Company believe it will have a material impact on future results of operations based on its current leasing arrangements. Other recently adopted accounting pronouncements 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 2018-13, 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 | 3 Months Ended |
Mar. 31, 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 solely of available-for-sale debt securities (in thousands): Amortized Cost Unrealized Gains Unrealized Losses Fair Value March 31, 2019 U.S. government and federal agency securities $ 105,533 $ 202 $ (3 ) $ 105,732 Certificates of deposit 9,237 42 — 9,279 Corporate bonds 272,988 502 (45 ) 273,445 $ 387,758 $ 746 $ (48 ) $ 388,456 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 March 31, 2019 and December 31, 2018, no available-for-sale securities had remaining maturities greater than three years. The amortized cost of available-for-sale 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 March 31, 2019 and December 31, 2018, the balance in the Company’s accumulated other comprehensive loss consisted solely of net unrealized gains and losses on available-for-sale securities, net of income tax effects and reclassification adjustments for realized gains and losses. During the three months ended March 31, 2019, the Company recognized net unrealized gains on available-for-sale securities of $1.0 million and income tax expense of $0.4 million in other comprehensive income for the period. The Company recognized net realized gains of less than $0.1 million on the sale or maturity of available-for-sale securities during the three months ended March 31, 2019, which were reclassified out of accumulated other comprehensive loss during the period and are included in investment income in the consolidated statements of operations and comprehensive income (loss). During the three months ended March 31, 2018, the Company recognized net unrealized losses on available-for-sale securities of $0.2 million and income tax expense of zero in other comprehensive loss for the period. The Company did not recognize any realized gains or losses on the sale or maturity of available-for-sale securities during the three months ended March 31, 2018. Realized gains and losses from the sale or maturity of marketable securities are determined based on the specific identification method. The following tables present the 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 (in thousands): Less than 12 Months 12 Months or Greater Total Fair Value Unrealized Losses Fair Value Unrealized Losses Fair Value Unrealized Losses March 31, 2019 U.S. government and federal agency securities $ 13,923 $ (3 ) $ — $ — $ 13,923 $ (3 ) Corporate bonds 110,103 (44 ) 1,999 (1 ) 112,102 (45 ) $ 124,026 $ (47 ) $ 1,999 $ (1 ) $ 126,025 $ (48 ) 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 March 31, 2019, marketable securities held by the Company which were in an unrealized loss position consisted of 40 investment grade security positions. The Company has the intent and ability to hold such securities until recovery and has determined that none of its investments were other-than-temporarily impaired as of March 31, 2019 or December 31, 2018. |
Fair Value of Financial Instrum
Fair Value of Financial Instruments | 3 Months Ended |
Mar. 31, 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 March 31, 2019 Cash equivalents: Money market mutual funds $ — $ 55,834 $ — $ 55,834 Total cash equivalents — 55,834 — 55,834 Marketable securities: U.S. government and federal agency securities — 105,732 — 105,732 Certificates of deposit — 9,279 — 9,279 Corporate bonds — 273,445 — 273,445 Total marketable securities — 388,456 — 388,456 Total cash equivalents and marketable securities $ — $ 444,290 $ — $ 444,290 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 three months ended March 31, 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 March 31, 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. The Company’s 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 March 31, 2019 and December 31, 2018, non-marketable equity securities had carrying values of $0.4 million and were included in other assets on the consolidated balance sheets. Since the acquisition of the securities, the Company has not identified any observable price changes or changes in circumstances that would have had an adverse effect on the fair value of the securities as of March 31, 2019 and December 31, 2018. No remeasurements or impairment losses were recorded on non-marketable equity securities during the three months ended March 31, 2019 and 2018. |
Property and Equipment, Net
Property and Equipment, Net | 3 Months Ended |
Mar. 31, 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): March 31, 2019 December 31, 2018 Lab equipment $ 15,563 $ 14,417 Computer equipment and software 2,104 2,002 Furniture and fixtures 1,935 1,915 Leasehold improvements 12,371 11,751 Construction-in-progress — 5,854 Total property and equipment 31,973 35,939 Accumulated depreciation and amortization (8,833 ) (7,237 ) Property and equipment, net $ 23,140 $ 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 agreement for the facility at 9800 Medical Center Drive. |
Commitments and Contingencies
Commitments and Contingencies | 3 Months Ended |
Mar. 31, 2019 | |
Commitments And Contingencies Disclosure [Abstract] | |
Commitments and Contingencies | 6. Commitments and Contingencies Lease Commitments 9800 Medical Center Drive Lease In November 2018, the Company entered into a lease for approximately 132,000 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). Construction of the new building, which will be conducted by the landlord, is expected to be completed in 2020 and the lease will expire approximately 16 years from the delivery of the leased premises to the Company, subject to certain extension and termination options. Under the original terms of the 9800 Medical Center Drive Lease, which was amended in April 2019 as discussed further below, the Company was entitled to receive a $14.6 million tenant improvement allowance from the landlord to construct additional improvements to the leased premises. 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 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 the 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 March 31, 2019, the Company had incurred $0.3 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. In April 2019, the Company agreed to pay $4.0 million to the landlord to fund certain costs of construction related to material changes in the design and construction of the building requested by the Company. 