Document and Entity Information
Document and Entity Information - shares | 9 Months Ended | |
Sep. 30, 2021 | Oct. 29, 2021 | |
Cover [Abstract] | ||
Document Type | 10-Q | |
Amendment Flag | false | |
Document Period End Date | Sep. 30, 2021 | |
Document Fiscal Year Focus | 2021 | |
Document Fiscal Period Focus | Q3 | |
Trading Symbol | SRPT | |
Entity Registrant Name | Sarepta Therapeutics, Inc. | |
Entity Central Index Key | 0000873303 | |
Current Fiscal Year End Date | --12-31 | |
Entity Filer Category | Large Accelerated Filer | |
Entity Small Business | false | |
Entity Emerging Growth Company | false | |
Entity Common Stock, Shares Outstanding | 87,076,918 | |
Entity Current Reporting Status | Yes | |
Entity Shell Company | false | |
Entity File Number | 001-14895 | |
Entity Tax Identification Number | 93-0797222 | |
Entity Address, Address Line One | 215 First Street | |
Entity Address, Address Line Two | Suite 415 | |
Entity Address, State or Province | MA | |
Entity Address, City or Town | Cambridge | |
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, $0.0001 par value per share | |
Security Exchange Name | NASDAQ | |
Entity Incorporation, State or Country Code | DE | |
Document Quarterly Report | true | |
Document Transition Report | false |
Condensed Consolidated Balance
Condensed Consolidated Balance Sheets - USD ($) $ in Thousands | Sep. 30, 2021 | Dec. 31, 2020 |
Current assets: | ||
Cash and cash equivalents | $ 1,599,113 | $ 1,502,648 |
Short-term investments | 0 | 435,923 |
Accounts receivable | 149,787 | 101,340 |
Inventory | 288,469 | 231,961 |
Other current assets | 147,941 | 213,324 |
Total current assets | 2,185,310 | 2,485,196 |
Property and equipment, net | 199,249 | 190,430 |
Intangible assets, net | 14,204 | 13,628 |
Right of use assets | 72,663 | 91,761 |
Other non-current assets | 190,792 | 203,703 |
Total assets | 2,662,218 | 2,984,718 |
Current liabilities: | ||
Accounts payable | 44,163 | 111,090 |
Accrued expenses | 221,009 | 193,553 |
Deferred revenue, current portion | 89,244 | 89,244 |
Other current liabilities | 19,027 | 22,139 |
Total current liabilities | 373,443 | 416,026 |
Long-term debt | 1,094,912 | 992,493 |
Lease liabilities, net of current portion | 63,428 | 80,367 |
Deferred revenue, net of current portion | 596,738 | 663,488 |
Contingent consideration | 43,600 | 50,800 |
Other non-current liabilities | 20,569 | 19,785 |
Total liabilities | 2,192,690 | 2,222,959 |
Commitments and contingencies (Note 16) | ||
Stockholders’ equity: | ||
Preferred stock, $0.0001 par value, 3,333,333 shares authorized; none issued and outstanding | ||
Common stock, $0.0001 par value, 198,000,000 shares authorized; 79,958,527 and 79,374,247 issued and outstanding at September 30, 2021, and December 31, 2020, respectively | 8 | 8 |
Additional paid-in capital | 3,554,307 | 3,609,877 |
Accumulated other comprehensive (loss) income, net of tax | (20) | 3 |
Accumulated deficit | (3,084,767) | (2,848,129) |
Total stockholders’ equity | 469,528 | 761,759 |
Total liabilities and stockholders’ equity | $ 2,662,218 | $ 2,984,718 |
Condensed Consolidated Balanc_2
Condensed Consolidated Balance Sheets (Parenthetical) - $ / shares | Sep. 30, 2021 | Dec. 31, 2020 |
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 | 198,000,000 | 198,000,000 |
Common stock, issued | 79,958,527 | 79,374,247 |
Common stock, outstanding | 79,958,527 | 79,374,247 |
Condensed Consolidated Statemen
Condensed Consolidated Statements of Operations and Comprehensive Loss - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2021 | Sep. 30, 2020 | Sep. 30, 2021 | Sep. 30, 2020 | |
Revenues: | ||||
Products, net | $ 166,911 | $ 121,429 | $ 433,676 | $ 333,221 |
Type of Revenue [Extensible List] | us-gaap:ProductMember | us-gaap:ProductMember | us-gaap:ProductMember | us-gaap:ProductMember |
Collaboration | $ 22,495 | $ 22,495 | $ 66,750 | $ 61,740 |
Total revenues | 189,406 | 143,924 | 500,426 | 394,961 |
Cost and expenses: | ||||
Cost of sales (excluding amortization of in-licensed rights) | 23,444 | 15,015 | 65,305 | 40,978 |
Research and development | 139,115 | 190,438 | 573,886 | 515,104 |
Selling, general and administrative | 61,127 | 75,373 | 204,605 | 231,829 |
Settlement and license charges | 10,000 | |||
Amortization of in-licensed rights | 178 | 166 | 527 | 497 |
Total cost and expenses | 223,864 | 280,992 | 854,323 | 788,408 |
Operating loss | (34,458) | (137,068) | (353,897) | (393,447) |
Other (loss) income: | ||||
Gain (loss) on contingent consideration, net | 7,200 | (45,000) | 7,200 | (45,000) |
Other expense, net | (20,649) | (14,335) | (52,362) | (34,202) |
Gain from sale of Priority Review Voucher | 102,000 | 108,069 | ||
Total other (loss) income | (13,449) | (59,335) | 56,838 | 28,867 |
Loss before income tax expense (benefit) | (47,907) | (196,403) | (297,059) | (364,580) |
Income tax expense (benefit) | 237 | 96 | (260) | 231 |
Net loss | (48,144) | (196,499) | (296,799) | (364,811) |
Other comprehensive loss: | ||||
Unrealized losses on investments, net of tax | (22) | (12) | (23) | (56) |
Total other comprehensive loss | (22) | (12) | (23) | (56) |
Comprehensive loss | $ (48,166) | $ (196,511) | $ (296,822) | $ (364,867) |
Net loss per share - basic and diluted | $ (0.60) | $ (2.50) | $ (3.72) | $ (4.70) |
Weighted average number of shares of common stock used in computing basic and diluted net loss per share | 79,880 | 78,501 | 79,695 | 77,637 |
Condensed Consolidated Statem_2
Condensed Consolidated Statements of Stockholders' Equity - USD ($) $ in Thousands | Total | Cumulative Effect of Accounting Change [Member] | Common Stock [Member] | Additional Paid-In Capital [Member] | Additional Paid-In Capital [Member]Cumulative Effect of Accounting Change [Member] | Accumulated Other Comprehensive (Loss) Income [Member] | Accumulated Deficit [Member] | Accumulated Deficit [Member]Cumulative Effect of Accounting Change [Member] |
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 | |||||||
Exercise of options for common stock | 3,687 | 3,687 | ||||||
Exercise of options for common stock, shares | 97,000 | |||||||
Vest of restricted stock units | 98,000 | |||||||
Issuance of common stock to Roche, net of issuance costs | 312,053 | 312,053 | ||||||
Issuance of common stock to Roche, net of issuance costs, shares | 2,522,000 | |||||||
Issuance of common stock under employee stock purchase plan | 3,795 | 3,795 | ||||||
Issuance of common stock under employee stock purchase plan, shares | 56,000 | |||||||
Stock-based compensation | 24,024 | 24,024 | ||||||
Unrealized gains (losses) from available-for-sale securities, net of tax | 574 | 574 | ||||||
Net loss | (17,492) | 17,492 | ||||||
Balance at Mar. 31, 2020 | 1,144,828 | $ 8 | 3,455,689 | 624 | (2,311,493) | |||
Balance (in shares) at Mar. 31, 2020 | 77,958,000 | |||||||
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 | |||||||
Net loss | (364,811) | |||||||
Balance at Sep. 30, 2020 | 892,047 | $ 8 | 3,550,857 | (6) | (2,658,812) | |||
Balance (in shares) at Sep. 30, 2020 | 78,790,000 | |||||||
Balance at Mar. 31, 2020 | 1,144,828 | $ 8 | 3,455,689 | 624 | (2,311,493) | |||
Balance (in shares) at Mar. 31, 2020 | 77,958,000 | |||||||
Exercise of options for common stock | 21,860 | 21,860 | ||||||
Exercise of options for common stock, shares | 449,000 | |||||||
Vest of restricted stock units | 25,000 | |||||||
Stock-based compensation | 27,616 | 27,616 | ||||||
Unrealized gains (losses) from available-for-sale securities, net of tax | (618) | 618 | ||||||
Net loss | (150,820) | 150,820 | ||||||
Balance at Jun. 30, 2020 | 1,042,866 | $ 8 | 3,505,165 | 6 | (2,462,313) | |||
Balance (in shares) at Jun. 30, 2020 | 78,432,000 | |||||||
Exercise of options for common stock, shares | 292,000 | |||||||
Vest of restricted stock units | 20,000 | |||||||
Issuance of common stock under employee stock purchase plan | 3,670 | 3,670 | ||||||
Issuance of common stock under employee stock purchase plan, shares | 46,000 | |||||||
Stock-based compensation | 26,903 | 26,903 | ||||||
Unrealized gains (losses) from available-for-sale securities, net of tax | (12) | 12 | ||||||
Net loss | (196,499) | 196,499 | ||||||
Balance at Sep. 30, 2020 | 892,047 | $ 8 | 3,550,857 | (6) | (2,658,812) | |||
Balance (in shares) at Sep. 30, 2020 | 78,790,000 | |||||||
Balance at Dec. 31, 2020 | 761,759 | $ (96,792) | $ 8 | 3,609,877 | $ (156,953) | 3 | (2,848,129) | $ 60,161 |
Balance (in shares) at Dec. 31, 2020 | 79,374,000 | |||||||
Exercise of options for common stock | 4,683 | 4,683 | ||||||
Exercise of options for common stock, shares | 108,000 | |||||||
Vest of restricted stock units | 204,000 | |||||||
Issuance of common stock under employee stock purchase plan | 4,543 | 4,543 | ||||||
Issuance of common stock under employee stock purchase plan, shares | 62,000 | |||||||
Stock-based compensation | 28,508 | 28,508 | ||||||
Unrealized gains (losses) from available-for-sale securities, net of tax | (6) | (6) | ||||||
Net loss | (167,250) | (167,250) | ||||||
Balance at Mar. 31, 2021 | 535,445 | $ 8 | 3,490,658 | (3) | (2,955,218) | |||
Balance (in shares) at Mar. 31, 2021 | 79,748,000 | |||||||
Balance at Dec. 31, 2020 | 761,759 | $ (96,792) | $ 8 | 3,609,877 | $ (156,953) | 3 | (2,848,129) | $ 60,161 |
Balance (in shares) at Dec. 31, 2020 | 79,374,000 | |||||||
Net loss | (296,799) | |||||||
Balance at Sep. 30, 2021 | 469,528 | $ 8 | 3,554,307 | (20) | (3,084,767) | |||
Balance (in shares) at Sep. 30, 2021 | 79,959 | |||||||
Balance at Mar. 31, 2021 | 535,445 | $ 8 | 3,490,658 | (3) | (2,955,218) | |||
Balance (in shares) at Mar. 31, 2021 | 79,748,000 | |||||||
Exercise of options for common stock | 3,526 | 3,526 | ||||||
Exercise of options for common stock, shares | 72,000 | |||||||
Vest of restricted stock units | 28,000 | |||||||
Shares withheld for taxes | (1,432) | (1,432) | ||||||
Shares withheld for taxes, shares | (18,000) | |||||||
Stock-based compensation | 28,969 | 28,969 | ||||||
Unrealized gains (losses) from available-for-sale securities, net of tax | 5 | 5 | ||||||
Net loss | (81,405) | (81,405) | ||||||
Balance at Jun. 30, 2021 | 485,108 | $ 8 | 3,521,721 | 2 | (3,036,623) | |||
Balance (in shares) at Jun. 30, 2021 | 79,830,000 | |||||||
Exercise of options for common stock | 2,606 | 2,606 | ||||||
Exercise of options for common stock, shares | 52,000 | |||||||
Vest of restricted stock units | 27,000 | |||||||
Issuance of common stock under employee stock purchase plan | 3,296 | 3,296 | ||||||
Issuance of common stock under employee stock purchase plan, shares | 50,000 | |||||||
Stock-based compensation | 26,684 | 26,684 | ||||||
Unrealized gains (losses) from available-for-sale securities, net of tax | (22) | (22) | ||||||
Net loss | (48,144) | (48,144) | ||||||
Balance at Sep. 30, 2021 | $ 469,528 | $ 8 | $ 3,554,307 | $ (20) | $ (3,084,767) | |||
Balance (in shares) at Sep. 30, 2021 | 79,959 |
Condensed Consolidated Statem_3
Condensed Consolidated Statements of Cash Flows - USD ($) $ in Thousands | 9 Months Ended | |
Sep. 30, 2021 | Sep. 30, 2020 | |
Cash flows from operating activities: | ||
Net loss | $ (296,799) | $ (364,811) |
Adjustments to reconcile net loss to cash flows from operating activities: | ||
(Gain) loss on contingent consideration, net | (7,200) | 45,000 |
Gain from sale of Priority Review Voucher, net of commission | (102,000) | (108,069) |
Depreciation and amortization | 27,872 | 19,623 |
Reduction in the carrying amounts of the right of use assets | 10,112 | 8,749 |
Non-cash interest expense | 5,617 | 18,532 |
Stock-based compensation | 84,161 | 78,543 |
Impairment of equity investment | 4,488 | |
Other | 3,964 | (2,031) |
Changes in operating assets and liabilities, net: | ||
Net increase in accounts receivable | (48,447) | (30,948) |
Net increase in inventory | (56,508) | (48,739) |
Net decrease (increase) in other assets | 74,155 | (114,127) |
Net (decrease) increase in deferred revenue | (66,750) | 771,923 |
Net (decrease) increase in accounts payable, accrued expenses and other liabilities | (46,511) | 8,356 |
Net cash (used in) provided by operating activities | (413,846) | 282,001 |
Cash flows from investing activities: | ||
Purchase of property and equipment | (36,284) | (53,592) |
Proceeds from sale of Priority Review Voucher, net of commission | 102,000 | 108,069 |
Purchase of available-for-sale securities | (29,988) | (897,691) |
Maturity and sale of available-for-sale securities | 466,000 | 847,980 |
Other | (3,738) | (2,596) |
Net cash provided by investing activities | 497,990 | 2,170 |
Cash flows from financing activities: | ||
Proceeds from exercise of stock options and purchase of stock under the Employee Stock Purchase Program | 18,654 | 48,131 |
Taxes paid related to net share settlement of equity awards | (6,333) | (4,798) |
Net cash provided by financing activities | 12,321 | 355,386 |
Increase in cash, cash equivalents and restricted cash | 96,465 | 639,557 |
Cash, cash equivalents and restricted cash: | ||
Beginning of period | 1,511,713 | 843,645 |
End of period | 1,608,178 | 1,483,202 |
Reconciliation of cash, cash equivalents and restricted cash: | ||
Cash and cash equivalents | 1,599,113 | 1,474,637 |
Restricted cash in other assets | $ 9,065 | $ 8,565 |
Restricted Cash, Noncurrent, Statement of Financial Position [Extensible Enumeration] | us-gaap:OtherNoncurrentAssetsMember | us-gaap:OtherNoncurrentAssetsMember |
Total cash, cash equivalents and restricted cash | $ 1,608,178 | $ 1,483,202 |
Supplemental disclosure of cash flow information: | ||
