Document and Entity Information
Document and Entity Information - USD ($) | 12 Months Ended | ||
Dec. 31, 2019 | Feb. 21, 2020 | Jun. 28, 2019 | |
Cover [Abstract] | |||
Document Type | 10-K | ||
Amendment Flag | false | ||
Document Period End Date | Dec. 31, 2019 | ||
Document Fiscal Year Focus | 2019 | ||
Document Fiscal Period Focus | FY | ||
Trading Symbol | SRPT | ||
Entity Registrant Name | Sarepta Therapeutics, Inc. | ||
Entity Central Index Key | 0000873303 | ||
Current Fiscal Year End Date | --12-31 | ||
Entity Well-known Seasoned Issuer | Yes | ||
Entity Current Reporting Status | Yes | ||
Entity Voluntary Filers | No | ||
Entity Filer Category | Large Accelerated Filer | ||
Entity Small Business | false | ||
Entity Emerging Growth Company | false | ||
Entity Shell Company | false | ||
Entity File Number | 001-14895 | ||
Entity Tax Identification Number | 93-0797222 | ||
Entity Incorporation, State or Country Code | DE | ||
Entity Address, Address Line One | 215 First Street | ||
Entity Address, Address Line Two | Suite 415 | ||
Entity Address, City or Town | Cambridge | ||
Entity Address, State or Province | MA | ||
Entity Address, Postal Zip Code | 02142 | ||
City Area Code | 617 | ||
Local Phone Number | 274-4000 | ||
Entity Interactive Data Current | Yes | ||
Title of 12(b) Security | Common Stock, $0.0001 par value | ||
Security Exchange Name | NASDAQ | ||
Document Annual Report | true | ||
Document Transition Report | false | ||
Entity Common Stock, Shares Outstanding | 77,776,779 | ||
Entity Public Float | $ 11,294,104,196 | ||
Documents Incorporated by Reference | The registrant has incorporated by reference into Part II and Part III of this Annual Report on Form 10-K portions of its definitive Proxy Statement for the 2020 Annual Meeting of Stockholders to be filed no later than 120 days after the end of the fiscal year covered by this Annual Report on Form 10-K. |
Consolidated Balance Sheets
Consolidated Balance Sheets - USD ($) $ in Thousands | Dec. 31, 2019 | Dec. 31, 2018 |
Current assets: | ||
Cash and cash equivalents | $ 835,080 | $ 370,829 |
Short-term investments | 289,668 | 803,083 |
Accounts receivable | 90,879 | 49,044 |
Inventory | 171,379 | 125,445 |
Other current assets | 81,907 | 77,782 |
Total current assets | 1,468,913 | 1,426,183 |
Property and equipment, net | 129,620 | 97,024 |
Intangible assets, net | 12,497 | 11,574 |
Right of use assets, net | 37,933 | |
Other non-current assets | 173,859 | 107,294 |
Total assets | 1,822,822 | 1,642,075 |
Current liabilities: | ||
Accounts payable | 68,094 | 33,829 |
Accrued expenses | 185,527 | 134,095 |
Other current liabilities | 11,146 | 5,766 |
Total current liabilities | 264,767 | 173,690 |
Long-term debt | 681,900 | 420,554 |
Lease liabilities | 47,720 | |
Other non-current liabilities | 10,248 | 15,555 |
Total liabilities | 1,004,635 | 609,799 |
Commitments and contingencies (Note 21) | ||
Stockholders’ equity: | ||
Preferred stock, $.0001 par value, 3,333,333 shares authorized; none issued and outstanding | ||
Common stock, $.0001 par value, 99,000,000 shares authorized; 75,184,863 and 71,071,887 issued and outstanding at December 31, 2019 and 2018, respectively | 8 | 7 |
Additional paid-in capital | 3,112,130 | 2,611,294 |
Accumulated other comprehensive income (loss), net of tax | 50 | (99) |
Accumulated deficit | (2,294,001) | (1,578,926) |
Total stockholders’ equity | 818,187 | 1,032,276 |
Total liabilities and stockholders’ equity | $ 1,822,822 | $ 1,642,075 |
Consolidated Balance Sheets (Pa
Consolidated Balance Sheets (Parenthetical) - $ / shares | Dec. 31, 2019 | Dec. 31, 2018 |
Statement Of Financial Position [Abstract] | ||
Preferred stock, par value | $ 0.0001 | $ 0.0001 |
Preferred stock, shares authorized | 3,333,333 | 3,333,333 |
Preferred stock, shares issued | 0 | 0 |
Preferred stock, shares outstanding | 0 | 0 |
Common stock, par value | $ 0.0001 | $ 0.0001 |
Common stock, shares authorized | 99,000,000 | 99,000,000 |
Common stock, issued | 75,184,863 | 71,071,887 |
Common stock, outstanding | 75,184,863 | 71,071,887 |
Consolidated Statements of Oper
Consolidated Statements of Operations and Comprehensive Loss - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Revenues: | |||
Product, net | $ 380,833 | $ 301,034 | $ 154,584 |
Type of Revenue [Extensible List] | us-gaap:ProductMember | us-gaap:ProductMember | us-gaap:ProductMember |
Total revenues | $ 380,833 | $ 301,034 | $ 154,584 |
Cost and expenses: | |||
Cost of sales (excluding amortization of in-licensed rights) | 56,586 | 34,193 | 7,353 |
Research and development | 560,909 | 401,843 | 166,707 |
Selling, general and administrative | 284,812 | 207,761 | 122,682 |
Acquired in-process research and development | 173,240 | ||
Settlement and license charges | 10,000 | 28,427 | |
Amortization of in-licensed rights | 849 | 865 | 1,053 |
Total cost and expenses | 1,086,396 | 644,662 | 326,222 |
Operating loss | (705,563) | (343,628) | (171,638) |
Other (loss) income: | |||
Other expense, net | (8,317) | (18,982) | (1,990) |
Gain from sale of Priority Review Voucher | 125,000 | ||
Total other (loss) income | (8,317) | (18,982) | 123,010 |
Loss before income tax expense (benefit) | (713,880) | (362,610) | (48,628) |
Income tax expense (benefit) | 1,195 | (692) | 2,060 |
Net loss | (715,075) | (361,918) | (50,688) |
Other comprehensive loss: | |||
Unrealized gains (losses) on investments | 149 | 280 | (259) |
Total other comprehensive income (loss) | 149 | 280 | (259) |
Comprehensive loss | $ (714,926) | $ (361,638) | $ (50,947) |
Net loss per share — basic and diluted | $ (9.71) | $ (5.46) | $ (0.86) |
Weighted average number of shares of common stock used in computing basic and diluted net loss per share | 73,615,000 | 66,250,000 | 58,818,000 |
Consolidated Statements of Stoc
Consolidated Statements of Stockholders' Equity - USD ($) $ in Thousands | Total | Common Stock [Member] | Additional Paid-In Capital [Member] | Accumulated Other Comprehensive (Loss) Gain [Member] | Accumulated Deficit [Member] |
BALANCE at Dec. 31, 2016 | $ 336,691 | $ 5 | $ 1,503,126 | $ (120) | $ (1,166,320) |
BALANCE (in shares) at Dec. 31, 2016 | 54,759,000 | ||||
Exercise of options for common stock | $ 13,799 | 13,799 | |||
Exercise of options for common stock, shares | 792,845 | 793,000 | |||
Grant of restricted stock awards and vest of restricted stock units, net of cancellations, shares | 400,000 | ||||
Shares withheld for taxes | $ (2,227) | (2,227) | |||
Shares withheld for taxes, shares | (60,000) | ||||
Issuance of common stock for cash, net of offering costs | 353,959 | $ 1 | 353,958 | ||
Issuance of common stock for cash, net of offering costs, shares | 8,798,000 | ||||
Issuance of common stock under employee stock purchase plan | 1,425 | 1,425 | |||
Issuance of common stock under employee stock purchase plan, shares | 102,000 | ||||
Equity component of convertible notes | 156,953 | 156,953 | |||
Purchase of capped call share options | (50,901) | (50,901) | |||
Stock-based compensation | 30,465 | 30,465 | |||
Unrealized gain (loss) from available-for-sale securities | (259) | (259) | |||
Net loss | (50,688) | (50,688) | |||
BALANCE at Dec. 31, 2017 | 789,217 | $ 6 | 2,006,598 | (379) | (1,217,008) |
BALANCE (in shares) at Dec. 31, 2017 | 64,792,000 | ||||
Exercise of options for common stock | $ 47,916 | 47,916 | |||
Exercise of options for common stock, shares | 2,119,306 | 2,119,000 | |||
Grant of restricted stock awards and vest of restricted stock units, net of cancellations, shares | 58,000 | ||||
Shares withheld for taxes | $ (9,061) | (9,061) | |||
Shares withheld for taxes, shares | (79,000) | ||||
Issuance of common stock for cash, net of offering costs | 513,409 | $ 1 | 513,408 | ||
Issuance of common stock for cash, net of offering costs, shares | 4,107,000 | ||||
Issuance of common stock under employee stock purchase plan | 2,306 | 2,306 | |||
Issuance of common stock under employee stock purchase plan, shares | 75,000 | ||||
Stock-based compensation | 50,127 | 50,127 | |||
Unrealized gain (loss) from available-for-sale securities | 280 | 280 | |||
Net loss | (361,918) | (361,918) | |||
BALANCE at Dec. 31, 2018 | $ 1,032,276 | $ 7 | 2,611,294 | (99) | (1,578,926) |
BALANCE (in shares) at Dec. 31, 2018 | 71,072,000 | ||||
Exercise of options for common stock, shares | 1,055,715 | ||||
Exercise of options and stock appreciation rights for common stock | $ 31,522 | 31,522 | |||
Exercise of options and stock appreciation rights for common stock, shares | 1,125,000 | ||||
Grant of restricted stock awards and vest of restricted stock units, net of cancellations, shares | 68,000 | ||||
Shares withheld for taxes | (9,135) | (9,135) | |||
Shares withheld for taxes, shares | (78,000) | ||||
Issuance of common stock for cash, net of offering costs | 365,354 | $ 1 | 365,353 | ||
Issuance of common stock for cash, net of offering costs, shares | 2,604,000 | ||||
Issuance of common stock for collaboration agreement | 29,415 | 29,415 | |||
Issuance of common stock for collaboration agreement, shares | 302,000 | ||||
Issuance of common stock under employee stock purchase plan | 5,079 | 5,079 | |||
Issuance of common stock under employee stock purchase plan, shares | 92,000 | ||||
Stock-based compensation | 78,602 | 78,602 | |||
Unrealized gain (loss) from available-for-sale securities | 149 | 149 | |||
Net loss | (715,075) | (715,075) | |||
BALANCE at Dec. 31, 2019 | $ 818,187 | $ 8 | $ 3,112,130 | $ 50 | $ (2,294,001) |
BALANCE (in shares) at Dec. 31, 2019 | 75,185,000 |
Consolidated Statements of Cash
Consolidated Statements of Cash Flows - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Cash flows from operating activities: | |||
Net loss | $ (715,075) | $ (361,918) | $ (50,688) |
Adjustments to reconcile net loss to cash flows in operating activities: | |||
Acquired in-process research and development | 173,240 | ||
Non-cash up-front payment to StrideBio | 29,415 | ||
Gain from sale of Priority Review Voucher | (125,000) | ||
Depreciation and amortization | 30,547 | 12,245 | 8,092 |
Amortization of investment discount | (8,445) | (7,672) | (888) |
Loss from debt extinguishment | 2,322 | ||
Non-cash interest expense | 21,444 | 20,190 | 2,679 |
Stock-based compensation | 78,602 | 50,127 | 30,465 |
Other | 690 | 3,938 | 805 |
Changes in operating assets and liabilities, net: | |||
Net increase in accounts receivable | (41,835) | (19,576) | (24,240) |
Net increase in inventory | (45,934) | (41,840) | (70,792) |
Net increase in other assets | (102,091) | (136,638) | (15,354) |
Net increase in accounts payable, accrued expenses, lease liabilities and other liabilities | 122,979 | 90,162 | 12,925 |
Net cash used in operations | (456,463) | (388,660) | (231,996) |
Cash flows from investing activities: | |||
Purchase of property and equipment | (59,631) | (61,157) | (12,000) |
Purchase of intangible assets | (3,082) | (3,188) | (9,215) |
Purchase of available-for-sale securities | (1,193,632) | (1,171,603) | (589,520) |
Maturity and sales of available-for-sale securities | 1,715,626 | 865,813 | 296,225 |
Proceeds from sale of Priority Review Voucher | 125,000 | ||
Purchase of restricted investment | (353) | ||
Maturity of restricted investment | 10,695 | ||
Acquisition of Myonexus Therapeutics, Inc., net of cash acquired | (172,556) | ||
Net cash provided (used) in investing activities | 286,725 | (370,488) | (178,815) |
Cash flows from financing activities: | |||
Proceeds from exercise of options and employee stock purchase program | 36,601 | 50,222 | 15,224 |
Taxes paid related to net share settlement of equity awards | (4,337) | ||
Proceeds from sales of common stock, net of offering costs | 365,354 | 513,409 | 353,959 |
Repayment of June 2015 and July 2017 Term Loan | (30,000) | (15,000) | |
Proceeds from revolving line of credit | 235,872 | 39,708 | |
Repayment of revolving line of credit | (235,954) | (39,645) | |
Payment for debt extinguishment | (2,134) | ||
Repayment of mortgage loans and notes payable | (1,265) | (109) | |
Purchase of capped call options | (50,901) | ||
Proceeds from convertible debt offering | 570,000 | ||
Debt issuance costs | (689) | (15,154) | |
Net cash provided by financing activities | 642,554 | 530,150 | 888,082 |
Increase (decrease) in cash and cash equivalents | 472,816 | (228,998) | 477,271 |
Cash, cash equivalents and restricted cash: | |||
Beginning of period | 370,829 | 599,827 | 122,556 |
End of period | 843,645 | 370,829 | 599,827 |
Reconciliation of cash, cash equivalents and restricted cash: | |||
Cash and cash equivalents | 835,080 | $ 370,829 | 599,691 |
Restricted cash in other assets | 8,565 | $ 136 | |
Restricted Cash, Noncurrent, Asset, Statement of Financial Position [Extensible List] | us-gaap:OtherAssetsNoncurrent | us-gaap:OtherAssetsNoncurrent | |
Total cash, cash equivalents and restricted cash | 843,645 | $ 370,829 | $ 599,827 |
Supplemental disclosure of cash flow information: | |||
Cash paid during the period for interest | 8,550 | 11,308 | 1,912 |
Cash paid during the period for income taxes | 933 | 1,548 | 5,336 |
Supplemental schedule of non-cash investing activities and financing activities: | |||
Shares withheld for tax included in accrued expenses | 4,798 | ||
Accrued debt discount and issuance costs | 5,000 | 625 | |
Property and equipment included in accrued expenses | 1,181 | 5,421 | 2,525 |
Reclassification of long term investments to short term investments | $ 9,980 | ||
December 2019 Term Loan [Member] | |||
Cash flows from financing activities: | |||
Proceeds from Term Loan | $ 245,625 | ||
July 2017 Term Loan [Member] | |||
Cash flows from financing activities: | |||
Proceeds from Term Loan | $ 30,000 |
ORGANIZATION AND NATURE OF BUSI
ORGANIZATION AND NATURE OF BUSINESS | 12 Months Ended |
Dec. 31, 2019 | |
Accounting Policies [Abstract] | |
ORGANIZATION AND NATURE OF BUSINESS | 1. ORGANIZATION AND NATURE OF BUSINESS Sarepta Therapeutics, Inc. (together with its wholly-owned subsidiaries, “Sarepta” or the “Company”) is a commercial-stage biopharmaceutical company focused on helping patients through the discovery and development of unique RNA-targeted therapeutics, gene therapy and other genetic therapeutic modalities for the treatment of rare diseases. Applying its proprietary, highly-differentiated and innovative technologies, and through collaborations with its strategic partners, the Company is developing potential therapeutic candidates for a broad range of diseases and disorders, including Duchenne muscular dystrophy (“DMD”), Limb-girdle muscular dystrophies (“LGMDs”), Mucopolysaccharidosis type IIIA (“MPS IIIA”) and other neuromuscular and central nervous system (“CNS”) disorders. Its first and second commercial products in the U.S., EXONDYS 51 (eteplirsen) Injection (“EXONDYS 51”) and VYONDYS 53 (golodirsen) Injection (“VYONDYS 53”), respectively, were granted accelerated approval by the U.S. Food and Drug Administration (“FDA”) on September 19, 2016 and December 12, 2019, respectively. EXONDYS 51 and VYONDYS 53 are indicated for the treatment of DMD in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 and exon 53 skipping, respectively. EXONDYS 51 and VYONDYS 53 As of December 31, 2019, the Company had approximately $1,134.4 million of cash, cash equivalents and investments, consisting of $835.1 million of cash and cash equivalents, $289.7 million of short-term investments, and $9.6 million of restricted cash and investments. The Company believes that its balance of cash, cash equivalents and investments as of December 31, 2019 is sufficient to fund its current operational plan for at least the next twelve months, though it may pursue additional cash resources through public or private debt and equity financings, seek additional government contracts and establish collaborations with or license its technology to other companies. |
SUMMARY OF SIGNIFICANT ACCOUNTI
SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES AND RECENT ACCOUNTING PRONOUNCEMENTS | 12 Months Ended |
Dec. 31, 2019 | |
Accounting Policies [Abstract] | |
SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES AND RECENT ACCOUNTING PRONOUNCEMENTS | 2. SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES AND RECENT ACCOUNTING PRONOUNCEMENTS Basis of Presentation The accompanying consolidated financial statements, which have been prepared in accordance with accounting principles generally accepted in the U.S. (“U.S. GAAP”), reflect the accounts of Sarepta Therapeutics, Inc. and its wholly-owned subsidiaries. All intercompany transactions between and among its consolidated subsidiaries have been eliminated. Management has determined that the Company operates in one segment: helping patients through the discovery and development of unique RNA-targeted therapeutics, gene therapy and other genetic therapeutic modalities for the treatment of rare diseases Estimates and Uncertainties The preparation of the consolidated financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets, liabilities, equity, revenue, expenses and the disclosure of contingent assets and liabilities. Actual results could differ from those estimates. Fair Value Measurements The Company has certain financial assets that are recorded at fair value which have been classified as Level 1, 2 or 3 within the fair value hierarchy as described in the accounting standards for fair value measurements: • Level 1—quoted prices for identical instruments in active markets; • Level 2—quoted prices for similar instruments in active markets, quoted prices for identical or similar instruments in markets that are not active, and model-derived valuations in which all significant inputs and significant value drivers are observable in active markets; and • Level 3—valuations derived from valuation techniques in which one or more significant value drivers are unobservable. The fair value of the majority of the Company’s financial assets is categorized as Level 1 within the fair value hierarchy. These assets include money market funds, publicly traded debt, and equity securities. For additional information related to fair value measurements, please read Note 5, Fair Value Measurements Cash Equivalents Only investments that are highly liquid and readily convertible to cash and have original maturities of three months or less are considered cash equivalents. Investments Available-For-Sale Debt Securities Available-for-sale debt securities are recorded at fair value and unrealized gains and losses are included in accumulated other comprehensive income (loss) in stockholder’s equity. Realized gains and losses are reported in other expense, net, on a specific identification basis. Equity Investments The Company’s equity investments include its investments in a publicly traded biotechnology company and a privately held biotechnology company and are included in other non-current assets in the Company’s consolidated balance sheets. The equity investment in the publicly traded biotechnology company has a readily determinable fair value and is carried at fair value with changes in value recorded as a gain or loss in the Company’s consolidated statements of operations and comprehensive loss. The equity investment in the privately held biotechnology company does not have readily determinable fair value and is measured at cost less any impairment, plus or minus changes resulting from observable price changes for the identical or a similar investment of the same issuer, which is recorded as a gain or loss on the Company’s consolidated statements of operations and comprehensive loss. Accounts Receivable The Company’s accounts receivable primarily arise from product sales. They are generally stated at the invoiced amount and do not bear interest. Revenues from product sales are recorded at the net sales price (transaction price), which includes estimates of variable consideration for which reserves are established and which result from Medicaid rebates, governmental chargebacks including Public Health Services (“PHS”) chargebacks, prompt pay discounts, co-pay assistance and distribution fees. These reserves are based on the amounts earned or to be claimed on the related sales and are classified as reductions of accounts receivable (if no payments are required of the Company) for PHS chargebacks, prompt pay discounts and certain distribution fees, or a current liability (if a payment is required of us), for Medicaid rebates, co-pay assistance and certain distribution fees. The accounts receivable from product sales represents receivables due from the Company’s specialty distributor and specialty pharmacies in the U.S. as well as certain distributors in the EU, Brazil, Israel and the Middle East. The Company monitors the financial performance and creditworthiness of its customers so that it can properly assess and respond to changes in the customers’ credit profiles. The Company provides reserves against trade receivables for estimated losses that may result from a customer’s inability to pay. Amounts determined to be uncollectible are written-off against the established reserve. As of December 31, 2019, the credit profiles for the Company’s customers are deemed to be in good standing and an allowance for doubtful accounts receivable is not considered necessary. Concentration of Credit Risk Financial instruments which potentially subject the Company to concentrations of credit risk consist of accounts receivable from customers and cash, cash equivalent and investments held at financial institutions. For the year ended December 31, 2019 42 47 51 December 31, 2019 As of December 31, 2019 Inventories Inventories are stated at the lower of cost and net realizable value with cost determined on a first-in, first-out basis. The Company capitalizes inventory costs associated with products following regulatory approval when future commercialization is considered probable and the future economic benefit is expected to be realized. EXONDYS 51 and VYONDYS 53 inventory that may be used in clinical development programs is charged to research and development expense when the product enters the research and development process and no longer can be used for commercial purposes. The Company periodically reviews its inventories for excess amounts or obsolescence and writes down obsolete or otherwise unmarketable inventory to its estimated net realizable value. Additionally, though the Company’s product is subject to strict quality control and monitoring which it performs throughout the manufacturing processes, certain batches or units of product may not meet quality specifications resulting in a charge to cost of sales. For products which are under development and have not yet been approved by regulatory authorities, purchased drug product is charged to research and development expense upon delivery. Delivery occurs when the inventory passes quality inspection and ownership transfers to the Company. Nonrefundable advance payments for research and development activities, including production of purchased drug product, are deferred and capitalized until the goods are delivered. If the Company does not expect the goods to be delivered or services to be rendered, the advanced payment capitalized will be charged to expense. Property and Equipment Property and equipment are initially recorded at cost, including the acquisition cost and all costs necessarily incurred to bring the asset to the location and working condition necessary for its intended use. The cost of normal, recurring or periodic repairs and maintenance activities related to property and equipment are expensed as incurred. The cost for planned major maintenance activities, including the related acquisition or construction of assets, is capitalized if the repair will result in future economic benefits. Interest costs incurred during the construction period of major capital projects are capitalized until the asset is ready for its intended use, at which point the interest costs are amortized as depreciation expense over the life of the underlying asset. The Company generally depreciates the cost of its property and equipment using the straight-line method over the estimated useful lives of the respective assets, which are summarized as follows: Asset Category Useful lives Lab equipment 5 years Office equipment 5 years Software and computer equipment 3 - 5 years Furniture and fixtures 7 years Leasehold improvements Lesser of the useful life or the term of the respective lease Land improvements 25 years Land Not depreciated Building and improvements 30 years Construction in Progress Not depreciated until put into service Intangible assets The Company’s intangible assets consist of in-licensed rights, patent costs, and software licenses, which are stated in the Company’s consolidated balance sheets net of accumulated amortization and impairments, if applicable. The in-licensed rights relate to agreements with BioMarin Pharmaceutical, Inc. (“BioMarin”) and the University of Western Australia (“UWA”). The in-licensed rights are being amortized on a straight-line basis over the remaining life of the related patents because the life of the related patents reflects the expected time period that the Company will benefit from the in-licensed rights. Patent costs consist primarily of external legal costs, filing fees incurred to file patent applications and renewal fees on proprietary technology developed or licensed by the Company. Patent costs associated with applying for a patent, being issued a patent and annual renewal fees are capitalized. Costs to defend a patent and costs to invalidate a competitor’s patent or patent application are expensed as incurred. Patent costs are amortized on a straight-line basis over the shorter of the estimated economic lives or the initial term of the patents, which is generally 20 years. Impairment of Long-Lived Assets Long-lived assets held and used by the Company, intangible assets with definite lives and equity investments without a readily determinable fair value are reviewed for impairment whenever events or circumstances indicate that the carrying amount of assets may not be recoverable. The Company evaluates recoverability of assets to be held and used by comparing the carrying amount of an asset to future net undiscounted cash flows to be generated by the asset. If the asset is considered to be impaired, the impairment to be recognized is measured as the amount by which the carrying amount of the assets exceeds the fair value of the assets. Such reviews assess the fair value of the assets based upon estimates of future cash flows that the assets are expected to generate. Convertible Debt The Company separately accounts for the liability and equity components of convertible debt instruments that can be settled in cash by allocating the proceeds from issuance between the liability component and the embedded conversion option. The value of the equity component is calculated by first measuring the fair value of the liability component, using the interest rate of a similar liability that does not have a conversion feature, as of the issuance date. The difference between the proceeds from the convertible debt issuance and the amount measured as the liability component is recorded as the equity component with a corresponding discount recorded on the debt. The Company recognizes the amortization of the resulting discount as interest expense using the effective interest method. Simultaneously, the Company bought capped call options from certain counterparties to minimize the impact of potential dilution upon conversion. The premium for the capped call options was recorded as additional paid-in capital. For additional information related to the convertible debt transactions, please read Note 13, Indebtedness Revenue Recognition The Company recognizes revenue when a customer obtains control of promised goods or services, in an amount that reflects the consideration which the Company expects to receive in exchange for the goods or services provided. To determine revenue recognition for arrangements within the scope of ASC Topic 606, “Revenue from Contracts with Customers” Product revenues The Company distributes its products principally through its customers. The customers subsequently resell the product to patients and health care providers. The Company provides no right of return to the customers except in cases of shipping error or product defect. Product revenues are recognized when the customers take control of the product, which typically occurs upon delivery to the customers. For the years ended December 31, 2019, 2018 2017 Variable Consideration Product revenues are recorded at the net sales price (transaction price) which includes estimated reserves for variable consideration, such as Medicaid rebates, governmental chargebacks, including Public Health Service (“PHS”) chargebacks, prompt payment discounts, co-pay assistance and distribution fees. These reserves reflect the Company’s best estimates of the amount of consideration to which it is entitled based on the terms of the contracts. Additional details relating to variable consideration follows: • Medicaid rebates relate to the Company’s estimated obligations to states under established reimbursement arrangements. Medicaid rebate reserves are recorded in the same period the related revenue is recognized, resulting in a reduction of product revenue and the establishment of a liability which is included in accrued expenses. • Governmental chargebacks, including PHS chargebacks, relate to the Company’s estimated obligations resulting from contractual commitments to sell products to qualified healthcare providers at prices lower than the list prices that the Company charges to wholesalers. The wholesaler charges the Company for the difference between what the wholesaler pays for the products and the ultimate selling price to the qualified healthcare providers. Chargeback reserves are recorded in the same period the related revenue is recognized, resulting in a reduction of product revenue and accounts receivable. Chargeback amounts are generally determined at the time of resale to the qualified healthcare provider from the wholesaler, and the Company generally issues credits for such amounts within a few weeks of receiving notification of resale from the wholesaler. • Prompt payment discounts relate to the Company’s estimated obligations for credits to be granted to specialty pharmacies for remitting payment on their purchases within established incentive periods. Reserves for prompt payment discounts are recorded in the same period the related revenue is recognized, resulting in a reduction of product revenue and accounts receivable. • Co-pay assistance relates to financial assistance provided to qualified patients, whereby the Company may assist them with prescription drug co-payments required by the patient’s insurance provider. Reserves for co-pay assistance are recorded in the same period the related revenue is recognized, resulting in a reduction of product revenue and the establishment of a liability which is included in accrued expenses. • Distribution fees relate to fees paid to customers in the distribution channel that provide the Company with inventory management, data and distribution services and are generally accounted for as a reduction of revenue. To the extent that the services received are distinct from the Company’s sale of products to the customers, these payments are accounted for as selling, general and administrative expenses. Reserves for distribution fees result in an increase in a liability if payments are required of the Company or a reduction of accounts receivable if no payments are required of the Company. Research and Development Research and development expenses consist of costs associated with research activities as well as those with the Company’s product development efforts, conducting pre-clinical trials, clinical trials and manufacturing activities. Research and development expenses are expensed as incurred. Up-front fees and milestones paid to third parties in connection with technologies which have not reached technological feasibility and do not have an alternative future use are expensed when incurred. Direct research and development expenses associated with the Company’s programs include clinical trial site costs, clinical manufacturing costs, costs incurred for consultants and other external services, such as data management and statistical analysis support and materials and supplies used in support of clinical programs. Indirect costs of the Company’s clinical programs include salaries, stock-based compensation and an allocation of its facility and technology costs. When third-party service providers’ billing terms do not coincide with the Company’s period-end, the Company is required to make estimates of its obligations to those third parties, including clinical trial and pharmaceutical development costs, contractual services costs and costs for supply of its drug candidates, incurred in a given