Document and Entity Information
Document and Entity Information - shares | 9 Months Ended | |
Sep. 30, 2023 | Oct. 27, 2023 | |
Cover [Abstract] | ||
Document Type | 10-Q | |
Amendment Flag | false | |
Document Period End Date | Sep. 30, 2023 | |
Document Fiscal Year Focus | 2023 | |
Document Fiscal Period Focus | Q3 | |
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 Current Reporting Status | Yes | |
Entity Interactive Data Current | Yes | |
Entity Shell Company | false | |
Entity File Number | 001-14895 | |
Entity Tax Identification Number | 93-0797222 | |
Entity Incorporation, State or Country Code | DE | |
Entity Address, Address Line One | 215 First Street | |
Entity Address, Address Line Two | Suite 415 | |
Entity Address, City or Town | Cambridge | |
Entity Address, State or Province | MA | |
Entity Address, Postal Zip Code | 02142 | |
City Area Code | 617 | |
Local Phone Number | 274-4000 | |
Title of 12(b) Security | Common Stock, $0.0001 par value per share | |
Trading Symbol | SRPT | |
Security Exchange Name | NASDAQ | |
Document Quarterly Report | true | |
Document Transition Report | false | |
Entity Common Stock, Shares Outstanding | 93,546,681 |
Condensed Consolidated Balance
Condensed Consolidated Balance Sheets - USD ($) $ in Thousands | Sep. 30, 2023 | Dec. 31, 2022 |
Current assets: | ||
Cash and cash equivalents, Fair value | $ 541,932 | $ 966,777 |
Short-term investments | 1,191,610 | 1,022,597 |
Accounts receivable | 318,855 | 214,628 |
Inventory | 244,011 | 203,968 |
Other current assets | 154,441 | 149,891 |
Total current assets | 2,450,849 | 2,557,861 |
Property and equipment, net | 212,367 | 180,037 |
Right of use assets | 133,454 | 64,954 |
Non-current inventory | 176,112 | 162,545 |
Other non-current assets | 136,925 | 162,969 |
Total assets | 3,109,707 | 3,128,366 |
Current liabilities: | ||
Accounts payable | 87,948 | 95,875 |
Accrued expenses | 322,350 | 418,996 |
Deferred revenue, current portion | 22,494 | 89,244 |
Other current liabilities | 17,951 | 15,489 |
Total current liabilities | 450,743 | 619,604 |
Long-term debt | 1,236,755 | 1,544,292 |
Lease liabilities, net of current portion | 134,752 | 57,578 |
Deferred revenue, net of current portion | 485,000 | 485,000 |
Contingent consideration | 38,100 | 36,900 |
Other non-current liabilities | 0 | 42 |
Total liabilities | 2,345,350 | 2,743,416 |
Commitments and contingencies (Note 16) | ||
Stockholders’ equity: | ||
Preferred stock, $0.0001 par value, 3,333,333 shares authorized; none issued and outstanding | 0 | 0 |
Common stock, $0.0001 par value, 198,000,000 shares authorized; 93,537,355 and 87,950,117 issued and outstanding at September 30, 2023, and December 31, 2022, respectively | 9 | 9 |
Additional paid-in capital | 5,256,854 | 4,296,841 |
Accumulated other comprehensive loss, net of tax | (638) | (1,664) |
Accumulated deficit | (4,491,868) | (3,910,236) |
Total stockholders’ equity | 764,357 | 384,950 |
Total liabilities and stockholders’ equity | $ 3,109,707 | $ 3,128,366 |
Condensed Consolidated Balanc_2
Condensed Consolidated Balance Sheets (Parenthetical) - $ / shares | Sep. 30, 2023 | Dec. 31, 2022 |
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, shares issued | 93,537,355 | 87,950,117 |
Common stock, shares outstanding | 93,537,355 | 87,950,117 |
Condensed Consolidated Statemen
Condensed Consolidated Statements of Operations and Comprehensive Loss - USD ($) shares in Thousands, $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2023 | Sep. 30, 2022 | Sep. 30, 2023 | Sep. 30, 2022 | |
Revenues: | ||||
Products, net | $ 309,322 | $ 207,774 | $ 779,805 | $ 607,836 |
Type of Revenue [Extensible List] | us-gaap:ProductMember | us-gaap:ProductMember | us-gaap:ProductMember | us-gaap:ProductMember |
Collaboration | $ 22,495 | $ 22,495 | $ 66,750 | $ 66,750 |
Total revenues | 331,817 | 230,269 | 846,555 | 674,586 |
Cost and expenses: | ||||
Cost of sales (excluding amortization of in-licensed rights) | 37,026 | 39,952 | 106,167 | 109,190 |
Research and development | 194,301 | 216,707 | 681,870 | 663,286 |
Selling, general and administrative | 120,893 | 104,787 | 350,171 | 330,943 |
Amortization of in-licensed rights | 439 | 178 | 796 | 535 |
Total cost and expenses | 352,659 | 361,624 | 1,139,004 | 1,103,954 |
Operating loss | (20,842) | (131,355) | (292,449) | (429,368) |
Other loss, net: | ||||
Gain from sale of Priority Review Voucher | 0 | 0 | 102,000 | 0 |
Loss on debt extinguishment | 0 | (125,441) | (387,329) | (125,441) |
Other income (expense), net | (12,332) | 378 | 17,309 | (33,848) |
Total other (loss), net | (12,332) | (125,063) | (268,020) | (159,289) |
Loss before income tax expense (benefit) | (33,174) | (256,418) | (560,469) | (588,657) |
Income tax expense (benefit) | 7,763 | 1,320 | 21,163 | 5,587 |
Net loss | (40,937) | (257,738) | (581,632) | (594,244) |
Other comprehensive income (loss): | ||||
Unrealized gains (losses) on investments, net of tax | 417 | (720) | 1,026 | (3,185) |
Total other comprehensive income (loss) | 417 | (720) | 1,026 | (3,185) |
Comprehensive loss | $ (40,520) | $ (258,458) | $ (580,606) | $ (597,429) |
Net loss per share - basic | $ (0.46) | $ (2.94) | $ (6.56) | $ (6.79) |
Net loss per share - diluted | $ (0.46) | $ (2.94) | $ (6.56) | $ (6.79) |
Weighted average number of shares of common stock used in computing basic and diluted net loss per share | 88,889 | 87,628 | 88,609 | 87,465 |
Weighted average number of shares of common stock used in computing basic and diluted net loss per share | 88,889 | 87,628 | 88,609 | 87,465 |
Condensed Consolidated Statem_2
Condensed Consolidated Statements of Stockholders' Equity - USD ($) shares in Thousands, $ in Thousands | Total | Common Stock [Member] | Additional Paid-In Capital [Member] | Accumulated Other Comprehensive (Loss) Income [Member] | Accumulated Deficit [Member] |
Balance at Dec. 31, 2021 | $ 928,009 | $ 9 | $ 4,134,768 | $ (20) | $ (3,206,748) |
Balance (in shares) at Dec. 31, 2021 | 87,127 | ||||
Exercise of options for common stock | 997 | 997 | |||
Exercise of options for common stock, shares | 18 | ||||
Vest of restricted stock units | 289 | ||||
Issuance of common stock under employee stock purchase plan | 3,993 | 3,993 | |||
Issuance of common stock under employee stock purchase plan, shares | 62 | ||||
Stock-based compensation | 29,198 | 29,198 | |||
Unrealized gains from available-for-sale securities, net of tax | (286) | (286) | |||
Net Income (Loss) | (105,025) | (105,025) | |||
Balance at Mar. 31, 2022 | 856,886 | $ 9 | 4,168,956 | (306) | (3,311,773) |
Balance (in shares) at Mar. 31, 2022 | 87,496 | ||||
Balance at Dec. 31, 2021 | 928,009 | $ 9 | 4,134,768 | (20) | (3,206,748) |
Balance (in shares) at Dec. 31, 2021 | 87,127 | ||||
Partial settlement of capped call share options for 2024 Notes | 26,317 | ||||
Purchase of capped call share options for 2027 Notes | 127,305 | ||||
Net Income (Loss) | (594,244) | ||||
Balance at Sep. 30, 2022 | 430,840 | $ 9 | 4,235,028 | (3,205) | (3,800,992) |
Balance (in shares) at Sep. 30, 2022 | 87,766 | ||||
Balance at Mar. 31, 2022 | 856,886 | $ 9 | 4,168,956 | (306) | (3,311,773) |
Balance (in shares) at Mar. 31, 2022 | 87,496 | ||||
Exercise of options for common stock | 339 | 339 | |||
Exercise of options for common stock, shares | 11 | ||||
Vest of restricted stock units | 28 | ||||
Stock-based compensation | 102,892 | 102,892 | |||
Unrealized gains from available-for-sale securities, net of tax | (2,179) | (2,179) | |||
Net Income (Loss) | (231,481) | (231,481) | |||
Balance at Jun. 30, 2022 | 726,457 | $ 9 | 4,272,187 | (2,485) | (3,543,254) |
Balance (in shares) at Jun. 30, 2022 | 87,535 | ||||
Exercise of options for common stock | 9,934 | 9,934 | |||
Exercise of options for common stock, shares | 144 | ||||
Vest of restricted stock units | 33 | ||||
Partial settlement of capped call share options for 2024 Notes | 26,317 | 26,317 | |||
Issuance of common stock under employee stock purchase plan | 3,477 | 3,477 | |||
Issuance of common stock under employee stock purchase plan, shares | 54 | ||||
Stock-based compensation | 50,418 | 50,418 | |||
Purchase of capped call share options for 2027 Notes | (127,305) | (127,305) | |||
Unrealized gains from available-for-sale securities, net of tax | (720) | (720) | |||
Net Income (Loss) | (257,738) | (257,738) | |||
Balance at Sep. 30, 2022 | 430,840 | $ 9 | 4,235,028 | (3,205) | (3,800,992) |
Balance (in shares) at Sep. 30, 2022 | 87,766 | ||||
Balance at Dec. 31, 2022 | 384,950 | $ 9 | 4,296,841 | (1,664) | (3,910,236) |
Balance (in shares) at Dec. 31, 2022 | 87,950 | ||||
Exercise of options for common stock | 22,808 | 22,808 | |||
Exercise of options for common stock, shares | 267 | ||||
Vest of restricted stock units | 390 | ||||
Issuance of common stock for exchange of 2024 Notes | 693,377 | 693,377 | |||
Issuance of common stock for exchange of 2024 Notes, shares | 4,456 | ||||
Partial settlement of capped call share options for 2024 Notes | 80,645 | 80,645 | |||
Issuance of common stock under employee stock purchase plan | 5,229 | 5,229 | |||
Issuance of common stock under employee stock purchase plan, shares | 77 | ||||
Stock-based compensation | 41,250 | 41,250 | |||
Unrealized gains from available-for-sale securities, net of tax | 1,245 | 1,245 | |||
Net Income (Loss) | (516,755) | (516,755) | |||
Balance at Mar. 31, 2023 | 712,749 | $ 9 | 5,140,150 | (419) | (4,426,991) |
Balance (in shares) at Mar. 31, 2023 | 93,140 | ||||
Balance at Dec. 31, 2022 | 384,950 | $ 9 | 4,296,841 | (1,664) | (3,910,236) |
Balance (in shares) at Dec. 31, 2022 | 87,950 | ||||
Partial settlement of capped call share options for 2024 Notes | 80,645 | ||||
Purchase of capped call share options for 2027 Notes | 0 | ||||
Net Income (Loss) | (581,632) | ||||
Balance at Sep. 30, 2023 | 764,357 | $ 9 | 5,256,854 | (638) | (4,491,868) |
Balance (in shares) at Sep. 30, 2023 | 93,537 | ||||
Balance at Mar. 31, 2023 | 712,749 | $ 9 | 5,140,150 | (419) | (4,426,991) |
Balance (in shares) at Mar. 31, 2023 | 93,140 | ||||
Exercise of options for common stock | 5,861 | 5,861 | |||
Exercise of options for common stock, shares | 80 | ||||
Vest of restricted stock units | 54 | ||||
Stock-based compensation | 47,377 | 47,377 | |||
Unrealized gains from available-for-sale securities, net of tax | (636) | (636) | |||
Net Income (Loss) | (23,940) | (23,940) | |||
Balance at Jun. 30, 2023 | 741,411 | $ 9 | 5,193,388 | (1,055) | (4,450,931) |
Balance (in shares) at Jun. 30, 2023 | 93,274 | ||||
Exercise of options for common stock | 9,873 | 9,873 | |||
Exercise of options for common stock, shares | 142 | ||||
Vest of restricted stock units | 45 | ||||
Issuance of common stock under employee stock purchase plan | 5,532 | 5,532 | |||
Issuance of common stock under employee stock purchase plan, shares | 76 | ||||
Stock-based compensation | 48,061 | 48,061 | |||
Unrealized gains from available-for-sale securities, net of tax | 417 | 417 | |||
Net Income (Loss) | (40,937) | (40,937) | |||
Balance at Sep. 30, 2023 | $ 764,357 | $ 9 | $ 5,256,854 | $ (638) | $ (4,491,868) |
Balance (in shares) at Sep. 30, 2023 | 93,537 |
Condensed Consolidated Statem_3
Condensed Consolidated Statements of Cash Flows (Unaudited) - USD ($) $ in Thousands | 9 Months Ended | |
Sep. 30, 2023 | Sep. 30, 2022 | |
Cash flows from operating activities: | ||
Net loss | $ (581,632) | $ (594,244) |
Adjustments to reconcile net loss to cash flows from operating activities: | ||
Loss on debt extinguishment | 387,329 | 125,441 |
Impairment of equity investments | 27,821 | 0 |
Gain from sale of Priority Review Voucher | (102,000) | 0 |
Depreciation and amortization | 33,025 | 31,311 |
Reduction in the carrying amounts of the right of use assets | 10,636 | 9,182 |
Non-cash interest expense | 3,919 | 6,027 |
Stock-based compensation | 136,688 | 182,508 |
Accretion of Investment Discount, Net | (32,847) | (4,518) |
Other | 1,524 | (584) |
Changes in operating assets and liabilities, net: | ||
Net increase in accounts receivable | (104,227) | (48,519) |
Net increase in inventory | (53,610) | (41,900) |
Net decrease in other assets | 5,011 | 34,398 |
Net decrease in deferred revenue | (66,750) | (66,750) |
Net (decrease) increase in accounts payable, accrued expenses, lease liabilities and other liabilities | (111,223) | 134,694 |
Net cash used in operating activities | (446,336) | (232,954) |
Cash flows from investing activities: | ||
Proceeds from sale of Priority Review Voucher | 102,000 | 0 |
Purchase of property and equipment | (57,067) | (22,860) |
Purchase of available-for-sale securities | (1,475,404) | (1,509,316) |
Maturity of available-for-sale securities | 1,340,265 | 476,722 |
Purchase of intangible assets | (11,239) | (1,225) |
Net cash used in investing activities | (101,445) | (1,056,679) |
Cash flows from financing activities: | ||
Partial settlement of capped call share options for 2024 Notes | 80,645 | 26,317 |
Proceeds from exercise of stock options and purchase of stock under the Employee Stock Purchase Program | 49,303 | 18,740 |
Debt conversion costs for 2024 Notes | (6,887) | 0 |
Proceeds from 2027 Notes offering, net of commissions | 0 | 1,127,400 |
Debt issuance costs for 2027 Notes | 0 | (162) |
Repayment of principal amount due under 2019 Term Loan | 0 | (550,000) |
Payment on debt extinguishment of 2019 Term Loan | 0 | (25,364) |
Repurchase of 2024 Notes | 0 | (247,868) |
Purchase of capped call share options for 2027 Notes | 0 | (127,305) |
Net cash provided by financing activities | 123,061 | 221,758 |
Decrease in cash, cash equivalents and restricted cash | (424,720) | (1,067,875) |
Cash, cash equivalents and restricted cash: | ||
Beginning of period | 985,801 | 2,125,523 |
End of period | 561,081 | 1,057,648 |
Reconciliation of cash, cash equivalents and restricted cash: | ||
Total cash, cash equivalents and restricted cash | 561,081 | 1,057,648 |
Cash and cash equivalents, Fair value | 541,932 | 1,038,624 |
Restricted cash in other assets | 19,149 | 19,024 |
Supplemental disclosure of cash flow information: | ||
Cash paid during the period for income taxes | 9,569 | 1,356 |
Cash paid during the period for interest | 15,129 | 41,273 |
Supplemental schedule of non-cash investing and financing activities: | ||
Intangible assets and property and equipment included in accounts payable and accrued expenses | 16,059 | 5,511 |
Lease liabilities arising from obtaining right of use assets | 79,846 | 12,281 |
Common stock issued for exchange of 2024 Notes | 693,377 | 0 |
Accrued debt issuance costs for 2027 Notes | 0 | 553 |
Lease liabilities terminated | $ 0 | $ 3,807 |
Pay vs Performance Disclosure
Pay vs Performance Disclosure - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||||||
Sep. 30, 2023 | Jun. 30, 2023 | Mar. 31, 2023 | Sep. 30, 2022 | Jun. 30, 2022 | Mar. 31, 2022 | Sep. 30, 2023 | Sep. 30, 2022 | |
Pay vs Performance Disclosure | ||||||||