Cash paid during the period for interest | 39,727 | 20,462 |
Supplemental schedule of non-cash investing activities and financing activities: | ||
Intangible assets and property and equipment included in accounts payable and accrued expenses | 4,644 | 8,292 |
Shares withheld for tax included in accrued expenses | 1,432 | 0 |
Lease liabilities arising from obtaining right of use assets | 13,148 | 37,647 |
Lease liabilities terminated | $ 19,967 | 0 |
Roche | ||
Cash flows from financing activities: | ||
Proceeds from issuance of common stock to Roche, net of offering costs | $ 312,053 |
ORGANIZATION AND NATURE OF BUSI
ORGANIZATION AND NATURE OF BUSINESS | 9 Months Ended |
Sep. 30, 2021 | |
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 (“Duchenne”), Limb-girdle muscular dystrophies (“LGMDs”), and other neuromuscular and central nervous system (“CNS”) related disorders. Its products in the U.S., EXONDYS 51 (eteplirsen) Injection (“EXONDYS 51”), VYONDYS 53 (golodirsen) Injection (“VYONDYS 53”) and AMONDYS 45 (casimersen) Injection (“AMONDYS 45”), were granted accelerated approval by the U.S. Food and Drug Administration (the “FDA”) on September 19, 2016, December 12, 2019 and February 25, 2021, respectively. Indicated for the treatment of Duchenne in patients who have a confirmed mutation of the dystrophin gene that is amenable to exon 51, exon 53 and exon 45 skipping, respectively, EXONDYS 51, VYONDYS 53 and AMONDYS 45 use the Company’s phosphorodiamidate morpholino oligomer (“PMO”) chemistry and exon-skipping technology to skip exon 51, exon 53 and exon 45 of the dystrophin gene. Exon skipping is intended to promote the production of an internally truncated but functional dystrophin protein. As of September 30, 2021, the Company had approximately $ 1,608.4 million of cash, cash equivalents and investments, consisting of $ 1,599.1 million of cash and cash equivalents and $ 9.3 million of long-term restricted cash and investments. The Company believes that its balance of cash, cash equivalents and investments as of the date of the issuance of this report is sufficient to fund its current operational plan for at least the next twelve months, though it may pursue raising additional cash resources through public or private debt and equity financings, seek funded research and development arrangements and 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 | 9 Months Ended |
Sep. 30, 2021 | |
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 unaudited condensed consolidated financial statements, which have been prepared in accordance with accounting principles generally accepted in the United States (“U.S. GAAP”), reflect the accounts of Sarepta 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: discovering, developing, manufacturing and delivering therapies to patients with rare diseases. In the opinion of the Company’s management, all adjustments of a normal recurring nature necessary for a fair presentation have been reflected. Certain financial information that is normally included in annual financial statements prepared in accordance with the U.S. GAAP, but that is not required for interim reporting purposes, has been omitted. These unaudited condensed consolidated financial statements should be read in conjunction with the consolidated financial statements and related notes for the year ended December 31, 2020 which are contained in the Company’s Annual Report on Form 10-K for the year ended December 31, 2020, filed with the U.S. Securities and Exchange Commission on March 1, 2021. The results for the three and nine months ended September 30, 2021 are not necessarily indicative of the results to be expected for the full year. Estimates and Uncertainties The preparation of the unaudited condensed 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. Concentration of Credit Risk Financial instruments that potentially subject the Company to concentrations of credit risk consist of accounts receivable from customers, cash held at financial institutions and cash equivalents and investments. As of September 30, 2021 , the majority of the Company’s accounts receivable arose from product sales in the U.S. and all customers have standard payment terms that generally require payment within 60 to 91 days . Outside of the U.S., the majority of the Company’s customers have payment terms ranging between 45 and 150 days . Three individual c ustomers accounted for 45 %, 39 % and 11 % of net product revenues for the three months ended September 30, 2021 and 47 %, 40 % and 10 % of net pr oduct revenues for the nine months ended September 30, 2021. Three individual customers accounted for 47 %, 38 % and 10 % of product revenues for the three months ended September 30, 2020 and 47 %, 39 % and 11 % of net product revenues for the nine months ended September 30, 2020. Three individual customers accounted for 44 %, 39 % and 10 % of accounts receivable from product sales as of September 30, 2021 and 45 %, 41 % and 9 % of accounts receivable from product sales as of December 31, 2020. The Company monitors the financial performance and creditworthiness of its customers so that it can properly assess and respond to changes in its customers’ credit profile. As of September 30, 2021, the Company believes that such customers are of high credit quality. As of September 30, 2021, the Company’s cash was concentrated at three financial institutions in the U.S., which potentially exposes the Company to credit risks. However, the Company does not believe that there is significant risk of non-performance by the financial institutions. Significant Accounting Policies For details about the Company’s accounting policies, please read Note 2, Summary of Significant Accounting Policies and Recent Accounting Pronouncements of the Annual Report on Form 10-K for the year ended December 31, 2020. There have not been any material changes to the Company’s accounting policies through September 30, 2021, other than those noted below relating to the adoption of ASU 2020-06, “ Debt - Debt with Conversion and Other Options (Subtopic 470-20) and Derivatives and Hedging - Contracts in Entity's Own Equity (Subtopic 815-40): Accounting for Convertible Instruments and Contracts in an Entity's Own Equity .” Recent Accounting Pronouncements Recently adopted In August 2020, the Financial Accounting Standards Board (“FASB”) issued ASU 2020-06, “ Debt - Debt with Conversion and Other Options (Subtopic 470-20) and Derivatives and Hedging - Contracts in Entity's Own Equity (Subtopic 815-40): Accounting for Convertible Instruments and Contracts in an Entity's Own Equity .” This ASU simplifies the complexity associated with applying U.S. GAAP for certain financial instruments with characteristics of liabilities and equity. More specifically, the amendments focus on the guidance for convertible instruments and derivative scope exceptions for contracts in an entity’s own equity. Under ASU 2020-06, the embedded conversion features are no longer separated from the host contract for convertible instruments with conversion features that are not required to be accounted for as derivatives under Topic 815, or that do not result in substantial premiums accounted for as paid-in capital. Consequently, a convertible debt instrument, such as the Company’s senior notes due on November 15, 2024 (the “2024 Notes”), will be accounted for as a single liability measured at its amortized cost, as long as no other features require bifurcation and recognition as derivatives. The new guidance also requires the if-converted method to be applied for all convertible instruments and requires additional disclosures. This guidance is required to be adopted by January 1, 2022, and early adoption is permitted, but no earlier than fiscal years beginning after December 15, 2020. The Company elected to early adopt this guidance on January 1, 2021, using the modified retrospective method. Under this transition method, the cumulative effect of the accounting change removed the impact of recognizing the equity component of the Company’s convertible notes (at issuance and the subsequent accounting impact of additional interest expense from debt discount amortization). The cumulative effect of the accounting change as of January 1, 2021 increased the carrying amount of the convertible notes by $ 96.8 million, reduced accumulated deficit by $ 60.2 million and reduced additional paid-in capital by $ 157.0 million. Interest expense of the 2024 Notes will be lower as a result of adoption of this guidance. The if-converted method for such instruments will be used to compute diluted net earnings per share if and when profitability is achieved. As a result of the adoption of this guidance, interest expense and net loss was reduced by $ 5.6 million and $ 16.6 million, or $ 0.07 and $ 0.21 per share, respectively, for the three and nine months ended September 30, 2021. In December 2019, the FASB issued ASU 2019-12, “ Income Taxes (Topic 740): Simplifying the Accounting for Income Taxes”, which is intended to simplify the accounting for income taxes. This ASU removes certain exceptions to the general principles in Topic 740 and clarifies and amends existing guidance to improve consistent application. The new guidance was effective beginning January 1, 2021. The adoption of this guidance did not have a material effect on the Company’s condensed consolidated financial statements. |
LICENSE AND COLLABORATION AGREE
LICENSE AND COLLABORATION AGREEMENTS | 9 Months Ended |
Sep. 30, 2021 | |
Organization Consolidation And Presentation Of Financial Statements [Abstract] | |
LICENSE AND COLLABORATION AGREEMENTS | 3. LICENSE AND COLLABORATION AGREEMENTS F. Hoffman-La Roche Ltd. For the three and nine months ended September 30, 2021, the Company recogniz ed $ 22.5 million and $ 66.8 million of collaboration revenue, respectively, associated with the license, collaboration and option agreement (the “Roche Agreement”) with F. Hoffman-La Roche Ltd. (“Roche”). For the three and nine months ended September 30, 2020, the Company recognize d $ 22.5 million and $ 61.7 million, r espectively, of collaboration revenue primarily related to the Roche Agreement. As of September 30, 2021, the Company has total deferred revenue of $ 686.0 million associated with the Roche Agreement, of which $ 89.2 million i s classified as current. The portion of deferred revenue related to the separate material rights for the options to acquire ex-U.S. rights to certain Duchenne-specific programs was $ 485.0 million a s of September 30, 2021. The costs associated with co-development activities performed under the Roche Agreement are included in operating expenses, with any reimbursement of costs by Roche reflected as a reduction of such expenses when the related expense is incurred. For the three and nine months ended September 30, 2021, costs reimbursable by Roche and reflected as a reduction to operating expenses were $ 29.4 million and $ 60.8 million, respective ly. For the three and nine months ended September 30, 2020 , costs reimbursable by Roche and reflected as a reduction to operating expenses were $ 16.9 million and $ 42.2 million, respectively. As of September 30, 2021 , there was $ 29.1 million of collaboration receivable included in other current assets. 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 Duchenne and Becker muscular dystrophy. In October 2018, the Company exercised the option and entered into a license agreement with Nationwide, which granted the Company exclusive worldwide rights to develop, manufacture and commercialize a micro-dystrophin gene therapy product candidate. In July 2021, the Company entered into an agreement with Nationwide to settle a dispute relating to a sublicense payment owed by the Company resulting from the upfront payment received from Roche under the Roche Agreement. The total sublicense payment payable to Nationwide under the agreement is $ 38.0 million, which was paid in July 2021. Approximately $ 9.3 million of this amount was previously accrued as of December 31, 2020 with the remaining $ 28.7 million accrued during the three months ended June 30, 2021. The expense relating to this payment is recognized to research and development expense. As a result of this payment, the Company has no further financial obligations to Nationwide resulting from the upfront payment received under the Roche Agreement. BioMarin Pharmaceutical, Inc. The FDA approval of AMONDYS 45 (casimersen) in February 2021 resulted in a settlement charge to BioMarin Pharmaceutical, Inc. (“BioMarin”) of $ 10.0 million under the terms of the previous settlement agreement with BioMarin. This amount, which was expensed to operations as incurred, is separately presented as settlement and license charges in the Company’s unaudited condensed consolidated statement of operations and comprehensive loss for the nine months ended September 30, 2021. Research and Option Agreements The Company has research and option agreements with third parties in order to develop various technologies and biologics that may be used in the administration of the Company’s genetic therapeutics. The agreements generally provide for research services related to pre-clinical development programs, and options to license the technology for clinical development. Prior to the options under these agreements being executed, the Company may be required to make up to $ 11.0 million in research milestone payments. Under these agreements, there are $ 242.0 million in potential option payments to be made