accounting period and record accruals at the end of the period. The Company bases its estimates on its knowledge of the research and development programs, services performed for the period, past history for related activities and the expected duration of the third-party service contract, where applicable. Stock-Based Compensation The Company’s stock-based compensation programs include stock options, restricted stock awards (“RSAs”), restricted stock units (“RSUs”), stock appreciation rights (“SARs”) and an employee stock purchase program (“ESPP”). The Company accounts for stock-based compensation using the fair value method. The fair values of stock options and SARs are estimated on the date of grant using the Black-Scholes-Merton option-pricing model. The fair values of RSAs and RSUs are based on the fair market value of the Company’s common stock on the date of the grant. The fair value of stock awards, with consideration given to estimated forfeitures, is recognized as stock-based compensation expense on a straight-line basis over the vesting period of the grants. For stock awards with performance-vesting conditions, the Company does not recognize compensation expense until it is probable that the performance-vesting condition will be achieved. Under the Company’s ESPP, participating employees purchase common stock through payroll deductions. The purchase price is equal to 85% of the lower of the closing price of the Company’s common stock on the first business day and the last business day of the relevant purchase period. The fair value of stock purchase rights are estimated using the Black-Scholes-Merton option-pricing model. The fair value of the look-back provision with the 15% discount is recognized on a graded-vesting basis as stock-based compensation expense over the purchase period. In addition to stock options with service and performance conditions, the Company also granted its CEO options with service and market conditions. A market condition relates to the achievement of a specified price of the Company’s common stock, a specified amount of intrinsic value indexed to the Company’s common stock or a specified price of the Company’s common stock in terms of other similar equity shares. The grant date fair value for the options with service and market conditions is determined by a lattice model with Monte Carlo simulations and is recognized as stock-based compensation expense on a straight-line basis over the service period. Income Taxes The Company follows the asset and liability method of accounting for income taxes. Deferred tax assets and liabilities are recognized for the future tax consequences attributable to differences between the consolidated financial statement carrying amounts of existing assets and liabilities and their respective tax bases and operating loss and tax credit carryforwards. It is the intention of the Company to reinvest the earnings of its non-U.S. subsidiaries in those operations and not to repatriate the earnings to the U.S. Accordingly, the Company does not provide for deferred taxes on the excess of the financial reporting over the tax basis in its investments in foreign subsidiaries as they are considered permanent in duration. Deferred tax assets and liabilities are measured using enacted tax rates expected to apply to taxable income in the years in which those temporary differences are expected to be recovered and settled. A valuation allowance is recorded to reduce the net deferred tax asset to zero when it is more likely than not that the net deferred tax asset will not be realized. The Company recognizes the effect of income tax positions only if those positions are more likely than not of being sustained upon an examination. Leases Effective January 1, 2019, the Company adopted ASC Topic 842, “ Leases” Leases” As a result of adopting ASC 842, the Company recorded lease right-of-use (“ROU”) assets of $42.5 million and lease liabilities of $60.1 million as of January 1, 2019, primarily related to real estate leases, based on the present value of future lease payments on the date of adoption. The difference between the ROU assets and lease liabilities was due to previously recorded net deferred rent liabilities that were reclassified into the ROU assets. There was no impact to retained earnings upon adoption of ASC 842. Amounts related to finance leases were immaterial as of adoption . At the inception of an arrangement, the Company determines whether the arrangement is or contains a lease based on the unique facts and circumstances present in the arrangement. Leases with a term greater than 12 months are recognized on the balance sheet as ROU assets and short-term and long-term lease liabilities, as applicable. The Company has elected not to recognize on the balance sheet leases with terms of 12 months or less. The Company typically only includes an initial lease term in its assessment of a lease arrangement. Options to renew a lease are not included in the Company’s assessment unless there is reasonable certainty that the Company will renew. The Company monitors its plans to renew its leases no less than on a quarterly basis. In addition, the Company’s lease agreements generally do not contain any residual value guarantees or restrictive covenants. Operating lease liabilities and their corresponding ROU assets are recorded based on the present value of future lease payments over the expected remaining lease term at lease commencement. Lease cost for operating leases is recognized on a straight-line basis over the lease term as an operating expense. Certain adjustments to the ROU asset may be required for items such as lease prepayments or incentives received. The interest rate implicit in lease contracts is typically not readily determinable. As a result, the Company utilizes its incremental borrowing rate, which reflects the fixed rate at which the Company could borrow on a collateralized basis the amount of the lease payments in the same currency, for a similar term, in a similar economic environment. In transition to ASC 842, the Company utilized the remaining lease term of its leases in determining the appropriate incremental borrowing rate. In accordance with ASC 842, components of a lease should be bifurcated between lease components and non - lease components. The fixed and in-substance fixed contract consideration identified must then be allocated based on the respective relative fair values to the lease components and non - lease components. However, ASC 842 provides a practical expedient that allows an accounting policy election to not separate lease and non - lease components by class of underlying asset. In using this expedient, the lease component and non - lease component s are accounted for together as a single component. For real estate leases, the Company has elected to account for the lease and non - lease components together for existing classes of underlying assets and allocates the contract consideration to the lease component only . This practical expedient is not elected for manufacturing facilities and equipment embedded in product supply arrangements. Embedded Derivatives The Company evaluates certain of its financial and business development transactions to determine if embedded components of these contracts meet the definition of derivative under Topic ASC 815, “ Derivatives and Hedging Commitments and Contingencies The Company records liabilities for legal and other contingencies when information available to the Company indicates that it is probable that a liability has been incurred and the amount of loss can be reasonably estimated. Legal costs in connection with legal and other contingencies are expensed as costs are incurred. Recent Accounting Pronouncements In December 2019, the Financial Accounting Standards Board (“FASB”) issued ASU 2019-12, “ Income Taxes (Topic 740): Simplifying the Accounting for Income Taxes”, In November 2018, the FASB issued ASU 2018-18, “ Collaborative Arrangements (Topic 808): Clarifying the Interaction between Topic 808 and Topic 606 In August 2018, the FASB issued ASU No. 2018-13, “Fair Value Measurement (Topic 820), Disclosure Framework – Changes to the Disclosure Requirements for Fair Value Measurement”. In August 2018, the FASB issued ASU No. 2018-15, “ Intangibles – Goodwill and Other – Internal-Use Software (Subtopic 350-40): Customer’s Accounting for Implementation Costs Incurred in a Cloud Computing Arrangement That is a Service Contract”. In June 2016 , the FASB issued ASU No. 2016-13, “ Financial Instruments - Credit Losses (Topic 326): Measurement of Credit Losses on Financial Instruments ” . This ASU requires that credit losses be reported using an expected losses model rather than the incurred losses model that is currently used, and establishes additional disclosures related to credit risks. For available-for-sale debt securities with unrealized losses, this standard now requires allowances to be recorded instead of reducing the amortized cost of the investment. ASU 2016-13 limits the amount of credit losses to be recognized for available-for-sale debt securities to the amount by which carrying value exceeds fair value and requires the reversal of previously recognized credit losses if fair value increases. ASU 2016-13 will be effective for fiscal years beginning after December 15, 2019 with early adoption permitted , and requires adoption using a modified retrospective approach, with certain exceptions. As of December 31, 2019, the Company has not elected to early adopt this guidance. Based on the composition of the Company’s investment portfolio as of December 3 1 , 2019, current market conditions and historical credit loss activity, the adoption of this standard is not expected to have a material impact on the Company’s consolidated financial statements. Additionally, for trade receivables, due to their short duration and the credit profile of the Company’s customers, the effect of transitioning from the incurred losses model to the expected losses model is not expected to be material. |
LICENSE AND COLLABORATION AGREE
LICENSE AND COLLABORATION AGREEMENTS | 12 Months Ended |
Dec. 31, 2019 | |
Organization Consolidation And Presentation Of Financial Statements [Abstract] | |
LICENSE AND COLLABORATION AGREEMENTS | 3. LICENSE AND COLLABORATION AGREEMENTS Roche Holding A.G. On December 21, 2019, the Company entered into a license, collaboration and option agreement and a stock purchase agreement (collectively, the “Roche Agreements”) with F. Hoffman-La Roche Ltd (“Roche”), providing Roche with exclusive commercial rights to SRP-9001 (AAVrh74.MHCK7.micro-dystrophin) (the “Lead Product”), the Company’s investigational gene therapy for DMD, outside the U.S. The Company retains all rights to SRP-9001 in the U.S. and will perform all development activities directed to obtaining and maintaining regulatory approvals for SRP-9001 in the U.S. and the EU. Further, global development expenses for SRP-9001 will be equally shared between the two parties. The closing of the transaction was subject to the expiration or termination of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976 and other customary conditions. The agreement became effective as of February 4, 2020, and closed on February 14, 2020. When the Roche Agreements became effective, the Company received payments totaling approximately $1.2 billion, consisting of $750.0 million in an up-front payment and $400.0 million in an equity investment. Additionally, the Company may receive up to approximately $1.7 billion in regulatory and sales milestones related to the Lead Product. Upon commercialization, the Company is also eligible to receive tiered royalty payments based on net sales. In addition, Roche has options to in-license (1) certain exon-skipping products that target the dystrophin gene to induce exon skipping, including eteplirsen, golodirsen, casimersen and SRP-5051, (2) certain gene therapy products other than SRP-9001 that encode and directly express dystrophin or a derivative thereof and (3) certain gene-editing products that modify, repair, or activate an endogenous dysfunctional dystrophin gene (collectively, the “Option Products”). If and when Roche decides to exercise the options, it will be required to make an option exercise payment, on a per Option Product basis, in an amount ranging between $20.0 million and $125.0 million. As of December 31, 2019, there was no accounting impact as a result of the execution of the Roche Agreements because the closing of the transaction did not occur until subsequent to year-end. Genethon In May 2017, the Company entered into a sponsored research agreement (the “Research Agreement”) with Genethon for its micro-dystrophin gene therapy program for the treatment of DMD. The Research Agreement provided the Company with an option to in-license the corresponding technology. On November 22, 2019, the Company exercised the option and entered into a license and collaboration with Genethon (the “Genethon Collaboration Agreement”). The Genethon Collaboration Agreement grants the Company with exclusive rights in the majority of the world (primarily excluding the EU) to Genethon’s micro-dystrophin gene therapy products (“Genethon Products”) and other micro-dystrophin gene therapy products (“Other Licensed Products”). Under the Genethon Collaboration Agreement, a joint steering committee will be established to plan, monitor and coordinate development activities for Genethon Products and Other Licensed Products. The Company and Genethon will be responsible for 75% and 25%, respectively, of development costs related to both the Genethon Products and the Other Licensed Products. For the year ended December 31, 2019, the Company recorded $9.0 million of research and development expense related to reimbursable development costs incurred for Genethon Products to date. Upon exercise of the option, the Company made an up-front payment of $28.0 million and may be liable for up to $157.5 million and $78.8 million in development, regulatory and sales milestones for the Genethon Products and Other Licensed Products, respectively. Furthermore, upon commercialization, the Company will be required to make tiered royalty payments based on net sales of the Genethon Products and the Other Licensed Products. The up-front payment represents rights to potential future benefits associated with ongoing research and development activities that have no alternative future use. Accordingly, this amount has been recorded as research and development expense in the accompanying consolidated statements of operations and comprehensive loss for the year ended December 31, 2019. As of December 31, 2019, no development or regulatory milestones were deemed probable of being achieved and, accordingly, no additional expense has been recognized. StrideBio, Inc. On November 14, 2019 (the “StrideBio Effective Date”), the Company entered into a collaboration and license agreement (the “StrideBio Collaboration Agreement”) and a stock purchase agreement (collectively, the “StrideBio Agreements”) with StrideBio, Inc. (“StrideBio”). Under the terms of the StrideBio Collaboration Agreement, StrideBio granted the Company exclusive worldwide licenses to develop, collaborate and commercialize StrideBio’s adeno-associated viral capsids for gene therapy with respect to multiple initial development targets (“Initial Targets”), and, at the option of the Company, additional development targets (“Additional Targets”). The Company also may be required to participate in StrideBio’s next preferred equity round of financing, subject to certain conditions. Both the Initial Targets and the Additional Targets are comprised of targets to which the Company will have the exclusive right to perform development activities (“Sarepta Development Targets”) and targets that the two parties will jointly develop through completion of Phase 1/2 clinical trials (“Joint Development Targets”). The Company also has the right to select additional Sarepta Development Targets and Joint Development Targets. For each Sarepta Development Target, StrideBio is responsible for initial research activities and each party bears its own costs while the Company is responsible for all costs following transfer of responsibilities to the Company for additional development. For each Joint Development Target, the parties will be responsible to develop a joint development plan for which the parties will share equally all costs through Phase 1/2 of clinical trials after which the Company will be solely responsible for the continued development, regulatory approval and commercialization of the target, including all related costs. The Company and StrideBio will also form a joint steering committee to oversee the collaboration activities. As a result of execution of the StrideBio Agreements, the Company recognized an up-front expense of $46.9 million, consisting of a cash payment of $17.5 million and 301,980 shares of the Company’s common stock delivered to StrideBio equal to $29.4 million. For Sarepta Development Targets and Joint Development Targets, respectively, the Company may be liable for up to $450.0 million and $835.0 million in development, regulatory and sales milestone payments per target. Furthermore, the Company may be obligated to pay StrideBio up to $42.5 million in additional fees when and if Additional Targets are selected. Additionally, upon commercialization, the Company may be required to make tiered royalty payments based on net sales of each target. The total up-front payment of $46.9 million, representing the fair value of the common shares delivered and cash, represents rights to potential future benefits associated with ongoing research and development activities that have no alternative future use. Accordingly, this amount has been recorded as research and development expense in the accompanying consolidated statements of operations and comprehensive loss for the year ended December 31, 2019. As of December 31, 2019, no development or regulatory milestones were deemed probable of being achieved and, accordingly, no additional expense has been recognized. Myonexus Therapeutics In May 2018, the Company entered into a Warrant to Purchase Common Stock Agreement (“Warrant Agreement”) with Myonexus Therapeutics, Inc. (“Myonexus”), a clinical-stage gene therapy biotechnology company that was developing gene therapies for Limb-Girdle muscular dystrophies (“LGMD”). Pursuant to the terms of the Warrant Agreement, the Company made an up-front payment of $60.0 million to purchase an exclusive option to acquire Myonexus for $200.0 million plus sales-related and regulatory-related contingent payments. During the year ended December 31, 2018, the Company recorded $85.0 million to research and development expense in connection with the Warrant Agreement comprised of the $60.0 million up-front payment, two development milestone payments totaling $20.0 million, and a third development milestone for $5.0 million was deemed probable of being achieved as of December 31, 2018. On February 27, 2019, the Company announced that it exercised the exclusive option to acquire Myonexus. The final exercise price as negotiated between the Company and Myonexus was $165.0 million. In addition, the Company incurred transaction fees and other fees associated with the exercise of approximately $8.8 million. The Company may also be required to make up to $200.0 million in additional payments to selling shareholders of Myonexus based on the achievement of certain sales- and regulatory-related milestones. The acquisition closed on April 4, 2019. As a result of the acquisition, the Company added five LGMD gene therapy programs, including MYO-101, MYO-102 and MYO-201 that are currently in Phase 1/2 clinical trials, to its research and development portfolio. The acquisition of Myonexus has been accounted for as an asset acquisition as substantially all of the fair value of the gross assets acquired is concentrated in a group of similar identifiable assets (the five LGMD gene therapy programs). Additionally, the Company assessed whether any of the contingent payments met the definition of a derivative under ASC 815 and, therefore, should be accounted for as contingent consideration. The Company identified that one regulatory-related milestone (not solely based on drug approval by the FDA) met the definition of a derivative. The following table summarizes the total consideration for the asset acquisition and the value of assets acquired and liability assumed: Consideration (in thousands) Purchase price $ 165,000 Transactions costs and other fees 8,753 Contingent consideration 4,500 Total consideration $ 178,253 Assets Acquired (in thousands) Cash and cash equivalents $ 1,197 Prepaids 3,816 In-process research and development 173,240 Total assets acquired $ 178,253 Liability Assumed (in thousands) Contingent consideration 4,500 Total liability assumed $ 4,500 The acquired in-process research and development asset relates to the LGMD asset group. Due to the stage of development of this asset group, significant risk remains, and it is not yet probable that there is future economic benefit from this asset. Absent successful clinical results and regulatory approval, there is no alternative future use associated with the LGMD asset group. Accordingly, the value of this asset of $173.2 million The portion of the $200.0 million in contingent payments related to the sales milestone will be accrued when and if the sales milestone becomes probable of being achieved, and the related payment will be capitalized and amortized over the life of the patent. As of December 31, 2019, the sales milestone was not probable of being achieved. Lysogene S.A. In October 2018, the Company entered into a license and collaboration agreement to develop and commercialize LYS-SAF302, a gene therapy to treat MPS IIIA as well as an equity investment agreement with Lysogene S.A. (“Lysogene”). Under the license and collaboration agreement, in addition to the payment of up-front fees, the Company may be liable for a total of $102.8 million in development, regulatory and sales milestones. Furthermore, the Company may be required to make tiered royalty payments based on net sales of the LYS-SAF302 product subsequent to its commercialization. Under the equity investment agreement, the Company purchased 950,606 shares of common stock issued by Lysogene, representing 8% of the outstanding equity of Lysogene at the time of the transaction. As a result of execution of the agreements, for the year ended December 31, 2018, the Company recorded research and development expense of $44.8 million, consisting of $26.1 million related to the payment of up-front fees and $18.7 million related to the achievement of a development milestone. In addition, $1.9 million of the total up-front fees paid was allocated to the equity investment in Lysogene and recorded as an other non-current asset. Changes in the fair value of this equity investment are recorded to other (loss) income in the Company’s consolidated statements of operations and other comprehensive loss. As of December 31, 2019, no additional development or regulatory milestones were deemed probable of being achieved and, accordingly, no additional expense has been recognized. Further, t he changes in the fair value of the equity investment Lacerta Therapeutics In August 2018, the Company entered into a license, development and option agreement (the “Lacerta License Agreement”) and a Series A Preferred Stock Purchase Agreement (the “Stock Purchase Agreement”) with Lacerta Therapeutics, Inc. (“Lacerta”). Pursuant to the Lacerta License Agreement, the Company licensed exclusive worldwide rights to develop, manufacture and commercialize a pre-clinical Pompe product candidate (the “Pompe License”). Lacerta also granted the Company exclusive options to enter into exclusive license agreements to develop, manufacture and commercialize other gene therapy product candidates for Sanfilipo syndrome and L-Amino Acid Decarboxylase Deficiency for additional consideration of $42.0 million (collectively, the “Options”) when (and if) the Options are exercised. Additionally, the Company may be liable for up to approximately $44.0 million in development, regulatory and sales milestones associated with the Pompe License and may be required to make tiered royalty payments based on net sales of the Pompe product subsequent to its commercialization. Under the Stock Purchase Agreement, the Company purchased approximately 4.5 million shares of Series A preferred stock issued by Lacerta. Under the agreements, the Company made an up-front payment of $38.0 million to Lacerta, $30.0 million and $8.0 million of which were allocated to the Series A preferred stock investment and the Pompe License, respectively. The amount allocated to the Pompe License represents rights to potential future benefits associated with ongoing research and development activities that have no alternative future use. Accordingly, this amount was recorded as research and development expense in the accompanying consolidated statements of operations and comprehensive loss for the year ended December 31, 2018. As of December 31, 2019, no development or regulatory milestones were deemed probable of being achieved and, accordingly, no additional expense has been recognized. The $30.0 million allocated to the Series A preferred stock investment was initially measured at cost and is classified as an other non-current asset in the accompanying consolidated balance sheets. Changes in the carrying value of the investment are reported as a component of earnings whenever there are observable price changes in orderly transactions for identical or similar investments of Lacerta in the future. For the years ended December 31, 2019 and 2018, the Company did not record any changes in carrying value of the investment as Lacerta did not issue identical or similar shares during the corresponding periods. Nationwide Children’s Hospital In December 2016, the Company entered into an exclusive option agreement with Nationwide Children’s Hospital (“Nationwide”) from which the Company obtained an exclusive right to acquire a worldwide license of the micro-dystrophin gene therapy technology for DMD and Becker muscular dystrophy. In October 2018, the Company exercised the option and entered into a license agreement with Nationwide (“Nationwide License Agreement”). Pursuant to the Nationwide License Agreement, the Company licensed exclusive worldwide rights to develop, manufacture and commercialize micro-dystrophin gene therapy product candidates. Under the agreement, the Company made an up-front payment of $1.0 million to Nationwide, which was recorded as research and development expense in the accompanying consolidated statements of operations and comprehensive loss for the year ended December 31, 2018. Additionally, the Company may be required to make up to $29.0 million in development, regulatory and sales milestone payments per micro-dystrophin product and low-single-digit royalty payments based on net sales of the micro-dystrophin products upon commercialization. As of December 31, 2019, no development or regulatory milestones were deemed probable of being achieved and, accordingly, no additional expense has been recognized. BioMarin Pharmaceutical, Inc. In July 2017, the Company and the University of Western Australia (“UWA”) entered into a settlement agreement with BioMarin Pharmaceutical, Inc. (“BioMarin”). On the same day, the Company entered into a license agreement, which was subsequently amended in April 2019, with BioMarin and Academisch Ziekenhuis Leiden (“AZL”) (collectively with the Company, UWA and BioMarin, the “Settlement Parties”). Under these agreements and amendment, BioMarin agreed to provide the Company with an exclusive license to certain intellectual property with an option to convert the exclusive license into a co-exclusive license and the Settlement Parties agreed to stop most existing efforts to continue with ongoing litigation and opposition and other administrative proceedings concerning BioMarin’s intellectual property. As a result of execution of the agreements, the Company made total up-front payments of $35.0 million. Additionally, the Company may be liable for up to approximately $65.0 million in regulatory and sales milestones for eteplirsen as well as casimersen and golodirsen. BioMarin is also eligible to receive tiered royalty payments, ranging from 4% to 8%, based on the net sales for the two products and product candidate. The royalty terms under the license agreement will expire in March 2024 in the U.S., December 2024 in the EU and no later than December 2024 in other countries. Of the $35.0 million paid to BioMarin, $28.4 million was expensed as incurred and $6.6 million was recorded as an intangible asset, representing the fair value of the U.S. license to BioMarin’s intellectual property. The intangible asset is being amortized on a straight-line basis over the remaining life of the patent and has a carrying value of $4.2 million as of December 31, 2019. The FDA approval of VYONDYS 53 in December 2019 resulted in a settlement charge to BioMarin of $10.0 million and has been expensed as incurred. No regulatory or sales milestones were achieved for the years ended December 31, 2018 or 2017. For the years ended December 31, 2019, 2018 and 2017, the Company recognized royalty expense of $19.4 million, $15.1 million and $4.7 million, respectively. As of December 31, 2019, no other regulatory or sales milestones were deemed probable of being achieved and, accordingly, no additional in-licensed rights or expenses have been recognized. University of Western Australia In April 2013, the Company and UWA entered into an amendment to an existing exclusive license agreement relating to the treatment of DMD by inducing the skipping of certain exons. The agreement was further amended in June 2016. Under the amended agreement, the Company may be obligated to make payments to UWA totaling up to $26.0 million upon the achievement of certain development, regulatory and sales milestones. Additionally, the Company is required to pay a low-single-digit percentage royalty on net sales of products covered by issued patents licensed under the agreements with UWA. Corresponding with the FDA approval of EXONDYS 51 in 2016, the Company recorded a $1.0 million milestone payment as an in-licensed right intangible asset in its consolidated balance sheet. Similarly, corresponding to the milestone payments associated with the FDA approval of VYONDYS 53 in December 2019, the Company recorded a $0.5 million milestone payment as an in-licensed right intangible asset in its consolidated balance sheet. Both intangible assets are being amortized on a straight-line basis over the remaining life of the relevant patents and have a combined carrying value of $1.1 million as of December 31, 2019. For the year ended December 31, 2019, the Company recorded $3.5 million in royalty expense, which is included in cost of sales, related to agreements with UWA with no such an expense incurred in 2018 or 2017. As of December 31, 2019, no other development, regulatory or sales milestones were deemed probable of being achieved and, accordingly, no additional in-licensed rights or expenses have been recognized. Milestone Obligations As of December 31, 2019, the Company may be obligated to make up to $3.0 billion of future development, regulatory, commercial, and up-front royalty payments associated with its collaboration and license agreements. For the years ended December 31, 2019, 2018 and 2017, the Company recognized approximately $113.2 million, $142.4 million and $22.0 million relating to certain up-front, milestone and settlement payments as research and development expense, respectively, under these agreements. The Company is also obligated to pay royalties on net sales of certain of its products related to these collaboration and license agreements. The royalty rates range from the low-single-digit to high teens percentages for both inside and outside the U.S. |
GAIN FROM SALE OF PRIORITY REVI
GAIN FROM SALE OF PRIORITY REVIEW VOUCHER | 12 Months Ended |
Dec. 31, 2019 | |
Goodwill And Intangible Assets Disclosure [Abstract] | |
GAIN FROM SALE OF PRIORITY REVIEW VOUCHER | 4. GAIN FROM SALE OF PRIORITY REVIEW VOUCHER In March 2017, the Company completed a sale of its Rare Pediatric Disease Priority Review Voucher (“PRV”) to Gilead Sciences, Inc. for $125.0 million, which was recorded as a gain from sale of the PRV as it did not have a carrying value at the time of the sale. |
FAIR VALUE MEASUREMENTS
FAIR VALUE MEASUREMENTS | 12 Months Ended |
Dec. 31, 2019 | |
Fair Value Disclosures [Abstract] | |