Net Income (Loss) | $ (40,937) | $ (23,940) | $ (516,755) | $ (257,738) | $ (231,481) | $ (105,025) | $ (581,632) | $ (594,244) |
Insider Trading Arrangements
Insider Trading Arrangements | 9 Months Ended |
Sep. 30, 2023 | |
Trading Arrangements, by Individual | |
Rule 10b5-1 Arrangement Adopted | false |
Non-Rule 10b5-1 Arrangement Adopted | false |
Rule 10b5-1 Arrangement Terminated | false |
Non-Rule 10b5-1 Arrangement Terminated | false |
ORGANIZATION AND NATURE OF BUSI
ORGANIZATION AND NATURE OF BUSINESS | 9 Months Ended |
Sep. 30, 2023 | |
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 has developed multiple approved products for the treatment of Duchenne muscular dystrophy (“Duchenne”) and is developing potential therapeutic candidates for a broad range of diseases and disorders, including Duchenne, Limb-girdle muscular dystrophies (“LGMDs”) and other neuromuscular and central nervous system (“CNS”) disorders. The Company's products in the U.S., EXONDYS 51 (eteplirsen) Injection (“EXONDYS 51”), VYONDYS 53 (golodirsen) Injection (“VYONDYS 53”), AMONDYS 45 (casimersen) Injection (“AMONDYS 45”) and ELEVIDYS, were granted accelerated approval by the U.S. Food and Drug Administration (the “FDA”) on September 19, 2016, December 12, 2019, February 25, 2021 and June 22, 2023, 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. ELEVIDYS addresses the root genetic cause of Duchenne mutations in the dystrophin gene that result in the lack of dystrophin protein by delivering a gene that codes for a shortened form of dystrophin to muscle cells known as ELEVIDYS micro-dystrophin. As of September 30, 2023, the Company had approximately $ 1,752.6 million of cash, cash equivalents, restricted cash and investments, consisting of $ 541.9 million of cash and cash equivalents, $ 1,191.6 million of short-term investments and $ 19.1 million of long-term restricted cash. 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 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 | 9 Months Ended |
Sep. 30, 2023 | |
Accounting Policies [Abstract] | |
SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES | 2. SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES 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 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, 2022, which are contained in the Company’s Annual Report on Form 10-K for the year ended December 31, 2022, filed with the U.S. Securities and Exchange Commission on February 28, 2023. The results for the three and nine months ended September 30, 2023 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 cash held at financial institutions, cash equivalents, investments and accounts receivable from customers. As of September 30, 2023, the Company’s cash was concentrated at three financial institutions, 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. The Company also purchases commercial paper, government and government agency bonds, corporate bonds and certificates of deposit issued by highly rated corporations, financial institutions and governments and limits the amount of credit exposure to any one issuer. These amounts may at times exceed federally insured limits. The Company has not experienced any credit losses related to these financial instruments and does not believe to be exposed to any significant credit risk related to these instruments. Please refer to Note 7, Product Revenues, Net, Accounts Receivable and Reserves for Product Revenues for discussion of the credit risk associated with accounts receivable from customers. Significant Accounting Policies For details about the Company's accounting policies, please read Note 2, Summary of Significant Accounting Policies of the Annual Report on Form 10-K for the year ended December 31, 2022. There have not been any material changes to the Company's accounting policies through September 30, 2023 . |
LICENSE AND COLLABORATION AGREE
LICENSE AND COLLABORATION AGREEMENTS | 9 Months Ended |
Sep. 30, 2023 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
LICENSE AND COLLABORATION AGREEMENTS | 3. LICENSE AND COLLABORATION AGREEMENTS F. Hoffman-La Roche Ltd. For both the three and nine months ended September 30, 2023 and 2022, the Company recognized $ 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”). As of September 30, 2023, the Company had total deferred revenue of $ 507.5 million associated with the Roche Agreement, of which $ 22.5 million is 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 as of September 30, 2023 and December 31, 2022. 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, 2023, costs reimbursable by Roche and reflected as a reduction to operating expenses were $ 34.9 million and $ 83.4 million, respectively. For the three and nine months ended September 30, 2022, costs reimbursable by Roche and reflected as a reduction to operating expenses were $ 22.0 million and $ 66.1 million, respectively. As of September 30, 2023, there was $ 35.4 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 their 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 connection with the FDA approval of ELEVIDYS in June 2023, the Company recorded a milestone payment of $ 10.0 million to Nationwide as an in-licensed right intangible asset which is being amortized on a straight-line basis over the remaining life of the relevant patents and has a carrying value of approximately $ 9.8 million as of September 30, 2023. The Company also achieved a certain sales-based milestone related to this license agreement with Nationwide as of September 30, 2023 . As such, the Company recorded $ 2.0 million as an in-licensed right intangible asset in its unaudited condensed consolidated balance sheets as of September 30, 2023 . The in-licensed right is being amortized on a straight-line basis over the remaining life of the relevant patents and has a carrying value of approximately $ 2.0 million as of September 30, 2023. Royalty payments due to Nationwide associated with commercial sales of ELEVIDYS totaled $ 2.1 million for the three and nine months ended September 30, 2023 and were recorded as cost of sales in the accompanying unaudited condensed consolidated statements of operations and comprehensive loss. 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 $ 36.1 million in research milestone payments. Under these agreements, there are $ 221.3 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 exercised and additional license agreements are executed, the Company would incur additional contingent obligations and may be required to make development, regulatory, and sales milestone payments and royalty payments based on the net sales of the developed products upon commercialization. During the nine months ended September 30, 2023 , the Company exercised an option in a research and option agreement and recognized $ 7.5 million of up-front payment as research and development expense in the accompanying unaudited condensed consolidated statements of operations and comprehensive loss. During the three and nine months ended September 30, 2022 , the Company exercised options in an additional research and option agreement and recognized $ 5.0 million of up-front expense as research and development expense in the accompanying unaudited condensed consolidated statements of operations and comprehensive loss. For the three and nine months ended September 30, 2023, the Company recognized $ 3.8 million of research milestone expense in the accompanying unaudited condensed consolidated statements of operations and comprehensive loss. For the nine months ended September 30, 2022 , the Company recognized $ 6.0 million of research, option and milestone expense in the accompanying unaudited condensed consolidated statements of operations and comprehensive loss. 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, 2023, the Company may be obligated to make up to $ 3.2 billion of future development, regulatory, commercial and up-front royalty payments associated with its collaboration and license 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, 2023, which are discussed above. For the three and nine months ended September 30, 2023, the Company recognized up-front, development milestone and other expenses of $ 3.8 million and $ 11.9 million, respectively, as research and development expense in the accompanying unaudited condensed consolidated statements of operations and comprehensive loss. For the three and nine months ended September 30, 2022 , the Company recognized up-front and development milestone expenses of $ 5.5 million and $ 14.3 million, respectively, as research and development expense in the accompanying unaudited condensed consolidated statements of operations and comprehensive loss. |
GAIN FROM SALE OF PRIORITY REVI
GAIN FROM SALE OF PRIORITY REVIEW VOUCHER | 9 Months Ended |
Sep. 30, 2023 | |
Goodwill and Intangible Assets Disclosure [Abstract] | |
GAIN FROM SALE OF PRIORITY REVIEW VOUCHER | 4. GAIN FROM SALE OF PRIORITY REVIEW VOUCHER In June 2023, the Company entered into an agreement to sell the rare pediatric disease Priority Review Voucher (“ELEVIDYS PRV”) it received from the FDA in connection with the approval of ELEVIDYS for consideration of $ 102.0 million, with no commission costs. The closing of the transaction was not subject to the conditions set forth under the Hart-Scott-Rodino Antitrust Improvements Act of 1976, as amended, and closed in June 2023. The net proceeds were recorded as a gain from sale of the ELEVIDYS PRV during the nine months ended September 30, 2023 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, 2023 | |
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 and twelve months ended September 30, 2023 and December 31, 2022, there were no transfers into or out of Level 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 the valuation techniques it utilizes to determine such fair value: Fair Value Measurement as of September 30, 2023 Total Level 1 Level 2 Level 3 (in thousands) Assets Money market funds $ 139,682 $ 139,682 $ — $ — Commercial paper 88,450 — 88,450 — Government and government agency bonds 989,257 — 989,257 — Corporate bonds 145,898 — 145,898 — Strategic investments 7,500 — — 7,500 Certificates of deposit 45,066 — 45,066 — Total assets $ 1,415,853 $ 139,682 $ 1,268,671 $ 7,500 Liabilities Contingent consideration $ 38,100 $ — $ — $ 38,100 Total liabilities $ 38,100 $ — $ — $ 38,100 Fair Value Measurement as of December 31, 2022 Total Level 1 Level 2 Level 3 (in thousands) Assets Money market funds $ 467,553 $ 467,553 $ — $ — Commercial paper 211,369 — 211,369 — Government and government agency bonds 807,540 — 807,540 — Corporate bonds 125,741 — 125,741 — Strategic investments 31,321 321 — 31,000 Certificates of deposit 42,745 — 42,745 — Total assets $ 1,686,269 $ 467,874 $ 1,187,395 $ 31,000 Liabilities Contingent consideration $ 36,900 $ — $ — $ 36,900 Total liabilities $ 36,900 $ — $ — $ 36,900 The Company’s assets with a fair value categorized as Level 1 within the fair value hierarchy primarily include money market funds. The Company's assets with a fair value categorized as Level 2 within the fair value hierarchy consist of commercial paper, government and government agency bonds, corporate bonds and certificates of deposit. These assets have been initially valued at the transaction price and subsequently valued at the end of each reporting period utilizing third-party pricing services. The Company uses observable market inputs to determine value, which primarily consist of reportable trades. Certain of the short-term investments with maturities of less than three months at the date of acquisition are presented as cash equivalents on the unaudited condensed consolidated balance sheets as of September 30, 2023. 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, 2022 $ 31,000 Additions 4,000 Changes in estimated fair value ( 27,500 ) Fair value, as of September 30, 2023 $ 7,500 As of (in thousands) Fair value, as of December 31, 2021 $ 32,412 Additions 163 Fair value, as of September 30, 2022 $ 32,575 The Company’s assets with a 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 three other private companies. For more information related to Lacerta, please read Note 3, License and Collaboration Agreements to the financial statements included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2022. The fair value of the Lacerta investment was initially based on a cost approach corroborated by the Black-Scholes-Merton option-pricing model. The most significant assumptions in the initial 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 private companies are recorded at fair value at the time of purchase as measured by their respective investment cost. Included in the Company's strategic investments is a $ 4.0 million strategic investment in a private biotechnology company made during the three and nine months ended September 30, 2023, which was included in other non-current assets within the unaudited condensed consolidated balance sheets as of September 30, 2023. At the end of each reporting period, the fair value of the Company's strategic investments will be adjusted if the issuers were to issue similar or identical securities or when there is a triggering event for impairment. Equity securities without a readily determinable fair value are written down to its fair value if a qualitative assessment indicates that the investment is impaired and the fair value of the investment is less than its carrying value. During the three and nine months ended September 30, 2023, the Company recorded an impairment loss of $ 27.5 million related to the Company's strategic investment in Series A preferred stock of Lacerta after comparing the fair value of the Lacerta strategic investment to its carrying value. The Company's assessment performed during the three and nine months ended September 30, 2023 considered entity-specific impairment indicators, such as the future business prospects of Lacerta's existing programs and expected future cash flows. The fair value as of September 30, 2023 was estimated based on a range of future cash flows that are expected to be realized by the Company, which could range from $ 0 to $ 2.5 million and reflects a Level 3 fair value measurement given the significant unobservable inputs. The impairment loss is included in Other (expense) income, net within the unaudited condensed consolidated statements of operations and comprehensive loss. There were no additional valuation measurement events related to the fair value of the Company's Level 3 strategic investments during the three and nine months ended September 30, 2023 or 2022, as no additional impairment indicators were identified nor were similar securities issued. The Company’s contingent consideration liability with a 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 to the financial statements included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2022 . 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. This fair value measurement was based upon significant inputs not observable in the market and therefore represented a Level 3 measurement. 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, 2022 $ 36,900 Change in estimated fair value 2,000 Liabilities terminated ( 800 ) Fair value, as of September 30, 2023 $ 38,100 As of (in thousands) Fair value, as of December 31, 2021 $ 43,600 Change in estimated fair value ( 6,700 ) Fair value, as of September 30, 2022 $ 36,900 For the three and nine months ended September 30, 2023, the Company recorded a net increase of $ 1.2 million and $ 2.0 million to account for the change in fair value of the Company's existing contingent consideration liabilities. Thes e changes, which are recorded through earnings, were a result of updates made to certain inputs and assumptions impacting the probability-weighted expected cash flows, principally the discount rate utilized to determine the present value of future payments to be made, the probability of success of the underlying programs and the approval date of the underlying programs, offset by the termination of a license agreement with an academic institution that included an embedded derivative. As of September 30, 2023, the remaining 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 immediate or short-term maturity of these financial instruments. For fair value information related to the Company's debt facilities, please read Note 11, Indebtedness. |