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 as of March 31, 2019 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 facility to the Company. In April 2019, the Company amended the 9800 Medical Center Drive Lease to expand the leased premises to include an additional 5,975 square feet of the building over the term of the lease. As a result of the amendment, the total amount of future rent under the 9800 Medical Center Drive Lease was increased by $4.0 million and the Company’s tenant improvement allowance under the lease was increased to $15.3 million. Other Lease Commitments In March 2015, the Company entered into a non-cancelable 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 a 7.5-year, non-cancelable 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. 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. 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 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. The Company leases additional office and laboratory facilities in Rockville, Maryland and New York, New York, as well as laboratory and other equipment, under non-cancelable operating leases with various expiration dates through 2022 and which may contain annual escalations of rental payments. As required by the Company’s lease agreement for its office space in New York, New York, 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. 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 March 31, 2019 Operating lease cost $ 700 Variable lease cost 161 Total lease cost $ 861 Cash paid for amounts included in the measurement of operating lease liabilities $ 711 Right-of-use assets acquired through operating lease liabilities $ 36 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 March 31, 2019 Weighted-average remaining lease term (years) 3.1 Weighted-average discount rate 5.6 % The following table presents a reconciliation of the undiscounted future minimum lease payments remaining under the 9800 Medical Center Drive Lease and other operating leases to the amounts reported as operating lease liabilities on the consolidated balance sheet as of March 31, 2019 (in thousands): 9800 Medical Other Total Center Drive Operating Undiscounted Lease (a) Leases Fixed Payments Undiscounted future minimum lease payments: 2019 (remainder of year) $ — $ 1,995 $ 1,995 2020 — 3,088 3,088 2021 1,332 2,412 3,744 2022 4,308 623 4,931 2023 5,188 479 5,667 Thereafter 76,892 — 76,892 Total undiscounted future minimum lease payments $ 87,720 $ 8,597 $ 96,317 Amount representing imputed interest (717 ) Total operating lease liabilities 7,880 Current portion of operating lease liabilities (2,397 ) Operating lease liabilities, non-current $ 5,483 (a) Undiscounted future minimum lease payments under the 9800 Medical Center Drive Lease are not included in the lease liabilities reported on the consolidated balance sheet as of March 31, 2019 as the lease has not yet commenced. The actual timing and amounts of payments under the 9800 Medical Center Drive Lease are subject to adjustment based on the completion date of construction and actual square footage of the facility constructed. Accordingly, these amounts were estimates as of March 31, 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 completion date of construction and actual square footage of the facility constructed. Accordingly, these amounts were estimates as of December 31, 2018. 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). In consideration for the license, the Company issued Penn an equity interest in 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 is obligated to pay Penn an upfront fee, 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 March 31, 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 Revenue
License Revenue | 3 Months Ended |
Mar. 31, 2019 | |
License Agreement Revenue Recognition [Abstract] | |
License Revenue | 7. License Revenue As of March 31, 2019, the Company’s NAV Technology Platform was being applied in the development of more than 20 partnered product candidates by 11 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. To date the Company has not recognized any revenue from the achievement of sales-based milestones or royalties on sales of licensed products. 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 March 31, 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 $29.6 million upon the commencement of various stages of clinical trials, $47.5 million upon the submission of regulatory approval filings, $117.5 million upon the approval of commercial products by regulatory agencies and $232.0 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 March 31, 2019 Receivables and contract assets: Accounts receivable, current and non-current $ 31,599 $ (469 ) $ 31,130 Contract assets $ 750 $ 250 $ 1,000 Contract liabilities: Deferred revenue, current and non-current $ 3,933 $ — $ 3,933 Balance at Beginning Net Additions Balance at of Period (Deductions) End of Period Three Months Ended March 31, 2018 Receivables and contract assets: Accounts receivable, current and non-current $ 5,850 $ 52,762 $ 58,612 Contract assets $ 350 $ (100 ) $ 250 Contract liabilities: Deferred revenue, current and non-current $ — $ — $ — The net increase in the balance of accounts receivable during the three months ended March 31, 2018 was primarily attributable to the January 2018 amendment to the license agreement with AveXis, Inc. (AveXis) for the development and commercialization of treatments for spinal muscular atrophy (SMA). As of March 31, 2018, the Company had recorded accounts receivable, current and non-current, of $53.5 million related to the amended license agreement with AveXis. As of March 31, 2019, the Company had recorded deferred revenue, current and non-current, of $3.9 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 months ended March 31, 2019 and 2018, the Company recognized license revenue of $0.8 million and $0.3 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 price were primarily attributable to development milestones achieved or deemed probable of achievement during the period that were previously not considered probable of achievement. As of March 31, 2019, the Company had recorded total current and non-current accounts receivable of $31.1 million, of which $0.1 million had been billed to customers and $31.0 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 March 31, 2019 and December 2018 included $26.7 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 March 31, 2019 and December 31, 2018, the Company had not recognized any impairment losses on its receivables or contract assets from contracts with customers. 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 AG (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. 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 March 31, 2019, the transaction price of the original March 2014 License was $7.5 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 $7.0 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, if any. 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 March 31, 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, if any. During the three months ended March 31, 2019 and 2018, the Company recognized license revenue of zero and $132.1 million, respectively, and interest income from licensing of less than $0.1 million and $1.2 million, respectively, from the March 2014 License, as amended, which includes amounts from both the original March 2014 License and the January 2018 Amendment. As of March 31, 2019 and 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 on the consolidated balance sheets. |