FAIR VALUE MEASUREMENTS | 5. FAIR VALUE MEASUREMENTS The tables below present information about the Company’s financial assets that are measured and carried at fair value and indicate the level within the fair value hierarchy of the valuation techniques it utilizes to determine such fair value: Fair Value Measurement as of December 31, 2019 Total Level 1 Level 2 Level 3 (in thousands) Assets Money market funds $ 203,410 $ 203,410 $ — $ — Government and government agency bonds 809,159 809,159 — — Strategic equity investments 31,937 1,937 — 30,000 Certificates of deposit 1,001 1,001 — — Total assets $ 1,045,507 $ 1,015,507 $ — $ 30,000 Liabilities Contingent consideration 5,200 — — 5,200 Total liabilities $ 5,200 $ — $ — $ 5,200 Fair Value Measurement as of December 31, 2018 Total Level 1 Level 2 Level 3 (in thousands) Assets Money market funds $ 42,920 $ 42,920 $ — $ — Commercial paper 125,907 — 125,907 — Government and government agency bonds 760,235 760,235 — — Corporate bonds 43,468 43,468 — — Strategic equity investments 31,739 1,739 — 30,000 Certificates of deposit 1,001 1,001 — — Total $ 1,005,270 $ 849,363 $ 125,907 $ 30,000 The Company’s assets with fair value categorized as Level 1 within the fair value hierarchy include money market funds, government and government agency bonds, corporate bonds, certificates of deposit, and the Company’s strategic investment in Lysogene, a publicly traded company in France, as more fully described in Note 3, License and Collaboration Agreements The Company’s assets with fair value categorized as Level 2 within the fair value hierarchy consist of commercial paper and government and government agency bonds. These assets have been initially valued at the transaction price and subsequently valued, at the end of each reporting period, through income-based approaches utilizing market observable data. The Company’s assets with fair value categorized as Level 3 within the fair value hierarchy consists of a strategic investment in Series A preferred stock of Lacerta as more fully described in Note 3, License and Collaboration Agreements At the end of each reporting period, the fair value will be adjusted if Lacerta issues similar or identical equity securities or when there is a triggering event for impairment. There were no changes in the fair value of the Lacerta strategic investment during the year ended December 31, 2019. The Company’s contingent consideration liability with fair value categorized as Level 3 within the fair value hierarchy relate to the regulatory-related contingent payments to Myonexus selling shareholders as well as to an academic institution under a separate license agreement that meet the definition on a derivative. For more information related to Myonexus, please read Note 3, License and Collaboration Agreements The carrying amounts reported in the consolidated balance sheets for cash and cash equiva lents, accounts receivable and accounts payable approximate fair value because of the immediate or short-term maturity of these financial instruments. For fair value information related to the Company’s debt facilities, please read Note 1 3 , Indebtedness . |
CASH, CASH EQUIVALENTS AND MARK
CASH, CASH EQUIVALENTS AND MARKETABLE SECURITIES | 12 Months Ended |
Dec. 31, 2019 | |
Cash And Cash Equivalents [Abstract] | |
CASH, CASH EQUIVALENTS AND MARKETABLE SECURITIES | 6. CASH, CASH EQUIVALENTS AND MARKETABLE SECURITIES The following table summarizes the Company’s financial assets with maturities of equal to or less than three months from the date of purchase included in cash equivalents in the consolidated balance sheets for each of the periods indicated: As of December 31, 2019 2018 (in thousands) Money market funds $ 203,410 $ 42,920 Government and government agency bonds 519,491 111,587 Commercial paper — 14,940 Total $ 722,901 $ 169,447 It is the Company’s policy to mitigate credit risk in its financial assets by maintaining a well-diversified portfolio that limits the amount of exposure as to maturity and investment type. The weighted average maturity of the Company’s available-for-sale securities as of December 31, 2019 and 2018 was approximately two months. The following tables summarize the Company’s cash, cash equivalents and investments for each of the periods indicated: As of December 31, 2019 Amortized Cost Gross Unrealized Gains Gross Unrealized Losses Fair Market Value (in thousands) Cash and money market funds $ 315,589 $ — $ — $ 315,589 Government and government agency bonds 809,090 71 (2 ) 809,159 Total cash, cash equivalents and investments $ 1,124,679 $ 71 $ (2 ) $ 1,124,748 As reported: Cash and cash equivalents $ 835,044 $ 36 $ — $ 835,080 Short-term investments 289,635 35 (2 ) 289,668 Total cash, cash equivalents and investments $ 1,124,679 $ 71 $ (2 ) $ 1,124,748 As of December 31, 2018 Amortized Cost Gross Unrealized Gains Gross Unrealized Losses Fair Market Value (in thousands) Cash and money market funds $ 244,302 $ — $ — $ 244,302 Commercial paper 125,907 — — 125,907 Government and government agency bonds 760,258 12 (35 ) 760,235 Corporate bonds 43,544 — (76 ) 43,468 Total cash, cash equivalents and investments $ 1,174,011 $ 12 $ (111 ) $ 1,173,912 As reported: Cash and cash equivalents $ 370,827 $ 3 $ (1 ) $ 370,829 Short-term investments 803,184 9 (110 ) 803,083 Total cash, cash equivalents and investments $ 1,174,011 $ 12 $ (111 ) $ 1,173,912 |
ACCOUNTS RECEIVABLE AND RESERVE
ACCOUNTS RECEIVABLE AND RESERVES FOR PRODUCT SALES | 12 Months Ended |
Dec. 31, 2019 | |
Receivables Net Current [Abstract] | |
ACCOUNTS RECEIVABLE AND RESERVES FOR PRODUCT SALES | 7. ACCOUNTS RECEIVABLE AND RESERVES FOR PRODUCT SALES The following table summarizes the components of the Company’s accounts receivable for the periods indicated: As of December 31, 2019 2018 (in thousands) Product sales, net of discounts and allowances $ 90,409 $ 48,252 Government contract receivables 470 792 Total accounts receivable, net $ 90,879 $ 49,044 The balance for government contract receivables for both periods presented is subject to government audit and will not be collected until the completion of the audit. The following table summarizes an analysis of the change in reserves for discounts and allowances for the periods indicated: Chargebacks Rebates Prompt Pay Other Accruals Total (in thousands) Balance, as of December 31, 2017 $ 995 $ 6,959 $ 169 $ 464 $ 8,587 Provision 12,284 28,420 2,624 5,286 48,614 Payments/credits (11,901 ) (11,103 ) (2,255 ) (3,432 ) (28,691 ) Balance, as of December 31, 2018 $ 1,378 $ 24,276 $ 538 $ 2,318 $ 28,510 Provision 9,698 44,749 4,897 9,643 68,987 Payments/credits (10,488 ) (24,287 ) (3,929 ) (7,290 ) (45,994 ) Balance, as of December 31, 2019 $ 588 $ 44,738 $ 1,506 $ 4,671 $ 51,503 The following table summarizes the total reserves above included in the Company’s consolidated balance sheets for the periods indicated: As of December 31, 2019 2018 (in thousands) Reduction to accounts receivable $ 6,254 $ 2,364 Component of accrued expenses 45,249 26,146 Total reserves $ 51,503 $ 28,510 |
INVENTORY
INVENTORY | 12 Months Ended |
Dec. 31, 2019 | |
Inventory Disclosure [Abstract] | |
INVENTORY | 8. INVENTORY The following table summarizes the components of the Company’s inventory for each of the periods indicated: As of December 31, 2019 2018 (in thousands) Raw materials $ 82,030 $ 71,313 Work in progress 88,031 47,279 Finished goods 1,318 6,853 Total inventory $ 171,379 $ 125,445 |
OTHER ASSETS
OTHER ASSETS | 12 Months Ended |
Dec. 31, 2019 | |
Deferred Costs Capitalized Prepaid And Other Assets Disclosure [Abstract] | |
OTHER ASSETS | 9. OTHER ASSETS The following table summarizes the Company’s other current assets for each of the periods indicated: As of December 31, 2019 2018 (in thousands) Manufacturing-related deposits and prepaids $ 54,276 $ 39,036 Prepaid clinical and pre-clinical expenses 8,263 9,706 Prepaid maintenance services 4,366 2,994 Leasehold improvement receivable 3,059 13,474 Prepaid insurance 2,573 1,006 Prepaid income tax 2,114 2,130 Prepaid research expenses 2,007 1,932 Other 5,249 7,504 Total other current assets $ 81,907 $ 77,782 The following table summarizes the Company’s other non-current assets for each of the periods indicated: As of December 31, 2019 2018 (in thousands) Manufacturing-related deposits and prepaids $ 122,091 $ 62,821 Strategic investments 31,937 31,739 Restricted cash and investments 9,566 1,001 Prepaid clinical expenses 4,665 7,541 Alternative minimum tax credit 3,367 3,367 Other 2,233 825 Total other non-current assets $ 173,859 $ 107,294 |
PROPERTY AND EQUIPMENT, NET
PROPERTY AND EQUIPMENT, NET | 12 Months Ended |
Dec. 31, 2019 | |
Property Plant And Equipment [Abstract] | |
PROPERTY AND EQUIPMENT, NET | 10. PROPERTY AND EQUIPMENT, NET Property and equipment are recorded at historical cost, net of accumulated depreciation. The following table summarizes components of property and equipment, net for each of the periods indicated: As of December 31, 2019 2018 (in thousands) Leasehold improvements $ 53,950 $ 20,937 Software and computer equipment 30,683 15,774 Lab equipment 30,053 17,659 Building and improvements 23,108 22,972 Furniture and fixtures 7,090 3,227 Land 5,183 4,158 Land improvements 3,403 — Office equipment 1,157 436 Construction in progress 25,988 40,010 Property and equipment, gross 180,615 125,173 Less: accumulated depreciation (50,995 ) (28,149 ) Property and equipment, net $ 129,620 $ 97,024 For the years ended December 31, 2019, 2018 and 2017, depreciation expense totaled $22.8 million, $10.2 million and $6.4 million, respectively. |
INTANGIBLE ASSETS
INTANGIBLE ASSETS | 12 Months Ended |
Dec. 31, 2019 | |
Goodwill And Intangible Assets Disclosure [Abstract] | |
INTANGIBLE ASSETS | 11. INTANGIBLE ASSETS The following table summarizes the components of the Company’s intangible assets for each of the periods indicated: As of December 31, 2019 2018 (in thousands) Patents $ 8,902 $ 7,227 In-licensed rights 8,073 7,573 Software licenses 1,029 626 Intangible assets, gross 18,004 15,426 Less: accumulated amortization (5,507 ) (3,852 ) Intangible assets, net $ 12,497 $ 11,574 The in-licensed rights relate to agreements with BioMarin and UWA. As a result of the FDA approval of EXONDYS 51 and VYONDYS 53, the Company recorded in-licensed rights of $1.0 million and $0.5 million, respectively. Following the execution of the settlement and license agreements with BioMarin in July 2017, the Company recorded a $6.6 million intangible asset related to EXONDYS 51 in the U.S. The in-licensed rights are being amortized on a straight-line basis over the remaining life of the related patent because the life of the related patent reflects the expected time period that the Company will benefit from the in-licensed right. For more information about the in-licensed rights, please read Note 3, License and Collaboration Agreements Patent amortization expense was $0.4 million, $0.7 million and $0.6 million for the years ended December 31, 2019, 2018 and 2017, respectively. Total amortization expense was $1.7 million, $2.1 million and $1.7 million for the years ended December 31, 2019, 2018 and 2017, respectively. The Company also expensed the remaining net book value of previously capitalized patents that were later abandoned of $0.2 million, $0.1 million and $0.6 million for the years ended December 31, 2019, 2018 and 2017, respectively, which were included in research and development expenses on the consolidated statements of operations and comprehensive loss. Additionally, in 2018, the Company reviewed its patent portfolio and identified technology that the Company will no longer pursue. As a result, the Company impaired these patent assets and recorded $3.8 million in impairment loss for the year ended December 31, 2018, which was included in research and development expense on the consolidated statement of operations and comprehensive loss. There was no such loss recorded in the years ended December 31, 2019 and 2017. The following table summarizes the estimated future amortization for intangible assets: As of December 31, 2019 (in thousands) 2020 $ 1,358 2021 1,168 2022 1,156 2023 1,156 2024 1,149 Thereafter 6,510 Total $ 12,497 |
ACCRUED EXPENSES
ACCRUED EXPENSES | 12 Months Ended |
Dec. 31, 2019 | |
Payables And Accruals [Abstract] | |
ACCRUED EXPENSES | 12. ACCRUED EXPENSES The following table summarizes the Company’s accrued expenses for each of the periods indicated: As of December 31, 2019 2018 (in thousands) Product revenue related reserves $ 45,249 $ 26,146 Accrued employee compensation costs 43,240 24,692 Accrued contract manufacturing costs 27,622 15,794 Accrued milestone expense 18,390 24,020 Accrued clinical and pre-clinical costs 18,010 11,396 Accrued professional fees 10,707 11,319 Accrued collaboration cost-sharing 9,000 2,167 Accrued royalties 6,301 8,254 Other 7,008 10,307 Total accrued expenses $ 185,527 $ 134,095 |
INDEBTEDNESS
INDEBTEDNESS | 12 Months Ended |
Dec. 31, 2019 | |
Debt Disclosure [Abstract] | |
INDEBTEDNESS | 13. INDEBTEDNESS 2024 Convertible Notes On November 14, 2017, the Company issued $570.0 million senior notes due on November 15, 2024 (the “2024 Notes”). The 2024 Notes were issued at face value and bear interest at the rate of 1.50% per annum, payable semi-annually in cash on each May 15 and November 15, commencing on May 15, 2018. The 2024 Notes contain customary covenants and events of default, occurrence of which will permits the certain holders to accelerate all outstanding obligations, including principal and interest. Upon conversion, the Company may pay cash, shares of its common stock or a combination of cash and stock, as determined by the Company in its discretion. The 2024 Notes may be convertible into 7,763,552 shares of the Company’s common stock under certain circumstances prior to maturity at a conversion rate of 13.621 The Company allocated the proceeds received from issuance of the 2024 Notes between the liability component and the embedded conversion option, or equity component. The liability component was determined by measuring the fair value of similar notes that do not include the embedded conversion option. The Company allocated $161.1 million to the equity component, which was determined by deducting the fair value of the liability component from the par value of the 2024 Notes. The equity component, net of allocated offering costs of $4.2 million, was recorded as an increase additional paid-in capital. The equity component, plus $10.6 million of offering costs allocated to the liability component, represent the total debt discount on the 2024 Notes at issuance. The debt discount is amortized under the effective interest method and recorded as additional interest expense over the life of the 2024 Notes. The effective interest rate on the liability component of the 2024 Notes for the year ended December 31, 2019, 2018 and 2017 was 6.9%. Upon the occurrence of a “fundamental change”, which includes (1) change in beneficial ownership of the Company where any person/group possesses more than 50% of the voting power of the Company, (2) consolidation or merger of the Company, (3) shareholder approval of a liquidation plan or (4) the Company is delisted from NYSE or NASDAQ, the holders may require the Company to repurchase all or a portion of the 2024 Notes for cash at 100% of the principal amount of the 2024 Notes being purchased, plus any accrued and unpaid interest. Additionally, upon the occurrence of a “make-whole fundamental change” prior to the maturity date, the Company shall adjust the conversion rate on a sliding scale basis detailed in the agreement To minimize the impact of potential dilution upon conversion of the 2024 Notes, the Company separately entered into capped call transactions with certain counterparties. The capped calls have a strike price of $73.42 and a cap price of $104.88 and are exercisable when and if the 2024 Notes are converted. If, upon conversion of the 2024 Notes, the price of the Company’s common stock is between the strike price and the cap price of the capped calls, the counterparties will deliver shares of the Company’s common stock and/or cash with an aggregate value equal to the difference between the price of the Company’s common stock at the conversion date and the strike price, multiplied by the number of shares of the Company’s common stock related to the capped calls being exercised. The Company paid $50.9 million for these capped calls transactions, which was recorded as additional paid-in capital. Term Loan s and Revolving Line of Credit December 2019 Term Loan On December 13, 2019, the Company entered into a loan agreement (the “Loan Agreement”) which provides a term loan (“December 2019 Term Loan”) of $500.0 million with Biopharma Credit PLC and Biopharma Credit Investments V (Master) LP (collectively, the “Lenders”). The 2019 Term Loan has two tranches: A and B, each of which has a loan amount of $250.0 million. On December 20, 2019, Sarepta drew down tranche A of the 2019 Term Loan and has the option of draw down tranche B of the loan no later than December 31, 2020, subject to certain conditions. The December 2019 Term Loan matures on December 20, 2023, when the principal amount of the loan will become due. Borrowings under the Loan Agreement bore interest at a rate per annum equal to 8.5%, which shall be payable quarterly in arrears. The Company is also required to pay the Lenders (1) a fee of 1.75% of the amounts drawn under both tranche A and tranche B due on closing (if and when drawn down), (2) a fee of 2.0% of principal amount on the December 2019 Term Loan maturity date or prepayment amount on each prepayment date and (3) certain out-of-pocket expenses incurred by the Lenders. The Company may voluntarily prepay, in whole or in part, the outstanding balance under the December 2019 Term Loan at any time after the tranche A closing date. Upon occurrence of a change in control, the Company is required to repay any amounts outstanding under the December 2019 Term Loan. In the event of a permitted prepayment, the Company would be obligated to make the following premium payments: (1) an amount equal to the sum of all interest that would have been accrued and payable from the prepayment date through December 20, 2021 (“Makewhole Amount”), and (2) an amount equal to 1.0% to 2.0% of the prepayment amount depending on the date of the prepayment (“Prepayment Premium”). The Loan Agreement As of December 31, 2019, the Company recorded a debt discount of $9.4 million and debt issuance costs of $0.7 million, both of which are being treated as deduction to the carrying value of the December 2019 Term Loan and amortized as interest expense over the term of the loan based on an effective interest method. The debt discount of $9.4 million is inclusive of (1) the initial fee of 1.75% payable to the Lenders and (2) the 2.0% fee payable to the Lenders at maturity or prepayment of the December 2019 Term Loan. This amount is recorded within other long-term liabilities in the Company’s consolidated balance sheets. After certain debt discounts and debt issuance costs, the Company received net proceeds of $244.9 million. July 2017 Term Loan and Revolving Line of Credit In July 2017, the Company entered into an amended and restated credit agreement (the “Amended and Restated Credit and Security Agreement”) with MidCap Financial Trust (“MidCap”) which provided a term loan of $60.0 million, bearing interest at a rate of 6.25%, plus the one-month London Interbank Offered Rate (“LIBOR”). In addition, in July 2017, the Company entered into a revolving credit and security agreement (the “Revolving Credit Agreement”) with MidCap which provided an aggregate revolving loan commitment of $40.0 million, bearing interest at a rate of 3.95%, plus the one-month LIBOR. In September 2018, the Company terminated both the Amended and Restated Credit and Security Agreement and the Revolving Credit Agreement with MidCap and paid off all amounts due thereunder, including any accrued and unpaid interest. As a result, the Company recorded a debt extinguishment loss of $2.3 million primarily related to the write-off of unamortized debt issuance costs and prepayment fees. As of December 31, 2019, the Company recorded approximately $681.9 million as long-term debt on the consolidated balance sheets. For the years ended December 31, 2019, 2018 and 2017, the Company recorded $30.7 million, $33.7 million and $5.8 million of interest expense, respectively. The following table summarizes the Company’s debt facilities for the periods indicated: As of December 31, 2019 2018 (in thousands) Principal amount of the 2024 Notes $ 570,000 $ 570,000 Unamortized discount - equity component (120,182 ) (140,206 ) Unamortized discount - debt issuance costs (7,922 ) (9,240 ) Net carrying value of 2024 Notes 441,896 420,554 Principal amount of the 2019 Term Loan 250,000 — Unamortized discounts (9,996 ) — Net carrying value of 2019 Term Loan 240,004 — Total carrying value of debt facilities 681,900 420,554 Fair value of 2024 Notes 1,141,288 952,681 Fair value of 2019 Term Loan 250,000 — Total fair value of debt facilities $ 1,391,288 $ 952,681 The fair value of the 2024 Notes is based on open market trades and is classified as level 1 in the fair value hierarchy. The fair value of the December 2019 Term Loan, approximating its principal amount due to the close proximity of the reporting date and the tranche A close date, is classified as level 2 in the fair value hierarchy as it is based on market observable inputs. The following table summarizes the total gross payments due under the Company’s debt arrangements: As of December 31, 2019 (in thousands) 2020 $ — 2021 — 2022 — 2023 250,000 2024 570,000 Thereafter — Total payments $ 820,000 |
EQUITY
EQUITY | 12 Months Ended |
Dec. 31, 2019 | |
Equity [Abstract] | |
EQUITY | 14. EQUITY In March 2019, the Company sold approximately 2.6 million shares of common stock through an underwritten public offering. The offering price was $140.41 per share. The Company received net proceeds of approximately $365.4 million from the offering, net of commission and offering expenses of approximately $0.3 million. In November 2019, the Company issued approximated 0.3 million shares of common stock with a fair value of $29.4 million as part of the up-front consideration to StrideBio (see Note 3, License and Collaboration Agreements In November 2018, the Company sold approximately 4.1 million shares of common stock through an underwritten public offering, including 0.3 million shares sold to the underwriters. The offering price was $131.00 per share. The Company received net proceeds of approximately $513.4 million from the offering, net of commission and offering expenses of approximately $24.6 million. In July 2017, the Company sold approximately 8.8 million shares of common stock through an underwritten public offering, including 1.2 million shares sold to the underwriters. The offering price was $42.50 per share. The Company received net proceeds of approximately $354.0 million from the offering, net of commission and offering expenses of approximately $20.0 million. |
STOCK-BASED COMPENSATION
STOCK-BASED COMPENSATION | 12 Months Ended |
Dec. 31, 2019 | |
Disclosure Of Compensation Related Costs Sharebased Payments [Abstract] | |
STOCK-BASED COMPENSATION | 1 5 . STOCK-BASED COMPENSATION In June 2011, the Company’s stockholders approved the 2011 Equity Incentive Plan (“2011 Plan”). The 2011 Plan, which as amended authorized 16.0 million shares of common stock to be issued, allowed for the grant of stock options, stock appreciation rights (“SARs”), restricted stock awards (“RSAs”), restricted stock units (“RSUs”), performance shares and performance units. During 2018, the 2011 Plan was merged into the 2018 Plan (defined below). As a result, there were no shares of common stock remaining available for future grant under the 2011 Plan. In June 2013, the Company’s stockholders approved the 2013 Employee Stock Purchase Plan (“ESPP”) with approximately 0.3 million shares of common stock available to be issued. In June 2016 and 2019, the Company’s stockholders approved additional approximately 0.3 million and 0.5 million shares, respectively, of common stock available to be issued to be added to the 2013 ESPP. As of December 31, 2019, 0.6 million shares of common stock remain available for future grant under the 2013 ESPP. In September 2014, the Company initiated the 2014 Employment Commencement Incentive Plan (“2014 Plan”) with approximately 0.6 million shares of common stock available to be issued. In October 2015, June 2017 and July 2018, the 2014 Plan was increased by 1.0 million, 3.8 million and 1.2 million shares of common stock available to be issued, respectively. As of December 31, 2019, 0.6 million shares of common stock remain available for future grant under the 2014 Plan. In June 2018, the Company’s stockholders approved the 2018 Equity Incentive Plan (“2018 Plan”). The 2018 Plan, which authorized 2.9 million shares of common stock to be issued, allows for the grant of stock options, SARs, RSAs, RSUs, performance shares and performance units. The 2011 Plan was merged into the 2018 Plan and, as a result, all remaining shares in the 2011 Plan were transferred into the 2018 Plan. As of December 31, 2019, 3.4 million shares of common stock remain available for future grant under the 2018 Plan. Stock Options In general, stock options have a ten-year term and vest over a four-year period, with one-fourth of the underlying shares vesting on the first anniversary of the grant and 1/48th of the underlying shares vesting monthly thereafter, such that the underlying shares will be fully vested on the fourth anniversary of the grant, subject to the terms of the applicable plan under which they were granted. The fair values of stock options granted during the periods presented are measured on the date of grant using the Black-Scholes-Merton option-pricing model, with the following assumptions: For the Year Ended December 31, 2019 2018 2017 Risk-free interest rate (1) 1.4 - 2.5% 2.5 - 3.0% 1.6 - 2.1% Expected dividend yield (2) — — — Expected term (3) 5.04 years 5.06 years 4.2 - 4.8 years Expected volatility (4) 52.5 - 68.9% 52.4 - 60.8% 54.0 - 63.0% (1) The risk-free interest rate is estimated using an average of Treasury bill interest rates over a historical period commensurate with the expected term of the option that correlates to the prevailing interest rates at the time of grant. (2) The expected dividend yield is zero as the Company has not paid any dividends to date and does not expect to pay dividends in the future. (3) The expected term is estimated using historical exercise behavior. (4) The expected volatility is the implied volatility in exchange-traded options of the Company’s common stock. The amounts estimated according to the Black-Scholes-Merton option-pricing model may not be indicative of the actual values realized upon the exercise of these options by the holders. The following tables summarize the Company’s stock option activity for each of the periods indicated: For the Year Ended December 31, 2019 2018 2017 Weighted Weighted Weighted Average Average Average Exercise Exercise Exercise Shares Price Shares Price Shares Price Grants outstanding at beginning of the period 8,391,171 $ 46.09 8,806,204 $ 29.74 5,436,951 $ 22.70 Granted 1,429,652 132.97 2,152,439 90.15 4,805,722 (1) 35.09 Exercised (1,055,715 ) 30.73 (2,119,306 ) 22.89 (792,845 ) 17.40 Expired and forfeited (418,760 ) 84.15 (448,166 ) 46.11 (643,624 ) 25.44 Grants outstanding at end of the period 8,346,348 $ 61.01 8,391,171 $ 46.09 8,806,204 $ 29.74 Grants exercisable at end of the period 2,368,621 $ 45.33 2,304,791 $ 27.69 3,288,712 $ 24.76 Grants vested and expected to vest at end of the period 7,987,427 $ 58.65 6,643,835 $ 45.43 6,910,022 $ 28.49 (1) Includes 3,300,000 options with service and market conditions granted to the Company’s CEO. These options have a five-year The weighted-average grant date fair value per share of stock options granted during the years ended December 31, 2019, 2018 and 2017 was $70.93, $44.66 and $14.78, respectively. Weighted Aggregate Average Intrinsic Remaining Value Contractual (in thousands) Life (Years) Options outstanding at December 31, 2019 $ 587,191 7.5 Options exercisable at December 31, 2019 $ 199,438 6.0 Options vested and expected to vest at December 31, 2019 $ 578,938 7.4 The following table summarizes the Company’s stock options vested and exercised for each of the periods indicated: For the Year Ended December 31, 2019 2018 2017 (in thousands) Aggregate grant date fair value of stock options vested $ 50,878 $ 16,316 $ 18,225 Aggregate intrinsic value of stock options exercised $ 109,707 $ 158,936 $ 20,922 For the years ended December 31, 2019, 2018 and 2017, the Company has recognized approximately $0.1 million, $0.2 million and $0.9 million in stock-based compensation expense related to the options with performance-based criteria, respectively. Restricted Stock Awards The Company grants RSAs to members of its board of directors and certain employees. The following table summarizes the Company’s RSA activity for each of the periods indicated: For the Year Ended December 31, 2019 2018 2017 Weighted Weighted Weighted Average Average Average Grant Date Grant Date Grant Date Shares Fair Value Shares Fair Value Shares Fair Value Grants outstanding at beginning of the period 252,321 $ 42.37 411,781 $ 37.23 153,170 $ 34.53 Granted — — 27,590 98.57 341,500 34.58 Vested (100,840 ) 40.54 (187,050 ) 39.34 (63,264 ) 14.60 Cancelled (15,356 ) 48.94 — — (19,625 ) 43.02 Grants outstanding at end of the period 136,125 $ 42.98 252,321 $ 42.37 411,781 $ 37.23 In September 2016, the Company granted certain executives RSAs with a performance conditions relating to certain sales targets. If the sales targets are achieved within the required time frame, the number of RSAs may be increased from 71,925 to 89,906 shares. In December 2017, the Company modified the expiration date of these RSAs from June 30, 2018 to January 1, 2019. As a result of this modification, the fair value per RSA was changed from $48.94 to $54.29. Through December 31, 2017, the Company had not recorded any stock-based compensation expense associated with these awards as the achievement of the performance conditions was not deemed probable. As of December 31, 2018, the first sales target related to these RSAs was achieved and, accordingly, the Company recognized approximately $3.3 million of stock-based compensation expense during the year ended December 31, 2018. The second target was not achieved and the shares related to this target were subsequently cancelled and no expense was recognized. Restricted Stock Units The Company also grants RSUs to members of its board of directors and employees. The following table summarizes the Company’s RSU activity for the periods indicated: For the Year Ended December 31, 2019 2018 2017 Weighted Weighted Weighted Average Average Average Grant Date Grant Date Grant Date Shares Fair Value Shares Fair Value Shares Fair Value Grants outstanding at beginning of the period 251,298 $ 81.21 66,552 $ 33.72 — $ — Granted 511,283 131.18 230,736 87.95 181,029 33.03 Vested (84,068 ) 75.12 (30,276 ) 33.23 (78,017 ) 32.63 Cancelled (72,639 ) 103.03 (15,714 ) 71.45 (36,460 ) 32.63 Grants outstanding at end of the period 605,874 $ 121.61 251,298 $ 81.21 66,552 $ 33.72 In March 2017, the Company granted certain executives 156,029 RSUs with performance conditions relating to certain sales target and regulatory milestones, which were achieved between June 2017 and March 2019. As of December 31, 2019, there were no RSUs with performance conditions remaining to be vested. For the years ended December 31, 2019, 2018 and 2017, the Company recognized approximately $0.5 million, $0.2 million and $2.9 million of stock-based compensation expense, respectively. Stock Appreciation Rights The Company issues SARs on the same terms as options granted to employees. The grant date fair value of the SARs is determined using the same valuation assumptions as for stock options described above. Stock-based compensation expense is recognized on a straight-line basis over the vesting period of the SARs. The following table summarizes the Company’s SAR activity for each of the periods indicated: For the Year Ended December 31, 2019 2018 2017 Weighted Weighted Weighted Average Average Average Exercise Exercise Exercise Shares Price Shares Price Shares Price Grants outstanding at beginning of the period 100,000 $ 23.85 100,000 $ 23.85 100,000 $ 23.85 Exercised (100,000 ) $ (23.85 ) — $ — — $ — Grants outstanding at end of the period — $ — 100,000 $ 23.85 100,000 $ 23.85 Grants exercisable at end of the period — $ — 100,000 $ 23.85 100,000 $ 23.85 Grants vested and expected to vest at end of the period — $ — 100,000 $ 23.85 100,000 $ 23.85 2013 Employee Stock Purchase Plan Under the Company’s 2013 ESPP, participating employees purchase common stock through payroll deductions. The purchase price is equal to 85% of the lower of the closing price of the Company’s common stock on the first business day and the last business day of the relevant purchase period. The 24-month offering period will end between February 29, 2020 and August 31, 2021. The following table summarizes the Company’s ESPP activity for each of the periods indicated: For the Year Ended December 31, 2019 2018 2017 Number of shares purchased 92,086 75,094 102,698 Proceeds received (in millions) $ 5.1 $ 2.3 $ 1.4 Stock-based Compensation Expense For the years ended December 31, 2019, 2018 and 2017, total stock-based compensation expense was $78.6 million, $50.1 million and $30.5 million, respectively. Included in the amounts for the year ended December 31, 2017 is $2.1 million of stock-based compensation expense incurred in connection with the resignation of the Company’s former CEO. The following table summarizes stock-based compensation expense by function included within the consolidated statements of operations and comprehensive loss: For the Year Ended December 31, 2019 2018 2017 (in thousands) Research and development $ 27,681 $ 14,214 $ 8,542 Selling, general and administrative 50,921 35,913 21,923 Total stock-based compensation $ 78,602 $ 50,127 $ 30,465 The following table summarizes stock-based compensation expense by grant type included within the consolidated statements of operations and comprehensive loss: For the Year Ended December 31, 2019 2018 2017 (in thousands) Stock options $ 53,427 $ 37,671 $ 23,416 Restricted stock awards/units 20,103 10,632 5,295 Employee stock purchase plan 5,072 1,824 1,754 Total stock-based compensation $ 78,602 $ 50,127 $ 30,465 As of December 31, 2019, there was $181.1 million of total unrecognized stock-based compensation expense related to the Company’s stock-based compensation plans. The expense is expected to be recognized over a weighted-average period of approximately 3 years. Of this amount, $109.5 million relates to options with service conditions only, $22.1 million relates to awards with service and market conditions, less than $0.1 million relates to awards with performance conditions, and the remaining $49.5 million related to restricted stock awards or restricted stock units with service conditions only. |