by the Company upon the determination to exercise the options. Additionally, if the options for each agreement are executed, the Company would incur additional contingent obligations and may be required to make development, regulatory, and sales milestone payments and tiered royalty payments based on the sales of the developed products upon commercialization. For the nine months ended September 30, 2021 , the Company recognized $ 3.0 million of research milestone expenses, with no similar activity for the nine months ended September 30, 2020. As of September 30, 2021, the Company has not exercised any options nor have any additional research milestone payments become probable of occurring. Milestone Obligations The Company has license and collaboration agreements in place for which it could be obligated to pay, in addition to the payment of up-front fees upon execution of the agreements, certain milestone payments as a product candidate proceeds from the submission of an investigational new drug application through approval for commercial sale and beyond. As of September 30, 2021, the Company may be obligated to make up to $ 4.1 billion in future development, regulatory, commercial and up-front royalty milestone payments associated with its license and collaboration agreements. These obligations exclude potential future option and milestone payments for options that have yet to be exercised within agreements entered into by the Company as of September 30, 2021, which are discussed above. For the three and nine months ended September 30, 2021, the Company recognized up-front, development milestone, settlement and other expenses of $ 4.5 million and $ 50.2 million , respectively, as research and development expense in the accompanying unaudited condensed consolidated statement of operations and comprehensive loss, inclusive of the expenses discussed above. For the three and nine months ended September 30, 2020 , the Company recognized up-front, development milestone, and other expenses of $ 15.4 million and $ 36.7 million, respectively, as research and development expense in the accompanying unaudited condensed consolidated statement of operations and comprehensive loss. |
GAIN FROM SALE OF PRIORITY REVI
GAIN FROM SALE OF PRIORITY REVIEW VOUCHER | 9 Months Ended |
Sep. 30, 2021 | |
Goodwill And Intangible Assets Disclosure [Abstract] | |
GAIN FROM SALE OF PRIORITY REVIEW VOUCHER | 4. GAIN FROM SALE OF PRIORITY REVIEW VOUCHER I n February 2021, the Company entered into an agreement to sell the rare pediatric disease Priority Review Voucher (“PRV”) it received from the FDA in connection with the approval of AMONDYS 45. Following the termination of the applicable waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976, as amended, in April 2021, the Company completed its sale of the PRV and received proceeds of $ 102.0 million, with no commission costs, 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. In February 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. Following the early termination of the applicable waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976, as amended, in March 2020, the Company completed its sale of the PRV and received proceeds of $ 108.1 million, net of commission, 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 | 9 Months Ended |
Sep. 30, 2021 | |
Fair Value Disclosures [Abstract] | |
FAIR VALUE MEASUREMENTS | 5. FAIR VALUE MEASUREMENTS The Company has certain financial assets and liabilities 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. During the nine months ended September 30, 2021, there were no transfers between Levels 1, 2 and 3. The tables below present information about the Company’s financial assets and liabilities that are measured and carried at fair value and indicate the level within the fair value hierarchy of valuation techniques it utilizes to determine such fair value: Fair Value Measurement as of September 30, 2021 Total Level 1 Level 2 Level 3 (in thousands) Assets Money market funds $ 1,287,292 $ 1,287,292 $ — $ — Strategic equity investments 35,288 2,876 — 32,412 Certificates of deposit 250 250 — — Total assets $ 1,322,830 $ 1,290,418 $ — $ 32,412 Liabilities Contingent consideration $ 43,600 $ — $ — $ 43,600 Total liabilities $ 43,600 $ — $ — $ 43,600 Fair Value Measurement as of December 31, 2020 Total Level 1 Level 2 Level 3 (in thousands) Assets Money market funds $ 629,440 $ 629,440 $ — $ — Government and government agency bonds 1,037,981 1,037,981 — — Strategic equity investments 38,799 3,699 — 35,100 Certificates of deposit 250 250 — — Total assets $ 1,706,470 $ 1,671,370 $ — $ 35,100 Liabilities Contingent consideration $ 50,800 $ — $ — $ 50,800 Total liabilities $ 50,800 $ — $ — $ 50,800 The Company’s assets with fair value categorized as Level 1 within the fair value hierarchy include money market funds, the Company’s strategic investment in Lysogene S.A. (“Lysogene”) and certificates of deposit. The Company did not hold any government and government agency bonds as of September 30, 2021. Certain of the government and government agency bonds held as of December 31, 2020 are publicly traded fixed income securities and are presented as cash equivalents on the condensed consolidated balance sheet as of December 31, 2020. The Company’s assets with fair value categorized as Level 3 within the fair value hierarchy consist of a strategic investment in Series A preferred stock of Lacerta Therapeutics, Inc. (“Lacerta”) and strategic investments in another two private companies. For more information related to Lacerta, please read Note 3, License and Collaboration Agreements of the Company’s Annual Report on Form 10-K for the year ended December 31, 2020. The fair value of the Lacerta investment was initially based on a cost approach corroborated by the Black-Scholes option pricing model. The most significant assumptions in the option pricing model include historical volatility of similar public companies, estimated term through Lacerta’s potential exit and a risk-free rate based on certain U.S. Treasury rates. The investments in the other two private companies are recorded at fair value at the time of purchase as measured by their respective investment cost. At the end of each reporting period, the fair value of the Company's strategic investments will be adjusted if the issuers are to issue similar or identical equity securities or when there is a triggering event for impairment. During the three and nine months ended September 30, 2021, the Company recorded an impairment loss of $ 4.5 million related to its investment in one of the private companies. The following tables represent a roll-forward of the fair value of Level 3 financial assets for each of the periods indicated: As of (in thousands) Fair value, as of December 31, 2020 $ 35,100 Additions 1,800 Change in estimated fair value ( 4,488 ) Fair value, as of September 30, 2021 $ 32,412 As of (in thousands) Fair value, as of December 31, 2019 $ 30,000 Additions — Change in estimated fair value — Fair value, as of September 30, 2020 $ 30,000 The Company’s contingent consideration liability with fair value categorized as Level 3 within the fair value hierarchy relates to the regulatory-related contingent payments to Myonexus Therapeutics, Inc. (“Myonexus”) selling shareholders as well as to two academic institutions under separate license agreements that meet the definition of a derivative. For more information related to Myonexus, please read Note 3, License and Collaboration Agreements of the Company’s Annual Report on Form 10-K for the year ended December 31, 2020 . The contingent consideration liability was estimated using an income approach based on the probability-weighted expected cash flows that incorporated industry-based probability adjusted assumptions relating to the achievement of the milestone and thus the likelihood of making the payments. Significant changes which increase or decrease the probabilities of achieving the milestone or shorten or lengthen the time required to achieve the milestone would result in a corresponding increase or decrease in the fair value of the liability. At the end of each reporting period, the fair value is adjusted to reflect the most current assumptions through earnings. The following tables represent a roll-forward of the fair value of Level 3 financial liabilities for each of the periods indicated: As of (in thousands) Fair value, as of December 31, 2020 $ 50,800 Changes in estimated fair value, net ( 7,200 ) Fair value, as of September 30, 2021 $ 43,600 As of (in thousands) Fair value, as of December 31, 2019 $ 5,200 Additions of contingent consideration 300 Changes in estimated fair value 45,000 Fair value, as of September 30, 2020 $ 50,500 A net decrease of $ 7.2 million was recorded during the three and nine months ended September 30, 2021 to account for the change in fair value of existing contingent consideration liabilities. This change, which is recorded through earnings, was a result of updates made to certain inputs and assumptions impacting the probability-weighted expected cash flows, principally the expected approval date of the underlying programs and the estimate of the amount of payments to be ultimately made. As of September 30, 2021, the contingent consideration was recorded as a non-current liability on the Company’s unaudited condensed consolidated balance sheets. The carrying amounts reported in the unaudited condensed consolidated balance sheets for cash and cash equivalents, accounts receivable and accounts payable approximated fair value because of the short-term maturity of these financial instruments. The fair value of the outstanding debt is disclosed in Note 11, Indebtedness . |
CASH, CASH EQUIVALENTS AND MARK
CASH, CASH EQUIVALENTS AND MARKETABLE SECURITIES | 9 Months Ended |
Sep. 30, 2021 | |
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 less than 90 days from the date of purchase included in cash equivalents in the unaudited condensed consolidated balance sheets for each of the periods indicated: As of As of (in thousands) Money market funds $ 1,287,292 $ 629,440 Government and government agency bonds — 602,058 Total $ 1,287,292 $ 1,231,498 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 September 30, 2021 and December 31, 2020 was less than one month and approximately two months , respectively. The following tables summarize the Company’s cash, cash equivalents and short-term investments as of the periods indicated: As of September 30, 2021 Amortized Gross Gross Fair (in thousands) Cash and money market funds $ 1,599,113 $ — $ — $ 1,599,113 Total cash, cash equivalents and investments $ 1,599,113 $ — $ — $ 1,599,113 As reported: Cash and cash equivalents $ 1,599,113 $ — $ — $ 1,599,113 Total cash, cash equivalents and investments $ 1,599,113 $ — $ — $ 1,599,113 As of December 31, 2020 Amortized Gross Gross Fair (in thousands) Cash and money market funds $ 900,590 $ — $ — $ 900,590 Government and government agency bonds 1,037,959 22 — 1,037,981 Total cash, cash equivalents and investments $ 1,938,549 $ 22 $ — $ 1,938,571 As reported: Cash and cash equivalents $ 1,502,639 $ 9 $ — $ 1,502,648 Short-term investments 435,910 13 — 435,923 Total cash, cash equivalents and investments $ 1,938,549 $ 22 $ — $ 1,938,571 |
PRODUCT REVENUES, NET, ACCOUNTS
PRODUCT REVENUES, NET, ACCOUNTS RECEIVABLE AND RESERVES FOR PRODUCT SALES | 9 Months Ended |
Sep. 30, 2021 | |
Receivables Net Current [Abstract] | |
PRODUCT REVENUES, NET, ACCOUNTS RECEIVABLE AND RESERVES FOR PRODUCT SALES | 7. PRODUCT REVENUES, NET, ACCOUNTS RECEIVABLE AND RESERVES FOR PRODUCT SALES The following table summarizes the Company’s product revenues, net by product for the periods indicated: For the Three Months Ended For the Nine Months Ended 2021 2020 2021 2020 (in thousands) EXONDYS 51 $ 115,598 $ 111,802 $ 335,244 $ 314,332 AMONDYS 45 26,655 — 33,784 — VYONDYS 53 24,658 9,627 64,648 18,889 Product revenues, net $ 166,911 $ 121,429 $ 433,676 $ 333,221 The following table summarizes the components of the Company’s accounts receivable for the periods indicated: As of As of (in thousands) Product sales receivable, net of discounts and allowances $ 149,317 $ 100,870 Government contract receivables 470 470 Total accounts receivable, net $ 149,787 $ 101,340 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 tables summarize an analysis of the change in reserves for discounts and allowances for each of the periods indicated: Chargebacks Rebates Prompt Pay Other Accruals Total (in thousands) Balance, as of December 31, 2020 $ 2,281 $ 41,771 $ 1,949 $ 4,969 $ 50,970 Provision 9,350 54,686 6,603 10,232 80,871 Payments/credits ( 10,878 ) ( 37,818 ) ( 5,865 ) ( 9,595 ) ( 64,156 ) Balance, as of September 30, 2021 $ 753 $ 58,639 $ 2,687 $ 5,606 $ 67,685 Chargebacks Rebates Prompt Pay Other Accruals Total (in thousands) Balance, as of December 31, 2019 $ 588 $ 44,738 $ 1,506 $ 4,671 $ 51,503 Provision 7,137 38,763 4,599 8,046 58,545 Payments/credits ( 6,699 ) ( 39,489 ) ( 4,348 ) ( 7,694 ) ( 58,230 ) Balance, as of September 30, 2020 $ 1,026 $ 44,012 $ 1,757 $ 5,023 $ 51,818 The following table summarizes the total reserves included in the Company’s unaudited condensed consolidated balance sheets for the periods indicated: As of As of (in thousands) Reduction to accounts receivable $ 7,743 $ 8,352 Component of accrued expenses 59,942 42,618 Total reserves $ 67,685 $ 50,970 |
INVENTORY
INVENTORY | 9 Months Ended |
Sep. 30, 2021 | |
Inventory Disclosure [Abstract] | |