CASH, CASH EQUIVALENTS AND MARK
CASH, CASH EQUIVALENTS AND MARKETABLE SECURITIES | 9 Months Ended |
Sep. 30, 2023 | |
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 $ 139,682 $ 467,553 Corporate bonds 10,526 3,157 Government and government agency bonds 66,535 128,451 Commercial paper — 33,190 Total $ 216,743 $ 632,351 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 both September 30, 2023 and December 31, 2022 was approximately four months . The following tables summarize the Company ’s cash, cash equivalents and short-term investments for each of the periods indicated: As of September 30, 2023 Amortized Gross Gross Fair (in thousands) Cash and money market funds $ 464,871 $ — $ — $ 464,871 Commercial paper 88,450 — — 88,450 Government and government agency bonds 989,797 41 ( 581 ) 989,257 Corporate bonds 145,969 16 ( 87 ) 145,898 Certificates of deposit 45,066 — — 45,066 Total cash, cash equivalents and investments $ 1,734,153 $ 57 $ ( 668 ) $ 1,733,542 As reported: Cash and cash equivalents $ 541,926 $ 8 $ ( 2 ) $ 541,932 Short-term investments 1,192,227 49 ( 666 ) 1,191,610 Total cash, cash equivalents and investments $ 1,734,153 $ 57 $ ( 668 ) $ 1,733,542 As of December 31, 2022 Amortized Gross Gross Fair (in thousands) Cash and money market funds $ 801,979 $ — $ — $ 801,979 Commercial paper 211,369 — — 211,369 Government and government agency bonds 808,904 178 ( 1,542 ) 807,540 Corporate bonds 126,014 9 ( 282 ) 125,741 Certificates of deposit 42,745 — — 42,745 Total cash, cash equivalents and investments $ 1,991,011 $ 187 $ ( 1,824 ) $ 1,989,374 As reported: Cash and cash equivalents $ 966,768 $ 9 $ — $ 966,777 Short-term investments 1,024,243 178 ( 1,824 ) 1,022,597 Total cash, cash equivalents and investments $ 1,991,011 $ 187 $ ( 1,824 ) $ 1,989,374 |
PRODUCT REVENUES, NET, ACCOUNTS
PRODUCT REVENUES, NET, ACCOUNTS RECEIVABLE AND RESERVES FOR PRODUCT REVENUES | 9 Months Ended |
Sep. 30, 2023 | |
Receivables, Net, Current [Abstract] | |
PRODUCT REVENUES, NET, ACCOUNTS RECEIVABLE AND RESERVES FOR PRODUCT REVENUES | 7. PRODUCT REVENUES, NET, ACCOUNTS RECEIVABLE AND RESERVES FOR PRODUCT REVENUES Net product revenues, which includes revenues associated with EXONDYS 51, AMONDYS 45 and VYONDYS 53 (collectively, the “PMO Products”) and ELEVIDYS consisted of the following: For the Three Months Ended For the Nine Months Ended 2023 2022 2023 2022 (in thousands) PMO Products United States $ 198,069 $ 188,346 $ 602,415 $ 548,355 Rest of World 42,145 19,428 108,282 59,481 Total PMO product revenues, net $ 240,214 $ 207,774 $ 710,697 $ 607,836 ELEVIDYS United States 69,108 — 69,108 — Total ELEVIDYS product revenues, net $ 69,108 $ — $ 69,108 $ — Total product revenues, net $ 309,322 $ 207,774 $ 779,805 $ 607,836 The Company considers there to be revenue concentration risks for regions where net product revenues exceed 10 % of consolidated net product revenues. No individual rest of world country exceeded 10 % of total net product revenues for the three and nine months ended September 30, 2023 and 2022. Three individual customers accounted for 40 % , 23 % and 9 % of product revenues, net, for the three months ended September 30, 2023 and 45 % , 29 % and 8 % for the nine months ended September 30, 2023. Three individual customers accounted for 48 % , 35 % and 7 % of net product revenues for the three months ended September 30, 2022 and 49 % , 34 % and 7 % of net product revenues for the nine months ended September 30, 2022. The concentration of the Company’s net product revenues within a particular country may have a material adverse effect on the Company’s revenues and results of operations if sales in the respective regions experience difficulties. As of September 30, 2023 and December 31, 2022, the Company's accounts receivable were $ 318.9 million and $ 214.6 million, respectiv ely, both of which were related to product sales receivable, net of discounts and allowances. As of September 30, 2023, 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 100 days. Outside of the U.S., the majority of the Company’s customers have payment terms ranging between 60 and 150 days . Three individual customers accounted for 29 % , 25 % and 12 % of accounts receivable from product sales as of September 30, 2023 and 36 %, 35 % and 12 % of accounts receivable from product sales as of December 31, 2022. As of September 30, 2023, the Company believes that such customers are of high credit quality and has not experienced any material credit losses related to such customers. 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, 2022 $ 417 $ 67,493 $ 3,343 $ 23,445 $ 94,698 Provision 18,191 94,025 11,251 52,500 175,967 Adjustments relating to prior year 110 ( 4,154 ) — — ( 4,044 ) Payments/credits ( 9,386 ) ( 73,317 ) ( 10,586 ) ( 40,048 ) ( 133,337 ) Balance, as of September 30, 2023 $ 9,332 $ 84,047 $ 4,008 $ 35,897 $ 133,284 Chargebacks Rebates Prompt Pay Other Accruals Total (in thousands) Balance, as of December 31, 2021 $ 799 $ 60,506 $ 2,798 $ 6,363 $ 70,466 Provision 8,756 80,956 9,508 28,809 128,029 Adjustments relating to prior year — ( 4,111 ) — 30 ( 4,081 ) Payments/credits ( 9,193 ) ( 70,645 ) ( 8,930 ) ( 18,397 ) ( 107,165 ) Balance, as of September 30, 2022 $ 362 $ 66,706 $ 3,376 $ 16,805 $ 87,249 The following table summarizes the total reserves above included in the Company’s unaudited condensed consolidated balance sheets for each of the periods indicated: As of As of (in thousands) Reduction to accounts receivable $ 47,606 $ 25,914 Component of accrued expenses 85,678 68,784 Total reserves $ 133,284 $ 94,698 |
INVENTORY
INVENTORY | 9 Months Ended |
Sep. 30, 2023 | |
Inventory Disclosure [Abstract] | |
INVENTORY | 8. INVENTORY The following table summarizes the components of the Company’s inventory for each of the periods indicated: As of As of (in thousands) Raw materials $ 114,622 $ 59,181 Work in progress 258,258 269,185 Finished goods 47,243 38,147 Total inventory $ 420,123 $ 366,513 Non-current inventory consists of raw materials and work in progress that is anticipated to be consumed beyond the Company's normal operating cycle. The following table summarizes the balance sheet classification of the Company's inventory for each of the periods indicated: As of As of (in thousands) Balance sheet classification Inventory $ 244,011 $ 203,968 Non-current inventory 176,112 162,545 Total inventory $ 420,123 $ 366,513 |
OTHER ASSETS
OTHER ASSETS | 9 Months Ended |
Sep. 30, 2023 | |
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 $ 77,125 $ 66,455 Collaboration receivable 35,408 41,758 Prepaid maintenance services 12,282 9,815 Prepaid clinical and pre-clinical expenses 9,247 11,237 Prepaid commercial expenses 2,793 2,947 Prepaid research expenses 2,372 1,927 Prepaid insurance 1,545 3,717 Other 13,669 12,035 Total other current assets $ 154,441 $ 149,891 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 $ 78,842 $ 97,409 Intangible assets, net 19,501 7,578 Restricted cash 19,149 19,024 Strategic investments 7,500 31,321 Prepaid maintenance services 5,948 3,403 Prepaid clinical expenses 2,219 2,150 Other 3,766 2,084 Total other non-current assets $ 136,925 $ 162,969 |
ACCRUED EXPENSES
ACCRUED EXPENSES | 9 Months Ended |
Sep. 30, 2023 | |
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) Product revenue related reserves $ 85,678 $ 68,784 Accrued contract manufacturing costs 60,998 202,173 Accrued employee compensation costs 51,143 65,946 Accrued clinical and pre-clinical costs 40,930 28,884 Accrued income taxes 24,677 12,521 Accrued professional fees 24,050 12,061 Accrued royalties 10,501 8,636 Accrued property and equipment purchases 9,382 984 Accrued milestone and license costs 3,750 7,702 Accrued research costs 3,516 1,629 Other 7,725 9,676 Total accrued expenses $ 322,350 $ 418,996 |
INDEBTEDNESS
INDEBTEDNESS | 9 Months Ended |
Sep. 30, 2023 | |
Debt Disclosure [Abstract] | |
INDEBTEDNESS | 11. INDEBTEDNESS 2024 Convertible Notes and 2017 Capped Call Transactions On November 14, 2017, the Company issued $ 570.0 million aggregate principal amount of senior convertible notes due on November 15, 2024 (the “2024 Notes”) and, simultaneously, entered into capped call transactions with counterparties intended to minimize the impact of potential dilution upon conversion of the 2024 Notes (the “2017 Capped Calls”). On September 14, 2022, the Company entered into separate, privately negotiated transactions to repurchase a portion of the outstanding 2024 Notes. The holders exchanged $ 150.6 million in aggregate principal value of 2024 Notes held by them for an aggregate payment of $ 248.6 million for full settlement of the principal value and accrued interest on such date. As a result of the repurchases, the Company entered into agreements with the 2017 Capped Calls counterparties to terminate a corresponding portion of the 2017 Capped Calls and received $ 26.3 million in cash. On March 2, 2023, the Company entered into separate, privately negotiated exchange agreements with certain holders of the outstanding 2024 Notes (the “Exchange Agreements”). The Exchange Agreements resulted in an exchange of $ 313.5 million in aggregate principal value of the 2024 Notes for shares of the Company ’ s common stock (the “2024 Notes Exchange”), which closed on March 7, 2023 (the “Exchange Date”). In connection with the 2024 Notes Exchange, the Company issued approximately 4.5 million shares of the Company's common stock representing an agreed upon contractual exchange rate under each of the Exchange Agreements. The fair value of these shares issued was approximately $ 693.4 million. The Company also incurred approximately $ 6.9 million in third-party debt conversion costs. The exchange was not pursuant to the conversion privileges included in the terms of the debt at issuance and therefore was accounted for as a debt extinguishment. The Company accounted for the debt extinguishment by recognizing the difference between the fair value of the shares of common stock transferred on the Exchange Date and the net carrying amount of the extinguished debt as a loss on debt extinguishment. Accordingly, on the Exchange Date, the Company: (i) reduced the carrying value of the 2024 Notes by $ 311.5 million, (ii) eliminated accrued interest of $ 1.5 million, and (iii) recorded a loss on debt extinguishment of $ 387.3 million, inclusive of the $ 6.9 million in third-party debt conversion costs, which is included in the unaudited condensed consolidated statement of operations and comprehensive loss. The outstanding principal balance of the 2024 Notes as of September 30, 2023 is approximately $ 105.8 million, which is convertible into approximatel y 1.4 million sh ares of Company common stock. As a result of the exchange, the Company entered into agreements with the counterparties to the 2017 Capped Calls to terminate a portion of the 2017 Capped Calls in a notional amount corresponding to the principal amount of the 2024 Notes exchanged through the 2024 Notes Exchange and received approximately $ 80.6 million in cash from the counterparties, which was included in additional paid-in capital within the unaudited condensed consolidated balance sheets as of September 30, 2023. For additional details about the 2024 Notes, please read Note 13, Indebtedness to the financial statements included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2022. Total Debt Obligations As of September 30, 2023 and December 31, 2022, the Company recorded approximately $ 1,236.8 million and $ 1,544.3 million as long-term debt on the unaudited condensed consolidated balance sheets, respectively. The following table summarizes the Company’s debt facilities for each of the periods indicated: As of As of (in thousands) Principal amount of the 2024 Notes $ 105,847 $ 419,371 Principal amount of the 2027 Notes 1,150,000 1,150,000 Unamortized discount - debt issuance costs of 2024 Notes ( 465 ) ( 3,059 ) Unamortized discount - debt issuance costs of 2027 Notes ( 18,627 ) ( 22,020 ) Total carrying value of debt facilities $ 1,236,755 $ 1,544,292 Fair value of 2024 Notes $ 189,297 $ 765,046 Fair value of 2027 Notes 1,294,900 1,308,482 Total fair value of debt facilities $ 1,484,197 $ 2,073,528 For the three months ended September 30, 2023 and 2022, contractual interest expense from debt facilities was $ 5.2 million and $ 14.7 million, inclusive of $ 1.2 million and $ 2.0 million of amortization of debt discounts, respectively. For the nine months ended September 30, 2023 and 2022, contractual interest expense from debt facilities was $ 16.8 million and $ 46.5 million, inclusive of $ 3.9 million and $ 6.0 million of amortization of debt discounts, respectively. The fair value of the 1.25 % convertible senior notes due on September 15, 2027 (“2027 Notes”) and the 2024 Notes is based on open market trades and is classified as Level 1 in the fair value hierarchy. The following table summarizes the total principal and contractual interest payments due under the Company’s debt arrangements: As of September 30, 2023 Principal Interest Total Payments (in thousands) 2023 (October-December) $ — $ 794 $ 794 2024 105,847 15,963 121,810 2025 — 14,375 14,375 2026 — 14,375 14,375 2027 1,150,000 14,375 1,164,375 Total payments $ 1,255,847 $ 59,882 $ 1,315,729 |
STOCK-BASED COMPENSATION
STOCK-BASED COMPENSATION | 9 Months Ended |
Sep. 30, 2023 | |
Share-Based Payment Arrangement [Abstract] | |