401 (K) PLAN
401 (K) PLAN | 12 Months Ended |
Dec. 31, 2019 | |
Compensation And Retirement Disclosure [Abstract] | |
401 (K) PLAN | 16. 401 (K) PLAN The Company sponsors a 401(k) Plan (“the Plan”) in the U.S. and other retirement plans in the rest of the world, all of which are defined contribution plans. The Plan is available to all employees who are age 21 or older. Participants may make voluntary contributions and the Company makes matching contributions according to the Plan’s matching formula. Matching contributions fully Expense related to the Plan totaled $3.4 million, $2.1 million and $1.4 million for the years ended December 31, 2019, 2018 and 2017, respectively. |
OTHER (LOSS) INCOME
OTHER (LOSS) INCOME | 12 Months Ended |
Dec. 31, 2019 | |
Other Income And Expenses [Abstract] | |
OTHER (LOSS) INCOME | 17. OTHER (LOSS) INCOME The following table summarizes other income and loss for the periods indicated: For the Year Ended December 31, 2019 2018 2017 (in thousands) Interest expense $ (30,669 ) $ (33,709 ) $ (5,801 ) Interest income 7,238 6,810 1,809 Amortization of investment discount 15,350 8,573 1,401 Other expense (236 ) (656 ) 601 Gain from sale of Priority Review Voucher — — 125,000 Total other (loss) income $ (8,317 ) $ (18,982 ) $ 123,010 |
INCOME TAXES
INCOME TAXES | 12 Months Ended |
Dec. 31, 2019 | |
Income Tax Disclosure [Abstract] | |
INCOME TAXES | 18. INCOME TAXES The following table summarizes the loss before the provision for income taxes by jurisdiction for the periods indicated: For the Year Ended December 31, 2019 2018 2017 (in thousands) Domestic $ (489,747 ) $ (309,294 ) $ (45,686 ) Foreign (224,133 ) (53,316 ) (2,942 ) Total $ (713,880 ) $ (362,610 ) $ (48,628 ) The following table summarizes provision for income taxes in the accompanying consolidated financial stat ements for the periods indicated : For the Year Ended December 31, 2019 2018 2017 (in thousands) Current provision: Federal $ — $ (110 ) $ 204 State 521 (653 ) 1,856 Foreign 1,050 311 — Total current provision 1,571 (452 ) 2,060 Deferred benefit: Federal (15 ) — — State (5 ) — — Foreign (356 ) (240 ) — Total deferred benefit (376 ) (240 ) — Total current provision $ 1,195 $ (692 ) $ 2,060 The following table summarizes the reconciliation between the Company’s effective tax rate and the income tax rate for each of the periods indicated: For the Year Ended December 31, 2019 2018 2017 Federal income tax rate 21.0 % 21.0 % 34.0 % State taxes 6.3 12.3 (27.7 ) Research and development and other tax credits 3.3 3.1 8.5 Valuation allowance (16.8 ) (45.5 ) (93.2 ) Permanent differences 1.8 6.9 6.4 Sarepta International C.V. return to provision — (0.1 ) 62.1 Impact of tax reform, net of valuation allowance — — 5.9 Basis difference in subsidiary (8.4 ) — — Foreign rate differential (7.4 ) (0.9 ) (0.6 ) Other — 3.4 0.4 Effective tax rate (0.2 ) % 0.2 % (4.2 ) % Permanent differences affecting the Company’s effective tax rate primarily include excess stock-based compensation tax deductions, net of non-deductible stock-based compensation and limitation on officer compensation deduction. In February 2019, the Company exercised its option to acquire Myonexus. Accumulated costs of $253.7 million, associated with the Myonexus acquisition, were expensed for U.S. GAAP purposes. Of the $253.7 million in accumulated costs, $85.0 million relates to up-front and milestone payments as a result of the execution of the Warrant Agreement in May 2018 as well as certain development milestones being achieved or becoming probable of being achieved and $168.7 million relates to the exercise of the exclusive option to acquire Myonexus in February 2019. For U.S. income tax purposes, these costs are considered to be an outside investment in the subsidiary and are not currently deductible for tax purposes. The permanent difference related to this acquisition is separately stated in the rate reconciliation above. In December 2012, the Company licensed certain intellectual property of Sarepta Therapeutics, Inc. to its wholly owned Netherlands subsidiary, Sarepta International C.V. The parties also entered into a contract research agreement under which Sarepta Therapeutics, Inc. performs research services for Sarepta International C.V. In January 2016, Sarepta Therapeutics, Inc. entered into a manufacturing and distribution agreement as well as service agreement with Sarepta International C.V. In conjunction with its recent filings, it was determined that beginning in 2016, Sarepta International C.V. is effectively connected with the conduct of a trade or business by the entity in the U.S. and, accordingly, the 2016, 2017 and 2018 losses are subject to U.S. income taxes. In May 2018, Sarepta International C.V. merged into another wholly owned U.S. subsidiary of Sarepta Therapeutics, Inc. The following table summarizes the analysis of the deferred tax assets and liabilities for each of the periods indicated: As of December 31, 2019 2018 (in thousands) Deferred tax assets: Net operating loss carryforwards $ 304,033 $ 212,342 Difference in depreciation and amortization 40,095 55,162 Research and development tax credits 103,806 67,309 Stock-based compensation 24,114 15,538 Lease liabilities 15,796 4,831 Deferred revenue 939 888 Capitalized inventory 18,255 22,943 Other 16,270 15,727 Total deferred tax assets 523,308 394,740 Deferred tax liabilities: Right of use asset (10,782 ) — Debt discount (23,099 ) (25,162 ) Total deferred tax liabilities (33,881 ) (25,162 ) Valuation allowance (488,829 ) (369,345 ) Net deferred tax assets $ 598 $ 233 The Company has evaluated the positive and negative evidence bearing upon the realizability of its U.S. net deferred tax assets, which are comprised principally of federal and state net operating loss carryforwards, research and development tax credit carryforwards, stock-based compensation expense, capitalized inventory, and intangibles. Under the applicable accounting standards, management has considered the Company’s history of losses and concluded that it is more likely than not that the Company will not recognize the benefits of net federal and state deferred tax assets. Accordingly, a full valuation allowance of the U.S. net deferred tax asset had been established at December 31, 2019 and 2018. The net change in the valuation allowance for deferred tax assets was an increase of $119.5 million and $164.8 million for the years ended December 31, 2019 and 2018, respectively. This increase for the year ended December 31, 2019 was primarily due to the generation of federal and state net operating losses and income tax credits. The Company generated foreign deferred tax assets mainly consisting of net operating loss carryforwards, stock-based compensation and unrealized gain/losses. Based upon the income projections in the majority of the foreign jurisdictions, the Company believes it will realize the benefit of its future deductible differences in these jurisdictions. As such, the Company has not recorded a valuation allowance against these foreign jurisdictions. Brazil, the Netherlands, Czech Republic, and Spain have generated deferred tax assets, which consist of net operating loss carryforwards and stock-based compensation expense. The Company has concluded that it is more likely than not that we will not recognize the future benefits of the deferred tax assets, and accordingly, a full valuation allowance has been recorded against these foreign deferred tax assets. In 2019, the Company undertook an internal restructuring involving its subsidiary in Switzerland. The restructuring resulted in the utilization of all of its net operating loss carryforwards and the release of its previously established valuation allowance of $7.9 million. As of December 31, 2019, the Company had federal and state net operating loss carryforwards of $1,166.2 million and $859.0 million, respectively, available to reduce future taxable income. The federal and state net operating loss carry forwards of $579.9 million and $807.7 million will expire at various dates between 2020 and 2039. The federal and state net operating loss carry forwards of $586.3 million and $51.3 million, respectively, can be carried forward indefinitely. Utilization of these net operating losses could be limited under Section 382 of the Internal Revenue Code and similar state laws based on ownership changes and the value of the Company’s stock. Additionally, the Company has $74.7 million and $34.1 million of federal and state research and development credits, respectively, available to offset future taxable income. These federal and state research and development credits begin to expire between 2020 and 2039 and between 2020 and 2034, respectively. The Company also has foreign net operating loss carryforwards of $8.0 million, mainly derived from the net operating loss generated by its subsidiary in Brazil, which may be carried forward indefinitely. The Company or one of its subsidiaries files income tax returns in the U.S., and various state and foreign jurisdictions. The federal, state and foreign income tax returns are generally subject to tax examinations for the tax years ended December 31, 2016 through December 31, 2018. To the extent the Company has tax attribute carryforwards, the tax years in which the attribute was generated may still be adjusted upon examination by the Internal Revenue Service, state or foreign tax authorities to the extent utilized in a future period. The follow table summarizes the reconciliation of the beginning and ending amount of total unrecognized tax benefits for each of the periods indicated: For the Year Ended December 31, 2019 2018 2017 (in thousands) Balance at beginning of the period $ 37,544 $ 5,134 $ 4,644 Increase related to current year tax positions 4,275 2,164 735 Increase related to prior year tax positions 109 30,246 — Decrease related to prior year tax positions (175 ) — (245 ) Balance at end of the period $ 41,753 $ 37,544 $ 5,134 The balance of total unrecognized tax benefits at December 31, 2019, if recognized, would not affect the effective tax rate on income from continuing operations, due to a full valuation allowance against the Company’s U.S. deferred tax assets. The Company does not expect that the amount of unrecognized tax benefits to change significantly in the next twelve months. The Company’s policy is to recognize interest and/or penalties related to income tax matters in income tax expense. It had no accrual for interest or penalties on its balance sheet at December 31, 2019 or 2018. No interest and/or penalties were recognized in 2018 or 2017. |
LEASES
LEASES | 12 Months Ended |
Dec. 31, 2019 | |
Leases [Abstract] | |
LEASES | 19. LEASES The adoption of ASC 842 resulted in the recognition of operating lease liabilities and ROU assets of $60.1 million and $42.5 million, respectively, on the Company’s balance sheet relating to its leases for its corporate headquarters and its office and lab space on the January 1, 2019 transition date. Further, the Company reclassified upon adoption $18.0 million of deferred rent which reduced the ROU assets recognized on the balance sheet, in accordance with the transition guidance. As of December 31, 2019, operating lease assets were $37.9 million and operating lease liabilities were $55.6 million. Amounts related to financing leases were immaterial. The following table contains a summary of the lease costs recognized under ASC 842 and other information pertaining to the Company’s operating leases for the year ended December 31, 2019: For the Year Ended December 31, 2019 (in thousands) Lease cost Operating lease cost $ 10,335 Variable lease cost 3,967 Total lease cost $ 14,302 Other information Operating lease payments 10,416 Operating lease liabilities arising from obtaining ROU assets — Weighted average remaining lease term 5.5 Weighted average discount rate 7.50 % The following table summarizes maturities of lease liabilities and the reconciliation of lease liabilities as of December 31, 2019: As of December 31, 2019 (in thousands) 2020 $ 11,718 2021 12,891 2022 11,080 2023 11,230 2024 11,471 Thereafter 9,654 Total minimum lease payments 68,044 Less: imputed interest (12,478 ) Total operating lease liabilities $ 55,566 Included in the condensed consolidated balance sheet: Current portion of lease liabilities within other current liabilities $ 7,846 Lease liabilities 47,720 Total operating lease liabilities $ 55,566 For comparable purposes, aggregate future minimum non-cancellable commitments under leases as of December 31, 2018, are as follows: As of December 31, 2018 (in thousands) 2020 $ 11,395 2021 12,558 2022 10,757 2023 10,898 2024 11,128 Thereafter 9,396 Total minimum lease payments $ 66,132 Excluded from the table above are obligations under manufacturing agreements with Brammer Bio MA, LLC (“Brammer”) and Paragon Bioservices, Inc. (“Paragon”). The Company has determined that both agreements contain an embedded lease. However, both leases have not yet commenced as of December 31, 2019, and as such, right of use assets and lease liabilities have not yet been recognized on the Company’s consolidated balance sheets. Refer to Note 21, Commitments and Contingencies |
NET LOSS PER SHARE
NET LOSS PER SHARE | 12 Months Ended |
Dec. 31, 2019 | |
Earnings Per Share [Abstract] | |
NET LOSS PER SHARE | 20. NET LOSS PER SHARE Basic net loss per share is computed by dividing net loss by the weighted-average number of shares of common stock outstanding. Diluted net loss per share is computed by dividing net loss by the weighted-average number of shares of common stock and dilutive common stock equivalents outstanding. Given that the Company recorded a net loss for each of the periods presented, there is no difference between basic and diluted net loss per share since the effect of common stock equivalents would be anti-dilutive and are, therefore, excluded from the diluted net loss per share calculation. For the Year Ended December 31, 2019 2018 2017 (in thousands, except per share amounts) Net loss $ (715,075 ) $ (361,918 ) $ (50,688 ) Weighted-average common shares outstanding - basic 73,615 66,250 58,818 Effect of dilutive securities* — — — Weighted-average common shares outstanding - diluted 73,615 66,250 58,818 Net loss per share — basic and diluted $ (9.71 ) $ (5.46 ) $ (0.86 ) * For the years ended December 31, 2019, 2018 and 2017, stock options, RSAs, RSUs, SARs and ESPP to purchase approximately 9.1 million, 9.1 million and 9.4 million shares of common stock, respectively, were excluded from the net loss per share calculation as their effect would have been anti-dilutive. The Company accounts for the effect of the 2024 Notes on diluted net earnings per share using the if-converted method as they may be settled in cash or shares at the Company’s option. While the closing price on December 31, 2019 exceeded the conversion price of $73.42, the potential shares issuable under the 2024 Notes were excluded from the calculation of diluted loss per share as they were anti-dilutive using the if-converted method. In the period of conversion, the 2024 Notes will have no impact on diluted net loss if they are settled in cash and will have an impact on diluted earnings per share if the 2024 Notes are settled in shares upon conversion and when the Company is in an income position. |
COMMITMENTS AND CONTINGENCIES
COMMITMENTS AND CONTINGENCIES | 12 Months Ended |
Dec. 31, 2019 | |
Commitments And Contingencies Disclosure [Abstract] | |
COMMITMENTS AND CONTINGENCIES | 21. COMMITMENTS AND CONTINGENCIES Manufacturing Obligations The Company has entered into long-term contractual arrangements from time to time for the provision of goods and services. Brammer Bio MA, LLC The Company entered into a Development, Commercial Manufacturing and Supply Agreement (the “Brammer Manufacturing Agreement”) and, subsequently, entered into the first amendment (the “Amendment”) to the Brammer Manufacturing Agreement with Brammer in June 2018 and May 2019, respectively (collectively, “Brammer Supply Agreements”). Pursuant to the terms of the Brammer Supply Agreements, Brammer agreed to provide the Company with access to clinical and commercial manufacturing capacity for its gene therapy programs. Under the Brammer Manufacturing Agreement, the Company will purchase product in batches from Brammer, subject to minimum and maximum annual purchase requirements. Further, the Company: (i) was required to make a $20.0 million advance payment to Brammer upon execution of the agreement in June 2018, (ii) was required to make two non-refundable payments of $5.0 million each to Brammer in the third and fourth quarter of 2018 to be used in the specification, selection, and procurement of the related process equipment to be utilized under the agreement, and (iii) was required to make a $10.0 million quarterly capacity access fee payment to Brammer throughout the term of the agreement. As a result of the Amendment: (i) the Company now has access to substantially all of the related facility’s capacity, subject to certain minimum and maximum volume limitations, (ii) the Company was required to make a $6.0 million advance payment to Brammer upon execution of the Amendment, and (iii) the quarterly capacity access fee payments due to Brammer throughout the term of the agreement increased from $10.0 million to $13.3 million, starting January 1, 2020. However, through December 31, 2019, a reduced quarterly capacity access fee was in effect as Brammer worked towards achieving full capacity at its facility. In addition, the application of the advance payments will reduce the quarterly capacity access fees paid through 2021. The term of the Brammer Supply Agreements will continue for a period of six years following the first regulatory approval of a product manufactured under the agreements. The term will automatically renew for successive two years unless the Company notifies Brammer of its intention not to renew (no less than twenty-four months prior to the expiration of the term). The Company also has the ability to terminate the agreement prior to expiration but would be required to continue remitting capacity access fees to Brammer for up to eight additional quarters. Upon execution of the Amendment, the Company determined that the Brammer Supply Agreements contain an embedded lease because the Company now has the right to direct the use of the facility and related equipment therein. Further, the Company determined that it did not control the facility or related equipment during construction and, thus, the lease did not fall in the scope of “build-to-suit” accounting. The lease has not commenced as of December 31, 2019 because of the clean room suites at the Brammer facility are not yet available for use by the Company. Accordingly, total cumulative payments made to Brammer of $75.5 million have been recorded as an other non-current asset in the accompanying consolidated balance sheets Rent expense recognized prior to regulatory approval of the related product will be classified to research and development expense. Upon regulatory approval, rent expense will be classified to cost of inventory with the recognition in cost of sales as the sales of product occur. Paragon Bioservices, Inc. The Company entered into a manufacturing collaboration agreement (the “Paragon Collaboration Agreement”) and, subsequently, entered into a manufacturing and supply agreement (the “Paragon Supply Agreement”) with Paragon in October 2018 and February 2019, respectively (collectively, the “Paragon Agreements”). Pursuant to the terms of the Paragon Agreements, Paragon agreed to provide the Company with two dedicated clean room suites and an option to reserve two additional clean room suites for its gene therapy programs. In September 2019, the Company exercised the option to gain access to the additional clean room suites. The Paragon Agreements will expire on December 31, 2024. The Company has the ability to terminate the Paragon Agreements prior to expiration, subject to potential additional financial consideration. Under the Paragon Agreements, the Company will purchase product in batches from Paragon subject to minimum annual purchase requirements during two periods: the pre-launch period and the post-launch period. During the pre-launch period, the Company is obligated to purchase a minimum amount of $4.0 million of services per quarter per clean room. During the post-launch period, on an annual basis, the Company is obligated to purchase a minimum number of batches per clean room. Further, the Company is required to pay Paragon: (i) use fees of $1.0 million per year per clean room suite after the clean rooms are fully qualified and validated to manufacture the Company’s materials, and (ii) clean room reservation fees totaling $48.0 million. Additional use fees and reservation fees are required if the Company has equipment needs beyond the basic equipment package included in the initial clean room suites. In addition, Paragon will provide the Company with a credit of up to 100% of the clean room use fee if certain clean room capacity utilization thresholds are met. The Company has concluded that the Paragon Agreements contain an embedded lease as the Company has the right to direct the use of the facility and related equipment therein. The Company also determined that it did not control the facility or related equipment during construction and, thus, the lease did not fall in the scope of “build-to-suit” accounting. The lease has not commenced as of December 31, 2019 because the clean room suites at the Paragon facility are not yet available for use by the Company. Accordingly, cumulative payments totaling $40.1 million made to Paragon have been recorded as an other non-current asset in the accompanying consolidated balance sheets and will be considered in the initial measurement of the cost of the right-of-use asset at the lease commencement date. This amount, along with any additional payments made prior to the lease commencement date, will be amortized on a straight-line basis as rent expense over the term of the embedded lease, beginning on the lease commencement date, currently anticipated to occur in the first quarter of 2020. Use fees associated with the clean room suites are considered contingent rental payments and will be charged to rent expense when (and if) incurred. Rent expense recognized prior to regulatory approval of the related product will be classified to research and development expense. Upon regulatory approval, rent expense will be classified to cost of inventory with the recognition in cost of sales as the sales of product occur. Aldevron, LLC In December 2018, the Company entered into a Clinical and Commercial Supply Agreement (the “CCSA”) with Aldevron LLC (“Aldevron”) for the supply of plasmid DNA to fulfill its needs for gene therapy clinical trials and commercial supply. Pursuant to the terms of the CCSA, Aldevron agreed to reserve a certain number of manufacturing slots (“Reserved Slots”) on a quarterly basis. The initial term of the CCSA expired on December 31, 2019. The Company exercised the option to extend the CCSA to December 31, 2020 (the “2020 Option”) and has another option to extend the term of the CCSA for an additional year to December 31, 2021 (the “2021 Renewal Right”). The Company may be required to make an additional $20.0 million in prepayments associated with the CCSA should the Company exercise the 2021 Renewal Right. The prepayments will be credited back to Sarepta, until exhausted, for each batch of product delivered by Aldevron, in an amount equal to 50% of the batch invoice amount. The Company has determined that the CCSA does not contain an embedded lease because it does not convey the right to control the use of Aldevron’s facility or related equipment therein. As of December 31, 2019, the Company recorded $22.8 million in other current assets in the accompanying balance sheets related to the prepayments made to Aldevron under the CCSA. The gross cost of batches purchased from Aldevron since inception of the agreement have been classified as research and development expense. In the event the Company does not expect services under the CCSA to be rendered to fully exhaust any prepayments made to Aldevron, the applicable balance will be charged to expense at the time this determination is made. The following table presents non-cancelable contractual obligations arising from long-term contractual arrangements: As of December 31, 2019 (in thousands) 2020 $ 378,744 2021 183,661 2022 64,653 2023 58,319 2024 58,309 Thereafter 149,350 Total manufacturing commitments $ 893,036 Additionally, should the Company obtain regulatory approval for any drug product candidate produced as a part of the Company’s manufacturing obligations above, additional minimum batch requirements with the respective manufacturing parties would be required. Other Funding Commitments The Company has several on-going clinical trials in various clinical trial stages. Its most significant clinical trial expenditures are to contract research organizations (“CROs”). The CRO contracts are generally cancellable at the Company’s option. As of December 31, 2019, the Company has approximately $91.4 million in cancellable future commitments based on existing CRO contracts. For the years ended December 31, 2019, 2018 and 2017, the Company recognized approximately $31.6 million, $19.6 million and $13.9 million, respectively, for expenditures incurred by CROs. Litigation In the normal course of business, the Company may from time to time be named as a party to various legal claims, actions and complaints, including matters involving securities, employment, intellectual property, effects from the use of therapeutics utilizing its technology, or others. For example, on August 30, 2019, Plaintiff Andrew Salinger filed a putative class action complaint against the Company and two of its current officers, Douglas S. Ingram and Sandesh Mahatme (collectively, the “Defendants”), in the United States District Court for the Southern District of New York. The complaint alleges that the Defendants violated Section 10(b) of the Securities Exchange Act of 1934, as amended (“Exchange Act”), and Rule 10b-5 promulgated thereunder, as well as Section 20(a) of the Exchange Act, in connection with the Company’s disclosures related to golodirsen. The proposed class consists of all persons or entities who acquired Company securities between September 6, 2017 and August 19, 2019. On December 17, 2019, the district court appointed Bernard Portnoy as lead plaintiff, and set a briefing schedule requiring the amended complaint to be filed on February 18, 2020 and requiring Defendants to answer or otherwise respond to the amended complaint on April 17, 2020. Defendants’ motion to transfer the case to the United States District Court for the District of Massachusetts is pending. On February 14, 2020, the lead plaintiff filed a Notice of Voluntary Dismissal of his claims against all Defendants and the clerk referred the Notice to the court for review and approval. The court has taken no further action. The Company is unable to provide an estimate of possible loss or range of possible loss. On January 7, 2020, Plaintiff Al Lutzker filed a stockholder derivative complaint, purportedly on behalf of the Company, against two of the Company’s current officers, Douglas S. Ingram and Sandesh Mahatme, and six current members of Company’s Board of Directors, M. Kathleen Behrens, Richard J. Barry, Michael W. Bonney, Mary Ann Gray, Claude Nicaise, and Hans Wigzell (collectively, the “Defendants”), in the United States District Court for the District of Delaware. The complaint asserts claims for breach of fiduciary duty, insider selling, unjust enrichment, waste of corporate assets, and violations of Section 14(a) of the Securities Exchange Act of 1934, and Rule 14a-9 promulgated thereunder, in connection with the Company’s disclosures related to golodirsen. The Company is unable to provide an estimate of possible loss or range of possible loss. |
SUBSEQUENT EVENT
SUBSEQUENT EVENT | 12 Months Ended |
Dec. 31, 2019 | |
Subsequent Events [Abstract] | |
SUBSEQUENT EVENT | 22. SUBSEQUENT EVENT On February 14, 2020, the Company entered into an agreement to sell the rare pediatric disease PRV it received from the FDA in connection with the approval of VYONDYS 53 for consideration of $111.0 million. The closing of the transaction is subject to the expiration or termination of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976 and other customary conditions. When the transaction closes, the net proceeds The Company has evaluated subsequent events from the date of the consolidated balance sheet through the date the consolidated financial statements were issued. |
FINANCIAL INFORMATION BY QUARTE
FINANCIAL INFORMATION BY QUARTER (UNAUDITED) | 12 Months Ended |
Dec. 31, 2019 | |
Quarterly Financial Information Disclosure [Abstract] | |
FINANCIAL INFORMATION BY QUARTER (UNAUDITED) | 23. FINANCIAL INFORMATION BY QUARTER (UNAUDITED) 2019 for Quarter Ended December 31 September 30 June 30 March 31 (in thousands) Revenues: Product, net $ 100,113 $ 99,041 $ 94,668 $ 87,011 Total revenues 100,113 99,041 94,668 87,011 Cost and expenses: Cost of sales (excluding amortization of in-licensed rights) 15,567 13,037 15,919 12,063 Research and development 223,141 133,949 113,266 90,553 Selling, general and administrative 81,424 75,429 67,393 60,566 Acquired in-process research and development — — 173,240 — Settlement and license charges 10,000 — — — Amortization of in-licensed rights 200 216 217 216 Total cost and operating expenses 330,332 222,631 370,035 163,398 Operating loss (230,219 ) (123,590 ) (275,367 ) (76,387 ) Other loss: Other expense, net (4,773 ) (2,510 ) (862 ) (172 ) Other loss (4,773 ) (2,510 ) (862 ) (172 ) Loss before income tax expense (234,992 ) (126,100 ) (276,229 ) (76,559 ) Income tax expense 711 226 174 84 Net loss $ (235,703 ) $ (126,326 ) $ (276,403 ) $ (76,643 ) Net loss per share - basic and diluted $ (3.16 ) $ (1.70 ) $ (3.74 ) $ (1.07 ) Weighted average number of shares of common stock used in computing basic and diluted net loss per share 74,557 74,177 73,958 71,731 2018 for Quarter Ended December 31 September 30 June 30 March 31 (in thousands) Revenues: Product, net $ 84,415 $ 78,486 $ 73,529 $ 64,604 Total revenues 84,415 78,486 73,529 64,604 Cost and expenses: Cost of sales (excluding amortization of in-licensed rights) 13,135 8,741 6,735 5,582 Research and development 146,207 86,584 122,848 46,204 Selling, general and administrative 64,220 53,044 47,156 43,341 Amortization of in-licensed rights 216 216 217 216 Total cost and operating expenses 223,778 148,585 176,956 95,343 Operating loss (139,363 ) (70,099 ) (103,427 ) (30,739 ) Other loss: Interest expense and other, net (2,311 ) (6,968 ) (5,218 ) (4,485 ) Total other loss (2,311 ) (6,968 ) (5,218 ) (4,485 ) Loss before income (benefit) tax expense (141,674 ) (77,067 ) (108,645 ) (35,224 ) Income tax (benefit) expense (779 ) (674 ) 622 139 Net loss $ (140,895 ) $ (76,393 ) $ (109,267 ) $ (35,363 ) Net loss per share - basic and diluted $ (2.05 ) $ (1.15 ) $ (1.67 ) $ (0.55 ) Weighted average number of shares of common stock used in computing basic and diluted net loss per share 68,653 66,209 65,484 64,631 |