INVENTORY | 8. INVENTORY The following table summarizes the components of the Company’s inventory for the periods indicated: As of As of (in thousands) Raw materials $ 40,156 $ 71,717 Work in progress 221,479 139,704 Finished goods 26,834 20,540 Total inventory $ 288,469 $ 231,961 |
OTHER ASSETS
OTHER ASSETS | 9 Months Ended |
Sep. 30, 2021 | |
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 As of (in thousands) Manufacturing-related deposits and prepaids $ 84,275 $ 134,430 Collaboration receivable 29,084 34,184 Prepaid clinical and pre-clinical expenses 12,497 16,224 Prepaid maintenance services 7,197 6,411 Prepaid research expenses 3,895 5,854 Leasehold improvement receivable 2,687 3,059 Prepaid income tax 1,599 4,939 Prepaid insurance 1,029 4,158 Other 5,678 4,065 Total other current assets $ 147,941 $ 213,324 The following table summarizes the Company’s other non-current assets for each of the periods indicated: As of As of (in thousands) Manufacturing-related deposits and prepaids $ 140,688 $ 148,525 Strategic investments 35,288 38,799 Restricted cash and investments 9,315 9,315 Prepaid clinical expenses 2,030 3,395 Other 3,471 3,669 Total other non-current assets $ 190,792 $ 203,703 |
ACCRUED EXPENSES
ACCRUED EXPENSES | 9 Months Ended |
Sep. 30, 2021 | |
Payables And Accruals [Abstract] | |
ACCRUED EXPENSES | 10. ACCRUED EXPENSES The following table summarizes the Company’s accrued expenses for each of the periods indicated: As of As of (in thousands) Accrued contract manufacturing costs $ 69,055 $ 36,543 Product revenue related reserves 59,942 42,618 Accrued employee compensation costs 29,725 50,803 Accrued clinical and pre-clinical costs 27,942 22,169 Accrued royalties 11,142 7,793 Accrued professional fees 8,929 10,221 Accrued collaboration cost sharing 4,927 3,516 Accrued interest expense 3,182 1,045 Accrued property and equipment 130 4,993 Accrued milestone and license expense — 9,380 Other 6,035 4,472 Total accrued expenses $ 221,009 $ 193,553 |
INDEBTEDNESS
INDEBTEDNESS | 9 Months Ended |
Sep. 30, 2021 | |
Debt Disclosure [Abstract] | |
INDEBTEDNESS | 1. INDEBTEDNESS 2024 Convertible Notes On November 14, 2017, the Company issued $ 570.0 million senior notes due on November 15, 2024 . 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 permits the certain holders to accelerate all outstanding obligations, including principal and interest. The Company incurred $ 10.6 million of offering costs, which represents 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. 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 shares per $1,000 principal amount of the 2024 Notes, which represents a conversion price of $ 73.42 per share, subject to adjustment under certain conditions. For more information related to the 2024 Notes, please read Note 13, Indebtedness of the Company’s Annual Report on Form 10-K for the year ended December 31, 2020. Upon adoption of ASU 2020-06, the 2024 Notes are accounted for as a single liability measured at its amortized cost. The cumulative effect of the accounting change as of January 1, 2021 increased the carrying amount of the convertible notes by $ 96.8 million, reduced the accumulated deficit by $ 60.2 million and reduced additional paid-in capital by $ 157.0 million. Interest expense of the 2024 Notes will be lower as a result of adoption of this guidance. The effective interest rate on the liability component of the 2024 Notes for both the three and nine months ended September 30, 2021 wa s 1.9 %. Fo r the three and nine months ended September 30, 2021, the interest expense related to the 2024 Notes was $ 2.7 million and $ 8.0 million, resp ectively. For the three and nine months ended September 30, 2020, the interest expense related to the 2024 Notes was $ 7.9 million and $ 23.4 million, respectively. The fair value of the Company’s 2024 Notes as of September 30, 2021 wa s $ 844.9 million, bas ed on open market trades and is classified as Level 1 in the fair value hierarchy. December 2019 Term Loan There have been no material changes to the December 2019 Term Loan in the three and nine months ended September 30, 2021. For more information related to the term loan, please read Note 13, Indebtedness of the Company’s Annual Report on Form 10-K for the year ended December 31, 2020. The following table summarizes the Company’s debt facilities for the periods indicated: As of As of (in thousands) Principal amount of the 2024 Notes $ 569,993 $ 569,993 Unamortized discount - equity component — ( 98,721 ) Unamortized discount - debt issuance costs ( 6,853 ) ( 6,510 ) Net carrying value of 2024 Notes 563,140 464,762 Principal amount of the 2019 Term Loan 550,000 550,000 Unamortized discounts ( 18,228 ) ( 22,269 ) Net carrying value of 2019 Term Loan 531,772 527,731 Total carrying value of debt facilities $ 1,094,912 $ 992,493 The following table summarizes the total gross payments due under the Company’s debt arrangements: As of (in thousands) 2021 (October-December) $ — 2022 — 2023 250,000 2024 869,993 2025 — Thereafter — Total payments $ 1,119,993 |
STOCK-BASED COMPENSATION
STOCK-BASED COMPENSATION | 9 Months Ended |
Sep. 30, 2021 | |
Disclosure Of Compensation Related Costs Sharebased Payments [Abstract] | |
STOCK-BASED COMPENSATION | 2. STOCK-BASED COMPENSATION The following table summarizes the Company’s stock awards granted for each of the periods indicated: For the Three Months Ended September 30, For the Nine Months Ended September 30, 2021 2020 2021 2020 Grants Weighted Grants Weighted Grants Weighted Grants Weighted Stock options 91,200 $ 42.62 97,240 $ 73.88 1,522,947 $ 48.50 1,256,064 $ 59.08 Restricted stock units 47,594 $ 83.37 49,430 $ 148.63 774,201 $ 86.34 592,867 $ 119.36 Stock-based Compensation Expense For the three months ended September 30, 2021 and 2020, total stock-based compensation expense was $ 26.7 million and $ 26.9 million, respectively. For the nine months ended September 30, 2021 and 2020, total stock-based compensation expense was $ 84.2 million and $ 78.5 million, respectively. The following table summarizes stock-based compensation expense by function included within the unaudited condensed consolidated statements of operations and comprehensive loss: For the Three Months Ended For the Nine Months Ended 2021 2020 2021 2020 (in thousands) Research and development $ 12,031 $ 10,645 $ 36,017 $ 31,034 Selling, general and administrative 14,653 16,258 48,144 47,509 Total stock-based compensation expense $ 26,684 $ 26,903 $ 84,161 $ 78,543 The following table summarizes stock-based compensation expense by grant type included within the unaudited condensed consolidated statements of operations and comprehensive loss: For the Three Months Ended For the Nine Months Ended 2021 2020 2021 2020 (in thousands) Stock options $ 13,912 $ 16,474 $ 48,518 $ 49,677 Restricted stock awards/units 11,695 8,950 32,199 24,047 Employee stock purchase plan 1,077 1,479 3,444 4,819 Total stock-based compensation expense $ 26,684 $ 26,903 $ 84,161 $ 78,543 |
OTHER (LOSS) INCOME
OTHER (LOSS) INCOME | 9 Months Ended |
Sep. 30, 2021 | |
Other Income And Expenses [Abstract] | |
OTHER (LOSS) INCOME | 13. OTHER (LOSS) INCOME The following table summarizes other (loss) income for the periods indicated: For the Three Months Ended For the Nine Months Ended 2021 2020 2021 2020 (in thousands) Interest expense $ ( 15,995 ) $ ( 13,889 ) $ ( 47,477 ) $ ( 41,217 ) Interest income 148 122 259 2,899 Amortization of investment discount — 313 157 4,276 Gain from sale of Priority Review — — 102,000 108,069 Gain (loss) on contingent consideration, 7,200 ( 45,000 ) 7,200 ( 45,000 ) Impairment of equity investment ( 4,488 ) — ( 4,488 ) — Other expense, net ( 314 ) ( 881 ) ( 813 ) ( 160 ) Total other (loss) income $ ( 13,449 ) $ ( 59,335 ) $ 56,838 $ 28,867 * The gain (loss) on contingent consideration, net is related to the fair value adjustment of the regulatory-related contingent payments that are accounted for as derivatives. Please see Note 5, Fair Value Measurements for further details. |
LEASES
LEASES | 9 Months Ended |
Sep. 30, 2021 | |
Leases [Abstract] | |
LEASES | 4. LEASES The Company has real estate operating leases in Cambridge, Andover and Burlington, Massachusetts, Dublin and Columbus, Ohio, and Durham, NC that provide for scheduled annual rent increases throughout each lease’s term. There have been no significant changes to the real estate leases in the nine months ended September 30, 2021. The Company has also identified embedded leases in its manufacturing and supply agreements with Catalent, Inc. (“Catalent”) and Thermo Fisher Scientific, Inc. (“Thermo”) as the Company determined that it controls the use of clean room suites and the related equipment therein. For additional details relating to these two agreements, please read Note 21, Commitments and Contingencies of the Annual Report on Form 10-K for the year ended December 31, 2020. The lease on four of the eight dedicated clean room suites at Thermo commenced during the second quarter of fiscal 2020. The lease on the remaining four of the eight dedicated clean room suites at Thermo commenced during the second quarter of fiscal 2021, which is when the dedicated clean room suites became available for use by the Company. As a result, as of September 30, 2021, the Company had additional aggregate right of use (“ROU”) assets of $ 13.3 million and aggregate lease liabilities of $ 11.1 million relating to these additional dedicated clean room suites. The difference between the ROU assets and the lease liabilities results from certain prepayments made to Thermo by the Company prior to the commencement of the leases. During the three months ended March 31, 2021, the Company modified the terms of its manufacturing and supply agreement with Catalent. The modification decreased the Company’s right of use of certain dedicated clean room suites and reduced the fixed and in-substance fixed payments due over the remaining term of the agreement. The modification was accounted for as a partial lease termination, resulting in: (i) the derecognition of right of use assets of $ 22.8 million, (ii) the derecognition of lease liabilities of $ 20.0 million, and (iii) the recognition of a loss of $ 2.8 million, which is included in research and development expense. |
NET LOSS PER SHARE
NET LOSS PER SHARE | 9 Months Ended |
Sep. 30, 2021 | |
Earnings Per Share [Abstract] | |
NET LOSS PER SHARE | 15. 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. For the three and nine months ended September 30, 2021 and 2020, there were no differences between basic and diluted net loss per share since the effect of common stock equivalents would be anti-dilutive due to the net loss position and, therefore, would be excluded from the diluted net loss per share calculation. For the Three Months Ended For the Nine Months Ended 2021 2020 2021 2020 (in thousands, except per share amounts) Net loss $ ( 48,144 ) $ ( 196,499 ) $ ( 296,799 ) $ ( 364,811 ) Weighted-average common shares outstanding - basic 79,880 78,501 79,695 77,637 Effect of dilutive securities* — — — — Weighted-average common shares outstanding - diluted 79,880 78,501 79,695 77,637 Net loss per share - basic and diluted $ ( 0.60 ) $ ( 2.50 ) $ ( 3.72 ) $ ( 4.70 ) * For both the three and nine months ended September 30, 2021 and 2020, stock options, restricted stock awards, restricted stock units, and employee stock purchase plan to purchase 9.6 million shares of the Company’s common stock, respectively, were excluded from the diluted 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 this obligation may be settled in cash or shares at the Company’s option. The effect of potential share settlement is included in the diluted EPS calculation if the effect is more dilutive. During the three and nine months ended September 30, 2021 , the inclusion of the potential share settlement of the 2024 Notes was anti-dilutive. |
COMMITMENTS AND CONTINGENCIES
COMMITMENTS AND CONTINGENCIES | 9 Months Ended |
Sep. 30, 2021 | |
Commitments And Contingencies Disclosure [Abstract] | |
