Document and Entity Information
Document and Entity Information - shares | 6 Months Ended | |
Jun. 30, 2022 | Jul. 29, 2022 | |
Cover [Abstract] | ||
Document Type | 10-Q | |
Amendment Flag | false | |
Document Period End Date | Jun. 30, 2022 | |
Document Fiscal Year Focus | 2022 | |
Document Fiscal Period Focus | Q2 | |
Trading Symbol | SRPT | |
Entity Registrant Name | Sarepta Therapeutics, Inc. | |
Entity Central Index Key | 0000873303 | |
Current Fiscal Year End Date | --12-31 | |
Entity Filer Category | Large Accelerated Filer | |
Entity Small Business | false | |
Entity Emerging Growth Company | false | |
Entity Common Stock, Shares Outstanding | 87,567,857 | |
Entity Current Reporting Status | Yes | |
Entity Shell Company | false | |
Entity File Number | 001-14895 | |
Entity Tax Identification Number | 93-0797222 | |
Entity Address, Address Line One | 215 First Street | |
Entity Address, Address Line Two | Suite 415 | |
Entity Address, State or Province | MA | |
Entity Address, City or Town | Cambridge | |
Entity Address, Postal Zip Code | 02142 | |
City Area Code | 617 | |
Local Phone Number | 274-4000 | |
Entity Interactive Data Current | Yes | |
Title of 12(b) Security | Common Stock, $0.0001 par value per share | |
Security Exchange Name | NASDAQ | |
Entity Incorporation, State or Country Code | DE | |
Document Quarterly Report | true | |
Document Transition Report | false |
Condensed Consolidated Balance
Condensed Consolidated Balance Sheets - USD ($) $ in Thousands | Jun. 30, 2022 | Dec. 31, 2021 |
Current assets: | ||
Cash and cash equivalents | $ 868,565 | $ 2,115,869 |
Short-term investments | 1,059,454 | 0 |
Accounts receivable | 203,854 | 152,990 |
Inventory | 208,095 | 186,212 |
Other current assets | 129,332 | 149,028 |
Total current assets | 2,469,300 | 2,604,099 |
Property and equipment, net | 183,292 | 191,156 |
Intangible assets, net | 13,062 | 14,239 |
Right of use assets | 46,999 | 45,531 |
Other non-current assets | 284,200 | 292,949 |
Total assets | 2,996,853 | 3,147,974 |
Current liabilities: | ||
Accounts payable | 56,207 | 76,741 |
Accrued expenses | 383,699 | 271,697 |
Deferred revenue, current portion | 89,244 | 89,244 |
Other current liabilities | 16,416 | 15,051 |
Total current liabilities | 545,566 | 452,733 |
Long-term debt | 1,100,873 | 1,096,876 |
Lease liabilities, net of current portion | 39,368 | 41,512 |
Deferred revenue, net of current portion | 529,989 | 574,244 |
Contingent consideration | 43,600 | 43,600 |
Other non-current liabilities | 11,000 | 11,000 |
Total liabilities | 2,270,396 | 2,219,965 |
Commitments and contingencies (Note 13) | ||
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; 87,535,299 and 87,126,974 issued and outstanding at June 30, 2022, and December 31, 2021, respectively | 9 | 9 |
Additional paid-in capital | 4,272,187 | 4,134,768 |
Accumulated other comprehensive loss, net of tax | (2,485) | (20) |
Accumulated deficit | (3,543,254) | (3,206,748) |
Total stockholders’ equity | 726,457 | 928,009 |
Total liabilities and stockholders’ equity | $ 2,996,853 | $ 3,147,974 |
Condensed Consolidated Balanc_2
Condensed Consolidated Balance Sheets (Parenthetical) - $ / shares | Jun. 30, 2022 | Dec. 31, 2021 |
Statement of Financial Position [Abstract] | ||
Preferred stock, par value | $ 0.0001 | $ 0.0001 |
Preferred stock, shares authorized | 3,333,333 | 3,333,333 |
Preferred stock, shares issued | 0 | 0 |
Preferred stock, shares outstanding | 0 | 0 |
Common stock, par value | $ 0.0001 | $ 0.0001 |
Common stock, shares authorized | 198,000,000 | 198,000,000 |
Common stock, issued | 87,535,299 | 87,126,974 |
Common stock, outstanding | 87,535,299 | 87,126,974 |
Condensed Consolidated Statemen
Condensed Consolidated Statements of Operations and Comprehensive Loss - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2022 | Jun. 30, 2021 | Jun. 30, 2022 | Jun. 30, 2021 | |
Revenues: | ||||
Products, net | $ 211,237 | $ 141,839 | $ 400,062 | $ 266,765 |
Type of Revenue [Extensible List] | us-gaap:ProductMember | us-gaap:ProductMember | us-gaap:ProductMember | us-gaap:ProductMember |
Collaboration | $ 22,250 | $ 22,250 | $ 44,255 | $ 44,255 |
Total revenues | 233,487 | 164,089 | 444,317 | 311,020 |
Cost and expenses: | ||||
Cost of sales (excluding amortization of in-licensed rights) | 37,795 | 19,515 | 69,238 | 41,861 |
Research and development | 252,329 | 239,622 | 446,579 | 434,771 |
Selling, general and administrative | 154,316 | 72,347 | 226,156 | 143,478 |
Settlement and license charges | 0 | 0 | 0 | 10,000 |
Amortization of in-licensed rights | 179 | 179 | 357 | 349 |
Total cost and expenses | 444,619 | 331,663 | 742,330 | 630,459 |
Operating loss | (211,132) | (167,574) | (298,013) | (319,439) |
Other (loss) income, net: | ||||
Other expense, net | (16,961) | (16,185) | (34,226) | (31,713) |
Gain from sale of Priority Review Voucher | 0 | 102,000 | 0 | 102,000 |
Total other (loss) income, net | (16,961) | 85,815 | (34,226) | 70,287 |
Loss before income tax expense (benefit) | (228,093) | (81,759) | (332,239) | (249,152) |
Income tax expense (benefit) | 3,388 | (354) | 4,267 | (497) |
Net loss | (231,481) | (81,405) | (336,506) | (248,655) |
Other comprehensive loss: | ||||
Unrealized (losses) gains on investments, net of tax | (2,179) | 5 | (2,465) | (1) |
Total other comprehensive loss | (2,179) | 5 | (2,465) | (1) |
Comprehensive loss | $ (233,660) | $ (81,400) | $ (338,971) | $ (248,656) |
Net loss per share - basic and diluted | $ (2.65) | $ (1.02) | $ (3.85) | $ (3.12) |
Net loss per share - basic and diluted | $ (2.65) | $ (1.02) | $ (3.85) | $ (3.12) |
Weighted average number of shares of common stock used in computing basic and diluted net loss per share | 87,511 | 79,746 | 87,383 | 79,601 |
Weighted average number of shares of common stock used in computing basic and diluted net loss per share | 87,511 | 79,746 | 87,383 | 79,601 |
Condensed Consolidated Statem_2
Condensed Consolidated Statements of Stockholders' Equity - USD ($) shares in Thousands, $ in Thousands | Total | Cumulative Effect of Accounting Change [Member] | Common Stock [Member] | Additional Paid-In Capital [Member] | Additional Paid-In Capital [Member] Cumulative Effect of Accounting Change [Member] | Accumulated Other Comprehensive (Loss) Income [Member] | Accumulated Deficit [Member] | Accumulated Deficit [Member] Cumulative Effect of Accounting Change [Member] |
Balance at Dec. 31, 2020 | $ 761,759 | $ (96,792) | $ 8 | $ 3,609,877 | $ (156,953) | $ 3 | $ (2,848,129) | $ 60,161 |
Balance (in shares) at Dec. 31, 2020 | 79,374 | |||||||
Exercise of options for common stock | 4,683 | 4,683 | ||||||
Exercise of options for common stock, shares | 108 | |||||||
Vest of restricted stock units/awards | 204 | |||||||
Issuance of common stock under employee stock purchase plan | 4,543 | 4,543 | ||||||
Issuance of common stock under employee stock purchase plan, shares | 62 | |||||||
Stock-based compensation | 28,508 | 28,508 | ||||||
Unrealized losses from available-for-sale securities, net of tax | (6) | (6) | ||||||
Net loss | (167,250) | (167,250) | ||||||
Balance at Mar. 31, 2021 | 535,445 | $ 8 | 3,490,658 | (3) | (2,955,218) | |||
Balance (in shares) at Mar. 31, 2021 | 79,748 | |||||||
Balance at Dec. 31, 2020 | 761,759 | $ (96,792) | $ 8 | 3,609,877 | $ (156,953) | 3 | (2,848,129) | $ 60,161 |
Balance (in shares) at Dec. 31, 2020 | 79,374 | |||||||
Net loss | (248,655) | |||||||
Balance at Jun. 30, 2021 | 485,108 | $ 8 | 3,521,721 | 2 | (3,036,623) | |||
Balance (in shares) at Jun. 30, 2021 | 79,830 | |||||||
Balance at Mar. 31, 2021 | 535,445 | $ 8 | 3,490,658 | (3) | (2,955,218) | |||
Balance (in shares) at Mar. 31, 2021 | 79,748 | |||||||
Exercise of options for common stock | 3,526 | 3,526 | ||||||
Exercise of options for common stock, shares | 72 | |||||||
Vest of restricted stock units/awards | 28 | |||||||
Shares withheld for taxes | (1,432) | 1,432 | ||||||
Shares withheld for taxes, shares | 18 | |||||||
Stock-based compensation | 28,969 | 28,969 | ||||||
Unrealized losses from available-for-sale securities, net of tax | 5 | 5 | ||||||
Net loss | (81,405) | (81,405) | ||||||
Balance at Jun. 30, 2021 | 485,108 | $ 8 | 3,521,721 | 2 | (3,036,623) | |||
Balance (in shares) at Jun. 30, 2021 | 79,830 | |||||||
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/awards | 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 losses from available-for-sale securities, net of tax | (286) | (286) | ||||||
Net 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 | |||||||
Net loss | (336,506) | |||||||
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 | |||||||
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/awards | 28 | |||||||
Stock-based compensation | 102,892 | 102,892 | ||||||
Unrealized losses from available-for-sale securities, net of tax | (2,179) | (2,179) | ||||||
Net 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 |
Condensed Consolidated Statem_3
Condensed Consolidated Statements of Cash Flows - USD ($) $ in Thousands | 6 Months Ended | |
Jun. 30, 2022 | Jun. 30, 2021 | |
Cash flows from operating activities: | ||
Net loss | $ (336,506) | $ (248,655) |
Adjustments to reconcile net loss to cash flows from operating activities: | ||
Depreciation and amortization | 20,608 | 17,377 |
Reduction in the carrying amounts of the right of use assets | 5,514 | 6,023 |
Non-cash interest expense | 3,988 | 3,691 |
Stock-based compensation | 132,090 | 57,477 |
Loss on disposal of assets | 5,370 | 342 |
Gain from sale of Priority Review Voucher | 0 | (102,000) |
Other | (991) | 3,343 |
Changes in operating assets and liabilities, net: | ||
Net increase in accounts receivable | (50,864) | (26,180) |
Net increase in inventory | (15,364) | (36,795) |
Net decrease in other assets | 30,921 | 73,670 |
Net decrease in deferred revenue | (44,255) | (44,255) |
Net decrease in accounts payable, accrued expenses, lease liabilities and other liabilities | 81,499 | 6,445 |
Net cash used in operating activities | (167,990) | (289,517) |
Cash flows from investing activities: | ||
Purchase of property and equipment | (14,629) | (27,043) |
Proceeds from sale of Priority Review Voucher, net of commission | 0 | 102,000 |
Purchase of available-for-sale securities | (1,137,602) | (29,989) |
Maturity and sale of available-for-sale securities | 77,151 | 436,000 |
Other | (718) | (3,243) |
Net cash (used in) provided by investing activities | (1,075,798) | 477,725 |
Cash flows from financing activities: | ||
Proceeds from exercise of stock options and purchase of stock under the Employee Stock Purchase Program | 5,329 | 12,752 |
Taxes paid related to net share settlement of equity awards | 0 | (6,333) |
Net cash provided by financing activities | 5,329 | 6,419 |
Decrease in cash, cash equivalents and restricted cash | (1,238,459) | 194,627 |
Cash, cash equivalents and restricted cash: | ||
Beginning of period | 2,125,523 | 1,511,713 |
End of period | 887,064 | 1,706,340 |
Reconciliation of cash, cash equivalents and restricted cash: | ||
Cash and cash equivalents | 868,565 | 1,697,275 |
Restricted cash in other assets | 18,499 | 9,065 |
Total cash, cash equivalents and restricted cash | 887,064 | 1,706,340 |
Supplemental disclosure of cash flow information: | ||
Cash paid during the period for interest | 27,780 | 27,780 |
Supplemental schedule of non-cash investing activities and financing activities: | ||