SUMMARY OF SIGNIFICANT ACCOUN_2
SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES AND RECENT ACCOUNTING PRONOUNCEMENTS (Policies) | 12 Months Ended |
Dec. 31, 2019 | |
Accounting Policies [Abstract] | |
Basis of Presentation | Basis of Presentation The accompanying consolidated financial statements, which have been prepared in accordance with accounting principles generally accepted in the U.S. (“U.S. GAAP”), reflect the accounts of Sarepta Therapeutics, Inc. and its wholly-owned subsidiaries. All intercompany transactions between and among its consolidated subsidiaries have been eliminated. Management has determined that the Company operates in one segment: helping patients through the discovery and development of unique RNA-targeted therapeutics, gene therapy and other genetic therapeutic modalities for the treatment of rare diseases |
Estimates and Uncertainties | Estimates and Uncertainties The preparation of the consolidated financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets, liabilities, equity, revenue, expenses and the disclosure of contingent assets and liabilities. Actual results could differ from those estimates. |
Fair Value Measurements | Fair Value Measurements The Company has certain financial assets that are recorded at fair value which have been classified as Level 1, 2 or 3 within the fair value hierarchy as described in the accounting standards for fair value measurements: • Level 1—quoted prices for identical instruments in active markets; • Level 2—quoted prices for similar instruments in active markets, quoted prices for identical or similar instruments in markets that are not active, and model-derived valuations in which all significant inputs and significant value drivers are observable in active markets; and • Level 3—valuations derived from valuation techniques in which one or more significant value drivers are unobservable. The fair value of the majority of the Company’s financial assets is categorized as Level 1 within the fair value hierarchy. These assets include money market funds, publicly traded debt, and equity securities. For additional information related to fair value measurements, please read Note 5, Fair Value Measurements |
Cash and Cash Equivalents | Cash Equivalents Only investments that are highly liquid and readily convertible to cash and have original maturities of three months or less are considered cash equivalents. |
Investments | Investments Available-For-Sale Debt Securities Available-for-sale debt securities are recorded at fair value and unrealized gains and losses are included in accumulated other comprehensive income (loss) in stockholder’s equity. Realized gains and losses are reported in other expense, net, on a specific identification basis. Equity Investments The Company’s equity investments include its investments in a publicly traded biotechnology company and a privately held biotechnology company and are included in other non-current assets in the Company’s consolidated balance sheets. The equity investment in the publicly traded biotechnology company has a readily determinable fair value and is carried at fair value with changes in value recorded as a gain or loss in the Company’s consolidated statements of operations and comprehensive loss. The equity investment in the privately held biotechnology company does not have readily determinable fair value and is measured at cost less any impairment, plus or minus changes resulting from observable price changes for the identical or a similar investment of the same issuer, which is recorded as a gain or loss on the Company’s consolidated statements of operations and comprehensive loss. |
Accounts Receivable | Accounts Receivable The Company’s accounts receivable primarily arise from product sales. They are generally stated at the invoiced amount and do not bear interest. Revenues from product sales are recorded at the net sales price (transaction price), which includes estimates of variable consideration for which reserves are established and which result from Medicaid rebates, governmental chargebacks including Public Health Services (“PHS”) chargebacks, prompt pay discounts, co-pay assistance and distribution fees. These reserves are based on the amounts earned or to be claimed on the related sales and are classified as reductions of accounts receivable (if no payments are required of the Company) for PHS chargebacks, prompt pay discounts and certain distribution fees, or a current liability (if a payment is required of us), for Medicaid rebates, co-pay assistance and certain distribution fees. The accounts receivable from product sales represents receivables due from the Company’s specialty distributor and specialty pharmacies in the U.S. as well as certain distributors in the EU, Brazil, Israel and the Middle East. The Company monitors the financial performance and creditworthiness of its customers so that it can properly assess and respond to changes in the customers’ credit profiles. The Company provides reserves against trade receivables for estimated losses that may result from a customer’s inability to pay. Amounts determined to be uncollectible are written-off against the established reserve. As of December 31, 2019, the credit profiles for the Company’s customers are deemed to be in good standing and an allowance for doubtful accounts receivable is not considered necessary. |
Concentration of Credit Risk | Concentration of Credit Risk Financial instruments which potentially subject the Company to concentrations of credit risk consist of accounts receivable from customers and cash, cash equivalent and investments held at financial institutions. For the year ended December 31, 2019 42 47 51 December 31, 2019 As of December 31, 2019 |
Inventories | Inventories Inventories are stated at the lower of cost and net realizable value with cost determined on a first-in, first-out basis. The Company capitalizes inventory costs associated with products following regulatory approval when future commercialization is considered probable and the future economic benefit is expected to be realized. EXONDYS 51 and VYONDYS 53 inventory that may be used in clinical development programs is charged to research and development expense when the product enters the research and development process and no longer can be used for commercial purposes. The Company periodically reviews its inventories for excess amounts or obsolescence and writes down obsolete or otherwise unmarketable inventory to its estimated net realizable value. Additionally, though the Company’s product is subject to strict quality control and monitoring which it performs throughout the manufacturing processes, certain batches or units of product may not meet quality specifications resulting in a charge to cost of sales. For products which are under development and have not yet been approved by regulatory authorities, purchased drug product is charged to research and development expense upon delivery. Delivery occurs when the inventory passes quality inspection and ownership transfers to the Company. Nonrefundable advance payments for research and development activities, including production of purchased drug product, are deferred and capitalized until the goods are delivered. If the Company does not expect the goods to be delivered or services to be rendered, the advanced payment capitalized will be charged to expense. |
Property and Equipment | Property and Equipment Property and equipment are initially recorded at cost, including the acquisition cost and all costs necessarily incurred to bring the asset to the location and working condition necessary for its intended use. The cost of normal, recurring or periodic repairs and maintenance activities related to property and equipment are expensed as incurred. The cost for planned major maintenance activities, including the related acquisition or construction of assets, is capitalized if the repair will result in future economic benefits. Interest costs incurred during the construction period of major capital projects are capitalized until the asset is ready for its intended use, at which point the interest costs are amortized as depreciation expense over the life of the underlying asset. The Company generally depreciates the cost of its property and equipment using the straight-line method over the estimated useful lives of the respective assets, which are summarized as follows: Asset Category Useful lives Lab equipment 5 years Office equipment 5 years Software and computer equipment 3 - 5 years Furniture and fixtures 7 years Leasehold improvements Lesser of the useful life or the term of the respective lease Land improvements 25 years Land Not depreciated Building and improvements 30 years Construction in Progress Not depreciated until put into service |
Intangible assets | Intangible assets The Company’s intangible assets consist of in-licensed rights, patent costs, and software licenses, which are stated in the Company’s consolidated balance sheets net of accumulated amortization and impairments, if applicable. The in-licensed rights relate to agreements with BioMarin Pharmaceutical, Inc. (“BioMarin”) and the University of Western Australia (“UWA”). The in-licensed rights are being amortized on a straight-line basis over the remaining life of the related patents because the life of the related patents reflects the expected time period that the Company will benefit from the in-licensed rights. Patent costs consist primarily of external legal costs, filing fees incurred to file patent applications and renewal fees on proprietary technology developed or licensed by the Company. Patent costs associated with applying for a patent, being issued a patent and annual renewal fees are capitalized. Costs to defend a patent and costs to invalidate a competitor’s patent or patent application are expensed as incurred. Patent costs are amortized on a straight-line basis over the shorter of the estimated economic lives or the initial term of the patents, which is generally 20 years. |
Impairment of Long-Lived Assets | Impairment of Long-Lived Assets Long-lived assets held and used by the Company, intangible assets with definite lives and equity investments without a readily determinable fair value are reviewed for impairment whenever events or circumstances indicate that the carrying amount of assets may not be recoverable. The Company evaluates recoverability of assets to be held and used by comparing the carrying amount of an asset to future net undiscounted cash flows to be generated by the asset. If the asset is considered to be impaired, the impairment to be recognized is measured as the amount by which the carrying amount of the assets exceeds the fair value of the assets. Such reviews assess the fair value of the assets based upon estimates of future cash flows that the assets are expected to generate. |
Convertible Debt | Convertible Debt The Company separately accounts for the liability and equity components of convertible debt instruments that can be settled in cash by allocating the proceeds from issuance between the liability component and the embedded conversion option. The value of the equity component is calculated by first measuring the fair value of the liability component, using the interest rate of a similar liability that does not have a conversion feature, as of the issuance date. The difference between the proceeds from the convertible debt issuance and the amount measured as the liability component is recorded as the equity component with a corresponding discount recorded on the debt. The Company recognizes the amortization of the resulting discount as interest expense using the effective interest method. Simultaneously, the Company bought capped call options from certain counterparties to minimize the impact of potential dilution upon conversion. The premium for the capped call options was recorded as additional paid-in capital. For additional information related to the convertible debt transactions, please read Note 13, Indebtedness |
Revenue Recognition | Revenue Recognition The Company recognizes revenue when a customer obtains control of promised goods or services, in an amount that reflects the consideration which the Company expects to receive in exchange for the goods or services provided. To determine revenue recognition for arrangements within the scope of ASC Topic 606, “Revenue from Contracts with Customers” Product revenues The Company distributes its products principally through its customers. The customers subsequently resell the product to patients and health care providers. The Company provides no right of return to the customers except in cases of shipping error or product defect. Product revenues are recognized when the customers take control of the product, which typically occurs upon delivery to the customers. For the years ended December 31, 2019, 2018 2017 Variable Consideration Product revenues are recorded at the net sales price (transaction price) which includes estimated reserves for variable consideration, such as Medicaid rebates, governmental chargebacks, including Public Health Service (“PHS”) chargebacks, prompt payment discounts, co-pay assistance and distribution fees. These reserves reflect the Company’s best estimates of the amount of consideration to which it is entitled based on the terms of the contracts. Additional details relating to variable consideration follows: • Medicaid rebates relate to the Company’s estimated obligations to states under established reimbursement arrangements. Medicaid rebate reserves are recorded in the same period the related revenue is recognized, resulting in a reduction of product revenue and the establishment of a liability which is included in accrued expenses. • Governmental chargebacks, including PHS chargebacks, relate to the Company’s estimated obligations resulting from contractual commitments to sell products to qualified healthcare providers at prices lower than the list prices that the Company charges to wholesalers. The wholesaler charges the Company for the difference between what the wholesaler pays for the products and the ultimate selling price to the qualified healthcare providers. Chargeback reserves are recorded in the same period the related revenue is recognized, resulting in a reduction of product revenue and accounts receivable. Chargeback amounts are generally determined at the time of resale to the qualified healthcare provider from the wholesaler, and the Company generally issues credits for such amounts within a few weeks of receiving notification of resale from the wholesaler. • Prompt payment discounts relate to the Company’s estimated obligations for credits to be granted to specialty pharmacies for remitting payment on their purchases within established incentive periods. Reserves for prompt payment discounts are recorded in the same period the related revenue is recognized, resulting in a reduction of product revenue and accounts receivable. • Co-pay assistance relates to financial assistance provided to qualified patients, whereby the Company may assist them with prescription drug co-payments required by the patient’s insurance provider. Reserves for co-pay assistance are recorded in the same period the related revenue is recognized, resulting in a reduction of product revenue and the establishment of a liability which is included in accrued expenses. • Distribution fees relate to fees paid to customers in the distribution channel that provide the Company with inventory management, data and distribution services and are generally accounted for as a reduction of revenue. To the extent that the services received are distinct from the Company’s sale of products to the customers, these payments are accounted for as selling, general and administrative expenses. Reserves for distribution fees result in an increase in a liability if payments are required of the Company or a reduction of accounts receivable if no payments are required of the Company. |
Research and Development | Research and Development Research and development expenses consist of costs associated with research activities as well as those with the Company’s product development efforts, conducting pre-clinical trials, clinical trials and manufacturing activities. Research and development expenses are expensed as incurred. Up-front fees and milestones paid to third parties in connection with technologies which have not reached technological feasibility and do not have an alternative future use are expensed when incurred. Direct research and development expenses associated with the Company’s programs include clinical trial site costs, clinical manufacturing costs, costs incurred for consultants and other external services, such as data management and statistical analysis support and materials and supplies used in support of clinical programs. Indirect costs of the Company’s clinical programs include salaries, stock-based compensation and an allocation of its facility and technology costs. When third-party service providers’ billing terms do not coincide with the Company’s period-end, the Company is required to make estimates of its obligations to those third parties, including clinical trial and pharmaceutical development costs, contractual services costs and costs for supply of its drug candidates, incurred in a given accounting period and record accruals at the end of the period. The Company bases its estimates on its knowledge of the research and development programs, services performed for the period, past history for related activities and the expected duration of the third-party service contract, where applicable. |
Stock-Based Compensation | Stock-Based Compensation The Company’s stock-based compensation programs include stock options, restricted stock awards (“RSAs”), restricted stock units (“RSUs”), stock appreciation rights (“SARs”) and an employee stock purchase program (“ESPP”). The Company accounts for stock-based compensation using the fair value method. The fair values of stock options and SARs are estimated on the date of grant using the Black-Scholes-Merton option-pricing model. The fair values of RSAs and RSUs are based on the fair market value of the Company’s common stock on the date of the grant. The fair value of stock awards, with consideration given to estimated forfeitures, is recognized as stock-based compensation expense on a straight-line basis over the vesting period of the grants. For stock awards with performance-vesting conditions, the Company does not recognize compensation expense until it is probable that the performance-vesting condition will be achieved. Under the Company’s ESPP, participating employees purchase common stock through payroll deductions. The purchase price is equal to 85% of the lower of the closing price of the Company’s common stock on the first business day and the last business day of the relevant purchase period. The fair value of stock purchase rights are estimated using the Black-Scholes-Merton option-pricing model. The fair value of the look-back provision with the 15% discount is recognized on a graded-vesting basis as stock-based compensation expense over the purchase period. In addition to stock options with service and performance conditions, the Company also granted its CEO options with service and market conditions. A market condition relates to the achievement of a specified price of the Company’s common stock, a specified amount of intrinsic value indexed to the Company’s common stock or a specified price of the Company’s common stock in terms of other similar equity shares. The grant date fair value for the options with service and market conditions is determined by a lattice model with Monte Carlo simulations and is recognized as stock-based compensation expense on a straight-line basis over the service period. |
Income Taxes | Income Taxes The Company follows the asset and liability method of accounting for income taxes. Deferred tax assets and liabilities are recognized for the future tax consequences attributable to differences between the consolidated financial statement carrying amounts of existing assets and liabilities and their respective tax bases and operating loss and tax credit carryforwards. It is the intention of the Company to reinvest the earnings of its non-U.S. subsidiaries in those operations and not to repatriate the earnings to the U.S. Accordingly, the Company does not provide for deferred taxes on the excess of the financial reporting over the tax basis in its investments in foreign subsidiaries as they are considered permanent in duration. Deferred tax assets and liabilities are measured using enacted tax rates expected to apply to taxable income in the years in which those temporary differences are expected to be recovered and settled. A valuation allowance is recorded to reduce the net deferred tax asset to zero when it is more likely than not that the net deferred tax asset will not be realized. The Company recognizes the effect of income tax positions only if those positions are more likely than not of being sustained upon an examination. |
Leases | Leases Effective January 1, 2019, the Company adopted ASC Topic 842, “ Leases” Leases” As a result of adopting ASC 842, the Company recorded lease right-of-use (“ROU”) assets of $42.5 million and lease liabilities of $60.1 million as of January 1, 2019, primarily related to real estate leases, based on the present value of future lease payments on the date of adoption. The difference between the ROU assets and lease liabilities was due to previously recorded net deferred rent liabilities that were reclassified into the ROU assets. There was no impact to retained earnings upon adoption of ASC 842. Amounts related to finance leases were immaterial as of adoption . At the inception of an arrangement, the Company determines whether the arrangement is or contains a lease based on the unique facts and circumstances present in the arrangement. Leases with a term greater than 12 months are recognized on the balance sheet as ROU assets and short-term and long-term lease liabilities, as applicable. The Company has elected not to recognize on the balance sheet leases with terms of 12 months or less. The Company typically only includes an initial lease term in its assessment of a lease arrangement. Options to renew a lease are not included in the Company’s assessment unless there is reasonable certainty that the Company will renew. The Company monitors its plans to renew its leases no less than on a quarterly basis. In addition, the Company’s lease agreements generally do not contain any residual value guarantees or restrictive covenants. Operating lease liabilities and their corresponding ROU assets are recorded based on the present value of future lease payments over the expected remaining lease term at lease commencement. Lease cost for operating leases is recognized on a straight-line basis over the lease term as an operating expense. Certain adjustments to the ROU asset may be required for items such as lease prepayments or incentives received. The interest rate implicit in lease contracts is typically not readily determinable. As a result, the Company utilizes its incremental borrowing rate, which reflects the fixed rate at which the Company could borrow on a collateralized basis the amount of the lease payments in the same currency, for a similar term, in a similar economic environment. In transition to ASC 842, the Company utilized the remaining lease term of its leases in determining the appropriate incremental borrowing rate. In accordance with ASC 842, components of a lease should be bifurcated between lease components and non - lease components. The fixed and in-substance fixed contract consideration identified must then be allocated based on the respective relative fair values to the lease components and non - lease components. However, ASC 842 provides a practical expedient that allows an accounting policy election to not separate lease and non - lease components by class of underlying asset. In using this expedient, the lease component and non - lease component s are accounted for together as a single component. For real estate leases, the Company has elected to account for the lease and non - lease components together for existing classes of underlying assets and allocates the contract consideration to the lease component only . This practical expedient is not elected for manufacturing facilities and equipment embedded in product supply arrangements. |
Embedded Derivatives | Embedded Derivatives The Company evaluates certain of its financial and business development transactions to determine if embedded components of these contracts meet the definition of derivative under Topic ASC 815, “ Derivatives and Hedging |
Commitments and Contingencies | Commitments and Contingencies The Company records liabilities for legal and other contingencies when information available to the Company indicates that it is probable that a liability has been incurred and the amount of loss can be reasonably estimated. Legal costs in connection with legal and other contingencies are expensed as costs are incurred. |
Recent Accounting Pronouncements | Recent Accounting Pronouncements In December 2019, the Financial Accounting Standards Board (“FASB”) issued ASU 2019-12, “ Income Taxes (Topic 740): Simplifying the Accounting for Income Taxes”, In November 2018, the FASB issued ASU 2018-18, “ Collaborative Arrangements (Topic 808): Clarifying the Interaction between Topic 808 and Topic 606 In August 2018, the FASB issued ASU No. 2018-13, “Fair Value Measurement (Topic 820), Disclosure Framework – Changes to the Disclosure Requirements for Fair Value Measurement”. In August 2018, the FASB issued ASU No. 2018-15, “ Intangibles – Goodwill and Other – Internal-Use Software (Subtopic 350-40): Customer’s Accounting for Implementation Costs Incurred in a Cloud Computing Arrangement That is a Service Contract”. In June 2016 , the FASB issued ASU No. 2016-13, “ Financial Instruments - Credit Losses (Topic 326): Measurement of Credit Losses on Financial Instruments ” . This ASU requires that credit losses be reported using an expected losses model rather than the incurred losses model that is currently used, and establishes additional disclosures related to credit risks. For available-for-sale debt securities with unrealized losses, this standard now requires allowances to be recorded instead of reducing the amortized cost of the investment. ASU 2016-13 limits the amount of credit losses to be recognized for available-for-sale debt securities to the amount by which carrying value exceeds fair value and requires the reversal of previously recognized credit losses if fair value increases. ASU 2016-13 will be effective for fiscal years beginning after December 15, 2019 with early adoption permitted , and requires adoption using a modified retrospective approach, with certain exceptions. As of December 31, 2019, the Company has not elected to early adopt this guidance. Based on the composition of the Company’s investment portfolio as of December 3 1 , 2019, current market conditions and historical credit loss activity, the adoption of this standard is not expected to have a material impact on the Company’s consolidated financial statements. Additionally, for trade receivables, due to their short duration and the credit profile of the Company’s customers, the effect of transitioning from the incurred losses model to the expected losses model is not expected to be material. |
SUMMARY OF SIGNIFICANT ACCOUN_3
SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES AND RECENT ACCOUNTING PRONOUNCEMENTS (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Accounting Policies [Abstract] | |
Summary of Estimated Useful Lives of Property and Equipment | The Company generally depreciates the cost of its property and equipment using the straight-line method over the estimated useful lives of the respective assets, which are summarized as follows: Asset Category Useful lives Lab equipment 5 years Office equipment 5 years Software and computer equipment 3 - 5 years Furniture and fixtures 7 years Leasehold improvements Lesser of the useful life or the term of the respective lease Land improvements 25 years Land Not depreciated Building and improvements 30 years Construction in Progress Not depreciated until put into service |
LICENSE AND COLLABORATION AGR_2
LICENSE AND COLLABORATION AGREEMENTS (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Organization Consolidation And Presentation Of Financial Statements [Abstract] | |
Schedule of Total Consideration of Business Acquisition | The following table summarizes the total consideration for the asset acquisition and the value of assets acquired and liability assumed: Consideration (in thousands) Purchase price $ 165,000 Transactions costs and other fees 8,753 Contingent consideration 4,500 Total consideration $ 178,253 Assets Acquired (in thousands) Cash and cash equivalents $ 1,197 Prepaids 3,816 In-process research and development 173,240 Total assets acquired $ 178,253 Liability Assumed (in thousands) Contingent consideration 4,500 Total liability assumed $ 4,500 |
FAIR VALUE MEASUREMENTS (Tables
FAIR VALUE MEASUREMENTS (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Fair Value Disclosures [Abstract] | |
Assets and Liabilities Measured and Carried at Fair Value | The tables below present information about the Company’s financial assets that are measured and carried at fair value and indicate the level within the fair value hierarchy of the valuation techniques it utilizes to determine such fair value: Fair Value Measurement as of December 31, 2019 Total Level 1 Level 2 Level 3 (in thousands) Assets Money market funds $ 203,410 $ 203,410 $ — $ — Government and government agency bonds 809,159 809,159 — — Strategic equity investments 31,937 1,937 — 30,000 Certificates of deposit 1,001 1,001 — — Total assets $ 1,045,507 $ 1,015,507 $ — $ 30,000 Liabilities Contingent consideration 5,200 — — 5,200 Total liabilities $ 5,200 $ — $ — $ 5,200 Fair Value Measurement as of December 31, 2018 Total Level 1 Level 2 Level 3 (in thousands) Assets Money market funds $ 42,920 $ 42,920 $ — $ — Commercial paper 125,907 — 125,907 — Government and government agency bonds 760,235 760,235 — — Corporate bonds 43,468 43,468 — — Strategic equity investments 31,739 1,739 — 30,000 Certificates of deposit 1,001 1,001 — — Total $ 1,005,270 $ 849,363 $ 125,907 $ 30,000 |
CASH, CASH EQUIVALENTS AND MA_2
CASH, CASH EQUIVALENTS AND MARKETABLE SECURITIES (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Cash And Cash Equivalents [Abstract] | |
Summary of Company Financial Assets with Maturities of Less Than 90 Days Included in Cash Equivalents | The following table summarizes the Company’s financial assets with maturities of equal to or less than three months from the date of purchase included in cash equivalents in the consolidated balance sheets for each of the periods indicated: As of December 31, 2019 2018 (in thousands) Money market funds $ 203,410 $ 42,920 Government and government agency bonds 519,491 111,587 Commercial paper — 14,940 Total $ 722,901 $ 169,447 |
Summary of Company Cash, Cash Equivalents and Investments | The following tables summarize the Company’s cash, cash equivalents and investments for each of the periods indicated: As of December 31, 2019 Amortized Cost Gross Unrealized Gains Gross Unrealized Losses Fair Market Value (in thousands) Cash and money market funds $ 315,589 $ — $ — $ 315,589 Government and government agency bonds 809,090 71 (2 ) 809,159 Total cash, cash equivalents and investments $ 1,124,679 $ 71 $ (2 ) $ 1,124,748 As reported: Cash and cash equivalents $ 835,044 $ 36 $ — $ 835,080 Short-term investments 289,635 35 (2 ) 289,668 Total cash, cash equivalents and investments $ 1,124,679 $ 71 $ (2 ) $ 1,124,748 As of December 31, 2018 Amortized Cost Gross Unrealized Gains Gross Unrealized Losses Fair Market Value (in thousands) Cash and money market funds $ 244,302 $ — $ — $ 244,302 Commercial paper 125,907 — — 125,907 Government and government agency bonds 760,258 12 (35 ) 760,235 Corporate bonds 43,544 — (76 ) 43,468 Total cash, cash equivalents and investments $ 1,174,011 $ 12 $ (111 ) $ 1,173,912 As reported: Cash and cash equivalents $ 370,827 $ 3 $ (1 ) $ 370,829 Short-term investments 803,184 9 (110 ) 803,083 Total cash, cash equivalents and investments $ 1,174,011 $ 12 $ (111 ) $ 1,173,912 |
ACCOUNTS RECEIVABLE AND RESER_2
ACCOUNTS RECEIVABLE AND RESERVES FOR PRODUCT SALES (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Receivables Net Current [Abstract] | |