COMMITMENTS AND CONTINGENCIES | 6. COMMITMENTS AND CONTINGENCIES Manufacturing Obligations The following table summarizes the aggregate non-cancelable contractual obligations arising from the Company’s manufacturing obligations: As of (in thousands) 2021 (October - December) $ 378,723 2022 325,106 2023 232,934 2024 213,008 2025 108,090 Thereafter 262,612 Total manufacturing commitments* $ 1,520,473 * Total manufacturing commitments include both the Catalent and Thermo manufacturing and supply agreements. Related to the leases at Catalent and Thermo, the Company has ROU assets and lease liabilities recorded on the unaudited condensed consolidated balance sheets as of September 30, 2021. For more information, please read Note 19, Leases of the Company’s Annual Report on Form 10-K for the year ended December 31, 2020. 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. 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, arising from the use of therapeutics utilizing its technology, or others. We record a loss contingency reserve for a legal proceeding when we consider the potential loss probable and we can reasonably estimate the amount of the loss or determine a probable range of loss. We provide disclosure when we consider a loss reasonably possible or when we determine that a loss in excess of a reserve is reasonably possible. We provide an estimate of such reasonably possible losses or an aggregate range of such reasonably possible losses, unless we believe that such an estimate cannot be made. On September 15, 2020, REGENXBIO INC. (“RegenX”) and the Trustees of the University of Pennsylvania filed a lawsuit against the Company and Sarepta Therapeutics Three, LLC (together, “Sarepta”), in the U.S. District Court for the District of Delaware. The plaintiffs assert patent infringement of U.S. Patent No. 10,526,617 (“the ‘617 Patent”) under 35 U.S.C.§§ 271(a)-(c) based on Sarepta’s alleged direct or indirect manufacture and use of cultured host cell technology to make adeno-associated virus (“AAV”) gene therapy products, including SRP-9001. Specifically, the Complaint essentially includes the allegation that Sarepta’s use, and the use by its contract manufacturers on its behalf, of a host cell containing a recombinant acid molecule that encodes a capsid protein having at least 95% amino acid identity to AAVrh10 infringes upon the ‘617 Patent asserted by RegenX. Plaintiffs seek injunctive relief, a judgment of infringement, an unspecified amount of damages that is no less than a reasonable royalty, a judgment of willful infringement, attorneys’ fees and costs, and such other relief as the court deems just and proper. On November 4, 2020, Sarepta moved to dismiss the case pursuant to Federal Rule of Civil Procedure 12(b)(6) based on the Safe Harbor provision of non-infringement contained in 35 U.S.C. § 271(e)(1). On October 5, 2021, the Court scheduled a hearing on Sarepta’s motion to dismiss for December 2, 2021. On July 13, 2021, Nippon Shinyaku Co., Ltd. (“Nippon Shinyaku” or “NS”) filed a lawsuit against the Company in the U.S. District Court for the District of Delaware. NS asserts a claim for breach of contract arising from Sarepta filing seven petitions for Inter Partes Review (“IPR Petitions”) with the Patent Trial and Appeal Board at the USPTO (PTAB Case Nos. IPR2021-01134, IPR2021-01135, IPR2021-01136, IPR2021-01137, IPR2021-01138, IPR2021-01139, IPR2021-01140) in which Sarepta is seeking to invalidate certain NS patents concerning exon 53 skipping technology (U.S. Patent Nos. 9,708,361, 10,385,092, 10,407,461, 10,487,106, 10,647,741, 10,662,217, and 10,683,322, respectively, and collectively the “NS Patents”). In addition, NS asserts claims for patent infringement of each of the NS Patents arising from Sarepta’s alleged activities concerning, including the sale of, its exon 53 skipping product, VYONDYS 53 (golodirsen). NS further seeks a determination of non-infringement by NS arising from NS’s alleged activities concerning, including the sale of, its exon 53 skipping product, Viltepso (viltolarsen) and invalidity of certain patents licensed to the Company from UWA (U.S. Patent Nos. 9,994,851, 10,227,590, and 10,266,827). NS filed a motion for preliminary injunction solely seeking Sarepta’s withdrawal of the IPR Petitions. The district court denied the motion for preliminary injunction on September 24, 2021, and NS appealed to the U.S. Court of Appeals for the Federal Circuit. On September 10, 2021, NS filed an amended complaint, which Sarepta moved to dismiss on September 24, 2021. |
SUBSEQUENT EVENT
SUBSEQUENT EVENT | 9 Months Ended |
Sep. 30, 2021 | |
Subsequent Events [Abstract] | |
SUBSEQUENT EVENT | 17. SUBSEQUENT EVENTS In October 2021, the Company sold approximately 7.1 million shares of common stock through an underwritten public offering, including 0.9 million shares sold pursuant to the underwriters’ option to purchase additional shares. The offering price was $ 81.00 per share. The Company expects to receive net proceeds of approximately $ 548.5 million from the offering, net of underwriting commissions and offering expenses of approximately $ 26.5 millio n. The Company entered into a development, commercial manufacturing and supply agreement in June 2018 and, subsequently, entered into the first and second amendments in May 2019 and July 2020, respectively, with Thermo, formerly Brammer Bio MA, LLC (collectively, the “Thermo Supply Agreements”). On October 1, 2021, the Company entered into its third amendment with Thermo. This amendment primarily modifies the term of the Thermo Supply Agreements, the capacity dedicated to the Company, as well as the payments due over the remaining term of the agreement. The Company continues to have the ability to terminate the agreement prior to the expiration, subject to the payment of additional financial consideration. As of the date of this report, the Company is still assessing the financial impact of this amendment. For more information related to the Thermo Supply Agreements, please read Note 21, Commitments and Contingencies of the Annual Report on the Form 10-K for the year ended December 31, 2020. |
SUMMARY OF SIGNIFICANT ACCOUN_2
SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES AND RECENT ACCOUNTING PRONOUNCEMENTS (Policies) | 9 Months Ended |
Sep. 30, 2021 | |
Accounting Policies [Abstract] | |
Basis of Presentation | Basis of Presentation The accompanying unaudited condensed consolidated financial statements, which have been prepared in accordance with accounting principles generally accepted in the United States (“U.S. GAAP”), reflect the accounts of Sarepta 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: discovering, developing, manufacturing and delivering therapies to patients with rare diseases. In the opinion of the Company’s management, all adjustments of a normal recurring nature necessary for a fair presentation have been reflected. Certain financial information that is normally included in annual financial statements prepared in accordance with the U.S. GAAP, but that is not required for interim reporting purposes, has been omitted. These unaudited condensed consolidated financial statements should be read in conjunction with the consolidated financial statements and related notes for the year ended December 31, 2020 which are contained in the Company’s Annual Report on Form 10-K for the year ended December 31, 2020, filed with the U.S. Securities and Exchange Commission on March 1, 2021. The results for the three and nine months ended September 30, 2021 are not necessarily indicative of the results to be expected for the full year. |
Estimates and Uncertainties | Estimates and Uncertainties The preparation of the unaudited condensed 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. |
Concentration of Credit Risk | Concentration of Credit Risk Financial instruments that potentially subject the Company to concentrations of credit risk consist of accounts receivable from customers, cash held at financial institutions and cash equivalents and investments. As of September 30, 2021 , the majority of the Company’s accounts receivable arose from product sales in the U.S. and all customers have standard payment terms that generally require payment within 60 to 91 days . Outside of the U.S., the majority of the Company’s customers have payment terms ranging between 45 and 150 days . Three individual c ustomers accounted for 45 %, 39 % and 11 % of net product revenues for the three months ended September 30, 2021 and 47 %, 40 % and 10 % of net pr oduct revenues for the nine months ended September 30, 2021. Three individual customers accounted for 47 %, 38 % and 10 % of product revenues for the three months ended September 30, 2020 and 47 %, 39 % and 11 % of net product revenues for the nine months ended September 30, 2020. Three individual customers accounted for 44 %, 39 % and 10 % of accounts receivable from product sales as of September 30, 2021 and 45 %, 41 % and 9 % of accounts receivable from product sales as of December 31, 2020. The Company monitors the financial performance and creditworthiness of its customers so that it can properly assess and respond to changes in its customers’ credit profile. As of September 30, 2021, the Company believes that such customers are of high credit quality. As of September 30, 2021, the Company’s cash was concentrated at three financial institutions in the U.S., which potentially exposes the Company to credit risks. However, the Company does not believe that there is significant risk of non-performance by the financial institutions. Significant Accounting Policies For details about the Company’s accounting policies, please read Note 2, Summary of Significant Accounting Policies and Recent Accounting Pronouncements of the Annual Report on Form 10-K for the year ended December 31, 2020. There have not been any material changes to the Company’s accounting policies through September 30, 2021, other than those noted below relating to the adoption of ASU 2020-06, “ Debt - Debt with Conversion and Other Options (Subtopic 470-20) and Derivatives and Hedging - Contracts in Entity's Own Equity (Subtopic 815-40): Accounting for Convertible Instruments and Contracts in an Entity's Own Equity .” |
Recent Accounting Pronouncements | Recent Accounting Pronouncements Recently adopted In August 2020, the Financial Accounting Standards Board (“FASB”) issued ASU 2020-06, “ Debt - Debt with Conversion and Other Options (Subtopic 470-20) and Derivatives and Hedging - Contracts in Entity's Own Equity (Subtopic 815-40): Accounting for Convertible Instruments and Contracts in an Entity's Own Equity .” This ASU simplifies the complexity associated with applying U.S. GAAP for certain financial instruments with characteristics of liabilities and equity. More specifically, the amendments focus on the guidance for convertible instruments and derivative scope exceptions for contracts in an entity’s own equity. Under ASU 2020-06, the embedded conversion features are no longer separated from the host contract for convertible instruments with conversion features that are not required to be accounted for as derivatives under Topic 815, or that do not result in substantial premiums accounted for as paid-in capital. Consequently, a convertible debt instrument, such as the Company’s senior notes due on November 15, 2024 (the “2024 Notes”), will be accounted for as a single liability measured at its amortized cost, as long as no other features require bifurcation and recognition as derivatives. The new guidance also requires the if-converted method to be applied for all convertible instruments and requires additional disclosures. This guidance is required to be adopted by January 1, 2022, and early adoption is permitted, but no earlier than fiscal years beginning after December 15, 2020. The Company elected to early adopt this guidance on January 1, 2021, using the modified retrospective method. Under this transition method, the cumulative effect of the accounting change removed the impact of recognizing the equity component of the Company’s convertible notes (at issuance and the subsequent accounting impact of additional interest expense from debt discount amortization). The cumulative effect of the accounting change as of January 1, 2021 increased the carrying amount of the convertible notes by $ 96.8 million, reduced accumulated deficit by $ 60.2 million and reduced additional paid-in capital by $ 157.0 million. Interest expense of the 2024 Notes will be lower as a result of adoption of this guidance. The if-converted method for such instruments will be used to compute diluted net earnings per share if and when profitability is achieved. As a result of the adoption of this guidance, interest expense and net loss was reduced by $ 5.6 million and $ 16.6 million, or $ 0.07 and $ 0.21 per share, respectively, for the three and nine months ended September 30, 2021. In December 2019, the FASB issued ASU 2019-12, “ Income Taxes (Topic 740): Simplifying the Accounting for Income Taxes”, which is intended to simplify the accounting for income taxes. This ASU removes certain exceptions to the general principles in Topic 740 and clarifies and amends existing guidance to improve consistent application. The new guidance was effective beginning January 1, 2021. The adoption of this guidance did not have a material effect on the Company’s condensed consolidated financial statements. |
FAIR VALUE MEASUREMENTS (Tables
FAIR VALUE MEASUREMENTS (Tables) | 9 Months Ended |
Sep. 30, 2021 | |
Fair Value Disclosures [Abstract] | |
Assets and Liabilities Measured and Carried at Fair Value | The tables below present information about the Company’s financial assets and liabilities that are measured and carried at fair value and indicate the level within the fair value hierarchy of valuation techniques it utilizes to determine such fair value: Fair Value Measurement as of September 30, 2021 Total Level 1 Level 2 Level 3 (in thousands) Assets Money market funds $ 1,287,292 $ 1,287,292 $ — $ — Strategic equity investments 35,288 2,876 — 32,412 Certificates of deposit 250 250 — — Total assets $ 1,322,830 $ 1,290,418 $ — $ 32,412 Liabilities Contingent consideration $ 43,600 $ — $ — $ 43,600 Total liabilities $ 43,600 $ — $ — $ 43,600 Fair Value Measurement as of December 31, 2020 Total Level 1 Level 2 Level 3 (in thousands) Assets Money market funds $ 629,440 $ 629,440 $ — $ — Government and government agency bonds 1,037,981 1,037,981 — — Strategic equity investments 38,799 3,699 — 35,100 Certificates of deposit 250 250 — — Total assets $ 1,706,470 $ 1,671,370 $ — $ 35,100 Liabilities Contingent consideration $ 50,800 $ — $ — $ 50,800 Total liabilities $ 50,800 $ — $ — $ 50,800 |
Summary of Fair Value of Level 3 Financial Assets | The following tables represent a roll-forward of the fair value of Level 3 financial assets for each of the periods indicated: As of (in thousands) Fair value, as of December 31, 2020 $ 35,100 Additions 1,800 Change in estimated fair value ( 4,488 ) Fair value, as of September 30, 2021 $ 32,412 As of (in thousands) Fair value, as of December 31, 2019 $ 30,000 Additions — Change in estimated fair value — Fair value, as of September 30, 2020 $ 30,000 |