Stock-based Compensation
Stock-based Compensation | 3 Months Ended |
Mar. 31, 2019 | |
Disclosure Of Compensation Related Costs Sharebased Payments [Abstract] | |
Stock-based Compensation | 8. 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 March 31, 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 2,583,144 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 March 31, 2019 2018 Stock options $ 5,452 $ 3,122 Restricted stock units 68 68 Employee stock purchase plan 198 101 $ 5,718 $ 3,291 As of March 31, 2019, the Company had $68.9 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 3.1 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 March 31, 2019 2018 Research and development $ 2,347 $ 1,530 General and administrative 3,371 1,761 $ 5,718 $ 3,291 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 1,102 $ 48.22 Exercised (480 ) $ 7.81 Cancelled or forfeited (137 ) $ 30.72 Outstanding at March 31, 2019 5,340 $ 26.02 7.9 $ 170,518 Exercisable at March 31, 2019 2,491 $ 12.22 6.8 $ 112,324 Vested and expected to vest at March 31, 2019 5,340 $ 26.02 7.9 $ 170,518 (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 three months ended March 31, 2019 was $32.20. During the three months ended March 31, 2019, the total number of stock options exercised was 480,320, resulting in total proceeds of $3.8 million. The total intrinsic value of options exercised during the three months ended March 31, 2019 was $20.0 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 March 31, 2019 40 $ 20.90 Employee Stock Purchase Plan As of March 31, 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 three months ended March 31, 2019, 10,241 shares of common stock were issued under the 2015 ESPP. |
Income Taxes
Income Taxes | 3 Months Ended |
Mar. 31, 2019 | |
Income Tax Disclosure [Abstract] | |
Income Taxes | 9. 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 March 31, 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 March 31, 2019 and December 31, 2018. During the three months ended March 31, 2019, the Company recognized income tax benefit of $0.1 million and income tax expense in other comprehensive income of $0.4 million related to net unrealized gains on available-for-sale securities during the period. As of March 31, 2019, the Company had accrued $0.3 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 sheets. |
Related Party Transactions
Related Party Transactions | 3 Months Ended |
Mar. 31, 2019 | |
Related Party Transactions [Abstract] | |
Related Party Transactions | 10. 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 and which has a term of one year and is terminable by either party. Expenses incurred under the agreements with FOXKISER for the three months ended March 31, 2019 and 2018 were $0.8 million and $0.5 million, respectively, and were recorded as research and development expenses in the consolidated statements of operations and comprehensive income (loss). |
Net Income (Loss) Per Share
Net Income (Loss) Per Share | 3 Months Ended |
Mar. 31, 2019 | |
Earnings Per Share [Abstract] | |
Net Income (Loss) Per Share | 11. 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 March 31, 2019 2018 Basic net income (loss) per share: Net income (loss) applicable to common stockholders $ (32,228 ) $ 104,239 Shares used in computation: Weighted-average common shares outstanding 36,366 31,632 Basic net income (loss) per share $ (0.89 ) $ 3.30 Diluted net income (loss) per share: Net income (loss) applicable to common stockholders $ (32,228 ) $ 104,239 Shares used in computation: Weighted-average common shares outstanding 36,366 31,632 Stock options — 2,620 Restricted stock units — 22 Employee stock purchase plan — 1 Weighted-average diluted common shares 36,366 34,275 Diluted net income (loss) per share $ (0.89 ) $ 3.04 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 March 31, 2019 2018 Stock options issued and outstanding 5,340 1,582 Unvested restricted stock units outstanding 40 — Employee stock purchase plan 20 — 5,400 1,582 |
Supplemental Disclosures
Supplemental Disclosures | 3 Months Ended |
Mar. 31, 2019 | |
Payables And Accruals [Abstract] | |
Supplemental Disclosures | 12. Supplemental Disclosures Accrued expenses and other current liabilities consists of the following (in thousands): March 31, 2019 December 31, 2018 Accrued external research and development expenses $ 5,474 $ 4,274 Accrued personnel costs 5,357 9,484 Accrued income taxes payable 1,134 726 Accrued licensing costs 911 1,617 Accrued external general and administrative expenses 854 773 Accrued purchases of property and equipment 191 221 Other accrued expenses and current liabilities 268 69 $ 14,189 $ 17,164 Other liabilities of $1.8 million and $2.5 million reported as of March 31, 2019 and December 31, 2018, respectively, consist of accrued licensing costs payable in periods beyond 12 months from the reporting date. |
Summary of Significant Accoun_2
Summary of Significant Accounting Policies (Policies) | 3 Months Ended |
Mar. 31, 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 include all normal and recurring adjustments necessary for the fair statement of the Company’s financial position as of March 31, 2019, and the results of its operations and its cash flows for the interim periods ended March 31, 2019 and 2018. 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: revenue, stock-based compensation expense, accrued research and development expenses and other accrued liabilities, income taxes and the fair value of financial instruments. |
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): March 31, 2019 March 31, 2018 Cash and cash equivalents $ 55,852 $ 71,870 Restricted cash 1,053 225 Total cash and cash equivalents and restricted cash $ 56,905 $ 72,095 |