Summary of Components of Accounts Receivable | The following table summarizes the components of the Company’s accounts receivable for the periods indicated: As of December 31, 2019 2018 (in thousands) Product sales, net of discounts and allowances $ 90,409 $ 48,252 Government contract receivables 470 792 Total accounts receivable, net $ 90,879 $ 49,044 |
Summary of Change in Reserves for Discounts and Allowances | The following table summarizes an analysis of the change in reserves for discounts and allowances for the periods indicated: Chargebacks Rebates Prompt Pay Other Accruals Total (in thousands) Balance, as of December 31, 2017 $ 995 $ 6,959 $ 169 $ 464 $ 8,587 Provision 12,284 28,420 2,624 5,286 48,614 Payments/credits (11,901 ) (11,103 ) (2,255 ) (3,432 ) (28,691 ) Balance, as of December 31, 2018 $ 1,378 $ 24,276 $ 538 $ 2,318 $ 28,510 Provision 9,698 44,749 4,897 9,643 68,987 Payments/credits (10,488 ) (24,287 ) (3,929 ) (7,290 ) (45,994 ) Balance, as of December 31, 2019 $ 588 $ 44,738 $ 1,506 $ 4,671 $ 51,503 |
Summary of Total Reserves Included in Consolidated Balance Sheets | The following table summarizes the total reserves above included in the Company’s consolidated balance sheets for the periods indicated: As of December 31, 2019 2018 (in thousands) Reduction to accounts receivable $ 6,254 $ 2,364 Component of accrued expenses 45,249 26,146 Total reserves $ 51,503 $ 28,510 |
INVENTORY (Tables)
INVENTORY (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Inventory Disclosure [Abstract] | |
Summary of Components of Inventory | The following table summarizes the components of the Company’s inventory for each of the periods indicated: As of December 31, 2019 2018 (in thousands) Raw materials $ 82,030 $ 71,313 Work in progress 88,031 47,279 Finished goods 1,318 6,853 Total inventory $ 171,379 $ 125,445 |
OTHER ASSETS (Tables)
OTHER ASSETS (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Deferred Costs Capitalized Prepaid And Other Assets Disclosure [Abstract] | |
Summary of Other Current Assets | The following table summarizes the Company’s other current assets for each of the periods indicated: As of December 31, 2019 2018 (in thousands) Manufacturing-related deposits and prepaids $ 54,276 $ 39,036 Prepaid clinical and pre-clinical expenses 8,263 9,706 Prepaid maintenance services 4,366 2,994 Leasehold improvement receivable 3,059 13,474 Prepaid insurance 2,573 1,006 Prepaid income tax 2,114 2,130 Prepaid research expenses 2,007 1,932 Other 5,249 7,504 Total other current assets $ 81,907 $ 77,782 |
Summary of Other Non-current Assets | The following table summarizes the Company’s other non-current assets for each of the periods indicated: As of December 31, 2019 2018 (in thousands) Manufacturing-related deposits and prepaids $ 122,091 $ 62,821 Strategic investments 31,937 31,739 Restricted cash and investments 9,566 1,001 Prepaid clinical expenses 4,665 7,541 Alternative minimum tax credit 3,367 3,367 Other 2,233 825 Total other non-current assets $ 173,859 $ 107,294 |
PROPERTY AND EQUIPMENT, NET (Ta
PROPERTY AND EQUIPMENT, NET (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Property Plant And Equipment [Abstract] | |
Summarizes Components of Property and Equipment, Net | Property and equipment are recorded at historical cost, net of accumulated depreciation. The following table summarizes components of property and equipment, net for each of the periods indicated: As of December 31, 2019 2018 (in thousands) Leasehold improvements $ 53,950 $ 20,937 Software and computer equipment 30,683 15,774 Lab equipment 30,053 17,659 Building and improvements 23,108 22,972 Furniture and fixtures 7,090 3,227 Land 5,183 4,158 Land improvements 3,403 — Office equipment 1,157 436 Construction in progress 25,988 40,010 Property and equipment, gross 180,615 125,173 Less: accumulated depreciation (50,995 ) (28,149 ) Property and equipment, net $ 129,620 $ 97,024 |
INTANGIBLE ASSETS (Table)
INTANGIBLE ASSETS (Table) | 12 Months Ended |
Dec. 31, 2019 | |
Goodwill And Intangible Assets Disclosure [Abstract] | |
Summary of Components of Intangible Assets | The following table summarizes the components of the Company’s intangible assets for each of the periods indicated: As of December 31, 2019 2018 (in thousands) Patents $ 8,902 $ 7,227 In-licensed rights 8,073 7,573 Software licenses 1,029 626 Intangible assets, gross 18,004 15,426 Less: accumulated amortization (5,507 ) (3,852 ) Intangible assets, net $ 12,497 $ 11,574 |
Summary of Estimated Future Amortization for Intangible Assets | The following table summarizes the estimated future amortization for intangible assets: As of December 31, 2019 (in thousands) 2020 $ 1,358 2021 1,168 2022 1,156 2023 1,156 2024 1,149 Thereafter 6,510 Total $ 12,497 |
ACCRUED EXPENSES (Tables)
ACCRUED EXPENSES (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Payables And Accruals [Abstract] | |
Summary of Accrued Expenses | The following table summarizes the Company’s accrued expenses for each of the periods indicated: As of December 31, 2019 2018 (in thousands) Product revenue related reserves $ 45,249 $ 26,146 Accrued employee compensation costs 43,240 24,692 Accrued contract manufacturing costs 27,622 15,794 Accrued milestone expense 18,390 24,020 Accrued clinical and pre-clinical costs 18,010 11,396 Accrued professional fees 10,707 11,319 Accrued collaboration cost-sharing 9,000 2,167 Accrued royalties 6,301 8,254 Other 7,008 10,307 Total accrued expenses $ 185,527 $ 134,095 |
INDEBTEDNESS (Tables)
INDEBTEDNESS (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Debt Disclosure [Abstract] | |
Summary of Debt Facilities | The following table summarizes the Company’s debt facilities for the periods indicated: As of December 31, 2019 2018 (in thousands) Principal amount of the 2024 Notes $ 570,000 $ 570,000 Unamortized discount - equity component (120,182 ) (140,206 ) Unamortized discount - debt issuance costs (7,922 ) (9,240 ) Net carrying value of 2024 Notes 441,896 420,554 Principal amount of the 2019 Term Loan 250,000 — Unamortized discounts (9,996 ) — Net carrying value of 2019 Term Loan 240,004 — Total carrying value of debt facilities 681,900 420,554 Fair value of 2024 Notes 1,141,288 952,681 Fair value of 2019 Term Loan 250,000 — Total fair value of debt facilities $ 1,391,288 $ 952,681 |
Summarizes Total Gross Payments Due under Company's Debt Arrangements | The following table summarizes the total gross payments due under the Company’s debt arrangements: As of December 31, 2019 (in thousands) 2020 $ — 2021 — 2022 — 2023 250,000 2024 570,000 Thereafter — Total payments $ 820,000 |
STOCK-BASED COMPENSATION (Table
STOCK-BASED COMPENSATION (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Summary of Stock Option Activity | The following tables summarize the Company’s stock option activity for each of the periods indicated: For the Year Ended December 31, 2019 2018 2017 Weighted Weighted Weighted Average Average Average Exercise Exercise Exercise Shares Price Shares Price Shares Price Grants outstanding at beginning of the period 8,391,171 $ 46.09 8,806,204 $ 29.74 5,436,951 $ 22.70 Granted 1,429,652 132.97 2,152,439 90.15 4,805,722 (1) 35.09 Exercised (1,055,715 ) 30.73 (2,119,306 ) 22.89 (792,845 ) 17.40 Expired and forfeited (418,760 ) 84.15 (448,166 ) 46.11 (643,624 ) 25.44 Grants outstanding at end of the period 8,346,348 $ 61.01 8,391,171 $ 46.09 8,806,204 $ 29.74 Grants exercisable at end of the period 2,368,621 $ 45.33 2,304,791 $ 27.69 3,288,712 $ 24.76 Grants vested and expected to vest at end of the period 7,987,427 $ 58.65 6,643,835 $ 45.43 6,910,022 $ 28.49 (1) Includes 3,300,000 options with service and market conditions granted to the Company’s CEO. These options have a five-year The weighted-average grant date fair value per share of stock options granted during the years ended December 31, 2019, 2018 and 2017 was $70.93, $44.66 and $14.78, respectively. Weighted Aggregate Average Intrinsic Remaining Value Contractual (in thousands) Life (Years) Options outstanding at December 31, 2019 $ 587,191 7.5 Options exercisable at December 31, 2019 $ 199,438 6.0 Options vested and expected to vest at December 31, 2019 $ 578,938 7.4 |
Summary of Company's Stock Options Vested and Exercised | The following table summarizes the Company’s stock options vested and exercised for each of the periods indicated: For the Year Ended December 31, 2019 2018 2017 (in thousands) Aggregate grant date fair value of stock options vested $ 50,878 $ 16,316 $ 18,225 Aggregate intrinsic value of stock options exercised $ 109,707 $ 158,936 $ 20,922 |
Summary of Stock Appreciation Rights Activity | The following table summarizes the Company’s SAR activity for each of the periods indicated: For the Year Ended December 31, 2019 2018 2017 Weighted Weighted Weighted Average Average Average Exercise Exercise Exercise Shares Price Shares Price Shares Price Grants outstanding at beginning of the period 100,000 $ 23.85 100,000 $ 23.85 100,000 $ 23.85 Exercised (100,000 ) $ (23.85 ) — $ — — $ — Grants outstanding at end of the period — $ — 100,000 $ 23.85 100,000 $ 23.85 Grants exercisable at end of the period — $ — 100,000 $ 23.85 100,000 $ 23.85 Grants vested and expected to vest at end of the period — $ — 100,000 $ 23.85 100,000 $ 23.85 |
Summary of Employee Stock Purchase Plan Activity and Expense | The following table summarizes the Company’s ESPP activity for each of the periods indicated: For the Year Ended December 31, 2019 2018 2017 Number of shares purchased 92,086 75,094 102,698 Proceeds received (in millions) $ 5.1 $ 2.3 $ 1.4 |
Summary of Stock-Based Compensation Expense by Function Included within Consolidated Statements of Operations and Comprehensive Loss | The following table summarizes stock-based compensation expense by function included within the consolidated statements of operations and comprehensive loss: For the Year Ended December 31, 2019 2018 2017 (in thousands) Research and development $ 27,681 $ 14,214 $ 8,542 Selling, general and administrative 50,921 35,913 21,923 Total stock-based compensation $ 78,602 $ 50,127 $ 30,465 |
Summary of Stock-Based Compensation Expense by Grant Type Included within Consolidated Statements of Operations and Comprehensive Loss | The following table summarizes stock-based compensation expense by grant type included within the consolidated statements of operations and comprehensive loss: For the Year Ended December 31, 2019 2018 2017 (in thousands) Stock options $ 53,427 $ 37,671 $ 23,416 Restricted stock awards/units 20,103 10,632 5,295 Employee stock purchase plan 5,072 1,824 1,754 Total stock-based compensation $ 78,602 $ 50,127 $ 30,465 |
Restricted Stock Awards (RSAs) [Member] | |
Summary of Restricted Stock Award and Restricted Stock Units Activity | The following table summarizes the Company’s RSA activity for each of the periods indicated: For the Year Ended December 31, 2019 2018 2017 Weighted Weighted Weighted Average Average Average Grant Date Grant Date Grant Date Shares Fair Value Shares Fair Value Shares Fair Value Grants outstanding at beginning of the period 252,321 $ 42.37 411,781 $ 37.23 153,170 $ 34.53 Granted — — 27,590 98.57 341,500 34.58 Vested (100,840 ) 40.54 (187,050 ) 39.34 (63,264 ) 14.60 Cancelled (15,356 ) 48.94 — — (19,625 ) 43.02 Grants outstanding at end of the period 136,125 $ 42.98 252,321 $ 42.37 411,781 $ 37.23 |
Restricted Stock Units (RSUs) [Member] | |
Summary of Restricted Stock Award and Restricted Stock Units Activity | The following table summarizes the Company’s RSU activity for the periods indicated: For the Year Ended December 31, 2019 2018 2017 Weighted Weighted Weighted Average Average Average Grant Date Grant Date Grant Date Shares Fair Value Shares Fair Value Shares Fair Value Grants outstanding at beginning of the period 251,298 $ 81.21 66,552 $ 33.72 — $ — Granted 511,283 131.18 230,736 87.95 181,029 33.03 Vested (84,068 ) 75.12 (30,276 ) 33.23 (78,017 ) 32.63 Cancelled (72,639 ) 103.03 (15,714 ) 71.45 (36,460 ) 32.63 Grants outstanding at end of the period 605,874 $ 121.61 251,298 $ 81.21 66,552 $ 33.72 |
Stock Options [Member] | |
Assumptions for Measuring Fair Values of Stocks | The fair values of stock options granted during the periods presented are measured on the date of grant using the Black-Scholes-Merton option-pricing model, with the following assumptions: For the Year Ended December 31, 2019 2018 2017 Risk-free interest rate (1) 1.4 - 2.5% 2.5 - 3.0% 1.6 - 2.1% Expected dividend yield (2) — — — Expected term (3) 5.04 years 5.06 years 4.2 - 4.8 years Expected volatility (4) 52.5 - 68.9% 52.4 - 60.8% 54.0 - 63.0% (1) The risk-free interest rate is estimated using an average of Treasury bill interest rates over a historical period commensurate with the expected term of the option that correlates to the prevailing interest rates at the time of grant. (2) The expected dividend yield is zero as the Company has not paid any dividends to date and does not expect to pay dividends in the future. (3) The expected term is estimated using historical exercise behavior. (4) The expected volatility is the implied volatility in exchange-traded options of the Company’s common stock. |
OTHER (LOSS) INCOME (Tables)
OTHER (LOSS) INCOME (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Other Income And Expenses [Abstract] | |
Summary of Other Income and Loss | The following table summarizes other income and loss for the periods indicated: For the Year Ended December 31, 2019 2018 2017 (in thousands) Interest expense $ (30,669 ) $ (33,709 ) $ (5,801 ) Interest income 7,238 6,810 1,809 Amortization of investment discount 15,350 8,573 1,401 Other expense (236 ) (656 ) 601 Gain from sale of Priority Review Voucher — — 125,000 Total other (loss) income $ (8,317 ) $ (18,982 ) $ 123,010 |
INCOME TAXES (Tables)
INCOME TAXES (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Income Tax Disclosure [Abstract] | |
Summary of Loss before Provision for Income Taxes by Jurisdiction | The following table summarizes the loss before the provision for income taxes by jurisdiction for the periods indicated: For the Year Ended December 31, 2019 2018 2017 (in thousands) Domestic $ (489,747 ) $ (309,294 ) $ (45,686 ) Foreign (224,133 ) (53,316 ) (2,942 ) Total $ (713,880 ) $ (362,610 ) $ (48,628 ) |
Summary of Provision for Income Taxes | The following table summarizes provision for income taxes in the accompanying consolidated financial stat ements for the periods indicated : For the Year Ended December 31, 2019 2018 2017 (in thousands) Current provision: Federal $ — $ (110 ) $ 204 State 521 (653 ) 1,856 Foreign 1,050 311 — Total current provision 1,571 (452 ) 2,060 Deferred benefit: Federal (15 ) — — State (5 ) — — Foreign (356 ) (240 ) — Total deferred benefit (376 ) (240 ) — Total current provision $ 1,195 $ (692 ) $ 2,060 |
Reconciliation Between Effective Tax Rate and Federal Income Tax Rate | The following table summarizes the reconciliation between the Company’s effective tax rate and the income tax rate for each of the periods indicated: For the Year Ended December 31, 2019 2018 2017 Federal income tax rate 21.0 % 21.0 % 34.0 % State taxes 6.3 12.3 (27.7 ) Research and development and other tax credits 3.3 3.1 8.5 Valuation allowance (16.8 ) (45.5 ) (93.2 ) Permanent differences 1.8 6.9 6.4 Sarepta International C.V. return to provision — (0.1 ) 62.1 Impact of tax reform, net of valuation allowance — — 5.9 Basis difference in subsidiary (8.4 ) — — Foreign rate differential (7.4 ) (0.9 ) (0.6 ) Other — 3.4 0.4 Effective tax rate (0.2 ) % 0.2 % (4.2 ) % |
Analysis of Deferred Tax Assets and Liabilities | The following table summarizes the analysis of the deferred tax assets and liabilities for each of the periods indicated: As of December 31, 2019 2018 (in thousands) Deferred tax assets: Net operating loss carryforwards $ 304,033 $ 212,342 Difference in depreciation and amortization 40,095 55,162 Research and development tax credits 103,806 67,309 Stock-based compensation 24,114 15,538 Lease liabilities 15,796 4,831 Deferred revenue 939 888 Capitalized inventory 18,255 22,943 Other 16,270 15,727 Total deferred tax assets 523,308 394,740 Deferred tax liabilities: Right of use asset (10,782 ) — Debt discount (23,099 ) (25,162 ) Total deferred tax liabilities (33,881 ) (25,162 ) Valuation allowance (488,829 ) (369,345 ) Net deferred tax assets $ 598 $ 233 |
Reconciliation of Beginning and Ending Amount of Unrecognized Tax Benefits | The follow table summarizes the reconciliation of the beginning and ending amount of total unrecognized tax benefits for each of the periods indicated: For the Year Ended December 31, 2019 2018 2017 (in thousands) Balance at beginning of the period $ 37,544 $ 5,134 $ 4,644 Increase related to current year tax positions 4,275 2,164 735 Increase related to prior year tax positions 109 30,246 — Decrease related to prior year tax positions (175 ) — (245 ) Balance at end of the period $ 41,753 $ 37,544 $ 5,134 |
LEASES (Tables)
LEASES (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Leases [Abstract] | |
Summary of Lease Costs Recognized Under Topic 842 and Other Information Pertaining to Operating Leases | The following table contains a summary of the lease costs recognized under ASC 842 and other information pertaining to the Company’s operating leases for the year ended December 31, 2019: For the Year Ended December 31, 2019 (in thousands) Lease cost Operating lease cost $ 10,335 Variable lease cost 3,967 Total lease cost $ 14,302 Other information Operating lease payments 10,416 Operating lease liabilities arising from obtaining ROU assets — Weighted average remaining lease term 5.5 Weighted average discount rate 7.50 % |
Summary of Maturities of Lease Liabilities and Reconciliation of Lease Liabilities Recognized Under Topic 842 | The following table summarizes maturities of lease liabilities and the reconciliation of lease liabilities as of December 31, 2019: As of December 31, 2019 (in thousands) 2020 $ 11,718 2021 12,891 2022 11,080 2023 11,230 2024 11,471 Thereafter 9,654 Total minimum lease payments 68,044 Less: imputed interest (12,478 ) Total operating lease liabilities $ 55,566 Included in the condensed consolidated balance sheet: Current portion of lease liabilities within other current liabilities $ 7,846 Lease liabilities 47,720 Total operating lease liabilities $ 55,566 |
Summary of Aggregate Future Minimum Non-Cancellable Commitments Under Leases | For comparable purposes, aggregate future minimum non-cancellable commitments under leases as of December 31, 2018, are as follows: As of December 31, 2018 (in thousands) 2020 $ 11,395 2021 12,558 2022 10,757 2023 10,898 2024 11,128 Thereafter 9,396 Total minimum lease payments $ 66,132 |
NET LOSS PER SHARE (Tables)
NET LOSS PER SHARE (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Earnings Per Share [Abstract] | |
Basic and Diluted Net Loss Per Share | Given that the Company recorded a net loss for each of the periods presented, there is no difference between basic and diluted net loss per share since the effect of common stock equivalents would be anti-dilutive and are, therefore, excluded from the diluted net loss per share calculation. For the Year Ended December 31, 2019 2018 2017 (in thousands, except per share amounts) Net loss $ (715,075 ) $ (361,918 ) $ (50,688 ) Weighted-average common shares outstanding - basic 73,615 66,250 58,818 Effect of dilutive securities* — — — Weighted-average common shares outstanding - diluted 73,615 66,250 58,818 Net loss per share — basic and diluted $ (9.71 ) $ (5.46 ) $ (0.86 ) * For the years ended December 31, 2019, 2018 and 2017, stock options, RSAs, RSUs, SARs and ESPP to purchase approximately 9.1 million, 9.1 million and 9.4 million shares of common stock, respectively, were excluded from the net loss per share calculation as their effect would have been anti-dilutive. The Company accounts for the effect of the 2024 Notes on diluted net earnings per share using the if-converted method as they may be settled in cash or shares at the Company’s option. While the closing price on December 31, 2019 exceeded the conversion price of $73.42, the potential shares issuable under the 2024 Notes were excluded from the calculation of diluted loss per share as they were anti-dilutive using the if-converted method. In the period of conversion, the 2024 Notes will have no impact on diluted net loss if they are settled in cash and will have an impact on diluted earnings per share if the 2024 Notes are settled in shares upon conversion and when the Company is in an income position. |
COMMITMENTS AND CONTINGENCIES (
COMMITMENTS AND CONTINGENCIES (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Commitments And Contingencies Disclosure [Abstract] | |
Summary of Aggregate Non-Cancelable Contractual Obligations Arising from Manufacturing Obligations | The following table presents non-cancelable contractual obligations arising from long-term contractual arrangements: As of December 31, 2019 (in thousands) 2020 $ 378,744 2021 183,661 2022 64,653 2023 58,319 2024 58,309 Thereafter 149,350 Total manufacturing commitments $ 893,036 |
FINANCIAL INFORMATION BY QUAR_2
FINANCIAL INFORMATION BY QUARTER (UNAUDITED) (Tables) | 12 Months Ended |
Dec. 31, 2019 | |
Quarterly Financial Information Disclosure [Abstract] | |
Financial Information by Quarter | 2019 for Quarter Ended December 31 September 30 June 30 March 31 (in thousands) Revenues: Product, net $ 100,113 $ 99,041 $ 94,668 $ 87,011 Total revenues 100,113 99,041 94,668 87,011 Cost and expenses: Cost of sales (excluding amortization of in-licensed rights) 15,567 13,037 15,919 12,063 Research and development 223,141 133,949 113,266 90,553 Selling, general and administrative 81,424 75,429 67,393 60,566 Acquired in-process research and development — — 173,240 — Settlement and license charges 10,000 — — — Amortization of in-licensed rights 200 216 217 216 Total cost and operating expenses 330,332 222,631 370,035 163,398 Operating loss (230,219 ) (123,590 ) (275,367 ) (76,387 ) Other loss: Other expense, net (4,773 ) (2,510 ) (862 ) (172 ) Other loss (4,773 ) (2,510 ) (862 ) (172 ) Loss before income tax expense (234,992 ) (126,100 ) (276,229 ) (76,559 ) Income tax expense 711 226 174 84 Net loss $ (235,703 ) $ (126,326 ) $ (276,403 ) $ (76,643 ) Net loss per share - basic and diluted $ (3.16 ) $ (1.70 ) $ (3.74 ) $ (1.07 ) Weighted average number of shares of common stock used in computing basic and diluted net loss per share 74,557 74,177 73,958 71,731 2018 for Quarter Ended December 31 September 30 June 30 March 31 (in thousands) Revenues: Product, net $ 84,415 $ 78,486 $ 73,529 $ 64,604 Total revenues 84,415 78,486 73,529 64,604 Cost and expenses: Cost of sales (excluding amortization of in-licensed rights) 13,135 8,741 6,735 5,582 Research and development 146,207 86,584 122,848 46,204 Selling, general and administrative 64,220 53,044 47,156 43,341 Amortization of in-licensed rights 216 216 217 216 Total cost and operating expenses 223,778 148,585 176,956 95,343 Operating loss (139,363 ) (70,099 ) (103,427 ) (30,739 ) Other loss: Interest expense and other, net (2,311 ) (6,968 ) (5,218 ) (4,485 ) Total other loss (2,311 ) (6,968 ) (5,218 ) (4,485 ) Loss before income (benefit) tax expense (141,674 ) (77,067 ) (108,645 ) (35,224 ) Income tax (benefit) expense (779 ) (674 ) 622 139 Net loss $ (140,895 ) $ (76,393 ) $ (109,267 ) $ (35,363 ) Net loss per share - basic and diluted $ (2.05 ) $ (1.15 ) $ (1.67 ) $ (0.55 ) Weighted average number of shares of common stock used in computing basic and diluted net loss per share 68,653 66,209 65,484 64,631 |
Organization and Nature of Bu_2
Organization and Nature of Business - Additional Information (Detail) - USD ($) $ in Thousands | Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 |
Organization Consolidation And Presentation Of Financial Statements [Abstract] | |||
Cash, cash equivalents and investments | $ 1,134,400 | ||
Cash and cash equivalents | 835,080 | $ 370,829 | $ 599,691 |
Short-term investments | 289,668 | 803,083 | |
Restricted cash and investments | $ 9,566 | $ 1,001 |
Summary of Significant Accoun_4
Summary of Significant Accounting Policies and Recent Accounting Pronouncements - Additional Information (Detail) $ in Thousands | 12 Months Ended | |||
Dec. 31, 2019USD ($)Segment | Dec. 31, 2018 | Dec. 31, 2017 | Jan. 01, 2019USD ($) | |
Summary Of Significant Accounting Policies [Line Items] | ||||
Number of operating segments | Segment | 1 | |||
Right-of-use assets | $ 37,933 | $ 42,500 | ||
Operating lease liabilities | $ 55,566 | $ 60,100 | ||
Employee Stock Purchase Plan [Member] | ||||
Summary Of Significant Accounting Policies [Line Items] | ||||
Percentage of closing price of common stock | 85.00% | |||
Percentage of discount recognized on graded-vesting based on stock-based compensation expense over the purchase period | 15.00% | |||
Patents [Member] | ||||
Summary Of Significant Accounting Policies [Line Items] | ||||
Initial term of patents | 20 years | |||
Product Revenues [Member] | Customer One [Member] | ||||
Summary Of Significant Accounting Policies [Line Items] | ||||
Concentration of credit risk percentage | 43.00% | 42.00% | 47.00% | |
Product Revenues [Member] | Customer Two [Member] | ||||
Summary Of Significant Accounting Policies [Line Items] | ||||
Concentration of credit risk percentage | 41.00% | 38.00% | 34.00% | |
Product Revenues [Member] | Customer Three [Member] | ||||
Summary Of Significant Accounting Policies [Line Items] | ||||
Concentration of credit risk percentage | 13.00% | 18.00% | 19.00% | |
Accounts Receivable [Member] | Customer One [Member] | ||||
Summary Of Significant Accounting Policies [Line Items] | ||||
Concentration of credit risk percentage | 45.00% | 51.00% | ||
Accounts Receivable [Member] | Customer Two [Member] | ||||
Summary Of Significant Accounting Policies [Line Items] | ||||
Concentration of credit risk percentage | 37.00% | 28.00% | ||
Accounts Receivable [Member] | Customer Three [Member] | ||||
Summary Of Significant Accounting Policies [Line Items] | ||||
Concentration of credit risk percentage | 11.00% | 10.00% | ||
Minimum [Member] | ||||
Summary Of Significant Accounting Policies [Line Items] | ||||
Accounts receivable payment term | 60 days | |||
Maximum [Member] | ||||
Summary Of Significant Accounting Policies [Line Items] | ||||
Accounts receivable payment term | 91 days | |||
Outside of U.S. [Member] | Minimum [Member] | ||||
Summary Of Significant Accounting Policies [Line Items] | ||||
Accounts receivable payment term | 45 days | |||
Outside of U.S. [Member] | Maximum [Member] | ||||
Summary Of Significant Accounting Policies [Line Items] | ||||
Accounts receivable payment term | 150 days |
Summary of Estimated Useful Liv
Summary of Estimated Useful Lives of Plant and Equipment (Detail) | 12 Months Ended |
Dec. 31, 2018 | |
Lab Equipment [Member] | |
Property, Plant and Equipment [Line Items] | |
Property and equipment, Estimated useful life | 5 years |
Office Equipment [Member] | |
Property, Plant and Equipment [Line Items] | |
Property and equipment, Estimated useful life | 5 years |
Software and Computer Equipment [Member] | Minimum [Member] | |
Property, Plant and Equipment [Line Items] | |
Property and equipment, Estimated useful life | 3 years |
Software and Computer Equipment [Member] | Maximum [Member] | |
Property, Plant and Equipment [Line Items] | |
Property and equipment, Estimated useful life | 5 years |
Leasehold Improvements [Member] | |
Property, Plant and Equipment [Line Items] | |
Property and equipment, Estimated useful life | Lesser of the useful life or the term of the respective lease |
Furniture and Fixtures [Member] | |
Property, Plant and Equipment [Line Items] | |
Property and equipment, Estimated useful life | 7 years |
Land [Member] | |
Property, Plant and Equipment [Line Items] | |
Property and equipment, Estimated useful life | Not depreciated |
Land Improvements [Member] | |
Property, Plant and Equipment [Line Items] | |
Property and equipment, Estimated useful life | 25 years |
Construction in Progress [Member] | |
Property, Plant and Equipment [Line Items] | |
Property and equipment, Estimated useful life | Not depreciated until put into service |
Building and Improvements [Member] | |
Property, Plant and Equipment [Line Items] | |
Property and equipment, Estimated useful life | 30 years |
License and Collaboration Agr_3
License and Collaboration Agreements - Additional Information (Detail) | Dec. 21, 2019USD ($) | Nov. 14, 2019USD ($)shares | Apr. 04, 2019 | Feb. 27, 2019USD ($) | Oct. 31, 2018USD ($)shares | May 31, 2018USD ($) | Jul. 31, 2017USD ($) | May 31, 2017USD ($) | Jun. 30, 2016USD ($) | Dec. 31, 2019USD ($)shares | Sep. 30, 2019USD ($) | Jun. 30, 2019USD ($) | Mar. 31, 2019USD ($) | Dec. 31, 2018USD ($)shares | Sep. 30, 2018USD ($) | Jun. 30, 2018USD ($) | Mar. 31, 2018USD ($) | Dec. 31, 2019USD ($)Milestoneshares | Dec. 31, 2018USD ($)Milestoneshares | Dec. 31, 2017USD ($) | Nov. 22, 2019 | Aug. 08, 2018USD ($)shares |
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||||||||
Research and development expense | $ 223,141,000 | $ 133,949,000 | $ 113,266,000 | $ 90,553,000 | $ 146,207,000 | $ 86,584,000 | $ 122,848,000 | $ 46,204,000 | ||||||||||||||
Common stock, issued | shares | 75,184,863 | 71,071,887 | 75,184,863 | 71,071,887 | ||||||||||||||||||
Common stock, $.0001 par value, 99,000,000 shares authorized; 75,184,863 and 71,071,887 issued and outstanding at December 31, 2019 and 2018, respectively | $ 8,000 | $ 7,000 | $ 8,000 | $ 7,000 | ||||||||||||||||||
Contingent consideration liability of acquisition | 5,200,000 | 5,200,000 | ||||||||||||||||||||
Intangible asset, net | 12,497,000 | 11,574,000 | 12,497,000 | 11,574,000 | ||||||||||||||||||
Settlement and license charges | 10,000,000 | 10,000,000 | $ 28,427,000 | |||||||||||||||||||
Amortization of in-licensed rights | 200,000 | $ 216,000 | 217,000 | $ 216,000 | 216,000 | $ 216,000 | $ 217,000 | $ 216,000 | 849,000 | 865,000 | 1,053,000 | |||||||||||
Maximum [Member] | ||||||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||||||||
Percentage of royalty payments | 8.00% | |||||||||||||||||||||
Amortization of in-licensed rights | 1,100,000 | |||||||||||||||||||||
Minimum [Member] | ||||||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||||||||
Percentage of royalty payments | 4.00% | |||||||||||||||||||||
StrideBio, Inc. [Member] | ||||||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||||||||
Up-front cash payment under agreements | $ 17,500,000 | |||||||||||||||||||||
Up-front cash expense | $ 46,900,000 | |||||||||||||||||||||
Common stock, issued | shares | 301,980 | |||||||||||||||||||||
Common stock, $.0001 par value, 99,000,000 shares authorized; 75,184,863 and 71,071,887 issued and outstanding at December 31, 2019 and 2018, respectively | $ 29,400,000 | |||||||||||||||||||||
Up-front cash payment under development | 450,000,000 | |||||||||||||||||||||
Up-front cash payment under sales milestone | 835,000,000 | |||||||||||||||||||||
Up-front cash payment under agreements | 46,900,000 | |||||||||||||||||||||
StrideBio, Inc. [Member] | Maximum [Member] | ||||||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||||||||
Up-front cash payment under agreements | $ 42,500,000 | |||||||||||||||||||||
Myonexus Therapeutics, Inc. [Member] | ||||||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||||||||
Final exercise price | $ 165,000,000 | |||||||||||||||||||||
Additional transaction fees | 8,800,000 | 165,000,000 | 165,000,000 | |||||||||||||||||||
Additional development milestone payments to be paid | 200,000,000 | |||||||||||||||||||||
Date of acquisition | Apr. 4, 2019 | |||||||||||||||||||||
Contingent consideration liability of acquisition | $ 4,500,000 | 4,500,000 | 4,500,000 | |||||||||||||||||||
Lacerta Therapeutics [Member] | ||||||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||||||||
Research and development expense | $ 0 | |||||||||||||||||||||
Number of development milestone | Milestone | 0 | |||||||||||||||||||||
BioMarin Pharmaceutical, Inc. [Member] | ||||||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||||||||
Up-front cash payment under agreements | $ 35,000,000 | |||||||||||||||||||||
Regulatory and sales milestone payments | $ 0 | 0 | 0 | |||||||||||||||||||
Intangible asset, net | 6,600,000 | |||||||||||||||||||||
Settlement and license charges | 28,400,000 | |||||||||||||||||||||
Regulatory milestone payable | 10,000,000 | 10,000,000 | ||||||||||||||||||||
Sales milestone payment recorded as an in-license right | 0 | 0 | ||||||||||||||||||||
Royalty expense | 19,400,000 | 15,100,000 | 4,700,000 | |||||||||||||||||||
BioMarin Pharmaceutical, Inc. [Member] | Intellectual Property | ||||||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||||||||
Amortization of in-licensed rights | 4,200,000 | |||||||||||||||||||||
BioMarin Pharmaceutical, Inc. [Member] | US [Member] | ||||||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||||||||