STOCK-BASED COMPENSATION | 12. 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, 2023 2022 2023 2022 Grants Weighted Grants Weighted Grants Weighted Grants Weighted Stock options 50,875 $ 66.53 127,100 $ 59.84 1,135,799 $ 72.23 1,626,220 $ 47.54 Restricted stock units 27,539 $ 118.05 65,657 $ 105.28 1,137,202 (1) $ 153.02 925,167 (2) $ 80.66 (1) Included in restricted stock units (“RSUs”) for the nine months ended September 30, 2023 are 502,225 shares with performance conditions (the “March 2023 PSUs”) which are related to regulatory approval of certain of the Company's product candidates and achievement of a certain financial performance target. (2) Included in the RSUs for the nine months ended September 30, 2022 are 38,500 sh ares of PSUs (the “March 2022 PSUs”) with performance conditions related to regulatory approval of the Company's product candidates. As a result of the regulatory approval of ELEVIDYS in June 2023 and the achievement of the Company's financial performance target during the three months ended September 30, 2023 , the Company recorded stock-based compensation expense of $ 10.3 million and $ 17.7 million associated with the March 2023 PSUs during the three and nine months ended September 30, 2023, respectively. Vesting of the March 2023 PSUs is contingent on the fulfillment of remaining service conditions. The maximum remaining expense associated with the March 2023 PSUs, excluding forfeitures, is $ 62.2 million. The associated expense will be recognized over approximately the next 1.5 years. As of September 30, 2023, the performance conditions for the March 2022 PSUs were deemed as not probable of being achieved and, as such, no stock-based compensation related to these PSUs was recognized. If the performance conditions of the March 2022 PSUs are met within the required time frame, the Company may recognize up to $ 3.3 million of stock-based compensation expense, excluding forfeitures. Stock options and the remaining RSUs granted during the periods presented in the table have only service-based criteria and vest over four years . Grant Modification In June 2017, the Company granted its Chief Executive Officer 3,300,000 options with service and market conditions which were subject to a five-year cliff vesting schedule. On April 19, 2022 (the “Effective Date”), the Company entered into an agreement with its Chief Executive Officer to modify the vesting conditions of the options. Under the agreement, one-third of the options vested (the “Vested Tranche”) on the Effective Date with no required service or market conditions. Subject to the Chief Executive Officer's continued service through each applicable vesting date and the compound annual growth rate of the Company's common stock exceeding that of the Nasdaq Biotech Index in varying percentages, the remaining two-thirds of the options (the “Unvested Tranche”) shall vest in varying increments at any time between the Effective Date and June 26, 2025 (the “Measurement Period”) when (and if) the average of the closing price of the Company’s common stock during any consecutive 20 trading day period during the Measurement Period reaches certain pre-determined target stock prices. The Unvested Tranche represents awards with market conditions only. Both the pre- and post-modification fair values for the Unvested Tranche are determined by a lattice model with Monte Carlo simulations. The incremental compensation costs related to varying increments of the Unvested Tranche will be recognized as stock-based compensation expense over their respective derived service periods, an output from the Monte Carlo simulation, and will be fully recognized over a 1.3 year period from the Effective Date. During the nine months ended September 30, 2023 , 550,110 optio ns relating to the Unvested Tranche met the conditions for vesting as the average closing price of the Company's common stock exceeded $ 128.65 during 20 consecutive trading days in March 2023 and the compound annual growth rate of the Company's common stock exceeded that of the Nasdaq Biotech Index by greater than 5 % . For the three and nine months ended September 30, 2023, the Company recorded $ 0.6 million and $ 13.4 million of stock-based compensation expense in total related to the Chief Executive Officer's awards, respectively. For the three and nine months ended September 30, 2022 , the Company recorded $ 20.5 million and $ 94.2 million of stock-based compensation expense in total related to the Chief Executive Officer's awards, respectively. As of September 30, 2023, the Company has recognized all incremental compensation costs associated with the Unvested Tranche. Stock-based Compensation Expense For the three months ended September 30, 2023 and 2022, total stock-based compensation expense was $ 48.1 million and $ 50.4 million, respectively. For the nine months ended September 30, 2023 and 2022, total stock-based compensation expense was $ 136.7 million and $ 182.5 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 2023 2022 2023 2022 (in thousands) Research and development $ 22,325 $ 14,795 $ 60,315 $ 42,330 Selling, general and administrative 25,736 35,623 76,373 140,178 Total stock-based compensation expense $ 48,061 $ 50,418 $ 136,688 $ 182,508 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 2023 2022 2023 2022 (in thousands) Stock options $ 17,471 $ 35,686 $ 62,436 $ 141,247 Restricted stock units 28,924 13,321 70,101 37,064 Employee stock purchase plan 1,666 1,411 4,151 4,197 Total stock-based compensation expense $ 48,061 $ 50,418 $ 136,688 $ 182,508 |
OTHER LOSS, NET
OTHER LOSS, NET | 9 Months Ended |
Sep. 30, 2023 | |
Other Income and Expenses [Abstract] | |
OTHER LOSS, NET | 13. OTHER LOSS, NET The following table summarizes other loss, net f or each of the periods indicated: For the Three Months Ended For the Nine Months Ended 2023 2022 2023 2022 (in thousands) Accretion of investment discount, net $ 12,984 $ 3,435 $ 35,809 $ 4,808 Interest income 9,838 4,780 27,532 7,362 Interest expense ( 5,229 ) ( 14,736 ) ( 16,776 ) ( 46,560 ) (Loss) gain on contingent consideration, net ( 2,000 ) 6,700 ( 1,200 ) 6,700 Impairment of equity investments ( 27,500 ) — ( 27,821 ) — Other, net ( 425 ) 199 ( 235 ) ( 6,158 ) Other (expense) income, net $ ( 12,332 ) $ 378 $ 17,309 $ ( 33,848 ) Gain from sale of Priority Review Voucher — — 102,000 — Loss on debt extinguishment — ( 125,441 ) ( 387,329 ) ( 125,441 ) Total other loss, net $ ( 12,332 ) $ ( 125,063 ) $ ( 268,020 ) $ ( 159,289 ) |
LEASES
LEASES | 9 Months Ended |
Sep. 30, 2023 | |
Leases [Abstract] | |
LEASES | 14. LEASES The Company has real estate operating leases in Cambridge, Andover, Burlington and Bedford, Massachusetts, Dublin and Columbus, Ohio, and Durham, North Carolina that provide for scheduled annual rent increases over the lease term. The Company has also identified leases embedded in certain of its manufacturing and supply agreements as the Company determined that it controls the use of the facilities and related equipment therein. For more information related to the lease embedded in manufacturing and supply agreements with Catalent, Inc. (“Catalent”), please refer to Note 21, Commitments and Contingencies of the Company's Annual Report on Form 10-K for the year ended December 31, 2022. Bedford, Massachusetts On April 22, 2022, the Company entered into a lease agreement (the “Bedford Lease”) for 288,000 square feet of to-be-constructed research and development and manufacturing space in Bedford, Massachusetts. The term of the Bedford Lease commences upon the landlord’s completion of the initial construction of the core and shell of the building, at which time the Company will obtain control of the premises and commence internal construction activities. The Company has two options to extend the lease for a period of ten years each, exercisable under certain conditions and at a market rate determined in accordance with the lease agreement. In May 2022, in connection with the execution of the Bedford Lease, the Company issued a letter of credit collateralized by cash deposits of approximately $ 8.4 million, which was included in the other non-current assets of the Company’s unaudited condensed consolidated balance sheets. Such letter of credit shall be reduced to approximately $ 5.6 million at the commencement of the fourth rent year, provided certain conditions set forth in the Bedford Lease are satisfied. Undiscounted minimum rent payments due over the term of the lease aggregate to $ 307.4 million. Additionally, the Company is responsible for reimbursing the landlord for the Company’s share of the property’s operating expenses and property taxes. The Bedford Lease also provides for a tenant improvement allowance from the landlord of up to $ 72.0 million to be used towards costs incurred by the Company in the design and construction of the premises. The Bedford Lease commenced in May 2023 as the Company obtained control of the premises (the “Bedford Lease Commencement”). The Company has a lease liability and right-of-use (“ROU”) asset of $ 84.1 million and $ 71.7 million, respectively, on its unaudited condensed consolidated balance sheets as of September 30, 2023 related to the Bedford Lease. The Company recorded the $ 72.0 million tenant improvement allowance as a reduction to right-of-use assets and lease liabilities at the date of the Bedford Lease Commencement. Tenant improvement costs incurred by the Company that had been reimbursed by the landlord tot aled $ 6.1 million as of September 30, 2023 and are recorded as an increase to lease liabilities within the Company's unaudited condensed consolidated balance sheets. The initial gross ROU assets and lease liabilities recorded were based on the present value of estimated future payments associated with the Bedford Lease at a discount rate of 9.5 %, representing the rate at which the Company could borrow on a collateralized basis the amount of the lease payments in a similar term. The remaining lease term for the Bedford Lease is approximately 15.3 years. For the three and nine months ended September 30, 2023 , the Company had no operating lease costs and variable lease costs totaled less than $ 0.1 million related to the Bedford Lease. |
NET LOSS PER SHARE
NET LOSS PER SHARE | 9 Months Ended |
Sep. 30, 2023 | |
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. Given that the Company recorded a net loss for each of the periods presented, there is no difference between basic and diluted net loss per share since the effect of common stock equivalents would be anti-dilutive and are, therefore, excluded from the diluted net loss per share calculation. For the Three Months Ended For the Nine Months Ended 2023 2022 2023 2022 (in thousands, except per share amounts) Net loss $ ( 40,937 ) $ ( 257,738 ) $ ( 581,632 ) $ ( 594,244 ) Weighted-average common shares outstanding - basic 88,889 87,628 88,609 87,465 Effect of dilutive securities* — — — — Weighted-average common shares outstanding - diluted 88,889 87,628 88,609 87,465 Net loss per share - basic and diluted $ ( 0.46 ) $ ( 2.94 ) $ ( 6.56 ) $ ( 6.79 ) * For the three and nine months ended September 30, 2023 and 2022, stock options, RSUs and employee stock purchase plan to purchase of approximately 12.1 million and 11.2 million shares of 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 its 2027 Notes and 2024 Notes on diluted net earnings per share (“EPS”) 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 dilutive. During the three and nine months ended September 30, 2023 and 2022, the inclusion of the potential share settlement of the 2027 Notes and the 2024 Notes was anti-dilutive. Accordingly, the potential conversion o f approximately 1.4 million and 5.7 million shares related to the 2024 Notes has been excluded from the computation of diluted net loss per share for the three and nine months ended September 30, 2023 and 2022, respectively, and the potential conversion of approximatel y 8.1 million shar es related to the 2027 Notes has been excluded from the computation of diluted net loss per share for the three and nine months ended September 30, 2023 and 2022, respectively. |
COMMITMENTS AND CONTINGENCIES
COMMITMENTS AND CONTINGENCIES | 9 Months Ended |
Sep. 30, 2023 | |
Commitments and Contingencies Disclosure [Abstract] | |
COMMITMENTS AND CONTINGENCIES | 16. 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) 2023 (October-December) $ 380,762 2024 762,237 2025 151,525 2026 80,110 2027 77,497 Thereafter 72,244 Total manufacturing commitments* $ 1,524,375 * Total manufacturing commitments includes the Catalent manufacturing and supply agreement, for which the Company has ROU assets and lease liabilities recorded on the unaudited condensed consolidated balance sheets as of September 30, 2023. For more information, please read Note 21, Commitments and Contingencies to the financial statements included in the Company’ s Annual Report on Form 10-K for the year ended December 31, 2022. Thermo Fisher Scientific, Inc. The Company entered into a development, commercial manufacturing, and supply agreement in June 2018 and, subsequently, entered into the first, second and third amendments in May 2019, July 2020 and October 2021, respectively, with Brammer Bio MA, LLC, an affiliate of Thermo Fisher Scientific, Inc. (“Thermo”) (collectively, the “Thermo Agreement”). In March 2023, the Company executed a fourth amendment (the “Amendment”) that modified the terms of the Thermo Agreement. The Amendment removed the previous minimum batch purchase commitment of $ 54.7 million per annum and associated fee for the remaining term of the Thermo Agreement. In connection with the elimination of such commitment and fee, the Amendment implemented a fee of up to $ 60.0 million, to be paid in three installments of $ 20.0 million each by March 1, 2024, December 31, 2024 and December 31, 2025, respectively, unless waived in part as described below. The Company will recognize the first $ 20.0 million installment due March 1, 2024 as a nonrefundable advance payment over the term of the agreement as the Company believes it will receive future benefit from this contract. As the Company has yet to obtain regulatory approval to produce commercial supply of ELEVIDYS at Thermo manufacturing facilities as of September 30, 2023 , it recognized approximately $ 0.8 million and $ 1.7 million as research and development expense during the three and nine months ended September 30, 2023 related to this nonrefundable advanced payment. The second and third payment installments, which are associated with the years ending December 31, 2024 and 2025, will be waived if the Company meets certain minimum purchase thresholds under the Amendment. As of September 30, 2023, the Company believes it is probable that the minimum purchase thresholds will be met in the normal course of business throughout the term of the agreement and, therefore, no liabilities were recorded related to the second or third payment installments. For more information related to Thermo, please read Note 21, Commitments and Contingencies to the financial statements included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2022 . Litigation In the normal course of business, the Company from time to time is named as a party to various legal claims, actions and complaints, which have included or may include 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. The Company has not recorded any material accruals for loss contingencies and in management's opinion no material range of loss is estimable for the matters described below as of September 30, 2023. On September 15, 2020, REGENXBIO INC. (“Regenx”) and the Trustees of the University of Pennsylvania (“U-Penn”) filed a lawsuit against the Company and Sarepta Therapeutics Three, LLC, 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 the patented cultured host cell technology allegedly used to make adeno-associated virus (“AAV”) gene therapy products, including SRP-9001 (approved June 22, 2023 in the U.S. as ELEVIDYS ® ). 