Summary of Fair Value of Level 3 Financial Liabilities | The following tables represent a roll-forward of the fair value of Level 3 financial liabilities for each of the periods indicated: As of (in thousands) Fair value, as of December 31, 2020 $ 50,800 Changes in estimated fair value, net ( 7,200 ) Fair value, as of September 30, 2021 $ 43,600 As of (in thousands) Fair value, as of December 31, 2019 $ 5,200 Additions of contingent consideration 300 Changes in estimated fair value 45,000 Fair value, as of September 30, 2020 $ 50,500 |
CASH, CASH EQUIVALENTS AND MA_2
CASH, CASH EQUIVALENTS AND MARKETABLE SECURITIES (Tables) | 9 Months Ended |
Sep. 30, 2021 | |
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 less than 90 days from the date of purchase included in cash equivalents in the unaudited condensed consolidated balance sheets for each of the periods indicated: As of As of (in thousands) Money market funds $ 1,287,292 $ 629,440 Government and government agency bonds — 602,058 Total $ 1,287,292 $ 1,231,498 |
Summary of Company Cash, Cash Equivalents and Short-Term Investments | The following tables summarize the Company’s cash, cash equivalents and short-term investments as of the periods indicated: As of September 30, 2021 Amortized Gross Gross Fair (in thousands) Cash and money market funds $ 1,599,113 $ — $ — $ 1,599,113 Total cash, cash equivalents and investments $ 1,599,113 $ — $ — $ 1,599,113 As reported: Cash and cash equivalents $ 1,599,113 $ — $ — $ 1,599,113 Total cash, cash equivalents and investments $ 1,599,113 $ — $ — $ 1,599,113 As of December 31, 2020 Amortized Gross Gross Fair (in thousands) Cash and money market funds $ 900,590 $ — $ — $ 900,590 Government and government agency bonds 1,037,959 22 — 1,037,981 Total cash, cash equivalents and investments $ 1,938,549 $ 22 $ — $ 1,938,571 As reported: Cash and cash equivalents $ 1,502,639 $ 9 $ — $ 1,502,648 Short-term investments 435,910 13 — 435,923 Total cash, cash equivalents and investments $ 1,938,549 $ 22 $ — $ 1,938,571 |
PRODUCT REVENUES, NET, ACCOUN_2
PRODUCT REVENUES, NET, ACCOUNTS RECEIVABLE AND RESERVES FOR PRODUCT SALES (Tables) | 9 Months Ended |
Sep. 30, 2021 | |
Receivables Net Current [Abstract] | |
Summary of Product Revenues | The following table summarizes the Company’s product revenues, net by product for the periods indicated: For the Three Months Ended For the Nine Months Ended 2021 2020 2021 2020 (in thousands) EXONDYS 51 $ 115,598 $ 111,802 $ 335,244 $ 314,332 AMONDYS 45 26,655 — 33,784 — VYONDYS 53 24,658 9,627 64,648 18,889 Product revenues, net $ 166,911 $ 121,429 $ 433,676 $ 333,221 |
Summary of Components of Accounts Receivable | The following table summarizes the components of the Company’s accounts receivable for the periods indicated: As of As of (in thousands) Product sales receivable, net of discounts and allowances $ 149,317 $ 100,870 Government contract receivables 470 470 Total accounts receivable, net $ 149,787 $ 101,340 |
Summary of Change in Reserves for Discounts and Allowances | The following tables summarize an analysis of the change in reserves for discounts and allowances for each of the periods indicated: Chargebacks Rebates Prompt Pay Other Accruals Total (in thousands) Balance, as of December 31, 2020 $ 2,281 $ 41,771 $ 1,949 $ 4,969 $ 50,970 Provision 9,350 54,686 6,603 10,232 80,871 Payments/credits ( 10,878 ) ( 37,818 ) ( 5,865 ) ( 9,595 ) ( 64,156 ) Balance, as of September 30, 2021 $ 753 $ 58,639 $ 2,687 $ 5,606 $ 67,685 Chargebacks Rebates Prompt Pay Other Accruals Total (in thousands) Balance, as of December 31, 2019 $ 588 $ 44,738 $ 1,506 $ 4,671 $ 51,503 Provision 7,137 38,763 4,599 8,046 58,545 Payments/credits ( 6,699 ) ( 39,489 ) ( 4,348 ) ( 7,694 ) ( 58,230 ) Balance, as of September 30, 2020 $ 1,026 $ 44,012 $ 1,757 $ 5,023 $ 51,818 |
Summary of Total Reserves Included in Consolidated Balance Sheets | The following table summarizes the total reserves included in the Company’s unaudited condensed consolidated balance sheets for the periods indicated: As of As of (in thousands) Reduction to accounts receivable $ 7,743 $ 8,352 Component of accrued expenses 59,942 42,618 Total reserves $ 67,685 $ 50,970 |
INVENTORY (Tables)
INVENTORY (Tables) | 9 Months Ended |
Sep. 30, 2021 | |
Inventory Disclosure [Abstract] | |
Summary of Components of Inventory | The following table summarizes the components of the Company’s inventory for the periods indicated: As of As of (in thousands) Raw materials $ 40,156 $ 71,717 Work in progress 221,479 139,704 Finished goods 26,834 20,540 Total inventory $ 288,469 $ 231,961 |
OTHER ASSETS (Tables)
OTHER ASSETS (Tables) | 9 Months Ended |
Sep. 30, 2021 | |
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 As of (in thousands) Manufacturing-related deposits and prepaids $ 84,275 $ 134,430 Collaboration receivable 29,084 34,184 Prepaid clinical and pre-clinical expenses 12,497 16,224 Prepaid maintenance services 7,197 6,411 Prepaid research expenses 3,895 5,854 Leasehold improvement receivable 2,687 3,059 Prepaid income tax 1,599 4,939 Prepaid insurance 1,029 4,158 Other 5,678 4,065 Total other current assets $ 147,941 $ 213,324 |
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 As of (in thousands) Manufacturing-related deposits and prepaids $ 140,688 $ 148,525 Strategic investments 35,288 38,799 Restricted cash and investments 9,315 9,315 Prepaid clinical expenses 2,030 3,395 Other 3,471 3,669 Total other non-current assets $ 190,792 $ 203,703 |
ACCRUED EXPENSES (Tables)
ACCRUED EXPENSES (Tables) | 9 Months Ended |
Sep. 30, 2021 | |
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 As of (in thousands) Accrued contract manufacturing costs $ 69,055 $ 36,543 Product revenue related reserves 59,942 42,618 Accrued employee compensation costs 29,725 50,803 Accrued clinical and pre-clinical costs 27,942 22,169 Accrued royalties 11,142 7,793 Accrued professional fees 8,929 10,221 Accrued collaboration cost sharing 4,927 3,516 Accrued interest expense 3,182 1,045 Accrued property and equipment 130 4,993 Accrued milestone and license expense — 9,380 Other 6,035 4,472 Total accrued expenses $ 221,009 $ 193,553 |
INDEBTEDNESS (Tables)
INDEBTEDNESS (Tables) | 9 Months Ended |
Sep. 30, 2021 | |
Debt Disclosure [Abstract] | |
Summary of Debt Facilities | The following table summarizes the Company’s debt facilities for the periods indicated: As of As of (in thousands) Principal amount of the 2024 Notes $ 569,993 $ 569,993 Unamortized discount - equity component — ( 98,721 ) Unamortized discount - debt issuance costs ( 6,853 ) ( 6,510 ) Net carrying value of 2024 Notes 563,140 464,762 Principal amount of the 2019 Term Loan 550,000 550,000 Unamortized discounts ( 18,228 ) ( 22,269 ) Net carrying value of 2019 Term Loan 531,772 527,731 Total carrying value of debt facilities $ 1,094,912 $ 992,493 |
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 (in thousands) 2021 (October-December) $ — 2022 — 2023 250,000 2024 869,993 2025 — Thereafter — Total payments $ 1,119,993 |
STOCK-BASED COMPENSATION (Table
STOCK-BASED COMPENSATION (Tables) | 9 Months Ended |
Sep. 30, 2021 | |
Disclosure Of Compensation Related Costs Sharebased Payments [Abstract] | |
Summary of Stock Awards Granted | The following table summarizes the Company’s stock awards granted for each of the periods indicated: For the Three Months Ended September 30, For the Nine Months Ended September 30, 2021 2020 2021 2020 Grants Weighted Grants Weighted Grants Weighted Grants Weighted Stock options 91,200 $ 42.62 97,240 $ 73.88 1,522,947 $ 48.50 1,256,064 $ 59.08 Restricted stock units 47,594 $ 83.37 49,430 $ 148.63 774,201 $ 86.34 592,867 $ 119.36 |
Summary of Stock-Based Compensation Expense by Function Included within Condensed Consolidated Statements of Operations and Comprehensive Loss | The following table summarizes stock-based compensation expense by function included within the unaudited condensed consolidated statements of operations and comprehensive loss: For the Three Months Ended For the Nine Months Ended 2021 2020 2021 2020 (in thousands) Research and development $ 12,031 $ 10,645 $ 36,017 $ 31,034 Selling, general and administrative 14,653 16,258 48,144 47,509 Total stock-based compensation expense $ 26,684 $ 26,903 $ 84,161 $ 78,543 |
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 unaudited condensed consolidated statements of operations and comprehensive loss: For the Three Months Ended For the Nine Months Ended 2021 2020 2021 2020 (in thousands) Stock options $ 13,912 $ 16,474 $ 48,518 $ 49,677 Restricted stock awards/units 11,695 8,950 32,199 24,047 Employee stock purchase plan 1,077 1,479 3,444 4,819 Total stock-based compensation expense $ 26,684 $ 26,903 $ 84,161 $ 78,543 |
OTHER (LOSS) INCOME (Tables)
OTHER (LOSS) INCOME (Tables) | 9 Months Ended |
Sep. 30, 2021 | |
Other Income And Expenses [Abstract] | |
Summary of Other (Loss) Income | The following table summarizes other (loss) income for the periods indicated: For the Three Months Ended For the Nine Months Ended 2021 2020 2021 2020 (in thousands) Interest expense $ ( 15,995 ) $ ( 13,889 ) $ ( 47,477 ) $ ( 41,217 ) Interest income 148 122 259 2,899 Amortization of investment discount — 313 157 4,276 Gain from sale of Priority Review — — 102,000 108,069 Gain (loss) on contingent consideration, 7,200 ( 45,000 ) 7,200 ( 45,000 ) Impairment of equity investment ( 4,488 ) — ( 4,488 ) — Other expense, net ( 314 ) ( 881 ) ( 813 ) ( 160 ) Total other (loss) income $ ( 13,449 ) $ ( 59,335 ) $ 56,838 $ 28,867 * The gain (loss) on contingent consideration, net is related to the fair value adjustment of the regulatory-related contingent payments that are accounted for as derivatives. Please see Note 5, Fair Value Measurements for further details. |
NET LOSS PER SHARE (Tables)
NET LOSS PER SHARE (Tables) | 9 Months Ended |
Sep. 30, 2021 | |
Earnings Per Share [Abstract] | |
Basic and Diluted Net Loss Per Share | For the three and nine months ended September 30, 2021 and 2020, there were no differences between basic and diluted net loss per share since the effect of common stock equivalents would be anti-dilutive due to the net loss position and, therefore, would be excluded from the diluted net loss per share calculation. For the Three Months Ended For the Nine Months Ended 2021 2020 2021 2020 (in thousands, except per share amounts) Net loss $ ( 48,144 ) $ ( 196,499 ) $ ( 296,799 ) $ ( 364,811 ) Weighted-average common shares outstanding - basic 79,880 78,501 79,695 77,637 Effect of dilutive securities* — — — — Weighted-average common shares outstanding - diluted 79,880 78,501 79,695 77,637 Net loss per share - basic and diluted $ ( 0.60 ) $ ( 2.50 ) $ ( 3.72 ) $ ( 4.70 ) * For both the three and nine months ended September 30, 2021 and 2020, stock options, restricted stock awards, restricted stock units, and employee stock purchase plan to purchase 9.6 million shares of the Company’s common stock, respectively, were excluded from the diluted 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 this obligation may be settled in cash or shares at the Company’s option. The effect of potential share settlement is included in the diluted EPS calculation if the effect is more dilutive. During the three and nine months ended September 30, 2021 , the inclusion of the potential share settlement of the 2024 Notes was anti-dilutive. |
COMMITMENTS AND CONTINGENCIES (
COMMITMENTS AND CONTINGENCIES (Tables) | 9 Months Ended |
Sep. 30, 2021 | |
Commitments And Contingencies Disclosure [Abstract] | |
Summary of Aggregate Non-Cancelable Contractual Obligations Arising from Manufacturing Obligations | The following table summarizes the aggregate non-cancelable contractual obligations arising from the Company’s manufacturing obligations: As of (in thousands) 2021 (October - December) $ 378,723 2022 325,106 2023 232,934 2024 213,008 2025 108,090 Thereafter 262,612 Total manufacturing commitments* $ 1,520,473 * Total manufacturing commitments include both the Catalent and Thermo manufacturing and supply agreements. Related to the leases at Catalent and Thermo, the Company has ROU assets and lease liabilities recorded on the unaudited condensed consolidated balance sheets as of September 30, 2021. For more information, please read Note 19, Leases of the Company’s Annual Report on Form 10-K for the year ended December 31, 2020. |
Organization and Nature of Bu_2
Organization and Nature of Business - Additional Information (Detail) - USD ($) $ in Thousands | Sep. 30, 2021 | Dec. 31, 2020 | Sep. 30, 2020 |
Organization Consolidation And Presentation Of Financial Statements [Abstract] | |||
Cash, cash equivalents and investments | $ 1,608,400 | ||
Cash and cash equivalents | 1,599,113 | $ 1,502,648 | $ 1,474,637 |
Restricted cash and investments | $ 9,315 | $ 9,315 |
Summary of Significant Accoun_3
Summary of Significant Accounting Policies and Recent Accounting Pronouncements - Additional Information (Detail) $ / shares in Units, $ in Thousands | Jan. 02, 2021USD ($) | Sep. 30, 2021USD ($)$ / shares | Jun. 30, 2021USD ($) | Mar. 31, 2021USD ($) | Sep. 30, 2020USD ($) | Jun. 30, 2020USD ($) | Mar. 31, 2020USD ($) | Sep. 30, 2021USD ($)Segment$ / shares | Sep. 30, 2020USD ($) | Dec. 31, 2020 |
Summary Of Significant Accounting Policies [Line Items] | ||||||||||