Marketable Securities | Marketable Securities Marketable securities consist of debt securities and are classified as available-for-sale and carried at fair value. Marketable 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. Unrealized gains and losses, 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 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. 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. A decline in the fair value below cost of available-for-sale 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 investments 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 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 applies a dual approach to all leases in which it is a lessee and classifies 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 March 31, 2019 have been classified as operating leases. Operating lease expense is recognized on a straight-line basis over the term of the lease. Variable lease expense is 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. |
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. 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 months ended March 31, 2019, nor does the Company believe it will have a material impact on future results of operations based on its current leasing arrangements. Other recently adopted accounting pronouncements 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 2018-13, 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) | 3 Months Ended |
Mar. 31, 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): March 31, 2019 March 31, 2018 Cash and cash equivalents $ 55,852 $ 71,870 Restricted cash 1,053 225 Total cash and cash equivalents and restricted cash $ 56,905 $ 72,095 |
Marketable Securities (Tables)
Marketable Securities (Tables) | 3 Months Ended |
Mar. 31, 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 solely of available-for-sale debt securities (in thousands): Amortized Cost Unrealized Gains Unrealized Losses Fair Value March 31, 2019 U.S. government and federal agency securities $ 105,533 $ 202 $ (3 ) $ 105,732 Certificates of deposit 9,237 42 — 9,279 Corporate bonds 272,988 502 (45 ) 273,445 $ 387,758 $ 746 $ (48 ) $ 388,456 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 marketable 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 March 31, 2019 U.S. government and federal agency securities $ 13,923 $ (3 ) $ — $ — $ 13,923 $ (3 ) Corporate bonds 110,103 (44 ) 1,999 (1 ) 112,102 (45 ) $ 124,026 $ (47 ) $ 1,999 $ (1 ) $ 126,025 $ (48 ) 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) | 3 Months Ended |
Mar. 31, 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 March 31, 2019 Cash equivalents: Money market mutual funds $ — $ 55,834 $ — $ 55,834 Total cash equivalents — 55,834 — 55,834 Marketable securities: U.S. government and federal agency securities — 105,732 — 105,732 Certificates of deposit — 9,279 — 9,279 Corporate bonds — 273,445 — 273,445 Total marketable securities — 388,456 — 388,456 Total cash equivalents and marketable securities $ — $ 444,290 $ — $ 444,290 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) | 3 Months Ended |
Mar. 31, 2019 | |
Property Plant And Equipment [Abstract] | |
Schedule of Property and Equipment | Property and equipment, net consists of the following (in thousands): March 31, 2019 December 31, 2018 Lab equipment $ 15,563 $ 14,417 Computer equipment and software 2,104 2,002 Furniture and fixtures 1,935 1,915 Leasehold improvements 12,371 11,751 Construction-in-progress — 5,854 Total property and equipment 31,973 35,939 Accumulated depreciation and amortization (8,833 ) (7,237 ) Property and equipment, net $ 23,140 $ 28,702 |
Commitments and Contingencies (
Commitments and Contingencies (Tables) | 3 Months Ended |
Mar. 31, 2019 | |
Commitments And Contingencies Disclosure [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 March 31, 2019 Operating lease cost $ 700 Variable lease cost 161 Total lease cost $ 861 Cash paid for amounts included in the measurement of operating lease liabilities $ 711 Right-of-use assets acquired through operating lease liabilities $ 36 |
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 March 31, 2019 Weighted-average remaining lease term (years) 3.1 Weighted-average discount rate 5.6 % |
Reconciliation of Undiscounted Future Minimum Lease Payments Remaining Under 9800 Medical Center Drive Lease and Other Operating Leases | The following table presents a reconciliation of the undiscounted future minimum lease payments remaining under the 9800 Medical Center Drive Lease and other operating leases to the amounts reported as operating lease liabilities on the consolidated balance sheet as of March 31, 2019 (in thousands): 9800 Medical Other Total Center Drive Operating Undiscounted Lease (a) Leases Fixed Payments Undiscounted future minimum lease payments: 2019 (remainder of year) $ — $ 1,995 $ 1,995 2020 — 3,088 3,088 2021 1,332 2,412 3,744 2022 4,308 623 4,931 2023 5,188 479 5,667 Thereafter 76,892 — 76,892 Total undiscounted future minimum lease payments $ 87,720 $ 8,597 $ 96,317 Amount representing imputed interest (717 ) Total operating lease liabilities 7,880 Current portion of operating lease liabilities (2,397 ) Operating lease liabilities, non-current $ 5,483 (a) Undiscounted future minimum lease payments under the 9800 Medical Center Drive Lease are not included in the lease liabilities reported on the consolidated balance sheet as of March 31, 2019 as the lease has not yet commenced. The actual timing and amounts of payments under the 9800 Medical Center Drive Lease are subject to adjustment based on the completion date of construction and actual square footage of the facility constructed. Accordingly, these amounts were estimates as of March 31, 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 completion date of construction and actual square footage of the facility constructed. Accordingly, these amounts were estimates as of December 31, 2018. |
License Revenue (Tables)
License Revenue (Tables) | 3 Months Ended |
Mar. 31, 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 March 31, 2019 Receivables and contract assets: Accounts receivable, current and non-current $ 31,599 $ (469 ) $ 31,130 Contract assets $ 750 $ 250 $ 1,000 Contract liabilities: Deferred revenue, current and non-current $ 3,933 $ — $ 3,933 Balance at Beginning Net Additions Balance at of Period (Deductions) End of Period Three Months Ended March 31, 2018 Receivables and contract assets: Accounts receivable, current and non-current $ 5,850 $ 52,762 $ 58,612 Contract assets $ 350 $ (100 ) $ 250 Contract liabilities: Deferred revenue, current and non-current $ — $ — $ — |
Stock-based Compensation (Table
Stock-based Compensation (Tables) | 3 Months Ended |
Mar. 31, 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 March 31, 2019 2018 Stock options $ 5,452 $ 3,122 Restricted stock units 68 68 Employee stock purchase plan 198 101 $ 5,718 $ 3,291 |