Royalty payment expiry period | 2024-03 | |||||||||||||||||||||
BioMarin Pharmaceutical, Inc. [Member] | EU [member] | ||||||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||||||||
Royalty payment expiry period | 2024-12 | |||||||||||||||||||||
BioMarin Pharmaceutical, Inc. [Member] | Maximum [Member] | ||||||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||||||||
Regulatory and sales milestone payments | $ 65,000,000 | |||||||||||||||||||||
BioMarin Pharmaceutical, Inc. [Member] | Maximum [Member] | Other Countries [Member] | ||||||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||||||||
Royalty payment expiry period | 2024-12 | |||||||||||||||||||||
University of Western Australia [Member] | ||||||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||||||||
Regulatory and sales milestone payments | 0 | |||||||||||||||||||||
Sales milestone payment recorded as an in-license right | 0 | 0 | ||||||||||||||||||||
Royalty expense | 3,500,000 | 0 | 0 | |||||||||||||||||||
Carrying value of intangible assets | 1,100,000 | 1,100,000 | ||||||||||||||||||||
University of Western Australia [Member] | EXONDYS 51 | ||||||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||||||||
Sales milestone payment recorded as an in-license right | 1,000,000 | 1,000,000 | ||||||||||||||||||||
University of Western Australia [Member] | VYONDYS 53 [Member] | ||||||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||||||||
Sales milestone payment recorded as an in-license right | 500,000 | 500,000 | ||||||||||||||||||||
University of Western Australia [Member] | Maximum [Member] | EXONDYS 51 | ||||||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||||||||
Payments to UWA on successful achievement of certain development and regulatory milestones | $ 26,000,000 | |||||||||||||||||||||
Collaborative Arrangement | ||||||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||||||||
Percentage in development costs | 0.75 | |||||||||||||||||||||
Collaborative Arrangement | Roche Holding A.G. [Member] | ||||||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||||||||
Proceeds From License And Collaborative Agreement | $ 1,200,000,000 | |||||||||||||||||||||
Upfront Cash Payment Received | 750,000,000 | |||||||||||||||||||||
Stock Issued During Period Value Collaboration and License Agreement | 400,000,000 | |||||||||||||||||||||
Collaborative Arrangement | Roche Holding A.G. [Member] | Maximum [Member] | ||||||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||||||||
Regulatory and Sales Milestones Payment Received | 1,700,000,000 | |||||||||||||||||||||
Option exercise payment | 125,000,000 | |||||||||||||||||||||
Collaborative Arrangement | Roche Holding A.G. [Member] | Minimum [Member] | ||||||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||||||||
Option exercise payment | $ 20,000,000 | |||||||||||||||||||||
Collaborative Arrangement | Genethon [Member] | ||||||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||||||||
Percentage in development costs | 0.25 | |||||||||||||||||||||
Research and development expense | 9,000,000 | |||||||||||||||||||||
Up-front cash payment under agreements | $ 28,000,000 | |||||||||||||||||||||
Collaborative Arrangement | Genethon [Member] | Genethon Products [Member] | ||||||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||||||||
Development, regulatory and sales milestones payment | 157,500,000 | |||||||||||||||||||||
Collaborative Arrangement | Genethon [Member] | Other Licensed Products [Member] | ||||||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||||||||
Development, regulatory and sales milestones payment | $ 78,800,000 | |||||||||||||||||||||
Collaborative Arrangement | Lysogene S.A. [Member] | ||||||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||||||||
Research and development expense | 44,800,000 | |||||||||||||||||||||
Upfront License Fee | 26,100,000 | 26,100,000 | ||||||||||||||||||||
Development milestone payment | 18,700,000 | |||||||||||||||||||||
Collaborative Arrangement | University of Western Australia [Member] | ||||||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||||||||
Development Milestone and Settlement Upfront Fee Recognized as Research and Development Expense | 113,200,000 | 142,400,000 | $ 22,000,000 | |||||||||||||||||||
Collaborative Arrangement | University of Western Australia [Member] | Maximum [Member] | ||||||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||||||||
Development, regulatory, commercial milestone and up-front royalty payments | 3,000,000,000 | 3,000,000,000 | ||||||||||||||||||||
Warrant Agreement | Myonexus Therapeutics, Inc. [Member] | ||||||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||||||||
Research and development expense | 85,000,000 | |||||||||||||||||||||
Up-front cash payment under agreements | $ 60,000,000 | 60,000,000 | ||||||||||||||||||||
Cash consideration under agreement excluding contingent payments | $ 200,000,000 | |||||||||||||||||||||
Up-front cash payment under sales milestone | 20,000,000 | |||||||||||||||||||||
Milestone accrued payment | 5,000,000 | $ 5,000,000 | ||||||||||||||||||||
Number of development milestone | Milestone | 2 | |||||||||||||||||||||
Additional development milestone payments to be paid | 200,000,000 | 200,000,000 | ||||||||||||||||||||
Cost of asset | $ 173,200,000 | |||||||||||||||||||||
Equity Investment Agreement [Member] | Lysogene S.A. [Member] | ||||||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||||||||
Development, regulatory and sales milestones payments to be paid | $ 102,800,000 | |||||||||||||||||||||
Common stock purchased,shares | shares | 950,606 | |||||||||||||||||||||
Percentage of common stock outstanding | 8.00% | |||||||||||||||||||||
Equity Investment Agreement [Member] | Lysogene S.A. [Member] | Other Noncurrent Assets [Member] | ||||||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||||||||
Upfront License Fee | $ 1,900,000 | $ 1,900,000 | ||||||||||||||||||||
Equity Investment Agreement [Member] | Lacerta Therapeutics [Member] | ||||||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||||||||
Investment change in carrying value | 0 | $ 0 | ||||||||||||||||||||
License Development And Option Agreement | Lacerta Therapeutics [Member] | ||||||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||||||||
Up-front payment | $ 42,000,000 | |||||||||||||||||||||
Pompe License Agreement | Lacerta Therapeutics [Member] | ||||||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||||||||
Up-front payment | 8,000,000 | |||||||||||||||||||||
Pompe License Agreement | Lacerta Therapeutics [Member] | Maximum [Member] | ||||||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||||||||
Up-front payment | $ 44,000,000 | |||||||||||||||||||||
Series A Preferred Stock Purchase Agreement | Lacerta Therapeutics [Member] | Series A Preferred Stock | ||||||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||||||||
Up-front payment | shares | 4,500,000 | |||||||||||||||||||||
Series A Preferred Stock Purchase Agreement | Lacerta Therapeutics [Member] | Other Noncurrent Assets [Member] | ||||||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||||||||
Up-front payment | $ 30,000,000 | |||||||||||||||||||||
License And Purchase Agreement | Lacerta Therapeutics [Member] | ||||||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||||||||
Up-front payment | $ 38,000,000 | |||||||||||||||||||||
Nationwide License Agreement [Member] | Nationwide Children’s Hospital [Member] | ||||||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||||||||
Research and development expense | 0 | |||||||||||||||||||||
Up-front cash payment under agreements | $ 1,000,000 | |||||||||||||||||||||
Development, regulatory and sales milestones payments to be paid | $ 0 | $ 0 | ||||||||||||||||||||
Nationwide License Agreement [Member] | Nationwide Children’s Hospital [Member] | Maximum [Member] | ||||||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||||||||||||
Development, regulatory and sales milestones payments to be paid | $ 29,000,000 |
Schedule of Total Consideration
Schedule of Total Consideration of Business Acquisition (Details) - USD ($) $ in Thousands | 3 Months Ended | 12 Months Ended | |
Jun. 30, 2019 | Dec. 31, 2019 | Feb. 27, 2019 | |
Consideration | |||
Contingent consideration liability of acquisition | $ 5,200 | ||
Assets Acquired (in thousands) | |||
In-process research and development | $ 173,240 | 173,240 | |
Myonexus Therapeutics, Inc. [Member] | |||
Consideration | |||
Additional transaction fees | 165,000 | $ 8,800 | |
Transactions costs and other fees | 8,753 | ||
Contingent consideration liability of acquisition | 4,500 | $ 4,500 | |
Total consideration | 178,253 | ||
Assets Acquired (in thousands) | |||
Cash and cash equivalents | 1,197 | ||
Prepaids | 3,816 | ||
In-process research and development | 173,240 | ||
Total assets acquired | 178,253 | ||
Liability Assumed | |||
Contingent consideration | 4,500 | ||
Total liability assumed | $ 4,500 |
Gain From Sale of Priority Re_2
Gain From Sale of Priority Review Voucher - Additional Information (Detail) - USD ($) $ in Thousands | 3 Months Ended | 12 Months Ended |
Mar. 31, 2017 | Dec. 31, 2017 | |
Gain From Sale Of Intangible Asset [Line Items] | ||
Gain from sale of PRV asset | $ 125,000 | |
Gilead [Member] | ||
Gain From Sale Of Intangible Asset [Line Items] | ||
Gain from sale of PRV asset | $ 125,000 |
Assets and Liabilities Measured
Assets and Liabilities Measured and Carried at Fair Value (Detail) - USD ($) $ in Thousands | Dec. 31, 2019 | Dec. 31, 2018 |
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total assets | $ 1,045,507 | $ 1,005,270 |
Contingent consideration | 5,200 | |
Total liabilities | 5,200 | |
Money Market Funds [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total assets | 203,410 | 42,920 |
Government and Government Agency Bonds [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total assets | 809,159 | 760,235 |
Strategic Equity Investments [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total assets | 31,937 | 31,739 |
Certificates of Deposit [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total assets | 1,001 | 1,001 |
Commercial Paper [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total assets | 125,907 | |
Corporate Bonds [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total assets | 43,468 | |
Level 1 [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total assets | 1,015,507 | 849,363 |
Level 1 [Member] | Money Market Funds [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total assets | 203,410 | 42,920 |
Level 1 [Member] | Government and Government Agency Bonds [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total assets | 809,159 | 760,235 |
Level 1 [Member] | Strategic Equity Investments [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total assets | 1,937 | 1,739 |
Level 1 [Member] | Certificates of Deposit [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total assets | 1,001 | 1,001 |
Level 1 [Member] | Corporate Bonds [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total assets | 43,468 | |
Level 2 [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total assets | 125,907 | |
Level 2 [Member] | Commercial Paper [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total assets | 125,907 | |
Level 3 [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total assets | 30,000 | 30,000 |
Contingent consideration | 5,200 | |
Total liabilities | 5,200 | |
Level 3 [Member] | Strategic Equity Investments [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total assets | $ 30,000 | $ 30,000 |
Summary of Company Financial As
Summary of Company Financial Assets with Maturities of Less Than 90 Days Included in Cash Equivalents (Detail) - USD ($) $ in Thousands | Dec. 31, 2019 | Dec. 31, 2018 |
Cash and Cash Equivalents [Line Items] | ||
Cash equivalents | $ 722,901 | $ 169,447 |
Money Market Funds [Member] | ||
Cash and Cash Equivalents [Line Items] | ||
Cash equivalents | 203,410 | 42,920 |
Government and Government Agency Bonds [Member] | ||
Cash and Cash Equivalents [Line Items] | ||
Cash equivalents | $ 519,491 | 111,587 |
Commercial Paper [Member] | ||
Cash and Cash Equivalents [Line Items] | ||
Cash equivalents | $ 14,940 |
Cash, Cash Equivalents and Ma_3
Cash, Cash Equivalents and Marketable Securities - Additional Information (Detail) | 12 Months Ended | |
Dec. 31, 2019 | Dec. 31, 2018 | |
Cash And Cash Equivalents [Abstract] | ||
Weighted average maturity period of available-for-sale securities | 2 months | 2 months |
Summary of Company Cash, Cash E
Summary of Company Cash, Cash Equivalents and Investments (Detail) - USD ($) $ in Thousands | Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 |
Cash and Cash Equivalents [Line Items] | |||
Cash and cash equivalents, Amortized cost | $ 835,044 | $ 370,827 | |
Cash and cash equivalents, Gross unrealized gains | 36 | 3 | |
Cash and cash equivalents, Gross unrealized losses | (1) | ||
Cash and cash equivalents | 835,080 | 370,829 | $ 599,691 |
Cash, cash equivalents and investments, Amortized cost | 1,124,679 | 1,174,011 | |
Cash, cash equivalents and investments, Gross unrealized gains | 71 | 12 | |
Cash, cash equivalents and investments, Gross unrealized losses | (2) | (111) | |
Cash, cash equivalents and investments, Fair market value | 1,124,748 | 1,173,912 | |
Available for sale debt securities current, Amortized cost | 289,635 | 803,184 | |
Available for sale debt securities current, Gross unrealized gains | 35 | 9 | |
Available for sale debt securities current, Gross unrealized losses | (2) | (110) | |
Available for sale debt securities current, Fair market value | 289,668 | 803,083 | |
Cash and Money Market Funds [Member] | |||
Cash and Cash Equivalents [Line Items] | |||
Cash and cash equivalents, Amortized cost | 315,589 | 244,302 | |
Cash and cash equivalents | 315,589 | 244,302 | |
Commercial Paper [Member] | |||
Cash and Cash Equivalents [Line Items] | |||
Available for sale debt securities, Amortized cost | 125,907 | ||
Available for sale debt securities, Fair market value | 125,907 | ||
Government and Government Agency Bonds [Member] | |||
Cash and Cash Equivalents [Line Items] | |||
Available for sale debt securities, Amortized cost | 809,090 | 760,258 | |
Available for sale debt securities, Gross unrealized gains | 71 | 12 | |
Available for sale debt securities, Gross unrealized losses | (2) | (35) | |
Available for sale debt securities, Fair market value | $ 809,159 | 760,235 | |
Corporate Bonds [Member] | |||
Cash and Cash Equivalents [Line Items] | |||
Available for sale debt securities, Amortized cost | 43,544 | ||
Available for sale debt securities, Gross unrealized losses | (76) | ||
Available for sale debt securities, Fair market value | $ 43,468 |
Summary of Components of Accoun
Summary of Components of Accounts Receivable (Detail) - USD ($) $ in Thousands | Dec. 31, 2019 | Dec. 31, 2018 |
Receivables Net Current [Abstract] | ||
Product sales, net of discounts and allowances | $ 90,409 | $ 48,252 |
Government contract receivables | 470 | 792 |
Total accounts receivable, net | $ 90,879 | $ 49,044 |
Summary of Change in Reserves f
Summary of Change in Reserves for Discounts and Allowances (Detail) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2019 | Dec. 31, 2018 | |
Accounts Notes And Loans Receivable [Line Items] | ||
Beginning balance | $ 28,510 | $ 8,587 |
Provision | 68,987 | 48,614 |
Payments/credits | (45,994) | (28,691) |
Ending balance | 51,503 | 28,510 |
Chargebacks [Member] | ||
Accounts Notes And Loans Receivable [Line Items] | ||
Beginning balance | 1,378 | 995 |
Provision | 9,698 | 12,284 |
Payments/credits | (10,488) | (11,901) |
Ending balance | 588 | 1,378 |
Rebates [Member] | ||
Accounts Notes And Loans Receivable [Line Items] | ||
Beginning balance | 24,276 | 6,959 |
Provision | 44,749 | 28,420 |
Payments/credits | (24,287) | (11,103) |
Ending balance | 44,738 | 24,276 |
Prompt Pay [Member] | ||
Accounts Notes And Loans Receivable [Line Items] | ||
Beginning balance | 538 | 169 |
Provision | 4,897 | 2,624 |
Payments/credits | (3,929) | (2,255) |
Ending balance | 1,506 | 538 |
Other Accruals [Member] | ||
Accounts Notes And Loans Receivable [Line Items] | ||
Beginning balance | 2,318 | 464 |
Provision | 9,643 | 5,286 |
Payments/credits | (7,290) | (3,432) |
Ending balance | $ 4,671 | $ 2,318 |
Summary of Total Reserves Inclu
Summary of Total Reserves Included in Consolidated Balance Sheets (Detail) - USD ($) $ in Thousands | Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 |
Receivables Net Current [Abstract] | |||
Reduction to accounts receivable | $ 6,254 | $ 2,364 | |
Component of accrued expenses | 45,249 | 26,146 | |
Total reserves | $ 51,503 | $ 28,510 | $ 8,587 |
Inventory - Summary of Componen
Inventory - Summary of Components of Inventory (Detail) - USD ($) $ in Thousands | Dec. 31, 2019 | Dec. 31, 2018 |
Inventory Disclosure [Abstract] | ||
Raw materials | $ 82,030 | $ 71,313 |
Work in progress | 88,031 | 47,279 |
Finished goods | 1,318 | 6,853 |
Total inventory | $ 171,379 | $ 125,445 |
Summary of Other Current Assets
Summary of Other Current Assets (Detail) - USD ($) $ in Thousands | Dec. 31, 2019 | Dec. 31, 2018 |
Prepaid Expense And Other Assets Current [Abstract] | ||
Manufacturing-related deposits and prepaids | $ 54,276 | $ 39,036 |
Prepaid clinical and pre-clinical expenses | 8,263 | 9,706 |
Prepaid maintenance services | 4,366 | 2,994 |
Leasehold improvement receivable | 3,059 | 13,474 |
Prepaid insurance | 2,573 | 1,006 |
Prepaid income tax | 2,114 | 2,130 |
Prepaid research expenses | 2,007 | 1,932 |
Other | 5,249 | 7,504 |
Total other current assets | $ 81,907 | $ 77,782 |
Summary of Other Non-current As
Summary of Other Non-current Assets (Detail) - USD ($) $ in Thousands | Dec. 31, 2019 | Dec. 31, 2018 |
Prepaid Expense And Other Assets Noncurrent [Abstract] | ||
Manufacturing-related deposits and prepaids | $ 122,091 | $ 62,821 |
Strategic investments | 31,937 | 31,739 |
Restricted cash and investments | 9,566 | 1,001 |
Prepaid clinical expenses | 4,665 | 7,541 |
Alternative minimum tax credit | 3,367 | 3,367 |
Other | 2,233 | 825 |
Total other non-current assets | $ 173,859 | $ 107,294 |
Summarizes Components of Proper
Summarizes Components of Property and Equipment, Net (Detail) - USD ($) $ in Thousands | Dec. 31, 2019 | Dec. 31, 2018 |
Property, Plant and Equipment [Line Items] | ||
Property and equipment, gross | $ 180,615 | $ 125,173 |
Less: accumulated depreciation | (50,995) | (28,149) |
Property and equipment, net | 129,620 | 97,024 |
Land [Member] | ||
Property, Plant and Equipment [Line Items] | ||
Property and equipment, gross | 5,183 | 4,158 |
Building and Improvements [Member] | ||
Property, Plant and Equipment [Line Items] | ||
Property and equipment, gross | 23,108 | 22,972 |
Software and Computer Equipment [Member] | ||
Property, Plant and Equipment [Line Items] | ||
Property and equipment, gross | 30,683 | 15,774 |
Lab Equipment [Member] | ||
Property, Plant and Equipment [Line Items] | ||
Property and equipment, gross | 30,053 | 17,659 |
Furniture and Fixtures [Member] | ||
Property, Plant and Equipment [Line Items] | ||
Property and equipment, gross | 7,090 | 3,227 |
Office Equipment [Member] | ||
Property, Plant and Equipment [Line Items] | ||
Property and equipment, gross | 1,157 | 436 |
Leasehold Improvements [Member] | ||
Property, Plant and Equipment [Line Items] | ||
Property and equipment, gross | 53,950 | 20,937 |
Land Improvements [Member] | ||
Property, Plant and Equipment [Line Items] | ||
Property and equipment, gross | 3,403 | |
Construction in Progress [Member] | ||
Property, Plant and Equipment [Line Items] | ||
Property and equipment, gross | $ 25,988 | $ 40,010 |
Property and Equipment, Net - A
Property and Equipment, Net - Additional Information (Detail) - USD ($) $ in Millions | 12 Months Ended | ||
Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Property Plant And Equipment [Abstract] | |||
Depreciation expense | $ 22.8 | $ 10.2 | $ 6.4 |
Intangible Assets - Summary of
Intangible Assets - Summary of Components of Intangible Assets (Detail) - USD ($) $ in Thousands | Dec. 31, 2019 | Dec. 31, 2018 |
Finite Lived Intangible Assets [Line Items] | ||
Intangible assets, gross | $ 18,004 | $ 15,426 |
Less: accumulated amortization | 5,507 | 3,852 |
Intangible assets, net | 12,497 | 11,574 |
Patents [Member] | ||
Finite Lived Intangible Assets [Line Items] | ||
Intangible assets, gross | 8,902 | 7,227 |
In-Licensed Rights [Member] | ||
Finite Lived Intangible Assets [Line Items] | ||
Intangible assets, gross | 8,073 | 7,573 |
Software Licenses [Member] | ||
Finite Lived Intangible Assets [Line Items] | ||
Intangible assets, gross | $ 1,029 | $ 626 |
Intangible Assets - Additional
Intangible Assets - Additional Information (Detail) - USD ($) | 3 Months Ended | 12 Months Ended | ||||||||||
Dec. 31, 2019 | Sep. 30, 2019 | Jun. 30, 2019 | Mar. 31, 2019 | Dec. 31, 2018 | Sep. 30, 2018 | Jun. 30, 2018 | Mar. 31, 2018 | Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | Jul. 31, 2017 | |
Finite Lived Intangible Assets [Line Items] | ||||||||||||
Amortization of in-licensed rights | $ 200,000 | $ 216,000 | $ 217,000 | $ 216,000 | $ 216,000 | $ 216,000 | $ 217,000 | $ 216,000 | $ 849,000 | $ 865,000 | $ 1,053,000 | |
Patents [Member] | ||||||||||||
Finite Lived Intangible Assets [Line Items] | ||||||||||||
Amortization of in-licensed rights | 400,000 | 700,000 | 600,000 | |||||||||
Patents [Member] | Research and Development [Member] | ||||||||||||
Finite Lived Intangible Assets [Line Items] | ||||||||||||
Book value of patent abandoned | 200,000 | 100,000 | 600,000 | |||||||||
Impairment loss on patent assets | 0 | 3,800,000 | 0 | |||||||||
Bio Marin [Member] | ||||||||||||
Finite Lived Intangible Assets [Line Items] | ||||||||||||
Amortization of in-licensed rights | 1,700,000 | $ 2,100,000 | 1,700,000 | |||||||||
Maximum [Member] | ||||||||||||
Finite Lived Intangible Assets [Line Items] | ||||||||||||
Amortization of in-licensed rights | $ 1,100,000 | |||||||||||
EXONDYS 51 [Member] | ||||||||||||
Finite Lived Intangible Assets [Line Items] | ||||||||||||
In-lincensed Rights | 1,000,000 | |||||||||||
EXONDYS 51 [Member] | Maximum [Member] | Bio Marin [Member] | ||||||||||||
Finite Lived Intangible Assets [Line Items] | ||||||||||||
Sales milestone payment recorded as an in-license right | $ 6,600,000 | |||||||||||
VYONDYS 53 [Member] | ||||||||||||
Finite Lived Intangible Assets [Line Items] | ||||||||||||
In-lincensed Rights | $ 500,000 |
Intangible Assets - Summary o_2
Intangible Assets - Summary of Estimated Future Amortization for Intangible Assets (Detail) $ in Thousands | Dec. 31, 2019USD ($) |
Goodwill And Intangible Assets Disclosure [Abstract] | |
2020 | $ 1,358 |
2021 | 1,168 |
2022 | 1,156 |
2023 | 1,156 |
2024 | 1,149 |
Thereafter | 6,510 |
Total | $ 12,497 |
Summary of Accrued Expenses (De
Summary of Accrued Expenses (Detail) - USD ($) $ in Thousands | Dec. 31, 2019 | Dec. 31, 2018 |
Payables And Accruals [Abstract] | ||
Product revenue related reserves | $ 45,249 | $ 26,146 |
Accrued employee compensation costs | 43,240 | 24,692 |
Accrued contract manufacturing costs | 27,622 | 15,794 |
Accrued milestone expense | 18,390 | 24,020 |
Accrued clinical and pre-clinical costs | 18,010 | 11,396 |
Accrued professional fees | 10,707 | 11,319 |
Accrued collaboration cost-sharing | 9,000 | 2,167 |
Accrued royalties | 6,301 | 8,254 |
Other | 7,008 | 10,307 |
Total accrued expenses | $ 185,527 | $ 134,095 |
Indebtedness - Additional Infor
Indebtedness - Additional Information (Detail) | Dec. 13, 2019USD ($)Tranch | Nov. 14, 2017USD ($)shares$ / shares | Sep. 30, 2018USD ($) | Dec. 31, 2019USD ($)$ / shares | Dec. 31, 2018USD ($) | Dec. 31, 2017USD ($) | Jul. 31, 2017USD ($) |
Debt Instrument [Line Items] | |||||||
Debt instrument, face amount | $ 820,000,000 | ||||||
Proceeds for issuance of notes allocated to equity | $ 570,000,000 | ||||||
Amount paid for capped calls transactions | 50,901,000 | ||||||
Prepayment fee percentage | 2.00% | ||||||
Line of credit facility, lenders fee | 1.75% | ||||||
Debt extinguishment loss | $ (2,300,000) | $ (2,322,000) | |||||
Long-term debt | $ 681,900,000 | 420,554,000 | |||||
Convertible Debt, The Term Loan, The Revolver and The Mortgage Loans [Member] | |||||||
Debt Instrument [Line Items] | |||||||
Long-term debt | 681,900,000 | ||||||
Interest expense related to debt facilities | 30,700,000 | 33,700,000 | $ 5,800,000 | ||||
2024 Convertible Notes [Member] | |||||||
Debt Instrument [Line Items] | |||||||
Debt instrument, face amount | $ 570,000,000 | $ 570,000,000 | $ 570,000,000 | ||||
Debt instrument, maturity date | Nov. 15, 2024 | ||||||
Debt instrument, interest rate per annum | 1.50% | ||||||
Debt instrument, payment frequency | The 2024 Notes were issued at face value and bear interest at the rate of 1.50% per annum, payable semi-annually in cash on each May 15 and November 15, commencing on May 15, 2018. | ||||||
Debt instrument, conversion rate | 0.013621 | ||||||
Debt instrument, conversion price | $ / shares | $ 73.42 | $ 73.42 | |||||
Proceeds for issuance of notes allocated to equity | $ 161,100,000 | ||||||
Offering costs | 4,200,000 | ||||||
Debt issuance costs | $ 10,600,000 | ||||||
Effective interest rate percentage | 6.90% | 6.90% | 6.90% | ||||
Capped calls strike price | $ / shares | 73.42 | ||||||
Capped calls cap price | $ / shares | 104.88 | ||||||
Amount paid for capped calls transactions | $ 50,900,000 | ||||||
Debt discount | $ 120,182,000 | $ 140,206,000 | |||||
Debt issuance costs | 7,922,000 | $ 9,240,000 | |||||
Credit agreement interest rate per annum | 1.50% | ||||||
2024 Convertible Notes [Member] | Fundamental Change [Member] | |||||||
Debt Instrument [Line Items] | |||||||
Debt instrument, repurchase price percentage | 100.00% | ||||||
2024 Convertible Notes [Member] | Minimum [Member] | Beneficial Owner [Member] | Fundamental Change [Member] | |||||||
Debt Instrument [Line Items] | |||||||
Voting percentage | 50.00% | ||||||
2024 Convertible Notes [Member] | Common Stock [Member] | |||||||
Debt Instrument [Line Items] | |||||||
Debt instrument, convertible into shares | shares | 7,763,552 | ||||||
Biopharma Credit PLC and Biopharma Credit Investments V (Master) LP [Member] | December 2019 Term Loan [Member] | |||||||
Debt Instrument [Line Items] | |||||||
Debt instrument, maturity date | Dec. 20, 2023 | ||||||
Debt instrument, interest rate per annum | 8.50% | ||||||
Current borrowing capacity | $ 500,000,000 | ||||||
Number of tranches | Tranch | 2 | ||||||
Prepayment fee percentage | 2.00% | ||||||
Debt discount | 9,400,000 | ||||||
Debt issuance costs | 700,000 | ||||||
Proceeds after debt discount and issuance cost | $ 244,900,000 | ||||||
Credit agreement interest rate per annum | 8.50% | ||||||
Biopharma Credit PLC and Biopharma Credit Investments V (Master) LP [Member] | December 2019 Term Loan [Member] | Tranch A [Member] | |||||||
Debt Instrument [Line Items] | |||||||
Current borrowing capacity | $ 250,000,000 | ||||||
Line of credit facility, lenders fee | 1.75% | ||||||
Biopharma Credit PLC and Biopharma Credit Investments V (Master) LP [Member] | December 2019 Term Loan [Member] | Tranch B [Member] | |||||||
Debt Instrument [Line Items] | |||||||
Current borrowing capacity | $ 250,000,000 | ||||||
Line of credit facility, lenders fee | 1.75% | ||||||
Biopharma Credit PLC and Biopharma Credit Investments V (Master) LP [Member] | Minimum [Member] | December 2019 Term Loan [Member] | |||||||
Debt Instrument [Line Items] | |||||||
Prepayment fee percentage | 1.00% | ||||||
Biopharma Credit PLC and Biopharma Credit Investments V (Master) LP [Member] | Maximum [Member] | December 2019 Term Loan [Member] | |||||||
Debt Instrument [Line Items] | |||||||
Prepayment fee percentage | 2.00% | ||||||
MidCap Financial Amended and Restated Credit and Security Agreement [Member] | |||||||
Debt Instrument [Line Items] | |||||||
Debt instrument, interest rate per annum | 6.25% | ||||||
Current borrowing capacity | $ 60,000,000 | ||||||
Credit agreement interest rate per annum | 6.25% | ||||||
MidCap Financial Amended and Restated Credit and Security Agreement [Member] | Revolving Loan [Member] | |||||||
Debt Instrument [Line Items] | |||||||
Debt instrument, interest rate per annum | 3.95% | ||||||
Current borrowing capacity | $ 40,000,000 | ||||||
Credit agreement interest rate per annum | 3.95% |
Indebtedness - Summary of Debt
Indebtedness - Summary of Debt Facilities (Detail) - USD ($) | Dec. 31, 2019 | Dec. 31, 2018 | Nov. 14, 2017 |
Debt Instrument [Line Items] | |||
Principal amount | $ 820,000,000 | ||
Total carrying value of debt facilities | 681,900,000 | $ 420,554,000 | |
Total fair value of debt facilities | 1,391,288,000 | 952,681,000 | |
2024 Convertible Notes [Member] | |||
Debt Instrument [Line Items] | |||
Principal amount | 570,000,000 | 570,000,000 | $ 570,000,000 |
Unamortized discount | (120,182,000) | (140,206,000) | |
Unamortized discount - debt issuance costs | (7,922,000) | (9,240,000) | |
Net carrying value | 441,896,000 | 420,554,000 | |
Total fair value of debt facilities | 1,141,288,000 | $ 952,681,000 | |
2019 Term Loan [Member] | |||
Debt Instrument [Line Items] | |||
Principal amount | 250,000,000 | ||
Unamortized discount | (9,996,000) | ||
Net carrying value | 240,004,000 | ||
Total fair value of debt facilities | $ 250,000,000 |
Indebtedness - Summarizes Total
Indebtedness - Summarizes Total Gross Payments Due under Company's Debt Arrangements (Detail) $ in Thousands | Dec. 31, 2019USD ($) |
Debt Disclosure [Abstract] | |
2023 | $ 250,000 |
2024 | 570,000 |
Total payments | $ 820,000 |
Equity - Additional Information
Equity - Additional Information (Detail) - USD ($) $ / shares in Units, $ in Thousands, shares in Millions | 1 Months Ended | |||||
Nov. 30, 2019 | Mar. 31, 2019 | Nov. 30, 2018 | Jul. 31, 2017 | Dec. 31, 2019 | Dec. 31, 2018 | |
Class Of Stock [Line Items] | ||||||
Common stock issued | 0.3 | |||||
Common stock fair value | $ 8 | $ 7 | ||||
Common stock average price per share | $ 140.41 | $ 131 | $ 42.50 | |||
Net proceeds from issue of common stock | $ 365,400 | $ 513,400 | $ 354,000 | |||
Underwriting discounts, commission and offering expenses | $ 300 | $ 24,600 | $ 20,000 | |||
StrideBio, Inc. [Member] | ||||||
Class Of Stock [Line Items] | ||||||
Common stock fair value | $ 29,400 | |||||
Underwritten Public Offering [Member] | ||||||
Class Of Stock [Line Items] | ||||||
Common stock issued | 2.6 | 4.1 | 8.8 | |||
Underwritten Offering [Member] | ||||||
Class Of Stock [Line Items] | ||||||
Common stock issued | 0.3 | 1.2 |
Stock Based Compensation - Addi
Stock Based Compensation - Additional Information (Detail) - USD ($) | 1 Months Ended | 12 Months Ended | ||||||||||||||
Jul. 31, 2018 | Dec. 31, 2017 | Jun. 30, 2017 | Mar. 31, 2017 | Sep. 30, 2016 | Oct. 31, 2015 | Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | Jun. 30, 2019 | Jun. 30, 2018 | Dec. 31, 2016 | Jun. 30, 2016 | Sep. 30, 2014 | Jun. 30, 2013 | Jun. 30, 2011 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||||||||||||||
Term of award | 10 years | |||||||||||||||
Share based compensation granted under plan vest period | 4 years | |||||||||||||||
Weighted-average grant date fair value per share of stock-based awards, granted to employees | $ 70.93 | $ 44.66 | $ 14.78 | |||||||||||||
Stock-based compensation expense | $ 78,602,000 | $ 50,127,000 | $ 30,465,000 | |||||||||||||
Unrecognized stock based compensation expense | $ 181,100,000 | |||||||||||||||
Weighted average period expected to recognize unrecognized stock base compensation expense | 3 years | |||||||||||||||
Options with Service conditions [Member] | ||||||||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||||||||||||||
Unrecognized stock based compensation expense | $ 109,500,000 | |||||||||||||||
Awards with Service and Market Conditions [Member] | ||||||||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||||||||||||||
Unrecognized stock based compensation expense | 22,100,000 | |||||||||||||||
Stock Awards or Stock Units with Service Conditions [Member] | ||||||||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||||||||||||||
Unrecognized stock based compensation expense | 49,500,000 | |||||||||||||||
Former Chief Executive Officer [Member] | ||||||||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||||||||||||||
Stock-based compensation expense | 2,100,000 | |||||||||||||||
Maximum [Member] | Awards with Performance Conditions [Member] | ||||||||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||||||||||||||
Unrecognized stock based compensation expense | 100,000 | |||||||||||||||
Stock Options with Performance-based Conditions [Member] | ||||||||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||||||||||||||
Stock-based compensation expense | $ 100,000 | $ 200,000 | $ 900,000 | |||||||||||||