Number of operating segments | Segment | 1 | |||||||||
Net loss | $ (48,144) | $ (81,405) | $ (167,250) | $ (196,499) | $ (150,820) | $ (17,492) | $ (296,799) | $ (364,811) | ||
ASU 2020-06 | ||||||||||
Summary Of Significant Accounting Policies [Line Items] | ||||||||||
Increase in convertible notes | $ 96,800 | |||||||||
Decrease in accumulated deficit | (60,200) | |||||||||
Decrease in additional paid-in-capital | $ (157,000) | |||||||||
Interest expense reduced | 5,600 | 16,600 | ||||||||
Net loss | $ 5,600 | $ 16,600 | ||||||||
Increase in diluted earnings per share (in dollar per share) | $ / shares | $ 0.07 | $ 0.21 | ||||||||
Outside of U.S. [Member] | Product Revenues [Member] | Customer One [Member] | ||||||||||
Summary Of Significant Accounting Policies [Line Items] | ||||||||||
Concentration of credit risk percentage | 45.00% | 47.00% | 47.00% | 47.00% | ||||||
Outside of U.S. [Member] | Product Revenues [Member] | Customer Two [Member] | ||||||||||
Summary Of Significant Accounting Policies [Line Items] | ||||||||||
Concentration of credit risk percentage | 39.00% | 38.00% | 40.00% | 39.00% | ||||||
Outside of U.S. [Member] | Product Revenues [Member] | Customer Three [Member] | ||||||||||
Summary Of Significant Accounting Policies [Line Items] | ||||||||||
Concentration of credit risk percentage | 11.00% | 10.00% | 10.00% | 11.00% | ||||||
Outside of U.S. [Member] | Accounts Receivable [Member] | Customer One [Member] | ||||||||||
Summary Of Significant Accounting Policies [Line Items] | ||||||||||
Concentration of credit risk percentage | 44.00% | 45.00% | ||||||||
Outside of U.S. [Member] | Accounts Receivable [Member] | Customer Two [Member] | ||||||||||
Summary Of Significant Accounting Policies [Line Items] | ||||||||||
Concentration of credit risk percentage | 39.00% | 41.00% | ||||||||
Outside of U.S. [Member] | Accounts Receivable [Member] | Customer Three [Member] | ||||||||||
Summary Of Significant Accounting Policies [Line Items] | ||||||||||
Concentration of credit risk percentage | 10.00% | 9.00% | ||||||||
Minimum [Member] | ||||||||||
Summary Of Significant Accounting Policies [Line Items] | ||||||||||
Accounts receivable payment term | 60 days | |||||||||
Minimum [Member] | Outside of U.S. [Member] | ||||||||||
Summary Of Significant Accounting Policies [Line Items] | ||||||||||
Accounts receivable payment term | 45 days | |||||||||
Maximum [Member] | ||||||||||
Summary Of Significant Accounting Policies [Line Items] | ||||||||||
Accounts receivable payment term | 91 days | |||||||||
Maximum [Member] | Outside of U.S. [Member] | ||||||||||
Summary Of Significant Accounting Policies [Line Items] | ||||||||||
Accounts receivable payment term | 150 days |
License and Collaboration Agr_2
License and Collaboration Agreements - Additional Information (Detail) - USD ($) $ in Thousands | 1 Months Ended | 3 Months Ended | 9 Months Ended | 12 Months Ended | |||
Jul. 31, 2021 | Sep. 30, 2021 | Jun. 30, 2021 | Sep. 30, 2020 | Sep. 30, 2021 | Sep. 30, 2020 | Dec. 31, 2020 | |
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||
Revenue | $ 166,911 | $ 121,429 | $ 433,676 | $ 333,221 | |||
Settlement charge | 10,000 | ||||||
BioMarin Pharmaceutical, Inc. [Member] | Research and Development Expense [Member] | |||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||
Settlement charge | 10,000 | ||||||
Collaborative Arrangement | |||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||
Contingent research milestone payments | 3,000 | ||||||
Payment for option exercise | $ 242,000 | ||||||
Stock options exercised or expired | 0 | ||||||
Development Milestone and Upfront Fee Recognized as Research and Development Expense | 4,500 | 15,400 | $ 50,200 | 36,700 | |||
Collaborative Arrangement | Maximum [Member] | |||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||
Contingent research milestone payments | 11,000 | ||||||
Development Milestone and Settlement Upfront Fee Recognized as Research and Development Expense | 4,100 | 4,100 | |||||
Collaborative Arrangement | Roche | |||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||
Revenue | 22,500 | 22,500 | 66,800 | 61,700 | |||
Deferred Revenue | 686,000 | 686,000 | |||||
Deferred Revenue, Current | 89,200 | 89,200 | |||||
Deferred Revenue Separate Material Options Right | 485,000 | 485,000 | |||||
Research and development expense | 29,400 | $ 16,900 | 60,800 | $ 42,200 | |||
Collaboration Receivable | $ 29,100 | $ 29,100 | |||||
Nationwide License Agreement [Member] | NationWide Children Hospital [Member] | |||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||
Sublicense payment | $ 38,000 | ||||||
Nationwide License Agreement [Member] | NationWide Children Hospital [Member] | Research and Development Expense [Member] | |||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||
Sublicense payment | $ 28,700 | $ 9,300 |
Gain from Sale of Priority Re_2
Gain from Sale of Priority Review Voucher - Additional Information (Detail) $ in Millions | 9 Months Ended |
Sep. 30, 2021USD ($) | |
P R V | |
Gain From Sale Of Intangible Asset [Line Items] | |
Proceeds from sale of rare pediatric disease priority review voucher, Net of commission | $ 102 |
F D A | |
Gain From Sale Of Intangible Asset [Line Items] | |
Proceeds from sale of rare pediatric disease priority review voucher, Net of commission | $ 108.1 |
Assets and Liabilities Measured
Assets and Liabilities Measured and Carried at Fair Value (Detail) - USD ($) $ in Thousands | Sep. 30, 2021 | Dec. 31, 2020 | Sep. 30, 2020 | Dec. 31, 2019 |
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||||
Total assets | $ 1,322,830 | $ 1,706,470 | ||
Contingent consideration | 43,600 | 50,800 | ||
Total liabilities | 50,800 | |||
Money Market Funds [Member] | ||||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||||
Total assets | 1,287,292 | 629,440 | ||
Government and Government Agency Bonds [Member] | ||||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||||
Total assets | 1,037,981 | |||
Strategic Equity Investments [Member] | ||||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||||
Total assets | 35,288 | 38,799 | ||
Certificates of Deposit [Member] | ||||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||||
Total assets | 250 | 250 | ||
Level 1 [Member] | ||||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||||
Total assets | 1,290,418 | 1,671,370 | ||
Level 1 [Member] | Money Market Funds [Member] | ||||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||||
Total assets | 1,287,292 | 629,440 | ||
Level 1 [Member] | Government and Government Agency Bonds [Member] | ||||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||||
Total assets | 1,037,981 | |||
Level 1 [Member] | Strategic Equity Investments [Member] | ||||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||||
Total assets | 2,876 | 3,699 | ||
Level 1 [Member] | Certificates of Deposit [Member] | ||||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||||
Total assets | 250 | 250 | ||
Level 3 [Member] | ||||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||||
Total assets | 32,412 | 35,100 | $ 30,000 | $ 30,000 |
Contingent consideration | 43,600 | 50,800 | ||
Total liabilities | 43,600 | 50,800 | $ 50,500 | $ 5,200 |
Level 3 [Member] | Strategic Equity Investments [Member] | ||||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||||
Total assets | $ 32,412 | $ 35,100 |
Fair Value Measurements - Summa
Fair Value Measurements - Summary of Fair Value of Level 3 Financial Assets (Details) - USD ($) $ in Thousands | 9 Months Ended | |
Sep. 30, 2021 | Sep. 30, 2020 | |
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Fair value, at the beginning of the period | $ 1,706,470 | |
Fair value, at the end of the period | 1,322,830 | |
Fair Value Inputs Level3 [Member] | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Fair value, at the beginning of the period | 35,100 | $ 30,000 |
Additions | 1,800 | 0 |
Changes in estimated fair value | 4,488 | 0 |
Fair value, at the end of the period | $ 32,412 | $ 30,000 |
Fair Value Measurements - Sum_2
Fair Value Measurements - Summary of Fair Value of Level 3 Financial Liabilities (Details) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | |
Sep. 30, 2021 | Sep. 30, 2021 | Sep. 30, 2020 | |
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | |||
Fair value at the beginning of the period | $ 50,800 | ||
Changes in estimated fair value | $ (7,200) | (7,200) | |
Fair Value Inputs Level3 [Member] | |||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | |||
Fair value at the beginning of the period | 50,800 | $ 5,200 | |
Additions of contingent consideration | 300 | ||
Changes in estimated fair value | 7,200 | (45,000) | |
Fair value at the end of the period | $ 43,600 | $ 43,600 | $ 50,500 |
Fair Value Measurements - Addit
Fair Value Measurements - Additional Information (Detail) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2021 | Sep. 30, 2020 | Sep. 30, 2021 | Sep. 30, 2020 | |
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||||
Impairment of investment in private companies | $ 4,488 | $ 4,488 | ||
Business Combination, Contingent Consideration Arrangements, Change in Amount of Contingent Consideration, Liability | $ 7,200 | $ 7,200 |
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 | Sep. 30, 2021 | Dec. 31, 2020 |
Cash and Cash Equivalents [Line Items] | ||
Cash equivalents | $ 1,287,292 | $ 1,231,498 |
Money Market Funds [Member] | ||
Cash and Cash Equivalents [Line Items] | ||
Cash equivalents | 1,287,292 | 629,440 |
Government and Government Agency Bonds [Member] | ||
Cash and Cash Equivalents [Line Items] | ||
Cash equivalents | $ 602,058 |
Cash, Cash Equivalents and Ma_3
Cash, Cash Equivalents and Marketable Securities - Additional Information (Detail) | 9 Months Ended | 12 Months Ended |
Sep. 30, 2021 | Dec. 31, 2020 | |
Cash And Cash Equivalents [Line Items] | ||
Weighted average maturity period of available-for-sale securities | 2 months | |
Maximum [Member] | ||
Cash And Cash Equivalents [Line Items] | ||
Weighted average maturity period of available-for-sale securities | 1 month |
Summary of Company Cash, Cash E
Summary of Company Cash, Cash Equivalents and Short-term Investments (Detail) - USD ($) $ in Thousands | Sep. 30, 2021 | Dec. 31, 2020 | Sep. 30, 2020 |
Cash and Cash Equivalents [Line Items] | |||
Cash and cash equivalents, Amortized cost | $ 1,599,113 | $ 1,502,639 | |
Cash and cash equivalents, Gross unrealized gains | 9 | ||
Cash and cash equivalents | 1,599,113 | 1,502,648 | $ 1,474,637 |
Cash, cash equivalents and investments, Amortized cost | 1,599,113 | 1,938,549 | |
Cash, cash equivalents and investments, Gross unrealized gains | 22 | ||
Cash, cash equivalents and investments, Fair value | 1,599,113 | 1,938,571 | |
Available for sale debt securities current, Amortized cost | 435,910 | ||
Available for sale debt securities current, Gross unrealized gains | 13 | ||
Available for sale debt securities current, Fair value | 435,923 | ||
Cash and Money Market Funds [Member] | |||
Cash and Cash Equivalents [Line Items] | |||
Cash and cash equivalents, Amortized cost | 1,599,113 | 900,590 | |
Cash and cash equivalents | $ 1,599,113 | 900,590 | |
Government and Government Agency Bonds [Member] | |||
Cash and Cash Equivalents [Line Items] | |||
Available for sale debt securities, Amortized cost | 1,037,959 | ||
Available for sale debt securities, Gross unrealized gains | 22 | ||
Available for sale debt securities, Fair value | $ 1,037,981 |
Summary of Product Revenues (De
Summary of Product Revenues (Detail) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2021 | Sep. 30, 2020 | Sep. 30, 2021 | Sep. 30, 2020 | |
Disaggregation Of Revenue [Line Items] | ||||
Products, net | $ 166,911 | $ 121,429 | $ 433,676 | $ 333,221 |
EXONDYS 51 [Member] | ||||
Disaggregation Of Revenue [Line Items] | ||||
Products, net | 115,598 | 111,802 | 335,244 | 314,332 |
VYONDYS 53 [Member] | ||||
Disaggregation Of Revenue [Line Items] | ||||
Products, net | 24,658 | 9,627 | 64,648 | 18,889 |
AMONDYS 45 [Member] | ||||
Disaggregation Of Revenue [Line Items] | ||||
Products, net | $ 26,655 | $ 0 | $ 33,784 | $ 0 |
Summary of Components of Accoun
Summary of Components of Accounts Receivable (Detail) - USD ($) $ in Thousands | Sep. 30, 2021 | Dec. 31, 2020 |
Receivables Net Current [Abstract] | ||
Product sales receivable, net of discounts and allowances | $ 149,317 | $ 100,870 |
Government contract receivables | 470 | 470 |
Total accounts receivable, net | $ 149,787 | $ 101,340 |
Summary of Change in Reserves f
Summary of Change in Reserves for Discounts and Allowances (Detail) - USD ($) $ in Thousands | 9 Months Ended | |
Sep. 30, 2021 | Sep. 30, 2020 | |
Accounts Notes And Loans Receivable [Line Items] | ||
Beginning balance | $ 50,970 | $ 51,503 |
Provision | 80,871 | 58,545 |
Payments/credits | 64,156 | (58,230) |
Ending balance | 67,685 | 51,818 |
Chargebacks [Member] | ||
Accounts Notes And Loans Receivable [Line Items] | ||
Beginning balance | 2,281 | 588 |
Provision | 9,350 | 7,137 |
Payments/credits | 10,878 | 6,699 |
Ending balance | 753 | 1,026 |
Rebates [Member] | ||
Accounts Notes And Loans Receivable [Line Items] | ||
Beginning balance | 41,771 | 44,738 |
Provision | 54,686 | 38,763 |
Payments/credits | 37,818 | 39,489 |
Ending balance | 58,639 | 44,012 |
Prompt Pay [Member] | ||
Accounts Notes And Loans Receivable [Line Items] | ||
Beginning balance | 1,949 | 1,506 |
Provision | 6,603 | 4,599 |
Payments/credits | 5,865 | 4,348 |
Ending balance | 2,687 | 1,757 |