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 March 31, 2019 2018 Research and development $ 2,347 $ 1,530 General and administrative 3,371 1,761 $ 5,718 $ 3,291 |
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 March 31, 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 1,102 $ 48.22 Exercised (480 ) $ 7.81 Cancelled or forfeited (137 ) $ 30.72 Outstanding at March 31, 2019 5,340 $ 26.02 7.9 $ 170,518 Exercisable at March 31, 2019 2,491 $ 12.22 6.8 $ 112,324 Vested and expected to vest at March 31, 2019 5,340 $ 26.02 7.9 $ 170,518 (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) | 3 Months Ended |
Mar. 31, 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 March 31, 2019 2018 Basic net income (loss) per share: Net income (loss) applicable to common stockholders $ (32,228 ) $ 104,239 Shares used in computation: Weighted-average common shares outstanding 36,366 31,632 Basic net income (loss) per share $ (0.89 ) $ 3.30 Diluted net income (loss) per share: Net income (loss) applicable to common stockholders $ (32,228 ) $ 104,239 Shares used in computation: Weighted-average common shares outstanding 36,366 31,632 Stock options — 2,620 Restricted stock units — 22 Employee stock purchase plan — 1 Weighted-average diluted common shares 36,366 34,275 Diluted net income (loss) per share $ (0.89 ) $ 3.04 |
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 March 31, 2019 2018 Stock options issued and outstanding 5,340 1,582 Unvested restricted stock units outstanding 40 — Employee stock purchase plan 20 — 5,400 1,582 |
Supplemental Disclosures (Table
Supplemental Disclosures (Tables) | 3 Months Ended |
Mar. 31, 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): March 31, 2019 December 31, 2018 Accrued external research and development expenses $ 5,474 $ 4,274 Accrued personnel costs 5,357 9,484 Accrued income taxes payable 1,134 726 Accrued licensing costs 911 1,617 Accrued external general and administrative expenses 854 773 Accrued purchases of property and equipment 191 221 Other accrued expenses and current liabilities 268 69 $ 14,189 $ 17,164 |
Nature of Business - Additional
Nature of Business - Additional Information (Detail) - USD ($) $ in Thousands | Mar. 31, 2019 | Dec. 31, 2018 |
Organization Consolidation And Presentation Of Financial Statements [Abstract] | ||
Accumulated deficit | $ (115,317) | $ (83,016) |
Cash, cash equivalents and marketable securities | $ 444,300 |
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 | Mar. 31, 2019 | Dec. 31, 2018 | Mar. 31, 2018 |
Accounting Policies [Abstract] | |||
Cash and cash equivalents | $ 55,852 | $ 75,561 | $ 71,870 |
Restricted cash | 1,053 | $ 1,053 | 225 |
Total cash and cash equivalents and restricted cash | $ 56,905 | $ 72,095 |
Summary of Significant Accoun_5
Summary of Significant Accounting Policies - Additional Information (Detail) - USD ($) | Jan. 01, 2019 | Mar. 31, 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 | 6,858,000 | ||
Property and equipment, net | 23,140,000 | $ 28,702,000 | |
Financing lease obligation | 5,854,000 | ||
Accumulated deficit | $ (115,317,000) | $ (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] | 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 loss to accumulated deficit | $ 100,000 |
Marketable Securities - Summary
Marketable Securities - Summary of Company Marketable Securities (Detail) - USD ($) $ in Thousands | Mar. 31, 2019 | Dec. 31, 2018 |
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost | $ 387,758 | $ 395,304 |
Unrealized Gains | 746 | 129 |
Unrealized Losses | (48) | (414) |
Fair Value | 388,456 | 395,019 |
U.S. Government and Federal Agency Securities [Member] | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost | 105,533 | 103,410 |
Unrealized Gains | 202 | 93 |
Unrealized Losses | (3) | (37) |
Fair Value | 105,732 | 103,466 |
Certificates of Deposit [Member] | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost | 9,237 | 8,992 |
Unrealized Gains | 42 | |
Fair Value | 9,279 | 8,992 |
Corporate Bonds [Member] | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost | 272,988 | 282,902 |
Unrealized Gains | 502 | 36 |
Unrealized Losses | (45) | (377) |
Fair Value | $ 273,445 | $ 282,561 |
Marketable Securities - Additio
Marketable Securities - Additional Information (Detail) | 3 Months Ended | 12 Months Ended | |
Mar. 31, 2019USD ($)Security | Mar. 31, 2018USD ($) | Dec. 31, 2018USD ($) | |
Schedule Of Available For Sale Securities [Line Items] | |||
Available for sale securities remaining maturities greater than three years | $ 0 | $ 0 | |
Unrealized gains (losses) on available-for-sale securities, before tax | 1,000,000 | $ (200,000) | |
Unrealized gains (losses) on available-for-sale securities, income tax expense | $ 400,000 | $ 0 | |
Number of investment grade fixed income security | Security | 40 | ||
Other-than-temporary impaired | $ 0 | $ 0 | |
Investment Income [Member] | Maximum [Member] | |||
Schedule Of Available For Sale Securities [Line Items] | |||
Net realized gains or losses available for sale securities | $ 100,000 |
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 | Mar. 31, 2019 | Dec. 31, 2018 |
Schedule Of Available For Sale Securities [Line Items] | ||
Less than 12 Months, Fair Value | $ 124,026 | |
Less than 12 Months, Unrealized Losses | (47) | $ (391) |
12 Months or Greater, Fair Value | 1,999 | |
12 Months or Greater, Unrealized Losses | (1) | (23) |
Total, Fair Value | 126,025 | |
Total, Unrealized Losses | (48) | (414) |
Less than 12 Months, Fair Value | 298,407 | |
12 Months or Greater, Fair Value | 12,424 | |
Total, Fair Value | 310,831 | |
U.S. Government and Federal Agency Securities [Member] | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Less than 12 Months, Fair Value | 13,923 | |
Less than 12 Months, Unrealized Losses | (3) | (37) |
Total, Fair Value | 13,923 | |
Total, Unrealized Losses | (3) | (37) |
Less than 12 Months, Fair Value | 53,124 | |
Total, Fair Value | 53,124 | |
Corporate Bonds [Member] | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Less than 12 Months, Fair Value | 110,103 | |
Less than 12 Months, Unrealized Losses | (44) | (354) |
12 Months or Greater, Fair Value | 1,999 | |
12 Months or Greater, Unrealized Losses | (1) | (23) |
Total, Fair Value | 112,102 | |
Total, Unrealized Losses | $ (45) | (377) |
Less than 12 Months, Fair Value | 245,283 | |
12 Months or Greater, Fair Value | 12,424 | |
Total, Fair Value | $ 257,707 |
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 | Mar. 31, 2019 | Dec. 31, 2018 |
Marketable securities: | ||
Fair Value | $ 388,456 | $ 395,019 |