Intangible assets and property and equipment included in accounts payable and accrued expenses | 5,751 | 7,760 |
Lease liabilities arising from obtaining right of use assets | 11,407 | 10,582 |
Lease liabilities terminated | 3,807 | 19,967 |
Shares withheld for tax included in accrued expenses | $ 0 | $ 1,432 |
ORGANIZATION AND NATURE OF BUSI
ORGANIZATION AND NATURE OF BUSINESS | 6 Months Ended |
Jun. 30, 2022 | |
Accounting Policies [Abstract] | |
ORGANIZATION AND NATURE OF BUSINESS | 1. ORGANIZATION AND NATURE OF BUSINESS Sarepta Therapeutics, Inc. (together with its wholly-owned subsidiaries, “Sarepta” or the “Company”) is a commercial-stage biopharmaceutical company focused on helping patients through the discovery and development of unique RNA-targeted therapeutics, gene therapy and other genetic therapeutic modalities for the treatment of rare diseases. Applying its proprietary, highly-differentiated and innovative technologies, and through collaborations with its strategic partners, the Company is developing potential therapeutic candidates for a broad range of diseases and disorders, including Duchenne muscular dystrophy (“Duchenne”), Limb-girdle muscular dystrophies (“LGMDs”) and other neuromuscular and central nervous system (“CNS”) disorders. The Company's products in the U.S., EXONDYS 51 (eteplirsen) Injection (“EXONDYS 51”), VYONDYS 53 (golodirsen) Injection (“VYONDYS 53”) and AMONDYS 45 (casimersen) Injection (“AMONDYS 45”), were granted accelerated approval by the U.S. Food and Drug Administration (the “FDA”) on September 19, 2016, December 12, 2019 and February 25, 2021, respectively. Indicated for the treatment of Duchenne in patients who have a confirmed mutation of the dystrophin gene that is amenable to exon 51, exon 53 and exon 45 skipping, respectively, EXONDYS 51, VYONDYS 53 and AMONDYS 45 use the Company’s phosphorodiamidate morpholino oligomer (“PMO”) chemistry and exon-skipping technology to skip exon 51, exon 53 and exon 45 of the dystrophin gene. Exon skipping is intended to promote the production of an internally truncated but functional dystrophin protein. As of June 30, 2022, the Company had approximately $ 1,946.6 million of cash, cash equivalents, restricted cash and investments, consisting of $ 868.6 million of cash and cash equivalents, $ 1,059.5 million of short-term investments and $ 18.5 million of long-term restricted cash. The Company believes that its balance of cash, cash equivalents, restricted cash and investments as of the date of the issuance of this report is sufficient to fund its current operational plan for at least the next twelve months, though it may pursue raising additional cash resources through public or private debt and equity financings, seek funded research and development arrangements and additional government contracts and establish collaborations with or license its technology to other companies. |
SUMMARY OF SIGNIFICANT ACCOUNTI
SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES | 6 Months Ended |
Jun. 30, 2022 | |
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 the Company 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, 2021 which are contained in the Company’s Annual Report on Form 10-K for the year ended December 31, 2021, filed with the U.S. Securities and Exchange Commission on March 1, 2022. The results for the three and six months ended June 30, 2022 are not necessarily indicative of the results to be expected for the full year. Estimates and Uncertainties The preparation of the unaudited condensed consolidated financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets, liabilities, equity, revenue, expenses and the disclosure of contingent assets and liabilities. Actual results could differ from those estimates. Concentration of Credit Risk Financial instruments that potentially subject the Company to concentrations of credit risk consist of accounts receivable from customers, cash held at financial institutions and cash equivalents and investments. As of June 30, 2022 , the majority of the Company’s accounts receivable arose from product sales in the U.S. and all customers have standard payment terms that generally require payment within 60 to 91 days . Outside of the U.S., the majority of the Company’s customers have payment terms ranging between 45 and 150 days . Three individual c ustomers accounted for 50 %, 32 % and 7 % of net product revenues for the three months ended June 30, 2022 and 49 %, 34 % and 7 % o f net product revenues for the six months ended June 30, 2022 . Three individual customers accounted for 46 %, 40 % and 11 % of net product revenues for the three months ended June 30, 2021 and 48 %, 40 % and 9 % of net product revenues for the six months ended June 30, 2021. Three individual customers accounted for 40 %, 36 % and 7 % of accounts receivable from product sales as of June 30, 2022 and 41 %, 41 % and 10 % of accounts receivable from product sales as of December 31, 2021. The Company monitors the financial performance and creditworthiness of its customers so that it can properly assess and respond to changes in its customers’ credit profile. As of June 30, 2022, the Company believes that such customers are of high credit quality. As of June 30, 2022 , the Company’s cash was concentrated at three financial institutions in the U.S., which potentially exposes the Company to credit risks. However, the Company does not believe that there is significant risk of non-performance by the financial institutions. 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. Significant Accounting Policies For details about the Company's accounting policies, please read Note 2, Summary of Significant Accounting Policies and Recent Accounting Pronouncements of the Annual Report on Form 10-K for the year ended December 31, 2021. There have not been any material changes to the Company's accounting policies through June 30, 2022 . |
LICENSE AND COLLABORATION AGREE
LICENSE AND COLLABORATION AGREEMENTS | 6 Months Ended |
Jun. 30, 2022 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
LICENSE AND COLLABORATION AGREEMENTS | 3. LICENSE AND COLLABORATION AGREEMENTS F. Hoffman-La Roche Ltd. For the three and six months ended June 30, 2022 and 2021, the Company recognized $ 22.3 million and $ 44.3 million of collaboration revenue associated with the license, collaboration and option agreement (the “Roche Agreement”) with F. Hoffman-La Roche Ltd. (“Roche”). As of June 30, 2022, the Company has total deferred revenue of $ 619.2 million associated with the Roche Agreement, of which $ 89.2 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 June 30, 2022 and December 31, 2021. The costs associated with co-development activities performed under the Roche Agreement are included in operating expenses, with any reimbursement of costs by Roche reflected as a reduction of such expenses when the related expense is incurred. For the three months and six months ended June 30, 2022, costs reimbursable by Roche and reflected as a reduction to operating expenses were $ 26.4 million and $ 44.1 million, respectively. For the three and six months ended June 30, 2021 , costs reimbursable by Roche and reflected as a reduction to operating expenses were $ 18.0 million and $ 31.4 million, respectively. As of June 30, 2022, there was $ 26.4 million of collaboration receivable included in other current assets. Lysogene SA and Henogen SA In October 2018, the Company entered into a license and collaboration agreement to develop and commercialize LYS-SAF302, a gene therapy to treat Mucopolysaccharidosis type IIIA as well as an equity investment agreement with Lysogene SA (“Lysogene”). Under the license and collaboration agreement, in addition to the payment of up-front fees, the Company may be liable for a total of $ 102.8 million in development, regulatory and sales milestones. Furthermore, the Company may be required to make tiered royalty payments based on net sales of the LYS-SAF302 product subsequent to its commercialization. Beginning January 1, 2020, the Company began to reimburse Lysogene for expenses incurred in connection with development activities of LYS-SAF302. As of June 30, 2022, the Company owns 1,140,728 shares of common stock issued by Lysogene and recorded $ 0.8 million of equity investment in Lysogene as an other non-current asset in the Company’s consolidated balance sheets. The Company sent a termination notice to Lysogene on January 11, 2022 to notify them of the Company's intent to terminate the license and collaboration agreement. The termination became effective July 11, 2022. The Company is not obligated to pay early termination penalties to Lysogene but has agreed to pay certain research and development reimbursements incurred in the six months following termination, which are not expected to be material. The Company entered into a development, manufacturing and supply agreement with Henogen SA ("Henogen") in December 2019. Pursuant to the terms of the agreement, Henogen agreed to reserve manufacturing capacity within their facility to develop, manufacture and supply the Company with LYS-SAF302. On June 9, 2022, the Company and Henogen entered into an agreement to terminate the development, manufacturing and supply agreement. As a result, the Company recorded a charge of $ 17.1 million during the three months ended June 30, 2022, which was recorded in research and development expenses 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 exercised, the Company may be required to make up to $ 7.0 million in research milestone payments. Under these agreements, there are $ 187.1 million in potential option payments to be made by the Company upon the determination to exercise the options. Additionally, if the options for each agreement are executed, the Company would incur additional contingent obligations and may be required to make development, regulatory, and sales milestone payments and tiered royalty payments based on the net sales of the developed products upon commercialization. For the three and six months ended June 30, 2022 and 2021, the Company recognized $ 6.0 million and $ 3.0 million of research, option and milestone expense, respectively. As of June 30, 2022, the Company has not exercised any options nor have any additional research milestone payments become probable of occurring. Milestone Obligations The Company has license and collaboration agreements in place for which it could be obligated to pay, in addition to the payment of up-front fees upon execution of the agreements, certain milestone payments as a product candidate proceeds from the submission of an investigational new drug application through approval for commercial sale and beyond. As of June 30, 2022, the Company may be obligated to make up to $ 3.7 billion in future development, regulatory, commercial and up-front royalty milestone payments associated with its license and collaboration agreements. These obligations exclude potential future option and milestone payments for options that have yet to be exercised within agreements entered into by the Company as of June 30, 2022, which are discussed above. For the three months and six months ended June 30, 2022, the Company recognized up-front and development milestone expenses approximately $ 8.8 million as research and development expense in the accompanying unaudited condensed consolidated statements of operations and comprehensive loss. For the three and six months ended June 30, 2021 , the Company recognized up-front, development milestone, settlement and other expenses of $ 31.7 million and $ 45.7 million, respectively, as research and development expense in the accompanying unaudited condensed consolidated statements of operations and comprehensive loss. |