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 the ‘617 Patent asserted by Regenx. Plaintiffs seek injunctive relief, a judgment of infringement and willful infringement, an unspecified amount of damages that is no less than a reasonable royalty (treble damages), attorneys’ fees and costs, and such other relief as the court deems just and proper. On January 4, 2022, the Court denied Sarepta’s motion 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). Sarepta answered the Complaint on January 18, 2022, and a case schedule has been set with a trial commencing on January 29, 2024. The parties have filed cross motions for summary judgement, which are fully briefed. On June 20, 2023, Regenx and U-Penn commenced a second patent infringement lawsuit against Sarepta and its contract manufacturer, Catalent asserting patent alleged infringement of U. S. Patent No. 11,680,274 (“the ’274 Patent”). In the second lawsuit, Regenx and U-Penn allege that Sarepta and Catalent’s manufacture, use and commercial launch of ELEVIDYS ® (formerly/also known as SRP-9001) infringe the ’274 Patent. Sarepta answered the complaint on August 10, 2023, and a case schedule has been set with a trial commencing on November 17, 2025. 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 in which Sarepta sought 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 and willful infringement of each of the NS Patents allegedly arising from Sarepta’s activities, including the sale of, its exon 53 skipping product, VYONDYS 53 (golodirsen). NS further seeks a determination of non-infringement by NS alleged to arise from NS’s activities, including the sale of, its exon 53 skipping product, Viltepso (viltolarsen) and invalidity of certain patents licensed to the Company from University of Western Australia (“UWA”) (U.S. Patent Nos. 9,994,851, 10,227,590, and 10,266,827, collectively the “UWA Patents”). NS is seeking legal fees and costs, an unspecified amount of monetary relief (treble damages) attributed to Sarepta’s alleged infringement, and such other relief as the court deems just and proper. In January 2022, the PTAB granted institution of all claims of all NS Patents in response to Sarepta’s IPR Petitions and determined that Sarepta has demonstrated a reasonable likelihood of success in proving that the NS Patents are unpatentable. NS filed a motion for preliminary injunction solely seeking Sarepta’s withdrawal of the IPR Petitions, which was ultimately granted after the U.S. Court of Appeals for the Federal Circuit reversed and remanded to the district court on February 8, 2022. Sarepta subsequently withdrew the IPRs, which were terminated on June 14, 2022. On December 27, 2021, the district court partially granted and denied the motion to dismiss by Sarepta and ordered NS to file a Second Amended Complaint (“SAC”), which it did on January 14, 2022. In the SAC, NS maintains all claims of the original complaint of July 13, 2021, except a determination of non-infringement of the UWA Patents. On January 28, 2022, Sarepta filed its answer to the SAC, with defenses and counterclaims against NS and NS Pharma Inc. that include infringement of the UWA Patents allegedly arising from their activities concerning, including the sale of, its exon 53 skipping product, Viltepso (viltolarsen) and breach of contract. Sarepta is also seeking a determination of invalidity of the NS Patents. Sarepta is seeking an award of relief in its defenses to NS’ allegations, a judgment of breach of contract, a determination of invalidity of the NS Patents, a judgment of infringement and willful infringement of the UWA Patents, legal fees and costs, an unspecified amount of monetary relief (treble damages) attributable to NS’ alleged infringement, and such other relief as the court deems just and proper. UWA has since been joined as a Plaintiff in Sarepta’s counterclaims against NS. On August 14, 2023, the Court granted cross motions to amend the pleadings, allowing Sarepta to add a counterclaim against NS for inequitable conduct, and NS to add counterclaims against Sarepta for inequitable conduct and Walker Process fraud. The Court entered a scheduling order with a trial scheduled to commence on May 13, 2024. On or about June 5, 2023, Sarepta initiated a patent infringement lawsuit against Nippon Shinyaku in Japan, alleging that NS’s production, sales and offers to sell Viltepso infringe Sarepta’s Japanese Patent No. 6406782. NS filed its preliminary answer on July 13, 2023. Thereafter, the Court set an initial case schedule, with a final hearing set for July 11, 2024. |
SUMMARY OF SIGNIFICANT ACCOUN_2
SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES (Policies) | 9 Months Ended |
Sep. 30, 2023 | |
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 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, 2022, which are contained in the Company’s Annual Report on Form 10-K for the year ended December 31, 2022, filed with the U.S. Securities and Exchange Commission on February 28, 2023. The results for the three and nine months ended September 30, 2023 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 cash held at financial institutions, cash equivalents, investments and accounts receivable from customers. As of September 30, 2023, the Company’s cash was concentrated at three financial institutions, 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. The Company also purchases commercial paper, government and government agency bonds, corporate bonds and certificates of deposit issued by highly rated corporations, financial institutions and governments and limits the amount of credit exposure to any one issuer. These amounts may at times exceed federally insured limits. The Company has not experienced any credit losses related to these financial instruments and does not believe to be exposed to any significant credit risk related to these instruments. Please refer to Note 7, Product Revenues, Net, Accounts Receivable and Reserves for Product Revenues for discussion of the credit risk associated with accounts receivable from customers. |
Significant Accounting Policies | Significant Accounting Policies For details about the Company's accounting policies, please read Note 2, Summary of Significant Accounting Policies of the Annual Report on Form 10-K for the year ended December 31, 2022. There have not been any material changes to the Company's accounting policies through September 30, 2023 . |
FAIR VALUE MEASUREMENTS (Tables
FAIR VALUE MEASUREMENTS (Tables) | 9 Months Ended |
Sep. 30, 2023 | |
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 the valuation techniques it utilizes to determine such fair value: Fair Value Measurement as of September 30, 2023 Total Level 1 Level 2 Level 3 (in thousands) Assets Money market funds $ 139,682 $ 139,682 $ — $ — Commercial paper 88,450 — 88,450 — Government and government agency bonds 989,257 — 989,257 — Corporate bonds 145,898 — 145,898 — Strategic investments 7,500 — — 7,500 Certificates of deposit 45,066 — 45,066 — Total assets $ 1,415,853 $ 139,682 $ 1,268,671 $ 7,500 Liabilities Contingent consideration $ 38,100 $ — $ — $ 38,100 Total liabilities $ 38,100 $ — $ — $ 38,100 Fair Value Measurement as of December 31, 2022 Total Level 1 Level 2 Level 3 (in thousands) Assets Money market funds $ 467,553 $ 467,553 $ — $ — Commercial paper 211,369 — 211,369 — Government and government agency bonds 807,540 — 807,540 — Corporate bonds 125,741 — 125,741 — Strategic investments 31,321 321 — 31,000 Certificates of deposit 42,745 — 42,745 — Total assets $ 1,686,269 $ 467,874 $ 1,187,395 $ 31,000 Liabilities Contingent consideration $ 36,900 $ — $ — $ 36,900 Total liabilities $ 36,900 $ — $ — $ 36,900 |
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, 2022 $ 31,000 Additions 4,000 Changes in estimated fair value ( 27,500 ) Fair value, as of September 30, 2023 $ 7,500 As of (in thousands) Fair value, as of December 31, 2021 $ 32,412 Additions 163 Fair value, as of September 30, 2022 $ 32,575 |
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, 2022 $ 36,900 Change in estimated fair value 2,000 Liabilities terminated ( 800 ) Fair value, as of September 30, 2023 $ 38,100 As of (in thousands) Fair value, as of December 31, 2021 $ 43,600 Change in estimated fair value ( 6,700 ) Fair value, as of September 30, 2022 $ 36,900 |
CASH, CASH EQUIVALENTS AND MA_2
CASH, CASH EQUIVALENTS AND MARKETABLE SECURITIES (Tables) | 9 Months Ended |
Sep. 30, 2023 | |
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 $ 139,682 $ 467,553 Corporate bonds 10,526 3,157 Government and government agency bonds 66,535 128,451 Commercial paper — 33,190 Total $ 216,743 $ 632,351 |
Summary of Company Cash, Cash Equivalents and Short-Term Investments | The following tables summarize the Company ’s cash, cash equivalents and short-term investments for each of the periods indicated: As of September 30, 2023 Amortized Gross Gross Fair (in thousands) Cash and money market funds $ 464,871 $ — $ — $ 464,871 Commercial paper 88,450 — — 88,450 Government and government agency bonds 989,797 41 ( 581 ) 989,257 Corporate bonds 145,969 16 ( 87 ) 145,898 Certificates of deposit 45,066 — — 45,066 Total cash, cash equivalents and investments $ 1,734,153 $ 57 $ ( 668 ) $ 1,733,542 As reported: Cash and cash equivalents $ 541,926 $ 8 $ ( 2 ) $ 541,932 Short-term investments 1,192,227 49 ( 666 ) 1,191,610 Total cash, cash equivalents and investments $ 1,734,153 $ 57 $ ( 668 ) $ 1,733,542 As of December 31, 2022 Amortized Gross Gross Fair (in thousands) Cash and money market funds $ 801,979 $ — $ — $ 801,979 Commercial paper 211,369 — — 211,369 Government and government agency bonds 808,904 178 ( 1,542 ) 807,540 Corporate bonds 126,014 9 ( 282 ) 125,741 Certificates of deposit 42,745 — — 42,745 Total cash, cash equivalents and investments $ 1,991,011 $ 187 $ ( 1,824 ) $ 1,989,374 As reported: Cash and cash equivalents $ 966,768 $ 9 $ — $ 966,777 Short-term investments 1,024,243 178 ( 1,824 ) 1,022,597 Total cash, cash equivalents and investments $ 1,991,011 $ 187 $ ( 1,824 ) $ 1,989,374 |
PRODUCT REVENUES, NET, ACCOUN_2
PRODUCT REVENUES, NET, ACCOUNTS RECEIVABLE AND RESERVES FOR PRODUCT REVENUES (Tables) | 9 Months Ended |
Sep. 30, 2023 | |
Receivables, Net, Current [Abstract] | |
Summary of Product Revenues | Net product revenues, which includes revenues associated with EXONDYS 51, AMONDYS 45 and VYONDYS 53 (collectively, the “PMO Products”) and ELEVIDYS consisted of the following: For the Three Months Ended For the Nine Months Ended 2023 2022 2023 2022 (in thousands) PMO Products United States $ 198,069 $ 188,346 $ 602,415 $ 548,355 Rest of World 42,145 19,428 108,282 59,481 Total PMO product revenues, net $ 240,214 $ 207,774 $ 710,697 $ 607,836 ELEVIDYS United States 69,108 — 69,108 — Total ELEVIDYS product revenues, net $ 69,108 $ — $ 69,108 $ — Total product revenues, net $ 309,322 $ 207,774 $ 779,805 $ 607,836 |
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, 2022 $ 417 $ 67,493 $ 3,343 $ 23,445 $ 94,698 Provision 18,191 94,025 11,251 52,500 175,967 Adjustments relating to prior year 110 ( 4,154 ) — — ( 4,044 ) Payments/credits ( 9,386 ) ( 73,317 ) ( 10,586 ) ( 40,048 ) ( 133,337 ) Balance, as of September 30, 2023 $ 9,332 $ 84,047 $ 4,008 $ 35,897 $ 133,284 Chargebacks Rebates Prompt Pay Other Accruals Total (in thousands) Balance, as of December 31, 2021 $ 799 $ 60,506 $ 2,798 $ 6,363 $ 70,466 Provision 8,756 80,956 9,508 28,809 128,029 Adjustments relating to prior year — ( 4,111 ) — 30 ( 4,081 ) Payments/credits ( 9,193 ) ( 70,645 ) ( 8,930 ) ( 18,397 ) ( 107,165 ) Balance, as of September 30, 2022 $ 362 $ 66,706 $ 3,376 $ 16,805 $ 87,249 |
Summary of Total Reserves Included in Consolidated Balance Sheets | The following table summarizes the total reserves above included in the Company’s unaudited condensed consolidated balance sheets for each of the periods indicated: As of As of (in thousands) Reduction to accounts receivable $ 47,606 $ 25,914 Component of accrued expenses 85,678 68,784 Total reserves $ 133,284 $ 94,698 |
INVENTORY (Tables)
INVENTORY (Tables) | 9 Months Ended |
Sep. 30, 2023 | |
Inventory Disclosure [Abstract] | |
Summary of Components of Inventory | The following table summarizes the components of the Company’s inventory for each of the periods indicated: As of As of (in thousands) Raw materials $ 114,622 $ 59,181 Work in progress 258,258 269,185 Finished goods 47,243 38,147 Total inventory $ 420,123 $ 366,513 |
Summary Of Inventory Balance Sheet Classification | The following table summarizes the balance sheet classification of the Company's inventory for each of the periods indicated: As of As of (in thousands) Balance sheet classification Inventory $ 244,011 $ 203,968 Non-current inventory 176,112 162,545 Total inventory $ 420,123 $ 366,513 |
OTHER ASSETS (Tables)
OTHER ASSETS (Tables) | 9 Months Ended |
Sep. 30, 2023 | |
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 $ 77,125 $ 66,455 Collaboration receivable 35,408 41,758 Prepaid maintenance services 12,282 9,815 Prepaid clinical and pre-clinical expenses 9,247 11,237 Prepaid commercial expenses 2,793 2,947 Prepaid research expenses 2,372 1,927 Prepaid insurance 1,545 3,717 Other 13,669 12,035 Total other current assets $ 154,441 $ 149,891 |
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 $ 78,842 $ 97,409 Intangible assets, net 19,501 7,578 Restricted cash 19,149 19,024 Strategic investments 7,500 31,321 Prepaid maintenance services 5,948 3,403 Prepaid clinical expenses 2,219 2,150 Other 3,766 2,084 Total other non-current assets $ 136,925 $ 162,969 |
ACCRUED EXPENSES (Tables)
ACCRUED EXPENSES (Tables) | 9 Months Ended |
Sep. 30, 2023 | |
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) Product revenue related reserves $ 85,678 $ 68,784 Accrued contract manufacturing costs 60,998 202,173 Accrued employee compensation costs 51,143 65,946 Accrued clinical and pre-clinical costs 40,930 28,884 Accrued income taxes 24,677 12,521 Accrued professional fees 24,050 12,061 Accrued royalties 10,501 8,636 Accrued property and equipment purchases 9,382 984 Accrued milestone and license costs 3,750 7,702 Accrued research costs 3,516 1,629 Other 7,725 9,676 Total accrued expenses $ 322,350 $ 418,996 |