Cash Equivalents and Marketable Securities [Member] | ||
Marketable securities: | ||
Total cash equivalents and marketable securities | 444,290 | 470,561 |
Corporate Bonds [Member] | ||
Marketable securities: | ||
Fair Value | 273,445 | 282,561 |
Cash Equivalents [Member] | ||
Cash equivalents: | ||
Total cash equivalents | 55,834 | 75,542 |
U.S. Government and Federal Agency Securities [Member] | ||
Marketable securities: | ||
Fair Value | 105,732 | 103,466 |
Certificates of Deposit [Member] | ||
Marketable securities: | ||
Fair Value | 9,279 | 8,992 |
Money Market Mutual Funds [Member] | Cash Equivalents [Member] | ||
Cash equivalents: | ||
Total cash equivalents | 55,834 | 75,542 |
Significant Other Observable Inputs (Level 2) [Member] | ||
Marketable securities: | ||
Fair Value | 388,456 | 395,019 |
Significant Other Observable Inputs (Level 2) [Member] | Cash Equivalents and Marketable Securities [Member] | ||
Marketable securities: | ||
Total cash equivalents and marketable securities | 444,290 | 470,561 |
Significant Other Observable Inputs (Level 2) [Member] | Corporate Bonds [Member] | ||
Marketable securities: | ||
Fair Value | 273,445 | 282,561 |
Significant Other Observable Inputs (Level 2) [Member] | Cash Equivalents [Member] | ||
Cash equivalents: | ||
Total cash equivalents | 55,834 | 75,542 |
Significant Other Observable Inputs (Level 2) [Member] | U.S. Government and Federal Agency Securities [Member] | ||
Marketable securities: | ||
Fair Value | 105,732 | 103,466 |
Significant Other Observable Inputs (Level 2) [Member] | Certificates of Deposit [Member] | ||
Marketable securities: | ||
Fair Value | 9,279 | 8,992 |
Significant Other Observable Inputs (Level 2) [Member] | Money Market Mutual Funds [Member] | Cash Equivalents [Member] | ||
Cash equivalents: | ||
Total cash equivalents | $ 55,834 | $ 75,542 |
Fair Value of Financial Instr_4
Fair Value of Financial Instruments - Additional Information (Detail) - USD ($) | 3 Months Ended | ||
Mar. 31, 2019 | Mar. 31, 2018 | Dec. 31, 2018 | |
Fair Value Disclosures [Abstract] | |||
Fair value hierarchy level 1 to level 2 transfers amount | $ 0 | ||
Fair value hierarchy level 2 to level 1 transfers amount | 0 | ||
Non-marketable equity securities | 400,000 | $ 400,000 | |
Remeasurements or impairment losses on non-marketable equity securities | $ 0 | $ 0 |
Property and Equipment Net - Sc
Property and Equipment Net - Schedule of Property and Equipment (Detail) - USD ($) $ in Thousands | Mar. 31, 2019 | Dec. 31, 2018 |
Property Plant And Equipment [Line Items] | ||
Total property and equipment | $ 31,973 | $ 35,939 |
Accumulated depreciation and amortization | (8,833) | (7,237) |
Property and equipment, net | 23,140 | 28,702 |
Lab Equipment [Member] | ||
Property Plant And Equipment [Line Items] | ||
Total property and equipment | 15,563 | 14,417 |
Computer Equipment and Software [Member] | ||
Property Plant And Equipment [Line Items] | ||
Total property and equipment | 2,104 | 2,002 |
Furniture and Fixtures [Member] | ||
Property Plant And Equipment [Line Items] | ||
Total property and equipment | 1,935 | 1,915 |
Leasehold Improvements [Member] | ||
Property Plant And Equipment [Line Items] | ||
Total property and equipment | $ 12,371 | 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 | Mar. 31, 2019 | Jan. 01, 2019 | Dec. 31, 2018 |
Property Plant And Equipment [Line Items] | |||
Property and equipment, net | $ 23,140 | $ 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) |
Commitments and Contingencies -
Commitments and Contingencies - Additional Information (Detail) | 1 Months Ended | 3 Months Ended | |||||||
Apr. 30, 2019USD ($)ft² | Nov. 30, 2018USD ($)ft² | Apr. 30, 2018USD ($) | Nov. 30, 2017USD ($) | Jan. 31, 2016 | Mar. 31, 2015 | Mar. 31, 2019USD ($) | Jan. 01, 2019USD ($) | Dec. 31, 2018USD ($) | |
Other Commitments [Line Items] | |||||||||
Property and equipment, net | $ 23,140,000 | $ 28,702,000 | |||||||
Financing lease obligation | 5,854,000 | ||||||||
European Patent Office Proceeding [Member] | |||||||||
Other Commitments [Line Items] | |||||||||
Liability related to third party opposition | $ 0 | $ 0 | |||||||
Subsequent Event [Member] | University of Pennsylvania [Member] | Maximum [Member] | |||||||||
Other Commitments [Line Items] | |||||||||
Upfront fee, milestone fees | $ 20,500,000 | ||||||||
Rockville, Maryland and New York, New York [Member] | |||||||||
Other Commitments [Line Items] | |||||||||
Operating leases expiration year | 2022 | ||||||||
New York [Member] | |||||||||
Other Commitments [Line Items] | |||||||||
Letters of credit | $ 200,000 | ||||||||
9800 Medical Center Drive Lease [Member] | Subsequent Event [Member] | |||||||||
Other Commitments [Line Items] | |||||||||
Payment to landlord | $ 4,000,000 | ||||||||
9800 Medical Center Drive Lease [Member] | Leasehold Improvements [Member] | |||||||||
Other Commitments [Line Items] | |||||||||
Property and equipment, net | $ 300,000 | ||||||||
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] | |||||||||
Number of square feet to be constructed | ft² | 132,000 | ||||||||
Construction completion, description | Construction of the new building, which will be conducted by the landlord, is expected to be completed in 2020 | ||||||||
Lease term, from the delivery of leased premises | 16 years | ||||||||
Tenant improvement allowance entitled to received | $ 14,600,000 | ||||||||
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 | ||||||||
9800 Medical Center Drive Lease [Member] | Rockville, Maryland [Member] | Subsequent Event [Member] | |||||||||
Other Commitments [Line Items] | |||||||||
Number of square feet to be constructed | ft² | 5,975 | ||||||||
Change in tenant improvement allowance | $ 15,300,000 | ||||||||
Increased on future rent | $ 4,000,000 | ||||||||
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, existense 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, existense of option to extend the lease | true | ||||||||
Additional lease term under option to extend | 5 years | ||||||||
Tenant improvement allowance received | $ 800,000 | ||||||||
Lease period | 7 years 6 months | ||||||||
Lease expiration date | Sep. 30, 2023 | ||||||||
Monthly lease payments commencement period | 2016-09 | ||||||||
Required period to terminate lease after commencement period | 67 months |
Commitments and Contingencies_2
Commitments and Contingencies - Summary of Lease Costs and Supplemental Cash Flow Information of Operating Leases (Detail) $ in Thousands | 3 Months Ended |
Mar. 31, 2019USD ($) | |
Leases [Abstract] | |
Operating lease cost | $ 700 |
Variable lease cost | 161 |
Total lease cost | 861 |
Cash paid for amounts included in the measurement of operating lease liabilities | 711 |