FAIR VALUE MEASUREMENTS
FAIR VALUE MEASUREMENTS | 6 Months Ended |
Jun. 30, 2022 | |
Fair Value Disclosures [Abstract] | |
FAIR VALUE MEASUREMENTS | 4. 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 six months ended June 30, 2022 and June 30, 2021 , 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 valuation techniques it utilizes to determine such fair value: Fair Value Measurement as of June 30, 2022 Total Level 1 Level 2 Level 3 (in thousands) Assets Money market funds $ 464,060 $ 464,060 $ — $ — Commercial paper 226,674 — 226,674 — Government and government agency bonds 639,664 — 639,664 — Corporate bonds 199,103 — 199,103 — Strategic equity investments 33,234 822 — 32,412 Certificates of deposit 36,950 — 36,950 — Total assets $ 1,599,685 $ 464,882 $ 1,102,391 $ 32,412 Liabilities Contingent consideration $ 43,600 $ — $ — $ 43,600 Total liabilities $ 43,600 $ — $ — $ 43,600 Fair Value Measurement as of December 31, 2021 Total Level 1 Level 2 Level 3 (in thousands) Assets Money market funds $ 1,562,358 $ 1,562,358 $ — $ — Strategic equity investments 34,892 2,480 — 32,412 Certificates of deposit 250 250 — — Total assets $ 1,597,500 $ 1,565,088 $ — $ 32,412 Liabilities Contingent consideration $ 43,600 $ — $ — $ 43,600 Total liabilities $ 43,600 $ — $ — $ 43,600 The Company’s assets with fair value categorized as Level 1 within the fair value hierarchy include money market funds and the Company’s strategic investment in Lysogene. The Company's assets with 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 pricing services use observable market inputs to determine value, which primarily consist of reportable trades. Certain of the government and government agency bonds with original maturities of less than three months are presented as cash equivalents on the unaudited condensed consolidated balance sheets as of June 30, 2022. The Company’s assets with fair value categorized as Level 3 within the fair value hierarchy consist of a strategic investment in Series A preferred stock of Lacerta Therapeutics, Inc. (“Lacerta”) and strategic investments in two other private biotechnology companies. For more information related to Lacerta, please read Note 3, License and Collaboration Agreements of the Company’s Annual Report on Form 10-K for the year ended December 31, 2021. 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 option pricing model include historical volatility of similar public companies, estimated term through Lacerta’s potential exit and a risk-free rate based on certain U.S. Treasury rates. The investments in the other two private companies are recorded at fair value at the time of purchase as measured by their respective investment cost. At the end of each reporting period, the fair value of the Company's strategic investments will be adjusted if the issuers are to issue similar or identical equity securities or when there is a triggering event for impairment. There were no valuation measurement events related to the fair value of the strategic investments during the six months ended June 30, 2022 and 2021, respectively, as no impairment indicators were identified nor were similar securities issued. The Company’s contingent consideration liability with fair value categorized as Level 3 within the fair value hierarchy relates to the regulatory-related contingent payments to Myonexus Therapeutics, Inc. (“Myonexus”) selling shareholders as well as to two academic institutions under separate license agreements that meet the definition of a derivative. For more information related to Myonexus, please read Note 3, License and Collaboration Agreements of the Company’s Annual Report on Form 10-K for the year ended December 31, 2021 . 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. For the six months ended June 30, 2022 and 2021, there have been no changes to the fair value of the contingent consideration liability. As of June 30, 2022, the contingent consideration was recorded as a non-current liability on the Company's unaudited condensed consolidated balance sheets. The fair value of the senior notes due on November 15, 2024 (the “2024 Notes”) is based on open market trades and is classified as Level 1 in the fair value hierarchy. For more information related to the 2024 Notes, please read Note 13, Indebtedness of the Company’s Annual Report on Form 10-K for the year ended December 31, 2021. As of June 30, 2022 and December 31, 2021, the fair value of the 2024 Notes was approximately $ 726.2 million and $ 846.1 million, respectively. The fair value of the December 13, 2019 term loan (the “December 2019 Term Loan”) is classified as Level 2 in the fair value hierarchy and is determined using a discounted cash flow analysis with market interest rates adjusted for credit risk as a significant input. As of June 30, 2022 and December 31, 2021, the fair value of the December 2019 Term Loan was approximately $ 548.4 million and $ 576.1 million, respectively. The carrying values of the 2024 Notes and December 2019 Term Loan were $ 564.7 million and $ 536.1 million as of June 30, 2022 and $ 563.7 million and $ 533.2 million as of December 31, 2021, respectively. The carrying amounts reported in the unaudited condensed consolidated balance sheets for cash and cash equivalents, accounts receivable and accounts payable approximated fair value because of the short-term maturity of these financial instruments |
CASH, CASH EQUIVALENTS AND MARK
CASH, CASH EQUIVALENTS AND MARKETABLE SECURITIES | 6 Months Ended |
Jun. 30, 2022 | |
Cash and Cash Equivalents [Abstract] | |
CASH, CASH EQUIVALENTS AND MARKETABLE SECURITIES | 5. 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 $ 464,060 $ 1,562,358 Government and government agency bonds 42,937 — Total $ 506,997 $ 1,562,358 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 June 30, 2022 was approximately four months . T he Company did no t hold any short-term investments classified as available-for-sale securities as of December 31, 2021. The following tables summarize the Company ’s cash, cash equivalents and short-term investments as of the periods indicated: As of June 30, 2022 Amortized Gross Gross Fair (in thousands) Cash and money market funds $ 825,628 $ — $ — $ 825,628 Commercial paper 226,674 — — 226,674 Government and government agency bonds 641,219 5 ( 1,560 ) 639,664 Corporate bonds 200,013 2 ( 912 ) 199,103 Certificates of deposit 36,950 — — 36,950 Total cash, cash equivalents and investments $ 1,930,484 $ 7 $ ( 2,472 ) $ 1,928,019 As reported: Cash and cash equivalents $ 868,574 $ — $ ( 9 ) $ 868,565 Short-term investments 1,061,910 7 ( 2,463 ) 1,059,454 Total cash, cash equivalents and investments $ 1,930,484 $ 7 $ ( 2,472 ) $ 1,928,019 As of December 31, 2021 Amortized Gross Gross Fair (in thousands) Cash and money market funds $ 2,115,869 $ — $ — $ 2,115,869 Total cash and cash equivalents $ 2,115,869 $ — $ — $ 2,115,869 As reported: Cash and cash equivalents $ 2,115,869 $ — $ — $ 2,115,869 Total cash and cash equivalents $ 2,115,869 $ — $ — $ 2,115,869 |
ACCOUNTS RECEIVABLE AND RESERVE
ACCOUNTS RECEIVABLE AND RESERVES FOR PRODUCT SALES | 6 Months Ended |
Jun. 30, 2022 | |
Receivables, Net, Current [Abstract] | |
ACCOUNTS RECEIVABLE AND RESERVES FOR PRODUCT SALES | 6. ACCOUNTS RECEIVABLE AND RESERVES FOR PRODUCT SALES As of June 30, 2022 and December 31, 2021, the Company's accounts receivable were $ 203.9 million and $ 153.0 million, respectively, both of which were related to products sales receivable, net of 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, 2021 $ 799 $ 60,506 $ 2,798 $ 6,363 $ 70,466 Provision 5,642 50,361 6,192 19,416 81,611 Payments/credits ( 6,232 ) ( 43,293 ) ( 5,639 ) ( 11,460 ) ( 66,624 ) Balance, as of June 30, 2022 $ 209 $ 67,574 $ 3,351 $ 14,319 $ 85,453 Chargebacks Rebates Prompt Pay Other Accruals Total (in thousands) Balance, as of December 31, 2020 $ 2,281 $ 41,771 $ 1,949 $ 4,969 $ 50,970 Provision 5,665 33,221 4,005 6,848 49,739 Payments/credits ( 6,887 ) ( 26,992 ) ( 3,533 ) ( 7,236 ) ( 44,648 ) Balance, as of June 30, 2021 $ 1,059 $ 48,000 $ 2,421 $ 4,581 $ 56,061 The following table summarizes the total reserves included in the Company’s unaudited condensed consolidated balance sheets for the periods indicated: As of As of (in thousands) Reduction to accounts receivable $ 17,092 $ 8,321 Component of accrued expenses 68,361 62,145 Total reserves $ 85,453 $ 70,466 |
INVENTORY
INVENTORY | 6 Months Ended |
Jun. 30, 2022 | |
Inventory Disclosure [Abstract] | |
INVENTORY | 7. INVENTORY The following table summarizes the components of the Company’s inventory for the periods indicated: As of As of (in thousands) Raw materials $ 42,398 $ 58,822 Work in progress 253,200 230,194 Finished goods 35,499 26,717 Total inventory $ 331,097 $ 315,733 There were no material inventory reserves as of June 30, 2022 or December 31, 2021. Non-current inventory, which consists of raw materials and work in progress inventory, is included in other non-current assets in the Company's unaudited condensed consolidated balance sheets. Non-current inventory is anticipated to be consumed beyond our 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 $ 208,095 $ 186,212 Other non-current assets 123,002 129,521 Total inventory $ 331,097 $ 315,733 |
OTHER ASSETS
OTHER ASSETS | 6 Months Ended |
Jun. 30, 2022 | |
Deferred Costs, Capitalized, Prepaid, and Other Assets Disclosure [Abstract] | |
OTHER ASSETS | 8. 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 $ 69,502 $ 93,656 Collaboration receivable 26,421 18,647 Prepaid clinical and pre-clinical expenses 10,707 12,667 Prepaid maintenance services 7,644 8,452 Prepaid insurance 3,280 5,282 Prepaid research expenses 1,973 3,082 Other 9,805 7,242 Total other current assets $ 129,332 $ 149,028 The following table summarizes the Company’s other non-current assets for each of the periods indicated: As of As of (in thousands) Non-current inventory $ 123,002 $ 129,521 Manufacturing-related deposits and prepaids 103,819 112,765 Strategic investments 33,234 34,892 Restricted cash and investments 18,499 9,904 Prepaid clinical expenses 2,103 2,007 Other 3,543 3,860 Total other non-current assets $ 284,200 $ 292,949 |
ACCRUED EXPENSES
ACCRUED EXPENSES | 6 Months Ended |
Jun. 30, 2022 | |
Payables and Accruals [Abstract] | |
ACCRUED EXPENSES | 9. ACCRUED EXPENSES The following table summarizes the Company’s accrued expenses for each of the periods indicated: As of As of (in thousands) Accrued contract manufacturing costs $ 204,867 $ 104,311 Product revenue related reserves 68,361 62,145 Accrued clinical and pre-clinical costs 33,832 25,955 Accrued employee compensation costs 32,191 48,299 Accrued royalties 14,746 11,965 Accrued professional fees 13,574 9,381 Accrued collaboration cost sharing 2,868 2,887 Accrued income taxes 2,485 216 Accrued sponsored research agreements 1,651 3,377 Accrued fixed assets 1,462 127 Accrued interest expense 1,092 1,045 Other 6,570 1,989 Total accrued expenses $ 383,699 $ 271,697 |