INDEBTEDNESS (Tables)
INDEBTEDNESS (Tables) | 9 Months Ended |
Sep. 30, 2023 | |
Debt Disclosure [Abstract] | |
Summary of Debt Facilities | The following table summarizes the Company’s debt facilities for each of the periods indicated: As of As of (in thousands) Principal amount of the 2024 Notes $ 105,847 $ 419,371 Principal amount of the 2027 Notes 1,150,000 1,150,000 Unamortized discount - debt issuance costs of 2024 Notes ( 465 ) ( 3,059 ) Unamortized discount - debt issuance costs of 2027 Notes ( 18,627 ) ( 22,020 ) Total carrying value of debt facilities $ 1,236,755 $ 1,544,292 Fair value of 2024 Notes $ 189,297 $ 765,046 Fair value of 2027 Notes 1,294,900 1,308,482 Total fair value of debt facilities $ 1,484,197 $ 2,073,528 |
Summarizes Total Gross Payments Due under Company's Debt Arrangements | The following table summarizes the total principal and contractual interest payments due under the Company’s debt arrangements: As of September 30, 2023 Principal Interest Total Payments (in thousands) 2023 (October-December) $ — $ 794 $ 794 2024 105,847 15,963 121,810 2025 — 14,375 14,375 2026 — 14,375 14,375 2027 1,150,000 14,375 1,164,375 Total payments $ 1,255,847 $ 59,882 $ 1,315,729 |
STOCK-BASED COMPENSATION (Table
STOCK-BASED COMPENSATION (Tables) | 9 Months Ended |
Sep. 30, 2023 | |
Share-Based Payment Arrangement [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, 2023 2022 2023 2022 Grants Weighted Grants Weighted Grants Weighted Grants Weighted Stock options 50,875 $ 66.53 127,100 $ 59.84 1,135,799 $ 72.23 1,626,220 $ 47.54 Restricted stock units 27,539 $ 118.05 65,657 $ 105.28 1,137,202 (1) $ 153.02 925,167 (2) $ 80.66 (1) Included in restricted stock units (“RSUs”) for the nine months ended September 30, 2023 are 502,225 shares with performance conditions (the “March 2023 PSUs”) which are related to regulatory approval of certain of the Company's product candidates and achievement of a certain financial performance target. (2) Included in the RSUs for the nine months ended September 30, 2022 are 38,500 sh ares of PSUs (the “March 2022 PSUs”) with performance conditions related to regulatory approval of the Company's product candidates. As a result of the regulatory approval of ELEVIDYS in June 2023 and the achievement of the Company's financial performance target during the three months ended September 30, 2023 , the Company recorded stock-based compensation expense of $ 10.3 million and $ 17.7 million associated with the March 2023 PSUs during the three and nine months ended September 30, 2023, respectively. Vesting of the March 2023 PSUs is contingent on the fulfillment of remaining service conditions. The maximum remaining expense associated with the March 2023 PSUs, excluding forfeitures, is $ 62.2 million. The associated expense will be recognized over approximately the next 1.5 years. As of September 30, 2023, the performance conditions for the March 2022 PSUs were deemed as not probable of being achieved and, as such, no stock-based compensation related to these PSUs was recognized. If the performance conditions of the March 2022 PSUs are met within the required time frame, the Company may recognize up to $ 3.3 million of stock-based compensation expense, excluding forfeitures. Stock options and the remaining RSUs granted during the periods presented in the table have only service-based criteria and vest over four years . |
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 2023 2022 2023 2022 (in thousands) Research and development $ 22,325 $ 14,795 $ 60,315 $ 42,330 Selling, general and administrative 25,736 35,623 76,373 140,178 Total stock-based compensation expense $ 48,061 $ 50,418 $ 136,688 $ 182,508 |
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 2023 2022 2023 2022 (in thousands) Stock options $ 17,471 $ 35,686 $ 62,436 $ 141,247 Restricted stock units 28,924 13,321 70,101 37,064 Employee stock purchase plan 1,666 1,411 4,151 4,197 Total stock-based compensation expense $ 48,061 $ 50,418 $ 136,688 $ 182,508 |
OTHER LOSS, NET (Tables)
OTHER LOSS, NET (Tables) | 9 Months Ended |
Sep. 30, 2023 | |
Other Income and Expenses [Abstract] | |
Summary of Other Loss | The following table summarizes other loss, net f or each of the periods indicated: For the Three Months Ended For the Nine Months Ended 2023 2022 2023 2022 (in thousands) Accretion of investment discount, net $ 12,984 $ 3,435 $ 35,809 $ 4,808 Interest income 9,838 4,780 27,532 7,362 Interest expense ( 5,229 ) ( 14,736 ) ( 16,776 ) ( 46,560 ) (Loss) gain on contingent consideration, net ( 2,000 ) 6,700 ( 1,200 ) 6,700 Impairment of equity investments ( 27,500 ) — ( 27,821 ) — Other, net ( 425 ) 199 ( 235 ) ( 6,158 ) Other (expense) income, net $ ( 12,332 ) $ 378 $ 17,309 $ ( 33,848 ) Gain from sale of Priority Review Voucher — — 102,000 — Loss on debt extinguishment — ( 125,441 ) ( 387,329 ) ( 125,441 ) Total other loss, net $ ( 12,332 ) $ ( 125,063 ) $ ( 268,020 ) $ ( 159,289 ) |
NET LOSS PER SHARE (Tables)
NET LOSS PER SHARE (Tables) | 9 Months Ended |
Sep. 30, 2023 | |
Earnings Per Share [Abstract] | |
Basic and Diluted Net Loss Per Share | Given that the Company recorded a net loss for each of the periods presented, there is no difference between basic and diluted net loss per share since the effect of common stock equivalents would be anti-dilutive and are, therefore, excluded from the diluted net loss per share calculation. For the Three Months Ended For the Nine Months Ended 2023 2022 2023 2022 (in thousands, except per share amounts) Net loss $ ( 40,937 ) $ ( 257,738 ) $ ( 581,632 ) $ ( 594,244 ) Weighted-average common shares outstanding - basic 88,889 87,628 88,609 87,465 Effect of dilutive securities* — — — — Weighted-average common shares outstanding - diluted 88,889 87,628 88,609 87,465 Net loss per share - basic and diluted $ ( 0.46 ) $ ( 2.94 ) $ ( 6.56 ) $ ( 6.79 ) * For the three and nine months ended September 30, 2023 and 2022, stock options, RSUs and employee stock purchase plan to purchase of approximately 12.1 million and 11.2 million shares of 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 its 2027 Notes and 2024 Notes on diluted net earnings per share (“EPS”) 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 dilutive. During the three and nine months ended September 30, 2023 and 2022, the inclusion of the potential share settlement of the 2027 Notes and the 2024 Notes was anti-dilutive. Accordingly, the potential conversion o f approximately 1.4 million and 5.7 million shares related to the 2024 Notes has been excluded from the computation of diluted net loss per share for the three and nine months ended September 30, 2023 and 2022, respectively, and the potential conversion of approximatel y 8.1 million shar es related to the 2027 Notes has been excluded from the computation of diluted net loss per share for the three and nine months ended September 30, 2023 and 2022, respectively. |
COMMITMENTS AND CONTINGENCIES (
COMMITMENTS AND CONTINGENCIES (Tables) | 9 Months Ended |
Sep. 30, 2023 | |
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) 2023 (October-December) $ 380,762 2024 762,237 2025 151,525 2026 80,110 2027 77,497 Thereafter 72,244 Total manufacturing commitments* $ 1,524,375 * Total manufacturing commitments includes the Catalent manufacturing and supply agreement, for which the Company has ROU assets and lease liabilities recorded on the unaudited condensed consolidated balance sheets as of September 30, 2023. For more information, please read Note 21, Commitments and Contingencies to the financial statements included in the Company’ s Annual Report on Form 10-K for the year ended December 31, 2022. |
Organization and Nature of Bu_2
Organization and Nature of Business - Additional Information (Detail) - USD ($) $ in Thousands | Sep. 30, 2023 | Dec. 31, 2022 | Sep. 30, 2022 |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |||
Cash, cash equivalents and investments | $ 1,752,600 | ||
Cash and cash equivalents, Fair value | 541,932 | $ 966,777 | $ 1,038,624 |
Short-term investments | 1,191,610 | 1,022,597 | |
Restricted cash and investments | $ 19,149 | $ 19,024 |
Summary of Significant Accoun_3
Summary of Significant Accounting Policies - Additional Information (Detail) | 9 Months Ended |
Sep. 30, 2023 Segment | |
Summary Of Significant Accounting Policies [Line Items] | |
Number of operating segments | 1 |
License and Collaboration Agr_2
License and Collaboration Agreements - Additional Information (Detail) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | |||
Sep. 30, 2023 | Sep. 30, 2022 | Sep. 30, 2023 | Sep. 30, 2022 | Dec. 31, 2022 | |
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||
Product revenues, net | $ 309,322 | $ 207,774 | $ 779,805 | $ 607,836 | |
Cost of sales (excluding amortization of in-licensed rights) | 37,026 | 39,952 | 106,167 | 109,190 | |
Development Milestone and Upfront Fee Recognized as Research and Development Expense | 3,800 | 5,500 | 11,900 | 14,300 | |
Collaborative Arrangement | |||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||
Payment for option exercise | 221,300 | ||||
Research, option and milestone expense | 3,800 | 6,000 | 3,800 | 6,000 | |
Collaborative Arrangement | Maximum [Member] | |||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||
Contingent research milestone payments | 36,100 | ||||
Development Milestone and Settlement Upfront Fee Recognized as Research and Development Expense | 3,200,000 | 3,200,000 | |||
Collaborative Arrangement | Research and Development Expense [Member] | |||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||
Up-front and development milestone expenses | 5,000 | 7,500 | 5,000 | ||
Collaborative Arrangement | Roche Holding A.G. [Member] | |||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||
Product revenues, net | 22,500 | 66,800 | 22,500 | 66,800 | |
Deferred Revenue | 507,500 | 507,500 | |||
Deferred Revenue, Current | 22,500 | 22,500 | |||
Deferred Revenue Separate Material Options Right | 485,000 | 485,000 | $ 485,000 | ||
Research and development expense | 34,900 | $ 22,000 | 83,400 | $ 66,100 | |
Collaboration Receivable | 35,400 | 35,400 | |||
Nationwide License Agreement [Member] | |||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||
Licensed Right Intangible Asset | 2,000 | 2,000 | |||
Carrying value | 2,000 | 2,000 | |||
Cost of sales (excluding amortization of in-licensed rights) | 2,100 | 2,100 | |||
Nationwide License Agreement [Member] | Nationwide Childrens Hospital [Member] | |||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||
Sales milestone payment recorded as an in-license right | 9,800 | 9,800 | |||
Carrying value | $ 10,000 | $ 10,000 |
Gain from Sale of Priority Re_2
Gain from Sale of Priority Review Voucher - Additional Information (Detail) $ in Millions | 9 Months Ended |
Sep. 30, 2023 USD ($) | |
F D A [Member] | |
Gain From Sale Of Intangible Asset [Line Items] | |
Proceeds from sale of rare pediatric disease priority review voucher, Net of commission | $ 102 |
Fair Value Measurements - Asset
Fair Value Measurements - Assets and Liabilities Measured and Carried at Fair Value (Detail) - USD ($) $ in Thousands | Sep. 30, 2023 | Dec. 31, 2022 | Sep. 30, 2022 | Dec. 31, 2021 |
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||||
Total assets | $ 1,415,853 | $ 1,686,269 | ||
Contingent consideration | 38,100 | 36,900 | ||
Total liabilities | 38,100 | 36,900 | ||
Money Market Funds [Member] | ||||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||||
Total assets | 139,682 | 467,553 | ||
Commercial Paper [Member] | ||||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||||
Total assets | 88,450 | 211,369 | ||
Government and Government Agency Bonds [Member] | ||||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||||
Total assets | 989,257 | 807,540 | ||
Corporate Bonds [Member] | ||||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||||
Total assets | 145,898 | 125,741 | ||
Strategic Equity Investments [Member] | ||||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||||
Total assets | 7,500 | 31,321 | ||
Certificates of Deposit [Member] | ||||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||||
Total assets | 45,066 | 42,745 | ||
Level 1 [Member] | ||||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||||
Total assets | 139,682 | 467,874 | ||
Level 1 [Member] | Money Market Funds [Member] | ||||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||||
Total assets | 139,682 | 467,553 | ||
Level 1 [Member] | Strategic Equity Investments [Member] | ||||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||||
Total assets | 321 | |||
Level 2 [Member] | ||||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||||
Total assets | 1,268,671 | 1,187,395 | ||
Level 2 [Member] | Commercial Paper [Member] | ||||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||||
Total assets | 88,450 | 211,369 | ||
Level 2 [Member] | Government and Government Agency Bonds [Member] | ||||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||||
Total assets | 989,257 | 807,540 | ||
Level 2 [Member] | Corporate Bonds [Member] | ||||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||||
Total assets | 145,898 | 125,741 | ||
Level 2 [Member] | Certificates of Deposit [Member] | ||||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||||
Total assets | 45,066 | 42,745 | ||
Level 3 [Member] | ||||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||||
Total assets | 7,500 | 31,000 | $ 32,575 | $ 32,412 |
Contingent consideration | 38,100 | 36,900 | ||
Total liabilities | 38,100 | 36,900 | $ 36,900 | $ 43,600 |
Level 3 [Member] | Strategic Equity Investments [Member] | ||||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||||
Total assets | $ 7,500 | $ 31,000 |
Fair Value Measurements - Summa
Fair Value Measurements - Summary of Fair Value of Level 3 Financial Assets (Detail) - USD ($) $ in Thousands | 9 Months Ended | |
Sep. 30, 2023 | Sep. 30, 2022 | |
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Fair value, at the beginning of the period | $ 1,686,269 | |
Fair value, at the end of the period | 1,415,853 | |
Level 3 [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Fair value, at the beginning of the period | 31,000 | $ 32,412 |
Additions | 4,000 | 163 |
Changes in estimated fair value | (27,500) | |
Fair value, at the end of the period | $ 7,500 | $ 32,575 |
Fair Value Measurements - Sum_2
Fair Value Measurements - Summary of Fair Value of Level 3 Financial Liabilities (Detail) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | |
Sep. 30, 2023 | Sep. 30, 2023 | Sep. 30, 2022 | |
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | |||
Fair value at the beginning of the period | $ 36,900 | ||