Right-of-use assets acquired through operating lease liabilities | $ 36 |
Commitments and Contingencies_3
Commitments and Contingencies - Weighted-Average Remaining Lease Term and Weighted-Average Discount Rate of Operating Leases (Detail) | Mar. 31, 2019 |
Leases [Abstract] | |
Weighted-average remaining lease term (years) | 3 years 1 month 6 days |
Weighted-average discount rate | 5.60% |
Commitments and Contingencies_4
Commitments and Contingencies - Reconciliation of Undiscounted Future Minimum Lease Payments Remaining Under 9800 Medical Center Drive Lease and Other Operating Leases (Detail) $ in Thousands | Mar. 31, 2019USD ($) |
Undiscounted future minimum lease payments: | |
2019 (remainder of year) | $ 1,995 |
2020 | 3,088 |
2021 | 3,744 |
2022 | 4,931 |
2023 | 5,667 |
Thereafter | 76,892 |
Total undiscounted future minimum lease payments | 96,317 |
Current portion of operating lease liabilities | (2,397) |
Operating lease liabilities, non-current | 5,483 |
9800 Medical Center Drive Lease [Member] | |
Undiscounted future minimum lease payments: | |
2021 | 1,332 |
2022 | 4,308 |
2023 | 5,188 |
Thereafter | 76,892 |
Total undiscounted future minimum lease payments | 87,720 |
Other Operating leases [Member] | |
Undiscounted future minimum lease payments: | |
2019 (remainder of year) | 1,995 |
2020 | 3,088 |
2021 | 2,412 |
2022 | 623 |
2023 | 479 |
Total undiscounted future minimum lease payments | 8,597 |
Amount representing imputed interest | (717) |
Total operating lease liabilities | 7,880 |
Current portion of operating lease liabilities | (2,397) |
Operating lease liabilities, non-current | $ 5,483 |
Commitments and Contingencies_5
Commitments and Contingencies - 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 |
License Revenue - Additional In
License Revenue - Additional Information (Detail) | 1 Months Ended | 3 Months Ended | 12 Months Ended | ||||
May 31, 2018USD ($) | Jan. 31, 2018USD ($) | Mar. 31, 2014USD ($) | Mar. 31, 2019USD ($)ProductCandidateTechnologyLicensee | Mar. 31, 2018USD ($) | Dec. 31, 2018USD ($) | Dec. 31, 2017USD ($) | |
License Revenue [Line Items] | |||||||
Milestone payment upon commencement of clinical trials in humans | $ 29,600,000 | ||||||
Milestone payment upon submission of regulatory approval filings | 47,500,000 | ||||||
Milestone payment upon approval of commercial products by regulatory agencies | 117,500,000 | ||||||
Milestone payment upon achievement of specified sales targets for licensed products | 232,000,000 | ||||||
Deferred revenue, current and non-current | 3,933,000 | $ 3,933,000 | |||||
Impirment losses on receivables or contract assets | 0 | 0 | |||||
Total current and non-current accounts receivable | 31,130,000 | $ 58,612,000 | 31,599,000 | $ 5,850,000 | |||
Billed to customers | 100,000 | 400,000 | |||||
Billable to customers in future periods | $ 31,000,000 | 31,200,000 | |||||
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 March 31, 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, if any. | ||||||
Interest income from licensing | $ 613,000 | 1,355,000 | |||||
Revenue | 884,000 | 132,391,000 | |||||
Accounts receivable, current | $ 8,372,000 | 8,587,000 | |||||
NAV Technology Licensees [Member] | NAV Technology Platform [Member] | |||||||
License Revenue [Line Items] | |||||||
Number of technology licensees | TechnologyLicensee | 11 | ||||||
NAV Technology Licensees [Member] | NAV Technology Platform [Member] | Minimum [Member] | |||||||
License Revenue [Line Items] | |||||||
Number of development partnered product candidates | ProductCandidate | 20 | ||||||
January 2018 Amendment Agreement [Member] | AveXis, Inc. [Member] | |||||||
License Revenue [Line Items] | |||||||
Accounts receivable | 53,500,000 | ||||||
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 | ||||||
License fees received | $ 100,000,000 | ||||||
Transaction price of license | 132,100,000 | $ 172,100,000 | |||||
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 Agreement [Member] | License [Member] | |||||||
License Revenue [Line Items] | |||||||
License revenue | $ 800,000 | 300,000 | |||||
November 2018 License Agreement [Member] | Abeona Therapeutics Inc. [Member] | |||||||
License Revenue [Line Items] | |||||||
Accounts receivable | 26,700,000 | 26,000,000 | |||||
March 2014 License Agreement [Member] | AveXis, Inc. [Member] | |||||||
License Revenue [Line Items] | |||||||
Accounts receivable | 200,000 | 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 | 7,500,000 | ||||||
Payments for remaining development milestones | 7,000,000 | ||||||
Interest income from licensing | 1,200,000 | ||||||
Accounts receivable, non -current | 200,000 | 200,000 | |||||
March 2014 License Agreement [Member] | Maximum [Member] | AveXis, Inc. [Member] | |||||||
License Revenue [Line Items] | |||||||
Interest income from licensing | 100,000 | ||||||
Accounts receivable, current | 100,000 | $ 100,000 | |||||
March 2014 License Agreement [Member] | License [Member] | AveXis, Inc. [Member] | |||||||
License Revenue [Line Items] | |||||||
Revenue | $ 0 | $ 132,100,000 |
License Revenue - Summary of Ch
License Revenue - Summary of Changes in Balances of Receivables, Contract Assets and Contract Liabilities (Detail) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2019 | Mar. 31, 2018 | |
Receivables and contract assets: | ||
Accounts receivable, current and non-current, Balance at Beginning of Period | $ 31,599 | $ 5,850 |
Accounts receivable, current and non-current, Net Additions (Deductions) | (469) | 52,762 |
Accounts receivable, current and non-current, Balance at End of Period | 31,130 | 58,612 |
Contract assets, Balance at Beginning of Period | 750 | 350 |
Contract assets, Net Additions (Deductions) | 250 | (100) |
Contract assets, Balance at End of Period | 1,000 | $ 250 |
Contract liabilities: | ||
Deferred revenue, current and non-current, Balance at Beginning of Period | 3,933 | |
Deferred revenue, current and non-current, Balance at End of Period | $ 3,933 |
License Revenue - Additional _2
License Revenue - Additional Information (Detail1) | Mar. 31, 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 |
Stock-based Compensation - Addi
Stock-based Compensation - Additional Information (Detail) - USD ($) $ / shares in Units, $ in Thousands | 1 Months Ended | 3 Months Ended | |
Jan. 31, 2019 | Mar. 31, 2019 | Mar. 31, 2018 | |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||
Unrecognized stock-based compensation expense | $ 68,900 | ||
Unrecognized stock-based compensation, weighted-average period | 3 years 1 month 6 days | ||
Proceeds from exercise of stock options | $ 3,762 | $ 3,824 | |
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 | 2,583,144 | ||