STOCK-BASED COMPENSATION
STOCK-BASED COMPENSATION | 6 Months Ended |
Jun. 30, 2022 | |
Share-Based Payment Arrangement [Abstract] | |
STOCK-BASED COMPENSATION | 10. STOCK-BASED COMPENSATION The following table summarizes the Company’s stock awards granted for each of the periods indicated: For the Three Months Ended June 30, For the Six Months Ended June 30, 2022 2021 2022 2021 Grants Weighted Grants Weighted Grants Weighted Grants Weighted Stock options 207,583 $ 43.40 72,110 $ 39.79 1,499,120 $ 46.49 1,431,747 $ 48.87 Restricted stock units* 147,075 $ 72.99 37,240 $ 75.30 859,510 $ 78.78 726,607 $ 86.53 * Included in 2022 RSUs are 38,500 shares with performance conditions which are related to regulatory approval of certain of the Company's product candidates. As of June 30, 2022, none of the performance conditions are probable of being achieved. If the performance milestones are achieved within the required time frame, the Company may recognize up to $ 3.1 million of stock-based compensation related to these grants. 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. Additionally, the Chief Executive Officer is subject to a one-year post-exercise restriction to sell, transfer or dispose shares acquired upon the exercise of any options that vest after deduction of any shares withheld or sold to pay the applicable aggregate exercise price and/or withholding taxes. To determine the incremental compensation cost of the modification, the fair value of the modified awards was compared to the fair value of the original awards measured immediately before its terms or conditions were modified. As the Vested Tranche became immediately vested on the Effective Date, the Vested Tranche does not have service or market conditions. As such, the post-modification fair value for the Vested Tranche is based on the Black-Scholes-Merton option-pricing model, while the pre-modification fair value is based on a lattice model with Monte Carlo simulations. The incremental compensation cost of $ 43.9 million was immediately recognized as stock-based compensation expense in the three months ended June 30, 2022. 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. As a result of the modification, the Company is expected to recognize incremental compensation cost of $ 82.1 million associated with the Unvested Tranche. The incremental 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 approximately 1.3 years. For the three months ended June 30, 2022, the Company recorded $ 29.8 million of stock-based compensation expense related to the Unvested Tranche. Stock-based Compensation Expense For the three months ended June 30, 2022 and 2021, total stock-based compensation expense was $ 102.9 million and $ 29.0 million, respectively. For the six months ended June 30, 2022 and 2021, total stock-based compensation expense was $ 132.1 million and $ 57.5 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 Six Months Ended 2022 2021 2022 2021 (in thousands) Research and development $ 14,467 $ 12,860 $ 27,535 $ 23,986 Selling, general and administrative 88,425 16,109 104,555 33,491 Total stock-based compensation expense $ 102,892 $ 28,969 $ 132,090 $ 57,477 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 Six Months Ended 2022 2021 2022 2021 (in thousands) Stock options $ 89,155 $ 17,587 $ 105,561 $ 34,606 Restricted stock awards/units 12,378 10,573 23,743 20,504 Employee stock purchase plan 1,359 809 2,786 2,367 Total stock-based compensation expense $ 102,892 $ 28,969 $ 132,090 $ 57,477 |
OTHER (LOSS) INCOME, NET
OTHER (LOSS) INCOME, NET | 6 Months Ended |
Jun. 30, 2022 | |
Other Income and Expenses [Abstract] | |
OTHER (LOSS) INCOME, NET | 11. OTHER (LOSS) INCOME, NET The following table summarizes other loss, net f or the periods indicated: For the Three Months Ended For the Six Months Ended 2022 2021 2022 2021 (in thousands) Interest expense $ ( 16,028 ) $ ( 15,831 ) $ ( 31,824 ) $ ( 31,482 ) Interest income 2,409 52 2,582 111 Gain from sale of Priority Review — 102,000 — 102,000 Other expense, net ( 3,342 ) ( 406 ) ( 4,984 ) ( 342 ) Total other (loss) income, net $ ( 16,961 ) $ 85,815 $ ( 34,226 ) $ 70,287 In February 2021, the Company the Company entered into an agreement to sell the rare pediatric disease Priority Review Voucher (“PRV”) it received from the FDA in connection with the approval of AMONDYS 45. Following the termination of the applicable waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976, as amended, in April 2021, the Company completed its sale of the PRV and received proceeds of $ 102.0 million, with no commission costs, which was recorded as a gain from sale of the PRV as it did not have a carrying value at the time of the sale. For more information related to the PRV, please read Note 4, Gain from Sale of Priority Review Voucher of the Company’s Annual Report on Form 10-K for the year ended December 31, 2021. |
LEASES
LEASES | 6 Months Ended |
Jun. 30, 2022 | |
Leases [Abstract] | |
LEASES | 12. LEASES The Company has real estate operating leases in Cambridge, Andover and Burlington, Massachusetts, Dublin and Columbus, Ohio, and Durham, NC that provide for scheduled annual rent increases throughout each lease’s term. The Company has also identified a lease 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 manufacturing and supply agreements with Thermo Fisher Scientific, Inc. (“Thermo”) and Catalent, Inc. (“Catalent”), please read Note 21, Commitments and Contingencies of the Company's Annual Report on Form 10-K for the year ended December 31, 2021. 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 lease is expected to commence prior to December 31, 2022. The Company is not involved in the initial construction of the core and shell of the building and will record the lease liability and right-of-use (“ROU”) asset on its unaudited condensed consolidated balance sheets when it obtains control of the premises, which is currently expected to be during the second half of 2022. The initial term of the Bedford Lease is anticipated to be 15 years commencing at the earlier of (i) date the certificate of occupancy is issued; or (ii) January 1, 2024, representing the commencement of the Company’s obligation to pay rent for the premises. 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. Undiscounted rent payments due over the 15 -year 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 of $ 72.0 million to be used towards costs incurred by the Company in the design and construction of the premises. 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. Columbus, Ohio On December 22, 2018, the Company entered into a lease agreement for a research and development facility in Columbus, Ohio (the “Columbus Lease”). On May 19, 2022 (the "Columbus Lease Amendment Date"), the Company entered into an amendment to the Columbus Lease to expand the footprint and extend the lease term (the “Columbus Amendment”). The Columbus Amendment expands from its current form of approximately 78,000 square feet to 167,000 square feet through a series of expansion spaces commencing at various periods through January 1, 2025. Each expansion space commences on the date which approximates when the landlord will deliver control of that space for the Company to carry out design and construction activities (the “Columbus Commencement Date”). The Company is obligated to pay rent on each expansion space nine months after the Columbus Commencement Date. The Columbus Lease and Columbus Amendment expire on December 31, 2036, and the Company has options to extend the lease by five years in both 2036 and 2041. Each option is exercisable under certain conditions and at a market rate determined in accordance with the lease agreement. Undiscounted rent payments due over the 15 -year term from the Columbus Lease Amendment Date aggregate to $ 38.9 million. On June 1, 2022, the Company commenced design and constructions activities on an area of the premises of approximately 18,000 square feet (the “Second Expansion Space”) and, therefore, it was determined that the lease related to the Second Expansion Space had commenced on that date. As a result, the Company recorded an additional ROU asset and lease liability of $ 7.3 million related to the extended term of the lease of the original facility as of June 30, 2022 . |
NET LOSS PER SHARE
NET LOSS PER SHARE | 6 Months Ended |
Jun. 30, 2022 | |
Earnings Per Share [Abstract] | |
NET LOSS PER SHARE | 13. NET LOSS PER SHARE Basic net loss per share is computed by dividing net loss by the weighted-average number of shares of common stock outstanding. Diluted net loss per share is computed by dividing net loss by the weighted-average number of shares of common stock and dilutive common stock equivalents outstanding. For the three and six months ended June 30, 2022 and 2021, there were no differences between basic and diluted net loss per share since the effect of common stock equivalents would be anti-dilutive due to the net loss position and, therefore, would be excluded from the diluted net loss per share calculation. For the Three Months Ended For the Six Months Ended 2022 2021 2022 2021 (in thousands, except per share amounts) (in thousands, except per share amounts) Net loss $ ( 231,481 ) $ ( 81,405 ) $ ( 336,506 ) $ ( 248,655 ) Weighted-average common shares outstanding - basic 87,511 79,746 87,383 79,601 Effect of dilutive securities* — — — — Weighted-average common shares outstanding - diluted 87,511 79,746 87,383 79,601 Net loss per share - basic and diluted $ ( 2.65 ) $ ( 1.02 ) $ ( 3.85 ) $ ( 3.12 ) * For the three and six months ended June 30, 2022 and 2021, stock options, RSAs, RSUs, and ESPP to purchase 11.3 million and 9.8 million shares of the Company’s common stock, respectively, were excluded from the diluted net loss per share calculation as their effect would have been anti-dilutive. The Company accounts for the effect of its 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 more dilutive. During the three and six months ended June 30, 2022 and June 30, 2021 , respectively, the inclusion of the potential share settlement of the 2024 Notes was anti-dilutive. Accordingly, the potential conversion of 7,763,552 shares has been excluded from the computation of diluted net loss per share as of June 30, 2022 and June 30, 2021 , respectively. |
COMMITMENTS AND CONTINGENCIES
COMMITMENTS AND CONTINGENCIES | 6 Months Ended |
Jun. 30, 2022 | |
Commitments and Contingencies Disclosure [Abstract] | |