Change in estimated fair value | $ 1,200 | 2,000 | |
Fair value at the end of the period | 38,100 | 38,100 | |
Level 3 [Member] | |||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | |||
Fair value at the beginning of the period | 36,900 | $ 43,600 | |
Change in estimated fair value | 2,000 | (6,700) | |
Liabilities terminated | (800) | ||
Fair value at the end of the period | $ 38,100 | $ 38,100 | $ 36,900 |
Fair Value Measurements - Addit
Fair Value Measurements - Additional Information (Detail) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | |||
Sep. 30, 2023 | Sep. 30, 2022 | Sep. 30, 2023 | Sep. 30, 2022 | Dec. 31, 2022 | |
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | |||||
Impairment of equity investment | $ (27,500) | $ 0 | $ (27,821) | $ 0 | |
Fair value of contingent consideration liability | 1,200 | 2,000 | |||
Strategic investments | 7,500 | 7,500 | $ 31,321 | ||
Biotechnology Company | |||||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | |||||
Strategic investments | $ 4,000 | 4,000 | |||
Level 3 [Member] | |||||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | |||||
Fair value of contingent consideration liability | 2,000 | $ (6,700) | |||
Level 3 [Member] | Minimum [Member] | |||||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | |||||
Equity Method Investment Other Than Temporary Impairment Level 3 Input Min | 0 | ||||
Level 3 [Member] | Maximum [Member] | |||||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | |||||
Equity Method Investment Other Than Temporary Impairment Level 3 Input Max | $ 2,500 |
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, 2023 | Dec. 31, 2022 |
Cash and Cash Equivalents [Line Items] | ||
Cash equivalents | $ 216,743 | $ 632,351 |
Money Market Funds [Member] | ||
Cash and Cash Equivalents [Line Items] | ||
Cash equivalents | 139,682 | 467,553 |
Commercial Paper [Member] | ||
Cash and Cash Equivalents [Line Items] | ||
Cash equivalents | 0 | 33,190 |
Government and Government Agency Bonds [Member] | ||
Cash and Cash Equivalents [Line Items] | ||
Cash equivalents | 66,535 | 128,451 |
Corporate Bond [Member] | ||
Cash and Cash Equivalents [Line Items] | ||
Cash equivalents | $ 10,526 | $ 3,157 |
Cash, Cash Equivalents and Ma_3
Cash, Cash Equivalents and Marketable Securities - Additional Information (Detail) | 9 Months Ended | 12 Months Ended |
Sep. 30, 2023 | Dec. 31, 2022 | |
Cash and Cash Equivalents [Line Items] | ||
Weighted average maturity period of available-for-sale securities | 4 months | 4 months |
Summary of Company Cash, Cash E
Summary of Company Cash, Cash Equivalents and Short-term Investments (Detail) - USD ($) $ in Thousands | Sep. 30, 2023 | Dec. 31, 2022 | Sep. 30, 2022 |
Cash and Cash Equivalents [Line Items] | |||
Available for sale debt securities, Gross unrealized gains | $ 187 | ||
Available for sale debt securities, Gross unrealized losses | (1,824) | ||
Cash and cash equivalents, Amortized cost | $ 1,734,153 | 1,991,011 | |
Cash and cash equivalents, Gross unrealized gains | 8 | 9 | |
Cash and cash equivalents, Gross unrealized losses | (2) | ||
Cash and cash equivalents, Fair value | 541,932 | 966,777 | $ 1,038,624 |
Cash, cash equivalents and investments, Amortized cost | 541,926 | 966,768 | |
Cash, cash equivalents and investments, Gross unrealized gains | 57 | 187 | |
Cash, cash equivalents and investments, Gross unrealized losses | (668) | (1,824) | |
Cash, cash equivalents and investments, Fair value | 1,733,542 | 1,989,374 | |
Available for sale debt securities current, Amortized cost | 1,192,227 | 1,024,243 | |
Available for sale debt securities current, Gross unrealized gains | 49 | 178 | |
Available for sale debt securities current, Gross unrealized losses | 666 | (1,824) | |
Available for sale debt securities current, Fair value | 1,191,610 | 1,022,597 | |
Cash and Money Market Funds [Member] | |||
Cash and Cash Equivalents [Line Items] | |||
Cash and cash equivalents, Amortized cost | 464,871 | 801,979 | |
Cash and cash equivalents, Fair value | 464,871 | 801,979 | |
Commercial Paper [Member] | |||
Cash and Cash Equivalents [Line Items] | |||
Available for sale debt securities, Fair value | 88,450 | 211,369 | |
Cash and cash equivalents, Amortized cost | 88,450 | 211,369 | |
Government and Government Agency Bonds [Member] | |||
Cash and Cash Equivalents [Line Items] | |||
Available for sale debt securities, Gross unrealized gains | 41 | 178 | |
Available for sale debt securities, Gross unrealized losses | (581) | (1,542) | |
Available for sale debt securities, Fair value | 989,257 | 807,540 | |
Cash and cash equivalents, Amortized cost | 989,797 | 808,904 | |
Cash, cash equivalents and investments, Fair value | 1,989,374 | ||
Corporate Bond [Member] | |||
Cash and Cash Equivalents [Line Items] | |||
Available for sale debt securities, Gross unrealized gains | 16 | 9 | |
Available for sale debt securities, Gross unrealized losses | (87) | (282) | |
Available for sale debt securities, Fair value | 145,898 | 125,741 | |
Cash and cash equivalents, Amortized cost | 145,969 | 126,014 | |
Certificates of Deposit [Member] | |||
Cash and Cash Equivalents [Line Items] | |||
Available for sale debt securities, Fair value | 45,066 | 42,745 | |
Cash and cash equivalents, Amortized cost | $ 45,066 | $ 42,745 |
Product Revenues, Net, Accoun_3
Product Revenues, Net, Accounts Receivable and Reserves for Product Revenues (Additional Information) (Details) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | 12 Months Ended | ||
Sep. 30, 2023 | Sep. 30, 2022 | Sep. 30, 2023 | Sep. 30, 2022 | Dec. 31, 2022 | |
Products, net | $ 309,322 | $ 207,774 | $ 779,805 | $ 607,836 | |
Revenues | 331,817 | $ 230,269 | 846,555 | $ 674,586 | |
Product sales receivable, net of discounts and allowances | $ 318,900 | $ 318,900 | $ 214,600 | ||
Payment Terms | As of September 30, 2023, 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 100 days. Outside of the U.S., the majority of the Company’s customers have payment terms ranging between 60 and 150 days | ||||
Sales Revenue Net [Member] | Customer Concentration Risk [Member] | Customer One [Member] | |||||
Concentration of credit risk percentage | 40% | 48% | 45% | 49% | |
Sales Revenue Net [Member] | Customer Concentration Risk [Member] | Customer Two [Member] | |||||
Concentration of credit risk percentage | 23% | 35% | 29% | 34% | |
Sales Revenue Net [Member] | Customer Concentration Risk [Member] | Customer Three [Member] | |||||
Concentration of credit risk percentage | 9% | 7% | 8% | 7% | |
Sales Revenue Net [Member] | Customer Concentration Risk [Member] | No Individual [Member] | |||||
Concentration of credit risk percentage | 10% | 10% | 10% | 10% | |
Sales Revenue Net [Member] | Customer Concentration Risk [Member] | Total [Member] | |||||
Concentration of credit risk percentage | 10% | ||||
Accounts Receivable [Member] | Customer Concentration Risk [Member] | Customer One [Member] | |||||
Concentration of credit risk percentage | 29% | 36% | |||
Accounts Receivable [Member] | Customer Concentration Risk [Member] | Customer Two [Member] | |||||
Concentration of credit risk percentage | 25% | 35% | |||
Accounts Receivable [Member] | Customer Concentration Risk [Member] | Customer Three [Member] | |||||
Concentration of credit risk percentage | 12% | 12% |
Product Revenues, Net, Accoun_4
Product Revenues, Net, Accounts Receivable and Reserves for Product Revenues - Summary of Change in Reserves for Discounts and Allowances (Detail) - USD ($) $ in Thousands | 9 Months Ended | |
Sep. 30, 2023 | Sep. 30, 2022 | |
Accounts Notes And Loans Receivable [Line Items] | ||
Beginning balance | $ 94,698 | $ 70,466 |
Provision | 175,967 | 128,029 |
Adjustments relating to prior years | (4,044) | (4,081) |
Payments/credits | (133,337) | (107,165) |
Ending balance | 133,284 | 87,249 |
Chargebacks [Member] | ||
Accounts Notes And Loans Receivable [Line Items] | ||
Beginning balance | 417 | 799 |
Provision | 18,191 | 8,756 |
Adjustments relating to prior years | 110 | 0 |
Payments/credits | (9,386) | (9,193) |
Ending balance | 9,332 | 362 |
Rebates [Member] | ||
Accounts Notes And Loans Receivable [Line Items] | ||
Beginning balance | 67,493 | 60,506 |
Provision | 94,025 | 80,956 |
Adjustments relating to prior years | (4,154) | (4,111) |
Payments/credits | (73,317) | (70,645) |
Ending balance | 84,047 | 66,706 |
Prompt Pay [Member] | ||
Accounts Notes And Loans Receivable [Line Items] | ||
Beginning balance | 3,343 | 2,798 |
Provision | 11,251 | 9,508 |
Adjustments relating to prior years | 0 | 0 |
Payments/credits | (10,586) | (8,930) |
Ending balance | 4,008 | 3,376 |
Other Accruals [Member] | ||
Accounts Notes And Loans Receivable [Line Items] | ||
Beginning balance | 23,445 | 6,363 |
Provision | 52,500 | 28,809 |
Adjustments relating to prior years | 0 | 30 |
Payments/credits | (40,048) | (18,397) |
Ending balance | $ 35,897 | $ 16,805 |
Product Revenues, Net, Accoun_5
Product Revenues, Net, Accounts Receivable and Reserves for Product Revenues - Summary of Total Reserves Included in Consolidated Balance Sheets (Detail) - USD ($) $ in Thousands | Sep. 30, 2023 | Dec. 31, 2022 | Sep. 30, 2022 | Dec. 31, 2021 |
Receivables, Net, Current [Abstract] | ||||
Reduction to accounts receivable | $ 47,606 | $ 25,914 | ||
Component of accrued expenses | 85,678 | 68,784 | ||
Total reserves | $ 133,284 | $ 94,698 | $ 87,249 | $ 70,466 |
Net product revenues - summary
Net product revenues - summary of includes revenues associated with EXONDYS 51 AMONDYS 45 and VYONDYS 53 (collectively the PMO Products and ELEVIDYS (Details) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2023 | Sep. 30, 2022 | Sep. 30, 2023 | Sep. 30, 2022 | |
Disaggregation of Revenue [Line Items] | ||||
Revenue from Contract with Customer, Excluding Assessed Tax | $ 309,322 | $ 207,774 | $ 779,805 | $ 607,836 |
PMO Products [Member] | ||||
Disaggregation of Revenue [Line Items] | ||||
Revenue from Contract with Customer, Excluding Assessed Tax | 240,214 | 207,774 | 710,697 | 607,836 |
ELEVIDYS [Member] | ||||
Disaggregation of Revenue [Line Items] | ||||
Revenue from Contract with Customer, Excluding Assessed Tax | 69,108 | 0 | 69,108 | 0 |
United States [Member] | PMO Products [Member] | ||||
Disaggregation of Revenue [Line Items] | ||||
Revenue from Contract with Customer, Excluding Assessed Tax | 198,069 | 188,346 | 602,415 | 548,355 |
United States [Member] | ELEVIDYS [Member] | ||||
Disaggregation of Revenue [Line Items] | ||||
Revenue from Contract with Customer, Excluding Assessed Tax | 69,108 | 0 | 69,108 | 0 |
Rest of World [Member] | PMO Products [Member] | ||||
Disaggregation of Revenue [Line Items] | ||||
Revenue from Contract with Customer, Excluding Assessed Tax | $ 42,145 | $ 19,428 | $ 108,282 | $ 59,481 |
Inventory - Summary of Componen
Inventory - Summary of Components of Inventory (Detail) - USD ($) $ in Thousands | Sep. 30, 2023 | Dec. 31, 2022 |
Inventory Disclosure [Abstract] | ||
Raw materials | $ 114,622 | $ 59,181 |
Work in progress | 258,258 | 269,185 |
Finished goods | 47,243 | 38,147 |
Total inventory | $ 420,123 | $ 366,513 |
Inventory - Summarizes The Bala
Inventory - Summarizes The Balance Sheet Classification (Details) - USD ($) $ in Thousands | Sep. 30, 2023 | Dec. 31, 2022 |
Inventory Disclosure [Abstract] | ||
Inventory | $ 244,011 | $ 203,968 |
Non-current inventory | 176,112 | 162,545 |
Total inventory | $ 420,123 | $ 366,513 |
Other Assets - Summary of Other
Other Assets - Summary of Other Current Assets (Detail) - USD ($) $ in Thousands | Sep. 30, 2023 | Dec. 31, 2022 |
Prepaid Expense and Other Assets, Current [Abstract] | ||
Manufacturing-related deposits and prepaids | $ 77,125 | $ 66,455 |
Collaboration receivable | 35,408 | 41,758 |
Prepaid maintenance services | 12,282 | 9,815 |
Prepaid clinical and pre-clinical expenses | 9,247 | 11,237 |
Prepaid commercial expenses | 2,793 | 2,947 |
Prepaid research expenses | 2,372 | 1,927 |
Prepaid insurance | 1,545 | 3,717 |
Other | 13,669 | 12,035 |
Total other current assets | $ 154,441 | $ 149,891 |
Other Assets - Summary of Oth_2
Other Assets - Summary of Other Non-current Assets (Detail) - USD ($) $ in Thousands | Sep. 30, 2023 | Dec. 31, 2022 |
Prepaid Expense and Other Assets, Noncurrent [Abstract] | ||
Manufacturing-related deposits and prepaids | $ 78,842 | $ 97,409 |
Intangible assets, net | 19,501 | 7,578 |
Restricted cash | 19,149 | 19,024 |
Strategic investments | 7,500 | 31,321 |
Prepaid maintenance services | 5,948 | 3,403 |
Prepaid clinical expenses | 2,219 | 2,150 |
Other | 3,766 | 2,084 |
Total other non-current assets | $ 136,925 | $ 162,969 |
Accrued Expenses - Summary of A
Accrued Expenses - Summary of Accrued Expenses (Detail) - USD ($) $ in Thousands | Sep. 30, 2023 | Dec. 31, 2022 |
Payables and Accruals [Abstract] | ||
Product revenue related reserves | $ 85,678 | $ 68,784 |
Accrued contract manufacturing costs | 60,998 | 202,173 |
Accrued employee compensation costs | 51,143 | 65,946 |
Accrued clinical and pre-clinical costs | 40,930 | 28,884 |
Accrued income taxes | 24,677 | 12,521 |
Accrued professional fees | 24,050 | 12,061 |
Accrued royalties | 10,501 | 8,636 |
Accrued property and equipment purchases | 9,382 | 984 |
Accrued milestone and license costs | 3,750 | 7,702 |
Accrued research costs | 3,516 | 1,629 |
Other | 7,725 | 9,676 |
Total accrued expenses | $ 322,350 | $ 418,996 |
Indebtedness - Additional Infor
Indebtedness - Additional Information (Detail) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | |||||||
Mar. 02, 2023 | Sep. 14, 2022 | Nov. 14, 2017 | Sep. 30, 2023 | Mar. 31, 2023 | Sep. 30, 2022 | Sep. 30, 2023 | Sep. 30, 2022 | Dec. 31, 2022 | |
Debt Instrument [Line Items] | |||||||||
Debt instrument, face amount | $ 1,315,729 | $ 1,315,729 | |||||||
Partial settlement of capped call share options for 2024 Notes | $ 80,645 | $ 26,317 | 80,645 | $ 26,317 | |||||
Loss on debt extinguishment | 0 | (125,441) | (387,329) | (125,441) | |||||
Debt conversion costs for 2024 Notes | $ 6,900 | 6,887 | 0 | ||||||
Interest expense debt | 5,200 | 14,700 | 16,800 | 46,500 | |||||
Long-term debt | 1,236,755 | 1,236,755 | $ 1,544,292 | ||||||
Aggregate long-term debt | 1,236,755 | 1,236,755 | 1,544,292 | ||||||
Amortization of Debt Issuance Costs | 1,200 | $ 2,000 | 3,900 | $ 6,000 | |||||
Convertible Debt, The Term Loan, The Revolver and The Mortgage Loans [Member] | |||||||||
Debt Instrument [Line Items] | |||||||||
Long-term debt | $ 1,236,800 | $ 1,236,800 | 1,544,300 | ||||||
2027 Notes [Member] | |||||||||
Debt Instrument [Line Items] | |||||||||
Debt instrument, interest rate per annum | 1.25% | 1.25% | |||||||
2024 Convertible Notes [Member] | |||||||||
Debt Instrument [Line Items] | |||||||||
Debt instrument, face amount | $ 150,600 | $ 570,000 | $ 105,847 | $ 105,847 | 419,371 | ||||
Issuance of common stock for exchange of 2024 Notes, shares | 4,500,000 | ||||||||
Partial settlement of capped call share options for 2024 Notes | $ 80,600 | ||||||||