Restricted Stock Awards (RSAs) [Member] | ||||||||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||||||||||||||
Grants, Restricted stock units and awards | 27,590 | 341,500 | ||||||||||||||
Grant date fair value, restricted stock units and awards | $ 98.57 | $ 34.58 | ||||||||||||||
Restricted stock units and awards with performance conditions remaining to be vested | 411,781 | 136,125 | 252,321 | 411,781 | 153,170 | |||||||||||
Restricted Stock Awards (RSAs) [Member] | Minimum [Member] | Executives [Member] | ||||||||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||||||||||||||
Stock-based compensation expense | $ 0 | |||||||||||||||
Grants, Restricted stock units and awards | 71,925 | |||||||||||||||
Share-based compensation award, expiration date | Jun. 30, 2018 | |||||||||||||||
Grant date fair value, restricted stock units and awards | $ 48.94 | |||||||||||||||
Restricted Stock Awards (RSAs) [Member] | Maximum [Member] | Executives [Member] | ||||||||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||||||||||||||
Stock-based compensation expense | $ 3,300,000 | |||||||||||||||
Grants, Restricted stock units and awards | 89,906 | |||||||||||||||
Share-based compensation award, expiration date | Jan. 1, 2019 | |||||||||||||||
Grant date fair value, restricted stock units and awards | $ 54.29 | |||||||||||||||
Restricted Stock Units (RSUs) [Member] | ||||||||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||||||||||||||
Grants, Restricted stock units and awards | 511,283 | 230,736 | 181,029 | |||||||||||||
Grant date fair value, restricted stock units and awards | $ 131.18 | $ 87.95 | $ 33.03 | |||||||||||||
Restricted stock units and awards with performance conditions remaining to be vested | 66,552 | 605,874 | 251,298 | 66,552 | ||||||||||||
Restricted Stock Units (RSUs) [Member] | Executives [Member] | ||||||||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||||||||||||||
Stock-based compensation expense | $ 500,000 | $ 200,000 | $ 2,900,000 | |||||||||||||
Grants, Restricted stock units and awards | 156,029 | |||||||||||||||
Restricted stock units and awards with performance conditions remaining to be vested | 0 | |||||||||||||||
2011 Equity Incentive Plan [Member] | ||||||||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||||||||||||||
Common stock remain available for future grant | 0 | |||||||||||||||
Number of shares approved under the plan | 16,000,000 | |||||||||||||||
2013 Employee Stock Purchase Plan [Member] | ||||||||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||||||||||||||
Common stock remain available for future grant | 600,000 | |||||||||||||||
Number of shares approved under the plan | 500,000 | 300,000 | 300,000 | |||||||||||||
Term of award | 24 months | |||||||||||||||
Percentage of closing price of common stock | 85.00% | |||||||||||||||
2013 Employee Stock Purchase Plan [Member] | Minimum [Member] | ||||||||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||||||||||||||
Share-based compensation award, expiration date | Feb. 29, 2020 | |||||||||||||||
2013 Employee Stock Purchase Plan [Member] | Maximum [Member] | ||||||||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||||||||||||||
Share-based compensation award, expiration date | Aug. 31, 2021 | |||||||||||||||
2014 Employment Commencement Incentive Plan (2014 Plan) [Member] | ||||||||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||||||||||||||
Common stock remain available for future grant | 600,000 | |||||||||||||||
Number of shares approved under the plan | 600,000 | |||||||||||||||
Number of additional shares approved under the plan | 1,200,000 | 3,800,000 | 1,000,000 | |||||||||||||
2018 Equity Incentive Plan [Member] | ||||||||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||||||||||||||
Common stock remain available for future grant | 3,400,000 | |||||||||||||||
Number of shares approved under the plan | 2,900,000 |
Assumptions for Measuring Fair
Assumptions for Measuring Fair Values of Stocks (Detail) | 12 Months Ended | |||
Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | ||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Expected dividend yield | 0.00% | |||
Stock Options [Member] | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Risk-free interest rate, minimum | [1] | 1.40% | 2.50% | 1.60% |
Risk-free interest rate, maximum | [1] | 2.50% | 3.00% | 2.10% |
Expected term | [2] | 5 years 14 days | 5 years 21 days | |
Expected volatility, minimum | [3] | 52.50% | 52.40% | 54.00% |
Expected volatility, maximum | [3] | 68.90% | 60.80% | 63.00% |
Stock Options [Member] | Maximum [Member] | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Expected term | [2] | 4 years 9 months 18 days | ||
Stock Options [Member] | Minimum [Member] | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Expected term | [2] | 4 years 2 months 12 days | ||
[1] | The risk-free interest rate is estimated using an average of Treasury bill interest rates over a historical period commensurate with the expected term of the option that correlates to the prevailing interest rates at the time of grant. | |||
[2] | The expected term is estimated using historical exercise behavior. | |||
[3] | The expected volatility is the implied volatility in exchange-traded options of the Company’s common stock. |
Assumptions for Measuring Fai_2
Assumptions for Measuring Fair Values of Stocks (Parenthetical) (Detail) | 12 Months Ended |
Dec. 31, 2019 | |
Disclosure Of Compensation Related Costs Sharebased Payments [Abstract] | |
Expected dividend yield | 0.00% |
Summary of Stock Option Activit
Summary of Stock Option Activity (Detail) - USD ($) $ / shares in Units, $ in Thousands | 12 Months Ended | |||
Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | ||
Stock Option Shares | ||||
Grants outstanding at beginning of the period | 8,391,171 | 8,806,204 | 5,436,951 | |
Granted | 1,429,652 | 2,152,439 | 4,805,722 | [1] |
Exercised | (1,055,715) | (2,119,306) | (792,845) | |
Expired and forfeited | (418,760) | (448,166) | (643,624) | |
Grants outstanding at end of the period | 8,346,348 | 8,391,171 | 8,806,204 | |
Grants exercisable at end of the period | 2,368,621 | 2,304,791 | 3,288,712 | |
Grants vested and expected to vest at end of the period | 7,987,427 | 6,643,835 | 6,910,022 | |
Weighted Average Exercise Price | ||||
Grants outstanding at beginning of the period | $ 46.09 | $ 29.74 | $ 22.70 | |
Granted | 132.97 | 90.15 | 35.09 | |
Exercised | 30.73 | 22.89 | 17.40 | |
Expired and forfeited | 84.15 | 46.11 | 25.44 | |
Grants outstanding at end of the period | 61.01 | 46.09 | 29.74 | |
Grants exercisable at end of the period | 45.33 | 27.69 | 24.76 | |
Grants vested and expected to vest at end of the period | $ 58.65 | $ 45.43 | $ 28.49 | |
Aggregate Intrinsic Value | ||||
Outstanding at end of year | $ 587,191 | |||
Exercisable at end of year | 199,438 | |||
Vested and expected to vest | $ 578,938 | |||
Weighted Average Remaining Contractual Life (Years) | ||||
Outstanding at end of year | 7 years 6 months | |||
Exercisable at end of year | 6 years | |||
Vested and expected to vest | 7 years 4 months 24 days | |||
[1] | Includes 3,300,000 options with service and market conditions granted to the Company’s CEO. These options have a five-year |
Summary of Stock Option Activ_2
Summary of Stock Option Activity (Parenthetical) (Detail) - $ / shares | 12 Months Ended | ||
Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Fair value of options granted | $ 70.93 | $ 44.66 | $ 14.78 |
CEO [Member] | Stock Options [Member] | |||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Stock options granted | 3,300,000 | ||
Cliff vesting schedule | 5 years | ||
Fair value of options granted | $ 13.48 |
Summary of Company's Stock Opti
Summary of Company's Stock Options Vested and Exercised (Detail) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Disclosure Of Compensation Related Costs Sharebased Payments [Abstract] | |||
Aggregate grant date fair value of stock options vested | $ 50,878 | $ 16,316 | $ 18,225 |
Aggregate intrinsic value of stock options exercised | $ 109,707 | $ 158,936 | $ 20,922 |
Summary of Restricted Stock Awa
Summary of Restricted Stock Award Activity (Detail) - Restricted Stock Awards (RSAs) [Member] - $ / shares | 12 Months Ended | ||
Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Shares, Grants outstanding at beginning of the period | 252,321 | 411,781 | 153,170 |
Shares, Granted | 27,590 | 341,500 | |
Shares, Vested | (100,840) | (187,050) | (63,264) |
Shares, Cancelled | (15,356) | (19,625) | |
Shares, Grants outstanding at end of the period | 136,125 | 252,321 | 411,781 |
Weighted Average Grant Date Fair Value per Share, Grants outstanding at beginning of the period | $ 42.37 | $ 37.23 | $ 34.53 |
Weighted Average Grant Date Fair Value per Share, Granted | 98.57 | 34.58 | |
Weighted Average Grant Date Fair Value per Share, Vested | 40.54 | 39.34 | 14.60 |
Weighted Average Grant Date Fair Value per Share, Cancelled | 48.94 | 43.02 | |
Weighted Average Grant Date Fair Value per Share, Grants outstanding at end of the period | $ 42.98 | $ 42.37 | $ 37.23 |
Summary of Restricted Stock Uni
Summary of Restricted Stock Unit Activity (Detail) - Restricted Stock Units (RSUs) [Member] - $ / shares | 12 Months Ended | ||
Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Shares, Grants outstanding at beginning of the period | 251,298 | 66,552 | |
Shares, Granted | 511,283 | 230,736 | 181,029 |
Shares, Vested | (84,068) | (30,276) | (78,017) |
Shares, Cancelled | (72,639) | (15,714) | (36,460) |
Shares, Grants outstanding at end of the period | 605,874 | 251,298 | 66,552 |
Weighted Average Grant Date Fair Value per Share, Grants outstanding at beginning of the period | $ 81.21 | $ 33.72 | |
Weighted Average Grant Date Fair Value per Share, Granted | 131.18 | 87.95 | $ 33.03 |
Weighted Average Grant Date Fair Value per Share, Vested | 75.12 | 33.23 | 32.63 |
Weighted Average Grant Date Fair Value per Share, Cancelled | 103.03 | 71.45 | 32.63 |
Weighted Average Grant Date Fair Value per Share, Grants outstanding at end of the period | $ 121.61 | $ 81.21 | $ 33.72 |
Summary of Stock Appreciation R
Summary of Stock Appreciation Rights Activity (Detail) - Stock Appreciation Rights [Member] - $ / shares | 12 Months Ended | ||
Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Shares, Grants outstanding at beginning of the period | 100,000 | 100,000 | 100,000 |
Shares, Exercised | (100,000) | ||
Shares, Grants outstanding at end of the period | 100,000 | 100,000 | |
Shares, Grants exercisable at end of the period | 100,000 | 100,000 | |
Shares, Grants vested and expected to vest at end of the period | 100,000 | 100,000 | |
Weighted Average Exercise Price per Share, Grants outstanding at beginning of the period | $ 23.85 | $ 23.85 | $ 23.85 |
Weighted Average Exercise Price per Share, Exercised | $ (23.85) | ||
Weighted Average Exercise Price per Share, Grants outstanding at end of the period | 23.85 | 23.85 | |
Weighted Average Exercise Price per Share, Grants exercisable at end of the period | 23.85 | 23.85 | |
Weighted Average Exercise Price per Share, Grants vested and expected to vest at end of the period | $ 23.85 | $ 23.85 |
Summary of Employee Stock Purch
Summary of Employee Stock Purchase Plan Activity and Expense (Detail) - 2013 Employee Stock Purchase Plan [Member] - USD ($) $ in Millions | 12 Months Ended | ||
Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Number of shares purchased | 92,086 | 75,094 | 102,698 |
Proceeds received (in millions) | $ 5.1 | $ 2.3 | $ 1.4 |
Summary of Stock-Based Compensa
Summary of Stock-Based Compensation Expense by Function Included within Consolidated Statements of Operations and Comprehensive Loss (Detail) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | |||
Total stock-based compensation | $ 78,602 | $ 50,127 | $ 30,465 |
Research and Development [Member] | |||
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | |||
Total stock-based compensation | 27,681 | 14,214 | 8,542 |
Selling, General and Administrative [Member] | |||
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | |||
Total stock-based compensation | $ 50,921 | $ 35,913 | $ 21,923 |
Summary of Stock-Based Compen_2
Summary of Stock-Based Compensation Expense by Grant Type Included within Consolidated Statements of Operations and Comprehensive Loss (Detail) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | |||
Total stock-based compensation | $ 78,602 | $ 50,127 | $ 30,465 |
Stock Options [Member] | |||
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | |||
Total stock-based compensation | 53,427 | 37,671 | 23,416 |
Restricted Stock Awards/Units (RSAs/RSUs) [Member] | |||
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | |||
Total stock-based compensation | 20,103 | 10,632 | 5,295 |
Employee Stock Purchase Plan [Member] | |||
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | |||
Total stock-based compensation | $ 5,072 | $ 1,824 | $ 1,754 |
401 (K) PLAN - Additional Infor
401 (K) PLAN - Additional Information (Detail) - USD ($) $ in Millions | 12 Months Ended | |||
Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | Dec. 31, 2015 | |
Compensation And Retirement Disclosure [Abstract] | ||||
Expense related to 401 (K) Plan | $ 3.4 | $ 2.1 | $ 1.4 | |
Defined contribution plan, employers matching contribution, service period | 1 year | |||
Defined contribution plan, employers matching contribution, vesting percentage after one years of service | 100.00% | |||
Description of 401(k) plan | The Company sponsors a 401(k) Plan (“the Plan”) in the U.S. and other retirement plans in the rest of the world, all of which are defined contribution plans. The Plan is available to all employees who are age 21 or older. Participants may make voluntary contributions and the Company makes matching contributions according to the Plan’s matching formula. Matching contributions fully vest after one year of service for all employees. The expense related to the Plan primarily consists of the Company’s matching contributions. | |||
Age limit for eligibility to participate in the defined contribution plan | 21 years |
Summary of Other (Loss) Income
Summary of Other (Loss) Income (Detail) - USD ($) $ in Thousands | 3 Months Ended | 12 Months Ended | |||||||||
Dec. 31, 2019 | Sep. 30, 2019 | Jun. 30, 2019 | Mar. 31, 2019 | Dec. 31, 2018 | Sep. 30, 2018 | Jun. 30, 2018 | Mar. 31, 2018 | Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Nonoperating Income Expense [Abstract] | |||||||||||
Interest expense | $ (30,669) | $ (33,709) | $ (5,801) | ||||||||
Interest income | 7,238 | 6,810 | 1,809 | ||||||||
Amortization of investment discount | 15,350 | 8,573 | 1,401 | ||||||||
Other expense | (236) | (656) | 601 | ||||||||
Gain from sale of Priority Review Voucher | 125,000 | ||||||||||
Total other (loss) income | $ (4,773) | $ (2,510) | $ (862) | $ (172) | $ (2,311) | $ (6,968) | $ (5,218) | $ (4,485) | $ (8,317) | $ (18,982) | $ 123,010 |
Summary of Loss before Provisio
Summary of Loss before Provision for Income Taxes by Jurisdiction (Detail) - USD ($) $ in Thousands | 3 Months Ended | 12 Months Ended | |||||||||
Dec. 31, 2019 | Sep. 30, 2019 | Jun. 30, 2019 | Mar. 31, 2019 | Dec. 31, 2018 | Sep. 30, 2018 | Jun. 30, 2018 | Mar. 31, 2018 | Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Income Tax Disclosure [Abstract] | |||||||||||
Domestic | $ (489,747) | $ (309,294) | $ (45,686) | ||||||||
Foreign | (224,133) | (53,316) | (2,942) | ||||||||
Loss before income tax expense (benefit) | $ (234,992) | $ (126,100) | $ (276,229) | $ (76,559) | $ (141,674) | $ (77,067) | $ (108,645) | $ (35,224) | $ (713,880) | $ (362,610) | $ (48,628) |
Summary of Provision for Income
Summary of Provision for Income Taxes (Detail) - USD ($) $ in Thousands | 3 Months Ended | 12 Months Ended | |||||||||
Dec. 31, 2019 | Sep. 30, 2019 | Jun. 30, 2019 | Mar. 31, 2019 | Dec. 31, 2018 | Sep. 30, 2018 | Jun. 30, 2018 | Mar. 31, 2018 | Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Current provision: | |||||||||||
Federal | $ (110) | $ 204 | |||||||||
State | $ 521 | (653) | 1,856 | ||||||||
Foreign | 1,050 | 311 | |||||||||
Total current provision | 1,571 | (452) | 2,060 | ||||||||
Deferred benefit: | |||||||||||
Federal | (15) | ||||||||||
State | (5) | ||||||||||
Foreign | (356) | (240) | |||||||||
Total deferred benefit | (376) | (240) | |||||||||
Total current provision | $ 711 | $ 226 | $ 174 | $ 84 | $ (779) | $ (674) | $ 622 | $ 139 | $ 1,195 | $ (692) | $ 2,060 |
Reconciliation Between Effectiv
Reconciliation Between Effective Tax Rate and Federal Income Tax Rate (Detail) | 12 Months Ended | ||
Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Income Tax Disclosure [Abstract] | |||
Federal income tax rate | 21.00% | 21.00% | 34.00% |
State taxes | 6.30% | 12.30% | (27.70%) |
Research and development and other tax credits | 3.30% | 3.10% | 8.50% |
Valuation allowance | (16.80%) | (45.50%) | (93.20%) |
Permanent differences | 1.80% | 6.90% | 6.40% |
Sarepta International C.V. return to provision | (0.10%) | 62.10% | |
Impact of tax reform, net of valuation allowance | 5.90% | ||
Basis difference in subsidiary | (8.40%) | ||
Foreign rate differential | (7.40%) | (0.90%) | (0.60%) |
Other | 3.40% | 0.40% | |
Effective tax rate | (0.20%) | 0.20% | (4.20%) |
Income Taxes - Additional Infor
Income Taxes - Additional Information (Detail) - USD ($) | 1 Months Ended | 12 Months Ended | |||
Feb. 28, 2019 | Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | Dec. 31, 2016 | |
Income Taxes [Line Items] | |||||
Net change in valuation allowance for deferred tax assets | $ 119,500,000 | $ 164,800,000 | |||
Operating loss carryforwards, valuation allowance | 7,900,000 | ||||
Net operating loss carried forwards | 304,033,000 | 212,342,000 | |||
Accrual for interest or penalties | $ 0 | $ 0 | |||
Recognized interest and/or penalties | $ 0 | $ 0 | |||
Minimum [Member] | |||||
Income Taxes [Line Items] | |||||
Operating loss carryforwards, ending of expiration year | 2020 | ||||
Maximum [Member] | |||||
Income Taxes [Line Items] | |||||
Operating loss carryforwards, ending of expiration year | 2039 | ||||
Federal [Member] | |||||
Income Taxes [Line Items] | |||||
Net operating loss carryforwards | $ 1,166,200,000 | ||||
Net operating loss carryforwards subject to expiration | 579,900,000 | ||||
Net operating loss carried forwards | 586,300,000 | ||||
State [Member] | |||||
Income Taxes [Line Items] | |||||
Net operating loss carryforwards | 859,000,000 | ||||
Net operating loss carryforwards subject to expiration | 807,700,000 | ||||
Net operating loss carried forwards | 51,300,000 | ||||
Foreign [Member] | Sarepta International Holdings GmbH [Member] | |||||
Income Taxes [Line Items] | |||||
Net operating loss carryforwards | $ 8,000,000 | ||||
Research and Development Credits [Member] | |||||
Income Taxes [Line Items] | |||||
Research and development credits carryforward, Description | These federal and state research and development credits begin to expire between 2020 and 2039 and between 2020 and 2034, respectively. | ||||
Research and Development Credits [Member] | Federal [Member] | |||||
Income Taxes [Line Items] | |||||
Research and development credits carryforward, Amount | $ 74,700,000 | ||||
Research and Development Credits [Member] | Federal [Member] | Minimum [Member] | |||||
Income Taxes [Line Items] | |||||
Research and development credits carryforward, Expiration period | 2020 | ||||
Research and Development Credits [Member] | Federal [Member] | Maximum [Member] | |||||
Income Taxes [Line Items] | |||||
Research and development credits carryforward, Expiration period | 2039 | ||||
Research and Development Credits [Member] | State [Member] | |||||
Income Taxes [Line Items] | |||||
Research and development credits carryforward, Amount | $ 34,100,000 | ||||
Research and Development Credits [Member] | State [Member] | Minimum [Member] | |||||
Income Taxes [Line Items] | |||||
Research and development credits carryforward, Expiration period | 2020 | ||||
Research and Development Credits [Member] | State [Member] | Maximum [Member] | |||||
Income Taxes [Line Items] | |||||
Research and development credits carryforward, Expiration period | 2034 | ||||
Warrant Agreement | Myonexus Therapeutics, Inc. [Member] | |||||
Income Taxes [Line Items] | |||||
Accumulated costs related to acquisition | $ 253,700,000 | ||||
Up-front and milestone payments | 85,000,000 | ||||
Exercise option | $ 168,700,000 |
Analysis of Deferred Tax Assets
Analysis of Deferred Tax Assets and Liabilities (Detail) - USD ($) $ in Thousands | Dec. 31, 2019 | Dec. 31, 2018 |
Deferred tax assets: | ||
Net operating loss carryforwards | $ 304,033 | $ 212,342 |
Difference in depreciation and amortization | 40,095 | 55,162 |
Research and development tax credits | 103,806 | 67,309 |
Stock-based compensation | 24,114 | 15,538 |
Lease liabilities | 15,796 | 4,831 |
Deferred revenue | 939 | 888 |
Capitalized inventory | 18,255 | 22,943 |
Other | 16,270 | 15,727 |
Total deferred tax assets | 523,308 | 394,740 |
Deferred tax liabilities: | ||
Right of use asset | (10,782) | |
Debt discount | (23,099) | (25,162) |
Total deferred tax liabilities | (33,881) | (25,162) |
Valuation allowance | (488,829) | (369,345) |
Net deferred tax assets | $ 598 | $ 233 |
Reconciliation of Beginning and
Reconciliation of Beginning and Ending Amount of Unrecognized Tax Benefits (Detail) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Income Tax Disclosure [Abstract] | |||
Balance at beginning of the period | $ 37,544 | $ 5,134 | $ 4,644 |
Increase related to current year tax positions | 4,275 | 2,164 | 735 |
Increase related to prior year tax positions | 109 | 30,246 | |
Decrease related to prior year tax positions | (175) | (245) | |
Balance at end of the period | $ 41,753 | $ 37,544 | $ 5,134 |
Leases - Additional Information
Leases - Additional Information (Detail) - USD ($) $ in Thousands | Dec. 31, 2019 | Jan. 01, 2019 |
Lessee Lease Description [Line Items] | ||
Operating lease liabilities | $ 55,566 | $ 60,100 |
Right-of-use assets | 37,933 | $ 42,500 |
ASU No. 2016-02 [Member] | ||
Lessee Lease Description [Line Items] | ||
Operating lease liabilities | 60,100 | |
Right-of-use assets | 42,500 | |
ASU No. 2016-02 [Member] | Restatement Adjustment [Member] | ||
Lessee Lease Description [Line Items] | ||
De-recognition of deferred rent to reduce right-of-use assets | $ (18,000) |
Summary of Lease Costs Recogniz
Summary of Lease Costs Recognized Under Topic 842 and Other Information Pertaining to Operating Leases (Detail) $ in Thousands | 12 Months Ended |
Dec. 31, 2019USD ($) | |
Lease cost | |
Operating lease cost | $ 10,335 |
Variable lease cost | 3,967 |
Total lease cost | 14,302 |
Other information | |
Operating lease payments | $ 10,416 |
Weighted average remaining lease term | 5 years 6 months |
Weighted average discount rate | 7.50% |
Summary of Maturities of Lease
Summary of Maturities of Lease Liabilities and Reconciliation of Lease Liabilities Recognized Under Topic 842 (Detail) - USD ($) $ in Thousands | Dec. 31, 2019 | Jan. 01, 2019 |
Maturity of lease liability | ||
2020 | $ 11,718 | |
2021 | 12,891 | |
2022 | 11,080 | |
2023 | 11,230 | |
2024 | 11,471 | |
Thereafter | 9,654 | |
Total minimum lease payments | 68,044 | |
Less: imputed interest | (12,478) | |
Total operating lease liabilities | 55,566 | $ 60,100 |
Included in the condensed consolidated balance sheet: | ||
Current portion of lease liabilities within other current liabilities | $ 7,846 | |
Operating Lease, Liability, Current, Statement of Financial Position [Extensible List] | us-gaap:OtherLiabilitiesCurrent | |
Lease liabilities | $ 47,720 | |
Total operating lease liabilities | $ 55,566 | $ 60,100 |
Summary of Aggregate Future Min
Summary of Aggregate Future Minimum Non-Cancellable Commitments under Leases (Detail) $ in Thousands | Dec. 31, 2018USD ($) |
Maturity of lease liability | |
2020 | $ 11,395 |
2021 | 12,558 |
2022 | 10,757 |
2023 | 10,898 |
2024 | 11,128 |
Thereafter | 9,396 |
Total minimum lease payments | $ 66,132 |
Basic and Diluted Net Loss Per
Basic and Diluted Net Loss Per Share (Detail) - USD ($) $ / shares in Units, shares in Thousands, $ in Thousands | 3 Months Ended | 12 Months Ended | |||||||||
Dec. 31, 2019 | Sep. 30, 2019 | Jun. 30, 2019 | Mar. 31, 2019 | Dec. 31, 2018 | Sep. 30, 2018 | Jun. 30, 2018 | Mar. 31, 2018 | Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Earnings Per Share [Abstract] | |||||||||||
Net loss | $ (235,703) | $ (126,326) | $ (276,403) | $ (76,643) | $ (140,895) | $ (76,393) | $ (109,267) | $ (35,363) | $ (715,075) | $ (361,918) | $ (50,688) |
Weighted-average common shares outstanding - basic | 73,615 | 66,250 | 58,818 | ||||||||
Weighted-average common shares outstanding - diluted | 73,615 | 66,250 | 58,818 | ||||||||
Net loss per share — basic and diluted | $ (3.16) | $ (1.70) | $ (3.74) | $ (1.07) | $ (2.05) | $ (1.15) | $ (1.67) | $ (0.55) | $ (9.71) | $ (5.46) | $ (0.86) |
Basic and Diluted Net Loss Pe_2
Basic and Diluted Net Loss Per Share (Parenthetical) (Detail) - $ / shares shares in Millions | 12 Months Ended | |||
Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | Nov. 14, 2017 | |
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | ||||
Anti-dilutive securities excluded from computation of net loss per share | 9.1 | 9.1 | 9.4 | |
2024 Convertible Notes [Member] | ||||
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | ||||
Debt instrument, conversion price | $ 73.42 | $ 73.42 | ||
Effect on diluted net earnings (loss) per share | $ 0 |
Commitments and Contingencies -
Commitments and Contingencies - Additional Information (Detail) $ in Millions | 1 Months Ended | 12 Months Ended | |||||
May 31, 2019USD ($) | Oct. 31, 2018Suite | Jun. 30, 2018USD ($)PaymentMonthQuarter | Dec. 31, 2019USD ($) | Dec. 31, 2018USD ($) | Dec. 31, 2017USD ($) | Sep. 30, 2018USD ($) | |
CRO [Member] | |||||||
Commitments And Contingencies [Line Items] | |||||||
Cancellable future commitments | $ 91.4 | ||||||
CRO [Member] | Other Funding Commitments [Member] | |||||||
Commitments And Contingencies [Line Items] | |||||||
Expenditures incurred by CROs | 31.6 | $ 19.6 | $ 13.9 | ||||
Manufacturing Agreement [Member] | Brammer [Member] | |||||||
Commitments And Contingencies [Line Items] | |||||||
Advance upfront fee to be paid on execution of agreements | $ 20 | ||||||
Number of non-refundable process equipment fee payments | Payment | 2 | ||||||
Required non refundable payments at each time of procurement of equipment | $ 5 | $ 5 | |||||
Quarterly capacity access fee payments | $ 10 | ||||||
Manufacturing Agreement [Member] | Brammer [Member] | |||||||
Commitments And Contingencies [Line Items] | |||||||
Advance upfront fee to be paid on execution of agreements | $ 6 | ||||||
Quarterly capacity access fee payments | $ 13.3 | $ 10 | |||||
Supply agreement term | 6 years | ||||||
Successive renewal term of supply agreement | 2 years | ||||||
Minimum number of months prior to expiration of term for renewal | Month | 24 | ||||||
Maximum required additional quarters for remittance of capacity access fees before terminate agreement | Quarter | 8 | ||||||
Cash payment under agreements | 75.5 | ||||||
Manufacturing Collaboration Agreement [Member] | Paragon [Member] | |||||||
Commitments And Contingencies [Line Items] | |||||||
Number of dedicated clean room suites | Suite | 2 | ||||||
Number of additional clean room suites available on optional reserve | Suite | 2 | ||||||
Agreement expiration date | Dec. 31, 2024 | ||||||
Usage fees per annum per clean room suite | 1 | ||||||
Manufacturing Collaboration Agreement [Member] | Paragon [Member] | Other Noncurrent Assets [Member] | |||||||
Commitments And Contingencies [Line Items] | |||||||
Clean room suite reservation fees payments | 40.1 | ||||||
Manufacturing Collaboration Agreement [Member] | Paragon [Member] | Maximum [Member] | |||||||
Commitments And Contingencies [Line Items] | |||||||
Clean room suite reservation fees | $ 48 | ||||||
Percentage of credit on clean room use fee if threshold met | 100.00% | ||||||
Manufacturing Collaboration Agreement [Member] | |||||||
Commitments And Contingencies [Line Items] | |||||||
Pre-launch period purchase obligation, minimum amount per quarter per clean room | $ 4 | ||||||
Clinical and Commercial Supply Agreement [Member] | Aldevron LLC [Member] | |||||||
Commitments And Contingencies [Line Items] | |||||||
Agreement expiration date | Dec. 31, 2019 | ||||||
Agreement expiration description | The Company exercised the option to extend the CCSA to December 31, 2020 (the “2020 Option”) and has another option to extend the term of the CCSA for an additional year to December 31, 2021 (the “2021 Renewal Right”). | ||||||
Percentage of prepayments credited back equal to batch invoice amount | 50.00% | ||||||
Clinical and Commercial Supply Agreement [Member] | Aldevron LLC [Member] | Other Current Assets [Member] | |||||||
Commitments And Contingencies [Line Items] | |||||||
Prepayments to extend agreement term | $ 22.8 | ||||||
Clinical and Commercial Supply Agreement [Member] | Aldevron LLC [Member] | Maximum [Member] | |||||||
Commitments And Contingencies [Line Items] | |||||||
Advance upfront fee to be paid on execution of agreements | $ 20 |
Summary of Non-Cancelable Contr
Summary of Non-Cancelable Contractual Obligations Arising From Long-term Contractual Arrangements (Detail) $ in Thousands | Dec. 31, 2019USD ($) |
Commitments And Contingencies Disclosure [Abstract] | |
2020 | $ 378,744 |
2021 | 183,661 |
2022 | 64,653 |
2023 | 58,319 |
2024 | 58,309 |
Thereafter | 149,350 |
Total manufacturing commitments | $ 893,036 |
Subsequent Event - Additional I
Subsequent Event - Additional Information (Details) $ in Millions | Feb. 14, 2020USD ($) |
Subsequent Event | F D A | |
Subsequent Event [Line Items] | |
Agreement to sell | $ 111 |
Financial Information by Quar_3
Financial Information by Quarter (Detail) - USD ($) $ / shares in Units, $ in Thousands | 3 Months Ended | 12 Months Ended | |||||||||
Dec. 31, 2019 | Sep. 30, 2019 | Jun. 30, 2019 | Mar. 31, 2019 | Dec. 31, 2018 | Sep. 30, 2018 | Jun. 30, 2018 | Mar. 31, 2018 | Dec. 31, 2019 | Dec. 31, 2018 | Dec. 31, 2017 | |
Revenues: | |||||||||||
Product, net | $ 100,113 | $ 99,041 | $ 94,668 | $ 87,011 | $ 84,415 | $ 78,486 | $ 73,529 | $ 64,604 | $ 380,833 | $ 301,034 | $ 154,584 |
Type of Revenue [Extensible List] | us-gaap:ProductMember | us-gaap:ProductMember | us-gaap:ProductMember | us-gaap:ProductMember | us-gaap:ProductMember | us-gaap:ProductMember | us-gaap:ProductMember | us-gaap:ProductMember | us-gaap:ProductMember | us-gaap:ProductMember | us-gaap:ProductMember |
Total revenues | $ 100,113 | $ 99,041 | $ 94,668 | $ 87,011 | $ 84,415 | $ 78,486 | $ 73,529 | $ 64,604 | $ 380,833 | $ 301,034 | $ 154,584 |
Cost and expenses: | |||||||||||
Cost of sales (excluding amortization of in-licensed rights) | 15,567 | 13,037 | 15,919 | 12,063 | 13,135 | 8,741 | 6,735 | 5,582 | 56,586 | 34,193 | 7,353 |
Research and development expense | 223,141 | 133,949 | 113,266 | 90,553 | 146,207 | 86,584 | 122,848 | 46,204 | |||
Selling, general and administrative | 81,424 | 75,429 | 67,393 | 60,566 | 64,220 | 53,044 | 47,156 | 43,341 | 284,812 | 207,761 | 122,682 |
Acquired in-process research and development | 173,240 | 173,240 | |||||||||
Settlement and license charges | 10,000 | 10,000 | 28,427 | ||||||||
Amortization of in-licensed rights | 200 | 216 | 217 | 216 | 216 | 216 | 217 | 216 | 849 | 865 | 1,053 |
Total cost and expenses | 330,332 | 222,631 | 370,035 | 163,398 | 223,778 | 148,585 | 176,956 | 95,343 | 1,086,396 | 644,662 | 326,222 |
Operating loss | (230,219) | (123,590) | (275,367) | (76,387) | (139,363) | (70,099) | (103,427) | (30,739) | (705,563) | (343,628) | (171,638) |
Other (loss) income: | |||||||||||
Other expense, net | (4,773) | (2,510) | (862) | (172) | |||||||
Total other (loss) income | (4,773) | (2,510) | (862) | (172) | (2,311) | (6,968) | (5,218) | (4,485) | (8,317) | (18,982) | 123,010 |
Interest expense and other, net | (2,311) | (6,968) | (5,218) | (4,485) | |||||||
Loss before income tax expense (benefit) | (234,992) | (126,100) | (276,229) | (76,559) | (141,674) | (77,067) | (108,645) | (35,224) | (713,880) | (362,610) | (48,628) |
Income tax expense (benefit) | 711 | 226 | 174 | 84 | (779) | (674) | 622 | 139 | 1,195 | (692) | 2,060 |
Gain from sale of Priority Review Voucher | 125,000 | ||||||||||
Net loss | $ (235,703) | $ (126,326) | $ (276,403) | $ (76,643) | $ (140,895) | $ (76,393) | $ (109,267) | $ (35,363) | $ (715,075) | $ (361,918) | $ (50,688) |
Net loss per share — basic and diluted | $ (3.16) | $ (1.70) | $ (3.74) | $ (1.07) | $ (2.05) | $ (1.15) | $ (1.67) | $ (0.55) | $ (9.71) | $ (5.46) | $ (0.86) |
Weighted average number of shares of common stock used in computing basic and diluted net loss per share | 74,557 | 74,177 | 73,958 | 71,731 | 68,653 | 66,209 | 65,484 | 64,631 | 73,615,000 | 66,250,000 | 58,818,000 |