Other Accruals [Member] | ||
Accounts Notes And Loans Receivable [Line Items] | ||
Beginning balance | 4,969 | 4,671 |
Provision | 10,232 | 8,046 |
Payments/credits | 9,595 | 7,694 |
Ending balance | $ 5,606 | $ 5,023 |
Summary of Total Reserves Inclu
Summary of Total Reserves Included in Consolidated Balance Sheets (Detail) - USD ($) $ in Thousands | Sep. 30, 2021 | Dec. 31, 2020 | Sep. 30, 2020 | Dec. 31, 2019 |
Receivables Net Current [Abstract] | ||||
Reduction to accounts receivable | $ 7,743 | $ 8,352 | ||
Component of accrued expenses | 59,942 | 42,618 | ||
Total reserves | $ 67,685 | $ 50,970 | $ 51,818 | $ 51,503 |
Inventory - Summary of Componen
Inventory - Summary of Components of Inventory (Detail) - USD ($) $ in Thousands | Sep. 30, 2021 | Dec. 31, 2020 |
Inventory Disclosure [Abstract] | ||
Raw materials | $ 40,156 | $ 71,717 |
Work in progress | 221,479 | 139,704 |
Finished goods | 26,834 | 20,540 |
Total inventory | $ 288,469 | $ 231,961 |
Summary of Other Current Assets
Summary of Other Current Assets (Detail) - USD ($) $ in Thousands | Sep. 30, 2021 | Dec. 31, 2020 |
Prepaid Expense And Other Assets Current [Abstract] | ||
Manufacturing-related deposits and prepaids | $ 84,275 | $ 134,430 |
Collaboration receivable | 29,084 | 34,184 |
Prepaid clinical and pre-clinical expenses | 12,497 | 16,224 |
Prepaid maintenance services | 7,197 | 6,411 |
Prepaid research expenses | 3,895 | 5,854 |
Leasehold improvement receivable | 2,687 | 3,059 |
Prepaid income tax | 1,599 | 4,939 |
Prepaid insurance | 1,029 | 4,158 |
Other | 5,678 | 4,065 |
Total other current assets | $ 147,941 | $ 213,324 |
Summary of Other Non-current As
Summary of Other Non-current Assets (Detail) - USD ($) $ in Thousands | Sep. 30, 2021 | Dec. 31, 2020 |
Prepaid Expense And Other Assets Noncurrent [Abstract] | ||
Manufacturing-related deposits and prepaids | $ 140,688 | $ 148,525 |
Strategic investments | 35,288 | 38,799 |
Restricted cash and investments | 9,315 | 9,315 |
Prepaid clinical expenses | 2,030 | 3,395 |
Other | 3,471 | 3,669 |
Total other non-current assets | $ 190,792 | $ 203,703 |
Summary of Accrued Expenses (De
Summary of Accrued Expenses (Detail) - USD ($) $ in Thousands | Sep. 30, 2021 | Dec. 31, 2020 |
Payables And Accruals [Abstract] | ||
Accrued contract manufacturing costs | $ 69,055 | $ 36,543 |
Product revenue related reserves | 59,942 | 42,618 |
Accrued employee compensation costs | 29,725 | 50,803 |
Accrued clinical and pre-clinical costs | 27,942 | 22,169 |
Accrued royalties | 11,142 | 7,793 |
Accrued professional fees | 8,929 | 10,221 |
Accrued collaboration cost sharing | 4,927 | 3,516 |
Accrued interest expense | 3,182 | 1,045 |
Accrued property and equipment | 130 | 4,993 |
Accrued milestone and license expense | 9,380 | |
Other | 6,035 | 4,472 |
Total accrued expenses | $ 221,009 | $ 193,553 |
Indebtedness - Additional Infor
Indebtedness - Additional Information (Detail) | Jan. 01, 2021USD ($) | Nov. 14, 2017USD ($)shares$ / shares | Sep. 30, 2021USD ($) | Sep. 30, 2020USD ($) | Sep. 30, 2021USD ($) | Sep. 30, 2020USD ($) | Dec. 31, 2020USD ($) |
Debt Instrument [Line Items] | |||||||
Debt instrument, face amount | $ 1,119,993,000 | $ 1,119,993,000 | |||||
Debt instrument, conversion rate | 0.013621 | ||||||
2024 Convertible Notes [Member] | |||||||
Debt Instrument [Line Items] | |||||||
Debt instrument, face amount | $ 570,000,000 | $ 569,993,000 | $ 569,993,000 | $ 569,993,000 | |||
Debt instrument, maturity date | Nov. 15, 2024 | ||||||
Debt instrument, interest rate per annum | 1.50% | ||||||
Debt instrument, payment frequency | The 2024 Notes contain customary covenants and events of default, occurrence of which permits the certain holders to accelerate all outstanding obligations, including principal and interest. | ||||||
Debt issuance costs | $ 10,600,000 | ||||||
Debt instrument, conversion price | $ / shares | $ 73.42 | ||||||
2024 Convertible Notes [Member] | ASU 2020-06 | |||||||
Debt Instrument [Line Items] | |||||||
Increase in convertible notes | $ 96,800,000 | ||||||
Decrease in accumulated deficit | (60,200,000) | ||||||
Decrease in additional paid-in-capital | $ (157,000,000) | ||||||
Effective interest rate percentage | 1.90% | 1.90% | |||||
Interest expense reduced | $ 2,700,000 | $ 7,900,000 | $ 8,000,000 | $ 23,400,000 | |||
Debt Instrument Fair Value | $ 844,900,000 | $ 844,900,000 | |||||
2024 Convertible Notes [Member] | Common Stock [Member] | |||||||
Debt Instrument [Line Items] | |||||||
Debt instrument, convertible into shares | shares | 7,763,552 |
Indebtedness - Summary of Debt
Indebtedness - Summary of Debt Facilities (Detail) - USD ($) | Sep. 30, 2021 | Dec. 31, 2020 | Nov. 14, 2017 |
Debt Instrument [Line Items] | |||
Principal amount | $ 1,119,993,000 | ||
Total carrying value of debt facilities | 1,094,912,000 | $ 992,493,000 | |
2024 Convertible Notes [Member] | |||
Debt Instrument [Line Items] | |||
Principal amount | 569,993,000 | 569,993,000 | $ 570,000,000 |
Unamortized discount | (98,721,000) | ||
Unamortized discount - debt issuance costs | (6,853,000) | (6,510,000) | |
Net carrying value | 563,140,000 | 464,762,000 | |
2019 Term Loan [Member] | |||
Debt Instrument [Line Items] | |||
Principal amount | 550,000,000 | 550,000,000 | |
Unamortized discount | (18,228,000) | (22,269,000) | |
Net carrying value | $ 531,772,000 | $ 527,731,000 |
Indebtedness - Summarizes Total
Indebtedness - Summarizes Total Gross Payments Due under Company's Debt Arrangements (Detail) $ in Thousands | Sep. 30, 2021USD ($) |
Debt Disclosure [Abstract] | |
2023 | $ 250,000 |
2024 | 869,993 |
Total payments | $ 1,119,993 |
Summary of Stock Awards Granted
Summary of Stock Awards Granted (Detail) - $ / shares | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2021 | Sep. 30, 2020 | Sep. 30, 2021 | Sep. 30, 2020 | |
Stock Options [Member] | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Grants, Stock options | 91,200 | 97,240 | 1,522,947 | 1,256,064 |
Weighted Average Grant Date Fair Value, Stock options | $ 42.62 | $ 73.88 | $ 48.50 | $ 59.08 |
Restricted Stock Units [Member] | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Grants, Restricted stock units and awards | 47,594 | 49,430 | 774,201 | 592,867 |
Weighted Average Grant Date Fair Value, Restricted stock units and awards | $ 83.37 | $ 148.63 | $ 86.34 | $ 119.36 |
Stock Based Compensation - Addi
Stock Based Compensation - Additional Information (Detail) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2021 | Sep. 30, 2020 | Sep. 30, 2021 | Sep. 30, 2020 | |
Disclosure Of Compensation Related Costs Sharebased Payments [Abstract] | ||||
Stock-based compensation expense | $ 26,684 | $ 26,903 | $ 84,161 | $ 78,543 |
Summary of Stock-Based Compensa
Summary of Stock-Based Compensation Expense by Function Included within Condensed Consolidated Statements of Operations and Comprehensive Loss (Detail) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2021 | Sep. 30, 2020 | Sep. 30, 2021 | Sep. 30, 2020 | |
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | ||||
Total stock-based compensation expense | $ 26,684 | $ 26,903 | $ 84,161 | $ 78,543 |
Research and Development [Member] | ||||
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | ||||
Total stock-based compensation expense | 12,031 | 10,645 | 36,017 | 31,034 |
Selling, General and Administrative [Member] | ||||
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | ||||
Total stock-based compensation expense | $ 14,653 | $ 16,258 | $ 48,144 | $ 47,509 |
Summary of Stock-Based Compen_2
Summary of Stock-Based Compensation Expense by Grant Type Included within Condensed Consolidated Statements of Operations and Comprehensive Loss (Detail) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2021 | Sep. 30, 2020 | Sep. 30, 2021 | Sep. 30, 2020 | |
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | ||||
Total stock-based compensation expense | $ 26,684 | $ 26,903 | $ 84,161 | $ 78,543 |
Stock Options [Member] | ||||
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | ||||
Total stock-based compensation expense | 13,912 | 16,474 | 48,518 | 49,677 |
Restricted Stock Awards/Units (RSAs/RSUs) [Member] | ||||
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | ||||
Total stock-based compensation expense | 11,695 | 8,950 | 32,199 | 24,047 |
Employee Stock Purchase Plan [Member] | ||||
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | ||||
Total stock-based compensation expense | $ 1,077 | $ 1,479 | $ 3,444 | $ 4,819 |
Summary of Other (Loss) Income
Summary of Other (Loss) Income (Detail) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2021 | Sep. 30, 2020 | Sep. 30, 2021 | Sep. 30, 2020 | |
Nonoperating Income Expense [Abstract] | ||||
Interest expense | $ (15,995) | $ (13,889) | $ (47,477) | $ (41,217) |
Interest income | 148 | 122 | 259 | 2,899 |
Amortization of investment discount | 0 | 313 | 157 | 4,276 |
Gain from sale of Priority Review Voucher | 102,000 | 108,069 | ||
Gain (loss) on contingent consideration, net | 7,200 | (45,000) | 7,200 | (45,000) |
Impairment of equity investment | (4,488) | (4,488) | ||
Other (expense) income, net | (314) | (881) | (813) | (160) |
Total other (loss) income | $ (13,449) | $ (59,335) | $ 56,838 | $ 28,867 |
Leases - Additional Information
Leases - Additional Information (Detail) $ in Thousands | 3 Months Ended | ||||
Mar. 31, 2021USD ($) | Sep. 30, 2021USD ($) | Jun. 30, 2021Suite | Dec. 31, 2020USD ($) | Jun. 30, 2020Suite | |
Lessee Lease Description [Line Items] | |||||
Right of use assets | $ 72,663 | $ 91,761 | |||
Research and Development Expense [Member] | |||||
Lessee Lease Description [Line Items] | |||||
Derecognition of right of use asset | $ 22,800 | ||||
Derecognition of lease liabilities | 20,000 | ||||
Recognition of loss | $ 2,800 | ||||
Thermo Fisher Scientific Inc. [Member] | Thermo Agreements [Member] | |||||
Lessee Lease Description [Line Items] | |||||
Number of dedicated clean room suite | Suite | 8 | 8 | |||
Right of use assets | 13,300 | ||||
Operating Lease Liability | $ 11,100 | ||||
Thermo Fisher Scientific Inc. [Member] | Thermo Agreements [Member] | Other Assets [Member] | |||||
Lessee Lease Description [Line Items] | |||||
Number of dedicated clean room suite | Suite | 4 | 4 |
Basic and Diluted Net Loss Per
Basic and Diluted Net Loss Per Share (Detail) - USD ($) $ / shares in Units, $ in Thousands | 3 Months Ended | 9 Months Ended | |||||||
Sep. 30, 2021 | Jun. 30, 2021 | Mar. 31, 2021 | Sep. 30, 2020 | Jun. 30, 2020 | Mar. 31, 2020 | Sep. 30, 2021 | Sep. 30, 2020 | ||
Earnings Per Share [Abstract] | |||||||||
Net loss | $ (48,144) | $ (81,405) | $ (167,250) | $ (196,499) | $ (150,820) | $ (17,492) | $ (296,799) | $ (364,811) | |
Weighted-average common shares outstanding - basic | 79,880 | 78,501 | 79,695 | 77,637 | |||||
Effect of dilutive securities* | [1] | 0 | 0 | 0 | 0 | ||||
Weighted-average common shares outstanding - diluted | 79,880 | 78,501 | 79,695 | 77,637 | |||||
Net loss per share - basic and diluted | $ (0.60) | $ (2.50) | $ (3.72) | $ (4.70) | |||||
[1] | For both the three and nine months ended September 30, 2021 and 2020, stock options, restricted stock awards, restricted stock units, and employee stock purchase plan to purchase 9.6 million shares of the Company’s common stock, respectively, were excluded from the diluted 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 this obligation may be settled in cash or shares at the Company’s option. The effect of potential share settlement is included in the diluted EPS calculation if the effect is more dilutive. During the three and nine months ended September 30, 2021 , the inclusion of the potential share settlement of the 2024 Notes was anti-dilutive. |
Basic and Diluted Net Loss Pe_2
Basic and Diluted Net Loss Per Share (Parenthetical) (Detail) - shares shares in Millions | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2021 | Sep. 30, 2020 | Sep. 30, 2021 | Sep. 30, 2020 | |
Earnings Per Share [Abstract] | ||||
Anti-dilutive securities excluded from computation of net loss per share | 9.6 | 9.6 | 9.6 | 9.6 |
Summary of Aggregate Non-Cancel
Summary of Aggregate Non-Cancelable Contractual Obligations Arising from Manufacturing Obligations (Detail) - Manufacturing Commitments Agreement [Member] - Catalent and Thermo [Member] $ in Thousands | Sep. 30, 2021USD ($) | |
Commitments And Contingencies [Line Items] | ||
2021 (October - December)) | $ 378,723 | |
2022 | 325,106 | |
2023 | 232,934 | |
2024 | 213,008 | |
2025 | 108,090 | |
Thereafter | 262,612 | |
Total manufacturing commitments | $ 1,520,473 | [1] |
[1] | Total manufacturing commitments include both the Catalent and Thermo manufacturing and supply agreements. Related to the leases at Catalent and Thermo, the Company has ROU assets and lease liabilities recorded on the unaudited condensed consolidated balance sheets as of September 30, 2021. For more information, please read Note 19, Leases of the Company’s Annual Report on Form 10-K for the year ended December 31, 2020. |
Subsequent Event - Additional I
Subsequent Event - Additional Information (Details) - Subsequent Event [Member] - Underwriting [Member] $ / shares in Units, $ in Millions | 1 Months Ended |
Oct. 31, 2021USD ($)$ / sharesshares | |
Subsequent Event [Line Items] | |
Share issued underwritten public offering | shares | 7,100,000 |
Share sold to underwriters | shares | 900,000 |
Offering price | $ / shares | $ 81 |
Proceeds from issuance of common stock | $ | $ 548.5 |
Net of commission and offering expenses | $ | $ 26.5 |