Debt instrument, maturity date | Nov. 15, 2024 | ||||||||
Reduction in carrying value of convertible notes | 311,500 | ||||||||
Loss on debt extinguishment | 387,300 | ||||||||
Debt conversion costs for 2024 Notes | 6,900 | ||||||||
Conversion of shares excluded from computation of diluted EPS | 1,400,000 | 5,700,000 | 1,400,000 | 5,700,000 | |||||
Fair value convertible note exchange | 693,400 | ||||||||
Eliminated accrued interest | 1,500 | ||||||||
Payment for full settlement of the principal value and accrued interest | 248,600 | ||||||||
2024 Convertible Notes [Member] | Common Stock [Member] | |||||||||
Debt Instrument [Line Items] | |||||||||
Debt Instrument, Periodic Payment, Principal | $ 313,500 | ||||||||
2027 Convertible Notes [Member] | |||||||||
Debt Instrument [Line Items] | |||||||||
Debt instrument, face amount | $ 1,150,000 | $ 1,150,000 | $ 1,150,000 | ||||||
Conversion of shares excluded from computation of diluted EPS | 8,100,000 | 8,100,000 | 8,100,000 | 8,100,000 | |||||
2017 Capped Call Transactions [Member] | |||||||||
Debt Instrument [Line Items] | |||||||||
Partial settlement of capped call share options for 2024 Notes | $ 26,300 |
Indebtedness - Summary of Debt
Indebtedness - Summary of Debt Facilities (Detail) - USD ($) $ in Thousands | Sep. 30, 2023 | Dec. 31, 2022 | Sep. 14, 2022 | Nov. 14, 2017 |
Debt Instrument [Line Items] | ||||
Principal amount | $ 1,315,729 | |||
Net carrying value | 1,236,755 | $ 1,544,292 | ||
Total carrying value of debt facilities | 1,236,755 | 1,544,292 | ||
Total fair value of debt facilities | 1,484,197 | 2,073,528 | ||
2024 Convertible Notes [Member] | ||||
Debt Instrument [Line Items] | ||||
Principal amount | 105,847 | 419,371 | $ 150,600 | $ 570,000 |
Unamortized discount - debt issuance costs | (465) | (3,059) | ||
Total fair value of debt facilities | 189,297 | 765,046 | ||
2027 Convertible Notes [Member] | ||||
Debt Instrument [Line Items] | ||||
Principal amount | 1,150,000 | 1,150,000 | ||
Unamortized discount - debt issuance costs | (18,627) | (22,020) | ||
Total fair value of debt facilities | $ 1,294,900 | $ 1,308,482 |
Indebtedness - Summarizes Total
Indebtedness - Summarizes Total Gross Payments Due under Company's Debt Arrangements (Detail) $ in Thousands | Sep. 30, 2023 USD ($) |
Debt Conversion [Line Items] | |
2023 (October-December) | $ 794 |
2024 | 121,810 |
2025 | 14,375 |
2026 | 14,375 |
2027 | 1,164,375 |
Total payments | 1,315,729 |
Principal [Member] | |
Debt Conversion [Line Items] | |
2023 (October-December) | 0 |
2024 | 105,847 |
2025 | 0 |
2026 | 0 |
2027 | 1,150,000 |
Total payments | 1,255,847 |
Interest [Member] | |
Debt Conversion [Line Items] | |
2023 (October-December) | 794 |
2024 | 15,963 |
2025 | 14,375 |
2026 | 14,375 |
2027 | 14,375 |
Total payments | $ 59,882 |
Stock Based Compensation - Summ
Stock Based Compensation - Summary of Stock Awards Granted (Detail) - $ / shares | 3 Months Ended | 9 Months Ended | ||||
Sep. 30, 2023 | Sep. 30, 2022 | Sep. 30, 2023 | Sep. 30, 2022 | |||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||||
Grants, Stock options | 50,875 | 127,100 | 1,135,799 | 1,626,220 | ||
Weighted Average Grant Date Fair Value, Stock options | $ 66.53 | $ 59.84 | $ 72.23 | $ 47.54 | ||
Grants, Restricted stock units | 27,539 | 65,657 | 1,137,202 | [1] | 925,167 | [2] |
Weighted Average Grant Date Fair Value, Restricted stock units | $ 118.05 | $ 105.28 | $ 153.02 | $ 80.66 | ||
Restricted Stock Units [Member] | ||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||||
Grants, Stock options | 502,225 | 38,500 | ||||
[1] Included in restricted stock units (“RSUs”) for the nine months ended September 30, 2023 are 502,225 shares with performance conditions (the “March 2023 PSUs”) which are related to regulatory approval of certain of the Company's product candidates and achievement of a certain financial performance target. Included in the RSUs for the nine months ended September 30, 2022 are 38,500 sh ares of PSUs (the “March 2022 PSUs”) with performance conditions related to regulatory approval of the Company's product candidates. |
Stock Based Compensation - Su_2
Stock Based Compensation - Summary of Stock Awards Granted (Parenthetical) (Details) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2023 | Sep. 30, 2022 | Sep. 30, 2023 | Sep. 30, 2022 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Grants, Stock options | 50,875 | 127,100 | 1,135,799 | 1,626,220 |
Stock-based compensation Grants | $ 10,300 | $ 17,700 | ||
Stock-based compensation expense | 48,061 | $ 50,418 | $ 136,688 | $ 182,508 |
Employee Stock Option | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Share based compensation granted under plan vest period | 4 years | |||
Stock-based compensation expense | 17,471 | $ 35,686 | $ 62,436 | $ 141,247 |
Restricted Stock Units [Member] | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Grants, Stock options | 502,225 | 38,500 | ||
Stock-based compensation Grants | $ 3,300 | |||
Share based compensation granted under plan vest period | 1 year 6 months | |||
Share-based payment arrangement, Cost not yet recognized, Amount | $ 62,200 | $ 62,200 |
Stock Based Compensation - Addi
Stock Based Compensation - Additional Information (Detail) - USD ($) $ / shares in Units, $ in Millions | 1 Months Ended | 3 Months Ended | 9 Months Ended | |||
Jun. 30, 2017 | Sep. 30, 2023 | Mar. 31, 2023 | Sep. 30, 2022 | Sep. 30, 2023 | Sep. 30, 2022 | |
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items] | ||||||
Stock-based compensation expenses | $ 48.1 | $ 50.4 | ||||
Grants, Stock options | 50,875 | 127,100 | 1,135,799 | 1,626,220 | ||
Share-Based Compensation Arrangement by Share-Based Payment Award, Terms of Award | 550,110 options relating to the Unvested Tranche met the conditions for vesting as the average closing price of the Company's common stock exceeded $128.65 during 20 consecutive trading days in March 2023 and the compound annual growth rate of the Company's common stock exceeded that of the Nasdaq Biotech Index by greater than 5% | |||||
Chief Executive Officer | ||||||
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items] | ||||||
Stock-based compensation expenses | $ 13.4 | $ 94.2 | ||||
Grants, Stock options | 3,300,000 | |||||
Minimum [Member] | ||||||
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items] | ||||||
Compound annual growth rate | 5% | |||||
Unvested Tranche Member | ||||||
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items] | ||||||
Stock-based compensation expenses | $ 136.7 | $ 182.5 | ||||
Vesting, Stock options | 550,110 | |||||
Average closing price | $ 128.65 | |||||
Share-Based Compensation Arrangement by Share-Based Payment Award, Terms of Award | 550,110 options relating to the Unvested Tranche met the conditions for vesting as the average closing price of the Company's common stock exceeded $128.65 during 20 consecutive trading days in March 2023 and the compound annual growth rate of the Company's common stock exceeded that of the Nasdaq Biotech Index by greater than 5%. | |||||
Unvested Tranche Member | Chief Executive Officer | ||||||
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items] | ||||||
Stock-based compensation expenses | $ 0.6 | $ 20.5 |
Stock Based Compensation - Su_3
Stock Based Compensation - 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, 2023 | Sep. 30, 2022 | Sep. 30, 2023 | Sep. 30, 2022 | |
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | ||||
Total stock-based compensation expense | $ 48,061 | $ 50,418 | $ 136,688 | $ 182,508 |
Research and Development [Member] | ||||
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | ||||
Total stock-based compensation expense | 22,325 | 14,795 | 60,315 | 42,330 |
Selling, General and Administrative [Member] | ||||
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | ||||
Total stock-based compensation expense | $ 25,736 | $ 35,623 | $ 76,373 | $ 140,178 |
Stock Based Compensation - Su_4
Stock Based Compensation - 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, 2023 | Sep. 30, 2022 | Sep. 30, 2023 | Sep. 30, 2022 | |
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | ||||
Total stock-based compensation expense | $ 48,061 | $ 50,418 | $ 136,688 | $ 182,508 |
Employee Stock Option | ||||
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | ||||
Total stock-based compensation expense | 17,471 | 35,686 | 62,436 | 141,247 |
Restricted Stock Units [Member] | ||||
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | ||||
Total stock-based compensation expense | 28,924 | 13,321 | 70,101 | 37,064 |
Employee Stock Purchase Plan [Member] | ||||
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | ||||
Total stock-based compensation expense | $ 1,666 | $ 1,411 | $ 4,151 | $ 4,197 |
Other Loss, Net - Summary of Ot
Other Loss, Net - Summary of Other Loss (Detail) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2023 | Sep. 30, 2022 | Sep. 30, 2023 | Sep. 30, 2022 | |
Nonoperating Income (Expense) [Abstract] | ||||
Accretion of investment discount, net | $ 12,984 | $ 3,435 | $ 35,809 | $ 4,808 |
Interest income | 9,838 | 4,780 | 27,532 | 7,362 |
Interest expense | (5,229) | (14,736) | (16,776) | (46,560) |
(Gain) loss on contingent consideration, net | (2,000) | 6,700 | (1,200) | 6,700 |
Impairment of equity investment | (27,500) | 0 | (27,821) | 0 |
Other, net | (425) | 199 | (235) | (6,158) |
Other income (expense), net | (12,332) | 378 | 17,309 | (33,848) |
Gain from sale of Priority Review Voucher | 0 | 0 | 102,000 | 0 |
Loss on debt extinguishment | 0 | (125,441) | (387,329) | (125,441) |
Total other (loss), net | $ (12,332) | $ (125,063) | $ (268,020) | $ (159,289) |
Leases (Additional Information)
Leases (Additional Information) (Details) $ in Thousands | 3 Months Ended | 9 Months Ended | |||
May 31, 2022 USD ($) | Apr. 22, 2022 USD ($) ft² | Sep. 30, 2023 USD ($) | Sep. 30, 2023 USD ($) | Dec. 31, 2022 USD ($) | |
Lessee, Lease, Description [Line Items] | |||||
Right of use assets | $ 133,454 | $ 133,454 | $ 64,954 | ||
Bedford Massachusetts [Member] | |||||
Lessee, Lease, Description [Line Items] | |||||
Area of lease | ft² | 288,000 | ||||
Leases, term of contract extend | 10 years | ||||
Remaining lease term | 15 years 3 months 18 days | 15 years 3 months 18 days | |||
Lease discount rate | 9.50% | 9.50% | |||
Operating lease cost | $ 0 | $ 0 | |||
Variable Lease Cost | 100 | 100 | |||
Letter of Credit Reduce | $ 5,600 | ||||
Collateralized Letter of Credit Cash Deposit | $ 8,400 | ||||
Undiscounted rent payment | $ 307,400 | ||||
Tenant improvement allowance | $ 72,000 | 6,100 | |||
Reduction to right of use assets and lease liabilities | 72,000 | ||||
Right of use assets | 71,700 | 71,700 | |||
Operating lease liability | $ 84,100 | $ 84,100 |
Net Loss Per Share - Basic and
Net Loss Per Share - Basic and Diluted Net Loss Per Share (Detail) - USD ($) $ / shares in Units, shares in Thousands, $ in Thousands | 3 Months Ended | 9 Months Ended | |||||||
Sep. 30, 2023 | Jun. 30, 2023 | Mar. 31, 2023 | Sep. 30, 2022 | Jun. 30, 2022 | Mar. 31, 2022 | Sep. 30, 2023 | Sep. 30, 2022 | ||
Earnings Per Share [Abstract] | |||||||||
Net Income (Loss) | $ (40,937) | $ (23,940) | $ (516,755) | $ (257,738) | $ (231,481) | $ (105,025) | $ (581,632) | $ (594,244) | |
Weighted-average common shares outstanding - basic | 88,889 | 87,628 | 88,609 | 87,465 | |||||
Effect of dilutive securities* | [1] | 0 | 0 | 0 | 0 | ||||
Weighted-average common shares outstanding - diluted | 88,889 | 87,628 | 88,609 | 87,465 | |||||
Net loss per share - basic | $ (0.46) | $ (2.94) | $ (6.56) | $ (6.79) | |||||
Net loss per share - diluted | $ (0.46) | $ (2.94) | $ (6.56) | $ (6.79) | |||||
[1] For the three and nine months ended September 30, 2023 and 2022, stock options, RSUs and employee stock purchase plan to purchase of approximately 12.1 million and 11.2 million shares of 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 its 2027 Notes and 2024 Notes on diluted net earnings per share (“EPS”) 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 dilutive. During the three and nine months ended September 30, 2023 and 2022, the inclusion of the potential share settlement of the 2027 Notes and the 2024 Notes was anti-dilutive. Accordingly, the potential conversion o f approximately 1.4 million and 5.7 million shares related to the 2024 Notes has been excluded from the computation of diluted net loss per share for the three and nine months ended September 30, 2023 and 2022, respectively, and the potential conversion of approximatel y 8.1 million shar es related to the 2027 Notes has been excluded from the computation of diluted net loss per share for the three and nine months ended September 30, 2023 and 2022, respectively. |
Net Loss Per Share - Basic an_2
Net Loss Per Share - Basic and Diluted Net Loss Per Share (Parenthetical) (Detail) - shares shares in Millions | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2023 | Sep. 30, 2022 | Sep. 30, 2023 | Sep. 30, 2022 | |
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||||
Anti-dilutive securities excluded from computation of net loss per share | 12.1 | 11.2 | 12.1 | 11.2 |
2024 Convertible Notes [Member] | ||||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||||
Conversion of shares excluded from computation of diluted EPS | 1.4 | 5.7 | 1.4 | 5.7 |
2027 Convertible Notes [Member] | ||||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||||
Conversion of shares excluded from computation of diluted EPS | 8.1 | 8.1 | 8.1 | 8.1 |
Commitments and Contingencies -
Commitments and Contingencies - Summary of Aggregate Non-Cancelable Contractual Obligations Arising from Manufacturing Obligations (Detail) $ in Thousands | Sep. 30, 2023 USD ($) | |
Commitments And Contingencies [Line Items] | ||
2023 (October-December) | $ 380,762 | |
2024 | 762,237 | |
2025 | 151,525 | |
2026 | 80,110 | |
2027 | 77,497 | |
Thereafter | 72,244 | |
Total manufacturing commitments | $ 1,524,375 | [1] |
[1] Total manufacturing commitments includes the Catalent manufacturing and supply agreement, for which the Company has ROU assets and lease liabilities recorded on the unaudited condensed consolidated balance sheets as of September 30, 2023. For more information, please read Note 21, Commitments and Contingencies to the financial statements included in the Company’ s Annual Report on Form 10-K for the year ended December 31, 2022. |
Commitments and Contingencies_2
Commitments and Contingencies (Additional Information) (Details) - USD ($) $ in Millions | 3 Months Ended | 9 Months Ended | |
Sep. 30, 2023 | Mar. 31, 2023 | Sep. 30, 2023 | |
Commitments And Contingencies [Line Items] | |||
Purchase commitment | $ 54.7 | ||
Amendment Implemented Fee | 60 | ||
Nonrefundable Advance Payment | $ 0.8 | $ 1.7 | |
First Installments [Member] | |||
Commitments And Contingencies [Line Items] | |||
Amendment Implemented Fee | $ 20 | ||
Installments Date of implemented fee | Mar. 01, 2024 |