COMMITMENTS AND CONTINGENCIES | 14. 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) 2022 (July-December) $ 529,689 2023 321,520 2024 135,285 2025 98,798 2026 54,720 Thereafter 109,440 Total manufacturing commitments* $ 1,249,452 * Total manufacturing commitments includes the Catalent Inc. manufacturing and supply agreement, for which the Company has right of use assets and lease liabilities recorded on the unaudited condensed consolidated balance sheets as of June 30, 2022. For more information, please read Note 21, Commitments and Contingencies of the Company’s Annual Report on Form 10-K for the year ended December 31, 2021. Additionally, should the Company obtain regulatory approval for any drug product candidate produced as a part of the Company’s manufacturing obligations above, additional minimum batch requirements with the respective manufacturing parties would be required. Thermo Fisher Scientific, Inc. Under the development, commercial manufacturing, and supply agreement (as amended) with Thermo Fisher Scientific, Inc. (“Thermo”), the Company has committed to annual guaranteed purchases on a take-or-pay basis regardless of whether services or goods are ordered. During the three months ended June 30, 2022, the Company determined that it is probable that it will not satisfy the total guaranteed purchase requirements by December 31, 2022 based on work orders executed to date and the time remaining in the fiscal year 2022 to complete development work. As such, the Company recognized a loss of approximately $ 53.0 million during the three months ended June 30, 2022, reflecting the estimated shortfall related to the annual guaranteed purchase requirement for the manufacturing and supply of gene therapy materials. The loss has been classified as research and development expense in the accompanying unaudited condensed consolidated statement of operations and comprehensive loss. For more information related to Thermo, please read Note 21, Commitments and Contingencies of the Annual Report on Form 10-K for the year ended December 31, 2021 . 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 June 30, 2022. On September 15, 2020, REGENXBIO INC. (“RegenX”) and the Trustees of the University of Pennsylvania filed a lawsuit against the Company and Sarepta Therapeutics Three, LLC (together, “Sarepta”), in the U.S. District Court for the District of Delaware. The plaintiffs assert patent infringement of U.S. Patent No. 10,526,617 (“the ‘617 Patent”) under 35 U.S.C.§§ 271(a)-(c) based on Sarepta’s alleged direct or indirect manufacture and use of the patented cultured host cell technology allegedly used to make adeno-associated virus (“AAV”) gene therapy products, including SRP-9001. Specifically, the Complaint essentially includes the allegation that Sarepta’s use, and the use by its contract manufacturers on its behalf, of a host cell containing a recombinant acid molecule that encodes a capsid protein having at least 95% amino acid identity to AAVrh10 infringes upon the ‘617 Patent asserted by RegenX. Plaintiffs seek injunctive relief, a judgment of infringement 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. On July 13, 2021, Nippon Shinyaku Co., Ltd. (“Nippon Shinyaku” or “NS”) filed a lawsuit against the Company in the U.S. District Court for the District of Delaware. NS asserts a claim for breach of contract arising from Sarepta filing seven petitions for Inter Partes Review (“IPR Petitions”) with the Patent Trial and Appeal Board at the USPTO (PTAB Case Nos. IPR2021-01134, IPR2021-01135, IPR2021-01136, IPR2021-01137, IPR2021-01138, IPR2021-01139, IPR2021-01140) in which Sarepta 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. On July 29, 2022, the Court ordered the parties to submit a further updated proposed scheduling order. Case scheduling in district court, including a trial date, will follow. |
SUMMARY OF SIGNIFICANT ACCOUN_2
SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES (Policies) | 6 Months Ended |
Jun. 30, 2022 | |
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 the Company 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, 2021 which are contained in the Company’s Annual Report on Form 10-K for the year ended December 31, 2021, filed with the U.S. Securities and Exchange Commission on March 1, 2022. The results for the three and six months ended June 30, 2022 are not necessarily indicative of the results to be expected for the full year. |
Estimates and Uncertainties | Estimates and Uncertainties The preparation of the unaudited condensed consolidated financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets, liabilities, equity, revenue, expenses and the disclosure of contingent assets and liabilities. Actual results could differ from those estimates. |
Concentration of Credit Risk | Concentration of Credit Risk Financial instruments that potentially subject the Company to concentrations of credit risk consist of accounts receivable from customers, cash held at financial institutions and cash equivalents and investments. As of June 30, 2022 , the majority of the Company’s accounts receivable arose from product sales in the U.S. and all customers have standard payment terms that generally require payment within 60 to 91 days . Outside of the U.S., the majority of the Company’s customers have payment terms ranging between 45 and 150 days . Three individual c ustomers accounted for 50 %, 32 % and 7 % of net product revenues for the three months ended June 30, 2022 and 49 %, 34 % and 7 % o f net product revenues for the six months ended June 30, 2022 . Three individual customers accounted for 46 %, 40 % and 11 % of net product revenues for the three months ended June 30, 2021 and 48 %, 40 % and 9 % of net product revenues for the six months ended June 30, 2021. Three individual customers accounted for 40 %, 36 % and 7 % of accounts receivable from product sales as of June 30, 2022 and 41 %, 41 % and 10 % of accounts receivable from product sales as of December 31, 2021. The Company monitors the financial performance and creditworthiness of its customers so that it can properly assess and respond to changes in its customers’ credit profile. As of June 30, 2022, the Company believes that such customers are of high credit quality. As of June 30, 2022 , the Company’s cash was concentrated at three financial institutions in the U.S., which potentially exposes the Company to credit risks. However, the Company does not believe that there is significant risk of non-performance by the financial institutions. 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. |
Significant Accounting Policies | Significant Accounting Policies For details about the Company's accounting policies, please read Note 2, Summary of Significant Accounting Policies and Recent Accounting Pronouncements of the Annual Report on Form 10-K for the year ended December 31, 2021. There have not been any material changes to the Company's accounting policies through June 30, 2022 . |
FAIR VALUE MEASUREMENTS (Tables
FAIR VALUE MEASUREMENTS (Tables) | 6 Months Ended |
Jun. 30, 2022 | |
Fair Value Disclosures [Abstract] | |
Assets and Liabilities Measured and Carried at Fair Value | The tables below present information about the Company’s financial assets and liabilities that are measured and carried at fair value and indicate the level within the fair value hierarchy of valuation techniques it utilizes to determine such fair value: Fair Value Measurement as of June 30, 2022 Total Level 1 Level 2 Level 3 (in thousands) Assets Money market funds $ 464,060 $ 464,060 $ — $ — Commercial paper 226,674 — 226,674 — Government and government agency bonds 639,664 — 639,664 — Corporate bonds 199,103 — 199,103 — Strategic equity investments 33,234 822 — 32,412 Certificates of deposit 36,950 — 36,950 — Total assets $ 1,599,685 $ 464,882 $ 1,102,391 $ 32,412 Liabilities Contingent consideration $ 43,600 $ — $ — $ 43,600 Total liabilities $ 43,600 $ — $ — $ 43,600 Fair Value Measurement as of December 31, 2021 Total Level 1 Level 2 Level 3 (in thousands) Assets Money market funds $ 1,562,358 $ 1,562,358 $ — $ — Strategic equity investments 34,892 2,480 — 32,412 Certificates of deposit 250 250 — — Total assets $ 1,597,500 $ 1,565,088 $ — $ 32,412 Liabilities Contingent consideration $ 43,600 $ — $ — $ 43,600 Total liabilities $ 43,600 $ — $ — $ 43,600 |
CASH, CASH EQUIVALENTS AND MA_2
CASH, CASH EQUIVALENTS AND MARKETABLE SECURITIES (Tables) | 6 Months Ended |
Jun. 30, 2022 | |
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 $ 464,060 $ 1,562,358 Government and government agency bonds 42,937 — Total $ 506,997 $ 1,562,358 |
Summary of Company Cash, Cash Equivalents and Short-Term Investments | The following tables summarize the Company ’s cash, cash equivalents and short-term investments as of the periods indicated: As of June 30, 2022 Amortized Gross Gross Fair (in thousands) Cash and money market funds $ 825,628 $ — $ — $ 825,628 Commercial paper 226,674 — — 226,674 Government and government agency bonds 641,219 5 ( 1,560 ) 639,664 Corporate bonds 200,013 2 ( 912 ) 199,103 Certificates of deposit 36,950 — — 36,950 Total cash, cash equivalents and investments $ 1,930,484 $ 7 $ ( 2,472 ) $ 1,928,019 As reported: Cash and cash equivalents $ 868,574 $ — $ ( 9 ) $ 868,565 Short-term investments 1,061,910 7 ( 2,463 ) 1,059,454 Total cash, cash equivalents and investments $ 1,930,484 $ 7 $ ( 2,472 ) $ 1,928,019 As of December 31, 2021 Amortized Gross Gross Fair (in thousands) Cash and money market funds $ 2,115,869 $ — $ — $ 2,115,869 Total cash and cash equivalents $ 2,115,869 $ — $ — $ 2,115,869 As reported: Cash and cash equivalents $ 2,115,869 $ — $ — $ 2,115,869 Total cash and cash equivalents $ 2,115,869 $ — $ — $ 2,115,869 |
ACCOUNTS RECEIVABLE AND RESER_2
ACCOUNTS RECEIVABLE AND RESERVES FOR PRODUCT SALES (Tables) | 6 Months Ended |
Jun. 30, 2022 | |
Receivables, Net, Current [Abstract] | |
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, 2021 $ 799 $ 60,506 $ 2,798 $ 6,363 $ 70,466 Provision 5,642 50,361 6,192 19,416 81,611 Payments/credits ( 6,232 ) ( 43,293 ) ( 5,639 ) ( 11,460 ) ( 66,624 ) Balance, as of June 30, 2022 $ 209 $ 67,574 $ 3,351 $ 14,319 $ 85,453 Chargebacks Rebates Prompt Pay Other Accruals Total (in thousands) Balance, as of December 31, 2020 $ 2,281 $ 41,771 $ 1,949 $ 4,969 $ 50,970 Provision 5,665 33,221 4,005 6,848 49,739 Payments/credits ( 6,887 ) ( 26,992 ) ( 3,533 ) ( 7,236 ) ( 44,648 ) Balance, as of June 30, 2021 $ 1,059 $ 48,000 $ 2,421 $ 4,581 $ 56,061 |
Summary of Total Reserves Included in Consolidated Balance Sheets | The following table summarizes the total reserves included in the Company’s unaudited condensed consolidated balance sheets for the periods indicated: As of As of (in thousands) Reduction to accounts receivable $ 17,092 $ 8,321 Component of accrued expenses 68,361 62,145 Total reserves $ 85,453 $ 70,466 |
INVENTORY (Tables)
INVENTORY (Tables) | 6 Months Ended |
Jun. 30, 2022 | |
Inventory Disclosure [Abstract] | |
Summary of Components of Inventory | The following table summarizes the components of the Company’s inventory for the periods indicated: As of As of (in thousands) Raw materials $ 42,398 $ 58,822 Work in progress 253,200 230,194 Finished goods 35,499 26,717 Total inventory $ 331,097 $ 315,733 |
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 $ 208,095 $ 186,212 Other non-current assets 123,002 129,521 Total inventory $ 331,097 $ 315,733 |
OTHER ASSETS (Tables)
OTHER ASSETS (Tables) | 6 Months Ended |
Jun. 30, 2022 | |
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 $ 69,502 $ 93,656 Collaboration receivable 26,421 18,647 Prepaid clinical and pre-clinical expenses 10,707 12,667 Prepaid maintenance services 7,644 8,452 Prepaid insurance 3,280 5,282 Prepaid research expenses 1,973 3,082 Other 9,805 7,242 Total other current assets $ 129,332 $ 149,028 |
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) Non-current inventory $ 123,002 $ 129,521 Manufacturing-related deposits and prepaids 103,819 112,765 Strategic investments 33,234 34,892 Restricted cash and investments 18,499 9,904 Prepaid clinical expenses 2,103 2,007 Other 3,543 3,860 Total other non-current assets $ 284,200 $ 292,949 |