Weighted-average fair values of options granted | $ 32.20 | ||
Exercise of stock options, Shares | 480,320 | ||
Total intrinsic value of options exercised | $ 20,000 | ||
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 | |
Mar. 31, 2019 | Mar. 31, 2018 | |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||
Stock-based compensation expense | $ 5,718 | $ 3,291 |
Stock Option [Member] | ||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||
Stock-based compensation expense | 5,452 | 3,122 |
Restricted Stock Units [Member] | ||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||
Stock-based compensation expense | 68 | 68 |
Employee Stock Purchase Plan [Member] | ||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||
Stock-based compensation expense | $ 198 | $ 101 |
Stock-based Compensation - St_2
Stock-based Compensation - Stock-Based Compensation Expense (Detail) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2019 | Mar. 31, 2018 | |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||
Stock-based compensation expense | $ 5,718 | $ 3,291 |
Research and Development Costs [Member] | ||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||
Stock-based compensation expense | 2,347 | 1,530 |
General and Administrative [Member] | ||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||
Stock-based compensation expense | $ 3,371 | $ 1,761 |
Stock-based Compensation - Summ
Stock-based Compensation - Summary of Stock Option Activity (Detail) - 2014 and 2015 Equity Incentive Plan [Member] - USD ($) $ / shares in Units, shares in Thousands, $ in Thousands | 3 Months Ended | 12 Months Ended |
Mar. 31, 2019 | Dec. 31, 2018 | |
Share-based Compensation Arrangement by Share-based Payment Award, Options, Nonvested, Number of Shares [Roll Forward] | ||
Shares Outstanding, Beginning Balance | 4,855 | |
Shares, Granted | 1,102 | |
Shares, Exercised | (480) | |
Shares, Cancelled or forfeited | (137) | |
Shares Outstanding, Ending Balance | 5,340 | 4,855 |
Shares, Exercisable | 2,491 | |
Shares, Vested and expected to vest | 5,340 | |
Share-based Compensation Arrangement by Share-based Payment Award, Options, Outstanding, Weighted Average Exercise Price [Abstract] | ||
Weighted-average Exercise Price Outstanding, Beginning Balance | $ 19.31 | |
Weighted-average Exercise Price, Granted | 48.22 | |
Weighted-average Exercise Price, Exercised | 7.81 | |
Weighted-average Exercise Price, Cancelled or forfeited | 30.72 | |
Weighted-average Exercise Price, Outstanding, Ending Balance | 26.02 | $ 19.31 |
Weighted-average Exercise Price, Exercisable | 12.22 | |
Weighted-average Exercise Price, Vested and expected to vest | $ 26.02 | |
Share-based Compensation Arrangement by Share-based Payment Award, Options, Additional Disclosures [Abstract] | ||
Weighted-average Remaining Contractual Life (Years) Outstanding | 7 years 10 months 24 days | 7 years 7 months 6 days |
Weighted-average Remaining Contractual Life (Years), Exercisable | 6 years 9 months 18 days | |
Weighted-average Remaining Contractual Life (Years), Vested and expected to vest | 7 years 10 months 24 days | |
Aggregate Intrinsic Value Outstanding | $ 170,518 | $ 118,185 |
Aggregate Intrinsic Value, Exercisable | 112,324 | |
Aggregate Intrinsic Value, Vested and expected to vest | $ 170,518 |
Stock-based Compensation - Su_2
Stock-based Compensation - Summary of Unvested RSUs Activity Under 2015 Plan (Detail) - Restricted Stock Units [Member] shares in Thousands | 3 Months Ended |
Mar. 31, 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 | ||
Mar. 31, 2019 | Mar. 31, 2018 | Dec. 31, 2018 | |
Income Tax Disclosure [Line Items] | |||
Income tax benefit | $ 70 | ||
Unrealized gains (losses) on available-for-sale securities, income tax expense | 400 | $ 0 | |
Accrued income tax provision | 1,134 | $ 726 | |
Accrued Expenses and Other Current Liabilities [Member] | |||
Income Tax Disclosure [Line Items] | |||
Accrued income tax provision | $ 300 |
Related Party Transactions - Ad
Related Party Transactions - Additional Information (Detail) - USD ($) $ in Millions | 3 Months Ended | |
Mar. 31, 2019 | Mar. 31, 2018 | |
FOXKISER,LLP [Member] | Service Agreements [Member] | Research and Development Costs [Member] | ||
Related Party Transaction [Line Items] | ||
Related party transaction expense | $ 0.8 | $ 0.5 |
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 | |
Mar. 31, 2019 | Mar. 31, 2018 | |
Basic net income (loss) per share: | ||
Net income (loss) applicable to common stockholders | $ (32,228) | $ 104,239 |
Shares used in computation: | ||
Weighted-average common shares outstanding | 36,366 | 31,632 |
Basic net income (loss) per share | $ (0.89) | $ 3.30 |
Diluted net income (loss) per share: | ||
Net income (loss) applicable to common stockholders | $ (32,228) | $ 104,239 |
Shares used in computation: | ||
Weighted-average common shares outstanding | 36,366 | 31,632 |
Weighted-average diluted common shares | 36,366 | 34,275 |
Diluted net income (loss) per share | $ (0.89) | $ 3.04 |
Stock Option [Member] | ||
Shares used in computation: | ||
Weighted-average diluted common shares | 2,620 | |
Restricted Stock Units [Member] | ||
Shares used in computation: | ||
Weighted-average diluted common shares | 22 | |
Employee Stock Purchase Plan [Member] | ||
Shares used in computation: | ||
Weighted-average diluted common shares | 1 |
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 | |
Mar. 31, 2019 | Mar. 31, 2018 | |
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | ||
Anti-dilutive securities excluded from computation of diluted weighted average shares outstanding | 5,400 | 1,582 |
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 | 5,340 | 1,582 |
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 | |
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 | 20 |
Supplemental Disclosures - Sche
Supplemental Disclosures - Schedules of Accrued Expenses and Other Current Liabilities (Detail) - USD ($) $ in Thousands | Mar. 31, 2019 | Dec. 31, 2018 |
Payables And Accruals [Abstract] | ||
Accrued external research and development expenses | $ 5,474 | $ 4,274 |
Accrued personnel costs | 5,357 | 9,484 |
Accrued income taxes payable | 1,134 | 726 |
Accrued licensing costs | 911 | 1,617 |
Accrued external general and administrative expenses | 854 | 773 |
Accrued purchases of property and equipment | 191 | 221 |
Other accrued expenses and current liabilities | 268 | 69 |
Accrued expenses and other current liabilities | $ 14,189 | $ 17,164 |
Supplemental Disclosures - Addi
Supplemental Disclosures - Additional Information (Detail) - USD ($) $ in Thousands | Mar. 31, 2019 | Dec. 31, 2018 |
Payables And Accruals [Abstract] | ||
Other liabilities | $ 1,772 | $ 2,505 |