ACCRUED EXPENSES (Tables)
ACCRUED EXPENSES (Tables) | 6 Months Ended |
Jun. 30, 2022 | |
Payables and Accruals [Abstract] | |
Summary of Accrued Expenses | The following table summarizes the Company’s accrued expenses for each of the periods indicated: As of As of (in thousands) Accrued contract manufacturing costs $ 204,867 $ 104,311 Product revenue related reserves 68,361 62,145 Accrued clinical and pre-clinical costs 33,832 25,955 Accrued employee compensation costs 32,191 48,299 Accrued royalties 14,746 11,965 Accrued professional fees 13,574 9,381 Accrued collaboration cost sharing 2,868 2,887 Accrued income taxes 2,485 216 Accrued sponsored research agreements 1,651 3,377 Accrued fixed assets 1,462 127 Accrued interest expense 1,092 1,045 Other 6,570 1,989 Total accrued expenses $ 383,699 $ 271,697 |
STOCK-BASED COMPENSATION (Table
STOCK-BASED COMPENSATION (Tables) | 6 Months Ended |
Jun. 30, 2022 | |
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 June 30, For the Six Months Ended June 30, 2022 2021 2022 2021 Grants Weighted Grants Weighted Grants Weighted Grants Weighted Stock options 207,583 $ 43.40 72,110 $ 39.79 1,499,120 $ 46.49 1,431,747 $ 48.87 Restricted stock units* 147,075 $ 72.99 37,240 $ 75.30 859,510 $ 78.78 726,607 $ 86.53 |
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 Six Months Ended 2022 2021 2022 2021 (in thousands) Research and development $ 14,467 $ 12,860 $ 27,535 $ 23,986 Selling, general and administrative 88,425 16,109 104,555 33,491 Total stock-based compensation expense $ 102,892 $ 28,969 $ 132,090 $ 57,477 |
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 Six Months Ended 2022 2021 2022 2021 (in thousands) Stock options $ 89,155 $ 17,587 $ 105,561 $ 34,606 Restricted stock awards/units 12,378 10,573 23,743 20,504 Employee stock purchase plan 1,359 809 2,786 2,367 Total stock-based compensation expense $ 102,892 $ 28,969 $ 132,090 $ 57,477 |
OTHER (LOSS) INCOME, NET (Table
OTHER (LOSS) INCOME, NET (Tables) | 6 Months Ended |
Jun. 30, 2022 | |
Other Income and Expenses [Abstract] | |
Summary of Other Loss | The following table summarizes other loss, net f or the periods indicated: For the Three Months Ended For the Six Months Ended 2022 2021 2022 2021 (in thousands) Interest expense $ ( 16,028 ) $ ( 15,831 ) $ ( 31,824 ) $ ( 31,482 ) Interest income 2,409 52 2,582 111 Gain from sale of Priority Review — 102,000 — 102,000 Other expense, net ( 3,342 ) ( 406 ) ( 4,984 ) ( 342 ) Total other (loss) income, net $ ( 16,961 ) $ 85,815 $ ( 34,226 ) $ 70,287 |
NET LOSS PER SHARE (Tables)
NET LOSS PER SHARE (Tables) | 6 Months Ended |
Jun. 30, 2022 | |
Earnings Per Share [Abstract] | |
Basic and Diluted Net Loss Per Share | For the three and six months ended June 30, 2022 and 2021, there were no differences between basic and diluted net loss per share since the effect of common stock equivalents would be anti-dilutive due to the net loss position and, therefore, would be excluded from the diluted net loss per share calculation. For the Three Months Ended For the Six Months Ended 2022 2021 2022 2021 (in thousands, except per share amounts) (in thousands, except per share amounts) Net loss $ ( 231,481 ) $ ( 81,405 ) $ ( 336,506 ) $ ( 248,655 ) Weighted-average common shares outstanding - basic 87,511 79,746 87,383 79,601 Effect of dilutive securities* — — — — Weighted-average common shares outstanding - diluted 87,511 79,746 87,383 79,601 Net loss per share - basic and diluted $ ( 2.65 ) $ ( 1.02 ) $ ( 3.85 ) $ ( 3.12 ) * For the three and six months ended June 30, 2022 and 2021, stock options, RSAs, RSUs, and ESPP to purchase 11.3 million and 9.8 million shares of the Company’s common stock, respectively, were excluded from the diluted net loss per share calculation as their effect would have been anti-dilutive. The Company accounts for the effect of its 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 more dilutive. During the three and six months ended June 30, 2022 and June 30, 2021 , respectively, the inclusion of the potential share settlement of the 2024 Notes was anti-dilutive. Accordingly, the potential conversion of 7,763,552 shares has been excluded from the computation of diluted net loss per share as of June 30, 2022 and June 30, 2021 , respectively. |
COMMITMENTS AND CONTINGENCIES (
COMMITMENTS AND CONTINGENCIES (Tables) | 6 Months Ended |
Jun. 30, 2022 | |
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) 2022 (July-December) $ 529,689 2023 321,520 2024 135,285 2025 98,798 2026 54,720 Thereafter 109,440 Total manufacturing commitments* $ 1,249,452 * Total manufacturing commitments includes the Catalent Inc. manufacturing and supply agreement, for which the Company has right of use assets and lease liabilities recorded on the unaudited condensed consolidated balance sheets as of June 30, 2022. For more information, please read Note 21, Commitments and Contingencies of the Company’s Annual Report on Form 10-K for the year ended December 31, 2021. |
Organization and Nature of Bu_2
Organization and Nature of Business - Additional Information (Detail) - USD ($) $ in Thousands | Jun. 30, 2022 | Dec. 31, 2021 | Jun. 30, 2021 |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |||
Cash, cash equivalents and investments | $ 1,946,600 | ||
Cash and cash equivalents | 868,565 | $ 2,115,869 | $ 1,697,275 |
Short-term investments | 1,059,454 | 0 | |
Restricted cash and investments | $ 18,499 | $ 9,904 |
Summary of Significant Accoun_3
Summary of Significant Accounting Policies - Additional Information (Detail) $ / shares in Units, $ in Thousands | 3 Months Ended | 6 Months Ended | 12 Months Ended | ||||
Jun. 30, 2022 USD ($) $ / shares | Mar. 31, 2022 USD ($) | Jun. 30, 2021 USD ($) $ / shares | Mar. 31, 2021 USD ($) | Jun. 30, 2022 USD ($) Segment $ / shares | Jun. 30, 2021 USD ($) $ / shares | Dec. 31, 2021 | |
Summary Of Significant Accounting Policies [Line Items] | |||||||
Number of operating segments | Segment | 1 | ||||||
Net loss | $ | $ (231,481) | $ (105,025) | $ (81,405) | $ (167,250) | $ (336,506) | $ (248,655) | |
Increase in diluted earnings per share (in dollar per share) | $ / shares | $ (2.65) | $ (1.02) | $ (3.85) | $ (3.12) | |||
Outside of U.S. [Member] | Product Revenues [Member] | Customer One [Member] | |||||||
Summary Of Significant Accounting Policies [Line Items] | |||||||
Concentration of credit risk percentage | 50% | 46% | 49% | 48% | |||
Outside of U.S. [Member] | Product Revenues [Member] | Customer Two [Member] | |||||||
Summary Of Significant Accounting Policies [Line Items] | |||||||
Concentration of credit risk percentage | 32% | 40% | 34% | 40% | |||
Outside of U.S. [Member] | Product Revenues [Member] | Customer Three [Member] | |||||||
Summary Of Significant Accounting Policies [Line Items] | |||||||
Concentration of credit risk percentage | 7% | 11% | 7% | 9% | |||
Outside of U.S. [Member] | Accounts Receivable [Member] | Customer One [Member] | |||||||
Summary Of Significant Accounting Policies [Line Items] | |||||||
Concentration of credit risk percentage | 40% | 41% | |||||
Outside of U.S. [Member] | Accounts Receivable [Member] | Customer Two [Member] | |||||||
Summary Of Significant Accounting Policies [Line Items] | |||||||
Concentration of credit risk percentage | 36% | 41% | |||||
Outside of U.S. [Member] | Accounts Receivable [Member] | Customer Three [Member] | |||||||
Summary Of Significant Accounting Policies [Line Items] | |||||||
Concentration of credit risk percentage | 7% | 10% | |||||
Minimum [Member] | |||||||
Summary Of Significant Accounting Policies [Line Items] | |||||||
Accounts receivable payment term | 60 days | ||||||
Minimum [Member] | Outside of U.S. [Member] | |||||||
Summary Of Significant Accounting Policies [Line Items] | |||||||
Accounts receivable payment term | 45 days | ||||||
Maximum [Member] | |||||||
Summary Of Significant Accounting Policies [Line Items] | |||||||
Accounts receivable payment term | 91 days | ||||||
Maximum [Member] | Outside of U.S. [Member] | |||||||
Summary Of Significant Accounting Policies [Line Items] | |||||||
Accounts receivable payment term | 150 days |
License and Collaboration Agr_2
License and Collaboration Agreements - Additional Information (Detail) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | 9 Months Ended | ||||
Jun. 30, 2022 | Jun. 30, 2021 | Jun. 30, 2022 | Jun. 30, 2021 | Sep. 30, 2021 | Dec. 31, 2021 | Oct. 31, 2018 | |
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||
Product revenues, net | $ 211,237 | $ 141,839 | $ 400,062 | $ 266,765 | |||
Settlement charge | 0 | 0 | 0 | 10,000 | |||
Research, option and milestone expense | 6,000 | 3,000 | 6,000 | 3,000 | |||
Up-front and development milestone expenses | 8,800 | 8,800 | |||||
Henogen SA [Member] | |||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||
Research and development expenses charges | 17,100 | ||||||
Collaborative Arrangement | |||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||
Payment for option exercise | 187,100 | ||||||
Stock options exercised or expired | |||||||
Development Milestone and Upfront Fee Recognized as Research and Development Expense | 31,700 | 45,700 | |||||
Collaborative Arrangement | Maximum [Member] | |||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||
Contingent research milestone payments | 7,000 | ||||||
Development Milestone and Settlement Upfront Fee Recognized as Research and Development Expense | 3,700,000 | 3,700,000 | |||||
Collaborative Arrangement | Roche Holding A.G. [Member] | |||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||
Product revenues, net | 22,300 | 44,300 | 22,300 | 44,300 | |||
Deferred Revenue | 619,200 | 619,200 | |||||
Deferred Revenue, Current | 89,200 | 89,200 | |||||
Deferred Revenue Separate Material Options Right | 485,000 | 485,000 | $ 485,000 | ||||
Research and development expense | 26,400 | $ 18,000 | 44,100 | $ 31,400 | |||
Collaboration Receivable | 26,400 | $ 26,400 | |||||
Equity Investment Agreement [Member] | Lysogene S.A. [Member] | |||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||
Development, regulatory and sales milestones payments to be paid | $ 102,800 | ||||||
Common stock purchased,shares | 1,140,728 | ||||||
Equity Investment Agreement [Member] | Lysogene S.A. [Member] | Other Noncurrent Assets [Member] | |||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||
Equity investment publicly traded security | $ 800 | $ 800 |
Gain from Sale of Priority Revi
Gain from Sale of Priority Review Voucher - Additional Information (Detail) $ in Millions | 1 Months Ended |
Apr. 30, 2021 USD ($) | |
F D A | |
Gain From Sale Of Intangible Asset [Line Items] | |
Proceeds from sale of rare pediatric disease priority review voucher, Net of commission | $ 102 |
Assets and Liabilities Measured
Assets and Liabilities Measured and Carried at Fair Value (Detail) - USD ($) $ in Thousands | Jun. 30, 2022 | Dec. 31, 2021 |
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total assets | $ 1,599,685 | $ 1,597,500 |
Contingent consideration | 43,600 | 43,600 |
Total liabilities | 43,600 | 43,600 |
Money Market Funds [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total assets | 464,060 | 1,562,358 |
Commercial Paper [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total assets | 226,674 | |
Government and Government Agency Bonds [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total assets | 639,664 | |
Corporate Bonds [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total assets | 199,103 | |
Strategic Equity Investments [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total assets | 33,234 | 34,892 |
Certificates of Deposit [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total assets | 36,950 | 250 |
Level 1 [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total assets | 464,882 | 1,565,088 |
Level 1 [Member] | Money Market Funds [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total assets | 464,060 | 1,562,358 |
Level 1 [Member] | Strategic Equity Investments [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total assets | 822 | 2,480 |
Level 1 [Member] | Certificates of Deposit [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total assets | 250 | |
Level 2 [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total assets | 1,102,391 | |
Level 2 [Member] | Commercial Paper [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total assets | 226,674 | |
Level 2 [Member] | Government and Government Agency Bonds [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total assets | 639,664 | |
Level 2 [Member] | Corporate Bonds [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total assets | 199,103 | |
Level 2 [Member] | Certificates of Deposit [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total assets | 36,950 | |
Level 3 [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total assets | 32,412 | 32,412 |
Contingent consideration | 43,600 | 43,600 |
Total liabilities | 43,600 | 43,600 |
Level 3 [Member] | Strategic Equity Investments [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Total assets | $ 32,412 | $ 32,412 |
Fair Value Measurements - Addit
Fair Value Measurements - Additional Information (Detail) - USD ($) $ in Millions | Jun. 30, 2022 | Dec. 31, 2021 |
2024 Convertible Notes [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Debt Instrument Fair Value | $ 726.2 | $ 846.1 |
Net carrying value | 564.7 | 563.7 |
2019 Term Loan [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Debt Instrument Fair Value | 548.4 | 576.1 |
Net carrying value | $ 536.1 | $ 533.2 |
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 | Jun. 30, 2022 | Dec. 31, 2021 |
Cash and Cash Equivalents [Line Items] | ||
Cash equivalents | $ 506,997 | $ 1,562,358 |
Money Market Funds [Member] | ||
Cash and Cash Equivalents [Line Items] | ||
Cash equivalents | 464,060 | |
Government and Government Agency Bonds [Member] | ||
Cash and Cash Equivalents [Line Items] | ||
Cash equivalents | $ 42,937 | 1,562,358 |
Corporate Bond [Member] | ||
Cash and Cash Equivalents [Line Items] | ||
Cash equivalents | $ 0 |
Cash, Cash Equivalents and Ma_3
Cash, Cash Equivalents and Marketable Securities - Additional Information (Detail) - USD ($) $ in Thousands | 6 Months Ended | |
Jun. 30, 2022 | Dec. 31, 2021 | |
Cash and Cash Equivalents [Line Items] | ||
Weighted average maturity period of available-for-sale securities | 4 months | |
Available for sale debt securities current, Fair value | $ 1,059,454 | $ 0 |
Summary of Company Cash, Cash E
Summary of Company Cash, Cash Equivalents and Short-term Investments (Detail) - USD ($) $ in Thousands | Jun. 30, 2022 | Dec. 31, 2021 | Jun. 30, 2021 |
Cash and Cash Equivalents [Line Items] | |||
Cash and cash equivalents, Amortized cost | $ 1,930,484 | ||
Cash and cash equivalents, Gross unrealized losses | (9) | ||
Cash and cash equivalents | 868,565 | $ 2,115,869 | $ 1,697,275 |
Cash, cash equivalents and investments, Amortized cost | 868,574 | 2,115,869 | |
Cash, cash equivalents and investments, Gross unrealized gains | 7 | ||
Cash, cash equivalents and investments, Gross unrealized losses | (2,472) | ||
Cash, cash equivalents and investments, Fair value | 1,928,019 | 2,115,869 | |
Available for sale debt securities current, Amortized cost | 1,061,910 | ||
Available for sale debt securities current, Gross unrealized gains | 7 | ||
Available for sale debt securities current, Gross unrealized losses | (2,463) | ||
Available for sale debt securities current, Fair value | 1,059,454 | 0 | |
Cash and Money Market Funds [Member] | |||
Cash and Cash Equivalents [Line Items] | |||
Cash and cash equivalents, Amortized cost | 825,628 | 2,115,869 | |
Cash and cash equivalents | 825,628 | 2,115,869 | |
Commercial Paper [Member] | |||
Cash and Cash Equivalents [Line Items] | |||
Available for sale debt securities, Fair value | 226,674 | ||
Cash and cash equivalents, Amortized cost | 226,674 | ||
Government and Government Agency Bonds [Member] | |||
Cash and Cash Equivalents [Line Items] | |||
Available for sale debt securities, Gross unrealized gains | 5 | ||
Available for sale debt securities, Gross unrealized losses | (1,560) | ||
Available for sale debt securities, Fair value | 639,664 | ||
Cash and cash equivalents, Amortized cost | 641,219 | ||
Cash, cash equivalents and investments, Fair value | $ 2,115,869 | ||
Corporate Bond [Member] | |||
Cash and Cash Equivalents [Line Items] | |||
Available for sale debt securities, Gross unrealized gains | 2 | ||
Available for sale debt securities, Gross unrealized losses | (912) | ||
Available for sale debt securities, Fair value | 199,103 | ||
Cash and cash equivalents, Amortized cost | 200,013 | ||
Certificates of Deposit [Member] | |||
Cash and Cash Equivalents [Line Items] | |||
Available for sale debt securities, Fair value | 36,950 | ||
Cash and cash equivalents, Amortized cost | $ 36,950 |
Accounts Receivable and Reser_3
Accounts Receivable and Reserves for Product Sales (Additional Information) (Details) - USD ($) $ in Millions | Jun. 30, 2022 | Dec. 31, 2021 |
Receivables, Net, Current [Abstract] | ||
Product sales receivable, net of discounts and allowances | $ 203.9 | $ 153 |
Summary of Change in Reserves f
Summary of Change in Reserves for Discounts and Allowances (Detail) - USD ($) $ in Thousands | 6 Months Ended | |
Jun. 30, 2022 | Jun. 30, 2021 | |
Accounts Notes And Loans Receivable [Line Items] | ||
Beginning balance | $ 70,466 | $ 50,970 |
Provision | 81,611 | 49,739 |
Payments/credits | (66,624) | (44,648) |
Ending balance | 85,453 | 56,061 |
Chargebacks [Member] | ||
Accounts Notes And Loans Receivable [Line Items] | ||
Beginning balance | 799 | 2,281 |
Provision | 5,642 | 5,665 |
Payments/credits | (6,232) | (6,887) |
Ending balance | 209 | 1,059 |
Rebates [Member] | ||
Accounts Notes And Loans Receivable [Line Items] | ||
Beginning balance | 60,506 | 41,771 |
Provision | 50,361 | 33,221 |
Payments/credits | (43,293) | (26,992) |
Ending balance | 67,574 | 48,000 |
Prompt Pay [Member] | ||
Accounts Notes And Loans Receivable [Line Items] | ||
Beginning balance | 2,798 | 1,949 |
Provision | 6,192 | 4,005 |
Payments/credits | (5,639) | (3,533) |
Ending balance | 3,351 | 2,421 |
Other Accruals [Member] | ||
Accounts Notes And Loans Receivable [Line Items] | ||
Beginning balance | 6,363 | 4,969 |
Provision | 19,416 | 6,848 |
Payments/credits | (11,460) | (7,236) |
Ending balance | $ 14,319 | $ 4,581 |
Summary of Total Reserves Inclu
Summary of Total Reserves Included in Consolidated Balance Sheets (Detail) - USD ($) $ in Thousands | Jun. 30, 2022 | Dec. 31, 2021 | Jun. 30, 2021 | Dec. 31, 2020 |
Receivables, Net, Current [Abstract] | ||||
Reduction to accounts receivable | $ 17,092 | $ 8,321 | ||
Component of accrued expenses | 68,361 | 62,145 | ||
Total reserves | $ 85,453 | $ 70,466 | $ 56,061 | $ 50,970 |
INVENTORY - Summary of Componen
INVENTORY - Summary of Components of Inventory (Detail) - USD ($) $ in Thousands | Jun. 30, 2022 | Dec. 31, 2021 |
Inventory Disclosure [Abstract] | ||
Raw materials | $ 42,398 | $ 58,822 |
Work in progress | 253,200 | 230,194 |
Finished goods | 35,499 | 26,717 |
Total inventory | $ 331,097 | $ 315,733 |
INVENTORY - Summarizes The Bala
INVENTORY - Summarizes The Balance Sheet Classification (Details) - USD ($) $ in Thousands | Jun. 30, 2022 | Dec. 31, 2021 |
Inventory Disclosure [Abstract] | ||
Inventory | $ 208,095 | $ 186,212 |
Other non-current assets | 123,002 | 129,521 |
Total inventory | $ 331,097 | $ 315,733 |
OTHER ASSETS - Summary of Other
OTHER ASSETS - Summary of Other Current Assets (Detail) - USD ($) $ in Thousands | Jun. 30, 2022 | Dec. 31, 2021 |
Prepaid Expense and Other Assets, Current [Abstract] | ||
Manufacturing-related deposits and prepaids | $ 69,502 | $ 93,656 |
Collaboration receivable | 26,421 | 18,647 |
Prepaid clinical and pre-clinical expenses | 10,707 | 12,667 |
Prepaid maintenance services | 7,644 | 8,452 |
Prepaid insurance | 3,280 | 5,282 |
Prepaid research expenses | 1,973 | 3,082 |
Other | 9,805 | 7,242 |
Total other current assets | $ 129,332 | $ 149,028 |
OTHER ASSETS - Summary of Oth_2
OTHER ASSETS - Summary of Other Current Assets (Parenthetical) (Details) - USD ($) $ in Thousands | Jun. 30, 2022 | Dec. 31, 2021 |
Related Party Transaction [Line Items] | ||
Manufacturing-related deposits and prepaids | $ 69,502 | $ 93,656 |
OTHER ASSETS - Summary of Oth_3
OTHER ASSETS - Summary of Other Non-current Assets (Detail) - USD ($) $ in Thousands | Jun. 30, 2022 | Dec. 31, 2021 |
Prepaid Expense and Other Assets, Noncurrent [Abstract] | ||
Non-current inventory | $ 123,002 | $ 129,521 |
Manufacturing-related deposits and prepaids | 103,819 | 112,765 |
Strategic investments | 33,234 | 34,892 |
Restricted cash and investments | 18,499 | 9,904 |
Prepaid clinical expenses | 2,103 | 2,007 |
Other | 3,543 | 3,860 |
Total other non-current assets | $ 284,200 | $ 292,949 |
ACCRUED EXPENSES - Summary of A
ACCRUED EXPENSES - Summary of Accrued Expenses (Detail) - USD ($) $ in Thousands | Jun. 30, 2022 | Dec. 31, 2021 |
Payables and Accruals [Abstract] | ||
Accrued contract manufacturing costs | $ 204,867 | $ 104,311 |
Product revenue related reserves | 68,361 | 62,145 |
Accrued clinical and pre-clinical costs | 33,832 | 25,955 |
Accrued employee compensation costs | 32,191 | 48,299 |
Accrued royalties | 14,746 | 11,965 |
Accrued professional fees | 13,574 | 9,381 |
Accrued interest expense | 1,092 | 1,045 |
Accrued income taxes | 2,485 | 216 |
Accrued collaboration cost sharing | 2,868 | 2,887 |
Accrued sponsored research agreements | 1,651 | 3,377 |
Accrued fixed assets | 1,462 | 127 |
Other | 6,570 | 1,989 |
Total accrued expenses | $ 383,699 | $ 271,697 |
Indebtedness - Additional Infor
Indebtedness - Additional Information (Detail) $ in Millions | Nov. 14, 2017 | Jun. 30, 2022 USD ($) | Dec. 31, 2021 USD ($) |
Debt Instrument [Line Items] | |||
Debt instrument, conversion rate | 0.013621 | ||
2024 Convertible Notes [Member] | |||
Debt Instrument [Line Items] | |||
Debt Instrument Fair Value | $ 726.2 | $ 846.1 |
Indebtedness - Summary of Debt
Indebtedness - Summary of Debt Facilities (Detail) - USD ($) $ in Thousands | Jun. 30, 2022 | Dec. 31, 2021 |
Debt Instrument [Line Items] | ||
Total carrying value of debt facilities | $ 1,100,873 | $ 1,096,876 |
2024 Convertible Notes [Member] | ||
Debt Instrument [Line Items] | ||
Net carrying value | 564,700 | 563,700 |
2019 Term Loan [Member] | ||
Debt Instrument [Line Items] | ||
Net carrying value | $ 536,100 | $ 533,200 |
Summary of Stock Awards Granted
Summary of Stock Awards Granted (Detail) - $ / shares | 3 Months Ended | 6 Months Ended | |||
Jun. 30, 2022 | Jun. 30, 2021 | Jun. 30, 2022 | Jun. 30, 2021 | ||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||
Grants, Stock options | 207,583 | 72,110 | 1,499,120 | 1,431,747 | |
Weighted Average Grant Date Fair Value, Stock options | $ 43.40 | $ 39.79 | $ 46.49 | $ 48.87 | |
Grants, Restricted stock units and awards | [1] | 147,075 | 37,240 | 859,510 | 726,607 |
Weighted Average Grant Date Fair Value, Restricted stock units and awards | [1] | $ 72.99 | $ 75.30 | $ 78.78 | $ 86.53 |
Restricted Stock Units [Member] | |||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||
Grants, Stock options | 38,500 | ||||
[1] Included in 2022 RSUs are 38,500 shares with performance conditions which are related to regulatory approval of certain of the Company's product candidates. As of June 30, 2022, none of the performance conditions are probable of being achieved. If the performance milestones are achieved within the required time frame, the Company may recognize up to $ 3.1 million of stock-based compensation related to these grants. 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. Additionally, the Chief Executive Officer is subject to a one-year post-exercise restriction to sell, transfer or dispose shares acquired upon the exercise of any options that vest after deduction of any shares withheld or sold to pay the applicable aggregate exercise price and/or withholding taxes. To determine the incremental compensation cost of the modification, the fair value of the modified awards was compared to the fair value of the original awards measured immediately before its terms or conditions were modified. As the Vested Tranche became immediately vested on the Effective Date, the Vested Tranche does not have service or market conditions. As such, the post-modification fair value for the Vested Tranche is based on the Black-Scholes-Merton option-pricing model, while the pre-modification fair value is based on a lattice model with Monte Carlo simulations. The incremental compensation cost of $ 43.9 million was immediately recognized as stock-based compensation expense in the three months ended June 30, 2022. 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. As a result of the modification, the Company is expected to recognize incremental compensation cost of $ 82.1 million associated with the Unvested Tranche. The incremental 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 approximately 1.3 years. For the three months ended June 30, 2022, the Company recorded $ 29.8 million of stock-based compensation expense related to the Unvested Tranche. |
Summary of Stock Awards Grant_2
Summary of Stock Awards Granted (Parenthetical) (Details) - USD ($) $ in Millions | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2022 | Jun. 30, 2021 | Jun. 30, 2022 | Jun. 30, 2021 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Grants, Stock options | 207,583 | 72,110 | 1,499,120 | 1,431,747 |
Stock-based compensation Grants | $ 3.1 | |||
Stock Options [Member] | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Share based compensation granted under plan vest period | 4 years | |||
Restricted Stock Units [Member] | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Grants, Stock options | 38,500 | |||
Share based compensation granted under plan vest period | 4 years |
Stock Based Compensation - Addi
Stock Based Compensation - Additional Information (Detail) - USD ($) $ in Thousands | 1 Months Ended | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2017 | Jun. 30, 2022 | Jun. 30, 2021 | Jun. 30, 2022 | Jun. 30, 2021 | |
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items] | |||||
Stock-based compensation expense | $ 102,892 | $ 28,969 | $ 132,090 | $ 57,477 | |
Grants, Stock options | 207,583 | 72,110 | 1,499,120 | 1,431,747 | |
Incremental compensation cost | $ 43,900 | $ 82,100 | |||
Chief Executive Officer | |||||
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items] | |||||
Grants, Stock options | 3,300,000 | ||||
Unvested Tranche Member | |||||
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items] | |||||
Stock-based compensation expense | $ 29,800 |
Summary of Stock-Based Compensa
Summary of Stock-Based Compensation Expense by Function Included within Condensed Consolidated Statements of Operations and Comprehensive Loss (Detail) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2022 | Jun. 30, 2021 | Jun. 30, 2022 | Jun. 30, 2021 | |
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | ||||
Total stock-based compensation expense | $ 102,892 | $ 28,969 | $ 132,090 | $ 57,477 |
Research and Development [Member] | ||||
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | ||||
Total stock-based compensation expense | 14,467 | 12,860 | 27,535 | 23,986 |
Selling, General and Administrative [Member] | ||||
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | ||||
Total stock-based compensation expense | $ 88,425 | $ 16,109 | $ 104,555 | $ 33,491 |
Summary of Stock-Based Compen_2
Summary of Stock-Based Compensation Expense by Grant Type Included within Condensed Consolidated Statements of Operations and Comprehensive Loss (Detail) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2022 | Jun. 30, 2021 | Jun. 30, 2022 | Jun. 30, 2021 | |
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | ||||
Total stock-based compensation expense | $ 102,892 | $ 28,969 | $ 132,090 | $ 57,477 |
Stock Options [Member] | ||||
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | ||||
Total stock-based compensation expense | 89,155 | 17,587 | 105,561 | 34,606 |
Restricted Stock Awards/Units (RSAs/RSUs) [Member] | ||||
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | ||||
Total stock-based compensation expense | 12,378 | 10,573 | 23,743 | 20,504 |
Employee Stock Purchase Plan [Member] | ||||
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | ||||
Total stock-based compensation expense | $ 1,359 | $ 809 | $ 2,786 | $ 2,367 |
OTHER (LOSS) INCOME, NET - Summ
OTHER (LOSS) INCOME, NET - Summary of Other Loss (Detail) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2022 | Jun. 30, 2021 | Jun. 30, 2022 | Jun. 30, 2021 | |
Nonoperating Income (Expense) [Abstract] | ||||
Interest expense | $ (16,028) | $ (15,831) | $ (31,824) | $ (31,482) |
Interest income | 2,409 | 52 | 2,582 | 111 |
Gain from sale of Priority Review Voucher | 0 | 102,000 | 0 | 102,000 |
Other expense (income), net | (3,342) | (406) | (4,984) | (342) |
Total other (loss) income, net | $ (16,961) | $ 85,815 | $ (34,226) | $ 70,287 |
OTHER (LOSS) INCOME, NET (Addit
OTHER (LOSS) INCOME, NET (Additional Information) (Details) $ in Millions | 1 Months Ended |
Apr. 30, 2021 USD ($) | |
F D A | |
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |
Proceeds from sale of rare pediatric disease priority review voucher, Net of commission | $ 102 |
LEASES (Additional Information)
LEASES (Additional Information) (Details) $ in Millions | 6 Months Ended | ||||
May 31, 2022 USD ($) | Apr. 22, 2022 USD ($) ft² | Jun. 30, 2022 USD ($) | Jun. 01, 2022 USD ($) ft² | Dec. 22, 2018 ft² | |
Bedford Massachusetts [Member] | |||||
Lessee, Lease, Description [Line Items] | |||||
Area of lease | ft² | 288,000 | ||||
Initial lease term of contract | 15 years | 15 years | |||
Leases, term of contract extend | 10 years | ||||
Letter of Credit Reduce | $ 5.6 | ||||
Collateralized Letter of Credit Cash Deposit | $ 8.4 | ||||
Undiscounted rent payment | $ 307.4 | ||||
Tenant improvement allowance | $ 72 | ||||
Columbus Ohio [Member] | |||||
Lessee, Lease, Description [Line Items] | |||||
Area of lease | ft² | 18,000 | ||||
Initial lease term of contract | 15 years | ||||
Leases, term of contract extend | 5 years | ||||
Undiscounted rent payment | $ 38.9 | ||||
Right of use assets and lease liability | $ 7.3 | ||||
Columbus Ohio [Member] | Minimum [Member] | |||||
Lessee, Lease, Description [Line Items] | |||||
Area of lease | ft² | 78,000 | ||||
Columbus Ohio [Member] | Maximum [Member] | |||||
Lessee, Lease, Description [Line Items] | |||||
Area of lease | ft² | 167,000 |
Basic and Diluted Net Loss Per
Basic and Diluted Net Loss Per Share (Detail) - USD ($) $ / shares in Units, $ in Thousands | 3 Months Ended | 6 Months Ended | |||||
Jun. 30, 2022 | Mar. 31, 2022 | Jun. 30, 2021 | Mar. 31, 2021 | Jun. 30, 2022 | Jun. 30, 2021 | ||
Earnings Per Share [Abstract] | |||||||
Net loss | $ (231,481) | $ (105,025) | $ (81,405) | $ (167,250) | $ (336,506) | $ (248,655) | |
Weighted-average common shares outstanding - basic | 87,511 | 79,746 | 87,383 | 79,601 | |||
Effect of dilutive securities* | [1] | 0 | 0 | 0 | 0 | ||
Weighted-average common shares outstanding - diluted | 87,511 | 79,746 | 87,383 | 79,601 | |||
Net loss per share - basic and diluted | $ (2.65) | $ (1.02) | $ (3.85) | $ (3.12) | |||
Net loss per share - basic and diluted | $ (2.65) | $ (1.02) | $ (3.85) | $ (3.12) | |||
[1] For the three and six months ended June 30, 2022 and 2021, stock options, RSAs, RSUs, and ESPP to purchase 11.3 million and 9.8 million shares of the Company’s common stock, respectively, were excluded from the diluted net loss per share calculation as their effect would have been anti-dilutive. The Company accounts for the effect of its 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 more dilutive. During the three and six months ended June 30, 2022 and June 30, 2021 , respectively, the inclusion of the potential share settlement of the 2024 Notes was anti-dilutive. Accordingly, the potential conversion of 7,763,552 shares has been excluded from the computation of diluted net loss per share as of June 30, 2022 and June 30, 2021 , respectively. |
Basic and Diluted Net Loss Pe_2
Basic and Diluted Net Loss Per Share (Parenthetical) (Detail) - shares | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2022 | Jun. 30, 2021 | Jun. 30, 2022 | Jun. 30, 2021 | |
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||||
Anti-dilutive securities excluded from computation of net loss per share | 11,300,000 | 9,800,000 | 11,300,000 | 9,800,000 |
2024 Convertible Notes [Member] | ||||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||||
Conversion of shares excluded from computation of diluted EPS | 7,763,552 | 7,763,552 |
COMMITMENTS AND CONTINGENCIES -
COMMITMENTS AND CONTINGENCIES - Summary of Aggregate Non-Cancelable Contractual Obligations Arising from Manufacturing Obligations (Detail) - Manufacturing Commitments Agreement [Member] - Catalent and Thermo [Member] $ in Thousands | Jun. 30, 2022 USD ($) | |
Commitments And Contingencies [Line Items] | ||
2022 (April - December) | $ 529,689 | |
2023 | 321,520 | |
2024 | 135,285 | |
2025 | 98,798 | |
2026 | 54,720 | |
Thereafter | 109,440 | |
Total manufacturing commitments | $ 1,249,452 | [1] |
[1] Total manufacturing commitments includes the Catalent Inc. manufacturing and supply agreement, for which the Company has right of use assets and lease liabilities recorded on the unaudited condensed consolidated balance sheets as of June 30, 2022. For more information, please read Note 21, Commitments and Contingencies of the Company’s Annual Report on Form 10-K for the year ended December 31, 2021. |
COMMITMENTS AND CONTINGENCIES_2
COMMITMENTS AND CONTINGENCIES (Additional Information) (Details) $ in Millions | 6 Months Ended |
Jun. 30, 2022 USD ($) | |
Thermo Fisher Scientific Inc [Member] | |
Commitments And Contingencies [Line Items] | |
Research and Development Expense | $ 53 |